Ocular Therapeutix, Inc. · NASDAQ:OCUL
Ocular Therapeutix — Investor Day (June 2026)
146 slides from Ocular Therapeutix's investor day presentation, Jun 17, 2026
Slide 1: Ocular Therapeutix 2026 Investor Day
Ocular Therapeutix 2026 Investor Day June 17, 2026
Slide 2: Forward Looking Statements and Disclaimers
Forward Looking Statements and Disclaimers Any statements in this presentation about future expectations, plans, and prospects for the Company, including the development of, regulatory status of, and regulatory submissions regarding the Company’s product candidates, including the timing, design, enr
Slide 3: Delivering Results to Better Serve Our Patients
Delivering Results to Better Serve Our Patients Pravin U. Dugel, MD Executive Chairman, President & CEO 3
Slide 4: We Continue to Execute as Promised
We Continue to Execute as Promised 11H H 22024 024 SOL-1 Bear trial will be Narrative impossible to enroll OCULAR Outstanding RESULTS enrollment rate 4
Slide 5: We Continue to Execute as Promised
We Continue to Execute as Promised 1H 2024 2H 2024 SOL-1 SOL-1 will have Bear trial will be poor retention Narrative impossible and off-protocol to enroll rescues Exceptional OCULAR Outstanding retention and RESULTS enrollment on-protocol rate rescue rate 5
Slide 6: We Continue to Execute as Promised
We Continue to Execute as Promised 1H 2024 2H 2024 2025 SOL-1 SOL-1 will have Modifications Bear trial will be poor retention to SOL-1 Narrative impossible and off-protocol will negate to enroll rescues the SPA Exceptional SPA OCULAR Outstanding retention and retained with RESULTS enrollment on-prot
Slide 7: We Continue to Execute as Promised
We Continue to Execute as Promised 1H 2024 2H 2024 2025 11Q 2026 Q 2 026 SOL-1 SOL-1 will have Modifications Bear SOL-1 trial will be poor retention to SOL-1 is going Narrative impossible and off-protocol will negate to fail to enroll rescues the SPA Exceptional SPA Superiority OCULAR Outstanding re
Slide 8: We Continue to Execute as Promised
We Continue to Execute as Promised TODAY FDA will not Bear accept a single Narrative trial for NDA submission Per Type C meeting OCULAR minutes, will RESULTS submit NDA on a single trial NDA, New Drug Application 8
Slide 9: We Continue to Execute as Promised
We Continue to Execute as Promised TODAY 2025 CDER Single Trial Approvals1 FDA will not Bear accept a single Narrative trial for NDA submission 28 Single Trial Approvals Per Type C meeting 61% 48% OCULAR minutes, will of Evaluable of Non-Orphan RESULTS submit NDA Approvals Approvals on a single tria
Slide 10: We Continue to Execute as Promised
We Continue to Execute as Promised TODAY TOMORROW FDA will not Bear accept a single Some new Narrative trial for NDA bear narrative submission Per Type C meeting Intend OCULAR minutes, will to continue RESULTS submit NDA proving the on a single bears wrong trial NDA, New Drug Application 10
Slide 11: What You Will Hear Today About AXPAXLI
What You Will Hear Today About AXPAXLI 11
Slide 12: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina Groundbreaking Profile Unmatched Durability with Sustained Disease Control in SOL-1 12
Slide 13: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina Groundbreaking Clear Path Profile to Market Unmatched Durability FDA Aligned on NDA with Sustained Disease Submission in 4Q 2026 Control in SOL-1 NDA, New Drug Application 13
Slide 14: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina Why haven’t we submitted the NDA yet? Goal: Fastest path to FDA Groundbreaking Clear Path approval with least Profile to Market regulatory risk Adhering to FDA guidance Unmatched Durability FDA Aligned on NDA by including >300 subjects with Sustained Diseas
Slide 15: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina Aligned w/ FDA on AXPAXLI NDA submission plan NDA to be based on SOL-1 Groundbreaking Clear Path efficacy & safety data plus Profile to Market interim SOL-R safety data Goal: Fastest path to FDA Unmatched Durability FDA Aligned on NDA approval with least
Slide 16: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina Groundbreaking Clear Path Focused on Profile to Market Commercial Success Unmatched Durability FDA Aligned on NDA Preparing to Meet with Sustained Disease Submission in 4Q 2026 Immediate Demand Control in SOL-1 (if Approved) NDA, New Drug Application 16
Slide 17: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina SOL-R efficacy data no longer part of NDA submission Will now evaluate superiority vs Focused on aflibercept (8mg) Q6M at Week 96 Commercial as a key secondary endpoint in Success SOL-R Preparing to Meet Goal: Best-in-disease product Immediate Demand (if
Slide 18: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina Groundbreaking Clear Path Focused on Profile to Market Commercial Success Unmatched Durability FDA Aligned on NDA Preparing to Meet with Sustained Disease Submission in 4Q 2026 Immediate Demand Control in SOL-1 (if Approved) NDA, New Drug Application 18
Slide 19: Renowned Retina Leaders Will Provide Unique Perspectives
Renowned Retina Leaders Will Provide Unique Perspectives Arshad M. Khanani, Lejla Vajzovic, MD, FASRS Darius M. Moshfeghi, MD MD, MA, FASRS Duke University Stanford University Sierra Eye Associates Durham, North Carolina Stanford, California Reno, Nevada 19
