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Disc Medicine Announces Collaboration with National Institutes of Health for Phase 2 Clinical Study of Bitopertin in Patients with Diamond-Blackfan Anemia (DBA)
IND accepted for phase 2 clinical trial in DBA patients; study is expected to initiate mid-year 2023 WATERTOWN, Mass., March 21, 2023 (GLOBE NEWSWIRE) -- Disc
About this update from Disc Medicine, Inc.
[{"type":"text","content":"IND accepted for phase 2 clinical trial in DBA patients; study is expected to initiate mid-year 2023\nWATERTOWN, Mass., March 21, 2023 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, announced today a collaboration with the National Heart Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) to evaluate bitopertin, a therapeutic candidate designed to modulate heme biosynthesis, in a phase 2 clinical study of patients with Diamond-Blackfan anemia (DBA). The study will be conducted and funded by the NIH under a Cooperative Research and Development Agreement (CRADA) and is expected to initiate mid-year 2023. “We’re thrilled to have the support of the NHLBI for this clinical trial as there is growing evidence to suggest that reducing excess levels of heme may be an effective treatment strategy for DBA. This collaboration will enable us to benefit from the experience of the NHLBI in designing and conducting clinical studies of DBA.” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “Disc now has ongoing development programs of bitopertin in both erythropoietic porphyria and DBA. We believe controlling heme synthesis has the potential to address a wide range of hematologic conditions and are planning studies in additional indications.” Under the CRADA, the NHLBI will serve as the regulatory sponsor and be responsible for conducting a phase 2 clinical study of bitopertin in DBA patients. The study will be jointly funded by the NHLBI and Disc. The study will be under the direction of Dr. Cynthia Dunbar, M.D., the NIH Distinguished Investigator and Chief Translational Stem Cell Biology Branch, and Head, Molecular Hematopoiesis Section, NHLBI. The phase 2 study will be a pilot, single-arm, dose-escalation trial of bitopertin in DBA patients who either have steroid-refractory and/or relapsed disease or are unable to tolerate systemic corticosteroids. The study includes planned dose escalation within each participant to continually assess for hematologic response. Upon completion of the main treatment period, patients may continue on extended treatment within the trial. About Diamond-Blackfan Anemia Diamon...