Disc Medicine's Bitopertin Meets All Endpoints in Pivotal Phase 3 EPP Trial

Disc Medicine, Inc. announced positive topline results from its pivotal Phase 3 APOLLO trial of bitopertin for treating erythropoietic protoporphyria (EPP). The study successfully met all of its co-primary and secondary endpoints, demonstrating statistically significant and clinically meaningful benefits.

Key Details

• Primary Endpoints: The trial achieved both co-primary endpoints, showing a 41.4% reduction in protoporphyrin IX (PPIX) versus placebo (p<0.0001) and a 12.2-hour increase in average monthly time in light without pain versus placebo (p=0.0010).
• Secondary Endpoints & Safety: The drug also met all secondary endpoints, including a significant reduction in the rate of phototoxic reactions (p<0.0001). Bitopertin was generally well-tolerated, with dizziness being the most common adverse event.
• Regulatory Timeline: Based on these results, the company plans to resubmit a New Drug Application (NDA) to the U.S. FDA by the end of 2026, with a potential decision anticipated by mid-2027.