Core Insights - Oculis Holding AG's neuroprotective candidate Privosegtor has received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for treating optic neuritis (ON), a serious condition often linked to multiple sclerosis [1][2] - This designation follows the Breakthrough Therapy designation granted by the U.S. FDA, indicating strong regulatory support for Privosegtor [2] - Privosegtor is positioned to be the first neuroprotective therapy for optic neuropathies, addressing a significant unmet medical need with a potential market opportunity of $7 billion in the U.S. alone [3][4] Regulatory Designations - The EMA's PRIME designation is awarded to a limited number of programs each year, aimed at expediting the development of promising medicines that can provide major therapeutic advantages [2][11] - Privosegtor has also received Orphan Drug status from both the EMA and FDA for ON, further enhancing its development prospects [2][8] Clinical Development - Positive results from the Phase 2 ACUITY trial demonstrated substantial improvements in low-contrast visual acuity and anatomical benefits, supporting the potential of Privosegtor as a neuroprotective treatment [4][7] - Oculis is advancing the PIONEER program, which includes three global registrational trials for ON and non-arteritic anterior ischemic optic neuropathy (NAION) [5][8] Market Opportunity - Optic neuritis affects up to 8 in 100,000 people globally, with an estimated annual incidence of over 30,000 in the U.S., highlighting the significant market potential for effective treatments [9] - The absence of current neuroprotective treatments for optic neuropathies underscores the urgency for novel therapies that can prevent vision loss [9][10] Company Overview - Oculis is a biopharmaceutical company focused on innovations in neuro-ophthalmology and ophthalmology, with a late-stage clinical pipeline that includes Privosegtor, OCS-01, and Licaminlimab [10] - The company is headquartered in Switzerland and operates in the U.S. and Iceland, led by an experienced management team [10]

Core Insights - Oculis Holding AG announced the presentation of Phase 2 data for Privosegtor at the NANOS 52nd Annual Meeting, highlighting its commitment to addressing unmet medical needs in neuro-ophthalmology [1][2]. Company Overview - Oculis is a global biopharmaceutical company focused on innovations in ophthalmology and neuro-ophthalmology, with a late-stage clinical pipeline that includes three core product candidates: Privosegtor, OCS-01, and Licaminlimab [10]. - Privosegtor is a novel peptoid small-molecule candidate that has received Breakthrough Therapy designation from the FDA for optic neuritis (ON) and is entering registrational trials for ON and non-arteritic anterior ischemic optic neuropathy (NAION) [6][10]. Clinical Trial Results - The ACUITY Phase 2 trial results demonstrated significant improvements in low contrast visual acuity and neuroprotective effects in patients with optic neuritis, indicating Privosegtor's potential as the first neuroprotective therapy for this condition [4][6]. - The trial showed anatomical preservation of the retina and improvements in visual function after acute episodes of optic neuritis, with consistent results observed in animal models [6]. Event Participation - Oculis served as a Platinum Sponsor at the NANOS 2026 Congress, emphasizing its role in advancing scientific progress in neuro-ophthalmology [2][5]. - The presentation at NANOS represents a significant milestone for Oculis and the scientific community, showcasing groundbreaking data from the ACUITY trial [5]. Expert Involvement - Professor Martin Zinkernagel, a recognized expert in ophthalmology, presented the trial results, underscoring the importance of the findings in the context of neuro-ophthalmic science [5][7].

