Document_and_Entity_Informatio
Document and Entity Information | 6 Months Ended | |
Jun. 30, 2014 | Aug. 06, 2014 | |
Document And Entity Information [Line Items] | ' | ' |
Document Type | '10-Q | ' |
Amendment Flag | 'false | ' |
Document Period End Date | 30-Jun-14 | ' |
Document Fiscal Year Focus | '2014 | ' |
Document Fiscal Period Focus | 'Q2 | ' |
Trading Symbol | 'RARE | ' |
Entity Registrant Name | 'Ultragenyx Pharmaceutical Inc. | ' |
Entity Central Index Key | '0001515673 | ' |
Current Fiscal Year End Date | '--12-31 | ' |
Entity Filer Category | 'Non-accelerated Filer | ' |
Entity Common Stock, Shares Outstanding | ' | 31,703,990 |
CONDENSED_BALANCE_SHEETS
CONDENSED BALANCE SHEETS (USD $) | Jun. 30, 2014 | Dec. 31, 2013 |
In Thousands, unless otherwise specified | ||
Current assets: | ' | ' |
Cash and cash equivalents | $17,452 | $7,427 |
Short-term investments | 135,819 | 45,950 |
Prepaid expenses and other current assets | 4,945 | 1,848 |
Total current assets | 158,216 | 55,225 |
Property and equipment, net | 2,497 | 1,325 |
Restricted cash | 744 | 451 |
Other assets | 1,129 | 2,648 |
Total assets | 162,586 | 59,649 |
Liabilities, Convertible Preferred Stock and Stockholders’ Equity (Deficit) | ' | ' |
Accounts payable | 3,590 | 1,437 |
Accrued liabilities | 5,278 | 4,406 |
Deferred rent—current portion | 75 | 78 |
Total current liabilities | 8,943 | 5,921 |
Convertible preferred stock warrant liability | ' | 3,419 |
Other liabilities | 560 | 200 |
Total liabilities | 9,503 | 9,540 |
Commitments and contingencies (Note 10) | ' | ' |
Stockholders’ equity (deficit): | ' | ' |
Preferred stock, par value of $0.001 per share—25,000,000 shares authorized; nil outstanding as of June 30, 2014 and December 31, 2013 | ' | ' |
Common stock, par value of $0.001 per share—250,000,000 shares authorized; 30,059,288 and 3,766,289 shares issued and outstanding as of June 30, 2014 and December 31, 2013 | 30 | 4 |
Additional paid-in capital | 259,555 | ' |
Accumulated other comprehensive income (loss) | -48 | 11 |
Accumulated deficit | -106,454 | -74,836 |
Total stockholders’ equity (deficit) | 153,083 | -74,821 |
Total liabilities, convertible preferred stock and stockholders’ equity (deficit) | 162,586 | 59,649 |
Series A Convertible Preferred Stock | Redeemable Convertible Preferred Stock | ' | ' |
Liabilities, Convertible Preferred Stock and Stockholders’ Equity (Deficit) | ' | ' |
Convertible preferred stock | ' | 51,001 |
Series B Convertible Preferred Stock | Convertible Preferred Stock | ' | ' |
Liabilities, Convertible Preferred Stock and Stockholders’ Equity (Deficit) | ' | ' |
Convertible preferred stock | ' | $73,929 |
CONDENSED_BALANCE_SHEETS_Paren
CONDENSED BALANCE SHEETS (Parenthetical) (USD $) | Jun. 30, 2014 | Dec. 31, 2013 |
Preferred stock, par value | $0.00 | $0.00 |
Preferred stock, shares authorized | 25,000,000 | 25,000,000 |
Preferred stock, shares issued | 0 | 0 |
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 | 30,059,288 | 3,766,289 |
Common stock, shares outstanding | 30,059,288 | 3,766,289 |
Series A Convertible Preferred Stock | Redeemable Convertible Preferred Stock | ' | ' |
Convertible preferred stock, par value | $0.00 | $0.00 |
Convertible preferred stock, shares authorized | 0 | 35,377,566 |
Convertible preferred stock, shares issued | 0 | 34,349,894 |
Convertible preferred stock, shares outstanding | 0 | 34,349,894 |
Series B Convertible Preferred Stock | Convertible Preferred Stock | ' | ' |
Convertible preferred stock, par value | $0.00 | $0.00 |
Convertible preferred stock, shares authorized | 0 | 27,081,680 |
Convertible preferred stock, shares issued | 0 | 27,081,680 |
Convertible preferred stock, shares outstanding | 0 | 27,081,680 |
CONDENSED_STATEMENTS_OF_OPERAT
CONDENSED STATEMENTS OF OPERATIONS (USD $) | 3 Months Ended | 6 Months Ended | ||
In Thousands, except Share data, unless otherwise specified | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 |
Operating expenses: | ' | ' | ' | ' |
Research and development | $11,239 | $7,199 | $19,592 | $12,863 |
General and administrative | 2,422 | 1,048 | 4,408 | 2,131 |
Total operating expenses | 13,661 | 8,247 | 24,000 | 14,994 |
Loss from operations | -13,661 | -8,247 | -24,000 | -14,994 |
Other income (expense), net: | ' | ' | ' | ' |
Interest income | 149 | 63 | 242 | 89 |
Other expense, net | -73 | -406 | -3,457 | -420 |
Total other income (expense), net | 76 | -343 | -3,215 | -331 |
Net loss | -13,585 | -8,590 | -27,215 | -15,325 |
Net loss attributable to common stockholders | ($13,585) | ($10,829) | ($32,023) | ($19,034) |
Net loss per share attributable to common stockholders, basic and diluted | ($0.45) | ($3.32) | ($1.25) | ($6.19) |
Shares used in computing net loss per share attributable to common stockholders, basic and diluted | 30,055,943 | 3,257,806 | 25,697,407 | 3,076,907 |
CONDENSED_STATEMENTS_OF_COMPRE
CONDENSED STATEMENTS OF COMPREHENSIVE LOSS (USD $) | 3 Months Ended | 6 Months Ended | ||
In Thousands, unless otherwise specified | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 |
Net loss | ($13,585) | ($8,590) | ($27,215) | ($15,325) |
Other comprehensive income: | ' | ' | ' | ' |
Unrealized loss on available-for-sale securities | -2 | -66 | -59 | -77 |
Total comprehensive loss | ($13,587) | ($8,656) | ($27,274) | ($15,402) |
CONDENSED_STATEMENTS_OF_CASH_F
CONDENSED STATEMENTS OF CASH FLOWS (USD $) | 6 Months Ended | |
Jun. 30, 2014 | Jun. 30, 2013 | |
Operating activities: | ' | ' |
Net loss | ($27,215,000) | ($15,325,000) |
Adjustments to reconcile net loss to net cash used in operating activities: | ' | ' |
Depreciation and amortization | 278,000 | 227,000 |
Amortization of premium (discount) on investment securities | 1,396,000 | 365,000 |
Stock-based compensation | 1,741,000 | 376,000 |
Revaluation of convertible preferred stock warrant liability | 3,324,000 | 372,000 |
Changes in operating assets and liabilities: | ' | ' |
Prepaid expenses and other current assets | -3,097,000 | -1,288,000 |
Other assets | 1,519,000 | -129,000 |
Accounts payable | 1,960,000 | 886,000 |
Accrued liabilities and other liabilities | 1,229,000 | -164,000 |
Net cash used in operating activities | -18,865,000 | -14,680,000 |
Investing activities: | ' | ' |
Purchase of property and equipment | -1,257,000 | -75,000 |
Purchase of investments | -129,298,000 | -57,935,000 |
Proceeds from maturities of investments | 37,974,000 | ' |
Increase in restricted cash | -293,000 | -50,000 |
Net cash used in investing activities | -92,874,000 | -58,060,000 |
Financing activities: | ' | ' |
Net proceeds from issuance of common stock | 126,110,000 | 57,000 |
Payment of preferred stock dividend | -4,346,000 | ' |
Net cash provided by financing activities | 121,764,000 | 57,000 |
Net increase (decrease) in cash and cash equivalents | 10,025,000 | -72,683,000 |
Cash and cash equivalents at beginning of period | 7,427,000 | 86,190,000 |
Cash and cash equivalents at end of period | 17,452,000 | 13,507,000 |
Supplemental disclosures of non-cash investing and financing information: | ' | ' |
Reclassification of warrant liability to equity upon conversion to common stock warrants | 6,743,000 | ' |
Series A and Series B | ' | ' |
Supplemental disclosures of non-cash investing and financing information: | ' | ' |
Conversion of Series A and Series B preferred stock to common stock | $129,360,000 | ' |
Organization
Organization | 6 Months Ended | |
Jun. 30, 2014 | ||
Organization | ' | |
1 | Organization | |
