PD-001R: A First-in-Class Candidate for the Treatment of Neurodegenerative Diseases
Principle Investigators:
Prof. Simon LEE Ming-yuen, Cally Kwong Mei Wan Professor in Biomedical Sciences and Chinese Medicine Innovation, Chair Professor of Biomedical Sciences, Department of Food Science and Nutrition, Director of PolyU-BGI Joint Research Centre for Genomics and Synthetic Biology in Global Ocean Resources (RCOcean); Founder, AIM Pharmaceutical International Limited (a PolyU Start-up)
Dr ZHAO Chen, Postdoctoral Fellow, Department of Food Science and Nutrition; Chief Technology Officer, AIM Pharmaceutical International Limited (a PolyU Start-up)
A first-in-class, disease-modifying candidate for Parkinson’s disease (PD). Derived from Alpinia oxyphylla fruits and chemically synthesised via a novel scaffold, PD-001R activates the immunoproteasome to degrade pathological α-synuclein aggregates.
Preclinical studies demonstrate neuroprotection, reduced neuronal loss, mitigated dopamine depletion and improved motor, behavioural and cognitive function in PD and Alzheimer’s disease (AD) mouse models. Pharmacokinetic and toxicology studies in rats and beagles show rapid absorption, high oral bioavailability, blood–brain barrier penetration and favourable safety. CMC advances include GMP-aligned kilogramme-scale synthesis with IND-ready documentation. Patents in the US, EU, China and Japan cover PD-001R, its treatment claims and manufacturing. Preclinical work for PD IND submission is being finalised.
Key results: PD mice—50% motor improvement, 140% dopamine recovery, 32% higher substantia nigra neuron survival; AD mice—44% shorter water-maze latency, 78% longer target-quadrant time, 50% reduced Aβ₁₋₄₂; α-synuclein mice—60% degradation of Triton-insoluble α-syn and 56% increased dopaminergic neuron preservation.