Summary

Indications

Savara Inc. is a clinical-stage specialty pharmaceutical company focused on the development and commercialization of novel therapies for the treatment of serious or life-threatening rare respiratory diseases. Savara’s strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, in identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.

Autoimmune Pulmonary Alveolar Proteinosis (aPAP) is a rare lung disease, which affects approximately 7 people per million. It is characterizsed by the build-up of grainy material in the air sacs (alveoli) in the lungs. The grainy material consists of proteins and lipids from lung surfactant – an important substance that coats the inside of the air sacs to prevent the lungs from collapsing. The air sacs need to be inflated for the lungs to absorb the oxygen that the patient breathes and transfer it to the blood circulation. The body continuously produces new active surfactant. In healthy lungs the old, inactivated surfactant is digested by immune cells called alveolar macrophages. They are the dustmen or garbage collectors of the body and they have a very important task of keeping the alveoli clean. In aPAP lungs, however, the macrophages fail to clean the air sacs. Consequently, the old surfactant material builds up gradually in the lungs and eventually fills the alveoli causing the patient to feel breathless. Scientific research shows that the macrophages need to be stimulated by the GM-CSF protein in order to function. In a diseased lung, however, the GM-CSF protein is either inactivated or defective, rendering the macrophages or dustmen unable to perform their cleaning tasks. The patient is commonly a man in early middle age who experiences increasing breathing difficulty, first with exertion, later at rest while developing a cough. There may be episodes of fever, chest pain, or coughing blood, especially if secondary lung infection develops. The best available treatment today is periodic whole lung lavage (WLL), i.e. washing out the lungs under general anesthesia. This requires admission to intensive care, which is an invasive and inconvenient procedure that can only be conducted effectively and safely by highly experienced physicians at a few specialist sites.
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Frontal chest radiograph in patient with cystic fibrosis

There are approximately 30,000 people living with cystic fibrosis in the U.S. Cystic fibrosis is a life-shortening genetic disease characterized by thick, sticky mucus in the lungs and chronic lung infections resulting in gradual loss of lung function. The most prevalent lung pathogen in cystic fibrosis patients is Pseudomonas aeruginosa, which is commonly treated using inhaled antibiotics. In recent years, methicillin-resistant Staphylococcus aureus (MRSA), a bacterium that is resistant to conventional antibiotics (1), has become increasingly common, with a prevalence of almost 30% of the U.S. cystic fibrosis patient population. Recent publications indicate that cystic fibrosis patients with chronic MRSA infection have more hospitalizations, faster decline in lung function, and reduced life expectancy. (2,3,4) [1] Gorwitz RJ et al. Journal of Infectious Diseases. 2008:197:1226-34. [2] Prevalence and impact on FEV1 decline of chronic methicillin-resistant Staphylococcus aureus (MRSA) coloniation in patients with Cystic Fibrosis: A single-center case control study of 165 patients. Vanderhelst E, De Meirleir L, Verbanck S. et al. s.l. : J Cystic Fibrosis, 2012, Vol. 11, pp. 2-7. [3] Persistent Methicillin-resistant Staphylococcus aureus and Rate of FEV1 Decline in Cystic Fibrosis. Dasenbrook EC, Merlo CA, Diener-West M, et. al. s.l. : Am J Respir Crit Care Med, 2008, Vol. 178, pp. 814-821. [4] Elliott C. Dasenbrook; William Checkley; Christian A. Merlo; Michael W. Konstan; Noah Lechtzin; Michael P. Boyle. Association Between Respiratory Tract Methicillin-Resistant Staphylococcus aureus and Survival in Cystic Fibrosis. JAMA, 2010; 303 (23): 2386-2392.

Non-CF bronchiectasis is a chronic lung disease characterized by abnormal widening of the airways associated with chronic infection. Patients with bronchiectasis typically experience a chronic productive cough and lung infection, malaise and increased hospitalizations due to pulmonary exacerbations, all of which negatively impact quality of life. The underlying pathology in bronchiectasis is a mix of infection, reduced immune response and inflammatory damage of the lungs. Amikacin is a potent antibiotic against common pathogens in bronchiectasis and fosfomycin is an antibiotic that improves the killing activity of amikacin against a number of Gram-negative bacteria, and also has activity against Gram-positive bacteria common in bronchiectasis, including MRSA. Currently, there are no therapies specifically approved for bronchiectasis.
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Savara Nasdaq Opening Bell Ceremony - July 23, 2018
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