Summit Therapeutics has released interim results from their ongoing PhaseOut DMD study. The 48-week phase 2 trial is assessing the safety and efficacy of utrophin modulator, ezutromid, in boys with Duchenne muscular dystrophy.
Muscle biopsies taken before treatment and after 24 weeks of treatment were compared. This showed that 14 out of 22 participants had a noticeable reduction in muscle damage. In addition, levels of the utrophin protein had increased.
The company also reported that ezutromid has been well tolerated to date. Initial results from the final 48-week time point are expected to be reported in the third quarter of 2018.
Professor Dame Kay Davies from the University of Oxford and co-founder of Summit said in a press release:
These data provide the first evidence of utrophin modulation working in patients. If further findings build on this evidence they could establish ezutromid as a universal, disease-modifying treatment and bring hope to all patients and families living with DMD.
Whilst these preliminary results are positive, it is not yet known if ezutromid will be able to improve muscle function. Summit is planning to conduct a larger, placebo-controlled trial that is intended to provide evidence for marketing approval (known as a pivotal trial).
The company will provide further information on their pivotal trial once PhaseOut DMD is completed.
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