Atsena Aligns with FDA on Regulatory Pathway for ATSN-201 in XLRS

Atsena就XLRS的ATSN-201监管途径与FDA达成一致

July 10, 2025

2025年7月10日

Atsena Therapeutics has announced a key regulatory milestone, reporting that the US Food and Drug Administration (FDA) has agreed to expand the ongoing phase 1/2 LIGHTHOUSE study of ATSN-201 into a continuous phase 1/2/3 trial. This change allows the study to serve as a pivotal trial supporting a future Biologics License Application (BLA) submission for the treatment of X-linked retinoschisis (XLRS).

Atsena Therapeutics宣布了一项关键的监管里程碑，报告称美国食品药品监督管理局（FDA）已同意将正在进行的ATSN-201一期/二期LIGHTHOUSE研究扩展为连续的一期/二期/三期试验。这一变更使该研究能够作为支持未来生物制品许可申请（BLA）的关键试验，用于治疗X连锁视网膜劈裂症（XLRS）。

Atsena anticipates submitting the BLA in early 2028..

Atsena 预计将在 2028 年初提交 BLA。

Accelerating the Path to Potential First Treatment for XLRS

加速通往XLRS潜在首个治疗方案的道路

“This regulatory milestone marks another significant step toward delivering a potentially first- and best-in-class gene therapy for patients living with XLRS,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “The agency’s agreement will expedite the clinical development of ATSN-201 by at least 1.5 years, compared to a separate phase 3 clinical trial.

“这一监管里程碑标志着为XLRS患者提供潜在的第一和最佳基因疗法又迈出了重要一步，” Atsena Therapeutics首席执行官Patrick Ritschel表示，“与单独进行的第三阶段临床试验相比，该机构的协议将使ATSN-201的临床开发至少加快1.5年。”

If approved, this would be the first available treatment for XLRS, offering hope to patients and families affected by this inherited retinal disease. We’re grateful for the FDA’s continued guidance as we advance this trial and prepare for filing a BLA in early 2028.”.

如果获得批准，这将是首个可用于治疗XLRS的疗法，为受这种遗传性视网膜疾病影响的患者和家庭带来希望。我们感谢FDA在我们推进这项试验并准备在2028年初提交BLA时给予的持续指导。”

Regulatory Support and Expanded Study Design

监管支持与扩展研究设计

ATSN-201 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the FDA. This recent regulatory advancement follows an RMAT meeting during which the agency agreed on the proposed study design, endpoints, and patient population to support potential registration..

ATSN-201 已获得 FDA 的再生医学先进疗法 (RMAT)、快速通道、罕见儿科疾病和孤儿药资格认定。这一最新的监管进展是在一次 RMAT 会议之后取得的，会议上该机构同意了拟议的研究设计、终点指标和患者人群，以支持潜在的注册申请。

The LIGHTHOUSE study will now include an additional cohort of approximately 30 adult and pediatric patients, randomized 1:1 between treatment and control groups. Patients in the control arm will have the opportunity to receive treatment after one year. The study will assess efficacy and safety across all patients using measures such as microperimetry, visual acuity, and macular structure..

LIGHTHOUSE 研究现在将增加一个额外的约30名成人和儿童患者的队列，以1:1的比例随机分配到治疗组和对照组。对照组的患者将在一年后有机会接受治疗。研究将通过微视野、视力和黄斑结构等指标评估所有患者的疗效和安全性。

The pivotal cohort is expected to begin enrollment in the first quarter of 2026, with pivotal data anticipated in the second half of 2027.

关键队列预计将于2026年第一季度开始招募，关键数据预计将在2027年下半年发布。

Promising Early Data and First Use of Novel Vector

早期数据令人鼓舞且新型载体首次使用

Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics, highlighted the program’s progress:

藤田健二医学博士，Atsena Therapeutics首席医学官，强调了该项目的进展：

“Data from the ongoing trial show that subretinal delivery of ATSN-201 has been well tolerated to date, including in patients with severe schisis cavities, and has led to encouraging improvements in both retinal structure and visual function. This program marks the first clinical use of our laterally spreading subretinal vector, offering important proof of principle for our ocular gene therapy platform.

“正在进行的试验数据表明，ATSN-201 的视网膜下递送迄今为止已被良好耐受，包括在严重裂隙腔患者中，并且在视网膜结构和视觉功能方面带来了令人鼓舞的改善。该计划标志着我们横向扩散视网膜下载体的首次临床应用，为我们的眼科基因治疗平台提供了重要的原理证明。”

With the study’s expansion, we’re positioned to generate the pivotal data needed to potentially bring the first treatment for XLRS across the finish line.”.

随着研究的扩展，我们有望生成关键数据，以潜在地推动首个XLRS治疗方法的研发走向成功。

About XLRS and the LIGHTHOUSE Study

关于XLRS和LIGHTHOUSE研究

XLRS is an inherited retinal disease typically diagnosed in early childhood, affecting approximately 30,000 males in the US and EU combined. There are currently no approved treatments for this condition.

XLRS是一种遗传性视网膜疾病，通常在儿童早期被诊断出来，影响美国和欧盟大约3万名男性。目前尚无针对该疾病的获批治疗方法。

The LIGHTHOUSE study is a dose-escalation and dose-expansion clinical trial enrolling male patients aged 6 and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for the study remains ongoing.

灯塔研究是一项剂量递增和剂量扩展临床试验，招募6岁及以上由RS1基因突变引起的XLRS临床诊断的男性患者。该研究的招募仍在进行中。