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 that the U.S. Food and Drug Administration (FDA) has granted Enanta’s drug candidate EDP-305, an FXR agonist, Fast Track designation for the treatment of patients with non-alcoholic steatohepatitis (NASH) with liver fibrosis.
Fast track is a process designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. A drug that receives Fast Track designation is also eligible for more frequent meetings and communications with the FDA to discuss the drug's development plan.1
“We are extremely pleased to receive this Fast Track designation from the FDA and look forward to working with the agency to bring this investigational treatment to patients as soon as possible,” stated Jay R. Luly, Ph.D.
EDP-305 is currently in Phase 1 clinical development. Enanta’s 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 90 subjects and is planned to evaluate up to 5 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) 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 steroid and non-steroid 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 is currently in Phase 1 clinical
development.
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 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 also develops
liver cell injury and inflammation. This condition is called
non-alcoholic steatohepatitis (NASH). Patients with NASH can develop
fibrosis (the first stage of scarring of the liver) and ultimately
cirrhosis of the liver, potentially leading to hepatocellular carcinoma
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. 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 Enanta
Enanta 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 four disease
targets: Hepatitis C Virus (HCV), Non-alcoholic Steatohepatitis (NASH),
Respiratory Syncytial Virus (RSV) and Hepatitis B Virus (HBV).
Enanta has discovered novel protease inhibitors that are members of the direct-acting-antiviral (DAA) inhibitor classes designed for use against the hepatitis C virus (HCV). These protease inhibitors, developed through Enanta’s collaboration with AbbVie, include paritaprevir, which is contained in AbbVie’s marketed DAA regimens for HCV, and glecaprevir (ABT-493), Enanta’s second protease inhibitor product, which has been developed in Phase 3 studies as part of an investigational, pan-genotypic, once-daily, ribavirin-free, fixed-dose combination (G/P) with pibrentasvir (ABT-530), AbbVie’s second NS5A inhibitor. AbbVie has announced it has filed an NDA for G/P with the FDA and is on track to submit a marketing authorization application for G/P in the European Union in early 2017. Enanta has also discovered EDP-305, an FXR agonist product candidate for NASH, currently in Phase 1 clinical development, as well as a cyclophilin inhibitor, EDP-494, a novel host-targeting mechanism for HCV, which is also in Phase 1 clinical development. In addition, Enanta has early lead candidates for HBV and RSV in preclinical development. Please visit www.enanta.com for more information on Enanta’s programs and pipeline.
Forward Looking Statements Disclaimer
This 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 new disease areas such as NASH; the
impact of development, regulatory and marketing efforts of others with
respect to competitive treatments for NASH; regulatory and reimbursement
actions affecting any competitive treatment for NASH; 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,
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.
________________________________
1 http://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm