AzurRx BioPharma has initiated a Phase 2 clinical trial testing its oral capsule MS1819-SD, combined with standard porcine pancreatic enzyme replacement therapy (PERT), in people with cystic fibrosis (CF) who have exocrine pancreatic insufficiency (EPI).
PERT is the standard of care for EPI associated with CF or chronic pancreatitis. It was designed to adequately replace pancreatic enzymes that are missing in people with CF, so that digestion can proceed normally. Specifically, the therapy helps fat to be absorbed properly, thus allowing patients to eat a diet with a normal amount of fat and not experience gastrointestinal symptoms.
However, a “substantial number” of CF patients with EPI can’t attain normal fat absorption with PERT alone, the company said.
The new Phase 2 clinical trial is designed to assess the safety, tolerability, and efficacy of increasing doses of MS1819-SD combined with an unchanging, standard PERT dosage in people with CF who have severe EPI. Patients enrolled in the study should have persistent malnutrition and fat malabsorption despite daily treatment with PERTs.
MS1819-SD, like PERT, is an enzyme-based therapy. However, MS1819-SD is derived from a species of yeast, Yarrowia lipolytica. AzurRx has made a point of emphasizing that MS1819-SD does not contain any animal products (yeast are fungi). PERT is a pig-derived enzyme.
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Outcomes of the study will include relieving gastrointestinal symptoms, and increasing the coefficient of fat absorption — the percentage of fat in the diet that the body absorbs. Results from this study are expected in early 2020.
“The study of MS1819-SD in combination with PERT, the existing standard of care for treating severe EPI in CF patients, focuses on the clinical needs of the one-third of patients whose nutritional needs cannot be met with PERT alone,” Thijs Spoor, CEO of AzurRx, said in a press release.
“Together with our Phase 2 OPTION study to investigate MS1819-SD as a replacement for PERT, we are seeking to address the needs of CF patients by exploring the efficacy of both MS1819-SD replacement and combination therapies,” Spoor added.
Planned enrollment in the new trial is expected to include approximately 24 CF patients with severe EPI. The first patient visits have been completed in Hungary, the company said.
“The clinical nutritional needs of CF patients with severe EPI have long been underserved,” said James Pennington, MD, chief medical officer of AzurRx.
“We are optimistic that the planned study can provide these patients with a treatment plan that utilizes a safe, non-porcine therapy to potentially bolster the efficacy of existing PERTs,” he added.