Visual acuity gains reported in phase 3 trial of Leber hereditary optic neuropathy treatment

GenSight Biologics’ gene therapy treatment candidate for Leber hereditary optic neuropathy showed continuous recovery of visual function in a phase 3 trial, according to a press release.

Best corrected visual acuity improved by an equivalent of 21 ETDRS letter from nadir between weeks 48 and 72 in eyes treated with GS010 (rAAV2/2-ND4) in the 39-patient RESCUE trial.

Sham-treated eyes has a BCVA evolution of 21.7 ETDRS letters, so a statistically significant difference in visual acuity between GS010 and sham-treated eyes could not be shown, the release said.

“We are excited and extremely gratified to see a picture of a sustained recovery of visual function emerge from RESCUE results at week 72,” Bernard Gilly, co-founder and CEO of GenSight, in the release. “The findings continue to be consistent with what was observed in REVERSE at 48 and 72 weeks as well as with RESCUE at 48 weeks, and bolster our confidence in the benefits that GS010 can deliver to patients and motivate us to work with the authorities to bring GS010 as early as possible to the market.”

The trial evaluated GS010 in patients within 6 months of vision loss due to LHON. The parallel REVERSE trial is evaluating its efficacy in patients with an onset of vision loss within 7 to 12 months.

GenSight Biologics’ gene therapy treatment candidate for Leber hereditary optic neuropathy showed continuous recovery of visual function in a phase 3 trial, according to a press release.

Best corrected visual acuity improved by an equivalent of 21 ETDRS letter from nadir between weeks 48 and 72 in eyes treated with GS010 (rAAV2/2-ND4) in the 39-patient RESCUE trial.

Sham-treated eyes has a BCVA evolution of 21.7 ETDRS letters, so a statistically significant difference in visual acuity between GS010 and sham-treated eyes could not be shown, the release said.

“We are excited and extremely gratified to see a picture of a sustained recovery of visual function emerge from RESCUE results at week 72,” Bernard Gilly, co-founder and CEO of GenSight, in the release. “The findings continue to be consistent with what was observed in REVERSE at 48 and 72 weeks as well as with RESCUE at 48 weeks, and bolster our confidence in the benefits that GS010 can deliver to patients and motivate us to work with the authorities to bring GS010 as early as possible to the market.”

The trial evaluated GS010 in patients within 6 months of vision loss due to LHON. The parallel REVERSE trial is evaluating its efficacy in patients with an onset of vision loss within 7 to 12 months.