#USA #Airway Therapeutics #Zelpultide Alfa #pediatric approval #Marietta, Georgia

Airway Therapeutics Secures Approval for Zelpultide Alfa Study

In a major milestone, Airway Therapeutics, Inc. has received approval from the Pediatric Committee of the European Medicines Agency (EMA) for its pediatric investigation plan (PIP) regarding the experimental biologic product, Zelpultide Alfa. This innovative therapy is aimed at preventing bronchopulmonary dysplasia (BPD) in very preterm infants born between 22 and 27 weeks of gestation.

The ongoing phase 2b dose selection and phase 3 expansion study for Zelpultide Alfa is slated to commence at the end of the third or fourth quarter of 2025. This program marks an important step toward addressing a critical unmet medical need, as no preventive treatment for BPD has received approval to date.

Dr. Marc Salzberg, President and CEO of Airway Therapeutics, expressed his enthusiasm for the approval, stating, “We are delighted to have reached an agreement with the PDCO on the pediatric investigation plan and pivotal study design for Zelpultide Alfa. This revolutionary therapy aims to prevent BPD in this highly vulnerable population.” Zelpultide Alfa is a recombinant human surfactant D protein designed to modulate immune responses, decrease inflammation, and enhance pathogen clearance, ultimately supporting lung function.

The approved PIP outlines a comprehensive design and execution plan for a pivotal clinical trial featuring a phase 2b trial that will test two different doses of Zelpultide Alfa over a period of up to seven days, compared to a placebo. Following treatment for 150 patients, with 50 patients in each trial arm, a suitable dose will be chosen for continuation into phase 3. In this subsequent stage, an additional 216 patients will be recruited, contributing to the understanding of BPD through a focus on consecutive three-day evaluations during the trial.

BPD remains one of the most challenging complications faced in neonatal intensive care, often leading to long-term respiratory and neurodevelopmental issues. Dr. Daniele De Luca, the lead investigator and a professor of pediatrics (neonatology) at the University of Paris Saclay, highlighted the novel potential of Zelpultide Alfa, saying, “This study will allow us to rigorously evaluate its potential to change clinical practice by tackling the underlying causes of lung injury in extremely preterm infants.”

Following the EMA's approval, Airway Therapeutics plans to promptly submit the approved protocol to health authorities in Italy (AIFA), Spain (AEMPS), and Israel (MoH), aiming to initiate clinical trials in these regions by late 2025. Additional submissions will follow in France, Germany, Belgium, and Poland, with regulatory filings also expected in Argentina, Australia, and the United States.

About Zelpultide Alfa

Zelpultide Alfa is an innovative recombinant human surfactant D protein that mimics the complete quaternary structure and biological function of the native protein. It employs advanced cell line technology to ensure optimal glycosylation. The therapeutic action of Zelpultide Alfa relies on three critical mechanisms: modulating immune responses to reduce excessive inflammation, enhancing the recognition and clearance of pathogens, and preserving surfactant homeostasis to support lung function. As a versatile biological platform, Zelpultide Alfa is being developed for patients across various age groups, addressing not only BPD prevention in very preterm infants but also treating acute and chronic respiratory, inflammatory, and infectious conditions in adults.

The primary indication of Zelpultide Alfa—preventing BPD—is undergoing pivotal phase 2b/3 trials following a phase 1 study that demonstrated a favorable safety and tolerability profile.

About Airway Therapeutics

Airway Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing a new class of biological therapies aimed at redefining the prevention and treatment of respiratory, inflammatory, and infectious diseases. Its lead candidate, Zelpultide Alfa, is currently advancing through the final stages of clinical development for preventing BPD in extremely preterm infants—a disease for which no preventive treatment exists. Airway Therapeutics is actively pursuing broader applications of the Zelpultide Alfa platform to enhance outcomes for vulnerable patient populations.

For more information, please visit Airway Therapeutics.