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KV-101 (formerly PAX-101) is an intravenous formulation of suramin, a polysulfonated naphthylurea originally developed in the early 20th century for the treatment of parasitic infections such as African sleeping sickness. Kuvatris Therapeutics (formerly PaxMedica) is repurposing KV-101 for the treatment of Autism Spectrum Disorder (ASD). The drug's mechanism of action involves the non-selective antagonism of purinergic receptors (P2X and P2Y), which are hypothesized to mediate the "cell danger response" (CDR). In ASD, it is proposed that cells remain in a persistent state of metabolic defense, and by blocking extracellular ATP signaling through these receptors, KV-101 aims to normalize cellular communication and improve neurodevelopmental symptoms. Kuvatris has also explored KV-101 for Human African Trypanosomiasis (HAT) via the 505(b)(2) regulatory pathway.
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