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

Key Points

• Ocular Therapeutix remains on track to file for FDA approval of AXPAXLI in Q4 2026 for wet age-related macular degeneration, supported by positive SOL-1 Phase 3 results and safety data from the ongoing SOL-R study. The company is targeting a potential 2027 commercial launch.
• SOL-1 met the company’s stated FDA evidentiary requirements, including a p-value below 0.001, and showed fewer AXPAXLI-treated patients experienced significant vision loss versus EYLEA. Ocular Therapeutix also reported that roughly two-thirds of patients remained rescue-free one year after a single injection.
• The company is preparing manufacturing and commercial operations while pursuing additional opportunities in diabetic retinopathy. Ocular Therapeutix reported approximately $666 million in cash and said it is funded through 2028 on a conservative basis.

Ocular Therapeutix NASDAQ: OCUL said it remains on track to submit a U.S. marketing application for AXPAXLI in the fourth quarter of this year, following what Chief Executive Officer Pravin Dugel described as alignment with the Food and Drug Administration on the company’s planned submission package for wet age-related macular degeneration, or wet AMD.

Speaking at the Baird Healthcare Conference, Dugel said the company expects to seek approval for AXPAXLI with data from its SOL-1 Phase 3 trial and safety data from the ongoing SOL-R study. The company has said it is targeting a potential 2027 commercial launch.

FDA Submission Plan

Dugel said Ocular Therapeutix has maintained ongoing discussions with the FDA since the current management team joined roughly 2.5 years ago. He said the SOL-1 trial was conducted under a Special Protocol Assessment, or SPA, and that the agency confirmed in a Type C meeting that the company could pursue a single-trial submission if certain evidentiary requirements were met.

According to Dugel, the requirements included a properly masked and adequately powered trial before the clinical data cutoff, followed by a p-value below 0.001 and safety exposure in at least 300 patients at the time of submission. He said SOL-1 produced a p-value below 0.0006 and that Ocular Therapeutix expects to have the required safety database available.

“We checked all the boxes,” Dugel said. “We are in line with all the evidentiary standards.”

The company plans to obtain safety data from approximately 130 participants in SOL-R while keeping the study masked. Dugel said only safety information would be provided to an independent third party for transmission to the FDA, with Ocular Therapeutix not participating in that data-transfer chain. The company also plans to apply a small alpha-spending adjustment to protect the trial’s statistical integrity.

Dugel said preserving SOL-R’s integrity is particularly important for regulatory filings outside the U.S., even though the company does not view the study as necessary for a U.S. approval decision.

SOL-1 Results and Repeat-Dose Data

AXPAXLI is an investigational tyrosine kinase inhibitor delivered through Ocular Therapeutix’s proprietary hydrogel platform. Dugel said the product is intended to inhibit VEGF, a validated treatment target in wet AMD and diabetic retinopathy. The company’s objective is to reduce the treatment burden associated with frequent anti-VEGF injections while potentially improving longer-term disease control.

Dugel said SOL-1 compared a single injection of AXPAXLI with a single injection of EYLEA after a loading phase. The primary endpoint measured the percentage of patients who lost 15 or more letters of vision, a threshold that represents a doubling of the visual angle.

He said significantly fewer AXPAXLI-treated patients reached that vision-loss threshold than EYLEA-treated patients. Dugel also cited secondary findings that he said were clinically relevant, including OCT disease control within 30 microns at nine months after one injection and a result showing that about two-thirds of patients remained rescue-free at one year.

All SOL-1 participants were redosed at week 52, Dugel said. Ocular Therapeutix expects to provide the FDA with repeat-dosing information from SOL-1, as well as additional repeat-dose information from SOL-R. While the company has not discussed potential labeling with the FDA, Dugel said it hopes a future label could support repeat administration and dosing flexibility ranging from six to 12 months.

SOL-R and International Strategy

SOL-R is designed as a non-inferiority study, with high-dose EYLEA as a comparator. Dugel said the trial uses an extended run-in process intended to identify patients with stable disease before randomization. Participants receive three loading doses, followed by two observational periods; those showing disease fluctuations are excluded before randomization, he said.

The study will remain masked through its clinical data cutoff at week 96. Dugel said the company also sees the potential for a superiority result on measures that could include visual acuity, injection frequency, retinal thickness, atrophy or fibrosis, though he said it would be premature to predict such an outcome.

Commercial Readiness and Diabetic Retinopathy

Dugel said Ocular Therapeutix has retained and expanded its commercial team that markets DEXTENZA and has scaled manufacturing internally at its Bedford, Massachusetts, facility. He said the company has sufficient inventory to support a wet AMD launch and is increasing production in anticipation of a potential diabetic retinopathy opportunity.

In diabetic retinopathy, Dugel cited results from the HELIOS study, saying approximately 27% of control-arm patients experienced a vision-threatening complication by month 12, compared with zero in the AXPAXLI study arm after a single injection. He said the company’s registrational program includes patients with non-center-involving diabetic macular edema, but did not provide timing guidance for pivotal-study data.

Ocular Therapeutix reported approximately $666 million in cash in its latest earnings release and said it was funded into 2028 on what Dugel characterized as a conservative basis. Near-term milestones include the planned fourth-quarter submission and additional updates related to diabetic retinopathy, he said.

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 technology to design sustained-release treatments intended to deliver medication to ocular tissues over extended periods and potentially reduce the need for frequent eye drops or injections.

Its commercial product, Dextenza, is a resorbable intracanalicular insert that delivers dexamethasone to the ocular surface for the treatment of inflammation and pain following ophthalmic surgery.

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