Summit Announces New Analysis Showing Ezutromid Significantly Reduced Muscle Inflammation in Phase 2 Clinical Trial in DMD

Summit has announced additional positive data from its PhaseOut DMD clinical trial today, showing a significant reduction in muscle inflammation after 24 weeks of ezutromid dosing. The findings in their 24-week interim data are consistent with the expected activity of ezutromid to stabilize muscle fiber membranes and thereby reduce muscle fiber damage and inflammation. A statistically significant and meaningful reduction in muscle fiber damage was observed in previously reported 24-week findings from patient biopsies in PhaseOut DMD, and now Summit is reporting a decrease in muscle inflammation.

Read the announcement from Summit: 


Summit Announces New Analysis Showing Ezutromid Significantly Reduced Muscle Inflammation in Phase 2 Clinical Trial in DMD

  • MRS-T2 Data Provide Evidence of Early Impact of Ezutromid on Downstream Muscle Health
  • Data Build on Positive Biopsy Findings that Showed Significant Decrease in Muscle Damage


OXFORD, United Kingdom, Feb. 26, 2018 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM) announces further positive findings from PhaseOut DMD, a Phase 2 open-label, multi-centre clinical trial of the utrophin modulator ezutromid in Duchenne muscular dystrophy (‘DMD’). Further analysis of the 24-week interim dataset showed a statistically significant decrease in muscle inflammation as measured by magnetic resonance spectroscopy transverse relaxation time T2 (‘MRS-T2’).


“MRS-T2 is an objective technique used to monitor DMD disease progression as it allows for the precise quantification of changes in muscle breakdown and inflammation. MRS-T2 values typically increase over time in DMD,” 
commented Dr H Lee Sweeney, Director of the Myology Institute at the University of Florida and Co-Director of Imaging DMD. “The decrease in MRS-T2 seen in PhaseOut DMD is encouraging and suggests ezutromid is having a positive effect on muscle health. These data could be an early indication that these patients are experiencing a decrease in disease severity and highlight ezutromid’s potential as a disease modifying treatment. I look forward to seeing further findings from PhaseOut DMD.”


The reduction in MRS-T2 measured in PhaseOut DMD is consistent with the expected activity of ezutromid to stabilise muscle fibre membranes and thereby reduce muscle fibre damage and inflammation. A statistically significant and meaningful reduction in muscle fibre damage was observed in previously reported 24-week findings from patient biopsies in PhaseOut DMD.


Published research has shown reductions in MRS-T2 in DMD patients treated with steroids.[1]All patients in PhaseOut DMD have been on stable steroid regimens and therefore the MRS-T2 reductions observed are in addition to any anti-inflammatory effect provided by steroids.


The new MRS data showed a statistically significant decrease from baseline in the T2-relaxation time in the soleus (calf muscle) in patients (n=38) treated with ezutromid. The mean decrease was -0.861 milliseconds from baseline to 24 weeks (31.850 milliseconds to 30.989 milliseconds, 95% CI, -1.440, -0.281). The soleus is one of the most reliable leg muscles for monitoring disease progression via T2 relaxation time in DMD with increases shown to correlate to loss of functional ability.[1,2] Published natural history data show that T2 relaxation times increase with disease progression due to the relentless cycle of muscle damage and repair leading to inflammation.[1,2] A mean decrease of -0.470 milliseconds in MRS-T2 was also observed in the vastus lateralis (thigh muscle) in ezutromid-treated patients (n=37) from baseline to 24 weeks (32.265 milliseconds to 31.795 milliseconds, 95% CI, -1.158, 0.218).


“The 24-week interim analysis has shown encouraging signs of ezutromid activity in PhaseOut DMD. These MRS-T2 findings show a positive impact on downstream muscle health,” 
added Dr David Roblin, Chief Medical Officer and President of R&D at Summit. “This, combined with the evidence that ezutromid can modulate production of utrophin protein and significantly reduce muscle damage, is further evidence of the potential of ezutromid as a disease modifying approach for the treatment of all genetic forms of DMD.”


[1] Arpan et al., Neurology, 2014, 83: 974-980. 
[2] Willcocks et al., Neuromuscul Disord. 2014, 25(5): 393-401.



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