Drug intelligence / Profile preview

KM-391

Development stage
Preclinical
Lead developer
Innovation Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

**KM-391** is a novel small molecule compound developed by Cellceutix Corporation for the treatment of autism spectrum disorders. In preclinical animal studies using rat models with neonatal serotonin depletion induced by 5,7-dihydroxytryptamine (5,7-DHT), oral administration of KM-391 at doses of 2.5-10 mg/kg over 40-90 days significantly improved brain plasticity, serotonin levels in the cortex and hippocampus, and behavioral functions including reduced anxiety, enhanced exploration, and mitigation of autism-like repetitive behaviors, self-injury, touch sensitivity, and social deficits, outperforming the active control fluoxetine. Additional studies confirmed its efficacy in counteracting oxytocin antagonist-induced exacerbation of autism-related behaviors, positioning it as a potential first-in-class therapy targeting core autism pathophysiology, though no human clinical trials have been reported.[1][3][7][10]

02

Targets

SERT (Sodium-dependent serotonin transporter)

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