albo-10q_20180630.htm

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

FORM 10-Q

 

(Mark One)

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

For the quarterly period ended June 30, 2018

OR

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 001-33451

 

Albireo Pharma, Inc.

(Exact name of registrant as specified in its charter)

 

 

Delaware
(State or other jurisdiction of incorporation or organization)

 

90-0136863
(IRS Employer Identification No.)

 

 

 

10 Post Office Square, Suite 502 South, Boston, MA

(Address of principal executive offices)

 

02109
(Zip code)

 

Registrant’s telephone number, including area code: (857) 254-5555

 

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 Web site, if any, every Interactive Data File required to be submitted and posted 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 and post such files).    Yes      No  

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.

 

Large accelerated filer

 

Accelerated filer

Non-accelerated filer

(Do not check if a smaller reporting company)

Smaller reporting company

 

 

 

Emerging growth company

 

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. 

 

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

As of August 1, 2018, the registrant had 11,969,928 shares of common stock, $0.01 par value per share, outstanding.

 

 


Albireo Pharma, Inc.

 

 

 

Page

 

 

 

PART I — FINANCIAL INFORMATION

 

 

 

 

 

Cautionary Note Regarding Forward-Looking Statements

 

3

Item 1. Financial Statements

 

5

Condensed Consolidated Balance Sheets (unaudited) at June 30, 2018 and December 31, 2017

 

5

Condensed Consolidated Statements of Operations (unaudited) for the Three and Six Months Ended June 30, 2018 and 2017

 

6

Condensed Consolidated Statements of Comprehensive Loss (unaudited) for the Three and Six Months Ended June 30, 2018 and 2017

 

7

Condensed Consolidated Statements of Cash Flows (unaudited) for the Six Months Ended June 30, 2018 and 2017

 

8

Notes to Condensed Consolidated Financial Statements (unaudited)

 

9

 

 

 

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

 

17

 

 

 

Item 3. Quantitative and Qualitative Disclosures About Market Risk

 

26

 

 

 

Item 4. Controls and Procedures

 

26

 

 

 

PART II — OTHER INFORMATION

 

 

 

 

 

Item 6. Exhibits

 

29

 

 

 

Signatures

 

30

 

 

 

All brand names, trademarks or service marks appearing in this quarterly report are the property of their respective owners.  Registrant’s use or display of another party’s trademark, service mark, trade dress or product in this quarterly report is not intended to, and does not, imply a relationship with, or endorsement or sponsorship of, the registrant by such other party.

 

 

 

2


CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

This quarterly report includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, which we refer to as the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, which we refer to as the Exchange Act, that relate to future events or to our future operations or financial performance.  Any forward-looking statement involves known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by such forward-looking statement. Forward-looking statements include statements, other than statements of historical fact, about, among other things:

 

the progress, number, scope, cost, duration or results of our development activities, nonclinical studies and clinical trials of A4250, elobixibat, A3384 or any of our other product candidates or programs, such as the target indication(s) for development or approval, the size, design, population, conduct, cost, objective or endpoints of any clinical trial, or the timing for initiation or completion of or availability of results from any clinical trial (including our Phase 3 clinical trial of A4250 in patients with progressive familial intrahepatic cholestasis, or PFIC), for submission or approval of any regulatory filing, or for meeting with regulatory authorities;

 

the potential benefits that may be derived from any of our product candidates;

 

the timing of and our ability to obtain and maintain regulatory approval of our existing product candidates, any product candidates that we may develop, and any related restrictions, limitations, or warnings in the label of any approved product candidates;

 

any payment that HealthCare Royalty Partners III, L.P., or HCR, or EA Pharma Co., Ltd., or EA Pharma, may make to us or any other action or decision that EA Pharma may make concerning elobixibat or our business relationship;

 

our future operations, financial position, revenues, costs, expenses, uses of cash, capital requirements, our need for additional financing or the period for which our existing cash resources will be sufficient to meet our operating requirements; or

 

our strategies, prospects, plans, expectations, forecasts or objectives.

Words such as, but not limited to, “believe,” “expect,” “anticipate,” “estimate,” “forecast,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “targets,” “likely,” “will,” “would,” “could,” “should,” “continue,” “scheduled” and similar expressions or phrases, or the negative of those expressions or phrases, are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.  Although we believe that we have a reasonable basis for each forward-looking statement contained in this report, we caution you that these statements are based on our estimates or projections of the future that are subject to known and unknown risks and uncertainties and other important factors that may cause our actual results, level of activity, performance, experience or achievements to differ materially from those expressed or implied by any forward-looking statement.  Actual results, level of activity, performance, experience or achievements may differ materially from those expressed or implied by any forward-looking statement as a result of various important factors, including our critical accounting policies and risks and uncertainties relating, among other things, to:

 

the design, size, duration and endpoints for, and results from, our double blind Phase 3 clinical trial of A4250 in patients with PFIC or related extension study, or any other trials that will be required to obtain marketing approval for A4250 to treat patients with PFIC or any other pediatric cholestatic liver disease, for elobixibat to treat nonalcoholic steatohepatitis, or NASH, or for A3384 to treat bile acid malabsorption, or BAM;

 

whether favorable findings from clinical trials of A4250 to date, including findings in indications other than PFIC, will be predictive of results from future clinical trials, including the trials comprising our Phase 3 PFIC program for A4250;

 

whether either or both of the U.S. Food and Drug Administration, or FDA, and European Medicines Agency, or EMA, will determine that the primary endpoint and treatment duration of the Phase 3 trial in patients with PFIC are sufficient, even if such primary endpoint is met with statistical significance, to support approval of A4250 in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or otherwise;

 

the outcome and interpretation by regulatory authorities of an ongoing third-party study pooling and analyzing long-term PFIC patient data;

 

the timing for completion of, or for availability of data from, the trials comprising the Phase 3 PFIC program for A4250, and the outcomes of such trials;

 

delays or other challenges in the recruitment of patients for the Phase 3 trial of A4250;

3


 

whether we will receive a rare pediatric disease priority review voucher for A4250 from the FDA when applicable, whether a rare pediatric disease priority review voucher that we may receive in the future for A4250, if any, will be valuable to us, and, if necessary, whether the rare pediatric disease priority review voucher program will be renewed beyond 2020;

 

the competitive environment and commercial opportunity for a potential treatment for PFIC and other orphan pediatric cholestatic liver diseases;

 

the conduct and results of clinical trials and nonclinical studies and assessments of A4250, elobixibat, A3384 or any of our other product candidates and programs, including the performance of third parties engaged to execute them and difficulties or delays in patient enrollment and data analysis;

 

the medical benefit that may be derived from A4250, elobixibat, A3384 or any of our other product candidates;

 

the extent to which our agreements with HCR and EA Pharma for elobixibat generate nondilutive income for us;

 

the timing and success of submission, acceptance and approval of regulatory filings and any related restrictions, limitations or warnings in the label of any approved product candidates;

 

the significant control or influence that EA Pharma has over the commercialization of elobixibat in Japan and the development and commercialization of elobixibat in EA Pharma’s other licensed territories;

 

whether we elect to seek and, if so, our ability to establish a license or other partnering transaction with a third party for elobixibat in the United States or Europe;

 

whether findings from nonclinical studies and clinical trials of IBAT inhibitors will be predictive of future clinical success for a product candidate of ours in the treatment of NASH;

 

the accuracy of our estimates regarding expenses, costs, future revenues, uses of cash and capital requirements;

 

our ability to obtain additional financing on reasonable terms, or at all;

 

our ability to establish additional licensing, collaboration or similar arrangements on favorable terms and our ability to attract collaborators with development, regulatory and commercialization expertise;

 

the success of competing third-party products or product candidates;

 

our ability to successfully commercialize any approved product candidates, including their rate and degree of market acceptance;

 

our ability to expand and protect our intellectual property estate;

 

regulatory developments in the United States and other countries;

 

our ability to fully remediate our identified internal control material weaknesses by our planned timeframe;

 

the performance of our third-party suppliers, manufacturers and contract research organizations and our ability to obtain alternative sources of raw materials;

 

our ability to attract and retain key personnel; and

 

our ability to comply with regulatory requirements relating to our business, and the costs of compliance with those requirements, including those on data privacy and security.

