Synspira and CF Foundation Collaborate to Develop Enzyme Replacement for Malabsorption Syndromes

Rare Daily Staff

Synspira Therapeutics said the Cystic Fibrosis Foundation has agreed to support the development of SNSP003, Synspira’s experimental therapy 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 such as fat, protein, and carbohydrates. They are associated with diseases of the pancreas, liver, and gastrointestinal tract.

Despite advances in the treatment of cystic fibrosis (CF), malabsorption syndromes, including exocrine pancreatic insufficiency, continue to present a substantial burden to the patient community. CF is a rare life-threatening 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.

SNSP003 is an orally-delivered, non-porcine enzyme replacement could represent the first significant enzyme replacement therapy advancement in 40 years. It has 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 precise combination of three purified enzymes (lipase, protease, and amylase) with potential to improve clinical outcomes and 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, CEO of Synspira.

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 percent of people with CF require PERT, according to Synspira, yet current treatments rarely eliminate malabsorption, while undesirable GI symptoms, fatty acid abnormalities and malnutrition continue. In 2018, more than $1.3 billion of PERT therapies were sold in the United States.