Esperion Therapeutics, Inc. - Quarter Report: 2018 September (Form 10-Q)

Table of Contents

 

 

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

Form 10-Q

 

x      QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

 

For the quarterly period ended September 30, 2018

 

OR

 

o         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: 001-35986

 

Esperion Therapeutics, Inc.

(Exact name of registrant as specified in its charter)

 

 

3891 Ranchero Drive, Suite 150

Ann Arbor, MI 48108

(Address of principal executive office) (Zip Code)

 

Registrant’s telephone number, including area code:

(734) 887-3903

 

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 x  No o

 

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).  Yes x  No o

 

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

 

 

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o

 

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act).  Yes o  No x

 

As of October 31, 2018, there were 26,811,002 shares of the registrant’s Common Stock, $0.001 par value per share, outstanding.

 

 

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Esperion Therapeutics, Inc.

 

INDEX

 

	Page
PART I — FINANCIAL INFORMATION
Item 1. Financial Statements
Condensed Balance Sheets at September 30, 2018 and December 31, 2017	3
Condensed Statements of Operations and Comprehensive Loss for the three and nine month periods ended September 30, 2018 and 2017	4
Condensed Statements of Cash Flows for the nine month periods ended September 30, 2018 and 2017	5
Notes to Condensed Financial Statements	6
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations	13
Item 3. Quantitative and Qualitative Disclosures About Market Risk	29
Item 4. Controls and Procedures	30
PART II — OTHER INFORMATION
Item 1. Legal Proceedings	31
Item 1A. Risk Factors	31
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds	32
Item 6. Exhibits	32
Signatures	34

 

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Esperion Therapeutics, Inc.

 

Condensed Balance Sheets

(in thousands, except share data)

 

		September 30, 2018			December 31, 2017
		(unaudited)
Assets
Current assets:
Cash and cash equivalents		$	26,755		$	34,468
Short-term investments		132,463			165,731
Prepaid clinical development costs		7,411			2,072
Other prepaid and current assets		1,581			1,653
Total current assets		168,210			203,924
Property and equipment, net		583			435
Intangible assets		56			56
Long-term investments		5,181			73,420
Total assets		$	174,030		$	277,835
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable		$	18,630		$	20,375
Current portion of long-term debt		—			1,045
Accrued clinical development costs		20,030			10,506
Other accrued liabilities		3,625			1,218
Total current liabilities		42,285			33,144
Total liabilities		42,285			33,144
Commitments and contingencies (Note 5)
Stockholders’ equity:
Preferred stock, $0.001 par value; 5,000,000 shares authorized and no shares issued or outstanding as of September 30, 2018 and December 31, 2017		—			—
Common stock, $0.001 par value; 120,000,000 shares authorized as of September 30, 2018 and December 31, 2017; 26,804,300 shares issued and outstanding at September 30, 2018 and 26,304,669 shares issued and outstanding at December 31, 2017		27			26
Additional paid-in capital		670,340			641,801
Accumulated other comprehensive loss		(560		)	(845		)
Accumulated deficit		(538,062		)	(396,291		)
Total stockholders’ equity		131,745			244,691
Total liabilities and stockholders’ equity		$	174,030		$	277,835

 

See accompanying notes to the condensed financial statements.

 

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Esperion Therapeutics, Inc.

 

Condensed Statements of Operations and Comprehensive Loss

(in thousands, except share and per share data)

(unaudited)

 

		Three Months Ended September 30,						Nine Months Ended September 30,
		2018			2017			2018			2017
Operating expenses:
Research and development		$	41,551		$	40,056		$	122,015		$	114,164
General and administrative		9,011			5,681			21,921			16,122
Total operating expenses		50,562			45,737			143,936			130,286
Loss from operations		(50,562		)	(45,737		)	(143,936		)	(130,286		)
Other income, net		651			518			2,165			1,189
Net loss		$	(49,911	)	$	(45,219	)	$	(141,771	)	$	(129,097	)
Net loss per common share (basic and diluted)		$	(1.86	)	$	(1.86	)	$	(5.30	)	$	(5.57	)
Weighted-average shares outstanding (basic and diluted)		26,804,026			24,311,844			26,732,733			23,161,847
Other comprehensive loss:
Unrealized gain (loss) on investments		$	216		$	(112	)	$	285		$	(179	)
Total comprehensive loss		$	(49,695	)	$	(45,331	)	$	(141,486	)	$	(129,276	)

 

See accompanying notes to the condensed financial statements.

 

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Esperion Therapeutics, Inc.

 

Condensed Statements of Cash Flows

(in thousands)

(unaudited)

 

		Nine Months Ended September 30,
		2018			2017
Operating activities
Net loss		$	(141,771	)	$	(129,097	)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation expense		191			196
Amortization of premiums and discounts on investments		(134		)	314
Stock-based compensation expense		17,139			14,071
Changes in assets and liabilities:
Prepaids and other assets		(5,267		)	(1,458		)
Accounts payable		(1,808		)	14,906
Other accrued liabilities		11,704			4,640
Net cash used in operating activities		(119,946		)	(96,428		)
Investing activities
Purchases of investments		(25,481		)	(212,973		)
Proceeds from sales/maturities of investments		127,408			128,740
Purchase of property and equipment		(46		)	(19		)
Net cash provided by (used in) investing activities		101,881			(84,252		)
Financing activities
Proceeds from issuance of common stock, net of issuance costs		—			164,079
Proceeds from exercise of common stock options		11,401			732
Payments on long-term debt		(1,049		)	(1,272		)
Net cash provided by financing activities		10,352			163,539
Net decrease in cash and cash equivalents		(7,713		)	(17,141		)
Cash and cash equivalents at beginning of period		34,468			38,165
Cash and cash equivalents at end of period		$	26,755		$	21,024
Supplemental disclosure of cash flow information:
Purchase of property and equipment not yet paid		$	293		$	—
Offering costs not yet paid		$	—		$	111

 

See accompanying notes to the condensed financial statements.

 

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Esperion Therapeutics, Inc.

Notes to the Condensed Financial Statements

(unaudited)

 

1. The Company and Basis of Presentation

 

The Company is the Lipid Management Company, a late-stage pharmaceutical company focused on developing and commercializing complementary, convenient, cost-effective, once-daily, oral therapies for the treatment of patients with elevated low density lipoprotein cholesterol (“LDL-C”). Through scientific and clinical excellence, and a deep understanding of cholesterol biology, the experienced lipid management team at Esperion is committed to developing new LDL-C lowering therapies that will make a substantial impact on reducing global cardiovascular disease (“CVD”); the leading cause of death around the world. Bempedoic acid and the Company’s lead product candidate, the bempedoic acid / ezetimibe combination pill, are targeted therapies that have been shown to significantly lower elevated LDL-C levels in patients with hypercholesterolemia, including patients inadequately treated with current lipid-modifying therapies.

