ENTA Enanta Pharmaceuticals Inc.

49.94
-1.31  -3%
Previous Close 51.25
Open 52.07
52 Week Low 38.4
52 Week High 89.25
Market Cap $999,012,645
Shares 20,004,258
Float 16,336,020
Enterprise Value $679,057,222
Volume 149,836
Av. Daily Volume 161,630
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Upcoming Catalysts

Drug Stage Catalyst Date
EDP-938
Respiratory Syncytial Virus
Phase 2b
Phase 2b
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Drug Pipeline

Drug Stage Notes
EDP-305 INTREPID
Primary biliary cholangitis (PBC)
Phase 2
Phase 2
Phase 2 top-line data did not meet primary endpoint - May 6, 2020.
EDP-297
Non-alcoholic steatohepatitis (NASH)
Phase 1
Phase 1
Phase 1 trial to commence 3Q 2020.
EDP-514
Hepatitis B virus (HBV)
Phase 1b
Phase 1b
Phase 1b trial to be initiated 2Q 2020.
EDP-305 ARGON-2
Non-alcoholic steatohepatitis (NASH)
Phase 2
Phase 2
Phase 2b trial initiation has been delayed due to COVID-19 - March 26, 2020.
VIEKIRA PAK - once-daily, fixed-dose formulation through Abbvie
HCV - genotype
Approved
Approved
Approved July 25, 2017.
VIEKIRA PAK
HCV - genotype 1
Approved
Approved
Approved December 19, 2014.
Glecaprevir/Pibrentasvir (G/P)
Hepatitis C virus (HCV)
Approved
Approved
Approval announced August 3, 2017.

Latest News

  1. Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced the appointment of Mark G. Foletta to its Board of Directors. Mr. Foletta will also serve as the Chair of the Audit Committee and will be a member of the Nominating and Corporate Governance Committee. The appointment of Mr. Foletta increases the number of Enanta Directors to seven.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200629005071/en/

    Enanta Pharmaceuticals Announces the Appointment of Mark G. Foletta to its Board of Directors (Photo: Business Wire)

    Enanta Pharmaceuticals Announces the Appointment of Mark G. Foletta to its Board of Directors (Photo: Business Wire)

    "We are pleased to welcome Mark to our Board…

    Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced the appointment of Mark G. Foletta to its Board of Directors. Mr. Foletta will also serve as the Chair of the Audit Committee and will be a member of the Nominating and Corporate Governance Committee. The appointment of Mr. Foletta increases the number of Enanta Directors to seven.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200629005071/en/

    Enanta Pharmaceuticals Announces the Appointment of Mark G. Foletta to its Board of Directors (Photo: Business Wire)

    Enanta Pharmaceuticals Announces the Appointment of Mark G. Foletta to its Board of Directors (Photo: Business Wire)

    "We are pleased to welcome Mark to our Board during an important time in Enanta's growth," commented Jay R. Luly, Ph.D., President and Chief Executive Officer of Enanta Pharmaceuticals. "Mark's extensive financial, operational and governance leadership will be invaluable as we continue to develop our broad pipeline of small molecule drugs for viral infections and liver diseases."

    "Enanta is well-positioned for enduring success in advancing its virology and liver disease treatments," said Mr. Foletta. "I look forward to collaborating with the leadership team and members of the Board to further Enanta's mission and bring these critical therapies to patients in need."

    Mr. Foletta brings to Enanta more than 25 years of experience in financial, operational and governance leadership roles in the pharmaceutical and biotechnology industry. Most recently, he was Executive Vice President and Chief Financial Officer of Tocagen, a clinical-stage biopharmaceutical company focused on advancing a cancer-selective gene therapy platform, prior to its merger with Forte Biosciences. Prior to Tocagen, Mr. Foletta was Chief Financial Officer of Biocept, Inc. and served as Senior Vice President, Finance and Chief Financial Officer of Amylin Pharmaceuticals, Inc., where he helped drive the evolution of the company from a research and development organization to a fully integrated biopharmaceutical company. Mr. Foletta has held other management positions including Senior Vice President, Chief Financial Officer and Corporate Secretary of Intermark, Inc. and Triton Group Ltd. Earlier in his career he worked at Ernst & Young, LLP.