Ultragenyx Pharmaceutical Inc. (the Company) is a clinical-stage biotechnology 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 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 1/2 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 for the treatment of 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 a Phase 2 study of KRN23, an antibody targeting fibroblast growth factor 23, or FGF23, in pediatric patients with X-linked hypophosphatemia (XLH), a rare genetic disease that impairs bone growth. | ||
On January 30, 2014, the Company’s registration statements on Form S-1 (File Nos. 333-192244 and 333-193675) relating to its initial public offering (IPO) of its common stock were declared effective by the Securities and Exchange Commission (SEC). The shares began trading on The NASDAQ Global Select Market on January 31, 2014. The public offering price of the shares sold in the offering was $21.00 per share. The IPO closed on February 5, 2014 and included 6,624,423 shares of common stock, which included 864,054 shares of common stock issued pursuant to the over-allotment option granted to the underwriters. The Company received total proceeds from the offering of $129.4 million, net of underwriting discounts and commissions of $9.7 million. After deducting offering expenses of approximately $3.3 million and a cash dividend of $4.3 million, which was paid to the preferred stockholders on the closing date, net proceeds were approximately $121.7 million. Upon the closing of the IPO, all shares of convertible preferred stock then outstanding converted into 19,598,486 shares of common stock and the Series A convertible preferred stock warrants were converted into warrants to purchase common stock. | ||
Upon the effectiveness of the Amended and Restated Certificate of Incorporation of the Company on February 5, 2014, the number of shares of capital stock the Company is authorized to issue was increased to 275,000,000 shares, of which 250,000,000 shares are common stock and 25,000,000 shares are preferred stock. Both the common stock and preferred stock have a par value of $0.001 per share. There are no shares of preferred stock outstanding at June 30, 2014. |
Summary_of_Significant_Account
Summary of Significant Accounting Policies | 6 Months Ended | |
Jun. 30, 2014 | ||
Summary of Significant Accounting Policies | ' | |
2 | Summary of Significant Accounting Policies | |
Basis of Presentation | ||
The accompanying unaudited condensed consolidated financial statements 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 24, 2014 with the SEC. | ||
The results of operations for the three and six months ended June 30, 2014 are not necessarily indicative of the results to be expected for the year ending December 31, 2014. The condensed balance sheet as of December 31, 2013 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. | ||
Reverse Stock Split | ||
In January 2014, the Company’s board of directors and its stockholders approved an amendment to the Company’s amended and restated certificate of incorporation to effect a reverse split of shares of the Company’s common stock on a 1-for-3.1345 basis (the “Reverse Stock Split”). The par values and the authorized shares of the common and convertible preferred stock were not adjusted as a result of the Reverse Stock Split, nor were the outstanding shares of preferred stock. All issued and outstanding common stock and related per share amounts contained in the financial statements have been retroactively adjusted to reflect this Reverse Stock Split for all periods presented. A proportional adjustment to the conversion ratio for each series of convertible preferred stock was also effected in connection with the Reverse Stock Split. The Reverse Stock Split was effected on January 17, 2014. | ||
Use of Estimates | ||
The preparation of condensed 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. | ||
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, respectively. The cost of securities sold is based on the specific-identification method. Interest on marketable securities is included in interest income. | ||
Deferred Offering Costs | ||
Deferred offering costs, which primarily consist of direct incremental accounting, legal and printing fees relating to the IPO and to the Company’s underwritten public offering in July 2014, were initially capitalized. IPO deferred offering costs of $3.3 million were subsequently offset against IPO proceeds upon the completion of the IPO in February 2014. Underwritten public offering costs of $444,000 were subsequently offset against offering proceeds on completion of our underwritten public offering in July 2014. As of June 30, 2014 and December 31, 2013, deferred offering costs of $316,000 and $2.3 million, respectively, were capitalized and included in prepaid and other current assets on the balance sheet. | ||
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 marketable securities. 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 and corporate bond issuers 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. | ||
Recently Issued Accounting Pronouncements | ||
In June 2014, the Financial Accounting Standards Board issued Accounting Standards Update 2014-10, Development Stage Enterprises: Elimination of Certain Financial Reporting Requirements, Including an Amendment to Variable Interest Entities Guidance in Topic 810, Consolidation (ASU 2014-10). ASU 2014-10 removes the definition of a development stage entity from the Master Glossary of the Accounting Standards Codification, thereby removing the financial reporting distinction between development stage entities and other reporting entities from U.S. GAAP. ASU 2014-10 also eliminates the requirements for development stage entities to (1) present inception-to-date information in the statements of income, cash flows, and shareholder equity, (2) label the financial statements as those of a development stage entity, (3) disclose a description of the development stage activities in which the entity is engaged, and (4) disclose in the first year in which the entity is no longer a development stage entity that in prior years it had been in the development stage. The amendments also clarify that the guidance in Topic 275, Risks and Uncertainties, is applicable to entities that have not commenced planned principal operations. The amendments in ASU 2014-10 will be effective retrospectively except for the clarification to Topic 275, which shall be applied prospectively for annual reporting periods beginning after December 15, 2014, and interim periods therein. Early application of each of the amendments is permitted for any annual reporting period or interim period for which the entity’s financial statements have not yet been issued. The Company elected to early adopt the provisions of ASU 2014-10 in the second quarter of 2014, as reflected in this Quarterly Report. | ||
Fair_Value_Measurements
Fair Value Measurements | 6 Months Ended | |||||||||||||||
Jun. 30, 2014 | ||||||||||||||||
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 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): | ||||||||||||||||
30-Jun-14 | ||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 6,753 | $ | — | $ | — | $ | 6,753 | ||||||||
Commercial paper | — | 7,988 | — | 7,988 | ||||||||||||
Corporate bonds | — | 125,840 | — | 125,840 | ||||||||||||
U.S. Government securities | — | 3,002 | — | 3,002 | ||||||||||||
Total financial assets | $ | 6,753 | $ | 136,830 | $ | — | $ | 143,583 | ||||||||
December 31, 2013 | ||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 6,847 | $ | — | $ | — | $ | 6,847 | ||||||||
