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Document and Entity Information - shares	9 Months Ended
Document and Entity Information - shares	Sep. 30, 2017	Nov. 06, 2017
Document And Entity Information
Entity Registrant Name	Ophthotech Corp.
Entity Central Index Key	0001410939
Document Type	10-Q
Document Period End Date	Sep. 30, 2017
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Entity Filer Category	Large Accelerated Filer
Entity Common Stock, Shares Outstanding (in shares)		36,038,683
Document Fiscal Year Focus	2017
Document Fiscal Period Focus	Q3

Unaudited Balance Sheets - USD ($) $ in Thousands	Sep. 30, 2017	Dec. 31, 2016
Current assets
Cash and cash equivalents	$ 180,217	$ 133,930
Available for sale securities	0	155,348
Due from Novartis Pharma AG	0	3,531
Prepaid expenses and other current assets	1,436	3,078
Total current assets	181,653	295,887
Property and equipment, net	1,302	3,281
Other assets	27	462
Total assets	182,982	299,630
Current liabilities
Accrued research and development expenses	6,212	47,240
Accounts payable and accrued expenses	7,895	12,032
Deferred revenue	0	6,646
Total current liabilities	14,107	65,918
Deferred revenue, long-term	0	203,330
Royalty purchase liability	125,000	125,000
Total liabilities	139,107	394,248
Stockholders’ equity (deficit)
Preferred stock - $0.001 par value, 5,000,000 shares authorized, no shares issued or outstanding	0	0
Common stock - $0.001 par value, 200,000,000 shares authorized, 36,032,883 and 35,733,276 shares issued and outstanding at September 30, 2017 and December 31, 2016, respectively	36	36
Additional paid-in capital	519,051	504,517
Accumulated deficit	(475,212)	(598,959)
Accumulated other comprehensive loss	0	(212)
Total stockholders’ equity (deficit)	43,875	(94,618)
Total liabilities and stockholders’ equity (deficit)	$ 182,982	$ 299,630

Unaudited Balance Sheets (Parenthetical) - $ / shares	Sep. 30, 2017	Dec. 31, 2016
Statement of Financial Position [Abstract]
Preferred stock, par value (in dollars per share)	$ 0.001	$ 0.001
Preferred stock, shares authorized (in shares)	5,000,000	5,000,000
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)	200,000,000	200,000,000
Common stock, shares issued (in shares)	36,032,883	35,733,276
Common stock, shares outstanding (in shares)	36,032,883	35,733,276

Unaudited Statements of Operations - USD ($) shares in Thousands, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Income Statement [Abstract]
Collaboration revenue	$ 206,654	$ 1,668	$ 209,977	$ 45,587
Operating expenses
Research and development	10,707	50,854	58,343	136,886
General and administrative	7,059	12,024	28,770	37,209
Total operating expenses	17,766	62,878	87,113	174,095
Income (loss) from operations	188,888	(61,210)	122,864	(128,508)
Interest income	391	409	1,113	1,301
Other loss	(12)	(20)	(34)	(88)
Income (loss) before income tax provision (benefit)	189,267	(60,821)	123,943	(127,295)
Income tax provision (benefit)	194	70	196	(158)
Net income (loss)	$ 189,073	$ (60,891)	$ 123,747	$ (127,137)
Net income (loss) per common share
Basic (in dollars per share)	$ 5.26	$ (1.71)	$ 3.45	$ (3.59)
Diluted (in dollars per share)	$ 5.25	$ (1.71)	$ 3.44	$ (3.59)
Weighted average common shares outstanding
Basic (in shares)	35,971	35,594	35,878	35,415
Diluted (in shares)	36,047	35,594	35,984	35,415

Unaudited Statements of Comprehensive Income (Loss) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Statement of Comprehensive Income [Abstract]
Net income (loss)	$ 189,073	$ (60,891)	$ 123,747	$ (127,137)
Other comprehensive income (loss)
Unrealized gain (loss) on available for sale securities, net of tax	186	(41)	212	314
Other comprehensive income (loss)	186	(41)	212	314
Comprehensive income (loss)	$ 189,259	$ (60,932)	$ 123,959	$ (126,823)

Unaudited Statements of Cash Flows - USD ($) $ in Thousands	9 Months Ended
Unaudited Statements of Cash Flows - USD ($) $ in Thousands	Sep. 30, 2017	Sep. 30, 2016
Operating Activities
Net income (loss)	$ 123,747	$ (127,137)
Adjustments to reconcile net income (loss) to net cash used in operating activities
Depreciation	1,979	539
Amortization of premium and discounts on investment securities	140	454
Deferred income taxes	163	22,916
Share-based compensation	14,463	24,889
Changes in operating assets and liabilities
Due from Novartis Pharma AG	3,531	4,383
Income tax receivable	(52)	(20,844)
Prepaid expense and other current assets	1,694	569
Accrued interest receivable	465	157
Other assets	435	19
Accrued research and development expenses	(41,028)	21,197
Accounts payable and accrued expenses	(4,137)	(1,629)
Deferred revenue	(209,976)	(1,298)
Net cash used in operating activities	(108,576)	(75,785)
Investing Activities
Purchase of marketable securities	(12,014)	(48,123)
Maturities of marketable securities	166,806	62,500
Purchase of property and equipment	0	(247)
Net cash provided by investing activities	154,792	14,130
Financing Activities
Proceeds from stock option/employee stock purchase plan exercises	71	5,398
Net cash provided by financing activities	71	5,398
Net change in cash and cash equivalents	46,287	(56,257)
Cash and cash equivalents
Beginning of period	133,930	221,861
End of period	180,217	165,604
Supplemental disclosures of non-cash information related to investing activities
Change in unrealized gain (loss) on available for sale securities, net of tax	$ 212	$ 314

Business	9 Months Ended
Business	Sep. 30, 2017
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Business	1. Business Description of Business and Organization Ophthotech Corporation (the “Company” or “Ophthotech”) was incorporated on January 5, 2007, in Delaware. The Company is a biopharmaceutical company specializing in the development of novel therapeutics to treat ophthalmic diseases, with a focus on age-related and orphan retinal diseases. The Company currently has two product candidates in development, Zimura® (avacincaptad pegol), an anti-complement factor C5 aptamer, and Fovista® (pegpleranib), an anti-platelet derived growth factor, or PDGF, aptamer. Prior to 2017, the Company's primary focus was on developing Fovista and Zimura for various types of age-related macular degeneration, or AMD, which is a disorder of the central portion of the retina, known as the macula, that may result in blindness. In December 2016, the Company announced that two of its three pivotal Phase 3 clinical trials for Fovista in combination with the anti-vascular endothelial growth factor, or anti-VEGF, drug Lucentis® (ranibizumab) for the treatment of wet AMD failed to meet their primary endpoint. In August 2017, the Company announced that the third pivotal Phase 3 clinical trial for Fovista in combination with the anti-VEGF drugs Eylea® (aflibercept) or Avastin® (bevacizumab) for the treatment of wet AMD, referred to as the OPH1004 trial, failed to meet its primary endpoint. The Company is in the process of winding down the OPH1004 Fovista trial and has no future plans to develop Fovista in wet AMD and only very limited Fovista development activity outside of wet AMD. In early 2017, the Company announced that it had engaged a financial advisor to assist it in reviewing the Company’s strategic alternatives, including identifying potential business development opportunities. Also beginning in early 2017, the Company undertook a reassessment of its development plans for Zimura and Fovista, which included an evaluation of the scientific rationale for potentially developing these product candidates in one or more other ophthalmic indications for which there is a high unmet need. In July 2017, the Company announced that it is pursuing a strategy to leverage its clinical experience and retina expertise to identify and develop therapies to treat multiple ophthalmic orphan diseases for which there are limited or no treatment options available. To this end, the Company is preparing to initiate a randomized, controlled Phase 2b clinical trial of Zimura as a monotherapy for the treatment of autosomal recessive Stargardt disease ("STGD1"), an inherited retinal orphan disease causing vision loss during childhood or adolescence, before the end of 2017. In parallel, the Company is continuing its on-going Zimura programs in age-related retinal diseases. The Company is also continuing its business development efforts to identify and potentially obtain rights to additional products, product candidates and technologies that would complement its strategic goals as well as other compelling ophthalmology opportunities.

Summary of Significant Accounting Policies

9 Months Ended

Sep. 30, 2017

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

2. Summary of Significant Accounting Policies

The Company’s significant accounting policies are described in Note 2, “Significant Accounting Policies,” in the notes to the audited consolidated financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2016.

Basis of Presentation

The accompanying unaudited financial information as of September 30, 2017 and for the three and nine months ended September 30, 2017 and 2016 has been prepared by the Company pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) have been condensed or omitted pursuant to such rules and regulations. The December 31, 2016 Balance Sheet was derived from the Company’s audited financial statements. These interim financial statements should be read in conjunction with the notes to the financial statements contained in the Company’s Annual Report on Form 10-K (“Annual Report”) for 2016, as filed with the SEC on February 28, 2017.

In the opinion of management, the unaudited financial information as of September 30, 2017 and for the three and nine months ended September 30, 2017 and 2016, reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of the financial position, results of operations and cash flows of the Company. The results of operations for the three and nine months ended September 30, 2017 and 2016 are not necessarily indicative of the operating results for the full fiscal year or any future period.

Segment and geographic information

Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating and reporting segment.

Use of Estimates

The preparation of financial statements and related disclosures in conformity with GAAP requires management to make estimates and judgments that affect the amounts reported in the financial statements and accompanying notes. The Company bases its estimates and judgments on historical experience and on various other assumptions that it believes are reasonable under the circumstances. The amounts of assets and liabilities reported in the Company’s Balance Sheets and the amount of expenses reported for each of the periods presented are affected by estimates and assumptions, which are used for, but not limited to, accounting for research and development costs, revenue recognition, accounting for share-based compensation and accounting for income taxes. Actual results could differ from those estimates.

Cash and Cash Equivalents

The Company considers all highly liquid investments with an original maturity of 90 days or less when purchased to be cash equivalents. The carrying amounts reported in the Balance Sheets for cash and cash equivalents are valued at cost, which approximates their fair value.

As of September 30, 2017, the Company had cash and cash equivalents of approximately $180.2 million. The Company believes that its existing cash and cash equivalents as of September 30, 2017 will be sufficient to fund its operations and capital expenditure requirements as currently planned for at least the next 12 months.

Available for Sale Securities

The Company considers securities with original maturities of greater than 90 days when purchased to be available for sale securities. Available for sale securities with original maturities of greater than one year are recorded as non-current assets. Available for sale securities are recorded at fair value and unrealized gains and losses are recorded within accumulated other comprehensive income (loss). The estimated fair value of the available for sale securities is determined based on quoted market prices or rates for similar instruments. In addition, the cost of debt securities in this category is adjusted for amortization of premium and accretion of discount to maturity. The Company evaluates securities with unrealized losses to determine whether such losses, if any, are other than temporary.

Revenue Recognition

Collaboration Revenue

In May 2014, the Company received an upfront payment of $200.0 million in connection with its licensing and commercialization agreement (the “Novartis Agreement”) with Novartis Pharma AG ("Novartis"). Prior to the third quarter of 2017, this payment had not been recorded as revenue due to the existence of a contingency with respect to the Company's right to terminate the agreement in certain circumstances and the associated termination fee equivalent to the entire $200.0 million upfront payment, which the Company would have been required to pay if it had elected to exercise this termination option. The Company and Novartis entered into a letter agreement in July 2017 (the "Letter Agreement") that waived the Company's termination right, thereby resolving the contingency and allowing the Company to immediately recognize as revenue the portion of the upfront payment allocated using the relative selling price method to deliverables completed during prior periods. See "Note 5-Licensing and Commercialization Agreement with Novartis Pharma AG" below for a further description of the Letter Agreement. During the third quarter of 2017, the Company completed the remaining deliverables under the Novartis Agreement and the Letter Agreement and recognized as revenue the balance of all of the payments previously received from Novartis related to licensing, research and development, manufacturing and joint operating committee activities that had been previously deferred using the relative selling price method. In total, during the third quarter of 2017, the Company recognized $206.7 million in previously deferred collaboration revenue in connection with the Novartis Agreement. The recognition of this revenue during the period did not impact the Company's cash balance. On October 23, 2017, following the failure of the Phase 3 Fovista program and pursuant to the terms of the Letter Agreement, Novartis elected to terminate the Novartis Agreement with immediate effect.

