Gensight Biologics Sa · EURONEXT:SIGHT
Gensight Biologics Sa — Investor Presentation (January 2021)
33 slides from Gensight Biologics Sa's investor presentation, January 2021
Slide 1: Corporate Presentation
Corporate Presentation January 2021 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: Corporate Overview – Transitioning from R&D to Commercial Organization
Corporate Overview – Transitioning from R&D to Commercial Organization Established in 2012 / IPO in 2016 GenSight at the forefront of Gene Therapy in Ophthalmology • Publicly traded Biotech company EuroNext Paris: SIGHT • Seasoned management team with strong BioPharma and Financial markets experienc
Slide 4: Seasoned Executive Team
Seasoned Executive Team Bernard Gilly Thomas Gidoin Magali Taiel Leigh Shaw Chief Executive Officer Chief Financial Officer Chief Medical Officer VP of Regulatory Affairs PIXIUM VISION (Since 2011) DBV TECHNOLOGIES (2012-2015) ProQR THERAPEUTICS (2016-2018) UNITED NEUROSCIENCE (2017-2020) FOVEA PHAR
Slide 5: Pipeline: solid and advanced product portfolio in ophthalmic Gene Therapy
Pipeline: solid and advanced product portfolio in ophthalmic Gene Therapy Product Technology Candidate Indication Research Preclinical Phase I/II Phase III Registration REVERSE: Phase III top-line data reported in Apr (48w) & Oct (72w) 2018 LHON ND4 (EU) and in May 2019 (96w) MAA Filed LUMEVOQ® (FDA
Slide 6: Rich upcoming news flow with numerous inflexion points
Rich upcoming news flow with numerous inflexion points Q4 Q2 MAA REFLECT decision Q1 September Week 78 BLA Commercial MAA filing readout filing launch Europe 2020 2021 2022 GS030 Q3 Q1 H2 Completion of patient Early findings Phase I-II PIONEER enrollment in PIONEER from first patients interim top-
Slide 7: LUMEVOQ (GS010) in LHON-ND4
LUMEVOQ® (GS010) in LHON-ND4 Last Phase III ongoing in Leber Hereditary Optic Neuropathy Commercial preparation ongoing for 2022 European launch
Slide 8: 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 9: 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 10: Unparalleled clinical benefit demonstrated with LUMEVOQ in two Phase III studies
Unparalleled clinical benefit demonstrated with LUMEVOQ® (GS010) in LHON in two Phase III studies 5 lines bilateral improvement of visual acuity Change from NADIR in ETDRS letter equivalents Change from NADIR in ETDRS letter equivalents Week 96 Week 96 n Mean (SD) +5 Lines n Mean (SD) Considered cli
Slide 11: Other data complement the finding on sustained bilateral improvement
Other data complement the finding on sustained bilateral improvement Insights on Mechanism of Bilateral Effect Excellent Tolerability • Non-human primate study • No serious adverse events in detected/quantified GS010 viral LUMEVOQ®-treated eyes, and no vector DNA in many tissue discontinuation
Slide 12: Indirect comparison as a cornerstone for EMA Filing
Indirect comparison as a cornerstone for EMA Filing External control group needed because of bilateral improvement in RESCUE and REVERSE trials • Contralateral effect eliminated the control group formed by the sham eyes, as defined in the studies’ designs • EMA scientific advice highlighted the impo
Slide 13: LUMEVOQ modifies disease outcome
LUMEVOQ® modifies disease outcome 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 (LogMAR) * * * Treated: All pati
Slide 14: LUMEVOQ shows meaningful improvement on Quality of Life metrics
LUMEVOQ® shows meaningful improvement on Quality of Life metrics NEI VFQ-25 Results from REVERSE study Mean change from baseline (absolute score) at week 96 Composite score** Near activities Distance activities Dependency Role difficulties General vision Mental health 10 5,8 8 14 8,6 35 12 6,7 35 20
Slide 15: Last ongoing Phase III trial: REFLECT to assess efficacy and safety of bilateral injection
Last ongoing Phase III trial: REFLECT to assess efficacy and safety of bilateral injection Double-masked, confirmatory study under Special Protocol Assessment from FDA Q2 2021 LUMEVOQ® Patient inclusion criteria Design Endpoints at Week 78 REFLECT Week 78 Group 1 Primary Read-out • Difference in cha
Slide 16: European Commercial Strategy – Leveraging LUMEVOQ Trial Centers to Build Network of LHON Centers
European Commercial Strategy – Leveraging LUMEVOQ® Trial 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 patient m
Slide 17: European Reimbursement Strategy – Short Term Revenues Generation Expected in H1 2022
