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, and anemia of chronic kidney disease, or CKD; and DISC-3405 for the treatment of polycythemia vera, or PV, and other hematologic disorders. In addition, our preclinical programs also 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, and Diamond-Blackfan Anemia, or DBA; DISC-0974 for the treatment of anemia of myelofibrosis, or MF, and anemia of chronic kidney disease, or CKD; and DISC-3405 for the treatment of polycythemia vera, or PV, 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 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. Under the FDA’s Accelerated Approval Program, we would have the potential to submit a New Drug Application, or NDA, for bitopertin in EPP and XLP based on our existing data, and we would be required to conduct a post-marketing confirmatory clinical trial. 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. We have now initiated the APOLLO trial and anticipate submitting an NDA for accelerated approval of bitopertin in EPP and XLP in the second half of 2025. 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 U.S. We have also entered into a collaborative research and development agreement with the National Ins…

Excerpt shortened here; the source contains the full paragraph.

Current 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. Under the FDA’s Accelerated Approval Program, we would have the potential to submit a New Drug Application, or NDA, for bitopertin in EPP and XLP based on our existing data, and we would be required to conduct a post-marketing confirmatory clinical trial. 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. We initiated the APOLLO trial in May 2025. Based on feedback from the FDA at our pre-NDA meeting, we plan to submit an NDA in October 2025 for accelerated approval of bitopertin in EPP and XLP in the United State…

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 initiated the open-label Phase 2 portion of this clinical trial. We expect to report initial data from this Phase 2 trial in the second half of 2025. We also expect to report data from the multiple-ascending dose, or MAD, portion of the Phase 1b trial in patients with non-dialysis dependent CKD and anemia in the second half of 2025. We are also planning additional trials of DISC-0974 in other anemias of inflammation. In addition, we are 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.

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 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 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 in the second half of 2025. We also expect to report data from the multiple-ascending dose, or MAD, portion of the Phase 1b trial in patients with non-dialysis dependent CKD and anemia in the second half of 2025. We are also planning additional trials of DISC-0974 in other anemias of inflammation. In addition, we are 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 MAD portion of this Phase 1 healthy volunteer study of DISC-3405 in December 2024. We expect to develop DISC-3405 for the treatment of PV and other hematologic disorders and plan to initiate a Phase 2 clinical trial of DISC-3405 in the first half of 2025.

Current 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 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 and other hematologic disorders and initiated a Phase 2 clinical trial of DISC-3405 in PV in the first half of 2025.

Previous wording

In January 2025 we completed an underwritten offering of shares of our common stock and pre-funded warrants for net proceeds of $243.4 million, after deducting offering expenses. As of March 31, 2025, we had cash, cash equivalents and marketable securities of $694.7 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 2028, 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

In January 2025 we completed an underwritten offering of shares of our common stock and pre-funded warrants for net proceeds of $243.4 million, after deducting offering expenses. As of June 30, 2025, we had cash, cash equivalents and marketable securities of $650.0 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 2028, 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

Comparison of the Three Months Ended March 31, 2025 and 2024

Current wording

Comparison of the Three and Six Months Ended June 30, 2025 and 2024

Previous wording

The following table summarizes our results of operations for the three months ended March 31, 2025 and 2024 (in thousands):

Current wording

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

Previous wording

The following table summarizes our research and development expenses for the three months ended March 31, 2025 and 2024 (in thousands):

Current wording

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

Previous wording

Research and development expenses were $27.8 million for the three months ended March 31, 2025, compared to $23.7 million for the three months ended March 31, 2024. The increase of $4.1 million was primarily due to increases of $2.4 million and $2.1 million in DISC-0974 and bitopertin development expense, respectively, related to advancing the clinical trials and drug manufacturing activity. In addition, personnel-related costs increased $2.5 million related to higher research and development headcount, including an increase of $1.4 million in stock-based compensation driven by awards granted under our equity compensation plans. These increases were partially offset by a decrease of $3.0 million in DISC-3405 development expense related to manufacturing costs incurred in the three months ended March 31, 2024 that did not recur in the three months ended March 31, 2025.

