A drug from Disc Medicine to treat erythropoietic protoporphyria (EPP) in adults and adolescents has met its primary endpoints in a phase 3 trial.
Bitopertin is an investigational, oral glycine transporter 1 (GlyT1) inhibitor that modulates heme biosynthesis, being developed for EPP, a rare, inherited metabolic disorder that causes severe, painful photosensitivity.
In the APOLLO trial, the drug reduced protoporphyrin IX (PPIX) and increased total time in light. It also demonstrated positive results for secondary endpoints, including by reducing phototoxic reactions and improving multiple measures of light tolerance. Lastly, it led to a statistically-significant quality-of-life improvement and was generally well tolerated.
These results follow a rejection of bitopertin in February, when the FDA issued a CRL for Disc’s NDA to treat patients with EPP, citing a lack of evidence associating percent change in PPIX and sunlight exposure. Disc intends to resubmit the NDA by the end of this year.