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EYLEA 8mg in focus: Key learnings from the pivotal trials

Retina World Congress 2025 will take place May 8-11. As researchers prepare for their, presentation, we are highlighting the upcoming sessions that are scheduled for this meeting, which will be held in Fort Lauderdale, Florida.

Note: The following list of presentations are in alphabetical order by company. Updates will be added to this article as new information becomes available.

The company highlight Phase 2 results for migaldendranib (MGB), Ashvattha’s investigational subcutaneous treatment for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). The presentation, entitled, "Emerging Data om Anti-angiogenic Nanomedicine (Migaldendranib MGB/D-4517.2) for AMD and DME" will be given by Michael Singer, MD on Sunday, May 11, 2025; 11:11 – 11:16 am ET.

Phase 2 clinical data demonstrated that MGB administration lowered production of subretinal fluid reducing or eliminating the need for intravitreal anti-VEGF treatment in both the study eye and fellow eye in wet AMD and DME subjects with bilateral disease. New data indicates that MGB may be the first safe systemic therapy for the treatment of wet AMD and DME without ocular adverse events.

Huma Qamar, Chief Medical Officer at Ocugen will represent Ocugen at Retina World Congress to share a presentation and join retinal surgeons and industry peers during a panel discussion.

Inherited and Rare Retinal Diseases Session
Moderated by Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD on Thursday, May 8, 2025, 10:35 – 11:12 AM ET

Ocular Therapeutix will present information at the meeting in Fort Lauderdale regarding the axitinib intravitreal hydrogel clinical trials, the use of its candidate OTX-TKI for diabetic retinopathy, and the HELIOS clinical trial for diabetic retinopathy.

Axitinib Intravitreal Hydrogel: Update on Clinical Trials

Presented by Carl C. Awh, MD, FASRS, on Friday, May 9, 2025, 4:33 – 4:38 PM ET.

Presented by Dilsher S. Dhoot, MD, FASRS on Saturday, May 10, 2025, 8:44 – 8:49 AM ET

Volumetric Macular Fluid Analysis of the Impact of a Single Axitinib Intravitreal Injection (OTX-TKI) from the HELIOS Clinical Trial for Diabetic Retinopathy

Presented by Carl J. Danzig, MD on Saturday, May 10, 2025, 8:52 – 8:57 AM ET

The company will present further insights from Stage 1 of the Phase 3 DIAMOND program reinforcing the efficacy and safety profile of OCS-01 eye drops for DME will also be shared. OCS-01 is being developed to be the first non-invasive treatment for DME, addressing key unmet needs for earlier treatment intervention and for inadequate responders to the current standard of care.

Ashvattha Therapeutics to Present at ARVO, Retina World Congress, and AOS Annual Meeting. Ashvattha Therapeutics. April 15, 2025. Accessed April 16, 2025. https://www.globenewswire.com/news-release/2025/04/15/3061682/0/en/Ashvattha-Therapeutics-to-Present-at-ARVO-Retina-World-Congress-and-AOS-Annual-Meeting.html

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress. Ocugen Inc. April 29, 2025. Accessed April 30, 2025. https://www.globenewswire.com/news-release/2025/04/29/3070074/0/en/Ocugen-to-Present-on-Modifier-Gene-Therapy-Platform-at-Association-for-Research-in-Vision-and-Ophthalmology-2025-Annual-Meeting-and-Retina-World-Congress.html

Ocular Therapeutix™ to Participate in Upcoming Investor and Scientific Conferences. Ocular Therapeutix. April 29, 2025. Accessed April 29, 2025. https://www.globenewswire.com/news-release/2025/04/29/3070022/0/en/Ocular-Therapeutix-to-Participate-in-Upcoming-Investor-and-Scientific-Conferences.html

Oculis to Present its Innovative Late-Stage Pipeline Candidates at Upcoming Ophthalmology Conferences. Oculis Holding AG. May 1, 2025. Accessed May 1, 2025. https://www.biospace.com/press-releases/oculis-to-present-its-innovative-late-stage-pipeline-candidates-at-upcoming-ophthalmology-conferences

Companies working in the retina space have announced that data from clinical trials will be shared at this year’s meeting in Fort Lauderdale, Florida.

RWC 2025: Companies to share data at meeting in Fort Lauderdale

Ocugen recently announced new members to its Retina Scientific Advisory Board (SAB) and Executive Leadership Team.

