Savara has announced positive late-stage results for its daily molgramostim nebuliser solution (molgramostim) treatment for a rare lung disease.
The phase 3 IMPALA-2 clinical trial has been evaluating the efficacy and safety of molgramostim 300mcg administered once daily by inhalation versus placebo in adult patients living with autoimmune pulmonary alveolar proteinosis (aPAP).
Chronic respiratory diseases were the third-leading cause of global deaths in 2019 and can be caused by infection, smoking tobacco, radon, asbestos or other forms of air pollution.
Affecting around one in every 150,000 people in the US, aPAP is characterised by myeloid cell dysfunction, abnormal pulmonary surfactant accumulation and innate immune deficiency.
The trial met its primary endpoint after the therapy improved lung function in haemoglobin-adjusted percent predicted DLCO, a test that measures the diffusing capacity of the lung for carbon monoxide, achieving statistical significance compared to placebo at 24 weeks, which was maintained at 48 weeks.
Bruce Trapnell, lead investigator of the trial, commented: “With convincing data from two large clinical trials, the evidence now clearly demonstrates molgramostim has the potential to be a safe and efficacious treatment option for [aPAP] patients.”
Matt Pauls, chair and chief executive officer, Savara, said: “The strong efficacy data and favourable benefit-risk profile potentially positions molgramostim to be the first and only approved therapeutic for aPAP in the US and Europe.”
The inhaled form of recombinant human granulocyte-macrophage colony-stimulating factor has already been granted Orphan Drug, Fast Track and Breakthrough Therapy designations by the US Food and Drug Administration (FDA), Orphan Drug Designation from the European Medicines Agency (EMA), as well as the Innovative Passport and Promising Innovative Medicine designations from the UK’s Medicines and Healthcare Products Regulatory Agency for the treatment of aPAP.
Most recently, the FDA approved Verona Pharma’s Ohtuvayre (ensifentrine) as a maintenance treatment for adults living with chronic obstructive pulmonary disease (COPD), a common lung disease that affects more than 390 million people worldwide.
Prior to this, in May, Sanofi and Regeneron’s Dupixent (dupilumab) was recommended by the EMA’s human medicines committee as an add-on maintenance treatment for COPD, following positive late-stage results from the NOTUS trial, which were presented at the 2024 American Thoracic Society International Conference.