Slide 20: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 21: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 22: SOL-1 Data & AXPAXLI Clinical Program Review
SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH Chief Medical Officer 22
Slide 23: SOL-1: Superiority Trial Evaluated Head-to-Head Durability of AXPAXLI vs Aflibercept
SOL-1: Superiority Trial Evaluated the Head-to-Head Durability of AXPAXLI vs Aflibercept (2 mg) Primary Endpoint Week -8 -4 R 1:1 0 4 8 12 16 20 24 28 32 36 Head-To-Head Comparison AXPAXLI* (0.45 mg) Single injection of AXPAXLI vs single injection of Aflibercept Aflibercept (2 mg) (2 mg) Loading Ran
Slide 24: SOL-1: Superiority Trial Evaluated Head-to-Head Durability of AXPAXLI vs Aflibercept
SOL-1: Superiority Trial Evaluated the Head-to-Head Durability of AXPAXLI vs Aflibercept (2 mg) Key Primary Secondary Endpoint Endpoint Week -8 -4 R 1:1 0 4 8 12 16 20 24 28 32 36 40 44 48 52 AXPAXLI* (0.45 mg) Aflibercept (2 mg) Loading Randomization Monthly Visits (Day 1) AXPAXLI (0.45 mg) Afliber
Slide 25: SOL-1: Superiority Trial Evaluated Head-to-Head Durability of AXPAXLI vs Aflibercept
SOL-1: Superiority Trial Evaluated the Head-to-Head Durability of AXPAXLI vs Aflibercept (2 mg) Key Masked Primary Secondary Safety Endpoint Endpoint Follow-up** Week -8 -4 R 1:1 0 4 8 12 16 20 24 28 32 36 40 44 48 52 … 76 … 104 AXPAXLI* (0.45 mg) End of year 2 Aflibercept (2 mg) Loading Randomizati
Slide 26: SOL-1 Results Highlight AXPAXLI's Groundbreaking Product Profile
SOL-1 Results Highlight AXPAXLI’s Groundbreaking Product Profile First Successful Superiority Trial of Novel Agent vs Approved anti-VEGF Sustained Unmatched Disease Well-Tolerated Durability Control VEGF, vascular endothelial growth factor 26
Slide 27: AXPAXLI Successfully Met Superiority Primary Endpoint
AXPAXLI Successfully Met Superiority Primary Endpoint Vision Maintenance* with a Single Injection of AXPAXLI at Week 36 P = 0.0006 100% Clear benefit† 18.3% with AXPAXLI vs 74.1% Observed Aflibercept 2 mg Proportion of Subjects difference Maintaining Vision, % 75% 55.8% 50% Exceptionally high 0.0006
Slide 28: AXPAXLI Demonstrated Strong Potential for Annual Dosing
AXPAXLI Demonstrated Strong Potential for Annual Dosing Month 9 Results ~3 of 4 AXPAXLI subjects maintained vision* with a single injection 74.1% P = 0.0006 *Maintained vision defined as <15 ETDRS letters of loss. ETDRS, early treatment diabetic retinopathy study 28
Slide 29: AXPAXLI Demonstrated Strong Potential for Annual Dosing
AXPAXLI Demonstrated Strong Potential for Annual Dosing Month 9 Results Month 12 Results ~3 of 4 AXPAXLI subjects ~2 of 3 AXPAXLI subjects maintained vision* with a maintained vision* with a single injection single injection 74.1% 65.9% P = 0.0006 P < 0.0001 *Maintained vision defined as <15 ETDRS l
Slide 30: AXPAXLI Demonstrated Strong Potential for Annual Dosing
AXPAXLI Demonstrated Strong Potential for Annual Dosing Month 9 Results Month 12 Results Continued Response ~3 of 4 AXPAXLI subjects ~2 of 3 AXPAXLI subjects Of subjects that maintained maintained vision* with a maintained vision* with a vision at Month 9, ~90% single injection single injection main
Slide 31: Significantly Lower Risk of Rescue During Year with AXPAXLI
Significantly Lower Risk of Rescue During Year 1 with AXPAXLI 1.0 P = 0.0005* Time to First 0.9 Rescue Treatment Probability of Not Having Rescue Injection 0.8 0.7 0.6 0.5 0.4 0.3 0.2 0.1 0.0 AXPAXLI 0.45 mg (n=170) 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 60 Aflibercept 2 mg (n=172) Time from Base
Slide 32: Significantly Lower Risk of Rescue During Year with AXPAXLI
Significantly Lower Risk of Rescue During Year 1 with AXPAXLI 1.0 P = 0.0005* Time to First 0.9 Rescue Treatment Probability of Not Having Rescue Injection 0.8 0.7 ~6 MONTHS ~6 Month Difference 0.6 0.5 AXPAXLI Week 52 rescue rate reflects ~6-month 0.4 advantage over aflibercept 0.3 No approved wet A
Slide 33: SOL-1 Results Highlight AXPAXLI's Groundbreaking Product Profile
SOL-1 Results Highlight AXPAXLI’s Groundbreaking Product Profile First Successful Superiority Trial of Novel Agent vs Approved anti-VEGF Sustained Unmatched Disease Well-Tolerated Durability Control VEGF, vascular endothelial growth factor 33
Slide 34: Sustained Fluid Control Generally Leads to Strong Vision Control
Sustained Fluid Control Generally Leads to Strong Vision Control Fluid Control Vision Control Anatomic deterioration often precedes visual loss & drives retreatment in clinical practice 34
Slide 35: Sustained Fluid Control Generally Leads to Strong Vision Control
Sustained Fluid Control Generally Leads to Strong Vision Control Fluid Control Vision Control Anatomic deterioration often precedes visual loss & drives retreatment in clinical practice 35
Slide 36: AXPAXLI Provided Sustained Disease Control Measured Via OCT
AXPAXLI Provided Sustained Disease Control Measured Via OCT Time to ≥ 75 µm Increase in CSFT 1.0 P < 0.0001* 0.9 What does it all mean? 0.8 75 µm CSFT gains often 0.7 used as re-treatment criteria ≥ 75 µm Increase in CSFT Probability of Not Having 0.6 in wet AMD trials 0.5 0.4 0.3 0.2 0.1 AXPAXLI 0.
