Disease Modifying Effects of ANAVEX®2-73 (blarcamesine) for Rett Syndrome Seen

 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 long-term clinical study results from the U.S. ANAVEX®2-73-RS-001 (NCT03758924) study demonstrate disease modifying effect of ANAVEX®2-73 (blarcamesine) for adult patients with Rett syndrome.

Usually, symptomatic therapies treat the symptoms of the disease but do not address the underlying cause of the disease. Disease-modifying therapies target the underlying cause of the disease.

ANAVEX®2-73 (blarcamesine) is an orally available, small-molecule activator of the sigma-1 receptor (SIGMAR1) which, data suggest, is pivotal to restoring cellular homeostasis and promoting neuroplasticity.1

In the U.S. ANAVEX®2-73-RS-001 trial, of all 25 patients who both started and completed the randomized, double-blind, placebo-controlled study, 24 patients voluntarily enrolled in the 12-week open label extension (OLE) study, receiving once daily oral liquid ANAVEX®2-73 (blarcamesine) formulation for further evaluation of long-term safety, tolerability, and effectiveness of ANAVEX®2-73 (blarcamesine) in patients with Rett syndrome. The 12-week extension study was subsequently further extended to 36 weeks.

The effect of ANAVEX®2-73 (blarcamesine) in the double-blind part of the U.S. ANAVEX®2-73-RS-001 study was maintained in the open label 12-week extension study. Results from pharmacometric modeling of the full clinical data (i.e., from baseline of the double-blind study to the end of the open label extension study) indicates that the data are best characterized by a combined symptomatic and disease modifying drug effect model. Meaning that ANAVEX®2-73 (blarcamesine) exhibited both symptomatic and disease modifying effects in the treatment of Rett syndrome in a clinical setting.

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