Savara Inc., a biopharmaceutical company specializing in
rare respiratory diseases, recently showcased significant research at the American Thoracic Society (ATS) 2024 International Conference held from May 17-22 in San Diego, CA. Savara's partner, Trillium Health LLC, also presented significant advancements through a poster highlighting a newly developed laboratory blood test for diagnosing
autoimmune Pulmonary Alveolar Proteinosis (aPAP).
One of the key presentations was Poster Board 701, titled “Healthcare Burden of
Pulmonary Alveolar Proteinosis,” presented by Dr. E. Lee. This study, sponsored by Savara Inc., involved a retrospective cohort analysis using a longitudinal claims database (IPM.ai). It compared 2,312 patients diagnosed with pulmonary alveolar proteinosis (PAP) to 9,247 matched controls without PAP. The findings illustrated that PAP patients have significantly higher healthcare resource utilization and costs. Specifically, PAP patients experienced a fourfold increase in hospital admissions and cost of care, a sixfold increase in inpatient visits, and a higher incidence of comorbidities, procedures, and therapy usage. These results indicate a substantial unmet medical need in this patient population, emphasizing the necessity for improved diagnostic and treatment options.
Another noteworthy presentation was Poster Board 702, titled “Development of a Novel Laboratory Test for the Detection of
GM-CSF Antibodies to Aid in the Diagnosis of Individuals At-risk for Autoimmune Pulmonary Alveolar Proteinosis (aPAP),” presented by A. Ali and J. Kim from Trillium Health LLC. This research introduced a new laboratory test designed to detect granulocyte-macrophage colony-stimulating factor (GM-CSF) antibodies in human serum. The test demonstrated a clinical accuracy of 100% in a cohort of 40 subjects, half of whom were positive and half negative. This particle-based flow cytometry immunoassay is highly precise and sensitive, making it a valuable tool for diagnosing aPAP by accurately determining GM-CSF antibody levels.
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare lung condition characterized by an abnormal accumulation of surfactant, a mix of proteins and lipids, in the alveoli. Normally, alveolar macrophages clear out used surfactant, but in aPAP, antibodies against GM-CSF hinder this process. This results in a build-up of surfactant, obstructing gas exchange and causing patients to suffer from
shortness of breath,
chronic cough,
fatigue, and other respiratory symptoms. Over time, aPAP can lead to complications like
lung fibrosis and may necessitate a lung transplant.
Savara Inc. is at the forefront of fighting rare respiratory diseases with its lead program,
molgramostim nebulizer solution, which is currently in Phase 3 development for treating aPAP. Molgramostim is an inhaled GM-CSF delivered through an investigational eFlow® Nebulizer System from
PARI Pharma GmbH. The company's management team has extensive expertise in rare respiratory diseases, ensuring the identification of unmet needs and advancing promising treatments toward regulatory approval and commercialization.
These presentations at the ATS International Conference underscore the company's commitment to addressing significant health challenges associated with
rare respiratory conditions through advanced research and innovative solutions.
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