LANGHORNE, PA — In a significant advancement for patients suffering from autoimmune Pulmonary Alveolar Proteinosis (aPAP), Savara Inc. has revealed promising outcomes from its IMPALA-2 Phase 3 clinical trial. The study, focusing on the efficacy and safety of molgramostim, an inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), met its primary endpoint and demonstrated sustained benefits, marking a potential turning point in aPAP treatment.
The IMPALA-2 trial, a rigorous 48-week, double-blind, placebo-controlled study, was designed to assess the impact of daily inhaled molgramostim treatments on adult patients with aPAP. The trial’s primary measure was the improvement in lung function, specifically the mean change from baseline to Week 24 in hemoglobin-adjusted percent predicted diffusing capacity for carbon monoxide (DLCO). Results showed a statistically significant improvement, a success that was also observed at Week 48, confirming the treatment’s lasting effects.
Importantly, the trial also noted significant improvements in the St. George’s Respiratory Questionnaire (SGRQ) Total Score and Activity Score at Week 24, alongside improved exercise capacity at Week 48. These findings underscore molgramostim’s potential to enhance the quality of life for aPAP patients significantly.
Dr. Bruce Trapnell, Professor of Medicine and Pediatrics at the University of Cincinnati College of Medicine and Lead Clinical Investigator of the IMPALA-2 trial, highlighted the urgent need for effective treatments for aPAP, stating, “With convincing data from two large clinical trials, the evidence now clearly demonstrates molgramostim has the potential to be a safe and efficacious treatment option for these patients.”
Safety results were encouraging, with molgramostim demonstrating a tolerable profile comparable to the placebo group. Adverse events, including COVID-19, cough, and pyrexia, were generally similar across both treatment and placebo groups, with only two patients discontinuing due to unrelated adverse events.
Matt Pauls, Chair and CEO of Savara, expressed his optimism regarding the trial’s outcomes, noting, “The IMPALA-2 results not only met but exceeded our expectations, validating our hypothesis that molgramostim provides clear, durable improvement in gas exchange, and beyond that, clinical benefits that positively impact the quality of life for aPAP patients.”
Molgramostim’s development as a treatment for aPAP has received significant regulatory endorsements, including Orphan Drug, Fast Track, and Breakthrough Therapy designations from the U.S. Food and Drug Administration, among other international recognitions. These endorsements reflect the potential of molgramostim to fill a critical gap in aPAP treatment, offering hope to patients grappling with this challenging autoimmune condition.
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