 These updates support Ocugen in its efforts to pursue its goal of three biologics license applications (BLA) in the next 3 years.

“As our novel modifier gene therapy programs continue to progress and demonstrate positive data in the clinic, it is now more important than ever to solidify and optimized Retina SAB reflecting the absolute best guidance in this space as we move closer to commercialization,” Shankar Musunuri, PhD, MBA, chairman, CEO, and cofounder of Ocugen, said in a press release. “Additionally, it’s imperative that we ensure our leadership team includes top talent to most effectively execute our corporate strategy.”

Three new members of the Retina SAB are retina surgeons Jeffrey S. Heier, MD, Peter K. Kaiser, MD, and Arshad N. Khanani, MA, MA, FASRS. Drs. Heier, Kaiser, and Khanani join SAB chair Lejla Vajzovic, MD, FASRS, and existing SAB members David S. Boyer, MD, and Carl D. Regillo, MD, FACS.

One of the leading retinal clinical researchers in the US, Heier, is the Director of the Vitroretinal Service, and Director of Retina Research at Ophthalmic Consultants of Boston. Heier’s research focuses on new treatments in exudative and non-exudative macular degeneration, diabetic macular edema, venous occlusive disease, vitreoretinal surgical techniques and instrumentation, and diagnostic imaging of the retina.

Kaiser comes to the SAB with a resume of authoring seven textbooks, 30 book chapters, and more than 400 peer-reviewed manuscripts. Associate editor of 

, Kaiser is also the Chaney Family Endowed Chair in Ophthalmology Research at the Cleveland Clinic’s Cole Eye Institute, and professor of ophthalmology at Case Western Reserve School of Medicine. Additionally, he is the director of the Center for Ocular Research and Evaluation (CORE).

Managing partner, director of clinical research, and director of Fellowship at Sierra Eye Associates, and clinical professor at the University of Nevada, Reno School of Medicine, Khanani has been recognized among the top 10 researchers globally on The Ophthalmologist Power List 2025. In 2021, Khanani founded the Clinical Trials at the Summit meeting and has received numerous rewards including the Macular Society’s Lawrence J. Singerman Medal in 2025 and the American Society of Retina Specialists Presidential Award.

In addition to the new SAB members, Ocugen has made new leadership appointments, including a newly created Chief Development Officer (CDO) position.

Joining Ocugen in the new CDO role, Vijay Tammara, PhD, brings more than 32 years of global regulatory leadership experience with knowledge in biotechnology, biosimilars, 505(b)(2), and complex regulatory submissions. He has made contributions toward the approval of 9 marketing authorizations (MAs), biologics license applications (BLAs) and new drug applications (NDAs), 2 biosimilar MAs in emerging markets, 3 abbreviated new drug applications, 12 orphan drug designations, 7 qualified infections drug product designations, and filed over 62 investigational NDAs with clearance in the first review cycle with no clinical holds.

 Tammara has held senior advisory and leadership roles at the FDA and life sciences companies including Sanofi, Wyeth/Pfizer, and Merck, as well as led regulatory strategy and operations across the US, EU, Latin America and Asia.

Following the retirement of Mike Shine, Abhi Gupta, MBA, has been named Executive Vice President, Commercial and Business Development, bringing more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry. Gupta has led commercialization planning for rAAV gene therapies and played a key role in Pfizer’s $5.5B gene therapy portfolio across neuromuscular, hematologic, and cardiovascular indications. He previously served as SVP and Head of Cell and Gene Therapies and Syneos Health, as well as held leadership roles at Pfizer, Regeneron, and Johnson & Johnson.

“Ocugen is honored to partner with this distinguished group of advisors and delighted to welcome Dr. Tammara and Mr. Gupta as the Company sharpens its patient-centric focus to potentially deliver paradigm-changing gene therapies,” the company said in a press release.

Ocugen. Ocugen, Inc. Announces New Appointments to Retina Scientific Advisory Board & Executive Leadership Team. GlobeNewswire News Room. Published July 21, 2025. Accessed July 21, 2025. 

https://www.globenewswire.com/news-release/2025/07/21/3118634/0/en/Ocugen-Inc-Announces-New-Appointments-to-Retina-Scientific-Advisory-Board-Executive-Leadership-Team.html

Ocugen announces new SAB members and leadership changes

For decades, I had to deliver the difficult message to patients with inherited retinal diseases (IRDs) that there was no treatment available for them. The cruel reality was that their condition would continue to cause irreversible vision loss.

