FDA approves first IND for RNA exon editor

The FDA approved an investigational new drug (IND) application from Ascidian Therapeutics for ACDN-01, an RNA exon editor, that will now enter clinical trials for treating the genetic cause of Stargardt disease and other ABCA4retinopathies. According to the company, a Phase 1/2 study will begin within the first half of this year. The company reports that this is the first IND granted for an RNA exon editor. ACDN-01 was also granted a Fast Track designation from the FDA.

Back to top

SPA granted for Phase 3 trial of an intravitreal implant for wet AMD

Ocular Therapeutix announced that the FDA has agreed to a special protocol assessment (SPA) agreement modificationfor its pivotal Phase 3 clinical trial of AXPAXLI (OTX-TKI, axitinib intravitreal implant) for treatment of wet AMD. According to the company’s press release, this SPA agreement modification allows the trial to include treatment-naïve patients with a visual acuity of 20/80 or better upon initial screening. Among other criteria to qualify for enrollment, these patients would then need to gain at least 10 ETDRS letters or achieve 20/20 visual acuity from initial screening, after two aflibercept injections, to Day 1 of the trial. According to the company, AXPAXLI is an implant carrying 450 µg of axitinib that is more soluble and “expected to provide for a slightly increased daily release of the drug and is designed to improve synchronization of axitinib drug depletion with hydrogel bioresorption.”

Back to top

Study: retinal imaging helps predict risk of developing a host of diseases

Researchers at Mass Eye and Ear, MIT, and Harvard, published a study in Science Translational Medicine that shows the ability of retinal imaging to help predict a patient’s risk of developing various diseases, including ocular, cardiac, pulmonary, metabolic, and neuropsychiatric. According to the organization’s press release, the investigators used retinal imaging, genetics, and large data to make these estimates, finding significant associations between retinal thinning of different layers and increased risk of developing ocular or systemic diseases.

Back to top

European Medicines Agency recommends rejection of pegcetacoplan for GA

The European Medicines Agency recommended refusing the marketing authorization application for Syfovre (pegcetacoplan injection, Apellis Pharmaceuticals) for treatment of geographic atrophy (GA). Syfovre is FDA approved in the U.S. for treatment of GA secondary to AMD. In a press release, Apellis announced its plans to seek re-examination of their application with the Committee for Medicinal Products for Human Use of the European Medicines Agency, noting the positive data from Phase 3 studies published in The Lancet as well as post-hoc Phase 3 analyses.

Back to top

ASCRS news and events

• ASCRS Annual Meeting: The Preview Program for the ASCRS Annual Meeting, April 5–8 in Boston, Massachusetts, is available online. Find the program, registration information, and more here.
• Eyecelerator @ ASCRS: Eyecelerator, a partnership between ASCRS and the American Academy of Ophthalmology, is bringing back The Winning Pitch Challenge to Eyecelerator @ ASCRS, April 4, in Boston, Massachusetts. Today, February 2, is the last day submissions will be accepted for The Winning Pitch Challenge. Cash prizes for winners are worth up to $25,000. Find more information on submissions here.

Back to top

Research highlights

• A prospective, examiner-masked, randomized clinical trial published in the Journal of Cataract & Refractive Surgery compared the visual performance and photic visual disturbances of patients who received two different EDOF IOLs for a mini-monovision effect. The patients received either bilateral Symfony (Johnson & Johnson Vision) or Vivity (Alcon) with one eye targeted for –0.75 D of myopia. Nearly 130 patients completed follow up (Symfony: n=60; Vivity: n=66). Eighty percent and 84% of patients in the Symfony and Vivity groups, respectively, achieved J2 or better. There were not significant differences between the two groups in terms of distance visual acuity, defocus profiles, contrast sensitivity, reading speed, or patient-reported spectacle independence. In terms of quality of vision, 45% of patients in the Symfony group experienced no bothersome visual disturbances; 66% of patients in the Vivity group had no bothersome visual disturbances. With this, the authors concluded that mini-monovision was well tolerated but the “data suggest that Vivity IOL is associated with a lower probability of bothersome visual disturbances compared with Symfony IOL.”
• The first clinical results for a biotissue stent to facilitate supraciliary outflow in patients with open-angle glaucoma was published in the British Journal of Ophthalmology. The study included 10 patients who had combined phacoemulsification cataract surgery followed by implantation of the endoscleral supraciliary biostent, which the paper describes as being composed of “decellularized scleral allograft tissue” that was “microtrephined into a polymer tubular implant.” Implantation was successful in all patients without any significant adverse events. At 12 months postop, IOP was lowered 40% from baseline to 14.6±3.2 mm Hg, according to the paper, with 80% of participants achieving a more than 20% reduction in IOP. Medication use was reduced 62% from baseline with a mean of 0.5 medications being used at postop month 12. The authors describe the implant as well tolerated and having 11% endothelial cell loss at 12 months, which they note is similar to phacoemulsification alone. Mean BCVA went from 20/130 at baseline to 20/36 at postop month 12.

Back to top

---