Drug intelligence / Profile preview

AR-001

Development stage
Preclinical
Lead developer
Aeon Respire
Modality
Small Molecules
Administration
Inhalation
01

Overview

AR-001 is an inhaled dry powder formulation of niclosamide being developed by Aeon Respire for the treatment of various severe respiratory diseases, including severe asthma, chronic obstructive pulmonary disease (COPD), and idiopathic pulmonary fibrosis (IPF). Niclosamide, originally an approved anthelmintic, was identified through target-based screening as a potent inhibitor of the calcium-activated chloride channel TMEM16A (also known as Anoctamin-1). TMEM16A is a key regulator of calcium flux in the airways and is essential for airway smooth muscle contraction and mucin hypersecretion. By pharmacologically blocking TMEM16A, AR-001 aims to reduce bronchoconstriction, decrease mucus production, and inhibit TGF-β-mediated fibrosis. Additionally, the drug has shown potential in blocking infections by airway viruses. AR-001 is currently in the preclinical stage of development.

Other names
niclosamide dry powder inhaled formulationinhaled niclosamide
02

Targets

ANO1 (Anoctamin-1)

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