- In September 2023, Anavex Life Sciences Corp. announced promising results from a long-term clinical study displaying the disease-modifying effects of ANAVEX 2-73 (blarcamesine) for Rett syndrome. The study demonstrated sustained improvements in clinical symptoms and biomarkers over 148 weeks, highlighting blarcamesine's potential as a novel drug activating sigma-1 receptors associated with neuroprotection and neuroplasticity
- In August 2023, Neuren Pharmaceuticals received FDA approval for Daybue (trofinetide), marking it as the first treatment for Rett syndrome. Daybue, a synthetic analog of a natural neurotrophic peptide, enhances synaptic function and reduces neuroinflammation. The approval was based on positive outcomes from two phase 3 clinical trials, indicating Daybue's efficacy in improving core symptoms such as breathing, communication, mood, and motor function in Rett syndrome patients
- In June 2020, Otsuka Pharmaceuticals Co., Ltd. disclosed a strategic Research and Development (R&D) collaboration with Axcelead Drug Discovery, specifically focusing on neuroscience research. This collaboration has proven fruitful, significantly enriching Otsuka's drug development pipelines with a broader range of potential candidates. The collaboration emphasizes Otsuka's commitment to advancing neuroscience solutions, leveraging Axcelead's expertise to explore innovative therapeutic avenues and enhance the company's capabilities in addressing neurological disorders
