Strongbridge Biopharma (SBBP) 10-Q 1 May 19 2019 Q1 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	3 Months Ended
	Mar. 31, 2019	Apr. 29, 2019
Document and Entity Information [Abstract]
Entity Registrant Name	Strongbridge Biopharma plc
Entity Central Index Key	0001634432
Document Type	10-Q
Current Fiscal Year End Date	--12-31
Document Period End Date	Mar. 31, 2019
Document Fiscal Year Focus	2019
Document Fiscal Period Focus	Q1
Amendment Flag	false
Entity Current Reporting Status	Yes
Entity Filer Category	Accelerated Filer
Entity Small Business	true
Entity Emerging Growth Company	true
Entity Ex Transition Period	true
Entity Common Stock, Shares Outstanding		54,172,061

Consolidated Balance Sheets

Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2019	Dec. 31, 2018
Current assets:
Cash and cash equivalents	$ 104,306	$ 122,490
Accounts receivable	3,916	1,626
Inventory	4,202	3,946
Prepaid expenses and other current assets	2,726	4,236
Total current assets	115,150	132,298
Property and equipment, net	292	294
Right to use assets, net	1,046
Intangible assets, net	28,876	30,132
Goodwill	7,256	7,256
Other assets	862	305
Total assets	153,482	170,285
Current liabilities:
Accounts payable	7,494	1,184
Accrued liabilities and other current liabilities	14,854	16,065
Total current liabilities	22,348	17,249
Warrant liability	17,333	15,513
Supply agreement liability, noncurrent	15,454	24,568
Other long-term liabilities	1,364
Total liabilities	56,499	57,330
Commitments and contingencies (Note 7)
Stockholders’ equity:
Deferred shares, $1.098 par value, 40,000 shares authorized, issued and outstanding at March 31, 2019 and December 31, 2018	44	44
Ordinary shares, $0.01 par value, 600,000,000 shares authorized at March 31, 2019 and December 31, 2018; 54,167,948 and 54,122,074 shares issued and outstanding at March 31, 2019 and December 31, 2018	542	541
Additional paid-in capital	325,863	323,402
Accumulated deficit	(229,466)	(211,032)
Total stockholders’ equity	96,983	112,955
Total liabilities and stockholders’ equity	$ 153,482	$ 170,285

Consolidated Balance Sheets (Pa

Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2019	Dec. 31, 2018
Consolidated Balance Sheets
Deferred shares, par value (in dollars per share)	$ 1.098	$ 1.098
Deferred shares, shares authorized	40,000	40,000
Deferred shares, shares issued	40,000	40,000
Deferred shares, shares outstanding	40,000	40,000
Ordinary shares, par value (in dollars per share)	$ 0.01	$ 0.01
Ordinary shares, shares authorized	600,000,000	600,000,000
Ordinary shares, shares issued	54,167,948	54,122,074
Ordinary shares, shares outstanding	54,167,948	54,122,074

Consolidated Statements of Oper

Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Revenues:
Revenues	$ 4,343	$ 3,870
Cost and expenses:
Cost of sales (excluding amortization of intangible assets)	813	667
Selling, general and administrative	12,100	12,362
Research and development	6,583	4,881
Amortization of intangible assets	1,256	1,769
Total cost and expenses	20,752	19,679
Operating loss	(16,409)	(15,809)
Other expense, net:
Income from field services agreement	2,016
Expense from field services agreement	(2,229)
Unrealized loss on fair value of warrants	(1,820)	(9,700)
Interest expense		(2,874)
Loss on extinguishment of debt		(500)
Other income, net	685	160
Total other expense, net	(1,348)	(12,914)
Loss before income taxes	(17,757)	(28,723)
Income tax expense	(677)
Net loss	(18,434)	(28,723)
Net loss attributable to ordinary shareholders:
Basic	(18,434)	(28,723)
Diluted	$ (18,434)	$ (28,723)
Net loss per share attributable to ordinary shareholders:
Basic and diluted (in dollars per share)	$ (0.34)	$ (0.66)
Weighted-average shares used in computing net loss per share attributable to ordinary shareholders:
Basic and diluted	54,155,034	43,620,746
Product
Revenues:
Revenues	$ 4,333	$ 3,870
Royalty
Revenues:
Revenues	$ 10

Consolidated Statement of Share

Consolidated Statement of Shareholders' (Deficit) Equity - USD ($) $ in Thousands	Ordinary SharesMaximum	Ordinary Shares	Deferred Shares	Additional Paid-In Capital	Accumulated Deficit	Total
Balance at beginning of period at Dec. 31, 2017		$ 401	$ 44	$ 230,524	$ (242,883)	$ (11,914)
Balance (in shares) at Dec. 31, 2017		40,149,812	40,000
Increase (Decrease) in Stockholders' Equity
Net loss					(28,723)	(28,723)
Stock-based compensation				1,688		1,688
Issuance of shares, net of costs		$ 53		33,455		33,508
Issuance of shares, net of costs (in shares)		5,255,683
Exercise of stock options				59		59
Exercise of stock options (in shares)		37,169
Issuance of warrants related to the loan agreements				7,663		7,663
Shares issued, net of shares withheld for employee taxes		$ 1		(429)		(428)
Shares issued, net of shares withheld for employee taxes (in shares)		89,163
Balance at end of period at Mar. 31, 2018		$ 455	$ 44	272,960	(271,606)	1,853
Balance (in shares) at Mar. 31, 2018		45,531,827	40,000
Balance at beginning of period at Dec. 31, 2018		$ 541	$ 44	323,402	(211,032)	112,955
Balance (in shares) at Dec. 31, 2018		54,122,074	40,000
Increase (Decrease) in Stockholders' Equity
Net loss					(18,434)	(18,434)
Stock-based compensation				2,323		2,323
Exercise of stock options	$ 1	$ 1		165		166
Exercise of stock options (in shares)		39,728
Shares issued, net of shares withheld for employee taxes				(27)		(27)
Shares issued, net of shares withheld for employee taxes (in shares)		6,146
Balance at end of period at Mar. 31, 2019		$ 542	$ 44	$ 325,863	$ (229,466)	$ 96,983
Balance (in shares) at Mar. 31, 2019		54,167,948	40,000

Consolidated Statements of Cash

Consolidated Statements of Cash Flow - USD ($) $ in Thousands	3 Months Ended		12 Months Ended
	Mar. 31, 2019	Mar. 31, 2018	Dec. 31, 2018
Cash flows from operating activities:
Net loss	$ (18,434)	$ (28,723)
Adjustments to reconcile net income (loss) to net cash used in operating activities:
Change in fair value of warrant liability	1,820	9,700
Stock-based compensation	2,323	1,688
Amortization of intangible assets	1,256	1,769	$ 7,187
Interest and related guarantee fees paid in kind		766
Amortization of debt discounts and debt issuance costs		314
Loss on extinguishment of debt		500
Depreciation	18	3
Changes in operating assets and liabilities:
Accounts receivable	(2,290)	(432)
Inventory	(649)	(1,150)
Prepaid expenses and other current assets	1,510	(567)
Other assets	(1,210)	325
Accounts payable	539	921
Accrued liabilities and other liabilities	(3,191)	(3,133)
Net cash used in operating activities	(18,308)	(18,019)
Cash flows from investing activities:
Payment for acquisitions		(24,655)
Purchases of property and equipment	(15)
Net cash provided by (used in) investing activities	(15)	(24,655)
Cash flows from financing activities:
Proceeds from long-term debt, net		44,930
Payment for loss on extinguishment of debt		(500)
Proceeds from issuance of ordinary shares, net		33,508
Proceeds from exercise of stock options	166	59
Payments related to tax withholding for net-share settled equity awards	(27)	(428)
Net cash provided by financing activities	139	77,569
Net (decrease) increase in cash and cash equivalents	(18,184)	34,895
Cash and cash equivalents—beginning of period	122,490	57,510	57,510
Cash and cash equivalents—end of period	$ 104,306	92,405	$ 122,490
Supplemental disclosures of cash flow information - Cash paid during the year for:
Interest		$ 1,642

