Ocular Therapeutix Randomizes First Patient in HELIOS-3 Phase 3 Study for AXPAXLI in NPDR

Ocular Therapeutix 在HELIOS-3第三阶段研究中为NPDR的AXPAXLI随机选定了首位患者。

November 25, 2025

2025年11月25日

Ocular Therapeutix has announced the randomization of the first patient in HELIOS-3, the second Phase 3 registrational study evaluating AXPAXLI (OTX-TKI) for the treatment of nonproliferative diabetic retinopathy (NPDR). The HELIOS clinical program is designed to assess AXPAXLI’s potential in providing long-lasting treatment effects with less frequent dosing..

Ocular Therapeutix 宣布了在HELIOS-3试验中首位患者已完成随机分组，这是评估AXPAXLI（OTX-TKI）治疗非增殖性糖尿病视网膜病变（NPDR）的第二项III期注册研究。HELIOS临床项目旨在评估AXPAXLI以较少频率给药提供持久治疗效果的潜力。

HELIOS Program Structure and Study Design

HELIOS项目结构与研究设计

The HELIOS program includes two complementary superiority trials:

HELIOS 计划包括两项互补的优效性试验：

• HELIOS-2:

• HELIOS-2:

Compares AXPAXLI administered every 12 months to ranibizumab (0.3 mg)

每12个月给药一次的AXPAXLI与雷珠单抗（0.3 mg）进行比较

• HELIOS-3:

• 赫利俄斯-3：

Compares 6- and 12-month dosing of AXPAXLI to sham treatment

比较AXPAXLI六个月和十二个月的剂量与假治疗

Both studies use a novel ordinal ≥2-step diabetic retinopathy severity score (DRSS) as the primary endpoint. This endpoint is designed to evaluate whether infrequent treatment with AXPAXLI can significantly impact the progression of NPDR.

两项研究均使用一种新的序数≥2级糖尿病视网膜病变严重程度评分（DRSS）作为主要终点。该终点旨在评估使用AXPAXLI进行低频率治疗是否能显著影响NPDR的进展。

Company Perspective on AXPAXLI’s Role in Diabetic Retinopathy

公司对AXPAXLI在糖尿病视网膜病变中作用的看法

According to Pravin U. Dugel, MD, Executive Chairman, President, and CEO of Ocular Therapeutix, the initiation of HELIOS-3 is a key step in the development of AXPAXLI:

根据Ocular Therapeutix的执行主席、总裁兼首席执行官Pravin U. Dugel博士的说法，HELIOS-3的启动是AXPAXLI开发中的关键一步：

“Initiating the HELIOS registrational program marks a pivotal step toward redefining treatment for diabetic retinal disease. AXPAXLI’s potential to deliver efficacy with attractive durability could help hundreds of thousands, if not millions more patients preserve vision.”

“启动HELIOS注册计划标志着重新定义糖尿病视网膜疾病治疗的关键一步。AXPAXLI在提供疗效和持久性方面的潜力，可以帮助成千上万，甚至数百万患者保留视力。”

“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.”

“通过追求涵盖整个疾病连续体（包括NPDR和DME）的基于优越性的DR标签，我们相信AXPAXLI能够带来不仅仅是渐进式的，而是对全球患者、医生和支付方而言具有变革意义的市场机会。”

Investigator Commentary: Addressing Treatment Burden

研究人员评论：解决治疗负担问题

Allen Hu, MD, Principal Investigator at Cumberland Valley Retina Consultants, commented on the relevance of the HELIOS program in clinical practice:

Cumberland Valley Retina Consultants 的首席研究员胡艾伦博士评论了 HELIOS 项目在临床实践中的相关性：

“The HELIOS program has the potential to transform care for patients with diabetic retinal disease. Current DR treatments are burdensome and unsustainable – particularly for this working age population, unlike wet AMD – requiring numerous intravitreal injections every year.”

“HELIOS计划有可能改变糖尿病视网膜病变患者的护理。目前的糖尿病视网膜病变治疗方法繁琐且不可持续——特别是对于这个工作年龄的人群，不同于湿性AMD——每年需要进行多次玻璃体内注射。”

“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.”.

“基于令人鼓舞的HELIOS-1数据，AXPAXLI显示出显著的潜力，可以满足对具有持久疗效的有效治疗的未满足需求。鉴于AXPAXLI在湿性AMD的SOL研究中迅速招募和出色的执行，我完全相信眼科临床团队将在NPDR领域再次交付一个非常成功的项目。”

Use of Ordinal DRSS Endpoint in Clinical Trials

临床试验中序数DRSS终点的使用

The HELIOS studies introduce an ordinal ≥2-step DRSS endpoint, which differs from traditional binary endpoints. Dilsher S. Dhoot, MD, of California Retina Consultants, discussed the significance of this approach:

HELIOS 研究引入了一个序数 ≥2 级 DRSS 终点，这与传统的二元终点不同。加州视网膜顾问公司的迪尔舍尔·S·杜特医学博士讨论了这种方法的重要性：

“This new DRSS endpoint reflects clinical goals more effectively and may offer greater sensitivity in capturing changes in disease severity. Unlike binary measures, it evaluates improvement, stability, and progression, allowing every patient to contribute to the analysis.”

“这个新的DRSS终点能更有效地反映临床目标，并且在捕捉疾病严重程度的变化时可能提供更高的敏感性。与二元指标不同，它评估改善、稳定和进展，让每位患者都能为分析做出贡献。”

“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.”

“如果HELIOS试验成功，我预计AXPAXLI将因持久的疗效和较低的治疗负担而在糖尿病视网膜病变中得到广泛应用，这对患者、医生和支付方都非常有吸引力。”