Sep 13, 2023
Marco Meglio
Over a 2-year treatment period, patients on ALZ-801 demonstrated significant correlations between effects on volumetric MRI outcomes and 3 cognitive scales, suggesting cognitive gains are a result of preservation of brain structures from neurodegeneration and atrophy.
Alzheon, the drug makers of ALZ-801, announced positive topline data from its phase 2 biomarker study (NCT04693520), with findings that showed significant reductions in plasma phosphorylated tau (p-tau) levels and improved cognitive scores after 2 years of treatment with the agent. Patients on ALZ-801 also demonstrated a statistically significant reduction in measures of hippocampal volume in comparison with an external control arm of matched ADNI patients, which were consistent with previously reported 12-month data.
The open-label, single-arm phase 2 trial included 84 patients with early-stage Alzheimer disease (AD) who carried either 1 or 2 copies of the ε4 allele of apolipoprotein E gene (APOE3/4 heterozygotes and APOE4/4 homozygotes, respectively) and were treated over a 104-week period. Treatment with the agent resulted in a statistically significant 43% reduction in plasma p-tau181 (P <.009) after 52 weeks and a 31% reduction at week 104 (P <.045). Plasma amyloid-β42 levels decreased throughout the treatment period, reaching a statistically significant 4% reduction from baseline (P <.042) at week 104, while the reduction in plasma Aβ40 levels stabilized at 52 weeks at a new homeostatic level.