Full Press Release Details
BioPharma Presents Positive Secondary Endpoint Data in its Phase II
MS1819-SD Chronic Pancreatitis Study at the 2019 Digestive Disease
significance achieved on multiple secondary endpoints
underscore the potential of MS1819 to provide meaningful clinical
benefits to patients suffering from exocrine pancreatic
YORK, May 20, 2019 (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 released new data for its
lead development candidate MS1819-SD at the 2019 Digestive Disease Week (DDW)
presentation entitled Results from a Phase II Study of
MS1819, a Recombinant Lipase for the Treatment of Exocrine
Pancreatic Insufficiency in Patients with Chronic
Pancreatitis was delivered by Dr. Nam Q. Nguyen, et al., on
addition to previously released coefficient of fat absorption (CFA)
results and safety assessments, Dr. Nguyen discussed newly released
positive secondary endpoint data from the study.
Summary of Key Secondary Endpoints Analysis
highest dose of 2,240 mg/day used in the study, statistical
significance was achieved with the following
Improvement in stool consistency
consistency on the Bristol scale decreased by 20%, from a baseline
of 5.1 to 4.1, with a p value of <0.001.
Reduction in the number of bowel movements
daily evacuations decreased by 32%, from a baseline of 2.8 to 1.9
per day, with a p value of 0.006.
Reduction in the incidence of steatorrhea
of steatorrhea, the presence of excess fat in feces, decreased by
18%, from a baseline of 12.3 to 10.1, with a p value of
was a trend in the reduction of abdominal discomfort on the visual
analogue scale (VAS), recorded from a mean of 21.0 during baseline
to 14.5 at the highest dose of 2,240 mg, at a p value of
regard to CFA, it was observed that improvements were most
pronounced in patients with baseline CFA of less than
| Baseline | @ Highest Dose of MS1819-SD (2240 mg) | Mean Change | p value | |
| Coefficient of Fat Absorption (CFA)* | 21.8% | 0.002 | ||
| Stool Consistency (on Bristol Scale) | 5.1 | 4.1 | -20% | 0.006 |
| Bowel Movements | 2.8 | 1.9 | -32% | 0.006 |
| Steatorrhea | 12.3 | 10.1 | -18% | 0.008 |
| Abdominal Discomfort (on VAS) | 21.0 | 14.5 | -31% | 0.148 |
*Per protocol analysis
Nguyen, an investigator in the study and the Head of Education and
Research, Interventional & EUS endoscopist, at the Royal
Adelaide Hospital in Australia commented that the efficacy
results of the MS1819-SD study point to the potential of a novel
and non-porcine based recombinant therapy to safely and effectively
improve the nutritional status and ease of digestion of CP patients
with EPI and markedly reduce associated
new MS1819-SD secondary endpoint data presented at DDW have both
statistical and clinical significance for the treatment of EPI in
chronic pancreatitis patients, said Thijs Spoor, CEO of
AzurRx. The study has demonstrated that MS1819-SD has a
favorable safety profile, as demonstrated by the primary endpoint
outcome, and efficacy in increasing the coefficient of fat
absorption, improving stool consistency, reducing the number of
bowel movements and decreasing the incidence of fatty stools as
secondary endpoints. These results underscore MS1819's
potential to provide meaningful clinical benefits on several fronts
to patients suffering from EPI.
About Phase 2 MS1819-SD in Chronic Pancreatitis
Company previously reported the completion of this open-label,
multi-center, dose escalation Phase IIa study, whose primary
endpoint was to evaluate the safety of escalating doses of
MS1819-SD in patients with chronic pancreatitis. The secondary
endpoint for the study was to investigate the efficacy of MS1819-SD
in these patients by analysis of the CFA and its change from
baseline. The Company enrolled 11 chronic pancreatitis
patients in France, Australia and New Zealand. During the course of
the trial, patients washed-out of their standard of
care treatment for EPI to establish a baseline and then were
subsequently treated with escalating doses of study drug in
two-week increments.
data from the Phase IIa study show a favorable safety profile with
no severe adverse events. Although the study was not powered for
efficacy, in a pre-planned analysis, the highest dose cohort of
MS1819-SD showed statistically significant and clinically
meaningful increases in CFA compared to baseline with a mean
increase of 21.8% and a p value of p=0.002 on a per protocol
Phase II OPTION Study of MS1819-SD in Cystic
Company announced that it continues to dose patients in the
Company's Phase II OPTION study to investigate MS1819-SD in cystic
fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI)
with the 50% enrollment mark having been reached several weeks
Phase II, open-label, multi-center, 2x2 crossover study is designed
to investigate the safety, tolerability and efficacy of MS1819-SD
in a head-to-head comparison against the current porcine enzyme
replacement therapy (PERT) standard of care. The primary efficacy
endpoint will be a comparison of CFAs after each of the two
crossover periods. Planned enrollment is expected to include
approximately 30 CF patients, with the results expected in the
information about the ongoing OPTION Study can be found
supplied as an oral non-systemic biologic capsule, is a recombinant
enzyme that is derived from the yarrowia lipolytica lipase, and
unlike the standard of care, does not contain any animal
About Exocrine Pancreatic Insufficiency:
a condition characterized by deficiency of the
exocrine pancreatic enzymes, resulting in the inability
to digest food properly, or maldigestion. This deficiency can be
responsible for greasy diarrhea, fecal urge and weight
are approximately 90,000 patients in the U.S. with EPI caused by
chronic pancreatitis according to the National Pancreas Foundation,
and more than 30,000 patients with EPI caused by cystic fibrosis
according to the Cystic Fibrosis Foundation. Patients are currently
treated with porcine pancreatic enzyme replacement