Below is a summary of the components of the Company’s collaboration revenue for the three and nine months ended September 30, 2017 and 2016:



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

License revenue	$	152,912	$	—	$	152,912	$	22,937
Research and development activity revenue	52,864		1,664		56,180		8,089
API transfer revenue	754		—		754		14,545
Joint operating committee revenue	124		4		131		16
Total collaboration revenue	$	206,654	$	1,668	$	209,977	$	45,587

Concentration of Credit Risk

The Company’s financial instruments that are exposed to concentration of credit risk consist primarily of cash and cash equivalents and available for sale securities. The Company maintains its cash in bank accounts, which generally exceed federally insured limits. The Company maintains its cash equivalents in investments in money market funds and, at times, in U.S. Treasury securities and investment-grade corporate debt securities with original maturities of 90 days or less.

The Company’s available for sale securities are also invested in U.S. Treasury securities and investment-grade corporate debt securities. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents and available for sale securities. As of September 30, 2017, the Company did not hold any available for sale securities.

Concentration of Suppliers

The Company currently relies exclusively upon a single third-party manufacturer to provide supplies of the active pharmaceutical ingredient, or API, for Zimura on a purchase order basis. The Company also engages a single third-party manufacturer to provide fill/finish services for clinical supplies of Zimura. In addition, the Company currently relies upon a single third-party supplier to supply it with the proprietary polyethylene glycol, or PEG, reagent used to manufacture Zimura on a purchase order basis. Furthermore, the Company and its contract manufacturers currently rely upon sole-source suppliers of certain raw materials and other specialized components of production used in the manufacture and fill/finish of Zimura. If the Company’s third-party manufacturers or fill/finish service providers should become unavailable to the Company for any reason, including as a result of capacity constraints, financial difficulties or insolvency, the Company believes that there are a limited number of potential replacement manufacturers, and the Company likely would incur added costs and delays in identifying or qualifying such replacements.

Foreign Currency Translation

The Company considers the U.S. dollar to be its functional currency. Expenses denominated in foreign currencies are translated at the exchange rate on the date the expense is incurred. The effect of exchange rate fluctuations on translating foreign currency assets and liabilities into U.S. dollars is included in the Statements of Operations. Foreign exchange transaction gains and losses are included in the results of operations and are not material in the Company’s financial statements.

Financial Instruments

Cash equivalents and available for sale securities are reflected in the accompanying financial statements at fair value. The carrying amount of accounts payable and accrued expenses, including accrued research and development expenses, approximates fair value due to the short-term nature of those instruments.

Property and Equipment

Property and equipment, which consists mainly of manufacturing and clinical equipment, furniture and fixtures, computers, software, and other office equipment, and leasehold improvements, are carried at cost less accumulated depreciation. Depreciation is computed over the estimated useful lives of the respective assets, generally three to ten years, using the straight-line method. Amortization of leasehold improvements is recorded over the shorter of the lease term or estimated useful life of the related asset.

Research and Development

Research and development expenses primarily consist of costs associated with the manufacturing, development and clinical testing of Zimura and Fovista as well as costs associated with the preclinical development of other product candidates and formulations. Research and development expenses consist of:


•	external research and development expenses incurred under arrangements with third parties, such as contract research organizations (“CROs”) and other vendors and contract manufacturing organizations (“CMOs”) for the production of drug substance and drug product; and


•	employee-related expenses, including salaries, benefits and share-based compensation expense.

Research and development expenses also include costs of acquired product licenses and related technology rights where there is no alternative future use, costs of prototypes used in research and development, consultant fees and amounts paid to collaborative partners.

All research and development expenses are charged to operations as incurred in accordance with Financial Accounting Standards Board Accounting Standards Codification Topic, or ASC, 730, Research and Development. The Company accounts for non-refundable advance payments for goods and services that will be used in future research and development activities as expenses when the service has been performed or when the goods have been received, rather than when the payment is made.

Income Taxes

The Company utilizes the liability method of accounting for deferred income taxes, as set forth in ASC 740, Income Taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities.

Share-Based Compensation

The Company follows the provisions of ASC 718, Compensation—Stock Compensation, which requires the measurement and recognition of compensation expense for all share-based payment awards made to employees and non-employee directors, including employee stock options, restricted stock units (“RSUs”) and the option granted to employees to purchase shares under the 2016 Employee Stock Purchase Plan (the “ESPP”). Share-based compensation expense is based on the grant date fair value estimated in accordance with the provisions of ASC 718 and is generally recognized as an expense over the requisite service period, net of estimated forfeitures. For grants containing performance-based vesting provisions, expense is recognized over the estimated achievement period.

Stock Options

The Company estimates the fair value of stock options granted to employees and non-employee directors on the date of grant using the Black-Scholes option-pricing model. Due to the lack of trading history, the Company’s computation of stock-price volatility is based on the volatility rates of comparable publicly held companies over a period equal to the expected term of the options granted by the Company. The Company’s computation of expected term is determined using the “simplified” method, which is the midpoint between the vesting date and the end of the contractual term. The Company believes that it does not have sufficient reliable exercise data in order to justify the use of a method other than the “simplified” method of estimating the expected exercise term of employee stock option grants. The Company utilizes a dividend yield of zero based on the fact that the Company has never paid cash dividends to stockholders and has no current intentions to pay cash dividends. The risk-free interest rate is based on the zero-coupon U.S. Treasury yield at the date of grant for a term equivalent to the expected term of the option.

For stock options granted as consideration for services rendered by consultants, the Company recognizes expense in accordance with the requirements of ASC 505-50, Equity Based Payments to Non-Employees. Consultant stock option grants are recorded as an expense over the vesting period of the underlying stock options. At the end of each financial reporting period prior to vesting, the value of these options, as calculated using the Black-Scholes option-pricing model, will be re-measured using the fair value of the Company’s common stock and the non-cash expense recognized during the period will be adjusted accordingly. Since the fair value of options granted to consultants is subject to change in the future, the amount of the future expense will include fair value re-measurements until the stock options are fully vested.

The weighted-average assumptions used to estimate grant date fair value of stock options using the Black-Scholes option pricing model were as follows for the three and nine months ended September 30, 2017 and 2016:



	Three months ended September 30,		Nine months ended September 30,
	2017	2016	2017	2016
Expected common stock price volatility	82%	71%	81%	71%
Risk-free interest rate	1.95%-2.06%	1.14% -1.32%	1.82%-2.38%	1.14%-1.92%
Expected term of options (years)	6.1	6.0	6.1	6.1
Expected dividend yield	—	—	—	—

RSUs

The Company estimates the fair value of RSUs granted to employees using the closing market price of the Company’s common stock on the date of grant.

ESPP

In April 2016, the board of directors adopted the ESPP pursuant to which the Company may sell up to an aggregate of 1,000,000 shares of common stock. The ESPP was approved by the Company’s stockholders in June 2016. The ESPP is considered compensatory and the fair value of the discount and look back provision are estimated using the Black-Scholes option-pricing model and recognized over the six month withholding period prior to purchase.

Share-based compensation expense includes expenses related to stock options and RSUs granted to employees, non-employee directors and consultants, as well as the option granted to employees to purchase shares under the ESPP, all of which have been reported in the Company’s Statements of Operations as follows:



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

Research and development	$	1,840	$	5,664	$	8,887	$	16,732
General and administrative	1,571		2,584		5,576		8,157
Total	$	3,411	$	8,248	$	14,463	$	24,889

Recent Accounting Pronouncements

In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-9, Revenue from Contracts with Customers (Topic 606) (“ASU 2014-9”). ASU 2014-9 outlines a new, single comprehensive model for entities to use in accounting for revenue arising from contracts with customers and supersedes most current revenue recognition guidance, including industry-specific guidance. This new revenue recognition model provides a five-step analysis in determining when and how revenue is recognized. The new model will require revenue recognition to depict the transfer of promised goods or services to customers in an amount that reflects the consideration a company expects to receive in exchange for those goods or services. The FASB subsequently issued additional clarifying standards to address issues arising from implementation of the new revenue standard, including a one-year deferral of the effective date for the new revenue standard. Public companies should now apply the guidance in ASU 2014-9 to annual reporting periods beginning after December 15, 2017 and interim periods within those annual periods. Earlier application is permitted only as of annual reporting periods beginning after December 15, 2016, including interim periods within that annual period. Companies may use either a full retrospective or a modified retrospective approach to adopt ASU 2014-9. Due to the recent development and termination of the Company's collaboration contracts with Novartis, the Company does not expect the standard to have a material impact upon adoption.

In February 2016, the FASB issued ASU No. 2016-2, Leases (Topic 842). Under the new guidance, lessees will be required to recognize the following for all leases (with the exception of short-term leases) at the commencement date: (1) a lease liability, which is a lessee’s obligation to make lease payments arising from a lease, measured on a discounted basis; and (2) a right-of-use asset, which is an asset that represents the lessee’s right to use, or control the use of, a specified asset for the lease term. Under the new guidance, lessor accounting is largely unchanged. Certain targeted improvements were made to align, where necessary, lessor accounting with the lessee accounting model and Topic 606, Revenue from Contracts with Customers. The new lease guidance simplified the accounting for sale and leaseback transactions primarily because lessees must recognize lease assets and lease liabilities. Lessees will no longer be provided with a source of off-balance sheet financing. Publicly-traded business entities should apply the amendments in ASU 2016-2 for fiscal years beginning after December 15, 2018, including interim periods within those fiscal years (i.e., January 1, 2019, for a calendar year entity). Early application is permitted for all publicly-traded business entities and all nonpublicly-traded business entities upon issuance. Lessees (for capital and operating leases) and lessors (for sales-type, direct financing, and operating leases) must apply a modified retrospective transition approach for leases existing at, or entered into after, the beginning of the earliest comparative period presented in the financial statements. The modified retrospective approach would not require any transition accounting for leases that expired before the earliest comparative period presented. Lessees and lessors may not apply a full retrospective transition approach. The Company is currently assessing the impact that adopting this new accounting guidance will have on its financial statements and footnote disclosures.

In August 2016, the FASB issued ASU No. 2016-15, Statement of Cash Flows (Topic 230): Classification of Certain Cash Receipts and Cash Payments, which clarifies the presentation of certain specific cash flow issues in the Statement of Cash Flows. For public companies, the amendments are effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual periods and early adoption is permitted. This new guidance is not expected to have a material impact on the Company’s Consolidated Financial Statements.

In January 2017, the FASB issued ASU No. 2017-01, Business Combinations (Topic 805): Clarifying the Definition of a Business, in an effort to clarify the definition of a business with the objective of adding guidance to assist entities with evaluating whether transactions should be accounted for as acquisitions (or disposals) of assets or businesses. The amendments of this ASU are effective for fiscal years beginning after December 15, 2017, and interim periods within those fiscal years. Early adoption is permitted. This new guidance will be applicable for the Company’s acquisitions on or after January 1, 2018.

Net Income (Loss) Per Common Share

9 Months Ended

Sep. 30, 2017

Earnings Per Share [Abstract]

Net Income (Loss) Per Common Share

3. Net Income (Loss) Per Common Share

Basic and diluted net income (loss) per common share is determined by dividing net income (loss) by the weighted average common shares outstanding during the period. For the periods where there is a net loss, stock options and RSUs have been excluded from the calculation of diluted net loss per common share because their effect would be anti-dilutive. Therefore, the weighted average common shares used to calculate both basic and diluted net loss per common share would be the same. The following table sets forth the computation of basic and diluted net income (loss) per common share for the periods indicated:



	Three months ended September 30,					Nine months ended September 30,
	2017		2016			2017		2016

Basic and diluted net income (loss) per common share calculation:
Net income (loss)	$	189,073	$	(60,891	)	$	123,747	$	(127,137	)
Weighted average common shares outstanding - basic	35,971		35,594			35,878		35,415
Plus: net effect of dilutive stock options and unvested restricted stock units	76		—			106		—
Weighted average common shares outstanding - dilutive	36,047		35,594			35,984		35,415
Net income (loss) per share of common stock - basic	$	5.26	$	(1.71	)	$	3.45	$	(3.59	)
Net income (loss) per share of common stock - diluted	$	5.25	$	(1.71	)	$	3.44	$	(3.59	)

The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding for the periods presented, as they would be anti-dilutive:



	Three months ended September 30,		Nine months ended September 30,
	2017	2016	2017	2016

Stock options outstanding	3,640	3,423	3,813	3,423
Restricted stock units	269	721	332	721
Total	3,909	4,144	4,145	4,144

Cash, Cash Equivalents and Available for Sale Securities

9 Months Ended

Sep. 30, 2017

Cash, Cash Equivalents, and Short-term Investments [Abstract]

Cash, Cash Equivalents and Available for Sale Securities

4. Cash, Cash Equivalents and Available for Sale Securities

The Company considers all highly liquid investments purchased with original maturities of 90 days or less at the date of purchase to be cash equivalents. Cash and cash equivalents included cash of $13.2 million and $25.8 million at September 30, 2017 and December 31, 2016, respectively. Cash and cash equivalents at September 30, 2017 and December 31, 2016 also included $167.1 million and $108.1 million, respectively, of investments in money market funds, U.S. Treasury securities and certain short-term investment-grade corporate debt securities with original maturities of 90 days or less.

