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`U.S. SECURITIES AND EXCHANGE COMMISSION
`WASHINGTON, DC 20549
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`FORM 10-K
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`(Mark One)
` ANNUAL REPORT PURSUANT TO SECTION 13 OR 15 (d) OF THE SECURITIES EXCHANGE ACT OF 1934
`For the fiscal year ended December 31, 2012
` TRANSITION REPORT PURSUANT TO SECTION 13 OR 15 (d) OF THE SECURITIES EXCHANGE ACT OF 1934
`For the transition period from to
`Commission file Number: 001-34921
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`AEGERION PHARMACEUTICALS, INC.
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`(Exact Name of Registrant as Specified in Its Charter)
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`20-2960116
`Delaware
`(IRS Employer
`(State or Other Jurisdiction of
`Incorporation or Organization)
`Identification Number)
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`101 Main Street, Suite 1850, Cambridge, Massachusetts 02142
`(Address of Principal Executive Offices, including Zip Code)
`617-500-7867
`(Registrant’s telephone number, including area code)
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`SECURITIES REGISTERED PURSUANT TO SECTION 12(b) OF THE ACT:
`Common Stock, $0.001 Par Value
`The NASDAQ Global Select Market
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`SECURITIES REGISTERED PURSUANT TO SECTION 12(g) OF THE ACT:
`None
`(Title of Class)
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`Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes  No 
`Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or 15(d) of the Act. Yes  No 
`Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange
`Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been
`subject to such filing requirements for the past 90 days. Yes  No 
`Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Website, if any, every Interactive
`Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T during the preceding 12 months (or for such shorter period
`that the registrant was required to submit and post such files). Yes  No 
`Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K is not contained herein, and will not be
`contained, to the best of registrant’s knowledge, in definitive proxy or information statements incorporated by reference in Part III of this Form
`10-K or any amendment to this Form 10-K. 
`Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting
`company. See definitions of “large accelerated filer,” “accelerated filer,” and “smaller reporting company” in Rule 12b-2 of the Exchange Act.
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`Large accelerated filer 
` Accelerated filer
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`Non-accelerated filer  (Do not check if a smaller reporting company)
` Smaller reporting company 
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`Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes  No 
`The aggregate market value of the voting and non-voting common equity held by non-affiliates of the registrant as of June 29, 2012 was
`approximately $357,420,821, based upon the closing price on the NASDAQ Global Market reported for such date.
`As of March 8, 2013, 28,810,589 shares of the registrant’s common stock were outstanding.
`DOCUMENTS INCORPORATED BY REFERENCE
`Portions of the registrant’s definitive Proxy Statement for its 2013 Annual Meeting of Shareholders are incorporated by reference into Part
`III of this Annual Report on Form 10-K
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`FORM 10-K
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`TABLE OF CONTENTS
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`PART I
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` Business
` Risk Factors
` Unresolved Staff Comments
` Properties
` Legal Proceedings
` Mine Safety Disclosures
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`PART II
` Market for Registrant’s Common Equity, Related Shareholder Matters and Issuer Purchases of Equity Securities
` Selected Financial Data
` Management’s Discussion and Analysis of Financial Condition and Results of Operations
` Quantitative and Qualitative Disclosures About Market Risk
` Financial Statements and Supplementary Data
` Changes in and Disagreements With Accountants on Accounting and Financial Disclosure
` Controls and Procedures
` Other Information
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`PART III
` Directors, Executive Officers and Corporate Governance
` Executive Compensation
` Security Ownership of Certain Beneficial Owners and Management and Related Shareholder Matters
` Certain Relationships and Related Transactions, and Director Independence
` Principal Accounting Fees and Services
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`Item 1.
`Item 1A.
`Item 1B.
`Item 2.
`Item 3.
`Item 4.
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`Item 5.
`Item 6.
`Item 7.
`Item 7A.
`Item 8.
`Item 9.
`Item 9A.
`Item 9B.
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`Item 10.
`Item 11.
`Item 12.
`Item 13.
`Item 14.
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`Item 15.
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` Exhibits and Financial Statement Schedules
`SIGNATURES
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`PART IV
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`Forward-Looking Statements
`All statements included or incorporated by reference into this Annual Report on Form 10-K, or Annual Report, other than statements or
`characterizations of historical fact, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.
