Key insights
- Disc Medicine (IRON) shares surged 60% following overwhelmingly positive Phase 3 trial results for its EPP treatment, bitopertin. The trial met all primary and secondary endpoints, showing significant reductions in toxic buildup, increased pain-free light exposure, fewer reactions, and improved quality of life. The drug was generally well-tolerated. Disc Medicine plans to resubmit its NDA to the FDA by the end of 2026, anticipating a decision by mid-2027.
Investing.com -- Shares of Disc Medicine, Inc. (NASDAQ: IRON) jumped 60% after the company announced overwhelmingly positive topline results from its Phase 3 APOLLO trial. The trial evaluated bitopertin, an investigational treatment for adults and adolescents with erythropoietic protoporphyria (EPP).
The randomized, double-blind trial of 183 global patients met every primary and secondary endpoint, demonstrating statistically significant improvements across all study measures compared to a placebo.
Key Trial Highlights:
Reduced toxic buildup: Patients saw a 41.4% reduction in whole-blood metal-free protoporphyrin IX (the compound that causes symptoms).
More pain-free light exposure: Patients experienced a 12.2-hour increase in their average monthly total time spent in light without pain by the sixth month.
Fewer reactions: The drug significantly reduced phototoxic reactions over the six-month treatment period.
Improved quality of life: Exploratory measures, including Patient Global Impression of Change scores, showed statistically significant improvements.
Safety Profile Bitopertin was generally well-tolerated. The most common side effect was dizziness, reported in 50% of the treatment group. These cases were characterized as mild or moderate and were generally transient. No adverse events above Grade 3 were reported during the trial.
Armed with this data, Disc Medicine plans to resubmit a New Drug Application (NDA) to the FDA by the end of 2026. The company anticipates a regulatory decision by mid-2027.
Original Article