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PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements In Chloride Channel Function In Cystic Fibrosis Patients
PTC
Therapeutics, Inc. (PTC) announced that results from a randomized
Phase 2a European study demonstrated that treatment with the
investigational drug PTC124 caused statistically significant improvements
in the chloride channel function of children with cystic fibrosis (CF)
caused by a particular genetic mutation, called a nonsense mutation.
Results from the study were presented today by Isabelle SermetGaudelus,
M.D., Ph.D., principal investigator at l'Hopital Necker-Enfants Malade. In
addition, results from an Israeli Phase 2a study demonstrated statistically
significant improvements in the function of the cystic fibrosis
transmembrane conductance regulator (CFTR) protein and a statistically
significant decrease in the frequency of cough, one of the most prominent
and burdensome CF-related symptoms. Data from the Israeli study were
presented by Eitan Kerem, M.D., principal investigator and head of the
department of pediatrics and Cystic Fibrosis Center at Hadassah University
Hospital.
PTC124 is also being featured in a plenary presentation given by
Preston Campbell, III, M.D., Executive Vice President of Medical Affairs at
the Cystic Fibrosis Foundation, on promising developments in CF research.
"We are very pleased by the data continuing to emerge from the Phase 2
clinical development program for PTC124 in CF," noted Dr. Campbell. "Our
alliance with PTC and the development of PTC124 are key elements of our
strategic work supporting novel therapeutic approaches with the potential
to modify the course of CF. We look forward to continuing our work with PTC
to move PTC124 to the next stage of development, including pivotal clinical
trials that evaluate long-term clinical benefit for CF patients."
Patients with CF lack adequate levels of the CFTR protein, a chloride
channel which is required for normal function of the lung, pancreas, liver
and other organs. Nonsense mutations are single-point alterations in the
genetic code that when transcribed into mRNA prematurely stop the mRNA
translation process, preventing production of a full-length, functional
protein. Patients with nonsense-mutation CF make virtually no CFTR protein
and thus often have a more severe form of CF. Nonsense mutations are
responsible for approximately 10 percent of the cases of CF worldwide.
By inducing the production of functional CFTR, PTC124 addresses one of
the underlying genetic defects responsible for CF. Earlier studies of
PTC124 in adults with CF evaluated nasal transepithelial potential
difference (TEPD) as a surrogate for the presence and activity of the CFTR
protein. Across a variety of studies, TEPD assessments showed statistically
significant improvements of mean CFTR-dependent chloride secretion in the
airways.
European Phase 2a Study Results
The multicenter, randomized, open-label Phase 2a study demonstrated
that treatment with PTC124 caused improvements in CFTR-mediated chloride
conductance and in apical expression of CFTR protein in nasal epithelium.
The study enrolled 30 children ages 6 to 18 years to evaluate the activity,
safety and pharmacokinetic activity of PTC124 in pediatric patients. All of
the patients had nonsense-mutation CF, pathological lung infection, and
CF-induced pancreatic insufficiency. In a cross-over design testing 14 days
of PTC124 at two dose levels, patients were randomized to receive either
the lower or higher dose first. PTC124 treatment across both dose levels
was associated with a statistically significant (p=0.03) increase in the
proportion of epithelial cells showing cell surface CFTR protein. PTC124
also induced significantly improved CFTR-mediated chloride channel activity
as measured by nasal potential difference, with 40 percent of patients
achieving values in the normal range during PTC124 administration. The
study demonstrated improvements in patients with multiple nonsense mutation
types and indicated a similar PTC124 pharmacokinetic profile as that seen
in previous studies in adults. PTC124 was generally well tolerated in this
study and compliance was greater than or equal to 93 percent.
"We are encouraged by the results of this study showing PTC124 to be as
pharmacologically active and well tolerated in children with CF as it is in
adults," said Dr. Sermet-Gaudelus. "There is a serious need for new methods
to treat the underlying cause of CF, particularly in younger patients who
have not yet experienced irreversible disease-related lung injury. These
data in pediatric patients support inclusion of children with CF in
long-term studies of PTC124."
Israeli Phase 2a 12-Week Study Results
The Phase 2a extension study evaluated 12 weeks of oral PTC124 at two
different dose levels in 19 adults with nonsense-mutation CF who
participated in a prior short-term PTC124 Phase 2a study. Greater than 85
percent of the patients had chronic CF-related lung infection and
pancreatic insufficiency. Study results showed that treatment with PTC124
resulted in statistically significant improvements in CFTR function as
measured by nasal potential difference in both dose groups. Improvements in
lung function, including forced expiratory volume in one second (FEV1) and
forced vital capacity (FVC), were also observed. Baseline data showed that
the patients coughed a remarkable 630 times per day on average, with a
range of 320 to 1,330 coughs per day, compared to healthy individuals who
typically cough fewer than 16 times per day, according to the European
Respiratory Journal (Hsu 1994). Patients in this study experienced a mean
decrease in cough frequency of almost 200 coughs per day by the end of the
study (p95%).