Organization

Organization	3 Months Ended
	Mar. 31, 2019
Organization
Organization	1. Organization We are a global, commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Our first commercial product is Keveyis (dichlorphenamide), the first and only treatment approved by the U.S. Food and Drug Administration (the “FDA”) for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis (“PPP”), a group of rare hereditary disorders that cause episodes of muscle weakness or paralysis. In January 2018, Strongbridge Ireland Ltd., one of our wholly-owned subsidiaries, acquired the U.S. and Canadian rights to Macrilen (macimorelin), the first and only oral drug approved by the FDA for the diagnosis of patients with adult growth hormone deficiency. We launched Macrilen in the United States in July 2018. In December 2018, we sold Strongbridge Ireland Ltd. to Novo Nordisk Healthcare AG (“Novo”) for $145 million plus the right to receive tiered royalties on net sales of Macrilen through 2027. In addition, Strongbridge U.S. Inc, another of our wholly-owned subsidiaries, entered into an agreement with Novo Nordisk Inc., subsidiary of Novo (“NNI”), pursuant to which NNI will fund the costs of 23 of our field-based employees to provide full-time ongoing services to NNI, including the promotion of Macrilen in the United States, for a period of three years. W e have two clinical-stage product candidates for rare endocrine diseases, Recorlev and veldoreotide. Recorlev (levoketoconazole) is a cortisol synthesis inhibitor currently being studied for the treatment of endogenous Cushing's syndrome. Veldoreotide is a next-generation somatostatin analog being investigated for potential applications in conditions amenable to somatostatin receptor activation, such as acromegaly. Both Recorlev and veldoreotide have received orphan designation from the FDA and the European Medicines Agency (“EMA”). Liquidity We believe that our cash resources of $104.3 million at March 31, 2019 will be sufficient to allow us to fund planned operations for at least 12 months beyond the issuance date of these financial statements. We may never achieve profitability, and unless and until we do, we will continue to need to raise additional capital. We plan to continue to fund our operations and capital funding needs through equity or debt financing along with revenues from Keveyis and royalty revenues from Macrilen . There can be no assurances, however, that additional funding will be available on terms acceptable to us.

Summary of significant accounti

Summary of significant accounting policies and basis of presentation	3 Months Ended
	Mar. 31, 2019
Summary of significant accounting policies and basis of presentation
Summary of significant accounting policies and basis of presentation	2. Summary of significant accounting policies and basis of presentation Basis of presentation These unaudited consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States (“U.S. GAAP”). The unaudited consolidated financial statements reflect all adjustments, which include only normal recurring adjustments that are, in the opinion of management, necessary to present a fair statement of the operating results and financial position for the periods presented. The preparation of the unaudited consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect reported amounts and disclosures in the consolidated financial statements. Actual results could differ from those estimates. Results for the three months ended March 31, 2019 are not necessarily indicative of the results that may be expected for the year ending December 31, 2019. These unaudited consolidated financial statements should be read in conjunction with the accounting policies and notes to the audited consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2018 filed with the U.S. Securities and Exchange Commission on February 27, 2019 (the “2018 Annual Report”). Our significant accounting policies are described in Note 2 of the notes to the audited consolidated financial statements included in our 2018 Annual Report. Since the date of those financial statements, there have been no changes to our significant accounting policies. Royalty Revenues Royalty revenues are from commercial sales of Macrilen by Novo Nordisk Healthcare AG, based on net sales. Leases We account for leases in accordance with Accounting Standards Codification Topic 842, Leases , (“ASC 842”). We determine if an arrangement is a lease at contract inception. A lease exists when a contract conveys to us the right to control the use of identified property, plant, or equipment for a period of time in exchange for consideration. The definition of a lease embodies two conditions: (1) there is an identified asset in the contract that is land or a depreciable asset (i.e., property, plant, and equipment), and (2) we have the right to control the use of the identified asset. Operating leases where we are the lessee are included in Right of use (“ROU”) assets and Other current liabilities and Other long-term liabilities on our Consolidated Balance Sheets. The lease liabilities are initially and subsequently measured at the present value of the unpaid lease payments at the lease commencement date. Key estimates and judgments include how we determined (1) the discount rate we use to discount the unpaid lease payments to present value, (2) lease term and (3) lease payments. ASC 842 requires a lessee to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. Our leases do not provide an implicit rate, we use our incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. Our incremental borrowing rate for a lease is the rate of interest we would have to pay on a collateralized basis to borrow an amount equal to the lease payments under similar terms. The lease term for all of our leases includes the noncancellable period of the lease. Lease payments included in the measurement of the lease asset or liability comprise of our fixed payments The ROU asset is initially measured at cost, which comprises the initial amount of the lease liability adjusted for lease payments made at or before the lease commencement date less any lease incentives received. For operating leases, the ROU asset is subsequently measured throughout the lease term at the carrying amount of the lease liability, plus (minus) any prepaid (accrued) lease payments, less the unamortized balance of lease incentives received. Lease expense for lease payments is recognized on a straight-line basis over the lease term. We monitor for events or changes in circumstances that require a reassessment of a lease. If a reassessment results in the remeasurement of a lease liability, a corresponding adjustment is made to the carrying amount of the corresponding ROU asset unless doing so would reduce the carrying amount of the ROU asset to an amount less than zero. In that case, the amount of the adjustment that would result in a negative ROU asset balance is recorded in profit or loss. We have elected not to recognize ROU assets and lease liabilities for all short-term leases that have a lease term of 12 months or less. We recognize the lease payments associated with our short-term leases as an expense on a straight-line basis over the lease term. Variable lease payments associated with these leases are recognized and presented in the same manner as for all our other leases. We adopted ASC 842 using a modified retrospective transition approach as of the effective date, as permitted by the amendments in ASU 2018-11, which provides an alternative modified retrospective transition method. As a result, we were not required to adjust our comparative period financial information for effects of the standard or make the new required lease disclosures for periods before the date of adoption ( i.e., January 1, 2019). We have elected to adopt the package of transition practical expedients and, therefore, have not reassessed (1) whether existing or expired contracts contain a lease, (2) lease classification for existing or expired leases or (3) the accounting for initial direct costs that were previously capitalized. We did not elect the practical expedient to use hindsight for leases existing at the adoption date. Further, we do not expect the amendments in ASU 2018-01: Land Easement Practical Expedient to have an effect on us because we do not enter into land easement arrangements. Income and Expense from Field Services Agreement For our field services agreement with NNI, our income and expense are being recorded as non-operating income and expense, respectively. Segment information Operating segments are identified as components of an enterprise for which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision making group, in making decisions on how to allocate resources and assess performance. We view our operations and manage our business in one operating segment. Net loss per share Basic net loss per share is calculated by dividing the net loss attributable to shareholders by the weighted average number of ordinary shares outstanding during the period. Diluted net loss per share is calculated by dividing the net loss attributable to shareholders by the weighted‑average number of ordinary shares outstanding for the period, including any dilutive effect from outstanding stock options or other equity-based awards. Shares used in the diluted net loss per share calculations exclude anti‑dilutive ordinary share equivalents, which currently consist of outstanding stock options, unvested restricted stock units (“RSUs”) and warrants. The following potentially dilutive securities have been excluded from the computations of diluted weighted average shares outstanding as of March 31, 2019 and 2018, as they would be anti-dilutive: Three Months Ended March 31, 2019 2018 Warrants 6,833,253 8,803,253 Stock options issued and outstanding 10,205,851 7,960,469 Unvested RSUs 758,850 173,400 Recent accounting pronouncements – not yet adopted In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other : Simplifying the Accounting for Goodwill Impairment . ASU 2017-04 removes Step 2 of the goodwill impairment test, which requires a hypothetical purchase price allocation. A goodwill impairment will now be the amount by which a reporting unit’s carrying value exceeds its fair value, not to exceed the carrying amount of goodwill. This standard, which will be effective for us beginning in the first quarter of fiscal year 2020, is required to be applied prospectively. Early adoption is permitted for interim or annual goodwill impairment tests performed on testing dates after January 1, 2017. We are currently evaluating the impact this new accounting guidance will have on our consolidated financial statements.

