美國
證券交易委員會
華盛頓特區20549
表格
（標記一）
根據1934年證券交易法第13或15(d)節的季度報告 |
截至季度結束日期的財務報告
或者
根據1934年證券交易法第13或15(d)節的轉型報告書 |
針對從__________________到__________________的過渡期
委託文件編號：001-39866
（依據其憲章指定的註冊名稱）
（國家或其他管轄區的 公司成立或組織） |
（IRS僱主 唯一識別號碼） |
，（主要行政辦公地址） |
(郵政編碼) |
公司電話，包括區號：(
在法案第12(b)條的規定下注冊的證券：
每一類的名稱 |
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交易 符號: |
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在其上註冊的交易所的名稱 |
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請在以下複選框中打勾，指示註冊人：(1)在前12個月(或註冊人被要求提交這些報告的更短期間內)已經提交了1934年證券交易法第13或15(d)條規定需要提交的所有報告；以及(2)在過去的90天內一直受到了此類文件提交要求的限制。
請在以下複選框中打勾，指示註冊人是否已經電子提交了根據Regulation S-T規則405條(本章節的§232.405條)需要提交的所有互動數據文件在過去的12個月內(或註冊人被要求提交這些文件的更短期間內)。
勾選以下選框，指示申報人是大型加速評估提交人、加速評估提交人、非加速評估提交人、小型報告公司或新興成長型公司。關於「大型加速評估提交人」、「加速評估提交人」、「小型報告公司」和「新興成長型公司」的定義，請參見《交易所法規》第12億.2條。
大型加速報告人 |
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加速文件提交人 |
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☐ |
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☒ |
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較小的報告公司 |
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新興成長公司 |
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如果是新興成長型企業，請勾選複選標記，表明註冊者已選擇不使用延長過渡期來符合根據證券交易法第13(a)條規定提供的任何新財務會計準則。
請在以下空格內打勾，表示註冊人是不是外殼公司（按交易所法則120億.2條定義）。 是 ☐ 否
截至2024年11月1日，註冊公司的普通股流通數量爲
目錄
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頁面 |
第一部分 |
1 |
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第 1 項。 |
1 |
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1 |
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2 |
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3 |
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4 |
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5 |
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第 2 項。 |
12 |
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第 3 項。 |
19 |
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第 4 項。 |
19 |
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第二部分。 |
21 |
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第 1 項。 |
21 |
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第 1A 項。 |
21 |
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第 5 項。 |
21 |
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第 6 項。 |
23 |
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24 |
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i
關於公司參考的說明
在本第10-Q表格的季度報告中，「公司」、「Vor」、「Vor Bio」、「Vor生物製藥公司」、「我們」、「我們的」，除非上下文另有要求，均指Vor生物製藥公司及其合併子公司，「我們的董事會」指的是Vor生物製藥公司的董事會。
關於前瞻性聲明的特別說明
本季度10-Q表格中包含涉及重大風險和不確定性的前瞻性聲明。除歷史事實聲明外，本季度10-Q報告中包含的所有聲明，包括關於我們的戰略、未來運營、未來財務狀況、未來營業收入、預計成本、前景、計劃和管理目標，均屬於前瞻性聲明。在某些情況下，您可以通過諸如「可能」、「將」、「應該」、「期望」、「計劃」、「預期」、「可以」、「可能」、「打算」、「目標」、「持續」、「項目」、「估計」、「認爲」、「估計」、「預測」、「潛在」或「繼續」等術語識別前瞻性聲明，或使用這些術語的負面形式或其他類似表達意圖識別關於未來的聲明。這些聲明僅代表本季度10-Q表格日期，並涉及已知和未知的風險、不確定性以及可能導致我們的實際結果、活動水平、表現或成就與前瞻性聲明所反映或暗示的任何未來結果、表現或成就存在實質性差異的其他重要因素。
我們在很大程度上是基於我們對未來事件和金融趨勢的當前期望和預測，我們相信這些可能會影響我們的業務、財務狀況和經營業績。這些前瞻性聲明包括但不限於以下內容：
ii
您應該仔細閱讀此表格10-Q季度報告以及我們作爲附件提交的文件，並理解我們未來實際結果可能與預期有很大不同。此表格10-Q季度報告中包含的前瞻性聲明是根據此10-Q季度報告的日期作出的，我們不承擔更新任何前瞻性聲明的義務，除非根據適用法律的規定需要。您應查閱此季度報告中的「風險因素」部分，以及我們截至2023年12月31日年度報告中的「概要風險因素」和「風險因素」部分，討論可能導致實際結果或事件與我們提出的前瞻性聲明有實質不同的重要因素。
本季度10-Q表格中包含統計數據以及其他行業和市場數據，這些數據來自我們自身的內部估計和研究，也來自行業和一般出版物、研究、第三方調查和研究。行業出版物、研究和調查通常陳述這些信息來源被認爲是可靠的，儘管他們並不保證這些信息的準確性或完整性。雖然我們相信每一份研究和出版物都是可靠的，但我們並未獨立核實第三方來源的市場和行業數據。雖然我們認爲我們的內部公司研究是可靠的，市場定義是合適的，但這些研究或定義都未經任何獨立來源驗證過。
所有板塊或商標出現在本季度10-Q表格中，包括Mylotarg，在此歸屬於其各自所有者。
iii
第一部分—財政財務信息
項目 1. 財務報表財務報表（未經審計）
VOR生物製藥公司。
壓縮的綜合合併資產負債表
(未經審計)
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9月30日， |
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12月31日, |
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(以千爲單位，除每股數據外) |
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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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$ |
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$ |
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有價證券 |
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預付費用 |
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其他資產 |
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總流動資產 |
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限制性現金等價物 |
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資產和設備，淨值 |
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經營租賃權使用資產 |
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其他 |
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資產總額 |
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$ |
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$ |
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負債和股東權益 |
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流動負債: |
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應付賬款 |
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$ |
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$ |
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應計負債 |
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經營租賃負債 |
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其他流動負債 |
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流動負債合計 |
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長期負債： |
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經營租賃負債—非流動 |
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負債合計 |
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股東權益: |
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優先股，$0.0001 |
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普通股，每股面值爲 $0.0001； |
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額外實收資本 |
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累計其他綜合收益(虧損) |
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( |
) |
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累積赤字 |
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( |
) |
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( |
) |
股東權益總額 |
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負債和股東權益總額 |
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$ |
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$ |
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隨附說明是這些簡明合併財務報表的一部分。
1
VOR BIOPHARMA INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(UNAUDITED)
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Three Months Ended |
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Nine Months Ended |
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(in thousands, except share and per share amounts) |
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2024 |
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2023 |
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2024 |
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2023 |
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Operating expenses: |
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Research and development |
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$ |
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$ |
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$ |
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$ |
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General and administrative |
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Total operating expenses |
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$ |
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$ |
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$ |
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$ |
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Loss from operations |
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$ |
( |
) |
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$ |
( |
) |
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$ |
( |
) |
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$ |
( |
) |
Other income: |
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Interest income |
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Total other income |
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Net loss |
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$ |
( |
) |
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$ |
( |
) |
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$ |
( |
) |
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$ |
( |
) |
Net loss per share attributable to common stockholders, |
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$ |
( |
) |
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$ |
( |
) |
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$ |
( |
) |
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$ |
( |
) |
Weighted-average common shares outstanding, |
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Other comprehensive income: |
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Unrealized gain on available for sale marketable securities |
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Total other comprehensive income |
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Comprehensive loss |
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$ |
( |
) |
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$ |
( |
) |
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$ |
( |
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$ |
( |
) |
The accompanying notes are an integral part of these condensed consolidated financial statements.
