Gensight Biologics Sa · EURONEXT:SIGHT
Gensight Biologics Sa — Investor Presentation (September 2024)
22 slides from Gensight Biologics Sa's investor presentation, September 2024
Slide 1: Corporate presentation
A LEADING GENE THERAPY BIOTECHNOLOGY COMPANY Corporate presentation SEPTEMBER 2024 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: 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 VP, Regulatory & Quality SANOFI (2011-2021) ProQR THERAPEUTICS (2016-2018) REDPIN THERAPEUTICS (2021-2022) S
Slide 4: International board of directors with extensive industry and geographic expertise
International board of directors with extensive industry and geographic expertise Michael Wyzga Prof. José-Alain Sahel Maritza McIntyre, Ph.D. Simone Seiter, M.D., Ph.D. Chairman since March 2016 Observer and Co-Founder Independent Director Independent Director Corporate strategy Research and develo
Slide 5: Investment case
Investment case Gene therapy company with a pivotal stage lead product candidate Seasoned LUMEVOQ® LUMEVOQ® Cutting-edge Late-stage management team/ Robust Defining registration optogenetics in Biotech company Solid investor base clinical data in LHON pathway Retinitis Pigmentosa Public company foun
Slide 6: Pipeline: solid and advanced product portfolio in ophthalmic gene therapy
Pipeline: solid and advanced product portfolio in ophthalmic gene therapy ________ Phase III ________ Ph III data Additional Preclinical Phase I/II available trial Registration Market opportunity MTS platform LHON ND4 (UK) Not needed ~45-50 new patients per year1 Orphan Drug Designation LHON ND4 (US
Slide 7: LUMEVOQ in ND4-LHON
01 LUMEVOQ® in ND4-LHON • Therapeutic effect and favorable safety in over 250 patients (Phase I/II, three completed Phase III and one Phase III ongoing) • UK regulatory submission planned for H2 2025 • Additional Phase III for US and EU targeted to start in H2 2025 SEPTEM B E R | 2024 7
Slide 8: LUMEVOQ introduces Gene Therapy solution for mitochondrial diseases
LUMEVOQ® introduces Gene Therapy solution for mitochondrial diseases Replacing affected mitochondrial mRNA via proprietary MTS* technology The product of research PCMV MTS1 cDNA_ND4 MTS2 collaboration with MTS in action for LUMEVOQ®: ITR ITR STEP 1 STEP 2 STEP 3 STEP 4 SEPTEM B E R | 2024 Wild-type
Slide 9: ND4-LHON: acute, blinding bilateral mitochondrial disease with high unmet medical need
ND4-LHON: acute, blinding bilateral mitochondrial disease with high unmet medical need Devastating impact Acute, rapidly progressing and irreversible: high unmet need • Major cause of blindness in young Evolution of vision from onset of disease adults Onset 3M 6M 12M • Causal mutation affects severi
Slide 10: Clinically meaningful and durable visual function improvement shown in four Phase III studies
Clinically meaningful and durable visual function improvement shown in four Phase III studies Meaningful visual acuity improvement 98 patients 37 patients 39 patients vision loss ≤ 1 year vision loss 6 ≤ 12 months vision loss ≤ 6 months Mean change from NADIR* in Mean change from nadir* in Mean chan
Slide 11: Visual function improvement for the majority of patients
Visual function improvement for the majority of patients Results not confined to a small number of super-responders At least +15 letters / 3 lines improvement from nadir Switch from off-chart to on-chart 100 REVERSE/ RESCUE/ 100 REVERSE/ RESCUE/ REFLECT RESTORE RESTORE REFLECT RESTORE RESTORE 4 year
Slide 12: LUMEVOQ real world experience: consistent with clinical trial results
LUMEVOQ® real world experience: consistent with clinical trial results Evolution of mean visual acuity over time (n = 63) Vision function improvement after injection (n=53) Mean change in +21 ETDRS letters visual acuity (vs. nadir) % of eyes with 63.2% On Chart on-chart vision Off Chart Improvement
Slide 13: LUMEVOQ: favorable safety and tolerability profile
LUMEVOQ®: favorable safety and tolerability profile 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 ●
Slide 14: Continuous optimization of manufacturing process for clinical and commercial supply
Continuous optimization of manufacturing process for clinical and commercial supply Corrective & Preventive Process improvements in Actions (CAPA) to close downstream unit Q3 2024 execution gaps in past operations • Successful blending and fill process to runs obtain DP for patients in Early Access
Slide 15: Manufacturing to enable resumption of paid early access in France
Manufacturing to enable resumption of paid early access in France Perform blending Implement step to optimize # CAPAs to of vials; close transfer fill and execution finish to new CDMO gaps ✓ ✓ ✓ ✓ Packaged and Manufacture Manufacture Quarantine Package and Release for released drug Drug Drug Product
Slide 16: Advancing towards providing access to LUMEVOQ
Advancing towards providing access to LUMEVOQ® Next steps on LUMEVOQ® regulatory path • Supply for Early Access • Scientific advice meeting • Defining the next steps for a • Scientific advice received on program outcome (December 2023) new MAA new Phase III study RECOVER confirmed that there will be
Slide 17: GS030
02 GS030 • Optogenetic treatment1 for photoreceptor degenerative diseases, such as: - Retinitis Pigmentosa (RP) - Age-Related Macular Degeneration (AMD) • PIONEER Phase I/II dose escalation study ongoing • Promising first results published in peer-reviewed journal2 show fully blind patients regainin
Slide 18: Phase I/II trial (safety and dose finding) for Retinitis Pigmentosa
Phase I/II trial (safety and dose finding) for Retinitis Pigmentosa Retinitis Pigmentosa (RP) Study design Extension Cohort 1 Cohort 2 Cohort 3 Cohort (N = 3) (N = 3) (N = 3) (N =1) 5E10 vg/eye 1.5E11 vg/eye 5E11 vg/eye Highest dose of 5E11 vg/eye DSMB confirmed good safety profile 4 weeks post-inje
Slide 19: Favorable safety profile and promising early efficacy signals from PIONEER
Favorable safety profile and promising early efficacy signals from PIONEER Safety Efficacy (at one year) • No study discontinuations related to treatment or • Vision improvement observed in some patients study procedure o Before treatment: barely able to perceive light • Excellent systemic tolerance
Slide 20: Corporate & Finance
03 Corporate & Finance SEPTEM B E R | 2024 20
Slide 21: GenSight Biologics financial highlights
GenSight Biologics financial highlights Company Overview Shareholder Structure Headquarter: Paris, France Sofinnova 22,3% Listing: Euronext Paris Other Tickers: SIGHT 47,7% Invus 16,7% ISIN: FR0013183985 Market Cap: €42,2m Director and employees UPMC 9,6% (August 30, 2024) 0,2% Armistice 3,5% Cash P
Slide 22: Recapitulation: GenSight Biologics investment case
Recapitulation: GenSight Biologics investment case Gene therapy company with a pivotal stage lead product candidate Seasoned LUMEVOQ® LUMEVOQ® Cutting-edge Late-stage management team/ Robust Defining registration optogenetics in Biotech company Solid investor base clinical data in LHON pathway Retin
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