9 June 2023
New research with initial positive results has been published by Disc Medicine on Bitopertin, a potential treatment for Erythropoietic Protoporphyria (EPP).
Disc Medicines presented the preliminary results of their ongoing BEACON Phase II open-label trial at the European Hematology Association Conference in Frankfurt on 9 June 2023. The trial is evaluating bitopertin, an orally administered glycine transporter 1 (GlyT1) inhibitor, in patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP).
Highlights of the initial data presented were:
- Consistent and dose-dependent reductions of protoporphyrin IX (PPIX), the disease-causing metabolite in EPP, were observed in patients treated with bitopertin
- Patients reported significant improvements in sunlight tolerance and measures of quality-of-life
- Bitopertin was well-tolerated, with no meaningful changes in hemoglobin observed