At a symposium during the 49th meeting of the Macula Society in Coronado, California, Ocular Therapeutix presented detailed results from its phase 3 SOL-1 trial evaluating Axpaxli (OTX-TKI) for neovascular age-related macular degeneration (nAMD). The intravitreal insert met its primary endpoint and demonstrated superiority to aflibercept. The company said the results are intended to support a new drug application to the US Food and Drug Administration (FDA) through an accelerated pathway.

SOL-1 enrolled 344 treatment-naïve patients and compared a single injection of Axpaxli, a bioresorbable hydrogel incorporating the tyrosine kinase inhibitor axitinib, with aflibercept 2 mg (Eylea; Regeneron) following an 8-week loading regimen. At week 36, 74.1% of Axpaxli-treated patients maintained vision, defined as loss of fewer than 15 ETDRS letters, compared with 55.8% in the aflibercept arm (risk difference, 17.5%; P=.0006). At week 52, 65.9% of Axpaxli-treated patients maintained vision vs 44.2% in the aflibercept arm (risk difference, 21.1%; P<.0001).

Rescue-free rates and central subfield thickness control were also higher with Axpaxli through week 36. Safety findings were consistent with prior studies, with no treatment-related ocular serious adverse events reported.

During the symposium, lead investigator Arshad M. Khanani, MD, said Axpaxli is the first investigational agent with a novel mechanism of action to demonstrate superiority over an approved anti-VEGF therapy in an FDA-aligned study in wet AMD. Additional presentations included Dilsher S. Dhoot, MD, who discussed real-world treatment challenges in nAMD; Peter K. Kaiser, MD, who reviewed the mechanism of action; Jeffrey S. Heier, MD, who outlined the trial design; and Patricio G. Schlottmann, MD, who presented safety data. Mark R. Barakat, MD, and Adnan Tufail, MBBS, MD, presented case examples and key insights. The symposium concluded with a panel discussion.

The study was conducted under a special protocol assessment agreement. A second phase 3 trial, SOL-R, is ongoing, with topline results expected in the first half of 2027.

Prior to the symposium, Pravin U. Dugel, MD, executive chairman, president, and CEO of Ocular Therapeutix, spoke with Retinal Physician, Ophthalmology Management's sister publication, about the trials, the regulatory pathway for Axpaxli, and what the results could mean for the future of wet AMD care. That interview is available here.