These and other risks and uncertainties are described in greater detail under the caption “Risk Factors” in Item 1A of Part I of our Annual Report on Form 10-K for the fiscal year ended December 31, 2017, in item 1A of Part II of this quarterly report, and in other filings that we make with the Securities and Exchange Commission, or SEC. As a result of the risks and uncertainties, the results or events indicated by the forward-looking statements may not occur. We caution you not to place undue reliance on any forward-looking statement.

In addition, any forward-looking statement in this quarterly report represents our views only as of the filing date of this quarterly report and should not be relied upon as representing our views as of any subsequent date. We anticipate that subsequent events and developments may cause our views to change. Although we may elect to update these forward-looking statements publicly at some point in the future, we specifically disclaim any obligation to do so, except as required by applicable law. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make.


4


PART I — FINANCIAL INFORMATION

Item 1.  Financial Statements

Albireo Pharma, Inc.

Condensed Consolidated Balance Sheets

(in thousands, except share and per share data)

(unaudited)

 

 

 

June 30, 2018

 

 

December 31, 2017

 

ASSETS

 

 

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

 

 

Cash and cash equivalents

 

$

183,228

 

 

$

53,231

 

Prepaid expenses and other assets

 

 

610

 

 

 

1,054

 

Other receivables

 

 

2,406

 

 

 

726

 

Total current assets

 

 

186,244

 

 

 

55,011

 

Property and equipment, net

 

 

210

 

 

 

178

 

Goodwill

 

 

17,260

 

 

 

17,260

 

Other noncurrent assets

 

 

422

 

 

 

775

 

Total assets

 

$

204,136

 

 

$

73,224

 

LIABILITIES AND STOCKHOLDERS' EQUITY

 

 

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

 

 

Trade payables

 

$

3,020

 

 

$

1,350

 

Accrued expenses

 

 

4,353

 

 

 

6,105

 

Other liabilities

 

 

541

 

 

 

474

 

Total current liabilities

 

 

7,914

 

 

 

7,929

 

Liability related to sale of future royalties

 

 

46,736

 

 

 

 

Long-term liabilities

 

 

39

 

 

 

42

 

Total liabilities

 

 

54,689

 

 

 

7,971

 

Stockholders' Equity:

 

 

 

 

 

 

 

 

Common stock, $0.01 par value per share — 30,000,000 authorized at June 30,

   2018 and December 31, 2017; 11,957,491 and 8,902,784

   issued and outstanding at June 30, 2018 and December 31, 2017, respectively

 

 

120

 

 

 

89

 

Additional paid in capital

 

 

211,140

 

 

 

114,522

 

Accumulated other comprehensive income

 

 

4,768

 

 

 

1,001

 

Accumulated deficit

 

 

(66,581

)

 

 

(50,359

)

Total stockholders’ equity

 

 

149,447

 

 

 

65,253

 

Total liabilities and stockholders’ equity

 

$

204,136

 

 

$

73,224

 

 

See accompanying notes to Condensed Consolidated Financial Statements.

5


Albireo Pharma, Inc.

Condensed Consolidated Statements of Operations

(in thousands, except share and per share data)

(unaudited)

 

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

 

 

2018

 

 

2017

 

 

2018

 

 

2017

 

Revenue

 

$

730

 

 

$

1

 

 

$

11,932

 

 

$

2

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

 

6,411

 

 

 

2,962

 

 

 

12,562

 

 

 

5,774

 

General and administrative

 

 

4,238

 

 

 

3,713

 

 

 

8,366

 

 

 

6,925

 

Other operating (income) expense, net

 

 

487

 

 

 

(65

)

 

 

1,991

 

 

 

9

 

Total operating expenses

 

 

11,136

 

 

 

6,610

 

 

 

22,919

 

 

 

12,708

 

Operating loss

 

 

(10,406

)

 

 

(6,609

)

 

 

(10,987

)

 

 

(12,706

)

Interest income (expense), net

 

 

(1,666

)

 

 

(152

)

 

 

(2,682

)

 

 

(401

)

Other income (expense), net

 

 

(2,531

)

 

 

585

 

 

 

(2,553

)

 

 

260

 

Net loss before income taxes

 

 

(14,603

)

 

 

(6,176

)

 

 

(16,222

)

 

 

(12,847

)

Income tax

 

 

 

 

 

 

 

 

 

 

 

 

Net loss

 

$

(14,603

)

 

$

(6,176

)

 

$

(16,222

)

 

$

(12,847

)

Net loss per share - basic and diluted

 

$

(1.22

)

 

$

(0.86

)

 

$

(1.42

)

 

$

(1.91

)

Weighted average shares outstanding - basic and diluted

 

 

11,938,357

 

 

 

7,171,610

 

 

 

11,417,463

 

 

 

6,734,555

 

 

See accompanying notes to Condensed Consolidated Financial Statements.

6


Albireo Pharma, Inc.

Condensed Consolidated Statements of Comprehensive Loss

(in thousands)

(unaudited)

 

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

 

 

2018

 

 

2017

 

 

2018

 

 

2017

 

Net loss

 

$

(14,603

)

 

$

(6,176

)

 

$

(16,222

)

 

$

(12,847

)

Other comprehensive loss:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Foreign currency translation adjustment

 

 

2,573

 

 

 

389

 

 

 

3,767

 

 

 

(90

)

Total other comprehensive income (loss)

 

 

2,573

 

 

 

389

 

 

 

3,767

 

 

 

(90

)

Total comprehensive loss

 

$

(12,030

)

 

$

(5,787

)

 

$

(12,455

)

 

$

(12,937

)

 

See accompanying notes to Condensed Consolidated Financial Statements.

7


Albireo Pharma, Inc.

Condensed Consolidated Statements of Cash Flows

(in thousands)

(unaudited)

 

 

 

Six Months Ended June 30,

 

 

 

2018

 

 

2017

 

Cash flows from operating activities:

 

 

 

 

 

 

 

 

Net loss

 

$

(16,222

)

 

$

(12,847

)

Adjustments to reconcile net loss to net cash used in operating activities:

 

 

 

 

 

 

 

 

Non cash interest expense on liability related to royalty monetization

 

 

2,028

 

 

 

 

Accretion of debt discount and amortization of issuance costs

 

 

 

 

 

301

 

Depreciation and amortization

 

 

22

 

 

 

13

 

Change in fair value of financial instruments

 

 

 

 

 

(251

)

Stock-based compensation expense

 

 

2,244

 

 

 

2,051

 

Unrealized foreign exchange (gain) loss

 

 

6,718

 

 

 

 

Changes in operating assets and liabilities:

 

 

 

 

 

 

 

 

Trade receivables

 

 

(754

)

 

 

27

 

Prepaid expenses and other current assets

 

 

432

 

 

 

94

 

Other receivables

 

 

(1,055

)

 

 

(292

)

Other non current assets

 

 

354

 

 

 

(12

)

Trade payables

 

 

1,867

 

 

 

280

 

Accrued expenses

 

 

(2,223

)

 

 

(3,864

)

Other liabilities and long-term liabilities

 

 

70

 

 

 

29

 

Other non current liabilities

 

 

 

 

 

43

 

Net cash used in operating activities

 

 

(6,519

)

 

 

(14,428

)

Cash flows from investing activities:

 

 

 

 

 

 

 

 

Purchase of property, plant and equipment

 

 

(61

)

 

 

