Summit Presenting Duchenne Biomarker Validation Data and Details of PhaseOut DMD Trial at Meeting
The drug discovery and development biopharma Summit Therapeutics is giving several presentations related to the ongoing PhaseOut DMD clinical trial at the 22nd International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France.
The study is evaluating Summit’s lead investigative product, the utrophin modulator ezutromid, in about 40 ambulatory boys ages 5-9 with Duchenne MD.
Summit is also presenting three posters on muscle biopsy biomarkers, developed in collaboration with Flagship Biosciences, that were designed to determine the effect of utrophin modulators in clinical trials.
Utrophin functions in a similar way to dystrophin, which is lacking in patients with DMD. The trial hopes to provide proof-of-concept for ezutromid by measuring endpoints related to muscle fiber regeneration, health, and function.
Ezutromid may potentially be able to slow down or stop the progression of DMD, regardless of genetics. It may also be administered as a complementary approach along with other drugs for Duchenne.
The PhaseOUT DMD trial is being conducted at nine sites in the United States and seven in the United Kingdom. It includes a screening and baseline phase of up to 28 days; a 48-week open-label treatment phase; and a 30-day safety follow-up phase. An optional extension study is available to participants at the end of the 48-week treatment period.
The biomarkers Summit developed in collaboration with Flagship Biosciences are determined using an automated immunohistochemical analysis process that employs muscle biopsies to measure utrophin protein and myosin, a biomarker of muscle immaturity. The biomarkers have shown high consistency among images and across samples.
Summit has tried to ease the process of biomarker determination in biopsy samples by developing a standardized protocol to handle, process, and ship the biopsies. These biomarkers are currently being used in the PhaseOUT DMD trial.
“Developing new biomarkers capable of more reliably analyzing muscle biopsies in clinical trials has been a major priority for Summit, and I am delighted with the progress made in our collaboration with Flagship,” Dr. David Roblin, the chief operating and medical officer of Summit, said in a news release.
“These automated techniques are capable of analyzing thousands of muscle fibers in whole muscle biopsy sections, and we believe that their use could help us establish proof of mechanism for our utrophin modulator, ezutromid, in our ongoing PhaseOut DMD clinical trial,” Roblin said.
He said Summit is coming up on an important period in its utrophin modulation program. The biopharma expects to report the first 24-week biopsy, MRI, and functional data from PhaseOut DMD in the first quarter of 2018, with top-line data from the complete 48-week clinical trial expected in the third quarter of next year.
Dr. David Young, the CEO of Flagship Biosciences, said Flagship “proudly supports” Summit’s work in developing a therapy “potentially applicable to all patients with DMD with our advanced tissue analysis solutions.
“The progress we have made in developing these tools could advance the understanding of how patients are responding to treatment with ezutromid in this challenging rare disease,” Young added.