Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2016 | Oct. 28, 2016 | |
Document and Entity Information | ||
Entity Registrant Name | AMICUS THERAPEUTICS INC | |
Entity Central Index Key | 1,178,879 | |
Document Type | 10-Q | |
Document Period End Date | Sep. 30, 2016 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Entity Current Reporting Status | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Common Stock, Shares Outstanding | 142,326,195 | |
Document Fiscal Year Focus | 2,016 | |
Document Fiscal Period Focus | Q3 |
Consolidated Balance Sheets (Un
Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Current assets: | ||
Cash and cash equivalents | $ 33,115 | $ 69,485 |
Investments in marketable securities | 179,284 | 144,548 |
Accounts receivable | 864 | |
Inventories | 3,251 | 0 |
Prepaid expenses and other current assets | 5,198 | 2,568 |
Total current assets | 221,712 | 216,601 |
Property and equipment, less accumulated depreciation of $15,181 and $13,353 at September 30, 2016 and December 31, 2015, respectively | 10,183 | 6,178 |
In-process research & development | 486,700 | 486,700 |
Goodwill | 197,797 | 197,797 |
Other non-current assets | 1,788 | 1,108 |
Total Assets | 918,180 | 908,384 |
Current liabilities: | ||
Accounts payable and accrued expenses | 29,013 | 32,216 |
Contingent consideration payable, current portion | 55,992 | 41,400 |
Other current liabilities | 607 | |
Total current liabilities | 85,612 | 73,616 |
Deferred reimbursements | 35,756 | 35,756 |
Due to related party | 44,047 | 41,601 |
Unsecured notes payable | 21,977 | |
Contingent consideration payable, less current portion | 216,198 | 232,677 |
Deferred tax liability | 176,219 | 176,219 |
Other non-current liabilities | 1,816 | 681 |
Commitments and contingencies | ||
Stockholders' equity: | ||
Common stock, $.01 per value, 250,000,000 authorized, 142,273,085 shares issued and outstanding at September 30, 2016, 250,000,000 shares authorized, 125,027,034 shares issued and outstanding at December 31, 2015 | 1,478 | 1,306 |
Additional paid-in capital | 1,038,613 | 917,454 |
Accumulated other comprehensive loss: | ||
Foreign currency translation adjustment, less tax benefit of $706 at September 30, 2016 | 1,062 | |
Unrealized gain/(loss) on available-for securities | 287 | (115) |
Warrants | 16,076 | 8,755 |
Accumulated deficit | (720,961) | (579,566) |
Total stockholders' equity | 336,555 | 347,834 |
Total Liabilities and Stockholders' Equity | $ 918,180 | $ 908,384 |
Consolidated Balance Sheets (U3
Consolidated Balance Sheets (Unaudited) (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2016 | Dec. 31, 2015 | |
Consolidated Balance Sheets (Unaudited) | |||
Accumulated depreciation of property and equipment (in dollars) | $ 15,181 | $ 15,181 | $ 13,353 |
Common stock, par value (in dollars per share) | $ 0.01 | $ 0.01 | $ 0.01 |
Common stock, shares authorized | 250,000,000 | 250,000,000 | 250,000,000 |
Common stock, shares issued | 142,273,085 | 142,273,085 | 125,027,034 |
Common stock, shares outstanding | 142,273,085 | 142,273,085 | 125,027,034 |
Foreign currency translation adjustment, tax | $ 253 | $ 706 |
Consolidated Statements of Oper
Consolidated Statements of Operations (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Consolidated Statements of Operations (Unaudited) | ||||
Net product sales | $ 2,127 | $ 2,127 | ||
Cost of goods sold | 344 | 344 | ||
Gross profit | 1,783 | 1,783 | ||
Operating Expenses: | ||||
Research and development | 32,457 | $ 20,971 | 74,163 | $ 54,318 |
Selling, general and administrative | 17,469 | 15,372 | 52,470 | 30,077 |
Changes in fair value of contingent consideration payable | (4,110) | 1,300 | 9,228 | 2,400 |
Restructuring charges | 11 | 7 | 69 | 44 |
Loss on extinguishment of debt | 952 | |||
Depreciation | 896 | 395 | 2,336 | 1,256 |
Total operating expenses | 46,723 | 38,045 | 138,266 | 89,047 |
Loss from operations | (44,940) | (38,045) | (136,483) | (89,047) |
Other income (expenses): | ||||
Interest income | 460 | 316 | 1,098 | 645 |
Interest expense | (1,517) | (17) | (3,517) | (727) |
Other expense | (910) | (54) | (3,199) | (93) |
Loss before income tax benefit | (46,907) | (37,800) | (142,101) | (89,222) |
Income tax benefit | 253 | 706 | ||
Net loss | $ (46,654) | $ (37,800) | $ (141,395) | $ (89,222) |
Net loss per common share - basic and diluted | $ (0.33) | $ (0.32) | $ (1.07) | $ (0.85) |
Weighted-average common shares outstanding - basic and diluted | 140,656,109 | 118,724,882 | 131,675,690 | 104,885,956 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Consolidated Statements of Comprehensive Loss (Unaudited) | ||||
Net loss | $ (46,654) | $ (37,800) | $ (141,395) | $ (89,222) |
Other comprehensive gain/(loss) | ||||
Foreign currency translation adjustment, net of tax $253 and $706, respectively | 220 | 1,062 | ||
Unrealized gain/(loss) on available-for-sale securities | 86 | (91) | 402 | (10) |
Other comprehensive gain/(loss) | 306 | (91) | 1,464 | (10) |
Comprehensive loss | $ (46,348) | $ (37,891) | $ (139,931) | $ (89,232) |
Consolidated Statements of Com6
Consolidated Statements of Comprehensive Loss (Unaudited) (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended |
Sep. 30, 2016 | Sep. 30, 2016 | |
Consolidated Statements of Comprehensive Loss (Unaudited) | ||
Foreign currency translation adjustment, tax | $ 253 | $ 706 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Operating activities | ||
Net loss | $ (141,395) | $ (89,222) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Non-cash interest expense | 1,744 | 136 |
Depreciation | 2,336 | 1,256 |
Stock-based compensation | 13,087 | 6,929 |
Charges to research expense for stock issued in asset acquisition | 4,607 | |
Restructuring charges | 69 | 44 |
Loss on extinguishment of debt | 952 | |
Loss on disposal of asset | 17 | |
Non-cash changes in the fair value of derivative liability | 324 | |
Non-cash changes in the fair value of contingent consideration payable | 9,228 | 2,400 |
Foreign currency remeasurement loss | 2,207 | |
Non-cash income tax benefit | (706) | |
Changes in operating assets and liabilities: | ||
Accounts receivable | (863) | |
Inventories | (3,505) | |
Prepaid expenses and other current assets | (2,803) | (668) |
Other non-current assets | (660) | (540) |
Accounts payable and accrued expenses | (2,920) | 14,118 |
Non-current liabilities | 684 | (33) |
Net cash used in operating activities | (118,549) | (64,628) |
Investing activities | ||
Sale and redemption of marketable securities | 165,495 | 133,418 |
Purchases of marketable securities | (199,829) | (220,861) |
Purchases of property and equipment | (5,520) | (2,246) |
Acquisitions, net of cash acquired | (141,060) | |
Net cash used in investing activities | (39,854) | (230,749) |
Financing activities | ||
Proceeds from issuance of common stock, net of issuance costs | 97,068 | 243,042 |
Proceeds from unsecured note agreement | 30,000 | |
Payments of secured loan agreement | (15,291) | |
Proceeds from related party | 50,000 | |
Payment of capital lease | (118) | |
Payment of contingent consideration | (5,000) | |
Proceeds from exercise of stock options | 1,456 | 10,673 |
Purchase of vested restricted stock units | (1,010) | (1,682) |
Proceeds from exercise of warrants | 4,000 | |
Net cash provided by financing activities | 122,396 | 290,742 |
Effect of exchange rate changes on cash and cash equivalents | (363) | |
Net decrease in cash and cash equivalents | (36,370) | (4,635) |
Cash and cash equivalents at beginning of period | 69,485 | 24,074 |
Cash and cash equivalents at end of period | 33,115 | 19,439 |
Supplemental disclosures of cash flow information | ||
Cash paid during the period for interest | 284 | $ 605 |
Contingent consideration resolution in shares | 6,115 | |
Capital expenditures funded by capital lease borrowings | $ 850 |
Description of Business
Description of Business | 9 Months Ended |
Sep. 30, 2016 | |
Description of Business | |
Description of Business | Note 1. Corporate Information, Status of Operations, and Management Plans Amicus Therapeutics, Inc. (the “Company,” “we,” “us,” or “our”) is a global patient-focused biotechnology company engaged in the discovery, development, and commercialization of a diverse set of novel treatments for patients living with devastating rare and orphan diseases. The lead product, migalastat HCl is a small molecule that can be used as a monotherapy and in combination with enzyme replacement of therapy (“ERT”) for Fabry disease. The Company’s Fabry franchise strategy is to develop migalastat HCl (which the Company may refer to as “migalastat”) for all patients with Fabry disease - as a monotherapy for patients with amenable mutations and in combination with ERT for all other patients. In May 2016, the Company announced that the European Commission (“EC”) had granted full approval for the oral small molecule pharmacological chaperone Galafold™ (migalastat) as a first-line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation. The approved label includes 269 Fabry-causing mutations, which represent up to half of all patients with Fabry disease. The Company commenced commercial shipments of Galafold in the EU in the second quarter of 2016 and recognized net product sales of $2.1 million in the third quarter of 2016. Also in the pipeline, SD-101 is, a product candidate in late-stage development, as a potential first-to-market therapy for the chronic, rare connective tissue disorder Epidermolysis Bullosa (“EB”). The Company is also leveraging its biologics and Chaperone-Advanced Replacement Therapy (“CHART™”) platform technologies to develop novel ERT products for Pompe disease, Fabry disease, and potentially other lysosomal storage disorders (“LSDs”). The Company is also investigating preclinical and discovery programs in other rare and devastating diseases including CDKL5 deficiency. The Company believes that the platform technologies and advanced product pipeline uniquely position the Company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases. In July 2016, the Company expanded its biologics pipeline with a new preclinical program for CDKL5 deficiency, a rare and devastating genetic neurological disease for which there is no currently approved treatment. The Company has obtained the rights and related intellectual property to a preclinical CDKL5 program through its acquisition of MiaMed, Inc (“MiaMed”). Under the terms of the MiaMed Agreement and Plan of Merger (the “MiaMed Agreement”), with MiaMed and certain other parties signatory thereto, in connection with the closing of the transactions contemplated by the MiaMed Agreement, the former holders of MiaMed’s capital stock (collectively, the “MiaMed Stockholders”) received an aggregate of $6.5 million, comprised of (i) approximately $1.8 million in cash (plus MiaMed’s cash and cash equivalents at closing and less any of MiaMed’s unpaid third-party fees and expenses related to the transaction), and (ii) 825,603 shares of Amicus common stock. In addition, Amicus also agreed to pay up to an additional $83.0 million in connection with the achievement of certain clinical, regulatory and commercial milestones, for a potential aggregate deal value of $89.5 million. On June 30, 2016, the Company entered into a Joinder to and Amendment of Note and Warrant Purchase Agreement (the “Amended Purchase Agreement”) with Redmile Capital Fund, LP and certain of its affiliates (collectively referred to as “Redmile”). Such amendment joined Grosvenor Special Opportunities Master Fund, Ltd. (“GCM”) to the Note and Warrant Purchase Agreement, dated as of February 19, 2016. At closing, the Company sold (a) $30.0 million principal amount of additional notes and (b) five-year warrants to purchase 42 shares of common stock of the Company, par value $0.01 per share (“Common Stock”) for every $1,000 of the principal amount of Additional Notes purchased by each Purchaser (“Additional Warrants”), for an aggregate of approximately 1.3 million shares of Common Stock issuable under the Additional Warrants. For additional information, see “—Note 7. Debt Instruments and Related Party Transactions.” Beginning in April 2016 and through July 2016, the Company sold 15.0 million shares of Common Stock under an at-the-market (“ATM”) equity program with Cowen and Company, LLC (“Cowen”) acting as sales agent. Cowen was compensated at a fixed commission rate up to 3.0%. The ATM sales agreement resulted in net proceeds of $97.1 million, after Cowen’s commission of $2.7 million and other expenses of $0.2 million. The Company has completed all sales under the ATM equity program. The Company had an accumulated deficit of approximately $721.0 million at September 30, 2016 and anticipates incurring losses through the fiscal year ending December 31, 2016 and beyond. The Company has funded its operating losses to date through the sale of its redeemable convertible preferred stock, issuance of convertible notes, net proceeds from its initial public offering and subsequent stock offerings, payments from partners during the terms of the collaboration agreements and other financing arrangements. The Company commenced commercial shipments of Galafold in the EU in the second quarter of 2016 and recognized net product sales of $2.1 million in the third quarter of 2016. The Company believes that its existing cash and cash equivalents and short-term investments will be sufficient to fund the current operating plan into the fourth quarter of 2017. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2016 | |
Summary of Significant Accounting Policies | |
Summary of Significant Accounting Policies | Note 2. The consolidated financial statements include the accounts of Amicus Therapeutics, Inc. and its wholly-owned subsidiaries, after the elimination of intercompany transactions. Basis of Presentation The Company has prepared the accompanying unaudited consolidated financial statements in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulations S-X. Accordingly, they do not include all of the information and disclosures required by generally accepted accounting principles for complete financial statements. In the opinion of management, the accompanying unaudited financial statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s interim financial information. The accompanying unaudited consolidated financial statements and related notes should be read in conjunction with the Company’s financial statements and related notes as contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015. For a complete description of the Company’s accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2015. Foreign Currency Transactions The functional currency for most of our foreign subsidiaries is their local currency. For our non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of our foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of equity. The Company transacts business in various foreign countries and therefore, is subject to risk of foreign currency exchange rate fluctuations. As such, in June 2016 the Company entered into one forward contract to economically hedge transactional exposure associated with commitments arising from trade accounts payable denominated in a currency other than the functional currency of the respective operating entity. The Company does not designate this forward contract as a hedging instrument under applicable accounting guidance and, therefore, changes in fair value are recorded in the Consolidated Statements of Operations. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. Concentration of Credit Risk The Company’s financial instruments that are exposed to concentration of credit risk consist primarily of cash and cash equivalents and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on cash and cash equivalents or its marketable securities. The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold. The majority of the Company’s accounts receivable at September 30, 2016 have arisen from product sales in Germany. