Ocular Therapeutix Axpaxli Succeeds in Phase 3 Trial; Oculis Plans Q2 2026 Readouts

Ocular Therapeutix announced its experimental Axpaxli eye disease drug succeeded in a Phase 3 study, outperforming a low dose of the blockbuster medicine 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 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.

The success in the low-dose Eylea arm was higher than analysts anticipated, and investors sent Ocular shares down 23% in early trading Tuesday. 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.

The CEO 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," the CEO said in the company's press release.

Ocular is trying to break into a market dominated by Eylea until the 2022 approval of Vabysmo, which offered a less frequent dosing schedule and quickly became a top choice for doctors. The Eylea franchise 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.

Separately, Oculis Holding AG, a clinical-stage ophthalmology-based biopharmaceutical company, announced fourth-quarter and full-year financial results. Net loss for the fourth quarter narrowed to 23.51 million Swiss Francs or 0.42 Franc per share, from 28.66 million Francs or 0.67 Franc per share in the prior year.

For the full year 2025, net loss expanded to 98.96 million Francs, or 1.89 Franc per share, from 85.78 million Francs, or 2.12 Franc per share, in the year ago. In terms of US dollars, this amounts to a net loss of $119.1 million in 2025, compared to $97.4 million in 2024.

As of December 31, 2025, the firm reported having cash, cash equivalents, and short-term investments of $268.7 million or 213.0 million Francs compared to $109.0 million or 98.7 million Francs in the prior year, providing a cash runway into 2029.

The company's lead product candidate, OCS-01, is a topical dexamethasone ophthalmic formulation in Phase 3 clinical trials, DIAMOND1 and DIAMOND 2, for the treatment of Diabetic Macular Edema. Both the Phase 3 DIAMOND trials with OCS-01 have enrolled over 800 patients and are nearing completion.

The trial readouts from Phase 3 trials with OCS-01 are expected in Q2 2026. If positive, a subsequent NDA submission to the FDA is planned for Q4 2026. While the US DME market is currently valued at approximately $3 billion, only a fraction of the 1.8 million people diagnosed with the disease are successfully managed according to the firm.

Privosegtor, a neuroprotective candidate of Oculis intended for the treatment of optic neuropathies like optic neuritis and non-arteritic anterior ischemic optic neuropathy, recently received Breakthrough Therapy designation based on the Phase 2 ACUITY results in optic neuritis. This sight-threatening neuro-ophthalmic condition is often the first clinical manifestation of multiple sclerosis.

In the Phase 2 ACUITY trial results, Privosegtor + steroid showed substantial improvements in vision and consistent anatomical and biological neuroprotective benefits compared with placebo + steroid. After a successful meeting with the FDA in the fall of 2025, Oculis launched the PIONEER program, which includes three pivotal trials in optic neuritis and non-arteritic anterior ischemic optic neuropathy.

The first registrational trial in the program, PIONEER-1 in ON, was initiated in Q4 of 2025, with clinical sites activation progressing as planned. PIONEER's registrational program in optic neuropathies is estimated to have a potential US market opportunity of over $7 billion, according to Oculis.

Licaminlimab, a topical anti-TNFa which is being developed with a genotype-based approach to drive precision medicine in dry eye disease, was recently initiated in the first genotype-based registrational trial, PREDICT-1. In Phase 2 studies, Licaminlimab showed a substantially greater treatment effect in patients carrying a specific TNFR1 genotype, with profound improvements ranging from 5-fold greater in signs to 7-fold greater in symptoms according to the firm.

The PREDICT-1 trial results for Licaminlimab, with a precision medicine approach in dry eye disease, are planned for Q4, 2026.