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STP1 is a fixed-dose combination of two small molecule drugs—ibudilast and bumetanide—being developed by STALICLA for the treatment of Autism Spectrum Disorder (ASD), specifically in a biologically defined subgroup known as ASD Phenotype 1 (ASD-Phen1)[2][4][5]. Ibudilast acts as a pan-phosphodiesterase (PDE) inhibitor with anti-inflammatory properties, while bumetanide is a modulator of the sodium-potassium-chloride cotransporter NKCC1[5][7]. The combination targets neuroinflammation and abnormal neuronal signaling implicated in ASD. Mechanistically, it functions through inhibition of phosphoric diester hydrolases (phosphodiesterases) and sodium-potassium-chloride symporters[2], aiming to address core biological dysfunctions in ASD-Phen1. Clinical studies have shown that STP1 is well-tolerated and demonstrates EEG-based target engagement in relevant brain regions[4]. The drug was identified using an AI-driven precision medicine platform to match specific patient subgroups with targeted therapies[5].
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