Revenue recognition

Revenue recognition	3 Months Ended
	Mar. 31, 2019
Revenue recognition
Revenue recognition	3. Revenue recognition Product sales, net We sell Keveyis to one specialty pharmacy provider (the “Customer”), who is the exclusive distributor of Keveyis in the United States. The Customer subsequently resells Keveyis to patients, most of whom are covered by payors that may provide for government-mandated or privately negotiated rebates with respect to the purchase of Keveyis. Revenues from sales of Keveyis are recognized when we satisfy a performance obligation by transferring control of the product to our customers. Transfer of control occurs upon receipt of the product by the customer. We expense incremental costs related to the set-up of contracts with our customers when incurred, as these costs do not meet the criteria for capitalization. Reserves for variable consideration Revenues from sales of Keveyis are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and that result from rebates, co-pay assistance and other allowances that are offered between us and the patients’ payors. There is no variable consideration reserve for returns as we do not accept returns of Keveyis. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if the amount is payable to the Customer) or a current liability (if the amount is payable to a party other than the Customer). Where appropriate, these estimates may take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained, and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. We reassess our estimates on an ongoing basis. If actual results in the future vary from our estimates, we will adjust our estimates. Any such adjustments would affect net product revenue and earnings in the period such variances become known. Trade Discount : We provide the Customer with a discount that is explicitly stated in our contract and is recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive sales order management, data and distribution services from the Customer. To the extent, the services received are distinct from our sale of Keveyis to the Customer, these payments are classified in selling, general and administrative expenses in our consolidated statement of operations and comprehensive loss. Funded Co-pay Assistance Program : We contract with a third-party to manage the co-pay assistance program intended to provide financial assistance to qualified insured patients. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with Keveyis that has been recognized as revenue, but remains in the distribution channel inventories at the end of each reporting period. These payments are consideration payable to the Customer and the related reserve is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses on the consolidated balance sheet. Government Rebates : We are subject to discount obligations under state Medicaid programs and Medicare. We estimate our Medicaid and Medicare rebates based upon a range of possible outcomes that are probability-weighted for the estimated patient mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability, which is included in accrued expenses on the consolidated balance sheet. For Medicaid, accruals are based on estimates of future Medicaid beneficiary utilization applied to the Medicaid unit rebate formula established by the Center for Medicaid and Medicare Services. Effective January 1, 2011, manufacturers of pharmaceutical products are responsible for 50% of the patient’s cost of branded prescription drugs related to the Medicare Part D Coverage Gap. In order to estimate the cost to us of this Medicare coverage gap responsibility, we estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program. Our liability for these rebates consists of estimates of claims for the current quarter and estimated future claims that will be made for Keveyis that have been recognized as revenue, but remains in the distribution channel inventories at the end of each reporting period. Temporary Supply and Patient Assistance Programs : We provide free Keveyis to uninsured patients who satisfy pre-established criteria for either the Temporary Supply Program or the Patient Assistance Program. Patients who meet the Temporary Supply Program eligibility criteria may receive a temporary supply of free Keveyis for no more than sixty days while there is a determination of the patient’s third-party insurance, prescription drug benefit or other third-party coverage for Keveyis. The Patient Assistance Program provides free Keveyis for up to twelve months to uninsured patients who satisfy pre-established criteria for financial need. We do not recognize any revenue related to these free products and the associated costs are classified in selling, general and administrative expenses in our consolidated statements of operations and comprehensive loss. Royalty Revenues Royalty revenues are from commercial sales of Macrilen by Novo Nordisk Healthcare AG, based on net sales.

Fair value measurement

Fair value measurement	3 Months Ended
	Mar. 31, 2019
Fair value measurement
Fair value measurement	4. Fair value measurement We follow FASB accounting guidance on fair value measurements for financial assets and liabilities measured on a recurring basis. Because of their short-term nature, the amounts reported in the balance sheet for cash and accounts payable approximate fair value. The guidance requires fair value measurements to maximize the use of “observable inputs.” The three-level hierarchy of inputs to measure fair value are as follows: Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities. Because of their short-term nature, the amounts reported in the balance sheet for cash and accounts payable approximate fair value. Level 2: Significant observable inputs other than Level 1 prices such as quoted prices in markets that are not active, or inputs that are observable, either directly or indirectly, for substantially the full term of the asset or liability Level 3: Prices or valuation techniques that require inputs that are both significant to the fair value measurement and unobservable (i.e., supported by little or no market activity). The fair values of the outstanding warrants were measured using the Black-Scholes option-pricing model. Inputs used to determine estimated fair value of the warrant liabilities include the estimated fair value of the underlying stock at the valuation date, the estimated term of the warrants, risk-free interest rates, expected dividends and the expected volatility of the underlying stock. The significant unobservable inputs used in the fair value measurement of the warrant liabilities were the volatility rate and the estimated term of the warrants. Generally, increases (decreases) in the fair value of the underlying stock and estimated term would result in a directionally similar impact to the fair value measurement. We did not have any transfers between the different levels. The following table presents our assets and liabilities that are measured at fair value on a recurring basis for the periods presented (in thousands) : As of March 31, 2019 Level I Level II Level III Total Cash equivalents 103,939 — — 103,939 Total assets $ 103,939 $ — $ — $ 103,939 Warrant liability — — 17,333 17,333 Total liabilities $ — $ — $ 17,333 $ 17,333 As of December 31, 2018 Level I Level II Level III Total Cash equivalents 122,300 — — 122,300 Total assets $ 122,300 $ — $ — $ 122,300 Warrant liability — — 15,513 15,513 Total liabilities $ — $ — $ 15,513 $ 15,513 The following table presents a reconciliation of our level 3 Warrant liability (in thousands): March 31, 2019 Balance as of 12/31/2018 $ 15,513 Unrealized loss on fair value of warrants for the three months ended March 31, 2019 1,820 Balance as of 3/31/2019 $ 17,333

Intangible assets and goodwill

Intangible assets and goodwill	3 Months Ended
	Mar. 31, 2019
Intangible assets and goodwill
Intangible assets and goodwill	5. Intangible assets and goodwill The following represents the balance of our intangible assets as follows (in thousands): As of March 31, 2019 Beginning of Period Additions Sold Amortization End of Period Keveyis $ 30,132 $ — $ — $ (1,256) $ 28,876 Goodwill 7,256 — — — 7,256 Total $ 37,388 $ — $ — $ (1,256) $ 36,132 As of December 31, 2018 Beginning of Period Additions Sold Amortization End of Period Keveyis $ 35,155 $ — $ — $ (5,023) $ 30,132 Macrilen — 24,834 (22,670) (2,164) — Goodwill 7,256 — — — 7,256 Total $ 42,411 $ 24,834 $ (22,670) $ (7,187) $ 37,388 Our finite lived intangible assets consist of acquired developed product rights obtained from our acquisition of Keveyis (dichlorphenamide) from a subsidiary of Taro Pharmaceutical Industries Ltd. (“Taro”) . Pursuant to the terms of the Asset Purchase Agreement and Supply Agreement that we entered into with Taro, we paid Taro an upfront payment in two installments of $1 million in December 2016 and $7.5 million in March 2017. We concluded that the supply price payable by us exceeds fair value and, therefore, used a discounted cash flow method with a probability assumption to value the payments in excess of fair value at $29.3 million, for which we have recorded an intangible asset and corresponding liability. This liability will be reduced as we purchase inventory over the term of the Supply Agreement. In addition, we incurred transaction costs of $2.4 million. The overall recording of the transaction resulted in the recording of an intangible asset of $40.2 million. This asset is being amortized over an eight-year period using the straight-line method. We recorded amortization expense of $1.3 million and $1.8 for the three months ended March 31, 2019 and 2018, respectively

