BIIB100

BIIB100 is an orally bioavailable small molecule inhibitor of Exportin 1 (XPO1), a protein responsible for the nuclear export of many proteins and RNA. By inhibiting XPO1, BIIB100 aims to regulate abnormal nucleocytoplasmic transport implicated in neurodegenerative diseases. Preclinical studies demonstrated broad neuroprotective effects in models of multiple sclerosis, traumatic brain injury, and Huntington’s disease. The drug was developed primarily for amyotrophic lateral sclerosis (ALS) by Biogen after acquiring rights from Karyopharm Therapeutics. A Phase 1 clinical trial evaluated its safety and pharmacokinetics in ALS patients but development was discontinued after completion of the study[1][3][5].