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Also known as ANAVEX2-73 oral liquid
ANAVEX2-73 liquid · 1 trial · 1 indication
Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
Change from baseline to End of Treatment (EOT)
| Arm | Type | Description |
|---|---|---|
| ANAVEX2-73 Active | EXPERIMENTAL | ANAVEX2-73 liquid oral solution |
| ANAVEX2-73 Placebo | PLACEBO_COMPARATOR | Placebo liquid oral solution |
| Name | Type | Description |
|---|---|---|
| ANAVEX2-73 oral liquid | DRUG | Liquid oral solution |
| Placebo oral liquid | DRUG | Liquid oral solution |
Inclusion Criteria: * Aged ≥ 5 years to 17 (inclusive). * Diagnosis of classic RTT, according to 2010 criteria, and a MECP2 mutation. * Post-regression stage, defined as ≥ 6 months since last loss of spoken language or motor (fine or gross) skills. * Clinical Global Impression - Severity (CGI-S) sc...
Top 2 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Akebia Therapeutics, Inc. | AKBA | 1 | PHASE2 | Vadadustat |
| Rigel Pharmaceuticals, Inc. | RIGL | 1 | PHASE2 | Fostamatinib |
ANAVEX2-73 liquid is an investigational small molecule being studied for the treatment of Rett syndrome, a rare pediatric neurodevelopmental disorder. It is a liquid formulation developed by Anavex Life Sciences Corp. for pediatric patients. The program holds Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.
ANAVEX2-73 liquid is developed by Anavex Life Sciences Corp., which trades on the Nasdaq under the ticker AVXL. The company is the sponsor of the clinical program evaluating the liquid formulation in Rett syndrome.
ANAVEX2-73 liquid is in Phase 2 clinical development. The single Phase 2 trial of the liquid formulation in Rett syndrome has been completed, and the drug is investigational rather than approved. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.
ANAVEX2-73 liquid has been evaluated in one Phase 2 trial, NCT04304482, titled ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome. That trial is completed and enrolled 92 female participants aged 5 years and older across Australia, Canada, and the United Kingdom. It was randomized, double-blind, and placebo-controlled.
Yes. ANAVEX2-73 liquid and ANAVEX2-73 oral liquid refer to the same investigational formulation of the drug, developed by Anavex Life Sciences Corp. for oral administration in pediatric Rett syndrome patients. The liquid formulation is distinct from other formulations of ANAVEX2-73 that the company has studied.
ANAVEX2-73 liquid is not approved; it is an investigational drug still in clinical development. Its Phase 2 trial in Rett syndrome has been completed, and the FDA has granted the program Fast Track, Orphan Drug, and Rare Pediatric Disease designations, which are intended to support and expedite development.