Oculis Holding Ag · NASDAQ:OCS
Oculis — Investor Day (April 2025)
54 slides from Oculis's investor day presentation, Apr 15, 2025
Slide 1: Oculis Rethinking Ophthalmology R&D Day
Oculis Rethinking Ophthalmology R&D Day April 15, 2025
Slide 2: Safe Harbor Statements
Safe Harbor Statements Cautionary note on forward-looking statements These slides and the accompanying oral presentation contain forward-looking statements and information. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “i
Slide 3: Oculis Management
Oculis Management RA & A Riad Sherif, MD Sylvia Cheung Snehal Shah, PharmD Sharon Klier, MD Chief Executive Officer Chief Financial Officer President of R&D Chief Development Officer Oculis
Slide 4: World Renowned Ophthalmology and Neuro-Ophthalmology Experts
World Renowned Ophthalmology and Neuro-Ophthalmology Experts Va Retina Arshad M. Khanani, MD Clinical Professor, University of Nevada, and Director of Clinical Research, Sierra Eye Associates Baruch D. Kuppermann, MD, PhD Prof. and Chair, Dept of Ophthalmology Director, Gavin Herbert Eye Institute U
Slide 5: Stephen Hauser, MD, PhD Professor, Neurology
Stephen Hauser, MD, PhD Professor, Neurology Director, UCSF Weill Institute for Neuroscience A neuroimmunologist, Dr. Hauser’s research has advanced our understanding of the genetic basis, immune mechanisms, and treatment of multiple sclerosis (MS). Dr. Hauser has received numerous awards and honors
Slide 6: Agenda & Speakers
Agenda & Speakers Opening Remarks: 5 mins. Oculis Corporate Update: 5 mins. OCS-01 in DME: 30 mins. + DIAMOND Phase 3 trials update * Q&A Licaminlimab (OCS-02) in DED: 20 mins. + Rethinking DED treatment with precision medicine approach * Q&A Sylvia Cheung, Chief Financial Officer Riad Sherif, MD, C
Slide 7: Agenda & Speakers
Agenda & Speakers Privosegtor (OCS-05) — New Frontier in 5] Neuroprotection: 55 mins. + Phase 2 ACUITY expanded data analysis + Next steps in acute optic neuritis + Neuroprotection beyond acute optic neuritis * Q&A 6 Closing Remarks: 5 mins. Riad Sherif, MD, CEO Prof. Stephen Hauser, MD, PhD Prof. M
Slide 8: Corporate Update
Corporate Update
Slide 9: Strong Momentum in 2025
Strong Momentum in 2025 Privosegtor: Positive Ww OCS-01: Completed enrollment Phase 2 ACUITY results in Phase 3 DIAMOND program in in Acute Optic Neuritis DME with >800 patients Jan Feb | Mar Apr Cs Licaminlimab: Confirmed W s100 million raised precision medicine in an over-subscribed approach in DE
Slide 10: Major Innovations Addressing Substantial Unmet Medical Needs
3 Major Innovations Addressing Substantial Unmet Medical Needs Highly differentiated late-stage pipeline focused on multi-billion-dollar market opportunities OCS-01 OPTIREACH® Eye Drops in DME ~y Topical treatment for DME enabling early intervention and for patients with inadequate response to curre
Slide 11: Oculis Pipeline Development Strategic Evolution
Oculis Pipeline Development Strategic Evolution Explore Potential for Privosegtor in Expa n d multiple additional neuro- ophthalmology and Adva nce Privosegtor in neurology conditions neuro-ophthalmology OCS-01 would be the first ever and beyond topical treatment in DME, if approved * NAION Licaminl
Slide 12: OCS-01 in DME
OCS-01 in DME DIAMOND Phase 3 trials update Oculis
Slide 13: Positive and Consistent Results Across Previously Completed Studies
Positive and Consistent Results Across 4 Previously Completed Studies Four (4) clinical trials successfully completed showing positive outcomes Patient Case (Phase 2 DX211) OCS-01 showed consistent biological effect in CMT reduction and BCVA improvement? Age 55 w Treatment Group | OCS-01 DME Duratio
Slide 14: Key Takeaways From OCS-01 DIAMOND Phase 2/3 Stage
5 Key Takeaways From OCS-01 DIAMOND Phase 3 Stage 1 Consistent with previous trials with robust statistically significant improvement in vision and reduction in retinal edema vs. vehicle 4) 7.2 letter gain in BCVA vs. baseline at Week 6, increasing to 7.6 at Week 12 6 25.3% of patients gained 215 le
Slide 15: OCS-01 Phase 3 DIAMOND Program in DME
OCS-01 | Phase 3 DIAMOND Program in DME Completed enrollment in both Diamond 1 & 2 Phase 3 Studies Study Design Key Endpoints Study Population + Randomized, double-blind, placebo- + Primary endpoint: Change in BCVA + Age 18-85 years controlled study (registrational trials) ETDRS letter score at Week
