Ocular Therapeutix, Inc. · NASDAQ:OCUL
Ocular Therapeutix — Euretina 2026
88 slides from Ocular Therapeutix's conference presentation, Oct 2, 2026
Slide 1: Redefining the Management of nAMD
Redefining the Management of nAMD The Impact of Durability and Sustained Disease Control October 2, 2026 Euretina | Vienna, Austria MC-US-OTX-0298 (v1.0)
Slide 2: Forward-Looking Statements
Forward-Looking Statements Any statements in this presentation about future expectations, plans, and prospects for the Company, including statements regarding the development and regulatory status of the Company’s or its competitors’ product candidates, including statements regarding the SOL-1 Phase
Slide 3: Disclaimer
Disclaimer The following presentation discusses OTX-TKI - an investigational product candidate that has not been approved by the FDA or any other regulatory body; safety and effectiveness have not been established 3
Slide 4: Faculty
Faculty Prof. Arshad M. Khanani Prof. Adnan Tufail Prof. Dilsher S. Dhoot Dr. Patricio G. Schlottmann MD, MA, FASRS MBBS, MD, FRCOphth MD, FASRS MD Managing Partner, Director of Clinical Consultant Ophthalmic Surgeon at Adjunct Clinical Assistant Professor at Director of Research, Research, Director
Slide 5: Disclosures
Disclosures • Consultant: 4DMT, Abbvie, Adverum, Alcon Pharmaceuticals, Alimera Sciences, Inc., Alkeus, Allergan, ANI Pharmaceuticals, Amgen, Dilsher S. Dhoot Annexon, Apellis Pharmaceuticals, Inc., Astellas, Bausch and Lomb, Bayer Healthcare Pharmaceuticals, Inc., Biocryst, Coherus, EyePoint Pharma
Slide 6: Agenda
Agenda Opening Remarks SOL-1 Clinical Evidence Arshad M. Khanani, MD, MA, FASRS Dilsher S. Dhoot, MD, FASRS The Unmet Need Key Insights Adnan Tufail, MBBS, MD, FRCOphth Adnan Tufail MBBS, MD, FRCOphth Science Behind the Therapy Clinical Program Update Dilsher S. Dhoot, MD, FASRS Arshad M. Khanani, M
Slide 7: The Unmet Need
The Unmet Need Adnan Tufail MBBS, MD, FRCOphth 7
Slide 8: Anti-VEGF has Transformed Our Management of Neovascular AMD
Anti-VEGF has Transformed Our Management of Neovascular AMD Delivering anti-VEGF in clinical practice and achieving optimal outcomes remain challenging Brown DM, et al. N Engl J Med. 2006;355(14):1432–1444. Anti-VEGF, anti-vascular endothelial growth factor; AMD, age-related macular degeneration 8
Slide 9: Real World Outcomes Do Not Reflect Clinical Trial Results
However, Real World Outcomes Do Not Reflect Clinical Trial Results Report 1 Ophthalmology 2014 12,951 eyes; 11,135 patients; 92,976 ranibizumab injections. PRN regimen. Mean VA: baseline 55 → yr1 +2 (57) → yr2 +1 (56) → yr3 −2 (53). Median injections: 6/4/4 per year. 90%/84%/82% avoided moderate vis
Slide 10: Visual Acuity Outcomes in the Real World
Visual Acuity Outcomes in the Real World Evidence Base: Pivotal Trials, UK EMR Users Group, FRB & IRIS Registries Pivotal RCTs UK EMR data FRB Registry AAO IRIS® Registry Gold-standard efficacy data UK EMR. · NOD AMD Fight Retinal Blindness! — International USA National Registry • 14 UK NHS centres,
Slide 11: Injections per Year: Clinical Trials vs Real-World Practice
Injections per Year: Clinical Trials vs Real-World Practice VA Δ +11 +6 +6 +6 +5 +3 +2 +4 +11 +6 +4 +6 +4 +1 +1 14 14 12 12 12 12 Number of injections per year 10 10 8 7 8 7 7 7 6 6 6 6 6 6 5 5 4 4 4 4 3 2 2 0 0 Year 1 Year 2 RCT (monthly fixed)1 Brolucizumab (HAWK/HARRIER)2-4 Faricimab T&E (TENAYA/
Slide 12: Injections per Year: Clinical Trials vs Real-World Practice
Injections per Year: Clinical Trials vs Real-World Practice VA Δ +11 +6 +6 +6 +5 +3 +2 +4 +11 +6 +4 +6 +4 +1 +1 +2 +0 −2 -3 −2 14 14 14 14 12 12 12 12 12 12 Number of injections per year 10 10 10 10 8 7 8 8 8 7 7 7 6 6 6 6 6 6 6 6 5 5 5 5 5 5 4 4 4 4 4 3 4 4 2 2 2 2 0 0 0 0 Year 1 Year 2 Year 3 Year
Slide 13: Eyes with Excellent Baseline Vision Decline on Average in Real-World Studies
Eyes with Excellent Baseline Vision Decline on Average in Real-World Studies Visual Acuity Over Time N=92,976 Despite initial gains, eyes showed a sustained decline in VA over 3 years Writing Committee for the UK Age-Related Macular Degeneration EMR Users Group. The neovascular age-related macular d
Slide 14: Eyes with Excellent Baseline Vision Decline on Average in Real-World Studies
Eyes with Excellent Baseline Vision Decline on Average in Real-World Studies Visual Acuity Change in Vision Over Time Stratified by Baseline VA Worst baseline vision N=92,976 Best baseline vision Change in vision is dependent on Despite initial gains, eyes baseline status with eyes starting showed a
Slide 15: Actual Visual Acuity, NOT Change, is What is Meaningful to Patients
Actual Visual Acuity, NOT Change, is What is Meaningful to Patients Visual Acuity Change in Vision Mean Vision Over Time Stratified by Baseline VA Stratified by Baseline VA Worst baseline vision Best baseline vision N=92,976 Best baseline Worst vision baseline vision Change in vision is dependent on
Slide 16: Recent Data Supports Similar Association
Recent Data Supports Similar Association • Visual outcomes were evaluated by treatment exposure • ≥7 or <7 intravitreal anti-VEGF injections • 295,561 eligible eyes • 184,258 actively treated Barikian et al. Ophthalmology Retina 2026;10:71-80 BCVA, best-corrected visual acuity; VEGF, vascular endoth
Slide 17: The Injection–Outcome Relationship: More Injections = Better Vision
The Injection–Outcome Relationship: More Injections = Better Vision 5 4 4 Linear relationship 4 4 Between 4–10 injections, each additional injection 3 3 adds ~0.6 ETDRS letters Mean BCVA change (letters) 2 (n=49,485 eyes) 1 1 Under-treatment threshold -1 -2 ≤5 injections associated with mean letter
Slide 18: Why do Patients Discontinue Anti-VEGF Treatment?
Why do Patients Discontinue Anti-VEGF Treatment? • Multi-centre study of nAMD patients (n=2,302 patients) • 655 (28.5%) discontinued intravitreal anti-VEGF therapy • Early discontinuers were older and had higher baseline VA Reported reasons for discontinuation Contributing factors in the nAMD popula
Slide 19: Greater Retinal Thickness Variability Associated with Suboptimal Outcomes
Greater Retinal Thickness Variability During Anti-VEGF Therapy is Associated with Suboptimal Anatomic and Visual Outcomes Repeated cycles of fluid resolution and recurrence are reflected in fluctuations in retinal Retinal Thickness thickness over time Fluctuations Anti-VEGF Injection Weeks Developme
Slide 20: Greater Retinal Thickness Variability Associated with Suboptimal Outcomes
Greater Retinal Thickness Variability During Anti-VEGF Therapy is Associated with Suboptimal Anatomic and Visual Outcomes Retinal Thickness Fluctuations Development of Fibrosis and Atrophy Poorer Long-Term Visual Outcomes Highest retinal thickness variability had approximately double the odds of dev
Slide 21: Greater Retinal Thickness Variability Associated with Suboptimal Outcomes
Greater Retinal Thickness Variability During Anti-VEGF Therapy is Associated with Suboptimal Anatomic and Visual Outcomes Retinal Thickness Fluctuations Development of Vision deteriorates by ~9 letters Fibrosis and Atrophy between patients with most and least stable retinal Poorer Long-Term thicknes
Slide 22: Clinical Trials to Real-world Management with New Agents
