Ocular Therapeutix (OCUL) Investor presentation summary

Unmet need in nAMD management

• High treatment burden and suboptimal visual outcomes persist with current anti-VEGF therapies, leading to poor adherence and under-treatment.

• Real-world data show frequent injections, high non-persistence rates, and significant patient-reported barriers such as dissatisfaction, financial burden, and logistical challenges.

• Under-treatment is associated with worse long-term visual outcomes, with visual gains eroding over time.

• Greater retinal thickness variability during anti-VEGF therapy correlates with poorer anatomic and visual outcomes.

• Innovations have improved durability, but the need for longer-acting, effective therapies remains.

OTX-TKI: Mechanism and delivery

• OTX-TKI is a hydrogel-based, bioresorbable implant delivering axitinib, a highly potent, selective pan-VEGF tyrosine kinase inhibitor.

• The implant provides continuous, consistent intraocular drug levels for up to 12 months, with complete and predictable bioresorption.

• Axitinib demonstrates superior potency and broad inhibition of VEGF, PDGF, and FGF receptors compared to other TKIs.

• Designed for seamless adoption, the platform uses familiar intravitreal injection techniques and aims to improve adherence.

SOL-1 Phase 3 trial design and population

• SOL-1 is a Phase 3, FDA-aligned, superiority trial comparing a single OTX-TKI injection to a single aflibercept 2 mg injection in treatment-naïve nAMD patients.

• Both arms followed the same dosing schedule, with no sham injections, and primary endpoint was maintenance of visual acuity at 36 weeks.

• The enrolled cohort had excellent baseline vision, distinct from prior nAMD trials.