Ocular Therapeutix Receives FDA Approval for Registrational Trial of AXPAXLI in NPDR, Plans to Outline Clinical Trial Design at Investor Day

Ocular Therapeutix has received FDA agreement under Special Protocol Assessment (SPA) for registrational trial of AXPAXLI in NPDR. The trial will focus on the treatment of non-proliferative diabetic retinopathy (NPDR). The company plans to outline clinical trial design, timing, and development strategy for NPDR and diabetic macular edema (DME) at its Investor Day on September 30, 2025. AXPAXLI has the potential to be a transformative approach to improving outcomes while preventing or substantially reducing the rate of vision-threatening complications in patients with diabetic retinopathy.

Ocular Therapeutix, Inc. (NASDAQ: OCUL) has received a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) granting a Special Protocol Assessment (SPA) agreement for the registrational trial of AXPAXLI in the treatment of non-proliferative diabetic retinopathy (NPDR). This agreement provides a clear regulatory pathway for AXPAXLI's development in diabetic eye disease, expanding its market potential beyond wet AMD.

The Phase 1 HELIOS trial results were promising, showing that no NPDR patients receiving AXPAXLI experienced disease progression or vision-threatening complications at 48 weeks, compared to 25% disease worsening and 40% vision-threatening complications in the control group. The treatment, designed for annual dosing, targets a substantial market with nearly 9 million Americans affected by diabetic retinopathy. The company will present detailed clinical trial design, timing, and development strategy for NPDR and diabetic macular edema (DME) at its upcoming Investor Day on September 30, 2025.

The FDA's SPA agreement for AXPAXLI represents a critical regulatory milestone, substantially derisking Ocular Therapeutix's development pathway in NPDR. This formal agreement essentially locks in the trial design, endpoints, and statistical analyses that will support a future approval application, eliminating significant regulatory uncertainty. The company can now proceed with confidence that their registration trial design meets FDA standards.

The expansion into diabetic eye disease opens a massive market opportunity. With nearly 9 million Americans affected by diabetic retinopathy and only about 1% currently receiving treatment, AXPAXLI could address a substantially underserved population. The current standard involves frequent anti-VEGF injections, which creates a significant treatment burden that many asymptomatic NPDR patients are unwilling to accept despite the risk of disease progression.

The preliminary Phase 1 HELIOS data mentioned is particularly compelling. At 48 weeks after a single AXPAXLI injection, 0% of treated NPDR patients showed disease progression or vision-threatening complications, compared to 25% disease worsening and 40% developing vision-threatening complications in the control group. This suggests potential for disease modification, not just symptom management. Moreover, all treated patients with non-center involved DME showed improvement, suggesting efficacy across the spectrum of diabetic eye disease. Annual dosing represents a paradigm-shifting advantage that could transform the treatment landscape from reactive to proactive care.

The potential to require just one injection per year versus the current standard of 6-12 injections annually could dramatically improve both treatment adherence and real-world outcomes while reducing healthcare system burden. Ocular plans to outline clinical trial design, timing, and development strategy for non-proliferative diabetic retinopathy (NPDR) and diabetic macular edema (DME) at its Investor Day on September 30, 2025.

References:
[1] https://www.stocktitan.net/news/OCUL/ocular-therapeutix-tm-receives-fda-agreement-under-special-protocol-tceh1k25uoad.html