Vertex Presented Successful Results for Cystic Fibrosis Treatment


Sep. 1 2017, Updated 7:38 a.m. ET

VX-152 phase two study

Vertex Pharmaceuticals (VRTX) conducted a phase two study to evaluate the safety and efficacy of VX-152 (100mg, 200mg, and 300mg) along with tezacaftor and ivacaftor in individuals with cystic fibrosis (or CF) aged at least 18 years and who have one F508del mutation and one minimal function mutation. Another group of individuals had CF with two copies of the F508del mutation. The primary goal of the phase two study was to evaluate the safety and tolerability of VX-152.

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Safety and efficacy results

Patients in the phase two trial, the triple combination regimen of VX-152 and tezacaftor/ivacaftor demonstrated good tolerance. The phase two study on CF patients with two copies of the F508del mutation is ongoing. Success in these clinical trials will strengthen Vertex Pharmaceuticals’ CF product portfolio.

Vertex Pharmaceuticals’ peers that are actively conducting research and development for CF drugs include Pfizer (PFE), PTC Therapeutics (PTCT), Proteostasis Therapeutics (PTI), Novartis, Roche, and others. The iShares Nasdaq Biotechnology ETF (IBB) invests ~4.9% of its total portfolio holdings in Vertex Pharmaceuticals.


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