    Mr. Foletta currently serves as the Lead Independent Director of DexCom, Inc. and is Chairman of the Audit Committee of AMN Healthcare Services, Inc. He has previously served on the Boards of Directors and as Audit Committee Chair of Regulus Therapeutics, Inc., Ambit Biosciences, Inc. and Anadys Pharmaceuticals, Inc. Mr. Foletta received a B.A. in Business Economics from the University of California, Santa Barbara and is a member of the Corporate Directors Forum.

    About Enanta

    Enanta 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 have produced clinical candidates for the following disease targets: respiratory syncytial virus (RSV), non-alcoholic steatohepatitis (NASH), hepatitis B virus (HBV), human metapneumovirus (hMPV) and SARS-CoV-2 (COVID-19).

    Enanta's research and development activities are funded by royalties from hepatitis C virus (HCV) products developed under its collaboration with AbbVie. Glecaprevir, a protease inhibitor discovered by Enanta, is sold by AbbVie in numerous countries as part of its leading treatment for chronic HCV infection under the tradenames MAVYRET® (U.S.) and MAVIRET® (ex-U.S.) (glecaprevir/pibrentasvir). Please visit www.enanta.com for more information.

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  2. Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced that Jay R. Luly, Ph.D., President and Chief Executive Officer, will present at the Raymond James Human Health Innovation Conference on June 18, 2020 at 10:20 a.m. ET.

    A live webcast will be accessible by visiting the "Events and Presentations" section on the "Investors" page of Enanta's website at www.enanta.com. A replay of the webcast will be available following the presentation and will be archived for approximately 30 days.

    About Enanta

    Enanta Pharmaceuticals is using its robust, chemistry-driven approach and drug discovery capabilities to become a leader…

    Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced that Jay R. Luly, Ph.D., President and Chief Executive Officer, will present at the Raymond James Human Health Innovation Conference on June 18, 2020 at 10:20 a.m. ET.

    A live webcast will be accessible by visiting the "Events and Presentations" section on the "Investors" page of Enanta's website at www.enanta.com. A replay of the webcast will be available following the presentation and will be archived for approximately 30 days.

    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), hepatitis B virus (HBV), human metapneumovirus (hMPV) and SARS-CoV-2 (COVID-19).

    Enanta's research and development activities are funded by royalties from hepatitis C virus (HCV) products developed under its collaboration with AbbVie. Glecaprevir, a protease inhibitor discovered by Enanta, is sold by AbbVie in numerous countries as part of its leading treatment for chronic HCV infection, under the tradenames MAVYRET® (U.S.) and MAVIRET® (ex-U.S.) (glecaprevir/pibrentasvir). Please visit www.enanta.com for more information.

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  3. Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced that Jay R. Luly, Ph.D., President and Chief Executive Officer, will participate in a fireside chat at the 2020 RBC Capital Markets Global Healthcare Virtual Conference on May 19, 2020 at 9:45 a.m. ET.

    A live webcast of the fireside chat will be accessible by visiting the "Events and Presentations" section on the "Investors" page of Enanta's website at www.enanta.com. A replay of the webcast will be available following the presentation and will be archived for approximately 30 days.

    About Enanta

    Enanta Pharmaceuticals is using its robust, chemistry-driven…

    Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced that Jay R. Luly, Ph.D., President and Chief Executive Officer, will participate in a fireside chat at the 2020 RBC Capital Markets Global Healthcare Virtual Conference on May 19, 2020 at 9:45 a.m. ET.

    A live webcast of the fireside chat will be accessible by visiting the "Events and Presentations" section on the "Investors" page of Enanta's website at www.enanta.com. A replay of the webcast will be available following the presentation and will be archived for approximately 30 days.

    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), hepatitis B virus (HBV), human metapneumovirus (hMPV) and SARS-CoV-2.

