View Filing Data

Document and Entity Information - shares	3 Months Ended
Document and Entity Information - shares	Mar. 31, 2015	May 01, 2015
Document and Entity Information
Entity Registrant Name	Ophthotech Corp.
Entity Central Index Key	0001410939
Document Type	10-Q/A
Document Period End Date	Mar. 31, 2015
Amendment Flag	true
Amendment Description	We are filing this Amendment No. 1 to our Quarterly Report on Form 10-Q/A (the "Amended Report"), which was originally filed with the Securities and Exchange Commission on Form 10-Q on May 11, 2015 (the "Original Report" or "Quarterly Report on Form 10-Q"), to reflect a restatement of our financial statements. As discussed in Note 13 to our financial statements, subsequent to the issuance of the Original Report, we and the Audit Committee of our Board of Directors concluded that we should restate our balance sheets as of December 31, 2014 and March 31, 2015, and our statements of operations, comprehensive income (loss), and cash flows for the three months ended March 31, 2015 to correct the following errors: • In the second quarter of 2014, we recorded an income tax benefit by reducing a portion of our valuation allowance against our gross deferred tax assets. In determining the amount of the valuation allowance release, we considered anticipated 2015 tax losses which would generate a refund of a portion of federal income taxes paid in 2014. We have now determined that, as a matter of accounting principle, the net deferred tax asset recorded on our balance sheets was overstated as of December 31, 2014 and March 31, 2015 and the income tax provision on our statement of operations was understated for the three months ended March 31, 2015. Our cash position and operating expenses were not affected. For the reasons discussed above, we are filing this Amended Report in order to amend our financial statements and the following items in Part I of the Original Report to the extent necessary to correct the errors discussed above and make corresponding revisions to our financial information cited elsewhere in this Amended Report: • Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations • Item 4. Controls and Procedures In addition, in accordance with applicable Securities and Exchange Commission rules, this Amended Report includes updated certifications from our chief executive officer and chief financial officer as Exhibits 31.3, 31.4, 32.3 and 32.4. Other than minor conforming revisions made to the financial information contained in Part II Item 1A. Risk Factors, none of the remaining Items of our Original Report are amended hereby. Items that have not been amended have been omitted herefrom. In order to preserve the nature and character of the disclosures set forth in the Original Report, except as expressly noted above, this Amended Report speaks as of the date of the filing of the Original Report, May 11, 2015, and we have not updated the disclosures in the Amended Report to speak as of a later date. All information contained in this Amended Report is subject to updating and supplementing as provided in our reports filed with the Securities and Exchange Commission subsequent to the date of the Original Report.
Current Fiscal Year End Date	--12-31
Entity Current Reporting Status	Yes
Entity Filer Category	Large Accelerated Filer
Entity Common Stock, Shares Outstanding		34,298,236
Document Fiscal Year Focus	2015
Document Fiscal Period Focus	Q1

Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2015	Dec. 31, 2014
Current assets
Cash and cash equivalents	$ 39,568	$ 39,814
Due from Novartis Pharma, AG	50,249	960
Available for sale securities	393,718	423,746
Prepaid expenses and other current assets	8,900	8,812
Deferred tax assets	70	50
Total current assets	492,505	473,382
Property and equipment, net	1,563	1,555
Deferred tax assets, non-current	4,412	4,467
Security deposits	282	282
Other Assets	79	100
Total assets	498,841	479,786
Current liabilities
Accounts payable and accrued expenses	5,757	8,707
Accrued research and development expenses	8,103	7,918
Deferred revenue	6,637	3,206
Total current liabilities	20,497	19,831
Deferred revenue, long-term	211,309	206,418
Royalty purchase liability	125,000	125,000
Total liabilities	356,806	351,249
Stockholders' equity
Preferred stock - $0.001 par value, 5,000,000 shares authorized, no shares issued or outstanding
Common stock - $0.001 par value, 200,000,000 shares authorized, 34,245,730 and 33,994,520 shares issued and outstanding at March 31, 2015 and December 31, 2014, respectively	34	34
Additional paid-in capital	435,204	428,390
Accumulated deficit	(293,186)	(299,822)
Accumulated other comprehensive loss	(17)	(65)
Total stockholders' equity	142,035	128,537
Total liabilities and stockholders' equity	$ 498,841	$ 479,786

Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2015	Dec. 31, 2014
Balance Sheets
Preferred stock, par value (in dollars per share)	$ 0.001	$ 0.001
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, shares authorized	200,000,000	200,000,000
Common stock, shares issued	34,245,730	33,994,520
Common stock, shares outstanding	34,245,730	33,994,520

Statements of Operations - USD ($) shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Statements of Operations
Collaboration Revenue	$ 41,678
Costs and expenses:
Research and development	24,557	$ 14,377
General and administrative	9,584	6,349
Total costs and expenses	34,141	20,726
Income (loss) from operations	7,537	(20,726)
Interest income	73	44
Income (loss) before income tax provision	7,610	(20,682)
Income tax provision	974
Net income (loss)	$ 6,636	$ (20,682)
Net income (loss) per common share:
Basic (in dollars per share)	$ 0.19	$ (0.64)
Diluted ( in dollars per share)	$ 0.19	$ (0.64)
Weighted average common shares outstanding:
Basic (in shares)	34,154	32,282
Diluted (in shares)	35,239	32,282

Statements Comprehensive Income (Loss) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Statements Comprehensive Income (Loss)
Net income (loss)	$ 6,636	$ (20,682)
Other comprehensive income:
Unrealized gain on available for sale securities, net of taxes	48	18
Other comprehensive income	48	18
Comprehensive income (loss)	$ 6,684	$ (20,664)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References + Details

Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
Statements of Cash Flows - USD ($) $ in Thousands	Mar. 31, 2015	Mar. 31, 2014
Operating Activities
Net income (loss)	$ 6,636	$ (20,682)
Adjustments to reconcile net income(loss) to net cash used in operating activities
Depreciation	56	12
Amortization of premium and discounts on investment securities	991
Share-based compensation	5,054	2,729
Changes in operating assets and liabilities:
Due from Novartis Pharma, AG	(49,289)
Prepaid expense and other current assets	(88)	1,535
Accrued interest receivable	(447)
Other assets	21
Accrued research and development expenses	185	317
Accounts payable and accrued expenses	(2,950)	(57)
Deferred revenue	8,322
Net cash used in operating activities	(31,509)	(16,146)
Investing Activities
Purchase of marketable securities	(144,433)	(224,247)
Maturities of marketable securities	174,000
Purchase of property and equipment	(64)	(728)
Net cash provided by (used in) investing activities	29,503	(224,975)
Financing Activities
Proceeds from stock option/warrant exercises	1,760	11
Proceeds from follow-on public offering, net		55,409
Proceeds from royalty purchase agreement		41,667
Net cash provided by financing activities	1,760	97,087
Net change in cash and cash equivalents	(246)	(144,034)
Cash and cash equivalents
Beginning of period	39,814	210,596
End of period	39,568	66,562
Supplemental disclosures of non-cash information related to investing activities
Change in unrealized gains in available for sale securities, net of tax	$ 48	$ 18

Business	3 Months Ended
Business	Mar. 31, 2015
Business
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 diseases of the back of the eye, with a focus on developing therapeutics for age-related macular degeneration, or AMD. The Company’s most advanced product candidate is Fovista, which is in Phase 3 clinical development for use in combination with anti-VEGF therapy that represent the current standard of care for the treatment of wet AMD. The Company has completed one Phase 1 and one Phase 2b clinical trial of Fovista administered in combination with the anti-VEGF drug Lucentis (ranibizumab). The Company is also developing its product candidate Zimura for the treatment of patients with geographic atrophy, a form of dry AMD; in combination with anti-VEGF therapy for the treatment of polypoidal choroidal vasculopathy, a specific type of wet AMD, in patients who do not respond adequately to treatment with anti-VEGF monotherapy or for whom anti-VEGF monotherapy fails; and, potentially, in combination with anti-VEGF therapy and Fovista for the treatment of anti-VEGF resistant wet AMD patients who are believed to have complement mediated inflammation.

Summary of Significant Accounting Policies

3 Months Ended

Mar. 31, 2015

Summary of Significant Accounting Policies

Summary of Significant Accounting Policies

2.Summary of Significant Accounting Policies

Restatement of Previously Issued Financial Statements

As discussed in Note 13 to the Company’s financial statements, subsequent to the issuance of the Company’s Annual Report on Form 10-K for the year ended December 31, 2014, the Company and the Audit Committee of its Board of Directors concluded that the Company should restate its balance sheets as of December 31, 2014 and March 31, 2015, and its statements of operations, comprehensive income (loss), and cash flows for the three months ended March 31, 2015 to correct the following. In the second quarter of 2014, the Company recorded an income tax benefit by reducing a portion of its valuation allowance against its gross deferred tax assets. In determining the amount of the valuation allowance release, the Company considered anticipated 2015 tax losses which would generate a refund of a portion of federal income taxes paid in 2014. The Company has now determined that, as a matter of accounting principle, the net deferred tax asset recorded on its balance sheets was overstated as of December 31, 2014 and March 31, 2015 and the income tax provision on its statement of operations was understated for the period ended March 31, 2015. The Company’s cash position and operating expenses are not affected.