2
Vor Biopharma Inc.
CONDENSED CONSOLIDATED Statements of stockholders’ EQUITY
(Unaudited)
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Common |
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Additional |
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Accumulated other |
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Accumulated |
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Total |
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(in thousands, except share amounts) |
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Shares |
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Amount |
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Capital |
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(loss) income |
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Deficit |
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Equity |
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Balance at December 31, 2023 |
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$ |
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$ |
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$ |
( |
) |
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$ |
( |
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$ |
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Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, and exercise of stock options |
|
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— |
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( |
) |
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— |
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— |
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( |
) |
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Issuance of common stock from at-the-market sales agreement |
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— |
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— |
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— |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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Other comprehensive loss |
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— |
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— |
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— |
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( |
) |
|
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— |
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( |
) |
Net loss |
|
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— |
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— |
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— |
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— |
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( |
) |
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( |
) |
Balance at March 31, 2024 |
|
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$ |
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$ |
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$ |
( |
) |
|
$ |
( |
) |
|
$ |
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Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, exercise of stock options, and issuance of common stock under ESPP |
|
|
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— |
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— |
|
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— |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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Other comprehensive income |
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— |
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— |
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— |
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|
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— |
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Net loss |
|
|
— |
|
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— |
|
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— |
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— |
|
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( |
) |
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( |
) |
Balance at June 30, 2024 |
|
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$ |
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$ |
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$ |
( |
) |
|
$ |
( |
) |
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$ |
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Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, and exercise of stock options |
|
|
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— |
|
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( |
) |
|
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— |
|
|
|
— |
|
|
|
( |
) |
|
Issuance of common stock from at-the-market sales agreement |
|
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|
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— |
|
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|
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— |
|
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— |
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|||
Stock-based compensation expense |
|
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— |
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— |
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— |
|
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— |
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Other comprehensive income |
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— |
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— |
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— |
|
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— |
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Net loss |
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— |
|
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— |
|
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— |
|
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— |
|
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|
( |
) |
|
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( |
) |
Balance at September 30, 2024 |
|
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$ |
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$ |
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$ |
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$ |
( |
) |
|
$ |
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Common |
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Additional |
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Accumulated other comprehensive |
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Accumulated |
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Total |
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(in thousands, except share amounts) |
|
Shares |
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Amount |
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Capital |
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loss |
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Deficit |
|
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Equity |
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Balance at December 31, 2022 |
|
|
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|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
|
||||
Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, and exercise of stock options |
|
|
|
|
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— |
|
|
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( |
) |
|
|
— |
|
|
|
— |
|
|
|
( |
) |
|
Issuance of common stock from at-the-market sales agreement |
|
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|
|
— |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
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|
|||
Stock-based compensation expense |
|
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— |
|
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— |
|
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— |
|
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— |
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Other comprehensive income |
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— |
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— |
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— |
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— |
|
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||
Net loss |
|
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— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
( |
) |
|
|
( |
) |
Balance at March 31, 2023 |
|
|
|
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
|
||||
Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, exercise of stock options, and issuance of common stock under ESPP |
|
|
|
|
|
— |
|
|
|
( |
) |
|
|
— |
|
|
|
— |
|
|
|
( |
) |
|
Issuance of common stock from at-the-market sales agreement |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
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|||
Stock-based compensation expense |
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|
— |
|
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— |
|
|
|
|
|
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— |
|
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— |
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Other comprehensive loss |
|
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— |
|
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— |
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— |
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( |
) |
|
|
— |
|
|
|
( |
) |
Net loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
( |
) |
|
|
( |
) |
Balance at June 30, 2023 |
|
|
|
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
|
||||
Issuance of common stock upon vesting of RSUs, vesting and exercise of stock options, net of shares withheld for taxes |
|
|
|
|
|
— |
|
|
|
( |
) |
|
|
— |
|
|
|
— |
|
|
|
( |
) |
|
Issuance of common stock from at-the-market sales agreement |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
|||
Stock-based compensation expense |
|
|
— |
|
|
|
— |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
||
Other comprehensive income |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
|
|
|
— |
|
|
|
|
||
Net loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
( |
) |
|
|
( |
) |
Balance at September 30, 2023 |
|
|
|
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
|
||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
3
Vor Biopharma Inc.