Commercial paper | — | 1,000 | — | 1,000 | ||||||||||||
Corporate bonds | — | 44,950 | — | 44,950 | ||||||||||||
Total financial assets | $ | 6,847 | $ | 45,950 | $ | — | $ | 52,797 | ||||||||
Financial Liabilities: | ||||||||||||||||
Convertible preferred stock warrant liability | $ | — | $ | — | $ | 3,419 | $ | 3,419 | ||||||||
Total financial liabilities | $ | — | $ | — | $ | 3,419 | $ | 3,419 | ||||||||
The convertible preferred stock warrant liability was classified as a Level 3 liability. As of December 31, 2013, the Company determined the estimated fair value of the warrants using an option-pricing method to allocate the equity value of the Company to the warrants based on the Company’s capital structure. The equity value was estimated using the back-solve method, whereby the equity value was derived from a recent transaction involving the Company’s own securities. The key inputs used to determine value of the warrants was an estimated fair value of the Company’s common stock of $12.14 per share, expected volatility of 70%, the expected time to liquidity event of 0.43 years and risk-free interest rate of 0.11%. The significant unobservable input used in the fair value measurement of the convertible preferred stock warrant liability was the equity value of the Company. 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. | ||||||||||||||||
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%. 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): | ||||||||||||||||
Six Months Ended June 30, | ||||||||||||||||
2014 | 2013 | |||||||||||||||
Fair value, beginning of period | $ | 3,419 | $ | 518 | ||||||||||||
Change in fair value recorded as a loss in other expense, net | 3,324 | 372 | ||||||||||||||
Reclassification of warrant liability to additional paid-in capital | (6,743 | ) | — | |||||||||||||
Fair value, end of period | $ | — | $ | 890 | ||||||||||||
Balance_Sheet_Components
Balance Sheet Components | 6 Months Ended | |||||||||||||||
Jun. 30, 2014 | ||||||||||||||||
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: | ||||||||||||||||
30-Jun-14 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 6,753 | $ | — | $ | — | $ | 6,753 | ||||||||
Corporate bonds classified as cash equivalents | 1,011 | — | — | 1,011 | ||||||||||||
Commercial Paper classified as short-term investments | 7,988 | — | — | 7,988 | ||||||||||||
Corporate bonds classified as short-term investments | 124,883 | 20 | (74 | ) | 124,829 | |||||||||||
U.S Government securities classified as short-term investments | 2,996 | 6 | — | 3,002 | ||||||||||||
Total | $ | 143,631 | $ | 26 | $ | (74 | ) | $ | 143,583 | |||||||
December 31, 2013 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 6,847 | $ | — | $ | — | $ | 6,847 | ||||||||
Commercial Paper classified as short-term investments | 1,000 | — | — | 1,000 | ||||||||||||
Corporate bonds classified as short-term investments | 44,939 | 17 | (6 | ) | 44,950 | |||||||||||
Total | $ | 52,786 | $ | 17 | $ | (6 | ) | $ | 52,797 | |||||||
At June 30, 2014, 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): | ||||||||||||||||
June 30, | December 31, | |||||||||||||||
2014 | 2013 | |||||||||||||||
Research and clinical study expenses | $ | 2,852 | $ | 1,886 | ||||||||||||
Payroll and related expenses | 2,208 | 2,360 | ||||||||||||||
Other | 218 | 160 | ||||||||||||||
Total accrued liabilities | $ | 5,278 | $ | 4,406 | ||||||||||||
License_and_Research_Agreement
License and Research Agreements | 6 Months Ended | |
Jun. 30, 2014 | ||
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 $110,500 (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 June 30, 2014. The remaining total aggregate payments, if all milestones are achieved by Nobelpharma, would be 200 million Yen (approximately $2.0 million based on the exchange rate at June 30, 2014). 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. | ||
AAI Pharma License Agreement | ||
In March 2011, the Company entered into a license agreement with AAI Pharma Services Corp. (AAI Pharma). Under the terms of this license agreement, AAI Pharma 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 AAI Pharma’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 AAI Pharma 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 $1.3 million and $2.0 million for its share of the costs as research and development expenses for the three and six months ended June 30, 2014 respectively. | ||
Convertible_Preferred_Stock_Wa
Convertible Preferred Stock Warrants and Common Stock Warrants | 6 Months Ended | ||||||||||||
Jun. 30, 2014 | |||||||||||||
Convertible Preferred Stock Warrants and Common Stock Warrants | ' | ||||||||||||
6 | Convertible Preferred Stock Warrants and Common Stock Warrants | ||||||||||||
Upon the closing of the Company’s IPO, the Convertible Preferred Stock warrants were converted into warrants to purchase common stock. | |||||||||||||
As of June 30, 2014, outstanding common stock warrants consisted of the following: | |||||||||||||
Common Stock Warrants: | Number of Warrants | Date Issued | Term | Exercise Price | |||||||||
Common stock | 83,167 | Jun-10 | 10 years | $ | 3.006 | ||||||||
Common stock | 203,759 | Feb-11 | 10 years | 3.006 | |||||||||
Common stock | 66,533 | Jun-11 | 10 years | 3.006 | |||||||||
Total common stock warrants | 353,459 | ||||||||||||
As of December 31, 2013, outstanding preferred stock warrants consisted of the following: | |||||||||||||
Convertible Preferred Stock Warrants: | Number of Warrants | Date Issued | Term | Exercise Price | |||||||||
Series A | 241,803 | Jun-10 | 10 years | $ | 1.034 | ||||||||
Series A | 592,417 | Feb-11 | 10 years | 1.034 | |||||||||
Series A | 193,442 | Jun-11 | 10 years | 1.034 | |||||||||
Total convertible preferred stock warrants | 1,027,662 | ||||||||||||
The fair value of the warrants was estimated to be $6.7 million and $3.4 million as of January 30, 2014 (pricing date of IPO) and December 31, 2013, respectively. | |||||||||||||
The Company recorded $0 and ($374,000), to other income (expense) for three months ended June 30, 2014 and 2013 and ($3.3 million) and($372,000) for the six months ended June 30, 2014 and 2013, representing the change in fair value of the warrants for the respective period. |
Convertible_Preferred_Stock
Convertible Preferred Stock | 6 Months Ended | |
Jun. 30, 2014 | ||
Convertible Preferred Stock | ' | |
7 | Convertible Preferred Stock | |
The holders of the Series A and Series B convertible preferred stock were entitled to receive cumulative dividends at the rate of $0.062 per share per annum, payable in the form of cash. Dividends accrued from day to day, whether or not declared, but were paid only when, as, and if declared by the Board of Directors. During 2012, $2.1 million of dividends were declared and paid to holders of Series A convertible preferred stock in the form of additional Series A convertible preferred stock. Dividends in arrears as of December 31, 2013 were $4.0 million for both series of preferred stock. Upon the closing of the IPO in February 2014, all shares of convertible preferred stock then outstanding automatically converted in 19,598,486 shares of common stock. In connection with the conversion of the convertible preferred stock, all accrued and outstanding dividends in the amount of $4.3 million were paid. | ||
