Oculis Holding (OCS) investor relations material

Strategic focus and pipeline overview

• Transitioned from pure ophthalmology to dual franchises in neuro-ophthalmology and ophthalmology, with three late-stage programs targeting DME, dry eye disease, and neuroprotection in optic neuropathies.

• Privosegtor demonstrated strong efficacy in acute optic neuritis, showing significant functional and structural improvements over steroids alone, and is advancing to pivotal phase III trials (PIONEER-1, PIONEER-2).

• PIONEER-3 will target NAION, with a similar design, and a third indication for acute MS relapse is in preparation for next year.

• The DME program (OCS-01) is the first eye drop for retina, with a phase III readout expected in Q2 2026, targeting both early and VEGF non-responsive patients.

• OCS-02, a topical anti-TNF for dry eye, is being developed as a personalized medicine based on a genetic biomarker, aiming for higher efficacy and smaller, more targeted trials.

Clinical and regulatory progress

• Privosegtor’s phase II data showed an 18-letter improvement in vision at three months, sustained at 15 letters at six months, and 42% better preservation of retinal ganglion cells compared to steroids.

• FDA is highly supportive, allowing direct progression to pivotal trials for both AON and NAION due to the unique neuroprotective mechanism and strong data.

• The acute optic neuritis trial enrolls patients within 12 days of symptom onset, with high engagement from both the medical community and patients, especially those with MS.

• Readouts for Privosegtor are at three months (efficacy), six and twelve months (safety), with additional biomarker data at one month.

• OCS-01 has shown consistent efficacy across global trials, with a 7.6-letter gain in vision, comparable to LUCENTIS but without injections.

Market opportunity and commercialization

• The U.S. market for optic neuropathies is estimated at $7 billion annually, with orphan pricing and no current competition.

• Privosegtor addresses a major unmet need, offering neuroprotection where steroids only reduce inflammation but do not prevent vision loss.

• OCS-01 is positioned for broad adoption, with strong support from retina specialists and potential expansion to ophthalmologists, enabling earlier intervention in DME.

• OCS-02’s genotype-based approach could deliver five to seven times better response in selected patients, improving trial efficiency and commercial outcomes.

• Upcoming catalysts include phase III readouts for DME in Q2 2026 and further precision medicine data in dry eye disease.