NeuDirection announces that its innovative drug Neu-001 for the treatment of amblyopia has successfully completed its Phase I clinical trial. The trial results demonstrated favorable safety and tolerability profiles, establishing a solid foundation for further clinical development.
Neu-001 is a highly selective GPCR agonist capable of crossing the blood-brain barrier. By activating neuroplasticity in the visual cortex, it offers a potential new pharmacological approach for adult amblyopia patients. Current amblyopia treatment relies primarily on patching therapy during childhood, leaving adult patients with limited effective options. Neu-001 has the potential to address this unmet clinical need.
The Phase I clinical trial was conducted at the Phase I Clinical Trial Center of Shanghai General Hospital, using a single-center, randomized, double-blind, placebo-controlled design. The trial was initiated in August 2025 and completed data review in December, enrolling a total of 53 healthy adult subjects across 8 dose groups (0.002 mg to 0.3 mg).
Regarding safety, the incidence of adverse events was 65% in the Neu-001 group compared to 53.85% in the placebo group, with no significant difference between the two groups. Adverse events in the Neu-001 group were predominantly Grade 1, mainly manifesting as mild changes in laboratory parameters, with no observed dose-related increase in adverse events. Pharmacokinetic data showed rapid absorption, with time to peak concentration (Tmax) of approximately 10 to 30 minutes. Peak concentration (Cmax) and area under the curve (AUC) increased proportionally with dose, demonstrating favorable linear pharmacokinetic characteristics.
On January 28, a symposium on Neu-001 Phase I Clinical Trial and Innovative Drug Development was successfully held in Hong Kong, hosted by the Centre for Regenerative Medicine and Health (CRMH) of the Hong Kong Institute of Science & Innovation, Chinese Academy of Sciences. Dr. Zhang Mingjian from Shanghai General Hospital presented a detailed report on the Phase I clinical trial results. Attending experts engaged in in-depth discussions on the data and began planning for the Phase IIa clinical trial protocol.
Based on the positive Phase I results, NeuDirection plans to initiate the Phase IIa clinical trial as soon as possible to further evaluate the safety and preliminary efficacy of Neu-001 in amblyopia patients.