`Forward-looking statements are often identified by words such as “anticipates,” “expects,” “intends,” “plans,” “predicts,” “believes,” “seeks,”
`“estimates,” “forecasts,” “may,” “will,” “should,” “would,” “could,” “potential,” “continue,” “ongoing” and similar expressions, and variations
`or negatives of these words. Examples of forward-looking statements contained in this Annual Report include our statements regarding: the
`commercial potential for JUXTAPID
`(lomitapide) capsules, also referred to as lomitapide (“JUXTAPID”); our estimates as to the potential
`™
`number of patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia (“HoFH”); the possibility
`of named patient sales outside the United States (“U.S.”); the potential for and possible timing of approval of lomitapide in the European Union
`(“EU”) and other international markets; plans for further clinical development of JUXTAPID; our expectations regarding a possible future filing
`for approval in Japan; our plans for commercial marketing, sales, manufacturing and distribution; our expectations with respect to
`reimbursement of JUXTAPID in the U.S. and elsewhere; our expectations with respect to the impact of competition on our future operations and
`results; our beliefs with respect to our intellectual property portfolio and the extent to which it protects us; our expectations regarding the
`availability of data and marketing exclusivity in various countries; and our forecasts regarding our future expenses, our cash position and the
`timing of any future need for additional capital to fund operations.
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`The forward-looking statements contained in this Annual Report and in the documents incorporated into this Annual Report by reference
`are based on our current beliefs and assumptions with respect to future events, all of which are subject to change. Forward-looking statements
`are not guarantees of future performance, and are subject to risks, uncertainties and assumptions that are difficult to predict, including those
`discussed in “ Risk Factors ” in Part I, Item 1A of this Annual Report. It is not possible for us to predict all risks, nor can we assess the impact of
`all factors on our business or the extent to which any factor, or combination of factors may impact our operations or results. New risks may
`emerge from time to time. Past financial or operating performance is not necessarily a reliable indicator of future performance. Given these risks
`and uncertainties, we can give no assurances that any of the events anticipated by the forward-looking statements will occur or, if any of them
`does, what impact it will have on our results of operations and financial condition. Our actual results could differ materially and adversely from
`those expressed in any forward-looking statement in this Annual Report or in our other filings with the Securities and Exchange Commission
`(“SEC”).
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`Except as required by law, we undertake no obligation to revise our forward-looking statements to reflect events or circumstances that arise
`after the date of this Annual Report or the respective dates of documents incorporated into this Annual Report by reference that include forward-
`looking statements. Thus, you should not assume that our silence over time means that actual events are bearing out as expressed or implied in
`these forward-looking statements.
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`In this Annual Report, “Aegerion Pharmaceuticals, Inc.,” “Aegerion,” the “Company,” “we,” “us” and “our” refer to Aegerion
`Pharmaceuticals, Inc. taken as a whole, unless otherwise noted.
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`Business.
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`Item 1.
`Overview
`We are a biopharmaceutical company dedicated to the development and commercialization of novel, life-altering therapies for patients
`with debilitating, often fatal, rare diseases.
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`PART I
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`Our first product, JUXTAPID received marketing approval from the U.S. Food and Drug Administration (“FDA”) on December 21, 2012,
`as an adjunct to a low-fat diet and other lipid-lowering treatments, including low-density lipoprotein (“LDL”) apheresis, where available, to
`reduce low-density lipoprotein cholesterol (“LDL-C”), total cholesterol (“TC”), apolipoprotein B (“apo B”) and non-high-density lipoprotein
`cholesterol (“non-HDL-C”) in patients with homozygous familial hypercholesterolemia (“HoFH”). We launched JUXTAPID in the U.S. in late
`January 2013. In the first quarter of 2012, we submitted a Marketing Authorization Application (“MAA”) to the European Medicines Agency
`(“EMA”) requesting approval to market lomitapide as an adjunct to a low-fat diet and other lipid-lowering therapies, with or without apheresis,
`to reduce LDL-C, TC, apo B and triglycerides (“TG”) in adults with HoFH.
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`We expect that our near-term efforts will be focused on:
` commercializing JUXTAPID as a treatment for HoFH in the U.S.;
`•
` gaining regulatory approval of lomitapide for adult patients with HoFH in the EU and in other international markets, and launching
`•
`lomitapide in those countries in which we receive marketing approval;
` supporting and facilitating expanded access to JUXTAPID in countries where named patient supply or compassionate use can occur
`as a result of the FDA approval of JUXTAPID;
` clinical development activities to support a potential marketing authorization application for lomitapide in HoFH in Japan; and
` activities in support of our planned clinical study of lomitapide in pediatric HoFH patients.
