phase 1
Severe Asthma
(Chronic Bronchitis)
Cystic Fibrosis
New Chemical


AR-001, our lead product candidate is a best-in-class dry powder formulation of niclosamide optimized for self delivery using a capsule-based dry powder inhalation device. This proprietary drug-device combination product benefits from the established safety on niclosamide and the optimal target coverage that will be achieved by direct delivery to the airways. AR-001 is currently being tested in IND enabling in vivo studies to establish safety and determine optimal clinical dose ranges.

Tier 1 Indications

Severe Asthma

Our lead indication is severe asthma. We expect to demonstrate improvements in lung function and symptoms by relaxing tightened airways and reducing mucus, something that no existing asthma treatment adequately achieves. Moreover, by blocking viral infection and upstream inflammation we believe that AR-001 will reduce exacerbations and compete well against expensive biologics that require injection.


(Chronic Bronchitis)
COPD patients also suffer from poor breathing due to inflammation and airway narrowing and achieve benefit from bronchodilators. We expect AR-001 to improve breathing in COPD patients by blocking multiple smooth muscle contractiles and reducing bronchitis-associated mucus and inflammation.

Anti Viral

(SARS - CoV-2)
Niclosamide has been discovered to be a potent inhibitor of SARS-CoV-2 and other respiratory viruses, including influenza, rhinovirus and RSV. Our shelf stable inhaled formulation of niclosamide is ideal for treating lung infections and drug evasion through mutation is not likely because it targets a host factor.


In addition to our primary indications of severe asthma and COPD, AR-001 holds promise to treat other diseases that involve pathologic calcium signaling in the airways. These Tier 2 indications are highlighted below and the company will seek opportunities to generate supporting preclinical data as appropriate.

Tier 2 Indications

Pulmonary Hypertension

TMEM16A is over-expressed in pulmonary arteries during pulmonary hypertension and inhibition may provide therapeutic benefit both by inducing smooth-muscle relaxation and reducing cell proliferation.


(Idiopathic Pulmonary Fibrosis)
Besides epithelial and smooth muscle cells, TMEM16 is expressed by lung fibroblasts and knockdown of TMEM16A expression ameliorates fibrosis in disease models. Therefore, there is a reason to believe IPF can be treated with AR-001.

Cystic Fibrosis

Cystic fibrosis is a disease associated with pathologic accumulation of dehydrated mucus in the airways. AR-001 represents a novel approach to treat CF because or TMEM16's role in mucus production and the fact that bronchodilators are known to improve the lung function of CF patients.

Building a Respiratory Disease Pipeline

Our work to date has led to a patent protected therapy directed against a validated new target for respiratory disease. We are strategically positioned to advance quickly to clinical trials.

Our small molecule and deep target biology knowledge positions us uniquely for identifying follow-on new chemical entities with improved activity. Our intention is to be the leaders in the development of inhaled TMEM16 inhibitors.