Nivalis Therapeutics Announces First Patient Dosed in Second Phase 2 Study of N91115 for Treatment of Cystic Fibrosis

https://globenewswire.com/news-release/2016/05/16/840427/0/en/Nivalis-Therapeutics-Announces-First-Patient-Dosed-in-Second-Phase-2-Study-of-N91115-for-Treatment-of-Cystic-Fibrosis.html

Nivalis Therapeutics, Inc. (NASDAQ:NVLS), a clinical stage pharmaceutical company focused on treating people with cystic fibrosis (“CF”), today announced the first patient was dosed in a second Phase 2 clinical study of its lead investigational drug, N91115, a stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This proof-of-concept study will evaluate the effect of N91115 as add-on therapy to Kalydeco™ (ivacaftor) in adult patients who have one copy of the F508del mutation and a second mutation that results in a gating defect in the CFTR protein.

“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. “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.”

N91115 works through a novel mechanism of action called S-nitrosoglutathione reductase (GSNOR) inhibition that is presumed to modulate the unstable and defective CFTR protein responsible for CF. GSNOR inhibition restores GSNO levels, thereby modifying the chaperones responsible for CFTR protein degradation. In preclinical testing, this stabilizing effect was shown to increase and prolong the function of the CFTR protein and may lead to an increase in net chloride secretion. This effect is both complementary and agnostic to other CFTR modulators, like Orkambi™.

Study Design
The 12-week, double-blind, randomized, placebo-controlled, parallel group study is designed to investigate the efficacy and safety of 400mg of N91115 administered twice daily in adult patients with CF who are heterozygous for F508del-CFTR and a gating mutation that is approved for treatment with Kalydeco. This multicenter study will randomize approximately 20 CF patients to either N91115 with Kalydeco or placebo with Kalydeco.

The primary efficacy endpoint is the within group absolute change from baseline in percent predicted FEV1 in the N91115 treatment group. The expected completion date is the first half of 2017. For more information on this study, please visit ClinicalTrials.gov and reference Identifier: NCT02724527.N91115.

About Nivalis Therapeutics, Inc.
Nivalis Therapeutics, Inc. (http://www.nivalis.com) is a clinical-stage pharmaceutical company committed to the discovery, development and commercialization of therapeutics for people with CF. In addition to developing innovative solutions intended to extend and improve the lives of people with CF, Nivalis plans to utilize its proprietary S-nitrosoglutathione reductase (GSNOR) inhibitor portfolio to develop therapeutics for other diseases.

About N91115
N91115 works through a novel mechanism of action called GSNOR inhibition that is presumed to modulate the unstable and defective CFTR protein responsible for CF. GSNOR inhibition restores GSNO levels thereby modifying the chaperones responsible for CFTR protein degradation. This stabilizing effect has been shown to increase and prolong the function of the CFTR chloride channel and increase net chloride secretion in preclinical experiments. Nivalis discovered and owns exclusive rights to N91115 in the United States (U.S.) and all other major markets, including U.S. composition of matter patent protection until at least 2031. N91115 was granted Orphan Drug and Fast Track designations by the FDA earlier this year.  Nivalis Therapeutics has completed clinical studies with N91115, including a Phase 1a dose-escalation safety study in healthy volunteers, and a Phase 1b safety study in people with CF who have two copies of the F508del-CFTR mutation. In preclinical studies, N91115 has been shown to increase the function of F508del-CFTR, the mutant protein that is estimated to be present in approximately 86 percent of people with CF in the United States and Europe.

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