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In her keynote address, Prof Schmidt-Erfurth focused on new artificial intelligence tools and their usage in addressing age-related macular degeneration.

Ursula Schmidt-Erfurth, MD, discusses her keynote EURETINA Lecture

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

EURETINA 2025: Aleksandra Rachitskaya and Danilo Ianetta on the Vit-Buckle Symposium

The Vit-Buckle Symposium will be a part of the at EURETINA annual meeting in Paris, France again this year. To discuss this symposium, Aleksandra Rachitskaya, MD, FASRS, from Cleveland Clinic Cole Eye Institute and Danilo Iannetta, MD, PhD, from Rome Sapienza University spoke with the Eye Care Network. 

The symposium was designed to showcase challenging surgical cases from presenters worldwide, with a significant portion dedicated to open and honest discussions. Participants believed that learning was most effective when sharing complex medical scenarios rather than just highlighting successes. Iannetta described the session as highly dynamic and interactive, structured around 2-minute surgical video presentations followed by extensive discussions. The format encouraged surgeons to learn from each other's experiences and perspectives. 

The Vit-Buckle Society distinguished itself by welcoming members at all training levels, including medical students, residents, and fellows. Members gained access to extensive educational resources, including webinars in surgical and medical retina, and opportunities to participate in international meetings. 

Iannetta shared his long-standing connection with the society, having first engaged with it in 2014 during a year-long stay in New York. He observed the organization's consistent growth and commitment to creating a multicultural, international scientific community. 

The society will host its annual meeting for April 9-11, 2026, in Las Vegas, Nevada, USA, and is inviting abstracts from participants worldwide. The members emphasized their commitment to supporting young professionals, offering free membership for trainees and opportunities for international participants to present their work. 

Key highlights included the society's presence at multiple international conferences, including recent appearances at the Pan-American meeting and an upcoming event in Asia. Both Rachitskaya and Iannetta stressed the importance of collaboration, knowledge sharing, and supporting emerging medical professionals. The symposium represented more than just a medical conference; it was a platform for global medical professionals to connect, learn, and grow together. By creating an inclusive, interactive environment, the Vit-Buckle Society aimed to advance medical knowledge and support the next generation of eye care specialists.

clinical

EURETINA 2025: Aleksandra Rachitskaya and Danilo Iannetta on the Vit-Buckle Symposium

The European Medicines Agency (EMA) granted Orphan designations to MCO-010 (sonpiretigene isteparvovec) across 5 categories of retinal dystrophies. The US Food and Drug Administration (FDA) also granted RMAT designation for MCO-010 in Stargardt disease (SD). The EMA and FDA actions suggest regulatory agency recognition of the potential of MCO-010 to address significant unmet needs.

The EMA Orphan designations cover several categories. These include nonsyndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies. This has the potential to provide disease-agnostic regulatory paths forward for dozens of retinal conditions. In the European Union, EMA Orphan status provides opportunities for protocol assistance and up to 10 years of market exclusivity following approval.

The new FDA RMAT designation for SD builds on prior Orphan Drug and Fast Track designations for both retinitis pigmentosa (RP) and SD. In the United States, Orphan, Fast Track, and RMAT designations provide opportunities for accelerated development and review, flexibility in trial design, and 7 years of market exclusivity upon approval.

Regarding this designation, Sulagna Bhattacharya, CEO of Nanoscope, ins quoted in the company’s press release

, saying, “Securing RMAT designation for Stargardt disease in addition to our prior FDA designations for SD and RP is a major validation for our therapies that warrant expedited development and review. Combined with 5 EMA Orphan designations, these achievements highlight the global momentum behind our MCO platform as a potential vision-restoring therapy for patients with few or no treatment options.”

MCO (multicharacteristic opsin) is a one-time, in-office, intravitreal disease-agnostic therapy platform that is designed to restore vision in patients with photoreceptor degeneration, including RP, SD, and geographic atrophy (GA). This therapy works by activating highly dense bipolar retinal cells to become light sensitive and employing the remaining visual circuitry following photoreceptor death. Notably, MCO does not require genetic testing, surgical intervention, or repeat dosing, which may enable broad patient applicability within existing retina office workflows.

