Anavex presents preclinical results of ANAVEX 2-73 in Rett Syndrome

Company exploring potential to advance ANAVEX 2-73 into human clinical studies in rare disease indication

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NEW YORK—Anavex Life Sciences Corp., a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s Disease, other central nervous system (CNS) diseases, pain and various types of cancer, recently announced positive data for ANAVEX 2-73 in an exploratory study in a Rett syndrome model at the 2016 Epilepsy Pipeline Conference held February 25-26, 2016 in San Francisco.
Chronic oral daily dosing of ANAVEX 2-73 starting at ~5.5 weeks of age was conducted in the MECP2 Rett syndrome disease mouse model and continued through a 12-week behavioral testing time point 60 minutes pre-treatment during the behavioral testing. Behavioral paradigms measure different aspects of muscular coordination, balance, motor learning and muscular strengths, some of the core deficits observed in Rett syndrome. The experiment was sponsored by the Scout Program and performed by PsychoGenics, Inc., NY.
“The data demonstrate dose-related and significant improvements in an array of behavioural and gait paradigms in a mouse model with a MECP2-null mutation that causes neurological symptoms that mimic Rett syndrome. There is a tremendous need for therapeutic solutions for the individuals living with Rett syndrome and their families, and we are very encouraged by the data we have seen with ANAVEX 2-73,” said Steven Kaminsky, Ph.D., science officer of
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 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.
Leader of the Rett syndrome Natural History Study, Alan Percy, M.D, stated in a release, “Given the strong clinical safety data profile of ANAVEX 2-73, it would be encouraging to explore the compound in patients with Rett syndrome, which is a very vulnerable patient population that in addition to its neurodevelopmental symptoms also experience a significant number of seizures.”
The presentation titled Assessment of ANAVEX 2-73 in a MECP2 Rett Syndrome Mouse Model was scheduled to be presented by Christopher U. Missling, Ph.D., at the Epilepsy Pipeline Conference in San Francisco in the oral session and will be available on the publication’s page of the Anavex website.
“The positive preclinical signal in Rett syndrome seems to support the upstream mechanism of action of ANAVEX 2-73 with the potential to target not only neurodegenerative diseases such as Alzheimer’s, but also neurodevelopmental rare diseases like Rett syndrome,” said Missling, president and CEO of Anavex. is accelerating research for treatments and a cure for Rett syndrome. As the world’s leading private funder of Rett research, they have funded more than $35 million in peer-reviewed research grants and programs to date. They are a 501(c) 3 organization, earning Charity Navigator’s most prestigious 4-star rating. empowers families to make a difference.
Anavex Life Sciences Corp. is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative diseases, including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidates, ANAVEX 2-73 and ANAVEX PLUS, the combination of ANAVEX 2-73 and donepezil (Aricept), are currently in a Phase 2a clinical trial for Alzheimer’s disease. The drug combination ANAVEX PLUS produced up to 80 percent greater reversal of memory loss in Alzheimer’s disease models versus when the drugs were used individually. 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 convulsive epileptic 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.

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