GenSight Biologics Confirms Sustained Efficacy and Safety of Bilateral LUMEVOQ Injections Four Years After One-Time Administration

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Graph 1: Evolution of Best-Corrected Visual Acuity (BCVA) Over Time – REFLECT Phase III Study (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 reported initial efficacy and safety results at 4 years^a post-treatment administration in the REFLECT Phase III clinical trial with LUMEVOQ (GS010; lenadogene nolparvovec). The results show that four years after a one-time administration of the gene therapy, the visual acuity improvement has been sustained while maintaining a favorable safety profile. Bilateral injection provides an additional effect compared to unilateral treatment, demonstrated across all analyses of visual acuity improvement and responder rates.

“The latest REFLECT data confirms that the improvement seen with lenadogene nolparvovec is sustained 4 years after treatment has been given, including the additional benefit observed in participants receiving a bilateral intravitreal injection of the gene therapy,” said Patrick Yu-Wai-Man, MD, PhD, Professor of Ophthalmology, University of Cambridge and Moorfields Eye Hospital, and International Principal Investigator of REFLECT. “Importantly, REFLECT participants receiving a bilateral injection had a comparable safety profile to those treated unilaterally.”

The findings reinforce the results observed at 3 years post-treatment administration, which were reported in March 2023.^b

Sustained and meaningful efficacy at Year 4

The evolution of the visual acuity over time shows that visual improvement after lenadogene nolparvovec treatment was maintained over 4 years in all subjects, with the visual acuity of bilateral injected patients remaining better than that of patients who received a unilateral injection. This difference has been observed since Year 1.5.

Graph 1: Evolution of Best-Corrected Visual Acuity (BCVA) Over Time – REFLECT Phase III Study

(See Graph 1)

Compared against nadir (i.e., the worst BCVA recorded from baseline to Year 4), average visual acuity for all LUMEVOQ-treated eyes increased beyond the +15 letter threshold that conventionally defines clinically meaningful improvement. The improvement of placebo eyes highlights the consistent contralateral treatment effect observed in all clinical trials (also documented in sham-treated eyes in the REVERSE¹ and RESCUE² trials).