GenSight Biologics Withdraws its EMA Application for LUMEVOQ®

Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible) (Paris:SIGHT), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announces that the Committee for Advanced Therapies (CAT)¹ of the Committee Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) assessed the data presented during the oral explanation² on LUMEVOQ^® European regulatory dossier.

As provided by the review procedure and following responses to the D180 questions, an oral explanation was held on April 19. GenSight invited the world renowned LHON³ experts, Patrick Yu-Wai-Man, MD, PhD (Cambridge University, UK) and José-Alain Sahel, MD (University of Pittsburgh School of Medicine, USA) to share their clinical practice and perspective on LUMEVOQ^® data.

Following interactions with the CAT indicating that the data provided thus far would not be sufficient to support a positive opinion of the marketing authorization of LUMEVOQ^® by EMA, GenSight decided to withdraw its application ahead of a final opinion by the CAT. This decision enables the Company to discuss the best possible path forward for LUMEVOQ^® with the EMA in the coming weeks, aiming at submitting a new application addressing remaining objections as soon as possible, in Europe and other countries. The company is exploring options including generating new clinical data, which may induce material delays and additional costs.

“GenSight teams have gathered a huge dataset of 252 ND4-LHON patients treated with LUMEVOQ showing that 70% of LUMEVOQ-treated patients present a visual recovery, in contrast to the poor and limited recovery observed in the natural history of the disease,” said José-Alain Sahel, Co-founder of GenSight Biologics and of the Institut de la Vision, Paris, France, “In light of the study results, confirmed by real-life data, LUMEVOQ is the current best therapeutic option for ND4-LHON patients given the 3-fold difference in vision function in treated patients. It is disappointing that the contralateral effect of the therapy limited the perceived strength of these data, published in top-tier peer-reviewed journals by leaders in the field.”

“We disagree with the current CAT assessment and remain highly confident in the clinical benefit of LUMEVOQ for LHON patients, which is supported by extensive evidence from multiple clinical trials and real-world data,” commented Bernard Gilly, Chief Executive Officer and Co-Founder of GenSight Biologics. “The decision to withdraw our application allows us to continue to work with EMA to agree as soon as possible on a regulatory path forward. I want to thank the patient communities for their support and reaffirm our determination to bring this innovative therapy to ND4-LHON patients in need of an efficacious treatment. I also want to thank the scientific community and our teams for their long-lasting commitment.”

GenSight confirms that the manufacturing validation campaign is on track with its partner in the United-States, as planned, with a product released for human use by the end of the year.

GenSight's Marketing Authorization Application (MAA) submission of LUMEVOQ^® to the EMA was based on the benefit-risk balance established by results from 252 patients treated through clinical trials or compassionate use. This unprecedented data set for ND4-LHON establishes that LUMEVOQ^® efficacy compares positively to both natural history and idebenone.

GenSight management team will host a live webcast at 4:30 pm CEST (10:30 am EST) today to further comment on this decision. The webcast will be held in English, and a simultaneous French translation will also be available.

Live webcast in English, with a simultaneous French translation also available: https://bit.ly/3LcxrMD
The webcast will be available in replay using the same link above.

The Company will report its cash position as of March 31, 2023, in a separate press release later today.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing 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 an investigational compound and has not been registered in any country at this stage; a marketing authorization application is currently under review by the EMA for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. 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.

About LUMEVOQ^® (GS010; lenadogene nolparvovec)

LUMEVOQ® (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. “LUMEVOQ” was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. LUMEVOQ® (GS010; lenadogene nolparvovec), is an investigational compound and has not been registered in any country at this stage.

¹ The Committee for Advanced Therapies (CAT) is the European Medicines Agency's (EMA) committee responsible for assessing the quality, safety and efficacy of advanced therapy medicinal products (ATMPs) and following scientific developments in the field.
² After the second clock-stop, an oral explanation in which the applicant directly addresses the committee can be requested either by the applicant or by the CHMP - https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/evaluation-medicines-step-step#clock-stop-2-section
³ Leber hereditary optic neuropathy (LHON) and ND4 is the most frequent mutation with worst prognosis for LHON patients

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