Biosight Ltd., an Israel-based pharmaceutical development company focused on the development of innovative therapeutics for haematological malignancies and disorder, announced on Monday that it has received Orphan Drug Designation from the United States Food & Drug Administration (FDA) for its aspacytarabine (BST-236), an investigational novel antimetabolite, to treat myelodysplastic syndromes (MDS).

The product was also granted Orphan Drug Designation in 2019 for aspacytarabine to treat acute myeloid leukaemia (AML). It is a novel proprietary anti-metabolite and includes cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine.

Subsequent to the product's encouraging single-agent efficacy and safety profile in the recently completed Phase 2b trial in front-line treatment of AML patients unfit for standard induction chemotherapy, the aspacytarabine development program is being expanded to second-line treatment of AML and MDS in two ongoing Phase two clinical trials, one in the US and Israel and one in France, in partnership with the European cooperative group, GFM.