Basis of Presentation

The accompanying unaudited financial information as of March 31, 2015 and for the three months ended March 31, 2015 and 2014 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, 2014 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 2014, as originally filed with the SEC on March 2, 2015, and as amended on Form 10-K/A and filed with the SEC on July 28, 2015.

In the opinion of management, the unaudited financial information as of March 31, 2015 and for the three months ended March 31, 2015 and 2014, reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results or operations and cash flows. The results of operations for the three months ended March 31, 2015 and 2014 are not necessarily indicative of the operating results for the full fiscal year or any future period.

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 share-based compensation and accounting for research and development costs. 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.

Available for Sale Securities

The Company considers securities with original maturities of greater than 90 days to be available for sale securities. 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

Prior to 2014, the Company had not generated any revenue. In May 2014, the Company received an up-front payment of $200.0 million in connection with its licensing and commercialization agreement with Novartis Pharma, AG (the “Novartis Agreement”), which has not been recorded as revenue due to certain contingencies associated with the payment. In each of September 2014 and March 2015, the Company achieved a $50.0 million enrollment-based milestone, or $100.0 million in the aggregate, under the Novartis Agreement. The Company recognized revenue of approximately $41.7 million during the three months ended March 31, 2015, which primarily related to the $50.0 million milestone it achieved in March 2015. The balance of the milestone payment was recorded as deferred revenue. The Company uses the relative selling price method to allocate arrangement consideration to the Company’s performance obligations under the Novartis Agreement. Below is a summary of the components of the Company’s collaboration revenue for the three months ended March 31, 2015 and 2014:

	Three months ended March 31,
	2015		2014

License revenue	$	38,083	$	—
Research and development activity revenue	3,585		—
Joint operating committee revenue	10		—

Total collaboration revenue	$	41,678	$	—

The Company did not recognize any revenue related to its obligation to supply active pharmaceutical ingredient, or API, for Fovista to Novartis during the three months ended March 31, 2015.

In the future, the Company may generate additional revenues from a combination of product sales and license fees, milestone payments, research and development activity-related payments, payments for manufactured material and royalties in connection with the Novartis Agreement. The terms of this agreement and other potential collaboration or commercialization agreements the Company may enter into generally contain multiple elements, or deliverables, which may include (i) licenses, or options to obtain licenses, to certain of the Company’s technology and products, (ii) research and development activities to be performed on behalf of the collaborative partner, and (iii) in certain cases, services in connection with the manufacturing of pre-clinical, clinical or commercial material. Payments to the Company under these arrangements typically include one or more of the following: non-refundable, up-front license fees; option exercise fees; funding of research and/or development efforts; milestone payments; payments for manufactured material; and royalties on future product sales.

When evaluating multiple element arrangements, the Company considers whether the deliverables under the arrangement represent separate units of accounting. This evaluation requires subjective determinations and requires management to make judgments about the individual deliverables and whether such deliverables are separable from the other aspects of the contractual relationship. In determining the units of accounting, management evaluates certain criteria, including whether the deliverables have standalone value, based on the relevant facts and circumstances for each arrangement. The consideration received is allocated among the separate units of accounting using the relative selling price method, and the applicable revenue recognition criteria are applied to each of the separate units.

The Company determines the estimated selling price for deliverables within each agreement using vendor-specific objective evidence (“VSOE”) of selling price, if available, third-party evidence (“TPE”) of selling price if VSOE is not available, or best estimate of selling price (“BESP”) if neither VSOE nor TPE is available. Determining the best estimate of selling price for a deliverable requires significant judgment. The Company uses BESP to estimate the selling price for licenses to the Company’s proprietary technology, since the Company often does not have VSOE or TPE of selling price for these deliverables. In those circumstances where the Company utilizes BESP to determine the estimated selling price of a license to the Company’s proprietary technology, the Company considers market conditions as well as entity-specific factors, including those factors contemplated in negotiating the agreements as well as internally developed models that include assumptions related to the market opportunity, estimated development costs, probability of success and the time needed to commercialize a product candidate that is subject to the license. In validating the Company’s BESP, the Company evaluates whether changes in the key assumptions used to determine the BESP will have a significant effect on the allocation of arrangement consideration among multiple deliverables.

When management believes the license to its intellectual property and products has stand-alone value, the Company generally recognizes revenue attributed to the license upon delivery. When management believes such a license does not have stand-alone value from the other deliverables to be provided in the arrangement, the Company generally recognizes revenue attributed to the license on a straight-line basis over the Company’s contractual or estimated performance period, which is typically the term of the Company’s research and development obligations. If management cannot reasonably estimate when the Company’s performance obligation ends, then revenue is deferred until management can reasonably estimate when the performance obligation ends. The periods over which revenue should be recognized are subject to estimates by management and may change over the course of the research and development agreement. Such a change could have a material impact on the amount of revenue the Company records in future periods.

At the inception of arrangements that include milestone payments, the Company evaluates whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether (a) the consideration is commensurate with either (1) the entity’s performance to achieve the milestone, or (2) the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone, (b) the consideration relates solely to past performance, and (c) the consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. The Company evaluates factors such as the scientific, regulatory, commercial and other risks that must be overcome to achieve the respective milestone, the level of effort and investment required to achieve the respective milestone and whether the milestone consideration is reasonable relative to all deliverables and payment terms in the arrangement in making this assessment.

The Company aggregates its milestones into three categories: (i) clinical and development milestones, (ii) regulatory milestones, and (iii) commercial milestones. Clinical and development milestones are typically achieved when a product candidate advances into a defined phase of clinical research or completes such phase or when a contractually specified clinical trial enrollment target is attained. Regulatory milestones are typically achieved upon acceptance of the submission of an application for marketing approval for a product candidate or upon approval to market the product candidate by the U.S. Food and Drug Administration (the “FDA”) or other regulatory authorities. For example, a milestone payment may be due to the Company upon the FDA’s acceptance of a New Drug Application (“NDA”). Commercial milestones are typically achieved when an approved pharmaceutical product reaches certain defined levels of net sales by the licensee, such as when a product first achieves global sales or annual sales of a specified amount.

Revenues from clinical and development and regulatory milestone payments, if the milestones are deemed substantive and the milestone payments are nonrefundable, are recognized upon successful accomplishment of the milestones. With regard to the Novartis Agreement, the Company has concluded that the clinical and development milestones and certain regulatory milestones are not substantive and that the regulatory approval milestones are substantive. Milestone payments received that are not considered substantive are included in the allocable arrangement consideration and are recognized as revenue in proportion to the relative-selling price allocation established at the inception of the arrangement. Revenues from commercial milestone payments are accounted for as royalties and are recorded as revenue upon achievement of the milestone, assuming all other revenue recognition criteria are met.

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 U.S. Treasury securities with maturities of 90 days or less and in money market funds that invest primarily in U.S. Treasury securities.

The Company’s available for sale securities are also invested in U.S. Treasury securities. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents and available for sale securities.

Foreign Currency Translation

The Company considers the U.S. dollar to be its functional currency. Expenses 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

The carrying amounts of the Company’s financial instruments, which include cash and cash equivalents, available for sale securities, accounts payable and accrued expenses approximate their respective fair value due to their short maturities.

Property and Equipment

Property and equipment, which consists mainly of manufacturing and clinical equipment, furniture and fixtures, computers and other 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 rather than a method based on when the payments are made.

Research and Development

Research and development expenses primarily consist of costs associated with the manufacturing, development and clinical testing of Fovista, an anti-platelet derived growth factor (“PDGF”) aptamer that the Company is developing for use in combination with anti-VEGF therapy for treatment of wet AMD, and Zimura, an inhibitor of complement factor C5 that the Company is developing for the treatment of patients with geographic atrophy, a form of dry AMD; in combination with anti-VEGF therapy for the treatment of polypoidal choroidal vasculopathy, a specific type of wet AMD, in patients who do not respond adequately to treatment with anti-VEGF monotherapy or for whom anti-VEGF monotherapy fails; and, potentially, in combination with anti-VEGF therapy and Fovista for the treatment of anti-VEGF resistant wet AMD patients who are believed to have complement mediated inflammation. 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 for the production of drug substance and drug product; and

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

Research and development costs 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 costs 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.

