According to a story from pm360online.com, the orphan lung disease company Savara recently announced that they had completed their enrollment target of 135 patients for a Phase 3 clinical trial that will test Molgradex, its most advanced drug candidate, as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP). Molgradex is an inhaled formulation of human granulocyte-macrophage colony-stimulating factor.
About Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Autoimmune pulmonary alveolar proteinosis (aPAP) is a disorder of the lung which is most characterized by the unusual buildup of lipoprotein compounds in a portion of the lung called the alveoli. These areas play an important role in getting oxygen into the blood stream. In aPAP, the cause is generally connected to some sort of malfunction of the alveolar macrophage; the body develops an autoimmune response to granulocyte-macrophage colony-stimulating factor, and as a result, alveolar macrophages do not develop properly. Symptoms of this disorder include shortness of breath, weight loss, fever, and coughing. The course of the disease varies greatly. Some patients see their disease go into remission spontaneously, while others have steady symptoms. Patients are more vulnerable to lung infections, and the progression of the disease is potentially lethal. It is usually treated with a process called whole lung lavage, in which the entire lung is filled with sterile fluid and withdrawn. To learn more about aPAP, click here.
About The Study
The company expects to have the results of this study available in mid 2019. Molgradex is also being tested in a long term extension study that is intended to monitor the effects of long term use of the medication for patients with aPAP. This study is expected to continue for three years after the original Phase 3 trial is completed. The effectiveness of the drug will be assessed based on improvements to the lung function of the patients. Secondary endpoints, which will help measure the extent of potential improvement, will include the time to need for whole lung lavage, St. George’s respiratory questionnaire, and the six minute walk distance test.
The current standard whole lung lavage procedure is good at providing symptom relief, but it is also a major inconvenience to the patient. It is a rather invasive process and it must be performed under general anesthesia. Hopefully Molgradex can be a more convenient alternative.