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Biotechnology: Pharmaceutical Preparations
Clinical TrialJun 12, 2026, 08:31 AM

Disc Medicine DISC-0974 & bitopertin trials show positive data

AI Summary

Disc Medicine announced updated positive data from multiple clinical programs at the EHA Annual Meeting. The RALLY-MF trial of DISC-0974 in myelofibrosis and anemia demonstrated meaningful and durable anemia responses across all patient subgroups, regardless of baseline transfusion status or JAK inhibitor use. Additionally, updated data from the HELIOS open-label extension trial of bitopertin in erythropoietic protoporphyria showed sustained reductions in PPIX, significant improvement in light tolerance, and a favorable longer-term safety profile. The company anticipates a catalyst-rich second half of the year, including End of Phase 2 discussions for DISC-0974 and Phase 3 readout for bitopertin.

Key Highlights

  • DISC-0974 RALLY-MF trial showed 55% of nTD patients achieved ">1.5 g/dL hemoglobin increase for ">12 weeks.
  • 64% of TD Low patients achieved transfusion independence over 16 weeks with DISC-0974.
  • 50% of TD High patients achieved transfusion independence over 12 weeks with DISC-0974.
  • Bitopertin HELIOS trial demonstrated sustained reductions in PPIX and significant improvement in light tolerance.
  • Bitopertin showed a favorable longer-term safety profile with up to 2.5+ years of exposure.
  • Management to host a corporate update conference call on Monday, June 15, at 8:00 am ET.
  • Initial data from RESTORE-PV Phase 2 trial of DISC-3405 in polycythemia vera expected in Q4 2026.