AIR001 is a sodium nitrite solution for intermittent inhalation via a nebulizer. It was initially developed by Mast Therapeutics to treat heart failure with preserved ejection fraction (HFpEF), but more recently, it has been the focus of clinical trials as a potential new therapy for cystic fibrosis (CF) and non-CF bronchiectasis.

Bronchiectasis is an irreversible condition that affects the respiratory tract. It is characterized by the progressive dilation, expansion, inflammation, and scarring of the airways that bring air to the lungs (the bronchi). This damage causes the obstruction of the airways and is detrimental for mucus clearance, which leads to the accumulation and growth of bacteria, further increasing the inflammatory state. Patients with bronchiectasis are normally prescribed a combination of bronchodilators, corticosteroids, and antibiotics.

How does AIR001 work?

AIR001 helps reduce bronchiectasis symptoms in two ways: by lessening inflammation and by improving pulmonary vasculature.

Bronchiectasis leads to an accumulation of mucus that tends to generate bacterial growth and cause inflammation. AIR001 reduces infection by blocking microbial growth by inhibiting bacterial oxygen consumption by nitrite.

It is also able to increase pulmonary vasodilation (dilatation of blood vessels in the lungs) through the action of nitrogen, and therefore reduce pulmonary vascular resistance, which increases the oxygenation of the blood and other major organs.

AIR001 dual action would help patients reduce inflammation in the bronchi and allow for a better oxygention of all tissues, therefore assisting with the major symptoms of bronchiectasis (cough, chest pain, shortness of breath, and fatigue).

Studies of AIR001 in non-CF bronchiectasis

Mast Therapeutics developed AIR001 for the treatment of HFpEF. Later, the company merged with Savara Pharmaceuticals, a company that’s focused on the development and commercialization of new therapies for the treatment of serious or life-threatening rare respiratory diseases. It will be tested in a Phase 2 clinical trial as a potential therapy for CF and non-CF bronchiectasis.

The aim of the Phase 2 trial is to determine the safety and correct dosage of AIR001 in adults with cystic fibrosis and airway infection. Lung function and bacterial density will also be analyzed to help determine the effectiveness of AIR001 in reducing infection and improving pulmonary function.

The study is being conducted by the University of Pittsburgh and the University of Pittsburgh Medical Center.

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