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined and the Company evaluates the creditworthiness of each customer on a regular basis. To date, the Company has not incurred any credit losses. Significant Accounting Policies There have been no material changes to the Company’s significant accounting policies during the nine months ended September 30, 2016, as compared to the significant accounting policies disclosed in Note 2 of the Consolidated Financial Statements in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015. However, the following accounting policies are the most critical in fully understanding and evaluating the Company’s financial condition and results of operations. Additionally, the Company added new policies on inventory and product sales in the current quarter. Revenue Recognition The Company recognizes revenue when amounts are realized or realizable and earned. Revenue is considered realizable and earned when persuasive evidence an arrangement exists, title to product and associated risk of loss has passed to the customer, the price is fixed or determinable, collection of the amounts due are reasonably assured and the Company has no further performance obligations. Net Product Sales The Company’s net product sales consist solely of sales of Galafold for the treatment of Fabry disease in the EU. The Company has recorded revenue on sales where Galafold is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold are generally received from pharmacies and the ultimate payor is typically a government authority. The Company records revenue net of estimated third party discounts and rebates. Allowances are recorded as a reduction of revenue at the time revenues from product sales are recognized. These allowances are adjusted to reflect known changes in factors and may impact such allowances in the quarter those changes are known. Collaboration Revenue In multiple element arrangements, revenue is allocated to each separate unit of accounting and each deliverable in an arrangement is evaluated to determine whether it represents separate units of accounting. A deliverable constitutes a separate unit of accounting when it has standalone value and there is no general right of return for the delivered elements. In instances when the aforementioned criteria are not met, the deliverable is combined with the undelivered elements and the allocation of the arrangement consideration and revenue recognition is determined for the combined unit as a single unit of accounting. Allocation of the consideration is determined at arrangement inception on the basis of each unit’s relative selling price. In instances where there is determined to be a single unit of accounting, the total consideration is applied as revenue for the single unit of accounting and is recognized over the period of inception through the date where the last deliverable within the single unit of accounting is expected to be delivered. The Company’s current revenue recognition policies provide that, when a collaboration arrangement contains multiple deliverables, such as license and research and development services, the Company allocates revenue to each separate unit of accounting based on a selling price hierarchy. The selling price hierarchy for a deliverable is based on (i) its vendor specific objective evidence (“VSOE”) if available, (ii) third party evidence (“TPE”) if VSOE is not available, or (iii) best estimated selling price (“BESP”) if neither VSOE nor TPE is available. The Company would establish the VSOE of selling price using the price charged for a deliverable when sold separately. The TPE of selling price would be established by evaluating largely similar and interchangeable competitor products or services in standalone sales to similarly situated customers. The BESP would be established considering internal factors such as an internal pricing analysis or an income approach using a discounted cash flow model. The Company also considers the impact of potential future payments it makes in its role as a vendor to its customers and evaluates if these potential future payments could be a reduction of revenue from that customer. If the potential future payments to the customer are: · a payment for an identifiable benefit; · the identifiable benefit is separable from the existing relationship between the Company and its customer; · the identifiable benefit can be obtained from a party other than the customer; and · the Company can reasonably estimate the fair value of the identifiable benefit then the payments are accounted for separate from the revenue received from that customer. If, however, all these criteria are not satisfied, then the payments are treated as a reduction of revenue from that customer. If the Company determines that any potential future payments to its customers are to be considered as a reduction of revenue, it must evaluate if the total amount of revenue to be received under the arrangement is fixed and determinable. If the total amount of revenue is not fixed and determinable due to the uncertain nature of the potential future payments to the customer, then any customer payments cannot be recognized as revenue until the total arrangement consideration becomes fixed and determinable. The reimbursements for research and development costs under collaboration agreements that meet the criteria for revenue recognition are included in Research Revenue and the costs associated with these reimbursable amounts are included in research and development expenses. In order to determine the revenue recognition for contingent milestones, the Company evaluates the contingent milestones using the criteria as provided by the Financial Accounting Standards Boards (“FASB”) guidance on the milestone method of revenue recognition at the inception of a collaboration agreement. The criteria requires that (i) the Company determines if the milestone is commensurate with either its performance to achieve the milestone or the enhancement of value resulting from the Company’s activities to achieve the milestone, (ii) the milestone be related to past performance, and (iii) the milestone be reasonable relative to all deliverable and payment terms of the collaboration arrangement. If these criteria are met then the contingent milestones can be considered as substantive milestones and will be recognized as revenue in the period that the milestone is achieved. Inventories and Cost of Cost of Goods Sold Until regulatory approval of Galafold, the Company expensed all manufacturing costs as research and development expense. Upon regulatory approval, the Company began capitalizing costs related to the purchase and manufacture of Galafold. Inventories are stated at the lower of cost or market determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of product sales in the consolidated statements of operations. Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as estimated royalties payable. A portion of the inventory available for sale was expensed as research and development costs prior to regulatory approval and as such the cost of goods sold and related gross margins are not necessarily indicative of future cost of goods sold and gross margin. Fair Value Measurements The Company records certain asset and liability balances under the fair value measurements as defined by the FASB guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity’s own assumptions that market participants assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Contingent Liabilities On an ongoing basis, the Company may be involved in various claims, and legal proceedings. On a quarterly basis, the Company reviews the status of each significant matter and assesses its potential financial exposure. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount can be reasonably estimated, the Company will accrue a liability for the estimated loss. Because of uncertainties related to claims and litigation, accruals will be based on the Company’s best estimates based on available information. On a periodic basis, as additional information becomes available, or based on specific events such as the outcome of litigation or settlement of claims, the Company may reassess the potential liability related to these matters and may revise these estimates, which could result in a material adverse adjustments to the Company’s operating results. New Accounting Pronouncements In August 2016, the FASB issued ASU 2016-15, Statement of Cash Flows (Topic 230): Classification of Certain Cash Receipts and Cash Payments. This Accounting Standards Update addresses the following eight specific cash flow issues including Debt prepayment or debt extinguishment costs, contingent consideration payments made after a business combination and separately identifiable cash flows and application of the predominance principle. The amendments in this ASU apply to all entities. The amendments in this Update are effective for public business entities for fiscal years beginning after December 15, 2017, and interim periods within those fiscal years. Early adoption is permitted, including adoption in an interim period. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In May 2016, the FASB issued ASU 2016-12, Revenue from Contracts with Customers (Topic 606): Narrow-Scope Improvements and Practical Expedients. The amendments address narrow-scope improvements to the guidance on collectability, noncash consideration, and completed contracts at transition. Additionally, the amendments provide a practical expedient for contract modifications at transition and an accounting policy election related to the presentation of sales taxes and other similar taxes collected from customers. These amendments are effective at the same date that Topic 606 is effective. Topic 606 is effective for public entities for annual reporting periods beginning after December 15, 2017, including interim reporting periods therein (i.e., January 1, 2018, for a calendar year entity). This Accounting Standards Update is the final version of Proposed Accounting Standards Update 2015-230—Revenue from Contracts with Customers (Topic 606)—Narrow-Scope Improvements and Practical Expedients, which has been deleted. The Company will adopt the new ASU on January 1, 2018. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In April 2016, the FASB issued ASU 2016-10, Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing. The amendments clarify the following two aspects of Topic 606: (a) identifying performance obligations; and (b) the licensing implementation guidance. The amendments do not change the core principle of the guidance in Topic 606. The effective date and transition requirements for the amendments are the same as the effective date and transition requirements in Topic 606. Public entities should apply the amendments for annual reporting periods beginning after December 15, 2017, including interim reporting periods therein (i.e., January 1, 2018, for a calendar year entity). Early application for public entities is permitted only as of annual reporting periods beginning after December 15, 2016, including interim reporting periods within that reporting period. 2018. The Company will adopt the new ASU on January 1, 2018. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In March 2016, the FASB issued ASU 2016-09, Compensation - Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting . The amendments are intended to improve the accounting for employee share-based payments and affect all organizations that issue share-based payment awards to their employees. Several aspects of the accounting for share-based payment award transactions are simplified, including: ( a ) income tax consequences; ( b ) classification of awards as either equity or liabilities; and ( c ) classification on the statement of cash flows. For public companies, the amendments are effective for annual periods beginning after December 15, 2016, and interim periods within those annual periods. Early adoption is permitted for any organization in any interim or annual period. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In March 2016, the FASB issued ASU 2016-08, Revenue from Contracts with Customers (Topic 606): Principal versus Agent Considerations (Reporting Revenue Gross versus Net) . The amendments relate to when another party, along with the entity, is involved in providing a good or service to a customer. Topic 606 Revenue from Contracts with Customers requires an entity to determine whether the nature of its promise is to provide that good or service to the customer (i.e., the entity is a principal) or to arrange for the good or service to be provided to the customer by the other party (i.e., the entity is an agent). The amendments are intended to improve the operability and understandability of the implementation guidance on principal versus agent considerations. The effective date and transition of these amendments is the same as the effective date and transition of ASU 2014-09, Revenue from Contracts with Customers (Topic 606). Public entities should apply the amendments in ASU 2014-09 for annual reporting periods beginning after December 15, 2017, including interim reporting periods therein (i.e., January 1, 2018, for a calendar year entity). The Company will implement the new ASU in the first quarter of 2018. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) . This update requires the recognition of lease assets and lease liabilities on the balance sheet for all lease obligations and disclosing key information about leasing arrangements. This update requires the recognition of lease assets and lease liabilities by lessees for those leases classified as operating leases under previous generally accepted accounting principles. This update will be effective for the Company for all annual and interim periods beginning after December 15, 2018, including interim periods within those fiscal years. Early application is permitted for all public business entities and all nonpublic business entities upon issuance. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. |
Cash, Money Market Funds and Ma
Cash, Money Market Funds and Marketable Securities | 9 Months Ended |
Sep. 30, 2016 | |
Cash, Money Market Funds and Marketable Securities | |
Cash, Money Market Funds and Marketable Securities | Note 3. As of September 30, 2016, the Company held $33.1 million in cash and cash equivalents and $179.3 million in available-for-sale securities which are reported at fair value on the Company’s balance sheet. Unrealized gains and losses are reported within accumulated other comprehensive income/ (loss) in the statements of comprehensive loss. If a decline in the fair value of a marketable security below the Company’s cost basis is determined to be other than temporary, such marketable security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in earnings as an impairment charge. To date, only temporary impairment adjustments have been recorded. The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds both of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as many of these securities are either government backed or of the highest credit rating. Investments that have original maturities or greater than 3 months but less than 1 year are classified as short-term and investments with maturities that are greater than 1 year are classified as long-term. The Company transacts business in various foreign countries and therefore, is subject to risk of foreign currency exchange rate fluctuations. As such, in June 2016 the Company entered into a forward contract to economically hedge transactional exposure associated with commitments arising from trade accounts payable denominated in a currency other than the functional currency of the respective operating entity. The Company does not designate these forward contracts as hedging instruments under applicable accounting guidance and, therefore, changes in fair value are recorded as other income (expense) in the Consolidated Statements of Operations, with the corresponding liability in current liabilities on the Consolidated Balance Sheet. For the three and nine months ended September 30, 2016, the Company recognized a gain of $22 thousand and a loss of $324 thousand related to the derivative instruments not designated as hedging instruments in other income (expense) in the Consolidated Statements of Operations and the corresponding liability of $324 thousand is recorded as other current liability in the Consolidated Balance Sheets. Cash and available-for-sale securities are all current unless mentioned otherwise and consisted of the following as of September 30, 2016 and December 31, 2015 (in thousands): As of September 30, 2016 Cost Unrealized Unrealized Fair Value Cash balances $ $ — $ — $ Corporate debt securities ) Commercial paper — Certificate of deposit — — $ $ $ ) $ Included in cash and cash equivalents $ $ — $ — $ Included in marketable securities $ $ $ ) $ Total cash and marketable securities $ $ $ ) $ As of December 31, 2015 Cost Unrealized Unrealized Fair Value Cash balances $ $ — $ — $ Corporate debt securities ) Commercial paper — Certificate of deposit — — $ $ $ ) $ Included in cash and cash equivalents $ — — $ Included in marketable securities ) Total cash and marketable securities $ $ $ ) $ For the nine months ended September 30, 2016 and the year ended December 31, 2015, there were no realized gains or losses. The cost of securities sold is based on the specific identification method. Unrealized loss positions in the available for sale securities as of September 30, 2016 and December 31, 2015 reflect temporary impairments that have not been recognized and have been in a loss position for less than twelve months and as such are recognized in other comprehensive gain/ (loss). The fair value of these available for sale securities in unrealized loss positions was $60.3 million and $118.5 million as of September 30, 2016 and December 31, 2015, respectively. The Company holds available-for-sale investment securities which are reported at fair value on the Company’s balance sheet. Unrealized holding gains and losses are reported within accumulated other comprehensive income (“AOCI”) in the Statements of Comprehensive Loss. |
