Paris, France - GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced initial results of new meta-analyses in Leber Hereditary Optic Neuropathy (LHON), which show those treated with LUMEVOQ (GS010; lenadogene nolparvovec) gene therapy experienced a rate of visual recovery greater than that of idebenone-treated patients and untreated (natural history) patients.

The meta-analyses are the first to focus solely on patients with the m.11778G>A ND4 mutation, which is the most common mutation and one with a poor visual prognosis[1].

Dr. Nancy J. Newman, LeoDelle Jolley Professor of Ophthalmology and Neurology at the Emory University School of Medicine in Atlanta, United-States, presented the findings at NANOS 2024. She noted, 'This study is very important in order to compare these therapies' abilities to achieve equivalent end-point metrics. It is clear that the degree of efficacy of the gene therapy was greater than the other currently available options of no treatment and idebenone use as previously administered in studies.'

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 patients[6] 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.

Contact:

Ivan Tortet

Email: itortet@gensight-biologics.com

Guillaume van Renterghem

Tel: +41 (0)76 735 01 31

Email: gvanrenterghem@lifesciadvisors.com

(C) 2024 Electronic News Publishing, source ENP Newswire