UK Duchenne research charity to invest $1.2M in US biotech’s R&D

Funding

Action Duchenne, a UK research charity raising funds to find a cure for Duchenne Muscular Dystrophy (DMD), is teaming up with US biotech company AVI BioPharma, Inc. to accelerate research and development of AVI’s discovery stage exon-skipping drugs for the treatment of DMD.

“We have teamed up with AVI to help accelerate these clinical programmes as part of our commitment to cure this devastating disease,” said Nick Catlin, CEO of Action Duchenne. “Our primary goal is to help provide treatment options for the many children and adults afflicted with DMD. We believe we can [add] significant value to AVI’s ground breaking efforts.”

Leslie Hudson, President and Chief Executive Officer of AVI BioPharma, said, “We believe this collaboration has high potential and we are also very pleased to receive the financial support from Action Duchenne which will help accelerate our research and development efforts for new exon skipping therapeutics.”

The agreement has a one-year term, with an option to extend for additional years, and will provide approximately $1.2 million in support to AVI over the initial term for advancement of research, regulatory work and clinical trial recruitment.

AVI is currently conducting a dose-finding clinical trial evaluating the systemic delivery of AVI-4658. The open label, 12 week safety study includes measures of drug efficacy and pharmacokinetics. It is being conducted at the UCL Institute of Child Health, Great Ormond Street Hospital NHS Trust London and at the Royal Victoria Infirmary, Newcastle, UK, which is the centre for the European Treat Neuromuscular Diseases (Treat-NMD) initiative. The clinical costs for the trial are being met in part by the UK Medical Research Council.

DMD is the most common fatal genetic disorder, affecting approximately one in every 3,500 boys worldwide. The incurable muscle-wasting disease is associated with errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fibre function

AVI-4658 is designed to override the error by skipping exon 51 (an exon is a sequence of DNA that is expressed in a protein) of the dystrophin gene. 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 of these patients.

Results from a Phase I proof of concept trial showed that injection of the drug into the muscles of boys with DMD successfully induced dystrophin production in a dose-responsive manner. The drug was well tolerated, with no significant drug–related adverse events detected. The clinical trial was conducted in London.

Action Duchenne was set up by Duchenne families in 2001 to promote new research for a cure for Duchenne. The charity has funded 9 major projects costing over £1m and has been a leading partner in the £1.6m DMD project funded by the UK Department of Health.

In 2005 Action Duchenne launched the Duchenne Registry, the first National Duchenne database that holds gene information of people living with Duchenne and can be used to speed up the recruitment of patients for clinical trials.