The Company considers securities with original maturities of greater than 90 days at the date of purchase to be available for sale securities. The Company held no available for sale securities at September 30, 2017. The Company held available for sale securities with a fair value totaling $155.3 million at December 31, 2016. These available for sale securities consisted of U.S. Treasury securities and investment-grade corporate debt securities. During the quarter ended September 30, 2017, the Company's investments matured and were reinvested in money market funds.

Available for sale securities, including carrying value and estimated fair values as of December 31, 2016, are summarized as follows:



	As of December 31, 2016
	Cost		Unrealized Gains		Unrealized Losses			Fair Value

U.S. Treasury securities	$	120,288	$	6	$	(33	)	$	120,261
Corporate debt securities	35,114		—		(27		)	35,087
Total	$	155,402	$	6	$	(60	)	$	155,348

The Company’s available for sale securities are reported at fair value on the Company’s Balance Sheets. Unrealized gains (losses) are reported within accumulated other comprehensive income (loss) in the statements of comprehensive income (loss). The cost of securities sold and any realized gains/losses from the sale of available for sale securities are based on the specific identification method. The changes in accumulated other comprehensive income (loss) associated with the unrealized gain (loss) on available for sale securities during the three and nine months ended September 30, 2017 and September 30, 2016 were as follows:



	Three months ended September 30,						Nine months ended September 30,
	2017			2016			2017			2016

Beginning balance	$	(186	)	$	(118	)	$	(212	)	$	(473	)
Current period changes in fair value before reclassifications, net of tax	186			(41		)	212			314
Amounts reclassified from accumulated other comprehensive income (loss), net of tax	—			—			—			—
Total other comprehensive income (loss)	186			(41		)	212			314
Ending balance	$	—		$	(159	)	$	—		$	(159	)

Licensing and Commercialization Agreement with Novartis Pharma AG

9 Months Ended

Sep. 30, 2017

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Licensing and Commercialization Agreement with Novartis Pharma AG

5. Licensing and Commercialization Agreement with Novartis Pharma AG

In May 2014, the Company entered into the Novartis Agreement. Under the Novartis Agreement, the Company granted Novartis exclusive rights under specified patent rights, know-how and trademarks controlled by the Company to manufacture, from bulk API supplied by the Company, standalone Fovista products and products combining Fovista with an anti-VEGF drug to which Novartis has rights in a co-formulated product, for the treatment, prevention, cure or control of any human disease, disorder or condition of the eye, and to develop and commercialize those licensed products in all countries outside of the United States (the “Novartis Territory”). The Company agreed to use commercially reasonable efforts to complete its ongoing pivotal Phase 3 clinical program for Fovista and Novartis agreed to use commercially reasonable efforts to develop a standalone Fovista product and a co-formulated product containing Fovista and an anti-VEGF drug to which Novartis has rights, as well as a pre-filled syringe presentation of such products and to use commercially reasonable efforts, subject to obtaining marketing approval, to commercialize licensed products in the Novartis Territory in accordance with agreed development and marketing plans.

In July 2017, the Company and Novartis entered into the Letter Agreement to streamline the process and timeline for evaluating data from the OPH1004 trial once it became available. On October 23, 2017, following the failure of the Phase 3 Fovista program and pursuant to the terms of the Letter Agreement, Novartis elected to terminate the Novartis Agreement with immediate effect. See "—Note 13—Subsequent Events" for a description of the termination notice.

In May 2014, Novartis paid the Company a $200.0 million upfront payment. In each of September 2014 and March 2015, the Company achieved a $50.0 million enrollment-based milestone, and in June 2016, the Company achieved a $30.0 million enrollment-based milestone, for an aggregate total of $130.0 million in enrollment-based milestones under the Novartis Agreement. The Company used the relative selling price method to allocate these payments to contract deliverables based on its performance obligations under the Novartis Agreement.

Activities under the Novartis Agreement were evaluated under ASC 605-25, Revenue Recognition—Multiple Element Arrangements (“ASC 605-25”) (as amended by ASU 2009-13, Revenue Recognition (“ASU 2009-13”)) to determine if they represented a multiple element revenue arrangement. The Novartis Agreement included the following deliverables: (1) an exclusive license to commercialize Fovista outside the United States (the “License Deliverable”); (2) the performance obligation to conduct research and development activities related to the Phase 3 Fovista clinical trials and certain Phase 2 trials for Fovista (the “R&D Activity Deliverable”); (3) the performance obligation to supply API to Novartis for development and manufacturing purposes (the “Manufacturing Deliverable”) and (4) the Company’s obligation to participate on the joint operating committee established under the terms of the Novartis Agreement and related subcommittees (the “Joint Operating Committee Deliverable”). The Company’s obligation to provide access to clinical and regulatory information as part of the License Deliverable included the obligation to provide access to all clinical data, regulatory filings, safety data and manufacturing data to Novartis which was necessary for the commercialization of Fovista in the Novartis Territory. The R&D Activity Deliverable included the right and responsibility for the Company to conduct the Phase 3 Fovista clinical program and other Phase 2 studies of Fovista which were necessary or desirable for regulatory approval or commercialization of Fovista. The Manufacturing Deliverable included the obligation for the Company to supply API to Novartis for clinical purposes, for which Novartis agreed to pay the Company’s manufacturing costs. The Joint Operating Committee Deliverable included the obligation to participate in the Joint Operating Committee and related subcommittees at least through the first anniversary of regulatory approval in the European Union. All of these deliverables were deemed to have stand-alone value and to meet the criteria to be accounted for as separate units of accounting under ASC 605-25. Factors considered in this determination included, among other things, the subject of the licenses and the research and development and commercial capabilities of Novartis. Accordingly, each unit was accounted for separately.

The Novartis Agreement included a termination right for the Company in the event that specified governmental actions prevented the parties from materially progressing the development or commercialization of licensed products. If the Company elected to exercise this termination option, it would have been required to pay a substantial termination fee equivalent to the entire upfront payment amount. The Company concluded that this termination provision constituted a contingent event that was unknown at the inception of the agreement. As such, the Company recorded the $200.0 million upfront payment in deferred revenue, long-term until such time that the contingency related to this termination provision was resolved. In July 2017, the contingency was resolved when the Company permanently waived this termination right as part of the Letter Agreement.

The Letter Agreement also provided Novartis with a shorter notice period in the event Novartis determined to terminate the Novartis Agreement in certain circumstances. In addition, the Letter Agreement provided Novartis with a fully paid-up, royalty free license to use data from the Lucentis monotherapy arms of the Company's Phase 2b OPH1001 trial and Phase 3 OPH1002 and OPH1003 trials in the Novartis Territory in connection with the development, manufacturing and commercialization of Novartis-controlled anti-VEGF products. The Lucentis study data license continues until the fifth anniversary of the Letter Agreement.

The Company evaluated the Letter Agreement under ASC 605-25 and determined that the Letter Agreement does not create any new deliverables. The Company is treating the Fovista license granted at the inception of the Novartis Agreement and the Lucentis study data license granted under the Letter Agreement as one collective technology license (the "Licenses") delivered at the inception of the Novartis Agreement. In addition, as the waiver of its right to terminate the Novartis Agreement as a result of specified governmental actions resolved the Company’s contingency with respect to such termination right and the associated termination fee, the Company allocated the entire previously deferred amount, $200.0 million, to the deliverables that were determined based on the relative selling price at contract inception. Upon entry into the Letter Agreement in July 2017, the Company immediately recognized as revenue $189.8 million of the upfront payment allocated to contract deliverables completed during prior periods. Upon termination of the OPH1004 trial in August 2017, the Company recognized the remaining $16.9 million of collaboration revenue, attributable to the R&D Deliverable, previously deferred under the Novartis Agreement. In total, during the third quarter of 2017, the Company recognized $206.7 million in previously deferred collaboration revenue in connection with the Novartis Agreement. The recognition of this revenue during the period did not impact the Company's cash balance.

Below is a summary of the components of the Company’s collaboration revenue for the three and nine months ended September 30, 2017 and September 30, 2016:



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

License revenue	$	152,912	$	—	$	152,912	$	22,937
Research and development activity revenue	52,864		1,664		56,180		8,089
API transfer revenue	754		—		754		14,545
Joint operating committee revenue	124		4		131		16
Total collaboration revenue	$	206,654	$	1,668	$	209,977	$	45,587

Financing Agreement with Novo A/S	9 Months Ended
Financing Agreement with Novo A/S	Sep. 30, 2017
Related Party Transactions [Abstract]
Financing Agreement with Novo A/S	6. Financing Agreement with Novo A/S In May 2013, the Company entered into a Purchase and Sale Agreement with Novo A/S, which is referred to as the Novo Agreement, pursuant to which the Company had the ability to obtain financing in three tranches in an amount of up to $125.0 million in return for the sale to Novo A/S of aggregate royalties of worldwide sales of (a) Fovista, (b) Fovista-Related Products, and (c) Other Products (each as defined in the Novo Agreement), calculated as mid-single digit percentages of net sales. The Novo Agreement provided for up to three separate purchases for a purchase price of $41.7 million each, at a first, second and third closing, for an aggregate purchase price of $125.0 million. In each purchase, Novo A/S would acquire rights to a low single digit percentage of net sales. In each of May 2013, January 2014 and November 2014, the Company received cash payments of $41.7 million, or $125.0 million in the aggregate, and Novo A/S received, in the aggregate, a right to receive royalties on net sales of Fovista at a mid-single digit percentage. The royalty payment period covered by the Novo Agreement begins on commercial launch and ends, on a product-by-product and country-by-country basis, on the latest to occur of (i) the 12th anniversary of the commercial launch, (ii) the expiration of certain patent rights and (iii) the expiration of the regulatory exclusivity for each product in each country. The Company's obligations under the Novo agreement are secured by a lien on certain of the Company's intellectual property and other rights related to Fovista and other anti-PDGF products the Company may develop. Under the terms of the Novo Agreement, the Company is not required to reimburse or otherwise compensate Novo A/S through any means other than the agreed royalty entitlement. In addition, the Company does not, under the terms of the Novo Agreement, have the right or obligation to prepay Novo A/S in connection with a change of control of the Company or otherwise. The $125.0 million in aggregate proceeds from the three financing tranches under the Novo Agreement represents the full funding available under the Novo Agreement, and has been recorded as a liability on the Company’s Balance Sheet as of September 30, 2017, in accordance with ASC 730, Research and Development. Because there is a significant related party relationship between the Company and Novo A/S, the Company is treating its obligation to make royalty payments under the Novo Agreement as an implicit obligation to repay the funds advanced by Novo A/S. As the Company makes royalty payments in accordance with the Novo Agreement, it will reduce the liability balance. At the time that such royalty payments become probable and estimable, and if such amounts exceed the liability balance, the Company will impute interest accordingly on a prospective basis based on such estimates. The Novo Agreement requires the establishment of a Joint Oversight Committee in the event that Novo A/S does not continue to have a representative on the Company’s board of directors. The Joint Oversight Committee would have responsibilities that include “discussion and review” of all matters related to Fovista research, development, regulatory approval and commercialization, but there is no provision either implicit or explicit that gives the Joint Oversight Committee or its members decision-making authority.

