美國證券交易委員會
華盛頓特區20549
表格
[mark one]
根據1934年證券交易法第13或15(d)條，本季度報告 |
截止季度結束日期：
根據1934年證券交易法第13或15（d)條的轉型報告 |
過渡期從______________到______________
委員會文件號
(公司註冊名完全按照其章程規定)
(註冊地或其他司法管轄區 | (稅務局僱主 |
組建或組織) | 標識號碼) |
(主要行政辦公室地址，包括郵政編碼)
(
(公司電話號碼，包括區號)
(先前名稱、先前地址和先前財政年度，如自上次報告以來發生變更)
根據證券法第12(b)條註冊的證券：
每一類的名稱 |
| 交易標的 |
| 註冊交易所的名稱 |
請在以下方框內打勾：(1) 在過去的12個月內(或者在註冊公司需要提交此類報告的較短時期內)，公司已經提交了根據證券交易法1934年第13或15(d)條規定需要提交的所有報告；以及 (2) 在過去的90天內，公司一直受到了此類報告提交的要求。
請確認是否在過去12個月內（或該註冊人需要提交此類文件的更短期間內）已經以電子方式提交了每個交互式數據文件，這些文件需要根據規則405（S-T條例§232.405）進行提交。
在交易所法案第120億.2條中，勾選表示報告人爲大型加速文件提交人、加速文件提交人、非加速文件提交人、小型報告公司或新成長公司。請參閱「大型加速文件提交者」、「加速文件提交者」、「小型報告公司」和「新興成長公司」的定義。
大型加速報告人 | ☐ | 加速文件提交人 | ☐ | ☒ | 較小的報告公司 | 新興成長公司 |
如果是一家新興成長型公司，請在覈對標記內指示公司是否已選擇不使用從根據證券交易法第13(a)條提供的依據進行遵守任何新的或修訂的財務會計準則的擴展過渡期。 ☐
請在以下方框內打勾：公司是否是空殼公司(根據證券交易法第12b-2條規定定義）。是
截至最近可行日期，發行人普通股的流通股數爲：
前瞻性聲明
Cellectar Biosciences公司的Form 10-Q季度報告中包含根據1934年修正的證券交易法第21E條的前瞻性聲明，我們將其稱爲交易所法案。我們的前瞻性陳述示例包括：
| ● | 我們對業務策略、業務計劃和研發活動的當前看法； |
| ● | 我們產品開發項目的進展，包括臨床測試以及啓動和結果的時間安排； |
| ● | 我們的預計操作結果，包括研究和開發費用； |
| ● | 我們有能力繼續開發 iopofosine I 131（iopofosine，也稱爲CLR 131），CLR 1900 系列，CLR 2000 系列和CLR 12120 的計劃； |
| ● | 我們有能力繼續開發我們的磷脂酸藥物結合物（PDC）™ 的計劃； |
| ● | 我們有能力在美國繼續維持對孤兒藥物的指定 iopofosine 作爲治療多發性骨髓瘤、神經母細胞瘤、骨肉瘤、橫紋肌肉瘤、尤因氏肉瘤和淋巴漿細胞淋巴瘤的治療藥物，以及孤兒藥品地位的預期好處； |
| ● | 我方唯一供應商發生任何中斷情況 iopofosine; |
| ● | 我們通過出售股權和/或債務證券、戰略交易或其他方式獲得額外資金的能力； |
| ● | 我們推進技術成爲產品候選人的能力； |
| ● | 我們當前資源的增強和消費，以及獲得額外資金的能力； |
| ● | 我們對於一般經濟和市場狀況，包括我們的競爭優勢的當前看法； |
| ● | 由於衝突、軍事行動、恐怖襲擊、自然災害、公共衛生危機(包括傳染病或疾病如COVID-19大流行的發生)、網絡攻擊和一般不穩定性所導致的不確定性和經濟不穩定性； |
| ● | 美國立法和監管發展對我們產品候選品定價和報銷的未來影響； |
| ● | 我們達到納斯達克的持續上市標準的能力； |
| ● | 任何上述內容的假設；以及 |
| ● | 任何其他涉及我們打算或相信將來會發生的事件或發展的聲明。 |
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在某些情況下，您可以通過術語識別前瞻性聲明，例如「預計」，「預計」，「打算」，「估計」，「計劃」，「相信」，「尋求」，「可能」，「應該」，「可以」，「願意」或類似表達。因此，這些聲明涉及估計，假設和不確定性，可能導致實際結果與其中表達的結果有實質差異。前瞻性聲明還涉及風險和不確定性，其中許多超出我們的控制範圍。任何前瞻性聲明都應完全參考本季度報告中討論的因素。
您應該完整閱讀本報告，並理解我們實際未來的結果可能與我們的預期有實質不同。您應該假定出現在本報告中的信息僅截至本日期爲準確。因爲本報中提到的風險因素可能導致實際結果或結果與我們或代表我們提出的任何前瞻性聲明有實質不同，您不應過度依賴任何前瞻性聲明。此外，任何前瞻性聲明僅表明其發表之日，我們沒有義務更新任何前瞻性聲明以反映發表聲明之日之後的事件或情況，或反映不可預測事件的發生。新的因素不時出現，我們無法預測哪些因素會出現。此外，我們無法評估每個因素對我們業務的影響，或任何因素，或多個因素的影響，可能導致實際結果與任何前瞻性聲明中包含的結果有實質不同。
這份Form 10-Q季度報告中包含Cellectar Biosciences, Inc.的商標和服務標記。除非在本10-Q季度報告中另有規定，以™標識的商標屬於Cellectar Biosciences, Inc.。所有其他商標均爲其各自所有者的財產。
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第一部分 財務信息
項目1：基本報表
CELLECTAR BIOSCIENCES, INC.
簡明合併資產負債表
(未經審計)
6月30日， | 運營租賃負債： | |||||
| 2024 |
| 2023 | |||
資產 |
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流動資產： |
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現金及現金等價物 | $ | |
| $ | | |
預付費用和其他流動資產 |
| |
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總流動資產 |
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固定資產、廠房和設備，淨額 |
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經營租賃資產使用權 |
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其他長期資產 |
| | | |||
資產總計 | $ | |
| $ | | |
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負債和股東權益（赤字） |
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流動負債： |
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應付賬款及應計費用 | $ | |
| $ | | |
認股權負債 | |
| | |||
租賃負債，流動 |
| |
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流動負債合計 |
| |
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長期租賃負債，扣除應付本金 |
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負債合計 | | | ||||
承諾和 contingencies（見注 7） |
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中間資本: |
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D輪優先股， |
| | | |||
股東權益（赤字）： | ||||||
E-2系列優先股， |
| |
|
| | |
E-3系列優先股 |
| |
| — | ||
普通股，每股面值爲 $0.0001； |
| |
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額外實收資本 |
| |
|
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累積赤字 |
| ( |
| ( | ||
3660 | | ( | ||||
負債合計及股東權益（虧損） | $ | | $ | | ||
隨附說明是這些簡明合併財務報表的一部分。
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CELLECTAR BIOSCIENCES, INC.
簡明合併利潤表
(未經審計)
截至6月30日的三個月： | 截至6月30日的六個月： | |||||||||||
| 2024 |
| 2023 |
| 2024 |
| 2023 | |||||
營業費用: |
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研發 | $ | | $ | | $ | | $ | | ||||
ZSCALER, INC. |
| |
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營業費用總計 |
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營業虧損 |
| ( |
| ( |
| ( |
| ( | ||||
其他收益（費用）： |
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認股權證估值收益（損失） | | ( | ( | ( | ||||||||
利息收入 |
| |
| |
| |
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其他收入（支出）總額 |
| |
| ( |
| ( |
| ( | ||||
淨損失 | $ | ( | $ | ( | $ | ( | $ | ( | ||||
每股虧損 - 基本 | $ | ( | $ | ( | $ | ( | $ | ( | ||||
每股稀釋淨損失 | $ | ( | $ | ( | $ | ( | $ | ( | ||||
基本加權平均普通股流通量 | | | | | ||||||||
稀釋後加權平均每股優先股實際已發行數量 |
| |
| |
| |
| | ||||
隨附說明是這些簡明合併財務報表的一部分。
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CELLECTAR BIOSCIENCES, INC.
