GenSight Biologics
Key Opinion Leaders Highlight GS010 Efficacy and Patient Benefits in Discussion of Findings from REVERSE Phase III Clinical Trial
Regulatory News:
GenSight Biologics (Paris: SIGHT)(Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported key highlights from its recent Key Opinion Leader (KOL) Event hosted in New York City on June 12 and dedicated to GS010 and the REVERSE Phase III clinical trial in the treatment of Leber Hereditary Optic Neuropathy (LHON).
The panel of medical experts included Nancy J. Newman, MD1, Robert C. Sergott, MD2, Mark Moster, MD3, and José-Alain Sahel, MD4. In addition, Lissa Poincenot, LHON patient advocate and mother of an LHON patient, presented the patient perspective on the results.
“This is revolutionary work that is being done,” said Dr. Sergott, who presented the study’s key findings. “We are witnessing the best of translational, precision medicine.” The topline findings on Spectral-Domain Optical Coherence Tomography (SD-OCT) parameters are “the first demonstrations of neuro-protection of neurons and of central nervous system axons in a human genetic disease,” according to Dr. Sergott.
Neuro-protection is an important outcome for LHON, a disease that in her disease overview, Dr. Newman had characterized as “a disease in motion” that inexorably resulted in bilateral vision loss in 97% of cases within a year of onset.
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The evidence that GS010 engaged its biologic targets was coupled with a functional outcome. Low-contrast sensitivity, measured using the Pelli-Robson chart, almost doubled in the GS010-treated eyes compared to sham-treated eyes. Dr. Sergott noted that low-contrast sensitivity is a better indicator of real-life visual function than the more widely-used measure of visual acuity, which is based on the number of letters that can be read on a Snellen chart and which measures high-contrast sensitivity. He added: “LHON patients treated with GS010 have a statistically significant improvement in their ability to discern subtle shades of gray, a very precise measure of visual function, the ability to see better in low light environments that approximate real life conditions, both indoors and outdoors.”