FDA's Office Of Orphan Products Development Awards PTC Therapeutics, Inc. A Grant To Support Clinical Development Of PTC124 For The Treatment Of Nonsense-Mutation-Mediated Cystic Fibrosis

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 ).

Photo: NewsCom: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGOAP Archive: http://photoarchive.ap.orgPRN Photo Desk, photodesk@prnewswire.comPTC Therapeutics, Inc.

CONTACT: Investors & Media: Jane Baj of PTC Therapeutics, Inc.,+1-908-222-7000, ext. 167, jbaj@ptcbio.com; or Robert Stanislaro of NoonanRusso, +1-212-845-4268, robert.stanislaro@eurorscg.com; Patients, Patients'Families, Investigators and Patient Organizations: Kerri Donnelly of PTCTherapeutics, Inc., +1-908-222-7000, ext. 112, kdonnelly@ptcbio.com

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