Synspira Therapeutics and Cystic Fibrosis Foundation Sign Agreement to Develop Broad-spectrum Enzyme Replacement for Malabsorption Syndromes
13 February 2020
Synspira Therapeutics Inc., a clinical-stage biopharmaceutical company dedicated to improving the lives of people with cystic fibrosis (CF) and other rare diseases, announced today the company has signed an agreement with the Cystic Fibrosis Foundation to support the development of SNSP003, Synspira's orally delivered non-porcine enzyme replacement therapy (ERT) designed to treat Malabsorption Syndromes. Malabsorption Syndromes are a group of life-threatening disorders that result from defects in the digestion and absorption of macronutrients (fat, protein, carbohydrate).
SNSP003 could represent the first significant enzyme replacement therapy advancement in 40 years with the potential to be the only broad-spectrum non-porcine product that addresses malabsorption of fat, protein and carbohydrates – each macronutrient is critical to maintaining proper nutritional status and eliminating negative clinical outcomes. SNSP003 is a rationally designed treatment including a precise combination of three purified enzymes (lipase, protease and amylase). SNSP003 has the potential to improve clinical outcomes and also to dramatically reduce treatment burden by improving dosing convenience and providing a formulation for pediatric and adult patients unable to swallow capsules.
"For more than 20 years, our team has collaborated with the CF Foundation on novel therapies to improve the lives of people with CF. We are focused on a rational approach to designing and rapidly advancing effective treatment options that improve outcomes and reduce patient burden for people with CF and others with Malabsorption Syndromes," said Robert Gallotto, Chief Executive Officer of Synspira. "Synspira brings an unmatched expertise to the development and delivery of oral enzyme therapies for the treatment of Malabsorption Syndromes. Now, with the CF Foundation's support, we can quickly advance SNSP003, and address an urgent unmet need."
Synspira's oral enzyme delivery technology is expected to provide a significant advancement over current porcine-based pancreatic enzyme replacement therapies (PERT). PERT has inherent limitations due to the porcine pancreas source which leads to variable response, a high pill burden (15-40 capsules per day) and an inability to provide innovative formulations. Approximately 90% of people with CF require PERT, yet current treatments rarely eliminate malabsorption, while undesirable GI symptoms, fatty acid abnormalities and malnutrition continue. In 2018, U.S. sales of PERT therapies were over $1.3 billion.
Malabsorption Syndromes and Exocrine Pancreatic Insufficiency
Malabsorption Syndromes are a group of life-threatening disorders that result from a defect in one or more of the steps in the digestion and subsequent absorption of macronutrients (fat, protein, carbohydrate). Malabsorption Syndromes are associated with diseases of the pancreas, liver and gastrointestinal tract. Despite advances in the treatment of CF, Malabsorption Syndromes, including exocrine pancreatic insufficiency (EPI), continue to present a substantial burden to the patient community. Cystic fibrosis is a life-threatening disease that affects approximately 35,000 children and adults in the United States and nearly 100,000 people worldwide. CF is a genetic disease caused by an abnormal gene that affects the lungs, pancreas and other organs. Malabsorption develops early in the course of CF and remains a significant unmet need.
About Synspira Therapeutics
Synspira Therapeutics Inc. is a clinical-stage biopharmaceutical company dedicated to significantly improving the lives of people with cystic fibrosis (CF) and other rare diseases. Inspired by patients, Synspira is focused on developing a rationally designed portfolio of products that can make a life-changing impact and improve the quality of life of our patients. Synspira is working to advance two novel products (SNSP113 and SNSP003) intended to provide better clinical outcomes and reduce treatment burden in Progressive Pulmonary Disease (PPD) and Malabsorption Syndromes. SNSP113, Synspira's lead product, is entering Phase 2 clinical studies in people with cystic fibrosis and PPD. Synspira is a privately held company headquartered in Framingham, MA.