Slide 16: Rapid Enrollment Completed for Both DIAMOND Phase 3 Trials
Rapid Enrollment Completed for Both DIAMOND Phase 3 Trials Strong execution supported by DIAMOND Committees with world-renowned experts 119 DIAMOND Phase 3 trials topline results Sites Activated (anticipated) Page 864 >800 Subjects Enrolled Ww Enrollment completed for both DIAMOND Phase 3 trials Ww
Slide 17: 2025 DIAMOND Team Focused on Execution and Oversight
2025 DIAMOND Team Focused on Execution and Oversight fot) Driving strong adherence to the protocol © Ensuring patient compliance and retention © Collaborating closely with highly experienced investigators and committees & Investigator’s perspective: Dr. David Boyer Oculis
Slide 18: DIAMOND Experts Committees
DIAMOND Experts Committees Worldwide renowned retina specialists Steering Committee Arshad M. Khanani, MD David Almeida, MD, PhD David S. Boyer, MD Saradha Chexal, MD Carl Danzig, MD Dilsher Dhoot, MD Frank Holz, MD Anat Loewenstein, MD Patricio Schlottmann, MD Ashish Sharma, MD Veeral Sheth, MD Mic
Slide 19: Licaminlimab in DED
Licaminlimab In DED Rethinking DED Treatment with a Genotype-Base Development for Personalized Medicine Oculis
Slide 20: Large Unsatisfied Market Creates Significant Opportunity for Personalized Medicine
20 Large Unsatisfied Market Creates Significant Opportunity for Personalized Medicine Only 13% experiencing lasting relief after 12 months with current treatments! 2024 Dry Eye Rx drug market in G7 countries? and U.S. Rx split? Driven by trial and error with significant unmet needs Others 13% 1.07 C
Slide 21: Licaminlimab: Three Positive DED Phase 2 Trials Completed
Licaminlimab: Three Positive DED Phase 2 Trials Completed Consistent positive results in signs and symptoms with potential for a genotype-based development to drive precision medicine Phase 2 Randomized Controlled Consistent positive results across studies and Studies in DED unique precision medicin
Slide 22: First Genotype-Based Development in DED Fully Aligned with FDA Guidance
22 Mas First Genotype-Based Development in DED Fully Aligned with FDA Guidance — Meeting Completed in Q1 25 Precision medicine strategy Registrational path forward Study design First genotype-based development in DED to drive precision medicine Primary endpoint in TNFR1 genotype positive patients; s
Slide 23: Licaminlimab First Genotype-based Development to Drive Precision Medicine in DED
Licaminlimab First Genotype-based Development to Drive Precision Medicine in DED Registration Program to Start in 2H 2025 Phase 2/3 Study Design Key endpoints Study Population + Randomized, multicenter, double-masked, | | * Primary endpoint: Global ocular discomfort + TNFR1 genotype: ~2/3 positive v
Slide 24: Privosegtor — Neuroprotection
Privosegtor — Neuroprotection Phase 2 ACUITY expanded data analysis Next steps in Acute Optic Neuritis Neuroprotection beyond Acute Optic Neuritis
Slide 25: Privosegtor (OCS-05) Key Takeaways
Privosegtor (OCS-05) Key Takeaways New class of drug potentially unlocking neuroprotection therapy era Privosegtor is a small molecule peptoid that penetrates Blood Brain and Retinal Barrier Pre-clinical data in various in vivo models validated preservation of neurons and axons Acute optic neuritis
Slide 26: Neuroprotection the New Frontier in Neuro-Ophthalmology and Beyond
Neuroprotection the New Frontier in Neuro-Ophthalmology and Beyond Privosegtor is a neuroprotective candidate activating neurotrophic signaling pathways supporting neuronal and axonal survival and preservation * Privosegtor is a small molecule peptoid that penetrates Blood Brain and Retinal Barrier
Slide 27: Acute Optic Neuritis a Predictive Model for Neuroprotection
Acute Optic Neuritis a Predictive Model for Neuroprotection Shares a similar pathophysiology triggered by acute RGC/Axon injuries Similar pathophysiological mechanisms across AON, MS relapses, and acute RGC & Axons insults « MS relapses: regardless of their anatomical location, they share fundamenta
Slide 28: Acute Optic Neuritis
