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Savara's rare lung disease therapy succeeds in late-stage study
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Savara's rare lung disease therapy succeeds in late-stage study
Jun 26, 2024 7:37 AM

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)

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