(146

)

Net cash used in investing activities

 

 

(61

)

 

 

(146

)

Cash flows from financing activities:

 

 

 

 

 

 

 

 

Proceeds from issuance of common stock, net of issuance costs

 

 

94,149

 

 

 

48,508

 

Proceeds from royalty agreement

 

 

44,525

 

 

 

 

Exercise of options

 

 

255

 

 

 

62

 

Payments of principal on borrowings

 

 

 

 

 

(1,648

)

Net cash provided by financing activities

 

 

138,929

 

 

 

46,922

 

Effect of exchange rate changes on cash and cash equivalents

 

 

(2,352

)

 

 

319

 

Net increase in cash and cash equivalents

 

 

129,997

 

 

 

32,667

 

Cash and cash equivalents—beginning of period

 

 

53,231

 

 

 

29,931

 

Cash and cash equivalents—end of period

 

$

183,228

 

 

$

62,598

 

Supplemental disclosures of cash flow information:

 

 

 

 

 

 

 

 

Cash paid for interest

 

$

 

 

$

136

 

Shares issued upon cashless exercise of Kreos warrants

 

 

 

 

 

617

 

 

See accompanying notes to Condensed Consolidated Financial Statements.

8


Albireo Pharma, Inc.

Notes to Condensed Consolidated Financial Statements

(unaudited)

1.  Summary of significant accounting policies and basis of presentation

Organization

Albireo Pharma, Inc. (Parent), together with its direct and indirect subsidiaries (the Company), is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel bile acid modulators to treat orphan pediatric liver diseases and other liver and gastrointestinal diseases and disorders. The Company’s clinical pipeline includes a Phase 3 product candidate, a Phase 2 product candidate, and elobixibat, which is approved in Japan for the treatment of chronic constipation.  We also anticipate commencing a development program with elobixibat in nonalcoholic steatohepatitis or NASH.  A4250, the Company’s Phase 3 lead product candidate, is in development initially for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC), a rare, life-threatening genetic disorder affecting young children.

Basis of presentation

The accompanying unaudited Condensed Consolidated Financial Statements have been prepared in accordance with U.S. generally accepted accounting principles (GAAP) for interim financial information, and the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements and should be read in conjunction with the audited consolidated financial statements and accompanying notes included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2017.  In the opinion of management, all adjustments (including normal recurring accruals) considered necessary for fair presentation have been included in the Condensed Consolidated Financial Statements.  The results of operations for the three and six months ended June 30, 2018 are not necessarily indicative of the results that may be expected for the full fiscal year, any other interim period or any future fiscal year.  The condensed consolidated financial statements are prepared on a basis consistent with prior periods except for the adoption of the new revenue recognition standard discussed below.

Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (ASC) and Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB).  

Principles of consolidation

The accompanying Condensed Consolidated Financial Statements include the accounts of Parent and its direct or indirect wholly owned subsidiaries, Albireo Limited, Albireo AB, Elobix AB, Albireo, Inc., Albireo Security Corp. and Biodel UK Limited. All intercompany balances and transactions have been eliminated in consolidation.

Foreign currency translation

Functional and presentation currency

Items included in the financial statements of each entity comprising the Company are measured using the currency of the primary economic environment in which the entity operates (the functional currency). The functional currency for Parent, Albireo, Inc. and Albireo Security Corp. is the U.S. Dollar (USD), the functional currency for Albireo Limited, Elobix AB and Biodel UK Limited is the Euro, and the functional currency for Albireo AB is the Swedish Krona (SEK). The Company consolidates its financial statements in USD.

Transactions and balances

Foreign currency transactions in each entity comprising the Company are remeasured into the functional currency of the entity using the exchange rates prevailing at the respective transaction dates. Foreign exchange gains and losses resulting from the settlement of such transactions and from the translation at period-end exchange rates of monetary assets and liabilities denominated in foreign currencies are recognized within Other (income) expense, net in the Condensed Consolidated Statements of Operations as part of operating expenses.

9


The results and financial position of the Company that have a functional currency different from the USD are translated into the presentation currency as follows:

 

a.

assets and liabilities presented are translated at the closing exchange rate as of June 30, 2018 and December 31, 2017;

 

b.

income and expenses for each statement of comprehensive income (loss) are translated at the average exchange rate for the applicable period;

 

c.

significant transactions use the closing exchange rate on the date of the transaction; and

 

d.

all resulting exchange differences arising from such translations are recognized directly in other comprehensive income (loss) and presented as a separate component of equity.

Use of estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts of assets, liabilities, revenues and expenses reported in the financial statements and accompanying notes. Management must apply significant judgment in this process. On an ongoing basis, the Company evaluates its estimates and assumptions, including but not limited to accruals, deferred tax assets and royalty monetization liability estimated at fair value.  Actual results could materially differ from these estimates.

Fair value of financial instruments

When measuring the fair value of financial instruments, the Company evaluates valuation techniques such as the market approach, the income approach and the cost approach. A three-level valuation hierarchy, which prioritizes the inputs to valuation techniques that are used to measure fair value, is based upon whether such inputs are observable or unobservable.

Observable inputs reflect market data obtained from independent sources, while unobservable inputs reflect market assumptions made by the reporting entity. The three-level hierarchy for the inputs to valuation techniques is briefly summarized as follows:

Level 1—Observable inputs such as quoted prices (unadjusted) for identical instruments in active markets;

Level 2—Observable inputs such as quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, or model-derived valuations whose significant inputs are observable for substantially the full term of the assets or liabilities; and

Level 3—Unobservable inputs that reflect the reporting entity’s estimate of assumptions that market participants would use in pricing the asset or liability.

Research and development expenses

Research and development costs are expensed as incurred and include primarily salaries, benefits and other staff-related costs; clinical trial and related clinical manufacturing costs; contract services and other outside costs.

The Company’s nonclinical studies and clinical trials are performed by third-party contract research organizations (CROs). Some of these expenses are billed monthly for services performed, while others are billed based upon milestones achieved. For nonclinical studies, the significant factors used in estimating accruals include the percentage of work completed to date and contract milestones achieved. For clinical trial expenses, the significant factors used in estimating accruals include the number of patients enrolled and percentage of work completed to date or contract milestones achieved. The Company’s estimates are highly dependent upon the timeliness and accuracy of the data provided by the respective CROs regarding the status of the contracted activity, with adjustments made when deemed necessary.

Revenue recognition

In 2012, the Company entered into a license agreement (the Agreement) with EA Pharma Co., Ltd. (EA Pharma, formerly Ajinomoto Pharmaceuticals Co., Ltd.) to develop a select product candidate (elobixibat) for registration and subsequent commercialization in select markets. In conjunction with the Agreement, the Company granted EA Pharma an exclusive license to its intellectual property for development and commercialization activities in the designated field and territories. The Company is entitled to payments resulting from pharmaceutical ingredient or related procurement services if provided as part of a development plan. Revenue related to these payments is recorded on a net basis; in this instance, the Company acts as an agent, as it does not have discretion to change suppliers and does not perform any part of the services or manufacture of the subject pharmaceutical ingredients.  The costs associated with these activities are netted against the related revenue in the condensed consolidated statements of operations.

10


In 2012, EA Pharma made an upfront cash payment to the Company of €10.0 million under the Agreement. The parties amended the agreement in April 2016, pursuant to which EA Pharma made an additional cash payment to the Company of $8.0 million.  As of June 30, 2018, the Company is eligible to receive an additional regulatory-based milestone payment under the Agreement of €4.3 million ($5.0 million based on the Euro to USD exchange rate as of June 30, 2018) if a specified regulatory event is achieved for elobixibat.  In December 2017, the Company executed a royalty interest acquisition agreement (RIAA) with HealthCare Royalty Partners III, L.P. (HCR) relating to certain royalties and sales milestone payments due under the Agreement and which is detailed below. The cash payments and any other payments for milestones and royalties from EA Pharma are non-refundable, non-creditable and not subject to set-off.

