Disc Medicine is a clinical-stage biopharmaceutical company focused on developing novel treatments for patients suffering from serious hematologic diseases. The company's innovative approach targets fundamental pathways of red blood cell biology, aiming to restore red blood cell production in inherited and acquired anemias.
Disc Medicine's lead investigational product, bitopertin, is an orally administered inhibitor of glycine transporter one (GlyT1) designed to modulate heme biosynthesis. By inhibiting GlyT1, bitopertin is intended to reduce the accumulation of protoporphyrin IX (PPIX), a toxic metabolite that causes disease pathology in conditions like erythropoietic protoporphyria (EPP). Bitopertin is currently being evaluated in two ongoing Phase 2 studies for the treatment of EPP.
Another key candidate in Disc Medicine's pipeline is DISC-0974, an antibody targeting hemojuvelin, a protein involved in iron regulation. By downregulating hemojuvelin, DISC-0974 aims to release iron from the body's stores, thereby refreshing red blood cell production. DISC-0974 is being investigated as a potential treatment for severe anemia associated with myelofibrosis and other inflammatory conditions.
In December 2022, Disc Medicine received Orphan Drug Designation from the US FDA for bitopertin for the treatment of EPP, highlighting the drug's potential in addressing this rare disease. The company also initiated a Phase 1 clinical trial for DISC-3405, an anti-TMPRSS6 antibody licensed from Mabwell Therapeutics, to treat the rare blood cancer polycythemia vera in October 2023.
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