Anavex Presents Preclinical Results of ANAVEX 2-73 in Rett Syndrome
- Data presentation at 2016 Epilepsy Pipeline Conference -
- Company exploring potential to advance ANAVEX 2-73 into human clinical studies
in rare disease indication -
New York, NY – February 25, 2016 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), 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, today announced positive data of ANAVEX 2-73 in an exploratory study in a Rett syndrome model at the 2016 Epilepsy Pipeline Conference being held February 25-26, 2016 in San Francisco, CA.
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 Rettsyndrome.org Scout Program and performed by PsychoGenics, Inc., NY.
“The data demonstrates 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. 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, PhD, Chief Science Officer of Rettsyndrome.org.
Rett syndrome is 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, MD, stated: “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” will be presented by Christopher U. Missling, PhD at the 2016 Epilepsy Pipeline Conference in San Francisco, CA at 4:25 p.m. PST February 26, 2016 in the oral session and will be available on the publications 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 Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.
About Rettsyndrome.org
Rettsyndrome.org 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. Rettsyndorme.org empowers families to make a difference. Visit www.rettsyndrome.org to learn more, or call (800) 818-7388 (RETT).
