Health Canada Special Access Program (SAP) Authorizes ANAVEX®2-73 (blarcamesine) for patients after successful completion of Open-Label Extension (OLE) study
Granting of requests are also ongoing in United Kingdom and Australia – representing all regions of Rett Syndrome study participation
NEW YORK – June 15, 2023
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. 
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.
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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