美國
證券交易委員會
華盛頓特區20549
形式
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1934年證券交易法 |
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截至季度 |
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或 |
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1934年證券交易法 |
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對於到的過渡期 |
委員會檔案編號
TG治療, Inc.
(章程中規定的註冊人的確切名稱)
| |
(成立或組織的州或其他司法管轄區) | (國稅局僱主識別號) |
(地址包括主要行政辦公室的郵政編碼)
(
(註冊人電話號碼,包括地區代碼)
根據該法第12(b)條登記的證券:
班級名稱 | 交易符號 | 交易所名稱 |
| | |
通過勾選標記確定註冊人是否:(1)在過去12個月內(或在註冊人被要求提交此類報告的較短期限內)提交了1934年證券交易法第13或15(d)條要求提交的所有報告,以及(2)在過去90天內是否遵守此類提交要求。
通過勾選標記檢查註冊人是否已在過去12個月內(或在註冊人被要求提交此類文件的較短期限內)以電子方式提交了根據S-t法規第405條(本章第232.405條)要求提交的所有交互數據文件。
☒
通過複選標記來確定註冊人是大型加速申報人、加速申報人、非加速申報人、小型報告公司還是新興成長型公司。請參閱《交易法》第120條第2條中「大型加速申報人」、「加速申報人」、「小型報告公司」和「新興成長型公司」的定義。
| 加速文件收件箱 |
非加速文件收件箱 | 小型上市公司 |
新興成長型公司 |
如果是新興成長型公司,請通過勾選標記表明註冊人是否選擇不利用延長的過渡期來遵守根據《交易法》第13(a)條規定的任何新的或修訂的財務會計準則。☐
通過勾選標記確認註冊人是否是空殼公司(定義見《交易法》第120條第2款)。
是的
有
TG治療, Inc.
形式 10-Q
截至2024年9月30日的季度
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本報告中討論的某些事項,包括在“風險因素”和“管理層對財務狀況和經營結果的討論和分析”標題下討論的事項,可能構成前瞻性陳述,就修訂後的19證券法(證券法)和1934年修訂的證券交易法(交易法)而言,涉及已知和未知的風險、不確定因素和其他因素,可能會導致我們的實際結果、業績或成就與這些前瞻性陳述明示或暗示的未來結果、業績或成就大不相同。在某些情況下,您可以通過“預期”、“相信”、“考慮”、“繼續”、“可能”、“估計”、“預期”、“打算”、“可能”、“計劃”、“潛在”、“預測”、“專案”、“尋求”、“應該”、“目標”、“將”、“將”或這些詞語的否定或其他類似術語來識別前瞻性陳述,儘管並不是所有的前瞻性陳述都包含這些識別詞語。
所有歸因於我們的書面或口頭前瞻性陳述均由這些警示性陳述明確限定。此外,對於我們的所有前瞻性陳述,我們主張1995年《私人證券訴訟改革法案》所載前瞻性陳述的安全港保護。此類前瞻性陳述包括但不限於以下陳述:
● |
我們有能力為我們的候選產品獲得並保持監管部門的批准,包括AZER-CEL,以及我們有能力在美國(美國)、歐盟(EU)和英國(英國)保持對BRIUMVI®單抗(Ublituximab)用於治療復發性多發性硬化症(RMS)或任何其他未來適應症的監管批准; |
● |
我們有能力調整和擴展我們的商業基礎設施,以成功地推出、營銷和銷售BRIUMVI和我們的其他候選產品; |
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我們有能力維持可靠的產品供應,以滿足市場需求; |
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BRIUMVI或任何未來產品或產品組合正在進行的商業化成功,包括預期的市場接受率和程度以及定價和補償; |
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我們臨床前研究和臨床試驗的啟動、時間、進展和結果; |
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我們推動候選藥物進入並成功完成臨床試驗的能力; |
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我們開發、配製、製造和商業化我們的候選產品的能力; |
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我們有能力在商業上合理的條件下與第三方建立和維持合同關係和夥伴關係,以製造、分銷、營銷和供應以及為我們的臨床開發和商業化努力提供一系列其他支持職能; |
● |
為我們的業務和候選藥物實施我們的商業模式和戰略計劃; |
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我們能夠為涵蓋我們的產品和候選產品的知識產權建立和維護的保護範圍; |
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對我們的費用、未來收入、資本需求和額外融資需求的估計; |
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如果需要,我們保持和建立合作並達成戰略安排的能力; |
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我們滿足任何財務預測或指導的能力,包括但不限於短期和長期收入預測或指導以及對這些預測或指導所依據的假設的變更; |
● |
我們有能力獲得足夠的資本來資助我們的計劃運營; |
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我們的財務業績和現金消耗管理; |
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我們維持或獲得足夠的產品責任和其他保險的能力; |
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與我們的競爭對手和我們的行業相關的發展; |
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未來監管發展或立法行動對我們公司的影響,包括我們所遵守的醫療保健、環境和其他法律法規的變化; |
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當前的經濟、市場和商業狀況; |
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我們保留、吸引和雇用關鍵人員的能力; |
● | 我們的競爭地位; |
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我們普通股交易價格的波動; |
● | 我們使用現金和其他資源;以及 |
● | 我們成功實施戰略的能力。 |
我們的業務面臨多種風險,您在做出投資決定之前應該了解這些風險。下文描述的風險是與投資我們相關的主要風險的總結,並且不是我們面臨的唯一風險。您應仔細考慮這些風險、第1A項中的風險因素以及我們向美國證券交易委員會(SEC)提交的其他報告和文件中描述的任何其他風險。
與商業化相關的風險
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如果我們無法維持目前對BRIUMVI的批准,我們的業務將受到實質性損害。 |
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我們無法預測何時或是否會獲得監管部門的批准,將我們的候選產品商業化,包括非腫瘤學適應症的Azer-cel。 |
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作為一家商業公司,我們的運營經驗有限,因此,用於治療RMS的BRIUMVI的營銷和銷售可能不如預期的成功。 |
● |
如果BRIUMVI或我們未來的任何候選產品(如果獲得批准)不能在醫生、患者、付款人或醫學界獲得廣泛的市場接受,我們從產品銷售中產生的收入將是有限的。 |
● |
如果BRIUMVI和我們可能獲得批准的任何未來產品(包括非腫瘤適應症的Azer-cel)的市場機會比我們估計的要小,或者如果我們獲得的任何批准是基於較窄的患者群體,或者標籤包括患者或醫療保健提供者無法接受的警告或限制,我們的收入將受到不利影響。 |
● |
我們將面臨針對我們目標適應症的治療的激烈競爭,包括來自擁有比我們更多資源的公司的競爭,這可能會導致其他人比我們更早或更成功地將藥物商業化,這可能會減少或消除我們的商業機會。 |
● |
如果我們無法產生足夠的收入,我們可能需要籌集大量額外資本來維持我們的業務。 |
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產品責任訴訟可能會導致我們招致重大責任,並限制產品商業化。 |
與藥物開發和監管批准相關的風險
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如果我們無法繼續維護或獲得監管部門對我們的產品或候選產品的批准,最終無法將我們的產品或候選產品商業化,或者如果我們在這樣做的過程中遇到重大延誤,我們的業務將受到實質性損害。 |
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我們的新產品和候選產品可能會造成不良的副作用,可能會推遲或阻止他們的監管批准,或者在營銷批准(如果有)後顯著限制他們的商業形象,或者如果獲得批准,可能會導致退出市場。 |
● |
因為臨床前研究和早期臨床試驗的結果不一定能預測未來的結果,所以我們開發的任何候選產品在以後的臨床試驗中都可能不會有有利的結果。此外,隨著分析更多的患者數據或更多的終端,我們宣佈或公佈的臨床試驗的臨時、最重要的資訊和初步數據可能會發生變化,或者可能會影響感知的產品概況。 |
● |
我們可能通過臨床開發推進的任何產品或候選產品都受到廣泛的監管,這可能是昂貴和耗時的,可能會導致意想不到的延誤,或者阻止收到所需的批准。 |
與政府監管制藥行業相關的風險
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我們受到廣泛的監管,包括新的立法和監管提案,包括控制、設定或限制已批准藥物的定價的努力,這可能會增加我們的成本並對我們營銷產品、獲得合作者和籌集資本的能力產生不利影響。 |
● |
如果我們未能遵守各種醫療保健法律和法規,我們可能會遭受損失或承擔責任。 |
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如果我們未能遵守監管要求,任何候選產品都可能無法獲得監管批准,我們獲得上市批准的任何產品都可能受到限制或退出市場,我們可能會受到處罰。 |
與我們對第三方的依賴相關的風險
● |
我們依賴第三方進行原材料、產品和候選產品的商業和臨床供應增加了我們無法以可接受的成本或質量獲得足夠數量的產品或候選產品或此類數量的風險,這可能會延遲、阻止或損害我們的開發或商業化工作。 |
● |
如果我們依賴進行臨床試驗並生成支持我們的監管申請所需的臨床、臨床前和其他數據的第三方沒有按要求提供服務,那麼我們可能無法在預期或根本無法獲得監管批准或商業化我們的產品或候選產品。 |
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由於我們已從第三方獲得了我們的產品和候選產品的許可,因此與我們的許可方的任何爭議或不履行義務將對我們開發和商業化適用產品的能力產生不利影響。 |
智慧財產權相關風險
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我們的成功取決於我們獲得和保護我們知識產權的能力,如果我們獲得的專利保護範圍不夠廣泛,我們的競爭對手可能會開發和商業化與我們類似或相同的產品,我們成功將我們的產品商業化的能力可能會受到損害。 |
● |
如果不遵守政府專利機構施加的各種程序和其他要求,我們的專利保護可能會減少或取消。 |
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我們可能需要從第三方獲得某些知識產權的許可,而此類許可可能無法獲得,也可能無法以商業合理的條款獲得。 |
● |
如果我們或我們的合作伙伴被起訴侵犯第三方的知識產權,對抗此類訴訟將是昂貴和耗時的,任何此類訴訟的不利結果都將對我們的業務產生實質性的不利影響。 |
● |
如果我們無法保護我們的商業祕密的機密性,我們的業務可能會受到重大損害。 |
與我們的財務狀況和額外資本需求相關的風險
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雖然我們最近實現了淨收入,但自成立以來我們已經出現了重大運營虧損,並且未來可能會出現虧損。 |
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雖然我們預計不需要籌集額外資本,但我們可能需要這樣做。如果我們無法籌集資金,如果需要,我們可能會被要求推遲、限制、減少或取消我們的一些藥物開發計劃或商業化工作。 |
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我們的負債水平和償債義務可能會對我們的財務狀況產生不利影響,並可能使我們更難爲我們的運營提供資金。 |
一般風險因素
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患者和醫療保健提供者擔心,免疫抑制產品,如抗CD20抗體和其他b細胞靶向藥物,可能會增加感染病毒的風險,或在感染後導致更嚴重的併發症。這些擔憂可能會影響BRIUMVI和我們正在開發的其他免疫抑制產品的商業潛力。 |
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我們將需要發展和擴大我們的業務,我們在管理這種發展和擴大時可能會遇到困難。 |
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我們繼續臨床開發和商業化活動的能力將取決於我們吸引和保持關鍵管理層和其他人員的能力。 |
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我們的某些高管、董事和其他股東擁有我們已發行普通股的5%以上,並可能能夠影響我們的管理層和提交給股東批准的事項的結果。 |
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我們的憲章文件和特拉華州法律中的某些反收購條款可能會使第三方收購變得更加困難,這可能會限制投資者可能願意爲我們的普通股支付的價格。 |
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我們目前的股價是波動的,我們預計它將繼續波動,這可能會限制投資者出售股票獲利的能力,並可能使我們面臨證券和股東衍生品訴訟。 |
● |
信息技術系統的重大網絡中斷、數據安全漏洞或未經授權披露敏感數據可能會損害我們的業務,並使我們承擔責任或聲譽損害。 |
上述僅是我們部分風險的總結。這些和其他風險在第二部分第1A項題爲「風險因素」的部分以及本季度報告10-Q表格(我們的風險因素)的其他地方進行了更全面的討論。
簡明綜合資產負債表
(單位爲千,不包括每股和每股金額)
| 9月30日, | 十二月三十一日, | ||||||
| 2024 | 2023 | ||||||
| (未經審計) | (注1) | ||||||
資產 | | |||||||
流動資產: | | | ||||||
現金及現金等價物 | $ | $ | ||||||
短期投資證券 | ||||||||
應收賬款淨額 | ||||||||
庫存 | ||||||||
其他流動資產 | ||||||||
流動資產總額 | ||||||||
受限現金 | ||||||||
長期投資證券 | ||||||||
使用權資產 | ||||||||
其他非流動資產(1) | ||||||||
總資產 | $ | $ | ||||||
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負債和股東權益 | ||||||||
流動負債: | ||||||||
應付賬款和應計費用 | $ | $ | ||||||
其他流動負債 | ||||||||
租賃負債-流動部分 | ||||||||
遞延收入--當期部分 | ||||||||
應計補償 | ||||||||
流動負債總額 | ||||||||
遞延收入,非當期部分 | ||||||||
應付貸款-非流動 | ||||||||
租賃負債-非流動 | ||||||||
總負債 | ||||||||
承付款和或有事項 | ||||||||
股東權益: | ||||||||
普通股,$ 每股面值( 和 授權股份, 和 發行的股票, 和 分別於2024年9月30日和2023年12月31日發行在外的股份) | ||||||||
額外實收資本 | ||||||||
國庫股,按成本價計算, 和 分別於2024年9月30日和2023年12月31日的股票 | ( | ) | ( | ) | ||||
累計赤字 | ( | ) | ( | ) | ||||
股東權益總額 | ||||||||
總負債和股東權益 | $ | $ |
附註是簡明綜合財務報表的組成部分。
(1)截至2023年12月31日的金額已重新分類,以符合本期列報方式。
簡明綜合業務報表
(單位爲千,不包括每股和每股金額)
(未經審計)
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截至三個月 |
九個月結束 |
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9月30日, |
9月30日, |
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2024 |
2023 |
2024 |
2023 |
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收入: |
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產品收入,淨額 |
$ | $ | $ | $ | ||||||||||||
許可證、里程碑、版稅和其他收入 |
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總收入 |
$ | $ | $ | $ | ||||||||||||
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成本和支出: |
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收入成本 |
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研究和開發: |
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非現金補償 |
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其他研究和開發 |
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總研發 |
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銷售、總務和行政: |
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非現金補償 |
$ | |||||||||||||||
其他銷售,一般和行政 |
$ | |||||||||||||||
銷售總額,一般和行政 |
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總成本和費用 |
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營業收入 |
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其他費用(收入): |
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利息開支 |
$ | |||||||||||||||
其他收入 |
( |
) | ( |
) | $ | ( |
) | ( |
) | |||||||
其他費用合計(淨額) |
||||||||||||||||
|
|
|
||||||||||||||
稅前淨收益 |
$ | $ | $ | $ | ||||||||||||
所得稅費用 |
$ | $ | ||||||||||||||
淨收入 |
$ | $ | $ | $ | ||||||||||||
|
||||||||||||||||
每股普通股淨收入: |
||||||||||||||||
基本信息 |
$ | $ | $ | $ | ||||||||||||
稀釋 |
$ | $ | $ | $ | ||||||||||||
加權平均普通股流通股 |
||||||||||||||||
基本信息 |
||||||||||||||||
稀釋 |
附註是簡明綜合財務報表的組成部分。
簡明合併股東權益變動表
(單位爲千,不包括每股和每股金額)
(未經審計)
其他內容 | ||||||||||||||||||||||||||||
普通股 | 已繳費 | 庫存股 | 累計 | |||||||||||||||||||||||||
| 股份 | 量 | 資本 | 股份 | 量 | 赤字 | 總 | |||||||||||||||||||||
2023年1月1日的餘額 | $ | $ | $ | ( | ) | $ | ( | ) | $ | |||||||||||||||||||
與期權行使相關的普通股發行 | * | |||||||||||||||||||||||||||
發行限制性股票 | ( | ) | ||||||||||||||||||||||||||
債務融資發行的憑證 | — | — | ||||||||||||||||||||||||||
沒收限制性股票 | ( | ) | * | — | ||||||||||||||||||||||||
授予員工、董事和顧問的限制性股票的補償 | — | — | ||||||||||||||||||||||||||
淨虧損 | — | — | ( | ) | ( | ) | ||||||||||||||||||||||
2023年3月31日的餘額 | ( | ) | ( | ) | ||||||||||||||||||||||||
與期權行使相關的普通股發行 | * | |||||||||||||||||||||||||||
發行限制性股票 | * | * | ||||||||||||||||||||||||||
沒收限制性股票 | ( | ) | * | * | ||||||||||||||||||||||||
公開發行普通股(扣除發行成本美元 百萬美元) | ||||||||||||||||||||||||||||
授予員工、董事和顧問的限制性股票的補償 | — | — | ||||||||||||||||||||||||||
淨虧損 | — | — | ( | ) | ( | ) | ||||||||||||||||||||||
2023年6月30日的餘額 | ( | ) | ( | ) | ||||||||||||||||||||||||
與期權行使相關的普通股發行 | * | |||||||||||||||||||||||||||
限制性股票的發行 | * | * | ||||||||||||||||||||||||||
限制性股票的沒收 | ( | ) | * | * | ||||||||||||||||||||||||
授予員工、董事和顧問的限制性股票的補償 | — | — | ||||||||||||||||||||||||||
淨虧損 | — | — | ||||||||||||||||||||||||||
2023年9月30日的餘額 | ( | ) | ( | ) |
其他內容 |
||||||||||||||||||||||||||||
普通股 |
已繳費 |
庫存股 |
累計 |
|||||||||||||||||||||||||
|
股份 |
量 |
資本 |
股份 |
量 |
赤字 |
總 |
|||||||||||||||||||||
2024年1月1日餘額 |
$ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||||||
與期權行使相關的普通股發行 |
* | |||||||||||||||||||||||||||
發行限制性股票 |
( |
) | ||||||||||||||||||||||||||
沒收限制性股票 |
( |
) | * | — | ||||||||||||||||||||||||
授予員工、董事和顧問的限制性股票的補償 |
— | — | ||||||||||||||||||||||||||
淨虧損 |
— | — | ( |
) | ( |
) | ||||||||||||||||||||||
2024年3月31日的餘額 |
|
|
( |
) | ( |
) | |
|||||||||||||||||||||
與期權行使相關的普通股發行 |
* | |||||||||||||||||||||||||||
發行限制性股票 |
* | — | ||||||||||||||||||||||||||
沒收限制性股票 |
( |
) | * | — | ||||||||||||||||||||||||
授予員工、董事和顧問的限制性股票的補償 |
— | — | ||||||||||||||||||||||||||
淨收入 |
— | — | ||||||||||||||||||||||||||
2024年6月30日的餘額 |
( |
) | ( |
) | ||||||||||||||||||||||||
與期權行使相關的普通股發行 |
* | |||||||||||||||||||||||||||
限制性股票的發行 |
( |
) | ||||||||||||||||||||||||||
限制性股票的沒收 |
( |
) | * | * | ||||||||||||||||||||||||
普通股回購 |
— | ( |
) | ( |
) | |||||||||||||||||||||||
授予員工、董事和顧問的限制性股票的補償 |
— | — | ||||||||||||||||||||||||||
淨收入 |
— | — | ||||||||||||||||||||||||||
2024年9月30日餘額 |
( |
) | ( |
) |
* 金額低於一千美元
隨附的附註是簡明綜合財務報表的組成部分。
現金流量表簡明合併報表
(單位:千)
(未經審計)
|
止九個月 |
|||||||
|
9月30日, |
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|
2024 |
2023 |
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經營活動產生的現金流量 |
|
|
||||||
|
|
|
||||||
淨收入 |
$ | $ | ||||||
將淨虧損與經營活動中使用的淨現金進行調節的調整: |
||||||||
債務消滅損失 |
||||||||
非現金股票補償費用 |
||||||||
折舊及攤銷 |
||||||||
投資證券溢價(折扣)攤銷 |
( |
) | ( |
) | ||||
債務發行成本攤銷 |
||||||||
租賃權益攤銷 |
||||||||
租賃負債和使用權資產的非現金變化 |
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股權投資公允價值變化 |
||||||||
應付票據公允價值變化 |
( |
) | ||||||
資產和負債變化: |
||||||||
庫存增加 |
( |
) | ( |
) | ||||
其他易變現資產增加 |
( |
) | ( |
) | ||||
應收賬款增加 |
( |
) | ( |
) | ||||
應付賬款和應計費用增加(減少) |
( |
) | ||||||
租賃負債減少 |
( |
) | ( |
) | ||||
其他流動負債增加 |
||||||||
遞延收入增加 |
||||||||
經營活動所用現金淨額 |
( |
) | ( |
) | ||||
|
||||||||
投資活動產生的現金流量 |
||||||||
短期證券到期收益 |
||||||||
持有至到期證券的投資 |
( |
) | ( |
) | ||||
長期證券投資 |
( |
) | ||||||
購買財產、廠房和設備 |
( |
) | ||||||
投資活動所用現金淨額 |
( |
) | ( |
) | ||||
|
||||||||
融資活動現金流量 |
||||||||
應付貸款的支付 |
( |
) | ||||||
普通股發行,淨值 |
||||||||
行使期權收益 |
||||||||
債務融資收益 |
||||||||
支付的融資費用 |
( |
) | ( |
) | ||||
購買庫藏股 |
( |
) | ||||||
融資活動提供的淨現金 |
||||||||
|
||||||||
現金、現金等值和限制現金淨增加 |
||||||||
|
|
|||||||
現金、現金等值和期限開始時的限制現金 |
||||||||
|
||||||||
期末現金、現金等值和限制現金 |
$ | $ | ||||||
|
||||||||
|
||||||||
與簡明綜合資產負債表金額的對賬: |
||||||||
現金及現金等價物 |
$ | $ | ||||||
受限制現金 |
||||||||
現金、現金等值物和限制現金總額 |
$ | $ | ||||||
|
||||||||
已付現金: |
||||||||
利息 |
$ | $ | ||||||
非現金交易 |
||||||||
遞延融資成本 |
$ | $ | ||||||
債務融資發行的憑證 |
$ | $ | ||||||
隨附的附註是簡明綜合財務報表的組成部分。
TG治療, Inc.
簡明合併財務報表附註(未經審計)
除非上下文另有要求,否則本報告中提及 “TG,” “這個 公司,” “我們,” “我們”和“我們” 請參閱TG Therapeutics, Inc.及其子公司。
業務描述
TG Therapeutics是一家完全整合的商業階段生物製藥公司,專注於b細胞疾病新型治療方法的收購、開發和商業化。除了包括多種研究藥物在內的研究管道外,TG還獲得了FDA的BRIUMVI(ublituximab-xiiy)的批准,用於治療成人RMS患者,包括臨床孤立綜合徵、復發緩解性疾病和成人活動性次要進展性疾病。TG還獲得了歐盟(EU)歐盟委員會(EC)和英國(UK)藥品和醫療保健產品監管局(MHRA)對BRIUMVI的批准,用於治療患有活動性疾病的成年RMS患者,由臨床或影像學特徵定義。TG繼續積極評估補充產品、技術和公司,以獲得許可、合作伙伴關係、收購和/或投資機會。
.
呈列基準
隨附的未經審計簡明合併財務報表是根據美國公認會計原則(GAAP)、中期財務信息以及季度報告表格的說明編制的 10-Q和文章 10 法規S-X 《交易法》。故該等股份認購 可以 不 包括GAAP完整財務報表所需的所有信息和腳註。管理層認爲屬正常經常性且公平列報簡明綜合財務報表所需的所有調整均已納入。然而,這些簡明綜合財務報表應與我們的年度報告中包含的經審計綜合財務報表一併閱讀 10-k爲年終年度 2023年12月31日.隨附的截至日期的簡明資產負債表 2023年12月31日 都源自於這些陳述。運營結果 三和九 截至 2024年9月30日 是 不 必然表明 可以 預計將在整個財年或任何其他中期期間進行。
流動資金及資本資源
從歷史上看,我們曾遭受過運營虧損;然而,在 三和九 截至 2024年9月30日, 公司產生了淨利潤。此外,在 十二 截至 2023年12月31日, 公司因美元而實現淨利潤
我們的主要現金來源是私募和公開發行股票證券的收益、貸款和擔保協議的收益、商業化協議的預付款(見附註2(了解更多信息),以及BRIUMVI的藥品銷售產品收入。在這段時間裏九 截至 2024年9月30日,我們產生了$
截至 2024年9月30日,我們有美元
我們需要運營的實際現金數量取決於許多因素,包括,但是 不 僅限於我們對BRIUMVI的商業化工作、爲我們其他候選藥物潛在商業化所做的準備、我們候選藥物臨床試驗的時機、設計和進行以及與許可或以其他方式獲取新候選產品相關的成本。我們 可以 依賴於大量的未來融資來提供執行我們當前和未來運營所需的現金,包括我們任何候選藥物的商業化。
我們的普通股在納斯達克資本市場上市,交易代碼爲“TGTX”。
主要會計政策概要
我們的重要會計政策在註釋中描述 1 我們的年度報告中包含的合併財務報表註釋 10-k爲年終年度 2023年12月31日, 除非此處更新或與收入確認、應收賬款、庫存、收入成本、股權證券以及在年內採用新會計準則有關 九 截至 2024年9月30日.管理層並 不 相信任何最近發佈的,但 不 但有效的會計公告,如果目前採用,將對公司的合併財務報表產生重大影響。
財務報表重新分類
過往期間的某些帳戶餘額已在這些財務報表中重新分類,以符合本期分類。
收入確認
根據主題 606, 我們確認收入是爲了描述向客戶轉讓承諾的商品或服務的金額,該金額反映了我們預計有權換取這些商品或服務的對價。爲了實現這一核心原則,主題 606 包括a內的規定 五- 步驟模型,包括i)識別與客戶的合同,ii)識別合同中的履行義務,iii)確定交易價格,iv)將交易價格分配給履行義務,以及v)在實體履行履行義務時確認收入。
在合同開始時,我們評估每份合同中承諾的商品或服務,並評估每個承諾的商品或服務是否獨特,並確定哪些商品或服務是履行義務。然後,當履行履行義務時,我們將分配給各自履行義務的交易價格金額確認爲收入。
產品收入,淨-當客戶控制產品時(通常在交付給客戶時),公司確認產品收入,扣除與某些津貼和應計費用相關的可變對價。產品收入按淨銷售價格或交易價格記錄。公司記錄產品收入儲備(歸類爲產品收入減少),以計入可變對價的組成部分。可變對價包括以下組成部分,具體描述如下:退款、政府回扣、貿易折扣和津貼、產品退貨和自付援助。
這些準備金是基於對相關銷售所賺取或將索賠的金額的估計,並被歸類爲應收賬款的減少(如果該金額預計將從公司的客戶帳戶中貸記)或負債(如果該金額預計將以現金支付)。本公司對可變對價準備的估計是根據預期值法的一致應用而計算的,期望值法是一系列可能的對價金額中的概率加權金額的總和。這些估計反映了公司目前的合同和法律要求、特定的已知市場事件和趨勢、行業數據以及預測的客戶購買和支付模式。交易價格中包含的可變對價金額 可以 受限制,並僅在以下情況下才計入淨產品收入:已確認的累計收入數額的重大逆轉很可能將不發生在未來的一段時間。實際收到的對價金額 可以 最終與公司的估計不同。如果實際結果不同,公司會調整這些估計,這可能會對調整期內的收益產生影響。
退款:折扣退款代表公司因合同承諾向合格的醫療保健提供者和政府機構銷售產品而產生的估計義務,該價格低於向直接從公司購買產品的客戶收取的標價。客戶向公司收取客戶就產品向公司支付的費用與客戶最終合同承諾或政府要求的合格醫療保健提供者的較低售價之間的差額。
政府回扣:政府回扣包括醫療保險、Tricare和醫療補助回扣。這些儲備在確認相關收入的同期記錄。對於醫療保險,該公司還估計了處方藥覆蓋缺口中將根據醫療保險D部分計劃欠回扣的患者數量。
商業付款人回扣:該公司與各種私人付款人組織(主要是保險公司和藥房福利經理)簽訂合同,支付有關使用我們產品和合同處方集狀態的回扣。公司估計這些回扣並在確認相關收入的同期記錄此類估計,導致產品收入減少並建立流動負債。
貿易折扣和津貼:公司向客戶提供折扣,折扣在適用合同中明確規定,並記錄在相關產品收入確認期間。此外,該公司還從客戶那裏獲得銷售訂單管理、庫存管理和數據服務,以換取一定的費用。
產品退貨:根據行業慣例,公司通常爲客戶提供從公司購買的產品的有限退貨權。該公司估計其產品銷售額 可以 由其客戶退回並在相關產品收入確認期間記錄此估計。該公司目前根據類似產品的數據和其他定性考慮(例如分銷渠道中剩餘庫存的可見性)來估計產品退貨負債。
在某些限制的情況下,公司的退貨政策允許在以下情況下對BRIUVI進行符合條件的退貨以獲得積分:
●收到損壞的產品;
●由於公司錯誤而導致的發貨錯誤;
●開始期間退回的過期產品 三 產品到期和結束前的幾個月 六 有效期後數月;
●可能召回的產品;以及
●公司自行決定指定的產品可以退回以獲得積分。
截至 2024年9月30日,公司已
收到了與BRIUMVI銷售相關的任何退貨。
自付援助計劃:向符合條件的擁有商業保險的患者提供自付援助,公司 可以 爲患者提供經濟援助,並支付患者保險提供商要求的處方藥自付費用。自付援助準備金在確認相關收入的同期記錄。
許可協議
該公司通過與製藥公司就某些產品的開發和商業化達成的許可證或類似協議產生收入。此類協定 可以 包括以許可證形式轉讓知識產權。客戶付款 可以 包括不可退還的預付費、基於實現規定里程碑的付款以及產品銷售的特許權使用費。
知識產權許可:如果確定公司知識產權的許可與安排中確定的其他履行義務不同,公司將分配給該許可的交易價格確認爲許可控制權轉讓後的收入。對協議中所有其他承諾的商品或服務進行評估,以確定它們是否獨特。如果他們是 不 不同的是,它們與其他承諾的商品或服務結合起來,創建一套獨特的承諾商品或服務。
里程碑付款:合同開始時的或有里程碑估計金額爲不可能發生材料沖銷,並使用最可能金額法計入交易價格。具有里程碑意義的付款不在公司的控制範圍內,如監管批准,不被認爲在收到這些批准之前有可能實現,因此可變對價受到限制。然後,交易價格按相對獨立的銷售價格分配給每項履約義務,公司在履行合同項下的履約義務時確認收入。在每個報告期結束時,公司重新評估實現以開發或銷售爲基礎的里程碑付款的可能性 可以 不受到重大逆轉的影響,如有必要,調整整體交易價格的估計。任何此類調整均按累積追趕原則入賬,這將影響許可證和其他收入以及調整期間的收益。
基於銷售的版稅:對於包括基於銷售的特許權使用費和被視爲特許權使用費相關主要項目的知識產權許可的安排,收入在相關銷售發生時或已分配部分或全部特許權使用費的履行義務已履行(或部分履行)時(較晚)時確認。
可選購買:公司的安排 可以 向被許可人提供選擇性購買許可產品的權利。當被許可人確定將進行此類購買時,這些選擇性購買將作爲單獨的合同覈算,除非該選擇權傳達了重大權利。可選購買記錄爲產品淨收入。
其他收入
收入還來自爲客戶提供監管支持和開發服務而確認的服務費用。服務費收入在服務轉移給客戶時隨時間確認。
延期產品收入
當公司在合同條款下完成對客戶的履行義務之前從客戶收到對價或無條件到期該對價時,合同負債被記錄爲遞延收入。預計將在年內確認爲收入的遞延收入 12 資產負債表日後的幾個月被歸類爲流動負債。遞延收入 不 預計將在 12 資產負債表日後的幾個月被歸類爲長期負債。
應收帳款
一般來說,應收賬款包括應收客戶款項,扣除現金折扣、產品退貨和退款的客戶備抵。我們與客戶的合同有標準付款條款。我們分析逾期未收的帳戶,並定期評估客戶的信譽,以便我們能夠正確評估和應對他們信用狀況的變化。截至 2024年9月30日,我們確定了與未償應收賬款相關的預期信用損失撥備,目前 不 根據我們對合同付款條款和個人客戶情況的審查而要求。
收入成本
收入成本主要包括 三- 因BRIUMVI銷售而欠我們許可合作伙伴的方製造成本、分銷、管理費用和特許權使用費。 收入成本 可以 還包括與超額或過時的庫存調整費用、異常成本、未吸收的製造和管理費用以及製造差異相關的成本。根據我們在監管機構批准之前承擔與產品製造相關成本的政策,迄今爲止銷售的BRIUMVI生產成本的一部分在FDA批准BRIUMVI之前已作爲研究和開發費用,因此 不 反映在收入成本上。 我們的收入成本還與向客戶提供監管支持和開發服務有關。
庫存
庫存按成本或估計可變現淨值中的較低者列賬,成本基於 第一- 在-第一-out方法(FIFA)。公司在每個報告期內對資本化庫存的可收回性進行評估,並將任何超額和廢棄庫存減記至其在發生損失期間的估計可變現淨值 第一 鑑定此類減損費用(如果發生)將記錄在收入成本中。
在獲得監管機構批准之前,我們將與庫存生產相關的成本作爲發生期間的研發費用。監管機構批准後,這些批准產品的製造成本將資本化。當確定用於臨床試驗時,可用於臨床或商業產品生產的庫存將作爲研發成本支出。
股本證券
我們的股權證券由精密生物科學公司的普通股組成。(精確)。根據ASC,股權證券按公允價值確認 321, 投資-股票證券。購買具有以下功能的股票證券的遠期合同 不 符合ASC下的衍生品資格 815 根據ASC覈算 321. 該等遠期合同按資產負債表日的公允價值記錄。見附註 5 了解更多詳情。
每股普通股淨利潤(損失)
我們普通股的每股基本淨利潤(損失)是通過將適用於普通股的淨利潤(損失)除以本期已發行普通股的加權平均股數來計算的。每股普通股稀釋淨收益(損失)包括潛在行使或轉換(例如認購證、股票期權和限制性股票)的影響(如果有的話),這將導致發行增量普通股股份。這些項目的影響在淨虧損期間具有反稀釋作用。因此,所列所有期間的每股基本和稀釋淨利潤(虧損)將相同。但對於 三和九 截至 2024年9月30日和2023, 該公司在未經審計的簡明綜合經營報表中擁有淨利潤。
.
