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 investigation.
OPTION 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.
Discussing 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 our trial goal.”
Mr. 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.”
“To 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 digested.”
Mr. 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.
We 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.”
An 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 days.
Phase 2 OPTION 2 Trial
DesignThe 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, and for safety.
About MS1819MS1819 is a
recombinant lipase enzyme for the treatment of exocrine pancreatic
insufficiency associated with cystic fibrosis and chronic
pancreatitis. MS1819, supplied as an oral non-systemic biologic
capsule, is derived from the Yarrowia lipolytica yeast lipase and
breaks up fat molecules in the digestive tract of EPI patients so
that they can be absorbed as nutrients. Unlike the standard of
care, the MS1819 synthetic lipase does not contain any animal
products.
About Exocrine Pancreatic
InsufficiencyEPI is a condition characterized by
deficiency of the exocrine pancreatic enzymes, resulting in a
patient’s inability to digest food properly, or maldigestion. The
deficiency in this enzyme can be responsible for greasy diarrhea,
fecal urge and weight loss.
There are more than 30,000 patients in the U.S.
with EPI caused by cystic fibrosis according to the Cystic Fibrosis
Foundation and approximately 90,000 patients in the U.S with EPI
caused by chronic pancreatitis according to the National Pancreas
Foundation. Patients are currently treated with porcine pancreatic
enzyme replacement pills.
About AzurRx BioPharma,
Inc.AzurRx BioPharma, Inc. (NASDAQ: AZRX) is a clinical
stage biopharmaceutical company specializing in the development of
targeted, non-systemic therapies for gastrointestinal (GI)
diseases. The Company has a pipeline of three gut-restricted GI
assets. The lead therapeutic candidate is MS1819, a recombinant
lipase for the treatment of exocrine pancreatic insufficiency (EPI)
in patients with cystic fibrosis and chronic pancreatitis,
currently in two Phase 2 CF clinical trials. AzurRx is launching
two clinical programs using proprietary formulations of
niclosamide, a pro-inflammatory pathway inhibitor; FW-1022, for
COVID-19 gastrointestinal infections, and FW-420, for grade 1
Immune Checkpoint Inhibitor-Associated Colitis and diarrhea in
oncology patients. The Company is headquartered in Delray Beach,
Florida with clinical operations in Hayward, California. For more
information visit www.azurrx.com.
Forward-Looking StatementThis
press release may contain certain statements relating to future
results which are forward-looking statements. 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, depending
on factors including whether results obtained in preclinical and
nonclinical studies and clinical trials will be indicative of
results obtained in future clinical trials; whether preliminary or
interim results from a clinical trial will be indicative of the
final results of the trial; and the impact of the coronavirus
(COVID-19) pandemic on the Company’s operations and current and
planned clinical trials, including potential delays in clinical
trial recruitment and participation. Additional information
concerning the Company and its business, including a discussion of
factors that could materially affect the Company’s financial
results are contained in the Company’s Annual Report on Form 10-K
for the year ended December 31, 2019 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 forward-looking statements to reflect events
or circumstances that subsequently occur or of which we hereafter
become aware.
For more information:
AzurRx BioPharma, Inc.1615 South Congress AvenueSuite 103Delray
Beach, Florida 33445Phone: (646) 699-7855info@azurrx.com
Media contact:
Tiberend Strategic Advisors, Inc.Johanna Bennett/Ingrid
Mezo(212) 375-2665/(646)
604-5150jbennett@tiberend.com/imezo@tiberend.com
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