可轉換優先股和股東（赤字）權益綜合報表
(未經審計)
D系列優先股 | 總費用 | ||||||||||||||||||||||||
股票 | 優先股 | 普通股 | 額外的 | 累積的 | 股東的 | ||||||||||||||||||||
| 股份 |
| 數量 |
|
| 股份 |
| 金額 |
| 股份 |
| 面值 |
| 實收資本 |
| $ |
| （虧損）股權 | |||||||
2022年12月31日結存餘額 |
| | $ | | — | $ | — | | $ | | $ | | $ | ( | $ | | |||||||||
將預先融資權證轉換爲普通股 |
| — | — | — | — | | | — | — | | |||||||||||||||
以股票爲基礎的報酬計劃 | — | — | — | — | — | — | | — | | ||||||||||||||||
淨虧損 | — | — | — | — | — | — | — | ( | ( | ||||||||||||||||
2023年3月31日的餘額 | | | — | — | | | | ( | | ||||||||||||||||
以股票爲基礎的報酬計劃 |
| — | — | — | — | — | — | | — | | |||||||||||||||
淨虧損 | — | — | — | — | — | — | — | ( | ( | ||||||||||||||||
2023年6月30日的餘額 |
| | $ | | — | $ | — | | $ | | $ | | $ | ( | $ | ( | |||||||||
| |||||||||||||||||||||||||
2023年12月31日結餘爲 | | $ | | | $ | | | $ | | $ | | $ | ( | $ | ( | ||||||||||
以股票爲基礎的報酬計劃 | — | — | — | — | — | — | | — | | ||||||||||||||||
將預付權證轉換爲普通股 | — | — | — | — | | | | — | | ||||||||||||||||
行使優先股認股權，扣除發行成本（注2） | — | — | | | — | — | — | — | | ||||||||||||||||
將E-3系列優先股轉換爲普通股 | — | — | ( | ( | | | | — | — | ||||||||||||||||
行使認股權證以換取普通股 | — | — | — | — | | | | — | | ||||||||||||||||
將E-2系列優先股轉換爲普通股 |
| — | — | ( | ( | | | | — | — | |||||||||||||||
已退市股票 | — | — | — | — | ( | — | — | — | — | ||||||||||||||||
淨虧損 | — | — | — | — | — | — | — | ( | ( | ||||||||||||||||
2024年3月31日結存餘額 | | | | | | | | ( | | ||||||||||||||||
以股票爲基礎的報酬計劃 | — | — | — | — | — | — | | — | | ||||||||||||||||
將E-3系列優先股轉換爲普通股 | — | — | ( | ( | | | | — | — | ||||||||||||||||
淨虧損 |
| — | — | — | — | — | — | — | ( | ( | |||||||||||||||
2024年6月30日餘額 |
| | $ | | | $ | | | $ | | $ | | $ | ( | $ | | |||||||||
隨附說明是這些簡明合併財務報表的一部分。
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CELLECTAR 生物科學有限公司
簡明的合併現金流量表
（未經審計）
六個月已結束 | ||||||
6月30日 | ||||||
|
| 2024 |
| 2023 | ||
來自經營活動的現金流： |
|
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|
| ||
淨虧損 | $ | ( |
| $ | ( | |
爲將淨虧損與經營活動中使用的現金進行覈對而進行的調整： |
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折舊和攤銷 |
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股票薪酬支出 |
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經營租賃使用權資產的變化 |
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認股權證公允價值的變化 |
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以下方面的變化： | ||||||
預付費用和其他流動資產 | ( | | ||||
租賃責任 |
| ( |
| ( | ||
應付賬款和應計負債 |
| ( |
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用於經營活動的現金 |
| ( |
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| ( | |
來自投資活動的現金流： |
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購買不動產、廠房和設備 |
| ( | — | |||
用於投資活動的現金 |
| ( |
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| — | |
來自融資活動的現金流量: |
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行使認股權證的收益，扣除發行成本 |
| |
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融資活動提供的現金 | | | ||||
現金和現金等價物的淨增長 | | ( | ||||
期初的現金和現金等價物 |
| |
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期末的現金和現金等價物 | $ | | $ | | ||
現金流信息的補充披露 | ||||||
認股權證與股權的結算 | $ | | $ | — | ||
將優先股轉換爲普通股 | $ | | $ | — | ||
所附附附註是這些簡明合併財務報表不可分割的一部分。
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CELLECTAR 生物科學有限公司
簡明合併財務報表附註（未經審計）
1。業務和組織的性質
Cellectar Biosciences, Inc.（以下簡稱 「公司」）是一家處於後期階段的臨床生物製藥公司，專注於癌症治療藥物的發現、開發和商業化，利用公司專有的磷脂藥物偶聯物™（PDC™）交付平台，該平台專門靶向癌細胞，通過減少脫靶效應來提高療效和安全性。
繼續關注 — 作爲一家新興成長型公司，該公司自成立以來一直遭受巨額經常性虧損，並在運營中使用淨現金，因爲該公司將幾乎所有的精力都用於研究、開發和尋求批准其候選產品在市場上商業化。由於這些努力，該公司的累計赤字約爲 $
爲了爲其研究、開發和審批工作提供資金，公司自成立以來一直嚴重依賴私人投資者和公衆股東通過發行證券提供的資金，例如普通股、可轉換優先股和認股權證（外部資本）。該公司預計，在可預見的將來，該公司將繼續嚴重依賴外部資本爲公司的運營提供資金，直到其一種或多種候選產品獲得批准並在市場上成功商業化。儘管管理層認爲將根據需要獲得額外的外部資本，但無法保證額外的外部資本會得到擔保，也無法保證以公司可接受的條件進行擔保。
截至隨附的合併財務報表發佈之日（「發行日期」），公司在發行之後的未來十二個月中爲公司運營提供資金的可用流動性僅限於約美元
這些不確定性使人們對公司繼續經營的能力產生了極大的懷疑。隨附的財務報表是在公司將繼續作爲持續經營企業運營的基礎上編制的，這意味着在可預見的將來，它將能夠在正常業務過程中變現資產並結清負債和承諾。因此，隨附的合併財務報表不包括可能因這些不確定性結果而產生的任何調整。
簡明合併財務報表由Cellectar Biosciences, Inc.根據美利堅合衆國普遍接受的中期財務信息會計原則（美國公認會計原則）以及表格10-Q和第S-X條例第10條的說明編制。因此，它們不包括美國公認會計原則要求的完整財務報表的所有信息和腳註。管理層認爲，本文件中的披露足以滿足中期報告要求。
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截至2023年12月31日的附註簡明合併資產負債表來源於公司經審計的基本報表。截至2024年6月30日的附註簡明合併資產負債表，以及截至2024年6月30日和2023年的前六個月的簡明合併損益表、現金流量表和股東權益合併報表，以及基本報表附註中包含的相關中期信息，均按照美國一般公認會計原則（美國U.S. GAAP）和證券交易委員會（SEC）有關中期財務信息的指導、規則和法規編制。因此，它們不包括美國U.S. GAAP要求的所有細節和附註，以形成完整的基本報表。在管理層看來，未經審計的中期簡明合併財務報表反映了截至2024年6月30日公司的合併財務狀況以及截至2024年和2023年的前六個月公司的合併經營業績、現金流量和股東權益的公平呈現所需的本質性調整。截至2024年6月30日的六個月業績並不一定代表未來業績。
這些未經審計的簡明合併財務報表應與公司於2024年10月29日提交給SEC的年度財務報表附註一起閱讀，該報表包括截至2023年12月31日的財年10-K/A表。
使用估計根據通用會計準則編制附帶的合併基本報表，需要管理層對資產和負債的報告金額以及在合併基本報表日期當日的或有資產和負債的披露以及報告期間費用金額做出一定的估計和假設。重要的估計包括用於潛在負債的計提假設，權證負債的估值，債務和權益工具的估值，用於服務發行的股票期權的估值以及遞延稅資產準備金的估值。實際結果可能與這些估計值有所不同。
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固定資產 —— 固定資產以成本計量。折舊額按照資產的預計可用年限採用直線法提供，
使用權（ROU） 資產和租賃負債 公司按照FASB會計準則 codification（ASC）第842號主題《租賃》的規定，計提所有重大租約。使用權資產按照其估計的有用壽命攤銷，其表示租約的全期。詳見註釋8。
以股票爲基礎的補償公司使用Black-Scholes期權定價模型計算授予日期的股票期權獎勵的公允價值。由此產生的補償費用，扣除非基於績效的獎勵的棄權，按照獎勵的服務期間以直線方式確認，截至2024年和2023年6月30日結束的三個月和六個月批准日期範圍內。
迄今爲止，我們的研究和開發費用與AV-101的開發有關。研究和開發費用按照發生的原則確認，並將在收到將用於研究和開發的貨物或服務之前支付的款項資本化，直至收到這些貨物或服務。研發成本會在發生的時候支出。公司確認當有可能公司會遵守補助安排附帶的條件並且補助款項會收到時，公司會確認收入和成本報銷。政府補助款項會按照公司確認與政府補助款項有關的成本的期間，系統性地確認。具體來說，當政府補助款項與收入成本或營業費用的報銷相關時，政府補助會在綜合損益表中確認爲相關費用的減少。公司會在資產負債表中的預付費用和其他流動資產中記錄應收政府補助款項。
率所得稅會使用負債會計方法來覈算。根據這種方法，延緩稅資產和負債是根據資產和負債的財務報表基礎和稅基礎之間的暫時差異以及淨營運虧損和信貸轉嫁使用實施的稅率來確定的，這種差異預計會在稅率會恢復的年份生效。對於稅率變更對延緩稅資產和負債的影響會在包括批准日期的那個期間在收入中確認。一旦有更加可能不實現公司毛延緩稅資產的一部分，就會設立估值準備金。在準備納稅申報表的過程中所採取或預計會採取的稅收立場需要評估，以確定這些稅收立場是否更可能會被適用納稅權威維持。未能達到更可能會達到的門檻的稅收立場會在當年記錄爲稅收支出。截至2024年6月30日和2023年12月31日，沒有需要計入或在財務報表中披露的不確定的稅收立場。
金融工具的公允價值ASC主題825指南要求披露某些金融工具的公允價值。附表中的金融工具包括貨幣資金、預付費用和其他資產、應付賬款及應計負債、長期負債等。由於其短期性質，貨幣資金、預付費用、其他流動資產和應付賬款的賬面價值近似於其公允價值（見附註2和3）。
權證 公司根據ASC480《區分負債與權益》和ASC815《衍生工具和套期交易》中的具體條款和適用的權威指引，將權證分類爲權益類或負債類工具。評估考慮了權證是否根據ASC480獨立存在的金融工具，是否符合ASC480對負債的定義，以及權證是否符合ASC815對權益分類的所有要求，包括權證是否與公司自身的普通股掛鉤，以及權證持有人是否可能在公司無控制權的基本交易中要求淨現金結算等權益分類條件。這一評估需要專業判斷，分別在發行權證時和權證有效期內的每個後續季度結束日期進行（見附註2）。如果權證屬於負債分類，估值變動以及發行權證的成本將計入基本報表中的其他收入（費用）（見附註3）。如果初始將這些工具分類爲負債或者權益，後續評估確定分類已更改，公司將在基本報表中反映這一變化。
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優先股 公司根據ASC 480和ASC 815的權威指導，基於特定條款對優先股進行覈算，包括它們是否是獨立工具，是否存在任何贖回或轉換方面，以及它們如何要求結算（特別是是否存在現金結算方面），以及它們是否具有主要類似債務或類似股權的特徵，它們是否具有嵌入式衍生工具，以及是否具有贖回特徵。根據這些標準的分析，優先股將被分類爲債務、臨時（或「中層」）股權或永久股權。然後每季度評估結果分類，判斷是否需要更改分類。
信貸風險集中—— 使公司面臨信用風險的金融工具主要包括存放在金融機構的現金和現金等價物。截至2024年6月30日和2023年12月31日，公司的多餘現金存放在與知名金融機構的利息收入帳戶中。有時，這些金額可能超過FDIC保險限額。截至2024年6月30日和2023年12月31日，未投保的現金餘額約爲$
政府援助 根據政府援助計劃，符合條件的支出報銷會被記錄爲營業成本減少，前提是公司有合理保證會遵守撥款安排附加條件，並且已經提出了報銷。確定報銷金額，以及因此而產生的應收金額，需要管理層根據其對符合計劃條款的支出的解釋進行計算。公司提交的報銷申請將由相關政府機構進行審核。 公司目前通過新華保險獲得了一項癌症治療研究獎，總額約爲1000萬美元，爲期約
2024年6月30日結束的六個月內，公司獲得了約
最近頒佈但尚未頒佈的會計準則— 2023年12月，FASB發佈了《會計準則更新》（ASU）2023-09，所得稅（第740號課題）—改進所得稅披露，旨在增強所得稅披露的透明度和決策效用。公衆企業必須在2024年12月15日後開始的年度財政期間採納此標準，允許提前採納。公司正在評估採納此指導對其合併財務報表和相關披露的影響。
2023年11月，FASB發佈了ASU 2023 - 07，分部報告（課題280）。此更新的修訂擴展了分部披露要求，包括針對具有單個可報告分部的實體的新分部披露要求，以及其他披露要求。此更新對於2023年12月15日後開始的財政年度和2024年12月15日後開始的財政年度內的中期時段有效。公司目前正在評估採納ASU 2023 - 07將對其簡明合併財務報表產生的影響。
公司評估了FASB發佈的所有ASU，以考慮其對財務報表的適用性。公司評估了所有已發佈但尚未採納的ASU，並得出結論，未披露的不相關於公司或預計不會對公司產生實質影響。
