在微軟授權下，我們是否可以存取該項
美國
證券交易委員會
華盛頓特區20549
表格
截止至本季度結束
或
過渡期從 到 .
委員會檔案編號
（依憑章程所載的完整登記名稱）
| ||
(成立或組織的州或其他轄區) | （聯邦稅號） | |
（總部地址） | （郵政編碼） |
(
(註冊人電話號碼，包括區號)
根據法案第12(b)條註冊的證券：
每種類別的名稱 |
| 交易標的（s） |
| 每個註冊交易所的名稱 |
|
|
勾選表示登記者在過去12個月（或所需提交此類文件的更短期間）期間，根據S-t規則405（本章第232.405條）提交了每個互動數據文件。
請用勾選標記來指示：登記者是否為大型加速發行人、加速發行人、非加速發行人、較小的報告公司或新興成長公司。請參見《交易所法》第1202條對「大型加速發行人」、「加速發行人」、「較小的報告公司」和「新興成長公司」的定義：
大型加速文件提交者 | ☐ | 加速檔案提交者 | ☐ |
| |||
☒ | 較小報告公司 | ||
| |||
新興成長型企業 |
如果一家新興成長型企業，請勾選“是”表示註冊人選擇不使用根據證券交易所法第13(a)條所提供的任何新的或修改後的財務會計準則的延長過渡期來遵守。
請勾選表示該申報人是否為外殼公司（如法案第120億2條所定義）。 是
請指示在最近切實可行的日期，申報人各類普通股的股份總數。
|
|
|
普通股類 |
| 截至2024年11月1日的優秀股份 |
普通股，面值$0.00001 |
|
1
風險因素總結
投資於我們普通股的範圍涉及廣泛的風險，您應該在仔細考慮這些風險後才進行投資。在購買我們的普通股之前，建議您仔細閱讀並考慮“項目控制項1A. 風險因素”一節中的一些風險討論。
我們的業務受到多種風險的影響，包括可能阻止我們實現業務目標或對我們的業務、財務狀況、營業收入、現金流和前景造成不利影響的風險，您應在決定是否投資我們的普通股之前考慮這些風險。這些風險在本報告的第26頁開始的“風險因素”一節中有更詳細的討論，包括以下內容：
與我們的財務狀況和資本需求相關的風險
| ● | 我們有有限的運營歷史，僅開展了有限數量的臨床試驗，目前只有有限數量的患者參與我們正在進行的試驗，並且迄今尚未完成任何臨床試驗。我們沒有任何產品獲得商業銷售批准，也沒有產生任何營業收入，這可能使投資者難以評估我們當前業務的狀況、成功的機會和可行性。 |
| ● | 我們自成立以來一直虧損，並未創造任何營業收入。我們預計在可預見的未來將繼續虧損，並可能永遠無法實現或維持盈利。 |
| ● | 我們的營業收入和實現盈利能力很大程度上取決於我們實現與當前或未來產品候選者的發現或識別、臨床前和臨床研究、監管批准和商品化相關的多項目標。 |
| ● | 我們將需要大量額外資本來資助我們的業務和實現我們的目標。如果我們無法及時籌措到資金，或無法接受我們可接受的條款，我們可能被迫延遲、減少或取消我們的研究或產品開發計畫、任何未來的商品化努力或其他業務。 |
| ● | 在COVID-19大流行期間實施的政策，今天仍然存在，可能對我們的業務產生不利影響，包括我們的臨床前開發、臨床試驗和臨床試驗操作。 |
與我們產品候選者開發相關的風險
| ● | 我們極大程度上依賴我們的產品候選者NXP800和NXP900的成功。 |
| ● | 臨床試驗非常昂貴、耗時、難以設計和實施，並涉及不確定的安全性、耐受性和功效結果。此外，較早的臨床前研究和臨床試驗的結果可能無法預測未來臨床前研究或臨床試驗的結果。我們目前或未來的產品候選者在早期或後期臨床試驗中可能沒有良好的結果，如果有的話，也可能無法獲得監管機構的批准。 |
| ● | 如果我們未能證明我們的任何或所有產品候選者的安全性和/或功效，我們可能需要終止開發計劃，這可能損害我們的聲譽和業務。 |
| ● | 藥品產品的開發和商業化受到廣泛的規定，我們可能無法為NXP800、NXP900或任何未來的產品候選者獲得監管機構的批准。 |
| ● | 如果我們無法取得或維持產品候選藥品的監管批准，最終無法商業化其中一個或多個產品，或者在此過程中遇到重大延遲，我們的業務將受到重大損害。 |
3
關於我們對第三方的依賴的風險
| ● | 我們當前和未來產品候選品的製造過程複雜。出於各種原因，我們的第三方製造商可能會遇到困難或中斷，這可能會延遲或完全阻止他們製造我們當前和未來產品候選品用於臨床試驗，如果獲得批准，則用於商業銷售。 |
與管理增長和員工事項相關的風險
| ● | 我們未來的成功取決於我們保留高管和關鍵員工的能力，吸引、留住和激勵合格人才，並管理我們的人力資本。 |
| ● | 我們目前有13名全職員工，需要擴大組織規模和能力。我們可能會在管理這種增長方面遇到困難。 |
與知識產權相關的風險
| ● | 如果我們無法獲得和維持產品和技術的專利保護或其他必要的權利，或者獲得的專利保護範圍不夠廣泛，或者在許可專利下的權利不夠廣泛，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和技術，我們成功商業化產品和技術的能力可能會受到不利影響。 |
4
第I部分-財務信息
項目1。 基本報表
NUVECTIS PHARMA, INC.
簡明資產負債表
(以千美元計算，每股和股數除外)
(未經審計)
| 2021年9月30日 |
| 運營租賃負債： | ||||
2024 | 2023 |
| |||||
資產 |
|
|
|
| |||
流動資產 |
|
|
|
|
| ||
現金及現金等價物 |
| $ | |
| $ | |
|
其他資產 |
| |
| |
| ||
總流動資產 |
| |
| |
| ||
資產總計 |
| $ | |
| $ | |
|
負債和股東權益 |
|
|
|
|
| ||
流動負債 | |||||||
應付賬款 |
| $ | |
| $ | |
|
應計負債 |
| |
| |
| ||
員工薪酬福利 |
| |
| |
| ||
流動負債合計 |
| |
| |
| ||
負債合計 |
| |
| |
| ||
承諾和事務，參見注釋3 |
|
|
|
|
| ||
股東權益，請參閱附註4 |
|
|
|
|
| ||
2,885.3 |
| ||||||
股票認購應收款項。 |
| |
| |
| ||
累積赤字 |
| ( |
| ( |
| ||
總股東權益 |
| |
| |
| ||
負債合計和股東權益 |
| $ | |
| $ | |
|
* |
附註是這些未經審計的基本財務報表的一部分。
5
NUVECTIS PHARMA，INC。
捷凱收購公司二期有限公司
(以千美元計算，每股和股數除外)
(未經審計)
| |||||||||||||
截至9月30日止三個月 | 截至九月三十日的九個月 | ||||||||||||
| 2024 |
| 2023 |
| 2024 |
| 2023 | ||||||
營業費用 |
|
|
| ||||||||||
研發 |
| $ | |
| $ | |
| $ | |
| $ | |
|
一般行政 | |
| | |
| |
| ||||||
|
| ||||||||||||
營業虧損 |
| ( |
| ( |
| ( |
| ( |
| ||||
財務收益 | |
| | |
| |
| ||||||
|
| ||||||||||||
淨損失 |
| $ | ( |
| $ | ( |
| $ | ( |
| $ | ( |
|
歸屬於普通股股東的淨虧損 | $ | ( |
| $ | ( | $ | ( |
| $ | ( |
| ||
每股普通股的基本和稀釋淨損失，詳見附註6 | $ | ( |
| $ | ( | $ | ( |
| $ | ( |
| ||
基本和稀釋後的平均流通股本 | |
| | |
| |
| ||||||
附註是這些未經審計的基本財務報表的一部分。
6
努維克蒂斯製藥公司，INC。
股東權益變動簡明報表
（以千美元計，每股份額除外）
(未經審計)
|
|
|
| ||||||||||
普通股 | 額外的 | 總計 | |||||||||||
0.00001美元的面值 | 實繳 | 累積的 | 股東的 | ||||||||||
| 股份 |
| 金額 |
| 資本 |
| 赤字 |
| 股權 | ||||
2022年12月31日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 | — |
| * |
|
| |
| — | | ||||
限制性股票授予 | | * | — | — | — | ||||||||
優先投資期權的行使 | | * | | | |||||||||
認證股證權行權 | | * | | | |||||||||
本期淨損失 |
| — |
| — |
| - |
| ( |
| ( | |||
2023年3月31日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 | — | * | | — |
| | |||||||
發行受限制股獎項 | | — | — | — | — | ||||||||
行使優先投資期權，減去$的發行成本 | | * | | — | | ||||||||
認證股證權行權 | | * | | — | | ||||||||
發行普通股，減去發行成本$ | | * | | — | | ||||||||
期權的行權 | | * | | — | | ||||||||
本期淨損失 |
| — |
| — |
| - |
| ( |
| ( | |||
2023年6月30日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 | — |
| * | |
| — | | ||||||
發行受限制股獎勵 | | * | — | — |
| — | |||||||
普通股的發行，減去發行成本爲$ | | * | | — | | ||||||||
本期淨損失 |
| — |
| — |
| - |
| ( |
| ( | |||
2023年9月30日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
* | 表示低於1美元美元的金額。 |
附註是這些未經審計的基本財務報表的一部分。
7
NUVECTIS PHARMA, INC.
股東權益變動簡明報表
(美元指數以千爲單位，股份數量除外)
(未經審計)
|
|
|
| ||||||||||
普通股 | 額外的 | 總計 | |||||||||||
面值$0.00001 | 實繳 | 累積的 | 股東的 | ||||||||||
| 股份 |
| 金額 |
| 資本 |
| 赤字 |
| 股權 | ||||
2023年12月31日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 |
| — |
| — |
| |
| — |
| | |||
限制性股票授予 | | — | — | — | — | ||||||||
發行普通股，減去發行成本$ | | * | | — | | ||||||||
本期淨損失 |
| — |
| — |
| — |
| ( |
| ( | |||
2024年3月31日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 |
| — |
| * |
| |
| — | | ||||
發行受限制的股票獎勵 | | * | — | — | — | ||||||||
發行普通股，減去發行成本$ | | * | | — | | ||||||||
本期淨損失 |
| — |
| — |
| — |
| ( |
| ( | |||
2024年6月30日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 |
| — |
| * |
| |
| — |
| | |||
發行受限制的股票獎勵 | | * | — | — | — | ||||||||
普通股股份發行，減去$發行成本 | | * | | — | | ||||||||
本期淨損失 |
| — |
| — |
| — |
| ( |
| ( | |||
2024年9月30日的餘額 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
* | 表示小於1美元的金額。 |
附註是這些未經審計的基本財務報表的一部分。
8
NUVECTIS PHARMA, INC.
精簡現金流量表
(以千美元計算，每股和股數除外)
(未經審計)
截至9月30日的前九個月 | |||||||
| 2024 |
| 2023 | ||||
經營活動產生的現金流量 |
|
|
|
|
| ||
淨損失 |
| $ | ( |
| $ | ( |
|
調整以將虧損調和到經營活動中使用的淨現金： |
| |
| |
| ||
經營性資產和負債變動: |
|
|
|
|
| ||
其他流動資產的（增加）/減少 |
| ( |
| |
| ||
應付賬款減少/增加 | ( | | |||||
應付負債減少 | ( | ( | |||||
應計的薪酬和福利減少 |
| ( |
| ( |
| ||
經營活動使用的淨現金流量 |
| $ | ( |
| $ | ( |
|
投資活動產生的現金流量 |
|
|
|
|
| ||
投資活動產生的淨現金流量 |
|
|
| ||||
籌資活動產生的現金流量 |
|
|
|
|
| ||
普通股發行收益 - 市場發行 |
| $ | |
| $ | |
|
與市場發行相關的發行成本 | ( | ( | |||||
與首次公開發行相關的發行成本 | — | ( | |||||
行使權證、期權和優先投資選項所得款項 | — | | |||||
與行使權證和優先投資選項相關的發行成本 | — | ( | |||||
定向增發相關的發行成本 | — | ( | |||||
籌資活動產生的現金淨額 |
| $ | |
| $ | |
|
現金及現金等價物的(減少)/增加 |
| $ | ( |
| $ | |
|
期初現金及現金等價物 |
| $ | |
| $ | |
|
期末現金及現金等價物 |
| $ | |
| $ | |
|
附註是這些未經審計的基本財務報表的一部分。
9
NUVECTIS PHARMA, INC.
