Data supports study design for ALZ-801 clinical programme

Results from studies of ALZ-801 and its active molecule tramiprosate support the study design for the clinical programme, reports Alzheon.

ALZ-801 is an oral amyloid-targeted medicine that has been studied in a genetically-defined population of patients at the mild stage of Alzheimer’s disease (AD) with ongoing amyloid accumulation. The clinical programme for this treatment uses a precision medicine approach to drug development for AD, targeting patients who are genetically defined as homozygous for the e4 allele of apolipoprotein E (APOE4/4 patients).

“Alzheon’s new insight are defining a more targeted and higher probability path for developing innovative medicines by directing Alzheimer’s R&D to the right patients, with the right drug and at the right stage of their disease,” said Dr Martin Tolar, PhD, founder, president and CEO of Alzheon. “We are pioneering a precision medicine approach to Alzheimer’s disease with our pivotal programme for ALLZ-801, by focusing on a well-recognised, genetically-defined subset of Alzheimer’s patients with the APOE4 genotype and by targeting patients at an early point in their disease progression, who have shown the greatest response to our lead molecule.”

The company has completed the Phase I bridging programme with ALZ-801, a novel prodrug of tramiprosate, and has presented pharmacokinetic and safety data supporting selection of the optimal dose of ALZ-801 that will be used in the upcoming confirmatory study.

Further, the company has presented clinical analyses for tramiprosate, which evaluated AD patients in previous Phase III 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 the US and Europe. These analyses support the refinement of patient selection for the upcoming ALZ-801 studies to focus on APOE4/4 homozygous patients at the mild stage of AD.

“Our latest clinical analyses suggest that tramiprosate efficacy is highest in APOE4/4 homozygous AD patients at the mild stage of disease, which is consistent with the findings from the studies with other amyloid-targeted therapies. The potential for tramiprosate efficacy to be sustained over 130 weeks is also very encouraging,” added Dr Susan Abushakra, chief medical officer of Alzheon. “These analyses have informed the selection criteria for the upcoming study of ALZ-801 in homozygous AD patients. With these promising efficacy data, the favourable long-term safety profile of tramiprosate, and the improved gastrointestinal tolerability of ALZ-801, we are poised to initiate the pivotal programme with ALZ-801.”