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Anavex Shares Surge 87.4% on Phase II Trial Results of Its Rett Syndrome Drug

Updated 15 Dec 2020
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Shares of Anavex Life Sciences Corp (NASDAQ: AVXL) surged 87.4% after reporting positive results from a U.S Phase II trial of its ANAVEX®2-73 (blarcamesine) in adult female patients with Rett syndrome.


The main goal of the trial was safety and the convenient oral liquid once-daily dosing of 5 mg ANAVEX®2-73 was well-tolerated and demonstrated dose-proportional PK (pharmacokinetics). Adverse events related to study drug were similar between ANAVEX®2-73 (13.3%) and placebo (10%), with no reported serious adverse events (SAEs).

All twenty-five patients in this randomized study elected to enter a 12-week ANAVEX®2-73 extension study. Anavex will be advancing its Expanded Access Policy in order to provide long-term therapy to current participants with Rett syndrome under an expanded access program for ANAVEX®2-73.

Walter E Kaufmann, MD, Anavex’s Chief Medical Officer., commented: “The outcome of this trial is very promising in terms of both safety and clinical improvement. Despite the challenges of the older age of the cohort (patients were over 18 years of age) and the relatively low dose (5 mg daily), ANAVEX®2-73 demonstrated clinically meaningful improvements in outcome measures evaluating multiple impairments,”


“Moreover, the convergent clinical evidence was supported by parallel changes in a key biomarker of disease. This strong body of data opens the possibility of successful treatment for both adults and children with Rett syndrome and early interventions for modifying the course of the disease.”

Based on today’s results, the company is planning to meet with the FDA given that 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.

Anavex share price

Tradingview chart of Anavex share price 15122020

Anavex shares today surged 87.4% to trade at $9.73 having rallied from Friday’s closing price of $5.19.

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