基本報表附註
注1 - 一般：
| a. | Nuvectis Pharma, Inc.（以下簡稱「公司」）於2020年7月27日根據德拉華州法律成立，於2021年5月開始主要業務。 公司的主要執行辦公室位於新澤西州Fort Lee。 |
公司是一家專注於開發創新精準藥物治療腫瘤等嚴重未滿足醫療需要狀況的生物製藥公司。
| b. | 2021年5月，公司與CRt Pioneer Fund（「CRT」）簽訂了全球獨家許可協議（見附註3a）。 2021年8月，公司與蘇格蘭愛丁堡大學達成了全球獨家許可協議，用於公司的第二藥物候選品（見附註3a）。 |
| c. | 2022年2月，公司股票開始在納斯達克交易，代碼爲「NVCT」. |
| d. | 流動性和資本資源 |
公司自成立以來一直出現淨營業虧損，並截至2024年9月30日，累計赤字爲$
在截至2024年9月30日的三個月內，公司總共出售了
截至2024年9月30日的九個月內，公司共出售了
管理層認爲，截至2024年9月30日，公司現有的現金及現金等價物可支持至少12個月的計劃運營，包括這些簡明財務報表發行日期後的時間。
公司需要籌集額外資本以完成旨在開發產品候選藥物的臨床試驗，直至獲得監管和營銷批准。不能保證公司將能夠獲得此類額外融資，或以對公司滿意的條件獲得，並且此融資是否足以滿足其需求。如果公司未能成功獲取足夠資金，這可能迫使公司延遲、限制或減少其產品開發、臨床試驗、商業化努力或其他運營，甚至關閉或清算。
10
注 2 - 重要會計政策:
| a. | 報告範圍 |
附帶的簡明財務報表未經審計。公司的未經審計的簡明財務報表是根據美國通用會計準則（「U.S. GAAP」）編制的，以美元表示，並遵循證券交易委員會（「SEC」）有關中期財務報告的要求。因此，它們不包括美國通用會計準則爲完整財務報表所要求的所有信息和披露，因爲通常需要的某些腳註或其他財務信息可以簡化或省略。未經審計的簡明財務報表是按照已經審計的財務報表的基礎進行準備的。未經審計的簡明財務報表包括公司的賬目。這些附註中對適用指南的任何參考，是指財務會計準則委員會（「FASB」）的會計準則編碼（「ASC」）和會計準則更新（「ASU」）中找到的權威美國通用會計準則。
管理層認爲，未經審計的簡明財務報告包括認爲對中期期間結果的真實陳述所必需的所有正常和經常性調整。截至2024年9月30日的期間結果並不一定代表預期的截至2024年12月31日或任何未來期間的結果。2023年12月31日的簡明資產負債表包含於此處的，是從該日期的已經審計的財務報表導出的，但並不包括美國通用會計準則要求的所有披露。這些未經審計的簡明財務報表應與公司的審計財務報表和相關附註一起閱讀，其中包括公司在2024年3月5日向SEC提交的年度10-k表上的截至2023年12月31日的財年報告。
基礎報表中採用並使用的重要會計政策與上一財政年度的一致。
| b. | 財務報表的編制符合美國一般公認的會計原則，要求管理層進行估計和假設，這些估計和假設影響資產和負債的報告金額和變動以及潛在的資產和負債的披露。實際結果可能與估計的結果不一致。 |
公司財務報表的編制要求管理層進行估計和假設，影響公司財務報表及附註中資產、負債和費用的報告金額。公司財務報表中最重要的估計涉及研發費用的計提、股權獎勵的估值和遞延稅款資產的準備金。這些估計和假設基於當前事實、未來預期以及其他各種因素，被認爲在情況下是合理的，其結果構成了就資產和負債的賬面價值以及從其他來源無法明顯得知的費用錄入的判斷依據。實際結果可能與這些估計有重大和不利的差異。
c. | 公允價值計量 |
公司遵循權威會計指引，其中定義了公允價值，建立了一致的公允價值衡量框架，並擴大了對每個按公允價值計量的主要資產和負債類別的披露，無論是按照重複性還是非重複性計量。公允價值被定義爲在測量日期在資產或負債的主要或最有利的市場上以有序交易方式在市場參與者之間交易時將獲得的交換價格（按退出價格計算）。可能用於衡量公允價值的三個輸入級別包括：
第1級：在測量日期對於資產或負債可訪問的相同資產或負債在活躍市場中報出（未調整）的價格。公允價值層次結構最優先考慮第1級輸入。公司的第1級資產包括貨幣市場基金。
11
二級：除一級價格以外的可觀察輸入，如活躍市場中類似資產或負債的報價價格或其他可觀察或可以通過可觀察市場數據證實的輸入，可以基本完整地覆蓋資產或負債的全部期限。
三級：由市場活動幾乎沒有支持的不可觀察輸入。公平價值層次結構最低地對三級輸入優先。
在確定公允價值時，公司利用估值技術最大程度地利用可觀察輸入，並儘可能減少不可觀察輸入的使用，並在評估公允價值時考慮交易對手信用風險。
現金及現金等價物中包括的貨幣市場帳戶被視爲一級。
截至2024年和2023年9月30日止三個月和九個月內，未發生在公允價值衡量水平之間的轉移。其他金融工具主要包括現金及現金等價物、其他流動資產、應付賬款和應計負債。這些金融工具的公允價值接近其賬面價值。
d. | 最近發佈的未採納會計準則 |
管理層認爲，儘管尚未生效，但最近發佈的會計準則，如果目前採納，對公司財務狀況或業績不會產生重大影響。在 期內行權。
備註3 - 承諾和 contingencies:
| a. | 許可協議 |
CRt 先鋒基金許可協議
CRt 先鋒基金許可協議沒有發生重大變化，如公司年度報告（2023年12月31日結束的財年）中披露，該報告於2024年3月5日提交給證監會（請查看我們年度報告中財務報表注 5a）。
由於與這些事件或里程碑的實現相關的不確定性，截至2024年9月30日和2023年12月31日，任何潛在的里程碑或版稅支付金額尚未計提。
愛丁堡大學許可協議
與公司提交給證監會的2023年12月31日年度報告中披露的愛丁堡大學（UoE）許可協議相比，目前尚未發生重大變化（請查看我們年度報告中的財務報表註釋5a）。
由於與這些事件、里程碑或額外研究承諾的實現相關的不確定性，截至2024年9月30日和2023年12月31日，任何未來可能的研究支持、里程碑或版稅支付金額均未予計提。截至2024年9月30日，公司已向UoE支付了$
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| b. | 關聯交易 |
除正常業務過程中已披露在公司2023財年12月31日提交給SEC的10-K表格中的相關方交易外，無其他相關方交易（請參閱我們在年度報告10-K中財務報表附註中的附註10）。
| c. | 備用金 |
截至2024年9月30日和2023年12月31日
註釋 4 – 股東權益:
| a. | 公開實體的定向增發 |
於2022年7月29日，公司完成了一項定向增發（「2022年7月定向增發」），根據2022年7月27日簽訂的證券購買協議（「協議」）的條款和條件。有關2022年7月定向增發，公司發行了
| b. | 市價購買計劃 |
2024年9月30日結束的九個月內，公司總共出售了
在截至2024年9月30日的三個月內，公司共出售了
截至2024年9月30日，ATm計劃尚有約百萬美元的證券可供出售。
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注5 - 基於股份的報酬：
| a. | 2021年全球股票激勵計劃（「激勵計劃」） |
以下表格總結了公司在截至2024年9月30日的九個月內激勵計劃中的股票期權活動：
|
|
| 已授予和預期於2021年1月2日授予股份 |
| ||||||
股數 | Risks and Uncertainties | 價格 | 聚合的 | |||||||
分shéarsunder | 行權價格每股 | 剩餘期限 | 內在價值 | |||||||
時間租船 | 時間租船 | life | （以千爲單位） | |||||||
2023年12月31日餘額 | |
| $ | |
|
| $ | | ||
已批准 |
| — |
| - |
|
|
|
| ||
行使 |
| — |
| - |
|
|
|
| ||
被取消 |
| — |
| - |
|
|
|
| ||
優秀 - 2024年9月30日 |
| |
| $ | |
|
| $ | | |
2024年9月30日到期可行權 |
| |
|
|
| |||||
2024年9月30日預計歸屬 |
| |
| $ | |
|
| $ | | |
截至2024年9月30日，未確認的股權報酬支出爲$
限制性股票授予
已向員工授予限制性股票獎勵(RSAs)。RSA獎勵的價值取決於授予日公司的股價。公司根據激勵計劃授予了RSAs。
以下表格總結了截至2024年9月30日爲止的九個月內公司的RSA活動情況，如上述激勵計劃所述：
|
| 已授予和預期於2021年1月2日授予股份 |
| Risks and Uncertainties |
| 聚合的 | ||||
股數 | 平均津貼 | 合同條款 | 內在價值 | |||||||
股份 | 日期 公允價值 | （以年爲單位） | （以千爲單位） | |||||||
2023年12月31日餘額 | |
| $ | |
|
| $ | | ||
已批准 |
| |
| |
|
|
|
| ||
被取消 | ( | | ||||||||
34,105 |
| ( |
| |
|
|
|
| ||
未來表現優越 – 2024年9月30日 |
| |
| $ | |
|
| $ | | |
預計歸屬於 – 2024年9月30日 |
| |
| $ | |
|
| $ | | |
截至2023年7月31日，續借貸款協議下未償還的借款額爲
截至2024年9月30日，未確認的RSA相關補償成本總額爲$
2024年1月4日，公司發行
2023年1月12日，公司發行
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在第三個確定日期上，潛在標的的收盤價雖增長了25％，但您僅收到提前贖回支付，無法從此類增值中受益。 在授予日期的每個週年紀念日。於2024年1月4日，將2023年1月授予的首個三分之一的權益延長至2024年7月15日。於2024年7月12日，將2023年1月授予的首個三分之一的權益延長至2025年1月3日。
於2022年4月1日，公司發行
2021年7月27日，Bentsur先生，Poradosu博士和Shemesh先生被授予
股份報酬支出
截至2024年9月30日的三個月，公司確認了支出，金額爲$
截至2024年9月30日的九個月，公司確認了$
注6 - 每股虧損：
| a. | Basic |
基本每股淨虧損是通過將歸屬於公司股東的淨虧損除以平均未行使普通股份的加權平均數來計算的。
| |||||||||||||
三個月 | 三個月 | 在這九個月中 | 在這九個月中 | ||||||||||
截至2024年9月30日 |
| 截至2023年9月30日 | 截至2024年9月30日 |
| 截至2023年9月30日 | ||||||||
以千美元爲單位，除每股和股份數量外 | |||||||||||||
歸屬於普通股股東的淨虧損 | $ | ( |
| $ | ( | $ | ( |
| $ | ( |
| ||
每股普通股基本和稀釋淨虧損 | ( |
| ( | ( |
| ( |
| ||||||
普通股加權平均持股量 | |
| | |
| |
| ||||||
15
基本每股虧損是通過將歸屬於公司股東的結果除以期間內發行的普通股權重平均數量來計算的。
| 截至九個月結束時 | ||||||
2024年9月30日 |
| 2024年9月30日 | |||||
普通股加權平均 | |
| |
| |||
未獲授予的限制性股票的加權 | ( |
| ( |
| |||
普通股加權平均股數 | |
| |
| |||
| b. | Diluted |
由於其對所示期間的稀釋淨損失每股影響將屬於反稀釋性，以下可能具有稀釋性的證券未包括在稀釋每股普通股淨損失的計算中：
2021年9月30日 | ||||
| 2024 |
| 2023 | |
與以下相關的普通股份可分享： |
|
|
|
|
權證 |
| |
| |
Options |
| |
| |
未獲頒 RSAs* |
| |
| |
| * | 包括 |
附註7 - 關聯交易：
| a. | 除正常業務外，無關聯方交易。 |
附註8 - 後續事項：
| a. | 請參閱注5，了解有關延長Bentsur先生，Poradosu博士和Shemesh先生獲得期權的期限的信息。 |
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項目2。經營管理層討論及經營成果分析
您應閱讀本報告其他地方出現的財務報表及相關附註與本報告一起出現的下面的討論和分析。以下討論和分析含有在1933年證券法第27A條和1934年證券交易法第21E條的意義內的前瞻性聲明，包括但不限於，關於我們的期望、信念、意圖或未來策略的聲明，這些聲明由「期望」、「預期」、「打算」、「相信」、「可能」、「計劃」、「尋求」或類似語言表示。本文件中包含的所有前瞻性聲明均基於我們在本文件日期可得的信息，我們不承擔更新任何此類前瞻性聲明的義務。對於此類前瞻性聲明，我們宣稱受1995年私人證券訴訟改革法中包含的前瞻性聲明安全港保護的保護。我們的業務和財務表現面臨重大風險和不確定性。實際結果可能與前瞻性聲明中的預測結果大不相同。在評估我們的業務時，您應仔細考慮本報告「風險因素」標題下以及我們於2023年12月31日結束的年度10-k表中提供的信息。在下文中，「我們」、「我們」和「我們的」一詞可能指的是Nuvectis Pharma公司。
概述
我們是一家專注於開發用於治療腫瘤領域未滿足的嚴重醫療需求的創新精準藥物的生物製藥公司。
NXP800（GCN2激酶激活劑）
我們已經獲得了NXP800的全球獨家開發和商業權利許可。 在倫敦英格蘭的癌症研究所發現的一種口服小分子 在倫敦英格蘭的癌症研究所（ICR）發現。
在臨床前研究中，NXP800治療抑制了人類卵巢、子宮內膜和胃癌異種移植模型中的腫瘤生長，在其中，At-rich interactive domain-containing protein 1A（ARID1a）基因的遺傳突變存在，可能將ARID1a作爲治療敏感性的生物標誌物，並因此提供一種患者丰度的潛在策略。基於這項工作，我們已經開始在鉑金抵抗性ARID1a突變的卵巢癌中進行NXP800的臨床研究，這種癌症主要由兩種組織學類型構成，即卵巢透明細胞癌（OCCC）和卵巢子宮內膜癌（OEC）。我們正在探索利用ARID1a缺陷作爲其他腫瘤類型患者選擇標誌物的實用性。對ARID1a突變的遺傳篩查可以使用商用的基於下一代測序的體外診斷測試進行檢測，這些測試通常在臨床上爲癌症患者所使用。
2021年12月，位於英國的一期研究已經開始，包括兩部分：劑量遞增（1a期），隨後是擴展期（1b期）。在1a期中，對患有晚期實體瘤的患者評估了NXP800的安全性、耐受性和藥代動力學特性，以確定1億階段的劑量和給藥方案。該研究的10億階段始於2023年第二季度，旨在評估NXP800對鉑金抵抗性ARID1a突變的卵巢癌患者的安全性和初步抗腫瘤活性。
2022年6月，NXP800的新藥申請（IND）獲得了美國食品和藥物管理局（FDA）的批准，包括1期臨床試驗方案。2022年12月，我們宣佈FDA授予NXP800用於治療鉑金抵抗性ARID1a突變的卵巢癌的開發計劃快速通道認定地位。
2023年8月，我們宣佈FDA授予NXP800孤兒藥物認定，用於膽管癌患者的治療。
2023年12月，我們宣佈與梅奧診所合作，在膽管癌患者中進行一項由調查員贊助的臨床試驗。
2024年8月，我們宣佈FDA授予NXP800孤兒藥物認定，用於治療ARID1a缺乏的卵巢、輸卵管和原發性腹膜癌患者。
17
NXP900（SRC/YES1 激酶抑制劑）
我們已授權 NXP900 的全球獨家開發和商業版權，這是一種 SRC 家族激酶（「SFK」）抑制劑，可有效抑制 c-src（「SRC」）和 YES1 激酶。NXP900 是在蘇格蘭愛丁堡大學發現的。
SRC 在許多癌症類型中會被異常激活，包括乳腺癌、結腸癌、前列腺瘤、胰腺瘤和卵巢瘤等實體瘤，而在非癌細胞中仍然主要處於非活性狀態。SRC 活性的增加通常與晚期癌症、轉移潛能和治療耐藥性有關，並與不良的臨床預後相關。據報道，YES1基因擴增與多種腫瘤有關，包括肺癌、頭頸癌、膀胱癌和食道癌。此外，YES1 直接磷酸化和激活 Yes相關蛋白（「YAP1」），Hippo通路的主要影響因子，已被確定爲包括鱗狀細胞、間皮瘤和乳頭狀腎癌在內的多種癌症類型的耐藥性、癌症進展和轉移的啓動子。
在體內，NXP900 治療可抑制乳腺癌、宮頸癌、食道癌、頭頸癌和髓母細胞瘤異種移植模型中的原發性和轉移性腫瘤的生長，並顯示出靶向藥效學效應。此外，已經發現YES1基因擴增是表皮生長因子受體（「EGFR」）、人類表皮生長因子受體2（「HER2」）和間變性淋巴瘤激酶（「ALK」）耐藥的關鍵機制。2022年4月發表在《自然通訊》（非公司贊助）上的一項同行評審研究表明，與奧西替尼聯合使用時，NXP900 能夠使耐藥性非小細胞肺癌（「NSCLC」）細胞對奧西替尼（Tagrisso® 中的活性成分）重新敏感，奧西替尼是治療表皮生長因子突變陽性非小細胞肺癌的主要抑制劑。我們在細胞系模型中複製和擴展了這些發現，表明與奧西替尼聯合使用以及單獨與alK抑制劑阿樂替尼（Alecensa® 中的活性成分）聯合使用具有統計學意義的協同作用。
2023 年 5 月，美國食品藥品管理局批准了我們的 NXP900 臨床試驗申請，其中包括 1 期臨床試驗方案。
第 1 階段研究於 2023 年 9 月啓動，由兩部分組成：劑量遞增（第 1a 階段），然後是擴展階段（第 1b 階段）。在正在進行的 1a 期中，正在評估 NXP900 在晚期實體瘤患者中的安全性、耐受性和藥代動力學特性，以確定第 10期的劑量和給藥時間表。
運營結果
從2020年7月27日成立到2024年9月30日，我們沒有產生任何收入。 從那時起ur 從頭開始 2024 年 9 月 30 日 我們的主要活動是開發我們的兩種候選藥物，即 NXP800 和 NXP900，包括完成許可協議、支持IND的研究、對每種候選藥物進行正在進行的1期臨床試驗以及包括籌集資金在內的其他組織活動。對於 NXP800，我們進行了支持臨床試驗的研究，隨後我們的臨床試驗申請和英國藥品和醫療保健監管局的受理，IND 申請並獲得 FDA 的認可，以及西班牙藥品和醫療器械管理局的臨床試驗申請和受理。NXP800 的 1a 期和 10期臨床試驗分別於 2021 年 12 月和 2023 年 4 月開始。對於 NXP900，我們進行了支持臨床試驗的研究，隨後我們申請了 IND 並獲得了 FDA 的認可，併爲 2023 年 9 月開始的 1a 期臨床試驗做了準備。
研究和開發費用
研發費用包括直接歸因於開展研發計劃的成本，包括許可費、工資成本、基於股份的薪酬支出、工資稅和其他員工福利、分包商以及用於研發活動的材料和服務，包括臨床試驗、製造成本和專業服務。與研究與開發有關的所有費用均按發生時列爲支出。
