Wave Life Sciences have today announced the results of their early stage phase 1 clinical trial of Suvodirsen, as well as their plans for a much larger phase 2/3 clinical trial of the potential drug.

The phase 1 trial was the the first test of Suvodirsen in patients. The company tested different doses of the potential drug and found that it was safe and well-tolerated. However, at the higher doses patients experienced side effects that included headaches and sickness after receiving Suvodirsen.

Wave has also announced plans to start a phase 2/3 trial of Suvodirsen. The trial aims to recruit 150 boys living with Duchenne who are aged between 5 and 12 and who are amenable to exon 51 skipping. Importantly, Wave has used the data from the phase 1 trial to choose doses of Suvodirsen that are lower than those that caused more serious side effects.

So far, Wave has not collected data to investigate the effectiveness of Suvodirsen, but the phase 2/3 trial will include a wide range of functional measures including the Northstar Ambulatory Assessment and muscle biopsies that will allow the levels of dystrophin in the muscles to be examined. Everybody who took part in the phase 1 trial has now moved into an open label extension study (at the doses chosen for the phase 2/3 trial) and these individuals will also undertake muscle biopsies. Using this data, Wave hopes to be able to announce later this year how much dystrophin is being produced.

The sites of the new trial have not yet been announced, but as we find out more we will let you know.

Find out more

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

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