Document_and_Entity_Informatio
Document and Entity Information | 3 Months Ended | |
Mar. 31, 2015 | 6-May-15 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | FALSE | |
Document Period End Date | 31-Mar-15 | |
Document Fiscal Year Focus | 2015 | |
Document Fiscal Period Focus | Q1 | |
Trading Symbol | RARE | |
Entity Registrant Name | Ultragenyx Pharmaceutical Inc. | |
Entity Central Index Key | 1515673 | |
Current Fiscal Year End Date | -19 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Common Stock, Shares Outstanding | 35,902,599 |
CONDENSED_CONSOLIDATED_BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
Current assets: | ||
Cash and cash equivalents | $178,040 | $24,324 |
Short-term investments | 164,526 | 163,163 |
Prepaid expenses and other current assets | 6,877 | 5,929 |
Restricted cash | 1,071 | |
Total current assets | 350,514 | 193,416 |
Property and equipment, net | 3,514 | 3,033 |
Restricted cash | 1,031 | 744 |
Other assets | 781 | 774 |
Total assets | 355,840 | 197,967 |
Current liabilities: | ||
Accounts payable | 5,755 | 4,857 |
Accrued liabilities | 8,398 | 7,575 |
Deferred rent—current portion | 90 | 85 |
Total current liabilities | 14,243 | 12,517 |
Other liabilities | 478 | 505 |
Total liabilities | 14,721 | 13,022 |
Commitments and contingencies (Note 9) | ||
Stockholders’ equity: | ||
Preferred stock, par value of $0.001 per share—25,000,000 shares authorized; nil outstanding as of March 31, 2015 and December 31, 2014 | ||
Common stock, par value of $0.001 per share—250,000,000 shares authorized; 35,612,019 and 31,934,682 shares issued and outstanding as of March 31, 2015 and December 31, 2014 | 36 | 32 |
Additional paid-in capital | 501,597 | 324,128 |
Accumulated other comprehensive loss | -94 | -174 |
Accumulated deficit | -160,420 | -139,041 |
Total stockholders’ equity | 341,119 | 184,945 |
Total liabilities and stockholders’ equity | $355,840 | $197,967 |
CONDENSED_CONSOLIDATED_BALANCE1
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $0.00 | $0.00 |
Preferred stock, shares authorized | 25,000,000 | 25,000,000 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $0.00 | $0.00 |
Common stock, shares authorized | 250,000,000 | 250,000,000 |
Common stock, shares issued | 35,612,019 | 31,934,682 |
Common stock, shares outstanding | 35,612,019 | 31,934,682 |
CONDENSED_CONSOLIDATED_STATEME
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) (USD $) | 3 Months Ended | |
In Thousands, except Share data, unless otherwise specified | Mar. 31, 2015 | Mar. 31, 2014 |
Operating expenses: | ||
Research and development | $17,364 | $8,353 |
General and administrative | 4,138 | 1,986 |
Total operating expenses | 21,502 | 10,339 |
Loss from operations | -21,502 | -10,339 |
Other income (expense), net: | ||
Interest income | 273 | 93 |
Other expense, net | -150 | -3,384 |
Total other income (expense), net | 123 | -3,291 |
Net loss | -21,379 | -13,630 |
Net loss attributable to common stockholders | ($21,379) | ($18,438) |
Net loss per share attributable to common stockholders, basic and diluted | ($0.63) | ($0.85) |
Shares used in computing net loss per share attributable to common stockholders, basic and diluted | 34,008,830 | 21,582,435 |
CONDENSED_CONSOLIDATED_STATEME1
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) (USD $) | 3 Months Ended | |
In Thousands, unless otherwise specified | Mar. 31, 2015 | Mar. 31, 2014 |
Statement Of Income And Comprehensive Income [Abstract] | ||
Net loss | ($21,379) | ($13,630) |
Other comprehensive income: | ||
Unrealized gain (loss) on available-for-sale securities | 80 | -57 |
Total comprehensive loss | ($21,299) | ($13,687) |
CONDENSED_CONSOLIDATED_STATEME2
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) (USD $) | 3 Months Ended | |
Mar. 31, 2015 | Mar. 31, 2014 | |
Operating activities: | ||
Net loss | ($21,379,000) | ($13,630,000) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 201,000 | 117,000 |
Amortization of premium (discount) on investment securities, net | 965,000 | 479,000 |
Stock-based compensation | 2,408,000 | 795,000 |
Revaluation of convertible preferred stock warrant liability | 0 | 3,324,000 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | -948,000 | -2,391,000 |
Other assets | -7,000 | -187,000 |
Accounts payable | 437,000 | 2,058,000 |
Accrued liabilities and other liabilities | 664,000 | -1,186,000 |
Net cash used in operating activities | -17,659,000 | -10,621,000 |
Investing activities: | ||
Purchase of property and equipment | -459,000 | -569,000 |
Purchase of investments | -77,254,000 | -87,998,000 |
Proceeds from the sale of investments | 12,766,000 | |
Proceeds from maturities of investments | 62,240,000 | 23,462,000 |
Increase in restricted cash | -1,358,000 | -293,000 |
Net cash used in investing activities | -4,065,000 | -65,398,000 |
Financing activities: | ||
Proceeds from issuance of common stock, net of issuance costs | 175,440,000 | 128,385,000 |
Payment of preferred stock dividend | -4,346,000 | |
Net cash provided by financing activities | 175,440,000 | 124,039,000 |
Net increase in cash and cash equivalents | 153,716,000 | 48,020,000 |
Cash and cash equivalents at beginning of period | 24,324,000 | 7,427,000 |
Cash and cash equivalents at end of period | 178,040,000 | 55,447,000 |
Supplemental disclosures of non-cash investing and financing information: | ||
Reclassification of warrant liability to equity upon conversion to common stock warrants | 6,743,000 | |
Conversion of Series A and Series B preferred stock to common stock | $129,360,000 |
Organization
Organization | 3 Months Ended | |
Mar. 31, 2015 | ||
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Organization | 1 | Organization |
Ultragenyx Pharmaceutical Inc. (the Company) is a biopharmaceutical company incorporated in California on April 22, 2010. The Company subsequently reincorporated in the state of Delaware in June 2011. | ||
The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating metabolic genetic diseases. The Company is currently conducting a Phase 2 extension study of sialic acid, extended release (SA-ER) in patients with hereditary inclusion body myopathy (HIBM), a progressive muscle-wasting disorder; a Phase 3 study of recombinant human beta-glucuronidase (rhGUS) in patients with mucopolysaccharidosis 7, or MPS 7, a rare lysosomal storage disease; a Phase 2 clinical study for triheptanoin in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS), a brain energy deficiency; a Phase 2 clinical study of triheptanoin in patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD), a genetic disorder in which the body is unable to convert long chain fatty acids into energy; and Phase 2 studies of KRN23, an antibody targeting fibroblast growth factor 23, or FGF23, in patients with X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), both rare genetic diseases that impair bone growth mineralization. The Company is in the clinical stage as of December 31, 2014, and since inception has been engaged in developing its product candidates, raising capital and recruiting personnel. The Company operates in the United States of America in one reportable segment. | ||
In February 2015, the Company completed an underwritten public offering in which the Company sold 3,450,000 shares of common stock, which included 450,000 shares of common stock purchased by the underwriters pursuant to an option granted to them in connection with the offering, at a public offering price of $54.00 per share. The total proceeds that the Company received from the offering were approximately $175.1 million, net of underwriting discounts and commissions of approximately $11.2 million. After deducting estimated offering expenses payable of approximately $0.6 million, net proceeds were $174.5 million. |
Summary_of_Significant_Account
Summary of Significant Accounting Policies | 3 Months Ended | |
Mar. 31, 2015 | ||
Accounting Policies [Abstract] | ||
