AzurRx BioPharma (AZRX) Reports First Two Patients Dosed in Phase 2b OPTION 2 Extension Study of MS1819 in CF Patients

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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 first two patients have been dosed in the Phase 2b OPTION 2 extension study of MS1819 using immediate release capsules for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis.

The company recently announced an additional study arm in the Phase 2b OPTION 2 study utilizing immediate release capsules in efforts to identify the optimal dose and delivery method of MS1819. The OPTION 2 extension phase design will test patients 18 years or older, who have already completed the OPTION 2 crossover trial, at higher doses relative to the previously conducted OPTION 1 trial. As of today, two patients have been dosed and will continue to be treated for the duration of the next two weeks.

“As expected, the Data Monitoring Committee (DMC) remains supportive of our program with no safety concerns regarding the design and protocol of the study,” said Dr. James Pennington, Chief Medical Officer of AzurRx BioPharma. “Due to enrollment in the initial crossover trial being ahead of schedule, we are given an opportunity to thoroughly explore optimal doses and capsule types that will help guide our future development plans for MS1819 in a Phase 3 program. In the meantime, we will continue our preparation for a successful FDA meeting later this year.”

Topline data results for this study are anticipated in Q1 2021, as enrollment remains strong.

About MS1819

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 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 colonpathy. 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 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.

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