Vertex gets NDA going for CF triple combo; Disarm woos rare disease expert Alvin Shih as CEO
→ Vertex’s has officially set sail with its latest cystic fibrosis triple combo, filing an NDA to the FDA. Armed with a breakthrough therapy designation, the regimen comprises ivacaftor and tezacaftor — elements of the biotech’s other CF drugs Kalydeco and Symdeko — as well as the novel compound elexacaftor (VX-445). An approval can arrive as soon as March 2020 assuming the priority review request is granted, Jefferies’ Michael Yee notes, adding that the new drug could contribute at least $6 billion in peak sales as it taps into a new population of patients with one F508del mutation and one minimal function mutation.
→ Five months after leaving Vivek Ramaswamy’s rare disease offshoot Enzyvant, Alvin Shih has landed his next CEO job at Disarm Therapeutics. Incubated out of Atlas Venture’s Cambridge, MA hatching grounds, the biotech is zeroing in on SARM1 in hopes of disrupting the process of axonal degeneration. Shih’s experience with rare neurological diseases preps him for the challenge of applying this technology to a broad range of diseases including multiple sclerosis and ALS to peripheral neuropathies, Disarm said.