處於臨床開發後期階段的候選產品的開發成本通常高於臨床開發早期階段的候選產品，這主要是由於後期臨床試驗的規模和持續時間的延長。我們預計，在短期和將來，由於我們正在進行和計劃中的臨床前和臨床開發活動，我們的研發費用將大幅增加。我們的候選產品的成功開發是高度不確定的。目前，我們無法準確
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估計或了解完成我們任何產品候選藥的臨床前和臨床開發所需工作的性質、時間和成本，我們可能永遠無法獲得任何產品候選藥的監管批准。
一般行政費用
一般和行政費用主要包括我們行政、財務和會計人員的工資和與人員相關的成本，包括基於股票的補償，在專利和公司事務方面的法律費用；支付給會計、審計、諮詢和稅務服務的專業費用；保險費用；投資者關係活動；旅行費用；以及未包括在研發費用中的設施費用。
我們預計隨着增加人數以支持我們持續的研發活動，我們的一般和行政費用將在未來增加。
以下表格總結了我們截至2024年9月30日和2023年9月30日三個月的營業費用（以千爲單位）。
截至9月30日的三個月 | |||||||||
| 2024 |
| 2023 |
| 變化 | ||||
營業費用: |
|
|
|
|
|
| |||
研發 |
| $ | 2,819 |
| $ | 4,486 |
| $ | (1,667) |
一般行政 |
| 1,540 |
| 1,672 |
| (132) | |||
營業虧損 |
| (4,359) |
| (6,158) |
| 1,799 | |||
財務收益 |
| 206 |
| 277 |
| (71) | |||
淨損失 |
| $ | (4,153) |
| $ | (5,881) |
| $ | 1,728 |
研發費用
以下表格總結了我們截至2024年9月30日和2023年三個月的研發費用（以千元計）
| 截至9月30日的三個月 |
| 增長 | ||||||
| 2024 |
| 2023 |
| （減少） | ||||
臨床費用 | $ | 1,058 |
| $ | 1,209 | $ | (151) | ||
員工薪酬福利 | 1,414 |
| 1,219 | 195 | |||||
製造業-半導體 | 333 |
| 1,494 | (1,161) | |||||
許可費 | — |
| 500 | (500) | |||||
專業服務和其他 | 14 |
| 65 | (51) | |||||
所有研發費用 | $ | 2,819 |
| $ | 4,486 | $ | (1,667) | ||
在2024年9月30日結束的三個月內，研發支出下降了170萬美元，降低了37%，與2023年同期相比。2024年9月30日結束的三個月內，研發支出的減少主要是由於製造相關成本減少了120萬美元，這是由於製造活動減少，NXP900的一次性許可費減少了50萬美元，臨床試驗費用減少了20萬美元，部分抵消了2023年的臨床試驗啓動費用，員工薪酬增加了20萬美元，其中包括與僱員股票補償相關的10萬美元的增加。
以下表格總結了我們在2024年9月30日和2023年結束的三個月的一般和行政支出（以千爲單位）。
截至9月30日的三個月 |
| 增長 | |||||||
| 2024 | 2023 |
| （減少） | |||||
其他 |
| $ | 764 |
| $ | 831 | $ | (67) | |
員工薪酬福利 |
| 391 |
| 410 | (19) | ||||
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保險和其他 |
| 385 |
| 431 | (46) | ||||
總管理費用 |
| $ | 1,540 |
| $ | 1,672 | $ | (132) |
截至2024年9月30日的三個月內，一般和行政費用減少了$10萬，或8%，與2023年同期相比。2024年9月30日結束的三個月內，一般和行政費用的減少主要是由於與上市公司相關費用的專業和諮詢服務減少$10萬引起的。
因上述原因，截至2024年9月30日的三個月內，我們的營業虧損減少了$170萬，或29%，相較於2023年同期，其中由於利率期貨增加，財務收入增加$20萬。
以下表總結了截至2024年9月30日的九個月的營業費用結果2024年和2023年：
（以千爲單位）
截至9月30日的九個月內， | |||||||||
| 2024 |
| 2023 |
| 變化 | ||||
營業費用: |
|
|
|
|
|
| |||
研發 |
| $ | 8,422 |
| $ | 11,115 |
| $ | (2,693) |
一般行政 |
| 4,976 |
| 4,916 |
| 60 | |||
營業虧損 |
| (13,398) |
| (16,031) |
| 2,633 | |||
財務收益 |
| 646 |
| 393 |
| 253 | |||
淨損失 |
| $ | (12,752) |
| $ | (15,638) |
| $ | 2,886 |
研發費用
以下表格總結了截至2024年9月30日和2023年的研發費用情況：
（以千爲單位）
| 截至9月30日的九個月， |
| 增長 | ||||||
| 2024 |
| 2023 |
| （減少） | ||||
員工薪酬福利 | $ | 4,358 |
| $ | 3,805 | $ | 553 | ||
臨床費用 | 2,651 | 3,085 | (434) | ||||||
製造業-半導體 | 1,382 |
| 3,094 | (1,712) | |||||
許可費 | — |
| 1,001 | (1,001) | |||||
專業服務和其他 | 31 |
| 131 | (100) | |||||
所有研發費用 | $ | 8,422 |
| $ | 11,115 | $ | (2,693) | ||
2024年9月30日結束的九個月裏，研發費用下降了270萬美元，與2023年同期相比。2024年9月30日結束的九個月裏，研發費用的減少主要是由於與製造業相關成本減少了170萬美元，NXP900一次性許可費減少了100萬美元，臨床試驗費用減少了40萬美元，部分抵消了員工薪酬增加了50萬美元，其中包括員工股票補償增加了40萬美元。
一般行政費用
以下表格總結了我們在2024年9月30日和2023年結束的九個月裏的一般和管理費用（以千美元計）。
截至9月30日止的九個月 |
| 增長 | |||||||
| 2024 | 2023 |
| （減少） | |||||
其他 |
| $ | 2,609 |
| $ | 2,272 | $ | 337 | |
員工薪酬福利 |
| 1,277 |
| 1,405 | (128) | ||||
保險和其他 |
| 1,090 |
| 1,239 | (149) | ||||
20
總管理費用 |
| $ | 4,976 |
| $ | 4,916 | $ | 60 |
截至2024年9月30日的九個月內，總部和行政費用比2023年同期增加了10萬美元。2024年9月30日的九個月內，總部和行政費用的增加主要是由於與公司相關費用中的專業和諮詢服務增加了30萬美元，部分抵消了與員工薪酬和福利減少10萬美元以及其他費用減少10萬美元有關。
由於上述原因，2024年9月30日結束的九個月內，與2023年同期相比，我們的營業虧損減少了290萬美元，這主要是由員工薪酬和福利、臨床試驗費用、製造費用以及財務收入增加30萬美元推動的。
流動性和資本資源
截至2024年9月30日，我們的現金及現金等價物爲1720萬美元。截至2024年9月30日和2023年的三個月，我們分別報告了淨損失爲420萬美元和590萬美元。截至2024年和2023年的九個月，我們的淨損失分別爲1280萬美元和1560萬美元。
2022年2月4日，我們宣佈以每股5.00美元的價格定價了我們的首次公開發行普通股(IPO)，共發行320萬股普通股，扣除一定的承銷折讓和佣金。根據UoE許可協議，我們需要支付UoE未來基金籌集的總額的2.5％，直到累計總額達到300萬美元。根據IPO，我們向UoE支付了與這次籌款有關的40萬美元。
IPO於2022年2月8日結束，毛收益爲1600萬美元，扣除承銷折讓和費用後淨收益爲1260萬美元。
此外，2022年7月29日，我們完成了2022年7月的定向增發，獲得了毛收益1590萬美元，在扣除費用和開支後淨收益爲1420萬美元，不包括根據我們許可協議要求支付的款項。作爲該交易的一部分，我們發行了優先投資期權，該期權於2023年1月23日行使，至2026年1月29日到期，行使價格爲每股9.65美元，根據證券購買協議中定義的某些調整。截至2023年12月31日，100萬1091個優先投資期權以淨收益890萬美元行使。截至2024年9月30日止三個月和九個月，未行使任何優先投資期權，淨收益爲0美元。此外，作爲2022年7月的定向增發的一部分，我們向代銷商發行了認股權證，最多可購買115,481股普通股。代銷商認股權證與優先投資期權基本相同，唯一的區別是行使價格爲每股10.31美元。截至2024年9月30日，79,104份代銷商認股權證已行使，淨收益80萬美元。
2023年3月17日，我們以S-3表格提交了一個無註冊許可書登記聲明（「登記聲明」）。根據登記聲明，我們可以發行總共高達15000萬美元的證券。與登記聲明的提交相對應，我們還與H.C.Wainwright & Co.（「銷售代理」）簽訂了銷售協議，根據該協議，我們可以通過一個總額高達4000萬美元的現場發行計劃(ATM)發行和銷售我們的普通股股票，這包括在登記聲明下可提供的15000萬美元的證券中。根據ATM，我們將向銷售代理支付高達總收益的3.0％的佣金率作爲我們的普通股任何銷售的佣金。我們無需在ATM下出售任何股票。截至2024年9月30日，我們已售出了1,337,654股普通股，並在ATM下收到了淨收益1290萬美元。
我們相信IPO、定向增發和ATm所籌集的資金將至少支持我們在財務報表公佈後的未來12個月內支付營業費用和資本支出。我們這一估計是基於可能被證明錯誤的假設，我們可能會比預期更早耗盡可用資本資源。我們長期的未來生存取決於我們籌集額外資金以資助我們的運營能力。
我們預計隨着我們不斷進行的活動，特別是推進當前或未來產品候選藥物的臨床試驗，包括根據我們與NXP800和NXP900許可協議所涉及的里程碑和贊助研究承諾支付，我們的支出將大幅增加。此外，我們預計作爲公開公司運作會導致成本費用增加。
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隨着我們的發展，公司的法律、會計、投資者關係和其他費用都在增加。我們的運營支出的時間和金額將在很大程度上取決於我們能力：
| ● | 推進我們的臨床和臨床前項目的發展； |
| ● | 製造或採購我們的臨床前和臨床藥物材料，並開發後期和商業製造的流程； |
| ● | 爲成功完成臨床試驗的任何當前或未來產品候選品尋求監管批准； |
| ● | 根據我們的許可協議實現里程碑 |
| ● | 建立銷售、市場營銷、醫務和分銷基礎設施，用於商業化我們可能獲得營銷批准的任何現有或未來候選產品 |
| ● | 招聘更多的臨床、質量控制和科學人員； |
| ● | 擴大我們的運營、財務和管理系統，增加人員，包括支持我們臨床開發、製造和商業化努力以及作爲上市公司的運營的人員 |
| ● | 獲取、保護、擴大和保護我們的知識產權組合；和 |
| ● | 收購額外的產品候選者。 |
我們預計在尋求監管批准我們的產品候選者以及如果選擇追求入許可協議或收購其他產品候選者時，將需要額外的資本。如果我們的當前或未來產品候選者獲得監管批准，我們預計將承擔與產品製造、銷售、營銷和分銷有關的重大商業化費用，具體取決於我們選擇進行商業化的地點。
由於與我們的產品候選者的研究、開發和商業化相關的衆多風險和不確定性，我們無法估計我們的運營資本需求的確切金額。我們未來的資金需求將取決於許多因素，並且可能會因此顯著增加，包括：
| ● | 研究和開發我們當前或未來產品候選者的範圍、進展和成本，包括我們的臨床前和臨床試驗的時間安排以及安全性、耐受性和有效性結果。 |
| ● | 我們當前或未來產品候選者的監管審查成本、時間和結果； |
| ● | 我們當前或未來產品候選者製造的成本、時間以及爲支持我們的臨床試驗和臨床預研工作的能力； |
| ● | 未來活動的成本，包括我們任何獲得營銷批准的當前或未來產品候選者的銷售、醫療事務、營銷、製造和分銷成本； |
| ● | 生產商業級產品的成本以及支持商業上市所需的庫存成本； |
| ● | 有能力獲得額外的非稀釋性資金，包括來自組織和基金會的資助； |
| ● | 我們的產品如果獲得營銷批准，從商業銷售中獲得的營業收入（如果有）； |
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| ● | 準備、申請和代表我們進行專利申請，獲取、維護、擴展和執法我們的知識產權以及捍衛知識產權相關訴訟的成本； |
| ● | 我們建立和維護合作伙伴關係並達成有利條款的能力，如果可能的話；而且 |
| ● | 我們獲得或通過許可獲取其他候選產品和技術的程度。 |
在我們能夠產生大量產品營業收入之前，我們預計將通過公開或私人股權發行、債務融資、政府資助、合作、戰略合作伙伴關係和聯盟，或與第三方進行市場營銷、分銷或許可安排等多種途徑來籌措運營資金。如果我們通過出售股權或可轉換債務證券籌集額外資本，您的所有權可能會受到重大稀釋，而這些證券的條款可能包括清算或其他優先權，這可能不利於您作爲普通股股東的權益。債務融資和優先股權融資（如果有）可能涉及限制我們能夠採取特定行動的限制性契約，比如增加額外債務、進行資本支出或宣佈分紅等。此外，債務融資會導致固定的支付義務。
如果我們通過政府資助、合作、戰略伙伴關係、聯盟、市場營銷、分銷或許可安排與第三方合作方籌集額外資金，我們可能不得不放棄對我們的技術、未來營業收入、研究項目或產品候選者的寶貴權利，或者以可能對我們不利的條款授予許可。如果我們無法及時通過股權或債務融資等其他安排籌集額外資金，可能需要延遲、限制、減少或終止我們的研究、產品開發或未來商業化努力，或授予開發和推廣產品候選者的權利，而這些產品我們本來更願意自己開發和推廣。
現金流量
下表提供了所述期間我們的現金流量信息：（以千爲單位）
截至9月30日止的九個月 | ||||||
| 2024 |
| 2023 | |||
經營活動使用的淨現金流量 |
| $ | (9,782) |
| $ | (12,104) |
投資活動產生的淨現金流出 |
| — |
| — | ||
籌資活動產生的現金淨額 |
| $ | 7,825 |
| $ | 14,170 |
經營活動
在2024年9月30日結束的九個月內，運營活動中使用了980萬美元現金。這主要歸因於我們1280萬美元的淨虧損，部分抵消了370萬美元的非現金費用。我們經營資產和負債的變動主要是由於向供應商支付60萬美元，以及支付董事和高級管理人員保險費用10萬美元。
在2023年9月30日結束的九個月內，運營活動中使用了1210萬美元現金。這主要歸因於我們1560萬美元的淨虧損，部分抵消了350萬美元的非現金費用。我們經營資產和負債的變動主要是由於支付員工薪酬和福利50萬美元。
籌資活動
在2024年9月30日結束的九個月中，融資活動提供的淨現金爲780萬美元，主要包括通過ATm出售普通股所得的淨收入。
在2023年9月30日結束的九個月中，融資活動提供的淨現金爲1420萬美元，主要包括我們定向增發相關認股權證所得的1030萬美元淨收入以及通過ATm出售普通股所得的420萬美元淨收入，減少了支付的40萬美元的遞延發行成本。
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與業務組合有關，在扣除發行成本後，我們獲得了約38390萬美元的現金收益。截至2021年9月30日，我們的現金及現金等價物爲28230萬美元。業務組合之後，管理層認爲其現有的財務資源足以滿足其至少在發佈這些財務報表之日起12個月內運營和資本需求。公司可能需要額外的資本來追求某些商業機會或響應技術進步，競爭動態或技術，客戶需求，挑戰，收購或不可預見的情況。此外，公司已經發生了，並預計將繼續發生與成爲一家上市公司有關的重大成本。因此，公司可能在未來進行股權或債務融資或進入信貸機構以實現上述或其他原因；但是，公司可能無法及時以有利可圖的條件安全地獲得額外的債務或股權融資。如果公司通過股權融資獲得了額外資金，則其現有股東可能會面臨重大稀釋。此外，公司在未來獲得的任何債務融資都可能涉及與公司的資本籌集活動和其他財務和運營事項有關的限制性契約，這可能會使公司更難以獲得額外的資本並追求商業機會。如果公司無法在需要時以令人滿意的條件獲得充足的融資，公司繼續增長或支持業務及應對業務挑戰的能力可能會受到嚴重限制。
我們與臨床研究機構、合同製造組織和其他第三方簽訂合同，用於臨床試驗、臨床前研究和測試以及製造服務，在正常業務過程中。這些合同可以在提前書面通知的情況下由我們取消。取消時應付的款項僅包括截至取消日期提供的服務或發生的費用，包括我們的服務提供商不可取消的義務。此類付款的金額和時間尚不清楚。
我們還與第三方簽訂了許可和合作協議，這在正常業務過程中。我們未包括這些協議下的未來付款，因爲這些協議下的義務取決於未來事件，例如我們達到指定的發展、監管和商業里程碑，或者淨產品銷售的版稅。
根據NXP800許可協議，我們需要向ICR支付某些與發展和監管里程碑相關的費用，包括最多2200萬美元的預批准里程碑費用，最多17800萬美元（另加2200萬美元）的監管和商業銷售里程碑費用，以及根據銷售淨額分層基礎上的中位數到10％的版稅，除非終止開發。此外，我們最初同意向ICR提供高達額外50萬美元的研究和開發。2022年3月31日，我們同意向ICR提供額外40萬美元的研究和開發支持（總計90萬美元）。
根據NXP900許可協議，我們需要向UoE支付某些與發展和監管里程碑相關的費用，包括最多4500萬美元的預批准里程碑費用，最多27960萬美元（另外4500萬美元）的監管和商業銷售里程碑費用，淨銷售額的中位數到8％的版稅以及我們將來基金募集總額的2.5％，最高累計達300萬美元，除非停止開發。此外，我們將向UoE提供額外高達754,000美元的研究和開發支持。
我們目前沒有任何長期租賃協議。我們在2024年5月1日簽訂的一年協議基礎上租用新澤西州Fort Lee的辦公空間。
不設爲資產負債表賬目之離線安排
在所呈現期間，我們並沒有，也目前沒有任何符合證券交易委員會規定的表外安排。
關鍵會計政策和重大判斷和估計
我們在本季度報告的形式10-Q中的簡明財務報表及相關附註，是根據美國通用會計原則編制的。簡明財務報表的編制還要求我們進行對資產、負債、成本、費用及相關披露的數額產生影響的估計和假設。我們的估計基於歷史經驗和我們認爲在該情況下合理的各種其他假設。實際結果可能與管理層所做的估計有重大不同。在我們的估計與實際結果之間存在差異的範圍內，我們未來的財務報表呈現、財務狀況、經營成果和現金流量將受到影響。
