Biotech Entrepreneur Tony Coles Takes Aim At Parkinson's And Alzheimer's

Tony Coles, the chief executive who sold Onyx Pharmaceuticals to Amgen  for $10.4 billion in 2013, is starting a new company that will use yeast to try and discover new treatments for Parkinson’s, Alzheimer’s, and amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease.)

The new startup, Yumanity, will be based in Cambridge, Mass., and is built around technologies developed by Susan Lindquist, the former director of the MIT-affiliated Whitehead Institute for Biomedical Research. Three researchers who worked in Lindquist’s lab will serve as founding scientists for the company, which currently has five employees. Coles says he hopes to scale up to 25.

“I love a big problem and a good challenge and I think that this challenge is sized about right,” Coles says. “We’ve got 50 million people around the world suffering from these diseases, $650 billion in economic costs, and lots of families like mine that have been affected.”

Three specific technologies were created at the Lindquist lab that may allow drug researchers to search more effectively for drugs against neurodegenerative diseases. In all of these disorders, proteins that play important roles in the brain become mis-folded – they are literally misshapen. (Lindquist won the National Medal of Science for her work in how proteins fold in the brain.)

When these misfolded proteins are put into yeast cells, they die. The first step taken using the Yumanity technology is to find drug compounds that keep the yeast cells from dying. These are then put into human neurons derived from induced pluripotent stem cells created from people who have the neurodegenerative disease being studied.

If the drug seems effective in human cells, it is then put back into yeast to try and figure out what molecular target it affects. By this back-and-forth approach (yeast, then human, then yeast, then human again) it should be possible to find and understand new drugs for the disease.

The approach was honed in a trio of Science publications (one, two, three) over the past three years. Even working in an academic lab, Lindquist’s researchers have been able to screen 500,000 compounds against each of three diseases (Parkinson’s, Alzheimer’s, ALS). A bigger effort will be possible at a company, especially as drug companies may be willing to share their proprietary compound libraries as part of the process.

Coles and Lindquist met as members of the board of directors at a previous company she started, FoldRx, which was sold to Pfizer PFE +0.26%. She called Coles to ask him if he would be interested in commercializing her new work. “I couldn’t have imagined anyone who I would rather work with on this than Tony,” she says.

Despite the fact that the work is far more research-based than what he had previously done—Yumanity is a research-stage company, whereas Onyx didn’t invent compounds but bought and developed cancer drugs others had discovered – Coles took the job. He also brought on Kenneth Rhodes, a former Biogen Idec executive, to serve as Chief Scientific Officer.

Yumanity is initially being funded by the founders (Coles received more than $57 million from the Onyx deal) but a funding round is expected to close early next year. Coles says that there is also interest in large pharmaceutical firms who might partner with Yumanity.

Yumanity is still in the very early stages of research. None of its work is ready to be tested in humans. It does have one lead in Parkinson’s, and the company is conducting some research in animal models, not just in cell lines. But the potential is big. Coles says: “I think we’ve got a way to transform the way new drugs are identified and potentially the way new drugs can be discovered.”

http://health.einnews.com/article/239840679/1G1S_NrxwrBIQcEB