In a separate presentation, Dr. Kerem provided a review of all PTC124
studies to date, including studies in patients with Duchenne muscular
dystrophy, a genetic disorder that can also be caused by a nonsense
mutation.
"We are enthusiastic about the growing body of data on PTC124, and this
study specifically, which suggests the benefits of PTC124 treatment may
increase over time," said Dr. Kerem. "The safety profile of the 12-week
administration of PTC124 in patients with CF supports further
investigations in long-term clinical trials."
"We are pleased to see PTC124 highlighted in so many presentations at
NACFC," said Langdon Miller, M.D., Chief Medical Officer of PTC. "These
data demonstrate the activity of PTC124 across a broad range of patients
and suggest that it could be a valuable option for many patients living
with CF. There is currently no available therapy to correct defective CFTR
production and function. Instead, available treatments for CF are designed
to alleviate the symptoms of the disease. We hope that PTC124 will be among
the first treatments that addresses the underlying cause of the disease."
About Cystic Fibrosis
Cystic fibrosis (CF) is a life-threatening genetic disease that causes
serious lung infections and digestive complications. According to the
Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and
children in the United States and nearly 70,000 people worldwide. There is
a commercially available genetic test to determine if a patient's CF is
caused by a nonsense mutation, and it is estimated that nonsense mutations
are the cause of CF in approximately 10 percent of patients in the United
States and Europe and over 50 percent of patients in Israel. There is
currently no available therapy to correct defective CFTR production and
function. Instead, available treatments for CF are designed to alleviate
the symptoms of the disease. These treatments include chest physical
therapy to clear the thick mucus from the lungs, antibiotics to treat lung
infections, and a mucus-thinning drug designed to reduce the number of lung
infections and improve lung function. In addition, the majority of cystic
fibrosis patients take pancreatic enzyme supplements to assist with food
absorption in digestion. There is a significant unmet medical need for
treatments that address the underlying cause of CF. More information
regarding CF is available through the Cystic Fibrosis Foundation
(http://www.cff.org).
About PTC124
PTC124 is an orally delivered, investigational new drug discovered by
PTC Therapeutics. The drug is being developed for the treatment of nonsense
mutation genetic disorders. Nonsense mutations are single-point alterations
in the genetic code that prematurely stop the mRNA translation process,
leading to production of truncated, non-functional proteins. PTC124 induces
the cellular translation machinery to read through nonsense mutations in
mRNA, inducing production of full-length, functional proteins. PTC124 has
demonstrated proof of concept in Phase 2a clinical trials. Across all
clinical studies to date, PTC124 has been generally well tolerated. PTC124
is currently in Phase 2b development with the goal of demonstrating that
increasing functional protein levels in patients with nonsense mutation
genetic disorders will safely provide clinical benefits.
PTC124 has been granted orphan drug status by the FDA and the European
Commission for the treatment of cystic fibrosis and Duchenne muscular
dystrophy due to nonsense mutations. The FDA has also granted PTC124
Subpart E designation for expedited development, evaluation, and marketing.
PTC has an exclusive collaboration with Genzyme Corporation to develop
and commercialize PTC124 outside the United States and Canada. The
development of PTC124 has also been supported by grants from the Muscular
Dystrophy Association, Parent Project Muscular Dystrophy, FDA's Office of
Orphan Products Development, the National Center for Research Resources and
notably, the Cystic Fibrosis Foundation Therapeutics, Inc. (the nonprofit
affiliate of the Cystic Fibrosis Foundation), which recently expanded
support of PTC124 to include funding up to $25 million.
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery,
development and commercialization of orally administered, proprietary,
small-molecule drugs that target post-transcriptional control processes.
Post-transcriptional control processes regulate the rate and timing of
protein production and are of central importance to proper cellular
function. PTC's internally-discovered pipeline addresses multiple
therapeutic areas, including genetic disorders, oncology and infectious
diseases. PTC has extensive knowledge of post-transcriptional control
processes and has developed proprietary technologies that it applies in its
drug discovery activities, including the Gene Expression Modulation by
Small-molecules (GEMS) technology, which has been the basis for
collaborations with leading biopharmaceutical companies such as Genzyme,
Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information,
visit the company's website http://www.ptcbio.com.
PTC Therapeutics, Inc.
http://www.ptcbio.com
PTC Announces de date care sã arate cã PTC124 Cauzele statistic semnificative îmbunãtãþiri în clor Canal Funcþia În fibroza chistica Pacienþii - PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements In Chloride Channel Function In Cystic Fibrosis Patients - articole medicale engleza - startsanatate