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Blanton Eye Institute at Houston Methodist Hospital in Houston, Texas

Andrew G. Lee, MD, shares essential insights on neuro-ophthalmology for retina specialists, emphasizing diagnosis techniques and collaboration with neuro-ops.

Q&A: Andrew G. Lee, MD, shares what every retina specialist should know about neuro-ophthalmology

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

Justis Ehlers, MD, FASRS presents on PER-001, a Long-acting Endothelin Antagonist Intravitreal Implant, Improved Structure and Visual Function in Patients With Diabetic Retinopathy

Justis Ehlers, MD, FASRS, a retina surgeon from the Cole Eye Institute at the Cleveland Clinic, presented at the 2025 annual meeting of the American Society of Retina Specialists (ASRS). The meeting was held in Long Beach, California, from July 30 through August 2, 2025. 

His presentation, titled "PER-001, a Long-acting Endothelin Antagonist Intravitreal Implant, Improved Structure and Visual Function in Patients With Diabetic Retinopathy," focused on a study of PER-001, a novel therapeutic targeting the endothelin-1 receptor, a potent vasoconstrictor in the human body. 

This treatment candidate aims to address challenges in diabetic retinopathy and potentially improve retinal function. The phase 2A study was designed to evaluate the effectiveness of PER-001 through a comparative analysis between 2 different dosage groups and a sham group. The research specifically targeted patients with diabetic retinopathy without diabetic macular edema (DME), who typically maintain good central visual acuity but may experience early-stage visual complications. Key research findings revealed promising results across functional and structural end points. Functionally, the study observed clear trends in the treatment groups, with potential improvements in:

Structurally, quantitative ultrawidefield angiography demonstrated positive signals in:

The sham group consistently showed progressive worsening across these parameters, highlighting the potential efficacy of the PER-001 therapeutic approach. A notable aspect of the study is the extended-release implant system, similar to existing dexamethasone implants. This innovative delivery mechanism suggests the possibility of limited dosing, potentially every 6 months, which could significantly improve patient treatment convenience and adherence. 

The research represents an important step in understanding how endothelin-1 receptor antagonists might address vascular and inflammatory challenges in retinal conditions. The mechanism's potential to impact ischemia, inflammation, and vascular leakage opens promising avenues for future therapeutic interventions. Moving forward, the research team plans to conduct a phase 2B/3 study, with a primary focus on evaluating the duration and durability of the PER-001 therapeutic approach. This next phase will be crucial in determining the long-term potential of this innovative treatment strategy for patients with diabetic retinopathy.

Justis Ehlers, MD, FASRS, presented "PER-001, a Long-acting Endothelin Antagonist Intravitreal Implant, Improved Structure and Visual Function in Patients With Diabetic Retinopathy," during the 2025 ASRS meeting held in Long Beach, California.

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ASRS 2025: PER-001 improved structure and visual function in patients with diabetic retinopathy

The 2025 annual meeting of the American Society of Retina Specialists was held in Long Beach, California, from July 30 through August 2, 2025. At this event, Robert A. Sisk, MD, FACS, FASRS, gave a presentation titled, "Subretinal Laru-zova Gene Therapy for XLRP: 24-Month Results of the Randomized, Controlled, Multicenter Phase 2 SKYLINE Trial."

caught up with Sisk at the meeting to discuss these trial results and how they might impact future candidates and research into X-linked retinitis pigmentosa (XLRP).

Note: The following conversation has been lightly edited for clarity.

Can you share key highlights from your presentation on the phase 2 SKYLINE trial at ASRS 2025?

SKYLINE was a phase 2 trial looking at the dose-dependent response of laru-zova, which is a full-length RPGR gene therapy, and trying to look at groups from the previous phase 1/2 study that showed some benefit, groups 2 and 5 from HORIZON. Ultimately, we're trying to see with a larger patient base, does that stratify out to a benefit that's greater in 1 group than the other? And we did see that in the high-dose group, there was a significant improvement in microperimetry response, which is essentially visual sensitivity in the central retina, the portion that's treated with the gene therapy, compared to the low-dose group.

What patient population makes up this study?

Males aged 8 to 50 years, who had a demonstrated pathogenic variance in the gene 

, which is the most common gene that causes X-linked retinitis pigmentosa, were able to participate, but they also had to have measurable microperimetry sensitivity so that they could demonstrate an increase with treatment or a decrease if there was a safety issue. And an intact foveal ellipsoid band, which is an anatomic marker that shows the integrity and health of the light-catching cells, or photoreceptors, at the area of the retina that provides the best vision?

Were there any surprising or unexpected results in these data?

Well, I think not so much from this particular study, but when we look at the HORIZON patients, it was clear that those patients who had the measurable microperimetry and the intact ellipsoid band were those who were more likely to benefit from the gene therapy. And that has been something that we've seen in a variety of other gene therapy trials. It seems like the ellipsoid zone is important. Having near-normal retinal anatomy in that area is important to see the benefit of the gene therapy.

How might these results shape a phase 3 trial or future XLRP research?

 So the exciting thing is that the phase 3 trial is not just under way, but it's actually already fully recruited. So this trial was important in verifying the vision benefit from microperimetry. An almost parallel trial called DAWN helped to establish low luminance visual acuity is another important metric of potential visual improvement, and both of those are being looked at as primary end points for the VISTA study.

Did this study provide any insights into retinitis pigmentosa as a disease state? If so, what have we learned?

I think what we're learning is that there is, at least, with the gene therapy trials, somewhat of a narrow window in which we can treat these patients. Patients have to have some diminished vision. In the trial, it was 20/32 to 20/200. They have to have some diminished vision. Otherwise, there's a ceiling effect in terms of being able to see improvement in visual acuity. But as the visual acuity declines, there's also typically loss of the ellipsoid band. So if there's total loss of the ellipsoid band, those patients are not candidates. So from a clinical trial perspective, it's a narrow window where there's a beginning of vision decline, but not so much that it's taken away that anatomic parameter that would allow the vision to benefit. I'm hopeful that if this gets approved, following the phase 3 study, that we'll be able to treat patients at an earlier age who may be able to benefit rather than having such a narrow recruiting window.

 As you consider the pipeline potential therapeutics for treating patients with retinitis pigmentosa, what do you hope the future holds?

 I'm hopeful that we'll see a variety of different therapeutic options. It's very exciting to see one trial making it all the way to phase 3. But we really want patients to not just have gene augmentation therapy, but also, we want some gene agnostic approaches, like stem cell studies, optogenetic gene therapy for patients with really late-stage disease. So I think in the end, in clinical practice, we'll have therapies that harmonize and really give a more complete outcome for those patients.

Q&A: 24-month results of the phase 2 SKYLINE trial for XLRP

Christina Y. Weng, MD, MBA, FASRS, a professor of ophthalmology at Baylor College of Medicine in Houston, Texas, spoke with 

 about her presentation at the American Society of Retina Specialists (ASRS) meeting. This meeting is taking place from July 30 through August 2, 2025. 

Her presentation is titled, "Postoperative Day 0 as a Substitute for Postoperative Day 1 Examination Following Vitreoretinal Surgery: A Comparative Prospective Study." This research examines the real-life need to conduct day 0 postop exams and how patients present similarly or differently on day 0 to day 1 postop exams. 

Note: The following presentation explanation and review of data has been lightly edited for clarity.

I am presenting a paper that's comparing exam findings on postoperative day 0 and postoperative day 1, following vitreoretinal surgery. And I'd also like to acknowledge my coauthors, Dr Will Park and Dr Lupe Torres. 

There's been a lot of interest in this topic, because every vitreoretinal surgeon has encountered situations where a postop, day 1 exam just simply isn't feasible. And I'll give you 2 real examples of patients of mine. I had a patient from New Orleans who lives 5 hours away from the surgery center, and her caretaker was unable to drive her back the next day after emergent surgery. Another example I think of is a few years ago when one of my longtime patients had a mac-on retinal detachment on a Friday, and I had an early flight I had to catch on Saturday morning, but she really, really wanted to have surgery with me. So in these types of situations, many of us have wondered, "Is it okay to see the patient on postoperative day 0 instead of day 1?" 

