Disc Medicine 的 bitopertin III期APLLO试验在EPP中达到全部主要及次要终点，拟2026年底重新递交NDA

Disc Medicine, Inc. (IRON) reported positive topline results from the pivotal Phase 3 APOLLO trial of bitopertin in adults and adolescents with erythropoietic protoporphyria (EPP), with the investigational therapy meeting all primary and secondary endpoints.
The results support the company's plan to resubmit a New Drug Application (NDA) to the U.S. FDA by the end of 2026.
Erythropoietic protoporphyria (EPP) is a rare inherited disorder caused by the buildup of protoporphyria IX (PPIX), a substance that can trigger severe pain and skin reactions after exposure to sunlight. The condition can significantly limit outdoor activities and daily life.
The APOLLO study enrolled 183 patients aged 12 years and older with EPP or X-lined protoporphyria (XLP) across the United States, Canada, Europe and Australia. Participants were randomized to receive either bitopertin or placebo once daily for six months.
Bitopertin achieved statistical significance on both co-primary endpoints. Patients receiving the drug experienced a 41.4% reduction in whole-blood metal-free PPIX levels compared with placebo p<0.0001). The treatment also increased average monthly time spent in light without pain by 12.2 hours versus placebo (p<0.0001). The treatment also increased average monthly time spent in light without pain by 12.2 hours versus placebo during the sixth month of treatment (p=0.0010).
The trial also met all secondary endpoints. According to the company, patients treated with bitopertin experienced fewer phototoxic reactions during the six-month study period, greater cumulative time spent in light without pain and longer daily sunlight exposure before the onset of symptoms compared with placebo.
In addition, exploratory quality-of-life measures showed statistically significant improvements, including better Patient Global Impression of Change (PGIC) and Patient Global Impression of Severity (PGIS) scores.
The positive Phase 3 results follow a Complete Response Letter (CRL) issued by the U.S. FDA earlier this year for Disc Medicine's previous application seeking approval of bitopertin in EPP. At the time, the FDA acknowledged the drug's ability to reduce PPIX levels but said the available data did not adequately establish a relationship between PPIX reduction and clinical benefit. The agency indicated the results from the Phase 3 APOLLO study could support a future regulatory submission.
Disc Medicine said bitopertin was generally well tolerated and no new safety signals were identified. Dizziness was the most commonly reported treatment-emerged adverse event in the treatment group and was typically mild to moderate and transient. The company reported no adverse events above Grade 3 severity.
Bitopertin is an investigational oral inhibitor of glycine transporter 1 (GlyT1) that is designed to reduce the production of PPIX by modulating heme biosynthesis. The drug has previously been evaluated in the Phase 2 BEACON and AURORA studies, the HELIOS extension study and the Phase 3 APOLLO trial.
Following the APOLLO results, Disc Medicine plans to resubmit the NDA to the FDA by year-end 2026, with a regulatory decision anticipated by mid-2027.
IRON has traded between $40.00 and $99.50 over the past year. The stock closed Friday's trading (October 16, 2026) at $56.84, up 0.34%. In pre-market trading the stock is at $83.16, up 45.19%.
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October 16, 2026 16:06 ET Monthly consumer price inflation data and retail sales figures were the main news from the U.S. economy this week. In Europe, monthly GDP figures from the U.K. gained attention. External trade data from China was in focus in Asia. Elsewhere, the International Monetary Fund released its latest projections for the global economy.