Disc Medicine Inc. presented preliminary findings from its ongoing, Phase 2 open-label BEACON trial evaluating bitopertin, an orally administered glycine transporter 1 (GlyT1) inhibitor, in patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) at the European Hematology Association (EHA) 2023 Congress in Frankfurt, Germany. The initial trial data demonstrated consistent decreases in PPIX, significant increases in reported sunlight tolerance and improvements in measures of patient quality of life. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy.

There are currently two ongoing Phase 2 clinical trials of bitopertin in patients with erythropiietic porphyria, including an open-label trial called BEACON and a randomized, double-blind placebo-controlled trial called AURORA. Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. There is currently no cure for EPP and only one FDA-approved therapy, a surgically implanted synthetic hormone designed to stimulate melanin production called Scenesse(R) (afamelanotide).