– Rolling submission for suzetrigine
moderate-to-severe acute pain NDA granted by FDA; first module
submitted and on track to complete filing this quarter –
– Successful completion of end-of-phase 2 FDA
meeting for pain associated with diabetic peripheral neuropathy
(DPN); Phase 3 program to initiate in 2H 2024 –
– Breakthrough Therapy designation for pain
associated with DPN granted by FDA –
– Enrollment of Phase 2 study in patients with
painful lumbosacral radiculopathy on track to complete by end of
year –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced important advancements across its suzetrigine pain
program, which has the potential to be the first new class of
medicine for acute and neuropathic pain in more than two decades.
Suzetrigine is an oral selective NaV1.8 pain signal inhibitor
(formerly known as VX-548).
Following the positive Phase 3 results in acute pain announced
in January 2024, the Food and Drug Administration (FDA) has granted
a rolling New Drug Application (NDA) submission for suzetrigine in
moderate-to-severe acute pain. Vertex has started the rolling
submission process and is on track to complete the submission in
the second quarter of 2024. Suzetrigine was previously granted FDA
Fast Track and Breakthrough Therapy designations in
moderate-to-severe acute pain.
In neuropathic pain, Vertex released positive results from its
Phase 2 study in December 2023 and recently completed a successful
end-of-phase 2 meeting with the FDA. Vertex is now preparing to
initiate a Phase 3 pivotal program of suzetrigine in patients with
DPN in 2H 2024. In addition, the FDA recently granted suzetrigine
Breakthrough Therapy designation for the treatment of pain
associated with DPN.
The Phase 3 program will include two identical 12-week
randomized, double-blind, placebo-controlled studies evaluating the
efficacy and safety of suzetrigine (70 mg once daily) in patients
with DPN. The primary endpoint for both studies will be the change
from baseline in weekly average of daily pain intensity on the
numeric pain rating scale (NPRS) assessed at Week 12 compared to
placebo. Both studies will also include a key secondary endpoint of
change from baseline in the weekly average of daily pain intensity
on the NPRS at Week 12 compared to pregabalin. Approximately 1,100
patients are expected to enroll in each Phase 3 study. After
completing participation in the randomized controlled studies,
patients may roll over into an open-label study to evaluate the
long-term safety and effectiveness of suzetrigine in DPN.
Additionally, Vertex continues to enroll its Phase 2 study of
suzetrigine in patients with lumbosacral radiculopathy, or LSR,
which is pain caused by impairment or injury to nerve roots in the
area of the lumbar spine. The company is on track to complete
enrollment in the Phase 2 LSR study by the end of the year.
“Today marks a significant milestone on our journey to redefine
the treatment of pain,” said Carmen Bozic, M.D., Executive Vice
President, Global Medicines Development and Medical Affairs, and
Chief Medical Officer at Vertex. “Given the favorable benefit/risk
profile demonstrated by suzetrigine across the entire clinical
program and the positive interactions with regulators, we are
excited by the opportunity to rapidly advance suzetrigine, a new
non-opioid potential treatment, for the millions of patients
suffering from acute and peripheral neuropathic pain.”
Next Steps for the Pain Portfolio
In line with its pain portfolio serial innovation strategy,
Vertex continues to advance preclinical and clinical development of
additional NaV1.8 and NaV1.7 pain signal inhibitors, for use alone
or in combination, in acute and neuropathic pain. The company
intends to advance its next generation NaV1.8 pain signal inhibitor
VX-993 oral formulation into Phase 2 acute pain and peripheral
neuropathic pain studies later this year. Vertex also anticipates
initiating a Phase 1 study of an intravenous formulation of VX-993
later this year.
About Acute Pain
Acute pain is a disabling condition and is defined as pain
lasting less than 3 months. It is estimated that over 80 million
people are prescribed a medicine for acute pain every year in the
U.S. Due to limited treatment options, there is an unmet need in
acute pain management to improve the patient experience and reduce
the economic and societal burden.
