Anavex Life Sciences Corp. announced on October 15, 2026, that it has received notification from the U.S. Food and Drug Administration (FDA) allowing the company to proceed with a planned clinical study for its investigational drug ANAVEX®2-73 (blarcamesine) in patients with Fragile X syndrome (FXS).
The FDA clearance enables the company to open its Investigational New Drug (IND) application for blarcamesine in the United States. The company noted that this approval establishes an open IND in all three of its core indications: Alzheimer’s disease, Rett syndrome, and Fragile X syndrome.
The planned Phase 2a study, designated ANAVEX2-73-FXS-001, is a randomized, double-blind, placebo-controlled, dose-ranging trial. The study will evaluate the safety, tolerability, and pharmacokinetics of blarcamesine administered as an oral solution. The trial is expected to enroll approximately 60 adult males with Fragile X syndrome.
Secondary endpoints for the study include behavioral assessments using the Aberrant Behavior Checklist revised for Fragile X (ABC FX) and cognitive measures using the NIH Toolbox.
According to the filing, blarcamesine previously received Orphan Drug Designation from the FDA for the treatment of Fragile X syndrome. The company stated that newly published peer-reviewed data in Frontiers in Pharmacology in September 2026 supports the scientific rationale for the clinical development program. The study reported that 14 days of oral blarcamesine produced dose-related improvements in EEG abnormalities in a mouse model of Fragile X syndrome.
The company emphasized that blarcamesine is an investigational drug that has not been approved by the FDA or any other regulatory authority for any indication, and its safety and efficacy in Fragile X syndrome have not been established.
