AzurRx BioPharma Announces Positive Interim Data in Phase 2 Clinical Trial of MS1819 in Combination with PERT in the Treatment of Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency

Rhea-AI Summary

AzurRx BioPharma (NASDAQ: AZRX) announced positive interim results from its Phase 2 trial evaluating MS1819 in combination with porcine-derived pancreatic enzyme replacement therapy (PERT) for treating severe exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients. The first 18 patients showed a significant average improvement of 5.9 points in the Coefficient of Fat Absorption (CFA), exceeding the clinically significant threshold. Full results from 20 patients are expected in Q2 2021, with potential implications for patients unable to achieve adequate nutrition using PERT alone.

Positive

• MS1819 and PERT combination therapy led to a significant average improvement of 5.9 points in CFA from baseline.
• Interim results indicate potential benefits for 25-30% of CF patients with severe EPI not responding adequately to PERT.
• All patients reported positive responses to MS1819, improving overall quality of life.

Negative

• None.

Data from first 18 patients indicate MS1819 and PERT combination led to clinically meaningful improvements in primary efficacy endpoint

Full top-line data expected in Q2 2021

DELRAY BEACH, Fla., May 13, 2021 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc. (NASDAQ: AZRX), (“AzurRx” or the “Company”), a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced positive interim data from the first 18 out of 20 patients in its Phase 2 trial evaluating 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).

The interim topline data revealed that the combination therapy led to clinically meaningful improvements in the primary efficacy endpoint, the Coefficient of Fat Absorption (CFA), with an average gain of 5.9 points from baseline. According to the clinical literature, a five-point improvement in CFA is considered clinically significant.¹

“We are encouraged by the promising interim data from the first 18 patients in our Phase 2 clinical trial, evaluating MS1819 in combination with PERT for the treatment of EPI in cystic fibrosis patients,” said James Sapirstein, President, CEO and Chairman of AzurRx. “Based on the clinical results to date, we believe that the combination therapy has significant potential to help the 25-30% of refractory cystic fibrosis patients with severe EPI who are unable to achieve adequate nutrition using PERT alone. Adding MS1819 to the treatment regimen appears to enable these patients to meet their nutritional needs, reduce the debilitating symptoms of EPI, and improve their overall quality of life, with an acceptable safety profile. We expect to report full topline results from all 20 patients enrolled in the trial in the second quarter of 2021.”

Dr. Bulent Karadag, the principal investigator in the trial from the Marmara University Faculty School of Medicine in Istanbul, Turkey, stated, "Many of my patients suffer from severe EPI and a number are not adequately controlled. They frequently reported that they felt better after taking MS1819 over the course of the six-week trial."

Dr. James Pennington, Chief Medical Officer of AzurRx, commented, “The overarching goal of our MS1819 program is to provide a safe and effective therapy to control EPI, a debilitating gastrointestinal condition common to patients with cystic fibrosis that can result in numerous, life-altering complications, including malnutrition. Based on the clinical evidence from the combination trial to-date, we have been able to improve the nutritional status of the severe EPI patients who need the most help. It turns out that the patients with the lowest baseline CFAs had the largest improvements in CFA in the study. It is also very encouraging to note that all the patients responded well and that many reported that they felt better overall when MS1819 was added to their daily dose of PERT.”

The Phase 2 combination clinical trial is a multi-center study designed to investigate the safety, tolerability and efficacy of escalating doses of MS1819, in conjunction with a stable dose of PERT, in order to increase the patient’s coefficient of fat absorption (CFA) levels and relieve abdominal symptoms. The study enrolled 20 patients, 12 years of age or older, with severe EPI who were treated escalating doses of MS1819 (700mg, 1200mg, and 2240mg) once daily for 15 days per dosing level, in addition to their standard PERT dose. Baseline CFA is established by measuring CFA levels while on standard of care therapy only, before beginning combination therapy. Trial eligibility requires a CFA of less than 80%. The primary efficacy endpoint of the trial is improvement in CFA; secondary endpoints of the study are improvements in the stool weight, stool consistency, number of bowel movements, the incidence of steatorrhea, and increase of body weight.

Additional information about this clinical trial can be found at: https://clinicaltrials.gov/ct2/show/NCT04302662

About the MS1819 Combination Therapy Study
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 1Q 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 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 assets. 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 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 Statement
This press release may contain certain statements relating to future results which are forward-looking statements. 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 will be indicative of the final results of the trial; the size of the potential markets for the Company’s drug candidates and its ability to service those markets; and the Company’s current and future capital requirements and its ability to raise additional funds to satisfy its capital needs. Additional information concerning the Company and its business, including a discussion of factors that could materially affect the Company’s financial results are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020 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

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

¹ Brady, M.S et al, 2006, Journal of American Dietetic Association, 2006, 1181-1185.
² Freedman, S.D., Am. J. Manag. Care, 2017; 23: S2220-S228
³ Littlewood, J. et al, 2006, Pediatric Pulmonology, 41:35-49
⁴ Engelen, M. et al, 2014, Curr. Opin. Clin. Nutr. Metab. Care; 17(6):515-520
⁵ Vandenbranden, S.L. et al, 2012, Pediatric Pulmonology, 2012; 47(2): 135-143

FAQ

What are the interim results of the Phase 2 trial for AZRX's MS1819?

The interim results show a significant average improvement of 5.9 points in Coefficient of Fat Absorption (CFA) from baseline among the first 18 patients.

How many patients were enrolled in the AZRX Phase 2 trial for MS1819?

The Phase 2 trial enrolled a total of 20 patients with severe exocrine pancreatic insufficiency.

When will the full results of the AZRX trial be available?

Full top-line results from the trial are expected to be reported in Q2 2021.

What is the significance of the CFA improvement in the AZRX trial?

An improvement of five points in CFA is considered clinically significant, indicating potential therapeutic benefits from the combination therapy.

What condition does MS1819 aim to treat in the AZRX trial?

MS1819 aims to treat severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis.