Tuesday 6
August 2013
Sarepta Therapeutics eteplirsen
results accepted for publication
The
results of Sarepta Therapeutics' phase 2b clinical trial of eteplirsen - a
potential exon skipping drug (or molecular patch) - have
been accepted for publication in a medical journal. The study focuses on the
first 48 weeks of the trial. After boys with Duchenne muscular dystrophy received
the potential drug, production of the dystrophin protein was restored
in up to 50% of the muscle fibres that
were examined. Researchers also noted that boys who took eteplirsen for 48
weeks were able to walk, on average, 67.3 metres further in six minutes than those
who took a placebo (an inactive
drug) for 24 weeks followed by eteplirsen for 24 weeks. Importantly, the
results show that eteplirsen was safe, with no serious side effects observed in
any boy in the trial.
Whilst these results are promising, but the
trial was small, with only 12 boys in total. It is therefore possible that a
larger trial will be required to confirm these results. Importantly, the
company's paper has been accepted for publication in a scientific journal
(called Annals of Neurology). This is the first time the results of the trial
have been subjected to peer review. Peer review is a process of quality control
for science which lets independent scientists (peers) examine the methods and
results of a study to check that the conclusions reached are correct. The
scientists can highlight inaccuracies or problems in the study which the
authors must address before publication.
Dr
Marita Pohlschmidt, Director of Research at the Muscular Dystrophy Campaign,
said:
It is really encouraging that Sarepta
Therapeutics has chosen to publish these results in a peer-reviewed journal.
Peer review is 'quality control' for the scientific community. By publishing
its results in this way Sarepta is allowing independent scientists to
scrutinise the study and its conclusions.
What we need to see now is eteplirsen tested
in a larger group - this trial included only twelve boys - to confirm these
promising results.