    Enanta's research and development activities are funded by royalties from hepatitis C virus (HCV) products developed under its collaboration with AbbVie. Glecaprevir, a protease inhibitor discovered by Enanta, is sold by AbbVie in numerous countries as part of its leading treatment for chronic 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.

    View Full Article Hide Full Article
    • On Track to Initiate Phase 1b Study of EDP-514 in Viremic Hepatitis B Patients in 2Q 2020 and Phase 1 Study of EDP-297 in 3Q 2020
    • Royalty Revenue for the Quarter was $27.6 Million
    • Cash and Marketable Securities Totaled $435.4 Million at March 31, 2020

    Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today reported financial results for its fiscal second quarter ended March 31, 2020.

    "Enanta is fortunate to have advanced several of its programs in the recent quarter and it continues to have a strong balance sheet and ongoing royalty funding to support its business plans going forward," said Jay R. Luly, President and…

    • On Track to Initiate Phase 1b Study of EDP-514 in Viremic Hepatitis B Patients in 2Q 2020 and Phase 1 Study of EDP-297 in 3Q 2020
    • Royalty Revenue for the Quarter was $27.6 Million
    • Cash and Marketable Securities Totaled $435.4 Million at March 31, 2020

    Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today reported financial results for its fiscal second quarter ended March 31, 2020.

    "Enanta is fortunate to have advanced several of its programs in the recent quarter and it continues to have a strong balance sheet and ongoing royalty funding to support its business plans going forward," said Jay R. Luly, President and Chief Executive Officer of Enanta Pharmaceuticals. "The current pandemic has underscored our commitment as a company to bring forth novel anti-viral therapies for respiratory viruses and liver infections, and we remain highly dedicated to the clinical advancement of our innovative small molecule pipeline. We are on schedule to initiate our Phase 1b study of EDP-514 in viremic hepatitis B patients this quarter, and we also expect to initiate a first-in-human study of our follow-on Farnesoid X receptor agonist, EDP-297, next quarter. Additionally, we have plans for two additional studies of EDP-938 for respiratory syncytial virus to start by year-end, one in pediatric patients and one in adult transplant patients. With these catalysts in mind, we continue to monitor any impact of COVID-19 on all of our clinical studies."

    Fiscal Second Quarter Ended March 31, 2020 Financial Results

    Total revenue for the three months ended March 31, 2020 was $27.6 million and consisted of royalty revenue derived primarily from worldwide net sales of AbbVie's hepatitis C virus (HCV) regimen MAVYRET®/MAVIRET® (glecaprevir/pibrentasvir). For the three months ended March 31, 2019, total revenue was $39.6 million, which consisted of royalty revenue earned on AbbVie's global net sales of its HCV regimens. AbbVie has reported that the decrease in its first quarter 2020 HCV sales was due to lower patient volumes in select international markets where patients are treated in hospitals affected by COVID-19, as well as increased competition affecting pricing and market share within the U.S. Managed Medicaid segment.

    Research and development expenses totaled $32.6 million for the three months ended March 31, 2020, compared to $34.2 million for the three months ended March 31, 2019. The decrease in research and development expenses was primarily due to a decrease in clinical trial expense due to timing of Phase 2 studies in non-alcoholic steatohepatitis (NASH)/primary biliary cholangitis (PBC) conducted in the prior year.

    General and administrative expenses totaled $6.9 million for the three months ended March 31, 2020, compared to $6.8 million for the three months ended March 31, 2019.

    Enanta recorded an income tax benefit of $3.9 million for the three months ended March 31, 2020 compared to an income tax benefit of $3.2 million for the same period of 2019. The income tax benefit for the three months ended March 31, 2020 was due to a pre-tax loss and increased research and development tax credits. In the prior year, Enanta recorded an income tax benefit despite reporting pre-tax income due to tax deductions from employee stock award-related activity during the quarter.

    Net loss for the three months ended March 31, 2020 was $6.0 million, or a loss of $0.30 per diluted common share, compared to net income of $4.1 million, or $0.20 per diluted common share, for the corresponding period in 2019.

    Enanta's cash, cash equivalents and short-term and long-term marketable securities totaled $435.4 million at March 31, 2020. This compares to a total of $400.3 million at September 30, 2019. Enanta expects that its current cash, cash equivalents and marketable securities, as well as its continuing royalty revenue, will be sufficient to meet the anticipated cash requirements of its existing business and development programs for the foreseeable future.

    Pipeline Programs and Near-term Milestones

    • Respiratory Syncytial Virus (RSV): N-Protein Inhibitor EDP-938, Human Metapneumovirus (hMPV) and SARS-CoV-2
      • Continue with plans to broaden the RSVP study into the Southern Hemisphere, and to expand to trials sites in Europe and North America in the fall and winter RSV season, with the goal of having data in the third quarter of 2021
      • Plan to initiate Phase 2 dose ranging study in pediatric patients with RSV in 4Q 2020
      • Plan to initiate Phase 2 study in adult transplant patients with RSV in 4Q 2020
      • Perform optimization of Enanta's current nanomolar hMPV inhibitor leads
      • Advance efforts for discovery of direct-acting antiviral compounds for SARS-CoV-2
    • Hepatitis B (HBV): Core Inhibitor EDP-514
      • Initiate Phase 1b study in viremic HBV patients in 2Q 2020
      • Resume recruitment in Phase 1b study in nuc-suppressed HBV patients, currently paused
    • Non-Alcoholic Steatohepatitis (NASH): Farnesoid X Receptor (FXR) Agonists EDP-305 and EDP-297
      • Resume recruitment and dosing in ARGON-2 Phase 2b study of EDP-305 in NASH, currently paused
      • Plan to initiate Phase 1 study of EDP-297 (follow-on FXR for NASH) in 3Q 2020
      • Advance efforts for discovery of non-FXR compounds for NASH
    • Hepatitis C (HCV) Collaboration with AbbVie
      • AbbVie announced that the European Commission approved a change to the marketing authorization for MAVIRET® (glecaprevir/pibrentasvir) to shorten once-daily treatment duration from 12 to 8 weeks in treatment-naïve, compensated cirrhotic, chronic HCV patients with genotype (GT) 3 infection. The decision makes MAVIRET the only pan-genotypic (GTs 1-6) 8-week treatment option for treatment-naïve, chronic HCV patients, without cirrhosis or with compensated cirrhosis.

    Upcoming Events and Presentations

    • May 19, 2020 – RBC Capital Markets Global Healthcare Conference, Virtual
    • June 18, 2020 – Raymond James Healthcare Conference, Virtual
    • Enanta plans to issue its fiscal third quarter financial results press release, and hold a conference call regarding those results, on August 4, 2020.

    Conference Call and Webcast Information

    Enanta will host a conference call and webcast today at 4:30 p.m. ET. To participate in the live conference call, please dial (855) 840-0595 in the U.S. or (518) 444-4814 for international callers. A replay of the conference call will be available starting at approximately 7:30 p.m. ET on May 6, 2020, through 11:59 p.m. ET on May 8, 2020 by dialing (855) 859-2056 from the U.S. or (404) 537-3406 for international callers. The passcode for both the live call and the replay is 4261269. A live audio webcast of the call and replay can be accessed by visiting the "Events and Presentation" section on the "Investors" page of Enanta's website at www.enanta.com.

    About Enanta Pharmaceuticals, Inc.

    Enanta 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 have produced clinical candidates for the following disease targets: respiratory syncytial virus (RSV), non-alcoholic steatohepatitis (NASH) and hepatitis B virus (HBV). Enanta is also conducting research in human metapneumovirus (hMPV) and emerging coronaviruses, including SARS-CoV-2.

    Enanta's research and development activities are funded by royalties from hepatitis C virus (HCV) products developed under its collaboration with AbbVie. Glecaprevir, a protease inhibitor discovered by Enanta and now marketed by AbbVie as part of its leading treatment for chronic HCV infection, is sold under the brand names MAVYRET® (U.S.) and MAVIRET® (ex-U.S.) (glecaprevir/pibrentasvir). Please visit www.enanta.com for more information.

