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Fragile X disappointments upset autism ambitions

Nature Reviews Drug Discovery volume 14, pages 151–153 (2015)Cite this article

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The clinical failure of two large mGluR5-antagonist programmes in Fragile X syndrome has forced drug developers to rethink trial design and target selection for neurodevelopmental indications.

In September, Roche pulled the plug on its mGluR5 antagonist RG7090 in FXS, citing a lack of efficacy. Just 5 months earlier, Novartis had reported that its mGluR5 antagonist AFQ056 hadn't worked either and discontinued development in FXS. A smaller biotech, Seaside Therapeutics, who had partnered with Roche, discontinued trials of its own mGluR5 antagonist STX107 (and of its γ-aminobutyric acid type B receptor agonist STX209) nearly a year earlier when it ran out funding, and has since practically suspended its operations. What went wrong?

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  1. Asher Mullard
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Mullard, A. Fragile X disappointments upset autism ambitions. Nat Rev Drug Discov 14, 151–153 (2015). https://doi.org/10.1038/nrd4555

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