AzurRx BioPharma Announces Completion of Enrollment in Phase 2 Clinical Trial of MS1819 in Combination with PERT in the Treatment of Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency - Seite 2

The Phase 2 combination clinical trial of MS1819 is a multi-center study designed to investigate the safety, tolerability and efficacy of escalating doses of MS1819, in conjunction with a stable dose of PERT in order to increase the patient’s coefficient of fat absorption (CFA) levels and relieve abdominal symptoms. The study enrolled 18 patients, 12 years of age or older, with severe EPI. Patients enrolled in the study 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 on standard of care therapy only, before beginning combination therapy. Eligibility requires a CFA of less than 80%. The primary efficacy endpoint of the trial is improvement in CFA; secondary endpoints of the study are improvements in the stool weight, stool consistency, number of bowel movements, the incidence of steatorrhea, and increase of body weight.

Previously reported results from the initial five patients in the trial revealed that the combination therapy led to clinically meaningful improvements in the primary efficacy endpoint, the CFA, and improvements in the key secondary endpoints, including body weight, stool consistency and reductions in the number of bowel movements and the incidence of steatorrhea (excessive amounts of fat in the feces).

Additional information about this clinical trial can be found at: https://clinicaltrials.gov/ct2/show/NCT04302662

About the MS1819 Combination Therapy Study
The digestive standard of care for both CF and chronic pancreatitis (CP) patients with EPI are commercially-available PERTs. Ideally, a stable daily dose of PERT will enable CF patients to eat a normal to high-fat diet and minimize unpleasant gastrointestinal symptoms. In practice, however, a substantial number of CF patients do not achieve normal absorption of fat with PERTs^1,2. Achieving an optimal nutritional status, including normal fat absorption levels, in CF patients is important for maintaining better pulmonary function, physical performance and prolonging survival. Furthermore, a decline of body mass index around the age of 18 years predicts a substantial drop in lung function^3,4.