UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, DC 20549
FORM
(Mark One)
For the quarterly period ended
OR
For the transition period from to .
Commission File No.
(Exact name of registrant as specified in its charter)
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(State or other jurisdiction of incorporation or organization) |
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(I.R.S. Employer Identification No.) |
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(Address of principal executive offices) |
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(Zip Code) |
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(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 |
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.
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).
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.
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Accelerated filer |
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Non-accelerated filer |
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Smaller reporting company |
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Emerging growth company |
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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 May 1, 2023, the registrant had
ULTRAGENYX PHARMACEUTICAL INC.
FORM 10-Q FOR THE QUARTER ENDED MARCH 31, 2023
INDEX
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Part I – |
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Item 1. |
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8 |
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Item 2. |
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Management’s Discussion and Analysis of Financial Condition and Results of Operations |
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21 |
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Item 3. |
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31 |
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Item 4. |
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31 |
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Part II – |
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Item 1. |
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31 |
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Item 1A. |
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32 |
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Item 2. |
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69 |
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Item 3. |
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69 |
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Item 4. |
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69 |
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Item 5. |
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69 |
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Item 6. |
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69 |
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71 |
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q , or 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:
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 such industry, business, market, and other data from
1
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.
2
PART I. FINANCIAL INFORMATION
Item 1. Financial Statements
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(Unaudited)
(In thousands, except share amounts)
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March 31, |
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December 31, |
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2023 |
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2022 |
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ASSETS |
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Current assets: |
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Cash and cash equivalents |
$ |
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$ |
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Marketable debt securities |
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Accounts receivable, net |
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Inventory |
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Prepaid expenses and other current assets |
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Total current assets |
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Property, plant, and equipment, net |
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Equity investments |
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Marketable debt securities |
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Right-of-use assets |
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Intangible assets, net |
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Goodwill |
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Other assets |
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Total assets |
$ |
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$ |
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LIABILITIES AND STOCKHOLDERS' EQUITY |
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Current liabilities: |
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Accounts payable |
$ |
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$ |
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Accrued liabilities |
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Contract liabilities |
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Lease liabilities |
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Liabilities for sales of future royalties |
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Total current liabilities |
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Lease liabilities |
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Deferred tax liabilities |
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Liabilities for sales of future royalties |
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Other liabilities |
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Total liabilities |
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Stockholders’ equity: |
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Preferred stock — |
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Common stock — |
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Treasury stock, at cost, |
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Deferred compensation obligation |
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Additional paid-in capital |
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Accumulated other comprehensive loss |
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( |
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( |
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Accumulated deficit |
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( |
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( |
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Total stockholders’ equity |
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Total liabilities and stockholders’ equity |
$ |
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$ |
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See accompanying notes.
3
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
(In thousands, except share and per share amounts)
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Three Months Ended March 31, |
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2023 |
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2022 |
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Revenues: |
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Collaboration and license |
$ |
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$ |
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Product sales |
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Royalty revenue |
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Total revenues |
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Operating expenses: |
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Cost of sales |
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Research and development |
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Selling, general and administrative |
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Total operating expenses |
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Loss from operations |
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( |
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( |
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Interest income |
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Change in fair value of equity investments |
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( |
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Non-cash interest expense on liabilities for sales of future royalties |
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( |
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Other income |
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Loss before income taxes |
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( |
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Provision for income taxes |
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( |
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( |
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Net loss |
$ |
( |
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$ |
( |
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Net loss per share, basic and diluted |
$ |
( |
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$ |
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Weighted-average shares used in computing net loss per share, |
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See accompanying notes.
4
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(Unaudited)
(In thousands)
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Three Months Ended March 31, |
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2023 |
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2022 |
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Net loss |
$ |
( |
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$ |
( |
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Other comprehensive income (loss): |
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Foreign currency translation adjustments |
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( |
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Unrealized gain (loss) on available-for-sale securities |
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( |
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Other comprehensive loss: |
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( |
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Total comprehensive loss |
$ |
( |
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$ |
( |
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See accompanying notes.
5
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY
(Unaudited)
(In thousands, except share amounts)
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Common Stock |
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Additional |
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Accumulated |
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Accumulated |
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Treasury |
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Deferred Compensation |
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Total |
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Shares |
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Amount |
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Capital |
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Income (Loss) |
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Deficit |
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Stock |
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Obligation |
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Equity |
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Balance as of December 31, 2022 |
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$ |
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$ |
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$ |
( |
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$ |
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$ |
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$ |
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$ |
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Stock-based compensation |
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— |
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— |
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— |
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— |
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— |
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— |
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Issuance of common stock under |
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( |
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— |
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— |
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— |
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— |
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( |
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Deferred compensation |
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— |
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— |
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— |
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— |
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— |
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( |
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— |
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Other comprehensive income |
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— |
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— |
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— |
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— |
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— |
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— |
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Net loss |
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— |
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— |
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— |
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— |
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( |
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— |
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— |
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( |
) |
Balance as of March 31, 2023 |
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$ |
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$ |
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$ |
( |
) |
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$ |
( |
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$ |
( |
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$ |
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$ |
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Common Stock |
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Additional |
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Accumulated |
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Accumulated |
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Treasury |
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Deferred Compensation |
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Total |
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Shares |
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Amount |
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Capital |
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Income (Loss) |
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Deficit |
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Stock |
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Obligation |
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Equity |
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Balance as of December 31, 2021 |
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$ |
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$ |
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$ |
( |
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$ |
( |
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$ |
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$ |
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$ |
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Stock-based compensation |
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— |
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— |
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— |
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— |
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— |
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— |
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Issuance of common stock under |
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— |
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— |
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— |
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— |
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Other comprehensive loss |
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— |
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— |
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— |
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( |
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— |
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— |
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— |
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( |
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Net loss |
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— |
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— |
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— |
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— |
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( |
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— |
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— |
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( |
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Balance as of March 31, 2022 |
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$ |
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$ |
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$ |
( |
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$ |
( |
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$ |
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$ |
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$ |
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See accompanying notes.
6
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(Unaudited)
(In thousands)
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Three Months Ended March 31, |
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2023 |
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2022 |
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Operating activities: |
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Net loss |
$ |
( |
) |
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$ |
( |
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Adjustments to reconcile net loss to net cash used in operating activities: |
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Stock-based compensation |
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Amortization of (discount) premium on marketable debt securities, net |
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( |
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Depreciation and amortization |
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Change in fair value of equity investments |
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Non-cash royalty revenue |
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( |
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( |
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Non-cash interest expense on liabilities for sales of future royalties |
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Other |
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Changes in operating assets and liabilities: |
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Accounts receivable |
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( |
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Inventory |
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( |
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Prepaid expenses and other assets |
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( |
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Accounts payable, accrued, and other liabilities |
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( |
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( |
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Contract liabilities |
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( |
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( |
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Net cash used in operating activities |
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( |
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( |
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Investing activities: |
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Purchase of property, plant, and equipment |
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( |
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( |
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Purchase of marketable debt securities |
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( |
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( |
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Proceeds from sale of marketable debt securities |
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Proceeds from maturities of marketable debt securities |
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Payment for intangible asset |
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( |
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Other |
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( |
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Net cash provided by (used in) investing activities |
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( |
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Financing activities: |
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Proceeds from the issuance of common stock under equity plan awards, net of tax |
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( |
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Other |
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( |
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Net cash (used in) provided by financing activities |
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( |
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Effect of exchange rate changes on cash |
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( |
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Net decrease in cash, cash equivalents and restricted cash |
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( |
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( |
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Cash, cash equivalents and restricted cash at beginning of period |
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