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AzurRx BioPharma, Inc. (NASDAQ: AZRX), a company specializing in the development of targeted non-systemic, recombinant therapies for gastrointestinal (GI) diseases, today announced the completion of patient enrollment in the first cohort of the Phase 2b OPTION 2 extension study evaluating immediate-release capsules of MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF).
The Phase 2b OPTION 2 trial extension arm is currently dosing patients at clinical trial sites in the U.S. and Europe who have participated in the previous arms of the OPTION 2 trial. The goal of the MS1819 clinical program is to provide cystic fibrosis patients with a safe and effective therapy to control EPI, a debilitating gastrointestinal condition common to patients with cystic fibrosis that can lead to a chronic nutritional deficiency. For cystic fibrosis patients, maintaining proper nutritional levels is essential to ensure healthy growth, weight management and good lung function.
James Sapirstein, President and Chief Executive Officer of AzurRx BioPharma, stated, “The extension arm of the Phase 2b OPTION 2 trial is providing us with an opportunity to determine both the optimized dosage and delivery mechanism of MS1819. We have fully enrolled the most important cohort of the extension study and would like to thank both our clinical collaborators and patients for their continued participation in the study. We continue to be encouraged by the absence of any reports of serious adverse events to date, and eagerly look forward to reporting topline results from this trial in Q1 2021.”
OPTION 2 Extension StudyThe Phase 2b OPTION 2 study is an 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 for EPI, porcine pancreatic enzyme replacement therapy (PERT). The primary efficacy endpoint is the coefficient of fat absorption (CFA). AzurRx BioPharma initiated the additional study arm, the Phase 2b OPTION 2 extension trial, to identify both the optimal dose and the optimal delivery method for MS1819 using immediate-release capsules at higher dose levels than previous early-stage clinical trials.
To date, there have been no serious adverse events reported during either the Phase 2b OPTION 2 trial or its extension arm. The Phase 2b OPTION 2 extension trial is enrolling volunteers 18 years or older at trial sites in the U.S. and Poland who have completed the OPTION 2 crossover trial at higher dose levels relative to the previous OPTION 1 trial. Trial participants will be treated with MS1819 over a two-week-period. AzurRx BioPharma expects to report topline data by the end of first quarter 2021.
About MS1819MS1819 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 more than $2 billion globally in 2019. There currently is no non-animal-based 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 InsufficiencyEPI 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.