That's what this study explores. This was a prospective study of eyes operated on by 2 retina surgeons that were seen on both postop day 0 and again on postop day 1. On each of the days, we collected 5 main outcomes of interest, intraocular pressure, visual acuity, postop complications, pain level, and view of retina. So what do we find? 

So as a nod to my pilot series, launching scene with 

, The TLDR, is that a postop day 0 exam may be a safe and effective substitute for the traditional postop day 1 exam. More specifically, we included 90 eyes with a variety of conditions, with the most common being vitreous hemorrhage, tractional retinal detachment, and rhegmatogenous retinal detachment, over half received gas or oil tamponade. The mean time from the end of surgery to the postop day 0 evaluation was about 7 hours. So if a case ended at 9 AM, let's say, we saw them at 4 PM later that day. 

Now walking through each of these 5 outcome measures, we found that the visual acuity did improve slightly from day 0 to day 1, probably in due part to the retrobulbar block wearing off. On average, the IOP did increase from 13.8 to 17.5 mm of mercury from day 0 to day 1. But both values, as you can see, were obviously in the normal range. More eyes had IOP elevation on day 1 compared to day 0, but the proportion of eyes with IOP 30 or greater was small and comparable between the 2 days. And of course, what we really want to know is how many eyes had an IOP that was okay on day 0, but then high on day 1. This happened in 9 eyes out of the 90, and regression analysis showed an association with IOL placement, TRD primary diagnosis, or C3F8, gas use. And even so, none of these 9 eyes had an IOP that exceeded 34. There were also no differences in pain levels, visualization of the retina, or postop complications, and there were no cases of endophthalmitis. 

So to recap, the postop day 1 exam, while it's important, can occasionally pose real-life challenges, and there have been a number of smaller retrospective studies published on this topic. But what's unique about our study is that it looks at the same patient cohort on both days, and we found that the risk of developing significant complications on postop day 1 after an exam on postop day 0 is low. In fact, the only clinically meaningful differences were with IOP, and they affected a small proportion of eyes only. 

Of course, we have sample size limitations and the case mix variability, but our finding support that a postop day 0 exam is a safe and effective substitute for the traditional postop day 1 exam.


Christina Weng, MD, MBA, FASRS, explores the viability of postoperative day 0 exams as a substitute for day 1 after vitreoretinal surgery, revealing promising findings.

Christina Y. Weng, MD, MBA, FASRS, on comparative study of postop day 0 and day 1 exams

Association of Serum Vitamin D Insufficiency With Proliferative Vitreoretinopathy After Rhegmatogenous Retinal Detachment Repair

Steven D. Ness, MD, an attending physician at the VA Boston Healthcare System and assistant professor at Boston University, presented a study examining the relationship between vitamin D levels and proliferative vitreoretinopathy (PVR) risk in retinal detachment surgery patients. 

The study involved 313 patients who underwent surgical treatment for primary rhegmatogenous detachments, with serum vitamin D levels measured within 1 year of surgery. Of these patients, 42 developed PVR after their initial surgery. The patient population was notably homogeneous, comprising over 95% males and over 80% Caucasians. 

Key findings revealed a significant correlation between low vitamin D levels and PVR risk. Patients with low vitamin D experienced over 4 times higher risk of developing PVR post surgery. Moreover, for each 1 ng/mL decrease in vitamin D levels, the PVR risk increased by 4%. These results were both statistically significant and potentially clinically meaningful. The research methodology was rigorous. 

Researchers were able to conduct individual chart reviews to ensure data accuracy, specifically screening out patients with preexisting PVR and focusing on straightforward retinal detachment cases. This approach provided a cleaner dataset compared to typical database studies. 

While acknowledging the study's limitations—primarily its narrow demographic—Ness highlighted the potential clinical implications. Vitamin D therapy represents a low-risk, low-cost intervention that could potentially reduce severe surgical complications and improve vision outcomes. 