Acute Optic Neuritis An acute inflammation of the optic nerve that can lead to permanent visual impairment Orphan indication with Acute inflammation of the optic nerve ~ 65k patients a year (US/EU)! impacting retinal ganglion cells y = Inflammation in the optic nerve = Anatomy of a retinal ganglion
Slide 29: Acute Optic Neuritis: Orphan Indication With No Approved Neuroprotective Therapy
Acute Optic Neuritis: Orphan Indication With No Approved Neuroprotective Therapy Current treatment landscape Visual Sequelae + Decreased contrast * Decreased visual fields * Decreased visual acuity * Uhthoff phenomenon Current High-dose corticosteroids Treatment to resolve acute inflammation Neuropr
Slide 30: Acute Optic Neuritis: OCT Imaging Biomarker Predicts Outcome
Acute Optic Neuritis: OCT Imaging Biomarker Predicts Outcome Change in GCIPL thickness in the first month predicts visual impairment by month 61 GCIPL and RNFL are measured Decrease of 2 4.5 ym in GCIPL predicts poor using OCT imaging technique LCVA and 27 um predicts poor VF and CVA RNFL . = —» Axo
Slide 31: Phase 2 ACUITY Trial in Acute Optic Neuritis
Phase 2 ACUITY Trial in Acute Optic Neuritis Proof-of-concept for neuroprotection Study Design Key endpoints Study Population Primary endpoint: Cardiac safety + Randomized, double-masked, placebo- + Patients diagnosed with a unilateral controlled study Secondary endpoints: acute optic neuritis with
Slide 32: Patient Demographics and Baseline Characteristics
Patient Demographics and Baseline Characteristics Privosegtor + steroid 3 mg/kg/day (N = 15) Placebo + steroid (N = 14) Age, mean (SD), years 33.7 (9.8) 32.7 (10.3) Female, n (%) 9 (60.0) 10 (71.4) GCIPL thickness, mean (SD), um 89.3 (8.3) 84.3 (13.8) RNFL thickness, mean (SD), ym 104.6 (13.1) 115.5
Slide 33: Primary Endpoint of Cardiac Safety Showed No difference
Primary Endpoint of Cardiac Safety Showed No difference in % of Patients that had a change in ECG Outside of Normal Percentage of subjects with shift from normal (baseline) to outside of normal value in any ECG parameters* from Visit 3 (after treatment) through Visit 4 Events observed in the Privose
Slide 34: Safety Summary
Safety Summary + NoAEs leading to drug withdrawal or study discontinuation + No drug-related serious adverse events (SAEs) + 2 Unrelated SAEs: + Hospitalization due to MS relapse (Privosegtor (OCS-05 + steroid) and due to my (placebo + steroid) Event, n (%) Privosegtor + steroid Placebo + 2 mg/kg/da
Slide 35: Relapses or Worsening of CNS Inflammatory Disorders
Relapses or Worsening of CNS Inflammatory Disorders Adverse events related to new relapses or worsening of CNS inflammatory disorders Event, n (%) Privosegtor + steroid Placebo + steroid 2 mg/kg/day 3 mg/kg/day 1g per day (N= 4) (N= 15) (N= 14) At least one new relapse of CNS 0/ 0) 0, inflammatory d
Slide 36: Patients in the Privosegtor 3mg/kg/day Arm Achieved Less GCIPL Thickness Decrease
36 Patients in the Privosegtor 3mg/kg/day Arm Achieved Less GCIPL Thickness Decrease GCIPL Thickness in the Affected Eye: MMRM, LS Mean Change From Baseline, mITT 5 *P = 0.049 —e-Placebo + steroid (N = 14) 0 ¢ -ePrivosegtor (OCS-05) 3mg/kg/day + steroid (N = 15) *P = 0.052 GCIPL Thickness Change, LS
Slide 37: Patients in the Privosegtor 3mg/kg/day Arm Achieved Less RNFL Thickness Decrease
Patients in the Privosegtor 3mg/kg/day Arm Achieved Less RNFL Thickness Decrease RNEL Thickness in the Affected Eye: MMRM, LS Mean Change from Baseline, mITT yum 04 _ -e-Placebo + steroid (N = 14) wW -e-Privosegtor (OCS-05) 3mg/kg/day + steroid (N = 15) = -10 5 oO o = -21.1 *P = 0.045 ” -20 4 * *P =
Slide 38: Patients in the Privosegtor 3mg/kg/day Arm Achieved Clinically Meaningful Improvement in Visual Function
Patients in the Privosegtor 3mg/kg/day Arm Achieved Clinically Meaningful Improvement in Visual Function 2.5% ETDRS LCVA in the Affected Eye: MMRM, LS Mean Change From Baseline, mITT +18 ETDRS Letters +15 ETDRS Letters “P= 0.012 Letters } *P=0.072 | 50 4 48.1 + 51.7 or 40.3 40 4 c 36.9 o = 30 5 314
Slide 39: Privosegtor Arm Showed a Robust LCVA Improvement Across all Subgroups
Privosegtor Arm Showed a Robust LCVA Improvement Across all Subgroups and Maintained through Month 6 LCVA letters subgroup analyses of Privosegtor 3mg + steroid vs placebo + steroid LS Mean LS Mean difference difference Subgroup LSM difference at month 3 (P-value*) LSM difference at month 6 (P-value