 In 2017, voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) was the first in human gene therapy to receive US approval for Leber congenital amaurosis (LCA) due to biallelic 

mutation. This ushered in a new era of gene therapy across medicine. LCA is one of hundreds of genetically characterized IRDs. The population with LCA is limited; about 1000 to 2000 individuals in the US are affected.

 Although I still communicate to patients with other IRDs that no treatment is available for their conditions, I now can say one may be on the horizon. Optogenetics offers a promising mutation-agnostic approach to gene therapy, helping us deliver hope to more patients.

Degenerative retinal disease progressively damages the photoreceptors responsible for capturing light and transmitting visual signals to the brain. Generally—and because of associated and progressive vision loss—IRDs are considered a degenerative disease. 

Studies show that IRDs account for about 20% to 25% of the world’s blindness in the working-age population.

 The most common IRD is retinitis pigmentosa (RP).

RP is generally characterized by retinal pigmentary changes and a gradual loss of night and peripheral vision. Black peripheral retinal “bone spicules” are a classic clinical feature, but pigmentary changes may be subtle to absent. Patients often progress to complete blindness.

 mutation—is another type of IRD clinically characterized by yellow flecks in the macula and retinal midperiphery, progressing to macular “beaten bronze” atrophic changes, leading to central vision loss,

 and severely impacting daily tasks like reading and recognizing faces.

Despite the prevalence and impact of RP and Stargardt disease, treatment options remain limited. This is especially true for individuals with advanced vision loss.

Optogenetics uses gene therapy strategies to introduce light-sensitive transmembrane proteins called opsins into the retina. Unlike gene replacement therapies targeting specific genetic mutations, optogenetics is both mutation- and disease-agnostic. Optogenetic strategies use remaining retinal cells (eg, bipolar and ganglion cells) to regain light sensitivity and restore a degree of functional vision,

 opening new possibilities for individuals with severe vision loss and potentially transforming the management of conditions such as RP and Stargardt disease.

The field of optogenetics is evolving. Current therapies in development include, in order of most advanced clinical status to preclinical therapies, MCO-010 (Nanoscope Therapeutics), RST-001 (RetroSense/Allergan), GS030 (GenSight Biologics), BS01 (Bionic Sight), and RTx-015 (Ray Therapeutics). These therapies differ with respect to their cellular targets, kinetics, and spectral and light sensitivity (

The most advanced optogenetic in development is MCO-010. Multicharacteristic opsin (MCO) is a transgene for a high-performing opsin that sensitizes bipolar cells to light.

 It is sensitive across the visible light spectrum, has rapid kinetics for tracking movement, and responds to ambient light levels. It is delivered by a proprietary adeno-associated virus (AAV2) in a single office-based intravitreal injection and, unlike some other treatments in development, does not require a light-intensifying device to mimic photoreceptor function (

). In a phase 1/2a clinical study, intravitreal treatment with MCO-010 has demonstrated restoration of vision in patients with advanced RP.

In RESTORE (NCT04945772), a randomized, multicenter, sham-controlled phase 2 clinical trial of 27 patients with severe vision loss from advanced RP, no better than logMAR +1.9 (20/1600 Snellen equivalent, counting fingers), MCO-010 demonstrated a clinically meaningful improvement in vision. Patients received either a single intravitreal injection (IVT) of a high- or low-dose MCO-010 or sham IVT. At the 52-week primary end point, approximately 40% of patients who received MCO-010 experienced a mean change in best-corrected visual acuity (BCVA) improvement from baseline of at least +0.3 logMAR, equivalent to 3 lines of Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity improvement, which was statistically significant in both the high- and low-dose MCO-010 groups compared with the sham (

Vision gains were durable, with sustained results at the 76-week key secondary end point. Vision improvement relative to baseline continued to be observed through 2.5 years, including about a 5× greater BCVA area under the curve at the end of 126 weeks in both groups treated with MCO-010 vs those treated with sham. The difference was statistically significant. MCO-010 was well tolerated, and there were no serious or severe adverse events, including severe inflammation, vasculitis, and hypotony. Although inflammation was seen in some patients, it was effectively treated with topical steroids. Based on these results, Nanoscope is preparing a biologics licensing application for marketing approval through the FDA. Additionally, a phase 4 RP postapproval study will begin later in 2025.

What makes MCO-010 particularly exciting is its visible light sensitivity and fast on-and-off kinetics. Unlike other optogenetic therapies that require external laser-stimulation goggles or a light amplification device, MCO-010 works with ambient light. This significantly enhances the therapy’s practicality for patients in their everyday lives.

Although the focus has been on RP, the use of MCO-010 for Stargardt disease is also under investigation. Early clinical data in this population show promising improvements in visual acuity and functional vision. In the open-label, phase 2 STARLIGHT study (NCT05417126) enrolling 6 individuals with severe vision loss (visual acuity no better than 20/800) from advanced Stargardt disease, MCO-010 continued to show a positive safety profile with improvements in visual function across multiple parameters such as visual acuity, visual field, and patient-reported outcome measures. The findings in STARLIGHT support continued development. A randomized, controlled phase 3 registrational study will begin dosing this year. MCO-020 (Nanoscope Therapeutics) is under an investigational new drug (IND)–ready phase for geographic atrophy (GA).

Companies like Ray Therapeutics and GenSight Biologics are also advancing optogenetic programs that target retinal ganglion cells. A phase 1 study (NCT06460844) of RTx-015 (Ray Therapeutics) for RP is under way to evaluate the safety and preliminary efficacy of a single uniocular intravitreal injection. Up to 3 dose cohorts are planned. RTx-021 (Ray Therapeutics) is under an IND-enabling study phase for GA and Stargardt disease. BS01 (Bionic Sight) and GS030 (GenSight Biologics) combine optogenetic therapy with goggles enabling laser stimulation of the transduced ganglion cells. GenSight has initiated a dose-escalation study to evaluate the safety and tolerability of GS030 in patients with RP and plans to investigate the potential for GS030 to treat GA thereafter.

Each optogenetics approach has unique advantages. As the field progresses, we will better understand how best to tailor these therapies for different retinal conditions.

Paradigm shift and potential for a brighter future

Optogenetics represents a potential paradigm shift for retina specialists. Historically, our role was limited to monitoring disease progression and offering low-vision support for patients with IRDs. An approved optogenetic therapy may deliver an opportunity to restore some functional vision in some patients with significant vision loss.

From a clinical perspective—and if an optogenetic strategy is approved—we will need to study and understand who the most suitable candidates for optogenetic therapy will be and who will most likely benefit. Additionally, managing patient expectations and providing comprehensive counseling on potential outcomes will be essential.

Integrating potential optogenetic treatments into clinical practice will require ongoing education and collaboration within the ophthalmology community. Retina specialists will be key in posttreatment care, monitoring patients for efficacy and safety and collaborating with low-vision specialists to maximize functional outcomes.

The prospect of offering mutation-agnostic vision restoration to a population that has long been without options is exciting. For the countless patients with RP, Stargardt disease, and other IRDs without current available treatment, we may be able to provide hope and brighter days based on randomized controlled clinical trial evidence.

Retinal degeneration. Chapter: Stem cell therapy and retinal regeneration. Science Direct. Accessed March 18, 2025. https://www.sciencedirect.com/topics/neuroscience/retinal-degeneration

Hu ML, Edwards TL, O’Hare F, et al. Gene therapy for inherited retinal diseases: progress and possibilities. 

. 2021;104(4):444-454. doi:10.1080/08164622.2021.1880863

Crewe JM, Morlet N, Morgan WH, et al. Mortality and hospital morbidity of working-age blind. 

. 2013;97(12):1579-1585. doi:10.1136/bjophthalmol-2013-303993

Liew G, Michaelides M, Bunce C. A comparison of the causes of blindness certifications in England and Wales in working age adults (16-64 years), 1999-2000 with 2009-2010. 

 2014;4(2):e004015. doi:10.1136/bmjopen-2013-004015

Inherited retinal dystrophies including retinitis pigmentosa. Royal National Institute of Blind People. Reviewed October 12, 2022. Accessed March 18, 2025. https://www.rnib.org.uk/your-eyes/eye-conditions-az/retinitis-pigmentosa

Diagnosis, clinical trials and treatments for inherited retinal diseases. Mayo Clinic. October 14, 2023. Accessed March 18, 2025. https://www.mayoclinic.org/medical-professionals/ophthalmology/news/diagnosis-clinical-trials-and-treatments-for-inherited-retinal-diseases/mac-20556115

Stargardt disease. National Eye Institute. Updated December 4, 2024. Accessed March 18, 2025. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/stargardt-disease

Stargardt disease. Royal National Institute of Blind People. Updated February 1, 2024. Accessed March 18, 2025. https://www.rnib.org.uk/your-eyes/eye-conditions-az/stargardt-disease

Wood EH, Kreymerman A, Kowal T, et al. Cellular and subcellular optogenetic approaches towards neuroprotection and vision restoration. 

