Study successfully achieved both primary and secondary objectives
ANAVEX®2-73 treatment resulted in improvements of all efficacy endpoints over 48 Weeks
Anavex plans to proceed to ANAVEX®2-73 pivotal trial for Parkinson’s disease
NEW YORK, March 30, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other Central Nervous System (CNS) disorders, reports preliminary 48-week open-label extension Parkinson’s disease dementia ANAVEX®2-73-PDD-EP-001 Phase 2 study data which demonstrated longitudinal beneficial effects of ANAVEX®2-73 on the prespecified primary and secondary objectives, as well as planned primary and key secondary endpoints which will be utilized in a forthcoming pivotal study of ANAVEX®2-73 in Parkinson’s disease.
ANAVEX®2-73 (blarcamesine) is an oral small-molecule activator of the sigma-1 receptor (SIGMAR1), which data suggests is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.1
Parkinson’s disease (PD) is a chronic, debilitating CNS disease and the second largest age-related disorder after Alzheimer’s disease.2 This study demonstrates for the first-time that patients’ clinical symptoms consistently improve longitudinally during the 48-week ANAVEX2-73-PDD-EP-001 Phase 2 study under active ANAVEX®2-73 treatment in Parkinson’s disease.
The 48-week ANAVEX2-73-PDD-EP-001 (NCT04575259) Phase 2 study assessed safety, tolerability and efficacy, measuring among others, Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)3 Parts I, II, III, REM Sleep Behavior Disorder Screening Questionnaire (RBDSQ), Clinical Global Impression – Improvement (CGI-I), as well as cognitive efficacy endpoint Montreal Cognitive Assessment (MoCA) over a 48-week period.
Preliminary analysis reveals that ANAVEX®2-73 (blarcamesine) was found to be generally safe and well tolerated; and safety findings in this study are consistent with the known safety profile of ANAVEX®2-73. In respect to efficacy, across all efficacy endpoints, patients performed better while on ANAVEX®2-73.
The 48-week Open Label Extension (OLE) ANAVEX2-73-PDD-EP-001 Phase 2 study was offered to participants after completion of the double-blind placebo-controlled ANAVEX2-73-PDD-001 Phase 2 study. Study participants were allowed to stay on a stable regimen of anti-Parkinson's disease medications (including levodopa, dopamine agonists, MAO-B inhibitors, or entacapone).
Previously, in the double-blind ANAVEX2-73-PDD-001 Phase 2 study, ANAVEX®2-73 treatment demonstrated statistically significant improvements compared to placebo (ITT population) for MDS-UPDRS Total score. From baseline to the end of the trial at 14 weeks, the MDS-UPDRS Total score improved by -10.98 points in the ANAVEX®2-73 high dose group and worsened by 3.53 points in the placebo group, an adjusted mean difference of -14.51 points (p = 0.034). This corresponds to a relative improvement of 18.9% over 14 weeks.4 This data was also consistent with expression levels of pathological dysregulated neurodegenerative genes, including Parkinson’s disease genes, which were significantly restored by the therapeutic effect of ANAVEX®2-73 (p