Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2016 | May. 02, 2016 | |
Document And Entity Information [Abstract] | ||
Entity Registrant Name | Raptor Pharmaceutical Corp | |
Entity Central Index Key | 1,070,698 | |
Trading Symbol | RPTP | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Document Fiscal Year Focus | 2,016 | |
Document Fiscal Period Focus | Q1 | |
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2016 | |
Entity Common Stock, Shares Outstanding | 85,306,787 |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2016 | Dec. 31, 2015 |
Current assets: | ||
Cash and cash equivalents | $ 132,038 | $ 157,352 |
Restricted cash | 1,371 | 1,055 |
Accounts receivable | 17,862 | 13,267 |
Inventories | 9,596 | 6,424 |
Prepaid expenses and other assets | 4,558 | 3,301 |
Total current assets | 165,425 | 181,399 |
Noncurrent assets: | ||
Property and equipment, net | 7,594 | 7,644 |
Goodwill | 12,223 | 12,223 |
Intangible assets, net | 176,731 | 216,463 |
Other assets | 1,994 | 1,761 |
Total Assets | 363,967 | 419,490 |
Current liabilities: | ||
Accounts payable | 4,857 | 5,423 |
Accrued liabilities | 23,639 | 22,630 |
Note payable, current portion | 10,959 | 10,846 |
Total current liabilities | 39,455 | 38,899 |
Noncurrent liabilities: | ||
Contingent consideration liability | 152,070 | 166,800 |
Deferred tax liability | 615 | 303 |
Note payable, net of current portion | 33,479 | 36,296 |
Convertible notes | 60,000 | 60,000 |
Total liabilities | 285,619 | 302,298 |
Stockholders' equity: | ||
Preferred stock, $0.001 par value per share, 15,000,000 shares authorized, zero shares issued and outstanding | 0 | |
Common stock, $0.001 par value per share, 150,000,000 shares authorized, 85,289,311 and 85,235,591 shares issued and outstanding at March 31, 2016 and December 31, 2015, respectively | 85 | 85 |
Additional paid-in capital | 444,299 | 441,601 |
Accumulated other comprehensive loss | (1,334) | (1,377) |
Accumulated deficit | (364,702) | (323,117) |
Total stockholders' equity | 78,348 | 117,192 |
Total Liabilities and Stockholders' Equity | $ 363,967 | $ 419,490 |
CONDENSED CONSOLIDATED BALANCE3
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) (Parenthetical) - $ / shares | Mar. 31, 2016 | Dec. 31, 2015 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 15,000,000 | 15,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 150,000,000 | 150,000,000 |
Common stock issued (in shares) | 85,289,311 | 85,235,591 |
Common stock, shares outstanding (in shares) | 85,289,311 | 85,235,591 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Income Statement [Abstract] | ||
Product revenue | $ 27,470 | $ 20,453 |
Cost of sales | 4,331 | 3,722 |
Gross profit | 23,139 | 16,731 |
Operating expenses: | ||
Research and development | 14,019 | 16,552 |
Selling, general and administrative | 20,388 | 14,839 |
Impairment of IPR&D | 39,600 | |
Change in fair value of contingent consideration related to Quinsair acquisition | (14,730) | |
Total operating expenses | 59,277 | 31,391 |
Loss from operations | (36,138) | (14,660) |
Interest income | 132 | 27 |
Interest expense | (5,018) | (4,498) |
Foreign currency transaction loss | (213) | (476) |
Adjustment to fair value of common stock warrants | (55) | |
Loss before provision for income taxes | (41,237) | (19,662) |
Provision for income taxes | 348 | 21 |
Net Loss | (41,585) | (19,683) |
Other comprehensive income (loss): | ||
Foreign currency translation gain (loss), net of tax | 43 | (547) |
Comprehensive Loss | $ (41,542) | $ (20,230) |
Net loss per share: | ||
Basic and diluted | $ (0.49) | $ (0.28) |
Weighted-average shares outstanding: | ||
Basic and diluted | 85,255,212 | 69,140,642 |
CONDENSED CONSOLIDATED STATEME5
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Cash flows from operating activities: | ||
Net loss | $ (41,585) | $ (19,683) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 2,658 | 2,853 |
Fair value adjustment of common stock warrants | 55 | |
Amortization of intangible asset | 132 | 60 |
Depreciation of property and equipment | 373 | 302 |
Deferred income taxes | 312 | |
Amortization of debt issuance cost | 295 | 302 |
Impairment of IPR&D | 39,600 | |
Change in fair value of contingent consideration related to Quinsair acquisition | (14,730) | |
Changes in assets and liabilities: | ||
Accounts receivable | (4,559) | (3,395) |
Inventories | (3,178) | 4,817 |
Prepaid expenses and other assets | (1,336) | 869 |
Accounts payable | (698) | (1,305) |
Accrued liabilities | 791 | 386 |
Net cash used in operating activities | (21,925) | (14,739) |
Cash flows from investing activities: | ||
Net purchase of property and equipment | (307) | (2,045) |
Change in restricted cash | (316) | 70 |
Net cash used in investing activities | (623) | (1,975) |
Cash used in financing activities: | ||
Proceeds from the exercise of common stock options and ESPP | 41 | 2,185 |
Principal payments on debt | (3,000) | |
Net cash (used in) provided by financing activities | (2,959) | 2,185 |
Effect of exchange rates on cash and cash equivalents | 193 | (547) |
Net decrease in cash and cash equivalents | (25,314) | (15,076) |
Cash and cash equivalents, beginning of period | 157,352 | 149,613 |
Cash and Cash Equivalents, End of Period | 132,038 | 134,537 |
Supplemental cash flow information: | ||
Interest paid | 2,793 | 2,869 |
Income taxes paid | $ 85 | $ 120 |
DESCRIPTION OF BUSINESS AND SUM
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 3 Months Ended |
Mar. 31, 2016 | |
Accounting Policies [Abstract] | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 1. DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES The accompanying condensed consolidated financial statements reflect the financial position and results of operations of Raptor Pharmaceutical Corp. (the "Company" or "Raptor") and have been prepared in accordance with the accounting principles generally accepted in the United States of America ("GAAP") pursuant to the rules and regulations of the Securities and Exchange Commission (the "SEC"). Certain information and footnote disclosures have been condensed or omitted pursuant to such rules and regulations. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and in the opinion of management reflect all adjustments, which include only normal recurring adjustments, necessary for a fair presentation of the periods presented. The condensed consolidated balance sheet as of December 31, 2015 has been derived from the Company’s audited financial statements as of such date but does not include all disclosures required by GAAP. This Form 10-Q should be read in conjunction with the audited financial statements and accompanying notes in the Company's Annual Report on Form 10-K for the year ended December 31, 2015. Raptor is a biopharmaceutical company focused on developing and commercializing transformative treatments for people affected by rare and debilitating diseases. The Company's first commercial product, PROCYSBI® (cysteamine bitartrate) delayed-release capsules (“PROCYSBI”), received marketing approval from the U.S. Food and Drug Administration (“FDA”) on April 30, 2013 for the management of nephropathic cystinosis in adults and children six years and older. On August 14, 2015, we received FDA approval for the expanded use of PROCYSBI to treat children two to six years of age with nephropathic cystinosis. In Europe, PROCYSBI® gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate), received marketing authorization on September 6, 2013 from the European Commission (“EC”), for marketing in the European Union (“EU”) as an orphan medicinal product for the management of proven nephropathic cystinosis. The EU marketing authorization allows the Company to commercialize PROCYSBI in the 28 Member States of the EU plus Norway, Liechtenstein and Iceland (which are not EU Member States but are part of the European Economic Area or EEA). PROCYSBI received seven years of market exclusivity, through 2020 for patients six years and older as an orphan drug in the United States and ten years of market exclusivity, through 2023, as an orphan drug in Europe. Recently, PROCYSBI received orphan drug designation for the treatment of patients ages two years to six years, through 2022. The Company commenced commercial sales of PROCYSBI in the United States in June 2013 and in Europe in April 2014. For at least the near term, the Company's ability to generate revenue is primarily dependent upon sales of PROCYSBI in the United States for the management of nephropathic cystinosis in adults and children two years and older and in the EU for the management of proven nephropathic cystinosis. In October 2015, the Company acquired various assets and rights related to levofloxacin solution for inhalation, a pharmaceutical product also known as “MP-376” and commercially as “QUINSAIR,” from Tripex Pharmaceuticals, LLC (“Tripex”). QUINSAIR received marketing authorization by the EC for treating chronic lung infection caused by the bacteria Pseudomonas aeruginosa Pseudomonas aeruginosa Pseudomonas aeruginosa The Company is subject to a number of risks, including: the level of commercial sales of PROCYSBI in the United States and Europe; the ability to successfully launch PROCYSBI in other international markets; the ability to successfully launch and commercialize QUINSAIR in Europe and Canada; uncertainty whether the Company's research and development efforts will result in expanded labeling for PROCYSBI and additional commercialization for RP103 or MP-376 in various indications or additional commercial products; competition from other organizations; reliance on other entities for manufacturing; reliance on licensing the proprietary technology of others; uncertain patent protection; and the need to raise capital through equity and/or debt financings. Funding may not be available when needed, if at all, or on terms acceptable to the Company. If the Company exhausts its cash reserves and is unable to obtain adequate financing, it may be required to curtail planned operating expenditures, including its development programs. Basis of Presentation The Company's consolidated financial statements include the accounts of the Company's direct and indirect wholly owned subsidiaries, Raptor Pharmaceuticals Inc., formerly known as Raptor Therapeutics Inc. which merged with Raptor Discoveries Inc. in December 2012 prior to changing its name, and Raptor European Products, LLC, such subsidiaries incorporated in Delaware on August 1, 2007, and February 14, 2012, respectively, and Raptor Pharmaceuticals Europe B.V. (“BV”), Raptor Pharmaceuticals France SAS (“SAS”), Raptor Pharmaceuticals Germany GmbH (“GMBH”) and RPTP European Holdings C.V. (“CV”), domiciled in the Netherlands on December 15, 2009, in France on October 30, 2012, in Germany on October 16, 2013 and in the Cayman Islands on February 16, 2012, respectively. All inter-company accounts have been eliminated. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make certain estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the dates of the consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. Functional Currency The Company's consolidated functional currency is the U.S. dollar. BV, SAS, and GMBH, the Company's Dutch subsidiary, French subsidiary, and German Subsidiary, respectively, use the European Euro as their functional currency. The CV subsidiary, a Cayman-based subsidiary, uses the dollar as its functional currency. At each quarter end, each foreign subsidiary's balance sheets are translated into U.S. dollars based upon the quarter-end exchange rate, while their statements of operations and comprehensive loss are translated into U.S. dollars based upon an average exchange rate during the period. Segment Information The Company has determined that it operates in only one segment, as it only reports profit and loss information on an aggregate basis to its chief operating decision maker. The Company's long-lived assets maintained outside the U.S. are not material. Fair Value of Financial Instruments and Contingent Consideration Liability The carrying amounts of certain of the Company's financial instruments including cash equivalents, restricted cash, accounts payable, accrued liabilities, and capital lease liability approximate fair value due either to length of maturity or interest rates that approximate prevailing market rates. Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less, when purchased, to be cash equivalents. The Company maintains cash and cash equivalents, which consist principally of money market funds, with high credit quality financial institutions. Such amounts exceed Federal Deposit Insurance Corporation insurance limits. As of March 31, 2016, the Company had $132 million in cash and cash equivalents, of which $5.6 million was held by its foreign subsidiaries. Restricted Cash Restricted cash represents certificates of deposit and compensating balances required by the Company’s U.S. and European banks as collateral for credit cards and for access to a value-added tax deferral program. Accounts Receivable and Allowance for Doubtful Accounts Accounts receivable are recorded and carried at the original invoiced amount less an allowance for any potential uncollectible amounts. The Company estimates its allowance for doubtful accounts based upon an assessment of various factors, including historical experience, the age of the accounts receivable balances, credit quality of customers, current economic conditions, and other factors that may affect customers’ ability to pay. To date, the Company has not experienced significant losses with respect to the collection of accounts receivable. Revenue Recognition The Company recognizes revenue in accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Codification (“ASC”) 605, Revenue Recognition, when the following criteria have been met: persuasive evidence of an arrangement exists; delivery has occurred and risk of loss has passed; the seller's price to the buyer is fixed or determinable and collectability is reasonably assured. The Company determines that persuasive evidence of an arrangement exists based on written contracts that define the terms of the arrangements. Pursuant to the contract terms, the Company determines when title to products and associated risk of loss has passed on to the customer. The Company assesses whether the fee is fixed or determinable based on the payment terms associated with the transaction and whether the sales price is subject to refund or adjustment. The Company assesses collectability based primarily on the customer's payment history and creditworthiness. PROCYSBI is currently available for U.S. distribution from the Company's