Ocular Therapeutix (OCUL) OSN New York & Retina presentation summary

nAMD treatment burden and unmet need

• Anti-VEGF care can require up to 12 injections yearly; 90% of patients need injections every 1–3 months, and 40% discontinue in year 1 due to treatment burden.

• Real-world datasets show injection intervals increasing over time alongside declining vision: by year 7, mean BCVA change was −2.1 letters in Vestrum and −5.0 in IRIS.

• Retinal thickness variability was associated with poorer outcomes; the most variable quartile had roughly twice the odds of fibrosis or macular atrophy, and about 9 fewer letters of vision gain in a separate analysis.

• The proposed treatment goals are lower burden, continuous VEGF suppression and sustained disease control that accommodates real-world adherence.

OTX-TKI rationale and clinical program

• OTX-TKI combines axitinib, an intracellular tyrosine kinase inhibitor, with a bioresorbable hydrogel designed for sustained release, complete resorption and potential drug delivery up to 12 months.

• SOL-1 compared a single OTX-TKI 0.45 mg dose with a single aflibercept 2 mg dose; its superiority design was conducted under a Special Protocol Assessment.

• SOL-1 met its primary endpoint: at Week 36, 74.1% on OTX-TKI vs 55.8% on aflibercept maintained vision, defined as <15 ETDRS letters lost (P=0.0006); Week 52 rates were 65.9% vs 44.2% (P<0.0001).

• Time to first supplemental treatment favored OTX-TKI (nominal P=0.0005); Week 52 supplemental-free results were described as an ~6-month advantage over aflibercept.

• Post hoc analysis found 74.7% of OTX-TKI subjects remained supplemental-free through Week 36, with visual and anatomic stability reported among subjects remaining supplement-free.

Safety and treatment response

• 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.

• Vitreous floaters were reported in 21 OTX-TKI subjects and were generally mild to moderate; onset coincided with the hydrogel drug-elution stage, with no associated intraocular inflammation reported.

• In post hoc SOL-1 analysis, 71% of OTX-TKI subjects and 75% of aflibercept subjects met the quantitative dry-retina threshold at Week 8.

• Among OTX-TKI subjects dry at Week 8, post hoc analyses estimated ~75% could reach a 6-month and ~70% a 9-month dosing interval; these were exploratory estimates based on observed cases.