美國
證券交易委員會
華盛頓特區20549
表格
根據1934年證券交易法第13或15(d)條款的季度報告。 |
截至2024年6月30日季度結束
或
根據1934年證券交易法第13或15(d)條款的過渡報告 |
到 天從發票日期計算，被視為商業合理。 .
委員會檔案編號：
（依憑章程所載的完整登記名稱）
(依據所在地或其他管轄區) 的註冊地或組織地點) |
(國稅局雇主識別號碼) 識別號碼) |
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不適用 |
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(總部辦公地址) |
（郵政編碼） |
+
(註冊人電話號碼，包括區號)
根據法案第12(b)條規定註冊的證券：
每種類別的名稱 |
交易標的（s） |
每個註冊交易所的名稱 |
請勾選以下選項以表示申報人（1）已提交證券交易法1934年第13條或15(d)條所要求提交的所有報告，且在過去12個月中（或申報人需要提交此類報告的較短期間）已提交；（2）已受到過去90天內此類提交要求的限制。
請打勾號表明註冊人是否根據《S-t條例405條規定（本章節232.405號）的規定，在過去12個月內（或註冊人需要提交此類文件的更短期限內），已提交每個交互數據文件。
請檢視勾選，以指出登記者是否為大幅增加的存檔者、加速增長的存檔者、非加速增長的存檔者、較小的報告公司或新興成長公司。請查看《交易所法》第120條2的「大幅增加的存檔者」、「加速增長的存檔者」、「較小的報告公司」和「新興成長公司」的定義。
☒ |
加速歸檔人 |
☐ |
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非加速歸檔人 |
☐ |
小型報告公司 |
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新興成長型企業 |
如果是新興成長公司，請用勾選表示該註冊人已選擇不使用根據《交易所法》第13(a)條提供的任何新的或修訂的財務會計標準的擴展過渡期來遵守。 ☐
□標記是否申報人屬於貝殼公司（如《交易所法》第120億2條所定）。 是 ☐ 否
截至11月1日，2024年有
在本第10-Q表格的季度報告中，“公司”，“CRISPR”，“CRISPR Therapeutics”，“我們”，“我們的”和“我們的”，除非情境要求，均指CRISPR Therapeutics AG及其合併子公司; “我們的董事會”指的是CRISPR Therapeutics AG的董事會; 一般而言，我們將CASGEVY（exagamglogene autotemcel [exa-cel]，以前稱為CTX001）稱為“CASGEVY”。
“CRISPR Therapeutics®” 標準文字標記和設計標誌，“CRISPRX™,” “CRISPR TX™,” “CTX112™,” “CTX131™,” “CTX310™,” “CTX320™,” and “CTX211™” 是 crispr therapeutics ag 的商標和註冊商標。CASGEVY™ 文字標記和設計是福泰製藥的商標。所有其他商標和註冊商標 均屬於本季度10-Q表格中的各自所有者財產. 為了方便起見，在本季度10-Q表格中提到的商標、服務標記和商業名稱可能會出現不帶符號，這樣的省略並不意味著放棄任何此類權利。 ® 或 ™ 符號和任何這樣的遺漏均不是為了表明對任何此類權利的放棄。
前瞻性陳述
這份10-Q表格的季度報告包含涉及重大風險和不確定性的「前瞻性陳述」。所有陳述中，除了關於歷史事實的陳述外，在這份10-Q表格的季度報告內容包含的都是前瞻性陳述。這些陳述通常通過使用「預料」、「相信」、「繼續」、「可能」、「估計」、「期望」、「打算」、「可能」、「計劃」、「預測」、「項目」、「潛在」、「將會」、「將」這類字詞或這些字詞的否定形式或複數形式或類似表達方式來進行確定。這份10-Q表格的季度報告中的前瞻性陳述包括，但不限於，有關以下事項的陳述：
本季度報告表格10-Q中的任何前瞻性陳述均反映我們對未來事件或我們未來財務表現的觀點，涉及已知和未知風險、不確定性和假設，可能導致我們的實際結果和某些事件的時間與前瞻性陳述所暗示的未來結果有實質不同。可能導致或有助於產生這種差異的因素包括但不限於本季度報告表格10-Q第二部分第1A項中所確定的因素，以及在本季度報告表格10-Q中設定的“風險因素”部分中討論的因素，如有的話，我們於2024年2月21日向證券交易委員會（SEC）提交的年度報告表格10-K，以及其他SEC提交的文件。您不應該將前瞻性陳述作為未來事件的預測。這些前瞻性陳述僅於本報告日期發表。我們的前瞻性陳述不反映我們可能進行的任何未來收購、合併、處分、合資或投資的潛在影響。 反映我們目前對未來事件或我們未來財務表現的看法，並涉及已知和未知風險、不確定性和假設，可能導致我們的實際結果和某些事件的時間與前瞻性陳述所暗示的未來結果有實質不同。可能導致或有助於產生這種差異的因素包括但不限於本季度報告表格10-Q第二部分第1A項中所確定的因素，以及在本季度報告表格10-Q中設定的“風險因素”部分中討論的因素，如有的話，我們於2024年2月21日向證券交易委員會（SEC）提交的年度報告表格10-K，以及其他SEC提交的文件。您不應該將前瞻性陳述作為未來事件的預測。這些前瞻性陳述僅於本報告日期發表。我們的前瞻性陳述不反映我們可能進行的任何未來收購、合併、處分、合資或投資的潛在影響。
您應該完整閱讀本季度10-Q表格以及我們作為本季度10-Q報告附件提交的文件，並且要明白我們未來的實際結果、表現或成就可能與我們的預期大不相同。除法律要求外，我們不承擔任何更新前瞻性陳述以反映該等陳述日期之後事件或情況的義務。
投資者和其他人應該注意，我們通過我們的投資者關係網站（https://crisprtx.gcs-web.com/）、SEC文件、新聞稿、公開電話會議和網路研討會公佈重要信息。我們利用這些建渠道以及社交媒體與公眾溝通我們公司、業務、產品候選者和其他事項。我們發佈在社交媒體上的信息可能被視為重要信息。因此，我們鼓勵投資者、媒體和對我們公司感興趣的其他人查看我們在投資者關係網站上列出的社交媒體渠道上發佈的信息。
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第一部分—財務L INFORMATION
第一項。財務報表。財務報表
crispr therapeutics ag
綜合總表簡明總賬表
(未經審核，以千為單位，股份和每股數據除外)
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截至日期 |
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九月三十日， |
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12月31日， |
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2024 |
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2023 |
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資產 |
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流動資產: |
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現金及現金等價物 |
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有價證券 |
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應收帳款 |
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預付費用及其他流動資產 |
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全部流動資產 |
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物業及設備，扣除折舊後淨值 |
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非流動市場證券投資 |
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無形資產，扣除累計攤銷 |
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限制性現金 |
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經營租賃資產 |
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其他非流動資產 |
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資產總額 |
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負債及股東權益 |
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流動負債： |
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應付賬款 |
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$ |
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應計費用 |
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營業收入待確認收入，當期 |
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應付稅務負債 |
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營業租賃負債 |
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其他流動負債 |
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流動負債合計 |
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逾期收入，非流動資產 |
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扣除當期償還後之經營租賃負債淨額 |
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總負債 |
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股東權益： |
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普通股，瑞士法郎 |
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庫藏股，以成本計量， |
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資本公積額額外增資 |
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累積虧損 |
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其他綜合收益累計額 |
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股東權益總額 |
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負債總額及股東權益 |
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$ |
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附帶說明是這些未經審計的簡化合並財務報表的組成部分。
2
CRISPR Therapeutics AG
經過簡化的綜合損益表（未經審核） 運營和綜合虧損
（未審計，以千爲單位，除每股數據外）
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三個月已結束 |
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九個月已結束 |
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九月三十日 |
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九月三十日 |
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2024 |
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2023 |
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2024 |
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2023 |
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收入： |
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協作收入 |
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補助金收入 |
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總收入 |
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運營費用： |
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研究和開發 |
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一般和行政 |
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運營費用總額 |
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運營損失 |
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其他收入，淨額 |
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其他收入總額，淨額 |
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所得稅前淨虧損 |
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所得稅準備金 |
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淨虧損 |
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外幣折算調整 |
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有價證券的未實現收益 |
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綜合損失 |
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普通股每股淨虧損——基本 |
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$ |
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$ |
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$ |
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$ |
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已發行基本加權平均普通股 |
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普通股每股淨虧損——攤薄 |
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攤薄後的加權平均已發行普通股 |
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附帶說明是這些未經審計的簡化合並財務報表的組成部分。
3
CRISPR Therapeutics AG
彙編的並表報表 股東權益
（未審計，以千爲單位，除每股數據外）
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普通股份。 |
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庫藏股 |
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股份 |
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0.03瑞士法郎 |
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股份 |
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金額, |
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額外的 |
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累積的 |
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累積的 |
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總費用 |
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2022年12月31日結存餘額 |
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$ |
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$ |
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$ |
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$ |
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$ |
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$ |
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限制股股份解除限制 |
