Vor Biopharma Inc.
For the period ended Sep 30, 2025. Filed Nov 13, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 13, 2025
VOR$19.88+6.5%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-12.
0000950170-25-107276
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-13.
0001193125-25-280104
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 316 previous paragraphs and 134 current paragraphs.
86 unchanged; 27 reworded; 21 added; 201 removed; 2 previous and 0 current paragraphs rearranged.
Tables are not compared: 5 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (21)
Current report
Patients on telitacicept throughout the trial achieved a -7.5 mean MG-ADL change from baseline, while placebo crossover patients achieved -6.3; 96.2% of continuous patients and 90.2% of crossover patients reached ≥3-point improvement.
Current report
Patients on telitacicept throughout the trial achieved a -9.8 mean QMG change from baseline, while placebo crossover patients achieved -9.3; 94.2% of continuous patients and 90.2% of crossover patients reached ≥5-point improvement.
Current report
Telitacicept demonstrated a favorable safety profile comparable to placebo and consistent with prior clinical trials and post-marketing data across other autoimmune indications, including SLE, RA, SD and IgAN. No new safety signals were observed. Most adverse events were mild to moderate in severity.
Current report
No injection site reactions were reported during the open-label extension period in patients previously on telitacicept. Injection site reactions in placebo crossover patients were mild, self-resolving and did not lead to discontinuation.
Current report
systemic therapies for the treatment of Sjögren’s disease.
Current report
The data from the RemeGen-sponsored Phase 3 trial conducted in China evaluating telitacicept in SD were presented at the American College of Rheumatology (“ACR”) Convergence meeting in October 2025. The 48-week results demonstrated:
Current report
Mean change in ESSDAI score from baseline: At week 24, -4.4 (160mg), -3.0 (80mg), and -0.6 (placebo); at week 48, -4.6 (160mg), -3.2 (80mg), and -0.4 (placebo). These data demonstrate durable, dose-dependent improvement in systemic disease activity.
Current report
Mean change in ESSPRI score from baseline: At week 24, -1.88 (160mg), -1.31 (80mg), and -0.36 (placebo); at week 48, -2.56 (160mg), -1.74 (80mg), and -0.41 (placebo). These data showed sustained symptomatic benefit in dryness, fatigue, and pain.
Current report
≥3-point ESSDAI score improvement: At week 24, 71.8% (160mg), 47.1% (80mg), and 19.3% (placebo); at week 48, 73.0% (160 mg), 49.1% (80mg), and 16.5% (placebo).
Current report
Participants with ESSDAI score <5 (low disease activity): At week 24, 49.6% (160mg), 28.8% (80mg), and 10.9% (placebo); at week 48, 55.0% (160mg), 32.7% (80mg), and 12.2% (placebo).
Current report
Participants with ≥1-point or ≥15% ESSPRI score reduction: At week 24, 86.2% (160mg), 63.0% (80mg), and 32.2% (placebo); at week 48, 89.1% (160mg), 75.4% (80mg), and 33.3% (placebo).
Current report
Change from baseline in MFI-20 total (fatigue): At weeks 24 and 48, telitacicept 160mg and telitacicept 80mg each produced a statistically significant reduction in fatigue versus 80mg and placebo, with improvements sustained through the open-label extension period.
Current report
Telitacicept demonstrated a favorable safety profile comparable to placebo and consistent with prior studies across other autoimmune indications, including systemic lupus erythematosus, rheumatoid arthritis, myasthenia gravis, and IgA nephropathy. No new safety signals were observed. Most adverse events were mild to moderate in severity.
Current report
Other Income
Current report
Other income represents the proceeds received from the sale of certain intellectual property related to the Company’s previous product candidates trem-cel, VCAR33 and VADC45.
Current report
Research and development expenses were $14.1 million for the three months ended September 30, 2025, compared to $21.8 million for the three months ended September 30, 2024. The decrease of $7.7 million was primarily due to lower stock-based compensation and personnel costs as the Company had lower headcount following the implementation of the Restructuring Plan and reduced spend on its previous programs, trem-cel and VCAR33, partially offset by the $13.1 million increase in spend on telitacicept - gMG, as the Company began research and development activities for the new program.
Current report
General and administrative expenses were $14.0 million for the three months ended September 30, 2025, compared to $6.7 million for the three months ended September 30, 2024. The increase of $7.3 million was primarily due to an increase in stock-based compensation expense.
Current report
During October 2025, we sold an additional 1,370,111 shares of our common stock under the Stifel ATM Facility at a weighted-average price per share of $36.73 for aggregate net proceeds of $49.8 million, after deducting commissions. Following these sales, $48.9 million remained available to be sold under the Stifel ATM Facility.
Current report
Underwritten Public Offering
Current report
On November 12, 2025, we issued and sold 10,000,000 shares of common stock in an underwritten public offering at a price to the public of $10.00 per share, for net proceeds of approximately $93.7 million, after deducting the underwriting discounts and commissions and estimated offering expenses. In addition, on November 12, 2025, the underwriters exercised in full their option to purchase 1,500,000 additional shares of common stock at the public offering price, less the underwriting discounts and commissions. The closing of the purchase of the additional shares is expected to occur on November 14, 2025, subject to satisfaction of customary closing conditions, for additional expected net proceeds of $14.1 million, after deducting the underwriting discounts and commissions (collectively, the “November 2025 Offering”).
