They will shortly start the phase 2 as an open label study in both the US and EU
SUMMIT THERAPEUTICS PRESENTS ADDITIONAL DATA FROM PHASE 1B MODIFIED DIET CLINICAL TRIAL OF SMT C1100 IN DMD PATIENTS AT WMS
Oxford, UK, 30 September 2015 - Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, will today present detailed pharmacokinetic data from the Phase 1b modified diet clinical trial of SMT C1100 for the treatment of DMD, as well as preclinical data supporting both SMT C1100 and the broader utrophin modulator pipeline, at the 20th International Congress of the World Muscle Society ('WMS'). SMT C1100 is a utrophin modulator that the Company believes has potential to treat all patients with DMD.
"We believe utrophin modulation has significant potential as a universal treatment for DMD. For example, in preclinical studies, continual production of utrophin has resulted in disease-modifying effects in DMD models.Those preclinical findings, together with the detailed pharmacokinetic data from this recent trial, further our belief that SMT C1100 could have clinical benefit across all patients with DMD," said Ralf Rosskamp, MD, Chief Medical Officer of Summit. "We are thrilled to have the opportunity to provide hope for the patients and families living with this devastating disease, and look forward to initiating a Phase 2 trial, which aims to provide proof of concept for SMT C1100 through measurements of muscle health and function, in the fourth quarter of this year."
As previously reported, the Phase 1b modified diet trial met its primary objective. In this trial, increases in plasma absorption of SMT C1100 were observed in DMD patients who received SMT C1100 while following specific dietary guidance providing for a balanced diet of fats, proteins and carbohydrates. In the detailed pharmacokinetic data to be presented at WMS, ten of 12 patients achieved plasma exposure levels above 30 ng/ml for a mean of 14 hours in a 22-hour period, and six of those patients achieved plasma exposure levels above 67 ng/ml for a mean of 8.2 hours in the 22-hour period. These plasma levels correlate to an in vitro increase in utrophin levels of approximately 30% and 50%, respectively, in DMD myoblast cells and human myotubes. Since sustaining utrophin production is likely to be key in providing therapeutic benefit, the remaining two patients may also achieve some clinical benefit as they had plasma levels that exceeded 20 ng/ml. Summit's upcoming Phase 2 proof of concept trial will evaluate the potential benefit of SMT C1100 with longer-term dosing. Based on the detailed analysis of the Phase 1b modified diet trial, drug plasma exposure will not be used as an entry criterion for the Phase 2 proof of concept trial.
Summit plans to initiate its Phase 2 proof of concept trial of SMT C1100 in patients with DMD in the fourth quarter of this year. The 48-week open-label study is expected to enroll up to 40 patients between the ages of five and 10 years old at sites in Europe and in the US. Planned endpoints are expected to include changes in the fat content of muscle and in muscle inflammation as measured by magnetic resonance imaging ('MRI') of the leg muscles, measurements of utrophin protein and muscle fibre regeneration from muscle biopsies, and functional measures such as the six minute walk test and the North Star Ambulatory Assessment. The Company expects to report data from this trial periodically over the course of the study. The design and initiation of this study remain subject to regulatory approval. In addition, Summit continues to evaluate the timing and design for a larger, placebo-controlled trial of SMT C1100.
Additional Highlights from the WMS Presentations
SMT C1100 Clinical Development - Phase 1b Modified Diet Clinical Trial
Biomarker Development Programme
Second Generation Utrophin Modulator Pipeline Development
Copies of the posters are now available from the 'Programmes' section of Summit's website, www.summitplc.com.
Do you have any specific info more than this? I had heard it was to be 4th qtr this year, but know nothing more specific.
Lori, no more than is in the above where they say 4th Qtr
Yeah, I am literally holding my breath for tomorrow (4th qtr begins!). They released the last results 6 weeks early, so I am hoping we will hear about this EARLY into Q4!