PhaseOut DMD - A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with Ezutromid (SMT C1100) in Ambulatory Boys with Duchenne

Frequently Asked Questions (FAQs) about this study:


What stage is this research?

  • Ezutromid is in a Phase 2 proof-of-concept clinical trial, called PhaseOut DMD.


What is utrophin and utrophin modulation?

  • Utrophin is a naturally occurring Glossary Link protein that is functionally and structurally similar to dystrophin. Utrophin is produced naturally during the early stages of muscle fiber development but is turned off in maturing muscle fibers, as dystrophin steadily increases to perform the same functional role. When a muscle fiber is damaged, utrophin is produced during the repair process. Summit’s utrophin modulation approach aims to use small Glossary Link molecule drugs to maintain the production of utrophin to compensate for the absence of dystrophin in patients with Duchenne and so has the potential to maintain healthy muscle function.
  • Ezutromid is an orally administered utrophin modulator that has the potential to benefit all patients regardless of their dystrophin Glossary Link mutation. This is Summit’s most advanced utrophin modulator while the Company is also advancing an earlier-stage pipeline of future generation molecules. Summit believes that the approach of utrophin modulation could be complementary to other therapeutic approaches currently in development or that are approved.


What is the goal or purpose of this research?

  • PhaseOut DMD is a 48-week open label trial that aims to provide proof of concept for ezutromid and utrophin modulation through measurements of muscle fat infiltration using magnetic resonance imaging (MRI), and measuring utrophin protein and muscle fiber regeneration by analysis of muscle biopsies. All patients will have two muscle biopsies – one at baseline and a second after either 24-weeks or 48-weeks of dosing with ezutromid. Exploratory endpoints include the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes.
    At the end of the 48-week period, patients participating in the trial will have the opportunity to be transitioned into an open-label extension phase.


What is the current status of the PhaseOut DMD trial?

  • This trial has recently completed enrolment of patients with dosing of patients still ongoing. Some of the key inclusion criteria for being able to participate in this trial included:
    • age ranging from 5th to 10th birthday with confirmation of Duchenne diagnosis through genetic testing or muscle biopsy,
    • ability to walk a prespecified distance that is at least 300 meters unassisted in a 6-minute walk test, and
    • being on a stable regimen of corticosteroids for at least 6 months prior to enrollment.


When will data from PhaseOut be reported?

  • Summit expects to report interim data from trial in the first quarter of 2018. This is planned to include data from all of the patients who will have their second muscle biopsy after 24-weeks of dosing, as well as the 24-week analysis of MRI and functional data from all patients in the trial.  We plan to analyze all 24-week treatment biopsies once all samples have been collected. In addition to reporting on the 24-week biopsy data, we also expect to announce the 24-week analysis of MRI and functional data from all patients in the trial.


What are the next steps for the development of ezutromid?

  • In addition to PhaseOut DMD, Summit plans to conduct a randomized, placebo controlled trial designed with the potential to support accelerated and conditional approvals in the US and Europe, respectively. It is anticipated that this trial would start after positive data from PhaseOut DMD, and the Company would plan to provide timing guidance following the release of the 24-week dataset.


How long until ezutromid could be available on the market?

  • Summit is currently focused on the PhaseOut DMD trial. If this is successful, Summit would expect to run a larger, global registration trial, and the timing and length of that trial would be largely determined by what is seen in PhaseOut DMD and discussions with the regulatory authorities. 


Why should I consider participating in clinical trials of a utrophin modulator?

  • While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to new research treatments before they are widely available, having access to medical specialists that are normally not available to you or your child, and helping others by contributing to the better understanding of Duchenne.


Where can I learn more about this study?


(Updated 5/15/2017)