Inventories
Inventories | 9 Months Ended |
Sep. 30, 2016 | |
Inventories | |
Inventories | Note 4. Inventories consist of work in process and finished goods related to the manufacture of Galafold. The following table summarizes the components of inventories at September 30, 2016 (in thousands): (Dollars in thousands) September 30, 2016 Work-in-process Finished goods Total inventories $ There were no inventories on-hand as of December 31, 2015. Inventory manufactured prior to commercialization was expensed to research and development. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity, as well as product shelf-life. In evaluating the recoverability of inventories produced, the Company considers the probability that revenue will be obtained from the future sale of the related inventory. Inventory becomes obsolete when it has aged past its shelf-life, cannot be recertified and is no longer usable or able to be sold, or the inventory has been damaged. In such instances, a full reserve is taken against such inventory. Expired inventory is disposed of and the related costs are recognized as cost of product sales in the consolidated statement of operations. There have been no write-downs of inventory from the time inventory was first capitalized. |
Acquisitions
Acquisitions | 9 Months Ended |
Sep. 30, 2016 | |
Acquisitions | |
Acquisitions | Note 5. Acquisition of Miamed, Inc On July 5, 2016, the Company entered into an Agreement and Plan of Merger (the “MiaMed Agreement”) with MiaMed, Inc., (“MiaMed”). MiaMed is a pre-clinical biotechnology company focused on developing protein replacement therapy for CDKL5 and related diseases. Under the terms of the MiaMed Agreement, the former holders of MiaMed’s capital stock received an aggregate of $6.5 million, comprised of (i) approximately $1.8 million in cash (plus MiaMed’s cash and cash equivalents at closing and less any of MiaMed’s unpaid third-party fees and expenses related to the transaction), and (ii) 825,603 shares of the Company’s Common Stock. In addition, the Company also agreed to pay up to an additional $83.0 million in connection with the achievement of certain clinical, regulatory and commercial milestones, for a potential aggregate deal value of $89.5 million. The Company evaluated the transaction based on the guidance of ASC 805 , Business Combinations and concluded that it only acquired inputs and did not acquire any processes. The Company will need to develop its own processes in order to produce an output. Therefore, the Company accounted the transaction as an asset acquisition and accordingly $6.5 million was expensed to research and development. Acquisition of Scioderm, Inc. On September 30, 2015, the Company acquired Scioderm, a privately-held biopharmaceutical company focused on developing innovative therapies for treating the rare disease EB. The acquisition leverages the Scioderm development team’s EB expertise with the Company’s global clinical infrastructure to advance SD-101 toward regulatory approvals and the Company’s commercial, patient advocacy, and medical affairs infrastructure to support a successful global launch. The acquisition of Scioderm was accounted for as a purchase of a business in accordance with FASB Accounting Standard Codification 805 Business Combinations . The Company acquired Scioderm with cash and stock. At closing, the Company paid Scioderm stockholders, option holders, and warrant holders approximately $223.9 million, of which approximately $141.1 million was paid in cash and approximately $82.8 million was paid through the issuance of approximately 5.9 million newly issued shares of the Company. The Company had agreed to pay up to an additional $361 million to Scioderm stockholders, option holders, and warrant holders upon achievement of certain clinical and regulatory milestones, and $257 million to Scioderm stockholders, option holders, and warrant holders upon achievement of certain sales milestones. If SD-101 is approved, EB qualifies as a rare pediatric disease under The Food and Drug Administration Safety and Innovation Act (“FDSIA”) and the Company will request a Priority Review Voucher (“PRV”) under the FDSIA, if available. If the PRV is obtained and subsequently sold, the Company will pay Scioderm stockholders, option holders, and warrant holders the lesser of $100 million in the aggregate or 50% of the proceeds of such sale. If the Company obtains the PRV and has not entered into an agreement to sell or otherwise transfer to a third party the PRV within one year of its receipt, the shareholders’ agent may appoint a financial advisor to conduct a process to sell the PRV. If the Company determines in its sole discretion to use the PRV, the Company shall give the shareholders’ agent written notice thereof and shall pay to the Scioderm stockholders, option holders, and warrant holders $100 million. The inability to sell the PRV after complying with the provisions, shall not give rise to any payment. The fair value of the contingent consideration payments on the acquisition date was $259.0 million. This was an estimate based on significant inputs that are not observable in the market, referred to as Level 3 inputs. Key assumptions included a range of discount rates between 0.4% and 1.1% as interpolated from the U.S. Treasury constant maturity yield curve over the time frame for clinical and regulatory milestones and a range of discount rates between 1.0% and 2.2% for revenue-based milestones. The range of outcomes and assumptions used to develop these estimates have been updated to better reflect the probability of certain milestone outcomes and updated timelines related to clinical development and anticipated approval assumptions as of September 30, 2016 without limitation, the $5 million milestone paid in the second quarter and milestone payments projected for 2017 (See “— Note 9. Assets and Liabilities Measured at Fair Value”, for additional discussion regarding fair value measurements of the contingent acquisition consideration payable). In April 2016, while the total clinical and regulatory approval milestone payments remain unchanged at $361 million, the allocation between the clinical and regulatory approval milestone payments were revised as follows: clinical milestones of up to $81 million and regulatory approval milestones of up to $280 million. The commercial milestone payments of up to $257 million remained unchanged. The Company determined the fair value of the contingent consideration to be $262.2 million at September 30, 2016, of which $56.0 million is payable in the next twelve months, resulting in an increase in the contingent consideration payable and related expense of $9.4 million for the nine months ended September 30, 2016. The expense is recorded in the Consolidated Statement of Operations as the change within fair value of contingent consideration payable. See “— Note 9. Assets and Liabilities Measured at Fair Value”, for additional discussion regarding fair value measurements of the contingent acquisition consideration payable. For additional information, see “— Note 6. Goodwill and Intangible Assets.” The purchase price allocation was subject to completion of our analysis of the fair value of the assets and liabilities as of the effective date of the acquisition. The final valuation was completed as of December 31, 2015. A substantial portion of the assets acquired consisted of intangible assets related to SD-101. The Company determined that the estimated acquisition-date fair value of the indefinite lived IPR&D related to the SD-101 was $463.7 million. Acquisition of Callidus Biopharma, Inc. In November 2013, the Company acquired Callidus a privately-held biologics company focused on developing best-in-class ERTs for LSDs with its lead ERT ATB200 for Pompe disease in late preclinical development. The acquisition of the Callidus assets and technology complements the Company’s CHART™ platform for the development of next-generation ERTs. The fair value of the contingent acquisition consideration payments on the acquisition date was $10.6 million and was estimated by applying a probability-based income approach utilizing an appropriate discount rate. This estimation was based on significant inputs that are not observable in the market, referred to as Level 3 inputs. As of September 30, 2016, the range of outcomes and assumptions used to develop these estimates has changed to better reflect the probability of certain milestone outcomes; see “— Note 9. Assets and Liabilities Measured at Fair Value”, for additional discussion regarding fair value measurements of the contingent acquisition consideration payable. The Company determined the fair value of the contingent consideration to be $10.0 million at September 30, 2016, of which $9.6 million relates to ATB-200 Pompe program. The change in fair value of contingent consideration payable is recorded in the Consolidated Statement of Operations. All of the contingent consideration is payable beyond the next twelve months. During the second quarter of 2016, the Company reached the first clinical milestone, which was the dosing of the first patient in a Phase 1 or 2 study. The milestone for this event was $6.0 million which was paid in Company stock during the second quarter of 2016, resulting in $6.1 million impact on stockholder’s equity. For further information, see “— Note 6. Goodwill and Intangible Assets.” |
Goodwill and Intangible Assets
Goodwill and Intangible Assets | 9 Months Ended |
Sep. 30, 2016 | |
Goodwill and Intangible Assets | |
Goodwill and Intangible Assets | Note 6. In connection with the acquisitions discussed in “—Note 5. Acquisitions”, the Company has recognized goodwill of $197.8 million. The following table represents the changes in goodwill for the nine months ended September 30, 2016: ( in millions) Balance at December 31, 2015 $ Change in goodwill — Balance at September 30, 2016 $ In connection with the acquisitions discussed in “—Note 5. Acquisitions,” the Company recognized IPR&D of $486.7 million. Intangible assets related to IPR&D assets are considered to be indefinite-lived until the completion or abandonment of the associated research and development efforts. During the period the assets are considered indefinite-lived, they will not be amortized but will be tested for impairment on an annual basis and between annual tests if the Company becomes aware of any events occurring or changes in circumstances that would indicate a reduction in the fair value of the IPR&D assets below their respective carrying amounts. The following table represents the changes in IPR&D for the nine months ended September 30, 2016: ( in millions) Balance at December 31, 2015 $ Change in IPR&D — Balance at September 30, 2016 $ Goodwill and intangible assets are assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. If it is determined that the full carrying amount of an asset is not recoverable, an impairment loss is recorded in the amount by which the carrying amount of the asset exceeds its fair value. For the nine months ended September 30, 2016, there were no indicators of impairment. |
Debt Instruments and Related Pa
Debt Instruments and Related Party Transactions | 9 Months Ended |
Sep. 30, 2016 | |
Debt Instruments and Related Party Transactions | |
Debt Instruments and Related Party Transactions | Note 7. In October 2015, the Company entered into a Note and Warrant Purchase Agreement (the “October 2015 Purchase Agreement”) with Redmile Capital Fund, LP and certain of its affiliates, whereby it sold, on a private placement basis, (a) $50.0 million aggregate principal amount of its unsecured promissory notes (“Notes”) and (b) five-year warrants (“Warrants”) for approximately 1.3 million shares of Common Stock. The payment terms under the purchase agreement contains two installments, the first $15.0 million in October 2017 and the balance $35.0 million in October 2020. Interest was payable at 4.1% on a monthly basis over the term of the loan. The promissory notes are recorded as due to related party on the consolidated balance sheets. Due to the embedded redemption (put and/or call) features in the note agreement, it was determined that the fair value of the warrants should be bifurcated from the value of the notes payable and recorded as a debt discount. The relative fair value of the warrants and the debt discount as related to the October 2015 purchase agreement was determined to be $8.8 million. On February 19, 2016, the Company entered into a Note and Warrant Purchase Agreement (the “February 2016 Purchase Agreement”) with Redmile for an aggregate amount of up to $75.0 million. The Company has agreed with Redmile that in full consideration of the purchase price for the notes issued under the February 2016 Purchase Agreement, Redmile surrendered for cancellation all notes and warrants acquired from the October 2015 Purchase Agreement and the Company paid Redmile the interest accrued thereunder. Upon entering the February 2016 Agreement Redmile beneficially owned approximately 10% of the Company’s outstanding shares of Common Stock and warrants. As such the promissory notes are presented as due to related party on the consolidated balance sheets. Pursuant to the February 2016 agreement, at closing, it sold, on a private placement basis (a) $50.0 million aggregate principal amount of unsecured promissory notes (“Initial Notes”) and (b) five year warrants to purchase up to 37 shares of the Company’s Common Stock for every $1,000 of the principal amount of Initial Notes purchased (“Initial Warrants”), for an aggregate of up to 1,850,000 shares of Common Stock issuable under the Initial Warrants. The payment terms contain two installments, the first $15.0 million in October 2017 and the balance $35.0 million in October 2021. The interest rate is 3.875% and payable upon of maturity. This transaction was accounted for as a debt modification in accordance with ASC 470-50. The incremental fair value between the warrants that were cancelled and the February issued warrants of $3.5 million was recorded as additional unamortized debt discount on the balance sheet and added to the prior warrant balance within equity. The debt discount will be amortized over the life of the Initial Notes using the effective interest rate method. On June 30, 2016, following the positive CHMP opinion for migalastat in Europe and the subsequent EC marketing approval, the Company entered into the Amended Purchase Agreement with Redmile, which joined GCM to the February 2016 Purchase Agreement. There was no change to the previously issued debt. Pursuant to the Amended Purchase Agreement, the Company sold an additional $30.0 million unsecured promissory notes and five year warrants to purchase up to 42 shares of the Company’s Common Stock for every $1,000 of the principal amount of additional Notes purchased (“Additional Warrants”), for an aggregate of up to 1,260,000 shares of Common Stock. The $30.0 million payment is due in October 2021. The interest rate is 3.875% and payable upon of maturity. The fair value of the warrants was determined to be $3.8 million and recorded as a debt discount. The fair value of the warrants were calculated utilizing the Black-Scholes valuation model using the following six inputs: (1) the closing price of the Company’s Common Stock on the day of evaluation of $5.46; (2) the exercise price of the warrants of $7.06; (3) the remaining term of the warrants of 5 years; (4) the volatility of the Company’s Common Stock for the five year term of 86.02%; (5) the annual rate of dividends of 0%; and (6) the risk-free rate of return of 1.01%. The outstanding debt as of September 30, 2016 between Redmile and GCM as of September 30, 2016 is as follows (in thousands): Creditor Gross amount of Net unamortized Net carrying RedMile $ $ ) $ GCM ) Total Debt $ $ ) $ The debt discount amortization for the three and nine months ended September 30, 2016 was $0.7 million and $1.7 million, respectively. As of September 30, 2016, the total warrants were recorded at $16.1 million. See “—Note 8. Stockholders’ Equity” for more details. |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2016 | |
