Disc Medicine, Inc.

Previous wording

We are a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. We have assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, specifically heme biosynthesis and iron homeostasis. Our current pipeline includes bitopertin for the treatment of erythropoietic porphyrias, or EPs, including erythropoietic protoporphyria, or EPP, and X-linked protoporphyria, or XLP, and Diamond-Blackfan Anemia, or DBA; DISC-0974 for the treatment of anemia of myelofibrosis, or MF, anemia of inflammatory bowel disease, or IBD, and anemia of chronic kidney disease, or CKD; and DISC-3405 for the treatment of polycythemia vera, or PV, sickle cell disease, or SCD, and other hematologic disorders. In addition, our preclinical programs include DISC-0998 for the treatment of anemia associated with inflammatory diseases. Our approach to product candidate development leverages well-understood molecular mechanisms that have been validated in humans. We believe that each of our product candidates, if approved, has the potential to improve the lives of patients suffering from hematologic diseases.

Current wording

We are a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. We have assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, specifically heme biosynthesis and iron homeostasis. Our current pipeline includes bitopertin for the treatment of erythropoietic porphyrias, or EPs, including erythropoietic protoporphyria, or EPP, and X-linked protoporphyria, or XLP; DISC-0974 for the treatment of anemia of myelofibrosis, or MF, and anemia of inflammatory bowel disease, or IBD; and DISC-3405 for the treatment of polycythemia vera, or PV, sickle cell disease, or SCD, and other hematologic disorders. In addition, our preclinical programs include DISC-0998 for the treatment of anemia associated with inflammatory diseases. Our approach to product candidate development leverages well-understood molecular mechanisms that have been validated in humans. We believe that each of our product candidates, if approved, has the potential to improve the lives of patients suffering from hematologic diseases.

Previous wording

Bitopertin is the lead product candidate in our heme biosynthesis modulation portfolio. Bitopertin was previously evaluated by F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., or collectively, Roche, in a comprehensive clinical program in over 4,000 individuals in other indications which demonstrated the activity of bitopertin as a glycine transporter 1, or GlyT1, inhibitor and its effect on heme biosynthesis. We are initially developing bitopertin for the treatment of EPs, including EPP and XLP, which are part of a group of severe diseases, known as porphyrias, caused by defects in the heme biosynthesis pathway that cause an accumulation of toxic metabolites referred to as porphyrins, resulting in skin hypersensitivity to sunlight and some types of artificial light. In June and December 2023, we presented interim data from BEACON, a Phase 2 open-label, parallel-dose clinical trial of bitopertin in EPP and XLP patients conducted at sites in Australia. In April 2024, we presented topline data from AURORA, a Phase 2, randomized, double-blind, placebo-controlled clinical trial of bitopertin in EPP patients conducted at sites in the United States. Additional analyses of the BEACON and AURORA trials were presented in June 2024 and in December 2024. In both trials, bitopertin significantly reduced the toxic metabolite, protoporphyrin IX, or PPIX, and was associated with improvements in measures of time spent in sunlight and quality of life, demonstrating a clear association between PPIX reduction and clinical endpoints. In addition, bitopertin was generally well-tolerated. All participants who complete AURORA or BEACON are eligible to participate in HELIOS, an ongoing open-label, long-term extension study of bitopertin in EPP and XLP. In June 2025, we presented interim data from HELIOS, in which longer term treatment of bitopertin was associated with sustained reductions in PPIX, as well as improvements in quality of life, and improved liver biomarkers. In our end-of-Phase 2 meeting, the U.S. Food & Drug Administration, or the FDA, agreed with the potential for reduction of PPIX to serve as a surrogate endpoint to support a potential accelerated approval of bitopertin in EPP and XLP. In our Type C meeting with the FDA in December 2024, we aligned with the FDA on the design of APOLLO, a randomized, double-blind, placebo-controlled, clinical trial of bitopertin in EPP and XLP patients that in the setting of an accelerated approval would serve as a post-marketing confirmatory clinical trial. APOLLO is also intended to serve as a registrational trial with respect to any potential future marketing applications for bitopertin in EPP and XLP in jurisdictions outside the United States. We initiated the APOLLO trial in May 2025, and on September 29, 2025 we submitted an NDA for accelerated approval of bitopertin in EPP and XLP in the United States based on our existing data. In October 2025, we were awarded a Commissioner’s National Priority Voucher, or CNP…

Excerpt shortened here; the source contains the full paragraph.

