U.S. FDA Grants Orphan Drug Designation to ANAVEX 3-71 for the Treatment of Frontotemporal Dementia

NEW YORK, April 08, 2016  -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ANAVEX 3-71 for the treatment of Frontotemporal dementia (FTD).

 

Frontotemporal dementia describes a clinical syndrome associated with progressive shrinking of the frontal and temporal anterior lobes of the brain.  It causes a decline in language and/or behavioral changes, which include inappropriate social conduct, lack of empathy, blunted emotions or agitation, neglect of personal hygiene and a decrease in energy and motivation.  To date, no treatment has been shown to slow its progression and the prognosis for people with FTD is poor.  FTD afflicts an estimated 50,000 to 60,000 patients in the United States and represents an estimated 10 to 20 percent of all dementia cases.

 

“We believe that Orphan Drug Designation for ANAVEX 3-71 for the treatment of Frontotemporal dementia is a significant achievement,” said Kristina M. Capiak, Vice President of Regulatory Affairs at Anavex.

 

FDA Orphan Drug Designation is evaluated for drugs from all classes that are intended for the treatment of rare diseases, defined as diseases affecting fewer than 200,000 people in the United States.  The designation provides sponsors with development and commercial incentives, including seven years of market exclusivity in the U.S., prioritized consultation by FDA on clinical studies and certain exemptions from or reductions in regulatory fees.

 

“This regulatory milestone allows us to continue to advance in the rare disease space while expanding our portfolio within neurodegenerative diseases,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.

 

About ANAVEX 3-71

ANAVEX 3-71 (previously AF710B) is a preclinical drug candidate targeting both sigma-1 and M1 muscarinic receptors demonstrating neuroprotection and improved cognition in Alzheimer’s disease models.  It is a CNS-penetrable mono-therapy that bridges treatment of both cognitive impairments with potential disease modifications. ANAVEX 3-71 is effective in very low doses against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies, and has also demonstrated beneficial effects on neuroinflammation and mitochondrial dysfunctions.

 

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (AVXL) is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX 2-73 is currently in a Phase 2a clinical trial for Alzheimer’s disease. ANAVEX 2-73 is an orally available drug candidate that targets sigma-1 and muscarinic receptors and successfully completed Phase 1 with a clean safety profile. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. It has also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy and others. The Michael J. Fox Foundation (MJFF) for Parkinson’s Research has awarded Anavex a research grant to develop ANAVEX 2-73 for the treatment of Parkinson’s disease to fully fund a preclinical study, which could justify moving ANAVEX 2-73 into a Parkinson’s disease clinical trial.  ANAVEX 3-71, also targeting sigma-1 and M1 muscarinic receptors, is a promising preclinical drug candidate demonstrating disease modifications against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies, and also with beneficial effects on neuroinflammation and mitochondrial dysfunctions. Further information is available atwww.anavex.com.

 

Forward-Looking Statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

 

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