修正前發出的合併財務報表 — 在2024年第三季度，並在提交本10-Q表格之前，公司確定有必要重新評估公司以往發行的某些認股權證和優先股的會計處理方式。此外，公司確定部分先前列爲研發費用的運營成本更適合歸類爲一般和管理費用。根據《會計理論公告》第99號（SAb No. 99）第1.m，「重要性」和SAb No. 99 第1.N 「在計量當年財務報表中的錯報後考慮錯報的影響」條款，公司評估了這些錯誤對其先前發行的合併財務報表的重要性。基於公司對定量和定性因素的評估，公司得出結論，這些錯誤對公司截至2023年12月31日和2022年的合併財務報表，以及2024年第一季度的財務報表具有重要性。因此，本10-Q表格呈現了公司截至2023年12月31日已反映在公司10-K/A表中的6月30日爲止的重新說明的公司簡明合併財務報表。
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2. 股東權益
2023年9月定向增發
2023年9月8日，在私募定向增發中，公司向某些機構投資者發行了
優先股的轉換價格如下：對於E-1或E-2系列優先股，每股美元
| ● | A檔認股權證，總行使價格爲 $ |
| ● | B檔認股權證，總行使價格爲 $ |
截至2023年12月31日，Tranche A和Tranche b認股權證不符合衍生品的資格；但是，它們不符合公司股票可被編入指數的要求。因此，根據FASB ASC 815的指導，認股權證被視爲負債。截至2024年6月30日，Tranche A認股權證結算後，Tranche b認股權證不再符合衍生品，符合公司股票可被編入指數的要求。然而，由於現金結算功能需要在公司無法控制的基本交易事件中進行現金結算，導致與其他證券持有人獲得的結算方式不一致，認股權證不符合權益分類指導。因此，根據ASC 815的指導，Tranche b認股權證繼續被視爲負債。所有此類負債必須按公允價值列示，變動應反映在財務期間的財務結果中。有關估值請參閱附註3。
發行時，Series E-1優先股具有贖回特性；因此，截至2023年9月30日，它被歸類爲中間資本。Series E-1優先股還設有清算特權，計算方法爲每股金額等於（i）原始每股價格的兩倍（2倍），加上任何已宣佈但未支付的股利，或（ii）如果所有Series E-1優先股立即在清算前轉換爲普通股，則應支付的每股金額。在Series E-1優先股未償還時，由於清算特權影響了這些認股權證在清算事件中的處理，導致Tranche A和Tranche b認股權證被視爲可處置的。根據ASC 480的指導，可處置認股權證被視爲負債。這些特徵僅適用於Series E-1優先股未償還時；在公司於2023年10月25日舉行的股東特別會議上獲得股東批准該交易後，Series E-1優先股立即轉換爲Series E-2優先股和/或普通股，具體取決於持有人的受益所有權位置。
2023年9月定向增發的淨收益首先分配給Tranche A和Tranche b認股權證的公平價值，其發行時的公允價值爲$
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E-2系列優先股可在持有人的請求下轉換爲普通股，但轉換需遵守融資協議規定的持有人不得超過一定的受益所有權比例。E-2系列優先股發行後，2023年12月31日前，優先股持有人進行了轉換。
在2024年6月30日結束的六個月內，沒有發行股份。
2024年1月，公司發佈了其關鍵性、第20億項CLOVER Wam試驗的前期數據。根據第A筆認股權證的條款，認股權的到期日提前至 在前期數據發佈後的交易日後，認股權持有人全部行使了A筆認股權，導致公司發行
2024年6月30日結束的六個月內，持有
2022年10月公開發行和定向增發
於2022年10月25日，公司完成了每股
在2024年6月30日結束的六個月內，沒有發行股份。
權證
以下表格總結了截至2024年6月30日持有的購買股票的未行使認股權證信息：
股份數量 | ||||||||
可發行的 |
| |||||||
行使 |
| |||||||
未償還金額 | 行權 |
| ||||||
增發計劃 |
| 權證 |
| 價格 |
| 到期日期 | ||
2023 年份 B 類優先認股權證 | | $ | | |||||
2022年普通認股權證 | | $ | ||||||
2020年6月H系列普通認股權證 | | $ | ||||||
2017年10月份D系列普通認股權證 | | $ |
| |||||
總費用 |
| |
|
|
|
| ||
以上表格中的所有認股權證均被分類爲負債。
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3. 公允價值
根據ASC 820中對公允價值衡量和披露主題的規定，公司將其一般以公允價值衡量的金融資產和金融負債分爲三個級別，根據資產和負債交易的市場以及確定公允價值所使用的假設的可靠性:
| ● | 第1級：在活躍市場中報價的相同金融資產或負債的輸入價格。 |
| ● | 第2級：除第1級報價之外的輸入，例如在活躍市場中報價的類似金融資產和負債的價位，以及在不活躍市場中的相同資產和負債的價格，或者可由可觀察的市場數據證實或經可觀察的市場數據證實的其他輸入。 |
| ● | 三級：輸入報價對於金融資產或負債的公允價值具有重要意義，但並非通過活躍市場觀察或支持。 |
在估值基於市場上不太可觀察或難以觀測的模型或輸入時，公允價值的判斷需要更多判斷。因此，公司在確定公允價值時所行使的判斷程度最大的是分類爲三級的工具。金融工具在公允價值層次結構中的級別是基於對公允價值測量具有重要意義的任何輸入的最低級別。現金及現金等價物的賬面價值接近公允價值，因爲到期期限不到三個月。其他流動財務資產和負債的報告賬面價值接近公允價值，因爲其爲短期性質。截至2024年6月30日，公司沒有任何一級或二級負債。
作爲2023年9月融資的一部分（請參見附註2），公司發行了A檔和B檔權證（2023權證）來購買優先股的股票，根據轉換政策，代表了
2023年認股權證被分類到第3級層次中，因爲這些輸入的性質和使用的估值技術，並且在2024年6月30日和2023年12月31日分別具有50美元的公允價值，這些數值包含在相關資產負債表上的認股權責任標題中。
以下表總結了2024年6月30日和2023年12月31日使用的修改期權定價假設。
| 6月30日, |
| 12月31日 | ||
2024 | 2023 | ||||
波動性 | % | % | |||
無風險利率 |
| % | % | ||
預計壽命（年） |
| ||||
分紅 |
| % | % |
在行使A檔認股權證時，根據E-3系列優先股的普通股換股比率與認股權證行使時的股票交易價格之間的差額計算，其公允價值被判斷爲$
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根據ASC 815的規定，根據協議中的現金結算功能，第二特權證繼續被列爲負債。重新分類爲股本。
2022年10月，公司共發行了
2022年普通認股權證的公平價值是通過使用Black-Scholes期權定價模型確定的。影響2022年普通認股權證公平價值測量的定量要素包括基礎普通股每股價值、無風險利率、預期分紅派息和公司股票的預期波動率。無風險利率是根據與認股權證剩餘合約期限大致相等的時間段的美國國債收益率曲線確定的。公司根據預期的分紅派息和公司從未支付或宣佈現金分紅的事實，估算了一定的分紅率。根據公司普通股的歷史波動率確定了預期波動率。由於這些輸入的性質和使用的估值技術，這些認股權證屬於第3級層次結構。下表總結了每個財務報告日期使用的假設。
| 6月30日， |
| 運營租賃負債： |
| |
2024 | 2023 | ||||
波動性 | % | % | |||
無風險利率 |
| | % | | % |
預計壽命（年） |
| |
| | |
分紅 |
| % | % |
以下表格總結了屬於第3級公允價值層級內的 warrants 的公允市場價值變化，包括2023年9月份的 Warrants 和 2022年10月份的 Warrants:
| 三級 | ||
2023年12月31日Level 3負債的公允價值 | $ | | |
期權公允價值變動 | | ||
將2023年Tranche A 期權結算爲股本 | ( | ||
行使2022年10月份的期權 |
| ( | |
2024年6月30日，三級負債的公允價值 | $ | | |
4. 基於股票的薪酬
股票激勵計劃會計處理
2021年股票激勵計劃
公司保留了2021年股票激勵計劃（「2021計劃」）。公司將股票作爲僱員和非僱員董事和高管薪酬政策的組成部分之一。董事會的一個委員會確定所授予獎勵的條款，並可能授予各種形式的基於股權的激勵薪酬。目前，這些激勵主要包括期權和受限制股份。根據2015年股票激勵計劃（「2015計劃」）的條款，所有未使用的獎勵仍然有效。根據2015計劃的條款，當前適用於2015計劃的股份以及用於授予2021計劃股份的任何股份，如果在2015計劃下被沒收、取消、公司再收購或以其他方式終止，則將納入可供授予的股份中。
根據當前的股票期權授予計劃，所有期權在發行後一到三年後可行使，並在十年後到期。每份股票期權獎勵的公允價值是根據授予日期使用Black-Scholes期權定價模型估計的。波動率基於公司歷史普通股波動率。無風險利率基於當時實際的美國國債收益曲線。授予的股票期權的預期期限是基於預計未來期權將被行使的估計。沒收在發生時進行記錄。過去未記錄任何分紅派息。
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At the annual meeting of stockholders held on June 14, 2024, the Company’s stockholders approved an increase in the number of shares of common stock available for issuance under the 2021 Stock Incentive Plan by
The following table summarizes amounts charged to expense for stock-based compensation related to employee and director stock option grants:
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Research and development | $ | | $ | | $ | | $ | | ||||
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Total stock-based compensation | $ | | $ | | $ | | $ | | ||||
In December 2023, the Company granted
Assumptions Used in Determining Fair Value
Valuation and amortization method. The fair value of each stock award is estimated on the grant date using the Black-Scholes option-pricing model. The estimated fair value of employee stock options is amortized to expense using the straight-line method over the required service period which is generally the vesting period. The estimated fair value of the non-employee options is amortized to expense over the period during which a non-employee is required to provide services for the award (usually the vesting period).
Volatility. The Company estimates volatility based on the Company’s historical volatility since its common stock is publicly traded.
Risk-free interest rate. The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the time of grant commensurate with the expected term assumption.
Expected term. The expected term of stock options granted is based on an estimate of when options will be exercised in the future. The Company applies the simplified method of estimating the expected term of the options, as described in the SEC’s Staff Accounting Bulletins 107 and 110, as the historical experience is not indicative of the expected behavior in the future. The expected term, calculated under the simplified method, is applied to groups of stock options that have similar contractual terms. Using this method, the expected term is determined using the average of the vesting period and the contractual life of the stock options granted. The Company applied the simplified method to non-employees who have a truncation of term based on termination of service and utilizes the contractual life of the stock options granted for those non-employee grants which do not have a truncation of service.
Forfeitures. The Company records stock-based compensation expense only for those awards that are expected to vest and accounts for forfeitures as they occur.
Dividends. The Company has not historically recorded dividends related to stock options.
Exercise prices for all grants made during the six months ended June 30, 2024 and June 30, 2023, were equal to the market value of the Company’s common stock on the date of grant.