Slide 37: AXPAXLI Provided Sustained Disease Control Measured Via OCT
AXPAXLI Provided Sustained Disease Control Measured Via OCT Time to ≥ 75 µm Increase in CSFT 1.0 P < 0.0001* 0.9 What does it all mean? 0.8 75 µm CSFT gains often 0.7 used as re-treatment criteria ≥ 75 µm Increase in CSFT Probability of Not Having 0.6 in wet AMD trials 0.5 ~5 MONTHS ~5-month delay i
Slide 38: At Even Tighter Anatomic Thresholds, AXPAXLI Demonstrates Sustained Disease Control
At Even Tighter Anatomic Thresholds – AXPAXLI Demonstrates Stellar Sustained Disease Control Time to ≥ 30 µm Increase in CSFT 1.0 P < 0.0001* 0.9 What does it all mean? 0.8 0.7 ~6-month difference in time ≥ 30 µm Increase in CSFT Probability of Not Having 0.6 to a 30 µm CSFT gain in 0.5 favor of AXP
Slide 39: Sustained Fluid Control Generally Leads to Strong Vision Control
Sustained Fluid Control Generally Leads to Strong Vision Control Fluid Control Vision Control Anatomic deterioration often precedes visual loss & drives retreatment in clinical practice 39
Slide 40: AXPAXLI Demonstrated Strong Vision Control With Fewer Rescues Than Aflibercept
AXPAXLI Demonstrated Strong Vision Control Up to 9 Months With Significantly Fewer Rescues Than Aflibercept (2 mg) Mean Change in BCVA from Screening in All Subjects 10 Screening , ETDRS Letters Mean (±SE) Change from 78.0 76.6 5 Baseline 80.8 79.2 0 Primary Weeks -8 -4 0 4 8 12 16 20 24 28 32 36 36
Slide 41: AXPAXLI Demonstrated Strong Vision Control With Fewer Rescues Than Aflibercept
AXPAXLI Demonstrated Strong Vision Control Up to 9 Months With Significantly Fewer Rescues Than Aflibercept (2 mg) Mean Change in BCVA from Screening in All Subjects 10 Rescue Free Rate Screening , ETDRS Letters Mean (±SE) Change from at Week 36 78.0 76.6 74.7% 56.4% Rescue Rescue 5 free free Baseli
Slide 42: SOL-1 Results Highlight AXPAXLI's Groundbreaking Product Profile
SOL-1 Results Highlight AXPAXLI’s Groundbreaking Product Profile First Successful Superiority Trial of Novel Agent vs Approved anti-VEGF Sustained Unmatched Disease Well-Tolerated Durability Control VEGF, vascular endothelial growth factor 42
Slide 43: No Treatment or Procedure Related Ocular SAEs
No Treatment or Procedure Related Ocular SAEs Ocular Events in the Study Eye Subjects with Ocular AEs AXPAXLI 0.45 mg Aflibercept 2 mg Through Week 52, n (%) n = 170 n = 172 Ocular AEs in the study eye ≥ 1 AE 90 (52.9) 58 (33.7) ≥ 1 SAE* 1 (0.6) 0 ≥ 1 treatment-related AE 15 (8.8) 1 (0.6) ≥ 1 treatm
Slide 44: AXPAXLI was Generally Well Tolerated
AXPAXLI was Generally Well Tolerated Ocular Adverse Events in the Study Eye Subjects with Ocular AEs (> 2%) AXPAXLI 0.45 mg Aflibercept 2 mg Through Week 52, n (%) Safety is Essential n = 170 n = 172 in Wet AMD Vitreous floaters 21 (12.4) 2 (1.2) Cataract 12 (7.1) 5 (2.9) Conjunctival hemorrhage 11
Slide 45: Year 2 Safety: DSMC Recommends Trial Continuation
Year 2 Safety: DSMC Recommends Trial Continuation THROUGH Week 52 FOLLOWING Week 52 AXPAXLI was generally well-tolerated SOL-1 remains masked in Year 2, evaluating Q6M dosing No cases of endophthalmitis, occlusive or non-occlusive retinal vasculitis Masked safety data being Detailed results presente
Slide 46: Single Injection of AXPAXLI Demonstrated Superiority to Aflibercept (2 mg) in SOL-1
Single Injection of AXPAXLI Demonstrated Superiority to a Single Injection of Aflibercept (2 mg) in SOL-1 Results Highlight AXPAXLI’s Groundbreaking Profile First Phase 3 trial to Up to 52 Week visual Well-tolerated demonstrate durability & anatomic stability safety profile out to >9 months On avera
Slide 47: What Value Does Each SOL Trial Bring?
