Nivalis Therapeutics Announces Results from Phase 2 Clinical Trial of Cavosonstat for Treatment of Cystic Fibrosis
November 28 2016 - 4:05PM
Nivalis Therapeutics, Inc. (NASDAQ:NVLS), a clinical stage
pharmaceutical company focused on treating people with cystic
fibrosis (“CF”), today 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.
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 CavosonstatCavosonstat works through a
novel mechanism of action called GSNOR inhibition. 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 in
CF by the FDA earlier this year. Nivalis has completed clinical
studies with cavosonstat, including a Phase 1a dose-escalation
safety study in healthy volunteers, a Phase 1b safety study and a
phase 2 trial, added to Orkambi™ in people with CF who have two
copies of the F508del-CFTR mutation.
About Cystic FibrosisCF 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 StatementsThis press release
contains “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements regarding plans to complete Nivalis’ CF research
program, plans to investigate the therapeutic potential of
cavosonstat and our other GSNOR inhibitors, 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 ability of Nivalis to fund continued development
of cavosonstat, uncertainty in the potential for cavosonstat or any
other GSNOR inhibitors to address other therapeutic areas, 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, 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.
Contacts:
Investor Relations
John Graziano
1-646-378-2942
jgraziano@troutgroup.com
Media Relations
Melyssa Weible
1-201-723-5805
mweible@elixirhealthpr.com
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