24-Week Analysis from PhaseOut DMD Presented at AAN

At the 70^th American Academy of Neurology (AAN) Annual Meeting this morning Summit Therapeutics plc presented new 24-week interim data from PhaseOut DMD, its Phase 2 trial of ezutromid in Duchenne muscular dystrophy (DMD).

The new analysis show a high correlation between a decrease in muscle damage and a reduction in muscle inflammation in patients treated with ezutromid.

Reductions in developmental myosin, a biomarker of muscle damage which was measured by muscle biopsy, and reductions in muscle inflammation, which were measured by magnetic resonance, in patients after 24 weeks of treatment with ezutromid, a potential first-inclass utrophin modulator.

The data was presented in a poster titled, “Ezutromid significantly reduced muscle damage whilst maintaining utrophin in patients with Duchenne muscular dystrophy (DMD) after 24-weeks of treatment,” and authored by authored by Professor Francesco Muntoni on behalf of the PhaseOut DMD Study Group.¹

PhaseOut DMD is an open-label, multi-center trial that has enrolled 40 patients between the US and UK, between ages 5 and 10 years. The Phase 2 study is 48 weeks in length, after which patients have the option of enrolling into an extension phase and continuing to be dosed with ezutromid.

The change from baseline in magnetic resonance parameters related to the leg muscles serves as the primary endpoint, and biopsy measures evaluating utrophin and muscle damage are included as secondary endpoints, with patients having 2 biopsies: the first at baseline and the second after either 24 or 48 weeks of treatment. Exploratory endpoints include the 6-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes.

This interim analysis suggests that ezutromid is reducing muscle damage and improving muscle health while also continuing to be safe and well-tolerated.

“The correlation observed between decreases in developmental myosin, a biomarker of muscle damage, and decreases in muscle fiber inflammation, is highly encouraging, and we believe further supports that ezutromid is breaking the DMD disease cycle of muscle damage and repair,” commented Dr David Roblin, Chief Medical Officer and President of R&D of Summit in a press release. “We look forward to reporting the full results of this trial, expected in the third quarter of 2018.”²

Top-line 48-week results are expected to be reported in the third quarter of 2018.

Summit is preparing for the week 48 readout, and accelerating activities to prepare for a Phase 3 placebo controlled trial that is expected to be initiated in 2019.

References:

1. Muntoni F, et al. Ezutromid significantly reduced muscle damage whilst maintaining utrophin in patients with Duchenne muscular dystrophy (DMD) after 24-weeks of treatment. Presented at: 70^th American Academy of Neurology (AAN) Annual Meeting; April 21-27, 2018; Los Angeles, California.
2. Summit Presents New 24-Week Analyses from PhaseOut DMD at the 2018 American Academy of Neurology Annual Meeting. https://www.summitplc.com/wp-content/uploads/2018/04/2018_RNS_20-AAN-data-FINAL.pdf. Accessed April 20, 2018.