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

Optogenetics for retinal disease

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

Kiora Pharmaceuticals recently announced the receipt of a patent for the company’s ocular therapy KIO-104.

 KIO-104 is for the treatment of a wide range of ocular diseases, including ocular inflammation, uveitis, age-related macular degeneration, and complications as a result of refractive surgery. Along with other novel treatment methods for ocular inflammatory diseases, the patent (US Patent No. 12,364,680) also covers varying dosing schedules and necessary excipients.

“Securing this patent builds on our IP strategy to protect KIO-104 around its proprietary structure, delivery methods, formulation, and therapeutic applications,” Brian M. Strem, PhD, president and CEO of Kiora, said in a press release. “This specific patent is significant in its breadth of therapeutic applications.”

A small molecule in development for inflammatory retinal diseases, KIO-104 offers an alternative treatment method to steroids or systemic anti-inflammatory drugs. It works by inhibiting the mitochondrial enzyme DHODH, which plays a key role in the synthesis of key building blocks of DNA and RNA.

The KLARITY study, a multicenter phase 2 clinical trial, is currently screening patients to evaluate the role of KIO-104 in up to 28 patients at 2 dose levels across various forms of inflammatory retinal diseases.

 The results from the initial stage study will inform dose expansion.

The patent issuance is expected to further strengthen the protection of KIO-104 and extend market exclusivity into 2043, the company said in a press release.

Kiora Pharmaceuticals, Inc. Kiora Pharmaceuticals, Inc. Published July 22, 2025. Accessed July 23, 2025. 

https://ir.kiorapharma.com/news-events/news-releases/detail/235/kiora-pharmaceuticals-granted-u-s-patent-expanding-protection-for-kio-104-in-the-treatment-of-ocular-disease

Kiora Pharmaceuticals secures a patent for KIO-104, enhancing treatment options for ocular diseases and extending market exclusivity until 2043.

clinical

Kiora Pharmaceuticals receives patent for KIO-104

ANI Pharmaceuticals announced results from its NEW DAY clinical trial of ILUVIEN (fluocinolone acetonide intravitreal implant), 0.19 mg for patients with 

ILUVIEN is a corticosteroid indicated for treating DME in patients previously treated with a course of corticosteroids who did not have a clinically significant rise in intraocular pressure (IOP).

NEW DAY is a prospective, multicenter, masked, randomized, active-controlled trial that enrolled 306 eyes of treatment-naive, or almost naive, DME patients at approximately 42 sites around the US. Patients enrolled were older than 18 years with a diagnosis of type 1 or type 2 diabetes with a center-involving DME confirmed by SD-OCT, and CST of ≥ 350 µm and best corrected visual acuity (BCVA) of ≥35 ETDRS letters and ≤80 ETDRS letters in the study eye at the screening visit. The trial's primary end point was the mean total number of supplemental aflibercept injections needed for the treatment of DME in the ILUVIEN arm compared to the aflibercept arm over the 18-month study period in the intent-to-treat (ITT) population.

Patients were randomly assigned to either an ILUVIEN arm (n=154), where they received an ILUVIEN intravitreal implant, or an aflibercept arm (n=152), where they received 5 injections of anti-VEGF therapy (intravitreal aflibercept 2 mg) at 4-week intervals for the first 16 weeks of the trial during an induction period.

At the end of the 16-week induction period, patients in both arms were evaluated every 4 weeks during a 13-month maintenance period and received supplemental aflibercept injections only as needed.

According to the company, treatment with ILUVIEN demonstrated a numerical reduction in the mean number of supplemental aflibercept injections compared with the aflibercept arm. However, it did not reach the threshold for statistical significance (2.4 vs. 2.5; 

 = .756), and therefore the primary end point was not met. The secondary end point of mean time from last treatment injection to first supplemental aflibercept injection was met with a mean time of 185.4 days in the ILUVIEN arm, compared to 132.8 days in the aflibercept arm (

 < .001). Additionally, 33% of patients in the ILUVIEN arm did not require a supplemental injection during the study compared to 30% in the aflibercept arm.

Secondary end points that assessed visual acuity and anatomic changes in the ITT population demonstrated noninferiority between the ILUVIEN arm and the aflibercept arm, and additional secondary end points will be further evaluated by the company.

A post hoc analysis was conducted on a subset of randomly assigned patients without major protocol deviations, which showed that the ILUVIEN arm (n=128) demonstrated a statistically significant difference in the mean number of supplemental aflibercept injections compared to the aflibercept arm (n=134), with 1.8 vs 2.5 injections, respectively (

 = .029). Therefore, patients in the post hoc analysis received a total of 2.8 injections in the ILUVIEN arm, whereas the aflibercept arm patients received a total of 7.5 injections.

Michael A. Singer, MD, clinical professor of ophthalmology at the University of Texas Health Science Center and director of clinical research at Medical Center Ophthalmology Associates in Texas, commented on the results in a press release.

