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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, DC 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, 2021

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 No. 001-36276

 

ULTRAGENYX PHARMACEUTICAL INC.

(Exact name of registrant as specified in its charter)

 

 

Delaware

 

 

27-2546083

(State or other jurisdiction of incorporation or organization)

 

 

(I.R.S. Employer Identification No.)

 

60 Leveroni Court
Novato, California

 

94949

(Address of principal executive offices)

 

(Zip Code)

(415) 483-8800

(Registrant’s telephone number, including area code)

Not Applicable

(Former Name, Former Address and Former Fiscal Year, if Changed Since Last Report)

 

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading Symbol

Name of each exchange on which registered

Common Stock, $0.001 par value

RARE

The Nasdaq Global Select Market

 

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 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      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

 

  

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 July 28, 2021, the registrant had 67,836,060 shares of common stock issued and outstanding.

  

 


 

ULTRAGENYX PHARMACEUTICAL INC.

FORM 10-Q FOR THE QUARTER ENDED JUNE 30, 2021

INDEX

 

 

 

 

 

 

  

Page

 

 

 

 

 

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

  

1

 

 

 

 

 

Part I –

 

Financial Information

  

 

 

 

 

 

 

 

 

 

 

Item 1.

 

Financial Statements

  

 

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Balance Sheets

  

2

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Operations

  

3

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Comprehensive Loss

  

4

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Stockholders’ Equity

 

5

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Cash Flows

  

7

 

 

 

 

 

 

 

 

 

 

 

Notes to Condensed Consolidated Financial Statements

  

8

 

 

 

 

 

 

 

 

 

Item 2.

 

Management’s Discussion and Analysis of Financial Condition and Results of Operations

  

21

 

 

 

 

 

 

 

 

 

Item 3.

 

Quantitative and Qualitative Disclosures About Market Risk

  

32

 

 

 

 

 

 

 

 

 

Item 4.

 

Controls and Procedures

  

32

 

 

 

 

 

Part II –

 

Other Information

  

 

 

 

 

 

 

 

 

 

 

Item 1.

 

Legal Proceedings

  

33

 

 

 

 

 

 

 

 

 

Item 1A.

 

Risk Factors

  

33

 

 

 

 

 

 

 

 

 

Item 2.

 

Unregistered Sales of Equity Securities and Use of Proceeds

  

67

 

 

 

 

 

 

 

 

 

Item 3.

 

Defaults Upon Senior Securities

  

68

 

 

 

 

 

 

 

 

 

Item 4.

 

Mine Safety Disclosures

  

68

 

 

 

 

 

 

 

 

 

Item 5.

 

Other Information

  

68

 

 

 

 

 

 

 

 

 

Item 6.

 

Exhibits

  

69

 

 

 

 

 

 

 

 

 

Signatures

 

 

  

70

 

 

 

 


 

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q (the Quarterly Report) contains forward-looking statements that involve risks and uncertainties. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report are forward-looking statements. In some cases, you can identify forward-looking statements by words such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will,” “would,” or the negative of these words, or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:

 

our commercialization, marketing, and manufacturing capabilities and strategy;

 

our expectations regarding the timing of clinical study commencements and reporting results from same;

 

the timing and likelihood of regulatory approvals for our product candidates;

 

the anticipated indications for our product candidates, if approved;

 

the potential market opportunities for commercializing our products and product candidates;

 

our expectations regarding the potential market size and the size of the patient populations for our products and product candidates, if approved for commercial use;

 

the impact of the COVID-19 pandemic and related health measures on our business, financial condition and liquidity;

 

estimates of our expenses, revenue, capital requirements, and our needs for additional financing;

 

our ability to develop, acquire, and advance product candidates into, and successfully complete, clinical studies;

 

the implementation of our business model and strategic plans for our business, products and product candidates and the integration and performance of any businesses we have acquired or may acquire;  

 

the initiation, timing, progress, and results of ongoing and future preclinical and clinical studies, and our research and development programs;

 

the scope of protection we are able to establish and maintain for intellectual property rights covering our products and product candidates;

 

our ability to maintain and establish collaborations or strategic relationships or obtain additional funding;

 

our ability to maintain and establish relationships with third parties, such as contract research organizations, contract manufacturing organizations, suppliers, and distributors;

 

our financial performance and the expansion of our organization;

 

our ability to obtain supply of our products and product candidates;

 

the scalability and commercial viability of our manufacturing methods and processes;

 

developments and projections relating to our competitors and our industry; and

 

other risks and uncertainties, including those listed under Part II, Item 1A. Risk Factors.

