Anavex Life Sciences Corp. announced positive top-line results from its Phase 1 clinical trial of ANAVEX3-71, an oral small molecule agonist of both SIGMAR1 and CHRM1 (Cholinergic Receptor Muscarinic M1) in development for the treatment of neurodegenerative diseases including Frontotemporal Dementia (FTD), for which ANAVEX3-71 has been granted Orphan Drug Designation (ODD) by the FDA. The study was a double-blind, randomized, placebo-controlled, Phase 1 trial to evaluate safety and tolerability, and pharmacokinetics (PK) of oral escalating doses of ANAVEX3-71 including effects of food and gender in healthy volunteers.

ANAVEX3-71 was well tolerated in all cohorts receiving ANAVEX3-71 in single doses ranging from 5 mg to 200 mg daily with no serious adverse events (SAEs) and no significant lab abnormalities in any subject. In the study, ANAVEX3-71 exhibited linear pharmacokinetics. Its pharmacokinetics was also dose proportional for doses up to 160 mg.

Gender had no effect on the PK of the drug and food had no effect on the bioavailability of ANAVEX3-71. The study also met the secondary objective of characterizing the effect of ANAVEX3-71 on electrocardiogram (ECG) parameters. There were no clinically significant ECG parameters throughout the study.

Participant QTcF measures were normal across all dose groups with no difference between ANAVEX3-71 and Placebo. Based on these results, and ANAVEX3-71 pre-clinical profile, the Company intends to advance ANAVEX3-71 into a biomarker-driven clinical development dementia program for the treatment of FTD, schizophrenias and Alzheimer's disease, evaluating longitudinal effect of treatment with ANAVEX3-71 initiating in 2022. Anavex believes the results of this study, could serve as the basis for advancing into respective registration studies in the U.S.