Stockholders' Equity | |
Stockholders' Equity | Note 8. Stockholders’ Equity Common Stock and Warrants As of September 30, 2016, the Company was authorized to issue 250 million shares of Common Stock. Dividends on Common Stock will be paid when, and if, declared by the board of directors. Each stockholder is entitled to vote on all matters that are appropriate for stockholder voting and is entitled to one vote for each share held. Beginning in April 2016 and through July 2016, the Company sold 15.0 million shares of Common Stock under an at-the-market (“ATM”) equity program with Cowen and Company, LLC (“Cowen”) acting as sales agent. Cowen was compensated at a fixed commission rate up to 3.0%. The ATM sales agreement resulted in net proceeds of $97.1 million, after Cowen’s commission of $2.7 million and other expenses of $0.2 million. The Company has completed all sales under the ATM equity program. As discussed in “—Note 7. Debt instruments and Related Party Transactions,” the Company issued approximately 1.8 million and 1.3 million of warrants in February 2016 and June 2016, respectively. The total outstanding warrants as of September 30, 2016 is as follows (in thousands): Creditor Warrant shares Warrant fair RedMile $ GCM Total warrants $ The closing balance of the warrants was $16.1 million as of September 30, 2016 on the Consolidated Balance Sheets. Nonqualified Cash Plan The Company’s Deferral Plan, (the “Deferral Plan”) provides certain key employees and members of the Board of Directors as selected by the Compensation Committee, with an opportunity to defer the receipt of such participant’s base salary, bonus and director’s fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code of 1986, as amended. Deferred compensation amounts under the Deferral Plan as of September 30, 2016 were approximately $1.3 million, as compared to $0.7 million on December 31, 2015 and are included in other long-term liabilities. Deferral Plan assets as of September 30, 2016 were $1.3 million, as compared to $0.7 million as of December 31, 2015 and are classified as trading securities. The Deferred Plan assets are recorded at fair value with changes in the investments’ fair value recognized in the period they occur. The income from investment for the three and nine months ended September 30, 2016 and 2015 was de minimis. Unrealized gain approximated $30 thousand and $37 thousand for the three and nine months ended September 30, 2016, respectively as compared to unrealized loss of $54 thousand and $64 thousand for the three and nine months ended September 30, 2015, respectively. Equity Incentive Plan Stock Option Grants The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions: Three months ended Nine months ended 2016 2015 2016 2015 Expected stock price volatility % % % % Risk free interest rate % % % % Expected life of options (years) Expected annual dividend per share $ $ $ $ A summary of the Company’s stock options for the nine months ended September 30, 2016 is as follows: Number of Weighted Weighted Aggregate Intrinsic Balance at December 31, 2015 $ Options granted $ Options exercised ) $ Options forfeited ) $ Balance at September 30, 2016 $ 7.5 years $ Vested and unvested expected to vest September 30, 2016 $ 7.4 years $ Exercisable at September 30, 2016 $ 5.9 years $ As of September 30, 2016, the total unrecognized compensation cost related to non-vested stock options granted was $35.8 million and is expected to be recognized over a weighted average period of 2.9 years. Restricted Stock Units A summary of non-vested Restricted Stock Units (“RSU”) activity under the Company’s Amended and Restated 2007 Equity Incentive Plan for the nine months ended September 30, 2016 is as follows: Number of Shares Weighted Weighted Aggregate Intrinsic Non-vested units as of December 31, 2015 $ Granted $ Vested ) $ Forfeited ) $ Non-vested units as of September 30, 2016 $ $ — Non-vested units expected to vest at September 30, 2016 $ $ — For the nine months ended September 30, 2016, 199,266 of the RSUs vested and all non-vested units are expected to vest over their normal term. As of September 30, 2016, there was $4.8 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of 2.69 year. Compensation Expense Related to Equity Awards The following table summarizes information related to compensation expense recognized in the statements of operations related to the equity awards (in thousands): Three Months Nine Months Ended September 30, Ended September 30, 2016 2015 2016 2015 Equity compensation expense recognized in: Research and development expense $ $ $ $ General and administrative expense Total equity compensation expense $ $ $ $ |
Assets and Liabilities Measured
Assets and Liabilities Measured at Fair Value | 9 Months Ended |
Sep. 30, 2016 | |
Assets and Liabilities Measured at Fair Value | |
Assets and Liabilities Measured at Fair Value | Note 9. Assets and Liabilities Measured at Fair Value The Company’s financial assets and liabilities are measured at fair value and classified within the fair value hierarchy, which is defined as follows: Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly. Level 3 — Inputs that are unobservable for the asset or liability. A summary of the fair value of the Company’s assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2016, are identified in the following table (in thousands): Level 1 Level 2 Total Assets: Cash/ money market funds $ $ — $ Corporate debt securities — Commercial paper — Certificate of deposit — Market exchanged mutual funds — $ $ $ Level 2 Level 3 Total Liabilities: Contingent consideration payable — $ $ Derivative liability $ — Deferred compensation plan liability $ — $ $ $ $ A summary of the fair value of the Company’s assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2015, are identified in the following table (in thousands): Level 1 Level 2 Total Assets: Cash/ money market funds $ $ — $ Corporate debt securities — Commercial paper — Certificate of deposit — Market exchanged mutual funds $ $ $ Level 2 Level 3 Total Liabilities: Contingent consideration payable — Deferred compensation plan liability — $ $ $ Cash, Money Market Funds and Marketable Securities The Company classifies its cash and money market funds within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities. No changes in valuation techniques or inputs occurred during the nine months ended September 30, 2016. No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the nine months ended September 30, 2016. Note Payable to Related Party and GCM In connection with the notes payable to Redmile, as disclosed in “—Note 7. Debt Instruments and Related Party Transactions”, and Warrants as disclosed in “— Note 8. Stockholders’ Equity,” the Company recorded the notes as a liability of $66.0 million on an amortized cost basis. The warrants issued in connection with the Amended Purchase Agreement were determined to be a component of equity based on the current accounting guidance. As such, these warrants which are considered Level 3 instruments were valued at the issuance date using the Black-Scholes valuation model using the following six inputs: (1) the closing price of the Company’s Common Stock on the day of evaluation of $5.46; (2) the exercise price of the warrants of $7.06; (3) the remaining term of the warrants of 5 years; (4) the volatility of the Company’s Common Stock for the five year term of 86.02%; (5) the annual rate of dividends of 0%; and (6) the risk-free rate of return of 1.01%. The Black-Scholes value of the warrants was $3.8 million. As of September 30, 2016, the warrants are recorded at $16.1 million and the notes at $66.0 million, net of discount of $14.0 million. Contingent Consideration Payable The contingent consideration payable resulted from the acquisitions of Scioderm and Callidus, as discussed in “—Note 5. Acquisitions.” The most recent valuation was determined using a probability weighted discounted cash flow valuation approach. Using this approach, expected future cash flows are calculated over the expected life of the agreement, are discounted, and then exercise scenario probabilities are applied. The valuation is performed quarterly. Gains and losses are included in the statement of operations. As discussed in “—Note 5. Acquisitions,” on July 5, 2016, the Company entered into the MiaMed Agreement with MiaMed. MiaMed is a pre-clinical biotechnology company focused on developing protein replacement therapy (“product candidate”) for CDKL5 and related diseases. Under the terms of the MiaMed Agreement, the Company agreed to pay up to an additional $83.0 million in connection with the achievement of certain clinical, regulatory and commercial milestones, for a potential aggregate deal value of $89.5 million. The MiaMed Agreement was accounted for as an asset acquisition and as such the Company determined that a liability for future milestone payments is not required to be recorded until the actual contingencies are met and will be recorded to research and development expenses when the contingency is resolved. The contingent consideration payable for Scioderm and Callidus has been classified as a Level 3 recurring liability as its valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions were used for the various inputs to the valuation approach the estimated fair value could be significantly higher or lower than the fair value the Company determined. The Company may be required to record losses in future periods, including expenses related to CDKL5. The following significant unobservable inputs were used in the valuation of the contingent consideration payable to former Scioderm stockholders: Contingent Fair value as of Valuation Unobservable Input Range Clinical and regulatory milestones $238.3 million Probability Discount rate 0.5%-3.1% Revenue-based milestones $23.9 million Monte Carlo Revenue volatility 58% The following significant unobservable inputs were used in the valuation of the contingent consideration payable to former Callidus shareholders for the ATB-200 Pompe program: Contingent Fair value as of Valuation Technique Unobservable Input Range Clinical and regulatory milestones $9.6 million Probability weighted discounted cash flow Discount rate 10.5% Contingent consideration liabilities are remeasured to fair value each reporting period using projected revenues, discount rates, probabilities of payment and projected payment dates. Projected contingent payment amounts related to clinical and regulatory based milestones are discounted back to the current period using a discounted cash flow model. Revenue-based payments are valued using a monte-carlo valuation model, which simulates future revenues during the earn-out-period using management’s best estimates. Projected revenues are based on our most recent internal operational budgets and long-range strategic plans. Increases in projected revenues and probabilities of payment may result in higher fair value measurements. Increases in discount rates and the time to payment may result in lower fair value measurements. Increases or decreases in any of those inputs together, or in isolation, may result in a significantly lower or higher fair value measurement. There is no assurance that any of the conditions for the milestone payments will be met. The following table shows the change in the balance of contingent consideration payable for the nine months ended September 30, 2016 and 2015, respectively (in thousands): Three months Nine months 2016 2015 2016 2015 Balance, beginning of the period $ $ $ $ Additions, from business acquisitions — — Payment of contingent consideration in cash — — ) — Payment of contingent consideration in stock — — ) — Change in fair value change during the period, included in Statement of Operations ) Balance, end of the period $ $ $ $ Deferred Compensation Plan- Investment and Liability The Company considers its investments in marketable securities, as available-for-sale and classifies these assets and related liability within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities. Foreign Currency Exchange Rate Exposure The Company transacts business in various foreign countries and therefore, is subject to risk of foreign currency exchange rate fluctuations. As such, in June 2016, the Company entered into a forward contract to economically hedge transactional exposure associated with commitments arising from trade accounts payable denominated in a currency other than the functional currency of the respective operating entity. The Company did not designate this forward contract as a hedging instrument under applicable accounting guidance and, therefore, the change in fair value is recorded in the Consolidated Statements of Operations. The forward contract settles in monthly installments with the final installment settlement in June 2017. There were no outstanding forward contracts at December 31, 2015. For the three and nine months ended September 30, 2016, the Company recognized a gain of $22 thousand and a loss of $324 thousand, respectively, related to the derivative instruments not designated as hedging instruments in the Consolidated Statements of Operations and the corresponding liability of $324 thousand is recorded as other current liability in the Consolidated Balance Sheet. The impact of gains and losses on foreign exchange contracts not designated as hedging instruments related to changes in the fair value of assets and liabilities denominated in foreign currencies are generally offset by net foreign exchange gains and losses, which are also included on the Consolidated Statements of Operations in other income (expense), net for all periods presented. When the Company enters into foreign exchange contracts not designated as hedging instruments to mitigate the impact of exchange rate volatility in the translation of foreign earnings, gains and losses will generally be offset by fluctuations in the U.S. Dollar translated amounts of each Income Statement account in current and/or future periods. |
Restructuring Charges
Restructuring Charges | 9 Months Ended |
Sep. 30, 2016 | |
Restructuring Charges | |
Restructuring Charges | Note 10. Restructuring Charges In December 2013, the Company initiated and completed a facilities consolidation effort, closing one of its leased locations in San Diego, CA. The Company recorded a charge of $0.7 million related to the net present value of the net future minimum lease payments at the cease-use date. The following table summarizes the restructuring charges and utilization for the nine months ended September 30, 2016 (in thousands): Balance as of Charges Cash Payments Fair Value Balance as of Facilities consolidation $ $ — $ ) $ $ — As of September 30, 2016, the lease had expired and the Company completed the restructuring process. |
Basic and Diluted Net Loss per
Basic and Diluted Net Loss per Common Share | 9 Months Ended |
Sep. 30, 2016 | |
Basic and Diluted Net Loss per Common Share | |
Basic and Diluted Net Loss per Common Share | Note 11. Basic and Diluted Net Loss per Common Share The Company calculates net loss per share as a measurement of the Company’s performance while giving effect to all dilutive potential common shares that were outstanding during the reporting period. The Company has a net loss for all periods presented; accordingly, the inclusion of Common Stock options and warrants would be anti-dilutive. Therefore, the weighted average shares used to calculate both basic and diluted earnings per share are the same. The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss per common share: (In thousands, except per share Three months Ended Nine months Ended amounts) 2016 2015 2016 2015 Historical Numerator: Net loss $ ) $ ) $ ) $ ) Denominator: Weighted average common shares outstanding — basic and diluted Dilutive common stock equivalents would include the dilutive effect of common stock options, restricted stock units and warrants for common stock equivalents. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method (in thousands): As of September 30, 2016 2015 Options to purchase common stock Outstanding warrants, convertible to common stock — Unvested restricted stock units Total number of potentially issuable shares |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2016 | |
Commitments and Contingencies. | |