condensed CONSOLIDATED StatementS of Cash Flows
(unaudited)
|
|
Nine Months Ended September 30, |
|
|||||
(in thousands) |
|
2024 |
|
|
2023 |
|
||
Cash flows from operating activities |
|
|
|
|
|
|
||
Net loss |
|
$ |
( |
) |
|
$ |
( |
) |
Adjustments to reconcile net loss to net cash used in operations: |
|
|
|
|
|
|
||
Depreciation expense |
|
|
|
|
|
|
||
Non-cash lease expense |
|
|
|
|
|
|
||
Stock-based compensation |
|
|
|
|
|
|
||
Interest amortization on marketable securities |
|
|
( |
) |
|
|
( |
) |
Changes in operating assets and liabilities: |
|
|
|
|
|
|
||
Operating lease liabilities |
|
|
( |
) |
|
|
( |
) |
Prepaid expenses and other current assets |
|
|
( |
) |
|
|
|
|
Accounts payable and accrued liabilities |
|
|
( |
) |
|
|
|
|
Other assets |
|
|
|
|
|
( |
) |
|
Net cash used in operating activities |
|
|
( |
) |
|
|
( |
) |
Cash flow from investing activities |
|
|
|
|
|
|
||
Purchases of marketable securities |
|
|
( |
) |
|
|
( |
) |
Proceeds from maturities of marketable securities |
|
|
|
|
|
|
||
Purchases of property and equipment |
|
|
( |
) |
|
|
( |
) |
Net cash provided by investing activities |
|
|
|
|
|
|
||
Cash flow from financing activities |
|
|
|
|
|
|
||
Payment of issuance costs related to underwritten public offering and concurrent private placement |
|
|
— |
|
|
|
( |
) |
Proceeds from the issuance of common stock from at-the-market sales agreement, net of issuance costs |
|
|
|
|
|
|
||
Repurchases of shares for tax withholdings upon vesting of restricted stock unit awards |
|
|
( |
) |
|
|
( |
) |
Proceeds from stock option exercises and the issuance of shares under ESPP |
|
|
|
|
|
|
||
Net cash provided by financing activities |
|
|
|
|
|
|
||
Net increase (decrease) in cash, cash equivalents and restricted cash equivalents |
|
|
|
|
|
( |
) |
|
Cash, cash equivalents and restricted cash equivalents, |
|
$ |
|
|
$ |
|
||
Cash, cash equivalents and restricted cash equivalents, end of period |
|
$ |
|
|
$ |
|
||
Supplemental disclosure of non-cash activities |
|
|
|
|
|
|
||
Right-of-use assets obtained in exchange for lease obligations |
|
$ |
— |
|
|
$ |
|
|
Purchases of property and equipment in accounts payable and accrued liabilities |
|
$ |
— |
|
|
$ |
|
|
Financing costs associated with the sale of common stock included in accounts payable and accrued expenses |
|
$ |
|
|
$ |
|
||
A reconciliation of the cash, cash equivalents and restricted cash equivalents reported within the condensed consolidated balance sheets that sum to the total of the same amounts shown in the statements of cash flows is as follows:
|
|
For the Nine Months Ended September 30, |
|
|||||
(in thousands) |
|
2024 |
|
|
2023 |
|
||
Cash and cash equivalents |
|
$ |
|
|
$ |
|
||
Restricted cash equivalents |
|
|
|
|
|
|
||
Total cash, cash equivalents and restricted cash equivalents as shown on the |
|
$ |
|
|
$ |
|
||
The accompanying notes are an integral part of these condensed consolidated financial statements.
4
VOR BIOPHARMA INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(UNAUDITED)
1. Nature of the Business
Vor Biopharma Inc. (the “Company”) is a clinical-stage cell and genome engineering company that combines a novel patient engineering approach with targeted therapies to provide a single company solution for patients suffering from hematological malignancies. The Company’s proprietary platform leverages its expertise in hematopoietic stem cell (“HSC”) biology, genome engineering and targeted therapy development to genetically modify HSCs to remove surface targets expressed by cancer cells. The Company is headquartered in Cambridge, Massachusetts. The Company was incorporated on
Risks and Uncertainties
The Company is subject to a number of risks common to development stage companies in the biotechnology industry, including, but not limited to, risks of failure of preclinical studies and clinical trials, dependence on key personnel, protection of proprietary technology, reliance on third party organizations, risks of obtaining regulatory approval for any product candidate that it may develop, development by competitors of technological innovations, compliance with government regulations, adverse macroeconomic conditions and the need to obtain additional financing.
The Company anticipates that it will continue to incur significant operating losses for the next several years as it continues to develop its product candidates. As a result, the Company’s continued operations are dependent on its ability to raise additional funding. If the Company is unable to obtain additional funding on a timely basis, it may be forced to significantly curtail, delay, or discontinue one or more of its planned research or development programs or be unable to expand its operations.
Liquidity and Capital Resources
As of September 30, 2024, the Company had $
If, for any reason, the Company utilizes its capital resources more quickly than anticipated or is unable to obtain additional funding on a timely basis, it may be required to revise its business plan and strategy. This may result in the Company significantly curtailing, delaying or discontinuing one or more of its research and development programs. As a result, the Company’s business, financial condition, and results of operations could be materially affected. The accompanying condensed consolidated financial statements do not include any adjustments to the carrying amounts and classification of assets and liabilities that may be necessary if the Company were unable to continue as a going concern.
2. Summary of Significant Accounting Policies
Basis of Presentation
The accompanying condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Certain comparative amounts have been reclassified to conform to the current period presentation, including the presentation of non-cash lease expense in the condensed consolidated statements of cash flows. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) issued by the Financial Accounting Standards Board (“FASB”).
5
Use of Estimates
The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the condensed consolidated financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the condensed consolidated financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: accrued expenses and research and development expenses.
Unaudited Interim Financial Information
The condensed consolidated financial statements of the Company included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (the “SEC”). The unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiary. All intercompany transactions and balances have been eliminated in consolidation. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period.
The accompanying condensed consolidated balance sheet as of December 31, 2023 has been derived from the Company’s audited consolidated financial statements for the year ended December 31, 2023. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. These unaudited interim condensed consolidated financial statements should be read in conjunction with the audited annual consolidated financial statements and related notes included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 (the “2023 Annual Report”).
During the nine months ended September 30, 2024, there have been no changes to the Company’s significant accounting policies as described in the 2023 Annual Report.
3. Marketable Securities
The amortized cost and estimated fair value of marketable securities, by remaining contractual maturity, are as follows:
|
|
September 30, 2024 |
|
|||||||||||||
(in thousands) |
|
Amortized Cost |
|
|
Gross Unrealized Holding Gains |
|
|
Gross Unrealized Holding Losses |
|
|
Fair Value |
|
||||
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Maturing after one year through five years |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasuries |
|
|
|
|
|
|
|
|
— |
|
|
|
|
|||
Total |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
|
|||
|
|
December 31, 2023 |
|
|||||||||||||
(in thousands) |
|
Amortized Cost |
|
|
Gross Unrealized Holding Gains |
|
|
Gross Unrealized Holding Losses |
|
|
Fair Value |
|
||||
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Maturing in one year or less |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasury Bill |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
|
|||
U.S. Treasuries |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total |
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|||
6
|
|
December 31, 2023 |
|
|||||||||||||||||||||
|
|
Less than twelve months |
|
|
Greater than twelve months |
|
|
Total |
|
|||||||||||||||
(in thousands) |
|
Fair value |
|
|
Unrealized loss |
|
|
Fair value |
|
|
Unrealized loss |
|
|
Fair value |
|
|
Unrealized loss |
|
||||||
U.S. Treasuries |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|
$ |
( |
) |
||
Total |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|
$ |
( |
) |
||
The Company did
4. Fair Value Measurements
The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis:
|
|
September 30, 2024 |
|
|||||||||||||
(in thousands) |
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
|
Total |
|
||||
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash equivalents |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
||
Marketable securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasuries |
|
|
— |
|
|
|
|
|
|
— |
|
|
|
|
||
Total marketable securities |
|
|
— |
|
|
|
|
|
|
— |
|
|
|
|
||
Restricted cash equivalents |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
||
Total |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
|
|||
|
|
December 31, 2023 |
|
|||||||||||||
(in thousands) |
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
|
Total |
|
||||
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash equivalents |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
||
Marketable securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
U.S. Treasury Bill |
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
||
U.S. Treasuries |
|
|
— |
|
|
|
|
|
|
— |
|
|
|
|
||
Total marketable securities |
|
|
|
|
|
|
|
|
— |
|
|
|
|
|||
Restricted cash equivalents |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
||
Total |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
|
|||
The fair value of the Company’s cash equivalents and restricted cash equivalents is based on quoted market prices in active markets with no valuation adjustment. The fair value of marketable securities was determined based on observable market inputs. There were
Prepaid expenses, accounts payable and accrued expenses are stated at their respective historical carrying values, which approximate fair value due to their short-term nature.