Summary of Significant Accounting Policies | 2 | Summary of Significant Accounting Policies |
Basis of Presentation | ||
The accompanying unaudited condensed consolidated financial statements include the amounts of the Company and our wholly-owned subsidiary and have been prepared in accordance with U.S. general accepted accounting principles (“U.S. GAAP”) for interim financial information and in accordance with the instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by generally accepted accounting principles for complete financial statements. The unaudited interim consolidated financial statements have been prepared on the same basis as the annual financial statements. In the opinion of management, the accompanying unaudited condensed consolidated financial statements reflect all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation. These financial statements should be read in conjunction with the audited financial statements and notes thereto for the preceding fiscal year contained in the Company’s Annual Report on Form 10-K filed on March 27, 2015 with the SEC. | ||
The results of operations for the three months ended March 31, 2015 are not necessarily indicative of the results to be expected for the year ending December 31, 2015. The condensed balance sheet as of December 31, 2014 has been derived from audited financial statements at that date but does not include all of the information required by U.S. GAAP for complete financial statements. | ||
Use of Estimates | ||
The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the financial statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical study accruals, fair value of assets and liabilities, convertible preferred stock and related warrants, common stock, income taxes and stock-based compensation. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates. | ||
Cash and Cash Equivalents | ||
The Company considers all highly liquid investments with original maturities of three months or less from the date of purchase to be cash equivalents. Cash equivalents consist primarily of amounts invested in money market accounts and corporate bonds. | ||
Short-Term Investments | ||
All investments have been classified as “available-for-sale” and are carried at estimated fair value as determined based upon quoted market prices or pricing models for similar securities. Management determines the appropriate classification of its investments in debt securities at the time of purchase and reevaluates such designation as of each balance sheet date. Unrealized gains and losses are excluded from earnings and were reported as a component of comprehensive loss. Realized gains and losses and declines in fair value judged to be other than temporary, if any, on available-for-sale securities are included in interest income and other expense, net, respectively. The cost of securities sold is based on the specific-identification method. Interest on marketable securities is included in interest income. | ||
Concentration of Credit Risk and Other Risks and Uncertainties | ||
Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents, and short-term investments. The Company’s cash, cash equivalents, and short-term investments are held by financial institutions that management believes are of high credit quality. The Company’s investment policy limits investments to fixed income securities denominated and payable in U.S. dollars such as U.S. government obligations, money market instruments and funds, corporate bonds, and asset-backed securities and places restrictions on maturities and concentrations by type and issuer. Such deposits may, at times, exceed federally insured limits. The Company has not experienced any losses on its deposits of cash and cash equivalents and its accounts are monitored by management to mitigate risk. The Company is exposed to credit risk in the event of default by the financial institutions holding its cash and cash equivalents, corporate bond issuers and other financial instruments to the extent recorded in the balance sheets. | ||
Income Taxes | ||
The Company uses the liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the differences between the financial reporting and the tax bases of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The Company must then assess the likelihood that the resulting deferred tax assets will be realized. A valuation allowance is provided when it is more likely than not that some portion or all of a deferred tax asset will not be realized. Due to the Company’s lack of earnings history, the net deferred tax assets have been fully offset by a valuation allowance. | ||
The Company recognizes benefits of uncertain tax positions if it is more likely than not that such positions will be sustained upon examination based solely on their technical merits, as the largest amount of benefit that is more likely than not to be realized upon the ultimate settlement. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have been no interest or penalties charged in relation to the unrecognized tax benefits. | ||
Net Loss per Share Attributable to Common Stockholders | ||
Basic net loss per share attributable to common stockholders is calculated by dividing the net loss attributable to common stockholders by the weighted-average number of shares of common stock outstanding during the period, without consideration for common stock equivalents. The net loss attributable to common stockholders is calculated by adjusting the net loss of the Company for the accretion on the Series A convertible preferred stock and cumulative dividends paid on Series A and B convertible preferred stock. Diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since the effects of potentially dilutive securities are antidilutive. In periods when we have incurred a net loss, convertible preferred stock, options and warrants to purchase common stock and convertible preferred stock warrants are considered common stock equivalents, but have been excluded from the calculation of diluted net loss per share attributable to common stockholders, as their effect is antidilutive. | ||
Recently Issued Accounting Pronouncements | ||
In August 2014, the FASB issued ASU No. 2014-15, Presentation of Financial Statements — Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, which requires management to evaluate, in connection with preparing financial statements for each annual and interim reporting period, whether there are conditions or events, considered in the aggregate, that raise substantial doubt about an entity’s ability to continue as a going concern within one year after the date that the financial statements are issued and provide related disclosures. This ASU will be effective for the Company in fiscal year 2016. Early adoption is permitted. We are currently assessing the future impact of this ASU in the financial statements. | ||
Fair_Value_Measurements
Fair Value Measurements | 3 Months Ended | ||||||||||||||||
Mar. 31, 2015 | |||||||||||||||||
Fair Value Disclosures [Abstract] | |||||||||||||||||
Fair Value Measurements | |||||||||||||||||
3 | Fair Value Measurements | ||||||||||||||||
Financial assets and liabilities are recorded at fair value. The carrying amount of certain financial instruments, including cash and cash equivalents, accounts payable and accrued liabilities approximate fair value due to their relatively short maturities. Assets and liabilities recorded at fair value on a recurring basis in the condensed consolidated balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows: | |||||||||||||||||
Level 1—Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date; | |||||||||||||||||
Level 2—Inputs are observable, unadjusted quoted prices in active markets for similar assets or liabilities, unadjusted quoted prices for identical or similar assets or liabilities in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities; and | |||||||||||||||||
