AzurRx BioPharma (AZRX) Reports Complete Enrollment in Phase 2b OPTION 2 Clinical Trial of MS1819 in Cystic Fibrosis

March 16, 2021 7:06 AM EDT

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AzurRx BioPharma, Inc. (NASDAQ: AZRX) today announced it has completed enrollment for its Phase 2b OPTION 2 clinical trial to investigate MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI). The trial enrolled 30 CF patients and top line data from the study is anticipated by the end of Q1 2021.

The Phase 2b multi-center study is designed to investigate the safety, tolerability and efficacy of MS1819 in enteric capsules) in a head-to-head comparison against the current porcine enzyme replacement therapy (PERT) standard of care. The primary efficacy endpoint is the coefficient of fat absorption (CFA), with secondary endpoints of stool weight, signs and symptoms of malabsorption and coefficient of nitrogen absorption (CNA). The trial also includes an extension arm that uses an immediate release MS1819 capsule, allowing the Company to compare data from the existing arm that uses enteric (delayed release) capsules with data from the new arm, and ultimately select the optimal delivery method.

“Completion of enrollment in our Phase 2b OPTION 2 trial is an important milestone in the clinical development of MS1819 as an EPI monotherapy for patients with cystic fibrosis,” said James Sapirstein, CEO, President and Chairman of AzurRx. “Data from this trial will inform the optimal dose for a potential pivotal Phase 3 study, and we are on target to report topline results from this study at the end of the first quarter of 2021.”

Dr. James Pennington, Chief Medical Officer of AzurRx, added, “The overarching goal of our MS1918 program is to provide a safe and effective therapy to control EPI and improve upon the current standard of care, PERT, which is porcine-derived and requires patients to take upwards of 40 capsules per day to control symptoms. MS1819 is a synthetic lipase that does not contain any animal products and offers the potential to substantially reduce the daily pill burden currently required to manage EPI.”

Additional information about this clinical trial can be found at: https://www.clinicaltrials.gov/ct2/show/NCT04375878

Phase 2 OPTION 2 Trial DesignThe Phase 2b OPTION 2 multi-center trial is designed to investigate the safety, tolerability and efficacy of MS1819 (2.2 and 4.4 gram doses in enteric capsules) in a head-to-head comparison versus the current standard of care, porcine pancreatic enzyme replacement therapy pills. The OPTION 2 trial is an open-label, crossover study, conducted in 15 sites in the U.S. and Europe. A total of 30 CF patients 18 years or older are enrolled. MS1819 will be administered in enteric capsules to provide gastric protection and allow optimal delivery of enzyme to the duodenum. Patients will first be randomized into two cohorts: to either the MS1819 arm, where they receive a 2.2 gram daily oral dose of MS1819 for three weeks; or to the PERT arm, where they receive their pre-study dose of PERT pills for three weeks. After three weeks, stools will be collected for analysis of coefficient of fat absorption. Patients will then be crossed over for another three weeks of the alternative treatment. After three weeks of cross-over therapy, stools will again be collected for analysis of CFA. A parallel group of patients will be randomized and studied in the same fashion, using a 4.4 gram daily dose of MS1819. All patients will be followed for an additional two weeks after completing both crossover treatments for post study safety observation. Patients will be assessed using descriptive methods for efficacy, comparing CFA between MS1819 and PERT arms, and for safety.

About MS1819MS1819 is a recombinant lipase enzyme for the treatment of exocrine pancreatic insufficiency associated with cystic fibrosis and chronic pancreatitis. MS1819, supplied as an oral non-systemic biologic capsule, is derived from the Yarrowia lipolytica yeast lipase and breaks up fat molecules in the digestive tract of EPI patients so that they can be absorbed as nutrients. Unlike the standard of care, the MS1819 synthetic lipase does not contain any animal products.

About Exocrine Pancreatic InsufficiencyEPI is a condition characterized by deficiency of the exocrine pancreatic enzymes, resulting in a patient’s inability to digest food properly, or maldigestion. The deficiency in this enzyme can be responsible for greasy diarrhea, fecal urge and weight loss.

There are more than 30,000 patients in the U.S. with EPI caused by cystic fibrosis according to the Cystic Fibrosis Foundation and approximately 90,000 patients in the U.S with EPI caused by chronic pancreatitis according to the National Pancreas Foundation. Patients are currently treated with porcine pancreatic enzyme replacement pills.



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