PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular...(ase 1 clinical trials PTC124 was general...),Health

Date:10/11/2007

ase 1 clinical trials, PTC124 was generally well tolerated, achieved target plasma concentrations that have been associated with activity in preclinical models and did not induce ribosomal read through of normal stop codons. PTC is currently conducting Phase 2 clinical trials of PTC124 in nonsense-mutation-mediated cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD).

It is estimated that 10% of the cases of CF and 13% of the cases of DMD are due to nonsense mutations. PTC believes that PTC124 is potentially applicable to a broad range of other genetic disorders in which a nonsense mutation is the cause of the disease. The FDA has granted PTC124 Fast-Track designations 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 CF and DMD by the European Commission. PTC124's development is supported by grants from the Muscular Dystrophy Association (MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), Parent Project Muscular Dystrophy (PPMD), FDA's Office of Orphan Products Development (OOPD) and by General Clinical Research Center grants from the National Center for Research Resources (NCRR).

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. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-m
'/>"/>

SOURCE PTC Therapeutics, Inc. Copyright©2007 PR Newswire. All rights reserved

2 GOOD

Page: 1 2 3 4
Related medicine technology :

1. Cell Therapeutics, Inc. Management to Discuss Todays Announcement of Interim Pixantrone Study Results
2. Halozyme Therapeutics Enhanze Technology Large Protein Molecule Therapeutic Clinical Trial Results Presented at the 34th Annual Meeting of the Controlled Release Society
3. CV Therapeutics Identifies Potential Anti-Diabetic Mechanism of Action for Ranexa
4. GTC Biotherapeutics to Webcast Corporate Presentation at the C.E. Unterberg, Towbin Emerging Growth Life Opportunities Conference
5. Fat Kills Cancer: Turning Stem Cells from Fat Tissue into Personalized, Cancer-Targeted Therapeutics
6. A 4-Week Therapy with Transition Therapeutics E1-I.N.T. Leads to Sustained Reductions in Blood Glucose Levels for 6 Months Post-treatment in Type 2 Diabetes Patients
7. Clinical Trial Suggests Bone Marrow Stem Cells Are Useful for Spinal Cord Injury; PrimeCell Therapeutics Provided Pre-Clinical Study
8. Pieris Progresses Development of its Proprietary Biotherapeutics Platform: Successful Demonstration of Dual Targeting With Duocalin(R) Technology
9. Opexa Therapeutics Reports Positive Top-line Data in Phase I/II Extension Trial with Tovaxin for Multiple Sclerosis
10. Orexigen Therapeutics to Present Data on Contrave Impact on Visceral Fat and Insulin Resistance at American Diabetes Association 67th Scientific Sessions in Chicago June 23
11. Allos Therapeutics Reports Results for Phase 3 Enrich Study of Efaproxyn in Women With Brain Metastases Originating From Breast Cancer