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news.PTC Therapeutics has been awarded $1.7 million from the drug discovery and development affiliate of the Cystic Fibrosis Foundation to fund development of its oral drug candidate, PTC124.
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PTC124 will be developed for the treatment of cystic fibrosis (CF) due to a nonsense mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. The product candidate is a novel, orally administered drug that targets nonsense mutations and is currently being evaluated in phase I clinical trials involving healthy volunteers.
“Unlike therapies that alleviate symptoms, PTC124 targets the root cause of CF in these patients,” explained Dr Robert Beall, president and CEO of the CF Foundation and Cystic Fibrosis Foundation Therapeutics Inc (CFFT). “We look forward to seeing the results from the phase I clinical trial of PTC124.”
Established in 2000, CFFT supports and assists activities related to CF drug discovery and development. Total support of CFFT is provided by the CF Foundation.
“We are honored to receive this award from CFFT and for the ongoing support of the CF Foundation,” said Dr Stuart Peltz, president and CEO of PTC. “The funding and guidance by CFFT are pivotal to the development of PTC124 in CF.”