Gensight Biologics Sa · EURONEXT:SIGHT
Gensight Biologics Sa — Investor Presentation (October 2023)
26 slides from Gensight Biologics Sa's investor presentation, October 2023
Slide 1: Corporate Presentation
Corporate Presentation October 2023 A LEADING Gene Therapy BIOTECHNOLOGY COMPANY GENSIGHT-BIOLOGICS.COM 2023-NPM-O-001 / October 2023 – Non-Confidential
Slide 2: Disclaimer
Disclaimer This document contains forward-looking statements and depend upon factors that are beyond the Company’s estimates made by the GenSight Biologics S.A. (the control. Therefore, actual results, the financial condition, “Company”), including with respect to the anticipated performance or achi
Slide 3: Investment Case – Last Mile to LUMEVOQ Approval
Investment Case – Last Mile to LUMEVOQ® Approval Public company founded in 2012 dedicated to developing and commercializing gene therapies for Late-stage Biotech 1 company neurodegenerative retinal diseases and diseases of the central nervous system. Euronext Paris Listed SIGHT Seasoned Management t
Slide 4: Seasoned Executive Team
Seasoned Executive Team Bernard Gilly Thomas Gidoin Magali Taiel Chief Executive Officer Chief Financial Officer Chief Medical Officer PIXIUM VISION (Since 2011) DBV TECHNOLOGIES (2012-2015) ProQR THERAPEUTICS (2016-2018) FOVEA PHARMA (2005-2009) IPSEN (2008-2011) ELI LILLY (2004-2016) SOFINNOVA PAR
Slide 5: Pipeline: solid and advanced product portfolio in ophthalmic Gene Therapy
Pipeline: solid and advanced product portfolio in ophthalmic Gene Therapy Expected Preclinical Phase I / II Phase III Registration Approval Market Opportunity MTS Platform LHON ND4 (EU) * ~ 450 new patients per year EMA Orphan Drug Designation LHON ND4 (US) ~ 250 new patients per year FDA Orphan Dru
Slide 6: LUMEVOQ in LHON-ND4
LUMEVOQ® in LHON-ND4 • 3 Phase III completed; additional Phase III to be initiated in Q2 2024 • New European regulatory submission planned in Leber Hereditary Optic Neuropathy
Slide 7: 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 GS010: ITR ITR cDNA_ND4 MTS1 MTS2 Gene encapsulated in AAV Step 1 Step 2 Ste
Slide 8: ND4 LHON: blinding bilateral mitochondrial disease
ND4 LHON: blinding bilateral mitochondrial disease Devastating impact Acute, rapidly progressing and irreversible • Major cause of blindness in young adults Evolution of vision from onset ONSET 3M 6M 12M “The evolution of natural 1st EYE 2nd EYE history eyes shows an absence of recovery” Incidence 0
Slide 9: Clinically meaningful and persistent visual function improvement across 3 Phase III studies
Clinically meaningful and persistent visual function improvement across 3 Phase III studies RESTORE RESTORE 98 patients 37 patients 39 patients with vision loss ≤ 1 year with vision loss 6 ≤ 12 months with vision loss ≤ 6 months 3+ lines of visual acuity improvement vs Nadir is Change from NADIR* in
Slide 10: Meaningful and sustained visual function improvement for the majority of patients
Meaningful and sustained visual function improvement for the majority of patients *Definition of “responder” At least -0.3 LogMAR (+15 letters) improvement from nadir Patient Responder Rates* Across LUMEVOQ® Phase III trials (%) Clinically relevant recovery (CRR)** from nadir 100 Responder rates 3 y
Slide 11: Indirect comparison to assess LUMEVOQ treatment effect
Indirect comparison to assess LUMEVOQ® treatment effect RESTORE Contralateral effect eliminated the control group formed by the set of eyes treated with sham/placebo injection →Indirect comparison of Lumevoq data versus an external control group to assess efficacy of the gene therapy Treated Group U
Slide 12: Visual improvement after LUMEVOQ treatment contrasts sharply with natural history
Visual improvement after LUMEVOQ® treatment contrasts sharply with natural history Clinically significant improvement of visual acuity persistent + over time for Lumevoq® efficacy pool versus Natural History • Evidence of efficacy drawn from three Phase III trials – unprecedented for a rare disease
Slide 13: Prospective data from early access programs confirm results of LUMEVOQ trials
Prospective data from early access programs (EAP) confirm results of LUMEVOQ® trials Evolution of mean visual acuity over time (n=63) Treated patients under EAP (n=63) Results at last observation 63 treated patients Last Observation = time post- Mean (SD): 13.37 (5.94) treatment (in months) Median:
