Savara Presents Phase 3 IMPALA-2 Trial Data for Molgramostim in aPAP at ERS Congress 2024

Savara Inc., a biopharmaceutical company focused on rare respiratory diseases, has announced new findings from its Phase 3 IMPALA-2 clinical trial. This trial investigates the efficacy of molgramostim for patients with autoimmune Pulmonary Alveolar Proteinosis (aPAP). These results were presented at the European Respiratory Society (ERS) Congress 2024 in Vienna, Austria.

The IMPALA-2 trial achieved its primary endpoint, showing a statistically significant improvement in the hemoglobin-adjusted percent predicted diffusing capacity of the lungs for carbon monoxide (DLCO%) at Week 24. This significant benefit was maintained through Week 48, demonstrating the durability of molgramostim's effects. Additionally, molgramostim showed significant improvements over placebo in the St. George’s Respiratory Questionnaire (SGRQ) Total Score at Week 24 and in exercise capacity at Week 48.

Molgramostim was well tolerated, with 97% of patients completing the 48-week double-blind treatment period. Only two patients discontinued due to adverse events not related to the treatment. All patients who completed the double-blind phase chose to continue into the 96-week open-label period.

Additional data presented at the ERS Congress further support molgramostim’s benefits. Improvements were seen in the Disease Severity Score (DSS) at Weeks 24 and 48, DLCO% responder analysis at both weeks, and the Ground Glass Opacification (GGO) score at Week 24. These results underscore molgramostim’s ability to reduce surfactant burden, increase pulmonary gas transfer, enhance quality of life, and improve exercise capacity in aPAP patients.

The promising results from IMPALA-2 suggest that molgramostim effectively addresses the pathobiology of aPAP, a rare lung disease marked by the build-up of surfactant in the alveoli. This build-up is due to the neutralization of granulocyte-macrophage colony-stimulating factor (GM-CSF) by antibodies, leading to the accumulation of surfactant and impaired gas exchange. Symptoms include shortness of breath, cough, fatigue, and potentially severe complications like lung fibrosis.

Savara plans to submit a Biologics License Application (BLA) for molgramostim in the first half of 2025. The drug has received Orphan Drug, Fast Track, and Breakthrough Therapy designations from the U.S. Food and Drug Administration, as well as Orphan Drug designation from the European Medicines Agency. It also holds Innovative Passport and Promising Innovative Medicine designations from the UK's Medicines and Healthcare Products Regulatory Agency.

The IMPALA-2 trial is a significant global study involving 43 sites across 16 countries, including the U.S., Canada, Japan, South Korea, Australia, and several European nations. The trial's primary efficacy assessment focuses on the change in DLCO% from baseline to Week 24, with additional evaluations at Week 48 to measure the durability of the treatment’s effect. The study also assesses safety through Week 48, while all participants who completed this period are continuing with a 96-week open-label treatment with molgramostim.

Savara remains dedicated to advancing treatments for rare respiratory diseases. Their lead program, molgramostim nebulizer solution, is specifically developed for inhalation using the investigational eFlow® Nebulizer System. The company is committed to addressing unmet medical needs and efficiently advancing potential treatments to approval and commercialization.