與我們在於2024年3月5日提交給美國證券交易委員會的年度報告中所述的「注2 - 重要會計政策摘要」中描述的相比，我們的關鍵會計政策和估計沒有重大變化。
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最近發佈的會計聲明
請參閱本季度報告在表格10-Q中包含的我們精簡財務報表附註2，討論最近的會計準則。
新興成長公司和小型報告公司的地位
2012年《創業公司啓動法案》允許像我們這樣的「新興成長公司」利用延長的過渡期來遵守適用於公開公司的新或修訂的會計準則，直到這些準則否則將適用於私人公司。我們選擇不「選擇退出」此規定，因此，我們將在私人公司採納新或修訂的會計準則時採納新或修訂的會計準則，直到我們（i）不可撤銷地選擇「選擇退出」這種延長過渡期，或者（ii）不再符合「新興成長公司」資格。
我們還是一家「較小的報告公司」，意味着我們非關聯方持有的股票市值加上我們首次公開發行的擬議票據總額少於70000萬美元，我們的年度營業收入在最近完成的財政年度內少於10000萬美元。只要我們（i）非關聯方持有的股票市值低於25000萬美元，或者（ii）我們的年度營業收入在最近完成的財政年度內少於10000萬美元且非關聯方持有的股票市值低於70000萬美元，我們將繼續是較小的報告公司。如果在我們不再是新興成長公司時我們仍是一家較小的報告公司，我們可以繼續依靠適用於較小報告公司的某些豁免披露要求。具體來說，作爲一家較小的報告公司，我們可以選擇在我們的年度報告10-K中只呈現最近兩個財政年度的審計財務報表，與新興成長公司類似，較小的報告公司在執行薪酬方面有減少的披露義務。
項目3。關於市場風險的定量和定性披露
根據SEC規則和法規，由於我們被視爲「較小的報告公司」，所以在本報告中不需要提供本項目要求的信息。
項目4。控制和程序
披露控件和程序的評估
我們保持「披露控制和程序」，如《證券交易法》第13a-15(e)條和第15d-15(e)條所定義，旨在確保公司在根據證券交易法規定提交的報告中需要披露的信息在SEC規則和表格規定的時間內被記錄、處理、總結和報告。披露控制和程序包括但不限於旨在確保公司在根據證券交易法規定提交的報告中需要披露的信息被積累並傳達給我們的管理層，包括我們的首席執行官和首席財務官，以便及時作出有關所需披露的決定。
截至2024年9月30日，管理層在我們的首席執行官和首席財務官的監督和參與下，評估了我們披露控制和程序的設計和運行的有效性（如《證券交易法》第13a-15(e)條和第15d-15(e)條所定義）。我們的披露控制和程序旨在合理保證我們在根據適用規則和表格規定提交的報告中需要披露的信息在規定的時間內被記錄、處理、總結和報告。根據該評估，我們的首席執行官和首席財務官得出結論，截至2024年9月30日，我們的披露控制和程序是有效的。
關於基本報表的內部控制變化及管理報告
在適用於2024年9月30日結束的九個月內進行的根據《交易所法》規定的評估中，未發現對我們的內部財務報告控制產生實質性影響或可能產生實質性影響的內部控制變化。
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控制和程序的有效性存在固有限制
我們的管理層，包括首席執行官和財務副總裁，相信我們的披露控制程序和內部財務報告控制設計旨在提供合理保證達到其目標，並在合理保證級別上是有效的。但是，管理層並不認爲我們的披露控制程序或我們的內部財務報告控制能夠防止或發現所有錯誤和欺詐。無論控制系統構思和操作得多好，都只能提供合理的、而非絕對的保證，確保控制系統的目標得以實現。由於任何控制系統的固有侷限性，並沒有一項控制評估能夠絕對保證在公司內是否已檢測到所有的控制問題和欺詐行爲。任何控制系統的設計也部分基於對未來事件發生可能性的假設，而且無法保證任何設計在各種潛在未來情況下都能成功實現其既定目標。隨着時間推移，由於條件變化或遵守政策或程序的程度可能惡化，控制可能會變得不足。由於成本效益型控制系統的固有侷限性，由於錯誤或欺詐而導致的錯誤可能會發生而不會被發現。
第二部分-其他信息
項目1。法律訴訟。
我們可能不時捲入在業務日常中發生的法律訴訟，我們預計解決這些訴訟不會對我們的財務狀況、經營業績或現金流產生重大不利影響。然而，無法確定任何可能發生的未來訴訟是否會對我們的業務產生重大的財務影響。截至本報告日期，我們並非涉及任何重大法律事項或索賠。
第1A項。風險因素。
本報告中包含的風險因素可能導致實際結果與我們在本報告中提出的或不時提出的前瞻性陳述有所不同。在做出投資決策之前，您應仔細考慮以下風險，除本報告及我們的其他公開文件中所包含的其他信息外。我們的業務、財務狀況或業務成果可能會受到任何這些風險的損害。以下所述的風險和不確定因素並不是我們面臨的唯一風險。我們目前還不知道或目前還不認爲對我們業務造成重大風險的其他風險也可能影響我們的業務運營。
與我們的財務和資本需求有關的風險
由於我們的有限經營歷史，您可能難以評估我們迄今爲止的業務成功和評估我們未來的生存能力。我們的活動始於2010年，迄今爲止，我們的工作主要集中在籌集資本、尋找和開發我們的產品和臨床前計劃、拓展我們在開發產品方面的專業知識以及進行臨床前研究並進行早期臨床試驗。由於FDA於2019年12月發佈的重新分類規定，我們不得不暫停在美國銷售用於治療焦慮和失眠的Gen-1器械。我們目前正在評估是否繼續爲失眠和焦慮治療修訂FDA的舊申請或爲我們的下一代器械新申請510(k)。雖然我們開發了第二代和第三代的器械，但這些器械尚未獲得FDA在美國市場銷售的批准。因此，如果我們的運營歷史更長，您對我們未來成功或生存能力的任何預測可能不夠準確。
我們是一家臨床階段的生物製藥公司，經營歷史有限。我們於2020年7月在特拉華州註冊成立，至今的運營範圍僅限於組織和人員配備、業務規劃、籌集資金、識別、調查、許可和評估潛在藥物候選品，以及與第三方達成協議，生產我們的主要藥物候選品和組件材料的初始數量。我們的兩個藥物候選品均處於早期臨床開發階段。我們尚未證明能夠成功進行或完成任何藥物候選品的臨床開發計劃，獲得上市批准，生產商業規模產品，或安排第三方代表我們進行，或進行必要的銷售、市場營銷和分銷活動，以便成功將產品商業化。因此，對於我們未來成功或生存能力的任何預測可能不夠準確。
在某個時候，我們可能需要從一個以研發爲重點的公司過渡爲能夠支持完整產品生命週期相關商業活動的公司。我們可能在這種轉變中不會取得成功。
自成立以來，我們一直虧損，並預計在可預見的將來會繼續虧損。我們可能永遠無法實現或維持盈利。
對生物製藥產品開發的投資是一項高度投機性的事業，需要大量前期資本支出，並存在當前或潛在未來產品候選者可能無法表現出充分療效或
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具有可接受的安全性能，並獲得監管批准並具備商業化的可行性。我們仍處於產品候選品開發的早期階段，並於2021年12月啓動了首個臨床試驗。我們沒有任何獲批用於商業銷售的產品，也沒有從產品銷售中產生任何營業收入。我們將繼續承擔與我們正在進行業務相關的重大研發和其他支出。此外，作爲一家經營歷史有限的企業，我們可能會遇到未預料的支出、困難、複雜情況、延遲以及其他已知和未知因素，如新冠肺炎疫情。
自我們開始業務運營以來，各個時期都虧損。自創立以來至2024年9月30日結束，我們累積虧損達6700萬美元。我們預計在可預見的未來將繼續承擔重大虧損，並且我們預計如果繼續進行我們的主導產品候選品的研發工作； 爲我們目前和未來的產品候選品進行臨床前研究和臨床試驗； 尋求任何成功完成臨床試驗的目前或未來產品候選品的市場批准； 遇到任何延遲或在上述任何一個方面遇到任何問題； 建立銷售、營銷和分銷基礎設施和擴大製造能力以推廣我們可能獲得監管批准的任何目前或未來產品候選品； 取得、擴展、保持、實施和保護我們的知識產權組合； 僱傭更多臨床、監管和科學人員； 並作爲一家上市公司經營。
我們的產品候選品NXP800和NXP900均處於臨床研發階段。這兩個產品候選品將需要進行額外的臨床前和臨床研究，經過監管審查和批准，大量投資，獲得足夠的臨床和商業生產能力，以及在我們可能從產品銷售中潛在獲得任何營收之前進行重大的營銷工作。NXP800的1期研究於2021年12月開始，NXP900於2023年9月開始。迄今爲止，我們尚未從我們的產品候選品獲得任何營業收入。我們創收的能力將取決於多個因素，包括但不限於：
| ● | 我們能夠及時且成功地完成我們的臨床前研究和臨床試驗，這可能比預期更爲緩慢或更爲昂貴，將取決於多個因素，包括第三方承包商的表現，我們招募患者參與臨床試驗的能力，以及在臨床試驗中產生的安全性、耐受性和療效結果； |
| ● | 成功向FDA提交IND申請以及任何額外的類似申請； |
| ● | 完成爲IND或類似提交所需的非臨床研究，必要時； |
| ● | 我們是否被FDA或類似的外國監管機構要求進行額外的臨床試驗或其他研究，以支持我們當前或未來產品候選藥物的批准和商業化； |
| ● | 美國食品藥品監督管理局和類似的外國監管機構對我們當前或未來產品候選者的安全性、效力、純度、功效和風險受益配置的接受情況; |
| ● | 我們當前或未來產品候選者可能出現的副作用或其他安全問題的盛行率、持續時間和嚴重程度，如果有的話; |
| ● | 從FDA和相似的外國監管機構獲得必要的營銷批准的時間; |
| ● | 我們當前或未來產品候選者（如果獲批）的實際和感知可得性、成本、風險配置以及相對於現有和未來可替代的癌症治療和競爭產品候選者和技術的安全性和功效，以及競爭者的產品候選者和技術; |
| ● | 我們和第三方承包方的製造業-半導體能力足夠，可以製造出我們現有或未來的產品候選的足夠臨床和商業供應，保持良好的與監管機構合規，開發、驗證和維護符合cGMP商業可行製造流程； |
| ● | 我們有能力成功制定商業策略，並在美國和國際市場上銷售和分銷任何現有或未來產品候選，是否獲批上市、報銷、銷售和在這些國家和地區分銷，無論是獨自還是與他人合作； |
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| ● | 當批准後，患者對我們當前或未來產品候選者的需求；和 |
| ● | 我們在我們當前或未來產品候選者中建立和執行知識產權的能力。 |
上述許多因素超出了我們的控制範圍，可能導致我們經歷重大延遲或阻止我們獲得監管批准或商業化我們當前和未來產品候選者。即使我們能夠商業化任何當前或未來產品候選者，我們可能不會在生成產品銷售後不久或永遠實現盈利。
我們將需要大額額外資金。籌集額外資金可能導致我們現有股東的稀釋，或要求我們放棄專有權利。如果我們無法按需籌集資本，我們可能被迫延遲、減少或取消我們的產品開發計劃或商業化努力。
我們預計我們的支出將隨着我們當前活動的增加而增加，特別是在我們繼續活動以確定新的產品候選者並啓動臨床試驗，以及尋求對我們當前或將來的任何產品候選者進行市場批准之際。此外，如果我們爲我們當前或將來的任何產品候選者獲得市場批准，我們預計將承擔與產品銷售、市場營銷、製造和分銷相關的重大商業化支出。此外，我們預計將承擔作爲一家上市公司運營的重大額外成本。因此，我們將需要在持續運營過程中獲得大額額外資金。我們無法確定是否會獲得額外資金以可接受的條件提供，或根本不會提供。直到在某種時候，如果有的話，我們可以獲得大量產品收入，我們預計將通過公開或私人股權發行，ATM融資，債務融資，政府資助，合作，戰略合作伙伴關係或與第三方的市場、分銷或許可安排等方式融資我們的運營。在通過出售股權或可轉換債務證券進行籌集額外資本的情況下，您的所有權權益將進一步被稀釋，並且這些證券的條款可能包括可能不利影響您作爲股東的權利的清算或其他優先權。債務融資和優先股權金融，如果可用，可能包括限制或限制我們採取特定行動的協議，例如增加債務、進行資本支出或宣佈分紅。
如果我們通過與合作伙伴、戰略聯盟或營銷、分銷或許可安排籌集額外的資金，我們可能不得不放棄我們的技術、未來收入流、研究計劃或產品候選者的寶貴權利，或以對我們不利的條件授予許可。
如果我們在需要資本時無法融到資金，或者融資條件不佳，我們可能被迫推遲、減少或取消我們的發現和臨床前開發計劃，或任何未來的商業化努力。
重大的公共衛生事件，特別是仍在實施中的COVID-19大流行期間所採取的政策和程序，可能對我們的臨床試驗、財務狀況、經營業績和業務的其他方面產生不利影響。
COVID-19大流行對全球社會、經濟、金融市場和商業實踐產生了廣泛、迅速發展和不可預測的影響。在2020年和2021年期間，採取了政策和程序，至今仍在執行，包括多種疫苗和藥物的廣泛分發以應對大流行。我們已將運營方式調整爲與大流行共存。
COVID-19大流行對公司未來業務產生影響的不確定性影響了管理層對各種依賴於這些估計和假設的會計估計的判斷和假設。自公司於2021年開始運營以來，COVID-19對這些估計和判斷沒有起到實質性的影響。
公司繼續面臨相對不確定性，關於繼續實施的政策和程序、大流行的強度和持續時間，以及從COVID-19大流行中恢復的性質和時間表，以及對公司的影響，包括潛在的永久性變化臨床試驗運營由大流行引起的程度。公司採取了管理這種不確定性的方式（在這種情況下繼續是重要因素）通過強化其資產負債表和現金資產，避免債務，同時專注於成本控制。COVID-19爆發或任何爆發形成的一些因素
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由COVID-19的任何變種引起的延誤或其他不利影響我們的臨床試驗計劃，以及總體不利影響我們的業務，包括：
| ● | 臨床試驗現場啓動時的延遲或困難，包括招募臨床試驗現場的困難，以及由於感染COVID-19或任何變種而導致臨床試驗中的患者出現增加的退出率，被迫隔離，或者無法完成研究評估，尤其是對於年長患者或其他更容易感染COVID-19或任何變種的人； |
| ● | 醫療資源（包括臨床試驗研究員和工作人員）轉移，從開展臨床試驗轉向關注大流行引起的擔憂，可能導致合作伙伴公司的臨床試驗出現延遲； |
| ● | 旅行限制，包括國內和國際旅行的限制，以及政府強制實行的隔離或由重要第三方實施的限制，可能會中斷關鍵試驗活動，如臨床試驗現場啓動和監測； |
| ● | 由於人手短缺、生產放緩或停頓，我們從合同製造組織獲取產品候選品的供應中斷或延遲； |
| ● | 由潛在的工作場所、實驗室和辦公室關閉以及整個醫療體系員工雲辦公增加導致的中斷和延遲； |
| ● | 由於COVID-19或任何變種傳播導致的疫情對FDA或其他監管機構的運作產生影響，導致監管批准、檢查、審核或其他監管活動中斷或延遲； |
我們目前依賴第三方爲我們臨床試驗和業務關鍵領域的某些功能或服務提供支持。如果這些第三方受COVID-19疫情限制的影響，我們可能會遇到延遲和/或額外成本。因此，我們啓動和完成臨床試驗、獲得當前和未來產品候選品的監管批准以及商業化的能力可能會延遲或受到干擾。
與我公司藥品候選品開發相關的風險
我們的發展方法可能永遠無法導致市場化產品。
我們產品候選人群和潛在未來產品候選人群的患者人群，可能僅限於具有特定靶點突變的人群，可能尚未完全定義，但明顯小於總體治療癌症患者人群，並且我們將需要積極篩選和識別這些患者。成功識別患者取決於幾個因素，包括確定具有特定遺傳突變的疾病如何響應我們目前的產品候選人或任何未來的產品候選人，並且如有必要，開發配套診斷來識別這類遺傳突變。此外，即使我們成功識別患者，我們也不能肯定爲我們目標適應症而產生的患者群體是否足夠大以實現盈利。此外，即使我們的方法成功，我們也可能永遠無法成功識別我們的產品候選人可能有效的額外疾病。我們不知道我們用特定基因定義的癌症患者對患者的治療方法是否會成功；如果我們的方法不成功，我們的業務將受到影響。
我們在開發工作的早期階段，極大地依賴於推進NXP800、NXP900或任何未來的其他產品候選人通過臨床前和臨床開發，確定我們藥物候選人的安全和有效劑量和用藥方案，獲得監管批准並最終商業化NXP800、NXP900或任何未來的其他產品候選人的能力；如果我們在此方面遇到延誤，我們的業務將受到重大損害。
我們從NXP800、NXP900或任何未來的其他產品候選人中產生產品收入的能力，極大地依賴於成功的臨床開發和最終商業化。此外，我們的藥物開發計劃可能考慮開發配套診斷，這是基於基因突變和其他改變來識別適當患者人群的檢測或測試。配套診斷屬於醫療設備監管範圍，必須在可以營銷之前從FDA或某些其他外國監管機構獲得營銷授權。如果配套診斷
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對於我們目前和未來的所有產品候選品，爲了能夠安全有效地使用，FDA必須得出結論，配套診斷工具符合適用的安全和有效性標準或用於產品候選品的實質等同性標準，才能在美國市場推廣產品候選品或配套診斷工具。
在開發我們的產品候選品的臨床前期或臨床研究中出現負面結果可能會阻止或延遲我們繼續進行臨床項目或獲得監管批准的能力。例如，儘管我們基於ARID1a緘默卵巢癌模型的臨床前期研究表明有抑制腫瘤生長的跡象，認爲這種癌症類型可能對NXP800的治療特別敏感，但在臨床測試中可能並非如此，可能由於安全性、耐受性問題和/或療效不足，在任何目標適應症中同樣成立。此外，抗腫瘤活性在我們計劃在臨床試驗中評估的每種腫瘤類型中可能不同。因此，我們可能需要停止開發我們的藥物候選品或投入大量額外資源，延遲我們的臨床試驗，並最終延遲任何我們未來產品候選品的批准（如果有的話）。
由於在早期臨床試驗中出現安全性、耐受性和/或療效不足等原因，我們目前或未來的產品候選品可能在早期臨床試驗中沒有良好的結果。此外，早期臨床試驗的積極結果並不一定能預測未來的結果，我們推進的任何產品候選品可能在後續臨床試驗中沒有良好的結果或獲得監管批准。2024年3月，我們宣佈了正在進行中的NXP800鉑金耐藥ARID1a突變卵巢癌10億期研究的初步安全性和療效結果。在這一初步分析中觀察到的任何結果可能並不能預測未來的結果。
我們可能遇到挫折，可能會延遲或阻止我們的當前或未來產品候選品獲得監管批准或我們進行商業化，其中包括：
| ● | 負面或不確定的功效結果和/或臨床試驗中的不良安全性發現，或他人的臨床試驗取得積極結果，類似於我們的產品候選品，導致其批准，並發展爲決定或要求進行額外的臨床前測試或臨床試驗，或放棄一個項目； |
| ● | 由我們的臨床試驗中的患者或被試者或正在使用我們、FDA、其他監管機構或他人視爲與我們當前或未來產品候選品開發相關的藥物或治療品的個人體驗到的與產品相關的副作用； |
| ● | 提交IND申請或類似的國外申請時出現延遲，或者在開始臨床試驗之前未能獲得監管機構的必要批准，或者一旦開始就暫停或終止臨床試驗的延遲或失敗； |
| ● | FDA或與我們的臨床試驗範圍或設計有關的類似國外機構施加的條件，包括我們的臨床終點； |
| ● | 臨床試驗中受試者招募出現延遲，包括難以或延遲識別合適的患者，或由於健康相關緊急事件，如COVID-19大流行，以及按照GCP或良好實驗室規範（「GLP」）要求及時完成臨床試驗； |
