NEW YORK – Biopharmaceutical company Alzheon on Tuesday said it is moving ahead with regulatory filing plans for its lead Alzheimer's disease candidate ALZ-801 (valiltramiprosate), an oral tablet for patients with early-stage disease who carry two copies of the APOE4 allele.
ALZ-801, currently in Phase III development, is an investigational disease-modifying treatment that blocks formation of neurotoxic soluble beta-amyloid oligomers in the brain, a sign of disease onset and an initial step in the amyloid cascade many say causes the progressive neurodegenerative disease.
Framingham, Massachusetts-based Alzheon said it expects a top-line data readout from the pivotal, randomized placebo-controlled Phase III trial, dubbed APOLLOE4, in the third quarter of 2024. Soon after, the firm said it will file a new drug application with the US Food and Drug Administration, and if the agency approves ALZ-801, Alzheon hopes to launch a commercial product in 2025.
"ALZ-801 is potentially two years from a US commercial launch as the first oral agent that can slow or even stop and prevent Alzheimer's pathology in patients and healthy individuals at risk for the disease," Alzheon Founder, President, and CEO Martin Tolar said in a statement.
While the ALZ-801 development program currently focuses on patients who are homozygous carriers of the APOE4 allele and therefore at higher risk for developing Alzheimer's, Alzheon is hoping in the future to study the drug for treating Alzheimer's and even preventing disease onset in patients who carry one copy of the APOE4 allele as well as in non-carriers.
In preliminary results from a Phase II trial, investigators found Alzheimer's patients with early-stage disease who are APOE4 carriers experienced significant plasma p-tau181 reduction 13 weeks after initiating treatment and significant plasma amyloid-beta42 and amyloid-beta40 reduction at 52 weeks. Investigators also observed a reduction in hippocampal atrophy by about 20 percent at 12 months compared to matched controls. The Phase II study is expected to be completed in Q3.
Alzheon will present updated biomarker results from the Phase II trial and data on baseline imaging characteristics from its Phase III trial at the Alzheimer's Association International Conference in Amsterdam next week.