Income Taxes	9 Months Ended
Income Taxes	Sep. 30, 2017
Income Tax Disclosure [Abstract]
Income Taxes	7. Income Taxes For the three and nine months ended September 30, 2017, the Company recorded a provision for income taxes of $0.2 million primarily due to discrete income tax items associated with changes in the fair value of the Company's available for sale marketable securities which are reflected in other comprehensive income (loss). Although the Company had $189.3 million and $123.9 million of net income before income taxes for the three and nine months ended September 30, 2017 as a result of the recognition of deferred revenue under the Novartis Agreement, the Company expects a net loss for tax purposes for 2017 with minimal taxes due. For tax purposes, the Company treated payments received under the Novartis Agreement as revenue at the time the payments were received. The Company recorded a provision for income taxes of $0.1 million for the three months ended September 30, 2016, and a benefit from income taxes of $0.2 million for the nine months ended September 30, 2016 primarily due to discrete income tax items in each period associated with changes in the fair value of the Company's available for sale marketable securities which are reflected in other comprehensive income (loss). In the second quarter of 2017, the IRS concluded an audit of the Company’s U.S. federal income tax returns for the years 2013, 2014 and 2015, resulting in an immaterial amount of additional tax due. Federal net operating losses for 2016 and general business credits generated between 2007 and 2016 remain subject to audit. During the nine months ended September 30, 2017, the Company amended certain state and local income tax returns to claim a refund for taxes previously paid. These claims may result in refunds to the Company of up to approximately $7.9 million. As the Company believes that the likelihood of its position in claiming the refunds does not rise to the level of more likely than not at this time, the Company has not recorded a current tax receivable for the refund claims as of September 30, 2017. The Company considers all available evidence, both positive and negative, to determine whether, based on the weight of that evidence, a valuation allowance is needed to reduce its deferred tax assets to the amount that is more likely than not to be realized. The Company placed significant weight on the fact that the Company expects to be in a cumulative net book loss position for the three-year period ending December 31, 2017 in recording valuation allowances on its deferred tax assets as of September 30, 2017. Pursuant to ASC 740, Income Taxes, the Company routinely evaluates the likelihood of success if challenged on income tax positions claimed on its income tax returns. During the three months ended September 30, 2017, the Company reduced certain deferred tax assets by $5.9 million and reduced the corresponding valuation allowance by an equivalent amount. During the three months ended September 30, 2016, the Company reduced certain deferred tax assets by $3.8 million and reduced the corresponding valuation allowance by an equivalent amount. These items have not been recognized in the financial statements and if disallowed by the tax authorities, would not result in an adjustment to the Company’s effective tax rate, its balance sheet or its cash flow statements for the current year. The Company will continue to evaluate its ability to realize its deferred tax assets on a periodic basis and will adjust such amounts in light of changing facts and circumstances including, but not limited to, future projections of taxable income, tax legislation, rulings by relevant tax authorities, the progress of ongoing tax audits and the regulatory approval of products currently under development. Any additional changes to the valuation allowance recorded on deferred tax assets in the future would impact the Company’s income taxes.

Fair Value Measurements

9 Months Ended

Sep. 30, 2017

Fair Value Disclosures [Abstract]

Fair Value Measurements

8. Fair Value Measurements

ASC 820, Fair Value Measurements and Disclosures (“ASC 820”), defines fair value as the price that would be received to sell an asset, or paid to transfer a liability, in the principal or most advantageous market in an orderly transaction between market participants on the measurement date. The fair value standard also establishes a three-level hierarchy, which requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value.

The Company reviews investments on a periodic basis for other than temporary impairments. This review is subjective as it requires management to evaluate whether an event or change in circumstances has occurred in the period that may have a significant adverse effect on the fair value of the investment. The Company uses the market approach to measure fair value for its financial assets. The market approach uses prices and other relevant information generated by market transactions involving identical or comparable assets. The Company classifies its corporate debt securities within the fair value hierarchy as Level 2 assets, as it primarily utilizes quoted market prices or rates for similar instruments to value these securities.

The valuation hierarchy is based upon the transparency of inputs to the valuation of an asset or liability on the measurement date. The three levels are defined as follows:


•	Level 1—inputs to the valuation methodology are quoted prices (unadjusted) for an identical asset or liability in an active market. The Company’s Level 1 assets consist of investments in money market funds and U.S. Treasury securities.


•	Level 2—inputs to the valuation methodology include quoted prices for a similar asset or liability in an active market or model-derived valuations in which all significant inputs are observable for substantially the full term of the asset or liability. The Company’s Level 2 assets consist of investments in investment-grade corporate debt securities.


•	Level 3—inputs to the valuation methodology are unobservable and significant to the fair value measurement of the asset or liability. The Company does not hold any assets that are measured using Level 3 inputs.

The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company’s assets and liabilities that are measured at fair value on a recurring basis as of September 30, 2017:



	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)

Assets
Investments in money market funds*	$	167,052	$	—	$	—



	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)

Assets
Investments in money market funds*	$	108,096	$	—	$	—
Investments in U.S. Treasury securities	$	120,261	$	—	$	—
Investments in Corporate debt securities	$	—	$	35,087	$	—

* Investments in money market funds, U.S. Treasury securities and corporate debt securities with maturities less than 90 days are reflected in cash and cash equivalents in the accompanying Balance Sheets.

No transfer of assets between Level 1 and Level 2 of the fair value hierarchy occurred during the three and nine months ended September 30, 2017.

Stock Option and Compensation Plans

9 Months Ended

Sep. 30, 2017

Disclosure of Compensation Related Costs, Share-based Payments [Abstract]

Stock Option and Compensation Plans

9. Stock Option and Compensation Plans

The Company adopted its 2007 Stock Incentive Plan (the “2007 Plan”) for employees, non-employee directors and consultants for the purpose of advancing the interests of the Company’s stockholders by enhancing its ability to attract, retain and motivate persons who are expected to make important contributions to the Company. The 2007 Plan provided for the granting of stock option awards, RSUs, and other stock-based and cash-based awards. Following the effectiveness of the 2013 Stock Incentive Plan described below in connection with the closing of the Company’s initial public offering, the Company is no longer granting additional awards under the 2007 Plan.

In August 2013, the Company’s board of directors adopted and the Company’s stockholders approved the 2013 stock incentive plan (the “2013 Plan”), which became effective immediately prior to the closing of the Company’s initial public offering. In June 2015, the Company’s board of directors adopted a first amendment to the 2013 Plan. The 2013 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, RSUs, restricted stock awards and other stock-based awards. Upon the effectiveness of the 2013 Plan, the number of shares of the Company’s common stock that were reserved for issuance under the 2013 Plan was the sum of (1) such number of shares (up to approximately 3,359,641 shares) as is equal to the sum of 739,317 shares (the number of shares of the common stock then available for issuance under the 2007 Plan), and such number of shares of the Company’s common stock that are subject to outstanding awards under the 2007 Plan that expire, terminate or are otherwise surrendered, canceled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right plus (2) an annual increase, to be added the first business day of each fiscal year, beginning with the fiscal year ending December 31, 2014 and continuing until, and including, the fiscal year ending December 31, 2023, equal to the lowest of 2,542,372 shares of the Company’s common stock, 4% of the number of shares of the Company’s common stock outstanding on the first day of the fiscal year and an amount determined by its board of directors. The Company’s employees, officers, directors, consultants and advisors are eligible to receive awards under the 2013 Plan. However, incentive stock options may only be granted to employees of the Company.

Annual increases under the evergreen provisions of the 2013 Plan have resulted in the addition of an aggregate of approximately 5,454,000 additional shares to the 2013 Plan, including for 2017, an increase of approximately 1,429,000 shares, or 4% of the total number of shares of the Company's common stock outstanding as of January 1, 2017. As of September 30, 2017, the Company had approximately 2,124,000 shares available for grant under the 2013 Plan.

In April 2016, the board of directors adopted the ESPP pursuant to which the Company may sell up to an aggregate of 1,000,000 shares of common stock. The ESPP was approved by the Company’s stockholders in June 2016. The ESPP allows eligible employees to purchase common stock at a price per share equal to 85% of the lower of the fair market value of the common stock at the beginning or end of each six month offering period during the term of the ESPP. The first offering period began in September 2016.

A summary of the stock option activity, weighted average exercise prices, options outstanding and exercisable as of September 30, 2017 is as follows:



	Common Stock Options		Weighted Average Exercise Price

Outstanding, December 31, 2016	3,359		$	39.92
Granted	1,294		$	4.25
Exercised	(20	)	$	1.64
Expired or forfeited	(995	)	$	38.79
Outstanding, September 30, 2017	3,638		$	27.74

Options exercisable at September 30, 2017			1,839
Weighted average grant date fair value (per share) of options granted during the period			$	2.95

As of September 30, 2017, there were approximately 3,354,000 options outstanding, net of estimated forfeitures that had vested or are expected to vest. The weighted average exercise price of these options was $28.48 per option; the weighted average remaining contractual life of these options was 7.5 years; and the aggregate intrinsic value of these options was approximately $0.1 million.

A summary of the stock options outstanding and exercisable as of September 30, 2017 is as follows:



			As of September 30, 2017
			Options Outstanding			Options Exercisable
Range of Exercise Prices	Total Options Outstanding		Weighted Average Remaining Life (Years)	Weighted Average Exercise Price		Number Exercisable		Weighted Average Exercise Price

$0.12-$10.03	1,364		8.8	$	4.63	194		$	6.93
$10.04-$20.00	204		5.7	$	13.52	147		$	13.63
$20.01-$30.00	127		6.1	$	25.13	122		$	25.14
$30.01-$40.00	836		5.8	$	32.81	759		$	32.89
$40.01-$55.00	727		7.8	$	46.47	431		$	46.11
$55.01-$73.22	380		8.3	$	72.18	186		$	71.13
	3,638		7.6	$	27.74	1,839		$	35.07

Aggregate Intrinsic Value	$	114				$	95

Cash proceeds from, and the aggregate intrinsic value of, stock options exercised during the three and nine months ended September 30, 2017 and 2016, respectively, were as follows:



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

Cash proceeds from options exercised	$	—	$	1,591	$	33	$	5,398
Aggregate intrinsic value of options exercised	$	—	$	5,185	$	43	$	12,607

In connection with stock option awards granted to employees, the Company recognized approximately $2.3 million and $5.6 million in share-based compensation expense during the three months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. In connection with stock option awards granted to employees, the Company recognized approximately $10.3 million and $17.3 million in share-based compensation expense during the nine months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. As of September 30, 2017, there were approximately $15.0 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option awards granted to employees, which are expected to be recognized over a remaining weighted average period of 2.2 years.

In connection with stock option awards granted to consultants, the Company recognized approximately $0.1 million and $0.6 million in share-based compensation expense during the three months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. In connection with stock option awards granted to consultants, the Company recognized approximately $0.3 million and $1.5 million in share-based compensation expense during the nine months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. As of September 30, 2017, there were approximately $0.2 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option awards granted to consultants, which are expected to be recognized over a remaining weighted average period of 1.9 years.

The following table presents a summary of the Company’s outstanding RSU awards granted as of September 30, 2017:



	Restricted Stock Units		Weighted Average Grant-Date Fair Value

Outstanding, December 31, 2016	721		$	55.33
Awarded	248		$	4.42
Vested	(263	)	$	24.24
Forfeited	(288	)	$	50.17
Outstanding, September 30, 2017	418		$	42.85

As of September 30, 2017, there were approximately 262,000 RSUs outstanding, net of estimated forfeitures that are expected to vest. The weighted average fair value of these RSUs was $36.82 per share and the aggregate fair value of these RSUs was approximately $0.7 million.

In connection with RSUs granted to employees, the Company recognized approximately $0.9 million and $2.0 million in share-based compensation expense during the three months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. In connection with RSUs granted to employees, the Company recognized approximately $3.4 million and $6.1 million in share-based compensation expense during the nine months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. As of September 30, 2017, there were approximately $6.8 million of unrecognized compensation costs, net of estimated forfeitures, related to RSUs granted to employees, which are expected to be recognized over a remaining weighted average period of 2.2 years.

In connection with RSUs granted to consultants, the Company recognized approximately $0.1 million in share-based compensation expense during the three months ended September 30, 2017, net of expected forfeitures. In connection with RSUs granted to consultants, the Company recognized $0.3 million of share-based compensation expense during the nine months ended September 30, 2017, net of expected forfeitures. There were no RSUs granted to consultants during the three and nine months ended September 30, 2016. As of September 30, 2017, there were approximately $0.1 million of unrecognized compensation costs, net of estimated forfeitures, related to RSUs granted to consultants, which are expected to be recognized over a remaining weighted average period of 1.9 years.

In connection with the ESPP made available to employees, the Company recognized a de minimis amount of share-based compensation expense during the three months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. In connection with the ESPP made available to employees, the Company recognized $0.1 million and a de minimis amount of share-based compensation expense during the nine months ended September 30, 2017 and 2016, respectively, net of expected forfeitures. As of September 30, 2017, there was a de minimis amount of unrecognized compensation costs, net of estimated forfeitures, related to the ESPP, which are expected to be recognized over 0.5 years. There were 11,612 and 16,358 shares of common stock issued under the ESPP during the three and nine months ended September 30, 2017. Cash proceeds from ESPP purchases were $25 thousand and $38 thousand during the three and nine months ended September 30, 2017. There were no shares of common stock issued under the ESPP plan during the three and nine months ended September 30, 2016, as the first offering period under the ESPP commenced in September 2016. As of September 30, 2017, 983,642 shares were available for future purchases under the ESPP.