Current report
The $44.3 million increase in net cash used in operating activities for the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 was primarily due to the $45.0 million up-front license payment due under the Telitacicept License Agreement.
Removed paragraphs (201)
Previous report
Our Clinical Development Programs & Pipeline
Previous report
Our Team
Previous report
We are led by a seasoned management team with deep expertise in autoimmune disease drug development, commercialization, and corporate leadership.
Previous report
Jean-Paul Kress, M.D., Chief Executive Officer (“CEO”) and Chairman of the Board, was appointed in June 2025. Dr. Kress most recently served as Chief Executive Officer of MorphoSys AG from September 2019 until August 2024 when it was acquired by Novartis. Dr. Kress currently serves the board of directors of Sanofi and previously served as chairman of the board of Erytech Pharma from June 2019 until June 2023. He served as President and Chief Executive Officer of Syntimmune Inc. from January 2018 until November 2018. Prior to joining Syntimmune, Dr. Kress served as Executive Vice President of International and Head of Global Therapeutic Operations at Biogen Inc from June 2017 to January 2018. He previously served as a member of the board of directors of Sarepta Therapeutics, Inc. from September 2015 to June 2017. From September 2015 to June 2017, Dr. Kress served as Senior Vice President, Head of North America at Sanofi Genzyme. From July 2011 to September 2015, Dr. Kress served as President and Chief Executive Officer of Sanofi Pasteur MSD, one of the leading European vaccine companies. Prior to then, Dr. Kress worked at Gilead, Abbvie and Eli Lilly in senior commercial and business development roles in the United States and in Europe. Dr. Kress holds an M.D. degree from Faculté Necker-Enfants Malades in Paris, and graduate and post-graduate degrees in pharmacology and immunology from École Normale Supérieure in Paris.
Previous report
Sandy Mahatme, J.D., LL.M., Chief Financial Officer and Chief Business Officer, joined the Company in July 2025. Mr. Mahatme most recently served as President, Chief Operating Officer and Chief Financial Officer of National Resilience Inc., a technology focused biomanufacturing company dedicated to broadening access to complex medicines from July 2020 to June 2024. From November 2012 to July 2020, Mr. Mahatme served in various executive positions, including as Executive Vice President, Chief Financial Officer and Chief Business Officer of Sarepta Therapeutics, Inc., a publicly traded biopharmaceutical company. Prior to those roles, he worked at Celgene Corporation, where he served in various positions including Senior Vice President of Corporate Development, Senior Vice President of Finance, Corporate
Previous report
Treasurer and Head of Tax. Mr. Mahatme served in senior roles in business development and corporate finance at Pfizer after starting his career at Ernst & Young LLP. Mr. Mahatme has served as a director of Idorsia Pharmaceuticals Ltd. since May 2020 and of CRISPR Therapeutics AG since May 2024. In addition, Mr. Mahatme previously served as a director on the boards of Aeglea Bio Therapeutics, Inc. from June 2015 to July 2022 and Flexion Therapeutics, Inc. from July 2014 to June 2021. Mr. Mahatme earned Master of Laws degrees from Cornell Law School and the New York University School of Law and is a member of the New York State Bar Association.
Previous report
Qing Zuraw, M.D., M.P.H., M.B.A., Chief Development Officer, joined the Company in July 2025. Dr. Zuraw joins Vor Bio with over 25 years of experience leading complex global and U.S. clinical development programs across autoimmune, inflammatory, and immunologic diseases. Most recently, she served as Chief Development Officer and Head of Global Clinical Development for Autoimmune Diseases at RemeGen Co., Ltd. from December 2022 to July 2025, where she was one of the key leaders of the successful development and execution of clinical trials for telitacicept across three key indications—SLE, Sjögren’s syndrome, and myasthenia gravis (“MG”)—Dr. Zuraw has also previously held senior leadership roles at Janssen Research & Development from December 2018 to December 2022, Teva Pharmaceutical Industries Ltd. from August 2017 to December 2018, and Akebia Therapeutics, Inc. from June 2015 to July 2017, where she led global clinical development programs across rheumatology, nephrology, respiratory, and immunology. She played a key role in the U.S. FDA approval of Guselkumab for psoriatic arthritis and contributed to multiple NDA and BLA submissions across therapeutic areas. Dr. Zuraw earned her M.D. from Shandong University Cheeloo College of Medicine, M.P.H from Johns Hopkins University and M.B.A. from Drexel University.
Previous report
Dallan Murray, M.B.A., Chief Commercial Officer, joined the Company in August 2025. Mr. Murray brings over 20 years of commercial leadership experience in the biopharmaceutical industry. Most recently, he worked at Sarepta Therapeutics Inc. from October 2013 to August 2025, serving in various roles including Senior Vice President and Chief Commercial Officer. Mr. Murray has also previously held multiple positions at Vertex Pharmaceuticals Inc. from April 2010 to October 2013, including Senior Director of HCV Marketing. Other roles included Director, Hepatitis Marketing Team at Gilead Sciences Inc. from March 2008 to April 2010, Associate Director at Biogen Inc. from December 2004 to March 2008, and multiple product roles at Janssen-Ortho Inc. from February 1999 to December 2004. Mr. Murray earned his M.B.A. from Queen’s University.
