Ocular Therapeutix reported exceptionally high physician interest in AXPAXLI as the company prepares to seek U.S. approval for the investigational treatment for wet age-related macular degeneration.
Company-sponsored market research indicated that approximately 80% of surveyed retina specialists would likely use a treatment with AXPAXLI’s profile.
More than 90% of the surveyed physicians were expected to adopt the treatment during its first year on the market, assuming it receives regulatory approval.
The findings suggest that Ocular believes AXPAXLI could achieve rapid acceptance among retina specialists because of its potential durability, sustained disease control, and compatibility with existing clinical practices.
However, the percentages represent physician responses to market research rather than actual prescriptions, revenue, reimbursement coverage, or commercial market share.
The eventual adoption rate will depend on the approved label, clinical data, pricing, insurance coverage, manufacturing capacity, physician experience, and competition from existing and emerging treatments.
AXPAXLI, also known as OTX-TKI, is an investigational bioresorbable intravitreal hydrogel containing axitinib.
Axitinib is a small-molecule tyrosine kinase inhibitor with anti-angiogenic properties. Ocular is developing AXPAXLI for wet AMD and diabetic retinal disease.
The treatment is designed to provide sustained drug delivery through a procedure that is similar to the intravitreal injections retina specialists already administer.
That familiarity could reduce the operational barriers associated with adopting a new therapy because physicians would not need to introduce surgery, additional steroids, or a substantially different administration process.
The potential reduction in injection frequency is central to the commercial opportunity.
Ocular said up to 40% of patients discontinue or become non-adherent to current wet AMD treatments during the first year, partly because frequent injections and office visits create a significant burden.
Missed treatments can allow disease activity to return and contribute to irreversible vision loss.
AXPAXLI is intended to provide more predictable and durable disease control while reducing the need for repeated anti-VEGF injections.
The physician-interest findings follow positive results from Ocular’s Phase 3 SOL-1 trial.
The study evaluated a single 0.45-milligram dose of AXPAXLI against a single two-milligram dose of aflibercept after patients completed an initial loading period.
At Week 36, 74.1% of AXPAXLI-treated patients maintained vision, compared with 55.8% in the aflibercept group.
At Week 52, 65.9% of patients receiving AXPAXLI maintained vision, compared with 44.2% of patients receiving aflibercept.
The SOL-1 trial met its superiority primary endpoint with a p-value of 0.0006.
Ocular said all six prespecified sensitivity analyses for the primary endpoint were statistically significant, while the first three hierarchically controlled secondary endpoints also achieved statistical significance.
Those results support management’s view that AXPAXLI could offer a differentiated profile rather than merely matching an established treatment.
Ocular is preparing to submit a New Drug Application for AXPAXLI during the fourth quarter of 2026.
The proposed application is expected to include SOL-1 efficacy and safety results, interim safety information from SOL-R, and additional confirmatory evidence.
Ocular plans to conduct an interim SOL-R safety analysis to provide data from more than 300 patients with at least one year of treatment exposure across SOL-1 and SOL-R.
The company also plans to hold a pre-NDA meeting with the FDA to confirm the format and contents of the application.
The FDA has indicated that it will consider an application based on the single pivotal SOL-1 efficacy trial together with confirmatory evidence.
Ocular intends to use the 505(b)(2) regulatory pathway, which could shorten the review period by as much as 60 days compared with the traditional pathway for a new molecular entity.
SOL-R efficacy results are no longer expected to be required for the initial application.
That allowed Ocular to keep the trial masked through Week 96 and add endpoints intended to provide additional differentiation after a potential approval.
SOL-R will evaluate whether AXPAXLI can demonstrate superior vision outcomes compared with eight-milligram aflibercept when both treatments are administered every 24 weeks.
The trial will also examine whether AXPAXLI can reduce fibrosis and atrophy compared with two-milligram aflibercept.
Topline SOL-R results are expected during the first quarter of 2028.
The extended study could provide important evidence about repeat dosing, long-term durability, anatomical outcomes, and how AXPAXLI compares with newer high-dose anti-VEGF treatments.
Ocular is also evaluating AXPAXLI in diabetic retinopathy through the Phase 3 HELIOS-3 trial.
The company streamlined the program to prioritize a potential single registrational study evaluating one AXPAXLI treatment every 12 months against sham injections.
The once-yearly regimen could create another substantial commercial opportunity because fewer than 1% of the estimated 6.4 million U.S. patients with non-proliferative diabetic retinopathy currently receive treatment.
Ocular attributes contribute to the low treatment rate partly due to the burden of repeated injections for patients who may not yet be experiencing meaningful vision loss.
The physician survey results indicate that retina specialists may be receptive to a durable treatment that fits into existing clinical workflows.
However, the actual commercial outcome will not be known until AXPAXLI receives approval, obtains reimbursement, and is used in routine clinical practice.
The high stated adoption expectations therefore provide evidence of physician interest, but they should not be treated as a forecast of guaranteed prescriptions or revenue.
Ocular’s next major milestones include its pre-NDA meeting, completion of the required safety analysis, and submission of the AXPAXLI application during the fourth quarter.
A successful review could position the company to introduce AXPAXLI in 2027, subject to FDA approval.
KEY QUOTES:
“Our market research shows retina specialists strongly favor a once-yearly regimen for rapid uptake in diabetic retinopathy.”
Pravin U. Dugel, MD, Executive Chairman, President And Chief Executive Officer Of Ocular Therapeutix
“That simplicity is key to broad and rapid adoption.”
Jeffrey S. Heier, MD, Chief Scientific Officer Of Ocular Therapeutix
