FDA grants orphan drug designation to ANAVEX 3-71 to treat Frontotemporal dementia

ANAVEX 3-71 is a preclinical drug candidate with a novel mechanism of action through sigma-1 receptor activation and M1 muscarinic allosteric modulation, which has demonstrated to enhance neuroprotection and cognition in Alzheimer’s disease.

It is a central nervous system (CNS)-penetrable mono-therapy that bridges treatment of both cognitive impairments with potential disease modifications, Anavex said.

ANAVEX 3-71 is found to be effective in very low doses against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, such as cognitive deficits, amyloid and tau pathologies.

Beneficial effects were also seen on neuroinflammation and mitochondrial dysfunctions.

Anavex Life Sciences president and CEO Christopher Missling said: "This regulatory milestone allows us to continue to advance in the rare disease space while expanding our portfolio within neurodegenerative diseases."

Frontotemporal dementia refers to a group of disorders caused by progressive nerve cell loss in the brain’s frontal lobes or its temporal lobes.

It afflicts a projected 50,000 to 60,000 patients in the US and represents nearly 10% to 20% of all dementia cases.

Anavex Life Sciences develops differentiated therapeutics to treat neurodegenerative diseases including Alzheimer’s disease, other CNS diseases, pain and various types of cancer.

The company’s lead drug candidate, ANAVEX 2-73 is currently in a phase 2a clinical trial for Alzheimer’s disease.