On Monday, Disc Medicine Inc ( IRON ) released topline data from the AURORA Phase 2 study of bitopertin in patients with Erythropoietic Protoporphyria (EPP).
Erythropoietic protoporphyria is a rare condition in which a toxic substance, protoporphyrin IX (PPIX), builds up in the blood. Patients experience severe reactions in the sunlight, including pain, swelling, burning sensations, and blistering.
Treatment with bitopertin resulted in statistically significant reductions in PPIX, the primary endpoint, and significant improvements in the rate of phototoxic reactions with pain and the Patient Global Impression of Change (PGIC).
Bitopertin resulted in significant, dose-dependent, and sustained reductions in whole blood PPIX levels: -21.6% for 20 mg (p=0.003 vs. placebo) and -40.7% for 60 mg (p