U.S. specialty pharmacy partner, the Accredo Health Group, Inc. ("Accredo") which is currently the Company's only U.S. customer and ships directly to patients. The Company's distributor in the EU is the Almac Group, Ltd. PROCYSBI is not available in U.S. retail pharmacies. Authorization of coverage by patients' commercial insurance plans, Raptor's patient assistance program ("PAP") or government payors is a prerequisite to the shipment of PROCYSBI to U.S. patients. The Company is able to reasonably estimate and determine sales allowances; therefore the Company recognizes PROCYSBI revenue in the United States at the point of sale to the specialty pharmacy. Revenue is currently recognized in the EU once confirmed orders from the pharmacies have been shipped and invoiced for payment by the distributor on the Company’s behalf. The Company records revenue net of expected discounts, distributor fees, and rebates, including government rebates such as Medicare and Medicaid in the United States. Allowances are recorded as a reduction of revenue at the time product sales are recognized. Allowances for government rebates and discounts are established based on the actual payor and payor mix information, which is known in the United States at the time of shipment to the distributor and in Europe at the time of shipment to the pharmacy, and the government-mandated discount rates applicable to government-funded programs. The allowances are adjusted to reflect known changes in the factors that may impact such allowances in the quarter the changes are known. Inventories and Cost of Sales Inventories are stated at the lower of cost or market price, with cost determined on a first-in, first-out basis. Inventories are reviewed periodically to identify slow-moving inventory based on sales activity, both projected and historical, as well as product shelf-life. Products that have been approved by the FDA or other regulatory authorities are also used in clinical programs, to assess the safety and efficacy of the products for usage in diseases or patients that have not been approved by the FDA or other regulatory authorities. The forms of PROCYSBI and QUINSAIR that are utilized for both commercial and clinical programs are identical and, as a result, the inventory has an “alternative future use” as defined in authoritative accounting guidance. Raw materials and purchased drug product associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and/or no longer can be used for commercial purposes and, therefore, does not have an “alternative future use.” Upon launching PROCYSBI in June 2013 in the United States and in April 2014 in the EU, the Company began recognizing cost of sales. Cost of sales includes the cost of inventory sold or reserved; manufacturing, manufacturing overhead and supply chain costs; inventory variance amortization; product shipping and handling costs; and amortization of licensing approval milestone payments and licensing royalties payable to the University of California, San Diego (“UCSD”). QUINSAIR is approved in the EU and Canada and marketing commenced in Germany and Denmark in April 2016. Property and Equipment Property and equipment, which mainly consist of leasehold improvements, office furniture, lab equipment and computer hardware and software, are stated at cost. Depreciation is computed using the straight-line method over the related estimated useful lives, except for leasehold improvements, which are depreciated over the shorter of the useful life of the asset or the lease term. Significant additions and improvements that have useful lives estimated at greater than one year are capitalized, while repairs and maintenance are charged to expense as incurred. Goodwill and Intangible Assets Goodwill represents the excess of the consideration transferred over the estimated fair value of assets acquired and liabilities assumed in a business combination. Intangible assets with indefinite useful lives are related to purchased in-process research and development (IPR&D) projects and are measured at their respective fair values as of the acquisition date. The Company does not amortize goodwill and intangible assets with indefinite useful lives. Intangible assets related to IPR&D projects are considered to be indefinite-lived until the completion or abandonment of the associated R&D efforts. If and when development is complete, which generally occurs if and when marketing approval is obtained, the associated assets are deemed finite-lived and are amortized based on their respective estimated useful lives at that point in time. The Company tests goodwill and other indefinite-lived intangible assets for impairment on an annual basis and in between annual tests if any events or changes occur that would indicate the fair values of the assets are below their carrying amounts. Intangible assets with finite useful lives are amortized over their estimated useful lives on a straight-line basis, and are reviewed for impairment when facts or circumstances suggest that the carrying value of these assets may not be recoverable. Common Stock Warrant Liabilities The Company previously issued common stock warrants that contained conditional obligations that may have required the Company to transfer cash to settle the warrants upon the occurrence of certain fundamental transactions. Therefore, the Company classified such warrants as liabilities. At each reporting period, the Company re-measured the common stock warrant liability at the end of every reporting period with the change in value reported in the Company’s consolidated statements of operations and comprehensive loss. At the exercise date, the fair values of these warrants were re-measured and reclassified to equity. As of December 31, 2015, all common stock warrants had been exercised or expired. Debt Issuance Costs Debt issuance costs are expenses associated with the issuance of the loan agreements with HealthCare Royalty Partners ("HC Royalty") and the convertible notes. Debt issuance costs which were capitalized are being amortized over the life of the respective debt to interest expense using the interest method. Debt issuance costs are presented as a reduction in the carrying amount of notes payable on the Company’s condensed consolidated balance sheets. Net Loss per Share Net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding and potential shares of common stock during the period. For all periods presented, potentially dilutive securities are excluded from the computation of fully diluted net loss per share as their effect is anti-dilutive. Potentially dilutive securities include: Three months Ended March 31, 2016 2015 Warrants to purchase common stock — 334,764 Options to purchase common stock 9,573,631 9,663,956 Restricted stock unit awards outstanding 820,005 212,980 Convertible debt 3,428,571 3,428,571 Total Potentially Dilutive Securities 13,822,207 13,640,271 Stock-Based Compensation Compensation costs related to the Company's stock incentive plans are measured at the grant date based on the fair value of the equity instruments awarded and are recognized over the period during which an employee is required to provide service in exchange for the award, or the requisite service period, which is usually the vesting period. Research and Development Costs Research and development costs are charged to expense as incurred. Research and development expenses primarily include salaries and benefits for medical, clinical, regulatory, quality, pharmacovigilance and research personnel, preclinical studies, clinical trials, and certain commercial drug manufacturing expenses prior to obtaining marketing approval. Income Taxes Income taxes are recorded under the liability method, under which deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to affect taxable income. Valuation allowances are established when necessary to reduce deferred tax assets to the amount expected to be realized. Based on the weight of available evidence, including cumulative losses since inception and expected future losses, the Company has determined that it is more likely than not that the deferred tax asset amount will not be realized and therefore a full valuation allowance has been provided on the Company's net deferred tax assets. The Company identifies uncertain tax positions and discloses any potential tax liability on its financial statements. The Company recognizes interest and/or penalties related to income tax matters as a component of income tax expense. As of March 31, 2016, there were no accrued uncertain tax positions or interest and penalties related to uncertain tax positions. The Company files U.S. Federal, California, various other state and other income tax returns and various foreign country income tax returns. The Company is currently not subject to any income tax examinations. Due to the Company's net operating losses ("NOLs"), generally all tax years remain open. Reclassifications Certain amounts previously reported under specific financial statement captions have been reclassified to be consistent with the current period presentation. Disclosure of Change in Accounting Policy and Retroactive Restatement Disclosure As Simplifying the Presentation of Debt Issuance Costs The Company has adopted this standard as management believes this presentation more accurately reflects the costs of borrowing for arrangements in which debt issuance costs are incurred. The implementation resulted in the decrease of assets and debt liabilities of $3.6 million and $3.9 million as of March 31, 2016 and December 31, 2015, respectively. This restatement only impacted the Consolidated Balance Sheets as of March 31, 2016 and December 31, 2015. See Notes 8 and 9 of “Notes to Consolidated Financial Statements” for more information on the unamortized debt issuance costs related to the Company's debt. Recent Accounting Pronouncements In July 2015, the FASB issued ASU 2015-11, Simplifying the Measurement of Inventory, which requires entities to measure most inventory “at the lower of cost and net realizable value,” thereby simplifying the current guidance under which an entity must measure inventory at the lower of cost or market. ASU 2015-11 is effective for the Company in the first quarter of 2017 and is to be applied prospectively. Early adoption is permitted. The Company does not expect the adoption of this amendment to have a material effect on its financial condition and results of operations. In November 2015, the FASB issued ASU 2015-17, Balance Sheet Classification of Deferred Taxes. This is part of FASB’s simplification initiative. The amendments in this ASU require that deferred tax liabilities and assets be classified as noncurrent in a classified statement of financial position. This ASU is effective for the Company in the first quarter of 2017. Early adoption is permitted. The Company does not expect the adoption of this amendment to have a material effect on its financial condition and results of operations. In January 2016, the FASB issued ASU 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities. The purpose is to enhance the reporting model for financial instruments to provide users of financial statements with more decision-useful information. This ASU is effective for the Company in the first quarter of 2018. Early adoption is not permitted except for limited provisions. The Company does not expect the adoption of this amendment to have a material effect on its financial condition and results of operations. In February 2016, the FASB issued ASU 2016-02, Leases, which for operating leases, requires a lessee to recognize a right-of-use asset and a lease liability, initially measured at the present value of the lease payments, in its balance sheet. The standard also requires a lessee to recognize a single lease cost, calculated so that the cost of the lease is allocated over the lease term, on a generally straight-line basis. ASU 2016-02 will be effective for the Company in the first quarter of 2019, and early adoption is permitted. The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements and footnote disclosures. In March 2016, the FASB issued ASU 2016-09, Improvements to Employee Share-Based Payment Accounting, which simplifies several aspects of the accounting for employee share-based payment transactions for both public and nonpublic entities, including the accounting for income taxes, forfeitures, and statutory tax withholding requirements, as well as classification in the statement of cash flows. The ASU will be effective for the Company in the first quarter of 2017, and early adoption is permitted. The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements and footnote disclosures. |
FAIR VALUE MEASUREMENT
FAIR VALUE MEASUREMENT | 3 Months Ended |
Mar. 31, 2016 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENT | 2. FAIR VALUE MEASUREMENT The Company uses a fair value approach to value certain assets and liabilities. Fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. The Company uses a fair value hierarchy, which distinguishes between assumptions based on market data (observable inputs) and an entity's own assumptions (unobservable inputs). The hierarchy consists of three levels: · Level 1 – Quoted market prices in active markets for identical assets or liabilities; · Level 2 – Inputs other than level one inputs that are either directly or indirectly observable; and · Level 3 – Unobservable inputs developed using estimates and assumptions, which are developed by the reporting entity and reflect those assumptions that a market participant would use. Determining which category an asset or liability falls within the hierarchy requires significant judgment. The Company evaluates its hierarchy disclosures each reporting period. There were no transfers between Level 1, 2 or 3 of the fair value hierarchy during the quarter ended March 31, 2016. The following table presents the assets and liabilities recorded that are reported at fair value on our consolidated balance sheets on a recurring basis. Assets and Liabilities Measured at Fair Value on a Recurring Basis (In thousands) March 31, 2016 Level 1 Level 2 Level 3 Total Assets Cash equivalents (1) $ 118,792 $ — $ — $ 118,792 Total $ 118,792 $ — $ — $ 118,792 Liabilities Contingent consideration liability $ — $ — $ 152,070 $ 152,070 Total $ — $ — $ 152,070 $ 152,070 December 31, 2015 Level 1 Level 2 Level 3 Total Assets Cash equivalents (1) $ 147,007 $ — $ — $ 147,007 Total $ 147,007 $ — $ — $ 147,007 Liabilities Contingent consideration liability $ — $ — $ 166,800 $ 166,800 Total $ — $ — $ 166,800 $ 166,800 (1) Cash equivalents represent the fair value of the Company’s investments in money market funds at March 31, 2016 and December 31, 2015. The following tables present a reconciliation of the Company’s recurring fair value measurements categorized within Level 3 of the fair value hierarchy. See Note 10 for additional information regarding the fair value of the contingent consideration liability. Changes in Level 3 Liabilities Measured at Fair Value on a Recurring Basis – Common Stock Warrants Three Months Ended March 31, (In thousands) 2016 2015 Beginning fair value — $ 711 Change in fair value recognized in earnings — 55 Exercises $ — $ — Ending Fair Value $ — $ 766 Changes in Level 3 Liabilities Measured at Fair Value on a Recurring Basis – Contingent Consideration Liability (In thousands) Balance as of December 31, 2015 $ 166,800 Fair value adjustment (14,730 ) Balance as of March 31, 2016 $ 152,070 Effect of Raptor’s Stock Price and Volatility Assumptions on the Calculation of Fair Value of Warrant Liabilities As discussed above, the Company uses the Black-Scholes option pricing model as its method of valuation for warrants that are subject to warrant liability accounting. The determination of fair value as of the reporting date is affected by Raptor’s stock price as well as assumptions regarding a number of subjective variables. These variables include, but are not limited to, expected stock price volatility over the term of the security and risk-free interest rate. The primary factors affecting the fair value of the warrant liability are the Company’s stock price and volatility. |
