Ocular Therapeutix (NASDAQ:OCUL) reported positive top-line results from its SOL-1 Phase 3 clinical trial evaluating AXPAXLI (also referred to as OTX-TKI) in wet age-related macular degeneration (wet AMD), highlighting sustained disease control, visual and anatomic outcomes, and a safety profile the company described as well tolerated.
SOL-1 design and primary endpoint
On the conference call, Executive Chairman, President, and CEO Dr. Pravin Dugel said SOL-1 was a large, randomized, well-controlled Phase 3 registrational trial designed with a superiority primary endpoint under a special protocol assessment (SPA) agreed with the FDA. The study evaluated a single injection of AXPAXLI (0.45 mg) versus a single injection of aflibercept (2 mg) after an eight-week loading segment, with the primary endpoint assessed at week 36.
Top-line efficacy: vision and anatomy
Dugel said SOL-1 met its primary endpoint, with 74.1% of subjects in the AXPAXLI arm maintaining vision at week 36 versus 55.8% in the aflibercept arm. He cited an observed difference of 18.3% and a risk difference of 17.5% using the pre-specified statistical model, with a p-value of 0.0006.
Management and investigators also emphasized week 52 results, describing continued durability. Dugel reported that 65.9% of AXPAXLI subjects maintained vision at week 52.
Beyond vision endpoints, speakers repeatedly pointed to optical coherence tomography (OCT)-based anatomic measures. Dugel reported that 55.9% of AXPAXLI-treated subjects maintained central subfield thickness (CSFT) within 30 microns of baseline through week 36, with a nominal p-value of 0.0013 and a risk difference of 17.1% in favor of AXPAXLI. At week 52, 44.1% of AXPAXLI subjects maintained CSFT within 30 microns of baseline, he said. Steering Committee Chair Dr. Arshad Khanani added that 37.8% of patients in the aflibercept arm maintained CSFT within 30 microns at week 36.
Rescue-free rates and durability observations
Several speakers highlighted rescue-free rates as an indicator of durability after a single dose. Dr. Darius Moshfeghi, an independent rescue monitor for SOL-1, said on-protocol rescue-free rates in the AXPAXLI arm were 80.6%, 74.7%, and 68.8% at weeks 24, 36, and 52, respectively. Khanani also referenced a 56.4% rescue-free rate for aflibercept at week 36.
Both investigators and management discussed “read-through” to Ocular’s ongoing SOL-R trial, which is prospectively evaluating fixed six-month dosing. Khanani and Moshfeghi said that when SOL-R rescue criteria were applied to the SOL-1 dataset, approximately 80% of AXPAXLI-treated patients would have remained rescue-free at six months; Dugel later cited 77.1% at week 24 using those criteria. They argued SOL-R enrolls a more stable population due to an extensive screening and loading phase designed to exclude early persistent fluid or significant fluid fluctuations.
Safety and tolerability discussion
On safety, Dugel said AXPAXLI was generally well tolerated in SOL-1, with no observed treatment-related ocular or systemic serious adverse events. Moshfeghi said the safety profiles between study arms were “nearly indistinguishable” and noted that more than 95% of patients reached the week 52 durability assessment, with no meaningful discontinuations following repeat dosing in that period.
In Q&A, analysts asked about cataracts. Khanani said the study population was elderly and that the numbers were “very low,” adding he did not view the imbalance as clinically meaningful based on what had been presented, while noting the data would be reviewed in more detail as available. He also said he looked for intraocular inflammation and events such as retinal vasculitis, occlusive retinal vasculitis, and endophthalmitis, and stated none of those were reported in the dataset discussed.
Regulatory and upcoming disclosures
Dugel said the company believes SOL-1 represents the type of single, well-controlled, FDA-aligned registrational trial that can support an NDA filing and that Ocular plans to hold formal discussions with the FDA and pursue approval through the accelerated 505(b)(2) pathway. He added this strategy does not alter the ongoing SOL-R non-inferiority trial, which completed randomization in December 2025 and is expected to have top-line data in the first quarter of 2027.
Ocular said more detailed SOL-1 results are expected to be presented at the 49th Macula Society Annual Meeting. During Q&A, management said it was still assembling the complete dataset and planned to share additional curves and analyses as soon as possible.
Separately, Dugel provided an update that CFO and COO Donald Notman, who is on temporary medical leave, is “doing very well,” though he does not yet have a specific timetable to return.
About Ocular Therapeutix (NASDAQ:OCUL)
Ocular Therapeutix, Inc is a biopharmaceutical company dedicated to the development of innovative therapies for diseases and conditions of the eye. Founded in 2011 and headquartered in Bedford, Massachusetts, the company focuses on sustained-release drug delivery platforms designed to address key unmet needs in ophthalmology. Its proprietary hydrogel-based inserts and sealants aim to improve patient compliance and outcomes by providing controlled release of active pharmaceutical ingredients directly to ocular tissues.
The company’s flagship product, DEXTENZA®, is a preservative-free, sustained-release dexamethasone intracanalicular insert approved by the U.S.