Previous report
Our Strategy
Previous report
Vor Bio is executing a focused strategy to become a leading global company in autoimmune therapeutics. Our strategic priorities include:
Previous report
Rapid Global Development of Telitacicept: We are advancing telitacicept through global Phase 3 trials, with the near-term goal of regulatory approval in the United States, the European Union and Japan for gMG. Our strategy leverages clinical de-risking from prior trials of telitacicept conducted in China by RemeGen.
Previous report
Pipeline Expansion: Telitacicept’s dual inhibition of BAFF/APRIL provides a platform to address a broad range of B cell-driven autoimmune diseases. We are evaluating opportunities to develop telitacicept in additional indications based on scientific rationale and unmet market need.
Previous report
Global Commercial Preparedness: With telitacicept already approved in China for SLE, RA and gMG and a registrational program underway in major global markets, we are building commercial capabilities and infrastructure to support potential launches.
Previous report
Our Product Candidate
Previous report
Telitacicept
Previous report
Telitacicept is approved in China for the treatment of SLE, RA and gMG. A global Phase 3 clinical trial in gMG is currently underway across the United States, Europe, South America, and Asia including Japan to support potential approval in the United States, Europe, and Japan.
Previous report
gMG is a rare, chronic autoimmune neuromuscular disorder characterized by fluctuating skeletal muscle weakness resulting from pathogenic autoantibodies, most frequently the targeting components of the neuromuscular junction. These autoantibodies, about 85% of which are acetylcholine receptor (“AChR”) and 5-8% of which are muscle-specific tyrosine kinase (“MuSK”), impair synaptic transmission, leading to a disabling and potentially life-threatening condition that requires long-term immunomodulation to control disease activity.
Previous report
gMG has a prevalence of approximately 150 to 250 individuals per million worldwide, with a rising incidence among aging populations. In the United States, based on a 2021 claims analysis, the prevalence is thought to be comparable to Europe at approximately 370 individuals per million, and it is estimated that approximately 100,000 individuals have MG in the United States.
Previous report
The treatment goals for gMG include achieving minimal symptoms while minimizing treatment side effects. Even with newer treatments, MG poses significant physical, psychological, and economic impacts despite available treatments, with potential for exacerbations, myasthenia crises which could be life threatening, and complications such as infections or autoimmune comorbidities. Drug therapy for gMG typically begins with acetylcholinesterase inhibitors (such as pyridostigmine). However, as acetylcholinesterase inhibitors may improve symptoms, they are often insufficient. Traditional immunosuppressive therapies, such as corticosteroids, azathioprine, and mycophenolate mofetil, remained first-line treatments for decades, despite their delayed onset of action, cumulative toxicity, and inconsistent disease control. Recent biologics have introduced targeted immune modulation with improved clinical outcomes. Currently there are two classes of biologics in clinical development that have already obtained approval: neonatal fragment crystallizable receptor (“FcRn”) antagonists and complement inhibitors.
Previous report
FcRn antagonists enhance immunoglobulin G (“IgG”) catabolism and have demonstrated clinical benefit in AChR antibody positive patients. They are administered in cycles of four weeks or six weeks due to the deep reduction in IgG level, which increases the potential for severe infection. Complement inhibitors block terminal complement activation and have shown efficacy in reducing disease severity. However, complement inhibitors carry a black box warning on the label, which requires a Risk Evaluation and Mitigation Strategies (“REMS”) program requiring patients to be vaccinated to prevent serious brain infection prior to initiating the therapy. Most recently, a monoclonal antibody targeting CD19, depleting a broad range of B-cells, showed clinical benefit in Phase 3 clinical trials.
Previous report
However, none of these agents directly modulates the cytokine environment that sustains autoreactive B cells and plasma cells. B cell maturation and plasma cell survival are critically regulated by two cytokines of the TNF ligand superfamily: BAFF and APRIL. Elevated circulating levels of BAFF and APRIL have been detected in autoimmune diseases, including gMG. In gMG specifically, BAFF has been shown to be elevated in both serum and thymic tissues of patients with active disease and correlates with higher anti-AChR antibody titers.
Previous report
Telitacicept is a novel recombinant fusion protein. By binding to both BAFF and APRIL, telitacicept selectively inhibits B cell survival and plasma cell function. This dual-targeting approach has demonstrated clinical benefit in autoimmune conditions, including SLE, RA, IgA nephropathy, primary Sjögren’s syndrome (“pSS”), and gMG.
Previous report
Phase 3 Clinical Trial in patients with gMG in China
Previous report
Telitacicept was evaluated by RemeGen in a Phase 3 clinical trial in patients with gMG in China. The trial enrolled 114 patients and consisted of a double-blind treatment period (“Part A”) and an open-label treatment period (“Part B”). In Part A, patients were randomized 1:1 to receive either subcutaneous telitacicept 240 mg or placebo once a week for 24 doses. After completing Part A, patients were automatically entered into Part B, in which all patients receive weekly subcutaneous telitacicept 240 mg for 24 weeks.
