Phase III data shows benefits of ALZ 801 (tramiprosate prodrug) in Alzheimer's- Alzheon

Alzheon has announced new data analyses from two Phase III studies of ALZ 801 (tramiprosate prodrug) in Alzheimer's disease (AD) patients based on their APOE4 genotype. Tramiprosate is the biologically active agent of ALZ-801, a prodrug developed by Alzheon and currently being advanced into pivotal Phase III studies in AD patients with the APOE4/4 homozygous genotype, a patient population known to have high prevalence and burden of cortical amyloid pathology, a hallmark of Alzheimer's disease.

The new subgroup analyses of the tramiprosate Phase III trials showed that patients with one or two copies of the E4 allele of apolipoprotein E, referred to as APOE4, who received tramiprosate had clinically meaningful benefit on cognition and/or function on top of standard of care through 78 weeks of treatment. Data from the APOE4/4 homozygous subgroup in the North American study suggested a dose-dependent clinical benefit at the high dose of tramiprosate (150mg twice daily) compared to placebo (N = 147, nominal p-value = 0.04). APOE4 heterozygous subjects showed lower magnitude of effect with a significant clinical benefit on function at both doses, and positive trends on cognition at the 100mg twice-daily dose of tramiprosate. Patients who were APOE4 non-carriers did not show benefit from tramiprosate at either dose.

The safety profile was similar in the APOE4 carriers and non-carriers, and consistent with published safety analyses in the overall study population. These analyses are being presented at the 14th Annual International Athens/Springfield Symposium on Advances in Alzheimer Therapies Conference.