NEW YORK, May 20, 2016 (GLOBE NEWSWIRE) -- 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 diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of
cancer, today reports the U.S. Food and Drug Administration’s (FDA) Office of Orphan Product Development (OOPD) has granted Orphan
Drug Designation (ODD) to ANAVEX 2-73 for the treatment of Rett syndrome.
“The Orphan Drug Designation marks the first U.S. movement for ANAVEX 2-73. Rett syndrome is a devastating
disease occurring in early childhood and almost exclusively in girls. As there is currently no cure and limited treatment,
Rett syndrome represents a clear unmet clinical need,” commented Christopher U. Missling, PhD, President and Chief Executive
Officer. “The acknowledgement from the Agency for this orphan designation allows us to formally move forward with the planned
human clinical study in this disease indication, thereby potentially expanding the reach for ANAVEX 2-73 in addition to Alzheimer’s
disease.”
The FDA’s Office of Orphan Products Development advances the evaluation of drugs that show promise for the safe
treatment of rare diseases, defined as those 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.
About Rett Syndrome
Rett syndrome is rare non-inherited genetic postnatal progressive neurodevelopmental disorder that occurs almost exclusively in
girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat, and
even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. It is characterized by
normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands,
distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability. There is
currently no cure for Rett syndrome and treatment of the disorder is symptomatic. Management of the symptoms is done through a
multidisciplinary approach utilizing medication for motor difficulties, breathing irregularities, and control of seizures through
anticonvulsant drugs. Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups and occurs
worldwide in approximately 1 in every 10,000-15,000 live female births.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq:AVXL) is a publicly traded biopharmaceutical company dedicated to the
development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental 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 at www.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.
For Further Information:
Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939
Email: info@anavex.com
Investors:
866-505-2895
Email: ir@anavex.com
Media:
Jules Abraham
JQA Partners, Inc.
917-885-7378
jabraham@jqapartners.com
