Industry Partner News: Summit Announces that Ezutromid Significantly Reduced Muscle Damage in DMD Patients in 24-week Interim Data from Summit's PhaseOut DMD Clinical Trial


This morning, Summit Therapeutics plc announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid. This data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies. Summit plans to have a webinar with the community – please stay tuned for details.


1.       What does this data mean? This is an early look at data from PhaseOut DMD. In these data, we see that ezutromid treatment led to a significant reduction in muscle damage and increased production of utrophin in muscle fibers. This fits with our expectation that utrophin modulation maintains utrophin production in mature muscle fibers, enabling utrophin to replace the need for dystrophin in Duchenne muscular dystrophy muscles. We don’t yet know if ezutromid will be able to produce long term functional or other clinical benefits, but this is a very exciting step for ezutromid and utrophin modulation. Importantly, ezutromid has been well tolerated to date in all patients in PhaseOut DMD. We look forward to the results from the full 48 weeks of the trial expected in the third quarter of 2018.

2.       Will there be another clinical trial? When will that start/where will it take place? We expect to have to conduct another clinical trial aimed at getting regulatory approval for ezutromid to be marketed in the US and Europe. With these positive interim data in hand, we are actively planning for the next trial and expect to provide a timeline for the start of that trial once we have the 48-week data. It is expected to be a global trial, and the participating countries and sites will be announced closer to the initiation of the trial.

3.       When will ezutromid be available on the market? We are awaiting the 48-week data from PhaseOut DMD prior to finalizing our plans for the next trial. Once we have these plans in place, we will be able to better predict when we could potentially file for regulatory approvals. These filings would need to be approved by the applicable regulatory authorities before ezutromid would be available on the market.

4.       Can I get access to ezutromid before it’s approved? Running clinical trials that can support regulatory approvals is the best way to ultimately ensure wide access for patients to ezutromid. All of our efforts are therefore focused on conducting rigorous clinical trials to establish the safety and potential clinical benefits that ezutromid may have. At this point in the development, we cannot support any use of ezutromid outside of our clinical trials and any associated extension phases.

For more information, please see the full Summit press release by clicking here.