Conference Presentation

2024 Industry Updates

00 h 35 m

On July 27, 2024, at the 19th NFXF International Fragile X Conference, three of our industry partners working on treatments for Fragile X syndrome presented updates on their research: Tetra Therapeutics, Harmony Biosciences, and Spinogenix. A panel discussion and moderated Q&A followed presentations.

About the Webinar

With Gregg Harper, Cate McCanless, Kristen Bzdek, Craig Erickson, Chad Coberly, and Hilary Rosselot
Learn more about the presenters

Following each industry update, Hilary Rosselot, executive director at the NFXF, moderated a brief Q&A and panel discussion.

Tetra Therapeutics, a Shionogi Group Company

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 in their clinical trials.

Chad Coberly 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.

Update on the Development of Zatolmilast (BPN14770) for Fragile X Syndrome

Harmony Biosciences

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

Dr. Kristen Bzdek, Senior Medical Director at Harmony Biosciences, 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 to 29 may be eligible to participate in this study.

Also presenting: Gregg Harper, former Congressman, patient advocate, and dad to a son with FXS; and Cate McCanless, senior VP, Chief Corporate Affairs Officer, Harmony Biosciences.

Editorʼs Note: The team at Harmony Biosciences is excited to share that they have reached their enrollment goal for the Phase 3 RECONNECT trial and screening has officially closed.

Patients at Heart: How Harmony Biosciences Connext with Caregivers and Communities to Develop Rare Disease Treatments and an Update on the Open Label Extension Study and RECONNECT

Spinogenix

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

Dr. Craig Erickson, Chief Medical Advisor, Spinogenix, shared details on SPG601, a BK channel mechanism, and explained 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 to 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).

Editorʼs Note: The Phase 2 study is now completed. For more, see Spinogenix Reports Positive Phase 2 Results for SPG601 in Fragile X Syndrome.

SPG601 for Fragile X Syndrome

Industry Update Panel Discussion

Hilary Rosselot, executive director at the NFXF, moderated a brief Q&A and panel with industry partners discussing specific timelines for completing their studies and bringing their findings to the FDA for approval.

  • Tetra’s BPN14770 targets cognition/cognitive ability.
  • Harmony’s ZYN002 targets the behavioral symptoms of FXS. (Editor’s Note: The team at Harmony Biosciences is excited to share that they have reached their enrollment goal for the Phase 3 RECONNECT trial and screening has officially closed.)
  • 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. (Editor’s Note: The Phase 2 study is now completed.)

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:

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!

About the Presenters

Craig A. Erickson

Craig A. Erickson, MD, is a professor of Psychiatry at Cincinnati Children’s Hospital Medical Center and the University of Cincinnati College of Medicine-Affiliated. Dr. Erickson leads a neurodevelopmental clinical and research group focused on improving clinical care through research discovery. He is the director of the Cincinnati Fragile X Research and Treatment Center, one of the largest such programs in the world. He serves as the chair of the Clinical Trials Committee organized by the National Fragile X Foundation and is a leader in translational medicine efforts in Fragile X syndrome, autism, and related disorders. Additionally, he is the director of research in the Division of Psychiatry at Cincinnati Children’s Hospital. 

Gregg Harper

Gregg is a Fragile X dad to Livingston and is an NFXF team member, assisting with our advocacy efforts. He has long advocated for Fragile X, notably during his five terms representing Mississippi’s 3rd Congressional District. During his time in Congress, he served as a member of the influential House Committee on Energy and Commerce which has jurisdiction over a broad swath of the economy including healthcare, energy, transportation, and telecommunications. During his time in Congress, most of his colleagues had an opportunity to meet Livingston and be introduced to Fragile X. In 2010, Gregg established the Congressional Internship Program for Individuals with Intellectual Disabilities. It is a unique program designed to give students with varying intellectual disabilities an opportunity to gain Congressional work experience. Upon his retirement, the name was changed to the Gregg and Livingston Harper Congressional Internship Program for Individuals with Intellectual Disabilities.

Hilary Rosselot

Hilary joined the NFXF team in 2019. Prior to joining the NFXF team, she worked at the Cincinnati Fragile X Research and Treatment Center for several years. She has experience as a clinical research coordinator across many types of clinical trials and served as the clinical research manager for the Cincinnati program. She earned a bachelor’s in psychology, an MBA, and was previously a SOCRA-certified clinical research professional (CCRP). She enjoys spending time with her family, including her young daughter and Boston Terrier, as well as curling up with a good book.

Other Presenters

Chad Coberly has more than 30 years of experience in biotech/life science management including executive level roles at multiple emerging and late stage companies.

Cate McCanless is the Chief Corporate Affairs Officer for Harmony Biosciences, overseeing communications, patient advocacy, government relations and public policy, industry and alliance management and corporate philanthropy.

Kristen Bzdek is an MS/neuroimmunology-fellowship trained neurologist with over 13 years of clinical and research experience in academic and private practice in multiple sclerosis and general neurology and several years of pharmaceutical industry clinical development, business development, and medical affairs experience at Harmony Biosciences, IQVIA, Sanofi, and WCT.