Current wording

We have completed two Phase 2 clinical trials of bitopertin: BEACON, an open-label, parallel-dose clinical trial in EPP and XLP patients conducted at sites in Australia, and AURORA, a randomized, double-blind, placebo-controlled clinical trial in EPP patients conducted at sites in the United States. In both trials, bitopertin significantly reduced the toxic metabolite, protoporphyrin IX, or PPIX, and was associated with improvements in measures of time spent in sunlight and quality of life. In addition, bitopertin was generally well-tolerated. All participants in AURORA and BEACON were eligible to participate in HELIOS, an ongoing open-label, long-term extension study of bitopertin in EPP and XLP patients. Interim data from HELIOS, presented in June 2025, demonstrated that longer term treatment of bitopertin was associated with sustained reductions in PPIX, as well as improvements in quality of life and liver biomarkers. In our end-of-Phase 2 meeting in September 2024, the U.S. Food & Drug Administration, or the FDA, agreed with the potential for reduction of PPIX to serve as a surrogate endpoint to support a potential accelerated approval of bitopertin in EPP and XLP. In our Type C meeting in December 2024, we aligned with the FDA on the design of APOLLO, a Phase 3, randomized, double-blind, placebo-controlled, clinical trial of bitopertin in EPP and XLP patients that in the setting of an accelerated approval would serve as a post-marketing confirmatory trial. We initiated the APOLLO trial in May 2025 and in September 2025 we submitted a New Drug Application, or NDA, for accelerated approval of bitopertin in EPP and XLP in the United States based on our existing data. In October 2025, we were awarded a Commissioner’s National Priority Voucher, or CNPV, from the FDA for bitopertin in EPP and XLP. The CNPV program, announced in June 2025, is designed to accelerate the development and review of certain drugs aligned with US national health priorities. The FDA accepted our NDA for review in November 2025 and in February 2026 issued a complete response letter, or CRL. Although the FDA agreed that clinical data from AURORA and BEACON provided sufficient evidence that bitopertin significantly lowers whole blood metal-free PPIX, the FDA concluded that the trials did not show evidence of association between percent change in PPIX and sunlight exposure-based endpoints as measured in the trials. As such, the FDA determined that there is uncertainty regarding whether bitopertin’s effect on PPIX is reasonably likely to predict clinical benefit, despite the strong mechanistic and biological plausibility supporting the use of the PPIX biomarker in protoporphyria. The FDA indicated that results of the ongoing APOLLO trial could serve as evidence to support a potential traditional approval, and we have scheduled a Type A meeting with the FDA during the second quarter of 2026 to review our approach for potentially resubmitting our NDA. We completed enrollment in …

Excerpt shortened here; the source contains the full paragraph.

