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Disc Medicine Announces First Patient Enrolled in Phase 1/2 Clinical Trial of Bitopertin in Diamond-Blackfan Anemia (DBA)
WATERTOWN, Mass., July 27, 2023 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery,
About this update from Disc Medicine, Inc.
[{"type":"text","content":"WATERTOWN, Mass., July 27, 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 that the first patient has been enrolled in the National Institutes of Health-sponsored Phase 1/2 clinical trial of bitopertin in Diamond-Blackfan anemia (DBA). Bitopertin is an investigational, orally administered glycine transporter 1 (GlyT1) inhibitor designed to modulate heme biosynthesis. “The start of this trial represents another step toward demonstrating the potential of bitopertin to address a wide range of hematologic conditions.” said Will Savage, M.D., Ph.D., Chief Medical Officer of Disc. “We are excited to test the potential of treating DBA by modulating heme synthesis with bitopertin, particularly in light of our recent positive initial data from an open label trial of bitopertin in erythropoietic protoporphyria (EPP) at EHA. We appreciate the support and leadership of the National Heart, Lung, and Blood Institute and look forward to continued collaboration on this trial.” The Phase 1/2 study will be a 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. This study will be conducted and funded by the NIH under a Cooperative Research and Development Agreement (CRADA), 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, with Dr. David Young, M.D., Ph.D., NHLBI Staff Clinician as Principal Investigator. The content of this research is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. About Diamond-Blackfan Anemia Diamond-Blackfan Anemia (DBA) is a rare, inherited blood disorder characterized by the failure of bone marrow to produce red blood cells. The incidence of DBA is approximately 1:...