Clinical Trials to Real-world Management with New Agents Limitation of recent clinical trial designs1 • Clinical trials aimed to get regulatory approval • Suggest extended interval in subgroups • Investigational drug subgroup extended but not active control • Non-inferiority 1. Talks J, et al. Eye (
Slide 23: Clinical Trials to Real-world Management with New Agents
Clinical Trials to Real-world Management with New Agents Limitation of recent clinical trial designs1 • Clinical trials aimed to get regulatory approval • Suggest extended interval in subgroups • Investigational drug subgroup extended but not active control • Non-inferiority Real-world evidence sugg
Slide 24: Summary: Current Status of nAMD Treatment
Summary: Current Status of nAMD Treatment Real-world outcomes do not match clinical trials We are not getting much better since their inception Visual acuity change from baseline dependent on starting acuity Ideally, more treatment should be given BUT limited by patient/caregiver/healthcare system f
Slide 25: Science Behind the Therapy
Science Behind the Therapy Dilsher S. Dhoot, MD, FASRS 25
Slide 26: Current Anti-VEGF Therapies Selectively Target Only Extracellular VEGF
Current Anti-VEGF Therapies Selectively Target Only Extracellular VEGF Aflibercept Faricimab Ang 2 Ranibizumab Bevacizumab VEGF-A VEGF-A Brolucizumab VEGF-B VEGF-C VEGF-C Current Anti-VEGF agents act PIGF VEGF-E VEGF-D extracellularly by binding selective ligands, like VEGF-A, Extracellular preventi
Slide 27: Tyrosine Kinase Inhibitors Act Intracellularly to Inhibit Receptor Signaling
Tyrosine Kinase Inhibitors Act Intracellularly to Inhibit Receptor Signaling VEGF-A VEGF-A VEGF-B VEGF-C VEGF-C Tyrosine Kinase Inhibitors (TKI) PIGF VEGF-E VEGF-D bind at the intracellular tyrosine kinase domains of VEGF receptors, inhibiting ATP binding Extracellular and preventing activation of p
Slide 28: OTX-TKI: Axitinib via Hydrogel Allows for Tunable Sustained Release
OTX-TKI: Axitinib via Hydrogel Allows for Tunable Sustained Release AXITINIB HYDROGEL TECHNOLOGY Multi-target Tyrosine Kinase Inhibitor (TKI) Bioresorbable, Sustained Drug Delivery Highly selective pan VEGF inhibitor1 Proprietary hydrogel Highly potent TKI2 Versatile, biocompatible, tunable platform
Slide 29: Axitinib: Highly Selective, Very Potent VEGFR Inhibitor
Axitinib: Highly Selective, Very Potent VEGFR Inhibitor IC50 for VEGF Receptor 2 (nM)1-3 VEGF Challenge Rabbit Model2 100 Lower IC50 values indicate higher potency VEGFR-2 10 VEGFR-2 2.9nM 3.3nM 1 Axitinib Axitinib 90X more 100X more potent potent 0.1 VEGFR-2 0.032nM 0.01 Axitinib Sunitinib Vorolani
Slide 30: Axitinib demonstrates strongest inhibition of all VEGF/PDGF/FGF Receptors
Axitinib demonstrates strongest inhibition of all VEGF/PDGF/FGF Receptors AssayQuant (Fluorescence Assay)* Reaction Biology (Radiometric Assay)** 1 mM ATP Physiologic Testing Conditions 1 mM ATP Physiologic Testing Conditions Axitinib intraocular conc = 1.03 to 1.13 µM Axitinib intraocular conc = 1.
Slide 31: Comparison of Hydrogel vs Other Retinal Polymer Implants
Comparison of Hydrogel vs Other Retinal Polymer Implants Implant Implant Dimensions Needle Matrix Degradation Polymer Implant Remnants (Full View) (Length x Diameter) Size & Release^^ Byproduct at 12 Months 6mm x OZURDEX Lactic acid and Remnant 0.46mm* 22-gauge* glycolic acid† lasts ~12 months ‡ Two
Slide 32: OTX-TKI is Designed to Release Axitinib and Maintain Intraocular Drug Levels
OTX-TKI is Designed to Release Axitinib and Maintain Intraocular Drug Levels Over Time Axitinib concentrations following a single OTX-TKI in NHPs1 OTX-TKI In Vivo Delivery Profile1 Hydrogel Drug Zero-order kinetics Bioresorption Clearance Concentration (ng/g) 100000 Rapid, continuous and consistent