Level 3—Unobservable inputs that are significant to the measurement of the fair value of the assets or liabilities that are supported by little or no market data. | |||||||||||||||||
The following tables set forth the fair value of the Company’s financial assets and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): | |||||||||||||||||
31-Mar-15 | |||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | ||||||||||||||
Financial Assets: | |||||||||||||||||
Money market funds | $ | 142,829 | $ | — | $ | — | $ | 142,829 | |||||||||
Corporate bonds | — | 170,452 | — | 170,452 | |||||||||||||
Asset backed securities | — | 9,520 | — | 9,520 | |||||||||||||
U.S. Government agency securities | — | 2,998 | — | 2,998 | |||||||||||||
Commercial paper | — | 2,497 | — | 2,497 | |||||||||||||
Total financial assets | $ | 142,829 | $ | 185,467 | $ | — | $ | 328,296 | |||||||||
31-Dec-14 | |||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | ||||||||||||||
Financial Assets: | |||||||||||||||||
Money market funds | $ | 8,627 | $ | — | $ | — | $ | 8,627 | |||||||||
Corporate bonds | — | 152,942 | — | 152,942 | |||||||||||||
Asset backed securities | — | 9,542 | — | 9,542 | |||||||||||||
U.S. Government agency securities | — | 4,485 | — | 4,485 | |||||||||||||
Other | — | 209 | — | 209 | |||||||||||||
Total financial assets | $ | 8,627 | $ | 167,178 | $ | — | $ | 175,805 | |||||||||
The convertible preferred stock warrant liability was classified as a Level 3 liability. As of January 30, 2014, the Company determined the estimated fair value of the warrants using the Black-Scholes option-pricing model. Inputs used to determine the fair value included the value of the Company’s common stock upon closing of the IPO of $21.00, the remaining contractual term of the warrants of 7.0 years, risk-free interest rate of 2.19% and expected volatility of 70%. Generally, increases (decreases) in the equity value of the Company would result in a directionally similar impact to the fair value measurement of the preferred stock warrant liability. The preferred stock warrants were converted to common stock warrants upon the completion of the IPO and are no longer subject to remeasurement. | |||||||||||||||||
The following table sets forth a summary of the changes in the estimated fair value of the Company’s convertible preferred stock warrants, which were measured at fair value on a recurring basis until their conversion to common stock warrants and related reclassification to additional paid-in capital (in thousands): | |||||||||||||||||
Three Months Ended March 31, | |||||||||||||||||
2015 | 2014 | ||||||||||||||||
Fair value, beginning of period | $ | — | $ | 3,419 | |||||||||||||
Change in fair value recorded as a loss in other expense, net | — | 3,324 | |||||||||||||||
Reclassification of warrant liability to additional paid-in capital | — | (6,743 | ) | ||||||||||||||
Fair value, end of period | $ | — | $ | — | |||||||||||||
The Company recorded $0 and $3.3 million in other expense for the three months ended March 31, 2015 and 2014, representing the change in fair value of the warrants for the respective periods. | |||||||||||||||||
Balance_Sheet_Components
Balance Sheet Components | 3 Months Ended | |||||||||||||||
Mar. 31, 2015 | ||||||||||||||||
Investments Debt And Equity Securities [Abstract] | ||||||||||||||||
Balance Sheet Components | 4.Balance Sheet Components | |||||||||||||||
Cash Equivalents and Short-term Investments | ||||||||||||||||
The fair values of cash equivalents and short-term investments classified as available-for-sale securities, consisted of the following (in thousands): | ||||||||||||||||
31-Mar-15 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 142,829 | $ | — | $ | — | $ | 142,829 | ||||||||
Corporate bonds classified as cash equivalents | 20,941 | — | — | 20,941 | ||||||||||||
Commercial paper classified as short-term investments | 2,497 | 2 | (2 | ) | 2,497 | |||||||||||
Corporate bonds classified as short-term investments | 149,607 | 8 | (104 | ) | 149,511 | |||||||||||
Asset backed securities classified as short-term investments | 9,522 | — | (2 | ) | 9,520 | |||||||||||
U.S. Government agency securities classified as short-term | 2,994 | 4 | — | 2,998 | ||||||||||||
investments | ||||||||||||||||
Total | $ | 328,390 | $ | 14 | $ | (108 | ) | $ | 328,296 | |||||||
31-Dec-14 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 8,627 | $ | — | $ | — | $ | 8,627 | ||||||||
Corporate bonds classified as cash equivalents | 3,806 | 1 | — | 3,807 | ||||||||||||
Corporate bonds classified as short-term investments | 149,303 | 4 | (172 | ) | 149,135 | |||||||||||
Asset backed securities classified as short-term investments | 9,546 | — | (4 | ) | 9,542 | |||||||||||
U.S. Government agency securities classified as short-term | 4,488 | 1 | (4 | ) | 4,485 | |||||||||||
investments | ||||||||||||||||
Other classified as cash equivalents | 209 | — | — | 209 | ||||||||||||
Total | $ | 175,979 | $ | 6 | $ | (180 | ) | $ | 175,805 | |||||||
At March 31, 2015, the remaining contractual maturities of available-for-sale securities were less than two years. There have been no significant realized gains or losses on available-for-sale securities for the periods presented. | ||||||||||||||||
Accrued Liabilities | ||||||||||||||||
Accrued liabilities consist of the following (in thousands): | ||||||||||||||||
March 31, | December 31, | |||||||||||||||
2015 | 2014 | |||||||||||||||
Research and clinical study expenses | $ | 4,271 | $ | 2,703 | ||||||||||||
Payroll and related expenses | 3,217 | 4,205 | ||||||||||||||
Other | 910 | 667 | ||||||||||||||
Total accrued liabilities | $ | 8,398 | $ | 7,575 | ||||||||||||
License_and_Research_Agreement
License and Research Agreements | 3 Months Ended | |
Mar. 31, 2015 | ||
Research Grant Agreement [Abstract] | ||
License and Research Agreements | ||
5 | License and Research Agreements | |
Nobelpharma License Agreement | ||
In September 2010, the Company entered into a collaboration and license agreement with Nobelpharma Co., Ltd. (Nobelpharma). Under the terms of this collaboration and license agreement, each party granted the other party a worldwide exclusive license under certain of that party’s intellectual property related to the compound identified as N-acetylneuraminic acid, also known as sialic acid, to develop, manufacture, and commercialize products. Nobelpharma’s licensed territory includes Japan and certain other Asian countries, and the Company’s licensed territory includes the rest of the world. | ||
Under the collaboration and license agreement, the Company paid Nobelpharma $111,000 (10 million Yen) for the license, which was recorded as research and development expense in 2010, and also issued 76,567 shares of common stock to Nobelpharma with a minimal value. The Company is required to pay Nobelpharma royalties based on net sales upon product sales commencement. In addition, the Company is required to make certain payments to Nobelpharma based upon achievement of certain development and approval milestones. The Company paid $495,000 in development milestone payments since the inception of the agreement through March 31, 2015. The remaining total aggregate payments, if all milestones are achieved by Nobelpharma, would be 200 million Yen (approximately $1.7 million based on the exchange rate at March 31, 2015). The Company will pay a high single digit royalty on net sales in the Company’s territory and will receive a mid-single digit royalty on net sales in the Nobelpharma territory, excluding Japan, if such product sales are ever achieved. Net sales, as defined in the collaboration and license agreement, represent the net sales of products whereby the licensed compound is the active ingredient. If the products include other active ingredients, the portion of the net sales allocated to the licensed compound would be used in determining the royalty payments. | ||
Saint Louis University License Agreement | ||
