- Paul Negulescu, Fredrick Van Goor and Sabine
Hadida receive Breakthrough Prize for developing transformative
medicines for people with cystic fibrosis –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that three of its researchers have been awarded the 2024
Breakthrough Prize in Life Sciences “for developing
life-transforming drug combinations that repair the defective
chloride channel protein in patients with cystic fibrosis.”
Specifically, the award celebrates the work of Paul Negulescu,
Ph.D., Fredrick Van Goor, Ph.D., and Sabine Hadida, Ph.D., who have
worked together to lead cystic fibrosis (CF) discovery for over 20
years.
“The research teams led by Paul, Fred and Sabine discovered the
first and only medicines that address the underlying cause of
cystic fibrosis. This remarkable effort required discovery of three
novel mechanisms of action, resulting in the first disease
modifying therapies that act by restoring function to a misfolded
protein,” said David Altshuler, M.D., Ph.D. Executive Vice
President, Global Research, and Chief Scientific Officer. “This
prize recognizes the dedication and creativity of thousands of
people at Vertex spanning research, development, regulatory,
manufacturing and patient access, as well as our partners in the CF
community, who have worked tirelessly to bring these medicines to
people with CF around the world.”
The gene responsible for cystic fibrosis was discovered in 1989,
but until the work of Vertex scientists, treatment of CF addressed
only its symptoms rather than the underlying cause of the disease.
Today, Vertex’s four approved oral medicines treat CF by improving
the function of the defective CFTR protein. The first of these
medicines, KALYDECO® (ivacaftor), was approved in 2012, and today
is approved for people with CF ages 1 month and older carrying
responsive CFTR mutations. The most recently developed medicine,
TRIKAFTA®, is a triple combination therapy
(elexacaftor/tezacaftor/ivacaftor and ivacaftor) first approved in
2019 that today can treat ~90% of people with CF who have
responsive mutations.
Vertex is currently developing a next-in-class investigational
CF triple combination therapy, now in Phase 3 trials, that has the
potential for enhanced clinical benefit. The company is also in
early clinical trials in collaboration with Moderna to develop an
mRNA therapeutic designed to treat the approximately 5,000 people
with CF who do not produce any CFTR protein. If this mRNA therapy
is successful, it will be possible for all people with CF to have
medicines to treat the underlying cause of their disease.
The Breakthrough Prize in Life Sciences was founded in 2013 by
Sergey Brin, Priscilla Chan and Mark Zuckerberg, Yuri and Julia
Milner, and Anne Wojcicki. Breakthrough Prizes are also awarded
annually in the fields of Fundamental Physics and Mathematics. To
learn more, visit breakthroughprize.org.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 88,000 people globally. CF is a progressive,
multi-organ disease that affects the lungs, liver, pancreas, GI
tract, sinuses, sweat glands and reproductive tract. CF is caused
by a defective and/or missing CFTR protein resulting from certain
mutations in the CFTR gene. Children must inherit two defective
CFTR genes — one from each parent — to have CF, and these mutations
can be identified by a genetic test. While there are many different
types of CFTR mutations that can cause the disease, the vast
majority of people with CF have at least one F508del mutation. CFTR
mutations lead to CF by causing CFTR protein to be defective or by
leading to a shortage or absence of CFTR protein at the cell
surface. The defective function and/or absence of CFTR protein
results in poor flow of salt and water into and out of the cells in
a number of organs. In the lungs, this leads to the buildup of
abnormally thick, sticky mucus, chronic lung infections and
progressive lung damage that eventually leads to death for many
patients. The median age of death is in the early 30s.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor)
In people with certain types of mutations in the CFTR gene, the
CFTR protein is not processed or folded normally within the cell,
and this can prevent the CFTR protein from reaching the cell
surface and functioning properly. TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral
medicine designed to increase the quantity and function of the CFTR
protein at the cell surface. Elexacaftor and tezacaftor work
together to increase the amount of mature protein at the cell
surface. Ivacaftor, which is known as a CFTR potentiator, is
designed to facilitate the ability of CFTR proteins to transport
salt and water across the cell membrane. The combined actions of
elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus
from the airways.
