Disc Medicine, NHLBI Partner for DBA Therapy Trial


Disc Medicine has partnered with the National Heart Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH) to conduct a Phase II clinical trial of bitopertin in patients with Diamond Blackfan Anemia (DBA).

DBA, a rare inherited blood disorder, is characterized by the inability of the bone marrow to produce red blood cells.

NIH conducts and funds the study under a Collaborative Research and Development Agreement (CRADA).

Under the terms of CRADA, NHLBI will act as regulatory sponsor and will also conduct a Phase II clinical trial co-funded by Disc Medicine.

The trial, which is expected to begin mid-year, is under the direction of Dr. Cynthia Dunbar, Director of the NHLBI’s Molecular Hematopoiesis Section, Chief of the Division of Translational Stem Cell Biology, and Distinguished NIH Investigator. be carried out.

Disc Medicine CEO and President John Quisel said:

“This collaboration will allow DBAs to benefit from NHLBI experience in designing and conducting clinical studies.

“Disc now has ongoing development programs for Bitoperatin in both erythropoietic porphyria and DBA.”

A dose-escalating, single-arm, pilot phase II clinical trial of bitopertin will be conducted in DBA patients with steroid-resistant and/or recurrent disease or who cannot tolerate systemic corticosteroids.

This includes planned dose escalation for each participant for continuous assessment of hematologic response.

Participants may continue on extended treatment within the study after the main treatment period has ended.





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