Drug intelligence / Profile preview

nni-362

Development stage
Phase 1
Lead developer
Neuronascent
Modality
Small Molecules
Administration
Oral
01

Overview

NNI-362 is a first-in-class, orally administered small molecule under development by NeuroNascent for the treatment of neurodegenerative diseases, primarily Alzheimer's disease. Identified through phenotypic screening for its ability to promote neurogenesis, NNI-362 acts as an allosteric stimulator of p70S6 kinase (70 kDa ribosomal protein S6 kinase). This mechanism promotes the proliferation and differentiation of human neural progenitor cells into mature neurons, potentially enabling regeneration of lost neurons in aging and disease. In preclinical models, NNI-362 restored cognitive function and increased new neuron formation in aged mice and models of Down syndrome and Parkinson’s disease. In a Phase 1a clinical trial in healthy older adults, NNI-362 was well tolerated at multiple doses up to 240 mg orally and significantly reduced plasma levels of phosphorylated tau (p-tau181), an Alzheimer’s biomarker associated with disease progression. The drug is blood-brain barrier penetrant in animal studies. Further clinical trials are planned or ongoing for mild-to-moderate Alzheimer’s disease and early Parkinson’s disease[1][3][4][5][7][9].

02

Targets

RPS6KB1 (Ribosomal protein S6 kinase beta-1)

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