What Value Does Each SOL Trial Bring? 47
Slide 48: SOL Program Potential: SOL-1
SOL Program Potential: Each Trial Designed to Support Unique Value Proposition SOL-1 Phase 3 Superiority Trial Demonstrates effectiveness in wet AMD for NDA submission Provides safety data in wet AMD for NDA submission (Y2, evaluating Q6M dosing) Supports Ex-US regulatory filings Commercially releva
Slide 49: SOL Program Potential: SOL-1 and SOL-R
SOL Program Potential: Each Trial Designed to Support Unique Value Proposition SOL-1 SOL-R Phase 3 Superiority Trial Phase 3 Non-Inferiority Trial Demonstrates effectiveness in Provides safety data in wet AMD for NDA submission wet AMD for NDA submission Provides safety data in Supports Ex-US wet AM
Slide 50: SOL Program Potential: SOL-1, SOL-R, SOL-X
SOL Program Potential: Each Trial Designed to Support Unique Value Proposition SOL-1 SOL-R SOL-X Phase 3 Superiority Trial Phase 3 Non-Inferiority Trial Open-Label Extension Long-term safety and Demonstrates effectiveness in Provides safety data in evaluation of protective impact wet AMD for NDA sub
Slide 51: How Does SOL-1 Success Impact the HELIOS DR Program?
How Does SOL-1 Success Impact the HELIOS DR Program? 51
Slide 52: Streamlining Diabetic Retinopathy Program
Streamlining Diabetic Retinopathy Program 2025 Investor Day Sep. 30, 2025 Announced HELIOS-2* & HELIOS-3 DR Registrational Trials Evaluations of Q12M and Q6M AXPAXLI Regimens Superiority Trials Target Broad DR Label, Including DME Novel Primary Endpoint: Ordinal DRSS 2-Step Change Status Alignment o
Slide 53: Streamlining Diabetic Retinopathy Program
Streamlining Diabetic Retinopathy Program 2025 Investor Day Today Sep. 30, 2025 Announced HELIOS-2* & Prioritizing One DR HELIOS-3 DR Registrational Trials Registrational Trial (HELIOS-3) Evaluations of Q12M and Q6M Evaluation of only Q12M AXPAXLI Regimens AXPAXLI Regimen vs Sham Superiority Trials
Slide 54: Updated HELIOS-3 Study Design
Updated HELIOS-3 Study Design* HELIOS-3 Superiority Study Design Primary Endpoint (Week 56) Study of AXPAXLI in ~620 Ordinal DRSS 2-step change status at Week 56 from baseline subjects with moderately-severe (≥ 2 step improvement, ≥ 2-step worsening, < 2-step change in either direction) to severe NP
Slide 55: Rationale for Changes to HELIOS Program
HELIOS Program Update Rationale for Changes to HELIOS Program 1 2 3 4 Strong SOL-1 Demonstrated Q12M preferred HELIOS-3 results plus AXPAXLI regimen in DR supports global HELIOS-1 data durability up to 12 regulatory months in SOL-1 objectives Q12M, every 12 months; DR, diabetic retinopathy 55
Slide 56: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 57: U.S. Regulatory Strategy in Wet AMD
U.S. Regulatory Strategy in Wet AMD Peter K. Kaiser, MD Chief Development Officer 57
Slide 58: FDA Substantial Evidence of Effectiveness Requirement Likely Met by SOL-1
FDA “Substantial Evidence of Effectiveness” Requirement Likely Met by SOL-1 FDA Pathway Exists SOL-1 Trial Confirmatory Evidence FDA Modernization Act Adequate and well-controlled Mechanistic Evidence of 1997 Pharmacodynamic/ Pharmacokinetic Evidence SPA alignment FDA Guidance Animal Models of 1998
Slide 59: Substantial Evidence of Effectiveness with a Single Trial is Well-Established
Substantial Evidence of Effectiveness with a Single Trial is Well-Established Precedent is Well-Established Section 115 of the FDA Modernization Act of 1997 amended and clarified FD&C Act § 505(d) to define “substantial evidence” and explicitly allow one adequate and well-controlled trial plus confi
Slide 60: Approvals based on a Single Trial are Common and Increasingly Frequent
Approvals based on a Single Trial are Common and Increasingly Frequent Percent of All CDER NDAs Percent of Non-Orphan CDER NDAs Approved with a Single Trial1 Approved with a Single Trial1 100% 100% 66% 61% 50% 50% 42% 48% 0% 0% 2024 2025 2024 2025 Majority of recent CDER approvals Over 40% of recent
Slide 61: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data Code of Federal Regulation Adequate & Well-Controlled Trial 21 CFR § Part 314 defined what qualifies as an “adequate and well-controlled” study Protocol Aligned with FDA Evidence from clinical trials that are Pos
Slide 62: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data Code of Federal Regulation Adequate & Well-Controlled Trial 21 CFR § Part 314 defined what qualifies as an “adequate and well-controlled” study Protocol Aligned with FDA Evidence from clinical trials that are Pos
Slide 63: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data Adequate & Well-Controlled Trial A SPA means the FDA has agreed that the study Protocol Aligned with FDA design, endpoints, study size, and analysis plan are adequate to address the Post-Data scientific and regul
Slide 64: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data Adequate & Well-Controlled Trial SOL-1 conducted under SPA Protocol Aligned with FDA Only active wet AMD trial conducted under SPA Post-Data “The special protocol assessment is an official agreement between a spo
Slide 65: FDA Considers p values ≤ 0.001 Potentially Supportive
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data Adequate & Well-Controlled Trial Protocol Aligned with FDA Post-Data FDA Division of Ophthalmology considers p values ≤ 0.001 to be potentially supportive Superiority Primary Endpoint Met for single trial approva