Previous wording

DISC-0974 is the lead product candidate in our iron homeostasis portfolio and was licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. DISC-0974 is designed to suppress hepcidin production and increase serum iron levels. We completed a Phase 1 clinical trial in healthy volunteers in the U.S. in June 2022 with results showing an acceptable tolerability profile and evidence of target engagement, iron mobilization and augmented erythropoiesis. We initiated a Phase 1b/2 clinical trial in June 2022 in patients with anemia of MF, and initiated a separate Phase 1b/2 clinical trial in February 2023 in patients with non-dialysis dependent CKD and anemia. We presented interim data from both of these trials in December 2023 as well as additional interim data for anemia of MF in June 2024 and for non-dialysis dependent CKD and anemia in October 2024. This additional interim data included safety data and changes in hepcidin, iron, and hemoglobin levels for additional patients, as well as longer follow-up. In December 2024, we presented additional analyses of the Phase 1b study in anemia of MF, and in June 2025 we presented longer term data from the continuation phase of this study. We initiated RALLY-MF, the open-label Phase 2 portion of this clinical trial, in December 2024 and we expect to report initial data from this Phase 2 trial at the American Society of Hematology Annual Meeting in December 2025. Topline data from this trial is expected in 2026, which, if positive, is expected to support discussions with regulatory agencies on the potential regulatory path for DISC-0974 in anemia of MF. In October 2025, we announced that our recently completed Phase 1b, double-blind, placebo-controlled study of DISC-0974 in non-dialysis dependent CKD and anemia demonstrated engagement of mechanism with variable effects on hemoglobin. In this trial, DISC-0974 was generally well-tolerated and associated with substantial decreases in hepcidin, increases in iron, and improvements in markers of erythropoiesis. Meaningful hemoglobin increases observed in only a subset of patients were in part driven by those with higher baseline erythropoietin levels. We expect to report full data from this trial at the American Society of Nephrology (ASN) Kidney Week in November 2025, and we are assessing options for our DISC-0974 non-dialysis dependent CKD and anemia program based on full analysis of this data. We are also planning additional trials of DISC-0974 in other anemias of inflammation. For example, we plan to initiate a Phase 2 clinical trial of DISC-0974 in patients with IBD and anemia in the first quarter of 2026, and we are planning exploratory studies in additional patient populations with anemia of chronic disease. We are also developing a preclinical anti-hemojuvelin, or HJV, monoclonal antibody, DISC-0998, which also targets hepcidin suppression and was licensed from AbbVie. DISC-0998 is designed to increase serum iron levels and has an extended serum half-life as c…

Excerpt shortened here; the source contains the full paragraph.

Current wording

DISC-0974 is the lead product candidate in our iron homeostasis portfolio and was licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. DISC-0974 is designed to suppress hepcidin production and increase serum iron levels. We completed a Phase 1 clinical trial in healthy volunteers in the United States in June 2022, with results showing an acceptable tolerability profile and evidence of target engagement, iron mobilization and augmented erythropoiesis. We initiated a Phase 1b/2 clinical trial in June 2022 in patients with anemia of MF, with results from the Phase 1b portion of this clinical trial showing substantial and sustained reductions in hepcidin levels and increases in iron alongside strong hematologic responses across patient types and an acceptable tolerability profile. We initiated RALLY-MF, the open-label Phase 2 portion of this clinical trial, in December 2024 and reported initial data in December 2025, with results demonstrating meaningful overall anemia responses across all patient subgroups, regardless of baseline transfusion status and independent of concomitant Janus Kinase, or JAK, inhibitor therapy use. We expect to share updated data from this trial in June 2026 and topline data in the fourth quarter of 2026. If positive, we expect to hold an end-of-Phase 2 meeting with the FDA for DISC-0974 in anemia of MF by the end of 2026. We also initiated a Phase 2 clinical trial of DISC-0974 in patients with IBD and anemia in the first quarter of 2026, with initial data anticipated in 2027, and we are planning exploratory studies in additional patient populations with anemia of chronic disease. We are also developing a preclinical anti-hemojuvelin, or HJV, monoclonal antibody, DISC-0998, which also targets hepcidin suppression and was licensed from AbbVie. DISC-0998 is designed to increase serum iron levels and has an extended serum half-life as compared to DISC-0974. We believe this profile may be desirable in certain subsets of patients with anemia associated with inflammatory diseases.

