REVERSE Phase III trial of GS 010 shows efficacy at 72 weeks for Leber Hereditary Optic Neuropathy.- GenSight Biologics.

GenSight Biologics reported additional results at Week 72 from the REVERSE Phase III clinical trial, which evaluates 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.

At 72 weeks, a clinically meaningful improvement from baseline in mean visual acuity of +15 letters (-0.294 LogMAR) was observed in GS010-treated eyes, with concomitant contralateral improvement of +12 letters (-0.246 LogMAR)1 in sham-treated eyes. This improvement, which extends the positive trend that had been reported at Week 48, points to a sustained functional outcome for the trial subjects. Continued improvement was also observed in contrast sensitivity as determined by Pelli-Robson low-contrast testing. At 72 weeks, GS010-treated eyes and sham-treated eyes gained on average +0.21 LogCS and +0.15 LogCS versus baseline, respectively. The proportion of treated eyes that achieved a clinically meaningful improvement of at least 0.3 LogCS (45.9%) was statistically significantly higher than that of sham-treated eyes (24.3%; p=0.0047). The visual function outcomes were accompanied by evidence that GS010 was engaging its anatomic targets, the ganglion cells.

At 72 weeks, high-resolution Spectral-Domain Optical Coherence Tomography (SD-OCT) objectively demonstrated sustained preservation of the retina anatomy relevant to LHON in GS010-treated eyes. The ganglion cell layer macular volume was preserved (+0.000 mm3) in treated eyes, while sham-treated eyes deteriorated from baseline (-0.044 mm3). The difference was statistically significant (p=0.0060). Drug-treated eyes also showed a limited loss in thickness of the temporal quadrant of the retinal fiber layer of -1.6 �m, compared to a loss of -3.6 �m in sham-treated eyes (p=0.0521).