Vor Biopharma Inc.
For the period ended Jun 30, 2025. Filed Aug 12, 2025.
View on SEC.govAbout this filing
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.
Jun 30, 2025 · Filed Aug 12, 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 2025-03-31; filed 2025-05-14.
0000950170-25-071349
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent 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 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 74 previous paragraphs and 316 current paragraphs.
42 unchanged; 18 reworded; 250 added; 14 removed; 0 previous and 6 current paragraphs rearranged.
Tables are not compared: 3 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 (250)
Current report
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 report
Telitacicept is a novel fusion protein for treating autoimmune diseases. It is constructed with the extracellular domain of the human transmembrane activator and calcium modulator and cyclophilin ligand interactor (“TACI”) receptor and the fragment crystallizable (“Fc”) domain of human immunoglobulin G (“IgG”). Telitacicept targets and acts on two cell signaling molecules critical for B lymphocyte development: B cell lymphocyte stimulator (“BLyS”), also known as B cell activating factor (“BAFF”), and a proliferation inducing ligand (“APRIL”), which allows it to effectively reduce B cell mediated autoimmune responses that are implicated in several autoimmune diseases.
Current report
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 report
Our Clinical Development Programs & Pipeline
Current report
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 report
Our Team
Current report
We are led by a seasoned management team with deep expertise in autoimmune disease drug development, commercialization, and corporate leadership.
Current 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.
Current 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
Current 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.
Current 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.
Current 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.
Current report
Our Strategy
Current report
Vor Bio is executing a focused strategy to become a leading global company in autoimmune therapeutics. Our strategic priorities include:
Current 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.
Current 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.
Current 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.
Current report
Our Product Candidate
Current report
Telitacicept
Current 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.
Current 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.
Current 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.
Current 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.
Current 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.
Current 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.
Current 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.
Current report
Phase 3 Clinical Trial in patients with gMG in China
Current 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.
Current 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.
Current report
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 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).
Current 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.
Current 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.
Current 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.
Current report
The overall adverse event (“AE”) rate in the telitacicept group was comparable to that in the placebo group, indicating telitacicept was well-tolerated.
Current report
Global Phase 3 Clinical Trial in patients with gMG:
Current 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.
Current report
In July 2024, the clinical trial enrolled the first patient in the United States.
Current report
Regulatory Overview
Current 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.
Showing the first 40 of 250 passages. Open the source sections for the rest.
Removed paragraphs (14)
Previous report
Vor Bio is a clinical-stage company harnessing the power of cell and genome engineering to develop potentially transformative therapies in acute myeloid leukemia (“AML”), a devastating disease with few treatment options. AML is the most common type of acute leukemia in adults and one of the deadliest and most aggressive blood cancers, affecting approximately 20,000 newly diagnosed patients each year in the United States.
Previous report
On May 8, 2025, we announced that we are winding down our clinical and manufacturing operations, including our ongoing clinical trials, and conducting a process to explore strategic alternatives to maximize shareholder value, including, among other alternatives, a potential sale or licensing of our assets, an in-license arrangement, sale of the company, business combination, merger or other strategic action. In conjunction with this strategic process, we also announced a reduction of our workforce by 147 full-time employees, or approximately 95% of our then-current employee base. The reduction of workforce is expected to result in approximately $10.9 million in severance costs and such costs are primarily expected to be incurred in the second quarter of 2025. We estimate we will incur additional costs related to our wind down activities, including but not limited to impairment charges, losses on disposals of assets, contract termination costs and other incremental expenses as a result of the strategic process. The amount of such charges and costs could be material to our results of operations and financial condition. The exact amounts and timing of recognition of such charges and costs is not yet known.
Previous report
Research and development activities have been central to our business model, prior to our decision to wind down clinical and manufacturing operations in May 2025. We generally expect that our research and development expenses will decrease significantly as we wind down these operations. We may also incur other charges or cash expenditures due to events that may occur as a result of, or associated with, our wind down of clinical and manufacturing operations and review of strategic alternatives. The exact amounts of the charges and costs that we expect to incur, and the timing thereof, are currently unknown and such costs and charges could be material to our results of operation and financial condition.
Previous report
We generally expect that our general and administrative expenses will decrease significantly as we wind down our clinical and manufacturing operations, including through the implementation of our workforce reduction in May 2025. However, we may also incur other charges or cash expenditures due to events that may occur as a result of, or associated with, our wind down of clinical and manufacturing operations and review of strategic alternatives. The exact amounts of the charges and costs that we expect to incur, and the timing thereof, are currently unknown and such costs and charges could be material to our results of operation and financial condition.
Previous report
Other Income
Previous report
Research and development expenses were $26.7 million for the three months ended March 31, 2025, compared to $24.3 million for the three months ended March 31, 2024. The increase of $2.4 million was primarily due to an increase in clinical trial costs to support our trem-cel and VCAR33 programs as well as an increase in lab supplies and consumables.
