- New preclinical data support
investigation of new indication for EDP-305 in primary sclerosing
cholangitis (PSC)
Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a research and
development-focused biotechnology company dedicated to creating
small molecule drugs for viral infections and liver diseases, today
announced new data from its lead FXR agonist candidate EDP-305 for
NASH and PBC. This new data is being presented during The
International Liver Congress™ (ILC) 2017, April 19-23, in
Amsterdam.
Data from three poster presentations being presented at the
Congress will demonstrate that EDP-305 is a potent Farnesoid X
Receptor (FXR) agonist that has been shown to reduce expression of
fibrogenic genes, reduce fibrosis progression and improve
non-alcoholic fatty liver disease (NAFLD) activity scores (NAS) in
a variety of preclinical models.
NAFLD is the accumulation of excessive fat in the form of
triglycerides in patients’ liver cells (steatosis) that is not
caused by alcohol. NAFLD is widely considered to be the liver
expression of metabolic disease associated with type 2 diabetes,
insulin resistance, obesity, and hyperlipidemia. A subgroup of
NAFLD patients has liver cell injury and inflammation in addition
to excessive fat (steatohepatitis). Progression of this condition
leads to non-alcoholic steatohepatitis (NASH). Patients with NASH
can develop fibrosis and ultimately cirrhosis of the liver,
potentially leading to hepatocellular carcinoma or requiring a
liver transplant.
The first poster will be presented by Bryan C. Fuchs, Ph.D.,
Assistant Professor of Surgery, Harvard Medical School,
Massachusetts General Hospital. Poster #THU-377, titled “A Novel
Farnesoid X Receptor (FXR) Agonist, EDP-305, Reduces Fibrosis
Progression in Animal Models of Fibrosis”, demonstrates that
EDP-305 reduced fibrosis progression in a choline-deficient,
high-fat-diet mouse model of NASH and a rat model of PBC induced by
bile duct ligation. Fibrosis progression was measured by
quantitative molecular imaging of collagen crosslinking and Type 1
collagen, markers that are sensitive to changes in fibrosis.
The second poster will be presented by Lijuan Jiang, Ph.D.,
Executive Director DMPK and Bioanalysis, Enanta Pharmaceuticals,
Inc. Poster #FRI-363, titled “EDP-305, a Novel and Highly Potent
Farnesoid X Receptor (FXR) Agonist, Improves Liver Steatosis,
Ballooning and Non-Alcoholic Fatty Liver Disease (NAFLD) Activity
Score (NAS) in a Diet-Induced Murine Model of Non-Alcoholic
Steatohepatitis”, demonstrates that EDP-305 exerts beneficial
pharmacological effects in a dietary-induced NASH (DIN) mouse model
that mimics the human NAFLD/NASH physiological setting. In addition
to reducing plasma and liver lipid content, EDP-305 showed a
significant decrease in hepatocyte ballooning, and NAS in DIN mice,
suggesting that EDP-305 may have potential beneficial effects in
treating NASH.
The third poster will be presented by Yury Popov, M.D., Ph.D.,
Staff Scientist, Beth Israel Deaconess Medical Center and Assistant
Professor of Medicine, Harvard Medical School. Poster #SAT-459,
titled “A Novel FXR Agonist EDP-305 Potently Suppresses Liver
Injury and Fibrosis in Mouse Models of Biliary and Metabolic Liver
Disease”, demonstrates that treatment with EDP-305 potently
improved pre-established liver injury and hepatic fibrosis in
biliary (BALBc.Mdr2-/-) and metabolic (MCD) models of liver disease
in mice.
“The BALBc.Mdr2-/- mouse model may represent the most relevant
model that we have to evaluate the potential of new agents for the
treatment of primary sclerosing cholangitis (PSC). We are very
encouraged by the results we have observed with EDP-305 showing a
very robust response in this biliary disease model,” stated Yury
Popov, M.D., Ph.D.
“The extensive preclinical profiling of EDP-305 in a variety of
in vitro and in vivo models has given Enanta the confidence to
continue to advance EDP-305 in the clinic and to consider new
areas, such as PSC, for exploration,” stated Jay R. Luly, Ph.D.,
President and CEO, Enanta. “We expect to present clinical data from
our ongoing clinical study in healthy volunteers and presumed NAFLD
subjects and to initiate NASH-enabling studies in the second half
of this year, and also to begin phase 2 studies in PBC in the
fourth quarter of calendar 2017. We are planning for phase 2
studies in NASH in early 2018.”
EDP-305 is currently in Phase 1 clinical development. Enanta’s
ongoing double-blind, placebo-controlled Phase 1a/b study is
designed to evaluate the safety, tolerability and pharmacokinetics
of single ascending doses (SAD) and multiple ascending doses (MAD)
of EDP-305 in healthy adults, and in adults with presumptive
non-alcoholic fatty liver disease (NAFLD) (obese, with or without
pre-diabetes or type 2 diabetes). The study will enroll
approximately 150 subjects and is planned to evaluate at least 5
single and multiple dose cohorts, with EDP-305 administered orally,
once daily.
