Summary of Vor Biopharma Conference Call Company Overview - **Company**: Vor Biopharma (NasdaqGS:VOR) - **New Direction**: The company is undergoing a transformation referred to as "Vor 2.0," focusing on a late-stage asset in immunology, telitacicept, licensed from RemeGen, a Chinese biotech firm [4][5] Key Points and Arguments Asset and Mechanism - **Telitacicept**: A dual BAFF/APRIL inhibitor that normalizes the immune system and provides durable efficacy in autoimmune diseases, addressing limitations of current therapies [4][5] - **Data**: Over 70,000 patients treated in China and around 3,000 in clinical trials, with proven efficacy across three indications and two additional PLAs being filed [4][5] - **Market Opportunity**: Targeting myasthenia gravis and Sjögren's disease, both of which have high unmet medical needs [5] Competitive Advantage - **Differentiation**: Telitacicept is the most advanced BAFF/APRIL inhibitor, closely resembling the wild type TACI receptor, which enhances its efficacy and safety [6][7] - **Holistic Approach**: The drug targets both upstream and downstream B-cell pathways, potentially offering a more comprehensive treatment compared to FCRN antagonists and complement inhibitors [10][11] Myasthenia Gravis (MG) Development - **Patient Population**: Approximately 90,000 patients in the U.S. with significant unmet medical needs despite existing therapies [10] - **Phase 3 Trial**: Previous trials showed unprecedented activity on the primary endpoint (MGADL), with long-term data indicating sustained benefits [11][12] - **Global Phase 3 Trial**: Currently enrolling patients, leveraging data from China while ensuring quality and replicability in Western populations [17][20] Sjögren's Disease Development - **Patient Demographics**: Affects around 300,000 patients in the U.S., predominantly women, with significant symptoms and high rates of underdiagnosis [23][24] - **Phase 2 Results**: Telitacicept has shown best-in-disease results in previous trials, with a focus on multi-component scoring systems [25][26] - **Market Potential**: High unmet medical need and potential for multiple therapies in the space, with telitacicept positioned as a leading candidate [28] Financial Overview - **Capital Structure**: As of June, Vor Biopharma has $190 million in cash, providing a runway into Q1 2027, sufficient to support ongoing studies [40] - **Equity Units**: 1.2 billion equity units fully diluted [40] Upcoming Catalysts - **Data Presentations**: Anticipated long-term data from the MG study at AANEM and phase 3 data for Sjögren's at a major rheumatology conference [41] - **IgA Data**: Upcoming presentation of phase 3 data from China, showcasing consistent efficacy across autoimmune diseases [41] Conclusion - **Strategic Focus**: Vor Biopharma aims to change the standard of care in autoimmune diseases through its innovative approach and strong pipeline, with a disciplined strategy for capital allocation and development opportunities [34][41]

Core Insights - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases [2] - The company is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development [2] Event Participation - Vor Bio will participate in a fireside chat at the Stifel 2025 Virtual Immunology and Inflammation Forum on September 15, 2025 [1] - The fireside chat will take place from 4:00-4:25 pm ET and will be available via live webcast and archived replay on the company's website [1]

Summary of Vor Biopharma (VOR) Conference Call - September 02, 2025 Company Overview - **Company**: Vor Biopharma (VOR) - **Key Asset**: Telitacicept (Teli), a BAFF inhibitor for autoimmune diseases - **Partnership**: RemeGen, a large Chinese biopharma company Industry Insights - **Market Dynamics**: - Significant licensing deals for Chinese assets in the biotech sector - China has a rapidly growing biotech industry, with a pipeline comparable to the U.S. [6][11] - China Health 2030 initiative aims to streamline regulatory processes and clinical trials [12] Core Points and Arguments - **Asset Strength**: - Teli is a dual BAFF and APRIL inhibitor, addressing unmet needs in autoimmune diseases [6][16] - Over 70,000 patients treated in China, providing a robust real-world data set [7][25] - Advanced late-stage product with extensive clinical trial data [7][15] - **Market Potential**: - Myasthenia Gravis (MG) is identified as a key indication with a projected U.S. market of $4 billion, expected to grow to $10 billion by 2030 [19] - Sjögren's syndrome is highlighted as a significant opportunity due to lack of targeted treatments [33] - **Competitive Landscape**: - Teli is positioned as the most advanced BAFF/APRIL inhibitor globally, with a strong safety profile and efficacy data [25][30] - The product aims to address the shortcomings of existing therapies, particularly FCRN inhibitors [26][30] - **Regulatory and Development Strategy**: - Plans to initiate a global Phase III trial for Sjögren's syndrome, already approved by the FDA [35][44] - Ongoing Phase III trial for MG with promising long-term data expected [47] Additional Important Insights - **Data Quality and Transferability**: - Concerns about the transferability of Chinese clinical data to global populations have diminished, with increasing confidence in data integrity [10][11] - The company emphasizes the quality of its partnership with RemeGen, which has strong manufacturing capabilities [13][30] - **Future Milestones**: - Key upcoming data releases include Phase III results for Sjögren's syndrome and long-term MG data [47][48] - The company has approximately $199 million in cash, positioning it well for upcoming trials [49] - **Geopolitical Considerations**: - The partnership with RemeGen is viewed positively despite geopolitical tensions, with a focus on mutual benefits in innovation and market access [53][55] - **Immunology Pipeline**: - There is a growing interest in immunology deals from China, with a significant number of innovative modalities in development [56][57] This summary encapsulates the key points discussed during the conference call, highlighting Vor Biopharma's strategic positioning, market opportunities, and future plans in the context of the evolving biotech landscape.

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] Leadership Appointment - Adi Osovsky, S.J.D. has been appointed as General Counsel, bringing 17 years of experience in corporate counsel and law firm roles within the biotechnology and pharmaceutical sectors [1][2] - Dr. Osovsky previously served as Executive Vice President and Head of Legal & Corporate Secretary at iTeos Therapeutics, overseeing corporate legal and compliance activities [2] - Her experience includes senior legal roles at Sarepta Therapeutics, where she supported corporate transactions and governance during a period of rapid growth [2] Strategic Importance - The appointment of Dr. Osovsky is seen as crucial for Vor Bio's growth, particularly as the company advances its late-stage clinical programs [2] - The CEO, Jean-Paul Kress, emphasized that Dr. Osovsky's expertise in governance, compliance, and strategic transactions will be invaluable for the company's future [2]

Core Insights - Vor Bio's collaborator, RemeGen, achieved the primary endpoint in a Phase 3 clinical study of telitacicept for IgA nephropathy, demonstrating a 55% reduction in proteinuria at 39 weeks compared to placebo [2][3][4] - Telitacicept shows consistent disease-modifying activity across various autoimmune diseases, including myasthenia gravis, Sjögren's disease, and IgA nephropathy, indicating its potential as a best-in-class treatment [3][6] - The study involved 318 adult patients and utilized UPCR as a regulatory marker, reinforcing telitacicept's strong dataset and safety profile [3][4] Company Overview - Vor Bio is a clinical-stage biotechnology company focused on advancing telitacicept, a dual-target fusion protein, through Phase 3 clinical development for autoimmune diseases [5] - RemeGen plans to submit a Biologics License Application (BLA) for telitacicept in IgAN, which would mark its fifth approved indication in China [4][7] Product Details - Telitacicept is designed to treat autoimmune diseases by inhibiting BLyS and APRIL, which are critical for B cell survival, thereby reducing autoreactive B cells and autoantibody production [6][9] - The drug is already approved in China for systemic lupus erythematosus, rheumatoid arthritis, and generalized myasthenia gravis, with ongoing global trials to support further approvals [7][8] Disease Context - IgA nephropathy is a leading cause of chronic kidney disease, with significant unmet needs for effective therapies, as current treatments primarily slow disease progression without addressing underlying causes [8][9] - The overproduction of galactose-deficient IgA1 is identified as a central driver of IgAN, with BAFF and APRIL promoting its production [9]

Core Insights - Vor Bio, a clinical-stage biotechnology company, is focused on transforming the treatment of autoimmune diseases through innovative therapies [3] Stock Options and RSUs - The Compensation Committee granted stock options to purchase 6,959,013 shares and restricted stock units (RSUs) for 1,491,217 shares to seven new employees as part of the Inducement Plan [1] - Stock options have a ten-year term with exercise prices between $2.11 and $2.04 per share, equal to the closing price on grant dates [2] - Both stock options and RSUs will vest over four years, with 25% vesting after 12 months and the remainder vesting monthly or quarterly thereafter, contingent on continued employment [2] Company Overview - Vor Bio is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development to address serious autoantibody-driven conditions globally [3]

Core Insights - Vor Bio's stock is experiencing an increase, trading at $2.15, up 29.52% during premarket sessions, with a session volume of 10.83 million shares compared to an average of 17.99 million shares [1][6] - The company’s collaborator, RemeGen Co. Ltd, has successfully achieved the primary endpoint in a Phase 3 clinical study in China for telitacicept, targeting primary Sjögren's disease [1][2] - Telitacicept has shown a favorable safety profile and is set to have its fourth approved indication in China, with a Biologics License Application (BLA) planned for submission [3] Clinical Study Results - The Phase 3 study demonstrated significant improvement in disease activity, measured by a reduction in the EULAR Sjögren's syndrome disease activity index (ESSDAI) [2][5] - Novartis AG also reported positive topline data from two Phase 3 trials for ianalumab, which met the primary endpoint of improving disease activity [4][5] - Johnson & Johnson's investigational nipocalimab received Fast Track designation from the FDA for moderate-to-severe Sjögren's disease, showing over 70% relative average improvement in systemic disease activity [5][6]

Core Insights - Telitacicept shows potential as a best-in-disease treatment for primary Sjögren's disease, achieving the primary endpoint in a Phase 3 clinical study in China [2][3] - The drug targets both BAFF and APRIL, addressing the autoimmune signaling cascade, which may allow for disease modification rather than just symptom management [3] - Vor Bio plans to submit a Biologics License Application (BLA) for telitacicept in primary Sjögren's disease, marking its fourth approved indication in China [4] Company Overview - Vor Bio is a clinical-stage biotechnology company focused on advancing telitacicept, a dual-target fusion protein, through Phase 3 clinical development to treat autoimmune diseases [5] - The company aims to transform treatment options for serious autoantibody-driven conditions globally [5] Product Details - Telitacicept is designed to selectively inhibit BLyS (BAFF) and APRIL, reducing autoreactive B cells and autoantibody production, which are key drivers of autoimmune diseases [6] - The drug is already approved in China for systemic lupus erythematosus, rheumatoid arthritis, and generalized myasthenia gravis [7] Disease Context - Primary Sjögren's disease is a chronic autoimmune condition characterized by overactive B cells, leading to inflammation and damage to moisture-producing glands [8] - The disease is often underdiagnosed, with significant impacts on patients' daily lives, and currently lacks systemic disease-modifying therapies [10]

PART I. FINANCIAL INFORMATION This section presents the unaudited condensed consolidated financial statements, management's discussion and analysis, and disclosures on market risk and internal controls [Item 1. Financial Statements (Unaudited)](index=9&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements, including the balance sheets, statements of operations and comprehensive loss, statements of stockholders' equity (deficit), and statements of cash flows, along with their accompanying notes, providing a detailed financial overview for the periods ended June 30, 2025, and December 31, 2024 [Condensed Consolidated Balance Sheets](index=9&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheets (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | **Assets** | | | | Cash and cash equivalents | $190,574 | $81,949 | | Restricted cash equivalents | $2,413 | — | | Marketable securities | $9,991 | $9,977 | | Total current assets | $204,927 | $96,507 | | Total assets | $205,371 | $142,891 | | **Liabilities** | | | | Accounts payable | $669 | $1,505 | | Accrued liabilities | $58,104 | $12,892 | | Warrant liabilities | $1,652,298 | — | | Total liabilities | $1,711,071 | $46,227 | | **Stockholders' Equity (Deficit)** | | | | Total stockholders' equity (deficit) | $(1,505,700) | $96,664 | | Accumulated deficit | $(2,063,149) | $(456,994) | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share amounts) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development expenses | $261,499 | $21,823 | $288,200 | $46,145 | | General and administrative expenses | $12,785 | $7,212 | $19,375 | $15,216 | | Total operating expenses | $274,284 | $29,035 | $307,575 | $61,361 | | Loss from operations | $(274,284) | $(29,035) | $(307,575) | $(61,361) | | Interest income | $537 | $1,196 | $1,342 | $2,718 | | Change in fair value of warrant liabilities | $(1,299,922) | — | $(1,299,922) | — | | Net loss | $(1,573,669) | $(27,839) | $(1,606,155) | $(58,643) | | Net loss per share, basic and diluted | $(12.56) | $(0.41) | $(12.84) | $(0.86) | | Weighted-average common shares outstanding | 125,271,447 | 68,299,170 | 125,049,032 | 68,165,068 | [Condensed Consolidated Statements of Stockholders' Equity (Deficit)](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity%20(Deficit)) Changes in Stockholders' Equity (Deficit) (in thousands, except share amounts) | Item | Balance at Dec 31, 2024 | Balance at June 30, 2025 | | :------------------------------------------ | :---------------------- | :--------------------- | | Common Stock Shares | 124,776,152 | 125,645,952 | | Common Stock Amount | $13 | $13 | | Additional Paid-In Capital | $553,623 | $557,422 | | Accumulated Other Comprehensive Income | $22 | $14 | | Accumulated Deficit | $(456,994) | $(2,063,149) | | Total Stockholders' Equity (Deficit) | $96,664 | $(1,505,700) | **Key Changes for Six Months Ended June 30, 2025:** * Net loss: **$(1,606,155) thousand** (includes **$(32,486) thousand** for Q1 2025 and **$(1,573,669) thousand** for Q2 2025) * 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: **869,790 shares**, resulting in **$10 thousand** increase in Additional Paid-In Capital * Stock-based compensation expense: **$3,777 thousand** * Other comprehensive loss: **$(8) thousand** [Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Condensed Consolidated Statements of Cash Flows (in thousands) | Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------ | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(66,250) | $(52,312) | | Net cash provided by investing activities | $416 | $96,963 | | Net cash provided by financing activities | $174,459 | $65 | | Net increase in cash, cash equivalents and restricted cash equivalents | $108,625 | $44,716 | | Cash, cash equivalents and restricted cash equivalents, beginning of period | $84,362 | $33,773 | | Cash, cash equivalents and restricted cash equivalents, end of period | $192,987 | $78,489 | [Notes to Condensed Consolidated Financial Statements (Unaudited)](index=13&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements%20(Unaudited)) [1. Nature of the Business](index=13&type=section&id=1.%20Nature%20of%20the%20Business) - Vor Biopharma Inc. is a clinical-stage biotechnology company focused on advancing telitacicept, a novel dual-target recombinant fusion protein that inhibits BLyS and APRIL for autoimmune diseases. The company was incorporated on December 30, 2015, and is headquartered in Massachusetts[29](index=29&type=chunk) - The Company has incurred significant operating losses and anticipates continued losses, making its operations dependent on raising additional funding. As of June 30, 2025, the Company had **$200.6 million** in cash, cash equivalents, and marketable securities, and an accumulated deficit of **$2,063.1 million**[31](index=31&type=chunk)[32](index=32&type=chunk) - The issuance of **$174.4 million** in net cash proceeds from the 2025 PIPE Warrants in June 2025 has alleviated substantial doubt about the Company's ability to continue as a going concern for the next twelve months[32](index=32&type=chunk)[33](index=33&type=chunk) [2. Summary of Significant Accounting Policies](index=13&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) - The condensed consolidated financial statements are prepared in conformity with GAAP, requiring management to make estimates and assumptions, particularly for accrued expenses, stock-based compensation, fair value of financial instruments (including warrants), and R&D expenses[34](index=34&type=chunk)[35](index=35&type=chunk)[36](index=36&type=chunk) - Warrants are classified as either equity or liability based on specific terms and accounting guidance. Liability-classified warrants are revalued at each balance sheet date, with changes in fair value recorded in the statement of operations[40](index=40&type=chunk)[41](index=41&type=chunk) - Restructuring costs, including employee severance, are recognized based on whether benefits are ongoing or one-time, in accordance with ASC 712 or ASC 420[43](index=43&type=chunk) [3. Marketable Securities](index=14&type=section&id=3.