- IRB protocol approval provides green-light to initiate trial at U.S. clinical sites
NEW YORK, April 30, 2020 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc. (NASDAQ:AZRX) (“AzurRx” or the “Company”), a company specializing in the development of non-systemic, recombinant therapies for gastrointestinal diseases, today announced that it has received Institutional Review Board (IRB) approval of its Phase 2 OPTION 2 clinical trial protocol to investigate MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI).
“We are very pleased to receive IRB approval and look forward to entering the clinic,” said James Pennington, M.D., Chief Medical Officer of AzurRx. “We believe it is important to have a synthetic recombinant alternative to the current porcine derived PERT treatment, and optimizing our MS1819 dose is one more step forward towards realizing this goal. We look forward to working with our clinical collaborators and participating patients to test the efficacy of a higher dose of MS1819 and enteric capsules in this study.”
James Sapirstein, Chief Executive Officer of AzurRx added, “We are moving all the critical regulatory, clinical and manufacturing pieces into place to initiate the OPTION 2 trial. The FDA has reviewed the clinical trial protocol with no comments; IRB protocol approval allows us to initiate the trial at U.S. clinical sites; and the recently announced support by the Therapeutics Development Network (TDN) to conduct our clinical study allows for access to its 91 accredited U.S. care centers with established expertise in CF clinical research. We are now actively engaging clinical sites in the U.S. and Europe for the trial and, barring any coronavirus delays, look forward to initiating this trial in the coming weeks.”
Phase 2 OPTION 2 Trial Design
The Phase 2 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 manner versus the current standard of care, porcine pancreatic enzyme replacement therapy (PERT) pills. The OPTION 2 trial will be 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 will be 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 (CFA). 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.
MS1819 is a recombinant lipase enzyme for the treatment of exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP). 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
Exocrine Pancreatic Insufficiency (EPI) is a condition characterized by a deficiency of the pancreatic digestive enzymes in the intestinal tract needed to breakdown the nutrients in food so that they can be properly absorbed. This deficiency results in abdominal discomfort and pain, frequent bowel movements, steatorrhea, poor fat absorption, weight loss and chronic malnutrition.
There are approximately 90,000 patients in the U.S. with EPI caused by chronic pancreatitis according to the National Pancreas Foundation, and more than 30,000 patients with EPI caused by CF according to the Cystic Fibrosis Foundation. The current therapeutic standard of care for these patients are porcine pancreatic enzyme replacement therapy (PERT) pills.
About AzurRx BioPharma, Inc.
AzurRx BioPharma, Inc. (NASDAQ:AZRX) is a biopharmaceutical company engaged in the research and development of non-systemic biologics for the treatment of patients with gastrointestinal disorders. The Company is headquartered in New York, NY, with scientific operations based in Langlade, France and clinical operations in Hayward, California. Additional information on the Company can be found at www.azurrx.com.
This press release may contain certain statements relating to future results which are forward-looking statements. These statements are not historical facts, but instead represent only the Company’s belief regarding future events, many of which, by their nature, are inherently uncertain and outside of the Company’s control. 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. Additional information concerning the Company and its business, including a discussion of factors that could materially affect the Company’s financial results, including those related to the clinical development of MS1819, the results of its clinical trials, and the impact of the coronavirus (COVID-19) pandemic on the Company’s operations and current and planned clinical trials, including, but not limited to delays in clinical trial recruitment and participation 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.
AzurRx BioPharma, Inc.
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