Benzinga | Nov 30, 2020 08:03AM EST

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

* 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.