Previous wording

Lastly, we are developing DISC-3405, a monoclonal antibody against Transmembrane Serine Protease 6, or TMPRSS6, that we licensed from Mabwell Therapeutics, Inc., or Mabwell. DISC-3405 is part of our iron homeostasis portfolio and is designed to induce hepcidin production and reduce serum iron levels. We initiated a Phase 1 clinical trial of DISC-3405 in healthy adult volunteers in October 2023 and presented interim data from the single-ascending dose, or SAD, portion of this clinical trial in June 2024. We presented data from the multiple ascending dose, or MAD, portion of this Phase 1 healthy volunteer study of DISC-3405 in December 2024, and presented updated data from both the SAD and MAD portions of this clinical trial in June 2025. We are developing DISC-3405 for the treatment of PV, SCD, and other hematologic disorders. We initiated a Phase 2 clinical trial of DISC-3405 in PV in the first half of 2025, with initial data expected in 2026. We also initiated a Phase 1b clinical trial of DISC-3405 in patients with SCD in October 2025, with initial data expected in 2026, and plan to explore the role of therapeutic iron restriction in other indications.

Current wording

In addition, we are developing DISC-3405, a monoclonal antibody against Transmembrane Serine Protease 6, or TMPRSS6, that we licensed from Mabwell Therapeutics, Inc., or Mabwell. DISC-3405 is part of our iron homeostasis portfolio and is designed to induce hepcidin production and reduce serum iron levels. We initiated a Phase 1 clinical trial of DISC-3405 in healthy adult volunteers in October 2023 and presented interim data from the single-ascending dose, or SAD, portion of this clinical trial in June 2024. We presented data from the multiple ascending dose, or MAD, portion of this clinical trial in December 2024, and presented updated data from both the SAD and MAD portions of this clinical trial in June 2025, with results showing an acceptable tolerability profile and evidence of target engagement, including dose-related increases in serum hepcidin and corresponding reductions in serum iron across all dose levels. We are developing DISC-3405 for the treatment of PV, SCD, and other hematologic disorders. We initiated RESTORE-PV, an open-label Phase 2 clinical trial of DISC-3405 in patients with PV, in the first half of 2025, with initial data expected in the fourth quarter of 2026. We also initiated a Phase 1b, open-label clinical trial of DISC-3405 in patients with SCD in October 2025, with initial data expected in the fourth quarter of 2026, and plan to explore the role of therapeutic iron restriction in other indications.

Previous wording

As of September 30, 2025, we had cash, cash equivalents and marketable securities of $615.9 million. In October 2025, we completed an underwritten public offering of shares of our common stock and pre-funded warrants for net proceeds of approximately $210.9 million, after deducting estimated offering expenses payable by us. We believe that our cash, cash equivalents and marketable securities, including the net proceeds of our October 2025 underwritten public offering, will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029, without taking into account any potential net cash inflows from bitopertin or any other marketed product, if approved during such period. We have based these estimates on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. See "Liquidity and Capital Resources" for additional details.

Current wording

As of March 31, 2026, we had cash, cash equivalents and marketable securities of $730.2 million. We believe that our cash, cash equivalents and marketable securities will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029, without taking into account any potential net cash inflows from bitopertin or any other marketed product, if approved during such period. We have based these estimates on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. See "Liquidity and Capital Resources" for additional details.

Previous wording

We expect that our research and development expenses will increase substantially as we advance our programs into and through clinical development. At this time, we cannot accurately estimate or know the nature, timing and costs of the efforts that will be necessary to complete the preclinical and clinical development of any product candidates we may develop. A change in the outcome of any number of variables with respect to product candidates we may develop could significantly change the costs and timing associated with the development of that product candidate. We may never succeed in obtaining regulatory approval for any product candidates we may develop. The successful development of any product candidate is highly uncertain. This is due to the numerous risks and uncertainties associated with product development, including the following:

Current wording

We expect that our research and development expenses will increase substantially as we advance our programs into and through clinical development. At this time, we cannot accurately estimate or know the nature, timing or costs of the efforts that will be necessary to complete their preclinical and clinical development. Changes in the outcome of any number of variables with respect to these programs could significantly affect the costs and timing associated with their development. We may never succeed in obtaining regulatory approval for any product candidates that have not yet been approved. The successful development of any product candidate is highly uncertain due to the numerous risks and uncertainties inherent in product development, including the following:

Previous wording

We anticipate that our selling, general and administrative expenses will increase substantially in the future as we increase our headcount to support our continued research and development and potential commercialization activities, including establishing a sales, marketing and distribution infrastructure to commercialize any product candidates for which we may obtain marketing approval.