%20Marketable%20Securities) Marketable Securities (in thousands) | Item | June 30, 2025 (Fair Value) | December 31, 2024 (Fair Value) | | :---------------- | :------------------------- | :----------------------------- | | U.S. Treasuries | $9,991 | $9,977 | | Maturing in one year or less | $9,991 | $4,993 | | Maturing after one year through five years | — | $4,984 | * No individual securities were in an unrealized loss position as of June 30, 2025, or December 31, 2024 * No impairments or credit loss reserves were recorded for marketable securities during the periods presented [4. Fair Value Measurements](index=15&type=section&id=4.%20Fair%20Value%20Measurements) Fair Value Measurements (in thousands) as of June 30, 2025 | Item | Level 1 | Level 2 | Level 3 | Total | | :------------------------ | :------ | :------ | :------ | :------ | | Cash equivalents (Money market funds) | $190,508 | — | — | $190,508 | | Marketable securities (U.S. Treasuries) | — | $9,991 | — | $9,991 | | Restricted cash equivalents (Money market funds) | $2,413 | — | — | $2,413 | | Warrant liabilities | — | $1,652,298 | — | $1,652,298 | * Fair value of cash equivalents and restricted cash equivalents is based on quoted market prices (Level 1) * Fair value of marketable securities and warrant liabilities is based on observable market inputs (Level 2) * No transfers between fair value levels occurred during the six months ended June 30, 2025 [5. Property and Equipment, Net](index=16&type=section&id=5.%20Property%20and%20Equipment,%20Net) Property and Equipment, Net (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------------- | :------------ | :---------------- | | Laboratory equipment | — | $9,625 | | Manufacturing equipment | — | $7,082 | | Computer equipment | — | $446 | | Furniture, fixtures and other | — | $606 | | Construction in progress | — | $36 | | Total | — | $17,795 | | Less: Accumulated depreciation | — | $(11,214) | | Property and equipment, net | $— | $6,581 | * Depreciation expense for the six months ended June 30, 2025, was **$2.9 million**, compared to **$1.8 million** for the same period in 2024 * In connection with the Restructuring Plan, the Company recognized a **$3.3 million** loss on disposal of long-lived assets and accelerated depreciation of approximately **$1.5 million** during the three and six months ended June 30, 2025[48](index=48&type=chunk)[49](index=49&type=chunk) [6. Accrued Liabilities](index=16&type=section&id=6.%20Accrued%20Liabilities) Accrued Liabilities (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :------------------------------------------ | :------------ | :---------------- | | Employee-related expenses (including Restructuring Liability) | $11,136 | $5,852 | | Professional fees | $1,615 | $1,461 | | Clinical expenses | $79 | $3,835 | | Manufacturing expenses | $50 | $516 | | Research and development expenses | $83 | $872 | | Accrued up-front license payment (Note 10) | $45,000 | — | | Other | $141 | $356 | | Total accrued liabilities | $58,104 | $12,892 | [7. Stockholders' Equity and Warrants](index=16&type=section&id=7.%20Stockholders'%20Equity%20and%20Warrants) - In December 2024, the Company completed a private placement, issuing **55,871,260 common shares** and warrants (2024 Warrants) to purchase up to **69,839,075 shares**, generating **$52.7 million** in net proceeds. These warrants are equity-classified[51](index=51&type=chunk)[52](index=52&type=chunk)[53](index=53&type=chunk) - In June 2025, the Company issued 2025 PIPE Warrants (**700,000,000 shares**) and RemeGen Warrants (**320,000,000 shares**) in a private placement, raising **$174.4 million** in net proceeds. These 2025 Warrants are liability-classified and had a fair value of **$1,652.3 million** as of June 30, 2025, resulting in a **$1,299.9 million** change in fair value recognized as an expense[54](index=54&type=chunk)[55](index=55&type=chunk)[56](index=56&type=chunk) - Neither the 2024 nor 2025 Warrants were included in diluted net loss per share for the period ended June 30, 2025, as their effect would be antidilutive[57](index=57&type=chunk) [8. Stock-Based Compensation](index=17&type=section&id=8.%20Stock-Based%20Compensation) - As of June 30, 2025, the Company had **69,668,889 shares** available under the 2023 Inducement Plan and **6,027,849 shares** under the Amended and Restated 2021 Equity Incentive Plan[58](index=58&type=chunk)[59](index=59&type=chunk) - During the six months ended June 30, 2025, the Company granted **87,549,888 stock options** (weighted-average fair value **$0.73/share**) and **1,224,000 restricted stock units** (weighted-average fair value **$1.33/share**). Total unrecognized compensation expense for stock options was **$66.3 million** (**2.1 years** weighted-average period) and for RSUs was **$2.9 million** (**2.3 years** weighted-average period)[60](index=60&type=chunk)[61](index=61&type=chunk)[65](index=65&type=chunk) - An Option Repricing in February 2025 reduced the exercise price of approximately **6.76 million options** to **$1.34/share**, but the associated incremental compensation cost of **$1.9 million** was reversed as conditions for modified terms were not met[63](index=63&type=chunk)[64](index=64&type=chunk) Stock-Based Compensation Expense (in thousands) | Allocation | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $599 | $1,272 | $1,641 | $2,677 | | General and administrative | $1,245 | $1,521 | $2,136 | $3,197 | | Total stock-based compensation expense | $1,844 | $2,793 | $3,777 | $5,874 | [9. Leases](index=20&type=section&id=9.%20Leases) - In June 2025, the Company entered into an early termination agreement for its Cambridgepark Lease, effective August 4, 2025, incurring an **$8.5 million** non-refundable termination fee. This resulted in accelerated amortization of the right-of-use asset and de-recognition of remaining balances[70](index=70&type=chunk) Lease Costs (in thousands) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :---------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Operating lease cost | $13,085 | $1,951 | $15,036 | $3,902 | | Variable lease cost | $774 | $679 | $1,485 | $1,209 | | Total lease cost | $13,859 | $2,630 | $16,521 | $5,111 | **Lease Liabilities (in thousands):** | Item | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Operating lease right-of-use assets | $24 | $35,007 | | Total lease liabilities | $— | $31,830 | * Weighted-average remaining lease term decreased from **5.7 years** (Dec 31, 2024) to **0.1 years** (June 30, 2025) [10. Significant Agreements](index=22&type=section&id=10.%20Significant%20Agreements) - On June 25, 2025, the Company entered into a license agreement with RemeGen Co., Ltd. for exclusive rights to develop and commercialize telitacicept outside Greater China. The Company is responsible for all development, regulatory, and commercialization activities in the licensed territory[72](index=72&type=chunk)[73](index=73&type=chunk) - Consideration for the license included an upfront cash payment of **$45.0 million** (accrued as of June 30, 2025) and the issuance of RemeGen Warrants initially valued at **$177.4 million**. The agreement was accounted for as an asset acquisition, resulting in a **$222.6 million** charge to R&D expense[74](index=74&type=chunk)[75](index=75&type=chunk) - RemeGen is eligible for up to **$330 million** in regulatory milestone payments, up to **$3.775 billion** in sales milestone payments, and tiered royalties on net sales. No milestone or royalty payments have been accrued as of June 30, 2025[76](index=76&type=chunk) [11. Net Loss Per Share](index=22&type=section&id=11.%20Net%20Loss%20Per%20Share) Net Loss Per Share Attributable to Common Stockholders (in thousands, except per share amounts) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Net loss attributable to common stockholders | $(1,573,669) | $(27,839) | $(1,606,155) | $(58,643) | | Weighted-average common shares outstanding | 125,271,447 | 68,299,170 | 125,049,032 | 68,165,068 | | Net loss per share, basic and diluted | $(12.56) | $(0.41) | $(12.84) | $(0.86) | * Potentially dilutive securities (stock options, RSUs, warrants) were excluded from diluted EPS calculation as their effect would be anti-dilutive[79](index=79&type=chunk)[80](index=80&type=chunk) [12. Restructuring Plan](index=24&type=section&id=12.%20Restructuring%20Plan) - On May 5, 2025, the Board approved a Restructuring Plan to wind down existing clinical and manufacturing operations and explore strategic alternatives, publicly announced on May 8, 2025[81](index=81&type=chunk) - The plan included a workforce reduction of **154 full-time employees** (approximately **99% of the employee base**), incurring **$14.1 million** in severance and benefit costs as of June 30, 2025[82](index=82&type=chunk) Total Restructuring Costs (in thousands) for Three and Six Months Ended June 30, 2025 | Item | Research and Development Expense | General and Administrative Expense | Total Restructuring Costs | | :------------------------------------------ | :------------------------------- | :------------------------------- | :------------------------ | | Severance and Benefits Costs | $8,957 | $5,188 | $14,145 | | Stock-based Compensation | — | $549 | $549 | | Accelerated Depreciation on Long-lived Assets | $1,230 | $284 | $1,514 | | Accelerated Amortization on Right-of-use Assets | $10,159 | $1,064 | $11,223 | | Loss on Disposal of Long-lived Assets | $3,303 | — | $3,303 | | **Total** | **$23,649** | **$7,085** | **$30,734** | * The restructuring resulted in a total of **$30.7 million** in costs for the three and six months ended June 30, 2025[84](index=84&type=chunk) - The remaining restructuring liability included in accrued liabilities as of June 30, 2025, was **$11.5 million**[85](index=85&type=chunk) [13. Segments](index=26&type=section&id=13.