Nivalis Therapeutics (NVLS) Announces Cavosonstat Phase 2 in CF Missed Primary Endpoint

November 28, 2016 4:06 PM EST
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Nivalis Therapeutics, Inc. (Nasdaq: NVLS) announced topline results from the Company's Phase 2 trial evaluating the efficacy and safety of two doses of cavosonstat, 200 mg and 400 mg, in adult patients with CF who had two copies of the F508del-CFTR mutation and were being treated with Orkambi™. There were no dose limiting toxicities and cavosonstat was well tolerated at all doses in the trial. The trial failed, however, to demonstrate benefit in absolute change in percent predicted FEV1, the trial’s primary endpoint, or in sweat chloride reduction at 12 weeks.

“While we are disappointed in the outcome of this trial, we plan to continue to investigate the therapeutic potential of cavosonstat and our S-nitrosoglutathione reductase (GSNOR) inhibitor portfolio to determine next steps,” said Jon Congleton, president and chief executive officer of Nivalis.

Summary of Key DataThe data announced today are from a Phase 2, double-blind, randomized, placebo-controlled, parallel-group trial that evaluated the efficacy and safety of two doses of cavosonstat administered twice daily (BID) in adult patients with CF who were homozygous for the F508del-CFTR mutation and being treated with Orkambi. The trial included a total of 138 adults who received treatment with cavosonstat (200 mg) with Orkambi (n=44), cavosonstat (400 mg) with Orkambi (n=48) and placebo with Orkambi (n=46) for 16 weeks. The trial included a 4-week withdrawal and follow-up period once patients had completed 12-weeks of dosing.

The primary endpoint of the trial was change in absolute percent predicted FEV1 from baseline to week 12. This and key secondary endpoints are shown in the table below.

Primary and Key Secondary Endpoints at Week 12
Placebo(N=46) 200 mg BID(N=44) 400 mg BID(N=48) Pooled Active(N=92)
Absolute Change in FEV1 (% predicted) (Within group p-value) 0.97(0.36) 0.39(0.72) 0.35(0.73) 0.37(0.62)
Relative Change in FEV1 (% predicted) (Within group p-value) 1.87(0.31) 0.66(0.72) 1.11(0.53) 0.91(0.48)
Absolute Change in Sweat Chloride (mmol/L) (Within group p-value) -2.3(0.16) -1.2(0.46) -0.6(0.69) -0.8(0.44)
Absolute Change in CFQ-R respiratory domain (Within group p-value) -3.03(0.24) -3.15(0.23) 3.16(0.21) 0.16(0.93)
Absolute change in BMI (kg/m2) (Within group p-value) -0.09(0.39) 0.17(0.09) 0.17(0.08) 0.17(0.02)

“We would like to express our sincere gratitude to everyone who participated in this trial, including the patients, their families, the trial investigators and our employees,” said Dave Rodman, chief medical officer and executive vice president of discovery at Nivalis. “Although we did not meet the primary endpoint, these data help inform the overall body of CF research, and we remain dedicated to completing our current clinical CF research program.”

Conference Call DetailsNivalis will host a conference call today at 5:00 pm Eastern Time to discuss the results and provide an update on the cavosonstat development program. The call can be accessed by dialing 877-407-2987 for callers in the United States and Canada and 201-378-4918 for international callers. The conference call will also be webcast live and can be accessed on the “Investors” section of the Nivalis website, www.nivalis.com. The webcast will be available on Nivalis’ website for 90 days.



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