Pharma Focus America

Disc Medicine Commences Phase 1/2 Clinical Trial of Bitopertin for Diamond-Blackfan Anemia (DBA) with First Patient Enrollment

Friday, July 28, 2023

Disc Medicine, Inc. (NASDAQ:IRON) has recently commenced a Phase 1/2 clinical trial for bitopertin, an investigational drug targeting Diamond-Blackfan anemia (DBA). Disc Medicine is a clinical-stage biopharmaceutical company with a focus on discovering, developing, and commercializing novel treatments for serious hematologic diseases.

DBA is a severe hematologic disorder, and the company aims to address this condition through the modulation of heme biosynthesis using bitopertin, an orally administered glycine transporter 1 (GlyT1) inhibitor.

The initiation of this trial marks a significant step in exploring the potential of bitopertin to treat a wide range of hematologic conditions, according to Dr. Will Savage, M.D., Ph.D., Chief Medical Officer of Disc Medicine. The company is particularly enthusiastic about investigating the modulation of heme synthesis with bitopertin for DBA, especially after observing positive initial data from an open label trial of bitopertin in erythropoietic protoporphyria (EPP) presented at the European Hematology Association (EHA).

The Phase 1/2 study is designed as a single-arm, dose-escalation trial in DBA patients who have either steroid-refractory and/or relapsed disease or who cannot tolerate systemic corticosteroids. The trial includes planned dose escalation within each participant to continually assess for hematologic response. After the main treatment period, patients may have the opportunity to continue on extended treatment within the trial. The National Heart, Lung, and Blood Institute (NHLBI), a part of the National Institutes of Health (NIH), will conduct and fund the study under a Cooperative Research and Development Agreement (CRADA).

The trial will be overseen by Dr. Cynthia Dunbar, M.D., the NIH Distinguished Investigator, Chief of the Translational Stem Cell Biology Branch, and Head of the Molecular Hematopoiesis Section at NHLBI, with Dr. David Young, M.D., Ph.D., NHLBI Staff Clinician, serving as the Principal Investigator.

The authors emphasize that the responsibility for the research's content lies solely with them and does not necessarily reflect the official views of the National Institutes of Health.

Bitopertin is a clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) under investigation by Disc Medicine, Inc. The drug is designed to modulate heme biosynthesis and targets GlyT1, a membrane transporter expressed on developing red blood cells. Its role is to supply sufficient glycine for heme biosynthesis, supporting erythropoiesis, which is the process of red blood cell production.

Disc Medicine aims to develop bitopertin as a potential treatment for various hematologic diseases, including erythropoietic porphyrias. If successful, it could become the first disease-modifying therapy for this condition. Erythropoietic porphyrias encompass a group of disorders characterized by abnormalities in heme biosynthesis, leading to the accumulation of certain compounds that can be harmful to the body.

The company has initiated two Phase 2 clinical trials of bitopertin in patients with erythropoietic porphyria. The ongoing trials include an open-label study named BEACON and a randomized, double-blind placebo-controlled trial called AURORA.

As of now, bitopertin is an investigational agent and has not been approved for therapeutic use in any jurisdiction worldwide. Disc Medicine acquired global rights to bitopertin through a licensing agreement with Roche in May 2021.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing novel treatments for serious hematologic diseases. Their portfolio consists of innovative therapeutic candidates targeting fundamental biological pathways involved in red blood cell biology, specifically heme biosynthesis and iron homeostasis.

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