Synspira's Cystic Fibrosis Treatment Receives Orpan Drug Designation

Synspira, a privately held company developing a new class of inhaled glycopolymer-based therapeutics for the treatment of pulmonary disease, today announced that it has been granted Orphan Designation by the United States Food and Drug Administration (FDA) for poly (acetyl, arginyl) glucosamine (PAAG15A), for the treatment of cystic fibrosis. Synspira is developing PAAG15A as SNSP113, a potential inhaled treatment to improve lung function in patients with cystic fibrosis (CF).

“The orphan designation granted for PAAG15A by the FDA reflects the need for new treatment options for patients living with cystic fibrosis, and in particular for those patients with relentless and antibiotic resistant bacterial infections such as Burkholderia and non-tuberculous Mycobacteria,” said Shenda Baker, Ph.D., Chief Executive Officer of Synspira. “We are developing SNSP113 to treat pulmonary infection and airway congestion in cystic fibrosis patients to improve pulmonary health. Because SNSP113 treats the drivers of pulmonary decline rather than the underlying CFTR mutation, it has the potential to treat a broad population of CF patients regardless of genetic mutation.”

SNSP113 is a glycopolymer-based therapeutic being developed as an inhaled treatment to improve lung function in patients with cystic fibrosis. PAAG15A is the active modified polysaccharide in SNSP113 that interacts with structural polymers in protective bacterial biofilms, breaking them apart, and with native glycoproteins in mucus, normalizing mucus viscosity. SNSP113 also interacts with the cell walls of invading bacteria increasing their permeability, thereby reducing their inherent viability and potentiating the efficacy of antibiotics. SNSP113 is designed to reduce infection, airway congestion and inflammation, the key drivers of pulmonary exacerbations and pulmonary decline in cystic fibrosis patients.

SNSP113 is the lead candidate being developed by Synspira. The company is also developing three additional glycopolymer-based therapeutics.