We join with all of you in expressing our disappointment at learning the news of the negative phase II clinical study results from Roche. The drug under study was a metabotropic glutamate receptor subtype 5 (mGluR5) negative allosteric modulator. This means that it was designed to reverse the over-activity in this pathway that is brought about by the absence of FMRP.
Roche released a statement this morning confirming the discontinuation of the study. The company has also scheduled a webcast to discuss the clinical study results for September 18, please see webcast details below.
While this news comes as a disappointment, we must persevere. We know that Fragile X syndrome has been reversed in animals time after time. Now, more than ever, it is critically important that we as a community continue to support ongoing efforts to bring drugs through clinical trials to market.
Without our participation in these trials this will never occur.
The knowledge gained from the trials to date, about both the science and the process, will prove invaluable moving forward. None of this knowledge could have been gained without the participation of families in clinical trials.
The Fragile X community at large and indeed the autism and broader intellectual and developmental disability communities owe a deep debt of gratitude to those of you who have blazed this trail thus far.
To offer some perspective, consider the following comment made today following the Roche announcement from Jonathan Rubin, MD, MBA, Chief Medical Officer of Alcobra Ltd:
“We are currently enrolling adult and adolescent Fragile X patients in a multi-center Phase II placebo-controlled study. Our drug candidate, Metadoxine Extended Release (MDX), a novel monoamine-independent GABA modulator, has demonstrated significant pro-cognitive effects in three successful Phase II studies of adults with Attention Deficit Hyperactivity Disorder and has shown positive results in the Fmr1 knock-out mouse model for Fragile X syndrome. MDX has a different mechanism of action than compounds previously studied in clinical trials of Fragile X or ADHD patients. Building upon successful pre-clinical and clinical studies, we are eager to complete enrollment in the study and evaluate the potential of MDX in patients with Fragile X Syndrome.”
While other drug companies may be leaving the market, new ones are entering and they are eager to get trials underway. While we cannot predict the results of future trials, we are encouraged by the continued and robust interest in FX.
The National Fragile X Foundation will continue to work alongside these companies to bring you the most up-to-date information available at research.fragilex.org. We are here to provide support and hope for a brighter tomorrow.
We will move forward together.
Chief Executive Officer
Director of Government Affairs and Advocacy
For more information, Roche has scheduled a webcast and all are invited to join.
Roche Webcast Information
Webcast: Thursday, September 18 from 12:30 pm – 1:30 pm EDT
To pre-register and download this event to your calendar, please go to:
To access the live webcast, please go to:
To listen to the webcast call, please dial in 10-15 minutes prior to the scheduled start, using the following numbers:
+1 (1)631 570 5613 (USA)
+41 (0)58 310 50 00 (Europe)
+44 (0)203 059 58 62 (UK)