GEXVal Inc., a collaborator of FRAXA Research Foundation, continues its research to develop an investigational compound for Fragile X syndrome (FXS). The company’s Phase 2 Proof of Concept (POC) trial for GXV-001, an investigational candidate, has been selected for the Japan Agency for Medical Research and Development’s (AMED) “Strengthening Program for Pharmaceutical Startup Ecosystem.”
This program will provide GEXVal with critical funding to advance the global trial of GXV-001, an investigational compound being studied for Fragile X syndrome, where there are significant unmet needs. With support from FRAXA and AMED, GEXVal continues the development of the compound.
Research Updates on GXV-001 in Fragile X Development Program
GXV-001 is another investigational compound being evaluated by the FRAXA Drug Validation Initiative (FRAXA-DVI), a program dedicated to advancing research for Fragile X syndrome. Unlike other treatments in development, GXV-001 has a distinct mechanism of action that is being studied in relation to Fragile X.
The research collaboration has completed assessments across seven behavioral parameters, including evaluation of post-treatment observations.
Given the complex nature of FXS manifestations, the research program includes comprehensive gene expression analyses in brain tissues, particularly in hippocampal regions. The collaborative research efforts continue with ongoing studies examining potential effects on neural tissue structure.
Following completion of Phase 1 trials that evaluated safety, tolerability, pharmacokinetics, and biomarker parameters, GXV-001 is being studied for Fragile X syndrome. The research program includes investigations into related neurodevelopmental and cognitive disorders that share common symptoms and underlying disease mechanisms.
How FRAXA Brings Promising Fragile X Treatments to Trials
As GEXVal prepares for its Phase 2a trials, FRAXA is actively supporting trial site selection and leveraging its research network for this next development stage. FRAXA’s commitment to accelerating innovative treatments remains central to its mission to improve the lives of families affected by Fragile X syndrome.
This collaboration highlights how FRAXA’s targeted research programs like FRAXA-DVI are driving new treatments into clinical trials and bringing hope to families worldwide.
A Brighter Future for Fragile X Families
GEXVal’s inclusion in AMED’s program and the progress of GXV-001 demonstrate what’s possible when innovative science meets collaborative support. FRAXA is proud to work alongside partners like GEXVal to move us closer to meaningful treatments for Fragile X syndrome.
