GenSight Biologics Publishes Meta-Analysis on Efficacy of Treatments for Leber Hereditary Optic Neuropathy

GenSight Biologics announced the publication of a meta-analysis evaluating visual outcomes in patients with Leber Hereditary Optic Neuropathy (LHON) due to the MT-ND4 mitochondrial gene mutation. This mutation, known as ND4-LHON, is the most common cause of the disease and is associated with the most severe visual prognosis.

The study, published in the journal Survey of Ophthalmology, is the first to compare the effectiveness of various LHON treatments, both approved and experimental, specifically for the ND4-LHON patient population, according to GenSight. This analysis assesses visual recovery outcomes for patients receiving GenSight’s Lumevoq gene therapy, those treated with idebenone, and untreated patients following the natural course of the disease.

The findings highlight a clear “gradient of efficacy” across two key measures of visual recovery. Lumevoq gene therapy demonstrates significantly better visual outcomes compared to idebenone and both treatments outperform the natural progression of ND4-LHON, which typically leads to a severe and chronic reduction in visual acuity, affecting quality of life in both eyes.

One of the primary metrics analyzed in the study is the rate of Clinically Relevant Recovery (CRR), a measure of visual recovery consistency across the various studies reviewed. According to the analysis, the CRR rate following Lumevoq therapy is three times higher than that seen in untreated ND4-LHON patients and significantly surpasses that achieved with idebenone treatment.

“This study is very important for comparing these therapies’ abilities to achieve visual function outcomes in ND4-LHON patients," Nancy J. Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology at Emory University School of Medicine in Atlanta, and lead author of the study, said in a company news release. “For the first time, we clearly demonstrate a gradient of efficacy in visual outcomes, more marked for Clinically Relevant Recovery (CRR) than for final best-corrected visual acuity (BCVA), with lenadogene nolparvovec gene therapy superior to idebenone treatment, and both superior to the natural history of the disease.”

The publication also updates earlier findings presented at the 2024 North American Neuro-Ophthalmology Society annual meeting, integrating data from the recently completed LEROS trial for idebenone and new analyses of BCVA outcomes for each treatment group.