Previous report
The primary endpoint of the trial was change from baseline in the Myasthenia Gravis Activities of Daily Living (“MG-ADL”) score at week 24 of Part A. MG-ADL is an 8-item patient-reported scale that measures MG symptoms and functional status. Each item ranges from 0-3 for a total score range of 0-24. The MG-ADL scale quantifies the impact of MG symptoms on daily life quality, focusing on patients’ subjective experience and daily function. A secondary endpoint was change from baseline in the Quantitative Myasthenia Gravis (“QMG”) score, which is a 13-item scale used to quantify disease severity in MG, with total QMG scores ranging from 0-39. The QMG score assesses the strength and endurance of the whole body muscle group, focusing on objective measurement. There is a strong correlation between MG-ADL and QMG in evaluating treatment response, and the combination of MG-ADL and QMG can comprehensively reflect the disease severity.
Previous report
The “MG-ADL score decreased by 5.74 points from baseline in the telitacicept group, compared to a decrease of 0.91 points in the placebo group (note: missing data imputed as non-response).
Previous report
The QMG score decreased by 8.66 points from baseline in the telitacicept group, compared to a decrease of 2.27 points in the placebo group.
Previous report
98.1% of patients in the telitacicept group demonstrated a ≥ 3-point improvement in MG-ADL score, compared to 12.0% in the placebo group.
Previous report
87.0% of patients in the telitacicept group demonstrated a ≥ 5-point improvement in QMG score, compared to 16.0% in the placebo group.
Previous report
The overall adverse event (“AE”) rate in the telitacicept group was comparable to that in the placebo group, indicating telitacicept was well-tolerated.
Previous report
Global Phase 3 Clinical Trial in patients with gMG:
Previous report
Telitacicept is currently being evaluated in a global Phase 3 clinical trial, for which we will assume responsibility from RemeGen in connection with the license agreement, for the treatment of gMG. The trial is currently recruiting in the United States, Canada, Europe, Australia and South America. The trial is a randomized, double-blind, placebo controlled trial with an open-label extension period. The primary endpoint is change from baseline in MG-ADL at week 24. Key secondary endpoints include change from baseline in QMG score, proportion of patients achieving MG-ADL score reduction of at least 2 points, QMG score reduction of at least 3 points at week 24 and change from baseline in MG Quality of Life (“MG-QOL15r”) scale at week 24. The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale is scored on a 0-2 point scale, with the total score ranging from 0 to 30. Higher scores indicate a more severe impact of the disease on aspects of the patient's life. A decrease from baseline score indicates improvement.
Previous report
Regulatory Overview
Previous report
Telitacicept has received Orphan Drug Designation (“ODD”) from both the U.S. Food and Drug Administration (“FDA”) and European Medicines Agency (“EMA”) for the treatment of gMG and is now the first in class, dual inhibitor of BAFF and APRIL biologic drug for gMG with ODD from both FDA and EMA.
Previous report
In January 2024, the FDA cleared the Investigational New Drug (“IND”) application for the global multi-center Phase 3 clinical trial of telitacicept for the treatment of adult patients with pSS. In March 2024, telitacicept received Fast-Track Designation (“FTD”) from the FDA for the treatment of adult patients with pSS.
Previous report
Sales and Marketing
Previous report
Given our stage of development, we have not yet established a commercial organization or distribution capabilities. We plan to build focused capabilities in various regions worldwide (the United States, South America, Europe, Japan, the Middle East and North Africa) to commercialize our product candidate, if approved. We plan to focus our efforts in these regions because we believe the patient populations and medical specialists for the indications we are targeting are sufficiently concentrated to allow us to effectively promote our product candidate, if approved for commercial sale, with a targeted sales team.
Previous report
Manufacturing
Previous report
We plan to rely on third-party contract manufacturers for clinical manufacturing of required raw materials, manufacturing devices, active pharmaceutical ingredients and finished product for our research and clinical manufacturing. We do not have long-term agreements with any of these third parties. We also do not have any current contractual relationship for the manufacture of material for clinical trials beyond Phase 3 or commercial supplies. We intend to enter into agreements with third-party contract manufacturers and one or more backup manufacturers for future production. We continue to analyze the feasibility of building additional manufacturing capabilities for future development and commercial quantities of any products that we develop. Such products will need to be manufactured in facilities, and by processes, that comply with the requirements of the FDA and the regulatory authorities of other jurisdictions in which we are seeking approval.
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Reworded paragraphs (27)
Previous wording
Vor Bio is a clinical-stage biopharmaceutical company focused on developing a novel therapy in the treatment of autoimmune diseases. In June 2025, we in-licensed telitacicept from RemeGen Co., Ltd. (“RemeGen”). Telitacicept is approved in China for the treatment of systemic lupus erythematosus (“SLE”), rheumatoid arthritis (“RA”), and generalized myasthenia gravis (“gMG”). A global Phase 3 clinical trial in gMG is currently underway across the United States, Europe, South America, and Asia including Japan to support potential approval in the United States, Europe, and Japan.
