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FOCUS Interim Results: GT005 Gene Therapy Phase I/II Study for the Treatment of Geographic Atrophy

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FOCUS Interim Results: GT005 Gene Therapy Phase I/II Study for the Treatment of Geographic Atrophy FOCUS Interim Results: GT005 Gene Therapy Phase I/II Study for the Treatment of Geographic Atrophy Angiogenesis, Exudation, and Degeneration 2021 Nadia K. Waheed, MD, MPH Associate Professor, Tufts University School of Medicine Chief Medical Officer, Gyroscope Therapeutics FEBRUARY 12, 2021 Financial Disclosures Office Holder • Gyroscope Therapeutics Consultant • Apellis, Nidek, Boehringer Ingelheim Grants/Research Support • Carl Zeiss Meditec, Heidelberg, Nidek, Optovue, Regeneron, Topcon, Shareholder • Ocudyne Angiogenesis, Exudation, and Degeneration 2021 2 CFI Take Home Messages CFH • Complement over-activation has been implicated in AMD disease pathogenesis C3b • Complement Factor I (CFI) is a natural down-regulator of the complement system • GT005 is an investigational gene therapy designed to induce expression of CFI after subretinal delivery • GT005 has been well tolerated to date in the FOCUS Phase I/II trial in patients with GA • Early data show majority of patients had increased vitreous CFI and downstream modulation of complement biomarkers • Randomized controlled Phase II trials evaluating safety and efficacy of GT005 are ongoing Angiogenesis, Exudation, and Degeneration 2021 3 Strong Evidence of Role of Overactive Complement System in AMD Pathology Genetics Clinical Data Investigational complement inhibitors have shown a 28-29% reduction in GA progression (vs sham) in recent Phase 2 studies* Forest DL et al, 2015, Dis Model Mech, 8(5):421-427 Fritsche et al, 2016, Nature Genetics, 48 (2): 134-143 Lao DS et al, 2020, Ophthalmology, 127 (2): 186-195 • Biochemical analysis of drusen shows • Variants in multiple complement genes presence of multiple components of (including CFI) shown to be associated the complement pathway with increased risk of developing AMD *Liao DS et al, 2020, Ophthalmology.127(2):186-195 Jaffe GJ et al, 2020, Ophthalmology.1:S0161-6420(20)30845-9 Angiogenesis, Exudation, and Degeneration 2021 4 CFI: A Natural Inhibitor of the Alternative Pathway CFI – THE BODY’S DOW N NATURAL REGULATOR REGULATION C3b • CFI’s function is to keep the complement system in balance • Given this critical role, we believe CFI is well suited for gene therapy CFI • GT005 is designed to induce production AMPLIFICATION of this natural regulator TERMINAL PATHWAY AND CELL DEATH CFD CFB Angiogenesis, Exudation, and Degeneration 2021 5 GT005: Using Clinically Proven AAV2 Vector to Produce CFI • Designed to enable cellular transduction and induce secretion of CFI • Potential to allow for constitutive expression of CFI after single administration • Potential to avoid saw tooth dynamics of repeated intravitreal injections of medications • AAV2 clinically predicated with persistent durability at 3 years in other programs* ITR = inverse terminal repeat; CAG = CAG promotor; CMV = CMV promotor; CFI = complement factor I; WPRE = Woodchuck Hepatitis Virus (WHP) Posttranscriptional Regulatory Element which is a DNA sequence that, when transcribed creates a tertiary structure enhancing expression; bGHpA = poly A signal. *Maguire AM et al, 2019, Ophthalmology; 126:1273-1285 Angiogenesis, Exudation, and Degeneration 2021 6 FOCUS Phase I/II Open-Label Study to Evaluate GT005 Safety and Tolerability • Completed dose escalation and currently enrolling dose-expansion cohort • 22 patients dosed as of December 2020; data collection ongoing • Measuring transgene expression and pharmacodynamic markers by vitreous sampling • Interim data available from patients in Cohorts 1, 2, 3 and initial patients dosed in Cohort 4 Dose escalation Dose expansion OrbitTM Subretinal Delivery System OrbitTM Subretinal Delivery System (Transvitreal) (Transvitreal) Integration Expansion First patient dosed January 2019 First patient dosed March 2020 First patient dosed October 2020 Planned Dosing complete Enrolling Cohort 5 complete Cohort 3 Cohort 4 Cohort 6 Cohort 7 Dose 3 Doses 1, 2, 3 Dose 3 Dose TBD n=4 n = up to 20 n=3 n= up to 20 Cohort 2 Cohort 5 Dose 2 n=4 Dose 2 n=3 Cohort 1 Dose 1 n=3 Safety Evaluation (after 3 participants complete 5 w eeks follow-up) Angiogenesis, Exudation, and Degeneration 2021 7 3 Dose Levels of GT005 Were Well Tolerated in Initial Cohorts Ocular Treatment-Emergent Adverse Events (TEAEs) in Cohorts 1-4 Cohort 1 (n=3) Cohort 2 (n=4) Cohort 3 (n=4) Cohort 4 (n=8) Dose: (2E10 vg) Dose: (5E10 vg) Dose: (2E11 vg) Dose: (5E10-2E11vg) TEAEs (n=4) TEAEs (n=7) TEAEs (n=6) TEAEs (n=4) • Cataract • Age-related macular degeneration* • Cataract • Cataract • Cataract (fellow eye) • Blepharitis (both eyes) • Choroidal neovascularisation • Hallucination • Dry eye • Cataract • Eye contusion (fellow eye) • Intraocular