Positive topline data in Phase 1/2 allogenic cell therapy trial

Aurion Biotech announced positive topline data from its Phase 1/2 clinical trial for AURN001, an allogenic cell therapy being investigated for treatment of corneal edema secondary to corneal endothelial dysfunction. According to the company’s press release, the trial evaluated three different doses of the allogenic human corneal endothelial cells (neltependocel)/rho kinase inhibitor (Y-27632) treatment as well as neltependocel and Y-27632 independently. The company reported that the proportion of responders in the high-dose AURN001 group showed a 15 letter or more improvement in BCVA that was statistically significant compared to the Y-27632-only arm. There was also a statistically significant improvement in change in BCVA and central corneal thickness in the high-dose group compared to the Y-27632-only group. All doses were well tolerated with no serious ocular adverse events, and a dose-dependent improvement was observed in the primary endpoint. The study was prospective, multicenter, randomized, double masked, parallel arm, and dose ranging, assessing safety, tolerability, and efficacy; 97 subjects were enrolled and randomized. With these results, the company plans on bringing the high-dose of AURN001 (1.0 × 106 neltependocel + 100 μM Y-27632) into its proposed Phase 3 pivotal trials.

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Investigational, light-based treatment for infectious keratitis

The University of Miami Bascom Palmer Eye Institute and Provectus Biopharmaceuticals formed the company VisiRose to develop and commercialize a novel, light-based treatment for infectious keratitis and other severe eye infections: Rose Bengal Photodynamic Antimicrobial Therapy (RB PDAT). According to the company’s press release, the investigational, non-invasive treatment combines rose bengal sodium with a light-based device to treat bacterial, fungal, and parasitic infections. The company reported that the treatment has already shown “rapid infection resolution and improved patient outcomes in clinical trials across the U.S., India, Brazil, and Mexico.”

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Topline results from Phase 3 clinical trial for chronic thyroid eye disease

Viridian Therapeutics announced that veligrotug met all primary and secondary endpoints in its Phase 3 clinical trial for patients with chronic thyroid eye disease. According to the company’s press release, the week 15 proptosis responder rate with veligrotug was 56%. The trial was the first global Phase 3 study for patients with thyroid eye disease to demonstrate a “statistically significant and clinically meaningful 56% diplopia responder rate (placebo-adjusted rate of 31%, p=0.0006) and 32% rate of diplopia complete resolution (placebo-adjusted rate of 18%, p=0.0152).” Ninety-four percent of participants completed their treatment course, with a 9.6% placebo-adjusted rate experiencing hearing impairment. The company plans to submit a BLA in the second half of 2025. Veligrotug is an anti-insulin-like growth factor-1 receptor antibody delivered intravenously.

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Primary endpoint met in trial investigating extended dosing interval for retinal vein occlusion treatment

Regeneron announced that the primary endpoint was met in its trial investigating Eylea HD (aflibercept), 8 mg, dosed every 8 weeks compared to Eylea (aflibercept), 2 mg, dosed every 4 weeks. According to the company, the visual gains on the 8-week Eylea HD dosing regimen were non-inferior compared to that in the 4-week Eylea standard-of-care group. The company plans on submitting a BLA to the FDA in the first quarter of 2025.

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Rare Pediatric Disease Designation granted to investigational retina gene therapy

eyeDNA Therapeutics, a wholly owned subsidiary of Coave Therapeutics, was granted a Rare Pediatric Disease Designation by the FDA for its novel gene therapy (HORA-PDE6b) being investigated for patients with inherited retinitis pigmentosa caused by PDE6b gene mutations. According to the company’s press release, HORA-PDE6b is an AAV5-based gene therapy that will deliver a functional copy of the PDE6b gene into the subretinal space with the hope of delaying or stopping degeneration of photoreceptor cells in patients with this mutation.

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Merger and acquisition news

• Tenpoint Therapeutics and Visus Therapeutics completed a merger.

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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.
• Video submission opportunities: Several symposia at the upcoming ASCRS Annual Meeting are seeking case videos. Learn more about these submission opportunities here.
• 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. Stay tuned each week through April 2025 for a new video.

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Research highlights

• A retrospective cost analysis published in the Journal of Cataract & Refractive Surgery sought to determine whether dropless cataract surgery with an intracameral antibiotic and subconjunctival steroid could reduce healthcare system and patient out-of-pocket costs, compared to topical drop regimens. The costs for topical regimens were analyzed from the 2020 Medical Expenditure Panel Survey and dropless from pharmaceutical invoices/catalogs. According to the study, there were 583 prophylactic topical ophthalmic purchases for a mean system cost per eye of $76.20±SD 39.07 for the lowest cost topical steroid (prednisolone). The lowest cost for subconjunctival steroid (triamcinolone acetonide) was $4.01. The authors reported that “per eye, the lowest cost dropless, injection-based regimen, at $15.91, results in an $87.99 (84.7%) reduction in overall healthcare costs and a $43.64 (100%) reduction in patient out-of-pocket costs relative to the lowest cost topical regimen ($103.90±43.14 mean system cost and $43.64±37.32 mean out-of-pocket cost per eye).” Totaling this to the annual national costs for the healthcare system and patients’ out of pocket, use of intracameral moxifloxacin and subconjunctival triamcinolone acetonide could reduce costs by $450,000,000 and $225,000,000, respectively, according to the paper.
• The effect of topical cyclosporine A, 1%, as an adjuvant therapy in patients with acute Stevens-Johnson syndrome was evaluated in a randomized controlled trial that included 44 patients (88 eyes). Patients in the study presented within 3 months of acute disease onset. Group A received the cyclosporine with standard therapy and Group B received saline drops with standard therapy. Ocular surface parameters were assessed after 6 months. After 6 months, the authors reported that both groups showed a significant improvement in mean severity grades of conjunctival hyperemia, mucocutaneous junction involvement, and meibomian gland involvement. Corneal keratinization grade and neovascularization grade were worse at 6 months in the cyclosporine group. Other intergroup comparisons did not show significant differences. The authors concluded that adjuvant treatment with topical cyclosporine was not superior to standard therapy alone. This paper was published in the British Journal of Ophthalmology.

This is the last EyeWorld Weekly of 2024. EyeWorld Weekly will return on January 10. The EyeWorld Team wishes you a Happy Holidays!

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