In November 2010, the Company entered into a license agreement with Saint Louis University (SLU). Under the terms of this license agreement, SLU granted the Company an exclusive worldwide license to make, have made, use, import, offer for sale, and sell therapeutics related to SLU’s beta-glucuronidase product for use in the treatment of human diseases. | ||
Under the license agreement, the Company paid SLU an up-front fee of $10,000, which was recorded as research and development expense in 2010. The Company will be required to make a milestone payment of $100,000 upon approval of a glucuronidase-based enzyme therapy for treatment of MPS 7. Additionally, upon reaching a certain level of cumulative worldwide sales of the product, the Company will be required to pay to SLU a low single-digit royalty on net sales of the licensed products in any country or region, if such product sales are ever achieved. | ||
AAIPharma License Agreement | ||
In March 2011, the Company entered into a license agreement with AAIPharma Services Corp. (AAIPharma). Under the terms of this license agreement, AAIPharma granted the Company a fully paid-up, royalty-free, exclusive, perpetual, and irrevocable license to research, develop, make, have made, use, import, offer for sale, and sell products incorporating AAIPharma’s controlled release matrix solid dose oral tablet. Under the license agreement, the Company will pay a mid-single digit percentage of any sublicense revenue received by Ultragenyx related to the sublicense of AAIPharma technology that had been initially licensed by Ultragenyx. | ||
HIBM Research Group License Agreement | ||
In April 2012, the Company entered into an exclusive license agreement with HIBM Research Group (HRG). Under the terms of this license agreement, HRG granted the Company an exclusive worldwide license to certain intellectual property related to the treatment of HIBM. Under the license agreement, the Company paid HRG an up-front fee of $25,000 which was recorded as research and development expense during the year ended December 31, 2012. The Company may make future payments that aggregate up to $300,000 and that are contingent upon attainment of various development and approval milestones. Additionally, the Company will pay to HRG a royalty of less than 1% of net sales of the licensed products in the licensed territories, if such product sales are ever achieved. | ||
St. Jude Children’s Research Hospital License Agreement | ||
In September 2012, the Company entered into a license agreement with St. Jude Children’s Research Hospital (St. Jude). Under the terms of this license agreement, St. Jude granted the Company an exclusive license under certain know-how to research, develop, make, use, offer to sell, import, and otherwise commercialize and exploit St. Jude’s protective protein, cathepsin, a protein product to treat, prevent, and/or diagnose galactosialidosis and other monogenetic diseases. | ||
Under the license agreement, the Company paid St. Jude an up-front fee of $10,000 which was recorded as research and development expense during the year ended December 31, 2012. Additionally, the Company will pay to St. Jude a royalty of less than 1% on net sales of the licensed products in the licensed territories, if such product sales are ever achieved. | ||
Baylor Research Institute License Agreement | ||
In September 2012, the Company entered into a license agreement with Baylor Research Institute (BRI). Under the terms of this license agreement, BRI exclusively licensed to the Company certain intellectual property related to triheptanoin for North America. Under the license agreement, the Company paid BRI an up-front fee of $250,000 which was recorded as research and development expense during the year ended December 31, 2012. In June 2013, the Company notified BRI that it was exercising its option pursuant to the agreement to license the rights to triheptanoin in all territories outside of the United States, Canada and Mexico and paid the option exercise fee of $750,000. | ||
The Company may make future payments of up to $10.5 million contingent upon attainment of various development milestones and $7.5 million contingent upon attainment of various sales milestones. Additionally, the Company will pay to BRI a mid-single digit royalty on net sales of the licensed product in the licensed territories, if such product sales are ever achieved. | ||
Kyowa Hakko Kirin Collaboration and License Agreement | ||
In August 2013, the Company entered into a collaboration and license agreement with Kyowa Hakko Kirin Co., Ltd. (KHK). Under the terms of this collaboration and license agreement, the Company and KHK will collaborate on the development and commercialization of certain products containing KRN23, an antibody directed towards FGF23, in the field of orphan diseases in the United States and Canada, or the profit share territory, and in the European Union, Switzerland, and Turkey, or the European territory, and the Company will have the right to develop and commercialize such products in the field of orphan diseases in Mexico and Central and South America, or Latin America. In the field of orphan diseases, and except for ongoing studies being conducted by KHK, the Company will be the lead party for development activities in the profit share territory and in the European territory until the applicable transition date. The Company will share the costs for development activities in the profit share territory and European territory conducted pursuant to the development plan before the applicable transition date equally with KHK. On the applicable transition date in the relevant territory, KHK will become the lead party and be responsible for these costs. However, the Company will continue to share the costs of the studies commenced prior to the applicable transition date equally with KHK. The Company has the primary responsibility for conducting certain research and development services. The Company is obligated to provide assistance in accordance with the agreed upon development plan as well as participate on various committees. If KRN23 is approved, the Company and KHK will share commercial responsibilities and profits in the profit share territory until the applicable transition date, KHK will commercialize KRN23 in the European territory and the Company will develop and commercialize KRN23 in Latin America. KHK will manufacture and supply KRN23 for clinical use globally and will manufacture and supply KRN23 for commercial use in the profit share territory and Latin America. | ||
The Company is accounting for the agreement as a collaboration arrangement as defined in ASC 808, Collaborative Agreements; accordingly, the Company recognized $1.4 million and $710,000 for its share of the costs as research and development expenses for the three months ended March 31, 2015 and 2014, respectively. As of March 31, 2015 and 2014, the Company had a receivable in the amount of $1.5 million and $878,000 from KHK and an accrued liability of $25,000 and $201,000, respectively, for this collaboration arrangement. | ||
Common_Stock_Warrants
Common Stock Warrants | 3 Months Ended | ||||||||||||||
Mar. 31, 2015 | |||||||||||||||
Warrants And Rights Note Disclosure [Abstract] | |||||||||||||||
Common Stock Warrants | 6 | Common Stock Warrants | |||||||||||||
Upon the closing of the Company’s IPO in February 2014, the convertible preferred stock warrants were converted into warrants to purchase common stock. Accordingly, the warrants were reclassified from a liability to permanent equity and were no longer subject to remeasurement. | |||||||||||||||
The table sets forth the outstanding common stock warrants for the periods presented: | |||||||||||||||
Outstanding at March 31, | Outstanding at December 31, | Date Issued | Term | Exercise Price | |||||||||||
2015 | 2014 | ||||||||||||||
83,167 | 83,167 | Jun-10 | 10 years | $ | 3.006 | ||||||||||
174,651 | 174,651 | Feb-11 | 10 years | 3.006 | |||||||||||
66,533 | 66,533 | Jun-11 | 10 years | 3.006 | |||||||||||
