Disc Medicine, Inc.

Disc Medicine Announces Success on All Primary and Secondary Endpoints in the Pivotal Phase 3 APOLLO Trial of Bitopertin in Adults and Adolescents with Erythropoietic Protoporphyria (EPP)

Disc Medicine's pivotal Phase 3 APOLLO trial of bitopertin in erythropoietic protoporphyria met all primary and secondary endpoints with statistical significance. Bitopertin achieved a -41.4% reduction in whole-blood PPIX vs placebo (p<0.0001) and a +12.2 hour increase in monthly time in light without pain (p=0.0010), with no new safety signals and no TEAEs above Grade 3 in the 183-patient study. The company plans to resubmit an NDA to the FDA by end of 2026, with a decision anticipated by mid-2027; bitopertin has potential to be the first disease-modifying therapy for EPP.

•6d ago

Disc Medicine Presents Initial Results from RESTORE-PV Phase 2 Trial in Patients with Polycythemia Vera (PV) at the 14th Society of Hematologic Oncology (SOHO) Annual Meeting

Disc Medicine presented initial Phase 2 RESTORE-PV data at the SOHO annual meeting showing its anti-TMPRSS6 antibody DISC-3405 sharply reduced phlebotomy dependence in polycythemia vera: mean phlebotomy events fell from 4.0 to 0.6 per 26 weeks (p<0.0001), with 61.5% of patients entirely phlebotomy-free through week 26. Hematocrit was maintained stably below 45% through week 26 in Cohort A alongside dose-proportional PK, hepcidin elevation, and serum iron suppression, and the drug was generally well-tolerated with mild, self-limited injection site reactions. Disc now has two myeloproliferative programs advancing toward potential pivotal development — DISC-3405 in PV and selcodebart (DISC-0974) in anemia of myelofibrosis — with a RESTORE-PV update, Phase 1b sickle cell data, and FDA end-of-Phase-2 meeting feedback on selcodebart all expected by year-end 2026.

•1mo ago

Disc Medicine Announces Multiple Presentations Across Portfolio at the Society of Hematologic Oncology (SOHO) 2026 Annual Meeting

Disc Medicine announced it will present initial clinical data from its Phase 2 RESTORE-PV trial of DISC-3405 in polycythemia vera at the SOHO Annual Meeting in September. The oral presentation will cover baseline and safety data for 40 patients from Cohorts A and B, along with pharmacokinetic and pharmacodynamic endpoints for 20 patients from Cohort A. The company will also present updated efficacy data for its DISC-0974 program in myelofibrosis through both an oral presentation and poster sessions.

•1mo ago

Disc Medicine Reports Second Quarter 2026 Financial Results and Provides Business Update

Disc Medicine reported a Q2 net loss of $59.5 million, or $(1.54) per share, with R&D expenses of $46.9 million, as the company advances its pipeline of hematology therapeutics. The company ended the quarter with $717.7 million in cash, providing a runway into 2029, and confirmed that the Phase 3 APOLLO study for bitopertin can serve as the basis for a CRL response, with topline data expected in Q4. Pipeline updates included full enrollment in the RESTORE-PV study for DISC-3405 with data due in Q3, and Phase 2 data for selcodebart showing a 72% overall anemia response rate in myelofibrosis.

•2mo ago

Disc Medicine Presents Positive Clinical Updates at the 2026 European Hematology Association (EHA) Annual Meeting

Disc Medicine presented positive Phase 2 data for DISC-0974 in myelofibrosis anemia at the EHA Annual Meeting, showing durable anemia responses across all patient subgroups. The RALLY-MF trial data showed 55% of non-transfusion dependent patients achieved major hemoglobin response, while 64% of low-burden transfusion dependent patients achieved transfusion independence. Updated data for bitopertin in erythropoietic protoporphyria showed sustained PPIX reductions and favorable long-term safety, supporting the upcoming APOLLO Phase 3 readout in Q4.

•4mo ago