Disc Medicine announces feedback from FDA End of Phase 2 meeting for bitopertin

Disc Medicine (IRON) announced feedback from its end-of-Phase 2 meeting with the U.S. Food and Drug Administration, supporting the regulatory path forward for bitopertin in EPP. The meeting resulted in agreement on all proposed attributes of the APOLLO study, which the company plans to initiate by mid-2025, including the: Sufficiency of a single, randomized, double-blind, placebo-controlled trial; Primary endpoint of average monthly total time in sunlight without pain during the last month following 6 months of treatment; Additional measures such as change in PPIX, occurrence of phototoxic reactions, cumulative total pain-free time in sunlight, and patient global impression of change; Selection of 60 mg dose of bitopertin and 6-month treatment duration; and Inclusion of patients aged 12+ with EPP, including X-linked protoporphyria. In addition, the FDA also agreed with the potential for reduction of PPIX to serve as a surrogate endpoint to support an accelerated approval. Under this pathway, Disc would have the potential to submit an NDA based on the existing data package and the APOLLO trial would serve as a confirmatory trial. Disc will be meeting with the FDA to finalize the details of APOLLO and plans to provide an update in Q1 2025 on this discussion as well as timing for NDA filing under an accelerated pathway.