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December 2012

Sarepta Therapeutics Enters Into Collaboration for the Development of Additional Exon-Skipping Product for Duchenne Muscular Dystrophy

Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, announced on November 26 a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in development, in support of Sarepta’s broad-based program for the treatment of Duchenne muscular dystrophy (DMD). Sarepta’s collaboration is with University College London’s (UCL) scientist, Professor Francesco Muntoni, MD, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the EU and US. The EU Health Innovation-1 2012 Collaborative research grant will support certain IND-enabling activities and clinical proof of concept studies for an exon 53-skipping therapeutic. Sarepta recently announced positive results from its extension study of its Phase IIb trial of eteplirsen, its exon 51-skipping therapeutic candidate for the treatment of DMD. Sarepta is also developing other PMO-based exon-skipping drug candidates for exons 45 and 50.

“The recent compelling clinical data on eteplirsen targeting exon 51, which started with our work on the Phase I study in the UK, provides a strong foundation for using Sarepta’s technology against exon 53,” said Francesco Muntoni, professor of pediatric neurology and head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health, London. “We are pleased to be working with Sarepta to bring this exciting exon-skipping therapeutic approach to Europe and the broader DMD population.”

This program will be based on Sarepta’s advance proprietary RNA-based platform, Phosphorodiamidate Morpholino Oligomers (PMOs), which is a novel backbone chemistry that provides enhanced target tissue specificity, increased potency to allow for more efficient dosing, and greater uptake within a cell to further increase protein production. Targeting exon 53 with this technology will potentially address one of the most prevalent sets of mutations in DMD that are amenable to exon-skipping (deletion of exons 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, or 52) by restoring the cellular machinery’s ability to produce a functional dystrophin protein.

“The initiation of this program, along with our other collaborations for exons 45 and 50, continues to advance Sarepta’s 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 Therapeutics. “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.”

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