The Agreement will continue until the last royalty period for any product in the territory, which is defined as the period when there are no remaining patent rights or regulatory exclusivity in place for any products subject to royalties. EA Pharma may terminate the Agreement at will upon 180 days’ prior written notice to the Company. Either party may terminate the Agreement for the other party’s uncured material breach or insolvency and in certain other circumstances agreed to by the parties.

The Company assessed this arrangement in accordance with Accounting Standards Codification (ASC) Topic 606, Revenue from Contracts with Customers (ASC 606), and concluded that the contract counterparty, EA Pharma, is a customer. The Company identified the following material promises under the arrangement: (1) a sub-licensable and exclusive license to use the Company’s intellectual property and collaboration compounds to conduct development and commercialization activities in the designated fields and territories and (2) the technology transfer of the Albireo intellectual property and compound. Participation on the joint development committee (“JDC”) and joint commercialization committee (“JCC”) was determined to be quantitatively and qualitatively immaterial and therefore is excluded from performance obligations. The license was determined to not be distinct from the technology transfer; as such, the Company determined that these promises should be combined into a single performance obligation.

Under the Agreement, in order to evaluate the appropriate transaction price, the Company determined that the upfront amount constituted the entirety of the consideration to be included in the transaction price as of the outset of the arrangement, which was allocated to the single performance obligation. At the outset of the arrangement, the transaction price included only the €10.0 million upfront consideration received, and was increased to include the $8.0 million received in conjunction with the 2016 amendment. The potential milestone payments were excluded from the transaction price, as all milestone amounts were fully constrained. In April 2013, December 2015, and October 2016, various development milestone events were achieved, and the Company recognized revenue related to these events; because the Company previously satisfied its performance obligation to deliver the license, the Company recorded these milestone payments as received. The Company will reevaluate the transaction price at the end of each reporting period and as other uncertain events are resolved or other changes in circumstances occur, and, if necessary, adjust its estimate of the transaction price.

 

In January 2018, the Japanese Ministry of Health Labour and Welfare (MHLW) approved a new drug application filed by EA Pharma for elobixibat for the treatment of chronic constipation, for which the Company received a milestone payment of $11.2 million. Based on the regulatory approval, the Company determined that the milestone was no longer at risk of significant reversal. As such, because the single performance obligation had previously been satisfied, the Company recognized this amount in full in the first quarter of 2018 and there was no deferred revenue or contract asset as of June 30, 2018.

   

11


Monetization of Future Royalties

In December 2017, the Company executed the RIAA with HCR pursuant to which it sold to HCR the right to receive all royalties from sales in Japan and sales milestones achieved from any covered territory potentially payable to the Company under the Agreement, up to a specified maximum “cap” amount of $78.8 million, based on the funds the Company received from HCR to date.  The Company received $44.5 million from HCR, net of certain transaction expenses, under the RIAA and the Company is eligible to receive an additional $15.0 million under the RIAA if a specified sales milestone is achieved for elobixibat in Japan. If the cap amount is reached, the Company will again become eligible to receive royalties from Japanese sales and sales milestones from covered territories for elobixibat from EA Pharma under the Agreement.  The Company is obligated to make royalty interest payments to HCR under the RIAA only to the extent it receives future Japanese royalties, sales milestones or other specified payments from EA Pharma.  Although the Company sold its rights to receive royalties from the sales of elobixibat in Japan, as a result of its ongoing involvement in the cash flows related to these royalties, the Company will continue to account for these royalties as revenue.  The Company recorded the $44.5 million as a liability related to sale of future royalties (royalty obligation) in the balance sheet at June 30, 2018.  The royalty obligation will be amortized using the effective interest rate method, based on the Company's best estimate of the time it will take to reach the capped amount. The following table shows the activity within the liability account during the period from the inception of the royalty transaction in December 2017 to June 30, 2018:

 

 

 

June 30, 2018

 

 

 

(in thousands)

 

Liability related to sale of future royalties—beginning balance

 

$

 

Proceeds from sale of future royalties, net

 

 

44,525

 

Unrealized foreign currency (gain)/loss on remeasurement of the liability

 

 

2,440

 

Foreign currency translation (gain)/loss

 

 

(2,418

)

Accretion of interest expense on liability related to royalty monetzation

 

 

2,919

 

Liability related to sale of future royalties—ending balance

 

$

47,466

 

Less current portion classified within other current liabilities

 

 

(730

)

Net ending Liability related to sale of future royalties

 

$

46,736

 

 

The Company records estimated royalties to accrued other until the payment is received from EA Pharma at which time the Company then remits payment to HCR.  As royalties are remitted to HCR, the balance of the royalty obligation will be effectively repaid over the life of the RIAA. In order to determine the amortization of the royalty obligation, the Company is required to estimate the total amount of future royalty payments to be received and submitted to HCR, as noted above, based on the Company's best estimate of the time it will take to reach the cap amount. The sum of these amounts less the $44.5 million proceeds the Company received will be recorded as interest expense over the life of the royalty obligation. Since inception, the Company's estimate of its total interest expense resulted in a quarterly effective interest rate of approximately 4.15%. The Company periodically assesses the estimated royalty payments to HCR and to the extent such payments are greater or less than its initial estimates or the timing of such payments is materially different than its original estimates, the Company will prospectively adjust the accretion of interest on the royalty obligation. There are a number of factors that could materially affect the amount and the timing of royalty payments, most of which are not within the Company's control. Such factors include, but are not limited to, the rate of elobixibat prescriptions, the number of doses administered, the introduction of competing products, manufacturing or other delays, patent protection, adverse events that result in governmental health authority imposed restrictions on the use of the drug products, significant changes in foreign exchange rates as the royalties remitted to HCR are in U.S. dollars while sales of elobixibat are in Japanese yen, and sales never achieving forecasted numbers, which would result in reduced royalty payments, reduced non-cash royalty revenues and reduced non-cash interest expense over the life of the royalty obligation.

Loss contingencies

Loss contingencies are recorded as liabilities when it is probable that a liability has occurred and the amount of loss is reasonably estimable. Disclosure is required when there is a reasonable possibility that an ultimate loss will be material. Contingent liabilities are often resolved over long periods of time. Estimating probable losses requires analysis that often depends on judgments about potential actions by third parties, such as regulators.

Recently adopted accounting pronouncements

 

Effective January 1, 2018, the Company adopted ASC Topic 606, Revenue from Contracts with Customers, using the modified retrospective transition method. Under this method, results for reporting periods beginning after January 1, 2018 are presented under ASC 606, while prior period amounts are not adjusted and continue to be reported in accordance with ASC 605. This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements and financial instruments. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those

12


goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of ASC 606, the entity performs the following five step analysis: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step analysis to contracts when it is probable that the entity will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract, determines those that are performance obligations, and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.

 

The Company enters into licensing agreements which are within the scope of ASC 606, under which it may exclusively license rights to research, develop, manufacture and commercialize its product candidates to third parties. The terms of these arrangements may include payment to the Company of one or more of the following: non-refundable, upfront license fees; reimbursement of certain costs; development, regulatory and commercial milestone payments; and royalties on net sales of licensed products.

In determining the appropriate amount of revenue to be recognized as the Company fulfills its obligations under each of its agreements, the Company performs the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations, including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. As part of the accounting for these arrangements, the Company must use significant judgment to determine: (a) the number of performance obligations based on the determination under step (ii) above and (b) the transaction price under step (iii) above. The Company uses judgment to determine whether milestones or other variable consideration, except for royalties, should be included in the transaction price as described further below. The transaction price is allocated to each performance obligation on a relative stand-alone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied.