The Company initially recorded the Series A and Series B convertible preferred stock at their issuance price, which represents the carrying value. The Series A convertible preferred stock was redeemable at any time after June 16, 2017 once a written request to redeem such stock was received by the Company from holders of not less than seventy-five percent of the then outstanding Series A convertible preferred stock. As only the passage of time was required for the Series A convertible preferred stock to become redeemable, the difference in the initial carrying value of the Series A convertible preferred stock and their total redemption value was being accreted from the issuance date through the first redemption date of June 16, 2017. The Company recorded accretion of $0 and $1.8 million for the three months ended June 30, 2014 and 2013, respectively and $4.4 million and $2.9 million for the six months ended June 30, 2014 and 2013, respectively. As a result of the conversion of the preferred stock to common stock in connection with the Company’s IPO, the Company is no longer accreting the Series A convertible preferred stock to its previously calculated redemption value. |
StockBased_Compensation
Stock-Based Compensation | 6 Months Ended | ||||||||||||||||
Jun. 30, 2014 | |||||||||||||||||
Stock-Based Compensation | ' | ||||||||||||||||
8 | Stock-Based Compensation | ||||||||||||||||
2011 Equity Incentive Plan | |||||||||||||||||
In 2011, the Company adopted the 2011 Equity Incentive Plan (the 2011 Plan). The 2011 Plan provides for the granting of stock-based awards to employees, directors, and consultants under terms and provisions established by the Board of Directors. Under the terms of the 2011 Plan, options may be granted at an exercise price not less than fair market value. For employees holding more than 10% of the voting rights of all classes of stock, the exercise prices for incentive stock options must be at least 110% of fair market of the common stock on the grant date, as determined by the Board of Directors. The terms of options granted under the 2011 Plan may not exceed ten years. Options granted generally vest over a period of four years. Typically, the vesting schedule for option grants to newly hired employees provides that 1/4 of the grant vests upon the first anniversary of the employee’s date of hire, with the remainder of the shares vesting monthly thereafter at a rate of 1/48 of the total shares subject to the option. All other employee options typically vest in equal monthly installments over the four-year vesting schedule. In connection with the Company’s IPO, no further grants will be made under this plan and all remaining shares available for grant were transferred to the 2014 Incentive Plan. | |||||||||||||||||
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 had 2,250,000 shares of common stock available for future issuance at the time of its inception, which included 655,038 shares available under the 2011 Plan, which were transferred to the 2014 Plan upon adoption. The 2014 Plan provides for automatic annual increases in shares available for grant, beginning on January 1, 2015 through January 1, 2024. The 2014 Plan provides for the granting of stock-based awards to employees, directors, and consultants under similar terms, conditions and provisions as the 2011 Plan. | |||||||||||||||||
Founder’s Stock | |||||||||||||||||
In connection with the Series A preferred stock financing, the Company entered into a stock repurchase agreement with the founder on June 16, 2011, whereby 2,552,241 shares of common stock previously owned by the founder were subject to repurchase by the Company at the original issuance price in the event that the founder’s employment is terminated either voluntarily or involuntarily. The repurchase rights lapsed over a period of two years from June 16, 2011. The Company calculated the estimated fair value of these restricted shares at the time the restriction was added to the shares as $1,199,000 and recorded this amount as stock-based compensation ratably over the period that the repurchase rights lapsed. Stock-based compensation expense pertaining to the founder’s stock was $0, and $119,000 for the three months ended June 30, 2014 and 2013, respectively and $0 and $268,000 for the six months ended June 30, 2014 and 2013, respectively. | |||||||||||||||||
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 June 30, | Six Months Ended June 30, | ||||||||||||||||
2014 | 2013 | 2014 | 2013 | ||||||||||||||
Research and development | $ | 652 | $ | 52 | $ | 1,357 | $ | 82 | |||||||||
General and administrative | 294 | 133 | 384 | 294 | |||||||||||||
Total stock-based compensation | $ | 946 | $ | 185 | $ | 1,741 | $ | 376 | |||||||||
Defined_Contribution_Plan
Defined Contribution Plan | 6 Months Ended | |
Jun. 30, 2014 | ||
Defined Contribution Plan | ' | |
9 | 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. The Company has not provided any contributions to the plan since its inception through June 30, 2014. |
Commitment_and_Contingencies
Commitment and Contingencies | 6 Months Ended | |
Jun. 30, 2014 | ||
Commitments and Contingencies | ' | |
10 | 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. As of June 30, 2014, the Company had a binding obligation for approximately $780,000 with a manufacturing vendor for the production of a drug substance for one of its product candidates. All other significant contracts as of June 30, 2014 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 balance sheet. | ||
Net_Loss_per_Share_Attributabl
Net Loss per Share Attributable to Common Stockholders | 6 Months Ended | |||||||||||||||
Jun. 30, 2014 | ||||||||||||||||
Net Loss per Share Attributable to Common Stockholders | ' | |||||||||||||||
11 | 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 June 30, | Six Months Ended June 30, | |||||||||||||||
2014 | 2013 | 2014 | 2013 | |||||||||||||
Numerator: | ||||||||||||||||
Net loss | $ | (13,585 | ) | $ | (8,590 | ) | $ | (27,215 | ) | $ | (15,325 | ) | ||||
Accretion and dividends on convertible preferred stock | - | (2,239 | ) | (4,808 | ) | (3,709 | ) | |||||||||
Net loss attributable to common stockholders | $ | (13,585 | ) | $ | (10,829 | ) | $ | (32,023 | ) | $ | (19,034 | ) | ||||
Denominator: | ||||||||||||||||
Weighted-average common shares outstanding | 30,055,943 | 3,503,214 | 25,697,407 | 3,482,304 | ||||||||||||
Less: weighted-average unvested common shares subject to repurchase | — | (245,408 | ) | — | (405,397 | ) | ||||||||||
Weighted-average shares used to compute net loss per share attributable | 30,055,943 | 3,257,806 | 25,697,407 | 3,076,907 | ||||||||||||
to common stockholders, basic and diluted | ||||||||||||||||
Net loss per share attributable to common stockholders, basic and diluted | $ | (0.45 | ) | $ | (3.32 | ) | $ | (1.25 | ) | $ | (6.19 | ) | ||||
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 June 30, | Six Months Ended June 30, | |||||||||||||||
2014 | 2013 | 2014 | 2013 | |||||||||||||
Convertible preferred stock | — | 19,598,486 | 3,248,368 | 19,598,486 | ||||||||||||
Stock options to purchase common stock | 2,561,357 | 1,647,753 | 2,438,351 | 1,543,954 | ||||||||||||
Common stock subject to repurchase | — | 245,408 | — | 405,397 | ||||||||||||
Warrants to purchase convertible preferred stock | — | 353,459 | 294,875 | 353,459 | ||||||||||||
(as if converted) | ||||||||||||||||
Warrants to purchase common stock | 353,459 | — | 58,584 | — | ||||||||||||
2,914,816 | 21,845,106 | 6,040,178 | 21,901,296 | |||||||||||||
Subsequent_Events
Subsequent Events | 6 Months Ended | |
Jun. 30, 2014 | ||
Subsequent Events | ' | |
12 | Subsequent Events | |