7
5. Property and Equipment, Net
Property and equipment, net consisted of the following:
|
|
September 30, |
|
|
December 31, |
|
||
(in thousands) |
|
2024 |
|
|
2023 |
|
||
Laboratory equipment |
|
$ |
|
|
$ |
|
||
Manufacturing equipment |
|
|
|
|
|
|
||
Computer equipment |
|
|
|
|
|
|
||
Furniture, fixtures and other |
|
|
|
|
|
|
||
Construction in progress |
|
|
|
|
|
|
||
Total |
|
|
|
|
|
|
||
Less: Accumulated depreciation |
|
|
( |
) |
|
|
( |
) |
Property and equipment, net |
|
$ |
|
|
$ |
|
||
Depreciation expense for the three and nine months ended September 30, 2024 was $
6. Accrued Liabilities
Accrued liabilities consisted of the following:
|
|
September 30, |
|
|
December 31, |
|
||
(in thousands) |
|
2024 |
|
|
2023 |
|
||
Employee-related expenses |
|
$ |
|
|
$ |
|
||
Professional fees |
|
|
|
|
|
|
||
Clinical expenses |
|
|
|
|
|
|
||
Manufacturing expenses |
|
|
|
|
|
|
||
Research and development expenses |
|
|
|
|
|
|
||
Other |
|
|
|
|
|
|
||
Total accrued liabilities |
|
$ |
|
|
$ |
|
||
7. Stock-Based Compensation
2023 Inducement Plan
As of September 30, 2024, the Company had
2021 Equity Incentive Plan
As of September 30, 2024, the Company had
Stock Options
The Company’s stock options generally
|
|
Nine Months Ended September 30, |
|
|||||
|
|
2024 |
|
|
2023 |
|
||
Expected term (in years) |
|
|
|
|
|
|
||
Expected volatility |
|
|
% |
|
|
% |
||
Risk-free interest rate |
|
|
% |
|
|
% |
||
Dividend yield |
|
|
|
|
|
|
||
During the nine months ended September 30, 2024 and 2023, the Company granted stock options to purchase
8
respectively. As of September 30, 2024, total unrecognized compensation expense related to stock options was $
As of September 30, 2024, options for
Restricted Stock Units
During the nine months ended September 30, 2024 and 2023, the Company granted
Employee Stock Purchase Plan
As of September 30, 2024, the Company had
During the nine months ended September 30, 2024, the Company issued
Stock-Based Compensation
Stock-based compensation expense was allocated as follows:
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
||||||||||
(in thousands) |
|
2024 |
|
|
2023 |
|
2024 |
|
|
2023 |
|
||||
Research and development |
|
$ |
|
|
$ |
|
$ |
|
|
$ |
|
||||
General and administrative |
|
|
|
|
|
|
|
|
|
|
|
||||
Total stock-based compensation expense |
|
$ |
|
|
$ |
|
$ |
|
|
$ |
|
||||
8. Leases
Cambridgepark Lease Amendments
On June 15, 2021, the Company entered into the first lease amendment (“First Lease Amendment”) and second lease amendment (“Second Lease Amendment” and, together with the First Lease Amendment, the “Lease Amendments”) with PPF Off 100 Cambridge Park Drive, LLC (the “Landlord”). The Lease Amendments amended the Company’s lease agreement for its corporate office and laboratory facilities with the Landlord in Cambridge, Massachusetts to add additional leased space in the same building (the “Amended Cambridgepark Lease”).
The First Lease Amendment and Second Lease Amendment commenced for accounting purposes on
Payments due associated with the Lease Amendments include fixed and variable payments. Variable payments relate to the Company’s share of the Landlord’s operating costs associated with the underlying assets and are recognized when the event on which those payments are assessed occurs. The Amended Cambridgepark Lease does not contain a residual value guarantee. The Lease Amendments term end dates are coterminous with the existing lease agreement. In conjunction with the Lease Amendments, the Company was required to increase its irrevocable standby letter of credit to $
9
For further information regarding the Company’s Cambridgepark lease, please see Note 9 to the consolidated financial statements included in the 2023 Annual Report.
The elements of lease expense were as follows:
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
(in thousands) |
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
Operating lease cost |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Variable lease cost |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total lease cost |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Amounts reported in the condensed consolidated balance sheets and the weighted-average lease term and discount rate information were as follows:
(in thousands except weighted-average amounts) |
|
September 30, 2024 |
|
|
December 31, 2023 |
|
||
Assets |
|
|
|
|
|
|
||
Operating lease right-of-use assets |
|
$ |
|
|
$ |
|
||
Liabilities |
|
|
|
|
|
|
||
Operating lease liabilities, current |
|
$ |
|
|
$ |
|
||
Operating lease liabilities, non-current |
|
|
|
|
|
|
||
Total lease liabilities |
|
$ |
|
|
$ |
|
||
Weighted-Average Lease Term and Discount Rate |
|
|
|
|
|
|
||
Weighted-average remaining lease term (years) |
|
|
|
|
|
|||
Weighted-average discount rate |
|
|
% |
|
|
% |
||
The following table represents other lease activity:
|
|
Nine Months Ended September 30, |
|
|||||
(in thousands) |
|
2024 |
|
|
2023 |
|
||
Other Information |
|
|
|
|
|
|
||
Cash paid for amounts included in the measurement of lease liabilities |
|
|
|
|
|
|
||
Operating cash flows for operating leases |
|
$ |
|
|
$ |
|
||
Right-of-use assets obtained in exchange for lease obligations |
|
$ |
— |
|
|
$ |
|
|
9. Significant Agreements
Since December 31, 2023, there have been no material changes to the key terms of the Company’s license agreements. For further information regarding the Company’s existing license agreements, please see Note 10 to the consolidated financial statements included in the 2023 Annual Report.
