Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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...
ANAVEX2-73 liquid is an investigational small molecule being developed for the treatment of Rett Syndrome, a rare neurological disorder. It is currently in Phase 2 clinical development and has not been approved by the FDA. The drug is being studied specifically in pediatric patients with Rett Syndrome.
ANAVEX2-73 liquid is being developed by Anavex Life Sciences Corp., a biopharmaceutical company. The company's stock is traded under the ticker symbol AVXL. Anavex Life Sciences is conducting clinical trials to evaluate the safety and efficacy of this investigational drug for Rett Syndrome.
ANAVEX2-73 liquid is currently in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The drug has been granted Fast Track designation, Orphan Drug designation, and Rare Pediatric Disease designation by the FDA for the treatment of Rett Syndrome.
ANAVEX2-73 liquid has been studied in a Phase 2 clinical trial with the identifier NCT04304482, titled "ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome." This trial was completed and enrolled 92 female participants aged 5 years and older. The study was conducted in Australia, Canada, and the United Kingdom.
ANAVEX2-73 liquid is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for Rett Syndrome. The FDA has granted it Fast Track designation, Orphan Drug designation, and Rare Pediatric Disease designation, which are intended to expedite the development and review of treatments for serious conditions.
ANAVEX2-73 liquid is a small molecule being studied for Rett Syndrome. The specific molecular target of this drug has not been disclosed in available information. Clinical trials are evaluating its safety and efficacy in pediatric patients with Rett Syndrome.