AzurRx BioPharma Announces Completion of Enrollment in Phase 2b OPTION 2 Clinical Trial of MS1819 in Cystic Fibrosis
March 16 2021 - 7:00AM
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 announced it has completed
enrollment for its Phase 2b OPTION 2 clinical trial to investigate
MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic
insufficiency (EPI). The trial enrolled 30 CF patients and top line
data from the study is anticipated by the end of Q1 2021.
The Phase 2b multi-center study is designed 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. The primary efficacy
endpoint is 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 includes
an extension arm that uses 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.
“Completion of enrollment in our Phase 2b OPTION 2 trial is an
important milestone in the clinical development of MS1819 as an EPI
monotherapy for patients with cystic fibrosis,” said James
Sapirstein, CEO, President and Chairman of AzurRx. “Data from this
trial will inform the optimal dose for a potential pivotal Phase 3
study, and we are on target to report topline results from this
study at the end of the first quarter of 2021.”
Dr. James Pennington, Chief Medical Officer of AzurRx, added,
“The overarching goal of our MS1918 program is to provide a safe
and effective therapy to control EPI and improve upon the current
standard of care, PERT, which is porcine-derived and requires
patients to take upwards of 40 capsules per day to control
symptoms. MS1819 is a synthetic lipase that does not contain any
animal products and offers the potential to substantially reduce
the daily pill burden currently required to manage
EPI.”
Additional information about this clinical trial can be found
at: https://www.clinicaltrials.gov/ct2/show/NCT04375878
Phase 2 OPTION 2 Trial DesignThe Phase 2b
OPTION 2 multi-center trial is 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 is 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 are enrolled. MS1819 will be
administered in enteric capsules to provide gastric protection and
allow optimal delivery of enzyme to the duodenum. Patients will
first be randomized into two cohorts: to either the MS1819 arm,
where they receive a 2.2 gram daily oral dose of MS1819 for three
weeks; or to the PERT arm, where they receive their pre-study dose
of PERT pills for three weeks. After three weeks, stools will be
collected for analysis of coefficient of fat absorption. Patients
will then be crossed over for another three weeks of the
alternative treatment. After three weeks of cross-over therapy,
stools will again be collected for analysis of CFA. A parallel
group of patients will be randomized and studied in the same
fashion, using a 4.4 gram daily dose of MS1819. All patients will
be followed for an additional two weeks after completing both
crossover treatments for post study safety observation. Patients
will be 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 clinical
trials. AzurRx is launching two clinical programs using proprietary
formulations of niclosamide, a pro-inflammatory pathway
inhibitor, FW-420, for grade 1 Immune Checkpoint Inhibitor
Colitis and diarrhea in oncology patients and FW-1022, for
COVID-19 gastrointestinal infections. 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
AzurRx BioPharma (NASDAQ:AZRX)
Historical Stock Chart
From Aug 2024 to Sep 2024
AzurRx BioPharma (NASDAQ:AZRX)
Historical Stock Chart
From Sep 2023 to Sep 2024