Full Press Release Details
AzurRx BioPharma Announces Positive CFF DSMB Review of Final Phase
Company plan to proceed to higher 4-gram dose of MS1819-SD in next
Phase 2 clinical trial
concerns cited for MS1819-SD
NEW YORK, October 17, 2019 (GLOBE NEWSWIRE) -- AzurRx BioPharma,
( AzurRx or the Company ), a company
specializing in the development of non-systemic,
recombinant therapies for gastrointestinal diseases,
today announced that the Cystic Fibrosis Foundation (CFF) Data
Safety Monitoring Board (DSMB) has completed its review of the
Company's final results of the Phase 2 OPTION trial and has
found no safety concerns for MS1819-SD. Additionally, the group
supports the Company's plan to proceed to a higher 4-gram
dose of MS1819-SD in its next planned Phase 2 clinical
We are very pleased with the support of the CFF DSMB to
proceed with a new study using a higher 4-gram dose of MS1819-SD.
The safety profile of MS1819 at the 2-gram dose in both the Phase 2
study in chronic pancreatitis and Phase 2 OPTION trial in cystic
fibrosis has been excellent, with no reported serious adverse
events, said Dr. James Pennington, Chief Medical Officer of
AzurRx. We thank the DSMB for its diligence, and guidance
and support of our development plan as we move forward with this
paradigm changing therapy.
James Sapirstein, CEO of AzurRx added, The positive safety
review of the CFF DSMB is an important milestone for the
development of MS1819-SD as a monotherapy and for the AzurRx team.
We look forward to our planned meeting with the FDA in December and
to initiating our next Phase 2 study of MS1819-SD at a higher dose
The CFF DSMBis an independent
committee of experts in cystic fibrosis (CF) clinical care,
clinical and basic science research, bioethics, and biostatistics,
charged with protecting the safety and welfare of subjects
participating in clinical trials sanctioned by the Cystic Fibrosis
Foundation's Therapeutics Development Network (TDN) and with
ensuring the scientific integrity of those
Phase 2 OPTION Study Design
The Phase 2 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-SD arm, where they received a 2 gram daily oral dose of
MS1819-SD for three weeks; or to the porcine enzyme replacement
therapy (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-SD treatment phase,
56%, to the CFA of the PERT treatment phase, 86%. In
addition, CNAs were 93% in the MS1819-SD group, compared to 97% in
the PERT group. Of note, MS1819-SD contains no
protease. Safety in the OPTION trial was excellent, with no
severe adverse events (SAEs) and few overall adverse
MS1819-SD, supplied as an oral non-systemic biologic capsule, is a
recombinant enzyme that is derived from
lipolytica lipase, and
unlike the standard of care, does not contain any animal
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
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
EPI is the Company's lead development program, and additional early
stage research is being conducted for the prevention of
hospital-acquired infections. The Company is headquartered in
Brooklyn, NY, with scientific operations based in Langlade,
France. Additional information on the Company can be found
Forward-Looking Statements
This press release may contain certain statements relating to
future results which are forward-looking statements. These
statements are not historical facts, but instead represent only the
Company's belief regarding future events, many of which, by
their nature, are inherently uncertain and outside of the
Company's control. It is possible that the Company's
actual results and financial condition may differ, possibly
materially, from the anticipated results and financial condition
indicated in these forward-looking statements. Additional
information concerning the Company and its business, including a
discussion of factors that could materially affect the
Company's financial results, including those related to the
clinical development of MS1819-SD and final results of the Phase 2
OPTION study, are contained in the Company's Annual Report on
Form 10-K for the year ended December 31, 2018 under the
heading Risk Factors, as well as the
Company's subsequent filings with the Securities and Exchange
Commission. All forward-looking statements included in this press
release are made only as of the date of this press release, and we
do not undertake any obligation to publicly update or correct any