Current wording
Vor Bio is a clinical-stage biopharmaceutical company focused on developing a novel therapy in the treatment of autoimmune diseases. In June 2025, we in-licensed telitacicept from RemeGen Co., Ltd. (“RemeGen”). Telitacicept is approved in China for the treatment of generalized myasthenia gravis (“gMG”), systemic lupus erythematosus (“SLE”) and rheumatoid arthritis (“RA”). RemeGen has also filed Biologics License Applications (“BLAs”) in China for the treatment of Sjögren’s disease (“SD”) and IgA nephropathy (“IgAN”).
Previous wording
Pursuant to our license agreement with RemeGen, we were granted an exclusive license to develop and commercialize telitacicept outside of the Greater China region, which includes mainland China, Hong Kong, Macau and Taiwan. RemeGen retains development and commercialization rights in Greater China. The terms of the license agreement are more fully described in our Current Report on Form 8-K filed with the SEC on June 26, 2025. A copy of the license agreement is filed herewith as Exhibit 10.9.
Current wording
Pursuant to our license agreement with RemeGen, we were granted an exclusive license to develop and commercialize telitacicept outside of the Greater China region, which includes mainland China, Hong Kong, Macau and Taiwan. RemeGen retains development and commercialization rights in Greater China.
Previous wording
In July 2024, the clinical trial enrolled the first patient in the United States.
Current wording
Telitacicept is currently being evaluated in a global Phase 3 clinical trial, for which we are assuming responsibility from RemeGen in connection with the license agreement, for the treatment of gMG. The trial is currently recruiting patients in North America, Europe, Latin America, and Asia to support potential approval in the United States, Europe, Japan and other countries. In July 2024, the clinical trial enrolled a patient in the United States, the first in the global clinical trial.
Previous wording
The data from the results of the Phase 3 trial were presented at the 2025 American Academy of Neurology (“AAN”) meeting in April 2025 and in European Academy of Neurology in June 2025. The data at week 24 demonstrated:
Current wording
The data from the open-label treatment period (Part B) of the Phase 3 trial were presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (“AANEM”) meeting in October 2025. The data at week 48 demonstrated:
Previous wording
Our broader development plan includes exploring development of telitacicept in additional autoantibody-driven diseases where BAFF / APRIL signaling is a validated target, supported by prior clinical experience and mechanistic rationale.
Current wording
Our broader development plan includes exploring development of telitacicept in additional autoantibody-driven diseases where BAFF / APRIL signaling is a validated target, supported by prior clinical experience and mechanistic rationale. This includes Sjögren’s disease, one of the most common systemic autoimmune diseases globally. There are no approved disease-modifying
Previous wording
During the three and six months ended June 30, 2025, we incurred restructuring costs of $30.7 million which is comprised of severance payments, stock-based compensation modifications, loss on disposal of long-lived assets and accelerated depreciation and amortization on long-lived assets and right-of-use assets. We expect to incur an aggregate of $30.9 million of restructuring costs, with the additional $0.2 million of costs recognized in the third quarter of 2025 for certain costs which met the criteria for one-time termination benefits and require employees to provide future service.
Current wording
During the nine months ended September 30, 2025, we incurred restructuring costs of $29.7 million which is comprised of severance payments, stock-based compensation modifications, loss on disposal of long-lived assets and accelerated depreciation and amortization on long-lived assets and right-of-use assets.
Previous wording
We have incurred significant operating losses since inception, including net losses of $1,573.7 million and $1,606.2 million for the three and six months ended June 30, 2025, respectively and $116.9 million for the year ended December 31, 2024. As of June 30, 2025, we had an accumulated deficit of $2,063.1 million.
Current wording
We have incurred significant operating losses since inception, including net losses of $812.7 million and $2,418.8 million for the three and nine months ended September 30, 2025, respectively and $116.9 million for the year ended December 31, 2024. As of September 30, 2025, we had an accumulated deficit of $2,875.8 million.
Previous wording
As of June 30, 2025, we had cash, cash equivalents and marketable securities of $200.6 million. Based on our current operating plan, we expect that our cash, cash equivalents and marketable securities at June 30, 2025 will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2027.
Current wording
As of September 30, 2025, we had cash, cash equivalents and marketable securities of $170.5 million. Based on our current operating plan and considering the net proceeds of $49.8 million from the sale of shares of common stock under our at-the-market sales facility during October 2025 and the approximately $107.8 million in net proceeds and expected net proceeds from the November 2025 Offering (as defined below), we expect that our cash, cash equivalents and marketable securities will enable us to fund our operating expenses and capital expenditure requirements into the second quarter of 2027.
Previous wording
Our management’s discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with United States generally accepted accounting principles. The preparation of our condensed consolidated financial statements requires us to make judgments and estimates that affect the reported amounts of assets, liabilities, costs, and expenses, and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events, and various other factors that we believe to be reasonable under the circumstances. Actual results may differ from these estimates under different assumptions or conditions. On an ongoing basis, we evaluate our judgments and estimates in light of changes in circumstances, facts, and experience. The effects of material revisions in estimates, if any, will be reflected in the condensed consolidated financial statements prospectively from the date of change in estimates. As of June 30, 2025, in connection with our decision to wind down clinical and manufacturing operations related to previous product candidates, we have revised certain estimates related to accrued research and development. There have been no other material changes to our critical accounting estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2024 Annual Report.
