Full Press Release Details
AzurRx BioPharma Provides Key Takeaways from Phase 2b OPTION 2
Clinical Trial Topline Results Conference Call
BEACH, Fla., March 31, 2021 (GLOBE NEWSWIRE) -- AzurRx BioPharma,
Inc. (NASDAQ: AZRX),
( AzurRx or the Company ), a clinical
stage biopharmaceutical company specializing in the development of
targeted, non-systemic therapies for gastrointestinal (GI)
diseases, today provided key takeaways from its conference call
reporting on the topline results from its Phase 2b OPTION 2
clinical trial investigating MS1819 in cystic fibrosis (CF)
patients with exocrine pancreatic insufficiency (EPI). The
conference call, held on March 31, 2021, at 4:30 p.m. ET, featured
James Sapirstein, President, CEO and Chairman of AzurRx, and Dr.
James Pennington, Chief Medical Officer, discussing the recently
completed OPTION 2 study, and the company's plans to develop
an optimized formulation of MS1819 for ongoing clinical
2 was designed as a Phase 2b multi-center study to investigate the
safety, tolerability and efficacy of MS1819 (in enteric capsules)
in a head-to-head comparison against the current porcine enzyme
replacement therapy (PERT) standard of care for the treatment of
exocrine pancreatic insufficiency (EPI) in patients with cystic
fibrosis. The primary efficacy endpoint was the coefficient of fat
absorption (CFA), with secondary endpoints of stool weight, signs
and symptoms of malabsorption and coefficient of nitrogen
absorption (CNA). The trial also included an extension arm that
used an immediate release MS1819 capsule, allowing the Company to
compare data from the existing arm that uses enteric (delayed
release) capsules with data from the new arm, and ultimately select
the optimal delivery method.
the topline results of OPTION 2 during the conference call, Mr.
Sapirstein commented, To summarize, the best word to
describe the OPTION 2 topline results is mixed. MS1819 demonstrated
itself to be safe and well-tolerated and data from OPTION 2, and
other Phase 2 clinical trials, clearly demonstrate drug activity.
However, OPTION 2 did not consistently meet the primary efficacy
endpoint. Some patients were able to achieve CFA at levels beyond
what is required to demonstrate non-inferiority with PERT
therapies, but the majority did not, and as such, we did not meet
Sapirstein continued, The underlying cause of the
drug's uneven efficacy performance in OPTION 2, we believe,
lies with the enteric capsule formulation. While the enteric
coating protects the capsule from breaking down in the stomach
acid, it also appears to dissolve too slowly in the small intestine
to release the lipase enzyme in time to aid with proper digestion
and nutrient absorption.
that end, we are planning to pursue a new formulation for MS1819,
this one a capsule filled with acid-resistant granules, or
microbeads, similar to what is used in CREON , ZENPEP
and other PERT therapies. Such a capsule would dissolve in the
stomach, disperse the beads, and then pass through to the small
intestine where the beads would break down and release the lipase
enzyme so that it thoroughly mixes with food as it is being
Sapirstein concluded, We are moving full force with
developing the optimal formulation technology for MS1819 and have
already initiated discussions with contract manufacturers to
accelerate the process. This will require additional time and
resources. Yet we are fortunate, through financing efforts
that have raised an aggregate of approximately $22.5 million
in the first quarter of 2021, to have sufficient
capital currently on hand to fund this development and, within
the next year or so, initiate a further Phase 2 study to evaluate
efficacy, without substantially delaying our clinical
development initiatives in other areas.
firmly believe the cost-benefit ratio with MS1819 is clearly in our
favor. The drug's mechanism of action is known and proven, it
offers numerous therapeutic, safety and compliance advantages over
today's standard of care, and remains less cumbersome to
manufacture. Based on these factors, we believe that should this
optimized formulation prove successful in the clinic and we
have every reason to believe it will MS1819 could
eventually become the gold standard treatment for EPI in patients
with cystic fibrosis and chronic pancreatitis.
audio webcast of the conference call will be accessible via the
Investors section of the AzurRx website at www.azurrx.com. An
archive of the webcast will remain available for approximately 90
Phase 2 OPTION 2 Trial Design
Phase 2b OPTION 2 multi-center trial was designed to investigate
the safety, tolerability and efficacy of MS1819 (2.2 and 4.4 gram
doses in enteric capsules) in a head-to-head comparison versus the
current standard of care, porcine pancreatic enzyme replacement
therapy pills. The OPTION 2 trial was an open-label, crossover
study, conducted in 15 sites in the U.S. and Europe. A total of 30
CF patients 18 years or older were enrolled. MS1819 was
administered in enteric capsules to provide gastric protection and
allow optimal delivery of enzyme to the duodenum. Patients were
first randomized into two cohorts: to either the MS1819 arm, where
they received a 2.2 gram daily oral dose of MS1819 for three weeks;
or to the PERT arm, where they received their pre-study dose of
PERT pills for three weeks. After three weeks, stools were
collected for analysis of coefficient of fat absorption. Patients
were then crossed over for another three weeks of the alternative
treatment. After three weeks of cross-over therapy, stools were
again collected for analysis of CFA. A parallel group of patients
were randomized and studied in the same fashion, using a 4.4 gram
daily dose of MS1819. All patients were followed for an additional
two weeks after completing both crossover treatments for post study
safety observation. Patients were assessed using descriptive
methods for efficacy, comparing CFA between MS1819 and PERT arms,
is a recombinant lipase enzyme for the treatment of exocrine
pancreatic insufficiency associated with cystic fibrosis and