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AzurRx Doses First Two People in Expanded Phase 2b Trial of Cystic Fibrosis Patients

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AzurRx BioPharma shares traded 45% higher after the firm advised it had dosed the first two patients in its Phase 2b OPTION 2 Extension Study of MS1819 for treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis.

Clinical stage biopharmaceutical company AzurRx BioPharma Inc. (AZRX:NASDAQ), which focuses its efforts on developing targeted, non-systemic recombinant therapies for treating gastrointestinal (GI) diseases, yesterday announced that "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 (EPI) in patients with cystic fibrosis (CF)."

AzurRx BioPharma noted that it recently announced that it had established an additional study arm in the Phase 2b OPTION 2 study. This study is using immediate release capsules to select the optimal dose and delivery method of MS1819. The firm stated that the OPTION 2 extension phase will test patients who are 18 years or older who already completed the OPTION 2 crossover trial. The company relayed that the first two patients have now been dosed at higher dosage levels and will continue treatment for a total of two weeks. The firm stated that enrollment remains strong in the OPTION Phase 2 extension study and that it expects to receive topline data from trial in Q1/21.

AzurRx BioPharma's Chief Medical Officer Dr. James Pennington commented, "As expected, the Data Monitoring Committee (DMC) remains supportive of our program with no safety concerns regarding the design and protocol of the study...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."

The company indicated that "MS1819 is a recombinant lipase enzyme for the treatment of EPI associated with CF and chronic pancreatitis." The firm explained that "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." The company noted that MS1819 is a synthetic lipase, which, unlike the currently accepted standard of care now, porcine pancreatic enzyme replacement therapy (PERT), and is not derived from or contain any animal products.

AzurRx estimated that in 2019 the global market for PERT could be greater than $2 billion worldwide. The company believes that "MMS1819 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."

The firm advised that EPI is characterized by deficiency of the exocrine pancreatic enzymes that does not allow a person to properly digest food, resulting in a condition called maldigestion. The company pointed out that according to the Cystic Fibrosis Foundation there are over 30,000 patients in the U.S. with EPI caused by CF and the National Pancreas Foundation lists that there are around 90,000 people in the U.S. with EPI caused by chronic pancreatitis.

AzurRx BioPharma is headquartered in Delray Beach, Fla., and is a clinical stage biopharmaceutical company that concentrates its effort on developing targeted, non-systemic therapies for GI related diseases. The firm noted that it is concurrently working on three separated gut-restricted GI clinical programs. These include two Phase 2 clinical trials for its leading therapeutic candidate MS1819 for treatment of EPI in patients with CF and chronic pancreatitis. The company noted that it is additionally launching two other clinical programs in the areas of associated colitis and diarrhea in oncology patients and COVID-19 GI infections.

AzurRx began the day with a market cap of around $43.4 million with approximately 34.9 million shares outstanding. AZRX shares opened slightly lower today at $1.22 (-$0.0178, -1.44%) from yesterday's $1.24 closing price, but then turned positive and reached a new 52-week high this morning of $2.63. The stock has traded today between $1.22 and $2.63 per share and is presently trading at $1.84 (+$0.60, +48.79%).


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