Savara at a Glance
Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, AeroVanc and amikacin/fosfomycin.
Molgradex is an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP, and in Phase 2a development for nontuberculous mycobacteria, or NTM, in both non-cystic fibrosis (CF) and CF-affected individuals with chronic NTM lung infection.
AeroVanc, is a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin-resistant Sstaphylococcus aureus, or MRSA, lung infection in CF.
Amikacin/fosfomycin is a Phase 2, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens.
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, 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 aPAP, and Phase 2a development for NTM in both non-CF and CF-affected individuals with chronic NTM lung infection.
- AeroVanc, an inhaled vancomycin in Phase 3 development for MRSA infection in CF.
- amikacin/fosfomycin, an aerosolized combination antibiotic in Phase 2 development for non-CF bronchiectasis
Autoimmune 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 aPAP 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. The disease has a meaningful impact on a person’s quality of life. Symptoms can include breathlessness, significantly reduced exercise capacity, frequent fatigue and, in severe cases, infection, progressive respiratory failure or even pulmonary fibrosis requiring a lung transplant. Currently, there are no approved pharmacological treatment options for aPAP, with whole lung lavage (WLL) being the current standard of care. A WLL is an invasive surgical procedure performed under anesthesia to physically remove surfactant from a patient’s lungs.
Based on published investigator-sponsored treatment experience with inhaled GM-CSF, Savara believes Molgradex has the potential to replace the inactivated GM-CSF in aPAP patients, and thereby to restoring 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 develops 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. Unlike antibiotics that target bacteria directly, Molgradex attempts to boost the body’s natural defenses, essentially helping the immune system clear the infection. Savara believes that Molgradex has the potential to help eradicate NTM lung infection in both non-CF and CF-affected individuals, with or without the concomitant use of antibiotics.
About MRSA in CF
CF is a life-shortening genetic disease characterized by thick, sticky mucus in the lungs and frequent lung infections, which result in loss of lung function. As the disease progresses, the lungs of people with CF are typically infected with bacteria that are difficult to eradicate. Physicians have been using nebulized antibiotics to treat these infections and have sought new options for treatment in the chronic care setting. Persistent infection by MRSA has become increasingly common in the U.S. and is associated with faster decline in lung function and reduced survival. Currently there are no approved inhaled therapies for MRSA infection in people living with CF. AeroVanc (vancomycin hydrochloride inhalation powder), is an inhaled dry powder form of vancomycin to treat persistent (MRSA) lung infection in people living with CF. By delivering AeroVanc directly to the site of infection in the lungs, Savara believes that it may will improve clinical efficacy and reduce adverse effects due to lessor systemic drug exposure as compared to IV antibiotic treatment. AeroVanc would be is the first inhaled antibiotic for management of MRSA lung infection in CF.
About Non-CF Bronchiectasis
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. Research has shown that patients with bronchiectasis often have defective function of neutrophils, the most abundant type of immune cells fighting infection and neutrophil dysfunction can be reversed by treatment with GM-CSF. Savara is investigating the efficacy of amikacin/fosfomycin and Molgradex separately, and in combination, in reducing the bacterial infection load in non-CF bronchiectasis patients experiencing recurrent exacerbations. Currently, the unmet medical need is high as there are no therapies specifically approved for bronchiectasis.