Santhera Pharmaceuticals AG (“Santhera”), a Swiss-based biopharmaceutical company focused on neuromuscular diseases, announced today that it has started a European Phase III clinical study with its lead product SNT-MC17 (idebenone) in Friedreich’s Ataxia (FRDA). The design of this study reflects the company’s discussions with EMEA which took place earlier this year and will evaluate both the cardiacand neurological benefits of SNT-MC17 (idebenone).
The Phase III trial with SNT-MC17 (idebenone) is a double-blind, randomised, placebo- controlled study to determine its efficacy, safety and tolerability in the treatment of Friedreich’s Ataxia patients with hypertrophic card iomyopathy. The study will be performed in Germany, the UK and the Netherlands with possible extension to other EU territories. It will involve approximately 10 study centers and about 200 patients are expected to be enrolled. Patients will receive SNT-MC17 (idebenone) for a period of one year. Recruitment of patients into the trial has already started.
The Phase III study will compare the efficacy of three different doses of SNT-MC17(idebenone) with placebo on the left ventricular mass index of FRDA patients. Hypertrophic cardiomyopathy, as measured by increased left ventricular mass indices is a prominent factor in both the morbidity and mortality of FRDA patients. In parallel this study will assess the efficacy of SNT-MC17 (idebenone) on the neurological symptoms of FRDA. This will be determined by measuring the changes in the International Cooperative Ataxia Rating Scale(ICARS).
In addition to the Phase III study, Santhera has an on-going collaboration with the National Institute of Health in the USA for FRDA and plans to initiate its own Phase III studies in the US next year for this indication with SNT-MC17 (idebenone). Santhera is also currently running a Phase IIa study in Belgium investigating the efficacy of SNT-MC17 (idebenone) for an additional neuromuscular disease indication, Duchenne Muscular Dystrophy.
Earlier this year, Santhera concluded a development and marketing agreement with Takeda Pharmaceuticals for SNT-MC17 (idebenone) in Friedreich ́s Ataxia. Under the terms of this agreement Takeda will support the overall clinical development of SNT-MC17 (idebenone) in FRDA and, upon approval, will be responsible for marketing the drug for this indication Friedreich’s Ataxia in all EU countries and Switzerland. Santhera will be fully responsible for development, registration and supply of the product. Santhera continues to own the rights to the product outside the EU and plans to market the product in the US under its own name. Santhera also retains all rights to other potential indications for SNT-MC17 (idebenone) such as Duchenne Muscular Dystrophy.
Thomas Meier, Ph.D., Chief Scientific Officer of Santhera commented: “SNT-MC17(idebenone) is the first compound with a clear beneficial pharmacological profile in FRDA and therefore has the potential to become the first-line treatment for Friedreich’s Ataxia, a
disease for which there is currently no effective treatment or cure available. The protocol of our Phase III trial takes into account our discussions with the EMEA earlier this year. We are very pleased to collaborate in this study with numerous distinguished physicians and clinicalexperts who have in the past already contributed to the understanding of the FRDA-pathology.”
Klaus Schollmeier, Ph.D., Chief Executive Officer of Santhera declared: “The start of the first Phase III trial with our lead compound SNT-MC17 is a key milestone in the development of Santhera Pharmaceuticals. Based on the growing pool of positive data supporting the product’s use in FRDA and the strong commitment of our partners including Takeda, the NIH, and patient organizations in the US and Europe we are confident that we will soon be in a position to help FRDA patients suffering from this devastating disease.