- Three OPTION 2 clinical trial sites activated in the U.S.
- Topline data expected in Q1 2021
NEW YORK, July 22, 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 initiated its Phase 2b OPTION 2 clinical trial to investigate MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI) with the activation of three clinical sites and initial screening of the first patient. Top line data from the study are anticipated in Q1 2021.
The Phase 2b multi-center study 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 against the current porcine enzyme replacement therapy (PERT) standard of care. The primary efficacy endpoint is the coefficient of fat absorption (CFA). Planned enrollment is expected to include approximately 30 CF patients in the U.S. and Poland.
“We are excited to be a clinical trial site in the OPTION 2 study and to continue participating in AzurRx’s ongoing efforts to find a safe non-porcine enzyme replacement therapy for our patients with cystic fibrosis,” commented Steven R. Boas, M.D., FAAP, FACCP, President and CEO, The Cystic Fibrosis Institute. “I believe that the clinical protocol provides optimal safety conditions for participating patients and care providers, given concerns about COVID-19, and I am confident that we will be able to meet our enrollment targets for the trial in a timely fashion.”
Dr. James Pennington, Chief Medical Officer of AzurRx, added, “We have designed the OPTION 2 efficacy study based on the results from the OPTION bridging dose safety study, and are optimistic that the enteric capsules delayed-release delivery of the 2.2g and 4.4g doses of MS1819 will enable us to achieve our primary and secondary efficacy endpoints in this study. We thank our clinical collaborators and participating patients for their support of OPTION 2 and look forward to our clinical results.”
“The initiation of the OPTION 2 trial is a major clinical milestone for the Company,” said James Sapirstein, Chief Executive Officer of AzurRx. “Previous clinical trials have demonstrated that MS1819 is safe and well tolerated. With this trial, we are moving ahead to determine the optimal dose for MS1819 in preparation for a pivotal Phase 3 study. We look forward to the successful completion of the trial and to reporting our topline results of OPTION 2 in Q1 2021.”
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 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. 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 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 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 cystic fibrosis according to the Cystic Fibrosis Foundation. Patients are currently treated with porcine pancreatic enzyme replacement pills.
About AzurRx BioPharma, Inc.
AzurRx BioPharma, Inc. (NASDAQ: AZRX) is a biopharmaceutical company specialized in the research and development of non-systemic biologics for gastrointestinal disorders. The Company is focused on the development of its lead drug candidate, MS1819. AzurRx is currently conducting two Phase 2 clinical trials of MS1819: the OPTION 2 monotherapy trial, and the Combination therapy trial, consisting of MS1819 in conjunction with porcine-derived pancreatic enzyme replacement therapy, the current standard of care. 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.
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AzurRx BioPharma, Inc.
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