====Anavex (AVXL) : FDA approval of IND for phase 2 trial of ANAVEX®2-73 in patients with Rett Syndrome

• Anavex has received confirmation from the FDA that its IND is now open for ANAVEX2-73 for the treatment of Rett syndrome

Study will incorporate genomic precision medicine ANAVEX®2-73-specific biomarkers

NEW YORK, Oct. 19, 2018 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that the Company has received confirmation from the U.S. Food and Drug Administration (FDA) that its Investigational New Drug application (IND) is now open for ANAVEX®2-73 for the treatment of Rett syndrome, a rare and catastrophic neurodevelopmental disease.

ANAVEX®2-73 has already received orphan drug designation from the FDA for the treatment of Rett syndrome.

“The acceptance of this IND by the FDA is a significant milestone for ANAVEX®2-73,” stated Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “This is an important step toward achieving clinical data for the third indication for ANAVEX®2-73 also incorporating genomic precision medicine biomarkers.”

FDA has allowed Anavex to proceed with the Phase 2 study protocol, ANAVEX2-73-RS-001, A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients with Rett Syndrome using experimental drug ANAVEX®2-73 for the treatment of patients with Rett syndrome. The Phase 2 study is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX®2-73 formulation to treat Rett syndrome. Pharmacokinetic and dose finding will be investigated in a total of 15 patients over a 7-week treatment period including ANAVEX®2-73-specific genomic precision medicine biomarkers. All patients who participate in the study will be eligible to receive ANAVEX®2-73 under a voluntary open label extension protocol. This study will be followed by a planned placebo-controlled safety and efficacy evaluation of ANAVEX®2-73 over a 3 month treatment period.
In addition to Rett syndrome, Anavex has clinical development programs for ANAVEX®2-73 for the treatment of Alzheimer’s disease and Parkinson’s disease dementia.

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.