Commitments and Contingencies | Note 12. Commitments and Contingencies Since October 1, 2015, three purported securities class action lawsuits have been commenced in the United States District Court for New Jersey, naming as defendants the Company, its Chairman and Chief Executive Officer, and in one of the actions, its Chief Medical Officer. The lawsuits allege violations of the Securities Exchange Act of 1934 in connection with allegedly false and misleading statements made by the Company related to the regulatory approval path for migalastat. The plaintiffs seek, among other things, damages for purchasers of the Company’s Common Stock during different periods, all of which fall between March 19, 2015 and October 1, 2015. It is possible that additional suits will be filed, or allegations received from stockholders, with respect to similar matters and also naming the Company and/or its officers and directors as defendants. On May 26, 2016, the Court consolidated these lawsuits into a single action and appointed a lead plaintiff. The lead plaintiff filed a Consolidated Amended Complaint on July 11, 2016. Defendants’ motion to dismiss was fully briefed on October 28, 2016. The Company believes that it has meritorious defenses and intends to defend the lawsuits vigorously. These lawsuits and any other related lawsuits are subject to inherent uncertainties, and the actual cost will depend upon many unknown factors. The outcome of the litigation is necessarily uncertain, the Company could be forced to expend significant resources in the defense of these lawsuits and it may not prevail. On or about November 2, 2015, a derivative lawsuit was filed by an Amicus shareholder purportedly on Amicus’ behalf in the Superior Court of New Jersey, Middlesex County, Chancery Division, against the individuals who serve on the Amicus Board of Directors. Amicus itself was named as a nominal defendant. The derivative lawsuit alleged claims for breach of state law fiduciary duties, waste of corporate assets, and unjust enrichment based on allegedly false and misleading statements made by Amicus related to the regulatory approval path for migalastat HCl. On February 19, 2016, the complaint was dismissed by the Court and plaintiffs have not refiled. On or about March 3, 2016, a derivative lawsuit was filed by an Amicus shareholder purportedly on Amicus’ behalf in the Superior Court of New Jersey, Middlesex County, Chancery Division, against various officers and directors of the Company. Amicus itself is named as a nominal defendant. The derivative lawsuit alleges similar facts and circumstances as the three purported securities class action lawsuits described above and further alleges claims for breach of state law fiduciary duties, waste of corporate assets, unjust enrichment, abuse of control, and gross mismanagement based on allegedly false and misleading statements made by Amicus related to the regulatory approval path for migalastat HCl. The plaintiff seeks, among other things, to require the Amicus Board to take certain actions to reform its corporate governance procedures, including greater shareholder input and a provision to permit shareholders to nominate candidates for election to the Board, along with restitution, costs of suit and attorney’s fees. The parties have entered into a stipulation to stay the time to respond to the derivative complaint until the resolution of any motion to dismiss in the above-referenced securities action. This lawsuit and any other related lawsuits are subject to inherent uncertainties and the actual cost will depend upon many unknown factors. The outcome of the litigation is necessarily uncertain and the Company could be forced to expend significant resources in the defense of this suit, and the Company may not prevail. The Company is not currently able to estimate the possible cost to it from this matter, as this lawsuit is currently at an early stage and the Company cannot ascertain how long it may take to resolve this matter. |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2016 | |
Subsequent Events | |
Subsequent Events | Note 13. Subsequent Events The Company evaluated events that occurred subsequent to September 30, 2016 and there were no material recognized or non-recognized subsequent events during this period. |
Summary of Significant Accoun21
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2016 | |
Summary of Significant Accounting Policies | |
Basis of Presentation | Basis of Presentation The Company has prepared the accompanying unaudited consolidated financial statements in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulations S-X. Accordingly, they do not include all of the information and disclosures required by generally accepted accounting principles for complete financial statements. In the opinion of management, the accompanying unaudited financial statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s interim financial information. The accompanying unaudited consolidated financial statements and related notes should be read in conjunction with the Company’s financial statements and related notes as contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015. For a complete description of the Company’s accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2015. |
Foreign Currency Transactions | Foreign Currency Transactions The functional currency for most of our foreign subsidiaries is their local currency. For our non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of our foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of equity. The Company transacts business in various foreign countries and therefore, is subject to risk of foreign currency exchange rate fluctuations. As such, in June 2016 the Company entered into one forward contract to economically hedge transactional exposure associated with commitments arising from trade accounts payable denominated in a currency other than the functional currency of the respective operating entity. The Company does not designate this forward contract as a hedging instrument under applicable accounting guidance and, therefore, changes in fair value are recorded in the Consolidated Statements of Operations. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. |
Concentration of Credit Risk | Concentration of Credit Risk The Company’s financial instruments that are exposed to concentration of credit risk consist primarily of cash and cash equivalents and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on cash and cash equivalents or its marketable securities. The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold. The majority of the Company’s accounts receivable at September 30, 2016 have arisen from product sales in Germany. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined and the Company evaluates the creditworthiness of each customer on a regular basis. To date, the Company has not incurred any credit losses. |
Revenue Recognition | Revenue Recognition The Company recognizes revenue when amounts are realized or realizable and earned. Revenue is considered realizable and earned when persuasive evidence an arrangement exists, title to product and associated risk of loss has passed to the customer, the price is fixed or determinable, collection of the amounts due are reasonably assured and the Company has no further performance obligations. Net Product Sales The Company’s net product sales consist solely of sales of Galafold for the treatment of Fabry disease in the EU. The Company has recorded revenue on sales where Galafold is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold are generally received from pharmacies and the ultimate payor is typically a government authority. The Company records revenue net of estimated third party discounts and rebates. Allowances are recorded as a reduction of revenue at the time revenues from product sales are recognized. These allowances are adjusted to reflect known changes in factors and may impact such allowances in the quarter those changes are known. Collaboration Revenue In multiple element arrangements, revenue is allocated to each separate unit of accounting and each deliverable in an arrangement is evaluated to determine whether it represents separate units of accounting. A deliverable constitutes a separate unit of accounting when it has standalone value and there is no general right of return for the delivered elements. In instances when the aforementioned criteria are not met, the deliverable is combined with the undelivered elements and the allocation of the arrangement consideration and revenue recognition is determined for the combined unit as a single unit of accounting. Allocation of the consideration is determined at arrangement inception on the basis of each unit’s relative selling price. In instances where there is determined to be a single unit of accounting, the total consideration is applied as revenue for the single unit of accounting and is recognized over the period of inception through the date where the last deliverable within the single unit of accounting is expected to be delivered. The Company’s current revenue recognition policies provide that, when a collaboration arrangement contains multiple deliverables, such as license and research and development services, the Company allocates revenue to each separate unit of accounting based on a selling price hierarchy. The selling price hierarchy for a deliverable is based on (i) its vendor specific objective evidence (“VSOE”) if available, (ii) third party evidence (“TPE”) if VSOE is not available, or (iii) best estimated selling price (“BESP”) if neither VSOE nor TPE is available. The Company would establish the VSOE of selling price using the price charged for a deliverable when sold separately. The TPE of selling price would be established by evaluating largely similar and interchangeable competitor products or services in standalone sales to similarly situated customers. The BESP would be established considering internal factors such as an internal pricing analysis or an income approach using a discounted cash flow model. The Company also considers the impact of potential future payments it makes in its role as a vendor to its customers and evaluates if these potential future payments could be a reduction of revenue from that customer. If the potential future payments to the customer are: · a payment for an identifiable benefit; · the identifiable benefit is separable from the existing relationship between the Company and its customer; · the identifiable benefit can be obtained from a party other than the customer; and · the Company can reasonably estimate the fair value of the identifiable benefit then the payments are accounted for separate from the revenue received from that customer. If, however, all these criteria are not satisfied, then the payments are treated as a reduction of revenue from that customer. If the Company determines that any potential future payments to its customers are to be considered as a reduction of revenue, it must evaluate if the total amount of revenue to be received under the arrangement is fixed and determinable. If the total amount of revenue is not fixed and determinable due to the uncertain nature of the potential future payments to the customer, then any customer payments cannot be recognized as revenue until the total arrangement consideration becomes fixed and determinable. The reimbursements for research and development costs under collaboration agreements that meet the criteria for revenue recognition are included in Research Revenue and the costs associated with these reimbursable amounts are included in research and development expenses. In order to determine the revenue recognition for contingent milestones, the Company evaluates the contingent milestones using the criteria as provided by the Financial Accounting Standards Boards (“FASB”) guidance on the milestone method of revenue recognition at the inception of a collaboration agreement. The criteria requires that (i) the Company determines if the milestone is commensurate with either its performance to achieve the milestone or the enhancement of value resulting from the Company’s activities to achieve the milestone, (ii) the milestone be related to past performance, and (iii) the milestone be reasonable relative to all deliverable and payment terms of the collaboration arrangement. If these criteria are met then the contingent milestones can be considered as substantive milestones and will be recognized as revenue in the period that the milestone is achieved. |
Inventories and Cost of Cost of Goods Sold | Inventories and Cost of Cost of Goods Sold Until regulatory approval of Galafold, the Company expensed all manufacturing costs as research and development expense. Upon regulatory approval, the Company began capitalizing costs related to the purchase and manufacture of Galafold. Inventories are stated at the lower of cost or market determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of product sales in the consolidated statements of operations. Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as estimated royalties payable. A portion of the inventory available for sale was expensed as research and development costs prior to regulatory approval and as such the cost of goods sold and related gross margins are not necessarily indicative of future cost of goods sold and gross margin. |
Fair Value Measurements | Fair Value Measurements The Company records certain asset and liability balances under the fair value measurements as defined by the FASB guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity’s own assumptions that market participants assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. |
Contingent Liabilities | Contingent Liabilities On an ongoing basis, the Company may be involved in various claims, and legal proceedings. On a quarterly basis, the Company reviews the status of each significant matter and assesses its potential financial exposure. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount can be reasonably estimated, the Company will accrue a liability for the estimated loss. Because of uncertainties related to claims and litigation, accruals will be based on the Company’s best estimates based on available information. On a periodic basis, as additional information becomes available, or based on specific events such as the outcome of litigation or settlement of claims, the Company may reassess the potential liability related to these matters and may revise these estimates, which could result in a material adverse adjustments to the Company’s operating results. |