INVENTORIES
INVENTORIES | 3 Months Ended |
Mar. 31, 2016 | |
Inventory Disclosure [Abstract] | |
INVENTORIES | 3. INVENTORIES Inventories consist of raw materials, work-in-process and finished goods related to the manufacture of PROCYSBI and QUINSAIR. Raw materials include the active pharmaceutical ingredients (“API”), cysteamine bitartrate and Levofloxacin, for PROCYSBI and QUINSAIR, respectively. Work-in-process includes third party manufacturing and associated labor costs relating to the Company's personnel directly involved in the production process of both products. Also included in inventories are raw materials that may be used for clinical trials, which are charged to research and development (“R&D”) expense when consumed. The following table summarizes the components of inventories. March 31, December 31, (In thousands) 2016 2015 Raw materials $ 3,153 $ 2,681 Work-in-process 2,621 1,824 Finished goods 3,822 1,919 Total Inventories $ 9,596 $ 6,424 |
PROPERTY AND EQUIPMENT
PROPERTY AND EQUIPMENT | 3 Months Ended |
Mar. 31, 2016 | |
Property Plant And Equipment [Abstract] | |
PROPERTY AND EQUIPMENT | 4. PROPERTY AND EQUIPMENT The following table presents the components of property and equipment and their estimated useful lives. March 31, December 31, Estimated (In thousands) 2016 2015 useful lives Manufacturing equipment $ 4,342 $ 4,262 10 years Office furniture 2,332 2,344 7 years Laboratory equipment 1,768 1,721 5 years Computer hardware and software 1,572 1,364 3 years Leasehold improvements 586 583 Lease term Total at cost 10,600 10,274 Less: accumulated depreciation (3,006 ) (2,630 ) Total Property and Equipment, Net $ 7,594 $ 7,644 Depreciation expense for the three months ended March 31, 2016 and 2015 was approximately $373 thousand and $302 thousand, respectively. |
NET PRODUCT REVENUES BY GEOGRAP
NET PRODUCT REVENUES BY GEOGRAPHIC REGION AND BY SIGNIFICANT CUSTOMERS | 3 Months Ended |
Mar. 31, 2016 | |
Segment Reporting [Abstract] | |
NET PRODUCT REVENUES BY GEOGRAPHIC REGION AND BY SIGNIFICANT CUSTOMERS | 5. NET PRODUCT REVENUES BY GEOGRAPHIC REGION AND BY SIGNIFICANT CUSTOMERS Net product revenues by Geographic Region For Three Months Ended March 31, (In millions) 2016 2015 United States $ 25.3 $ 19.3 International 2.2 1.2 Total Net Product Revenues by Geographic Region $ 27.5 $ 20.5 Net Product Revenues by Significant Customer Sales to our significant customer, Accredo Health Services, totaled $25.3 million, or 92% for March 31, 2016 and $19.3 million, or 94.2% for March 31, 2015 of net product revenue. |
GOODWILL AND INTANGIBLE ASSETS
GOODWILL AND INTANGIBLE ASSETS | 3 Months Ended |
Mar. 31, 2016 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
GOODWILL AND INTANGIBLE ASSETS | 6. GOODWILL AND INTANGIBLE ASSETS On December 14, 2007, the Company acquired the intellectual property and other rights to develop RP103 to treat various clinical indications from UCSD by way of a merger with Encode Pharmaceuticals, Inc., a privately held development stage company ("Encode"), which held the intellectual property license with UCSD. The fair value of the intangible assets at the time of acquisition was approximately $2.6 million. Pursuant to the license agreement with UCSD, the Company is obligated to pay an annual maintenance fee until the commencement of commercial sales of any licensed products developed. The Company is also obligated to pay milestone payments upon the occurrence of certain events, royalties on net sales from products developed pursuant to the license agreement and a percentage of sublicense fees or royalties, if any. The Company is obligated to fulfill predetermined milestones within a specified number of years from the effective date of the license agreement, depending on the indication. To the extent that the Company fails to perform any of its obligations under the agreement, UCSD may terminate the license or otherwise cause the license to become non-exclusive. In April 2013, the Company announced that the FDA has approved PROCYSBI (cysteamine bitartrate) delayed release capsules for the management of nephropathic cystinosis in adults and children 6 years and older. Subsequently, the Company announced that the EC has approved PROCYSBI® gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate) as an orphan medicinal product for the management of proven nephropathic cystinosis for marketing in the EU. In conjunction with these approvals, the Company paid milestone payments to UCSD during the second and third quarters of 2013 of $0.8 million and $0.5 million, respectively, pursuant to this license, which were capitalized as intangible assets. In October 2015, the Company acquired the intellectual property and other rights to develop QUINSAIR for the management of chronic pulmonary infections due to Pseudomonas aeruginosa In addition, the purchase agreement provides for contingent payments of up to $350 million associated with development, regulatory and commercial milestones, a portion of which is also payable in Raptor common stock at the Company’s election, and a single digit royalty on future global net sales. A summary of intangible assets acquired is as follows: Useful Life March 31, December 31, (In thousands) (Years) 2016 2015 IPR&D QUINSAIR Indefinite $ 171,000 $ 210,600 Developed technology - QUINSAIR 11.0 3,200 3,200 IP license for RP103 related to the Encode merger 20.0 2,620 2,620 UCSD license - FDA and EC approval milestones 14.0 1,250 1,250 Other intangible assets 16.0 240 240 Total intangible assets 178,310 217,910 Less accumulated amortization (1,579 ) (1,447 ) Intangible Assets, Net $ 176,731 $ 216,463 The intangible assets related to the QUINSAIR developed technology are being amortized over an estimated useful life of 11 years, which is the life of the intellectual property patents. The intangible assets related to RP103 are being amortized over an estimated useful life of 20 years, which is the life of the intellectual property patents. The 14 year estimated useful life for the FDA and EMA approval milestones is based upon the typical development, approval, marketing and life cycle management timelines of pharmaceutical drug products. Other intangible assets are being amortized using the straight-line method over an estimated useful life of 16 years, which is the life of the intellectual property patents. The above definite-lived intangibles do not have any residual value beyond the assets’ useful lives. The QUINSAIR IPR&D will continue to be evaluated on a quarterly basis. During the three months ended March 31, 2016, the Company recognized an impairment in the QUINSAIR IPR&D of $39.6 million related to revisions of its clinical plans (See Note 10). During the three months ended March 31, 2016 and 2015, the Company amortized approximately $132 thousand and $60 thousand of intangible assets, respectively. Amortization expense for intangible assets for each of the next five years is expected to be as follows: (In thousands) Amortization Expense 2016 (remaining 9 months) $ 397 2017 529 2018 529 2019 529 2020 529 The Company tested the carrying value of goodwill for impairment as of December 31, 2015 and determined that there was no impairment. |
ACCRUED LIABILITIES
ACCRUED LIABILITIES | 3 Months Ended |
Mar. 31, 2016 | |
Payables And Accruals [Abstract] | |
ACCRUED LIABILITIES | 7. ACCRUED LIABILITIES Accrued liabilities consisted of: March 31, December 31, (In thousands) 2016 2015 Personnel-related costs $ 6,014 $ 7,601 Rebates and other sales deductions 2,738 2,833 Clinical trials and research and development costs 3,053 2,076 License royalty payable 2,491 1,951 Royalty-based interest payable 1,511 1,410 Manufacturing costs 2,115 1,577 Deferred rent 1,082 1,086 Travel 998 213 Business development & legal costs 822 1,030 Other 2,815 2,853 Total Accrued Liabilities $ 23,639 $ 22,630 The roll forward of significant estimated accrued rebates, reserve for cash discounts and product returns for the period ended March 31, 2016 and December 31, 2015 were as follows: Beginning Balance Provision for Current Period Sales Provision for Prior Period Sales Actual Returns/ Credits Related to Current Period Sales Actual Returns/ Credits Related to Prior Period Sales Outstanding Balance March 31, 2016 Accrued rebates $ 2,538 $ 2,566 $ — $ (18 ) $ (2,644 ) $ 2,442 Reserve for cash discounts 258 573 — (229 ) (258 ) 344 Product returns 296 — — — — 296 December 31, 2015 Accrued rebates $ 2,935 $ 6,250 $ 239 $ (3,712 ) $ (3,174 ) $ 2,538 Reserve for cash discounts 215 1,821 41 (1,563 ) (256 ) 258 Product returns 296 — — — — 296 The Company accrued approximately $2.4 million and $2.5 million for estimated rebate payments at March 31, 2016 and December 31, 2015, respectively. The Company evaluates its historical rebate payments by product as a percentage of historical sales in order to estimate its accrued rebates in proportion to revenue. Management has determined that a one-year look back represents a reasonable approach for assessing its rebate liabilities, and as of March 31, 2016 believes that it has adequately reserved for known and potentially unknown incurred rebates. The Company accrued approximately $0.3 million and $0.3 million for the estimated cost of prompt-payment discounts at March 31, 2016 and December 31, 2015, respectively. These amounts are estimated based upon payment terms with each of the Company’s customers. The Company considered the need for a reserve for possible product returns sold during the periods ended March 31, 2016 and December 31, 2015, respectively. The Company determined an allowance of $0.3 million at March 31, 2016 and December 31, 2015, respectively, was necessary for possible product returns from its distributor. These amounts are estimated based upon the timing and history of similar product sales in the pharmaceutical industry. As of March 31, 2016, no products had been returned. |
NOTE PAYABLE
NOTE PAYABLE | 3 Months Ended |
Mar. 31, 2016 | |
Debt Disclosure [Abstract] | |
NOTE PAYABLE | 8. NOTE PAYABLE On December 20, 2012, the Company entered into a loan agreement with HC Royalty, as lender, under which it agreed to borrow $50.0 million in two $25.0 million tranches. The Company received $23.4 million in net proceeds from the first tranche of the loan at closing in December 2012 and an additional $23.7 million in net proceeds in May 2013 from the second tranche upon FDA approval of PROCYSBI. In July 2014, the Company entered into an amended and restated loan agreement with HC Royalty which revised the terms of the 2012 loan agreement between the Company and HC Royalty, and also provided for an additional $10 million in term loan funding. The interest rate was revised to an annual fixed rate of 8.0%, compared to the original interest rate of 10.75%. The loan also contains a synthetic royalty component based on net product revenues in a calendar year, and such royalty is payable quarterly. The variable royalty rate under the amended and restated loan agreement has been revised to 8.0% on the first $50 million of revenue and 2.0% on revenue in excess of $50 million. The first quarterly principal payment of $3 million was due in June 2015. All term loans under the amended and restated loan agreement mature on March 31, 2020. The loan and the Company’s obligation to make payments thereunder shall terminate immediately when all payments received by HC Royalty equal $120.0 million. Prior to July 1, 2014, with respect to the first $25.0 million tranche, for each calendar year (prorated for any portion thereof), the loan bore a royalty rate of 6.25% of the first $25.0 million of product net revenues, 3.0% of product net revenues for such calendar year in excess of $25.0 million and up to $50.0 million, and 1.0% of product net revenues for such calendar year in excess of $50.0 million, payable quarterly. Prior to July 1, 2014, with respect to the second $25.0 million tranche, for each calendar year (prorated for any portion thereof), the loan bore a royalty rate of 6.0% of the first $25.0 million of net product revenues for such calendar year, 3.0% of product net revenues for such calendar year in excess of $25.0 million and up to $50.0 million, and 1.0% of product net revenues for such calendar year in excess of $50.0 million, payable quarterly. The Company’s amended and restated loan agreement with HC Royalty includes affirmative and negative covenants, including the use of commercially reasonable efforts to exploit RP103 in specific markets and compliance with laws, as well as restrictions on mergers and sales of assets, incurrence of liens, incurrence of indebtedness and transactions with affiliates and other requirements. To secure the performance of the Company’s obligations under the loan, the Company granted a security interest to HC Royalty in substantially all of its assets, the assets of its domestic subsidiaries and a pledge of stock of certain of its domestic subsidiaries. The Company’s failure to comply with the terms of the loan and related documents, the occurrence of a change of control of the Company or the occurrence of an uncured material adverse effect on the Company or the occurrence of certain other specified events, will result in an event of default under the loan that, if not cured or waived, could result in the acceleration of the payment of all of its indebtedness, as well as prepayment penalties, to HC Royalty and interest thereon. Under the terms of the security agreement, in an event of default, the lender can potentially take possession of, foreclose on, sell, assign or grant a license to use, the Company’s pledged collateral and assign and transfer the pledged stock of certain of its subsidiaries. The Company received marketing approval of PROCYSBI from the FDA on April 30, 2013 and commenced shipment of PROCYSBI during June 2013, and as a result, royalties became payable to HC Royalty based upon net revenues of PROCYSBI. Interest expense on the loan, excluding amortization of debt issuance costs, for the three months ended March 31, 2016 and 2015 was approximately $3.5 million and $3.0 million, respectively. The following table presents contractual principal payments of the note payable at March 31, 2016. (In thousands) Note 2016 (remaining 9 months) $ 9,000 2017 12,000 2018 12,000 2019 12,000 2020 3,000 Total $ 48,000 Unamortized debt issuance costs on the loan agreement totaled $1.4 million and $1.5 million at March 31, 2016 and December 31, 2015, respectively. Amortization expense was $0.2 million and $0.2 million for the three months ended March 31, 2016 and 2015, respectively. |