Amounts received prior to revenue recognition are recorded as deferred revenue. Amounts expected to be recognized as revenue within the 12 months following the balance sheet date are classified as current portion of deferred revenue in the accompanying consolidated balance sheets. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred revenue, net of current portion.

 

Exclusive Licenses

 

If the license to the Company’s intellectual property is determined to be distinct from the other promises or performance obligations identified in the arrangement, the Company recognizes revenue from non-refundable, upfront fees allocated to the license when the license is transferred to the customer and the customer is able to use and benefit from the license. In assessing whether a promise or performance obligation is distinct from the other promises, the Company considers factors such as the research, development, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. In addition, the Company considers whether the collaboration partner can benefit from a promise for its intended purpose without the receipt of the remaining promise, whether the value of the promise is dependent on the unsatisfied promise, whether there are other vendors that could provide the remaining promise, and whether it is separately identifiable from the remaining promise. For licenses that are combined with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. The measure of progress, and thereby periods over which revenue should be recognized, are subject to estimates by management and may change over the course of the research and development and licensing agreement. Such a change could have a material impact on the amount of revenue the Company records in future periods.

 

Milestone Payments

 

At the inception of each arrangement that includes development milestone payments, the Company evaluates whether the milestones are considered probable of being achieved and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. The Company evaluates factors such as the scientific, clinical, regulatory, commercial, and other risks that must be overcome to achieve the particular milestone in making this assessment. There is considerable judgment involved in determining whether it is probable that a significant revenue reversal would not occur. At the end of each subsequent reporting period, the Company reevaluates the probability of achievement of all

13


milestones subject to constraint and, if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment.

 

Royalties

 

For arrangements that include sales-based royalties, including milestone payments based on a level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied).

 

Impact of Adoption

 

The most significant change relates to the Company’s accounting for contingent milestone payments. Under ASC 605, the Company recognized revenue related to contingent milestone payments as the milestone was achieved, using the milestone method. Under ASC 606, the Company performs an assessment of the probability of milestone achievement at each reporting date, and determines whether the cumulative revenue related to the milestone is at risk of significant reversal.

As a result of adopting ASC 606 on January 1, 2018, the Company did not record any cumulative changes in the current period, as the performance obligation related to the Agreement with EA Pharma was fully satisfied in 2012.  Additionally, there was no difference in the revenue recognized or costs recorded in the six months ended June 30, 2018 as what would have been recognized or recorded under ASC 605.

In September 2016, the FASB issued ASU 2016-15, “Statement of Cash Flows (Topic 230): Classification of Certain Cash Receipts and Cash Payments (a consensus of the Emerging Issues Task Force),” which changes how certain cash receipts and cash payments are presented and classified in the statement of cash flows. The new standard is effective for fiscal years beginning after December 15, 2017 and for interim periods therein.  The Company has adopted this standard as of January 1, 2018 and determined there is no impact of this standard on the Company’s condensed consolidated financial statements as of the date of the adoption.

In May 2017, the FASB issued ASU 2017-09, “Compensation – Stock Compensation (Topic 718), Scope of Modification Accounting,” (ASC 718), which amends the scope of modification accounting for share-based payment arrangements and provides guidance on the types of changes to the terms or conditions of share-based payment awards to which an entity would be required to apply modification accounting under ASC 718.  The new standard is effective for fiscal years beginning after December 15, 2017 and for interim periods therein.  The Company has adopted this standard as of January 1, 2018 and determined there is minimal impact on the Company’s condensed consolidated financial statements.

Accounting pronouncements issued but not yet adopted

In February 2016, the FASB issued ASU 2016-02, “Leases (Topic 842).” The new standard establishes a right-of-use (ROU) model that requires a lessee to record a ROU asset and a lease liability on the balance sheet for all leases with terms longer than 12 months. Leases will be classified as either finance or operating, with classification affecting the pattern of expense recognition in the income statement. The new standard is effective for fiscal years beginning after December 15, 2018, including interim periods within those fiscal years. A modified retrospective transition approach is required for lessees for capital and operating leases existing at, or entered into after, the beginning of the earliest comparative period presented in the financial statements. The Company is currently evaluating the impact this ASU will have on its consolidated financial statements and currently does not plan to early adopt this standard.

 

2.  Commitments and contingencies

Operating lease commitments    

As of June 30, 2018, future minimum commitments under facility operating leases were $1,088,000.

Rent expense recognized under the Company’s operating leases was $204,000 and $185,000 for the six months ended June 30, 2018 and 2017, respectively.

Agreements with CROs

As of June 30, 2018, the Company had various agreements with CROs for the conduct of specified research and development activities.  Based on the terms of the respective agreements, the Company may be required to make future payments of up to $17.6 million to CROs upon the completion of contracted work.

14


Other Commitments

In connection with the spin-off of Albireo Limited from AstraZeneca in 2008 and associated transfer agreements, the Company became party to an assignment agreement between AstraZeneca and a named inventor on a patent related to elobixibat. In connection with this agreement, in April 2018, the Company was required to pay a one-time “launch fee” of $457,000.      

 

3.  Net loss per share

Basic net loss per share, or Basic EPS, is calculated by dividing the net loss by the weighted average number of shares of common stock outstanding. Diluted net loss per share, or Diluted EPS, is calculated by dividing the net loss by the weighted-average number of shares of common stock plus dilutive common stock equivalents outstanding.

The following table sets forth the computation of Basic EPS and Diluted EPS (in thousands, except for share and per share data): 

 

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

 

 

2018

 

 

2017

 

 

2018

 

 

2017

 

Basic and Diluted EPS:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Numerator

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Net loss

 

$

(14,603

)

 

$

(6,176

)

 

$

(16,222

)

 

$

(12,847

)

Denominator

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Weighted average number of shares

 

 

11,938,357

 

 

 

7,171,610

 

 

 

11,417,463

 

 

 

6,734,555

 

Basic and Diluted EPS

 

$

(1.22

)

 

$

(0.86

)

 

$

(1.42

)

 

$

(1.91

)

 

The following outstanding common stock equivalents were excluded from the computation of Diluted EPS for the three and six months ended June 30, 2018 and 2017 because including them would have been anti-dilutive:

 

 

 

For the Three Months Ended June 30,

 

For the Six Months Ended June 30,

 

 

 

2018

 

 

2017

 

2018

 

 

2017

 

Options to purchase common stock

 

 

1,393,297

 

 

 

767,662

 

 

1,393,297

 

 

 

767,662

 

 

4.  Income taxes

The Company did not record a tax provision or benefit for the three months or six months ended June 30, 2018 or 2017.  The Company has continued to maintain a full valuation allowance against its net deferred tax assets.  The Company has had an overall net operating loss position since its inception.      

On December 22, 2017, the SEC staff issued Staff Accounting Bulletin No. 118 (SAB 118), which provides guidance on accounting for the tax effects of the Tax Cuts and Jobs Act (TCJA). SAB 118 provides a measurement period that should not extend beyond one year from the TCJA enactment date for companies to complete the accounting under ASC Topic 740, Income Taxes. Although no changes were made to provisional amounts during the three or six months ended June 30, 2018, the Company will continue to refine its estimates related to the new legislation as clarifying guidance and interpretations are issued and the Company’s 2017 tax returns are completed.

15


5.  Stock-based Compensation

The Company recognized stock-based compensation expense for employees of $1.1 million and $1.4 million for the three months ended June 30, 2018 and 2017, respectively, and $2.2 million and $2.1 million for the six months ended June 30, 2018 and 2017, respectively.   