Property and Equipment

9 Months Ended

Sep. 30, 2017

Property, Plant and Equipment [Abstract]

Property and Equipment

10. Property and Equipment

Property and equipment as of September 30, 2017 and December 31, 2016 were as follows:



	Useful Life (Years)	September 30, 2017			December 31, 2016

Manufacturing and clinical equipment	7 - 10	$	412		$	617
Computer, software and other office equipment	5	1,711			1,711
Furniture and fixtures	1 - 7	774			774
Leasehold improvements	1 - 5	1,835			1,835
		4,732			4,937
Accumulated depreciation		(3,430		)	(1,656		)
Property and equipment, net		$	1,302		$	3,281

For the three and nine months ended September 30, 2017, depreciation expense was $0.6 million and $2.0 million, respectively. For the three and nine months ended September 30, 2016, depreciation expense was $0.2 million and $0.5 million, respectively.

Commitments and Contingencies

9 Months Ended

Sep. 30, 2017

Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies

11. Commitments and Contingencies

Under various agreements, the Company may be required to pay royalties and make milestone payments. These agreements include the following:

•

Under a license agreement with Archemix with respect to pharmaceutical products comprised of or derived from anti-C5 aptamers, for each anti-C5 aptamer product that the Company may develop under the agreement, including Zimura, the Company is obligated to make future payments to Archemix of up to an aggregate of $57.5 million if the Company achieves specified development, clinical and regulatory milestones and up to an aggregate of $22.5 million if the Company achieves specified commercial milestones. The Company is also obligated to pay Archemix a double-digit percentage of specified non-royalty payments the Company may receive from any sublicensee of the Company’s rights under this license agreement. No royalties are payable to Archemix under this license agreement.

•

Under the Company’s divestiture agreement with OSI (Eyetech), Inc., which agreement is now held by OSI Pharmaceuticals, LLC., or OSI Pharmaceuticals, a subsidiary of Astellas US, LLC, for rights to particular anti-PDGF aptamers, including Fovista, the Company is obligated to pay to OSI Pharmaceuticals future one-time payments of $12.0 million in the aggregate upon marketing approval in the United States and the European Union of a covered anti-PDGF product. The Company is also obligated to pay to OSI Pharmaceuticals a royalty at a low single-digit percentage of net sales of any covered anti-PDGF product the Company successfully commercializes.

•

Under a license agreement with Archemix Corp., or Archemix, with respect to pharmaceutical products comprised of or derived from any anti-PDGF aptamer, the Company is obligated to make future payments to Archemix of up to an aggregate of $14.0 million if the Company achieves specified clinical and regulatory milestones with respect to Fovista, up to an aggregate of $3.0 million if the Company achieves specified commercial milestones with respect to Fovista and, for each other anti-PDGF aptamer product that it may develop under the agreement, up to an aggregate of approximately $18.8 million if the Company achieves specified clinical and regulatory milestones and up to an aggregate of $3.0 million if the Company achieves specified commercial milestones. No royalties are payable to Archemix under this license agreement.

•

Under a license, manufacturing and supply agreement with Nektar for specified pegylation reagents used to manufacture Fovista, the Company was obligated to make future payments to Nektar of up to an aggregate of $6.5 million if the Company achieved specified clinical and regulatory milestones, and an additional payment of $3.0 million if the Company achieved a specified commercial milestone with respect to Fovista. The Company was obligated to pay Nektar tiered royalties at low to mid-single-digit percentages of net sales of any licensed product the Company successfully commercializes, with the royalty percentage determined by the Company’s level of licensed product sales, the extent of patent coverage for the licensed product and whether the Company has granted a third-party commercialization rights to the licensed product. In June 2014, the Company paid Nektar $19.8 million in connection with its entry into the Novartis Agreement. On October 27, 2017, Nektar and the Company agreed to terminate the license, manufacturing and supply agreement effective immediately. Neither party incurred any additional costs in connection with the termination of the license, manufacturing and supply agreement.


•	Under the Novo Agreement, with respect to Fovista, the Company will be obligated to pay Novo A/S a mid-single-digit percentage royalty based on worldwide sales of Fovista. See "Note 6—Financing Agreement with Novo A/S" above for further information about Novo Agreement.

The Company also has letter agreements with certain employees that require the funding of a specific level of payments, if certain events, such as a termination of employment in connection with a change in control or termination of employment by the employee for good reason or by the Company without cause, occur.

In addition, in the course of normal business operations, the Company has agreements with contract service providers to assist in the performance of the Company’s research and development and manufacturing activities. Expenditures to CROs and CMOs represent significant costs in clinical development. Subject to required notice periods and the Company’s obligations under binding purchase orders, the Company can elect to discontinue the work under these agreements at any time.

Legal Proceedings

On January 11, 2017, a putative class action lawsuit was filed against the Company and certain of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Frank Micholle v. Ophthotech Corporation, et al., No. 1:17-cv-00210. The complaint purports to be brought on behalf of shareholders who purchased the Company's common stock between May 11, 2015 and December 12, 2016. The complaint generally alleges that the Company and certain of its officers violated Sections 10(b) and/or 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by making allegedly false and/or misleading statements concerning the prospects of the Company's Phase 3 trials for Fovista in combination with anti-VEGF drugs for the treatment of wet AMD. The complaint seeks equitable and/or injunctive relief, unspecified damages, attorneys’ fees, and other costs.

On March 9, 2017, a second putative class action lawsuit was filed against the Company and the same group of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Wasson v. Ophthotech Corporation, et al., No. 1:17-cv-01758. The complaint purports to be brought on behalf of shareholders who purchased the Company’s common stock between May 11, 2015 and December 9, 2016. The allegations made in the complaint are similar to those made in the Micholle complaint. Putative lead plaintiffs in the Micholle action have moved to consolidate the Micholle and Wasson actions.

The Company denies any allegations of wrongdoing and intends to vigorously defend against these lawsuits. The Company is unable, however, to predict the outcome of these matters at this time. Moreover, any conclusion of these matters in a manner adverse to the Company and for which it incurs substantial costs or damages not covered by the Company's directors’ and officers’ liability insurance would have a material adverse effect on its financial condition and business. In addition, the litigation could adversely impact the Company's reputation and divert management’s attention and resources from other priorities, including the execution of business plans and strategies that are important to the Company's ability to grow its business, any of which could have a material adverse effect on the Company's business.

On May 30, 2017, a shareholder derivative action was filed against the members of the Company’s Board of Directors in the United States District Court for the Southern District of New York, captioned Etelmendorf v. Bolte, et al., No. 1:17-cv-04042. The complaint alleges that defendants breached their fiduciary duties to the Company by causing or permitting the Company to make allegedly false and/or misleading statements concerning the prospects of the Company’s Phase 3 trials for Fovista in combination with anti-VEGF drugs for the treatment of wet AMD, and by approving certain executive compensation. The complaint also alleges that defendants were unjustly enriched as a result of the alleged conduct. The complaint purports to seek unspecified damages on behalf of the Company, as well as an order directing the Company to reform and comply with its governance obligations, attorneys’ fees, and other costs. On October 17, 2017, the plaintiff filed a notice of voluntary dismissal without prejudice for this action.

Restructuring Activities

9 Months Ended

Sep. 30, 2017

Restructuring and Related Activities [Abstract]

Restructuring Activities

12. Restructuring Activities

In December 2016, the Company announced its intention to implement a reduction in personnel to focus on an updated business plan. In January 2017, the Board of Directors approved a plan to implement a reduction in personnel involving approximately 80% of the Company’s workforce based on the number of employees at the time the plan was approved. During the first nine months of 2017, the Company's workforce has been reduced by 112 employees in connection with the reduction in personnel and through natural attrition. The Company expects to complete the reduction in personnel during the fourth quarter of 2017. In connection with such reduction in personnel, the Company estimates that it will incur approximately $13.3 million of pre-tax charges through the third quarter of 2017, of which approximately $12.4 million in the aggregate is expected to result in cash expenditures. These pre-tax charges relate to (a) expected severance, stock compensation and other employee costs of approximately $11.2 million and (b) expected lease termination costs of approximately $2.1 million. As of September 30, 2017, the Company's cash expenditures related to such reduction in personnel totaled $8.7 million.

In connection with the reduction in personnel, the Company recognized approximately $1.4 million and $11.9 million of severance, stock compensation and other employee costs for the three and nine months ended September 30, 2017, of which $0.9 million and $6.8 million were recorded in "Research and development" expense and $0.5 million and $5.1 million were recorded in "General and administrative" expense in the Company's Statements of Operations.

As of September 30, 2017, the Company's accrual balance for severance and benefit costs was $2.3 million which was recorded in "Accounts payable and accrued expenses" in the Company's Balance Sheet. The severance and other employee cost accruals as of September 30, 2017 are expected to be paid through to the first half of 2018.

The following is a reconciliation of the severance-related accrual activity for the nine months ended September 30, 2017:



	Accrued Severance and Other Employee Costs
Beginning Balance	$	—
Accrued restructuring expenses	11,076
Payments	(8,727		)
Ending Balance	$	2,349

In January 2017, the Company issued a notice of termination under the Lease Agreement, dated as of September 30, 2007, between the Company and One Penn Plaza LLC, as previously supplemented and amended (as so supplemented and amended, the “Lease”) for office space at One Penn Plaza in New York, New York. The termination of the Lease triggered an early termination payment by the Company of approximately $0.9 million and will be effective in January 2018, through which time the Company will be responsible for paying continuing rental fees, as well as taxes, operating expenses and utility and other charges related to the leased premises. On November 1, 2017, the Company and One Penn Plaza LLC executed a further amendment to the Lease. See "—Note 13—Subsequent Events" for a description of the further lease amendment. Payments under the further lease amendment will not constitute restructuring charges.

On January 26, 2017, the Company issued a notice of termination under the Sublease Agreement between the Company and Otsuka America Pharmaceutical, Inc. (the “Sublease”) for office space at One University Square, Princeton, New Jersey. The termination of the Sublease triggered an early termination payment by the Company of approximately $1.2 million and will be effective in February 2018, through which time the Company will be responsible for paying continuing rental fees, as well as taxes, operating expenses and utility and other charges related to the subleased premises.

On January 26, 2017, the Company issued a notice of termination under its Office Lease Agreement between the Company and PSN Partners, L.P. (the “Office Lease”) for office space in Palmer Square in Princeton, New Jersey. The termination of the Office Lease did not trigger any early termination payment and is effective October 2017.

During January 2017, the Company made the early termination payments as described above and recognized $2.1 million of additional facilities costs which were recorded in "General and administrative" expense in the Company's Statements of Operations.

Subsequent Event	9 Months Ended
Subsequent Event	Sep. 30, 2017
Subsequent Events [Abstract]
Subsequent Event	13. Subsequent Events On October 24, 2017, Novartis delivered to the Company a notice of termination of the Novartis Agreement dated October 23, 2017. The termination notice was effective immediately. On October 27, 2017, Nektar and the Company agreed to terminate the License, Manufacturing and Supply Agreement with respect to PEG reagent used to manufacture Fovista. The termination was effective immediately. On November 1, 2017, the Company entered into a fifth amendment of lease (the “Fifth Lease Amendment”) with One Penn Plaza LLC, as landlord, amending, effective as of October 1, 2017, the Lease Agreement, dated as of September 30, 2007, between the Company and One Penn Plaza LLC, as previously supplemented and amended (as so supplemented and amended, the “Lease”). The Fifth Lease Amendment provides for a different, smaller premises to be made available to the Company under the Lease at One Penn Plaza, New York, NY, resulting in total office space available under the Lease of approximately 13,500 square feet. The Company intends to use the office space for corporate and clinical operations. The new office space will be available to the Company following the completion of construction being performed by the landlord, which is expected to be completed prior to the end of January 2018. Notwithstanding the Company's prior termination notice under the Lease, the Lease is now scheduled to expire at the end of 2018. The incremental rental fees, which includes utilities costs, over the extended term of the Lease from January through December 2018 are expected to be approximately $650 thousand. The Company is also liable for taxes, and other charges related to the leased premises. The Company previously provided the landlord with a letter of credit in an amount of approximately $138 thousand to secure the Company’s obligations under the Lease.

