Anavex Life Sciences Corp (AVXL)

[A_Good_Day_2]

Abstract:
15-MONTHS SAFETY AND EXPLORATORY EFFICACY DATA OF ANAVEX 2-73 IN A PHASE 2A STUDY IN MILD-TO-MODERATE ALZHEIMER’S DISEASE PATIENTS

Aims
ANAVEX2-73, a selective sigma-1 and muscarinic receptor agonist was tested in a Phase 2a-study in patients with mild-to-moderate AD. Adverse Events (AEs) were recorded for assessment of safety and maximum tolerated dose (MTD). Cognitive (MMSE, Cogstate, QEEG/ERP) and the functional (ADCS-ADL) marker were measured so as to establish a functional relationship between dosing regimen and exploratory efficacy outcomes.

Method
Thirty-two AD patients 55-85 years old with MMSE 16-28 were recruited. After establishing MTD and dose-response all patients received ANAVEX2-73 between 10mg and 50mg daily orally and re-assessed every 3 months. For safety, mathematical modeling was fitted to the recorded AEs to establish the dose-risk relationship. For efficacy, statistical hypothesis tests were performed on cognitive and functional markers and compared to the corresponding baselines.

Results

Safety: ANAVEX2-73 demonstrated a favorable safety profile in AD patients. The most common AEs were grade 1. Positive unexpected therapeutic response events, such as improved mood, improved social engagement and increased independent activities were recorded.

Exploratory Efficacy: 15-months data demonstrate that ANAVEX2-73 preserves average MMSE and ADCS-ADL scores and shows benefits over baseline for Cogstate and QEEG/ERP.

Conclusion
The safety of ANAVEX2-73 was assessed and MTD was determined. Despite not optimal dosing, both cognitive and functional performance is sustained over at least 12 months, suggesting that the effect of the compound does not seem to worsen AD symptoms with repeated dosing. In a progressive degenerative disease this is considered a positive outcome. The data support further clinical development of ANAVEX2-73 and preparation for a larger confirmatory study is underway.


Co-authors
C. Missling 1
1, USA