Phase III clinical trial of Lumevoq in Leber Hereditary Optic Neuropathy published in Ophthalmology journal.- GenSight Biologics

GenSight Biologics announced that the journal of the American Academy of Ophthalmology, Ophthalmology, has published results from the RESCUE pivotal Phase III clinical trial of Lumevoq gene therapy in ND4 Leber Hereditary Optic Neuropathy (LHON) subjects. The paper published in the January issue is the second peer-reviewed article based on Phase III clinical trial data to document comparable bilateral improvement in visual outcomes from a unilateral injection of a gene therapy. Efficacy analysis from 38 subjects1 show that best-corrected visual acuity (BCVA) evolved along parallel trajectories for Lumevoq-treated and sham-treated eyes, deteriorating to the worst levels at Week 24, followed by a plateau phase until Week 48, then showing improvement up to Week 96. By Week 96, average change against the worst recorded BCVA, or nadir, was -0.53 LogMAR (+26 ETDRS letters equivalent) in Lumevoq-treated eyes and -0.46 LogMAR (+23 ETDRS letters equivalent) in sham-treated eyes. This improvement was statistically significant in both eye groups (p<0.0001). at week 96, 71% of subjects had an improvement of at least -0.3 logmar (+15 etdrs letters equivalent) from the nadir in at least one eye and 71% of subjects had clinically relevant recovery (crr) from nadir in at least one eye. crr, a measure of treatment response established by an international consensus meeting on the management of lhon2, is defined as either an improvement from off-chart bcva to on-chart, or an on-chart improvement bcva of at least -0.2 logmar, or +10 etdrs letters. improvement in quality of life metrics relative to baseline values taken before treatment, as assessed by the well-established national eye institute visual function questionnaire-25 (nei vfq-25), was clinically relevant3 in the subscales related to mental health, dependency, and role difficulties. see: "efficacy and safety of intravitreal gene therapy for leber hereditary optic neuropathy treated within 6 months of disease onset." nancy j.newman et al. ophthalmology available online 12 january 2021. https: doi.org 10.1016 j.ophtha.2020.12.012>