Any forward-looking statements in this Quarterly Report reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties, and other factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by these forward-looking statements. Factors that may cause actual results to differ materially from current expectations include, among other things, those discussed under Part II, Item 1A. Risk Factors and elsewhere in this Quarterly Report. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Except as required by law, we assume no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future.

This Quarterly Report also contains estimates, projections, and other information concerning our industry, our business, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research, or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market, and other data from reports, research surveys, studies, and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data, and similar sources.

1


PART I. FINANCIAL INFORMATION

Item 1. Financial Statements

 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(Unaudited)

(In thousands, except share amounts)

 

 

June 30,

 

 

December 31,

 

 

2021

 

 

2020

 

Assets

 

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

 

Cash and cash equivalents

$

290,875

 

 

$

713,526

 

Marketable debt securities

 

554,881

 

 

 

488,007

 

Accounts receivable, net

 

24,720

 

 

 

23,093

 

Inventory

 

15,083

 

 

 

13,048

 

Prepaid expenses and other current assets

 

63,933

 

 

 

57,630

 

Total current assets

 

949,492

 

 

 

1,295,304

 

Property, plant, and equipment, net

 

104,568

 

 

 

73,515

 

Equity investments

 

103,710

 

 

 

155,375

 

Marketable debt securities

 

128,031

 

 

 

10,506

 

Right-of-use assets

 

38,619

 

 

 

40,524

 

Intangible assets, net

 

130,950

 

 

 

131,113

 

Goodwill

 

44,406

 

 

 

44,406

 

Other assets

 

12,668

 

 

 

8,812

 

Total assets

$

1,512,444

 

 

$

1,759,555

 

 

 

 

 

 

 

 

 

Liabilities and Stockholders’ Equity

 

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

 

Accounts payable

$

21,790

 

 

$

12,923

 

Accrued liabilities

 

91,060

 

 

 

108,491

 

Contract liabilities

 

3,912

 

 

 

59,219

 

Lease liabilities

 

10,305

 

 

 

8,976

 

Total current liabilities

 

127,067

 

 

 

189,609

 

Contract liabilities

 

 

 

 

7,349

 

Lease liabilities

 

35,861

 

 

 

39,251

 

Deferred tax liabilities

 

33,306

 

 

 

33,306

 

Liability related to the sale of future royalties

 

344,039

 

 

 

335,665

 

Total liabilities

 

540,273

 

 

 

605,180

 

Stockholders’ equity:

 

 

 

 

 

 

 

Preferred stock — 25,000,000 shares authorized; nil outstanding as of June 30, 2021 and

   December 31, 2020

 

 

 

 

 

Common stock — 250,000,000 shares authorized; 67,764,950 and 66,818,520 shares issued

   and outstanding as of June 30, 2021 and December 31, 2020, respectively

 

68

 

 

 

67

 

Additional paid-in capital

 

2,850,016

 

 

 

2,773,195

 

Accumulated other comprehensive income

 

232

 

 

 

689

 

Accumulated deficit

 

(1,878,145

)

 

 

(1,619,576

)

Total stockholders’ equity

 

972,171

 

 

 

1,154,375

 

Total liabilities and stockholders’ equity

$

1,512,444

 

 

$

1,759,555

 

See accompanying notes.