 

The clinical development program for the bempedoic acid / ezetimibe combination pill consisted of a single pivotal Phase 3 clinical study (1002FDC-053) in patients with hypercholesterolemia and with atherosclerotic cardiovascular disease (“ASCVD”) and/or heterozygous familial hypercholesterolemia (“HeFH”), including high CVD risk primary prevention patients, whose LDL-C is not adequately controlled despite receiving maximally tolerated lipid-modifying background therapy. 1002FDC-053 initiated in November 2017, fully enrolled 382 patients in March 2018, and the Company reported top-line results in August 2018.

 

The global pivotal Phase 3 clinical development program for bempedoic acid, consisting of four clinical studies, is complete with 3,621 high CVD risk patients with hypercholesterolemia and ASCVD and/or HeFH, or who are high CVD risk primary prevention, on optimized background lipid-modifying therapy and with elevated levels of LDL-C. These patients are on two distinct types of background lipid-modifying therapy: 1) patients on their maximally tolerated statin therapy, and 2) patients who are only able to tolerate less than the lowest approved daily starting dose of a statin, and can be considered stain intolerant. In March 2018, the Company reported top-line results from the first of the Phase 3 studies, Study 4 (1002-048). In May 2018, the Company reported top-line results from the 52-week long-term safety study, Study 1 (1002-040), and from Study 3 (1002-046). In October 2018, the Company reported top-line results from Study 2 (1002-047).

 

The Company intends to use positive results from the Phase 3 bempedoic acid / ezetimibe combination pill and bempedoic acid programs with a total of 4,003 patients to support global regulatory submissions for tandem LDL-C lowering indications in the U.S. no later than the first quarter of 2019 and in Europe no later than the second quarter of 2019.

 

The Company is also conducting a global cardiovascular outcomes trial (“CVOT”)—known as Cholesterol Lowering via BEmpedoic Acid, an ACL-inhibiting Regimen (CLEAR) Outcomes, for bempedoic acid in 12,604 patients with hypercholesterolemia and high CVD risk and who can be considered statin intolerant. The Company initiated the CLEAR Outcomes CVOT in December 2016 and expects the study to be fully enrolled in 2019, and intends to use positive results from this CVOT to support submissions for a CV risk reduction indication in the U.S. and Europe by 2022.

 

The Company’s primary activities since incorporation have been conducting research and development activities, including nonclinical, preclinical and clinical testing, performing business and financial planning, recruiting personnel, and raising capital. Accordingly, the Company has not commenced principal operations and is subject to risks and uncertainties which include the need to research, develop, and clinically test potential therapeutic products; obtain regulatory approvals for its products and commercialize them, if approved; expand its management and scientific staff; and finance its operations with an ultimate goal of achieving profitable operations.

 

The Company has sustained operating losses since inception and expects such losses to continue over the foreseeable future. Management plans to continue to fund operations through public or private equity or debt financings or through other sources, which may include collaborations with third parties. If adequate funds are not available, the Company may not be able to continue the development of its current or future product candidates, or to commercialize its current or future product candidates, if approved.

 

Basis of Presentation

 

The accompanying condensed financial statements are unaudited and were prepared by the Company in accordance with generally accepted accounting principles in the United States of America (“GAAP”). In the opinion of management, the Company has made all adjustments, which include only normal recurring adjustments necessary for a fair statement of the

 

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Company’s financial position and results of operations for the interim periods presented. Certain prior year amounts have been reclassified to conform with current year presentation. Certain information and disclosures normally included in the annual financial statements prepared in accordance with GAAP have been condensed or omitted. These condensed interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2017, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2017. The results of operations for the interim periods are not necessarily indicative of the results to be expected for a full year, any other interim periods or any future year or period.

 

2. Summary of Significant Accounting Policies

 

In January 2016, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) 2016-01 which includes provisions to accounting for equity investments, financial liabilities under the fair value option, and presentation and disclosure requirements for financial instruments. The updated guidance requires equity investments with determinable fair values to be measured at fair value with changes in fair value recognized in income. Equity investments without determinable fair values are to be measured at cost, less any impairment determined to be other than temporary. The Company adopted ASU 2016-01 effective January 1, 2018. Prospectively, unrealized gains or losses from equity investments with readily determinable fair values will be reflected in earnings through “Other income, net” on the statement of operations and any equity investments owned by the Company without readily determinable fair values will be measured at cost, less any impairment determined to be other than temporary. The adoption of the ASU did not have a material impact to the Company’s balance sheets, statements of operations or statements of cash flows.

 

In May 2017, the FASB issued ASU 2017-09 which includes provisions to clarify when to account for a change to terms or conditions of a share-based payment award as a modification. Under the updated guidance, modification accounting is required only if the fair value, the vesting conditions, or the classification of the award changes as a result of the change in terms or conditions. The Company adopted ASU 2017-09 effective January 1, 2018. The adoption of the ASU did not have a material impact to the Company’s balance sheets, statements of operations or statements of cash flows.

 

In August 2018, the FASB issued ASU 2018-15 which includes provisions to clarify customer’s accounting for implementation costs incurred in a cloud computing arrangement. Under the updated guidance, a customer in a cloud computing arrangement that is a service contract should follow the internal-use software guidance to determine how to account for costs incurred in implementation. The updated guidance also requires certain classification on the balance sheets, statements of operations and statements of cash flows as well as additional quantitative and qualitative disclosures. The standard is effective for public companies for fiscal years beginning after December 15, 2019, and interim periods within those years. Early adoption is permitted and entities can choose to adopt the new guidance prospectively or retrospectively. The Company does not believe the adoption of this standard to have a material impact to the Company’s balance sheets, statements of operations or statements of cash flows.

 

There have been no other material changes to the significant accounting policies previously disclosed in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2017.

 

3. Debt

 

In June 2014, the Company entered into a loan and security agreement (the “Credit Facility”) with Oxford Finance LLC which provided for initial borrowings of $5.0 million under the term loan (the “Term A Loan”). On June 30, 2014, the Company received proceeds of $5.0 million from the issuance of secured promissory notes under the Term A Loan. The secured promissory notes issued under the Credit Facility bear interest at an annual rate of 6.40% and were due on July 1, 2018. A final payment equal to 8.0% of the Term A Loan was due upon the earlier of the maturity date or prepayment of the term loan. The Company recognized the final payment as interest expense using the effective interest method over the life of the Credit Facility. The Term A Loan was fully repaid in July 2018.

