NEW YORK, NY – May 22, 2017
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, other central nervous system (CNS) diseases, pain, and various types of cancer, today announced new preclinical data for ANAVEX 2-73 in the neurodevelopmental disorders Angelman syndrome, Fragile X syndrome and Rett syndrome. The data was presented at the Antiepileptic Drug Trials XIV 2017 Conference in Aventura, Florida.
Characterized as an autism spectrum disorder, Angelman syndrome is a rare neuro-genetic disorder that occurs in one in 15,000 live births. Individuals with Angelman syndrome exhibit severe cognitive and physical impairments, including ataxia, intellectual disability, speech impairment, sleep disorders, and seizures (the latter being present in over 80 percent of affected individuals).
ANAVEX 2-73 (10 mg/kg IP dosed daily for 14 days) was assessed in the Ubiquitin-protein ligase E3A (Ube3a) mouse model of Angelman syndrome for the development of audiogenic seizures in 3-4-month-old animals. Sponsored by FAST (Foundation for Angelman Syndrome Therapeutics) and conducted in the laboratory of Associate Professor Anne Anderson, MD, at Baylor College of Medicine in Houston, Texas. The results indicated that ANAVEX 2-73 administration significantly reduced audiogenic-induced seizures (p<0.01).
“We are impressed with the positive anti-seizure signal of ANAVEX 2-73,” said Paula M. Evans, Chairperson of FAST. “Most people with Angelman syndrome have recurrent seizures throughout their lives, which is a serious challenge for the individual and caretakers.”
ANAVEX 2-73 has previously been shown to normalize an array of behavioral impairments in a mouse model of Fragile X (see announcement from June 6, 2016). In a recent study sponsored by Fraxa Research Foundation (FRAXA), the effects of ANAVEX 2-73 (1 mg/kg IP dosed twice daily for 14 days) on potential biomarkers were evaluated in Fragile X mental retardation 1 knockout (Fmr1 KO) mice. Vehicle-treated Fmr1 KO mice demonstrate significantly lower brain-derived neurotrophic factor (BDNF) expression in the hippocampus compared to wild-type mice. The experiments demonstrated that ANAVEX 2-73 restored hippocampal BDNF expression to normal levels (p<0.05). BDNF under-expression has been observed in many neurodevelopmental and neurodegenerative pathologies. BDNF signaling promotes maturation of both excitatory and inhibitory synapses. ANAVEX 2-73 normalization of BDNF expression could be a contributing factor for the positive data observed in both neurodevelopmental and neurodegenerative disorders.
In addition, ANAVEX 2-73 has been further evaluated in the MECP2 Rett syndrome disease mouse model. In an experiment sponsored by Rettsyndrome.org, 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 (30 mg/kg/day PO) for four weeks significantly increased the automatic visual response in the MECP2 Rett syndrome disease mouse (p<0.05).
In Rett syndrome there are disturbances in respiration characterized by an irregular breathing pattern and frequent apnea that are common and debilitating. A trend was observed in MECP2 mice treated with ANAVEX 2-73 – that is, a reduction in apnea counts to levels comparable to those observed in wild-type animals.
“We are encouraged by these converging positive preclinical findings, confirming the potential therapeutic effect of ANAVEX 2-73 in neurodevelopmental diseases,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We plan to continue exploring the links between preclinical findings and clinical manifestations in our ongoing translational research efforts.”
The presentation is available on the Anavex website.
About Angelman Syndrome Angelman syndrome (AS) is a rare neuro-genetic disorder that affects approximately one in 15,000 people – about 500,000 individuals worldwide. Children and adults with Angelman syndrome typically have balance issues, motor impairment and debilitating seizures. Some individuals never walk. Most do not speak. Individuals with Angelman syndrome require continuous care and are unable to live independently. They have a normal life expectancy. Typical characteristics of Angelman syndrome are not usually evident at birth. People with the disorder have feeding difficulties as infants and noticeable delayed development around 6-12 months of age. They need intensive therapies to help develop functional skills. In most cases, Angelman syndrome isn’t genetically inherited. Angelman syndrome affects every race and both genders. It is often misdiagnosed as autism or cerebral palsy. For more information about Angelman syndrome, please visit www.CureAngelman.org.
About FAST (Foundation for Angelman Syndrome Therapeutics) FAST is a Section 501(c)(3) nonprofit research organization singularly focused on funding research that holds the greatest promise of treating Angelman syndrome. FAST is the largest, non-governmental funder of Angelman-specific research. Paula Evans, the mother of a young girl with Angelman syndrome, founded FAST in 2008. The foundation is based in Downers Grove, Ill.
About Fragile X Syndrome and Autism Spectrum Disorders Fragile X syndrome is the most common form of inherited intellectual disability and the most frequent single gene cause of autism, affecting approximately one in 4,000 males and one in 6,000 females. The disorder is caused by the unstable expansion of a CGG repeat in the FMR1 gene and abnormal methylation, which results in suppression of FMR1 transcription and decreased protein levels in the brain. The average age for diagnosis of Fragile X syndrome in boys and girls is 35 to 37 months and 42 months, respectively. Behavioral abnormalities, including autism spectrum disorder, are common.
Autism spectrum disorder is a behavioral diagnosis while Fragile X syndrome is a medical/genetic diagnosis. Many studies have evaluated the Fragile X syndrome-autism spectrum disorders link over the past decade. Since many children with Fragile X syndrome are interested in social interactions, they may not meet the diagnostic criteria for autism spectrum disorders, even though they exhibit some features of autism spectrum disorders such as poor eye contact, shyness, social anxiety, hand-flapping and sensory issues. Autism is much more common in boys with Fragile X syndrome than in girls with Fragile X syndrome. According to the Centers for Disease Control and Prevention (CDC), a national parent survey found that 46% of males and 16% of females with Fragile X syndrome have been diagnosed or treated for autism spectrum disorders.
About FRAXA FRAXA’s mission is to find effective treatments and a cure for children and adults with Fragile X and related disorders such as autism. FRAXA has funded over $25,000,000 in biomedical research, yielding discoveries which can change the lives of all families struggling with Fragile X. FRAXA Research Foundation is a non-profit, 501(c)(3) organization which is dedicated to funding biomedical research for improved treatment and a cure for Fragile X, the leading inherited cause of intellectual disability and autism.
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 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, other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX 2-73, recently completed successfully a 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. It has also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy and others. The Michael J. Fox Foundation 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. Further information is available at www.anavex.com. You can also connect with the company on Twitter, Facebook and LinkedIn.
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