Aer Therapeutics Begins Phase 2 Trial of Fexlamose for COPD

Aer Therapeutics, a biopharmaceutical company based in Raleigh, North Carolina, has announced the commencement of a Phase 2a proof-of-concept clinical study for its novel drug, fexlamose, targeting Chronic Obstructive Pulmonary Disease (COPD). This study, known as AER-01-002, is notable for employing a pioneering precision imaging technique designed to identify and include COPD patients with high levels of mucus plugging. The research aims to enroll 100 individuals diagnosed with moderate to severe COPD who exhibit significant mucus plug presence. This is determined through a specialized scoring system that analyzes computed tomography (CT) scans, developed at the University of California, San Francisco.

The clinical trial is structured as a randomized, double-blind, placebo-controlled study, wherein fexlamose will be administered once daily via nebulizer over a 28-day period. The primary objective is to ascertain the safety and efficacy of fexlamose in improving lung function, with outcomes being measured by forced expiratory volume in one second (FEV1) and patient-reported outcomes such as the St. George's Respiratory Questionnaire (SGRQ-C). These primary measures are recognized by regulatory bodies like the United States Food and Drug Administration and the European Medicines Agency for COPD treatment approval. The study, conducted across sites in Australia and New Zealand with plans to extend to the United Kingdom, expects to release top-line data in January 2026.

Fexlamose, previously referred to as AER-01, is positioned as a chronic treatment for both COPD and asthma, intended to enhance lung performance, alleviate symptoms, and elevate quality of life. There is potential for the drug to be applied to other mucus-related lung diseases, such as cystic fibrosis and non-CF bronchiectasis. Earlier Phase 1 trials involving 96 healthy volunteers demonstrated fexlamose's safety and tolerability, with no serious adverse effects reported. The pharmacokinetic profile supports its suitability for once-daily dosing, aided by its proprietary chemical makeup that offers a favorable taste and smell profile compared to first-generation thiol-based mucolytics like Mucomyst.

The mechanism of action for fexlamose involves cleaving disulfide bridges in mucin polymers, which are responsible for the elasticity and formation of mucus plugs. As a thiol-modified carbohydrate, fexlamose dissolves these plugs, making it a fast-acting mucolytic suitable for pulmonary delivery. This approach could potentially fill a gap in the treatment of COPD, where current therapies do not address mucus plugs.

COPD, encompassing conditions such as chronic bronchitis and emphysema, is a major health concern, ranking as one of the leading causes of death worldwide. Despite the availability of medications that target muscle inflammation and airway constriction, no treatments have been specifically approved to tackle mucus plugs. The AER-01-002 study aims to leverage CT mucus plug scoring to identify patients who could benefit most from fexlamose, potentially heralding a new era in COPD management.

Aer Therapeutics is committed to developing innovative solutions for mucus-associated lung diseases. The company draws on the specialized knowledge of its founders in airway mucus biology and medicinal chemistry, alongside a management team experienced in lung disease drug development. Supported by prominent life science investors such as Canaan, OrbiMed, and Hatteras Venture Partners, Aer Therapeutics is poised to make significant contributions to the field of respiratory medicine.