Slide 66: FDA Considers p values ≤ 0.001 Potentially Supportive
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data Adequate & Well-Controlled Trial Protocol Aligned with FDA Post-Data FDA Division of Ophthalmology considers p values ≤ 0.001 to be potentially supportive Superiority Primary Endpoint Met for single trial approva
Slide 67: FDA Considers p values ≤ 0.001 Potentially Supportive
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data Adequate & Well-Controlled Trial Protocol Aligned with FDA Post-Data FDA Division of Ophthalmology considers p values ≤ 0.001 to be potentially supportive Superiority Primary Endpoint Met for single trial approva
Slide 68: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data • First trial to show superiority in preventing vision loss Adequate & Well-Controlled Trial against an active anti-VEGF control, aflibercept 2mg Protocol Aligned with FDA • 69% of AXPAXLI Subjects Rescue-Free at
Slide 69: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data • FDA notes one trial may establish effectiveness, but FDA still requires adequate safety exposure Adequate & Well-Controlled Trial • The 2023 nAMD Guidance defines a minimum of 300 patients with at least 9 month
Slide 70: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data • FDA notes one trial may establish effectiveness, but FDA still requires adequate safety exposure Adequate & Well-Controlled Trial • The 2023 nAMD Guidance defines a minimum of 300 patients with at least 9 month
Slide 71: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data 2023 FDA Guidance1 defined “confirmatory evidence” Adequate & Well-Controlled Trial 1. Clinical Evidence from a Related Indication 2. Mechanistic or Pharmacodynamic Evidence Protocol Aligned with FDA 3. Evidence
Slide 72: Meeting The Evidentiary Standard with a Single Pivotal Trial
Meeting The Evidentiary Standard for Effectiveness with a Single Pivotal Trial Pre-Data 2023 FDA Guidance1 defined “confirmatory evidence” Adequate & Well-Controlled Trial 1. Clinical Evidence from a Related Indication 2. Mechanistic or Pharmacodynamic Evidence Protocol Aligned with FDA 3. Evidence
Slide 73: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI VEGF-A is the key Mechanistic Evidence driver of wet AMD1 Class Consistency Vabysmo FDA review Pharmacodynamic Evidence “VEGF-A has been shown to cau
Slide 74: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI VEGF-A is the key Axitinib is a potent Mechanistic Evidence driver of wet AMD1 VEGFR inhibitor2 Class Consistency Inlyta Vabysmo FDA review FDA revie
Slide 75: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI Mechanistic Evidence FDA recognizes evidence from other members of the same pharmacologic class Class Consistency Observed effect is consistent with
Slide 76: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI Highly selective for all VEGF and PDGF receptors, minimal off target inhibition Mechanistic Evidence Physiologic Levels for Testing Axitinib intraocu
Slide 77: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI Axitinib inhibits VEGF & PDGF mediated angiogenic sprouts in human retinal microvascular endothelial cells and pericytes Mechanistic Evidence Class C
Slide 78: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI AXPAXLI Demonstrated Leakage Suppression in Rabbit VEGF Challenge model Mechanistic Evidence Class Consistency • Rabbit VEGF-challenge model a well-e
Slide 79: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI Natural History Evidence1 Mechanistic Evidence VEGF inhibition in wet AMD Class Consistency maintains vision Pharmacodynamic Evidence Untreated wet A
Slide 80: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD Confirmatory Evidence for AXPAXLI Real World Evidence of anti-VEGF Treatment Mechanistic Evidence • 295,561 eligible eyes • 184,258 actively treated Class Consistency ~SOL-1 Baseline
Slide 81: Confirmatory Evidence Supports Substantial Evidence of Effectiveness
Confirmatory Evidence Supports Substantial Evidence of Effectiveness for the Use of AXPAXLI in the Treatment of nAMD SOL-1 Emulated Population Confirmatory IRIS Registry Evidence for AXPAXLI 10 79.0 Mechanistic Evidence +7.3 +6.8 Mean Change from Screening , ETDRS Letters 5 Class Consistency Subject
Slide 82: What's Going into the AXPAXLI NDA Package, per Type C Meeting Minutes
What’s Going into the AXPAXLI NDA Package, per Type C Meeting Minutes AXPAXLI NDA Package AXPAXLI Efficacy for NDA Efficacy Data from SOL-1 Week 52 data Efficacy Week 52 Data None No SOL-R Efficacy Data included in the NDA Safety Week 52 Data Interim Analysis 120 Day Week 104 Data None Safety Update
Slide 83: What's Going into the AXPAXLI NDA Package, per Type C Meeting Minutes
What’s Going into the AXPAXLI NDA Package, per Type C Meeting Minutes AXPAXLI NDA Package AXPAXLI Safety for NDA Aligned with FDA to conduct Efficacy Week 52 Data None unscheduled interim safety review of SOL-R Will incur 0.0001 alpha penalty Safety Week 52 Data Interim Analysis for unscheduled anal
Slide 84: What's Going into the AXPAXLI NDA Package, per Type C Meeting Minutes
What’s Going into the AXPAXLI NDA Package, per Type C Meeting Minutes AXPAXLI NDA Package 120 Day Safety Update Efficacy Week 52 Data None Year 2 Safety from SOL-1 for repeat dosing Safety Week 52 Data Interim Analysis No additional data that would trigger a major amendment will 120 Day be submitted
