NEW YORK – GenSight Biologics on Wednesday said it plans to launch a new Phase III trial of its lead candidate, a gene therapy for a rare inherited mitochondrial disorder associated with vision loss, after receiving feedback from the European Medicines Agency.
The Paris-based gene therapy developer in April withdrew its EMA application for Lumevoq (lenadogene nolparvovec), which aims to treat Leber hereditary optic neuropathy (LHON) by delivering a functional copy of the ND4 gene, after an assessment by the agency's Committee for Advanced Therapies indicated data provided by the firm wasn't sufficient.
The Phase III results that GenSight submitted at the time failed to meet the study's primary endpoint when comparing the visual acuity change from baseline between participants who received Lumevoq in both eyes versus those who only received Lumevoq in one eye. However, investigators observed an improvement in visual acuity from baseline for Lumevoq-treated patients.
GenSight is continuing to finalize the design for a new proposed Phase III study, dubbed RECOVER, which it expects to kick off in the second quarter of next year. The company will also discuss its intended study design for the RECOVER trial with authorities in the UK and US, where it will also seek regulatory approval.
GenSight plans for the proposed Phase III trial to be a randomized, placebo-controlled trial with a treatment arm in which patients will receive intravitreal injections of Lumevoq in both eyes and a sham control arm. The study will also contain an open-label provision, in which patients in the control arm would be eligible to receive the treatment if the primary endpoint is met.
"We are grateful that key aspects of RECOVER have been endorsed so that we can work on refining and optimizing the design," GenSight CEO and Cofounder Bernard Gilly said in a statement.
GenSight expects to have the new Phase III trial completed in the second half of 2026.