下表總結了我們的潛在稀釋證券 2024年9月30日和 2023:
| 自.起 | |||||||
| 9月30日, | |||||||
| 2024 | 2023 | ||||||
未歸屬的限制性股票 | ||||||||
選項 | ||||||||
認股權證 | ||||||||
票據轉換後可發行的股份 | ||||||||
總 |
基本和稀釋每股收益的計算如下:
| 止三個月 | 止九個月 | ||||||||||||||
| 9月30日, | 9月30日, | ||||||||||||||
(in數千,份額和每股數據除外) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
淨利潤(虧損) | ||||||||||||||||
加權平均流通普通股 | ||||||||||||||||
潛在普通股的稀釋效應 | ||||||||||||||||
假設稀釋的加權平均流通普通股 | ||||||||||||||||
| ||||||||||||||||
每股淨利潤(虧損)-基本 | ||||||||||||||||
每股淨利潤(虧損)-稀釋 |
注意 2 收入
如註釋中所討論 1, 收入根據ASC內的指導確認 606. 下表列出了我們在所列期間的分類收入(以千計):
(in數千) | 截至9月30日的三個月裏, | 截至9月30日的九個月裏, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
產品總收入,淨 | $ | $ | $ | $ | ||||||||||||
許可證收入 | ||||||||||||||||
里程碑收入 | ||||||||||||||||
使用費收入 | ||||||||||||||||
其他收入 | ||||||||||||||||
總收入 | $ | $ | $ | $ |
產品收入,淨
為 三和九 截至 2024年9月30日,我們唯一的產品收入來源是在美國銷售BRIUVI,我們於年開始向客戶發貨 2023年1月。
截至 2024年9月30日,約為$
許可協議
Neuraxpharm商業化協議
對 2023年7月28日, 該公司與Neuraxpharm簽訂了一項美國以外的商業化協議(商業化協議)。公司授予Neuraxpharm在美國、加拿大和墨西哥以外的地區商業化BRIUMVI的獨家權利,這些地區由公司保留,但不包括公司之前合作的某些亞洲國家(地區)。此外,該公司將爲Neuraxpharm開展某些開發和監管活動,以支持其在商業化協議下的義務,以確保和維持在該地區出售BRIUVI所需的監管批准。作爲整體安排的一部分,公司已同意在整個商業化協議期限內向Neuraxpharm供應BRIUMVI。
作爲簽訂商業化協議的對價,公司收到了不可退還的預付款美元
公司評估了ASC項下的商業化協議 606 並得出結論,Neuraxpharm代表交易中的客戶。根據該指南,公司根據該安排確定了以下承諾:(i)在該地區開發、銷售、出售要約和進口產品的獨家權利(許可證);(ii)某些開發和監管活動(開發和監管活動)。
公司知識產權的許可代表了獨特的履行義務,因此,美元
開發和監管活動履行義務會隨着時間的推移而得到滿足,因爲Neuraxpharm同時接收並消耗公司履行服務所提供的利益。因此,收入在公司完成活動時確認。 爲 三和九 截至 2024年9月30日,公司確認其他收入爲美元
該安排還爲Neuraxpharm提供了選擇性購買BRIUMVI(許可產品供應)的權利。當行使購買BRIUMVI的權利時,這些選擇性購買將作爲單獨的合同覈算。Neuraxpharm選擇購買BRIUMVI的對價接近該地區客戶願意爲這些商品支付的價格。
其餘的對價形式是可變的,因爲它們取決於基於銷售的目標或其他里程碑的實現情況。該公司評估了可變對價的限制,並得出結論,里程碑付款高度依賴於公司控制之外的因素。因此,在合同開始時,里程碑是不按原樣包含在交易價格中不很可能收入的顯著逆轉將不發生。以銷售爲基礎的里程碑將在達到相關銷售門檻的期間確認爲收入。所有其他里程碑將在基本里程碑可能實現的期間立即確認爲收入。2024年3月,該公司收到了一美元
注 3 投資證券
截至目前我們的短期投資 2024年9月30日和 2023年12月31日 被歸類爲持有至到期。持有至到期投資按攤銷成本記錄。
下表總結了我們的短期投資證券 2024年9月30日和 2023年12月31日:
| 2024年9月30日 | |||||||||||||||
| 攤銷 | 總收入 | 總收入 | | ||||||||||||
| 成本,作爲 | 未實現 | 未實現 | 估計 | ||||||||||||
(in數千) | 調整後 | 持有收益 | 持有損失 | 公平值 | ||||||||||||
短期投資: | | | | | ||||||||||||
國內政府機構的債務(2024年10月至2025年3月到期)(持有至到期) | $ | $ | $ | $ | ||||||||||||
短期投資總額 | $ | $ | $ | $ |
| 2023年12月31日 | |||||||||||||||
| 攤銷 | 總收入 | 總收入 | | ||||||||||||
| 成本,作爲 | 未實現 | 未實現 | 估計 | ||||||||||||
| 調整後 | 持有收益 | 持有損失 | 公平值 | ||||||||||||
短期投資: | | | | | ||||||||||||
國內政府機構的債務(2024年1月至2024年6月到期)(持有至到期) | $ | $ | $ | $ | ||||||||||||
短期投資總額 | $ | $ | $ | $ |
截至目前我們的長期投資 2024年9月30日 包括精密普通股股份。截至2011年股本證券的公平市場價值 2024年9月30日 為$
注意 4 庫存
下表列出了截至2013年的庫存 2024年9月30日 (in數千):
| 2024年9月30日 | |||
原材料 | $ | |||
Work in Process | ||||
成品 | ||||
總庫存 | $ |
庫存以成本或可變現淨值中的較低者列報,由原材料、在製品和產成品組成。成本是使用標準成本法確定的,該方法接近實際成本,並假設貨物的先進先出流。用於臨床開發目的的庫存在消耗時計入研發費用。
在 2024年9月30日,我們所有的庫存都僅與BRIUMVI相關,該公司於2011年獲得FDA批准 2022年12月28日, 當時我們開始利用成本來製造BRIUMVI。在FDA批准BRIUMVI之前,與BRIUMVI和相關材料的製造相關的所有成本均在發生期間計入研發費用。
過程材料主要由散裝原料藥組成,其保質期爲多年。 當散裝原料藥生產成BRIUMVI成品時,這些成品的保質期爲 三 自制造之日起數年。 我們的期望是至少能銷售成品 十二 到期前幾個月。由於我們的製造交貨時間較長,有必要建立庫存以支持BRIUMVI的預測銷售,以確保適當的安全庫存水平,並履行我們向Neuraxpharm供應BRIUMVI的承諾。由於處於BRIUMVI產品推出的早期階段,該公司正在繼續評估其運營週期的長度。
該公司每季度分析我們的庫存水平,以確定超額數量和報廢(到期),並考慮歷史和預期未來銷售額與現有數量和剩餘保質期相比等因素。在 2024年9月30日,我們確定與BRIUMVI庫存相關的超額數量和報廢儲備爲
必需的.此外,自FDA批准BRIUMVI以來,該公司已 不 記錄了任何庫存減記。
注 5 公平值計量
我們在簡明綜合財務報表中按經常性基準計量某些金融資產和負債。公允價值等級制度對用於確定公允價值的輸入數據或假設的質量和可靠性進行排名,並要求按公允價值列賬的金融資產和負債分類和披露 一 以下 三 類別:
● | 水平1 相同資產和負債在活躍市場上的報價; |
● | 水平2 級別以外的輸入 1 可直接或間接觀察的報價;以及 |
● | 水平3 不可觀察的輸入 不 市場數據證實了這一點。 |
股權投資和遠期合同負債
在……上面 2024年1月7日,(精度生效日期)該公司及其全資子公司TG Cell Therapy,Inc.,(TG Cell)與Quality簽訂了許可協議(「Quality許可協議」),根據該協議,Quality授予該公司某些獨家和非獨家許可權,以開發、製造和商業化Quality的同種基因CAR t療法azercabtagene zapoparucel(azer-cel),用於治療自身免疫性和其他非腫瘤性疾病和病症。
根據精密許可協議,公司向精密預付了美元
內 12 精度生效日期後幾個月,公司將制定 一- 向精度支付的時間相當於美元
臨牀和監管里程碑事件(里程碑事件 1),公司將制定 一- 向精度支付的時間相當於美元
5%備註
在我們合併時(我們當時被稱爲曼哈頓製藥公司。(曼哈頓))與Ariston Pharmaceuticals,Inc.(阿里斯頓)在 三月 2010, 阿里斯頓發行美元
公司的金融工具包括現金、由貨幣市場基金組成的現金等值物、應收賬款、應付賬款和應付貸款。截至 2024年9月30日和 2023年12月31日現金及現金等值物、受限制現金、應收賬款以及應付貸款和利息的公允價值與其公允價值接近。由於利率接近具有類似條款和風險特徵的貸款的市場利率,公司資產負債表上應付貸款的公允價值估計接近其公允價值。
The following tables provide the fair value measurements of applicable financial assets and liabilities as of September 30, 2024 and December 31, 2023:
截至2024年9月30日按公允價值計算的金融資產和負債 | ||||||||||||||||
(單位:千) | 1級 | 2級 | 3級 | 總 | ||||||||||||
股權投資 | $ | $ | $ | $ | ||||||||||||
總資產 | $ | $ | $ | $ | ||||||||||||
遠期合同負債 | $ | $ | $ | $ | ||||||||||||
5%筆記 | $ | $ | $ | $ | ||||||||||||
總負債 | $ | $ | $ | $ |
截至2023年12月31日按公允價值計算的金融資產和負債 | ||||||||||||||||
1級 | 2級 | 3級 | 總 | |||||||||||||
5%筆記 | $ | $ | $ | $ | ||||||||||||
總負債 | $ | $ | $ | $ |
我們的股權投資分類爲級別 1 使用各自在納斯達克股市的收盤價進行估值。我們做 不 期間經歷公允價值層級之間的任何金融工具轉移 三 截至 2024年9月30日.我們的遠期合同負債分類爲級別 2 使用精密在納斯達克股市的收盤價進行估值。我們的水平 3 工具金額代表 5% 票據和相關應計利息。
該級別公允價值的變化 1 資產和級別 2 和水平 3 負債在隨附的簡明綜合經營報表中的其他(收入)費用中報告。
注意 6 股東’ 股權
優先股
我們修訂和重述的公司註冊證書授權發行最多
普通股
我們修訂和重述的公司註冊證書授權發行最多
對 2022年9月2日, 我們在表格S上提交了自動“貨架登記”聲明-3 ( 2022 WKSI Shelf)作爲規則中定義的“知名經驗豐富的發行人” 405 根據《證券法》,該法登記了無限和不確定金額的債務或股權證券以供未來發行和銷售。的 2022 WKSI貨架於年宣佈生效 2022年9月。 關於2022 WKSI貨架上,我們簽訂了一份市場發行銷售協議( 2022 ATM)與Cantor Fitzgerald & Co.和b.萊利證券公司(each一 2022 代理人和總的來說, 2022 代理人),與我們普通股股份的銷售有關。下 2022 ATM,我們將支付 2022 代理商佣金率高達
期間 六 截至 2023年6月30日, 我們總共售出了
股票回購計劃和國庫券
對 2024年8月2日, 該公司宣佈,其董事會(董事會)已授權並批准了一項高達美元的股份回購計劃
股權激勵計劃
TG Therapeutics,Inc 2022 激勵計劃( 2022 激勵計劃)於年獲得股東批准 2022年6月 與
TG Therapeutics,Inc修訂及重列 2012 激勵計劃( 2012 激勵計劃)於年獲得股東批准 六月 2020. 截至 2024年9月30日,
簡明綜合經營報表中包含的股票補償費用爲美元
期間 七月 的 2024, 公司發現了與授予的單一限制性股票獎勵的費用確認有關的錯誤 2021. 該錯誤的影響是低估了年底前的非現金薪酬費用(SG & A) 2022年12月31日, 和 2021 並相應少報額外實繳資本(APIC)。該公司已得出結論,該錯誤確實造成了 不 導致公司先前發佈的合併財務報表出現重大錯誤陳述。該錯誤的累積影響已被糾正,作爲對 2023年12月31日 通過增加累計赤字和亞太地區消費總值約美元來精簡合併資產負債表
股票期權與限制性股票
下表總結了股票期權和限制性股票的活動 九 截至 2024年9月30日:
| 股票期權 | 限制性股票 | ||||||
年初股權獎勵優秀 | ||||||||
年內變化: | ||||||||
授予 | ||||||||
鍛鍊或被賦予 | ( | ) | ( | ) | ||||
到期或被沒收 | ( | ) | ||||||
期末傑出股權獎勵 |
截至 2024年9月30日、與未授予的時間獎勵相關的總補償成本 不 尚未確認以及預計確認獎項的加權平均期如下:
(in數千) | 股票期權 | 限制性股票 | ||||||
未確認的賠償成本 | $ | $ | ||||||
預計加權平均期(以年數計)將確認的補償成本 |
截至 2024年9月30日, 與未歸屬的市場獎勵相關的總報酬成本 不 尚未確認以及預計確認獎項的加權平均期如下:
(in數千) | 限制性股票 | |||
未確認的賠償成本 | $ | |||
預計加權平均期(以年數計)將確認的補償成本 |
權證
該公司唯一未執行的認購證是作爲先前貸款協議的一部分向Hercules發行的認購證
公司根據以下關鍵假設使用布萊克-斯科爾斯模型估計了第一修正案令狀的公允價值:
修改後的定期貸款 | ||||
行使價 | $ | |||
發行日普通股價格 | $ | |||
波動 | % | |||
無風險利率 | % | |||
預期股息收益率 | — | |||
合同期限(年) |
注意 7 應付貸款
對 2023年3月31日, 公司與Hercules Capital,Inc.簽訂了修訂後的貸款協議的第一修正案(第一修正案)。(赫拉克勒斯)。第一修正案修改了與Hercules簽訂的修訂和重述的貸款和擔保協議(修訂後的貸款協議)的條款,該協議於2010年結束。 2021年12月30日。
On August 2, 2024 (the New Closing Date), the Company entered into a term loan facility of $
The Initial Term Loan is governed by a financing agreement, dated as of the New Closing Date (the Financing Agreement), which provides for (i) a single draw of the Initial Term Loan on the New Closing Date and (ii) an uncommitted additional facility in an aggregate principal amount of up to $
The Initial Term Loan is secured by a lien on substantially all of the assets of the Company and certain subsidiaries of the Company as guarantors and contains customary covenants and representations.
The events of default under the Financing Agreement are customary for financings of this type. If an event of default occurs, the Administrative Agent is entitled to take enforcement action, including acceleration of amounts due under the Financing Agreement.
The Company evaluated whether the Initial Term Loan represented a debt modification or extinguishment of the First Amendment with Hercules with ASC 470-50, Debt – Modifications and Extinguishments. As a result of the Initial Term Loan and effective termination of the First Amendment with Hercules, this transaction was accounted for by the Company under the extinguishment accounting model. The Company recorded a loss on extinguishment of debt of approximately $
該公司發生的融資總額和前期成本爲美元
第一修正案的應付貸款餘額 2024年9月30日和 2023年12月31日 如下:
第一修正案 | ||||||||
| 9月30日, | 12月31日, | ||||||
(單位:千) | 2024 | 2023 | ||||||
應付貸款 | $ | $ | ||||||
添加:最終付款費的確認責任 | ||||||||
| ||||||||
減:未攤銷債務發行和債務貼現成本 | ) | |||||||
| ||||||||
減:本金付款 | ) | |||||||
應付貸款總額 | ||||||||
減:當前部分 | ||||||||
非流動應付貸款 | $ | $ |
The loan payable balance of the Initial Term Loan as of September 30, 2024, is as follows:
初始定期貸款 | ||||
| 9月30日, | |||
(單位:千) | 2024 | |||
應付貸款 | $ | |||
添加:最終付款費的確認責任 | ||||
| ||||
減:未攤銷債務發行和債務貼現成本 | ) | |||
| ||||
減:本金付款 | ||||
應付貸款總額 | ||||
減:當前部分 | ||||
非流動應付貸款 | $ |
注意 8 租賃
在 2014年10月, 我們與Fortress Biotech,Inc.簽訂了一份協議(辦公室協議)。(F比奧)大約佔據
在 2019年10月, 我們最終確定了
在 2021年10月, 我們最終確定了
在 2019年1月1日, 我們確認了租賃負債和相應的使用權(ROU)資產爲美元
| 止三個月 | 止九個月 | ||||||||||||||
| 9月30日, | 9月30日, | 9月30日, | |||||||||||||
(in數千) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
經營租賃成本 | $ | $ | $ | $ | ||||||||||||
淨租賃成本 | $ | $ | $ | $ |
截至 2024年9月30日,加權平均剩餘經營租賃期限爲
| 9月30日, | 12月31日, | ||||||
(in數千) | 2024 | 2023 | ||||||
負債 | | | ||||||
租賃負債流動部分 | $ | $ | ||||||
非流動租賃負債 | ||||||||
租賃負債總額 | $ | $ |
截至 2024年9月30日,租賃負債的到期日如下:
| 操作 | |||
(in數千) | 租賃 | |||
2024年剩下的 | $ | |||
2025 | ||||
2026 | ||||
2027 | ||||
2028 | ||||
2029年之後 | ||||
租賃付款總額 | ||||
減:利息 | ( | ) | ||
租賃負債現值(*) | $ |
(*) | As our leases do not provide an implicit rate, we use our incremental borrowing rate based on the information available at commencement date and considering the term of the lease to determine the present value of lease payments. We used the incremental borrowing rate of |
注意 9 許可協議
BRIUMVI(Ublituximab)
在 2012年1月, 我們與LFb集團的全資子公司LFb Biotechnologies、GTC Biotherapeutics和LFB/GTC LLC簽訂了一份關於ublituximab開發的獨家許可協議(LFb許可協議)。根據LFb許可協議的條款,我們獲得了ubilituximab的開發和商業化全球獨家權利(法國/比利時除外)。截至 2024年9月30日,我們已經花費了大約美元
LFb Group有資格獲得ublituximab淨銷售額的特許權使用費,特許權使用費率從中個位數上升到高個位數。許可證將在最後一次許可的專利權到期後根據國家而終止,或
在 2012年11月, 我們與Ildong Pharmaceutical Co. Ltd.(Ildong)就ublituximab在韓國和東南亞的開發和商業化達成獨家(境內)分許可協議。根據分許可協議的條款,Ildong獲得了在韓國、臺灣、新加坡、印度尼西亞、馬來西亞、泰國、菲律賓、越南和緬甸開發和商業化ublituximab的特許權,包括授予分許可的權利。
預付美元
我們 可以 最多可獲得額外$
在 2023年7月, 該公司與Neuraxpharm簽訂了商業化協議。該公司授予Neuraxpharm在美國、加拿大和墨西哥以外地區商業化BRIUMVI的獨家權利,這些權利由TG保留,但不包括之前合作的某些亞洲國家。根據商業化協議的條款,公司收到了一份 一- 時間,不可退還$
尤利西斯
對 2024年1月7日, 該公司及其全資子公司TG Cell Therapy,Inc.,與精密簽訂了《精密許可協議》,根據該協議,精密授予該公司某些獨家和非獨家許可權,以開發、製造和商業化精密的異基因CAR t療法阿澤卡太烯扎普魯塞爾(azer-cel),用於治療自身免疫和其他非腫瘤疾病和病症。
根據Precision許可協議,公司向Precision支付了一筆預付款$
注意 10 關聯交易
在 七月 2015, 我們與F貝洛簽訂了共享服務協議(共享服務協議),以分擔某些服務的成本,例如設施使用、人員成本以及其他管理費用和行政成本。共享服務協議要求我們支付各自所使用的服務份額。就共享服務協議而言,我們發生了約美元的費用
請參閱備註8 - 有關與F比奧的辦公室協議的詳細信息的租賃。
以下討論和分析包含有關我們的計劃和對未來可能發生的事情的預期的前瞻性陳述。前瞻性陳述基於一系列假設和估計,這些假設和估計固有地受到重大風險和不確定性的影響,由於許多已知或未知因素,我們的結果可能與前瞻性陳述預期的結果存在重大差異,包括但不限於風險因素中討論的那些因素。另請參閱本報告開頭提出的關於前瞻性陳述的特別警告通知。
您應結合本報告其他地方出現的未經審計的簡明綜合財務報表及其相關腳註,以及管理層的討論和分析以及我們截至2023年12月31日年度10-k表格年度報告中包含的經審計綜合財務報表,閱讀以下討論和分析。
概述
TG治療公司是一家完全整合的、處於商業階段的生物製藥公司,專注於b細胞疾病新型治療方法的收購、開發和商業化。除了包括幾種研究藥物的研究流水線外,TG還獲得了FDA對BRIUMVI(ublituximab-xiy)的批准,用於治療成人複發性多發性硬化症(RMS),包括臨牀孤立綜合徵、復發-緩解性疾病和活動性繼發性進展性疾病。TG還獲得了歐盟(EU)的歐洲委員會(EC)和英國(UK)的藥品和保健產品監管機構(MHRA)對BRIUMVI的批准,用於治療具有臨牀或影像特徵定義的活動性疾病的RMS成年患者。TG繼續積極評估互補產品、技術和公司的內部許可、夥伴關係、收購和/或投資機會。
最近的業務更新亮點
BRIUMVI五年數據更新
2024年9月,我們在2024年歐洲多發性硬化症治療和研究委員會(ECTRIMS)年會上展示了ULTIMATE I & II III期試驗的新五年數據,該試驗評估了BRIUMVI®(ublituximab-xiiy)治療RMS患者。這些數據表明,經過五年的BRIUVI治療後,92%的RMS患者沒有出現殘疾進展,治療第五年的年複發率爲0.02(相當於患者治療每五十年發生一次復發),並且總體安全性特徵在五年的持續治療中保持一致,長期治療沒有出現新的安全信號。
Ublituximab Development
2024年8月,我們宣佈啓動一項I期臨牀試驗,評估Ublituximab皮下製劑治療RMS患者,並且患者已接受給藥。
管道
2024年8月,我們宣佈FDA批准阿澤卡烯扎普盧塞爾(azer-cel)用於治療進行性多發性硬化症的研究性新藥申請(IND)。此前,我們於2024年1月與精密生物科學公司達成了協議。(精密)將獲得精密azer-cel的全球許可,用於治療自身免疫性疾病和所有其他非腫瘤適應症。Deliver-cel是一種同種異基因(現成)CAR t計劃,該公司近期計劃在多種自身免疫適應症中評估該計劃。
我司的產品
我們目前在全球範圍內對我們所有正在開發的產品進行開發和商業權許可,但須遵守某些有限的地理限制。下表總結了截至2024年11月我們主要候選藥物的當前臨床試驗狀態。
臨床候選藥物: |
初始目標疾病 |
發展階段/狀況 |
Ublituximab(抗CD 20單克隆抗體) |
多發性硬化症(RMS)的復發形式 |
批准 |
皮下注射烏布利妥昔單抗 |
RMS |
階段1 |
尤利西斯 |
多發性硬化的進展形式 |
IND已接受 |
BRIUMVI(ublituximab-xiiy)概覽
BRIUMVI是第一個也是唯一一個獲准用於治療RMS的抗CD 20單克隆抗體,包括成人臨床孤立綜合徵、復發緩解性疾病和活動性次要進展性疾病,可以在開始劑量後每年兩次、一小時輸注給藥。
BRIUMVI的批准主要基於ULTIMATE I和ULTIMATE II 3期試驗。每項試驗都是一項獨立的全球、隨機、多中心、雙盲、雙模擬、活性對照研究,比較ublituximab的療效和安全性/耐受性(450毫克劑量,每6個月靜脈輸注1小時,第1天在4小時內輸注150毫克和第15天在1小時內輸注450毫克)與特立氟胺(14毫克口服片劑,每天一次)治療患有RMS的受試者。這些試驗是在FDA的特殊方案評估(SPA)下進行的。ULTIMATE I和II試驗由斯坦福大學神經病學和神經科學、兒科和遺傳學教授Lawrence Steinman醫學博士領導。全面入組於2018年10月完成,兩項研究總共入組了約1,100名受試者。
● |
2020年12月,我們宣佈了終極I&II試驗的陽性背線結果。這兩項研究都達到了在96周內顯著降低ARR的主要終點(在每項研究中P<0.005),BRIUMVI在每項研究中顯示ARR<0.1。與特氟米特相比,終末期I和II的ARR分別減少了約60%和50%。還滿足了關鍵的次要MRI終點。 | |
● |
2022年8月22日,I&II終極試驗的全部結果發表在《新英格蘭醫學雜誌》上。 |
|
● | 2024年2月27日,我們宣佈美國專利商標局(USPTO)爲BRIUMVI額外頒發了三項專利,將專利保護延長至2042年。 | |
● | 2024年8月,我們宣佈啓動一項第一階段臨床試驗,評估烏布妥昔單抗皮下製劑在RMS患者中的應用,第一批患者已經服藥。 | |
● |
來自最終I和II期3期試驗的其他數據集繼續在主要醫學會議上公佈,包括在9月份舉行的2024年ECTRIMS會議上首次提交的5年長期數據,以及來自Enhance 30期億試驗的最新數據,該試驗評估了從IV抗CD20療法切換到BRIUMVI的複發性硬化症患者,並評估了BRIUMVI的更短輸液時間(低至30分鐘)。 |
BRIUMVI的美國商業化
2022年12月28日,我們宣佈FDA批准BRIUVI用於治療成人RMS,包括臨床孤立綜合徵、復發緩解性疾病和活動性次要進展性疾病,主要基於ULTIMATE I & II 3期試驗的結果。2023年1月26日,我們宣佈在美國商業推出BRIUMVI,供醫生和患者使用,第一位RMS患者於2023年2月1日接受了BRIUMVI輸注。
Ex-U.S. Commercialization of BRIUMVI
On June 1, 2023, we announced that the EC granted approval of BRIUMVI for the treatment of adult patients with RMS who have active disease defined by clinical or imaging features. With this approval, the centralized marketing authorization is valid in all EU member states, Iceland, Norway and Liechtenstein.
On August 1, 2023, we announced an agreement with Neuraxpharm Pharmaceuticals, S.L. (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of CNS disorders, for the Ex-U.S. commercialization of BRIUMVI (Commercialization Agreement). Under the terms of the Commercialization Agreement, we received an upfront payment of $140 million, and $12.5 million upon launch in the first EU country (February 2024) and up to an additional $492.5 million in milestone-based payments on achievement of certain launch and commercial milestones. The total deal is valued at up to $645 million in upfront and milestone payments. In addition, we will receive tiered double-digit royalties on net product sales up to 30%. In exchange, Neuraxpharm will have the exclusive right to commercialize BRIUMVI in territories outside the U.S., Canada and Mexico, which are retained by TG, and excluding certain Asian countries of which the Company previously partnered. We retain an option to buy back all rights under the Commercialization Agreement for a period of two years in the event of a change in control of TG.
On November 1, 2023, we announced that we also received approval by the MHRA for BRIUMVI to treat adult patients with RMS with active disease defined by clinical or imaging features in the UK.
On February 26, 2024, we announced the commercial launch of BRIUMVI in the EU by Neuraxpharm, with BRIUMVI made available for commercial sale in Germany, with additional EU markets expected to follow.
For more information, please refer to our Annual Report on Form 10-K for the quarter and year ended December 31, 2023.
