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Disc Medicine's Phase 3 Bitopertin Trial Meets Primary and Secondary Endpoints in EPP

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IRON$79.66 0.92%

Disc Medicine, Inc. (NASDAQ:IRON) reported positive topline results from its pivotal Phase 3 APOLLO trial testing oral bitopertin in adults and adolescents with erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). The 183-patient trial met both co-primary endpoints and all secondary endpoints, demonstrating statistical significance over placebo across all key efficacy measures.

EPP is a rare genetic disorder that causes severe pain and skin reactions upon sunlight exposure due to the accumulation of toxic protoporphyrin IX. Patients taking a once-daily 60 mg dose of bitopertin achieved a 41.4% reduction in blood protoporphyrin IX levels relative to placebo and gained an average of 12.2 additional hours of pain-free time in light during the sixth month. The drug was generally well-tolerated, with dizziness as the most frequent side effect, and no high-grade adverse events reported.

Following the trial outcomes, Disc Medicine plans to resubmit a New Drug Application to the U.S. Food and Drug Administration by the end of 2026. A regulatory decision is anticipated by mid-2027, while detailed study findings will be presented at an upcoming medical conference.

Key points

  • The Phase 3 APOLLO trial of bitopertin met all primary and secondary endpoints in 183 patients with erythropoietic protoporphyria.
  • Bitopertin reduced blood protoporphyrin IX by 41.4% and added 12.2 monthly hours of pain-free light exposure versus placebo.
  • Bitopertin was generally well-tolerated, with dizziness being the most common adverse event and no Grade 3 or higher treatment-emergent side effects.
  • Disc Medicine plans to resubmit a New Drug Application to the U.S. FDA by the end of 2026, with a decision expected by mid-2027.

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Questions and answers

What were the results of Disc Medicine's Phase 3 APOLLO trial?

The Phase 3 APOLLO trial met all primary and secondary endpoints, showing a 41.4% reduction in blood PPIX and 12.2 additional monthly hours of pain-free light exposure for bitopertin compared to placebo.

When does Disc Medicine plan to seek FDA approval for bitopertin?

Disc Medicine plans to resubmit a New Drug Application to the U.S. FDA by the end of 2026, expecting a regulatory decision by mid-2027.

What side effects were observed in the bitopertin study?

Bitopertin was generally well-tolerated. Dizziness was the most common adverse event occurring in 50% of patients, and it was mild or moderate, with no adverse events above Grade 3.