AeroRx Therapeutics Reports Positive Phase 2a Results for Inhaled AERO-007, the First Nebulized LABA/LAMA Combination Therapy in Development for COPD
AeroRx Therapeutics, Inc., a clinical-stage biopharmaceutical company specializing in proprietary nebulized combination therapies for chronic respiratory diseases, has announced encouraging topline results from its Phase 2a proof-of-concept study of AERO-007. This investigational candidate represents a significant advancement in the treatment of chronic obstructive pulmonary disease (COPD) as the first nebulized long-acting beta2 agonist (LABA) and long-acting muscarinic antagonist (LAMA) combination therapy in clinical development.
The trial results, which will be presented in full at an upcoming scientific conference, revealed that AERO-007 was well tolerated and delivered rapid-onset bronchodilation sustained over a full 24-hour period. Importantly, these effects were consistent across both low and high doses tested in the study. AERO-007 is a fixed-dose combination of two well-established bronchodilators—indacaterol (a LABA) and glycopyrrolate (a LAMA)—delivered in a proprietary formulation via a standard jet nebulizer already cleared under the U.S. FDA’s 510(k) pathway.
Addressing a Widespread Unmet Need in COPD Management
COPD affects over 16 million people in the United States alone, according to data from the American Lung Association, and it is widely regarded as one of the leading causes of morbidity and mortality worldwide. Despite the availability of multiple FDA-approved maintenance therapies—many of which include LABA/LAMA combinations administered via handheld inhalers—up to half of COPD patients continue to experience troubling symptoms such as breathlessness, coughing, wheezing, and fatigue. These symptoms often lead to reduced quality of life, frequent hospitalizations, and accelerated disease progression.
One major issue lies in the method of drug delivery. While handheld inhalers remain the standard mode of administration, they are not always suitable for a significant subset of patients. Clinical studies indicate that up to 75% of individuals using handheld inhalers do not receive the full intended therapeutic dose due to improper technique. Most inhalers require precise coordination, breath-holding, and a forceful inhalation, tasks that are often challenging for elderly patients or those with severe disease-related respiratory impairment. These patients may struggle with dexterity, cognition, or breathing strength—limitations that significantly impact their ability to use inhalers effectively.
Nebulized therapy presents a compelling alternative for such patients. Unlike inhalers, nebulizers convert liquid medication into an aerosol mist that can be inhaled during normal breathing, requiring no special coordination or breath control. This method is particularly advantageous for older adults, patients with advanced COPD, or those recovering from exacerbations who are at high risk for hospital readmission and poor outcomes.
Yet despite the clear benefits, the current landscape of nebulized therapies lacks a fixed-dose LABA/LAMA combination product. This gap has limited clinicians’ ability to follow the Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines, which recommend dual bronchodilation with a LABA/LAMA combination as first-line maintenance treatment for many patients. AeroRx Therapeutics, through its development of AERO-007, aims to fill this critical void.
Phase 2a Study Design and Findings
The AeroRx recently completed Phase 2a trial was designed as a randomized, double-blind, placebo-controlled, single-dose, three-period crossover study. A total of 16 adult participants diagnosed with moderate to severe COPD were enrolled. Each subject received both low-dose and high-dose AERO-007 treatments, as well as a placebo, at different points during the study, allowing for within-subject comparisons and robust efficacy assessments.
The primary efficacy endpoint was the change in forced expiratory volume in one second (FEV1), a standard spirometry-based measure of lung function that reflects a patient’s ability to exhale forcefully. Both doses of AERO-007 led to statistically significant and clinically meaningful improvements in FEV1 compared to placebo, demonstrating the drug’s rapid onset and sustained bronchodilatory effect over a 24-hour period.
Safety and tolerability were equally promising. No serious adverse events were reported, and the tolerability profile of AeroRx -007 was consistent with the known safety characteristics of its individual components—indacaterol and glycopyrrolate—both of which are already approved by the FDA as monotherapies delivered via inhalers. The systemic drug exposure observed in the study was within acceptable limits and aligned with previously published data on the active ingredients.
Expert and Executive Perspectives
Dr. Dave Singh, principal investigator of the study and Professor of Respiratory Medicine and Clinical Pharmacology at the University of Manchester in the United Kingdom, emphasized the significance of the results and the unmet need being addressed.