10. Net Loss Per Share
The following table sets forth the computation of the Company’s basic and diluted net loss per share for the three and nine months ended September 30, 2024 and 2023:
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
(in thousands, except share and per share amounts) |
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
Numerator: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Net loss attributable to common stockholders |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
Denominator: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Weighted-average number of common shares outstanding, basic and diluted |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Net loss per share attributable to common stockholders, basic and diluted |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
10
The Company’s potentially dilutive securities were stock options, unvested restricted stock and restricted stock units. Based on the amounts outstanding as of September 30, 2024 and 2023, the Company excluded the following potential common shares from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:
|
|
As of September 30, |
|
|||||
|
|
2024 |
|
|
2023 |
|
||
Options to purchase common stock |
|
|
|
|
|
|||
Unvested restricted stock |
|
|
|
|
|
|
||
Restricted stock units |
|
|
|
|
|
|||
11
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations.
You should read the following discussion and analysis of our financial condition and results of operations together with the unaudited condensed consolidated financial statements and related notes included elsewhere in this Quarterly Report on Form 10-Q and with the consolidated financial statements and related notes included in our Annual Report on Form 10-K for the year ended December 31, 2023 (the “2023 Annual Report”). Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the section titled “Risk Factors” in our 2023 Annual Report and in other reports we have filed or may file with the SEC, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.
Overview
Vor Bio is a clinical-stage company harnessing the power of cell and genome engineering to develop potentially transformative therapies in acute myeloid leukemia (“AML”), a devastating disease with few treatment options. AML is the most common type of acute leukemia in adults and one of the deadliest and most aggressive blood cancers, affecting approximately 20,000 newly diagnosed patients each year in the United States.
Leveraging our expertise in HSC biology and genome engineering, we genetically modify HSCs to remove surface targets and then provide these cells as hematopoietic cell transplants (“HCTs”) to patients. Once these cells engraft into bone marrow, the patient’s healthy cells are shielded because they no longer express the surface target, leaving only the cancerous cells exposed. We believe this will unlock the potential of targeted therapies to selectively destroy cancerous cells while shielding healthy cells. As a result, our shielded transplants are designed to limit the on-target toxicities associated with these targeted therapies, thereby enhancing their utility, and broadening their applicability. We intend to pair our shielded transplants with targeted therapeutics such as antibody drug conjugates (“ADCs”) or VCAR33ALLO, a chimeric antigen receptor (“CAR”)-T therapy designed to target CD33, to bring potentially transformative outcomes to patients and establish a new standard of care Treatment System in AML.
We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML and other blood cancers. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. We released clinical data on 18 patients treated in this trial in September 2024. The data demonstrated:
We expect to provide our next clinical data update at the American Society of Hematology (ASH) 2024 annual meeting in December 2024.
VCAR33ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. In January 2024 we dosed the first patient with VCAR33ALLO in VBP301 and are continuing to dose patients in this study. In September 2024, we announced encouraging in vivo CAR-T expansion data from the first three patients treated, all at the lowest dose of 1 x 106 CAR+ cells/kg.
We believe that the combination of trem-cel followed by treatment with VCAR33ALLO in the post-transplant setting, which we refer to as the trem-cel + VCAR33 Treatment System, may transform patient outcomes and offer the potential for cures for patients that have limited treatment options. The trem-cel + VCAR33 Treatment System would utilize the same healthy donor allogenic cell source for both trem-cel and VCAR33ALLO. We plan to collect initial data on trem-cel from the VBP101 clinical trial and initial
12
clinical data from the VCAR33ALLO program prior to IND submission for the trem-cel + VCAR33 Treatment System. However, the VBP301 protocol allows for patients who have received a trem-cel transplant on the VBP101 study to enroll in VBP301 and receive VCAR33ALLO. This may provide valuable early insights into the potential of the trem-cel + VCAR33 Treatment System to enable a more potent therapy and durable responses post-transplant.
In September 2024, we announced a new preclinical asset, VADC45, which has a number of potential opportunities in oncology, gene therapy, and autoimmune disorders. VADC45 is an ADC designed to target the CD45 protein. CD45 is a well-validated target for a wide variety of blood cancers with clinical proof of concept. The linker-payload used in VADC45 is also clinically validated. VADC45 has the potential to treat a number of diseases, including treatment of hematologic malignancies, as a targeted conditioning agent for gene therapies such as for sickle cell disease, holistic immune reset for autoimmune disorders, and for our approach of combining this asset with epitope modification of CD45 to shield healthy stem cells. We already have robust preclinical data for VADC45 and are progressing IND-enabling studies to enable future Phase 1 studies.
We operate an in-house clinical manufacturing facility in Cambridge, Massachusetts to support development of our shielded transplants and CAR-T therapeutic candidates for patients with blood cancers. While this facility is now operational, we continue to rely on third-party contract manufacturers for our required raw materials, manufacturing devices, active pharmaceutical ingredients and finished product for our research and clinical manufacturing. Since our inception in December 2015, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring or discovering product candidates, establishing and protecting our intellectual property portfolio, developing and progressing our product candidates and preparing for clinical trials, establishing arrangements with third parties for the manufacture of our product candidates and component materials, building out our internal clinical manufacturing facility, and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Through September 30, 2024, we funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $464.4 million.
We have incurred significant operating losses since inception, including net losses of $27.6 million and $86.2 million, respectively, for the three and nine months ended September 30, 2024, and $117.9 million for the year ended December 31, 2023. As of September 30, 2024, we had an accumulated deficit of $426.3 million.
As of September 30, 2024, we had cash, cash equivalents and marketable securities of $62.8 million. We expect that our cash, cash equivalents and marketable securities at September 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025.
Critical Accounting Estimates
Our management’s discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with United States generally accepted accounting principles. The preparation of our condensed consolidated financial statements requires us to make judgments and estimates that affect the reported amounts of assets, liabilities, costs, and expenses, and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events, and various other factors that we believe to be reasonable under the circumstances. Actual results may differ from these estimates under different assumptions or conditions. On an ongoing basis, we evaluate our judgments and estimates in light of changes in circumstances, facts, and experience. The effects of material revisions in estimates, if any, will be reflected in the condensed consolidated financial statements prospectively from the date of change in estimates. There have been no material changes to our critical accounting estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2023 Annual Report.
Financial Operations Overview
Revenue
We have not generated any revenue since our inception and do not expect to generate any revenue from the sale of products in the near future, if at all. If our development efforts for our product candidates are successful and result in marketing approval, or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from a combination of product sales or payments from such agreements.
13
Expenses
Research and Development Expenses
Research and development expenses consist primarily of external and internal expenses incurred in connection with our research and development activities, including our drug discovery efforts and the development of our product candidates. External expenses include:
Internal expenses include:
We expense research and development costs as incurred. We recognize external development costs based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our condensed consolidated financial statements as prepaid expenses or accrued research and development expenses. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized, even when there is no alternative future use for the research and development. The capitalized amounts are expensed as the related goods are delivered or the services are performed.