Previous report
General and administrative expenses were $6.6 million for the three months ended March 31, 2025, compared to $8.0 million for the three months ended March 31, 2024. The decrease of $1.4 million was primarily due to a decrease in stock-based compensation expense and personnel costs.
Previous report
Other Income
Previous report
Other income decreased by $0.7 million during the three months ended March 31, 2025, compared to the three months ended March 31, 2024. The decrease in other income was due to decreases in interest earned from our cash, cash equivalents, restricted cash equivalents and marketable securities as we held lower balances of cash, cash equivalents and marketable securities during the 2025 period.
Previous report
However, as of the date of filing our Shelf Registration Statement, our public float was less than $75 million. As a result, we are subject to the limitations of General Instruction I.B.6 to Form S-3 until such time as our public float exceeds $75 million, which means we only have the capacity to sell shares up to one-third of our public float under shelf registration statements in any twelve-month period. We will remain constrained by the limitations of General Instruction I.B.6 to Form S-3 until such time as our public float exceeds $75 million, at which time the number of securities we may sell under a Form S-3 registration statement will no longer be constrained by the limitations of General Instruction I.B.6 to Form S-3.
Previous report
Based on our current business plan and current capital resources, management has concluded that there is substantial doubt regarding our ability to continue as a going concern for a period of 12 months from the date of issuance of the accompanying condensed consolidated financial statements. The accompanying financial statements have been prepared on a going concern basis and do not include any adjustments to the carrying amounts and classification of assets and liabilities that may be necessary if we were unable to continue as a going concern.
Previous report
We expect our expenses to decrease substantially in connection with the wind down of our clinical and manufacturing operations. However, we may incur substantial additional expenses and other charges as we conduct our strategic alternatives review and evaluate and seek to consummate strategic transactions to maximize shareholder value.
Previous report
If, for any reason, we utilize our capital resources more quickly than anticipated or are unable to obtain additional funding on a timely basis and/or complete a strategic transaction, we may be required to further revise our business plan and strategy or cease operations. This may limit the strategic options available to us. As a result, our business, financial condition, and results of operations could be materially affected.
Previous report
As discussed within Note 13, Subsequent Events, to our unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q, on May 5, 2025, the Company’s board of directors approved the wind down of our clinical and manufacturing operations. As a result, our existing contractual obligations and commitments may be renegotiated, amended or altered as we wind down operations and explore strategic alternatives to maximize shareholder value. The estimated impact of such activities was described within our Current Report on Form 8-K filed on May 8, 2025.
Reworded paragraphs (18)
Previous wording
We have incurred significant operating losses since inception, including net losses of $32.5 million for the three months ended March 31, 2025 and $116.9 million for the year ended December 31, 2024. As of March 31, 2025, we had an accumulated deficit of $489.5 million.
Current 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.
Previous wording
As of March 31, 2025, we had cash, cash equivalents and marketable securities of $60.0 million. We do not expect that our cash, cash equivalents and marketable securities at March 31, 2025 will enable us to fund our operations beyond one year from the date that these condensed consolidated financial statements are issued.
Current 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.
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. There have been no material changes to our critical accounting estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 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. 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.
Previous wording
We have not generated any revenue since our inception and do not expect to generate any revenue from the sale of products in the near future, if at all. If our development efforts for any product candidates that we may in-license as part of our strategic review process are successful and result in marketing approval, or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from a combination of product sales or payments from such agreements.
Current wording
We have not generated any revenue since our inception and do not expect to generate any revenue from the sale of products in the near future, if at all. If our development efforts for our current or future product candidates are successful and result in marketing approval, or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from a combination of product sales or payments from such agreements.
Previous wording
payments made under third party licensing agreements.
Current wording
payments made and consideration issued under third party licensing agreements.
Previous wording
The successful development of our product candidates or any product candidates that we may in-license as part of our strategic review in the future is highly uncertain. Therefore, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development and commercialization of any of our product candidates. We are also unable to predict when, if ever, material net cash inflows will commence from the sale of our product candidates, if approved. This is due to the numerous risks and uncertainties associated with developing product candidates, many of which are outside of our control, including the uncertainty of successfully completing clinical trials with safety, tolerability and efficacy profiles that are satisfactory to the FDA or any comparable foreign regulatory authority; the timing, receipt and terms of any regulatory approvals from applicable regulatory authorities; establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; government regulation and regulatory guidance; obtaining, maintaining, defending and enforcing intellectual property rights; launching commercial sales of product candidates, if approved; and the availability of funding sufficient for foreseeable and unforeseeable operating expenses and capital expenditure requirements. Any changes in the outcome of any of these variables could mean a significant change in the costs and timing associated with the development of our product candidates.
