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news.Vertex has declared that it is going to start phase III registration program for VX-770, an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, which targets the defective CFTR protein that causes cystic fibrosis (CF).
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The VX-770 registration program will consist of three clinical trials, including a primary 48 week phase III trial that is currently open to enroll patients aged 12 years and older, who carry the G551D mutation on at least one allele.
However, the other two trials will evaluate VX-770 in patients aged 6 to 11 years with the G551D mutation on at least one allele and in patients homozygous for the F508del mutation.
In the registration program, the primary endpoint for patients with the G551D mutation is forced expiratory volume in one second (FEV1), which will be measured through 24 weeks, and additional FEV1 measurements will be taken through 48 weeks as a secondary endpoint.
VX-770 came into existence as part of collaboration with Cystic Fibrosis Foundation Therapeutics (CFFT) to develop CFTR modulators.
Robert J. Beall, Ph.D., President and CEO, Cystic Fibrosis Foundation, said: The initiation of the VX-770 Phase 3 registration program is a major advancement in our efforts to bring forward new therapies aimed at treating the underlying cause of CF.