Data from RESTORE study shows Lumevoq improves vision in Leber hereditary optical neuropathy.
GenSight Biologics reported that after 5 years of follow-up, Leber Hereditary Optical Neuropathy (LHON) subjects treated with Lumevoq (GS010) continued to experience significantly improved vision as a result of a one-time injection of the gene therapy treatment.
Compared to the trend in vision observed among untreated patients, the findings are a significant divergence from the natural outcomes of LHON. The data from RESTORE (CLIN06), the long-term follow-up study to which all participants in the RESCUE3 and REVERSE4 Phase III pivotal trials were invited, also continue to show that the treatment is well-tolerated over the 5-year follow-up period.
FIve years’ data on efficacy and safety shows substantial durability evidence and is more extensive than what is typically submitted in a data package for a gene therapy. When RESTORE subjects enrolled in the study 2 years after the one-time injection, they had already experienced clinically meaningful improvements relative to the lowest point of their best-corrected visual acuity (BCVA): +18.8 ETDRS letters equivalent in their Lumevoq-treated eyes and +17.3 letters equivalent in their sham-treated eyes. FIve years after treatment, the bilateral improvement from nadir was sustained, with Lumevoq-treated eyes achieving a mean improvement against nadir of +22.0 letters equivalent and sham-treated eyes demonstrating a mean improvement of +19.5 letters equivalent.
The impact of such results on patients is demonstrated by increases in the self-reported quality of life (QoL) scores at Year 5 vs. baseline. Mean overall QoL increased by a clinically meaningful magnitude relative to baseline, driven by increases in the sub-scores corresponding to mental health and the ability to carry out activities autonomously (e.g., composite score, mental health, role difficulties, dependency, near and distance activities, general vision, social functioning).
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