BioXcel gains orphan drug status re leukemia

BioXcel Therapeutics reports that the FDA has granted Orphan Drug Designation for BXCL701, an investigational orally-available systemic innate immunity activator with dual mechanisms of action, for the treatment of Acute Myeloid Leukemia.

BXCL701 is designed to activate innate immune cells, specifically macrophages, by inhibiting the dipeptidyl dipeptidases DPP8 and DPP9, a novel mechanism, and exerts immune stimulatory activity through a pro-inflammatory form of programmed cell death known as pyroptosis. more