LANGHORNE, PA — Savara Inc. (Nasdaq: SVRA) reported new Phase 3 clinical trial data showing its experimental therapy molgramostim improved lung function and reduced disease-associated biomarkers in patients with autoimmune pulmonary alveolar proteinosis, a rare respiratory disorder with limited treatment options.
The findings, presented at the ATS 2026 International Conference in Orlando, provide additional evidence supporting the drug’s potential effectiveness as Savara advances efforts to bring a targeted treatment to market for the rare disease.
The data came from the double-blind portion of the IMPALA-2 Phase 3 study, which enrolled 164 patients globally. Participants received either inhaled molgramostim or a placebo once daily for 48 weeks.
Researchers measured pulmonary gas transfer using diffusing capacity of the lungs for carbon monoxide, or DLco%, a key indicator of how effectively oxygen moves from the lungs into the bloodstream.
According to the trial results, patients receiving molgramostim demonstrated statistically significant improvements in DLco% compared with placebo at both 24 weeks and 48 weeks.
The study also found significant reductions in several biomarkers associated with disease severity, including lactate dehydrogenase (LDH), cytokeratin 19 fragments (CYFRA 21-1), and Krebs von den Lungren protein-6 (KL-6).
Post-hoc analyses showed those biomarker reductions were accompanied by improvements in pulmonary gas transfer. Researchers reported strong correlations between declining biomarker levels and improved DLco% measurements across the overall study population.
The relationship was strongest for KL-6, a biomarker commonly associated with lung injury and disease activity in patients with autoimmune pulmonary alveolar proteinosis.
Changes in hemoglobin, hematocrit, and carcinoembryonic antigen (CEA) were similar between treatment groups.
The results add to the growing body of evidence supporting molgramostim’s therapeutic activity in autoimmune pulmonary alveolar proteinosis, a condition in which antibodies block granulocyte-macrophage colony-stimulating factor (GM-CSF), impairing the lungs’ ability to clear excess surfactant.
The disease can cause progressive shortness of breath, fatigue, chronic respiratory impairment, and, in severe cases, lung fibrosis or the need for transplantation.
The biomarker findings were presented by Dr. Y. Inoue during the conference and were derived from blood samples collected throughout the 48-week study period.
Savara has not yet disclosed regulatory filing timelines tied to the newly reported analyses. However, the data provide additional clinical support for molgramostim as the company pursues development of what could become one of the first targeted therapies specifically approved for autoimmune pulmonary alveolar proteinosis.
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