ICPT Intercept Pharmaceuticals Inc.

36.61
+0.92  (+3%)
Previous Close 35.69
Open 35.6
52 Week Low 27.0201
52 Week High 125
Market Cap $1,207,853,082
Shares 32,992,436
Float 20,812,122
Enterprise Value $1,245,629,402
Volume 545,676
Av. Daily Volume 827,602
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Upcoming Catalysts

Drug Stage Catalyst Date
Ocaliva - Obeticholic acid (OCA) - REVERSE
NASH patients with compensated cirrhosis
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
Ocaliva (Obeticholic acid (OCA)) - REGENERATE
Adult nonalcoholic steatohepatitis (NASH) patients.
Phase 3
Phase 3
CRL announced June 29, 2020.
Obeticholic acid (Ocaliva)
Primary biliary cirrhosis (PBC) - POISE
Approved
Approved
Approved May 27 2016. Updated U.S. label for Ocaliva anticipated by early 2018
Obeticholic acid (OCA) AESOP
Primary Sclerosing Cholangitis (PSC)
Phase 2
Phase 2
Phase 2 data released July 31, 2017 - primary endpoint met. Late breaker at AASLD October 23, 2017.
Obeticholic acid (OCA) - CONTROL
NASH patients taking statins
Phase 2
Phase 2
Phase 2 primary endpoint met - July 31, 2017. One patient death noted but DSMB noted unlikely to be related to OCA (company unsure).

Latest News

  1. NEW YORK, Nov. 24, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that Mark Pruzanski, M.D., President and Chief Executive Officer, Sandip Kapadia, Chief Financial Officer, and Gail Cawkwell, M.D., Ph.D., SVP, Medical Affairs, Safety and Pharmacovigilance of Intercept, will participate in a fireside chat at the Piper Sandler 32nd Annual Virtual Healthcare Conference on Tuesday, December 1, 2020 from 3:30 – 3:55 p.m. ET.

    A live webcast of the event will be available on the investor page of Intercept's website at http://ir.interceptpharma.com. An audio archive…

    NEW YORK, Nov. 24, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that Mark Pruzanski, M.D., President and Chief Executive Officer, Sandip Kapadia, Chief Financial Officer, and Gail Cawkwell, M.D., Ph.D., SVP, Medical Affairs, Safety and Pharmacovigilance of Intercept, will participate in a fireside chat at the Piper Sandler 32nd Annual Virtual Healthcare Conference on Tuesday, December 1, 2020 from 3:30 – 3:55 p.m. ET.

    A live webcast of the event will be available on the investor page of Intercept's website at http://ir.interceptpharma.com. An audio archive of the webcast will also be available on Intercept's website for approximately two weeks.

    About Intercept

    Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Founded in 2002 in New York, Intercept has operations in the United States, Europe and Canada. For more information, please visit www.interceptpharma.com or connect with the company on Twitter and LinkedIn.

    Contact

    For more information about Intercept, please contact:

    Investor inquiries:  

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  2. NEW YORK, Nov. 16, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced multiple new analyses supporting the use of routine noninvasive tests (NITs) to identify patients with advanced fibrosis due to NASH and measure obeticholic acid (OCA) treatment response. The new analyses, which include an oral presentation of REGENERATE interim analysis data showing that OCA helped patients achieve marked improvements in key noninvasive measures of liver fibrosis, are being presented at The Liver Meeting Digital Experience™, the Annual Meeting of the American Association for…

    NEW YORK, Nov. 16, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced multiple new analyses supporting the use of routine noninvasive tests (NITs) to identify patients with advanced fibrosis due to NASH and measure obeticholic acid (OCA) treatment response. The new analyses, which include an oral presentation of REGENERATE interim analysis data showing that OCA helped patients achieve marked improvements in key noninvasive measures of liver fibrosis, are being presented at The Liver Meeting Digital Experience™, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which will be held virtually from November 13, 2020 to November 16, 2020.

    "NITs are rapidly replacing liver biopsy for identifying and monitoring patients with advanced fibrosis due to NASH in routine clinical practice, and this year's Liver Meeting features a wealth of new data reinforcing the value of noninvasive strategies to manage patients treated with OCA," said Naim Alkhouri, M.D., Chief of Transplant Hepatology, and Director of the Fatty Liver Program at Arizona Liver Health in Phoenix. "Using a simple, sequential algorithm with two common NITs, we were able to identify a higher-risk subgroup of patients with fibrosis due to NASH and evaluate their treatment response noninvasively; these patients achieved marked reductions in measures of liver biochemistry and liver stiffness as assessed by transient elastography through 18 months of treatment."

    As previously reported, once-daily OCA 25 mg met the primary composite endpoint of fibrosis improvement (≥1 stage) with no worsening of NASH at the planned 18-month interim analysis of the Phase 3 REGENERATE study with high statistical significance (p=0.0002 vs. placebo). The new post hoc analysis being presented at The Liver Meeting evaluated the NIT-based efficacy of OCA in patients from the intent-to-treat population of the REGENERATE interim analysis who had Fibrosis-4 (FIB-4) and transient elastography data available at baseline. FIB-4 and transient elastography were applied sequentially to categorize patients' fibrosis severity; patients with possible advanced fibrosis (indeterminant status) or advanced fibrosis were pooled (OCA 25 mg, n=266; placebo, n=277). At month 18, OCA reduced mean alanine aminotransferase (ALT) scores and median transient elastography scores by 50.1% and 25.6%, respectively; reductions for placebo were 30.2% and 4.2%, respectively, suggesting that such noninvasive assessments can be utilized to monitor fibrosis improvement in OCA-treated patients.

    Additional Analyses Support the Role of NITs for Management of Advanced Fibrosis Due to NASH

    Multiple additional analyses being presented at the virtual Liver Meeting reinforce the value of noninvasive strategies for managing patients with advanced fibrosis due to NASH:

    • In an oral presentation (Abstract 56), an analysis of more than 4,000 patients screened for the REGENERATE study found that application of two sequential NITs improved the accuracy of identification and reduced misclassification of disease as compared to two simultaneous NITs. The authors concluded that sequential NIT strategies may decrease the need for liver biopsy, while maintaining the accuracy of diagnosis in patients with advanced fibrosis due to NASH.



    • An analysis (Abstract 1589) comparing FIB-4, liver stiffness measurement by transient elastography, and liver biopsy to predict the incidence of liver-related outcomes (e.g., cirrhosis complications and/or hepatocellular carcinoma) in patients with nonalcoholic fatty liver disease concluded that the predictive accuracy of FIB-4 and transient elastography is similar to that of liver biopsy for predicting liver-related events.



    • A review (Abstract 1576) of data from a large U.S. claims database that included approximately 21,500 patients diagnosed with NASH who met the study's inclusion criteria found that only 11% had a liver biopsy, underscoring the fact that liver biopsy is infrequently performed in the real world clinical practice setting. 

    "Strong collaboration among patient groups, academic centers and industry, coupled with large datasets from Phase 3 clinical trials have accelerated our ability to identify and validate noninvasive alternatives to biopsy for our patients with fibrosis due to NASH," said Jerome Boursier, M.D., Ph.D., professor of Medicine, Hepato-Gastroenterology Department of Angers University Hospital, and head of HIFIH laboratory, Angers University in France. "The new NIT data from the interim analysis of the REGENERATE study being presented at the Liver Meeting represent a major step forward. Clearly, the field is coalescing around a sequential testing strategy that combines two commonly used NITs; this approach addresses the major limitations of liver biopsy because it is both scalable and patient-friendly without appearing to sacrifice predictive accuracy. Sequential use of NITs starting with a simple test confirmed by a specialized one will also help to organize and optimize the patient pathway."