The Company anticipates that it will continue to incur significant research and development expenses in connection with conducting its pivotal Phase 3 clinical program for Fovista and if such trials are successful, seeking marketing approval for Fovista. In addition, the Company expects that it will incur significant expenses related to the completion of process development and manufacturing scale-up activities for Fovista. The Company also anticipates that its research and development expenses will increase as a result of its plan to initiate a Phase 2/3 clinical trial to evaluate the safety and efficacy of Zimura in patients with geographic atrophy in the second half of 2015; the very small Phase 2 trial recently commenced investigating Zimura in combination with anti-VEGF therapy for the treatment of polypoidal choroidal vasculopathy, a specific type of wet AMD, in patients who do not respond adequately to treatment with anti-VEGF monotherapy or for whom anti-VEGF monotherapy fails; and the planned Phase 2 study of Zimura in combination with anti-VEGF therapy and Fovista for the treatment of anti-VEGF resistant wet AMD patients who are believed to have complement mediated inflammation. In addition, the Company also expects its research and development expenses to increase as it further evaluates the potential benefit of Fovista in wet AMD, when administered in combination with anti-VEGF therapy, and in other ophthalmic diseases and conditions with unmet medical need. The Company expects these expenses to increase as patient enrollment increases in these trials.

Income Taxes

The Company utilizes the liability method of accounting for deferred income taxes, as set forth in ASC 740-10, Income Taxes—Overall. Under this method, deferred tax liabilities and assets are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities. The Company incurred U.S. federal net operating losses in each year from its inception in 2007 through 2013 and as such, all prior tax years since 2007 remain subject to potential tax examination as the utilization of net operating losses from prior years opens the relevant year to potential audit.

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 and restricted stock units (“RSUs”). 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 forfeitures.

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 option grants that do not vest immediately upon grant 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.

Restricted Stock Units

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

Share-based compensation expense includes expenses related to stock options and restricted stock units granted to employees, non-employee directors and consultants, and has been reported in the Company’s Statements of Operations as follows:

	Three months ended March 31,
	2015		2014

Research and development	$	3,115	$	1,662
General and administrative	1,939		1,067

Total	$	5,054	$	2,729

Recent Accounting Pronouncements

In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-09, “Revenue from Contracts with Customers (Topic 606),” (“ASU 2014-09”). ASU 2014-09 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. ASU 2014-09 is effective for public entities for annual reporting periods beginning after December 15, 2016 and interimperiods within those periods. Early adoption is not permitted. Companies may use either a full retrospective or a modified retrospective approach to adopt ASU 2014-09. The Company is currently assessing the impact that adopting this new accounting guidance will have on its financial statements and footnote disclosures.

In August 2014, the FASB issued ASU 2014-15, Presentation of Financial Statements-Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, which defines management’s responsibility to assess an entity’s ability to continue as a going concern, and to provide related footnote disclosures if there is substantial doubt about its ability to continue as a going concern. The pronouncement is effective for annual reporting periods ending after December 15, 2016 with early adoption permitted. The adoption of this guidance is not expected to have a significant impact on the Company’s financial statements.

Capitalization	3 Months Ended
Capitalization	Mar. 31, 2015
Capitalization
Capitalization	3.Capitalization On September 30, 2013, the Company closed its initial public offering of 8,740,000 shares of common stock at a price of $22.00 per share of common stock. The net proceeds to the Company were $175.6 million, after deducting underwriters’ discounts and commissions and other offering expenses. In connection with the closing of the IPO, all of the Company’s shares of redeemable convertible preferred stock outstanding at the time of the offering were automatically converted into 21,038,477 shares of common stock. On February 18, 2014, the Company closed a follow-on public offering of 2,628,571 shares of common stock at a public offering price of $31.50 per share of common stock. The Company sold 1,900,000 shares and 728,571 shares were sold by selling stockholders, including 342,857 shares sold by the selling stockholders upon the full exercise by the underwriters of their option to purchase additional shares in the follow-on public offering. The net proceeds to the Company were $55.4 million, after deducting underwriters’ discounts and commissions and other offering expenses. The Company did not receive any proceeds from the sale of shares by the selling stockholders in the follow-on public offering.

Net Income (Loss) Per Common Share

3 Months Ended

Mar. 31, 2015

Net Income (Loss) Per Common Share

Net Income (loss) Per Common Share

4.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, restricted stock units and warrants have been excluded from the calculation of diluted loss per common share because their effect would be anti-dilutive. Therefore, the weighted average shares used to calculate both basic and diluted loss per 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 March 31,
	2015 (Restated)		2014

Basic and diluted net income (loss) per common share calculation:
Net income (loss)	$	6,636	$	(20,682	)
Weighted average common shares outstanding - basic	34,154		32,282
Plus: effect of dilutive stock options, warrants and unvested restricted stock units	1,085		—

Weighted average common shares outstanding - dilutive	35,239		32,282

Net income (loss) per share of common stock - basic	$	0.19	$	(0.64	)

Net income (loss) per share of common stock - diluted	$	0.19	$	(0.64	)

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 March 31,
	2015	2014
Stock options	929	3,726
Warrants	—	88

Total	929	3,814

Cash, Cash Equivalents and Available for Sale Securities

3 Months Ended

Mar. 31, 2015

Cash, Cash Equivalents and Available for Sale Securities

Cash, Cash Equivalents and Available for Sale Securities

5.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 $5.3 million and $4.7 million at March 31, 2015 and

December 31, 2014, respectively. Cash and cash equivalents at March 31, 2015 and December 31, 2014 also included investments of $34.3 million and $35.1 million, respectively, in U.S. Treasury securities with original maturities of 90 days or less and investments in money market funds that invest in U.S. Treasury Securities.

The Company held available for sale securities with a fair value totaling $393.7 million and $423.7 million at March 31, 2015 and December 31, 2014, respectively. These available for sale securities consisted of U.S. Treasury securities and had maturities of greater than 90 days and less than one year. The Company evaluates securities with unrealized losses, if any, to determine whether such losses are other than temporary. The Company has determined that there were no other than temporary declines in fair values of its investments as of March 31, 2015. The Company classifies these securities as available for sale, however, the Company does not currently intend to sell its investments and the Company believes it is more likely than not that the Company will recover the carrying value of these investments.

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

	As of March 31, 2015
	Cost		Fair Value		Carrying Value		Unrealized Loss

U.S. Treasury securities - maturities < 1 year	$	393,747	$	393,718	$	393,718	$	(29	)
U.S. Treasury securities - maturities > 1 year	—		—		—		—

Total	$	393,747	$	393,718	$	393,718	$	(29	)

	As of December 31, 2014
	Cost		Fair Value		Carrying Value		Unrealized Loss

U.S. Treasury securities - maturities < 1 year	$	423,859	$	423,746	$	423,746	$	(113	)
U.S. Treasury securities - maturities > 1 year	$	—	$	—	$	—	$	—

Total	$	423,859	$	423,746	$	423,746	$	(113	)

The Company’s available for sale securities are reported at fair value on the Company’s balance sheet. Unrealized gains and losses are reported within accumulated other comprehensive income (loss) in the statements of comprehensive income (loss). The changes in accumulated other comprehensive loss associated with the unrealized loss on available for sale securities during the three months ended March 31, 2015 and March 31,2014 were as follows:

	Three months ended March 31,
	2015			2014

Beginning balance	$	(65	)	$	—
Current period changes in fair value, net of tax	48			18

