EW Weekly, January 13, 2017

January 13, 2017
Volume 22 , Number 2

Lucentis approved for mCNV

Lucentis (ranibizumab, Genentech, South San Francisco) has been approved in the U.S. for the treatment of myopic choroidal neovascularization (mCNV), Genentech said in a press release. This is the first anti-vascular endothelial growth factor (VEGF) therapy to be approved for this indication in the U.S. This approval is based on the results of the Phase 3 RADIANCE study (N=276), which demonstrated that treatment with Lucentis provided superior visual acuity gains in people with mCNV compared to verteporfin photodynamic therapy (vPDT). At 3 months, average visual acuity gains for patients treated with Lucentis were more than 12 letters, compared to 1.4 letters for those treated with vPDT.

Spark granted orphan drug status for IRD compound

Spark Therapeutics (Philadelphia) received an amended orphan drug designation from U.S. regulators for voretigene neparvovec to encompass all Inherited Retinal Disease (IRD) caused by biallelic RPE65 mutations, the company announced. The decision came on the heels of new 4-year data from a Phase 1 clinical trial and natural history study findings that enhance the understanding of investigational voretigene neparvovec, a one-time adeno-associated viral (AAV) gene therapy for IRD caused by the RPE65 gene mutation. Voretigene neparvovec has received breakthrough therapy and orphan product designations from the FDA, as well as orphan product designation from the European Medicines Agency. Spark Therapeutics initiated a rolling submission of the Biologics License Application and expects to complete the application early this year. Mean improvements in functional vision and visual function were maintained through 4 years as measured by both the multi-luminance mobility test and full field light sensitivity threshold testing in a Phase 1 clinical trial in which investigational voretigene neparvovec was administered to the contralateral eye. This cohort of participants (n=8) received the same dose of voretigene neparvovec that was administered in the Phase 3 trial and would have met the Phase 3 eligibility criteria, Spark noted. Results after 4 years showed a sustained average durability of effect across the Phase 1 clinical trial cohort, with a mean lux score change of 2.4±0.46 at year 4, compared to 2.6±0.56 at year 1. No serious adverse events associated with voretigene neparvovec or deleterious immune responses have been observed.

Emixustat granted orphan drug designation for Stargardt

Emixustat hydrochloride has been granted orphan drug designation in the U.S. for the treatment of Stargardt disease, developer Acucela (Seattle) said in a news release. Vitamin A is crucial to the visual process, and emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65), Acucela said. Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of A2E and related compounds. In animal models of Stargardt disease and retinal degeneration, emixustat was found to stop and reverse the accumulation of A2E and to preserve the integrity of the retina. Emixustat when delivered orally was found to be generally well tolerated in human clinical studies with delayed dark adaptation being the most common ocular adverse event.

First patients enrolled in Phase 2 study of THR-317 for DME

A Phase 2, single-masked, multicenter exploratory study evaluating the safety and efficacy of two dose levels of THR-317 for the treatment of diabetic macular edema (DME) has enrolled its first patients, developer ThromboGenics (Leuven, Belgium) said in a press release. THR-317 is a recombinant human monoclonal antibody directed against the receptor-binding site of human placental growth factor (PIGF). The Phase 2 study will evaluate the safety of three intravitreal injections of two dose levels of THR-317 (4 mg or 8 mg). The trial will also assess the anti-PIGF compound's ability to improve best corrected visual acuity and to reduce central retinal thickness in subjects with DME. The study plans to enroll a total of 50 patients over a period of about 12 months. The first results from the study are expected in the first quarter of 2018.

Mt. Sinai establishes Eye and Vision Research Institute

The Mount Sinai Health System announced the creation of the Mount Sinai/New York Eye and Ear (NYEE) Eye and Vision Research Institute (New York). The Institute is the first of its kind in the New York City metropolitan region, the group said. The Institute "will foster collaborations between faculty at Icahn School of Medicine at Mount Sinai (ISMMS) and academic departments throughout the Mount Sinai Health System," and will combine expertise from other fields of medicine to find breakthrough treatments and cures for eye conditions and disease, and will further research that can preserve vision and/or reverse blindness. Planned areas of research expansion include stem cell and regenerative biology of the visual system, ocular imaging, and genetics and genomics of eye disease.

