AzurRx BioPharma Announces Completion of Enrollment in First Cohort of Phase 2b OPTION 2 Extension Study of MS1819

Rhea-AI Summary

AzurRx BioPharma (NASDAQ: AZRX) has completed patient enrollment in the first cohort of the Phase 2b OPTION 2 extension study for MS1819, aimed at treating exocrine pancreatic insufficiency (EPI) in cystic fibrosis patients. This study, currently dosing patients in the U.S. and Europe, follows earlier trials and seeks to optimize dosage and delivery of MS1819. No serious adverse events have been reported thus far. The company anticipates releasing topline results by the end of Q1 2021, with MS1819 potentially filling a significant market gap valued at over $2 billion globally for non-animal-based therapies.

Positive

• Completion of patient enrollment in Phase 2b OPTION 2 trial extension.
• No serious adverse events reported in both the current and previous trials.
• Anticipation of topline results by end of Q1 2021, which could enhance market valuation.

Negative

• None.

DELRAY BEACH, Fla., Jan. 28, 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 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 Study
The 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 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 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 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.

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
(646) 699-7855
info@azurrx.com 

Investor Relations contact:

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

Media contact:

Tiberend Strategic Advisors, Inc.
Johanna Bennett/Ingrid Mezo
(212) 375-2665/(646) 604-5150
jbennett@tiberend.com/imezo@tiberend.com

FAQ

What is the purpose of the Phase 2b OPTION 2 trial for AzurRx (AZRX)?

The trial is designed to evaluate the safety, tolerability, and efficacy of MS1819 compared to the standard of care for treating exocrine pancreatic insufficiency in cystic fibrosis patients.

When will AzurRx (AZRX) report the results of the Phase 2b OPTION 2 trial?

Topline results from the trial are expected to be reported by the end of Q1 2021.

How does MS1819 differ from current therapies for exocrine pancreatic insufficiency?

MS1819 is a non-animal derived recombinant lipase, unlike the standard porcine pancreatic enzyme replacement therapy, potentially offering safer and more effective treatment options.

What is the market potential for MS1819 in treating exocrine pancreatic insufficiency?

The global market for porcine pancreatic enzyme replacement therapy was valued at over $2 billion, indicating significant potential for MS1819 as a non-animal based alternative.

What are the implications of the completion of patient enrollment in the trial for AzurRx (AZRX)?

Completion of enrollment signifies progress in the clinical development of MS1819, potentially enhancing investor confidence and market interest leading up to the results.