Savara reported on Wednesday that its experimental therapy met the primary endpoint of a phase 3 trial by improving a measure of lung function in patients with a rare disease that causes breathing difficulties.
The study included a total of 184 patients with autoimmune pulmonary alveolar proteinosis (aPAP), a disease that can cause shortness of breath, scarring in the lungs, and even lead to a need for a lung transplant.
Savara's inhaled therapy, molgramostim, improved a measure of lung capacity that tracks the ability of the lungs to transfer gas from inhaled air to red blood cells in blood capillaries in the lung, compared with a placebo.
The disease, which currently has no approved treatments, is estimated to affect fewer than 5,000 people in the U.S., according to government data.
Analyst Andrew Tsai predicted a likely approval for the therapy by early 2026 and more than $400 million in peak U.S. sales.
Tsai noted that the therapy's safety profile appeared clean, and patient discontinuation rates due to its common adverse events of coughing and chest pains were low.
The therapy also met the secondary endpoint of helping improve overall respiratory health, based on a questionnaire designed to measure health impairment in patients.
Molgramostim could fundamentally change the way that aPAP is treated, according to CEO Matt Pauls during a conference call. The current standard of care for the disease is an invasive medical procedure in which the patient's lungs are washed with saline.
Pennsylvania-based Savara planned to submit a marketing application for the therapy in the first half of 2025 and stated that it expected to present full data from the trial at a scientific conference later that year.