Nanoscope Therapeutics has announced three-year follow-up data from the REMAIN study, the long-term extension of its pivotal Phase 2b/3 RESTORE trial investigating MCO-010, a proprietary optogenetic therapy developed to treat retinitis pigmentosa (RP). The new data highlight the durability of vision improvements and reinforce the long-term safety of the therapy..

Nanoscope Therapeutics宣布了REMAIN研究的三年随访数据，这是其关键的2b/3期RESTORE试验的长期延续，研究对象是该公司开发的专有光遗传学疗法MCO-010，用于治疗视网膜色素变性（RP）。新数据显示了视力改善的持久性，并进一步验证了该疗法的长期安全性。

Long-Term Vision Gains Sustained Through Week 152

长期视力提升持续至第152周

The REMAIN study showed that a single intravitreal injection of MCO-010 resulted in clinically meaningful and sustained vision improvements in RP patients for up to 152 weeks. Participants maintained an average Best Corrected Visual Acuity (BCVA) gain of approximately 0.3 LogMAR, equivalent to an improvement of three lines (15 letters) on a standard ETDRS chart..

REMAIN 研究表明，单次玻璃体内注射 MCO-010 在 RP 患者中带来了临床意义上的持续视力改善，效果长达 152 周。参与者平均最佳矫正视力 (BCVA) 提高了约 0.3 LogMAR，相当于在标准 ETDRS 视力表上提升了三行（15 个字母）。

Compared with sham-treated patients, the BCVA Area Under the Curve (AUC) data from the RESTORE and REMAIN trials demonstrated a fivefold greater vision gain in the MCO-010 group, underscoring the therapy’s durable efficacy.

与假手术治疗的患者相比，RESTORE 和 REMAIN 试验的 BCVA 曲线下面积 (AUC) 数据显示，MCO-010 组的视力提升是前者的五倍，凸显了该疗法的持久疗效。

“The sustained visual gains demonstrated over 3 years are truly remarkable for this population,” said Allen C. Ho, MD, Director of Retina Research at Wills Eye Hospital and Chief Medical Advisor to Nanoscope. “These patients typically lose about 1.5 lines of vision every five years and often hit legal blindness as early as age 20.

“这一群体在三年内持续展现出的视力改善确实令人瞩目，”威尔斯眼科医院视网膜研究主任、Nanoscope首席医学顾问Allen C. Ho博士说道。“这些患者通常每五年会失去大约1.5行视力，并且往往早在20岁就会陷入法定失明。”

The ability to not just slow loss but restore visual function for several years represents a significant therapeutic advance.”.

“这种不仅能够减缓视力丧失，而且还能在数年内恢复视力的能力代表了一项重大的治疗进展。”

Strong Safety and Tolerability Profile Maintained

良好的安全性和耐受性特征保持不变

Throughout the 152-week follow-up, MCO-010 continued to show a favorable safety and tolerability profile. No serious ocular adverse events were reported. Only one case of mild inflammation required short-term treatment with topical steroids, and 14 of 15 treated patients needed no ongoing inflammation management at the study’s final time point..

在整个152周的随访期间，MCO-010持续表现出良好的安全性和耐受性。没有报告严重的眼部不良事件。仅有一例轻度炎症需要短期使用局部类固醇治疗，在研究的最后时间点，15名患者中有14名无需持续的炎症管理。

Clinical and Regulatory Momentum

临床和监管势头

“These long-term results underscore the lasting impact of MCO-010 in patients who currently have no treatment options,” stated Sulagna Bhattacharya, CEO of Nanoscope Therapeutics. “As we advance our rolling Biologics License Application (BLA) with the FDA, we are preparing for a potential commercial launch of what could become the first approved optogenetic therapy for retinal disease.”.

“这些长期结果强调了MCO-010对目前尚无治疗选择的患者的持久影响，”Nanoscope Therapeutics首席执行官Sulagna Bhattacharya表示。“随着我们推进与FDA的滚动生物制品许可申请（BLA），我们正在为可能推出首个获批的视网膜疾病光遗传学疗法做准备。”

She also noted that the durability observed in REMAIN highlights the broader promise of the MCO platform as a one-time treatment for a wide range of retinal degenerative diseases.

她还指出，REMAIN 研究中观察到的持久性突显了 MCO 平台作为一次性治疗多种视网膜退行性疾病的广阔前景。

MCO Platform: Intravitreal Therapy for Retinal Degenerative Conditions

MCO平台：视网膜退行性疾病的玻璃体内治疗

Nanoscope’s Multi-Characteristic Opsin (MCO) platform is a disease-agnostic, optogenetic therapy designed to restore vision in patients with advanced retinal degenerative diseases, including retinitis pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA).

纳米镜公司的多特性视蛋白（MCO）平台是一种与疾病无关的光遗传学疗法，旨在恢复晚期视网膜退行性疾病患者的视力，包括视网膜色素变性（RP）、斯塔加特病（SD）和地理萎缩（GA）。

Delivered via a one-time intravitreal injection, MCO-010 activates bipolar retinal cells to become light-sensitive, bypassing degenerated photoreceptors and restoring visual function. The platform is designed to function without the need for genetic testing, avoids invasive surgery, does not require repeat dosing, and is compatible with standard retina clinic workflows, making it a practical option for broad clinical adoption..

通过一次性玻璃体内注射给药，MCO-010激活双极视网膜细胞使其对光敏感，绕过退化的感光细胞并恢复视觉功能。该平台设计无需基因测试，避免了侵入性手术，不需要重复给药，并且与标准视网膜诊所工作流程兼容，成为广泛临床应用的实用选择。

Regulatory Designations Support MCO-010’s Advancement

监管指定支持MCO-010的进展

MCO-010 has received both Fast Track and Orphan Drug Designations from the FDA. In Europe, the EMA has granted five Orphan designations for MCO-010 across multiple retinal degenerative indications, supporting its potential as a first-in-class treatment.

MCO-010 已获得 FDA 的快速通道和孤儿药资格认定。在欧洲，EMA 已授予 MCO-010 在多个视网膜退行性适应症上的五个孤儿药资格认定，支持其作为首创疗法的潜力。