SOUTH PLAINFIELD, NJ - December 6, 2005 - 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 initiation of a Phase 2 study of PTC124 in patients with cystic fibrosis (CF) due to a nonsense mutation. PTC124 is a novel, orally administered drug that targets nonsense mutations and is being investigated initially as a treatment for CF and Duchenne muscular dystrophy (DMD), with the potential to treat a number of other genetic disorders.
Langdon L. Miller, M.D., PTC's Chief Medical Officer, commented, "Through conduct of the Phase 2 trial, we intend to establish proof of principle for PTC124 by demonstrating production of full-length, functional cystic fibrosis transmembrane conductance regulator (CFTR) in patients with CF due to a nonsense mutation. We hope that the pharmacodynamic effects of PTC124 can eventually be translated into clinical benefit for CF patients with this life-threatening disease."
The Phase 2 clinical study is enrolling patients who have CF due to the presence of a nonsense mutation in the CFTR gene. The primary endpoint of this Phase 2 clinical study is assessment of nasal transepithelial potential difference (NPD or TEPD) as a measure of CFTR function in response to treatment with PTC124. Secondary assessments of the induction of CFTR cellular protein, pulmonary function, safety, pharmacokinetics, and compliance will also be performed.
"The initiation of Phase 2 is an important milestone in the development of PTC124 and a wonderful achievement for PTC and our multiple collaborators," stated Stuart W. Peltz, Ph.D., President and CEO of PTC. "PTC124 is a new type of treatment, aimed at the root cause of the disease, and the progress we have been able to achieve is due to the dedication and support of multiple researchers, investigators, clinicians, and patient advocacy groups."
"We are excited that this innovative therapy, that addresses the basic CF defect, has moved into the next stage of clinical trials," said Preston W. Campbell, III, M.D., Executive Vice President for Medical Affairs at the Cystic Fibrosis Foundation. "PTC has been a great partner every step of the way."
PTC has commenced recruitment for the Phase 2 study in CF at the University of Alabama, Birmingham (UAB), AL and the Denver Children's Hospital in Denver CO. Additional sites in the United States include the Johns Hopkins Hospital in Baltimore, MD; and the Rainbow Babies' and Children's Hospital in Cleveland, OH; More details regarding the design and conduct of this study is available at www.clinicaltrials.gov.
"We are thrilled to have begun the Phase 2 study of PTC124 in CF patients. It is particularly rewarding for us because Dr. David Bedwell and his team here at UAB were the first scientists to investigate the concept of readthrough of nonsense mutations as a potential treatment strategy for CF, and developed the preclinical data that support the PTC124 clinical research program in CF," commented JP Clancy, M.D., Director of Pediatric Pulmonary Medicine at UAB. "The protocol will be performed at four sites across the US, and there has already been quite a bit of interest expressed by patients from other CF centers about how they can participate. All of the centers are nearly ready to go, and we hope to have preliminary results to report in 2006."
PTC also has plans to initiate a separate CF study of PTC124 in Israel, where nonsense-mutation-mediated CF is particularly prevalent, and hopes to initiate a Phase 2 study of PTC124 in DMD within the fourth quarter of 2005.