Patient enrollment has been completed for a Phase 2 clinical trial testing the investigational therapy MS1819 in combination with pancreatic enzyme replacement therapy, called PERT, for severe exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF).
Pancreatic insufficiency, known as EPI, is a complication of CF characterized by a deficiency of the enzymes that break down food and allow the body to absorb nutrients, such as fats. Without these enzymes, CF patients with EPI are unable to properly digest food and may require daily PERT.
But even that treatment, which involves digestive enzymes taken by mouth in a capsule, often is not sufficient to meet patients’ nutritional needs.
This study (NCT04302662) will assess the safety and efficacy of AzurRx BioPharma‘s MS1819, given orally along with pig-derived PERT — the current standard treatment — in 18 CF patients, ages 12 and up, with severe EPI. Top-line data from the trial are expected later this year.
“Based on the very encouraging clinical results to date, we believe that the combination therapy has significant potential to help the 25-30% of refractory [hard-to-treat] cystic fibrosis patients with severe EPI who are unable to achieve adequate nutrition using PERT alone,” James Sapirstein, president, CEO, and chairman of AzurRx, said in a press release.
Many patients taking PERTs fail to achieve optimal fat absorption or nutritional status, which impacts lung function, physical functioning, and survival. MS1819 is a synthetic, yeast-derived digestive enzyme intended to aid fat digestion in CF patients with severe EPI.
“Adding a small dose of MS1819 can help these patients meet their nutritional needs, reduce the debilitating symptoms of EPI and improve their overall quality of life, with an increased safety profile,” Sapirstein said.
The MS1819-PERT combination study will assess whether the two treatments, taken together, increase fat absorption and ease abdominal symptoms in CF patients with EPI. Participants will receive once-daily, increasing oral doses of MS1819 (700 mg, 1,200 mg, and 2,240 mg) for a total of 15 days (just over two weeks) along with their standard PERT dose.
The primary goal of the AzurRx-sponsored trial, underway in Turkey and Hungary, is improvement in fat absorption. Among the secondary goals are improvements in stool weight and consistency, number of bowel movements, a reduced incidence of steatorrhea, or excessive fat in the stool, and increased body weight.
AzurRx last year reported positive results from the first five patients treated in the study. Those findings included clinically meaningful benefits in fat absorption as well as improvements in the secondary goals with no adverse effects reported.
The company also is conducting the Phase 2b OPTION 2 study (NCT04375878) to compare the efficacy of MS1819 to PERT in people with EPI. That trial is fully enrolled with 30 adults, AzurRx announced earlier this month. Top-line results are expected by April.
“The overarching goal of our MS1819 program is to provide a safe and effective therapy to control EPI,” said James Pennington, MD, chief medical officer of AzurRx. “With the combination and OPTION 2 monotherapy trials progressing, we may soon have the opportunity to introduce a drug product that could potentially improve the lives of thousands of patients suffering from cystic fibrosis.”
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