GenSight Biologics (Euronext: SIGHT), a biopharmaceutical company that develops and commercialises gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced on Wednesday that it can confirm sustained efficacy and safety of bilateral LUMEVOQ (GS010; lenadogene nolparvovec) injections four years after one-time administration.

In the company's REFLECT Phase III trial, patients experienced clinically meaningful visual improvement, with 73% of bilaterally treated patients showing significant benefit. The results, reported on 20 February 2024, demonstrate that the visual acuity improvement remains consistent over time. Bilateral injection yielded additional benefits compared to unilateral treatment, with patients experiencing at least a 15-letter improvement relative to their observed nadir.

The findings underscore the importance of LUMEVOQ as a durable treatment option for retinal neurodegenerative diseases. Laurence Rodriguez, CEO of GenSight Biologics, emphasized the significance of sustained vision improvement from a single administration, which enhances patient adherence and quality of life.

The REFLECT trial, involving 98 subjects with Leber Hereditary Optic Neuropathy (LHON), confirmed a favorable safety profile for LUMEVOQ at the four-year mark. Adverse events were mostly mild, with no study discontinuations related to systemic or ocular issues.

The efficacy of LUMEVOQ was particularly notable in bilaterally treated patients, where 73% experienced clinically meaningful improvement, and 81% were able to read letters on a screen. This underscores the potential of bilateral treatment in enhancing patient outcomes.

A randomised, double-masked, placebo-controlled Phase III study, the REFLECT trial aimed to evaluate the safety and efficacy of bilateral injections of GS010 in LHON patients. It involved multiple centres across Europe/UK, the US and Taiwan, with subjects receiving intravitreal injections of LUMEVOQ.

Long-term follow-up will continue to monitor the efficacy and safety of LUMEVOQ over time, offering hope for patients suffering from LHON and other retinal diseases.