Anavex Life Sciences, a clinical-stage biopharmaceutical company, experienced a significant setback on Tuesday as its stock plummeted by approximately 38% in premarket trading. This sharp decline was a direct result of disappointing topline results from the Phase 2/3 EXCELLENCE clinical trial, revealing that the company’s leading asset, ANAVEX 2-73, intended for the treatment of the neurodevelopmental disorder Rett syndrome, failed to achieve its primary objectives.
As of the market opening on Tuesday, Anavex Life Sciences Corp’s stock, which had closed at $9.31 in Friday’s trading, saw a stark decline, commencing the day at $5.90, reflecting a 34.59% drop. The latest figures as of 01:06 PM EST indicated a continuation of this downward trend.
The EXCELLENCE trial focused on 92 children aged 5–17 diagnosed with Rett syndrome, aiming to evaluate the efficacy of ANAVEX 2-73. Dishearteningly, the trial did not meet one of its co-primary endpoints, the Clinical Global Impression-Improvement Scale (CGI-I). While the trial showed improvement in the Rett Syndrome Behaviour Questionnaire (RSBQ), this improvement lacked statistical significance.
Walter Kaufmann, Anavex’s Chief Scientific Officer, attributed the setback to a notable placebo response that obscured the therapeutic effect of ANAVEX 2-73, also known as blarcamesine. Kaufmann highlighted ongoing data analysis but offered an early assessment, indicating that the placebo rate may have been influenced by a slight imbalance in disease severity at baseline across treatment arms and the 2 to 1 drug-to-placebo randomization ratio.
Despite the setback, there were positive aspects in the trial results. The Anxiety, Depression, and Mood Scale (ADAMS), a key secondary endpoint, displayed favorable trends. Additionally, more than 91% of participants who completed the 12-week trial progressed to a 48-week open-label extension study (OLE).
Notably, the trial uncovered new safety signals that warrant further investigation. These findings add a layer of complexity to the overall assessment of ANAVEX 2-73’s viability as a therapeutic option for Rett syndrome.
Investors reacted swiftly to the news, prompting a substantial drop in the company’s stock value. The market’s response underscores the significance of these clinical trial results for Anavex Life Sciences, as the company grapples with the implications and adjusts its strategic approach moving forward. The fate of ANAVEX 2-73 as a potential treatment for Rett syndrome now hangs in the balance, awaiting further analysis and potential adjustments to future clinical trials.
