Therapy Areas: Hereditary Disorders
Vertex Begins Phase 3 Study of Triple Combination Regimen for People with Most Common Genetic Form of Cystic Fibrosis
5 March 2018 - - Boston, Massachusetts-based serious disease therapeutics developer Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) is initiating a Phase 3 study of VX-659, tezacaftor, and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis who have two copies of the F508del mutation, the company said.
The randomized, double-blind, controlled study will enroll approximately 100 patients ages 12 years or older. The primary endpoint of the study is the mean absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1) at week four of treatment.
The study is designed to support the submission of an application for approval in patients with two copies of the F508del mutation in the US using data from the four-week primary efficacy endpoint together with 24-week safety data generated from the recently initiated Phase 3 study in patients with one F508del mutation and one minimal function mutation.
The initiation of the Phase 3 study is based on data from a Phase 2 study that showed a mean absolute improvement in ppFEV1 of 9.7 percentage points from baseline through week four of treatment when VX-659 (400 mg) was added in people with CF who have two F508del mutations and were already receiving tezacaftor in combination with ivacaftor.
In the Phase 2 study, the VX-659 triple combination regimen was generally well tolerated, the majority of adverse events were mild to moderate in severity and there were no discontinuations due to adverse events.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
The company has research and development sites and commercial offices in the United States, Europe, Canada, and Australia.
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