Full Press Release Details
AzurRx BioPharma Announces Initiation of Phase 2 Clinical Study for
MS1819-SD in combination with standard PERT for Cystic Fibrosis
Patients with Severe Exocrine Pancreatic Insufficiency
Dose escalation study of MS1819-SD in combination with standard
porcine pancreatic enzyme replacement therapy (PERT) in CF patients
with severe exocrine pancreatic insufficiency (EPI).
Patients in study have persistent malnutrition and clinical
symptoms of fat malabsorption despite being on the maximum daily
First patient visits have been completed in Hungary.
Preliminary data expected in early 2020.
MS1819 R&D Update webcast call scheduled for Monday, July
8th at 4:30 p.m. EST
NEW YORK, July 8, 2019 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc.
(NASDAQ:AZRX) (AzurRx or the Company), a company specializing in
the development of non-systemic, recombinant therapies
for gastrointestinal diseases, today announced that it has
initiated a Phase 2 clinical trial to investigate MS1819-SD in
combination with standard porcine enzyme replacement therapy (PERT)
for patients with cystic fibrosis (CF) that suffer from severe
exocrine pancreatic insufficiency (EPI), but continue to experience
clinical symptoms of fat malabsorption
despite taking the maximum daily dose
The 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
The Phase 2 multi-center study is designed to investigate the
safety, tolerability and efficacy of escalating doses of MS1819-SD,
in conjunction with a stable dose of PERTs, in order to increase
the coefficient of fat absorption (CFA) and relieve abdominal
symptoms. A combination therapy of PERT and MS1819-SD 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 2020.
Thijs Spoor, Chief Executive Officer of AzurRx, commented,
The study of MS1819-SD in combination with PERT, the
existing standard of care for treating severe EPI in CF patients,
focuses on the clinical needs of the one-third of patients whose
nutritional needs cannot be met with PERT alone. Together with our
Phase 2 OPTION Study to investigate MS1819-SD as a replacement for
PERT, we are seeking to address the needs of CF patients by
exploring the efficacy of both MS1819-SD replacement and
combination therapies.
Dr. James Pennington, Chief Medical Officer of AzurRx, added,
The clinical nutritional needs of CF patients with severe
EPI have long been underserved. These patients cannot use higher
doses of commercially-available PERTs to alleviate these issues due
to the potential risk of fibrosing colonopathy. We are optimistic
that the planned study can provide these patients with a treatment
plan that utilizes a safe, non-porcine therapy to potentially
bolster the efficacy of existing PERTs.
The Company's Investor Relations firm, LifeSci Advisors, will
be hosting a MS1819 R&D update call with AzurRx's
management team on Monday, July 8th at 4:30 p.m. EST. Investors can
join the call using the following numbers and webcast
U.S. Investors: 877-407-0784
International Investors: 201-689-8560
Conference ID: 13692348
MS1819-SD, supplied as an oral non-systemic biologic capsule, is a
recombinant enzyme that is derived from
lipolytica lipase, and
unlike the PERT-based standard of care, does not contain any
In a Phase 2 trial of MS1819-SD in patients with CP, MS1819-SD
showed a favorable safety profile with good tolerability.
Additionally, a statistically significant (p=0.002) improvement in
the coefficient of fat absorption of 21.8% was observed the highest
studied dose (per protocol).
The Company is also currently conducting the OPTION Study, the
Company's Phase 2 multi-center study to investigate the
safety, tolerability and efficacy of MS1819-SD in a head-to-head
comparison against the current PERT standard of care for EPI in CF
patients. The Company recently announced the completion of
patient enrollment in the OPTION Study, with top-line results
expected late Summer 2019.
About Exocrine Pancreatic Insufficiency:
EPI is a condition characterized by deficiency of the
exocrine pancreatic enzymes, resulting in the inability
to digest food properly, or maldigestion. The deficiency in this
enzyme can be responsible for greasy diarrhea, fecal urge and
There are approximately 90,000 patients in the U.S. with EPI caused
by chronic pancreatitis according to the National Pancreas
Foundation and more than 30,000 patients with EPI caused by cystic
fibrosis according to the Cystic Fibrosis Foundation. Patients are
currently treated with porcine pancreatic enzyme replacement
AzurRx BioPharma, Inc.
AzurRx BioPharma, Inc. (NASDAQ:AZRX) is engaged in the research and
development of non-systemic biologics for the treatment of patients
with gastrointestinal disorders. MS1819-SD recombinant lipase for
EPI is the Company's lead development program, and additional early