Ocular Therapeutix (NASDAQ:OCUL) President and CEO Pravin Dugel said the company remains focused on gaining U.S. Food and Drug Administration approval for AXPAXLI and intends to seek approval based on its pivotal SOL-1 trial alone, while continuing discussions with the agency and maintaining its SOL-R study.
Speaking during a company session, Dugel repeatedly emphasized that Ocular’s regulatory strategy is based on alignment with FDA guidance issued in February 2023. He said the agency’s position was shaped by concerns that sham controls do not provide proper masking in retina studies and that superiority trials should not be based on drug labels that do not reflect how physicians use anti-VEGF therapies in practice.
Company Points to SOL-1 Results and FDA Alignment
Dugel said Ocular followed the FDA’s guidance “precisely” in designing SOL-1, which he described as a head-to-head superiority study. He said the company has a Special Protocol Assessment, or SPA, with the FDA, calling it “the highest level of agreement that you can get with the FDA.” He also said Ocular has two SPAs.
According to Dugel, SOL-1 met the FDA’s evidentiary standard for efficacy, with a p-value of less than 0.0006, compared with what he described as an FDA threshold of less than 0.001. He also said the study showed a “completely clean safety profile.”
From a clinical perspective, Dugel highlighted two measures he said are important to retina specialists: disease control and durability. He said SOL-1 showed optical coherence tomography stability within 30 microns at month nine after a single injection in a majority of patients, and that two-thirds of patients were rescue-free at month 12 after a single injection.
“We are more confident than ever that we will be approved with a single trial,” Dugel said. “We will be not only best in class, but first in class.”
Dugel Says Physicians Will Focus on Durability and Safety
Asked how retina specialists might compare AXPAXLI with other drugs studied under different trial designs, Dugel said physicians do not typically use retina drugs according to the exact label or clinical trial regimen. He argued that doctors will focus on whether the drug controls disease, how long it lasts and whether it is safe.
Dugel said he expects AXPAXLI to be adopted quickly if approved because it would not require changes to physician workflow. He described the product as using a “self-sealing needle at 25 gauge” and said the treatment experience would be similar to existing intravitreal therapy.
He said physicians may differ in how they initially use the product, with some potentially moving to a fixed six-month regimen and others using treat-and-extend approaches. Dugel said his view is that AXPAXLI is suited to every-six-month dosing because a drug used on that schedule should last nine to 10 months to provide flexibility if patients or physicians miss visits.
Label Strategy and SOL-R
Dugel said Ocular continues to have “ongoing formal discussions” with the FDA regarding a single-trial approval strategy. He described four evidentiary standards that he believes the company has met: proper masking, adequate power, a p-value below 0.001 and at least 300 patients available for safety review at the time of submission.
Regarding repeat dosing and label language, Dugel said Ocular will discuss its labeling strategy “when appropriate,” but added that there is redosing experience in both SOL-1 and SOL-R. He said the company’s goal is to obtain “a superiority label with flexibility of dosing every 6-12 months with repeatability.”
Dugel said SOL-R will continue unchanged. While he reiterated that Ocular intends to file based on SOL-1 alone, he said SOL-R may provide flexibility on safety data, commercial leverage and information useful for regulatory agencies outside the United States. He also noted that SOL-R includes a head-to-head comparison with EYLEA HD, though he said it is without statistical analysis.
SOL-X and Longer-Term Outcomes
Dugel also discussed SOL-X, saying execution and retention are “fantastic.” He said the study is intended to address both durability and longer-term outcomes. In particular, he said Ocular will evaluate visual acuity in patients who cross over after two years of pulsatile therapy.
He said the company believes patients treated with repeated anti-VEGF therapy may not fully catch up after crossing over because of fibrosis and atrophy, which he said can be detectable as early as 90 days. Dugel said Ocular believes SOL-X can show maintained vision and improved long-term outcomes related to fibrosis and atrophy compared with long-term anti-VEGF studies.
Diabetic Retinal Disease Opportunity
Asked about opportunities beyond wet macular degeneration, Dugel said Ocular believes AXPAXLI could be used once yearly in diabetic retinal disease, a market he described as three and a half times the size of wet macular degeneration.
He said the company believes it can obtain a broad label covering all diabetic retinal disease, noting that patients with non-center-involving diabetic macular edema are being recruited. He also referred to the earlier HELIOS study, saying that if future results duplicate those findings, the therapy could reduce the risk of vision-threatening complications “literally to zero.”
Dugel closed by thanking the host for the opportunity to discuss the company’s plans, while reiterating Ocular’s focus on advancing AXPAXLI toward FDA approval.
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.