    About Liver Fibrosis due to NASH

    Nonalcoholic steatohepatitis (NASH) is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death. Advanced fibrosis is associated with a substantially higher risk of liver-related morbidity and mortality in patients with NASH. In the United States, NASH is currently the second leading cause for liver transplantation overall, and in females, the leading cause. NASH is anticipated to become the leading indication for liver transplantation in Europe within the next decade. There are currently no medications approved for the treatment of NASH.

    About the REGENERATE Study

    REGENERATE is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study assessing the safety and efficacy of obeticholic acid (OCA) on clinical outcomes in patients with liver fibrosis due to NASH, an investigational use. A pre-specified 18-month interim analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. The intent-to-treat population for the interim analysis included 931 patients with stage 2 and 3 fibrosis (placebo, n=311; OCA 10 mg, n=312; OCA 25 mg, n=308). REGENERATE has completed target enrollment for the clinical outcomes cohort, with 2,480 adult NASH patients randomized at 339 qualified centers worldwide, and is expected to continue through clinical outcomes for verification and description of clinical benefit. The end-of-study analysis will evaluate the effect of OCA on all-cause mortality and liver-related clinical outcomes, as well as its long-term safety.

    The safety population of the interim analysis included 1,968 randomized patients who received at least one dose of investigational product (OCA or placebo) with exposures up to 37 months. Adverse events were generally mild to moderate in severity and the most common were consistent with the known profile of OCA. The frequency of serious adverse events was similar across treatment arms (11% in placebo, 11% in OCA 10 mg and 14% in OCA 25 mg), and no serious adverse event occurred in > 1% of patients in any treatment group. There were 3 deaths in the study (2 in placebo: bone cancer and cardiac arrest and 1 in OCA 25 mg: glioblastoma) and none were considered related to treatment. The most common adverse event reported was dose-related pruritus (placebo, 19%; OCA 10 mg, 28%; OCA 25 mg, 51%). The large majority of pruritus events were mild to moderate, with severe pruritus occurring in a small number of patients (< 1% in placebo, < 1% in OCA 10 mg and 5% in OCA 25 mg). A higher incidence of pruritus-associated treatment discontinuation was observed for OCA 25 mg (< 1% in placebo, < 1% in OCA 10 mg, and 9% in OCA 25 mg). Consistent with observations from previous NASH studies, OCA treatment was associated with an increase in low density lipoprotein (LDL) cholesterol, with a peak increase of 22.6 mg/dL at 4 weeks and subsequently reversing and approaching baseline at month 18 (4.0 mg/dL increase from baseline). Triglycerides rapidly and continually decreased in the OCA treatment groups through month 18. There were few and varied serious cardiovascular events and incidence was balanced across the three treatment groups (2% in placebo, 1% in OCA 10 mg and 2% in OCA 25 mg). In patients with type 2 diabetes, OCA treatment was associated with an early transient increase in glucose and hemoglobin A1c with a return to levels similar to placebo by month 6. No clinically meaningful changes were noted in non-diabetic patients. With respect to hepatobiliary events, more patients (3%) on OCA 25 mg experienced gallstones or cholecystitis compared to <1% on placebo and 1% on OCA 10 mg. While hepatic serious adverse events were rare (<1% incidence in each of the three treatment groups), more occurred in the OCA 25 mg group with no pattern attributable to OCA.

    About Intercept

    Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Founded in 2002 in New York, Intercept has operations in the United States, Europe and Canada. For more information, please visit www.interceptpharma.com or connect with the company on Twitter and LinkedIn.

    Cautionary Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements, including, but not limited to, statements regarding the progress, timing and results of our clinical trials, including our clinical trials for the treatment of nonalcoholic steatohepatitis ("NASH"), the safety and efficacy of our approved product, Ocaliva (obeticholic acid or "OCA") for primary biliary cholangitis ("PBC"), and our product candidates, including OCA for liver fibrosis due to NASH, the timing and acceptance of our regulatory filings and the potential approval of OCA for liver fibrosis due to NASH, the review of our New Drug Application for OCA for the treatment of liver fibrosis due to NASH by the U.S. Food and Drug Administration (FDA), our intent to work with the FDA to address the issues raised in the complete response letter (CRL), the potential commercial success of OCA, as well as our strategy, future operations, future financial position, future revenue, projected costs, financial guidance, prospects, plans and objectives.

    These statements constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "possible," "continue" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and we undertake no obligation to update any forward-looking statement except as required by law. These forward-looking statements are based on estimates and assumptions by our management that, although believed to be reasonable, are inherently uncertain and subject to a number of risks. The following represent some, but not necessarily all, of the factors that could cause actual results to differ materially from historical results or those anticipated or predicted by our forward-looking statements: our ability to successfully commercialize Ocaliva for PBC; our ability to maintain our regulatory approval of Ocaliva for PBC in the United States, Europe, Canada, Israel, Australia and other jurisdictions in which we have or may receive marketing authorization; our ability to timely and cost-effectively file for and obtain regulatory approval of our product candidates on an accelerated basis or at all, including OCA for liver fibrosis due to NASH following the issuance of the CRL by the FDA; any advisory committee recommendation or dispute resolution determination that our product candidates, including OCA for liver fibrosis due to NASH, should not be approved or approved only under certain conditions; any future determination that the regulatory applications and subsequent information we submit for our product candidates, including OCA for liver fibrosis due to NASH, do not contain adequate clinical or other data or meet applicable regulatory requirements for approval; conditions that may be imposed by regulatory authorities on our marketing approvals for our products and product candidates, including OCA for liver fibrosis due to NASH, such as the need for clinical outcomes data (and not just results based on achievement of a surrogate endpoint), any risk mitigation programs such as a REMS, and any related restrictions, limitations and/or warnings contained in the label of any of our products or product candidates; any potential side effects associated with Ocaliva for PBC, OCA for liver fibrosis due to NASH or our other product candidates that could delay or prevent approval, require that an approved product be taken off the market, require the inclusion of safety warnings or precautions, or otherwise limit the sale of such product or product candidate; the initiation, timing, cost, conduct, progress and results of our research and development activities, preclinical studies and clinical trials, including any issues, delays or failures in identifying patients, enrolling patients, treating patients, retaining patients, meeting specific endpoints in the jurisdictions in which we intend to seek approval or completing and timely reporting the results of our NASH or PBC clinical trials; our ability to establish and maintain relationships with, and the performance of, third-party manufacturers, contract research organizations and other vendors upon whom we are substantially dependent for, among other things, the manufacture and supply of our products, including Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH, and our clinical trial activities; our ability to identify, develop and successfully commercialize our products and product candidates, including our ability to successfully launch OCA for liver fibrosis due to NASH, if approved; our ability to obtain and maintain intellectual property protection for our products and product candidates, including our ability to cost-effectively file, prosecute, defend and enforce any patent claims or other intellectual property rights; the size and growth of the markets for our products and product candidates and our ability to serve those markets; the degree of market acceptance of Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH or our other product candidates among physicians, patients and healthcare payors; the availability of adequate coverage and reimbursement from governmental and private healthcare payors for our products, including Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH, and our ability to obtain adequate pricing for such products; our ability to establish and maintain effective sales, marketing and distribution capabilities, either directly or through collaborations with third parties; competition from existing drugs or new drugs that become available; our ability to prevent system failures, data breaches or violations of data protection laws; costs and outcomes relating to any disputes, governmental inquiries or investigations, regulatory proceedings, legal proceedings or litigation, including any securities, intellectual property, employment, product liability or other litigation; our collaborators' election to pursue research, development and commercialization activities; our ability to establish and maintain relationships with collaborators with development, regulatory and commercialization expertise; our need for and ability to generate or obtain additional financing; our estimates regarding future expenses, revenues and capital requirements and the accuracy thereof; our use of cash and short-term investments; our ability to acquire, license and invest in businesses, technologies, product candidates and products; our ability to attract and retain key personnel to manage our business effectively; our ability to manage the growth of our operations, infrastructure, personnel, systems and controls; our ability to obtain and maintain adequate insurance coverage; the impact of COVID-19, including any impact on our results of operations or financial position, related quarantines and government actions, delays relating to our regulatory applications, disruptions relating to our ongoing clinical trials or involving our contract research organizations, study sites or other clinical partners, disruptions relating to our supply chain or involving our third-party manufacturers, distributors or other distribution partners, facility closures or other restrictions, and the extent and duration thereof; the impact of general U.S. and foreign economic, industry, market, regulatory or political conditions, including the potential impact of Brexit; and the other risks and uncertainties identified in our periodic filings filed with the U.S. Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2019 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020.