Slide 85: Timeline to AXPAXLI NDA Submission Package
Timeline to AXPAXLI NDA Submission Package 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: 3Q 2026 To align on format and content of
Slide 86: Next Steps: AXPAXLI NDA Submission Leveraging the 505(b)(2) Pathway
Next Steps: AXPAXLI NDA Submission Leveraging the 505(b)(2) Pathway 505(b)(2) Pathway • Hybrid new drug application for approval of drugs that are similar to, but not identical, to previously approved drugs • Allows reliance on established safety and efficacy • Accelerates approval for new formulati
Slide 87: The 505(b)(2) Pathway vs 505(b)(1) Pathway
The 505(b)(2) Pathway vs 505(b)(1) Pathway 505(b)(2) 505(b)(1) Hybrid NDA relies partially on Full NDA relies entirely on data from previously preclinical and clinical data approved drug generated by the sponsor Goldstein, Beth, and Science Analyst. OVERVIEW of the 505(B)(2) REGULATORY PATHWAY for N
Slide 88: The 505(b)(2) Pathway Provides Up to 60 Days Advantage over 505(b)(1)
The 505(b)(2) Pathway Provides Up to 60 Days Advantage over 505(b)(1) 505(b)(2) 505(b)(1) Hybrid NDA relies partially on Full NDA relies entirely on data from previously preclinical and clinical data approved drug generated by the sponsor Up to 60 days Using the 505(b)(2) pathway compared to 505(b)(
Slide 89: AXPAXLI NDA Package Designed to Meet FDA Effectiveness and Safety Requirements
AXPAXLI NDA Package Designed to Meet FDA “Substantial Evidence of Effectiveness” and Safety Requirements Confirmatory Highly Positive SOL-1 Trial Aligned with FDA through SPA Safety Evidence Positive, Highly Clinical Sufficient Broad Adequate, Well- Stat Sig Coherence & Safety Confirmatory Controlle
Slide 90: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 91: Discussion: AXPAXLI NDA Submission Strategy
Discussion: AXPAXLI NDA Submission Strategy Peter K. Kaiser, MD Arthur Ciociola, PhD Chief Regulatory and Chief Development Officer Quality Officer 91
Slide 92: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 93: Preparing for a Successful Launch
Preparing for a Successful Launch David Robinson Global Chief Commercial Officer 93
Slide 94: Preparing For An Accelerated Launch of AXPAXLI
Preparing For An Accelerated Launch of AXPAXLI Following successful Type C meeting with FDA, NDA submission planned for 4Q 2026 Preparing for launch in 2027, if approved NDA, New Drug Application 94
Slide 95: AXPAXLI Potential
AXPAXLI Potential 95
Slide 96: Current Anti-VEGF Landscape
Current Anti-VEGF Landscape Global Branded Anti-VEGF Revenue1 Strong growth despite shortfalls $15B Vabysmo Eylea Globally, the branded anti-VEGF market has grown to ~$15B Lucentis Treatment is currently defined by short acting aVEGF therapies • Lucentis – 30 days2 • EYLEA - 44 days3 (+ 2 weeks) • V
Slide 97: Durability of AXPAXLI is Unmatched Relative to Current Agents
Durability of AXPAXLI is Unmatched Relative to Current Agents Potential for AXPAXLI Label to be 3X more Durable in Year 1 Lucentis AXPAXLI Eylea Demonstrated potential for up to annual dosing in SOL-1: Vabysmo Incremental durability vs 1st gen 69% Rescue-Free at Year 1 Eylea HD Incremental durabilit
Slide 98: AXPAXLI Potential to Significantly Improve Long-Term Treatment Dynamics
AXPAXLI Potential to Significantly Improve Long-Term Treatment Dynamics 100% 90% 30% 80% 43% 52% 70% 60% 65% 70% 14% 75% 76% 60% 79% 50% 100% 16% 40% 17% aVEGF Patients That 30% 16% Could Be Candidates 56% 16% 15% for AXPAXLI 20% 41% 14% 15% 31% 24% 15% 10% 19% 15% 11% 9% 6% 0% Initiation Year 1 Yea
Slide 99: AXPAXLI Potential to Significantly Improve Long-Term Treatment Dynamics
AXPAXLI Potential to Significantly Improve Long-Term Treatment Dynamics 100% 90% 30% 80% 43% 44% discontinue or are 52% non-adherent at year 1 70% 60% 65% 70% 14% 75% 76% 60% 79% 50% 100% 16% 40% 17% aVEGF Patients That 30% 16% Could Be Candidates 56% 16% 15% for AXPAXLI 20% 41% 14% 15% 31% 24% 15%
Slide 100: AXPAXLI Anticipated Use By Patient Segment
AXPAXLI Anticipated Use By Patient Segment Projected Dynamics: Early-Launch Phase Projected Mature Market Dynamics Greatest near-term Market expansion & opportunity post- increase in Tx naïve launch is in aVEGF starts expected over conversions time with improved adherence & long-term outcomes Illust
Slide 101: Positive Feedback
AXPAXLI Potential TIX redefined” 401
Slide 102: Key Insights: Retina Specialists
Key Insights: Retina Specialists Rapid Broad Seamless Practice Adoption Utilization Integration >90% of retina specialists Improved disease control Pre-filled intravitreal injection would adopt AXPAXLI and predictable 6+ month facilitates smooth practice within 1st year of launch1,2 durability drive
Slide 103: Retina Specialist Feedback Post-SOL-1
Retina Specialist Feedback Post-SOL-1 Following SOL-1 data, we have been very busy gathering feedback from the Retina Specialist community ~80% Of Retina Specialists surveyed1 were likely or extremely likely to utilize AXPAXLI based on SOL-1 Data Alone 1.Ocular Therapeutix Market Research (n=53 Reti
Slide 104: Early Payer Engagement is Validating the Unmet Need