Slide 14: Favorable safety and tolerability profile
Favorable safety and tolerability profile • No study discontinuations related to treatment or study procedure1 • Excellent systemic tolerance, related to the limited biodissemination2 • Mostly mild intraocular inflammation3, which was responsive to conventional treatment4, mostly corticosteroid eye
Slide 15: Regulatory path forward for LUMEVOQ
Regulatory path forward for LUMEVOQ® • Defining the next steps for a new MAA • Scientific advice received and further interactions planned • Interactions (scientific advice) ongoing to explore a UK-MA regulatory path • Phase 3 trial design discussions ongoing • Supply for Early Access program “Autor
Slide 16: Second product candidate targeting photoreceptor degenerative diseases: GS030
GS030 Second product candidate targeting photoreceptor degenerative diseases: - Retinitis Pigmentosa (RP) - Age-Related Macular Degeneration (AMD)
Slide 17: Treating the main degenerative diseases of photoreceptors that lead to blindness
Treating the 2 main degenerative diseases of photoreceptors that lead to blindness Geographic Atrophy (GA) in AMD Retinitis Pigmentosa (RP) (Age-Related Macular Degeneration) • Blinding genetic disease • Early (dry-form) AMD evolves with age into • Mutations in over 100 different genes late AMD, one
Slide 18: GS030 Optogenetic Therapy: Combining Gene Therapy and Medical Device
GS030 Optogenetic Therapy : Combining Gene Therapy and Medical Device Drug Product GS030-DP ChrimsonR gene Medical Device GS030-MD AAV2.7m8 vector + Light Projector ➢ Restore light sensitivity to diseased retina, independently of causative mutation 18 October 2023 - Non-Confidential
Slide 19: GS030 leads to functional vision restoration in monkey and rats
GS030 leads to functional vision restoration in monkey and rats Localization of light-sensitive protein in NHP retina Dose-ranging response to firing relationship in NHP Expression of ChrR-tdT in midget cells of Active dose range : 5x1010 and 5x1011 VG/eye monkey perifovea MEA assessment 6 months af
Slide 20: PIONEER Phase I/II clinical trial: In late-stage Retinitis Pigmentosa
PIONEER Phase I/II clinical trial: In late-stage Retinitis Pigmentosa Study design Cohort 1 (N = 3) Cohort 2 (N = 3) Cohort 3 (N = 3) Extension Cohort (N = 3-9) 5E10 vg/eye 1.5E11 vg/eye 5E11 vg/eye Highest dose of 5E11 vg/eye Recruitment ongoing 1 patient injected DSMB confirmed good safety profile
Slide 21: PIONEER Phase I/II clinical trial: A favorable safety and tolerability profile
PIONEER Phase I/II clinical trial: A favorable safety and tolerability profile • No study discontinuations related to treatment or study procedure • Excellent systemic tolerance, related to the limited biodissemination • Mostly mild intraocular inflammation, which was responsive to conventional trea
Slide 22: PIONEER Phase I/II clinical trial: Encouraging signs of efficacy in the highest dose cohort
PIONEER Phase I/II clinical trial: Encouraging signs of efficacy in the highest dose cohort Efficacy signal at one year • Released in Feb 2023, when the patients from the highest dose cohort have reached 1-year post gene therapy treatment • Encouraging signals of efficacy at one year post treatment:
Slide 23: Corporate & Finance
Corporate & Finance
Slide 24: Rich upcoming news flow with numerous inflection points
Rich upcoming news flow with numerous inflection points Q4 FDA Type-C H2 2026 September Meeting Topline Results 1st GMP Batch RECOVER Manufacturing November UK MHRA January Q4 Scientific REFLECT 4Yrs REFLECT 5Yrs April Withdrawal Advice 2027 of EMA Meeting Q1 Q2 Q1 Expected EMA Application Expected
Slide 25: GenSight Biologics in numbers
GenSight Biologics in numbers Key financial information Company Overview Analyst Coverage Market Cap* : € 33m Cash Position : € 1.0m + €10m (financing announced on Aug 3) (June 30, 2023) Daniil Gataulin (US) Outstanding Shares: 46.3m Alex Cogut (FR) Latest Equity Raised : € 30m (March 2021) Damien C
Slide 26: GenSight Biologics: Take Away
GenSight Biologics: Take Away • Late-stage biotech with revolutionary solutions fulfilling the promise of gene therapy and optogenetics • Seasoned management team and solid investor base to back up the focused portfolio • LUMEVOQ® for Leber Hereditary Optic Neuropathy ○ Significant and sustained vis
GenSight Biologics — Investor Presentation (October 2023) on 6ix