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5. INCOME TAXES
The Company accounts for income taxes in accordance with the liability method of accounting. Deferred tax assets or liabilities are computed based on the difference between the financial statement and income tax basis of assets and liabilities, and net operating loss carryforwards (“NOLs”), using the enacted tax rates. Deferred income tax expense or benefit is based on changes in the asset or liability from period to period. The Company did not record a provision or benefit for federal, state or foreign income taxes for the six months ended June 30, 2024 or 2023 because the Company has experienced losses on a tax basis since inception. Management has provided a full allowance against the value of its gross deferred tax assets in light of the continuing losses and uncertainty associated with the utilization of the NOLs in the future.
The Company also accounts for the uncertainty in income taxes related to the recognition and measurement of a tax position taken or expected to be taken in an income tax return. The Company follows the applicable accounting guidance on derecognition, classification, interest and penalties, accounting in interim periods, disclosure and transition related to the uncertainty in income tax positions. No uncertain tax positions have been identified.
6. NET LOSS PER SHARE
Basic net loss per share is computed by dividing net loss attributable to common stockholders by the weighted average number of shares of common stock and pre-funded warrants outstanding during the period. The pre-funded warrants are considered common shares outstanding for the purposes of the basic net loss per share calculation due to the nominal cash consideration and lack of other contingencies for issuance of the underlying common shares. Diluted net loss attributable to common stockholders per share is computed by dividing net loss attributable to common stockholders, as adjusted, by the sum of the weighted average number of shares of common stock and the dilutive potential common stock equivalents then outstanding. Potential common stock equivalents consist of stock options, warrants, and convertible preferred shares. In accordance with ASC Topic 260, Earnings per Share, diluted earnings per share are the amount of earnings for the period available to each share of common stock outstanding during the reporting period and to each share that would have been outstanding assuming the issuance of common shares for all dilutive potential common shares outstanding during the reporting period. In the quarter ended June 30, 2024, the common warrants issued in October 2022 were dilutive. In all other periods presented, all outstanding warrants were antidilutive.
Three months ended June 30, 2024 |
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Net loss | $ | ( | |
Dilutive effect of warrant liability |
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Net loss allocated to common shares | $ | ( | |
Weighted average common shares outstanding - basic |
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Dilutive effect of warrant liability |
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Weighted average common shares outstanding - diluted |
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Net loss per share - diluted | $ | ( |
The following potentially dilutive securities have been excluded from the computation of diluted net loss per share since their inclusion would be antidilutive:
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7. COMMITMENTS AND CONTINGENCIES
Legal
The Company may be involved in legal matters and disputes in the ordinary course of business. It is not anticipated that the outcome of such matters and disputes will materially affect the Company’s financial statements.
8. LEASES
Operating Lease Liability
In June 2018, the Company executed an agreement for office space in the Borough of Florham Park, Morris County, New Jersey to be used as its headquarters (HQ Lease). The HQ Lease commenced upon completion of certain improvements in October 2018.
On December 30, 2022, the Company entered into an Amended Agreement of Lease of the HQ Lease (Amended HQ Lease), with CAMPUS 100 LLC (the “Landlord”). Under the Amended HQ Lease, which was accounted for as a modification of the initial lease, the Company will continue to lease
Under the terms of the Amended Lease, the Company Company’s previously paid security deposit of $
Discount Rate
The Company has determined an appropriate interest rate to be used in evaluating the present value of the Amended Lease liability considering factors such as the Company’s credit rating, borrowing terms offered by the U.S. Small Business Administration, amount of lease payments, quality of collateral and alignment of the borrowing term and lease term. The Company considers
Maturity Analysis of Short-Term and Operating Leases
The following table approximates the dollar maturity of the Company’s undiscounted payments for its short-term leases and operating lease liabilities as of June 30, 2024:
Years ending June 30, |
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Present value of lease liabilities | $ | |
9. SUBSEQUENT EVENTS
On July 21, 2024, the Company, entered into a warrant exercise inducement (the “Inducement”) with certain holders (each a “Holder”) of its Tranche B warrants (the “Existing Warrants”), which were originally issued in September 8, 2023, pursuant to which the Holders agreed to exercise for cash their Existing Warrants to purchase an amount of shares of the Company’s Series E-4 Convertible Voting Preferred Stock, par value $
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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
Overview
You should read the following discussion and analysis of our financial condition and results of operations together with the unaudited financial information and notes thereto included in this Quarterly Report on Form 10-Q. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report, including information with respect to our plans and strategy for our business, include forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the “Risk Factors” section in our Annual Report on Form 10-K/A for the year ended December 31, 2023, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.
We are a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer. Our core objective is to leverage our proprietary phospholipid ether drug conjugate™ (PDC™) delivery platform to develop PDCs that are designed to specifically target cancer cells and deliver improved efficacy and better safety as a result of fewer off-target effects. We believe that our PDC platform possesses the potential for the discovery and development of the next generation of cancer-targeting treatments, and we plan to develop PDCs both independently and through research and development collaborations.
Our lead PDC therapeutic, iopofosine I 131 (iopofosine) is a small-molecule PDC designed to provide targeted delivery of iodine-131 directly to cancer cells, while limiting exposure to healthy cells. We believe this profile differentiates iopofosine from many traditional on-market treatments and radiotherapeutics. Our CLOVER-WaM Phase 2 pivotal study of iopofosine in patients with relapsed/refractory (r/r) Waldenstrom’s macroglobulinemia (WM) has completed, and our Phase 2b studies in r/r multiple myeloma (MM) patients and r/r central nervous system lymphoma (CNSL) are ongoing. The CLOVER-2 Phase 1a study for a variety of pediatric cancers has concluded and a Phase 1b study in pediatric patients with high grade glioma is enrolling. Additionally, a Phase 1 Investigator-initiated study conducted by the University of Wisconsin Madison of iopofosine I 131 in combination with external beam radiation in patients with recurrent head and neck cancer has also completed. As with all clinical trials, adverse events, serious adverse events or fatalities may arise during a clinical trial resulting from medical problems that may not be related to clinical trial treatments.
The U.S. Food and Drug Administration (FDA) granted iopofosine Fast Track Designation for lymphoplasmacytic lymphoma (LPL) and WM patients having received two or more prior treatment regimens, as well as r/r MM and r/r diffuse large B-cell lymphoma (DLBCL). Orphan Drug Designations (ODDs) have been granted for LPL/WM, MM, neuroblastoma, soft tissue sarcomas including rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. Iopofosine was also granted Rare Pediatric Disease Designation (RPDD) for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. The European Commission granted ODD to iopofosine for treatment of r/r MM and WM, as well as PRIME designation for WM.
Additionally, in June 2020, the European Medicines Agency (EMA) granted us Small and Medium-Sized Enterprise (SME) status by the EMA’s Micro, Small and Medium-sized Enterprise office. SME status allows us to participate in significant financial incentives that include a 90% to 100% EMA fee reduction for scientific advice, clinical study protocol design, endpoints and statistical considerations, quality inspections of facilities and fee waivers for selective EMA pre-and post-authorization regulatory filings, including orphan drug and PRIME designations. We are also eligible to obtain EMA certification of quality and manufacturing data prior to review of clinical data. Other financial incentives include EMA-provided translational services of all regulatory documents required for market authorization, further reducing the financial burden of the market authorization process.
Our product pipeline also includes a PDC-based targeted alpha-emitter therapy utilizing actinium-225 as the payload (CLR121225) currently in IND enabling studies. We are also evaluating other alpha emitting isotopes such as astatine-211 and lead-212 preclinically. Additionally, our preclinical PDC programs include small molecule chemotherapeutic compounds, oligonucleotide programs and peptide conjugate programs as well as several partnered PDC assets. These other programs are being developed primarily for solid tumors.
We have leveraged our PDC platform to establish three ongoing collaborations featuring four unique payloads and mechanisms of action. Through research and development collaborations, our strategy is to generate near-term capital, supplement internal resources, gain access to novel molecules or payloads, accelerate product candidate development, and broaden our proprietary and partnered product pipelines.
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Our PDC platform is designed to provide selective delivery of a diverse range of oncologic payloads to cancerous cells, whether a hematologic cancer or solid tumor; a primary tumor, or a metastatic tumor; and cancer stem cells. The PDC platform’s mechanism of entry is designed not to rely upon a specific cell surface epitope or antigen as are required by other targeted delivery platforms but rather a unique change in the tumor cell membrane. Our PDC platform takes advantage of a metabolic pathway (beta oxidation) utilized by nearly all tumor cell types in all stages of the tumor cycle. Tumor cells modify the cell membrane to create specific, highly organized microdomains by which to transport lipids and long chain fatty acids into the cytoplasm, as a result of the utilization of this metabolic pathway. Our PDCs are designed to bind to these regions and directly enter the intracellular compartment. This mechanism allows the PDC molecules to accumulate in tumor cells over time, which we believe can enhance drug efficacy. The direct intracellular delivery allows our molecules to avoid the specialized, highly acidic cellular compartment known as lysosomes, which allows a PDC to deliver payloads that previously could not be delivered in this targeted manner. Additionally, molecules targeting specific cell surface epitopes face challenges in completely eliminating a tumor because the targeted antigens are limited in the total number presented on the cell surface, limiting total potential uptake and resulting in heterogenous uptake across the tumor, have longer cycling time from internalization to relocation on the cell surface, again diminishing their availability for binding, and are not present on all of the tumor cells because of the heterogenous nature of cancer cells, further increasing the unequal distribution of the drug across the tumor. This means a subpopulation of tumor cells always exists that cannot be addressed by therapies targeting specific surface epitopes. Additionally, many epitopes utilized are also present on other normal tissue, resulting in off-target toxicities.
Beyond the benefits provided by the mechanism of entry, the PDC platform features include the capacity to link with almost any molecule, provide a significant increase in targeted oncologic payload delivery, a more uniform delivery, and the ability to target virtually all types of tumor cells. As a result, we believe that we can create PDCs to treat a broad range of cancers with the potential to improve the therapeutic index of oncologic drug payloads, enhance or maintain efficacy while also reducing adverse events by minimizing drug delivery to healthy cells, and increasing delivery to cancerous cells and cancer stem cells.
We employ a drug discovery and development approach that allows us to efficiently design, research and advance drug candidates. Our iterative process allows us to rapidly and systematically produce multiple generations of incrementally improved targeted drug candidates without the expense of having to generate significant compound libraries.
A description of our PDC product candidates follows:
Clinical Pipeline
Our lead PDC therapeutic, iopofosine, is a small-molecule PDC designed to provide targeted delivery of iodine-131 directly to cancer cells, while limiting exposure to healthy cells. We believe this profile differentiates iopofosine from many traditional on-market treatments and treatments in development. Iopofosine was recently evaluated in the completed CLOVER-WaM Phase 2 pivotal study in patients with r/r WM, while evaluation is ongoing in a Phase 2b study in r/r MM and CNS lymphoma patients and the CLOVER-2 Phase 1b study for pediatric patients with high grade gliomas. Adverse events across all studies have been largely restricted to fatigue (39%), and cytopenias; specifically, thrombocytopenia (75%), anemia (61%), neutropenia (54%), leukopenia (56%), and lymphopenia (34%). Fatalities have occurred in patients post-treatment with iopofosine.
