GenSight Biologics reports positive data from REVERSE Phase III trial
GenSight Biologics has reported further results from its REVERSE Phase III clinical trial for the treatment of Leber hereditary optic neuropathy (LHON).
GenSight Biologics has reported further results from its REVERSE Phase III clinical trial for the treatment of Leber hereditary optic neuropathy (LHON).
The trial analyses the safety and efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in 37 subjects suffering with visual loss due to 11778-ND4 LHON.
At 72 weeks, the study observed an improvement from baseline in mean visual acuity in eyes treated with GS010. Concomitant contralateral improvement was found to be of +12 letters (-0.246 LogMAR) in sham-treated eyes.
These results extend the positive trend reported at 48 weeks, indicating a sustained outcome for the trial patients.
An improvement was also seen in contrast sensitivity, as per the Pelli-Robson low-contrast testing.
In week 72, GS010-treated eyes gained on average +0.21 LogCS versus baseline, while sham-treated eyes had seen +0.15 LogCS.
"We’re seeing visual function continue to improve one and a half years after eyes are treated with GS010."
The proportion of treated eyes that experienced a clinical improvement of at least 0.3 LogCS (45.9%) was statistically higher than that of the sham-treated eyes (24.3%).
The outcome for visual function was also complemented by evidence that GS010 was engaging its anatomic targets, the ganglion cells.
High-resolution Spectral-Domain Optical Coherence Tomography (SD-OCT) demonstrated preservation of the retina anatomy at 72 weeks.
The ganglion cell layer macular volume was preserved in treated eyes, while it deteriorated in sham-treated eyes from baseline.
Drug-treated eyes also indicated a reduced loss in thickness of the temporal quadrant of the retinal fiber layer of -1.6µm, as against a loss of -3.6µm in sham-treated eyes.
GenSight co-founder and CEO Bernard Gilly said: “We’re seeing visual function continue to improve one and a half years after eyes are treated with GS010, and at the same time, objective tests continue to establish neuroprotection of the retina in treated eyes. This is, beyond any doubt, a great benefit for patients and their families."
"These results strengthen our determination to work with regulatory agencies to bring GS010 to market within our defined timelines."