Anavex Life Sciences Reports the Results of Review by the Independent Data Safety Monitoring Board for its Phase 2/3 Trials of ANAVEX2-73 in Patients with Rett Syndrome - Seite 2

The respective ongoing placebo-controlled Phase 2 and Phase 2/3 ANAVEX2-73 clinical studies in adult (AVATAR) and pediatric (EXCELLENCE) patients with Rett syndrome are expected to be completed by mid 2021 and 2H 2021, respectively.

ANAVEX2-73 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 ANAVEX2-73.⁵

Anavex Life Sciences’ product portfolio includes small drug molecule lead candidate ANAVEX2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease and Rett syndrome.

About ANAVEX2-73-RS-002 AVATAR Clinical Study (NCT03941444)

The Phase 2 trial is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX2-73 to treat Rett syndrome in a total of 33 adult patients with Rett syndrome over a 7-weeks treatment period incorporating precision medicine biomarkers. Preceding the placebo-controlled randomization of 30 patients (Part B), a 3-patient cohort (Part A) underwent a pharmacokinetic (PK) assessment with safety, tolerability, pharmacokinetic and efficacy evaluation of ANAVEX2-73. All patients who completed the study are eligible to continue to receive ANAVEX2-73 for additional 48 weeks within the open label extension protocol.

About ANAVEX2-73-RS-003 EXCELLENCE Clinical Study (NCT04304482)

The Phase 2/3 trial is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX2-73 to treat Rett syndrome in at least 69 pediatric patients with Rett syndrome over a 12-weeks treatment period incorporating precision medicine biomarkers. All patients who completed the study are eligible to continue to receive ANAVEX2-73 for additional 48 weeks within the open label extension protocol.

About ANAVEX2-73-RS-001 U.S. Clinical Study (NCT03758924)

The Phase 2 trial is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX2-73 to treat Rett syndrome in a total of 31 adult patients with Rett syndrome over a 7-weeks treatment period were evaluated incorporating precision medicine biomarkers. Preceding the placebo-controlled randomization of 25 patients (Part B), a 6-patient cohort (Part A) underwent a 7-weeks pharmacokinetic (PK) assessment with safety, tolerability, pharmacokinetic and efficacy evaluation of ANAVEX2-73. All patients who completed in the study were eligible to continue to receive ANAVEX2-73 under a 36-week open label extension protocol (ANAVEX2-73-RS-EP-001).