Full Press Release Details
AzurRx BioPharma Announces First Patients Dosed in Phase II OPTION
Initial top line data of MS1819-SD in Cystic Fibrosis
expected in mid-2019.
First patients dosed in open-label, cross-over Phase II OPTION
study with MS1819-SD for exocrine pancreatic insufficiency in
Five clinical trial sites in the U.S. activated for OPTION
Additional European sites now expected with Ministry of Health
YORK, February 20, 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 provided a clinical development and strategic corporate
Clinical Development
Company announced that it has dosed the first patients in the
Company's Phase II OPTION study to investigate MS1819-SD in cystic
fibrosis (CF) patients with exocrine pancreatic insufficiency
Phase II multi-center study is designed to investigate the safety,
tolerability and efficacy of MS1819-SD in a head-to-head comparison
against the current porcine enzyme replacement therapy standard of
care. Planned enrollment is expected to include approximately
30 CF patients, with the results expected in mid-2019.
highlights of OPTION include:
is a six-week non-inferiority, coefficient of fat absorption (CFA)
assessment comparing MS1819-SD to the standard of care porcine
pancreatic enzyme replacement therapy (PERT) in patients with
exocrine pancreatic insufficiency due to cystic
design leverages input from the U.S. Food and Drug Administration
(FDA) and the CF community;
of approximately 30 patients 18 years of age and older;
CF community has long recognized the unmet need for a non-porcine
based pancreatic enzyme alternative to existing therapies for
treating EPI in CF , stated Michael W. Konstan, M.D., Vice
Dean for Translational Research and Professor of Pediatrics and of
Population & Quantitative Health Sciences at Case Western
Reserve University School of Medicine.
Konstan, the overall PI for the study, further added I am
thrilled to hear that the OPTION trial is enrolling subjects, and
look forward to seeing the results of the study later this
James Pennington, Chief Medical Officer of AzurRx, added, We
would like to thank the patients and our clinical collaborators and
their staffs for their enthusiasm, energy and willingness to work
with us in insuring that the OPTION study is successfully
completed. This is truly a team effort and we're greatly
appreciative of everyone's dedication and
recently announced, in a Phase II trial in the chronic pancreatitis
setting, 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 in the highest studied dose (per protocol).
Spoor, Chief Executive Officer of AzurRx, added, Having the
first patients dosed in our OPTION study of MS1819-SD in patients
with CF represents a significant milestone for AzurRx and
demonstrates that we are on track to complete our Phase 2 study by
mid-2019 as previously announced.
OPTION Study now enrolling patients at 5 sites in the U.S., Expands
for the OPTION study are currently being enrolled at five clinical
trial centers in the U.S., with up to six additional U.S. sites
initiating in the near future.
Company has received approval from the Polish Ministry of Health to
expand enrollment to include European patients, with the first
European clinical trial sites being allowed to enroll
Company also announced that it has raised $2 million from the sale
of a convertible note to its largest shareholder, convertible at
$2.50 per share. These additional funds allow the company to
continue to execute its operational plan, added Mr.
OPTION trial is a Phase II, open-label, multicenter, 2x2 crossover
study assessing the safety and efficacy of MS1819-SD versus porcine
PERT given at the same dose that was being administered during the
pre-study period. Approximately 30 evaluable patients will
complete both crossover periods. The primary efficacy
endpoint will be a comparison of CFAs after each of the two
information about the ongoing OPTION MS1819-SD can be found at
supplied as an oral non-systemic biologic capsule, is a recombinant
enzyme that is derived from the yarrowia lipolytica lipase, and
unlike the standard of care, does not contain any animal
About Exocrine Pancreatic Insufficiency:
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
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.
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
Forward-Looking Statements
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