Full Press Release Details
AzurRx BioPharma Announces First
Patients Dosed in Clinical Study for MS1819-SD in combination with
PERT for Cystic Fibrosis Patients with Severe Exocrine
Pancreatic Insufficiency
First cystic fibrosis (CF) patients with severe exocrine pancreatic
insufficiency (EPI) treated in dose escalation study of MS1819-SD
in combination with standard porcine pancreatic enzyme replacement
Patients in study have persistent malnutrition and clinical
symptoms of fat malabsorption despite being on the maximum daily
Approximately one-third of CF patients have severe EPI
Preliminary data expected in early 2020
YORK, October 15, 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,
that it has dosed the first patients in its Phase 2 clinical
trial to investigate MS1819-SD in combination with standard porcine
enzyme replacement therapy (PERT) for patients with cystic fibrosis
(CF) who suffer from severe exocrine pancreatic insufficiency
(EPI). This subset of CF patients experiences clinical symptoms of
fat malabsorption, despite taking the maximum daily dose
The Phase 2 multi-center study is designed to investigate the
safety, tolerability and efficacy of escalating doses of MS1819-SD
(700 mg, 1120 mg and 2240 mg per day, respectively), which is a
yeast-derived lipase, in conjunction with a stable dose of porcine
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.
We are very pleased to have dosed the first patients in our
combination therapy study for cystic fibrosis patients with severe
EPI, commented Dr. James Pennington, Chief Medical Officer
of AzurRx. Many patients with severe EPI are not gaining
adequate control of their symptoms despite receiving doses of PERT
which are at or near the maximum levels specified in the dosing
guidelines recommended by the Cystic Fibrosis Foundation. Adding a
synthetic lipase like MS1819 to PERT therapy has the potential to
address inadequately controlled malabsorption. We are encouraged by
the strong safety profile of MS1819, as demonstrated in both our
recent Phase 2 chronic pancreatitis and CF trials, and believe that
using MS1819 in combination with PERTs will improve fat absorption
and overall nutrition of CF patients, while decreasing the risk of
fibrosing colonopathy.
James Sapirstein, Chief Executive Officer of AzurRx, added,
The initiation of our combination study of MS1819-SD with
PERT in CF patients with severe EPI represents a significant
milestone for the Company. This study, together with the recent
completion of and top-line data read-out from our OPTION
monotherapy study, demonstrate AzurRx's commitment to finding
the best solutions for meeting the needs of CF patients with both
severe and mild-moderate EPI conditions.
supplied as an oral non-systemic biologic capsule, is a recombinant
enzyme that is derived from the yarrowia lipolytica lipase, and
unlike PERTS, which are the current standard of care, does not
contain any animal products.
About Exocrine Pancreatic
a condition characterized by deficiency of the
exocrine pancreatic enzymes, resulting in the inability
to digest food properly, or maldigestion. This deficiency can be
responsible for greasy diarrhea, fecal urge and weight
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 (~30%) 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
In many cases, patients with severe EPI cannot use higher
doses of commercially-available PERTs to alleviate these issues
because they are on doses that are at or near the maximum dose
recommended by the CFF guidelines. The concern for higher doses is
the risk of fibrosing colonopathy.
are approximately 90,000 patients in the U.S. with EPI caused by CP
according to the National Pancreas Foundation and more than 30,000
patients with EPI caused by CF according to the Cystic Fibrosis
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 stage
research is being conducted for the prevention of hospital-acquired
infections. The Company is headquartered in Brooklyn, NY, with
scientific operations based in Langlade, France. Additional
information on the Company can be found
This press release may contain certain statements relating to
future results which are forward-looking statements. These
statements are not historical facts, but instead represent only the
Company's belief regarding future events, many of which, by
their nature, are inherently uncertain and outside of the
Company's control. It is possible that the Company's
actual results and financial condition may differ, possibly