pressure increased • Eye Oedema • Conjunctivitis viral (both eyes) • Iridocyclitis • Visual acuity reduced • Dry age-related macular • Retinal haemorrhage degeneration (fellow eye) † • Vitreous floaters • Glaucoma (fellow eye) • Glaucoma • No dose-related trends in frequency/type • 1 possible GT005-related adverse event of AEs - Choroidal neovascularization treated with anti-VEGF (moderate severity) • No GT005-related SAEs • 12 surgery-related adverse events • No safety signal on laboratory - 9 mild (1 corneal abrasion post op causing vision decrease) parameters - 3 moderate (2 cataracts; 1 sub-RPE injection) • Other ocular AEs in study eye of note - 2 study eye AEs reported with increased IOP - 1 with history of glaucoma, Tmax 27, resolved with drops - 1 with IOP elevation at wk1 visit. Tmax 29, resolved at 5 weeks *Miscoded (Sub-RPE injection) †BCVA drop in fellow eye secondary to GA progression Data as of December 2020 Angiogenesis, Exudation, and Degeneration 2021 8 Interim Data Shows No Signs of GT005-Related Inflammation Interim FOCUS Immunogenicity Data • GT005 associated with clinically benign • AAV2 antibodies (n=12) immunogenicity • Pre-existing antibodies to AAV2 vector in 9 patients • No increases in AAV2 antibody titers post-treatment • No antibody-mediated immunogenicity to the • T-cell immunogenicity (n=11) CFI transgene • Pre-existing T-cell immunogenicity to AAV2 vector in 2 patients • Increases in T-cell response post-treatment that were transient in • No association of immune responses with nature and at a low magnitude adverse events • Anti-CFI antibodies (n=8) • No anti-CFI antibodies seen in patients tested to date Data as of December 2020 Angiogenesis, Exudation, and Degeneration 2021 9 Secondary Endpoints Data Visual Acuity Preserved at 48 Weeks Post Treatment Mean (95% CI) BCVA Mean (95% CI) BCVA change from Baseline 80 10 70 5 60 0 50 40 -5 BCVA 30 BCVA change (ETDRS (ETDRS letters) -10 (ETDRS (ETDRS letters) 20 -15 10 0 -20 0 4 8 12 16 20 24 28 32 36 40 44 48 0 4 8 12 16 20 24 28 32 36 40 44 48 n=22 n=10 n=8 n=22 n=10 n=8 Study Week Study Week Study Eye Fellow Eye Study Eye Fellow Eye Data as of December 2020 Angiogenesis, Exudation, and Degeneration 2021 10 Transgene Expression: Consistent and Durable Increase in Vitreous CFI levels • 9/10 patients had elevated CFI levels post-GT005 administration; average increase of 146% from baseline • 1st patient treated continues to have sustained CFI increase at 84 weeks • Increases in vitreal CFI seen in both RV and non-RV patients 2500 1200 Low dose (2E10 vg) e n 2000 Medium dose (5E10 vg) i l m e ) High dose (2E11 vg) o 1200 s r L a f Open symbol = RV GA b I m / Closed symbol = Broad GA F 800 m g ) 800 C o L n r ( f n m I / i e F g n e n C i ( 400 l s n e a i 400 s e e r a s c a b 0 e n I r c n I 0 -400 2e10 vg 5e10 vg 2e11 vg (n=2) (n=2) (n=6) 0 10 20 30 40 50 60 70 80 90 Dose Study week Maximum CFI level (at any timepoint) above baseline; DataD asata of c uDecembert: 05OCT202 20200 Bars=mean SEM; only data from 12 weeks included DataData ascu oft: 0December5OCT202 20200; Angiogenesis, Exudation, and Degeneration 2021 11 Previously Published Data Implies Significant Impact on Amplification Loop Proc Natl Acad Sci U S A. 2011 May 24;108(21):8761-6. Classical Pathway Lectin Pathway Alternative Pathway Ba Ba • Published data indicate that protective variants* Ba C3 in C3, CFB and CFH decrease complement Ba activity by 48% when compared to common risk Amplification Loop Ba variants [PNAS (2011) 108(21):8761-6] CFI CFH C3b CFB CFD • Ba is a direct readout of the amplification loop** CFH+CFI C5 • Inhibition of the amplification loop by CFI (GT005) iC3b C3c leads to a 41% drop in vitreal Ba levels, indicating significant inhibition of the amplification loop + C3d CR1+CFI MAC Degradation Cell lysis and death * C3 R102G, CFH V62I, CFB R102G ** It is unclear whether the relationship between Ba levels and lytic activity is linear Angiogenesis, Exudation, and Degeneration 2021 12 Significant Decreases in Ba and C3 Breakdown Product Vitreous Levels After GT005 Administration Average Decrease of 41% in Ba and 42% in C3 Breakdown Product Vitreous Levels Data as of December 2020 The assay used for Ba and C3 breakdown products measurements are standard and validated Statistical analysis: Wilcoxon matched pairs test (post-treatment compared to baseline); Data shown as Mean ± SD Angiogenesis, Exudation, and Degeneration 2021 13 Significant Correlation Between Increases in Local CFI Levels and Decreases in Ba with GT005 Notes: Data as of December 2020 Statistical analysis: Spearman correlation; Data used: latest available data point up to week 56 for individual patients Angiogenesis, Exudation, and Degeneration 2021 14 Phase II Studies Evaluate Two Patient Populations Primary Endpoint: GA Progression at 48 Weeks • 75 GA patients with CFI rare variants • 180 GA patients (broad
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