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Disc Medicine collaborates with NIH for phase 2 study of bitopertin in patients with Diamond-Blackfan anaemia

Watertown, Massachusetts
Thursday, March 23, 2023, 18:00 Hrs  [IST]

Disc Medicine, Inc., a clinical-stage biopharmaceutical company, announced 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 anaemia (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.

Diamond-Blackfan Anaemia (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 1:100,000 to 1:200,000 live births every year. Patients are usually diagnosed during infancy and commonly present with severe anaemia, pallor, fatigue, as well as other potential abnormalities. DBA is chronic and requires lifelong management with corticosteroids and blood transfusions, which are associated with serious toxicities and long-term complications. Evidence suggests that the anaemia of DBA may be caused by the accumulation of excess heme in developing red blood cells, which is toxic and leads to their premature death. Preclinical studies have shown that targeting elevated heme levels has the potential as a therapeutic strategy for DBA.

Bitopertin is a clinical-stage, orally administered inhibitor of GlyT1 that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, Diamond-Blackfan Anaemia (DBA) and others. There are currently two ongoing phase 2 clinical studies of bitopertin in patients with erythropoietic porphyria, including an open-label trial called BEACON and a randomized, double-blind placebo controlled trial called AURORA.

Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021.

Disc Medicine is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases.


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