324,351 | 324,351 | ||||||||||||||
StockBased_Compensation
Stock-Based Compensation | 3 Months Ended | ||||||||
Mar. 31, 2015 | |||||||||
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |||||||||
Stock-Based Compensation | 7 | Stock-Based Compensation | |||||||
2014 Incentive Plan | |||||||||
In 2014, the Company adopted the 2014 Incentive Plan (the 2014 Plan), which became effective upon the closing of the Company’s IPO in February 2014. The 2014 Plan provides for automatic annual increases in shares available for grant, beginning on January 1, 2015 through January 1, 2024. As of March 31, 2015, there were 2,595,430 shares reserved under the 2014 Plan for the future issuance of equity awards. | |||||||||
The table below sets forth the functional classification of stock-based compensation expense, net of estimated forfeitures, for the periods presented (in thousands): | |||||||||
Three Months Ended March 31, | |||||||||
2015 | 2014 | ||||||||
Research and development | $ | 1,841 | $ | 705 | |||||
General and administrative | 567 | 90 | |||||||
Total stock-based compensation | $ | 2,408 | $ | 795 | |||||
Defined_Contribution_Plan
Defined Contribution Plan | 3 Months Ended | |
Mar. 31, 2015 | ||
Defined Contribution Pension And Other Postretirement Plans Disclosure [Abstract] | ||
Defined Contribution Plan | 8 | Defined Contribution Plan |
In March 2013, the Company began to sponsor a 401(k) retirement plan, in which substantially all of its full-time employees are eligible to participate. Eligible participants may contribute a percentage of their annual compensation to this plan, subject to statutory limitations. Prior to 2015, the Company had not provided any contributions to the plan. In 2015, the Company began to make contributions to the Plan for eligible participants, and recorded $123,000 as contribution expenses for the three months ended March 31, 2015. |
Commitment_and_Contingencies
Commitment and Contingencies | 3 Months Ended | |
Mar. 31, 2015 | ||
Commitments And Contingencies Disclosure [Abstract] | ||
Commitments and Contingencies | 9 | Commitments and Contingencies |
Commitments | ||
The Company has various manufacturing, clinical, research, and other contracts with vendors in the conduct of the normal course of its business. All other significant contracts as of March 31, 2015 were terminable, with varying provisions regarding termination. If a contract with a specific vendor were to be terminated, the Company would only be obligated for the products or services that the Company had received at the time the termination became effective. | ||
Contingencies | ||
While there are no legal proceedings the Company is aware of, the Company may become party to various claims and complaints arising in the ordinary course of business. Management does not believe that any ultimate liability resulting from any such claims will have a material adverse effect on its results of operations, financial position, or liquidity. However, management cannot give any assurance regarding the ultimate outcome of such claims, and their resolution could be material to the Company for any particular period, depending upon the level of income or loss for the period, as well as the Company’s consolidated balance sheet. | ||
Net_Loss_per_Share_Attributabl
Net Loss per Share Attributable to Common Stockholders | 3 Months Ended | |||||||
Mar. 31, 2015 | ||||||||
Earnings Per Share [Abstract] | ||||||||
Net Loss per Share Attributable to Common Stockholders | 10 | Net Loss per Share Attributable to Common Stockholders | ||||||
The following table sets forth the computation of the basic and diluted net loss per share attributable to common stockholders (in thousands, except share and per share data): | ||||||||
Three Months Ended | ||||||||
March 31, | ||||||||
2015 | 2014 | |||||||
Numerator: | ||||||||
Net loss | $ | (21,379 | ) | $ | (13,630 | ) | ||
Accretion and dividends on convertible preferred stock | — | (4,808 | ) | |||||
Net loss attributable to common stockholders | $ | (21,379 | ) | $ | (18,438 | ) | ||
Denominator: | ||||||||
Weighted-average common shares outstanding | 34,008,830 | 21,582,435 | ||||||
Less: weighted-average unvested common shares subject to repurchase | — | — | ||||||
Weighted-average shares used to compute net loss per share attributable | 34,008,830 | 21,582,435 | ||||||
to common stockholders, basic and diluted | ||||||||
Net loss per share attributable to common stockholders, basic and diluted | $ | (0.63 | ) | $ | (0.85 | ) | ||
The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been antidilutive: | ||||||||
Three Months Ended March 31, | ||||||||
2015 | 2014 | |||||||
Convertible preferred stock (as if converted) | — | 6,532,828 | ||||||
Stock options to purchase common stock | 2,698,846 | 2,315,345 | ||||||
Unvested restricted stock units | 33,856 | — | ||||||
Convertible preferred stock warrants (as if converted) | — | 117,820 | ||||||
Common stock warrants | 324,351 | 235,639 | ||||||
3,057,053 | 9,201,632 | |||||||
Summary_of_Significant_Account1
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2015 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation |
The accompanying unaudited condensed consolidated financial statements include the amounts of the Company and our wholly-owned subsidiary and have been prepared in accordance with U.S. general accepted accounting principles (“U.S. GAAP”) for interim financial information and in accordance with the instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by generally accepted accounting principles for complete financial statements. The unaudited interim consolidated financial statements have been prepared on the same basis as the annual financial statements. In the opinion of management, the accompanying unaudited condensed consolidated financial statements reflect all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation. These financial statements should be read in conjunction with the audited financial statements and notes thereto for the preceding fiscal year contained in the Company’s Annual Report on Form 10-K filed on March 27, 2015 with the SEC. | |
The results of operations for the three months ended March 31, 2015 are not necessarily indicative of the results to be expected for the year ending December 31, 2015. The condensed balance sheet as of December 31, 2014 has been derived from audited financial statements at that date but does not include all of the information required by U.S. GAAP for complete financial statements. | |
Use of Estimates | Use of Estimates |
The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the financial statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical study accruals, fair value of assets and liabilities, convertible preferred stock and related warrants, common stock, income taxes and stock-based compensation. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates. | |
Cash and Cash Equivalents | Cash and Cash Equivalents |
The Company considers all highly liquid investments with original maturities of three months or less from the date of purchase to be cash equivalents. Cash equivalents consist primarily of amounts invested in money market accounts and corporate bonds. | |
Short-Term Investments | |
Short-Term Investments | |
All investments have been classified as “available-for-sale” and are carried at estimated fair value as determined based upon quoted market prices or pricing models for similar securities. Management determines the appropriate classification of its investments in debt securities at the time of purchase and reevaluates such designation as of each balance sheet date. Unrealized gains and losses are excluded from earnings and were reported as a component of comprehensive loss. Realized gains and losses and declines in fair value judged to be other than temporary, if any, on available-for-sale securities are included in interest income and other expense, net, respectively. The cost of securities sold is based on the specific-identification method. Interest on marketable securities is included in interest income. | |
