Anavex Life Sciences (AVXL) Reports Results of Review by Independent DSMB for its Phase 2/3 Trials of ANAVEX®2-73 in Patients with Rett Syndrome

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 the Independent Data Safety Monitoring Board (DSMB) for the Company's ongoing clinical trial program, including the late stage AVATAR (ANAVEX®2-73-RS-002)1, EXCELLENCE (ANAVEX®2-73-RS-003)2 and the U.S. Rett syndrome extension study (ANAVEX®2-73-RS-EP-001) of its investigational compound ANAVEX®2-73 (blarcamesine) has completed its recent pre-planned review of the respective interim safety data for these three separate clinical studies.

Upon review of the interim safety data, the DSMB made the following recommendation for the randomized, double-blind, placebo-controlled AVATAR (ANAVEX®2-73-RS-002) study in adult patients with Rett syndrome:

• The DSMB recommendation is to continue the studies without modification.

Upon review of the interim safety data, the DSMB made the following recommendation for the randomized, double-blind, placebo-controlled EXCELLENCE (ANAVEX®2-73-RS-003) study in pediatric patients with Rett syndrome:

• The DSMB recommendation is to continue the studies without modification.

Upon review of the interim safety data, the DSMB made the following recommendation for the open-label extension U.S. Rett syndrome (ANAVEX®2-73-RS-EP-001) study in adult patients with Rett syndrome:

• The DSMB recommendation is to continue the studies without modification.

DSMBs are committees commonly used in clinical trials to protect the interests of the patients and the integrity of the study data in ongoing trials.

Based on the previously reported results of the first placebo-controlled U.S. Phase 2 (ANAVEX®2-73-RS-001)3 study in adult patients with Rett syndrome, Anavex is planning to meet with the FDA to discuss the approval pathway. There are no FDA-approved drugs for Rett syndrome. ANAVEX®2-73 has Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome and may be considered for accelerated approval.

The respective ongoing placebo-controlled Phase 2 and Phase 2/3 ANAVEX®2-73 clinical studies in adult (AVATAR) and pediatric (EXCELLENCE) patients with Rett syndrome are expected to be completed by mid 2021 and 2H 2021, respectively.

ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.4

PET scan data previously confirmed dose-dependent target engagement of SIGMAR1 with ANAVEX®2-73.5

Anavex Life Sciences’ product portfolio includes small drug molecule lead candidate ANAVEX®2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease and Rett syndrome.

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