Summary of Significant Accounting Policies (Policies)

9 Months Ended

Sep. 30, 2017

Accounting Policies [Abstract]

Basis of Presentation

Segment and geographic information

Use of Estimates

Cash and Cash Equivalents

Available for Sale Securities

Revenue Recognition

Collaboration Revenue

Concentration of Credit Risk

Concentration of Suppliers

Foreign Currency Translation

Financial Instruments

Property and Equipment

Research and Development


•	external research and development expenses incurred under arrangements with third parties, such as contract research organizations (“CROs”) and other vendors and contract manufacturing organizations (“CMOs”) for the production of drug substance and drug product; and


•	employee-related expenses, including salaries, benefits and share-based compensation expense.

Income Taxes

Share-Based Compensation

Stock Options



	Three months ended September 30,		Nine months ended September 30,
	2017	2016	2017	2016
Expected common stock price volatility	82%	71%	81%	71%
Risk-free interest rate	1.95%-2.06%	1.14% -1.32%	1.82%-2.38%	1.14%-1.92%
Expected term of options (years)	6.1	6.0	6.1	6.1
Expected dividend yield	—	—	—	—

RSUs

The Company estimates the fair value of RSUs granted to employees using the closing market price of the Company’s common stock on the date of grant.

ESPP

Recent Accounting Pronouncements

Summary of Significant Accounting Policies (Tables)

9 Months Ended

Sep. 30, 2017

Accounting Policies [Abstract]

Summary of the components of collaboration revenue

Below is a summary of the components of the Company’s collaboration revenue for the three and nine months ended September 30, 2017 and 2016:



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

License revenue	$	152,912	$	—	$	152,912	$	22,937
Research and development activity revenue	52,864		1,664		56,180		8,089
API transfer revenue	754		—		754		14,545
Joint operating committee revenue	124		4		131		16
Total collaboration revenue	$	206,654	$	1,668	$	209,977	$	45,587

Schedule of weighted-average assumptions used to estimate grant date fair value of stock options



	Three months ended September 30,		Nine months ended September 30,
	2017	2016	2017	2016
Expected common stock price volatility	82%	71%	81%	71%
Risk-free interest rate	1.95%-2.06%	1.14% -1.32%	1.82%-2.38%	1.14%-1.92%
Expected term of options (years)	6.1	6.0	6.1	6.1
Expected dividend yield	—	—	—	—

Schedule of share-based compensation expense



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

Research and development	$	1,840	$	5,664	$	8,887	$	16,732
General and administrative	1,571		2,584		5,576		8,157
Total	$	3,411	$	8,248	$	14,463	$	24,889

Net Loss Per Common Share (Tables)

9 Months Ended

Sep. 30, 2017

Earnings Per Share [Abstract]

Schedule of computation of basic and diluted net income (loss) per common share

The following table sets forth the computation of basic and diluted net income (loss) per common share for the periods indicated:



	Three months ended September 30,					Nine months ended September 30,
	2017		2016			2017		2016

Basic and diluted net income (loss) per common share calculation:
Net income (loss)	$	189,073	$	(60,891	)	$	123,747	$	(127,137	)
Weighted average common shares outstanding - basic	35,971		35,594			35,878		35,415
Plus: net effect of dilutive stock options and unvested restricted stock units	76		—			106		—
Weighted average common shares outstanding - dilutive	36,047		35,594			35,984		35,415
Net income (loss) per share of common stock - basic	$	5.26	$	(1.71	)	$	3.45	$	(3.59	)
Net income (loss) per share of common stock - diluted	$	5.25	$	(1.71	)	$	3.44	$	(3.59	)

Schedule of potentially dilutive securities that have been excluded from the computations of diluted weighted average common shares outstanding

The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding for the periods presented, as they would be anti-dilutive:



	Three months ended September 30,		Nine months ended September 30,
	2017	2016	2017	2016

Stock options outstanding	3,640	3,423	3,813	3,423
Restricted stock units	269	721	332	721
Total	3,909	4,144	4,145	4,144

Cash, Cash Equivalents and Available for Sale Securities (Tables)

9 Months Ended

Sep. 30, 2017

Cash, Cash Equivalents, and Short-term Investments [Abstract]

Summary of available for sale securities, including carrying value and estimated fair values

Available for sale securities, including carrying value and estimated fair values as of December 31, 2016, are summarized as follows:



	As of December 31, 2016
	Cost		Unrealized Gains		Unrealized Losses			Fair Value

U.S. Treasury securities	$	120,288	$	6	$	(33	)	$	120,261
Corporate debt securities	35,114		—		(27		)	35,087
Total	$	155,402	$	6	$	(60	)	$	155,348

Schedule of changes in accumulated other comprehensive income (loss) associated with the unrealized gain (loss) on available for sale securities

The changes in accumulated other comprehensive income (loss) associated with the unrealized gain (loss) on available for sale securities during the three and nine months ended September 30, 2017 and September 30, 2016 were as follows:



	Three months ended September 30,						Nine months ended September 30,
	2017			2016			2017			2016

Beginning balance	$	(186	)	$	(118	)	$	(212	)	$	(473	)
Current period changes in fair value before reclassifications, net of tax	186			(41		)	212			314
Amounts reclassified from accumulated other comprehensive income (loss), net of tax	—			—			—			—
Total other comprehensive income (loss)	186			(41		)	212			314
Ending balance	$	—		$	(159	)	$	—		$	(159	)

- Definition

Tabular disclosure of available-for-sale securities which includes, but is not limited to, changes in the cost basis and fair value, fair value and gross unrealized gain (loss), fair values by type of security, contractual maturity and classification, amortized cost basis, contracts to acquire securities to be accounted for as available-for-sale, debt maturities, transfers to trading, change in net unrealized holding gain (loss) net of tax, continuous unrealized loss position fair value, aggregate losses qualitative disclosures, other than temporary impairment (OTTI) losses or other disclosures related to available for sale securities.

+ References + Details

Licensing and Commercialization Agreement with Novartis Pharma AG (Tables)

9 Months Ended

Sep. 30, 2017

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Summary of the components of the Company's collaboration revenue

Below is a summary of the components of the Company’s collaboration revenue for the three and nine months ended September 30, 2017 and September 30, 2016:



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

License revenue	$	152,912	$	—	$	152,912	$	22,937
Research and development activity revenue	52,864		1,664		56,180		8,089
API transfer revenue	754		—		754		14,545
Joint operating committee revenue	124		4		131		16
Total collaboration revenue	$	206,654	$	1,668	$	209,977	$	45,587

Fair Value Measurements (Tables)

9 Months Ended

Sep. 30, 2017

Fair Value Disclosures [Abstract]

Schedule of assets and liabilities that are measured at fair value on a recurring basis



	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)

Assets
Investments in money market funds*	$	167,052	$	—	$	—



	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)

Assets
Investments in money market funds*	$	108,096	$	—	$	—
Investments in U.S. Treasury securities	$	120,261	$	—	$	—
Investments in Corporate debt securities	$	—	$	35,087	$	—

Stock Option and Compensation Plans (Tables)

9 Months Ended

Sep. 30, 2017

Disclosure of Compensation Related Costs, Share-based Payments [Abstract]

Summary of the stock option activity including weighted average exercise prices, options outstanding and exercisable

A summary of the stock option activity, weighted average exercise prices, options outstanding and exercisable as of September 30, 2017 is as follows:



	Common Stock Options		Weighted Average Exercise Price

Outstanding, December 31, 2016	3,359		$	39.92
Granted	1,294		$	4.25
Exercised	(20	)	$	1.64
Expired or forfeited	(995	)	$	38.79
Outstanding, September 30, 2017	3,638		$	27.74

Options exercisable at September 30, 2017			1,839
Weighted average grant date fair value (per share) of options granted during the period			$	2.95

Summary of the stock options outstanding and exercisable by range of exercise prices

A summary of the stock options outstanding and exercisable as of September 30, 2017 is as follows:



			As of September 30, 2017
			Options Outstanding			Options Exercisable
Range of Exercise Prices	Total Options Outstanding		Weighted Average Remaining Life (Years)	Weighted Average Exercise Price		Number Exercisable		Weighted Average Exercise Price

$0.12-$10.03	1,364		8.8	$	4.63	194		$	6.93
$10.04-$20.00	204		5.7	$	13.52	147		$	13.63
$20.01-$30.00	127		6.1	$	25.13	122		$	25.14
$30.01-$40.00	836		5.8	$	32.81	759		$	32.89
$40.01-$55.00	727		7.8	$	46.47	431		$	46.11
$55.01-$73.22	380		8.3	$	72.18	186		$	71.13
	3,638		7.6	$	27.74	1,839		$	35.07

Aggregate Intrinsic Value	$	114				$	95

Schedule of cash proceeds from, and the aggregate intrinsic value of, stock options exercised

Cash proceeds from, and the aggregate intrinsic value of, stock options exercised during the three and nine months ended September 30, 2017 and 2016, respectively, were as follows:



	Three months ended September 30,				Nine months ended September 30,
	2017		2016		2017		2016

Cash proceeds from options exercised	$	—	$	1,591	$	33	$	5,398
Aggregate intrinsic value of options exercised	$	—	$	5,185	$	43	$	12,607

Summary of outstanding RSU awards granted

The following table presents a summary of the Company’s outstanding RSU awards granted as of September 30, 2017:



	Restricted Stock Units		Weighted Average Grant-Date Fair Value

Outstanding, December 31, 2016	721		$	55.33
Awarded	248		$	4.42
Vested	(263	)	$	24.24
Forfeited	(288	)	$	50.17
Outstanding, September 30, 2017	418		$	42.85

Property and Equipment (Tables)

9 Months Ended

Sep. 30, 2017

Property, Plant and Equipment [Abstract]

Schedule of property and equipment

Property and equipment as of September 30, 2017 and December 31, 2016 were as follows:



	Useful Life (Years)	September 30, 2017			December 31, 2016

Manufacturing and clinical equipment	7 - 10	$	412		$	617
Computer, software and other office equipment	5	1,711			1,711
Furniture and fixtures	1 - 7	774			774
Leasehold improvements	1 - 5	1,835			1,835
		4,732			4,937
Accumulated depreciation		(3,430		)	(1,656		)
Property and equipment, net		$	1,302		$	3,281

Restructuring Activities (Tables)

9 Months Ended

Sep. 30, 2017

Restructuring and Related Activities [Abstract]

Reconciliation of severance-related accrual activity

The following is a reconciliation of the severance-related accrual activity for the nine months ended September 30, 2017:



	Accrued Severance and Other Employee Costs
Beginning Balance	$	—
Accrued restructuring expenses	11,076
Payments	(8,727		)
Ending Balance	$	2,349

Business (Details)	9 Months Ended
Business (Details)	Sep. 30, 2017 candidate	Dec. 31, 2016 trial
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Number of product candidates \| candidate	2
Phase Three Trials Failure to Meet Primary Endpoint
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Number of trials completed		2
Phase Three Clinical Trial
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Number of trials completed		3

Summary of Significant Accounting Policies - Segment and Geographic Information (Details)	9 Months Ended
	Sep. 30, 2017 segment
Accounting Policies [Abstract]
Number of operating segments	1
Number of reportable segments	1

Summary of Significant Accounting Policies - Cash and Cash Equivalents (Details) $ in Millions	Sep. 30, 2017 USD ($)
Accounting Policies [Abstract]
Cash and cash equivalents	$ 180.2

Summary of Significant Accounting Policies - Collaboration Revenue (Details) - USD ($) $ in Thousands	1 Months Ended			3 Months Ended		9 Months Ended
	Aug. 31, 2017	Jul. 31, 2017	May 31, 2014	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Upfront payments received	$ 16,900
Components of collaboration revenue
Total collaboration revenue				$ 206,654	$ 1,668	$ 209,977	$ 45,587
License revenue
Components of collaboration revenue
Total collaboration revenue				152,912	0	152,912	22,937
Research and development activity revenue
Components of collaboration revenue
Total collaboration revenue				52,864	1,664	56,180	8,089
API transfer revenue
Components of collaboration revenue
Total collaboration revenue				754	0	754	14,545
Joint operating committee revenue
Components of collaboration revenue
Total collaboration revenue				$ 124	$ 4	$ 131	$ 16
Novartis Pharma AG \| Licensing and Commercialization Agreement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Upfront payments received		$ 189,800	$ 200,000

Summary of Significant Accounting Policies - Property and Equipment (Details)	9 Months Ended
	Sep. 30, 2017
Minimum
Property and equipment
Estimated useful lives	3 years
Maximum
Property and equipment
Estimated useful lives	10 years

