Synspira Therapeutics, CF Foundation Agree to Support Development of SNSP003 for Malabsorption Syndromes

The Cystic Fibrosis Foundation has reached an agreement with Synspira Therapeutics to support the further development of SNSP003, an enzyme replacement therapy (ERT) designed to treat malabsorption syndromes, including those affecting patients with cystic fibrosis (CF).

Malabsorption syndromes are a group of disorders that develop when there is a defect in the digestion and absorption of macronutrients — such as fat, protein, and carbohydrates — due to diseases of the pancreas, liver, and gastrointestinal (GI) tract.

Malabsorption syndromes, such as exocrine pancreatic insufficiency (EPI),  develop early in patients with CF, and represent a significant unmet medical need in these patients.

Current standard care for malabsorption includes porcine-based pancreatic enzyme replacement therapies (PERT). It is estimated that approximately 90% of people with CF require treatment with PERT. However, this treatment method usually does not eliminate malabsorption completely, and can be associated with unwanted GI symptoms, and fatty acid abnormalities.

In addition, because PERT is produced from porcine (pig) pancreas, the response can vary greatly. Other PERT limitations are the high pill burden (15–40 capsules per day), and an inability to develop innovative formulations.

Synspira’s SNSP003 is an oral treatment that represents, according to the company, the first meaningful advance in ERT in the past 40 years.

SNSP003 includes a combination of three different purified enzymes: lipase (for digestion of fats); protease (for digestion of proteins); and amylase (for digestion of carbohydrates). So, it has the potential to separately address malabsorption of each macronutrient (fat, protein, and carbohydrates), which are vital to maintain proper nutrition.

SNSP003 can improve the patients’ clinical outcomes and decrease treatment burden, while improving the convenience of dosing by delivering a formula for pediatric and adult patients who cannot 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,” Robert Gallotto, CEO of Synspira, said in a press release.

“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,” Gallotto said.

Besides SNSP003, Synspira’s lead therapeutic candidate, SNSP113, is an inhaled therapy being developed to ease pulmonary infections, airway congestion, and inflammation in patients with CF. SNSP113 has successfully completed a Phase 1 study (NCT03309358), and will advance to Phase 2 clinical trials.