Roche begins Ph II Fragile X studies

Swiss drugmaker Roche has begun a Phase II study of a candidate to treat Fragile X syndrome, a condition that is the most common cause of autism yet identified, the FRAXA Research Foundation announced this week.

Fragile X is a family of genetic conditions caused by changes in the FMR1 gene, according to the National Fragile X Foundation's website. Roche's placebo-controlled study is enrolling 60 patients. The targeted completion date is in June, according to clinicaltrials.gov.

Currently, there are four pharmaceutical companies in active clinical trials of mGluR5 antagonists for a Fragile X indication--Neuropharm, Novartis, Seaside Therapeutics and Roche. The drugs being tested are mGluR5 antagonists that work by blocking an overactive receptor that plays a key role in weakened synapses, according to an AP report.

Scientists are closely watching the drugmakers' progress because "this looks like a really promising pathway" for some types of autism, too, Dr. Andrea Beckel-Mitchener of the National Institute of Mental Health tells the AP.

 According to FRAXA, The drugmakers' clinical trial progress is as follows:

• Novartis completed its first Phase II trial of an mGluR5 antagonist for Fragile X in Europe last summer and they are proceeding ahead with more trials.


• Seaside Therapeutics recently completed Phase I of its lead mGluR5 antagonist, licensed from Merck, and is now preparing for Phase II in the U.S. this spring. Seaside has also just finished a Phase II trial of arbaclofen in patients with Fragile X and autism and is planning for Phase III.


• Neuropharm, which completed the first trial of an mGluR5 antagonist, fenobam, in patients with Fragile X, is planning follow up trials.

- check out FRAXA's announcement
- read more about the condition the foundation's website
- here's AP's coverage

Related Article:
Autism drugs head into human clinical trials