LANGHORNE, PA — Savara Inc. (Nasdaq: SVRA) announced that the results of its Phase 3 IMPALA-2 clinical trial for molgramostim, an inhaled therapy for autoimmune pulmonary alveolar proteinosis (autoimmune PAP), will be published in the New England Journal of Medicine on August 21, 2025. The findings highlight significant clinical benefits and a strong safety profile for patients with this rare, chronic lung disease.
Significant Improvements in Lung Function
The trial achieved statistical significance on its primary endpoint, demonstrating that molgramostim significantly improved pulmonary gas transfer compared to placebo. At Week 24, patients receiving molgramostim showed an average 9.8% improvement in the diffusing capacity of the lungs for carbon monoxide (DLco%), compared with 3.8% for placebo—a treatment difference of 6.0% (P<0.001).
These benefits persisted through Week 48, where the treatment group maintained an 11.6% improvement versus 4.7% for placebo (P<0.001). These results are comparable to improvements considered clinically meaningful in other serious lung conditions, such as pulmonary fibrosis.
Enhanced Quality of Life and Functional Capacity
Molgramostim also demonstrated significant gains in respiratory health-related quality of life. Patients reported greater improvements in total scores on the St. George’s Respiratory Questionnaire (SGRQ), with reductions of -11.5 points at Week 24 versus -4.9 points for placebo (P=0.007). The therapy similarly improved SGRQ activity scores and exercise capacity, measured via peak metabolic equivalents (peak-METs). At Week 48, molgramostim-treated patients improved 1.1 METs, nearly double the gains seen in the placebo group.
Reduction in Disease Burden
The study also found molgramostim reduced pulmonary surfactant buildup, a hallmark of autoimmune PAP. Ground glass opacity (GGO) scores—an indicator of surfactant burden measured via CT scan—declined more significantly in treated patients. Additionally, fewer patients in the treatment group required whole lung lavage, a rescue therapy often used for severe cases (7% vs. 13%).
Strong Safety Profile
The therapy was well tolerated, with most adverse events reported as mild or moderate. Completion rates were high, with 98% of patients in the molgramostim group and 96% of those in the placebo group completing the 48-week treatment period.
Autoimmune PAP
Autoimmune PAP is a rare lung disease caused by an immune response that impairs the clearance of surfactant in the lungs, leading to breathing difficulties, fatigue, and potentially long-term complications such as fibrosis and lung failure. Current treatment options are limited, making molgramostim’s results particularly significant for patients and clinicians.
Global Impact
The IMPALA-2 trial, conducted at 43 clinical sites across 16 countries, represents the largest and longest Phase 3 study ever undertaken for autoimmune PAP. The promising results position molgramostim as a potential first-in-class therapy, offering a safer, more effective alternative to invasive procedures.
With publication in the NEJM, Savara’s findings could accelerate the path toward regulatory approvals and bring hope to patients worldwide affected by this rare and debilitating disease.
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