A summary of the outstanding stock options as of June 30, 2018 is as follows:

 

 

 

Stock Options Outstanding

 

 

 

Number of

Shares

 

 

Weighted

Average

Exercise

Price Per

Share

 

 

Weighted

Average

Remaining

Contractual

Term (Years)

 

 

Aggregate

Intrinsic

Value (in

thousands)

 

Outstanding—December 31, 2017

 

 

1,035,361

 

 

$

17.78

 

 

 

8.71

 

 

$

11,896

 

Granted

 

 

486,050

 

 

$

31.12

 

 

 

 

 

$

 

Expirations/forfeitures

 

 

(42,491

)

 

$

41.93

 

 

 

 

 

$

 

Exercises

 

 

(60,345

)

 

$

4.23

 

 

 

 

 

$

 

Outstanding—June 30, 2018

 

 

1,418,575

 

 

$

21.94

 

 

 

8.47

 

 

$

20,587

 

Exercisable—June 30, 2018

 

 

444,425

 

 

$

15.72

 

 

 

7.03

 

 

$

10,142

 

Vested or expected to vest at—June 30, 2018

 

 

1,399,153

 

 

$

22.23

 

 

 

8.48

 

 

$

19,917

 

 

Aggregate intrinsic value represents the difference between the estimated fair value of the underlying common stock and the exercise price of outstanding, in-the-money options.

Options to purchase 19,422 shares of common stock are performance based and vest upon the date the Company files a drug approval application for its product candidate A4250 for any orphan indication, if such filing occurs prior to a specified date. This unvested performance-based option is excluded from the vested or expected to vest balance as of June 30, 2018.

As of June 30, 2018, the total unrecognized compensation expense related to unvested options was $17.1 million, which the Company expects to recognize over a weighted average vesting period of 2.6 years.

In determining the estimated fair value of the stock-based awards, the Company uses the Black-Scholes option pricing model and assumptions discussed below. Each of these inputs is subjective and generally requires significant judgment to determine.

The fair value of stock option awards granted during the three and six months ended June 30, 2018 was estimated with the following assumptions: 

 

 

 

Three Months

Ended June 30,

2018

 

Six Months

Ended June 30,

2018

 

Price per share of common stock

 

$30.68-$32.53

 

30.68-32.57

 

Expected term (in years)

 

5.5-6.0

 

5.3-6.0

 

Risk-free interest rate

 

2.8

 

2.6-2.8

 

Expected volatility

 

84.7-86.9

 

84.7-86.9

 

Dividend rate

 

0%

 

0%

 

 

6.  Financings

At-the-Market Sales

In October 2017, the Company entered into an at-the-market offering program Sales Agreement with Cowen and Company, LLC (Cowen) relating to the sale of shares of the Company’s common stock having an aggregate offering price of up to $50.0 million from time to time through Cowen, acting as its agent. In February 2018, the Company sold an aggregate of 728,862 shares of common stock pursuant to the Sales Agreement and received proceeds, net of offering expenses, of approximately $24.2 million.

January 2018 Underwritten Public Offering

On January 29, 2018, the Company completed an underwritten public offering of 2,265,500 shares of its common stock, at a price to public of $33.00 per share.  The Company received net proceeds from this offering of $70.0 million, after deducting underwriting discounts, commission and offering expenses.

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

You should read the following discussion and analysis of our financial condition and results of operations together with our condensed consolidated financial statements and the related notes included elsewhere in this quarterly report and our audited financial statements and Management’s Discussion and Analysis of Financial Condition and Results of Operations included in our Annual Report on Form 10-K for the year ended December 31, 2017, filed with the SEC. In addition to historical information, the following discussion contains forward-looking statements that involve risks, uncertainties and assumptions. Our actual results, performance or experience could differ materially from what is indicated by any forward-looking statement due to various important factors, risks and uncertainties, including, but not limited to, those set forth under “Cautionary Note Regarding Forward-Looking Statements” included elsewhere in this quarterly report or under “Risk Factors” in Item 1A of Part I of our Annual Report on Form 10-K for the year ended December 31, 2017 in item 1A of Part II of this quarterly report or in other filings that we make with the SEC.

Overview

We are a biopharmaceutical company focused on the development and commercialization of novel bile acid modulators to treat orphan pediatric liver diseases and other liver or gastrointestinal diseases and disorders.  The initial target indication for our lead product candidate, A4250, is progressive familial intrahepatic cholestasis, or PFIC, a rare, life-threatening genetic disorder affecting young children for which there is currently no approved drug treatment. We completed a Phase 2 clinical trial of A4250 in children with chronic cholestasis and pruritus and enrolled the first patient in our Phase 3 clinical trial of A4250 in patients with PFIC in May of 2018.  In June of 2018, the FDA granted a rare pediatric disease designation to A4250, which affirms our eligibility to apply for a rare pediatric disease priority review voucher upon submission of a new drug application for A4250.  In addition to PFIC, we plan to consider conducting future clinical development of A4250 as a treatment for one or more other pediatric cholestatic liver diseases and disorders. Our most advanced product candidates in addition to A4250 include elobixibat, which is approved in Japan for the treatment of chronic constipation and which for we anticipate conducting a Phase 2 clinical trial of as a treatment for nonalcoholic steatohepatitis, or NASH, and A3384, which is a product candidate to treat bile acid malabsorption.  In June 2018, the Company was granted a patent for a method of using elobixibat to treat NASH in both the US and Europe. We also have a preclinical program in NASH.

The precise prevalence of PFIC is unknown, and we are not aware of any patient registries or other method of establishing with precision the actual number of patients with PFIC in any geography.  PFIC has been estimated to affect between one in every 50,000 to 100,000 children born worldwide. Benign recurrent familial intrahepatic cholestasis, or BRIC, is a disease that is caused by the same genetic defect, and patients who manifest the same symptoms as PFIC but their symptomatology tends to be episodic in nature. BRIC has also been estimated to affect between one in every 50,000 to 100,000 children born worldwide. Based on the published incidence, published regional populations, and estimated median life expectancies, we estimate the prevalence of PFIC together with BRIC to be approximately 8,000 to 10,000 patients in the U.S. and E.U. but we are not able to estimate the prevalence of PFIC with precision.  There are currently no drugs approved for the treatment of PFIC. First-line treatment for PFIC is typically off-label ursodeoxycholic acid, or UDCA, which is approved in the U.S. and elsewhere for the treatment of primary biliary cholangitis, or PBC. However, many PFIC patients do not respond well to UDCA, undergo partial external bile diversion, or PEBD, surgery and often require liver transplantation. PEBD surgery is a life-altering and undesirable procedure in which bile is drained outside the body to a stoma bag that must be worn by the patient 24 hours a day.

We intend to consider conducting future clinical development of A4250 as a treatment for other pediatric cholestatic liver diseases and disorders in addition to PFIC.  These indications may include any or all of Alagille syndrome or ALGS, biliary atresia and primary sclerosing cholangitis.

ALGS is a genetic condition associated with liver, heart, eye and skeletal abnormalities. In particular, ALGS patients have fewer than normal bile ducts inside the liver, which leads to cholestasis and the accumulation of bile and causes scarring in the liver. We estimate the incidence of ALGS to be one in 55,000 newborns and the prevalence to be approximately 2,100 patients in the U.S. and 1,600 patients in the E.U. There are currently no drugs approved for the treatment of ALGS. Current treatment for ALGS is generally in line with current treatments for PFIC as described above.

Biliary atresia is a partial or total blocking or absence of large bile ducts that causes cholestasis and resulting accumulation of bile that damages the liver. The estimated worldwide incidence of biliary atresia is 7.3 in every 100,000 births in the U.S. and one in every 18,000 births in the E.U and the prevalence is estimated to be approximately 9,600 patients in the U.S. and 6,600 patients in the E.U.. There are currently no drugs approved for the treatment of biliary atresia. The current standard of care is a surgery known as the Kasai procedure, or hepatoportoenterostomy, in which the obstructed bile ducts are removed and a section of the small intestine is connected to the liver directly. However, only an estimated 25% of those initially undergoing the Kasai procedure will survive to their twenties without need for liver transplantation.