. 2023;96:101153. doi:10.1016/j.preteyeres.2022.101153

Wright W, Gajjeraman S, Batabyal S, et al. Restoring vision in mice with retinal degeneration using multicharacteristic opsin. 

. 2017;4(4):041505. doi:10.1117/1.NPh.4.4.041505

Batabyal S, Gajjeraman S, Pradhan S, Bhattacharya S, Wright W, Mohanty S. Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice. 

2021;28(3-4):162-176. doi:10.1038/s41434-020-00200-2

Batabyal S, Kim S, Carlson M, et al. Multi-characteristic opsin therapy to functionalize retina, attenuate retinal degeneration, and restore vision in mouse models of retinitis pigmentosa. 

. 2024;13(10):25. doi:10.1167/tvst.13.10.25

Mahapatra S, Batabyal S,et al. A synthetic opsin restores vision in patients with severe retinal degeneration. 

. 2025;33(5):2279-2290. doi:10.1016/j.ymthe.2025.03.031

Allen C. Ho, MD, FACS, FASRS, is director of retina research, Mid Atlantic Retina, Wills Eye Hospital, and professor of ophthalmology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia. Ho is a consultant and/or receives grant funding from Adverum Biotechnologies, Apellis Pharmaceuticals, AsclepiX Therapeutics, Astellas Pharma, Clearside Biomedical, Genentech/Roche, Gyroscope Therapeutics, Kodiak Sciences, Lineage, Nanoscope Therapeutics, and Regenxbio.

A mutation-agnostic gene therapy approach offers new possibilities for patients with advanced retinal disease.

clinical

Optogenetics for retinal disease

An Indonesian study of the characteristics of the retinal vasculature found that the retinal vessel tortuosity (VT) was significantly higher and the fractal dimension (FD) was significantly lower in patients with 

 Lead author Fariztah Sukainah Nur Fathimah, MD, from the Department of Ophthalmology, Faculty of Medicine, Universitas Airlangga, Surabaya, East Java, Indonesia, and colleagues reported their findings in the 

International Journal of Retina and Vitreous.

Because diabetes is so rampant worldwide, the push is on to be able to identify DR earlier than what is currently possible, with the goal of saving vision.

The investigators commented, “DR is a common microvascular condition that frequently leads to blindness in both industrialized and poor countries. The current method of screening for DR involves visually examining fundus pictures to clinically assess the presence of DR. However, there is an urgent requirement for a screening method that can detect DR at an early stage, before it becomes clinically apparent.”

They explained that retinal VT is an early sign of vascular changes in retinopathy, and low FD values indicate less frequent blood vessel branching patterns associated with the risk of cardiovascular mortality and advanced stages of DR.

Fathimah and colleagues investigated retrospectively the differences in retinal VT and FD between patients with and without DR. They analyzed the medical records and optical coherence tomography (OCT)-A images of patients with and without DR. A vitreoretinal specialist graded the disease severity according to the International Clinical Diabetic Retinopathy and Diabetic Macular Edema Severity Scales. The patients were divided into 5 groups: as no DR, mild nonproliferative DR (NPDR), moderate NPDR, severe NPDR, and proliferative DR (PDR).

The study analyzed 96 patients (161 eyes) who were a mean age of 52.7 ± 9.9 years. The investigators found that the VT was significantly higher in all DR groups (

 < 0.05): mild NPDR (β = +0.0621), moderate NPDR (β = +0.0412), severe NPDR (β = +0.0441), and proliferative DR (β = +0.0404). The FD of the superficial capillary plexus (SCP) did not differ significantly among the groups; the FD of the deep capillary plexus (DCP) was significantly lower compared with the groups with no PDR: moderate NPDR (β = -0.0131), severe NPDR (β = -0.0316), and PDR (β = -0.0326).

