Gensight Biologics Sa · EURONEXT:SIGHT
Gensight Biologics Sa — Investor Presentation (May 2025)
26 slides from Gensight Biologics Sa's investor presentation, May 2025
Slide 1: A Leading Gene Therapy Biotechnology Company: Corporate presentation
A LEADING GENE THERAPY BIOTECHNOLOGY COMPANY Corporate presentation MAY 2025 GENSIGHT-BIOLOGICS.COM
Slide 2: Disclaimer
Disclaimer This document contains forward-looking based on financial and non-financial information, estimates. Forward-looking statements, statements and estimates made by the GenSight including projections as to the future regulatory forecasts and estimates only speak as of the Biologics S.A. (the
Slide 3: The investment case for GenSight Biologics
The investment case for GenSight Biologics: Gene therapy company with promising candidate for pivotal trial Late-stage Seasoned Last mile of clinical Proof-of-concept in biotech company management team development for Phase III humans for cutting-edge focused on realizing the and supportive investor
Slide 4: A new chapter, led by a seasoned international management team
A new chapter, led by a seasoned international management team … Laurence Rodriguez Magali Taiel Scott Jeffers Magali Gibou Chief Executive Officer Chief Medical Officer Chief Technical Officer Chief Regulatory & Quality Affairs Officer SANOFI (2011-2021) ProQR THERAPEUTICS (2016-2018) REDPIN THERAP
Slide 5: Overseen by a board of international industry experts as directors
… and overseen by a board of international industry experts as directors Michael Wyzga Prof. José-Alain Sahel Maritza McIntyre, Ph.D. Simone Seiter, M.D., Ph.D. Chairman since March 2016 Director Independent Director Independent Director Corporate strategy Research and development CMC and Regulatory
Slide 6: LUMEVOQ in ND4-LHON
LUMEVOQ® in ND4-LHON • Compelling data on treatment benefit and safety from treating 252 patients M AY | 2025 6
Slide 7: LHON: rare mitochondrial disease with no effective options
LHON: rare mitochondrial disease with no effective options against acute and irreversible loss of vision Natural history: Devastating impact acute, rapidly progressing and irreversible blindness Evolution of vision from onset of disease • Major cause of blindness in young adults Onset 3M 6M 12M • Vi
Slide 8: LUMEVOQ: enhanced AAV-based gene therapy to restore mitochondrial function
LUMEVOQ®: enhanced AAV-based gene therapy to restore mitochondrial function and restore visual function Exclusive MTS* technology to deliver gene therapy solution to damaged mitochondria Conventional gene therapy mechanism … … Enhanced with MTS* STEP 1 STEP 2 STEP 3 STEP 4 Wild-type mRNA Wild-type m
Slide 9: Clinically meaningful and durable visual recovery, instead of inevitable blindness
Clinically meaningful and durable visual recovery, instead of inevitable blindness Results consistent across 3 Phase III clinical trials (n=174) and early access/compassionate use programs (n=63) Mean improvement vs. vision nadir*, PHASE III** ETDRS chart 5 years after one-time injection BILATERAL I
Slide 10: For majority of patients, meaningful and durable visual recovery after one-time treatment
For majority of patients, meaningful and durable visual recovery after one-time treatment with LUMEVOQ® Results consistent across 3 Phase III clinical trials (n=174) and early access/compassionate use programs (n=63) Visual improvement vs. vision nadir*, PHASE III** ETDRS chart 5 years after one-tim
Slide 11: Evidence from real life settings consistent with clinical trial data
Evidence from real life settings consistent with clinical trial data Evolution of mean visual acuity over time (n = 63) Vision function improvement at last available observation 1.4 (n=63 patients; 126 eyes) Mean +/- std. err. of BCVA value (LogMAR) 1.5 Average change in 24 ETDRS letters visual acui
Slide 12: Highly favorable safety record
Highly favorable safety record Comprehensive evidence from 252 patients treated in Phase I/II , four Phase III trials and early access ● No study discontinuations related to treatment or study procedure1 ● Excellent systemic tolerance, related to the limited biodissemination2 ● Mostly mild intraocul
Slide 13: Next for LUMEVOQ: Bold Steps for Big Wins
