Therapy Areas: Central Nervous System
GenSight Biologics Claims Sustained Efficacy and Safety at 96 Weeks in RESCUE Phase III Clinical Trial of GS010 for the Treatment of Leber Hereditary Optic Neuropathy
27 September 2019 - - French biopharma company GenSight Biologics (PAR: SIGHT) (Euronext: SIGHT, ISIN: FR0013183985) has reported the first set of results from Week 96 of the RESCUE Phase III clinical trial.

The trial evaluated the efficacy and safety of a single intravitreal injection of GS010 (rAAV2/2-ND4) in 39 subjects whose visual loss due to 11778-ND4 Leber Hereditary Optic Neuropathy commenced up to 6 months prior to study treatment.

Week 96, which marks the time when individual patient profiles can be analyzed, is the last of the scheduled readouts for the RESCUE trial and completes the data collection from GS010's pivotal trials in Europe.

The results point to continued efficacy of GS010 two years past injection, with best-corrected visual acuity sustaining a clinically meaningful improvement over nadir.

Having been treated early in the course of the disease, RESCUE patients' vision initially deteriorated to a worst point, or nadir, before beginning to recover.

A mixed model of analysis of covariance (ANCOVA) was used with change from baseline as the response, and subject, eyes of the subject as random factor, treatment and the baseline LogMAR value as covariates in the model.

When visual acuity is measured from the post-baseline nadir, the visual acuity of GS010-treated eyes in fact recovered significantly from the worst BCVA reading post-baseline.

GS010-treated eyes regained more than two-thirds of the initial loss occurring in the most acute phase of the disease.

This improvement from nadir (-0.498 LogMAR mean improvement, or +24.9 ETDRS letters equivalent) corresponds to five lines of Snellen acuity and is far above the 3-line threshold commonly accepted as a clinically meaningful level of visual improvement.

Such recovery of vision is unprecedented in any gene therapy trial. Moreover, these results demonstrate the durability of improvement seen in earlier readouts of this trial.

GenSight is planning to schedule a pre-submission meeting with the EMA in early 2020 and expects to submit application for marketing approval in Europe in 3Q20.

An End of Phase II meeting with the US Food and Drug Administration has been requested and is expected for November 2019.

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.