Early Payer Engagement is Validating the Unmet Need for Durability, Adherence and Long-Term Visual Outcomes Experienced payer team early engagements have covered: 100% 100% of Tier 1 MEDICARE ADVANTAGE Lives of Tier 1 COMMERCIAL Lives 104
Slide 105: Payer Insights Indicate Strong Potential for Premium Pricing
Payer Insights Indicate Strong Potential for Premium Pricing Superiority Label Payers acknowledge that a label with superior Superiority in durability would get our durability potentially transforms the market and attention relative to may command premium pricing Total Cost of Care Adherence and LT
Slide 106: Launch Preparation
AXPAXLI Potential eteeeegee OCULAR =. *, experience see THERAPEUTIX redefined” 106
Slide 107: Current Treatment Challenges Represent a Clear Market Opportunity
Current Treatment Challenges Represent a Clear Market Opportunity No Superiority Label Market Dynamics Approvals based on non- Biosimilar entrants inferiority trial design Minimal efficacy or Increased competitive durability differentiation pressure to rebate/discount Payers cite lack of Focus on fi
Slide 108: Current Market Dynamics Put Ocular In A Position to Win
Current Market Dynamics Put Ocular In A Position to Win 1.8M wet AMD Patients in the U.S. Alone Concentrated Call Point High Treatment Initiation Rate High Switch Rate 2.5 – 3K Retina 71% initiate within 1st Patients often request Specialists in U.S. month of diagnosis2 most durable product3 ~600 HC
Slide 109: Our Team is Ready
Our Team is Ready Powered by Existing Field Expertise World Class Retina Leadership Existing National Coverage Total Team of 65 ~50-person Field Team ~15-person Reimbursement Team David Robinson Steve Meyers Namrata Saroj Global CCO CCO CBO Nicole Harris Kelley Allison Andrea Gibson John France SVP,
Slide 110: Key Launch Preparation Activities - Ongoing
Key Launch Preparation Activities - Ongoing Commercial Launch Organizational Strategy Infrastructure Enablement Key Account Segmentation Talent Planning and and Prioritization Patient Support Organization Hub Buildout Cross-functional Launch Scenario Planning Operating Model Data and Analytics Distr
Slide 111: Path to Commercial Readiness
Path to Commercial Readiness LAUNCH T-18 Months T-15 Months T-12 Months T-9 Months T-6 Months T-3 Months Hire Field Train Field Deploy Field Marketing Team Marketing Team Marketing Team Prepare Our Team Hire Field Market Train Field Market Access Deploy Field Market Access Team Access Team Team Cond
Slide 112: AXPAXLI Potential to Redefine the Retina Market
AXPAXLI Potential to Redefine the Retina Market 1 Large Market Size and a Global Opportunity 2 Redefines Retina Treatment Dosing 3 Superiority Label Positions AXPAXLI in a Class of One Superiority 4 Economic Predictability for Payers, Physicians & Patients 112
Slide 113: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 114: SOL-R: Defining Non-Inferiority Success
SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD Chief Scientific Officer 114
Slide 115: FDA Alignment, Careful Patient Selection, Interpreting Outcomes
FDA Careful Interpreting Alignment Patient Outcomes Selection 115
Slide 116: FDA Alignment
SieL-R Careful Patient Selection Interpreting Outcomes
Slide 117: SOL-R Design: Updates & Considerations
SOL-R Design: Updates & Considerations 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 AXPAXLI Visits (0.45 mg) SOL-R Aflibercept Q8W Q8W End of (2 mg) Year 2 Loading Aflibercept (8 mg) Random
Slide 118: SOL-R Design: Updates & Considerations
SOL-R Design: Updates & Considerations 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 AXPAXLI Visits (0.45 mg) SOL-R Aflibercept Q8W Q8W End of (2 mg) Year 2 Loading Aflibercept (8 mg) Random
Slide 119: SOL-R Design: Updates & Considerations
SOL-R Design: Updates & Considerations 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 AXPAXLI Visits (0.45 mg) SOL-R Aflibercept Q8W Q8W End of (2 mg) Year 2 Loading Aflibercept (8 mg) Random
Slide 120: SOL-R Design: Updates & Considerations
SOL-R Design: Updates & Considerations 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 AXPAXLI Visits (0.45 mg) SOL-R Aflibercept Q8W Q8W End of (2 mg) Year 2 Loading Aflibercept (8 mg) Random
Slide 121: Careful Patient Selection
Careful Patient Selection 121
Slide 122: Patients with Early Persistent Fluid Can Disrupt Non-Inferiority Trials
Patients with Early Persistent Fluid Can Disrupt Non-Inferiority Trials Vision in Patients Vision in Patients with Early Persistent Fluid without Early Persistent Fluid Delta = 0.9 Delta = 4.2 Screening to exclude subjects with early persistent fluid may be critical for trial success “Early Persiste
Slide 123: Learnings from Kodiak's DAZZLE Trial in Wet AMD
Learnings from Kodiak’s DAZZLE Trial in Wet AMD DAZZLE Trial Failed To Meet Primary Endpoint1 Kodiak’s Original Zenkuda/KSI-301 Formulation* Commentary1 “Allowing treatment with KSI-301 no more often than every 12 weeks after the loading phase turned out to be insufficient for some patients” *Kodiak
Slide 124: SOL-R Population Carefully Selected
SOL-R Population Carefully Selected Primary 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 Evaluation AXPAXLI Visits SOL-R Aflibercept (2 mg) Loading Aflibercept (8 mg) Randomization Monthly Visits (Day 1) Two Evaluation Visits SOL-R Trial Design CSFT ≤350 μm a
Slide 125: Why do we insist on monotherapy after randomization?
