FRAXA Research Foundation

Shionogi has announced that enrollment for the adult trial (EXPERIENCE-301) of zatolmilast is now closed, and the adolescent trial (EXPERIENCE-204) is in its final phase. This level of participation is a testament to the dedication of families, researchers, and advocates working toward real treatment options for Fragile X syndrome. ---------- Update from Shionogi: "We have an update on the progress of Shionogi’s EXPERIENCE clinical trials (also known as BPN14770-CNS-204, BPN14770-CNS-301, BPN14770-CNS-302 and the Tetra studies): As a result of an unprecedented surge of interest by this incredible community, enrollment for the adult study in Fragile X syndrome (EXPERIENCE-301) is nearly at capacity and screening will close today. Clinical trial sites are working with Shionogi to accommodate previously scheduled appointments for potential new study participants. Notably, clinical trial sites are no longer able to accommodate new appointments as the clinical trial cannot recruit far beyond the planned 150 individuals without putting the protocol integrity of the trial at risk and exhausting the total clinical trial drug supply. The adolescent study in Fragile X syndrome (EXPERIENCE-204) is in its final phase of enrollment. All scheduled screening appointments will proceed as planned; however, no additional screening appointments are available. On behalf of our team and our partners at Shionogi, we are grateful for the support of the community in both raising awareness of and participating in these studies. Please note the open-label study (EXPERIENCE-302) is ongoing for individuals who have completed EXPERIENCE-301 or 204. We look forward to sharing the results from these studies." ---------- At FRAXA, we’ve been committed to advancing PDE4 inhibitors for Fragile X research for years, from early studies in animal models to co-funding the initial clinical trials of zatolmilast (BPN14770). Seeing this work progress into Phase 3 trials is an incredible milestone for our community. While enrollment is closing, the research continues. We’re looking forward to seeing the results of these trials. #FragileX #FXResearch #ClinicalTrials #RareDisease #Neuroscience #DrugDevelopment #Biotech #PrecisionMedicine #MedicalResearch #PDE4

?? NIH funding delays are putting Fragile X research at risk. ?? Fragile X syndrome research is at a critical turning point, but without funding, progress may grind to a halt. The Centers for Collaborative Research in Fragile X, which drive key discoveries, face uncertainty. Researchers are left in limbo, unable to continue promising studies that could lead to new treatments and a cure. FRAXA Research Foundation and the National Fragile X Foundation are working together to urge Congress and the NIH to act now. Funding delays don’t just slow science—they set research back for years. The Fragile X community deserves better. ?? Your voice can make an impact. Support the fight for continued Fragile X research funding. #FragileX #FXResearch #NIHfunding #RareDisease #Neuroscience #CureFX #BiomedicalResearch #Genetics #SciencePolicy #Neurodevelopment

Calling all families navigating the Fragile X journey! Join us on April 22 at 12:00 pm ET for Fragile X Family Conversations, a dedicated space for families and caregivers to connect, share experiences, and support one another. These virtual meetups have been incredibly valuable in bringing families together, and we’ve truly enjoyed each one. They provide a unique opportunity to exchange ideas with others who understand the challenges of Fragile X. As Fragile X parents ourselves, the FRAXA team fosters a welcoming space where families can feel heard, supported, and part of a community. Space is limited – register now to secure your spot. #FragileX #FXResearch #GeneticResearch #RareDisease #CommunitySupport #FamilySupport #NeurologicalDisorders #InclusiveCommunity #HopeInResearch #FamilyConnections

Today, on Rare Disease Day, we celebrate the millions of individuals and families affected by Fragile X syndrome and other rare disorders. Progress is happening every day! Pivotal clinical trials of new treatments are moving closer to completion. Behind the scenes, FRAXA is reviewing a exciting batch of new Fragile X research proposals, with new awards to be announced in early April. And other companies are working to develop even better treatments. Together, we keep moving forward. ?? #FragileX #FXResearch #RareDiseaseDay #RareDiseases #Neuroscience #GeneTherapy #MedicalResearch #DrugDevelopment #ClinicalTrials #RareDiseaseCommunity

