GenSight Biologics receives approval to include teenage patients in RESCUE and REVERSE Phase III trials with GS010 in Leber’s Hereditary Optic Neuropathy
Paris, France, September 8, 2016 – GenSight Biologics (Euronext: SIGHT, FR0013183985, PEA-PME eligible), a biopharma company that discovers and develops innovative gene therapies for neurodegenerative retinal diseases and diseases of the central nervous system, today announced that regulatory agencies and ethics committees in the United States, France and the United Kingdom, have authorized a protocol amendment to include teenage patients (15-18 years) in RESCUE and REVERSE, two ongoing Phase III clinical trials of GS010 in the treatment of Leber’s Hereditary Optic Neuropathy (LHON).
The Investigational New Drug (IND) Application had been cleared by the U.S. Food and Drug Administration (FDA) in August 2015, and Clinical Trial Applications (CTAs) had been accepted in France, Germany, Italy and the United Kingdom by national agencies in the first half of 2016, allowing GenSight Biologics to initiate the two Phase III clinical trials in the US and in Europe. The initial protocols were designed to enroll adult patients, whereas this amendment now allows inclusion of patients from the age of 15.
Bernard Gilly, Chief Executive Officer of GenSight Biologics, commented “Given the brutality of the onset of vision loss and the neurodegenerative nature of the disease, we are thrilled to be able to consider all patients irrespective of their age.”
“Considering that affected adolescents with LHON present in a similar fashion to adults with the disease, and the current good safety profile and potential prospect for benefit, it is extremely important to be able to include these teenage patients with LHON,” also commented Pr. Nancy J. Newman, MD, Director of the Section of Neuro-Ophthalmology, Emory University School of Medicine, Atlanta, Georgia (USA).
Available epidemiologic data suggest that teenage patients (15-18 years) may represent between 14 to 22% of all LHON patients (all mutations included).
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