Summary of Significant Accounting Policies - Share-Based Compensation (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016	Apr. 30, 2016
Share-Based Compensation
Expected dividend yield (as a percent)			0.00%
Share-based compensation expense	$ 3,411	$ 8,248	$ 14,463	$ 24,889
Research and development
Share-Based Compensation
Share-based compensation expense	1,840	5,664	8,887	16,732
General and administrative
Share-Based Compensation
Share-based compensation expense	$ 1,571	$ 2,584	$ 5,576	$ 8,157
Employee Stock Option
Share-Based Compensation
Expected common stock price volatility	82.00%	71.00%	81.00%	71.00%
Risk free interest rate, minimum	1.95%	1.14%	1.82%	1.14%
Risk free interest rate, maximum	2.06%	1.32%	2.38%	1.92%
Expected term of options (years)	6 years 1 month 6 days	6 years	6 years 1 month 6 days	6 years 1 month 6 days
Expected dividend yield (as a percent)	0.00%	0.00%	0.00%	0.00%
ESPP
Share-Based Compensation
Share-based compensation expense			$ 100
ESPP \| Maximum
Share-Based Compensation
Number of shares reserved for issuance under the Plan (in shares)					1,000,000

Net Loss Per Common Share (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Basic and diluted net income (loss) per common share calculation
Net income (loss)	$ 189,073	$ (60,891)	$ 123,747	$ (127,137)
Weighted average common shares outstanding - basic (in shares)	35,971	35,594	35,878	35,415
Plus: net effect of dilutive stock options and unvested restricted stock units (in shares)	76	0	106	0
Weighted average common shares outstanding - dilutive (in shares)	36,047	35,594	35,984	35,415
Net income (loss) per share of common stock - basic (in dollars per share)	$ 5.26	$ (1.71)	$ 3.45	$ (3.59)
Net income (loss) per share of common stock - diluted (in dollars per share)	$ 5.25	$ (1.71)	$ 3.44	$ (3.59)
Anti-dilutive securities
Total (in shares)	3,909	4,144	4,145	4,144
Stock options outstanding
Anti-dilutive securities
Total (in shares)	3,640	3,423	3,813	3,423
Restricted stock units
Anti-dilutive securities
Total (in shares)	269	721	332	721

Cash, Cash Equivalents and Available for Sale Securities (Details) - USD ($) $ in Thousands	Sep. 30, 2017	Dec. 31, 2016
Cash, Cash Equivalents, and Short-term Investments [Abstract]
Cash	$ 13,200	$ 25,800
Investments in money market funds, U.S. Treasury securities and certain short-term investment-grade corporate debt securities with original maturities of 90 days or less	167,100	108,100
Fair Value	$ 0	$ 155,348

Cash, Cash Equivalents and Available for Sale Securities - Summary of Available for Sale Securities (Details) - USD ($) $ in Thousands	Sep. 30, 2017	Dec. 31, 2016
Available for sale securities, including carrying value and estimated fair values
Cost		$ 155,402
Unrealized Gains		6
Unrealized Losses		(60)
Fair Value	$ 0	155,348
U.S. Treasury securities
Available for sale securities, including carrying value and estimated fair values
Cost		120,288
Unrealized Gains		6
Unrealized Losses		(33)
Fair Value		120,261
Corporate debt securities
Available for sale securities, including carrying value and estimated fair values
Cost		35,114
Unrealized Gains		0
Unrealized Losses		(27)
Fair Value		$ 35,087

Cash, Cash Equivalents and Available for Sale Securities - Schedule of Changes in Accumulated Other Comprehensive Income (Loss) (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Changes in accumulated other comprehensive income (loss)
Beginning balance			$ (94,618)
Current period changes in fair value before reclassifications, net of tax	$ 186	$ (41)	212	$ 314
Amounts reclassified from accumulated other comprehensive income (loss), net of tax	0	0	0	0
Other comprehensive income (loss)	186	(41)	212	314
Ending balance	43,875		43,875
Accumulated Other Comprehensive Income (Loss)
Changes in accumulated other comprehensive income (loss)
Beginning balance	(186)	(118)	(212)	(473)
Ending balance	$ 0	$ (159)	$ 0	$ (159)

Licensing and Commercialization Agreement with Novartis Pharma AG (Details) - USD ($) $ in Thousands	1 Months Ended						3 Months Ended		9 Months Ended		22 Months Ended
	Aug. 31, 2017	Jul. 31, 2017	Jun. 30, 2016	Mar. 31, 2015	Sep. 30, 2014	May 31, 2014	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016	Jun. 30, 2016
Agreement
Upfront fees received	$ 16,900
Total collaboration revenue							$ 206,654	$ 1,668	$ 209,977	$ 45,587
License revenue
Agreement
Total collaboration revenue							152,912	0	152,912	22,937
Research and development activity revenue
Agreement
Total collaboration revenue							52,864	1,664	56,180	8,089
API transfer revenue
Agreement
Total collaboration revenue							754	0	754	14,545
Joint operating committee revenue
Agreement
Total collaboration revenue							124	$ 4	131	$ 16
Licensing and Commercialization Agreement \| Novartis Pharma AG
Agreement
Upfront fees received		$ 189,800				$ 200,000
Licensing and Commercialization Agreement \| Novartis Pharma AG \| Research and development activity revenue \| Up-front payment
Agreement
Deferred revenue							$ 200,000		$ 200,000
Licensing and Commercialization Agreement \| Novartis Pharma AG \| Achievement of specified patient enrollment milestones
Agreement
Enrollment-based milestone revenue achieved			$ 30,000	$ 50,000	$ 50,000						$ 130,000

Financing Agreement with Novo A/S (Details)	1 Months Ended			19 Months Ended
Financing Agreement with Novo A/S (Details)	Nov. 30, 2014 USD ($)	Jan. 31, 2014 USD ($)	May 31, 2013 USD ($) tranche purchase	Nov. 30, 2014 USD ($)	Sep. 30, 2017 USD ($)	Dec. 31, 2016 USD ($)
Agreement
Royalty purchase liability					$ 125,000,000	$ 125,000,000
Novo A/S \| Novo Agreement \| Fovista, Fovista-Related Products, and Other Products
Agreement
Number of tranches in financing \| tranche			3
Aggregate royalty rights			$ 125,000,000
Number of separate purchases provided for in financing agreement \| purchase			3
Proceeds from royalty purchase agreement	$ 41,700,000	$ 41,700,000	$ 41,700,000	$ 125,000,000
Royalty purchase liability			$ 125,000,000

Income Taxes (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Income Taxes (Details) - USD ($) $ in Thousands	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Operating Loss Carryforwards [Line Items]
Income tax provision (benefit)	$ 194	$ 70	$ 196	$ (158)
Income (loss) from before income tax provision (benefit)	189,267	(60,821)	123,943	$ (127,295)
Reduction in deferred tax assets and corresponding valuation allowance	5,900	$ 3,800
Domestic Tax Authority
Operating Loss Carryforwards [Line Items]
Refunds claimed on amended tax returns	$ (7,900)		$ (7,900)

Fair Value Measurements (Details) - USD ($)	Sep. 30, 2017	Dec. 31, 2016
Fair Value Measurements
Investments in U.S. Treasury securities and Corporate debt securities	$ 0	$ 155,348,000
Transfer of assets from Level 1 to Level 2 of the fair value measurement hierarchy	0
Transfer of assets from Level 2 to Level 1 of the fair value measurement hierarchy	0
Fair Value, Measurements, Recurring \| Quoted prices in active markets for identical assets (Level 1) \| Money market funds
Fair Value Measurements
Investments in money market funds, U.S. Treasury securities and corporate debt securities with maturities less than 90 days	167,052,000	108,096,000
Fair Value, Measurements, Recurring \| Quoted prices in active markets for identical assets (Level 1) \| U.S. Treasury securities
Fair Value Measurements
Investments in U.S. Treasury securities and Corporate debt securities		120,261,000
Fair Value, Measurements, Recurring \| Quoted prices in active markets for identical assets (Level 1) \| Corporate debt securities
Fair Value Measurements
Investments in U.S. Treasury securities and Corporate debt securities		0
Fair Value, Measurements, Recurring \| Significant other observable inputs (Level 2) \| Money market funds
Fair Value Measurements
Investments in money market funds, U.S. Treasury securities and corporate debt securities with maturities less than 90 days	0	0
Fair Value, Measurements, Recurring \| Significant other observable inputs (Level 2) \| U.S. Treasury securities
Fair Value Measurements
Investments in U.S. Treasury securities and Corporate debt securities		0
Fair Value, Measurements, Recurring \| Significant other observable inputs (Level 2) \| Corporate debt securities
Fair Value Measurements
Investments in U.S. Treasury securities and Corporate debt securities		35,087,000
Fair Value, Measurements, Recurring \| Significant unobservable inputs (Level 3) \| Money market funds
Fair Value Measurements
Investments in money market funds, U.S. Treasury securities and corporate debt securities with maturities less than 90 days	$ 0	0
Fair Value, Measurements, Recurring \| Significant unobservable inputs (Level 3) \| U.S. Treasury securities
Fair Value Measurements
Investments in U.S. Treasury securities and Corporate debt securities		0
Fair Value, Measurements, Recurring \| Significant unobservable inputs (Level 3) \| Corporate debt securities
Fair Value Measurements
Investments in U.S. Treasury securities and Corporate debt securities		$ 0

Stock Option and Compensation Plans - Stock Incentive Plans and Stock Option Activity Narrative (Details) - USD ($) $ / shares in Units, $ in Millions	1 Months Ended			9 Months Ended
	Jan. 31, 2017	Apr. 30, 2016	Aug. 31, 2013	Sep. 30, 2017
Options outstanding, net of estimated forfeitures, vested or expected to vest
Options outstanding (in shares)				3,354,000
Weighted average exercise price (in dollars per share)				$ 28.48
Weighted average remaining contractual life				7 years 6 months 8 days
Aggregate intrinsic value				$ 0.1
ESPP
Stock Option and Compensation Plans
Number of shares of common stock available for issuance under the Plan (in shares)				983,642
Purchase price of common stock as percentage of fair market value		85.00%
Maximum \| ESPP
Stock Option and Compensation Plans
Number of shares reserved for issuance under the Plan (in shares)		1,000,000
2013 Plan
Stock Option and Compensation Plans
Number of shares of common stock available for issuance under the Plan (in shares)				2,124,000
Annual increase in shares reserved for issuance under the Plan (in shares)			2,542,372
Annual increase in shares reserved for issuance under the Plan (as a percent)	4.00%		4.00%
Increase in number of shares available under the Plan (in shares)	1,429,000			5,454,000
2013 Plan \| Maximum
Stock Option and Compensation Plans
Number of shares reserved for issuance under the Plan (in shares)			3,359,641
2007 Plan
Stock Option and Compensation Plans
Number of shares of common stock available for issuance under the Plan (in shares)			739,317

Stock Option and Compensation Plans - Summary of Stock Option Activity (Details) shares in Thousands	9 Months Ended
	Sep. 30, 2017 $ / shares shares
Common Stock Options
Outstanding, beginning of period (in shares) \| shares	3,359
Granted (in shares) \| shares	1,294
Exercised (in shares) \| shares	(20)
Expired or forfeited (in shares) \| shares	(995)
Outstanding, end of period (in shares) \| shares	3,638
Weighted Average Exercise Price
Outstanding, beginning of period (in dollars per share) \| $ / shares	$ 39.92
Granted (in dollars per share) \| $ / shares	4.25
Exercised (in dollars per share) \| $ / shares	1.64
Expired or forfeited (in dollars per share) \| $ / shares	38.79
Outstanding, end of period (in dollars per share) \| $ / shares	$ 27.74
Options exercisable at end of period (in shares) \| shares	1,839
Weighted average grant date fair value (per share) of options granted during the period (in dollars per share) \| $ / shares	$ 2.95