Concentration of Credit Risk and Other Risks and Uncertainties | Concentration of Credit Risk and Other Risks and Uncertainties |
Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents, and short-term investments. The Company’s cash, cash equivalents, and short-term investments are held by financial institutions that management believes are of high credit quality. The Company’s investment policy limits investments to fixed income securities denominated and payable in U.S. dollars such as U.S. government obligations, money market instruments and funds, corporate bonds, and asset-backed securities and places restrictions on maturities and concentrations by type and issuer. Such deposits may, at times, exceed federally insured limits. The Company has not experienced any losses on its deposits of cash and cash equivalents and its accounts are monitored by management to mitigate risk. The Company is exposed to credit risk in the event of default by the financial institutions holding its cash and cash equivalents, corporate bond issuers and other financial instruments to the extent recorded in the balance sheets. | |
Income Taxes | Income Taxes |
The Company uses the liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the differences between the financial reporting and the tax bases of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The Company must then assess the likelihood that the resulting deferred tax assets will be realized. A valuation allowance is provided when it is more likely than not that some portion or all of a deferred tax asset will not be realized. Due to the Company’s lack of earnings history, the net deferred tax assets have been fully offset by a valuation allowance. | |
The Company recognizes benefits of uncertain tax positions if it is more likely than not that such positions will be sustained upon examination based solely on their technical merits, as the largest amount of benefit that is more likely than not to be realized upon the ultimate settlement. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have been no interest or penalties charged in relation to the unrecognized tax benefits. | |
Net Loss per Share Attributable to Common Stockholders | Net Loss per Share Attributable to Common Stockholders |
Basic net loss per share attributable to common stockholders is calculated by dividing the net loss attributable to common stockholders by the weighted-average number of shares of common stock outstanding during the period, without consideration for common stock equivalents. The net loss attributable to common stockholders is calculated by adjusting the net loss of the Company for the accretion on the Series A convertible preferred stock and cumulative dividends paid on Series A and B convertible preferred stock. Diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since the effects of potentially dilutive securities are antidilutive. In periods when we have incurred a net loss, convertible preferred stock, options and warrants to purchase common stock and convertible preferred stock warrants are considered common stock equivalents, but have been excluded from the calculation of diluted net loss per share attributable to common stockholders, as their effect is antidilutive. | |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements |
In August 2014, the FASB issued ASU No. 2014-15, Presentation of Financial Statements — Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, which requires management to evaluate, in connection with preparing financial statements for each annual and interim reporting period, whether there are conditions or events, considered in the aggregate, that raise substantial doubt about an entity’s ability to continue as a going concern within one year after the date that the financial statements are issued and provide related disclosures. This ASU will be effective for the Company in fiscal year 2016. Early adoption is permitted. We are currently assessing the future impact of this ASU in the financial statements. |
Fair_Value_Measurements_Tables
Fair Value Measurements (Tables) | 3 Months Ended | ||||||||||||||||
Mar. 31, 2015 | |||||||||||||||||
Fair Value Disclosures [Abstract] | |||||||||||||||||
Summary of Financial Assets and Liabilities Measured on Recurring Basis | The following tables set forth the fair value of the Company’s financial assets and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands): | ||||||||||||||||
31-Mar-15 | |||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | ||||||||||||||
Financial Assets: | |||||||||||||||||
Money market funds | $ | 142,829 | $ | — | $ | — | $ | 142,829 | |||||||||
Corporate bonds | — | 170,452 | — | 170,452 | |||||||||||||
Asset backed securities | — | 9,520 | — | 9,520 | |||||||||||||
U.S. Government agency securities | — | 2,998 | — | 2,998 | |||||||||||||
Commercial paper | — | 2,497 | — | 2,497 | |||||||||||||
Total financial assets | $ | 142,829 | $ | 185,467 | $ | — | $ | 328,296 | |||||||||
31-Dec-14 | |||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | ||||||||||||||
Financial Assets: | |||||||||||||||||
Money market funds | $ | 8,627 | $ | — | $ | — | $ | 8,627 | |||||||||
Corporate bonds | — | 152,942 | — | 152,942 | |||||||||||||
Asset backed securities | — | 9,542 | — | 9,542 | |||||||||||||
U.S. Government agency securities | — | 4,485 | — | 4,485 | |||||||||||||
Other | — | 209 | — | 209 | |||||||||||||
Total financial assets | $ | 8,627 | $ | 167,178 | $ | — | $ | 175,805 | |||||||||
Summary of Changes in Estimated Fair Value | The following table sets forth a summary of the changes in the estimated fair value of the Company’s convertible preferred stock warrants, which were measured at fair value on a recurring basis until their conversion to common stock warrants and related reclassification to additional paid-in capital (in thousands): | ||||||||||||||||
Three Months Ended March 31, | |||||||||||||||||
2015 | 2014 | ||||||||||||||||
Fair value, beginning of period | $ | — | $ | 3,419 | |||||||||||||
Change in fair value recorded as a loss in other expense, net | — | 3,324 | |||||||||||||||
Reclassification of warrant liability to additional paid-in capital | — | (6,743 | ) | ||||||||||||||
Fair value, end of period | $ | — | $ | — | |||||||||||||
Balance_Sheet_Components_Table
Balance Sheet Components (Tables) | 3 Months Ended | |||||||||||||||
Mar. 31, 2015 | ||||||||||||||||
Investments Debt And Equity Securities [Abstract] | ||||||||||||||||
Summary of Fair Values of Cash Equivalents and Short-Term Investments Classified as Available-for-Sale Securities | The fair values of cash equivalents and short-term investments classified as available-for-sale securities, consisted of the following (in thousands): | |||||||||||||||
31-Mar-15 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 142,829 | $ | — | $ | — | $ | 142,829 | ||||||||
Corporate bonds classified as cash equivalents | 20,941 | — | — | 20,941 | ||||||||||||
Commercial paper classified as short-term investments | 2,497 | 2 | (2 | ) | 2,497 | |||||||||||
Corporate bonds classified as short-term investments | 149,607 | 8 | (104 | ) | 149,511 | |||||||||||
Asset backed securities classified as short-term investments | 9,522 | — | (2 | ) | 9,520 | |||||||||||
U.S. Government agency securities classified as short-term | 2,994 | 4 | — | 2,998 | ||||||||||||
investments | ||||||||||||||||
Total | $ | 328,390 | $ | 14 | $ | (108 | ) | $ | 328,296 | |||||||
31-Dec-14 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 8,627 | $ | — | $ | — | $ | 8,627 | ||||||||
Corporate bonds classified as cash equivalents | 3,806 | 1 | — | 3,807 | ||||||||||||
Corporate bonds classified as short-term investments | 149,303 | 4 | (172 | ) | 149,135 | |||||||||||
Asset backed securities classified as short-term investments | 9,546 | — | (4 | ) | 9,542 | |||||||||||
