Company Overview - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases [3] - The company is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and commercialization [3] Executive Compensation - The Board of Directors granted CEO Jean-Paul Kress an option to purchase 83,296,638 shares of common stock as an inducement for his employment [1] - The stock option has a ten-year term with an exercise price of $0.89 per share, equal to the closing price on June 26, 2025 [2] - The option will vest over a four-year period, with 25% vesting after 12 months and the remaining shares vesting monthly over the following 36 months [2]

Core Insights - Vor Biopharma Inc. has been upgraded by H.C. Wainwright, marking a significant turnaround for the company following the in-licensing of telitacicept [1][4] - The company has entered into a strategic licensing agreement with RemeGen Co., Ltd. for the development and commercialization of telitacicept outside Greater China [2] Financial Details - Vor Biopharma will pay $125 million upfront, which includes $45 million in cash and $80 million in stock warrants, along with potential milestone payments exceeding $4 billion and tiered royalties [3] - The stock price of Vor Biopharma has increased by 49.1%, reaching $1.58 [7] Market Opportunity - The market potential for telitacicept in generalized myasthenia gravis (gMG) is significant, with an estimated 82,700 people in the U.S. affected in 2021, and 75% of these patients receiving treatment [5] - If priced at $225,000 per patient annually and capturing 15% of the U.S. market, peak annual sales for telitacicept could reach $1.8 billion [5] - Additional sales of $2.7 billion could be generated if the drug successfully launches in the EU and Japan, achieving a 10% market share [6] Management Changes - Jean-Paul Kress has been appointed as CEO and Chairman, replacing Dr. Robert Ang, who will remain as a strategic advisor until October [3]

Core Insights - Vor Bio's shares have surged by 265.5% in the past week, significantly outperforming the industry average increase of 0.8% [1] - The company has entered a licensing agreement with RemeGen to develop and commercialize telitacicept in markets outside of China [1][6] Licensing Agreement Details - The licensing deal is valued at $125 million upfront, which includes $45 million in cash and $80 million in stock warrants, along with potential milestone payments exceeding $4 billion and tiered royalties on future sales [6][7] - Vor Bio will focus on developing telitacicept, a dual-target fusion protein that blocks two proteins, BAFF and APRIL, to treat autoimmune diseases [2][7] Clinical Development - A global phase III study for telitacicept in treating generalized myasthenia gravis (gMG) is currently ongoing in the United States, Europe, and South America, with initial data expected in the first half of 2027 [3] Leadership Changes - Vor Bio has appointed Jean-Paul Kress as the new CEO and chairman, following the resignation of the previous CEO, Robert Ang [3][6] Company Outlook - The licensing agreement is seen as a pivotal move that may prevent Vor Bio from shutting down its operations, which was previously announced due to financial difficulties [8]

Core Viewpoint - Rongchang Biopharmaceutical's significant licensing agreement with Vor Bio, valued at up to $4.23 billion, led to an unexpected 18.36% drop in its stock price, raising concerns about the market's perception of the deal [2]. Group 1: Licensing Agreement Details - On June 25, Rongchang Biopharmaceutical announced a licensing agreement with Vor Bio, granting exclusive rights to develop and commercialize the drug Tai Tasi Pi outside of Greater China [2]. - The agreement includes an upfront payment of $125 million, which consists of a $45 million initial payment and $80 million in warrants, along with potential milestone payments of up to $4.1 billion and sales royalties in the high single to double-digit percentage range [2]. Group 2: Product and Financial Performance - Tai Tasi Pi, Rongchang's core innovative drug, received conditional approval in March 2021 and is currently approved for indications such as myasthenia gravis, systemic lupus erythematosus, and rheumatoid arthritis [3]. - In 2024, Rongchang's revenue reached 1.717 billion yuan, a year-on-year increase of 58.54%, although it reported a net loss of 1.468 billion yuan [4]. - The significant revenue growth is attributed to increased sales of Tai Tasi Pi and another product, with R&D expenses amounting to 1.54 billion yuan, representing 89.69% of total revenue [4]. Group 3: Vor Bio Company Concerns - Vor Bio has not generated any revenue since its inception and reported net losses of $118 million and $117 million for 2023 and 2024, respectively, with a cumulative deficit of $457 million as of December 31, 2024 [5]. - The company announced significant layoffs, planning to cut 147 full-time employees, approximately 95% of its workforce, and to gradually shut down its clinical and manufacturing operations [5]. - Vor Bio also experienced major management changes, including the departure of its CFO, Chief Scientific Officer, and head of technical operations [6].

