Cystic fibrosis patients
Vertex Pharmaceuticals (VRTX) is currently involved in evaluating four triple combination regimens comprised of tezacaftor, ivacaftor, and one of the next-generation correctors such as VX-152, VX-440, VX-445, and VX-659 for cystic fibrosis (or CF) patients with either het/min or homozygous gene mutations. On July 18, 2017, the company announced positive Phase 1 and Phase 2 data for three combination regimens in CF patients with one F508del mutation and one minimal function mutation. The company demonstrated robust efficacy and tolerability for these regimens, for both two-week and four-week time frames.
Vertex Pharmaceuticals aims to initiate pivotal trials evaluating two of the triple combination regimens in the first half of 2018. The company expects to expand its addressable market size to almost 90% of the CF patient population by launching its triple combination regimens.
Vertex Pharmaceuticals accounts for 0.53% of the PowerShares QQQ’s (QQQ) total portfolio holdings.
Vertex Pharmaceuticals has amended its Phase 2 studies evaluating triple combination regimens comprised of next-generation correctors VX-659 and VX-445 to evaluate the efficacy of a combination regimen comprised of tezacaftor, a next-generation corrector, and a potentiator, VX-561. This addition to the original study may enable Vertex Pharmaceuticals to launch a once-daily combination regimen for CF patients. The company expects the triple combination regimens to give it a significant lead in the CF space, ahead of peers Pfizer (PFE), Sanofi (SNY), and Novartis (NVS).
Vertex Pharmaceuticals has announced results from its Phase 2 trial that evaluated the combination of inhaled ENaC (epithelial sodium channel) inhibitor VX-371 with Orkambi in 142 CF patients ages 12 and older who have two F508del mutations. This trial, however, failed to meet its endpoint. Vertex Pharmaceuticals is currently studying VX-371 as a monotherapy in primary ciliary dyskinesia.