Phase 2 trial of therapeutic for patients with early AD yields positive results
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Alzheon Inc. reported statistically significant and clinically relevant results in patients with Alzheimer’s disease following 12 months of treatment with the oral investigational therapeutic ALZ-801 in its phase 2 trial.
According to a release from Alzheon, ALZ-801 is an agent currently in phase 3 development as a potentially disease-modifying treatment for AD that blocks formation of neurotoxic soluble beta amyloid oligomers that cause cognitive decline in patients with AD.
In mechanism of action studies, ALZ-801 fully inhibited the formation of amyloid oligomers at the phase 3 clinical dose, the company reported. ALZ-801 has shown potential for robust efficacy in the highest-risk AD population — patients with two copies of the apolipoprotein epsilon 4 allele (APOE4/4 homozygotes) — and favorable safety with no events of brain vasogenic edema, seen in trials with plaque-clearing antibodies, the release additionally revealed.
The ongoing, fully enrolled phase 2 biomarker study is evaluating ALZ-801 in 84 patients with early AD, who carry either one or two copies of the epsilon 4 allele of apolipoprotein E gene (APOE3/4 heterozygotes and APOE4/4 homozygotes, respectively), representing two-thirds of AD patients. APOE4 genotype, the leading risk factor for AD after aging, is associated with a several-fold higher brain burden of neurotoxic amyloid oligomers.
All participants received oral ALZ-801 265 mg twice daily, with 75 individuals (mean age, 69 years; 52% female) completing the week 52 visit and were included in this prespecified analysis. In this population, ALZ-801 demonstrated a significant 41% reduction from baseline in plasma p-tau181 at 52 weeks. ALZ-801 also significantly reduced the plasma p-tau181/beta amyloid-42 ratio by 37% at 52 weeks.
“Rather than slowing the cognitive decline of patients as seen in trials with other agents, subjects treated with ALZ-801 demonstrated cognitive gain from baseline status on memory tests and maintained their cognitive skills over 1 year,” Martin Tolar, MD, PhD, founder, president, and CEO of Alzheon. “These results position ALZ-801 to potentially become 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.”
Per the release, Alzheon has received support from the National Institute on Aging in the form of a $47M grant to fund the APOLLOE4 phase 3 study with ALZ-801.