REGENXBIO Inc. 10-Q

Item 1. Financial Statements. REGENXBIO INC. CONSOLIDATED BALANCE SHEETS (unaudited) (in thousands, except per share data) September 30, 2021 December 31, 2020 Assets Current assets Cash and cash equivalents $ 228,387 $ 338,426 Marketable securities 111,473 137,314 Accounts receivable, net 46,017 42,999 Prepaid expenses 18,401 10,505 Other current assets 5,886 1,953 Total current assets 410,164 531,197 Marketable securities 193,640 46,809 Accounts receivable, net 2,730 3,267 Property and equipment, net 122,231 56,467 Operating lease right-of-use assets 61,742 63,815 Restricted cash 1,330 1,330 Other assets 8,558 5,279 Total assets $ 800,395 $ 708,164 Liabilities and Stockholders’ Equity Current liabilities Accounts payable $ 19,845 $ 10,622 Accrued expenses and other current liabilities 49,694 49,082 Deferred revenue 395 449 Operating lease liabilities 1,329 2,500 Liability related to sale of future royalties 35,508 18,794 Total current liabilities 106,771 81,447 Deferred revenue 3,531 3,783 Operating lease liabilities 83,880 70,153 Liability related to sale of future royalties 144,315 174,504 Other liabilities 484 524 Total liabilities 338,981 330,411 Stockholders’ equity Preferred stock; $ 0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at September 30, 2021 and December 31, 2020 — — Common stock; $ 0.0001 par value; 100,000 shares authorized at September 30, 2021 and December 31, 2020; 42,752 and 37,476 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively 4 4 Additional paid-in capital 917,950 667,181 Accumulated other comprehensive loss ( 1,285 ) ( 360 ) Accumulated deficit ( 455,255 ) ( 289,072 ) Total stockholders’ equity 461,414 377,753 Total liabilities and stockholders’ equity $ 800,395 $ 708,164 The accompanying notes are an integral part of these unaudited consolidated financial statements. 3 Table of Contents REGENXBIO INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (unaudited) (in thousands, except per share data) Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Revenues License and royalty revenue $ 30,773 $ 98,912 $ 71,692 $ 133,122 Total revenues 30,773 98,912 71,692 133,122 Operating Expenses Cost of revenues 14,105 17,364 28,775 25,457 Research and development 47,855 43,968 133,459 119,114 General and administrative 21,030 15,859 57,293 46,246 Provision for credit losses and other 5,131 7,770 5,781 7,887 Total operating expenses 88,121 84,961 225,308 198,704 Income (loss) from operations ( 57,348 ) 13,951 ( 153,616 ) ( 65,582 ) Other Income (Expense) Interest income from licensing 117 1,444 700 4,141 Investment income (loss) 5,535 ( 6,607 ) 6,514 ( 4,071 ) Interest expense ( 6,709 ) — ( 19,777 ) — Total other income (expense) ( 1,057 ) ( 5,163 ) ( 12,563 ) 70 Income (loss) before income taxes ( 58,405 ) 8,788 ( 166,179 ) ( 65,512 ) Income Tax Benefit (Expense) — 3 ( 4 ) 503 Net income (loss) $ ( 58,405 ) $ 8,791 $ ( 166,183 ) $ ( 65,009 ) Other Comprehensive Income (Loss) Unrealized gain (loss) on available-for-sale securities, net ( 30 ) ( 487 ) ( 925 ) 58 Total other comprehensive income (loss) ( 30 ) ( 487 ) ( 925 ) 58 Comprehensive income (loss) $ ( 58,435 ) $ 8,304 $ ( 167,108 ) $ ( 64,951 ) Net income (loss) per share: Basic $ ( 1.37 ) $ 0.24 $ ( 3.93 ) $ ( 1.75 ) Diluted $ ( 1.37 ) $ 0.23 $ ( 3.93 ) $ ( 1.75 ) Weighted-average common shares outstanding: Basic 42,629 37,342 42,324 37,234 Diluted 42,629 38,877 42,324 37,234 The accompanying notes are an integral part of these unaudited consolidated financial statements. 4 Table of Contents REGENXBIO INC. CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY (unaudited) (in thousands) Three Months Ended September 30, 2021 Accumulated Additional Other Total Common Stock Paid-in Comprehensive Accumulated Stockholders’ Shares Amount Capital Loss Deficit Equity Balances at June 30, 2021 42,555 $ 4 $ 905,346 $ ( 1,255 ) $ ( 396,850 ) $ 507,245 Exercise of stock options 163 — 1,729 — — 1,729 Issuance of common stock under employee stock purchase plan 35 — 1,141 — — 1,141 Stock-based compensation expense — — 9,734 — — 9,734 Unrealized loss on available-for-sale securities, net — — — ( 30 ) — ( 30 ) Net loss — — — — ( 58,405 ) ( 58,405 ) Balances at September 30, 2021 42,752 $ 4 $ 917,950 $ ( 1,285 ) $ ( 455,255 ) $ 461,414 Three Months Ended September 30, 2020 Accumulated Additional Other Total Common Stock Paid-in Comprehensive Accumulated Stockholders’ Shares Amount Capital Income Deficit Equity Balances at June 30, 2020 37,291 $ 4 $ 648,729 $ 750 $ ( 251,622 ) $ 397,861 Exercise of stock options 74 — 268 — — 268 Issuance of common stock under employee stock purchase plan 38 — 1,192 — — 1,192 Stock-based compensation expense — — 8,035 — — 8,035 Unrealized loss on available-for-sale securities, net — — — ( 487 ) — ( 487 ) Net income — — — — 8,791 8,791 Balances at September 30, 2020 37,404 $ 4 $ 658,224 $ 263 $ ( 242,831 ) $ 415,660 The accompanying notes are an integral part of these unaudited consolidated financial statements. 