CONVERTIBLE NOTES
CONVERTIBLE NOTES | 3 Months Ended |
Mar. 31, 2016 | |
Convertible Notes [Abstract] | |
CONVERTIBLE NOTES | 9. CONVERTIBLE NOTES In July 2014, the Company sold $60 million aggregate principal amount of 8.0% convertible senior notes due 2019 to HC Royalty and other purchasers. These convertible notes require quarterly interest distributions at a fixed coupon rate equal to 8.0% until maturity or conversion, which will be no later than August 1, 2019. The convertible senior notes are convertible at the option of the holder at a conversion rate of 57.14 common shares per $1,000 principal amount of convertible senior notes at issuance (equivalent to a conversion price of $17.50 per common share), subject to adjustment in certain events. Upon conversion of these convertible senior notes by a holder, the holder will receive shares of the Company’s common stock. In addition, the Company may elect to exercise the optional redemption, as defined in the note purchase agreement, in which case the convertible senior notes will convert into shares of common stock if the price of the common stock is at or above 175% of the applicable conversion price over a 30 consecutive day period. Upon the occurrence of a “change of control”, as defined in the note purchase agreement, the holders may require the Company to repurchase all or a portion of the notes for cash at 100% of the principal amount of the notes being purchased, plus a repayment premium and any accrued and unpaid interest. To secure the performance of the Company's obligations under the convertible notes agreement, the Company has assigned certain of its assets as collateral. Interest expense on convertible notes, excluding amortization of debt issuance costs, was $1.2 million for the three months ended March 31, 2016 and March 31, 2015. Unamortized debt issuance costs on these convertible notes totaled $2.2 million and $2.3 million at March 31, 2016 and December 31, 2015, respectively. Amortization expense was $0.1 million and $0.1 million for the three months ended March 31, 2016 and 2015, respectively. |
BUSINESS COMBINATION
BUSINESS COMBINATION | 3 Months Ended |
Mar. 31, 2016 | |
Business Combinations [Abstract] | |
BUSINESS COMBINATION | 10. BUSINESS COMBINATION (a) Acquisition of QUINSAIR Acquisition Overview On October 5, 2015, the Company completed the acquisition of QUINSAIR from Tripex. The Company acquired exclusive global rights and assets to develop, manufacture and commercialize QUINSAIR, a levofloxacin solution for inhalation. At closing, the Company paid Tripex approximately $35.4 million in cash consideration, subject to a deduction for payment of costs for representations and warranties insurance, and an amount to be held in escrow, and issued to Tripex 3,448,001 shares of Raptor common stock. In addition, the purchase agreement provides for contingent payments of up to $350 million associated with development, regulatory and commercial milestones, a portion of which is also payable in Raptor common stock at the Company’s election, and a single digit royalty on future global net sales. The Company has single-digit royalty and contingent obligations to two additional parties involved in QUINSAIR’s development. Consideration transferred The acquisition-date fair value of the consideration transferred consisted of the following items: (In thousands) Cash consideration $ 35,370 Stock consideration 20,860 Contingent consideration 166,800 Total purchase consideration $ 223,030 Fair Value Estimate of Assets Acquired and Liability Assumed Property and equipment $ 282 Developed Technology 3,200 In-Process Research and Development 210,600 Goodwill 8,948 $ 223,030 Value of Intangible Assets Acquired Amortization Period* Developed technology $ 3,200 132 months IPR&D 210,600 (1 ) Total identifiable intangible assets $ 213,800 * Recognized on a straight-line basis. (1) IPR&D is an intangible asset classified as indefinite-lived until the completion or abandonment of the associated research and development effort, and will be amortized over an estimated useful life to be determined at the date the project is completed. IPR&D is not amortized during this period, but is periodically tested for impairment. The fair value of the acquired developed technology and IPR&D assets were estimated using the income approach. The income approach uses valuation techniques to convert future amounts to a single present amount (discounted). The measurement is based on the value indicated by current market expectations about those future amounts. Direct costs of the QUINSAIR acquisition included consulting, legal, and accounting fees which aggregated to $3.9 million. The Company estimated the acquisition date fair value of the contingent consideration payable of $166.8 million on October 5, 2015 has been calculated on a discounted and probability adjusted basis. The gross amount is payable upon the achievement of specified development, regulatory approval, sales-based milestone events or financial results. The model used in valuing this contingent consideration liability requires the use of significant estimates and assumptions including but not limited to: · estimates of revenues and operating profits related to the products or product candidates; · the probability of success for unapproved product candidates considering their stages of development; · the time and resources needed to complete the development and approval of product candidates; · the life of the potential commercialized products and associated risks, including the inherent difficulties and uncertainties in developing a product candidate such as obtaining FDA and other regulatory approvals; and · risks related to the viability of and potential alternative treatments in any future target markets. Goodwill Goodwill presented above of $8.9 million represents the difference of the QUINSAIR total purchase consideration of $223.0 million minus QUINSAIR IPR&D and Contingent Consideration Liability Fair Value as of March 31, 2016 and December 31, 2015 The QUINSAIR IPR&D and Contingent Consideration Liability will continue to be evaluated on a quarterly basis. During March 2016, t he Company revised its clinical development plans for MP-376 for the Bronchiectasis (BE) and Nontuberculous Mycobacteria (NTM) indications, the programs which drive the majority of the fair value of the IPR&D intangible assets balance related to the QUINSAIR acquisition. The revisions to the clinical development plans were a result of the following: - increased and further assessment of possible development plan options from external clinical advisory boards, external consultants, and internal clinical teams; and - strategic consideration and related reduction of the Company’s near-term operating cash expenditures and a prioritization of those programs or strategies that could have more near-term data readouts. As a result of these revisions the tables below represent the change in fair values for IPR&D and Contingent Consideration Liability: In-Process Research and Development (In thousands) Balance as of December 31, 2015 $ 210,600 Impairment loss recognized in earnings (39,600 ) Balance as of March 31, 2016 $ 171,000 Contingent Consideration Liability (In thousands) Balance as of December 31, 2015 $ 166,800 Change in fair value recognized in earnings (14,730 ) Balance as of March 31, 2016 $ 152,070 |
CAPITAL STRUCTURE
CAPITAL STRUCTURE | 3 Months Ended |
Mar. 31, 2016 | |
Stockholders Equity Note [Abstract] | |
CAPITAL STRUCTURE | 11. CAPITAL STRUCTURE Common Stock Issuance under At-The-Market ("ATM") Agreement On April 30, 2012, the Company entered into an "At-the-Market" ("ATM") Sales Agreement, with Cowen and Company, LLC ("Cowen"), under which the Company could, at its discretion, sell its common stock with a sales value of up to a maximum of $40.0 million through ATM offerings on the NASDAQ Stock Market. On July 3, 2013, the Company and Cowen amended and restated the Sales Agreement (the "Amended and Restated Sales Agreement") to increase the aggregate gross sales proceeds that could be raised to $100 million. Cowen was the sole sales agent for any sales made under the ATM for a 3.0% commission on gross proceeds. The common stock was sold at prevailing market prices at the time of the sale of common stock, and, as a result, prices varied. During the three months ended March 31, 2014, there were no shares sold under the ATM. As of December 31, 2014, the Company did not have any remaining shares available under the ATM for future sales of the Company's common stock. On September 4, 2015, the Company entered into a new ATM sales agreement, with Cowen, under which the Company may, at its discretion, sell its common stock with a sales value of up to a maximum of $75.0 million through ATM offerings on the NASDAQ Stock Market (the “2015 Sales Agreement”). Cowen is the sole sales agent for any sales made under the 2015 Sales Agreement, and the Company will pay Cowen a commission, or allow a discount, for its services in acting as agent in the sale of our common stock of up to 3.0% of the gross sales price per share of all shares sold through it as agent under the 2015 Sales Agreement. The common stock will be sold at prevailing market prices at the time of the sale of common stock, and, as a result, prices will vary. There have been no shares sold under the 2015 Sales Agreement and $75.0 million was available for issuance under the 2015 Sales Agreement at March 31, 2016. 2015 Follow-on Public Offering On April 8, 2015, the Company closed an underwritten public offering of shares of the Company’s common stock at a price to the public of $9.00 per share. The shares sold in the offering included 9.5 million shares of common stock plus an additional 1.43 million shares of common stock pursuant to the exercise by the underwriters of the over-allotment option the Company granted to them. Total gross proceeds to the Company in the offering (including in connection with the sale of the shares of common stock pursuant to the exercise of the over-allotment option) totaled $98.3 million, before underwriting discounts and commissions. The offering resulted in net proceeds to the Company of approximately $92.0 million after deduction of underwriting discounts of 6% and other offering expenses paid by the Company. |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 3 Months Ended |
Mar. 31, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
STOCK-BASED COMPENSATION | 12. STOCK-BASED COMPENSATION 2010 Stock Incentive Plan The Company's 2010 Stock Incentive Plan, as amended, provides for stock options, restricted shares or restricted share units to be granted to its employees, independent contractors, consultants or directors. On November 25, 2014, as a key requirement of the Company's strategy of strengthening its leadership team and employee base, continuing the expansion of its commercial activities into new territories, and increasing the expansion of its product development programs, the Company's Board of Directors approved the Raptor Pharmaceutical Corp. 2014 Employment Commencement Stock Incentive Plan. The plan was approved pursuant to Rule 5635(c)(4) of the Nasdaq Global Select Market for equity grants to induce new employees to enter into employment with the Company. Up to 2,400,000 shares may be issued under this plan. On May 19, 2015, at the Company’s Annual Meeting of Stockholders, the stockholders approved amendments to the Company’s 2010 Stock Incentive Plan. These amendments were previously approved by the Company’s Board of Directors in February 2015. Among other things, the 2015 Plan Amendment increased the share reserve available for issuance under the 2010 Stock Incentive Plan by 3,456,620 to an aggregate of approximately 15.4 million shares plus any shares which are subject to awards under the 2014 Commencement Plan which are forfeited or lapse unexercised and which are not issued under the 2014 Commencement Plan, all of which may be used for any form of award under the 2010 Stock Incentive Plan. Following the approval of the 2015 Plan Amendment by the Company’s stockholders, no new equity grants will be made under the 2014 Commencement Plan. During the three months ended March 31, 2016 and 2015, the Company received approximately $0.04 million and $2.2 million, respectively, from the exercise of stock options. At March 31, 2016, there were 2,553,867 shares remaining available for issuance under the 2010 Stock Incentive Plan. The Company recorded employee stock-based compensation expense as follows: Three Months Ended March 31, (In thousands) 2016 2015 Cost of goods sold $ 120 $ 32 Research and development 590 584 General and administrative 1,948 2,237 Total Stock-Based Compensation Expense $ 2,658 $ 2,853 A summary of the activity in the 2010 Equity Incentive Plan, the 2006 Equity Compensation Plan, as amended and the Company's other stock option plans, is as follows: For Three Months Ended March 31,2016 Option Shares Weighted- average Exercise Price Beginning balance 8,790,474 $ 8.49 Granted 1,436,631 3.83 Exercised (15,773 ) 2.57 Canceled (637,701 ) 10.15 Outstanding Balance at Year End 9,573,631 7.69 Restricted Stock Units At March 31, 2016, there were 820,005 restricted stock units (“RSUs”) outstanding. There were 450,426 RSUs granted, 38,523 vested/distributed, and 40,675 RSUs forfeited related to employee departures during the three months ended March 31, 2016. During the twelve months ended December 31, 2015, there were 509,967 RSUs granted, 10,044 RSUs distributed, and 51,146 shares forfeited. Unvested RSUs at March 31, 2016 vest through 2020. Employee Stock Purchase Plan The Employee Stock Purchase Plan (“ESPP”) allows a maximum of 1,000,000 shares of common stock to be purchased in aggregate for all employees. At March 31, 2016, 162,498 shares had been purchased, and there remained a negligible amount of uninvested employee contributions in the ESPP. As of March 31, 2016, there were approximately 837,502 shares reserved for future issuance under the ESPP. |
