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AzurRx BioPharma Announces Completion of Enrollment in Phase 2 Clinical Trial of MS1819 in Combination with PERT in the Treat...
March 22 2021 - 07:00AM
GlobeNewswire Inc.

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 it has completed enrollment 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).

“We are very pleased to have completed enrollment in our Phase 2 MS1819 plus PERT combination therapy clinical trial,” said James Sapirstein, President, CEO and Chairman of AzurRx. “Based on the very encouraging 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 a small dose of MS1819 can help these patients meet their nutritional needs, reduce the debilitating symptoms of EPI and improve their overall quality of life, with an increased safety profile.”

Mr. Sapirstein continued, “The combination therapy program represents a second therapeutic opportunity for the MS1819 program. We reported the completion of enrollment of our Phase 2 monotherapy program – OPTION 2 – last week and will announce topline results for that trial at the end of March. We continue to remain on target to report topline results from the combination therapy study during the second quarter of 2021.”

“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,” added Dr. James Pennington, Chief Medical Officer of AzurRx. “Early clinical evidence has been promising, and with the combination and OPTION 2 monotherapy trials progressing, we may soon have the opportunity to introduce a drug product that could potentially improve the lives of thousands of patients suffering from cystic fibrosis.”

The Phase 2 combination clinical trial of MS1819 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 18 patients, 12 years of age or older, with severe EPI. Patients enrolled in the study receive escalating doses of 700mg, 1200mg, and 2240mg of MS1819 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. 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.

Previously reported results from the initial five patients in the trial revealed that the combination therapy led to clinically meaningful improvements in the primary efficacy endpoint, the 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).

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 PERTs1,2. 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 function3,4.

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 20 CF patients with severe EPI, with study completion anticipated in 1H 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.