Current wording

Research and development expenses were $46.3 million for the three months ended June 30, 2025, compared to $23.5 million for the three months ended June 30, 2024. The increase of $22.8 million was primarily due to an increase of $16.6 million in bitopertin development expense, including a $10.0 million milestone payment triggered by the initiation of our APOLLO trial. The remaining $6.6 million increase reflects ongoing costs associated with both the APOLLO study and the HELIOS long-term extension trial. Additionally, DISC-0974 development expenses increased $1.3 million primarily due to clinical trial advancement and drug manufacturing activities. Personnel-related costs increased $4.4 million related to higher research and development headcount, including an increase of $2.2 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also an increase of $2.5 million in other research programs and expenses related to expanded research initiatives and ongoing program support. These increases were partially offset by a decrease of $2.0 million in DISC-3405 development expense related to manufacturing costs incurred in the three months ended June 30, 2024 that did not recur in the three months ended June 30, 2025.

Previous wording

The following table summarizes our selling, general and administrative expenses for the three months ended March 31, 2025 and 2024 (in thousands):

Current wording

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

Previous wording

Selling, general and administrative expenses were $12.2 million for the three months ended March 31, 2025, compared to $7.8 million for the three months ended March 31, 2024. The increase of $4.4 million was due to an increase of $2.0 million in personnel-related costs due to higher selling, general and administrative headcount, including an increase of $0.9 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $2.1 million increase in legal, consulting, and professional fees primarily related to developing our commercialization capabilities.

Current wording

Selling, general and administrative expenses were $15.1 million for the three months ended June 30, 2025, compared to $7.4 million for the three months ended June 30, 2024. The increase of $7.7 million was due to an increase of $3.6 million in personnel-related costs due to higher selling, general and administrative headcount, including an increase of $2.2 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $3.6 million increase in legal, consulting, and professional fees primarily related to developing our commercialization capabilities.

Previous wording

Income tax expense was $0.1 million for the three months ended March 31, 2025, compared to less than $0.1 million for the three months ended March 31, 2024. This expense relates to state income tax resulting from an increase in interest income.

Current wording

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

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 March 31, 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, $721.0 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, 2025, we had cash, cash equivalents and marketable securities of $694.7 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 June 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, $721.0 million from various private and public sales of our equity securities, and $27.6 million from borrowings under the Hercules Loan Agreement. As of June 30, 2025, we had cash, cash equivalents and marketable securities of $650.0 million.

Previous wording

We have incurred significant operating losses since inception and, as of March 31, 2025, had an accumulated deficit of $332.1 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 2028 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 financings.

Current wording

We have incurred significant operating losses since inception and, as of June 30, 2025, had an accumulated deficit of $387.3 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 2028 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 our Term Loan Facility.

Previous 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.

Current wording

During the six months ended June 30, 2025, net cash used in operating activities of $89.3 million was primarily due to our net loss of $89.3 million and the change in operating assets and liabilities of $11.2 million. These were partially offset by non-cash expenses of $11.2 million which included $14.8 million in stock-based compensation, reduced by $5.1 million from amortization and accretion of investment securities.

Previous wording

During the three months ended March 31, 2024, net cash used in operating activities of $33.6 million was primarily due to our net loss of $26.9 million, which includes interest income of $4.5 million; and changes in operating assets and liabilities of $10.9 million, including a milestone payment of $5.0 million to Mabwell; offset by non-cash expenses of $4.2 million primarily related to stock-based compensation expense of $4.1 million.

Current wording

During the six months ended June 30, 2024, net cash used in operating activities of $49.0 million was primarily due to our net loss of $53.3 million, which included interest income of $9.1 million, and changes in operating assets and liabilities of $3.7 million, which included a reduction in accounts payable related to a milestone payment to Mabwell of $5.0 million. These were partially offset by non-cash expenses of $8.0 million primarily related to stock-based compensation expense of $8.1 million.

Previous 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.

Current wording

During the six months ended June 30, 2025, net cash used in investing activities was primarily due to purchases of marketable securities of $405.9 million, partially offset by maturities of marketable securities of $185.8 million.

Previous wording

During the three months ended March 31, 2024, net cash used in investing activities was due to purchases of property and equipment.

Current wording

During the six months ended June 30, 2024, net cash used in investing activities was primarily due to purchases of marketable securities.

Previous 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.

Current wording

During the six months ended June 30, 2025, net cash provided by financing activities of $245.1 million consisted primarily of aggregate net proceeds of $243.4 million from our underwritten offering in January 2025.

Previous wording

During the three months ended March 31, 2024, net cash provided by financing activities of $15.9 million consisted primarily of aggregate net proceeds of $14.8 million from the at-the-market offerings.

Current wording

During the six months ended June 30, 2024, net cash provided by financing activities of $189.1 million consisted primarily of cash net proceeds of $172.7 million from the underwritten offering.