FDA Grants Orphan Drug Designation to Synspira’s Investigational Cystic Fibrosis Therapy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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 the lead candidate being developed by Synspira as part of a new class of proprietary glycopolymer-based therapeutics. SNSP113 is designed to specifically target and disrupt the cohesion of bacterial biofilms, potentiating the activity of antibiotics, and normalizing thick mucus. SNSP113 is intended to improve lung function in CF patients by targeting the key drivers of pulmonary decline – infection, airway congestion and inflammation.

The Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

About SNSP113
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.

About Cystic Fibrosis
Cystic fibrosis (CF) is a progressive genetic disease that causes airway obstruction, persistent lung infections and chronic inflammation of pulmonary tissue leading to permanent lung damage and ultimately resulting in respiratory failure. CF is characterized by the accumulation of thick, sticky mucus in the lungs and clogged airways which impede breathing. Bacteria are not easily cleared and create protective biofilms that are difficult for antibiotics to penetrate and often lead to the emergence of multi-drug resistant bacteria. More than 30,000 people in the United States, and a similar number in Europe, live with cystic fibrosis1.

About Synspira
Synspira is developing a new class of inhaled glycopolymer-based therapeutics to reduce pulmonary inflammation, airway obstruction and infection, key drivers of pulmonary diseases including cystic fibrosis (CF), chronic obstructive pulmonary disease (COPD), and pneumonia. Synspira has an exclusive license from Synedgen to the Glycomics Technology Platform for the development of inhaled therapeutics in pulmonary indications. Synspira is dedicated to developing drugs with new mechanisms of action to target and change the course of pulmonary diseases.

1 Cystic Fibrosis Foundation. About Cystic Fibrosis. https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/.

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