Current wording
Our management’s discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with United States generally accepted accounting principles. The preparation of our condensed consolidated financial statements requires us to make judgments and estimates that affect the reported amounts of assets, liabilities, costs, and expenses, and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events, and various other factors that we believe to be reasonable under the circumstances. Actual results may differ from these estimates under different assumptions or conditions. On an ongoing basis, we evaluate our judgments and estimates in light of changes in circumstances, facts, and experience. The effects of material revisions in estimates, if any, will be reflected in the condensed consolidated financial statements prospectively from the date of change in estimates. During the nine months ended September 30, 2025, in connection with our decision to wind down clinical and manufacturing operations related to previous product candidates, we have revised certain estimates related to accrued research and development. There have been no other material changes to our critical accounting estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2024 Annual Report.
Previous wording
successful patient enrollment in, and the initiation of, clinical trials, as well as drop out or discontinuation rates or complications with donors;
Current wording
successful patient enrollment in, and the initiation of, clinical trials, as well as drop out or discontinuation rates;
Previous wording
Comparison of Three and Six Months Ended June 30, 2025 and 2024
Current wording
Comparison of Three and Nine Months Ended September 30, 2025 and 2024
Previous wording
Research and development expenses were $261.5 million for the three months ended June 30, 2025, compared to $21.8 million for the three months ended June 30, 2024. The increase of $239.7 million was primarily due to the upfront consideration transferred for the Telitacicept License Agreement of $222.6 million, $3.3 million in loss on disposal of long-lived assets, and $11.2 million in accelerated depreciation and amortization due to the abandonment of long-lived assets (including the right-of-use asset) and the early termination penalty for the lease.
Current wording
Research and development expenses were $302.3 million for the nine months ended September 30, 2025, compared to $68.0 million for the nine months ended September 30, 2024. The increase of $234.4 million was primarily due to the upfront consideration transferred for the license agreement with RemeGen for telitacicept (the “Telitacicept License Agreement”) of $222.6 million, $13.1 million in research and development activities on telitacicept-MG as the Company began research and development activities for the new program, $3.3 million in loss on disposal of long-lived assets, and $11.2 million in accelerated depreciation and amortization due to the abandonment of long-lived assets (including the right-of-use asset) and the early termination penalty for the lease, partially offset by lower stock-based compensation and personnel costs as the Company had lower headcount following the Restructuring Plan.
Previous wording
General and administrative expenses were $12.8 million for the three months ended June 30, 2025, compared to $7.2 million for the three months ended June 30, 2024. The increase of $5.6 million was primarily due to an increase in personnel costs from the termination payments owed to employees subject to the reduction in force, and an increase in facilities and other expenses from accelerated depreciation and loss on disposals of long-lived assets.
Current wording
General and administrative expenses were $33.3 million for the nine months ended September 30, 2025, compared to $21.9 million for the nine months ended September 30, 2024. The increase of $11.4 million was primarily due to an increase in stock-based compensation expense, an increase in personnel costs from the termination payments owed to employees subject to the Restructuring Plan, and an increase in facilities and other expenses from accelerated depreciation and loss on disposals of long-lived assets.
Previous wording
Other income (expense) decreased by $1,300.6 million during the three months ended June 30, 2025, compared to the three months ended June 30, 2024. Other income (expense) decreased by $1,301.3 million during the six months ended June 30, 2025, compared to the six months ended June 30, 2024. The decrease in other income (expense) in both periods was due to the issuance of liability-classified warrants and the subsequent change in fair value of the warrant liabilities resulting from changes in the quoted price of the Company’s common stock underlying the warrants.
Current wording
Other income (expense) decreased by $785.5 million during the three months ended September 30, 2025, compared to the three months ended September 30, 2024. Other income (expense) decreased by $2,086.8 million during the nine months ended September 30, 2025, compared to the nine months ended September 30, 2024. The decrease in other income (expense) in both periods was primarily due to the issuance of liability-classified warrants and the subsequent changes in fair value of the warrant liabilities resulting from changes in the quoted price of the Company’s common stock underlying the warrants, offset in part by the proceeds from the sale of intellectual property related to the Company’s previous programs.
Previous wording
Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and have received aggregate net proceeds from financing transactions of approximately $691.5 million as of June 30, 2025.
Current wording
Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and have received aggregate net proceeds from financing transactions of approximately $711.7 million as of September 30, 2025.
Previous wording
On March 20, 2025, we filed a universal shelf registration statement (the “Shelf Registration Statement”), to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of June 30, 2025, $350.0 million remains available under this Shelf Registration Statement, including $119.7 million reserved for at-the market offerings discussed below.