Stock Option and Compensation Plans - Options Outstanding and Exercisable by Exercise Price and Options Exercised (Details) $ / shares in Units, shares in Thousands, $ in Thousands	9 Months Ended
	Sep. 30, 2017 USD ($) $ / shares shares
Options Outstanding
Total Options Outstanding (in shares) \| shares	3,638
Weighted Average Remaining Life (Years)	7 years 6 months 28 days
Weighted Average Exercise Price (in dollars per share)	$ 27.74
Aggregate Intrinsic Value of Options Outstanding \| $	$ 114
Options Exercisable
Number Exercisable (in shares) \| shares	1,839
Weighted Average Exercise Price (in dollars per share)	$ 35.07
Aggregate Intrinsic Value of Options Exercisable \| $	$ 95
$0.12-$10.03
Options Outstanding
Total Options Outstanding (in shares) \| shares	1,364
Weighted Average Remaining Life (Years)	8 years 9 months 13 days
Weighted Average Exercise Price (in dollars per share)	$ 4.63
Options Exercisable
Number Exercisable (in shares) \| shares	194
Weighted Average Exercise Price (in dollars per share)	$ 6.93
Exercise prices, low end of range (in dollars per share)	0.12
Exercise prices, high end of range (in dollars per share)	$ 10.03
$10.04-$20.00
Options Outstanding
Total Options Outstanding (in shares) \| shares	204
Weighted Average Remaining Life (Years)	5 years 8 months 23 days
Weighted Average Exercise Price (in dollars per share)	$ 13.52
Options Exercisable
Number Exercisable (in shares) \| shares	147
Weighted Average Exercise Price (in dollars per share)	$ 13.63
Exercise prices, low end of range (in dollars per share)	10.04
Exercise prices, high end of range (in dollars per share)	$ 20.00
$20.01-$30.00
Options Outstanding
Total Options Outstanding (in shares) \| shares	127
Weighted Average Remaining Life (Years)	6 years 1 month 12 days
Weighted Average Exercise Price (in dollars per share)	$ 25.13
Options Exercisable
Number Exercisable (in shares) \| shares	122
Weighted Average Exercise Price (in dollars per share)	$ 25.14
Exercise prices, low end of range (in dollars per share)	20.01
Exercise prices, high end of range (in dollars per share)	$ 30.00
$30.01-$40.00
Options Outstanding
Total Options Outstanding (in shares) \| shares	836
Weighted Average Remaining Life (Years)	5 years 9 months 11 days
Weighted Average Exercise Price (in dollars per share)	$ 32.81
Options Exercisable
Number Exercisable (in shares) \| shares	759
Weighted Average Exercise Price (in dollars per share)	$ 32.89
Exercise prices, low end of range (in dollars per share)	30.01
Exercise prices, high end of range (in dollars per share)	$ 40.00
$40.01-$55.00
Options Outstanding
Total Options Outstanding (in shares) \| shares	727
Weighted Average Remaining Life (Years)	7 years 9 months 9 days
Weighted Average Exercise Price (in dollars per share)	$ 46.47
Options Exercisable
Number Exercisable (in shares) \| shares	431
Weighted Average Exercise Price (in dollars per share)	$ 46.11
Exercise prices, low end of range (in dollars per share)	40.01
Exercise prices, high end of range (in dollars per share)	$ 55.00
$55.01-$73.22
Options Outstanding
Total Options Outstanding (in shares) \| shares	380
Weighted Average Remaining Life (Years)	8 years 3 months 11 days
Weighted Average Exercise Price (in dollars per share)	$ 72.18
Options Exercisable
Number Exercisable (in shares) \| shares	186
Weighted Average Exercise Price (in dollars per share)	$ 71.13
Exercise prices, low end of range (in dollars per share)	55.01
Exercise prices, high end of range (in dollars per share)	$ 73.22

Stock Option and Compensation Plans - Schedule of Cash Proceeds From and Aggregate Intrinsic Value of Stock Options Exercised (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Disclosure of Compensation Related Costs, Share-based Payments [Abstract]
Cash proceeds from options exercised	$ 0	$ 1,591	$ 33	$ 5,398
Aggregate intrinsic value of options exercised	$ 0	$ 5,185	$ 43	$ 12,607

Stock Option and Compensation Plans - Stock Compensation Expense (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Stock Option and Compensation Plans
Share-based compensation expense	$ 3,411	$ 8,248	$ 14,463	$ 24,889
Stock options \| Employees
Stock Option and Compensation Plans
Share-based compensation expense	2,300	5,600	10,300	17,300
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures	15,000		$ 15,000
Expected weighted average period to recognize share-based compensation costs			2 years 2 months 20 days
Non-employee Options \| Consultants
Stock Option and Compensation Plans
Share-based compensation expense	100	600	$ 300	1,500
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures	200		$ 200
Expected weighted average period to recognize share-based compensation costs			1 year 11 months 4 days
Restricted stock units
Stock Option and Compensation Plans
Share-based compensation expense	$ 100
RSUs expected to vest (in shares)	262,000		262,000
Weighted average fair value of RSUs expected to vest (in dollars per share)	$ 36.82		$ 36.82
Aggregate intrinsic value of RSUs expected to vest	$ 700		$ 700
Number of shares granted (in shares)			248,000
Restricted stock units \| Employees
Stock Option and Compensation Plans
Share-based compensation expense	900	$ 2,000	$ 3,400	$ 6,100
Expected weighted average period to recognize share-based compensation costs			2 years 2 months 18 days
Unrecognized compensation costs, net of estimated forfeitures	6,800		$ 6,800
Restricted stock units \| Consultants
Stock Option and Compensation Plans
Share-based compensation expense			$ 300
Expected weighted average period to recognize share-based compensation costs			1 year 11 months 6 days
Unrecognized compensation costs, net of estimated forfeitures	$ 100		$ 100
Number of shares granted (in shares)		0		0
ESPP
Stock Option and Compensation Plans
Share-based compensation expense			$ 100
Expected weighted average period to recognize share-based compensation costs			6 months
Shares of common stock issued under plan (in shares)	11,612		16,358
Proceeds from employee stock purchase plan	$ 25		$ 38
Number of shares available for future purchases under the plan (in shares)	983,642		983,642

Stock Option and Compensation Plans - Summary of Outstanding RSU Awards Granted (Details) - Restricted stock units shares in Thousands	9 Months Ended
	Sep. 30, 2017 $ / shares shares
Restricted Stock Units
Outstanding, beginning of period (in shares) \| shares	721
Awarded (in shares) \| shares	248
Vested (in shares) \| shares	(263)
Forfeited (in shares) \| shares	(288)
Outstanding, end of period (in shares) \| shares	418
Weighted Average Grant-Date Fair Value
Outstanding, beginning of period (in dollars per share) \| $ / shares	$ 55.33
Awarded (in dollars per share) \| $ / shares	4.42
Vested (in dollars per share) \| $ / shares	24.24
Forfeited (in dollars per share) \| $ / shares	50.17
Outstanding, end of period (in dollars per share) \| $ / shares	$ 42.85

Property and Equipment (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Property and Equipment (Details) - USD ($) $ in Thousands	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016	Dec. 31, 2016
Property and Equipment
Property and equipment, gross	$ 4,732		$ 4,732		$ 4,937
Accumulated depreciation	(3,430)		(3,430)		(1,656)
Property and equipment, net	1,302		1,302		3,281
Depreciation expense	600	$ 200	$ 1,979	$ 539
Minimum
Property and Equipment
Useful life			3 years
Maximum
Property and Equipment
Useful life			10 years
Manufacturing and clinical equipment
Property and Equipment
Property and equipment, gross	412		$ 412		617
Manufacturing and clinical equipment \| Minimum
Property and Equipment
Useful life			7 years
Manufacturing and clinical equipment \| Maximum
Property and Equipment
Useful life			10 years
Computer, software and other office equipment
Property and Equipment
Useful life			5 years
Property and equipment, gross	1,711		$ 1,711		1,711
Furniture and fixtures
Property and Equipment
Property and equipment, gross	774		$ 774		774
Furniture and fixtures \| Minimum
Property and Equipment
Useful life			1 year
Furniture and fixtures \| Maximum
Property and Equipment
Useful life			7 years
Leasehold improvements
Property and Equipment
Property and equipment, gross	$ 1,835		$ 1,835		$ 1,835
Leasehold improvements \| Minimum
Property and Equipment
Useful life			1 year
Leasehold improvements \| Maximum
Property and Equipment
Useful life			5 years

Commitments and Contingencies (Details) - USD ($)	1 Months Ended
Commitments and Contingencies (Details) - USD ($)	Jun. 30, 2014	Sep. 30, 2017
Acquisition agreement \| OSI Pharmaceuticals \| PDGF Licensed Products
Commitments and contingencies
Amount to be paid on achievement of milestone		$ 12,000,000
License Agreements \| Archemix \| PDGF Licensed Products
Commitments and contingencies
Royalty payable		0
License Agreements \| Archemix \| Fovista \| Achievement of specified clinical and regulatory milestones \| Maximum
Commitments and contingencies
Amount to be paid on achievement of milestone		14,000,000
License Agreements \| Archemix \| Fovista \| Achievement of specified commercial milestones \| Maximum
Commitments and contingencies
Amount to be paid on achievement of milestone		3,000,000
License Agreements \| Archemix \| Other anti-PDGF aptamer \| Achievement of specified clinical and regulatory milestones \| Maximum
Commitments and contingencies
Amount to be paid on achievement of milestone		18,800,000
License Agreements \| Archemix \| Other anti-PDGF aptamer \| Achievement of specified commercial milestones \| Maximum
Commitments and contingencies
Amount to be paid on achievement of milestone		3,000,000
License Agreements \| Archemix \| C5 Licensed Product
Commitments and contingencies
Royalty payable		0
License Agreements \| Archemix \| C5 Licensed Product \| Achievement of specified commercial milestones \| Maximum
Commitments and contingencies
Amount to be paid on achievement of milestone		22,500,000
License Agreements \| Archemix \| C5 Licensed Product \| Achievement of specified development, clinical and regulatory milestones \| Maximum
Commitments and contingencies
Amount to be paid on achievement of milestone		57,500,000
Supply Agreement \| Nektar
Commitments and contingencies
Payment upon entry into Novartis Agreement	$ 19,800,000
Supply Agreement \| Nektar \| Fovista \| Achievement of specified clinical and regulatory milestones \| Maximum
Commitments and contingencies
Amount to be paid on achievement of milestone		6,500,000
Supply Agreement \| Nektar \| Fovista \| Achievement of specified commercial milestones
Commitments and contingencies
Amount to be paid on achievement of milestone		$ 3,000,000

Restructuring Activities (Details) $ in Thousands		1 Months Ended	3 Months Ended	9 Months Ended
Restructuring Activities (Details) $ in Thousands	Jan. 26, 2017 USD ($)	Jan. 31, 2017 USD ($)	Sep. 30, 2017 USD ($) employee	Sep. 30, 2017 USD ($) employee	Dec. 31, 2016 USD ($)
Restructuring Cost and Reserve [Line Items]
Expected costs			$ 13,300	$ 13,300
Costs expected to result in future cash expenditures				$ 12,400
Severance and other employee costs
Restructuring Cost and Reserve [Line Items]
Percent of personnel expected to be effected by reduction in workforce		80.00%
Number of employee positions eliminated in reduction of personnel \| employee			112	112
Expected costs			$ 11,200	$ 11,200
Cash expenditures related to reduction in personnel				8,727
Costs related to reduction in personnel			1,400	11,900
Accrual balance for restructuring activity			2,349	2,349	$ 0
Accrued restructuring expenses				11,076
Severance and other employee costs \| Research and development
Restructuring Cost and Reserve [Line Items]
Costs related to reduction in personnel			900	6,800
Severance and other employee costs \| General and administrative
Restructuring Cost and Reserve [Line Items]
Costs related to reduction in personnel			500	5,100
Lease termination costs
Restructuring Cost and Reserve [Line Items]
Expected costs			$ 2,100	$ 2,100
Lease termination costs \| One Penn Plaza LLC
Restructuring Cost and Reserve [Line Items]
Accrued restructuring expenses	$ 900
Lease termination costs \| Otsuka America Pharmaceutical, Inc.
Restructuring Cost and Reserve [Line Items]
Accrued restructuring expenses	$ 1,200
Lease termination costs \| General and administrative
Restructuring Cost and Reserve [Line Items]
Accrued restructuring expenses		$ 2,100

Restructuring Activities - Reconciliation of Severance-related Accrual Activity (Details) - Accrued Severance and Other Employee Costs $ in Thousands	9 Months Ended
	Sep. 30, 2017 USD ($)
Restructuring Reserve [Roll Forward]
Beginning Balance	$ 0
Accrued restructuring expenses	11,076
Payments	(8,727)
Ending Balance	$ 2,349

Subsequent Event (Details) - Subsequent Event $ in Thousands	Nov. 01, 2017 USD ($) ft²
Subsequent Event [Line Items]
Office space (in square feet) \| ft²	13,500
Incremental rent fees	$ 650
Landlord letter of credit	$ 138