Oculis Holding (OCS) investor relations material

Pipeline and Regulatory Progress

• Achieved full FDA alignment to advance Privosegtor into registration trials for acute optic neuritis (AON) and non-arteritic anterior ischemic optic neuropathy (NAION), using a single IND for both indications.

• PIONEER-1 (AON) to start in Q4 2025, PIONEER-2 (AON) in H1 2026, and PIONEER-3 (NAION) in mid-2026, all mirroring the successful phase II ACUITY trial design.

• Privosegtor has received Orphan Drug designation from both FDA and EMA for AON.

• Plans to cross-reference Privosegtor AON IND for a new IND submission in MS relapse in 2026.

• OCS-01 for DME is in a phase III registration trial with readout expected in Q2 2026; company maintains a strong balance sheet with cash runway into end of 2027.

Clinical Trial Design and Endpoints

• PIONEER-1 and -2 will use the same dose, patient population, and endpoints as ACUITY, focusing on LCVA change at month three as the primary endpoint.

• Secondary endpoints include proportion of 15-letter gainers at three months and LCVA change at six months.

• NAION trial (PIONEER-3) will leverage operational synergies with AON trials, using similar centers and data collection methods.

• Each indication will have independent statistical powering to maximize success probability.

• Privosegtor is a first-in-class neuroprotective peptoid targeting trophic factor pathways.

Market Opportunity and Unmet Need

• AON and NAION are orphan indications with over 30,000 annual U.S. cases each and no approved therapies.

• U.S. market potential estimated at over $3B for AON and $4B for NAION, with rare disease pricing analogs of $100k–$400k per treatment.

• Both indications target concentrated prescriber bases and represent significant unmet needs in neuro-ophthalmology.

• Privosegtor could become the first neuroprotective therapy to improve vision outcomes in these conditions.

• ACUITY Phase 2 trial showed improvement in visual function and anatomical preservation of the retina.