Can a 12-person Framingham startup compete with major drug makers like Merck & Co., Eli Lilly & Co., and Biogen Inc. in developing medicines for Alzheimer’s disease?
Martin Tolar, chief executive of Alzheon Inc., thinks so. At the Alzheimer’s Association International Conference in Toronto, his little-known Massachusetts company on Monday outlined ambitious plans to bring its drug candidate into late-stage clinical trials.
Three years after it was created to license an Alzheimer’s drug compound from a Canadian company that had given up on it, Alzheon reported clinical data that sets the stage for two studies of its drug candidate, called ALZ-801, in a subset of Alzheimer’s patients.
Tolar said in an interview that Alzheon hopes to launch the three-year studies, each involving about 500 patients around the world, at the end of this year. But first the company hopes to raise about $100 million to bankroll its clinical trials from private investors, an initial public offering, or a partnership with a larger drug maker.
“Our goal is to change the course of the disease,” Tolar said. “This disease is not only very expensive, but it’s going to destroy the fabric of our society. If you look at the field of Alzheimer’s, everything has failed. But the field has been learning from those failures.”
Like experimental treatments developed by Cambridge-based Biogen Inc. and other companies, ALZ-801 targets the amyloid plaque that builds up in the brain, which many neurologists suspect triggers Alzheimer’s, a neurodegenerative disease that robs people of their memories. An estimated 5.4 million mostly older Americans have been diagnosed with Alzheimer’s.
But unlike the monoclonal antibodies used by rival companies, which cause side effects such as nausea and stomach upset, Alzheon’s twice-a-day pill is based on an anticlumping agent that company scientists believe will be easier for patients to tolerate. It is also aimed at a subset of patients with a specific gene who are more sensitive to side effects from other drugs.
Anton Porsteinsson, who directs the Memory Disorders Clinic at the University of Rochester School of Medicine and sits on Alzheon’s scientific advisory board, had been an investigator for clinical trials of what is now ALZ-801 when it was being developed by Neurochem Inc. in Saint-Laurent, Quebec. Porsteinsson said Neurochem leaders, disappointed in their trial results for a broader population, may have erred by not studying the drug’s effect on subgroups.
“It was a very small company and they bet everything on this, and the top-line results weren’t what they wanted,” he said. “They didn’t do the right kind of follow-up analysis on the subgroups. . . . Alzheon was on the hunt for a good drug candidate that didn’t have to be reinvented. They looked at the [Neurochem] data and decided they could work with it.”
Tolar said Alzheon, which has raised a total of about $20 million to date from angel investors and venture capital firms, decided to license the compound from Neurochem in part because many Big Pharma companies, with little to show from expensive clinical trials of would-be therapies, appeared to be scaling back on their Alzheimer’s research and development.
Alzheon’s scientists have been able to advance the study of the drug candidate, he said, by narrowing the patient population to a subgroup that could most benefit and by altering the formulation and dosing to increase its effectiveness.
“Amyloid is the best understood and validated target in Alzheimer’s research,” Tolar said. “We have a drug that targets amyloid and we have a very good understanding of how to apply it.”Robert Weisman can be reached at email@example.com. Follow him on Twitter @GlobeRobW.