Ocular claims a win with eye drug data even as shares sink

Dive Brief:

Ocular Therapeutix on Tuesday said its experimental Axpaxli eye disease drug succeeded in a Phase 3 study, outperforming a low dose of the blockbuster medicine Eylea.
The trial followed 344 patients who were newly diagnosed with wet age-related macular degeneration and deemed likely to suffer from vision loss while meeting other criteria. After an initial period in which all the participants received two injections of 2 mg Eylea, subjects randomly received either one dose of Axpaxli or another 2 mg dose of Eylea.
After 36 weeks, 74% of patients on Axpaxli maintained a prespecified level of visual acuity compared with about 56% in the Eylea arm. The success in the low-dose Eylea arm was higher than analysts anticipated, and investors sent Ocular shares down 23% in early trading Tuesday.

Dive Insight:

Ocular is trying to break into a market dominated by Regeneron’s Eylea until the 2022 approval of Roche’s Vabysmo, which offered a less frequent dosing schedule and quickly became a top choice for doctors. Regeneron answered with a longer-acting version of its own medicine, Eylea HD, the next year.

The Eylea franchise – marketed by Bayer outside the U.S. – still brings in billions of dollars a year, though revenue has been slipping with the new competition. In 2025, net U.S. sales of Eylea and Eylea HD dropped 27% to $4.4 billion.

Ocular is now hoping to enter the fray with a different type of drug known as a tyrosine kinase inhibitor, or TKI. Company executives worked with the Food and Drug Administration on a Special Protocol Assessment agreement, which outlined the parameters of the study with the goal for Ocular to seek a superiority label.

CEO Pravin Dugel said company executives were “thrilled” with Ocular’s study results and claimed the drug had met a difficult bar. “Many pivotal programs have failed at demonstrating superiority in a Phase 3 trial, and we do not foresee other programs attempting this challenge or pursuing a superiority label in this indication,” Dugel said in the company’s press release.

But it’s unclear how successful Ocular will be. RBC Capital Markets analyst Brian Abrahams, who covers Regeneron, sees a limited effect on the Eylea franchise. While the Ocular trial succeeded, “the benefits seem somewhat modest,” Abrahams wrote in a note to clients. The study design also didn’t reflect the way many retina specialists manage their patients, he said.

The RBC analyst who covers Ocular, Lisa Walter, had a more positive take, saying a highly statistically significant result “is a win” and highlighting Ocular’s plan to use the data to seek FDA approval. The study “should be positive for [Ocular] in the long term, but we understand investors might be initially disappointed,” she wrote to clients.