    Contact

    For more information about Intercept, please contact:

    Lisa DeFrancesco

    +1-646-565-4833

    Christopher Frates

    +1-646-757-2371



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  3. Worldwide Ocaliva net sales of $79.5 million in the third quarter 2020, representing 29% growth over the prior year quarter

    Intercept updates 2020 Ocaliva Net Sales Guidance to narrow the range to $310 million to $320 million

    Intercept updates 2020 non-GAAP Adjusted Operating Expenses Guidance to narrow the range to $460 million to $480 million

    Productive Type A Meeting held with FDA on NASH Fibrosis Program

    Conference call scheduled for 8:30 a.m. ET today

    NEW YORK, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced its financial results for the…

    Worldwide Ocaliva net sales of $79.5 million in the third quarter 2020, representing 29% growth over the prior year quarter

    Intercept updates 2020 Ocaliva Net Sales Guidance to narrow the range to $310 million to $320 million

    Intercept updates 2020 non-GAAP Adjusted Operating Expenses Guidance to narrow the range to $460 million to $480 million

    Productive Type A Meeting held with FDA on NASH Fibrosis Program

    Conference call scheduled for 8:30 a.m. ET today

    NEW YORK, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced its financial results for the quarter ended September 30, 2020.

    "We were pleased to reengage with FDA at our recent Type A end-of-review meeting to discuss the Agency's benefit-risk assessment in the CRL based on its review of the available data, as well as our proposed way forward to resubmitting our NDA for OCA in NASH fibrosis," said Mark Pruzanski, M.D., President and Chief Executive Officer of Intercept. "The meeting was constructive and FDA has provided us with helpful guidance regarding supplemental data we can provide to further characterize OCA's efficacy and safety profile that could support resubmission based on our Phase 3 REGENERATE 18-month biopsy data, together with a safety update from our ongoing studies. We are advancing accordingly and plan to hold additional meetings with the Agency with the goal of achieving sufficient alignment to proceed on this basis and potentially resubmit our NDA next year. Of note, the REGENERATE outcomes phase is ongoing and our Phase 3 REVERSE study in NASH patients with compensated cirrhosis is expected to read out by the end of next year. We continue to lead the NASH field and believe that, if approved, OCA has the potential to become an important treatment for patients with advanced fibrosis due to NASH. Meanwhile, our PBC business continued its strong performance in the third quarter and we have updated our 2020 financial guidance with anticipated Ocaliva net sales of $310 to $320 million and non-GAAP adjusted operating expenses of $460 to $480 million, providing us with a strong financial footing for the future."

    Ocaliva® (obeticholic acid) Commercial Highlights

    We recognized $79.5 million of Ocaliva net sales in the third quarter of 2020, as compared to $61.5 million in the prior year quarter. Ocaliva net sales in the third quarter of 2020 were comprised of U.S. net sales of $58.6 million and ex-U.S. net sales of $20.9 million, as compared to U.S. net sales of $45.2 million and ex-U.S. net sales of $16.3 million in the prior year quarter.

    Selected Third Quarter 2020 Financial Results 

    Revenues 

    We recognized $79.5 million in total revenue in the third quarter of 2020, as compared to $61.9 million in total revenue in the prior year quarter. Total revenue in the third quarter of 2019 included approximately $0.4 million of licensing revenue.

    Operating Expenses

    Our cost of sales was $1.8 million in the third quarter of 2020, as compared to $0.5 million in the prior year quarter. Our cost of sales for the quarters ended September 30, 2020 and 2019 consisted primarily of packaging, labeling, materials and related expenses.

    Our selling, general and administrative expenses decreased to $70.6 million in the third quarter of 2020, from $76.8 million in the prior year quarter. The decrease was primarily driven by reductions in spend resulting from the delay of the potential approval and commercialization of obeticholic acid (OCA) for liver fibrosis due to nonalcoholic steatohepatitis (NASH).

    Our research and development expenses decreased to $48.9 million in the third quarter of 2020, from $60.2 million in the prior year quarter. The decrease was primarily driven by lower NASH development costs, including the conclusion of enrollment activities for the REGENERATE and REVERSE studies and reduced costs related to our preparations for regulatory interactions.

    Restructuring expenses were $13.4 million and $0 for the three months ended September 30, 2020 and 2019, respectively. The increase between periods was driven primarily by severance costs and other related termination benefits incurred in conjunction with the previously announced plan to reduce workforce in response to the receipt of the complete response letter.

    In the quarters ended September 30, 2020 and 2019, we recorded $134.7 million and $137.5 million, respectively, in total operating expenses and $118.1 million and $123.4 million, respectively, in non-GAAP adjusted operating expenses, which excludes non-cash stock-based compensation expense of $15.8 million and $13.1 million, respectively, and depreciation expense of $0.7 million and $0.9 million, respectively.

    References in this press release to "non-GAAP adjusted operating expenses" mean our total operating expenses, as calculated and presented in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"), adjusted for the effects of two non-cash items: stock-based compensation and depreciation. See "Non-GAAP Financial Measures" below. A reconciliation of non-GAAP adjusted operating expenses to total operating expenses for all historical periods presented is included below under the heading "Reconciliation of Non-GAAP Adjusted Operating Expenses to Total Operating Expenses."

    Interest Expense

    Interest expense in the quarters ended September 30, 2020 and 2019 was $12.1 million and $11.8 million, respectively. For the quarter ended September 30, 2020, interest expense related to the $460.0 million aggregate principal amount of 3.25% Convertible Senior Notes due 2023 that we issued in July 2016 and the $230.0 million aggregate principal amount of 2.00% Convertible Senior Notes due 2026 (the "2026 Convertible Notes") that we issued in May 2019.



    Net Loss

    In the third quarter of 2020 we reported a net loss of $66.5 million, a decrease compared to a net loss of $84.8 million in the third quarter 2019.

    Cash Position

    As of September 30, 2020, we had cash, cash equivalents, restricted cash, and investment debt securities available for sale of approximately $496.8 million. As of December 31, 2019, we had cash, cash equivalents, restricted cash, and investment debt securities available for sale of approximately $657.3 million.

    2020 Financial Guidance

    We are updating our 2020 Ocaliva net sales guidance to narrow the range at the upper end of our previously announced guidance, and now anticipate Ocaliva net sales of $310 million to $320 million. We are also updating our non-GAAP adjusted operating expenses guidance to narrow the range at the lower end of the previously announced guidance, and now anticipate non-GAAP adjusted operating expenses of $460 million to $480 million.

    See "Non-GAAP Financial Measures" below. A quantitative reconciliation of projected non-GAAP adjusted operating expenses to total operating expenses is not available without unreasonable effort primarily due to our inability to predict with reasonable certainty the amount of future stock-based compensation expense.

    Conference Call on November 9, 2020 at 8:30 a.m. ET

    We are hosting our third quarter 2020 financial results conference call and webcast on November 9, 2020 at 8:30 a.m. ET. The conference call will be available on the investor page of our website at http://ir.interceptpharma.com or by calling (855) 232-3919 (toll-free domestic) or (315) 625-6894 (international) passcode 3798851. A replay of the call will be available on our website shortly following the completion of the call and will be available for two weeks.

    About Intercept

    Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Founded in 2002 in New York, Intercept has operations in the United States, Europe and Canada. For more information, please visit www.interceptpharma.com or connect with the company on Twitter and LinkedIn.