Current wording
On March 20, 2025, we filed a universal shelf registration statement (the “Shelf Registration Statement”), to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of September 30, 2025, $329.5 million remains available under this Shelf Registration Statement, including $99.2 million reserved for at-the-market offerings discussed below. Following the additional at-the-market sales during October 2025 and the November 2025 Offering, assuming the closing of the purchase of the 1,500,000 additional shares by the underwriters occurs, $158.9 million remains available under this Shelf Registration Statement, with $48.9 million reserved for at-the-market offerings.
Previous wording
In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million, subject to our filing a prospectus supplement to the base prospectus included in our Shelf Registration Statement covering the offer and sale of shares of our common stock pursuant to the Stifel ATM Facility. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We did not sell any shares of our common stock under the Stifel ATM Facility during the six months ended June 30, 2025. As of June 30, 2025, $119.7 million remained available to be sold under the Stifel ATM Facility.
Current wording
In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 540,750 shares of our common stock under the Stifel ATM Facility during the nine months ended September 30, 2025 at a weighted-average price per share of $37.89 for aggregate net proceeds of $20.3 million, after deducting commissions. As of September 30, 2025, $99.2 million remained available to be sold under the Stifel ATM Facility.
Previous wording
As of June 30, 2025, we had cash, cash equivalents and marketable securities of $200.6 million. We will need to raise additional capital in the future to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us. In the event that we are unable to obtain sufficient additional funding, there can be no assurance that we will be able to continue as a going concern.
Current wording
As of September 30, 2025, we had cash, cash equivalents and marketable securities of $170.5 million. We will need to raise additional capital in the future to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us. In the event that we are unable to obtain sufficient additional funding, there can be no assurance that we will be able to continue as a going concern.
Previous wording
Based on our current operating plan, we expect that our existing cash, cash equivalents and marketable securities at June 30, 2025 will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2027. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.
Current wording
Based on our current operating plan and considering the additional at-the-market sales during October 2025 and the net proceeds and expected net proceeds from the November 2025 Offering, we expect that our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses and capital expenditure requirements into the second quarter of 2027. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.
Previous wording
As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the public or private sale of our equity, government or private party grants, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions. To the extent that we raise additional capital through the sale of our equity or convertible debt securities, including through the use of the Stifel ATM Facility, the ownership interest of our shareholders will be or could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders. Debt financing and equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we are unable to obtain additional funding, we could be forced to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or any commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations. If we raise funds through strategic collaborations or other similar arrangements with third parties, we may have to relinquish valuable rights to our platform technology, future revenue streams, research programs or product candidates or may have to grant licenses on terms that may not be favorable to us and/or may reduce the value of our common stock. Our ability to raise additional funds may be adversely impacted by worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from geopolitical tensions and adverse macroeconomic conditions or otherwise. Because of the numerous risks and uncertainties associated with product development, we cannot predict the timing or amount of increased expenses, and there is no assurance that we will ever be profitable or generate positive cash flow from operating activities.
Current wording
As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the public or private sale of our equity, government or private party grants, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions. To the extent that we raise additional capital through the sale of our equity or convertible debt securities, including through the use of the Stifel ATM Facility, the ownership interest of our shareholders may be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders. Debt financing and equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we are unable to obtain additional funding, we could be forced to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or any commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations. If we raise funds through strategic collaborations or other similar arrangements with third parties, we may have to relinquish valuable rights to our platform technology, future revenue streams, research programs or product candidates or may have to grant licenses on terms that may not be favorable to us and/or may reduce the value of our common stock. Our ability to raise additional funds may be adversely impacted by worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from geopolitical tensions and adverse macroeconomic conditions or otherwise. Because of the numerous risks and uncertainties associated with product development, we cannot predict the timing or amount of increased expenses, and there is no assurance that we will ever be profitable or generate positive cash flow from operating activities.
Previous wording
Net cash used in operating activities was $66.3 million for the six months ended June 30, 2025, reflecting a net loss of $1,606.2 million, offset by changes in operating assets and liabilities of $2.0 million and non-cash charges of $1,537.9 million. The non-cash charges primarily consisted of the change in fair value of warrant liabilities of $1,299.9 million and the acquisition of in-process research and development of $222.6 million.
Current wording
Net cash used in operating activities was $119.9 million for the nine months ended September 30, 2025, reflecting a net loss of $2,418.8 million, offset by changes in operating assets and liabilities of $7.0 million and non-cash charges of $2,291.9 million. The non-cash charges primarily consisted of the change in fair value of warrant liabilities of $2,090.4 million and the issuance of pre-funded warrants in connection with the acquisition of in-process research and development of $177.4 million.
Previous wording
Net cash used in operating activities was $52.3 million for the six months ended June 30, 2024, reflecting a net loss of $58.6 million and net cash used of $2.7 million for operating assets and liabilities, which were offset by non-cash charges of $9.0 million. The non-cash charges primarily consisted of stock-based compensation expense of $5.9 million, non-cash lease expense of $2.5 million and depreciation expense of $1.8 million, offset by $1.1 million of non-cash interest earned on marketable securities.
