Presenter of 1 Presentation
STUDY DESIGN AND PARTICIPANT CHARACTERISTICS OF A PHASE 2 TRIAL OF ATH-1017, A NOVEL TREATMENT FOR MILD-TO-MODERATE ALZHEIMER’S DISEASE
Abstract
Aims
To describe the ongoing ACT-AD study, a 26-week, randomized, double-blind, placebo-controlled study in mild-to-moderate Alzheimer’s disease (AD) with ATH-1017. ATH-1017 is a small-molecule enhancer of HGF/MET, a neurotrophic pathway impaired in AD. Neuronal MET expression is decreased in the cortex and hippocampus, which may contribute to synaptic loss, neurodegeneration, and functional decline. Enhancement of HGF/MET activity has the potential to restore neuronal connections and improve cognition. The ACT-AD study seeks to replicate and extend the previously shown improvement of ERP P300 latency, the primary endpoint and a non-invasive neurophysiological biomarker of cognitive performance.
Methods
ACT-AD has a target enrollment of 75 patients (aged 55-85, CDR 1 and 2, MMSE 14-24) in the US and Australia. Subjects are randomized 1:1:1 to placebo, 40 mg, or 70 mg dose of ATH-1017 with a once daily subcutaneous injection. In addition to ERP P300 latency and safety, ADAS-Cog11, ADCS-CGIC, ADCS-ADL23, COWAT, NPI, and other measures are protocoled. Summary statistics for demographic and baseline characteristics for the overall patient population are presented.
Results
The first 67 enrolled patients have a mean(SD) age of 71.9 (7.2), of whom 54% are female and 46% male, and 91% are white. Mean (SD) baseline MMSE score is 19.2 (3.0), with a minimum of 14 and a maximum of 24. ApoE4 carriers make up 56% of the population, with 44% as non-carriers. Data will be updated once enrollment is complete.
Conclusions
Enrolled subjects represent an appropriate population to evaluate the treatment effects of ATH-1017 on ERP P300.