Disc Medicine Reports Positive Results from RESTORE-PV Trial in Polycythemia Vera

Disc Medicine announced positive initial results from its RESTORE-PV Phase 2 trial, showing that the company’s investigational monoclonal antibody, DISC-3405, successfully increased hepcidin and reduced serum iron levels in patients with polycythemia vera (PV). These findings were presented at the 14th Society of Hematologic Oncology (SOHO) Annual Meeting in Houston, Texas.

The RESTORE-PV trial enrolled 40 adult participants with PV, divided into two cohorts: Cohort A and Cohort B. In Cohort A, 20 participants received DISC-3405 subcutaneously at 300 mg every two weeks for 20 weeks, followed by an additional 20 weeks at the same dose. Cohort B participants received the same treatment, but every four weeks. By the time of the data cut, 13 patients in Cohort A had completed 26 weeks of the study, and 18 participants in Cohort B were dosed.

Key results from the trial showed that DISC-3405 effectively reduced phlebotomy events, a common and burdensome treatment for PV patients. Specifically, the mean total phlebotomy events significantly decreased from 4.0 in 26 weeks at baseline to 0.6 in 26 weeks post-Day 1 (p<0.0001). Additionally, 61.5% of participants in Cohort A remained entirely phlebotomy-free post-baseline through 26 weeks. For those who completed the first maintenance period (weeks 12-32), 77.8% were phlebotomy-free during this period.

Moreover, DISC-3405 maintained hematocrit levels at a stable <45% through week 26, leading to an initial improvement in symptom burden among patients. The drug was generally well-tolerated, with adverse events consistent with the underlying disease and a low rate of mild, self-limiting injection site reactions.

Disc Medicine also presented positive results from the Phase 2 RALLY-MF trial of selcodebart (DISC-0974) in patients with anemia of myelofibrosis (MF) and a new systematic literature review characterizing the humanistic burden of anemia associated with MF through patient-reported outcomes.

John Quisel, JD, PhD, President and CEO of Disc Medicine, commented, "The first look at data from the RESTORE-PV trial shows that the established pharmacodynamics of DISC-3405 are translating well on important clinical measures. Iron restriction is proving to be a transformative treatment approach for a large population of patients with polycythemia vera, with the potential to address their crucial need for hematocrit control while managing symptom burden and reducing reliance on phlebotomy."

Disc Medicine plans to provide an update on the RESTORE-PV trial and initial data from the Phase 1b trial of DISC-3405 in sickle cell disease by the end of 2026. The company also expects to share feedback from an end-of-phase 2 meeting with the U.S. Food and Drug Administration (FDA) and plans for pivotal development in anemia of MF by the end of the year.

Polycythemia vera (PV) is a chronic and rare myeloproliferative neoplasm characterized by the abnormal proliferation of red blood cells. PV affects approximately 150,000 patients in the U.S. and has a similar prevalence in Europe. Current therapy for PV involves phlebotomy to reduce blood volume and iron to limit erythropoiesis, or treatment with cytoreductive agents, with the goal of reducing red blood cell count and managing symptoms.

Disc Medicine, a clinical-stage biopharmaceutical company, is committed to discovering, developing, and commercializing novel treatments for serious hematologic diseases. The company aims to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis.