Anavex Life Sciences Receives Grant From Rettsyndrome.org to Commence U.S. Phase 2 Trial in 2017
January 19 2017 - 7:00AM
ANAVEX 2-73 has U.S. FDA Orphan Drug
Designation for Rett Syndrome
Anavex Life Sciences Corp. (Nasdaq:AVXL) and the International Rett
Syndrome Foundation, doing business as Rettsyndrome.org today
announced that Rettsyndrome.org has committed a financial grant of
a minimum of $0.6 million to cover the majority of a planned U.S.
multicenter Phase 2 clinical trial of ANAVEX 2-73 for the treatment
of Rett syndrome.
Scheduled to begin in 2017, the trial will be a
randomized, double blind, placebo-controlled study of ANAVEX 2-73
in patients with Rett syndrome lasting up to 12 weeks. Primary and
secondary endpoints include safety as well as Rett syndrome
conditions such as cognitive impairment, motor impairment,
behavioral symptoms and seizure activity. Existing independent
pre-clinical research demonstrated that ANAVEX 2-73 improves all of
these measures. Hence, ANAVEX 2-73 might have a chance to
positively affect a wide range of the symptoms associated with Rett
syndrome.
Steven Kaminsky, PhD, Chief Science Officer of
Rettsyndrome.org, stated, “This is a very exciting time for us.
Pre-clinical data from ANAVEX 2-73 is highly encouraging and
demonstrates the potential of this investigational drug to
significantly improve Rett patients’ lives. Success in the Phase 2
clinical trial could result in a truly safe and effective drug for
this patient population, for which no effective treatment currently
exists. We are very motivated to support this trial financially and
by involving our knowledgeable clinical and educated community
network.”
Rettsyndrome.org sponsored Anavex’s pre-clinical
studies of Rett syndrome, which successfully demonstrated benefits
from treatment with ANAVEX 2-73. As reported at the 2016 Epilepsy
Pipeline Conference and the 2016 Rett Syndrome Research Symposium,
ANAVEX 2-73 demonstrated dose-related and significant improvements
in an array of behavioral and gait paradigms in a mouse model with
a MECP2-null mutation that causes neurological symptoms that mimic
Rett syndrome.
“We are delighted to continue our partnership
with Rettsyndrome.org and its network of experts to accelerate the
clinical development of ANAVEX 2-73 for the treatment of Rett
syndrome,” said Christopher U. Missling, PhD, President and Chief
Executive Officer of Anavex. “We look forward to initiating the
planned Phase 2 clinical trial as soon as reasonably possible in
2017.”
Anavex received Orphan Drug Designation from the
U.S. Food and Drug Administration (FDA) for ANAVEX 2-73 in Rett
syndrome on May 18, 2016. The designation provides sponsors with
development and commercial incentives, including seven years of
market exclusivity in the U.S., prioritized consultation by the FDA
on clinical studies and certain exemptions from or reductions in
regulatory fees.
About Rett Syndrome
Rett syndrome is a 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 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 to
15,000 live female births.
About Rettsyndrome.org
Rettsyndrome.org is the most comprehensive
nonprofit organization dedicated to accelerating research of
treatments and a cure for Rett syndrome and related disorders while
providing information and family empowerment. As the world's
leading private funder of Rett syndrome research, Rettsyndrome.org
has funded over $40M in high-quality, peer-reviewed research grants
and programs to date. The organization hosts the largest global
gathering of Rett researchers and clinicians to establish research
direction for the future. Rettsyndrome.org, a 501(c)3 organization,
has earned Charity Navigator's most prestigious 4 star rating year
after year. To learn more about our work and Rett syndrome, visit
www.rettsyndrome.org or call (800) 818-7388 (RETT).
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, recently completed successfully a Phase 2a clinical
trial for Alzheimer’s disease. ANAVEX 2-73 is an orally available
drug candidate that restores cellular homeostasis by targeting
sigma-1 and muscarinic receptors and successfully completed Phase
1. 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 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 and connect with the
company on Twitter, Facebook and LinkedIn.
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
info@anavex.com
Anavex Investors:
Matthew Haines
River East Investor Relations, LLC
(917) 733-9297
mhaines@rivereastir.com
Anavex Media:
Dennis Dobson, Jr.
Dobson Media Group
(203) 258-0159
dennisdobsonjr@dobsonmediagroup.com
Rettsyndrome.org:
Steven Kaminsky, PhD, Chief Science Officer
(800) 818-7388
skaminsky@rettsyndrome.org
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