Anavex Life Sciences Announces FDA Approval of IND for Phase 2 Trial of ANAVEX®2-73 in Patients with Rett Syndrome
October 19 2018 - 7:00AM
Study will incorporate genomic precision
medicine ANAVEX®2-73-specific biomarkers
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, Rett
syndrome and other central nervous system (CNS) diseases, today
announced that the Company has received confirmation from the U.S.
Food and Drug Administration (FDA) that its Investigational New
Drug application (IND) is now open for ANAVEX®2-73 for the
treatment of Rett syndrome, a rare and catastrophic
neurodevelopmental disease.
ANAVEX®2-73 has already received orphan drug
designation from the FDA for the treatment of Rett syndrome.
“The acceptance of this IND by the FDA is a
significant milestone for ANAVEX®2-73,” stated Christopher U
Missling, PhD, President and Chief Executive Officer of Anavex.
“This is an important step toward achieving clinical data for the
third indication for ANAVEX®2-73 also incorporating genomic
precision medicine biomarkers.”
FDA has allowed Anavex to proceed with the Phase
2 study protocol, ANAVEX2-73-RS-001, A Double-Blind, Randomized,
Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients
with Rett Syndrome using experimental drug ANAVEX®2-73 for the
treatment of patients with Rett syndrome. The Phase 2 study is a
randomized double-blind, placebo-controlled safety, tolerability,
pharmacokinetic and efficacy study of oral liquid ANAVEX®2-73
formulation to treat Rett syndrome. Pharmacokinetic and dose
finding will be investigated in a total of 15 patients over a
7-week treatment period including ANAVEX®2-73-specific genomic
precision medicine biomarkers. All patients who participate in the
study will be eligible to receive ANAVEX®2-73 under a voluntary
open label extension protocol. This study will be followed by a
planned placebo-controlled safety and efficacy evaluation of
ANAVEX®2-73 over a 3 month treatment period.In addition to Rett
syndrome, Anavex has clinical development programs for ANAVEX®2-73
for the treatment of Alzheimer’s disease and Parkinson’s disease
dementia.
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 one in every 10,000 to
15,000 live female births.
About ANAVEX®2-73
ANAVEX®2-73 activates the Sigma-1 receptor (S1R)
protein, which serves as a molecular chaperone and functional
modulator involved in restoring homeostasis. In a Phase 2a
Alzheimer’s disease (AD) study, ANAVEX®2-73 has shown dose
dependent improvement in exploratory endpoints of cognition (MMSE)
and activities of daily living (ADCS-ADL). Full genomic analysis of
ANAVEX®2-73 Phase 2a AD patients was performed. The ANAVEX®2-73
Phase 2 Rett syndrome study design includes genomic biomarkers
identified in the ANAVEX®2-73 Phase 2a AD study. Studies of
ANAVEX®2-73 in a mouse model with a MECP2-null mutation that causes
neurological symptoms that mimic Rett syndrome, ANAXEX®2-73 was
evaluated in automatic visual response and respiration tests in
7-month old mice, an age at which advanced pathology is evident.
Vehicle-treated methyl-CpG binding protein 2 (MECP2) mice
demonstrated fewer automatic visual responses than wild-type mice.
Treatment with ANAVEX®2-73 for four weeks significantly increased
the automatic visual response in the MECP2 Rett syndrome disease
mouse (p<0.05). Additionally, chronic oral dosing daily for 6.5
weeks of ANAVEX®2-73 starting at ~5.5 weeks of age was conducted in
the MECP2 HET Rett syndrome disease mouse model assessed the
different aspects of muscular coordination, balance, motor learning
and muscular strengths, some of the core deficits observed in Rett
syndrome. Administration of ANAVEX®2-73 resulted in both
significant and dose related improvements in an array of these
behavioral paradigms in the MECP2 HET Rett syndrome disease model.
These experiments were sponsored by Rettsyndrome.org.
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, Rett syndrome and other central nervous system
(CNS) diseases, pain and various types of cancer. Anavex’s lead
drug candidate, ANAVEX®2-73, recently completed a successful 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. Preclinical studies
demonstrated its potential to halt and/or reverse the course of
Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant,
anti-amnesic, neuroprotective and anti-depressant properties in
animal models, indicating its potential to treat additional CNS
disorders, including epilepsy. The Michael J. Fox Foundation for
Parkinson’s Research previously awarded Anavex a research grant,
which fully funded a preclinical study to develop ANAVEX®2-73 for
the treatment of Parkinson’s disease. ANAVEX®3-71, which targets
sigma-1 and M1 muscarinic receptors, is a promising preclinical
drug candidate demonstrating disease-modifying activity against the
major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD)
mice, including cognitive deficits, amyloid and tau pathologies. In
preclinical trials, ANAVEX®3-71 has shown beneficial effects on
neuroinflammation and mitochondrial dysfunction. Further
information is available at www.anavex.com. You can also 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 DevelopmentToll-free: 1-844-689-3939Email:
info@anavex.com
Investors & Media:Scott
GordonCore IRscottg@coreir.com
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