Your browser doesn't support javascript. This means that the content or functionality of our website will be limited or unavailable. If you need more information about Vinnova, please contact us.

Melatonin MT2-Selective Agonists as a Novel Treatment for Fragile X Syndrome (FXS) and Phelan-McDermid Syndrome (PMS)

Reference number
Coordinator CTC Clinical Trial Consultants AB
Funding from Vinnova SEK 2 169 764
Project duration January 2026 - January 2029
Status Ongoing
Venture European Joint Programme on Rare Diseases
Call International collaborations with preclinical therapy studies of rare diseases

Purpose and goal

This project aims to produce results showing that COS01, a brain-penetrating partial selective melatonin MT2 receptor agonist, can alleviate insomnia, anxiety, and social impairments in autism spectrum disorders (ASD) such as Fragile X-syndrome (FSX) and Phelan-McDermid syndrome (PMS).

Expected effects and result

1) Mapped effect of COS01 in sleep disorders in animal models 2) Mapped effect of COS01 in autism-like animal models 3) Mapped effect of how COS01 modulates neuronal pathways in animal models 4) Mapped molecular mechanisms for COS01 in vivo and in vitro 5) Completed preparations to bring COS01 to First-In-Human clinical trial including formulation of COS01.

Planned approach and implementation

This project is a three-year international collaboration between five parties, organized in five partly parallel and collaborative work packages. Regular meetings ensure coordination, follow-up of results and identification of potential problem areas. The consortium actively involves patients with ASD and their families, who will work together with the researchers as partners at different stages during the project.

The project description has been provided by the project members themselves and the text has not been looked at by our editors.

Last updated 9 July 2026

Reference number 2025-01795