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Savara Inc. (NASDAQ:SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, announced that the results from the Phase 3 IMPALA-2 clinical trial will be published online in NEJM. The manuscript, titled "A Phase 3 Trial of Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis" will appear in the August 21, 2025, online version of the publication and can be found at www.nejm.org. Following the online publication in NEJM, the manuscript will be available on the Congresses & Publications page of the Company's corporate website.
"IMPALA-2, the largest and longest Phase 3 clinical trial conducted in patients with autoimmune PAP, demonstrated that 48 weeks of once daily administration of inhaled molgramostim addresses the underlying pathophysiology of this chronic rare lung disease," said Bruce Trapnell, M.D., Professor of Medicine and Pediatrics, University of Cincinnati College of Medicine and Lead Clinical Investigator of the IMPALA-2 trial. "Treatment with molgramostim improved the cardinal manifestations of autoimmune PAP, namely it reduced pulmonary surfactant burden and improved pulmonary gas transfer, respiratory health-related quality of life, and patient functionality. Additionally, molgramostim was well tolerated with no notable safety concerns."
IMPALA-2 achieved statistical significance on its primary endpoint, change from baseline in the hemoglobin-adjusted percent predicted diffusing capacity of the lungs for carbon monoxide (DLco%) at Week 24. Molgramostim significantly improved pulmonary gas transfer as measured by DLco% at Week 24 compared with placebo (9.8% vs. 3.8%; estimated treatment difference, 6.0%; P<0.001 by comparison of least-squares means [LSMs]). The beneficial effect of molgramostim on pulmonary gas transfer was maintained to Week 48 as shown by the difference in LSM change from baseline in DLco% at Week 48 between molgramostim and placebo groups (11.6% vs. 4.7%; estimated treatment difference, 6.9%; P<0.001). The mean improvements in DLco% from baseline for molgramostim (9.8% at Week 24 and 11.6% at Week 48) are similar to the minimal clinically meaningful difference (MCID) in DLco% of 10% reported for pulmonary fibrosis.1
Molgramostim improved respiratory health-related quality of life as measured by the St. George's Respiratory Questionnaire (SGRQ) Total and Activity scores. The magnitude of LSM reduction from baseline in SGRQ Total score was significantly greater in the molgramostim group than placebo at Week 24 (-11.5 points vs. -4.9 points, estimated difference, -6.6 points; P=0.007) and remained numerically greater at Week 48 (-10.7 points vs. -5.9 points, estimated difference, -4.9 points [95% confidence interval, -10.8, 1.0]). The LSM change from baseline in SGRQ Activity score was greater for molgramostim compared with placebo at Week 24 (-13.0 points vs. -5.2 points, estimated difference, -7.8 points [95% confidence interval, -14.1, -1.5]) and at Week 48 (-13.4 vs. -7.4 points, estimated difference, -6.0 points [95% confidence interval, -13.6, 1.6]).
Molgramostim improved patient function as measured by exercise capacity (expressed as peak metabolic equivalents [METs]). A MET is a unit of resting oxygen uptake which measures energy expenditure. The LSM change from baseline in peak-METs was greater at Week 48 for molgramostim than placebo (1.1 vs. 0.6; estimated treatment difference, 0.6; [95% confidence interval, 0.1, 1.0]). Peak-METs is a clinically useful unit of measure reflecting exercise capacity, prognosis, and mortality in cardiovascular disease for which a 0.5-MET increase comprises a clinically useful measure of rehabilitation.2 Using the treadmill test in IMPALA-2, the LSM change in peak-METs from baseline to Week 48 was 1.1 in molgramostim-treated patients and the between-group difference observed at Week 48 was 0.6 peak-METs.
Molgramostim reduced surfactant burden as measured by ground glass opacity (GGO) score, an exploratory endpoint in IMPALA-2; GGO scores were determined by two blinded radiologists from a chest CT scan, with scores ranging from 0 to 15 (higher scores are worse). The mean reduction from baseline in GGO score was greater in the molgramostim group than the placebo group at Week 24 (-2.1 vs -1.1). Further, the number of patients who received ≥1 whole lung lavage as rescue therapy during the double-blind intervention period was lower with molgramostim than placebo (6 [7%] vs. 11 [13%]).
In IMPALA-2, molgramostim was well tolerated. Most treatment-emergent adverse events (AEs) were mild or moderate, and few led to discontinuation, with 98% of patients in the molgramostim group and 96% of patients in the placebo group completing the 48-week double-blind period on treatment.
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