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行使已獲得的期權，扣除發行費用$ |
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購買普通股的ESPP計劃 |
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股票補償費用 |
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其他綜合收益 |
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淨虧損 |
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2023年3月31日的餘額 |
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$ |
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$ |
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$ |
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$ |
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$ |
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$ |
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限制股份的認購權 |
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行權已取得的期權，扣除發行成本 $ |
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股票補償費用 |
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其他綜合收益 |
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淨虧損 |
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— |
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2023年6月30日的餘額 |
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$ |
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$ |
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$ |
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$ |
( |
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$ |
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$ |
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限制股份的分配 |
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|
行使已獲期權 |
|
|
|
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||||
購買普通股的ESPP計劃 |
|
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
|||
股票補償費用 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||
其他綜合收益 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
|
||
淨虧損 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
( |
) |
|
— |
|
|
( |
) |
2023年9月30日結餘 |
|
|
$ |
|
|
|
$ |
( |
) |
$ |
|
$ |
( |
) |
$ |
( |
) |
$ |
|
|||||
4
|
普通股份。 |
|
庫藏股 |
|
|
|
|
|
|
|
|
|
||||||||||||
|
股份 |
|
0.03瑞士法郎 |
|
股份 |
|
金額, |
|
額外的 |
|
累積的 |
|
累積的 |
|
總費用 |
|
||||||||
2023年12月31日結餘爲 |
|
|
$ |
|
|
|
$ |
( |
) |
$ |
|
$ |
( |
) |
$ |
|
$ |
|
||||||
普通股發行淨額，減去發行成本$ |
|
|
|
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||||
限制股份的分配 |
|
|
|
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|||
行權已取得的期權，扣除發行成本 $ |
|
|
|
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||||
購買普通股的ESPP計劃 |
|
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
|||
股票補償費用 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||
其他綜合損失 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
( |
) |
|
( |
) |
淨虧損 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
( |
) |
|
— |
|
|
( |
) |
2024年3月31日結存餘額 |
|
|
$ |
|
|
|
$ |
( |
) |
$ |
|
$ |
( |
) |
$ |
( |
) |
$ |
|
|||||
限制股份的分配 |
|
|
|
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|||
行權已取得的期權，扣除發行成本 $ |
|
|
|
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||||
股票補償費用 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||
其他綜合損失 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
( |
) |
|
( |
) |
淨虧損 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
( |
) |
|
— |
|
|
( |
) |
2024年6月30日餘額 |
|
|
$ |
|
|
|
$ |
( |
) |
$ |
|
$ |
( |
) |
$ |
( |
) |
$ |
|
|||||
普通股發行 |
|
|
|
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||||
限制股份的分配 |
|
|
|
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|||
行使已獲期權 |
|
|
|
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||||
購買普通股的ESPP計劃 |
|
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
|||
股票補償費用 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
— |
|
|
— |
|
|
|
||
其他綜合收益 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
|
||
淨虧損 |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
( |
) |
|
— |
|
|
( |
) |
2024年9月30日的餘額 |
|
|
$ |
|
|
|
$ |
( |
) |
$ |
|
$ |
( |
) |
$ |
|
$ |
|
||||||
附帶說明是這些未經審計的簡化合並財務報表的組成部分。
5
CRISPR Therapeutics AG
壓縮的合併現金流量表現金流量表
（未經審計，以千爲單位）
|
|
截至9月30日的九個月 |
|
|||||
|
|
2024 |
|
|
2023 |
|
||
經營活動： |
|
|
|
|
|
|
||
淨虧損 |
|
$ |
( |
) |
|
$ |
( |
) |
淨虧損與經營活動使用現金的調節： |
|
|
|
|
|
|
||
折舊和攤銷 |
|
|
|
|
|
|
||
以股票爲基礎的補償 |
|
|
|
|
|
|
||
其他非現金項目，淨額 |
|
|
( |
) |
|
|
( |
) |
收購的未完成研發項目 |
|
|
|
|
|
|
||
變動情況： |
|
|
|
|
|
|
||
應收賬款 |
|
|
|
|
|
|
||
預付款項和其他資產 |
|
|
|
|
|
|
||
應付賬款及應計費用 |
|
|
( |
) |
|
|
( |
) |
遞延收入 |
|
|
( |
) |
|
|
|
|
營運租賃資產和負債 |
|
|
( |
) |
|
|
( |
) |
其他淨負債 |
|
|
( |
) |
|
|
( |
) |
經營活動使用的淨現金流量 |
|
|
( |
) |
|
|
( |
) |
投資活動： |
|
|
|
|
|
|
||
購買固定資產和設備 |
|
|
( |
) |
|
|
( |
) |
購買進行中的研究和開發 |
|
|
|
|
|
( |
) |
|
股權投資 |
|
|
( |
) |
|
|
|
|
購買有市場流通的證券 |
|
|
( |
) |
|
|
( |
) |
有價證券到期收益 |
|
|
|
|
|
|
||
投資活動的淨現金流量(使用)/提供的淨現金流量 |
|
|
( |
) |
|
|
|
|
籌資活動： |
|
|
|
|
|
|
||
發行普通股收到的款項，減去發行成本 |
|
|
|
|
|
|
||
行權期權和ESPP(員工股票購買計劃)認購款淨額扣除發行成本 |
|
|
|
|
|
|
||
籌資活動產生的現金淨額 |
|
|
|
|
|
|
||
匯率變動對現金的影響 |
|
|
|
|
|
|
||
現金減少或增加 |
|
|
( |
) |
|
|
|
|
期初現金、現金等價物及受限制的現金 |
|
|
|
|
|
|
||
期末現金、現金等價物及受限制的現金 |
|
$ |
|
|
$ |
|
||
補充披露的非現金投融資活動 |
|
|
|
|
|
|
||
應付賬款及應計費用中的財產和設備購買 |
|
$ |
|
|
$ |
|
||
股權發行成本包括應付賬款、應計費用及其他長期負債 |
|
$ |
|
|
$ |
|
||
|
|
截至2022年9月30日， |
|
|||||
協調至合併資產負債表中的金額 |
|
2024 |
|
|
2023 |
|
||
現金及現金等價物 |
|
$ |
|
|
$ |
|
||
受限現金 |
|
|
|
|
|
|
||
期末現金、現金等價物及受限制的現金餘額 |
|
$ |
|
|
$ |
|
||
所附附附註是這些未經審計的簡明合併財務報表不可分割的一部分。
6
CRISPR Therapeutics AG
基本報表附註 基本報表
(未經審計)
1. 報告的基礎和重要會計政策
報告範圍
附表中的簡明綜合財務報表未經審計，按照美國通用會計準則或U.S. GAAP由公司編制。
附表中簡明綜合財務報表包括公司及其全資子公司的賬目。所有公司間的餘額和交易在合併中已經予以消除。公司將其業務視爲一個經營板塊，在其中進行業務發現、開發和商業化，這一業務源自或融合基因編輯技術。公司的年度財務報表通常包括的一些信息及附註披露在這些中期財務報表中已經被精簡或省略。據管理層的意見，這些中期財務報表反映了爲公正呈現截至2024年和2023年9個月的財務狀況和經營業績所需的所有正常週期性調整。
其經營板塊的管理。.
使用估計
根據美國通用會計準則編制財務報表需要管理層進行估計和假設，這些估計和假設會影響財務報表及附註中報告的金額。 公司管理層會定期評估包括但不限於營業收入確認、基於股權的報酬費用以及期間內報告金額在內的估計。這些綜合財務報表中的重要估計涉及營業收入確認和基於股權的報酬費用。公司的估計依據歷史經驗和其他市場特定或其他相關假設，認爲在當時環境下是合理的。實際結果可能與這些估計或假設有所不同。 估計變動將在其知曉的期間反映在報告結果中。
重要會計政策
爲截至2024年9月30日的三個月和九個月期間編制這些簡明綜合財務報表所使用的重要會計政策與公司於2024年2月21日向SEC提交的2023年度10-K表格附註2中討論的相一致。
新會計準則-最近採納
從時間到時間，財務會計準則委員會或其他標準制定機構發佈新的會計準則，公司將在指定的生效日期採納。公司不認爲最近發佈的準則的採納對其簡明綜合財務報表和披露可能產生或可能產生重大影響。
7
2. Marketable Securities
The following table summarizes cash equivalents and marketable securities held at September 30, 2024 and December 31, 2023 (in thousands), which are recorded at fair value. The table below excludes $
|
|
Amortized |
|
|
Gross |
|
|
Gross |
|
|
Fair Value |
|
||||
September 30, 2024 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Certificates and term deposits |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Commercial paper |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total cash equivalents |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Marketable securities: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasury securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Corporate debt securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Certificates and term deposits |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Government-sponsored enterprise securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Commercial paper |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total marketable debt securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Corporate equity securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total marketable securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total cash equivalents and marketable securities |
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|||
December 31, 2023 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
U.S. Treasury securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total cash equivalents |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Marketable securities: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasury securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Corporate debt securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Certificates and term deposits |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Government-sponsored enterprise securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Commercial paper |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total marketable securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total cash equivalents and marketable securities |
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|||
As of September 30, 2024, marketable debt securities were in a net unrealized gain position of $
As of September 30, 2024 and December 31, 2023, the aggregate fair value of marketable securities that were in an unrealized loss position for less than twelve months was $
The Company determined that there is no material credit risk associated with the above investments as of September 30, 2024.