Slide 33: OTX-TKI Release with Complete Bioresorption
OTX-TKI is Designed to Release Axitinib and Maintain Intraocular Drug Levels Over Time with Complete Bioresorption Axitinib concentrations following a single OTX-TKI in NHPs1 OTX-TKI In Vivo Delivery Profile1 Hydrogel Drug Zero-order kinetics Bioresorption Clearance Concentration (ng/g) 100000 Rapid
Slide 34: Histology of Ocular Tissues Demonstrates Axitinib Elution with No Inflammatory Cells
Histology of Ocular Tissues Demonstrates Axitinib Elution with No Inflammatory Cells Histology of Ocular Tissues from 0.7 mg OTX-TKI in NHPs Microscopic Findings 5002 5603 White, refractile, basophilic, granular axitinib in the vitreous No inflammatory cells in vitreous, retina, or AC 500 um 1000 um
Slide 35: OTX-TKI is Designed for Seamless, Immediate Adoption
OTX-TKI is Designed for Seamless, Immediate Adoption Ideal Target Single Bioresorbable Optimizing for Product Profile Hydrogel Retina Practices Pan-VEGF and PDGF Familiar IVT No remnants3 inhibitory activity1 injection with 25g needle6 Axitinib has best-in-class Predictable Tunable hydrogel4 (TKI) p
Slide 36: SOL-1: Trial Design and Baseline Characteristics
SOL-1: Trial Design and Baseline Characteristics Arshad M. Khanani, MD, MA, FASRS 36
Slide 37: SOL: OTX-TKI Phase 3 Clinical Program in nAMD
SOL: OTX-TKI Phase 3 Clinical Program in nAMD Evaluating the efficacy, durability and safety of OTX-TKI in nAMD Registrational Trials Extension Study SOL-1 SOL-R SOL-X Phase 3 Phase 3 Superiority Trial Non-Inferiority Trial Open-Label Extension Durability of a single Repeat dosing Long-term safety a
Slide 38: SOL: OTX-TKI Phase 3 Clinical Program in nAMD
SOL: OTX-TKI Phase 3 Clinical Program in nAMD Evaluating the efficacy, durability and safety of OTX-TKI in nAMD Registrational Trials Extension Study SOL-1 SOL-R SOL-X Phase 3 Phase 3 Superiority Trial Non-Inferiority Trial Open-Label Extension Durability of a single Repeat dosing Long-term safety a
Slide 39: OTX-TKI Evaluated in a Superiority nAMD Trial Designed to Align with FDA Guidance
OTX-TKI Evaluated in a Superiority nAMD Trial Designed to Align with FDA Guidance FDA Guidance for nAMD Trials SOL-1 Trial Design One comparator arm should have the same dosing Both study arms have Dosing Schedule schedule as the investigational drug1 same dosing schedule Sham injections are not rec
Slide 40: Eligibility and Enrollment Criteria
Eligibility and Enrollment Criteria Key Inclusion Criteria Week -8 -4 R 1:1 Aflibercept 2 mg OTX-TKI* (0.45 mg) Study visit SOL-1 OTX-TKI 0.45 mg Aflibercept (2 mg) Randomization (Day 1) Enrollment Criteria Randomization Criteria Screening Visit (Week -8) Baseline Visit (Day 1) Treatment naïve CNV d
Slide 41: Strong Subject Retention Maintained through Week 52
Strong Subject Retention Maintained through Week 52 Subject Disposition Randomized N=344 OTX-TKI 0.45 mg Aflibercept 2 mg N=172 N=172 Discontinued before Week 52 (n=11) Discontinued before Week 52 (n=5) • Adverse event (n=1) • Adverse event (n=2) • Subject withdrew consent* (n= 6) • Subject withdrew
Slide 42: Excellent Vision at Baseline; Majority Demonstrated ≥10-letter Gain from Screening
Excellent Vision at Baseline; Majority Demonstrated ≥10-letter Gain from Screening Best-Corrected Visual Acuity Screening Visit (Week -8) OTX-TKI 0.45 mg Aflibercept 2 mg Study Eye Characteristics N = 172 N = 172 BCVA, ETDRS letter score, mean (SD) 70.9 (11.3) 69.5 (10.8) Snellen Equivalent ~20/40 ~
Slide 43: Anatomic Improvement Observed After Two Aflibercept Injections