FRAXA-funded researchers at Stanford University have been investigating how ISRIB, an experimental drug, might help restore brain function and social behavior in Fragile X syndrome. In Fragile X, neurons struggle to regulate protein production, leading to disrupted learning, memory, and communication between brain cells. This study found that ISRIB: >> Reduced the number of immature dendritic spines, improving brain connectivity >> Strengthened synaptic function, enhancing neural communication >> Improved social behavior in Fragile X mouse models While ISRIB has not yet been tested in humans with Fragile X, early trials in other neurological conditions, including traumatic brain injury and Alzheimer’s disease, are already underway. Could ISRIB be part of future treatment strategies for Fragile X? Researchers continue to explore the possibilities. #FragileX #FXResearch #Neuroscience #MedicalResearch #BrainHealth #RareDisease #ScientificBreakthrough #DrugDiscovery #SynapticFunction #Neurodevelopment

There’s still time to sign up for our virtual Fragile X Family Conversations happening Tuesday, February 11, at 12:00 pm ET! This special online gathering is a chance for families and caregivers to share experiences, offer support, and connect with others who truly understand the Fragile X journey. As parents ourselves, the FRAXA team knows how valuable it is to be part of a welcoming and supportive community. Space is limited – register now to save your spot! #FragileX #FXResearch #FragileXCommunity #CaregiverSupport #RareDisease #ParentingSpecialNeeds #Neurodevelopment #FamilySupport #DisabilityAwareness #CommunityMatters

Changes are happening with the Zatolmilast clinical trials for Fragile X syndrome. The studies previously known as the Tetra trials are now called the EXPERIENCE clinical studies — a name that reflects the goal of further evaluating the safety and efficacy of an investigational study drug on cognition and other aspects of Fragile X syndrome. Now led by Shionogi, these Phase 2b/3 clinical trials are still enrolling males aged 9-45 across the U.S. who meet the eligibility criteria. Participants who complete the placebo-controlled study may have the opportunity to continue treatment in an open-label extension. Learn more about the study and find clinical trial sites through FRAXA: ?? https://lnkd.in/epfe68_6

The RECONNECT clinical trial, led by Harmony Biosciences, is enrolling individuals with Fragile X syndrome between the ages of 3-29. This study is evaluating an investigational treatment for behavioral symptoms associated with FXS. With both at-home and in-clinic options, participation is more accessible than ever. Find out more: https://lnkd.in/eu4ZyrFd #FragileX #FXResearch #HarmonyBiosciences #BehavioralHealth #ClinicalStudy

Calling all families navigating the Fragile X journey! Join us on Feburary 11 at 12:00 pm ET for Fragile X Family Conversations: Sharing, Learning, Supporting. This exclusive event is a space just for families and caregivers to connect, share experiences, and offer each other support. These sessions provide unique opportunities to brainstorm with others who truly understand the daily challenges of Fragile X. With each of us on FRAXA's team is a Fragile X parent and we’re here to offer a welcoming community where families can feel seen, heard, and hopeful. Space is limited – register now to reserve your spot! #FragileX #FXResearch #GeneticResearch #RareDisease #CommunitySupport #FamilySupport #NeurologicalDisorders #InclusiveCommunity #HopeInResearch #FamilyConnections

In 2025, FRAXA is taking a bold step toward a cure for Fragile X syndrome with a new grant program focused on curative therapies. This initiative supports groundbreaking approaches such as gene therapy, gene repair, protein replacement, mRNA therapeutics, and targeted ASO treatments. Researchers are invited to apply for funding of up to $100,000 per year. Let’s drive meaningful advances in Fragile X therapeutics and deliver life-changing solutions to Fragile X families. #FragileX #FXResearch #GeneTherapy #mRNATherapeutics #Neuroscience #RareDiseaseResearch #CurativeTherapies #BiomedicalResearch #TherapeuticDevelopment #InnovativeScience