RARE Daily

Disc Medicine Raises $90 Million to Advance Therapies for Hematologic Diseases

September 2, 2021

Disc Medicine, a clinical-stage biotech developing therapies to treat rare and more prevalent hematologic diseases, raised $90 million in a series B funding round.

OrbiMed led the financing with participation from new investors Arix Bioscience, Janus Henderson Investors, 5AM Ventures, Rock Springs Capital, Nantahala Capital Management, Willett Advisors, and Alexandria Venture Investments. Existing investors Atlas Venture, Novo Holdings and Access Biotechnology also participated in the financing.

 “We have made immense progress this year and have established a strong foundation for growth, including building a portfolio of first-in-class, clinical-stage treatments and assembling a strong leadership team with the capabilities to advance these programs deep into development,” said John Quisel, CEO at Disc Medicine. “This financing will accelerate the next stage of our journey as we enter clinical studies in patients next year.”

The proceeds of the financing will be used to advance the development of Disc’s 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.

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, which was shelved by Roche in 2014 after failing a late-stage trial to treat schizophrenia, has been evaluated in more than 4,000 healthy volunteers and patients in more than 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.

The financing will also support a phase 2 clinical study of DISC-0974, an anti-hemojuvelin monoclonal antibody designed to suppress hepcidin, to treat myelofibrosis patients with transfusion-dependent anemia. 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.

Author: Rare Daily Staff

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