In July 2014, we completed an underwritten public offering in which we sold 1,613,879 shares of our common stock, which included 302,602 shares purchased by the underwriters pursuant to an option granted to them in connection with the offering, at a public offering price of $40.00 per share. In addition, certain existing stockholders sold 706,072 shares of common stock in the underwritten public offering at the same per-share price. The total proceeds the Company received from the offering were approximately $60.7 million, net of underwriting discounts and commissions of approximately $3.9 million. After deducting estimated offering expenses payable by us of approximately $0.4 million, net proceeds to us were $60.2 million. | ||
Summary_of_Significant_Account1
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2014 | |
Basis of Presentation | ' |
Basis of Presentation | |
The accompanying unaudited condensed consolidated financial statements 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 24, 2014 with the SEC. | |
The results of operations for the three and six months ended June 30, 2014 are not necessarily indicative of the results to be expected for the year ending December 31, 2014. The condensed balance sheet as of December 31, 2013 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. | |
Reverse Stock Split | ' |
Reverse Stock Split | |
In January 2014, the Company’s board of directors and its stockholders approved an amendment to the Company’s amended and restated certificate of incorporation to effect a reverse split of shares of the Company’s common stock on a 1-for-3.1345 basis (the “Reverse Stock Split”). The par values and the authorized shares of the common and convertible preferred stock were not adjusted as a result of the Reverse Stock Split, nor were the outstanding shares of preferred stock. All issued and outstanding common stock and related per share amounts contained in the financial statements have been retroactively adjusted to reflect this Reverse Stock Split for all periods presented. A proportional adjustment to the conversion ratio for each series of convertible preferred stock was also effected in connection with the Reverse Stock Split. The Reverse Stock Split was effected on January 17, 2014. | |
Use of Estimates | ' |
Use of Estimates | |
The preparation of condensed 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. | |
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, respectively. The cost of securities sold is based on the specific-identification method. Interest on marketable securities is included in interest income. | |
Deferred Offering Costs | ' |
Deferred Offering Costs | |
Deferred offering costs, which primarily consist of direct incremental accounting, legal and printing fees relating to the IPO and to the Company’s underwritten public offering in July 2014, were initially capitalized. IPO deferred offering costs of $3.3 million were subsequently offset against IPO proceeds upon the completion of the IPO in February 2014. Underwritten public offering costs of $444,000 were subsequently offset against offering proceeds on completion of our underwritten public offering in July 2014. As of June 30, 2014 and December 31, 2013, deferred offering costs of $316,000 and $2.3 million, respectively, were capitalized and included in prepaid and other current assets on the balance sheet. | |
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 marketable securities. 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 and corporate bond issuers 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. | |
Recently Issued Accounting Pronouncements | ' |
Recently Issued Accounting Pronouncements | |
In June 2014, the Financial Accounting Standards Board issued Accounting Standards Update 2014-10, Development Stage Enterprises: Elimination of Certain Financial Reporting Requirements, Including an Amendment to Variable Interest Entities Guidance in Topic 810, Consolidation (ASU 2014-10). ASU 2014-10 removes the definition of a development stage entity from the Master Glossary of the Accounting Standards Codification, thereby removing the financial reporting distinction between development stage entities and other reporting entities from U.S. GAAP. ASU 2014-10 also eliminates the requirements for development stage entities to (1) present inception-to-date information in the statements of income, cash flows, and shareholder equity, (2) label the financial statements as those of a development stage entity, (3) disclose a description of the development stage activities in which the entity is engaged, and (4) disclose in the first year in which the entity is no longer a development stage entity that in prior years it had been in the development stage. The amendments also clarify that the guidance in Topic 275, Risks and Uncertainties, is applicable to entities that have not commenced planned principal operations. The amendments in ASU 2014-10 will be effective retrospectively except for the clarification to Topic 275, which shall be applied prospectively for annual reporting periods beginning after December 15, 2014, and interim periods therein. Early application of each of the amendments is permitted for any annual reporting period or interim period for which the entity’s financial statements have not yet been issued. The Company elected to early adopt the provisions of ASU 2014-10 in the second quarter of 2014, as reflected in this Quarterly Report. |
Fair_Value_Measurements_Tables
Fair Value Measurements (Tables) | 6 Months Ended | |||||||||||||||
Jun. 30, 2014 | ||||||||||||||||
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): | ||||||||||||||||
30-Jun-14 | ||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 6,753 | $ | — | $ | — | $ | 6,753 | ||||||||
Commercial paper | — | 7,988 | — | 7,988 | ||||||||||||
Corporate bonds | — | 125,840 | — | 125,840 | ||||||||||||
U.S. Government securities | — | 3,002 | — | 3,002 | ||||||||||||
Total financial assets | $ | 6,753 | $ | 136,830 | $ | — | $ | 143,583 | ||||||||
December 31, 2013 | ||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 6,847 | $ | — | $ | — | $ | 6,847 | ||||||||
Commercial paper | — | 1,000 | — | 1,000 | ||||||||||||
Corporate bonds | — | 44,950 | — | 44,950 | ||||||||||||
Total financial assets | $ | 6,847 | $ | 45,950 | $ | — | $ | 52,797 | ||||||||
Financial Liabilities: | ||||||||||||||||
Convertible preferred stock warrant liability | $ | — | $ | — | $ | 3,419 | $ | 3,419 | ||||||||
Total financial liabilities | $ | — | $ | — | $ | 3,419 | $ | 3,419 | ||||||||
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): | ||||||||||||||||
Six Months Ended June 30, | ||||||||||||||||
2014 | 2013 | |||||||||||||||
Fair value, beginning of period | $ | 3,419 | $ | 518 | ||||||||||||
Change in fair value recorded as a loss in other expense, net | 3,324 | 372 | ||||||||||||||
Reclassification of warrant liability to additional paid-in capital | (6,743 | ) | — | |||||||||||||
Fair value, end of period | $ | — | $ | 890 | ||||||||||||
Balance_Sheet_Components_Table
Balance Sheet Components (Tables) | 6 Months Ended | |||||||||||||||
Jun. 30, 2014 | ||||||||||||||||
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: | ||||||||||||||||
30-Jun-14 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 6,753 | $ | — | $ | — | $ | 6,753 | ||||||||
Corporate bonds classified as cash equivalents | 1,011 | — | — | 1,011 | ||||||||||||
Commercial Paper classified as short-term investments | 7,988 | — | — | 7,988 | ||||||||||||
Corporate bonds classified as short-term investments | 124,883 | 20 | (74 | ) | 124,829 | |||||||||||
