Ophthalmology pipeline watch: Key trial results and PDUFA dates for Q1 2026

Editor's Note: This content was generated with the assistance of AI.

Clinical trial watchers can anticipate the release of data from a number of trials in the first quarter of 2026. The trials focus on Leber hereditary optic neuropathy, dry eye, wet age-related macular degeneration (AMD), thyroid eye disease (TED), and meibomian gland dysfunction, among others.

Dry eye disease is a particular focus of ophthalmologists. The following two data sets are expected in early 2026.

• Reproxalap (Aldeyra Therapeutics) for dry eye disease has an extended PDUFA date of March 16, 2026.
• AZR-MD-001 (selenium sulfide ophthalmic ointment, Azura Ophthalmics) is a treatment for meibomian gland dysfunction. The 12-month long-term safety phase 3 data from the ASTRO study are expected during the first quarter of 2026.

Ernest Kornmehl, MD, is anticipating these data from the ocular surface disease treatments. He is in private practice in Wellesley Hills, MA.

“Selenium sulfide ointment will be a welcome addition to our armamentarium in the management of meibomian gland dysfunction (MGD). It reduces the signs and symptoms of MGD with only twice-weekly application and results in more glands secreting meibum.”

He continued, “Reproxalop, for dry eye and allergy, works rapidly in reducing symptoms by blocking reactive aldehydes, but patients will have to follow the treatment schedule of four times a day for 4 weeks, then twice a day for 2 weeks. This should not be an issue if they are highly motivated," he commented.

Gene therapies are also advancing through the approval process. Raxone is one of them.

• Raxone (idebenone, Chiesi Farmaceutici), a treatment for Leber hereditary optic neuropathy, has a PDUFA date of February 28, 2026.

“It is important to find a treatment for devastating, rare diseases. These conditions are difficult to study, and some can progress rapidly and be difficult to diagnose, especially in the pediatric population in retina. Clinical trials are in several stages, and we are hopeful the treatments will lead to improved outcomes. When FDA approval occurs, continued scientific evaluation of outcomes in the real world is important. We are hopeful for Raxone, especially when we think of pediatric eye disease,” commented Mary Elizabeth Hartnett, MD. She is the Michael F. Marmor, MD, Professor in Retinal Science and Diseases and a Professor of Ophthalmology at Stanford University, Stanford, CA.

Other data releases to watch for include the following:

• Brimochol PF (brimonidine tartrate and carbachol, Tenpoint Therapeutics) is a fixed-dose combination drop intended to treat presbyopia. The company anticipates a January 28, 2026, Prescription Drug User Fee Act (PDUFA) date. The potential drug approval is based on positive results from the phase 3 BRIO trials.
• The Axpaxli/Duravyu (axitinip/vorolanib, Ocular Therapeutix), a sustained-release intravitreal implant for wet AMD, and 1-year topline data from the phase 3 SOL-1 trial are expected during the first quarter of 2026. The company will submit a New Drug Application afterward based on their expected positive results.
• Elegrobart (VRDN-003, Viridian Therapeutics) is a subcutaneous drug for TED. The topline data from the phase 3 REVEAL-1 trial are expected in the first quarter of 2026.
• Tarcocimab (KSI-301, Kodiak Science) is a drug for diabetic retinopathy. The company is expected to release the topline data from the phase 3 GLOW2 trial in the first quarter of 2026.
• OPGx-BEST1 (Opus Genetics) is a gene therapy to treat Best vitelliform macular dystrophy. The treatment’s preliminary data from the phase 1/2 trial (BIRD-1) are expected during the first quarter.
• OCU410 (Ocugen, Inc.) is a subretinal injection for geographic atrophy secondary to AMD. Ocugen is expected to release the results of its phase 2 study this quarter.

The following are a few clinical trials of interest to ophthalmologists that are in the patient-recruitment phase.

• NCT03131726: Treatment of newly diagnosed Graves´ ophthalmopathy with simvastatin.
• NCT05941182: Evaluation of a Real-world Implementation Model for Remote Consultation with Vision Self-testing (ReVise)
• NCT06985264: Dry Eye in Upper Eyelid Surgery: Muscle Versus Fat Removal (enrollment by invitation)
• NCT06693856: Multi-center Prospective Clinical Study on Glucocorticoid Pulse Therapy for Graves' Ophthalmopathy (enrollment by invitation)
• NCT05694858: Transdermal Microneedle Lignocaine Delivery Versus EMLA Patch for Topical Analgesia Before Venipuncture Procedure to Adults in Clinical Setting
• NCT06812845: Patterns of Eye Diseases Among Patients Attending the Eye Investigation Unit of Sohag University Hospital
• NCT05126147: Hydroxychloroquine in Mild Graves' Orbitopathy

New study launch

• A new study, the PAINT Trial for chronic central serous chorioretinopathy, is expected to launch in January 2026. The first data would not be available until later in the year. The PAINT study is a prospective, multicenter, placebo-controlled trial to evaluate half-dose photodynamic therapy.

For more information on the progress of specific ongoing studies, visit the ClinicalTrials.gov database.

Security and Exchange Commission filings

Expected Security and Exchange Commission filings and quarterly financial updates early in 2026 for the 4^th quarter of 2025 include earnings reports from Bausch + Lomb, Alcon, Carl Zeiss Meditec, and Hoya Corporation, primarily in February and March.

• Hoya Corporation (7741.T): Friday, January 30, 2026.
• Bausch + Lomb Corporation: Estimated for Wednesday, February 18, 2026, or February 24, 2026.
• Alcon AG: Estimated for Tuesday, February 24, 2026.