    Non-GAAP Financial Measures

    This press release presents non-GAAP adjusted operating expenses on a historical and projected basis. For the periods presented, non-GAAP adjusted operating expenses exclude from total operating expenses, as calculated and presented in accordance with GAAP, the effects of two non-cash items: stock-based compensation and depreciation. Non-GAAP adjusted operating expenses is a financial measure that has not been prepared in accordance with GAAP. Accordingly, investors should consider non-GAAP adjusted operating expenses in addition to, but not as a substitute for, total operating expenses that we calculate and present in accordance with GAAP. Among other things, our management uses non-GAAP adjusted operating expenses to establish budgets and operational goals and to manage our business. Other companies may define or use this measure in different ways. We believe that the presentation of non-GAAP adjusted operating expenses provides investors and management with helpful supplemental information relating to operating performance and trends. A table reconciling non-GAAP adjusted operating expenses to total operating expenses for all historical periods presented is included below under the heading "Reconciliation of Non-GAAP Adjusted Operating Expenses to Total Operating Expenses". A quantitative reconciliation of projected non-GAAP adjusted operating expenses to total operating expenses is not available without unreasonable effort primarily due to our inability to predict with reasonable certainty the amount of future stock-based compensation expense.

    About Liver Fibrosis due to NASH

    Nonalcoholic steatohepatitis (NASH) is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death. Advanced fibrosis is associated with a substantially higher risk of liver-related morbidity and mortality in patients with NASH. In the United States, NASH is currently the second leading cause for liver transplantation overall, and in females, the leading cause. NASH is anticipated to become the leading indication for liver transplantation in Europe within the next decade. There are currently no medications approved for the treatment of NASH.

    About the REGENERATE Study

    REGENERATE is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study assessing the safety and efficacy of obeticholic acid (OCA) on clinical outcomes in patients with liver fibrosis due to NASH. A pre-specified 18-month analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. REGENERATE has completed target enrollment for the clinical outcomes cohort, with 2,480 adult NASH patients randomized at over 300 qualified centers worldwide, and is expected to continue through clinical outcomes for verification and description of clinical benefit. The end-of-study analysis will evaluate the effect of OCA on all-cause mortality and liver-related clinical outcomes, as well as long-term safety.

    About Ocaliva® (obeticholic acid)

    Ocaliva is indicated in the United States for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.

    This indication is approved under the accelerated approval pathway based on a reduction in alkaline phosphatase (ALP) as a surrogate endpoint which is reasonably likely to predict clinical benefit, including an improvement in liver transplant free-survival. An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. We are conducting a Phase 4 clinical outcomes trial, which we refer to as our COBALT trial, of OCA in patients with PBC with the goal of confirming clinical benefit on a post-marketing basis.

    In December 2016, Ocaliva received conditional marketing authorization in Europe for the treatment of PBC in combination with UDCA in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA, conditioned upon us providing further data post-approval to confirm benefit. For detailed safety information for Ocaliva 5 mg and 10 mg tablets including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European Summary of Product Characteristics that can be found on www.ema.europa.eu.

    U.S. IMPORTANT SAFETY INFORMATION FOR OCALIVA IN PBC

    WARNING: HEPATIC DECOMPENSATION AND FAILURE IN INCORRECTLY DOSED PBC PATIENTS WITH CHILD-PUGH CLASS B OR C OR DECOMPENSATED CIRRHOSIS

    • In postmarketing reports, hepatic decompensation and failure, in some cases fatal, have been reported in patients with Primary Biliary Cholangitis (PBC) with decompensated cirrhosis or Child-Pugh Class B or C hepatic impairment when OCALIVA was dosed more frequently than recommended.
    • The recommended starting dosage of OCALIVA is 5 mg once weekly for patients with Child-Pugh Class B or C hepatic impairment or a prior decompensation event.

    Contraindications

    OCALIVA is contraindicated in PBC patients with complete biliary obstruction.

    Warnings and Precautions

    Hepatic Decompensation and Failure in Incorrectly-Dosed PBC Patients with Child-Pugh Class B or C or Decompensated Cirrhosis

    In postmarketing reports, hepatic decompensation and failure, in some cases fatal, have been reported in PBC patients with decompensated cirrhosis or Child-Pugh B or C hepatic impairment when OCALIVA was dosed more frequently than the recommended starting dosage of 5 mg once weekly. Reported cases typically occurred within 2 to 5 weeks after starting OCALIVA and were characterized by an acute increase in total bilirubin and/or ALP concentrations in association with clinical signs and symptoms of hepatic decompensation (e.g., ascites, jaundice, gastrointestinal bleeding, worsening of hepatic encephalopathy).

    Routinely monitor patients for progression of PBC disease, including liver-related complications, with laboratory and clinical assessments. Dosage adjustment, interruption or discontinuation may be required. Close monitoring is recommended for patients at an increased risk of hepatic decompensation. Severe intercurrent illnesses that may worsen renal function or cause dehydration (e.g., gastroenteritis), may exacerbate the risk of hepatic decompensation. Interrupt treatment with OCALIVA in patients with laboratory or clinical evidence of worsening liver function indicating risk of decompensation, and monitor the patient's liver function. Consider discontinuing OCALIVA in patients who have experienced clinically significant liver-related adverse reactions. Discontinue OCALIVA in patients who develop complete biliary obstruction.

    Liver-Related Adverse Reactions

    Dose-related, liver-related adverse reactions including jaundice, worsening ascites and primary biliary cholangitis flare have been observed in clinical trials, as early as one month after starting treatment with OCALIVA 10 mg once daily up to 50 mg once daily (up to 5-times the highest recommended dosage). Monitor PBC patients during treatment with OCALIVA for elevations in liver biochemical tests and for the development of liver-related adverse reactions.

    Severe Pruritus

    Severe pruritus was reported in 23% of PBC patients in the OCALIVA 10 mg arm, 19% of PBC patients in the OCALIVA titration arm, and 7% of PBC patients in the placebo arm in a 12-month double-blind randomized controlled trial of 216 PBC patients. Severe pruritus was defined as intense or widespread itching, interfering with activities of daily living, or causing severe sleep disturbance, or intolerable discomfort, and typically requiring medical interventions. Consider clinical evaluation of PBC patients with new onset or worsening severe pruritus. Management strategies include the addition of bile acid resins or antihistamines, OCALIVA dosage reduction, and/or temporary interruption of OCALIVA dosing.

    Reduction in HDL-C

    Patients with PBC generally exhibit hyperlipidemia characterized by a significant elevation in total cholesterol primarily due to increased levels of high-density lipoprotein-cholesterol (HDL-C). Dose-dependent reductions from baseline in mean HDL-C levels were observed at 2 weeks in OCALIVA-treated PBC patients, 20% and 9% in the 10 mg and titration arms, respectively, compared to 2% in the placebo arm. Monitor PBC patients for changes in serum lipid levels during treatment. For PBC patients who do not respond to OCALIVA after 1 year at the highest recommended dosage that can be tolerated (maximum of 10 mg once daily), and who experience a reduction in HDL-C, weigh the potential risks against the benefits of continuing treatment.

    Adverse Reactions

    The most common adverse reactions from subjects taking OCALIVA for PBC were pruritus, fatigue, abdominal pain and discomfort, rash, oropharyngeal pain, dizziness, constipation, arthralgia, thyroid function abnormality, and eczema.

    Drug Interactions

    Bile Acid Binding Resins

    Bile acid binding resins such as cholestyramine, colestipol, or colesevelam adsorb and reduce bile acid absorption and may reduce the absorption, systemic exposure, and efficacy of OCALIVA. If taking a bile acid binding resin, take OCALIVA at least 4 hours before or 4 hours after taking the bile acid binding resin, or at as great an interval as possible.

