Inhalable gene therapy meets Phase II endpoint in CF

Data published Thursday in The Lancet Respiratory Medicine from a Phase II study of pGM169/GL67A to treat cystic fibrosis showed that the inhalable gene therapy led to a significant benefit in lung function. The U.K. Cystic Fibrosis Gene Therapy Consortium conducted the trial, which was funded by a partnership between the U.K.'s Medical Research Council and National Institute for Health Research.

The study's primary endpoint was relative percent change after 12 months in predicted forced expiratory volume in one second (FEV1) in patients receiving monthly doses of pGM169/GL67A vs. placebo. The primary analysis was per protocol; in that 116-patient population, the treatment group showed a significant improvement of 3.7% (95% CI: 0.1, 7.3, p=0.046) vs. placebo, meeting the endpoint. The treatment group had a relative FEV1 change of -0.4% (95% CI: -2.8, 2.1), while the placebo group had -4% (95% CI: -6.6, -1.4). ...