 

4. Warrants

 

In connection with the Credit Facility entered into in June 2014, the Company issued a warrant to purchase 8,230 shares of common stock at an exercise price of $15.19. The warrant will terminate on the earlier of June 30, 2019, and the closing of a merger or consolidation transaction in which the Company is not the surviving entity. The warrant was recorded at fair value of $0.1 million to additional-paid-in-capital in accordance with ASC 815-10 based upon the allocation of the debt proceeds.

 

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Upon the closing of the Company’s Initial Public Offering, all warrants exercisable for 1,940,000 shares of Series A preferred stock, at an exercise price of $1.00 per share (unadjusted for stock splits), were automatically converted into warrants exercisable for 277,690 shares of common stock, at an exercise price of $6.99 per share. During the nine months ended September 30, 2018, the remaining 177,123 warrants were net exercised for 159,944 shares of the Company’s common stock. During the year ended December 31, 2017, 71,237 warrants were net exercised for 62,525 shares of the Company’s common stock.

 

As of September 30, 2018, the Company had warrants outstanding that were exercisable for a total of 8,230 shares of common stock at a weighted-average exercise price of $15.19 per share.

 

5. Commitments and Contingencies

 

On January 12, 2016, a purported stockholder of the Company filed a putative class action lawsuit in the United States District Court for the Eastern District of Michigan, against the Company and Tim Mayleben, captioned Kevin L. Dougherty v. Esperion Therapeutics, Inc., et al. (No. 16-cv-10089). The lawsuit alleges that the Company and Mr. Mayleben violated Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule 10b-5 by allegedly failing to disclose in an August 17, 2015, public statement that the FDA would require a cardiovascular outcomes trial before approving the Company’s lead product candidate. The lawsuit seeks, among other things, compensatory damages in connection with an allegedly inflated stock price between August 18, 2015 and September 28, 2015, as well as attorneys’ fees and costs. On May 20, 2016, an amended complaint was filed in the lawsuit and on July 5, 2016, the Company filed a motion to dismiss the amended complaint. On December 27, 2016, the court granted the Company’s motion to dismiss with prejudice and entered judgment in the Company’s favor. On January 24, 2017, the plaintiffs in this lawsuit filed a motion to alter or amend the judgment.  In May 2017, the court denied the plaintiff’s motion to alter or amend the judgment.  On June 19, 2017, the plaintiffs filed a notice of appeal to the Sixth Circuit Court of Appeals and on September 14, 2017, they filed their opening brief in support of the appeal. The appeal was fully briefed on December 7, 2017, and it was argued before the Sixth Circuit on March 15, 2018. On September 27, 2018, the Sixth Circuit issued an opinion in which it reversed the district court’s dismissal and remanded for further proceedings. On October 11, 2018, the Company filed a petition for rehearing en banc and, on October 23, 2018, the Sixth Circuit Court of Appeals directed plaintiffs to respond to that petition. The Company is unable to predict the outcome of this matter and is unable to make a meaningful estimate of the amount or range of loss, if any, that could result from an unfavorable outcome.

 

On May 7, 2018, a purported stockholder of the Company filed a putative class action lawsuit in the United States District Court for the Eastern District of Michigan, captioned Kevin Bailey v. Esperion Therapeutics, Inc., et al. (No. 18-cv-11438).  An amended complaint was filed on October 22, 2018, against the Company and certain directors and officers. The amended complaint alleges violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule 10b-5 based on allegedly making false and misleading statements about the facts and circumstances surrounding the Phase 3 trial results for bempedoic acid that the Company announced on May 2, 2018. The lawsuit seeks, among other things, compensatory damages in connection with an allegedly inflated stock price between February 22, 2017, and May 22, 2018, as well as attorneys’ fees and costs. The Company is unable to predict the outcome of this matter and is unable to make a meaningful estimate of the amount or range of loss, if any, that could result from an unfavorable outcome.

 

On July 6, 2018, the Company entered into the first amendment of the lease for the Company’s principal executive office in Ann Arbor, Michigan. The amended lease is to increase the current 7,941 rentable square feet of office space by 11,471 rentable square feet, together with the right to use common areas and facilities in common with the landlord and other tenants. The term of the lease commences with respect to all of the space in the leased premises on the later to occur of (i) the date upon which landlord delivers the premises to the Company under the terms of the lease with the delivery conditions set forth in the lease satisfied and (ii) November 1, 2018 (the “Lease Commencement Date”). The term of the lease shall end 60 months after the Lease Commencement Date. Under the terms of the lease, following the first month (during which the base rent is $0) and the second month (during which the base rent is $15,990), the base rent, subject to certain adjustments, for the leased premises will start at approximately $19,412 per month, plus certain operating expenses and taxes, and shall increase on an annual basis and/or as otherwise provided in the lease agreement. In addition, on May 14, 2018, the Company provided notice of early lease termination for its second Ann Arbor lease of 5,500 square feet to end its tenancy effective November 15, 2018.

 

There have been no other material changes to the Company’s contractual obligations and commitments and contingencies outside the ordinary course of business from those previously disclosed in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2017.

 

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6. Investments

 

The following table summarizes the Company’s cash equivalents and investments:

 

		September 30, 2018
		Amortized Cost			Gross Unrealized Gains			Gross Unrealized Losses			Estimated Fair Value
					(in thousands)
Cash equivalents:
Money market funds		$	22,071		$	—		$	—		$	22,071
Short-term investments:
Certificates of deposit		6,749			—			(13		)	6,736
U.S. treasury notes		57,401			—			(248		)	57,153
U.S. government agency securities		68,864			—			(290		)	68,574
Long-term investments:
Certificates of deposit		245			—			(2		)	243
U.S. treasury notes		4,945			—			(7		)	4,938
Total		$	160,275		$	—		$	(560	)	$	159,715

 

		December 31, 2017
		Amortized Cost			Gross Unrealized Gains			Gross Unrealized Losses			Estimated Fair Value
					(in thousands)
Cash equivalents:
Money market funds		$	27,302		$	—		$	—		$	27,302
U.S. treasury notes		2,999			—			—			2,999
Short-term investments:
Certificates of deposit		12,429			1			(13		)	12,417
U.S treasury notes		97,537			—			(225		)	97,312
U.S. government agency securities		56,143			—			(141		)	56,002
Long-term investments:
Certificates of deposit		3,863			—			(10		)	3,853
U.S. treasury notes		27,983			—			(209		)	27,774
U.S. government agency securities		42,041			—			(248		)	41,793
Total		$	270,297		$	1		$	(846	)	$	269,452

 

At September 30, 2018, remaining contractual maturities of investments classified as current on the balance sheets were less than 12 months and remaining contractual maturities of investments classified as long-term were less than two years.

