GenSight Biologics has released final results from the REFLECT phase 3 clinical trial, confirming that its gene therapy, Lumevoq (lenadogene nolparvovec), provides sustained visual acuity improvement in patients with Leber Hereditary Optic Neuropathy (LHON) 5 years after a single administration. The study also found that bilateral injections resulted in a greater proportion of patients experiencing meaningful recovery compared to unilateral treatment.
The findings, presented by Patrick Yu-Wai-Man, MD, PhD, of the University of Cambridge, showed that 75% of patients who received bilateral treatment achieved clinically relevant visual recovery, compared to 60% of those treated unilaterally. Additionally, 79% of bilaterally treated patients were able to read letters on a vision chart, vs 72% in the unilateral group.
Evolution of visual acuity in the REFLECT phase 3 Study. Graphic courtesy Business Wire.
The safety profile remained consistent, with no serious ocular adverse events reported. Intraocular inflammation was the most common adverse event but was generally mild and responsive to standard treatment.
The REFLECT trial enrolled 98 patients with LHON caused by the ND4 gene mutation. All participants received an intravitreal injection of Lumevoq in the first affected eye, while the second eye was randomized to either a second Lumevoq injection or a placebo. The 5-year data reinforce the therapy’s potential as a long-term treatment for LHON, the investigators concluded.