A significant portion of our research and development costs have been external costs, which we track by stage of development, preclinical or clinical. However we do not track our internal research and development expenses on a program specific basis because these costs are deployed across multiple projects and, as such, are not separately classified.
Research and development activities are central to our business model. We expect that our research and development expenses will increase significantly for the foreseeable future as we continue to identify and develop product candidates, particularly as more of our product candidates move into clinical development and later stages of clinical development.
The successful development of our product candidates in the future is highly uncertain. Therefore, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development and commercialization of any of our product candidates. We are also unable to predict when, if ever, material net cash inflows will commence from the sale of our product candidates, if approved. This is due to the numerous risks and uncertainties associated with developing product candidates, many of which are outside of our control, including the uncertainty of:
14
Any changes in the outcome of any of these variables could mean a significant change in the costs and timing associated with the development of our product candidates.
General and Administrative Expenses
General and administrative expenses consist primarily of personnel-related costs, including salaries, bonuses, benefits and stock-based compensation expenses for employees involved in our executive, finance, corporate, business development and administrative functions, as well as expenses for outside professional services, including legal, audit, accounting and tax-related services and other consulting fees, facility-related expenses, which include depreciation costs and other allocated expenses for rent and maintenance of facilities, insurance costs, recruiting costs, travel expenses and other general administrative expenses.
We expect that our general and administrative expenses will increase as our business expands and we hire additional personnel to support our continued research and development activities, including our clinical programs.
Other Income
Interest Income
Interest income consists of interest income earned on our cash, cash equivalents, restricted cash and marketable securities held in financial institutions.
Results of Operations
Comparison of Three and Nine Months Ended September 30, 2024 and 2023
The following table summarizes our results of operations for the periods indicated (amounts in thousands):
|
|
Three Months Ended |
|
|
|
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
Operating expenses: |
|
|
|
|
|
|
|
|
|
|||
Research and development |
|
$ |
21,817 |
|
|
$ |
27,606 |
|
|
$ |
(5,789 |
) |
General and administrative |
|
|
6,696 |
|
|
|
7,710 |
|
|
|
(1,014 |
) |
Total operating expenses |
|
|
28,513 |
|
|
|
35,316 |
|
|
|
(6,803 |
) |
Loss from operations |
|
|
(28,513 |
) |
|
|
(35,316 |
) |
|
|
6,803 |
|
Other income: |
|
|
|
|
|
|
|
|
|
|||
Interest income |
|
|
954 |
|
|
|
2,126 |
|
|
|
(1,172 |
) |
Total other income |
|
|
954 |
|
|
|
2,126 |
|
|
|
(1,172 |
) |
Net loss |
|
$ |
(27,559 |
) |
|
$ |
(33,190 |
) |
|
$ |
5,631 |
|
15
|
|
Nine Months Ended |
|
|
|
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
Operating expenses: |
|
|
|
|
|
|
|
|
|
|||
Research and development |
|
$ |
67,962 |
|
|
$ |
73,418 |
|
|
$ |
(5,456 |
) |
General and administrative |
|
|
21,912 |
|
|
|
24,494 |
|
|
|
(2,582 |
) |
Total operating expenses |
|
|
89,874 |
|
|
|
97,912 |
|
|
|
(8,038 |
) |
Loss from operations |
|
|
(89,874 |
) |
|
|
(97,912 |
) |
|
|
8,038 |
|
Other income: |
|
|
|
|
|
|
|
|
|
|||
Interest income |
|
|
3,672 |
|
|
|
6,310 |
|
|
|
(2,638 |
) |
Total other income |
|
|
3,672 |
|
|
|
6,310 |
|
|
|
(2,638 |
) |
Net loss |
|
$ |
(86,202 |
) |
|
$ |
(91,602 |
) |
|
$ |
5,400 |
|
Research and Development Expenses
The following table summarizes our research and development expenses incurred for the periods indicated (amounts in thousands):
|
|
Three Months Ended |
|
|
|
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
External expenses |
|
$ |
7,856 |
|
|
$ |
14,550 |
|
|
$ |
(6,694 |
) |
Internal expenses: |
|
|
|
|
|
|
|
|
|
|||
Personnel expenses (including stock-based compensation) |
|
|
9,409 |
|
|
|
9,143 |
|
|
|
266 |
|
Manufacturing, facilities, and other expenses |
|
|
4,552 |
|
|
|
3,913 |
|
|
|
639 |
|
Total research and development expenses |
|
$ |
21,817 |
|
|
$ |
27,606 |
|
|
$ |
(5,789 |
) |
|
|
Nine Months Ended |
|
|
|
|
||||||
|
|
2024 |
|
|
2023 |
|
|
Change |
|
|||
External expenses |
|
$ |
24,449 |
|
|
$ |
34,568 |
|
|
$ |
(10,119 |
) |
Internal expenses: |
|
|
|
|
|
|
|
|
|
|||
Personnel expenses (including stock-based compensation) |
|
|
29,202 |
|
|
|
27,566 |
|
|
|
1,636 |
|
Manufacturing, facilities, and other expenses |
|
|
14,311 |
|
|
|
11,284 |
|
|
|
3,027 |
|
Total research and development expenses |
|
$ |
67,962 |
|
|
$ |
73,418 |
|
|
$ |
(5,456 |
) |
Research and development expenses were $21.8 million for the three months ended September 30, 2024, compared to $27.6 million for the three months ended September 30, 2023. The decrease of $5.8 million was due to a decrease in license payments, preclinical expenses, and manufacturing starting materials, offset in part by an increase in clinical trial costs to support our trem-cel and VCAR33ALLO programs.
Research and development expenses were $68.0 million for the nine months ended September 30, 2024, compared to $73.4 million for the nine months ended September 30, 2023. The decrease of $5.5 million was primarily due to a decrease in license payments, preclinical expenses, and stock-based compensation expense, partially offset by an increase in personnel-related costs driven by additional headcount to support our clinical and manufacturing activities and an increase in clinical trial costs to support our trem-cel and VCAR33ALLO programs.
General and Administrative Expenses
General and administrative expenses were $6.7 million for the three months ended September 30, 2024, compared to $7.7 million for the three months ended September 30, 2023. The decrease of $1.0 million was primarily due to a decrease in professional fees and stock-based compensation expense.
General and administrative expenses were $21.9 million for the nine months ended September 30, 2024, compared to $24.5 million for the nine months ended September 30, 2023. The decrease of $2.6 million was primarily due to a decrease in professional fees and stock-based compensation expense, partially offset by an increase in personnel costs.
16
Other Income
Other income decreased by $1.2 million during the three months ended September 30, 2024, compared to the three months ended September 30, 2023. Other income decreased by $2.6 million for the nine months ended September 30, 2024, compared to the nine months ended September 30, 2023. The decrease in other income in both periods was due to decreases in interest earned from our cash, cash equivalents, restricted cash and marketable securities as the Company held lower balances of cash, cash equivalents and marketable securities.
