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PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements in Chloride Channel Function in Cystic Fibrosis Patients
Date:10/24/2008

al 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-media
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SOURCE PTC Therapeutics, Inc.
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