    FORWARD LOOKING STATEMENTS

    This press release contains forward-looking statements, including statements with respect to the prospects for advancement of Enanta's clinical programs in RSV, NASH and HBV, as well as discovery programs in hMPV and SARS-CoV-2, and prospects for future royalty revenue from sales of AbbVie's MAVYRET®/MAVIRET® HCV regimen. 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: Enanta's revenues in the short-term are dependent upon the level of AbbVie's sales of its MAVYRET®/MAVIRET® HCV regimen; the impact of development, regulatory and marketing efforts of others with respect to competitive treatments for RSV, NASH, and HBV; competitive pricing, market acceptance and reimbursement rate actions affecting MAVYRET®/MAVIRET® compared to competitive HCV products on the market; the discovery and development risks of Enanta's programs in RSV, NASH, HBV, hMPV, SARS-CoV-2; the competitive impact of development, regulatory and marketing efforts of others in those disease areas; Enanta's lack of clinical development experience; Enanta's need to attract and retain senior management and key research and development 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-Q for the quarter ended December 31, 2019, 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.

    ENANTA PHARMACEUTICALS, INC.
    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
    UNAUDITED
    (in thousands, except per share amounts)
     
     
    Three Months Ended Six Months Ended
    March 31, March 31,

    2020

    2019

    2020

    2019

     
    Revenue

    $ 27,619

    $ 39,631

    $ 80,189

    $ 109,517

    Operating expenses
    Research and development

    32,610

    34,155

    65,388

    69,033

    General and administrative

    6,884

    6,780

    13,805

    13,932

    Total operating expenses

    39,494

    40,935

    79,193

    82,965

    Income (loss) from operations

    (11,875)

    (1,304)

    996

    26,552

    Other income, net

    1,950

    2,245

    4,026

    4,130

    Income (loss) before income taxes

    (9,925)

    941

    5,022

    30,682

    Income tax (expense) benefit

    3,920

    3,204

    2,416

    (526)

    Net income (loss)

    $ (6,005)

    $ 4,145

    $ 7,438

    $ 30,156

     
     
    Net income (loss) per share
    Basic

    $ (0.30)

    $ 0.21

    $ 0.37

    $ 1.55

    Diluted

    $ (0.30)

    $ 0.20

    $ 0.36

    $ 1.44

    Weighted average common shares outstanding
    Basic

    19,922

    19,549

    19,836

    19,487

    Diluted

    19,922

    21,084

    20,692

    20,946

    ENANTA PHARMACEUTICALS, INC.
    CONDENSED CONSOLIDATED BALANCE SHEETS
    UNAUDITED
    (in thousands)
                 
            March 31,   September 30,
           

    2020

     

    2019

    Assets            
    Current assets          
      Cash and cash equivalents  

     $                   74,338

     

     $                    51,230

      Short-term marketable securities  

                        280,917

     

                         284,006

      Accounts receivable  

                           27,619

     

                            51,313

      Prepaid expenses and other current assets  

                           19,835

     

                            15,299

        Total current assets  

    402,709

     

    401,848

    Long-term marketable securities  

                           80,099

     

                            65,013

    Property and equipment, net  

                             9,738

     

                            10,927

    Deferred tax assets    

                           12,418

     

                            11,341

    Operating lease, right-of-use assets  

                             7,837

                                       —
    Restricted cash    

                                 608

     

                                  608

    Other long-term assets    

                                   92

     

                                    92

        Total assets  

    $                  513,501

     

    $                   489,829

    Liabilities and Stockholders' Equity        
    Current liabilities          
      Accounts payable  

     $                      5,945

     

     $                       6,689

      Accrued expenses and other current liabilities  

                             9,807

     

                            15,920

      Operating lease liabilities  

                             3,764

                                       —
        Total current liabilities  

                           19,516

     

                            22,609

    Operating lease liabilities, net of current portion  

                             5,330

                                       —
    Series 1 nonconvertible preferred stock  

                             1,628

     

                              1,628

    Other long-term liabilities  

                             1,036

     

                              3,100

        Total liabilities  

                           27,510

     

                            27,337

    Total stockholders' equity  

                        485,991

     

                         462,492

        Total liabilities and stockholders' equity

    $                  513,501

     

    $                   489,829

     

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  4. Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced topline results from its INTREPID Phase 2 study of EDP-305, a Farnesoid X receptor (FXR), in subjects with primary biliary cholangitis (PBC).

