Savara's rare lung disease therapy succeeds in late-stage study

By Pratik Jain

(Reuters) -Savara said on Wednesday its experimental therapy met the main goal of a late-stage trial by improving a measure of lung function in patients with a rare disease that causes breathing difficulties.

The study tested 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, helped improve 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 less than 5,000 people in the United States, according to government data.

Jefferies analyst Andrew Tsai expects a likely approval for the therapy by early 2026, and more than $400 million in peak U.S. sales.

Tsai said the therapy's safety profile looked clean and patient discontinuation rates due to its common adverse events of coughing and chest pains were low.

The therapy also met the secondary goal 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, CEO Matt Pauls said on 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 salt water.

Pennsylvania-based Savara plans to submit a marketing application for the therapy in the first half of 2025 and said it expects to present full data from the trial at a scientific conference later this year.

(Reporting by Pratik Jain in Bengaluru; Editing by Tasim Zahid and Devika Syamnath)