Full Press Release Details
AzurRx BioPharma Announces Positive Interim Data From First Patient
Cohort in Phase 2 Clinical Trial of MS1819 in Combination with PERT
Therapy in the Treatment of Cystic Fibrosis Patients with Severe
Exocrine Pancreatic Insufficiency
Data from first five patients demonstrate positive results with
primary efficacy and secondary safety endpoints
Clinically meaningful improvement (>80%) in coefficient of fat
absorption (CFA) for all patients across all dose levels of
Topline data from full dataset anticipated in 1Q 2021
Management to discuss the interim results on call scheduled for
today, August 11, 2020, at 4:30 p.m. Eastern Time
YORK, August 11, 2020 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc.
(NASDAQ:AZRX) ( AzurRx or the
company specializing in the development of non-systemic,
recombinant therapies for gastrointestinal diseases,
today announced positive results from the first five
patients in its Phase 2 combination therapy trial of MS1819 for the
treatment of severe exocrine pancreatic insufficiency (EPI) in
patients with cystic fibrosis (CF).
primary efficacy analysis, patients receiving MS1819 in combination
with PERT therapy achieved a clinically meaningful improvement in
the coefficient of fat absorption (CFA) at each of the three dose
levels, with no adverse safety events reported. Additionally,
patients showed improvements in the key secondary endpoints,
including body weight, stool consistency and reductions in the
number of bowel movements and the incidence of
Results from the initial five patients in the trial show an
improvement in CFA above 80% (the FDA's measure for adequate
nutrition) across all dose levels and on all visits. Mean CFA at
the 700mg/day, 1200 mg/day and 2240 mg/day doses of MS1819 plus
standard of care were 88.4%, 87.2% and 86.5% respectively, compared
to baseline level (78.4%). This represents an average increase of 9
points in CFA during the trial.
are thrilled to see such consistently positive responses from these
initial patients in our ongoing Phase 2 combination therapy trial
with MS1819. EPI is a very challenging disease with many CF
patients unable to achieve healthy nutrition even when using the
maximum number of allowed or tolerated PERT capsules, especially
those in the moderate-severe categories, said James Sapirstein, Chief Executive
Officer of AzurRx. We look forward to completing treatment
of all patients and announcing top line data in 2021.
Dr. James Pennington, Chief Medical Officer of AzurRx added,
To see these results in the first five patients is quite
compelling. With its safety and efficacy profile, MS1819 has the
potential to meaningfully improve the quality of life for many
patients with severe EPI. The pill burden of PERT therapy alone is
quite challenging for most patients, and for severe EPI patients
the symptoms of the disease can be debilitating. We believe
that a small daily dose of MS1819, when added to their daily dose
of PERT, has the potential to safely help CF patients meet their
nutritional needs, decrease abdominal pain and alleviate multiple
morbidities caused by severe EPI. We are excited to continue developing
this exciting therapy and are committed to bringing this therapy to
Phase 2 clinical trial of MS1819 in combination with PERT therapy
in the treatment of CF patients with severe exocrine pancreatic
insufficiency 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 PERTs in order to increase the
patient's CFA levels and relieve abdominal symptoms. The
study is expected to enroll 24 patients, 12 years of age or older,
with severe EPI, with the goal of having at least 20 patients
completing. Patients enrolled in the study receive escalating doses
of 700mg, 1200mg, and 2240mg of MS1819 once a day for 15 days per
dosing level, in addition to their standard PERT therapy. 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.
information about the ongoing Combination Therapy study can be
Conference Call and Webcast Information
The management team will host a conference call to discuss results
from the five patients in the Phase 2 study of MS1819 in
combination with PERT therapy, today, Tuesday, August 11, 2020 at
4:30 pm Eastern Time. To participate in the call, dial
877-407-0784 (domestic) or 201-689-8560 (international) fifteen
minutes before the conference call begins and reference the
conference passcode 13707811. The live conference call can be
accessed via audio webcast at http://public.viavid.com/index.php?id=141064.
A replay of the call will be
available on the Investor
of the Company's website
About the MS1819 Combination Therapy Study
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 PERTs(1,2). 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 function(3,4).
combination therapy of PERT and MS1819 has the potential to: (i)
correct macronutrient and micronutrient maldigestion; (ii)
eliminate abdominal symptoms attributable to maldigestion; and