Alzheon Alzheimer’s asset ready for phase III with high hopes

Alzheon today released positive results from its Phase II biomarker trial on ALZ-801 (valiltramiprosate) in patients diagnosed with neurodegenerative disorders such as Alzheimer’s disease.

ALZ-801 is administered orally and works by blocking the formation of neurotoxic soluble amyloid oligomers that later lead to cognitive decline in patients with Alzheimer’s disease. It has also shown potential for safety and efficacy in Alzheimer’s patients who are most at risk, particularly those who have two copies of the apolipoprotein ε4 (APOE4/4) allele. Alzheon is about to move into its Phase III APOLLOE4 trial, which is focused on this population.

The Phase II biomarker study is fully enrolled with 84 patients and is still ongoing. It is evaluating the use of ALZ-801 in patients with early Alzheimer’s disease who have one or two copies of APOE3/4 heterozygotes and APOE4/4 homozygotes. APOE4 is one of the main risk factors for Alzheimer’s disease after aging.

Patients received ALZ-801 265 mg orally twice daily and were assessed for the primary outcome of change from baseline in plasma p-tau181. The biomarker analysis was based on results from 80 patients who completed the week 26 visit.

In this study, participants saw a 29% reduction in plasma p-tau181 after 26 weeks and an 18% reduction after 13 weeks. The plasma p-tau181/Aβ42 ratio fell by 30% after 26 weeks and by 21% after 13 weeks. The researchers said there is strong potential for disease modification with effects on these biomarkers.

“Rather than slowing patients’ cognitive decline as seen in trials with other agents, subjects treated with ALZ-801 demonstrated significant cognitive gain from baseline on memory tests, showing improvement during treatment.These well-differentiated results position ALZ-801 to potentially become the first oral agent capable of slowing or even stopping and preventing Alzheimer’s disease in patients and healthy individuals at risk for the disease,” said Martin Tolar, MD, Ph.D., founder, president and CEO of Alzheon, in a statement.

Besides the biomarker results, Alzheon’s phase II study also identified a memory and standard of living test called Rey Auditory Verbal Learning Test (RAVLT) as a secondary outcome. Patients showed significant improvement from baseline records for immediate and delayed recall after week 26. No serious events or signs of vasogenic edema were observed.

“ALZ-801 is in a class of its own as an easy-to-administer oral tablet that has shown the potential for robust efficacy with a favorable safety profile, avoiding the vascular complications of cerebral edema and microhemorrhage seen with plaque-clearing infusions of anti-amyloid antibodies,” added John Hey, Ph.D., chief scientific officer of Alzheon, in the same press release.

the National Institute of Aging gave Alzheon a $47 million grant to fund its Phase III trial, which examines the efficacy, safety, biomarkers and imaging effects of taking 265 mg of ALZ-801 twice daily. This randomized double-blind trial will last 78 weeks.

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