Ending balance	$	(17	)	$	18

Licensing and Commercialization Agreement with Novartis Pharma AG	3 Months Ended
	Mar. 31, 2015
Licensing and Commercialization Agreement with Novartis Pharma AG
Licensing and Commercialization Agreement with Novartis Pharma AG	6.Licensing and Commercialization Agreement with Novartis Pharma AG On May 19, 2014, the Company entered into a licensing and commercialization agreement with Novartis Pharma AG. 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 active pharmaceutical ingredient, or API, supplied by the Company, standalone Fovista products and products combining Fovista with an anti-VEGF product 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 has agreed to use commercially reasonable efforts to complete its ongoing pivotal Phase 3 clinical program for Fovista and Novartis has agreed to use commercially reasonable efforts to develop a standalone Fovista product and a co-formulated product containing Fovista and an anti-VEGF product 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. Novartis has also granted the Company options, subject to specified limitations, and to the extent such rights are controlled by Novartis, to obtain exclusive rights from Novartis to develop and commercialize in the United States the co-formulated and pre-filled syringe products developed by Novartis. The Company and Novartis have each granted the other options, subject to specified limitations, to obtain access to study data from certain clinical trials of licensed products that the Company or Novartis may conduct, including for use by the other in regulatory filings in its territory. The Company has agreed to exclusively supply Novartis, and Novartis has agreed to exclusively purchase from the Company, its clinical and commercial requirements for the bulk API for Fovista for use in licensed products in the Novartis Territory. The Company has agreed not to commercialize any product comprising Fovista or any other anti-PDGF product in the ophthalmic field in the Novartis Territory. Novartis paid the Company a $200.0 million upfront fee upon execution of the Novartis Agreement. Novartis is also obligated to pay the Company up to an aggregate of $130.0 million if the Company achieves specified patient enrollment-based milestones for its Phase 3 clinical program for Fovista, of which, $50.0 million was received by the Company in October 2014 and $50.0 million was achieved in March 2015 and received in April 2015, and up to an aggregate of an additional $300.0 million upon achievement of specified regulatory approval milestones, including reimbursement approval, in certain countries in the Novartis Territory. In addition, Novartis has agreed to pay the Company up to an aggregate of an additional $400.0 million if Novartis achieves specified sales milestones in the Novartis Territory. Novartis also is obligated to pay the Company royalties with respect to standalone Fovista products and combination Fovista products that Novartis successfully commercializes. The Company will receive royalties at a mid-thirties percentage of net sales of standalone Fovista products and a royalty of approximately equal value for sales of combination Fovista products. Such royalties are subject to customary deductions, credits, and reductions for lack of patent coverage or market exclusivity. Novartis’s obligation to pay such royalties will continue on a licensed product-by-licensed product and country-by-country basis until Novartis’s last actual commercial sale of such licensed product in such country. The Company will continue to be responsible for royalties it owes to third parties on sales of Fovista products. Novartis has agreed to pay the Company’s manufacturing costs plus a specified percentage margin for supplies of the bulk API for Fovista that the Company supplies to Novartis. If the Company or Novartis exercises each of their respective rights to obtain access to study data from clinical trials conducted by the other party, the party exercising the option will be obligated to pay the other party’s associated past development costs and share with such other party any future associated development costs. If the Company exercises its option to obtain Novartis-controlled rights to develop, manufacture and commercialize any co-formulated Fovista product in the United States, the Company will be obligated to pay a specified percentage of Novartis’s associated past development costs and share with Novartis any future associated development costs. The Company and Novartis will also need to negotiate and agree on financial and other terms that would apply to such rights. If the Company exercises its option to obtain Novartis-controlled rights to develop and commercialize a pre-filled syringe product in the United States, the Company will be obligated to either enter into a supply agreement with Novartis under which the Company will pay Novartis its manufacturing cost plus a specified percentage margin for supplies of Fovista products in pre-filled syringes that Novartis supplies to the Company, or obtain supplies of products in pre-filled syringes from a third party manufacturer and pay Novartis a low single-digit percentage of the Company’s net sales of such products. The Company has retained control over the design and execution of its pivotal Phase 3 clinical program for Fovista and remains responsible for funding the costs of that program, subject to Novartis’s responsibility to provide Lucentis, an anti-VEGF agent to which Novartis has rights in the Novartis Territory, for use in the Phase 3 trials already initiated and in other Phase 2 and Phase 3 clinical trials in the Novartis Territory following the effective date of the Novartis Agreement. Novartis will have control over, and will be responsible for the costs of, all other clinical trials that may be required to obtain marketing approvals in the Novartis Territory for licensed products under the agreement. Novartis is also responsible for costs associated with co-formulation development, pre-filled syringe development and other development costs in the Novartis Territory, excluding regulatory filing fees in the European Union for the standalone Fovista product, for which the Company will be responsible. The Novartis Agreement, unless earlier terminated by the Company or Novartis, will expire upon the expiration of Novartis’s obligation to pay the Company royalties on net sales of licensed products. The Company and Novartis each may terminate the Novartis Agreement if the other party materially breaches the agreement and does not cure such breach within a specified cure period, if the other party experiences any specified insolvency event, if the other party challenges or assists a third party in challenging the validity or enforceability of certain patent rights controlled by the terminating party, or if the parties are prevented in any manner that materially adversely affects the progression of the development or commercialization of licensed products for a specified period as a result of specified governmental actions. Novartis may terminate the Novartis Agreement at any time without cause, or within a specified period after a change in control of the Company, as defined in the Novartis Agreement, or for specified safety reasons, effective at the end of a specified period following Novartis’s written notice to the Company of Novartis’s election to terminate the agreement. The Company may also terminate the agreement if Novartis determines to seek marketing approval of an alternative anti-PDGF product in the Novartis Territory as more fully described below. If the Company elects to terminate the Novartis Agreement because specified governmental actions prevent the parties from materially progressing the development or commercialization of licensed products as described above, the Company will be required to pay a substantial termination fee, with the specific amount of such fee determined based on the effective date of the termination. Following any termination, all rights to Fovista that the Company granted to Novartis, including, without limitation, the right to commercialize standalone Fovista products in the Novartis Territory, will revert to the Company, Novartis will perform specified activities in connection with transitioning to the Company the rights and responsibilities for the continued development, manufacture and commercialization of the standalone Fovista product for countries in the Novartis Territory, and the parties will cooperate on an orderly wind down of development and commercialization activities for other licensed products in the Novartis Territory. Novartis has agreed to specified limitations on its ability to in-license, acquire or commercialize any anti-PDGF product that does not contain Fovista (an “Alternative Anti-PDGF Product”) in the Novartis Territory and, to the extent Novartis develops, in-licenses or acquires such a product, to make such product available to the Company in the United States under specified option conditions. If the Company exercises its option, the Company will be obligated to make certain payments to Novartis, including specified milestone and royalty payments. The amounts of such payments will vary based on the product’s stage of clinical development at the time the Company exercise its option, whether the product is a standalone or combination product and whether Novartis exercises an option to co-promote such product in the United States. If Novartis determines to seek marketing approval of an Alternative Anti-PDGF Product in the Novartis Territory, the Company will, subject to specified limitations, have the option to terminate the Novartis Agreement, convert Novartis’s exclusive licenses into non-exclusive licenses, or elect to receive a royalty on sales of such product by Novartis. If the Company elects to terminate the Novartis Agreement, Novartis will, subject to specified limitations, be required to pay to the Company, certain payments based on achievement, with respect to such product, of the milestones that would have otherwise applied to licensed products under the Novartis Agreement. Activities under the licensing and commercialization 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 includes 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”). Novartis has the right, subject to the certain approval rights of the Company, to sublicense the exclusive royalty-bearing license to commercialize Fovista in the Novartis Territory. The Company’s obligation to provide access to clinical and regulatory information as part of the License Deliverable includes the obligation to provide access to all clinical data, regulatory filings, safety data and manufacturing data to Novartis which is necessary for commercialization of Fovista in the Novartis Territory. The R&D Activity Deliverable includes the right and responsibility for the Company to conduct the Phase 3 Fovista clinical program and other studies of Fovista in the Novartis Territory which are necessary or desirable for regulatory approval or commercialization of Fovista. The Manufacturing Deliverable includes the obligation for the Company to supply API to Novartis for development and manufacturing purposes, for which Novartis has agreed to pay the Company’s manufacturing costs, plus a specified margin. The Joint Operating Committee Deliverable includes 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 will be accounted for separately. Options are considered substantive if, at the inception of the arrangement, the Company is at risk as to whether the collaboration partner will choose to exercise the option. Factors that the Company considers in evaluating whether an option is substantive include the overall objective of the arrangement, the benefit the collaborator might obtain from the arrangement without exercising the option, the cost to exercise the option and the likelihood that the option will be exercised. For arrangements under which an option is considered substantive, assuming the option is not priced at a significant and incremental discount, the Company does not consider the item underlying the option to be a deliverable at the inception of the arrangement and the associated option fees are not included in allocable arrangement consideration. Conversely, for arrangements under which an option is not considered substantive or if an option is priced at a significant and incremental discount, the Company would consider the item underlying the option to be a deliverable at the inception of the arrangement and a corresponding amount would be included in allocable arrangement consideration. All of the options included in the Novartis Agreement have been determined to be substantive, and none of the options are priced at a significant and incremental discount. The Novartis Agreement provides that, if the Company elects to terminate the Novartis Agreement because specified governmental actions prevent the parties from materially progressing the development or commercialization of licensed products as described above, the Company will be required to pay a substantial termination fee, with the specific amount of such fee determined based on the effective date of the termination. The Company has concluded that this termination provision constitutes a contingent event that was unknown at the inception of the agreement. As such, the Company has recorded the $200.0 million upfront payment in deferred revenue, long-term until such time that the contingency related to this termination provision is resolved. The Company believes the enrollment milestones and certain of the regulatory milestones that may be achieved under the Novartis Agreement do not meet the recognition criteria within the definition of a milestone included in ASU 2010-17, Revenue Recognition—Milestone Method, and therefore, payments received for the achievement of the enrollment milestones in excess of the termination fee will be included in the allocable arrangement consideration and allocated to the deliverables based upon BESP using the relative selling price method. The Company believes the remaining regulatory approval milestones that may be achieved under the Novartis Agreement are consistent with the definition of a milestone included in ASU 2010-17, Revenue Recognition—Milestone Method, and, accordingly, the Company will recognize payments related to the achievement of such milestones, if any, when the applicable milestone is achieved. Factors considered in this determination included scientific and regulatory risks that must be overcome to achieve each milestone, the level of effort and investment required to achieve each milestone, and the monetary value attributed to each milestone. In each of September 2014 and March 2015, the Company achieved a $50.0 million enrollment-based milestone, or $100.0 million in aggregate, under the Novartis Agreement. The Company recognized revenue of approximately $41.7 million during the three months ended March 31, 2015, which primarily related to the $50.0 million milestone it achieved in March 2015. The balance of the milestone payment was recorded as deferred revenue. Using the relative selling price method, the Company allocated revenue of $38.1 million to the license it delivered to Novartis under the Novartis Agreement, $3.6 million to research and development activities that the Company performed under the Novartis Agreement during the three months ended March 31, 2015, and a de minimis amount of revenue associated with its joint operating committee participation obligation during the same period.