Accrued liabilities and other c

Accrued liabilities and other current liabilities	3 Months Ended
	Mar. 31, 2019
Accrued liabilities and other current liabilities
Accrued liabilities and other current liabilities	6. Accrued liabilities and other current liabilities Accrued liabilities and other current liabilities consist of the following (in thousands): March 31, December 31, 2019 2018 Supply agreement - current portion $ 5,375 $ 1,638 Consulting and professional fees 2,659 4,145 Employee compensation 1,711 5,717 Accrued sales allowances 1,815 2,233 Accrued royalties 1,367 802 Accrued Taxes 1,193 535 Lease liability - current portion 326 — Other 408 995 Total accrued liabilities $ 14,854 $ 16,065

Commitments and contingencies

Commitments and contingencies	3 Months Ended
	Mar. 31, 2019
Commitments and contingencies
Commitments and contingencies	7. Commitments and contingencies (a) Commitments to Taro Pharmaceuticals Industries Ltd. In December 2016, we acquired the U.S. marketing rights to Keveyis (dichlorphenamide) from Taro. Under the terms of an asset purchase agreement, we paid Taro an upfront payment in two installments of $1 million in December 2016 and $7.5 million in March 2017, and will pay an aggregate of $7.5 million in potential milestones upon the achievement of certain product sales targets. Taro has agreed to continue to manufacture Keveyis for us under an exclusive supply agreement through the orphan exclusivity period. We are obligated to purchase certain annual minimum amounts of product totaling approximately $29 million over a six-year period. As of March 31, 2019, our remaining obligation was $22.1 million. The supply agreement may extend beyond the orphan exclusivity period unless terminated by either party pursuant to the terms of the agreement. If terminated by Taro at the conclusion of the orphan exclusivity period, we have the right to manufacture the product on our own or have the product manufactured by a third party on our behalf. We are required to reimburse Taro for their royalty obligation resulting from their sale of Keveyis to us. (b) Indemnifications In the ordinary course of business and in connection with the sale of assets and businesses and other transactions, we often indemnify our counterparties against certain liabilities that may arise in connection with the transaction or that are related to events and activities prior to or following a transaction, such as breaches of contracts, unfavorable tax consequences and employee liabilities. If the indemnified party were to make a successful claim pursuant to the terms of the indemnification, we may be required to reimburse the loss and such amount could be material to our financial statements. Where appropriate, the obligation for such indemnifications is recorded as a liability. Because the amount of these types of indemnifications generally is not specifically stated, the overall maximum amount of the obligation under such indemnifications cannot be reasonably estimated. However, we believe that the likelihood of a material liability being triggered under these indemnification obligations is not probable at this time.

Leases

Leases	3 Months Ended
	Mar. 31, 2019
Leases
Leases	8. Leases We lease office space under operating leases. Our leases have initial lease terms ranging from one to five years. Our lease agreements contain provisions for future rent increases. As of March 31, 2019, future minimum commitments under facility operating leases were as follows (in thousands): Operating leases 2019 326 2020 470 2021 481 2022 492 2023 207 Total minimum lease payments $ 1,976 The components of lease cost for the quarter ended March 31, 2019 are as follows (in thousands): Three Months Ended March 31, 2019 Lease costs Amortization of right of use assets $ 110 Interest on lease liabilities 33 Total lease cost $ 143 Amounts reported in the Consolidated Balance Sheets for leases where we are the lessee as of the quarter ended March 31, 2019 were as follows (in thousands): March 31, 2019 Operating Leases Right of use asset $ 1,046 Lease liability $ 1,690 Remaining lease term Operating leases 4 years Discount rate Operating leases

Income taxes

Income taxes	3 Months Ended
	Mar. 31, 2019
Income taxes
Income taxes	9. Income taxes Deferred tax assets and liabilities are recognized for the future tax consequences of differences between the carrying amounts and tax bases of assets and liabilities and operating loss carryforwards and other attributes using enacted rates expected to be in effect when those differences reverse. Valuation allowances are provided against deferred tax assets that are not more likely than not to be realized. We assess our ability to realize deferred tax assets. Changes in future earnings projections, among other factors, may cause us to adjust our valuation allowance on deferred tax assets. Any such adjustments would impact our income tax expense in the period in which it is determined that these factors have changed. We recorded income tax expense of $0.7 million for the three months ended March 31, 2019, as a result of tax liability expected in connection with the intercompany transfer of intellectual property. The global intangible low-taxed income tax and base erosion provisions are effective for taxable years beginning after December 31, 2017. We do not currently expect these provisions to have a material impact on our tax rate as we do not own any controlled foreign corporations and they are currently below the gross receipts threshold for purposes of the base erosion provisions.

Warrants

Warrants	3 Months Ended
	Mar. 31, 2019
Warrants and Rights Note Disclosure [Abstract]
Warrants	10. Warrants Warrants Our outstanding warrants as of March 31, 2019 are as follows: Warrants Outstanding Exercise Expiration Warrants Warrants March 31, Classification Price Date Issued Exercised 2019 Warrants in connection with private equity placement Liability $ 2.50 6/28/2022 7,000,000 (1,970,000) 5,030,000 Warrants in connection with Horizon and Oxford loan agreement Equity $ 2.45 12/28/2026 428,571 (267,857) 160,714 Warrants in connection with CRG loan agreement Equity $ 7.37 7/14/2024 394,289 — 394,289 Warrants in connection with CRG loan amendment in January 2018 Equity $ 10.00 1/16/2025 1,248,250 — 1,248,250 9,071,110 6,833,253

