Ocular Therapeutix (OCUL) 14th Annual Vit-Buckle Society Meeting presentation summary

Mechanism of action and product profile

• OTX-TKI is a hydrogel-based, bioresorbable implant delivering axitinib, a highly selective and potent pan-VEGFR tyrosine kinase inhibitor, for sustained intraocular release up to 12 months.

• Axitinib demonstrates >90% inhibition of VEGFR1/2/3 and >85% inhibition of PDGFRα/β at physiologic ATP and drug concentrations, with minimal off-target activity.

• The hydrogel platform leaves no long-term remnants and is designed for predictable, complete bioresorption, supporting redosing.

• OTX-TKI is administered via a familiar intravitreal injection using a 25-gauge needle, aiming for seamless adoption in retina practices.

• The implant is intended to improve adherence and reduce treatment burden compared to current anti-VEGF therapies.

Clinical program and trial design

• The SOL-1 Phase 3 trial is a multicenter, double-masked, randomized, parallel-group superiority study comparing a single OTX-TKI (0.45 mg) dose to a single aflibercept (2 mg) dose in treatment-naïve nAMD patients.

• The trial design aligns with FDA guidance, with both arms on identical dosing schedules and no sham injections, conducted under a Special Protocol Assessment (SPA) agreement.

• The primary endpoint is the proportion of subjects maintaining visual acuity (<15 ETDRS letter loss) at Week 36.

• Key secondary endpoints include maintenance of visual acuity at Week 52, time to rescue injection, and anatomic outcomes such as central subfield thickness (CSFT).

• The study enrolled 344 subjects, well balanced in demographics and baseline characteristics, with high retention through Week 52.

Efficacy results

• OTX-TKI met the primary endpoint, with 74.1% of subjects maintaining visual acuity at Week 36 versus 55.8% for aflibercept (p=0.0006).

• At Week 52, 65.9% of OTX-TKI subjects maintained visual acuity versus 44.2% for aflibercept (p<0.0001).

• Approximately 75% of OTX-TKI subjects remained rescue-free through Week 36, and 72% through Week 52, significantly higher than aflibercept.

• OTX-TKI delayed time to first rescue injection and provided sustained control of retinal anatomy and fluid.

• Rescue-free subjects maintained their initial vision gains and demonstrated better disease control.