    Warfarin

    The International Normalized Ratio (INR) decreased following coadministration of warfarin and OCALIVA. Monitor INR and adjust the dose of warfarin, as needed, to maintain the target INR range when coadministering OCALIVA and warfarin.

    CYP1A2 Substrates with Narrow Therapeutic Index

    Obeticholic acid, the active ingredient in OCALIVA, may increase the exposure to concomitant drugs that are CYP1A2 substrates. Therapeutic monitoring of CYP1A2 substrates with a narrow therapeutic index (e.g. theophylline and tizanidine) is recommended when coadministered with OCALIVA.

    Inhibitors of Bile Salt Efflux Pump

    Avoid concomitant use of inhibitors of the bile salt efflux pump (BSEP) such as cyclosporine. Concomitant medications that inhibit canalicular membrane bile acid transporters such as the BSEP may exacerbate accumulation of conjugated bile salts including taurine conjugate of obeticholic acid in the liver and result in clinical symptoms. If concomitant use is deemed necessary, monitor serum transaminases and bilirubin.

    Please see Full Prescribing Information, including Boxed WARNING and Medication Guide for OCALIVA.

    To report SUSPECTED ADVERSE REACTIONS, contact Intercept Pharmaceuticals, Inc. at 1-844-782-ICPT or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    Cautionary Note Regarding Forward-Looking Statements



    This press release contains forward-looking statements, including, but not limited to, statements regarding the progress, timing and results of our clinical trials, including our clinical trials for the treatment of nonalcoholic steatohepatitis ("NASH"), the safety and efficacy of our approved product, Ocaliva (obeticholic acid or "OCA") for primary biliary cholangitis ("PBC"), and our product candidates, including OCA for liver fibrosis due to NASH, the timing and acceptance of our regulatory filings and the potential approval of OCA for liver fibrosis due to NASH, the review of our New Drug Application for OCA for the treatment of liver fibrosis due to NASH by the U.S. Food and Drug Administration (FDA), our intent to work with the FDA to address the issues raised in the complete response letter (CRL), the potential commercial success of OCA, as well as our strategy, future operations, future financial position, future revenue, projected costs, financial guidance, prospects, plans and objectives.

    These statements constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "possible," "continue" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and we undertake no obligation to update any forward-looking statement except as required by law. These forward-looking statements are based on estimates and assumptions by our management that, although believed to be reasonable, are inherently uncertain and subject to a number of risks. The following represent some, but not necessarily all, of the factors that could cause actual results to differ materially from historical results or those anticipated or predicted by our forward-looking statements: our ability to successfully commercialize Ocaliva for PBC; our ability to maintain our regulatory approval of Ocaliva for PBC in the United States, Europe, Canada, Israel, Australia and other jurisdictions in which we have or may receive marketing authorization; our ability to timely and cost-effectively file for and obtain regulatory approval of our product candidates on an accelerated basis or at all, including OCA for liver fibrosis due to NASH following the issuance of the CRL by the FDA; any advisory committee recommendation or dispute resolution determination that our product candidates, including OCA for liver fibrosis due to NASH, should not be approved or approved only under certain conditions; any future determination that the regulatory applications and subsequent information we submit for our product candidates, including OCA for liver fibrosis due to NASH, do not contain adequate clinical or other data or meet applicable regulatory requirements for approval; conditions that may be imposed by regulatory authorities on our marketing approvals for our products and product candidates, including OCA for liver fibrosis due to NASH, such as the need for clinical outcomes data (and not just results based on achievement of a surrogate endpoint), any risk mitigation programs such as a REMS, and any related restrictions, limitations and/or warnings contained in the label of any of our products or product candidates; any potential side effects associated with Ocaliva for PBC, OCA for liver fibrosis due to NASH or our other product candidates that could delay or prevent approval, require that an approved product be taken off the market, require the inclusion of safety warnings or precautions, or otherwise limit the sale of such product or product candidate; the initiation, timing, cost, conduct, progress and results of our research and development activities, preclinical studies and clinical trials, including any issues, delays or failures in identifying patients, enrolling patients, treating patients, retaining patients, meeting specific endpoints in the jurisdictions in which we intend to seek approval or completing and timely reporting the results of our NASH or PBC clinical trials; our ability to establish and maintain relationships with, and the performance of, third-party manufacturers, contract research organizations and other vendors upon whom we are substantially dependent for, among other things, the manufacture and supply of our products, including Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH, and our clinical trial activities; our ability to identify, develop and successfully commercialize our products and product candidates, including our ability to successfully launch OCA for liver fibrosis due to NASH, if approved; our ability to obtain and maintain intellectual property protection for our products and product candidates, including our ability to cost-effectively file, prosecute, defend and enforce any patent claims or other intellectual property rights; the size and growth of the markets for our products and product candidates and our ability to serve those markets; the degree of market acceptance of Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH or our other product candidates among physicians, patients and healthcare payors; the availability of adequate coverage and reimbursement from governmental and private healthcare payors for our products, including Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH, and our ability to obtain adequate pricing for such products; our ability to establish and maintain effective sales, marketing and distribution capabilities, either directly or through collaborations with third parties; competition from existing drugs or new drugs that become available; our ability to prevent system failures, data breaches or violations of data protection laws; costs and outcomes relating to any disputes, governmental inquiries or investigations, regulatory proceedings, legal proceedings or litigation, including any securities, intellectual property, employment, product liability or other litigation; our collaborators' election to pursue research, development and commercialization activities; our ability to establish and maintain relationships with collaborators with development, regulatory and commercialization expertise; our need for and ability to generate or obtain additional financing; our estimates regarding future expenses, revenues and capital requirements and the accuracy thereof; our use of cash and short-term investments; our ability to acquire, license and invest in businesses, technologies, product candidates and products; our ability to attract and retain key personnel to manage our business effectively; our ability to manage the growth of our operations, infrastructure, personnel, systems and controls; our ability to obtain and maintain adequate insurance coverage; the impact of COVID-19, including any impact on our results of operations or financial position, related quarantines and government actions, delays relating to our regulatory applications, disruptions relating to our ongoing clinical trials or involving our contract research organizations, study sites or other clinical partners, disruptions relating to our supply chain or involving our third-party manufacturers, distributors or other distribution partners, facility closures or other restrictions, and the extent and duration thereof; the impact of general U.S. and foreign economic, industry, market, regulatory or political conditions, including the potential impact of Brexit; and the other risks and uncertainties identified in our periodic filings filed with the U.S. Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2019 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020.

    Contact

    For more information about Intercept, please contact:

    Lisa DeFrancesco

    +1-646-565-4833

    Christopher Frates

    +1-646-757-2371





    Intercept Pharmaceuticals, Inc.

    Condensed Consolidated Statements of Operations

    (Unaudited)

    (In thousands, except per share data)

      Three Months Ended

    September 30,


      Nine Months Ended

    September 30,


      2020   2019   2020   2019 
    Revenue:               
    Product revenue, net$79,521  $61,545  $229,422  $179,286 
    Licensing revenue -   405   -   1,216 
    Total revenue 79,521   61,950   229,422   180,502 
                    
    Operating expenses:               
    Cost of sales 1,826   487   4,555   1,738 
    Selling, general and administrative 70,619   76,828   262,537   223,738 
    Research and development 48,858   60,168   139,587   178,163 
    Restructuring 13,381   -   13,381   - 
    Total operating expenses 134,684   137,483   420,060   403,639 
    Operating loss (55,163)  (75,533)  (190,638)  (223,137)
                    
    Other income (expense):               
    Interest expense (12,091)  (11,795)  (35,801)  (29,518)
    Other income, net 785   2,495   3,706   6,132 
      (11,306)  (9,300)  (32,095)  (23,386)
    Net loss$(66,469) $(84,833) $(222,733) $(246,523)
                    
    Net loss per common and potential common share:               
    Basic and diluted$(2.01) $(2.59) $(6.76) $(7.88)
                    
    Weighted average common and potential common shares outstanding:               
    Basic and diluted 32,989   32,717   32,957   31,275 





    Condensed Consolidated Balance Sheet Information

    (In thousands)

      September 30,

    2020
       December 31,

    2019 (1)
     
      (Unaudited)     
    Cash, cash equivalents, restricted cash and investment debt securities, available for sale$496,760  $657,347 
    Total assets$591,352  $754,886 
    Total liabilities (2)$721,639  $703,330 
    Stockholders' (deficit) equity$(130,287) $51,556 

    ______________

    (1)



    Derived from the audited financial statements included in Intercept's Annual Report on Form 10-K for the year ended December 31, 2019



    (2)Includes $553.2 million and $532.1 million related to the 2023 Convertible Notes and the 2026 Convertible Notes (together, the "Convertible Notes") as of September 30, 2020 and December 31, 2019, respectively. Intercept separately accounts for the debt and equity components of the Convertible Notes. The aggregate outstanding principal amount of the Convertible Notes was $690.0 million as of September 30, 2020, and December 31, 2019.





