Santhera Pharmaceuticals AG (“Santhera”) of Switzerland announced today the start of a collaborative clinical trial with the US National Institutes of Neurological Disorders and Stroke (NINDS) at the National Institute of Health (NIH) to evaluate SNT-MC17 (idebenone) in patients affected by Friedreich’s ataxia (FRDA), a devastating life-threatening neuromuscular disease.
This phase II study is designed to provide data on the safety and efficacy of SNT-MC17 in FRDA patients at various doses. These results will supplement previous clinical work which suggested that the product is efficacious in treating the cardiac complications that are the main cause of death in FRDA patients.
Under the collaboration the NIH will conduct the clinical trial, which is expected to enroll 48 patients with FRDA and last six months. Santhera will supply the drug material to be tested and contribute to the data management and analysis as well as regulatory support. The primary endpoint of the study is the reduction of oxidative stress markers. Importantly, this study also aims to analyze changes in several neurological and functional parameters as a result of treatment with SNT-MC17. These parameters will be secondary endpoints of the study. Data obtained from this study will be used as part of the development program for SNT-MC17 (idebenone) both in the US and in Europe; Santhera plans to start a pivotal Phase III trial in Europe later this year.
Nicolas Di Prospero MD, who is the principal investigator at NINDS, declared: “Friedreich’s ataxia is a life-threatening disease for which no effective treatment is approved. Previous studies have suggested that idebenone can slow or arrest the development of hypertrophic cardiomyopathy and may also have beneficial effects on disease-specific neurological symptoms. By conducting this study, we hope to get an indication of whether neurological benefits are conferred by idebenone/SNT-MC17 in FRDA patients.”
Mr. Ron Bartek, President of the Friedreich’s Ataxia Research Alliance (FARA), the patient organization in the US dedicated to supporting efforts to find treatments for FRDA concluded: “We are pleased that Santhera and the scientists at NINDS have teamed up to accelerate the clinical development and finally approval of SNT-MC17 (idebenone) which is currently the only pharmacological treatment option with clear proof of concept in clinical trials.”
Thomas Meier, Ph.D., Santhera’s Chief Scientific Officer commented: “We are very pleased to be collaborating with Dr. Di Prospero and his colleagues at the NIH, who have experience with this drug from previous Phase I trials. In this new trial, we will evaluate the neurological impact of our lead compound SNT-MC17 (idebenone); this is an important additional step in determining its full benefit to FRDA patients. Independently, we will enroll our Phase III clinical program in Europe and will subsequently commence a Phase III study in the US.”