The results suggest that PTC124 can restore function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in airway cells and significantly reduce blood neutrophil counts that are a hallmark of cystic fibrosis (CF). Patients with CF lack the CFTR protein, a chloride channel that maintains proper hydration of epithelial cells in the lung, pancreas, and liver.
Across the two studies, at two PTC124 dose levels tested, assessments showed statistically significant improvements of mean CFTR-dependent chloride secretion in the airways.
“These results are very exciting because they provide the first indication that an oral therapy may address the underlying cause of CF through restoration of CFTR function,” said J.P. Clancy, director, Pediatric Pulmonary Division, University of Alabama.
“When considered together with the encouraging results recently announced in patients with Duchenne muscular dystrophy, we have established a strong basis for advancing the clinical development of PTC124 and we are currently conducting preclinical studies with the intent to extend this concept into other disorders,” said Stuart Peltz, CEO, PTC Therapeutics.
PTC Therapeutics intends to initiates an international phase III trial program within 2007.