Positive results in Phase 1 trial for geographic atrophy cell-based therapy

Eyestem Research announced positive Phase 1 results for its Eyecyte-RPE therapy for patients with geographic atrophy secondary to dry age-related macular degeneration. According to the company’s press release, no patients in any of the three cohorts experienced any serious adverse events. In addition, the first six patients had an improvement of 14.9 letters within 4–6 months post-transplant. The company also reported retinal scans showing “early signs of disease reversal in a handful of patients.” According to the company’s press release, Eyecyte-RPE is a “cryopreserved, allogenic retinal pigment epithelium cell suspension.”

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Enrollment complete for Phase 3 DME drop trials

Oculis Holding AG announced that it completed enrollment for its two Phase 3 trials for OCS-01 eye drops for the treatment of diabetic macular edema (DME). The trials enrolled more than 800 patients at 119 U.S. and international sites. Both trials are double masked and randomized to evaluate the safety and efficacy of OCS-01 in patients with DME for 52 weeks. Topline data is expected in the second quarter of 2026. If approved, the company reported that OCS-01 would be the first topical treatment for this indication, addressing an unmet need for “early treatment intervention or for patients with inadequate response to anti-VEGF therapy.” Separately, in an R&D event, Oculis also provided updates about its plans to begin a Phase 2/3 study for licaminlimab (OCS-02) in the second quarter of this year. It also announced expanded analysis from its Phase 2 trial for privosegtor (OCS-05) for acute optic neuritis and two new programs investigating use of this drug as a neuroprotective treatment for non-arthritic anterior ischemic optic neuritis and for acute treatment relapses in patients with multiple sclerosis.

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Enrollment complete in study for hydrogel retinal sealant

Pykus Therapeutics announced enrollment completion in its pilot study of PYK-2101, a hydrogel retinal sealant for patients with retinal detachment. According to the company’s press release, the study, taking place in Australia with 11 patients, is measuring safety and efficacy of PYK-2101 16 weeks after vitrectomy. PYK-2101 is intended to accelerate recovery following retinal detachment surgery by eliminating the need for patients to remain face down after surgery as with other endotamponade techniques. The company plans to submit an Investigational Device Exemption application to the FDA in the fourth quarter of this year to begin a multicenter pivotal trial in the U.S.

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First patient dosed in Phase 2 trial studying oral diabetic retinopathy therapy

Vantage Biosciences announced that the first patient was dosed in its Phase 2 clinical trial evaluating VX-01, an oral therapy for non-proliferative diabetic retinopathy that targets inflammatory mechanisms underlying this disease progression. The Phase 2 study is multicenter, double masked, randomized, and placebo controlled, evaluating the oral therapy in patients with moderate to severe non-proliferative diabetic retinopathy without central diabetic macular edema. Patients will take VX-01 or placebo BID for 52 weeks and will be followed for a 12-week post-treatment period.

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Regenerative Medicine Advanced Therapy designation granted to gene therapy candidate for X-linked retinoschisis

Atsena Therapeutics announced that it received a Regenerative Medicine Advanced Therapy designation from the FDA for ATSN-201 for treatment of X-linked retinoschisis (XLRS). According to the company’s press release, this gene therapy candidate uses a novel “spreading capsid,” which “spreads laterally beyond the subretinal injection site,” to achieve gene expression in central retina photoreceptors and avoid risks of foveal detachment. The company’s Phase 1/2 dose-escalation, dose-expansion trial is ongoing and enrolling male patients 6 years old and older with a diagnosis of XLRS caused by RS1 gene mutations.

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ASCRS news and events

• 2025 ASCRS Annual Meeting: One week until the ASCRS Annual Meeting and ASCRS Subspecialty Day begin. Registration will be available online and onsite 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. Registration available online and onsite 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. Registration available online and onsite. Learn more.

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Research highlights

• The refractive prediction accuracy of the Zeiss AI IOL Calculator was compared to the Barrett Universal II and Kane formulas in a retrospective case series conducted at the Cullen Eye Institute, Baylor College of Medicine, in Houston, Texas. The authors of the paper published in the Journal of Cataract & Refractive Surgery described the Zeiss AI IOL Calculator as an artificial intelligence-based IOL-optimized formula. Refractive prediction errors were calculated in the entire dataset as well as subgroups (short eyes, long eyes). The dataset included 10,838 eyes. The investigators wrote that the Barrett Universal II “produced significantly greater SDs, [root mean square absolute error], and [mean absolute error] in the whole group and short eyes, and the Kane had greater SD, [root mean square absolute error], and [mean absolute error] in short eyes (all adjusted P<.05); the [Barrett Universal II] had significantly lower percentages of eyes within ±0.50 D of [prediction errors] in the whole group (80.0% vs 81.2%) and in short eyes (71.3% vs 76.1%), and the Kane had lower percentage of eyes within ±0.50 D of [prediction errors] in short eyes (71.9% vs 76.1%) (all adjusted P<.05).” With this the authors concluded that the Zeiss AI IOL Calculator had superior performance to both comparator formulas, especially in short eyes.
• A review published in the journal Eye conducted a systematic search of various research databases to gain a better understanding of the possible efficacy of botulinum toxin A (BTX-A) as a treatment for dry eye disease. The meta-analysis included 10 studies with 513 patients, finding that BTX-A “significantly improved” tear breakup time by 1.79 seconds, Schirmer test scores by 3.72 mm, and OSDI scores by –7.51. Tear meniscus height was also increased by 0.10 mm. These findings, according to the authors, support “enhanced tear stability, production, and symptom relief” and the “use of BTX-A in clinical practice as a promising treatment for DED.”

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