    Reconciliation of Non-GAAP Adjusted Operating Expenses to Total Operating Expenses

    (Unaudited)

    (In thousands)

     Three Months Ended

    September 30,

     Nine Months Ended

    September 30,

      2020   2019   2020   2019 
    Total operating expenses$134,684  $137,483  $420,060  $403,639 
                    
    Adjustments:               
    Stock-based compensation 15,825   13,130   44,381   42,809 
    Depreciation 710   914   2,282   2,839 
    Non-GAAP adjusted operating expenses$118,149  $123,439  $373,397  $357,991 

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  4. NEW YORK, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, will announce its third quarter 2020 financial results prior to market open on Monday, November 9, 2020. The announcement will be followed by a conference call and audio webcast hosted by Intercept management at 8:30 a.m. ET to discuss the results.

    The conference call will be available on the investor page of our website at http://ir.interceptpharma.com or by calling (855) 232-3919 (toll-free domestic) or (315) 625-6894 (international) passcode 3798851. Archived webcasts will be available on Intercept's website…

    NEW YORK, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, will announce its third quarter 2020 financial results prior to market open on Monday, November 9, 2020. The announcement will be followed by a conference call and audio webcast hosted by Intercept management at 8:30 a.m. ET to discuss the results.

    The conference call will be available on the investor page of our website at http://ir.interceptpharma.com or by calling (855) 232-3919 (toll-free domestic) or (315) 625-6894 (international) passcode 3798851. Archived webcasts will be available on Intercept's website for approximately two weeks.

    About Intercept

    Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Founded in 2002 in New York, Intercept has operations in the United States, Europe and Canada. For more information, please visit www.interceptpharma.com or connect with the company on Twitter and LinkedIn.

    CONTACT

    For more information about Intercept, please contact:

    Investor inquiries: 

    Media inquiries:

    Source: Intercept Pharmaceuticals, Inc.

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  5. Presentation of long-term efficacy and safety data of obeticholic acid (OCA) for the treatment of patients with PBC

    Analysis from Phase 3 REGENERATE study examines the antifibrotic efficacy of OCA on noninvasive measures of liver fibrosis in patients with fibrosis due to NASH

    NEW YORK, Oct. 29, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that multiple abstracts evaluating OCA for the treatment of primary biliary cholangitis (PBC) and fibrosis due to nonalcoholic steatohepatitis (NASH) will be presented at The Liver Meeting Digital Experience™, the Annual…

    Presentation of long-term efficacy and safety data of obeticholic acid (OCA) for the treatment of patients with PBC

    Analysis from Phase 3 REGENERATE study examines the antifibrotic efficacy of OCA on noninvasive measures of liver fibrosis in patients with fibrosis due to NASH

    NEW YORK, Oct. 29, 2020 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that multiple abstracts evaluating OCA for the treatment of primary biliary cholangitis (PBC) and fibrosis due to nonalcoholic steatohepatitis (NASH) will be presented at The Liver Meeting Digital Experience™, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which will be held virtually from November 13-16, 2020.

    "These 13 presentations at this year's AASLD meeting demonstrate the continuing strong interest in the advancement of clinical research in both PBC and fibrosis due to NASH," said Mark Pruzanski, M.D., President and CEO of Intercept. "In particular, we look forward to the presentation of the Ocaliva efficacy and safety data in patients with PBC treated for up to six years. We're also pleased to see the presentation of new data from the interim analysis of the Phase 3 REGENERATE study highlighting the potential role of noninvasive tests in the identification of patients with advanced fibrosis due to NASH and the measurement of OCA treatment response."

    Presentations at The Liver Meeting Digital Experience™ include:

    NASH

    Oral presentations

    Sunday, November 15, 2020 10:30 a.m. ET

    Evaluation of obeticholic acid efficacy in patients with NASH who were monitored using noninvasive tests: a post hoc analysis of the REGENERATE trial (Abstract 70)

    Naim Alkhouri, Arun J. Sanyal, Vlad Ratziu, Mary Rinella, Rohit Loomba, Jean-Francois Dufour, Essy Mozaffari, Tanya Granston, Huafeng Zhou, Reshma Shringarpure, Leigh MacConell, Pierre Bedossa, Zachary Goodman, Zobair Younossi, Quentin M. Anstee, Natasha von Roenn, Stephen Harrison

    Saturday, November 14, 2020 5:30 p.m. ET

    Noninvasive assessments to identify patients with advanced fibrosis due to NASH: screened population from the REGENERATE trial (Abstract 56)

    Jerome Boursier, Arun J. Sanyal, Vlad Ratziu, Mary Rinella, Rohit Loomba, Jean-Francois Dufour, Essy Mozaffari, Reshma Shringarpure, Leigh MacConell, Tanya Granston, Huafeng Zhou, Aldo Trylesinski, Stephen A. Harrison, Pierre Bedossa, Zachary Goodman, Zobair Younossi, Mazen Noureddin, Elisabetta Bugianesi, Quentin M. Anstee

    Poster presentations

    Economic burden of NASH-associated cirrhosis: US payor's perspective (Abstract 1584)

    Essy Mozaffari, Amy Law, Melissa Lingohr-Smith, Amarita Randhawa, Bryan Velez de Villa, Jay Lin

    Predicted long-term clinical outcomes of obeticholic acid (OCA) for the treatment of patients with advanced fibrosis without cirrhosis due to non-alcoholic steatohepatitis (NASH) compared to standard of care in the USA (Abstract 1542)

    A. Sidney Barritt, Diana Brixner, Mazen Noureddin, Nathorn Chaiyakunapruk, Raluca Pais, William Green, Pablo Anaya, Sandrine Cure, Marcie Strauss, Richard Zur

    Identification of patients with non-alcoholic steatohepatitis (NASH) in an electronic health record (EHR) database (Abstract 1599)

    Monica L. Bertoia, John D. Seeger, Erik Ness, Thomas Capozza, Bruce Wong, Lina Titievsky

    Characteristics and care patterns of real-world non-alcoholic steatohepatitis (NASH) patients with and without liver biopsy (Abstract 1576)

    Diana Esposito, Lina Titievsky, Simo Du, Yuval A. Patel, Bruce Wong, Thomas Capozza, Erik Ness, Kerrin Gallagher, Aziza Jamal-Allial, Mindie H. Nguyen

    FIB-4 and FibroScan can identify patients with nonalcoholic fatty liver disease who are at risk of liver-related events: results of a longitudinal analysis with comparison with liver biopsy (Abstract 1589)

    Jerome Boursier, Hannes Hagström, Mattias Ekstedt, Clemence Moreau, Martin Bonacci, Sandrine Cure, Aldo Trylesinski, Manuel Romero-Gómez

    Identification of undiagnosed NASH: development and application of a real-world prediction model (Abstract 1520)

