Benzinga | Jun 28, 2021 07:04AM EDT

Anavex Life Sciences Announces ANAVEX2-73 Improved both Primary Cognitive and Secondary MDS-UPDRS Efficacy Endpoints with Significant Biomarker Correlation in Placebo-Controlled Phase 2 Clinical Trial for the Treatment of Patients with Par

ANAVEX(r)2-73 treatment resulted in significant increase in the expression of the SIGMAR1 mRNA biomarker that significantly correlated with improvements in the primary and secondary clinical efficacy endpoints CoA (p = 0.029) and MDS-UPDRS Part III (p = 0.024) and MDS-UPDRS Total (p = 0.038)



14.51-point MDS-UPDRS (Movement Disorder Society- Unified Parkinson Disease Rating Scale) Total score improvement compared to placebo (p = 0.034)

Data strengthens regulatory pathway for Parkinson's disease as new therapeutic target population for ANAVEX(r)2-73

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, today reported that the predictive biomarker of response established with SIGMAR1 mRNA expression correlates significantly with responses in primary and secondary clinical efficacy endpoints from the proof-of-concept randomized, double-blind, placebo-controlled Phase 2 trial that randomized 132 patients with Parkinson's disease dementia equally (ratio of 1:1:1) to target doses of 30mg, 50mg ANAVEX(r)2-73 or placebo, respectively.

ANAVEX(r)2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.1 Recent independent findings strengthen the understanding of the beneficial effect of SIGMAR1 activation as compensatory mechanism to chronic CNS diseases.2

Parkinson's disease (PD) is a chronic CNS disease and the second largest age-related disorder after Alzheimer's disease.3 This study demonstrates for the first-time that a drug-specific biomarker correlates with clinical efficacy endpoints in Parkinson's disease.

ANAVEX(r)2-73 treatment resulted in significant (p = 0.035) mRNA expression increase of SIGMAR1, the gene encoding for the receptor targeted by ANAVEX(r)2-73, which correlated with clinical efficacy as measured by primary cognitive efficacy endpoints, CDR system Continuity of Attention (CoA) (p = 0.029) and CDR system Power of Attention (PoA) (p = 0.015), and secondary Parkinson's efficacy endpoints Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)4, MDS-UPDRS Part III (p = 0.024) and MDS-UPDRS Total (p = 0.038).