Oculis Holding Update Summary Company Overview - **Company**: Oculis Holding AG - **Ticker**: NasdaqGM: OCS - **Industry**: Biopharmaceuticals focusing on neuro-ophthalmology - **Market Potential**: Over $25 billion in neuro-ophthalmology with specific indications for OCS-05 targeting acute optic neuritis (AON) and non-arteritic anterior ischemic optic neuropathy (NAION) [4][12][14] Key Developments - **FDA Interaction**: Successful meeting with the U.S. FDA allowing OCS-05 to advance into the registration phase for AON and NAION [5][6] - **Registration Trials**: - **Pioneer 1**: Planned to start in Q4 2025 for AON - **Pioneer 2**: Planned to start in the first half of 2026 for AON - **Pioneer 3**: Planned to start in mid-2026 for NAION [6][7][15] Clinical Trial Design - **Pioneer Trials**: - Pioneer 1 and 2 will mirror the successful Phase 2 Acuity trial with the same dose and patient population [6][9] - Primary endpoint: Change from baseline in Low Contrast Visual Acuity (LCVA) at month three [10][12] - Secondary endpoints include the proportion of 15-letter gainers at month three and change from baseline in LCVA at month six [10][12] Market Opportunity - **Acute Optic Neuritis (AON)**: - Estimated 30,000 cases per year in the U.S. - Potential market size of over $3 billion with treatment costs between $100,000 to $400,000 per year [12][13] - **Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)**: - Estimated 30,000 to 35,000 cases per year in the U.S. - Potential market size of over $4 billion [14][15] Financial Position - **Balance Sheet**: Strong with no debt and cash runway extending to the end of 2027 without utilizing loan facilities [5] Strategic Focus - **Pipeline**: Multiple pivotal milestones focusing on significant unmet medical needs in neuro-ophthalmology [15][16] - **Future Plans**: - Launching Predict, a Phase 3 trial in genotype-based development for dry eye [15] - Aiming to leverage data from AON trials for potential MS relapse indications [9][15] Regulatory and Clinical Insights - **Regulatory Pathway**: Full alignment with FDA on trial designs and IND submissions for both AON and NAION [5][6] - **Patient Population**: Trials will include both MS and non-MS patients, with a focus on all-comers for AON [9][12] - **Treatment Window**: Treatment should ideally start within 12 days of symptom onset, with an average of 9.5 days observed in previous studies [61] Community Engagement - **Feedback from Neuro-Ophthalmologists**: Positive reception and engagement from the medical community regarding OCS-05, with plans for strong presence at upcoming medical congresses [73][74] Conclusion - Oculis is positioned to be a leader in neuro-ophthalmology with a robust pipeline and significant market opportunities in AON and NAION, aiming to address critical unmet medical needs while maintaining a strong financial foundation [15][16][60]