• Regeneron Pharmaceuticals announced The Lancet published one-year results from the pivotal PULSAR and PHOTON trials for EYLEA HD (aflibercept) Injection 8 mg. The publications detailed data demonstrating that EYLEA HD extended dosing regimens were non-inferior to EYLEA (aflibercept) Injection 2 mg for both the treatment of wet AMD and diabetic macular edema (DME). Both PULSAR in wet AMD (N=1,009) and PHOTON in DME (N=658) met their primary endpoints, with EYLEA HD demonstrating non-inferior and clinically equivalent vision gains at 48 weeks with both 12- and 16-week dosing regimens after only 3 initial monthly doses, compared to an EYLEA 8-week dosing regimen after initial monthly doses (3 in PULSAR and 5 in PHOTON). Additionally, 79% and 77% of wet AMD patients and 91% and 89% of DME patients, who were respectively randomized to 12- and 16-week dosing, maintained these extended dosing intervals through 48 weeks.

• MediPrint Ophthalmics completed its Glaucoma SIGHT-2, Phase 2b study that evaluated the company’s lead candidate LL-BMT, a preservative-free, weekly drug-eluting contact lens vs a control group on bimatoprost 0.01% ophthalmic solution. The results showed clinically meaningful and sustained IOP reduction (of about 30%) over the duration of 3 weeks, which is statistically comparable to 0.01% bimatoprost eyedrops. No serious treatment emergent adverse events were reported, and the lenses were well tolerated. CLDEQ-8 questionnaire also showed significant improvements in dry eye and contact lens comfort.

• Telios Pharma announced topline results from its Phase 2 study assessing TL-925, a first-in-class topical Bruton’s tyrosine kinase inhibitor for individuals with moderate to severe dry eye disease (DED). TL-925 was safe and well tolerated, and the intention-to-treat analysis demonstrated clinically meaningful and statistically significant efficacy relative to vehicle control across multiple signs and symptoms. The rapid onset of efficacy across multiple ocular regions, coupled with favorable and consistent results in different environments, confirm TL-925’s differentiated profile as a safe, well-tolerated and effective treatment for patients with DED. Complete results will be presented at the ARVO annual meeting in May. A Phase 2b study of TL-925 in DED has been initiated based on these results and is currently enrolling patients. 

• NovaSight published a study demonstrating long-term vision gains achieved with CureSight, the company’s digitized, at-home amblyopia treatment, in the American Journal of Ophthalmology. The study found that children with various types of amblyopia showed significant improvement in both visual acuity (VA) and stereoacuity following short-term binocular treatment with CureSight, and that the gains were maintained for at least one year. The pivotal randomized controlled trial compared vision and stereoacuity improvement outcomes with CureSight vs eye patching. At 12-weeks post-treatment, improvement in amblyopic eye VA was maintained with no statistically significant change compared to the end-of-treatment visit. At one year, there was a partial reduction in the amblyopic eye VA gain compared to end-of-treatment but with a statistically significant residual gain of two lines compared to baseline (0.20±0.14 LogMar Mean, SD). For more, see the study results here.

• Exonate Ltd. announced positive results from its Phase 1b/2a trial for its lead candidate EXN407. The data demonstrated the safety and tolerability of EXN407 as well as clear indications of biological activity, positioning it for further development as a topical treatment for retinal vascular diseases such as diabetic retinopathy (DR) and DME. In addition to the primary safety and tolerability endpoints, the study concluded that there were promising signals of biological response from EXN407, demonstrating sustained decreases in macular thickness relative to the placebo group and comparable to previously reported anti-VEGF injections. Additionally, EXN407 treatment led to a significant decrease in vascular leakage (60% of EXN407-treated patients relative to 20% placebo) and EXN407 inhibited further increases to vascular leakage (10% of EXN407-treated patients relative to 50% placebo). Full results of the trial will be presented at the ARVO annual meeting.

• Statistics & Data Corp. (SDC) announced a strategic decision to enhance focus and efficiency by transitioning its ophthalmic biometric division to its partner Ora Inc. SDC’s tech-enabled service offering brings to market an AI/ML enabled clinical ecosystem to unify disparate technologies into a single data lake (SDC DataHub) and a single point of access reporting platform (SDC Insights), paired with a mobile ePRO/eCOA Solution (SDC Capture). SDC’s tech-enabled service offerings feature eSource, eConsent, ePRO, safety processing, custom workflows, integrated IRT and EDC options and solutions to strengthen oversight and reduce timelines.

• The FDA cleared iView Therapeutics’ investigational new drug application for the initiation of a Phase 1/2 clinical trial. The trial will evaluate the safety, tolerability and efficacy of IVW-1001, a novel TRPM8 agonist, to treat the signs and symptoms of dry eye diseases. The company says IVW-1001 offers potential as a therapeutic intervention, leveraging its TRPM8 agonist mechanism to target the underlying causes of dry eye.

• GenSight Biologics announced initial results of new meta-analyses in Leber Hereditary Optic Neuropathy, which show those treated with LUMEVOQ (GS010; lenadogene nolparvovec) gene therapy experienced a rate of visual recovery greater than that of idebenone-treated patients and untreated (natural history) patients. The meta-analyses focused solely on patients with the m.11778G>A ND4 mutation. The meta-analyses depict a gradient of efficacy of visual recovery with LUMEVOQ resulting in greater recovery rates than that of idebenone treatment, and both greater than that in the natural history of the disease. This gradient of recovery, based on the CRR measure of visual improvement, is observed at both eye level and patient level. There is no overlap in confidence intervals when LUMEVOQ is compared to idebenone and to natural history, indicating a positive difference in visual outcomes. The company is currently engaging with authorities in the United States, EU and United Kingdom to align on the regulatory path for LUMEVOQ. 

• Bascom Palmer Eye Institute researchers are using a $1 million gift to galvanize their work on whole eye transplants. Eduardo Alfonso, MD, director of Bascom Palmer and chair of the Department of Ophthalmology at the Miller School, says the whole eye transplant project aims to provide blind patients with a seeing eye, perhaps by using a biological eye modified to make it functional for vision. The “bionic eye” will likely include an electronic chip, with gene therapy to prevent allograft rejection, stem cell therapy to replace degenerating eye tissue and electronic connections to the brain. David T. Tse, MD, professor of ophthalmology, has designed a surgical technique to transplant and preserve the globe of the eye after it is removed from a donor’s blood supply. Read more about the whole eye transplant research initiative here. 

• According to new research from Oregon Health & Science University (OHSU) and collaborators, published in JAMA Ophthalmology, an artificial intelligence (AI) technology can accurately and independently detect 100% of severe cases of a blindness-causing condition that affects prematurely born babies. The technology has the potential to expand worldwide screening — and sight-saving treatment — for retinopathy of prematurity (ROP). According to OHSU, the study marks the first time that autonomous AI screening for ROP has been shown to work in a real-world population. If the technology is approved by regulatory agencies, ROP would become the second eye disease that can be independently detected by AI, says OHSU. For more, see the study results here. 

Quick Notes is published weekly. Unless otherwise noted, the information presented is based on press releases. Find earlier editions here. To submit a press release to be considered for publication, click here.