    Kalé Kponee-Shovein, Priyanka Bobbili, Amy Law, Bruce Wong, Lina Titievsky, Essy Mozaffari, Amarita Randhawa, Pamela Davis, François Laliberté, Maya Mahin, Iman Fakih, Mei Sheng Duh

    Fecal microbiome signature for NASH based on analysis of a REGENERATE sub-study compared with three healthy control populations (Abstract 1510)

    Gary Wu, Yun Li, Hongzhe Li, Kyle Bittinger, Rotonya Carr, Lillian Chau, Elliot S. Friedman, Jung-Jin Lee, Luciano Adorini, Mary Erickson, Luna Zaru, Reshma Shringarpure, Leigh MacConell

    PBC

    Poster presentations

    Long-term efficacy and safety of obeticholic acid in patients with PBC from the POISE trial grouped biochemically by risk of disease progression (Abstract 1250)

    Christopher L. Bowlus, Michael Trauner, Alexander Liberman, Elizabeth Malecha, Leigh MacConell, Andreas E. Kremer, Frederik Nevens

    Long-term efficacy and safety of obeticholic acid in primary biliary cholangitis: responder analysis of over 5 years of treatment in the POISE trial (Abstract 1251)

    Bettina Hansen, David Jones, Marco Carbone, Christopher L. Bowlus, Frederik Nevens, Andreas E. Kremer, Alexander Liberman, Leigh MacConell, Gideon Hirschfield

    Primary biliary cholangitis: patient characteristics and the healthcare economic burden in the United States (Abstract 1259)

    Robert G. Gish, Amy Law, Melissa Lingohr-Smith, Femi Adekunle, Darren Wheeler, Bryan Velez de Villa, Chiara Bassanelli, Jay Lin

    Real-world effectiveness of obeticholic acid in patients with primary biliary cholangitis (Abstract 1268)

    Robert G. Gish, Amy Law, Femi Adekunle, Darren Wheeler, Melissa Lingohr-Smith, Chiara Bassanelli, Bryan Velez de Villa, Jay Lin

    A full list of sessions at The Liver Meeting Digital Experience™ is available at https://tlmdx.aasld.org/.

    About Intercept

    Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Founded in 2002 in New York, Intercept has operations in the United States, Europe and Canada. For more information, please visit www.interceptpharma.com or connect with the company on Twitter and LinkedIn.

    About Liver Fibrosis due to NASH

    Nonalcoholic steatohepatitis (NASH) is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death. Advanced fibrosis is associated with a substantially higher risk of liver-related morbidity and mortality in patients with NASH. In the United States, NASH is currently the second leading cause for liver transplantation overall, and in females, the leading cause. NASH is anticipated to become the leading indication for liver transplantation in Europe within the next decade. There are currently no medications approved for the treatment of NASH.

    About the REGENERATE Study

    REGENERATE is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study assessing the safety and efficacy of obeticholic acid (OCA) on clinical outcomes in patients with liver fibrosis due to NASH. A pre-specified 18-month analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. REGENERATE has completed target enrollment for the clinical outcomes cohort, with 2,480 adult NASH patients randomized over 300 qualified centers worldwide, and is expected to continue through clinical outcomes for verification and description of clinical benefit. The end-of-study analysis is designed to evaluate the effect of OCA on all-cause mortality and liver-related clinical outcomes, as well as long-term safety.

    About Primary Biliary Cholangitis

    Primary biliary cholangitis (PBC) is a chronic, progressive liver disorder that mostly affects women, afflicting approximately one in 1,000 women over the age of 40. If left untreated, survival of PBC patients is significantly worse than the general population.

    About Ocaliva® (obeticholic acid) 

    Ocaliva is indicated in the United States for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.

    This indication is approved under the accelerated approval pathway based on a reduction in alkaline phosphatase (ALP) as a surrogate endpoint which is reasonably likely to predict clinical benefit, including an improvement in liver transplant free-survival. An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. We are conducting a Phase 4 clinical outcomes trial, which we refer to as our COBALT trial, of OCA in patients with PBC with the goal of confirming clinical benefit on a post-marketing basis.

    In December 2016, Ocaliva received conditional marketing authorization in Europe for the treatment of PBC in combination with UDCA in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA, conditioned upon us providing further data post-approval to confirm benefit. For detailed safety information for Ocaliva 5 mg and 10 mg tablets including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European Summary of Product Characteristics that can be found on www.ema.europa.eu.

    U.S. IMPORTANT SAFETY INFORMATION FOR OCALIVA IN PBC

    WARNING: HEPATIC DECOMPENSATION AND FAILURE IN INCORRECTLY DOSED PBC PATIENTS WITH CHILD-PUGH CLASS B OR C OR DECOMPENSATED CIRRHOSIS

    • In postmarketing reports, hepatic decompensation and failure, in some cases fatal, have been reported in patients with Primary Biliary Cholangitis (PBC) with decompensated cirrhosis or Child-Pugh Class B or C hepatic impairment when OCALIVA was dosed more frequently than recommended.
    • The recommended starting dosage of OCALIVA is 5 mg once weekly for patients with Child-Pugh Class B or C hepatic impairment or a prior decompensation event.

    Contraindications

    OCALIVA is contraindicated in PBC patients with complete biliary obstruction.

    Warnings and Precautions

    Hepatic Decompensation and Failure in Incorrectly-Dosed PBC Patients with Child-Pugh Class B or C or Decompensated Cirrhosis

    In postmarketing reports, hepatic decompensation and failure, in some cases fatal, have been reported in PBC patients with decompensated cirrhosis or Child-Pugh B or C hepatic impairment when OCALIVA was dosed more frequently than the recommended starting dosage of 5 mg once weekly. Reported cases typically occurred within 2 to 5 weeks after starting OCALIVA and were characterized by an acute increase in total bilirubin and/or ALP concentrations in association with clinical signs and symptoms of hepatic decompensation (e.g., ascites, jaundice, gastrointestinal bleeding, worsening of hepatic encephalopathy).

    Routinely monitor patients for progression of PBC disease, including liver-related complications, with laboratory and clinical assessments. Dosage adjustment, interruption or discontinuation may be required. Close monitoring is recommended for patients at an increased risk of hepatic decompensation. Severe intercurrent illnesses that may worsen renal function or cause dehydration (e.g., gastroenteritis), may exacerbate the risk of hepatic decompensation. Interrupt treatment with OCALIVA in patients with laboratory or clinical evidence of worsening liver function indicating risk of decompensation, and monitor the patient's liver function. Consider discontinuing OCALIVA in patients who have experienced clinically significant liver-related adverse reactions. Discontinue OCALIVA in patients who develop complete biliary obstruction.

    Liver-Related Adverse Reactions

    Dose-related, liver-related adverse reactions including jaundice, worsening ascites and primary biliary cholangitis flare have been observed in clinical trials, as early as one month after starting treatment with OCALIVA 10 mg once daily up to 50 mg once daily (up to 5-times the highest recommended dosage). Monitor PBC patients during treatment with OCALIVA for elevations in liver biochemical tests and for the development of liver-related adverse reactions.

    Severe Pruritus

    Severe pruritus was reported in 23% of PBC patients in the OCALIVA 10 mg arm, 19% of PBC patients in the OCALIVA titration arm, and 7% of PBC patients in the placebo arm in a 12-month double-blind randomized controlled trial of 216 PBC patients. Severe pruritus was defined as intense or widespread itching, interfering with activities of daily living, or causing severe sleep disturbance, or intolerable discomfort, and typically requiring medical interventions. Consider clinical evaluation of PBC patients with new onset or worsening severe pruritus. Management strategies include the addition of bile acid resins or antihistamines, OCALIVA dosage reduction, and/or temporary interruption of OCALIVA dosing.

