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SMT C1100 Preliminary Results from Phase 1B Clinical Trial for Treatment of Duchenne

In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profile in twelve boys with Duchenne. Plasma levels of the drug were variable and the company speculates that diet and the course of the disease may be the cause.

The preliminary trial data will be reviewed further by Summit and is expected to lead to a revision of future clinical trial plans in order to determine the optimal way, either through dietary means or drug formulation changes, to address the drug uptake differences between Duchenne patients and healthy volunteers. The next patient study is now expected to start in Q4 2014.

"Our goal with utrophin modulation is to treat all patients with DMD. Armed with a greater understanding of the importance of diet and other disease related factors, our future clinical trial plans will be modified and will increase the potential likelihood of achieving this goal."
--Glyn Edwards, Chief Executive Officer of Summit

Summit will host a webinar next week to discuss this news and answer your questions - more details to come. For now though, you can submit your questions to info@parentprojectmd.org, RE: SUMMIT.



Read Summit's Announcement:

SUMMIT REPORTS PRELIMINARY RESULTS FROM PHASE 1B CLINICAL TRIAL OF SMT C1100 FOR DUCHENNE MUSCULAR DYSTROPHY

  • Safe and well tolerated at all doses tested in the study
  • Reduction in CK levels, an enzyme associated with muscle damage
  • Next patient clinical trial expected to start in Q4 2014

Oxford, UK, 21 May 2014 - Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy ('DMD') and C. difficile infection, announces that SMT C1100 for the treatment of DMD has successfully met its primary endpoint of safety and tolerability in a Phase 1b clinical trial in patients with the disease.  SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease.

The Phase 1b dose-escalating trial was conducted in 12 patients with DMD aged between 5 and 11 years old.  These preliminary results show that SMT C1100 was safe and well tolerated at all doses tested in the study, and that there were no issues with patient compliance.  All the boys had variable blood plasma concentrations of SMT C1100 with only two of the boys achieving concentrations similar to those of the adult volunteers in the 2012 Phase 1 study.  Initial evidence suggests that the variability in drug uptake may be due to differences in diet and to other disease-related factors.

The non-placebo controlled trial also measured creatine kinase ('CK') levels, an enzyme that is associated with muscle fibre damage and elevated in boys with DMD.  In the majority of patients there was a reduction in CK levels during dosing with SMT C1100.  These data are consistent with non-clinical in vivo efficacy studies in the mdx model of DMD that showed SMT C1100 reduced CK levels after only 15 days. 

"This first-ever trial of a utrophin modulator in DMD patients demonstrated the excellent safety profile of SMT C1100 with the study successfully achieving its primary endpoint," commented Glyn Edwards, Chief Executive Officer of Summit.  "In addition, the positive impact on the enzyme markers of muscle health was both unexpected and exciting with these data potentially representing SMT C1100's first signs of activity in DMD patients."

These preliminary trial data will be reviewed further by Summit and is expected to lead to a revision of future clinical trial plans in order to determine the optimal way, either through dietary means or drug formulation changes, to address the drug uptake differences between DMD patients and healthy volunteers.  The next patient study is now expected to start in Q4 2014.

Glyn Edwards continued, "Our goal with utrophin modulation is to treat all patients with DMD.  Armed with a greater understanding of the importance of diet and other disease related factors, our future clinical trial plans will be modified and will increase the potential likelihood of achieving this goal.

Webcast Presentation
A webcast presentation by Glyn Edwards on the preliminary Phase 1b results is available by clicking on the following link: http://www.brrmedia.co.uk/event/123676?popup=true

About the Phase 1b Clinical Trial
The Phase 1b trial was a dose-escalating, open-label study conducted in paediatric patients with DMD to evaluate safety, tolerability and drug exposure.  The trial enrolled 12 patients aged between 5 and 11 years, divided equally into three dose cohorts.  Each cohort received daily oral doses of SMT C1100 for a total of ten days with a dose escalating safety review taking place after each cohort had completed dosing.  The trial was conducted at four NHS hospitals in the UK with the Chief Investigator being Professor Francesco Muntoni at Great Ormond Street Hospital, London.   Further information about the trial is available at www.clintrial.gov.

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