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October 18, 2017

Synspira Announces First Patient Dosed in Phase 1a Study of SNSP113 in Cystic Fibrosis

— First-in-class drug candidate for treatment of cystic fibrosis regardless of genetic mutation —

CAMBRIDGE, Mass. – October 18, 2017 – Synspira, a privately held company developing a new class of inhaled glycopolymer-based therapeutics for the treatment of pulmonary disease, today announced initiation of a Phase 1a first-in-human, multi-center study of SNSP113, its lead drug candidate, in development for use in pulmonary complications of cystic fibrosis (CF). SNSP113 has the potential to improve pulmonary function by reducing the viability of recalcitrant infectious bacteria, reducing inflammation, and facilitating clearance of associated biofilms and mucus.

“SNSP113 is the first clinical candidate from a new class of drugs that have the potential to transform the treatment of pulmonary diseases including cystic fibrosis, chronic obstructive pulmonary disease and pneumonia,” said Shenda Baker, Ph.D., Chief Scientific Officer of Synspira. “By simultaneously reducing infections, inflammation and the build-up of mucus and biofilms associated with cystic fibrosis, we believe SNSP113 could improve outcomes for patients. The initiation of our first clinical trial represents a major milestone for Synspira and a significant step forward toward improving the lives of CF patients.”

The Phase 1a first-in-human trial will assess the safety and tolerability of single ascending doses in healthy individuals and will advance into stable CF patients in 2018. ( ID#NCT03309358)

Pre-clinical studies suggest that SNSP113 has the potential to target significant infections in cystic fibrosis, including those driven by Burkholderia and non-tuberculous Mycobacterium for which there are currently no approved therapeutic treatments. Recurring pulmonary infection and inflammation result in permanent lung damage and can eventually lead to pulmonary failure.

“By targeting biofilms and mucus buildup in the lungs and allowing for the potentiation of antibiotics, SNSP113 has the potential to be effective across a wide range of pulmonary complications in CF patients,” said Dr. Steven M. Rowe, Director of the Gregory Fleming James Cystic Fibrosis Research Center at the University of Alabama, Birmingham. “If effective, this treatment could change the progression of this life-shortening disease and improve the lives of people living with cystic fibrosis.”

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 where there is a high unmet treatment need. Synspira is a privately held company headquartered in Framingham, MA. Our lead product, SNSP113, is a first-of-its-kind product 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 life-threatening genetic disorder that results in the accumulation of thick, sticky mucus in the lungs, clogging airways and leading to infection and chronic inflammation. Moreover, because of the inability to clear the airways, bacteria colonize and form biofilms that are difficult for antibiotics to penetrate. More than 30,000 people in the United States, and a similar number in Europe, live with cystic fibrosis.