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The CLOVER-WaM pivotal Phase 2b study completed enrollment of WM patients that have received at least two previous lines of therapy including those that failed or had a suboptimal response to a BTKi therapy in 4Q 2023. Topline safety data was reported on 45 patients meeting the criteria for the mITT population with a data cut-off date of January 3, 2024. Topline efficacy evaluable population (n=41) was defined as patients who were in the mITT and had follow up of at least 60 days post last dose. The CLOVER-WaM study met its primary endpoint with a MRR of 61% (95% confidence interval [44.50%, 75.80%, two-sided p value < 0.0001]) exceeding the agreed-upon statistical hurdle of 20%. The ORR in evaluable patients was 75.6%, and 100% of patients experienced disease control. Responses were durable, with median duration of response not reached and 76% of patients remaining progression free at a median follow-up of eight months. These outcomes exceed real world data, which demonstrate a 4-12% MRR and a duration of response of approximately six months or less despite continuous treatment in a patient population that is less pretreated and not refractory to multiple classes of drugs. Notably, iopofosine monotherapy achieved a 7.3% complete remission (CR) rate in this highly refractory WM population. Iopofosine I 131 was well tolerated and its toxicity profile was consistent with the Company’s previously reported safety data. There were no treatment-related adverse events (TRAEs) leading to discontinuation. The rates of Grade 3 or greater TRAEs observed in more than 10% of patients included thrombocytopenia (55%), neutropenia (37%), and anemia (26%). All patients recovered from cytopenias with no reported aplastic sequalae. Importantly, there were no clinically significant bleeding events, and the rate of febrile neutropenia was 2%. There were no treatment-related deaths in the study.
The CLOVER-1 Phase 2 study met the primary efficacy endpoints from the Part A dose-finding portion, conducted in r/r B-cell malignancies, and is now enrolling an MM and CNSL expansion cohort (Phase 2b). The Phase 2b study will evaluate highly refractory MM patients in triple class, quad- and penta-drug refractory patients, including post-BCMA immunotherapy patients and r/r CNSL patients. The initial Investigational New Drug (IND) application was accepted by the FDA in March 2014 with multiple INDs submitted since that time. The Phase 1 study was designed to assess the compound’s safety and tolerability in patients with r/r MM and to determine maximum tolerated dose (MTD) and was initiated in April 2015. The study completed enrollment and the final clinical study report is expected in the first half of 2025. Initiated in March 2017, the primary goal of the Phase 2a study was to assess the compound’s efficacy in a broad range of hematologic cancers.
The CLOVER-2 Phase 1a pediatric study was conducted internationally at seven leading pediatric cancer centers. The study was an open-label, sequential-group, dose-escalation study to evaluate the safety and tolerability of iopofosine in children and adolescents with relapsed or refractory cancers, including malignant brain tumors, neuroblastoma, sarcomas, and lymphomas (including Hodgkin’s lymphoma). The maximum tolerated dose was determined to be greater than 60mCi/m2 administered as a fractionated dose. CLOVER-2 Phase 1b study is an open-label, international dose-finding study evaluating two different doses and dosing regiments of iopofosine in r/r pediatric patients with high grade gliomas. These cancer types were selected for clinical, regulatory and commercial rationales, including the radiosensitive nature and continued unmet medical need in the r/r setting, and the rare disease determinations made by the FDA based upon the current definition within the Orphan Drug Act.
In December 2014, the FDA granted ODD for iopofosine for the treatment of MM. In 2018, the FDA granted ODD and RPDD for iopofosine for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma, and osteosarcoma. In May 2019, the FDA granted Fast Track Designation for iopofosine for the treatment of MM and in July 2019 for the treatment of DLBCL. In September 2019 iopofosine received ODD from the European Union for MM. In December 2019, the FDA and the European Union each granted ODD for iopofosine for the treatment of WM. In September 2023, the European Union granted PRIME designation for iopofosine for the treatment of r/r WM. The FDA granted Fast Track designation for iopofosine for the treatment of r/r LPL and WM in May 2020.
As the result of iopofosine’s RPDD designation, we may be eligible to receive a priority review voucher (PRV) if the product receives approval for any of the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma, or osteosarcoma. The FDA may award PRV to sponsors of a product application for a RPDD that meet its specified criteria. The key criteria to receiving PRV is that the drug be approved for a rare pediatric disease and treat a serious or life-threatening manifestation of the disease or condition that primarily affects individuals under the age of 18. In order to receive a PRV, a sponsor must obtain approval of a “rare pediatric disease product application,” which is a human drug application for prevention or treatment of a rare pediatric disease and which contains no active ingredient, including any ester or salt thereof, that has been approved by the FDA; is deemed eligible for priority review; is submitted under section 505(b)(1) of the Federal Food, Drug, and Cosmetic Act (FDCA) or section 351(a) of the Public Health Service Act (PHSA); relies on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population; does not seek approval for an adult indication in the original rare pediatric disease application; and is approved after September 30, 2016. Under this program, a sponsor who receives an approval for a drug or biologic for a rare pediatric disease can receive a PRV that can be redeemed to receive a priority review of a subsequent marketing application for a different product. Additionally, the PRV’s can be exchanged or sold to other companies so that the receiving company may use the voucher. Congress has only authorized the rare pediatric disease priority review voucher program until September 30, 2024. However, if a drug candidate receives RPDD before September 30, 2024, it is eligible to receive a voucher if it is approved before September 30, 2026.
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CLOVER-WaM: Phase 2 Pivotal Study in: Patients with r/r Waldenstrom’s Macroglobulinemia
We participated in a Type C guidance meeting with the FDA in September 2020. The results of that guidance meeting provided us with an agreed upon path for conducting the CLOVER-WaM study; a single arm, pivotal study in WM patients that have received and relapsed or were refractory to two prior lines of therapy, including having failed or had a suboptimal response to BTKi therapy. WM is a rare, indolent, and incurable form of non-Hodgkin’s lymphoma (NHL) that is composed of a patient population in need of new and better treatment options.
The study enrolled 65 WM patients who have received at least two prior lines of therapy, failed both lines of therapy including having failed or had a suboptimal response to a BTKi (i.e. ibrutinib). Patients in the trial received 4-doses of iopofosine over two cycles (cycle one: days 1, 15, and cycle two: days 57, 71) with each dose administered as a 15mCi/m2 infusion. The primary endpoint of the trial is major response rate (MRR) defined as a partial response (a minimum of a 50% reduction in IgM) or better in patients that receive a minimum total body dose (TBD) of 60 mCi with secondary endpoints of treatment-free survival (treatment-free remission), duration of response and progression-free survival. An independent data monitoring committee (iDMC) performed an interim safety and futility evaluation on the first 10 patients enrolled. If three of the 10 patients experienced a Clinically Significant Toxicity (CST) then the dose would have been reduced to 12.5 mCi/m2. We believe this design aligned with the feedback received from the FDA during the guidance meeting held in September 2020 and subsequent interactions. The FDA accepted the dose to be tested, our proposal for a safety and futility assessment to be conducted on the first 10 patients, the endpoint to be assessed, the statistical analysis plan and study size of approximately 50 patients in the mITT population (>60mCi TBD). Based upon this agreement, the pivotal study was initiated. The interim futility and safety assessment occurred in 2022 and the iDMC determined the study exceeded the futility threshold and that the CST threshold was not met, therefore the study should continue to enroll with no change to the dosing regimen. The study achieved full enrollment in the fourth quarter 2023 and topline safety data was reported on 45 patients meeting the criteria for the mITT population with a data cut-off date of January 3, 2024. Among mITT patients, median age was 71 years, median IgM level prior to treatment with iopofosine was 2,185, 90% were refractory to either a BTKi (18/36 50%) or anti-CD20 therapy (18/41 40%), with 26.7% multiclass refractory, and 80% of patients were previously treated with a BTKi therapy. Topline efficacy evaluable population (n=41) was defined as patients who were in the mITT and had follow up of at least 60 days post last dose. The CLOVER WaM study met its primary endpoint with a major response rate (MRR) of 61% (95% confidence interval [44.50%, 75.80%, two-sided p value < 0.0001]) exceeding the agreed upon statistical hurdle of 20%. The overall response rate (ORR) in evaluable patients was 75.6%, and 100% of patients experienced disease control. Responses were durable, with median duration of response not reached and 76% of patients remaining progression free at a median follow-up of eight months. These outcomes exceed real world data, which demonstrate a 4-12% MRR and a duration of response of approximately six months or less despite continuous treatment in a patient population that is less pretreated and not refractory to multiple classes of drugs. Notably, iopofosine monotherapy achieved an 7.3% complete remission (CR) rate in this highly refractory WM population. Iopofosine I 131 was well tolerated and its toxicity profile was consistent with the Company’s previously reported safety data. There were no treatment-related adverse events (TRAEs) leading to discontinuation. The rates of Grade 3 or greater TRAEs observed in more than 10% of patients included thrombocytopenia (55%), neutropenia (37%), and anemia (26%). All patients recovered from cytopenias with no reported aplastic sequalae. Importantly, there were no clinically significant bleeding events, and the rate of febrile neutropenia was 2%. There were no treatment-related deaths in the study.
CLOVER-1: Phase 2 Study in Select B-Cell Malignancies
The Phase 2 CLOVER-1 study was an open-label study designed to determine the efficacy and safety of CLR 131 in select B-cell malignancies (multiple myeloma (MM), indolent chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), lymphoplasmacytic lymphoma (LPL)/Waldenstrom’s macroglobulinemia (WM), marginal zone lymphoma (MZL), mantle cell lymphoma (MCL), DLBCL, and central nervous system lymphoma (CNSL) who have been previously treated with standard therapy for their underlying malignancy. As of March 2022, the study arms for CLL/SLL, LPL/WM, MZL, MCL, and DLBCL were closed. Dosing of patients varied by disease state cohort and was measured in terms of TBD.
In July 2016, we were awarded a $2,000,000 National Cancer Institute (NCI) Fast-Track Small Business Innovation Research grant to further advance the clinical development of iopofosine. The funds supported the Phase 2 study initiated in March 2017 to define the clinical benefits of iopofosine in r/r MM and other niche hematologic malignancies with unmet clinical need. These niche hematologic malignancies include CLL, SLL, MZL, LPL/WM and DLBCL. The study was conducted in approximately 10 U.S. cancer centers in patients with orphan-designated relapse or refractory hematologic cancers. The planned study enrollment was up to 80 patients.
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The study’s primary endpoint was clinical benefit response (CBR), with secondary endpoints of ORR, PFS, time to next treatment (TtNT), median Overall Survival (mOS), DOR and other markers of efficacy following patients receiving one of three TBDs of iopofosine (<50mCi, ~50mCi and >60mCi), with the option for a second cycle approximately 75-180 days later. Dosages were provided either as a single bolus or fractionated (the assigned dose level split into two doses) given day 1 and day 15. Over the course of the study the dosing regimen of iopofosine advanced from a single bolus dose to two cycles of fractionated administrations of 15 mCi/m2 per dose on days 1, 15 (cycle 1), and days 57, 71 (cycle 2). Adverse events occurring in at least 25% of subjects were fatigue (39%) and cytopenias, specifically, thrombocytopenia (75%), anemia (61%), neutropenia (54%), leukopenia (51%), and lymphopenia (25%). Serious adverse events occurring in greater than 5% of subjects were restricted to thrombocytopenia (9%) and febrile neutropenia (7.5%).