Current wording

We anticipate that our selling, general and administrative expenses will increase substantially in the future as we increase our headcount to support our continued research and development and potential commercialization activities, including establishing the infrastructure to commercialize any product candidates for which we may obtain marketing approval.

Previous wording

Interest income primarily consists of interest earned on cash equivalents, consisting of money market funds, U.S. treasury securities and certificates of deposit, as well as marketable securities, consisting of U.S. treasury securities, U.S. government agency securities and corporate debt securities.

Current wording

Interest income primarily consists of interest earned on cash equivalents, consisting of money market funds and corporate debt securities, as well as marketable securities, consisting of U.S. treasury securities, U.S. government agency securities and corporate debt securities.

Previous wording

Comparison of the Three and Nine Months Ended September 30, 2025 and 2024

Current wording

Comparison of the Three Months Ended March 31, 2026 and 2025

Previous wording

The following table summarizes our results of operations for the three and nine months ended September 30, 2025 and 2024 (in thousands):

Current wording

The following table summarizes our results of operations for the three months ended March 31, 2026 and 2025 (in thousands):

Previous wording

The following table summarizes our research and development expenses for the three and nine months ended September 30, 2025 and 2024 (in thousands):

Current wording

The following table summarizes our research and development expenses for the three months ended March 31, 2026 and 2025 (in thousands):

Previous wording

Research and development expenses were $50.3 million for the three months ended September 30, 2025, compared to $24.7 million for the three months ended September 30, 2024. The increase of $25.6 million was primarily due to an increase of $8.7 million in DISC-3405 development expenses driven by a $10.0 million milestone payment triggered by the first administration to a patient in a Phase 2 clinical trial. Personnel-related costs increased $7.0 million related to higher research and development headcount, including an increase of $2.7 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $4.6 million increase in bitopertin development expense reflecting ongoing costs associated with both the APOLLO trial and the HELIOS long-term extension trial. Other research programs and expenses increased $3.7 million related to expanded research initiatives and ongoing program support. Additionally, DISC-0974 development expenses increased $1.7 million primarily due to clinical trial advancement and drug manufacturing activities.

Current wording

Research and development expenses were $45.9 million for the three months ended March 31, 2026, compared to $27.8 million for the three months ended March 31, 2025. The increase of $18.1 million was primarily due to an increase of $7.5 million in DISC-3405 development expenses. This increase was primarily driven by a $5.0 million milestone payment triggered by the first administration to a patient in the Phase 1b clinical trial of DISC-3405 in a second indication. Personnel-related costs increased $7.1 million related to increased research and development headcount, including an increase of $2.8 million in stock-based compensation driven by awards granted under our equity compensation plans and an increase of $0.2 million related to an organizational restructuring. DISC-0974 development expenses increased $2.0 million primarily due to clinical trial advancement and drug manufacturing activities. There was also a $0.9 million increase in bitopertin development expense reflecting ongoing costs associated with both the APOLLO trial and the HELIOS long-term extension trial. Other research programs and expenses increased $0.6 million related to expanded research initiatives and ongoing program support.

Previous wording

The following table summarizes our selling, general and administrative expenses for the three and nine months ended September 30, 2025 and 2024 (in thousands):

Current wording

The following table summarizes our selling, general and administrative expenses for the three months ended March 31, 2026 and 2025 (in thousands):

Previous wording

Selling, general and administrative expenses were $17.4 million for the three months ended September 30, 2025, compared to $8.2 million for the three months ended September 30, 2024. The increase of $9.2 million was due to an increase of $5.6 million in personnel-related costs due to higher selling, general and administrative headcount, including an increase of $2.4 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $3.0 million increase in legal, consulting, and professional fees primarily related to developing our commercialization capabilities.

