Results of first fenobam trial in adults with Fragile X Published

1/7/2009

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 everywhere 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. Not only will this exciting ongoing research and testing on new treatments get us closer to a solution for Fragile X — it will also aid in research and treatments for all Autism Spectrum disorders.” Related information: National Public Radio (NPR) Story: Potential Reversal Of Autism? Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients (an mGluR5 antagonist from Novartis) FRAXA’s research strategy: reaching for a cure More news releases