First Drug Candidate from NIH Programme Acquired by Biopharmaceutical Company

A drug candidate developed by researchers at the NIH’s National Center for Advancing Translational Sciences (NCATS) and its collaborators to treat sickle cell disease has been acquired by Baxter International’s BioScience business. The drug candidate, Aes-103, is the first specifically developed to target the underlying molecular mechanism of sickle cell disease. Baxter now will advance the clinical development activities required for regulatory approval and commercialisation.

This is the first time a company has acquired a drug candidate developed with NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) programme resources. Baxter International recently acquired AesRx, LLC, Newton, Massachusetts — the TRND programme collaborator — including Aes-103. TRND and AesRx researchers worked together to develop Aes-103 through a Phase II clinical trial to evaluate safety and effectiveness. The trial data indicated that Aes-103 significantly reduced patients’ pain.

“This is a wonderful example of why NCATS was created,” said NIH Director Francis S. Collins, MD, PhD. “The progress made thus far in the development of Aes-103 demonstrates NCATS’ catalytic role in bringing together the necessary players, whether academic, nonprofit or industry, to overcome obstacles to translation and advance badly needed treatments to patients.”

Currently, the only drug approved by the FDA to treat sickle cell disease is hydroxyurea, a drug initially developed to treat cancer. However, the clinical utility of hydroxyurea is limited. Many individuals with sickle cell disease either do not respond to the drug, or they may experience undesirable side-effects.

“Sickle cell was the first disease to ever have its molecular cause discovered — more than 65 years ago — and now a potential treatment based on that discovery has at last been developed,” said NCATS Director Christopher P. Austin, MD. “This success validates the NCATS model, which is based on a novel collaborative approach that de-risks intervention development programs to enable private-sector investment. We look forward to applying this model to the thousands of rare diseases that are currently untreatable so that we realize the NCATS mission of getting more treatments to more patients more quickly.”

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