GenSight’s Leber hereditary optic neuropathy treatment shows long-term positive visual acuity gains

A phase 1/2 clinical trial of GS010 showed visual acuity improvement in subjects with vision loss due to Leber hereditary optic neuropathy, according to a press release from GenSight Biologics.

A single intravitreal injection of GS010 was administered to the eye more severely affected by the disease in 15 subjects. At the time of treatment, subjects had an onset of vision loss of 6 years, according to the release.

Long-term follow-up, which is ongoing, showed relatively better vision at 2.5 years after injection for subjects who had an onset of vision loss of less than 2 years. Treated eyes had a mean gain of 28 ETDRS letters (–0.55 logMAR) compared with baseline, and untreated eyes had a mean gain of 13 ETDRS letters (–0.25 logMAR) compared with baseline, the release said. This difference was clinically significant.

A significant gain of 15 or more letters in treated eyes was demonstrated in 60% of subjects with vision loss of less than 2 years.

“Not only do we see a significant clinical benefit in recently diagnosed subjects, but this benefit is sustained after 2.5 years with a single injection of GS010,” Bernard Gilly, CEO and co-founder of GenSight, said in the release. “This is particularly encouraging, as we are now less than 6 months away from phase 3 efficacy data with RESCUE and REVERSE.”

A phase 1/2 clinical trial of GS010 showed visual acuity improvement in subjects with vision loss due to Leber hereditary optic neuropathy, according to a press release from GenSight Biologics.

A single intravitreal injection of GS010 was administered to the eye more severely affected by the disease in 15 subjects. At the time of treatment, subjects had an onset of vision loss of 6 years, according to the release.

Long-term follow-up, which is ongoing, showed relatively better vision at 2.5 years after injection for subjects who had an onset of vision loss of less than 2 years. Treated eyes had a mean gain of 28 ETDRS letters (–0.55 logMAR) compared with baseline, and untreated eyes had a mean gain of 13 ETDRS letters (–0.25 logMAR) compared with baseline, the release said. This difference was clinically significant.

A significant gain of 15 or more letters in treated eyes was demonstrated in 60% of subjects with vision loss of less than 2 years.

“Not only do we see a significant clinical benefit in recently diagnosed subjects, but this benefit is sustained after 2.5 years with a single injection of GS010,” Bernard Gilly, CEO and co-founder of GenSight, said in the release. “This is particularly encouraging, as we are now less than 6 months away from phase 3 efficacy data with RESCUE and REVERSE.”