New Accounting Pronouncements | New Accounting Pronouncements In August 2016, the FASB issued ASU 2016-15, Statement of Cash Flows (Topic 230): Classification of Certain Cash Receipts and Cash Payments. This Accounting Standards Update addresses the following eight specific cash flow issues including Debt prepayment or debt extinguishment costs, contingent consideration payments made after a business combination and separately identifiable cash flows and application of the predominance principle. The amendments in this ASU apply to all entities. The amendments in this Update are effective for public business entities for fiscal years beginning after December 15, 2017, and interim periods within those fiscal years. Early adoption is permitted, including adoption in an interim period. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In May 2016, the FASB issued ASU 2016-12, Revenue from Contracts with Customers (Topic 606): Narrow-Scope Improvements and Practical Expedients. The amendments address narrow-scope improvements to the guidance on collectability, noncash consideration, and completed contracts at transition. Additionally, the amendments provide a practical expedient for contract modifications at transition and an accounting policy election related to the presentation of sales taxes and other similar taxes collected from customers. These amendments are effective at the same date that Topic 606 is effective. Topic 606 is effective for public entities for annual reporting periods beginning after December 15, 2017, including interim reporting periods therein (i.e., January 1, 2018, for a calendar year entity). This Accounting Standards Update is the final version of Proposed Accounting Standards Update 2015-230—Revenue from Contracts with Customers (Topic 606)—Narrow-Scope Improvements and Practical Expedients, which has been deleted. The Company will adopt the new ASU on January 1, 2018. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In April 2016, the FASB issued ASU 2016-10, Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing. The amendments clarify the following two aspects of Topic 606: (a) identifying performance obligations; and (b) the licensing implementation guidance. The amendments do not change the core principle of the guidance in Topic 606. The effective date and transition requirements for the amendments are the same as the effective date and transition requirements in Topic 606. Public entities should apply the amendments for annual reporting periods beginning after December 15, 2017, including interim reporting periods therein (i.e., January 1, 2018, for a calendar year entity). Early application for public entities is permitted only as of annual reporting periods beginning after December 15, 2016, including interim reporting periods within that reporting period. 2018. The Company will adopt the new ASU on January 1, 2018. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In March 2016, the FASB issued ASU 2016-09, Compensation - Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting . The amendments are intended to improve the accounting for employee share-based payments and affect all organizations that issue share-based payment awards to their employees. Several aspects of the accounting for share-based payment award transactions are simplified, including: ( a ) income tax consequences; ( b ) classification of awards as either equity or liabilities; and ( c ) classification on the statement of cash flows. For public companies, the amendments are effective for annual periods beginning after December 15, 2016, and interim periods within those annual periods. Early adoption is permitted for any organization in any interim or annual period. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In March 2016, the FASB issued ASU 2016-08, Revenue from Contracts with Customers (Topic 606): Principal versus Agent Considerations (Reporting Revenue Gross versus Net) . The amendments relate to when another party, along with the entity, is involved in providing a good or service to a customer. Topic 606 Revenue from Contracts with Customers requires an entity to determine whether the nature of its promise is to provide that good or service to the customer (i.e., the entity is a principal) or to arrange for the good or service to be provided to the customer by the other party (i.e., the entity is an agent). The amendments are intended to improve the operability and understandability of the implementation guidance on principal versus agent considerations. The effective date and transition of these amendments is the same as the effective date and transition of ASU 2014-09, Revenue from Contracts with Customers (Topic 606). Public entities should apply the amendments in ASU 2014-09 for annual reporting periods beginning after December 15, 2017, including interim reporting periods therein (i.e., January 1, 2018, for a calendar year entity). The Company will implement the new ASU in the first quarter of 2018. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) . This update requires the recognition of lease assets and lease liabilities on the balance sheet for all lease obligations and disclosing key information about leasing arrangements. This update requires the recognition of lease assets and lease liabilities by lessees for those leases classified as operating leases under previous generally accepted accounting principles. This update will be effective for the Company for all annual and interim periods beginning after December 15, 2018, including interim periods within those fiscal years. Early application is permitted for all public business entities and all nonpublic business entities upon issuance. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. |
Cash, Money Market Funds and 22
Cash, Money Market Funds and Marketable Securities (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Cash, Money Market Funds and Marketable Securities | |
Schedule of cash and available-for-sale securities | Cash and available-for-sale securities are all current unless mentioned otherwise and consisted of the following as of September 30, 2016 and December 31, 2015 (in thousands): As of September 30, 2016 Cost Unrealized Unrealized Fair Value Cash balances $ $ — $ — $ Corporate debt securities ) Commercial paper — Certificate of deposit — — $ $ $ ) $ Included in cash and cash equivalents $ $ — $ — $ Included in marketable securities $ $ $ ) $ Total cash and marketable securities $ $ $ ) $ As of December 31, 2015 Cost Unrealized Unrealized Fair Value Cash balances $ $ — $ — $ Corporate debt securities ) Commercial paper — Certificate of deposit — — $ $ $ ) $ Included in cash and cash equivalents $ — — $ Included in marketable securities ) Total cash and marketable securities $ $ $ ) $ |
Inventories (Tables)
Inventories (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Inventories | |
Schedule of inventories for the period | The following table summarizes the components of inventories at September 30, 2016 (in thousands): (Dollars in thousands) September 30, 2016 Work-in-process Finished goods Total inventories $ |
Goodwill and Intangible Assets
Goodwill and Intangible Assets (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Goodwill and Intangible Assets | |
Schedule of changes in goodwill | ( in millions) Balance at December 31, 2015 $ Change in goodwill — Balance at September 30, 2016 $ |
Schedule of changes in IPR&D | ( in millions) Balance at December 31, 2015 $ Change in IPR&D — Balance at September 30, 2016 $ |
Debt Instruments and Related 25
Debt Instruments and Related Party Transactions (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Debt Instruments and Related Party Transactions | |
Schedule of outstanding debt | The outstanding debt as of September 30, 2016 between Redmile and GCM as of September 30, 2016 is as follows (in thousands): Creditor Gross amount of Net unamortized Net carrying RedMile $ $ ) $ GCM ) Total Debt $ $ ) $ |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Stockholders' Equity | |
Schedule of total outstanding warrants | The total outstanding warrants as of September 30, 2016 is as follows (in thousands): Creditor Warrant shares Warrant fair RedMile $ GCM Total warrants $ |
Schedule of fair value weighted-average assumptions | Three months ended Nine months ended 2016 2015 2016 2015 Expected stock price volatility % % % % Risk free interest rate % % % % Expected life of options (years) Expected annual dividend per share $ $ $ $ |
Summary of stock options | Number of Weighted Weighted Aggregate Intrinsic Balance at December 31, 2015 $ Options granted $ Options exercised ) $ Options forfeited ) $ Balance at September 30, 2016 $ 7.5 years $ Vested and unvested expected to vest September 30, 2016 $ 7.4 years $ Exercisable at September 30, 2016 $ 5.9 years $ |
Summary of non-vested Restricted Stock Units activity | Number of Shares Weighted Weighted Aggregate Intrinsic Non-vested units as of December 31, 2015 $ Granted $ Vested ) $ Forfeited ) $ Non-vested units as of September 30, 2016 $ $ — Non-vested units expected to vest at September 30, 2016 $ $ — |
Summary of the compensation expense recognized in the statements of operations | The following table summarizes information related to compensation expense recognized in the statements of operations related to the equity awards (in thousands): Three Months Nine Months Ended September 30, Ended September 30, 2016 2015 2016 2015 Equity compensation expense recognized in: Research and development expense $ $ $ $ General and administrative expense Total equity compensation expense $ $ $ $ |
Assets and Liabilities Measur27
Assets and Liabilities Measured at Fair Value (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Summary of assets and liabilities subject to fair value measurements | A summary of the fair value of the Company’s assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2016, are identified in the following table (in thousands): Level 1 Level 2 Total Assets: Cash/ money market funds $ $ — $ Corporate debt securities — Commercial paper — Certificate of deposit — Market exchanged mutual funds — $ $ $ Level 2 Level 3 Total Liabilities: Contingent consideration payable — $ $ Derivative liability $ — Deferred compensation plan liability $ — $ $ $ $ A summary of the fair value of the Company’s assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2015, are identified in the following table (in thousands): Level 1 Level 2 Total Assets: Cash/ money market funds $ $ — $ Corporate debt securities — Commercial paper — Certificate of deposit — Market exchanged mutual funds $ $ $ Level 2 Level 3 Total Liabilities: Contingent consideration payable — Deferred compensation plan liability — $ $ $ |
Schedule of changes in contingent consideration payable | The following table shows the change in the balance of contingent consideration payable for the nine months ended September 30, 2016 and 2015, respectively (in thousands): Three months Nine months 2016 2015 2016 2015 Balance, beginning of the period $ $ $ $ Additions, from business acquisitions — — Payment of contingent consideration in cash — — ) — Payment of contingent consideration in stock — — ) — Change in fair value change during the period, included in Statement of Operations ) Balance, end of the period $ $ $ $ |
Scioderm | |
Schedule of significant unobservable inputs used in the valuation of the contingent consideration payable | Contingent Fair value as of Valuation Unobservable Input Range Clinical and regulatory milestones $238.3 million Probability Discount rate 0.5%-3.1% Revenue-based milestones $23.9 million Monte Carlo Revenue volatility 58% |
Callidus | |
Schedule of significant unobservable inputs used in the valuation of the contingent consideration payable | Contingent Fair value as of Valuation Technique Unobservable Input Range Clinical and regulatory milestones $9.6 million Probability weighted discounted cash flow Discount rate 10.5% |
Restructuring Charges (Tables)
Restructuring Charges (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Restructuring Charges | |
Summary of restructuring charges and utilization | The following table summarizes the restructuring charges and utilization for the nine months ended September 30, 2016 (in thousands): Balance as of Charges Cash Payments Fair Value Balance as of Facilities consolidation $ $ — $ ) $ $ — |
Basic and Diluted Net Loss pe29
Basic and Diluted Net Loss per Common Share (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Basic and Diluted Net Loss per Common Share | |
Schedule of reconciliation of the numerator and denominator used in computing basic and diluted net loss per common share | (In thousands, except per share Three months Ended Nine months Ended amounts) 2016 2015 2016 2015 Historical Numerator: Net loss $ ) $ ) $ ) $ ) Denominator: Weighted average common shares outstanding — basic and diluted |
Schedule of potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method | The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method (in thousands): As of September 30, 2016 2015 Options to purchase common stock Outstanding warrants, convertible to common stock — Unvested restricted stock units Total number of potentially issuable shares |
Description of Business (Detail
Description of Business (Details) | Jul. 05, 2016USD ($) | Jun. 30, 2016USD ($)$ / sharesshares | Feb. 19, 2016USD ($)shares | Jun. 30, 2016USD ($)$ / sharesshares | Feb. 29, 2016 | Oct. 31, 2015USD ($)shares | Sep. 30, 2016USD ($)$ / shares | Jul. 31, 2016USD ($)shares | Sep. 30, 2016USD ($)$ / shares | Sep. 30, 2015USD ($) | Apr. 01, 2016item | Dec. 31, 2015USD ($)$ / shares |
Corporate Information, Status of Operations and Management Plans | ||||||||||||
Number of Fabry causing mutations included in the label approved by the CHMP | item | 269 | |||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Common Stock, Par or Stated Value Per Share | $ / shares | $ 0.01 | $ 0.01 | $ 0.01 | |||||||||
Accumulated deficit | $ (720,961,000) | $ (720,961,000) | $ (579,566,000) | |||||||||
Proceeds from the issuance of common stock (in dollars) | 97,068,000 | $ 243,042,000 | ||||||||||
Net product sales | 2,127,000 | $ 2,127,000 | ||||||||||
Galafold | EU | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Net product sales | $ 2,100,000 | |||||||||||
Cowen and Company, LLC | Sales Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Number of shares issued from ATM transactions (in shares) | shares | 15,000,000 | |||||||||||
Net proceeds from stock issued at ATM transactions | $ 97,100,000 | |||||||||||
Commission expenses incurred on issue of common stock | 2,700,000 | |||||||||||
Other expenses paid | $ 200,000 | |||||||||||
October 2015 Purchase Agreement | Redmile Group | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Unsecured notes | $ 50,000,000 | |||||||||||
October 2015 Purchase Agreement | Warrants | Redmile Group | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Warrant term | 5 years | |||||||||||
Shares issuable for warrants (in shares) | shares | 1,300,000 | |||||||||||
February 2016 Purchase Agreement | Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Unsecured notes | $ 50,000,000 | |||||||||||
February 2016 Purchase Agreement | Warrants | Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Warrant term | 5 years | |||||||||||
Increment used for debt conversion | $ 1,000 | |||||||||||
Additional Note and Warrant Agreement June 2016 | Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Common Stock, Par or Stated Value Per Share | $ / shares | $ 0.01 | $ 0.01 | ||||||||||
Unsecured notes | $ 30,000,000 | $ 30,000,000 | ||||||||||
Additional Note and Warrant Agreement June 2016 | Warrants | Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Warrant term | 5 years | 5 years | ||||||||||
Increment used for debt conversion | $ 1,000 | $ 1,000 | ||||||||||
Maximum | ||||||||||||
Corporate Information, Status of Operations and Management Plans | ||||||||||||
Percentage of patients with Fabry disease caused by mutations included in the label approved by the CHMP | 0.5 | |||||||||||
Maximum | Cowen and Company, LLC | Sales Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Fixed commission rate as a percentage of gross proceeds per Share sold | 3.00% | |||||||||||
Maximum | February 2016 Purchase Agreement | Redmile Group | Beneficial Owner | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Unsecured notes | $ 75,000,000 | |||||||||||
Maximum | February 2016 Purchase Agreement | Warrants | Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Shares of common stock issued per increment of note principal | shares | 37 | |||||||||||
Shares issuable for warrants (in shares) | shares | 1,850,000 | |||||||||||
Maximum | Additional Note and Warrant Agreement June 2016 | Warrants | Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Shares of common stock issued per increment of note principal | shares | 42 | 42 | ||||||||||
Shares issuable for warrants (in shares) | shares | 1,260,000 | 1,260,000 | ||||||||||
MiaMed Inc | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Total consideration, stock and cash | $ 6,500,000 | |||||||||||
Cash consideration paid | 1,800,000 | |||||||||||
Potential aggregate deal value | 89,500,000 | |||||||||||
MiaMed Inc | Clinical, Regulatory and Commercial milestones | Maximum | ||||||||||||
Acquisition of Miamed, Inc. | ||||||||||||
Contingent consideration payable upon achievement of milestones | $ 83,000,000 |
Summary of Significant Accoun31
Summary of Significant Accounting Policies (Details) - item | 1 Months Ended | 12 Months Ended |
Jun. 30, 2016 | Dec. 31, 2015 | |
Summary of Significant Accounting Policies | ||
Number of forward contracts | 1 | 0 |
Cash, Money Market Funds and 32
Cash, Money Market Funds and Marketable Securities (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2016 | Dec. 31, 2015 | Sep. 30, 2015 | Dec. 31, 2014 | |
Cash, Money Market Funds, and Marketable Securities | |||||
Cash and cash equivalents, Amortized Cost | $ 33,115 | $ 33,115 | $ 69,485 | $ 19,439 | $ 24,074 |
Cash and cash equivalents, Fair Value | 33,115 | 33,115 | 69,485 | ||
Marketable securities, Amortized Cost | 178,997 | 178,997 | 144,663 | ||
Unrealized Gain | 332 | 332 | 39 | ||
Unrealized Loss | (45) | (45) | (154) | ||
Marketable securities, Fair Value | 179,284 | 179,284 | 144,548 | ||
Cash and marketable securities, Amortized Cost | 212,112 | 212,112 | 214,148 | ||
Cash and marketable securities, Fair Value | 212,399 | 212,399 | 214,033 | ||
Available-for-sale investments | |||||
Realized gain (loss) on securities available-for-sale | 0 | 0 | |||
Fair value of available for sale securities in unrealized loss positions | 60,300 | 60,300 | 118,500 | ||
Other current liabilities | |||||
Cash, Money Market Funds, and Marketable Securities | |||||
Derivative liabilities | 324 | 324 | |||
Other income (expense) | |||||
Cash, Money Market Funds, and Marketable Securities | |||||
Gain (loss) on derivative instruments | 22 | (324) | |||
Corporate debt securities | |||||
Cash, Money Market Funds, and Marketable Securities | |||||
Marketable securities, Amortized Cost | 79,658 | 79,658 | 118,627 | ||
