October 11, 2018
Synspire Pharmaceuticals Receives U.S. FDA Orphan Designation for SNSP113 for the Treatment of Cystic Fibrosis
Cambridge, Mass. – October 11, 2018 – Synspire Pharmaceuticals, 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 SNSP113 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., Board Member of Synspire. “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
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 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 fibrosis.