    Reduction in HDL-C

    Patients with PBC generally exhibit hyperlipidemia characterized by a significant elevation in total cholesterol primarily due to increased levels of high-density lipoprotein-cholesterol (HDL-C). Dose-dependent reductions from baseline in mean HDL-C levels were observed at 2 weeks in OCALIVA-treated PBC patients, 20% and 9% in the 10 mg and titration arms, respectively, compared to 2% in the placebo arm. Monitor PBC patients for changes in serum lipid levels during treatment. For PBC patients who do not respond to OCALIVA after 1 year at the highest recommended dosage that can be tolerated (maximum of 10 mg once daily), and who experience a reduction in HDL-C, weigh the potential risks against the benefits of continuing treatment.

    Adverse Reactions

    The most common adverse reactions from subjects taking OCALIVA for PBC were pruritus, fatigue, abdominal pain and discomfort, rash, oropharyngeal pain, dizziness, constipation, arthralgia, thyroid function abnormality, and eczema. 

    Drug Interactions

    Bile Acid Binding Resins

    Bile acid binding resins such as cholestyramine, colestipol, or colesevelam adsorb and reduce bile acid absorption and may reduce the absorption, systemic exposure, and efficacy of OCALIVA. If taking a bile acid binding resin, take OCALIVA at least 4 hours before or 4 hours after taking the bile acid binding resin, or at as great an interval as possible.

    Warfarin

    The International Normalized Ratio (INR) decreased following coadministration of warfarin and OCALIVA. Monitor INR and adjust the dose of warfarin, as needed, to maintain the target INR range when coadministering OCALIVA and warfarin.

    CYP1A2 Substrates with Narrow Therapeutic Index

    Obeticholic acid, the active ingredient in OCALIVA, may increase the exposure to concomitant drugs that are CYP1A2 substrates. Therapeutic monitoring of CYP1A2 substrates with a narrow therapeutic index (e.g. theophylline and tizanidine) is recommended when coadministered with OCALIVA.

    Inhibitors of Bile Salt Efflux Pump

    Avoid concomitant use of inhibitors of the bile salt efflux pump (BSEP) such as cyclosporine. Concomitant medications that inhibit canalicular membrane bile acid transporters such as the BSEP may exacerbate accumulation of conjugated bile salts including taurine conjugate of obeticholic acid in the liver and result in clinical symptoms. If concomitant use is deemed necessary, monitor serum transaminases and bilirubin.

    Please see Full Prescribing Information, including Boxed WARNING and Medication Guide for OCALIVA.

    To report SUSPECTED ADVERSE REACTIONS, contact Intercept Pharmaceuticals, Inc. at 1-844-782-ICPT or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    Cautionary Note Regarding Forward-Looking Statements



    This press release contains forward-looking statements, including, but not limited to, statements regarding the progress, timing and results of our clinical trials, including our clinical trials for the treatment of nonalcoholic steatohepatitis ("NASH"), the safety and efficacy of our approved product, Ocaliva (obeticholic acid or "OCA") for primary biliary cholangitis ("PBC"), and our product candidates, including OCA for liver fibrosis due to NASH, the timing and acceptance of our regulatory filings and the potential approval of OCA for liver fibrosis due to NASH, the review of our New Drug Application for OCA for the treatment of liver fibrosis due to NASH by the U.S. Food and Drug Administration (FDA), our intent to work with the FDA to address the issues raised in the complete response letter (CRL), the potential commercial success of OCA, as well as our strategy, future operations, future financial position, future revenue, projected costs, financial guidance, prospects, plans and objectives.

    These statements constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "possible," "continue" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and we undertake no obligation to update any forward-looking statement except as required by law. These forward-looking statements are based on estimates and assumptions by our management that, although believed to be reasonable, are inherently uncertain and subject to a number of risks. The following represent some, but not necessarily all, of the factors that could cause actual results to differ materially from historical results or those anticipated or predicted by our forward-looking statements: our ability to successfully commercialize Ocaliva for PBC; our ability to maintain our regulatory approval of Ocaliva for PBC in the United States, Europe, Canada, Israel, Australia and other jurisdictions in which we have or may receive marketing authorization; our ability to timely and cost-effectively file for and obtain regulatory approval of our product candidates on an accelerated basis or at all, including OCA for liver fibrosis due to NASH following the issuance of the CRL by the FDA; any advisory committee recommendation or dispute resolution determination that our product candidates, including OCA for liver fibrosis due to NASH, should not be approved or approved only under certain conditions; any future determination that the regulatory applications and subsequent information we submit for our product candidates, including OCA for liver fibrosis due to NASH, do not contain adequate clinical or other data or meet applicable regulatory requirements for approval; conditions that may be imposed by regulatory authorities on our marketing approvals for our products and product candidates, including OCA for liver fibrosis due to NASH, such as the need for clinical outcomes data (and not just results based on achievement of a surrogate endpoint), any risk mitigation programs such as a REMS, and any related restrictions, limitations and/or warnings contained in the label of any of our products or product candidates; any potential side effects associated with Ocaliva for PBC, OCA for liver fibrosis due to NASH or our other product candidates that could delay or prevent approval, require that an approved product be taken off the market, require the inclusion of safety warnings or precautions, or otherwise limit the sale of such product or product candidate; the initiation, timing, cost, conduct, progress and results of our research and development activities, preclinical studies and clinical trials, including any issues, delays or failures in identifying patients, enrolling patients, treating patients, retaining patients, meeting specific endpoints in the jurisdictions in which we intend to seek approval or completing and timely reporting the results of our NASH or PBC clinical trials; our ability to establish and maintain relationships with, and the performance of, third-party manufacturers, contract research organizations and other vendors upon whom we are substantially dependent for, among other things, the manufacture and supply of our products, including Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH, and our clinical trial activities; our ability to identify, develop and successfully commercialize our products and product candidates, including our ability to successfully launch OCA for liver fibrosis due to NASH, if approved; our ability to obtain and maintain intellectual property protection for our products and product candidates, including our ability to cost-effectively file, prosecute, defend and enforce any patent claims or other intellectual property rights; the size and growth of the markets for our products and product candidates and our ability to serve those markets; the degree of market acceptance of Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH or our other product candidates among physicians, patients and healthcare payors; the availability of adequate coverage and reimbursement from governmental and private healthcare payors for our products, including Ocaliva for PBC and, if approved, OCA for liver fibrosis due to NASH, and our ability to obtain adequate pricing for such products; our ability to establish and maintain effective sales, marketing and distribution capabilities, either directly or through collaborations with third parties; competition from existing drugs or new drugs that become available; our ability to prevent system failures, data breaches or violations of data protection laws; costs and outcomes relating to any disputes, governmental inquiries or investigations, regulatory proceedings, legal proceedings or litigation, including any securities, intellectual property, employment, product liability or other litigation; our collaborators' election to pursue research, development and commercialization activities; our ability to establish and maintain relationships with collaborators with development, regulatory and commercialization expertise; our need for and ability to generate or obtain additional financing; our estimates regarding future expenses, revenues and capital requirements and the accuracy thereof; our use of cash and short-term investments; our ability to acquire, license and invest in businesses, technologies, product candidates and products; our ability to attract and retain key personnel to manage our business effectively; our ability to manage the growth of our operations, infrastructure, personnel, systems and controls; our ability to obtain and maintain adequate insurance coverage; the impact of COVID-19, including any impact on our results of operations or financial position, related quarantines and government actions, delays relating to our regulatory applications, disruptions relating to our ongoing clinical trials or involving our contract research organizations, study sites or other clinical partners, disruptions relating to our supply chain or involving our third-party manufacturers, distributors or other distribution partners, facility closures or other restrictions, and the extent and duration thereof; the impact of general U.S. and foreign economic, industry, market, regulatory or political conditions, including the potential impact of Brexit; and the other risks and uncertainties identified in our periodic filings filed with the U.S. Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2019 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020.

    Contact

    For more information about Intercept, please contact:

    Lisa DeFrancesco

    +1-646-565-4833

    interceptpharma.com

            

    Christopher Frates

    +1-646-757-2371

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