Future research plans include examining larger, more diverse patient populations to validate these initial findings. The goal is to determine whether the observed vitamin D-PVR relationship holds true across different demographic groups. The study's significance lies in being the first to demonstrate an association between vitamin D levels and PVR risk. Ness suggested that simple interventions like vitamin D supplementation or increased sunlight exposure might help mitigate surgical risks, offering a promising avenue for further investigation in ophthalmological care.

ASRS 2025: An association of vitamin D insufficiency with proliferative vitreoretinopathy

As part of the 2025 American Society of Retina Specialists annual meeting, Andrew G. Lee, MD, was invited to give a talk titled "What Every Retina Doctor Needs to Know About Neuro-ophthalmology." Following his presentation, 

 had the chance to speak with Lee about what he shared and the field of neuro-ophthalmology.

 We were invited to give a talk here at ASRS about what every retina doctor needs to know about neuro-ophthalmology. Can you provide some highlights from that talk?

Sure, what every retina doctor needs to know about neuro-ophthalmology is, ironically, when it's not the retina. The super power that we emphasize today was swinging a flashlight looking for a relative afferent pupillary defect [RAPD], and so when the patient sees nothing and the doctor sees nothing in the retina, that's actually neuro-op, and that is what we emphasize today. It's when it's not the retina that is a super important, super power, detecting the RAPD, and that person needs a neuro-op workup.

 Can you highlight what it's like to work with a neuro-op for these doctors when they should call in, or obviously, it's a much smaller specialty even than retina, so how can someone get in touch with a neuro-op if they don't have a connection any of kind?

So the neuro-ophthalmologist community is quite small, and it's a demand specialty, and we don't have neuro-ops in every city. A lot of times, we're serving in a consulting role and helping retina docs and community docs and comprehensive ophthalmologists to know what to do, even if we're not seeing the patient. You should definitely engage with and try and have a neuro-ophthalmologist help you walk through a case, even if they're not going to see it, because the next available neuro-op appointment is usually 3 to 6 months away. If, however, it's an emergency problem, that person should go to the hospital. You still need to call your neuro-op, but the way to get a neuro-op consult is at the hospital. Somebody is going to see them on call. Neurology is going to see them. They're going to have the MRI. The hospital is going to admit them. So that's actually the fast track to getting a neuro-ophthalmologist, if it's an emergency, is admit, but all the other neuro-ops, you should use your neuro-op locally in a consulting role, and the amazing thing about the internet now is you don't actually have to be in the same city or even in the same country. You can access neuro-ops remotely anywhere in the world now.

As a neuro-op, what is the most common question you get, and how do you answer that?

The most common question that we get in neuro-op is, "Should we scan this person?" The answer is, if it's an optic neuropathy or a cranial neuropathy, or it's something that could be intracranial or intraorbital, you definitely need to scan the person. So "where is the lesion?" is a much more important question than "what is the lesion?" The MR will tell you what it is. It's your job as a clinician to determine whether it is the eye, in the orbit, or in the brain. If it's in the brain or the orbit that needs a scan, and that scan has a name: MR, head, orbit, gadolinium fat-sat, MR, head, orbit, gadolinium fat-sat. That is the most common question that we receive in neuro-op.

 From a technology standpoint, what do you think are the most powerful tools when it comes to diagnosing and managing conditions that might align with a neuro-op? Is it that flashlight test? What technology do you see as being crucial?

We have a lot of low-tech things like the flashlight, where we're just swinging that light to look for the relative afferent pupillary effect, the RAPD, and that hasn't changed since the 1800s. What's changed is OCT has completely changed the landscape on "is it the retina or is it the optic nerve?" Optical coherence tomography (OCT) sees better than me. It sees better than you. It sees better than any person who's ever lived because it sees to the micron level. So having an OCT in the macula and an OCT of the optic nerve has been a game changer on is it neuro-op: yes or no, and that is part of what we talked about today at the lecture.

To hear more from Andrew G. Lee, MD, or to learn more about neuro-ophthalmology cases, watch his series, 

Andrew G. Lee, MD

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