Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company, has announced positive additional data for bitopertin in erythropoietic protoporphyria (EPP), including analyses from the AURORA study that demonstrated clinical activity of bitopertin. The study highlighted meaningful improvements in light tolerance, phototoxic reactions, and quality of life. Additionally, updated data from a Phase 1b trial of DISC-0974 in MF anemia showed durable increases in hemoglobin and reduced transfusion burden in a majority of evaluable patients. Initial single-ascending dose (SAD) data from a Phase 1 healthy volunteer trial of DISC-3405 provided proof of mechanism, showing potential for deep and sustained increases in hepcidin and reductions in iron.
Bitopertin in EPP
The AURORA study, a randomized, double-blind, placebo-controlled Phase 2 clinical trial, enrolled 75 adult subjects with EPP. Key findings include significant reductions in protoporphyrin IX (PPIX), meaningful improvements in key aspects of EPP, and substantial, dose-dependent reductions in the rate of phototoxic reactions compared to placebo. Bitopertin was generally well-tolerated with no reported serious adverse events (SAEs) to date.
DISC-0974 in MF
The Phase 1b/2a multi-center, open-label, ascending-dose clinical trial of DISC-0974 is enrolling patients with MF and severe anemia. Updated data from 34 patients showed substantial and sustained reductions in hepcidin levels and increases in iron, with strong hematologic response observed across all patient types at 28-100 mg doses. DISC-0974 was generally well-tolerated at all evaluated dose levels.
DISC-3405
Initial data from the SAD portion of the Phase 1 clinical trial of DISC-3405 in healthy volunteers showed a meaningful dose-dependent increase in hepcidin and corresponding reduction in serum iron across all dose levels. DISC-3405 was generally well-tolerated with no SAEs, AEs higher than Grade 2, or AEs leading to trial withdrawal reported to date.
The data were presented at the European Hematology Association (EHA) 2024 Congress, showcasing the activity and therapeutic potential of each of Disc Medicine's programs. Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.