“The NEW DAY study provides clinically meaningful data on ILUVIEN’s potential impact on treatment burden, including a statistically significant difference in time to first supplemental injection of aflibercept in the ILUVIEN arm compared to the aflibercept arm. This potential reduction in treatment burden can be critical for our patients with diabetic macular edema, as their disease is multifactorial, necessitating multiple visits to medical specialists.”

Safety data were reported as “consistent with data from prior ILUVIEN clinical trials and real-world use.” In the ITT patient population, 41% of patients in the ILUVIEN arm experienced treatment-related treatment-emergent adverse events, compared with 3% in the aflibercept arm. The company notes that these were mainly associated with cataract/subcapsular cataract (n=50) and an increase in IOP (n=24). Additionally, 16% of patients in the ILUVIEN arm experienced an increase in IOP compared with 3% in the aflibercept arm, whereas 11% of patients experienced an increase in IOP of ≥25 mm Hg compared with 3% in the aflibercept arm.

ANI Pharmaceuticals Announces Results from NEW DAY Clinical Trial of ILUVIEN® for Use in Patients with Diabetic Macular Edema (DME). Published July 23, 2025. Accessed July 24, 2025. 

https://www.globenewswire.com/de/news-release/2025/07/23/3120083/0/en/ANI-Pharmaceuticals-Announces-Results-from-NEW-DAY-Clinical-Trial-of-ILUVIEN-for-Use-in-Patients-with-Diabetic-Macular-Edema-DME.html

ANI Pharmaceuticals releases results for ILUVIEN in treating diabetic macular edema.

Canonical

NEW DAY trial of ILUVIEN for DME does not meet primary end point; post hoc analysis suggests potential benefits

In a press release, Aldeyra Therapeutics, Inc, announced that the European Medicines Agency (EMA) has granted orphan designation for its methotrexate intravitreal injection (ADX-2191). The therapeutic candidate is formulated to treat inherited retinal dystrophies, including retinitis pigmentosa (RP). ADX-2191 is being investigated for performance in 

In 2021, the FDA granted orphan drug designation to ADX-2191.

 The phase 2 trial supporting the EMA's decision this week was originally announced in 2023. Data from that trial demonstrated improvements from baseline in retinal sensitivity. The company, based in Lexington, Massachusetts, plans to initiate a phase 2/3 trial further examining ADX-2191 for RP later in 2025.

In the European Union, orphan designation grants companies a number of benefits: reduced regulatory fees, research grants, clinical protocol support, and potential long-term market exclusivity throughout the EU. The EMA grants orphan designation to drugs/biologics intended to address life-threatening or chronically debilitating diseases that affect fewer than 5 in 10,000 EU residents.

Todd C. Brady, MD, PhD, president and CEO of Aldeyra Therapeutics, said the orphan designation will expand access to therapies for RP and other inherited retinal dystrophies. “Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology,” Brady said in the press release.

 “The receipt of orphan designation from the EMA, in conjunction with the previously announced orphan drug designation from the [FDA], highlights the importance of developing a treatment option for patients suffering from retinitis pigmentosa.”

Currently, there are no approved treatments for RP, a group of genetic eye diseases characterized by retinal cell death and subsequent loss of visual acuity. In the press release, Aldeyra Therapeutics provided information about the functional utility of its methotrexate intravitreal injection. In vivo preclinical research identified methotrexate as an agent to induce misfolded rhodopsin clearance. Rhodopsin, a protein found in the rod cells, is light-sensitive and crucial to the visual cycle. ADX-2191 is a sterile, noncompounded intravitreal formulation of methotrexate that is preservative-free and vitreous-compatible. The US FDA's orphan drug designation applies to the use of ADX-2191 in treating RP and primary vitreoretinal lymphoma.

Aldeyra Therapeutics designs pharmaceuticals that modulate protein systems, rather than functioning with a protein inhibitor/activator model. Along with ADX-2191, the company's other late-stage product candidate is reactive aldehyde species (RASP) modulator reproxalap, indicated as a potential therapy for dry eye disease and allergic conjunctivitis. Other product candidates from the company include RASP modulators ADX-629, ADX-248, ADX-743, ADX-631, and ADX-246.

Aldeyra Therapeutics Receives Orphan Designation from the European Medicines Agency for ADX-2191 for the Treatment of Inherited Retinal Dystrophies including Retinitis Pigmentosa. Press release. Aldeyra Therapeutics. Published July 24, 2024. Accessed July 24, 2025. 

https://www.businesswire.com/news/home/20250724869031/en/Aldeyra-Therapeutics-Receives-Orphan-Designation-from-the-European-Medicines-Agency-for-ADX-2191-for-the-Treatment-of-Inherited-Retinal-Dystrophies-including-Retinitis-Pigmentosa

Kaiser Maharjan E. U.S. FDA grants orphan drug designation to ADX-2191 from Aldeyra Therapeutics. 

