PTC Therapeutics has landed a $25 million award from Cystic Fibrosis Foundation to support Phase II development of PTC124. The drug is under investigation for cystic fibrosis caused by nonsense mutations. PTC gained attention last year when it announced that PTC124 was effective in treating a particular group of patients with Duchenne muscular dystrophy triggered by nonsense mutations, which is found in about 10 to 15 percent of patients. More importantly, however, the study highlighted that PTC124 could be the key to dealing with the nonsense mutations involving more than 1,800 genetic disorders like cystic fibrosis (10 percent of cases) and Hurler's disease (70 percent of cases).
"The encouraging data from the Phase IIa studies indicate that PTC may become a very important drug for the treatment of CF in the 10 percent of patients who carry nonsense mutations," said Robert J. Beall, president and CEO of the Cystic Fibrosis Foundation. "These exciting results lead us to believe PTC124 has the potential to be one of the first oral drugs on the market to address the basic defect in cystic fibrosis." PTC was a 2007 Fierce 15 winner.
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