Anatomic Improvement Observed After Two Aflibercept Injections Central Subfield Thickness Screening Visit (Week -8) OTX-TKI 0.45 mg Aflibercept 2 mg Study Eye Characteristics N = 172 N = 172 BCVA, ETDRS letter score, mean (SD) 70.9 (11.3) 69.5 (10.8) Snellen Equivalent ~20/40 ~20/40 CSFT, µm, mean (
Slide 44: SOL-1 Evaluated a Distinct nAMD Cohort with Excellent Baseline Vision
SOL-1 Evaluated a Distinct nAMD Cohort with Excellent Baseline Vision Baseline BCVA Across nAMD Trials 85 80 75 Baseline BCVA 70 65 TENAYA & LUGANO6 LUCERNE2 60 PULSAR3 nAMD Trials mean BCVA: 52 to 66 letters 55 HAWK & VIEW 1 & CEDAR & (~20/100 to 20/50) HARRIER5 VIEW 21 SEQUOIA4 50 45 150 200 250 3
Slide 45: SOL-1 Evaluated a Distinct nAMD Cohort with Excellent Baseline Vision
SOL-1 Evaluated a Distinct nAMD Cohort with Excellent Baseline Vision Baseline BCVA Across nAMD Trials 85 Baseline SOL-1 80 mean BCVA: 81 letters (~20/25) 75 mean CSFT: 219 µM Screening Baseline BCVA 70 65 TENAYA & LUGANO6 LUCERNE2 60 PULSAR3 nAMD Trials mean BCVA: 52 to 66 letters 55 HAWK & VIEW 1
Slide 46: SOL-1: Results
SOL-1: Results Patricio G. Schlottmann, MD 46
Slide 47: Primary Endpoint Successfully Met
Primary Endpoint Successfully Met Statistically Significant Higher Proportion of Subjects Maintained Visual Acuity* with OTX-TKI vs. Aflibercept 2 mg at Week 36 100% P = 0.0006 Proportion of Subjects, % 74.1% 75% 55.8% 50% 25% 0% Week 36 OTX-TKI 0.45 mg (n=170) Aflibercept 2 mg (n=172) *Maintenance
Slide 48: Sustained Through Week 52
Difference in Proportion of Subjects Maintaining Visual Acuity Sustained Through Week 52 Key Secondary Endpoint: Maintenance of Visual Acuity* at Week 52 P = 0.0006 100% P < 0.0001 100% Proportion of Subjects, % Proportion of Subjects, % 74.1% 75% 75% 65.9% 55.8% 50% 50% 44.2% 25% 25% 0% 0% Week 36
Slide 49: Most Subjects (~75%) in the OTX-TKI Arm Did Not Receive Rescue Injections at Week 36
Most Subjects (~75%) in the OTX-TKI Arm Did Not Receive Rescue Injections at Week 36 Proportion of Rescue-Free Subjects Time to First Rescue Treatment Post Hoc Analysis Key Secondary Endpoint 100% P = 0.0856* P = 0.0006* 1.0 80.6% 0.9 74.7% P = 0.0005† 72.1% 0.8 Probability of Not Having 75% Proport
Slide 50: Treatment Burden Reduction vs 2 mg Aflibercept Q8W at ~1 Year
Treatment Burden Reduction vs 2 mg Aflibercept Q8W at ~1 Year LOADING DOSES INCLUDED LOADING DOSES NOT INCLUDED Faricimab 14% (6.4 vs 7.4) 45% (2.4 vs 4.4) TENAYA/LUCERNE1,2 Phase 3 - to Week 48 Aflibercept 8mg 29% (5.49 vs 7.7) 47% (2.49 vs 4.7) PULSAR3,4 Phase 3 – up to Week 48 (weighted mean Q12
Slide 51: OTX-TKI Subjects Maintained Vision and Anatomy, Most Rescue-Free
On Average, OTX-TKI Subjects Maintained Vision and Anatomy, with Most Remaining Rescue-Free Post Hoc Analysis: Mean Change in BCVA and CSFT from Screening 10 from Screening BCVA, Mean (±SE) Change ETDRS Letters 78.0 Baseline 5 BCVA 80.8 74.7% Rescue free 0 Weeks -8 -4 0 4 8 12 16 20 24 28 32 36 36 0
Slide 52: OTX-TKI Subjects Maintained Vision and Anatomy, Most Rescue-Free
On Average, OTX-TKI Subjects Maintained Vision and Anatomy, with Most Remaining Rescue-Free Post Hoc Analysis: Mean Change in BCVA and CSFT from Screening 10 from Screening BCVA, Mean (±SE) Change ETDRS Letters Baseline 5 BCVA 76.6 79.2 0 Weeks -8 -4 0 4 8 12 16 20 24 28 32 36 36 0 56.4% from Screen
Slide 53: OTX-TKI Subjects Maintained Vision and Anatomy, Most Rescue-Free
On Average, OTX-TKI Subjects Maintained Vision and Anatomy, with Most Remaining Rescue-Free Post Hoc Analysis: Mean Change in BCVA and CSFT from Screening 10 from Screening BCVA, Mean (±SE) Change ETDRS Letters 78.0 Baseline 5 BCVA 76.6 80.8 74.7% Rescue free 79.2 0 Weeks -8 -4 0 4 8 12 16 20 24 28
