Alzheon announced the peer-reviewed publication of full results from its APOLLOE4 Phase 3 trial of oral valiltramiprosate (ALZ-801) in individuals homozygous for the APOE4 gene, with early Alzheimer’s disease. The trial enrolled 325 participants over 78 weeks across North America and Europe, focusing on those with mild cognitive impairment (MCI) or mild Alzheimer’s dementia who carry two copies of APOE4.
The APOLLOE4 trial did not meet its primary endpoint of slowing cognitive decline across the overall study population, as measured by the ADAS-Cog13. However, in a prespecified subgroup of patients at the MCI stage, the company claims nominally statistically significant benefits in cognition and clinically meaningful effects in functional measures. Brain imaging analyses reportedly showed slowing of atrophy in multiple regions.
On safety, Alzheon says ALZ-801 was not associated with an increased risk of amyloid-related imaging abnormalities (ARIA), such as brain edema or microhemorrhages, which have been concerns in other anti-amyloid therapies. APOE4/4 homozygotes represent about 15 % of Alzheimer’s patients and are considered high risk.
The long-term extension of APOLLOE4 is ongoing, with up to 104 additional weeks of treatment. The company’s next steps include further clinical development and exploration of regulatory pathways for ALZ-801, potentially targeting patients earlier in disease progression.
Independent assessment will be needed to confirm whether the observed subgroup effects translate into a viable treatment path.