17


Primary sclerosing cholangitis refers to swelling (inflammation), scarring, and destruction of bile ducts inside and outside of the liver. The first symptoms are typically fatigue, itching and jaundice, and many patients with sclerosing cholangitis also suffer from inflammatory bowel disease.  The estimated incidence of sclerosing cholangitis is 6.3 cases per 100,000 people.  There are currently no drugs approved for the treatment of sclerosing cholangitis. First-line treatment is typically off-label UDCA, although UDCA has not been established to be safe and effective in patients with sclerosing cholangitis in well controlled clinical trials.

 

In June 2018, our shareholders elected Anne Klibanski, M.D., and Stephanie Okey, M.S., to our Board of Directors, and we added Jason G. Duncan, J.D., as General Counsel and Secretary.

 

In July 2018, we added Patrick Horn, M.D. who succeeded Paresh Soni, M.D. as our Chief Medical Officer.  Dr. Soni has agreed to serve as an active consultant to us, and during such time as he remains a consultant will continue to be covered as a “Participant” under our 2018 Equity Incentive Plan.

Since inception, we have incurred significant operating losses. As of June 30, 2018, we had an accumulated deficit of $66.6 million.  We expect to continue to incur significant expenses and increasing operating losses as we continue our development of, and seek marketing approvals for, our product candidates, prepare for and begin the commercialization of any approved products, and add infrastructure and personnel to support our product development efforts and operations as a public company in the United States.

As a clinical-stage company, our revenues, expenses and results of operations are likely to fluctuate significantly from quarter to quarter and year to year. We believe that period-to-period comparisons of our results of operations should not be relied upon as indicative of our future performance.

As of June 30, 2018, we had approximately $183.2 million in cash and cash equivalents.

Financial Operations Overview

The following discussion sets forth certain components of our consolidated statements of operations as well as factors that impact those items.

Revenue

We generate revenue primarily from the receipt of royalty revenue, upfront or license fees, milestone payments and payment for pharmaceutical ingredient or related procurement services that are made pursuant to license agreements or related supply agreements. License agreements with commercial partners generally include nonrefundable upfront fees and milestone payments, the receipt of which is dependent upon the achievement of specified development, regulatory or commercial milestone events, as well as royalties on product sales of licensed products, if and when such product sales occur, and payments for pharmaceutical ingredient or related procurement services. For these agreements, management applies judgment in the allocation of total agreement consideration to the performance obligations on a reliable basis that reasonably reflects the selling prices that might be expected to be achieved in stand-alone transactions.  For additional information about our revenue recognition, refer to Note 1 to our condensed consolidated financial statements included in this quarterly report.

Operating Expenses

Research and Development Expenses

Research and development expenses consist primarily of personnel costs (including salaries, benefits and stock-based compensation) for employees in research and development functions, costs associated with nonclinical and clinical development services, including clinical trials and related manufacturing costs, third-party contract research organizations, or CROs, and related services and other outside costs, including fees for third-party professional services such as consultants. Our nonclinical studies and clinical studies are performed by CROs. We expect to continue to focus our research and development efforts on nonclinical studies and clinical trials of our product candidates. As a result, we expect our research and development expenses to continue to increase for the foreseeable future.

Our direct research and development expenses are tracked on a program-by-program basis and consist primarily of external costs such as fees paid to CROs and others in connection with our nonclinical and clinical development activities and related manufacturing. We do not allocate employee costs or facility expenses, including depreciation or other indirect costs, to specific product development programs because these costs are deployed across multiple product development programs and, as such, are not separately classified.

18


Successful development of our current and potential future product candidates is highly uncertain. Completion dates and costs for our programs can vary significantly by product candidate and are difficult to predict. As a result, we cannot estimate with any degree of certainty the costs we will incur in connection with development of any of our product candidates. We anticipate we will make determinations as to which programs and product candidates to pursue and how much funding to direct to each program and product candidate on an ongoing basis in response to the results of ongoing and future clinical trials, our ability to enter into licensing, collaboration and similar arrangements with respect to current or potential future product candidates, the success of research and development programs and our assessments of commercial potential.

General and Administrative Expenses

General and administrative expenses consist primarily of personnel costs (including salaries, benefits and stock-based compensation) for our executive, finance and other administrative employees. In addition, general and administrative expenses include fees for third-party professional services, including consulting, information technology, legal and accounting services and other corporate expenses and allocated overhead.

Critical Accounting Policies and Estimates

Our management’s discussion and analysis of financial condition and results of operations is based on our unaudited condensed consolidated financial statements, which have been prepared in accordance with United States generally accepted accounting principles for interim financial information. The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses. We base our estimates and assumptions on historical experience and on various assumptions that we believe are reasonable under the circumstances, and we evaluate them on an ongoing basis. These estimates and assumptions form the basis for making judgments about the carrying values of assets and liabilities and the recording of revenues and expenses that are not readily apparent from other sources. Actual results and experiences may differ materially from these estimates and judgments. In addition, our reported financial condition and results of operations could vary if new accounting standards are enacted that are applicable to our business.

Our significant accounting policies are described in Note 1 to our audited consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2017 and in Note 1 to our condensed consolidated financial statements included in this quarterly report.  We believe that our accounting policies relating to revenue recognition and the adoption of ASC 606 (see Note 1 to our condensed consolidated financial statements included in this quarterly report), royalty monetization and related estimates (see Note 1 to our condensed consolidated financial statements included in this quarterly report), research and development expenses and stock-based compensation are the most critical to understanding and evaluating our reported financial results. We have identified these policies as critical because they are both important to the presentation of our financial condition and results of operations and require us to make judgments and estimates on matters that are inherently uncertain and may change in future periods. These policies are described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations — Critical Accounting Policies and Estimates” in our Annual Report on Form 10-K for the year ended December 31, 2017.  

Results of Operations

Three Months Ended June 30, 2018 and June 30, 2017

Revenue

 

 

 

Three Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Revenue

 

$

730

 

 

$

1

 

 

$

729

 

 

There was $730,000 in revenue for the three months ended June 30, 2018 compared with $1,000 for the three months ended June 30, 2017, an increase of $729,000.  The higher revenue is due to the estimated royalty revenue from EA Pharma for elobixibat for the 2018 period.

 

Research and development expenses

 

 

 

Three Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Research and development expenses

 

$

6,411

 

 

$

2,962

 

 

$

3,449

 

 

19


Research and development expenses were $6.4 million for the three months ended June 30, 2018 compared with $3.0 million for the three months ended June 30, 2017, an increase of $3.4 million.  The higher research and development expenses for the 2018 period were principally due to an increase of $1.6 million in costs associated with development of A4250, including costs incurred for manufacturing and clinical development activities for our Phase 3 clinical trial in patients with PFIC, and an increase of $865,000 in personnel and related expenses as we continue to increase our headcount.

 

The following table summarizes our principal product development programs and the out-of-pocket third-party expenses incurred with respect to each clinical-stage product candidate and our preclinical programs for the three months ended June 30, 2018 and 2017.

 

 

 

Three Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Direct third-party project costs:

 

 

 

 

 

 

 

 

 

 

 

 

A4250

 

$

3,461

 

 

$

1,872

 

 

$

1,589

 

Elobixibat

 

 

72

 

 

 

(7

)

 

 

79

 

A3384

 

 

205

 

 

 

62

 

 

 

143

 

Preclinical

 

 

462

 

 

 

61

 

 

 

401

 

Total

 

$

4,200

 

 

$

1,988

 

 

$

2,212

 

Other project costs(1):

 

 

 

 

 

 

 

 

 

 

 

 

Personnel costs

 

$

1,347

 

 

$

482

 

 

$

865

 

Other costs(2)

 

 

864

 

 

 

492

 

 

 

372

 

Total

 

$

2,211

 

 

$

974

 

 

$

1,237

 

Total research and development costs

 

$

6,411

 

 

$

2,962

 

 

$

3,449

 

 

(1)

Other project costs are leveraged across multiple programs.

