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 positive interim
data from the first 18 out of 20 patients 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).
The interim topline data revealed that the combination therapy
led to clinically meaningful improvements in the primary efficacy
endpoint, the Coefficient of Fat Absorption (CFA), with an average
gain of 5.9 points from baseline. According to the clinical
literature, a five-point improvement in CFA is considered
clinically significant.1
“We are encouraged by the promising interim data from the first
18 patients in our Phase 2 clinical trial, evaluating MS1819 in
combination with PERT for the treatment of EPI in cystic fibrosis
patients,” said James Sapirstein, President, CEO and Chairman of
AzurRx. “Based on the 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 MS1819 to
the treatment regimen appears to enable these patients to meet
their nutritional needs, reduce the debilitating symptoms of EPI,
and improve their overall quality of life, with an acceptable
safety profile. We expect to report full topline results from all
20 patients enrolled in the trial in the second quarter of
2021.”
Dr. Bulent Karadag, the principal investigator in the trial from
the Marmara University Faculty School of Medicine in Istanbul,
Turkey, stated, "Many of my patients suffer from severe EPI and a
number are not adequately controlled. They frequently reported
that they felt better after taking MS1819 over the course of the
six-week trial."
Dr. James Pennington, Chief Medical Officer of AzurRx,
commented, “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. Based on the clinical evidence from the combination
trial to-date, we have been able to improve the nutritional status
of the severe EPI patients who need the most help. It turns out
that the patients with the lowest baseline CFAs had the largest
improvements in CFA in the study. It is also very encouraging to
note that all the patients responded well and that many reported
that they felt better overall when MS1819 was added to their daily
dose of PERT.”
The Phase 2 combination clinical trial 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 20 patients, 12 years of age or older, with severe EPI who
were treated escalating doses of MS1819 (700mg, 1200mg, and 2240mg)
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. Trial 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.
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 24 CF patients with severe EPI, with study completion
anticipated in 1Q 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; the size of the potential markets for the Company’s drug
candidates and its ability to service those markets; and the
Company’s current and future capital requirements and its ability
to raise additional funds to satisfy its capital needs. 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, 2020 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 Brady, M.S et al, 2006, Journal of American Dietetic
Association, 2006, 1181-1185.2 Freedman, S.D., Am. J. Manag. Care,
2017; 23: S2220-S2283 Littlewood, J. et al, 2006, Pediatric
Pulmonology, 41:35-494 Engelen, M. et al, 2014, Curr. Opin. Clin.
Nutr. Metab. Care; 17(6):515-5205 Vandenbranden, S.L. et al, 2012,
Pediatric Pulmonology, 2012; 47(2): 135-143
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