EW Weekly, August 28, 2020

FDA approves drops for patients with corneal issues due to cystinosis
The FDA has approved Recordati Rare Diseases’ CYSTADROPS (cysteamine ophthalmic solution, 0.37%) for treatment of corneal cystine crystal depositions in patients with cystinosis. According to a press release from the company, this is the first and only FDA-approved drop with this indication for QID dosing. The cystine crystal buildup associated with this rare genetic condition can cause light sensitivity, ocular discomfort, and pain. A Phase 3 open-label, randomized, controlled, two-arm multicenter trial that helped lead to the drop’s FDA approval saw a 40% reduction in in vivo confocal microscopy corneal cystine density from baseline after 90 days of use, according to the company’s press release. In addition, a Phase 1/2a open-label, adaptive dose-response trial showed that the drops resulted in a 30% decrease in the in vivo confocal microscopy score maintained for 5 years.

Subretinal delivery system for future gene therapies receives FDA approval
Gyroscope Therapeutics received FDA approval for its Orbit Subretinal Delivery System, which the company explained in a press release is a system designed to avoid the need for a vitrectomy and a retinotomy. The company stated that the Orbit system accesses the subretinal space through a suprachoroidal approach with a flexible cannula that has a microneedle inside that delivers the dose of a therapy. Gyroscope Therapeutics noted in its announcement that this FDA approval is based on injection of Balanced Salt Solution (BSS, Alcon) or BSS Plus (Alcon). The company press release stated that Gyroscope plans to license the Orbit system to other companies developing ocular gene and cell therapies. It is also investigating its own gene therapy.

FDA clears IND application for diabetic retinopathy gene therapy
REGENXBIO announced the FDA clearance of its IND application for the diabetic retinopathy therapy RGX-314. According to the company, RGX-314, which is delivered into the suprachoroidal space, will enter its Phase 2 clinical trial in the second half of this year. The dose-escalation study is multicentered, open-label, and randomized. It will enroll 40 patients with diabetic retinopathy in two cohorts with patients randomized to receive either the gene therapy or the control at a 3:1 ratio. Two dose levels of RGX-314 will be evaluated, according to the press release.

Trial started for regenerative therapy for corneal endothelial dystrophies
Trefoil Therapeutics began a Phase 1/2 trial for a regenerative treatment for patients with corneal endothelial dystrophies, including Fuchs. The study, according to a press release from the company, will evaluate TTHX1114, an engineered fibroblast growth factor-1 that is injected intracamerally. The company press release explained that the engineered growth factor is meant to have an increased half-life compared to the naturally occurring version, which is known to stimulate cell proliferation and migration and offer cellular protection. The study, which is prospective, multicentered, randomized, masked, and placebo-controlled, will include up to 71 patients with moderate to severe corneal endothelial dystrophy. Patients involved will receive four injections of the therapy. The company stated that the primary endpoint is endothelial cell count from baseline to day 56.

20/Happy in 2020: ASCRS launches master class
ASCRS continues its “20/Happy in 2020: The ASCRS Master Class in Refractive Cataract Surgery,” this Saturday with Module 2, “Our Refractive IOL Armamentarium – More Choices than Ever.” Offering up to 18 AMA PRA Category 1 Credits TM as well as non-CME symposia that attendees can view live or watch later, attendees who complete the multi-week master class will receive a frameable certificate. A total of nine CME modules will cover topics including preoperative diagnostics, IOL calculations, IOL design, patient selection, education, and counseling, efficiency, and more, with discussion and expertise provided by a faculty of 80 surgeons from around the world. Sessions will consist of lectures, audience Q&A, and live panel discussions. Registered attendees who miss a session or can’t attend live can view the program later online. Learn more and register at https://ascrs.org/20happy.

Research highlights
  • Real-world outcomes of different treatments in initially treatment-naïve patients with diabetic macular edema (DME) were analyzed in a retrospective study that included 27 institutions in Japan. Records included a total of 2,049 eyes between 2010–2015 that were followed for 2 years. Interventions included anti-VEGF therapy, local corticosteroids, macular photocoagulation, and vitrectomy. The mean improvement of BCVA over 2 years among all the groups was −0.04±0.40, with 46.3% of eyes among all groups coming in at >20/40 as a final BCVA. Mean improvement in BCVA was −0.09±0.39, –0.02±0.40, and −0.05±0.39, among the group that received anti-VEGF only, the group that received combination therapy, and the group without anti-VEGF therapy, respectively. Nearly 50% of eyes in the anti-VEGF monotherapy group (49.6%) had a final BCVA of >20/40, compared to 38.9% in the combination group and 52% in the group without an anti-VEGF treatment. The study authors concluded that eyes treated with anti-VEGF monotherapy had a better visual prognosis. The study is published in the British Journal of Ophthalmology.
  • A study published in the journal Cornea found that speed of Descemet membrane stripping for an endothelial keratoplasty graft can influence the graft scroll width and endothelial cell density. The experiment included 20 corneas from 10 donors divided into two groups: group 1, slow stripping speed (greater than 150 seconds, speed <0.057 mm/s) and group 2, fast stripping speed (less than 75 seconds, speed >0.11 mm/s). Mean total tissue preparation time in group 1 was 282.7±28 seconds and 126±50 seconds for group 2, with a mean stripping speed of 0.045±0.006 mm/s and 0.266±0.093 mm/s for group 1 and 2, respectively. Group 1 saw a graft width of 6.4±0.92 mm and mean endothelial cell loss of 389±149 cells/mm2 compared to a graft width of 2.87±0.32 mm and mean endothelial cell loss of 186±63.44 cells/mm2 in group 2. The study authors said this research showed a correlation between stripping speed, scroll width, and endothelial cell loss, with slower stripping associated with wider scrolls and a reduced endothelial cell density.
  • A prospective, single-center, three-arm, randomized trial of 99 eyes of 99 patients with open-angle glaucoma evaluated the extent of ab interno suture trabeculotomy and location of Schlemm’s canal incisions. Thirty-four patients received a 360-degree trabeculotomy, 34 patients a 180-degree trabeculotomy in the upper quadrants, and 31 patients a 180-degree trabeculotomy in the lower quadrants. IOP, number of medications, and complications were evaluated out to 12 months postop, with surgical success being an IOP of ≤21 mm Hg and a ≥20% IOP reduction or an IOP of ≤15 mm Hg and ≥20% IOP reduction with or without medications. Overall, the extent of the suture trabeculotomy and the incision locations did not affect IOP reduction. According to the study authors, each group had comparable mean reductions in IOP and number of medications, from a mean of 18.6 mm Hg on 3.1 medications at baseline to 13.7 mm Hg on 1.4 medications at month 12.

This issue of EyeWorld Weekly was edited by Stacy Jablonski and Vanessa Caceres.

EyeWorld Weekly (ISSN 1089-0319), a digital publication of the American Society of Cataract and Refractive Surgery (ASCRS), is published every Friday, distributed by email, and posted live on Friday.

Medical Editors: Eric Donnenfeld, MD, Chief Medical Editor; Rosa Braga-Mele, MD, Cataract Editor; Clara Chan, MD, Cornea Editor; Nathan Radcliffe, MD, Glaucoma Editor; and Vance Thompson, MD, Refractive Editor

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