U.S Government securities classified as short-term investments | 2,996 | 6 | — | 3,002 | ||||||||||||
Total | $ | 143,631 | $ | 26 | $ | (74 | ) | $ | 143,583 | |||||||
December 31, 2013 | ||||||||||||||||
Gross Unrealized | ||||||||||||||||
Amortized | Gains | Losses | Estimated | |||||||||||||
Cost | Fair Value | |||||||||||||||
Money market funds classified as cash equivalents | $ | 6,847 | $ | — | $ | — | $ | 6,847 | ||||||||
Commercial Paper classified as short-term investments | 1,000 | — | — | 1,000 | ||||||||||||
Corporate bonds classified as short-term investments | 44,939 | 17 | (6 | ) | 44,950 | |||||||||||
Total | $ | 52,786 | $ | 17 | $ | (6 | ) | $ | 52,797 | |||||||
Accrued Liabilities | ' | |||||||||||||||
Accrued liabilities consist of the following (in thousands): | ||||||||||||||||
June 30, | December 31, | |||||||||||||||
2014 | 2013 | |||||||||||||||
Research and clinical study expenses | $ | 2,852 | $ | 1,886 | ||||||||||||
Payroll and related expenses | 2,208 | 2,360 | ||||||||||||||
Other | 218 | 160 | ||||||||||||||
Total accrued liabilities | $ | 5,278 | $ | 4,406 | ||||||||||||
Convertible_Preferred_Stock_Wa1
Convertible Preferred Stock Warrants and Common Stock Warrants (Tables) | 6 Months Ended | ||||||||||||
Jun. 30, 2014 | |||||||||||||
Common Stock Warrants | ' | ||||||||||||
Summary of Outstanding Warrants | ' | ||||||||||||
As of June 30, 2014, outstanding common stock warrants consisted of the following: | |||||||||||||
Common Stock Warrants: | Number of Warrants | Date Issued | Term | Exercise Price | |||||||||
Common stock | 83,167 | Jun-10 | 10 years | $ | 3.006 | ||||||||
Common stock | 203,759 | Feb-11 | 10 years | 3.006 | |||||||||
Common stock | 66,533 | Jun-11 | 10 years | 3.006 | |||||||||
Total common stock warrants | 353,459 | ||||||||||||
Convertible Preferred Stock Warrants | ' | ||||||||||||
Summary of Outstanding Warrants | ' | ||||||||||||
As of December 31, 2013, outstanding preferred stock warrants consisted of the following: | |||||||||||||
Convertible Preferred Stock Warrants: | Number of Warrants | Date Issued | Term | Exercise Price | |||||||||
Series A | 241,803 | Jun-10 | 10 years | $ | 1.034 | ||||||||
Series A | 592,417 | Feb-11 | 10 years | 1.034 | |||||||||
Series A | 193,442 | Jun-11 | 10 years | 1.034 | |||||||||
Total convertible preferred stock warrants | 1,027,662 | ||||||||||||
StockBased_Compensation_Tables
Stock-Based Compensation (Tables) | 6 Months Ended | ||||||||||||||||
Jun. 30, 2014 | |||||||||||||||||
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 June 30, | Six Months Ended June 30, | ||||||||||||||||
2014 | 2013 | 2014 | 2013 | ||||||||||||||
Research and development | $ | 652 | $ | 52 | $ | 1,357 | $ | 82 | |||||||||
General and administrative | 294 | 133 | 384 | 294 | |||||||||||||
Total stock-based compensation | $ | 946 | $ | 185 | $ | 1,741 | $ | 376 | |||||||||
Net_Loss_per_Share_Attributabl1
Net Loss per Share Attributable to Common Stockholders (Tables) | 6 Months Ended | |||||||||||||||
Jun. 30, 2014 | ||||||||||||||||
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 June 30, | Six Months Ended June 30, | |||||||||||||||
2014 | 2013 | 2014 | 2013 | |||||||||||||
Numerator: | ||||||||||||||||
Net loss | $ | (13,585 | ) | $ | (8,590 | ) | $ | (27,215 | ) | $ | (15,325 | ) | ||||
Accretion and dividends on convertible preferred stock | - | (2,239 | ) | (4,808 | ) | (3,709 | ) | |||||||||
Net loss attributable to common stockholders | $ | (13,585 | ) | $ | (10,829 | ) | $ | (32,023 | ) | $ | (19,034 | ) | ||||
Denominator: | ||||||||||||||||
Weighted-average common shares outstanding | 30,055,943 | 3,503,214 | 25,697,407 | 3,482,304 | ||||||||||||
Less: weighted-average unvested common shares subject to repurchase | — | (245,408 | ) | — | (405,397 | ) | ||||||||||
Weighted-average shares used to compute net loss per share attributable | 30,055,943 | 3,257,806 | 25,697,407 | 3,076,907 | ||||||||||||
to common stockholders, basic and diluted | ||||||||||||||||
Net loss per share attributable to common stockholders, basic and diluted | $ | (0.45 | ) | $ | (3.32 | ) | $ | (1.25 | ) | $ | (6.19 | ) | ||||
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 June 30, | Six Months Ended June 30, | |||||||||||||||
2014 | 2013 | 2014 | 2013 | |||||||||||||
Convertible preferred stock | — | 19,598,486 | 3,248,368 | 19,598,486 | ||||||||||||
Stock options to purchase common stock | 2,561,357 | 1,647,753 | 2,438,351 | 1,543,954 | ||||||||||||
Common stock subject to repurchase | — | 245,408 | — | 405,397 | ||||||||||||
Warrants to purchase convertible preferred stock | — | 353,459 | 294,875 | 353,459 | ||||||||||||
(as if converted) | ||||||||||||||||
Warrants to purchase common stock | 353,459 | — | 58,584 | — | ||||||||||||
2,914,816 | 21,845,106 | 6,040,178 | 21,901,296 | |||||||||||||
Organization_Additional_Inform
Organization - Additional Information (Details) (USD $) | 6 Months Ended | 0 Months Ended | 6 Months Ended | 0 Months Ended | |||
Jun. 30, 2014 | Jun. 30, 2013 | Dec. 31, 2013 | Feb. 05, 2014 | Jun. 30, 2014 | Jan. 30, 2014 | Feb. 05, 2014 | |
IPO | IPO | IPO | IPO | ||||
Underwriters Overallotment Option | |||||||
Organization And Nature Of Business [Line Items] | ' | ' | ' | ' | ' | ' | ' |
Effective date of registration | ' | ' | ' | ' | 30-Jan-14 | ' | ' |
Public offering price of the shares sold | ' | ' | ' | ' | ' | $21 | ' |
Common stock issued in initial public offering | ' | ' | ' | 6,624,423 | ' | ' | 864,054 |
Net proceeds from issuance of common stock | $126,110,000 | $57,000 | ' | $129,400,000 | ' | ' | ' |
Underwriting discounts and commissions | ' | ' | ' | 9,700,000 | ' | ' | ' |
Offering expenses | ' | ' | ' | 3,300,000 | ' | ' | ' |
Cash dividends paid to preferred stockholders | ' | ' | ' | 4,300,000 | ' | ' | ' |
Net proceeds from initial public offering | ' | ' | ' | $121,700,000 | ' | ' | ' |
Number of preferred stock converted into common stock | ' | ' | ' | 19,598,486 | ' | ' | ' |
Authorized capital stock including preferred shares | 275,000,000 | ' | ' | ' | ' | ' | ' |
Common stock, shares authorized | 250,000,000 | ' | 250,000,000 | ' | ' | ' | ' |
Preferred stock, shares authorized | 25,000,000 | ' | 25,000,000 | ' | ' | ' | ' |
Preferred stock, par value | $0.00 | ' | $0.00 | ' | ' | ' | ' |
Common stock, par value | $0.00 | ' | $0.00 | ' | ' | ' | ' |
Preferred stock, shares outstanding | 0 | ' | 0 | ' | ' | ' | ' |
Summary_of_Significant_Account2
Summary of Significant Accounting Policies - Additional Information (Details) (USD $) | 1 Months Ended | |||||
Jan. 17, 2014 | Jun. 30, 2014 | Mar. 31, 2014 | Feb. 05, 2014 | Jun. 30, 2014 | Dec. 31, 2013 | |
Underwritten Offering Costs | IPO | IPO | Prepaid and Other Current Assets | Prepaid and Other Current Assets | ||
Costs Capitalized | Costs Capitalized | |||||
Summary Of Significant Accounting Policies [Line Items] | ' | ' | ' | ' | ' | ' |
Reverse stock split ratio on preferred and common stock | 0.319 | ' | ' | ' | ' | ' |
Deferred offering Costs | ' | ' | $3,300,000 | ' | $316,000 | $2,300,000 |
Offering expenses | ' | $444,000 | ' | $3,300,000 | ' | ' |
Fair_Value_Measurements_Summar
Fair Value Measurements - Summary of Financial Assets and Liabilities Measured on Recurring Basis (Details) (USD $) | Jun. 30, 2014 | Dec. 31, 2013 |
In Thousands, unless otherwise specified | ||
Financial Assets: | ' | ' |
Fair value | $143,583 | $52,797 |
Financial Liabilities: | ' | ' |
Fair value | ' | 3,419 |
Money Market Funds | ' | ' |
Financial Assets: | ' | ' |
