CFRx, scientists and entrepreneurs started to approach him with their ideas for CF treatments. But the nonprofit wasn’t an appropriate vehicle for funding those ideas—hence the new venture group.
“It’s something that has evolved out of necessity more than anything,” says Flatley, whose real estate firm is based in Braintree, MA. “People are coming to me with ideas and they need funding. In order to get these things launched, somebody needs to take that first risky step. I decided to take on that role in the CF community.”
Flatley is also continuing to provide financial support to the Cystic Fibrosis Foundation, the Bethesda, MD-based nonprofit that has a track record of stepping up and funding drug developers in need of cash for new CF therapies. The CF Foundation has given funding for drugs under development at Cambridge, MA-based companies Vertex Pharmaceuticals (NASDAQ:[[ticker:VRTX]]) and Alnara Pharmaceuticals. Flatley says his lab also provides workspace for an employee of the CF Foundation, Martin Mense, and that he sees his nonprofit and venture group as adding to the foundation’s efforts.
The Flatley Discovery Lab is focused on discovering small molecule drugs that target the underlying defect in the disease, the CFTR protein, similar to treatments in development at Vertex, says Richard Fitzpatrick, the lab’s chief scientist and operating officer. (Vertex’s lead molecule for the disease, VX-770, is in late-stage clinical trials, and the company aims to file for U.S. regulatory approval in the second half of this year. In mid-stage clinical trials the drug showed that it could improve lung function without causing serious side effects.)
Even with a great need for new therapies, there’s significant risk in trying to get drugs approved for cystic fibrosis, just like with treatments for any other disease. Last week I reported the outcry in the CF community after an FDA panel gave a negative opinion of a dietary supplement for cystic fibrosis patients that was developed in the Boston area and is now owned by the drugmaker Eli Lilly (NYSE:[[ticker:LLY]]).
With his new venture group, Flatley is poised to take on big risks and seed the next generation of drugs for this awful disease.
