AzurRx BioPharma Announces First Patients Dosed in Turkey for Phase 2 Combination Therapy Trial of MS1819 in Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency

Rhea-AI Summary

AzurRx BioPharma, Inc. (NASDAQ: AZRX) has dosed its first patients in Turkey for the Phase 2 Combination Trial evaluating MS1819 with standard pancreatic enzyme therapy for cystic fibrosis patients suffering from severe exocrine pancreatic insufficiency (EPI). The drug aims to improve fat absorption and thus enhance quality of life. Topline data is expected in Q2 2021. The trial, being conducted across multiple centers, has already shown positive results in initial patients, indicating potential to improve body weight and reduce bowel-related issues.

Positive

• First two patients dosed in Turkey for Phase 2 Combination Trial.
• Topline results expected in Q2 2021.
• Initial data shows significant improvements in coefficient of fat absorption and other secondary endpoints.

Negative

• None.

• Patient screening and dosing ongoing in Turkey and Hungary
  
• Topline data expected in Q2 2021 for the Combination Trial

DELRAY BEACH, Fla., Nov. 30, 2020 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc. (NASDAQ:AZRX) (“AzurRx” or the “Company”), a company specializing in the development of non-systemic, recombinant therapies for gastrointestinal diseases, today announced that the first two patients have been dosed in Turkey for its Phase 2 trial evaluating its investigational drug, MS1819, in combination with the current standard of care, porcine-derived pancreatic enzyme replacement therapy (PERT), for the treatment of severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF).

“In the midst of a global pandemic and unpredictable obstacles, we are very encouraged to see our Combination therapy trial moving forward at one of the clinical testing sites in Turkey,” said James Sapirstein, President & CEO of AzurRx.  Mr. Sapirstein continued, “We expect to release topline results in the second quarter of next year. We are optimistic the results will build on the encouraging data we received earlier this year and hope we will be one step closer to improving the lives of thousands of patients suffering from cystic fibrosis.”

Earlier last month, the Company reported that additional clinical sites were activated in Turkey, which follows Hungary as the second country where patients have been screened and dosed in the Combination trial.  As of today, there are five sites activated in Turkey that are currently screening for patients.

The Phase 2 Combination Trial, open-label, multicenter study is assessing the safety and efficacy of MS1819 administered over 15 days in immediate release capsules in increasing doses of 700mg, 1,200mg and 2,240 mg per day, in combination with a patient’s daily dose of PERT.  Previously reported results on the initial five patients in the trial revealed that the combination therapy led to clinically meaningful improvements in the primary efficacy endpoint, the coefficient of fat absorption (CFA), and improvements in the key secondary endpoints, including body weight, stool consistency and reductions in the number of bowel movements and the incidence of steatorrhea (excessive amounts of fat in the feces).

About the MS1819 Combination Therapy Trial
The digestive standard of care for both CF and chronic pancreatitis (CP) patients with EPI are commercially-available PERTs. Ideally, a stable daily dose of PERT will enable CF patients to eat a normal to high-fat diet and minimize unpleasant gastrointestinal symptoms. In practice, however, a substantial number of CF patients do not achieve normal absorption of fat with PERTs^(1,2).  Achieving an optimal nutritional status, including normal fat absorption levels, in CF patients is important for maintaining better pulmonary function, physical performance and prolonging survival. Furthermore, a decline of body mass index around the age of 18 years predicts a substantial drop in lung function^(3,4).

A combination therapy of PERT and MS1819 has the potential to: (i) correct macronutrient and micronutrient maldigestion; (ii) eliminate abdominal symptoms attributable to maldigestion; and (iii) sustain optimal nutritional status on a normal diet in CF patients with severe EPI. Planned enrollment is expected to include approximately 24 CF patients with severe EPI, with study completion anticipated in Q2 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, the MS1819 synthetic lipase does not contain any animal products.

About AzurRx BioPharma, Inc.
AzurRx BioPharma, Inc. (NASDAQ: AZRX) is a biopharmaceutical company specialized in the research and development of non-systemic biologics for gastrointestinal disorders. The Company is focused on the development of its lead drug candidate, MS1819. AzurRx is currently conducting two Phase 2 clinical trials of MS1819: the OPTION 2 monotherapy trial, and the Combination therapy trial, consisting of MS1819 in conjunction with porcine-derived pancreatic enzyme replacement therapy, the current standard of care. The Company is headquartered in Delray Beach, Florida with clinical operations in Hayward, California. Additional information on the Company can be found at 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, 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. 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

References
(1) Freedman, S.D., Am. J. Manag. Care, 2017; 23: S2220-S228
(2) Littlewood, J. et al, 2006, Pediatric Pulmonology, 2006, 41:35-49
(3) Engelen, M. et al, 2014, Curr. Opin. Clin. Nutr. Metab. Care; 17(6):515-520
(4) Vandenbranden, S.L. et al, 2012, Pediatric Pulmonology, 2012; 47(2): 135-143

FAQ

What are the goals of AzurRx's MS1819 Combination Trial?

The trial aims to assess the safety and efficacy of MS1819 in conjunction with standard pancreatic enzyme therapy to improve fat absorption in cystic fibrosis patients with severe EPI.

When will the topline results of the MS1819 trial be released?

Topline results from the trial are expected to be released in the second quarter of 2021.

How many patients are expected to be enrolled in the MS1819 trial?

Approximately 24 patients with cystic fibrosis and severe EPI are expected to be enrolled in the trial.

What is the significance of MS1819 for cystic fibrosis patients?

MS1819 aims to enhance nutrient absorption and improve gastrointestinal symptoms, potentially leading to better overall health and quality of life for patients with cystic fibrosis.