Exclusive Summary

• First-in-Class Potential with Novel MOA: Selective inhibition of Agmatinase combined with Imidazoline and NMDA receptor modulation to precisely regulate endogenous Agmatine levels.
• Efficacy on Core ASD Symptoms: Pre-clinical data in VPA mice models demonstrates significant amelioration of social deficits and repetitive restricted behaviors (RRBs).
• Patent-Protected NCE: A New Chemical Entity with substance patent protection, offering higher exclusivity and commercial value compared to repositioned drugs.
• Restoration of E/I Balance: Targets the underlying pathology of Excitatory/Inhibitory imbalance in ASD through a unique metabolic pathway regulation.

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