INCOME TAXES
INCOME TAXES | 3 Months Ended |
Mar. 31, 2016 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | 13. INCOME TAXES We apply an estimated annual effective tax rate (“ETR”) approach for calculating a tax provision for interim periods, as required under GAAP. We recorded a provision for income taxes of $0.3 million and $0.02 million for the three months ended March 31, 2016 and 2015, respectively. Our ETR differs from the U.S. federal statutory tax rate of 34% primarily as a result of nondeductible expenses, state income taxes, foreign income taxes, and the impact of a valuation allowance on our deferred tax assets. We recognize excess tax benefits associated with share-based compensation to stockholders’ equity only when realized. When assessing whether excess tax benefits relating to share-based compensation have been realized, we follow the with-and-without approach, excluding any indirect effects of the excess tax deductions. Under this approach, excess tax benefits related to share-based compensation are not deemed to be realized until after the utilization of all other tax benefits available to us. We recognize the impact of a tax position in our financial statements only if that position is more likely than not of being sustained upon examination by taxing authorities, based on the technical merits of the position. Any interest and penalties related to uncertain tax positions will be reflected in income tax expense. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 3 Months Ended |
Mar. 31, 2016 | |
Commitments And Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | 14. COMMITMENTS AND CONTINGENCIES Contractual Obligations with UCSD Relating to the Acquisition of the DR Cysteamine (RP103) License Pursuant to the license agreement with UCSD, the Company is obligated to pay milestone payments upon the occurrence of certain events, royalties on net sales from products developed pursuant to the license agreement and a percentage of sublicense fees or sublicense royalties, if any. The Company is obligated to fulfill predetermined milestones within a specified number of years from the effective date of the license agreement, depending on the indication. Cumulatively, the Company has expensed $2.2 million in milestone payments to UCSD based upon the initiation of clinical trials in cystinosis, Huntington’s disease and NASH and on regulatory filings in cystinosis. To the extent that the Company fails to perform any of its obligations under the license agreement, then UCSD may terminate the license or otherwise cause the license to become non-exclusive. Quinsair Contingent Consideration Liability The Quinsair purchase agreement provides for contingent payments of up to $350 million associated with development, regulatory and commercial milestones, a portion of which is also payable in Raptor common stock at the Company’s election, and a single digit royalty on future global net sales. Leases In January 2016, the Company entered into a four-year lease for additional office space in South San Francisco. The Company took occupancy of such facilities in February 2016. The Company will record such rent on a straight-line basis. In April 2013, the Company executed a seven-year lease for its corporate office facilities in Novato, California. The Company took occupancy of such facilities at the end of June 2013. On June 10, 2013, the Company amended the lease to add space to accommodate its research laboratory ad relocated to this space in July 2014. The Company records such rent on a straight-line basis. In October 2014, the Company executed a three-year lease for its European sales, marketing and administrative headquarters in Utrecht, Netherlands. The Company records such rent on a straight-line basis. |
DESCRIPTION OF BUSINESS AND S20
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 3 Months Ended |
Mar. 31, 2016 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The Company's consolidated financial statements include the accounts of the Company's direct and indirect wholly owned subsidiaries, Raptor Pharmaceuticals Inc., formerly known as Raptor Therapeutics Inc. which merged with Raptor Discoveries Inc. in December 2012 prior to changing its name, and Raptor European Products, LLC, such subsidiaries incorporated in Delaware on August 1, 2007, and February 14, 2012, respectively, and Raptor Pharmaceuticals Europe B.V. (“BV”), Raptor Pharmaceuticals France SAS (“SAS”), Raptor Pharmaceuticals Germany GmbH (“GMBH”) and RPTP European Holdings C.V. (“CV”), domiciled in the Netherlands on December 15, 2009, in France on October 30, 2012, in Germany on October 16, 2013 and in the Cayman Islands on February 16, 2012, respectively. All inter-company accounts have been eliminated. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make certain estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the dates of the consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. |
Functional Currency | Functional Currency The Company's consolidated functional currency is the U.S. dollar. BV, SAS, and GMBH, the Company's Dutch subsidiary, French subsidiary, and German Subsidiary, respectively, use the European Euro as their functional currency. The CV subsidiary, a Cayman-based subsidiary, uses the dollar as its functional currency. At each quarter end, each foreign subsidiary's balance sheets are translated into U.S. dollars based upon the quarter-end exchange rate, while their statements of operations and comprehensive loss are translated into U.S. dollars based upon an average exchange rate during the period. |
Segment Information | Segment Information The Company has determined that it operates in only one segment, as it only reports profit and loss information on an aggregate basis to its chief operating decision maker. The Company's long-lived assets maintained outside the U.S. are not material. |
Fair Value of Financial Instruments and Contingent Consideration Liability | Fair Value of Financial Instruments and Contingent Consideration Liability The carrying amounts of certain of the Company's financial instruments including cash equivalents, restricted cash, accounts payable, accrued liabilities, and capital lease liability approximate fair value due either to length of maturity or interest rates that approximate prevailing market rates. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less, when purchased, to be cash equivalents. The Company maintains cash and cash equivalents, which consist principally of money market funds, with high credit quality financial institutions. Such amounts exceed Federal Deposit Insurance Corporation insurance limits. As of March 31, 2016, the Company had $132 million in cash and cash equivalents, of which $5.6 million was held by its foreign subsidiaries. |
Restricted Cash | Restricted Cash Restricted cash represents certificates of deposit and compensating balances required by the Company’s U.S. and European banks as collateral for credit cards and for access to a value-added tax deferral program. |
Accounts Receivable and Allowance for Doubtful Accounts | Accounts Receivable and Allowance for Doubtful Accounts Accounts receivable are recorded and carried at the original invoiced amount less an allowance for any potential uncollectible amounts. The Company estimates its allowance for doubtful accounts based upon an assessment of various factors, including historical experience, the age of the accounts receivable balances, credit quality of customers, current economic conditions, and other factors that may affect customers’ ability to pay. To date, the Company has not experienced significant losses with respect to the collection of accounts receivable. |
Revenue Recognition | Revenue Recognition The Company recognizes revenue in accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Codification (“ASC”) 605, Revenue Recognition, when the following criteria have been met: persuasive evidence of an arrangement exists; delivery has occurred and risk of loss has passed; the seller's price to the buyer is fixed or determinable and collectability is reasonably assured. The Company determines that persuasive evidence of an arrangement exists based on written contracts that define the terms of the arrangements. Pursuant to the contract terms, the Company determines when title to products and associated risk of loss has passed on to the customer. The Company assesses whether the fee is fixed or determinable based on the payment terms associated with the transaction and whether the sales price is subject to refund or adjustment. The Company assesses collectability based primarily on the customer's payment history and creditworthiness. PROCYSBI is currently available for U.S. distribution from the Company's U.S. specialty pharmacy partner, the Accredo Health Group, Inc. ("Accredo") which is currently the Company's only U.S. customer and ships directly to patients. The Company's distributor in the EU is the Almac Group, Ltd. PROCYSBI is not available in U.S. retail pharmacies. Authorization of coverage by patients' commercial insurance plans, Raptor's patient assistance program ("PAP") or government payors is a prerequisite to the shipment of PROCYSBI to U.S. patients. The Company is able to reasonably estimate and determine sales allowances; therefore the Company recognizes PROCYSBI revenue in the United States at the point of sale to the specialty pharmacy. Revenue is currently recognized in the EU once confirmed orders from the pharmacies have been shipped and invoiced for payment by the distributor on the Company’s behalf. The Company records revenue net of expected discounts, distributor fees, and rebates, including government rebates such as Medicare and Medicaid in the United States. Allowances are recorded as a reduction of revenue at the time product sales are recognized. Allowances for government rebates and discounts are established based on the actual payor and payor mix information, which is known in the United States at the time of shipment to the distributor and in Europe at the time of shipment to the pharmacy, and the government-mandated discount rates applicable to government-funded programs. The allowances are adjusted to reflect known changes in the factors that may impact such allowances in the quarter the changes are known. |
Inventories and Cost of Sales | Inventories and Cost of Sales Inventories are stated at the lower of cost or market price, with cost determined on a first-in, first-out basis. Inventories are reviewed periodically to identify slow-moving inventory based on sales activity, both projected and historical, as well as product shelf-life. Products that have been approved by the FDA or other regulatory authorities are also used in clinical programs, to assess the safety and efficacy of the products for usage in diseases or patients that have not been approved by the FDA or other regulatory authorities. The forms of PROCYSBI and QUINSAIR that are utilized for both commercial and clinical programs are identical and, as a result, the inventory has an “alternative future use” as defined in authoritative accounting guidance. Raw materials and purchased drug product associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and/or no longer can be used for commercial purposes and, therefore, does not have an “alternative future use.” Upon launching PROCYSBI in June 2013 in the United States and in April 2014 in the EU, the Company began recognizing cost of sales. Cost of sales includes the cost of inventory sold or reserved; manufacturing, manufacturing overhead and supply chain costs; inventory variance amortization; product shipping and handling costs; and amortization of licensing approval milestone payments and licensing royalties payable to the University of California, San Diego (“UCSD”). QUINSAIR is approved in the EU and Canada and marketing commenced in Germany and Denmark in April 2016. |
Property and Equipment | Property and Equipment Property and equipment, which mainly consist of leasehold improvements, office furniture, lab equipment and computer hardware and software, are stated at cost. Depreciation is computed using the straight-line method over the related estimated useful lives, except for leasehold improvements, which are depreciated over the shorter of the useful life of the asset or the lease term. Significant additions and improvements that have useful lives estimated at greater than one year are capitalized, while repairs and maintenance are charged to expense as incurred. |
Goodwill and Intangible Assets | Goodwill and Intangible Assets Goodwill represents the excess of the consideration transferred over the estimated fair value of assets acquired and liabilities assumed in a business combination. Intangible assets with indefinite useful lives are related to purchased in-process research and development (IPR&D) projects and are measured at their respective fair values as of the acquisition date. The Company does not amortize goodwill and intangible assets with indefinite useful lives. Intangible assets related to IPR&D projects are considered to be indefinite-lived until the completion or abandonment of the associated R&D efforts. If and when development is complete, which generally occurs if and when marketing approval is obtained, the associated assets are deemed finite-lived and are amortized based on their respective estimated useful lives at that point in time. The Company tests goodwill and other indefinite-lived intangible assets for impairment on an annual basis and in between annual tests if any events or changes occur that would indicate the fair values of the assets are below their carrying amounts. Intangible assets with finite useful lives are amortized over their estimated useful lives on a straight-line basis, and are reviewed for impairment when facts or circumstances suggest that the carrying value of these assets may not be recoverable. |
