Cystic Fibrosis Pulmonary Infection
Cystic Fibrosis (CF) lung disease, which is estimated to affect more than 30,000 in the US and 70,000 worldwide1, is characterized by chronic bacterial infection and severe inflammation that leads to progressive deterioration in lung function. Chronic pulmonary infections in CF patients, a significant portion which involves Pseudomonas aeruginosa, are characterized by persistence, biofilm formation, evasion of the host’s immune response, and resistance to multiple therapeutic agents. Multispecies biofilm formed by combinations of CF-relevant bacteria have demonstrated greater resistance, virulence and pathogenicity than comparable single-species biofilms. The presence of such complex biofilms in the lungs of CF patients are considered to be largely responsible for the chronic, persistent nature of these pulmonary infections, which are not only responsible for chronic, ongoing and progressive morbidity, but are also ultimately responsible for mortality in this population. Chronic, CF-related pulmonary infections represent a major health care burden, being responsible for: significant loss of productivity; morbidity; reduced quality of life; and premature mortality frequently related to pulmonary infections.
Microbion’s compounds with their dual antimicrobial and anti-biofilm properties, represent a unique approach to treating resistant, chronic CF infections. Pre-clinical studies with an inhalation formulation of pravibismane, that will enable clinical studies, are ongoing. Pravibismane has the potential to address critical areas of immediate and advancing unmet medical needs with CF patients. Orphan drug designation was granted by the US FDA for inhaled pravibismane for the treatment (management) of pulmonary infections in patients with cystic fibrosis.
In addition to CF-related lung infection, there are multiple lower respiratory tract infections and conditions where the presence of microbial pathogens and their associated biofilm contribute to lung function decline. Infectious pathogens such as Pseudomonas spp., Staphylococcus spp., Streptococcus spp., Mycobacterium spp., or Haemophilus spp. may contribute to lung diseases such as non-CF bronchiectasis, bronchitis, community- or ventilator-associated pneumonia, non-tuberculous mycobacterium infection, etc., that are expected to benefit from an inhaled application of pravibismane or other Microbion compounds.
Microbion was awarded up to US$17 million from CARB-X (Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator) and the Cystic Fibrosis Foundation to advance the development of inhaled pravibismane through completion of pre-clinical and Phase 1 studies, including in patients with CF-related pulmonary infection.
Cystic Fibrosis Foundation. Cystic Fibrosis Foundation by the Numbers. July 1, 2017.