FDA Approves Eye Drops for Pharmacologically Induced Mydriasis

■ Ocuphire and Viatris announced that the FDA has approved Ryzumvi (phentolamine ophthalmic solution) 0.75% for the treatment of pharmacologically induced mydriasis produced by adrenergic agonists or parasympatholytic agents. Ryzumvi is expected to be commercially available in the United States in the first half of 2024.

Ryzumvi was evaluated in the comprehensive MIRA clinical trial program involving more than 600 patients, including the MIRA-1 phase 2b trial, MIRA-2 and MIRA-3 phase 3 pivotal trials, and MIRA-4 phase 3 pediatric trial. In the MIRA-2 and MIRA-3 trials, 553 patients aged 12 to 80 years who had mydriasis induced by instillation of phenylephrine or tropicamide or a combination of hydroxyamphetamine hydrobromide and tropicamide were randomized. Two drops (study eye) or 1 drop (fellow eye) of Ryzumvi or placebo were administered 1 hour after instillation of the mydriatic agent. The percentage of patients with study eyes returning to ≤0.2 mm from baseline pupil diameter was statistically significantly greater (P<.01) at all time points measured from 60 minutes through 24 hours in the Ryzumvi group compared with the placebo group across both of the MIRA-2 and MIRA-3 trials. The efficacy of Ryzumvi was similar for all age ranges including pediatric subjects.

“Our hope is that by addressing patient dilation barriers, we’re empowering eye care professionals to broaden exam availability, leading to enhanced eye health outcomes,” said Viatris Eye Care Division President Jeffrey Nau, PhD, in a news release.

Adverum Announces Positive Aflibercept Data From LUNA Phase 2 Trial

■ Adverum Biotechnologies announced initial aflibercept protein expression data from its ongoing phase 2 LUNA trial evaluating ixoberogene soroparvovec (Ixo-vec) for the treatment of wet age-related macular degeneration (AMD). Ixo-vec is an aflibercept-encoding AAV.7m8 vector designed to cross the inner limiting membrane, enabling in-clinic intravitreal delivery and enhanced retinal transduction. The new data include aflibercept protein levels for both the 2E11 and 6E10 vg/eye doses, suggesting that both doses are within the therapeutically active range. Notably, these protein expression levels are consistent with levels observed in the OPTIC trial, in which aflibercept levels and corresponding clinical activity have been sustained in patients through multiple years of follow-up after a single Ixo-vec injection.

In a news release, trial investigator Arshad M. Khanani, MD, MA, explained, “These results from the LUNA study could be clinically meaningful as the aflibercept levels measured in this trial are comparable to the sustained aflibercept levels seen in patients treated in the OPTIC trial.”

Apellis Receives Permanent J-Code for Syfovre for Geographic Atrophy

■ Apellis Pharmaceuticals announced that the Centers for Medicare and Medicaid Services has assigned a permanent and product-specific J-code (J2781) for Syfovre (pegcetacoplan injection), the first treatment for geographic atrophy (GA) secondary to AMD. By targeting C3, Syfovre is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. The recommended dose is 15 mg of Syfovre administered intravitreally in each affected eye once every 25 to 60 days to achieve a reduction in the mean rate of GA lesion growth.

“The permanent J-code is a significant milestone that will help ensure accurate and efficient reimbursement of Syfovre in all treatment settings, building on our goal of bringing this important treatment to GA patients in need,” Adam Townsend, chief commercial officer of Apellis, said in a news release.

Orbis Joins Forces With Heidelberg to Fight Avoidable Blindness Around the World

■ Sight-saving charity Orbis has announced a partnership with high-tech imaging solutions company Heidelberg Engineering. Heidelberg will fund the joint venture that aims to improve vision services by training eyecare professionals around the world using Cybersight, Orbis’s telemedicine and e-learning platform. In the first half of 2023, Cybersight webinars reached more than 15,000 attendees. The new sessions supported by Heidelberg have the potential to equip thousands of ophthalmic professionals worldwide to provide higher quality eye care for their communities. Heidelberg’s funding will also allow Orbis to continue its research project into retinoblastoma. The project will also use Cybersight to investigate the impact of artificial intelligence on worldwide retinoblastoma care.

Atsena Doses First Patient in Phase 1/2 of X-linked Retinoschisis Trial

■ Atsena Therapeutics announced the first patient has been dosed in the LIGHTHOUSE study, its phase 1/2 clinical trial evaluating subretinal injection of ATSN-201, for the treatment of X-linked retinoschisis (XLRS). The study is being conducted in male patients ages 6 to 64 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. ATSN-201 leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina, while avoiding the surgical risks of foveal detachment.