Roche buys ForSight VISION4

ForSight VISION4, a privately held biotechnology company in Menlo Park, California, has been acquired by Roche Holdings (Basel, Switzerland), the company said. ForSight is developing port delivery system (PDS) platforms for the treatment of retinal diseases. In 2010, ForSight VISION4 inked a deal with Genentech (a Roche subsidiary) to use the PDS platform on its ranibizumab compound. The PDS is a durable intravitreal implant that is placed through a scleral incision in a one-time surgical procedure. It is then refilled using a proprietary refill needle by a physician in an office setting. The ranibizumab PDS is currently in a Phase 2 study to define the duration of drug delivery possible with the PDS technology.


  • Myopia in early life is one of the most common ocular sequelae in type 1 retinopathy of prematurity survivors, according to J.Y. Lok and colleagues. In their retrospective case series, the clinical records of all infants with type 1 ROP who had undergone laser therapy between 2007 and 2012 in Hong Kong were reviewed. Among 494 babies screened, 14 Chinese babies (26 eyes) were recruited with 1:1 male-to-female ratio. All eyes showed gradual progression of myopia in the first 3 years of age but no significant change of astigmatism. Further correlation analysis showed no correlation with laser energy consumed, birth weight (p=0.14), head circumference growth (p=0.57) and body weight growth (p=0.71). However, severity of myopia was related to the post-conceptual age when receiving laser therapy (p<0.005), gestation age (p=0.02) and possibly body height growth with age (p=0.05). The study is published in International Ophthalmology.
  • Central field index (CFI) is less variable than visual field index (VFI) in stable eyes with fixation involvement, especially in severe glaucoma, indicating a need to include CFI calculations for monitoring advanced disease in eyes with central defects, according to a new study. A. Rao and colleagues retrospectively identified multiple visual fields (VFs) of patients with repeatable central fixation involvement on Humphrey VFs (24-2 and 10-2 program) that were stable (clinically and on VFs) over a very short period of 2 to 3 months. The inter-visit difference for VFI was greater than CFI ranging from -4% to 9% versus -1% to 8% in early (P=0.9), -13% to 18% versus -6% to 17% (P=0.056) in moderate, and -21% to 19% versus -9% to 9% (P<0.001) in severe glaucoma. The CFI within each group had narrower range than VFI with maximum range in severe glaucoma (33% to 95%). The divergence of CFI from VFI started at MD 24-2 beyond (worse) -10 dB. This difference between CFI and VFI was associated significantly with number of points with P<1% on 24-2 (R=80.3%). The study is published in the Journal of Glaucoma.
  • Patients on tamsulosin had an increased risk of a 1-day postoperative IOP spike after cataract surgery, showing the importance of identifying patients on tamsulosin preoperatively to better manage and potentially mitigate IOP spikes, according to Levi Bonnell, MPH, and colleagues. In their retrospective cohort study, registry data from men who had cataract surgery were evaluated and included if they had been taking tamsulosin at the time of surgery. The study comprised 584 men (864 eyes). An IOP increase greater than 10 mm Hg or IOP 30 mm Hg or higher after cataract surgery occurred in 12.4% and 9.3%, respectively, of eyes in the tamsulosin group versus 4.4% and 2.1%, respectively, in the control group (all P=.001). After adjusting for significant covariates, patients on tamsulosin were 2.6 times (P=.01) and 3.8 (P=.01) more likely to have a 1-day postoperative IOP increase greater than 10 mm Hg or a 1-day postoperative IOP of 30 mm Hg or higher. The study is published in the Journal of Cataract & Refractive Surgery.

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