About KALYDECO® (ivacaftor)
In people with certain types of mutations in the CFTR gene, the
CFTR protein at the cell surface does not function properly. Known
as a CFTR potentiator, ivacaftor is an oral medicine designed to
facilitate the ability of CFTR proteins to transport salt and water
across the cell membrane, which helps hydrate and clear mucus from
the airways. KALYDECO® (ivacaftor) was the first medicine to treat
the underlying cause of cystic fibrosis in people with specific
mutations in the CFTR gene.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a
prescription medicine used for the treatment of cystic fibrosis
(CF) in patients aged 2 years and older who have at least one copy
of the F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene or another mutation that is
responsive to treatment with TRIKAFTA. Patients should talk to
their doctor to learn if they have an indicated CF gene mutation.
It is not known if TRIKAFTA is safe and effective in children under
2 years of age.
IMPORTANT SAFETY INFORMATION
Before taking TRIKAFTA, patients should tell their doctor
about all of their medical conditions, including if they: are
allergic to TRIKAFTA or any ingredients in TRIKAFTA, have kidney
problems, have or have had liver problems, are pregnant or plan to
become pregnant because it is not known if TRIKAFTA will harm an
unborn baby, or are breastfeeding or planning to breastfeed because
it is not known if TRIKAFTA passes into breast milk.
Patients should tell their doctor about all the medicines
they take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements. TRIKAFTA may affect
the way other medicines work, and other medicines may affect how
TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when
taken with certain medicines. Patients should ask their doctor or
pharmacist for a list of these medicines if they are not sure.
Patients should especially tell their doctor if they take:
antibiotics such as rifampin or rifabutin; seizure medicines such
as phenobarbital, carbamazepine, or phenytoin; St. John’s wort;
antifungal medicines including ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; antibiotics including
telithromycin, clarithromycin, or erythromycin.
Patients should avoid food or drink that contains
grapefruit while taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
Liver damage and worsening of liver function in patients
with severe liver disease that can be serious and may require
transplantation. Liver damage has also happened in patients without
liver disease.
High liver enzymes in the blood, which is a common side
effect in patients treated with TRIKAFTA. These can be
serious and may be a sign of liver injury. The patient’s doctor
will do blood tests to check their liver before they start
TRIKAFTA, every 3 months during the first year of taking TRIKAFTA,
and every year while taking TRIKAFTA. Patients should call their
doctor right away if they have any of the following symptoms of
liver problems: pain or discomfort in the upper right stomach
(abdominal) area; yellowing of the skin or the white part of the
eyes; loss of appetite; nausea or vomiting; dark, amber-colored
urine.
Serious allergic reactions have happened to patients who
are treated with TRIKAFTA. Call your healthcare provider or go to
the emergency room right away if you have any symptoms of an
allergic reaction. Symptoms of an allergic reaction may include:
rash or hives; tightness of the chest or throat or difficulty
breathing; swelling of the face, lips and/or tongue; difficulty
swallowing; and light-headedness or dizziness.
Abnormality of the eye lens (cataract) has been noted in
some children and adolescents treated with TRIKAFTA. If the patient
is a child or adolescent, their doctor should perform eye
examinations before and during treatment with TRIKAFTA to look for
cataracts.
The most common side effects of TRIKAFTA include
headache, upper respiratory tract infection (common cold) including
stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash,
increase in liver enzymes, increase in a certain blood enzyme
called creatine phosphokinase, flu (influenza), inflamed sinuses,
and increase in blood bilirubin.
Patients should tell their doctor if they have any side effect
that bothers them or that does not go away. These are not all the
possible side effects of TRIKAFTA. For more information, patients
should ask their doctor or pharmacist.
Please click here to see the full Prescribing
Information for TRIKAFTA.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
KALYDECO® (ivacaftor)
INDICATIONS AND USAGE
KALYDECO (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 1 month and older
who have at least one mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene that is responsive to KALYDECO.
Patients should talk to their doctor to learn if they have an
indicated CF gene mutation. It is not known if KALYDECO is safe and
effective in children under 1 month of age.