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`We also expect to build our business in the future by acquiring rights to one or more product candidates targeted at life-threatening or
`substantially debilitating rare diseases that leverage our infrastructure and expertise.
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`As of December 31, 2012, we had not generated revenue from the sale of any product. In the near-term, our ability to generate revenues is
`entirely dependent upon sales of JUXTAPID in the U.S. and in countries where JUXTAPID is available for sale on a named patient basis as a
`result of the approval of JUXTAPID in the U.S. As of December 31, 2012 we had an accumulated deficit of approximately $192.7 million and
`approximately $82.2 million in cash, cash equivalents and marketable securities. In January 2013, we sold 3,110,449 shares of our common
`stock in an underwritten public offering at a price to the public of $26.64 per share. The net proceeds to us from this offering were approximately
`$78.3 million after deducting underwriting discounts and commissions.
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`HoFH
`HoFH is a serious, rare genetic disease that impairs the function of the receptor responsible for removing LDL-C (“bad” cholesterol) from
`the blood. A loss of low density lipoprotein receptor (“LDL-R”) function results in extreme elevation of blood cholesterol levels.
`
`Cholesterol is a naturally occurring molecule which is transported in the blood. The liver and the intestines are the two main sites where
`cholesterol is packaged and released within the body. The liver synthesizes cholesterol, and provides the body’s intrinsic supply. The intestines
`are the conduit through which cholesterol
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`enters the body for metabolism. The delivery of cholesterol to peripheral cells in the body provides necessary sources of cellular energy and cell
`structure. However, excess levels of cholesterol in the blood, also known as hypercholesterolemia, can be the source of significant diseases in
`humans.
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`HoFH is most commonly caused by genetic mutations in both alleles of the LDL-R gene, but can also be caused by mutations in other
`genes. To date, greater than 1,600 mutations have been identified that can impair the functioning of the LDL-R, with some mutations leading to a
`total lack of LDL-R activity and others leading to severely reduced activity in LDL-R. Untreated HoFH patients typically have LDL-C levels in
`the range of 400 mg/dL to 1,000 mg/dL. As a result of elevated levels of LDL-C, HoFH patients often develop premature and progressive
`atherosclerosis, a narrowing or blocking of the arteries, and are at very high risk of experiencing premature cardiovascular events, such as heart
`attack or stroke, often experiencing their first cardiovascular event in their twenties. If untreated, patients with HoFH generally die before the age
`of 30.
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`There are no universally accepted criteria for the diagnosis of HoFH. Diagnosis is typically made clinically, using the following criteria:
` Assessment of cholesterol levels (TC or LDL-C);
`•
` Physical examination for the presence of xanthomas; and
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` Assessment of the family history of the patient.
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`Although not widely used, HoFH may also be diagnosed through an assessment of LDL-R function in cultured skin fibroblasts. Genetic
`testing may be performed to make a diagnosis of HoFH, but current genetic tests only detect approximately 80% of cases. Genetic testing is not
`widely available and is not routinely used if there are sufficient clinical findings and family history to make a clinical diagnosis of HoFH.
`
`Physicians treating patients with hypercholesterolemia, including HoFH, are highly focused on lowering levels of LDL-C in their patients.
`In the U.S., for example, organizations such as the National Cholesterol Education Program (“NCEP”), the American Heart Association, and the
`American College of Cardiology have emphasized aggressive management of LDL-C. NCEP guidelines currently recommend that patients at
`high risk of experiencing a heart attack achieve LDL-C levels of 100 mg/dL or lower through lifestyle changes and drug therapy as appropriate
`based on their starting levels. Both the Canadian Cardiovascular Society and the Joint British Society have supported LDL-C treatment targets as
`low as 70mg/dL for high-risk patients. The clinical approach taken with HoFH patients has typically involved an aggressive treatment plan to
`reduce lipid levels as much as possible through dietary modifications and a combination of available lipid lowering drug therapies. Conventional
`drug therapies include statins, cholesterol absorption inhibitors and bile acid sequestrants. Less frequently, other drugs, such as niacin and
`fibrates, have been added to provide some incremental reductions in LDL-C levels, although these agents are typically used to modify lipids
`other than LDL-C. Because many of these therapies, including statins, act by increasing the activity of LDL-R, HoFH patients, given their
`defective LDL-R function, are often resistant, or refractory, to standard therapies. For example, high dose statin therapies that typically produce
`46% to 55% reductions in LDL-C levels in the broad hypercholesterolemic patient population, on average, produce 14% to 30% reductions in
`patients with HoFH. Patients with HoFH who are unable to reach their recommended target LDL-C levels on drug therapy are sometimes treated
`using LDL apheresis in which cholesterol is removed from the body through mechanical filtration. Although levels of LDL-C are reduced
`acutely using apheresis, there is a rapid rebound. Because apheresis provides only temporary reductions in LDL-C levels, it must be repeated
`frequently, typically one or two times per month. In addition, apheresis is not readily available to all patients, particularly in the U.S. due to the
`limited number of treatment centers that perform this procedure.
