Disc Medicine reports positive Phase 3 results for bitopertin in erythropoietic protoporphyria, setting stage for FDA resubmission

Disc Medicine's bitopertin, being developed for erythropoietic protoporphyria (EPP), a rare blood disorder that causes painful light sensitivity, had its accelerated FDA application rejected in February.

In the Phase 3 APOLLO trial, the drug significantly lowered the disease biomarker protoporphyrin IX and increased the amount of time patients could spend in sunlight or artificial light.

The trial met all primary and secondary endpoints, and the company’s CEO John Quisel expressed enthusiasm about the clean results.

Analysts had anticipated a positive readout, noting the drug’s ability to reduce PPIX could improve patient quality of life and may pave the way for a new FDA submission.