AVI BioPharma Announces Department Of Defense Funding To Accelerate Development Of AVI-4658 For Duchenne Muscular Dystrophy
"We are pleased to collaborate with Dr. Hoffman, a distinguished researcher and expert in the field of DMD, and advance AVI's efforts toward the development of therapeutics utilizing exon skipping for the treatment of DMD," said Steve Shrewsbury, M.D., Chief Medical Officer and Senior Vice President of Clinical and Regulatory Affairs of AVI BioPharma. "Through this collaboration, AVI hopes to provide the additional data as requested by the FDA to allow clinical studies with AVI-4658 to begin in the U.S."
The collaboration will support a series of GLP toxicology studies for AVI's exon skipping drug candidates based on phosphorodiamidate morpholino oligomers (PMO) chemistry. The funding is part of the Department of Defense's Congressionally Directed Medical Research Program to identify and pursue research with the most promise for treatment of DMD.
"Exon skipping represents one of the most promising investigational approaches for the treatment of Duchenne muscular dystrophy, and it is imperative to accelerate the clinical development of therapeutics that could improve the care and quality of life for boys with this disease,' said Dr. Hoffman. "We are excited to be collaborating with AVI, both a pioneer and a leading developer of RNA-based drugs."
"A strong pre-clinical GLP toxicology package is a critical part of a robust drug development program, and this new grant is designed to help enable both intravenous and subcutaneous clinical trials in DMD patients in the U.S.," said Dr. Connor.
AVI is currently evaluating AVI-4658 in human clinical trials in Europe. The drug is designed to skip exon 51 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. By skipping this exon, a truncated, yet potentially functional form of the dystrophin protein is produced, which could ameliorate the disease process and possibly prolong and improve the quality of life in these patients. Results from a Phase 1 proof-of-concept trial showed that injection of the drug into the muscles of a series of DMD boys successfully induced dystrophin production in a dose-responsive manner. Further, the drug was well tolerated, with no significant drug-related adverse events reported. The clinical trial was conducted in collaboration with the MDEX Consortium in London UK. AVI is currently sponsoring an ongoing clinical trial in the UK evaluating the systemic delivery of AVI-4658. This is an open label, 12 week safety trial, which includes measures of drug efficacy and pharmacokinetics, being conducted in London, UK at the UCL Institute of Child Health / Great Ormond Street Hospital NHS Trust facilities by members of the MDEX Consortium.
About Duchenne Muscular Dystrophy (DMD)
DMD is the most common fatal genetic disorder to affect children around the world. Approximately one in every 3,500 boys worldwide is afflicted with Duchenne muscular dystrophy with 20,000 new cases reported each year. It is a devastating and incurable muscle-wasting disease associated with specific inborn errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Symptoms usually appear in male children before age six. Progressive muscle weakness of the legs and pelvis eventually spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients are confined to a wheelchair by age 12. Eventually, this progresses to complete paralysis and increasing difficulty in breathing. The condition is terminal and death usually occurs before the age of 30. The outpatient cost of care for a non-ambulatory DMD boy is among the highest of any disease. There is currently no cure for DMD, but for the first time in decades, there are promising therapies in or moving into development.
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