May 18, 2025 at 2:30 p.m. PT
LANGHORNE, Pa.—(BUSINESS WIRE)—Savara Inc. (NASDAQ: SVRA), a biopharmaceutical company targeting rare respiratory diseases, unveiled promising Phase 3 trial results for its experimental therapy molgramostim in autoimmune pulmonary alveolar proteinosis (aPAP) at the ATS International Conference 2025. The data, drawn from 157 patients, underscore molgramostim’s potential to address a critical unmet need in a condition affecting fewer than 1,000 individuals annually in the U.S.
ATS 2025 Poster Highlights
Poster Title: Molgramostim Reduces Surfactant Burden and Number of Whole Lung Lavage Procedures in aPAP Patients: IMPALA-2 Phase 3 Clinical Trial Results
Presenter: Dr. Tisha S. Wang, Professor of Clinical Medicine and Senior Executive Clinical Vice Chair at UCLA Department of Medicine
Poster Number: 918
Location: PD05, Room 3009/3011 West Building, Level 3
Summary:
- Molgramostim demonstrated a statistically significant reduction in pulmonary surfactant buildup, as quantified by ground-glass opacification (GGO) scores—a critical radiological biomarker in aPAP. Patients receiving the drug experienced mean GGO score reductions of 2.1 compared to 1.1 in placebo cohorts (P=0.0004).
- Clinically relevant outcomes showed 7.4% of molgramostim-treated patients required rescue whole-lung lavage (WLL) procedures over 48 weeks, versus 13.3% in the placebo group. The drug cohort underwent 15 WLLs compared to 24 for controls.
- Reinforcing its mechanism of action, molgramostim targets the root cause of aPAP—impaired GM-CSF signaling—which hinders macrophage function responsible for surfactant clearance.
Poster Title: The Effects of Molgramostim on Respiratory Health-related Quality of Life and Patient-reported Outcomes in aPAP
Presenter: Dr. Ali Ataya, Associate Professor of Medicine, University of Florida Division of Pulmonary and Critical Care
Poster Number: P31
Location: TP22, Area A, Hall F (North Building, Exhibition Level)
Summary:
- Patients reported measurable improvements in health-related quality of life (HRQoL) metrics, including the St. George’s Respiratory Questionnaire Impact Score (P=0.0084) and Symptom Score, with these benefits sustaining through the trial’s 48-week period.
- The EuroQol 5D-5L survey—a standardized HRQoL assessment—showed numerical advantages for molgramostim in three key dimensions: mobility, self-care, and usual activities.
- Patient-reported severity assessments (PGIS) revealed significant reductions in breathing difficulties at Weeks 24 (P=0.0305) and 48 (P=0.0049). Physical activity improvements were quantified through higher self-ratings of “Much better” or “A little better” among 62% of the molgramostim group versus 47% in placebo at conclusion.
Implications for Care Pathways
Researchers suggest these findings could disrupt aPAP’s standard of care, which has relied solely on invasive and resource-intensive WLL procedures since the disease’s identification in 1958. Dr. Wang noted, “For a condition with no approved therapies, demonstrating sustained surfactant clearance through an inhalation device represents a paradigm shift.”
Publication Details
IMPALA-2 trial abstracts were published in American Journal of Respiratory and Critical Care Medicine and will be discussed further this week at ATS 2025. Trial data will form the basis of future regulatory submissions with peak exposure positioning Savara to capitalise on a rare disease void in respiratory oncology.
Subluxation of Market Opportunity
The 48-week Phase 3 readouts extend molgramostim’s efficacy timeline, with open-label extension studies now evaluating long-term safety across 96-week periods. This positions Savara to potentially leverage accelerated FDA pathways for rare disease treatments, targeting cost efficiencies in a market currently dependent on hospitalization-based WLL specialists.
DSNU Rare Respiratory Development
Savara’s focus spans aPAP and other niche pulmonary diseases through its proprietary eFlow® nebulization system. The team deployed includes veterans of pivotal trials for orphan respiratory drugs, with CEO Robert Savage stating earlier this year that “Our platform enables delivery of large molecules directly to the lungs—a space emerging from decades of limited innovation.”
For additional information about aPAP, the IMPALA-2 trial, or Savara’s pipeline, visit their website.
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