“Current COPD management guidelines universally endorse the use of dual bronchodilators—specifically LABA/LAMA combinations—as first-line therapy for moderate-to-severe patients,” said Dr. Singh. “Yet in real-world practice, we lack nebulized options that combine these agents. AERO-007 is the first investigational therapy that promises to close this gap, providing an important alternative for patients who cannot optimally use handheld inhalers. This could ultimately reduce symptom burden, exacerbation frequency, and hospitalizations.”
AeroRx Therapeutics’ Co-founder and Chief Executive Officer, Dr. Ahmet Tutuncu, M.D., Ph.D., echoed those sentiments and outlined the company’s strategic vision.
“With AERO-007, we are developing a best-in-class nebulized combination therapy that leverages two FDA-approved, clinically validated bronchodilators. Delivered through a widely available, reimbursable standard jet nebulizer, AERO-007 has the potential to become a new standard of care for a highly underserved segment of the COPD population,” said Dr. Tutuncu. “The favorable tolerability, consistent pharmacokinetic profile, and compelling efficacy data from our Phase 2a study provide a solid foundation for advancement into late-stage development.”
Dr. Tutuncu further confirmed the company’s plans to present the full dataset at a forthcoming scientific meeting and noted that preparations are underway for a late-stage, NDA-enabling clinical program.
A Platform for the Future
Beyond AERO-007, AeroRx Therapeutics is leveraging its proprietary formulation technology and platform capabilities to explore additional combination therapies and novel delivery mechanisms targeting chronic respiratory conditions. The company’s long-term goal is to build a diversified pipeline of inhaled therapeutics that optimize efficacy, convenience, and patient adherence, particularly for older populations and those living with complex, progressive pulmonary diseases.
AERO-007 stands at the forefront of this strategy. By combining two clinically proven bronchodilators in a single inhaled dose using a nebulizer—a device already familiar to many patients and caregivers—AeroRx aims to overcome longstanding barriers to effective COPD treatment. This includes not only the mechanical challenges of using inhalers but also systemic disparities in access, education, and healthcare delivery that often leave the most vulnerable COPD patients behind.
A Market with Urgent Demand
The commercial opportunity for a nebulized LABA/LAMA combination therapy is substantial. COPD is the third leading cause of death worldwide and generates tens of billions of dollars in annual healthcare costs in the U.S. alone. Rehospitalization rates following exacerbations remain unacceptably high, and treatment adherence continues to be a major challenge—one closely tied to the limitations of current delivery systems.
By offering a maintenance therapy that aligns more closely with patient needs and clinical realities, AERO-007 could improve long-term disease control, reduce emergency interventions, and enhance quality of life for millions of patients. Moreover, the use of an existing, reimbursable nebulizer platform simplifies the adoption pathway for both providers and payers.
As AeroRx prepares to initiate Phase 3 studies, the momentum behind AERO-007 continues to build. The company’s focus on combining clinical innovation with real-world applicability positions it as a leader in the emerging field of nebulized combination respiratory therapies.
“Our ultimate goal is to reshape the treatment paradigm in COPD and related chronic lung diseases,” Dr. Tutuncu concluded. “With AERO-007, we are one step closer to delivering on that promise—bringing a safe, effective, and accessible therapy to patients who need it the most.”
About AeroRx Therapeutics
AeroRx Therapeutics is a clinical-stage biopharmaceutical company developing proprietary, combination products for nebulized delivery to improve the treatment of chronic respiratory diseases. The company’s lead product candidate, inhaled AERO-007, is the first nebulized LABA/LAMA fixed-dose combination drug in clinical development as a first-line maintenance therapy for COPD and has the potential to help millions of patients who remain symptomatic, or poorly served, under existing drugs delivered via handheld inhalers or nebulized LABA or LAMA monotherapy.
A second program, AERO-111, is in development as the first nebulized LABA/LAMA/ICS triple therapy, for patients who escalate beyond dual bronchodilators. AeroRx’s leadership team has decades of expertise in aerosol drug delivery, clinical development, and regulatory strategy and guided multiple respiratory therapies from concept to regulatory approval. AeroRx is headquartered in the Avalon BioVentures Accelerator in La Jolla, California. For more visit, www.aerorxtx.com.
