Nivalis Therapeutics: Ivalis Therapeutics Announces Completion Of Enrollment In Phase 2 Study

The following excerpt is from the company's SEC filing.

of N91115 for Treatment of Cystic Fibrosis

- Study

is Evaluating N91115 in Patients Homozygous for the F508del-CFTR Mutation and

Being Treated with Orkambi™

- “Cavosonstat” Approved as the Unique Non-proprietary or Generic Name for N91115

- Data Expected

Late 2016

BOULDER, Colo., July

, 2016 –


Therapeutics, Inc. (NASDAQ: NVLS), a clinical stage pharmaceutical company focused on treating people with cystic fibrosis (“CF”), today announced completion of enrollment, defined as the last patient enrolled receiving their first study dose, in the larger of the two Phase 2 clinical studies of N91115, a novel stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In this study, N91115 is being evaluated for the treatment of CF patients, who have two copies of the

mutation, when added to Orkambi™ (lumacaftor/ivacaftor). A total of 138 patients have been enrolled at 46 sites in the U.S. and topline results are expected by the end of this year.

Company also announced that the United States Adopted Names (USAN) Council has approved “cavosonstat” as the unique non-proprietary or generic name for N91115, which the Company will use going forward to refer to N91115.

Completing enrollment in this Phase 2 study represents a critical milestone in our clinical development program for cavosonstat and moves us closer to our goal of bringing our novel stabilizer to people with CF,” said Jon Congleton, president and chief executive officer of Nivalis. “We would like to thank the study participants and their families, the clinical trial sites and the CF community for their continued commitment to this effort and we look forward to the topline results of this study by the end of this year.”

Cavosonstat 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

16-week, double-blind, randomized, placebo-controlled, parallel group study is designed to investigate the efficacy and safety of two doses of cavosonstat, 200mg and 400mg, administered twice daily in adult patients with CF who are homozygous for the

mutation and being treated with Orkambi™. The study includes a 4-week withdrawal and follow-up period once patients have completed 12-weeks of dosing. The primary clinical efficacy outcome will be the absolute change from baseline in percent predicted FEV1. For more information on this study, please visit and reference Identifier NCT02589236.

About Nivalis Therapeutics, Inc.

Therapeutics, Inc. ( 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.

avosonstat 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 cavosonstat in the United States (U.S.) and all other major markets, including U.S. composition of matter patent protection until at least 2031. Cavosonstat was granted Orphan Drug and Fast Track designations by the FDA earlier this year. Nivalis Therapeutics has completed clinical studies with cavosonstat, 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

mutation. In preclinical studies, cavosonstat has been shown to increase the function of

, the mutant protein that is estimated to be present in approximately 86 percent of people with CF in the United States and Europe.

About Cystic Fibrosis

is a life-shortening genetic disease that affects an estimated 70,000 people worldwide, predominantly in the United States and Europe, according to the Cystic Fibrosis Foundation (

). CF is characterized by a defect in the chloride channel known as the “cystic fibrosis transmembrane conductance regulator,” or CFTR, and is caused by mutations in the CFTR gene.

Forward Looking Statements

press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding Nivalis’ development plans and potential opportunities, statements regarding the timing of completion of and availability of topline clinical results, and expectations that early stage clinical trials are indicative of later stage clinical trial results or will result in an approved drug. These forward-looking statements are based on management’s current expectations of future events and involve substantial risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by the forward-looking statements. These risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, including that preclinical responses may not be predictive of clinical results, delays in the timing of regulatory filings and approvals, delays in the commercialization or availability of lumacaftor/ivacaftor, and other matters that could affect the completion of the clinical development and commercial potential of the company’s product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Nivalis’ business in general, see the risk factors contained in the company’s annual report on Form 10-K filed with the Securities and Exchange Commission on March 8, 2016 and in other reports filed by Nivalis with the Securities and Exchange Commission. All information in this press release is as of the date of this release, and Nivalis undertakes no duty to update or revise this information unless required by law.


Investor Relations

John Graziano


Media Relations

Lindsay Rocco


The above information was disclosed in a filing to the SEC. To see the filing, click here.

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