Core Viewpoint - Vor Bio has announced a private placement in public equity financing (PIPE) expected to generate approximately $175 million in gross proceeds to advance its clinical pipeline and for general corporate purposes [1][3]. Group 1: Private Placement Details - Vor Bio will issue prefunded warrants to purchase a total of 700 million shares of common stock at a price of $0.25 per warrant [2]. - The PIPE includes participation from notable investors such as RA Capital Management, Mingxin Capital, Forbion, Venrock Healthcare Capital Partners, Caligan Partners, and NEXTBio [2]. - The expected closing date for the PIPE is June 27, 2025, pending customary closing conditions [3]. Group 2: Warrants and Stockholder Approval - The warrants have an exercise price of $0.0001 per share and will be exercisable upon stockholder approval for the issuance of the underlying shares [4]. - Vor Bio plans to hold a special stockholder meeting to seek approval for the issuance of the shares related to the warrants [4]. Group 3: Company Overview - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, particularly through its novel dual-target fusion protein, telitacicept, which is in Phase 3 clinical development [7].

Core Insights - Vor Bio has secured exclusive global rights (excluding Greater China) to develop and commercialize telitacicept, a dual-target recombinant fusion protein for autoimmune diseases [1][6] - RemeGen has received an initial payment of $125 million, which includes a $45 million upfront payment and $80 million in warrants, along with potential milestones exceeding $4 billion and tiered royalties [1][5] - Jean-Paul Kress, MD, has been appointed as the new CEO and Chairman of Vor Bio, bringing extensive experience in clinical development and commercialization [3][4] Company Developments - Vor Bio is focused on advancing telitacicept through Phase 3 clinical development to address serious autoantibody-driven conditions globally [7] - RemeGen is conducting a global Phase 3 clinical trial for telitacicept, with initial results expected in the first half of 2027 [2][6] - The strategic out-licensing of telitacicept's ex-China rights is aimed at maximizing its clinical and commercial potential on a global scale [5] Product Information - Telitacicept targets key immune pathways by inhibiting BlyS (BAFF) and APRIL, which are critical for B cell survival, thereby reducing autoreactive B cells and autoantibody production [2][5] - In a Phase 3 trial in China for generalized myasthenia gravis, telitacicept showed a 4.8-point improvement in the MG-ADL scale compared to placebo at 24 weeks [5]

