Alzheon says data back plans for phase 3 program

Amyloid plaques in Alzheimer's disease
Amyloid plaques in Alzheimer's disease (Jensflorian/Creative Commons)

Alzheon is still trying to build a case for a pivotal trial of its Alzheimer’s candidate, and the company says it has new data backing up the drug’s use in patients genetically predisposed to develop the disease.

The tiny biotech says it will be presenting new findings this week from a pivotal trial of its beta-amyloid-targeting drug tramiprosate (ALZ-801) in patients with two copies of the APOE4 gene and mild Alzheimer’s, which suggest it does better than placebo at stabilizing symptoms over 18 months.

Alzheon has been trying to raise the capital it needs to run another pivotal trial of tramiprosate, but had to withdraw plans for a $80 million-plus IPO earlier this year after a lackluster investor response. That stemmed no doubt from the ever-extending list of amyloid drugs that have failed to move the needle on memory and cognition measures in clinical trials—including tramiprosate itself when it was owned by Neurochem (now known as Bellus Health).

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Framingham, Massachusetts-based Alzheon has latched onto data from a North American trial showing that, while the drug flunked overall, it did much better in homozygous APOE4/4 patients, a group that represents around 10% of all Alzheimer’s patients and who tend to be at high risk of developing the disease at an earlier age.

Since then, it’s been trying to raise funds to test its theory with a confirmatory trial specifically designed to include only APOE4/4 individuals. It remains to be seen whether Alzheon will take another stab at a public listing or try to raise funds for a pivotal trial another way but, in the meantime, it is trumpeting what it says are “large clinical benefits” with its drug.

Specifically, the new analysis shows that according to the widely used ADAS-cog rating scale, 57% of patients with mild Alzheimer’s remained “cognitively stable” over the 18-month trial period, compared to just 20% of those on placebo.

A similar pattern was seen when it came to the proportion of patients with minimal or zero decline on the Disability Assessment for Dementia  scale over the course of the study, at 46% for tramiprosate versus 18% in the placebo group. The results will be presented on Wednesday at the Alzheimer’s Association International Conference in Chicago.

The data “strengthens the confidence in our pioneering precision medicine approach for ALZ-801 in the genetically-defined population of APOE4/4 homozygous patients with mild or early Alzheimer’s disease,” said Martin Tolar, M.D., Ph.D., Alzheon’s founder and CEO.

He believes a pattern is emerging around drugs that target soluble amyloid fibrils, which include not only tramiprosate but also other candidates such as Biogen’s aducanumab and BAN-2401.

That is “the only therapeutic approach to date that has shown a disease modifying effect in Alzheimer’s patients,” according to Tolar.