The following excerpt is from the company's SEC filing.
Completion of Enrollment in Phase 2 Study of Cavosonstat for Treatment of
Cystic Fibrosis Achieved During the Quarter
BOULDER, Colo., November 7, 2016
Nivalis Therapeutics, Inc. (NASDAQ: NVLS), a clinical-stage pharmaceutical company focused on treating people with cystic fibrosis (CF), today reported financial results and recent business highlights for the third quarter 2016. Nivalis continues to advance the clinical development of cavosonstat (N91115), a novel stabilizer of the cystic fibrosis transmembrane conductance regulator protein, or CFTR.
“Our cavosonstat Phase 2 development programs continue to progress on schedule with data due by the end of this year for our add-on to Orkambi™ study and in the first quarter of 2017 for our add-on to Kalydeco™ study,” said Jon Congleton, president and chief executive officer of Nivalis. “We continue to believe that cystic fibrosis requires multiple mechanisms to achieve optimal outcomes for people managing this disease. The cavosonstat add-on to Orkambi study will produce the first Phase 2 triple therapy data set aimed at validating this belief,” Congleton added.
Third Quarter Financial Results
For the third quarter ended September 30, 2016, Nivalis reported a net loss of $7.4 million, or ($0.48) per share, compared to a net loss of $6.1 million, or ($0.39) per share, in the same quarter of the prior year. The increased loss was primarily the result of higher costs associated with progressing cavosonstat in two Phase 2 clinical studies, including completing enrollment, as planned, in the study adding cavosonstat to Orkambi.
Cash used in operating activities was $7.5 million during the third quarter and $21.1 million for the nine months year to date. As of September 30, 2016, Nivalis has $66.2 million in cash and marketable securities. Nivalis has no outstanding debt and there are 15.5 million shares of common stock issued and outstanding.
Recent Business Highlights and Upcoming Milestones
early July, the Company completed enrollment of the Phase 2 clinical study in which cavosonstat is being evaluated for the treatment of CF patients, who have two copies of the
mutation, in combination with Orkambi. The 16-week (including a 4-week follow-up), double-blind, randomized, placebo-controlled, parallel group study is designed to investigate the efficacy and safety of two doses of cavosonstat, 200 mg and 400 mg twice daily, compared to placebo when added to Orkambi. A total of 138 patients were enrolled at 46 sites in the U.S., and topline results are expected by the end of this year. More information on this Phase 2 study is available on ClinicalTrials.gov, reference Identifier:
In May 2016, Nivalis also began dosing patients in a second Phase 2 trial to evaluate the effect of cavosonstat in patients who have one copy of the
mutation and a second mutation that results in a gating defect in the CFTR protein. This proof-of-concept study is intended to evaluate the efficacy and safety of cavosonstat, 400mg twice daily, in adult patients who have these mutations and who are being treated with Kalydeco. This multicenter study will randomize approximately 20 CF patients to either cavosonstat with Kalydeco or placebo with Kalydeco. The primary efficacy endpoint is the within-group absolute change from baseline in ppFEV1 in the cavosonstat treatment group. Data is expected in the first quarter of 2017. More information on this Phase 2 study is available on ClinicalTrials.gov, reference Identifier:
This month, the Company announced that Cynthia Smith, chief commercial officer (CCO) of ZS Pharma, Inc., a subsidiary of AstraZeneca, has been appointed to the Company’s Board of Directors. Ms. Smith has more than 20 years of broad leadership experience within the healthcare industry. Since 2013, Ms. Smith has served as CCO and a member of the executive team of ZS Pharma, where she led efforts to transition the company from the
development stage to a commercial enterprise. Ms. Smith also served as vice president, market access and commercial development at Affymax, Inc., and has held various senior leadership positions in market access, corporate strategy, government relations and external affairs at Merck & Co.
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.
Cavosonstat 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 and all other major markets, and has composition of matter patent protection in the United States 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
CF 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 (www.cff.org). 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
This 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 for its clinical trials, 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.
Nivalis Therapeutics, Inc.
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The above information was disclosed in a filing to the SEC. To see the filing, click here.
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