Anavex Life Sciences

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a clinical-stage biopharmaceutical company focused on developing novel small molecule therapies for challenging central nervous system (CNS) diseases, including neurodegenerative aging diseases (Alzheimer’s disease (AD), Parkinson’s dementia (PDD), and frontotemporal dementia. Their therapeutic approach is centered on targeting the sigma-1 receptor (σ1/SIGMAR1) using a proprietary SIGMACEPTOR discovery platform.

The sigma-1 receptor is expressed in many different tissue types, but is especially enriched in the cerebellum, nucleus accumbens, and cerebral cortex. Variants of the sigma-1 receptor have been associated with increased risk of AD, and several sigma-1 receptor agonists have been found to ameliorate learning impairments in several animal models, including transgenic models of AD, in which they are also neuroprotective.

Localized at the endoplasmic reticulum (ER), the sigma-1 receptor regulates calcium signaling between it and the mitochondria. More recently, it has been identified as a key regulator of autophagy, particularly in autophagosome biogenesis and autophagosome-lysosome fusion. In September 2025, a new paper delineated blarcamesine’s specific biochemical mechanism in restoring autophagy via sigma-1 receptor activation.

Consistent with these mechanistic findings, Anavex’s most advanced candidate, blarcamesine, enhances the complete autophagy process in human cells and in C. elegans and ameliorates beta-amyloid-induced muscle paralysis in C. elegans. In cell models, blarcamesine counteracts beta-amyloid-induced impairments in mitochondrial function. Blarcamesine protects mice against spatial working and contextual long-term memories induced by injecting beta-amyloid 25-35 into their brain ventricles and ameliorates the increase in hippocampal lipid peroxidation.

Additionally, Anavex asserts that sigma-1 receptor agonism “protects healthy gene expression through Chromatin Remodeling” and “prevents toxic RNA from translating into proteins (post-transcription).”

The President and CEO of Anavex is Christopher U. Missling, PhD, MS, MBA; Juan Carlos Lopez-Talavera, MD, PhD, is Senior Vice President and Head of Research and Development at Anavex.

Blarcamesine (ANAVEX2-73)
Blarcamesine (ANAVEX2-73) is an orally available drug candidate under development by Anavex Life Sciences for neurodegenerative aging of the Alzheimer’s (AD) and Parkinson’s dementia (PDD) types and several other CNS disorders. It is designed to restore cellular homeostasis by targeting the sigma-1 (σ1/SIGMAR1) and muscarinic receptors. It is in late-stage development for AD, having apparently succeeded in a Phase III clinical trial and an open-label extension (OLE), and was submitted to the European Medicines Agency (EMA) for approval in December 2024.

In November 2025, Anavex issued an update on their discussions with regulators regarding blarcamesine for early AD. They stated that the EMA’s Committee for Medicinal Products for Human Use (CHMP) had come to “a negative trend vote on the Marketing Authorisation Application (MAA) for blarcamesine following its CHMP oral explanation” and that they expected the CHMP to come to a formal opinion in its December meeting. Anavex stated that “based on feedback and continued guidance from the CHMP, EMA and the Alzheimer’s disease community” and in accordance with EMA procedures, it intended to request a new, independent CHMP review by a different set of reviewers, supplying them with relevant biomarker data.

In the same update, Anavex stated that following preliminary discussions, FDA’s Center for Drug Evaluation and Research (CDER) had advised them to request a meeting to discuss their blarcamesine AD clinical trial results.

In addition to AD, PDD, and PD, blarcamesine is in development for Rett syndrome, infantile spasms, Fragile X Syndrome, Angelman syndrome, and an undisclosed rare disease.

ANAVEX 3-71 (AF710B)

Like all the drugs in Anavex’s stable, AF710B is a sigma-1 receptor agonist. It is in development for frontotemporal dementia (fr which AF710B has received orphan drug designation) and AD, and has undergone Phase I trials for both of these indications; it has also Phase II for schizophrenia.

ANAVEX 1-41

This agent is in preclinical development for depression and stroke.

ANAVEX®1066

This agent is in preclinical development for visceral, acute, and neuropathic pain.