AzurRx BioPharma is planning a multi-center Phase 2 trial to be conducted in the United States and Europe to evaluate its lead candidate MS1819-SD as a treatment for patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).
This announcement follows the approval by the U.S. Food and Drug Administration (FDA) of an Investigational New Drug (IND) application for MS1819-SD submitted by the company.
The trial is expected to start by the end of 2018.
“We are moving forward aggressively with our plans for the Phase 2 multi-center study in cystic fibrosis, which we expect will include approximately 30 patients in a head-to-head test against the current standard of care,” James Pennington, MD, chief medical officer of AzurRx, said in a press release. “We expect to complete this study in 2019 and look forward to reporting data shortly thereafter.”
Many CF patients have EPI because mucus blocks the pancreatic ducts, which control the release of pancreatic enzymes needed for digestion. These patients must take enzymes to overcome this deficit and maintain normal digestion and nutritional health.
The current treatment for EPI is porcine pancreatic enzyme replacement pills, which consist of enzymes derived from pigs.
MS1819-SD is an oral investigational therapy designed to restore the balance of digestive enzymes that degrade fatty molecules. It is a man-made version of the lipase enzyme from the yeast Yarrowia lipolytica, meaning that unlike the currently available therapies, it does not contain animal products.
Using this strategy, researchers expect to overcome one of the major risks of products of animal origin — contamination with potential hazards from the animal source.
“Current treatments utilize animal-based products, which can carry inherent risks, including transmission of pathogens and manufacturing/supply chain inconsistency. In contrast, MS1819-SD could reduce a patient’s pill burden and, since it is derived from yeast, we believe the manufacturing process should be more scalable in larger volumes,” said Thijs Spoor, CEO of AzurRx.
Results from a Phase 2a study, conducted in collaboration with Mayoly Spindler, have demonstrated the potential of this investigational therapy to improve fat absorption. The study (NCT03481803) enrolled 11 patients with chronic pancreatitis — another cause of EPI — across France, Australia, and New Zealand. Patients were treated with escalating doses of MS1819-SD in two-week increments.
Although the study was not powered to determine the effectiveness of the treatment, patients who received the highest dose of MS1819-SD had an average increase of 21.8% in the coefficient of fat absorption — a measure of fat digestion — compared with their initial values.
Other tests, including the Bristol stool scale, number of daily evacuations, and stool weight, also showed to be improved upon treatment with MS1819-SD.
“FDA clearance for our Phase 2 study of MS1819-SD in cystic fibrosis patients represents a major milestone for the company,” Spoor said. “This approval follows on the heels of our successful Phase 2a EPI trial in patients with chronic pancreatitis (CP), which demonstrated both safety and statistically significant efficacy. As a result, we are excited to move ahead with our planned Phase 2 multi-center EPI trial in patients with CF.”