TKI axitinib beats anti-VEGF therapy in wet AMD in phase 3 trial | ASRS 2026

Patients with wet age-related macular degeneration (AMD) treated with a sustained-release formulation of the tyrosine kinase inhibitor (TKI) axitinib were able to spread out their injections by up to nine months, according to data from a phase 3 trial. These data were presented at the American Society of Retina Specialists Annual Scientific Meeting, held July 15 through July 18, 2026, in Montreal.

“We saw unmatched durability. This is the first trial to demonstrate durability greater than nine months,” Dilsher Dhoot, M.D., a vitreoretinal surgeon at California Retina Consultants, said during the session.

Developed by Ocular Therapeutix, axitinib is a small-molecule TKI that has been combined with the company’s hydrogel implant technology. The hydrogel technology allows for a targeted release of axitinib over time.

TKIs are a class of approved targeted oral cancer medications that block the signals that lead to cancer growth. They are emerging as a possible therapy for ophthalmology as an alternative to anti-VEGF injections. TKIs have the potential to inhibit pathways involved in retinal diseases. There are no TKI therapies currently available for eye diseases, but several are in late-stage development.

Treatments for eye disease such as AMD or diabetic macular edema include anti-VEGF agents such as Avastin (bevacizumab), Lucentis (ranibizumab) and Eylea (aflibercept). They work by binding to VEGF proteins or their receptors on the cell and stop abnormal blood vessels from forming.

TKIs, on the other hand, bind to the VEGF receptor in the cell and prevent activation of receptor signaling. When combined with the hydrogel implant, axitinib is able to target several VEGF receptors and provide continuous delivery. The hydrogel developed by Ocular Therapeutix is bioresorbable.

SOL-1 is a phase 3 registrational trial comparing a single dose of axitinib with aflibercept 2 mg in treatment-naive patients with wet AMD. In the trial, 344 patients were randomly assigned to either axitinib or aflibercept. The primary end point was the proportion of patients who maintained best corrected visual acuity (BCVA) at week 36. Secondary end points included change from baseline BCVA, central subfield thickness (CST) and proportion of patients who needed rescue therapy at weeks 36 and 52. CST is a key measurement of retinal thickness.

Patients received a loading dose at eight weeks and four weeks before the trial began, and they were only eligible to be randomly assigned if they had a 10-letter gain or 20/20 vision at baseline.

In the trial, 74% of patients treated with axitinib maintained vision compared with 56% for those who received aflibercept 2 milligrams at 36 weeks. This difference was maintained for 52 weeks, at 66% for those treated with axitinib versus 44% treated with aflibercept, Dhoot said.

The proportion of those who were rescue-free was also higher in the axitinib group. At 36 weeks, 74.7% of patients who had received axitinib were rescue-free compared with 56.4% treated with aflibercept. At 52 weeks, 68.8% of axitinib patients were rescue-free compared with 47.7% for aflibercept.

A common adverse event was vitreous floaters, which was mild to moderate. “The onset coincided with the OTX-TKI hydrogel bioresorption process, and for all subjects with vitrified floaters, the drug particles are no longer visible later in the trial,” Dhoot said.

There were nine events of immune-related inflammation seen in seven patients; all cases were mild or moderate in severity and resolved. There were no cases of endophthalmitis or occlusive or nonocclusive retinal vasculitis.

During the session, moderator Charles C. Wykoff, M.D., Ph.D., FASRS, said the criteria used in SOL-1 for when patients would be rescued with another medication are not how patients are managed in the clinic. Wykoff is director of research at Retina Consultants of Texas and the Greater Houston Retina Research Foundation. Dhoot pointed out, however, that the trial design was based on the FDA draft guidance from 2023.

Ocular Therapeutix plans to submit a new drug application for axitinib in wet AMD to the FDA in the fourth quarter of 2026, under the brand name Axpaxli.