Friday 15 April 2011
New exon skipping trial for Duchenne announced
Following the release of the encouraging results of the UK exon skipping clinical trial in June 2010, AVI Biopharma has announced that it will start another trial in the third quarter of 2011. This trial will test higher doses of the exon skipping drug and will be conducted in the USA.
The trial will involve injecting a small pieces of DNA called "antisense oligonucleotides" or "molecular patches" into the bloodstream to restore the production of the dystrophin - the protein that is missing in boys with Duchenne muscular dystrophy. The molecular patch tested in this trial is designed to skip exon 51 which could potentially be used to treat 13% of boys with Duchenne muscular dystrophy. The molecular patch was previously called AVI-4658 and has now been named eteplirsen.
Eteplirsen (AVI-4658) was well tolerated by all 19 boys participating in the previous trial which tested six different doses of the drug. The eight boys receiving the highest doses produced new dystrophin protein in their muscles. Encouragingly, three of the boys produced considerable amounts of dystrophin, but unfortunately some boys produced very little. So this new trial will test a higher dose to try to achieve more consistent dystrophin production amongst patients.
This new trial will prepare for a large international phase 3 clinical trial of eteplirsen which is predicted to start in the second half of 2012.
Further information and links
Read about the results of the previous AVI Biopharma exon skipping trial.
News from the Prosensa/GSK exon skipping trials.
More information about Duchenne muscular dystrophy.
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