Alzheon reports positive interim results from phase 2 trial of oral AD therapeutic
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Key takeaways:
- Twice daily ALZ-801 resulted in a –41% change from baseline in plasma p-tau181 at 52 weeks.
- Bilateral hippocampal volume atrophy at 1 year was reduced by roughly 20% compared with matched controls.
Alzheon Inc. has announced positive interim findings from an ongoing phase 2 study evaluating ALZ-801, an investigational disease-modifying treatment for Alzheimer’s disease.
According to a release from Alzheon, ALZ-801 (valiltramiprosate) blocks formation of neurotoxic soluble beta-amyloid oligomers in the brain associated with cognitive symptoms and AD progression. It received fast track designation from the FDA in 2017.
The open-label trial for ALZ-801 enrolled 84 apolipoprotein E-4 (APOE4) carriers at seven sites in the Czech Republic and Netherlands to evaluate the effects of twice daily 265 mg ALZ-801 on plasma biomarkers of AD and hippocampal volume. Researchers are also assessing clinical outcomes and the drug’s safety, efficacy, tolerability and pharmacokinetics over 104 weeks of treatment.
According to the release, 80 participants completed 26 weeks of treatment and 75 completed 52 weeks. Researchers reported significant change from baseline in plasma p-tau181 at 13 and 26 weeks, reaching –41% at 52 weeks.
Data additionally showed bilateral hippocampal volume atrophy at 1 year was reduced by roughly 20% compared with matched controls. The most common adverse events were mild nausea and COVID infection, with no serious events related to drug administration.
“By inhibiting the misfolding of amyloid monomers and subsequent formation of neurotoxic amyloid oligomers, we observed a rapid and sustained reduction in plasma p-tau181 as early as 13 weeks, with continued reduction through 52 weeks,” John Hey, PhD, chief scientific officer at Alzheon, said in the release. “Preventing oligomer formation with an oral tablet is a simplified approach to disease modification in Alzheimer’s.”
The company also announced a phase 3, double-blind randomized trial for ALZ-801, which will compare the oral therapeutic with placebo over 78 weeks in patients with the APOE4/4 genotype. That study is supported by a $47 million grant from the National Institute on Aging.