8
The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable securities for the three and nine months ended September 30, 2024 and 2023.
3. Fair Value Measurements
The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of September 30, 2024 and December 31, 2023 (in thousands):
|
|
Fair Value Measurements at |
|
|||||||||||||
|
|
September 30, 2024 |
|
|||||||||||||
|
|
Total |
|
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
||||
Cash and cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
||
Money market funds |
|
|
|
|
|
|
|
|
— |
|
|
|
— |
|
||
Certificates and term deposits |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Commercial paper |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Marketable securities: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasury securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Corporate debt securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Certificates and term deposits |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Government-sponsored enterprise securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Commercial paper |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Corporate equity securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Total |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
|
|
Fair Value Measurements at |
|
|||||||||||||
|
|
December 31, 2023 |
|
|||||||||||||
|
|
Total |
|
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
||||
Cash and cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
||
Money market funds |
|
|
|
|
|
|
|
|
— |
|
|
|
— |
|
||
U.S. Treasury securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Marketable securities: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasury securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Corporate debt securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Certificates and term deposits |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Government-sponsored enterprise securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Commercial paper |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Total |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
— |
|
|||
Marketable securities classified as Level 2 within the valuation hierarchy generally consist of U.S. treasury securities and government agency securities, certificates of deposit, corporate bonds and commercial paper. The Company estimates the fair value of these marketable securities by taking into consideration valuations obtained from third-party pricing sources.
9
4. Property and Equipment, net
Property and equipment, net, consists of the following (in thousands):
|
|
As of |
|
|||||
|
|
September 30, |
|
|
December 31, |
|
||
|
|
2024 |
|
|
2023 |
|
||
Computer equipment |
|
$ |
|
|
$ |
|
||
Furniture, fixtures and other |
|
|
|
|
|
|
||
Laboratory equipment |
|
|
|
|
|
|
||
Leasehold improvements |
|
|
|
|
|
|
||
Construction work in process |
|
|
|
|
|
|
||
Total property and equipment, gross |
|
|
|
|
|
|
||
Accumulated depreciation |
|
|
( |
) |
|
|
( |
) |
Total property and equipment, net |
|
$ |
|
|
$ |
|
||
Depreciation expense for the three and nine months ended September 30, 2024 was $
5. Accrued Expenses
Accrued expenses consist of the following (in thousands):
|
|
As of |
|
|||||
|
|
September 30, |
|
|
December 31, |
|
||
|
|
2024 |
|
|
2023 |
|
||
Payroll and employee-related costs |
|
$ |
|
|
$ |
|
||
Research and development costs |
|
|
|
|
|
|
||
Collaboration costs |
|
|
|
|
|
|
||
Licensing fees |
|
|
|
|
|
|
||
Professional fees |
|
|
|
|
|
|
||
Intellectual property costs |
|
|
|
|
|
|
||
Accrued property and equipment |
|
|
|
|
|
|
||
Other |
|
|
|
|
|
|
||
Total |
|
$ |
|
|
$ |
|
||
6. Significant Contracts
Agreements with Vertex
2015 collaboration
In 2015, the Company entered into a strategic collaboration, option and license agreement, or the 2015 Collaboration Agreement, with Vertex Pharmaceuticals Incorporated, or Vertex. The 2015 Collaboration Agreement is focused on the use of the Company’s CRISPR/Cas9 gene editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. The Company and Vertex amended the 2015 Collaboration Agreement in 2017 and 2019 with Amendment No. 1 and Amendment No. 2, respectively, namely to clarify Vertex’s option rights under the 2015 Collaboration Agreement and to modify certain definitions and provisions of the 2015 Collaboration Agreement to make them consistent with the JDA (as defined below) and the 2019 Collaboration Agreement (as defined below). In 2017, Vertex exercised an option granted to it under the 2015 Collaboration Agreement to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, and in 2019, Vertex exercised the remaining options granted to it under the 2015 Collaboration Agreement to exclusively license certain collaboration targets developed under the 2015 Collaboration Agreement.
Hemoglobinopathies collaboration
In 2017, following Vertex's exercise of its option to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, the Company and Vertex entered into a joint development and commercialization agreement, or the JDA, and agreed for potential hemoglobinopathy treatments, including CASGEVY, the Company and Vertex would share equally all research and development costs and worldwide revenues. In 2021, the Company and Vertex amended and restated the JDA, or the A&R Vertex JDCA (as amended and in effect, from time to time), pursuant to which the parties agreed to, among
10
other things, (a) adjust the governance structure for the collaboration and adjust the responsibilities of each party thereunder, whereby Vertex leads and has all decision making (i.e., control) in relation to the CASGEVY program prospectively; (b) adjust the allocation of net profits and net losses between the parties with respect to CASGEVY only, which will be allocated
In connection with the closing of the transaction contemplated by the A&R Vertex JDCA, the Company received a $
Letter Agreement
In May 2024, Vertex and the Company entered into a letter agreement, or the Letter Agreement, with respect to the priority review voucher issued by the FDA to Vertex as the sponsor of the rare pediatric disease product application for CASGEVY. Vertex and the Company agreed that if Vertex utilizes or transfers the priority review voucher prior to the first calendar year in which the CASGEVY program generates a net profit, Vertex will pay the Company $
DMD and DM1 exclusive license
In 2019, the Company and Vertex entered into a series of agreements, including a strategic collaboration and license agreement, or the 2019 Collaboration Agreement, for the development and commercialization of products for the treatment of Duchenne muscular dystrophy, or DMD, and myotonic dystrophy Type 1, or DM1. For the DMD and DM1 programs, Vertex is responsible for all research, development, manufacturing and commercialization activities and all related costs. Upon investigational new drug, or IND, filing, the Company has the option to forego the DM1 milestones and royalties, and instead, co-develop and co-commercialize all DM1 products globally in exchange for payment of
Collaboration in the field of diabetes
In 2021, the Company and ViaCyte, Inc., or ViaCyte, entered into a joint development and commercialization agreement, or the ViaCyte JDCA, to jointly develop and commercialize product candidates and shared products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. In the third quarter of 2022, Vertex acquired ViaCyte, and ViaCyte became a wholly-owned subsidiary of Vertex. In March 2023, (1) the Company and ViaCyte entered into an amendment to the ViaCyte JDCA, or the ViaCyte JDCA Amendment, and adjusted certain rights and obligations of the Company and ViaCyte under the ViaCyte JDCA, and (2) the Company and Vertex entered into a non-exclusive license agreement, or the Non-Ex License Agreement, pursuant to which the Company agreed to license to Vertex, on a non-exclusive basis, certain of its gene editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. Subsequently, ViaCyte elected to opt-out of the ViaCyte JDCA. Per the opt-out terms, the on-going collaboration assets are now wholly owned by the Company, subject to a royalty on future sales owed to ViaCyte. The opt-out became effective in early February 2024.
In connection with entering into the Non-Ex License Agreement, the Company received a $
11
Accounting Analysis
For purposes of this Note 6, the 2015 Collaboration Agreement, Amendment No. 1, Amendment No. 2, A&R Vertex JDCA, Amendment No. 1 to the A&R Vertex JDCA and 2019 Collaboration Agreement are collectively referred to as the “Vertex Agreements” and the Non-Ex License Agreement and ViaCyte JDCA Amendment are collectively referred to as the “March 2023 Agreements.”
The Vertex Agreements and the March 2023 Agreements include components of a customer-vendor relationship as defined under ASC 606, Revenue from Contracts with Customers, or ASC 606, collaborative arrangements as defined under ASC 808, Collaborative Agreements, or ASC 808, and research and development costs as defined under ASC 730, Research and Development, or ASC 730. Specifically, with regards to the March 2023 Agreements, the Company concluded that the non-exclusive license is a performance obligation under ASC 606 and the ongoing research and development services under the ViaCyte JDCA Amendment are a unit of account under ASC 808.
The Company has determined that recognition criteria for the Letter Agreement has not been met and will not be met until the priority review voucher is (i) utilized or (ii) there is sufficient profitability such that Vertex is obligated to pay the Company under the Letter Agreement.
Accounting Analysis Under ASC 606
March 2023 Agreements
Identification of the Contract
The March 2023 Agreements were negotiated as a package with a single commercial objective and, as such, the March 2023 Agreements were combined for accounting purposes and treated as a single arrangement. The Company determined for accounting purposes that the combined contract terminates the original ViaCyte JDCA and created a new contract.
