Positive long-term results reported for Leber’s hereditary optic neuropathy treatment

GenSight Biologics has reported that GS010, an intravitreal injection treatment for Leber’s hereditary optic neuropathy, has shown positive long-term visual acuity and safety results after 96 weeks, according to a company press release.

In the phase 1/2 trial, a single intravitreal injection of GS010 was administered in the eye most severely affected by the disease.

In patients with an onset of vision loss of less than 2 years, there was a mean gain of 29 ETDRS letters compared with baseline in the treated eye and a mean gain of 15 letters in the untreated eye, according to the release.

The mean change in visual acuity from baseline was statistically significant in the treated eye of all patients.

“We continue to see a sustained clinical benefit for patients in this long-term follow-up after 2 years with a single injection, and this is very encouraging,” Bernard Gilly, GenSight CEO and co-founder, said in the release. “Furthermore, this data continues to support the design of our two phase 3 studies with GS010 for the treatment of Leber’s hereditary optic neuropathy, which are currently ongoing in the U.S. and Europe, addressing patients with an early onset of vision loss. We are now less than a year away from phase 3 efficacy data.”

Regarding safety, there were no reports of vision worsening or ocular sequelae, serious treatment-emergent adverse events or systemic adverse events.