Financing Agreement with Novo A/S	3 Months Ended
Financing Agreement with Novo A/S	Mar. 31, 2015
Financing Agreement with Novo A/S
Financing Agreement with Novo A/S	7.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 low to mid-single digit percentages of net sales, with the royalty percentage determined by the amount of funding provided by Novo A/S. 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. 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 March 31, 2015, 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, which would result in a corresponding increase in the liability balance. 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	3 Months Ended
Income Taxes	Mar. 31, 2015
Income Taxes
Income Taxes	8.Income Taxes The Company utilizes the liability method of accounting for deferred income taxes. Under this method, deferred tax liabilities and assets are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities. A valuation allowance is established against deferred tax assets when, based on the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company’s policy is to record interest and penalties on uncertain tax positions as income tax expense. As of March 31, 2015 and December 31, 2014, the Company does not believe any material uncertain tax positions were present. Accordingly, interest and penalties have not been accrued due to an uncertain tax position. In assessing the realizability of deferred tax assets, the Company considers whether it is more likely than not that some portion or all of the deferred tax assets will not be realized. The ultimate realization of deferred tax assets is dependent upon the generation of taxable income during the periods in which the temporary differences representing net future deductible amounts become deductible, and is impacted by the Company’s ability to carryback losses to 2014, the only year in which the Company had taxable income. The Company is currently projecting tax losses in 2015 and in future years. The Company recorded net income of $6.6 million during the three months ended March 31, 2015, which included a provision for income taxes of approximately $1.0 million. However, due to the Company’s projected tax loss in 2015, the Company expects to record an income tax benefit for the full year. The Company currently expects to realize certain of its net deferred tax assets recorded as of March 31, 2015 due to the Company’s ability to carryback its projected federal tax losses to 2014. Because of the Company’s history of losses and lack of other positive evidence to support taxable income after the 2014 tax year, the Company has recorded a valuation allowance against those remaining deferred tax assets that are not expected to be realized. Deferred tax assets relating to employee share-based compensation deductions were reduced to reflect exercises of non-qualified stock option grants and vesting of restricted stock. Although certain of these deductions were reported on the corporate tax returns and increased net operating losses, these related tax benefits were not recognized for financial reporting purposes.

Fair Value Measurements

3 Months Ended

Mar. 31, 2015

Fair Value Measurements

Fair Value Measurements

9.Fair Value Measurements

ASC 820, Fair Value Measurements and Disclosures, 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 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 U.S. Treasury 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.

Level 3—inputs to the valuation methodology are unobservable and significant to the fair value measurement of the asset or liability.

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 March 31, 2015:

	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 U.S. Treasury money market funds*	$	34,347	$	—	$	—
Investments in U.S. Treasury securities maturities < 1 year	$	393,718	$	—	$	—

	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 U.S. Treasury money market funds*	$	35,111	$	—	$	—
Investments in U.S. Treasury securities maturities < 1 year	$	423,746	$	—	$	—

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

Stock Option and Compensation Plans

3 Months Ended

Mar. 31, 2015

Stock Option and Compensation Plans

Stock Option and Compensation Plans

10.Stock Option and Compensation Plans

The Company adopted its 2007 Stock Incentive Plan (the “2007 Plan”) for employees, directors and consultants for the purpose of advancing the interests of the Company 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, restricted stock awards, 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. The 2013 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock unit awards, and other stock-based awards. Upon 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.

In connection with the evergreen provisions of the 2013 Plan, the number of shares available for issuance under the 2013 Plan was increased by approximately 1,360,000 effective as of January 1, 2015. As of March 31, 2015, the Company had approximately 1,350,000 shares available for grant under the 2013 Plan.

Cash proceeds from, and the aggregate intrinsic value of, stock options exercised during the three months ended March 31, 2015 and 2014, respectively, were as follows:

	Three months ended March 31,
	2015		2014

Cash Proceeds from options exercised	$	1,760	$	11
Aggregate intrinsic value of options exercised	$	10,368	$	369

A summary of the stock options outstanding and exercisable as of March 31, 2015 is as follows:

			As of March 31, 2015
			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	1,057		6.8	$	5.85	639		$	3.77
$ 10.04-$20.00	495		8.0	$	13.39	128		$	13.44
$ 20.01-$30.00	176		8.6	$	25.64	55		$	25.49
$ 30.01-$40.00	1,595		9.0	$	33.59	338		$	32.36
$ 40.01-$53.95	473		9.7	$	45.63	—		$	—

	3,796					1,160


Aggregate Intrinsic Value	$	84,600				$	37,497

In connection with stock option awards granted to employees, the Company recognized share-based compensation expense of approximately $3.9 million and $2.5 million for the three months ended March 31, 2015 and 2014, respectively, net of expected forfeitures. As of March 31, 2015, there was approximately $44.3 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option awards to employees, which are expected to be recognized over a remaining weighted average period of 3.1 years.

In connection with stock options awards granted to consultants, the Company recognized approximately $0.7 million and $0.2 million in share-based compensation expense during the three months ended March 31, 2015 and 2014, respectively, net of expected forfeitures. As of March 31, 2015, there was approximately $5.1 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option award granted to consultants which are expected to be recognized over a remaining weighted average period of 2.2 years.

As of March 31, 2015, the Company had approximately 172,000 restricted stock units outstanding. In connection with restricted stock units granted to employees, the Company recognized share-based compensation of approximately $0.5 million during the three months ended March 31, 2015, net of expected forfeitures. The Company did not recognize any share-based compensation expense related to restricted stock units during the three months ended March 31, 2014. As of March 31, 2015, there was approximately $6.3 million of unrecognized compensation costs, net of estimated forfeitures, related to restricted stock units granted to employees to be recognized over a remaining weighted average period of 3.6 years.

Property and Equipment

3 Months Ended

Mar. 31, 2015

Property and Equipment

Property and Equipment

11.Property and Equipment

Property and equipment at March 31, 2015 and December 31, 2014 were as follows:

	Useful Life
	(Years)	March 31, 2015			December 31, 2014
Manufacturing and clinical equipment	7-10	$	617		$	617
Computer and other office equipment	5	299			292
Furniture and fixtures	7	628			591
Leasehold improvements	3-5	377			357

		1,921			1,857
Accumulated depreciation		(358		)	(302		)

Property and equipment, net		$	1,563		$	1,555

For the three months ended March 31, 2015 and 2014, depreciation expense was $56 thousand and $12 thousand, respectively.

Subsequent event	3 Months Ended
Subsequent event	Mar. 31, 2015
Subsequent event
Subsequent Event	12.Subsequent event In April 2015, the Company entered into a sublease agreement (the “Sublease”) with Otsuka America Pharmaceutical, Inc., (“Otsuka”), as sublandlord, pursuant to which the Company will sublease from Otsuka approximately 35,200 square feet of office space in Princeton, New Jersey. The term of the Sublease will expire in March 2021, and is subject to an early termination right in favor of the Company. The termination right is exercisable prior to April 2017 and, if exercised, would be effective in February 2018 and would trigger a termination payment by the Company of approximately $1.2 million. The Company has agreed to pay aggregate rental fees of approximately $6.9 million over the term of the Sublease, subject to certain partial rent abatements and offsets by the sublandlord during the first three years of the Sublease. The Company is also liable for taxes, operating expenses and utility and other charges related to the subleased premises.