Phase 2a Study: Patients with r/r Waldenstrom’s Macroglobulinemia Cohort
Patients in the r/r WM cohort all received TBD of ≥ 60 mCi (25 mCi/m2 single bolus, 31.25 mCi/m2 fractionated, 37.5 mCi/m2 fractionated, or two cycles of mCi/m2 fractionated) either as a bolus dose or fractionated. Current data from our Phase 2a CLOVER-1 clinical study show a 100% ORR in six WM patients and an 83.3% major response rate with one patient achieving a complete response (CR), which reached 39 months post-last treatment. While median treatment free survival (TFS), also known as treatment free remission (TFR), and DOR have not been reached, the average treatment TFS/TFR is currently at 330 days. We believe this may represent an important improvement in the treatment of r/r WM as we believe no approved or late-stage development treatments for second- and third-line patients have reported a CR to date. Based on study results to date, patients continue to tolerate iopofosine well, with the most common adverse events being cytopenias and fatigue.
Phase 2a Study: Patients with r/r Multiple Myeloma Cohort
In September 2020, we announced that a 40% ORR was observed in the subset of refractory MM patients deemed triple class refractory who received 60 mCi or greater TBD. Triple class refractory is defined as patients that are refractory to immunomodulatory, proteasome inhibitors and anti-CD38 antibody drug classes. The 40% ORR (6/15 patients) represents triple class refractory patients enrolled in Part A of Cellectar’s CLOVER-1 study and additional patients enrolled in Part B from March through May 2020 and received >60mCi TBD (25 mCi/m2 single bolus, 31.25 mCi/m2 fractionated, 37.5 mCi/m2 fractionated, or two cycles of mCi/m2 fractionated) either as a bolus dose or fractionated. Patients with MM received 40 mg of dexamethasone concurrently beginning within 24 hours of the first CLR 131 infusion. All MM patients enrolled in the expansion cohort are required to be triple class refractory. The additional six patients enrolled in 2020 were heavily pre-treated with an average of nine prior multi-drug regimens. Three patients received a TBD of > 60 mCi and three received less than 60 mCi. Consistent with the data released in February 2020, patients receiving > 60 mCi typically exhibit greater responses. Based on study results to date, patients continue to tolerate iopofosine well, with the most common and almost exclusive treatment-emergent adverse events are cytopenias, such as thrombocytopenia, neutropenia, and anemia.
In December 2021, we presented data from 11 MM patients from our ongoing Phase 2 CLOVER-1 study in a poster at the American Society of Hematology (ASH) Annual Meeting and Exposition. The MM patients were at least triple class refractory (defined as refractory to an immunomodulatory agent, proteasome inhibitor and monoclonal antibody) with data current as of May 2021. Patients had a median of greater than 7 prior therapies with 50% classified as high risk. Initial results in these patients showed an ORR of 45.5%, a CBR of 72.7%, and a disease control rate (DCR) of 100%. Median PFS was 3.4 months. In a subset of five quad/penta drug refractory patients, efficacy increased, demonstrating an ORR of 80% and CBR of 100% in this highly treatment refractory group. The most commonly observed treatment emergent adverse events were cytopenias that included Grade 3 or 4 thrombocytopenia (62.5%), anemia (62.5%), neutropenia (62.5%) and decreased white blood cell count (50%). Treatment emergent adverse events were mostly limited to bone marrow suppression in line with prior observations. No patients experienced treatment emergent adverse events of neuropathy, arrhythmia, cardiovascular event, bleeding, ocular toxicities, renal function, alterations in liver enzymes, or infusion-site reactions or adverse events. We continue to enrich the r/r MM patient cohort with patients that are even more refractory, specifically enrolling patients that are quad-class refractory (triple class plus refractory to any of the recent approved product classes) and have relapsed post-BCMA immunotherapy. We reported in the Blood Cancer Journal in August 2022 that iopofosine demonstrated a 50% ORR in patients receiving >60mCi total administered dose (3/6 patients).
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Phase 2a: Patients with r/r non-Hodgkin’s lymphoma Cohort
In February 2020, we announced positive data from our Phase 2a CLOVER-1 study in patients with NHL patients were treated with three different doses (<50mCi, ~50mCi and >60mCi TBD. Patients in the r/r NHL cohort received TBD of either ≥ 60 mCi or < 60 mCi (25 mCi/m2 single bolus, 31.25 mCi/m2 fractionated, 37.5 mCi/m2 fractionated, or two cycles of mCi/m2 fractionated) either as a bolus dose or fractionated. Patients with r/r NHL who received <60mCi TBD and the >60mCi TBD had a 42% and 43% ORR, respectively and a combined rate of 42%. These patients were also heavily pre-treated, having a median of three prior lines of treatment (range, 1 to 9) with the majority of patients being refractory to rituximab and/or ibrutinib. The patients had a median age of 70 with a range of 51 to 86. All patients had bone marrow involvement with an average of 23%. In addition to these findings, subtype assessments were completed in the r/r B-cell NHL patients. Patients with DLBCL demonstrated a 30% ORR with one patient achieving a CR, which continues at nearly 24 months post-treatment. The ORR for CLL/SLL and MZL patients was 33%.
Based upon the dose response observed in the Phase 2a study for patients receiving TBDs of 60mCi or greater, we determined that patient dosing of iopofosine in the pivotal study would be >60mCi TBD. Therefore, patients are now grouped as receiving <60mCi or >60mCi TBD.
The most frequently reported adverse events in all patients were cytopenias, which followed a predictable course and timeline. The frequency of adverse events did not increase as doses were increased and the profile of cytopenias remained consistent. Importantly, our assessment is that these cytopenias have had a predictable pattern to initiation, nadir and recovery and are treatable. The most common grade ≥3 events at the highest dose (75mCi TBD) were hematologic toxicities including thrombocytopenia (65%), neutropenia (41%), leukopenia (30%), anemia (24%) and lymphopenia (35%). No patients experienced cardiotoxicities, neurological toxicities, infusion site reactions, peripheral neuropathy, allergic reactions, cytokine release syndrome, keratopathy, renal toxicities, or changes in liver enzymes. The safety and tolerability profile in patients with r/r NHL was similar to r/r MM patients except for fewer cytopenias of any grade. Based upon iopofosine being well tolerated across all dose groups, the observed response rate, and especially in difficult to treat patients such as high risk and triple class refractory or penta-refractory, and corroborating data showing the potential to further improve upon current ORRs and durability of those responses, the study has been expanded to test a two-cycle dosing optimization regimen with a target TBD >60 mCi/m2 of iopofosine.
In May 2020, we announced that the FDA granted Fast Track Designation for iopofosine in WM in patients having received two or more prior treatment regimens.
Phase 1 Study in Patients with r/r Multiple Myeloma
In February 2020, final results from a multicenter, Phase 1 dose escalation clinical trial of iopofosine in r/r MM were presented. The trial was designed to evaluate the safety and potential initial efficacy of iopofosine administered in an up to 30-minute I.V. infusion either as a single bolus dose or as a fractionated dose in heavily pretreated MM patients. The study enrolled a total of 26 evaluable patients at three trial sites. For the trial, which used a modified three-plus-three dose escalation design, 15 evaluable patients were dosed in single bolus doses from 12.5mCi/m2 up to 31.25mCi/m2 (TBD 20.35-59.17 mCi) and 11 evaluable patients were dosed in fractionated dosing cohorts of 31.25mCi/m2 to 40mCi/m2 (TBD 54.915-89.107 mCi). An iDMC did not identify dose-limiting toxicities in any cohort. Of the 26 evaluable patients in the trial, a partial response was observed in 4 of 26 patients (15.4%) and stable disease or minimal response in 22 of 26 patients (84.6%), for a disease control rate of 100%. A significant decrease in M-protein and free light chain (FLC) was also observed.
Iopofosine in combination with dexamethasone was under investigation in adult patients with r/r MM. MM is an incurable cancer of the plasma cells and is the second most common form of hematologic cancer. Patients had to be refractory to or relapsed from at least one proteasome inhibitor and at least one immunomodulatory agent. The clinical study was a standard three-plus-three dose escalation safety study to determine the maximum tolerable dose. We use the International Myeloma Working Group (IMWG) definitions of response, which involve monitoring the surrogate markers of efficacy, M protein and FLC. The IMWG defines a PR as a 50% or greater decrease in M protein or to 50% or greater decrease in FLC levels (for patients in whom M protein is unmeasurable). Secondary objectives included the evaluation of therapeutic activity by assessing surrogate efficacy markers, which include M protein, FLC, PFS and OS. All patients were heavily pretreated with an average of five prior lines of therapy. An iDMC assessed the safety of iopofosine up to its planned maximum single, bolus dose of 31.25 mCi/m2 or a TBD of ~63 mCi. The four single dose cohorts examined were: 12.5 mCi/m2 (~25mCi TBD), 18.75 mCi/m2 (~37.5mCi TBD), 25 mCi/m2(~50mCi TBD), and 31.25 mCi/m2(~62.5mCi TBD), all in combination with low dose dexamethasone (40 mg weekly). Of the five patients in the first cohort, four were assessed as achieving stable disease and one patient progressed at Day 15 after administration and was taken off the study. Of the five patients admitted to the second cohort, all five were assessed as achieving stable disease; however, one patient progressed
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at Day 41 after administration and was taken off the study. Four patients were enrolled to the third cohort, and all were assessed as achieving stable disease. In September 2017, we announced safety and tolerability data for cohort 4, in which patients were treated with a single infusion up to 30-minutes of 31.25mCi/m2 of iopofosine, which was tolerated by the three patients in the cohort. Additionally, all three patients experienced CBR with one patient achieving a partial response (PR). The patient experiencing a PR had an 82% reduction in FLC. This patient did not produce M protein, had received seven prior lines of treatment including radiation, stem cell transplantation and multiple triple combination treatments including one with daratumumab that was not tolerated. One patient experiencing stable disease attained a 44% reduction in M protein. In January 2019, we announced that the pooled mOS data from the first four cohorts was 22.0 months. In late 2018, we modified this study to evaluate a fractionated dosing strategy to potentially increase efficacy and decrease adverse events.