Identification of Performance Obligations
The Company concluded the transfer of the non-exclusive license, including certain modified rights and obligations provided as part of the ViaCyte JDCA Amendment to support the delivery of the license, was both capable of being distinct and distinct within the context of the contract.
Determination of Transaction Price
The initial transaction price was comprised of the upfront payment of $
In the second quarter of 2023, the Company adjusted the transaction price to include $
Allocation of Transaction Price to Performance Obligations
The Company identified one performance obligation for the March 2023 Agreements and, as a result, no allocation of the transaction price was required.
Recognition of Revenue
The Company determined the non-exclusive license, including certain modified rights and obligations provided as part of the ViaCyte JDCA Amendment to support the delivery of the license, represented functional intellectual property, as the intellectual property provides Vertex with the ability to perform a function or task in the form of research and development in the field of diabetes. In 2023, the Company recognized revenue of $
In 2023, revenue from variable consideration of $
12
Milestones under the Non-Ex License Agreement
As of September 30, 2024, the Company is eligible to receive potential future milestone payments from Vertex of up to $
Each of the remaining milestones under the Non-Ex License Agreement are fully constrained as of September 30, 2024. There is uncertainty as to whether the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.
Vertex Agreements
Deferred revenue
As of September 30, 2024 and December 31, 2023, there was
Milestones
The Company has evaluated the milestones that may be received in connection with the Vertex Agreements.
Under the 2015 Collaboration Agreement and subsequent amendments, the Company is eligible to receive up to $
The Company is eligible to receive potential future payments of up to $
The Company has the option to conduct research at its own cost in certain defined areas. If such research is beneficial to the CASGEVY program and CASGEVY ultimately achieves regulatory approval in such areas, the Company could be entitled to receive from Vertex certain milestone payments aggregating to high eight digits.
Each of the remaining milestones described above are fully constrained as of September 30, 2024. There is uncertainty that the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.
Accounting Analysis under ASC 808
Vertex Agreements
In connection with the Vertex Agreements, the Company identified the following collaborative elements, which are accounted for under ASC 808: (i) development and commercialization services for shared products, including any transition services related to CASGEVY under the A&R Vertex JDCA; (ii) R&D Services for follow-on products; and (iii) committee participation. The related impact of the cost sharing is included within collaboration expense, net, in the condensed consolidated statements of operations and comprehensive loss. During the three and nine months ended September 30, 2024, the Company recognized $
13
During the three and nine months ended September 30, 2023, the Company recognized $
7. Commitments and Contingencies
Leases
Refer to Note 7 to the consolidated financial statements in the Company’s 2023 Annual Report on Form 10-K filed with the SEC on February 21, 2024 for discussion of the Company’s lease arrangements.
Litigation
In the ordinary course of business, the Company is from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, the Company’s intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office involving the Company’s intellectual property estate including certain in-licensed intellectual property. The outcome of any of the foregoing, regardless of the merits, is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of Company’s management and other resources that would otherwise be engaged in other activities. If the Company is unable to prevail in any such proceedings, the Company’s business, results of operations, liquidity and financial condition could be adversely affected.
Letters of Credit
As of September 30, 2024, the Company had restricted cash of $
Research, Manufacturing, License and Intellectual Property Agreements
The Company has engaged several research institutions and companies to identify new delivery strategies and applications of the Company’s gene editing technology. The Company is also a party to a number of license agreements which require significant upfront payments and may be required to make future royalty payments and potential milestone payments from time to time. In addition, the Company is also a party to intellectual property agreements, which require maintenance and milestone payments from time to time. Further, the Company is a party to a number of manufacturing agreements that require upfront payments for the future performance of services.
In association with these agreements, on a product-by-product basis, the counterparties are eligible to receive up to low eight-digit potential payments upon specified research, development and regulatory milestones. In addition, on a product-by-product basis, the counterparties are eligible to receive potential commercial milestone payments based on specified annual sales thresholds. The potential payments are low-single digit percentages of the specified annual sales thresholds. The counterparties are also eligible to receive low single-digit royalties on future net sales.
Under certain circumstances and if certain contingent future events occur, Vertex is eligible to receive up to $
Under the A&R Vertex JDCA, the Company has an option to defer specified costs on the CASGEVY program in excess of $
In the third quarter of 2024, the Company exercised its option to defer specified costs on the CASGEVY program in excess of the deferral limit under the A&R Vertex JDCA, as amended. As of September 30, 2024, the Company has deferred $
14
which resulted in a deferral of $
8. Share Capital
All of the Company's common shares are authorized under Swiss corporate law with a nominal value of
As of September 30, 2024, the Company's share capital consists of
Common Share Issuances
At-the-Market Offering
In August 2019, the Company entered into an Open Market Sale AgreementSM with Jefferies LLC, or Jefferies, under which the Company was able to offer and sell, from time to time at its sole discretion through Jefferies, as its sales agent, its common shares, or the August 2019 Sales Agreement.
In January 2021, in connection with the August 2019 Sales Agreement, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $
As of September 30, 2024, the Company has issued and sold an aggregate of
Registered Direct Offering
In February 2024, the Company entered into an investment agreement for the sale of approximately $
9. Stock-based Compensation
During the three and nine months ended September 30, 2024 and 2023, the Company recognized the following stock-based compensation expense (in thousands):
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
Research and development |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
General and administrative |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
15
Stock option activity
The following table summarizes stock option activity for the nine months ended September 30, 2024:
|
|
Shares |
|
|
Weighted- |
|
||
Outstanding at December 31, 2023 |
|
|
|
|
$ |
|
||
Granted |
|
|
|
|
|
|
||
Exercised |
|
|
( |
) |
|
|
|
|
Cancelled or forfeited |
|
|
( |
) |
|
|
|
|
Outstanding at September 30, 2024 |
|
|
|
|
$ |
|
||
Exercisable at September 30, 2024 |
|
|
|
|
$ |
|
||
Vested and expected to vest at September 30, 2024 |
|
|
|
|
$ |
|
||
As of September 30, 2024, total unrecognized compensation expense related to stock options was $
Restricted stock activity
The following table summarizes restricted stock activity for the nine months ended September 30, 2024:
|
|
Shares |
|
|
Weighted- |
|
||
Unvested balance at December 31, 2023 |
|
|
|
|
$ |
|
||
Granted |
|
|
|
|
|
|
||
Vested |
|
|
( |
) |
|
|
|
|
Cancelled or forfeited |
|
|
( |
) |
|
|
|
|
Unvested balance at September 30, 2024 |
|
|
|
|
$ |
|
||
10. Net Loss Per Share Attributable to Common Shareholders
Basic net loss per share is calculated by dividing net loss attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net loss per share is calculated by dividing the net loss attributable to common shareholders by the weighted-average number of common share equivalents outstanding for the period, including any dilutive effect from outstanding stock options and warrants using the treasury stock method. The Company’s net loss is net loss attributable to common shareholders for all periods presented.
The following common stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect:
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
Outstanding options |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Unvested restricted common shares |
|
|
|
|
|
|
|
|
|
|
|
|
||||
ESPP |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total |
|
|
|
|
|
|
|
|
|
|
|
|
||||
16
11. Income Taxes
17
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with (i) our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and (ii) our audited consolidated financial statements and related notes and management’s discussion and analysis of financial condition and results of operations included in our Annual Report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission, or the SEC, on February 21, 2024. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and impact and potential impacts on our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including, without limitation, those factors set forth in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2023 and the “Risk Factors” section of subsequent Quarterly Reports on Form 10-Q, our actual results or timing of certain events could differ materially from the results or timing described in, or implied by, these forward-looking statements.
Overview
We are a leading gene editing company focused on the development of CRISPR/Cas9-based therapeutics. CRISPR/Cas9 is a revolutionary technology for gene editing, the process of precisely altering specific sequences of genomic DNA. We aim to apply this technology to disrupt, delete, correct and insert genes to treat diseases and to engineer advanced cellular therapies. We have advanced this technology from discovery to an approved medicine with unparalleled speed, culminating in the landmark first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023 with our collaborators at Vertex Pharmaceuticals Incorporated, or Vertex. We believe that the combination of our technology, research and development capabilities, and proven ability to execute may enable us to create an entirely new class of highly effective and potentially curative therapies for patients with both rare and common diseases for whom current biopharmaceutical approaches have had limited success.
