GenSight Biologics Announces Initial Results from New Meta-Analyses on Visual Outcomes with LUMEVOQ Gene Therapy at NANOS 2024 - Seite 2

Based on the pre-defined statistical analysis plan, the three meta-analyses were performed on three groups of ND4-LHON patients according to therapy: LUMEVOQ gene therapy one-time intravitreal injection, idebenone therapy and no treatment (natural history). To be included in the meta-analyses, studies had to satisfy a number of criteria that ensured comparability: for example, at least 3 data points per eye, information on the type of mutation with inclusion of ND4 patients only, and availability of visual outcomes data such as CRR. Clinically Relevant Recovery (CRR) is an accepted criterion of clinically meaningful visual improvement in LHON patients⁶ and could be extracted from all three patient samples. The final set of qualifying studies consisted of five natural history studies (173 patients), six idebenone studies (201 patients) and three LUMEVOQ studies (174 patients).

“The meta-analyses formalize the patient testimonies we have received that attest to the difference LUMEVOQ can make in patients’ lives: this treatment transforms the natural history of the disease,” said Laurence Rodriguez, Chief Executive Officer of GenSight. “The team is working hard to make sure that patients and clinicians facing the visual emergency that is LHON have access to an efficacious treatment.”

The Company is currently engaging with authorities in the US, EU, and UK to align on the regulatory path for LUMEVOQ. The Company aims to restart the Early Access Program (AAC) in France in Q3 2024. Follow-up of patients in the Phase III REFLECT study of LUMEVOQ is ongoing, with topline results at Year 4 of follow-up expected this month.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.