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Figure 1. Evolution of BCVA In LUMEVOQ®-treated Patients (RESCUE/REVERSE/RESTORE) vs. Untreated Patients Note: The Locally Estimated Scatterplot Smoothing (LOESS) curves show the evolution of BCVA, from 12 months to 60 months after onset of vision loss, in all eyes (LUMEVOQ®- and sham-treated) from REVERSE / RESCUE / RESTORE studies and all eyes from a matched cohort of patients not treated with LUMEVOQ®. The shaded areas represent the 95% confidence interval for the BCVA values. The values >60 months were set to 60 months. The curve starts at 12 months after onset when 92.7% of eyes in RESCUE and REVERSE had received treatment, either with LUMEVOQ® or a sham injection. The untreated cohort consisted of 208 ND4-LHON patients that were followed in the REALITY registry and from two prospective and eight retrospective natural history studies. 6 * p0.01 for Kruskal-Wallis test and Repeated measures on patient. (Graphic: Business Wire)
GenSight Biologics (PARIS:SIGHT) ((Euronext: SIGHT, OTC:GSGTFPEA-PME eligible)), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today 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 patients1the findings are a significant divergence from the natural outcomes of LHON.
The data from RESTORE (CLIN06)2the long-term follow-up study to which all participants in the RESCUE3 and REVERSE4 Phase III pivotal trials were invited, also continued to show that the treatment is well-tolerated over the 5-year follow-up period.
5 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 (the “nadir”) 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. 5 years after treatment, the 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 (eg, composite score, mental health, role difficulties, dependency, near and distance activities, general vision, social functioning).
“The 5-year data from the RESTORE long-term extension study illustrates that the efficacy and safety data previously reported following treatment with a one-time injection of LUMEVOQ® is being maintained, providing hope to patients affected with this debilitating blinding disease” said Patrick Yu-Wai-ManMD, PhD, Professor of Ophthalmology and Honorary Consultant Neuro-ophthalmologist at the University of CambridgeMoorfields Eye Hospital, and the UCL Institute of…
Full story available on Benzinga.com
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