. Published August 5, 2021. Accessed July 24, 2025. https://europe.ophthalmologytimes.com/view/u-s-fda-grants-orphan-drug-designation-to-adx-2191-from-aldeyra-therapeutics

Findings from a phase 2 clinical trial support the therapeutic potential of ADX-2191 in patients with retinitis pigmentosa.

ADX-2191 receives EMA orphan drug designation for inherited retinal dystrophies

The phase 3 ARCHER II trial of vonaprument (formerly ANX007) for dry age-related macular degeneration (AMD) with geographic atrophy (GA) has completed enrollment. This aligns with the previously shared timeline, and Annexon remains on track to report topline data in the second half of 2026.

Vonaprument is a first-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q locally in the eye with an intravitreal formulation. The neuroprotective effect and consistent benefits of C1q inhibition were previously demonstrated by vonaprument in a phase 2 trial.

According to the company’s press release

, ARCHER II is a global, randomized, double-masked, sham-controlled pivotal trial that has enrolled more than 630 patients with advanced dry AMD/geographic atrophy (GA). Patients will be randomized 2:1 to receive a monthly dose of vonaprument or sham procedure.

The primary end point is the prevention of ≥15-letter loss of best corrected visual acuity (BCVA), which represents 3 lines on the standard Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart. The primary analysis will occur at least 12 months from dosing initiation. Secondary end points in ARCHER II include safety, low-luminance visual acuity (LLVA), and photoreceptor integrity (EZ). Topline data are expected in the second half of 2026. Vonaprument has received priority medicine (PRIME) designation in Europe and fast track designation in the US.

David Eichenbaum, MD, director of research at the Retina Vitreous Associates of Florida, will give an oral presentation on the design and rationale of the pivotal phase 3 ARCHER II trial at the American Society of Retina Specialists (ASRS) annual meeting held in Long Beach, California.

Eichenbaum, a principal investigator of the ARCHER II trial, is quoted in the company’s press release

, saying, “The global reach of ARCHER II has increased awareness of vonaprument’s innovative mechanism and compelling phase 2 results. With no approved therapies in Europe and limited options in the US, there is an urgent need for a treatment for GA that protects vision, the most clinically meaningful outcome for patients. I’m encouraged by the robust design of ARCHER II and look forward to helping advance this differentiated therapy worldwide.”

 ARCHER II, a Phase 3 Randomized Clinical Trial of ANX007 in Patients With Dry AMD and GA: Study Design and Rationale Informed by Outcomes From ARCHER

David A. Eichenbaum, MD, FASRS, Retina Specialist, Retina Vitreous Associates of Florida

Thursday, July 31 at 2:59 PM Pacific Time (PT)

Annexon Announces Completion of Enrollment in Pivotal Phase 3 ARCHER II Trial of Vonaprument (formerly ANX007) for Dry Age-Related Macular Degeneration (AMD) with Geographic Atrophy. July 24, 2025. Accessed July 24, 2025. News Release. https://finance.yahoo.com/news/annexon-announces-completion-enrollment-pivotal-110000694.html

The phase 3 ARCHER II trial completes enrollment for vonaprument, targeting vision preservation in dry AMD, with results expected in 2026.

Phase 3 ARCHER II trial completes enrollment

Carl D. Regillo, MD, shares updates on the VERONA trial for DME

Carl D. Regillo, MD, Director of the Retina Service at Wills Eye Hospital and partner at Mid Atlantic Retina, met with Modern Retina at the 2025 Retina World Congress meeting held in Fort Lauderdale, Florida. He discussed the VERONA study and its implications for ophthalmology. The study focused on EYP-1901, a novel treatment for center-involved diabetic macular edema (DME) with decreased vision. This innovative therapy involves vorolanib packaged in a bio-erodible polymer and administered via intravitreal injection. The results were notably promising, outperforming aflibercept in both vision improvement and disease control. Key highlights of the study include:

Exceptional durability, with most patients not requiring supplemental anti-VEGF therapy for 6 months

Notable performance compared to a single dose of aflibercept

Potential to address current limitations in retinal treatment

Regillo emphasized the broader significance of clinical trials in ophthalmology, noting that collaborative efforts with industry have driven substantial progress in therapeutics over recent decades. Despite these advancements, significant challenges remain in the field. Current limitations in retinal treatments include:

Suboptimal treatment responses for some conditions

Challenges in maintaining consistent patient engagement

The study represents a promising step toward addressing these challenges by potentially:

Minimizing the logistical challenges of frequent medical visits

Regillo's perspective on the trajectory of ophthalmology is fundamentally optimistic. He views the ongoing research as a critical pathway to enhancing patient care, particularly for sight-threatening diseases like wet AMD and diabetic retinopathy. The conversation underscores the dynamic nature of ophthalmological research, highlighting how innovative approaches and persistent scientific exploration can progressively improve medical treatments and patient experiences.

Allen C. Ho, MD, FACS, FASRS

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