Vertex Announces FDA Clearance of Investigational New Drug Application for VX-407 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
21 Marzo 2024 - 1:00PM
Business Wire
– ADPKD is the most common inherited kidney
disease, with no treatments currently available that address the
underlying cause of disease –
– Vertex to initiate a Phase 1 clinical trial
in healthy volunteers this month –
– ADPKD is Vertex’s 10th disease area in the
clinic, further expanding the company’s pipeline of potentially
transformative medicines for serious diseases –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) has
cleared the Investigational New Drug Application (IND) for VX-407,
an investigational first-in-class small molecule corrector that
targets the underlying cause of autosomal dominant polycystic
kidney disease (ADPKD) in patients with a subset of PKD1 genetic
variants. ADPKD is the most common inherited kidney disease, with
an estimated 250,000 people in the U.S. and Europe living with
ADPKD; however, there are no treatments currently available that
address the underlying causal biology of the disease.
ADPKD is a life-shortening genetic kidney disease characterized
by the growth of numerous kidney-enlarging cysts that impair kidney
function and can ultimately lead to kidney failure, requiring
dialysis or kidney transplantation, and premature death.
The majority of ADPKD cases are caused by variants in the PKD1
gene, which encodes the polycystin 1 (PC1) protein. These inherited
variants lead to a loss of PC1 function that results in cyst
growth. VX-407 is a first-in-class small molecule corrector that is
designed to target the underlying cause of ADPKD in a subset of
patients with PKD1 variants, estimated at ~25,000 (or ~10%) of the
overall ~250,000 ADPKD patient population, by restoring function to
the variant PC1 protein. Vertex plans to initiate a Phase 1
clinical trial of VX-407 in healthy volunteers this month.
“The advancement of VX-407, a first-in-class molecule for the
treatment of ADPKD, into the clinic represents another important
opportunity to transform the treatment of a serious disease,” said
Carmen Bozic, M.D., Executive Vice President, Global Medicines
Development and Medical Affairs, and Chief Medical Officer at
Vertex. “Just as our approach in cystic fibrosis allowed us to
reach more patients over time, our goal here is to serially
innovate to reach the 250,000 people suffering from ADPKD.”
About Autosomal Dominant Polycystic Kidney Disease
(ADPKD)
ADPKD is the most common inherited kidney disease and one of the
most common severe Mendelian genetic diseases, affecting
approximately 250,000 diagnosed people in the U.S. and Europe. As
an autosomal dominant disease, one affected parent can pass on the
disease to their children.
In most cases, ADPKD is caused by variants in the PKD1 and PKD2
genes, which express proteins known as polycystins. The majority of
ADPKD patients (~80%) have a variant in the PKD1 gene, resulting in
a loss of function of polycystin 1 (PC1). This leads to the
proliferation of kidney epithelial cells, increased fluid secretion
and the formation and expansion of numerous fluid-filled cysts. The
progressive cyst formation causes an increase in kidney size and
decline in kidney function. Around half of patients with ADPKD
experience kidney failure by the age of 60. Kidney cysts can also
lead to severe abdominal pain, cyst infection, blood in the urine
and kidney stones, all of which significantly impair quality of
life.
About VX-407
VX-407 is a first-in-class small molecule corrector that is
designed to treat ADPKD in patients with a subset of PKD1 variants,
estimated at ~25,000 (or ~10%) of the overall ~250,000 ADPKD
patient population, by correcting defective PC1 folding to restore
function. In doing so, the aim is to stop growth of kidney cysts
and reduce kidney volume, thereby preventing progression to kidney
failure.
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 plans to initiate
a Phase 1 clinical trial of VX-407 in healthy volunteers this
month, and estimates regarding the potential eligible patient
population. 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 risks and
uncertainties that could cause actual events or results to differ
materially from those expressed or implied by such forward-looking
statements. Those risks and uncertainties include, among other
things, 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.
(VRTX-GEN)
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Vertex Pharmaceuticals Incorporated Investors:
InvestorInfo@vrtx.com Susie Lisa, CFA: +1 617-341-6108 or
Manisha Pai: +1 617-961-1899 or Miroslava Minkova: +1
617-341-6135
Media: mediainfo@vrtx.com or U.S.: +1 617-341-6992
or Heather Nichols: +1 617-839-3607 or International: +44 20 3204
5275
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