    The INTREPID study was a 12-week, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics and efficacy of EDP-305 in subjects with PBC, with or without an inadequate response to ursodeoxycholic acid. The primary endpoint of the study was to evaluate the proportion of subjects with at least 20% reduction in alkaline phosphatase (ALP) from pre-treatment value (ALP response…

    Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced topline results from its INTREPID Phase 2 study of EDP-305, a Farnesoid X receptor (FXR), in subjects with primary biliary cholangitis (PBC).

    The INTREPID study was a 12-week, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics and efficacy of EDP-305 in subjects with PBC, with or without an inadequate response to ursodeoxycholic acid. The primary endpoint of the study was to evaluate the proportion of subjects with at least 20% reduction in alkaline phosphatase (ALP) from pre-treatment value (ALP response), or normalization of ALP, at week 12.

    In the intent-to-treat (ITT) analysis, EDP-305 1 mg and 2.5 mg treatment arms resulted in 45% (n=14/31, p=0.106) and 46% (n=13/28, p=0.063) ALP response, respectively, compared to 11% (n=1/9) in the placebo arm. Absolute changes from baseline in ALP at week 12 in both the EDP-305 1 mg arm (p=0.017) and the 2.5 mg arm (p= 0.021) compared to the change from baseline in the placebo arm were statistically significant. In a post-hoc analysis, the proportions of ALP responders among those who completed treatment with no missing value at week 12 were 50% (n=14/28, p=0.039) and 62% (n=13/21, p=0.011), respectively, compared to 11% in placebo (n=1/9).

    In the ITT analysis, key secondary endpoints, which included changes from baseline in liver enzymes alanine aminotransferase (ALT), aspartate aminotransferase (AST) and gamma-glutamyl transferase (GGT) at week 12, were met with a statistically significant difference compared to placebo in both the EDP-305 1 mg arm and the 2.5 mg arm. Absolute changes from baseline at week 12 in these key secondary endpoints in the EDP-305 1 mg and 2.5 mg arms compared to placebo were respectively: ALT (p=0.001, p=0.009), AST (p=0.000, p=0.001) and GGT (p=0.000, p=0.000). A statistically significant difference in the percent change from baseline of these key biomarkers at week 12 was also observed in both EDP-305 arms compared to placebo.

    Overall, EDP-305 was generally safe in subjects with PBC, with the majority of treatment-emergent adverse events (TEAEs) being mild to moderate. Five patients in the 2.5 mg arm experienced severe pruritus. The most common (≥10% or >1 subject/arm) TEAEs included pruritus, gastrointestinal-related symptoms (abdominal pain, diarrhea, gastro-esophageal reflux), headache and insomnia. These TEAEs are consistent with the safety profile observed across more than 400 subjects exposed to EDP-305 for up to 12 weeks. The incidence of treatment discontinuation due to pruritus in INTREPID was approximately 3% for the 1 mg EDP-305 treatment group and 18% for the 2.5 mg EDP-305 treatment group. Treatment with EDP-305 had no apparent effect on lipids, including cholesterol, low-density lipoproteins, high-density lipoproteins and triglycerides.

