Summit stops development of utrophin drug

Published Date
Jenny Sharpe

We are disappointed to hear that Summit Therapeutics has decided to discontinue the development of its utrophin modulator, ezutromid. The company recently completed a 48 -week phase 2 trial, PhaseOUT DMD, testing ezutromid in boys with Duchenne.

After reviewing the results from the trial, Summit has reported that whilst ezutromid was well tolerated, it unfortunately did not provide a benefit to the boys. This is disappointing given the positive interim results reported earlier this year.

Summit is currently working with clinicians involved in PhaseOut DMD to bring the trial and associated extension phase to a conclusion. If your son is enrolled in this trial, please consult his doctor for more information.

Summit also plans to explore ways that information gathered as part of PhaseOut DMD can be made available to other researchers. It’s important to share knowledge in this way, even if the data is negative, in order to help design better drugs and trials for Duchenne.

Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said:

This will be extremely disappointing news for families living with Duchenne. It’s important that we recognise the huge commitment made by the 40 boys and their families who participated in this 48 week trial, and thank them for their part in furthering Duchenne research. We welcome Summit’s plans to make the trial data more widely available – this will hopefully be valuable information that will help the development of other therapies for Duchenne.


We will share any further news on our website when it is available from Summit Therapeutics.  Further, we will continue to support research and promising steps to an effective Duchenne treatment as every day counts when you’re living with Duchenne.

Professor Dame Kay Davies, who works on utrophin research at Oxford University, said:

We intend to continue the utrophin approach. Ezutromid failed but everyone agrees that the utrophin modulation strategy did not. We remain committed to a solution for all boys wherever in the world they are and at whatever age.

You can read more about this news in Summit’s community letter and press release.

If you have any further questions please contact our Clinical Research Officer, Dr Sofia Nnorom, on 020 7803 4813 or at

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