PART I. FINANCIAL INFORMATION [Financial Statements (Unaudited)](index=5&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements for the quarter ended March 31, 2025, highlighting a net loss of **$32.5 million** and management's conclusion of substantial doubt about the company's ability to continue as a going concern Condensed Consolidated Balance Sheets (in thousands) | | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $50,047 | $81,949 | | Total current assets | $64,847 | $96,507 | | Total assets | $109,312 | $142,891 | | **Liabilities and Stockholders' Equity** | | | | Total current liabilities | $16,727 | $18,612 | | Total liabilities | $43,243 | $46,227 | | Total stockholders' equity | $66,069 | $96,664 | | Accumulated deficit | $(489,480) | $(456,994) | Condensed Consolidated Statements of Operations (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Research and development | $26,701 | $24,322 | | General and administrative | $6,590 | $8,004 | | **Loss from operations** | **$(33,291)** | **$(32,326)** | | Interest income | $805 | $1,522 | | **Net loss** | **$(32,486)** | **$(30,804)** | | Net loss per share, basic and diluted | $(0.26) | $(0.45) | - On May 8, 2025, the company announced the wind-down of its clinical and manufacturing operations and a plan to reduce its workforce while it explores strategic alternatives to maximize shareholder value[25](index=25&type=chunk)[71](index=71&type=chunk) - Management has concluded that there is substantial doubt regarding the Company's ability to continue as a going concern within one year, citing the need to consummate a strategic transaction or raise additional funding, which is uncertain[28](index=28&type=chunk) - In conjunction with the strategic process, the company announced a workforce reduction of approximately **95%** (147 full-time employees), which is expected to result in about **$10.9 million** in severance costs, primarily in Q2 2025[72](index=72&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=18&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial performance, highlighting the strategic decision made in May 2025 to wind down clinical and manufacturing operations and explore alternatives to maximize shareholder value, alongside significant liquidity challenges and a "going concern" warning [Overview](index=18&type=section&id=Overview) Vor Bio, a clinical-stage company focused on AML, announced on May 8, 2025, it is winding down clinical and manufacturing operations, including a **95%** workforce reduction, while exploring strategic alternatives, having incurred a net loss of **$32.5 million** for Q1 2025 and an accumulated deficit of **$489.5 million** - On May 8, 2025, the company announced it is winding down clinical and manufacturing operations to explore strategic alternatives, including a potential sale, licensing of assets, merger, or other business combination[76](index=76&type=chunk) - A workforce reduction of **147** full-time employees (approx. **95%**) was announced, with expected severance costs of around **$10.9 million**, primarily to be incurred in Q2 2025[76](index=76&type=chunk) - As of March 31, 2025, the company had an accumulated deficit of **$489.5 million** and incurred a net loss of **$32.5 million** for the quarter[77](index=77&type=chunk) [Results of Operations](index=22&type=section&id=Results%20of%20Operations) For Q1 2025, the net loss increased to **$32.5 million** from **$30.8 million** in Q1 2024, primarily due to a **$2.4 million** rise in R&D expenses, partially offset by a **$1.4 million** decrease in G&A expenses Comparison of Operations (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | Change | | :--- | :--- | :--- | :--- | | Research and development | $26,701 | $24,322 | $2,379 | | General and administrative | $6,590 | $8,004 | $(1,414) | | **Loss from operations** | **$(33,291)** | **$(32,326)** | **$(965)** | | **Net loss** | **$(32,486)** | **$(30,804)** | **$(1,682)** | - R&D expenses increased by **$2.4 million**, primarily due to higher clinical trial costs supporting the trem-cel and VCAR33 programs, along with increased lab supplies and consumables[90](index=90&type=chunk) - G&A expenses decreased by **$1.4 million**, mainly due to a reduction in stock-based compensation expense and personnel costs[91](index=91&type=chunk) [Liquidity and Capital Resources](index=24&type=section&id=Liquidity%20and%20Capital%20Resources) As of March 31, 2025, the company held **$60.0 million** in cash, cash equivalents, and marketable securities, which management deems insufficient for the next 12 months, leading to substantial doubt about its going concern ability, further restricted by SEC rules on capital raising due to its public float being below **$75 million** - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities of **$60.0 million**[78](index=78&type=chunk)[98](index=98&type=chunk) - Management concluded there is substantial doubt about the company's ability to continue as a going concern for 12 months from the financial statement issuance date[99](index=99&type=chunk) - The company is subject to SEC's General Instruction I.B.6 to Form S-3, which limits its capacity to sell shares under its shelf registration to one-third of its public float in any twelve-month period, as its public float is less than **$75 million**[96](index=96&type=chunk) [Cash Flows](index=26&type=section&id=Cash%20Flows) In Q1 2025, net cash used in operating activities was **$31.1 million**, while net cash used in investing activities was **$0.2 million**, a significant change from **$44.0 million** provided in Q1 2024 due to non-recurring proceeds from marketable securities Cash Flow Summary (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(31,066) | $(30,465) | | Net cash (used in) provided by investing activities | $(230) | $43,973 | | Net cash (used in) provided by financing activities | $(606) | $96 | - Net cash used in operating activities increased by **$0.6 million**, primarily due to higher R&D expenses and lower stock-based compensation[107](index=107&type=chunk) - The significant change in investing cash flow from Q1 2024 to Q1 2025 was due to **$54.0 million** in proceeds from maturities of marketable securities in the 2024 period, which did not recur in 2025[108](index=108&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=28&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is classified as a smaller reporting company and, as such, is not required to provide the information for this item - As a smaller reporting company, Vor Biopharma is not required to provide quantitative and qualitative disclosures about market risk[116](index=116&type=chunk) [Controls and Procedures](index=28&type=section&id=Item%204.%20Controls%20and%20Procedures) Based on an evaluation as of March 31, 2025, the company's management concluded that its disclosure controls and procedures were effective, with no material changes in internal control over financial reporting during the quarter - The Chief Executive Officer and Chief Financial Officer concluded that the company's disclosure controls and procedures were effective as of March 31, 2025[118](index=118&type=chunk) - No changes in internal control over financial reporting occurred during the quarter ended March 31, 2025, that have materially affected, or are reasonably likely to materially affect, internal controls[119](index=119&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=29&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not currently a party to any material legal proceedings but may become involved in litigation arising from the ordinary course of business from time to time - The company is not currently a party to any material legal proceedings[122](index=122&type=chunk) [Risk Factors](index=29&type=section&id=Item%201A.%20Risk%20Factors) This section highlights significant new and updated risks, including potential Nasdaq delisting for failing to meet the **$1.00** minimum bid price, and uncertainties associated with the strategic review and operational wind-down, such as transaction completion and negative consequences from workforce reduction - The company received a notice from Nasdaq on April 22, 2025, for non-compliance with the minimum bid price of **$1.00** per share, posing a risk of delisting if compliance is not regained by October 20, 2025[124](index=124&type=chunk) - There is no assurance that the strategic review process will result in any transaction, or that any pursued transaction will be completed on attractive terms or at all[127](index=127&type=chunk) - The workforce reduction and operational wind-down carry risks such as loss of institutional knowledge, attrition beyond the intended scope, decreased morale, and potential impairment charges and contract termination costs that could be material[128](index=128&type=chunk)[129](index=129&type=chunk) [Other Information](index=31&type=section&id=Item%205.%20Other%20Information) The company states that during the first quarter of 2025, no director or officer adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement - During the quarter ended March 31, 2025, no director or officer adopted or terminated a "Rule 10b5-1 trading arrangement" or a "non-Rule 10b5-1 trading arrangement"[130](index=130&type=chunk) [Exhibits](index=32&type=section&id=Item%206.%20Exhibits) This section provides a list of all exhibits filed with the Quarterly Report on Form 10-Q, including the company's Amended and Restated Certificate of Incorporation, Bylaws, and certifications by the Principal Executive and Financial Officers - This section lists exhibits filed with the Form 10-Q, including corporate governance documents, officer certifications, and Inline XBRL data files[132](index=132&type=chunk)

