Collaboration
Orphan diseases specialist Santhera Pharmaceuticals has agreed a collaboration with Columbia University in New York to investigate Catena in a Phase II study as treatment of MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes).
MELAS is a rare but devastating multisystem disorder that affects the brain, nervous system, muscles and cognitive abilities. Currently, there are no approved therapies.
This announcement followed shortly after a similar collaboration between Santhera and the US National Institutes of Health to investigate Catena in Primary Progressive Multiple Sclerosis.
The MELAS trial is a Phase II, double-blind, placebo-controlled study investigating the safety and efficacy of two doses of Catena (idebenone) versus placebo. The primary endpoint of the proof-of-concept study is the change in cerebral lactate concentration from baseline to week four as assessed by Magnetic Resonance Spectroscopy.
Under the agreement, the Neurological Institute of Columbia University is conducting the clinical trial, while Santhera is supplying the study medication and contributing data management and other support. The company has exclusive rights to the data for regulatory purposes.
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