Sarepta partners on fourth drug aimed at Duchenne

Monday, November 26, 2012

Sarepta Therapeutics (Nasdaq:SRPT) says its collaborating with a London scientist and other organizations for development of a fourth drug candidate for the treatment of Duchenne muscular dystrophy (DMD), a rare, degenerative neuromuscular disorder causing muscle loss and a premature death.

The move is spurred in part by good results of a Phase 2b study announced last month of eteplirsen, Sareptas lead drug candidate using its proprietary technology to skip a sequence of DNA, known as exon 51, which is hoped to enable the repair of genetic mutations that affect about 13 percent of the total DMD population. The results of the trial showed significant improvement over a placebo, according to the company, in a 48-week Phase 2b study measuring increase in dystrophin, a protein which slows the progression of the disease, as well as the so-called six minute walk test, measuring the distance a DMD patient can walk in six minutes.

The aim of the new collaboration - with professor Francesco Muntoni, M.D., of the University College London, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the European Union and United States- is to develop a drug to similarly skip another sequence of DNA known as exon 53 to address a different genetic mutation thought to cause DMD.

The initiation of this program, along with our other collaborations for exons 45 and 50, continues to advance Sareptas strategy in pursuing exon-skipping therapeutics for all of the DMD patients who may benefit from this drug technology, said Chris Garabedian, president and CEO of Sarepta in a statement. Our goal of demonstrating that the success of eterplirsen can be reproduced across other exon-skipping targets is a critical step in being able to treat more boys and young men affected with this devastating disease.

Sareptas stock was up 5 percent Monday to $30.80, double its value as of Oct. 2, before the results of the Phase 2b study were announced. The company did not respond to a request for comment as of deadline.

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Sarepta partners on fourth drug aimed at Duchenne