| ● | 無法保持與監管要求的合規性，包括cGMP合規，並有效遵守與我們當前或未來產品候選品質相關的其他要求； |
| ● | 由於安全性和耐受性問題和/或療效不足，臨床試驗中受試者的高退出率； |
| ● | 我們當前或未來產品候選品的供應或質量不足，或進行臨床試驗所需的其他材料不足； |
| ● | 臨床試驗成本高於預期; |
| ● | 無法與其他療法競爭； |
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| ● | 我們當前或未來的產品候選者效果不佳，存在安全性和/或耐受性問題，或在臨床試驗過程中需要不同劑量或給藥計劃； |
| ● | 試驗結果所需時間超出預期； |
| ● | 試驗遭受欺詐、數據採集故障或其他技術故障導致試驗無效； |
| ● | 我們試驗的結果不支持在歐盟申請條件批准； |
| ● | 不利的FDA或其他監管機構對臨床試驗現場的檢查和審查； |
| ● | 我們的第三方承包商或調查人員未能遵守監管要求或未能及時履行其合同義務，或根本沒有履行； |
| ● | 由於COVID-19大流行的影響而導致的延遲，包括COVID-19對FDA繼續正常運作的影響； |
| ● | 監管要求、政策和指導方針的延遲和變更，包括針對臨床開發通常或針對我們的技術特別加強額外監管監督；或 |
| ● | FDA和類似的外國監管機構對數據的不同解讀。 |
此外，由於我們財務和人員資源有限，主要專注於開發NXP800和NXP900，因此我們可能放棄或推遲追求其他未來可能擁有更大商業潛力的產品候選藥物，可能未能充分利用可行的商業產品或有利可圖的市場機會。如果我們不能準確評估未來產品候選藥物的商業潛力或目標市場，我們可能通過合作、許可或其他版稅安排放棄對這些未來產品候選藥物有價值的權利，在這些情況下，我們保留獨家開發和商業化權利可能更爲有利。
臨床藥物開發涉及漫長而昂貴的過程，結果不確定，臨床試驗難以設計和實施，我們任何一項臨床試驗都可能產生失敗的結果或在任何階段失敗。
臨床試驗在人體上進行，費用昂貴，可能需要多年才能完成，結果固有地不確定。失敗可能發生在任何時間。此外，藥物候選物的早期研究和早期臨床數據的任何積極結果可能不能預測早期臨床試驗的最終結果或後續臨床試驗的結果，使得藥物候選物可能無法根據早期臨床試驗的結果達到後續階段的臨床試驗，或者在後續的臨床試驗中未能展現出期望的安全性和有效性特徵，儘管在早期研究和早期臨床試驗中顯示出這些特徵的跡象。製藥行業中許多公司由於缺乏功效或不良的安全性特徵，在早期和愛文思控股臨床試驗中遭受重大挫折，儘管在早期研究或初步臨床研究中取得了有希望的結果。因此，我們進行的任何現有和未來的臨床試驗的結果可能會失敗。臨床試驗可能由於成本高於預期或出於各種原因而延遲、暫停或過早終止，例如：
| ● | 藥品審批達成一致或與FDA或類似的外國監管機構達成一致，以執行我們能夠執行的試驗設計可能存在延遲或失敗； |
| ● | 延遲或未能獲得啓動試驗的授權，包括獲得適當獨立審查委員會(IRB)批准在人體上對候選者進行測試，或無法遵守監管機構就臨床試驗的範圍或設計所施加的條件； |
| ● | 延遲達成或未能達成與潛在CRO和臨床試驗地點就可接受條款達成一致的協議，這些條款可能經過廣泛談判並在不同的CRO和試驗地點之間可能有很大差異； |
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| ● | 在確定和維護足夠數量的試驗點方面存在無力、延遲或失敗的情況，其中許多試驗點可能已經參與了其他臨床項目； |
| ● | 招募和註冊合適的志願者或患者參與試驗方面可能存在延遲或失敗的情況； |
| ● | 如果被視爲必要，在及時基礎上開發和驗證伴隨診斷工具可能存在延遲或失敗的情況； |
| ● | 患者未能完成試驗或未能返回進行治療後的隨訪; |
| ● | 無法在治療期間或治療後充分監測患者; |
| ● | 臨床試驗現場和研究者偏離試驗方案, 未能根據監管要求進行試驗或退出試驗; |
| ● | 由於觀察到的安全問題或其他原因而由FDA或類似外國監管機構實施的臨床控件導致未能啓動或延遲或無法完成臨床試驗; |
| ● | 臨床試驗中出現負面或不確定結果，我們決定符合監管機構的要求進行額外的臨床前研究、臨床試驗或放棄一個或多個產品開發項目；或者 |
| ● | 無法制造出足夠數量和符合質量標準的藥物候選品用於臨床試驗。 |
我們依靠並計劃繼續依靠CROs、合同製造組織（「CMOs」）和臨床試驗點來確保對我們臨床試驗的適當和及時進行。儘管我們已經並預期將來會與CROs和CMOs簽訂管理其承包活動和行爲的協議，但我們對其實際表現具有有限影響力。因此，我們最終並不會對CRO或CMO遵守與我們可能有的任何協議條款，遵守適用的監管要求，或遵守已經達成的時間表和期限負有控制權，而未來CRO或CMO未能執行這些義務可能導致我們任何臨床試驗受到延遲或失敗。
此外，如果臨床試驗被暫停，被放棄或被我們、IRB或倫理委員會、數據安全監控委員會、FDA或歐洲藥品管理局（「EMA」）或其他監管機構終止，我們也可能遇到延遲。暫停或終止可能由許多因素引起，包括未能按照法規要求進行臨床試驗，FDA、EMA或其他監管機構對臨床試驗操作或試驗地點的檢查，使參與者面臨由意外安全問題或不良副作用引起的健康風險，先前未曾見過的安全問題的發展，未能證明使用藥物候選品具有益處，或者政府法規或行政行動的變化。因此，我們無法確定當前正在進行、計劃或未來任何臨床試驗的開始或完成時間表。
導致臨床試驗啓動或完成延遲的許多因素，也可能最終導致否決我方當前或未來產品候選者的營銷批准。
如果我們在啓動或完成任何藥物候選者的臨床試驗中遇到延遲，或者出現暫停、中止、暫停或終止，該藥物候選者的商業前景可能會受到損害，我們從該藥物候選者中產生產品收入的能力可能會延遲或被消滅。此外，臨床試驗完成的任何延誤都將增加我們的成本，使我們的藥物候選者開發和批准流程減慢，並危及我們藥物候選者的監管批准以及開始銷售和產生收入的能力。凡此種種事件的發生都可能嚴重損害我們的業務、財務狀況、運營結果和前景。
招募患者的困難可能會延遲或阻止我們當前或未來產品候選者的臨床試驗。
確定和資格認定參與我們當前或未來產品候選者臨床研究的患者對我們的成功至關重要。我們臨床研究完成的時機在一定程度上取決於我們能夠招募患者參與測試我們當前或未來產品候選者的速度，如果我們在招募方面遇到困難，我們可能會在臨床試驗中遇到延遲。
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此外，由於我們目前專注於患有特定和罕見疾病的患者，我們招募符合條件的患者的能力可能受限，可能導致招募速度比預期慢。我們的臨床試驗將與其他臨床試驗競爭，即當前或未來的產品候選品與我們當前或未來的產品候選品屬於相同的治療領域，這可能減少可供我們使用的患者數量和類型。
由於患者招募所需時間超出預期或患者退出超出預期，臨床試驗可能會出現延遲。如果我們無法找到並招募足夠數量的符合FDA或國外監管機構要求參與這些試驗的符合條件的患者，我們可能無法啓動或繼續當前或未來產品候選品的臨床試驗。我們無法預測我們未來在招募受試者方面將取得多大成功。患者的招募取決於許多因素，包括：
| ● | 患者識別和符合/不符合方案中定義的資格和包含/排除標準； |
| ● | 臨床試驗主要終點分析所需的患者人口規模以及識別患者的流程； |
| ● | COVID-19大流行可能導致的潛在干擾包括啓動臨床試驗點困難、招募和保留參與者困難、醫療保健資源轉移至臨床試驗、可能實施的旅行或隔離政策以及其他因素； |
| ● | 患者與臨床試驗點的距離； |
| ● | 試驗的設計； |
| ● | 我們招募具有適當能力和專業知識的臨床試驗研究者的能力； |
| ● | 臨床醫生和患者對正在研究的產品候選品相對於其他可用療法的潛在優勢和風險的看法，包括任何可能獲得批准用於我們正在研究適應症的新產品； |
| ● | 市場上競爭性可用療法的可獲得性和其他競爭性產品候選品的臨床試驗； |
| ● | 我們獲取和保持臨床試驗受試者知情同意的能力； |
| ● | 臨床試驗中被招募的受試者在完成之前退出試驗的風險。 |
如果我們無法找到並招募足夠符合FDA或類似監管機構要求參與的合格患者，我們可能無法啓動或繼續我們當前或未來產品候選品的臨床試驗。如有必要，我們打算與第三方合作開發伴隨診斷產品，用於我們的臨床試驗。如果這些第三方無功而返，我們在識別患有我們臨床試驗所針對的遺傳突變的患者方面可能會遇到困難。如果我們無法納入患有目標遺傳突變或有明確定義嚴重未滿足醫療需求的患者，我們可能無法參與FDA的加速審核和發展計劃，包括突破性療法認定和快速路徑認定，或尋求加速臨床開發和監管時間表。
我們的研究可能無法充分證明我們當前或未來任何產品候選品的安全性、效力、純度、功效或其他必要的藥理特性，這可能會阻礙或延遲開發、監管批准和商業化。
在獲得對我們當前或未來產品候選品進行商業銷售的監管批准之前，包括NXP800和NXP900，我們必須通過冗長、複雜和昂貴的研究證明我們當前或未來產品候選品在每個目標適應症中既安全又有效。臨床前和臨床測試費用高昂，可能需要多年才能完成，其結果本質上是不確定的。失敗可能發生在任何時候，包括臨床前研究和臨床試驗過程中，因爲我們當前的產品候選品處於早期開發階段，所以存在很高的失敗風險。
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The results of preclinical studies and early clinical trials of a drug candidate may not be predictive of the final results of later-stage clinical trials. Results of our trials could reveal a high and unacceptable severity and prevalence of adverse safety issues which may result in suspension or termination, and the FDA or comparable foreign regulatory authorities could, through a clinical hold or otherwise, order us to halt or cease further development of or deny approval. Drug-related side effects could also affect patient recruitment into the study or patient willingness to remain in the study and therefore affect our ability to complete clinical trials. Drug-related side effects could also result in potential product liability claims. Any of these occurrences may harm our business, financial condition and prospects significantly.
Moreover, 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.
The FDA and comparable foreign regulatory authorities may not accept data from any preclinical or clinical trials we may conduct in foreign countries.
The FDA’s acceptance of data generated for patients recruited outside the United States from clinical trials conducted in whole or in part outside the United States may be subject to certain conditions, if accepted at all.
Although the FDA has the authority to accept foreign data as part or even the sole basis for marketing approval, the FDA generally does not approve an application on the basis of foreign data alone unless (i) the data is applicable to the U.S. population and U.S. medical practice, (ii) the trials were performed by clinical investigators of recognized competence and pursuant to GCP regulations, and (iii) the FDA’s clinical trial requirements were met. Many foreign regulatory authorities have similar approval requirements. In addition, any clinical study conducted in whole or in part outside of the United States would be subject to the applicable local laws of the jurisdiction where the trial was conducted. We cannot guarantee that the FDA or comparable foreign regulatory authority will accept data from trials conducted in whole or in part outside of the United States, which may result in the need for additional trials.
We may not be able to submit IND applications to commence additional clinical trials on the timelines we expect, and even if we are able to, the FDA may not permit us to proceed.
Our CTAs and INDs for NXP800 and NXP900, have been approved. However, we may be unable to submit additional CTAs, IND applications or other clinical research on our expected timelines. Moreover, while we have obtained CTA and IND approvals, we cannot be sure that issues will not arise that may lead to the delay, suspension or termination of such clinical trials. Any failure to file CTAs, IND applications or other clinical research authorizations will adversely impact our expected timelines to obtain regulatory acceptance for the commencement of our trials and may prevent us from completing our clinical trials or commercializing our products on a timely basis, if at all.
We currently have no marketing and sales organization and have limited experience in marketing products. If we are unable to establish marketing and sales capabilities or enter into agreements with third parties to market and sell any approved product candidates, we may not be able to generate product revenue.
We will have to compete with other pharmaceutical and biotechnology companies to recruit, hire, train and retain marketing and sales personnel. If we are unable or decide not to establish internal sales, marketing, and distribution capabilities, we may pursue arrangements with third-party sales, marketing, and distribution collaborators regarding the sales and marketing of our products, if approved.
There can be no assurance that we will be able to develop in-house sales and distribution capabilities or establish or maintain relationships with third-party collaborators to commercialize any product in the United States or overseas.
We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.
