Allos Pharma
Dr. Randy Carpenter of Allos discusses how they obtained the license for arbaclofen (STX209), a drug that reached clinical trial status for Fragile X syndrome with Seaside Pharmaceuticals.
Families and professionals expressed their disappointment in the STX209 program ending. There was hope that the data would be made available for future research and possibly to even help with future trials to study arbaclofen in FXS for a drug approval. Until now, this has not been an option. Allos plans to meet with FDA about the future of arbaclofen in FXS.
We have been able to work with the organization storing the data to move forward with obtaining your data, but we need your help! If your child participated in the trial, please consider giving your permission to provide the data for future research. (Editor’s Note: Sign-up is longer available.)
Allos Pharma Industry Updates Webinar
Tetra Therapeutics
Tetra is developing BPN14770/zatomilast for treating cognition and daily function in Fragile X syndrome.
Tetra previously completed a trial of BPN14770 in adult men with FXS. You can read our summary of a paper published on the research this past summer.
According to presenter Dr. Chad Coberly, Tetra plans to move forward with BPN14770 in FXS, likely opening future trials with broader age ranges in late 2021.
Editor’s Note: The Phase 2B/3 Studies of BPN14770 in Fragile X syndrome is now enrolling.
BPN14770/Zatolmilast in Fragile X Syndrome
Zynerba Pharmaceuticals
Zynerba previously completed a trial of ZYN002, a clear cannabidiol gel applied to the skin, in children and adolescents with FXS.
According to presenter Dr. Joseph Palumbo, Zynerba is moving forward with ZYN002 in FXS and is actively recruiting 3 to 17 year olds with FXS.
Editor’s Note: The ZYN002 trial reached its enrollment goal in May 2025.
Zynerba Pharmaceuticals Industry Updates Webinar
Developing New Treatments for Fragile X is a Team Effort
We will not be able to have more effective treatments or a drug on the market to target Fragile X without your help.
Consider participating in research opportunities – which range from surveys to more involved clinical trials. Researchers work with us to connect participants to them for their research and trials because, without you, research would not happen.
We are dedicated to advancing treatment research. We believe in scientifically sound research that has the community at the heart of the work. After all, you are the ones who will be using the treatments!
We advance treatment research by doing what we do best:
- Growing the pool of dedicated research and clinicians (Randi J. Hagerman Summer Scholars Research Awards, NFXF International Fragile X Conference, FXCRC)
- Connecting the right groups of people, like our industry partners with our FXCRC clinics and Clinical Trials Committee
- Educating these groups on what you, the community, want and need from a future treatment and providing them the right tools to design and execute meaningful clinical programs that will impact you. This is part of our Research Readiness Program.
- Advocating for regulatory understanding of what life is like with Fragile X and continued and increased research funding to contribute to that understanding through initiatives such as Advocacy and the Patient-Focused Drug Development meeting with FDA.
- Providing the right tools for you to make the best decisions for your family, such as Research 101, MyFXResearch portal, IFXPR, treatment recommendations.
While there are drugs and non-drug interventions that help address the symptoms of FXS, we do not have a drug for FXS. And we will likely need more than one treatment to help every person living with Fragile X. This process, the work of our industry and academic research partners, and the FDA’s oversight ensure that when there is a drug for FXS it will be safe and effective. Together, We’re Stronger!