Nanoscope Therapeutics saw positive results in the RESTORE phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), a rolling BLA submission to the FDA has been initiated.

Promising results have also been seen in the STARLIGHT phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126), and the company plans to initiate a Phase 3 registrational trial in 2025.

A phase 2 program for MCO in GA is expected to start by the end of 2025.

Other IND-ready programs include Leber congenital amaurosis (LCA).

Nanoscope Strengthens Global Regulatory Pathways for MCO-010 with FDA RMAT and Five EMA Orphan Designations. Nanoscope Therapeutics. News Release. September 2, 2025. Accessed September 2, 2025. https://www.prnewswire.com/news-releases/nanoscope-strengthens-global-regulatory-pathways-for-mco-010-with-fda-rmat-and-five-ema-orphan-designations-302543344.html

Nanoscope Therapeutics. Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa. Prnewswire.com. Published July 14, 2025. Accessed July 14, 2025. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-initiates-rolling-submission-of-biologics-license-application-to-fda-for-mco-010-the-first-gene-agnostic-therapy-to-treat-retinitis-pigmentosa-302503892.html

MCO-010 receives Orphan and RMAT designations, highlighting its potential as a groundbreaking therapy for various retinal dystrophies.

Canonical

EMA grants Orphan designations to MCO-010, FDA grants RMAT in Stargardt disease

Outlook Therapeutics has requested a Type A meeting with the US Food and Drug Administration following the agency issuing a complete response letter (CRL) to its biologics license application (BLA) resubmission for ONS-5010 (bevacizumab-vikg) for the treatment of 

wet age-related macular degeneration (AMD)

Bevacizumab-vikg is a recombinant humanized monoclonal antibody (mAb) that selectively binds with high affinity to all isoforms of human VEGF and neutralizes VEGF’s biologic activity through a steric blocking of the binding of VEGF to its receptors Flt-1 (VEGFR-1) and KDR (VEGFR-2) on the surface of endothelial cells.

According to Outlook, the Type A meeting seeks to clarify details of the confirmatory evidence required by the FDA in the CRL issued August 27, 2025. In the CRL, the FDA advised that, because ONS-5010 did not meet the primary efficacy end point in NORSE EIGHT, it is recommended that confirmatory evidence of efficacy be submitted to support the application for ONS-5010. In the CRL, the FDA did acknowledge the NORSE TWO pivotal trial met its safety and efficacy endpoints.

The company previously resubmitted the BLA in February 2025, and the FDA accepted the resubmission in April 2025.

 The BLA resubmission was based on the efficacy and safety demonstrated in the NORSE EIGHT clinical trial, as well as additional CMC information requested by the FDA.

Bob Jahr, CEO of Outlook Therapeutics, commented on the request, saying, “We believe this Type A meeting will provide us with additional clarity on the FDA’s requirements to remedy the deficiency identified in the CRL. The Outlook Therapeutics team remains dedicated to our mission to provide patients in the US with a safe and effective alternative to compounded bevacizumab, which has been used off-label as a first-line treatment for wet-AMD for years despite concerns around potency, sterility, consistency, and regulatory oversight associated with the use of compounded products.”

This is the second Type A meeting requested by Outlook regarding ONS-5010 and the second CRL issued by the FDA for ONS-5010.

 The first Type A meeting request in September 2023 followed the first CRL issued on August 29, 2023. For the 2023 submission, it was noted that the FDA “could not approve the BLA during the review cycle due to several CMC issues, open observations from preapproval manufacturing inspections, and a lack of substantial evidence.”

After the CRL in 2023, Outlook Therapeutics stock underwent a 1-for-20 reverse stock split as “part of the Company’s plan to regain compliance with the minimum bid price ($4 per share) requirement for continued listing on the Nasdaq Capital Market.”

In March 2024, the European Medicines Agency (EMA) issued a positive Committee for Medicinal Products for Human Use (CHMP) opinion on ONS-5010. In June 2025, LYTENAVA (bevacizumab gamma) became commercially available in Germany and the UK for the treatment of wet AMD.