We have established a portfolio of therapeutic programs spanning four core franchises: hemoglobinopathies, immuno-oncology and autoimmune, in vivo approaches and type 1 diabetes. Our most advanced program, CASGEVY, has received approval in the United States and other countries for the treatment of eligible patients with severe sickle cell disease, or SCD, or transfusion-dependent beta thalassemia, or TDT, two genetic disorders of hemoglobin, or hemoglobinopathies, with high unmet medical need. In addition, we have further research efforts on targeted conditioning and in vivo editing of hematopoietic stem cells that have the potential to expand the number of patients that could benefit significantly. We are also progressing multiple next-generation gene-edited cell therapy programs, including allogeneic chimeric antigen receptor T cell, or CAR T, candidates for the treatment of hematological and solid tumor cancers and autoimmune diseases. In addition, we are advancing a portfolio of programs leveraging in vivo editing for both common and rare diseases, starting with the treatment and prevention of cardiovascular disease. Further, we have multiple parallel efforts using allogeneic, gene-edited, hypoimmune, stem cell-derived beta cells to address type 1 diabetes, or T1D, without the need for chronic immunosuppression.
Hemoglobinopathies
CASGEVY
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin in red blood cells, which can compensate for the defective adult hemoglobin in patients with SCD and TDT. CASGEVY is the first therapy to emerge from our strategic partnership with Vertex and is being advanced under a joint development and commercialization agreement between us and Vertex and certain of its affiliates.
In 2023, CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in the world. To date, CASGEVY has been approved in the United States, European Union, Great Britain, Canada, Switzerland, Kingdom of Saudi Arabia and Kingdom of Bahrain for the treatment of eligible patients 12 years and older with SCD or TDT. We and Vertex continue to investigate CASGEVY, including (1) three clinical trials designed to assess the safety and efficacy of a single dose of CASGEVY in patients 12 to 35 years of age with severe SCD and TDT, respectively, (2) two clinical trials in patients 5 to 11 years of age, one in severe SCD and a second in TDT, and (3) long-term follow-up clinical trials designed to follow participants for up to 15 years after CASGEVY infusion. CASGEVY safety data presented to date is generally consistent with an autologous stem cell transplant and myeloablative conditioning. Efficacy data presented to date support the profile of this therapy as a potential one-time functional cure for people with severe SCD and TDT.
Additional candidates
Building upon CASGEVY, we have next-generation efforts in targeted conditioning and in vivo editing of hematopoietic stem cells, either of which could broaden the number of patients that could benefit from our hemoglobinopathies product candidates.
18
Immuno-Oncology and Autoimmune
We believe CRISPR/Cas9 has the potential to create the next generation of CAR T cell therapies that may have a superior product profile and allow broader patient access compared to current autologous therapies. We are advancing several cell therapy programs for oncology and/or autoimmune indications, including two next-generation allogeneic CAR T programs, CTX112 targeting Cluster of Differentiation 19, or CD19, and CTX131 targeting Cluster of Differentiation 70, or CD70. These product candidates incorporate two novel gene edits—knock-out of Regnase-1 and knock-out of transforming growth factor-beta receptor type 2, or TGFBR2—designed to enhance CAR T potency and reduce CAR T exhaustion. Emerging pharmacology data, including pharmacokinetics, from ongoing clinical trials of CTX112 and CTX131, indicate that the novel potency gene edits lead to significantly higher CAR T cell expansion and functional persistence in patients compared to our first-generation candidates that did not incorporate these edits. In addition, the next-generation candidates exhibit increased manufacturing robustness, with a higher and more consistent number of CAR T cells produced per batch. We are producing CTX112 and CTX131 for clinical trials at our internal GMP manufacturing facility.
CD19 Candidates
CTX112 is being developed for both oncology and autoimmune indications. It is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the candidate in adult patients with relapsed or refractory CD19-positive B-cell malignancies who have received at least two prior lines of therapy, as well as an ongoing clinical trial in adult patients with systemic lupus erythematosus. Early clinical studies conducted by third parties have shown that CD19-directed autologous CAR T therapy can produce long-lasting remissions in multiple autoimmune indications by deeply depleting B cells. Our first generation allogeneic CD19-directed CAR T program has demonstrated effective depletion of B cells in oncology settings, which supports the potential for CTX112 in autoimmune diseases.
CD70 Candidates
CTX131 is being developed for both solid tumors and hematologic malignancies, including T cell lymphomas, or TCL. It is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the candidate in adult patients with relapsed or refractory solid tumors, as well as in hematologic malignancies, including TCL. We believe allogeneic CAR T approaches for TCL may have greater potential to meet the unmet need in this patient population given the patients’ own T cells are not suitable for autologous manufacturing.
Additional candidates
Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR T product candidates.
In Vivo
Our in vivo gene editing strategy focuses on gene disruption and whole gene correction – the two technologies required to address the vast majority of the most prevalent severe monogenic diseases as well as many common diseases. We have established a leading platform for in vivo gene editing and are rapidly advancing a broad portfolio of in vivo programs. Our first in vivo programs target the liver, taking advantage of validated lipid nanoparticle, or LNP, delivery technologies, and aim to treat diseases where we can produce a strong therapeutic effect by safely disrupting a gene with well-understood genetic association
Cardiovascular disease
Our first two in vivo programs utilizing our proprietary LNP platform, CTX310 and CTX320, are directed towards validated therapeutic targets associated with cardiovascular disease. CTX310 is being investigated in an ongoing clinical trial targeting ANGPTL3 in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia. Natural loss-of-function mutations in ANGPTL3 are associated with reduced low-density lipoprotein, triglycerides and atherosclerotic cardiovascular disease risk without any negative impact on overall health. In addition, CTX320 is being investigated in an ongoing clinical trial targeting LPA, the gene encoding apo(a), a critical component of lipoprotein(a), or Lp(a), in patients with elevated Lp(a), which has shown to have an independent association with major adverse cardiovascular events. Up to 20% of the global population has elevated Lp(a) levels.
Additional candidates
Building upon CTX310 and CTX320, we have a number of earlier stage investigational in vivo programs leveraging gene disruption in the liver for both rare and common diseases. In addition, we have programs focused on gene correction in the liver, including programs leveraging technologies developed by our CRISPR-X research team. Finally, we are pursuing additional delivery technologies, including further advancements to nanoparticle technology and adeno-associated virus, or AAV, vectors, for delivery to tissues beyond the liver, including hematopoietic stem cells.
19
Type 1 Diabetes
We are developing gene-edited stem cell-derived therapies for the treatment of T1D. We believe our gene editing capabilities have the potential to enable a beta-cell replacement product candidate that may deliver durable benefit to patients without the need for long-term immunosuppression. We have three parallel efforts to achieve this goal. First, our most advanced product candidate, CTX211, is an allogeneic, gene-edited, hypoimmune, stem cell derived product candidate in a device that is implanted into patients and intended to produce insulin in a glucose-dependent manner. This program, formerly known as VCTX211, originated from our collaboration with ViaCyte, Inc., or ViaCyte, a subsidiary of Vertex, and was developed by applying our gene editing technology to ViaCyte’s proprietary stem cell capabilities. CTX211 is being investigated in an ongoing Phase 1/2 clinical trial designed to assess the safety, tolerability and efficacy of CTX211 in adult patients with T1D. Second, we have research efforts focused on a deviceless beta cell replacement approach consisting of unencapsulated beta cells derived from edited stem cells. Third, we have granted a non-exclusive license to certain of our CRISPR/Cas9 intellectual property to Vertex to accelerate Vertex’s development of hypoimmune cell therapies for T1D, for which we received $170 million in upfront and milestone payments in 2023 and remain eligible to receive additional research and development milestones and royalties on future products.
CRISPR-X
While we have made significant progress with our current portfolio of programs, we recognize that we need to continue to innovate to unlock the full power of gene editing and bring potentially transformative therapies to even more patients. We have a dedicated early-stage research team called CRISPR-X that focuses on innovating next-generation editing modalities. CRISPR-X is developing technologies to enable whole gene correction and insertion without requiring homology-directed repair or viral delivery of DNA, such as all-RNA gene correction, non-viral delivery of DNA and novel gene insertion techniques.
Partnerships
Given the numerous potential therapeutic applications for CRISPR/Cas9, we have partnered strategically to broaden the indications we can pursue and accelerate development of programs by accessing specific technologies and/or disease-area expertise. We maintain broad partnerships to develop gene editing-based therapeutics in specific disease areas.
Vertex. We established our initial collaboration agreement in 2015 with Vertex, which focused on TDT, SCD, cystic fibrosis and select additional indications. In December 2017, we entered into a joint development and commercialization agreement with Vertex pursuant to which, among other things, we are co-developing and co-commercializing CASGEVY for TDT and SCD. In April 2021, we and Vertex amended and restated our existing joint development and commercialization agreement, pursuant to which, among other things, we will continue to develop and commercialize CASGEVY for TDT and SCD in partnership with Vertex. We also entered into a strategic collaboration and license agreement with Vertex in June 2019 for the development and commercialization of products for the treatment of Duchenne muscular dystrophy, or DMD, and myotonic dystrophy type 1, or DM1 and, in March 2023, we entered into a non-exclusive license agreement with Vertex for Vertex to utilize our gene editing technology in diabetes.
