Announcement • Jun 02
Disc Medicine Presents Updated Data from RALLY-MF Phase 2 Trial of DISC-0974 in Anemia of Myelofibrosis At Asco Annual Meeting Disc Medicine presented updated data from the RALLY-MF Phase 2 trial of DISC-0974 in anemia of myelofibrosis at the ASCO Annual Meeting in Chicago, IL. 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 myelofibrosis 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 (non-transfusion dependent receiving no transfusions (nTD, n=31), transfusion dependent with low transfusion burden (TD Low, n=11) and transfusion dependent with high transfusion burden (TD High, n=8)). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=25) and not receiving JAK inhibitor therapy (n=25). 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 >75% reduction from baseline and corresponding increases in serum iron. 55% (N=17 of 31) of baseline nTD patients achieved a hemoglobin increase of =1.5 g/dL for =12 weeks (major response) and 68% had an increase of =1 g/dL for =12 weeks (overall response). 64% (N=7 of 11) of TD Low patients achieved transfusion independence (TI, major response) over a 16-week period and 73% achieved a =50% reduction in transfusions (overall response). 50% (N=4 of 8) of TD High patients achieved transfusion independence (TI, major response) over a 12-week period and 88% achieved a =50% reduction in transfusion requirement (overall response). 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 (AE) 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 Fourth Quarter 2026, 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. IRON
Live News • Jun 02
Disc Medicine Advances With Positive Myelofibrosis Data and Expanded Access for Bitopertin Disc Medicine reported positive Phase 2 RALLY-MF trial results for DISC-0974 in myelofibrosis-associated anemia, showing meaningful and durable hemoglobin improvements in 55% of non-transfusion dependent patients and favorable tolerability.
Anemia and transfusion responses to DISC-0974 were seen across all subgroups, including patients on JAK inhibitors, and the company plans an end-of-Phase 2 meeting with the FDA by the end of 2026.
The company launched a U.S. expanded access program for bitopertin, allowing eligible EPP and XLP patients aged 12 and older to access the drug ahead of potential approval. Phase 3 APOLLO topline data are expected in Q4 2026, with a possible FDA decision by mid-2027.
Taken together, Disc Medicine now has encouraging mid-stage data in myelofibrosis-related anemia and a late-stage asset in EPP/XLP moving toward key regulatory milestones. This places considerable focus on upcoming clinical and FDA interactions over the next year.
Investors may wish to monitor how the FDA responds to the DISC-0974 Phase 2 package and whether APOLLO meets its endpoints, since setbacks in either program could materially affect sentiment on the stock. Announcement • Jun 01
Disc Medicine Launches Expanded Access Program for Bitopertin for Epp Patients Disc Medicine, Inc. announced the launch of an expanded access program (EAP) for bitopertin for eligible patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) in the U.S. Bitopertin is being evaluated for the treatment of EPP and XLP in patients age 12 and above in the ongoing double-blind, placebo-controlled Phase 3 APOLLO study. Data from APOLLO is expected in Fourth Quarter 2026, after which a CRL response will be submitted with an FDA decision expected by mid-2027. EAPs are intended to serve as a potential pathway for patients with serious conditions to gain access to an investigational medical treatment outside of clinical trials. The EAP for bitopertin may be available for patients with EPP ages 12 and above who lack satisfactory therapeutic alternatives and meet additional eligibility criteria. 