Outlook Therapeutics Requests Type A Meeting with FDA. Published September 2, 2025. Accessed September 3, 2025. 

https://ir.outlooktherapeutics.com/news-releases/news-release-details/outlook-therapeutics-requests-type-meeting-fda

Harp MD. FDA issues complete response letter to Outlook Therapeutics for resubmitted BLA for ONS-5010. Published August 28, 2025. Accessed September 3, 2025. 

https://www.ophthalmologytimes.com/view/fda-issues-complete-response-letter-to-outlook-therapeutics-for-resubmitted-bla-for-ons-5010

Harp MD. FDA accepts biologics license application from Outlook Therapeutics for ONS-5010 for the treatment of wet AMD. Published April 9, 2025. Accessed August 28, 2025. 

https://www.ophthalmologytimes.com/view/fda-accepts-biologics-license-application-from-outlook-therapeutics-for-ons-5010-for-the-treatment-of-wet-amd

Hutton D. FDA issues CRL for Outlook Therapeutics’ ONS-5010. Published August 30, 2025. Accessed August 28, 2025. 

https://www.ophthalmologytimes.com/view/fda-issues-crl-for-outlook-therapeutics-ons-5010

Harp MD. Outlook Therapeutics stock undergoes 1-for-20 reverse stock split. Published March 14, 2024. Accessed August 28, 2025. 

https://www.ophthalmologytimes.com/view/outlook-therapeutics-stock-undergoes-1-for-20-reverse-stock-split

Outlook Therapeutics seeks FDA clarity on ONS-5010 after a complete response letter, aiming to address efficacy concerns for wet AMD treatment.

Outlook Therapeutics requests Type A meeting with FDA following second CRL for ONS-5010

Now is an exciting time to be a retina specialist. A wave of innovations and regulatory approvals has emerged in the past year that has the potential to change our practice patterns. Although it can be difficult to keep up with what often seems like an endless font of presentations and publications, I would like to highlight a few recent developments that I think are particularly noteworthy.

Aflibercet-abzv: An established therapy in a new package

In August 2024, aflibercept-abzv (Enzeevu; Sandoz) was approved to treat 

neovascular age-related macular degeneration (nAMD)

 As a biosimilar, aflibercept-abzv is considered interchangeable with its reference product, aflibercept (Eylea; Regeneron Pharmaceuticals, Inc), which is reassuring to those of us who have been prescribing aflibercept 2 mg for our patients for years. This similarity was confirmed by the results of the phase 3 Mylight study (

), which found that aflibercept-abzv had comparable efficacy, safety, and pharmacokinetics to aflibercept 2 mg in patients with nAMD after 52 weeks.

A notable advantage of aflibercept-abzv is that it will come in a prefilled syringe. From a workflow perspective, this should help optimize clinic efficiency, especially considering the high number of treatments many practices perform daily. From a safety perspective, using a prefilled syringe may pose a lower risk of endophthalmitis, with fewer failure points during which contamination could occur. Ultimately, it is great to have another option to integrate into our portfolio of care. Hopefully, the approval of this and other biosimilars will continue to expand access to the best possible therapies for a greater number of patients.

Photobiomodulation: A new approach for dry AMD

Another exciting development in retina has been the approval of the ValedaLight Delivery System (LumiThera) for the treatment of dry AMD.

 is estimated to make up as many as 90% of all AMD cases,

 and a significant proportion of these patients can go on to have vision loss from progression to 

 Despite the enormity of this problem, historically, there has been no approved therapy for preventing the progression of intermediate dry AMD beyond lifestyle considerations, such as smoking cessation and a good diet, and the use of the AREDS/AREDS2 study antioxidant vitamin and mineral supplements. The latter, although accessible and low-risk, has demonstrated only a moderate benefit in reducing the risk of progression by approximately 25%.

The approval of the Valeda Light Delivery System was based on the results of the randomized, controlled LIGHTSITE III trial (

), wherein patients with dry AMD were given either multiwavelength photobiomodulation or sham treatments over 24 months. We currently have the 13-month results of this trial, which were promising. In LIGHTSIDE III, patients who received photobiomodulation experienced visual acuity gains of 5.4 letters compared with 3.0 letters in patients who received sham. Even more promising, patients who received photobiomodulation had significantly decreased odds of going on to develop GA.

