Summit Therapeutics has completed patient dosing in the PhaseOut DMD clinical trial, a Phase II study of ezutromid to treat patients with duchenne muscular dystrophy (DMD), the most common and severe form of muscular dystrophy.

The 48-week trial of the company’s utrophin modulator, ezutromid, enrolled 40 patients aged between five and ten years in the US and UK.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

Ezutromid, an orally administered, small molecule drug, is Summit’s lead utrophin modulator.

“These results, if positive, could form the basis of a regulatory filing of ezutromid, bringing this universal treatment to patients more rapidly.”

It has previously received orphan drug status from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

The primary objective of the PhaseOut DMD trial is the change from baseline in magnetic resonance parameters related to the leg muscles.

The trial’s secondary objectives are biopsy measures evaluating utrophin and muscle damage.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Its exploratory objectives comprise the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes.

Summit Therapeutics Research and Development president and chief medical officer Dr David Roblin said: “We believe the early improvements seen in muscle health in the interim data from PhaseOut DMD indicate ezutromid is reducing DMD disease severity.

“In the full trial results, we aim to see continued utrophin modulation and sustained changes in magnetic resonance parameters.

“These results, if positive, could form the basis of a regulatory filing of ezutromid, bringing this universal treatment to patients more rapidly.”

Following the completion of the treatment period, patients included in the trial have the option to enrol themselves into an extension phase to continue to receive ezutromid.

Top-line data from the trial is expected to be available by the third quarter of this year.