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Fragile X syndrome

Cannabinoid CB1 receptor (CNR1); CNR2

Mouse studies suggest blocking the endocannabinoid system could help treat fragile X syndrome. In mouse models for fragile X syndrome, the small molecule CNR1 antagonist rimonabant decreased cognitive impairment and susceptibility to noise-induced seizures compared with vehicle. In the mouse model, a research-grade small molecule CNR2 antagonist decreased anxiolytic behaviors compared with vehicle. Next steps include seeking a partner to carry out clinical studies and characterizing the effects of other molecules that antagonize the endocannabinoid system in animals.
Sanofi discontinued marketing Acomplia/Zimulti rimonabant to treat obesity in 2007 due to psychiatric side effects.
At least five companies have CNR1 and/or CNR2 antagonists in Phase I testing or earlier to treat endocrine/metabolic, hepatic or neurological indications.

SciBX 6(14); doi:10.1038/scibx.2013.341
Published online April 11, 2013

Patent application filed; available for licensing

Busquets-Garcia, A. et al. Nat. Med.; published online March 31, 2013;
Contact: Andrés Ozaita, Pompeu Fabra University, Barcelona, Spain