Summary of Savara’s U.S. Expanded Access Policy
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 for aPAP
Molgradex is the first inhaled GM-CSF being developed for the treatment of a rare respiratory disease called autoimmune pulmonary alveolar proteinosis (aPAP). A pivotal Phase 3 clinical study, IMPALA, is currently underway.
Molgradex for NTM
Molgradex is being evaluated in a Phase 2a clinical study, OPTIMA, for the treatment of patients with nontuberculous mycobacterial (NTM) lung infection, a rare and serious lung disorder associated with increased rates of morbidity and mortality.
Molgradex for NTM in CF
Molgradex is being evaluated in a Phase 2a clinical study, ENCORE, for the treatment of people living with CF who have nontuberculous mycobacterial (NTM) lung infection, a rare and serious lung disorder associated with increased rates of morbidity and mortality.
AeroVanc for MRSA in CF
AeroVanc is the first inhaled antibiotic being developed to address the growing problem of chronic methicillin-resistant Staphylococcus aureus (MRSA) lung infection in people with CF. A pivotal Phase clinical study, AVAIL, is currently underway.
Amikacin/fosfomycin for non-CF bronchiectasis
Savara is preparing to initiate a Phase 2 clinical study in the U.S. in non-CF bronchiectasis patients experiencing recurrent exacerbations. The study is expected to begin in late 2019 or early 2020.
Savara Nasdaq Opening Bell Ceremony - July 23, 2018×