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Cystic Fibrosis Canada is excited to hear that data from early stage clinical trials led by Vertex Pharmaceuticals Inc. resulted in significantly improved lung function in people with one F508del mutation and one minimal function mutation (F508del/Min).
The triple combination therapy uses a combination of ivacaftor (known as Kalydeco), Vertex’s new product tezacaftor, and a third compound to tackle the mutation of the CF gene, which is the underlying cause of cystic fibrosis (CF). This is the first data to demonstrate the potential to treat the underlying cause of CF in people with F508del/Min mutations, a severe and difficult-to-treat type of the disease.
This next generation combination therapy has the potential to be more effective than currently approved modulators and could benefit up to 90% of the CF population. Phase 2 data showed improvements in ppFEV1 of 9.7 and 12.0 percentage points for VX-152 and VX-440 in triple combination with tezacaftor and ivacaftor in F508del/Min patients. Initial data from Phase 1 study showed improvement in ppFEV1 of 9.6 percentage points with VX-659 triple combination in F508del/Min patients.
Initial Phase 2 data also showed improvements in ppFEV1 of 7.3 and 9.5 percentage points when VX-152 or VX-440 was added in people with two copies of the F508del mutation (F508del/F508del), who were already receiving tezacaftor and ivacaftor.
All 3 triple combination regimens were generally well tolerated across the studies.
CF Canada has provided millions in research funding to Vertex and has invested over $244 million in CF research, innovation and care.
CF is the most common fatal genetic disease affecting Canadian children and young adults. At present, there is no cure. CF causes various effects on the body, but mainly affects the digestive system and lungs. The degree of CF severity differs from person to person, however, the persistence and ongoing infection in the lungs, with destruction of lungs and loss of lung function, will eventually lead to death in the majority of people with CF.
“CF Canada’s mission has always been to support the CF community by, among other things, investing in leading research that could result in new treatments, and ultimately a cure for CF. Whenever new data points to the potential for improved treatment of CF, we are obviously very pleased. While it is still early on, it is our hope that we will continue to see positive results from further phase 3 and phase 4 clinical trials, which will allow us to determine the effectiveness of this treatment in larger populations.”
President and CEO, Cystic Fibrosis Canada
For more information, please consult Vertex’s Press Release