Summit Therapeutics plc announced that its Phase 1b modified diet clinical trial of SMT C1100 for the treatment of Duchenne met its primary objective with half of the patients who received the higher dose of SMT C1100 achieving desired plasma levels while following specific dietary guidance. Based on these results, Summit will advance SMT C1100 into a Phase 2 open-label clinical trial. Congratulations to our friends at Summit and we look forward to hearing more about next steps!
Read Summit's Press Release:
Summit Therapeutics Announces Phase 1b Modified Diet Clinical Trial Achieves Primary Objective in DMD
- Primary Objective Met; Plasma Absorption of SMT C1100 Observed at a Level Suitable for Further Development
- SMT C1100 to Progress into Phase 2 Open-label Trial
- Conference Call Scheduled for 1:00pm BST / 8:00am EDT
Oxford, UK, 17 August 2015 - Summit Therapeutics plc (AIM: SUMM, NASDAQ: SMMT), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and C. difficile infection, announces that its Phase 1b modified diet clinical trial of SMT C1100 for the treatment of DMD met its primary objective with half of the patients who received the higher dose of SMT C1100 achieving desired plasma levels while following specific dietary guidance. Based on these results, Summit will advance SMT C1100 into a Phase 2 open-label clinical trial.
The Phase 1b clinical trial was designed to increase plasma levels of SMT C1100 in patients by recommending a diet with balanced proportions of fat, proteins and carbohydrates, combined with consuming a small glass of full fat, whole milk at the time of dosing. Initial analysis shows six of 12 patients achieved the desired plasma level after receiving a 2,500 mg dose of SMT C1100 twice daily for 14 days. In a prior Phase 1b trial, only two of 12 patients dosed with SMT C1100 achieved the desired plasma level. SMT C1100 was well tolerated at all doses tested in the modified diet trial with no serious adverse events reported. This outcome increases the human safety database for this investigational drug. SMT C1100 is a small molecule utrophin modulator being developed for the potential treatment of all patients with DMD. SMT C1100 has received orphan drug designation in the US and Europe.
"DMD has a devastating impact on its patients and families, and current treatment options are merely palliative in nature or are only applicable to small subsets of patients. A universal DMD treatment, like SMT C1100 if successfully developed, would have a broad impact for these patients and families, either alone or in combination with other DMD therapies," said Principal Investigator Professor Francesco Muntoni from Great Ormond Street Hospital, London. "I am, therefore, looking forward to future clinical studies with SMT C1100."
"These data clear an important hurdle in the development of SMT C1100 by demonstrating it is a viable drug candidate to bring proof-of-concept for the Company's burgeoning utrophin modulator pipeline and more importantly, hope to all patients and their families living with this devastating disease," said Glyn Edwards, Chief Executive Officer of Summit. "Our priority is advancing SMT C1100 efficiently through future patient clinical trials designed to evaluate the potential clinical benefit of this promising treatment, and this will start with an open-label Phase 2 trial that is expected to start by the end of this year."
The trial also measured enzyme biomarkers of muscle damage, such as creatine kinase. In this modified diet study, there was no change in the enzyme levels when patients received SMT C1100 compared to when they received a placebo. The Company plans to evaluate creatine kinase as well as additional biomarkers over a longer duration of exposure to SMT C1100 in the upcoming Phase 2 open-label trial.
Summit plans to commence the Phase 2 open-label trial during the fourth quarter of 2015. The trial will evaluate the longer-term benefits of SMT C1100 on muscle health, function and safety. Further details of the trial will be provided at a future date.