    "While INTREPID did not meet the primary endpoint in subjects with PBC, as defined by at least a 20% reduction in ALP in the ITT set analysis, there were numerically higher response rates with 1 mg and 2.5 mg compared to placebo," said Jay Luly, Ph.D., President and Chief Executive Officer of Enanta Pharmaceuticals. "In addition, as shown in the completer analysis, those subjects who finished treatment had a significant ALP response. We were also able to obtain actionable data from this study to help us advance EDP-305 and were encouraged that the lower dose of 1 mg could achieve much better tolerability, in terms of pruritus, without reducing the number of ALP responders or key biomarkers of target engagement also achieved at the 2.5 mg dose. This is an encouraging finding, particularly for the intermediate doses of 1.5 mg and 2.0 mg that we plan to take into our ARGON-2 study in non-alcoholic steatohepatitis (NASH) patients. One of those two doses in NASH could potentially achieve an efficacy and tolerability profile acceptable in NASH patients."

    Dr. Luly continued, "Rather than conducting further dose selection studies with EDP-305 in PBC, a disease for which there is already an approved second-line FXR agonist therapy, we intend to focus our future efforts with EDP-305 on NASH, a disease where FXR agonists like EDP-305 have the potential to be important components of drug combinations designed to give maximum benefit to patients. Our ARGON-2 study in NASH will explore new intermediate doses with the potential to optimize efficacy and tolerability, thereby maximizing the opportunity to develop EDP-305 in combination with other mechanisms of action against NASH."

    "Based on these data, it's clear that EDP-305 demonstrated evidence of target engagement with robust reductions in markers of liver injury," said Kris Kowdley, M.D., FACP, FACG, AGAF, FAASLD, Director, Liver Institute Northwest and Clinical Professor, Elson S. Floyd College of Medicine, Washington State University, the Principal Investigator for the study. "These data suggest that a dose between 1.0 mg and 2.5 mg could hit an optimal level of efficacy and tolerability, which could bode well for future studies in NASH. I look forward to seeing future data on EDP-305."

    About Primary Biliary Cholangitis

    Primary biliary cholangitis (PBC) is a chronic disease of the liver that slowly destroys the medium-sized bile ducts within the liver. Bile is a digestive liquid that is made in the liver. It travels through the bile ducts to the small intestine, where it helps digest fats and fatty vitamins.

    In patients with PBC, the bile ducts are destroyed by inflammation. This causes bile to remain in the liver, where its increased levels gradually damage liver cells and cause cirrhosis or scarring of the liver. As cirrhosis progresses and the amount of scar tissue in the liver increases, the liver loses its ability to function, leading to potential liver failure, liver transplantation or hepatocellular carcinoma. PBC affects mostly women, but more men are now being diagnosed. The disorder usually becomes apparent during middle age, initially affecting most individuals between the ages of 45 to 65 years. However, the disorder has been diagnosed in females as young as 22 years of age and in females in their early 90s. It has been estimated that PBC is one of the most common autoimmune diseases, affecting nearly 1 in 1000 women over the age of 40.1

    About EDP-305, a Farnesoid X Receptor Agonist

    EDP-305 is a potent Farnesoid X receptor (FXR) agonist and Enanta's lead product candidate being developed primarily for the treatment of NASH. 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. EDP-305 represents a class of FXR agonists that has been designed to take advantage of increased binding interactions with the receptor. 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.

    About Enanta Pharmaceuticals, Inc.

    Enanta 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 have produced clinical candidates for the following disease targets: respiratory syncytial virus (RSV), non-alcoholic steatohepatitis (NASH) and hepatitis B virus (HBV). Enanta is also conducting research in human metapneumovirus (hMPV) and emerging coronaviruses, including SARS-CoV-2.

    Enanta's research and development activities are funded by royalties from hepatitis C virus (HCV) products developed under its collaboration with AbbVie. Glecaprevir, a protease inhibitor discovered by Enanta and now marketed by AbbVie as part of its leading treatment for chronic HCV infection, is sold under the brand names 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 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 the disease areas in Enanta's research and development pipeline, such as NASH; the impact of development, regulatory and marketing efforts of others with respect to competitive treatments 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-Q for the fiscal quarter ended December 31, 2019 and any 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 https://rarediseases.org/rare-diseases/primary-biliary-cholangitis/

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