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Topline Data from Phase 2b OPTION 2 Trial Expected in First Quarter 2021
DELRAY BEACH, Fla., Jan. 26, 2021 (GLOBE NEWSWIRE) — AzurRx BioPharma, Inc. (“AzurRx” or the “Company”) (NASDAQ: AZRX), a company specializing in the development of targeted non-systemic, recombinant therapies for gastrointestinal (GI) diseases, today announced the activation of two additional clinical trial sites in Poland for the extension arm of the Phase 2b OPTION 2 study investigating immediate-release capsules of MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF).
Additional sites were added in Europe to minimize any potential patient enrollment delays during the ongoing COVID-19 pandemic. The two sites in Poland, as well as the active clinical trial sites in the U.S., are actively enrolling patients, and the Company anticipates reporting topline data during the first quarter 2021.
OPTION 2 is a Phase 2b, open-label, multi-center, crossover clinical trial designed to investigate the safety, tolerability, and efficacy of MS1819 in a head-to-head comparison against the current standard of care, porcine pancreatic enzyme replacement therapy (PERT) pills. The primary efficacy endpoint is the coefficient of fat absorption (CFA).
The extension arm of the Phase 2b OPTION 2 trial is designed to test immediate release capsules to identify the optimal dose and delivery method of MS1819. The extension arm will enroll patients 18 years or older who have completed the OPTION 2 crossover trial at higher dose levels relative to the previous OPTION 1 trial. The first participants in the expansion arm were recently dosed and will be treated for a two-week period.
“MS1819 has the potential to redefine the treatment of exocrine pancreatic insufficiency, a debilitating gastrointestinal condition common to patients with cystic fibrosis that can lead to a chronic nutritional deficiency,” said James Sapirstein, President and Chief Executive Officer of AzurRx. “The goal of the MS1819 program is to provide cystic fibrosis patients with a safe and effective therapy that enables them to control EPI and achieve the nutrition necessary for healthy growth, weight maintenance, and good lung function. The recently initiated extension arm to the Phase 2b OPTION 2 trial provides the opportunity to investigate an immediate-release capsule formulation of MS1819, which we believe could offer important advantages to cystic fibrosis patients in their daily management of EPI. Overall, the OPTION 2 trial of MS1819 is proceeding ahead of schedule, and we look forward to reporting topline data in the first quarter of 2021.”
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, porcine pancreatic enzyme replacement therapy (PERT), the MS1819 synthetic lipase does not contain any animal products.
The global market for PERT was estimated to be approximately $1.4 billion in the U.S. and over $2 billion globally in 2019. There currently is no non-animal derived enzyme replacement therapy in the market for the treatment of exocrine pancreatic insufficiency. AzurRx believes that MS1819 has the potential to provide a safe and effective non-animal derived, or synthetic, alternative to PERT, without the risk of animal pathogen transmission or fibrosing colonopathy. In addition, we believe that MS1819 has the potential to improve patient compliance and quality of life given anticipated reductions in pill burden and pill size relative to PERT.
About Exocrine Pancreatic Insufficiency
EPI is a condition characterized by a 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 clinical programs. 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-Associated 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.
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, 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 such as the interim results presented will be indicative of the final results of the trial; and the Company’s success in raising additional financing to support its operations. 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.
For more information:
AzurRx BioPharma, Inc.
1615 South Congress Avenue
Delray Beach, Florida 33445
Phone: (646) 699-7855
Investor Relations contact:
LifeSci Advisors, LLC.
Hans Vitzthum, Managing Director
1 International Place, Suite 1480
Boston, MA 02110
Phone: (617) 430-7578
Tiberend Strategic Advisors
35 W. 35th Street, 11th Floor
New York, NY 10001-0080
Phone: (212) 375-2665