Molgradex is an investigational inhaled formulation of recombinant human GM-CSF. It is being evaluated for the treatment of autoimmune pulmonary alveolar proteinosis, or aPAP, 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 GM-CSF, a naturally occurring protein, suppressing the stimulating activity of GM-CSF on lung macrophages which function to clear excess surfactant from the alveoli.
Molgradex has been granted Orphan Drug Designation for the treatment of aPAP in the U.S. and the European Union. Orphan Drug Designation makes Molgradex eligible for seven years of exclusivity from approval in the U.S., and ten years of exclusivity in the European Union.
Additionally, the U.S. Food & Drug Administration (FDA) granted Molgradex Breakthrough Therapy Designation for the treatment of aPAP. This process is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary evidence indicates the drug may demonstrate a substantial improvement over available therapies.
Molgradex is an investigational inhaled formulation of recombinant human GM-CSF. It is being evaluated for the treatment of nontuberculous mycobacterial (NTM) lung infection in people living with cystic fibrosis (CF) and NTM infection in people who do not have CF. NTM is estimated to affect approximately 50,000 – 80,000 individuals in the U.S, the most common types involving Mycobacterium avium complex (MAC) and Mycobacterium abscessus. NTM lung infections are a considerable therapeutic challenge due to the unique ability of the bacteria to evade the normal killing mechanisms of alveolar macrophages (a type of immune cells responsible for killing bacteria in the lungs). 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.
Apulmiq is an investigational inhaled ciprofloxacin. It is being evaluated for the treatment of non-cystic fibrosis bronchiectasis (NCFB), a chronic lung disease characterized by abnormal widening of the airways associated with thickened and scarred walls due to infection and inflammation. Symptoms include persistent and productive cough, dyspnea, fatigue, and recurrent infective pulmonary exacerbations that often lead to hospitalization and are associated with higher mortality. With an estimated prevalence of more than 150,000 patients in the U.S. and no approved pharmaceutical therapies available, there is a high unmet medical need.
In the U.S., Apulmiq has been granted Orphan Drug Designation as well as designated a Qualified Infectious Disease Product (QIDP), making it eligible for up to 12 years of exclusivity.
AeroVanc is an investigational inhaled formulation of vancomycin. It is being evaluated for the treatment of persistent methicillin-resistant Staphylococcus aureus
, or MRSA, lung infection in people living with cystic fibrosis (CF). CF is a genetic disease that involves sticky mucus buildup in the lungs, persistent lung infections, and permanent and progressive respiratory disability. There are approximately 30,000 patients affected by CF in the U.S., and MRSA infection has become increasingly common in these patients—affecting ~26% of people living with CF. Persistent MRSA infection in CF is associated with increased use of intravenous (IV) antibiotics, increased hospitalizations, faster decline of lung function, as well as shortened life-expectancy. Due to the lung pathology associated with CF, persistent MRSA lung infection is difficult to eradicate or manage using oral or IV antibiotics. Currently, there is no standard of care to manage this condition and no approved inhaled antibiotics addressing MRSA lung infection.
In the U.S., AeroVanc has been granted Orphan Drug Designation and designated a Qualified Infectious Disease Product (QIDP) for the treatment of persistent MRSA lung infection in people living with CF. The Orphan Drug Designation makes AeroVanc eligible for seven years of exclusivity from approval and ten years of exclusivity in the European Union. QIDP designation makes AeroVanc eligible for an additional five years of exclusivity in the U.S.
Savara Nasdaq Opening Bell Ceremony - July 23, 2018×