Gensight Biologics Sa · EURONEXT:SIGHT
Gensight Biologics Sa — Investor Presentation (January 2022)
29 slides from Gensight Biologics Sa's investor presentation, January 2022
Slide 1: Corporate Presentation
Corporate Presentation January 2022 A LEADING Gene Therapy BIOTECHNOLOGY COMPANY GENSIGHT-BIOLOGICS.COM
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 – Transitioning from R&D to Commercial Organization
Investment Case – Transitioning from R&D to Commercial Organization 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 Listed SIGHT / €265m
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) End 2022 ~ 400 new patients per year EMA Orphan Drug Designation LHON ND4 (US) ~ 600 new patients per year FDA Orp
Slide 6: LUMEVOQ® in LHON-ND4
LUMEVOQ® in LHON-ND4 • 3 Phase III completed • European regulatory submission ongoing in Leber Hereditary Optic Neuropathy • Commercial preparation ongoing for early 2023 European launch
Slide 7: LUMEVOQ® introduces Gene Therapy solution
LUMEVOQ® introduces Gene Therapy solution 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 Step 3 Step 4 Retinal cell tra
Slide 8: Leber Hereditary Optic Neuropathy (LHON-ND4) high unmet medical need
Leber Hereditary Optic Neuropathy (LHON-ND4) high unmet medical need What is LHON-ND4 Progressive disease Current treatment paradigm • Rare inherited mitochondrial disease leading to • Rare recovery from vision nadir(1) reached during • No cure for LHON-ND4 degeneration of retinal ganglion cells (RG
Slide 9: Unparalleled clinical benefit demonstrated with LUMEVOQ® in LHON in 3 Phase III studies
Unparalleled clinical benefit demonstrated with LUMEVOQ® in LHON in 3 Phase III studies 34 patients 37 patients 98 patients with vision loss ≤ 6 months with vision loss 6 ≤ 12 months with vision loss ≤ 1 year 3+ lines of visual acuity improvement vs Nadir is Change from NADIR in ETDRS letter Change
Slide 10: The Bilateral Effect Demonstrated – LUMEVOQ also detected in contralateral eyes
The Bilateral Effect Demonstrated – LUMEVOQ also detected in contralateral eyes LUMEVOQ® Front Study1 conducted on 6 non-human primates : All received a single injection of LUMEVOQ® in one eye at a Left Anterior segment dose equivalent to that used in humans, while 2 control animals received a pla
Slide 11: LUMEVOQ® modifies disease outcome compared to natural history
LUMEVOQ® modifies disease outcome compared to natural history Sustained improvement after LUMEVOQ® injection vs. absence of recovery among untreated patients Figure 1. Evolution of Visual Acuity in LUMEVOQ®-treated Patients (N=76) versus Untreated Patients (N=208) * Best-Corrected Visual Acuity (Log
Slide 12: Better efficacy for bilaterally treated subjects demonstrated in REFLECT
Better efficacy for bilaterally treated subjects demonstrated in REFLECT Note: The LogMAR value of interest at 1.5 years was the first logMAR recorded after 518 days after IMP and for the 2 year analysis the LogMAR value of interest is the nearest LogMAR of 730 days recorded between 700 and 935 days
Slide 13: Driving patient and physicians' awareness through Compassionate Use for LUMEVOQ®
Driving patient and physicians' awareness through Compassionate Use for LUMEVOQ® • 18 individual patients Expanded Access • “ATU de Cohorte” or ATUc - Cohort Temporary Authorization for Use - for LUMEVOQ® granted by ANSM INDs so far approved by the FDA for to GenSight on July 5, 2021 LUMEVOQ® ○ “ATU
Slide 14: Real World Experience – US Compassionate Use Program
Real World Experience – US Compassionate Use Program AVG Worse Eye BCVA Mean ETDRS AVG Better Eye BCVA Mean ETDRS Change (LogMAR) Change (LogMAR) 11 lines of improvement 7.5 lines of improvement Baseline 1 Week 2 Weeks 1 Month 3 Months 6 Months 1 Year Baseline 1 Week 2 Weeks 1 Month 3 Months 6 Month
Slide 15: European Commercial Strategy – Leveraging LUMEVOQ® Clinical Centers
European Commercial Strategy – Leveraging LUMEVOQ® Clinical Centers to Build Network of LHON Centers of Excellence • LHON experts mapped in both major and smaller markets • Progressively build the LHON clinical network working with LHON experts o Recognize varying levels of LHON expertise and patien
Slide 16: European Reimbursement Strategy – Short Term Revenues Generation Expected in H1 2023
European Reimbursement Strategy – Short Term Revenues Generation Expected in H1 2023 Commercial Launch (L) L + 12 Months L + 24 Months H1 2023 Free pricing upon approval; benefit Negotiated price after 12 months; price assessment; price negotiations influence on other markets 3 Largest EU countries
Slide 17: Early Engagement Initiated with Key European payers
Early Engagement Initiated with Key European payers Post approval Recognition of Contact Meeting Discussion potential Pricing initiated scheduled engaged significant benefit evaluation Highly Specialized Promising Technology Innovative Medicine Recognition Cohort ATU 17 January 2022 - non confidenti
Slide 18: A Targeted Approach to Accelerate Diagnosis and Get More Patients Treated
A Targeted Approach to Accelerate Diagnosis and Get More Patients Treated Within 12 Months Aims to educate A&Es, Ophthalmologists and Neurologists to LHON ND4 disease and other forms of neurodegenerative diseases to help more ~ 1,000 patients be treated within the important first months of diagnosis
Slide 19: 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 20: 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 21: GS030: using Gene Therapy to rejuvenate production of light-sensitive protein
GS030: using Gene Therapy to rejuvenate production of light-sensitive protein and restore vision AAV2.7m8 + ChrimsonR Na+ + The product of Step 1 Step 2 Step 3 research collaboration with Gene Therapy Stimulation with Retinal output sent transfer of the gene optoelectronic to brain for image that en
Slide 22: 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 23: PIONEER Phase I/II clinical trial: A First-in-Man study
PIONEER Phase I/II clinical trial: A First-in-Man study Study design Cohort 1 (N = 3) Cohort 2 (N = 3) Cohort 3 (N = 3) Extension Cohort 5E10 vg/eye 1.5E11 vg/eye 5E11 vg/eye Highest dose of 5E11 vg/eye DSMB confirmed good safety profile 4 weeks post-injection of 3rd (last) patient of each cohort •
Slide 24: PIONEER: encouraging preliminary findings from two patients
PIONEER: encouraging preliminary findings from two patients Outcome one year after gene therapy Video of treated patient Both treated patients experienced significant vision improvement, from being barely able to perceive light before treatment to being able to locate and count objects, one year aft
Slide 25: GS030 timeline
GS030 timeline PIONEER PIONEER PIONEER PIONEER 1yr cohort Phase 3 1st patient 2nd patient extension initiation in 1yr results report report results RP H1 2021 H2 2021 H1 2022 H2 2022 H1 2023 H2 2023 2024 Phase 3 Phase 1/2 FDA Fast Track goggles initiation in Designation prototype Dry AMD/GA Manufact
Slide 26: Building high strategic value
Building high strategic value
Slide 27: Rich upcoming news flow with numerous inflection points
Rich upcoming news flow with numerous inflection points Q3 April Response Response to to D180 Q4 June D120 EMA EMA CHMP Q1 REFLECT December Questions Questions Decision EU Approval & Launch August 1.5Yrs REFLECT January RESTORE 3Yrs readout FU RESCUE/REVERSE 2Yrs FDA Type-C Feedback 2021 2022 2023 2
Slide 28: GenSight Biologics in numbers
GenSight Biologics in numbers Key financial information Company Overview Share price evolution and trading volume Market Cap* : € 208m Analyst Coverage Share price € Trading volume € m Cash Position : € 44.3m 10 10 (December 31, 2021) • Chardan: Geulah Livshits (US) 5 5 Outstanding Shares: 46.3m • B
Slide 29: Impressive Development Track Record from Concept to Clinic
Impressive Development Track Record from Concept to Clinic 2021-2022 REGULATORY REVIEW IN EU AND IN THE US EU regulatory review ongoing - Potential approval in Q4 2022 FDA discussion ongoing 2023 LUMEVOQ® EXPECTED EUROPEAN LAUNCH 2016 European commercial team started EURONEXT IPO executing on fast l
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