Intercept Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results, Issues 2021 Financial Guidance and Provides Business Update
Worldwide Ocaliva® net sales of
Intercept provides initial 2021 Worldwide Ocaliva Net Sales Guidance of
FDA interactions remain ongoing regarding newly identified safety signal (NISS) for liver disorder in a subset of PBC patients with cirrhosis; Update to Ocaliva label expected
Anticipated readout of REVERSE Phase 3 study in compensated cirrhosis due to NASH by end of 2021
Conference call scheduled for 8:30 a.m. ET today
“As we turn the page on 2020, I am proud of the Intercept team’s efforts to support patients with PBC and grow our Ocaliva foundational rare disease franchise during a global pandemic,” said
“Regarding our NASH regulatory process, we are laser-focused on building alignment with FDA in advance of a potential NDA resubmission, with the goal of increasing the probability of a successful outcome for patients with advanced fibrosis due to NASH,” Durso continued. “We are also looking forward to topline results from our phase 3 REVERSE trial in patients with compensated cirrhosis due to NASH this year.”
Ocaliva® (obeticholic acid) Commercial Highlights
Full year 2020 Ocaliva net sales were $312.7 million, which represented growth of 25% as compared to the prior year. Ocaliva net sales in 2020 were comprised of
We recognized $83.3 million of Ocaliva net sales in the fourth quarter of 2020, as compared to
Selected Fourth Quarter and Full Year 2020 Financial Results
We recognized $83.3 million in total revenue in the fourth quarter of 2020, as compared to $71.5 million in total revenue in the prior year quarter. Total revenue in the fourth quarter of 2019 included approximately $1.2 million of licensing revenue.
We recognized $312.7 million in total revenue in 2020, as compared to $252.0 million in 2019. Total revenue in 2019 included $249.6 million of Ocaliva net sales and approximately $2.4 million of licensing revenue.
Our cost of sales was $0.8 million in the fourth quarter of 2020, as compared to $2.5 million in the prior year quarter. Cost of sales was $5.3 million in 2020, as compared to $4.2 million in 2019. Our cost of sales for the quarters and years ended December 31, 2020 and 2019 consisted primarily of packaging, labeling, materials and related expenses.
Our selling, general and administrative expenses decreased to $70.0 million in the fourth quarter of 2020, from $93.7 million in the prior year quarter. The fourth quarter period-over-period decrease was primarily driven by reductions in spend resulting from actions taken following the delay of the potential approval and commercialization of obeticholic acid (OCA) for liver fibrosis due to nonalcoholic steatohepatitis (NASH). Selling, general and administrative expenses increased to
Our research and development expenses decreased to $51.9 million in the fourth quarter of 2020, from $64.6 million in the prior year quarter. The fourth quarter period-over-period decrease was primarily driven by lower NASH development costs and the conclusion of enrollment activities for the REGENERATE and REVERSE studies. Research and development expenses decreased to
Restructuring expenses were
In the quarters ended December 31, 2020 and 2019, we recorded $123.9 million and $160.8 million, respectively, in total operating expenses and $106.6 million and $146.8 million, respectively, in non-GAAP adjusted operating expenses, which excludes non-cash stock-based compensation expense of $16.5 million and $13.2 million, respectively, and depreciation expense of $0.8 million and $0.8 million, respectively.
In the years ended December 31, 2020 and 2019, we recorded $543.9 million and $564.4 million, respectively, in total operating expenses and $480.0 million and $504.8 million, respectively, in non-GAAP adjusted operating expenses, which excludes non-cash stock-based compensation expense of $60.8 million and $56.0 million, respectively, and depreciation expense of $3.1 million and $3.7 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
Interest expense in the quarters ended December 31, 2020 and 2019 was $12.3 million and $11.6 million, respectively. Interest expense in the years ended
In the fourth quarter and full year of 2020 we reported a net loss of $52.1 million and
2021 Financial Guidance
Our sales guidance range for full year 2021 provided below reflects the potential impact of various Ocaliva label change scenarios, and we plan to refine this range throughout the year as we have more information from our discussions with FDA.
We are announcing 2021 Ocaliva net sales guidance of
Conference Call on
We are hosting our fourth quarter and full year 2020 financial results conference call and webcast on
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
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
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
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.
WARNING: HEPATIC DECOMPENSATION AND FAILURE IN INCORRECTLY DOSED PBC PATIENTS WITH CHILD-PUGH CLASS B OR
- 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 Bor 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 Bor C hepatic impairment or a prior decompensation event.
OCALIVA is contraindicated in PBC patients with complete biliary obstruction.
Warnings and Precautions
Hepatic Decompensation and Failure in Incorrectly-Dosed PBC Patients with Child-
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 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.
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.
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.
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
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
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, including in connection with the newly identified safety signal relating to Ocaliva identified by the FDA in
For more information about Intercept, please contact:
Condensed Consolidated Statements of Operations
(In thousands, except per share data)
|Three Months Ended
|Product revenue, net||$||83,268||$||70,284||$||312,690||$||249,570|
|Cost of sales||767||2,474||5,322||4,212|
|Selling, general and administrative||69,956||93,680||332,493||317,418|
|Research and development||51,898||64,636||191,485||242,799|
|Total operating expenses||123,870||160,790||543,930||564,429|
|Other income (expense):|
|Other income, net||708||2,758||4,414||8,890|
|Net loss per common and potential common share:|
|Basic and diluted||$||(1.58||)||$||(2.99||)||$||(8.34||)||$||(10.89||)|
|Weighted average common and potential common shares outstanding:|
|Basic and diluted||33,007||32,780||32,970||31,654|
Condensed Consolidated Balance Sheet Information
|Cash, cash equivalents, restricted cash and investment debt securities, available for sale||
|Total liabilities (1)||$||747,342||$||703,330|
|Stockholders’ (deficit) equity||$||(166,853||)||$||51,556|
Reconciliation of Non-GAAP Adjusted Operating Expenses to Total Operating Expenses
|Three Months Ended
|Total operating expenses||$||123,870||$||160,790||$||543,930||$||564,429|
|Non-GAAP adjusted operating expenses||$||106,565||$||146,793||$||479,962||$||504,784|
Source: Intercept Pharmaceuticals, Inc.