NKGen Biotech presented encouraging Phase 1/2a data on troculeucel, an autologous natural killer (NK) cell therapy, at the 17th Clinical Trials on Alzheimer's Disease (CTAD) conference. The data suggest that troculeucel may reduce key biomarkers associated with Alzheimer's disease (AD) and improve cognitive function in patients with moderate AD.
Biomarker Analysis
Analysis of data from a Phase 1 dose-escalation trial revealed that troculeucel reduces levels of GFAP, NfL, p-tau181, GDF-15, and LTBP2. These biomarkers are detectable up to a decade before dementia symptoms manifest, suggesting a potential role for troculeucel in delaying or preventing dementia onset in asymptomatic individuals with detectable biomarkers. The research builds upon recent publications highlighting the early detection of these biomarkers, allowing for potential early intervention strategies.
Phase 1/2a Trial Results
In the Phase 1/2a trial, the highest dose of cryopreserved troculeucel (6 billion cells) was administered, demonstrating a strong safety profile with no adverse reactions reported. Preliminary analysis showed that 100% of the first three subjects treated at this dose experienced stable or improved cognitive function. Notably, two of the three subjects showed significant improvement, progressing from moderate-stage disease to mild cognitive impairment after only three months of treatment.
Impact on Moderate Alzheimer's Disease
According to NKGen Biotech, 35% of all Alzheimer’s patients have moderate-stage disease, representing a significant unmet medical need. There is currently no approved therapy to improve cognitive function, let alone stop or slow cognitive decline, in this patient population. The Phase 1/2a trial aimed to address this gap by evaluating the safety and efficacy of troculeucel in moderate AD subjects. The observed improvements in cognitive function and biomarker profiles offer a promising avenue for further research and potential therapeutic development.
Study Design and Next Steps
The Phase 1/2a trial is designed to evaluate the safety and efficacy of troculeucel in patients with moderate Alzheimer's disease. The Phase 1 portion of the study enrolled three subjects who received the highest dose of troculeucel to date. Based on the promising initial data, NKGen Biotech has initiated the Phase 2 portion of the trial, a double-blind, randomized, placebo-controlled study. This phase will further evaluate the safety and efficacy of troculeucel, with data updates expected as the study progresses. The company is also exploring the potential of troculeucel in other tau- and synuclein-related neurodegenerative diseases.