RESULTS OF OPERATIONS
The following table summarizes the results of operations for the three months ended September 30, 2024 and 2023:
止三個月 |
||||||||
9月30日, |
||||||||
(in數千) |
2024 |
2023 |
||||||
產品收入,淨 |
$ | 83,297 | $ | 25,068 | ||||
許可證、里程碑、版稅和其他收入 |
582 | 140,747 | ||||||
總收入 |
$ | 83,879 | $ | 165,815 | ||||
成本和費用: |
||||||||
收入成本 |
9,341 | 3,509 | ||||||
研究與開發: |
||||||||
非現金補償 |
3,028 | 2,915 | ||||||
其他研究和開發 |
17,110 | 11,838 | ||||||
研究和發展的總 |
20,138 | 14,753 | ||||||
一般和行政: |
||||||||
非現金補償 |
8,745 | 6,269 | ||||||
其他銷售,一般和行政 |
33,221 | 26,500 | ||||||
總務和行政總計 |
41,966 | 32,769 | ||||||
總成本和支出 |
71,445 | 51,031 | ||||||
利息開支 |
10,832 | 3,713 | ||||||
其他收入 |
(2,666 | ) | (2,859 | ) | ||||
其他費用總額,淨額 |
8,166 | 854 | ||||||
稅前淨利潤 |
4,268 | 113,930 | ||||||
所得稅開支 |
388 | — | ||||||
淨收入 |
$ | 3,880 | $ | 113,930 |
產品收入,淨. 截至2024年9月30日的三個月,產品淨收入約爲8330加元,而截至2023年9月30日的三個月爲2510加元。截至2024年9月30日和2023年9月30日止三個月的產品淨收入包括BRIUVI在美國的產品淨銷售額。2023年1月,獲得FDA批准後,我們開始在美國境內商業銷售BRIUVI。
許可證、里程碑、版稅和其他收入.截至2024年9月30日的三個月,許可證、里程碑、特許權使用費和其他收入爲60加元,截至2023年9月30日的三個月爲1.407億美元。截至2024年9月30日止三個月的許可證、里程碑、特許權使用費和其他收入主要包括代表Neuraxpharm Pharmaceuticals,SL進行的開發和監管活動收到的對價(Neuraxpharm)根據與Neuraxpharm的商業化協議(更多信息請參閱注2)。截至2023年9月30日止三個月的許可證、里程碑、特許權使用費和其他收入主要包括根據與Neuraxpharm的商業化協議確認的一次性14000美元不可退還預付款。
收入成本。截至2024年9月30日的三個月的收入成本爲930萬,而截至2023年9月30日的三個月的收入成本約爲350美元萬。截至2024年9月30日和2023年9月30日的三個月的收入成本主要包括第三方製造、分銷、管理費用和欠我們的許可合作伙伴的BRIUMVI銷售版稅。迄今爲止銷售的BRIUMVI生產成本的一部分在食品和藥物管理局批准BRIUMVI之前作爲研究和開發支出,因此沒有反映在收入成本中。我們預計,隨着我們消耗這些庫存,BRIUMVI的收入成本將相對於產品收入增加。我們預計在2025年第一季度之前將剩餘的商業化前庫存用於產品銷售,之後我們的產品毛利率預計將略有下降。截至2024年9月30日和2023年9月30日的三個月的收入成本分別包括根據商業化協議向Neuraxpharm提供監管支持和開發服務相關的30美元萬和70美元萬。
非現金薪酬分配器(研究與開發).截至2024年9月30日的三個月,與股權激勵補助相關的非現金薪酬費用(研究與開發)總計300加元,而截至2023年9月30日的可比期間爲290加元。
其他研究與開發. 截至2024年9月30日的三個月,其他研發費用與截至2023年9月30日的期間相比增加了約530盧比至1710盧比。截至2024年9月30日的三個月內研發費用增加主要是由於本期人員、臨床試驗相關費用和製造成本增加。
非現金補償發票(銷售、一般和行政). 截至2024年9月30日的三個月,與股權激勵補助相關的非現金薪酬費用(銷售、一般和行政)總計870盧比,而截至2023年9月30日的同期爲630萬美元。非現金薪酬費用的增加主要是由於截至2024年9月30日的三個月內更多地確認了向高管發放的非現金薪酬費用。
其他銷售、一般和行政費用. 截至2024年9月30日的三個月,其他銷售、一般和行政費用總計3,320美元,而截至2023年9月30日的同期爲2,650萬美元。這一增加主要是由於截至2024年9月30日的三個月內與BRIUMVI商業化相關的人員、顧問和第三方的其他銷售、一般和行政成本。
利息開支. 截至2024年9月30日止三個月的利息費用增加了710盧比,達到1080盧比,而截至2023年9月30日止三個月的利息費用爲370萬美元。截至2024年9月30日的三個月的利息費用包括與Hercules的第一修正案相關的約460加元債務消除成本,以及與同期Blue Owl的初始期限貸款相關的440加元利息費用(更多信息請參閱註釋7)。
其他收入. 截至2024年9月30日的三個月,其他收入總計爲270盧比,而截至2023年9月30日的可比期間爲290盧比。
下表總結了截至2024年和2023年9月30日止九個月的經營業績:
止九個月 |
||||||||
9月30日, |
||||||||
(in數千) |
2024 |
2023 |
||||||
產品收入,淨 |
$ | 206,381 | $ | 48,868 | ||||
許可證、里程碑、版稅和其他收入 |
14,438 | 140,823 | ||||||
總收入 |
$ | 220,819 | $ | 189,691 | ||||
成本和費用: |
||||||||
收入成本 |
23,087 | 6,277 | ||||||
研究與開發: |
||||||||
非現金補償 |
8,000 | 10,162 | ||||||
其他研究和開發 |
62,417 | 48,581 | ||||||
研究和發展的總 |
70,417 | 58,743 | ||||||
一般和行政: |
||||||||
非現金補償 |
22,593 | 18,386 | ||||||
其他銷售,一般和行政 |
92,742 | 73,167 | ||||||
總務和行政總計 |
115,335 | 91,553 | ||||||
總成本和支出 |
208,839 | 156,573 | ||||||
利息開支 |
16,967 | 10,184 | ||||||
其他收入 |
(5,128 | ) | (4,154 | ) | ||||
其他費用總額,淨額 |
11,839 | 6,030 | ||||||
稅前淨利潤 |
141 | 27,088 | ||||||
所得稅開支 |
89 | — | ||||||
淨收入 |
$ | 52 | $ | 27,088 |
產品收入,淨. 截至2024年9月30日止九個月的產品淨收入約爲20640盧比,而截至2023年9月30日止九個月的產品淨收入爲4890盧比。截至2024年9月30日和2023年9月30日止九個月的產品淨收入包括BRIUVI在美國的產品淨銷售額。2023年1月,獲得FDA批准後,我們開始在美國境內商業銷售BRIUVI。
License, Milestone, Royalty and Other Revenue. License, milestone, royalty and other revenue was $14.4 million for the nine months ended September 30, 2024 compared to approximately $140.8 million for the nine months ended September 30, 2023. License, milestone, royalty and other revenue for the nine months ended September 30, 2024 is comprised of a $12.5 million milestone payment under the Neuraxpharm Commercialization Agreement for the first key market commercial launch of BRIUMVI in the EU, as well as consideration received for development and regulatory activities performed on behalf of Neuraxpharm in accordance with the Commercialization Agreement. License, milestone, royalty and other revenue for the nine months ended September 30, 2023 is predominantly comprised of recognition of the one-time $140.0 million non-refundable upfront payment under the Commercialization Agreement with Neuraxpharm (see Note 2 for more information).
Cost of Revenue. Cost of revenue for the nine months ended September 30, 2024 was $23.1 million compared to approximately $6.3 million for the nine months ended September 30, 2023. Cost of revenue for both the nine months ended September 30, 2024 and September 30, 2023 consists primarily of third-party manufacturing, distribution, overhead costs and royalties owed to our licensing partner for BRIUMVI sales. A portion of the costs of producing BRIUMVI sold to date was expensed as research and development prior to the FDA approval of BRIUMVI and therefore it is not reflected in the cost of revenue. We expect the cost of revenue for BRIUMVI to increase in relation to product revenues as we deplete these inventories. We expect to use the remaining pre-commercialization inventory for product sales through the first quarter of 2025, after which our product gross margin is anticipated to decrease modestly. The cost of revenue for the nine months ended September 30, 2024 and the nine months ended September 30. 2023 includes $1.4 million and $0.7 million, respectively, of costs related to delivering regulatory support and development services to Neuraxpharm in accordance with the Commercialization Agreement.
Noncash Compensation Expense (Research and Development). Noncash compensation expense (research and development) related to equity incentive grants totaled $8.0 million for the nine months ended September 30, 2024, as compared to $10.2 million during the comparable period ended September 30, 2023. The decrease in noncash compensation expense was primarily due to decreased vesting of milestone-based grants during the nine months ended September 30, 2024, as compared to the nine months ended September 30, 2023.
Other Research and Development Expense. Other research and development expense increased for the nine months ended September 30, 2024, by approximately $13.8 million to $62.4 million, as compared to $48.6 million during the comparable period ended September 30, 2023. The increase in other research and development expense during the nine months ended September 30, 2024 was primarily attributable to the Precision License Agreement, as well as manufacturing related expenses during the period, offset by a decrease in clinical trial expenses.
Noncash Compensation Expense (Selling, General and Administrative). Noncash compensation expense (selling, general and administrative) related to equity incentive grants totaled $22.6 million for the nine months ended September 30, 2024, as compared to $18.4 million during the comparable period ended September 30, 2023. The increase in noncash compensation expense was primarily due to greater recognition of noncash compensation expense for grants to executives during the nine months ended September 30, 2024.
其他銷售、一般和行政費用. 截至2024年9月30日的九個月內,其他銷售、一般和行政費用總計爲9,270美元,而截至2023年9月30日的同期爲7,320萬美元。這一增加主要是由於截至2024年9月30日的九個月內與BRIUMVI商業化相關的人員、顧問和第三方的其他銷售、一般和行政成本。
利息開支. 截至2024年9月30日止九個月的利息費用增加了680萬美元,達到1,700萬美元,而截至2023年9月30日止九個月的利息費用爲1,020美元。截至2024年9月30日止九個月的利息費用包括與Hercules的第一修正案相關的約460加元債務消除成本,以及同期與Blue Owl的初始期限貸款相關的增加利息費用(詳情請參閱註釋7)。
其他收入. 截至2024年9月30日止九個月的其他收入增加了100加元,達到510加元,而截至2023年9月30日止九個月的其他收入爲410加元。這一增加主要是由於截至2024年9月30日的九個月內,與截至2023年9月30日的可比期間相比,短期投資證券賺取的增值收入增加。
流動資金及資本資源
從歷史上看,我們曾出現過經營虧損;然而,在截至2024年9月30日的三個月和九個月內,公司實現了淨利潤。此外,在截至2023年12月31日的十二個月內,作爲我們與Neuraxpharm達成的美國前商業化協議(商業化協議)的一部分,該公司因2023年第三季度確認爲許可證收入的14000美元不可退還預付款而實現了淨利潤(更多信息請參閱注2)。我們可能會在短期內出現運營虧損,並且可能永遠無法盈利。截至2024年9月30日,我們累計赤字爲16億美元。
我們的主要現金來源是私募和公開發行股權證券的收益、我們的貸款和擔保協議、商業化協議的預付款(有關更多信息,請參閱附註2),以及BRIUMVI的藥品銷售產品收入。在截至2024年9月30日的前9個月裏,我們從BRIUMVI的藥品銷售中獲得了20640美元的萬產品收入。BRIUMVI是我們目前唯一上市的產品。我們幾乎所有的運營虧損都來自與我們的研發計劃相關的成本,以及與我們的運營相關的銷售、一般和管理成本,包括我們的商業化活動。隨着我們繼續將BRIUMVI商業化,我們預計將繼續產生大量的研發費用,以及大量的商業化和外包製造費用。由於與藥品開發和商業化相關的衆多風險和不確定性,我們無法預測未來虧損的程度,也無法預測我們可能在多長時間內繼續實現淨利潤。我們可能無法維持或提高我們的季度或年度盈利能力。我們盈利的能力取決於我們創造可觀收入的能力。
截至2024年9月30日,我們擁有34100美元的現金、現金等值物和投資證券。該公司相信,其現有現金、現金等值物和投資證券,加上與在美國和美國以外地區出售BRIOMVI相關的預計收入,足以滿足我們自本申請之日起至少十二個月內的預期運營現金需求。
我們運營所需的實際現金金額取決於許多因素,包括但不限於我們對BRIUMVI的商業化努力、爲我們其他候選藥物潛在商業化所做的準備,以及我們候選藥物臨床試驗的時機、設計和進行以及與許可或以其他方式收購新候選產品相關的成本。我們可能依賴大量的未來融資來提供執行我們當前和未來運營所需的現金,包括我們任何候選藥物的商業化。
現金流討論
下表總結了截至2024年和2023年9月30日止九個月的現金流:
|
止九個月 |
|||||||
9月30日, |
||||||||
(in數千) |
2024 |
2023 |
||||||
經營活動所用現金淨額 |
$ | (14,876 | ) | $ | (18,203 | ) | ||
投資活動所用現金淨額 |
$ | (16,824 | ) | $ | (5,896 | ) | ||
融資活動提供(用於)的淨現金 |
$ | 134,599 | $ | 72,706 |
截至2024年9月30日止九個月的經營活動中使用的現金爲1,490盧比,而截至2023年9月30日止九個月的經營活動中使用的現金爲1,820盧比。經營活動中使用的淨現金減少是由於截至2024年9月30日的九個月收入增加,但被同期經營支出增加和用於庫存採購的現金增加所抵消。
截至2024年9月30日止九個月投資活動中使用的淨現金爲1680盧比,而截至2023年9月30日止九個月投資活動中使用的淨現金爲590盧比。投資活動使用的淨現金增加主要是由於截至2024年9月30日的九個月內短期證券投資增加。
截至2024年9月30日止九個月的融資活動提供的淨現金約爲13460日元,而截至2023年9月30日止九個月的融資活動提供的淨現金爲7270日元。截至2024年9月30日的九個月內,融資活動提供的淨現金增加主要是由於與Blue Owl的新貸款的收益,但被我們之前與Hercules的貸款的償還所抵消。
OFF-BALANCE SHEET ARRANGEMENTS
我們沒有與未合併實體進行任何交易,因此我們提供財務擔保、次級保留權益、衍生工具或其他或有安排,使我們面臨重大持續風險、或有負債或爲我們提供融資、流動性、市場風險或信用風險支持的未合併實體可變權益下的任何其他義務。
關鍵會計政策和會計估計
關鍵會計政策既對描述我們的財務狀況和經營結果很重要,又要求管理層做出最困難、最主觀或最複雜的判斷,這往往是因爲需要對本質上不確定的事項的影響做出估計。有關我們的重要會計政策的說明,請參閱“第二部分,第8項.財務報表和補充數據,附註1--重要會計政策的組織和摘要” 在截至2023年12月31日的年度報告Form 10-k中,請參閱本季度報告Form 10-Q中的註釋1,了解由於收入確認、銷售總額與淨額調整、應收賬款、庫存和收入成本的商業化而產生的重大會計政策。在這些政策中,以下政策被認爲對理解我們的未經審計的簡明綜合財務報表至關重要,因爲它們需要應用最困難、最主觀和最複雜的判斷:基於股票的薪酬費用和金融負債的公允價值計量。詳情請分別參閱“附註2--收入”、“附註5--公允價值計量”和“附註6--股東權益”。
自我們在截至2023年12月31日止年度的10-k表格年度報告第7A項“關於市場風險的定量和定性披露”中披露以來,我們面臨的市場風險並未發生重大變化。
披露控制和程式的評估
截至2024年9月30日,管理層在首席執行官和首席財務官的監督和參與下,對我們的披露控制和程序(定義見《交易法》第13 a-15(e)條和第15 d-15(e)條)的設計和運作的有效性進行了評估。我們的披露控制和程序旨在提供合理的保證,即我們根據《交易法》提交或提交的報告中要求披露的信息在適用規則和表格指定的時間內被記錄、處理、總結和報告。根據該評估,我們的首席執行官兼首席財務官得出的結論是,截至2024年9月30日,由於下文描述的財務報告內部控制存在重大缺陷,我們的披露控制和程序並不有效。
鑑於下文所述的重大弱點,管理層進行了額外分析和其他程序,以確保我們的合併財務報表根據美國公認會計原則(美國GAAP)編制。因此,管理層認爲,本季度報告中包含的合併財務報表根據美國公認會計原則,在所有重大方面公平地反映了我們截至所列期間的財務狀況、經營業績和現金流量。
重大缺陷是財務報告內部控制的缺陷或缺陷的組合,使得我們的年度或中期財務報表的重大錯報有合理的可能性無法及時防止或發現。該公司發現,對基於股份的薪酬獎勵進行流程級控制的設計並不有效。這種設計不力的控制是由於對非常規股份支付獎勵的風險評估不足。這一控制缺陷導致相關賬目出現重大錯報。此外,上述控制缺陷造成了一種合理的可能性,即不能及時防止或發現合併財務報表的重大錯報。因此,我們得出的結論是,這一缺陷代表着公司財務報告內部控制的實質性弱點,我們對財務報告的內部控制無效。
管理’修復已確定的材料缺陷的計劃
公司將實施強化的風險評估程序,以確保所有非常規股票支付獎勵得到適當識別和評估。此外,公司將圍繞非常規股份支付獎勵設計額外的預防性控制,以確保此類獎勵得到適當的認可和衡量。管理層將定期向審計委員會報告補救計劃的進展和結果,包括內部控制缺陷的識別、狀態和解決方案。
我們預計,重大弱點將在2024年12月31日之前得到全面補救,但在更新的政策和培訓到位並運行足夠長的時間以使管理層和畢馬威會計師事務所能夠測試並得出控制措施的運營有效性之前,重大弱點不能被認爲已得到全面補救。
財務報告內部控制的變化
Other than the steps to remediate the material weakness discussed above, there were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) during the period ended September 30, 2024 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
We, and our subsidiaries, are not a party to, and our property is not the subject of, any material pending legal proceedings.
在投資我們的證券之前,您應仔細考慮以下風險因素和本季度報告其他地方包含的其他信息。如果發生以下任何風險,我們的業務、財務狀況或經營業績可能會受到重大損害。對我們的證券的投資本質上是投機性的,風險很高,不應該由無法無限期承擔投資經濟風險且無法承擔全部投資損失的投資者進行。以下描述的風險並不是我們業務面臨的唯一風險。我們目前未知或我們目前認爲不重大的其他風險可能會對我們未來的業務產生不利影響。投資者還應參閱本季度報告中包含或引用的其他信息,包括我們的財務報表和相關注釋,以及我們不時向SEC提交的其他文件。
與商業化相關的風險
如果我們獲得美國食品藥品監督管理局(FDA)或歐洲藥品管理局(EMA)對候選產品的批准,並且沒有在醫生、患者、醫療保健支付者和醫療界中獲得廣泛的市場接受,那麼我們從產品銷售中產生的收入將受到限制。
我們目前有一種已上市的產品BRIUMVI,該產品於2022年12月28日獲得FDA批准,用於治療復發型多發性硬化症(RMS),包括臨床孤立綜合徵、復發緩解疾病和成人活動性次級進展疾病。此外,BRIUMVI於2023年6月1日獲得歐盟委員會(EC)的批准,並於2023年晚些時候獲得藥品和保健產品監管局(MHRA)的批准,用於治療患有活動性疾病的成年RMS患者。歐盟和英國分別由臨床或影像特徵定義。
作爲一家商業公司,我們的經驗有限,並且我們成功克服與生物製藥行業藥物商業化相關的風險(包括我們的產品未達到足夠水平的風險)的能力仍然不確定。BRIUVI以及我們未來可能推向市場的其他藥物可能無法獲得醫生、患者、第三方付款人和醫療保健界其他人的市場接受。因此,我們可能無法產生大量收入或達到我們的收入預測或指導,並且可能無法盈利。BRIUMVI的市場接受程度以及我們可能獲得營銷批准的任何未來候選產品將取決於多種因素,包括:
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我們收到營銷批准的時間、此類批准的條款以及獲得此類批准的國家/地區; |
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臨床試驗中證明以及與替代治療相比的有效性、安全性和耐受性; |
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BRIUMVI和我們的任何候選產品以及競爭產品的市場引入時間; |
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我們產品獲得批准的適應症,以及此類產品批准標籤的其他方面; |
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醫生、高級從業者、神經病學診所的主要運營商和患者接受我們的產品作爲安全、可耐受和有效的治療方法; |
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與替代治療方法相比,我們的產品的潛在和感知的優點或缺點; |
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我們有能力以具有競爭力的價格出售我們的產品; |
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第三方付款人和政府當局是否能提供足夠的報銷; |
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患者費用分擔義務的程度,包括自付費用和免賠額; |
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政府當局對我們產品的監管要求發生變化; |
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相對方便和容易管理; |
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副作用和不良事件的發生率和嚴重程度; |
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目標患者人群嘗試新療法的意願以及醫生開出這些療法的意願; |
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我們以及任何當前或未來合作伙伴的銷售和營銷工作的有效性; |
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保護我們在智慧財產權組閤中的權利; |
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我們有能力維持可靠的產品供應以滿足市場需求;以及 |
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與我們的產品或與公司相關的有利或不利的宣傳。 |
此外,全球健康問題可能會影響BRIUMVI的商業化。患者和醫療保健提供者擔心抗CD 20抗體和其他b細胞靶向藥物等免疫抑制產品可能會增加感染病毒的風險或導致感染後更嚴重的併發症或後果,包括死亡。這些或其他類似的擔憂可能會影響BRIUMVI和我們正在開發的其他免疫抑制產品的商業潛力。
If BRIUMVI, or any future product candidates for which we receive regulatory approval, do not achieve an adequate level of acceptance by physicians, hospitals, healthcare payors and patients, we may not generate sufficient revenue from these products and we may not become or remain profitable, which would have a material adverse effect on our business.
We may be subject to limitations on the indicated uses or requirements to fulfill certain post-marketing requirements to the satisfaction of regulatory authorities or may be unable to maintain marketing approval for BRIUMVI or future products that we may bring to market.
Regulatory approvals for our product or any of our product candidates may be subject to conditions and limitations on the approved indicated uses for which the product may be marketed or contain requirements or commitments for potentially costly post-marketing testing, including Phase 4 clinical trials, and surveillance and pharmacovigilance to monitor the safety and efficacy of the approved product candidate. For example, with respect to the FDA’s approval of BRIUMVI for RMS, the approval is subject to certain post-marketing requirements and commitments, including long-term safety studies, as well as studies to evaluate the effects of BRIUMVI in pregnant women and pediatric populations, among others. Similar post-approval studies are required by other regulatory authorities outside of the U.S., including but not limited to, the EMA in the EU and the MHRA in the UK. These studies are highly specialized in their design and conduct and are associated with considerable expenses, and based on the outcome, could result in further labeling restrictions that could impair or restrict the way in which we are able to market BRIUMVI, or negatively impact its overall clinical profile. As of September 18, 2024, we announced updated and long-term data from the Open-Label Extension of our ULTIMATE I & II Phase 3 studies demonstrating a consistent safety profile, but the ultimate outcome of these and other studies remains uncertain.
In addition, with respect to BRIUMVI and any product candidate that the FDA or a comparable foreign regulatory authority approves, the manufacturing processes, testing, labeling, packaging, distribution, import, export, adverse event reporting, storage, advertising, promotion and recordkeeping for the product will be subject to extensive and ongoing regulatory requirements. These requirements include submissions of safety and other post-marketing information and reports, registration, as well as continued compliance with current Good Manufacturing Practices (cGMPs), with Good Clinical Practices (GCPs), for any clinical trials that we conduct post-approval, and with Good Laboratory Practices (GLPs) for any nonclinical studies. Later discovery of previously unknown problems with a product or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things, restrictions on the marketing or manufacturing of the product, withdrawal of the product from the market, mandatory safety labeling changes or product recalls, suspension or revocation of product approvals, product seizure or detention, refusal to permit the import or export of products, and injunctions or the imposition of civil or criminal penalties, all of which would adversely affect our business, prospects and ability to achieve or sustain profitability.
BRIUMVI以及我們未來獲得批准的任何候選產品在獲得批准後可能會被發現產生不良副作用,從而在商業化後可能會導致重大負面後果。
由於BRIUMVI或任何未來批准的產品在上市後使用更廣泛或更長的時間,臨床研究(包括驗證性或其他上市後研究)或不良事件報告或藥物警戒可能會產生數據,這可能會影響我們產品的商業潛力。例如,隨着更多患者在商業和臨床環境中接觸產品的時間更長,目前尚不清楚不良事件是否可能發生更高的頻率和/或嚴重程度,或者是否會繼續證明可接受的安全性和耐受性。如果我們或其他人發現BRIUMVI或RMS領域內的其他產品或候選產品在進入市場後引起了意外的副作用,則可能會導致一些潛在的重大負面後果,包括:
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監管部門可以撤銷批准或者限制批准的此類產品的使用適應症; |
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監管當局可要求添加新的或不同的標籤聲明,包括警告或方框警告、預防措施或禁忌症,這些可能會減少產品的使用或以其他方式限制受影響產品的商業成功; |
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我們可能被要求改變這些候選藥物的分配或給藥方式,或者進行額外的臨床試驗; |
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監管機構可能要求風險評估和緩解策略(REMS),這是一項降低風險的計劃,其中可能包括藥物指南、醫生溝通計劃或確保安全使用的要素,如受限分配方法、患者登記和其他風險最小化工具; |
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我們可能會受到監管調查和政府執法行動的影響; |
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我們可能會決定將這些候選藥物從市場上移除; |
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我們可能無法以可接受的條款簽訂合作協議並執行我們的商業模式; |
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我們可能會被起訴,並對接觸或服用我們產品的個人造成的傷害承擔責任;以及 |
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我們的聲譽可能會受到影響。 |
這些事件中的任何一個或一個組合都可能阻止我們維持監管批准以及實現或維持受影響產品的市場接受度,或者可能大幅增加受影響產品商業化的成本和費用,這反過來又可能會顯着影響我們成功商業化候選藥物和產生收入的能力。
BRIUMVI和我們的候選產品(包括非腫瘤適應症中的azer-cel)目標患者人群的發病率和患病率尚未精確確定。如果BRIUMVI和我們候選產品的市場機會小於我們的估計,或者如果我們獲得的任何批准是基於對患者人群的更窄定義,那麼我們的收入和實現盈利能力將受到不利影響。
RMS的確切發病率和/或患病率尚不清楚。我們對BRIMVI在RMS中的預測是基於估計以及我們當前對該疾病的了解和理解。這些估計通常基於與目標醫生和我們做出估計時可用的其他來源的一對一和群體互動,包括科學文獻、醫療保健利用數據庫和市場研究。儘管我們相信我們的估計是合理的,但許多因素可能會限制其準確性。例如,我們用於進行估計的來源可能被證明是不正確的。此外,新研究可能會改變這些疾病的估計發病率或患病率,受影響的患者數量可能會低於預期。
BRIUMVI和我們的候選產品的總體潛在市場機會如果獲得批准,最終取決於批准的處方信息、醫療界的接受程度、患者准入以及藥品定價和報銷等。主要市場的患者數量,包括這些市場中可解決的患者數量,可能會低於預期,患者可能無法接受我們的藥物治療,新患者可能變得越來越難以識別或獲得治療,患者和醫生可能會選擇使用有競爭力的產品或報銷可能不利,所有這些都將對我們的運營業績和業務產生不利影響。
我們面臨着巨大的競爭,這可能會導致其他人在我們之前或比我們更成功地商業化藥物,從而導致我們的商業機會減少或消除。
我們在生物技術和生物製藥市場競爭激烈的領域開展業務。我們面臨着來自衆多來源的競爭,包括商業製藥和生物技術企業、學術機構、政府機構以及私營和公共研究機構。我們的許多競爭對手擁有明顯更多的財務、產品開發、製造和商業化資源。大型製藥公司有豐富的產品商業化經驗,可能與客戶有重要的現有關係,並有更多的資源可供他們推廣其產品。許多人在與我們相同的疾病中表現活躍,包括在神經學和免疫學領域,有些人與我們直接競爭。我們還可能與這些組織在招聘商業和其他關鍵人員方面展開競爭。規模較小或處於初創階段的公司也可能成爲重要的競爭對手,特別是通過與大型和成熟公司的合作安排。
如果我們的競爭對手開發和商業化比我們或我們的合作者可能開發的任何藥物更有效、副作用更少或更不嚴重、更方便或定價或合同不同的藥物,我們的商業機會可能會減少或消失。我們的競爭對手也可能比我們更快地獲得FDA或其他監管機構對其藥品的批准,這可能會導致我們的競爭對手在我們或我們的合作者能夠進入市場之前建立強大的市場地位。在競爭環境中,根據法律、法規和指南,公司的通信也可能受到監管機構和競爭對手的嚴格審查,這些法律、法規和指南涉及促銷通信(廣告和促銷標籤)和非促銷通信(例如,某些教育和科學交流),以及根據聯邦法律(如《蘭漢姆法案》)和一致的州法律保護企業免受誤導性廣告或標籤的不公平競爭的潛在競爭對手行爲。
如果獲得批准,影響我們所有候選藥物成功的關鍵競爭因素可能是它們的功效、安全性、便利性、價格、仿製藥或生物仿製藥競爭水平以及政府和其他第三方付款人的報銷可用性。
製藥和生命科學行業正在快速發展,包括其他製藥技術和治療疾病方法的開發。這些發展可能會使我們的產品或候選產品過時或失去競爭力。與我們相比,我們的許多潛在競爭對手擁有更多:
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研發資源,包括人員和技術; |
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監管經驗; |
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藥物開發、臨床試驗和藥物商業化經驗; |
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利用知識產權的經驗和專業知識;以及 |
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資本資源。 |
我們還將在招聘和留住合格人員、建立臨床試驗地點、臨床試驗患者註冊以及識別和授權新產品和候選產品方面面臨這些第三方的競爭。
BRIUMVI以及我們未來能夠商業化的任何產品可能會受到不利的定價法規或第三方付款人保險和報銷政策的影響,這將損害我們的業務。
管理新藥的監管審批、定價和報銷的規定因國家而異。當前和未來的立法可能會大幅改變審批要求,這可能會涉及額外的成本,並導致獲得批准的延遲。一些國家要求藥品的銷售價格獲得批准後才能上市。在許多國家,定價審查期從批准上市後開始。在一些市場,處方藥定價即使在獲得初步批准後,仍然受到政府的持續控制。因此,我們可能會在特定國家/地區獲得產品的上市批准,但隨後會受到價格法規的約束,這些法規會推遲我們候選藥物的商業推出,可能會推遲很長一段時間,並對我們在該國家/地區銷售候選藥物所能產生的收入產生負面影響。不利的定價限制可能會阻礙我們收回在一個或多個產品上的投資的能力,即使我們的更多候選產品獲得了市場批准。有資格獲得報銷並不意味着任何藥物在所有情況下都會得到支付,或者支付的費率將涵蓋我們的成本,包括研究、開發、製造、銷售和分銷。如果適用,新藥的臨時報銷水平也可能不足以支付我們的成本,可能不會成爲永久性的。然而,一些第三方付款人可能仍然需要有文件證明患者符合某些資格標準才能獲得BRIUMVI的補償。
我們成功地將任何產品商業化的能力還將在一定程度上取決於政府當局、私人健康保險公司和其他組織爲我們的產品和相關治療提供保險和補償的程度。政府當局和第三方付款人,如私人健康保險公司和健康維護組織,決定他們將支付哪些藥物,並建立報銷和共同支付水平。美國醫療保健行業和其他地方的一個主要趨勢是成本控制。政府當局和第三方付款人試圖通過限制覆蓋範圍和限制特定藥品的報銷金額來控制成本。越來越多的第三方付款人要求製藥公司在標價的基礎上向他們提供預定的折扣,並對藥品的定價提出挑戰,除了檢查藥品的安全性和有效性外,還檢查藥品的成本效益。第三方商業付款人在設置自己的報銷政策時通常依賴於聯邦醫療保險覆蓋政策和支付限制。支付方可以限制某些產品的承保範圍,方法是使用僅涵蓋選定藥物的處方、使支付方不喜歡的藥物對患者來說更昂貴的可變共同支付,以及使用管理控制,例如要求事先授權或首先在另一種類型的治療中失敗。付款人可能會針對價格較高的藥物施加這些覆蓋障礙,因此我們的產品可能會受到付款人驅動的限制。此外,在患者可以獲得保險的國家,如美國,保險自付金額或其他福利限制可能會成爲獲得或繼續使用我們獲得監管批准的產品的障礙。如果我們無法獲得或維持保險,或者在一個或多個國家或地區的保險減少,我們的產品銷售額可能會低於預期,我們的財務狀況可能會受到損害。
Net prices for drugs may be reduced by mandatory discounts or rebates required by government healthcare programs or private payors and by any future relaxation of laws that presently restrict imports of drugs from countries where they may be sold at lower prices. In the United States, for example, we must offer discounted pricing or rebates on purchases of pharmaceutical products under various federal and state healthcare programs, such as the Medicaid Drug Rebate Program, the 340B drug pricing program and the Medicare Part D Program. We must also report specific prices to government agencies under healthcare programs, such as the Medicaid Drug Rebate Program and Medicare Part B. The calculations necessary to determine the prices reported are complex and the failure to report prices accurately may expose us to penalties.
