Clinical trial flops have become the norm for Alzheimer’s disease treatments, despite the billions put into research for the memory-robbing disorder. But a new Lexington, MA-based startup called Alzheon sees opportunity in all of those high-profile failures of the past. Its belief is that these failures have left behind a treasure trove of clinical data, and stalled drug candidates, that can be plucked, refined, and developed to work for more specific groups of patients.
Alzheon officially emerged from stealth today, backed by an undisclosed initial seed funding from two unnamed private investors.
“We’re taking on assets that are mature, but are usually in hands where it’s just not the right fit,” says Alzheon president and CEO Martin Tolar.
Alzheon is starting up with one such drug already in its portfolio. It in-licensed a compound called ALZ-801 (previously BLU8499) from Quebec-based Bellus Health that is the successor to an earlier Alzheimer’s drug that failed a big clinical trial in 2007. Alzheon didn’t disclose the financial terms of the deal, other than to say Bellus stands to receive a royalty stream on potential sales of the drug in the future, should things pan out. But Alzheon will now develop ALZ-801, believing it can design a trial program that targets the patients that are most likely to respond to it. And Tolar says the company has enough cash in the bank to add a few more programs as well.
Tolar is a neuroscientist by training and a Pfizer veteran that has worked on a number of Alzheimer’s drug programs. He and Alzheon chief scientific officer John Hey, for instance, helped develop an Alzheimer’s drug candidate for CoMentis that led to a big licensing deal with Astellas Pharma in 2009. Tolar later had stints leading Wellesley, MA-based startup NormOxys, and most recently Cambridge, MA-based genomics startup Knome. He says he always had his eye on coming back to drug development—and specifically, Alzheimer’s. So he stayed in contact with the teams he worked with at Pfizer and CoMentis, and came up with the idea for Alzheon. Tolar left his post at Knome, staying for just about a year and a half, and Alzheon was incorporated in August. Hey joined on as CSO, and former Pfizer coworker Mark Versavel came on as chief medical officer.
“This is a team that’s been together for a long time,” Tolar says. “We jelled before founding this. We felt we could provide a new approach to [Alzheimer’s drug development],”
Through that approach, Alzheon isn’t starting from scratch. Rather, it is targeting stalled drug programs that have been tested already in clinical trials. The drugs have already been shown to have good safety profiles, meaning they can quickly be advanced into studies that show whether they’re any good.
“The main criteria is to have clinical data in patients that could convince us that there is a signal here, that there is a population or subpopulation that would benefit from treatment, and that there is a trial design where we can, practically [speaking], make it happen,” Tolar says. “This tremendously reduces the risk, and allows us to run much smarter trials.”
Alzheon’s first foray into this plan is with ALZ-801. The compound is
