Nivalis Therapeutics, Inc., announced the first dosing of a cystic fibrosis (CF) patient enrolled in its second Phase 2 clinical trial evaluating the drug N91115, a stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein that is defective in people with the disease.
Study investigators are evaluating the efficacy of N91115 as an add-on therapy to Kalydeco (ivacaftor) in adult patients with one copy of the F508del mutation and another mutation that results in a gating defect in the CFTR protein.
N91115 is an investigational compound that works through a novel mechanism, S-nitrosoglutathione reductase (GSNOR) inhibition. The mechanism is designed to restore GSNO levels, modifying proteins responsible for CFTR protein degradation. It has been shown in preclinical studies to modulate and prolong function of the CFTR chloride channel.
The 12-week, randomized, placebo-controlled study (NCT02724527) is examining the efficacy and safety of 400 mg of N91115 administered twice daily in adult patients with CF and the specific mutations described above. About 20 enrolled CF patients will be randomized to either N91115 with Kalydeco or placebo with Kalydeco. The trial’s primary efficacy endpoint is the absolute change from baseline in forced expiratory volume in one second (FEV1, a measure of lung function), and it is expected to conclude in April 2017.
“The initiation of our second Phase 2 study sets us on the path to demonstrate that a multi-mechanism approach that includes N91115 as a novel stabilizer of the CFTR protein may help CF patients already being treated with Kalydeco achieve better clinical outcomes,” said Jon Congleton, president and chief executive officer of Nivalis, in a press release. “We hope to observe a positive impact of N91115 on lung function in this trial, and add to the important research targeted at increasing treatment options that address F508del, the most common mutation of the CFTR gene. We look forward to the results of this study in the first half of 2017.”
More information about the clinical trial, taking place at 10 sites across the U.S., is available through this link.
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