 

During the three and nine months ended September 30, 2018, other income, net in the statements of operations includes interest income on investments of $0.6 million and $2.1 million, and income for the accretion of premiums and discounts on investments of less than $0.1 million and $0.1 million, respectively. During the three and nine months ended September 30, 2017, other income, net in the statements of operations includes interest income on available-for-sale investments of $0.7 million and $1.7 million, and expense for the amortization of premiums and discounts on investments of less than $0.1 million and $0.3 million, respectively.

 

There were no unrealized gains or losses on investments reclassified from accumulated other comprehensive loss to other income in the statements of operations during the three and nine months ended September 30, 2018 and 2017.

 

7. Fair Value Measurements

 

The Company follows accounting guidance that emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Fair value is defined as “the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date.” Fair value measurements are defined on a three level hierarchy:

 

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Level 1 inputs:		Quoted prices for identical assets or liabilities in active markets;
Level 2 inputs:		Observable inputs other than Level 1 prices, such as quoted market prices for similar assets or liabilities or other inputs that are observable or can be corroborated by market data; and
Level 3 inputs:		Unobservable inputs that are supported by little or no market activity and require the reporting entity to develop assumptions that market participants would use when pricing the asset or liability.

 

The following table presents the Company’s financial assets and liabilities that have been measured at fair value on a recurring basis:

 

Description		Total			Level 1			Level 2			Level 3
					(in thousands)
September 30, 2018
Assets:
Money market funds		$	22,071		$	22,071		$	—		$	—
Investments:
Certificates of deposit		6,979			6,979			—			—
U.S. treasury notes		62,091			62,091			—			—
U.S. government agency securities		68,574			—			68,574			—
Total assets at fair value		$	159,715		$	91,141		$	68,574		$	—
December 31, 2017
Assets:
Money market funds		$	27,302		$	27,302		$	—		$	—
Available-for-sale securities:
Certificates of deposit		16,270			16,270			—			—
U.S. treasury notes		128,085			128,085			—			—
U.S. government agency securities		97,795			—			97,795			—
Total assets at fair value		$	269,452		$	171,657		$	97,795		$	—

 

There were no transfers between Levels 1, 2 or 3 during the three and nine months ended September 30, 2018 and 2017.

 

8. Stock Compensation

 

2017 Inducement Equity Plan

 

In May 2017, the Company’s board of directors approved the 2017 Inducement Equity Plan (the “2017 Plan”). The number of shares of common stock available for awards under the 2017 Plan was set to 750,000, with any shares of common stock that are forfeited, cancelled, held back upon the exercise or settlement of an award to cover the exercise price or tax withholding, reacquired by the Company prior to vesting, satisfied without the issuance of common stock, or otherwise terminated (other than by exercise) under the 2017 Plan added back to the shares of common stock available for issuance under the 2017 Plan.

 

2013 Stock Option and Incentive Plan

 

In May 2015, the Company’s stockholders approved the amended and restated 2013 Stock Option and Incentive Plan (as amended, the “2013 Plan”). The number of shares of common stock available for awards under the 2013 Plan was set to 2,975,000 shares, plus (i) shares of common stock that are forfeited, cancelled, held back upon the exercise or settlement of an award to cover the exercise price or tax withholding, reacquired by the Company prior to vesting, satisfied without the issuance of common stock or otherwise terminated (other than by exercise) under the 2013 Plan and the Company’s 2008 Incentive Stock Option and Restricted Stock Plan are added back to the shares of common stock available for issuance under the 2013 Plan, and (ii) on January 1, 2016, and each January 1, thereafter, the number of shares of common stock reserved and available for issuance under the 2013 Plan will be cumulatively increased by 2.5% of the number of shares of common stock outstanding on the immediately preceding December 31, or such lesser number of shares of common stock determined by the compensation committee.

 

The 2017 Plan provides for the granting of stock options, stock appreciation rights, restricted stock awards, restricted stock units (“RSUs”), unrestricted stock awards and dividend equivalent rights. The 2013 Plan provides for the

 

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granting of stock options, stock appreciation rights, restricted stock awards, RSUs, unrestricted stock awards, cash-based awards, performance share awards and dividend equivalent rights. The Company incurs stock-based compensation expense related to stock options and RSUs. The fair value of RSUs is determined by the closing market price of the Company’s common stock on the date of grant. The fair value of stock options is calculated using a Black-Scholes option pricing model. The Company accounts for stock-based compensation in accordance with the provisions of ASC 718, Compensation—Stock Compensation. Accordingly, compensation costs related to equity instruments granted are recognized over the requisite service periods of the awards on a straight-line basis at the grant-date fair value. In accordance with the adoption of ASU 2016-09, the Company accounts for forfeitures as they occur.

 

The following table summarizes the activity relating to the Company’s options to purchase common stock for the nine months ended September 30, 2018:

 

		Number of Options		Weighted-Average Exercise Price Per Share			Weighted-Average Remaining Contractual Term (Years)		Aggregate Intrinsic Value
									(in thousands)
Outstanding at December 31, 2017		4,159,151		$	28.13		7.39		$	165,385
Granted		1,096,827		$	60.30
Forfeited or expired		(362,415	)	$	40.57
Exercised		(336,875	)	$	33.84
Outstanding at September 30, 2018		4,556,688		$	34.46		7.23		$	78,758

 

The following table summarizes information about the Company’s stock option plan as of September 30, 2018:

 

		Number of Options		Weighted-Average Exercise Price Per Share			Weighted-Average Remaining Contractual Term (Years)		Aggregate Intrinsic Value
									(in thousands)
Vested and expected to vest at September 30, 2018		4,556,688		$	34.46		7.23		$	78,758
Exercisable at September 30, 2018		2,738,990		$	28.19		6.18		$	59,605

 

During the three and nine months ended September 30, 2018, the Company recognized $5.3 million and $16.6 million, respectively, of stock-based compensation expense related to stock options. During the three and nine months ended September 30, 2017, the Company recognized $4.9 million and $13.8 million, respectively, of stock-based compensation expense related to stock options. As of September 30, 2018, there was $46.3 million of unrecognized stock-based compensation expense related to unvested options, which will be recognized over a weighted-average period of 2.9 years.

