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PTC receives $25 million from CFFT

This article was originally published in Scrip

Executive Summary

PTC Therapeuticsis to receive further financial support from Cystic Fibrosis Foundation Therapeutics(CFFT), the non-profit subsidiary of the Cystic Fibrosis Foundation, to support development of PTC124, PTC's investigational oral drug for cystic fibrosis caused by nonsense mutations. PTC will receive up to $25 million to fund Phase IIb clinical trials of PTC124. In 2005, it received $1.7 million from CFFT to fund the Phase I trials. Last month, PTC reported data from Phase IIa studies of PTC124 in adult and paediatric cystic fibrosis patients demonstrating significant improvements in the primary outcome measure – nasal potential difference. A three-month, Phase IIa extension study of adult patients showed a significant mean decrease in the frequency of cough, one of the most prominent symptoms of the disease. PTC124 is also in Phase IIb development for Duchenne muscular dystrophy.

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