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`JUXTAPID
`Mechanism of Action
`JUXTAPID is a small molecule microsomal triglyceride transfer protein, or MTP, inhibitor, or MTP-I. MTP exists in both the liver and
`intestines where it plays a role in the formation of cholesterol. Given the fact that MTP is involved in the formation of cholesterol-carrying
`lipoproteins from both liver-related, or hepatic, and intestinal sources, we believe the inhibition of MTP makes an attractive target for
`cholesterol-lowering therapy. JUXTAPID is the only MTP-I approved by the FDA for any indication.
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`United States
`The FDA approved JUXTAPID on December 21, 2012, as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL
`apheresis where available, to reduce LDL-C, TC, apo B and non-HDL-C in patients with HoFH. We launched JUXTAPID in the U.S. in late
`January 2013. The FDA has granted seven years of orphan drug exclusivity for JUXTAPID in the U.S. in the treatment of HoFH.
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`The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH or in
`pediatric patients. The effect of JUXTAPID on cardiovascular morbidity and mortality has not been determined. The prescribing information for
`JUXTAPID contains a boxed warning citing the risk of hepatic toxicity. The boxed warning warns physicians that JUXTAPID can cause
`hepatotoxicity as manifested by elevations in transaminases and increases in hepatic fat, and recommends that physicians measure alanine
`aminotranferease (“ALT”), aspartate aminotransferase (“AST”), alkaline phosphatase, and total bilirubin before initiating treatment and then
`ALT and AST regularly during treatment.
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`Because of the risk of liver toxicity, JUXTAPID is available only through the approved Risk Evaluation and Mitigation Strategies
`(“REMS”) program. We will certify all qualified healthcare providers who prescribe JUXTAPID and the pharmacies that will dispense the
`medicine. The goals of the REMS program are: to educate prescribers about the risk of hepatotoxicity associated with the use of JUXTAPID and
`the need to monitor patients during treatment with JUXTAPID as per product labeling; and to restrict access to therapy with JUXTAPID to
`patients with a clinical or laboratory diagnosis consistent with HoFH.
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`European Union
`In the first quarter of 2012, we submitted an MAA to the EMA, requesting approval to market lomitapide in the EU as an adjunct to a low-
`fat diet and other lipid-lowering therapies, with or without apheresis, to reduce LDL-C, TC, apo B and TG in adults with HoFH. In March 2012,
`the EMA accepted the MAA for review with a review start date of March 21, 2012. We expect a decision on our MAA filing in mid-2013.
`Lomitapide does not have orphan drug designation for the treatment of HoFH in the EU since the EMA considers the relevant condition for
`orphan drug purposes to include both HoFH and heterozygous familial hypercholesterolemia (“HeFH”).
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`Expanded Access
`We plan to file for marketing approval of lomitapide in a number of additional countries outside the U.S. and EU. We also plan to make
`lomitapide available in certain countries that allow use of a drug, on a named patient basis or under a compassionate use or other type of so-
`called expanded access program, before it has obtained marketing approval in such countries. We plan to seek reimbursement for lomitapide for
`authorized pre-approval uses in some of these countries, to the extent permitted by applicable law and local regulatory authorities. In other
`countries or under certain circumstances, we may provide lomitapide free of charge for permitted pre-approval uses.
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`Other Development
`During the fourth quarter of 2012, we initiated enrollment of patients into a Phase 1 study of the pharmacokinetic and pharmacodynamic
`properties of JUXTAPID in Japanese patients. Depending on the outcome of this study, we plan to conduct a therapeutic bridging study of
`JUXTAPID in Japanese HoFH patients in support of a planned filing for marketing authorization in Japan.
