New analyses of Phase III data for tramiprosate identifies biological phenotypes in Alzheimer's disease.- Alzheon Inc.

Alzheon, Inc. presented new analyses of previous Phase III data for tramiprosate, an investigational amyloid anti-aggregation agent and the active molecule of prodrug ALZ-801. The new results strengthen Alzheon's precision medicine approach which identifies distinct biological phenotypes in Alzheimer's disease (AD) patients. These phenotypes were described in the recently-published analyses of the tramiprosate Phase III data, showing that AD patients who carry two ?4 alleles of apolipoprotein E gene (APOE4/4 homozygotes) respond with the largest clinical benefits.

In order to refine the selection criteria of patients for the upcoming ALZ-801 studies, the new analyses further evaluated tramiprosate efficacy in APOE4/4 homozygous AD patients according to disease severity based on their Mini-Mental State Examination (MMSE) scores at baseline. The results show tramiprosate's largest efficacy signals on cognition and function in APOE4/4 homozygous patients with Mild AD, defined as MMSE of 22 and higher. These new results were discussed in an oral presentation at the 9th annual Clinical Trials on Alzheimer's Disease (CTAD) conference, held December 8-10 in San Diego. At CTAD, Alzheon also presented a poster on the Phase 1 clinical studies and bridging data for ALZ-801, an optimized prodrug of tramiprosate, that support the pivotal clinical program planned to begin in 2017, which will evaluate ALZ-801 as a potential disease-modifying treatment for APOE4/4 homozygous patients at the Mild stage of Alzheimer's disease.

The new analyses presented at CTAD evaluated Alzheimer's patients in the Phase 3 tramiprosate studies who were carriers of the homozygous APOE4/4 genotype, after segmenting them from the larger, all-comer study population involving more than 2,000 patients in North America and Europe. The data for APOE4/4 patients was then analyzed based on the severity of disease as measured by the MMSE scores, from the overall Mild and Moderate AD population (MMSE 16-26) to milder groups (MMSE 20-26 and 22-26). The analysis showed that the efficacy of tramiprosate was greatest in the least impaired patient group with MMSE scores of 22 to 26. In this population, large and nominally significant effects were observed on both cognition (measured by ADAS-cog scale) as well as function (measured by CDR-SB scale).