-Treatment with the NaV1.8 inhibitor VX-150
showed significant relief of pain in patients with small fiber
neuropathy and was generally well tolerated-
-Third positive Phase 2 proof-of-concept study
for VX-150 further validates the potential role of NaV1.8
inhibition for the treatment of multiple pain conditions-
-Vertex has initiated a Phase 2b dose-ranging
study to evaluate VX-150 for acute pain following bunionectomy
surgery and expects to advance additional NaV1.8 inhibitors into
clinical development beginning in 2019-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced positive results of a Phase 2 study of the
investigational NaV1.8 inhibitor VX-150 in patients with pain
caused by small fiber neuropathy. The study met its primary
endpoint and showed that treatment with VX-150 demonstrated
statistically significant and clinically meaningful pain reduction,
as measured by the within-group change from baseline in the weekly
average of daily pain intensity on the 11-point numeric rating
scale (NRS) at Week 6. VX-150 was generally well tolerated in this
study.
This Phase 2 study in patients with small fiber neuropathy is
the third positive proof-of-concept study for VX-150 and provides
further validation of the potential role of NaV1.8 inhibition in
the treatment of multiple pain conditions. A Phase 2b dose-ranging
study of VX-150 following bunionectomy surgery is currently ongoing
to support potential pivotal development. Additionally, the company
is advancing multiple pain molecules through late-stage preclinical
development and anticipates initiating clinical development with
the first of these molecules in 2019.
“We are excited to now have three positive proof-of-concept
studies that validate the potential role for NaV1.8 inhibitors to
treat a variety of pain conditions,” said Jeffrey Leiden, M.D.,
Ph.D., Chairman, President and Chief Executive Officer of Vertex.
“These results show a potential path for the treatment of pain at a
time when there is great clinical and societal need for new
medicines. We continue to progress VX-150 toward pivotal
development and also plan to advance additional NaV1.8 pain
medicines into clinical development beginning in 2019.”
About the Phase 2 Study in Patients with Small Fiber
Neuropathy
The data announced today were from a Phase 2, randomized,
double-blind, placebo-controlled, 6-week study that evaluated
treatment with VX-150 (n = 46) or placebo (n = 43) in patients with
chronic pain caused by small fiber neuropathy. In this study,
1250mg of VX-150 was dosed orally every 24 hours.
Efficacy Results
The study met its primary endpoint, showing a statistically
significant mean within-group change from baseline of -2.02 points
for those treated with VX-150 in the weekly average of daily pain
intensity on the 11-point NRS, as reported in the daily diary, at
Week 6 (p<0.0001). The mean within-group change from baseline
for those treated with placebo was -0.93.
A second pre-specified analysis compared those patients
randomized to VX-150 or placebo. This analysis demonstrated a
treatment difference in the mean change from baseline of -1.09 (95%
CI: -1.88 to -0.29) in the weekly average of daily pain intensity
on the 11-point NRS, as reported in the daily diary, at Week 6.
This treatment difference was observed as early as Week 1, and was
sustained through the six-week treatment period.
Data from the efficacy analyses are provided below:
Placebo
VX-150
(n=43)
(n=46)
Mean baseline NRS 5.99 6.43
Mean change
from baseline NRS at Week 6 -0.93 -2.02
Within-group
p-value 0.0017 <0.0001
Between-group treatment
difference
NA
-1.09
(95% confidence interval)
(-1.88, -0.29)
Safety Results
In this study, VX-150 was generally well tolerated. 91 percent
of patients in the VX-150 group and 81 percent of patients in the
placebo group completed treatment. Six patients discontinued
treatment due to adverse events (2 in VX-150 and 4 in placebo) and
three patients experienced serious adverse events (0 in VX-150 and
3 in placebo). The majority of adverse events were mild or
moderate. Adverse events occurred in 63 percent of patients who
received VX-150 and 56 percent of patients who received placebo.
The most common adverse event (≥10% in any treatment group) was
headache, which occurred in 24 percent of patients in the VX-150
group and 12 percent of patients in the placebo group.
Next Steps
Vertex also announced today that the U.S. Food and Drug
Administration (FDA) has granted Breakthrough Therapy designation
to VX-150 for the treatment of moderate-to-severe acute pain.
Breakthrough Therapy designation is designed to expedite the
development and review of drugs that are intended to treat a
serious condition and for which preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement
over available therapy on a clinically significant endpoint or
endpoints.
Vertex recently initiated a Phase 2b dose-ranging study
evaluating VX-150 in patients with acute pain following
bunionectomy surgery. The study is designed to evaluate multiple
oral doses of VX-150 to potentially support pivotal
development.
Vertex now has positive Phase 2 data for VX-150 in multiple pain
conditions, including chronic pain caused by osteoarthritis and
small fiber neuropathy, as well as acute pain following
bunionectomy surgery. The company continues to invest in the
discovery and development of other potential pain molecules that
target NaV1.8 and other new pain mechanisms and anticipates
initiating clinical development with the first of these molecules
in 2019.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is
now located in Boston's Innovation District. Today, the company has
research and development sites and commercial offices in the United
States, Europe, Canada, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including being named to Science magazine's Top Employers in
the life sciences ranking for nine years in a row.
For additional information and the latest updates from the
company, please visit www.vrtx.com.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995,
including, without limitation, the statements by Dr. Leiden in the
third paragraph of this press release, the statements under the
caption “Next Steps” and the statements regarding (i) the recently
initiated Phase 2b study of VX-150 and (ii) the advancement of the
multiple pain molecules through late-stage preclinical development.
While Vertex believes the forward-looking statements contained in
this press release are accurate, these forward-looking statements
represent the company's beliefs only as of the date of this press
release and there are a number of factors that could cause actual
events or results to differ materially from those indicated by such
forward-looking statements. Those risks and uncertainties include,
among other things, that data from the company's development
programs may not support registration or further development of its
compounds due to safety, efficacy or other reasons, and other risks
listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the Securities and Exchange Commission and
available through the company's website at www.vrtx.com. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470
Media:mediainfo@vrtx.comor617-341-6992
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