 

The following table summarizes the activity relating to the Company’s RSUs for the nine months ended September 30, 2018:

 

		Number of RSUs		Weighted-Average Fair Value Per Share
Outstanding and unvested at December 31, 2017		10,003		$	57.54
Granted		34,000		$	67.94
Forfeited or expired		(4,691	)	$	57.54
Vested		(2,812	)	$	57.54
Outstanding and unvested at September 30, 2018		36,500		$	67.23

 

During the three and nine months ended September 30, 2018, the Company recognized $0.2 million and $0.5 million, respectively, of stock-based compensation expense related to RSUs. During the three and nine months ended September 30, 2017, the Company recognized $0.1 million and $0.3 million, respectively, of stock-based compensation expense recognized related to RSUs. As of September 30, 2018, there was $2.1 million of unrecognized stock-based compensation expense related to unvested RSUs, which will be recognized over a weighted-average period of 3.3 years.

 

9. Income Taxes

 

There was no provision for income taxes for the three and nine months ended September 30, 2018 and 2017, because the Company has incurred operating losses since inception. At September 30, 2018, the Company concluded that it is

 

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not more likely than not that the Company will realize the benefit of its deferred tax assets due to its history of losses. Accordingly, a full valuation allowance has been applied against the net deferred tax assets.

 

10. Net Loss Per Common Share

 

Basic net loss per share is calculated by dividing net loss by the weighted-average number of common shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is computed by dividing net loss by the weighted-average number of common stock equivalents outstanding for the period determined using the treasury-stock method. For purposes of this calculation, warrants for common stock, stock options and unvested RSUs are considered to be common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive.

 

The shares outstanding at the end of the respective periods presented below were excluded from the calculation of diluted net loss per share due to their anti-dilutive effect:

 

		September 30, 2018		December 31, 2017
Warrants for common stock		8,230		185,353
Common shares under option		4,556,688		4,159,151
Unvested RSUs		36,500		10,003
Total potential dilutive shares		4,601,418		4,354,507

 

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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

 

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our condensed financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and our annual report on Form 10-K for the fiscal year ended December 31, 2017.

 

Forward-Looking Statements

 

This Quarterly Report on Form 10-Q contains “forward-looking statements” within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act). These forward-looking statements are based on our management’s belief and assumptions and on information currently available to management. Although we believe that the expectations reflected in these forward-looking statements are reasonable, these statements relate to future events, including our clinical development plans, or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements, including in relation to the clinical development of the bempedoic acid / ezetimibe combination pill and bempedoic acid to be materially different from any future results, performance or achievements, including in relation to the clinical development of the bempedoic acid / ezetimibe combination pill and bempedoic acid, expressed or implied by these forward-looking statements.

 

Forward-looking statements are often identified by the use of words such as, but not limited to, “may,” “will,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue” or the negative of these terms or other similar terminology. These statements are only predictions. You should not place undue reliance on forward-looking statements because they involve known and unknown risks, uncertainties and other factors, which are, in some cases, beyond our control and that could materially affect results. Factors that may cause actual results to differ materially from current expectations include, among other things, those referred to or discussed in or incorporated by reference into the section titled “Risk Factors” included in Item 1A of Part II of this Quarterly Report on Form 10-Q. If one or more of these risks or uncertainties occur, or if our underlying assumptions prove to be incorrect, actual events or results may vary significantly from those implied or projected by the forward-looking statements. No forward-looking statement is a guarantee of future performance.

 

The forward-looking statements in this report represent our views as of the date of this quarterly report. Except as required by law, we undertake no obligation to update any forward-looking statements to reflect events or circumstances after the date of such statements.

 

Overview

 

Corporate Overview

 

We are the Lipid Management Company, a late-stage pharmaceutical company focused on developing and commercializing complementary, convenient, cost-effective, once-daily, oral therapies for the treatment of patients with elevated low density lipoprotein cholesterol, or LDL-C. Through scientific and clinical excellence, and a deep understanding of cholesterol biology, the experienced lipid management team at Esperion is committed to developing new LDL-C lowering therapies that will make a substantial impact on reducing global cardiovascular disease, or CVD; the leading cause of death around the world. Bempedoic acid and our lead product candidate, the bempedoic acid / ezetimibe combination pill, are targeted therapies that have been shown to significantly lower elevated LDL-C levels in patients with hypercholesterolemia, including patients inadequately treated with current lipid-modifying therapies.

 

The clinical development program for the bempedoic acid / ezetimibe combination pill consisted of a single pivotal Phase 3 study (1002FDC-053) in patients with hypercholesterolemia and with atherosclerotic cardiovascular disease, or ASCVD, and/or heterozygous familial hypercholesterolemia, or HeFH, including high CVD risk primary prevention patients, whose LDL-C is not adequately controlled despite receiving maximally tolerated lipid-modifying background therapy. 1002FDC-053 initiated in November 2017, fully enrolled 382 patients in March 2018, and we reported top-line results in August 2018.

 

The global pivotal Phase 3 clinical development program for bempedoic acid, consisting of four clinical studies, is complete with 3,621 high CVD risk patients with hypercholesterolemia and ASCVD and/or HeFH, or who are high CVD risk primary prevention, on optimized background lipid-modifying therapy and with elevated levels of LDL-C. These patients are on two distinct types of background lipid-modifying therapy: 1) patients on their maximally tolerated statin therapy, and 2)

 

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patients who are only able to tolerate less than the lowest approved daily starting dose of a statin, and can be considered statin intolerant. In March 2018, we reported top-line results from the first of the Phase 3 studies, Study 4 (1002-048). In May 2018, we reported top-line results from the 52-week long-term safety study, Study 1 (1002-040), and from Study 3 (1002-046). In October 2018, we reported top-line results from Study 2 (1002-047).

 

We intend to use positive results from our Phase 3 bempedoic acid / ezetimibe combination pill and bempedoic acid programs with a total of 4,003 patients to support global regulatory submissions for tandem LDL-C lowering indications in the U.S. no later than the first quarter of 2019 and in Europe no later than the second quarter of 2019.

