AzurRx BioPharma is planning to expand the clinical development of its investigative therapy MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) caused by chronic inflammation of the pancreas and cystic fibrosis (CF) into Europe.
The company has submitted an Investigational Medicinal Product Dossier (IMPD) — similar to an Investigational New Drug (IND) application in the U.S. — to the European Medicines Agency (EMA). If approved, AzurRx will expand its Phase 2a trial (ACTRN12616000962437) into clinical centers in France.
The IMPD comprises information regarding quality, production, and control of MS1819, as well as data collected from preclinical and clinical studies.
“This IMPD submission reflects the continued geographic expansion of our MS1819-SD clinical trial program and upon approval will allow us to open clinical trial sites in France,” Thijs Spoor, CEO of AzurRx BioPharma, said in a press release. “We are proud of the outstanding efforts of our scientific team as we reach this important regulatory milestone.”
MS1819 is an engineered lipase (an enzyme that degrades fat molecules) derived from the yeast Yarrowia Lipolytica. The protein, developed by AzurRx in collaboration with Laboratoires Mayoly Spindler, is expected to help patients overcome exocrine pancreatic insufficiency (EPI), a condition that is often found in people with chronic pancreatitis, a chronic inflammation of the pancreas, and in cystic fibrosis.
EPI is characterized by an insufficient production of pancreatic enzymes, which affects patients’ ability to digest food properly.
According to the Cystic Fibrosis Foundation, more than 30,000 CF patients have EPI due to chronic pancreatitis and impaired discharge of pancreatic enzymes required for normal digestion. While the condition is commonly treated with porcine-derived enzyme replacement pills, these are associated with adverse effects and low stability and activity.
Early data from a Phase 1b trial showed preliminary signs of efficacy and a favorable safety profile in EPI patients receiving MS1819. Recent data from the ongoing Phase 2 trial continued to demonstrate MS1819’s potential to overcome this digestive disorder.
The trial is testing the safety and effectiveness of MS1819 in patients with chronic pancreatitis who have EPI. Data from the first six patients treated shows that oral MS1819 (2,240 mg per day, the highest dose tested) improved fat absorption by 21 percent, compared to baseline. This was accompanied by an improvement in the number of daily evacuations and weight of stool.