Disc Medicine, a Cambridge MA-based biotechnology company, raised $90M in Series B funding.
The round was led by OrbiMed with participation from new investors Arix Bioscience, Janus Henderson Investors, 5AM Ventures, Rock Springs Capital, Nantahala Capital Management, Willett Advisors, and Alexandria Venture Investments and existing investors Atlas Venture, Novo Holdings and Access Biotechnology. In conjunction with the funding, Mona Ashiya, PhD, Partner at OrbiMed, and Mark Chin, MS, MBA, Managing Director at Arix Bioscience, joined Disc’s Board.
Led by John Quisel, JD, PhD, Chief Executive Officer, Disc Medicine provides a clinical-stage biotechnology programs dedicated to the discovery and development of novel therapies for the treatment of serious and debilitating hematologic diseases.
The company intends to use the funds to develop its hematology pipeline of therapeutic candidates. This includes conducting a phase 2 study of its lead program bitopertin, an oral, clinical-stage GlyT1 inhibitor with potential to become the first disease-modifying treatment for erythropoietic porphyrias (EP), a family of rare and debilitating genetic disorders caused by dysregulated heme synthesis. It will also support a phase 2 clinical study of DISC-0974, an anti-hemojuvelin (HJV) monoclonal antibody designed to suppress hepcidin, to treat myelofibrosis patients with transfusion-dependent anemia. DISC-0974 is currently being studied in a phase 1 clinical trial of healthy volunteers, and the company expects to report data later this year. The company intends to expand clinical development of both programs and plans to initiate clinical studies in additional hematologic disorders.
Bitopertin is a clinical-stage, orally administered small molecule inhibitor of glycine transporter 1 (GlyT1). Glycine is an essential precursor for heme biosynthesis and GlyT1 is required to maintain adequate levels of intracellular glycine in developing erythrocytes. As a modulator of heme synthesis, bitopertin has the potential to provide benefit for a range of disorders caused by imbalances in the production of heme and its pathway intermediates. Bitopertin has been evaluated in over 4,000 healthy volunteers and patients in over 30 clinical trials across multiple indications, including several phase 2 and 3 trials in psychiatric disorders and in a rare blood cell disorder, and has a well-defined safety profile. Although CNS efficacy was not established in these studies, bitopertin demonstrated marked effects on heme synthesis. Moreover, in preclinical studies conducted by Disc Medicine, inhibition of GlyT1 by bitopertin was shown to decrease levels of the metabolites that are the underlying cause of EP. Bitopertin is an experimental agent and is not approved for use as a therapy in any jurisdiction worldwide.
DISC-0974 is an investigational, first-in-class monoclonal antibody designed to suppress hepcidin production by inhibiting the hemojuvelin (HJV) co-receptor, a highly selective and critical target of the hepcidin pathway. Hepcidin is the primary regulatory hormone of iron homeostasis and plays a central role by restricting iron absorption and preventing deployment from internal iron stores. DISC-0974 is being developed as a potential treatment for anemia of inflammation by suppressing hepcidin and enhancing iron availability for erythropoiesis. Preclinical studies of DISC-0974 and human genetic evidence validate that inhibition of HJV results in potent suppression of hepcidin and increased serum iron. Disc obtained global rights to DISC-0974 and related molecules under a license agreement from AbbVie in October 2019. DISC-0974 is an experimental agent and is not approved for use as a therapy in any jurisdiction worldwide.