Roche begins New Phase 2 Fragile X Clinical Trial in the US

Swiss pharmaceutical giant Hoffmann-LaRoche (“Roche”) has begun US trials of their lead mGluR5 antagonist, RO4917523, for Fragile X. Five US sites – Atlanta, Chicago, Nashville, Indianapolis, and Davis, CA – are currently recruiting participants; details here. Longtime FRAXA members will recall that we’ve been talking about the promise of mGluR5 drugs to treat Fragile X for years. Ever since FRAXA Postdoctoral Fellow Dr. Kim Huber made the original finding in Mark Bear’s lab at Brown University in 2000, FRAXA has been working nonstop to organize and fund the pivotal preclinical research which has validated these drugs as potential treatments for Fragile X. Starting in 2001, FRAXA organized a concerted effort in university labs around the world to confirm Mark Bear’s mGluR Theory of Fragile X and to demonstrate to pharmaceutical companies that mGluR5 antagonists have specific therapeutic effects for Fragile X. This strategy has exceeded expectations with trials currently in progress. Three pharmaceutical companies in now active clinical trials of mGluR5 antagonists for Fragile X. Multiple development programs are essential to FRAXA’s goal of bringing new Fragile X treatments to patients, because any one drug faces many risks in the long journey to FDA approval. The fact that many companies are also developing drugs in this class for other indications (like Parkinson’s Disease) makes it even more likely that one mGluR5 antagonist will be available in the not-too-distant future. To summarize progress to date: * Roche has started the first US multiple-dose trial of an mGluR5 antagonist at sites around the country (a Phase II trial). * Novartis completed its first Phase II trial of an mGluR5 antagonist for Fragile X in Europe last summer and are proceeding with more trials. * Seaside Therapeutics completed Phase I of its lead mGluR5 antagonist, licensed from Merck, and is preparing for Phase II in the US. Seaside has also finished a Phase II trial of arbaclofen in patients with Fragile X and autism. * Neuropharm, completed the first ever trial of an mGluR5 antagonist, fenobam, in patients with Fragile X. However, Neuropharm ran out of funds and closed in April, 2010. Roche and Novartis started work on Fragile X after a some of their key scientists were invited to FRAXA’s 2004 research meeting at Banbury. Graeme Bilbe, Ph.D., of Novartis, and Mark Bear, Ph.D., of MIT, at the FRAXA Banbury meeting at Cold Spring Harbor, NY, 2004 Fabrizio Gasparini, Ph.D., of Novartis, and Will Spooren, Ph.D., of Roche, Cold Spring Harbor, NY, 2004 Katie Clapp and Mike Tranfaglia, MD, of FRAXA, and Mark Bear, of MIT Roche trial in Fragile X, on Novartis trial in Fragile X, on Neuropharm trial of fenobam HEALTHBEAT: Drugs tested to improve learning in Fragile X syndrome, may give autism hints AP, February 1, 2010 New York Times: Promise Seen in Drug April 29, 2010 FRAXA’s research strategy: reaching for a cure National Public Radio(NPR): Potential Reversal Of Autism? FRAXA continues to work with these companies and others. More trials are coming soon, with mGluR-based treatments as well as other therapeutic strategies.

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