2023 Industry Updates — Webinar

Allos Pharma

Randy Carpenter, MD

Allos obtained the license for arbaclofen (STX209), a drug that reached clinical trial status for Fragile X syndrome with Seaside Pharmaceuticals, and is pursing a new clinical trial in Fragile X syndrome in Q1 2024.

Randy shares the status of arbaclofen, the goal of the future clinical trial, and high-level details with us during this year’s Industry Update.

Tetra Therapeutics

Chad Coberly

Tetra is developing BPN14770/zatomilast for treating cognition and daily function in Fragile X syndrome.

Tetra completed their initial trial last year and is currently enrolling males ages 12-45-years-old with Fragile X syndrome in their trials.

Chad shared they plan to let children as young as 9-years-old in soon and are committed to trials in even younger children and females once they have the data from their current trials. Tetra also announced their at-home trial participation option that will be available soon. You can learn more about the trials here: Clinical Trials: Phase 2B/3 Studies of BPN14770 in Fragile X syndrome | NFXF

Tetra will continue to move forward with BPN14770 in FXS, likely sharing an update at the NFXF’s 19th International Conference in July 2024. Stay tuned for more details.

Zynerba Pharmaceuticals

Stephen O’Quinn PhD

Zynerba previously completed a trial of ZYN002, a clear cannabidiol gel applied to the skin, in children and adolescents with FXS.

Zynerba completed their first trial and is now recruiting for their confirmatory study of ZYN002 in FXS.

Males and females with full mutation Fragile X syndrome ages 3-22-years old may be eligible to participate in the trial. Stephen shared their exciting at-home/virtual participation option, which is up and running for families who are not able to participate by going to a study site. You can learn more about the RECONNECT trial here: Clinical Trial RECONNECT: ZYN002 Gel | MyFXResearch Portal (fragilex.org)

Zynerba plans to share an update at the NFXF’s 19th International Conference in July 2024. Stay tuned for more details.

Autifony Therapeutics

Charles Large, PhD

Autifony is a clinical stage biotechnology company dedicated to developing new medicines to treat rare genetic CNS disorders by deploying its pioneering ion channel drug discovery platform.

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. Please take a moment to review currently available opportunities at our MyFXResearch page.

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 the things we do best: 

• Growing the pool of dedicated research and clinicians (Summer Scholars, 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!