 

We are also conducting a global cardiovascular outcomes trial, or CVOT, — known as Cholesterol Lowering via BEmpedoic Acid, an ACL-inhibiting Regimen (CLEAR) Outcomes, for bempedoic acid in 12,604 patients with hypercholesterolemia and high CVD risk and who can be considered statin intolerant.  We initiated the CLEAR Outcomes CVOT in December 2016 and expect the study to be fully enrolled in 2019, and intend to use positive results from this CVOT to support submissions for a CV risk reduction indication in the U.S. and Europe by 2022.

 

We were incorporated in Delaware in January 2008, and commenced our operations in April 2008. Since our inception, we have focused substantially all of our efforts and financial resources on developing the bempedoic acid / ezetimibe combination pill and bempedoic acid. We have funded our operations to date primarily through proceeds from sales of preferred stock, convertible promissory notes and warrants, public offerings of common stock and the incurrence of indebtedness, and we have incurred losses in each year since our inception. We own the exclusive worldwide rights to bempedoic acid.

 

We do not have any products approved for sale. To date, we have not generated any revenue. We have never been profitable and our net losses were $49.9 million and $45.2 million for the three months ended September 30, 2018 and 2017, respectively, and were $141.8 million and $129.1 million for the nine months ended September 30, 2018 and 2017, respectively. Substantially all of our net losses resulted from costs incurred in connection with research and development programs, general and administrative costs associated with our operations. We expect to incur significant expenses and increasing operating losses for the foreseeable future. We expect our expenses to increase in connection with our ongoing activities, including, among others:

 

·                  completing the clinical development of bempedoic acid, including the completion of the global pivotal Phase 3 LDL-C lowering program and the CLEAR Outcomes CVOT;

 

·                  completing the clinical development activities for the bempedoic acid / ezetimibe combination pill;

 

·                  seeking regulatory approval for the bempedoic acid / ezetimibe combination pill and bempedoic acid;

 

·                  commercializing the bempedoic acid / ezetimibe combination pill and bempedoic acid, if approved; and

 

·                  operating as a public company.

 

Accordingly, we will need additional financing to support our continuing operations. We will seek to fund our operations through public or private equity or debt financings or through other sources, which may include collaborations with third parties. Adequate additional financing may not be available to us on acceptable terms, or at all. Our failure to raise capital as and when needed would have a material adverse effect on our financial condition and our ability to pursue our business strategy or continue operations. We will need to generate significant revenues to achieve profitability, and we may never do so.

 

Product Overview

 

Through the complementary mechanisms of action of inhibition of cholesterol synthesis (bempedoic acid) and inhibition of cholesterol absorption (ezetimibe), the bempedoic acid / ezetimibe combination pill is our lead, non-statin, orally available, once-daily, LDL-C lowering therapy. Inhibition of ATP citrate lyase, or ACL, by bempedoic acid reduces cholesterol biosynthesis and lowers LDL-C by up-regulating the LDL receptor. Inhibition of Niemann-Pick C1-Like 1 by ezetimibe results in reduced absorption of cholesterol from the gastrointestinal tract, thereby reducing delivery of cholesterol to the liver, which in turn upregulates the LDL receptors. Phase 3 data demonstrated that this safe and well tolerated combination results in a 35 percent lowering of LDL-C when used with maximally tolerated statins, a 43 percent lowering of LDL-C when used as a monotherapy, and a 34 percent reduction in high sensitivity C-reactive protein, or hsCRP. The bempedoic acid / ezetimibe combination pill is being developed for patients at high CVD risk with hypercholesterolemia.

 

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With a targeted mechanism of action, bempedoic acid is a first-in-class, complementary, orally available, once-daily ACL inhibitor that, similar to statins, reduces cholesterol biosynthesis and lowers LDL-C by up-regulating the LDL receptor. Similar to statins, bempedoic acid also reduces hsCRP, a key marker of inflammation associated with cardiovascular disease. Completed Phase 2 and Phase 3 studies conducted in almost 4,800 patients, including approximately 3,100 patients treated with bempedoic acid, have produced an additional 20 percent LDL-C lowering when used with maximally tolerated statins, up to 30 percent LDL-C lowering as a monotherapy, 35 percent in combination with ezetimibe when used with maximally tolerated statins, and up to 48 percent LDL-C lowering in combination with ezetimibe as monotherapy. Bempedoic acid is being developed for patients at high CVD risk with hypercholesterolemia. We acquired the rights to bempedoic acid from Pfizer in 2008. We own the exclusive worldwide rights to bempedoic acid and we are not obligated to make any royalty or milestone payments to Pfizer.

 

During the nine months ended September 30, 2018, we incurred $90.1 million in expenses related to the four studies in our global pivotal Phase 3 LDL-C lowering program, our CLEAR Outcomes CVOT, our 1002FDC-053 study, our open-label extension study, our 1002FDC-058 study and our Phase 2 (1002-39) clinical study of bempedoic acid when added-on to an injectable proprotein convertase subtilisin/kexin type 9 inhibitor, or PCSK9i, therapy in patients with hypercholesterolemia.

 

During the nine months ended September 30, 2017, we incurred $85.9 million in expenses related to the four studies in our global pivotal Phase 3 LDL-C lowering program, our CLEAR Outcomes CVOT, our Phase 2 (1002-038) clinical study of the bempedoic acid / ezetimibe combination plus atorvastatin with bempedoic acid 180 mg, ezetimibe 10 mg and atorvastatin 20 mg in patients with hypercholesterolemia and our Phase 2 (1002-39) clinical study of bempedoic acid when added-on to a PCSK9i in patients with hypercholesterolemia.

 

Program Developments

 

On October 28, 2018, we announced the completion of the Phase 3 LDL-C lowering development program of bempedoic acid and positive cumulative results. The Phase 3 program included 3,621 high cardiovascular risk patients taking maximally tolerated statin, which could include no statin, who required additional LDL-C lowering. The program achieved its efficacy endpoints and other key measures at 12 weeks for bempedoic acid, including:

 

·            On-treatment LDL-C lowering of an additional 18 percent to 31 percent (vs. placebo, p<0.001), and in the intent to treat analysis LDL-C lowering of an additional 17 percent to 28 percent (p<0.001).

 

·            Reductions of 19 percent to 33 percent in hsCRP, an important marker of the underlying inflammation associated with cardiovascular disease.

 

·            Reductions in hemoglobin A1c, or HbA1c, of 0.19% to 0.31% vs. placebo in patients with diabetes.

