A clinical trial on utrophin modulation is completed

One of the objectives of our team is to inform you about new treatments. You can catch up on the last news in the latest press release by Summit Therapeutics company about ezutromid, a utrophin modulation. We wish you a happy reading and thank you for sharing those articles within the DMD community.


“PhaseOutDMD” clinical trial for ezutromid is completed

“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.”

Said Dr. David Roblin, Chief Medical Officer and President of R&D of Summit.

What is “PhaseOutDMD”?

PhaseOut DMD is a Phase 2 open-label, multi-centre trial of the Company’s utrophin modulator, ezutromid, in patients with DMD. Previously announced 24-week interim data from PhaseOut DMD showed evidence of activity across three different measures. Specifically, ezutromid:

• Maintained the production of utrophin, a naturally occurring protein that can potentially substitute for dystrophin, as measured by muscle biopsy;

• Significantly and meaningfully reduced muscle damage, as measured by muscle biopsy; and

• Significantly reduced muscle inflammation, as measured by magnetic resonance.

What is utrophin?

The human body naturally produces utrophin, a protein, when a muscle is first forming or when a muscle is repairing. As a muscle matures, dystrophin replaces utrophin. However, in people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly. Utrophin is functionally and structurally similar to dystrophin. Preclinical trials that have simulated sustained utrophin production have shown that it could potentially replace dystrophin in people with Duchenne muscular dystrophy (DMD). The replacement of dysfunctional dystrophin with functional utrophin might have a highly positive impact on muscle performance.

Summit Therapeutics believes that utrophin may slow or even stop the progression of DMD.


This video explains what is utrophin.

About Summit Therapeutics

Summit is a biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programmes focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).

What is DMD?

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,5000. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

Useful links

Here are a few additional links including the initial news release of the Summit Therapeutics company.

Link to the news release: www.summitplc.com

Link to additional information on utrophin modulation:

Positive data about utrophin modulator – La Force DMD

Portrait of Duchenne – Utrophin modulator – La Force DMD


  • 2018 > Summit Therapeutics ends development of ezutromid therapy for DMD after trial failure. Read more here.
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