SNSP113 was designated an orphan drug as a potential treatment of pulmonary decline in cystic fibrosis (CF) by the U.S. Food and Drug Administration (FDA) in October 2018, and given orphan medicinal product status by the European Medicines Agency in April 2019. Both designations give incentives to support development and testing of therapies for rare diseases.
How SNSP113 works
SNSP113 contains an active ingredient called poly N (acetyl, arginyl) glucosamine, which is a type of complex sugar molecule. The treatment is reported to have a multi-faceted mechanism of action that might protect pulmonary function in CF patients:
- Targeting and breaking apart bacterial biofilms. These are communities of bacteria that are attached to the airway epithelium and are usually resistant to antibiotics. By breaking the biofilms, SNSP113 could reduce antibiotic resistance to common disease pathogens, such as Burkholderia cepacia complex, Pseudomonas aeruginosa, and methicillin-resistant Staphylococcus aureus (MRSA).
- Normalizing the mucus viscosity of CF patients, and to improve mucus transport and airway clearance by interacting with the mucin polymers, which are the major components of mucus.
- Interacting with and disrupting the cell walls of the invading bacteria, to increase their permeability and allow greater uptake of antibiotics given to kill bacteria.
- Reducing the inflammatory cascade of neutrophils, immune cells whose activity causes pulmonary tissue damage and fibrosis.
Synspira reports that lab data suggests SNSP113 has strong anti-inflammatory effects at low doses.
SNSP113 in clinical trials
A single-ascending dose Phase 1 study (NCT03309358) tested the safety and tolerability at day eight of treatment with inhaled SNSP113 in 32 healthy people. This trial initially was a two-part study in healthy people and CF patients, but was stopped after what is reported to be the “successful completion” of its first part.
Synspira has announced that it plans a Phase 2 clinical trial of inhaled SNSP113 in people with CF in 2019.
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