While we believe that our scientific knowledge, technology, and development expertise provide us with competitive advantages, we face potential competition from many different sources, including major pharmaceuticals, specialty pharmaceuticals and biotechnology companies, academic institutions and government agencies, and public and private research institutes that conduct research, development, manufacturing, and commercialization. Many of our competitors have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, regulatory approvals, and product marketing than we do. Our
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競爭對手可能會在招聘和留住合格的科學和管理人員、建立臨床試驗中心和招募臨床試驗患者，以及獲取與我們項目相關的補充或必要的技術方面與我們競爭。結果可能是，我們的競爭對手可能會比我們更早或更成功地發現、開發、許可或商業化產品。
如果我們的產品候選藥NXP800和NXP900獲得批准，它們可能會與競爭對手藥物和其他目前正在開發中的藥物競爭。政府和其他第三方支付者對產品的報銷性也將顯著影響我們產品的定價和競爭力。我們的競爭對手可能會比我們更快地獲得FDA或其他監管部門對其產品的批准，這可能導致我們的競爭對手在我們進入市場之前建立強勁的市場地位。
政府監管風險
未能或延遲獲得必要的監管批准可能會阻止或推遲我們目前或未來產品候選品的商業化，我們產生營業收入的能力可能會受到實質性損害。
藥物產品的研究、測試、製造、標籤、批准、銷售、營銷和分銷在美國受FDA和其他國家/地區的監管機構的廣泛監管，各國的監管規定也不同。在我們收到FDA的NDA相應批准之前，我們將不被允許在美國推出我們目前或未來的產品候選品，也不會被允許在任何海外國家推出，直到我們收到各國家/地區的監管機構的批准。獲得FDA、EMA和類似外國機構批准的時間是不可預測的，通常需要很多年的臨床試驗後才確定，並取決於衆多因素，包括監管機構的廣泛裁量權和涉及的產品候選品的類型、複雜性和新穎性。監管機構在批准過程中具有相當大的裁量權，他們可能拒絕接受任何申請，或認爲我們的數據不足以獲得批准，需要額外的非臨床研究或臨床試驗。
獲取監管批准需要提交廣泛的非臨床和臨床數據以及支持信息給監管機構的每個治療適應症，以建立產品候選藥物的安全性和有效性。獲得監管批准還需要提交關於產品製造過程的信息，並且在許多情況下，監管機構會對製造、加工和包裝設施進行檢查。我們目前或未來的產品候選藥物可能無效，可能只是適度有效，或可能被證明具有不良或意外的副作用、毒性或其他特徵，這可能會阻止我們獲得上市批准，或阻止或限制商業使用，或者我們或我們的CMO在cGMP合規方面可能存在缺陷，這可能會導致候選藥物無法獲得批准。此外，我們尚未在任何司法管轄區獲得任何藥物候選品的監管批准，我們目前的藥物候選者或未來可能尋求開發的任何藥物候選者可能永遠都不會獲得監管批准。
出於許多原因，我們的藥物候選者可能無法獲得監管批准，或者可能被延遲獲得監管批准，包括以下任何一個或多個原因:
| ● | 美國食品藥品監督管理局（FDA）、歐洲藥品管理局（EMA）或類似的外國監管機構可能不同意我們臨床試驗的設計或實施; |
| ● | 我們可能無法向FDA、EMA或類似的外國監管機構證明一種藥物候選品對其擬議適應症是安全和有效的; |
| ● | 臨床試驗的結果可能不符合FDA、EMA或類似的外國監管機構批准所需的統計顯著性水平; |
| ● | 我們可能無法證明候選藥物的臨床和其他優勢超過其安全風險； |
| ● | FDA、EMA或類似的外國監管機構可能不同意我們對臨床前研究或臨床試驗數據的解釋； |
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| ● | 我們藥物候選品臨床試驗收集的數據可能不足以支持提交新藥申請或其他申請，也可能無法在美國或其他地方獲得監管批准； |
| ● | 在審查我們的臨床試驗現場和數據時，FDA或類似的外國監管機構可能會發現我們的記錄保存或我們臨床試驗現場的記錄保存不足； |
| ● | 我們與合同製造臨床和商業用品的第三方製造商的製造流程或設施可能無法滿足FDA、EMA或類似的外國監管機構的要求； |
| ● | 美國食品和藥物管理局（FDA）、歐洲藥品管理局（EMA）或類似的外國監管機構可能未能批准我們計劃與合作伙伴內部開發的伴隨診斷設備;且 |
| ● | 醫療標準變更或美國FDA、歐洲EMA或類似的外國監管機構的批准政策或法規可能發生顯著變化，使我們的臨床數據不足以獲得批准。 |
獲得市場許可的審批過程所需的時間和費用，以及未來臨床試驗結果和其他影響因素的不可預測性，可能導致我們未能獲得NXP800、NXP900或我們未來可能尋求開發的任何其他藥物候選品的監管批准，這將嚴重損害我們的業務、運營結果和前景。在這種情況下，我們可能也沒有資源進行新的臨床試驗和/或我們可能判斷進一步開發任何此類藥物候選品沒有正當理由，可能會中止任何此類項目。
此外，即使我們在一個或多個司法管轄區獲得監管批准，監管機構也可能只批准我們的任一藥物候選品用途較少或受限制的適應症，可能不會批准我們提出的產品收費價格，可能批准取決於昂貴的後市場臨床試驗表現（根據司法轄區不同分別稱爲「有條件」的或「加速」的批准），或可能批准的藥物候選品標籤中不包含對該藥物候選品成功商業化所必要或理想的標籤聲明。前述任何情況均可能對我們藥物候選品的商業前景造成重大損害。
在一個司法管轄區獲得我們當前或未來產品候選品的監管批准並不意味着我們將能夠成功在其他司法管轄區獲得我們當前或未來產品候選品的監管批准。
在一個司法管轄區獲得任何我們當前或未來產品候選品的監管批准並不保證我們將能夠在其他任何司法管轄區獲得或保持監管批准，而在一個司法管轄區獲得監管批准失敗或延遲可能對其他司法管轄區的監管批准流程產生負面影響。例如，即使FDA批准了產品候選品，類似的外國監管機構也必須批准在這些國家中的產品候選品的製造、營銷和促銷。各司法管轄區的藥品批准程序各不相同，並可能涉及比美國更多、更大的要求和行政審查期，包括額外的前期臨床研究或臨床試驗，因爲在一個司法管轄區進行的臨床試驗可能不被其他司法管轄區的監管機構接受。在美國以外的許多司法管轄區，產品候選品必須獲得報銷批准後才能在該司法管轄區銷售。在某些情況下，我們打算爲產品收費的價格也需獲得批准。
我們也可以在其他國家提交市場申請。美國以外的司法管轄區的監管機構對產品候選者的批准有要求，我們必須在那些司法管轄區開始營銷前遵守這些要求。獲得類似的國外監管批准，並符合類似的國外監管要求可能導致我們遇到重大延遲、困難和成本，這可能會延遲或阻止我們在某些國家推出我們的產品。我們目前沒有任何產品候選者獲得批准在任何司法管轄區出售，包括國際市場，也沒有獲得國際市場監管批准的經驗。如果我們未能遵守國際市場的監管要求和/或獲得適用的市場批准，我們的目標市場將受到影響，我們無法實現當前或未來產品候選者的全部市場潛力。
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即使我們的現有或未來產品候選獲得監管批准，我們將需要遵守持續的監管義務和持續的監管審查，這可能導致額外的重大開支，如果我們未能遵守監管要求或與我們的現有或未來產品候選出現意外問題，我們可能會受到處罰。
如果我們的任一現有或未來產品候選獲得批准，則對產品的製造、標籤、包裝、存儲、廣告、促銷、取樣和產品記錄將受到廣泛和持續的監管要求。這些要求包括提交安全性和其他上市後信息和報告，註冊，以及持續遵守cGMP法規。藥品製造商和負責任何產品製造過程的任何CMO都必須遵守廣泛的FDA和外國類似監管機構要求，包括確保質量控制和製造程序符合cGMP法規和任何適用的外國等效物。因此，我們及我們的CMO將接受持續的審查和檢查，以評估對cGMP的遵守情況和對在任何NDA、其他市場申請和先前對檢查觀察的回應中所做承諾的遵守情況。因此，我們和我們合作的其他人必須繼續在監管遵從的所有領域，包括製造、生產和質量控制方面，花費時間、金錢和精力。
FDA或類似的外國監管機構還可能對昂貴的上市後非臨床研究或臨床試驗（通常稱爲「第4期試驗」）和上市後監測提出要求，以監測產品的安全性或有效性。如果我們或監管機構發現產品存在先前未知的問題，如嚴重或頻繁的意外不良事件、生產問題或在生產或加工產品的設施存在問題，如產品污染或不符合適用的cGMP法規在內，監管機構可能對該產品、製造工廠或我們實施限制。如果我們或第三方提供商，包括我們的CMO未能充分遵守適用法規，則我們可能需要啓動產品的召回或下架。
對我們目前或未來產品候選項的原先未知問題的後期發現，包括意外嚴重程度或頻率的不良事件，或與我們的第三方製造商或製造流程有關的問題，或未能遵守監管要求，可能導致以下情況，以及其他事項：
| ● | 對產品的製造、已批准生產商或製造流程實施限制； |
| ● | 對產品的標籤或市場營銷有所限制； |
| ● | 對產品分銷或使用實施限制； |
| ● | 要求進行後市場研究或臨床試驗； |
| ● | 將產品從市場上撤回; |
| ● | 產品召回; |
| ● | 美國食品藥品監督管理局（FDA）的警告信或無標題信函，或來自國外監管當局的類似違規通知； |
| ● | FDA或其他適用的監管機構拒絕批准待批准申請或批准申請的補充； |
| ● | 罰款、賠償或返還利潤或收益； |
| ● | 撤銷或暫停市場準入批文； |
| ● | 暫停我們進行中的任何臨床試驗； |
| ● | 產品被扣押或拘留或拒絕允許進口或出口; 和 |
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| ● | consent decrees, injunctions or the imposition of civil or criminal penalties. |
In addition, regulatory authorities’ policies (such as those of the FDA or EMA) may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our current or future product candidates. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are otherwise not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained, which would adversely affect our business, prospects and ability to achieve or sustain profitability.
Non-compliance with European Union requirements regarding safety monitoring or pharmacovigilance can also result in significant financial penalties. Similarly, failure to comply with the European Union’s requirements regarding the protection of personal information can also lead to significant penalties and sanctions.
The FDA’s policies may change and additional government regulations may be enacted that could prevent, limit or delay marketing approval of our current or future product candidates. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, this may adversely affect, or even lead to the rescission of, the marketing approval that we may have obtained, which would adversely affect our business, prospects and ability to achieve or sustain profitability.
A variety of risks associated with marketing our current or future product candidates internationally could materially adversely affect our business.
We plan to seek regulatory approval of our current or future product candidates outside of the United States and expect that we will be subject to additional risks related to operating in foreign countries including: differing regulatory requirements; unexpected changes in tariffs, trade barriers, price and exchange controls; economic weakness, including inflation, or political instability in particular foreign economies and markets; compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; foreign currency fluctuations that result in increased operating expenses, reduced revenue, and other obligations incident to doing business in another country; potential liability under the Foreign Corrupt Practices Act of 1977 or comparable foreign regulations; and challenges enforcing our contractual and intellectual property rights, especially in countries that do not recognize intellectual property rights to the same extent as the United States.
The insurance coverage and reimbursement status of newly approved products is uncertain. Our current or future product candidates may become subject to unfavorable pricing regulations, third-party coverage and reimbursement practices, or healthcare reform initiatives, which would harm our business. Failure to obtain or maintain adequate coverage and reimbursement for new or current products could limit our ability to market those products and decrease our ability to generate revenue.
Adverse pricing limitations may hinder our ability to recoup our investment in one or more of our current or future product candidates, even if any such current or future product candidate we may develop obtains marketing approval.
Our ability to successfully commercialize any current or future product candidates will depend in part on the coverage and reimbursement for the products and related treatments from government health administration authorities and third-party payors, such as private health insurers and health maintenance organizations. These organizations decide which medications they will pay for and establish reimbursement levels. If coverage and adequate reimbursement is not available, or the approved reimbursement amount is not high enough, we may be unable to establish or maintain pricing sufficient to generate a return on our investment and may be unable to successfully commercialize our current or future product candidates. Reimbursement by a third-party payor may depend upon a number of factors, including, but not limited to, the third-party payor’s determination that use of a product is a covered benefit under its health plan, safe, effective and medically necessary, appropriate for the specific patient, cost-effective, and neither experimental nor investigational. If coverage and adequate reimbursement is not available, or the approved reimbursement amount is not high enough, we may be unable to establish or maintain pricing sufficient to generate a return on our investment and may be unable to successfully commercialize our current or future product candidates.
A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Government authorities and third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. In general, the prices of medicines under such systems are substantially lower than in the United States.
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There is also significant uncertainty related to the insurance coverage and reimbursement of newly approved products, and coverage may be more limited than the purposes for which the medicine is approved by the FDA or comparable foreign regulatory authorities. In the United States, the principal decisions about reimbursement for new medicines are typically made by CMS. As a result, the coverage determination process is often a time consuming and costly process that may require us to provide scientific and clinical support for the use of our products to each payor separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance. It is difficult to predict what CMS will decide with respect to reimbursement for fundamentally novel products such as ours. Reimbursement agencies in Europe may be more conservative than CMS. Our inability to promptly obtain coverage and profitable payment rates from both government-funded and private payors for any approved products we may develop could have a material adverse effect on our operating results, our ability to raise capital needed to commercialize our current or future product candidates, and our overall financial condition.
Healthcare legislative measures and changes in policies, funding, staffing and leadership at the FDA and other agencies could hinder or prevent the commercial success of our products.
In the United States, there have been a number of legislative and regulatory changes to the healthcare system that could affect our future results of operations and the future results of operations of our potential customers.
In recent years, there has been heightened governmental scrutiny over the manner in which biopharmaceutical manufacturers set prices for their marketed products, which has resulted in several recent government inquiries as well as federal and state legislation designed to, among other things, increase drug price transparency, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform government reimbursement for drug products. Congress and the executive branch have each indicated that they will continue to seek new legislative and/or administrative measures to control drug costs, making this area subject to ongoing uncertainty. At the state level in the United States, legislatures have also increasingly passed legislation and implemented regulations designed to control drug product pricing.
While we cannot predict what impact these laws or policies will have in general or specifically on any product we may commercialize in the future, such efforts by the government and payors may result in downward pressure on reimbursement, which could negatively affect market acceptance of new products. Any rebates, discounts, taxes costs or regulatory or systematic changes on healthcare may have a significant effect on our profitability in the future.
Given recent federal and state government initiatives directed at lowering the total cost of healthcare, the executive branch, Congress and state legislatures will likely continue to focus on healthcare reform and the reform of the Medicare and Medicaid programs. While we cannot predict the full outcome of any such government action or legislation, it may harm our ability to market our products and generate revenues.
Furthermore, regulatory authorities’ assessment of the data and results required to demonstrate safety and effectiveness can change over time and can be affected by many factors, such as the emergence of new information, including on other products, changing policies and agency funding, staffing and leadership. We cannot be sure whether future changes to the regulatory environment will be favorable or unfavorable to our business prospects.
Our future relationships with customers and third-party payors in the United States and elsewhere may be subject to applicable anti-kickback, fraud and abuse, false claims, transparency, health information privacy and security and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm, administrative burdens, diminished profits and future earnings.
Healthcare providers, physicians and third-party payors in the U.S. and elsewhere will play a primary role in the recommendation and prescription of any current or future product candidates for which we obtain marketing approval. Our future arrangements with third-party payors and customers may expose us to broadly applicable fraud and abuse and other healthcare laws and regulations, including, without limitation, the federal Anti-Kickback Statute and the federal False Claims Act, which may constrain the business or financial arrangements and relationships through which we sell, market and distribute any current or future product candidates for which we obtain marketing approval. In addition, we may be subject to transparency laws and patient privacy regulation by the federal and state
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governments and by governments in foreign jurisdictions in which we conduct our business. The applicable federal, state and foreign healthcare laws and regulations that may affect our ability to operate include, but are not necessarily limited to:
| ● | the federal Anti-Kickback Statute, which 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, or in return for, 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; |
| ● | federal civil and criminal false claims laws and civil monetary penalty laws, including the federal False Claims Act, which impose criminal and civil penalties, including civil whistleblower or qui tam actions, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, including the Medicare and Medicaid programs, 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; the federal Health Insurance Portability and Accountability Act of 1996 (“HIPAA”), which imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters; |
| ● | HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, and their respective implementing regulations, which impose obligations on covered healthcare providers, health plans, and healthcare clearinghouses, as well as their business associates that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity, with respect to safeguarding the privacy, security and transmission of individually identifiable health information; |
| ● | the federal Open Payments program, which requires manufacturers of certain drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to CMS, information related to “payments or other transfers of value” made to “covered recipients,” which include physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors, and teaching hospitals) and applicable manufacturers. Applicable group purchasing organizations also are required to report annually to CMS the ownership and investment interests held by the physicians and their immediate family members. The SUPPORT for Patients and Communities Act added to the definition of covered recipient practitioners including physician assistants, nurse practitioners, clinical nurse specialists, certified registered nurse anesthetists and certified nurse-midwives effective in 2022. Data collection began on August 1, 2013 with requirements for manufacturers to submit reports to CMS by March 31, 2014 and 90 days after the end of each subsequent calendar year. Disclosure of such information was made by CMS on a publicly available website beginning in September 2014; and |
| ● | analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers; state and foreign data privacy or data protection laws and regulations, such as state health data privacy legislation, state data breach legislation, or general state privacy legislation such as California’s Consumer Privacy Act (CCPA) and its implementing regulations; state and foreign laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restrict payments that may be made to healthcare providers; state and foreign laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; and state and foreign 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 HIPAA, thus complicating compliance efforts. |
In November 2020, HHS finalized significant changes to the regulations implementing the Anti-Kickback Statute, as well as the Physician Self-Referral Law and the civil monetary penalty rules regarding beneficiary inducements, with the goal of offering the healthcare industry more flexibility and reducing the regulatory burden associated with those fraud and abuse laws, particularly with respect to value-based arrangements among industry participants.
Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations may involve substantial costs. It is possible that governmental authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our
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operations are 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, including, without limitation, damages, fines, imprisonment, exclusion from participation in government healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations, which could have a material adverse effect on our businesses. If any of the physicians or other healthcare providers or entities with whom we expect to do business, including our collaborators, is found not to be in compliance with applicable laws, it may be subject to criminal, civil or administrative sanctions, including exclusions from participation in government healthcare programs, which could also materially affect our businesses.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
We are subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. Our operations may involve the use of hazardous and flammable materials, including chemicals and biological and radioactive materials. Our operations also may produce hazardous waste products. We currently contract with third parties for the conduct of our manufacturing efforts and preclinical studies and clinical trials and such third parties are responsible for disposal of these materials and wastes. However, we cannot eliminate our risk of contamination or injury from these materials. In the event of contamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines and penalties.
Although we maintain workers’ compensation insurance to cover us for costs and expenses, we may incur due to injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities. We do not maintain insurance for environmental liability or toxic tort claims that may be asserted against us in connection with our storage or disposal of biological, hazardous or radioactive materials.
Risks Related to our Intellectual Property
We currently hold a license to certain intellectual property rights relating to our lead product candidate, NXP800 and to NXP900, as well as intellectual property rights relating to other compounds that modulate HSF1 and the SRC and YES1 kinases. If we are unable to maintain patent and other intellectual property protection for NXP800 and NXP900, and to obtain and maintain patent and other intellectual property protections for our other current or future product candidates and technology, or if the scope of intellectual property protection obtained or maintained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to commercialize NXP800, NXP900 or any other current or future product candidates or technology may be adversely affected.