Fair value | 6,753 | 6,847 |
Commercial Paper | ' | ' |
Financial Assets: | ' | ' |
Fair value | 7,988 | 1,000 |
U.S. Government Securities | ' | ' |
Financial Assets: | ' | ' |
Fair value | 3,002 | ' |
Level 1 | ' | ' |
Financial Assets: | ' | ' |
Fair value | 6,753 | 6,847 |
Level 1 | Money Market Funds | ' | ' |
Financial Assets: | ' | ' |
Fair value | 6,753 | 6,847 |
Level 2 | ' | ' |
Financial Assets: | ' | ' |
Fair value | 136,830 | 45,950 |
Level 2 | Commercial Paper | ' | ' |
Financial Assets: | ' | ' |
Fair value | 7,988 | 1,000 |
Level 2 | U.S. Government Securities | ' | ' |
Financial Assets: | ' | ' |
Fair value | 3,002 | ' |
Level 3 | ' | ' |
Financial Liabilities: | ' | ' |
Fair value | ' | 3,419 |
Corporate Bonds | ' | ' |
Financial Assets: | ' | ' |
Fair value | 125,840 | 44,950 |
Corporate Bonds | Level 2 | ' | ' |
Financial Assets: | ' | ' |
Fair value | 125,840 | 44,950 |
Convertible Preferred Stock Warrant Liability | ' | ' |
Financial Liabilities: | ' | ' |
Fair value | ' | 3,419 |
Convertible Preferred Stock Warrant Liability | Level 3 | ' | ' |
Financial Liabilities: | ' | ' |
Fair value | ' | $3,419 |
Fair_Value_Measurements_Additi
Fair Value Measurements - Additional Information (Details) (USD $) | 1 Months Ended | |
Dec. 31, 2013 | Jan. 30, 2014 | |
Back Solve Method | 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 | $12.14 | $21 |
Expected volatility | 70.00% | 70.00% |
Expected time | '5 months 5 days | '7 years |
Expected risk free interest rate | 0.11% | 2.19% |
Fair_Value_Measurements_Summar1
Fair Value Measurements - Summary of Changes in Estimated Fair Value (Details) (USD $) | 6 Months Ended | |
In Thousands, unless otherwise specified | Jun. 30, 2014 | Jun. 30, 2013 |
Fair Value Inputs Assets Quantitative Information [Line Items] | ' | ' |
Fair value, beginning of period | $3,419 | $518 |
Change in fair value recorded as a loss in other expense, net | 3,324 | 372 |
Reclassification of warrant liability to additional paid-in capital | -6,743 | ' |
Fair value, end of period | ' | $890 |
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 $) | Jun. 30, 2014 | Dec. 31, 2013 |
In Thousands, unless otherwise specified | ||
Schedule Of Available For Sale Securities [Line Items] | ' | ' |
Amortized Cost | $143,631 | $52,786 |
Gross Unrealized Gains | 26 | 17 |
Gross Unrealized Losses | -74 | -6 |
Estimated Fair Value | 143,583 | 52,797 |
Money Market Funds | ' | ' |
Schedule Of Available For Sale Securities [Line Items] | ' | ' |
Amortized Cost | 6,753 | 6,847 |
Estimated Fair Value | 6,753 | 6,847 |
Corporate bonds classified as cash equivalents | ' | ' |
Schedule Of Available For Sale Securities [Line Items] | ' | ' |
Amortized Cost | 1,011 | ' |
Estimated Fair Value | 1,011 | ' |
Commercial Paper classified as short-term investments | ' | ' |
Schedule Of Available For Sale Securities [Line Items] | ' | ' |
Amortized Cost | 7,988 | 1,000 |
Estimated Fair Value | 7,988 | 1,000 |
Corporate bonds classified as short-term investments | ' | ' |
Schedule Of Available For Sale Securities [Line Items] | ' | ' |
Amortized Cost | 124,883 | 44,939 |
Gross Unrealized Gains | 20 | 17 |
Gross Unrealized Losses | -74 | -6 |
Estimated Fair Value | 124,829 | 44,950 |
U.S Government securities classified as short-term investments | ' | ' |
Schedule Of Available For Sale Securities [Line Items] | ' | ' |
Amortized Cost | 2,996 | ' |
Gross Unrealized Gains | 6 | ' |
Estimated Fair Value | $3,002 | ' |
Balance_Sheet_Components_Addit
Balance Sheet Components - Additional Information (Details) (USD $) | 6 Months Ended |
In Thousands, unless otherwise specified | Jun. 30, 2014 |
Schedule Of Available For Sale Securities [Line Items] | ' |
Available-for-sale Securities, Gross Realized Gain (Loss) | $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 $) | Jun. 30, 2014 | Dec. 31, 2013 |
In Thousands, unless otherwise specified | ||
Accrued Liabilities [Line Items] | ' | ' |
Research and clinical study expenses | $2,852 | $1,886 |
Payroll and related expenses | 2,208 | 2,360 |
Other | 218 | 160 |
Total accrued liabilities | $5,278 | $4,406 |
License_and_Research_Agreement1
License and Research Agreements - Additional Information (Details) | Jun. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2010 | Sep. 30, 2010 | Jun. 30, 2014 | Jun. 30, 2014 | Nov. 30, 2010 | Jun. 30, 2014 | Dec. 31, 2012 | Jun. 30, 2014 | Jun. 30, 2014 | Dec. 31, 2012 | Jun. 30, 2014 | Jun. 30, 2013 | Dec. 31, 2012 | Jun. 30, 2014 | Jun. 30, 2014 | Jun. 30, 2014 | Jun. 30, 2014 |
Nobelpharma License Agreement | Nobelpharma License Agreement | Nobelpharma License Agreement | Nobelpharma License Agreement | Saint Louis University License Agreement | Saint Louis University License Agreement | H I B M Research Group | H I B M Research Group | H I B M Research Group | St. Jude Children's Research Hospital License Agreement | St. Jude Children's Research Hospital License Agreement | Baylor Research Institute License Agreement | Baylor Research Institute License Agreement | Baylor Research Institute License Agreement | Baylor Research Institute License Agreement | Kyowa Hakko Kirin Collaboration | Kyowa Hakko Kirin Collaboration | |||
USD ($) | 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 ($) | USD ($) | |||||||||||||||
Research And Development [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
License agreement | ' | ' | $110,500 | ¥ 10,000,000 | ' | ' | $10,000 | ' | $25,000 | ' | ' | $10,000 | ' | ' | $250,000 | ' | ' | $1,300,000 | $2,000,000 |
Common stock, shares issued | 30,059,288 | 3,766,289 | 76,567 | 76,567 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Payments of milestone | ' | ' | ' | ' | 495,000 | ' | ' | 100,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Remaining total aggregate payments | ' | ' | ' | ' | 2,000,000 | 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 | ' | ' | ' | ' | ' |
Convertible_Preferred_Stock_Wa2
Convertible Preferred Stock Warrants and Common Stock Warrants - Summary of Outstanding Warrants (Details) | Jun. 30, 2014 | Jun. 30, 2014 | Jun. 30, 2014 | Jun. 30, 2014 | Dec. 31, 2013 | Dec. 31, 2013 | Dec. 31, 2013 | Dec. 31, 2013 |
Common Stock | Common Stock | Common Stock | Common Stock | Convertible Preferred Stock | Convertible Preferred Stock | Convertible Preferred Stock | Convertible Preferred Stock | |
June 2010 Common Stock Warrants | February 2011 Common Stock Warrants | June 2011 Common Stock Warrants | June 2010 Series A Convertible Preferred Stock Warrant | February 2011 Series A Convertible Preferred Stock Warrant | June 2011 Series A Convertible Preferred Stock Warrants | |||
Class Of Warrant Or Right [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' |
Number of Warrants | 353,459 | 83,167 | 203,759 | 66,533 | 1,027,662 | 241,803 | 592,417 | 193,442 |
Date Issued | ' | '2010-06 | '2011-02 | '2011-06 | ' | '2010-06 | '2011-02 | '2011-06 |
Term | ' | '10 years | '10 years | '10 years | ' | '10 years | '10 years | '10 years |
Exercise Price | ' | 3.006 | 3.006 | 3.006 | ' | 1.034 | 1.034 | 1.034 |
Convertible_Preferred_Stock_Wa3
Convertible Preferred Stock Warrants and Common Stock Warrants - Additional Information (Details) (USD $) | 3 Months Ended | 6 Months Ended | 3 Months Ended | 6 Months Ended | ||||||
Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 | Dec. 31, 2013 | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 | Jan. 30, 2014 | |
Warrant | Warrant | Warrant | Warrant | IPO | ||||||
