Ocular Therapeutix (OCUL) Investor presentation summary

Unmet need in nAMD

• UK real-world data showed ranibizumab-treated eyes gained 2 letters in Year 1, then lost 2 letters by Year 3; median injections were 6, 4 and 4 per year.

• Real-world treatment burden contributes to under-treatment and discontinuation; in one 2,302-patient study, 28.5% stopped anti-VEGF therapy.

• Higher retinal thickness variability was associated with poorer visual outcomes and roughly twice the odds of fibrosis or macular atrophy in the highest-variability group.

• The presentation identifies a need for more durable therapy that can sustain disease control while reducing treatment burden.

OTX-TKI approach

• OTX-TKI combines axitinib, an intracellular tyrosine kinase inhibitor, with a bioresorbable hydrogel insert designed for sustained, tunable release.

• Preclinical delivery data show axitinib in retina and choroid after a single implant; the hydrogel is designed to bioresorb completely and predictably.

• The insert is being developed to provide continuous delivery for up to 12 months, with a familiar intravitreal injection approach; it remains investigational and unapproved.

SOL-1 results

• Phase 3 SOL-1 met its primary endpoint: at Week 36, 74.1% on OTX-TKI versus 55.8% on aflibercept 2 mg maintained vision, defined as less than 15-letter BCVA loss (P=0.0006).

• At Week 52, vision maintenance was 65.9% with OTX-TKI versus 44.2% with aflibercept 2 mg (P<0.0001).

• In a post hoc analysis, 74.7% of OTX-TKI subjects were rescue-free at Week 36; at Week 52, rescue-free proportions were 68.8% versus 47.7%.

• Through Week 52, nine intraocular inflammation events occurred in seven OTX-TKI subjects; all were mild or moderate and resolved, with no endophthalmitis or retinal vasculitis cases reported.