Liprotamase (Sollpura) for Nutritional-GI in Cystic Fibrosis Patients

Liprotamase (sold under Anthera’s brand name Sollpura) is a soluble, stable, and experimental pancreatic enzyme replacement therapy (PERT) that does not come from animal sources. It is for the treatment of exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF), especially in young children and adults who are unable to swallow multiple pills or who use gastric tubes.

Sollpura consists of three soluble, nonporcine digestive enzymes: lipase, protease, and amylase, combined in a fixed ratio.

How Sollpura works

The enzymes in Sollpura digest food that the body was unable to digest within the gastrointestinal tract. This facilitates the absorption of nutrients into the bloodstream and helps maintain nutrition, with larger molecules cut into smaller ones, such as triglycerides into free fatty acids, proteins into small peptides and amino acids, and carbohydrates into simple sugars. Sollpura enzymes are not absorbed into the bloodstream.

Sollpura is a biotechnology product which eliminates the need for the use of porcine-derived enzymes. Its enzymes are very stable and do not need to be coated, which allows a reduction in the quantity of pills that need to be taken every day.

Sollpura studies

The Phase 3 SIMPLICITY clinical trial (NCT02734810) is evaluating the safety and effectiveness of a new, soluble formulation of Sollpura (liprotamase powder for oral solution), in cystic fibrosis patients with EPI. The open-label study is currently recruiting participants in 17 different locations across the U.S.

Primary outcomes of the study include the number of participants with adverse events in one week and the coefficient of fat absorption over seven weeks.

The study is being conducted in two parts based on age. Part A will evaluate the safety of Sollpura powder for oral solution in patients ages 7 and older with CF-related EPI.

Part B will evaluate the safety and effectiveness in pediatric patients ages 28 days up to 7 years, also with CF-related EPI, based on observed coefficient of fat absorption measured after seven weeks of treatment.

An ongoing Phase 3, randomized, open-label study called SOLUTION (NCT02279498) is evaluating the safety and effectiveness of Sollpura in participants with EPI due to CF. The study, whic was initiated in 2015, is a non-inferiority test of Sollpura compared to PERT in patients with EPI due to CF.

The primary endpoint is the change from baseline in coefficient of fat absorption (CFA) following seven weeks of treatment with either Sollpura or Pancreaze, an alternative porcine PERT to that being taken during screening.

After the seven-week period and CFA measurements, the participants enroll in the SOLUTION extension period (from week 7 to week 20). This extension period, known as the EASY trial (NCT02823964), was designed as an observational analysis of the long-term effects of Sollpura and Pancreaze in key measurements of height, weight, and body mass index (BMI).

At the end of March 2017, Anthera announced positive results from the EASY extension period of the SOLUTION study. Sollpura was well tolerated and the key measurements of height, weight, and BMI were maintained. According to Anthera, data from this extension period will serve as the basis for an FDA new drug application (NDA) for Sollpura. 

Future studies for Sollpura

Anthera initiated another Phase 3 study, named RESULT (NCT03051490). The study, which is not yet recruiting participants, aims to evaluate the non-inferiority of Sollpura at individualized doses compared to approved PERT. The study expects to enroll 150 patients with EPI due to CF.

The primary endpoint of this study is the change from baseline in CFA after four weeks of treatment with either Sollpura or Pancreaze. Participants randomized in the Sollpura group will start at 125% of the baseline PERT dose and may see their individualized doses corrected for differences in solubility between the lipases in Sollpura and porcine PERTs.

The Sollpura group will be enrolled in a 20-week extension period for measurements of weight, height, BMI and to evaluate the long-term safety of the drug. Data from this study is expected at the end of 2017 or in early 2018. 

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