Core Viewpoint - Vor Bio is exploring strategic alternatives to maximize shareholder value due to challenging fundraising conditions and current clinical data [1][2]. Group 1: Strategic Alternatives - The strategic alternatives being considered include potential asset sales, licensing, company sale, business combinations, mergers, or other strategic actions [2]. - Vor Bio has initiated a wind-down of its clinical and manufacturing operations, including ongoing clinical trials, which is not related to safety concerns regarding its product candidates [2]. Group 2: Workforce and Financials - The company has implemented a workforce reduction of approximately 95%, incurring costs of about $10.9 million, and will retain around 8 employees for compliance and strategic exploration [3]. - As of December 31, 2024, Vor Bio had cash, cash equivalents, and marketable securities totaling $91.9 million [5]. Group 3: Timeline and Legal Advisory - Vor Bio does not have a defined timeline for the exploration of strategic alternatives and will not disclose further developments unless a specific action is approved by the Board of Directors [4]. - The company has retained Cooley LLP as its legal advisor for this strategic process [4].

Core Insights - Vor Bio reported financial results for Q4 and full year 2024, highlighting advancements in their cell and genome engineering platform and clinical trials for trem-cel and VCAR33 [1][2] Corporate Updates - Trem-cel is being developed as a shielded transplant for patients with AML and MDS, genetically engineered to remove CD33, allowing for targeted therapies post-transplant [3] - VCAR33 is a transplant donor-derived anti-CD33 CAR-T cell therapy for AML patients who have relapsed after standard care or trem-cel transplant [5] - The combination of trem-cel and VCAR33 has the potential to improve patient outcomes in AML and set a new standard of care for high relapse risk patients [6][10] Clinical Trials and Data Updates - Upcoming clinical data updates for VCAR33 are expected in the first half of 2025, while updates for trem-cel + Mylotarg are anticipated in the second half of 2025 [7][16] - The latest data from the VBP101 trial showed durable engraftment and improved relapse-free survival in patients treated with trem-cel and Mylotarg [8] - Enrollment in the VBP301 study is ongoing, with encouraging CAR-T expansion data reported [9] Financial Position - Vor Bio completed a $55.6 million private placement in December 2024, enhancing its financial position [11] - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $91.9 million, projected to fund operations into Q1 2026 [16] - R&D expenses for Q4 2024 were $25.3 million, up from $20.9 million in Q4 2023, while total operating expenses for the year were $121.2 million, down from $126.0 million in 2023 [16][21] Board and Leadership Changes - Erez Kalir was appointed to Vor Bio's Board of Directors as part of the recent financing transaction, bringing extensive experience in life sciences and technology [12]