If we are unable to expand our commercialization operations, we may not be successful in commercializing BRIUMVI or any product candidate, if and when such product candidates are approved, and we may not be able to generate revenue.
Commercialization of pharmaceutical products is an extremely complex and highly capital and resource-intensive process. Even for established companies with existing infrastructure and significantly greater resources than we have, challenges have occurred.
We have made and continue to make significant investments in our commercial organization and infrastructure. We built processes and systems to support the commercialization of BRIUMVI following its commercial launch on January 26, 2023. There are risks involved with establishing our own commercialization capabilities. For example, if we are unable to recruit and retain adequate numbers of effective personnel to support the ongoing commercialization of BRIUMVI, we may not be successful in marketing and selling the product.
Additional factors that may inhibit our efforts to commercialize BRIUMVI and our other product candidates on our own, or through partnership, and generate product revenues include:
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the costs and time associated with the initial and ongoing training of commercialization personnel on the applicable disease states, products, competitors, and legal and regulatory compliance matters; |
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the inability of commercialization personnel to obtain access to physicians or to effectively promote or provide education about BRIUMVI and any future approved products; |
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the lack of complementary drugs to be offered by the Company, which may put us at a competitive disadvantage relative to companies with more extensive product lines; |
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decisions by third-party payors to deny reimbursement of or delay coverage decisions regarding BRIUMVI or following approval of any product candidates; |
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our inability to maintain a healthcare compliance program including effective mechanisms for compliance monitoring; |
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our inability to establish and maintain commercial partnerships outside the U.S.; |
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our inability, or the inability of a third party with whom we have partnered, to maintain the necessary regulatory approvals required to operate in markets outside of the U.S.; |
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the timing of product availability for commercial sale following approval and continued product supply; and |
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unforeseen costs and expenses associated with creating a commercialization organization. |
In addition, we have entered into a commercialization agreement, and may enter into additional agreements in the future, that facilitate commercialization of BRIUMVI and/or future products that receive approval in markets outside the U.S. through partnerships. On February 26, 2024, our partner commercially launched BRIUMVI in Germany, the first European market in which BRIUMVI has launched. However, there are also risks with entering into these types of arrangements with third parties to perform sales, marketing and distribution services. For example, we may not be able to enter into such arrangements on terms that are favorable to us. Our drug revenues or the profitability of these drug revenues to us are likely to be lower than if we were to market and sell any products or product candidates that we develop ourselves. In addition, we likely will have little control over such third parties, and any of them may fail to devote the necessary resources and attention to sell and market our product or product candidates effectively. If we do not establish sales and marketing capabilities successfully, either on our own or in collaboration with third parties, we will not be successful in commercializing our drug candidates. Further, our business, results of operations, financial condition and prospects will be materially adversely affected.
We believe there is potential market opportunity for BRIUMVI outside of the U.S., including in the EU. We have entered into a commercialization agreement for the sale of BRIUMVI in certain territories outside the U.S., Canada and Mexico, the commercialization rights for which had been previously retained by TG, thus excluding certain Asian countries subject to previously existing partnerships, and we also may enter into certain collaboration and/or commercialization agreements with third parties in the future to facilitate market expansion. To the extent we do expand into other markets outside of the U.S. in which we are responsible for building and maintaining a commercial infrastructure, we expect to incur significant expenses in establishing an infrastructure to commercialize our drug products. Depending on the expenses incurred, it could have a negative impact on our cash resources.
Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any drug candidates that we may develop.
我們面臨着與人體臨床試驗中候選產品測試相關的產品責任暴露的固有風險,以及與BRIUMVI和我們未來可能獲得營銷授權的任何其他產品的商業化相關的更大風險。如果我們無法成功保護自己免受有關BRIUMVI或我們的任何候選產品造成傷害的指控,我們可能會承擔重大責任。無論優點或最終結果如何,責任索賠可能會導致:
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對我們可能商業化的任何產品的需求減少; |
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損害我們的聲譽和嚴重的負面媒體關注; |
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臨牀試驗參與者退出; |
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爲相關訴訟辯護的巨額成本,包括任何可能面臨此類相關訴訟的個人可能反過來尋求向我們索賠的風險; |
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向試驗參與者或患者提供大量金錢獎勵; |
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收入損失;以及 |
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無法將我們可能開發的任何產品或候選產品商業化。 |
儘管我們維持產品責任保險,但它可能不足以涵蓋我們可能承擔的所有責任。保險範圍越來越昂貴,而且難以獲得和維護。我們可能無法以合理的成本或足以滿足可能產生的任何責任的金額維持保險範圍。
我們與政府實體簽訂的任何合同都可能涉及未來的資金和合規風險。
我們與政府實體簽訂的任何合同都可能涉及未來的資金和合規風險。與政府實體簽訂的此類合同通常會受到缺乏資金和遵守獨特要求等風險的影響。例如,政府合同採購義務通常取決於資金的可獲得性,這可能會被取消或減少。此外,政府客戶未來購買的產品或服務的數量往往是不確定的。我們的任何政府合同可能不會續簽,或者可能會爲了方便而終止,而事先幾乎沒有通知。與政府實體簽訂的合同通常要遵守採購法,其中包括社會經濟影響、就業做法、環境保護、記錄和會計義務以及其他要求。這些合同和法律要求可能會使我們的業務複雜化,並增加我們的合規負擔。任何此類風險的發生都可能損害我們的聲譽,並可能對我們的業務運營、財務狀況和/或運營結果產生重大不利影響。
與我們的財務狀況和額外資本需求相關的風險
自成立以來,我們已經出現了重大的運營虧損,未來我們可能會出現虧損。
生物製藥藥物開發是一項高度投機的事業,涉及很大程度的風險。我們於2012年1月開始運營。迄今爲止,我們的業務主要限於組織和配備我們的公司、業務規劃、籌集資金、開發我們的技術、識別潛在的候選藥物、進行臨床前研究和臨床試驗、將UKONIQ商業化(退出銷售)以及推出和商業化BRUMVI。我們正在從一家專注於腫瘤學研發和商業化能力的公司轉型爲一家能夠支持美國國內外神經學和免疫學商業活動的公司。這種轉型涉及多種風險,我們可能不會成功這樣的轉型。
自成立以來,我們一直將我們的努力和財力集中在臨床試驗、我們的產品和候選產品的製造、建立商業基礎設施和準備支持商業產品上。到目前爲止,我們主要通過公開發行普通股和債務融資來爲我們的運營提供資金。自成立以來,我們發生了重大的運營虧損。我們幾乎所有的運營虧損都來自與我們的研發計劃相關的成本,以及與我們的運營相關的銷售、一般和管理成本,包括我們的商業化活動。BRIUMVI是我們目前唯一上市的產品。隨着我們繼續將BRIUMVI商業化,我們預計將繼續產生大量的研發費用,以及大量的商業化和外包製造費用。由於與藥品開發和商業化相關的衆多風險和不確定性,我們無法預測未來虧損的程度,也無法預測我們可能在多長時間內繼續實現淨利潤。我們可能無法維持或提高我們的季度或年度盈利能力。我們盈利的能力取決於我們創造可觀收入的能力。我們之前的虧損已經並將繼續對我們的股東赤字和營運資本產生不利影響,如果我們無法在未來期間保持盈利的話。
爲了實現並保持盈利,我們必須成功開發(或授權)我們的產品或候選產品並商業化,並繼續成功商業化BRIUVI。目前尚不確定我們未來何時以及是否會從銷售我們的產品或任何候選產品(如果獲得批准)中產生或繼續產生任何重大收入。此外,如果獲得批准,我們無法保證我們將滿足有關BRIUVI或我們候選產品的收入預測或指導。爲了獲得可觀且持續的收入並滿足我們的收入預測或指導,我們必須單獨或與其他人一起成功:(i)獲得和維持對我們的產品和候選產品的監管批准;以及(ii)製造、營銷和銷售我們的產品和候選產品。我們產生持續收入的能力取決於多種因素,包括但不限於我們:
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successfully complete clinical trials that meet their clinical endpoints; |
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initiate and successfully complete all safety, pharmacokinetic, biodistribution, and non-clinical studies required to obtain U.S. and foreign marketing approval for our product and product candidates; |
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obtain approval from the FDA and foreign equivalents to market and sell our product and product candidates, and maintain FDA, MHRA and EMA approvals of BRIUMVI for RMS; |
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establish and maintain commercial manufacturing capabilities with third parties that are satisfactory to the regulatory authorities, cost effective, and that are capable of providing commercial supply of our product and product candidates; |
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expand on our commercialization infrastructure to commercialize BRIUMVI, and/or entering into collaborations with third parties; |
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achieve market acceptance of BRIUMVI and any other products for which we may receive regulatory approval in the medical community and with third-party payors. |
If we are unable to generate significant and sustained revenues, we will not become or remain profitable and we will be unable to continue our operations without continued funding.
While we do not expect to need to raise additional capital, we may need to do so. If we are unable to raise capital, if needed, we may be required to delay, limit, reduce or eliminate some of our drug development programs or commercialization efforts.
The development of pharmaceuticals is capital-intensive. We are also continuing to generate additional clinical data for BRIUMVI to support and potentially expand commercial adoption, including assessing long-term tolerability in our Open-Label Extension of the Phase 3 ULTIMATE I and II trials and Phase 4 clinical studies necessary to satisfy post-approval commitments for regulatory authorities or those undertaken voluntarily by the Company to evaluate the use of BRIUMVI in alternate settings or with alternate methods of administration. Moreover, now that we have launched BRIUMVI, we will need to expend substantial resources on maintaining approvals and continuing commercialization, manufacturing and distribution over the foreseeable future. Additionally, we expect to commence a trial evaluating azer-cel in autoimmune disease in 2024.
The amount and timing of our future funding requirements will depend on many factors, including, but not limited to, the following:
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the success of the commercialization of BRIUMVI and any other products for which we receive regulatory approval; |
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the costs and timing of clinical and commercial manufacturing supply arrangements for each product and product candidate; |
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the costs of expanding our sales, distribution, and other commercialization capabilities; |
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the costs and timing of regulatory approvals; |
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the progress of our clinical trials, including expenses to support the trials and milestone payments that may become payable under our license agreements; |
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our ability to establish and maintain strategic collaborations, including licensing and other arrangements; |
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the costs involved in enforcing or defending patent claims or other intellectual property rights; and |
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the extent to which we in-license or invest in other indications or product candidates. |
As a result, significant additional funding may be required. Additional sources of financing to continue our operations in the future might not be available on favorable terms, if at all. If we do not succeed in raising additional funds on acceptable terms, we could be forced to discontinue product development, reduce or forego commercialization efforts that are required for successful commercialization of BRIUMVI or any of our product candidates and otherwise forego attractive business opportunities. Any additional sources of financing may involve the issuance of our equity securities, which would have a dilutive effect to stockholders. Currently, other than BRIUMVI, our products are investigational and have not been approved by the FDA or any foreign regulatory authority for sale. For the foreseeable future, we will have to fund all our operations and capital expenditures from sales of BRIUMVI, cash on hand and amounts raised in future offerings or financings. Accordingly, our prospects must be considered in light of the uncertainties, risks, expenses and difficulties frequently encountered by companies in the early stages of commercial operations and the competitive environment in which we operate.
Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or drug candidates and occupy valuable management time and resources.
Until such time, if ever, as we can generate substantial revenues, we expect to finance our cash needs through a combination of public and private equity offerings, debt financings, collaborations, strategic alliances, licensing agreements or other arrangements. We do not have any committed external source of funds, other than funds already borrowed under the Financing Agreement that we entered into with Blue Owl Capital Corporation, HealthCare Royalty and Blue Owl Capital in August 2024 (see Note 7 to our condensed consolidated financial statements for more information). To the extent that we raise additional capital through the sale of common stock or securities convertible or exchangeable into common stock, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that materially adversely affect the rights of our common stockholders. We may also seek funds through collaborations, strategic alliances or licensing arrangements with third parties at a time that is not desirable to us and we may be required to relinquish valuable rights to some intellectual property, future revenue streams, research programs or products and product candidates or to grant licenses on terms that may not be favorable to us, any of which may have a material adverse effect on our business, operating results and prospects. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. We cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to us, if at all, which could limit our ability to expand our business operations and could harm our overall business prospects.
此外,籌款活動可能會分散我們管理層的日常活動,這可能會對我們開發和商業化候選藥物的能力產生不利影響。金融市場的混亂通常使股權和債務融資變得更加困難,並可能對我們滿足融資需求的能力產生重大不利影響。此外,我們發行額外證券(無論是股權還是債務),或此類發行的可能性,可能會導致我們股票的市場價格下跌。
由於資源有限,我們可能無法利用可能帶來更大商業機會或更有可能成功的計劃或候選產品。
由於我們的資源有限,我們可能會放棄或推遲對某些計劃或候選產品或後來被證明具有更大商業潛力的指示的機會的追求。我們對候選產品潛在市場的估計可能不準確,我們在當前和未來研發計劃上的支出可能不會產生任何商業上可行的產品。如果我們沒有準確評估特定候選產品的商業潛力,我們可能會通過戰略合作、許可、銷售或其他安排放棄對該候選產品有價值的權利,而在這種情況下,保留該候選產品的獨家開發權和商業化權利對我們更有利。我們可能會將我們的努力和資源集中在潛在的候選產品或其他最終被證明不成功的潛在計劃上。或者,我們可以將內部資源分配給某個治療領域的候選產品,在該領域中,達成合作安排會更有利。
無法保證我們能夠爲我們的候選產品找到額外的治療機會,或通過內部研究計劃開發合適的潛在候選產品,這可能會對我們未來的增長和前景產生重大不利影響。如果發生上述任何事件,我們可能會被迫放棄或推遲針對特定候選產品的開發工作,或者無法開發出潛在成功的候選產品,這可能會對我們的業務、財務狀況、運營業績和前景產生重大不利影響。
我們的負債水平和償債義務可能會對我們的財務狀況產生不利影響,並可能使我們更難爲我們的運營提供資金。
2019年2月,我們與Hercules Capital,Inc.簽訂了貸款和擔保協議,一家馬里蘭州公司(Hercules),於2021年12月30日,該公司與Hercules簽訂了經修訂和重述的貸款和擔保協議(經修訂的貸款協議),該協議於2023年3月31日進行了修訂。2024年8月2日,我們償還了經修訂的經修訂貸款協議項下的所有未償還本金和應計利息和費用,經修訂的貸款協議已有效終止。
2024年8月2日,該公司根據融資協議(定義如下)與Blue Owl Capital Corporation(作爲行政代理人)、HealthCare Royalty和Blue Owl Capital簽訂了價值25000加元的定期貸款融資(初始定期貸款)。
初始定期貸款受日期爲2024年8月2日的融資協議(融資協議)管轄,該協議規定:(i)在截止日期單次提取初始定期貸款;(ii)本金總額爲10000美元的未承諾額外融資(看見 更多信息請參閱我們的簡明綜合財務報表附註7)。
融資協議項下的所有義務均以作爲擔保人的公司和我們的某些子公司幾乎所有資產的優先權爲擔保。該債務可能會給我們帶來額外的融資風險,特別是如果我們的業務或當前的金融市場狀況不利於償還或爲其到期未償債務進行再融資。這種債務還可能產生重要的負面後果,包括:
如果額外債務增加到我們當前的債務水平上,上述風險可能會增加,包括以以下方式增加:
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我們將需要通過支付利息和本金來償還債務,這將減少可用於資助我們的運營、研發工作和其他一般企業活動的資金;和 |
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我們未能遵守融資協議中的限制性契約可能會導致違約事件,如果不糾正或豁免,將加速我們償還該債務的義務,而融資協議下的債權人可以尋求強制執行其在擔保此類債務的資產中的擔保權益。 |
如果額外債務增加到我們當前的債務水平上,上述風險可能會增加。
我們可能沒有足夠的可用現金來支付到期債務的利息或本金。
Failure to satisfy our current and future debt obligations under the Financing Agreement, or the breach of any of its covenants, subject to specified cure periods with respect to certain breaches, could result in an event of default and, as a result, Blue Owl Capital and HealthCare Royalty could accelerate all the amounts due. In the event of an acceleration of amounts due under the Financing Agreement, as a result of an event of default, we may not have enough available cash or be able to raise additional funds through equity or debt financings to repay such indebtedness at the time of such acceleration. In that case, we may be required to delay, limit, reduce or terminate our product candidate development or commercialization efforts or grant to others’ rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves. Blue Owl Capital Corporation could also exercise their rights as the Administrative Agent to take possession and dispose of the collateral securing the term loan for its benefit, which collateral includes substantially all of our assets and certain of our subsidiaries as guarantors. Our business, financial condition and results of operations could be materially adversely affected as a result of any of these events.
In addition, the Financing Agreement imposes operating and other restrictions on the Company. Such restrictions will affect, and in many respects limit or prohibit, our ability and the ability of any future subsidiary to, among other things (subject to the exceptions provided for in the Financing Agreement):
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dispose of certain assets; |
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change its lines of business; |
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engage in mergers, acquisitions, joint ventures or consolidations; |
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incur additional indebtedness; |
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create liens on assets; |
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pay dividends and make contributions or repurchase our capital stock; and |
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engage in certain transactions with affiliates. |
The breach of any of these restrictive covenants could have a material adverse effect on our business and prospects.
Our cash and cash equivalents could be adversely affected if the financial institutions in which we hold our cash and cash equivalents fail.
On March 10, 2023, the Federal Deposit Insurance Corporation (FDIC) announced that Silicon Valley Bank had been closed by the California Department of Financial Protection and Innovation, and on March 12, 2023, Signature Bank was closed by the New York State Department of Financial Services, and the FDIC was named receiver. Although we did not maintain any bank accounts with Silicon Valley Bank or Signature Bank, we regularly maintain cash balances at third-party financial institutions in excess of the FDIC insurance limit. Any failure of a depository institution to return any of our deposits, or any other adverse conditions in the financial or credit markets affecting depository institutions, could impact access to our invested cash or cash equivalents and could adversely impact our operating liquidity and financial performance.
與藥物開發和監管批准相關的風險
如果我們無法維持或獲得對我們的產品和候選產品的監管批准,並且最終無法成功將我們的產品或候選產品商業化,或者在這樣做時遇到重大延誤,我們的業務將受到重大損害。
我們從產品銷售中產生收入的能力將在很大程度上取決於BRIUVI的成功商業化。我們的每個候選產品都需要額外的非臨床或臨床開發、監管機構批准以及足夠的臨床和商業供應。我們開發計劃的成功以及候選產品獲得監管批准將取決於幾個因素,其中包括以下因素:
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成功完成我們的臨床項目並取得積極的結果,支持在我們預測的時間範圍內發現我們的候選產品在目標人群中的有效性和可接受的安全性特徵; |
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研究性新藥申請(IND)和臨床試驗申請(MTA)已獲得批准/發佈/批准,以便我們的候選產品可以開始臨床試驗; |
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成功啓動和完成臨床前研究以及成功啓動、入組和完成臨床試驗; |
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我們有足夠的財政和其他資源來完成必要的臨牀前研究和臨牀試驗; |
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收到適用監管機構對我們候選產品的監管批准; |
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與第三方製造商建立商業上可行的臨床供應和商業製造安排;以及 |
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爲我們的候選產品獲得並維護專利和商業祕密保護或監管排他性。 |
如果我們沒有及時或根本沒有實現其中一個或多個因素,我們的臨床項目和監管提交時間表可能會出現嚴重延遲,並且可能無法獲得監管機構對我們候選產品的批准。
由於臨床前研究和早期臨床試驗的結果不一定能預測未來的結果,因此我們開發的任何候選產品可能不會在後期的臨床試驗中取得有利的結果或獲得監管機構的批准。此外,臨時的, “頂線,” 隨着更多患者數據或其他終點(包括功效和安全性)的分析,我們宣佈或發佈的臨床試驗的初步數據可能會發生變化,或者感知的產品概況可能會受到負面影響。
醫藥開發具有內在的風險。臨床前開發試驗和早期臨床試驗的結果可能不能預測後來臨床試驗的結果,臨床試驗的中期結果也不一定能預測最終結果。此外,臨床前和臨床數據往往容易受到不同解釋和分析的影響,許多公司認爲他們的候選產品在臨床前研究中表現令人滿意,儘管如此,臨床試驗仍未能獲得上市批准。一旦候選產品顯示了足夠的臨床前數據以保證臨床研究,我們將被要求通過充分和良好控制的臨床試驗證明我們的候選產品是有效的,具有良好的益處-風險概況,用於其目標適應症的人群,然後我們才能尋求監管機構對其商業銷售的批准。許多候選藥物在臨床開發的早期階段因安全性和耐受性問題或臨床活性不足而失敗,儘管臨床前結果很有希望。因此,不能保證可以爲這些化合物找到安全有效的劑量,或者它們是否會單獨或與其他候選產品一起進入高級或關鍵的臨床試驗。此外,早期臨床試驗的成功並不意味着後來的臨床試驗也會成功,因爲後期臨床試驗中的候選產品可能無法證明足夠的安全性或有效性,儘管已經通過了初步臨床測試。公司經常在高級臨床試驗中遭遇重大挫折,即使在早期的臨床試驗顯示有希望的結果之後也是如此。通過臨床試驗的候選藥物的失敗率極高。
臨床試驗中接受治療的患者的個體報告結果可能無法代表此類研究中接受治療的患者的整個人群。此外,與早期研究相比,通常在國際上進行的更大規模的第三階段研究本質上面臨着更大的操作風險,包括結果可能因地區與地區或國家與國家而異的風險,這可能會對研究結果或適用監管機構對研究結果有效性的看法產生重大不利影響。
我們可能會不時公開披露我們的臨床試驗的主要或初步數據,這些數據基於對現有數據的初步分析,結果和相關的發現和結論可能會在對與特定研究或試驗相關的數據進行更全面的審查後發生變化。在分析此類數據時,我們也會做出假設、估計、計算和結論,我們可能沒有收到或沒有機會全面和仔細地評估來自特定研究或試驗的所有數據,例如後來的數據,包括所有終點和安全數據。因此,我們報告的主要或初步結果可能與相同研究的未來結果不同,或者一旦收到更多數據並進行充分評估,不同的結論或考慮因素可能會使這些結果合格。頂線或初步數據仍需接受審計和核實程序,這可能會導致最終數據與我們之前公佈的背線、中期或初步數據存在實質性差異。在提供頂線結果時,我們可能會在所有次要終點完全分析之前披露研究的主要終點。正的主端點不會轉換爲滿足所有或任何次要端點。因此,在最終數據可用之前,應謹慎查看主要數據和初步數據,包括來自全面安全分析和所有終端的最終分析的數據。
Further, from time to time, we may also disclose interim data from our preclinical studies and clinical trials. Interim data from clinical trials that we may complete are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and more patient data become available. For example, time-to-event based endpoints such as duration of response (DOR) and progression-free survival (PFS), and continuously observed data such as annualized relapse rate (ARR) have the potential to change with longer follow-up. In addition, as patients continue on therapy, there can be no assurance that the final safety data from studies, once fully analyzed, will be consistent with prior safety data presented, will be differentiated from other similar agents in the same class, will support continued development, or will be favorable enough to support regulatory approvals for the indications studied. Further, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the value of the particular program, the approvability or commercialization of the particular product candidate or product and our company in general. The information we choose to publicly disclose regarding a particular study or clinical trial is based on what is typically extensive information, and regulators or others may not agree with what we determine is material or otherwise appropriate information to include in our disclosure. If the interim, top-line or preliminary data that we report differ from final results, or if others, including regulatory authorities, disagree with the conclusions we have reached, our ability to obtain approval for, or successfully commercialize, our product or product candidates may be harmed, which could harm our business, operating results, prospects or financial condition.
Many of the results reported in our early clinical trials rely on local investigator-assessed efficacy outcomes which may be subject to greater variability or subjectivity than results assessed in a blinded, independent, centrally reviewed manner, often required of later phase, adequate and well-controlled registration-directed clinical trials. If the results from our registration-directed trials are different from the results found in the earlier studies, we may need to terminate or revise our clinical development plan, which could extend the time for conducting our development program and could have a material adverse effect on our business.
Clinical drug development involves a lengthy and expensive process, with an uncertain outcome. We may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates.
Before obtaining marketing approval from regulatory authorities for the sale of any product candidate, we must complete preclinical studies and then conduct extensive clinical trials to demonstrate the safety and efficacy of our product candidates in humans. Clinical testing is expensive, is difficult to design and implement, can take many years to complete and is uncertain as to outcome. It is impossible to predict when or if our product candidates will prove effective and safe in humans, will receive regulatory approval or will have a differentiated safety and tolerability profile. A failure of one or more clinical trials can occur at any stage of testing. Accordingly, our ongoing trials and future clinical trials may not be successful. Even if our clinical trials produce positive results, there can be no guarantee that the positive outcomes will be replicated in future studies either within the same indication as previously evaluated or in alternate indications and settings.
Successful completion of our clinical trials is a prerequisite to submitting a New Drug Application (NDA) or a Biologics License Application (BLA) to the FDA and a Marketing Authorization Application (MAA) to the EMA for each product candidate and, consequently, the ultimate approval and commercial marketing of our product candidates. We do not know whether any of our ongoing or future clinical trials for our product candidates will be completed on schedule, if at all.
Whether or not and how quickly we complete clinical trials depends in part upon the rate at which we are able to engage clinical research/trial sites and, thereafter, the rate of enrollment of patients, and the rate at which we collect, clean, lock and analyze the clinical trial database. Patient enrollment is a function of many factors, including the size of the patient population, the proximity of patients to clinical sites, the eligibility criteria for the study, the existence of competitive clinical trials, and whether existing or new drugs are approved for the indication we are studying. We are aware that other companies are currently conducting or planning clinical trials that seek to enroll patients with the same diseases that we are studying. We may experience unforeseen events that could delay or prevent our ability to complete current clinical trials, initiate new trials, receive marketing approval or commercialize our product candidates, including:
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the FDA or other regulatory authorities may require us to submit additional data or impose other requirements before permitting us to initiate a clinical trial; |
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the FDA or other regulatory authorities or institutional review boards (IRBs) or ethics committees (ECs) may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site or in a country; we may experience delays in reaching, or fail to reach, agreement on acceptable terms with prospective trial sites and prospective clinical research organizations (CROs), the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; |
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clinical trials of our drug candidates may produce negative or inconclusive results, and we may decide, or regulatory authorities may require us, to conduct additional preclinical studies or clinical trials or we may decide to abandon drug development programs; |
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the number of patients required for clinical trials of our drug candidates may be larger than we anticipate, and enrollment in these clinical trials may be slower than we anticipate or participants may drop out of these clinical trials or fail to return for post-treatment follow-up at a higher rate than we anticipate; |
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our third-party contractors, including our clinical trial sites, may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all, or may deviate from the clinical trial protocol or drop out of the trial, which may require that we add new clinical trial sites or investigators; |
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we may elect to or regulatory authorities or IRBs or ECs may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks; |
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the cost of clinical trials of our product candidates may be greater than we anticipate; |
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the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate, including, without limitation, as a result of disruptions to our supply chains caused by global health crises international conflicts such as the Russian invasion of Ukraine or the Israel-Hamas war, economic instability, or natural disasters; |
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regulatory authorities may revise the requirements applicable to our product candidates, or such requirements may not be as we anticipate; and |
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our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulatory authorities, IRBs or ECs to suspend or terminate the trials, or reports may arise from preclinical or clinical testing of other therapies in the same or a similar class that raise safety or efficacy concerns about our product candidates. |
We also could encounter delays if a clinical trial is suspended or terminated by us, by the IRBs of the institutions in which such trials are being conducted, by the Data and Safety Monitoring Board (DSMB) for such trial or by the FDA or other regulatory authorities. Such regulatory authorities may impose a clinical hold, suspension or termination due to a number of factors, including failure to conduct the clinical trial in accordance with regulatory requirements or our clinical protocols, inspection of the clinical trial operations or trial site by the FDA or other regulatory authorities, unforeseen safety issues or adverse side effects, failure to demonstrate a benefit from using a drug, changes in governmental regulations or administrative actions or lack of adequate funding to continue the clinical trial. In addition to the FDA, the IRB and/or the DSMB for our clinical trials may recommend modification to the study design or closure of the study entirely based on the IRB's and/or DSMB’s interpretation of the benefit-risk of the study. While we develop charters that guide the nature of the IRB and DSMB meetings, their analysis and interpretation of study data occurs independently from us and is wholly within their control. Even if the IRB or DSMB finds no safety concerns and recommends no modifications to the ongoing study, this does not mean the safety profile reported in the study may support a marketing approval or commercial acceptance if marketing approval is granted. Many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates.
Negative or inconclusive results from the clinical trials we conduct, unanticipated adverse medical events, or changes in regulatory policy could cause us to have to delay, repeat or terminate the clinical trials. If we are required to repeat or conduct additional clinical trials or other testing of our drug candidates beyond those that we currently contemplate, if we are unable to successfully complete clinical trials of our drug candidates or other testing, if the results of these trials or tests are not positive or are only modestly positive or if there are safety concerns, we may:
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be delayed in obtaining marketing approval for our product candidates; |
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not obtain marketing approval at all; |
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obtain marketing approval in some countries and not others; |
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obtain approval for indications or patient populations that are not as broad as intended or desired; |
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be subject to post-marketing requirements or post-marketing commitments; |
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be subject to increased pricing pressure; or |
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have the drug removed from the market after obtaining marketing approval. |
In addition, changes in regulatory policy could cause us to have to repeat or conduct additional clinical trials or change our clinical development strategy. For example, in December 2022, with the passage of Food and Drug Omnibus Reform Act, Congress required sponsors to develop and submit a diversity action plan for each Phase 3 clinical trial or any other “pivotal study” of a new drug or biological product. These plans are meant to encourage the enrollment of more diverse patient populations in late-stage clinical trials of FDA-regulated products. If we are not able to adhere to these new requirements, our ability to conduct clinical trials may be delayed or halted. Our drug development costs will also increase if we experience delays in testing or regulatory approvals. Certain clinical trials are designed to continue until a pre-determined number of events have occurred in the patients enrolled. Trials such as this are subject to delays stemming from patient withdrawal and from lower-than-expected event rates. Significant clinical trial delays could also shorten any periods during which we may have the exclusive right to commercialize our product candidates or allow our competitors to bring products to market before we do and impair our ability to successfully commercialize our product candidates. Any delays in our preclinical or future clinical development programs may harm our business, financial condition and prospects significantly. We may also incur additional costs if enrollment is increased.
