On June 12, 2026 Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, reported updated data from multiple clinical programs to be presented at the EHA (Free EHA Whitepaper) Annual Meeting in Stockholm, Sweden. Data from the RALLY-MF trial of DISC-0974 in patients with MF and anemia, to be presented in an oral session today, demonstrate meaningful, durable overall anemia responses across all patient subgroups, regardless of baseline transfusion status or concomitant JAK inhibitor use. Updated data from the HELIOS open-label extension trial of bitopertin in EPP, to be presented in a poster session tomorrow, June 13, show sustained reductions in PPIX, significant improvement in light tolerance measures, and favorable longer-term safety in patients treated with bitopertin.

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"The updates at this year’s EHA (Free EHA Whitepaper) highlight continued progress across our portfolio heading into a catalyst-rich second half of the year," said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. "For DISC-0974 in MF anemia, our Phase 2 dataset continues to strengthen as we prepare for End of Phase 2 discussions with FDA by the end of this year. For bitopertin, continued durability of PPIX reduction and light tolerance improvement in HELIOS is encouraging leading up to the APOLLO Phase 3 readout in Q4, which, as confirmed in a recent Type A meeting with FDA, can serve as the basis for a response to the CRL and could potentially support a traditional approval if successful. We also look forward to sharing initial data from the RESTORE-PV Phase 2 trial of DISC-3405 in polycythemia vera in Q4 this year, setting up the potential for a third program in pivotal-stage development in 2027."

Management will host a call following the EHA (Free EHA Whitepaper) meeting to review highlights of the presented data and next steps for the company on Monday, June 15 at 8:00am EDT. Please register for the event on the Events and Presentations page of Disc’s website (View Source).

Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.

Details of Presentations and Abstracts:

DISC-0974: RALLY-MF Oral Presentation

RALLY-MF, an 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 (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.

Bitopertin: HELIOS update poster

HELIOS is an ongoing Phase 2, open-label, long-term extension trial that enrolled 86 adult and adolescent patients with EPP from the BEACON and AURORA trials. Patients were randomized to receive 20 mg or 60 mg bitopertin in BEACON and 20 mg or 60 mg bitopertin or placebo in AURORA, with all patients transitioning to a 60 mg daily dose of bitopertin in HELIOS.

Longer term treatment with bitopertin was associated with sustained reductions in the disease-causing toxin PPIX, with additional benefit for patients receiving the 60 mg dose continuously
Treatment with bitopertin was associated with sustained, significant improvement in average light tolerance and time to prodrome measures
Bitopertin exhibited a favorable longer-term safety profile with up to 2.5+ years of exposure and similar safety across adults and adolescents with EPP and XLP
DISC-3405: RESTORE-PV Trial-in-Progress poster

RESTORE-PV is a Phase 2 open-label study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of DISC-3405 in patients with polycythemia vera. Initial data from the trial is expected in Q4 2026.

(Press release, Disc Medicine, JUN 12, 2026, View Sourcenews-releases/news-release-details/disc-medicine-presents-positive-clinical-updates-2026-european [SID1234668733])