Biosight announced the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation to BST-236, an investigational novel antimetabolite, for the treatment of acute myeloid leukemia (AML). BST-236 is being developed for the treatment of hematological malignancies and disorders, and is currently in Phase 2b development for front-line treatment of AML.
"We are very pleased to have received Orphan Drug Designation from the FDA for BST-236, our lead therapeutic candidate," said Ruth Ben Yakar, Ph.D., CEO of Biosight. "The emerging clinical data from a completed Phase 1/2a and an ongoing Phase 2b studies, suggest that BST-236 may provide a superior front-line treatment option for AML patients, especially for patients who are medically unfit to receive standard chemotherapy, and may establish BST-236 as the new core therapeutic in AML and other hematological malignancies and disorders."
Orphan Drug Designation by the FDA entitles Biosight to seven years of market exclusivity for the use of BST-236 for the treatment of AML, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.
BST-236 is a novel antimetabolite, a pro-drug of the chemotherapeutic drug cytarabine. Cytarabine has been the backbone of AML therapy for the past 40 years, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically unfit patients. BST-236 is designed to enable delivery of high cytarabine doses to leukemia cells with lower systemic exposure to the free drug and relative sparing of normal tissues. As such, BST-236 may serve as a superior core therapy for AML and other hematological malignancies and disorders, including to medically unfit or older adults.
