In a press release earlier, Sarepta Therapeutics announced results of an interim analysis of the casimersen part of their late stage, phase 3 ESSENCE trial.

Muscle biopsies were performed at the start of the trial and after 48 weeks. Sarepta has now completed an analysis of the total level of dystrophin protein in the muscle samples, and found levels increased in participants who received casimersen (a potential drug designed to skip exon 45 of the dystrophin gene) compared to their levels at start of the trial. Levels were also higher than those who received a placebo (an inactive form of the drug)

The company also reported that of the 22 samples tested, all showed evidence of exon skipping when the RNA - the messenger molecule that carries the instructions from the DNAto where proteins are produced in a cell - was decoded.

These initial results are encouraging, and we look forward to the complete analysis of all the data at the end of the trial, and its publication in a peer-reviewed journal. Sarepta has already announced that they plan to use these results to work towards filing an application to the FDA (the drug regulator in the USA) to market casimersen there.

The ESSENCE trial is a 96-week, global phase 3 trial which is testing the safety and effectiveness of casimersen and golodirsen (separately) in patients amenable to exon 45 or 53 skipping respectively. The casimersen part of the trial includes 43 patients - 27 received weekly intravenous infusion while 16 received a placebo.

Find out more

Contact Neil Bennett, Director of Research, to find out more on 020 7250 8240

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