Liquidity and Capital Resources
Sources of Liquidity
Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and have received aggregate net proceeds from financing transactions of approximately $464.4 million as of September 30, 2024.
In order to fund our future operations, including our planned clinical trials, on March 14, 2022, we filed a universal shelf registration statement (the “Shelf Registration Statement”), to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of September 30, 2024, $274.4 million remains available under this Shelf Registration Statement, including $119.7 million reserved for at-the market offerings discussed below.
At-the-Market Sales Agreement
In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 249,096 shares of our common stock under the Stifel ATM Facility during the nine months ended September 30, 2024 at a weighted-average price per share of $1.64 for aggregate net proceeds of $0.4 million, after deducting commissions. As of September 30, 2024, $119.7 million remained available to be sold under the Stifel ATM Facility.
Cash Requirements
As of September 30, 2024, there were no material changes in our short-term and long-term cash requirements from those disclosed in the “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our 2023 Annual Report.
As of September 30, 2024, we had cash, cash equivalents and marketable securities of $62.8 million. We will need to raise additional capital to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us.
Based on our current business plan and current capital resources, management has concluded that there is substantial doubt regarding our ability to continue as a going concern. We expect that our existing cash, cash equivalents and marketable securities at September 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.
We expect our expenses to increase substantially if, and as, we:
17
Developing pharmaceutical products, including conducting preclinical studies and clinical trials, is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval for any product candidates or generate revenue from the sale of any product candidate for which we may obtain marketing approval. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that we do not expect to be commercially available for at least several years, if ever.
As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the public or private sale of our equity, government or private party grants, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions. To the extent that we raise additional capital through the sale of our equity or convertible debt securities, including through the use of the Stifel ATM Facility, the ownership interest of our shareholders will be or could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders. Debt financing and equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we are unable to obtain additional funding, we could be forced to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or any commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations. If we raise funds through strategic collaborations or other similar arrangements with third parties, we may have to relinquish valuable rights to our platform technology, future revenue streams, research programs or product candidates or may have to grant licenses on terms that may not be favorable to us and/or may reduce the value of our common stock. Our ability to raise additional funds may be adversely impacted by worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from geopolitical tensions and adverse macroeconomic conditions or otherwise. Because of the numerous risks and uncertainties associated with product development, we cannot predict the timing or amount of increased expenses, and there is no assurance that we will ever be profitable or generate positive cash flow from operating activities.
We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements that have or are reasonably likely to have a material current or future effect on our financial condition, revenues or expenses, results of operations, liquidity, cash requirements or capital resources.
Cash Flows
The following table provides information regarding our cash flows for the periods presented (in thousands):
|
|
Nine Months Ended September 30, |
|
|||||
|
|
2024 |
|
|
2023 |
|
||
Net cash used in operating activities |
|
$ |
(75,597 |
) |
|
$ |
(76,266 |
) |
Net cash provided by investing activities |
|
|
96,929 |
|
|
|
60,673 |
|
Net cash provided by financing activities |
|
|
106 |
|
|
|
2,913 |
|
Net increase (decrease) in cash, cash equivalents and restricted cash equivalents |
|
$ |
21,438 |
|
|
$ |
(12,680 |
) |
Operating Activities
Net cash used in operating activities was $75.6 million for the nine months ended September 30, 2024, reflecting a net loss of $86.2 million and net cash used of $2.8 million for operating assets and liabilities, which were offset by non-cash charges of $13.4 million. The non-cash charges primarily consisted of stock-based compensation expense of $8.1 million, non-cash lease expense of $3.8 million and depreciation expense of $2.7 million, offset by $1.1 million of non-cash interest earned on marketable securities.
Net cash used in operating activities was $76.3 million for the nine months ended September 30, 2023, reflecting a net loss of $91.6 million, offset by non-cash adjustments to operating assets and liabilities of $1.4 million and non-cash charges of $13.9 million. The non-cash charges primarily consisted of stock-based compensation expense of $11.5 million, non-cash lease expense of $3.5 million and depreciation expense of $2.6 million, offset by $3.7 million of non-cash interest earned on marketable securities.
18
The $0.7 million decrease in net cash used in operating activities for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily due to a decrease in research and development expenses offset by differences in the timing of payments for costs incurred during each respective period.
Investing Activities
Net cash provided by investing activities was $96.9 million for the nine months ended September 30, 2024, which consisted of proceeds of $107.0 million from the maturity of marketable securities, offset by purchases of $9.9 million of marketable securities and $0.2 million of property and equipment. Net cash provided by investing activities was $60.7 million for the nine months ended September 30, 2023, which consisted of proceeds of $120.0 million from the maturity of marketable securities, offset by purchases of $58.4 million of marketable securities and $1.0 million of property and equipment.
Financing Activities
Net cash provided by financing activities was $0.1 million for the nine months ended September 30, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.3 million and proceeds from exercise of stock options and issuance of shares under the ESPP of $0.1 million, offset by $0.3 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $2.9 million for the nine months ended September 30, 2023, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $4.6 million, and proceeds from stock option exercises and purchases of common stock under the ESPP of $0.2 million, offset by the payment of $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022, and $1.2 million of taxes paid related to net share settlement of equity awards.
Contractual Obligations and Other Commitments
Contractual obligations relate to future minimum lease payments for existing non-cancellable leases primarily relating to corporate office and laboratory real estate, with terms expiring through February 2030. During the nine months ended September 30, 2024, there were no significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2023 Annual Report.
Other commitments include license and collaboration agreements we have entered into with certain parties. Such arrangements require ongoing payments, including payments upon the achievement of certain development, regulatory and commercial milestones, receipt of sublicense income, as well as royalties on commercial sales. Payments under these arrangements are expensed as incurred.
We also have agreements with certain vendors for various services, including services related to clinical operations and support, which we are not contractually able to terminate for convenience and avoid any and all future obligations to the vendors. Under such agreements, we are contractually obligated to make certain payments to vendors to reimburse them for their unrecoverable outlays incurred prior to cancellation. The exact amounts of such obligations are dependent on the timing of termination and the exact terms of the relevant agreement and cannot be reasonably estimated. We do not include these payments in this summary as they are not fixed and estimable.
Recent Accounting Pronouncements
There are no new significant recent accounting pronouncements which may materially impact our financial statements.
Item 3. Quantitative and Qualitative Disclosures About Market Risk.
We are a smaller reporting company as defined by Item 10 of Regulation S-K and are not required to provide the information otherwise required under this item.
Item 4. Controls and Procedures.