 

In the Phase 3 LDL-C lowering development program, adjudicated major adverse cardiovascular events, or MACE, in the bempedoic acid arm as compared to placebo were the following:

 

Adjudicated MACE Events in Bempedoic Acid Compared to Placebo

(Cumulative Phase 3 Study Data)

 

		% of Patients
Major Adverse Cardiovascular Events (MACE)		Bempedoic Acid N=2,424		Placebo N=1,197
3-component MACE		1.9	%	2.3	%
4-component MACE		3.8	%	4.2	%
5-component MACE		4.0	%	4.6	%

 

In the Phase 3 program, bempedoic acid was observed to be safe and well-tolerated. The vast majority (>80%) of patients were studied for 52 weeks. Across the program there were no clinically relevant differences between the bempedoic acid and placebo treatment groups in the occurrence of adverse events, with summarized results as follows:

 

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Adverse Events in Treatment Groups

(Cumulative Phase 3 Study Data)

 

		% of Patients
Treatment Emergent Adverse Events (AEs)		Bempedoic Acid N=2,424		Placebo N=1,197
Overview of AEs in All Patients (patient incidence)
Any AE(s)		73	%	73	%
Serious AE(s)		14	%	13	%
Discontinuation due to AE(s)		11	%	8	%

 

		Bempedoic Acid N=2,424		Placebo N=1,197
Fatal Adverse Events — Unrelated to Study Medication
Cardiovascular death		0.4	%	0.3	%
Non-Cardiovascular death
Neoplasms		0.2	%	0.0	%
Sepsis/septic shock		0.1	%	0.1	%
Other		0.1	%	0.0	%

 

Fatal adverse events were very low overall at 0.8% and 0.3%, respectively (compared to a 1.8% annual fatality rate for people 65-74 year olds according to the CDC).

 

·            No fatal adverse events were determined by the independent study investigator to be related to study medication.

 

·            The bempedoic acid arm included a case of gas poisoning and a case of pancreatitis resulting from a pancreatic pseudocyst.

 

Phase 3 Clinical Studies Completed in 2018

 

Study 2—Global pivotal Phase 3 long-term safety and tolerability study in patients with hypercholesterolemia on maximally tolerated background lipid-modifying therapy

 

On October 28, 2018, we announced the top-line results from the pivotal Phase 3 study, Study 2 (1002-047). The 52-week, global, pivotal, Phase 3 randomized, double-blind, placebo-controlled, multicenter study evaluated the efficacy and safety of bempedoic acid 180 mg/day versus placebo in high CVD risk patients with hypercholesterolemia with ASCVD and/or HeFH, whose LDL-C is inadequately controlled with current lipid-modifying therapies, and who are taking maximally tolerated statin therapy. The study was conducted at 93 sites in North America and Europe. A total of 779 patients were randomized 2:1 to receive bempedoic acid or placebo. The primary objective was to assess the 12-week LDL-C lowering efficacy of patients treated with bempedoic versus placebo. Secondary objectives included evaluating the safety and tolerability of bempedoic acid versus placebo, the 24-week and 52-week LDL-C lowering efficacy of bempedoic acid versus placebo, and its effect on other risk markers after 12 weeks of treatment, including hsCRP. While analyses of the complete efficacy and safety results from Study 2 are ongoing, the top-line results are summarized as follows:

 

LDL-Cholesterol Percent Change from Baseline to Week 12 Endpoint (LDL-C On-Treatment Analysis)

 

						LDL-C
				LDL-C		Week 12
				Baseline		Endpoint		Percent Change
		Number of		Mean (SD)		Mean (SD)		from Baseline
Treatment Group		Patients		mg/dL		mg/dL		LS Mean (SE)		P Value
Bempedoic Acid		476		119 (38	)	97 (34	)	–16% (1.1	)	<0.001
Placebo		245		122 (38	)	122 (43	)	+2% (1.4	)	—

 

LS = least squares; SD = standard deviation; SE = standard error; mITT population

 

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LDL-Cholesterol Percent Change from Baseline to Week 12 Endpoint (Intent to Treat Analysis)

 

						LDL-C
				LDL-C		Week 12
				Baseline		Endpoint		Percent Change
		Number of		Mean (SD)		Mean (SD)		from Baseline
Treatment Group		Patients		mg/dL		mg/dL		LS Mean (SE)		P Value
Bempedoic Acid		522		119 (38	)	98 (34	)	–15% (1.1	)	<0.001
Placebo		257		122 (38	)	123 (41	)	+2% (1.4	)	—

 

LS = least squares; SD = standard deviation; SE = standard error; ITT population

 

Adverse Events and Discontinuations at 52 Weeks

 

		% of Patients
Treatment Emergent Adverse Events (AEs)		Bempedoic Acid N=522		Placebo N=257
Overview of AEs in All Patients
Any AE(s)		70	%	71	%
Serious AE(s)		20	%	19	%
Discontinuation due to AE(s)		11	%	9	%

 

		Bempedoic Acid N=522		Placebo N=257
Fatal Adverse Events — Unrelated to Study Medication
Cardiovascular death		0.8	%	0.8	%
Non-Cardiovascular death
Septic shock(1)		0.2	%	0.0	%
Gas poisoning(2)		0.2	%	0.0	%

 

(1)Patient died from septic shock that was a complication of planned abdominal surgery

(2)Death was reported verbatim as CO2 gas poisoning

 

hsCRP Nonparametric Analysis

 

		Number of		Baseline		Percent Change from Baseline
Treatment Group		Patients		Level (mg/L)		Median Change		P Value
Bempedoic Acid		467		1.6		-19	%	.039
Placebo		240		1.9		-9	%	—

 

·            Bempedoic acid was observed to be safe and well-tolerated over a 52-week period. There were no clinically relevant differences between bempedoic acid and the placebo groups in the occurrence of AEs, SAEs or discontinuations due to AEs.

 

·            After twelve weeks of treatment with bempedoic acid, LDL-C levels were lowered by 18% (p<0.001), with a decrease of 16% from baseline for the patients treated with bempedoic acid and an increase of 2% for patients who received placebo.

 

·            hsCRP, a marker of the underlying inflammation associated with CVD, was reduced by 19% (p=.039) for patients dosed with bempedoic acid after twelve weeks of therapy, versus a 9% decrease with placebo.

 

·            Clinically significant reductions in total cholesterol, apoB and non-HDL-C were seen in the patients treated with bempedoic acid.