Summary of Oculis Holding AG Conference Call Company Overview - **Company**: Oculis Holding AG - **Industry**: Biopharmaceuticals, specifically focusing on ophthalmology Key Products and Programs 1. **OCS-01 for Diabetic Macular Edema (DME)** - **Phase**: Currently in Phase 3 trials (DIAMOND-1 and DIAMOND-2) with data expected in the second half of Q2 [3][19] - **Unique Selling Proposition**: First and only topical product for retina, allowing early intervention for naïve patients and combination treatment for those unresponsive to anti-VEGF [3][5] - **Market Potential**: Addresses a larger patient pool than current treatments, with over 40% of DME patients not responding to existing anti-VEGF therapies [4][5] - **Efficacy Data**: Phase 2 results showed a mean change in Best Corrected Visual Acuity (BCVA) of 7.8 letters at week 6, comparable to anti-VEGF treatments [10][11] - **Pricing Strategy**: Targeting a price of around $10,000 per year, which is competitive compared to existing treatments like Ozurdex priced between $8,000-$16,000 [71][75] 2. **Dry Eye Program** - **Focus**: Utilizing a genetic biomarker (TNFR1) to identify responsive patients, aiming to treat 20% of the dry eye population [96][100] - **Clinical Efficiency**: Reduced patient count in trials (160 patients) due to targeted approach, leading to higher probability of success [97] - **Compliance Improvement**: Expected compliance rate of 70% compared to the current 10%-15% for existing treatments, leading to increased patient value [100][142] - **Pricing**: Expected to be competitive with existing products like MIEBO, which costs around $11,000 per year [128][137] 3. **Privosegtor for Neuroprotection** - **Indication**: Targeting neurodegenerative diseases, with a focus on optic neuritis [168] - **Phase 2 Data**: Showed significant improvement in vision (doubling function) and preservation of retinal ganglion cells [176][177] - **Biomarkers**: Neurofilament levels will be used as a regulatory endpoint, indicating axonal damage [178][189] - **Pricing**: Anticipated treatment cost between $100,000 and $400,000, with a likely average around $150,000 to $200,000 [205][211] Market Strategy and Commercialization - **Commercialization Approach**: Aiming for a straightforward launch due to the existing awareness of DME among healthcare providers [61][70] - **Sales Force**: Planning to deploy a field force of 50-60 full-time equivalents (FTEs) for effective market penetration [54][55] - **Market Education**: No need for extensive education on DME, as the condition is well-known among practitioners [63][70] Additional Insights - **Regulatory Engagement**: Positive discussions with the FDA regarding the use of genetic biomarkers for patient selection in clinical trials [96] - **Patient-Centric Approach**: Emphasis on reducing trial-and-error in treatment for dry eye patients, enhancing the overall patient experience [99][100] - **Future Outlook**: Anticipation of positive data from ongoing trials, with plans for rapid filing post-results [40][41] This summary encapsulates the key points discussed during the conference call, highlighting Oculis Holding AG's innovative approaches in the biopharmaceutical sector, particularly in ophthalmology.

Core Insights - Oculis Holding AG is advancing its position in neuro-ophthalmology and ophthalmology with a late-stage portfolio and significant market opportunities [2][6] - The company has received breakthrough therapy designation for its product Privosegtor, targeting optic neuritis, with a potential market opportunity exceeding $7 billion in the U.S. [2] - Oculis is set to present pivotal trial results for its product OCS-01 in diabetic macular edema (DME) in Q2 2026 [2] - The company is also developing Licaminlimab, a precision medicine approach for dry eye disease (DED) [2][6] Company Overview - Oculis is a global biopharmaceutical company focused on addressing unmet medical needs in neuro-ophthalmology and ophthalmology [6] - The company has a strong balance sheet and a robust pipeline, aiming to deliver six pivotal readouts with current funding [3] - Oculis is headquartered in Switzerland and operates in the U.S. and Iceland, led by an experienced management team [6] Upcoming Events - Oculis management will participate in the Leerink Global Healthcare Conference from March 8-11, with a presentation scheduled for March 10 [4] - The company will also engage in one-on-one meetings at the Leerink Partners Mountain Meeting from March 22-25 [5] - Additional one-on-one meetings are planned during the LifeSci Capital Biotech Forum on March 26 [5]

Core Insights - Oculis Holding AG has made significant clinical advancements in 2025, particularly in neuro-ophthalmology, with the FDA granting Breakthrough Therapy designation for Privosegtor in optic neuritis, leading to the launch of the PIONEER registrational program [2][4] - The company anticipates three major registrational milestones in 2026, including results from the DIAMOND-1 and DIAMOND-2 trials for OCS-01 in diabetic macular edema (DME) and the PREDICT-1 trial for Licaminlimab in dry eye disease [2][3] - Oculis has a strong financial position with cash and short-term investments of $268.7 million as of December 31, 2025, providing a cash runway into 2029 [3][5] Clinical Highlights - Privosegtor has shown promising Phase 2 ACUITY results, leading to its Breakthrough Therapy designation and a potential U.S. market opportunity exceeding $7 billion for optic neuropathies [3][4] - OCS-01 is nearing completion of Phase 3 DIAMOND trials, with over 800 patients enrolled and topline results expected in Q2 2026, targeting a DME market valued at approximately $3 billion [4][5] - Licaminlimab is positioned to transform treatment for dry eye disease with a precision medicine approach, with topline results from the PREDICT-1 trial expected in Q4 2026 [10] Financial Performance - As of December 31, 2025, Oculis reported a net loss of CHF 99.0 million ($119.1 million) for the year, an increase from CHF 85.8 million ($97.4 million) in 2024, primarily due to rising operating expenses [5][15] - Research and development expenses for Q4 2025 were CHF 13.3 million ($16.6 million), up from CHF 11.8 million ($13.4 million) in Q4 2024, reflecting advancements in clinical development [5][15] - General and administrative expenses also increased to CHF 7.8 million ($9.7 million) in Q4 2025 from CHF 5.5 million ($6.3 million) in the same period in 2024, driven by personnel and professional service costs [5][15]

Core Insights - Oculis Holding AG has appointed Katie Kazem as Chief Legal Officer, enhancing its legal, compliance, and corporate governance functions [1][2] - The company is advancing its product Privosegtor into the PIONEER program and is expecting topline results for OCS-01 eye drops in diabetic macular edema (DME) in Q2 [2] - Oculis has a differentiated late-stage clinical pipeline, including three core product candidates: Privosegtor, OCS-01, and Licaminlimab [4] Company Overview - Oculis is a global biopharmaceutical company focused on innovations in neuro-ophthalmology and ophthalmology [4] - The company is headquartered in Switzerland and has operations in the U.S. and Iceland [4] - Oculis is supported by leading international healthcare investors and led by an experienced management team [4] Product Pipeline - Privosegtor is a neuroprotective candidate in the PIONEER program aimed at treating optic neuropathies [4] - OCS-01 is in pivotal registration studies to become the first non-invasive topical treatment for DME [4] - Licaminlimab is a novel topical anti-TNFα in Phase 2, focusing on precision medicine for dry eye disease [4] Leadership and Expertise - Katie Kazem brings over 15 years of experience in corporate and securities law, particularly in life sciences [2][5] - She has a strong background in advising on IPOs, capital raising, M&A, and regulatory compliance [2][5] - Kazem has previously served as external legal counsel to Oculis since its NASDAQ listing in March 2023 [2]

Summary of Oculis Conference Call Company Overview - **Company**: Oculis - **Industry**: Biopharmaceuticals, specifically in ophthalmology and neuro-ophthalmology - **Stock Exchange**: Nasdaq-listed - **Financial Position**: Strong balance sheet with no debt, cash runway until 2029 excluding a facility loan of CHF 100 million ($125 million) [2][3] Key Products and Pipeline Ocular Franchise - **OCS-01**: - First non-invasive self-administered eye drop for diabetic macular edema (DME) - Phase 3 readout planned for Q2 2023 - Targets a market of 1.8 million diagnosed patients in the US, with only half currently treated [2][5][6] - Demonstrated a 7.6 letters gain in best-corrected visual acuity (BCVA) at week 12 in trials, with 27% of patients gaining more than 15 letters [10] - Expected submission for approval in Q4 2023, with potential approval in 2027 [12] - **Licaminlimab**: - First precision medicine in ophthalmology for dry eye disease - Utilizes a biomarker to identify high responders, allowing for smaller and more efficient Phase 3 trials [12][13] - Expected top-line results in Q4 2026 [41] Neuro-Ophthalmology Franchise - **Privosegtor**: - First product in neuroprotection, targeting optic neuritis and NAION - Received Breakthrough Therapy Designation - Demonstrated preservation of retinal and ganglion cells in trials, with significant functional improvements [16][21] - Market potential of $7 billion for optic neuritis and NAION, with no current solutions available [18] - Phase 3 trials (Pioneer 1 and Pioneer 2) expected to start in 2026, with readouts planned for 2027 [23][35] Market Opportunity - **DME Market**: - Significant unmet medical need with a large patient pool, as DME is the leading cause of blindness in the working-age population in the US [34] - OCS-01 aims to address both early intervention and patients not responding to current treatments [7][9] - **Neuro-Ophthalmology Market**: - High potential due to the lack of existing treatments for conditions like optic neuritis and NAION, which are closely linked to multiple sclerosis (MS) [19][20] - Plans to expand treatment to all types of MS relapses, significantly increasing the addressable market [40] Strategic Focus - **Commercial Strategy**: - Focus on the US market for launch, with potential partnerships for ex-US markets [26] - Emphasis on innovative and differentiated product profiles to ensure successful commercial launches [26] - **Execution and Risk Management**: - Acknowledgment of execution risk as a primary concern, with a commitment to high-quality trial execution [31][32] - Confidence in the biological efficacy of products based on consistent preclinical and clinical data [39] Manufacturing and Partnerships - **Manufacturing Strategy**: - Oculis does not have in-house manufacturing capabilities; instead, it partners with established global manufacturers to maintain flexibility and quality [44] Conclusion - Oculis is positioned to make significant advancements in the ophthalmology and neuro-ophthalmology sectors with its innovative product pipeline, addressing critical unmet medical needs and leveraging a strong financial position for future growth [25]

Visionary Innovation Riad Sherif, M.D. Chief Executive Officer J.P. Morgan Healthcare Conference January 14, 2026 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," "intend," "future," "potential," or "continue," and other similar expressions are intended to identi ...

Core Insights - Oculis Holding AG is advancing its late-stage pipeline, particularly focusing on Privosegtor and OCS-01, which address significant unmet medical needs in ophthalmology and neuro-ophthalmology [1][6] Group 1: Privosegtor - Privosegtor has received breakthrough therapy designation from the U.S. FDA for treating optic neuritis, based on positive results from the ACUITY Phase 2 trial [2][10] - In the ACUITY trial, patients receiving Privosegtor showed an average gain of 18 letters in vision improvement compared to placebo, indicating a clinically meaningful two-fold improvement in visual resolution [3] - The PIONEER program has been launched to support registration for Privosegtor in optic neuritis and non-arteritic anterior optic neuropathy (NAION), with a potential market opportunity exceeding $7 billion in the U.S. [4][7] Group 2: OCS-01 - OCS-01 is in Phase 3 development as the first eye drop treatment for diabetic macular edema (DME), targeting a market opportunity of approximately $3 billion in the U.S. [5][15] - The diagnosed DME population in the U.S. is estimated to be around 1.8 million, highlighting the significant unmet medical need for effective treatments [5] - Topline results from the DIAMOND Phase 3 trials for OCS-01 are expected in Q2 2026, with an NDA submission planned for Q4 2026 [5][6] Group 3: Market Potential - The combined market potential for Oculis' neuroprotective therapies and DME treatments could exceed $30 billion, reflecting the substantial unmet needs in these areas [6][10] - The company is positioned to transform treatment landscapes in neuro-axonal diseases and diabetic eye conditions, which currently lack effective therapies [6][15]

Core Insights - Oculis Holding AG announced that its neuroprotective drug, Privosegtor, has received Breakthrough Therapy Designation from the FDA for the treatment of optic neuritis, addressing significant unmet medical needs in this area [1][7]. Company Overview - Oculis is a global biotechnology company focused on innovation for treating eye diseases, with a pipeline that includes three key development drugs: Privosegtor, OCS-01, and Licaminlimab [15]. - The company is headquartered in Switzerland and has operations in the United States and Iceland, backed by leading global healthcare investment funds [15]. Drug Development and Market Opportunity - Privosegtor is a small molecule peptide mimic that can cross the blood-brain and retinal barriers, potentially becoming the first neuroprotective treatment for optic nerve disorders, with a combined market opportunity of over $7 billion in the U.S. alone [2][5]. - The ACUITY Phase 2 study demonstrated significant improvements in visual acuity with Privosegtor, showing an average increase of 18 letters on the ETDRS visual acuity scale compared to placebo after three months [4][7]. Clinical Trial Insights - The ACUITY study involved 36 eligible patients aged 18 to 60 with recent onset optic neuritis, comparing Privosegtor with placebo alongside intravenous steroids [12][13]. - Results indicated that Privosegtor not only improved visual acuity but also maintained the structural integrity of the retina and optic nerve, which typically suffer damage during acute optic neuritis [4][9]. Regulatory Status - The Breakthrough Therapy Designation is intended to expedite FDA review for drugs that show substantial improvement over existing treatments for serious conditions [14]. - Following a successful meeting with the FDA in 2025, Oculis initiated the PIONEER program, which includes three registration studies to support the approval of Privosegtor for optic neuritis and NAION [5][6].