Santhera: Start of US phase III clinical trial with SNT-MC17
Fast-track designation granted by FDA.
Santhera Pharmaceuticals announced that it has reached an agreement with the US food and Drug Administration (FDA) under the Special Protocol Assessment (SPA) procedure relating to the Phase III clinical trial to evaluate SNT-MC17 (INN: idebenone) for the treatment of Friedreich's ataxia (FRDA). The protocol incorporates advice provided by the FDA on the design of the study, its endpoints, statistical analysis and conduct. The FDA granted a fast track designation to Santhera's compound in FRDA.
The positive clinical results from a recently reported study conducted in collaboration with the US National Institutes of Health (NIH) formed the basis for the design of the phase III trial. The trial with SNT-MC17, named IONIA (Idebenone effects On Neurological ICARS Assessments), is a double-blind, randomized, placebo-controlled study of six months duration investigating the efficacy of two doses of SNT-MC17 compared to placebo. The primary endpoint of IONIA will be a neurological endpoint, measured by the International Cooperative Ataxia Rating Scale (ICARS), comparing the change in the ICARS for each of the treatment groups with placebo over the 24 week study period. The study will also investigate additional neurological endpoints as well as activities of daily living parameters and cardiac measures.
Based on the efficacy data obtained in the NIH study, particularly for the neurological outcome measures, Santhera's first dose group in the IONIA trial will be 450 mg/day for patients below 45 kg body weight and a corresponding dose of 900 mg/day for patients above 45 kg body weight. The second dose group will be 1350 mg/day for patients below 45 kg of body weight and 2250 mg/day for patients above 45 kg. Using Santhera's 150 mg tablet, the daily dose of SNT-MC17 will be divided into three equal doses to be taken with a meal.
The IONIA study will recruit a minimum of 51 ambulatory FRDA patients between the ages of 8 and 17 years and will be conducted at two clinical centers in the US - the Children's Hospital of Philadelphia and the School of Medicine of the University of California, Los Angeles. Patient recruitment is expected to start soon. Santhera has committed to an open label extension study of 12 months duration offering FRDA patients who have enrolled and completed the IONIA trial, the continuation of treatment with SNT-MC17 at the 1350/2250 mg/day dose level. The purpose of this extension study is to generate additional safety and tolerability data on SNT-MC17 in longer use.
In response to FDA's advice under the SPA, Santhera has, to the extent possible, incorporated the Agency's recommendations into the design of the protocol for this pivotal clinical trial. Discussion points with the FDA have been the size of the study and the safety data base regarding the high dose at the time of filing for a new drug application (NDA). Santhera acknowledges both aspects as they are resulting primarily from the orphan nature of the disease.
Furthermore, the FDA granted a fast-track designation for the compound in FRDA that allows a rolling submission of the NDA enabling the Agency to review the product's NDA with a higher priority in a potentially shorter review time. The compound earlier received orphan drug designation for the indication FRDA in the US, allowing for a market exclusivity of 7 years after marketing approval.
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