The Science Behind AER-01

AER-01 is a novel, best-in-class therapeutic engineered to dissolve mucus plugs by targeting and cleaving excessive mucin disulfide bonds.

The Importance of Removing Mucus Plugs in Patients with Respiratory Disease

  • Mucus plugs obstruct airflow in patients with lung diseases such as COPD, asthma, cystic fibrosis (CF), and non-CF bronchiectasis.
  • Oxidative stress in these diseases causes mucus gels to become more viscous and elastic, making it harder to clear the mucus.
  • Severe COPD and asthma patients are especially prone to mucus plugs and often experience reduced airflow, lower exercise tolerance, and higher all-cause mortality when they are present. Current treatments for COPD and asthma have a limited effect against mucus plugs.
  • It is important to clear mucus plugs to improve lung function, lung health, and quality of life in these patients.

How AER-01 Works

Pathologic mucus is characterized by excessive disulfide cross-linking between mucin polymers, leading to formation of mucus plugs that block airways.

AER-01 is an inhaled thiol-containing drug that liquifies mucus plugs by cleaving excessive disulfide bonds effectively.

Cleaving Disulfide Bonds

Heavily cross-linked
mucin polymers

Lightly cross-linked
mucin polymers

AER-01
Effectively Liquifies
Mucus In Minutes

Before

After

The First and Only Mucolytic for COPD and Asthma

While other mucolytic drugs exist for diseases like cystic fibrosis, AER-01 is the only therapy designed to target mucus plugs at the common pathway: disulfide bonds. AER-01 has greater potency and faster action than existing therapies in vitro, and it represents a potential breakthrough in clearing mucus-obstructed airways.

plugged-airway

Plugged airway

Open airway

Precision Imaging to Confirm Effect

Aer Therapeutics uses state-of-the-art lung imaging to determine how effective AER-01 is in clearing mucus plugs in patients with COPD and asthma. Imaging provides a visualization of plugged airways and quantitative measure of plug decrease, in addition to tracking functional improvements in spirometry and symptom relief in patients.

Clinical Trial

AER-01 is currently being evaluated in a Phase 2a clinical trial underway in Australia, New Zealand, and the United Kingdom. The study is assessing the safety and efficacy of once-daily dosing over 28 days in patients with moderate-to-severe COPD, with a focus on improvement in lung function. Preliminary results are anticipated in Q2 2026.

Presentation

AER Therapeutics:
Targeting Mucus Obstruction to Improve Lung Health.

Exploratory Endpoints

Aer Therapeutics has initiated a Phase 2a proof-of-concept clinical study (AER-01-002) for AER-01 in chronic obstructive pulmonary disease.

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