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IAMA-6 is a selective small molecule inhibitor of the sodium-potassium-chloride cotransporter 1 (NKCC1), developed to restore neuronal excitability by modulating chloride ion transport in neurons. The drug is being developed by IAMA Therapeutics as a potential treatment for autism spectrum disorders and drug-resistant epilepsy, with additional exploration in other central nervous system (CNS) indications. Preclinical studies have demonstrated efficacy in models of idiopathic and syndromic autism as well as refractory epilepsy, and clinical development has advanced to Phase 1 trials evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy adults. The mechanism of action targets NKCC1-associated neuronal hyperexcitability—a process implicated in various neurological disorders—by selectively inhibiting this transporter to rebalance inhibitory signaling within the brain[1][3][4][7].
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