Stock-based compensation

Stock-based compensation	3 Months Ended
	Mar. 31, 2019
Stock-based compensation
Stock based compensation	11. Stock‑based compensation Our board of directors has adopted the 2017 Inducement Plan (the “Inducement Plan”). The Inducement Plan provides for the grant of equity-based awards to new employees. The purpose of the Inducement Plan is to attract valued employees by offering them a greater stake in our success and a closer identity with us, and to encourage ownership of our ordinary shares by such employees. The Inducement Plan became effective on February 23, 2017. As of March 31, 2019, 738,953 shares are available for issuance pursuant to the Inducement Plan. Our board of directors has adopted, and our shareholders have approved, the 2015 Equity Compensation Plan (the “2015 Plan”). The 2015 Plan provides for the grant of incentive stock options to our employees and any parent or subsidiary corporation’s employees, and for the grant of nonstatutory stock options, stock awards, and RSUs to our employees, directors and consultants and our parent or subsidiary corporations’ employees and consultants. The 2015 Plan became effective on September 3, 2015. As of March 31, 2019, 262,168 shares are available for issuance pursuant to the 2015 Plan. Our board of directors has adopted, and our shareholders have approved, the Non‑Employee Director Equity Compensation Plan (the “Non‑Employee Director Plan”). The Non‑Employee Director Plan provides for the grant of nonstatutory stock options, stock awards, and RSUs to our non‑employee directors. The Non‑Employee Director Plan became effective on September 3, 2015. As of March 31, 2019, 272,151 shares are available for issuance pursuant to the Non‑Employee Director Plan. A summary of our outstanding stock options as of March 31, 2019 is as follows: Options Outstanding Weighted- Average Weighted- Remaining Average Contractual Number of Exercise Term Aggregate Shares Price (Years) Intrinsic Value (in thousands) Outstanding—January 1, 2019 8,579,511 $ 7.35 7.57 $ 3,281 Granted 1,767,400 $ 4.70 Forfeited and cancelled (101,332) $ 6.31 Exercised (39,728) $ 4.19 Outstanding—March 31, 2019 10,205,851 $ 6.91 7.77 $ 5,385 Vested and exercisable—March 31, 2019 4,224,745 $ 8.63 6.25 $ 2,506 Included in the stock options outstanding at March 31, 2019 are unvested stock options to purchase 88,908 shares at a weighted average exercise price of $18.80 per share for which the vesting of certain tranches will accelerate if the fair value per share of our stock reaches $31.46. In addition, the options outstanding as of March 31, 2019 include 97,652 shares that vest upon a market appreciation event, so long as it occurs prior to the date specified in the applicable award agreement and 97,652 shares that will vest upon the one year anniversary of the market appreciation event. The market appreciation event, which had not yet occurred as of March 31, 2019, is defined as the last trading day in the period in which our closing stock price on each of 20 consecutive trading days reported on Nasdaq has been at least $30.14 or $33.66 for the respective grantee. Stock‑based compensation expense We recognized stock‑based compensation expense for employees and directors for stock options and RSUs as follows (in thousands): Three Months Ended March 31, 2019 2018 Selling, general and administrative $ 1,811 $ 1,280 Research and development 512 408 Total stock-based compensation $ 2,323 $ 1,688 As of March 31, 2019, the total unrecognized compensation expense related to unvested stock options, net of estimated forfeitures, is $19.0 million, which we expect to recognize over an estimated weighted‑average period of 2.95 years. In determining the estimated fair value of our service-based awards, we use the Black‑Scholes option‑pricing model and assumptions discussed below. Each of these inputs is subjective and generally requires significant judgment. The fair value of our service-based awards that were granted during the years was estimated with the following assumptions: Three Months Ended March 31, 2019 2018 Expected term (in years) 6.09 6.08 Risk-free interest rate 2.47% - 2.61% 2.25% - 2.71% Expected volatility 80.00% - 80.85% 85.00% Dividend rate —% —% Restricted stock units Our board of directors have approved grants of RSUs to employees. These RSUs vest two years from the date of issuance, provided that the employee is employed by us on such vesting date. All RSUs will fully vest upon a change of control of our company. If and when the RSUs vest, we will issue one ordinary share for each whole RSU that has vested, subject to satisfaction of the executive’s tax withholding obligations. The RSUs will cease to be outstanding upon such issuance of ordinary shares. We recorded expense, which is included in the stock-based compensation table above, of $239,000 and $149,000 for the three months ended March 31, 2019 and 2018, respectively. As of March 31, 2019, the total unrecognized compensation expense related to unvested RSUs is $3.1 million, which we expect to recognize over an estimated weighted‑average period of 1.8 years. A summary of our unvested RSUs as of March 31, 2019 is as follows: Number of Shares Outstanding—January 1, 2019 143,100 Granted 634,000 Forfeited (6,250) Vested (12,000) Unvested—March 31, 2019 758,850

Summary of significant accoun_2

Summary of significant accounting policies and basis of presentation (Policies)	3 Months Ended
	Mar. 31, 2019
Summary of significant accounting policies and basis of presentation
Basis of presentation	Basis of presentation These unaudited consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States (“U.S. GAAP”). The unaudited consolidated financial statements reflect all adjustments, which include only normal recurring adjustments that are, in the opinion of management, necessary to present a fair statement of the operating results and financial position for the periods presented. The preparation of the unaudited consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect reported amounts and disclosures in the consolidated financial statements. Actual results could differ from those estimates. Results for the three months ended March 31, 2019 are not necessarily indicative of the results that may be expected for the year ending December 31, 2019. These unaudited consolidated financial statements should be read in conjunction with the accounting policies and notes to the audited consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2018 filed with the U.S. Securities and Exchange Commission on February 27, 2019 (the “2018 Annual Report”). Our significant accounting policies are described in Note 2 of the notes to the audited consolidated financial statements included in our 2018 Annual Report. Since the date of those financial statements, there have been no changes to our significant accounting policies.
Revenues	Royalty Revenues Royalty revenues are from commercial sales of Macrilen by Novo Nordisk Healthcare AG, based on net sales.
Leases	Leases We account for leases in accordance with Accounting Standards Codification Topic 842, Leases , (“ASC 842”). We determine if an arrangement is a lease at contract inception. A lease exists when a contract conveys to us the right to control the use of identified property, plant, or equipment for a period of time in exchange for consideration. The definition of a lease embodies two conditions: (1) there is an identified asset in the contract that is land or a depreciable asset (i.e., property, plant, and equipment), and (2) we have the right to control the use of the identified asset. Operating leases where we are the lessee are included in Right of use (“ROU”) assets and Other current liabilities and Other long-term liabilities on our Consolidated Balance Sheets. The lease liabilities are initially and subsequently measured at the present value of the unpaid lease payments at the lease commencement date. Key estimates and judgments include how we determined (1) the discount rate we use to discount the unpaid lease payments to present value, (2) lease term and (3) lease payments. ASC 842 requires a lessee to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. Our leases do not provide an implicit rate, we use our incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. Our incremental borrowing rate for a lease is the rate of interest we would have to pay on a collateralized basis to borrow an amount equal to the lease payments under similar terms. The lease term for all of our leases includes the noncancellable period of the lease. Lease payments included in the measurement of the lease asset or liability comprise of our fixed payments The ROU asset is initially measured at cost, which comprises the initial amount of the lease liability adjusted for lease payments made at or before the lease commencement date less any lease incentives received. For operating leases, the ROU asset is subsequently measured throughout the lease term at the carrying amount of the lease liability, plus (minus) any prepaid (accrued) lease payments, less the unamortized balance of lease incentives received. Lease expense for lease payments is recognized on a straight-line basis over the lease term. We monitor for events or changes in circumstances that require a reassessment of a lease. If a reassessment results in the remeasurement of a lease liability, a corresponding adjustment is made to the carrying amount of the corresponding ROU asset unless doing so would reduce the carrying amount of the ROU asset to an amount less than zero. In that case, the amount of the adjustment that would result in a negative ROU asset balance is recorded in profit or loss. We have elected not to recognize ROU assets and lease liabilities for all short-term leases that have a lease term of 12 months or less. We recognize the lease payments associated with our short-term leases as an expense on a straight-line basis over the lease term. Variable lease payments associated with these leases are recognized and presented in the same manner as for all our other leases. We adopted ASC 842 using a modified retrospective transition approach as of the effective date, as permitted by the amendments in ASU 2018-11, which provides an alternative modified retrospective transition method. As a result, we were not required to adjust our comparative period financial information for effects of the standard or make the new required lease disclosures for periods before the date of adoption ( i.e., January 1, 2019). We have elected to adopt the package of transition practical expedients and, therefore, have not reassessed (1) whether existing or expired contracts contain a lease, (2) lease classification for existing or expired leases or (3) the accounting for initial direct costs that were previously capitalized. We did not elect the practical expedient to use hindsight for leases existing at the adoption date. Further, we do not expect the amendments in ASU 2018-01: Land Easement Practical Expedient to have an effect on us because we do not enter into land easement arrangements.
Income and expense from Field Services Agreement	Income and Expense from Field Services Agreement For our field services agreement with NNI, our income and expense are being recorded as non-operating income and expense, respectively.
Segment information	Segment information Operating segments are identified as components of an enterprise for which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision making group, in making decisions on how to allocate resources and assess performance. We view our operations and manage our business in one operating segment.
Net loss per share	Net loss per share Basic net loss per share is calculated by dividing the net loss attributable to shareholders by the weighted average number of ordinary shares outstanding during the period. Diluted net loss per share is calculated by dividing the net loss attributable to shareholders by the weighted‑average number of ordinary shares outstanding for the period, including any dilutive effect from outstanding stock options or other equity-based awards. Shares used in the diluted net loss per share calculations exclude anti‑dilutive ordinary share equivalents, which currently consist of outstanding stock options, unvested restricted stock units (“RSUs”) and warrants. The following potentially dilutive securities have been excluded from the computations of diluted weighted average shares outstanding as of March 31, 2019 and 2018, as they would be anti-dilutive: Three Months Ended March 31, 2019 2018 Warrants 6,833,253 8,803,253 Stock options issued and outstanding 10,205,851 7,960,469 Unvested RSUs 758,850 173,400
Recent accounting pronouncements - not yet adopted	Recent accounting pronouncements – not yet adopted In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other : Simplifying the Accounting for Goodwill Impairment . ASU 2017-04 removes Step 2 of the goodwill impairment test, which requires a hypothetical purchase price allocation. A goodwill impairment will now be the amount by which a reporting unit’s carrying value exceeds its fair value, not to exceed the carrying amount of goodwill. This standard, which will be effective for us beginning in the first quarter of fiscal year 2020, is required to be applied prospectively. Early adoption is permitted for interim or annual goodwill impairment tests performed on testing dates after January 1, 2017. We are currently evaluating the impact this new accounting guidance will have on our consolidated financial statements.

