Results of First trial of Fenobam in Adults with Fragile X Published in Major Journal
We are pleased to announce the publication of positive results of a Phase IIa clinical trial of fenobam in Fragile X. Fenobam belongs to a class of compounds known as mGluR5 antagonists. Neuropharm, a specialty pharmaceutical company based in the U.K., received Orphan Drug Designation in the US in 2006 for fenobam in the treatment of Fragile X, after acquiring rights to relevant data on the compound from FRAXA.
This trial was conducted in the US by Drs. Randi Hagerman of the UC Davis MIND Institute and Elizabeth Berry-Kravis of the RUSH University Medical Center, and initial results were first announced last summer. Their article in the Journal of Medical Genetics can be accessed free at: http://jmg.bmj.com/cgi/rapidpdf/jmg.2008.063701v1
Highlights of the Study:
1. This was a single dose open label study of fenobam in 6 male and 6 female adults with Fragile X.
2. The study investigated the tolerability and pharmacokinetics of fenobam in this patient group.
3. The study also investigated the effect of fenobam on efficacy related outcome measures. These measures had the potential to index efficacy after administration of a single dose of fenobam.
4. Fenobam was well tolerated – there were no significant adverse reactions.
5. Pharmacokinetic analysis showed that fenobam levels were dose dependent but variable.
6. Four of 6 males and 2 of 6 females were responders. Potential positive effects on indices of social function such as eye contact were also noted.
7. The investigators conclude that the favorable safety profile and the clinical effects noted in this study support implementation of further controlled trials of fenobam in adults with Fragile X.
This study supports the theory, first put forth by Mark Bear, at MIT, that drugs which block or reduce mGluR5 activity could specifically reverse core deficits seen in Fragile X. It has brought hope to families who are living with Fragile X, including Jim Cantore, meteorologist for The Weather Channel. Jim’s two children have Fragile X. He is also a member of FRAXA’s Honorary Board. “Being a part of FRAXA has helped me realize that I am not alone here. Having the ability to talk with other parents about Fragile X and brainstorm on ways to make our children’s lives as happy and fulfilling as we can is priceless! FRAXA has been instrumental in speeding up the pace of Fragile X research.”
FRAXA Research Foundation and Neuropharm, have been working together to explore the potential of fenobam to treat Fragile X and possibly autism. Fenobam is a compound which reduces mGluR5 activity, which is known to be overactive in Fragile X. Neuropharm received Orphan Drug Designation in the US for fenobam to treat Fragile X Syndrome after acquiring rights to relevant data on the compound from FRAXA.
The investigators concluded that the favorable safety profile and the clinical effects noted in this study support implementation of further controlled trials of NPL-2009 in adults with Fragile X Syndrome. Robert Mansfield, Neuropharm’s CEO, commented: “This represents a significant step forward in the Fragile X arena and we acknowledge this achievement by Professors Hagerman and Berry-Kravis. We are delighted to be working with the principal investigators and with the team at FRAXA on this exciting Orphan Drug project.”
After her son Andy was diagnosed with Fragile X, Katie, her husband Michael Tranfaglia, and a third parent founded FRAXA Research Foundation. She has served as FRAXA’s executive director since 1994. Katie has a BA in History from Harvard and an MA in Computer Science from UNC Chapel Hill. She worked as a software engineer and instructor in computer science before FRAXA.