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`We also plan to conduct a clinical study of lomitapide in pediatric HoFH patients. The FDA has established a post-marketing requirement
`for us to conduct a juvenile toxicology study in rodents. The study will seek to ascertain the impact, if any, of JUXTAPID on growth and
`development prior to initiating the clinical study of lomitapide in pediatric patients. In 2011, the Paediatric Committee of the EMA (“PDCO”)
`issued a positive opinion on our Pediatric Investigation Plan (“PIP”) for lomitapide. This enabled us to file the MAA for lomitapide without
`pediatric data. The PDCO opinion requires that, prior to initiation of a pediatric study in the EU, the data on lomitapide generated in the adult
`HoFH population must be evaluated by the Committee for Medicinal Products for Human Use (“CHMP”) and a positive conclusion on the
`benefit/risk balance and therapeutic benefit must be found. The pediatric study will then be reevaluated by the PDCO. In conjunction with our
`planning for a potential clinical study of lomitapide in pediatric patients, we are evaluating our formulation for lomitapide to determine whether
`any changes are necessary to facilitate administration to pediatric patients.
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`If, in the future, we elect to develop lomitapide for broader patient populations, such as for those patients with HeFH, who have severely
`elevated LDL-C levels despite current therapies, we would plan to do so selectively depending on, among other factors, the applicable
`indications, the related development costs and our available resources.
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`Patient Registry
`We have made a post-marketing commitment to the FDA to conduct an observational cohort study, or registry study, in order to further
`understand JUXTAPID’s long-term safety and effectiveness. The target enrollment for this study is 300 patients and patients will be enrolled
`globally, and followed by investigators in the study for a period of 10 years. The registry is voluntary, but patients who are treated with
`JUXTAPID will be encouraged to participate in the study.
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`Phase 3 Clinical Study (HoFH)
`The FDA based its approval of JUXTAPID on our Phase 3 clinical study, which evaluated the safety and effectiveness of JUXTAPID to
`reduce LDL-C levels in 29 adult patients with HoFH. The study was a multinational, single-arm, open-label, 78-week trial. The results of the
`study were published in the November 2, 2012 online version of the Lancet .
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`In the Phase 3 study, each patient’s background lipid-lowering therapies were stabilized during a six-week run-in phase prior to dosing,
`and were maintained through at least the end of the 26-week efficacy phase. All patients received dietary counseling and were instructed to
`consume a diet containing <20% of energy from total dietary fat. JUXTAPID was initiated at a dose of 5 mg daily and gradually escalated to
`doses of 10 mg, 20 mg, 40 mg, up to 60 mg daily, based on tolerability and acceptable liver enzymes levels. As set forth in the table below, when
`added to the existing lipid-lowering therapy of the HoFH patients in the study, JUXTAPID reduced LDL-C by an average of 40% at Week 26 in
`the intent-to-treat population with last observation carried forward for the patients who discontinued prematurely; and reduced LDL-C by an
`average of 50% for the 23 patients who completed the study through Week 26.
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`As shown in the table below, approximately 65% of all patients completing the study experienced LDL-C reductions of 50% to 93% from
`their baseline as measured at the end of Week 26.
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`After Week 26, during the 52-week safety phase of the study, adjustments to concomitant lipid-lowering treatments were allowed. Average
`reductions in LDL-C were sustained during chronic therapy.
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`The most common adverse reactions in the Phase 3 study were gastrointestinal, reported by 27 (93%) of 29 patients. Adverse reactions,
`reported by greater than or equal to 8 patients (28%) in the HoFH clinical trial, included diarrhea, nausea, vomiting, dyspepsia and abdominal
`pain. Other common adverse reactions, reported by five to seven (17-24%) patients, included weight loss, abdominal discomfort, abdominal
`distension, constipation, flatulence, increased ALT chest pain, influenza, nasopharyngitis, and fatigue. Elevations in liver enzymes and hepatic
`(liver) fat were also observed. Ten of the 29 patients in the study had at least one elevation in liver enzymes greater than or equal to three times
`the upper limit of normal (“ULN”), including four patients who experienced liver enzymes greater than or equal to five times the ULN. During
`the clinical trial, liver enzyme elevations were managed through dose reduction or temporary discontinuation of dose. There were no clinically
`meaningful elevations of total bilirubin, international normalized ratio (“INR”) or alkaline phosphatase, which are other markers of potential
`harmful effects on the liver. Hepatic fat increased from a baseline of 1% to a median absolute increase of 6% at 26 and 78 weeks.
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`Historical Clinical Development
`Lomitapide has also been evaluated in 14 Phase 1 and eight Phase 2 clinical trials, including a Phase 2 trial in HoFH patients.
`Approximately 940 patients have been treated with lomitapide as part of the clinical development program.