About Peripheral Neuropathic Pain
Peripheral neuropathic pain, or PNP, is a significant area of
unmet need for patients suffering from pain. PNP is a collection of
chronic conditions including pain associated with diabetic
peripheral neuropathy (DPN), lumbosacral radiculopathy (LSR), small
fiber neuropathy and trigeminal neuralgia. DPN and LSR are two of
the largest segments within the estimated 10 million patients who
are prescribed a medicine for PNP every year in the U.S.
About Suzetrigine (VX-548)
Suzetrigine (formerly VX-548) is an investigational oral,
selective NaV1.8 pain signal inhibitor that is highly selective for
NaV1.8 relative to other NaV channels. NaV1.8 is a voltage-gated
sodium channel that plays a critical role in pain signaling in the
peripheral nervous system. NaV1.8 is a genetically validated target
for the treatment of pain, and suzetrigine has demonstrated a
favorable benefit/risk profile in three Phase 3 studies and two
Phase 2 studies in moderate-to-severe acute pain. Suzetrigine also
demonstrated positive results and a well-tolerated profile in a
Phase 2 study in patients with pain associated with diabetic
peripheral neuropathy, a type of peripheral neuropathic pain.
Vertex’s approach is to selectively inhibit NaV1.8 using small
molecules with the objective of creating a new class of pain signal
inhibitors that have the potential to provide effective relief of
pain without the limitations of currently available therapies,
including the addictive potential of opioids.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious diseases. The company has approved medicines that
treat the underlying causes of multiple chronic, life-shortening
genetic diseases — cystic fibrosis, sickle cell disease and
transfusion-dependent beta thalassemia — and continues to advance
clinical and research programs in these diseases. Vertex also has a
robust clinical pipeline of investigational therapies across a
range of modalities in other serious diseases where it has deep
insight into causal human biology, including acute and neuropathic
pain, APOL1-mediated kidney disease, autosomal dominant polycystic
kidney disease, type 1 diabetes, myotonic dystrophy type 1 and
alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in
Boston, with international headquarters in London. Additionally,
the company has research and development sites and commercial
offices in North America, Europe, Australia, Latin America and the
Middle East. Vertex is consistently recognized as one of the
industry's top places to work, including 14 consecutive years on
Science magazine's Top Employers list and one of Fortune’s 100 Best
Companies to Work For. For company updates and to learn more about
Vertex's history of innovation, visit www.vrtx.com or follow us on
LinkedIn, Facebook, Instagram, YouTube and Twitter/X.
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, statements made by Carmen Bozic,
M.D., in this press release, statements regarding Vertex’s
expectations for the rolling submission to the FDA for suzetrigine
in acute pain, including expectations to complete the submission in
the second quarter of 2024, expectations to initiate the Phase 3
program of suzetrigine in patients with DPN in the second half of
2024, plans for the design of and enrollment expectations for the
Phase 3 DPN program, plans to evaluate the long-term efficacy and
safety of suzetrigine in patients with DPN, including enrollment
expectations for this open-label study, and enrollment expectations
for the Phase 2 study in LSR, including plans to complete
enrollment in this study by the end of the year, our expectations
for the anticipated benefits of suzetrigine, our plans to continue
advancing additional NaV1.8 and NaV1.7 pain signal inhibitors in
acute and neuropathic pain, our plans to advance VX-993 oral
formulation into Phase 2 acute pain and PNP studies later this
year, and our plans to initiate a Phase 1 study of an intravenous
formulation of VX-993 later this year. 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 our clinical studies for suzetrigine may be delayed,
and that data from the company's research and development programs
may not support registration or further development of its
compounds due to safety, efficacy, and other risks listed under the
heading “Risk Factors” in Vertex's annual report and in subsequent
filings filed with the Securities and Exchange Commission and
available through the company's website at www.vrtx.com and
www.sec.gov. You should not place undue reliance on these
statements. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
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version on businesswire.com: https://www.businesswire.com/news/home/20240418505520/en/
Vertex Pharmaceuticals Incorporated Investors:
InvestorInfo@vrtx.com Susie Lisa, CFA: +1 617-341-6108 or Miroslava
Minkova: +1 617-341-6135
Media: mediainfo@vrtx.com or International: +44 20 3204
5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607
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