DELRAY BEACH, Fla.—AzurRx BioPharma, Inc. has reported the completion of enrollment in its Phase 2 trial evaluating MS1819 in combination with porcine-derived pancreatic enzyme replacement therapy (PERT) for the treatment of severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF). MS1819 is a recombinant lipase enzyme, derived from the Yarrowia lipolytica yeast lipase, that breaks up fat molecules in the digestive tract of EPI patients so that they can be absorbed as nutrients.
“We are very pleased to have completed enrollment in our Phase 2 MS1819 plus PERT combination therapy clinical trial. 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 cystic fibrosis patients with severe EPI who are unable to achieve adequate nutrition using PERT alone,” said James Sapirstein, president, CEO, and chairman of AzurRx. “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.”
The Phase 2 combination clinical trial of MS1819 is a multi-center study to investigate the safety, tolerability, and efficacy of escalating doses of MS1819 in conjunction with a stable dose of PERT. The goal is to increase the patient’s coefficient of fat absorption (CFA) levels, and to relieve abdominal symptoms. The study has enrolled 18 patients with severe EPI who were 12 years or older. Patients receive escalating doses of 700mg, 1200mg, and 2240mg of MS1819 once daily for 15 days per dosing level, in addition to their standard PERT dose.
Baseline CFA is established by measuring CFA levels while only on standard of care therapy, before beginning combination therapy. Eligibility requires a CFA of less than 80 percent. The primary efficacy endpoint of the trial is improvement in CFA; secondary endpoints of the study include improvements in the stool weight, stool consistency, number of bowel movements, the incidence of steatorrhea, and increase of body weight.
“The combination therapy program represents a second therapeutic opportunity for the MS1819 program. We reported the completion of enrollment of our Phase 2 monotherapy program — OPTION 2 — last week, and will announce topline results for that trial at the end of March. We continue to remain on target to report topline results from the combination therapy study during the second quarter of 2021,” Sapirstein continued.
Previously reported results from the initial five patients in the trial revealed that the combination therapy led to clinically meaningful improvements in CFA, and improvements in key secondary endpoints — including body weight, stool consistency, reductions in number of bowel movements, and reductions in incidence of steatorrhea.
“The overarching goal of our MS1819 program is to provide a safe and effective therapy to control EPI, a debilitating gastrointestinal condition common to patients with cystic fibrosis that can result in numerous, life-altering complications, including malnutrition,” added Dr. James Pennington, chief medical officer of AzurRx. “Early clinical evidence has been promising, and 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.”