Initial patient experience shows no safety issues

MS1819 appears active, as demonstrated by CFA increases >20% at various dosing levels

BROOKLYN, N.Y., April 11, 2017 (GLOBE NEWSWIRE) — AzurRx BioPharma, Inc. (AZRX) (“AzurRx” or the “Company”), a company specializing in the development of non-systemic, recombinant therapies for gastrointestinal diseases, today is providing an update on the ongoing open-label Phase IIa trial of lead candidate MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) associated with chronic pancreatitis (CP). The trial is being conducted in partnership with Mayoly Spindler, a European pharmaceutical company.

Early results indicate that MS1819 exhibits a favorable safety profile, with no moderate or severe adverse events having been reported to date.  Additionally, no patients have dropped out of the study once they have qualified to take the active drug.  Regarding efficacy, results to date indicate that MS1819 is active, as measured by coefficient of fat absorption (CFA), which is the parameter being measured for the primary efficacy endpoint.  There was a favorable dose response of greater than 20% improvement in CFA seen in all patients at either the second or third of the four escalating dosage levels of MS 1819.

These early efficacy and favorable safety results from the ongoing Phase IIa trial confirm data from the previously performed Phase Ib trial of MS1819. The company expects study completion in the third-quarter of 2017.

Thijs Spoor, CEO of AzurRx BioPharma, said, “We are extremely encouraged by these early Phase IIa MS1819 data, observing a clear dose-response pattern with clean safety characteristics.  We continue to expect MS1819 to show a superior efficacy, safety, and dosing profile relative to the currently marketed pancreatic enzyme replacement therapies (PERT).”

Dr. Richard Stubbs, Managing Director of P3 Research, in Wellington, New Zealand and Clinical Investigator said, “Our patient satisfaction is very high in this study as underscored by an expressed interest in joining in further studies of MS1819. Physician response has also been favorable.  We are delighted to play a key role in developing products that have significant potential to change patient care.”

MS1819 Phase IIa Trial

AzurRx is currently conducting a Phase IIa trial of MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) associated with chronic pancreatic pancreatitis (CP).  The study is being performed at four sites in Australia and New Zealand with a target enrollment of 12-15 patients. In addition to assessing safety, AzurRx is evaluating four escalating dose increments of MS1819, with the aim of elucidating the optimal treatment dose to be taken into subsequent registration trials in EPI patients with chronic pancreatitis (CP) and cystic fibrosis (CF).  The primary efficacy endpoint is the change in CFA from baseline and measured at the end of the open label treatment part of the trial (Phase C) on standardized high-fat meals and stool collection for 3 days.  AzurRx continues to expect completion of this Phase IIa trial in 3Q17.   Information about the trial can be found on the following website:
http://www.anzctr.org.au/TrialSearch.aspx?searchTxt=ms1819&isBasic=True.

About MS1819, a Recombinant Lipase

MS1819, a recombinant lipase derived from the yeast Yarrowia Lipolytica, is in development for the treatment of exocrine pancreatic insufficiency (EPI) associated with chronic pancreatitis (CP) and cystic fibrosis (CF).  Early Phase Ib data in EPI patients treated with MS1819 showed a favorable safety profile and encouraging preliminary signals of efficacy.

There are approximately 100,000 patients in the U.S. with EPI caused by CP according to the National Pancreas Foundation and more than 30,000 patients with EPI caused by CF according to the Cystic Fibrosis Foundation. Patients are currently treated with porcine pancreatic enzyme replacement pills. The U.S. market for such EPI porcine replacement therapy pills in 2016 was estimated to be approximately $950 million in the U.S. and $1.5 billion globally according to IMS data and Wall Street estimates.