ViaCyte. We entered into a research and collaboration agreement in September 2018 with ViaCyte to pursue the discovery, development and commercialization of gene-edited allogeneic stem cell therapies for the treatment of diabetes, and in July 2021, we entered into a joint development and commercialization agreement with ViaCyte, or the ViaCyte JDCA. In connection with entering into the ViaCyte JDCA, our existing research collaboration agreement with ViaCyte expired in accordance with its terms. In the third quarter of 2022, Vertex announced it had acquired ViaCyte and ViaCyte’s rights to jointly develop and commercialize product candidates and shared products for use in the treatment of diabetes type 1, diabetes type 2 and insulin dependent/requiring diabetes throughout the world, and in March 2023, we entered into an amendment to the ViaCyte JDCA pursuant to which, among other things, we adjusted certain rights and obligations of the parties thereunder. In December 2023, ViaCyte elected to opt-out of the collaboration with us for the co-development and co-commercialization of gene-edited stem cell therapies for the treatment of diabetes. Per the opt-out terms, once the opt-out is complete, the on-going collaboration assets will be wholly owned by us, subject to a royalty on future sales owed to ViaCyte. The opt-out became effective in early February 2024. The ViaCyte collaboration assets include CTX211 (formerly VCTX211), an allogeneic, gene-edited, hypoimmune, stem cell derived product candidate in a device that is implanted into patients and intended to produce insulin in a glucose-dependent manner. A Phase 1 clinical trial for CTX211 for the treatment of T1D is ongoing.
Bayer. We entered into an option agreement in the fourth quarter of 2019 with Bayer pursuant to which Bayer has an option to co-develop and co-commercialize two products that we advance for the diagnosis, treatment, or prevention of certain autoimmune disorders, eye disorders, or hemophilia A disorders for a specified period of time, or, under certain circumstances, exclusively license such optioned products.
Other Partnerships. We have entered into a number of additional collaborations and license agreements to support and complement our hematopoietic stem cell, immuno-oncology and auto-immune, in vivo and T1D programs and platform, including agreements with: Nkarta, Inc. to develop and commercialize products leveraging donor-derived, gene-edited CAR-NK cells; Capsida Biotherapeutics, Inc. to develop in vivo gene editing therapies delivered with engineered AAV vectors; Roswell Park Comprehensive
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Cancer Center to advance a gene-edited autologous CAR T program against a new target; MaxCyte, Inc. on ex vivo delivery for our hemoglobinopathy and immuno-oncology programs; CureVac AG on optimized mRNA constructs and manufacturing for certain in vivo programs; and KSQ Therapeutics, Inc. on intellectual property for our allogeneic immuno-oncology programs.
Financial Overview
Since our inception in October 2013, we have devoted substantially all of our resources to our research and development efforts, identifying potential product candidates, undertaking drug discovery and preclinical development activities, building and protecting our intellectual property estate, establishing internal manufacturing capabilities, organizing and staffing our company, business planning, raising capital and providing general and administrative support for these operations. To date, we have primarily financed our operations through private placements of our preferred shares, common share issuances, convertible loans and license and collaboration agreements with strategic partners.
While we were in a net income position in certain previous years due to certain payments associated with our collaborations with Vertex, we have a history of recurring losses and expect to continue to incur losses for the foreseeable future. Our net losses may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase significantly as we continue our current research programs and development activities; seek to identify additional research programs and additional product candidates; conduct initial drug application supporting preclinical studies and initiate clinical trials for our product candidates; initiate preclinical testing and clinical trials for any other product candidates we identify and develop; seek regulatory approval for our product candidates; maintain, defend, protect and expand our intellectual property estate; further develop our gene editing platform; hire additional research, clinical and scientific personnel; incur facilities costs associated with such personnel growth; develop manufacturing infrastructure; and incur additional costs associated with operating as a public company.
Revenue Recognition
We have not generated any revenue to date from product sales and do not expect to do so in the near future. Revenue recognized for the three and nine months ended September 30, 2024 was not material. Revenue recognized for the nine months ended September 30, 2023 was $170.0 million related to our receipt of an upfront payment from Vertex in connection with entering into agreements with Vertex and ViaCyte relating to the research, development, manufacture and commercialization of therapeutic products in the diabetes field in the first quarter of 2023, as well as revenue recognized in the second quarter of 2023 related to a research milestone achieved in the second quarter of 2023. There was no revenue recognized for the three months ended September 30, 2023. For additional information about our revenue recognition policy, see Note 2, “Summary of Significant Accounting Policies,” in our Annual Report on Form 10-K for the year ended December 31, 2023 filed with the SEC on February 21, 2024, as well as Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
Research and Development Expenses
Research and development expenses consist primarily of costs incurred for our research activities, including our product discovery efforts and the development of our product candidates, which include:
Our external research and development expenses support our various preclinical and clinical programs, and, as such, we do not break down external research and development expenses further. Our internal research and development expenses consist of payroll and benefits expenses, facilities expense, and other indirect research and development expenses incurred in support of overall research and development activities and, as such, are not allocated to a specific development stage or therapeutic area. Research and development costs are expensed as incurred. Nonrefundable advance payments for research and development goods or services to be received in the future are deferred and capitalized. The capitalized amounts are expensed as the related goods are delivered or the services are performed. At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of any product candidates we may identify and develop. This is due to the numerous risks and uncertainties associated with developing such product candidates, including the uncertainty of:
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A change in the outcome of any of these variables with respect to the development of any product candidates or the subsequent commercialization of any product candidates we may successfully develop could significantly change the costs, timing and viability associated with the development of that product candidate.
Research and development activities are central to our business model. We expect to continue to incur research and development costs consistent with research and development at companies of our size and stage of development, which may increase in the foreseeable future as our current development programs progress, new programs are added and we continue to prepare regulatory filings. These increases will likely include the costs related to the implementation and expansion of clinical trial sites and related patient enrollment, monitoring, program management and manufacturing expenses for current and future clinical trials.
General and Administrative Expenses
General and administrative expenses consist primarily of employee related expenses, including salaries, benefits and equity-based compensation, for personnel in executive, finance, accounting, business development, human resources and other general and administrative functions. Other significant costs include facility costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters and fees for accounting and consulting services.
We expect to continue to incur general and administrative expenses consistent with general and administrative functions at research and development companies of our size and stage of development, which may increase in the future to support continued research and development activities, and potential commercialization of our product candidates. In addition, we anticipate ongoing expenses related to the reimbursements of third-party patent related expenses in connection with certain of our in-licensed intellectual property.
Collaboration Expense, Net
Collaboration expense, net, consists of operating expense related to the CASGEVY program under our collaboration with Vertex. Under the A&R Vertex JDCA, we have an option to defer our portion of specified costs on the CASGEVY program in excess of $110.3 million for the years ended December 31, 2022, 2023 and 2024. In the third quarter of 2024, we exercised our option to defer our portion of specified costs incurred in 2024 for the CASGEVY program in excess of $110.3 million. Any deferred amounts are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum amount per year. Additionally, we are permitted, under certain specified circumstances as set forth in Amendment No. 1 to the A&R Vertex JDCA, to adjust the timing of and portion of our share of costs we are permitted to defer under the agreement.
Other Income (Expense), Net
Other income (expense), net consists primarily of interest income earned on investments.
22
Results of Operations
Comparison of three months ended September 30, 2024 and 2023 (in thousands):
|
|
Three Months Ended September 30, |
|
|
Period to Period |
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
Revenue: |
|
|
|
|
|
|
|
|
|
|||
Collaboration revenue |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
— |
|
Grant revenue |
|
|
602 |
|
|
|
— |
|
|
|
602 |
|
Total revenue |
|
|
602 |
|
|
|
— |
|
|
|
602 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|||
Research and development |
|
|
82,160 |
|
|
|
90,698 |
|
|
|
(8,538 |
) |
General and administrative |
|
|
17,419 |
|
|
|
18,291 |
|
|
|
(872 |
) |
Collaboration expense, net |
|
|
11,153 |
|
|
|
23,422 |
|
|
|
(12,269 |
) |
Total operating expenses |
|
|
110,732 |
|
|
|
132,411 |
|
|
|
(21,679 |
) |
Loss from operations |
|
|
(110,130 |
) |
|
|
(132,411 |
) |
|
|
22,281 |
|
Other income, net |
|
|
25,064 |
|
|
|
20,671 |
|
|
|
4,393 |
|
Loss before income taxes |
|
|
(85,066 |
) |
|
|
(111,740 |
) |
|
|
26,674 |
|
Provision for income taxes |
|
|
(876 |
) |
|
|
(412 |
) |
|
|
(464 |
) |
Net loss |
|
$ |
(85,942 |
) |
|
$ |
(112,152 |
) |
|
$ |
26,210 |
|
Collaboration Revenue
There was no collaboration revenue for the three months ended September 30, 2024 and 2023. Please refer to Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.
