Data from Santhera's Catena in Leber's Hereditary Optic Neuropathy Will Be Presented at the mitoNET Congress for Mitoch

Professor Thomas Klopstock, University of Munich, and investigator of the RHODOS study will present the data to an international audience of experts in mitochondrial medicine. He commented: "Catena holds the promise to protect LHON patients from deterioration of vision. The RHODOS-data establish the drug as a first treatment option for LHON. The trial also has key importance for the whole field of mitochondrial medicine as it shows that randomized, placebo-controlled studies can be conducted in these rare disorders. The energy defect of mitochondria may be tackled by drugs."

LHON is a rare genetic eye disease that leads to a rapid loss of central vision and ultimately to blindness as a result of nerve cell degeneration in the retina and optic nerve. Catena supports cellular energy production and has the potential to mitigate the effects of the genetic mutations which lead to blindness in LHON. The RHODOS study (Rescue of Hereditary Optic Disease Outpatient Study) was a randomized, double blind, placebo controlled trial testing the efficacy and safety of Catena and placebo over a six-month treatment period.

About Leber's Hereditary Optic Neuropathy
Leber's Hereditary Optic Neuropathy (LHON) is an inherited atrophy of certain cells in the retina and optic nerves that leads to rapid loss of central vision and ultimately to blindness. Blurring of central vision and color desaturation usually mark the beginning of the symptomatic phase of this neuro-ophthalmological disorder. The effects of LHON are rapid and severe, typically leading to blindness within a few months of the onset. While symptoms initially develop in one eye, the second eye is usually involved within a few months. Patients are predominantly young adult males who typically have one of three different point mutations of the mitochondrial genome. These mutations lead to the reduction in cellular energy production, which in turn results in cell damage and death of certain optic nerve cells.

Catena is the first drug that has ever been clinically investigated in a randomized, placebo-controlled intervention study in LHON. Given the drug's principal mode of action, Catena may protect the retinal and optic nerve cells and thereby delay, lessen or prevent vision loss. LHON is found in all ethnic groups; an estimated 20,000 patients live in Europe and in the United States.

About mitoNET
mitoNET is a German network of clinicians and scientists aiming for the improvement, the medical care and research in the field of mitochondrial diseases. Such disorders are rare and variable in severity and characterization. The mitoNET 2010 congress builds a bridge from the rapid scientific advances in this field to the clinically relevant aspects of these devastating diseases.

For further information, contact
Thomas Meier, Chief Scientific Officer Phone: +41 (0)61 906 89 64 thomas.meier@santhera.com

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