 

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1002FDC-053 — Phase 3 efficacy and safety study of the bempedoic acid / ezetimibe combination pill in patients with hypercholesterolemia

 

On August 27, 2018, we announced the top-line results from the pivotal Phase 3 bempedoic acid / ezetimibe combination pill study (1002FDC-1003). The 12-week, pivotal Phase 3 randomized, double-blind, parallel group, multicenter study evaluated the efficacy and safety of bempedoic acid 180 mg / ezetimibe 10 mg combination pill compared to bempedoic acid 180 mg alone, ezetimibe 10 mg alone or placebo in high-risk patients with ASCVD and/or HeFH or with multiple risk factors for ASCVD being treated with maximally tolerated statins. The study was conducted at 78 sites in North America. A total of 382 patients were randomized 2:2:2:1 to receive bempedoic acid 180 mg / ezetimibe 10 mg combination pill, bempedoic acid 180 mg, ezetimibe 10mg or placebo. The co-primary objectives of the study were to assess LDL-C lowering efficacy in patients treated with the bempedoic acid / ezetimibe combination pill versus placebo, 180 mg of bempedoic acid and 10 mg of ezetimibe alone. The secondary objectives included assessments of hsCRP,  non-HDL-C, total cholesterol, or TC, and apoB after 12 weeks of treatment as well as characterizing the safety and tolerability of the combination pill versus placebo alone, bempedoic acid alone, and ezetimibe alone. While analyses of the complete efficacy and safety results from 1002FDC-053 are ongoing, the top-line results are summarized as follows:

 

LDL-Cholesterol Percent Change from Baseline to Week 12 Endpoint (LDL-C On-Treatment Analysis)

 

						LDL-C
				LDL-C		Week 12
				Baseline		Endpoint		Percent Change
		Number of		Mean (SD)		Mean (SD)		from Baseline
Treatment Group		Patients		mg/dL		mg/dL		LS Mean (SE)		P Value
Bempedoic Acid / Ezetimibe Combination Pill		97		152 (39	)			–35% (2.4	)	<0.001*
Bempedoic Acid		95		146 (36	)			–20% (2.4	)	—
Ezetimibe		95		147 (39	)			–24% (2.1	)	—
Placebo		49		153 (42	)			–3% (3.4	)	—

 

*vs. placebo

 

LS = least squares; SD = standard deviation; SE = standard error; mITT population

 

LDL-Cholesterol Percent Change from Baseline to Week 12 Endpoint (Intent to Treat Analysis)

 

						LDL-C
				LDL-C		Week 12
				Baseline		Endpoint		Percent Change
		Number of		Mean (SD)		Mean (SD)		from Baseline
Treatment Group		Patients		mg/dL		mg/dL		LS Mean (SE)		P Value
Bempedoic Acid / Ezetimibe Combination Pill		108		152 (39	)			–32% (2.5	)	<0.001*
Bempedoic Acid		110		146 (36	)			–18% (2.3	)	—
Ezetimibe		109		147 (39	)			–21% (2.0	)	—
Placebo		55		153 (42	)			–3% (3.1	)	—

 

*vs. placebo

 

LS = least squares; SD = standard deviation; SE = standard error; ITT population

 

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hsCRP Nonparametric Analysis

 

		Number of		Baseline		Percent Change from Baseline
Treatment Group		Patients		Level (mg/L)		Median Change		P Value
Bempedoic Acid / Ezetimibe Combination Pill		102		3.1		–34	%	<0.05*
Bempedoic Acid		101		3.0		-20	%	—
Ezetimibe		102		3.0		-9	%	—
Placebo		52		3.0		+4	%	—

 

*versus placebo and ezetimibe

 

·            After 12 weeks of treatment with the bempedoic acid / ezetimibe combination pill, LDL-C levels were lowered by 35% (p<0.001) compared to 24% for patients who received ezetimibe, 20% for patients that received bempedoic acid, and 3% for patients that received placebo in the on-treatment analysis. The study met its primary endpoint of LDL-C lowering of 32% at 12 weeks in the intent to treat (ITT) analysis, with a 21% lowering for patients who received ezetimibe (p<0.001), 18% for patients who received bempedoic acid (p<0.001), and 3% for patients who received placebo (p<0.001).

 

·            hsCRP, a marker of inflammation associated with CVD, was reduced by 34% (p<0.05 vs. placebo and ezetimibe) for patients dosed with the bempedoic acid / ezetimibe combination pill after twelve weeks of therapy, versus a 4% increase with placebo and reductions of 20% for bempedoic acid and 9% for ezetimibe.

 

·            Clinically significant lowering of total cholesterol, apoB and non-HDL-C were seen in the patients treated with the bempedoic acid / ezetimibe combination pill and bempedoic acid.

 

·            The bempedoic acid / ezetimibe combination pill and bempedoic acid were observed to be safe and well-tolerated in this study. There were no clinically relevant differences in the occurrence of SAEs and discontinuations due to AEs among the four patient groups.

 

Study 3— Global pivotal Phase 3 LDL-C lowering efficacy and safety study in patients with hypercholesterolemia not adequately controlled with current lipid-modifying therapy and considered statin intolerant

 

On May 23, 2018, we announced the top-line results from the pivotal Phase 3 study, Study 3 (1002-046). The 24-week, global, pivotal, Phase 3 randomized, double-blind, placebo-controlled, multicenter study evaluated the LDL-C lowering efficacy and safety of bempedoic acid 180 mg/day versus placebo added to background lipid-modifying therapy in patients with hypercholesterolemia who are considered statin intolerant. The study was conducted at 67 sites in the U.S. and Canada. A total of 345 patients were randomized 2:1 to receive bempedoic acid or placebo. The primary efficacy objective was to assess the 12-week LDL-C lowering efficacy of bempedoic acid versus placebo. Secondary objectives included evaluating the 24-week LDL-C lowering efficacy of bempedoic acid versus placebo, the safety and tolerability of bempedoic acid versus placebo, and its effects on other risk markers after 12 weeks of treatment, including hsCRP. While analyses of the complete efficacy and safety results from Study 3 are ongoing, the top-line results are summarized as follows:

 

LDL-Cholesterol Percent Change from Baseline to Week 12 Endpoint (LDL-C On-Treatment Analysis)

 

						LDL-C
				LDL-C		Week 12
				Baseline		Endpoint		Percent Change
		Number of		Mean (SD)		Mean (SD)		from Baseline
Treatment Group		Patients		mg/dL		mg/dL		LS Mean (SE)		P Value
Bempedoic Acid		204		158 (41	)	116 (36	)	–26% (1.3	)	<0.001
Placebo		101		157 (40	)	153 (43	)	–2% (1.4	)	—

 

LS = least squares; SD = standard deviation; SE = standard error; mITT population

 

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