Slide 54: OTX-TKI Delivered Sustained Fluid Control
OTX-TKI Delivered Sustained Fluid Control Prespecified Exploratory Analysis: Mean Change in Intra-Retinal and Sub-Retinal Fluid Volume Week 24 Week 36 Mean Volume Mean Volume 70 54 nL 59 nL Total Fluid Volume: 60 nL 60 Mean (±SE) Change from Baseline , nL 50 40 Total Fluid Volume: 30 nL 30 32 nL 31
Slide 55: No Treatment or Procedure Related SAEs
No Treatment or Procedure Related SAEs Non-Ocular and Ocular Adverse Events in the Study Eye OTX-TKI 0.45 mg Aflibercept 2 mg Subjects with AEs Through Week 52, n (%) n = 170 n = 172 Non-ocular AEs ≥ 1 AE 84 (49.4) 73 (42.4) ≥ 1 SAE 19 (11.2) 21 (12.2) ≥ 1 treatment-related AE 0 0 ≥ 1 treatment-rela
Slide 56: OTX-TKI was Generally Well Tolerated
OTX-TKI was Generally Well Tolerated Ocular Adverse Events in the Study Eye Subjects with Ocular AEs (> 2%) OTX-TKI 0.45 mg Aflibercept 2 mg Through Week 52, n (%) n = 170 n = 172 Vitreous floaters 21 (12.4) 2 (1.2) • Nine IOI events observed in study eyes of 7 subjects in the OTX-TKI Cataract 12 (7
Slide 57: Appearance of Floaters May Correspond to Hydrogel Dissolution and Drug Elution
Appearance of Floaters May Correspond to Hydrogel Dissolution and Drug Elution Evaluation of Vitreous Floaters Time to Onset OTX-TKI Aflibercept 0.45 mg 2 mg n = 23* n=2 Time to onset, days Mean (SD) 187.9 (64.0) 164.5 (164.8) Median 207.0 164.5 BCVA change from prior visit before onset of floaters,
Slide 58: Incidence of Macular Atrophy in SOL-1
Incidence of Macular Atrophy in SOL-1 Safety Analysis Any Macular Atrophy1 Macular Atrophy in the Central 1mm1 Percent Change from Baseline 10% Percent Change from Baseline 40% OTX-TKI 0.45mg: 1.2% OTX-TKI 0.45mg: 0.7% Aflibercept 2 mg: 2.7% 9% Aflibercept 2 mg: 0.6% 35% Proportion of Subjects, % 8%
Slide 59: SOL-1 Results Demonstrate Potential Treatment Benefit of OTX-TKI
SOL-1 Results Demonstrate Potential Treatment Benefit of OTX-TKI Superiority Primary Endpoint Successfully Met OTX-TKI Demonstrated Superior Maintenance of Vision* Compared to Aflibercept 2mg at Week 36 OTX-TKI demonstrated a First trial to demonstrate Visual and anatomic stability well-tolerated sa
Slide 60: Clinical Evidence
Clinical Evidence Dilsher S. Dhoot, MD, FASRS 60
Slide 61: OTX-TKI Case
-8 Weeks OTX-TKI Case 1 BCVA: 74 Ltrs (-10) CSFT: 467 µm BCVA Baseline 95 BCVA: 84 Ltrs Snellen: 20/20 90 20/20 20/20 CSFT: 285 µm ETDRS Letters 85 80 20/32 Week 4 BCVA (Δ): 83 (-1) Ltrs 75 CSFT (Δ): 320 (+35) µm 70 Week 12 65 BCVA (Δ): 82 (-2) Ltrs -8 -4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 CSFT
Slide 62: OTX-TKI Case
-8 Weeks OTX-TKI Case 2 BCVA: 75 Ltrs CSFT: 344 µm BCVA Baseline BCVA: 86 Ltrs 95 Snellen: 20/20 New hemorrhage CSFT: 247 µm 90 20/20 ETDRS Letters 85 20/25 20/25 Week 4 80 BCVA (Δ): 84 (-2) Ltrs CSFT (Δ): 254 (+7) µm 75 70 Week 12 65 BCVA (Δ): 85 (-1) Ltrs -8 -4 0 4 8 12 16 20 24 28 32 36 40 44 48
Slide 63: OTX-TKI Case
OTX-TKI Case 3 -8 Weeks BCVA BCVA: 74 Ltrs 95 CSFT: 448 µm 90 20/20 ETDRS Letters Baseline 85 BCVA: 85 Ltrs Snellen: 20/20 80 CSFT: 261 µm 20/25 75 20/25 70 Week 4 BCVA (Δ): 85 (0) Ltrs 65 CSFT (Δ): 257 (-4) µm -8 -4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 Time (Weeks) Week 12 CSFT BCVA (Δ): 84 (-1)
Slide 64: OTX-TKI Case
-8 Weeks OTX-TKI Case 4 BCVA: 64 Ltrs CSFT: 363 µm BCVA 95 Baseline BCVA: 85 Ltrs 90 20/20 20/16 Snellen: 20/20 85 CSFT: 273 µm ETDRS Letters 80 Week 4 75 20/25 BCVA (Δ): 82 (-3) Ltrs 70 CSFT (Δ): 274 (+1) µm 65 60 Week 16 -8 -4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 BCVA (Δ): 83 (-2) Ltrs Time (Wee
Slide 65: Aflibercept Case