%20Segments) - The Company operates as a single reportable and operating segment, with the CEO serving as the Chief Operating Decision Maker (CODM). The CODM reviews consolidated operating results and net loss to make resource allocation decisions[86](index=86&type=chunk)[87](index=87&type=chunk) Segment Expenses (in thousands) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Trem-cel (external R&D) | $3,095 | $3,619 | $9,477 | $8,480 | | VCAR33 (external R&D) | $2,419 | $1,762 | $6,055 | $4,362 | | Other research and development | $225,144 | $3,177 | $228,139 | $6,294 | | Salaries and benefits | $17,863 | $10,404 | $29,476 | $22,517 | | General corporate activities | $16,694 | $4,947 | $21,863 | $10,246 | | Other segment items | $1,308,454 | $3,930 | $1,311,145 | $6,744 | | **Total Segment Expenses** | **$1,573,669** | **$27,839** | **$1,606,155** | **$58,643** | | **Segment Net Loss** | **$(1,573,669)** | **$(27,839)** | **$(1,606,155)** | **$(58,643)** | * Other segment items primarily include changes in fair value of warrant liabilities, stock-based compensation, depreciation, and non-cash lease expense[88](index=88&type=chunk)[89](index=89&type=chunk) [14. Related Party Transactions](index=26&type=section&id=14.%20Related%20Party%20Transactions) - In June 2025, RA Capital Healthcare Fund, L.P., an affiliate of a board member, purchased warrants to acquire up to **200,000,000 shares** of the Company's common stock for **$50.0 million** in gross proceeds as part of the 2025 Private Placement[91](index=91&type=chunk) [15. Subsequent Events](index=26&type=section&id=15.%20Subsequent%20Events) - On August 12, 2025, the Company entered into a new lease agreement for approximately **8,391 square feet** of office space in Boston, Massachusetts, with a term expiring **72 months** after the September 1, 2025 commencement date. Aggregate base rental payments are expected to be **$3.8 million** during the initial term[92](index=92&type=chunk)[93](index=93&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=28&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides a comprehensive discussion and analysis of the Company's financial condition and results of operations, highlighting key events such as the in-licensing of telitacicept, the restructuring plan, and the financial performance for the three and six months ended June 30, 2025, compared to the same periods in 2024 [Overview](index=28&type=section&id=Overview) - Vor Bio is a clinical-stage biopharmaceutical company focused on autoimmune diseases, having in-licensed telitacicept from RemeGen Co., Ltd. in June 2025[95](index=95&type=chunk) - Telitacicept is a novel fusion protein approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG), and is currently in a global Phase 3 clinical trial for gMG[95](index=95&type=chunk)[96](index=96&type=chunk) - The Company holds exclusive development and commercialization rights for telitacicept outside of Greater China[97](index=97&type=chunk) [Our Clinical Development Programs & Pipeline](index=28&type=section&id=Our%20Clinical%20Development%20Programs%20%26%20Pipeline) - The Company's broader development plan includes exploring telitacicept in additional autoantibody-driven diseases where BAFF/APRIL signaling is a validated target, leveraging prior clinical experience and mechanistic rationale[98](index=98&type=chunk) [Our Team](index=28&type=section&id=Our%20Team) - Jean-Paul Kress, M.D., was appointed CEO and Chairman in June 2025, bringing extensive experience in autoimmune disease drug development and corporate leadership[99](index=99&type=chunk) - Sandy Mahatme, J.D., LL.M., joined as Chief Financial Officer and Chief Business Officer in July 2025, with a background in biomanufacturing and biopharmaceutical finance[99](index=99&type=chunk)[100](index=100&type=chunk) - Qing Zuraw, M.D., M.P.H., M.B.A., joined as Chief Development Officer in July 2025, having led global clinical development programs for telitacicept at RemeGen[104](index=104&type=chunk) - Dallan Murray, M.B.A., joined as Chief Commercial Officer in August 2025, bringing over **20 years** of commercial leadership experience in the biopharmaceutical industry[104](index=104&type=chunk) [Our Strategy](index=30&type=section&id=Our%20Strategy) - The Company's strategic priorities include rapid global development of telitacicept, pipeline expansion into additional B cell-driven autoimmune diseases, and building global commercial capabilities for potential launches[104](index=104&type=chunk) [Our Product Candidate (Telitacicept)](index=30&type=section&id=Our%20Product%20Candidate%20(Telitacicept)) This section details telitacicept, its mechanism of action, current approvals in China, ongoing global clinical trials for gMG, regulatory designations, commercialization strategy, manufacturing approach, competitive landscape, and intellectual property protection [Telitacicept Overview and gMG](index=30&type=section&id=Telitacicept%20Overview%20and%20gMG) - Telitacicept is approved in China for SLE, RA, and gMG, and is a novel recombinant fusion protein that dual-targets BLyS (BAFF) and APRIL to inhibit B cell survival and plasma cell function[101](index=101&type=chunk)[108](index=108&type=chunk) - Generalized Myasthenia Gravis (gMG) is a rare, chronic autoimmune neuromuscular disorder with a prevalence of approximately **150-250 individuals per million** worldwide, and an estimated **100,000 individuals** in the United States[102](index=102&type=chunk)[103](index=103&type=chunk) - Current gMG treatments include acetylcholinesterase inhibitors, traditional immunosuppressives, FcRn antagonists, and complement inhibitors, but none directly modulate the cytokine environment sustained by BAFF and APRIL[105](index=105&type=chunk)[106](index=106&type=chunk)[107](index=107&type=chunk) [Phase 3 Clinical Trial in patients with gMG in China](index=32&type=section&id=Phase%203%20Clinical%20Trial%20in%20patients%20with%20gMG%20in%20China) - RemeGen's Phase 3 trial in China for gMG enrolled **114 patients**, demonstrating significant improvements in MG-ADL and QMG scores for the telitacicept group compared to placebo at week **24**[109](index=109&type=chunk)[111](index=111&type=chunk) Key Results from China Phase 3 gMG Trial (Week 24) | Endpoint | Telitacicept Group | Placebo Group | | :------------------------------------------ | :----------------- | :------------ | | MG-ADL score decrease from baseline | 5.74 points | 0.91 points | | QMG score decrease from baseline | 8.66 points | 2.27 points | | Patients with ≥3-point MG-ADL improvement | 98.1% | 12.0% | | Patients with ≥5-point QMG improvement | 87.0% | 16.0% | * Telitacicept was well-tolerated, with an overall adverse event rate comparable to placebo [Global Phase 3 Clinical Trial in patients with gMG](index=34&type=section&id=Global%20Phase%203%20Clinical%20Trial%20in%20patients%20with%20gMG) - Telitacicept is currently being evaluated in a global Phase 3 clinical trial for gMG, with the Company assuming responsibility from RemeGen. The trial is recruiting in the United States, Canada, Europe, Australia, and South America[112](index=112&type=chunk) - The primary endpoint is change from baseline in MG-ADL at week **24**, with key secondary endpoints including QMG score reduction and MG Quality of Life (MG-QoL15r) scale changes[112](index=112&type=chunk) - The first patient in the United States was enrolled in July 2024[113](index=113&type=chunk) [Regulatory Overview (Product Candidate)](index=34&type=section&id=Regulatory%20Overview%20(Product%20Candidate)) - Telitacicept has received Orphan Drug Designation (ODD) from both the U.S. FDA and European Medicines Agency (EMA) for the treatment of gMG[114](index=114&type=chunk) - In January 2024, the FDA cleared the Investigational New Drug (IND) application for a global multi-center Phase 3 clinical trial of telitacicept for adult patients with primary Sjögren's syndrome (pSS), and granted Fast-Track Designation (FTD) in March 2024[115](index=115&type=chunk) [Sales and Marketing](index=34&type=section&id=Sales%20and%20Marketing) - The Company plans to build focused commercial capabilities and infrastructure in regions like the United States, South America, Europe, Japan, the Middle East, and North Africa to commercialize telitacicept, if approved[116](index=116&type=chunk) [Manufacturing](index=34&type=section&id=Manufacturing) - The Company plans to rely on third-party contract manufacturers for clinical manufacturing of raw materials, devices, active pharmaceutical ingredients, and finished products, and expects to enter into long-term agreements for future clinical trials and commercialization[117](index=117&type=chunk) [Competition (Product Candidate)](index=34&type=section&id=Competition%20(Product%20Candidate)) - The autoimmune field is highly competitive, with numerous monoclonal antibodies, biologics, CAR T cells, and small molecules from companies like Alexion, Amgen, Argenx, Dianthus, Johnson & Johnson, and UCB already marketed or in development[118](index=118&type=chunk) [Intellectual Property (Product Candidate)](index=34&type=section&id=Intellectual%20Property%20(Product%20Candidate)) - The