Molgramostim, from Savara Pharma, is an un-glycosylated GM-CSF produced in the Escherichia coli bacteria. Molgramostim has been genetically adapted to contain a human GM-CSF gene. It is currently undergoing Phase 1 studies for bronchiectasis treatment.
GM-CSF, also known as granulocyte macrophage-colony stimulating factor (rhGM-CSF), has two roles: One is to act as a messenger protein (cytokine) and the other is to serve as a growth factor for the production of blood cells (called hematopoietic growth factor).
Hematopoietic growth factors are involved in the production of blood cells from progenitors in the bone marrow, making them useful in different clinical situations. Cytokines are proteins that are produced by some cells of the immune system and are involved in immunity, inflammation, and blood production (hematopoiesis).
How does molgramostim work?
The body’s GM-CSF regulates both the balance of lungs’ surfactant, which is a complex substance produced in the lungs and serve as the lung’s defense through natural immune functions. Molgramostim influences the growth, differentiation, and function of granulocytes, macrophages, and eosinophils, which are types of white blood cells.
A nebulizer containing molgramostim has been developed by Serendex as inhalation treatment for respiratory conditions such as aPAP, bronchiectasis, and cystic fibrosis.
Molgramostim studies
A Phase 1, randomized, double-blind, placebo-controlled clinical trial (NCT02468908) in healthy adults has been completed in the United Kingdom. It assessed the safety and tolerability of single ascending and multiple ascending doses of molgramostim in humans.
The primary objective was the number and severity of adverse events resulting from the treatment. Secondary objectives were the evaluation of molgramostim effects in the human body. Other objectives were to check if antibodies against molgramostim developed during treatment; the change in white blood cell count; the change in the exhaled fraction of nitric oxide; and heart rhythm changes, measured by electrocardiogram changes.
Inhaled molgramostim was well-tolerated by the participants, was minimally absorbed into the blood stream, and had a clear effect on white blood cells counts within normal ranges. The most common adverse event reported was coughing.
Treatment with inhaled molgramostim may be a promising therapy for respiratory conditions with a high unmet medical need.
More studies were started in 2016 for other respiratory conditions, ARDS, and autoimmune pulmonary alveolar proteinosis.
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