Anavex clears FDA hurdle for blarcamesine in Fragile X syndrome

Anavex Life Sciences Corp. said on October 15, 2026 that the FDA notified the company it may move forward with its planned clinical study of ANAVEX®2-73 (blarcamesine) in Fragile X syndrome (FXS) under the compound's investigational new drug application. FXS is described as a genetic condition and the most common inherited cause of intellectual disability and developmental delay, as well as the most commonly known single-gene cause of autism, with no FDA-approved treatment currently available.¹

With the IND now active, the company can begin clinical testing of blarcamesine in FXS patients in the United States, and the drug previously received an FDA Orphan Drug Designation for this indication, according to the release.

Interim Chief Executive Officer Terrie Kellmeyer said the clearance means Anavex now holds open INDs in all three of its core indications: Alzheimer's Disease, Rett syndrome, and Fragile X syndrome¹. She added that the company is positioned to advance a planned Phase 2a randomized, double-blind, placebo-controlled, dose-ranging study, titled ANAVEX2-73-FXS-001, testing blarcamesine oral solution's safety, tolerability, and pharmacokinetics in roughly 60 adult males with FXS, with the Aberrant Behavior Checklist revised for FXS and NIH Toolbox measures serving as secondary endpoints¹.

The release also cited a new peer-reviewed paper published in Frontiers in Pharmacology in September 2026, which extended earlier 2024 data and reported that 14 days of oral blarcamesine treatment in a mouse model of FXS produced dose-related improvements in EEG measures, including elevated resting gamma power and impaired synchronization to auditory stimuli, abnormalities shared between the mouse model and people with FXS¹.

The company noted that blarcamesine remains investigational, with no regulatory approval anywhere and no established safety or efficacy data in FXS.