A Phase 3 study of Sollpura (liprotamase), a non-porcine enzyme replacement therapy aiming to treat exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF) better than existing porcine-derived therapies, is now recruiting about 150 pediatric and adult patients in the United States, Europe and Israel.
Specifically, the expanded trial will evaluate the efficacy — or non-inferiority — of Sollpura compared to pig-derived and enteric-coated pancreatic enzyme replacement therapies (PERTs) approved for CF patients with EPI, a condition caused by low enzyme levels in the digestive tract. Topline results are expected toward the end of 2017 or in early 2018.
As a PERT, Sollpura is being developed by Anthera to overcome nutrient malabsorption in CF patients due to chronic pancreatic inflammation and the thick mucus that blocks the discharge of pancreatic enzymes needed for digestion. It contains the enzymes lipase, protease and amylase in a specific ratio designed to promote a healthy digestion of food. Because it is of non-porcine origin, Sollpura’s therapeutic value should come without the risks associated with similar porcine-based drugs.
“We are very much looking forward to supporting Anthera in their efforts to bring this novel, biotech-derived pancreatic enzyme replacement therapy to patients,” Fernando Casals Seoane, MD, PhD, gastroenterology and hepatology specialist at Hospital Universitario de La Princesa, Madrid, said in a company press release.
In a previous Phase 3 clinical study, called SOLUTION (NCT02279498), researchers demonstrated the non-inferiority of Sollpura compared to Pancreaze, a porcine-derived PERT. The new clinical trial, RESULT (NCT03051490), will further assess the efficacy and safety of Sollpura capsules in comparison to this PERT in CF patients with EPI, adjusting for dose frequency and levels as needed by an individual.
“We have been involved with the Sollpura program since Anthera initiated its clinical development with the SOLUTION study and we are pleased that the RESULT study is now underway in Europe,” said Amparo Solé Jover, MD, PhD, pulmonologist at Hospital La Fe de Valencia, also in Spain, and president of the Sociedad Española de Fibrosis Quística.
The trial is expected to enroll about 150 patients, ages 7 and above, on a stable porcine PERT regime with well-controlled EPI, as measured by the coefficient of fat absorption (CFA). Participants will be randomized to receive either Sollpura or Pancreaze for four weeks. During the initial three weeks, researchers will conduct dose adjustments based on a patient’s symptoms and clinical evaluation to achieve the best therapeutic benefit.
Its primary endpoint, or objective, is treatment efficacy as seen in changes in CFA levels from baseline after four weeks of treatment. Those patients given Sollpura will be followed for an additional 20 weeks to evaluate long-term safety and efficacy.
Anthera also announced that the RESULT study was approved by the Protocol Review Committee of the Cystic Fibrosis Foundation’s Therapeutics Development Network, which may bring further testing sites to this Phase 3 trial and aid in patient recruitment within the U.S.
Sollpura was also evaluated as a drinkable formulation (a powder for oral solution) in the Phase 3 SIMPLICITY clinical trial (NCT02734810). This power formulation may provide a more easy-to-administer source of PERT for pediatric patients and for those who receive their nutrition through feeding tubes.
For more information about the trial and how to participate, please visit this link.