Research and Development Expenses
Research and development expenses were $82.2 million for the three months ended September 30, 2024, compared to $90.7 million for the three months ended September 30, 2023. The following table summarizes our research and development expenses for the three months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands):
|
|
Three Months Ended September 30, |
|
|
Period to Period |
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
External research and development expenses |
|
$ |
25,375 |
|
|
$ |
30,667 |
|
|
$ |
(5,292 |
) |
Employee related expenses |
|
|
18,443 |
|
|
|
20,016 |
|
|
|
(1,573 |
) |
Facility expenses |
|
|
25,651 |
|
|
|
25,656 |
|
|
|
(5 |
) |
Stock-based compensation expenses |
|
|
11,949 |
|
|
|
11,287 |
|
|
|
662 |
|
Other expenses |
|
|
607 |
|
|
|
733 |
|
|
|
(126 |
) |
Sublicense and license fees |
|
|
135 |
|
|
|
2,339 |
|
|
|
(2,204 |
) |
Total research and development expenses |
|
$ |
82,160 |
|
|
$ |
90,698 |
|
|
$ |
(8,538 |
) |
The decrease of approximately $8.5 million was primarily attributable to $5.3 million of decreased external research and development costs, primarily associated with a decrease in variable external research and manufacturing costs.
General and Administrative Expenses
General and administrative expenses were $17.4 million for the three months ended September 30, 2024, compared to general and administrative expenses of $18.3 million for the three months ended September 30, 2023. The decrease was primarily associated with a decrease in consulting and professional services costs.
Collaboration Expense, Net
Collaboration expense, net, was $11.2 million for the three months ended September 30, 2024, compared to $23.4 million for the three months ended September 30, 2023. In both the third quarter of 2024 and the third quarter of 2023, we exercised our option to defer specified costs on the CASGEVY program in excess of the $110.3 million deferral limit under the A&R Vertex JDCA, as amended. The decrease of approximately $12.3 million in collaboration expense, net, was primarily attributable to the timing of when we reached the deferral limit, as a result of increased commercial and manufacturing costs for CASGEVY when compared to the prior period. As of September 30, 2024, we have deferred $44.9 million in 2024 under the A&R Vertex JDCA, as amended, compared to a deferral of $23.4 million in 2023 as of September 30, 2023.
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Other Income, Net
Other income was $25.1 million for the three months ended September 30, 2024, compared to $20.7 million of income for the three months ended September 30, 2023. The increase of approximately $4.4 million was primarily due to interest income earned on cash, cash equivalents and marketable securities for the three months ended September 30, 2024.
Comparison of nine months ended September 30, 2024 and 2023 (in thousands):
|
|
Nine Months Ended September 30, |
|
|
Period to Period |
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
Revenue: |
|
|
|
|
|
|
|
|
|
|||
Collaboration revenue |
|
$ |
— |
|
|
$ |
170,000 |
|
|
$ |
(170,000 |
) |
Grant revenue |
|
|
1,623 |
|
|
|
— |
|
|
|
1,623 |
|
Total revenue |
|
|
1,623 |
|
|
|
170,000 |
|
|
|
(168,377 |
) |
Operating expenses: |
|
|
|
|
|
|
|
|
|
|||
Research and development |
|
|
238,498 |
|
|
|
292,188 |
|
|
|
(53,690 |
) |
General and administrative |
|
|
54,853 |
|
|
|
59,683 |
|
|
|
(4,830 |
) |
Collaboration expense, net |
|
|
110,250 |
|
|
|
110,250 |
|
|
|
— |
|
Total operating expenses |
|
|
403,601 |
|
|
|
462,121 |
|
|
|
(58,520 |
) |
Loss from operations |
|
|
(401,978 |
) |
|
|
(292,121 |
) |
|
|
(109,857 |
) |
Other income, net |
|
|
75,924 |
|
|
|
51,819 |
|
|
|
24,105 |
|
Loss before income taxes |
|
|
(326,054 |
) |
|
|
(240,302 |
) |
|
|
(85,752 |
) |
Provision for income taxes |
|
|
(2,887 |
) |
|
|
(2,655 |
) |
|
|
(232 |
) |
Net loss |
|
$ |
(328,941 |
) |
|
$ |
(242,957 |
) |
|
$ |
(85,984 |
) |
|
|
|
|
|
|
|
|
|
|
|||
Collaboration Revenue
There was no collaboration revenue for the nine months ended September 30, 2024. Collaboration revenue for the nine months ended September 30, 2023 was $170.0 million due to an upfront payment from Vertex in the first quarter of 2023, as well as revenue recognized in the second quarter of 2023 related to a research milestone which was achieved in the second quarter of 2023. Please refer to Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.
Research and Development Expenses
Research and development expenses were $238.5 million for the nine months ended September 30, 2024, compared to $292.2 million for the nine months ended September 30, 2023. The following table summarizes our research and development expenses for the nine months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands):
|
|
Nine Months Ended September 30, |
|
|
Period to Period |
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
External research and development expenses |
|
$ |
63,083 |
|
|
$ |
102,642 |
|
|
$ |
(39,559 |
) |
Employee related expenses |
|
|
58,015 |
|
|
|
63,576 |
|
|
|
(5,561 |
) |
Facility expenses |
|
|
73,486 |
|
|
|
81,662 |
|
|
|
(8,176 |
) |
Stock-based compensation expenses |
|
|
36,542 |
|
|
|
36,284 |
|
|
|
258 |
|
Other expenses |
|
|
1,467 |
|
|
|
2,191 |
|
|
|
(724 |
) |
Sublicense and license fees |
|
|
5,905 |
|
|
|
5,833 |
|
|
|
72 |
|
Total research and development expenses |
|
$ |
238,498 |
|
|
$ |
292,188 |
|
|
$ |
(53,690 |
) |
The decrease of approximately $53.7 million was primarily attributable to the following:
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General and Administrative Expenses
General and administrative expenses were $54.9 million for the nine months ended September 30, 2024, compared to general and administrative expenses of $59.7 million for the nine months ended September 30, 2023. The decrease of approximately $4.8 million was primarily attributable to decreased employee-related expenses and consulting and professional services-related expenses.
Collaboration Expense, Net
Collaboration expense, net, was $110.3 million for the nine months ended September 30, 2024 and 2023, which represents our share of costs for the CASGEVY program. In both the third quarter of 2024 and the third quarter of 2023, we exercised our option to defer specified costs on the CASGEVY program in excess of the $110.3 million deferral limit under the A&R Vertex JDCA, as amended. As of September 30, 2024, we have deferred $44.9 million in 2024 under the A&R Vertex JDCA, as amended, compared to a deferral of $23.4 million in 2023 as of September 30, 2023. This increase in the deferral is a result of increased commercial and manufacturing costs for CASGEVY when compared to the prior period.
Other Income, Net
Other income was $75.9 million for the nine months ended September 30, 2024, compared to $51.8 million of income for the nine months ended September 30, 2023. The increase of approximately $24.1 million was primarily due to interest income earned on cash, cash equivalents and marketable securities for the nine months ended September 30, 2024.
Liquidity and Capital Resources
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of September 30, 2024, we had $1,935.6 million in cash, cash equivalents and marketable securities, of which approximately $154.6 million was held outside of the United States, and an accumulated deficit of $1,328.6 million. We anticipate that we will continue to incur losses for at least the next several years. We expect to continue to incur research and development costs and general and administrative expenses consistent with costs associated with research and development at companies of our size and stage of development, and, as a result, we will need additional capital to fund our operations, which we may raise through public or private equity or debt financings, strategic collaborations, or other sources.
In August 2019, we entered into the August 2019 Sales Agreement with Jefferies and filed our current prospectus supplement for $378.6 million in August 2024. As of September 30, 2024, we have issued and sold an aggregate of 1.6 million common shares under the 2021 ATM at an average price of $134.76 per share for aggregate proceeds of $218.4 million, which were net of equity issuance costs of $2.9 million, excluding stamp taxes.
In February 2024, the Company entered into an investment agreement for the sale of approximately $280.0 million of its common shares to a group of institutional investors in a registered direct offering, at a price per share of $71.50. The Company received net proceeds of $279.0 million, excluding stamp taxes due of $2.8 million.
Funding Requirements
Our primary uses of capital are, and we expect will continue to be, research and development activities, manufacturing activities, compensation and related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution, filing, defense and intellectual property maintenance costs, and general overhead costs, including costs associated with operating as a public company. We expect to continue to incur operating expenses consistent with costs associated with research and development at companies of our size and stage of development, which may increase in the future to support continued research and development activities and potential commercialization of our product candidates.
