Sarepta Therapeutics, Inc. and the University of Western Australia (UWA) have entered into an exclusive, worldwide licensing agreement for intellectual property rights to support the development of exon-skipping drug candidates for the treatment of Duchenne muscular dystrophy (DMD). The agreement grants Sarepta rights to UWA's extensive patent portfolio in DMD and enables the Company to build out its exon-skipping pipeline with new candidates based on its proprietary phosphorodiamidate morpholino oligomer (PMO) technology to address the majority of patients with the disorder worldwide.

Under the terms of the agreement, UWA is eligible to receive up to $7.1 million in upfront and development milestone payments, as well as a low single-digit royalty on net sales of all approved medicines under the collaboration. Additional financial terms were not disclosed.

The deal expands an agreement first signed in 2008, which supported the development of several exon-skipping drugs including eteplirsen, Sarepta's lead clinical candidate for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51.

"This agreement underscores our commitment to pursue treatments for all DMD patients who can benefit from our exon-skipping technology, even those with rare genetic mutations," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We believe this collaboration allows us to apply our unique morpholino chemistry with UWA's groundbreaking research on the dystrophin gene to develop potentially best-in-class drugs for this disease across the globe."

"We are very encouraged by the recent progress of Sarepta's lead clinical program eteplirsen, which we believe demonstrates the strength of the underlying platform technology as well as our partner's drug development expertise in DMD," said Andy Sierakowski, director of the Office of Industry and Innovation at UWA. "This expanded collaboration with Sarepta enables us to translate our understanding of the dystrophin gene into additional potential exon-skipping therapeutics that address a majority of patients with DMD, representing a major contribution by the University of Western Australia to the field of genetic medicine."

DMD is a rare and severe genetic disorder that affects boys and young men. It is associated with errors in the gene for dystrophin, a protein that plays a key structural role in muscle fibers. Patients with DMD lack functional dystrophin, and regular activity causes progressive muscle damage leading to weakness, loss of ambulation, respiratory and cardiac dysfunction, and eventually premature death. There are no approved treatments.

Exon-skipping is an innovative disease-modifying treatment approach designed to skip an exon in the dystrophin gene, thereby enabling the repair of specific genetic mutations and the production of a functional, but shorter, form of dystrophin. Sarepta currently has four exon-skipping programs in DMD addressing patients with genotypes amenable to skipping of exons 51, 45, 50 and 53.

DMD is an X-linked rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders and a devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs eventually spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.

Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases.