Alzheon Lands $47M to Take Another Shot at a Pivotal Alzheimer’s Test

[Corrected 6:36 p.m. See below.] Alzheon, an Alzheimer’s disease drug developer that has twice filed and withdrawn its IPO plans, now has a $47 million commitment from the National Institutes of Health (NIH) to fund a pivotal study testing an experimental treatment in patients with the memory-robbing disorder.

The cash, a five-year grant from the NIH’s National Institute on Aging, represents another shot for the company and its drug, ALZ-801, which is based on a compound that previously failed in clinical trials. But the Framingham, MA-based biotech now plans to test its small molecule in a narrower group: patients who have are in the early stages of Alzheimer’s and also have two copies of APOE4/4, a genetic variant associated with a higher risk of developing the disease. The company says that this precision medicine approach increases the chances of the trial’s success. [Paragraph updated to clarify the distinction between ALZ-801 and tramiprosate.]

Alzheon aims to stop amyloid, a protein that forms plaques on the brains of Alzheimer’s patients and is a hallmark of the disease’s progression. It’s the same protein targeted by several drugs that previously failed in late-stage clinical testing. Many of those drugs aimed to clear away those protein clumps. The Alzheon drug is intended to stop them from forming altogether. It’s designed to block amyloid oligomers, which are precursors of the plaques.

The Alzheon drug is a prodrug of tramiprosate, the active ingredient in a drug that was initially developed and tested by Montreal-based Neurochem, a company now known as Bellus Health (NASDAQ: [[ticker:BLU]]). Under Bellus, tramiprosate failed in Phase 3 studies enrolling more than 2,000 patients with mild-to-moderate Alzheimer’s. But the Canadian company did not take into account APOE4 in conducting its study, which enrolled patients who were carriers of the gene and those who were not. Alzheon’s analysis of the data found “promising clinical signals” in patients who have two copies of the gene, the company said in its IPO filings. Those are the patients that Alzheon believes can be helped by ALZ-801, which as a prodrug, converts into the active drug tramiprosate inside the body. [Paragraph updated to clarify the distinction between ALZ-801 and tramiprosate.]

The Phase 3 study is designed to enroll 300 patients who will be randomly assigned to receive the Alzheon pill or a placebo twice a day for 18 months. The main goal of the study is assessing cognition measured according to a scale used to evaluate Alzheimer’s patients. The study will also track biological indicators, including the levels of amyloid and tau proteins in the blood and cerebrospinal fluid. Imaging technology will be used to assess the drug’s effects on the brain.

Alzheon formed in 2013, led by CEO Martin Tolar, a neuroscientist and Pfizer (NYSE: [[ticker:PFE]]) veteran. The company first filed to go public in 2018, setting a preliminary $80 million goal. It withdrew those plans but left the door open to revive them. It refiled IPO paperwork later that year, with the fundraising target cut in half. That filing was withdrawn in early 2019. Now with the NIH grant secured, Alzheon plans to start a Phase 3 test for ALZ-801 in the first quarter of 2021.

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