Common Stock Warrant Liabilities | Common Stock Warrant Liabilities The Company previously issued common stock warrants that contained conditional obligations that may have required the Company to transfer cash to settle the warrants upon the occurrence of certain fundamental transactions. Therefore, the Company classified such warrants as liabilities. At each reporting period, the Company re-measured the common stock warrant liability at the end of every reporting period with the change in value reported in the Company’s consolidated statements of operations and comprehensive loss. At the exercise date, the fair values of these warrants were re-measured and reclassified to equity. As of December 31, 2015, all common stock warrants had been exercised or expired. |
Debt Issuance Costs | Debt Issuance Costs Debt issuance costs are expenses associated with the issuance of the loan agreements with HealthCare Royalty Partners ("HC Royalty") and the convertible notes. Debt issuance costs which were capitalized are being amortized over the life of the respective debt to interest expense using the interest method. Debt issuance costs are presented as a reduction in the carrying amount of notes payable on the Company’s condensed consolidated balance sheets. |
Net Loss per Share | Net Loss per Share Net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding and potential shares of common stock during the period. For all periods presented, potentially dilutive securities are excluded from the computation of fully diluted net loss per share as their effect is anti-dilutive. Potentially dilutive securities include: Three months Ended March 31, 2016 2015 Warrants to purchase common stock — 334,764 Options to purchase common stock 9,573,631 9,663,956 Restricted stock unit awards outstanding 820,005 212,980 Convertible debt 3,428,571 3,428,571 Total Potentially Dilutive Securities 13,822,207 13,640,271 |
Stock-Based Compensation | Stock-Based Compensation Compensation costs related to the Company's stock incentive plans are measured at the grant date based on the fair value of the equity instruments awarded and are recognized over the period during which an employee is required to provide service in exchange for the award, or the requisite service period, which is usually the vesting period. |
Research and Development Costs | Research and Development Costs Research and development costs are charged to expense as incurred. Research and development expenses primarily include salaries and benefits for medical, clinical, regulatory, quality, pharmacovigilance and research personnel, preclinical studies, clinical trials, and certain commercial drug manufacturing expenses prior to obtaining marketing approval. |
Income Taxes | Income Taxes Income taxes are recorded under the liability method, under which deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to affect taxable income. Valuation allowances are established when necessary to reduce deferred tax assets to the amount expected to be realized. Based on the weight of available evidence, including cumulative losses since inception and expected future losses, the Company has determined that it is more likely than not that the deferred tax asset amount will not be realized and therefore a full valuation allowance has been provided on the Company's net deferred tax assets. The Company identifies uncertain tax positions and discloses any potential tax liability on its financial statements. The Company recognizes interest and/or penalties related to income tax matters as a component of income tax expense. As of March 31, 2016, there were no accrued uncertain tax positions or interest and penalties related to uncertain tax positions. The Company files U.S. Federal, California, various other state and other income tax returns and various foreign country income tax returns. The Company is currently not subject to any income tax examinations. Due to the Company's net operating losses ("NOLs"), generally all tax years remain open. |
Reclassifications | Reclassifications Certain amounts previously reported under specific financial statement captions have been reclassified to be consistent with the current period presentation. |
Disclosure of Change in Accounting Policy and Retroactive Restatement Disclosure | Disclosure of Change in Accounting Policy and Retroactive Restatement Disclosure As Simplifying the Presentation of Debt Issuance Costs The Company has adopted this standard as management believes this presentation more accurately reflects the costs of borrowing for arrangements in which debt issuance costs are incurred. The implementation resulted in the decrease of assets and debt liabilities of $3.6 million and $3.9 million as of March 31, 2016 and December 31, 2015, respectively. This restatement only impacted the Consolidated Balance Sheets as of March 31, 2016 and December 31, 2015. See Notes 8 and 9 of “Notes to Consolidated Financial Statements” for more information on the unamortized debt issuance costs related to the Company's debt. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In July 2015, the FASB issued ASU 2015-11, Simplifying the Measurement of Inventory, which requires entities to measure most inventory “at the lower of cost and net realizable value,” thereby simplifying the current guidance under which an entity must measure inventory at the lower of cost or market. ASU 2015-11 is effective for the Company in the first quarter of 2017 and is to be applied prospectively. Early adoption is permitted. The Company does not expect the adoption of this amendment to have a material effect on its financial condition and results of operations. In November 2015, the FASB issued ASU 2015-17, Balance Sheet Classification of Deferred Taxes. This is part of FASB’s simplification initiative. The amendments in this ASU require that deferred tax liabilities and assets be classified as noncurrent in a classified statement of financial position. This ASU is effective for the Company in the first quarter of 2017. Early adoption is permitted. The Company does not expect the adoption of this amendment to have a material effect on its financial condition and results of operations. In January 2016, the FASB issued ASU 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities. The purpose is to enhance the reporting model for financial instruments to provide users of financial statements with more decision-useful information. This ASU is effective for the Company in the first quarter of 2018. Early adoption is not permitted except for limited provisions. The Company does not expect the adoption of this amendment to have a material effect on its financial condition and results of operations. In February 2016, the FASB issued ASU 2016-02, Leases, which for operating leases, requires a lessee to recognize a right-of-use asset and a lease liability, initially measured at the present value of the lease payments, in its balance sheet. The standard also requires a lessee to recognize a single lease cost, calculated so that the cost of the lease is allocated over the lease term, on a generally straight-line basis. ASU 2016-02 will be effective for the Company in the first quarter of 2019, and early adoption is permitted. The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements and footnote disclosures. In March 2016, the FASB issued ASU 2016-09, Improvements to Employee Share-Based Payment Accounting, which simplifies several aspects of the accounting for employee share-based payment transactions for both public and nonpublic entities, including the accounting for income taxes, forfeitures, and statutory tax withholding requirements, as well as classification in the statement of cash flows. The ASU will be effective for the Company in the first quarter of 2017, and early adoption is permitted. The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements and footnote disclosures. |
DESCRIPTION OF BUSINESS AND S21
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Accounting Policies [Abstract] | |
Potentially dilutive securities | Potentially dilutive securities include: Three months Ended March 31, 2016 2015 Warrants to purchase common stock — 334,764 Options to purchase common stock 9,573,631 9,663,956 Restricted stock unit awards outstanding 820,005 212,980 Convertible debt 3,428,571 3,428,571 Total Potentially Dilutive Securities 13,822,207 13,640,271 |
FAIR VALUE MEASUREMENT (Tables)
FAIR VALUE MEASUREMENT (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Assets and Liabilities Measured at Fair Value on a Recurring Basis | Assets and Liabilities Measured at Fair Value on a Recurring Basis (In thousands) March 31, 2016 Level 1 Level 2 Level 3 Total Assets Cash equivalents (1) $ 118,792 $ — $ — $ 118,792 Total $ 118,792 $ — $ — $ 118,792 Liabilities Contingent consideration liability $ — $ — $ 152,070 $ 152,070 Total $ — $ — $ 152,070 $ 152,070 December 31, 2015 Level 1 Level 2 Level 3 Total Assets Cash equivalents (1) $ 147,007 $ — $ — $ 147,007 Total $ 147,007 $ — $ — $ 147,007 Liabilities Contingent consideration liability $ — $ — $ 166,800 $ 166,800 Total $ — $ — $ 166,800 $ 166,800 (1) Cash equivalents represent the fair value of the Company’s investments in money market funds at March 31, 2016 and December 31, 2015. |
Common Stock Warrants [Member] | |
Summary of Level 3 Liabilities Measured at Fair Value on Recurring Basis | Changes in Level 3 Liabilities Measured at Fair Value on a Recurring Basis – Common Stock Warrants Three Months Ended March 31, (In thousands) 2016 2015 Beginning fair value — $ 711 Change in fair value recognized in earnings — 55 Exercises $ — $ — Ending Fair Value $ — $ 766 |
Contingent Consideration Liability [Member] | |
Summary of Level 3 Liabilities Measured at Fair Value on Recurring Basis | Changes in Level 3 Liabilities Measured at Fair Value on a Recurring Basis – Contingent Consideration Liability (In thousands) Balance as of December 31, 2015 $ 166,800 Fair value adjustment (14,730 ) Balance as of March 31, 2016 $ 152,070 |
INVENTORIES (Tables)
INVENTORIES (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Inventory Disclosure [Abstract] | |
Components of Inventory | The following table summarizes the components of inventories. March 31, December 31, (In thousands) 2016 2015 Raw materials $ 3,153 $ 2,681 Work-in-process 2,621 1,824 Finished goods 3,822 1,919 Total Inventories $ 9,596 $ 6,424 |
PROPERTY AND EQUIPMENT (Tables)
PROPERTY AND EQUIPMENT (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Property Plant And Equipment [Abstract] | |
Components of Property and Equipment | The following table presents the components of property and equipment and their estimated useful lives. March 31, December 31, Estimated (In thousands) 2016 2015 useful lives Manufacturing equipment $ 4,342 $ 4,262 10 years Office furniture 2,332 2,344 7 years Laboratory equipment 1,768 1,721 5 years Computer hardware and software 1,572 1,364 3 years Leasehold improvements 586 583 Lease term Total at cost 10,600 10,274 Less: accumulated depreciation (3,006 ) (2,630 ) Total Property and Equipment, Net $ 7,594 $ 7,644 |
NET PRODUCT REVENUES BY GEOGR25
NET PRODUCT REVENUES BY GEOGRAPHIC REGION AND BY SIGNIFICANT CUSTOMERS (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Segment Reporting [Abstract] | |
Summary of Net Product Revenue by Geographic Region | Net product revenues by Geographic Region For Three Months Ended March 31, (In millions) 2016 2015 United States $ 25.3 $ 19.3 International 2.2 1.2 Total Net Product Revenues by Geographic Region $ 27.5 $ 20.5 |
GOODWILL AND INTANGIBLE ASSETS
GOODWILL AND INTANGIBLE ASSETS (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Summary of intangibles assets acquired | A summary of intangible assets acquired is as follows: Useful Life March 31, December 31, (In thousands) (Years) 2016 2015 IPR&D QUINSAIR Indefinite $ 171,000 $ 210,600 Developed technology - QUINSAIR 11.0 3,200 3,200 IP license for RP103 related to the Encode merger 20.0 2,620 2,620 UCSD license - FDA and EC approval milestones 14.0 1,250 1,250 Other intangible assets 16.0 240 240 Total intangible assets 178,310 217,910 Less accumulated amortization (1,579 ) (1,447 ) Intangible Assets, Net $ 176,731 $ 216,463 |
Estimated amortization expense for intangible assets | Amortization expense for intangible assets for each of the next five years is expected to be as follows: (In thousands) Amortization Expense 2016 (remaining 9 months) $ 397 2017 529 2018 529 2019 529 2020 529 |
ACCRUED LIABILITIES (Tables)
ACCRUED LIABILITIES (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Payables And Accruals [Abstract] | |
Components of Accrued Liabilities | Accrued liabilities consisted of: March 31, December 31, (In thousands) 2016 2015 Personnel-related costs $ 6,014 $ 7,601 Rebates and other sales deductions 2,738 2,833 Clinical trials and research and development costs 3,053 2,076 License royalty payable 2,491 1,951 Royalty-based interest payable 1,511 1,410 Manufacturing costs 2,115 1,577 Deferred rent 1,082 1,086 Travel 998 213 Business development & legal costs 822 1,030 Other 2,815 2,853 Total Accrued Liabilities $ 23,639 $ 22,630 |
Schedule of Significant Estimated Accrued Rebates Reserve for Cash Discounts and Product Returns | The roll forward of significant estimated accrued rebates, reserve for cash discounts and product returns for the period ended March 31, 2016 and December 31, 2015 were as follows: Beginning Balance Provision for Current Period Sales Provision for Prior Period Sales Actual Returns/ Credits Related to Current Period Sales Actual Returns/ Credits Related to Prior Period Sales Outstanding Balance March 31, 2016 Accrued rebates $ 2,538 $ 2,566 $ — $ (18 ) $ (2,644 ) $ 2,442 Reserve for cash discounts 258 573 — (229 ) (258 ) 344 Product returns 296 — — — — 296 December 31, 2015 Accrued rebates $ 2,935 $ 6,250 $ 239 $ (3,712 ) $ (3,174 ) $ 2,538 Reserve for cash discounts 215 1,821 41 (1,563 ) (256 ) 258 Product returns 296 — — — — 296 |
NOTE PAYABLE (Tables)
NOTE PAYABLE (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Debt Disclosure [Abstract] | |
Contractual principal payments of the note payable | The following table presents contractual principal payments of the note payable at March 31, 2016. (In thousands) Note 2016 (remaining 9 months) $ 9,000 2017 12,000 2018 12,000 2019 12,000 2020 3,000 Total $ 48,000 |
BUSINESS COMBINATION (Tables)
BUSINESS COMBINATION (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Summary of intangibles assets acquired | A summary of intangible assets acquired is as follows: Useful Life March 31, December 31, (In thousands) (Years) 2016 2015 IPR&D QUINSAIR Indefinite $ 171,000 $ 210,600 Developed technology - QUINSAIR 11.0 3,200 3,200 IP license for RP103 related to the Encode merger 20.0 2,620 2,620 UCSD license - FDA and EC approval milestones 14.0 1,250 1,250 Other intangible assets 16.0 240 240 Total intangible assets 178,310 217,910 Less accumulated amortization (1,579 ) (1,447 ) Intangible Assets, Net $ 176,731 $ 216,463 |