Current wording
Net cash used in operating activities was $75.6 million for the nine months ended September 30, 2024, reflecting a net loss of $86.2 million and net cash used of $2.8 million for operating assets and liabilities, which were offset by non-cash charges of $13.4 million. The non-cash charges primarily consisted of stock-based compensation expense of $8.1 million, non-cash lease expense of $3.8 million and depreciation expense of $2.7 million, offset by $1.1 million of non-cash interest earned on marketable securities.
Previous wording
Net cash provided by investing activities was $0.4 million for the six months ended June 30, 2025, which consisted of proceeds from the sales of property and equipment of $0.8 million, offset by $0.4 million in purchases of property and equipment. Net cash provided by investing activities was $97.0 million for the six months ended June 30, 2024, which consisted of proceeds of $107.0 million from the maturity of marketable securities, offset by purchases of $9.9 million of marketable securities and $0.1 million of property and equipment.
Current wording
Net cash provided by investing activities was $0.2 million for the nine months ended September 30, 2025, which consisted of proceeds from the sales of property and equipment of $0.8 million, partially offset by $0.6 million in purchases of property and equipment. Net cash provided by investing activities was $96.9 million for the nine months ended September 30, 2024, which consisted of proceeds of $107.0 million from the maturity of marketable securities, partially offset by purchases of $9.9 million of marketable securities and $0.2 million of property and equipment.
Previous wording
Net cash provided by financing activities was $174.5 million for the six months ended June 30, 2025, which consisted of $175.0 million in proceeds from the issuance of pre-funded warrants, offset by $0.6 million of issuance costs related to the December 2024 private placement. Net cash provided by financing activities was $0.1 million for the six months ended June 30, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.2 million and proceeds from exercise of stock options and issuance of shares under the ESPP of $0.1 million, offset by $0.2 million of taxes paid related to net share settlement of equity awards.
Current wording
Net cash provided by financing activities was $196.0 million for the nine months ended September 30, 2025, which primarily consisted of $175.0 million in proceeds from the issuance of pre-funded warrants, $20.3 million in proceeds from the issuance of common stock under the Stifel ATM Facility, and $2.1 million in proceeds from the exercise of stock options and issuance of shares under the ESPP, partially offset by $1.2 million of issuance costs related to the December 2024 private placement and June 2025 private placement. Net cash provided by financing activities was $0.1 million for the nine months ended September 30, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.3 million and proceeds from exercise of stock options and issuance of shares under the ESPP of $0.1 million, offset by $0.3 million of taxes paid related to net share settlement of equity awards.
Previous wording
As noted in Note 9 to the unaudited condensed consolidated financial statements and related notes included elsewhere in this Quarterly Report on Form 10-Q, we amended our lease for corporate office and laboratory real estate space to terminate on August 4, 2025. On June 30, 2025, we abandoned such space and have no remaining contractual payments related to the leased space. As noted in Note 15 to the unaudited condensed consolidated financial statements and related notes included elsewhere in this Quarterly Report on Form 10-Q, on August 12, 2025, we entered into a non-cancellable lease relating to corporate office space, with a term expiring in August 2031.
Current wording
As noted in Note 9 to the unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q, we amended our lease for corporate office and laboratory real estate space to terminate on August 4, 2025. On June 30, 2025, we abandoned such space and have no remaining contractual payments related to the leased space. As further noted in Note 9 to the unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q, on August 12, 2025, we entered into a non-cancellable lease relating to corporate office space, with a term expiring in August 2031.
Previous wording
Other commitments include license and collaboration agreements we have entered into with certain parties including the Telitacicept License Agreement discussed in Note 10 to the unaudited condensed consolidated financial statements and related notes included elsewhere in this Quarterly Report on Form 10-Q. Such arrangements require ongoing payments, including payments upon the achievement of certain development, regulatory and commercial milestones, receipt of sublicense income, as well as royalties on commercial sales. Payments under these arrangements will generally be expensed as incurred.
Current wording
Other commitments include license and collaboration agreements we have entered into with certain parties including the Telitacicept License Agreement discussed in Note 10 to the unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q. Such arrangements require ongoing payments, including payments upon the achievement of certain development, regulatory and commercial milestones, receipt of sublicense income, as well as royalties on commercial sales. Payments under these arrangements will generally be expensed as incurred.
Previous wording
Other than the changes discussed above, during the six months ended June 30, 2025, there were no additional significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2024 Annual Report.
Current wording
Other than the changes discussed above, during the nine months ended September 30, 2025, there were no additional significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2024 Annual Report.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
Research and development expenses were $288.2 million for the six months ended June 30, 2025, compared to $46.1 million for the six months ended June 30, 2024. The increase of $242.1 million was primarily due to the upfront consideration transferred for the Telitacicept License Agreement of $222.6 million, $3.3 million in loss on disposal of long-lived assets, and $11.2 million in accelerated depreciation and amortization due to the abandonment of long-lived assets (including the right-of-use asset) and the early termination penalty for the lease.
Previous report — wording carried elsewhere
General and administrative expenses were $19.4 million for the six months ended June 30, 2025, compared to $15.2 million for the six months ended June 30, 2024. The increase of $4.2 million was primarily due to an increase in personnel costs from the termination payments owed to employees subject to the reduction in force, and an increase in facilities and other expenses from accelerated depreciation and loss on disposals of long-lived assets.