Disc Medicine, Inc. (NASDAQ: IRON) announced initial results from the RESTORE-PV Phase 2 clinical trial of its investigational drug DISC-3405 on September 9, 2026. The data was presented at the 14th Society of Hematologic Oncology (SOHO) annual meeting in Houston, Texas.
The RESTORE-PV trial is a multi-center, open-label study enrolling 40 adult participants with polycythemia vera (PV). The study design includes a 4- to 12-week observation period, followed by a 12-week dose escalation and a maintenance period. Participants in Cohort A received DISC-3405 subcutaneously at 300 mg every two weeks, while those in Cohort B received the same dose every four weeks.
At the time of the data cut, all 20 participants in Cohort A had been dosed, with 13 patients completing 26 weeks of the study. Efficacy results were presented for Cohort A, while safety data was reported for both cohorts.
The company reported that DISC-3405 demonstrated dose-proportional pharmacokinetics and increased hepcidin levels while reducing serum iron. The treatment resulted in a controlled mean hematocrit maintained below 45% through week 26. The data indicated a significant reduction in phlebotomy events. For the 13 patients who completed 26 weeks, the mean total phlebotomy events decreased from 4.0 at baseline to 0.6 post-treatment (p<0.0001). Additionally, 61.5% of participants remained entirely phlebotomy-free through week 26, and 77.8% of those who completed the first maintenance period remained phlebotomy-free.
Disc Medicine stated that DISC-3405 was generally well-tolerated. Adverse events were consistent with the underlying disease, and injection site reactions were reported at a low rate, described as mild and self-limited.
The company plans to provide an update on RESTORE-PV and initial data from the Phase 1b trial of DISC-3405 in sickle cell disease by the end of 2026.
