Alzheimer's team tackles a daunting R&D field with a new game plan

Martin Tolar is an old hand when it comes to striking deals for experimental Alzheimer's drugs. As CEO at CoMentis, he reaped a $100 million cash windfall from a 2008 licensing pact with Astellas on a beta secretase inhibitor. And before that, he worked deals for Pfizer ($PFE). Today, after wrapping a short stint as CEO of Knome, the veteran biotech player is back in the Alzheimer's drug game, launching a new company and heading into a mid-stage study of a drug that will once again tackle the amyloid beta hypothesis.

Tolar's new company, which he is founding with a group of experienced Alzheimer's hands he's worked with in the past, is called Alzheon. The CSO is John Hey, who worked with Tolar at CoMentis. And the biotech is coming out of stealth mode today shorn of any impressive numbers. Alzheon has in-licensed a Phase II-ready Alzheimer's program from Canada's Bellus Health, committing a royalty stream on any future sales. And it's backed by private investors, with no mention of the amount of capital committed so far.

Tolar's new gamble on Alzheimer's, a disease that has so far delivered a pair of snake eyes for every biopharma company that's wagered big sums on a pivotal study, will be built on one of the earlier failures.

The drug is tramiprosate, which flunked a big study back in 2009. It's been rebuilt, so to speak, so it can be delivered through the gut in such a way as it can deliver a higher dose where it's needed in the brain. Delivered in higher concentrations, Tolar's team hopes to see the treatment prevent the development of amyloid beta, the toxic protein that builds up in the brains of Alzheimer's patients. The idea is that by preventing the damaging accumulation of amyloid beta, the drug can slow or prevent the disease in patients identified early on or eventually prior to the conditions needed to develop Alzheimer's. Tolar's readying a mid-stage trial of several hundred patients that should launch next year, he says. The study of ALZ-801 will cost about $20 million. And the company hopes to build a pipeline of Alzheimer's therapies.

Tolar is acutely aware of the long line of trial failures that have put a cloud over the field. But he's betting that the now sizeable amount of data from those failures has helped identify better biomarkers and led to a much better understanding of disease pathology. Like others, he's going earlier in the disease, gambling that the FDA's increased flexibility on the definition of success will assist his little company at a time giants like Eli Lilly ($LLY) and J&J ($JNJ) have been hit with high-profile setbacks.

"There's a treasure trove of data from large studies," Tolar tells FierceBiotech. And that's one resource he can tap for free. 

- here's the press release