licensed patent portfolio from RemeGen includes a first patent family for telitacicept's composition of matter, with granted patents in key regions (US, China, Europe, Japan, Korea, Russia, Brazil, India) expected to expire in **2028**[119](index=119&type=chunk) - Additional licensed families cover telitacicept formulations (expiring **2040-2044**) and methods/uses for specific conditions like SLE, IgA nephropathy, Sjögren's syndrome, myasthenia gravis, and other autoimmune diseases (expiring **2039-2045**)[120](index=120&type=chunk)[121](index=121&type=chunk)[123](index=123&type=chunk)[124](index=124&type=chunk)[125](index=125&type=chunk)[126](index=126&type=chunk) [Government Regulation](index=36&type=section&id=Government%20Regulation) This section outlines the extensive regulatory framework governing drug and biological product development, approval, and commercialization in the United States and the European Union, including preclinical and clinical trial requirements, review processes, special designations, and post-approval obligations [Regulatory Approval in the United States](index=36&type=section&id=Regulatory%20Approval%20in%20the%20United%20States) - Pharmaceutical and biological products in the U.S. are extensively regulated by the FDA under the FDCA and PHSA, requiring considerable data for quality, safety, and efficacy before marketing[127](index=127&type=chunk)[128](index=128&type=chunk) - The approval process for biologics involves extensive preclinical studies, submission of an Investigational New Drug (IND) application, well-controlled human clinical trials, and submission of a Biologics License Application (BLA) for marketing approval[129](index=129&type=chunk) [Preclinical Studies (US)](index=38&type=section&id=Preclinical%20Studies%20(US)) - Preclinical studies involve laboratory and animal testing to assess product chemistry, formulation, potential adverse events, and therapeutic rationale, conducted in accordance with GLP regulations[131](index=131&type=chunk) - An IND must be submitted to the FDA and become effective before human clinical trials can begin, allowing interstate shipment of unapproved product candidates for investigational use[132](index=132&type=chunk) [Clinical Trials (US)](index=38&type=section&id=Clinical%20Trials%20(US)) - Clinical trials involve administering investigational products to volunteers or patients under qualified investigators, adhering to federal regulations, GCPs, and IRB-approved protocols[133](index=133&type=chunk)[134](index=134&type=chunk) - Trials are generally conducted in **three sequential phases** (Phase 1, 2, 3) to assess safety, pharmacokinetics, efficacy, and establish the overall benefit/risk relationship for product labeling[139](index=139&type=chunk)[145](index=145&type=chunk) - Regulatory agencies require extensive monitoring, auditing, and reporting of clinical activities, data, and adverse events, with trials potentially suspended or terminated for non-compliance or health risks[142](index=142&type=chunk)[143](index=143&type=chunk) [FDA Review Processes](index=41&type=section&id=FDA%20Review%20Processes) - After clinical trials, a BLA is submitted to the FDA, including preclinical and clinical results, proposed labeling, and manufacturing information, requiring a substantial user fee[146](index=146&type=chunk)[147](index=147&type=chunk) - The FDA reviews the BLA for safety, purity, potency, and cGMP compliance, with standard review goals of **10 months** (**6 months** for priority review), which can be extended[148](index=148&type=chunk)[149](index=149&type=chunk)[150](index=150&type=chunk) - Approval may be limited to specific indications or conditions, and the FDA may issue a Complete Response Letter for deficiencies or require a REMS program to manage risks[152](index=152&type=chunk)[153](index=153&type=chunk) [Orphan Drug Designation (US)](index=41&type=section&id=Orphan%20Drug%20Designation%20(US)) - Orphan drug designation is granted for products treating rare diseases (fewer than **200,000 U.S. individuals**) or conditions where development costs are unlikely to be recovered from sales[155](index=155&type=chunk) - Designation, requested before BLA submission, does not shorten the review process but grants **seven years** of market exclusivity upon first FDA approval for the designated indication, with limited exceptions[156](index=156&type=chunk)[157](index=157&type=chunk) [Expedited Development and Review Programs (US)](index=43&type=section&id=Expedited%20Development%20and%20Review%20Programs%20(US)) - The FDA offers expedited review programs for products addressing unmet medical needs in serious or life-threatening conditions, including Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval[158](index=158&type=chunk)[159](index=159&type=chunk)[160](index=160&type=chunk)[161](index=161&type=chunk)[162](index=162&type=chunk) - Fast Track allows for greater FDA interaction and rolling review; Breakthrough Therapy provides intensive guidance; Priority Review aims for a **six-month** review; and Accelerated Approval uses surrogate endpoints for earlier approval, contingent on confirmatory studies[159](index=159&type=chunk)[160](index=160&type=chunk)[161](index=161&type=chunk)[162](index=162&type=chunk) - These programs may expedite development but do not change approval standards, and qualification can be revoked or review times may not be shortened[164](index=164&type=chunk) [Additional Controls for Biologics (US)](index=45&type=section&id=Additional%20Controls%20for%20Biologics%20(US)) - The PHSA emphasizes manufacturing controls for biologics to reduce the risk of adventitious agents and grants the FDA authority to suspend licenses for public health dangers[165](index=165&type=chunk) - Approved biologics may be subject to official lot release, requiring manufacturers to submit samples and test results to the FDA, which may also perform confirmatory tests[166](index=166&type=chunk) [Post-Approval Requirements (US)](index=45&type=section&id=Post-Approval%20Requirements%20(US)) - After BLA approval, products are subject to ongoing FDA regulation, including marketing and promotion standards, adverse event reporting, and cGMP compliance[167](index=167&type=chunk)[168](index=168&type=chunk) - The FDA may require post-marketing testing (Phase 4), REMS, or surveillance, and can withdraw approvals or request recalls for non-compliance or newly discovered problems, leading to various sanctions[168](index=168&type=chunk)[169](index=169&type=chunk)[173](index=173&type=chunk) [U.S. Marketing Exclusivity](index=47&type=section&id=U.S.%20Marketing%20Exclusivity) - The BPCIA established an abbreviated approval pathway for biosimilar or interchangeable biological products[170](index=170&type=chunk) - Reference biological products receive **12 years** of data exclusivity from first licensure, preventing FDA from accepting biosimilar/interchangeable applications for **four years**, and market entry for **10 years**[171](index=171&type=chunk) [Regulatory Approval in the European Union](index=47&type=section&id=Regulatory%20Approval%20in%20the%20European%20Union) - The EMA coordinates the evaluation and monitoring of centrally authorized medicinal products in the EU, responsible for scientific evaluation of marketing authorization applications[172](index=172&type=chunk) - The EU approval process involves preclinical tests, submission of a Clinical Trial Application (CTA), well-controlled clinical trials, and submission of a Marketing Authorization Application (MAA)[174](index=174&type=chunk)[183](index=183&type=chunk) [Preclinical Studies (EU)](index=49&type=section&id=Preclinical%20Studies%20(EU)) - Preclinical tests in the EU involve laboratory evaluations of product chemistry, formulation, stability, and animal toxicity studies, complying with international, EU, and national regulations[176](index=176&type=chunk) [Clinical Trials (EU)](index=49&type=section&id=Clinical%20Trials%20(EU)) - Clinical trials in the EU are governed by the Clinical Trials Regulation (CTR), which harmonizes authorizations, simplifies adverse-event reporting, and increases transparency through a single-entry EU portal (CTIS)[177](index=177&type=chunk)[178](index=178&type=chunk) - The CTR introduced a streamlined application procedure and a harmonized assessment process, with a **three-year** transition period ending January 31, 2025, after which all trials are subject to its provisions[178](index=178&type=chunk)[179](index=179&type=chunk) [Review and Approval (EU)](index=49&type=section&id=Review%20and%20Approval%20(EU)) - Medicinal products in the EU require a Marketing Authorization (MA), obtainable through a centralized procedure administered by the EMA or national procedures[180](index=180&type=chunk) - The centralized procedure, compulsory for specific products like biotechnological or orphan medicinal products, grants a single MA valid across the European Economic Area (EEA) based on the CHMP's opinion and European Commission's decision[181](index=181&type=chunk)[182](index=182&type=chunk)[184](index=184&type=chunk) [Validity of Marketing Authorizations (EU)](index=51&type=section&id=Validity%20of%20Marketing%20Authorizations%20(EU)) - An MA typically has an initial validity of **five years**, renewable based on a re-evaluation of the risk-benefit balance. After