Portrait of Duchenne – Utrophin Modulator

Michelle Avery, PhD

Director, Investor Relations at Summit Therapeutics plc

In this second interview of our series “Portrait of Duchenne,” La Fondation La Force talks with Michelle Avery, the Director of Investor Relations for Summit Therapeutics plc. She also heads patient relations and public relations. Michelle Avery answers our question about utrophin modulators – a potential disease-modifying approach to treat all patients with DMD.

Our journey at La Fondation La Force travels the road of discovery with families, researchers, pharmaceutical companies and more. Our mission is finding the best new experimental treatments and the most promising research for children and adults with Duchenne muscular dystrophy (DMD). In November 2016, we went to the 14th Action Duchenne International Conference in London, UK, to meet with the international DMD community on your behalf.  We hope this series of interviews will bring hope and inspiration to Canadian families.

What is utrophin?

The human body naturally produces utrophin, a protein, when muscle first forms or when it’s repairing. As muscle matures, dystrophin replaces utrophin. However, in people with DMD, dystrophin does not function properly. Utrophin is functionally and structurally similar to dystrophin.

In the video, Michelle Avery answers our questions about utrophin modulators


  • Utrophin modulation is:

R: Utrophin is a protein very similar to dystrophin. Utrophin is present when a muscle is first forming or when it’s repairing. But then, for whatever reason, it’s turned off, and dystrophin comes in to take its place. Now, this is still in place in the boys with DMD… They have a lot of utrophin being made, as the muscles are repairing, but they also have the signal to turn it off. So what we’re trying to do is keep that utrophin turned on. As the muscles mature, they can have utrophin instead of dystrophin.

  • Where will this treatment be available first?

R: Right now, our development program… we’re considering a global program… Regulatory-wise, the U.S. usually comes along first and more rapidly than other countries, but we have a collaboration with Sarepta Therapeutics for Europe… They’re going to do a great job… They’ve had a lot of experience in DMD so far, so we’re excited to use their expertise, and they will be taking over in Europe and potentially in Latin America as well. There are some countries in Latin America where they have that option to sell our drug.

  • Can utrophin replace dystrophin?

R: We believe that utrophin can replace dystrophin, and this is based on a lot of work that we’ve done in mice, where we’ve seen that these mice can look much like “normal” when they have utrophin in the place of dystrophin. Utrophin itself is just a little bit smaller than the dystrophin, and it is missing a couple of different bonding. What we know from studies with patients with Becker’s is that if they’re missing the part of dystrophin that is missing in utrophin, these patients seem to do pretty well. So that’s encouraging that you don’t necessarily need all of the parts of dystrophin to have a more normal muscle function.

  • How do you see the future for the DMD community?

R: It’s very exciting work. We’re hoping this is, like, the first trial that lets us know whether or not this has a chance to work in patients, and we’re hopeful… It seems like it makes sense from a scientific perspective.


Clinical trial information updated: PhaseOut DMD

PhaseOut DMD is a Phase II clinical trial evaluating ezutromid, a utrophin modulator, in patients with DMD. This 48-week, open-label study is ongoing in the U.K. and USA. The study plans to enrol approximately 40 patients by mid-2017.

In addition to PhaseOut DMD, Summit Therapeutics plc plans to conduct a randomized, placebo-controlled trial specifically designed to support accelerated and conditional approvals for ezutromid in the USA and Europe. If PhaseOut DMD yields positive interim data, planning of this trial would begin after the 24-week data are reported.

For more information about the PhaseOut DMD clinical trial of ezutromid: Utrophin Trials   –  Clinical trials

For more information about utrophin, you  can also read our previous article: Utrophin production might be a good option for people with DMD

More about Summit PLC Therapeutics

Summit plc is e a clinical-stage drug discovery and development company advancing innovative therapies to significantly advance the current standard of treatment for serious unmet medical needs. Their strategy focuses on two therapy areas: Duchenne muscular dystrophy, a fatal genetic muscle-wasting disease, and the infectious disease caused by the bacteria C. difficile.


Up next:

Our next portrait: Exon Skipping with Georges Dickson

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We thank the Action Duchenne UK team, who received us with open arms and gave us access to all key speakers at their conference. Because of their generosity, we can spread this hopeful information to the Canadian DMD community.


To know more about the Action Duchenne conference:


Special Thanks to Daniel K Cooper and Allain Lagadic

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