 

 

 

2


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(Unaudited)

(In thousands, except share and per share amounts)

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Revenues:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Collaboration and license

$

63,940

 

 

$

50,161

 

 

$

142,950

 

 

$

77,376

 

Product sales

 

18,346

 

 

 

8,066

 

 

 

34,859

 

 

 

14,545

 

Non-cash collaboration royalty revenue

 

4,689

 

 

 

3,482

 

 

 

8,561

 

 

 

6,097

 

Total revenues

 

86,975

 

 

 

61,709

 

 

 

186,370

 

 

 

98,018

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Cost of sales

 

3,136

 

 

 

1,803

 

 

 

8,324

 

 

 

(1,700

)

Research and development

 

113,205

 

 

 

80,709

 

 

 

260,723

 

 

 

193,670

 

Selling, general and administrative

 

53,410

 

 

 

42,252

 

 

 

106,668

 

 

 

89,768

 

Total operating expenses

 

169,751

 

 

 

124,764

 

 

 

375,715

 

 

 

281,738

 

Loss from operations

 

(82,776

)

 

 

(63,055

)

 

 

(189,345

)

 

 

(183,720

)

Interest income

 

441

 

 

 

1,797

 

 

 

1,080

 

 

 

4,716

 

Change in fair value of equity investments

 

(31,046

)

 

 

95,200

 

 

 

(51,665

)

 

 

102,868

 

Non-cash interest expense on liability related to the sale of future

    royalties

 

(8,517

)

 

 

(8,429

)

 

 

(16,935

)

 

 

(16,511

)

Other income (expense)

 

(67

)

 

 

217

 

 

 

(862

)

 

 

(239

)

Income (loss) before income taxes

 

(121,965

)

 

 

25,730

 

 

 

(257,727

)

 

 

(92,886

)

Provision for income taxes

 

(463

)

 

 

(415

)

 

 

(842

)

 

 

(824

)

Net income (loss)

$

(122,428

)

 

$

25,315

 

 

$

(258,569

)

 

$

(93,710

)

Net income (loss) per share:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Basic

$

(1.81

)

 

$

0.42

 

 

$

(3.84

)

 

$

(1.59

)

Diluted

$

(1.81

)

 

$

0.41

 

 

$

(3.84

)

 

$

(1.59

)

Weighted-average shares used in computing net income (loss)

    per share:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Basic

 

67,607,752

 

 

 

59,995,617

 

 

 

67,356,443

 

 

 

58,996,278

 

Diluted

 

67,607,752

 

 

 

61,146,231

 

 

 

67,356,443

 

 

 

58,996,278

 

See accompanying notes.

 

 

 

3


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS)

(Unaudited)

(In thousands)

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Net income (loss)

$

(122,428

)

 

$

25,315

 

 

$

(258,569

)

 

$

(93,710

)

Other comprehensive income (loss):

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Foreign currency translation adjustments

 

103

 

 

 

89

 

 

 

(182

)

 

 

40

 

Unrealized gain (loss) on available-for-sale securities

 

73

 

 

 

2,740

 

 

 

(275

)

 

 

1,391

 

Other comprehensive income (loss):

 

176

 

 

 

2,829

 

 

 

(457

)

 

 

1,431

 

Total comprehensive income (loss)

$

(122,252

)

 

$

28,144

 

 

$

(259,026

)

 

$

(92,279

)

See accompanying notes.


4


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(Unaudited)

(In thousands, except share amounts)

 

 

 

Common Stock

 

 

Additional

Paid-In

 

 

Accumulated

Other

Comprehensive

 

 

Accumulated

 

 

Total

Stockholders'

 

 

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

 

Balance as of March 31, 2021

 

 

67,439,477

 

 

$

67

 

 

$

2,810,176

 

 

$

56

 

 

$

(1,755,717

)

 

$

1,054,582

 

 

Stock-based compensation

 

 

 

 

 

 

 

 

27,099

 

 

 

 

 

 

 

 

 

27,099

 

 

Issuance of common stock under equity plan

    awards, net of tax

 

 

325,473

 

 

 

1

 

 

 

12,741

 

 

 

 

 

 

 

 

 

12,742

 

 

Other comprehensive income

 

 

 

