Repurposed cancer drug to enter Phase I trials for Alzheimer’s disease this year

alzheimer's disease

In the world of Alzheimer’s disease drugs, the past year has been marked by disappointing Phase 3 clinical trial results for a handful of much-anticipated antibody drugs.

But the failure of those drugs, designed to clear toxic beta amyloid plaques that accumulate in the brain, isn’t discouraging to Cleveland startup ReXceptor Inc., which has just obtained an exclusive license to commercialize a treatment method from Case Western Reserve University.

ReXceptor is taking a fundamentally different approach. “We’re not trying to take (beta amyloid) out of the body entirely; we’re trying to restore a natural function,” explained CEO Michael Haag, who’s also the interim director of technology management at CWRU. “Everyone produces a certain amount of amyloid beta which can be shuttled out of the brain, but as you age, you have less and less of those carrier proteins. We want to give the brain back these carrier proteins.”


ReXceptor thinks it can do that by using a drug that’s already been approved by the U.S. Food and Drug Administration. Bexarotene, marketed by Medicis as Targretin, is approved for treatment of cutaneous T-cell lymphoma. But last year, CWRU neuroscience professor Gary Landreth and then-graduate student Paige Cramer published a paper in Science detailing how the drug, when administered to mice, cleared 25 percent of amyloid beta build up in the brain and reversed some of the cognitive impairment of Alzheimer’s disease within 72 hours of a single dose.

So far, the company has worked with $1.4 million in grants and investments and is now ready to initiate a Phase I, proof of mechanism study in the second half of this year. According to Haag, it will involve 12 healthy volunteers and take six months to a year.

“Gary’s paper showed a robust response in mice, and we want to make sure that the same response is recapitulated in human subjects,” he said. “The endpoint is that the drug gets to brain, and that the biomarkers that we’re interested in move the way that we think they will.”

As the number of U.S. adults with Alzheimer’s is expected to nearly triple by 2050, and the risk of death from the disease appears to be going up. The need for disease-altering therapies led the FDA to issue more flexible guidelines to drug developers in March.

We’re bringing active investors and corporate development execs together with promising startups at MedCity News INVEST. Join us April 12-13, 2016, in Chicago for the premier national investing conference based in the Midwest.

No comments