Why do we insist on monotherapy after randomization? 125
Slide 126: Non-Inferiority Designs: Active Arm Dosing
Non-Inferiority Designs: Active Arm Dosing* Non-Inferiority Trials for 1st & 2nd Generation anti-VEGFs Randomization Only Investigational product used in active arm in post-randomization phase Eylea, Beovu, Vabysmo, KSI-301 & Eylea HD Investigational Investigational + *Active arm dosing is abbreviat
Slide 127: Non-Inferiority Designs: Active Arm Dosing
Non-Inferiority Designs: Active Arm Dosing* Non-Inferiority Trials for 1st & 2nd Generation anti-VEGFs Randomization Only Investigational product used in active arm in post-randomization phase Eylea, Beovu, Vabysmo, KSI-301 & Eylea HD Non-Inferiority Trials for Extended Duration Products SUSVIMO Ran
Slide 128: Combination Regimen Trials Were Designed to Evaluate Superiority
Combination Regimen Trials Were Designed to Evaluate Superiority Superiority Trials Evaluating Combination Regimens Fovista Randomization Sozinibercept Investigational product given in combination with control arm product Investigational Investigational + Active arm dosing is abbreviated and reflect
Slide 129: Interpreting Outcomes
Interpreting Outcomes 129
Slide 130: Key Learning From Susvimo's FDA Review in Wet AMD
Key Learning From Susvimo’s FDA Review in Wet AMD Susvimo FDA Review “The Agency stated that the primary analysis should include consideration of patients who receive rescue therapy that may impact the efficacy outcomes” Susvimo Archway Trial permitted rescue with Intravitreal Ranibizumab 1. U.S. Fo
Slide 131: Key Considerations about Rescues in Non-Inferiority Wet AMD Trials
Key Considerations about Rescues in Non-Inferiority Wet AMD Trials Number of Timing Rescues of Rescues AMD, age-related macular degeneration 131
Slide 132: Key Considerations about Rescues in Non-Inferiority Wet AMD Trials
Key Considerations about Rescues in Non-Inferiority Wet AMD Trials Number of Timing Rescues of Rescues Subjects requiring frequent rescue therapy may be considered treatment failures1 (or combination therapy) FDA generally does not consider a single rescue as a treatment failure1 1. Weiss J, Boyd B,
Slide 133: Key Considerations about Rescues in Non-Inferiority Wet AMD Trials
Key Considerations about Rescues in Non-Inferiority Wet AMD Trials Number of Timing Rescues of Rescues Rescues in close proximity (<3 months) to the primary endpoint will fall under greater regulatory scrutiny1 EX: A W48 rescue for a trial with a W52/56 endpoint may be considered to have impacted th
Slide 134: Key Considerations about Rescues in Non-Inferiority Wet AMD Trials
Key Considerations about Rescues in Non-Inferiority Wet AMD Trials Number of Timing Rescues of Rescues Clear interpretation of a non-inferiority trial requires understanding of each AMD, age-related macular degeneration 134
Slide 135: Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents
Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents 1 Did the trial meet the NI margin? NI, non-inferiority 135
Slide 136: Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents
Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents 1 2 Did the trial meet the How many subjects NI margin? were rescued? NI, non-inferiority 136
Slide 137: Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents
Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents 1 2 3 How many subjects Did the trial meet the How many subjects were rescued more NI margin? were rescued? than once? NI, non-inferiority 137
Slide 138: Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents
Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents 1 2 3 How many subjects Did the trial meet the How many subjects were rescued more NI margin? were rescued? than once? 4 How many rescues near the primary endpoint? NI, non-inferiority 138
Slide 139: Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents
Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents 1 2 3 How many subjects Did the trial meet the How many subjects were rescued more NI margin? were rescued? than once? 4 5 How many rescues Was the trial protocol near the primary in alignment with FDA? endpoint? NI, non-
Slide 140: Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents
Key Questions of a Non-Inferiority Trial Evaluating Extended Durability Agents 1 2 3 How many subjects Did the trial meet the How many subjects were rescued more NI margin? were rescued? than once? 4 5 6 Does the use of other How many rescues Was the trial protocol agents constitute a near the prima
Slide 141: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 142: KOL Perspectives: AXPAXLI's Potential to Redefine Retina
KOL Perspectives: AXPAXLI’s Potential to Redefine Retina MODERATOR PANELISTS Jeffrey S. Heier, MD Arshad M. Khanani, Lejla Vajzovic, MD, FASRS Darius M. Moshfeghi, MD Chief Scientific Officer MD, MA, FASRS Duke University Stanford University Sierra Eye Associates Durham, North Carolina Stanford, Cal
Slide 143: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu
Slide 144: Summary & Takeaways
Summary & Takeaways Pravin U. Dugel, MD Executive Chairman, President & CEO 144
Slide 145: AXPAXLI: Positioned to Redefine Retina
AXPAXLI: Positioned to Redefine Retina Groundbreaking Clear Path Focused on Profile to Market Commercial Success Unmatched Durability FDA Aligned on NDA Preparing to Meet with Sustained Disease Submission in 4Q 2026 Immediate Demand Control in SOL-1 (if Approved) NDA, New Drug Application 145
Slide 146: Ocular Therapeutix 2026 Investor Day Agenda
Ocular Therapeutix 2026 Investor Day Agenda Intro & Overview Pravin U. Dugel, MD SOL-R: Defining Non-Inferiority Success Jeffrey S. Heier, MD SOL-1 Data & AXPAXLI Clinical Program Review Nadia K. Waheed, MD, MPH KOL Perspectives: Potential to Redefine Retina Moderator: Jeffrey S. Heier, MD U.S. Regu