Beyond the promising clinical trial findings, photobiomodulation is also attractive to patients, as it is a quick treatment with no needles and no pills. Although this treatment requires a somewhat onerous schedule of 9 sessions over 3 to 5 weeks, repeated 3 times a year, the individual treatments each take only approximately 5 minutes. The patients I have treated with it thus far have found it to be a simple and easy procedure that is comfortable, and they like that they can come in for treatment and then continue the rest of their day without the disruption of blurry or distorted vision.

I am excited at the prospect of having an efficacious therapy to prevent the progression of dry AMD, and I am looking forward to seeing more data about this form of therapy emerge. Although the 13-month data we have so far are encouraging, I would be interested to see the effects on a large group of patients taken out to 2 to 5 years and beyond. It will obviously take us time to accrue these data, but if the results we have seen so far hold up over time, this has the potential to be an incredibly impactful form of therapy.

Encapsulated cell therapy: The first approved therapy for macular telangiectasia type 2

Another breakthrough we have seen this year has been the approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals Inc) for the treatment of adults with macular telangiectasia type 2 (MacTel).

MacTel is an uncommon condition in which the dysfunction of Müller cells, glial cells that support neurons in the retina, leads to destabilization of the outer retina and, ultimately, photoreceptor loss.

 Even when patients with MacTel have good performance on a Snellen chart, they often report significant visual disturbances, such as scotomas and metamorphopsia, and have difficulty with vision-related tasks of daily living.

Historically, there has been no treatment for patients with MacTel, and management has consisted solely of monitoring for the development of subretinal neovascularization in a small subset of patients. This can cause rapid vision loss from fluid and hemorrhage, and can be treated with anti-VEGF agents.

 However, we have not had any treatment for the underlying disease.

Now we finally have an option to offer patients with MacTel. Revakinagene taroretcel-lwey is an encapsulated cell therapy, in which retinal pigment epithelial cells that have been genetically modified to produce ciliary neurotrophic factor (CNTF) are encased in a semipermeable membrane, which is then anchored to the sclera. Once implanted, the cells within the capsule can subsist on nutrients present in the vitreous. The CNTF produced by these cells can then easily reach the retina.

The approval of revakinagene taroretcel-lwey was based on the results of 2 phase 3 clinical trials, which demonstrated a statistically significant difference in rate of change in ellipsoid zone area, a proxy measure for photoreceptor loss, through 24 months between eyes treated with revakinagene taroretcel-lwey and eyes treated with sham. Treatment with revakinagene taroretcel-lwey was also found to slow the loss of retinal sensitivity and reading speed. Revakinagene taroretcel-lwey was generally well tolerated, with the majority of treatment-emergent ocular adverse events being related to surgery.

Of course, treatment with revakinagene taroretcel-lwey only slows progression of the disease and does not reverse vision loss. As such, there will be a bit of a learning curve to figure out which patients will most benefit from this treatment and when in the disease course intervention is most beneficial, but the approval of this truly novel therapy after decades of research represents a significant milestone for our field. This platform is also interesting and can be used to treat other retinal diseases as well.

The confluence of many factors has led to the highly dynamic level of change we are currently experiencing in retina, including an aging population and an increase in our patient base, improvements in imaging techniques that allow us to better understand pathophysiology, and improvements in our ability to engineer biologic therapies. These changes show no sign of abating, and I look forward to how future advances will continue to reshape how we treat and care for our patients.

Garg is codirector of retina research on the Retina Service at Wills Eye Hospital, a partner with Mid Atlantic Retina, and is professor of ophthalmology at the Sidney Kimmel Medical College at Thomas Jefferson University, both in Philadelphia, Pennsylvania.