Unrealized Gain | 5 | 5 | 1 | ||
Unrealized Loss | (45) | (45) | (154) | ||
Marketable securities, Fair Value | 79,618 | 79,618 | 118,474 | ||
Commercial paper | |||||
Cash, Money Market Funds, and Marketable Securities | |||||
Marketable securities, Amortized Cost | 98,326 | 98,326 | 25,686 | ||
Unrealized Gain | 327 | 327 | 38 | ||
Marketable securities, Fair Value | 98,653 | 98,653 | 25,724 | ||
Certificate of deposit | |||||
Cash, Money Market Funds, and Marketable Securities | |||||
Marketable securities, Amortized Cost | 1,013 | 1,013 | 350 | ||
Marketable securities, Fair Value | $ 1,013 | $ 1,013 | $ 350 |
Inventories (Details)
Inventories (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Dec. 31, 2015 | |
Inventories | ||
Work-in-process | $ 3,111 | |
Finished goods | 140 | |
Total inventories | 3,251 | $ 0 |
Inventory write-downs | $ 0 |
Acquisitions (Details)
Acquisitions (Details) - USD ($) $ in Thousands | Jul. 05, 2016 | Sep. 30, 2015 | Apr. 30, 2016 | Jun. 30, 2016 | Sep. 30, 2016 | Sep. 30, 2015 | Dec. 31, 2015 | Nov. 30, 2013 |
Acquisitions | ||||||||
Contingent consideration payable | $ 272,190 | $ 274,077 | ||||||
Contingent consideration payable, current portion | 55,992 | 41,400 | ||||||
Changes in fair value of contingent consideration payable | 9,228 | $ 2,400 | ||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Payment of contingent consideration in stock, impact on stockholder's equity | 6,115 | |||||||
IPR&D | ||||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Intangible assets-IPR&D | 486,700 | 486,700 | ||||||
Scioderm | ||||||||
Acquisitions | ||||||||
Initial amount to Effective Time Holders | $ 223,900 | |||||||
Initial amount cash payment excluding Series B Preferred | 141,100 | |||||||
Initial amount paid in shares of Common Stock | $ 82,800 | |||||||
Priority review voucher transfer years | 1 year | |||||||
Payment to be made if Priority Review Voucher is obtained and used | $ 100,000 | |||||||
Contingent consideration payable | 259,000 | 262,200 | $ 259,000 | |||||
Contingent consideration payable, current portion | 56,000 | |||||||
Scioderm | Changes in fair value of contingent consideration payable | ||||||||
Acquisitions | ||||||||
Changes in fair value of contingent consideration payable | 9,400 | |||||||
Scioderm | IPR&D | ||||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Intangible assets-IPR&D | $ 463,700 | |||||||
Scioderm | Maximum | ||||||||
Acquisitions | ||||||||
Priority Review Voucher sale proceeds shared | $ 100,000 | |||||||
Priority Review Voucher sale proceeds shared (as a percent) | 50.00% | |||||||
Scioderm | Scioderm | Series B Preferred Stock | ||||||||
Acquisitions | ||||||||
Consideration paid in common stock | 5,900,000 | |||||||
Callidus | ||||||||
Acquisitions | ||||||||
Contingent consideration payable | $ 10,600 | |||||||
Contingent consideration payable, current portion | 10,000 | |||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Milestone payment, value of shares issued | $ 6,000 | |||||||
Payment of contingent consideration in stock, impact on stockholder's equity | 6,100 | |||||||
Callidus | ATB-200 Pompe program | ||||||||
Acquisitions | ||||||||
Contingent consideration payable, current portion | 9,600 | |||||||
MiaMed Inc | ||||||||
Acquisitions | ||||||||
Total consideration, stock and cash | $ 6,500 | |||||||
Cash consideration paid | 1,800 | |||||||
Potential aggregate deal value | 89,500 | |||||||
Research and development expense | $ 6,500 | |||||||
MiaMed Inc | Amicus | Common Stock | ||||||||
Acquisitions | ||||||||
Consideration paid in common stock | 825,603 | |||||||
Clinical, Regulatory and Commercial milestones | MiaMed Inc | Maximum | ||||||||
Acquisitions | ||||||||
Contingent consideration payable upon achievement of milestones | $ 83,000 | |||||||
Clinical and Regulatory Approval milestones | Scioderm | ||||||||
Acquisitions | ||||||||
Milestone payment | $ 5,000 | |||||||
Contingent consideration payable | 238,300 | |||||||
Clinical and Regulatory Approval milestones | Scioderm | Minimum | ||||||||
Acquisitions | ||||||||
Discount rate (as a percent) | 0.40% | |||||||
Clinical and Regulatory Approval milestones | Scioderm | Maximum | ||||||||
Acquisitions | ||||||||
Discount rate (as a percent) | 1.10% | |||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Upfront cash payments | $ 361,000 | $ 361,000 | ||||||
Clinical and Regulatory Approval milestones | Callidus | ATB-200 Pompe program | ||||||||
Acquisitions | ||||||||
Contingent consideration payable | 9,600 | |||||||
Revenue-based milestones | Scioderm | ||||||||
Acquisitions | ||||||||
Contingent consideration payable | 23,900 | |||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Upfront equity payments | $ 257,000 | |||||||
Revenue-based milestones | Scioderm | Minimum | ||||||||
Acquisitions | ||||||||
Discount rate (as a percent) | 1.00% | |||||||
Revenue-based milestones | Scioderm | Maximum | ||||||||
Acquisitions | ||||||||
Discount rate (as a percent) | 2.20% | |||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Upfront equity payments | $ 257,000 | |||||||
Clinical milestone | Scioderm | Maximum | ||||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Upfront cash payments | 81,000 | |||||||
Regulatory Approval milestone | Scioderm | Maximum | ||||||||
Allocation of the purchase consideration, including the contingent acquisition consideration payable | ||||||||
Upfront cash payments | $ 280,000 |
Goodwill and Intangible Asset35
Goodwill and Intangible Assets (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2016USD ($) | |
Goodwill | $ 197,797 |
Changes in goodwill | |
Balance at beginning of the period | 197,797 |
Balance at end of the period | 197,797 |
Business Acquisitions | |
Goodwill | 197,800 |
Changes in goodwill | |
Balance at beginning of the period | 197,800 |
Change in goodwill | 0 |
Balance at end of the period | $ 197,800 |
Goodwill and Intangible Asset36
Goodwill and Intangible Assets - IPR&D (Details) - IPR&D $ in Millions | 9 Months Ended |
Sep. 30, 2016USD ($) | |
Changes in IPR&D | |
Balance at beginning of the period | $ 486.7 |
Change in IPR&D | 0 |
Balance at end of the period | 486.7 |
Scioderm | |
Changes in IPR&D | |
Balance at beginning of the period | 463.7 |
Business Acquisitions | |
Changes in IPR&D | |
Balance at end of the period | $ 486.7 |
Debt Instruments and Related 37
Debt Instruments and Related Party Transactions (Details) | Jun. 30, 2016USD ($)$ / sharesshares | Feb. 19, 2016USD ($)itemshares | Jun. 30, 2016USD ($)item$ / sharesshares | Oct. 31, 2015USD ($)itemshares | Sep. 30, 2016USD ($)$ / shares | Sep. 30, 2016USD ($)$ / shares | Dec. 31, 2015USD ($)$ / shares |
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Debt discount amortization | $ 700,000 | $ 1,700,000 | |||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | $ 0.01 | ||||
Warrants | $ 16,076,000 | $ 16,076,000 | $ 8,755,000 | ||||
Private Placement Purchase Agreement | Additional Note and Warrant Agreement June 2016 | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Debt discount | 13,976,000 | 13,976,000 | |||||
Outstanding debt | |||||||
Gross amount of debt | 80,000,000 | 80,000,000 | |||||
Net unamortized discount | (13,976,000) | (13,976,000) | |||||
Net carrying value of debt | 66,024,000 | 66,024,000 | |||||
Redmile Group | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Warrants | 12,927,000 | 12,927,000 | |||||
Redmile Group | Private Placement Purchase Agreement | October 2015 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Proceeds from note and warrant purchase agreement | $ 50,000,000 | ||||||
Number of installments for repayment of debt | item | 2 | ||||||
Fixed interest rate (as a percent) | 4.10% | ||||||
Redmile Group | Private Placement Purchase Agreement | Additional Note and Warrant Agreement June 2016 | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Debt discount | 10,953,000 | 10,953,000 | |||||
Outstanding debt | |||||||
Gross amount of debt | 55,000,000 | 55,000,000 | |||||
Net unamortized discount | (10,953,000) | (10,953,000) | |||||
Net carrying value of debt | 44,047,000 | 44,047,000 | |||||
Redmile Group | Private Placement Purchase Agreement | October 2017 payment | October 2015 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Installment payment | $ 15,000,000 | ||||||
Redmile Group | Private Placement Purchase Agreement | October 2020 payment | October 2015 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Installment payment | $ 35,000,000 | ||||||
Redmile Group | Warrants | Private Placement Purchase Agreement | October 2015 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Warrant Term | 5 years | ||||||
Shares issuable for warrants (in shares) | shares | 1,300,000 | ||||||
Debt discount | 8,800,000 | 8,800,000 | |||||
Outstanding debt | |||||||
Net unamortized discount | (8,800,000) | (8,800,000) | |||||
Redmile Group | Beneficial Owner | February 2016 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Ownership position in the company (as a percent) | 10.00% | ||||||
Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | February 2016 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Proceeds from note and warrant purchase agreement | $ 50,000,000 | ||||||
Number of installments for repayment of debt | item | 2 | ||||||
Fixed interest rate (as a percent) | 3.875% | ||||||
Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | Additional Note and Warrant Agreement June 2016 | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Proceeds from note and warrant purchase agreement | $ 30,000,000 | $ 30,000,000 | |||||
Fixed interest rate (as a percent) | 3.875% | 3.875% | |||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | |||||
Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | October 2017 payment | February 2016 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Installment payment | $ 15,000,000 | ||||||
Redmile Group | Beneficial Owner | Private Placement Purchase Agreement | October 2021 payment | February 2016 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Installment payment | $ 35,000,000 | ||||||
Redmile Group | Beneficial Owner | Warrants | Private Placement Purchase Agreement | February 2016 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Warrant Term | 5 years | ||||||
Increment used for debt conversion | $ 1,000 | ||||||
Incremental fair value of warrant liability | 3,500,000 | ||||||
Redmile Group | Beneficial Owner | Warrants | Private Placement Purchase Agreement | Additional Note and Warrant Agreement June 2016 | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Warrant Term | 5 years | 5 years | |||||
Increment used for debt conversion | $ 1,000 | $ 1,000 | |||||
Number of inputs used in fair value classification | item | 6 | ||||||
Offering price (in dollars per share) | $ / shares | $ 5.46 | $ 5.46 | |||||
Exercise price of warrants (in dollars per share) | $ / shares | $ 7.06 | $ 7.06 | |||||
Remaining term of the warrants | 5 years | ||||||
Volatility (as a percent) | 86.02% | ||||||
Annual rate of dividends (as a percent) | 0.00% | ||||||
Risk-free rate of return (as a percent) | 1.01% | ||||||
Fair value of the warrant liability | $ 3,800,000 | $ 3,800,000 | |||||
GCM | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Warrants | 3,149,000 | 3,149,000 | |||||
GCM | Private Placement Purchase Agreement | Additional Note and Warrant Agreement June 2016 | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Debt discount | 3,023,000 | 3,023,000 | |||||
Outstanding debt | |||||||
Gross amount of debt | 25,000,000 | 25,000,000 | |||||
Net unamortized discount | (3,023,000) | (3,023,000) | |||||
Net carrying value of debt | $ 21,977,000 | $ 21,977,000 | |||||
Maximum | Redmile Group | Beneficial Owner | February 2016 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Proceeds from note and warrant purchase agreement | $ 75,000,000 | ||||||
Maximum | Redmile Group | Beneficial Owner | Warrants | Private Placement Purchase Agreement | February 2016 Purchase Agreement | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Shares issuable for warrants (in shares) | shares | 1,850,000 | ||||||
Shares of common stock issued per increment of note principal | shares | 37 | ||||||
Maximum | Redmile Group | Beneficial Owner | Warrants | Private Placement Purchase Agreement | Additional Note and Warrant Agreement June 2016 | |||||||
Debt Instruments and Related Party Transactions [Line Items] | |||||||
Shares issuable for warrants (in shares) | shares | 1,260,000 | 1,260,000 | |||||
Shares of common stock issued per increment of note principal | shares | 42 | 42 |
Stockholders' Equity - Common S
Stockholders' Equity - Common Stock and Warrants (Details) $ / shares in Units, $ in Thousands | Jun. 30, 2016$ / sharesshares | Feb. 19, 2016shares | Jun. 30, 2016$ / sharesshares | Feb. 29, 2016 | Oct. 31, 2015shares | Jul. 31, 2016USD ($)shares | Sep. 30, 2016USD ($)item$ / sharesshares | Sep. 30, 2015USD ($) | Dec. 31, 2015USD ($)$ / sharesshares |
Collaborative Agreements | |||||||||
Authorized number of shares of common stock | 250,000,000 | 250,000,000 | |||||||
Voting right for each share held, number | item | 1 | ||||||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | |||||||
Net proceeds from the issuance of common stock | $ | $ 97,068 | $ 243,042 | |||||||
Warrants outstanding (in shares) | 3,110,000 | ||||||||
Warrant liability | $ | $ 16,076 | $ 8,755 | |||||||
Cowen and Company, LLC | Sales Agreement | |||||||||
Collaborative Agreements | |||||||||
Number of shares issued from ATM transactions (in shares) | 15,000,000 | ||||||||
Net proceeds from stock issued at ATM transactions | $ | $ 97,100 | ||||||||
Commission expenses incurred on issue of common stock | $ | 2,700 | ||||||||
Other Expenses | $ | $ 200 | ||||||||
Cowen and Company, LLC | Sales Agreement | Maximum | |||||||||
Collaborative Agreements | |||||||||
Fixed commission rate as a percentage of gross proceeds per Share sold | 3.00% | ||||||||
Redmile Group | |||||||||
Collaborative Agreements | |||||||||
Warrants outstanding (in shares) | 2,060,000 | ||||||||
Warrant liability | $ | $ 12,927 | ||||||||
GCM | |||||||||
Collaborative Agreements | |||||||||
Warrants outstanding (in shares) | 1,050,000 | ||||||||
Warrant liability | $ | $ 3,149 | ||||||||
October 2015 Purchase Agreement | Redmile Group | Private Placement Purchase Agreement | Warrants | |||||||||
Collaborative Agreements | |||||||||
Warrant term | 5 years | ||||||||
Shares issuable for warrants (in shares) | 1,300,000 | ||||||||
February 2016 Purchase Agreement | Beneficial Owner | Redmile Group | Private Placement Purchase Agreement | Warrants | |||||||||
Collaborative Agreements | |||||||||
Warrant term | 5 years | ||||||||
February 2016 Purchase Agreement | Beneficial Owner | Redmile Group | Private Placement Purchase Agreement | Maximum | Warrants | |||||||||
Collaborative Agreements | |||||||||
Shares of common stock issued per increment of note principal | 37 | ||||||||
Shares issuable for warrants (in shares) | 1,850,000 | ||||||||
Additional Note and Warrant Agreement June 2016 | Beneficial Owner | Redmile Group | Private Placement Purchase Agreement | |||||||||
Collaborative Agreements | |||||||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | |||||||
Additional Note and Warrant Agreement June 2016 | Beneficial Owner | Redmile Group | Private Placement Purchase Agreement | Warrants | |||||||||
Collaborative Agreements | |||||||||
Warrant term | 5 years | 5 years | |||||||
Additional Note and Warrant Agreement June 2016 | Beneficial Owner | Redmile Group | Private Placement Purchase Agreement | Maximum | Warrants | |||||||||
Collaborative Agreements | |||||||||
Shares of common stock issued per increment of note principal | 42 | 42 | |||||||
Shares issuable for warrants (in shares) | 1,260,000 | 1,260,000 |
Stockholders' Equity - Equity I
Stockholders' Equity - Equity Incentive Plan (Details) - USD ($) | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | Dec. 31, 2015 | |
Nonqualified Cash Plan (the "Deferral Plan") | |||||
Stockholders' Equity | |||||
Deferred compensation liability, noncurrent | $ 1,300,000 | $ 1,300,000 | $ 700,000 | ||
Trading securities | 1,300,000 | 1,300,000 | $ 700,000 | ||
Unrealized gain/(loss) | $ 30,000 | $ (54,000) | $ 37,000 | $ (64,000) | |
Common stock options | |||||
Fair value weighted-average assumptions: | |||||
Expected stock price volatility (as a percent) | 81.60% | 74.40% | 81.30% | 75.40% | |
Risk free interest rate (as a percent) | 1.20% | 1.70% | 1.50% | 1.70% | |
Expected life of options | 6 years 3 months | 6 years 3 months | 6 years 3 months | 6 years 3 months | |
Expected annual dividend per share (in dollars per share) | $ 0 | $ 0 | $ 0 | $ 0 | |
