Alzheon, Inc. announced that it has achieved the enrollment target of 325 patients in its pivotal APOLLOE4 Phase 3 trial evaluating oral ALZ-801 in Early AD patients, who carry two copies of the e4 allele of apolipoprotein E gene (APOE4/4 homozygotes). These patients represent approximately 15% 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 (valiltramiprosate) is an oral investigational agent in Phase 3 development as a potentially disease modifying treatment for AD that blocks formation of the neurotoxic soluble beta amyloid oligomers causing cognitive decline in Alzheimer's patients.

In mechanism of action studies, ALZ-801 fully inhibited the formation of amyloid oligomers at the Phase 3 clinical dose. ALZ-801 has shown potential for robust efficacy in the highest-risk Alzheimer's population of APOE4/4 homozygotes, and favorable safety with no events of the brain vasogenic edema seen in trials with plaque-clearing antibodies. Alzheon has pioneered precision medicine in AD by targeting neurotoxic amyloid oligomers and has developed a well-differentiated solution to both treatment and prevention of Alzheimer's with a broad platform of small molecules, which act upstream on the same pathway as anti-amyloid antibodies.

These oral agents prevent the formation of neurotoxic soluble amyloid oligomers without disrupting the insoluble plaque deposits in brain tissue and small vessels, thereby avoiding the vascular complications of brain edema and microbleeds seen with infusions of plaque-clearing antibodies. Alzheon's lead investigational product, ALZ-801, is a first-in-class oral drug with a favorable safety profile that may not only slow, but also arrest the progression of Alzheimer's.