Evaluation of Disclosure Controls and Procedures
Our management, with the participation of our Chief Executive Officer and our Chief Financial Officer, evaluated, as of the end of the period covered by this Quarterly Report on Form 10-Q, the effectiveness of our disclosure controls and procedures. The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”), means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports
19
that it files or submits under the Exchange Act is accumulated and communicated to the company’s management, including its principal executive and principal financial officers, or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure. 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 our management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.
Based on the evaluation of our disclosure controls and procedures as of September 30, 2024, our Chief Executive Officer and our Chief Financial Officer concluded that our disclosure controls and procedures as of such date were effective at the reasonable assurance level.
Changes in Internal Control over Financial Reporting
There were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) during the quarter ended September 30, 2024 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
20
PART II—OTHER INFORMATION
Item 1. Legal Proceedings.
We are not currently a party to any material legal proceedings. From time to time, we may become involved in other litigation or legal proceedings relating to claims arising from the ordinary course of business.
Item 1A. Risk Factors.
Our business is subject to risks and events that, if they occur, could adversely affect our financial condition and results of operations and the trading price of our securities. Except as set forth below, there have been no material changes to the risk factors disclosed in “Part I. Item 1A. Risk Factors” in our 2023 Annual Report.
If we fail to satisfy all applicable requirements of Nasdaq and it determines to delist our common stock, the delisting could adversely affect the market liquidity of our common stock and the market price of our common stock could decrease.
To maintain the listing of our common stock on Nasdaq, we are required to meet certain listing requirements, including, a minimum closing bid price of $1.00 per share. On August 29, 2024, we received notice from Nasdaq that we are no longer in compliance with Nasdaq’s Listing Rule 5450(a)(1) because the closing bid price of our common stock had fallen below $1.00 per share for 31 consecutive days. In accordance with Nasdaq Listing Rule 5810(c)(3)(A), we have an initial period of 180 calendar days, or until February 25, 2025 (the “Compliance Date”), to regain compliance with the minimum bid price requirement. To regain compliance, the closing bid price of our common stock must be at least $1.00 per share for a minimum of 10 consecutive business days before the Compliance Date.
If our common stock does not achieve compliance by the Compliance Date, we may be eligible for an additional 180-day period to regain compliance if we apply to transfer the listing of our common stock to the Nasdaq Capital Market. To qualify, we would be required to meet the continued listing requirement for market value of publicly held shares and all other initial listing standards, with the exception of the bid price requirement, and provide written notice to Nasdaq of our intention to cure the deficiency during the second compliance period by effecting a reverse stock split, if necessary.
There can be no assurance that we will maintain compliance with the requirements for listing our common stock on Nasdaq. If we are unable to satisfy the Nasdaq criteria for continued listing, our common stock would be subject to delisting. A delisting of our common stock could negatively impact us by, among other things, (i) reducing the liquidity and market price of our common stock; (ii) reducing the number of investors willing to hold or acquire our common stock, which could negatively impact our ability to raise equity financing; (iii) decreasing the amount of news and analyst coverage of us; (iv) limiting our ability to issue additional securities or obtain additional financing in the future; (v) limiting our ability to use a registration statement to offer and sell freely tradable securities, thereby preventing us from accessing the public capital markets; and (vi) impairing our ability to provide equity incentives to our employees. In addition, delisting from Nasdaq may negatively impact our reputation and, consequently, our business.
Item 5. Other Information
During the quarter ended September 30, 2024, no director or officer, as defined in Rule 16a-1(f),
Appointment of Principal Accounting Officer
As previously disclosed, on June 8, 2024, our board of directors designated Amy Quinlan, our Vice President of Finance, as our interim principal accounting officer, effective as of September 30, 2024. On November 7, 2024, our board of directors removed Ms. Quinlan as interim principal accounting officer and designated Han Choi, M.D., LL.M., our Chief Financial Officer, as our principal accounting officer.
Information regarding Dr. Choi’s background and business experience is set forth under Item 5.02 of our Current Report on Form 8-K (File No. 001-39979) filed with the Securities and Exchange Commission on September 30, 2024, and is incorporated herein by reference. There are no arrangements or understandings between Dr. Choi and any other persons pursuant to which he was selected as an officer of Vor Bio. There are also no family relationships between Dr. Choi and any director or executive officer of Vor Bio, and Dr. Choi has no direct or indirect material interest in any related party transaction required to be disclosed pursuant to Item 404(a) of Regulation S-K.
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We did not enter into, or materially amend, any material plan, contract or arrangement to which Dr. Choi is a party or in which he participates in connection with Dr. Choi’s designation as principal accounting officer, or make or modify any grant or award to Dr. Choi under any such plan, contract or arrangement.
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Item 6. Exhibits.
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Incorporated by Reference |
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Exhibit Number |
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Description |
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Form |
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File No. |
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Exhibit Number |
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Filing Date |
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Filed Herewith |
3.1 |
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Amended and Restated Certificate of Incorporation of the Registrant |
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8-K |
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001-39979 |
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3.1 |
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February 9, 2021 |
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3.2 |
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8-K |
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001-39979 |
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3.2 |
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February 9, 2021 |
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4.1 |
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S-1/A |
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333-252175 |
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4.1 |
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February 1, 2021 |
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4.2 |
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S-1/A |
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333-252175 |
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4.2 |
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February 1, 2021 |
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10.1+ |
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Offer Letter, by and between the Registrant and Han Choi, dated September 19, 2024 |
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X |
31.1 |
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X |
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32.1† |
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X |
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101.INS |
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Inline XBRL Instance Document
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X |
101.SCH |
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Inline XBRL Taxonomy Extension Schema Document
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X |
101.CAL |
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Inline XBRL Taxonomy Extension Calculation Linkbase Document
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X |
101.DEF
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Inline XBRL Taxonomy Extension Definition Linkbase Document
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X |
101.LAB
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Inline XBRL Taxonomy Extension Label Linkbase Document
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X |
101.PRE |
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Inline XBRL Taxonomy Extension Presentation Linkbase Document
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X |
104 |
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The cover page from the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, formatted in Inline XBRL. |
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+ Indicates management contract or compensatory plan.
† The certifications furnished in Exhibit 32.1 hereto are deemed to be furnished with this Quarterly Report on Form 10-Q and will not be deemed to be “filed” for purposes of Section 18 of the Exchange Act. Such certifications will not be deemed to be incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Exchange Act, except to the extent that the Registrant specifically incorporates it by reference.
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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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VOR BIOPHARMA INC. |
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Date: November 7, 2024 |
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By: |
/s/ Robert Ang |
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Robert Ang President and Chief Executive Officer (Principal Executive Officer) |
Date: November 7, 2024 |
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By: |
/s/ Han Choi |
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Han Choi Chief Financial Officer (Principal Financial Officer and Principal Accounting Officer) |
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