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STX209

Seaside Therapeutics LLC

Drug Names(s): arbaclofen

Description: STX209 (arbaclofen) is the purified R-isomer form of the approved drug baclofen, a derivative of gamma-aminobutyric acid (GABA-B) that modulates the GABA-B receptor and is primarily used to treat spasticity. Arbaclofen is a selective gamma-amino butyric acid type B (GABA-B) receptor agonist, which inhibits glutamate signaling in the brain and should thereby indirectly inhibit excessive metabotropic glutamate receptor (mGluR) mediated protein synthesis. Patients with fragile X syndrome, the most common heritable mental retardation and a leading cause of autism, have a mutation in the FMRP gene, which normally inhibits protein synthesis stimulated by the metabotropic glutamate receptor 5, or mGluR5.

Deal Structure: In June 2012, Roche and Seaside Therapeutics entered into a collaboration to develop disease modifying treatments for fragile X syndrome (FXS) and autism spectrum disorders (ASD). Seaside will develop its GABA-B agonist program and retains exclusive rights to issued and pending patents covering the use of GABA-B agonists for the treatment of FXS and ASD. Roche may exercise options to commercialize STX209 upon completion of certain clinical development phases in FXS and ASD, but Seaside will continue to lead the clinical development of these programs. Additional terms of the transaction were not be disclosed. In June 2013, Roche cancelled its license agreement with Seaside for STX209.


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