NeuDirection Limited (“NeuDirection”) announced that the first healthy subject has been successfully dosed in the Phase I clinical trial for its lead pipeline candidate, Neu-001, a first-in-class small molecule drug for the treatment of amblyopia. The first dose was administered on August 30, 2025, at Shanghai General Hospital. This study aims to evaluate the safety, tolerability, and pharmacokinetic profile of Neu-001 in healthy adults, providing critical data for subsequent clinical development.
The Phase Ia clinical trial of Neu-001 is a single-center, randomized, double-blind, placebo-controlled, Single Ascending Dose (SAD) study. The study plans to enroll 53 healthy adult subjects to be evaluated across eight ascending dose cohorts. The successful administration of the first dose marks the official entry of Neu-001 into the clinical development stage. According to the current plan, this Phase Ia study is expected to be completed in the fourth quarter of 2025. Subsequently, the Phase Ib study will advance to a Multiple Ascending Dose (MAD) study to further evaluate the drug’s safety, tolerability, pharmacokinetics, and preliminary efficacy in amblyopia patients.
Neu-001 is a small molecule drug developed to target a novel GPCR for the treatment of both pediatric and adult amblyopia. As a common visual impairment in children, amblyopia currently has no FDA-approved pharmacological treatments. Conventional therapies, such as eye-patching, have significant limitations including poor compliance, inconsistent efficacy, and a restricted age window for treatment. This leaves a major unmet need, especially for adult patients who have passed the “critical period” for treatment. With its unique mechanism of enhancing neuroplasticity, Neu-001 has the potential to become the world’s first drug to treat amblyopia by breaking the age barrier, thereby reshaping the therapeutic landscape.
NeuDirection is a spin-off from the Centre for Regenerative Medicine and Health (CRMH) of the Hong Kong Institute of Science & Innovation (HKISI), Chinese Academy of Sciences (CAS), and City University of Hong Kong (CityUHK). The company was co-founded by Professor He Jufang, a leading expert in neuroscience from CityUHK, and Professor Micky Tortorella, who brings decades of drug development experience from global pharmaceutical companies. Professor He’s pioneering research in neuroplasticity serves as the scientific cornerstone of the Neu-001 pipeline. Anchored in the innovation ecosystem of the Hong Kong Science Park, NeuDirection has assembled a professional and diverse team that deeply integrates cutting-edge neuroscience discoveries with seasoned drug development expertise. The successful initiation of this clinical trial marks a major breakthrough for the company in its transition from scientific research to clinical application.
Leveraging the clinical advancement of Neu-001, NeuDirection will continue to deepen its industry-academia-research collaboration and accelerate the translation of scientific achievements. Supported by the Hong Kong SAR Government’s InnoHK innovation platform, the company is dedicated to providing solutions for neglected neurological diseases worldwide, with the goal of building a globally influential biopharmaceutical enterprise.