GenSight Biologics reports positive Phase III results in LHON

French firm GenSight Biologics (Euronext: SIGHT) has reported positive 96-week data from its late-stage, Phase III trial against LHON, a brutal inherited condition that often results in legal blindness for teens and young adults.

The trial evaluated the safety and efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in 37 subjects whose visual loss due to 11778-ND4 Leber Hereditary Optic Neuropathy (LHON) commenced between 6 and 12 months prior to study treatment. Week 96 is the last of the scheduled readouts for the trial and marks the time when the data are unmasked, providing access to individual patient profiles.

The results point to continued efficacy of GS010 two years past injection, with best-corrected visual acuity (BCVA) sustaining a clinically-meaningful improvement over baseline. At Week 96, GS010-treated eyes showed a mean improvement of -0.308 LogMAR compared to baseline, equivalent to +15.4 ETDRS letters or 3 lines on the ETDRS vision chart. This clinically-meaningful level of improvement in visual acuity maintains the gain observed at Week 72 (+14.7 ETDRS letters equivalent).