Savara reported that its daily treatment for a rare lung disease met the primary endpoint in a key late-stage study.
The company is developing molgramostim for autoimmune pulmonary alveolar proteinosis (aPAP) — a disease in which substances build up in the lung’s air sacs and cause breathing problems.
In the Phase 3 IMPALA-2 study, Savara’s therapy improved lung function as measured by a test called DLCO — diffusing capacity of the lungs for carbon monoxide — compared to placebo at 24 weeks. That difference was maintained at 48 weeks, Savara said Wednesday.
Bruce Trapnell“Patients typically experience breathlessness, which begins slowly and progresses over time, often accompanied by cough and fatigue, and in some patients, serious infections, pulmonary fibrosis and respiratory failure requiring lung transplantation,” Bruce Trapnell, a professor at the University of Cincinnati College of Medicine and lead investigator for the study, said in a statement.
Molgramostim, delivered via a nebulizer, is a recombinant form of an immune system regulator called granulocyte-macrophage colony-stimulating factor. In aPAP, autoantibodies block that factor’s signaling, which is needed for the body to clear the lung’s air sacs.
“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,” Trapnell said.
As for safety, those who received Savara’s therapy in the Phase 3 trial and those who received placebo experienced a similar frequency of adverse events.
Savara’s shares {SVRA} fell 3% Wednesday morning.
The company said it plans to file an application to the FDA for approval in the first half of 2025, and Jefferies analyst Andrew Tsai estimated the treatment could be on the market by early 2026. Tsai also projected peak sales of over $400 million.
Back in 2020, Savara reported success in a Phase 2 study of molgramostim in the rare lung disease that was published in the New England Journal of Medicine.