Garg reports being a consultant for the American Academy of Ophthalmology, Apellis, Bausch+Lomb, Boehringer Ingelheim, Merck Manuals, and West Pharmaceutical Services. Garg has reported performing contracted research for Adverum, Alexion, the American Academy of Ophthalmology, Annexon, Apellis, Astellas, Avonelle-X, Boehringer Ingelheim, Genentech, Gyroscope, Iveric Bio, Jefferson, KCRN, Kodiak, Lineage Cell Therapeutics, MUST Network, NGM Bio, Ocugen, OcuTerra, Opthea, Regeneron, REGENXBIO, and Roche/Genentech. Dr Garg reports having served on an advisory board for Coherus. Garg reports having received lecture fees from the Canadian Ophthalmological Society, Evolve, Physicians’ Education Resource

Sandoz receives FDA approval for Enzeevu (aflibercept-abzv), further strengthening US biosimilar position. Press release. Sandoz. August 12, 2024. Accessed May 27, 2025. https://www.sandoz.com/sandoz-receives-fda-approval-enzeevutm-aflibercept-abzv-further-strengthening-us-biosimilar/

Bordon AF, Kaiser PK, Wolf A, et al. Efficacy and safety of the proposed biosimilar aflibercept, SDZ-AFL, in patients with neovascular age-related macular degeneration: fifty-two-week results from the phase 3 Mylight study. 

. 2024;44(10):1704-1713. doi:10.1097/IAE.0000000000004174

LumiThera obtains FDA authorization of Valeda treatment for dry AMD patients to improve vision. Press release. LumiThera Inc. November 4, 2024. Accessed May 27, 2025. https://www.businesswire.com/news/home/20241104299697/en/LumiThera-Obtains-FDA-Authorization-of-Valeda-Treatment-for-Dry-AMD-Patients-to-Improve-Vision

Fernandes AR, Zielińska A, Sanchez-Lopez E, et al. Exudative versus nonexudative age-related macular degeneration: physiopathology and treatment options. 

. 2022;23(5):2592. doi:10.3390/ijms23052592

Leng T, Schwartz J, Nimke D, et al. Dry age-related macular degeneration: distribution of visual acuity and progression risk in a large registry. 

. 2023;12(1):325-340. doi:10.1007/s40123-022-00583-y

Evans J. Antioxidant supplements to prevent or slow down the progression of AMD: a systematic review and meta-analysis. 

. 2008;22(6):751-760. doi:10.1038/eye.2008.100

Boyer D, Hu A, Warrow D, et al. LIGHTSITE III: thirteen-month efficacy and safety evaluation of multiwavelength photobiomodulation in nonexudative (dry) age-related macular degeneration using the LumiThera Valeda Light Delivery System. 

. 2024;44(3):487-497. doi:10.1097/IAE.0000000000003980

Neurotech’s ENCELTO (revakinagene taroretcel-lwey) approved by the FDA for the treatment of macular telangiectasia type 2 (MacTel). Press release. Neurotech Pharmaceuticals Inc. March 6, 2025. Accessed May 27, 2025. https://www.neurotechpharmaceuticals.com/wp-content/uploads/Neurotech_Press-Release_BLA_Approval_FINAL.pdf

Kedarisetti KC, Narayanan R, Stewart MW, Reddy Gurram N, Khanani AM. Macular telangiectasia type 2: a comprehensive review. 

. 2022;16:3297-3309. doi:10.2147/OPTH.S373538

Clemons TE, Gillies MC, Chew EY, et al; Macular Telangiectasia Research Group. The National Eye Institute Visual Function Questionnaire in the Macular Telangiectasia (MacTel) Project. 

. 2008;49(10):4340-4346. doi:10.1167/iovs.08-1749

Tao W. Application of encapsulated cell technology for retinal degenerative diseases. 

. 2006;6(7):717-726. doi:10.1517/14712598.6.7.717

Albini TA. Phase 3, multicenter, randomized, sham-controlled studies of the efficacy and safety of revakinagene taroretcel in macular telangiectasia type 2. Presented at: Retina World Congress; May 9-12, 2024; Fort Lauderdale, FL.

Catch up on recent advancements in retina care, including new therapies for age-related macular degeneration and macular telangiectasia, enhancing patient outcomes.

Navigating the most recent FDA approvals in retina

The lesion characteristics of patients with 

 were found to affect the lesion growth rate, with fovea-sparing, multifocal, and bilateral lesions having the fastest growth rates in a study published in 

Translational Vision Science & Technology.