In addition, principal investigators for our clinical trials may serve as scientific advisors or consultants to us from time to time and receive compensation in connection with such services. If these relationships and any related compensation result in perceived or actual conflicts of interest, the integrity of the data generated at the applicable clinical trial site or the FDA’s acceptance of such data, may be jeopardized.
Biologics carry unique risks and uncertainties, which could have a negative impact on our business.
The successful development, manufacturing and sale of biologics is a long, expensive and uncertain process. There are unique risks and uncertainties with biologics. For example, access to and supply of necessary biological materials, such as cell lines, may be limited, and governmental regulations restrict access to and regulate the transport and use of such materials. In addition, the development, manufacturing and sale of biologics is subject to regulations that are often more complex and extensive than the regulations applicable to other pharmaceutical products. Manufacturing biologics, especially in large quantities, is often complex and may require the use of innovative technologies. Such manufacturing also requires facilities specifically designed and validated for this purpose and sophisticated quality assurance and quality control procedures. Biologics are also frequently costly to manufacture. Failure to successfully develop, manufacture and sell BRIUMVI or other biological product candidates we may develop could adversely affect our business.
Our product or product candidates may cause undesirable side effects that could delay or prevent their regulatory approval or impact their availability and commercial potential after approval.
Unexpected or undesirable adverse events caused by BRIUMVI or any of our product candidates that we take into clinical trials could cause either a DSMB or regulatory authorities to interrupt, delay, modify or suspend clinical trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA or other regulatory authorities. Even if a product candidate has obtained marketing approval, undesirable side effects may inhibit market acceptance of the approved product due to its tolerability versus other therapies. This could prevent us from commercializing the affected product candidate and generating revenues from its sale.
As is the case with all drugs, it is likely that there will be side effects associated with the use of our drug candidates. Results of our trials could reveal a higher than expected and unacceptable severity and prevalence of side effects. In such an event, our trials could be suspended or terminated and the FDA or comparable foreign regulatory authorities could order us to discontinue an ongoing trial or deny approval of our drug candidates for any or all targeted indications. The drug-related side effects could also affect patient recruitment or the ability of enrolled patients to complete the trial or result in potential product liability claims. In addition, data may emerge, from confirmatory or other post-marketing studies, or from pharmacovigilance reporting, as products are used more widely, or for a longer duration, after approval that may affect the commercial potential of our products. Any of these occurrences may harm our business, financial condition and prospects significantly.
Many compounds that initially showed promise in early-stage testing have later been found to cause side effects that prevented further development of the compound. Further, early clinical trials by their nature utilize a small sample of the potential patient population. With a limited number of patients and limited duration of exposure, rare and serious side effects of our drug candidates may only be uncovered when a significantly larger number of patients are exposed to the drug candidate in Phase 3 or registration-directed trials or when the drug candidate is on the market. If any of our product candidates cause unacceptable adverse events in clinical trials, we may not be able to obtain marketing approval and generate revenues from its sale, or even if approved for sale may lack differentiation from competitive products, which could have a material adverse impact on our business and operations. Further, clinical trials by their nature utilize a sample of the potential patient population. With a limited number of patients, rare and severe side effects of BRIUMVI or our other product candidates may only be uncovered with a significantly larger number of patients exposed to the product.
Any products or product candidates we may advance through clinical development are subject to extensive regulation, which can be costly and time consuming, cause unanticipated delays or prevent the receipt of the required approvals.
The clinical development, manufacturing, labeling, packaging, storage, record-keeping, advertising, promotion, import, export, marketing and distribution, and pharmacovigilance and adverse event reporting of our product or product candidates or any future product candidates are subject to extensive regulation by the FDA in the United States and by comparable regulatory authorities worldwide. In the United States, we are not permitted to market a new product candidate until we receive approval of a BLA or NDA from the FDA. The process of obtaining a BLA or NDA approval is expensive, often takes many years, and can vary substantially based upon the type, complexity and novelty of the products involved. In addition, approval policies or regulations may change over time. If we fail to gain approval to commercialize our product candidates from the FDA and other foreign regulatory authorities in the timelines we project or at all, we may be unable to generate the revenues that we may project or generate revenues at levels sufficient to sustain our business.
The FDA and foreign regulatory authorities exercise extensive control over the pharmaceutical product approval process, including substantial discretion to delay, limit or deny approval of a product candidate for many reasons. During the regulatory review process, the FDA or other regulatory authorities may disagree with or not accept our clinical trial design, may have questions about the potential impact of our study design on conclusions that can be drawn from the data, may interpret results differently than we do, may apply the results of our trials in one disease to the review of a regulatory application for a different disease even if the doses and therapeutic areas are distinct, and may change its view on the criteria that must be met for approval. This could happen even for a protocol used to support a trial that is subject to a Special Protocol Assessment (SPA) agreement with the FDA. There is no guarantee that the FDA will not delay, limit or deny approval of our product candidates in the future.
Furthermore, some of our clinical trials may be conducted as open-label studies, meaning that trial participants, investigators, site staff, some employees of our CROs, and our field-level employees (e.g., clinical research associates and monitors), among others, have knowledge of treatment arm assignments on a patient-level, which has the potential to introduce bias into study conduct. Further, even when our clinical trials are double-blind, double-dummy studies, unblinding of treatment arm assignment may occur from time to time, for example, on the occurrence of unexpected safety events which may necessitate understanding of study treatment. While we believe we have put in place adequate firewalls to prevent inappropriate unblinding of study data consistent with standard industry practice for these types of studies, no assurance can be given that issues related to study conduct will not be raised. The FDA may raise issues of safety, study conduct, bias, deviation from the protocol, statistical power, patient completion rates, changes in scientific or medical parameters or internal inconsistencies in the study design or data prior to making its final decision. The FDA may also seek the guidance of an outside advisory committee in evaluating (among other things) clinical data and safety and effectiveness considerations prior to making its final decision. These issues could cause a delay in the FDA’s review, lead the FDA to deny approval, or lead the Company to withdraw a regulatory application.
Other reasons that the FDA or regulatory authorities around the world may delay, limit or deny approval of a product candidate, include:
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we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a product candidate is tolerable and effective for an indication; |
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the FDA may not accept clinical data from trials conducted by individual investigators or in countries where the standard of care or the patient population, is potentially different from that of the United States; |
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the results of clinical trials may not meet the level of statistical significance required by the FDA or comparable foreign regulatory authorities for approval; |
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we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks; |
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the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data from preclinical studies and/or clinical trials; |
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the data collected from clinical trials of our product candidates may not be sufficient to support the submission of a BLA, NDA or other marketing authorization submission to obtain regulatory approval in the United States or elsewhere; |
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the FDA or comparable foreign regulatory authorities may identify issues related to the manufacturing processes or facilities of third-party manufacturers with which we or our collaborators currently contract for clinical supplies and plan to contract for commercial supplies; during the course of review, the FDA or foreign regulatory authorities may raise issues and request or require additional preclinical, clinical, chemistry, manufacturing, and control (CMC), or other data and information, and the development and provision of these data and information may be time consuming. We may not be able to generate the data within the time period necessary to obtain approval within the established regulatory review timelines, such as by a Prescription Drug User Fee Act (PDUFA) goal date or at all to satisfy the FDA or foreign regulatory authorities; |
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the approval processes of the FDA or comparable foreign regulatory authorities may significantly change in a manner rendering our clinical data insufficient for approval; or |
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interruptions or delays in the operations of the FDA and foreign regulatory authorities as a result of global health, inadequate government funding, or economic crises international conflict, or national disasters may negatively impact review, inspection, and approval timelines. |
Even if we succeed in obtaining regulatory approval for a product candidate, the FDA may require, or we may commit to, post-marketing studies, including additional clinical trials such as those necessary to assess drug interactions or activity of a product in specific populations, which may be costly. The outcomes of post-marketing studies may impact product labeling and therefore, there can be no guarantee that the product attributes contained in the initial prescribing information will be maintained as future studies produce data. This includes, without limitation, additional results from studies evaluating drug-drug interactions and patients with certain comorbidities that may restrict the use of an approved product in select populations or introduce dose modifications or contraindicated concomitant medications that have the potential to impact the utility of a product or its perceived product profile among prescribers. Post-marketing studies may also lead to the introduction of new warnings in the product prescribing information. The FDA may require adoption of a REMS program requiring prescriber training or a post-marketing registry or may restrict the marketing and dissemination of our products. Finally, failure to complete a post-marketing commitment by the applicable post-marketing milestone date may lead to withdrawal of the product or indication. Any requirements to conduct post-approval studies or fulfill special post-approval requirements could impact our ability to commercialize our product or product candidates and increase our costs.
We are currently focusing the majority of our efforts on developing BRIUMVI (ublituximab-xiiy) and azercabtagene zapreleucel (azer-cel) for particular indications. As a result, we may fail to capitalize on other indications or product candidates that may ultimately have proven to be more profitable.
We are currently focusing the majority of our resources and efforts on developing BRIUMVI (ublituximab-xiiy) and azercabtagene zapreleucel (azer-cel). As a result, we may forego or delay the pursuit of opportunities for other indications or with other product candidates that may have greater commercial potential. Our resource allocation decisions may cause us to fail to capitalize on viable commercial drugs or profitable market opportunities. If we do not accurately evaluate the commercial potential or target markets for BRIUMVI (ublituximab-xiiy) and azercabtagene zapreleucel (azer-cel), we may relinquish valuable rights to our product candidates or programs through collaboration, licensing or other strategic arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate(s) or program(s).
A Breakthrough Therapy or Fast Track designation by the FDA may not actually lead to a faster development or regulatory review or approval process.
We may seek Breakthrough Therapy or Fast Track designation for some of our drug candidates. If a drug is intended for the treatment of a serious or life-threatening condition, and the drug demonstrates the potential to address an unmet medical need for this condition, the Sponsor may apply for Fast Track designation or Breakthrough Therapy designation, the latter of which has more significant requirements. The FDA has broad discretion whether or not to grant these designations, so even if we believe a particular drug candidate is eligible for such a designation, we cannot be sure that the FDA would decide to grant it. Even if we receive Breakthrough Therapy or Fast Track designation for a drug candidate, we may not experience a faster development process, review or approval compared to conventional FDA procedures. A drug that receives Fast Track designation is eligible for more frequent interactions with the FDA, priority review if relevant criteria are met, and rolling submission of the BLA or NDA. Even if rolling review is allowed, there is no guarantee that the FDA will have commenced or completed review of the BLA or NDA modules submitted earlier in the rolling review process. Neither Breakthrough Therapy nor Fast Track designation guarantees Priority Review of an NDA or BLA.
We may seek orphan drug designation for some of our drug candidates. However, we may be unsuccessful in obtaining or may be unable to maintain the benefits associated with orphan drug designation, including the potential for market exclusivity.
Regulatory authorities in some jurisdictions, including the United States, the European Union, and the United Kingdom, may designate drugs for relatively small patient populations as orphan drugs. Under the U.S. Orphan Drug Act, the FDA may designate a drug as an orphan drug if it is a drug intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 individuals annually in the United States, or a patient population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States. In the United States, orphan drug designation entitles a party to financial incentives, such as opportunities for grant funding towards clinical trial costs, tax advantages, and user-fee waivers. Orphan drug designations are required to be maintained through annual reporting and are subject to re-evaluation. Based on the evolving data and development plans for our product candidates and changing incidence and prevalence rates for our intended indications, there can be no guarantee that we will be able to successfully maintain orphan drug designations that we have for certain of our drug candidates or that we will be successful in obtaining orphan designation for other drug candidates in the future.
Generally, if a product with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, the product is entitled to a period of marketing exclusivity, which precludes FDA or EMA from approving another marketing application for the same drug or biologic for that time period. Even if we obtain orphan drug exclusivity for a drug, that exclusivity may not effectively protect the designated drug from competition because different drugs can be approved for the same condition. Even after an orphan drug is approved, the FDA can subsequently approve another product that meets the definition of a “same drug” under 21 C.F.R. 316.3 for the same condition if the FDA concludes that the later product is clinically superior in that it is shown to be safer, more effective or makes a major contribution to patient care. In addition, a designated orphan drug may not receive orphan drug exclusivity if it is approved for a use that is broader than the indication for which it received orphan drug designation. Moreover, orphan drug exclusive marketing rights in the United States may be lost if the FDA exercises its authority to revoke orphan drug designation, which it may do on a variety of grounds, including that the request contained an untrue statement of material fact or omitted material information, or that the drug in fact was not eligible for orphan drug designation. Orphan drug designation neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process. While we intend to seek orphan drug designation for our other drug candidates, we may never receive such designations. Even if we receive orphan drug designation for any of our drug candidates, there is no guarantee that we will enjoy the benefits of those designations or obtain orphan drug exclusivity. In addition, the U.S. Orphan Drug Act may be subject to amendments that could reduce the period of marketing exclusivity or change the qualifications for orphan drug designation, which could adversely impact our products or product candidates that have or may be eligible for orphan drug designation.
We are conducting clinical trials and anticipate conducting additional clinical trials for our product and product candidates at sites outside the United States, and trials conducted in such locations or clinical trial activities in such locations may be impacted by political conditions, including international conflict.
Many of our clinical trials utilize international clinical research sites. We work with what we believe are reputable CROs and clinical research sites in conducting our studies internationally. Nevertheless, there can be heightened challenges to monitoring and oversight of global clinical trials and sponsors are subject to the risk that fraud, misconduct, incompetence, unexpected patient variability and other issues affecting the reliability, quality, and outcome of studies. Such challenges, if they were to occur, could negatively impact trial results, and depending on the circumstances and scope of concerns could potentially even prevent a trial from being useful or acceptable for regulatory approval. If such events were to occur with respect to any of our trials (and in particular with respect to registration-directed studies), they would have a substantial negative impact on our business.
In addition, our clinical studies with sites outside the United States may be adversely impacted by international conflict. For example, in February 2022, Russia initiated a full-scale military invasion of Ukraine. In one or both countries, as well as neighboring countries that may be impacted by this conflict (e.g. Poland, Slovakia, Belarus, Georgia), we have clinical trial sites for our RMS and/or oncology programs. While no clinical trials are actively enrolling patients in Russia or Ukraine, there are a number of trial subjects in long-term treatment and follow-up. The political and physical conditions in Russia and Ukraine have disrupted our ability to supply investigational drug product to impacted sites; impacted patients’ ability to partake in our clinical trials and our ability to gather data on those patients, including long-term follow-up data; and resulted in suspension of clinical trial activities at impacted sites. Furthermore, the United States and its European allies have imposed significant sanctions against Russia and Belarus, including regional embargoes, full blocking sanctions, and other restrictions targeting major Russian financial institutions. Specifically, such sanctions have included, among other things, a prohibition on doing business with certain Russian companies, officials, and oligarchs; a commitment by certain countries and the European Union to remove selected Russian banks from the Society for Worldwide Interbank Financial Telecommunications (SWIFT) electronic banking network that connects banks globally; and restrictive measures to prevent the Russian Central Bank from undermining the impact of the sanctions. Our ability to conduct clinical trials in Russia, Belarus, Ukraine and elsewhere in the region may also become restricted under applicable sanctions laws. The conflict, as well as government responses, has resulted in global economic instability, which could affect our supply chain and commercialization efforts. While we do not believe this conflict will have a material impact on product development or our overall business, given the rapidly evolving situation and the potential to expand beyond Ukraine and Russia, the full impact of the conflict remains uncertain.
The FDA, EMA and other comparable foreign regulatory authorities may not accept data from trials conducted in locations outside of their respective jurisdictions.
We have been and may continue to conduct clinical trials globally. The acceptance of study data by the FDA, EMA or other comparable foreign regulatory authorities from clinical trials conducted outside of their respective jurisdictions may be subject to certain conditions, which may include conditions related to the applicability and verifiability of the data and cooperation with foreign regulatory agencies. In cases where data from United States clinical trials are intended to serve as the basis for marketing approval in countries outside the United States, the standards for clinical trials and approval may be different. There can be no assurance that any United States or foreign regulatory authority would accept data from trials conducted outside of its applicable jurisdiction. If the FDA, EMA or any applicable foreign regulatory authority does not accept such data, it would result in the need for additional trials, which would be costly and time-consuming and delay aspects of our business plan, and may result in our product candidates not receiving approval or clearance for commercialization in the applicable jurisdiction.
Approval of one of our product candidates in the United States would not assure approval of that candidate in foreign jurisdictions.
We intend to seek additional product approvals in certain countries outside of the United States. The approval procedures for pharmaceuticals vary among countries and obtaining approval in one jurisdiction does not guarantee approval in another jurisdiction. For example, even if the FDA grants approval of a product candidate comparable regulatory authorities in foreign jurisdictions may not approve the same product candidate, or the same indications for use for the product candidate, or may require additional evidence for approval. The time required to obtain approval in other countries might differ from that required to obtain FDA approval. In many countries outside the United States, the product must be approved for reimbursement before it can be marketed. As a general matter, however, the foreign regulatory approval process involves a lengthy and challenging process with risks similar or identical to the risks associated with the FDA approval discussed above. Therefore, we cannot guarantee that we, or future collaborators, will obtain approvals of our product and product candidates in any foreign jurisdiction on a timely basis, if at all. Failure to receive approval in certain foreign markets could significantly impact the full market potential of our product and product candidates and may negatively impact the regulatory process in other countries. Furthermore, if we obtain regulatory approval for a product or product candidate in a foreign jurisdiction, we will be subject to the burden of complying with complex regulatory, legal, and other requirements that could be costly and could subject us to additional risks and uncertainties.
We have product candidates still under development and are also engaging manufacturing partners in commercial manufacturing activities, and as such clinical and commercial manufacturing site additions and process improvements implemented in the production of our product and product candidates may affect their timely delivery or quality.
We have limited experience in manufacturing products for clinical or commercial purposes. We currently do not have any manufacturing capabilities of our own. We have established a contract manufacturing relationship for the commercial supply of BRIUMVI with Samsung Biologics. As with any supply program, obtaining materials of sufficient quality and quantity to meet the requirements of the market demand for BRIUMVI and our development programs cannot be guaranteed and we cannot ensure that we will be successful in these endeavors.
To the extent possible and commercially practicable, we plan to develop back-up strategies for raw materials and manufacturing and testing services for our commercial products. Given the long lead times and cost of establishing additional commercial manufacturing sites we expect that we will rely on single contract manufacturers to produce our commercial products under current Good Manufacturing Practice, or cGMP, regulations for the foreseeable future. Our commercial manufacturing partners have a limited number of facilities in which our product candidates can be produced and will have limited experience in manufacturing our product candidates in quantities sufficient for commercialization. Our third-party manufacturers will have other clients and may have other priorities that could affect their ability to perform the work satisfactorily and/or on a timely basis. All of these occurrences would be beyond our control.
We expect to similarly rely on contract manufacturing relationships for our development programs and any products that we may in-license or acquire in the future. However, there can be no assurance that we will be able to successfully contract with such manufacturers on terms acceptable to us, or at all.
Contract manufacturers are subject to ongoing periodic and unannounced inspections by the FDA, the Drug Enforcement Administration, if applicable, and corresponding state agencies to ensure strict compliance with cGMP requirements and other state and federal regulations. Where manufactured products are globally registered, similar regulatory inspection burdens are applicable from each and every marketed territory. If our manufacturing partners are inspected and deemed out of compliance with cGMPs, product recalls could result, inventory could be destroyed, production could be stopped, and supplies could be delayed or otherwise disrupted.
If we need to change manufacturers either before or after commercialization, the FDA and corresponding foreign regulatory agencies may need to approve these new manufacturers in advance, which will involve testing, regulatory submissions, and additional inspections to ensure compliance with FDA and other regulations and standards, and may require significant lead times and delay. Furthermore, switching manufacturers may be difficult because the number of potential manufacturers is limited. It may be difficult or impossible for us to find a replacement manufacturer quickly or on terms acceptable to us, or at all.
Some of our product and product candidates are currently manufactured in relatively small batches for use in preclinical and clinical studies. Process improvements implemented to date have changed, and process improvements in the future may change, the activity and/or analytical profile of the product or product candidates, which may affect the safety and efficacy of the products. It is possible that additional and/or different adverse events may appear among patients exposed to drug product manufactured under one process compared to the other, or that adverse events may arise with greater frequency, intensity and duration among patients exposed to drug product manufactured under one process compared to the other.
Further, no assurance can be given that the material manufactured from any future optimized processes, if any, for BRIUMVI or any of our product candidates will perform comparably to the product or product candidates as manufactured to date which could result in an unexpected safety or efficacy outcome as compared to the data published or presented to date. Similarly, following each round of process improvements, if any, for any of our drug candidates, future clinical trial results conducted with the new material will be subject to uncertainty related to the effects, if any, of those additional process improvements that were made.
Risks Related to Governmental Regulation of Pharmaceutical Industry and Legal Compliance Matters
We are subject to new legislation, regulatory proposals and third-party payor initiatives that may increase our costs of compliance and adversely affect our ability to market our products, obtain collaborators and raise capital.
In both the United States and certain foreign countries, there have been a number of legislative and regulatory changes or proposed changes to the healthcare system, many of which have focused on prescription drug pricing and lowering overall healthcare costs, that could impact our ability to sell our products profitably and support future innovation. We expect prescription drug pricing and other healthcare costs to continue to be subject to intense political and social pressures on a global basis.
In the United States, the President, federal and state legislatures, health agencies and third-party payors continue to focus on containing the cost of healthcare and addressing public concern over access and affordability of prescription drugs. The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010 (collectively, the ACA) was enacted in 2010 and made significant changes to the U.S. healthcare system. ACA changes included expanding healthcare coverage through Medicaid expansion and implementation of the individual health insurance mandate; changing coverage and reimbursement of drug products under government healthcare programs; imposing an annual fee on manufacturers of branded drugs; and expanding government enforcement authority. Although the ACA has been the subject of a number of legislative and litigation challenges since it passed, it is expected that the Biden Administration will seek to strengthen and expand the ACA. We cannot predict what effect, if any, further changes to the ACA would have on our business or if any changes in an administration may have on the ACA or our business.
Beyond the ACA, there has been increasing legislative, regulatory and enforcement interest with respect to prescription drug pricing practices. Proposals that may garner bipartisan legislative support or become legislation through reconciliation include adding a cap on out-of-pocket spending under Medicare Part D, authorizing Medicare to negotiate certain drugs covered by Medicare Parts D and B directly with manufacturers, and imposing limits on increases in drug prices. In addition, President Biden may take executive action to introduce new drug pricing models and other drug pricing initiatives. The Biden Administration also may propose substantial changes to the U.S. healthcare system, including expanding government-funded health insurance options. We are uncertain of the impact or outcome of potential Executive Orders, rescission of rules and policy statements, or new legislation, especially any relative impact on the healthcare regulatory and policy landscape, or the impact they may have on our business. We expect drug pricing will continue to be a focus of the Biden Administration. At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
There have been several recent U.S. Congressional inquiries and proposed and enacted legislation designed to bring more transparency to drug pricing, reduce the cost of prescription drugs under Medicare, limit price increases, evaluate the relationship between pricing and manufacturer patient programs, and reform government health care program reimbursement methodologies for prescription drugs. For example, the Bipartisan Budget Act of 2018 (BBA) increased manufacturer point-of-sale discounts off negotiated prices of applicable brand drugs in the Medicare Part D coverage gap from 50% to 70% effective as of January 1, 2019, ultimately increasing the liability for brand drug manufacturers. In addition, on September 20, 2024, the Centers for Medicare & Medicaid Services issued a final rule titled “Medicaid Program; Misclassification of Drugs, Program Integrity Updates Under the Medicaid Drug Rebate Program” which may impact our reimbursement and rebate strategy. We expect that health care reform measures that may be adopted in the future, may result in more rigorous coverage criteria, increased manufactured financial liability and additional downward pressure on the price that we may receive for any of our product candidates, if approved. Any reduction in reimbursement from Medicare or other government health care programs may result in a similar reduction in payments from private payors.
There continue to be efforts to lower drug prices through increased competition, with policy proposals seeking to facilitate generic and biosimilar approval and marketing authorization. For example, in 2018, the FDA announced the Biosimilar Action Plan and sought input on how the agency can best facilitate greater availability of biosimilar products, including input on whether changes to an approved biologic (e.g., a new indication) would be protected by the remainder of the statutory 12-year exclusivity period (commonly referred to as umbrella exclusivity). In the event there is a modification to the biologic exclusivity period or other steps taken to facilitate biosimilar or generic approvals, we could experience biosimilar/generic competition of any products for which we receive FDA approval at an earlier time than currently anticipated.
On August 16, 2022, President Biden signed into law the Inflation Reduction Act of 2022 (the Act), which, among other provisions, included several measures intended to lower the cost of prescription drugs and related healthcare reforms. Specifically, the Act authorizes and directs the Department of Health and Human Services (DHHS) to set drug price caps for certain high-cost Medicare Part B and Part D qualified drugs, with the initial list of drugs selected on August 29, 2023, and the first year of maximum price applicability to begin in 2026. On October 3, 2023, the Centers for Medicare & Medicaid Services announced that all manufacturers of the initially selected drugs opted to participate. The Act further authorizes the DHHS to penalize pharmaceutical manufacturers that increase the price of certain Medicare Part B and Part D drugs faster than the rate of inflation. Finally, the Act creates significant changes to the Medicare Part D benefit design by capping Part D beneficiaries’ annual out-of-pocket spending at $2,000 beginning in 2025. We cannot be sure whether additional or related legislation or rulemaking will be issued or enacted, or what impact, if any, such changes will have on the profitability of any of our drug candidates, if approved for commercial use, in the future.
At the state level, individual states are experiencing significant economic pressure within their respective Medicaid programs and responding to public concern over the cost of healthcare. States, including California, Florida, Nevada and Maine, among others, have responded to these pressures with a range of legislative enactments and policy proposals designed to control prescription drug prices by, for example, allowing importation of pharmaceutical products from jurisdictions outside the U.S., imposing price controls on state drug purchases, consolidating state drug purchasing to a single purchaser, and imposing transparency measures around prescription drug prices and marketing costs. These measures, which vary by state, could reduce the ultimate demand for our products, if approved, or put pressure on our product pricing.
In addition, other legislative changes have been adopted that could have an adverse effect upon, and could prevent, our products’ or product candidates’ commercial success. More broadly, the Budget Control Act of 2011, as amended, or the Budget Control Act, includes provisions intended to reduce the federal deficit, including reductions in Medicare payments to providers through 2030 (except May 1, 2020 to December 31, 2020). Any significant spending reductions affecting Medicare, Medicaid or other publicly funded or subsidized health programs, or any significant taxes or fees imposed as part of any broader deficit reduction effort or legislative replacement to the Budget Control Act, or otherwise, could have an adverse impact on our anticipated product revenues.
Furthermore, legislative and regulatory proposals have been made to expand post-approval requirements, make changes the Orphan Drug Act and related guidance, reform the 340B Drug Pricing Program, and restrict sales and promotional activities for drugs. With respect to the 340B Drug Pricing Program, recent legislative proposals, as well as judicial challenges to DHHS’s policies, present both opportunities and challenges for drug manufacturers participating in the program. Further, we cannot be sure whether additional legislative changes will be enacted, or whether the FDA regulations, guidance or interpretations will be changed, or what the impact of such changes on the marketing approvals of our product candidates, if any, may be. In addition, increased scrutiny by Congress of the FDA’s approval process may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketing testing and other requirements.
In many international markets, including the European Union, the government regulates prescription drug prices, patient access, and/or reimbursement levels to control the biopharmaceutical budget of their government-sponsored healthcare system. The European Union and some individual countries have announced or implemented measures and may in the future implement new or additional measures, to reduce biopharmaceutical costs to contain the overall level of healthcare expenditures. These measures vary by country and may include, among other things, non-coverage decisions, patient access restrictions, international price referencing, mandatory discounts or rebates, and cross-border sales of prescription drugs. These measures may adversely affect our ability to generate revenues or commercialize our product or product candidates in certain international markets.
There likely will continue to be pressure on prescription drug prices globally and legislative and regulatory proposals, including at the federal and state levels in the U.S., directed at broadening the availability of health care and containing or lowering the cost of health care products and services. We cannot predict the initiatives that may be adopted in the future. The continuing efforts of the government, health insurance companies, managed care organizations and other payors of health care services to contain or reduce costs of health care may adversely affect, among other things:
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our ability to generate revenues and achieve or maintain profitability; |
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the demand for any products for which we may obtain regulatory approval; |
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our ability to set a price that we believe is fair for our products; |
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the level of taxes that we are required to pay; and |
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the availability of capital. |
In addition, governments may impose price controls, which may adversely affect our future profitability.
Inadequate funding for the FDA, the SEC or other government agencies could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, the ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory, and policy changes. Average review times at the agency have fluctuated in recent years as a result. In addition, government funding of the SEC and other government agencies on which our operations may rely, including those that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable.
Disruptions at the FDA and other agencies may also extend the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business. For example, over the last several years, the U.S. government shut down several times and certain regulatory agencies, such as the FDA and the SEC, had to furlough critical employees and stop critical activities. If a prolonged government shutdown occurs, it could significantly impact the ability of the FDA to review and process our regulatory submissions in a timely matter, which could have a material adverse effect on our business. Further, upon completion of this offering and in our operations as a public company, future government shutdowns could impact our ability to access the public markets and obtain necessary capital in order to properly capitalize and continue our operations.
Some of our relationships with customers and third-party payors are subject to applicable fraud and abuse laws, false claims laws, transparency and disclosure laws, health information and security laws, and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, exclusion from government healthcare programs, contractual damages, reputational harm and diminished profits and future earnings.
