STX209
STX209 (arbaclofen) is a selective gamma-amino butyric acid type B (GABA-B) receptor agonist.
Research suggests that individuals with autism and fragile X syndrome have abnormalities in synaptic transmission. Through the GABA-B receptor, STX209 may serve to restore the normal balance at the synapse and correct abnormalities associated with these disorders.
Program Status STX209 has successfully completed the largest ever randomized, blinded, placebo-controlled trial (Phase 2) in patients with FXS and an open-label exploratory trial in patients with ASD. We plan to initiate two Phase 3 FXS trials in North America and a Phase 2 trial in autism in mid 2011.
STX107
STX107 is a selective mGluR5 negative allosteric modulator (NAM).
Research indicates that mGluR5 NAMs normalize the excessive synaptic protein synthesis that is associated with fragile X syndrome. As fragile X syndrome is the most common known genetic cause of autism, it is hoped that mGlur5 NAMs may provide therapeutic benefit to some individuals with idiopathic autism. Treatments that target this receptor may also provide benefit for individuals with other neurodevelopmental disorders.
Program Status We plan to initiate a randomized, blinded, placebo-controlled Phase 2 trial in patients with FXS in 2011.
