- First three patients enrolled in dose escalation trial using delayed-release enteric capsules
- Topline data expected in 1H 2021
NEW YORK, Aug. 13, 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 is has enrolled the first three patients in its Phase 2b OPTION 2 clinical trial to investigate MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI). The trial will enroll approximately 30 CF patients with top line data from the study anticipated in 1H 2021.
The Phase 2b multi-center study is designed to investigate the safety, tolerability, and efficacy of MS1819 (2.2 gram 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), with secondary endpoints of stool weight, signs and symptoms of malabsorption and coefficient of nitrogen absorption (CNA).
“Enrolling the first patient in our Phase 2b OPTION 2 trial is an important milestone and we appreciate the support from our clinical trial specialists and centers. We are optimistic that our delayed-release delivery of MS1819 will achieve our primary and secondary efficacy endpoints in this study,” said Dr. James Pennington, Chief Medical Officer of AzurRx. “We anticipate data from this trial will inform the optimal dose for our pivotal Phase 3 study, and look forward to top line results in the first quarter of next year.”
James Sapirstein, Chief Executive Officer of AzurRx, added, “We are very pleased to announce that patient dosing in the OPTION 2 trial has begun in the U.S. This represents an important milestone in the clinical development of MS1819 as an EPI monotherapy for patients with cystic fibrosis. Despite the challenges posed by the COVID-19 pandemic, the AzurRx team has been able to successfully activate new clinical trial sites and work with investigators to safely admit patients into the trial.”
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 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 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, 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 such as the interim results presented will be indicative of the final results of the trial. 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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