Summary of significant accoun_3

Summary of significant accounting policies and basis of presentation (Tables)	3 Months Ended
	Mar. 31, 2019
Summary of significant accounting policies and basis of presentation
Schedule of potentially dilutive securities excluded from computations of diluted weighted average shares outstanding	Three Months Ended March 31, 2019 2018 Warrants 6,833,253 8,803,253 Stock options issued and outstanding 10,205,851 7,960,469 Unvested RSUs 758,850 173,400

Fair value measurement (Tables)

Fair value measurement (Tables)	3 Months Ended
	Mar. 31, 2019
Fair value measurement
Schedule of fair value of financial assets by level	The following table presents our assets and liabilities that are measured at fair value on a recurring basis for the periods presented (in thousands) : As of March 31, 2019 Level I Level II Level III Total Cash equivalents 103,939 — — 103,939 Total assets $ 103,939 $ — $ — $ 103,939 Warrant liability — — 17,333 17,333 Total liabilities $ — $ — $ 17,333 $ 17,333 As of December 31, 2018 Level I Level II Level III Total Cash equivalents 122,300 — — 122,300 Total assets $ 122,300 $ — $ — $ 122,300 Warrant liability — — 15,513 15,513 Total liabilities $ — $ — $ 15,513 $ 15,513
Schedule of reconciliation of level 3 Warrant liability	The following table presents a reconciliation of our level 3 Warrant liability (in thousands): March 31, 2019 Balance as of 12/31/2018 $ 15,513 Unrealized loss on fair value of warrants for the three months ended March 31, 2019 1,820 Balance as of 3/31/2019 $ 17,333

Intangible assets and goodwill

Intangible assets and goodwill (Tables)	3 Months Ended
	Mar. 31, 2019
Intangible assets and goodwill
Schedule of gross carrying amount of in process research and development and goodwill	The following represents the balance of our intangible assets as follows (in thousands): As of March 31, 2019 Beginning of Period Additions Sold Amortization End of Period Keveyis $ 30,132 $ — $ — $ (1,256) $ 28,876 Goodwill 7,256 — — — 7,256 Total $ 37,388 $ — $ — $ (1,256) $ 36,132 As of December 31, 2018 Beginning of Period Additions Sold Amortization End of Period Keveyis $ 35,155 $ — $ — $ (5,023) $ 30,132 Macrilen — 24,834 (22,670) (2,164) — Goodwill 7,256 — — — 7,256 Total $ 42,411 $ 24,834 $ (22,670) $ (7,187) $ 37,388

Accrued liabilities and other_2

Accrued liabilities and other current liabilities (Tables)	3 Months Ended
	Mar. 31, 2019
Accrued liabilities and other current liabilities
Schedule of accrued liabilities and other current liabilities	Accrued liabilities and other current liabilities consist of the following (in thousands): March 31, December 31, 2019 2018 Supply agreement - current portion $ 5,375 $ 1,638 Consulting and professional fees 2,659 4,145 Employee compensation 1,711 5,717 Accrued sales allowances 1,815 2,233 Accrued royalties 1,367 802 Accrued Taxes 1,193 535 Lease liability - current portion 326 — Other 408 995 Total accrued liabilities $ 14,854 $ 16,065

Leases (Tables)

Leases (Tables)	3 Months Ended
	Mar. 31, 2019
Leases
Schedule of future minimum commitments under facility operating leases	As of March 31, 2019, future minimum commitments under facility operating leases were as follows (in thousands): Operating leases 2019 326 2020 470 2021 481 2022 492 2023 207 Total minimum lease payments $ 1,976
Schedule of components of lease cost	The components of lease cost for the quarter ended March 31, 2019 are as follows (in thousands): Three Months Ended March 31, 2019 Lease costs Amortization of right of use assets $ 110 Interest on lease liabilities 33 Total lease cost $ 143
Schedule of amounts reported for leases in consolidated balance sheets	Amounts reported in the Consolidated Balance Sheets for leases where we are the lessee as of the quarter ended March 31, 2019 were as follows (in thousands): March 31, 2019 Operating Leases Right of use asset $ 1,046 Lease liability $ 1,690 Remaining lease term Operating leases 4 years Discount rate Operating leases

Warrants (Tables)

Warrants (Tables)	3 Months Ended
	Mar. 31, 2019
Warrants and Rights Note Disclosure [Abstract]
Schedule of warrants	Warrants Outstanding Exercise Expiration Warrants Warrants March 31, Classification Price Date Issued Exercised 2019 Warrants in connection with private equity placement Liability $ 2.50 6/28/2022 7,000,000 (1,970,000) 5,030,000 Warrants in connection with Horizon and Oxford loan agreement Equity $ 2.45 12/28/2026 428,571 (267,857) 160,714 Warrants in connection with CRG loan agreement Equity $ 7.37 7/14/2024 394,289 — 394,289 Warrants in connection with CRG loan amendment in January 2018 Equity $ 10.00 1/16/2025 1,248,250 — 1,248,250 9,071,110 6,833,253

