Ocular Therapeutix NASDAQ: OCUL Executive Chairman, President, and CEO Dr. Pravin Dugel said the company is engaged in formal discussions with the U.S. Food and Drug Administration as it works toward a New Drug Application submission for AXPAXLI in wet age-related macular degeneration (wet AMD), with the company aiming to support approval based on its SOL-1 phase 3 trial.
Speaking during an H.C. Wainwright virtual fireside chat hosted by equity research analyst Yi Chen, Dugel emphasized the company’s view that FDA leadership has signaled a “default position” favoring single-trial approvals when the pivotal study is rigorously designed. He also said the agency views the approach as “a raising of the bar,” calling for a “well-designed, well-masked, well-controlled study that’s properly powered.”
Regulatory pathway and SOL-1’s design
Dugel said Ocular has a Special Protocol Assessment (SPA) for SOL-1 and described the trial as aligned with FDA guidance issued in February 2023. He said the company has “begun the process of formal discussions with the FDA” and added that Ocular is “very, very happy with the way that the discussions are going,” while declining to provide a specific NDA submission timing.
He argued that SOL-1 provides what the FDA typically seeks for a pivotal trial: an “acceptable and excellent safety profile” and a primary endpoint met with “robust statistical significance.” Dugel noted the study was masked and conducted without a sham procedure, which he said is consistent with FDA guidelines and the SPA framework.
Asked about the risk the FDA could require a second pivotal trial given SOL-1’s superiority design, Dugel reiterated that Ocular’s strategy was to pursue what he called the potential for “the first and only superiority label” in this category, in contrast to what he described as a landscape of non-inferiority “me-too drugs.”
Label ambitions, clinical relevance, and adoption considerations
In discussing what might drive physician adoption, Dugel said retinal specialists may not focus on labeling language directly, but he contended a superiority label could matter indirectly by helping physicians choose a therapy “from day one” and by potentially reducing barriers such as step therapy. He also cited the potential for premium pricing protection if the product is differentiated.
Dugel declined to attribute SOL-1’s superiority to specific factors such as durability or rescue rates, saying the company was not yet in labeling discussions. Still, he repeatedly emphasized disease control measures that he said clinicians prioritize in practice, particularly optical coherence tomography (OCT) findings.
He said Ocular has provided additional analyses following SOL-1 to help explain the drug’s performance, arguing that while the primary endpoint is critical for regulators, physicians treat wet AMD based on disease control measures. Dugel highlighted what he described as OCT stability, saying that at nine months a single injection could provide disease control “within 30 microns of stability in a majority of patients,” which he called “absolutely unheard of.”
Safety profile and transparency
Dugel said AXPAXLI’s safety profile in SOL-1 meets the high bar set by existing anti-VEGF therapies, which he described as having “a superb safety profile.” He said Ocular went beyond typical disclosures by presenting safety information “from day one up to the patient level,” including every intraocular inflammation (IOI) event.
He also addressed questions raised around reported floaters, saying Ocular asked physicians to look for and code peripheral findings as floaters due to MedDRA coding limitations. According to Dugel, patients did not perceive the floaters, the findings appeared and resolved in a timing consistent with the drug’s presence, and there was “no impact on the visual acuity whatsoever.”
Dugel also stated that in Ocular’s dataset there were “no cases of endophthalmitis” and “no cases of ischemic or non-ischemic vasculitis.”
Ongoing trials: SOL-R, SOL-X, and diabetic retinopathy
Dugel said the company will continue running the SOL-R trial without changes, describing its execution as “going great.” While he said Ocular’s interactions with the FDA thus far suggest SOL-R may not be required for initial approval, he framed the study as a source of additional clinical information.
He also described SOL-X, the extension trial, as central to demonstrating long-term outcomes beyond durability. Dugel said AXPAXLI’s goals include reducing treatment dropout and improving long-term vision outcomes by avoiding what he called the “pulsatile nature” of repeated injections, which he linked to fibrosis and atrophy over time. He said SOL-X, which includes a crossover after two years of “pulsatile treatment,” is intended to evaluate whether continuous suppression can lead to better long-term outcomes, and he indicated more information would be shared at the company’s upcoming investor day.
On diabetic retinopathy, Dugel highlighted prior HELIOS study results in moderate to severe non-proliferative diabetic retinopathy (NPDR). He said control patients progressed to vision-threatening complications at a 30% to 40% rate, while after a single injection of AXPAXLI, the rate of vision-threatening complications after a year was “literally reduced to zero.” He argued that the primary barrier to treating NPDR broadly has been sustainability and follow-up, and suggested annual dosing could be feasible, with payer incentives driven by the high cost of blindness.
Commercial posture, manufacturing, IP, and partnering
On commercialization, Dugel said Ocular’s commercial team is led by David Robinson, whom he described as having led the launch of EYLEA, and noted the company already has an in-place team selling DEXTENZA. He said retina sales forces can be relatively small, citing his understanding that Lucentis’s sales force “consisted of 55 people,” and added that Ocular already has more than that based on DEXTENZA.
Dugel said the company is not currently considering partnerships or co-promotion deals for ex-U.S. markets, arguing Ocular does not need additional expertise or capital and intends to build a standalone company, while maintaining dialogue with potential strategic parties as part of his role.
He also said Ocular’s intellectual property is “ironclad till 2044” and that the company is filing additional IP related to scale-up and automation. On manufacturing, he said Ocular believes it can scale hydrogel-based products, noting the company manufactures the hydrogel and that DEXTENZA supports manufacturing continuity.
Asked about pricing relative to branded anti-VEGF therapies and the potential impact of biosimilars, Dugel said he did not believe biosimilar competition would affect AXPAXLI pricing “if it’s done properly,” again pointing to the potential significance of a superiority label. He added that Ocular has conducted payer advisory boards and that payers “love this” because of the cost burden of blindness, though he said it is too early for specific pricing discussions.
About Ocular Therapeutix NASDAQ: OCUL
Ocular Therapeutix, Inc is a biopharmaceutical company dedicated to the development of innovative therapies for diseases and conditions of the eye. Founded in 2011 and headquartered in Bedford, Massachusetts, the company focuses on sustained-release drug delivery platforms designed to address key unmet needs in ophthalmology. Its proprietary hydrogel-based inserts and sealants aim to improve patient compliance and outcomes by providing controlled release of active pharmaceutical ingredients directly to ocular tissues.
The company's flagship product, DEXTENZA®, is a preservative-free, sustained-release dexamethasone intracanalicular insert approved by the U.S.
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