Nacuity Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to NPI-001, the company’s investigational oral therapy for retinitis pigmentosa (RP). The designation recognizes early clinical evidence suggesting that NPI-001 may offer substantial benefit to patients with this progressive, inherited retinal condition..

Nacuity Pharmaceuticals 宣布，美国食品和药物管理局 (FDA) 已授予该公司在研口服药物 NPI-001 用于治疗视网膜色素变性（RP）的突破性疗法资格。该认定基于早期临床证据，表明 NPI-001 可能为这种进行性遗传性视网膜疾病患者带来显著益处。

A Novel Approach Targeting Oxidative Stress

一种针对氧化应激的新方法

NPI-001 is a GMP-grade formulation of N-acetylcysteine amide, designed to mitigate oxidative stress, a key factor contributing to photoreceptor degeneration in RP. According to Nacuity, preclinical data demonstrate that the therapy enhances glutathione levels, helping neutralize reactive oxygen species and protect retinal cells..

NPI-001是一种GMP级别的N-乙酰半胱氨酸酰胺制剂，旨在减轻氧化应激，这是导致RP中光感受器退化的关键因素。据Nacuity称，临床前数据表明，该疗法可提高谷胱甘肽水平，有助于中和活性氧物质并保护视网膜细胞。

“Breakthrough Therapy Designation represents an objective assessment by the FDA that early clinical evidence supports the potential of NPI-001 tablets to deliver substantial treatment effects for patients with retinitis pigmentosa, a serious blinding disease,” said G. Michael Wall, PhD, Senior Vice President and Chief Scientific Officer of Nacuity Pharmaceuticals.

“突破性疗法认定代表了FDA的客观评估，即早期临床证据支持NPI-001片剂对患有视网膜色素变性（一种严重的致盲疾病）的患者具有显著治疗效果的潜力，”Nacuity Pharmaceuticals高级副总裁兼首席科学官G. Michael Wall博士表示。

“This recognition represents a key value-creating milestone for Nacuity and underscores our commitment to efficiently advancing NPI-001 toward late-stage development.”.

“这一认可代表了Nacuity的一个关键价值创造里程碑，凸显了我们对高效推进NPI-001进入后期开发的承诺。”

Clinical Trial Results: Slowed Photoreceptor Loss

临床试验结果：减缓光感受器丧失

The designation follows results from a phase 1/2 randomized, placebo-controlled, double-masked, multicenter clinical trial (NCT04355689) involving 49 patients with RP across four sites in Australia. Participants received oral NPI-001 or placebo tablets twice daily for two years.

该指定是基于一项涉及澳大利亚四个地点的49名RP患者的1/2期随机、安慰剂对照、双盲、多中心临床试验（NCT04355689）的结果。参与者每天两次接受口服NPI-001或安慰剂片，持续两年。

Key findings included:

关键发现包括：

• More than 50% slower photoreceptor loss in the NPI-001 group compared to placebo over 24 months.

• 在24个月内，NPI-001组的光感受器损失速度比安慰剂组减缓了50%以上。

• While retinal sensitivity did not reach statistical significance, patients receiving NPI-001 experienced nearly 30% slower loss of visual function.

• 虽然视网膜敏感度未达到统计学显著性，但接受NPI-001治疗的患者视觉功能丧失速度减缓了近30%。

Regulatory Designations

监管指定

NPI-001 has also received Fast Track Designation, granted in early 2025, as well as Orphan Drug Designation, which provides seven years of U.S. regulatory exclusivity upon approval.

NPI-001 还获得了快速通道资格，该资格于 2025 年初授予，同时还获得了孤儿药资格，这将在获批后提供七年的美国监管独占期。