In 2013, worldwide rights to ALZ-801 (formerly BLU8499) were licensed to Alzheon Inc. (Alzheon) in return for royalties on sales and revenue sharing. BLU8499 was initially developed by BELLUS Health for the treatment of Alzheimer’s disease (AD). Alzheon is a private company focused on AD and other neurodegenerative disorders.
ALZ-801 is a prodrug of tramiprosate, a beta-amyloid targeting small molecule shown to improve cognition and function in AD patients who are carriers of apolipoprotein E4 (APOE4) AD genotype, and to reduce soluble beta-amyloid in the cerebral spinal fluid of AD patients.
Recent third-party positive clinical results for the treatment of early AD using anti-beta-amyloid antibodies lend further support to the concept of amyloid beta clearance as a promising approach for the treatment of AD.
Alzheon has completed two bridging Phase 1b clinical studies with ALZ-801 and is currently in preparation for a pivotal Phase 2/3 program focusing on treatment of mild AD patients who are homozygous for APOE4, the most important genetic risk factor for late-onset AD.