Although we and our partner, Vertex, received marketing approval of CASGEVY in 2023 in certain jurisdictions, and have received subsequent approvals in 2024, most of our programs are still in early stages of development and the outcome of these efforts is uncertain, we cannot estimate the actual amounts necessary to successfully complete the development, manufacture and commercialization of any current or future product candidates, if approved, or whether, or when, we may achieve profitability. Until such time as we can generate substantial product revenues, if ever, we expect to finance our cash needs through a combination of equity financings, debt financings and payments received in connection with our collaboration agreements. We intend to consider opportunities to raise additional funds through the sale of equity or debt securities when market conditions are favorable to us to do so. However, the trading prices for our common shares and other biopharmaceutical companies have been highly volatile. As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event, including resulting from the continued spread of the coronavirus or the recent failure of certain banks and financial institutions in the United States and globally, could materially and adversely affect our business and the value of our common shares. To the extent that we raise additional capital through the future sale of equity or debt securities, the ownership interests of our shareholders will be diluted, and the terms of these securities may include liquidation or other
25
preferences that adversely affect the rights of our existing shareholders. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Outlook
Based on our research and development plans and our timing expectations related to the progress of our programs, we expect our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses and capital expenditures for at least the next 24 months without giving effect to any additional proceeds we may receive under our collaboration with Vertex and any other capital raising transactions we may complete. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect. Given our need for additional financing to support the long-term clinical development of our programs, we intend to consider additional financing opportunities when market terms are favorable to us.
Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our delivery technologies and our gene editing technology platform; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance of our product candidates, either directly or with a collaborator or distributor, if approved, including for CASGEVY; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter; maintaining good relationships with our collaborators and licensors; maintaining, defending, protecting and expanding our estate of intellectual property rights, including patents, trade secrets and know-how; and attracting, hiring and retaining qualified personnel.
Cash Flows
The following table provides information regarding our cash flows for each of the periods below (in thousands):
|
|
Nine Months Ended September 30, |
|
|
Period to Period |
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
Net cash used in operating activities |
|
$ |
(92,743 |
) |
|
$ |
(164,302 |
) |
|
$ |
71,559 |
|
Net cash (used in) provided by investing activities |
|
|
(386,309 |
) |
|
|
456,100 |
|
|
|
(842,409 |
) |
Net cash provided by financing activities |
|
|
315,108 |
|
|
|
23,725 |
|
|
|
291,383 |
|
Effect of exchange rate changes on cash |
|
|
66 |
|
|
|
12 |
|
|
|
54 |
|
Net (decrease) increase in cash |
|
$ |
(163,878 |
) |
|
$ |
315,535 |
|
|
$ |
(479,413 |
) |
Operating Activities
Net cash used in operating activities was $92.7 million for the nine months ended September 30, 2024, compared to cash used in operating activities of $164.3 million for the nine months ended September 30, 2023. The decrease in net cash used in operating activities was primarily driven by cash received from a milestone recognized as revenue in 2023, offset by increased net losses.
Investing Activities
Net cash used in investing activities for the nine months ended September 30, 2024 was $386.3 million, compared to net cash provided by investing activities of $456.1 million for the nine months ended September 30, 2023. The change to a net cash used in investing activities from a net cash provided by investing activities was primarily driven by a net increase in purchases of our marketable securities.
Financing Activities
Net cash provided by financing activities for the nine months ended September 30, 2024 was $315.1 million, compared with $23.7 million for the nine months ended September 30, 2023. Net cash provided by financing activities for the nine months ended September 30, 2024 primarily consisted of proceeds from the sale of approximately $280.0 million of its common shares to a group of institutional investors in a registered direct offering, at a price per share of $71.50.
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Critical Accounting Policies and Significant Judgments and Estimates
This discussion and analysis of our financial condition and results of operations is based on our financial statements, which we have prepared in accordance with U.S. GAAP. We believe that several accounting policies are important to understanding our historical and future performance. We refer to these policies as critical because these specific areas generally require us to make judgments and estimates about matters that are uncertain at the time we make the estimate, and different estimates—which also would have been reasonable—could have been used. On an ongoing basis, we evaluate our estimates and judgments, including those described in greater detail below. We base our estimates on historical experience and other market-specific or other relevant assumptions that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
We believe that our most critical accounting policies are those relating to revenue recognition and equity-based compensation, and there have been no changes to our accounting policies discussed in our Annual Report on Form 10-K for the year ended December 31, 2023 filed with the SEC on February 21, 2024.
Recent Accounting Pronouncements
Refer to Note 1 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for a discussion of recent accounting pronouncements.
Item 3. Qualitative and Quantitative Disclosures about Market Risk
Interest Rate Sensitivity
We are exposed to market risk related to changes in interest rates. As of September 30, 2024, we had cash, cash equivalents and marketable securities of $1,935.6 million, primarily invested in U.S. treasury securities and government agency securities, corporate bonds, commercial paper and money market accounts invested in U.S. government agency securities. Due to the conservative nature of these instruments, we do not believe that we have a material exposure to interest rate risk. If interest rates were to increase or decrease by 1%, the fair value of our investment portfolio would increase or decrease by an immaterial amount.
Foreign Currency Exchange Rate Risk
As a result of our foreign operations, we face exposure to movements in foreign currency exchange rates, primarily the Swiss Franc and British Pound, against the U.S. dollar. The current exposures arise primarily from cash, accounts payable and intercompany receivables and payables. Changes in foreign exchange rates affect our consolidated statement of operations and distort comparisons between periods. To date, foreign currency transaction gains and losses have not been material to our financial statements, and we have not engaged in any foreign currency hedging transactions.
Inflation
Inflation generally affects us by increasing our cost of labor, clinical trial and manufacturing costs. We do not believe that inflation had a material effect on our business, financial condition or results of operations during the three and nine months ended September 30, 2024 and 2023.
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Item 4. Controls and Procedures.
Management’s Evaluation of our Disclosure Controls and Procedures
We maintain disclosure controls and procedures that are designed to ensure that information required to be disclosed in the reports that we file or submit under the Securities and Exchange Act of 1934 is (1) recorded, processed, summarized, and reported within the time periods specified in the SEC’s rules and forms and (2) accumulated and communicated to our management, including our principal executive officer and principal financial officer, to allow timely decisions regarding required disclosure.
As of September 30, 2024, our management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities and Exchange Act of 1934). Our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Our principal executive officer and principal financial officer have concluded based upon the evaluation described above that, as of September 30, 2024, our disclosure controls and procedures were effective at the reasonable assurance level.
Changes in Internal Control over Financial Reporting
During the quarter ended September 30, 2024, there were no changes in our internal control over financial reporting, as such term is defined in Rules 13a-15(f) and 15(d)-15(f) promulgated under the Securities Exchange Act of 1934, that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
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PART II—OTHER INFORMATION
Item 1. Legal Proceedings.
In the ordinary course of business, we are from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, our intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office involving our intellectual property estate including certain in-licensed intellectual property. There are currently no claims or actions pending against us that, in the opinion of our management, are likely to have a material adverse effect on our business.
There have been no material developments with respect to the legal proceedings previously disclosed in “Item 3. Legal Proceedings” of our Annual Report on Form 10-K for the fiscal year ended December 31, 2023 filed with the SEC on February 21, 2024.
Item 1A. Risk Factors.
In addition to the risks described in our Annual Report on Form 10-K and any quarterly report on Form 10-Q, you should carefully consider the other information set forth in this Form 10-Q and the information in our other filings with the SEC, as they could materially affect our business, financial condition or future results of operations. There have been no material changes to the risk factors previously disclosed in Part I, Item 1A (Risk Factors) of our Annual Report on Form 10-K.
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds.
None.
Item 3. Defaults Upon Senior Securities.
Not applicable.
Item 4. Mine Safety Disclosures.
Not applicable.
Item 5. Other Information.
From time to time, our officers (as defined in Rule 16a–1(f)) and directors may enter into Rule 10b5-1 or non-Rule 10b5-1 trading arrangements (as each such term is defined in Item 408 of Regulation S-K). During the three months ended September 30, 2024, none of our officers and directors
Item 6. Exhibits
The exhibits filed as part of this Quarterly Report on Form 10-Q are set forth in the Exhibit Index below.
Exhibit Number |
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Description of Document |
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10.1* |
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31.1* |
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Certification of Chief Executive Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 |
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31.2* |
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Certification of Chief Financial Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 |
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32.1*+ |
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101.INS* |
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Inline XBRL Instance Document – the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the inline XBRL document. |
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101.SCH* |
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Inline XBRL Taxonomy Extension Schema With Embedded Linkbase Documents |
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104* |
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Cover Page Interactive Data File (formatted as inline XBRL with applicable taxonomy extension information contained in Exhibits 101) |
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* Filed herewith.
+ The certification attached as Exhibit 32.1 that accompany this Quarterly Report on Form 10-Q are not deemed filed with the Securities and Exchange Commission and are not to be incorporated by reference into any filing of CRISPR Therapeutics AG under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Form 10-Q, irrespective of any general incorporation language contained in such filing.
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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.
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CRISPR Therapeutics AG |
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Dated: November 5, 2024 |
By: |
/s/ Samarth Kulkarni |
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Samarth Kulkarni, Ph.D. |
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Chief Executive Officer |
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(Principal Executive Officer) |
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Dated: November 5, 2024 |
By: |
/s/ Raju Prasad |
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Raju Prasad, Ph.D. |
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Chief Financial Officer |
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(Principal Financial Officer) |
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