Our success depends in large part on our ability to obtain and maintain patent and other intellectual property protection in the United States and other countries with respect to our current or future product candidates, including NXP800 and NXP900, their respective components, formulations, combination therapies, methods used to manufacture them and methods of treatment and development that are important to our business, as well as successfully defending these patents against third-party challenges. If we do not adequately protect our intellectual property rights, or if the intellectual property rights we are able to obtain are insufficiently broad and exclusive, competitors may be able to erode or negate any competitive advantage we may have, which could harm our business and ability to achieve profitability.
We intend to rely upon a combination of patents, patent applications, confidentiality agreements, trade secret protection and license agreements to protect the intellectual property related to our current or future product candidates and technologies. Any disclosure to or misappropriation by third parties of our confidential proprietary information could enable competitors to quickly duplicate or surpass our technological achievements, thus eroding our competitive position in our market. We, or any current or future partners, collaborators, or licensees, may fail to identify patentable aspects of inventions made in the course of development and commercialization activities before it is too late to obtain patent protection on them. We may be also unable to exclusively license relevant technology and associated intellectual property developed by others. Therefore, we may miss potential opportunities to establish our patent position.
If we are unable to secure additional patent protection or maintain existing or future patent protection with respect to NXP800, NXP900, or any other proprietary products and technology we develop, our business, financial condition, results of operations, and prospects would be materially harmed.
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We currently hold a license to certain intellectual property rights relating to NXP800, including its composition of matter and to other compounds that modulate HSF1 (activate the GCN2 kinase). In addition, we hold a license to certain intellectual property relating to NXP900, including its composition of matter and to other compounds that inhibit the SRC and YES1 kinases.
We have licensed one patent family covering the composition of matter for NXP800, including two issued U.S. patents covering the composition of matter for NXP800, as well as methods for using and making NXP800. Additionally, patents have been issued in major markets, including the U.S., the European Union, and Japan. The statutory expiration for the issued U.S. patents in this family is October 2034, without considering any patent extensions that may or may not be possible.
We have licensed a patent family directed to additional compounds that modulate HSF1. A patent from this family has been granted in the U.S., and has a statutory expiration of April 2036, without considering any patent extensions that may or may not be possible.
We have also licensed a patent family directed to deuterated compounds that modulate HSF1. Any U.S. patent that grants from this family would have a statutory expiration of October 2037, without considering any patent extensions or patent disclaimers that may or may not be possible.
We have licensed one patent family covering the composition of matter for NXP900, which has been granted in the U.S., EU, Japan, China and is pending in the United Kingdom and Canada. The statutory expiration for patents in this patent family is April 2036, without considering any possible patent term extension.
If the scope of our patent protection, whether now or in the future, with respect to NXP800, NXP900 or our future product candidates and technology is not sufficiently broad, we will be unable to prevent others from using our technology or from developing or commercializing technology and products similar or identical to ours or other competing products and technologies. Any failure to obtain or maintain patent protection, through our own patents or through in-licensing, with respect to NXP800, NXP900 and our future product candidates would have a material adverse effect on our business, financial condition, results of operations and prospects.
Even if they are unchallenged, our patent applications, if issued, and any patents we may own or in-license now or in the future, may not provide us with any meaningful protection or prevent competitors from designing around our patent claims to circumvent any patents we may own or in-license in the future by developing similar or alternative technologies or therapeutics in a non-infringing manner. If the patent protection provided by our patent applications or any patents we may pursue with respect to our current or future product candidates is not sufficiently broad to impede competition, our ability to successfully commercialize our current or future product candidates could be negatively affected, which would harm our business.
Additionally, we cannot be certain that the claims in our patent applications covering composition of matter (or other related aspects) of our current or future product candidates or technology will be considered patentable by the USPTO, or by patent offices in foreign countries, or that the claims in any issued patents we may own or in-license in the future will be considered patentable by courts in the United States or foreign countries.
The issuance of a patent does not foreclose challenges to its inventorship, scope, validity or enforceability. Therefore, our owned and in-licensed patents may be challenged in the courts or patent offices in the United States and elsewhere. Such challenges 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. Successful patent challenges 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 in-licensed patents may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
Moreover, patents or patent applications owned or filed by us, or by our licensors or other collaborators, may be challenged or narrowed 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. An adverse determination in any such submission, Patent Trial and Appeal Board 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
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our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.
If we fail to comply with our obligations in our current license agreements, or in any future agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our current or future licensors, we could lose license rights that are important to our business.
We are currently party to a license which grants us certain intellectual property rights relating to our lead product candidate, NXP800, as well as other related compounds, and to a license which grants us certain intellectual property rights relating to our second drug candidate, NXP900, as well as other compounds that inhibit the SRC and YES1 kinases. These agreements impose numerous obligations on us to maintain our licensing rights, including development, diligence, payment, commercialization, funding, milestone, royalty, sublicensing, insurance, patent prosecution, enforcement and other obligations. In spite of our efforts, our licensor might conclude that we have materially breached our license agreement and might therefore terminate the license agreement, thereby removing or limiting our ability to develop and commercialize NXP800 or NXP900 (and other compounds covered by the licenses).
Additionally, in the future, we may be party to other license or collaboration agreements with third parties to advance our research or allow commercialization of current or future product candidates. Such future agreements may impose numerous obligations, such as development, diligence, payment, commercialization, funding, milestone, royalty, sublicensing, insurance, patent prosecution, enforcement and other obligations on us and may require us to meet development timelines, or to exercise commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses. In spite of our efforts, our future licensors might conclude that we have materially breached our future license agreements and might terminate the license agreements, thereby removing or limiting our ability to develop and commercialize products and technologies covered by these license agreements.
Any termination of these current or future licenses, or failure of the underlying patents to provide the intended exclusivity, could result in the loss of significant rights and could harm our ability to commercialize our current or future product candidates, and competitors or other third parties would have the freedom to seek regulatory approval of, and to market, products identical to ours and we may be required to cease our development and commercialization of certain of our current or future product candidates. Any of the foregoing could have a material adverse effect on our competitive position, business, financial conditions, results of operations, and prospects.
If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
In addition to the protection afforded by patents we may own or in-license in the future, we seek to rely on trade secret protection, confidentiality agreements, and license agreements to protect proprietary know-how that is not patentable, processes for which patents are difficult to enforce, and any other elements of our product discovery and development processes that involve proprietary know-how, information, or technology that is not covered by patents. Although we require all of our employees, consultants, advisors and any third parties who have access to our proprietary know-how, information, or technology to enter into confidentiality agreements, trade secrets can be difficult to protect and we have limited control over the protection of trade secrets used by our collaborators and suppliers.
If we are unable to prevent unauthorized material disclosure of our intellectual property to third parties, we will not be able to establish or maintain a competitive advantage in our market, which could materially adversely affect our business, financial condition, results of operations and future prospects.
Third-party claims of intellectual property infringement, misappropriation or other violations may be costly and time consuming and may prevent or delay our product discovery and development efforts.
The intellectual property landscape around precision medicine is crowded, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights; the outcome of which would be uncertain and could have a material adverse effect on the success of our business. We or any of our future licensors or strategic partners may be party to, exposed to, or threatened with, future adversarial proceedings or litigation by third parties having patent or other intellectual property rights alleging that our current or future product candidates and/or proprietary technologies infringe, misappropriate or otherwise violate their intellectual property rights. Thus, because of the large number of patents issued and patent applications filed in our fields, there may be a risk that third parties may allege they have patent rights encompassing our current or future product candidates, technologies or methods.
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Third parties may assert that we are employing their proprietary technology without authorization. In addition, because some patent applications in the United States may be maintained in secrecy until the patents are issued, patent applications in the United States and many foreign jurisdictions are typically not published until 18 months after filing, and publications in the scientific literature often lag behind actual discoveries, we cannot be certain that others have not filed patent applications covering our current or future product candidates or technology. If any such patent applications issue as patents, and if such patents have priority over patent applications or patents we own or in-license, we may be required to obtain rights to such patents owned by third parties which may not be available on commercially reasonable terms or at all, or may only be available on a non-exclusive basis.
In the event of a successful claim of infringement, misappropriation or other violation against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, obtain one or more licenses from third parties, pay royalties or redesign our infringing products, which may be impossible or require substantial time and monetary expenditure.
Changes to patent law in the United States and in foreign jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.
Our success is heavily dependent on intellectual property, particularly patents. Recent U.S. Supreme Court rulings have narrowed the scope of patent protection available in certain circumstances and weakened the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained. Laws and regulations governing patents could further change in unpredictable ways that could weaken our ability to obtain new patents or to enforce patents that we own or in-license now, or that we might obtain or in-license in the future.
We may be subject to claims challenging the inventorship or ownership of our intellectual property, including any patents we may own or in-license currently or in the future.
We may be subject to claims that former employees, collaborators or other third parties have an interest in any patents we may own or in-license currently or in the future, trade secrets, or other intellectual property as an inventor or co-inventor. Litigation may be necessary to defend against these and other claims challenging inventorship of any patents we may own or in-license in the future, trade secrets or other intellectual property, which may require substantial time and monetary expenditure.
We may be subject to claims that we or our employees, consultants or independent contractors have wrongfully used or disclosed confidential information or alleged trade secrets of third parties or competitors or breached non-competition or non-solicitation agreements with our competitors.
We may be subject to claims that we or our employees, consultants or independent contractors have inadvertently or otherwise used or disclosed confidential information or trade secrets of third parties or competitors or our employees’ former employers or our consultants’ or contractors’ current or former clients or customers. Litigation or arbitration may be necessary to defend against these claims, which may require substantial time and monetary expenditure.
If we do not obtain patent term extension and data exclusivity for any of our current or future product candidates we may develop, our business may be materially harmed.
Depending upon the timing, duration and specifics of any FDA marketing approval of any of our current or future product candidates we may develop, one or more U.S. patents we may own or in-license in the future may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Act. If we are unable to obtain patent term extension or the term of any such extension is shorter than what we request, our competitors may obtain approval of competing products following expiration of any patents that issue from our patent applications, and our business, financial condition, results of operations, and prospects could be materially harmed.
If our trademarks and trade names are not adequately protected, we may not be able to build name recognition in our marks of interest and our business may be adversely affected.
Our trademarks or trade names may be challenged, infringed, diluted, circumvented or declared generic or determined to be infringing on other marks. We intend to rely on both registration and common law protection for our trademarks. We may not be able to protect
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our rights to these trademarks and trade names or may be forced to stop using these names, which we need for name recognition by potential partners or customers in our markets of interest. If we are unable to obtain a registered trademark or establish name recognition based on our trademarks and trade names, we may not be able to compete effectively and our business may be adversely affected.
Risks Related to our Reliance on Third Parties
We plan to rely on third parties to conduct our preclinical studies and clinical trials. If these third parties do not properly and successfully carry out their contractual duties or meet expected deadlines, we may not be able to obtain regulatory approval of or commercialize our current or future product candidates.
We plan to utilize and depend upon independent investigators and collaborators, such as medical institutions, CROs, CMOs, and strategic partners to conduct and support our preclinical studies and clinical trials under agreements with us. We rely upon, and plan to continue to rely upon, such third-party entities to execute our clinical trials and preclinical studies and to monitor and manage data produced by and relating to those studies and trials. However, in the future we may not be able to establish arrangements with CROs when needed or on terms that are acceptable to us, or at all, which could negatively affect our development efforts with respect to our drug candidates and materially harm our business, operations and prospects. As a result of the use of third-party contractors, we will have only limited control over certain aspects of their activities. Nevertheless, we are responsible for ensuring that each of our studies, including each of our clinical trials, is conducted in accordance with the applicable protocol, legal and regulatory requirements as well as scientific standards, and our reliance on any third-party entity will not relieve us of our regulatory responsibilities.
Based on our present expectations, we and our third-party contractors will be required to comply with GCP regulations for the clinical development of all of our drug candidates. If we or any of these third parties fail to comply with applicable GLP or GCP regulations, the clinical data generated in our preclinical and 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, which we may not have sufficient cash or other resources to support and which would delay our ability to generate revenue from future sales of such drug candidate. Any agreements governing our relationships with CROs or other contractors with whom we currently engage or may engage in the future may provide those outside contractors with certain rights to terminate a clinical trial under specified circumstances. If such an outside contractor terminates its relationship with us during the performance of a clinical trial, we would be forced to seek an engagement with a substitute contractor, which we may not be able to do on a timely basis or on commercially reasonable terms, if at all, and the applicable clinical trial would experience delays or may not be completed.
Large-scale clinical trials require significant additional financial and management resources and reliance on third-party clinical investigators, CROs, and consultants, which may cause us to encounter delays that are outside of our control. We may be unable to identify and contract with sufficient investigators, CROs, or consultants on a timely basis, if at all.
If these third parties 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, legal and regulatory requirements or for other reasons, our preclinical or clinical trials may be extended, delayed or terminated and we may not be able to complete development of, obtain regulatory approval for, or successfully commercialize, our current or future product candidates. In addition, we will be unable to control whether or not they devote sufficient time and resources to our preclinical and clinical programs. These outside contractors may not assign as great a priority to our programs or pursue them as diligently as we would if we were undertaking such programs ourselves. As a result, our operations and the commercial prospects for the effected drug candidates would be harmed, our costs could increase and our ability to generate revenues could be delayed. These contractors may also have relationships with other commercial entities, some of whom may compete with us. If our contractors assist our competitors to our detriment, our competitive position would be harmed.
If our relationships with any third parties conducting our studies are terminated, we may be unable to enter into arrangements with alternative third parties on commercially reasonable terms, or at all. Switching or adding third parties to conduct our studies involves substantial cost and requires extensive management time and focus. In addition, there is a natural transition period when a new third party commences work. As a result, delays occur, which can materially impact our ability to meet our desired clinical development timelines. Although we carefully manage our relationships with third parties conducting our studies, we cannot assure you that we will not encounter similar challenges or delays in the future or that these delays or challenges will not have a material and adverse effect on our business, financial condition and results of operations.
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We rely, and expect to continue to rely, on the third-party manufacturers to manufacture our current or future product candidates. Reliance on third parties increases the risk that we will not have sufficient quantities of our products or such quantities at an acceptable quality and cost, which could delay, prevent or impair our development or commercialization efforts.
We do not currently own any facility that may be used as our clinical-scale manufacturing and processing facility and must rely on outside vendors to manufacture our current or future product candidates. We rely on a single CMO to make the NXP800 drug substance and finished drug product, each performed at a different manufacturing facility. We rely on a single CMO to manufacture NXP900 drug substance and another single CMO to manufacture NXP900 drug product. There is no assurance that we will be able to retain these relationships, and if we are unable to maintain these relationships, we could experience delays in our development efforts. There is no assurance that our CMOs will be successful in manufacturing NXP800 and/or NXP900 drug substance or product. If NXP800, NXP900 or any other drug candidate we may develop or acquire in the future receives regulatory approval, we will likely rely on one or more CMOs to manufacture the commercial supply of such drugs.
Our anticipated reliance on a limited number of third-party manufacturers exposes us to a number of risks, including:
| ● | due to the limited number of potential manufacturers, and because the FDA requires inspection of any manufacturers’ cGMP compliance as part of our marketing application, we may be unable to identify manufacturers on acceptable terms, if at all; |
| ● | a new manufacturer would have to be educated in and develop substantially equivalent processes for, the production of our current or future product candidates; |
| ● | our third-party manufacturers might be unable to timely manufacture our current or future product candidates or produce the quantity and quality required to meet our clinical and commercial needs due to a variety of potential reasons including failure to achieve drug substance or drug product specifications, batch to batch inconsistencies, site or equipment contaminations, failure to scale up as needed in a cost-efficient manner, failed regulatory inspections, competition for production capacity and availability from other customers; |
| ● | we may not own, or may have to share, the intellectual property rights to any improvements made by our third-party manufacturers in the manufacturing process for our current or future product candidates; |
| ● | our third-party manufacturers could breach or terminate their agreements with us; |
| ● | our third-party manufacturers might be unable to formulate and manufacture our drugs in the volume and of the quality required to meet our clinical and commercial needs, if any; |
| ● | our third-party manufacturers may not perform as contractually agreed or may not remain in the contract manufacturing business for the time required to supply our clinical trials or to successfully produce, store and distribute our products; |
| ● | drug manufacturers are subject to ongoing periodic unannounced inspection by the FDA and some state agencies in the United States, as well as foreign regulatory authorities, to ensure strict compliance with cGMP regulations and other regulatory requirements; and |
| ● | raw materials and components used in the manufacturing process, particularly those for which we have no other source or supplier, may not be available or may not be suitable or acceptable for use due to material or component defects. |
Each of these risks could delay or prevent the completion of our preclinical or clinical trials or the approval of any of our current or future product candidates by the FDA or another foreign regulatory authority, result in higher costs or adversely impact commercialization of our current or future product candidates.
