August 30, 2025 — The first healthy subject in the Phase I clinical trial of Neu-001, an innovative drug jointly developed by the by the Centre for Regenerative Medicine and Health, Hong Kong Institute of Science & Innovation, Chinese Academy of Sciences (CRMH) and City University of Hong Kong (CityU), was dosed 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.
Clinical Trial Overview of Neu-001
The Phase Ia clinical trial of Neu-001 adopts a single-centre, randomized, double-blind, placebo-controlled, single ascending dose design. The study plans to enrol 53 healthy adult subjects, who will be evaluated across eight ascending dose cohorts starting from the lowest dose. The 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, after which it will advance to a multiple ascending dose study to further evaluate the drug’s safety, tolerability, pharmacokinetics, and preliminary efficacy in amblyopia patients.
A Milestone in Industry-Academia-Research Collaboration
Amblyopia is a common visual impairment disorder in children, affecting over 100 million patients globally. Current treatment options remain significantly limited. If subsequent clinical trials confirm the efficacy of Neu-001, the drug is expected to become the world’s first treatment for amblyopia leveraging a neuroplasticity mechanism, thereby reshaping the therapeutic landscape in this field.
Leveraging the clinical advancement of Neu-001 as an opportunity, CRMH will continue to deepen industry-academia-research collaborative innovation, accelerate the translation of scientific and technological achievements, and, with the support of the InnoHK initiative of the Innovation and Technology Commission of the Hong Kong Special Administrative Region Government, establish itself as a globally influential hub for biomedical and health technology innovation.
