June 26 (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)