Next for LUMEVOQ®: Bold Steps for Big Wins • RECOVER trial • Manufacturing • Regulatory path M AY | 2025 13
Slide 14: Why the RECOVER trial is required
Why the RECOVER trial is required: gain full acceptance of clinical results by regulatory authorities in the US and EU Unforeseen contralateral effect inadequate control arm requirement to demonstrate efficacy in new trial • Consistent contralateral effect in Phase III trials: untreated eyes (sh
Slide 15: The contralateral effect has underlying biological mechanisms
The contralateral effect has underlying biological mechanisms The design of the RECOVER trial needs to remove the confounding impact of the contralateral effect Type of st udy Finding Reference Animal Transcription of LUMEVOQ ND4 DNA in the contralateral McGrady Molecular Therapy 2023 (mice) untreat
Slide 16: RECOVER Phase III trial: designed to succeed
RECOVER Phase III trial: designed to succeed Leverage insights and experience; incorporate regulatory feedback; engage top LHON clinical experts Insights from previous trials: Feedback from the FDA and EMA: • Optimal treatment window = 6 • Bilateral placebo injection in control months to 1.5 years a
Slide 17: Manufacturing: incorporating 2024 lessons into 2025 priorities
Manufacturing: incorporating 2024 lessons into 2025 priorities >>> Optimization 2024 >>> >>> RECOVER Trial and MAA >>> Process Tech transfer and MAA and Address past French Early improvements Clinical Supply execution Access (AAC) process Commercial gaps (downstream supply (RECOVER, AAC) ops) optimi
Slide 18: Last mile of clinical development: advancing towards marketing authorization
Last mile of clinical development: advancing towards marketing authorization LUMEVOQ® regulatory path towards submission in key markets • Scientific advice received on new Phase • Scientific advice received on new Phase III study RECOVER – positive feedback III study RECOVER – positive feedback on o
Slide 19: GS030
GS030 • Optogenetic treatment approach* for photoreceptor degenerative diseases with novel, mutation-agnostic mechanism of action • Promising early results from PIONEER Phase I/II study show blind patients regaining ability to locate objects M AY | 2025 *GS030 is a combination treatment consisting o
Slide 20: Optogenetics for Retinitis Pigmentosa
Optogenetics for Retinitis Pigmentosa Retinitis Pigmentosa (RP) GS030 Therapy: Bypass Degenerated Cells through Optogenetics Retinal Ganglion Cells (RGCs) Degenerating Photoreceptors Gene Therapy GS030-DP Intravitreal injection 1 RGC transfection ChrimsonR Expression of light- • Leading cause of i
Slide 21: GS030 to date: favorable safety profile and promising early signals
GS030 to date: favorable safety profile and promising early signals of reactivating visual function Findings from PIONEER Phase I/II Clinical Trial Safety Efficacy signal (at one year) • No study discontinuations related to treatment or study • Vision improvement observed in some patients procedure
Slide 22: Corporate & Finance
Corporate & Finance M AY | 2025 22
Slide 23: GenSight Biologics Listed in Euronext Paris
GenSight Biologics Listed in EuroNext Paris Company Overview Shareholder Structure, as of Apr.16, 2025 Headquarter: Paris, France Sofinnova (22.8%) Listing: Euronext Paris 22,8% Invus (14.6%) Tickers: SIGHT 37,8% Heights (9.1%) UPMC (7.7%) ISIN: FR0013183985 14,6% Goldman Sachs Group (4.8%) BPIfranc
Slide 24: Backup
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Slide 25: Clinically meaningful and durable visual recovery, instead of inevitable blindness
Clinically meaningful and durable visual recovery, instead of inevitable blindness Mean Improvement vs. vision nadir*, 5 years after one-time injection RESTORE RESTORE BILATERAL INJECTION Unilateral Injection Unilateral Injection Unilateral Injection Mean improvement vs. Mean improvement vs. Mean im
Slide 26: Majority of patients experience meaningful, durable visual recovery after one-time treatment
Majority of patients experience meaningful, durable visual recovery after one- time treatment with LUMEVOQ REFLECT Trial REVERSE+RESTORE Trial RESCUE+RESTORE Trial @ 5 Years @ 5 Years @ 5 Years (injected ≤12 months after onset) (injected 6-12 months after onset) (up to 6 months after onset) Improvem
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