Financial Performance - Vor Bio reported a net loss of $116.9 million for the year ended December 31, 2024, compared to a net loss of $117.9 million for 2023, reflecting a decrease of $0.9 million [478][491]. - Total operating expenses decreased to $121.2 million in 2024 from $126.0 million in 2023, a reduction of $4.9 million [491]. - Interest income for 2024 was $4.3 million, down from $8.2 million in 2023, reflecting a decrease of $3.9 million [491]. - General and administrative expenses decreased to $27.9 million for the year ended December 31, 2024, from $31.7 million in 2023, a reduction of $3.8 million [493]. - Other income decreased by $3.9 million for the year ended December 31, 2024, primarily due to lower interest received from cash and marketable securities [494]. - The company reported a net cash used in operating activities of $99.7 million for the year ended December 31, 2024, compared to $100.3 million in 2023, reflecting a decrease of $0.6 million [506][507]. Cash and Funding - Cash, cash equivalents, and marketable securities amounted to $91.9 million as of December 31, 2024, expected to fund operations into the first quarter of 2026 [479]. - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $91.9 million, necessitating additional capital to fund future operations [499]. - Net cash provided by investing activities was $96.9 million for the year ended December 31, 2024, compared to $71.0 million in 2023, indicating an increase of $25.9 million [509]. - Net cash provided by financing activities was $53.4 million for the year ended December 31, 2024, significantly up from $2.9 million in 2023 [510]. - The company raised approximately $52.7 million in a private placement on December 30, 2024, with warrants potentially generating an additional $58.5 million if exercised [498]. - The company has $274.4 million remaining available under its universal shelf registration statement as of December 31, 2024, which is set to expire on March 18, 2025 [496]. - The company anticipates substantial additional funding will be required to support ongoing operations and growth strategies, with potential dilution of existing shareholders' interests [502]. Research and Development - Research and development expenses totaled $93.3 million for the year ended December 31, 2024, a slight decrease of $1.0 million from $94.3 million in 2023, primarily due to reduced pre-clinical costs [492]. - The VBP101 clinical trial for trem-cel included 25 patients, with 100% achieving primary neutrophil engraftment and a median engraftment time of 9.5 days [472]. - Trem-cel demonstrated preliminary evidence of improved relapse-free survival, with a median follow-up duration of 7.4 months [472]. - The company plans to initiate a Phase 1 clinical trial for the trem-cel+VCAR33 Treatment System in the second half of 2025 [475]. - VADC45, a new preclinical asset targeting CD45, has potential applications in oncology and autoimmune disorders, with robust preclinical data already available [475][476]. Company Status and Compliance - The company has an accumulated deficit of $457.0 million as of December 31, 2024 [478]. - The company expects to continue incurring significant costs associated with operating as a public company and developing pharmaceutical products, which may delay revenue generation for several years [501][502]. - The company may cease to qualify as an emerging growth company by December 31, 2026, or earlier if total annual gross revenues exceed $1.235 billion [520]. - The market value of the company's common stock held by non-affiliates must exceed $700 million for it to be deemed a "large accelerated filer" under SEC rules [520]. - The company has issued more than $1.0 billion of non-convertible debt over the prior three-year period, which could affect its status [520]. - As a smaller reporting company, the company may present only the two most recent fiscal years of audited consolidated financial statements in its Annual Report [521]. - The company has reduced disclosure obligations regarding executive compensation as a smaller reporting company [522]. - The company is exempt from compliance with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act [522]. - The company is not required to provide quantitative and qualitative disclosures about market risk for this reporting period [523].