Restatement of Previously Issued Financial Statements

3 Months Ended

Mar. 31, 2015

Restatement of Previously Issued Financial Statements

Restatement of Previously Issued Financial Statements

13.Restatement of Previously Issued Financial Statements

Subsequent to the issuance of the Company’s Quarterly Report on Form 10-Q for the three months ended March 31, 2015, the Company and the audit committee of the Board of Directors concluded that the Company should restate its balance sheets as of March 31, 2015 and December 31, 2014, and its statements of operations, comprehensive income (loss), and cash flows for the three months ended March 31, 2015 to correct the following errors:

In the second quarter of 2014, the Company recorded an income tax benefit by reducing a portion of its valuation allowance against its gross deferred tax assets. In determining the amount of the valuation allowance release, the Company considered anticipated 2015 tax losses which would generate a refund of a portion of federal income taxes paid in 2014. The Company has now determined that, as a matter of accounting principle, the net deferred tax asset recorded on its balance sheet was overstated as of and for the periods ending December 31, 2014 and March 31, 2015 and the income tax provision on its statement of operations was understated as of and for the period ending March 31, 2015. The Company’s cash position and operating expenses were not affected.

The following tables summarize the effect of the restatement by major financial statement line as of March 31, 2015 and for the three months then ended:

Balance Sheet

	March 31, 2015
	As Reported			As Restated
	(unaudited)
Deferred tax assets	$	469		$	70
Total current assets	$	492,904		$	492,505
Deferred tax assets, non- current	$	22,597		$	4,412
Total assets	$	517,425		$	498,841
Accounts payable and accrued expenses	$	4,783		$	5,757
Current liabilities	$	19,523		$	20,497
Total liabilities	$	355,832		$	356,806
Accumulated deficit	$	(273,628	)	$	(293,186	)
Total stockholders’ equity	$	161,593		$	142,035
Total liabilities and stockholders’ equity	$	517,425		$	498,841

Statement of Operations

	Three Months Ended March 31, 2015
	As Reported		As Restated
	(unaudited)
Income tax provision	$	—	$	974
Net income	$	7,610	$	6,636
Basic net income per common share	$	0.22	$	0.19
Diluted net income per common share	$	0.22	$	0.19

Statement of Comprehensive Income

	Three Months Ended March 31, 2015
	As Reported		As Restated
	(unaudited)
Net income	$	7,610	$	6,636
Comprehensive income	$	7,658	$	6,684

Statement of Cash Flows

	Three Months Ended March 31, 2015
	As Reported			As Restated
	(unaudited)
Net income	$	7,610		$	6,636
Accounts payable and accrued expenses	$	(3,924	)	$	(2,950	)

Summary of Significant Accounting Policies (Policies)

3 Months Ended

Mar. 31, 2015

Summary of Significant Accounting Policies

Restatement of Previously Issued Financial Statements

Basis of Presentation

Use of Estimates

Cash and Cash Equivalents

Available for Sale Securities

Revenue Recognition

Collaboration Revenue

	Three months ended March 31,
	2015		2014

License revenue	$	38,083	$	—
Research and development activity revenue	3,585		—
Joint operating committee revenue	10		—

Total collaboration revenue	$	41,678	$	—

The Company did not recognize any revenue related to its obligation to supply active pharmaceutical ingredient, or API, for Fovista to Novartis during the three months ended March 31, 2015.

Concentration of Credit Risk

Foreign Currency Translation

Financial Instruments

Property and Equipment

Research and Development

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

Income Taxes

Share-Based Compensation

Stock Options

Restricted Stock Units

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

	Three months ended March 31,
	2015		2014

Research and development	$	3,115	$	1,662
General and administrative	1,939		1,067

Total	$	5,054	$	2,729

Recent Accounting Pronouncements

Summary of Significant Accounting Policies (Tables)

3 Months Ended

Mar. 31, 2015

Summary of Significant Accounting Policies

Schedule of collaboration revenue components

	Three months ended March 31,
	2015		2014

License revenue	$	38,083	$	—
Research and development activity revenue	3,585		—
Joint operating committee revenue	10		—

Total collaboration revenue	$	41,678	$	—

Schedule of share-based compensation expense includes expenses related to stock options and restricted stock units granted to employees and non-employees

	Three months ended March 31,
	2015		2014

Research and development	$	3,115	$	1,662
General and administrative	1,939		1,067

Total	$	5,054	$	2,729

Net Income (Loss) Per Common Share (Tables)

3 Months Ended

Mar. 31, 2015

Net Income (Loss) Per Common Share

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

	Three months ended March 31,
	2015 (Restated)		2014

Basic and diluted net income (loss) per common share calculation:
Net income (loss)	$	6,636	$	(20,682	)
Weighted average common shares outstanding - basic	34,154		32,282
Plus: effect of dilutive stock options, warrants and unvested restricted stock units	1,085		—

Weighted average common shares outstanding - dilutive	35,239		32,282

Net income (loss) per share of common stock - basic	$	0.19	$	(0.64	)

Net income (loss) per share of common stock - diluted	$	0.19	$	(0.64	)

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

	Three months ended March 31,
	2015	2014
Stock options	929	3,726
Warrants	—	88

Total	929	3,814

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

3 Months Ended

Mar. 31, 2015

Cash, Cash Equivalents and Available for Sale Securities

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

	As of March 31, 2015
	Cost		Fair Value		Carrying Value		Unrealized Loss

U.S. Treasury securities - maturities < 1 year	$	393,747	$	393,718	$	393,718	$	(29	)
U.S. Treasury securities - maturities > 1 year	—		—		—		—

Total	$	393,747	$	393,718	$	393,718	$	(29	)

	As of December 31, 2014
	Cost		Fair Value		Carrying Value		Unrealized Loss

U.S. Treasury securities - maturities < 1 year	$	423,859	$	423,746	$	423,746	$	(113	)
U.S. Treasury securities - maturities > 1 year	$	—	$	—	$	—	$	—

Total	$	423,859	$	423,746	$	423,746	$	(113	)

Schedule of changes in accumulated other comprehensive loss associated with the unrealized loss on available-for-sale securities

	Three months ended March 31,
	2015			2014

Beginning balance	$	(65	)	$	—
Current period changes in fair value, net of tax	48			18

Ending balance	$	(17	)	$	18

- 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

Fair Value Measurements (Tables)

3 Months Ended

Mar. 31, 2015

Fair Value Measurements

Schedule of the Company's assets and liabilities that are measured at fair value on 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 U.S. Treasury money market funds*	$	34,347	$	—	$	—
Investments in U.S. Treasury securities maturities < 1 year	$	393,718	$	—	$	—

	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 U.S. Treasury money market funds*	$	35,111	$	—	$	—
Investments in U.S. Treasury securities maturities < 1 year	$	423,746	$	—	$	—

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

Stock Option and Compensation Plans (Tables)

3 Months Ended

Mar. 31, 2015

Stock Option and Compensation Plans

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

	Three months ended March 31,
	2015		2014

Cash Proceeds from options exercised	$	1,760	$	11
Aggregate intrinsic value of options exercised	$	10,368	$	369

Summary of the stock options outstanding and exercisable

			As of March 31, 2015
			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	1,057		6.8	$	5.85	639		$	3.77
$ 10.04-$20.00	495		8.0	$	13.39	128		$	13.44
$ 20.01-$30.00	176		8.6	$	25.64	55		$	25.49
$ 30.01-$40.00	1,595		9.0	$	33.59	338		$	32.36
$ 40.01-$53.95	473		9.7	$	45.63	—		$	—

	3,796					1,160


Aggregate Intrinsic Value	$	84,600				$	37,497

Property and Equipment (Tables)

3 Months Ended

Mar. 31, 2015

Property and Equipment

Schedule of property and equipment

	Useful Life
	(Years)	March 31, 2015			December 31, 2014
Manufacturing and clinical equipment	7-10	$	617		$	617
Computer and other office equipment	5	299			292
Furniture and fixtures	7	628			591
Leasehold improvements	3-5	377			357

		1,921			1,857
Accumulated depreciation		(358		)	(302		)

Property and equipment, net		$	1,563		$	1,555

Restatement of Previously Issued Financial Statements (Tables)

3 Months Ended

Mar. 31, 2015

Restatement of Previously Issued Financial Statements

Summary of effects of the restatement by major financial statement line item

Balance Sheet

	March 31, 2015
	As Reported			As Restated
	(unaudited)
Deferred tax assets	$	469		$	70
Total current assets	$	492,904		$	492,505
Deferred tax assets, non- current	$	22,597		$	4,412
Total assets	$	517,425		$	498,841
Accounts payable and accrued expenses	$	4,783		$	5,757
Current liabilities	$	19,523		$	20,497
Total liabilities	$	355,832		$	356,806
Accumulated deficit	$	(273,628	)	$	(293,186	)
Total stockholders’ equity	$	161,593		$	142,035
Total liabilities and stockholders’ equity	$	517,425		$	498,841

