Disc Medicine reported positive top-line results from its Phase 3 APOLLO trial of bitopertin, an investigational treatment for erythropoietic protoporphyria (EPP), a rare inherited disorder that can cause severe pain and skin reactions after light exposure.
The 183-person trial met both primary endpoints:
compared with placebo, bitopertin reduced blood levels of metal-free protoporphyrin IX (PPIX) by 41.4% and gave patients an average of 12.2 additional hours per month of pain-free time in light during month six.
The company said all secondary endpoints were also met, including fewer painful phototoxic reactions. Bitopertin was generally well tolerated, with no new safety concerns reported.
The FDA had issued a Complete Response Letter in February, agreeing that bitopertin lowered PPIX but saying earlier studies had not established that this translated into improved light tolerance. The APOLLO results directly address that concern.
Disc plans to resubmit its application by the end of 2026 and expects an FDA decision around mid-2027. The drug is not yet approved.
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