definitive renewal, it can be valid for an unlimited period[186](index=186&type=chunk) - A 'sunset clause' dictates that an authorization not followed by actual placing of the product on the EU market within **three years** will cease to be valid[186](index=186&type=chunk) [Manufacturing Regulation in the EU](index=51&type=section&id=Manufacturing%20Regulation%20in%20the%20EU) - Manufacturing and importing medicinal products in the EU require specific authorizations and compliance with EU cGMP standards and distribution regulations[187](index=187&type=chunk) - Non-compliance can lead to civil, criminal, or administrative sanctions, including suspension of manufacturing authorization[187](index=187&type=chunk) [Data and Market Exclusivity (EU)](index=51&type=section&id=Data%20and%20Market%20Exclusivity%20(EU)) - Innovative medicinal products in the EU generally receive **eight years** of data exclusivity and **10 years** of market exclusivity, preventing generic/biosimilar referencing and commercialization[188](index=188&type=chunk) - A special regime exists for biosimilars, requiring appropriate preclinical or clinical trial results to support marketing authorization applications[189](index=189&type=chunk) [Orphan Drug Designation (EU)](index=51&type=section&id=Orphan%20Drug%20Designation%20(EU)) - Orphan medicinal product designation in the EU is for products treating life-threatening or chronically debilitating conditions affecting not more than **five in 10,000 persons**, or where development costs are not justified by sales[190](index=190&type=chunk)[191](index=191&type=chunk) - Designation provides incentives like fee reductions, protocol assistance, and **10 years** of market exclusivity, which can be reduced to **six years** under certain conditions or if a similar, clinically superior product is approved[193](index=193&type=chunk) [Post-Authorization Requirements (EU)](index=53&type=section&id=Post-Authorization%20Requirements%20(EU)) - MA holders in the EU must comply with regulatory requirements for manufacturing, marketing, promotion, and sale, including establishing a pharmacovigilance system and submitting periodic safety update reports (PSURs)[194](index=194&type=chunk) - New MAAs require a risk management plan (RMP), and advertising/promotion must comply with EU and Member State laws, adhering to the product's Summary of Product Characteristics (SmPC)[195](index=195&type=chunk)[196](index=196&type=chunk) [European Data Collection and Processing](index=53&type=section&id=European%20Data%20Collection%20and%20Processing) - Processing of health-related and personal data in Europe is governed by the EU GDPR, imposing strict requirements on disclosures, consents, data security, breach notifications, and individual rights[197](index=197&type=chunk) - Non-compliance with EU GDPR can lead to private litigation, processing prohibitions, and heavy fines, potentially increasing compliance costs and limiting activities in Europe[198](index=198&type=chunk) [Marketing (EU)](index=55&type=section&id=Marketing%20(EU)) - The EU prohibits benefits or advantages to healthcare professionals to induce product prescription, with interactions governed by national anti-bribery laws, sunshine rules, and industry codes[199](index=199&type=chunk) - Payments to physicians in certain EU Member States must be publicly disclosed, and non-compliance can result in reputational risk, administrative penalties, fines, or imprisonment[200](index=200&type=chunk) [International Regulation](index=55&type=section&id=International%20Regulation) - Foreign regulations govern clinical trials, commercial sales, and distribution of product candidates, with approval processes varying by country and potentially differing from FDA or European Commission timelines[201](index=201&type=chunk) [Other Healthcare Laws and Regulations and Legislative Reform](index=55&type=section&id=Other%20Healthcare%20Laws%20and%20Regulations%20and%20Legislative%20Reform) - The Company's operations are subject to various U.S. federal and state healthcare fraud and abuse laws, including the Anti-Kickback Statute, False Claims Act, HIPAA, and Physician Payments Sunshine Act, which broadly regulate interactions with healthcare providers and third-party payors[202](index=202&type=chunk)[205](index=205&type=chunk) - Violations of these laws can lead to significant civil, criminal, and administrative penalties, including fines, imprisonment, exclusion from government healthcare programs, and reputational harm[204](index=204&type=chunk)[206](index=206&type=chunk) - Recent legislative reforms, such as the Affordable Care Act (ACA) and the Inflation Reduction Act of 2022 (IRA), have significantly impacted healthcare financing and drug pricing, including Medicare drug price negotiation and rebates for price increases[208](index=208&type=chunk)[209](index=209&type=chunk)[211](index=211&type=chunk) - The U.S. Supreme Court's decision in Loper Bright Enterprises v. Raimondo, overturning the Chevron doctrine, could lead to increased regulatory uncertainty and legal challenges to federal agency regulations, including those from the FDA[213](index=213&type=chunk) [Pharmaceutical Coverage, Pricing and Reimbursement](index=61&type=section&id=Pharmaceutical%20Coverage,%20Pricing%20and%20Reimbursement) - Sales of product candidates depend on coverage and adequate reimbursement by governmental and private third-party payors, which is a time-consuming and costly process requiring scientific, clinical, and cost-effectiveness data[215](index=215&type=chunk)[216](index=216&type=chunk) - Coverage and reimbursement decisions in the U.S. are often influenced by CMS, while in Europe, pricing and reimbursement schemes vary widely by country, potentially involving pricing negotiations and Health Technology Assessments (HTA)[219](index=219&type=chunk)[220](index=220&type=chunk) - Downward pressure on healthcare costs globally, including increased discounts and reference pricing, creates higher barriers to entry for new products and could negatively impact the Company's revenues and profitability[221](index=221&type=chunk)[222](index=222&type=chunk) [Restructuring Plan (MD&A)](index=65&type=section&id=Restructuring%20Plan%20(MD%26A)) - On May 5, 2025, the Company approved a Restructuring Plan to wind down existing clinical and manufacturing operations and reduce its workforce by approximately **99%** (**154 full-time employees**)[223](index=223&type=chunk) - Restructuring costs incurred during the three and six months ended June 30, 2025, totaled **$30.7 million**, including severance, stock-based compensation modifications, and asset disposals/accelerated depreciation[224](index=224&type=chunk) - The Company incurred net losses of **$1,573.7 million** and **$1,606.2 million** for the three and six months ended June 30, 2025, respectively, with an accumulated deficit of **$2,063.1 million** as of June 30, 2025[225](index=225&type=chunk) - Existing cash, cash equivalents, and marketable securities of **$200.6 million** as of June 30, 2025, are expected to fund operating expenses and capital expenditure requirements into the **first quarter of 2027**[226](index=226&type=chunk) [Critical Accounting Estimates](index=65&type=section&id=Critical%20Accounting%20Estimates) - The preparation of financial statements requires management to make judgments and estimates affecting reported amounts, particularly for accrued R&D expenses, stock-based compensation, and fair value of financial instruments[227](index=227&type=chunk) - In connection with the restructuring, certain estimates related to accrued research and development have been revised, but no other material changes to critical accounting estimates were reported from the 2024 Annual Report[227](index=227&type=chunk) [Financial Operations Overview](index=65&type=section&id=Financial%20Operations%20Overview) - The Company has not generated any revenue since inception and does not expect to generate product sales revenue in the near future, if at all, relying on potential future product sales or collaboration/license agreements[228](index=228&type=chunk) - Research and development expenses, expensed as incurred, consist of external costs (CROs, consultants, supplies, manufacturing, licensing payments) and internal costs (personnel, facilities, depreciation). These expenses are expected to increase significantly as product candidates advance[229](index=229&type=chunk)[230](index=230&type=chunk)[231](index=231&type=chunk)[233](index=233&type=chunk) - General and administrative expenses primarily include personnel costs, professional services (legal, audit), facility-related expenses, and are expected to increase with business expansion[237](index=237&type=chunk)[238](index=238&type=chunk) - Other income (expense) includes interest income on cash and marketable securities, and changes in the fair value of liability-classified warrant liabilities[239](index=239&type=chunk)[240](index=240&type=chunk) [Results of Operations](index=69&type=section&id=Results%20of%20Operations) Summary of Results of Operations (in thousands) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change (2025 vs 2024) | | :------------------------------------------ | :------------------------------- | :------------------------------- | :-------------------- | | Research and development | $261,499 | $21,823 | $239,676 | | General and administrative | $12,785 | $7,212 | $5,573 | | Total operating expenses | $274,284 | $29,035 | $245,249 | | Loss from operations | $(274,284) | $(29,035) | $(245,249) | | Interest income | $537 | $1,196 | $(659) | | Change in fair value of warrant liabilities | $(1,299,922) | — | $(1,299,922) | | Net loss | $(1,573,669) | $(27,839) | $(1,545,830) | | Item | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (2025 vs 2024) | | :------------------------------------------ | :----------------------------- | :----------------------------- | :-------------------- | | Research and development | $288,200 | $46,145 | $242,055 | | General and administrative | $19,375 | $15,216 | $4,159 | | Total operating expenses | $307,575 | $61,361 | $246,214 | | Loss from operations | $(307,575) | $(61,361) | $(246,214) | | Interest income | $1,342 | $2,718 | $(1,376) | | Change in fair value of warrant liabilities | $(1,299,922) | — | $(1,299,922) | | Net loss | $(1,606,155) | $(58,643) | $(1,547,512) | - Research and development expenses increased by **$239.7 million** for the three months and **$242.1 million** for the six months ended June 30, 2025, primarily due to the **$222.6 million** upfront consideration for the Telitacicept License Agreement, **$3.3 million** loss on asset disposal, and **$11.2 million** in accelerated depreciation/amortization[242](index=242&type=chunk)[243](index=243&type=chunk) - General and administrative expenses increased by **$5.6 million** for the three months and **$4.2 million** for the six months ended June 30, 2025, mainly due to increased personnel costs from termination payments and higher facilities/other expenses from accelerated depreciation and asset disposals[244](index=244&type=chunk)[245](index=245&type=chunk) - Other income (expense) decreased by **$1,300.6 million** for the three months and **$1,301.3 million** for the six months ended June 30, 2025, primarily due to the change in fair value of liability-classified warrant liabilities[246](index=246&type=chunk) [Liquidity and Capital Resources](index=72&type=section&id=Liquidity%20and%20Capital%20Resources) - Since inception, the Company has funded operations primarily through equity sales, raising approximately **$691.5 million** in net proceeds as of June 30, 2025[247](index=247&type=chunk) - As of June 30, 2025, the Company had **$200.6 million** in cash, cash equivalents, and marketable securities, expected to fund operations into the **first quarter of 2027**[250](index=250&type=chunk)[251](index=251&type=chunk) - Future expenses are expected to increase substantially with continued R&D, clinical trials, manufacturing, regulatory approvals, and commercialization efforts, necessitating additional funding through equity, debt, or collaborations[252](index=252&type=chunk)[254](index=254&type=chunk) Cash Flows (in thousands) | Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------ | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(66,250) | $(52,312) | | Net cash provided by investing activities | $416 | $96,963 | | Net cash provided by financing activities | $174,459 | $65 | | Net increase in cash, cash equivalents and restricted cash equivalents | $108,625 | $44,716 | * Net cash used in operating activities increased by **$14.0 million** in 2025, driven by higher net loss partially offset by non-cash charges (warrant liabilities, in-process R&D acquisition)[256](index=256&type=chunk)[257](index=257&type=chunk)[259](index=259&type=chunk) - Financing activities provided **$174.5 million** in 2025, primarily from **$175.0 million** in proceeds from pre-funded warrants, compared to **$0.1 million** in 2024[261](index=261&type=chunk) - Contractual obligations include ongoing payments under license and collaboration agreements, such as the Telitacicept License Agreement, which require payments upon achievement of milestones and royalties on sales[263](index=263&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=76&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, the registrant is not required to provide quantitative and qualitative disclosures about market risk - The Company is a smaller reporting company and is not required to provide quantitative and qualitative disclosures about market risk[267](index=267&type=chunk) [Item 4. Controls and Procedures](index=76&type=section&id=Item%204.%20Controls%20and%20Procedures) This section details the management's evaluation of the effectiveness of the Company's disclosure controls and procedures and reports on any changes in internal control over financial reporting - Management, with CEO and CFO participation, concluded that the Company's disclosure controls and procedures were effective at the reasonable assurance level as of June 30, 2025[268](index=268&type=chunk)[269](index=269&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended June 30, 2025, that materially affected, or are reasonably likely to materially affect, the Company's internal control over financial reporting[270](index=270&type=chunk) PART II. OTHER INFORMATION This section includes information on legal proceedings, risk factors, other disclosures, exhibits, and required signatures [Item 1. Legal Proceedings](index=78&type=section&id=Item%201.%20Legal%20Proceedings) This section states that the Company is not currently involved in any material legal proceedings - The Company is not currently a party to any material legal proceedings[273](index=273&type=chunk) [Item 1A. Risk Factors](index=78&type=section&id=Item%201A.%20Risk%20Factors) This section outlines various risks that could materially affect the Company's business, financial condition, results of operations, cash flows, and access to liquidity, categorized by financial position, development, regulatory, third-party relationships, intellectual property, legal compliance, employee matters, and common stock ownership [Risks Related to Our Financial Position and Need for Additional Capital](index=78&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) - The Company has incurred significant net losses since inception (**$1,606.2 million** for the six months ended June 30, 2025) and expects to continue incurring losses, potentially never achieving or maintaining profitability[275](index=275&type=chunk)[276](index=276&type=chunk) - Substantial additional funding will be needed to support ongoing operations and growth strategy, and inability to raise capital on acceptable terms could force delays or elimination of R&D programs or commercialization efforts[277](index=277&type=chunk)[278](index=278&type=chunk)[282](index=282&type=chunk) - Raising additional capital through equity or convertible debt securities, including warrants (e.g., 2024 and 2025 Warrants), will cause significant dilution to existing stockholders[283](index=283&type=chunk)[284](index=284&type=chunk) - The Company has a limited operating history and no history of commercializing products, making it difficult to evaluate future viability and success[286](index=286&type=chunk)[287](index=287&type=chunk) - The ability to utilize net operating loss carryforwards (federal **$243.9 million**, state **$234.7 million** as of Dec 31, 2024) may be limited by ownership changes under Section 382 of the Internal Revenue Code, potentially increasing future tax obligations[292](index=292&type=chunk) [Risks Related to Discovery, Development, Manufacturing and Commercialization](index=83&type=section&id=Risks%20Related%20to%20Discovery,%20Development,%20Manufacturing%20and%20Commercialization) - The Company is substantially dependent on the success of its lead product candidate, telitacicept, and failure to complete its development, obtain approval, or commercialize it in a timely manner would harm the business[293](index=293&type=chunk)[295](index=295&type=chunk) - Reliance on clinical trial data from RemeGen in China for telitacicept carries risks, as access to data may be limited, and there's no assurance the FDA or other regulatory authorities will accept or consider such data[296](index=296&type=chunk)[298](index=298&type=chunk) - Clinical trials may fail to demonstrate safety and efficacy, or serious adverse effects may be identified, leading to increased costs, abandonment of development, or limitations on use[300](index=300&type=chunk)[301](index=301&type=chunk)[303](index=303&type=chunk) - Interim, topline, or preliminary clinical trial data may change upon full analysis, and discrepancies could harm reputation and business prospects[302](index=302&type=chunk)[304](index=304&type=chunk)[306](index=306&type=chunk) - The Company's limited resources may be expended on less profitable or successful product candidates or indications, leading to missed opportunities[307](index=307&type=chunk) - Even if approved, product candidates may fail to achieve sufficient market acceptance by physicians, patients, and payors due to factors like efficacy, safety, pricing, or competition[308](index=308&type=chunk)[309](index=309&type=chunk) - Failure to establish internal sales and marketing capabilities or secure third-party agreements could hinder commercialization efforts[310](index=310&type=chunk)[312](index=312&type=chunk) - The Company faces significant competition from major pharmaceutical and biotechnology companies, with competitors potentially achieving regulatory approval faster or developing superior