Ocular Therapeutix eyes Q4 AXPAXLI NDA after FDA alignment

Ocular Therapeutix (NASDAQ:OCUL) said it remains on track to submit a New Drug Application for AXPAXLI in wet age-related macular degeneration in the fourth quarter, after what Executive Chairman, President and CEO Dr. Pravin Dugel described as alignment with the U.S. Food and Drug Administration on the planned filing package.

Speaking during a fireside chat at the H.C. Wainwright 6th Annual Ophthalmology Virtual Conference, Dugel said the company's recent FDA interactions were the result of more than two years of work and not a sudden change in regulatory strategy.

"What we formalized with the FDA was that we meet all of these evidentiary standards," Dugel said. "Therefore, what we will be submitting in the fourth quarter of this year constitutes a complete package submission for approval."

FDA Submission Pathway

Dugel distinguished between the FDA accepting a proposed package for submission and making an approval decision, which remains subject to agency review. He said the company believes it has met the evidentiary standards for a single-trial approval pathway, including study masking, statistical powering, a p-value threshold and safety exposure requirements.

According to Dugel, SOL-1 was conducted under a Special Protocol Assessment, or SPA, and he said the study met pre-specified standards for masking and powering. He also said the trial achieved a p-value below 0.0006, compared with the standard he cited of less than 0.001, and that Ocular can present at least 300 patients exposed to the drug by including patients from SOL-R for safety purposes.

Dugel said the FDA's written minutes stated that the company has "very compelling data" and that the agency would accept the company's package as a complete submission. He repeatedly emphasized, however, that the FDA review team will still conduct its review after submission.

Pre-NDA Meeting Described as Operational

Ocular Therapeutix expects a pre-NDA meeting in the third quarter. Dugel characterized that meeting as operational rather than substantive, saying it would address filing logistics such as table formatting, locations, font sizes and hyperlinks.

He said poor organization of those materials could cause delays of two to four weeks if reviewers require corrections, but he said the meeting would not involve content decisions. The company continues to expect a complete NDA submission in the fourth quarter.

Dugel declined to guide on whether the application might receive standard or priority review, whether an advisory committee would be convened, or the approximate review timeline, saying those decisions are up to the FDA.

505(b)(2) Pathway and Manufacturing

Dugel said the FDA has formally agreed that Ocular will use the 505(b)(2) pathway for AXPAXLI. He said that pathway could save time because the FDA has already approved the two components of the product: the tyrosine kinase inhibitor and the hydrogel.

He also pointed to Ocular's existing commercial product Dextenza, saying it uses the same hydrogel as AXPAXLI. Dugel said Dextenza has undergone multiple FDA reviews since commercialization and that the company has not had issues with the hydrogel in those reviews.

"We'll satisfy all of their requirements," Dugel said in reference to chemistry, manufacturing and controls. "We're ready to do that."

Label Expectations and SOL-1 Data

Dugel said it is too early for formal labeling discussions but said Ocular expects AXPAXLI could receive what he described as a "first and only superiority label," with dosing flexibility every six to 12 months and repeat dosing language. He said patients in SOL-1 were redosed at week 52 and that SOL-R patients included for safety would also have redosing experience.

Responding to questions about the SOL-1 trial design, which compared AXPAXLI with a single injection of aflibercept 2 mg, Dugel said the company followed FDA guidance for a superiority study and that the trial design was validated through the SPA.

He said SOL-1 provided evidence physicians would consider relevant, including safety, durability and disease control. Dugel said about two-thirds of patients were rescue-free after one year with a single injection and that the majority of patients showed optical coherence tomography stability within 30 microns at month nine.

Dugel also addressed safety findings, saying the trial reported no cases of endophthalmitis or retinal vasculitis through week 52. He said reported vitreous floaters were actually drug particles, coded as floaters because there was no MedDRA code for drug particles. He said the particles were far from the visual axis, were observed by physicians rather than patients, had no impact on vision and resolved over time.

Commercial Readiness and Capital Position

Dugel said Ocular already has commercial infrastructure in place through Dextenza and highlighted David Robinson, whom he described as the leader for the planned launch and the architect of EYLEA's launch. He said the company is preparing for wet AMD and diabetic retinal disease opportunities.

On financing, Dugel said the company had previously reported $667 million in cash at the end of the first quarter and remains "very well capitalized." He said Ocular has conservatively stated it is financed into 2028 and through launch, and that outlook has not changed.

Dugel closed by saying the company is in a "fantastic position" and reiterated that its regulatory strategy is based on a lengthy period of interaction with the FDA, supported by the SPA and preparations for a potential commercial launch.

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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