Drug intelligence / Profile preview

AEX-635

Development stage
Preclinical
Lead developer
NLS Pharmaceutics
Modality
Small Molecules
01

Overview

**AEX-635** is a lead compound from the AEX-6xx series within the DOXA platform, consisting of novel dihydroquinazoline and dihydrobenzothiazine derivatives developed by Aexon Labs and licensed to NLS Pharmaceutics. It acts as a dual orexin receptor agonist targeting OX1R and OX2R to regulate sleep-wake cycles, motivation, and arousal, while also inhibiting cathepsin H (CTSH) for neuronal resilience; additionally, it modulates Multidrug Resistance-Associated Protein 1 (MRP1) to enhance neuroprotection, improve blood-brain barrier penetration, and boost CNS drug bioavailability under oxidative stress. Preclinical data demonstrate robust neuroprotective activity in α-synuclein-induced stress models, supporting evaluation in narcolepsy, hypersomnia, ADHD, and neurodegenerative diseases like Parkinson's and Huntington's, with IND-enabling studies planned.[1][3][7]

02

Targets

CTSH (Cathepsin H)HCRTR1 (Orexin/hypocretin receptor type 1)HCRTR2 (Orexin/hypocretin receptor type 2)ABCC1 (MRP1)

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