Aflibercept Case 1 -8 Weeks BCVA BCVA: 78 Ltrs CSFT: 389 µm 90 20/20 ETDRS Letters 80 Baseline BCVA: 84 Ltrs 20/40 Snellen: 20/20 70 CSFT: 273 µm (15 letter loss) 60 20/50 Week 4 BCVA (Δ): 83 (-1) Ltrs 50 CSFT (Δ): 282 (+9) µm -8 -4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 Time (Weeks) CSFT Week 8 BCV
Slide 66: Aflibercept Case
Aflibercept Case 1 Screening Study Per-Protocol Rescue Investigator Aflibercept Aflibercept (2 mg) Aflibercept (2mg) Discretion Rescue (2mg) -8 Weeks Week 24 BCVA: 78 Ltrs BCVA (Δ): 74 (-10) Ltrs CSFT: 389 µm CSFT (Δ): 335 (+62) µm Baseline Week 28 BCVA: 84 Ltrs BCVA (Δ): 74 (-10) Ltrs Snellen: 20/2
Slide 67: Aflibercept Case
-8 Weeks Aflibercept Case 2 BCVA: 80 Ltrs CSFT: 394 µm BCVA 20/16 Baseline BCVA: 89 Ltrs 90 Snellen: 20/16 20/25 20/25 80 CSFT: 328 µm ETDRS Letters 70 (15 letter loss) Week 4 60 BCVA (Δ): 78 (-11) Ltrs CSFT (Δ): 349 (+21) µm 50 40 Week 16 30 BCVA (Δ): 83 (-6) Ltrs -8 -4 0 4 8 12 16 20 24 28 32 36 4
Slide 68: Aflibercept Case Study
Aflibercept Case 2 Study Screening Aflibercept Per-Protocol Rescue Investigator Aflibercept (2 mg) (2mg) injection Aflibercept (2mg) Discretion Rescue administration -8 Weeks BCVA: 80 Ltrs CSFT: 394 µm Week 28 Baseline BCVA: 89 Ltrs BCVA (Δ): 82 (-7) Ltrs Snellen: 20/16 CSFT (Δ): 421 (+93) µm CSFT:
Slide 69: Key Insights
Key Insights Adnan Tufail MBBS, MD, FRCOphth 70
Slide 70: SOL-1 Evaluated a Distinct nAMD Cohort with Excellent Baseline Vision
SOL-1 Evaluated a Distinct nAMD Cohort with Excellent Baseline Vision SOL-1 : Mean Change in BCVA from Screening Baseline BCVA Across nAMD Trials Post Hoc Analysis 10 Mean (±SE) Change from Screening BCVA, ETDRS 78.0 Letters Baseline 5 BCVA 76.6 80.8 79.2 0 -8 -4 0 4 8 12 16 20 24 28 32 36 36 Weeks
Slide 71: Does Reduced Potential for Visual Acuity Gain Occur in Pivotal Trials?
Does Reduced Potential for Visual Acuity Gain Occur in Pivotal Trials? Sub-Analysis of ANCHOR and MARINA In good VA group (baseline 70-85 letters), ~4 letter loss at M6 from peak Tufail A, et al.. Br J Ophthalmol. 2020;104(5):672-677. BCVA, best-corrected visual acuity; ETDRS, Early Treatment Diabet
Slide 72: Does Reduced Potential for Visual Acuity Gain Occur in Pivotal Trials?
Does Reduced Potential for Visual Acuity Gain Occur in Pivotal Trials? Sub-Analysis of ANCHOR and MARINA M2 M6 Tufail A, et al.. Br J Ophthalmol. 2020;104(5):672-677. BCVA, best-corrected visual acuity; ETDRS, Early Treatment Diabetic Retinopathy Study; M, month 73
Slide 73: Emulating SOL-1 Entry Criteria in the IRIS Registry
Using a similar methodology, we emulated SOL-1 entry criteria to the IRIS Registry Thomas DS, et al. Clin Transl Sci. 2021;14(3):1166-1175. EHRs, electronic health records; EM, exact matching; IPTW, inverse probability of treatment weighting; PSM, propensity score matching; SOC, standard of care; UC
Slide 74: How Does SOL-1 Data Align with Real World Outcomes?
How Does SOL-1 Data Align with Real World Outcomes? SOL-1 Clinical Trial SOL-1 Emulated Population Post Hoc Analysis IRIS Registry 10 10 79.0 from Screening BCVA, 80.8 Mean (±SE) Change Mean Change from Screening BCVA, ETDRS Letters ETDRS Letters 5 5 70.9 68.4 0 0 Weeks -8 -4 0 4 8 12 16 20 24 28 32
Slide 75: How Does SOL-1 Data Align with Real World Outcomes?
How Does SOL-1 Data Align with Real World Outcomes? SOL-1 Clinical Trial SOL-1 Emulated Population Post Hoc Analysis IRIS Registry 10 10 79.0 +7.3 from Screening BCVA, +6.5 +6.6 +6.8 80.8 Mean (±SE) Change Mean Change from Screening BCVA, ETDRS Letters ETDRS Letters 5 5 70.9 68.4 0 0 Weeks -8 -4 0 4
Slide 76: How Does SOL-1 Data Align with Real World Outcomes?