Current wording

Selling, general and administrative expenses were $23.6 million for the three months ended March 31, 2026, compared to $12.2 million for the three months ended March 31, 2025. The increase of $11.4 million was primarily due to an increase of $8.7 million in personnel-related costs due to increased selling, general and administrative headcount, including an increase of $2.8 million in stock-based compensation driven by awards granted under our equity compensation plans and an increase of $1.6 million related to an organizational restructuring. There was also a $2.1 million increase in legal, consulting, and professional fees primarily related to developing our commercialization capabilities.

Previous wording

Other income (expense), net was $17.7 million for the nine months ended September 30, 2025, compared to $15.4 million for the nine months ended September 30, 2024. This increase of $2.3 million was primarily due to higher interest income driven by larger cash, cash equivalents and marketable securities balances, partially offset by an increase in interest expense associated with our term loan.

Current wording

Other income (expense), net was $6.1 million for the three months ended March 31, 2026, compared to $6.0 million for the three months ended March 31, 2025. Other income (expense), net consists primarily of interest income from our cash, cash equivalents and marketable securities, partially offset by interest expense associated with our term loan.

Previous wording

Income tax expense was $0.1 million and $0.3 million for the three and nine months ended September 30, 2025, respectively, compared to $0.1 million and $0.2 million for the three and nine months ended September 30, 2024, respectively. This expense primarily relates to state income tax resulting from an increase in interest income.

Current wording

Income tax expense was $0.1 million for the three months ended March 31, 2026 and 2025, respectively. This expense primarily relates to state income tax resulting from an increase in interest income.

Previous wording

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses. We expect to continue to incur significant expenses and operating losses in the foreseeable future as we advance the clinical development of our product candidates. We expect that our research and development and selling, general and administrative costs will continue to increase significantly, including in connection with conducting clinical trials and manufacturing for our product candidates to support potential commercialization and providing selling, general and administrative support for our operations, including the costs associated with operating as a public company. As a result, we may need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources. See “Risk Factors” included within Item 1A of this Quarterly Report on Form 10-Q for additional risks associated with our substantial capital requirements.

Current wording

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses. We expect to continue to incur significant expenses and operating losses in the foreseeable future as we advance the clinical development of our product candidates. We expect that our research and development and selling, general and administrative costs will continue to increase significantly, including in connection with conducting clinical trials and manufacturing for our product candidates to support commercialization and providing selling, general and administrative support for our operations, including the costs associated with operating as a public company. As a result, we may need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources. See “Risk Factors” included within Item 1A of this Quarterly Report on Form 10-Q for additional risks associated with our substantial capital requirements.

Previous wording

To date, we have funded our operations primarily with proceeds from the sale of our convertible preferred stock and common stock, the proceeds from the merger with Gemini, proceeds from various private and public sales of our equity securities and proceeds from borrowings under the Hercules Loan Agreement. Through September 30, 2025, we have received net proceeds of $144.5 million from sales of our Series Seed, Series A and Series B convertible preferred stock, $89.5 million from the merger with Gemini, $730.8 million from various private and public sales of our equity securities, and $27.6 million from borrowings under the Hercules Loan Agreement. As of September 30, 2025, we had cash, cash equivalents and marketable securities of $615.9 million.

Current wording

To date, we have funded our operations primarily with proceeds from the sale of our convertible preferred stock and common stock, the proceeds from the merger with Gemini, proceeds from various private and public sales of our equity securities and proceeds from borrowings under the Hercules Loan Agreement. Through March 31, 2026, we have received net proceeds of $144.5 million from sales of our Series Seed, Series A and Series B convertible preferred stock, $89.5 million from the merger with Gemini, $941.8 million from various private and public sales of our equity securities, and $27.6 million from borrowings under the Hercules Loan Agreement. As of March 31, 2026, we had cash, cash equivalents and marketable securities of $730.2 million.

Previous wording

We have incurred significant operating losses since inception and, as of September 30, 2025, had an accumulated deficit of $449.7 million. In addition, we expect to continue to incur significant and increasing expenses and operating losses for the foreseeable future. We believe that our cash, cash equivalents and marketable securities, including the net proceeds of our October 2025 underwritten public offering, will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029 without taking into account any potential net cash inflows from bitopertin or any other marketed product, if approved during such period. We have based these estimates on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. We may also pursue additional cash resources through public or private equity offerings, collaborations or additional draws under the Hercules Loan Agreement.

