AzurRx Biopharma, along with partner Mayoly Spindler, announced that its lead asset met its primary and secondary endpoints in a phase 2a trial. The drug, a recombinant lipase, is designed to treat an enzyme condition linked to chronic pancreatitis.
The candidate, called MS1819-SD, is designed to treat exocrine pancreatic insufficiency (EPI), a condition caused by chronic pancreatitis. Scarring and inflammation stop the pancreas from being able to make enough exocrine enzymes, resulting in the inability to digest food properly—fats especially. It is currently treated with supplements that help patients keep up their levels of fat-soluble vitamins, as well as with pancreatic enzyme replacement therapies (PERTs). However, there is no FDA-approved PERT that is not derived from pigs.
“EPI is an underserved, billion-dollar market with limited alternatives, marked by limited effectiveness of current therapies, safety challenges, sourcing and supply issues, as well as a high pill burden, which is inconvenient for patients and results in non-adherence,” said AzurRx CEO Thijs Spoor in a statement. “Based on the positive results of our phase 2a study, we are even more confident in the outlook and market potential for MS-1819. If approved, MS-1819 would be the first non-porcine product available for EPI, with the potential to lower pill burden in patients with disease.”
AzurRx’s drug is a recombinant lipase: a fat-digesting enzyme. The companies enrolled 11 patients with chronic pancreatitis in France, Australia and New Zealand. The patients “washed out” of their standard-of-care treatment for EPI and then were treated with rising doses of MS1819-SD in two-week increments. The primary endpoint of the study was evaluating the safety of escalating doses of the drug in chronic pancreatitis patients, and the secondary endpoint was to assess the drug’s efficacy by analyzing patients’ coefficient of fat absorption (CFA) and how it changed from baseline.
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MS1819-SD met both its primary and secondary endpoints, with the highest dose achieving a statistically significant boost in CFA (21.8%), the companies reported. The study also posted encouraging data for other evaluated endpoints, including the Bristol stool scale, number of daily evacuations and stool weight, they said. There were no severe adverse events. AzurRx is planning a phase 2b trial later this year.
“We are very pleased that the phase 2a data show a statistically significant improvement in CFA of 21.8% at the highest dose, which compares quite favorably with historical data for the currently available porcine agents in patients with chronic pancreatitis,” Spoor said. “These results support our confidence in the next phase of MS1819’s clinical development as a new therapy for patients suffering from cystic fibrosis.”