“Compared to the group with no DR, this study discovered a substantial rise in VT in patients with mild, moderate, and severe NPDR as well as PDR. This suggests that microvascular changes advance with the severity of the DR. Furthermore, a significant decrease in FD of the DCP was found in moderate NPDR, severe NPDR, and PDR. These findings underscore the necessity of evaluating specific retinal layers to gain deeper insights into the vascular changes linked to DR. If validated in larger studies, retinal vascular geometry could become a dual-purpose screening tool for both diabetic eye disease and systemic cardiovascular risk assessment,” Fathimah and colleagues concluded.

Fathimah FSN, Widjaja SA, Sasono W, et al. Retinal vessel tortuosity and fractal dimension in diabetic retinopathy. 

https://doi.org/10.1186/s40942-025-00688-z

Quinn N, Jenkins A, Ryan C, Januszewski A, Peto T, Brazionis L. Imaging the eye and its relevance to diabetes care. 

Sayin N. Ocular complications of diabetes mellitus. 

 [homepage on the Internet]. 2015;6(1):92. Available from: 

http://www.wjgnet.com/1948-9358/full/v6/i1/92.htm

Saghiri MA, Suscha A, Wang S, Saghiri AM, Sorenson CM, Sheibani N. Noninvasive temporal detection of early retinal vascular changes during diabetes. 

Li M, Wang G, Xia H, Feng Z, Xiao P, Yuan J. Retinal vascular geometry detection as a biomarker in diabetes mellitus. 

Frizziero L, Midena G, Longhin E, et al. Early retinal changes by OCT angiography and multifocal electroretinography in diabetes. 

Agra CLDM, Lira RPC, Pinheiro FG, Sá LHSE, Filho VTFB. Optical coherence tomography angiography: microvascular alterations in diabetic eyes without diabetic retinopathy. 

Ari Widjaja S, Mieler WF, Sasono W et al. Retinal neurovascular alteration in type 2 diabetes with renal impairment in association with systemic arterial stiffness. 

Early diabetic retinopathy detection may be found in biomarker changes

Katherine Talcott, MD, of Cleveland Clinic in Cleveland, Ohio, presented results of the phase 2 DAVIO 2 trial (NCT05381948) of EYP-1901 (vorolanib intravitreal insert; a/k/a DURAVYU) for 

wet age-related macular degeneration (AMD)

Association for Research in Vision and Ophthalmology (ARVO)

Wet AMD has a high treatment burden, typically requiring frequent anti-VEGF injections for optimal outcomes. However, real-world clinical studies show that most patients are not receiving injections at a rate that maintains vision over time, and a significant proportion of patients discontinue treatment altogether.

Recently-approved therapies aim to reduce treatment burden in wet AMD; however, the sustained-delivery device Susvimo (port delivery system with ranibizumab; Genentech, Inc) requires surgery for implantation,

 and other recently-approved products (Vabysmo, Genentech, Inc; Eylea-HD®, Regeneron Pharmaceuticals) have achieved incremental improvements.

Talcott’s presentation focused on phase 2 clinical trial results of a therapeutic in development: EYP-1901, which aims to reduce treatment burden for wet AMD. This candidate is a bioerodible, sustained-release intravitreal insert delivering the potent and selective tyrosine kinase inhibitor (TKI) vorolanib over at least 6 months. TKIs, including vorolanib, inhibit the VEGF receptor intracellularly, thereby inhibiting angiogenesis and exudation.

drug delivery technology, a next-generation, bioerodible version of the Durasert intravitreal insert used in 4 FDA-approved intraocular indications to date.

 The insert, composed of 94% vorolanib/6% matrix, is designed to deliver a consistent daily dose of vorolanib for at least 6 months starting from the day of administration, with all drug released before matrix bioerosion to ensure no uncontrolled drug release. It does not require cold storage.

DAVIO 2, the only phase 2 and largest completed clinical trial of an intravitreal TKI to date, assessed efficacy and safety of a single injection of EYP-1901 compared with on-label aflibercept 2.0 mg every 8 weeks (q8W) in patients with previously treated wet AMD.

In total, DAVIO 2 enrolled 161 patients who received 3 monthly loading doses of aflibercept 2.0 mg, followed by either sham and aflibercept 2.0 mg Q8W thereafter; a single injection of EYP-1901 2 mg; or a single injection of EYP-1901 3 mg. Starting at week 12, all patients were assessed every 4 weeks for supplemental aflibercept 2.0 mg injections using prespecified best-corrected visual acuity (BCVA) and anatomic criteria; supplementation per investigator discretion was permitted. The primary end point was BCVA change from day 1 to week 28/32, averaged.