Cohorts five and six received fractionated dosing of 31.25 mCi/m2(~62.5mCi TBD) and 37.5 mCi/m2 (~75mCi TBD), each administered on day 1 and day 8. Following the determination that all prior dosing cohorts were tolerated, we initiated a cohort seven utilizing a 40mCi/m2 (~95mCi TBD) fractionated dose administered 20mCi/m2 (~40mCi TBD) on days 1 and day 8. Cohort seven was the highest pre-planned dose cohort and subjects have completed the evaluation period. The study completed enrollment and the final clinical study report is expected in the first half of 2022. Adverse events occurring in at least 25% of subjects were fatigue (26%) and cytopenias, specifically, thrombocytopenia (90%), anemia (65%), neutropenia (55%), leukopenia (61%), and lymphopenia (58%). Serious adverse events occurring in greater than two subjects were restricted to febrile neutropenia n=3 (9.7%).
In May 2019, we announced that the FDA granted Fast Track Designation for iopofosine in fourth line or later r/r MM. Iopofosine is currently being evaluated in our ongoing CLOVER-1 Phase 2 clinical study in patients with r/r MM and other select B-cell lymphomas. Patients in the study received up to four, approximately 20-minute, IV infusions of iopofosine over 3 months, with doses given 14 days apart in each cycle and a maximum of two cycles. Low dose dexamethasone 40 mg weekly (20mg in patients ≥ 75), was provided for up to 12 weeks. The planned study enrollment was up to 80 patients. Its primary endpoint was clinical benefit rate (CBR), with additional endpoints of ORR, PFS, median overall survival (OS) and other markers of efficacy. Over the course of the study the dosing regimen of iopofosine advanced from a single bolus dose to two cycles of fractionated administrations of 15 mCi/m2 per dose on days 1, 15 (cycle 1), and days 57, 71 (cycle 2). Following treatment with iopofosine, approximately 91% of patients experience a reduction in tumor marker with approximately 73% experiencing greater than 37% reduction.
CLOVER 2: Phase 1 Study in r/r Pediatric Patients with select Solid tumors, Lymphomas and Malignant Brain Tumors
In December 2017, the Division of Oncology at the FDA accepted our IND and study design for the Phase 1 study of iopofosine in children and adolescents with select rare and orphan designated cancers. This study was initiated during the first quarter of 2019. In December 2017, we submitted an IND application for r/r pediatric patients with select solid tumors, lymphomas and malignant brain tumors. The Phase 1 clinical study of iopofosine is an open-label, sequential-group, dose-escalation study evaluating the safety and tolerability of intravenous administration of iopofosine in children and adolescents with relapsed or refractory malignant solid tumors (neuroblastoma, Ewing’s sarcoma, osteosarcoma, rhabdomyosarcoma) and lymphoma or recurrent or refractory malignant brain tumors for which there are no standard treatments. Secondary objectives of the study are to identify the recommended efficacious dose of iopofosine and to determine preliminary antitumor activity (treatment response) of iopofosine in children and adolescents.
In August 2020, based on data on four dose levels from 15mCi/m2 up to 60mCi/m2, the iDMC permitted the beginning of the evaluation of the next higher dose cohort, at 75mCi/m2. The iDMC advised, based upon the initial data, to enrich the 60 mCi/m2 dose level for patients over the age of 10 with HGG and Ewing sarcoma. Changes in various tumor parameters appeared to demonstrate initial response and tumor uptake. This includes patients with relapsed HGGs with over five months of PFS. In November 2020, we announced clinical data providing that iopofosine had been measured in pediatric brain tumors, confirming that systemic administration of iopofosine crosses the blood brain barrier and is delivered into tumors and that the data show disease control in heavily pretreated patients with ependymomas. In November 2021, we announced favorable data on changes in various tumor parameters in a Phase 1 study in children and adolescents with relapsed and refractory high-grade gliomas (HGGs) and soft tissue sarcomas. Pediatric HGGs are a collection of aggressive brain and central nervous system tumor subtypes (i.e. diffuse intrinsic pontine gliomas, glioblastomas, astrocytomas, ependymomas, etc.) with about 400 new pediatric cases diagnosed annually in the U.S. Children with these tumors have a poor prognosis and limited 5-year survival. Adverse events occurring in at least 25% of subjects were fatigue, headache, nausea and vomiting (28% respectively), and cytopenias, specifically, thrombocytopenia (67%), anemia (67%), neutropenia (61%), leukopenia (56%), and lymphopenia (33%). There were no serious adverse events occurring in more than 2 subjects. The Part A portion of this Phase 1 study has concluded, and part B has initiated to determine the appropriate dosing regimen in pediatric patients with r/r HGG. In 2022, the NCI awarded Cellectar a $1,900,000 SBIR Phase 2 grant to explore iopofosine in pediatric HGG.
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In 2018, the FDA granted ODD and RPDD for iopofosine for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. If iopofosine should be approved for any of these pediatric indications, the first approved RPDD would enable us to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive priority review for a future New Drug Application (NDA) or Biologic License Application (BLA) submission, which would reduce the FDA review time from 12 months to six months. Currently, these vouchers can also be transferred or sold to another entity. In December 2020, the FDA extended the Priority Review Voucher Program through September 2026 for rare pediatric diseases.
Phase 1 Study in r/r Head and Neck Cancer
In August 2016, the University of Wisconsin Carbone Cancer Center (UWCCC) was awarded a five-year Specialized Programs of Research Excellence (SPORE) grant of $12,000,000 from the NCI and the National Institute of Dental and Craniofacial Research to improve treatments and outcomes for head and neck cancer (HNC) patients. HNC is the sixth most common cancer across the world with approximately 56,000 new patients diagnosed every year in the U.S. As a key component of this grant, the UWCCC researchers completed testing of iopofosine in various animal HNC models and initiated the first human clinical study enrolling up to 30 patients combining iopofosine and external beam radiation treatment (EBRT) with recurrent HNC in the fourth quarter of 2019. UWCCC has completed the part A portion of a safety and tolerability study of iopofosine in combination with EBRT and preliminary data suggest safety and tolerability in relapsed or refractory HNC. The reduction in the amount or fractions (doses) of EBRT has the potential to diminish the (number and severity of) adverse events associated with EBRT. Patients with HNC typically receive approximately 60-70 Grays (Gy) of EBRT given as 2 – 3 Gy daily doses over a six-week timeframe. Patients can experience long-term tumor control following re-irradiation in this setting; however, this approach can cause severe injury to normal tissue structures, significant adverse events and diminished quality of life. Part B of the study was to assess the safety and potential benefits of iopofosine in combination with EBRT in a cohort of up to 24 patients. This portion of the study has fully enrolled, and data were reported at the ASTRO 2024 conference on March 2, 2024. Complete remission was achieved in 64% of patients, with an ORR of 73% (n=11). Prior to treatment with iopofosine I 131, six patients had multiple recurrence and one had metastatic disease, both of which are indicative of poor outcomes. Additionally, the study demonstrated durability of tumor control with an overall survival of 67% and progression free survival of 42% at 12 months. Eleven patients (92%) experienced a treatment-related adverse event. Treatment-related adverse events of grade 3 or higher occurring in 20% or more patients were thrombocytopenia (75%), lymphopenia (75%), leukopenia (75%), neutropenia (67%), and anemia (42%). Observed adverse events were consistent with the known toxicity profile of iopofosine I 131, with cytopenias being the most common. All patients recovered. We believe that these data support the notion of enhanced patient outcomes when combining the use of iopofosine I 131 in combination with external beam radiation for a treatment of solid tumors.
Preclinical Pipeline
We believe our PDC platform has potential to provide targeted delivery of a diverse range of oncologic payloads, as exemplified by the product candidates listed below, that may result in improvements upon current standard of care (SOC) for the treatment of a broad range of human cancers:
| ● | CLR 12120 Series is an alpha emitting radio-conjugate program. The company has validated the in vivo potential of alpha emitting phospholipid radioconjugates and their potential to treat highly refractory and difficult to treat solid tumors. Cellectar is currently progressing with a lead molecule using actinium-225 as the alpha emitting payload. |
| ● | The company has developed a series of proprietary small molecule phospholipid drug conjugates. These programs employ either novel payload or novel linkers. Many of these molecules have demonstrated efficacy and tolerability in preclinical mouse models. The collaboration with IntoCell Inc. successfully met its agreed upon endpoint. The collaboration provided significant data which has led Cellectar to select a series of highly potent cytotoxic small molecule payloads for further development. |
| ● | In collaboration with other parties, Cellectar has also validated that the PLE is capable of delivering peptide payloads and oligonucleotide (siRNA, mRNA, etc.) payloads to the tumors when delivered systemically. These molecules have also been shown to demonstrate activity and safety in multiple preclinical mouse models. Based upon these collaborations and the data, the company has initiated internal proprietary programs with each of these treatment modalities. |
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Results of Operations
Research and development expense. Research and development expense consist of costs incurred in identifying, developing and testing, and manufacturing product candidates, which primarily include salaries and related expenses for personnel, cost of manufacturing materials and contract manufacturing fees paid to contract manufacturers and contract research organizations, fees paid to medical institutions for clinical studies, and costs to secure intellectual property. The Company analyzes its research and development expenses based on four categories as follows: clinical project costs, preclinical project costs, manufacturing and related costs, and general research and development costs that are not allocated to the functional project costs, including personnel costs, facility costs, related overhead costs and patent costs.
General and administrative expense. General and administrative expense consists primarily of salaries and other related costs for personnel in executive, finance and administrative functions. Other costs include insurance, costs for public company activities, investor relations, directors’ fees and professional fees for legal and accounting services.
Three Months Ended June 30, 2024 and 2023
Research and Development. Research and development expense for the three months ended June 30, 2024 was approximately $7,345,000, compared to approximately $6,135,000 for the three months ended June 30, 2023.
The following table is a summary comparison of approximate research and development costs for the three months ended June 30, 2024 and 2023:
Three Months Ended | |||||||||
June 30, | |||||||||
| 2024 |
| 2023 |
| Variance | ||||
Clinical project costs | $ | 3,951,000 | $ | 2,284,000 | $ | 1,667,000 | |||
Manufacturing and related costs |
| 2,628,000 |
| 2,258,000 |
| 370,000 | |||
Pre-clinical project costs |
| 31,000 |
| 33,000 |
| (2,000) | |||
General research and development costs |
| 735,000 |
| 1,560,000 |
| (825,000) | |||
$ | 7,345,000 | $ | 6,135,000 | $ | 1,210,000 | ||||
The overall increase in research and development expense of approximately $1,210,000, or 20%, was primarily a result of increased clinical project costs of approximately $1,667,000, driven by the timing of the activities related to our pivotal and pediatric trials and an increase in personnel.
General and administrative. General and administrative expense for the three months ended June 30, 2024 was approximately $6,358,000, compared to approximately $2,159,000 for the same period in 2023. The overall increase in general and administrative expense of approximately $4,199,000, or 194%, was primarily driven by costs associated with the development of infrastructure necessary to support commercialization upon anticipated NDA approval, including the related marketing and personnel cost.
Other income (expense), net. Other income (expense), net, for the three months ended June 30, 2024, was income of approximately $12,784,000, as compared to approximately $1,885,000 of expense in the same period of 2023, resulting almost exclusively from changes in warrant valuation. Fluctuations in the Company’s common stock price result in warrant valuation changes that are presented here. Interest income increased year-over-year to approximately $329,000 in 2024 as compared to approximately $73,000 in 2023. The Company’s cash on hand and increased interest rates drove the improved return.
