2024 Industry Updates — Presentation

Tetra Therapeutics, a Shionogi Group Company

Chad Coberly

Tetra Therapeutics, a Shionogi Group Company, is developing BPN14770/zatomilast for treating cognition and daily function in FXS.

Tetra is currently enrolling males living with FXS who are between the ages of 9 and 45 years in their clinical trials.

Chad shared updates on Tetra’s current FXS Clinical Program, which includes three clinical trials testing the study drug BPN14770 in FXS.  The extension study (available for participants who complete a previous clinical trial) has been extended to two years.  Children as young as 9 years old can now participate in trials, and at-home participation – where the medical staff come to you – is now being rolled out as an option.

Additionally, Chad shared that Tetra’s FXS Program recently received two designations from the FDA that will help to “speed up” the process when the time comes to submit to the FDA for approval of BPN14770.

You can learn more about the Tetra trials here.

Harmony Biosciences

Greg Harper, Former Congressman, Patient Advocate, Dad

Cate McCanless, Senior VP, Chief Corporate Affairs Officer

Kristen Bzdek, MD, Senior Medical Director

Harmony Biosciences, which acquired Zynerba Pharmaceuticals, is committed to continuing to test ZYN002, a clear cannabidiol gel applied to the skin, in children and adolescents – ages 3 to 29 years – living with FXS.

Dr. Kristen Bzdek shared that a data analysis focusing on safety and efficacy in the open-label extension study had significant and exciting findings.  Dr. Bzdek discussed the RECONNECT trial and its new fully at-home option for U.S. participants, where visits occur virtually and with a study nurse traveling to participants’ homes.  Males and females living with full mutation FXS ages 3-29 years old may be eligible to participate in this study.

You can learn more about the RECONNECT trial here.

Spinogenix

Craig Erickson, MD, Chief Medical Advisor

Spinogenix is testing SPG601, a novel drug mechanism, in a single-dose challenge study.

Dr. Craig Erickson shares details on SPG601, a BK channel mechanism, and explains the benefits of single-dose study models.

SPG601 is a BK channel activity modulator that Spinogenix is studying.  Currently, there is one clinical trial that is enrolling 10 adult males (ages 18-45) living with FXS to evaluate SPG601.  The study requires two study visits to the study center at Cincinnati Children’s Hospital Medical Center.  This study focuses on analyzing data from brain activity and brain function (non-invasive procedures).

Panel Discussion

Hilary Rosselot, Executive Director at the NFXF, moderated a brief Q&A and panel discussion following the industry updates.

Industry partners discussed specific timelines for completing their studies and bringing their findings to the FDA for approval.

Harmony’s ZYN002 targets the behavioral symptoms of FXS.  Tetra’s BPN14770 targets cognition/cognitive ability.  The Spinogenix trial is at the “proof of concept” level, testing the concept of quickly studying the effects of SPG601 after one single dose to see if its effects suggest it’s worth advancing into future trials.

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!