PTC124 proves promising in cystic fibrosis trial

PTC Therapeutics' PTC124 proved promising in an early stage cystic fibrosis trial in Israel. PTC124 has already spurred tremendous interest as a therapy for Duchenne muscular dystrophy. And the developer believes that the therapy could prove effective in a number of genetic diseases [1]. Last month Genzyme paid $100 million [2]upfront and promised to pay up to $337 million in development and sales milestones to obtain co-marketing rights to PTC124.

In the current trial, researchers tested PTC124 against a type of cystic fibrosis caused by a mutation in the salt channel gene that blocks critical protein development. The therapy stops the blocking and allows the development of the proteins. That particular type of cystic fibrosis triggers half of all cases in Israel and 10 percent of the cases in the United States. Researchers say they will now push ahead with a larger trial to test the efficacy of the drug.

"We are planning a larger long-term study that will be international, in the United States and Canada and also in Europe," said Dr. Langdon Miller, chief medical officer of PTC Therapeutics.

--read the report [3] in the Washington Post

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PTC sued by an angry parent in search of PTC124 [4]
PTC inks $437M pact with Genzyme [2]
PTC gets $25M drug development award [5]
Genetic therapy could cure 1800 diseases [1]