Ocular Therapeutix Eyes Q4 FDA Filing for AXPAXLI After SOL-1 Win

Key Points

• Ocular Therapeutix plans to file an FDA application for AXPAXLI in Q4 after its SOL-1 trial showed superiority over an approved anti-VEGF therapy. The company says it has aligned with the FDA on a single-trial 505(b)(2) pathway.
• AXPAXLI is designed as a sustained maintenance treatment for wet age-related macular degeneration, aiming to reduce the burden of repeated injections and address treatment dropouts and long-term vision loss.
• The company reported nearly $600 million in cash as of June 30 and expects its existing capital to last into 2028, although it may need additional funding to support commercialization.

Ocular Therapeutix NASDAQ: OCUL is preparing a fourth-quarter new drug application submission for AXPAXLI, its investigational maintenance treatment for wet age-related macular degeneration, following discussions with the U.S. Food and Drug Administration, President and Chief Executive Officer Pravin Dugel said during a fireside chat.

Dugel said the company’s SOL-1 trial showed superiority against an approved anti-VEGF therapy, describing it as the first time a novel agent had met such a superiority endpoint in wet AMD. He said the company has formalized a single-trial approval pathway with the FDA and remains on schedule to submit a full 505(b)(2) application package during the fourth quarter.

Addressing treatment durability in wet AMD

AXPAXLI is a tunable hydrogel containing a tyrosine kinase inhibitor, or TKI, designed as a maintenance treatment rather than a traditional bolus injection, according to Dugel. He said the program is intended to address the burden of repeated anti-VEGF injections and the loss of vision that can occur when patients discontinue treatment or experience long-term disease progression.

“In this country alone, in the first year, there is a 40% dropout rate,” Dugel said. “All of these patients who drop out, almost half of them end up going blind.”

He also said that even patients who remain on therapy can lose vision over time because of atrophy and fibrosis. Ocular Therapeutix believes sustained treatment could reduce those challenges, he said.

Dugel characterized AXPAXLI as part of a maintenance-drug category that is distinct from bolus injection treatments. He said the company does not view products such as high-dose aflibercept, faricimab, or Kodiak Sciences’ tarcocimab as direct competitors because those therapies are delivered as bolus injections.

Clinical-trial design and competitive developments

The discussion followed disappointing Phase III results reported by EyePoint Pharmaceuticals for its DURAVYU program. Dugel said there was “no joy” in another company’s failure, but argued that the outcome highlighted the importance of patient selection and trial design in retinal diseases.

He said retinal conditions such as wet AMD, diabetic macular edema and retinal vein occlusion can vary substantially among patients, while the field lacks established biomarkers or genetic markers to stratify disease. According to Dugel, Ocular Therapeutix selected different patient populations for its SOL-1 and SOL-R studies.

For SOL-1, the company used treatment-naive patients and provided loading treatment before randomization, Dugel said. He said this approach was designed to identify an appropriate patient population for the trial. Ocular Therapeutix is also using what he described as a different, stable patient population in SOL-R.

Dugel said he believes EyePoint’s results reflected both what he called a weaker TKI and patient-selection issues. He noted that EyePoint included both previously treated and treatment-naive patients in its studies before randomizing and loading patients, contrasting that approach with Ocular Therapeutix’s design.

On Kodiak’s DAYBREAK data, Dugel said more details would be needed to assess the results, including which patients reached six-month dosing, the number of injections received, retreatment requirements, adverse-event data and the regulatory path for a pro re nata, or as-needed, study arm.

FDA discussions and filing plans

Dugel said Ocular Therapeutix held a Type C meeting with the FDA in May and a pre-NDA meeting in September. The company has received minutes from the pre-NDA meeting and is “in lockstep” with the agency, he said.

He pointed to the statistical result from SOL-1, saying the FDA’s definition of a robust study included a p-value below 0.001, while the company reported a p-value below 0.0006. Dugel did not identify outstanding filing requirements and said the company had checked the evidentiary standards needed for its planned single-trial submission.

When asked about the possibility of an FDA advisory committee meeting, Dugel said it was too early to discuss the issue. In his view, an advisory committee should not be necessary because the treatment is intended to suppress VEGF, an approach used by anti-VEGF medicines for decades.

The company amended SOL-1 to add a key secondary endpoint measuring superiority versus 8-milligram aflibercept at week 96 and extended masking, moving topline data to the first quarter of 2028 from the first quarter of 2027. Dugel said the decision avoided the risk of submitting a major amendment during FDA review that could slow the approval process.

Capital position

Ocular Therapeutix had nearly $600 million in cash as of June 30 and has projected its existing capital runway into 2028. Dugel said the company’s guidance does not include the full cost of commercializing AXPAXLI, but said Ocular Therapeutix has built a team intended to support a launch.

He did not provide an estimate of additional launch funding needs. However, he said the company could access “all kinds of flexible ways” to raise capital, if needed, without issuing dilutive equity.

About Ocular Therapeutix (NASDAQ:OCUL)

Ocular Therapeutix, Inc is a biopharmaceutical company focused on developing and commercializing therapies for diseases and conditions affecting the eye. The company uses its proprietary hydrogel-based formulation and delivery technologies to create sustained-release treatments intended to improve drug delivery, reduce dosing frequency and address unmet needs in ophthalmology.

Its commercial product is DEXTENZA, a corticosteroid ophthalmic insert that is placed in the canaliculus by a healthcare professional.

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