Although our agreements with our CMOs require them to perform according to certain cGMP requirements such as those relating to quality control, quality assurance and qualified personnel, we cannot control the conduct of our CMOs to implement and maintain these standards. If any of our CMOs cannot successfully manufacture material that conforms to our specifications and the regulatory
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requirements of the FDA, EMA or other comparable foreign authorities, we could be prevented from obtaining regulatory approval for our drug candidates unless and until we engage a substitute CMO that can comply with such requirements, which we may not be able to do. Any such failure by any of our CMOs would significantly impact our ability to develop, obtain regulatory approval for or market our drug candidates, if approved.
If our third-party manufacturers use hazardous and biological materials in a manner that causes injury or violates applicable law, we may be liable for damages.
Although we believe that our manufacturers’ procedures for using, handling, storing, and disposing of hazardous and biological materials comply with legally prescribed standards, we cannot completely eliminate the risk of contamination or injury. In the event of an accident, local, city, state or federal authorities may curtail the use of these materials and interrupt our business operations. Further, we could be held liable for damages or penalized with fines, and the liability could exceed our resources. We do not have any insurance for liabilities arising from medical or hazardous materials.
Risks Related to Managing Growth and Employee Matters
We are highly dependent on our key personnel and anticipate hiring new key personnel. If we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.
Our ability to compete in the highly competitive biotechnology and pharmaceutical industries depends upon our ability to attract and retain highly qualified managerial, scientific and medical personnel. We are highly dependent on our management, scientific and medical personnel, including our Chairman, Chief Executive Officer and President, our Chief Scientific and Business Officer and our Chief Development and Operations Officer. While we expect to engage in an orderly transition process as we integrate newly appointed officers and managers, we face a variety of risks and uncertainties relating to management transition, including diversion of management attention from business concerns, failure to retain other key personnel or loss of institutional knowledge.
We will need to grow the size of our organization, and we may experience difficulties in managing this growth.
As of November 1, 2024, we had 13 full-time employees. We also contract for various services through consulting and vendor agreements. We intend to hire new employees to conduct our research and development activities in the future. Any delay in hiring such new employees could result in delays in our research and development activities and would harm our business. As our development and commercialization plans and strategies develop, and as we transition into operating as a public company, we expect to need additional managerial, operational, sales, marketing, financial and other personnel, as well as additional facilities to expand our operations.
If we are not able to effectively expand our organization by hiring new employees and expanding our groups of consultants and contractors, or we are not able to effectively build out new facilities to accommodate this expansion, we may not be able to successfully implement the tasks necessary to further develop and commercialize our current or future product candidates and, accordingly, may not achieve our research, development and commercialization goals.
We will incur significant increased costs as a result of operating as a public company, and our management will be required to devote substantial time to compliance activities and initiatives.
As a public company, we will incur significant legal, accounting, and other expenses that we did not incur as a private company. We are now subject to the reporting requirements of the Securities Exchange Act of 1934, as amended, which requires, among other things, that we file with the SEC, annual, quarterly, and current reports with respect to our business and financial condition. In addition, the Sarbanes-Oxley Act of 2002 (“SOX”), as well as rules subsequently adopted by the SEC and the Nasdaq Capital Market to implement provisions of SOX, impose significant requirements on public companies, including requiring establishment and maintenance of effective disclosure and financial controls and changes in corporate governance practices.
Moreover, these rules and regulations will increase our legal and financial compliance costs and make some activities more time-consuming and costly. For example, these rules and regulations make it more difficult and more expensive for us to obtain director and officer liability insurance, and we may be required to accept reduced policy limits and coverage or incur substantially higher costs to obtain the same or similar coverage. As a result, it may be more difficult for us to attract and retain qualified persons to serve on our Board of Directors, our Board committees or as executive officers.
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SOX 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 SOX. 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, which could have an adverse effect on the market price of our stock.
Our business and operations would suffer in the event of computer system failures, cyber-attacks, or deficiencies in our or third parties’ cybersecurity.
We are increasingly dependent upon information technology systems, infrastructure, and data to operate our business. In the ordinary course of business, we may collect, store, and transmit confidential information, including, but not limited to, information related to our intellectual property and proprietary business information, personal information, and other confidential information. We have outsourced elements of our operations to third party vendors, who each have access to our confidential information, which increases our disclosure risk. Although we have implemented internal security and business continuity measures, our information technology and other internal infrastructure systems may breakdown, incur damage or be interrupted by system malfunctions, natural disasters, terrorism, war, or telecommunication and electrical failures, as well as by inadvertent or intentional security breaches by our employees, contractors, consultants, business partners, and/or other third parties, or from cyber-attacks by malicious third parties, each of which could compromise our system infrastructure or lead to the loss, destruction, alteration, disclosure, or dissemination of, or damage or unauthorized access to, our data or other assets. Such a security breach may cause loss, damage, or disclosure of proprietary or confidential information, which could in turn result in significant legal and financial exposure and reputational damage that could adversely affect our business. Furthermore, the loss or corruption of clinical trial data from future clinical trials may result in delays in our regulatory approval efforts and could significantly increase our costs to recover or reproduce the data.
The costs related to significant security breaches or disruptions could be material and our insurance policies may not be adequate to compensate us for the potential losses arising from any such security breach. In addition, such insurance may not be available to us on economically reasonable terms, if at all, may not cover all claims made against us, and may have high deductibles. Furthermore, if the information technology systems of our third-party vendors and other contractors and consultants become subject to disruptions or security breaches, we may have insufficient recourse against such third parties and we may have to expend significant resources to mitigate the impact of such an event, and to develop and implement protections to prevent future events of this nature from occurring.
Risks Related to Commercial Activities
If any of our current or future product candidates do not achieve broad market acceptance among physicians, patients, healthcare payors and the medical community, the revenues from any such current or future product candidate may be limited.
The use of precision medicines as a potential cancer treatment is a recent development and may not become broadly accepted by physicians, patients, hospitals, cancer treatment centers, and others in the medical community. We cannot predict whether physicians, patients, hospitals, cancer treatment centers, and government agencies or third-party payors will determine that our product is safe, therapeutically effective, and cost effective as compared with competing treatments. If our current or potential future product candidates do not achieve an adequate level of market acceptance, we may not generate significant product revenues and may not become profitable. Factors influencing acceptance of our current or future product candidates in the market, include: the clinical indications for which our product candidates are licensed; whether our product candidates are viewed as a safe and effective treatment; our ability to demonstrate our product’s advantages, including cost advantages, over alternative treatments; the prevalence and severity of any side effects of our products and of other precision medicines; product labeling or product insert requirements of the FDA or other regulatory authorities and limitations or warnings contained in the labeling; the timing of market introduction of our product candidates and competitive products; patient willingness to pay out-of-pocket in the absence of coverage by third-party payors and government authorities; and the effectiveness of our sales and marketing efforts.
If our current or future product candidates are licensed but fail to achieve market acceptance among physicians, patients, hospitals, cancer treatment centers or others in the medical community, we will not be able to generate significant revenue. In addition, although
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our current or future product candidates may differ in certain ways from other precision medicine approaches, serious adverse events or deaths in other preclinical or clinical trials involving precision medicines, even if not ultimately attributable to our current or future products or product candidates, could result in increased government regulation, unfavorable public perception and publicity, potential regulatory delays in the testing or licensing of our current or future product candidates, stricter labeling requirements for those product candidates that are licensed, and a decrease in demand for any such product candidates.
If product liability lawsuits are brought against us, we may incur substantial liabilities and may be required to limit commercialization of our current or future product candidates.
We face an inherent risk of costly and time-consuming product liability lawsuits as a result of the planned clinical testing of our current or future product candidates and will face an even greater risk if we commercialize any products. For example, we may be sued if our current or future product candidates cause or are perceived to cause injury or are found to be otherwise unsuitable during clinical testing, manufacturing, marketing or sale. If we cannot successfully defend ourselves against product liability claims, we may incur substantial liabilities or be required to limit commercialization of our current or future product candidates. Failure to obtain or retain sufficient product liability insurance at an acceptable cost may prevent or inhibit the commercialization of products we may develop. Although we have clinical trial insurance, our insurance policies have various exclusions, and we may be subject to a claim for which we have no coverage. We may have to pay any amounts awarded by a court or negotiated in a settlement that are not covered by or which exceed our insurance coverage, and we may not have sufficient capital to pay such amounts.
Risks Related to Ownership of our Common Stock
We do not know whether an active, liquid and orderly trading market will develop for our Common Stock or what the market price of our Common Stock will be and, as a result, it may be difficult for you to sell your shares of our Common Stock.
Prior to the pricing of our initial public offering on February 4, 2022, there was no public trading market for shares of our Common Stock and after our IPO the trading price of our Common Stock has been volatile. Although our Common Stock is listed on the Nasdaq Capital Market, an active trading market for our shares is still developing and may not be sustained in the future. The lack of an active market for our Common Stock creates volatility in the price of the stock and may impair investors’ ability to sell their shares at the time they wish to sell them or at a price that they consider reasonable and may reduce the fair market value of their shares. Further, an inactive market may impair our ability to raise capital by selling shares of our Common Stock and to enter into strategic partnerships or acquire companies or products using our shares of common stock as consideration.
Our growth is subject to economic and political conditions.
Our business is affected by global and local economic and political conditions as well as the state of the financial markets, inflation, recession, financial liquidity, currency volatility, growth, and policy initiatives. There can be no assurance that global economic conditions and financial markets will not worsen and that we will not experience any adverse effects that may be material to our consolidated cash flows, results of operations, financial position or our ability to access capital, such as the adverse effects resulting from a prolonged shutdown in government operations both in the United States and internationally. Political changes, including war or other conflicts, some of which may be disruptive, could interfere with our supply chain, our customers and all of our activities in a particular location.
We do not intend to pay dividends on our Common Stock in the foreseeable future, so any returns will be limited to the value of our stock, which may be volatile.
We plan to retain future earnings for the development, operation, and expansion of our business and do not anticipate declaring or paying any cash dividends for the foreseeable future. Any return to stockholders will be limited to the appreciation of their stock, which may never occur. Further, the trading price of our common stock is likely to be highly volatile and may be subject to wide fluctuations in response to various factors, some of which are beyond our control. Broad market and industry factors may negatively affect the market price of our common stock, regardless of our actual operating performance.
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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. We may never obtain research coverage by industry or financial analysts. If no or few analysts publish research reports on the Company or if analysts publish negative research reports about the Company, our stock price may significantly decline.
Raising additional capital may cause dilution to our existing stockholders, restrict our operations, or require us to relinquish rights to our current or future technologies or product candidates.
We may seek additional capital through a combination of public and private equity offerings, ATM facility, debt financings, strategic partnerships and alliances and licensing arrangements. Any equity or equity-related financing may dilute our stockholders may subject us to restrictive covenants and interest costs. If we obtain funding through a strategic collaboration or licensing arrangement, we may be required to relinquish our rights to our current product candidates or any future product candidates that we may develop.
Additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our operations. If we are unable to raise additional capital as needed or on acceptable terms, we may be required to delay or discontinue any research, development or commercialization programs and may be unable to expand our operations or otherwise capitalize on our business opportunities. Further, we may be required to seek collaborators for potential product candidates earlier, or on less favorable terms, than might otherwise be desired, or to relinquish or license our rights to potential product candidates in markets where we otherwise would seek to pursue development or commercialization. Any of the above events could significantly harm our business, prospects, financial condition and results of operations and cause the price of our common stock to decline.
Our principal stockholders and management own a significant percentage of our stock and will be able to exert significant influence over matters subject to stockholder approval.
As of November 1, 2024, our executive officers, directors, and 5% stockholders beneficially owned approximately 56.9% of our voting stock and anticipate that the same group will hold a significant portion of our outstanding voting stock for the foreseeable future. These stockholders will have the ability to influence us through their ownership position. This may prevent or discourage unsolicited acquisition proposals or offers for our common stock.
Our failure to meet the continuing listing requirements of the NASDAQ Capital Market could result in a de-listing of our securities.
If we fail to satisfy the continuing listing requirements of NASDAQ, such as the corporate governance, stockholders’ equity or minimum closing bid price requirements, NASDAQ may take steps to delist our common stock. Such a delisting would likely have a negative effect on the price of our common stock and would impair our stockholders’ ability to sell or purchase our common stock. In the event of a delisting, we would likely take actions to restore our compliance with NASDAQ’s listing requirements, but we can provide no assurance that any such action taken by us would allow our common stock to become listed again, stabilize the market price or improve the liquidity of our securities, prevent our common stock from dropping below the NASDAQ minimum bid price requirement or prevent future non-compliance with NASDAQ’s listing requirements.
We are an emerging growth company and a smaller reporting company, and we cannot be certain if the reduced reporting requirements applicable to emerging growth companies and smaller reporting companies will make our common stock less attractive to investors.
We are an emerging growth company, as defined in the JOBS Act. For as long as we continue to be an emerging growth company, we may take advantage of exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies, including: exemption from the auditor attestation requirements of Section 404 of SOX, as amended; being permitted to provide only two years of our audited financial statements and correspondingly reduced “Management’s Discussion and Analysis of Financial Condition and Results of Operations”; exemption from any Public Company Accounting Oversight Board requirement regarding audit firm rotation or an auditor report supplement providing additional information about the audit and financial
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statements; reduced disclosure obligations regarding executive compensation; and exemption from the nonbinding advisory votes on executive compensation and stockholder approval of any golden parachute payments not previously approved.
We have elected to take advantage of certain of the reduced reporting obligations. We cannot predict whether investors will find our common stock less attractive if we rely on these exemptions. If some investors find our common stock less attractive as a result, there may be a less active trading market for our common stock and our stock price may be reduced or more volatile.
Provisions in our certificate of incorporation, our bylaws, and Delaware law may discourage, delay, or prevent a change in control of our Company or changes in our management and, as a result, depress the trading price of our stock.
Provisions of our certificate of incorporation, our bylaws and Delaware law may deter unsolicited takeovers and/or delay or prevent a change in control of our Company, including transactions in which our stockholders might otherwise receive a premium for their shares.
In addition, the Delaware General Corporation Law prohibits a publicly held Delaware corporation from engaging in a business combination with an interested stockholder, defined as a person who owns, or within the last three years has owned, 15% of our voting stock, for a period of three years after the date of the transaction in which the person became an interested stockholder, unless the business combination is approved in a prescribed manner.
The foregoing provisions and anti-takeover measures may limit the price that investors might be willing to pay in the future for shares of our Common Stock and may deter potential acquirers of our Company.
Item 2.Unregistered Sales of Equity Securities and Use of Proceeds.
During the period covered by this report, we have not issued any unregistered securities.
Use of Proceeds from Sales of Registered Securities
On February 4, 2022, our registration statement on Form S-1 (File No. 333-260099) and our registration statement on Form S-1MEF (File No. 333-262512) (collectively, the “Registration Statements”) were declared effective by the SEC. Pursuant to such Registration Statements, we sold an aggregate of 3,200,000 shares of our common stock at a price of $5.00 per share for aggregate net cash proceeds of approximately $13.1 million, which amount is net of $1.12 million in underwriter’s discounts, and commissions, and $1.8 million of other expenses incurred in connection with the offering. We closed the offering on February 8, 2022.
On August 24, 2022, our registration statement on Form S-1 (File No. 333-266857), which was filed on August 15, 2022, was declared effective by the SEC. Pursuant to such registration statement, we sold an aggregate of 1,015,598 shares of our common stock at a price of $8.25, and 909,091 of pre-funded warrants to purchase one share of common stock at $8.25 per share for aggregate net cash proceeds of approximately $14.3 million, which amount is net of $1.4 million in placement agent fees, and $0.3 million of other expenses incurred in connection with the offering. We closed the private placement on July 29, 2022. In this offering, we also issued to the investors who participated in the offering preferred investment options to purchase up to an aggregate of 1,924,689 shares of common stock, at an exercise price of $9.65 per share with a term of three and one-half years from the date of issuance.
We intend to use the net proceeds of these offerings to fund the Phase 1 development of NXP800 and NXP900, to continue development and sponsored research related to our current product candidates or any future product candidate, hiring of additional personnel, capital expenditures, costs of operating as a public company and other general corporate purposes.
There has been no material change in the expected use of the net proceeds from our initial public offering as described in our final prospectuses filed with the SEC on February 8, 2022 and August 15, 2022, respectively, pursuant to Rule 424(b) under the Securities Act. We invested the funds received in an interest-bearing money market account.
Item 3.Defaults Upon Senior Securities.
None.
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Item 6. Exhibits
Exhibit No. |
| Description |
31.1* | ||
31.2* | ||
32.1** | ||
32.2** | ||
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 (XBRL): (i) the Condensed Balance Sheets, (ii) the Condensed Statements of Operations, (iii) the Condensed Statement of Stockholders’ Equity, (iv) the Condensed Statements of Cash Flows, and (v) Notes to the Condensed Financial Statements. | |
104* | Cover Page Interactive Data File (formatted as Inline XBRL and contained in Exhibit 101). |
* Filed herewith.
** Furnished herewith.
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Signatures
Pursuant to the requirements of the Securities Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized, in the City of Fort Lee, State of New Jersey, on this 5th day of November 2024.
| Nuvectis Pharma, Inc. | ||
|
| ||
| By: | /s/ Ron Bentsur | |
|
| Name: | Ron Bentsur |
|
| Title: | Chairman, Chief Executive Officer and President |
By: | /s/ Michael J Carson | ||
| Name: | Michael J Carson | |
| Title: | Vice President of Finance (Principal Financial and Accounting Officer) | |
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