With the FDA, EMA and MHRA approval of BRIUMVI, we are subject to additional extensive healthcare statutory and regulatory requirements and oversight by the federal government and the states and foreign governments in which we conduct our business. Healthcare providers and third-party payors play a primary role in the recommendation and prescription of any drug candidates for which we obtain marketing approval. Our past, current and future relationships, arrangements and interactions with these professionals and entities, as well as with patients and patient advocacy organizations expose us to broadly applicable fraud and abuse and other healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market, sell and distribute our product and product candidates for which we obtain marketing approval. Restrictions under applicable federal and state healthcare laws and regulations include the following:
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the federal Anti-Kickback Statute prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under federal and state healthcare programs such as Medicare and Medicaid. A person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; |
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the federal False Claims Act imposes civil penalties, including through civil whistleblower or qui tam actions, against individuals or entities for, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government. For example, life sciences companies have faced enforcement actions under the False Claims Act in connection with their alleged off-label promotion of drugs, purportedly concealing price concessions in the pricing information submitted to the government for government price reporting purposes, and allegedly providing free product to customers with the expectation that the customers would bill federal health care programs for the product, among other activities. In addition, the government may assert that a claim including items and services resulting from a violation of the federal Anti-Kickback Statute or the Federal Food, Drug, and Cosmetic Act (FDCA) constitutes a false or fraudulent claim for purposes of the False Claims Act; |
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the federal Health Insurance Portability and Accountability Act of 1996 (HIPAA) as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (HITECH) and its implementing regulations, has fraud provisions that impose criminal and civil liability for knowingly and willingly executing, or attempting to execute, a scheme to defraud any healthcare benefit program, including private third-party payors, or knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement in connection with the delivery of or payment for healthcare benefits, items or services; similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation. A violation of this statute is a felony and may result in fines, imprisonment or exclusion from government sponsored programs, or integrity oversight and reporting obligations to resolve allegations of non-compliance; | |
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the Physician Payments Sunshine Act under section 6002 of the Affordable Care Act requires manufacturers of drugs, devices, biologics and medical supplies that are reimbursable under Medicare, Medicaid or the Children’s Health Insurance Program to monitor and report certain information related to payments and other transfers of value to and the ownership and investment interests of physicians and certain other healthcare providers as well as teaching hospitals to the federal government for redisclosure to the public. The Centers for Medicare & Medicaid Services (CMS) has the potential to impose penalties for violations of the Physician Payment Sunshine Act, depending on the circumstances, and reported payments also have the potential to draw scrutiny to our relationships with health care practitioners and academic medical institutions, which may have implications under the Anti-Kickback Statute and other healthcare laws; |
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HIPAA, as amended by the HITECH and other amendments, and its implementing regulations, which also imposes obligations on certain covered entity healthcare providers, health plans, and healthcare clearinghouses as well as their business associates that perform certain services involving the use or disclosure of individually identifiable health information, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information; |
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a wide range of federal and state consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers including those related to privacy; |
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the FDCA and its implementing regulations, which among other things, strictly regulate drug product marketing and prohibit manufacturers from promotion and marketing of products prior to approval or for uses inconsistent with the FDA-required labeling; |
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federal laws, including the Medicaid Drug Rebate Program, that require pharmaceutical manufacturers to report certain calculated product prices to the government or provide certain discounts or rebates to government authorities or private entities, often as a condition of reimbursement under government healthcare programs; |
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the Drug Supply Chain Security Act (DSCSA), which imposes obligations on entities in the commercial product supply chain, including manufacturers, to identify and track prescription drugs as they are distributed in the U.S.; and |
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state law equivalents of some of the above federal laws, such as anti-kickback and false claims laws that may apply to items or services reimbursed by any third-party payor, including commercial insurers, state transparency laws, state laws limiting interactions between pharmaceutical manufacturers and members of the healthcare industry, and state laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and often are not preempted by federal laws, thus complicating compliance efforts. |
As we continue commercialization of BRIUMVI, we are taking steps to provide patient support services to help patients access the product. Our patient support programs are administered in conjunction with a patient support program vendor and other third parties. There has been heightened governmental scrutiny over the scope of patient support programs and the manner in which drug manufacturers and their vendors operate such programs. We cannot ensure that our compliance controls, policies, and procedures will be sufficient to protect against acts of our employees, business partners or vendors that may violate the laws, regulations, or evolving government guidance on patient support programs. A government investigation, regardless of its outcome, could impact our business practices, harm our reputation, divert attention of management, increase our expenses and reduce availability of assistance to patients. If we or our vendors are deemed to fail to comply with relevant laws, regulations or government guidance in the operation of these programs, we could be subject to damages, fines, penalties or other criminal, civil or administrative sanctions or enforcement actions.
Ensuring that our business arrangements with third parties comply with applicable healthcare laws and regulations involves substantial costs. It is possible that governmental authorities will conclude that our business practices do not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. The compliance and enforcement landscape, and related risk, is informed by government enforcement precedent and settlement history, Advisory Opinions, and Special Fraud Alerts. Our approach to compliance may evolve over time in light of these types of developments. Additionally, the potential safe harbors available under the federal Anti-Kickback Statute are subject to change through legislative and regulatory action, and we may decide to adjust our business practices or be subject to heightened scrutiny as a result. If our operations, including activities to be conducted by our sales team, were to be found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines, exclusion from government-funded healthcare programs, such as Medicare and Medicaid, qui tam actions brought by individual whistleblowers in the name of the government, and the curtailment or restructuring of our operations.
If we violate applicable data privacy and security laws, we may be subject to penalties, including civil and criminal penalties, damages, fines, reputation harm and the curtailment or restructuring of our operations.
We may be subject to privacy and security laws in the various jurisdictions in which we operate, obtain or store personal information. The legislative and regulatory landscape for privacy and data protection continues to evolve, and there has been an increasing focus on privacy and data protection issues with the potential to affect our business.
Within the United States, HIPAA establishes a federal “floor” with respect to privacy, security, and breach notification requirements as it pertains to protected health information subject to HIPAA and does not supersede any state laws insofar as they are broader or more stringent than HIPAA. There are numerous other laws, regulations and legislative and regulatory initiatives at the federal and state levels addressing privacy and security of personal data. Depending on the data we receive, we may be subject to federal and state privacy-related laws that may be more restrictive or contain different requirements than the privacy regulations issued under HIPAA. These laws vary and could impose additional penalties and requirements related to such data. HIPAA affects the ability of healthcare providers and other entities with which we may interact, including clinical trial sites, to disclose patient health information to us. Under Section 5(a) of the Federal Trade Commission Act (FTCA), the Federal Trade Commission (FTC) expects a company’s data security measures to be reasonable and appropriate in light of the sensitivity and volume of consumer information it holds, the size and complexity of its business, and the cost of available tools to improve security and reduce vulnerabilities. The FTC has asserted authority and issued enforcement actions in response to actual or perceived unfair or deceptive practices by a company in the handling of consumer information. Medical data is considered sensitive data that merits stronger safeguards. States may also impose requirements. For example, the California Consumer Privacy Act (CCPA), went into effect in January 2020 creating data privacy obligations for covered companies and providing privacy rights to California residents, including the right to opt out of certain disclosures of their information. Colorado, Connecticut, Washington, Utah, Virginia and Iowa have also enacted data privacy statutes. Among other things, these state-specific laws create new data privacy obligations for covered companies and provide new privacy rights to state residents, including the right to opt out of certain disclosures of their information. The CCPA also created a private right of action with statutory damages for certain data breaches, thereby potentially increasing risks associated with a data breach. The Washington My Health My Data Act also contains a private right of actions. Draft regulations implementing certain of the state statutes have been published, but many questions remain as to how all of the new statutes will be interpreted. In addition to the laws discussed above, we may see more stringent federal and state privacy legislation passed in 2024 and beyond, as increased cyber-attacks have once again put a spotlight on data privacy and security in the U.S. and other jurisdictions. We cannot predict where new legislation might arise, the scope of such legislation, or the potential impact to our business and operations. We expect to incur additional costs to ensure that our data privacy and security policies, procedures, and activities comply with applicable and evolving legal requirements.
Numerous other jurisdictions regulate the privacy and security of personally identifiable data. For example, the processing of personal data in the European Economic Area (EEA), is subject to the General Data Protection Regulation (GDPR), which took effect in May 2018. The GDPR increases obligations with respect to clinical trials conducted in the EEA, such as in relation to the provision of fair processing notices, exercising data subject rights and reporting certain data breaches to regulators and affected individuals, as well as how we document our relationships with third parties that process GDPR-covered personal data on our behalf. The GDPR also increases the scrutiny applied to transfers of personal data from the EEA (including from clinical trial sites in the EEA) to countries that are considered by the EC to lack an adequate level of data protection, such as the United States. In July 2020, the Court of Justice of the European Union invalidated the EU-U.S. Privacy Shield framework, one of the mechanisms used to legitimize the transfer of personal data from the EEA to the U.S., which decision may lead to increased scrutiny on data transfers from the EEA to the U.S. generally and increase our costs of compliance with data privacy legislation.
If our operations are found to be in violation of any data privacy and security laws, rules or regulations that apply to us, we may be subject to penalties, including civil and criminal penalties, damages, fines and the curtailment or restructuring of our operations, which could adversely affect our ability to operate our business and our financial results. Although compliance programs can mitigate the risk of investigation and prosecution for violations of these laws, rules or regulations, we cannot be certain that our program will address all areas of potential exposure and the risks in this area cannot be entirely eliminated, particularly because the requirements and government interpretations of the requirements in this space are constantly evolving. Any action against us for violation of these laws, rules or regulations, even if we successfully defend against it, could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business, as well as damage our business or reputation. Moreover, achieving and sustaining compliance with applicable federal and state privacy, security, fraud and reporting laws may prove costly.
If we fail to adequately understand and comply with the local laws and customs as we expand into new international markets, these operations may incur losses or otherwise adversely affect our business and results of operations.
We expect to operate a portion of our business in certain countries through subsidiaries or through supply, marketing, and distributor arrangements. In those countries where we have limited experience in operating subsidiaries and in reviewing equity investees, we will be subject to additional risks related to complying with a wide variety of national and local laws, including restrictions on the import and export of certain intermediates, drugs, technologies and multiple and possibly overlapping tax laws. In addition, we may face competition in certain countries from companies that may have more experience with operations in such countries or with international operations generally. We may also face difficulties integrating new facilities in different countries into our existing operations, as well as integrating employees hired in different countries into our existing corporate culture. If we do not effectively manage our operations in these subsidiaries and review equity investees effectively, or if we fail to manage our alliances, we may lose money in these countries, and it may adversely affect our business and results of our operations. In all interactions with foreign regulatory authorities and other government agencies, we are exposed to liability risks under the Foreign Corrupt Practices Act (FCPA) or similar anti-bribery laws. We may participate in collaborations and relationships with third parties whose actions could potentially subject us to liability under the FCPA, or local anti-corruption laws. In addition, we cannot predict the nature, scope or effect of future regulatory requirements to which our international operations might be subject or the manner in which existing laws might be administered or interpreted. We are also subject to other laws and regulations governing our international operations, including regulations administered by the governments of the U.S., and authorities in the EU, including applicable export control regulations, economic sanctions on countries and persons, customs requirements, and currency exchange regulations, which we collectively refer to as Trade Control Laws. There is no assurance that we will be completely effective in ensuring our compliance with all applicable anti-corruption laws, including the FCPA, similar anti-bribery laws, or other legal requirements, including Trade Control Laws. If we are not in compliance with the FCPA, and other anti-corruption laws or Trade Control Laws, we may be subject to criminal and civil penalties, disgorgement and other sanctions and remedial measures, and legal expenses, which could have an adverse impact on our business, financial condition, results of operations and liquidity. The Securities and Exchange Commission, or SEC, also may suspend or bar issuers from trading securities on U.S. exchanges, including the Nasdaq Stock Market, for violations of the FCPA’s accounting provisions. Likewise, any investigation of any potential violations of the FCPA, other anti-corruption laws or Trade Control Laws by the U.S. or other authorities, could also have an adverse impact on our reputation, our business, results of operations and financial condition.
Any product for which we obtain marketing approval, including BRIUMVI, could be subject to restrictions or withdrawal from the market and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with products.
Any regulatory approvals that we receive for our drug candidates may be subject to limitations on the indicated uses for which the drug may be marketed or to conditions of approval that may require potentially costly post-marketing clinical trials or surveillance to monitor safety and efficacy of the drug candidate. In addition, any product for which we obtain marketing approval, along with the manufacturing processes and facilities, post-approval clinical data, labeling, advertising and promotional activities for such product, will be subject to continual requirements of, and review by, the FDA, EMA and comparable regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, registration requirements, cGMP requirements relating to quality control, quality assurance and corresponding maintenance of records and documents, and requirements regarding promotional interactions with healthcare professionals.
Failure to comply with these regulatory requirements or later discovery of previously unknown problems with products, manufacturers, or manufacturing processes, may result in actions such as:
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restrictions on product manufacturing, distribution or use; |
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restrictions on the labeling or marketing of a product; |
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requirements to conduct post-marketing studies or clinical trials; |
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warning letters or other advisory actions; |
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request for withdrawal of the products from the market; |
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refusal to approve pending applications or supplements to approved applications that we or our subsidiaries submit; |
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recalls; |
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suspension or termination of ongoing clinical trials; |
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fines, restitutions, or disgorgement of profits or revenues; |
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refusal to permit the import or export of products; |
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product seizure or detentions; |
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injunctions or the imposition of civil or criminal penalties; and |
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adverse publicity. |
Any internal or government investigation of alleged violations of law could require us to expend significant time and resources in response and could generate negative publicity. In addition, the FDA’s or EMA’s regulations, policies or guidance may change and new or additional statutes or government regulations may be enacted that could prevent or delay regulatory approval of our product candidates or further restrict or regulate post-approval activities. We also cannot predict the likelihood, nature, or extent of adverse government regulation that may arise from pending or future legislation or administrative action, either in the United States or abroad.
If we, or our respective suppliers, third-party contractors, clinical investigators or collaborators are slow to adapt, or are unable to adapt, to changes in existing regulatory requirements or adoption of new regulatory requirements or policies, we, our subsidiaries, or our respective collaborators may be subject to the actions listed above, including losing marketing approval for products, resulting in decreased revenue from milestones, product sales or royalties.
If we or any of our contract manufacturers and suppliers fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could seriously harm our business.
Our third-party manufacturers, suppliers, and we are subject to federal, state, and local laws and regulations governing the use, manufacture, storage, handling, release, disposal of, and exposure to, hazardous and regulated materials. Violation of these laws and regulations could lead to substantial fines and penalties. Although we believe that our safety procedures, and those of our third-party manufacturers, for handling and disposing of these materials comply with the standards prescribed by these laws and regulations, we cannot eliminate the risk of accidental contamination or injury from these materials. In the event of an accident, state or federal authorities may curtail our use of these materials and interrupt our business operations. In addition, we could become subject to potentially material liabilities relating to the investigation and cleanup of any contamination, whether currently unknown or caused by future incidents.
The U.S. political and economic environment could materially impact our business operations and financial performance, and uncertainty surrounding the potential legal, regulatory and policy changes by a new U.S. presidential administration may directly affect us and the global economy.
The political and economic environment in the United States and elsewhere has resulted in and will continue to result in some uncertainty. Changing regulatory policies because of the changing political environment could impact our regulatory and compliance costs and future revenues, all of which could materially and adversely affect our business, financial condition and operating results. Failure to adapt to or comply with evolving regulatory requirements or investor or stakeholder expectations and standards could negatively impact our reputation, ability to do business with certain partners, access to capital and our stock price.
Further, a change in the U.S. presidential administration and congressional seat turnover following the 2024 election cycle may result in increased regulatory and economic uncertainty. Changes in federal policy by the executive branch and regulatory agencies may occur over time through the new presidential administration’s and/or Congress’s policy and personnel changes, which could lead to changes involving the level of oversight and focus on the pharmaceutical industry; however, the nature, timing and economic and political effects of such potential changes remain highly uncertain. Any future changes in federal and state laws and regulations, as well as the interpretation and implementation of such laws and regulations, could affect us in substantial and unpredictable ways. At this time, it is unclear what laws, regulations and policies may change and whether future changes or uncertainty surrounding future changes will adversely affect our operating environment and therefore our business, financial condition and results of operations.
Our research and development activities could be affected or delayed as a result of shortages in animal availability or possible restrictions on animal testing. Compliance with governmental regulations regarding the treatment of animals used in research could increase our operating costs, which would adversely affect the commercialization of our products.
Certain laws and regulations may require us to test our product candidates on animals before initiating clinical trials involving humans. Failure to access or a significant delay in accessing animal research models that meet our needs or that fulfill regulatory requirements may materially adversely affect our ability to advance our preclinical and clinical programs and successfully develop our product candidates, which result in significant harm to our business.
Additionally, animal testing activities have been the subject of controversy and adverse publicity. Animal rights groups and other organizations and individuals have attempted to stop animal testing activities by pressing for legislation and regulation in these areas and by disrupting these activities through protests and other means. To the extent the activities of these groups are successful, our research and development activities may be interrupted, delayed or become more expensive. The Animal Welfare Act (AWA), is the federal law that covers the treatment of certain animals used in research. Currently, the AWA imposes a wide variety of specific regulations that govern the humane handling, care, treatment and transportation of certain animals by producers and users of research animals, most notably relating to personnel, facilities, sanitation, cage size, and feeding, watering and shipping conditions. Third parties with whom we contract are subject to registration, inspections and reporting requirements under the AWA. Furthermore, some states have their own regulations, including general anti-cruelty legislation, which establish certain standards in handling animals. Comparable rules, regulations, and obligations exist in many foreign jurisdictions. If we or our contractors fail to comply with regulations concerning the treatment of animals used in research, we may be subject to fines, penalties and adverse publicity, and our operations could be adversely affected.
Risks Related to Our Dependence on Third Parties
We rely on third parties to generate clinical, preclinical and other data necessary to support the regulatory applications needed to conduct clinical trials and submit for marketing approval. We rely on third parties to help conduct our planned clinical trials. If these third parties do not perform their services as required, we may not be able to obtain regulatory approval for or commercialize our product or product candidates when expected or at all.
In order to submit an IND, BLA, or NDA to the FDA and maintain these applications, it is necessary to submit all information on the clinical, non-clinical, chemistry, manufacturing, controls and quality aspects of the product candidate. Clinical trial applications and marketing authorization applications for foreign regulatory bodies have substantially similar requirements. We rely on our third-party contractors and our licensing partners to provide portions of this data. If we are unable to obtain this data, or the data is not sufficient to meet the regulatory requirements, we may experience significant delays in our development programs and commercialization efforts.
Additionally, we use CROs to assist in the conduct of our current clinical trials and expect to use such services for future clinical trials and we rely upon medical institutions, clinical investigators and contract laboratories to conduct our trials in accordance with our clinical protocols and appropriate regulations. Our current and future CROs, investigators and other third parties play a significant role in the conduct of our trials and the subsequent collection and analysis of data from the clinical trials. There is no guarantee that any CROs, investigators and other third parties will devote adequate time and resources to our clinical trials or perform as contractually required. If any third parties upon whom we rely for administration and conduct of our clinical trials fail to meet expected deadlines, fail to adhere to its clinical protocols or otherwise perform in a substandard manner, our clinical trials may be extended, delayed or terminated, and we may not be able to commercialize our product or product candidates. In addition to the third parties identified above, we are also heavily reliant on the conduct of our patients enrolled to our studies by our third-party investigators. We rely on our clinical trial sites and investigators to properly identify and screen eligible candidates for our clinical trials, and for them to ensure participants adhere to our clinical protocol requirements. The majority of our clinical trial conduct occurs in the outpatient setting, where patients are expected to continue to adhere to our study protocol specified requirements. The ability of our enrolled patients to properly identify, document, and report adverse events; take protocol specified study drugs at the correct quantity, time, and setting, as applicable; avoid contraindicated medications; and comply with other protocol specified procedures such as returning to the trial site for scheduled laboratory and disease assessments, is wholly out of our control. Deviations from protocol procedures, such as those identified previously, could materially affect the quality of our clinical trial data, and therefore ultimately affect our ability to develop and commercialize our drug candidates. If any of our clinical trial sites terminates for any reason, we may experience the loss of follow-up information on patients enrolled in our ongoing clinical trials unless we are able to transfer the care of those patients to another qualified clinical trial site. If any of our clinical trial sites is required by the FDA or IRB to close down due to data management or patient management or any other issues, we may lose clinical trial subjects.
Whether conducted through a CRO or through our internal staff, we are solely responsible for ensuring that each of our clinical trials is conducted in accordance with the applicable protocol, legal and regulatory requirements and scientific standards, and our reliance on CROs will not relieve us of our regulatory responsibilities. For any violations of laws and regulations during the conduct of our clinical trials, we could be subject to warning letters or other enforcement actions that may include civil penalties or criminal prosecution. We and our CROs are required to comply with regulations, including GCP guidelines for conducting, monitoring, recording and reporting the results of clinical trials to ensure that the data and results are scientifically credible and accurate, and that the trial patients are adequately informed of the potential risks of participating in clinical trials and their rights are protected. These regulations are enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area and comparable foreign regulatory authorities for any drug candidates in clinical development. The FDA enforces GCP regulations through periodic inspections of clinical trial sponsors, clinical investigators, CROs, institutional review boards, and non-clinical laboratories. If we, our CROs, our investigators or other third parties fail to comply with applicable GCPs, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. We cannot assure you that, upon inspection, the FDA will determine that our current or future clinical trials comply with GCPs. In addition, our clinical trials must be conducted with drug candidates produced under cGMP regulations. Our failure or the failure of our CROs or Contract Manufacturing Organizations (CMOs) to comply with these regulations may require us to repeat clinical trials, which would delay the regulatory approval process and could also subject us to enforcement action. We also are required to register most ongoing clinical trials and post the results of completed clinical trials on government-sponsored databases, e.g., ClinicalTrials.gov, within certain timeframes. Failure to do so can result in fines, adverse publicity and civil and criminal sanctions.
CROs play an important role in the conduct of our clinical trials, especially outside of the United States. As a result, many important aspects of our development programs, including their conduct and timing, will be outside of our direct control. Our reliance on third parties to conduct current or future clinical trials will also result in less direct control over the management of data developed through clinical trials than would be the case if we were relying entirely upon our own staff. Communicating with outside parties can also be challenging, potentially leading to mistakes as well as difficulties in coordinating activities. Outside parties may:
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have staffing difficulties; |
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fail to comply with contractual obligations; |
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experience regulatory compliance issues; |
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undergo changes in priorities or become financially distressed; or |
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form relationships with other entities, some of which may be our competitors. |
These factors may materially adversely affect the willingness or ability of third parties to conduct our clinical trials and may subject us to unexpected cost increases that are beyond our control. If the CROs do not perform clinical trials in a satisfactory manner, breach their obligations to us or fail to comply with regulatory requirements, the development, regulatory approval and commercialization of our drug candidates may be delayed, we may not be able to obtain regulatory approval and commercialize our drug candidates, or our development program may be materially and irreversibly harmed. If we are unable to rely on clinical data collected by our CROs, we could be required to repeat, extend the duration of, or increase the size of any clinical trials we conduct, and this could significantly delay commercialization and require significantly greater expenditures.
If any of our relationships with these third-party CROs terminate, we may not be able to enter into arrangements with alternative CROs. If CROs do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, any clinical trials such CROs are associated with may be extended, delayed or terminated, and we may not be able to obtain regulatory approval for or successfully commercialize our product or product candidates. As a result, we believe that our financial results and the commercial prospects for our product or product candidates in the subject indication would be harmed, our costs could increase and our ability to generate revenue could be delayed.
We contract with third parties for the manufacture of BRIUMVI for commercial supply, as well as all of our clinical product supply, and we expect to continue to do so. This reliance on third parties increases the risk that we will not have sufficient quantities of our products or product candidates or such quantities at an acceptable cost or quality, which could delay, prevent or impair our development or commercialization efforts.
We do not currently own or operate, nor do we have any plans to establish in the future, any manufacturing facilities. We rely, and expect to continue to rely, on third parties for the manufacture, testing, packaging and labeling of any products that we commercialize and our product candidates for preclinical development and clinical testing. For example, we currently rely on Samsung Biologics for clinical and commercial supply of BRIUMVI. In addition, we utilize multiple vendors who provide testing services. Our reliance on third parties increases the risk that we will not have sufficient quantities of our products or product candidates or such quantities at an acceptable cost or quality, which could delay, prevent or impair our development or commercialization efforts.
The facilities used by contract manufacturers to manufacture, test, package, and label our product and product candidates typically undergo periodic inspections by the FDA or a comparable foreign regulatory authority to verify compliance with applicable cGMP regulations. Additional inspections may be conducted after we submit our marketing applications to or receive marketing approval from the FDA or a comparable foreign regulatory authority. Although the FDA and other regulators impose requirements regarding our selection, qualification, oversight, and monitoring of our contract manufacturers and hold us responsible for the ultimate compliance of our products, we do not directly control the manufacturing process of our third-party contract manufacturers and are subject to risks associated with their ability to comply with cGMPs in connection with the manufacture of our products and product candidates. If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA or others and the compliance concerns cannot be resolved, remediated, or otherwise addressed to the FDA’s or others’ satisfaction in a timely manner during the review of any marketing applications that we submit, it may negatively impact our ability to obtain regulatory approval for our drug candidates or obtain approval within projected timelines. We cannot guarantee the ability of our third-party manufacturers to maintain compliance with cGMP regulations, including having adequate quality control, quality assurance and qualified personnel. Further, our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of products or product candidates, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect our business and supplies of our products or product candidates.
Our reliance on third-party manufacturers entails additional risks, including:
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reliance on the third party for regulatory compliance and quality assurance; |
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the possible breach of the manufacturing, supply or quality agreement by the third party; |
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the possible misappropriation of our proprietary information, including our trade secrets and know-how; and |
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the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us. |
Moreover, our current long-term supply agreement for BRIUMVI contains certain minimum purchases in what are commonly referred to as a “take or pay” provision, and it is possible that future supply agreements could contain such provisions. To the extent our demand does not meet the minimum supply required amounts, we would be forced to pay more than desired. This could create a situation where we are spending more than required and could impact our ongoing operations and entail curtailing other important research and development or commercialization efforts, all of which could have a material adverse effect on the Company. In negotiating our supply agreement for BRIUMVI, there is no guarantee that we have foreseen all eventualities or that our third-party manufacturer will be able to accommodate unforeseen changes in business direction in a timely fashion or at all. Scheduling of manufacturing at our third-party manufacturer is governed by contractual terms that require us to make investments in inventory of materials, with limited shelf-life, in advance of regulatory approval and based on preliminary commercial forecasting, and such inventory may not be used if timelines and supply needs shift.
Our drug candidates and any drugs that we may develop may compete with other drug candidates and approved drugs for access to manufacturing facilities. There are a limited number of manufacturers that operate under cGMP regulations and that might be capable of manufacturing for us. Any third-party manufacturer with which we contract will have other clients, and our relative importance as a customer may adversely impact contractual terms or the performance of services in a satisfactory manner or on a timely basis.
Any performance failure on the part of our existing or future manufacturers could delay clinical development or marketing approval or interrupt commercial distribution. If our current contract manufacturers cannot perform as agreed, we may be required to replace such manufacturers causing additional costs and delays in identifying and qualifying any such replacement. If a new contract manufacturer is not successful in replicating the product or experiences delays, or if regulatory authorities impose unforeseen requirements with respect to product comparability from multiple manufacturing sources, we may experience delays in clinical development or an interruption in our commercial supply. No assurance can be given that any new manufacturer will be successful or that material manufactured by a new manufacturer will perform comparably to product manufactured by the previous manufacturer or that the relevant regulatory agencies will agree with our interpretation of comparability. Any significant delays or gaps in supply of commercial or clinical products may adversely affect our clinical development program, our ability to commercialize any drugs that receive marketing approval on a timely and competitive basis, and our future profit margins.
We also rely on other third parties to store and distribute drug supplies for our clinical trials and for commercial demand for BRIUMVI and expect to continue to do so for any other potential commercial products. Any performance failure on the part of our distributors could delay clinical development or marketing approval of any future product candidates or commercialization of our products, producing additional losses and depriving us of potential product revenue.
The third parties upon whom we rely for the supply of starting materials, intermediates, active pharmaceutical ingredient (API)/drug substance, drug product, and other materials used in our drug candidates are our sole source of supply, and the loss or disruption of any of these suppliers could significantly harm our business.
The starting materials, intermediates, API/drug substance, and drug product used in many of our drug candidates are currently supplied to us from single-source suppliers. Our ability to successfully develop our drug candidates, supply our drug candidates for clinical trials and to ultimately supply our commercial drugs in quantities sufficient to meet the market demand, depends in part on our ability to obtain starting materials, intermediates, API/drug substance, and drug product for these drugs in accordance with regulatory requirements and in sufficient quantities for clinical testing and commercialization. It is expected that many of our manufacturing partners will be sole source suppliers from single site locations for the foreseeable future. Various raw materials, components, and testing services required for our product and product candidates may also be single sourced. We are not certain that our single-source suppliers will be able to supply sufficient quantities of their products or on the timelines necessary to meet our needs, either because of the nature of our agreements with those suppliers, our limited experience with those suppliers, our relative importance as a customer to those suppliers, international political conflicts that may impact trade or the supply chain within a particular region, public health emergencies or natural disasters that may cause those suppliers to stop work for a period of time or lead to a sudden increase in demand for selected materials resulting in short-term unavailability of such materials. If any of our suppliers ceases operations for any reason or is unable or unwilling to supply starting materials, intermediates, API/drug substance, and drug product in sufficient quantities or on the timelines necessary to meet our needs, it could significantly and adversely affect our business, the supply of our drug products and drug candidates and our financial condition. In addition, if our current or future supply of any of our products or product candidates should fail to meet specifications during its stability program there could be a voluntary or mandatory product recall if the product is approved and, even in the absence of a recall, there could be significant interruption of our supply of drug, which would adversely affect the clinical development and commercialization of the product.
We continually evaluate our supply chains to identify potential risks and needs for additional manufacturers and other suppliers for the production of our products and product candidates. Establishing additional or replacement suppliers for the API/drug substance, drug product, and certain raw materials, if required, may not be accomplished quickly, or at all, and may involve significant expense. If we are able to find a replacement supplier, we would need to evaluate and qualify such replacement supplier and its ability to meet quality and compliance standards. Any change in suppliers or the manufacturing process could require additional regulatory approval and result in operational delays. While we seek to maintain adequate inventory of materials necessary for the production of our products and product candidates, any supply interruption or delay, or our inability to identify alternate sources at acceptable prices in a timely manner could impede, delay, limit or prevent our commercialization and development efforts, which could harm our business, results of operations, financial condition and prospects.
Because we have in-licensed BRIUMVI and our product candidates from third parties, any dispute with or non-performance by our licensors will adversely affect our ability to develop and commercialize the applicable product or product candidate.
Because we license BRIUMVI and our product candidates from third parties and we expect to continue to in-license additional product candidates, if there is any dispute between us and our licensor regarding our rights under a license agreement, our ability to develop and commercialize the applicable product or product candidate may be adversely affected. Disputes may arise with the third parties from whom we license our products and product candidates for a variety of reasons, including:
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the scope of rights granted under the license agreement and other interpretation-related issues; |
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the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the license agreement; |
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the sublicensing of patent and other rights under our collaborative development relationships and obligations associated with sublicensing; |
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our diligence obligations under the license agreement and what activities satisfy those diligence obligations; |
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the ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners; and |
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the priority of invention of patented technology. |
In addition, the agreements under which we currently license BRIUMVI and our product candidates from third parties are complex, and certain provisions in such agreements may be susceptible to multiple interpretations, or may conflict in such a way that puts us in breach of one or more agreements, which would make us susceptible to lengthy and expensive disputes with one or more of our licensing partners. The resolution of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or other obligations under the relevant agreement, either of which could have a material adverse effect on our business, financial condition, results of operations, and prospects. Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product or product candidate, which could have a material adverse effect on our business, financial conditions, results of operations, and prospects.
If conflicts arise between us and our future collaborators or strategic partners, these parties may act in a manner adverse to us and could limit our ability to implement our strategies.
