Shionogi
With Dr. Juan-Carlos Gomez, Chief Medical Officer
Dr. Juan-Carlos Gomez shared updates on the company’s Fragile X EXPERIENCE program, and what to expect next. Shionogi, a nearly 150-year-old global pharmaceutical company with expertise in central nervous system (CNS) research, is gathering real-world insights from families affected by Fragile X syndrome to guide future therapies.
Dr. Gomez explained that data from the clinical trials is currently being analyzed. He emphasized Shionogi’s commitment to collaboration with researchers, clinicians, and advocacy groups like the National Fragile X Foundation to advance innovative treatment options for FXS.
Shionogi FXS Community Update
Mirum Pharma
With Dr. Larry Carter, Senior Vice President of Clinical Development
Dr. Larry Carter provided an update on the company’s Fragile X program. Mirum, historically focused on rare liver diseases, has recently expanded into rare neurological conditions, including FXS.
Dr. Carter explained how Mirum’s investigational medication, called MRM, is designed to increase levels of cyclic AMP (cAMP) in the brain — a molecule often found at lower levels in individuals with FXS. A new Phase 2 trial in the U.S. and Spain is testing different doses of MRM in males living with full mutation FXS. The study focuses on learning, memory, and behavior. Enrollment information is available on ClinicalTrials.gov. More study-specific information will be available on our Research Opportunities portal.
Mirum Pharma: National Fragile X Foundation Research Update
Kaerus Bioscience
With Dr. Rob Ring, CEO
Dr. Rob Ring highlighted how Kaerus was created specifically for developing treatment for FXS. He highlighted the science behind their approach: targeting brain ion channels — specifically the BK channel — to regulate activity disrupted in the Fragile X brain. After a successful Phase 1 trial, Kaerus’s Fragile X program was acquired by Servier Pharmaceuticals, which will advance it into patient trials.
Kaerus Bioscience: A Promising Future for KER-0193
Servier Pharmaceuticals
With Valérie Olivier, Asset Leader
Valérie Olivier shared exciting updates on the company’s expansion into neurology, marking a new chapter beyond its established expertise in cardiometabolism and oncology. Servier recently acquired Kearus Bioscience’s Fragile X molecule, KER-0193, and is planning Phase 2 trials in adult males, with future plans of studies for children and Phase 3 trials.
Emphasizing collaboration, Servier intends to work closely with experts, patient groups, and organizations like the NFXF to ensure the program meets the needs of the Fragile X community.
Servier: 2025 Industry Update
Industry Update Q&A
With Anna De Sonia, Director of Research Facilitation, NFXF
After presentations concluded, Anna De Sonia moderated a short Q&A with the panelists. These experts highlighted the power of community-driven research, stressing the importance of listening to families, collaborating with clinical experts like the Clinical Trials Committee, having conversations with regulatory experts, and exploring combination therapies.
Some of the key points included:
- Listening to families and the community to understand what outcomes matter most.
- Engaging regulatory authorities (FDA, EMA) while presenting community insights.
- Considering multiple endpoints and combination therapies rather than relying on a single treatment.
- Emphasizing collaborative sharing of learnings across the Fragile X research space.
- Integrating medications with behavioral interventions, occupational therapy, and other supportive approaches.
- Active engagement from families, clinical trial sites, and the broader community supports successful trials.
- Increased industry interest and collaboration make this a particularly promising time for Fragile X research.
- Collaboration and support from the Fragile X community.
Presenters wrapped up by sharing what they’re most excited about for the future of Fragile X research.
2025 Industry Updates Q&A
This webinar did more than update the Fragile X community on Fragile X research programs, it reinforced the strength of the Fragile X community — collaborative, engaged, and ready to advance meaningful research together.
