GenSight’s gene therapy treatment for patients living with Leber hereditary optic neuropathy (LHON) has demonstrated sustained efficacy and eyesight improvement over five years.
France-based GenSight Biologics has unveiled final data from its Phase III Reflect trial (NCT03293524), a randomised, placebo-controlled study to evaluate the safety and efficacy of Lumevoq, also known as lenadogene nolparvovec. The results showed that a single injection of the gene therapy sustained its efficacy throughout the trial.
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LHON is a relatively rare genetic disorder of the eye in which individuals can suffer sudden and rapid vision loss over a certain period. The onset of LHON typically occurs in late childhood or early adulthood. In the case of the Reflect study, researchers selected patients who had contracted LHON due to a mutation in the NADH dehydrogenase 4 (ND4) protein.
Patients were split into two arms, with the active arm receiving an injection of Lumevoq in both eyes, while the placebo group received treatment in only one. Five years after injection, 75% who were bilaterally treated saw a rate of clinically relevant recovery, compared with 60% for those who received only one injection.
Additionally, 79% of bilaterally treated patients were able to read letters on a screen while this figure was 72% in the placebo group.
The REFLECT trial’s principal investigator Patrick Yu-Wai-Man said: “The latest REFLECT data confirms that the improvement seen with lenadogene nolparvovec is sustained five years after treatment has been given, including the additional benefit observed in participants receiving a bilateral intravitreal injection of the gene therapy.
“Importantly, REFLECT participants receiving a bilateral injection had a comparable safety profile to those treated unilaterally.”
The trial was carried out at sites across the UK, Europe, the US and Taiwan, seeing up to 98 subjects between 2018 and 2019. The company is entering a relatively clear market. According to GlobalData’s Pharmaceutical Intelligence Center, there is only one other treatment in Phase III development.
Elsewhere in the field of treatments for ocular degeneration, Chinese biotechnology company Innovent Biologics has reported that its Phase II trial of efdamrofusp alfa high-dose to treat neovascular age-related macular degeneration (nAMD) met its primary endpoint. Meanwhile, French digital health company Tilak Healthcare has announced positive results from a clinical trial of a mobile app that allows patients to monitor their eyesight.