Statement of Operations

	Three Months Ended March 31, 2015
	As Reported		As Restated
	(unaudited)
Income tax provision	$	—	$	974
Net income	$	7,610	$	6,636
Basic net income per common share	$	0.22	$	0.19
Diluted net income per common share	$	0.22	$	0.19

Statement of Comprehensive Income

	Three Months Ended March 31, 2015
	As Reported		As Restated
	(unaudited)
Net income	$	7,610	$	6,636
Comprehensive income	$	7,658	$	6,684

Statement of Cash Flows

	Three Months Ended March 31, 2015
	As Reported			As Restated
	(unaudited)
Net income	$	7,610		$	6,636
Accounts payable and accrued expenses	$	(3,924	)	$	(2,950	)

Summary of Significant Accounting Policies (Details) $ in Thousands		1 Months Ended			3 Months Ended	7 Months Ended
	May 19, 2014 USD ($)	Mar. 31, 2015 USD ($)	Sep. 30, 2014 USD ($)	May 31, 2014 USD ($)	Mar. 31, 2015 USD ($) item	Mar. 31, 2015 USD ($)
Components of collaboration revenue
Total collaboration revenue					$ 41,678
Number of categories for milestones \| item					3
License revenue
Components of collaboration revenue
Total collaboration revenue					$ 38,083
Research and development activity revenue
Components of collaboration revenue
Total collaboration revenue					3,585
Joint operating committee revenue
Components of collaboration revenue
Total collaboration revenue					10
Novartis Pharma AG
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Upfront fees received				$ 200,000
Patient enrollment-based milestone revenue achieved		$ 50,000	$ 50,000			$ 100,000
Novartis Pharma AG \| Licensing and Commercialization Agreement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Upfront fees received	$ 200,000
Novartis Pharma AG \| Licensing and Commercialization Agreement \| Achievement of specified patient enrollment milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Patient enrollment-based milestone revenue achieved		$ 50,000	$ 50,000			$ 100,000
Recognized revenues					41,700
Novartis Pharma AG \| Licensing and Commercialization Agreement \| Achievement of specified patient enrollment milestones \| License revenue
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Recognized revenues					38,100
Novartis Pharma AG \| Licensing and Commercialization Agreement \| Achievement of specified patient enrollment milestones \| Research and development activity revenue
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Recognized revenues					$ 3,600

Summary of Significant Accounting Policies (Details 2)	3 Months Ended
Summary of Significant Accounting Policies (Details 2)	Mar. 31, 2015
Minimum
Property and equipment
Estimated useful lives	3 years
Maximum
Property and equipment
Estimated useful lives	10 years

Summary of Significant Accounting Policies (Details 3) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Summary of Significant Accounting Policies
Dividend yield (as a percent)	0.00%
Share-Based Compensation
Share-based compensation expense	$ 5,054	$ 2,729
Research and development
Share-Based Compensation
Share-based compensation expense	3,115	1,662
General and administrative
Share-Based Compensation
Share-based compensation expense	$ 1,939	$ 1,067

Capitalization (Details) - USD ($) $ / shares in Units, $ in Thousands			3 Months Ended
	Feb. 18, 2014	Sep. 30, 2013	Mar. 31, 2014
Capital structure
Net proceeds, after deducting underwriters' discounts and commissions and other offering expense			$ 55,409
Common stock
Capital structure
Issuance of common stock from offering, net (in shares)	1,900,000	8,740,000
Price of common stock issued in initial public offering (in dollars per share)		$ 22.00
Net proceeds from issuance of shares of common stock after deducting underwriters' discount and commissions and other offering expenses (in dollars)		$ 175,600
Shares issued on automatic conversion of redeemable convertible preferred stock		21,038,477
Shares of common stock offered in follow on offering	2,628,571
Offering price (in dollars per share)	$ 31.50
Shares sold by selling stockholders in follow on offering	728,571
Shares sold by selling stockholders upon full exercise by underwriters of their option to purchase additional shares	342,857
Net proceeds, after deducting underwriters' discounts and commissions and other offering expense	$ 55,400

Net Income (Loss) Per Common Share (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Basic and diluted net loss per common share calculation:
Net income	$ 6,636	$ (20,682)
Weighted average common shares outstanding - basic	34,154	32,282
Plus: effect of dilutive stock options, warrants and unvested restricted stock units	1,085
Weighted average common shares outstanding - dilutive	35,239	32,282
Net income (loss) per share of common stock - basic	$ 0.19	$ (0.64)
Net income (loss) per share of common stock - diluted	$ 0.19	$ (0.64)
Anti-dilutive securities
Total (in shares)	929	3,814
Options outstanding
Anti-dilutive securities
Total (in shares)	929	3,726
Warrants
Anti-dilutive securities
Total (in shares)		88

Cash, Cash Equivalents and Available for Sale Securities (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014	Dec. 31, 2014
Cash, Cash Equivalents and Available for Sale Securities
Cash	$ 5,300		$ 4,700
Investments in U.S. Treasury money market funds and U.S. Treasury securities with maturities less than 90 days	34,300		35,100
Available for sale securities, including carrying value and estimated fair values
Cost	393,747		423,859
Unrealized Loss	29		113
Changes in accumulated other comprehensive loss
Beginning balance	(65)
Current period changes in fair value, net of tax	48	$ 18
Ending balance	(17)	$ 18
Fair Value
Available for sale securities, including carrying value and estimated fair values
Value	393,718		423,746
Carrying Value
Available for sale securities, including carrying value and estimated fair values
Value	393,718		423,746
U.S. Treasury securities with maturities less than one year
Available for sale securities, including carrying value and estimated fair values
Cost	393,747		423,859
Unrealized Loss	29		113
U.S. Treasury securities with maturities less than one year \| Fair Value
Available for sale securities, including carrying value and estimated fair values
Value	393,718		423,746
U.S. Treasury securities with maturities less than one year \| Carrying Value
Available for sale securities, including carrying value and estimated fair values
Value	$ 393,718		$ 423,746

Licensing and Commercialization Agreement with Novartis Pharma AG (Details) - Novartis Pharma AG - USD ($) $ in Millions		1 Months Ended				3 Months Ended	7 Months Ended
	May 19, 2014	Mar. 31, 2015	Oct. 31, 2014	Sep. 30, 2014	May 31, 2014	Mar. 31, 2015	Mar. 31, 2015
Agreement
Upfront fees received					$ 200.0
Patient enrollment-based milestone revenue achieved		$ 50.0		$ 50.0			$ 100.0
Licensing and Commercialization Agreement
Agreement
Upfront fees received	$ 200.0
Licensing and Commercialization Agreement \| Achievement of specified patient enrollment milestones
Agreement
Milestone payment received			$ 50.0
Patient enrollment-based milestone revenue achieved		50.0		$ 50.0			100.0
Recognized revenues						$ 41.7
Licensing and Commercialization Agreement \| Achievement of specified patient enrollment milestones \| Maximum
Agreement
Aggregate amount receivable on achievement of milestone		130.0				130.0	130.0
Licensing and Commercialization Agreement \| Achievement of specified patient enrollment milestones \| License revenue
Agreement
Recognized revenues						38.1
Licensing and Commercialization Agreement \| Achievement of specified patient enrollment milestones \| Research and development activity revenue
Agreement
Recognized revenues						3.6
Licensing and Commercialization Agreement \| Achievement of specified marketing approval milestones \| Maximum
Agreement
Aggregate amount receivable on achievement of milestone		300.0				300.0	300.0
Licensing and Commercialization Agreement \| Achievement of specified commercial sale milestone \| Maximum
Agreement
Aggregate amount receivable on achievement of milestone		$ 400.0				$ 400.0	$ 400.0

Financing Agreement with Novo A/S (Details) $ in Thousands	1 Months Ended			3 Months Ended	23 Months Ended
Financing Agreement with Novo A/S (Details) $ in Thousands	Nov. 30, 2014 USD ($)	Jan. 31, 2014 USD ($)	May 31, 2013 USD ($) tranche	Mar. 31, 2014 USD ($)	Mar. 31, 2015 USD ($)	Dec. 31, 2014 USD ($)
Agreement
Royalty rights purchase price					$ 125,000	$ 125,000
Proceeds from royalty purchase agreement				$ 41,667
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
Royalty rights purchase price			41,700
Proceeds from royalty purchase agreement	$ 41,700	$ 41,700	$ 41,700		$ 125,000