Current wording

We have incurred significant operating losses since inception and, as of March 31, 2026, had an accumulated deficit of $573.7 million. In addition, we expect to continue to incur significant and increasing expenses and operating losses for the foreseeable future. We believe that our cash, cash equivalents and marketable securities will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029, without taking into account any potential net cash inflows from bitopertin or any other marketed product, if approved during such period. We have based these estimates on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. We may also pursue additional cash resources through public or private equity offerings, collaborations or additional debt financing.

Previous wording

During the nine months ended September 30, 2025, net cash used in operating activities of $137.1 million was primarily due to our net loss of $151.7 million and the change in operating assets and liabilities of $4.9 million. These were partially offset by non-cash expenses of $19.4 million which included $24.3 million in stock-based compensation, reduced by $6.9 million from amortization and accretion of investment securities.

Current wording

During the three months ended March 31, 2026, net cash used in operating activities of $62.2 million was primarily due to our net loss of $63.5 million and the change in operating assets and liabilities of $10.0 million. These were partially offset by non-cash expenses of $11.3 million which included $11.9 million in stock-based compensation, reduced by $1.1 million from amortization and accretion of investment securities.

Previous wording

During the nine months ended September 30, 2024, net cash used in operating activities of $66.4 million was primarily due to our net loss of $79.9 million, which includes interest income of $15.5 million; partially offset by non-cash expenses of $9.8 million primarily related to stock-based compensation expense of $12.5 million offset by amortization/accretion of investment securities of $3.1 million and changes in operating assets and liabilities of $3.7 million, including a reduction in accounts payable for the payment of a milestone payment of $5.0 million to Mabwell.

Current wording

During the three months ended March 31, 2025, net cash used in operating activities of $41.4 million was primarily due to our net loss of $34.1 million and the change in operating assets and liabilities of $11.5 million. These were partially offset by non-cash expenses of $4.2 million which included $6.4 million in stock-based compensation, reduced by $2.5 million from amortization and accretion of investment securities.

Previous wording

During the nine months ended September 30, 2024, net cash used in investing activities was primarily due to purchases of marketable securities of $313.9 million, partially offset by maturities of marketable securities of $23.0 million.

Current wording

During the three months ended March 31, 2026, net cash provided by investing activities was primarily due to maturities of marketable securities of $152.1 million, partially offset by purchases of marketable securities of $93.8 million.

Previous wording

During the nine months ended September 30, 2025, net cash used in investing activities was primarily due to purchases of marketable securities of $491.3 million, partially offset by maturities of marketable securities of $320.8 million.

Current wording

During the three months ended March 31, 2025, net cash used in investing activities was primarily due to purchases of marketable securities of $347.3 million, partially offset by maturities of marketable securities of $75.5 million.

Previous wording

During the nine months ended September 30, 2025, net cash provided by financing activities of $256.7 million consisted primarily of aggregate net proceeds of $243.4 million and $9.8 million from our underwritten offering in January 2025 and sales of shares of common stock in Cantor ATM offerings, respectively.

Current wording

During the three months ended March 31, 2025, net cash provided by financing activities of $244.3 million consisted primarily of aggregate net proceeds of $243.4 million from our underwritten offering in January 2025.

Previous wording

Until such time, if ever, as we can generate substantial revenue from product sales, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, and marketing, distribution or licensing arrangements with third parties. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders may be materially diluted, and the terms of such securities could include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include restrictive covenants that limit our ability to take specified actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings or other arrangements when needed, we may be required to delay, reduce or eliminate our product development or future commercialization efforts, or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Current wording

Until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, and marketing, distribution or licensing arrangements with third parties. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders may be materially diluted, and the terms of such securities could include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include restrictive covenants that limit our ability to take specified actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings or other arrangements when needed, we may be required to delay, reduce or eliminate our product development or future commercialization efforts, or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.