Stock-based compensation (Table

Stock-based compensation (Tables)	3 Months Ended
	Mar. 31, 2019
Stock-based compensation
Schedule of summary of outstanding stock options activity	Options Outstanding Weighted- Average Weighted- Remaining Average Contractual Number of Exercise Term Aggregate Shares Price (Years) Intrinsic Value (in thousands) Outstanding—January 1, 2019 8,579,511 $ 7.35 7.57 $ 3,281 Granted 1,767,400 $ 4.70 Forfeited and cancelled (101,332) $ 6.31 Exercised (39,728) $ 4.19 Outstanding—March 31, 2019 10,205,851 $ 6.91 7.77 $ 5,385 Vested and exercisable—March 31, 2019 4,224,745 $ 8.63 6.25 $ 2,506
Schedule of stock-based compensation	We recognized stock‑based compensation expense for employees and directors for stock options and RSUs as follows (in thousands): Three Months Ended March 31, 2019 2018 Selling, general and administrative $ 1,811 $ 1,280 Research and development 512 408 Total stock-based compensation $ 2,323 $ 1,688
Schedule of assumptions for estimating fair value of stock option awards	Three Months Ended March 31, 2019 2018 Expected term (in years) 6.09 6.08 Risk-free interest rate 2.47% - 2.61% 2.25% - 2.71% Expected volatility 80.00% - 80.85% 85.00% Dividend rate —% —%
Schedule of summary of unvested RSUs	Number of Shares Outstanding—January 1, 2019 143,100 Granted 634,000 Forfeited (6,250) Vested (12,000) Unvested—March 31, 2019 758,850

Organization - Sale of subsidia

Organization - Sale of subsidiary (Details) $ in Millions	1 Months Ended	3 Months Ended
	Dec. 31, 2018USD ($)employee	Mar. 31, 2018shares
Ordinary Shares
Agreements
Issuance of shares, net of costs (in shares) | shares		5,255,683
Macrilen Acquisition Agreement | Novo
Agreements
Consideration for sale of subsidiary | $	$ 145
Services Agreement | NNI
Agreements
Number of employees funded by counterparty | employee	23
Term of agreement	3 years

Organization - Products (Detail

Organization - Products (Detail)	Mar. 31, 2019product
Organization
Number of clinical-stage product candidates	2

Organization - Liquidity (Detai

Organization - Liquidity (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2019	Dec. 31, 2018
Liquidity
Cash and cash equivalents	$ 104,306	$ 122,490
Substantial doubt about going concern, within one year	false

Summary of significant accoun_4

Summary of significant accounting policies and basis of presentation - Leases (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2019USD ($)
Summary of significant accounting policies and basis of presentation
Operating Lease, Right-of-Use Asset, Statement of Financial Position	us-gaap:OperatingLeaseRightOfUseAsset
Operating Lease, Liability, Statement of Financial Position	us-gaap:OtherLiabilitiesCurrent us-gaap:OtherLiabilitiesNoncurrent
Lease, Practical Expedients, Package	true
Lease, Practical Expedient, Use of Hindsight	false
Right to use assets, net	$ 1,046
Lease liability	$ 1,690

Summary of significant accoun_5

Summary of significant accounting policies and basis of presentation - Segment information (Details)	3 Months Ended
	Mar. 31, 2019segment
Segment information
Number of operating segments	1

Summary of significant accoun_6

Summary of significant accounting policies and basis of presentation - Net loss per share (Details) - shares	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Warrant liability
Net income (loss) per share
Anti-dilutive shares of common stock (in shares)	6,833,253	8,803,253
Stock options
Net income (loss) per share
Anti-dilutive shares of common stock (in shares)	10,205,851	7,960,469
RSUs
Net income (loss) per share
Anti-dilutive shares of common stock (in shares)	758,850	173,400

Revenue recognition - Reserves

Revenue recognition - Reserves (Details) $ in Millions	3 Months Ended
	Mar. 31, 2019USD ($)
Reserve for returns	$ 0
Percentage of patient’s cost of branded prescription drugs related to the Medicare Part D Coverage Gap for which manufacturers of pharmaceutical products are responsible	50.00%
Maximum
Temporary supply period	60 days
Financial need period	12 months

Fair value measurement (Details

Fair value measurement (Details) - USD ($) $ in Thousands	Mar. 31, 2019	Dec. 31, 2018
Liabilities
Warrant liabilities	$ 17,333	$ 15,513
Recurring
Assets
Cash equivalents	103,939	122,300
Total Assets	103,939	122,300
Liabilities
Warrant liabilities	17,333	15,513
Total liabilities	17,333	15,513
Recurring | Level I
Assets
Cash equivalents	103,939	122,300
Total Assets	103,939	122,300
Recurring | Level III
Liabilities
Warrant liabilities	17,333	15,513
Total liabilities	$ 17,333	$ 15,513

Fair value measurement - Level

Fair value measurement - Level 3 (Details) - Warrant liability $ in Thousands	3 Months Ended
	Mar. 31, 2019USD ($)
Reconciliation of level 3:
Balance at beginning of period	$ 15,513
Unrealized loss on fair value of warrants	1,820
Balance at end of period	$ 17,333

Intangible assets and goodwil_2

Intangible assets and goodwill - Components (Details) - USD ($) $ in Thousands	3 Months Ended		12 Months Ended
	Mar. 31, 2019	Mar. 31, 2018	Dec. 31, 2018	Mar. 31, 2019
Intangible assets and goodwill
Finite-lived intangible assets, Amortization	$ (1,256)	$ (1,769)	$ (7,187)
Goodwill, Beginning of Period	7,256	7,256	7,256
Goodwill, End of Period	7,256		7,256
Total, Beginning of Period	37,388	42,411	42,411
Total, Additions			24,834
Sold			(22,670)
Amortization	(1,256)	(1,769)	(7,187)
Total	37,388	42,411	42,411	$ 36,132
Keveyis
Intangible assets and goodwill
Finite-lived intangible assets, Beginning of Period	30,132	$ 35,155	35,155
Finite-lived intangible assets, Amortization	(1,256)		(5,023)
Finite-lived intangible assets, End of Period	28,876		30,132
Amortization	$ (1,256)		(5,023)
Macrilen
Intangible assets and goodwill
Finite-lived intangible assets, Additions			24,834
Finite-lived intangible assets, Amortization			(2,164)
Sold			(22,670)
Amortization			$ (2,164)

Intangible assets and goodwil_3

Intangible assets and goodwill - Asset purchase (Details) $ in Thousands	1 Months Ended		3 Months Ended		12 Months Ended
	Mar. 31, 2017USD ($)	Dec. 31, 2016USD ($)item	Mar. 31, 2019USD ($)	Mar. 31, 2018USD ($)	Dec. 31, 2018USD ($)	Dec. 31, 2017USD ($)	Dec. 31, 2016installment	Dec. 31, 2016USD ($)
In-process research and development and Goodwill
Amortization of intangible assets			$ 1,256	$ 1,769	$ 7,187
Keveyis
In-process research and development and Goodwill
Acquired product rights			28,876		30,132	$ 35,155
Amortization of intangible assets			$ 1,256		5,023
Keveyis | Taro | Acquired product rights
In-process research and development and Goodwill
Acquired product rights								$ 40,200
Transaction costs								2,400
Estimated life		8 years
Macrilen
In-process research and development and Goodwill
Amortization of intangible assets					$ 2,164
Asset Purchase Agreement | Keveyis | Taro | Acquired product rights
In-process research and development and Goodwill
Number of installment payments		2					2
Installment payment	$ 7,500	$ 1,000
Supply Agreement | Keveyis | Taro | Acquired product rights
In-process research and development and Goodwill
Acquired product rights								$ 29,300

Accrued liabilities and other_3

Accrued liabilities and other current liabilities (Details) - USD ($) $ in Thousands	Mar. 31, 2019	Dec. 31, 2018
Accrued liabilities and other current liabilities
Supply agreement - current portion	$ 5,375	$ 1,638
Consulting and professional fees	2,659	4,145
Employee compensation	1,711	5,717
Accrued sales allowances	1,815	2,233
Accrued royalties	1,367	802
Accrued taxes	1,193	535
Lease liability - current portion	326
Other	408	995
Accrued liabilities and other current liabilities	$ 14,854	$ 16,065

