Startup Alzheon’s Proposal for Alzheimer’s Drug Success Adds New Science to Failed Trials

First published on MedCityNews.com. From a commercial perspective, the last few decades of Alzheimer’s drug development have largely been a wash of promising disease-altering drugs failing to demonstrate efficacy in late-stage trials.

First published on MedCityNews.com. From a commercial perspective, the last few decades of Alzheimer’s drug development have largely been a wash of promising disease-altering drugs failing to demonstrate efficacy in late-stage trials. For startup Alzheon, though, those failures have left behind a trail of valuable clinical and biomarker data that it thinks could lead to hidden treasure.

Alzheon launched today with a plan to scoop up abandoned drug candidates to which it thinks it can apply recent advances in the understanding of Alzheimer’s disease and new diagnostic and prognostic tools to refine the drugs and target them toward more specific groups of patients.

For its first clinical candidate, the Lexington, Massachusetts-based biotech has licensed a prodrug of the small molecule tramiprosate from Bellus Health (formerly Neurochem Inc.) of Quebec, Canada. It was designed to inhibit the formation of amyloid assemblies in the brain that experts have long considered characteristic of the disease.

In the late 2000s, Neurochem took the drug as far as Phase 3 studies involving more than a thousand Alzheimer’s disease patients. It failed to meet the trial’s efficacy endpoints.

But in a retrospective analysis of clinical data, researchers found that the drug seemed to slow atrophy of the hippocampus, a kind of brain damage associated with Alzheimer’s, especially in patients with a gene variation called APOE4.

Alzheon will piggyback on those clues and use a prodrug form of tramiprosate, or an inactive substance that is converted into an active drug inside the body. CEO Dr. Martin Tolar said the prodrug has better properties than its parent drug.

Tolar is a neurologist by training and spent six years in leadership positions at Pfizer before taking on roles at various smaller biotech companies including CoMentis Inc., NormOxys and Knome Inc. Working with him on this new venture is former CoMentis and NormOxys colleague John Hey, a 20-year drug development veteran, and Dr. Mark Versavel, who has led neurology projects at Bayer and Pfizer.

The team will use their combined science and business savvy and the existing clinical data behind ALZ-801 to design a Phase 2 efficacy study around specific subpopulations of Alzheimer’s patients who stand to benefit most from the drug. Tolar said the trial should start next year.

While smaller, more targeted trials should in theory be quicker and more likely to produce positive results than large trials, the trials will still be costly. Tolar declined to disclose details of his company’s initial financing but said his team is working with two private investors with a history success in biopharmaceuticals.

As plans for the trial get underway, Alzheon, which was incorporated in August, is also at work building a pipeline. Tolar said the team is in talks with other companies about in-licensing deals that would bring in other therapeutic approaches that have demonstrated safety and proof-of-concept activity in neurodegenerative disease.

Chief Scientific Officer Hey summed up the company’s long-term strategy in a statement: “Alzheon has unique capabilities to deploy our drug development platform and leverage the clinical knowledgebase from past Alzheimer’s programs to deliver the best of both worlds:  innovative treatments for challenging neurodegenerative diseases and well-designed clinical programs that have a high likelihood of success.”

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