FDA Fast-Tracks Alzheon’s Anti-Amyloid Alzheimer’s Agent
The FDA has granted Fast Track designation to Alzheon, Inc.’s investigational anti-amyloid drug ALZ-801 for the treatment of Alzheimer’s disease (AD). Using a precision medicine approach, Alzheon is evaluating ALZ-801 based on genetic markers and stage of the disease. ALZ-801 is an oral agent that has a novel molecular mechanism of action blocking the formation of toxic amyloid oligomers1 associated with the development and progression of AD. Clinical data for ALZ-801 and its active agent, tramiprosate, suggest long-term clinical efficacy in AD patients with the e4 allele of apolipoprotein E (APOE4) genotype, along with a favorable safety profile.
The initial pivotal Phase 3 program will focus on approval in the genetically-defined subpopulation of high risk patients who are homozygous for APOE4 at the mild stage of AD. Future clinical plans include expanding the evaluation of ALZ-801 in additional populations of AD patients, according to the company. To date, there are no approved drugs that target the underlying pathology and slow the progressive cognitive and functional decline of Alzheimer’s disease.