Tripex Pharmaceuticals LLC [Member] | |
Schedule of Acquisition-Date Fair Value of Consideration Transferred | The acquisition-date fair value of the consideration transferred consisted of the following items: (In thousands) Cash consideration $ 35,370 Stock consideration 20,860 Contingent consideration 166,800 Total purchase consideration $ 223,030 |
Schedule of Fair Value Estimate of Assets Acquired and Liability Assumed | Fair Value Estimate of Assets Acquired and Liability Assumed Property and equipment $ 282 Developed Technology 3,200 In-Process Research and Development 210,600 Goodwill 8,948 $ 223,030 |
Summary of intangibles assets acquired | Value of Intangible Assets Acquired Amortization Period* Developed technology $ 3,200 132 months IPR&D 210,600 (1 ) Total identifiable intangible assets $ 213,800 * Recognized on a straight-line basis. (1) IPR&D is an intangible asset classified as indefinite-lived until the completion or abandonment of the associated research and development effort, and will be amortized over an estimated useful life to be determined at the date the project is completed. IPR&D is not amortized during this period, but is periodically tested for impairment. |
Summary of Change in Fair Values for IPR&D | As a result of these revisions the tables below represent the change in fair values for IPR&D and Contingent Consideration Liability: In-Process Research and Development (In thousands) Balance as of December 31, 2015 $ 210,600 Impairment loss recognized in earnings (39,600 ) Balance as of March 31, 2016 $ 171,000 |
Summary of Change in Fair Values for Contingent Consideration Liability | Contingent Consideration Liability (In thousands) Balance as of December 31, 2015 $ 166,800 Change in fair value recognized in earnings (14,730 ) Balance as of March 31, 2016 $ 152,070 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 3 Months Ended |
Mar. 31, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Employee and consultant stock-based compensation expense | The Company recorded employee stock-based compensation expense as follows: Three Months Ended March 31, (In thousands) 2016 2015 Cost of goods sold $ 120 $ 32 Research and development 590 584 General and administrative 1,948 2,237 Total Stock-Based Compensation Expense $ 2,658 $ 2,853 |
Summary of the activity in stock option plan | A summary of the activity in the 2010 Equity Incentive Plan, the 2006 Equity Compensation Plan, as amended and the Company's other stock option plans, is as follows: For Three Months Ended March 31,2016 Option Shares Weighted- average Exercise Price Beginning balance 8,790,474 $ 8.49 Granted 1,436,631 3.83 Exercised (15,773 ) 2.57 Canceled (637,701 ) 10.15 Outstanding Balance at Year End 9,573,631 7.69 |
DESCRIPTION OF BUSINESS AND S31
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Additional Information (Details) | 3 Months Ended | |||
Mar. 31, 2016USD ($)Segment | Dec. 31, 2015USD ($) | Mar. 31, 2015USD ($) | Dec. 31, 2014USD ($) | |
Accounting Policies [Abstract] | ||||
Decrease of assets and debt liabilities | $ 3,600,000 | $ 3,900,000 | ||
Minimum age of children for management of nephropathic cystinosis | 6 years | |||
Period of orphan drug exclusivity for PROCYSBI in U.S. | 7 years | |||
Period of orphan drug exclusivity for PROCYSBI in EU | 10 years | |||
Segment Information [Abstract] | ||||
Number of segments | Segment | 1 | |||
Cash and Cash Equivalents [Abstract] | ||||
Cash and cash equivalents | $ 132,038,000 | $ 157,352,000 | $ 134,537,000 | $ 149,613,000 |
Cash and cash equivalents held by foreign subsidiaries | $ 5,600,000 | |||
Fixed Assets [Abstract] | ||||
Useful life of significant additions and improvements in fixed assets for capitalization, minimum | 1 year | |||
Income taxes [Abstract] | ||||
Accrued interest or penalties related to uncertain tax positions | $ 0 |
DESCRIPTION OF BUSINESS AND S32
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Potentially Dilutive Securities (Details) - shares | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total potentially dilutive securities (in shares) | 13,822,207 | 13,640,271 |
Warrants to Purchase Common Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total potentially dilutive securities (in shares) | 334,764 | |
Options to Purchase Common Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total potentially dilutive securities (in shares) | 9,573,631 | 9,663,956 |
Restricted stock unit awards outstanding [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total potentially dilutive securities (in shares) | 820,005 | 212,980 |
Convertible Debt [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total potentially dilutive securities (in shares) | 3,428,571 | 3,428,571 |
FAIR VALUE MEASUREMENT - Additi
FAIR VALUE MEASUREMENT - Additional Information (Details) | 3 Months Ended |
Mar. 31, 2016 | |
Fair Value Disclosures [Abstract] | |
Transfers between measurement levels | There were no transfers between Level 1, 2 or 3 of the fair value hierarchy during the quarter ended March 31, 2016. |
FAIR VALUE MEASUREMENT - Assets
FAIR VALUE MEASUREMENT - Assets and Liabilities Measured at Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands | Mar. 31, 2016 | Dec. 31, 2015 | Oct. 05, 2015 | |
Liabilities [Abstract] | ||||
Contingent consideration liability | $ 152,070 | $ 166,800 | $ 166,800 | |
Recurring [Member] | ||||
ASSETS | ||||
Cash equivalents | [1] | 118,792 | 147,007 | |
Total | 118,792 | 147,007 | ||
Liabilities [Abstract] | ||||
Contingent consideration liability | 152,070 | 166,800 | ||
Total | 152,070 | 166,800 | ||
Recurring [Member] | Level 1 [Member] | ||||
ASSETS | ||||
Cash equivalents | [1] | 118,792 | 147,007 | |
Total | 118,792 | 147,007 | ||
Liabilities [Abstract] | ||||
Contingent consideration liability | 0 | 0 | ||
Total | 0 | 0 | ||
Recurring [Member] | Level 2 [Member] | ||||
ASSETS | ||||
Cash equivalents | [1] | 0 | 0 | |
Total | 0 | 0 | ||
Liabilities [Abstract] | ||||
Contingent consideration liability | 0 | 0 | ||
Total | 0 | 0 | ||
Recurring [Member] | Level 3 [Member] | ||||
ASSETS | ||||
Cash equivalents | [1] | 0 | 0 | |
Total | 0 | 0 | ||
Liabilities [Abstract] | ||||
Contingent consideration liability | 152,070 | 166,800 | ||
Total | $ 152,070 | $ 166,800 | ||
[1] | Cash equivalents represent the fair value of the Company’s investments in money market funds at March 31, 2016 and December 31, 2015. |
FAIR VALUE MEASUREMENT - Summar
FAIR VALUE MEASUREMENT - Summary of Level 3 Liabilities Measured at Fair Value on Recurring Basis (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Common Stock Warrants [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Beginning balance | $ 711 | |
Change in fair value recognized in earnings | 55 | |
Ending balance | $ 766 | |
Contingent Consideration Liability [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Beginning balance | $ 166,800 | |
Fair value adjustment | (14,730) | |
Ending balance | $ 152,070 |
INVENTORIES - Components of Inv
INVENTORIES - Components of Inventory (Details) - USD ($) $ in Thousands | Mar. 31, 2016 | Dec. 31, 2015 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 3,153 | $ 2,681 |
Work-in-process | 2,621 | 1,824 |
Finished goods | 3,822 | 1,919 |
Total Inventories | $ 9,596 | $ 6,424 |
PROPERTY AND EQUIPMENT - Compon
PROPERTY AND EQUIPMENT - Components of Property and Equipment (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Dec. 31, 2015 | |
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 10,600 | $ 10,274 |
Less: accumulated depreciation | (3,006) | (2,630) |
Total Property and Equipment, Net | 7,594 | 7,644 |
Manufacturing Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 4,342 | 4,262 |
Estimated useful lives | 10 years | |
Office Furniture [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 2,332 | 2,344 |
Estimated useful lives | 7 years | |
Laboratory Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 1,768 | 1,721 |
Estimated useful lives | 5 years | |
Computer Hardware and Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 1,572 | 1,364 |
Estimated useful lives | 3 years | |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 586 | $ 583 |
Estimated useful lives | Lease term |
PROPERTY AND EQUIPMENT - Additi
PROPERTY AND EQUIPMENT - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Property Plant And Equipment [Abstract] | ||
Depreciation expense excluding foreign exchange | $ 373 | $ 302 |
NET PRODUCT REVENUES BY GEOGR39
NET PRODUCT REVENUES BY GEOGRAPHIC REGION AND BY SIGNIFICANT CUSTOMERS - Summary of Net Product Revenue by Geographic Region (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Entity Wide Revenue Major Customer [Line Items] | ||
Total Net Product Revenues | $ 27,470 | $ 20,453 |
United States [Member] | ||
Entity Wide Revenue Major Customer [Line Items] | ||
Total Net Product Revenues | 25,300 | 19,300 |
International [Member] | ||
Entity Wide Revenue Major Customer [Line Items] | ||
Total Net Product Revenues | $ 2,200 | $ 1,200 |
NET PRODUCT REVENUES BY GEOGR40
NET PRODUCT REVENUES BY GEOGRAPHIC REGION AND BY SIGNIFICANT CUSTOMERS - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Entity Wide Revenue Major Customer [Line Items] | ||
Total Net Product Revenues | $ 27,470 | $ 20,453 |
Accredo Health Services [Member] | ||
Entity Wide Revenue Major Customer [Line Items] | ||
Total Net Product Revenues | $ 25,300 | $ 19,300 |
Accredo Health Services [Member] | Sales [Member] | Customer Concentration Risk | ||
Entity Wide Revenue Major Customer [Line Items] | ||
Percentage of sales revenue | 92.00% | 94.20% |
GOODWILL AND INTANGIBLE ASSET41
GOODWILL AND INTANGIBLE ASSETS - Additional Information (Details) - USD ($) | Oct. 05, 2015 | Mar. 31, 2016 | Mar. 31, 2015 | Sep. 30, 2013 | Jun. 30, 2013 | Dec. 31, 2015 | Oct. 31, 2015 | Dec. 14, 2007 | |
Summary of intangible assets [Abstract] | |||||||||
Maximum age of children for management of nephropathic cystinosis | 6 years | ||||||||
Amortization charged to intangible assets | $ 132,000 | $ 60,000 | |||||||
Intangible asset impairment recognized | 39,600,000 | ||||||||
Goodwill impairment | $ 0 | ||||||||
IP license for QUINSAIR [Member] | |||||||||
Summary of intangible assets [Abstract] | |||||||||
Total intangible assets | $ 213,800,000 | ||||||||
Contingent payment | $ 350,000,000 | ||||||||
Encode Pharmaceuticals, Inc. [Member] | IP license for RP103 [Member] | |||||||||
Summary of intangible assets [Abstract] | |||||||||
Total intangible assets | $ 2,620,000 | 2,620,000 | $ 2,600,000 | ||||||
Useful Life | 20 years | ||||||||
Encode Pharmaceuticals, Inc. [Member] | UCSD license FDA and EC approval milestones [Member] | |||||||||
Summary of intangible assets [Abstract] | |||||||||
Total intangible assets | $ 1,250,000 | 1,250,000 | |||||||
Milestone payment based on drug approvals | $ 500,000 | $ 800,000 | |||||||
Useful Life | 14 years | ||||||||
Encode Pharmaceuticals, Inc. [Member] | Other Intangible Assets [Member] | |||||||||
Summary of intangible assets [Abstract] | |||||||||
Total intangible assets | $ 240,000 | 240,000 | |||||||
Useful Life | 16 years | ||||||||
Intangible assets, amortization method | straight-line method | ||||||||
Tripex Pharmaceuticals LLC [Member] | |||||||||
Summary of intangible assets [Abstract] | |||||||||
Contingent payment | $ 350,000,000 | ||||||||
Useful Life | [1] | 132 months | |||||||
Tripex Pharmaceuticals LLC [Member] | Developed Technology - QUINSAIR [Member] | |||||||||
Summary of intangible assets [Abstract] | |||||||||
Total intangible assets | $ 3,200,000 | $ 3,200,000 | |||||||
Useful Life | 11 years | ||||||||
[1] | Recognized on a straight-line basis. |
GOODWILL AND INTANGIBLE ASSET42
GOODWILL AND INTANGIBLE ASSETS - Summary of Intangible Assets Acquired (Details) - USD ($) $ in Thousands | Oct. 05, 2015 | Mar. 31, 2016 | Dec. 31, 2015 | Dec. 14, 2007 | |
Summary of intangible assets [Abstract] | |||||
Total intangible assets | $ 178,310 | $ 217,910 | |||
Less accumulated amortization | (1,579) | (1,447) | |||
Intangible Assets, Net | 176,731 | 216,463 | |||
Tripex Pharmaceuticals LLC [Member] | |||||
Summary of intangible assets [Abstract] | |||||
Useful Life | [1] | 132 months | |||
Tripex Pharmaceuticals LLC [Member] | IPR&D QUINSAIR [Member] | |||||
Summary of intangible assets [Abstract] | |||||
Indefinite intangible asset | $ 171,000 | 210,600 | |||
Useful Life | Indefinite | ||||
Tripex Pharmaceuticals LLC [Member] | Developed Technology - QUINSAIR [Member] | |||||
Summary of intangible assets [Abstract] | |||||
Total intangible assets | $ 3,200 | 3,200 | |||
Useful Life | 11 years | ||||
Encode Pharmaceuticals, Inc. [Member] | IP license for RP103 [Member] | |||||
Summary of intangible assets [Abstract] | |||||
Total intangible assets | $ 2,620 | 2,620 | $ 2,600 | ||
Useful Life | 20 years | ||||
Encode Pharmaceuticals, Inc. [Member] | UCSD license FDA and EC approval milestones [Member] | |||||
Summary of intangible assets [Abstract] | |||||
Total intangible assets | $ 1,250 | 1,250 | |||
Useful Life | 14 years | ||||
Encode Pharmaceuticals, Inc. [Member] | Other Intangible Assets [Member] | |||||
Summary of intangible assets [Abstract] | |||||
Total intangible assets | $ 240 | $ 240 | |||
Useful Life | 16 years | ||||
[1] | Recognized on a straight-line basis. |
GOODWILL AND INTANGIBLE ASSET43
GOODWILL AND INTANGIBLE ASSETS -Estimated Amortization Expense for Intangible Assets (Details) $ in Thousands | Mar. 31, 2016USD ($) |
Estimated Amortization Expense for Intangible Assets [Abstract] | |
2016 (remaining 9 months) | $ 397 |
2,017 | 529 |
2,018 | 529 |
2,019 | 529 |
2,020 | $ 529 |
ACCRUED LIABILITIES - Component
ACCRUED LIABILITIES - Components of Accrued Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2016 | Dec. 31, 2015 |
Accrued liabilities [Abstract] | ||
Personnel-related costs | $ 6,014 | $ 7,601 |
Rebates and other sales deductions | 2,738 | 2,833 |
Clinical trials and research and development costs | 3,053 | 2,076 |
License royalty payable | 2,491 | 1,951 |
Royalty-based interest payable | 1,511 | 1,410 |
Manufacturing costs | 2,115 | 1,577 |
Deferred rent | 1,082 | 1,086 |
Travel | 998 | 213 |
Business development & legal costs | 822 | 1,030 |
Other | 2,815 | 2,853 |
Total Accrued Liabilities | $ 23,639 | $ 22,630 |
ACCRUED LIABILITIES - Schedule
ACCRUED LIABILITIES - Schedule of Significant Estimated Accrued Rebates Reserve for Cash Discounts and Product Returns (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2016 | Dec. 31, 2015 | |