Slide 40: Patients in the Privosegtor Arm Achieved Lower Neurofilament Release
Patients in the Privosegtor Arm Achieved Lower Neurofilament Release, a Biological Sign of Less Neuronal and Axonal Death Neurofilaments are released into the CSF and blood as a result of axonal injury or neuronal death? Axonal damage L : 66 \ : Zz a 1 ; t time 7 q > vtVv3 M1 M3 M6 . \\ Blood Visit
Slide 41: ACUITY Phase 2 Topline Results Summary
ACUITY Phase 2 Topline Results Summary Privosegtor (OCS-05) achieved primary safety endpoint and key secondary endpoints showing functional vision improvement and neuroprotective anatomical & biological benefits on: Improvement in LCVA with 18 letters difference at mon Anatomy: GCIPL and RNFL with l
Slide 42: Acute Optic Neuritis — Next Steps
Acute Optic Neuritis — Next Steps FDA interaction planned in Q3 2025 following successful Phase 2 ACUITY trial 1. Meet with FDA to review ACUITY results and discuss full development program to support Acute Optic Neuritis registrational plan The current plan for a registrational trials: Primary endp
Slide 43: Privosegtor
Privosegtor Addressing unmet needs in MS: Neuroprotection for MS patients
Slide 44: Multiple Sclerosis
Multiple Sclerosis Most common CNS condition affecting young adults High prevalence of multiple sclerosis worldwide'? Large and growing market driven by immuno-modulators Existing therapies only slow disease progression while relapses continue to cause disability Worldwide 2.8m Characterized by rela
Slide 45: Relapsing-Remitting Multiple Sclerosis (RRMS)
45 Relapsing-Remitting Multiple Sclerosis (RRMS) Characterized by acute attacks (relapses) that may cause permanent disability BENS Estimated number of relapses in U.S. per year ~170K* RRMS represents ~85% of patients at initial diagnosis" Symptoms include loss of vision?, severe weakness or poor ba
Slide 46: Privosegtor Neuroprotective Effect Observed in Acute Optic Neuritis
Privosegtor Neuroprotective Effect Observed in Acute Optic Neuritis Could Be Translated into a Reduction of Relapse Associated Worsening 46 Disability * RAW: Relapse-Associated Relapsing Remitting Multiple Sclerosis Worsening ¢ Effective neuroprotection in Relapse relapses could translate to reduced
Slide 47: Privosegtor
Privosegtor Addressing unmet needs in NAION: Neuroprotection for patients to improve visual outcomes
Slide 48: Another Acute Optic Nerve Disorder: NAION
Another Acute Optic Nerve Disorder Non-arteritic Anterior Ischemic Optic Neuropathy No treatment approved, with severe vision loss in > 60% patients Orphan indication with US incidence of ~ 20k-30k' RGC, axons and optic nerve Permanent vision loss in atrophy caused by hypoperfusion? many patients An
Slide 49: NAION: Orphan Indication With No Approved Therapy
NAION: Orphan Indication With No Approved Therapy No medical or surgical treatment shown to improve prognosis of acute NAION other than risk factor modification’ Current There is no effective treatment for Treatments treating the disease or preventing it Neuroprotective treatment effect on retinal g
Slide 50: Privosegtor: Opening a New Era in Neuroprotection
Privosegtor: Opening a New Era in Neuroprotection with Acute treatments for AON, Relapses of MS and NAION Next steps to advance efficient development and reduce risk 1. Meet with FDA in 2H 2025 and Advance AON into registrational trials in 1H 2026 + pand into NAION and MS Relapse with Pre-IND intera
Slide 51: Conclusion
Conclusion
Slide 52: Oculis Pipeline Development Strategic Evolution
Oculis Pipeline Development Strategic Evolution Explore Potential for OCS-05 in Ex pa n d multiple additional neuro- ophthalmology and OCS-05 in neuro- neurology conditions ophthalmology and beyond Advance OCS-01 could be the first ever topical treatment in DME, if approved « NAION +$25B45 f Licamin
Slide 53: Advanced and Innovative Pipeline
Advanced and Innovative Pipeline Pre-clinical Phase 1 Phase 2 Phase 3 Next catalysts + DME TLR Q2 2026 DIABETIC MACULAR EDEMA + DME NDA submission 2H 2026 Ocs-01 OPTIREACH © * Ocular surgery NDA submission after DME * Initiate Ph2/3 as genotype-based development trial in 2H 2025 LICAMINLIMAB (Ocs-02
Slide 54: Oculis
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