Nivalis Therapeutics has announced positive data from its Phase 1b trial evaluating N91115 for cystic fibrosis.
N91115 is a first-in-class CFTR stabilizer that modulates CFTR activity through a novel mechanism of action that aims to be complementary to existing and future CFTR modulators. In Nivalis’ randomized, placebo-controlled, double-blind, parallel group clinical trial, a total of 51 adults with a diagnosis of CF were assigned at random to receive a drug placebo or N91115 at 50, 100 or 200 mg doses twice per day for a period of 28 days. Primary study endpoints were the tolerability, safety, and pharmacokinetic profile of N91115. Secondary endpoints of the study were the determination of doses for the Phase 2 study and the exploration of pharmacodynamic indicators of the disease’s biological activity. During the study, an independent Data Monitoring Committee monitored the patients’ safety.
Results from the study showed that at the examined dose range, the drug was well tolerated and is safe in adult CF patients. In terms of pharmacokinetic profile, the results revealed that N91115 completely reached the dose selection rationale and the targeted blood levels. The results also showed a tendency for a modest decline in sweat chloride at the highest dose analyzed, which may indicate a threshold effect for CFTR modulation.