Ophthalmology Month in Review: January 2025

With an already robust pipeline, and more available treatments expected in the coming years, ophthalmology kicked off 2025 with a plethora of key updates, providing more hope for patients with blinding eye diseases. Last month, the US Food and Drug Administration (FDA) accepted a revised supplemental New Drug Application (sNDA) for one of the first geographic atrophy (GA) drugs brought to market, granted a regenerative designation to a gene therapy for an incurable genetic eye disease, and paved the way another gene therapy to move to Phase 3 for diabetic macular edema (DME).

In our January 2025 month in review, we also provide a recap of other pipeline news and stories that defined this past month in ophthalmology, with a particular focus on company updates on drugs for disease states including neovascular (wet) age-related macular degeneration (nAMD), Stargardt disease, and acute optic neuritis. We also highlight the latest episodes of our flagship ophthalmology podcast, New Insight with Veeral Sheth, MD, on a new drug offering pharmacological hope for presbyopia.

Here’s what happened in January:

FDA News

FDA Accepts Revised Avacincaptad Pegol sNDA for Geographic Atrophy

The FDA accepted the revised supplemental new drug application (sNDA) for avacincaptad pegol intravitreal solution (IZERVAY) to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD) on January 6. Announced by Astellas Pharma, the FDA set a target action date for February 26, 2025. This announcement comes after the agency filed a Complete Response Letter (CRL) for the sNDA in November 2024.

FDA Grants Regenerative Medicine Advanced Therapy to Laru-Zova for XLRP

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to laruparetigene zovaparvovec (laru-zova) gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP). Announced by Beacon Therapeutics, on January 28, 2025, the RMAT designation expedites the development and review of therapies with the potential to address an unmet need in serious or life-threatening diseases, allowing for enhanced communications with the agency throughout development.

4D-150 Reduces Injection Burden in DME, Paving Way for FDA-Backed Phase 3

4DMT announced topline interim data from Part 1 of the SPECTRA clinical trial, demonstrating the positive safety and clinical activity of 4D-150 in patients with diabetic macular edema (DME). 4D-150 sustained gains in best-corrected visual acuity (BCVA) and a reduction in central subfield thickness (CST), while remaining tolerable without signs of intraocular inflammation (IOI). Feedback from the FDA supports the drug’s progression to Phase 3.

Pipeline News

OCS-05 for Acute Optic Neuritis Achieves Key Endpoints in Phase 2 Trial

Oculis announced OCS-05, an investigational neuroprotective therapy for acute optic neuritis, met its key primary safety endpoint and achieved statistical significance on efficacy-based secondary endpoints in the Phase 2 ACUITY trial. These topline results showed the peptidomimetic small molecule's favorable safety and tolerability profile versus placebo, and its neuroprotective structural benefit and capability to improve visual function.

Gildeuretinol Halts Stargardt Progression in Interim TEASE-3 Results

Alkeus Pharmaceuticals announced positive interim data from the TEASE-3 clinical trial found once-daily oral gildeuretinol halted disease progression in additional patients with early-stage Stargardt disease, stabilizing visual acuity over multiple years. These 2 patients recently completed 24 months of gildeuretinol treatment, achieving consistency with prior trial results, and bringing the total to 5 patients who completed TEASE-3.

Aflibercept 8 mg for RVO Hits Primary Endpoint in Phase 3 QUASAR Trial

Regeneron announced aflibercept 8 mg injection (EYLEA HD) met its primary endpoint in the Phase 3 QUASAR trial, showing improved vision with extended dosing in people with macular edema following retinal vein occlusion (RVO). The higher dose of aflibercept demonstrated non-inferior vision gains with an every 8-week dosing regimen compared with every 4-week dosing of standard-of-care aflibercept injection 2 mg (EYLEA).

OCU400 Improves 2-Year Vision in Retinitis Pigmentosa in Phase 1/2 Trial

Ocugen provided a 2-year Phase 1/2 trial update, reporting the positive efficacy and safety of OCU400, a novel modifier gene therapy platform, for early to advanced retinitis pigmentosa (RP) in pediatric and adult populations. All 9 patients with RP in the Phase 1/2 trial treated with OCU400 saw improved or preserved visual function at 1 and 2 years of study, compared with the untreated fellow eyes.

SING IMT Device Secures Positive 6-Month Data for Late-Stage AMD

Samsara Vision announced an intermediate assessment of visual and safety outcomes found the implantation of the Smaller-Incision New-Generation Implantable Miniature Telescope (SING IMT) benefited distance and near vision in patients with late-stage AMD 6 months post-surgery. The SING IMT showed a low impact on corneal endothelium cell density, with the company indicating an acceptable level of complications and adverse events.

ONS-5010 for Wet AMD Secures Noninferiority in NORSE EIGHT Trial

Outlook Therapeutics announced the complete safety and efficacy analysis of NORSE EIGHT revealed the noninferiority of ONS-5010 (LYTENAVA), an ophthalmic formulation of bevacizumab, to ranibizumab for the treatment of neovascular (wet) AMD at 12 weeks. The full 3-month analysis supported the company’s plan to resubmit the Biologics License Application (BLA) for ONS-5010 in the first quarter of 2025.

New Insight

New Insight: A Look at Qlosi for Presbyopia with Elad Kedar, MBA

In the latest episode of our flagship ophthalmology podcast, New Insight, host Veeral Sheth, MD, MBA, director of clinical research at University Retina and Macula Associates, explored the approval and impending launch of Qlosi (pilocarpine HCL ophthalmic solution) 0.4%, a groundbreaking pharmacologic option for managing presbyopia, with Elad Kedar, MBA, chief executive officer of Orasis Pharmaceuticals.