- October 21, 2018
- Posted by: PharmaScroll
- Category:
- Phase 2 results of VX-659 and VX-445 triple combination regimens concurrently published in The New England Journal of Medicine
Vertex Pharmaceuticals at the 32ndNorth American Cystic Fibrosis Conference presented Phase 2 data evaluating clinical safety and efficacy as well as in vitro data of two triple combination regimens (VX-659, tezacaftor and ivacaftor; VX-445, tezacaftor and ivacaftor) in patients with one F508del mutation and one minimal function mutation (F508del/Min) and in patients with two F508del mutations (F508del/F508del), as well as in once-daily triple combination regimens (VX-659, tezacaftor and VX-561; VX-445, tezacaftor and VX-561) in F508del/Min patients.
“The data we are presenting at this year’s Conference reinforce our belief that treating the underlying cause of CF early in life may modify the course of this disease, and demonstrate the rapid progress we are making toward our goal of developing a single medicine that will treat the underlying cause of CF in up to 90 percent of people with this devastating disease,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex.
VX-659/Tezacaftor/Ivacaftor – Phase 2 Triple Combination Results
This Phase 2 randomized, double-blind study assessed VX-659 in combination with tezacaftor and ivacaftor for 4 weeks in people with CF ages 18 and older with either F508del/Min or F508del/F508del mutations, and in combination with tezacaftor and VX-561 in patients with CF ages 18 and older with F508del/Min mutations. Primary endpoints included safety and tolerability, as well as efficacy measured by mean absolute change in percent predicted forced expiratory volume in one second (ppFEV1)from baseline. Secondary endpoints included change in sweat chloride (SwCl) and Cystic Fibrosis Questionnaire-Revised Respiratory Domain score (CFQ-R RD).
The study met its primary endpoint and showed that treatment with the triple combination regimen resulted in statistically significant and clinically meaningful increases in lung function, as well as improvements in sweat chloride and CFQ-R RD. The improvements in the F508del/F508del patients were observed when VX-659 was added in people already receiving tezacaftor/ivacaftor. Most adverse events (AEs) were mild to moderate in severity. The most common AEs in this group were cough, infective pulmonary exacerbation of cystic fibrosis, headache, oropharyngeal pain and sputum increased.
The Phase 3 ECLIPSE clinical trial program assessing VX-659/tezacaftor/ivacaftor in people with CF with F508del/Min, who today don’t have a medicine to treat the underlying cause of their disease, or F508del/F508del mutations is currently ongoing.
“Since the discovery of CFTR modulators, we have envisioned highly effective CFTR modulation therapy that could modify the progression of the disease for all CF patients,” said Steven M. Rowe, M.D., M.S.P.H., co-chair of Vertex’s Triple Combination Steering Committee and Director of the Cystic Fibrosis Research Center at the University of Alabama at Birmingham. “These impressive results showing marked improvements in lung function and a substantial reduction in sweat chloride in patients with one or two F508del mutations demonstrate we are an important step closer towards achieving that goal.”
VX-445/Tezacaftor/Ivacaftor – Phase 2 Triple Combination Results
This Phase 2, randomized, double-blind study assessed VX-445 in combination with tezacaftor and ivacaftor for 4 weeks in people with CF ages 18 and older with F508del/Min or F508del/F508del mutations, and in combination with tezacaftor and VX-561 in patients with CF ages 18 and older with F508del/Min mutations. Primary endpoints included safety and tolerability, as well as efficacy measured by mean absolute change in ppFEV1 from baseline. Secondary endpoints included change in SwCl and CFQ-R RD.
The study met its primary endpoint and showed that treatment with the triple combination regimen resulted in statistically significant and clinically meaningful increases in lung function, as well as improvements in sweat chloride and CFQ-R RD. The improvements in the F508del/F508del patients were observed when VX-445 was added in people already receiving tezacaftor/ivacaftor. Most adverse events (AEs) were mild to moderate in severity. The most common AEs include cough, sputum increased, infective pulmonary exacerbation of cystic fibrosis, hemoptysis and pyrexia.
The Phase 3 AURORA clinical trial program assessing VX-445/tezacaftor/ivacaftor in people with CF with F508del/Min, who today don’t have a medicine to treat the underlying cause of their disease, or F508del/F508del mutations is ongoing.
“The results from these two studies are truly exciting because they represent the potential for these regimens to provide significant clinical benefits for even more people with CF,” said Jennifer Taylor-Cousar, M.D., co-chair of Vertex’s Triple Combination Steering Committee and Associate Professor, Departments of Medicine and Pediatrics, Pulmonary Divisions, Medical Director of Clinical Research Services and Co-Director and Director of the CF Therapeutics Development Network, Adult CF Program, National Jewish Health, Colorado.
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News Source: Vertex Pharmaceuticals website
Image Source: https://www.glassdoor.co.in/Photos/Vertex-Pharmaceuticals-Office-Photos-IMG466489.htm