Study AER-01-002 is using a novel and precision imaging strategy to enroll 100 mucus plug-high chronic obstructive pulmonary disease (COPD) patients

AER-01-002 is well powered to determine if 28-days of once daily dosing of fexlamose is safe and improves lung function

Fexlamose has been shown in Phase 1 studies of 96 healthy volunteers to be safe, well tolerated and highly differentiated from first generation thiol-based mucolytic drugs

RALEIGH, N.C., Jan. 08, 2025 (GLOBE NEWSWIRE) — Aer Therapeutics (“Aer”), a biopharmaceutical company developing novel inhaled treatments for mucus-associated lung disease, announced today that the first patient has been dosed in its Phase 2a proof-of-concept (“POC”) clinical study of fexlamose (formerly “AER-01”) in Chronic Obstructive Pulmonary Disease (“COPD”).

The Phase 2a POC study (“AER-01-002”) is a randomized, double-blind, placebo controlled, parallel group study of fexlamose given once daily for 28 days to patients with COPD. Aer is utilizing a novel approach to identify and treat mucus plug-high COPD patients. Specifically, AER-01-002 will enroll 100 patients with moderate to severe COPD who are mucus plug-high. A mucus plug score is generated by analysis of computed tomography (CT) lung scans using a mucus plug scoring system developed at the University of California, San Francisco. This precision imaging approach to enrich AER-01-002 with mucus plug-high, moderate to severe COPD patients maximizes the sensitivity of the trial to detect the treatment benefits of fexlamose.

AER-01-002 was designed and powered to determine if once daily dosing of fexlamose, administered by nebulizer for 28 days, improves lung function as measured by forced expiratory volume in one second (“FEV1″), patient reported outcomes (“PRO”) including the St. George’s Respiratory Questionnaire (SGRQ-C), CT mucus plug score, and other secondary and exploratory functional, imaging and PRO endpoints. Both FEV1 and SGRQ-C are primary outcome measures accepted by regulatory agencies such as the United States Food and Drug Administration (“FDA”) and European Medicines Agency (“EMA”) for approval in COPD. AER-01-002 is being conducted in clinical sites in Australia and New Zealand, with plans to expand to the United Kingdom. Top-line data are expected in January 2026.

“We are highly encouraged by the rapid start-up of AER-01-002 and enrollment of our first patient in Australia. Our clinical investigators are very excited by the novel approach Aer is taking with fexlamose to potentially improve lung function and symptoms in COPD patients with moderate to severe disease. These patients have a large unmet medical need and targeting mucus plugs represents a rational and differentiated approach to increase their lung function and improve their lung health. We expect to complete AER-01-002 by the end of this year,” said Dr. John Fahy, founder and Chief Clinical Consultant for Aer.

About Fexlamose
Fexlamose is being developed as a chronic treatment for COPD and asthma in conjunction with standard therapies, to improve lung function, lessen symptoms and improve quality of life. Fexlamose may also be developed for the treatment of other muco-obstructive lung diseases, including cystic fibrosis (CF) and non-CF bronchiectasis.

Fexlamose previously demonstrated a strong safety and tolerability profile in a Phase 1 Single Ascending Dose and Multiple Ascending Dose clinical studies in 96 healthy volunteers. Adverse events were mild to moderate and self-resolving, and there were no serious adverse events (SAEs). There was no indication of bronchoconstriction and no clinically significant hematology or chemistry abnormalities. Pharmacokinetic data indicate suitability of fexlamose for once daily dosing. Importantly, fexlamose was deemed to be tolerable from a taste and smell profile due to its proprietary chemistry and manufacturing process and its physicochemical properties. This safety and tolerability profile potentially differentiates fexlamose from first generation thiol-based mucolytics such as Mucomyst® (N-Acetylcysteine, Bristol Myers Squibb).

Disulfide bridges cross-link mucin polymers to increase their elasticity and propensity to form mucus plugs. Fexlamose is a thiol-modified carbohydrate (“thiol-saccharide”) which cleaves mucin disulfide bridges to liquefy (“lyse”) mucus plugs. Carbohydrate scaffolds are natural and non-toxic, and their polar nature and high aqueous solubility allows them to easily penetrate mucus plugs. Fexlamose is a potent and fast acting mucolytic that is well-suited for pulmonary delivery as liquid inhalation solution by nebulizer or as dry powder.

Primary market research indicates that fexlamose sales could exceed $1 billion in the U.S. alone for the treatment of moderate to severe COPD and asthma.

About COPD
COPD is a group of lung diseases that include chronic bronchitis and emphysema that are characterized by breathlessness and chronic cough. COPD can be caused by inhaling pollutants, including tobacco smoke or workplace-related fumes, chemicals and dusts. Genetics can also play a causal role in some COPD patients. COPD is one of the leading causes of death in the United States. In 2021, it was the fourth leading cause of death worldwide, responsible for about 5% of all deaths. COPD, which affects millions of patients worldwide, has seen very limited treatment innovation in recent years. The airway component of COPD involves airway smooth muscle tightening, airway inflammation, and airway blockage with mucus plugs. Although several drugs are approved to treat the smooth muscle and inflammation components of COPD, none are approved to treat mucus plugs. The development of treatments for mucus plugs in COPD and other muco-obstructive lung diseases has been slowed by a lack of predictive and monitoring biomarkers for airway mucus plug pathology. AER-01-002 will use CT mucus plug scoring as a novel protocol design element to enrich the trial in patients who are more likely to demonstrate the treatment benefits of fexlamose.

About Aer Therapeutics
Aer Therapeutics is a clinical stage biopharmaceutical company with a mission to develop novel treatments for mucus-associated lung diseases such as COPD, asthma, cystic fibrosis (CF) and non-CF bronchiectasis. The company leverages the expertise of its scientific founders in airway mucus biology and pathology, medicinal glycochemistry, and inhaled pharmaceutics and the experience of its management team in drug development for lung disease. Aer is supported by a syndicate of premier life science industry investors including Canaan, OrbiMed, and Hatteras Venture Partners. Learn more at: www.aertherapeutics.com

Forward-Looking Statements
Certain of the statements made in this press release are forward looking, such as those, among others, relating to Aer’s expectations for fexlamose’s efficacy and safety, clinical trial enrollment goals and the timing of future clinical trials. Actual enrollment results, use of cash and other developments may occur that differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include risks that enrollment goals will not be met, trials may not be commenced or successful or may take longer to complete than anticipated, and projected cash needs and expected financial results may be different. Aer disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

CONTACT

Thomas P. Mathers
Executive Chairman
Aer Therapeutics
400 W North Street, Suite 112
Raleigh, NC 27603
+1 (617) 642-8677
tom.mathers@aertherapeutics.com