Disc Medicine Updates DISC-0974 Trial Data
Disc Medicine presented updated data from the RALLY-MF Phase 2 trial of DISC-0974 in anemia of MF at the ASCO Annual Meeting in Chicago. In this updated data set, treatment with DISC-0974 shows substantial reductions in hepcidin and increases in iron levels translating to positive impact on clinically meaningful measures of anemia across a broad range of patient types. This ongoing Phase 2 open-label study had enrolled 61 adult patients with MF and anemia as of the data cutoff date of April 27, including 50 patients with sufficient follow up to be included in the responder analysis. The trial was comprised of both patients receiving concomitant JAK inhibitor therapy and not receiving JAK inhibitor therapy. DISC-0974 was administered subcutaneously at 50 mg every 4 weeks for up to 6 treatments. The updated results demonstrated: consistent, substantial decreases in hepcidin reaching greater than 75% reduction from baseline and corresponding increases in serum iron; 55% of baseline nTD patients achieved a hemoglobin increase of 1.5 g/dL for 12 weeks and 68% had an increase of =1 g/dL for =12 weeks; 64% of TD Low patients achieved transfusion independence over a 16-week period and 73% achieved a 50% reduction in transfusions; 50% of TD High patients achieved transfusion independence over a 12-week period and 88% achieved a 50% reduction in transfusion requirement; 56% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response across transfusion groups and 72% achieved an overall response, with similar response rates regardless of which specific JAK inhibitor the patient received. Dosing with DISC-0974 was associated with improvements in patient-reported outcomes: clinically significant improvements in FACIT-Fatigue scores in nTD and TD Low participants that were correlated with hemoglobin change MPN-SAF TSS50 at EOS was achieved by 50% of nTD and TD low major responders. DISC-0974 was generally well-tolerated. Diarrhea, not considered serious, was the only adverse event that was reported as related to DISC-0974 and reported in two or more subjects. The majority of AEs were not considered related to DISC-0974. Additional data from the RALLY-MF study is to be shared in Q4, with an end of Phase 2 meeting with the FDA expected to occur by end of year. DISC-0974 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide.
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- Securities Claims Investigation: Rosen Law Firm is investigating potential securities claims against Disc Medicine (NASDAQ: IRON) for allegedly issuing materially misleading business information, allowing affected investors to seek compensation without upfront costs.
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- Securities Claims Investigation: Rosen Law Firm is investigating potential securities claims against Disc Medicine, Inc. (NASDAQ: IRON) for allegedly issuing misleading business information, indicating legal risks that could impact shareholder returns.
- FDA Response Letter: On February 13, 2026, the FDA issued a Complete Response Letter to Disc Medicine, rejecting its new drug application due to uncertainties requiring additional evidence, which led to a 22% drop in the company's stock price on the same day, reflecting market concerns about its product prospects.
- Class Action Preparation: The Rosen Law Firm is preparing a class action lawsuit against Disc Medicine, allowing affected investors to seek compensation without any out-of-pocket fees, potentially providing legal support to help recover losses.
- Law Firm Background: The Rosen Law Firm specializes in securities class actions and has recovered over $438 million for investors in 2019 alone, demonstrating its strong capabilities and successful track record in securities litigation, which investors should consider when selecting legal counsel.
- Securities Claims Investigation: Rosen Law Firm is investigating potential securities claims on behalf of shareholders of Disc Medicine, Inc. (NASDAQ:IRON) due to allegations of misleading business information, aiming to protect investor rights and seek compensation.
- FDA Response Letter Impact: On February 13, 2026, the FDA issued a Complete Response Letter to Disc Medicine, rejecting its new drug application, which caused the company's stock price to plummet 22% on the same day, reflecting market concerns about its product prospects.
- Class Action Preparation: The Rosen Law Firm is preparing a class action lawsuit, allowing investors to participate without any out-of-pocket fees, aiming to recover losses incurred due to the company's misleading information.
- Law Firm Background: The Rosen Law Firm specializes in securities class actions and has recovered over $438 million for investors in 2019 alone, demonstrating its strong capabilities and successful track record in the securities litigation field.
- RALLY-MF Trial Results: In the Phase 2 RALLY-MF trial, over 55% of non-transfusion dependent patients achieved a major hemoglobin response within 12 weeks, demonstrating the efficacy of DISC-0974 in treating myelofibrosis and enhancing the company's competitive edge in hematological disorders.
- Transfusion Independence Improvement: Among transfusion-dependent patients, 64% with low burden and 50% with high burden achieved transfusion independence, indicating significant potential for improving patient quality of life and possibly advancing further clinical development.
- HELIOS Trial Outcomes: The HELIOS extension trial showed sustained reductions in protoporphyrin IX (PPIX) and significant improvements in light tolerance for patients on continuous 60 mg dosing over 2.5 years, indicating long-term safety and efficacy of Bitopertin in treating erythropoietic protoporphyria.
- Upcoming Milestones: Disc expects to engage in end-of-Phase 2 discussions with the FDA for DISC-0974 by year-end and to report Phase 3 APOLLO trial results for Bitopertin in Q4 2026, potentially paving the way for traditional approval and furthering the company's strategic positioning in hematological therapies.
- RALLY-MF Trial Results: In the RALLY-MF trial, DISC-0974 demonstrated significant anemia responses in 61 patients with myelofibrosis (MF) and anemia, with 55% of non-transfusion dependent patients achieving a hemoglobin increase of ≥1.5 g/dL within 12 weeks, indicating the drug's potential to improve patient quality of life.
- HELIOS Trial Update: The HELIOS trial showed that patients treated with bitopertin experienced sustained reductions in PPIX levels and significant improvements in light tolerance, highlighting the long-term safety and efficacy of the drug in treating erythropoietic protoporphyria (EPP).
- Future Development Plans: Disc Medicine plans to share initial data from DISC-3405 in patients with polycythemia vera in Q4 2026, marking the company's ongoing innovation and potential market expansion in hematologic disease treatments.
- Management Conference Call: The company will host a conference call on June 15 to review highlights of the data presented at the EHA meeting and outline next steps, further enhancing investor confidence in the company's future growth.