Daniel R. Muth, MD, the first author of the study, and colleagues evaluated the long-term fundus autofluorescence–based growth rate of lesions in treatment-naive patients with GA. The authors conducted the study to observe the GA progression and establish a comprehensive data set on the long-term growth dynamics of GA as a reference for future preclinical treatment developments and clinical trial end points.

Muth practices in the Division of Eye and Vision, Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden; St Erik Eye Hospital, Solna, Sweden; and the Department of Ophthalmology, University Hospital of Zurich, University of Zurich, Switzerland.

Approximately 6 million individuals worldwide are estimated to have late-stage dry age-related macular degeneration (AMD) with GA.

 The FDA has approved 2 complement system inhibitors—C3 inhibitor pegcetacoplan (Syfovre; Apellis Pharmaceuticals, Inc) and C5 inhibitor avacincaptad pegol (Izervay; Astellas Pharma Inc)—that have successfully slowed the progression of GA. However, neither of those treatments can improve vision.

 In Europe, however, the clinical benefit of pegcetacoplan was considered inadequate by the European Medicines Agency, which resulted in the rescinding of its market approval.

 The manufacturer of avacincaptad pegol then withdrew the drug from the application process.

“This development highlighted the need for European long-term data on GA kinetics and new biomarkers,” Muth and colleagues stated.

The bottom line is that AMD still lacks sufficient treatment. GA growth kinetics are essential for understanding this disease’s pathophysiology and predicting individual future growth.

The authors then conducted the study under discussion, a prospective, single-center, observational study between February 2013 and September 2024 at Karolinska Institutet and St Erik Eye Hospital. The patients underwent a clinical examination, fundus autofluorescence, and optical coherence tomography. The authors recounted that the area and the absolute and square root transformed growth rates were analyzed every 6 months.

Ultimately, 204 eyes of 111 patients (median baseline age, 79 years; range, 56-93 years; 60% women) were enrolled in the study. The median follow-up time was 21 months (minimum-maximum, 5-123). Of 73 eyes in which the fovea was spared, 22 eyes later converted to involvement of the fovea over a median of 24 months.

“The mean growth for the total cohort was 1.597 mm

/year and 0.264 mm/year after square root transformation. Bilateral [1.621 mm

/year; 0.267 mm/year], multifocal [1.961 mm

/year; 0.322 mm/year], and fovea-sparing [1.987 mm

/year; 0.234 mm/year] lesions showed significantly faster growth when analyzed in isolation. In a mixed statistical model that controlled for bilaterality, only foveal status remained a significant influencer on the square root transformed growth rate [

This study indicated that by analyzing the data of one of the most extensive GA patient cohorts in the Nordic countries, it established a data set on the long-term treatment-naive GA growth dynamics. Muth and colleagues pointed out that it provides a reference for upcoming preclinical treatment developments and clinical trial end points.

“In this GA cohort, we demonstrated the influence of lesion characteristics on growth rates with follow-up times of up to more than 10 years. Evaluated individually, fovea-sparing, multifocal lesion configuration, and bilateral lesions showed significantly faster GA growth. In combined statistical models, sparing of the fovea remained a significant risk factor for faster growth that was corrected for baseline lesion size by square root transformation. Patients presenting with 1 or more of these lesion characteristics hold a high potential to benefit from future treatments because the slowing of lesion growth may be more likely to be achieved. In fovea-sparing cases, functional preservation may be possible,” they concluded.

Muth practices in the Division of Eye and Vision, Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden; St Erik Eye Hospital, Solna, Sweden; and the Department of Ophthalmology, University Hospital Zurich, University of Zurich, Switzerland. He is supported by Stiftung OPOS, St Gallen, Switzerland.

Muth DR, Quérat L, Venkataraman AP, et al. Long-term natural history of treatment-naïve geographic atrophy in age-related macular degeneration. 

Shen LL, Sun M, Grossetta Nardini HK, Del Priore LV. Progression of unifocal versus multifocal geographic atrophy in age-related macular degeneration: a systematic review and meta-analysis. 

. 2020;4(9):899-910. doi:10.1016/j.oret.2020.03.020

Heier JS, Lad EM, Holz FG, et al. Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials. 

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Fovea-sparing, multifocal, and bilateral lesions exhibited the fastest growth rates.

Ursula Schmidt-Erfurth, MD

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