GenSight Biologics Announces Update on Real-World Data from Early Access Programs of LUMEVOQ Gene Therapy at NANOS 2024

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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 that recent prospective real-world data from early access programs (EAP) confirm the benefit of LUMEVOQ in patients with Leber Hereditary Optic Neuropathy from the ND4 mutation (ND4-LHON), as observed in clinical trials.

One year after treatment, eyes of bilaterally injected patients experienced an average gain from nadir¹ in best-corrected visual acuity (BCVA) equal to +23 ETDRS letters, greater than the 18-letter average gain among patients with only one eye injected. In both groups, the clinical improvement exceeded the conventional definition of clinical meaningfulness, in which a 15-letter gain is considered meaningful. The safety profile of the gene therapy continues to be favorable and comparable between bilaterally treated patients and unilaterally treated patients.

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 medical conferences in Europe and the US later this year. Dr. Chiara La Morgia, MD, PhD, of the IRCCS Institute of Neurological Sciences of Bologna and the University of Bologna, Italy, presented the results at NANOS and commented, “It is very reassuring to clinicians that as successive waves of data become available, we continue to obtain a consistent picture of significant visual improvement among most patients treated with lenadogene nolparvovec. The evidence is becoming more robust, which is relevant for patients who are struck by a severe blinding disease such as LHON.”

These latest analyses are able to draw upon a more robust sample than the early view of the data released in March 2023². In particular, the improvements at one year post-treatment and beyond provide a more definitive view of the upward trajectory of mean visual acuity (see Figure 1).

One year after treatment, responder analyses show that the visual improvements accrue to the majority of patients: 63.2% of the eyes of bilaterally injected patients reached a clinically meaningful level of improvement in BCVA (≥0.3 LogMAR or +15 ETDRS letters), as did 57.9% of eyes of unilaterally injected patients. Nine months after injection, the average visual acuity corresponded to on-chart vision, compared to off-chart average visual acuity at baseline.