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Six Months Ended June 30, 2024 and 2023
Research and Development. Research and development expense for the six months ended June 30, 2024 was approximately $14,434,000, compared to approximately $12,494,000 for the six months ended June 30, 2023.
The following table is a summary comparison of approximate research and development costs for the six months ended June 30, 2024 and 2023:
| Six Months Ended |
| |||||||
June 30, | |||||||||
2024 |
| 2023 | Variance | ||||||
Clinical project costs | $ | 6,644,000 | $ | 4,909,000 | $ | 1,735,000 | |||
Manufacturing and related costs |
| 5,941,000 |
| 4,175,000 |
| 1,766,000 | |||
Pre-clinical project costs |
| 52,000 |
| 216,000 |
| (164,000) | |||
General research and development costs |
| 1,797,000 |
| 3,194,000 |
| (1,397,000) | |||
$ | 14,434,000 | $ | 12,494,000 | $ | 1,940,000 | ||||
The overall increase in research and development expense of approximately $1,940,000, or 16%, was primarily a result of increased manufacturing and related costs of approximately $1,766,000 related to production sourcing and increased clinical project costs of approximately $1,735,000 driven by the timing of the activities related to our pivotal and pediatric trial, partially offset by a decrease in general research and development costs.
General and administrative. General and administrative expense for the six months ended June 30, 2024 was approximately $11,272,000, compared to approximately $4,505,000 for the same period in 2023. The overall increase in general and administrative expense of $6,767,000, or 150%, was primarily driven by costs associated with the development of infrastructure necessary to support commercialization upon anticipated NDA approval, including the related marketing and personnel costs.
Other income (expense), net. Other income (expense), net, for the first six months of 2024 was an expense of approximately $1,856,000, while the expense for the same period in 2023 was approximately $370,000. A significant portion of the expense comes from changes in the valuation of the Company’s outstanding warrants. Warrant valuation consists of a number of aspects, but the most significant driver is the value at which the Comapany’s common stock is trading at the end of each reporting period. Interest income was approximately $649,000 year-to-date in 2024, and approximately $197,000 in 2023. The Company’s improved return on cash equivalents is a product of higher average cash balance and a higher interest rate environment.
Liquidity and Capital Resources
We have incurred losses since inception in devoting substantially all of our efforts toward research and development of drug candidates for which we are seeking FDA approval. During the six months ended June 30, 2024, we generated a net loss of approximately $27.6 million and used approximately $27.5 million in cash for operations. We expect that we will continue to generate operating losses for the foreseeable future. As of June 30, 2024, our consolidated cash balance was approximately $25.9 million. As of the date the accompanying consolidated financial statements were issued (the “issuance date”), the Company’s available liquidity to fund the Company’s operations over the next twelve months beyond the issuance date was limited to approximately $34.3 million of unrestricted cash and cash equivalents. Absent further action taken by management to increase its liquidity, the Company may be unable to fund its operations under normal course beyond the second quarter of 2025. To improve the Company’s liquidity, management plans to secure additional outside capital via the sale of equity and/or debt securities or execute a strategic transaction. Management also plans to preserve liquidity, as needed, by implementing temporary cost saving measures. While management believes their plans will be successful, no assurance can be provided such plans will be effectively implemented over the next twelve months beyond the issuance date. In the event management’s plans are not effectively implemented, the Company will be required to seek other alternatives which may include, among others, the sale of assets, discontinuance of certain operations, and/or filing for bankruptcy protection.
These uncertainties raise substantial doubt about the Company’s ability to continue as a going concern. The accompanying financial statements have been prepared on the basis that the Company will continue to operate as a going concern, which contemplates it will be able to realize assets and settle liabilities and commitments in the normal course of business for the foreseeable future.
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Accordingly, the accompanying consolidated financial statements do not include any adjustments that may result from the outcome of these uncertainties.
Item 3. Quantitative and Qualitative Disclosures about Market Risk
We are a smaller reporting company as defined by Rule 12b-2 of the Exchange Act and are not required to provide the information otherwise required under this item.
Item 4. Controls and Procedures
Evaluation of Disclosure Controls and Procedures
Under the supervision, and with the participation of our management, including our principal executive officer and principal financial officer, we conducted an evaluation of the effectiveness of the effectiveness of our disclosure controls and procedures as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”), in connection with the period ending March 31, 2024. Based on that evaluation, management has concluded that as of the respective period, our disclosure controls and procedures were not effective due to the material weaknesses in internal control over financial reporting described below.
Notwithstanding the material weaknesses in our internal control over financial reporting, management has concluded that the audited consolidated financial statements included in this Form 10-Q/A fairly present, in all material respects, our financial position, results of operations and cash flows for the periods presented in conformity with accounting principles generally accepted in the United States of America.
Management’s Report on Internal Control over Financial Reporting
Our management is responsible for establishing and maintaining adequate internal control over financial reporting as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act for the Company. Management assessed the effectiveness of internal control over financial reporting as of the year ended December 31, 2023. In making this assessment, our management used the criteria set forth in the Internal Control - Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission (the “COSO Framework”). Based on this evaluation, our management concluded that our internal control over financial reporting was not effective as of December 31, 2023, continuing through June 30, 2024, because of the material weaknesses described below.
Material Weaknesses
A material weakness is a deficiency, or a combination of deficiencies, in internal control over financial reporting, such that a reasonable possibility exists that a material misstatement of our annual or interim consolidated financial statements would not be prevented or detected on a timely basis.
Management concluded that material weaknesses existed as of the year ended December 31, 2023. Specifically, management identified deficiencies in the principles associated with the control environment, risk assessment, control activities, information and communication and monitoring components of internal control, based on the criteria established by the COSO Framework, that constitute material weaknesses, either individually or in the aggregate.
| ● | Control Environment: The Company lacked appropriate policies and resources to develop and operate effective internal control over financial reporting, which contributed to the Company’s inability to properly analyze, record and disclose accounting matters accurately and timely. This was further impacted by the limited number of staff in the Company’s accounting and finance function. This material weakness contributed to additional material weaknesses further described below. |
| ● | Risk Assessment: The Company does not have a formal process to identify, update, and assess risks, including risks around the accounting for complex transactions, that could significantly impact the design and operation of the Company’s control activities. |
| ● | Control Activities: Management did not design and implement effective control activities and identified the following material weaknesses: |
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| o | Management failed to design and implement adequate internal controls over financial reporting which resulted in the inaccurate accounting of preferred equity and warrants |
| o | Management failed to design and implement adequate internal controls over the recording of stock-based compensation expense related to the restricted stock awards granted in December 2023. |
| o | Management failed to design and implement adequate internal controls over financial reporting as it relates to the proper fair value methodologies and assumptions used to value financial instruments, specific to the assumptions utilized in the valuation of the preferred warrants. |
| ● | Information and Communication: As noted above, the Company had a limited number of staff in its finance and accounting function, and therefore was unable to design and maintain appropriate segregation of duties in the initiation, recording, and approval of transactions within its financial systems. This, coupled with management having not designed and maintained user access controls that adequately restrict user and privileged access to financial applications, and the absence of sufficient other mitigating controls, created a segregation of duties deficiencies. |
| ● | Monitoring Activities: Management did not appropriately select, develop, and perform ongoing evaluations to ascertain whether the components of internal controls are present and functioning |
These material weaknesses resulted in errors that required the restatement of the Company’s consolidated financial statements as of and for the fiscal years ended December 31, 2023 and December 31, 2022, as well as the restatement of the Company’s condensed consolidated financial statements as of and for the interim periods ended September 30, 2023, June 30, 2023, March 31, 2023, September 30, 2022, June 30, 2022, and March 31, 2022. Additionally, these material weaknesses could result in a misstatement of the account balances or disclosures that would result in a material misstatement to the annual or interim consolidated financial statements that would not be prevented or timely detected.
Management’s Plan to Remediate the Material Weaknesses
The process of designing and maintaining effective internal control over financial reporting is a continuous effort that requires management to anticipate and react to changes in our business, economic and regulatory environments and to expend significant resources. In early 2024, the Company began recruiting and hired qualified accounting and financial reporting personnel to supplement our level of knowledge and experience with internal control over financial reporting in order to begin to design and implement a formal control environment and risk assessment process. Such process includes identification of risks, the level of detail in our risk assessment, and the clarity of the linkage between risks and internal controls. The results of this effort are expected to enable us to effectively identify, develop, evolve and implement controls and procedures to address risks. Additionally, the Company has also initiated the implementation of an ERP system, which will provide a system-based control structure for all financial transactions.
As our remediation efforts are still on-going, we will continue to consider the need for additional resources and implement further enhancements to our policies and procedures as necessary to further improve our internal control over financial reporting. As we work to improve our internal control over financial reporting, we may modify our remediation plan and may implement measures as we continue to review, optimize and enhance our financial reporting controls and procedures in the ordinary course. The material weaknesses will not be considered remediated until the remediated controls have been operating for a sufficient period of time and can be evidenced through testing that these are operating effectively.
Changes in Internal Control over Financial Reporting
Except for the identification of the material weaknesses described above, there has been no change in our internal control over financial reporting during the period ended June 30, 2024, that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.
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PART II. OTHER INFORMATION
Item 1. Legal Proceedings
We may be a party to proceedings in the ordinary course of business, however, we do not anticipate that the outcome of such matters and disputes will materially affect our financial statements.
Item 1A. Risk Factors
Other factors that could materially adversely affect our business and our equity securities are described in the Risk Factors previously disclosed in Form 10-K/A, our Annual Report filed with the SEC on October 28, 2024, pursuant to Section 13 or 15(d) of the Exchange Act. That information should be considered carefully, together with other information in this report and other reports and materials we file with the SEC.
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds
None.
Item 3. Default Upon Senior Securities
None.
Item 4. Mine Safety Disclosures
Not applicable.
Item 5. Other Information
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Item 6. Exhibits
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| Incorporation by Reference | ||||
Exhibit |
| Description |
| Filed with |
| Form |
| Filing Date |
| Exhibit |
10.1 | Cellectar Biosciences, Inc. 2021 Stock Incentive Plan, as Amended | 8-K | June 29, 2023 | 10.1 | ||||||
31.1 |
| Certification of Chief Executive Officer pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 |
| X |
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31.2 |
| Certification of Chief Financial Officer pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 |
| X |
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32.1 |
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| X |
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101 |
| Interactive Data Files |
| X |
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104 | Cover Page Interactive Data File (Embedded within the Inline XBRL document and included in Exhibit). | X | ||||||||
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
| CELLECTAR BIOSCIENCES, INC. | |
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Date: October 29, 2024 | By: | /s/ James V. Caruso |
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| James V. Caruso |
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| President and Chief Executive Officer |
(Principal Executive Officer) | ||
Date: October 29, 2024 | By: | /s/ Chad J. Kolean |
Chad J. Kolean | ||
Chief Financial Officer | ||
(Principal Financial and Accounting Officer) | ||
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