Transepithelial crosslinking NDA submission accepted

Glaukos announced FDA acceptance of its New Drug Application (NDA) for Epioxa, an epi-on crosslinking therapy with its iLink technology for the treatment of keratoconus. According to the company, the Prescription Drug User Fee Act goal date is October 20. The NDA includes data from the company’s two Phase 3 pivotal trials, both of which achieved pre-specified primary efficacy endpoints and achieved “favorable tolerability and safety profiles.”

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Fast Track designation granted to drop being investigated for night driving impairment due to visual disturbances

Opus Genetics announced that it received a Fast Track designation from the FDA for its phentolamine ophthalmic solution, 0.75%, for treatment of vision loss in low light conditions after keratorefractive surgery, specifically for patients with significant chronic night driving impairment. The company is enrolling for its Phase 3 clinical trial for this indication and expects enrollment to be complete within the first half of this year. The company also announced that it completed enrollment in its Phase 3 clinical trial evaluating phentolamine ophthalmic solution, 0.75%, for treatment of presbyopia.

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First-in-human interventional study for gene therapy treating Leber congenital amaurosis 4

MeiraGTx Holdings announced publication of its first-in-human interventional study of rAAV8.hRKp.AIPL1 in children with AIPL1-associated severe retinal dystrophy (Leber congenital amaurosis 4, LCA4) in The Lancet. According to the company’s press release about the study, 4 children were treated unilaterally with the one-time, subretinal injection gene therapy that delivers functional copies of the AIPL1 gene to cone and rod photoreceptors. An additional 7 children with LCA4 were treated with the gene therapy and, according to the press release, all 11 children “who were blind at birth now have visual acuity in treated eyes.” According to the company, “the findings indicate that children under the age of 4 years old with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vision and evidence of protection against progressive retinal degeneration, without serious adverse effects.” The company stated that it plans to submit a Marketing Authorization Application under exceptional circumstances for the treatment based on these results with no additional clinical data required. It is meeting with the FDA on discussions for expedited approval in the U.S.

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Positive outcomes observed for geographic atrophy drug

Cognition Therapeutics announced positive results from its “futility analysis” for its Phase 2 trial of zervimesine (CT1812) for treatment of geographic atrophy secondary to dry AMD. According to the company, this analysis helps determine if a drug candidate is showing signals of efficacy. The results of this analysis included the first 57 participants in the Phase 2 study who completed at least 6 months of dosing; it showed that patients who were treated with zervimesine had slower lesion growth rates than patients in the in the placebo group. A final clinic visit is being completed this month, and complete analysis will be reported in the second quarter of 2025.

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Regenerative Medicine Advanced Therapy designation granted to stem cell therapy for dry AMD

Luxa Biotechnology announced that it received a Regenerative Medicine Advanced Therapy designation from the FDA for RPESC-RPE-4W transplantation, which it described as a cell product for the treatment of dry AMD. The cell product is derived from adult retinal pigment epithelial stem cells and is used to generate retinal pigment epithelium cell progeny. According to the company’s press release, “transplantation of progenitor-stage RPESC-RPE cells obtained after four weeks of differentiation (RPESC-RPE-4W) effectively replaces the native RPE cells lost during AMD disease progression.” The cell therapy is currently in a Phase 1/2a clinical trial. The Regenerative Medicine Advanced Therapy designation from the FDA is designed to accelerate development and review of therapies for serious or life-threatening diseases.

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ASCRS news and events

• 2025 ASCRS Annual Meeting: Registration is open for the ASCRS Annual Meeting, taking place April 25–28, in Los Angeles, California. Find the most up-to-date information on the Real-Time Program here.
• SightLine at ASCRS: This is a new day-long meeting April 24, ahead of the ASCRS Annual Meeting, for all commercial stakeholders in ophthalmology to learn about and collaborate on technologies and care delivery ideas that will forge a more sustainable financial future. Learn more.
• Business of Interventional Glaucoma: Surgeons and practice administrators alike are invited to the ASOA ASCRS Business of Interventional Glaucoma program, taking place Thursday, April 24 from 1:00–5:30 p.m. in Los Angeles ahead of the ASCRS Annual Meeting and ASOA Annual Meeting. Learn more.
• ASCRS 50th anniversary: ASCRS members from the 50 states are sending in their perspectives on the Society, its impact on their career, and its influence on the specialty as a whole. Tune in each week through April 2025 for a new video.

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Research highlights

• Research published in Nature Communications found that stem cell exhaustion could contribute to meibomian gland shrinkage due to aging that is then associated with evaporative dry eye. The investigators of the study used snRNA-seq, in vivo lineage tracing, ex vivo live imaging, and genetic studies in mice to identify biomarkers for stem cell populations “that maintain distinct regions of the gland and uncover Hedgehog (Hh) signaling as a key regulator of stem cell proliferation. Consistent with this, we show that human Meibomian gland carcinoma exhibits increased Hh signaling.” The authors report that aged glands show decreased signaling, deficient innervation, and loss of collagen I “in niche fibroblasts.” This, they continued, indicates that “alterations in both glandular epithelial cells and their surrounding microenvironment contribute to age-related degeneration.” The authors think these findings could lead to new approaches for treating meibomian gland loss.
• A multicenter, retrospective, database study based out of the U.K. evaluated the risk of and risk factors for zonular dialysis in a fellow eye during phacoemulsification cataract surgery. The study included 66,288 patients. Zonular dialysis occurred intraoperatively in 0.5% of these patients and in the fellow eye in 0.5% of patients. The risk of the fellow eye developing zonular dialysis was higher if the first eye experienced zonular dialysis compared to if the patient had not experienced zonular dialysis in their first surgery. Prior zonular dialysis was the most significant risk factor, followed by pseudoexfoliation in the fellow eye, previous pars plana vitrectomy, and poor pupil dilation. The authors consider these findings important for patient counseling purposes and for surgery allocation. The research is published in the Journal of Cataract & Refractive Surgery.

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Product news

• Alcon launched SYSTANE PRO Preservative-Free as an over-the-counter eye drop for dry eye in U.S. markets this month and will extend to additional markets in 2026.
• SightScore, the first polygenic risk score testing for primary open angle glaucoma, was launched by Seonix Bio. The saliva-based genetic test is intended to identify individuals who are at high risk for developing glaucoma vs. at low risk to allow for more personalized treatment and monitoring.

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