AzurRx BioPharma, in partnership with Mayoly Spindler, announced that a recent Phase 2a clinical trial testing MS1819-SD as a potential treatment for exocrine pancreatic insufficiency (EPI) achieved its goals, namely a statistically significant improvement in fat absorption.
EPI, often caused by cystic fibrosis, is a disease resulting from a lack of exocrine pancreatic enzymes, which play a role in breaking down food molecules. Consequently, patients with EPI have trouble digesting food. The current standard treatment for EPI is porcine pancreatic enzyme replacement pills, which consist of enzymes derived from pigs.
MS1819-SD, an investigational treatment given as an oral biologic capsule, is a recombinant form of an enzyme called lipase that is derived from the yeast Yarrowia lipolytica — a species of fungus — and therefore does not contain animal products.
To assess the safety and tolerability of MS1819-SD, researchers conducted an open-label, multicenter, dose escalation Phase 2a trial (NCT03481803) which included 11 chronic pancreatitis patients in France, Australia, and New Zealand.
Chronic pancreatitis, another cause of EPI, refers to a condition characterized by long-term inflammation of the pancreas.
During the trial, patients were “washed-out” of their standard EPI treatment to establish a starting point, and were then treated with escalating doses of MS1819-SD in two-week increments.
Final data from the study indicated that MS1819-SD has a favorable safety profile with no severe adverse events.
While the study was not powered for effectiveness, the group of patients treated with the highest dose of MS1819-SD showed a statistically significant and clinically meaningful increase in the coefficient of fat absorption (CFA) — a measure of fat digestion — compared with initial values, with an average increase of 21.8%.
Additionally, favorable trends were observed on other tests including the Bristol stool scale, number of daily evacuations, and stool weight — all of which were consistent with the CFA results.
“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,” Thijs Spoor, CEO of AzurRx, said in a press release. “These results support our confidence in the next phase of MS1819’s clinical development as a new therapy for patients suffering from cystic fibrosis.”
Currently available treatments for EPI are plagued with safety and tolerability issues, sourcing and supply issues, as well as a high pill burden, which results in inconvenience for patients and noncompliance to medication.
“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,” Spoor said.
“Our patient satisfaction is very high in this study as underscored by an expressed interest in joining in further studies of MS1819. Physician response has also been favorable. We are delighted to play a key role in developing products that have significant potential to change patient care,” said Nam Q. Nguyen, PhD, a professor at the Royal Adelaide Hospital and one of the investigators of the trial.
Full results from the Phase 2a trial are expected to be presented at a major upcoming conference. In the meantime, the company is planning to test MS1819-SD in patients with cystic fibrosis.
“The statistically significant improvement of MS1819-SD on CFA in patients with chronic pancreatitis is very encouraging. We look forward to initiating our Phase 2b trial of MS1819-SD in cystic fibrosis patients later this year,” said James Pennington, MD, chief medical officer of AzurRx.
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