“We are excited to be utilizing AAV.SPR in the clinic, as it has the potential to revolutionize the treatment of XLRS, as well as other inherited retinal disorders. Spreading laterally beyond the subretinal injection site, AAV.SPR facilitates the safe delivery of RS1 to photoreceptors in the central retina/fovea. We look forward to advancing the LIGHTHOUSE study and the continued development of our novel gene therapies to reverse or prevent blindness,” Kenji Fujita, MD, chief medical officer of Atsena Therapeutics, said in a news release.

First Patient Dosed in Leber Congenital Amaurosis Phase 1/2 Trial

■ The first patient has been dosed in Opus Genetics’ phase 1/2, first-in-human clinical trial of OPGx-LCA5, according to an announcement made by the company. OPGx-LCA5 is an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene. The open-label, dose-escalation trial is evaluating the subretinal delivery of OPGx-LCA5 in 9 adult patients with LCA5. The objective of the trial is to evaluate the safety and preliminary efficacy of OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

“Dosing our first patient establishes Opus as a clinical-stage company and is a point of progress in our mission to advance first-in-class gene therapies for inherited retinal diseases. Despite the severe retinal dysfunction in patients with LCA5, preclinical data suggest an opportunity for therapeutic intervention, including retinal structural and functional restoration when OPGx-LCA5 was administered prior to peak disease severity,” said Ben Yerxa, PhD, Opus CEO. Once safety in adults has been established and confirmed by the FDA, Opus plans to add a pediatric cohort.

Ocugen Shares Update From Phase 1/2 Trial of OCU400 for Inherited Retinal Diseases

■ Ocugen announced that OCU400 continues to be generally safe and well tolerated across different mutations and dose levels in the phase 1/2 trial of OCU400 for retinitis pigmentosa (RP) associated with NR2E3 and Rhodopsin (RHO) mutations and Leber congenital amaurosis (LCA) with mutations in the CEP290 gene. In the multicenter, open-label study of 18 subjects ranging in age from 18 to 77, subjects received a unilateral subretinal injection of either a low dose, medium dose, or high dose of OCU400. Key efficacy outcomes from 12 subjects demonstrated that 83% experienced stabilization or improvements of BCVA scores; 42% experienced 4-letter improvement, and 33% experienced 7-letter improvement.

“We remain dedicated to our mission of pioneering breakthroughs in biotechnology and believe that OCU400 has the potential to have an impact on the future treatment of patients with RP and LCA,” Shankar Musunuri, MD, chairman, CEO, and cofounder of Ocugen, said in a news release.

Izervay for GA Demonstrates Positive 24-month Results in Phase 3 Study

■ Astellas announced positive 24-month top-line results from the phase 3 GATHER2 clinical trial evaluating the efficacy and safety of Izervay (avacincaptad pegol intravitreal solution), a complement C5 inhibitor for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Top-line results demonstrated that an Izervay monthly dosing regimen met the primary objective to significantly slow GA growth compared to sham at 24 months. Additionally, the treatment effect with an every-other-month dosing regimen showed a similar reduction in the rate of GA growth vs sham. Safety after 24 months of treatment was consistent with previously reported 12-month data. The rate of choroidal neovascularization was 12% in patients treated with Izervay and 9% in the sham group.

“We are excited about these results, which show that Izervay continued to slow the rate of GA growth with a consistent safety profile after 2 years of treatment,” said Dhaval Desai, PharmD, senior vice president and chief development officer of Iveric Bio, an Astellas Company, in a news release.

EyePoint Reports Update on PAVIA and DAVIO 2 Phase 2 Trials

■ EyePoint Pharmaceuticals announced positive interim masked safety data from its phase 2 PAVIA trial evaluating EYP-1901 as a potential 9-month treatment for moderately severe to severe nonproliferative diabetic retinopathy (NPDR) and DAVIO 2 as a potential 6-month sustained delivery maintenance treatment for wet AMD. To date, all treatment arms in the PAVIA trial have reached at least 3 months post-dosing follow-up. EYP-1901 is being developed as an investigational sustained delivery treatment for retinal disease combining a bioerodible formulation of EyePoint’s Durasert delivery technology (Durasert E) with vorolanib, a tyrosine kinase inhibitor.

