Transforming the experience of retinal disease treatment
Decades in the clinic and a personal tie to macular degeneration give Pravin Dugel a rare perspective and a relentless drive to improve retinal care. For years, effective treatments for wet AMD were lacking, and the level of care was often negligible, worsening symptoms.
The advent of Lucentis and Eylea brought about much needed improvements. “It was truly miraculous. It's not just that we could preserve vision, but we could actually improve vision.”
Still, treatment remains unsustainable for many patients today. The required frequency of injections is a huge burden for patients, who have to spend an entire day in the clinic – resulting in a staggering 40% drop out rate. “These patients end up going blind, and that's simply not acceptable,” says Dugel.
Dugel observes that even patients who remain on treatment may lose their vision after two to five years due to fibrosis and atrophy. “It’s kind of like having multiple concussions on a regular basis. It [becomes] fibro[tic] and scars, and that's how patients lose vision.” He underscores evidence that continuous suppression of the vascular endothelial growth factor (VEGF) can drive better long-term outcomes.
“[Lucentis and Eylea were] truly miraculous. It's not just that we could preserve vision, but we could actually improve vision.”
Pravin Dugel, MD, Executive Chairman, President and CEO of Ocular Therapeutix
AXPAXLI poised to reshape treatment and expand the market
Dugel explains that while monthly injections were once thought impossible, they are now routine and safe. AXPAXLI addresses this challenge with an FDA-approved tyrosine kinase inhibitor delivered via Ocular’s tuneable ELUTYX™ hydrogel platform . Already used in other medical applications, the hydrogel is distinct in that it fully dissolves after releasing the drug.
“The hydrogel can be tuned to last a few months, nine months, even up to 12 months. That’s the beauty of it—it’s fully adjustable and leaves nothing behind once the drug is delivered.”
While Ocular’s initial focus is wet AMD, the VEGF-inhibiting mechanism of AXPAXLI means it could also be effective for other retinal vascular diseases like diabetic retinopathy and retinal vein occlusion.
“The potential exists for this drug to be the most impactful drug that we've ever had in this field. It’s a well-known target – we know it works.”
The growing burden of vision loss carries profound human and economic consequences. As blindness rates rise in the U.S. and around the world, so do healthcare costs due to increased demand on medical resources. “If we're able to reduce the dropout rate by even 10% – that means a quarter million more patients in this country alone will not go blind – it’s not just the human factor, which is most important, but the cost savings are tremendous,” says Dugel.
“The potential exists for this drug to be the most impactful drug that we've ever had in this field.”
Pravin Dugel, MD, Executive Chairman, President and CEO of Ocular Therapeutix
Infrastructure, technology and commercialization
Building on the tight-knit nature of the retina specialist community, Ocular’s extensive experience is rooted in a deep understanding of patient needs, doctor preferences, and the intricacies of commercialization.“One of the reasons I'm here is because I had the chance to get the best of the best retina specialists. Some of us have practiced for 20, 30 years. We know what patients need. We know what doctors look for.”
Despite the current high patient dropout rates, the wet AMD market is well established. Ocular aims to commercialize AXPAXLI independently, without the need for external collaboration for capital or expertise. “Our goal is to go all the way.”
Ocular’s propriety technology platform, ELUTYX, is what delivers the FDA-approved payload, axitinib.
The excellent safety profile observed in clinical trials for AXPAXLI and their glaucoma product, PAXTRAVA – which is deliberately tested in the eye's most sensitive area – is evidence of the platform’s versatility and potential effectiveness for future indications.
Dugel stresses Ocular's meticulous adherence to FDA draft guidelines issued in February 2023. “We have followed their guidelines absolutely obsessively, because we don't want to take any regulatory risk whatsoever.”
The SOL-1 study, a superiority study, has shown excellent recruitment and patient quality, with expected rescue rates, reviewed on a masked basis, indicating the targeted VEGF-dependent patient population was enrolled. This study is masked and on track to read out in Q1 2026. The SOL-R study, a non-inferiority study, is also recruiting remarkably well, though no guidance has been provided on read out timing yet.
“Some of us have practiced for 20, 30 years. We know what patients need. We know what doctors look for.”
Pravin Dugel, MD, Executive Chairman, President and CEO of Ocular Therapeutix
Unlocking business opportunities amid market tailwinds
The market opportunity in retinal disease treatment is vast. “Every [approved] drug that targets VEGF, that has worked in wet macular degeneration, has worked in diabetic retinopathy, diabetic macular edema, retinal vein occlusion – all of which represent massive markets.”
In its HELIOS study in non-proliferative diabetic retinopathy, Ocular observed that a single injection of AXPAXLI reduced the incidence of vision-threatening complications to zero after 48 weeks. This is particularly significant for working-age individuals – doctors, lawyers, truck drivers – who, despite a high risk of vision loss, currently receive almost no treatment due to the burden of frequent injections. “Compelling data like this makes conversations with investors easy,” notes Dugel.
The current macro environment remains unstable, however Ocular’s disciplined approach, including its FDA compliance and financial prudence – the company is well capitalized into 2028 – is well positioned to drive positive results for both SOL-1 and SOL-R trials, the company’s primary focus.
“We’ve got one shot on goal. We've got the team to do it. We have the expertise to do it, and we have the path to do it.”
