The newly founded Collaborative Trajectory Analysis Project (cTAP) coalition of stakeholders has invited Summit Therapeutics, a company specialized in the discovery and development of drugs targeting Duchenne muscular dystrophy (DMD), to support the development of new therapeutic products aiming to treat DMD, such as utrophin modulators.
cTAP was created to overcome the challenges of developing drugs for diseases characterized by heterogeneous progression. The project brings advanced data science to an alliance of all stakeholders, a first for the DMD community.
“In the Duchenne field, there is a strong community amongst the families, patient organizations, caregivers and industry that comes together to improve the lives of patients living with this disease, where time is of the essence,” David Roblin, president of research and development at Summit, said in a press release.
“cTAP is leveraging the natural history and clinical data collected within this community to create more predictive models of disease progression with the aim of enabling companies to interpret data and improve the design of clinical trials in DMD. We believe this could potentially benefit the development of our utrophin modulators for the treatment of all patients with DMD. In addition, we have the opportunity to contribute our own data to aid others as we all seek to improve the lives of DMD patients and their families,” said Roblin.
Because DMD is caused by genetic faults in the gene that encodes dystrophin, a protein like utrophin, which is functionally and structurally similar to dystrophin, has significant therapeutic potential.
In preclinical trials, the prolonged expression of utrophin had meaningful, positive effects on muscle performance. Utrophin modulation could thus offer a way to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Utrophin modulation also could be complementary to other therapeutic approaches.
Summit is researching the underlying powers of utrophin modulation with its lead candidate ezutromid (SMT C1100), an orally administered small molecule investigational drug.
Ezutromid is in a Phase 2 clinical trial called PhaseOut DMD (NCT02858362). The study aims to establish proof-of-concept through a range of muscle structure, muscle health and functional endpoints.
Summit also is planning to measure utrophin and a biomarker of muscle regeneration from muscle biopsies provided at baseline and again after either 24 or 48 weeks of treatment, to assess ezutromid’s activity.
A mid-trial report should be expected during the first quarter of 2018. Full data from the completed trial should be reported in late 2018.
Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan drug status to ezutromid, meaning Summit will receive certain benefits, additional regulatory support and a period of market exclusivity in the U.S. and Europe.
The FDA has additionally granted ezutromid its fast track designation (for accelerating review process) and rare pediatric disease designation (to help drugs intended to treat rare diseases in the U.S.)
“cTAP was started with the single mission of helping biopharmaceutical companies to bring treatments to our children living with DMD as quickly as we possibly can. This mission is only made possible through a collaboration of clinicians, patient advocates and the biopharma industry, and we welcome Summit in this effort,” said Debra Miller, founder and CEO of CureDuchenne and initial founder of cTAP.