The Cystic Fibrosis Foundation has signed an agreement to support the development of Synspira Therapeutics Inc‘s SNSP003, an 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) and 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.

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, according to Synspira.

SNSP003 is a rationally designed treatment including a precise combination of three purified enzymes (lipase, protease and amylase) and 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, Synspira says.

“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, US sales of PERT therapies were over $1.3 billion.