 

 

 

 

 

 

 

 

176

 

 

 

 

 

 

176

 

 

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(122,428

)

 

 

(122,428

)

 

Balance as of June 30, 2021

 

 

67,764,950

 

 

$

68

 

 

$

2,850,016

 

 

$

232

 

 

$

(1,878,145

)

 

$

972,171

 

 

 

 

 

Common Stock

 

 

Additional

Paid-In

 

 

Accumulated

Other

Comprehensive

 

 

Accumulated

 

 

Total

Stockholders'

 

 

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

 

Balance as of December 31, 2020

 

 

66,818,520

 

 

$

67

 

 

$

2,773,195

 

 

$

689

 

 

$

(1,619,576

)

 

$

1,154,375

 

 

Stock-based compensation

 

 

 

 

 

 

 

 

51,319

 

 

 

 

 

 

 

 

 

51,319

 

 

Issuance of common stock under equity plan

   awards, net of tax

 

 

946,430

 

 

 

1

 

 

 

25,502

 

 

 

 

 

 

 

 

 

25,503

 

 

Other comprehensive loss

 

 

 

 

 

 

 

 

 

 

 

(457

)

 

 

 

 

 

(457

)

 

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(258,569

)

 

 

(258,569

)

 

Balance as of June 30, 2021

 

 

67,764,950

 

 

$

68

 

 

$

2,850,016

 

 

$

232

 

 

$

(1,878,145

)

 

$

972,171

 

 

 

 

 

Common Stock

 

 

Additional

Paid-In

 

 

Accumulated

Other

Comprehensive

 

 

Accumulated

 

 

Total

Stockholders'

 

 

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

 

Balance as of March 31, 2020

 

 

59,488,873

 

 

$

59

 

 

$

2,162,667

 

 

$

(1,545

)

 

$

(1,552,035

)

 

$

609,146

 

 

Issuance of common stock in connection with

    at-the-market offering, net of issuance costs

 

 

283,333

 

 

 

 

 

 

20,391

 

 

 

 

 

 

 

 

 

20,391

 

 

Stock-based compensation

 

 

 

 

 

 

 

 

22,428

 

 

 

 

 

 

 

 

 

22,428

 

 

Issuance of common stock under equity plan

   awards, net of tax

 

 

641,550

 

 

 

1

 

 

 

15,477

 

 

 

 

 

 

 

 

 

15,478

 

 

Other comprehensive income

 

 

 

 

 

 

 

 

 

 

 

2,829

 

 

 

 

 

 

2,829

 

 

Net income

 

 

 

 

 

 

 

 

 

 

 

 

 

 

25,315

 

 

 

25,315

 

 

Balance as of June 30, 2020

 

 

60,413,756

 

 

$

60

 

 

$

2,220,963

 

 

$

1,284

 

 

$

(1,526,720

)

 

$

695,587

 

 

5


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(Unaudited)

(In thousands, except share amounts)

 

 

 

Common Stock

 

 

Additional

Paid-In

 

 

Accumulated

Other

Comprehensive

 

 

Accumulated

 

 

Total

Stockholders'

 

 

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

 

Balance as of December 31, 2019

 

 

57,838,220

 

 

$

58

 

 

$

2,086,863

 

 

$

(147

)

 

$

(1,433,010

)

 

$

653,764

 

 

       Issuance of common stock in connection

          with license agreement, net of issuance

          costs

 

 

1,243,913

 

 

 

1

 

 

 

55,267

 

 

 

 

 

 

 

 

 

55,268

 

 

Issuance of common stock in connection with

    at-the-market offering, net of issuance costs

 

 

283,333

 

 

 

 

 

 

20,391

 

 

 

 

 

 

 

 

 

20,391

 

 

Stock-based compensation

 

 

 

 

 

 

 

 

42,585

 

 

 

 

 

 

 

 

 

42,585

 

 

Issuance of common stock upon exercise of

    warrants and under equity plan awards, net

    of tax

 

 

1,048,290

 

 

 

1

 

 

 

15,857