Boston-based biopharma company Proteostasis Therapeutics has dosed the first patient in a phase 2 clinical trial of its cystic fibrosis transmembrane conductance regulator (CFTR) modulator combinations in F508del homozygous and heterozygous cystic fibrosis patients.
The 28-day phase 2 study has been initiated after getting positive results from the 14-day phase 1 clinical studies of the company’s doublet (PTI-808 and PTI-801) and triplet (PTI-808, PTI-801 and PTI-428) combinations.
According to Proteostasis Therapeutics, previous studies showed a favorable safety and tolerability profile for the combinations, and also statistically significant improvement in percent predicted FEV1 (ppFEV1) and sweat chloride concentration that was found to be superior to the present CFTR modulator standard of care for F508del homozygous patients.
The ongoing phase 2 cystic fibrosis trial will evaluate efficacy over a longer duration and in additional genotypes including patients who are heterozygous for the F508del mutation with the doublet and triplet combinations of Proteostasis Therapeutics.
The mid-stage clinical trial will be a randomized, placebo-controlled study, that is likely to have up to 30 F508del homozygous patients and up to 30 F508del heterozygous patients. Endpoints of the phase 2 cystic fibrosis trial are safety, changes in sweat chloride concentration and variations in ppFEV1. Results from the mid-stage trial are likely to be released in Q1 2020.
Geoffrey Gilmartin – Chief Medical Officer of Proteostasis Therapeutics said: “Despite the inclusion of CF subjects colonized with bacteria associated with a more rapid decline in lung function, our doublet and triplet studies have delivered a compelling signal of ppFEV1improvement.
“By focusing on European centers, our next stage in development will target the recruitment of patients comparable to those used in other CFTR modulator combination studies which, together with optimal dose levels and longer treatment duration, could potentially further enhance the magnitude of pulmonary benefit.”
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