Disc Medicine, Inc.

We are a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing new treatments for patients with serious blood diseases. We have a portfolio of product candidates in both clinical and preclinical stages. These candidates aim to change fundamental biological processes related to how red blood cells form and function, specifically heme biosynthesis and iron homeostasis. Our current pipeline includes bitopertin, which is being developed for erythropoietic porphyrias (EPs), including erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). Also in our pipeline is DISC-0974 for anemia of myelofibrosis (MF) and anemia of inflammatory bowel disease (IBD), and DISC-3405 for polycythemia vera (PV), sickle cell disease (SCD), and other blood disorders. Our preclinical programs include DISC-0998 for anemia associated with inflammatory diseases. Our development strategy uses molecular mechanisms that are well-understood and have been confirmed in humans. Bitopertin is the main product candidate in our heme biosynthesis modulation portfolio. F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (collectively, Roche) previously evaluated bitopertin in a clinical program involving over 4,000 individuals for other conditions. This program showed that bitopertin acts as a glycine transporter 1 (GlyT1) inhibitor and affects heme biosynthesis. We are initially developing bitopertin for EPs, including EPP and XLP. These are severe diseases called porphyrias, caused by flaws in the heme biosynthesis pathway, leading to a buildup of toxic metabolites called porphyrins. This results in skin hypersensitivity to sunlight and certain artificial light. We have completed two Phase 2 clinical trials for bitopertin: BEACON, an open-label, parallel-dose trial in EPP and XLP patients conducted in Australia, and AURORA, a randomized, double-blind, placebo-controlled trial in EPP patients conducted in the United States.