EW Weekly, August 7, 2020

Enrollment complete for pivotal clinical trial of glaucoma shunt
MicroOptx announced that enrollment is complete for its Beacon Study evaluating the Beacon Aqueous Microshunt, which has an FDA investigational device exemption. The shunt, according to the company, directs aqueous humor to the tear film vs. an enclosed space in the eye, potentially allowing for more precise, consistent IOP control. The study will evaluate safety, performance, and IOP reduction of the device in refractory glaucoma patients. According to MicroOptx, the initial clinical trial will evaluate the shunt as a standalone procedure, but the company noted that studies in Europe have evaluated it in conjunction with other procedures, such as cataract surgery.

Orphan Drug Designation granted for treatment of RHO mutation-associated retinal degenerative disease
Ocugen’s OCU400 for treatment of RHO mutation-associated retinal degeneration, which represents about 12% of retinitis pigmentosa patients, was granted Orphan Drug Designation by the FDA. OCU400 is a gene therapy candidate that includes a functional copy of the nuclear hormone receptor gene, NR2E3, which according to Ocugen could reset retinal homeostasis to stabilize and rescue degenerating photoreceptor cells.

Clinical trial design for investigational dry eye drug agreed upon
Novaliq announced that it reached an agreement with the FDA for a clinical trial design for CyclASol, a topical anti-inflammatory and immunomodulating ophthalmic solution containing 0.1% cyclosporine A in a water-free formulation. The company already completed the Phase 2/3 ESSENCE trial, which it noted supports filing of an NDA for the drug. It is planning ESSENCE-2 as a multicenter, randomized, double-masked, vehicle-controlled trial to assess efficacy, safety, and tolerability of CyclASol for the signs and symptoms of dry eye. This trial is expected to start in the second half of 2020, enrolling 835 patients across 25 U.S. centers. The study will evaluate change from baseline in corneal staining and eye dryness score at day 28 as well as reading speed.

Positive 1-year data from Phase 1/2a trial of gene therapy for wet AMD
REGENXBIO announced positive data from its Phase 1/2a trial of RGX-314, a gene therapy for wet AMD. The therapy was tolerated in 42 patients at all dosing levels in the trial, and 1-year data showed a 61% and 85% reduction in anti-VEGF injections in cohorts 4 and 5, respectively, a press release from the company reported. The study also showed that 73% of patients in cohort 5 were anti-VEGF injection-free at 1 year. RGX-314 protein expression was dose dependent at 1 year, the press release stated. With this info, REGENXBIO is planning to start a pivotal program for subretinal delivery of the therapy by the end of this year. The company’s active Phase 2 AAVIATE study for suprachoroidal delivery of RGX-314 for wet AMD is expected to start enrolling in the third quarter of 2020, according to the press release.

Topline data supports efficacy, safety of thyroid eye disease drug
The safety and efficacy of TEPEZZA (Horizon Therapeutics), the first and currently only FDA-approved drug for thyroid eye disease, was supported by topline data from two clinical trials. From the OPTIC and OPTIC-X trials, 89% of patients who received placebo in the OPTIC trial and then TEPEZZA in the OPTIC-X trial saw a 2 mm or more reduction in proptosis at week 24. In the OPTIC trial overall, 83% of patients who received TEPEZZA saw a 2 mm or more reduction in proptosis at 24 weeks. In the 48-week follow-up off treatment in the OPTIC trial, patients who were proptosis responders maintained their response without additional treatment at 72 weeks.

2021 Medicare Physician Fee Schedule (MPFS) proposed rule released
On August 3, the Centers for Medicare and Medicaid Services (CMS) released the 2021 MPFS proposed rule, which includes significant reductions in reimbursement for specialty medicine, including ophthalmology. The impact of the restructuring and revaluing of office-based Evaluation and Management Services (E/M), in addition to other CMS proposals to increase the valuation for specific services, results in a budget neutrality adjustment (10.61% reduction) to the conversion factor. Therefore, in order to offset the increased spending, the conversion factor for 2021 will be $32.26, which represents a $3.83 decrease from the 2020 conversion factor of $36.09. The estimated impact on total allowed charges from this proposal is a reduction of 6% for ophthalmology. The cataract code 66984 is reduced by 9%, from $557.58 to $505.84. In addition, the YAG code 66821 in the facility is reduced by 8%, $317.95 to $292.28, and by 7% in the non-facility, $339.24 to $315.19. These are estimated payments based on the projected 2021 conversion factor of $32.2605. In addition, CMS did not change its previous position regarding increases to the postop visits for the 10- and 90-day global surgical services to reflect the increases to the E/M codes. ASCRS will continue to work with the Surgical Coalition, the broader Provider Coalition, and the Alliance of Specialty Medicine in asking Congress to waive budget neutrality for the E/M changes and to address the 10- and 90-day global surgical codes.

Research highlights
  • A quality improvement study was conducted in the Izmir Tepecik Training and Research Hospital in Turkey after the first case of COVID-19 was reported to evaluate the spread of SARS-CoV-2 despite enhanced safety and sanitation measures. The research published in JAMA Ophthalmology describes how asymptomatic patients were triaged into exam rooms and real-time PCR of samples from the biomicroscope, slit lamp breath shield, phoropter, tonometer, and door handles were taken before exams began and after the last patient left the room for the day. A total of 33 people visited the room. The mean exam time was 9 minutes. Two samples post-exam from the slit lamp breath shield and phoropter were positive for the virus. The study authors note that PCR doesn’t confirm infectivity of viral material, only presence.
  • Stem cells (neural progenitor cells) were discovered in the optic nerve lamina region. These cells, according to the research published in the journal Proceedings of the National Academy of Sciences, are capable of generating all macroglial cell types for the anterior optic nerve. A press release about the research from the University of Maryland said that these cells could support repair and protection of the optic nerve fibers. The press release stated that this finding suggests a new theory for glaucoma development and could lead to new treatment therapies for age-related disorders causing vision loss.
  • Two Phase 2 clinical trials that compared faricimab vs. ranibizumab—STAIRWAY and AVENUE—were published in JAMA Ophthalmology. The STAIRWAY 52-week trial included 76 nAMD patients receiving two different dosing regimens of faricimab every 16 or 12 weeks compared to monthly ranibizumab. At 24 weeks, 65% of faricimab patients had no disease activity, which suggests patients could go 16 weeks between treatments. Faricimab patients in both treatment arms gained 9.3 letters (12-week dosing) and 12.5 letters (16-week dosing) at week 40 (ranibizumab patients gained 11.4 letters). Further gains were seen at 52 weeks, with 10.1, 11.4, and 9.6 letters in the 12-week faricimab, 16-week faricimab, and monthly ranibizumab groups, respectively. More patients (46.4%) in the 16-week faricimab group gained 15 or more letters at 52 weeks compared to the ranibizumab group (37.5%). The 36-week AVENUE study of 273 nAMD patients that looked at four dosing regimens of faricimab compared to monthly ranibizumab found comparable outcomes in BCVA among all the groups. The authors of this study concluded that a Phase 3 trial is merited.

Product News
  • EyePoint Pharmaceuticals and ImprimisRx partnered to promote DEXYCU (dexamethasone intraocular suspension) 9%.

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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