News &

November 02, 2017

Synspira Announces Data for Lead Candidate at the 31st Annual North American Cystic Fibrosis Conference

CAMBRIDGE, Mass. – November 2, 2017 – Synspira, a privately held company developing a new class of inhaled glycopolymer-based therapeutics for the treatment of pulmonary disease, today announced the presentation of the results of two studies of SNSP113, its lead drug candidate for use in pulmonary complications of cystic fibrosis (CF). The studies were led by the laboratory of Steven M. Rowe M.D. at the University of Alabama at Birmingham and will be presented at the 31st Annual North American Cystic Fibrosis Conference (NACFC), being held from November 2-4, 2017 in Indianapolis, IN.

In CF patients, mucus is overproduced, improperly formed and highly viscous, causing thickened airways and compromising pulmonary clearance. Results from one study presented today suggest that SNSP113 has the potential to enhance pulmonary clearance by improving the viscosity and transport of mucins through structure alteration.

Studies showed that SNSP113 (active pharmaceutical ingredient is PAAG) improved viscosity and transport of CF sputum on excised rat trachea and altered mucin conformation to resemble a more normal, linear form. In addition, pretreatments of SNSP113 prevented Pseudomonas aeruginosa infection in 70% of wild type rats, whereas all animals treated with control were infected. Enhanced pulmonary clearance is an important aspect of removing bacteria to prevent colonization and infection in the lung.

“People living with cystic fibrosis are challenged by abnormal mucus that precipitates chronic infection and dysregulated inflammation,” said Dr. Steven M. Rowe, Director of the Gregory Fleming James Cystic Fibrosis Research Center at the University of Alabama, Birmingham. “The effects on both mucus and Pseudomonas bacterial infection demonstrated by SNSP113 are very promising for cystic fibrosis patients, no matter their underlying CFTR mutation.”

According to the Cystic Fibrosis Foundation (, there are more than 1,700 mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene; some are common and others are rare and found only in a few people. Certain types of CF mutations are associated with less severe symptoms of the disease but remain chronic and progressive.

The first study results will be presented at Workshop #8 and Formal Poster Session I: New Therapies Poster Discussion Session/Workshop.

Presenter: Courtney M. Fernandez
Title: PAAG Alters the Viscoelasticity and Transport of CF Mucus
Date: Thursday, November 2, 2017
Time: 9:45 – 11:05 AM EST and 11:15-1:45 PM EST
Location: Room 143, Indiana Convention Center and The Exchange – Hall I, Indiana Convention Center

A second study demonstrates that SNSP113 could potentially reduce established Pseudomonas infections in CF patients. In studies both as a single agent and in combination with tobramycin, an inhaled antibiotic commonly used in the treatment of lung infections, SNSP113 is shown to reduce the thickness of biofilms and prevent biofilm persistence when grown on CF epithelial cells. These studies demonstrated that PAAG disrupted Pseudomonas aeruginosa biofilms grown in co-culture on CF and wild type respiratory epithelium. Co-treatment with PAAG and tobramycin at the time of inoculation on CF respiratory epithelium augmented tobramycin efficacy against both a laboratory strain, PAO1, and a highly mucoid clinical strain, Pa529.

“The results presented today further elucidate how SNSP113 can improve the treatment of cystic fibrosis by potentially reducing chronic infections that impair lung function while also improving the clearance of mucus and bacteria,” said Shenda Baker, Ph.D., Chief Scientific Officer of Synspira. “We are continuing to evaluate in vivo capabilities of SNSP113 and its safety in a Phase 1a clinical trial as part of our clinical program to advance the treatment of cystic fibrosis patients, regardless of genetic mutation.”

The second study results will be presented at a Formal Poster Session.

Lead author: Bryan Garcia, M.D.
Title: Anti- Pseudomonas Aeruginosa Biofilm Activity of the Novel Glycopolymer PAAG on Infected Cystic Fibrosis Respiratory Epithelium
Date: Thursday, November 2, 2017
Time: 11:15-1:45 PM EST
Location: The Exchange – Hall I, Indiana Convention Center

About SNSP113

The company’s lead product, SNSP113, is a first-in-class inhaled glycochemistry-based therapeutic with a broad spectrum mechanism of action intended to target the underlying cascade of events that lead to progressive pulmonary disease or other life-threatening pulmonary conditions, such as nontuberculous Mycobacteria(NTM), Burkholderia cepacia complex (BCC), Pseudomonas aeruginosaor methicillin-resistant Staphylococcus aureus(MRSA). SNSP113 is designed to normalize mucin viscosity and improve mucus transport to increase airway clearance. SNSP113 disrupts the cohesion of bacterial biofilms and interacts with the cell walls of invading bacteria to increase their permeability, reduce their viability and potentiate the efficacy of antibiotics. These actions of SNSP113 lead to a reduction in the inflammatory cascade of neutrophils that can lead to pulmonary damage and fibrosis. Progressive pulmonary disease leads to overwhelming symptoms, impacts quality of life (QoL) and results in debilitating progressive lung decline. Synspira is expected to initiate SNSP113 Phase 2 trials in 2019.

About Synspira

Synspira Therapeutics is a clinical-stage biopharmaceutical company dedicated to significantly improving the lives of people with cystic fibrosis and other rare diseases, such as primary ciliary dyskinesia and bronchiectasis, where there is a high unmet treatment need. Synspira is a privately held company headquartered in Framingham, MA. The company’s lead product, SNSP113, is a first-in-class inhaled glycochemistry-based therapeutic with a broad spectrum mechanism of action designed to target the underlying cascade of events that lead to progressive pulmonary disease or other life-threatening pulmonary conditions. At Synspira we are inspired by the patients we serve and are driven to make a difference.

About Cystic Fibrosis

Cystic fibrosis (CF) is a progressive genetic disease that causes 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 fibrosis.