RNA-based therapeutics developer, AVI BioPharma, Inc., enters  collaboration for the development of two additional exon-skipping drugs, one for exon 45 and one for exon 50, to support AVI's broad-based development programme for the treatment of DMD.

AVI's collaboration with Children's National Medical Center in Washington, D.C. and the Carolinas Medical Center (CMC) will support certain IND-enabling activities for an exon 45-skipping therapeutic.

The collaboration with the National Institutes of Health (NIH) will support IND-enabling activities for an exon 50-skipping therapeutic and will be supported through in-kind research conducted either by the Therapeutics for Rare and Neglected Diseases (TRND) programme or by contract research organizations. AVI is currently conducting a phase IIb trial of eteplirsen, its exon 51-skipping therapeutic candidate for the treatment of DMD.


"The initiation of these additional programs, with the financial support of, and in collaboration with, leading institutions, is a validation of AVI's exon-skipping drug platform and will help to accelerate the development of our DMD programme," said Chris Garabedian, president and CEO of AVI BioPharma. "These collaborations will lay the foundation for AVI's pan exon strategy for the development of therapeutics to treat a majority of the DMD population."

The collaboration with Children's National and CMC will be funded primarily through two grants, one from the US Department of Defense's (DoD) Congressionally Directed Medical Research Program to Children's National and the other from the National Institute of Neurological Disorders and Stroke to CMC. This funding is intended to pursue the most promising treatments for DMD. The collaboration will support a series of GLP toxicology studies for an exon 45-skipping drug candidate based on AVI's phosphorodiamidate morpholino oligomer (PMO) chemistry. The details of the research plan associated with this collaboration, as well as the details of the funding from the NIH and the DoD, will be finalized by the parties over the next few months.

"Children's National is committed to finding an effective treatment for Duchenne muscular dystrophy patients," said Eric Hoffman, PhD, Director of the Center for Genetic Medicine Research at Children's National Medical Center. "Working with partners like AVI and their PMO-based exon-skipping therapeutics may accelerate the clinical development of a specific exon 45-skipping therapeutic that could improve the care and quality of life for more boys with this disease."

Dr Qi Long Lu, director of the McColl-Lockwood Laboratory at Carolinas Medical Center, added, "A strong pre-clinical GLP toxicology package is a critical part of a robust drug development program. Our collaboration with AVI is designed to help support the IND-enabling work necessary to advance AVI's exon-skipping drug candidates into the clinic."

The TRND programme is a new initiative by NIH designed to assist in the development of new drugs for rare and neglected diseases. To develop new medicines, TRND establishes partnerships with leading academic, government, biopharmaceutical and patient advocacy groups to focus on the discovery, optimization and pre-clinical testing of new drugs. AVI was selected for the award through a national competitive process. Definitive details of the collaboration will be finalized upon execution of a Cooperative Research and Development Agreement (CRADA) between the two parties.

"TRND collaborates with researchers and companies with an aim of helping companies like AVI advance TRND's mission of accelerating the development of therapeutics for rare diseases like DMD," said John McKew, PhD, Chief of TRND's Therapeutic Development Branch. "TRND selected AVI because its innovative platform technology has the potential to move forward into later stage clinical trials in this disease for which there is little or no therapy."