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 in its Phase 2 trial evaluating MS1819 in combination
with the current standard of care, porcine-derived pancreatic
enzyme replacement therapy (PERT), for the treatment of severe
exocrine pancreatic insufficiency (EPI) in patients with cystic
fibrosis (CF).
“We are very pleased to have completed enrollment in our Phase 2
MS1819 plus PERT combination therapy clinical trial,” said James
Sapirstein, President, CEO and Chairman of AzurRx. “Based on the
very encouraging clinical results to date, we believe that the
combination therapy has significant potential to help the 25-30% of
refractory cystic fibrosis patients with severe EPI who are unable
to achieve adequate nutrition using PERT alone. Adding a small dose
of MS1819 can help these patients meet their nutritional needs,
reduce the debilitating symptoms of EPI and improve their overall
quality of life, with an increased safety profile.”
Mr. Sapirstein continued, “The combination therapy program
represents a second therapeutic opportunity for the MS1819 program.
We reported the completion of enrollment of our Phase 2 monotherapy
program – OPTION 2 – last week and will announce topline results
for that trial at the end of March. We continue to remain on target
to report topline results from the combination therapy study during
the second quarter of 2021.”
“The overarching goal of our MS1819 program is to provide a safe
and effective therapy to control EPI, a debilitating
gastrointestinal condition common to patients with cystic fibrosis
that can result in numerous, life-altering complications, including
malnutrition,” added Dr. James Pennington, Chief Medical Officer of
AzurRx. “Early clinical evidence has been promising, and with the
combination and OPTION 2 monotherapy trials progressing, we may
soon have the opportunity to introduce a drug product that could
potentially improve the lives of thousands of patients suffering
from cystic fibrosis.”
The Phase 2 combination clinical trial of MS1819 is a
multi-center study designed to investigate the safety, tolerability
and efficacy of escalating doses of MS1819, in conjunction with a
stable dose of PERT in order to increase the patient’s coefficient
of fat absorption (CFA) levels and relieve abdominal symptoms. The
study enrolled 18 patients, 12 years of age or older, with severe
EPI. Patients enrolled in the study receive escalating doses of
700mg, 1200mg, and 2240mg of MS1819 once daily for 15 days per
dosing level, in addition to their standard PERT dose. Baseline CFA
is established by measuring CFA levels while on standard of care
therapy only, before beginning combination therapy. Eligibility
requires a CFA of less than 80%. The primary efficacy endpoint of
the trial is improvement in CFA; secondary endpoints of the study
are improvements in the stool weight, stool consistency, number of
bowel movements, the incidence of steatorrhea, and increase of body
weight.
Previously reported results from the initial five patients in
the trial revealed that the combination therapy led to clinically
meaningful improvements in the primary efficacy endpoint, the CFA,
and improvements in the key secondary endpoints, including body
weight, stool consistency and reductions in the number of bowel
movements and the incidence of steatorrhea (excessive amounts of
fat in the feces).
Additional information about this clinical trial can be found
at: https://clinicaltrials.gov/ct2/show/NCT04302662
About the MS1819 Combination Therapy StudyThe
digestive standard of care for both CF and chronic pancreatitis
(CP) patients with EPI are commercially-available PERTs. Ideally, a
stable daily dose of PERT will enable CF patients to eat a normal
to high-fat diet and minimize unpleasant gastrointestinal symptoms.
In practice, however, a substantial number of CF patients do not
achieve normal absorption of fat with PERTs1,2. Achieving an
optimal nutritional status, including normal fat absorption levels,
in CF patients is important for maintaining better pulmonary
function, physical performance and prolonging survival.
Furthermore, a decline of body mass index around the age of 18
years predicts a substantial drop in lung function3,4.
A combination therapy of PERT and MS1819 has the potential to:
(i) correct macronutrient and micronutrient maldigestion; (ii)
eliminate abdominal symptoms attributable to maldigestion; and
(iii) sustain optimal nutritional status on a normal diet in CF
patients with severe EPI. Planned enrollment is expected to include
approximately 20 CF patients with severe EPI, with study completion
anticipated in 1H 2021.
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
1 Freedman, S.D., Am. J. Manag. Care, 2017; 23: S2220-S2282
Littlewood, J. et al, 2006, Pediatric Pulmonology, 2006, 41:35-493
Engelen, M. et al, 2014, Curr. Opin. Clin. Nutr. Metab. Care;
17(6):515-5204 Vandenbranden, S.L. et al, 2012, Pediatric
Pulmonology, 2012; 47(2): 135-143
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