Anavex' therapy for Rett syndrome candidate shows promise, but questions remain

The potential Food and Drug Administration acceptance of US biopharma Anavex Life Sciences’ (Nasdaq: AVXL) AVATAR as a pivotal Phase III trial would have an important bearing on the Rett syndrome market, as it could enable blarcamesine to launch at a similar time as Australia-based Neuren Pharmaceuticals’ (ASX: NEU) trofenitide, which is a major future competitor, says GlobalData, a leading data and analytics company.

Anavex Life Sciences recently announced positive results from the Phase III AVATAR (NCT03941444) trial of its innovative drug blarcamesine for the treatment of adult females with Rett syndrome. Currently, there is no cure for Rett syndrome, and no FDA approved drugs specifically for the indication. Treatment is focused on improving specific symptoms of the syndrome, such as anticonvulsants to manage seizures.

According to GlobalData’s neurology analyst Philippa Salter, “There is a huge unmet need in the Rett syndrome patient population and thus significant opportunity for drug developers. If the FDA accepts AVATAR as a pivotal trial, blarcamesine will likely be able to launch in lock-step with trofinetide, with the two drugs sharing the first-to-market advantage. However, if the FDA requests an additional Phase III trial for blarcamesine, this would certainly provide a competitive edge to Neuren Pharmaceuticals.”