U.S. Government agency securities classified as short-term | 4,488 | 1 | (4 | ) | 4,485 | |||||||||||
investments | ||||||||||||||||
Other classified as cash equivalents | 209 | — | — | 209 | ||||||||||||
Total | $ | 175,979 | $ | 6 | $ | (180 | ) | $ | 175,805 | |||||||
Accrued Liabilities | Accrued liabilities consist of the following (in thousands): | |||||||||||||||
March 31, | December 31, | |||||||||||||||
2015 | 2014 | |||||||||||||||
Research and clinical study expenses | $ | 4,271 | $ | 2,703 | ||||||||||||
Payroll and related expenses | 3,217 | 4,205 | ||||||||||||||
Other | 910 | 667 | ||||||||||||||
Total accrued liabilities | $ | 8,398 | $ | 7,575 | ||||||||||||
Common_Stock_Warrants_Tables
Common Stock Warrants (Tables) | 3 Months Ended | ||||||||||||||
Mar. 31, 2015 | |||||||||||||||
Warrants And Rights Note Disclosure [Abstract] | |||||||||||||||
Summary of Outstanding Warrants | The table sets forth the outstanding common stock warrants for the periods presented: | ||||||||||||||
Outstanding at March 31, | Outstanding at December 31, | Date Issued | Term | Exercise Price | |||||||||||
2015 | 2014 | ||||||||||||||
83,167 | 83,167 | Jun-10 | 10 years | $ | 3.006 | ||||||||||
174,651 | 174,651 | Feb-11 | 10 years | 3.006 | |||||||||||
66,533 | 66,533 | Jun-11 | 10 years | 3.006 | |||||||||||
324,351 | 324,351 | ||||||||||||||
StockBased_Compensation_Tables
Stock-Based Compensation (Tables) | 3 Months Ended | ||||||||
Mar. 31, 2015 | |||||||||
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |||||||||
Functional Classification of Stock-Based Compensation Expense, Net of Estimated Forfeitures | The table below sets forth the functional classification of stock-based compensation expense, net of estimated forfeitures, for the periods presented (in thousands): | ||||||||
Three Months Ended March 31, | |||||||||
2015 | 2014 | ||||||||
Research and development | $ | 1,841 | $ | 705 | |||||
General and administrative | 567 | 90 | |||||||
Total stock-based compensation | $ | 2,408 | $ | 795 | |||||
Net_Loss_per_Share_Attributabl1
Net Loss per Share Attributable to Common Stockholders (Tables) | 3 Months Ended | |||||||
Mar. 31, 2015 | ||||||||
Earnings Per Share [Abstract] | ||||||||
Computation of Basic and Diluted Net Loss per Share | The following table sets forth the computation of the basic and diluted net loss per share attributable to common stockholders (in thousands, except share and per share data): | |||||||
Three Months Ended | ||||||||
March 31, | ||||||||
2015 | 2014 | |||||||
Numerator: | ||||||||
Net loss | $ | (21,379 | ) | $ | (13,630 | ) | ||
Accretion and dividends on convertible preferred stock | — | (4,808 | ) | |||||
Net loss attributable to common stockholders | $ | (21,379 | ) | $ | (18,438 | ) | ||
Denominator: | ||||||||
Weighted-average common shares outstanding | 34,008,830 | 21,582,435 | ||||||
Less: weighted-average unvested common shares subject to repurchase | — | — | ||||||
Weighted-average shares used to compute net loss per share attributable | 34,008,830 | 21,582,435 | ||||||
to common stockholders, basic and diluted | ||||||||
Net loss per share attributable to common stockholders, basic and diluted | $ | (0.63 | ) | $ | (0.85 | ) | ||
Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share | The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been antidilutive: | |||||||
Three Months Ended March 31, | ||||||||
2015 | 2014 | |||||||
Convertible preferred stock (as if converted) | — | 6,532,828 | ||||||
Stock options to purchase common stock | 2,698,846 | 2,315,345 | ||||||
Unvested restricted stock units | 33,856 | — | ||||||
Convertible preferred stock warrants (as if converted) | — | 117,820 | ||||||
Common stock warrants | 324,351 | 235,639 | ||||||
3,057,053 | 9,201,632 | |||||||
Organization_Additional_Inform
Organization - Additional Information (Details) (USD $) | 1 Months Ended | 3 Months Ended | |
In Millions, except Share data, unless otherwise specified | Feb. 28, 2015 | Mar. 31, 2015 | Dec. 31, 2014 |
Segment | |||
Organization And Nature Of Business [Line Items] | |||
Common stock, shares issued | 3,450,000 | 35,612,019 | 31,934,682 |
Purchase of additional shares by underwriters | 450,000 | ||
Common stock issued price per share | $54 | ||
Proceeds from public offering before offering expenses | $175.10 | ||
Underwriting discounts and commissions | 11.2 | ||
Offering expenses | 0.6 | ||
Proceeds from public offering, net | $174.50 | ||
United States of America | |||
Organization And Nature Of Business [Line Items] | |||
Number of reportable segments | 1 |
Summary_of_Significant_Account2
Summary of Significant Accounting Policies - Additional Information (Details) (USD $) | 3 Months Ended |
Mar. 31, 2015 | |
Accounting Policies [Abstract] | |
Unrecognized tax benefits related to interest or penalties | $0 |
Fair_Value_Measurements_Summar
Fair Value Measurements - Summary of Financial Assets and Liabilities Measured on Recurring Basis (Details) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
Financial Assets: | ||
Fair value | $328,296 | $175,805 |
Money Market Funds | ||
Financial Assets: | ||
Fair value | 142,829 | 8,627 |
Asset Backed Securities | ||
Financial Assets: | ||
Fair value | 9,520 | 9,542 |
Other | ||
Financial Assets: | ||
Fair value | 209 | |
Level 1 | ||
Financial Assets: | ||
Fair value | 142,829 | 8,627 |
Level 1 | Money Market Funds | ||
Financial Assets: | ||
Fair value | 142,829 | 8,627 |
Level 2 | ||
Financial Assets: | ||
Fair value | 185,467 | 167,178 |
Level 2 | Asset Backed Securities | ||
Financial Assets: | ||
Fair value | 9,520 | 9,542 |
Level 2 | Other | ||
Financial Assets: | ||
Fair value | 209 | |
Corporate Bonds | ||
Financial Assets: | ||
Fair value | 170,452 | 152,942 |
Corporate Bonds | Level 2 | ||
Financial Assets: | ||
Fair value | 170,452 | 152,942 |
US Government Corporations and Agencies Securities | ||
Financial Assets: | ||
Fair value | 2,998 | 4,485 |
US Government Corporations and Agencies Securities | Level 2 | ||
Financial Assets: | ||
Fair value | 2,998 | 4,485 |
Commercial Paper | ||
Financial Assets: | ||
Fair value | 2,497 | |
Commercial Paper | Level 2 | ||
Financial Assets: | ||
Fair value | $2,497 |
Fair_Value_Measurements_Additi
Fair Value Measurements - Additional Information (Details) (USD $) | 3 Months Ended | 0 Months Ended | |
Mar. 31, 2015 | Mar. 31, 2014 | Jan. 30, 2014 | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis Valuation Techniques [Line Items] | |||
Revaluation of convertible preferred stock warrant liability | $0 | $3,324,000 | |
Black-Scholes Option Pricing Model | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis Valuation Techniques [Line Items] | |||
Estimated fair value of common stock, per share | $21 | ||
Expected time | 7 years | ||
Expected risk free interest rate | 2.19% | ||
Expected volatility | 70.00% |
Fair_Value_Measurements_Summar1
Fair Value Measurements - Summary of Changes in Estimated Fair Value (Details) (USD $) | 3 Months Ended |
In Thousands, unless otherwise specified | Mar. 31, 2014 |
Fair Value Disclosures [Abstract] | |
Fair value, beginning of period | $3,419 |
Change in fair value recorded as a loss in other expense, net | 3,324 |
Reclassification of warrant liability to additional paid-in capital | ($6,743) |
Balance_Sheet_Components_Summa
Balance Sheet Components - Summary of Fair Values of Cash Equivalents and Short-Term Investments Classified as Available-for-Sale Securities (Details) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $328,390 | $175,979 |
Gross Unrealized Gains | 14 | 6 |
Gross Unrealized Losses | -108 | -180 |
Estimated Fair Value | 328,296 | 175,805 |
Money Market Funds Classified as Cash Equivalents | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 142,829 | 8,627 |
Estimated Fair Value | 142,829 | 8,627 |
Corporate Bonds Classified as Cash Equivalents | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 20,941 | 3,806 |
