The Phase 3 RESULT study is evaluating the non-inferiority of Sollpura compared to approved, porcine-derived, enteric-coated pancreatic enzyme replacement therapy (PERT) when administered at individualized doses to patients with exocrine pancreatic insufficiency due to cystic fibrosis. The study was initiated in Q2 2017 and will enroll patients (N≈150) who are well-controlled on a stable, porcine PERT at screening, as demonstrated by a coefficient of fat absorption (CFA) of at least 80%. The primary efficacy variable will evaluate the non-inferiority of Sollpura to Pancreaze (an approved porcine-extracted PERT) in the change from baseline CFA following treatment through 4 weeks of therapy.
Patients randomized to Sollpura will begin at 125% of their baseline porcine lipase dose and may undergo individualized dose adjustments based upon gastrointestinal signs and symptoms. Anthera believes that this optimized dosing paradigm will correct for differences in solubility between the lipases in Sollpura and porcine PERTs in the more acidic upper intestinal pH of many patients with cystic fibrosis. Patients randomized to Sollpura will be followed for an additional 20-week extension period (total of 24 weeks on study) for additional assessments of weight, height, BMI, and safety. Topline data are expected Q4 2017 / Q1 2018.