Number of Shares | |||||
Balance at the beginning of the period (in shares) | 11,729,200 | ||||
Options granted (in shares) | 4,829,600 | ||||
Options exercised (in shares) | (371,700) | ||||
Options forfeited (in shares) | (413,900) | ||||
Balance at the end of the period (in shares) | 15,773,200 | 15,773,200 | |||
Vested and unvested expected to vest as of the end of the period (in shares) | 14,718,800 | 14,718,800 | |||
Exercisable at the end of the period (in shares) | 7,395,400 | 7,395,400 | |||
Weighted Average Exercise Price | |||||
Balance at the beginning of the period (in dollars per share) | $ 7.11 | ||||
Options granted (in dollars per share) | 7.70 | ||||
Options exercised (in dollars per share) | 4.05 | ||||
Options forfeited (in dollars per share) | 8.79 | ||||
Balance at the end of the period (in dollars per share) | $ 7.32 | 7.32 | |||
Vested and unvested expected to vest as of the end of the period (in dollars per share) | 7.24 | 7.24 | |||
Exercisable at the end of the period (in dollars per share) | $ 6.44 | $ 6.44 | |||
Weighted Average Remaining Contractual Life | |||||
Balance at the end of the period | 7 years 6 months | ||||
Vested and unvested expected to vest at the end of the period | 7 years 4 months 24 days | ||||
Exercisable at the end of the period | 5 years 10 months 24 days | ||||
Aggregate Intrinsic Value | |||||
Aggregate intrinsic value of options outstanding (in dollars) | $ 24,900 | $ 24,900 | |||
Aggregate intrinsic value of options vested and unvested expected to vest (in dollars) | 24,100 | 24,100 | |||
Aggregate intrinsic value of options exercisable (in dollars) | 16,300 | 16,300 | |||
Total unrecognized compensation cost related to non-vested stock options granted (in dollars) | $ 35,800,000 | $ 35,800,000 | |||
Period of recognition unrecognized compensation costs (in years) | 2 years 10 months 24 days | ||||
Restricted stock units (RSUs) | Amended and Restated 2007 Equity Incentive Plan | |||||
Aggregate Intrinsic Value | |||||
Period of recognition unrecognized compensation costs (in years) | 2 years 8 months 9 days | ||||
Restricted Stock Units, Number of Shares | |||||
Non-vested units as of the beginning of the period (in shares) | 478,500 | ||||
Granted (in shares) | 582,700 | ||||
Vested (in shares) | (199,300) | ||||
Forfeited (in shares) | (23,200) | ||||
Non-vested units as of the end of the period (in shares) | 838,700 | 838,700 | |||
Non-vested units expected to vest (in shares) | 838,700 | 838,700 | |||
Weighted Average Grant Date Fair Value | |||||
Non-vested units as of the beginning of the period (in dollars per share) | $ 10.38 | ||||
Granted (in dollars per share) | 6.21 | ||||
Vested (in dollars per share) | 7.25 | ||||
Forfeited (in dollars per share) | 8.52 | ||||
Non-vested units as of the end of the period (in dollars per share) | $ 8.28 | 8.28 | |||
Non-vested units expected to vest (in dollars per share) | $ 8.28 | $ 8.28 | |||
Non-vested units as of the end of the period | 2 years 8 months 9 days | ||||
Non-vested units expected to vest as of the end of the period | 2 years 8 months 9 days | ||||
Unrecognized compensation cost related to unvested RSU's (in dollars) | $ 4,800,000 | $ 4,800,000 |
Stockholders' Equity - Compensa
Stockholders' Equity - Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Equity compensation expense | ||||
Total equity compensation expense | $ 4,338 | $ 2,737 | $ 13,087 | $ 6,929 |
Research and development expense | ||||
Equity compensation expense | ||||
Total equity compensation expense | 2,109 | 1,232 | 6,011 | 3,224 |
General and administrative expense | ||||
Equity compensation expense | ||||
Total equity compensation expense | $ 2,229 | $ 1,505 | $ 7,076 | $ 3,705 |
Assets and Liabilities Measur41
Assets and Liabilities Measured at Fair Value (Details) $ / shares in Units, $ in Thousands | Jul. 05, 2016USD ($) | Sep. 30, 2015USD ($) | Jun. 30, 2016USD ($)item$ / shares | Sep. 30, 2016USD ($) | Jun. 30, 2016USD ($)$ / shares | Sep. 30, 2015USD ($) | Sep. 30, 2016USD ($) | Sep. 30, 2015USD ($) | Dec. 31, 2015USD ($)item | Feb. 19, 2016USD ($) | Oct. 31, 2015USD ($) | Dec. 31, 2014USD ($) | Nov. 30, 2013USD ($) |
Financial assets and liabilities subject to fair value measurements | |||||||||||||
Transfer of assets from Level 1 to Level 2 | $ 0 | $ 0 | |||||||||||
Transfer of assets from Level 2 to Level 1 | 0 | 0 | |||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Warrant liability | 16,076 | 16,076 | $ 8,755 | ||||||||||
Contingent consideration payable | 272,190 | 272,190 | 274,077 | ||||||||||
Contingent consideration payable | |||||||||||||
Balance, beginning of the period | 276,300 | $ 11,800 | 274,077 | $ 10,700 | 10,700 | ||||||||
Additions, from business acquisitions | 269,884 | 269,884 | |||||||||||
Payment of contingent consideration in cash | (5,000) | ||||||||||||
Payment of contingent consideration in stock | (6,115) | ||||||||||||
Change in fair value change during the period, included in Statement of Operations | (4,110) | 1,300 | 9,228 | 2,400 | |||||||||
Balance, end of the period | $ 282,984 | $ 276,300 | 272,190 | $ 276,300 | 282,984 | 272,190 | 282,984 | 274,077 | |||||
Assets: | |||||||||||||
Cash/money market funds | 19,439 | 33,115 | 19,439 | 33,115 | 19,439 | 69,485 | $ 24,074 | ||||||
Fair value of assets | 213,724 | 213,724 | 214,691 | ||||||||||
Liabilities: | |||||||||||||
Contingent consideration payable | 272,190 | 272,190 | 274,077 | ||||||||||
Derivative liability | 324 | 324 | |||||||||||
Deferred compensation plan liability | 1,350 | 1,350 | 667 | ||||||||||
Fair value of liabilities | 273,864 | 273,864 | $ 274,744 | ||||||||||
Note payable to related party | 21,977 | 21,977 | |||||||||||
Foreign Currency Exchange Rate Exposure | |||||||||||||
Number of forward contracts | item | 1 | 0 | |||||||||||
Non-cash changes in the fair value of derivative liability | (22) | 324 | |||||||||||
Other current liabilities | |||||||||||||
Foreign Currency Exchange Rate Exposure | |||||||||||||
Derivative liabilities | 324 | 324 | |||||||||||
Callidus | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Contingent consideration payable | $ 10,600 | ||||||||||||
Contingent consideration payable | |||||||||||||
Payment of contingent consideration in stock | (6,100) | ||||||||||||
Liabilities: | |||||||||||||
Contingent consideration payable | $ 10,600 | ||||||||||||
Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Contingent consideration payable | 259,000 | 262,200 | 259,000 | 262,200 | 259,000 | ||||||||
Liabilities: | |||||||||||||
Contingent consideration payable | $ 259,000 | 262,200 | $ 259,000 | 262,200 | $ 259,000 | ||||||||
MiaMed Inc | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Potential aggregate deal value | $ 89,500 | ||||||||||||
Clinical and Regulatory Approval milestones | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Contingent consideration payable | 238,300 | 238,300 | |||||||||||
Liabilities: | |||||||||||||
Contingent consideration payable | 238,300 | 238,300 | |||||||||||
Revenue-based milestones | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Contingent consideration payable | 23,900 | 23,900 | |||||||||||
Liabilities: | |||||||||||||
Contingent consideration payable | 23,900 | 23,900 | |||||||||||
Minimum | Clinical and Regulatory Approval milestones | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 0.40% | ||||||||||||
Minimum | Revenue-based milestones | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 1.00% | ||||||||||||
Maximum | Clinical and Regulatory Approval milestones | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 1.10% | ||||||||||||
Maximum | Revenue-based milestones | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 2.20% | ||||||||||||
Maximum | Clinical, Regulatory and Commercial milestones | MiaMed Inc | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Contingent consideration payable upon achievement of milestones | $ 83,000 | ||||||||||||
ATB-200 Pompe program | Clinical and Regulatory Approval milestones | Callidus | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Contingent consideration payable | 9,600 | 9,600 | |||||||||||
Liabilities: | |||||||||||||
Contingent consideration payable | 9,600 | 9,600 | |||||||||||
Cash/money market funds | |||||||||||||
Assets: | |||||||||||||
Cash/money market funds | 33,115 | 33,115 | $ 69,485 | ||||||||||
Corporate debt securities | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 79,618 | 79,618 | 118,474 | ||||||||||
Commercial paper | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 98,653 | 98,653 | 25,724 | ||||||||||
Certificate of deposit | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 1,013 | 1,013 | 350 | ||||||||||
Market exchanged mutual funds | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 1,325 | 1,325 | 658 | ||||||||||
Level 1 | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 33,115 | 33,115 | 69,485 | ||||||||||
Level 1 | Cash/money market funds | |||||||||||||
Assets: | |||||||||||||
Cash/money market funds | 33,115 | 33,115 | 69,485 | ||||||||||
Level 2 | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 180,609 | 180,609 | 145,206 | ||||||||||
Liabilities: | |||||||||||||
Derivative liability | 324 | 324 | |||||||||||
Deferred compensation plan liability | 1,350 | 1,350 | 667 | ||||||||||
Fair value of liabilities | 1,674 | 1,674 | 667 | ||||||||||
Level 2 | Corporate debt securities | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 79,618 | 79,618 | 118,474 | ||||||||||
Level 2 | Commercial paper | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 98,653 | 98,653 | 25,724 | ||||||||||
Level 2 | Certificate of deposit | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 1,013 | 1,013 | 350 | ||||||||||
Level 2 | Market exchanged mutual funds | |||||||||||||
Assets: | |||||||||||||
Fair value of assets | 1,325 | 1,325 | 658 | ||||||||||
Level 3 | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Contingent consideration payable | 272,190 | 272,190 | 274,077 | ||||||||||
Liabilities: | |||||||||||||
Contingent consideration payable | 272,190 | 272,190 | 274,077 | ||||||||||
Fair value of liabilities | 272,190 | $ 272,190 | $ 274,077 | ||||||||||
Level 3 | Revenue-based milestones | Monte Carlo | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Probability of achievement of milestones (as a percent) | 66.50% | ||||||||||||
Revenue volatility (as a percent) | 58.00% | ||||||||||||
Level 3 | Minimum | Clinical and Regulatory Approval milestones | Probability weighted discounted cash flow | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 0.50% | ||||||||||||
Probability of achievement of milestones (as a percent) | 66.50% | ||||||||||||
Level 3 | Minimum | Revenue-based milestones | Monte Carlo | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 0.90% | ||||||||||||
Level 3 | Maximum | Clinical and Regulatory Approval milestones | Probability weighted discounted cash flow | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 3.10% | ||||||||||||
Probability of achievement of milestones (as a percent) | 100.00% | ||||||||||||
Level 3 | Maximum | Revenue-based milestones | Monte Carlo | Scioderm | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 1.70% | ||||||||||||
Level 3 | ATB-200 Pompe program | Clinical and Regulatory Approval milestones | Probability weighted discounted cash flow | Callidus | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Discount rate (as a percent) | 10.50% | ||||||||||||
Level 3 | ATB-200 Pompe program | Minimum | Clinical and Regulatory Approval milestones | Probability weighted discounted cash flow | Callidus | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Probability of achievement of milestones (as a percent) | 30.00% | ||||||||||||
Level 3 | ATB-200 Pompe program | Maximum | Clinical and Regulatory Approval milestones | Probability weighted discounted cash flow | Callidus | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Probability of achievement of milestones (as a percent) | 43.00% | ||||||||||||
Additional Note and Warrant Agreement June 2016 | Private Placement Purchase Agreement | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Notes payable | 66,024 | $ 66,024 | |||||||||||
Debt discount | 13,976 | 13,976 | |||||||||||
Redmile Group | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Warrant liability | 12,927 | 12,927 | |||||||||||
Redmile Group | October 2015 Purchase Agreement | Private Placement Purchase Agreement | |||||||||||||
Financial assets and liabilities subject to fair value measurements | |||||||||||||
Proceeds from note and warrant purchase agreement | $ 50,000 | ||||||||||||
Redmile Group | October 2015 Purchase Agreement | Warrants | Private Placement Purchase Agreement | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Debt discount | 8,800 | 8,800 | |||||||||||
Redmile Group | February 2016 Purchase Agreement | Beneficial Owner | Maximum | |||||||||||||
Financial assets and liabilities subject to fair value measurements | |||||||||||||
Proceeds from note and warrant purchase agreement | $ 75,000 | ||||||||||||
Redmile Group | February 2016 Purchase Agreement | Beneficial Owner | Private Placement Purchase Agreement | |||||||||||||
Financial assets and liabilities subject to fair value measurements | |||||||||||||
Proceeds from note and warrant purchase agreement | $ 50,000 | ||||||||||||
Redmile Group | Additional Note and Warrant Agreement June 2016 | Private Placement Purchase Agreement | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Notes payable | 44,047 | 44,047 | |||||||||||
Debt discount | $ 10,953 | $ 10,953 | |||||||||||
Redmile Group | Additional Note and Warrant Agreement June 2016 | Beneficial Owner | Private Placement Purchase Agreement | |||||||||||||
Financial assets and liabilities subject to fair value measurements | |||||||||||||
Proceeds from note and warrant purchase agreement | $ 30,000 | $ 30,000 | |||||||||||
Redmile Group | Additional Note and Warrant Agreement June 2016 | Beneficial Owner | Warrants | Private Placement Purchase Agreement | |||||||||||||
Weighted average assumptions used in the Black-Scholes valuation model for the warrants | |||||||||||||
Number of inputs used in fair value classification | item | 6 | ||||||||||||
Share Price | $ / shares | $ 5.46 | $ 5.46 | |||||||||||
Exercise price of warrants (in dollars per share) | $ / shares | $ 7.06 | $ 7.06 | |||||||||||
Remaining term of the warrants | 5 years | ||||||||||||
Expected stock price volatility (as a percent) | 86.02% | ||||||||||||
Expected annual dividend per share (as a percent) | 0.00% | ||||||||||||
Risk free interest rate (as a percent) | 1.01% | ||||||||||||
Fair value of the warrant liability | $ 3,800 | $ 3,800 | |||||||||||
Redmile Group and GCM | Additional Note and Warrant Agreement June 2016 | |||||||||||||
Financial assets and liabilities subject to fair value measurements | |||||||||||||
Proceeds from note and warrant purchase agreement | $ 66,000 |
Restructuring Charges (Details)
Restructuring Charges (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | ||
Dec. 31, 2013USD ($)item | Sep. 30, 2016USD ($) | Sep. 30, 2015USD ($) | Sep. 30, 2016USD ($) | Sep. 30, 2015USD ($) | |
Summary of restructuring charges and utilization | |||||
Charges | $ (11) | $ (7) | $ (69) | $ (44) | |
Facilities consolidation | |||||
Restructuring charges | |||||
Number of subleased locations that were closed in consolidation | item | 1 | ||||
Lease payments | $ 700 | ||||
Summary of restructuring charges and utilization | |||||
Balance at the beginning of the period | 118 | ||||
Cash payments | (187) | ||||
Fair Value Adjustments | $ 69 |
Basic and Diluted Net Loss pe43
Basic and Diluted Net Loss per Common Share (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Numerator: | ||||
Net loss | $ (46,654) | $ (37,800) | $ (141,395) | $ (89,222) |
Denominator: | ||||
Weighted average common shares outstanding - basic and diluted | 140,656,109 | 118,724,882 | 131,675,690 | 104,885,956 |
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 19,722,000 | 12,322,000 | ||
Common stock options | ||||
Denominator: | ||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 15,773,000 | 11,574,000 | ||
Warrants | ||||
Denominator: | ||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 3,110,000 | |||
Restricted stock units (RSUs) | ||||
Denominator: | ||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 839,000 | 748,000 |
Commitments and Contingencies (
Commitments and Contingencies (Details) | Sep. 30, 2016lawsuit |
Commitments and Contingencies. | |
Number of purported securities class action lawsuits | 3 |
Number of purported securities class action lawsuits naming additional company officers as defendants | 1 |