If conflicts arise between our future corporate or academic collaborators or strategic partners and us, the other party may act in a manner adverse to us and could limit our ability to implement our strategies. Future collaborators or strategic partners, may develop, either alone or with others, products in related fields that are competitive with the products or potential products that are the subject of these collaborations. Competing products, either developed by the collaborators or strategic partners or to which the collaborators or strategic partners have rights, may result in the withdrawal of partner support for any future product candidates. Our current or future collaborators or strategic partners may preclude us from entering into collaborations with their competitors, fail to obtain timely regulatory approvals, terminate their agreements with us prematurely, or fail to devote sufficient resources to the development and commercialization of products. Any of these developments could harm any future product development efforts.
We are dependent upon our relationships with collaboration and commercialization partners to further develop, fund, manufacture and commercialize our drug products and our product candidates. If such relationships are unsuccessful, or if a collaboration or commercialization partner terminates its collaboration or commercialization agreement with us, it could negatively impact our ability to conduct our business and generate net product revenue. Failure by a collaboration or commercialization partner to perform its duties under its collaboration or commercialization agreement with us (e.g. financial reporting or internal control compliance) may negatively affect us.
On July 28, 2023, we entered into a commercialization agreement (the Commercialization Agreement) with Neuraxpharm Pharmaceuticals, S.L. (Neuraxpharm), pursuant to which Neuraxpharm has the right to commercialize BRIUMVI in certain markets outside of the U.S. On February 26, 2024, Neuraxpharm announced the commercial launch of BRIUMVI in Germany and expects to launch BRIUMVI in additional international markets as well. In addition to the Commercialization Agreement, we may enter into collaboration arrangements with other collaboration and commercialization partners.
We are subject to a number of risks associated with our dependence on our relationships with our collaboration and commercialization partners, including:
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decisions by our collaboration and commercialization partners to terminate their collaboration or commercialization agreements with us for reasons specified in the collaboration or commercialization agreements, including our breach; |
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the need for us to identify and secure on commercially reasonable terms the services of third parties to perform key activities, including development and commercialization activities, currently performed by our collaboration or commercialization partners in the event that a collaboration or commercialization partner terminates its agreement with us; |
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adverse decisions by a collaboration or commercialization partner regarding the amount and timing of resource expenditures for the commercialization, distribution, and sale of our drug products; |
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failure by a collaboration or commercialization partner to perform its duties under its agreement with us (e.g., its failure to comply with regulatory requirements which may disrupt its performance of its obligations under the agreement with us); |
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failure by a collaboration or commercialization partner to timely deliver accurate and complete financial information to us or to maintain adequate and effective internal control over its financial reporting may negatively affect our ability to meet our financial reporting obligations as required by the SEC; |
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failure by a collaboration or commercialization partner to timely deliver accurate and complete medical or clinical information to us or to maintain adequate and effective internal control over its pharmacovigilance activities and reporting may negatively affect our ability to meet our reporting obligations as required by the FDA and other regulatory bodies; |
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collaboration or commercialization partners’ and their affiliates’ development and commercialization of products that compete directly or indirectly with our products or product candidates; |
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decisions by a collaboration or commercialization partner to prioritize others of its current or future products more highly than our drug products or our product candidates when it performs its duties; |
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possible disagreements with a collaboration or commercialization partner as to the timing, nature and extent of our development plans or distribution and sales and marketing plans; and |
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the fact that financial returns to us, if any, under our collaboration agreement with Neuraxpharm depends in large part on the achievement of milestones and generation of product sales, and if Neuraxpharm fails to perform or satisfy its obligations under the collaboration agreements, the development and commercialization of our drug products could be delayed, hindered or may not occur, and our business and prospects could be materially and adversely affected. |
While the Commercialization Agreement contains provisions that allow for dispute resolution, arbitration, and/or termination of the agreement by the Company in the event of a breach by Neuraxpharm, there can be no assurance that the Company and Neuraxpharm will agree on a cure for such a breach, and in the event of termination, there can be no assurance that the Company would be appropriately compensated and/or recover any losses sustained. Due to these factors and other possible disagreements with our collaboration and commercialization partners, we may be delayed or prevented from further developing, manufacturing or commercializing our drug products or our product candidates or we may become involved in litigation or arbitration, which would be time consuming and expensive.
If any collaboration or commercialization partner were to terminate our relationship with it unilaterally, we would need to undertake development, commercialization or distribution or sale activities for our drug products and product candidates solely at our own expense, and/or seek one or more other partners for some or all of these activities in the U.S. or worldwide. If we pursued these activities on our own, it would significantly increase our capital and infrastructure requirements, might limit the indications we are able to pursue for our drug products and our product candidates, and could prevent us from effectively commercializing our drug products and our product candidates. If we sought to find one or more other pharmaceutical company partners for some or all of these activities, we may not be successful in such efforts, or they may result in collaborations that have us expending greater funds and efforts than our relationships with our current collaboration and commercialization partners.
We may seek to establish additional collaborations, and if we are not able to establish them on commercially reasonable terms, we may have to alter our development and commercialization plans.
Our drug development programs and the potential commercialization of our drug candidates will require substantial additional cash to fund related expenses. Therefore, for some of our drug candidates, we may decide to collaborate with additional pharmaceutical and biotechnology companies for the development and potential commercialization of those drug candidates.
We face significant competition in seeking appropriate collaborators. Whether we reach a definitive agreement for a collaboration will depend, among other things, upon our assessment of the proposed collaborator’s resources and expertise, the terms and conditions of the proposed collaboration with a third party, and the proposed collaborator’s evaluation of a number of factors. Those factors may include the design or results of our clinical trials, the likelihood of approval by the FDA, EMA or similar regulatory authorities outside the United States, the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing drugs, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge, and industry and market conditions generally. The collaborator may also consider alternative product candidates or technologies for similar indications that may be available to collaborate on and whether such a collaboration could be more attractive than the one with us for our product candidate. The terms of any additional collaborations or other arrangements that we may establish may not be favorable to us.
We may be restricted under our collaboration agreements from entering into future agreements on certain terms with potential collaborators. Collaborations are complex and time-consuming to negotiate and document. In addition, there have been a significant number of recent business combinations among large pharmaceutical companies that have resulted in a reduced number of potential future collaborators.
We may not be able to negotiate additional collaborations on a timely basis, on favorable terms to us, or at all. If we are unable to do so, we may have to curtail the development of the product candidate for which we are seeking to collaborate, reduce or delay its development program or one or more of our other development programs, delay its potential commercialization or reduce the scope of any sales or marketing activities, or increase our expenditures and undertake development or commercialization activities at our own expense. If we elect to increase our expenditures to fund development or commercialization activities on our own, we may need to obtain additional capital, which may not be available to us on favorable terms or at all. If we do not have sufficient funds, we may not be able to further develop our product candidates or bring them to market and we may ultimately not be able to generate revenue from their sales.
Risks Related to Our Intellectual Property
Our success depends upon our ability to obtain and protect our intellectual property and proprietary technologies. If the scope of our patent protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and drugs similar or identical to ours, and our ability to successfully commercialize our technology and drugs may be impaired.
Our commercial success in part depends on obtaining and maintaining patent protection and trade secret protection in the United States and other countries with respect to any product we commercialize, including BRIUMVI, our product candidates, their formulations and uses and the methods we use to manufacture them, as well as successfully defending these patents against third-party challenges. We seek to protect our proprietary and intellectual property position by filing patent applications in the United States and abroad related to our novel technologies and product candidates, and by maintenance of our trade secrets through proper procedures. Because we in-license our products and product candidates, we also rely on our licensors to protect the patent and other intellectual property rights necessary for commercialization.
We will only be able to protect our technologies from unauthorized use by third parties to the extent that valid and enforceable patents or trade secrets cover them in the market they are being used or developed. The degree of patent protection we require to successfully commercialize our products and product candidates may be unavailable or severely limited in some cases and may not adequately protect our rights or permit us to gain or keep any competitive advantage. We cannot provide any assurances that any of our patents have, or that any of our pending patent applications that mature into issued patents will include, claims with a scope sufficient to protect any of our products. In addition, the laws of foreign countries may not protect our patent rights to the same extent as the patent laws of the United States.
Furthermore, patents have a limited lifespan. In the United States, the natural expiration of a patent is generally twenty years after it is filed. Various extensions may be available; however, the life of a patent, and the protection it affords, is limited. Given the amount of time required for the development, testing and regulatory review of new drug candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our patent portfolio may not provide us with adequate and continuing patent protection sufficient to exclude others from commercializing drugs similar or identical to our product or product candidates, including generic versions of such drugs.
Currently, we have several granted patents in the United States and EU, among other countries, and several pending patent applications that have not yet been issued or have been issued in certain jurisdictions but not all jurisdictions in which such applications have been filed. There can be no guarantee that any pending patent applications, nor any patent applications filed in the future will be granted in any or all jurisdictions in which they were filed, or that all patent claims initially submitted for examination in such patent applications will be allowed in the patent that is eventually granted, if at all. The patent prosecution process is subject to numerous risks and uncertainties, and there can be no assurance of the scope of patent claims that will ultimately be allowed, if at all, and no assurance that we or our partners will be successful in protecting our product and product candidates by obtaining and defending patents.
These risks and uncertainties include the following:
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the patent applications that we or our licensors file may not issue as patent; |
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patents that may be issued or in-licensed may be challenged, invalidated, modified, revoked or circumvented, or otherwise may not provide any competitive advantage; |
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as of March 16, 2013, the United States converted from a first-to-invent to a first-to-file system. If we do not win the filing race, we will not be entitled to inventive priority; |
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our competitors, many of whom have substantially greater resources than we do, and many of whom have made significant investments in competing technologies, may seek, or may already have obtained, patents that will limit, interfere with, or eliminate our ability to file new patent applications covering our products, or make, use, and/or sell our products either in the United States or in international markets; |
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there may be significant pressure on the United States government and other international governmental bodies to limit the scope of patent protection both inside and outside the United States for disease treatments that prove successful as a matter of public policy regarding worldwide health concerns, which could limit our ability to fully monetize our intellectual property rights; and |
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countries other than the United States may have less restrictive patent laws than those of the United States, allowing foreign competitors to exploit such less restrictive patent laws to make, use, and/or sell competing products in their respective jurisdictions. |
If we are not able to obtain patents that protect our product and product candidates, it could have a material adverse effect on our financial condition and results of operations.
In addition, the patent prosecution process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. Further, with respect to some of the pending patent applications covering our drug candidates, prosecution has yet to commence. Patent prosecution is a lengthy process, during which the scope of the claims initially submitted for examination by the United States Patent and Trademark Office (USPTO) can be significantly narrowed by the time they issue, if at all. It is also possible that we will fail to identify any patentable aspects of our research and development output and methodology, and, even if we do, an opportunity to obtain patent protection may have passed. Given the uncertain and time-consuming process of filing patent applications and prosecuting them, it is possible that our product(s) or process(es) originally covered by the scope of our patent applications may change or be modified throughout the patent prosecution process, leaving our product(s) or process(es) without patent protection. Moreover, in some circumstances, we do not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the patents, that cover technology licensed from third parties. Therefore, these patents and applications may not be prosecuted and enforced in a manner consistent with the best interests of our business. If our licensors or we fail to appropriately prosecute and maintain patent protection or trade secret protection for one or more products or product candidates, our ability to develop and commercialize such drugs may be adversely affected and we may not be able to prevent competitors from making, using and selling competing products. This failure to properly protect the intellectual property rights relating to our product and product candidates could impair our ability to compete in the market and adversely affect our ability to generate revenues and achieve profitability, which would have a material adverse effect on our financial condition and results of operations. Furthermore, should we enter into other collaborations, including out-licensing, joint development projects, partnerships, or strategic alternatives, we may be required to consult with or cede control to collaborators regarding the prosecution, maintenance and enforcement of patents licensed or developed under such collaborations. Therefore, such patents and patent applications may not be prosecuted and enforced in a manner consistent with the best interests of our business.
The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions, and has in recent years been the subject of much litigation. In addition, no consistent policy regarding the breadth of claims allowed in pharmaceutical or biotechnology patents has emerged to date in the United States. The patent situation outside the United States is even more uncertain. The patent laws of foreign countries may not protect our patent rights to the same extent as the laws of the United States, and we may fail to seek or obtain patent protection in all major markets. For example, European patent law restricts the patentability of methods of treatment of the human body more than United States patent law does. Our pending and future patent applications may not result in patents being issued which protect our technology or products, in whole or in part, or which effectively prevent others from commercializing competitive technologies and products. Changes in either the patent laws or interpretation of the patent laws in the U.S. and other countries may diminish the value of our patents or narrow the scope of our patent protection. For example, the federal courts of the United States have taken an increasingly dim view of the patent eligibility of certain subject matter, such as naturally occurring nucleic acid sequences, amino acid sequences and certain methods of utilizing same, which include their detection in a biological sample and diagnostic conclusions arising from their detection. Such subject matter, which had long been a staple of the biotechnology and biopharmaceutical industry to protect their discoveries, is now considered, with few exceptions, ineligible in the first instance for protection under the patent laws of the United States. Accordingly, we cannot predict the breadth of claims that may be allowed or enforced in our patents or in those licensed from a third party.
In addition, U.S. patent laws may change, which could prevent or limit us, our subsidiaries, or our licensors from filing patent applications or patent claims to protect products and/or technologies or limit the exclusivity periods that are available to patent holders, as well as affect the validity, enforceability, or scope of issued patents. For example, on September 16, 2011, the Leahy-Smith America Invents Act was signed into law. The Leahy-Smith Act includes a number of significant changes to United States patent law. These include the transition from a first-to-invent system to a first-to-file system and changes to the way issued patents are challenged. The formation of the Patent Trial and Appeal Board now provides a quicker and less expensive process for challenging issued patents.
The patents or patent applications owned or filed by us, or by our licensors or other collaborators, may be affected by third-party pre-issuance submissions of prior art to the USPTO, or by opposition, derivation, reexamination, inter parties review, post-grant review or interference proceedings. The costs of these proceedings could be substantial, and it is possible that our efforts to establish priority of invention would be unsuccessful, resulting in a material adverse effect on our U.S. patent position. An adverse determination in any such submission, patent office trial, proceeding or litigation could reduce the scope of, render unenforceable, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize products without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by patents and patent applications for our drug candidates is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future products or product candidates.
The issuance of a patent does not foreclose challenges to its inventorship, scope, validity or enforceability. Therefore, our owned and licensed patents may be challenged in the courts or patent offices in the U.S. and abroad. Such challenges may result in loss of exclusivity or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the duration of the patent protection of our technology and products. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such product candidates might expire before or shortly after such product candidates are commercialized. As a result, our owned and licensed patent portfolio may not provide us with enough rights to exclude others from commercializing products similar or identical to ours.
Even if our patent applications issue as patents, and they are unchallenged, our issued patents and pending patent applications, if issued, may not provide us with any meaningful protection or prevent competitors from designing around our patent claims to circumvent our owned or licensed patents by developing similar or alternative technologies or drugs in a non-infringing manner. For example, a third party may develop a competitive drug that provides benefits similar to one or more of our products or product candidates but that has a different composition that falls outside the scope of our patent protection. If the patent protection provided by the patents and patent applications we hold or pursue with respect to our products or product candidates is not sufficiently broad to impede such competition, our ability to successfully commercialize our products or product candidates could be negatively affected, which would harm our business.
In addition, we may in the future be subject to claims by our former employees or consultants asserting an ownership right in our patents or patent applications, as a result of the work they performed on our behalf. Although we have entered into agreements with many of our employees, consultants and advisors and any other third parties who have access to our proprietary know-how, information or technology for the purpose of assigning or granting similar rights to their inventions to us, we cannot be certain that we have executed such agreements with all parties who may have contributed to our intellectual property, nor can we be certain that our agreements with such parties will be upheld in the face of a potential challenge, or that they will not be breached, for which we may not have an adequate remedy. An adverse determination in any such submission or proceeding may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and drugs, without payment to us, or could limit the duration of the patent protection covering our technology and drug candidates. Such challenges may also result in our inability to manufacture or commercialize our products and product candidates without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future drug candidates.
Patent protection and other intellectual property protection are crucial to the success of our business and prospects, and there is a substantial risk that such protections may prove inadequate.
Obtaining and maintaining patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
The USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other similar provisions during the patent application process. In addition, periodic maintenance fees on issued patents often must be paid to the USPTO and foreign patent agencies over the lifetime of the patent. While an unintentional lapse can in many cases be cured by payment of a late fee or by other methods in accordance with the applicable rules, there are situations in which noncompliance can result in abandonment or lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction.
Non-compliance events that could result in abandonment or lapse of a patent or patent application include, but are not limited to, failure to respond to official actions within prescribed time limits, non-payment of fees and failure to properly legalize and submit formal documents. If we fail to maintain the patents and patent applications covering our drugs or procedures, we may not be able to stop a competitor from marketing drugs that are the same as or similar to our products or product candidates, which would have a material adverse effect on our business.
If we do not obtain patent term extensions under the Hatch-Waxman Act and similar foreign legislation extending the terms of our licensed patents and any future patents we may own, our business may be materially harmed.
Depending on the timing, duration, and specifics of any FDA regulatory approval for our drug candidates, one or more of our licensed U.S. patents or future U.S. patents that we may license or own may be eligible for limited patent term restoration under the Hatch-Waxman Act. The Hatch-Waxman Act permit a patent term extension of up to five years as compensation for patent term lost during the FDA regulatory review process. A patent term extension cannot extend the remaining term of a patent beyond fourteen years from the date of product approval by the FDA, and only one patent covering the approved product may be extended.
The application for a patent term extension is subject to approval by the USPTO, in conjunction with the FDA. We may not be granted an extension because of, for example, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of the patent protection afforded could be less than what we request. If we are unable to obtain patent term extension or any term of such extension is less than we request, the period during which we will have the right to exclusively market our product will be shortened and our competitors may obtain earlier approval of competing products, and our ability to generate revenues could be materially adversely affected.
We may not be able to enforce our intellectual property rights throughout the world.
Filing, prosecuting and defending patents on drug candidates throughout the world would be prohibitively expensive. Competitors may use our licensed and owned technologies in jurisdictions where we have not licensed or obtained patent protection to develop their own products and, further, may export otherwise infringing products to territories where we may obtain or license patent protection, but where patent enforcement is not as strong as that in the United States. These products may compete with our products in jurisdictions where we do not have any issued or licensed patents and any future patent claims or other intellectual property rights may not be effective or sufficient to prevent them from so competing.
Moreover, our ability to protect and enforce our intellectual property rights may be adversely affected by unforeseen changes in foreign intellectual property laws. Additionally, laws of some countries outside of the United States and Europe do not afford intellectual property protection to the same extent as the laws of the United States and Europe. Many companies have encountered significant problems in protecting and defending intellectual property rights in certain foreign jurisdictions. The legal systems of some countries, including India, China and other developing countries, do not favor the enforcement of patents and other intellectual property rights. This could make it difficult for us to stop the infringement of our patents or the misappropriation of our other intellectual property. For example, many foreign countries have compulsory licensing laws under which a patent owner must grant licenses to third parties. Consequently, we may not be able to prevent third parties from practicing our inventions in certain countries outside the United States and Europe.
Proceedings to enforce our future patent rights, if any, in foreign jurisdictions could result in substantial cost and divert our resources and attention from other aspects of our business. Moreover, such proceedings could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be meaningful. Furthermore, while we intend to protect our intellectual property rights in major markets for our products, we cannot ensure that we will be able to initiate or maintain similar efforts in all jurisdictions in which we may wish to market our products. Accordingly, our efforts to protect our intellectual property rights in such countries may be inadequate.
We may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time consuming and unsuccessful.
Competitors may infringe our patents or the patents of our licensors. To counter infringement or unauthorized use, we may be required to file infringement claims, which typically are very expensive, time-consuming and disruptive to our day-to-day business operations. Any claims we assert against accused infringers could provoke these parties to assert counterclaims against us alleging invalidity of our or certain of our subsidiaries’ patents or that we infringe their patents; or provoke those parties to petition the USPTO to institute inter parties review against the asserted patents, which may lead to a finding that all or some of the claims of the asserted patents are invalid. In addition, in an infringement proceeding, a court may decide that a patent of ours or our licensors is not valid or is unenforceable or may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question. An adverse result in any litigation or defense proceedings could put one or more of our pending patents at risk of being invalidated, held unenforceable, or interpreted narrowly.
In patent litigation in the United States, defendant counterclaims challenging the validity, enforceability or scope of asserted patents are commonplace. In addition, third parties may initiate legal proceedings against us to assert such challenges to our intellectual property rights. The outcome of any such proceeding is generally unpredictable. Grounds for a validity challenge could be an alleged failure to meet any of several statutory requirements, including lack of novelty, obviousness or non-enablement. Patents may be unenforceable if someone connected with the prosecution of the patent withheld relevant information from the USPTO or made a misleading statement during prosecution. It is possible that prior art of which we and the patent examiner were unaware during prosecution exists, which could render our patents invalid. Moreover, it is also possible that prior art may exist that we are aware of but do not believe is relevant to our current or future patents, but that could nevertheless be determined to render our patents invalid.
Competing drugs may also be sold in other countries in which our patent coverage might not exist or be as strong as in the United States. If we lose a foreign patent lawsuit, alleging our infringement of a competitor’s patents, we could be prevented from marketing our drugs in one or more foreign countries. Any of these outcomes would have a material adverse effect on our business.
In addition, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. Furthermore, adverse results on United States patents may affect related patents in our global portfolio. The adverse result could also put related pending patent applications at risk of not issuing. Additionally, there could be public announcements of the results of hearings, motions or other interim proceedings or developments. If securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock.
Interference proceedings provoked by third parties or brought by the USPTO may be necessary to determine the priority of inventions with respect to our patents or pending patent applications or those of our collaborators or licensors. An unfavorable outcome could require us to cease using the related technology or to attempt to license rights to it from the prevailing party. The costs of these proceedings could be substantial. As a result, the issuance, scope, validity, enforceability and commercial value of our or any of our respective licensors’ patent rights are highly uncertain. Our business could be harmed if the prevailing party does not offer us a license on commercially reasonable terms. Litigation or interference proceedings may fail and, even if successful, may result in substantial costs and distract our management and other employees. We may not be able to prevent, alone or with our licensors, misappropriation of our trade secrets or confidential information, particularly in countries where the laws may not protect those rights as fully as in the United States.
We may not have sufficient financial or other resources to adequately conduct such litigation or proceedings. Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources and more mature and developed intellectual property portfolios. Accordingly, despite our efforts, we may not be able to prevent third parties from infringing upon or misappropriating or from successfully challenging our intellectual property rights. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could have a material adverse effect on our ability to compete in the marketplace.
If we or our partners are sued for infringing intellectual property rights of third parties, it will be costly and time consuming, and an unfavorable outcome in that litigation would have a material adverse effect on our business.
Our commercial success depends upon our ability and the ability of our collaborators to develop, manufacture, market and sell our drug candidates and use our proprietary technologies without infringing the proprietary rights and intellectual property of third parties. The biotechnology and pharmaceutical industries are characterized by extensive and frequent litigation regarding patents and other intellectual property rights. We may in the future become party to, or threatened with, adversarial proceedings or litigation regarding intellectual property rights with respect to our drug candidates and technology, including interference proceedings before the USPTO.
Our competitors or other third parties may assert infringement claims against us, alleging that our drugs are covered by their patents. Given the vast number of patents in our field of technology, we cannot be certain that we do not infringe existing patents or that we will not infringe patents that may be granted in the future. Numerous United States and foreign issued patents and pending patent applications, which are owned by third parties, exist in the fields in which we are developing products, some of which may be directed at claims that overlap with the subject matter of our intellectual property. In addition, because patent applications can take many years to issue, there may be currently pending applications, unknown to us, which may later result in issued patents that our product or product candidates or proprietary technologies may infringe. Similarly, there may be issued patents relevant to our product or product candidates of which we are not aware. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after a first filing, or in some cases not at all. Therefore, we cannot know with certainty whether we or our licensors were the first to make the inventions claimed in patents or pending patent applications that we own or licensed, or that we or our licensors were the first to file for patent protection of such inventions.
We are aware of certain patents that may pose issues for our commercialization of our product and product candidates. If we decide to initiate proceedings to challenge the validity of these patents in the future, we may be unsuccessful, as courts or patent offices in the United States and abroad could uphold the validity of any such patents. If we were to challenge the validity of any issued United States patent in court, we would need to overcome a statutory presumption of validity that attaches to every United States patent. This means that in order to prevail, we would have to present clear and convincing evidence as to the invalidity of the patent’s claims. If we are unable to do so, we may be forced to delay the launch of our product candidates or launch at the risk of litigation for patent infringement, which may have a material adverse effect on our business and results of operations.
If a third-party claims that we or any collaborators of ours infringe their intellectual property rights, we may have to defend litigation or administrative proceedings which may be costly whether we win or lose, and which could result in a substantial diversion of our financial and management resources. If we are found to infringe a third party’s intellectual property rights, we could be required to obtain a license from such third party to continue developing and marketing our drug candidates and technology. However, we may not be able to obtain any required license on commercially reasonable terms, or at all. Even if we were able to obtain such a license, it could be granted on non-exclusive terms, thereby providing our competitors and other third parties access to the same technologies licensed to us. Without such a license, we could be forced, including by court order, to cease developing and commercializing the infringing technology or drug candidates. In addition, we could be found liable for monetary damages, including treble damages and attorney’s fees if we are found to have willfully infringed such third-party patent rights. A finding of infringement could prevent us from commercializing our drug candidates or force us to cease some of our business operations, which could materially harm our business.
No assurance can be given that patents issued to third parties do not exist, have not been filed, or could not be filed or issued, which contain claims covering their products, technology or methods that may encompass all or a portion of our products and methods. Given the number of patents issued and patent applications filed in our technical areas or fields, we believe there is a risk that third parties may allege they have patent rights encompassing our products or methods.
Other products or product candidates that we may in-license or acquire could be subject to similar risks and uncertainties.
We may need to license certain intellectual property from third parties, and such licenses may not be available or may not be available on commercially reasonable terms.
A third party may hold intellectual property, including patent rights that are important or necessary to the development and commercialization of our products. It may be necessary for us to use the patented or proprietary technology of third parties to commercialize our products, in which case we would be required to obtain a license from these third parties, whom may or may not be interested in granting such a license, on commercially reasonable terms, in which case our business could be harmed, possibly materially. For example, we engage extensively with third parties, including academic institutions, to conduct non-clinical and clinical research on our product and product candidates. While we seek to ensure all material transfer and service agreements governing this research provide us with favorable terms covering newly generated intellectual property, a general principle under which much of this research with academic institutions is conducted provides third-party ownership of newly generated intellectual property, with an exclusive option available for us to obtain a license to such intellectual property. Through the conduct of this research, it is possible that valuable intellectual property could be developed by a third party, which we will then need to license in order to better develop or commercialize our products. No assurance can be given that we will be able to successfully negotiate such a license on commercially reasonable terms, or at all. Further, should we fail to successfully negotiate a license to such intellectual property, most institutions are then free to license such intellectual property to any other third party, including potentially direct competitors of ours. Should we fail to adequately secure a license to any newly generated intellectual property, our ability to successfully develop or commercialize our products may be hindered, possibly materially.
If we are unable to protect the confidentiality of our trade secrets, our business and competitive position may be harmed.
In addition to the protection afforded by patents, we rely upon unpatented trade secret protection, unpatented know-how and continuing technological innovation to develop and maintain our competitive position. With respect to the building of our proprietary compound library, we consider trade secrets and know-how to be our primary intellectual property. We seek to protect our proprietary technology and processes, in part, by entering into confidentiality agreements with our collaborators, scientific advisors, employees and consultants, and invention assignment agreements with our consultants and employees. We may not be able to prevent the unauthorized disclosure or use of our technical know-how or other trade secrets by the parties to these agreements, however, despite the existence of confidentiality agreements and other contractual restrictions. Monitoring unauthorized uses and disclosures is difficult, and we do not know whether the steps we have taken to protect our proprietary technologies will be effective. If any of the collaborators, scientific advisors, employees and consultants who are parties to these agreements breach or violate the terms of any of these agreements, we may not have adequate remedies for any such breach or violation, and we could lose our trade secrets as a result. Enforcing a claim that a third party illegally obtained and is using our trade secrets, like patent litigation, is expensive and time-consuming, and the outcome is unpredictable. In addition, courts outside the United States are sometimes less willing to protect trade secrets.
Our trade secrets could otherwise become known or be independently discovered by our competitors. Competitors could purchase our drug candidates and attempt to replicate some or all of the competitive advantages we derive from our development efforts, willfully infringe our intellectual property rights, design around our protected technology or develop their own competitive technologies that fall outside of our intellectual property rights. If any of our trade secrets were to be lawfully obtained or independently developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, from using that technology or information to compete with us. If our trade secrets are not adequately protected so as to protect our market against competitors’ drugs, our competitive position could be adversely affected, as could our business.
We may be subject to damages resulting from claims that we or our employees have wrongfully used or disclosed alleged trade secrets of our competitors or are in breach of non-competition or non-solicitation agreements with our competitors.
We could in the future be subject to claims that we or our employees have inadvertently or otherwise used or disclosed alleged trade secrets or other proprietary information of former employers or competitors. Although we try to ensure that our employees and consultants do not use the intellectual property, proprietary information, know-how or trade secrets of others in their work for us, we may in the future be subject to claims that we caused an employee to breach the terms of his or her non-competition or non-solicitation agreement, or that we or these individuals have, inadvertently or otherwise, used or disclosed the alleged trade secrets or other proprietary information of a former employer or competitor. Litigation may be necessary to defend against these claims. Even if we are successful in defending against these claims, litigation could result in substantial costs and could be a distraction to management. If our defenses to these claims fail, in addition to requiring us to pay monetary damages, a court could prohibit us from using technologies or features that are essential to our drug candidates, if such technologies or features are found to incorporate or be derived from the trade secrets or other proprietary information of the former employers. An inability to incorporate such technologies or features would have a material adverse effect on our business and may prevent us from successfully commercializing our drug candidates. In addition, we may lose valuable intellectual property rights or personnel as a result of such claims. Moreover, any such litigation or the threat thereof may adversely affect our ability to hire employees or contract with independent sales representatives. A loss of key personnel or their work product could hamper or prevent our ability to commercialize our drug candidates, which would have an adverse effect on our business, results of operations and financial condition.
Risks Related to Our Business Organization and Governance, Strategy, Employees and Growth Management
If we fail to attract and keep key management, commercial, and clinical development personnel, we may be unable to successfully develop or commercialize our product and product candidates.
We are highly dependent on the research and development, commercialization, manufacturing, quality, financial and legal expertise of our senior management team as well as the other principal members of our management. Although we have entered into an employment agreement with our chief executive officer and employment letters with our senior managers, each of our executive officers may terminate their employment with us at any time. We do not maintain key person insurance for any of our executives or other employees. In addition, we rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our research and development and commercialization strategy. Our consultants and advisors may be employed by employers other than us and may have commitments under consulting or advisory contracts with other entities that may limit their availability to us. If we are unable to continue to attract and retain high quality personnel, our ability to pursue our growth strategy will be limited.
Recruiting and retaining qualified scientific, clinical, manufacturing and medical affairs, and commercial personnel, particularly in MS, will be critical to our success. The loss of the services of our chief executive officer or other key employees could impede the achievement of our research, development and commercialization objectives and seriously harm our ability to successfully implement our business strategy. Furthermore, replacing key employees may be difficult and may take an extended period of time because of the limited number of individuals in our industry with the breadth of skills and experience required to successfully develop, gain regulatory approval of and commercialize products. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate these key personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies for similar personnel. If we are not able to attract and retain the necessary personnel to accomplish our business objectives, we may experience constraints that will significantly impede the achievement of our development and commercialization objectives, our ability to raise additional capital, and our ability to implement our business strategy.