Jay S. Duker, MD, president and CEO of EyePoint, said in a news release, “We remain very encouraged by the positive masked safety results that EYP-1901 has produced in the 17 patients dosed in the phase 1 DAVIO trial in wet AMD as well as approximately 150 additional patients in our 2 phase 2 clinical trials: DAVIO 2 and PAVIA. These results bolster our confidence in EYP-1901 as a potentially paradigm-shifting treatment for patients who would benefit from a safe, sustained therapeutic option for VEGF-mediated diseases. ... In the 77 patients enrolled in the phase 2 PAVIA trial, the investigators report no drug-related ocular SAEs and no drug-related systemic SAEs, demonstrating EYP-1901’s excellent safety profile in NPDR for the first time.”

Collaboration Focuses on Eye Disease Detection and Vision Loss Prevention

■ ZEISS Medical Technology and Boehringer Ingelheim announced that they have formed a strategic collaboration to develop predictive analytics to enable early detection of eye diseases and treatments to prevent vision loss for people with serious eye diseases. The new partnership will focus on identifying markers of early stages of retinal diseases by leveraging ZEISS’s cloud-connected devices and AI-assisted analysis of massive image data sets. This will provide a basis for clinical studies for development of more personalized and precise treatments of early stages of chronic retinal disease, as well as earlier detection and prediction capabilities to preserve vision through new treatment pathways.

Novartis Discontinues Geographic Atrophy Development Program

■ Novartis announced it has discontinued development of GT005 (PPY988) for treatment of geographic atrophy (GA) secondary to dry AMD. This decision was based on a recommendation from an independent data monitoring committee following an overall benefit risk assessment of available data from the program studies, which concluded futility criteria had been met. In its announcement, the company stressed that no new safety signals were identified, and said that although it has discontinued the studies, the company remains committed to ensuring patients treated with GT005 (PPY988) receive long-term safety follow-up.

In a news release, Novartis stated, “Our disappointment in this outcome is first and foremost for the GA community. We are subsequently communicating with study investigators, health authorities, and relevant bodies and are committed to sharing information from these early studies with the ophthalmology and scientific communities to help inform future development of gene therapies.”

Altris Information Management System Gets FDA 510(k) Clearance

■ Altris announced that the FDA has granted 510(k) clearance to the company’s information management system (IMS), Altris AI, an image and data management platform. The Altris platform stores, organizes, and supports in-depth analysis of optical coherence tomography (OCT) scans and provides secure cloud-stored, web-based access to facilitate OCT scan analysis. The Altris AI platform features a manual annotation and agnostic measurement capability, and features that enable pathology progression tracking and comparison of both eyes, among other things. The 510(k) designation indicates that Altris AI is safe and effective, a milestone that brings Altric closer to commercializing the platform in the United States.

In a news release, Maria Znamenska, MD, PhD, associate professor of ophthalmology and Altris chief medical officer, said that being equipped with the Altris AI platform, “means fewer human errors, early detection of pathologies that can lead to blindness, and better patient outcomes, as a result.”

Optometry Panel Releases Consensus Document for Managing DR

■ LKC Technologies announced the release of Modern Fundamentals of Diabetic Retinopathy Management in Optometry, a consensus document developed by an optometric taskforce comprised of medical optometry educators. The taskforce developed the document with the goal of simplifying and standardizing diabetic retinopathy (DR) assessment and management, to avoid preventable vision loss. The document presents a framework that simplifies DR management so that optometrists can confidently care for the growing population of patients with diabetes. The guidelines are based on 5 pillars: detect, grade, assess risk, manage, and support.

“The taskforce strove to develop recommendations that would be practical to implement. Our goal was to elevate the standard of care with proper management and vigilance, even given the challenges posed by today’s busy healthcare environments,” Paul Chous, MA, OD, FAAO, of Chous Eye Care Associates in Tacoma, WA, said in a news release.

Home OCT Can Improve Neovascular AMD Management for Patients

■ Patient data demonstrate strong heterogeneity both in fluid recurrence and resolution patterns of neovascular AMD with the use of Home OCT (Notal Vision), according to a study presented by Miguel Busquets, MD, at the 2023 American Society of Retina Specialists annual meeting. Analysis of retrospective data on 57 eyes of 54 patients with nAMD revealed significant heterogeneity in recurrence and response fluid volume trajectories for different eyes.

Response to treatment was quantified in a mean decrease rate of 8.3 nL per day, with a mean level of resolution of 91.6% and a mean time of 11 days to complete resolution of fluid. With respect to treatment timing vs fluid characteristics, the cohort treated within 1 week had statistically significantly lower values of mean volume at treatment, mean resolution time, and mean area under the curve for fluid trajectories. RP