Drug intelligence / Profile preview

NPC86

Development stage
Preclinical
Lead developer
University of South Florida
Modality
Small Molecules
Administration
Intranasal
01

Overview

NPC86 is a novel therapeutic agent, likely a small molecule, developed to modulate the levels of the long noncoding RNA (lncRNA) Growth Arrest-Specific Transcript 5 (GAS5). GAS5 is a key regulator of insulin signaling and glucose metabolism; its depletion is associated with Type 2 Diabetes (T2D) and neurodegenerative conditions like tauopathy. NPC86 has demonstrated the ability to elevate GAS5 levels in vivo, leading to improved glucose tolerance and enhanced insulin sensitivity in diabetic mouse models. Furthermore, intranasal administration of NPC86 in tauopathy models (such as PS19 mice) has been shown to increase brain GAS5 levels, enhance insulin signaling via the pAKT pathway, and reduce tau phosphorylation, suggesting potential utility in treating Alzheimer's disease and related dementias.

02

Targets

GAS5 (Growth arrest-specific 5)UPF1

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