Full Press Release Details
AzurRx BioPharma Announces Positive Results from
Phase 2 Trial with MS1819 in Cystic Fibrosis
results seen in CF patients, with CFA in line with previous studies
and no need for a protease
to discuss the results on a call scheduled for today, September 25,
2019 at 8:30 a.m. Eastern Time
NEW YORK, Sept. 25, 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
announced positive safety results from its Phase II OPTION Clinical
Trial of MS1819 for the treatment of exocrine pancreatic
insufficiency in cystic fibrosis (CF).
showed that the primary efficacy endpoint of coefficient of fat
absorption (CFA) was comparable to the CFA in a prior phase 2 study
in patients with chronic pancreatitis, while using the same dose of
MS1819. The dose used in both studies was 2 gram/day, which was
selected in agreement with FDA as a bridging dose. Although the study was not powered for statistical
significance, the data also demonstrated meaningful efficacy
results, with approximately 50% of the patients showing CFAs high
enough to reach non-inferiority with porcine enzyme replacement
coefficient of nitrogen absorption (CNA) was comparable between the
MS1819 and PERT arms, 93% vs. 97%, respectively, in the OPTION
trial. This important finding confirms that protease
supplementation is not likely to be required with MS1819
We are thrilled to have seen such favorable safety and
meaningful efficacy data in this Phase II study. Importantly, the
data were consistent and confirm results seen in prior clinical
studies. We are grateful to all the investigators who worked
diligently to bring this study to completion on time and also want
to thank the patients and their caregivers for taking the time to
participate in the trial, said
Jim Pennington, M.D., Chief Medical Officer of AzurRx.
We are eager to move forward with what we consider a
logical and promising next trial to increase the dose for CF
search for a non-porcine based pancreatic enzyme replacement for
patients with cystic fibrosis has been a challenging but important
endeavor, said Dr. Michael Konstan, Professor of Pediatrics
at Case Western Reserve University School of Medicine, and
principal investigator in the OPTION trial. With these data
showing MS1819 to be safe and to have the potential to support fat
absorption, we have considerable reason to be optimistic for the
next steps in non-porcine enzyme
Thijs Spoor, Chief Executive Officer of AzurRx stated, We
look forward to meeting with the FDA to discuss these results and
our next steps. Clearly, these data are exciting for patients, who
currently face debilitating symptoms, and we look to advancing to
market a therapy that has the potential to improve the quality of
life in these patients.
Phase II OPTION Study Design
Phase II OPTION trial was an open-label, crossover study, conducted
in 14 sites in the U.S. and Europe. Patients were first randomized
to either the MS1819 arm, where they received a 2 gram daily oral
dose of MS1819 for three weeks; or to the PERT arm, where they
received their pre-study dose of PERT for three weeks. After three
weeks, stools were collected for analysis, and patients were then
crossed over to another three weeks of the alternative treatment.
After three weeks of cross-over therapy, stools were collected
again for analysis. Patients were then followed for an additional
two weeks for post study safety observation. A total of 32
patients, ages 18 years or older, completed the study. Topline data
compared the CFA of the MS1819 treatment phase, 56%, to the CFA of
the PERT treatment phase, 86%. In addition, CNAs were 93% in the
MS1819 group, compared to 97% in the PERT group. Of note, MS1819
contains no protease. Safety in the OPTION trial was excellent,
with no severe adverse events (SAEs) and few overall adverse
upon prior communications with FDA, the Company is planning to meet
with the agency before year-end to discuss a Phase IIb/III trial
design exploring the use of higher doses and/or enteric-coated
capsules to ensure higher MS1819 activity in the
MS1819-SD, 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 products.
About Exocrine Pancreatic Insufficiency
Exocrine Pancreatic Insufficiency (EPI) is 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
There 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 CF
according to the Cystic Fibrosis Foundation. Patients are currently
treated with porcine pancreatic enzyme replacement
Conference Call and Webcast Information
Company management will discuss the Phase II OPTION clinical
results on a conference call, today, Wednesday, September 25, 2019
at 8:30 am Eastern Time. To participate in the call, dial
877-407-0784 (domestic) or 201-689-8560 (international) fifteen
minutes before the conference call begins and reference the
conference passcode 13694778. The live conference call can be
accessed via audio webcast at http://public.viavid.com/index.php?id=136209.
A replay of the call will be available on the
Investor Relations section of the
About AzurRx BioPharma, Inc.
AzurRx BioPharma, Inc. (NASDAQ:AZRX) is engaged in the research and
development of non-systemic biologics for the treatment of patients
with gastrointestinal disorders. MS1819-SD recombinant lipase for