European Reimbursement Strategy – Short Term Revenues Generation Expected in H1 2022 Commercial Launch (L) L + 12 Months L + 24 Months Q1 2022 Free pricing upon approval; benefit Negotiated price after 12 months; price assessment; price negotiations influence on other markets 3 Largest EU countries
Slide 18: Compassionate Use for LUMEVOQ (GS010)
Compassionate Use for LUMEVOQ® (GS010) Seeking use of an investigational medication under circumstances a patient may not be able to participate in a clinical trial and before MA/BLA approval by regulatory authorities • 4 individual patients Expanded Access INDs • “ATU Nominative” - named patient Te
Slide 19: Second product candidate targeting photoreceptor
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 (Age-Related Macular Degeneration) • Blinding genetic disease caused by mutations • Early (dry-form) AMD evolves with age into late in over 100 different genes AMD,
Slide 21: GS030: using Gene Therapy to rejuvenate production of light-sensitive protein and restore vision
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 Restoration of a functional vision in P23H rats Expression of ChrR-tdT in midget cells of Light-induced visual evoked cortical responses monkey perifovea In vivo in NHP assessment 6
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 Best- Tolerated Dose Data Safety Monitoring Prior to Dose Escalation 4 weeks post-injection of 3rd (last) patient of
Slide 24: Building high strategic value
Building high strategic value
Slide 25: A company developing innovative and versatile technology platforms nearing commercialization
A company developing innovative and versatile technology platforms nearing commercialization and evolving in an area where value is increasingly being recognized by the market GenSight at the forefront of Gene Therapy with Gene Therapy increasingly attracts interest LUMEVOQ® and Beyond: potential pr
Slide 26: GenSight Biologics in numbers
GenSight Biologics in numbers Key financial information Company Overview Share price evolution and trading volume Market Cap*: € 341m Analyst Coverage Share price € Trading volume m Cash Position** (Sep 30, 2020): € 18.1m 6 15 • Chardan: Gbola Amusa (US) 5 4 10 Outstanding Shares: 40.9m 3 • Bryan Ga
Slide 27: Appendix
Appendix
Slide 28: RESCUE & REVERSE Phase III trials with unilateral injection demonstrated unprecedented improvement
RESCUE & REVERSE Phase III trials with unilateral injection demonstrated unprecedented improvement Different patient Same design Visual recovery at Week 96 and vs natural history inclusion criteria • Double-masked, multi- center REVERSE • One eye randomized to GS010; other eye received sham injectio
Slide 29: Visual Acuity: Improvement of BCVA from NADIR
Visual Acuity: Improvement of BCVA from NADIR Visual Acuity deteriorates to a low point before recovering significantly in both eyes Change from NADIR in ETDRS letter Change from NADIR in ETDRS letter equivalents equivalents Week 96 Week 96 +5 n Mean (SD) n Mean (SD) Lines Considered All-GS010 eyes
Slide 30: 3-year long-term follow-up: sustained efficacy and safety
3-year long-term follow-up: sustained efficacy and safety Change from NADIR in ETDRS letter equivalents Year 2 Year 3 post-injection post-injection n Mean (SD) Mean (SD) n=37 n=34 All-GS010 eyes 61 +18.8 (15.3) +20.5 (18.3) CLIN06 – long-term follow-up study All-sham eyes 61 +17.3 (14.6) +19.4 (18.5
Slide 31: REVERSE and RESCUE demonstrate that over 70% of patients benefit from treatment
REVERSE and RESCUE demonstrate that over 70% of patients benefit from treatment REVERSE RESCUE 80% 70% 60% 76% of REVERSE subjects achieved at least 15 letters 50% improvement vs nadir in one or two eyes 71% 76% 40% 30% 71% of RESCUE subjects achieved at least 15 letters improvement vs nadir in one
Slide 32: GS010 viral vector DNA detection in uninjected eye of monkeys supports bilateral effect
GS010 (LUMEVOQ®) viral vector DNA detection in uninjected eye of monkeys supports bilateral effect in REVERSE and RESCUE Phase III trials Viral vector DNA detected in uninjected eye potential mechanism for bilateral effect in REVERSE and RESCUE • Three test monkeys injected in one eye using dose e
Slide 33: European Commercial Strategy - Facilitate and Speed Up Patient Access to LUMEVOQ
European Commercial Strategy - Facilitate and Speed Up Patient Access to LUMEVOQ® Initial consultations and Confirmed diagnosis and Onset Follow-up care misdiagnoses treatment Delay in recognizing onset of Lack of expertise in recognizing visual loss LHON Referral to wrong specialist Barrier
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