GenSight Biologics on Track to Submit LUMEVOQ for European Approval in September 2020 Following Pre-Submission Meeting With EMA

Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announces the completion of the pre-submission meeting with the European Medicines Agency (EMA) for the Company’s lead product LUMEVOQ (GS010; lenadogene nolparvovec).

The EMA pre-submission meeting is a procedural milestone in the preparation of a marketing authorization application (MAA). Based on its successful conclusion, GenSight Biologics confirms its intention to submit the MAA for LUMEVOQ in September 2020.

LUMEVOQ is the gene therapy developed by the Company for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare blinding disease marked by sudden, irreversible vision loss. The pivotal trials for LUMEVOQ in Europe, RESCUE and REVERSE, were completed in 2019; patients from those trials have been invited to participate in a long-term follow-up study. In addition, GenSight Biologics has been conducting a natural history study (REALITY) and mechanistic studies in animals to supplement the data from RESCUE and REVERSE.

In its meeting with GenSight Biologics, the EMA confirmed the information needed for the Company’s September submission. The agency also provided updated guidance on various administrative topics, as well as advice on topics to be discussed in meetings between the Company and the Rapporteurs and Co-rapporteurs planned for June 2020.

“We are excited about our progress towards our regulatory filing of LUMEVOQ in Europe, as this brings us closer to making a novel, effective and safe treatment available to a large proportion of LHON patients,” said Bernard Gilly, Co-founder and Chief Executive Officer of GenSight Biologics. “We have been and continue to be extremely grateful for the support and guidance we have received from the European authorities.”

As a final step before actual submission, and as part of EMA’s procedures, GenSight Biologics expects to re-confirm by July its intent to submit in September.

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, LUMEVOQ (GS010; lenadogene nolparvovec), 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 the eye by intravitreal injection to offer patients a sustainable functional visual recovery.