Bitopertin updates

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

John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc Medicine:

“We’re delighted to share these initial, positive data from BEACON, which provide the first clinical evidence supporting our therapeutic hypothesis of bitopertin in EPP. Over the next 12 months, we plan to build on this momentum with a series of additional clinical read-outs across our portfolio. This is an important moment for Disc as a company, and I want to extend my gratitude to our team, collaborators, and most importantly, the patients and families participating in BEACON.”

Will Savage, M.D., Ph.D., Chief Medical Officer at Disc Medicine:

“We are excited to share these initial data from the BEACON trial, where we observed consistent and sustained suppression of PPIX, the disease-causing metabolite in EPP, in patients treated with bitopertin. Importantly, this reduction translated into significant improvements in the time that patients can spend in sunlight without reporting pain or symptoms related to their disease. We’re encouraged by the data and plan to present additional data at the end of the year.”

Read the full press release on the Disc Medicine website, and view the data presented at the European Hematology Association Conference in this PowerPoint presentation.