Income Taxes (Details) - USD ($) $ in Thousands	3 Months Ended
Income Taxes (Details) - USD ($) $ in Thousands	Mar. 31, 2015	Mar. 31, 2014
Income Taxes
Net income	$ 6,636	$ (20,682)
Income tax provision	$ 974

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References + Details

Fair Value Measurements (Details) - Recurring basis - Quoted prices in active markets for identical assets (Level 1) - USD ($) $ in Thousands	Mar. 31, 2015	Dec. 31, 2014
U.S. Treasury money market funds
Fair Value Measurements
Investments in U.S. Treasury money market funds and U.S. Treasury securities with maturities less than 90 days	$ 34,347	$ 35,111
U.S. Treasury securities with maturities less than one year
Fair Value Measurements
Investments in U.S. Treasury securities	$ 393,718	$ 423,746

Stock Option and Compensation Plans (Details) - USD ($) $ in Thousands		1 Months Ended	3 Months Ended
	Jan. 01, 2015	Aug. 31, 2013	Mar. 31, 2015	Mar. 31, 2014
2007 Plan
Stock Option and Compensation Plans
Number of shares of common stock available for issuance under the Plan		739,317
2013 Plan
Stock Option and Compensation Plans
Number of shares of common stock available for issuance under the Plan			1,350,000
Annual increase in shares reserved for issuance under the Plan		2,542,372
Annual increase in shares reserved for issuance under the Plan (as a percent)		4.00%
Increase in number of shares available under the Plan	1,360,000
2013 Plan \| Maximum
Stock Option and Compensation Plans
Number of shares reserved for issuance under the Plan		3,359,641
Options outstanding
Cash proceeds from, and the aggregate intrinsic value of, stock options exercised
Cash proceeds from options exercised			$ 1,760	$ 11
Aggregate intrinsic value of options exercised			$ 10,368	$ 369

Stock Option and Compensation Plans (Details 2) - Options outstanding $ / shares in Units, shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2015 USD ($) $ / shares shares
Options Outstanding
Total Options Outstanding (in shares) \| shares	3,796
Aggregate Intrinsic Value \| $	$ 84,600
Options Exercisable
Number Exercisable (in shares) \| shares	1,160
Aggregate Intrinsic Value \| $	$ 37,497
$0.12-$10.03
Range of Exercise Prices
Exercise prices, low end of range (in dollars per share)	$ 0.12
Exercise prices, high end of range (in dollars per share)	$ 10.03
Options Outstanding
Total Options Outstanding (in shares) \| shares	1,057
Weighted Average Remaining Life	6 years 9 months 18 days
Weighted Average Exercise Price (in dollars per share)	$ 5.85
Options Exercisable
Number Exercisable (in shares) \| shares	639
Weighted Average Exercise Price (in dollars per share)	$ 3.77
$10.04-$20.00
Range of Exercise Prices
Exercise prices, low end of range (in dollars per share)	10.04
Exercise prices, high end of range (in dollars per share)	$ 20.00
Options Outstanding
Total Options Outstanding (in shares) \| shares	495
Weighted Average Remaining Life	8 years
Weighted Average Exercise Price (in dollars per share)	$ 13.39
Options Exercisable
Number Exercisable (in shares) \| shares	128
Weighted Average Exercise Price (in dollars per share)	$ 13.44
$20.01-$30.00
Range of Exercise Prices
Exercise prices, low end of range (in dollars per share)	20.01
Exercise prices, high end of range (in dollars per share)	$ 30.00
Options Outstanding
Total Options Outstanding (in shares) \| shares	176
Weighted Average Remaining Life	8 years 7 months 6 days
Weighted Average Exercise Price (in dollars per share)	$ 25.64
Options Exercisable
Number Exercisable (in shares) \| shares	55
Weighted Average Exercise Price (in dollars per share)	$ 25.49
$30.01-$40.00
Range of Exercise Prices
Exercise prices, low end of range (in dollars per share)	30.01
Exercise prices, high end of range (in dollars per share)	$ 40.00
Options Outstanding
Total Options Outstanding (in shares) \| shares	1,595
Weighted Average Remaining Life	9 years
Weighted Average Exercise Price (in dollars per share)	$ 33.59
Options Exercisable
Number Exercisable (in shares) \| shares	338
Weighted Average Exercise Price (in dollars per share)	$ 32.36
$40.01 - $53.95
Range of Exercise Prices
Exercise prices, low end of range (in dollars per share)	40.01
Exercise prices, high end of range (in dollars per share)	$ 53.95
Options Outstanding
Total Options Outstanding (in shares) \| shares	473
Weighted Average Remaining Life	9 years 8 months 12 days
Weighted Average Exercise Price (in dollars per share)	$ 45.63

Stock Option and Compensation Plans (Details 3) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Other information
Share-based compensation expense	$ 5,054	$ 2,729
Stock options
Other information
Share-based compensation expense	3,900	2,500
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures	$ 44,300
Expected weighted average period to recognize share-based compensation costs	3 years 1 month 6 days
Non-employee Options \| Consultants
Other information
Share-based compensation expense	$ 700	$ 200
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures	$ 5,100
Expected weighted average period to recognize share-based compensation costs	2 years 2 months 12 days
Unvested restricted stock
Other information
Units outstanding	172,000
Unvested restricted stock \| Employees
Other information
Share-based compensation expense	$ 500
Expected weighted average period to recognize share-based compensation costs	3 years 7 months 6 days
Unrecognized share-based compensation costs, net of estimated forfeitures	$ 6,300

Property and Equipment (Details) - USD ($) $ in Thousands	3 Months Ended
Property and Equipment (Details) - USD ($) $ in Thousands	Mar. 31, 2015	Mar. 31, 2014	Dec. 31, 2014
Property and Equipment
Property and equipment, gross	$ 1,921		$ 1,857
Accumulated depreciation	(358)		(302)
Property and equipment, net	1,563		1,555
Depreciation	$ 56	$ 12
Minimum
Property and Equipment
Useful Life	3 years
Maximum
Property and Equipment
Useful Life	10 years
Manufacturing equipment
Property and Equipment
Property and equipment, gross	$ 617		617
Manufacturing equipment \| Minimum
Property and Equipment
Useful Life	7 years
Manufacturing equipment \| Maximum
Property and Equipment
Useful Life	10 years
Computer and other office equipment
Property and Equipment
Useful Life	5 years
Property and equipment, gross	$ 299		292
Furniture and fixtures
Property and Equipment
Useful Life	7 years
Property and equipment, gross	$ 628		591
Leasehold improvements
Property and Equipment
Property and equipment, gross	$ 377		$ 357
Leasehold improvements \| Minimum
Property and Equipment
Useful Life	3 years
Leasehold improvements \| Maximum
Property and Equipment
Useful Life	5 years

Subsequent event (Details) - Sublease for office space - Subsequent event - Otsuka $ in Millions	1 Months Ended
	Apr. 30, 2015 USD ($) ft²
Subsequent event
Subleased area office space \| ft²	35,200
Sublease early termination fees	$ 1.2
Sublease rental payments over the sublease period	$ 6.9
Period of rent abatements and offsets	3 years

Restatement of Previously Issued Financial Statements (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014	Dec. 31, 2014
Balance Sheets
Deferred tax assets	$ 70		$ 50
Total current assets	492,505		473,382
Deferred tax assets, non-current	4,412		4,467
Total assets	498,841		479,786
Accounts payable and accrued expenses	5,757		8,707
Current liabilities	20,497		19,831
Total liabilities	356,806		351,249
Accumulated deficit	(293,186)		(299,822)
Total stockholders' equity	142,035		128,537
Total liabilities and stockholders' equity	498,841		$ 479,786
Statements of Operations
Income tax provision	974
Net income	$ 6,636	$ (20,682)
Basic (in dollars per share)	$ 0.19	$ (0.64)
Diluted ( in dollars per share)	$ 0.19	$ (0.64)
Statement of Comprehensive Income
Net income	$ 6,636	$ (20,682)
Comprehensive income	6,684	(20,664)
Statements of Cash Flows
Net income	6,636	(20,682)
Accounts payable and accrued expenses	(2,950)	$ (57)
Previously reported
Balance Sheets
Deferred tax assets	469
Total current assets	492,904
Deferred tax assets, non-current	22,597
Total assets	517,425
Accounts payable and accrued expenses	4,783
Current liabilities	19,523
Total liabilities	355,832
Accumulated deficit	(273,628)
Total stockholders' equity	161,593
Total liabilities and stockholders' equity	517,425
Statements of Operations
Net income	$ 7,610
Basic (in dollars per share)	$ 0.22
Diluted ( in dollars per share)	$ 0.22
Statement of Comprehensive Income
Net income	$ 7,610
Comprehensive income	7,658
Statements of Cash Flows
Net income	7,610
Accounts payable and accrued expenses	$ (3,924)