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`Estimated Prevalence of HoFH
`There is no patient registry or other method of establishing with precision the actual number of patients with HoFH in any geography.
`Medical literature has historically reported the prevalence rate of genotypic HoFH as one person in a million. However, we believe that the
`prevalence rate of HoFH is higher. The historically reported definition of HoFH used a narrow genotypic definition of HoFH. At the time the
`rate was first reported, many of the genetic mutations leading to defects in LDL-R function were not characterized, and some mutations remain
`uncharacterized even today. In addition, many physicians use a broader definition of HoFH that includes patients diagnosed through phenotypic
`criteria. In 2010, we commissioned an independent consultant in the healthcare industry to prepare a commercial assessment of the HoFH market
`for us. In its report, this consultant estimated that the total number of patients likely to seek treatment with symptoms, signs or laboratory
`findings consistent with HoFH in each of the U.S. and the EU is approximately 3,000 patients. This consultant’s estimates, however, included a
`segment of severe HeFH patients whose levels of LDL-C are not controlled by current therapies. These patients may be phenotypically indistinct
`from HoFH patients. JUXTAPID is indicated solely for HoFH. Our prescribing information in the U.S. specifies that the safety and effectiveness
`of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH. We are not permitted to promote
`JUXTAPID for any indication other than HoFH. In addition, as part of the prescriber authorization form under our REMS program in the U.S.,
`the prescriber must affirm that the patient has a clinical or laboratory diagnosis consistent with HoFH. We do not know how many patients will
`be determined to have a clinical or laboratory diagnosis consistent with HoFH, and there is no generally accepted and referenced definition of
`HoFH matching these criteria. However, rare diseases are often found to have a higher than expected prevalence rate once the first product
`available to treat the disease is introduced. We expect this may also be true for HoFH. As a result, we believe that, even if we exclude the
`patients who have a clinical phenotype consistent with HoFH, but as to whom the prescriber cannot conclude and affirm that the patient has a
`clinical or laboratory diagnosis consistent with HoFH, there still may be as many as 3,000 HoFH patients in the U.S. based on our belief that the
`base prevalence rate may be higher than our consultant estimated. There is no guarantee that our assumptions and beliefs are correct. The
`number of patients with HoFH in the U.S. could actually be significantly lower than we expect, and could be closer to the historically reported
`rates than to our estimate of 3,000 patients. Ultimately the actual size of the total addressable market in the U.S. will be determined only after we
`have substantial commercial history selling JUXTAPID and we are able to assess how it is being used
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`clinically. We believe that the prevalence rate in the EU is likely to be consistent with the prevalence rate in the U.S. However, the total
`addressable HoFH market in the EU will depend ultimately on whether the EMA requires a genetic diagnosis or imposes other narrow diagnosis
`criteria.
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`Commercialization
`We believe that the key priorities for a successful commercial launch of JUXTAPID in the U.S. include:
` identifying patients with HoFH;
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` educating and training healthcare providers about JUXTAPID;
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` supporting access to and obtaining coverage for JUXTAPID; and
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` providing patients with support services to help them maximize the benefits of and compliance with treatment while minimizing the
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`risks and possible side effects.
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`Our commercial initiatives are designed to support these priorities. We believe that it will be possible to commercialize JUXTAPID in the
`U.S. with a relatively small specialty sales force. As a result, we have 25 sales representatives who are experienced in marketing drugs for the
`treatment of rare disorders. Our sales representatives are responsible for identifying patients with HoFH, and educating and training healthcare
`providers about JUXTAPID. We have also hired a small team of national account managers, who are primarily responsible for working with
`insurance plans, health maintenance organizations and other payers on securing reimbursement and formulary status for JUXTAPID. We have
`developed a comprehensive patient support program, which includes educational resources about JUXTAPID and HoFH; insurance verification
`and reimbursement support; nutritional support and counseling; monitoring and support of adherence; providing patients with options for
`seeking financial assistance from independent organizations for co-payments and other out-of-pocket payments for JUXTAPID treatment; and a
`free drug program for certain eligible uninsured and underinsured patients. We have a small team of customer care managers who are
`responsible for working with patients who are prescribed JUXTAPID and for coordinating our patient support services.
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`We believe our pricing for JUXTAPID in the U.S. is consistent with the level of pricing for other ultra-orphan drugs that treat diseases
`with a comparable prevalence rate as HoFH. Based on our interactions with payers and market research, we believe that payers in the U.S. will
`generally provide coverage for JUXTAPID. We believe that most payers will not make coverage dec