Savara Inc. announced the presentation of results from its Phase 3 IMPALA-2 trial of molgramostim inhalation solution for patients with autoimmune pulmonary alveolar proteinosis (aPAP) at the British Thoracic Society (BTS) Winter Meeting 2024 in London. The presentation, titled "Inhaled Molgramostim Improves Pulmonary Gas Exchange and Respiratory Health-Related Quality of Life in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP): Results from IMPALA-2," was delivered by Cormac McCarthy, M.D., Ph.D., from University College Dublin.
IMPALA-2 Trial Highlights
The IMPALA-2 trial evaluated the efficacy and safety of molgramostim in patients with aPAP. Molgramostim, a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), aims to address the underlying pathophysiology of aPAP, where GM-CSF is neutralized by antibodies, impairing alveolar macrophage function and leading to surfactant accumulation.
The study's findings suggest that molgramostim improves pulmonary gas exchange and enhances the respiratory health-related quality of life for individuals affected by aPAP. These results underscore the potential of molgramostim as a therapeutic intervention for this rare lung disease.
Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Autoimmune PAP is characterized by the build-up of surfactant in the alveoli of the lungs, impairing gas exchange and causing shortness of breath, cough, and fatigue. In a healthy lung, alveolar macrophages clear excess surfactant, a process stimulated by GM-CSF. However, in aPAP, antibodies neutralize GM-CSF, preventing macrophages from functioning properly.
Molgramostim: A Targeted Approach
Molgramostim is delivered via an investigational eFlow® Nebulizer System, specifically designed for the inhalation of large molecules. By providing GM-CSF directly to the lungs, molgramostim aims to restore macrophage function and facilitate the clearance of excess surfactant in aPAP patients.
Savara's focus on rare respiratory diseases and its commitment to advancing molgramostim through clinical development highlight the company's dedication to addressing unmet needs in pulmonary medicine.
