Anavex Life Sciences (AVXL) Announces FDA Fast Track Designation Granted for ANAVEX2-73 Clinical Development Program for Treatment of Rett Syndrome

February 3, 2020 7:05 AM

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) disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ANAVEX®2-73 (blarcamesine) clinical development program for the treatment of Rett syndrome.

FDA Fast Track is a program designed to facilitate and expedite the development and review of a new drug to address unmet medical need in the treatment of a serious and life-threatening condition for which it demonstrates the potential to address unmet medical needs for such a disease or condition. The purpose of the program is to get important new therapies to the patients earlier in order to address the unmet medical needs in the treatment serious and life-threatening diseases.

In the U.S. Phase 2 Rett syndrome study to date, ANAVEX®2-73 (blarcamesine) demonstrated significant improvements of the two global efficacy endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score and the Clinical Global Impression – Improvement (CGI-I).

At the 6th Annual European Rett Syndrome Conference, Anavex presented data demonstrating also that ANAVEX®2-73 (blarcamesine) significantly improved the RSBQ Hand Behaviours and the RSBQ Breathing Abnormalities scores. In addition, efficacy signals on both caregiver- and clinician-based measures of severity correlated with a key biomarker related to disease pathogenesis (Glutamate levels).

This is one of three clinical studies in Anavex’s Rett Syndrome Program: U.S. RTT (ANAVEX®2-73-RS-001), AVATAR (ANAVEX®2-73-RS-002) and EXCELLENCE (ANAVEX®2-73-RS-003).

“With no currently approved agents to treat Rett syndrome, patients and their physicians have an urgent need for new therapeutic options,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We view this FDA Fast Track designation as continued support that ANAVEX®2-73 (blarcamesine) has the potential to address this unmet need. Importantly, Fast Track designation provides a number of important advantages that could expedite the development and review of ANAVEX®2-73 (blarcamesine).”

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

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