Disc Medicine, Inc. presented updated clinical data from multiple programs at the 2026 European Hematology Association Annual Meeting in Stockholm, Sweden, highlighting progress in its treatments for serious hematologic diseases. The RALLY-MF Phase 2 trial of DISC-0974 in patients with myelofibrosis and anemia demonstrated meaningful, durable overall anemia responses across all patient subgroups, regardless of baseline transfusion status or concomitant JAK inhibitor use. The HELIOS open-label extension trial of bitopertin in erythropoietic protoporphyria showed sustained reductions in protoporphyrin IX, significant improvement in light tolerance measures, and favorable longer-term safety.
RALLY-MF Trial Results
The RALLY-MF trial enrolled 61 adult patients with myelofibrosis and anemia as of the April 27 data cutoff, with 50 patients included in the responder analysis. The trial included non-transfusion dependent patients (n=31), transfusion dependent patients with low burden (n=11), and transfusion dependent patients with high burden (n=8). DISC-0974 was administered subcutaneously at 50 mg every 4 weeks for up to 6 treatments.
| Patient Subgroup |
Major Response |
Overall Response |
| Non-transfusion dependent (n=31) |
55% (N=17) achieved hemoglobin increase ≥1.5 g/dL for ≥12 weeks |
68% achieved hemoglobin increase ≥1 g/dL for ≥12 weeks |
| Transfusion dependent low (n=11) |
64% (N=7) achieved transfusion independence over 16 weeks |
73% achieved ≥50% reduction in transfusions |
| Transfusion dependent high (n=8) |
50% (N=4) achieved transfusion independence over 12 weeks |
88% achieved ≥50% reduction in transfusion requirement |
Patients receiving concomitant JAK inhibitor therapy (n=25) showed a 56% major hematologic response rate and 72% overall response rate, with similar results across different JAK inhibitors. DISC-0974 was generally well-tolerated, with diarrhea being the only adverse event reported in two or more subjects.
HELIOS Trial Update
The HELIOS trial enrolled 86 adult and adolescent patients with erythropoietic protoporphyria from previous BEACON and AURORA trials, all transitioning to a 60 mg daily dose of bitopertin. Longer-term treatment with bitopertin was associated with sustained reductions in protoporphyrin IX, with additional benefits for patients receiving the 60 mg dose continuously. Treatment also led to 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.
RESTORE-PV Trial Update
Disc Medicine also provided an update on the RESTORE-PV Phase 2 trial of DISC-3405 in patients with polycythemia vera. The trial is an open-label study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of DISC-3405. Initial data from the trial is expected in Q4 2026.
Management will host a conference call on Monday, June 15 at 8:00 am ET to review the presented data and discuss next steps for the company. Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.
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