GenSight Biologics Announces Initial Results from New Meta-Analyses on Visual Outcomes with LUMEVOQ Gene Therapy at NANOS 2024

Regulatory News:

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240311531952/en/

Figure 1. Visual Recovery (CRR from Nadir) Among ND4 LHON Patients – Results from Three New Meta-Analyses (Graphic: Business Wire)

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced initial results of new meta-analyses in Leber Hereditary Optic Neuropathy (LHON), which show those treated with LUMEVOQ (GS010; lenadogene nolparvovec) gene therapy experienced a rate of visual recovery greater than that of idebenone-treated patients and untreated (natural history) patients. The meta-analyses are the first to focus solely on patients with the m.11778G>A ND4 mutation, which is the most common mutation and one with a poor visual prognosis¹.

Figure 1. Visual Recovery (CRR¹ from Nadir²) Among ND4 LHON Patients – Results from Three New Meta-Analyses^3,4

(See Figure 1)

The meta-analyses depict a gradient of efficacy of visual recovery with LUMEVOQ intravitreal gene therapy resulting in greater recovery rates than that of idebenone treatment, and both greater than that in the natural history of the disease. This gradient of recovery, based on the CRR measure of visual improvement, is observed at both eye level and patient level (response in one or both eyes). There is no overlap in confidence intervals when LUMEVOQ is compared to idebenone and to natural history, indicating a positive difference in visual outcomes.

The results were presented at the 2024 annual meeting of the North American Neuro-Ophthalmology Society (NANOS) and will also be presented at other major ophthalmology, neurology and neuro-ophthalmology medical conferences in Europe and the US later this year.

Dr. Nancy J. Newman, LeoDelle Jolley Professor of Ophthalmology and Neurology at the Emory University School of Medicine in Atlanta, United-States, presented the findings at NANOS 2024. She noted, “This study is very important in order to compare these therapies’ abilities to achieve equivalent end-point metrics. It is clear that the degree of efficacy of the gene therapy was greater than the other currently available options of no treatment and idebenone use as previously administered in studies.”