PTC124 proves promising in cystic fibrosis trial

John Carroll — Thu, 21 Aug 2008 10:58:58 -0400

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. Last month Genzyme paid $100 million 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 in the Washington Post

PTC sued by an angry parent in search of PTC124

John Carroll — Fri, 18 Jul 2008 10:47:01 -0400

PTC Therapeutics has been on a roll over the last few months. But the biotech hit a loud bump when the New York Times featured the story of one mother's legal fight to gain access to PTC124 for her son, who suffers from Duchenne muscular dystrophy. PTC just inked a $437 million deal with Genzyme for PTC124, which has demonstrated promising results for treating a segment of the Duchenne MD population. Cheri Gunvalson says that the experimental therapy is her son's last and best hope. But PTC isn't providing the therapy to anyone who isn't in the clinical trial and the boy, who can no longer walk, isn't eligible. Gunvalson is asking for access as part of a compassionate use approach that a number of drug developers have in place, but PTC CEO Stuart Peltz has said that there's been too little experience with the mid-stage therapy to provide it outside of a trial.

The Times notes that for many people an experimental drug is their only hope. But the Abigail Alliance for Better Access to Developmental Drugs took their case for broader access to experimental cancer therapies to court last year--and lost.

- read the article in the New York Times

Muscular Dystrophy

PTC124 proves promising in cystic fibrosis trial

PTC sued by an angry parent in search of PTC124