therapies[313](index=313&type=chunk)[317](index=317&type=chunk)[318](index=318&type=chunk)[320](index=320&type=chunk) - Negative developments in protein-based therapies, especially fusion proteins like telitacicept, could damage public perception and adversely affect the business[322](index=322&type=chunk)[323](index=323&type=chunk) - Product liability lawsuits could result in substantial liabilities, requiring commercialization limits or significant monetary awards, and insurance coverage may be inadequate[324](index=324&type=chunk)[325](index=325&type=chunk) - Manufacturing fusion proteins is complex and difficult; problems could lead to delays in development or commercialization, and reliance on third-party CMOs increases risks of insufficient quantities, quality, or regulatory non-compliance[326](index=326&type=chunk)[327](index=327&type=chunk)[328](index=328&type=chunk)[330](index=330&type=chunk) - Failure to comply with environmental, health, and safety laws by the Company or its contractors could result in fines, penalties, or costs, materially affecting business success[331](index=331&type=chunk)[332](index=332&type=chunk)[333](index=333&type=chunk)[334](index=334&type=chunk) - Success in preclinical studies or early clinical trials, including those by RemeGen, may not be indicative of results in future clinical trials, especially with small patient numbers, potentially leading to abandonment of product candidates[335](index=335&type=chunk) [Risks Related to Regulatory Review](index=97&type=section&id=Risks%20Related%20to%20Regulatory%20Review) - Clinical trials may fail to demonstrate safety and efficacy to regulatory authorities' satisfaction, leading to additional costs, delays, or inability to complete development and commercialization[336](index=336&type=chunk)[337](index=337&type=chunk)[338](index=338&type=chunk) - Numerous unforeseen events during clinical trials, such as regulatory delays, enrollment difficulties, negative results, or third-party contractor failures, could delay or prevent marketing approval[339](index=339&type=chunk)[340](index=340&type=chunk) - Even with successful clinical trials, obtaining timely regulatory approval for telitacicept or other product candidates in the U.S. or other jurisdictions is uncertain, and approvals may be for narrower indications or subject to significant limitations[342](index=342&type=chunk)[343](index=343&type=chunk) - Marketing approval in one country does not guarantee approval in others, and seeking foreign regulatory approval involves additional costs, testing, and risks, potentially reducing the target market[344](index=344&type=chunk) - Significant delays or difficulties in patient enrollment or retention in clinical trials could increase development costs and delay or prevent necessary regulatory approvals[346](index=346&type=chunk)[347](index=347&type=chunk) [Risks Related to Our Relationships with Third Parties](index=103&type=section&id=Risks%20Related%20to%20Our%20Relationships%20with%20Third%20Parties) - Reliance on third parties (CROs, data management, investigators) for research, preclinical testing, and clinical trials reduces control over these activities and may lead to unsatisfactory performance, delays, or non-compliance with regulatory requirements[348](index=348&type=chunk)[349](index=349&type=chunk)[352](index=352&type=chunk)[353](index=353&type=chunk) - Dependence on third-party manufacturers for materials and product candidates increases the risk of insufficient quantities, quality, or acceptable cost, potentially delaying or impairing development and commercialization efforts[356](index=356&type=chunk)[357](index=357&type=chunk)[360](index=360&type=chunk)[361](index=361&type=chunk)[362](index=362&type=chunk)[363](index=363&type=chunk) - Collaborations with third parties for R&D and commercialization of product candidates may not be successful, as collaborators may not dedicate sufficient resources, delay trials, or abandon programs, impacting revenue generation[364](index=364&type=chunk)[365](index=365&type=chunk)[366](index=366&type=chunk) - Inability to establish collaborations on commercially reasonable terms could force alterations to development and commercialization plans, requiring increased expenditures or delays[368](index=368&type=chunk)[369](index=369&type=chunk)[371](index=371&type=chunk)[373](index=373&type=chunk) [Risks Related to Our Intellectual Property](index=111&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) - High dependence on intellectual property licensed from third parties, particularly the Telitacicept License Agreement with RemeGen, means termination or invalidation of these licenses could result in loss of significant rights and harm commercialization ability[374](index=374&type=chunk)[375](index=375&type=chunk)[376](index=376&type=chunk)[381](index=381&type=chunk) - Commercial success relies on obtaining, maintaining, and protecting intellectual property through patents, trademarks, and trade secrets; inadequate protection could allow competitors to erode competitive advantage[382](index=382&type=chunk)[383](index=383&type=chunk) - Patent protection may be insufficient or challenged, as patents may not provide meaningful protection, be circumvented, or be invalidated due to procedural defects, prior art, or legal challenges[387](index=387&type=chunk)[388](index=388&type=chunk)[392](index=392&type=chunk)[394](index=394&type=chunk) - Failure to protect the confidentiality of trade secrets could harm business and competitive position, as unauthorized disclosure or independent discovery by competitors could occur despite protective measures[396](index=396&type=chunk)[397](index=397&type=chunk)[398](index=398&type=chunk) - Inability to acquire or in-license necessary rights to key technologies from third parties on acceptable terms could force abandonment of relevant programs or product candidates[399](index=399&type=chunk)[401](index=401&type=chunk)[402](index=402&type=chunk) - Third-party claims of intellectual property infringement, misappropriation, or other violations could prevent or delay product development, leading to substantial damages, injunctions, or costly litigation[403](index=403&type=chunk)[405](index=405&type=chunk)[406](index=406&type=chunk)[407](index=407&type=chunk) - Compliance with procedural, document submission, and fee payment requirements for patents is critical; non-compliance can lead to abandonment or lapse of patent rights[408](index=408&type=chunk) - Intellectual property licensed from government-funded programs may be subject to federal regulations like 'march-in' rights and U.S.-based manufacturing preferences, limiting exclusive rights and manufacturing options[409](index=409&type=chunk)[410](index=410&type=chunk)[411](index=411&type=chunk) - Lawsuits to protect or enforce patents or other intellectual property can be expensive, time-consuming, and unsuccessful, potentially resulting in invalidation of patents or limited enforcement ability[412](index=412&type=chunk)[413](index=413&type=chunk) - Changes to patent law in the U.S. and other jurisdictions, including recent Supreme Court rulings, could diminish the value of patents and weaken the ability to protect product candidates[414](index=414&type=chunk) - Inability to protect intellectual property rights globally, due to varying laws and enforcement challenges, could allow competitors to use technologies in other jurisdictions and export infringing products[415](index=415&type=chunk)[416](index=416&type=chunk)[417](index=417&type=chunk)[418](index=418&type=chunk) - Patent terms may be inadequate to protect competitive position for a sufficient time, as patents might expire before or shortly after commercialization, leading to competition from generics[419](index=419&type=chunk) - Failure to obtain patent term extension and data exclusivity for product candidates could shorten the period of exclusive rights, allowing competitors to market sooner and reducing revenue[420](index=420&type=chunk) - Claims that employees or consultants have wrongfully used or disclosed confidential information or misappropriated trade secrets could lead to litigation, loss of intellectual property, or personnel[421](index=4

Company Overview - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases through innovative therapies [3] - The company is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and aims for commercialization to address serious autoantibody-driven conditions globally [3] Leadership Appointment - Dallan Murray has been appointed as Chief Commercial Officer, effective immediately, bringing over 25 years of experience in leading commercial strategy and product launches in the biotechnology and pharmaceutical sectors [2] - Murray previously served as Executive Vice President, Chief Customer Officer at Sarepta Therapeutics, where he led the commercial and medical affairs organizations, achieving approximately $1.8 billion in net product revenue in 2024 [2] Strategic Goals - The appointment of Murray is seen as crucial for Vor Bio as it prepares for the potential commercialization of telitacicept and shapes its broader growth strategy [2] - Murray expressed enthusiasm about the opportunity to bring much-needed treatments to patients worldwide and to help establish a strong commercial and strategic foundation for the company's long-term success [3]