AzurRx BioPharma Announces First Two Patients Dosed in Phase 2b OPTION 2 Extension Study of MS1819 in Cystic Fibrosis Patients

Rhea-AI Summary

AzurRx BioPharma (NASDAQ: AZRX) announced the dosing of two patients in the Phase 2b OPTION 2 extension study of MS1819, aimed at treating exocrine pancreatic insufficiency in cystic fibrosis patients. This study explores higher doses of MS1819 using immediate-release capsules and follows a successful initial crossover trial. The Data Monitoring Committee has expressed no safety concerns. Topline results are expected in Q1 2021, and AzurRx is preparing for a key FDA meeting later this year.

Positive

• Enrollment for the initial crossover trial was ahead of schedule, allowing for further exploration of dosing.
• No safety concerns reported by the Data Monitoring Committee.
• Topline data results for the MS1819 study expected in Q1 2021.

Negative

• None.

DELRAY BEACH, Fla., Jan. 21, 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 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.

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.

Forward-Looking Statements

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
Suite 103
Delray Beach, Florida 33445
Phone: (646) 699-7855
info@azurrx.com 

Investor Relations contact:

LifeSci Advisors, LLC. 
Hans Vitzthum, Managing Director 
1 International Place, Suite 1480 
Boston, MA 02110 
Phone: (617) 430-7578 
hans@lifesciadvisors.com 

FAQ

What is the purpose of the Phase 2b OPTION 2 study for AZRX?

The Phase 2b OPTION 2 study aims to evaluate the efficacy of MS1819 in treating exocrine pancreatic insufficiency in cystic fibrosis patients by testing immediate-release capsules at higher doses.

When will the results of the MS1819 study be announced?

Topline results for the MS1819 study are anticipated in the first quarter of 2021.

What did the Data Monitoring Committee report regarding the study's safety?

The Data Monitoring Committee reported no safety concerns regarding the design and protocol of the study.

What is the significance of the MS1819 drug for AZRX?

MS1819 is a recombinant lipase enzyme with potential to offer a non-animal based treatment for exocrine pancreatic insufficiency, aiming to improve patient compliance and quality of life.

What are AZRX's next steps following the Phase 2b study?

Following the Phase 2b study, AZRX will prepare for a significant meeting with the FDA later in the year.