Current wording
The successful development of our product candidates in the future is highly uncertain. Therefore, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development and commercialization of any of our product candidates. We are also unable to predict when, if ever, material net cash inflows will commence from the sale of our product candidates, if approved. This is due to the numerous risks and uncertainties associated with developing product candidates, many of which are outside of our control, including the uncertainty of:
Previous wording
Comparison of Three Months Ended March 31, 2025 and 2024
Current wording
Comparison of Three and Six Months Ended June 30, 2025 and 2024
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 $517.1 million as of March 31, 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 $691.5 million as of June 30, 2025.
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 and subject to the limitations of General Instruction I.B.6 of Form S-3. 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 three months ended March 31, 2025. As of March 31, 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, 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.
Previous wording
As of March 31, 2025, we had cash, cash equivalents and marketable securities of $60.0 million. Depending on the outcome of our strategic review process, we may need to raise substantial additional capital to fund our 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.
Current 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.
Previous wording
If we are successful in consummating a strategic transaction that enables us to continue to develop our current or new product candidates, we will require substantial additional funding to expand our operations, hire additional personnel, initiate and successfully complete preclinical studies and clinical trials, obtain marketing approval and generate revenue from sales of our product candidates, if approved, and protect our intellectual property. We would 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. Because of the uncertain outcome of our strategic review process and the numerous risks and uncertainties associated with product development, we cannot predict the timing or amount of our future expenses, and there is no assurance that we will ever be profitable or generate positive cash flow from operating activities.
Current wording
maintain, expand, enforce, defend and protect our intellectual property;
Previous wording
Net cash used in operating activities was $31.1 million for the three months ended March 31, 2025, reflecting a net loss of $32.5 million and net cash used of $2.6 million for operating assets and liabilities, which were partially offset by non-cash charges of $4.1 million. The non-cash charges primarily consisted of stock-based compensation expense of $1.9 million, non-cash lease expense of $1.3 million and depreciation expense of $0.8 million.
Current 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.
Previous wording
Net cash used in operating activities was $30.5 million for the three months ended March 31, 2024, reflecting a net loss of $30.8 million and net cash used of $4.1 million for operating assets and liabilities, which were partially offset by non-cash charges of $4.5 million. The non-cash charges primarily consisted of stock-based compensation expense of $3.1 million, non-cash lease expense of $1.2 million and depreciation expense of $0.9 million, partially offset by $0.8 million of non-cash interest earned on marketable securities.
Current 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.
Previous wording
The $0.6 million increase in net cash used in operating activities for the three months ended March 31, 2025 compared to the three months ended March 31, 2024 was primarily due to an increase in research and development expenses and decrease in stock-based compensation, partially offset by differences in the timing of payments for costs incurred during each respective period.
Current wording
The $14.0 million increase in net cash used in operating activities for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was due to an increase in net loss, partially offset by differences in the timing of payments for costs incurred during each respective period and an increase in non-cash charges from the acquired in-process research and development and change in fair value of warrant liabilities.
Previous wording
Net cash used in investing activities was $0.2 million for the three months ended March 31, 2025, which consisted primarily of purchases of $0.2 million of property and equipment. Net cash provided by investing activities was $44.0 million for the three months ended March 31, 2024, which consisted of proceeds of $54.0 million from the maturity of marketable securities, partially 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 $0.4 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.
Previous wording
Net cash used in financing activities was $0.6 million for the three months ended March 31, 2025, which consisted of $0.5 million of issuance costs related to the December 2024 private placement and $0.1 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $0.1 million for the three months ended March 31, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.3 million, partially 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 $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.
Previous wording
Other commitments include license and collaboration agreements we have entered into with certain parties. Such arrangements require ongoing payments, including payments upon the achievement of certain development, regulatory and commercial milestones, receipt of sublicense income, as well as royalties on commercial sales. Payments under these arrangements are expensed as incurred.
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 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.
Previous wording
Contractual obligations relate to future minimum lease payments for existing non-cancellable leases primarily relating to corporate office and laboratory real estate, with terms expiring through February 2030. During the three months ended March 31, 2025, there were no significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2024 Annual Report.
Current 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.
Rearranged current paragraphs (6)
Current report — wording carried from elsewhere
the successful completion of clinical trials with safety, tolerability and efficacy profiles that are satisfactory to the FDA or any comparable foreign regulatory authority;
Current report — wording carried from elsewhere
the timing, receipt and terms of any regulatory approvals from applicable regulatory authorities;
Current report — wording carried from elsewhere
establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers;
Current report — wording carried from elsewhere
significant and changing government regulation and regulatory guidance;
Current report — wording carried from elsewhere
obtaining, maintaining, defending and enforcing patent claims and other intellectual property rights;
Current report — wording carried from elsewhere
Any changes in the outcome of any of these variables could mean a significant change in the costs and timing associated with the development of our product candidate or any future product candidate.