IMPORTANT SAFETY INFORMATION
Before taking KALYDECO, patients should tell their doctor
about all their medical conditions, including if they: have
liver or kidney problems; are allergic to KALYDECO or any
ingredients; are pregnant or plan to become pregnant because it is
not known if KALYDECO will harm an unborn baby; and are
breastfeeding or planning to breastfeed because is not known if
KALYDECO passes into breast milk.
Patients should tell their doctor about all the medicines
they take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements. KALYDECO may affect
the way other medicines work, and other medicines may affect
how KALYDECO works. Patients should ask their doctor or pharmacist
for a list of these medicines if they are not sure. Patients should
especially tell their doctor if they take the antibiotics rifampin
or rifabutin; seizure medicines such as phenobarbital,
carbamazepine, or phenytoin; St. John’s wort; antifungal medicines
such as ketoconazole, itraconazole, posaconazole, voriconazole, or
fluconazole; or antibiotics such as telithromycin, clarithromycin,
or erythromycin.
KALYDECO can cause dizziness in some patients who take
it. If patients experience dizziness, they should not drive or
operate machines until symptoms improve.
Patients should avoid food or drink containing grapefruit
while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood, which have happened in
patients receiving KALYDECO. The patient’s doctor will do
blood tests to check their liver before starting KALYDECO, every 3
months during the first year of taking KALYDECO, and every year
while taking KALYDECO. For patients who have had high liver enzymes
in the past, the doctor may do blood tests to check the liver more
often.
Patients should call their doctor right away if they have any of
the following symptoms of liver problems: pain or discomfort in the
upper right stomach (abdominal) area; yellowing of their skin or
the white part of their eyes; loss of appetite; nausea or vomiting;
or dark, amber-colored urine.
Serious allergic reactions have happened to patients who
are treated with KALYDECO. Patients should call their healthcare
provider or go to the emergency room right away if they have
symptoms of an allergic reaction. Symptoms of an allergic reaction
may include rash or hives, tightness of the chest or throat or
difficulty breathing, and light-headedness or dizziness.
Abnormality of the eye lens (cataract), which has
happened in some children and adolescents receiving KALYDECO. The
patient’s doctor should perform eye examinations before and during
treatment with KALYDECO to look for cataracts.
The most common side effects include headache; upper
respiratory tract infection (common cold), which includes sore
throat, nasal or sinus congestion, and runny nose; stomach
(abdominal) pain; diarrhea; rash; nausea; and dizziness.
Use of KALYDECO in patients aged 1 month to less than 6 months
born from a pregnancy lasting (gestational age) less than 37 weeks
has not been evaluated.
These are not all the possible side effects of KALYDECO.
Please click here to see the full Prescribing
Information for KALYDECO.
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 multiple approved medicines
that treat the underlying cause of cystic fibrosis (CF) — a rare,
life-threatening genetic disease — and has several ongoing clinical
and research programs in CF. Beyond CF, Vertex has a robust
clinical pipeline of investigational small molecule, mRNA, cell and
genetic therapies (including gene editing) in other serious
diseases where it has deep insight into causal human biology,
including sickle cell disease, beta thalassemia, APOL1-mediated
kidney disease, acute and neuropathic pain, type 1 diabetes and
alpha-1 antitrypsin deficiency.
Founded in 1989 in Cambridge, Mass., Vertex's global
headquarters is now located in Boston's Innovation District and its
international headquarters is in London. Additionally, the company
has research and development sites and commercial offices in North
America, Europe, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including 13 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 Facebook, Twitter,
LinkedIn, YouTube and Instagram.
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, statements regarding our
plans and expectations for our pipeline, including our
next-in-class investigational CF triple combination therapy and our
mRNA therapy in collaboration with Moderna. 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 studies
may not be indicative of final clinical trial results, 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, that our development programs may
experience delays, and other risks listed under the heading “Risk
Factors” in Vertex's most recent annual report and subsequent
filings filed with the Securities and Exchange Commission at
www.sec.gov and available through the company's website at
www.vrtx.com. 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
Media: mediainfo@vrtx.com or U.S.: +1 617-341-6992 or
Heather Nichols: +1 617-839-3607
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