Commitments and contingencies -

Commitments and contingencies - Commitments to Taro (Details) - Acquired product rights - Taro - Keveyis $ in Millions	1 Months Ended
	Mar. 31, 2017USD ($)	Dec. 31, 2016USD ($)item	Mar. 31, 2019USD ($)	Dec. 31, 2016installment	Dec. 31, 2016USD ($)
Asset Purchase Agreement
Other Commitments
Number of installment payments		2		2
Installment payment	$ 7.5	$ 1
Potential milestone payments					$ 7.5
Supply Agreement
Other Commitments
Minimum amount of purchases obligated					$ 29
Purchase obligation period		6 years
Remaining obligation			$ 22.1

Leases - Future minimum commitm

Leases - Future minimum commitments (Details) $ in Thousands	Mar. 31, 2019USD ($)
Future minimum commitments under facility operating leases:
2019	$ 326
2020	470
2021	481
2022	492
2023	207
Total minimum lease payments	$ 1,976
Minimum
Lessee, Lease, Description [Line Items]
Initial lease term	1 year
Maximum
Lessee, Lease, Description [Line Items]
Initial lease term	5 years

Leases - Components of lease co

Leases - Components of lease cost (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2019USD ($)
Lease costs
Amortization of right of use assets	$ 110
Interest on lease liabilities	33
Total lease cost	$ 143

Leases - Balance sheet location

Leases - Balance sheet location (Details) $ in Thousands	Mar. 31, 2019USD ($)
Leases
Right to use assets	$ 1,046
Operating Lease, Right-of-Use Asset, Statement of Financial Position	us-gaap:OperatingLeaseRightOfUseAsset
Lease liability	$ 1,690
Operating Lease, Liability, Statement of Financial Position	us-gaap:OtherLiabilitiesCurrent us-gaap:OtherLiabilitiesNoncurrent
Remaining lease term - Operating leases	4 years
Discount rate - Operating leases	7.69%

Income taxes (Details)

Income taxes (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2019USD ($)
Components of income tax expense (benefit)
Income tax expense	$ 677

Warrants (Details)

Warrants (Details)	3 Months Ended
	Mar. 31, 2019$ / sharesshares
Warrants
Warrants Issued (in shares)	9,071,110
Warrants Outstanding (in shares)	6,833,253
Warrants in connection with private equity placement
Warrants
Exercise Price (in dollars per share) | $ / shares	$ 2.50
Warrants Issued (in shares)	7,000,000
Warrants Exercised (in shares)	(1,970,000)
Warrants Outstanding (in shares)	5,030,000
Warrants in connection with Horizon and Oxford loan agreement
Warrants
Exercise Price (in dollars per share) | $ / shares	$ 2.45
Warrants Issued (in shares)	428,571
Warrants Exercised (in shares)	(267,857)
Warrants Outstanding (in shares)	160,714
Warrants in connection with CRG loan agreement
Warrants
Exercise Price (in dollars per share) | $ / shares	$ 7.37
Warrants Issued (in shares)	394,289
Warrants Outstanding (in shares)	394,289
Warrants in connection with CRG loan amendment in January 2018
Warrants
Exercise Price (in dollars per share) | $ / shares	$ 10
Warrants Issued (in shares)	1,248,250
Warrants Outstanding (in shares)	1,248,250

Stock-based compensation - Gene

Stock-based compensation - General (Details)	Mar. 31, 2019shares
Inducement Plan
Stock-based compensation
Number of shares available for issuance	738,953
2015 Plan
Stock-based compensation
Number of shares available for issuance	262,168
Non-Employee Director Plan
Stock-based compensation
Number of shares available for issuance	272,151

Stock-based compensation - Stoc

Stock-based compensation - Stock options activity (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended	12 Months Ended
	Mar. 31, 2019	Dec. 31, 2018
Summary of outstanding stock options
Outstanding at beginning of period (in shares)	8,579,511
Granted (in shares)	1,767,400
Forfeited and cancelled (in shares)	(101,332)
Exercised (in shares)	(39,728)
Outstanding at end of period (in shares)	10,205,851	8,579,511
Vested and exercisable at end of period (in shares)	4,224,745
Weighted-Average Exercise Price
Granted (in dollars per share)	$ 4.70
Forfeited and cancelled (in dollars per share)	6.31
Exercised (in dollars per share)	4.19
Outstanding (in dollars per share)	6.91	$ 7.35
Vested and exercisable at end of period (in dollars per share)	$ 8.63
Additional Disclosures - Options
Weighted Average Remaining Contractual Term, Outstanding	7 years 9 months 7 days	7 years 6 months 26 days
Weighted Average Remaining Contractual Term, Vested and exercisable	6 years 3 months
Aggregate Intrinsic Value, Outstanding (in dollars)	$ 5,385	$ 3,281
Aggregate Intrinsic Value, Vested and exercisable at end of the period (in dollars)	$ 2,506

Stock-based compensation - Opti

Stock-based compensation - Options outstanding (Details) - $ / shares	3 Months Ended
	Mar. 31, 2019	Dec. 31, 2018
Stock options
Options outstanding (in shares)	10,205,851	8,579,511
Options outstanding, weighted-average exercise price (in dollars per share)	$ 6.91	$ 7.35
Vesting subject to acceleration
Stock options
Options outstanding (in shares)	88,908
Options outstanding, weighted-average exercise price (in dollars per share)	$ 18.80
Share price vesting threshold (in dollars per share)	31.46
Vesting based on market appreciation event | Grantee, One
Stock options
Share price vesting threshold (in dollars per share)	30.14
Vesting based on market appreciation event | Grantee, Two
Stock options
Share price vesting threshold (in dollars per share)	$ 33.66
Vesting based on market appreciation event | Vesting at market appreciation event
Stock options
Options outstanding (in shares)	97,652
Consecutive trading days threshold	20 days
Vesting based on market appreciation event | Vesting at one year anniversary of market appreciation event
Stock options
Options outstanding (in shares)	97,652
Vesting period following event	1 year
Consecutive trading days threshold	20 days

Stock-based compensation - St_2

Stock-based compensation - Stock-based compensation expense (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Stock-based compensation expense
Total stock-based compensation	$ 2,323	$ 1,688
Stock options
Stock-based compensation expense
Total unrecognized compensation expense	$ 19,000
Estimated weighted average period over which expense is expected to be recognized	2 years 11 months 12 days
Selling, general and administrative
Stock-based compensation expense
Total stock-based compensation	$ 1,811	1,280
Research and development
Stock-based compensation expense
Total stock-based compensation	$ 512	$ 408

Stock-based compensation - Fair

Stock-based compensation - Fair value assumptions (Details)	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Fair value assumptions
Expected term (in years)	6 years 1 month 2 days	6 years 29 days
Minimum Risk-free interest rate (as a percent)	2.47%	2.25%
Maximum Risk-free interest rate (as a percent)	2.61%	2.71%
Minimum Expected volatility (as a percent)	80.00%	85.00%
Maximum Expected volatility (as a percent)	80.85%
Expected volatility (as a percent)		85.00%

Stock-based compensation - Rest

Stock-based compensation - Restricted stock units (Details) - USD ($)	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Stock-based compensation
Stock-based compensation	$ 2,323,000	$ 1,688,000
RSUs
Stock-based compensation
Vesting period	2 years
Number of shares issued for each stock award vested	1
Stock-based compensation	$ 239,000	$ 149,000
Total unrecognized compensation expense	$ 3,100,000
Estimated weighted average period over which expense is expected to be recognized	1 year 9 months 18 days
Summary of unvested RSUs
Unvested - Beginning of period (in shares)	143,100
Granted (in shares)	634,000
Forfeited (in shares)	(6,250)
Vested (in shares)	(12,000)
Unvested - End of period (in shares)	758,850