How Does SOL-1 Data Align with Real World Outcomes? SOL-1 Clinical Trial SOL-1 Emulated Population Post Hoc Analysis IRIS Registry 10 10 79.0 +7.3 from Screening BCVA, +6.5 +6.6 +6.8 80.8 Mean (±SE) Change Mean Change from Screening BCVA, ETDRS Letters ETDRS Letters 5 5 70.9 68.4 0 0 Weeks -8 -4 0 4
Slide 77: How Does SOL-1 Data Align with Real World Outcomes?
How Does SOL-1 Data Align with Real World Outcomes? UK Dataset (2022 -2025) Age, mean = 78.9 years Screen VA, mean = 65 letters Baseline VA, mean = 78 letters Analysis of a UK real-world cohort (2022–2025): 182 nAMD eyes selected using key SOL-1 entry criteria ETDRS, Early Treatment Diabetic Retinop
Slide 78: Clinical Program Update
Clinical Program Update Arshad M. Khanani, MD, MA, FASRS 79
Slide 79: Timeline to OTX-TKI NDA Submission Package for nAMD
Timeline to OTX-TKI NDA Submission Package for nAMD Feb 2026 Positive SOL-1 Week 52 results announced on February 17, 2026 Early Alignment at Type C Meeting: May 2026 FDA agreed in writing on NDA submission with SOL-1 trial plus confirmatory evidence Pre-NDA meeting: Sept 2026 Aligned on format and
Slide 80: SOL: OTX-TKI Phase 3 Clinical Program in nAMD
SOL: OTX-TKI Phase 3 Clinical Program in nAMD Evaluating the efficacy, durability and safety of OTX-TKI in nAMD Registrational Trials Extension Study SOL-1 SOL-R SOL-X Phase 3 Phase 3 Superiority Trial Non-Inferiority Trial Open-Label Extension Durability of a single Repeat dosing Long-term safety a
Slide 81: SOL-R: Evaluation of OTX-TKI relative to aflibercept 2 mg and 8 mg
SOL-R: Evaluation of with OTX-TKI relative to aflibercept 2 mg and 8 mg Primary Safety Key Secondary Endpoint follow-up* Endpoint Week -24 -20 -16 -12 -8 -4 R 2:2:1 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 … 72 … 96 Evaluation OTX-TKI Visits (0.45 mg) SOL-R Aflibercept Q8W Q8W End of (2 mg) Year 2
Slide 82: SOL: OTX-TKI Phase 3 Clinical Program in nAMD
SOL: OTX-TKI Phase 3 Clinical Program in nAMD Evaluating the efficacy, durability and safety of OTX-TKI in nAMD Registrational Trials Extension Study SOL-1 SOL-R SOL-X Phase 3 Phase 3 Superiority Trial Non-Inferiority Trial Open-Label Extension Durability of a single Repeat dosing Long-term safety a
Slide 83: SOL-X: Unlocking Long-Term Value with OTX-TKI
SOL-X: Unlocking Long-Term Value with OTX-TKI Designed to evaluate safety and disease modifying potential of OTX-TKI Primary Trials SOL-X Initial Treatment Arms Three-year additional follow-up SOL-1 OTX-TKI Aflibercept (2 mg) Time (weeks) 0 4 12 24 36 48 60 72 84 96 108 120 132 144 SOL-R OTX-TKI Q24
Slide 84: HELIOS-3 Study Design
Diabetic Retinopathy Program HELIOS-3 Study Design Evaluation of only Q12M OTX-TKI regimen vs Sham Design Primary Endpoint (Week 56) Superiority study of OTX-TKI in ~620 subjects with Ordinal DRSS 2-step change status at Week 56 from baseline moderately-severe to severe NPDR without CI-DME (≥ 2 step
Slide 85: OTX-TKI Development Program Summary
OTX-TKI Development Program Summary nAMD Program DR Program OTX-TKI OTX-TKI demonstrated PHASE 3 PHASE 3 PHASE 3 superior maintenance of Superiority Trial Non-Inferiority Trial Superiority Trial vision* to aflibercept 2 mg at Week 36 in nAMD Durability of a single Repeat dosing Evaluating OTX-TKI in
Slide 86: Panel Discussion
Panel Discussion Moderated by Arshad M. Khanani, MD, MA, FASRS 87
Slide 87: Faculty
Faculty Prof. Arshad M. Khanani Prof. Adnan Tufail Prof. Dilsher S. Dhoot Dr. Patricio G. Schlottmann MD, MA, FASRS MBBS, MD, FRCOphth MD, FASRS MD Managing Partner, Director of Clinical Consultant Ophthalmic Surgeon at Adjunct Clinical Assistant Professor at Director of Research, Research, Director
Slide 88: Redefining the Management of nAMD
Redefining the Management of nAMD The Impact of Durability and Sustained Disease Control October 2, 2026 Euretina | Vienna, Austria