AzurRx BioPharma announced that its clinical trial testing MS1819-SD as a replacement enzyme therapy for exocrine pancreatic insufficiency (EPI) associated with chronic pancreatitis (inflammation of the pancreas) has enrolled three new patients, completing 75 percent of its goal.
Although patients with cystic fibrosis (CF) are excluded from the trial, CF is the second most common cause of EPI after chronic pancreatitis. In the United States alone, it affects more than 30,000 CF patients, according to the Cystic Fibrosis Foundation. Therefore, the results of this clinical trial are particularly important for CF patients.
Enrollment is made in collaboration with Mayoly Spindler, a French pharmaceutical company, and now nine of a total of 12 patients have joined the trial.
The Phase 2a study (ACTRN12616000962437) is an open-label study, meaning that all patients will receive MS1819-SD, and its primary objective is to assess the safety of escalating doses of MS1819-SD in patients with chronic pancreatitis.
The trial will test a total of four oral doses of MS1819-SD – 280 mg a day; 560 mg a day; 1,120 mg/day; and 2,240 mg/day – and the treatment will be for a total of 48 to 60 days.
Safety will be assessed at the end of each treatment period and includes testing for immunoallergic reactions, digestive symptoms and laboratory tests.
Additional (secondary) objectives include investigating the effectiveness of MS1819SD in chronic pancreatitis patients by analyzing the coefficient of fat absorption (CFA) – an indicator of fat malabsorption – and its change from the beginning of the trial (baseline). For that, researchers will analyze the fat content in stools collected throughout five days after ingestion of high-fat meals at the end of each treatment phase.
Before initiating the first of four treatments, participants will undergo a washout period to remove any porcine pancreatic extracts, a set of enzymes extracted from pig pancreas glands and currently the only therapy available for pancreatic insufficiency.
MS1819-SD is a recombinant lipase – an enzyme that breaks down fats in food so they can be absorbed in the intestines – extracted from the yeast Yarrowia lipolytica.
Tested in previous preclinical studies with animals and later on in human trials, MS1819-SD administration was capable of compensating for the deficiency of the pancreatic lipase that characterizes chronic pancreatitis patients. Lack of adequate levels of pancreatic lipase leads to greasy diarrhea, fecal urge, and weight loss.
“We are very pleased that our existing clinical sites have recruited these new patients. After a rapid clearance of the Investigational Medicinal Product Dossier (IMPD) for our site in France (La Timone Hospital, Marseille), we believe we have now identified enough potential patients to complete enrollment of the study in the first half of 2018, in line with previously published guidance,” Thijs Spoor, CEO of AzurRx BioPharma, said in a press release.