Tuesday 24 July 2012

Latest results from phase 2b exon skipping trial encouraging

Sarepta Therapeutics (the company formerly known as AVI Biopharma) has announced further encouraging results from their phase 2b clinical trial of the exon skipping drug eteplirsen in treating Duchenne muscular dystrophy. The new results have shown that boys taking the higher dose of eteplirsen could, on average, walk 69m further in the six minute walk test than those taking a placebo.

Sarepta Therapeutics today announced the results of muscle function tests carried out during the phase 2b trial of the exon skipping drug, eteplirsen. 12 boys were recruited for the trial, four of whom received high dose eteplirsen (50mg/kg), four received low dose eteplirsen (30mg/kg), and four received a placebo.

These new results show that in the six minute walk test, the higher dose of eteplirsen (50mg/kg) was able to slow the decline in walking ability in boys with Duchenne muscular dystrophy - and after 36 weeks of treatment, those treated with the drug could walk 69m further than those given a placebo. The company has previously announced that the potential drug was well tolerated and no serious side effects were seen, and have also shown that the drug could restore dystrophin production in a significant number of muscle fibres.

What are the next steps?

Sarepta Therapeutics is still monitoring the participants in the trial and hopes to perform further walking tests after 48 weeks of treatment. The company has stated that if these results confirm those seen after 36 weeks then they will meet with drug regulators in the USA to discuss the best way to get the drug to market and determine if further, larger trials of the drug will be needed.

Mr Robert Meadowcroft, Chief Executive of the Muscular Dystrophy Campaign said:

This is very encouraging news which underlines again the real promise of exon skipping as a potential route to a treatment and this news offers so much hope to so many people.

However, we need to bear in mind that the trial involved only 12 boys and lasted for 36 weeks hence data from a larger study over a longer period would give us a much clearer picture.

We are delighted that our long term support for exon skipping research has been instrumental in getting to this promising position and we will shortly announce a new initiative to coordinate efforts in the UK to win the fight against Duchenne muscular dystrophy.