Savara at a Glance
Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP, in Phase 2a development for nontuberculous mycobacteria, or NTM, lung infection, and in preparation for Phase 2a development in cystic fibrosis, or CF, affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in CF. 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. The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2 ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.
- Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for pulmonary alveolar proteinosis (PAP); and,
- Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in preparation for Phase 2a development for nontuberculous mycobacterial (NTM) lung infection.
- AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of MRSA infection in Cystic Fibrosis.
Molgradex is currently in Phase 3 development for PAP and in preparation for Phase 2a development for NTM lung infection.
PAP is a rare lung disease characterized by the build-up of lung surfactant in the alveoli, or air sacs, of the lungs. The disease process underlying PAP involves an autoimmune response against a naturally occurring protein, GM-CSF, suppressing the stimulating activity of GM-CSF on lung macrophages which function to clear excess surfactant from the alveoli. Based on published investigator-sponsored treatment experience with inhaled GM-CSF, Savara believes Molgradex has the potential to replace the inactivated GM-CSF in PAP patients, and thereby to restore the surfactant clearing activity of the alveolar macrophages.
NTM lung infection is a rare and serious lung disorder associated with increased rates of morbidity and mortality. Nontuberculous mycobacteria are naturally-occurring organisms and NTM lung infection can occur when an individual inhales the organism from their environment and develop a slowly progressive and destructive lung disease. NTM infections often become chronic and require long courses of multiple antibiotics, and despite the aggressive treatment regimens, treatment failure rates are high, and recurrence of infection common. NTM lung infections are a considerable therapeutic challenge due to the unique ability of these bacteria to evade the normal killing mechanisms of alveolar macrophages, a type of immune cells responsible for killing bacteria in the lungs. There is increasing scientific literature suggesting that GM-CSF plays an important role in enhancing the ability of macrophages to clear mycobacteria. Savara believes that Molgradex has the potential to help eradicate NTM lung infection, with or without the concomitant use of antibiotics by stimulation of the innate immune system in the lungs.
AeroVanc (vancomycin hydrochloride inhalation powder), is an inhaled dry powder form of vancomycin to treat Methicillin-resistant Staphylococcus aureus (MRSA) lung infection in cystic fibrosis patients. By delivering vancomycin directly to the site of infection in the lungs, the hope is that AeroVanc will improve clinical efficacy and reduce adverse effects due to lessor systemic drug exposure as compared to IV antibiotic treatment. AeroVanc is the first inhaled antibiotic for management of MRSA lung infection in CF patients.