Patients in DAVIO 2 had significant treatment experience prior to the trial, averaging approximately 2 years since wet AMD diagnosis with an annualized mean of 10 anti-VEGF injections in the year before enrollment. On average, patients began DAVIO 2 with baseline vision ~73–75 ETDRS letter score (~20/32 Snellen equivalent) and CST ~263–266 μm.

DAVIO 2 met its primary end point, with both EYP-1901 2 mg and EYP-1901 3 mg providing similar vision outcomes as aflibercept Q8W at months 7/8 (averaged), as well as stable CST. These vision and CST outcomes were maintained throughout the 1-year trial in all arms.

Notably, patients in the EYP-1901 arms experienced substantial reductions in treatment burden, with 63% of patients receiving no supplemental aflibercept injections up to month 8, the expected minimum duration of EYP-1901. The median time to first supplemental aflibercept injection was 37/41 weeks (~9-10 months) after EYP-1901 2 mg/3 mg administration, with EYP-1901-treated eyes receiving a median of 1.1 supplemental injections through the end of the 56-week trial. These results reflect a treatment burden reduction of ≥80% compared with the patients’ own pretrial treatment frequency and ≈70% vs aflibercept Q8W-treated eyes through 1 year.

EYP-1901 had a favorable safety profile in DAVIO 2, with no EYP-1901-related ocular or systemic serious adverse events, no insert migration into the anterior chamber, no cases of retinal occlusive vasculitis, and no discontinuations related to this treatment.

Overall, EYP-1901 demonstrated noninferiority to aflibercept with stable BCVA, a favorable safety profile, and substantially reduced treatment burden in DAVIO 2.

Following the positive results from DAVIO 2, pivotal phase 3 clinical trials LUGANO (NCT06668064) and LUCIA (NCT06683742) began in late 2024 and are currently enrolling. As presented in a separate poster by Nimesh Patel, MD, of Massachusetts Eye and Ear and Harvard Medical School in Boston, MA, LUGANO and LUCIA aim to demonstrate that EYP-1901, when administered every 6 months, achieves similar visual outcomes to on-label aflibercept while reducing treatment burden in patients with wet AMD.

These pivotal, parallel, global trials each aim to enroll ~400 patients with active AMD, including patients with treatment-naive or previously-treated wet AMD. Building upon the findings of DAVIO 2, all patients will receive active treatment; following 3 monthly aflibercept 2.0 mg injections, patients are randomly assigned to receive either EYP-1901 2.7 mg with redosing every 6 months or on-label aflibercept 2.0 mg. Patients will be assessed for supplemental aflibercept injections per prespecified criteria at each visit. The primary end point of these noninferiority trials is BCVA change from baseline at weeks 52/56 blended. Secondary end points include safety, anatomic stability, reduction in treatment burden, and proportion of eyes supplement-free.

EYP-1901 (DURAVYU) is designed to continuously provide VEGF receptor inhibition via the TKI vorolanib from the day of administration through at least 6 months. The phase 2 DAVIO 2 trial demonstrated the potential to substantially reduce treatment burden in wet AMD while maintaining vision. Ongoing phase 3 LUGANO and LUCIA trials, designed based on learnings from DAVIO 2, will further assess the potential of EYP-1901 as a novel therapeutic for wet AMD.

DURAVYU has been conditionally accepted by the FDA as the proprietary name for EYP-1901. DURAVYU is an investigational product; it has not been approved by the FDA. FDA approval and the timeline for potential approval are uncertain.

Talcott is a consultant for EyePoint Pharmaceuticals, Inc.

1. Talcott K, Ribeiro R. Assessment of treatment burden in wet age-related macular degeneration treated with EYP-1901 (vorolanib intravitreal insert) versus aflibercept: DAVIO 2 trial results. Presented at: Association of Research in Vision and Ophthalmology (ARVO) annual meeting; 2025; Salt Lake City, UT.

2. Ciulla TA, Hussain RM, Taraborelli D, Pollack JS, Williams DF. Longer-term anti-VEGF therapy outcomes in neovascular age-related macular degeneration, diabetic macular edema, and vein occlusion-related macular edema: Clinical outcomes in 130 247 eyes. 

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A hope for reducing treatment burden for wet age-related macular degeneration with a sustained-release vorolanib intravitreal insert

Professor Varun Chaudhary, McMaster University, Canada

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