SOUTH PLAINFIELD, N.J., Dec. 6 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery, development, and
commercialization of small-molecule drugs targeting post-transcriptional
control mechanisms, today announced the company has been awarded a two-year
grant from the Office of Orphan Products Development (OOPD) of the U.S. Food
and Drug Administration (FDA). The FDA grant will be used towards the funding
of the patient-related costs of a U.S.-based Phase 2 trial evaluating PTC124
as a therapy for nonsense-mutation-mediated cystic fibrosis (CF). The OOPD
funds clinical research to accelerate the development of products that
demonstrate promise to treat rare diseases.
The primary objective of the Phase 2 study will be to assess the activity
of PTC124 in restoring the production and function of the cystic fibrosis
transmembrane regulator (CFTR) chloride channel that is abnormal in patients
with CF. CFTR function will be assessed non-invasively by measurement of
nasal potential difference (NPD). Secondary endpoints will include other
measures of biological and clinical activity, evaluation of drug safety,
determination of drug levels in the blood, and assessment of patient
compliance.
"We are honored to receive this important grant from the FDA as we
continue to evaluate PTC124 as a potential treatment for
nonsense-mutation-mediated CF," stated Stuart Peltz, Ph.D., President and CEO
of PTC Therapeutics. "The award of this grant demonstrates the scientific
merit of our development program for PTC124 as a potential treatment for CF."
Dr. Langdon Miller, PTC's Chief Medical Officer, commented: "We are delighted
to receive this peer-reviewed competitive award from the FDA. The orphan drug
grant further supports PTC's commitment to addressing orphan diseases with
high unmet medical need."
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery, development,
and commercialization of small-molecule drugs targeting post-transcriptional
control mechanisms. Post-transcriptional control processes are the sequence
of events in the cell that ultimately regulate the rate and timing of all
protein production. PTC's compounds alter these processes by selectively
modulating how RNA is used to produce proteins. By applying this approach,
PTC has advanced its drug discovery programs rapidly from targets to
preclinical and clinical drug candidates, building a robust pipeline across
genetic disorders, oncology, and infectious diseases.
About The Office of Orphan Products Development
The Office of Orphan Products Development (OOPD) has been dedicated to
promoting the development of products that demonstrate promise for the
diagnosis and/or treatment of rare diseases or conditions since it was created
in 1982. OOPD interacts with the medical and research communities,
professional organizations, academia, and the pharmaceutical industry, as well
as rare disease groups. The OOPD administers the major provisions of the
Orphan Drug Act (ODA) which provide incentives for sponsors to develop
products for rare diseases. The ODA has been very successful -- more than 200
drugs and biological products for rare diseases have been brought to market
since 1983. In contrast, the decade prior to 1983 saw fewer than ten such
products come to market. In addition, the OOPD administers the Orphan
Products Grants Program which provides funding for clinical research in rare
diseases.
About PTC124
PTC124 represents a first-in-class, orally delivered investigational new
drug for the treatment of genetic disorders due to nonsense mutations.
Nonsense mutations are single-point alterations in the genetic code that
prematurely halt the translation process, producing a shortened,
non-functional protein. In pre-clinical trials, PTC124 allowed the cellular
machinery to bypass the nonsense mutation, continue the translation process,
and thereby restore the production of a full-length, functional protein.
PTC124 has demonstrated the ability to restore full-length functional protein
in preclinical genetic disease models harboring nonsense mutations. In
Phase 1 clinical studies, PTC124 was generally well tolerated, achieved target
plasma concentrations that have been associated with activity in preclinical
models, and did not induce ribosomal readthrough of normal stop codons.
Pharmacokinetic modeling of the Phase 1 results has allowed development of a
dosing regimen for the Phase 2 studies in cystic fibrosis (CF) and Duchenne
muscular dystrophy (DMD). It is estimated that 10% of the cases of CF and 15%
of the cases of DMD are due to nonsense mutations. In addition to CF and DMD,
other potential indications include hemophilia, neurofibromatosis, retinitis
pigmentosa, epidermolysis bullosa, and lysosomal storage disorders. PTC124
may represent a unique opportunity to use a single small-molecule drug to
address chronic and life-threatening diseases of high unmet medical need. The
FDA has granted PTC124 fast track designation for the treatment of CF and
orphan drug designations for the treatment of CF and DMD due to nonsense
mutations. PTC124 has also been granted orphan drug status for the treatment
of DMD and CF by the Committee for Orphan Medicinal Products (COMP) of the
European Medicines Agency (EMEA). PTC124's development is also supported by
grants from the Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) and the
Muscular Dystrophy Association (MDA), and by General Clinical Research Center
grants from the National Center for Research Resources (NCRR).
About Cystic Fibrosis (CF)
CF is a life-threatening, genetic disease affecting approximately 70,000
people worldwide. A defective gene coding the cystic fibrosis transmembrane
conductance regulator (CFTR) protein causes the body to produce abnormally
thick, sticky mucus that leads to chronic lung-infections and impairs
digestion. It is estimated that approximately ten percent of CF patients have
the disease due to a nonsense mutation. More information regarding CF is
available through the Cystic Fibrosis Foundation (http://www.cff.org ).
SOURCE PTC Therapeutics, Inc.
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CONTACT:
Investors & Media: Jane Baj of PTC
Therapeutics, Inc., +1-908-222-7000, ext. 167, jbaj@ptcbio.com;
or Robert Stanislaro of Noonan Russo, +1-212-845-4268,
robert.stanislaro@eurorscg.com; Patients, Patients' Families,
Investigators and Patient Organizations: Kerri Donnelly of PTC
Therapeutics, Inc., +1-908-222-7000, ext. 112,
kdonnelly@ptcbio.com
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