眼科治疗开始对可以预防工作年龄糖尿病患者失明的药物进行重大试验

Ocular Therapeutix, Inc. (NASDAQ:OCUL, "Ocular"))), an integrated biopharmaceutical company committed to redefining the retina experience, today announced that the first patient has been randomized in the HELIOS-3 Phase 3 registrational program for AXPAXLI (also known as OTX-TKI) for the treatment of non-proliferative diabetic retinopathy (NPDR).

"Initiating the HELIOS registrational program marks a pivotal step toward redefining treatment for diabetic retinal disease. While there are more than 6 million NPDR patients in the U.S., fewer than 1% receive therapy today, due mostly to the burden of frequent injections in this working age population. AXPAXLI's potential to deliver efficacy with attractive durability could help hundreds of thousands, if not millions more patients preserve vision," said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix. "HELIOS-2 and HELIOS-3 are complementary superiority studies targeting a comprehensive and broad DR label. The HELIOS study designs reflect our intentional and strategic clinical philosophy: combining scientific rigor, outstanding clinical execution, and a focus on de-risking, with regulatory alignment to optimize the potential for success and a label that readily translates into broad adoptability. By aiming for a superiority-based DR label that spans the full continuum of disease – including NPDR and DME – we believe AXPAXLI could deliver a market opportunity that is not merely incremental, but transformative for patients, physicians, and payors around the world."

The HELIOS program is comprised of two complementary superiority studies, HELIOS-2 and HELIOS-3, designed to evaluate whether early AXPAXLI treatment, as infrequent as every 12 months, can meaningfully alter the course of non-proliferative diabetic retinopathy (NPDR). Ocular is targeting a broad diabetic retinopathy (DR) label with the HELIOS program by including patients with non-center-involved diabetic macular edema (non-CI-DME). The studies utilize a novel ordinal ≥2-step diabetic retinopathy severity score (DRSS) primary endpoint. HELIOS-2 is a superiority study comparing AXPAXLI dosed every 12 months to ranibizumab (0.3 mg), while HELIOS-3 compares 6- and 12-month dosing regimens of AXPAXLI to sham. The primary endpoint for each study is assessed at Week 52. Ocular has aligned with the FDA on the novel ordinal DRSS endpoint in its SPA agreement for HELIOS-2.

"The HELIOS program has the potential to transform care for patients with diabetic retinal disease, and my patients are excited to participate in this study. Current DR treatments are burdensome and unsustainable – particularly for this working age population, unlike wet AMD – requiring numerous intravitreal injections every year. This limits treatment uptake, leaving many at risk for disease progression and irreversible vision loss," commented Allen Hu, MD, Principal Investigator at Cumberland Valley Retina Consultants. "AXPAXLI shows significant promise to address the unmet need for an effective treatment with durable efficacy, based on the encouraging HELIOS-1 data. Given the rapid enrollment and outstanding execution of the SOL studies for AXPAXLI in wet AMD, I have full confidence that the Ocular clinical team will deliver another highly successful program in NPDR."

"The new ordinal ≥2-step DRSS endpoint Ocular has developed is an important and creative advancement for the retina field. This approach reflects real-world treatment goals, and I expect it will become the gold standard for DR trials going forward," said Dilsher S. Dhoot, MD, of California Retina Consultants. "Unlike binary endpoints, this ordinal measure captures changes across the full DRSS spectrum, including improvement, stability, and progression. By allowing every patient to contribute to the primary analysis, it enables more efficient trials while generating clinically meaningful data. If the HELIOS trials are successful, I expect AXPAXLI will be widely adopted across diabetic retinal disease due to its potential for durable efficacy and low treatment burden, making it highly attractive to patients, physicians, and payors alike."