Disc Medicine Inc. (NASDAQ: IRON) presented positive phase 2 trial results for DISC-3405, an investigational anti-TMPRSS6 monoclonal antibody, at the Society of Hematologic Oncology annual meeting. The trial, known as RESTORE-PV, enrolled 40 adult participants with polycythemia vera across two cohorts. Cohort A received 300 mg of DISC-3405 subcutaneously every two weeks, while Cohort B received the same dose every four weeks. At the data cutoff, all 20 participants in Cohort A were dosed, and 13 of them completed 26 weeks of the study.
The results showed a significant reduction in phlebotomy events. For the 13 Cohort A participants who completed 26 weeks, the mean total phlebotomy events decreased from 4.0 events in 26 weeks at baseline to 0.6 events in 26 weeks post-Day 1. Additionally, 61.5% of participants remained phlebotomy-free through 26 weeks, and 77.8% of participants who completed the first maintenance period remained phlebotomy-free during that time. Treatment maintained mean hematocrit levels below 45% through week 26. The drug also increased hepcidin and lowered serum iron. Safety data indicated that DISC-3405 was generally well-tolerated, with adverse events consistent with the underlying disease and a low rate of mild, self-limited injection site reactions.
Disc Medicine in-licensed the drug from Mabwell Therapeutics in January 2023. The company plans to provide updates on the RESTORE-PV trial by year-end and share initial data from a Phase 1b trial in sickle cell disease.
