WATERTOWN, Mass.--(BUSINESS WIRE)--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 it has completed enrollment in two Phase 2 studies.
The Phase 2 study of EDP-938 in RSV is a randomized, double-blind, placebo-controlled, human challenge study in healthy adult subjects randomized into 1 of 2 dosing arms or a placebo arm. Subjects are dosed for 5 days and receive a once-daily 600 mg dose, a single 500 mg loading dose followed by 300 mg twice daily, or placebo. Primary and secondary outcome measures include changes in viral load measurements and changes in baseline symptoms. Topline data is expected mid-calendar 2019.
The ARGON-1 study in NASH is a 12-week, randomized, double-blind, placebo-controlled Phase 2 study evaluating the safety, tolerability, pharmacokinetics and efficacy of EDP-305 in subjects with NASH. The primary endpoint in the study is ALT reduction and safety. Subjects receive placebo or a 1 mg or 2.5 mg dose once daily for 12-weeks. Topline data is expected by the end of the third quarter of calendar 2019.
More information on the designs of these studies can be found at www.clinicaltrials.gov.
About EDP-938
EDP-938, Enanta’s lead N-protein inhibitor, is
being developed for the treatment of RSV infection. Enanta believes
EDP-938 is differentiated from fusion inhibitors currently in
development by others for RSV because this N-protein inhibitor targets
the virus’ replication machinery and has demonstrated high barriers to
resistance against the virus in vitro. EDP-938 has also been
shown to reduce viral load below the level of detection in vivo.
Additionally, it is possible that N-protein inhibitors may be effective
treatments at later stages of infection.
About RSV
Respiratory syncytial virus (RSV) is a virus that
infects the lungs and represents a serious unmet medical need in infants
and children, as well as immune-compromised individuals and the elderly.
RSV is the most common cause of bronchiolitis (inflammation of the small
airways in the lung) and pneumonia in children under 1 year of age in
the United States. Each year, 57,000 to 125,000 children in this group
are hospitalized due to RSV infection. Also, at increased risk of a
severe RSV infection, are children with compromised (weakened) immune
systems due to a medical condition or medical treatment, adults with
compromised immune systems and those 65 and older. There is currently no
safe and effective therapy for already established RSV infection.
About EDP-305, a Farnesoid X Receptor (FXR) Agonist
EDP-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 that can lead to the formation of
taurine and glycine conjugates normally associated with bile acids,
which may also be present in other classes of FXR agonists.
About NAFLD, NASH, and FXR
Non-alcoholic fatty liver disease
(NAFLD) is the accumulation of excessive fat in the form of
triglycerides in patients’ liver cells (steatosis) that is not
associated with alcohol abuse. 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 (steatohepatitis) in
addition to excessive fat. 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 (HCC) or requiring a liver transplant.
Farnesoid X receptor (FXR) is a nuclear receptor and a main regulator of
bile acid levels in the liver and small intestine. FXR 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 Enanta
Enanta Pharmaceuticals is using its robust,
chemistry-driven approach and drug discovery capabilities to become a
leader in the discovery and development of small molecule drugs for the
treatment of viral infections and liver diseases. Enanta’s research and
development efforts are currently focused on the following disease
targets: respiratory syncytial virus (RSV), non-alcoholic
steatohepatitis (NASH)/ primary biliary cholangitis (PBC), and hepatitis
B virus (HBV).
Enanta’s research and development activities are funded by royalties from HCV products developed under its collaboration with AbbVie. Glecaprevir, a protease inhibitor discovered by Enanta, is now sold by AbbVie in numerous countries as part of its newest treatment for chronic hepatitis C virus (HCV) infection. This leading HCV regimen is sold under the tradenames MAVYRET™ (U.S.) and MAVIRET™ (ex-U.S.) (glecaprevir/pibrentasvir). Please visit www.enanta.com for more information.
Forward Looking Statements Disclaimer
This press release
contains forward-looking statements, including statements with respect
to the prospects for further developments with respect to EDP-938 for
RSV and EDP-305 for NASH/PBC. 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 the disease areas
in Enanta’s research and development pipeline, such as RSV, NASH and
PBC; the impact of development, regulatory and marketing efforts of
others with respect to competitive treatments for RSV, NASH and PBC;
Enanta’s limited clinical development experience; Enanta’s need to
attract and retain senior management and key scientific personnel;
Enanta’s need to obtain and maintain patent protection for its 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, 2018 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.