Gross Unrealized Gains | 1 | |
Estimated Fair Value | 20,941 | 3,807 |
Commercial Paper Classified as Short-Term Investments | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 2,497 | |
Gross Unrealized Gains | 2 | |
Gross Unrealized Losses | -2 | |
Estimated Fair Value | 2,497 | |
Corporate Bonds Classified as Short-Term Investments | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 149,607 | 149,303 |
Gross Unrealized Gains | 8 | 4 |
Gross Unrealized Losses | -104 | -172 |
Estimated Fair Value | 149,511 | 149,135 |
Asset Backed Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 9,522 | 9,546 |
Gross Unrealized Losses | -2 | -4 |
Estimated Fair Value | 9,520 | 9,542 |
US Government Corporations and Agencies Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 2,994 | 4,488 |
Gross Unrealized Gains | 4 | 1 |
Gross Unrealized Losses | -4 | |
Estimated Fair Value | 2,998 | 4,485 |
Other Classified as Cash Equivalents | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 209 | |
Estimated Fair Value | $209 |
Balance_Sheet_Components_Addit
Balance Sheet Components - Additional Information (Details) (USD $) | 3 Months Ended |
Mar. 31, 2015 | |
Schedule Of Available For Sale Securities [Line Items] | |
Significant realized gains or losses on available-for-sale securities | $0 |
Maximum | |
Schedule Of Available For Sale Securities [Line Items] | |
Available-for-sale securities remaining contractual maturities | 2 years |
Balance_Sheet_Components_Accru
Balance Sheet Components - Accrued Liabilities (Details) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
Payables And Accruals [Abstract] | ||
Research and clinical study expenses | $4,271 | $2,703 |
Payroll and related expenses | 3,217 | 4,205 |
Other | 910 | 667 |
Total accrued liabilities | $8,398 | $7,575 |
License_and_Research_Agreement1
License and Research Agreements - Additional Information (Details) | Mar. 31, 2015 | Feb. 28, 2015 | Dec. 31, 2014 | Sep. 30, 2010 | Sep. 30, 2010 | Mar. 31, 2015 | Mar. 31, 2015 | Nov. 30, 2010 | Mar. 31, 2015 | Dec. 31, 2012 | Mar. 31, 2015 | Mar. 31, 2015 | Dec. 31, 2012 | Mar. 31, 2015 | Jun. 30, 2013 | Dec. 31, 2012 | Mar. 31, 2015 | Mar. 31, 2015 | Mar. 31, 2015 | Mar. 31, 2014 |
USD ($) | USD ($) | Nobelpharma License Agreement | Nobelpharma License Agreement | Nobelpharma License Agreement | Nobelpharma License Agreement | Saint Louis University License Agreement | Saint Louis University License Agreement | H I B M Research Group License Agreement | H I B M Research Group License Agreement | H I B M Research Group License Agreement | St. Jude Children's Research Hospital License Agreement | St. Jude Children's Research Hospital License Agreement | Baylor Research Institute License Agreement | Baylor Research Institute License Agreement | Baylor Research Institute License Agreement | Baylor Research Institute License Agreement | Kyowa Hakko Kirin Collaboration | Kyowa Hakko Kirin Collaboration | ||
USD ($) | JPY (¥) | USD ($) | JPY (¥) | USD ($) | USD ($) | USD ($) | Maximum | Development and Approval Milestones | USD ($) | Maximum | USD ($) | USD ($) | Development Milestones | Sales Milestones | License Agreement | License Agreement | ||||
USD ($) | USD ($) | USD ($) | USD ($) | USD ($) | ||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||
License agreement | $111,000 | ¥ 10,000,000 | $495,000 | $10,000 | $100,000 | $25,000 | $10,000 | $250,000 | $1,400,000 | $710,000 | ||||||||||
Common stock, shares issued | 35,612,019 | 3,450,000 | 31,934,682 | 76,567 | 76,567 | |||||||||||||||
Remaining total aggregate payments | 1,700,000 | 200,000,000 | ||||||||||||||||||
Future contingent milestone payments | 300,000 | 10,500,000 | 7,500,000 | |||||||||||||||||
Payments for royalty on net sales | 1.00% | 1.00% | ||||||||||||||||||
Payment of option exercise fee | 750,000 | |||||||||||||||||||
License agreement receivable | 1,500,000 | 878,000 | ||||||||||||||||||
Accrued liabilities | $8,398,000 | $7,575,000 | $25,000 | $201,000 |
Common_Stock_Warrants_Summary_
Common Stock Warrants - Summary of Outstanding Warrants (Details) (Common Stock [Member], USD $) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2015 | Dec. 31, 2014 | |
Class Of Warrant Or Right [Line Items] | ||
Number of Warrants Outstanding | 324,351 | 324,351 |
June 2010 Common Stock Warrant [Member] | ||
Class Of Warrant Or Right [Line Items] | ||
Number of Warrants Outstanding | 83,167 | 83,167 |
Date Issued | 2010-06 | 2010-06 |
Term | 10 years | 10 years |
Exercise Price | $3.01 | $3.01 |
February 2011 Common Stock Warrant [Member] | ||
Class Of Warrant Or Right [Line Items] | ||
Number of Warrants Outstanding | 174,651 | 174,651 |
Date Issued | 2011-02 | 2011-02 |
Term | 10 years | 10 years |
Exercise Price | $3.01 | $3.01 |
June 2011 Common Stock Warrant [Member] | ||
Class Of Warrant Or Right [Line Items] | ||
Number of Warrants Outstanding | 66,533 | 66,533 |
Date Issued | 2011-06 | 2011-06 |
Term | 10 years | 10 years |
Exercise Price | $3.01 | $3.01 |
StockBased_Compensation_Additi
Stock-Based Compensation - Additional Information (Details) (2014 Incentive Plan) | 3 Months Ended |
Mar. 31, 2015 | |
2014 Incentive Plan | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Share reserved for future issuance | 2,595,430 |
Automatic increases in shares available for grant effective date | 1-Jan-15 |
Shares available for grant, ending date | 1-Jan-24 |
StockBased_Compensation_Functi
Stock-Based Compensation - Functional Classification of Stock-Based Compensation Expense, Net of Estimated Forfeitures (Details) (USD $) | 3 Months Ended | |
In Thousands, unless otherwise specified | Mar. 31, 2015 | Mar. 31, 2014 |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Stock-based compensation expense | $2,408 | $795 |
Research and development | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Stock-based compensation expense | 1,841 | 705 |
General and administrative | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Stock-based compensation expense | $567 | $90 |
Defined_Contribution_Plan_Addi
Defined Contribution Plan - Additional Information (Details) (USD $) | 3 Months Ended |
Mar. 31, 2015 | |
Defined Contribution Pension And Other Postretirement Plans Disclosure [Abstract] | |
Contribution expenses | $123,000 |
Net_Loss_per_Share_Attributabl2
Net Loss per Share Attributable to Common Stockholders - Computation of Basic and Diluted Net Loss per Share (Details) (USD $) | 3 Months Ended | |
In Thousands, except Share data, unless otherwise specified | Mar. 31, 2015 | Mar. 31, 2014 |
Numerator: | ||
Net loss | ($21,379) | ($13,630) |
Accretion and dividends on convertible preferred stock | -4,808 | |
Net loss attributable to common stockholders | ($21,379) | ($18,438) |
Denominator: | ||
Weighted-average common shares outstanding | 34,008,830 | 21,582,435 |
Weighted-average shares used to compute net loss per share attributable to common stockholders, basic and diluted | 34,008,830 | 21,582,435 |
Net loss per share attributable to common stockholders, basic and diluted | ($0.63) | ($0.85) |
Net_Loss_per_Share_Attributabl3
Net Loss per Share Attributable to Common Stockholders - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details) | 3 Months Ended | |
Mar. 31, 2015 | Mar. 31, 2014 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 3,057,053 | 9,201,632 |
Convertible Preferred Stock | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 6,532,828 | |
Stock options to purchase common stock | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 2,698,846 | 2,315,345 |
Convertible Preferred Stock Warrants (as if converted) | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 117,820 | |
Unvested Restricted Stock Units | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 33,856 | |
Common Stock Warrants | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common stock equivalents excluded from computation of diluted net loss per share | 324,351 | 235,639 |