Alzheon, Inc. announced that it has achieved enrollment target of 80 patients for its Phase 2 biomarker study of ALZ-801 in Early AD patients, who carry either one or two copies of the e4 allele of apolipoprotein E gene (APOE3/4 heterozygotes and APOE4/4 homozygotes, respectively). These patients represent two-thirds of all AD patients, have an earlier onset of AD and higher risk of rapid disease progression, and carry a high burden of neurotoxic soluble beta amyloid oligomers. ALZ-801 is an oral agent in Phase 3 development as a potentially disease modifying treatment for AD. In mechanism of action studies, ALZ-801 has been shown to fully inhibit the formation of neurotoxic soluble amyloid oligomers at the Phase 3 clinical dose. This therapeutic approach has been validated by the FDA’s recent accelerated approval of Biogen’s anti-amyloid antibody infusion Aduhelm (aducanumab) that established toxic amyloid species as an effective therapeutic target in AD. The ongoing APOLLOE4 Phase 3 trial (NCT04770220), which began dosing patients earlier this month, is supported by a $47 milliongrant from the National Institute on Aging. The APOLLOE4 Phase 3 trial is focused on APOE4/4 homozygotes, who constitute approximately 15% of AD patients. The Phase 2 biomarker data will guide the design of future Phase 3 trials in APOE3/4 heterozygotes, increasing the potential patient population of ALZ-801 tablet to include 65-70% of all AD patients. Originally targeting an enrollment of 40 patients, the Phase 2 study size was doubled to 80 patients to strengthen the statistical power in each of the two APOE4 genotypes for the primary outcome of change from baseline of phosphorylated-tau (p-tau181), a key biomarker of AD pathology. The study enlargement has been facilitated by a high demand from patients and clinicians to participate in the trial, allowing accelerated enrollment across 7 clinical sites in the Czech Republic and Netherlands. The Phase 2 biomarker study (NCT04693520) is evaluating the effects of 265 mg twice daily oral dose of ALZ-801 on fluid biomarkers, imaging, and clinical outcomes over two years of treatment. The study objective is to confirm the disease modifying potential for ALZ-801 in both APOE4/4 and APOE3/4 AD patients by assessing its effects on cerebrospinal fluid and plasma biomarkers of amyloid, tau, neuronal injury and neuroinflammation. The study also evaluates drug effects on brain hippocampal volume using magnetic resonance imaging, and on cognitive and functional outcomes. The first interim data readout is expected in 2022.