(2)

Other costs include facility, supply, consultant and overhead costs that support multiple programs.

General and administrative expenses

 

 

 

Three Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

General and administrative expenses

 

$

4,238

 

 

$

3,713

 

 

$

525

 

 

General and administrative expenses were $4.2 million for the three months ended June 30, 2018 compared with $3.7 million for the three months ended June 30, 2017, an increase of $525,000.  The higher general and administrative expenses for the 2018 period were principally due to increases in recruiting costs and increases in professional fees.  

Other operating (income) expense, net

 

 

 

Three Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Other operating (income) expense, net

 

$

487

 

 

$

(65

)

 

$

552

 

 

Other operating (income) expense, net totaled $487,000 of expense for the three months ended June 30, 2018 compared with $65,000 of other operating income for the three months ended June 30, 2017, a difference of $552,000.  The difference resulted from differences in currency exchange rates in the two periods.

Interest income (expense), net

 

 

 

Three Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Interest income (expense), net

 

$

(1,666

)

 

$

(152

)

 

$

(1,514

)

 

20


Interest income (expense), net totaled $1.7 million of expense for the three months ended June 30, 2018 compared with $152,000 of expense for the three months ended June 30, 2017, a difference of $1.5 million.  The difference was principally attributable to $1.9 million in non-cash interest expense recorded in connection with the sale of future royalties, partially offset by interest income for the 2018 period, compared to interest expense related to our prior loan facility for the 2017 period.

Other income (expense), net

 

 

 

Three Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Other income (expense), net

 

$

(2,531

)

 

$

585

 

 

$

(3,116

)

 

Net other expense was $2.5 million for the three months ended June 30, 2018 compared with net other income of $585,000 for the three months ended June 30, 2017, a difference of $3.1 million.  The difference is primarily related to the foreign currency expense associated with our royalty monetization offset by the exercise of warrants by our lender in May 2017.

 

Six Months Ended June 30, 2018 and June 30, 2017

Revenue

 

 

 

Six Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Revenue

 

$

11,932

 

 

$

2

 

 

$

11,930

 

 

Revenue was $11.9 million for the six months ended June 30, 2018 compared with $2,000 for the six months ended June 30, 2017, an increase of $11.9 million.  The higher revenue is due to a milestone payment received from EA Pharma due to the approval by the Japanese MHLW of the new drug application for elobixibat for the treatment of chronic constipation and the estimated royalty revenue from EA Pharma for elobixibat for the period.

Research and development expenses

 

 

 

Six Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Research and development expenses

 

$

12,562

 

 

$

5,774

 

 

$

6,788

 

 

Research and development expenses were $12.6 million for the six months ended June 30, 2018 compared with $5.8 million for the six months ended June 30, 2017, an increase of $6.8 million.  The higher research and development expenses for the 2018 period were principally due to an increase of $3.4 million in costs associated with development of A4250, including costs incurred for manufacturing and clinical development activities for our Phase 3 clinical trial in patients with PFIC, and an increase of $1.8 million in personnel and related expenses as we continue to increase our headcount.

21


The following table summarizes our principal product development programs and the out-of-pocket third-party expenses incurred with respect to each clinical-stage product candidate and our preclinical programs for the six months ended June 30, 2018 and 2017.

 

 

 

Six Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Direct third-party project costs:

 

 

 

 

 

 

 

 

 

 

 

 

A4250

 

$

6,851

 

 

$

3,485

 

 

$

3,366

 

Elobixibat

 

 

95

 

 

 

(4)

 

 

 

99

 

A3384

 

 

333

 

 

 

108

 

 

 

225

 

Preclinical

 

 

800

 

 

 

84

 

 

 

716

 

Total

 

$

8,079

 

 

$

3,673

 

 

$

4,406

 

Other project costs(1):

 

 

 

 

 

 

 

 

 

 

 

 

Personnel costs

 

$

2,757

 

 

$

944

 

 

$

1,813

 

Other costs(2)

 

 

1,726

 

 

 

1,157

 

 

 

569

 

Total

 

$

4,483

 

 

$

2,101

 

 

$

2,382

 

Total research and development costs

 

$

12,562

 

 

$

5,774

 

 

$

6,788

 

 

(1)

Other project costs are leveraged across multiple programs.

(2)

Other costs include facility, supply, patent, consultant and overhead costs that support multiple programs.

General and administrative expenses

 

 

 

Six Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

General and administrative expenses

 

$

8,366

 

 

$

6,925

 

 

$

1,441

 

 

General and administrative expenses were $8.4 million for the six months ended June 30, 2018 compared with $6.9 million for the six months ended June 30, 2017, an increase of $1.4 million.  The higher general and administrative expenses for the 2018 period were principally attributable to increases of $840,000 in professional fees, $319,000 in recruiting costs, and $195,000 in salary and benefits resulting from new hires.

Other operating (income) expense, net

 

 

 

Six Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Other operating (income) expense, net

 

$

1,991

 

 

$

9

 

 

$

1,982

 

 

Other operating (income) expense, net totaled $2.0 million of expense for the six months ended June 30, 2018 compared with $9,000 of expense for the six months ended June 30, 2017, a difference of $2.0 million.  The difference resulted from changes in currency exchange rates between the two periods.

Interest expense, net

 

 

 

Six Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Interest expense, net

 

$

(2,682

)

 

$

(401

)

 

$

2,281

 

 

Interest expense, net totaled $2.7 million for the six months ended June 30, 2018 compared with $401,000 for the six months ended June 30, 2017, an increase of $2.3 million. The difference was principally attributable to $2.9 million in non-cash interest expense recorded in connection with the sale of future royalties relating to sales of elobixibat in Japan, partially offset by interest income for the 2018 period, compared to interest expense related to our prior loan facility for the 2017 period. 

22


Other income (expense), net 

 

 

 

Six Months Ended June 30,

 

 

Change

 

 

 

2018

 

 

2017

 

 

$

 

 

 

(in thousands)

 

Other income (expense), net

 

$

(2,553)

 

 

$

260

 

 

$

(2,813

)

 

Other income (expense), net was $2.6 million of expense for the six months ended June 30, 2018 compared with $260,000 of income for the six months ended June 30, 2017, a change of $2.8 million.  The difference is primarily related to the foreign currency expense associated with our royalty monetization offset by the exercise of warrants by our lender in May 2017.

 

Liquidity and Capital Resources

Sources of Liquidity

We do not expect to generate significant revenue from product sales unless and until we or a potential future licensee or collaborator obtains marketing approval for, and commercializes, one or more of our current or potential future product candidates (other than elobixibat as a treatment for chronic constipation in Japan), which we do not expect to occur until at least 2021, if at all. We anticipate that we will continue to generate losses for the foreseeable future, and we expect the losses to increase as we continue the development of and seek regulatory approvals for our product candidates. We are subject to all of the risks applicable to the development of new pharmaceutical products and may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may harm our business. We expect that we will continue to incur additional costs associated with operating as a public company and anticipate that we will need substantial additional funding to complete development of and potentially commercialize our product candidates.

Our operations have historically been financed primarily through issuances of shares of common stock, preference shares or convertible debt, upfront fees paid upon entering into license agreements, payments received upon the achievement of specified milestone events under license agreements, grants and venture debt borrowings. Our primary uses of capital are, and we expect will continue to be, personnel-related costs, third party expenses associated with our research and development programs, including the conduct of clinical trials, and manufacturing-related costs for our product candidates.

As of June 30, 2018, our cash and cash equivalents were approximately $183.2 million.

During the first quarter of 2018, following the Japanese MHLW’s approval of elobixibat for the treatment of chronic constipation in January 2018, we received a $44.5 million pay