AzurRx BioPharma Announces Completion of Enrollment in Phase 2b OPTION 2 Clinical Trial of MS1819 in Cystic Fibrosis

Top-line data expected by end of Q1 2021

DELRAY BEACH, Fla., March 16, 2021 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc. (NASDAQ: AZRX), (“AzurRx” or the “Company”), a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, 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 Design
The 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 MS1819
MS1819 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 Insufficiency
EPI 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.

About AzurRx BioPharma, Inc.
AzurRx BioPharma, Inc. (NASDAQ: AZRX) is a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. The Company has a pipeline of three gut-restricted GI assets. The lead therapeutic candidate is MS1819, a recombinant lipase for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis and chronic pancreatitis, currently in two Phase 2 clinical trials. AzurRx is launching two clinical programs using proprietary formulations of niclosamide, a pro-inflammatory pathway inhibitor, FW-420, for grade 1 Immune Checkpoint Inhibitor Colitis and diarrhea in oncology patients and FW-1022, for COVID-19 gastrointestinal infections. The Company is headquartered in Delray Beach, Florida with clinical operations in Hayward, California. For more information, visit www.azurrx.com.

Forward-Looking Statement
This press release may contain certain statements relating to future results which are forward-looking statements. It is possible that the Company’s actual results and financial condition may differ, possibly materially, from the anticipated results and financial condition indicated in these forward-looking statements, depending on factors including whether results obtained in preclinical and nonclinical studies and clinical trials will be indicative of results obtained in future clinical trials; whether preliminary or interim results from a clinical trial will be indicative of the final results of the trial; and the impact of the coronavirus (COVID-19) pandemic on the Company’s operations and current and planned clinical trials, including potential delays in clinical trial recruitment and participation. Additional information concerning the Company and its business, including a discussion of factors that could materially affect the Company’s financial results are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019 under the heading “Risk Factors,” as well as the Company’s subsequent filings with the Securities and Exchange Commission. All forward-looking statements included in this press release are made only as of the date of this press release, and we do not undertake any obligation to publicly update or correct any forward-looking statements to reflect events or circumstances that subsequently occur or of which we hereafter become aware.

For more information:
AzurRx BioPharma, Inc.
1615 South Congress Avenue
Suite 103
Delray Beach, Florida 33445
Phone: (646) 699-7855
info@azurrx.com

Media contact:
Tiberend Strategic Advisors, Inc.
Johanna Bennett/Ingrid Mezo
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jbennett@tiberend.com/imezo@tiberend.com