Accrued Rebates [Member] | ||
Accrued Liabilities [Line Items] | ||
Beginning Balance | $ 2,538 | $ 2,935 |
Provision for Current Period Sales | 2,566 | 6,250 |
Provision for Prior Period Sales | 239 | |
Actual Returns/ Credits Related to Current Period Sales | (18) | (3,712) |
Actual Returns/ Credits Related to Prior Period Sales | (2,644) | (3,174) |
Outstanding Balance | 2,442 | 2,538 |
Reserve for Cash Discounts [Member] | ||
Accrued Liabilities [Line Items] | ||
Beginning Balance | 258 | 215 |
Provision for Current Period Sales | 573 | 1,821 |
Provision for Prior Period Sales | 41 | |
Actual Returns/ Credits Related to Current Period Sales | (229) | (1,563) |
Actual Returns/ Credits Related to Prior Period Sales | (258) | (256) |
Outstanding Balance | 344 | 258 |
Product returns [Member] | ||
Accrued Liabilities [Line Items] | ||
Beginning Balance | 296 | 296 |
Outstanding Balance | $ 296 | $ 296 |
ACCRUED LIABILITIES (Additional
ACCRUED LIABILITIES (Additional Information) (Details) - USD ($) | 12 Months Ended | |
Mar. 31, 2016 | Dec. 31, 2015 | |
Payables And Accruals [Abstract] | ||
Accrual for estimated rebate payments | $ 2,400,000 | $ 2,500,000 |
Accrual for estimated cost of prompt payment | 300,000 | 300,000 |
Allowance for possible product returns | 300,000 | $ 300,000 |
Products returned | $ 0 |
NOTE PAYABLE - Additional Infor
NOTE PAYABLE - Additional Information (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||||
Jul. 31, 2014USD ($) | May. 31, 2013USD ($) | Dec. 31, 2012USD ($) | Mar. 31, 2016USD ($)Tranche | Mar. 31, 2015USD ($) | Jun. 30, 2014USD ($) | Dec. 31, 2015USD ($) | |
Debt Instrument [Line Items] | |||||||
Interest Expense | $ 5,018 | $ 4,498 | |||||
Loan Agreement with HC Royalty [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Total amount of loan | $ 10,000 | $ 50,000 | |||||
Number of tranches in loan | Tranche | 2 | ||||||
Amount of loan per tranche | $ 25,000 | ||||||
Annual fixed interest rate | 8.00% | ||||||
Original interest rate | 10.75% | ||||||
Revised annual interest rate | 8.00% | ||||||
Amount of first net revenues for calendar year | $ 50,000 | ||||||
Excess revenue | 2.00% | ||||||
First payment amount | $ 3,000 | ||||||
Maturity date | Mar. 31, 2020 | ||||||
Total payment required to terminate loan obligation immediately | $ 120,000 | ||||||
Interest Expense | $ 3,500 | 3,000 | |||||
Unamortized debt issuance costs | 1,400 | $ 1,500 | |||||
Amortization expense | $ 200 | $ 200 | |||||
Loan Agreement with HC Royalty [Member] | First Tranche [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Draw down on loan | $ 23,400 | ||||||
Royalty rate of first threshold of PROCYSBI and future approved product net revenues for calendar year | 6.25% | ||||||
Amount of first threshold of PROCYSBI and future approved product net revenues for calendar year | $ 25,000 | ||||||
Royalty rate of amount between first and second threshold of PROCYSBI and future approved product net revenues for calendar year | 3.00% | ||||||
Amount of second threshold of PROCYSBI and future approved product net revenues for calendar year | $ 50,000 | ||||||
Royalty rate for amount in excess of second threshold of PROCYSBI and future approved product net revenues for calendar year | 1.00% | ||||||
Loan Agreement with HC Royalty [Member] | Second Tranche [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Draw down on loan | $ 23,700 | ||||||
Royalty rate of first threshold of PROCYSBI and future approved product net revenues for calendar year | 6.00% | ||||||
Amount of first threshold of PROCYSBI and future approved product net revenues for calendar year | $ 25,000 | ||||||
Royalty rate of amount between first and second threshold of PROCYSBI and future approved product net revenues for calendar year | 3.00% | ||||||
Amount of second threshold of PROCYSBI and future approved product net revenues for calendar year | $ 50,000 | ||||||
Royalty rate for amount in excess of second threshold of PROCYSBI and future approved product net revenues for calendar year | 1.00% |
NOTE PAYABLE - Contractual Prin
NOTE PAYABLE - Contractual Principal Payments of the Note Payable (Details) $ in Thousands | Mar. 31, 2016USD ($) |
Long-term Debt, Fiscal Year Maturity [Abstract] | |
2016 (remaining 9 months) | $ 9,000 |
2,017 | 12,000 |
2,018 | 12,000 |
2,019 | 12,000 |
2,020 | 3,000 |
Total | $ 48,000 |
CONVERTIBLE NOTES - Additional
CONVERTIBLE NOTES - Additional Information (Details) - Convertible Debt [Member] | 1 Months Ended | 3 Months Ended | ||
Jul. 31, 2014USD ($)$ / shares | Mar. 31, 2016USD ($) | Mar. 31, 2015USD ($) | Dec. 31, 2015USD ($) | |
Debt Instrument [Line Items] | ||||
Aggregate principal amount | $ 60,000,000 | |||
Annual fixed interest rate | 8.00% | |||
Maturity Date | Aug. 1, 2019 | |||
Convertible senior notes conversion rate | 57.14 | |||
Debt instrument convertible base principal amount of conversion | $ 1,000 | |||
The price per share of the conversion feature embedded in the debt instrument (in dollars per share) | $ / shares | $ 17.50 | |||
Percentage of applicable conversion price | 175.00% | |||
Applicable conversion price consecutive period, days | 30 days | |||
Percentage of principal amount in repayment premium accrued unpaid interest | 100.00% | |||
Interest expense excluding amortization of debt issuance costs | $ 1,200,000 | $ 1,200,000 | ||
Unamortized debt issuance costs | 2,200,000 | $ 2,300,000 | ||
Amortization expense | $ 100,000 | $ 100,000 |
BUSINESS COMBINATION - Addition
BUSINESS COMBINATION - Additional Information (Details) - USD ($) $ in Thousands | Oct. 05, 2015 | Mar. 31, 2016 | Dec. 31, 2015 |
Business Acquisition [Line Items] | |||
Contingent Consideration Liability | $ 166,800 | $ 152,070 | $ 166,800 |
Goodwill | $ 12,223 | $ 12,223 | |
Tripex Pharmaceuticals LLC [Member] | |||
Business Acquisition [Line Items] | |||
Cash consideration | $ 35,370 | ||
Acquisition completed date | Oct. 5, 2015 | ||
Business acquisition, common stock issued (in shares) | 3,448,001 | ||
Business acquisition, contingent payment | $ 350,000 | ||
Goodwill | 8,948 | ||
Total purchase consideration | 223,030 | ||
Net assets acquired | 214,100 | ||
Tripex Pharmaceuticals LLC [Member] | Selling, General and Administrative Expenses [Member] | |||
Business Acquisition [Line Items] | |||
Direct costs acquisition included consulting, legal, and accounting fees | $ 3,900 |
BUSINESS COMBINATION - Schedule
BUSINESS COMBINATION - Schedule of Acquisition-Date Fair Value of Consideration Transferred (Details) - Tripex Pharmaceuticals LLC [Member] $ in Thousands | Oct. 05, 2015USD ($) |
Business Acquisition [Line Items] | |
Cash consideration | $ 35,370 |
Stock consideration | 20,860 |
Contingent consideration | 166,800 |
Total purchase consideration | $ 223,030 |
BUSINESS COMBINATION - Schedu52
BUSINESS COMBINATION - Schedule of Fair Value Estimate of Assets Acquired and Liability Assumed (Details) - USD ($) $ in Thousands | Mar. 31, 2016 | Dec. 31, 2015 | Oct. 05, 2015 |
Business Acquisition [Line Items] | |||
In-Process Research and Development | $ 171,000 | $ 210,600 | |
Goodwill | $ 12,223 | $ 12,223 | |
Tripex Pharmaceuticals LLC [Member] | |||
Business Acquisition [Line Items] | |||
Property and equipment | $ 282 | ||
Developed Technology | 3,200 | ||
In-Process Research and Development | 210,600 | ||
Goodwill | 8,948 | ||
Fair Value Estimate of Assets Acquired and Liability Assumed | $ 223,030 |
BUSINESS COMBINATION - Schedu53
BUSINESS COMBINATION - Schedule of Identifiable Intangible Assets Acquired Value and Amortization Period (Details) - USD ($) $ in Thousands | Oct. 05, 2015 | Mar. 31, 2016 | Dec. 31, 2015 | |
Business Acquisition [Line Items] | ||||
In-Process Research and Development | $ 171,000 | $ 210,600 | ||
Tripex Pharmaceuticals LLC [Member] | ||||
Business Acquisition [Line Items] | ||||
Developed Technology | $ 3,200 | |||
In-Process Research and Development | 210,600 | |||
Total identifiable intangible assets | $ 213,800 | |||
Developed technology, Amortization Period | [1] | 132 months | ||
[1] | Recognized on a straight-line basis. |
BUSINESS COMBINATION - Summary
BUSINESS COMBINATION - Summary of Change in Fair Values for IPR&D (Details) $ in Thousands | 3 Months Ended |
Mar. 31, 2016USD ($) | |
Business Combinations [Abstract] | |
Beginning balance | $ 210,600 |
Impairment loss recognized in earnings | (39,600) |
Ending balance | $ 171,000 |
BUSINESS COMBINATION - Summar55
BUSINESS COMBINATION - Summary of Change in Fair Values for Contingent Consideration Liability (Details) $ in Thousands | 3 Months Ended |
Mar. 31, 2016USD ($) | |
Business Combinations [Abstract] | |
Beginning balance | $ 166,800 |
Change in fair value recognized in earnings | (14,730) |
Ending balance | $ 152,070 |
CAPITAL STRUCTURE - Additional
CAPITAL STRUCTURE - Additional Information (Details) - USD ($) | Sep. 04, 2015 | Apr. 08, 2015 | Jul. 03, 2013 | Mar. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | Mar. 31, 2014 | Apr. 30, 2012 |
Class Of Stock [Line Items] | ||||||||
Common stock issued (in shares) | 85,289,311 | 85,235,591 | ||||||
At-the-Market Sales Agreement [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Common stock issued (in shares) | 0 | |||||||
Remaining common stock available for future sales under ATM sales (in shares) | 0 | |||||||
2015 Follow-on Public Offering [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Common stock issued (in shares) | 9,500,000 | |||||||
Stock price (in dollars per share) | $ 9 | |||||||
Number of shares issued to underwriters under over-allotment option (in shares) | 1,430,000 | |||||||
Gross proceeds from issuance of common stock | $ 98,300,000 | |||||||
Net proceeds from issuance of common stock | $ 92,000,000 | |||||||
Underwriting discount percentage (in hundredths) | 6.00% | |||||||
Shares Issued Under ATM Sales Agreement [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Maximum amount of common stock under ATM sales agreement | $ 75,000,000 | $ 100,000,000 | $ 40,000,000 | |||||
Sales commission percentage for sales made under ATM | 3.00% | 3.00% | ||||||
Common stock issued (in shares) | 0 | |||||||
Remaining value of common stock available for future issuance under ATM sales agreement | $ 75,000,000 |
STOCK-BASED COMPENSATION - Addi
STOCK-BASED COMPENSATION - Additional Information (Details) - USD ($) $ in Thousands | May. 19, 2015 | Nov. 25, 2014 | Mar. 31, 2016 | Mar. 31, 2015 | Dec. 31, 2015 |
Employee Stock Purchase Plan [Member] | |||||
Employee Stock Purchase Plan [Abstract] | |||||
Number of shares authorized for repurchase (in shares) | 1,000,000 | ||||
Stock issued under ESPP (in shares) | 162,498 | ||||
Stock reserved for future issuance under ESPP (in shares) | 837,502 | ||||
Restricted Stock Units [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Vested and Expected to Vest [Abstract] | |||||
Restricted stock units outstanding (in shares) | 820,005 | ||||
Restricted stock units granted (in shares) | 450,426 | 509,967 | |||
Restricted stock units vested/distributed (in shares) | 38,523 | 10,044 | |||
Restricted stock units forfeited (in shares) | 40,675 | 51,146 | |||
Restricted stock units, vesting year | 2,020 | ||||
2010 Equity Incentive Plan [Member] | Options to Purchase Common Stock [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Number of shares available for grant (in shares) | 2,553,867 | ||||
Proceeds from the exercise of stock options | $ 40 | $ 2,200 | |||
2010 Equity Incentive Plan [Member] | Maximum [Member] | Options to Purchase Common Stock [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Number of shares issued (in shares) | 2,400,000 | ||||
2015 Plan Amendment [Member] | Options to Purchase Common Stock [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Increase (decrease) in share reserve available for future issuance (in shares) | 3,456,620 | ||||
Number of shares available for grant (in shares) | 15,400,000 | ||||
2014 Employment Commencement Stock Incentive Plan [Member] | Options to Purchase Common Stock [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Increase (decrease) in share reserve available for future issuance (in shares) | 0 |
STOCK-BASED COMPENSATION - Comp
STOCK-BASED COMPENSATION - Components of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total Stock-Based Compensation Expense | $ 2,658 | $ 2,853 |
Cost of Goods Sold [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total Stock-Based Compensation Expense | 120 | 32 |
Research and Development Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total Stock-Based Compensation Expense | 590 | 584 |
Selling, General and Administrative Expenses [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total Stock-Based Compensation Expense | $ 1,948 | $ 2,237 |
STOCK-BASED COMPENSATION - Summ
STOCK-BASED COMPENSATION - Summary of Stock Option Plan Activity (Details) - Stock Options [Member] | 3 Months Ended |
Mar. 31, 2016$ / sharesshares | |
Option shares | |
Outstanding, beginning of period (in shares) | shares | 8,790,474 |
Granted (in shares) | shares | 1,436,631 |
Exercised (in shares) | shares | (15,773) |
Canceled (in shares) | shares | (637,701) |
Outstanding, end of period (in shares) | shares | 9,573,631 |
Weighted-average exercise price [Roll Forward] | |
Outstanding, beginning of period (in dollars per share) | $ / shares | $ 8.49 |
Granted (in dollars per share) | $ / shares | 3.83 |
Exercised (in dollars per share) | $ / shares | 2.57 |
Canceled (in dollars per share) | $ / shares | 10.15 |
Outstanding, end of period (in dollars per share) | $ / shares | $ 7.69 |
INCOME TAXES - Additional Infor
INCOME TAXES - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2016 | Mar. 31, 2015 | |
Income Tax Disclosure [Abstract] | ||
Provision for income taxes | $ 348 | $ 21 |
U.S. federal statutory tax rate | 34.00% |
COMMITMENTS AND CONTINGENCIES -
COMMITMENTS AND CONTINGENCIES - Additional Information (Details) - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended | |
Jan. 31, 2016 | Mar. 31, 2016 | Oct. 05, 2015 | |
Contractual Obligations [Line Items] | |||
Lease term | 7 years | ||
South San Francisco [Member] | |||
Contractual Obligations [Line Items] | |||
Lease term | 4 years | ||
Tripex Pharmaceuticals LLC [Member] | |||
Contractual Obligations [Line Items] | |||
Business acquisition, contingent payment | $ 350 | ||
UCSD [Member] | |||
Contractual Obligations [Line Items] | |||
Milestone payments based upon initiation of clinical trials | $ 2.2 | ||
European sales, marketing and administrative headquarter [Member] | |||
Contractual Obligations [Line Items] | |||
Lease term | 3 years |