The current study includes subjects with presumptive NAFLD in
order to obtain initial safety data and additional data regarding
the relationship between EDP-305 plasma concentration levels and
certain pharmacological effects in the context of fatty liver
disease. This relationship will be explored by using biomarkers
that are relevant to the disease and to the activity of EDP-305,
such as evaluation of lipids, glucose, insulin resistance and
specific markers of FXR activity.
About EDP-305, a Farnesoid X Receptor (FXR)
AgonistEDP-305 is a potent FXR agonist and Enanta’s lead
product candidate being developed for the treatment of NASH and
PBC. EDP-305 represents a new class of FXR agonists that has been
designed to take advantage of increased binding interactions with
the receptor. Further, this non-bile acid class contains steroidal
and non-steroidal components, and does not contain the carboxylic
acid group normally present in other classes of FXR agonists and
natural bile acids that can lead to the formation of taurine and
glycine conjugates. EDP-305 has been granted Fast Track Designation
by the U.S. Food and Drug Administration and is currently in Phase
1 clinical development.
About NAFLD, NASH, and FXRNon-alcoholic fatty liver
disease (NAFLD) is the accumulation of excessive fat in the form of
triglycerides in patients’ liver cells (steatosis) that is not
caused by alcohol. NAFLD is widely considered to be the liver
expression of metabolic disease associated with type 2 diabetes,
insulin resistance, obesity, and hyperlipidemia. A subgroup of
NAFLD patients has liver cell injury and inflammation in addition
to excessive fat (steatohepatitis). Progression of this condition
leads to non-alcoholic steatohepatitis (NASH). Patients with NASH
can develop fibrosis and ultimately cirrhosis of the liver,
potentially leading to hepatocellular carcinoma or requiring a
liver transplant. The Farnesoid X receptor (FXR) is a nuclear
receptor and a main regulator of bile acid levels in the liver and
small intestine. It responds to bile acids by regulating gene
transcription of key enzymes and transporters, many of which play
important roles in lipid metabolism, insulin resistance,
inflammation, and fibrosis.
About EnantaEnanta Pharmaceuticals is a research and
development-focused biotechnology company that uses its robust
chemistry-driven approach and drug discovery capabilities to create
small molecule drugs for viral infections and liver diseases.
Enanta’s research and development efforts are currently focused on
the following disease targets: non-alcoholic steatohepatitis
(NASH)/ primary biliary cholangitis (PBC), respiratory syncytial
virus (RSV) and hepatitis B virus (HBV).
Enanta has discovered novel protease inhibitors for use against
the hepatitis C virus (HCV). These protease inhibitors, developed
through Enanta’s collaboration with AbbVie, include paritaprevir,
part of AbbVie’s currently marketed HCV regimens, and glecaprevir
(ABT-493), Enanta’s second protease inhibitor product, which AbbVie
is developing as part of its investigational, pan-genotypic HCV
regimen of glecaprevir/pibrentasvir (G/P) now in registration in
the U.S., the E.U. and Japan. Royalties and any further milestone
payments from this collaboration will provide additional funding
for Enanta’s earlier development programs, including its Phase 1
FXR agonist program for NASH/PBC, and its preclinical programs for
HBV and RSV. Please visit www.enanta.com for more information on
Enanta’s programs and pipeline.
Forward Looking Statements DisclaimerThis press release
contains forward-looking statements, including statements with
respect to the prospects for Enanta’s further development of
EDP-305. Statements that are not historical facts are based on
management’s current expectations, estimates, forecasts and
projections about Enanta’s business and the industry in which it
operates and management’s beliefs and assumptions. The statements
contained in this release are not guarantees of future performance
and involve certain risks, uncertainties and assumptions, which are
difficult to predict. Therefore, actual outcomes and results may
differ materially from what is expressed in such forward-looking
statements. Important factors and risks that may affect actual
results include: the development risks of early stage discovery
efforts in disease areas such as NASH, PBC and PSC; the impact of
development, regulatory and marketing efforts of others with
respect to competitive treatments for NASH, PBC and/or PSC;
regulatory and reimbursement actions affecting any competitive
treatment for NASH, PBC and/or PSC; Enanta’s lack of clinical
development experience; Enanta’s need to attract and retain senior
management and key scientific personnel; the need to obtain and
maintain patent protection for EDP-305 and Enanta’s other product
candidates and avoid potential infringement of the intellectual
property rights of others; and other risk factors described or
referred to in “Risk Factors” in Enanta’s most recent Form 10-K for
the fiscal year ended September 30, 2016 and other periodic reports
filed more recently with the Securities and Exchange Commission.
Enanta cautions investors not to place undue reliance on the
forward-looking statements contained in this release. These
statements speak only as of the date of this release, and Enanta
undertakes no obligation to update or revise these statements,
except as may be required by law.
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version on businesswire.com: http://www.businesswire.com/news/home/20170419005634/en/
Investor ContactEnanta Pharmaceuticals, Inc.Carol Miceli,
617-607-0710cmiceli@enanta.comorMedia ContactMacDougall
Biomedical CommunicationsKari Watson,
781-235-3060kwatson@macbiocom.com
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