We will need to develop and expand our business, and we may encounter difficulties in managing this development and expansion, which could disrupt our operations.
We may attempt to expand our business by acquiring additional businesses or drugs, forming strategic alliances or creating joint ventures with third parties. We may encounter numerous difficulties in developing, manufacturing, and marketing any new products resulting from any such arrangement or transaction that may delay or prevent us from realizing their expected benefits. If we are unable to successfully integrate such acquired businesses with our existing operations and company culture, we may never realize the benefits of such acquisitions or strategic alliances. We cannot assure you that, following any such transaction, we will achieve the expected synergies to justify the transaction.
As of November 5, 2024, we had 319 employees. To manage our anticipated future growth and focus in neurology and immunology, we must continue to implement and improve our managerial, operational and financial systems, and continue to recruit and train additional qualified personnel. Also, our management may need to divert a disproportionate amount of its attention away from its day-to-day activities and devote a substantial amount of time to managing these activities. Due to our limited resources, we may not be able to effectively manage the expansion and shift of our operations or recruit and train additional qualified personnel. This may result in weaknesses in our infrastructure, give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity among remaining employees. If our management is unable to effectively manage our transition to a strategy primarily focused on neurology and immunology, our expenses may increase more than expected our ability to generate or increase our revenue could be reduced and we may not be able to implement our business strategy. Our future financial performance and our ability to commercialize our drug candidates, if approved, and compete effectively will depend, in part, on our ability to effectively manage the future development and changes to our business.
Additionally, to help manage the evolving needs, we may utilize the services of outside vendors or consultants to perform tasks including clinical trial management, statistics and analysis, regulatory affairs, formulation development, chemistry, manufacturing, controls, and other pharmaceutical development functions. Our growth strategy may also entail expanding our group of contractors or consultants to implement these tasks going forward. Because we rely on a substantial number of consultants, effectively outsourcing many key functions of our business, we will need to be able to effectively manage these consultants to ensure that they successfully carry out their contractual obligations and meet expected deadlines. However, if we are unable to effectively manage our outsourced activities or if the quality or accuracy of the services provided by consultants is compromised for any reason, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain regulatory approval for our product candidates or otherwise advance our business. There can be no assurance that we will be able to manage our existing consultants or find other competent outside contractors and consultants on economically reasonable terms, or at all. If we are not able to effectively expand our organization by hiring new employees and expanding our groups of consultants and contractors when needed, we may be unable to successfully implement the tasks necessary to achieve our research, development and commercialization goals.
Certain anti-takeover provisions in our governing documents and Delaware law could make a third-party acquisition of us difficult. This could limit the price investors might be willing to pay in the future for our common stock.
Certain provisions in our amended and restated certificate of incorporation and restated bylaws may make it more difficult for a third party to acquire us or discourage a third party from attempting to acquire or control us and may limit the price that certain investors might be willing to pay in the future for shares of our common stock. For example, our amended and restated certificate of incorporation allows us to issue preferred stock without the approval of our stockholders, the issuance of which could decrease the amount of earnings and assets available for distribution to, or affect the rights and powers, including voting rights, of our common stockholders. In certain circumstances, such issuance could have the effect of decreasing the market price of our common stock. In addition, our restated bylaws eliminate the right of stockholders to call a special meeting of stockholders, which could make it more difficult for stockholders to effect certain corporate actions. Any of these provisions could also have the effect of delaying or preventing a change in control.
Our ability to utilize our net operating loss carryforwards and certain other tax attributes may be limited.
Under Section 382 of the Internal Revenue Code of 1986, as amended, if a corporation undergoes an ownership change (generally defined as a greater than 50% change (by value) in the ownership of its equity over a three-year period), the corporation’s ability to use its pre-change net operating loss carryforwards and certain other pre-change tax attributes to offset its post-change income may be limited. We may have experienced such ownership changes in the past, and we may experience ownership changes in the future as a result of shifts in our stock ownership, some of which are outside our control. As of December 31, 2023, we had federal net operating loss carryforwards of approximately $1.4 billion, and our ability to utilize those net operating loss carryforwards could be limited by an ownership change as described above, which could result in increased tax liability to us. In addition, pursuant to the Tax Act, we may not use net operating loss carry-forwards to reduce our taxable income in any year by more than 80%, and we may not carry back any net operating losses to prior years. On March 27, 2020, the Coronavirus Aid, Relief, and Economic Security (CARES) Act was signed by President Trump. Certain provisions of the CARES Act alter the rules regarding net-operating losses for such losses arising in 2018, 2019 and 2020. Such losses may be carried back for five years. We cannot assure you, however, of our ability to utilize these favorable offset rules within the applicable time period. These rules apply regardless of the occurrence of an ownership change.
Certain of our executive officers, directors, principal stockholders and their affiliates maintain the ability to exercise significant influence over our company and all matters submitted to stockholders for approval.
Certain of our executive officers, directors and stockholders own more than 5% of our outstanding common stock and, together with their affiliates and related persons, beneficially own a significant percentage of our capital stock. If these stockholders were to choose to act together, they would be able to influence our management and affairs and the outcome of matters submitted to our stockholders for approval, including the election of directors and any sale, merger, consolidation, or sale of all or substantially all of our assets. This concentration of voting power could delay or prevent an acquisition of our company on terms that other stockholders may desire. In addition, this concentration of ownership might adversely affect the market price of our common stock by:
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delaying, deferring or preventing a change of control of us; |
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impeding a merger, consolidation, takeover or other business combination involving us; or |
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discouraging a potential acquirer from making a tender offer or otherwise attempting to obtain control of us. |
Our internal information technology systems, or those of our third-party CROs, CMOs, or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our drug candidates’ development programs and our commercialization of any products for which we receive regulatory approval.
Despite the implementation of security measures, our internal information technology systems and those of our third-party CROs, CMOs, and other contractors and consultants are vulnerable to damage from computer viruses, unauthorized access, cyber-attacks or cyber-intrusions over the Internet, natural disasters, terrorism, war and telecommunication and electrical failures. Although we have been the targets of cyber-attacks and cyber-intrusions, the impact on our operations and financial condition has not been material. We expect such cybersecurity threats to continue and become more sophisticated, even more so due to the conflict between Russia and Ukraine. A significant cyber-attack or cyber-intrusion could cause our systems to fail, leakage of confidential information, or business interruption, which could result in a material disruption of our operations, financial loss, or reputational harm. For example, the loss of clinical trial data for our drug candidates could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. We have invested in protections and monitoring practices of our data and information technology systems to reduce these risks and expect to continue do so as our information technology systems increase in magnitude and complexity. However, there can be no assurance that our efforts and investments will prevent breakdowns or breaches in our systems that could adversely affect our business.
Unfavorable global economic conditions could adversely affect our business, financial condition or results of operations.
Our results of operations could be adversely affected by general conditions in the global economy and in the global financial markets. Key national economies, including the United States, have been affected from time to time by economic downturns or recessions, supply chain constraints, rising inflation, restricted credit, poor liquidity, reduced corporate profitability, debt, equity and foreign exchange market volatility, bankruptcies, rising interest rates, unemployment rates and overall uncertainty with respect to the economy. Increasing interest rates in the United States to respond to inflationary pressures and market volatility, as well as the government closures of Silicon Valley Bank and Signature Bank and liquidity concerns at other financial institutions, could negatively impact our results of operations and financial condition. In addition, increased interest rates or a general economic downturn or recession could reduce our ability to raise additional capital when needed on acceptable terms, if at all. A weak or declining economy, supply disruptions or international trade disputes could also strain our third-party suppliers, possibly resulting in supply disruption.
Likewise, the capital and credit markets may be adversely affected by the conflicts between Russia and Ukraine and Israel and Hamas, the possibility of wider European, Middle Eastern or other geopolitical tensions, and the global sanctions imposed in response thereto. Other international events such as trade disputes, separatist movements, leadership changes and political and military conflicts could also adversely affect global financial activity and markets and could negatively affect the U.S. economy. These conditions could result in decreased economic activity, heightened risk of cyberattacks and inflation, as well as impact our ability to raise capital. Additionally, the Federal Reserve Board (FRB) and other major central banks have been consistently removing or reducing monetary accommodation, increasing the risk of recession and also potentially negatively impacting asset values and credit spreads that were boosted by extraordinary monetary stimulus. A severe or prolonged economic downturn could result in a variety of risks to our business, including, weakened demand for our drug candidates and our ability to raise additional capital when needed on acceptable terms, if at all. A weak or declining economy could also strain our suppliers, possibly resulting in supply disruption, or cause our customers to delay making payments for our marketed product and services. We cannot anticipate all of the ways in which the foregoing, and the current economic climate and financial market conditions, could adversely impact our business.
Our employees, principal investigators, CROs, CMOs and consultants may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading, which could have a material adverse effect on our business.
We are exposed to the risk that our employees, principal investigators, CROs, CMOs, and consultants may engage in fraudulent conduct or other illegal activity. Misconduct by these parties could include intentional failures to comply with FDA regulations, provide accurate information to the FDA, comply with manufacturing standards we have established, comply with federal and state healthcare fraud and abuse laws and regulations, report financial information or data accurately or disclose unauthorized activities to us. In particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Activities subject to these laws also involve the improper use of information obtained in the course of clinical trials or creating fraudulent data in our preclinical studies or clinical trials, which could result in regulatory sanctions and cause serious harm to our reputation. We have adopted a code of ethics applicable to all of our employees and have implemented a compliance program, but it is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations. In addition, we are subject to the risk that a person could allege such fraud or other misconduct, even if none occurred. If any such actions are instituted against us, regardless of the outcome, our reputation and our business may suffer. If we are not successful in defending ourselves or asserting our rights, those actions could lead to imposition of civil, criminal and administrative penalties, damages, monetary fines, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations, any of which could adversely affect our ability to operate our business.
We may acquire businesses or drugs, or form strategic alliances, in the future, and we may not realize the benefits of such acquisitions.
We may acquire additional businesses or drugs, form strategic alliances or create joint ventures with third parties that we believe will complement or augment our existing business. If we acquire businesses with promising markets or technologies, we may not be able to realize the benefit of acquiring such businesses if we are unable to successfully integrate them with our existing operations and company culture. We may encounter numerous difficulties in developing, manufacturing and marketing any new products resulting from a strategic alliance or acquisition that delay or prevent us from realizing their expected benefits or enhancing our business. We cannot assure you that, following any such acquisition, we will achieve the expected synergies to justify the transaction.
We may be subject to adverse legislative or regulatory tax changes that could negatively impact our financial condition.
The rules dealing with U.S. federal, state and local income taxation are constantly under review by persons involved in the legislative process and by the IRS and the U.S. Treasury Department. Changes to tax laws (which changes may have retroactive application) could adversely affect our stockholders or us. In recent years, many such changes have been made and changes are likely to continue to occur in the future. We cannot predict whether, when, in what form, or with what effective dates, tax laws, regulations and rulings may be enacted, promulgated or decided, which could result in an increase in our, or our stockholders, tax liability or require changes in the manner in which we operate in order to minimize increases in our tax liability.
On December 22, 2017, legislation commonly referred to as the Tax Act was signed into law and is generally effective after December 31, 2017. The Tax Act makes significant changes to the United States federal income tax rules for taxation of individuals and business entities. Most of the changes applicable to individuals are temporary and apply only to taxable years beginning after December 31, 2017 and before January 1, 2026. For corporations, the Tax Act reduces the top corporate income tax rate to 21% and repeals the corporate alternative minimum tax, limits the deduction for net interest expense, limits the deduction for net operating losses and eliminates net operating loss carrybacks, modifies or repeals many business deductions and credits, shifts the United States toward a more territorial tax system, and imposes new taxes to combat erosion of the U.S. federal income tax base. The Tax Act makes numerous other large and small changes to the federal income tax rules that may affect potential investors and may directly or indirectly affect us. We continue to examine the impact this tax reform legislation may have on our business. However, the effect of the Tax Act on us, whether adverse or favorable, is uncertain, and may not become evident for some period of time. This document does not discuss such legislation or the manner in which it might affect us or purchasers of our common stock. Prospective investors are urged to consult with their legal and tax advisors with respect to the Tax Act and any other regulatory or administrative developments and proposals, and their potential effects on them based on their unique circumstances.
As another example, in August 2022, the Inflation Reduction Act of 2022 (the IRA) was enacted, and, among other things, included a new 15% alternative minimum tax on the adjusted financial statement income of certain large corporations for tax years beginning after December 31, 2022. To the extent that such changes have a negative impact on us, including as a result of related uncertainty, these changes could adversely impact our business, results of operations and financial position. On December 14, 2023, President Biden announced that, under the IRA, dozens of pharmaceutical companies are required to pay rebates to Medicare for price hikes on prescription drugs.
Risks Related to Our Common Stock and Being a Publicly Traded Company
Our stock price is, and we expect it to remain, volatile, which could limit investors’ ability to sell our stock at a profit.
The trading price of our common stock has been and is likely to continue to be highly volatile and subject to wide fluctuations in price in response to various factors, many of which are beyond our control. These factors include, among others:
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reception and success of BRIUMVI in the U.S. market; |
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reception and success of BRIUMVI in the German market and the anticipated launch of BRIUMVI in additional European markets; |
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publicity regarding actual or potential clinical results relating to our product or products under development by our competitors or us; |
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delay or failure in initiating, completing or analyzing nonclinical or clinical trials or the unsatisfactory design or results of these trials; |
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achievement or rejection of regulatory approvals by us or our competitors; |
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any delay in our regulatory review for products and product candidates we may develop, and any adverse development or perceived adverse development with respect to the applicable regulatory authority’s review of such filings, including without limitation a change to the projected approval date, scheduling of an advisory committee meeting or issuance of a “refusal to file” letter; |
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announcements of technological innovations or new commercial products by our competitors or us; |
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developments concerning proprietary rights, including patents; |
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developments concerning our collaborations; |
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● | actual or anticipated variations in our operating results due to the level of development expenses and other factors; | |
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regulatory developments in the United States and foreign countries; |
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economic, political and market conditions or other crises and other external factors such as the disruptions in the global economy caused by health epidemics, the conflict between Russia and Ukraine, and the Israel-Hamas war; |
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period-to-period fluctuations in our revenues and other results of operations; |
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failure to meet our revenue projections or guidance; |
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changes in financial estimates by securities analysts; |
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sales of our common stock by us; and |
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● | the occurrences of any of the other risks described in this Quarterly Report. |
We will not be able to control many of these factors, and we believe that period-to-period comparisons of our financial results will not necessarily be indicative of our future performance.
In addition, the stock market in general, and the market for biotechnology companies in particular, has experienced extreme price and volume fluctuations that may have been unrelated or disproportionate to the operating performance of individual companies. These broad market and industry factors may seriously harm the market price of our common stock, regardless of our operating performance. As a result of this volatility, investors may not be able to sell their common stock at or above the price paid for the shares.
We are subject to risks related to corporate social responsibility and reputational matters.
Our reputation and the reputation of our brands, including the perception held by our customers, end-users, business partners, investors, other key stakeholders and the communities in which we do business are influenced by various factors. There is an increased focus from our stakeholders on Environmental, Social, and Governance (ESG) practices and disclosure - and if we fail, or are perceived to have failed, in any number of ESG matters, such as environmental stewardship, inclusion and diversity, workplace conduct and support for local communities, or if we fail, or are perceived to have failed, to effectively respond to changes in legal or regulatory requirements concerning climate change or other sustainability concerns, our reputation or the reputation of our brands may suffer. Such damage to our reputation and the reputation of our brands may negatively impact our business, financial condition and results of operations. In addition, negative or inaccurate postings or comments on social media or networking websites about the Company or our brands could generate adverse publicity that could damage our reputation or the reputation of our brands. If we are unable to effectively manage real or perceived issues, including concerns about product quality, safety, corporate social responsibility or other matters, sentiments toward the Company or our products could be negatively impacted, and our financial results could suffer.
Climate change or legal, regulatory or market measures to address climate change may negatively affect our business, supply chain, results of operations, financial condition and growth prospects.
We believe that climate change has the potential to negatively affect our business, results of operations, financial condition and growth prospects. The adverse impacts of climate change include (i) physical risks such as increased frequency and severity of natural disasters and extreme weather events such as hurricanes, tornados, wildfires (exacerbated by drought), flooding, and extreme heat, (ii) risks in transitioning to a low-carbon economy (such as additional legal or regulatory requirements, changes in technology, market risk and reputational risk) and (iii) social and human effects (such as population dislocations and harm to health and well-being).
Since we currently rely on single contract manufacturers to produce our commercial products, extreme weather and sea level rise pose physical risks to the facilities of our manufacturing partners. Such risks include losses incurred as a result of physical damage to facilities, loss or spoilage of inventory, and operational disruptions caused by such natural disasters and extreme weather events. Loss of access to the facilities of our manufacturing partners may result in increased costs, delays in the development of our products or interruption of our business operations. Any disaster recovery and business continuity plans that our we or our third-party manufacturers have in place may prove inadequate in the event of a serious natural disaster or similar event. We may incur substantial expenses as a result of the limited nature of these disaster recovery and business continuity plans, which could have a material adverse effect on our business.
In addition, the long-term effects of climate change on general economic conditions and the pharmaceutical industry in particular are unclear and may heighten or intensify the existing risk of natural disasters. As part of our risk management policy, we maintain insurance coverage at levels that we believe are appropriate for our business. However, we cannot assure you that such insurance coverage will be sufficient to satisfy any damages and losses we may directly or indirectly incur. If the manufacturing facilities of our third-party manufacturers are unable to operate for any reason, even for a short period of time, any or all of our research and development programs or commercialization efforts may be harmed. Any material interruption could have a material and adverse effect on our business.
New legal or regulatory requirements may also be enacted to prevent, mitigate, or adapt to the implications of a changing climate and its effects on the environment. These regulations, which may differ across jurisdictions, could result in our manufacturing partners being subject to new or expanded carbon pricing or taxes, increased compliance costs, restrictions on greenhouse gas emissions, investment in new technologies, increased carbon disclosure and transparency, upgrading of facilities to meet new building codes, and the redesign of utility systems, which could increase our third-party manufacturers’ operating costs, including the cost of electricity and energy used to develop our products. Our supply chain as a whole would likely be subject to these same transitional risks and would likely pass along any increased costs to us.
Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable future, capital appreciation, if any, will likely be the sole source of gain for our stockholders.
We have never declared or paid cash dividends on our capital stock. We currently intend to retain all of our future earnings, if any, to finance the growth and development of our business. However, any future determination relating to the use of our future earnings, if any, will be made at the discretion of the Board of Directors and will depend on a number of factors, including capital requirements, financial conditions, future prospects, contractual restrictions and covenants and other factors that the Board of Directors may deem relevant. In addition, under the Financing Agreement, with Blue Owl Capital and HealthCare Royalty, we are currently restricted from paying cash dividends, and we expect these restrictions to continue in the future. Furthermore, the terms of any future debt agreements may continue to preclude us from paying dividends. As a result, capital appreciation, if any, of our common stock will be likely the sole source of gain for our stockholders for the foreseeable future.
An active trading market for our common stock may not be sustained, and investors may not be able to resell their shares at or above the price they paid.
Although we have listed our common stock on the Nasdaq Capital Market, an active trading market for our shares may not be sustained. In the absence of an active trading market for our common stock, investors may not be able to sell their common stock at or above the price at which they acquired their shares or at the time that they would like to sell. An inactive trading market may also impair our ability to raise capital to continue to fund operations by selling shares and may impair our ability to acquire other companies or technologies by using our shares as consideration.
If equity research analysts do not publish research or reports about our business or if they publish negative evaluations of or downgrade our common stock, the price of our common stock could decline.
The trading market for our common stock relies in part on the research and reports that equity research analysts publish about us or our business. We do not control these analysts. If one or more of the analysts covering our business downgrade their evaluations of our common stock, the price of our common stock could decline. If one or more of these analysts cease to cover our common stock, we could lose visibility in the market for our common stock, which in turn could cause our common stock price to decline.
We incur significant increased costs as a result of operating as a public company, and our management is required to devote substantial time to compliance initiatives.
As a public company, we incur significant legal, accounting and other expenses under the Sarbanes-Oxley Act of 2002, as well as rules subsequently implemented by the SEC, and the rules of any stock exchange on which we are listed. These rules impose various requirements on public companies, including requiring establishment and maintenance of effective disclosure and financial controls and appropriate corporate governance practices. Our team has devoted and will continue to devote a substantial amount of time to these compliance initiatives. Moreover, these rules and regulations increase our legal and financial compliance costs and make some activities more time-consuming and costly.
The Sarbanes-Oxley Act of 2002 requires, among other things, that we maintain effective internal control over financial reporting and disclosure controls and procedures. As a result, we are required to periodically perform an evaluation of our internal control over financial reporting to allow management to report on the effectiveness of those controls, as required by Section 404 of the Sarbanes-Oxley Act. Additionally, our independent auditors are required to perform a similar evaluation and report on the effectiveness of our internal control over financial reporting. These efforts to comply with Section 404 will require the commitment of significant financial and managerial resources. While we anticipate maintaining the integrity of our internal control over financial reporting and all other aspects of Section 404, we cannot be certain that a material weakness will not be identified when we test the effectiveness of our control systems in the future. If a material weakness is identified, we could be subject to sanctions or investigations by the SEC or other regulatory authorities, which would require additional financial and management resources, costly litigation or a loss of public confidence in our internal control over financial reporting, which could have an adverse effect on the market price of our stock and result in a loss of investor confidence in our financial reports.
We have identified a material weakness in our internal control over financial reporting related to non-routine share-based payment awards, and if our remediation of such material weakness is not effective, or if we fail to develop and maintain an effective system of disclosure controls and internal control over financial reporting, our ability to produce timely and accurate financial statements or comply with applicable laws and regulations could be impaired.
During the preparation of our unaudited condensed consolidated financial statements for the period ended June 30, 2024, we identified a material weakness in our internal control over financial reporting related to controls around non-routine share-based payment awards. A material weakness is a deficiency, or combination of deficiencies, in internal control over financial reporting, such that there is a reasonable possibility that a material misstatement of the annual or interim financial statements will not be prevented or detected on a timely basis. The material weakness identified pertains to certain process-level controls over share-based payment awards for stock awards with market conditions. To remediate the material weakness in the Company’s internal control over financial reporting, the Company will implement enhanced risk assessment procedures to ensure that all non-routine share-based grants are appropriately identified and evaluated. Further, the Company will design additional preventative controls around non-routine share-based payment awards to ensure the appropriate recognition and measurement of such awards at the grant date.
The material weakness will not be considered remediated until the applicable controls operate for a sufficient period of time to enable management and our independent registered public accounting firm to test and to conclude that these controls are operating effectively. Until this material weakness is remediated, we plan to continue to perform additional analyses and other procedures to ensure that our consolidated financial statements are prepared in accordance with GAAP.
If we are unable to further implement and maintain effective internal control over financial reporting or disclosure controls and procedures, our ability to record, process and report financial information accurately, and to prepare financial statements within required time periods could be adversely affected, which could subject us to litigation or investigations requiring management resources and payment of legal and other expenses, negatively affect investor confidence in our financial statements and adversely impact our stock price. If we are unable to assert that our internal control over financial reporting is effective, investors may lose confidence in the accuracy and completeness of our financial reports, the market price of our common stock could be adversely affected and we could become subject to litigation or investigations by Nasdaq, the SEC or other regulatory authorities, which could require additional financial and management resources.
Furthermore, we cannot assure you that the measures we have taken to date, and actions we may take in the future, will be sufficient to remediate the control deficiencies that led to our material weakness in our internal control over financial reporting or that they will prevent or avoid potential future material weaknesses. Our current controls and any new controls that we develop may become inadequate because of changes in conditions in our business. Further, weaknesses in our disclosure controls and internal control over financial reporting may be discovered in the future. Any failure to develop or maintain effective controls or any difficulties encountered in their implementation or improvement could harm our operating results or cause us to fail to meet our reporting obligations and may result in a restatement of our financial statements for prior periods. Any failure to implement and maintain effective internal control over financial reporting could adversely affect the results of periodic management evaluations.
Volatility in the price of our common stock may subject us to securities and shareholder derivative litigation, which could cause us to incur substantial costs and divert management’s attention, financial resources and other company assets.
In the past, securities class action and shareholder derivative litigation has often been brought against a company following periods of volatility in the market price of its securities. This risk is especially relevant for us because pharmaceutical companies have experienced significant stock price volatility in recent years. Past lawsuits and any future lawsuits to which we may become a party are subject to inherent uncertainties and will likely be expensive and time-consuming to investigate, defend, and resolve, and will divert our management’s attention and financial and other resources. The outcome of litigation is necessarily uncertain, and we could be forced to expend significant resources in the defense of these and other suits in which we may not prevail. Any litigation to which we are a party may result in an onerous or unfavorable judgment that may not be reversed upon appeal or in payments of substantial monetary damages or fines, or we may decide to settle this or other lawsuits on similarly unfavorable terms, which could adversely affect our business, financial condition, results of operations or stock price.
Future sales of our common stock, including by us or our directors and executive officers or shares issued upon the exercise of currently outstanding options, could cause our stock price to decline.
A substantial portion of our outstanding common stock can be traded without restriction at any time. In addition, a portion of our outstanding common stock is currently restricted as a result of federal securities laws but can be sold at any time subject to applicable volume limitations. As such, sales of a substantial number of shares of our common stock in the public market could occur at any time. These sales, or the perception in the market that the holders of a large number of shares intend to sell shares, by us or others, could reduce the market price of our common stock or impair our ability to raise adequate capital through the sale of additional equity securities. In addition, we have a significant number of shares that are subject to outstanding options. The exercise of these options and the subsequent sale of the underlying common stock could cause a further decline in our stock price. These sales also might make it difficult for us to sell equity securities in the future at a time and at a price that we deem appropriate. We cannot predict the number, timing or size of future issuances or the effect, if any, that any future issuances may have on the market price for our common stock.
We cannot guarantee that our stock repurchase program will be further consummated or will enhance stockholder value, our share repurchase program could affect the price of our common stock and increase volatility and may be suspended or terminated at any time, which may result in a decrease in the trading price of our common stock.
In August 2024, we announced that our Board of Directors had authorized a share repurchase program of up to $100 million of our outstanding shares of common stock. We intend to repurchase shares of our common stock from time to time, as authorized by our Board of Directors, through open market purchases, in privately negotiated transactions or by other means, including through the use of trading plans intended to qualify under Rule 10b5-1 under the Exchange Act, in accordance with applicable securities laws and other restrictions. The timing and the amount of stock repurchases in the share repurchase program will be determined by our management, based on its evaluation of factors including business and market conditions, corporate and regulatory requirements, and other considerations. The share repurchase program does not have a fixed expiration date, may be suspended or discontinued at any time, and does not obligate us to acquire any amount of our common stock.
There can be no assurance of any future share repurchases or share repurchase program authorizations. The timing and manner of any share repurchases will depend upon, among other factors, our cash balances and potential future capital requirements, results of operations and financial condition, alternative investment opportunities, restrictions under any of our agreements, business economic and market conditions, corporate and regulatory requirements the price of our Common Stock on the NASDAQ Capital Market, and other factors that we may deem relevant. We can provide no assurance that we will repurchase shares of our Common Stock at favorable prices, if at all
Repurchases pursuant to our share repurchase program could affect our stock price and increase its volatility or diminish our cash reserves, which may impact our ability to finance our future operations. The existence of a share repurchase program could also cause our stock price to be higher than it would be in the absence of such a program and could potentially reduce the market liquidity for our common stock. There can be no assurance that any repurchases will enhance shareholder value, because the market price of our common stock may decline below the levels at which we repurchased our common stock. Although our share repurchase program is intended to enhance long-term shareholder value, short-term stock price fluctuations could reduce the share repurchase program’s effectiveness.
ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS.
ISSUER PURCHASES OF EQUITY SECURITIES |
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(c) |
(d) |
Total Number of Shares (or Units) Purchased |
Average Price Paid per Share (or Unit) |
Total Number of Shares (or Units) Purchased as Part of Publicly Announced Plans or Programs |
Maximum Number (or Approximate Dollar Value) of Shares (or Units) that May Yet Be Purchased Under the Plans or Programs* |
|
July 2024 | - | - | - | - |
(July 1st to July 31st, 2024) | ||||
August 2024 |
9,000 |
$23.58 |
9,000 |
$99,787,788 |
(August 1st to August 31st, 2024) |
||||
September 2024 |
83,301 |
$23.25 |
83,301 |
$97,851,216 |
(September 1st to September 30th, 2024) |
||||
Total | 92,301 | $23.28 | 92,301 | $97,851,216 |
*Transaction fees are excluded.
On August 2, 2024, the Company announced that its Board of Directors had authorized and approved a share repurchase program for up to $100 million of the currently outstanding shares of the Company’s common stock. Repurchases under the program may be made using open market purchases, privately negotiated transactions, block purchases or other methods in accordance with applicable federal securities laws, including Rule 10b-18 of the Exchange Act. The share repurchase program does not have a fixed expiration date, may be suspended or discontinued at any time, and does not obligate us to acquire any particular amount of our common stock.
ITEM 3. DEFAULTS OF SENIOR SECURITIES.
None.
ITEM 4. MINE SAFETY DISCLOSURES.
Not applicable.
Securities Trading Plans of Directors and Executive Officers
During the three months ended September 30, 2024,
of our directors or executive officers adopted or terminated any contract, instruction or written plan for the purchase or sale of the Company’s securities that was intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) or any “non-Rule 10b5-1 trading arrangement,” as each term is defined in Item 408(a) of Regulation S-K.
The exhibits listed on the Exhibit Index are included with this report.
10.1# | Master Services Agreement between FUJIFILM DIOSYNTH BIOTECHNOLOGIES NORTH CAROLINA, INC, FUJIFILM DIOSYNTH BIOTECHNOLOGIES DENMARK APS and TG Therapeutics, Inc., effective October 8, 2024. |
31.1* |
Certification of Chief Executive Officer pursuant to Rule 13a-14(a)/15d-14(a), as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002, dated November 7, 2024. |
31.2* |
|
32.1** |
|
32.2** |
Certification of Chief Financial Officer pursuant to 18 U.S.C. 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, dated November 7, 2024. |
101* |
The following financial information from the Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2024, formatted in Inline Extensible Business Reporting Language (iXBRL): (i) the Condensed Consolidated Balance Sheets, (ii) the Condensed Consolidated Statements of Operations, (iii) the Condensed Consolidated Statements of Changes in Stockholders’ Equity, (iv) the Condensed Consolidated Statements of Cash Flows, and (v) Notes to the Condensed Consolidated Financial Statements (filed herewith). |
104* |
Cover Page Interactive Data File (embedded within the Inline XBRL document). |
* Filed herewith.
** Furnished herewith.
# Certain confidential portions of this exhibit have been omitted pursuant to Item 601(b) of Regulation S-K.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
TG THERAPEUTICS, INC. |
||
Date: November 7, 2024 |
By: |
/s/ Sean A. Power |
Sean A. Power |
||
Chief Financial Officer |
||
Principal Financial and Accounting Officer |