ANAVEX®2-73 (blarcamesine) Receives Compassionate Use Authorization for Pediatric Patients with Rett Syndrome
June 15 2023 - 6:30AM
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 disorders including
Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other
central nervous system (CNS) diseases, today announced that the
first patients with Rett syndrome from the ANAVEX®2-73-RS-003 study
(EXCELLENCE) are able to continue treatment with ANAVEX®2-73
(blarcamesine) thanks to Health Canada’s approval of the Special
Access Program (SAP), allowing for compassionate use following
completion of the 48-week Open-Label Extension study.
Special Access Program (SAP) applications are
granted at a physicians’ request on behalf of patients. Based on
the safety profile of ANAVEX®2-73 (blarcamesine), as well as
clinical evidence that the study drug will continue to benefit
patients, Anavex will continue its responsibility to support
patients post study by facilitating the supply of ANAVEX®2-73
(blarcamesine) to patients in Canada. Approved compassionate
programs are also ongoing in the United Kingdom and Australia,
representing all regions of the Rett Syndrome study
participation.
“Today there is no specific treatment for Rett
syndrome available in Canada and the Ontario Rett Syndrome
Association (O.R.S.A.) is pleased to see Health Canada has just
granted several investigators’ requests to access for this study
drug that may be beneficial to their patients,” commented Sabrina
Millson, President of the Ontario Rett Syndrome Association.
Health Canada's Special Access Program (SAP) for
drugs enables drugs that are not marketed in Canada to be requested
by practitioners for the treatment, diagnosis, or prevention of
serious or life-threatening conditions when conventional therapies
have failed, are unsuitable, or are unavailable either as marketed
products, or through enrollment in clinical trials.1
Christopher U Missling, PhD, President and Chief
Executive Officer of Anavex said: “While our clinical studies with
ANAVEX®2-73 (blarcamesine) are very critical to collect clinical
information for regulatory purposes, we also recognize the
long-term commitment to our investigators, patients and families.
Therefore, post clinical studies, we are keen to provide supply to
the investigators that have requested compassionate use of
ANAVEX®2-73 (blarcamesine) for their patients on a global basis. In
compliance with local authorities, many patients from our clinical
studies are now able to continue treatment with ANAVEX®2-73
(blarcamesine).”
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a
publicly traded biopharmaceutical company dedicated to the
development of novel therapeutics for the treatment of
neurodegenerative and neurodevelopmental disorders, including
Alzheimer's disease, Parkinson'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 (blarcamesine),
has successfully completed a Phase 2a and recently a Phase 2b/3
clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept
study in Parkinson's disease dementia, and both a Phase 2 and a
Phase 3 study in adult patients with Rett syndrome. 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 clinical stage 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
mitochondrial dysfunction and neuroinflammation. Further
information is available at www.anavex.com. You can also connect
with the company on Twitter, Facebook, Instagram, 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:Andrew J.
BarwickiInvestor RelationsTel: 516-662-9461Email:
andrew@barwicki.com
1
https://www.canada.ca/en/health-canada/services/drugs-health-products/special-access/drugs/guidance.html
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