Class Of Warrant Or Right [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Fair value of the warrants | ' | ' | ' | ' | $3,400,000 | ' | ' | ' | ' | $6,700,000 |
Other income (expense) | $76,000 | ($343,000) | ($3,215,000) | ($331,000) | ' | $0 | ($374,000) | ($3,300,000) | ($372,000) | ' |
Convertible_Preferred_Stock_Ad
Convertible Preferred Stock - Additional Information (Details) (USD $) | 1 Months Ended | 3 Months Ended | 6 Months Ended | 12 Months Ended | 6 Months Ended | 12 Months Ended | |||
In Millions, except Share data, unless otherwise specified | Feb. 05, 2014 | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 | Dec. 31, 2013 | Dec. 31, 2012 | Jun. 30, 2014 | Dec. 31, 2013 |
Series A Convertible Preferred Stock | Series A Convertible Preferred Stock | Series A Convertible Preferred Stock | Series A Convertible Preferred Stock | Series A Convertible Preferred Stock | Series A Convertible Preferred Stock | Series B Convertible Preferred Stock | Series B Convertible Preferred Stock | ||
Convertible Preferred Stock [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Dividends per share | ' | ' | ' | $0.06 | ' | ' | ' | $0.06 | ' |
Amount of dividends declared and paid | ' | ' | ' | ' | ' | ' | $2.10 | ' | ' |
Amount of dividends in arrears paid | ' | ' | ' | ' | ' | 4 | ' | ' | 4 |
Conversion of preferred stock into common stock, shares | 19,598,486 | ' | ' | ' | ' | ' | ' | ' | ' |
Amount of dividends accrued and outstanding paid | 4.3 | ' | ' | ' | ' | ' | ' | ' | ' |
Convertible preferred stock redemption terms | ' | ' | ' | 'The Series A convertible preferred stock was redeemable at any time after June 16, 2017 once a written request to redeem such stock was received by the Company from holders of not less than seventy-five percent of the then outstanding Series A convertible preferred stock. | ' | ' | ' | ' | ' |
Convertible preferred stock redemption date | ' | ' | ' | 16-Jun-17 | ' | ' | ' | ' | ' |
Accretion of preferred stock redemption | ' | $0 | $1.80 | $4.40 | $2.90 | ' | ' | ' | ' |
StockBased_Compensation_Additi
Stock-Based Compensation - Additional Information (Details) (USD $) | 6 Months Ended | 3 Months Ended | 6 Months Ended | 6 Months Ended | 6 Months Ended | ||||||||
Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 16, 2011 | Jun. 30, 2014 | Feb. 05, 2014 | Jun. 30, 2014 | Jun. 30, 2014 | Jun. 30, 2014 | Feb. 05, 2014 | |
Founder's Stock | Founder's Stock | Founder's Stock | Founder's Stock | Founder's Stock | 2011 Equity Incentive Plan | 2011 Equity Incentive Plan | 2011 Equity Incentive Plan | 2011 Equity Incentive Plan | 2014 Incentive Plan | 2014 Incentive Plan | |||
First Anniversary | Maximum | ||||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Minimum percentage of voting rights of all classes of stock | ' | ' | ' | ' | ' | ' | ' | 10.00% | ' | ' | ' | ' | ' |
Percentage of statutory stock options | ' | ' | ' | ' | ' | ' | ' | 110.00% | ' | ' | ' | ' | ' |
Options expiration period | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | '10 years | ' | ' |
Options vesting period | ' | ' | ' | ' | ' | ' | ' | '4 years | ' | ' | ' | ' | ' |
Option vesting rights, percentage | ' | ' | ' | ' | ' | ' | ' | 2.08% | ' | 25.00% | ' | ' | ' |
Share of common stock available for future issuance | ' | ' | ' | ' | ' | ' | ' | ' | 655,038 | ' | ' | ' | 2,250,000 |
Automatic increases in shares available for grant effective date | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 1-Jan-15 | ' |
Shares available for grant, ending date | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 1-Jan-24 | ' |
Number of shares authorized to be repurchased | ' | ' | ' | ' | ' | ' | 2,552,241 | ' | ' | ' | ' | ' | ' |
Repurchase rights expiration period | ' | ' | ' | ' | '2 years | ' | ' | ' | ' | ' | ' | ' | ' |
Estimated fair value of restricted shares | ' | ' | ' | ' | $1,199,000 | ' | ' | ' | ' | ' | ' | ' | ' |
Stock-based compensation expense | $1,741,000 | $376,000 | $0 | $119,000 | $0 | $268,000 | ' | ' | ' | ' | ' | ' | ' |
StockBased_Compensation_Functi
Stock-Based Compensation - Functional Classification of Stock-Based Compensation Expense, Net of Estimated Forfeitures (Details) (USD $) | 3 Months Ended | 6 Months Ended | ||
In Thousands, unless otherwise specified | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ' | ' | ' | ' |
Stock-based compensation expense | $946 | $185 | $1,741 | $376 |
Research and development | ' | ' | ' | ' |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ' | ' | ' | ' |
Stock-based compensation expense | 652 | 52 | 1,357 | 82 |
General and administrative | ' | ' | ' | ' |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ' | ' | ' | ' |
Stock-based compensation expense | $294 | $133 | $384 | $294 |
Commitments_and_Contingencies_
Commitments and Contingencies - Additional Information (Details) (USD $) | Jun. 30, 2014 |
Long Term Purchase Commitment [Line Items] | ' |
Purchase Obligation | $780,000 |
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 | 6 Months Ended | ||
In Thousands, except Share data, unless otherwise specified | Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 |
Numerator: | ' | ' | ' | ' |
Net loss | ($13,585) | ($8,590) | ($27,215) | ($15,325) |
Accretion and dividends on convertible preferred stock | ' | -2,239 | -4,808 | -3,709 |
Net loss attributable to common stockholders | ($13,585) | ($10,829) | ($32,023) | ($19,034) |
Denominator: | ' | ' | ' | ' |
Weighted-average common shares outstanding | 30,055,943 | 3,503,214 | 25,697,407 | 3,482,304 |
Less: weighted-average unvested common shares subject to repurchase | ' | -245,408 | ' | -405,397 |
Weighted-average shares used to compute net loss per share attributable to common stockholders, basic and diluted | 30,055,943 | 3,257,806 | 25,697,407 | 3,076,907 |
Net loss per share attributable to common stockholders, basic and diluted | ($0.45) | ($3.32) | ($1.25) | ($6.19) |
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 | 6 Months Ended | ||
Jun. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2014 | Jun. 30, 2013 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ' | ' | ' | ' |
Common stock equivalents excluded from computation of diluted net loss per share | 2,914,816 | 21,845,106 | 6,040,178 | 21,901,296 |
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 | ' | 19,598,486 | 3,248,368 | 19,598,486 |
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 | 2,561,357 | 1,647,753 | 2,438,351 | 1,543,954 |
Common Stock Subject to Repurchase | ' | ' | ' | ' |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ' | ' | ' | ' |
Common stock equivalents excluded from computation of diluted net loss per share | ' | 245,408 | ' | 405,397 |
Warrants to Purchase Convertible Preferred Stock (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 | ' | 353,459 | 294,875 | 353,459 |
Warrants 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 | 353,459 | ' | 58,584 | ' |
Subsequent_Events_Additional_I
Subsequent Events - Additional Information (Details) (USD $) | Jun. 30, 2014 | Dec. 31, 2013 | Jul. 31, 2014 |
Subsequent Event | |||
Subsequent Event [Line Items] | ' | ' | ' |
Common stock, shares issued | 30,059,288 | 3,766,289 | 1,613,879 |
Common stock sold by existing shareholders | ' | ' | 706,072 |
Purchase of additional shares by underwriters | ' | ' | 302,602 |
Common stock issued price per share | ' | ' | $40 |
Proceeds from public offering before offering expenses | ' | ' | $60,700,000 |
Underwriting discounts and commissions | ' | ' | 3,900,000 |
Offering expenses | ' | ' | 400,000 |
Proceeds from public offering, net | ' | ' | $60,200,000 |