Ocular Therapeutix Inc Announces Additional Positive Week 52 Data from Landmark SOL-1 Phase 3 Trial of AXPAXLI in Wet AMD

Published: 2026-04-13 15:08:50 ET OCUL

Published on 04/13/2026 at 03:08 pm EDT

Ocular Therapeutix, Inc. announced additional positive Week 52 data from the SOL-1 Phase 3 superiority trial of AXPAXLI (also known as OTX-TKI), its investigational product candidate for the treatment of wet age-related macular degeneration (wet AMD). The additional post-hoc analyses, presented at the 14th Annual Vit-Buckle Society (VBS) Meeting, reinforce the superior durability, strong sustained disease control, and generally well-tolerated safety seen with AXPAXLI in SOL-1. Highlights from the data presented at VBS include: Strong overall efficacy profile with unmatched durability: Achieved statistical significance in the first three of five key secondary endpoints tested in hierarchical order. In addition, six other pre-specified secondary endpoints measuring clinically significant functional and anatomic outcomes were met with statistical significance.

Sustained disease control (post-hoc analysis): Subjects in the AXPAXLI arm had significantly lower risk in anatomic worsening from Week 8 compared to the aflibercept (2 mg) arm. Week 8 was chosen to give both arms adequate time from the last aflibercept (2 mg) injection and start at a similar reference timepoint. The median time to =30 uM Central Subfield Thickness (CSFT) increase from Week 8 was 39 weeks in the AXPAXLI group and 16 weeks in the aflibercept (2 mg) group, a 23-week difference.

An estimated hazard ratio of 0.7 indicates that, at any time from Week 8 to Week 52, subjects in the treatment group experienced a 30% lower risk of the event (descriptive p=0.0028) compared with the control group. The median time to =75 uM CSFT increase from Week 8 was 46 weeks in the AXPAXLI group and 24 weeks in the aflibercept (2 mg) group, a 22-week difference. An estimated hazard ratio of 0.5 indicates that, at any time from Week 8 to Week 52, subjects in the treatment group experienced a 50% lower risk of the event (descriptive p. In all AXPAXLI subjects who had a vitreous floater AE, drug particles are no longer visible (mean time of 20 weeks).

Subjects treated with AXPAXLI generally maintained loading phase visual acuity gains up to Week 52 across quartile subgroups based on BCVA at screening. Ocular Therapeutix remains on track to submit its New Drug Application (NDA) for AXPAXLI in wet AMD based on the SOL-1 trial alone, subject to planned formal discussions with U.S. FDA. This planned submission is consistent with recent FDA commentary, including a February 2026 New England Journal of Medicine editorial indicating that a single well-controlled Phase 3 trial is expected to become the new default standard for approval.

In early-April, 2026, the FDA Commissioner further noted that this framework is anticipated to be phased in over the next six months, highlighting that the same statistical power can be achieved with a well-designed, well controlled, appropriately powered, single pivotal trial. These recent comments further reinforce the Company?s confidence in NDA acceptability based on SOL-1 alone, aligning with its goal of bringing AXPAXLI to patients as quickly as possible. AXPAXLI (also known as OTX-TKI) is an investigational, bioresorbable, intravitreal hydrogel incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties, being evaluated for the treatment of wet AMD and diabetic retinal disease.

The registrational Phase 3 SOL-1 trial (NCT06223958) is designed to evaluate the safety and efficacy of AXPAXLI in a multi-center, double-masked, randomized (1:1), parallel group trial that involves more than 100 clinical trial sites located in the U.S. and Argentina. In December 2024, the trial completed randomization of 344 treatment-naïve subjects with a diagnosis of wet AMD in the study eye. Two randomized subjects withdrew from the trial prior to receiving Day 1 treatment.

The superiority trial has an eight-week loading segment prior to randomization. During the loading segment, subjects who have 20/80 vision or better and a central subfield thickness (CSFT) of =500 µm receive two doses of aflibercept (2 mg) at Week -8 and Week -4. Subjects who achieve best corrected visual acuity (BCVA) of 20/20 at Day 1 (baseline) or gain at least 10 Early Treatment Diabetic Retinopathy Study (ETDRS) letters at Day 1 along with a CSFT of =350 µm were then randomized to receive a single dose of AXPAXLI (0.45 mg) or a single dose of aflibercept (2 mg). At Week 52 and at Week 76, all subjects are re-dosed with their respective initial treatment of AXPAXLI (0.45 mg) or aflibercept (2 mg).

Subjects will be followed for safety until the end of Week 104. Throughout the trial, subjects are assessed monthly. Trial subjects and designated trial personnel will remain masked through the end of Week 104.

The clinical trial protocol requires that, during the trial, subjects in either arm meeting the pre-specified rescue criteria, which includes a BCVA loss of =15 ETDRS letters from baseline or new vision-threatening macular hemorrhage, will receive a supplemental dose of aflibercept (2 mg). The protocol provides that after the first rescue injection, rescue therapy may be provided at investigator discretion per their clinical judgement. The primary endpoint of SOL-1 is the proportion of subjects who maintain visual acuity, a loss of <15 ETDRS letters of BCVA from baseline, at Week 36.

Predefined statistical rules were applied to adjust for treatment discontinuation or deviation as per the pre-specified statistical analysis plan. The trial remained masked following Week 36 and subjects were evaluated for treatment durability at Week 52. The trial is being conducted under a Special Protocol Assessment (SPA) agreement with the FDA.

In February 2026, Ocular reported positive SOL-1 Week 52 topline data. The superiority primary endpoint was met with 74.1% of subjects in the AXPAXLI (0.45 mg) arm maintaining vision at Week 36, a 17.5% risk difference (p=0.0006), compared to aflibercept (2 mg) arm. 65.9% of subjects treated with AXPAXLI (0.45 mg) maintained vision at Week 52, a 21.1% risk difference (p<0.0001), compared to aflibercept (2 mg) arm.