Developing novel therapeutics for Alzheimer’s disease and neurodegenerative disorders

Model: NRT-001 (J4)

Exhibitor: NEURAL REJUVENATION TEAM, ACADEMIA SINICA

Booth No: N915

Characteristic

1. Technology/Product/Service Name:
Developing novel therapeutics for Alzheimer’s disease and neurodegenerative disorders
2. Application Field/Indications: Alzheimer’s disease and neurodegenerative disorders
3. Introduction:
Alzheimer's Disease (AD) is the most common neurodegenerative disorder in aging societies, affecting millions of people worldwide. With the aging population, the number of patients is continually increasing, posing a growing healthcare challenge. Current clinical medications only provide symptomatic relief with limited efficacy and cannot significantly alter the progression of the disease. Therefore, finding new therapeutic approaches has become a major research focus. The pathogenesis of AD is complicated, in which brain energy deficit is a key factor contributing the occurrence of AD. J4, an equilibrative nucleoside transporter 1 (ENT1) inhibitor, offers a novel mechanism to normalize energy imbalance in the brain and treat neuronal energy deficits. Non-clinical experiments have demonstrated remarkable therapeutic effects of J4 in different AD mouse models, improving cognitive impairments and spatial memory deficits, reducing abnormal accumulation of Aβ and tau proteins, alleviating oxidative stress and neuroinflammation, and enhancing mitochondrial and glucose metabolism activity. J4 has shown superior efficacy compared to conventional treatments like donepezil and memantine. The research team has completed non-clinical pharmacokinetics, pharmacodynamics, and safety assessments of J4, and successfully obtained Investigational New Drug (IND) approvals from both the U.S. FDA and Taiwan FDA in 2024. The compound is currently undergoing a Phase I clinical trial in Taiwan. J4, an oral small-molecule drug with a novel mechanism of action, offers ease of administration and cost-effectiveness. Its strong efficacy and favorable safety profile provide significant potential for further development. J4 has been granted patents in multiple countries. The team plans to spin off into a startup company and collaborate with domestic and international pharmaceutical companies and research institutions to accelerate clinical development through resource raising or patent licensing. The development of J4 aims to provide Alzheimer’s disease patients with more treatment options, improve disease outcomes and quality of life, and reduce the long-term caregiving burden and healthcare costs for families.