5 Table of Contents REGENXBIO INC. CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY (unaudited) (in thousands) Nine Months Ended September 30, 2021 Accumulated Additional Other Total Common Stock Paid-in Comprehensive Accumulated Stockholders’ Shares Amount Capital Loss Deficit Equity Balances at December 31, 2020 37,476 $ 4 $ 667,181 $ ( 360 ) $ ( 289,072 ) $ 377,753 Issuance of common stock upon public offering, net of transaction costs of $ 14,194 4,899 — 216,059 — — 216,059 Exercise of stock options 324 — 3,296 — — 3,296 Issuance of common stock under employee stock purchase plan 54 — 1,768 — — 1,768 Stock-based compensation expense — — 29,646 — — 29,646 Unrealized loss on available-for-sale securities, net — — — ( 925 ) — ( 925 ) Net loss — — — — ( 166,183 ) ( 166,183 ) Balances at September 30, 2021 42,752 $ 4 $ 917,950 $ ( 1,285 ) $ ( 455,255 ) $ 461,414 Nine Months Ended September 30, 2020 Accumulated Additional Other Total Common Stock Paid-in Comprehensive Accumulated Stockholders’ Shares Amount Capital Income Deficit Equity Balances at December 31, 2019 36,992 $ 4 $ 627,810 $ 205 $ ( 177,822 ) $ 450,197 Exercise of stock options 356 — 4,247 — — 4,247 Issuance of common stock under employee stock purchase plan 55 — 1,799 — — 1,799 Stock-based compensation expense — — 24,368 — — 24,368 Unrealized gain on available-for-sale securities, net — — — 58 — 58 Net loss — — — — ( 65,009 ) ( 65,009 ) Balances at September 30, 2020 37,404 $ 4 $ 658,224 $ 263 $ ( 242,831 ) $ 415,660 The accompanying notes are an integral part of these unaudited consolidated financial statements. 6 Table of Contents REGENXBIO INC. CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited) (in thousands) Nine Months Ended September 30, 2021 2020 Cash flows from operating activities Net loss $ ( 166,183 ) $ ( 65,009 ) Adjustments to reconcile net loss to net cash used in operating activities Stock-based compensation expense 29,646 24,368 Depreciation and amortization 6,958 6,302 Provision for credit losses 5,532 7,678 Net amortization of premiums on marketable debt securities 4,377 586 Net losses (gains) on investments ( 5,189 ) 8,207 Imputed interest income from licensing ( 341 ) ( 2,033 ) Non-cash interest expense 19,777 — Other non-cash adjustments ( 51 ) 263 Changes in operating assets and liabilities Accounts receivable ( 7,886 ) ( 89,442 ) Prepaid expenses ( 7,896 ) ( 9,637 ) Other current assets ( 3,307 ) ( 2,998 ) Operating lease right-of-use assets 3,780 1,986 Other assets ( 4,402 ) 1,318 Accounts payable 6,503 9,907 Accrued expenses and other current liabilities 473 18,400 Operating lease liabilities 10,849 ( 2,222 ) Other liabilities ( 2 ) ( 1,203 ) Net cash used in operating activities ( 107,362 ) ( 93,529 ) Cash flows from investing activities Purchases of marketable debt securities ( 262,671 ) ( 79,374 ) Maturities of marketable debt securities 136,386 189,882 Sales of marketable debt securities — 2,287 Sales of equity securities 5,563 12,374 Purchases of property and equipment ( 69,561 ) ( 13,980 ) Net cash provided by (used in) investing activities ( 190,283 ) 111,189 Cash flows from financing activities Proceeds from exercise of stock options 3,296 4,247 Proceeds from issuance of common stock under employee stock purchase plan 1,768 1,799 Proceeds from public offering of common stock, net of underwriting discounts and commissions 216,438 — Issuance costs for public offering of common stock ( 379 ) — Repayments under liability related to sale of future royalties ( 33,252 ) — Transaction costs for sale of future royalties ( 265 ) — Net cash provided by financing activities 187,606 6,046 Net increase (decrease) in cash and cash equivalents and restricted cash ( 110,039 ) 23,706 Cash and cash equivalents and restricted cash Beginning of period 339,756 70,844 End of period $ 229,717 $ 94,550 Supplemental disclosures of non-cash investing and financing activities Net additions to property and equipment through accounts payable and accrued expenses $ 3,250 $ 46 Non-cash additions to property and equipment through tenant improvement allowance $ — $ 2,217 Non-cash consideration received for licenses granted $ — $ 1,123 Proceeds due to Company from sales of non-marketable equity securities $ 646 $ — The accompanying notes are an integral part of these unaudited consolidated financial statements. 7 Table of Contents REGENXBIO INC. NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (unaudited) 1. Nature of Business REGENXBIO Inc. (the Company) is a clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. The Company has developed a broad pipeline of gene therapy product candidates using its proprietary adeno-associated virus (AAV) gene delivery platform (NAV Technology Platform), which consists of exclusive rights to over 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. In addition to its internal product development efforts, the Company also selectively licenses the NAV® Technology Platform to other leading biotechnology and pharmaceutical companies (NAV Technology Licensees). As of September 30, 2021, the NAV Technology Platform was being applied by NAV Technology Licensees in one commercially available product, Zolgensma®, and in the preclinical and clinical development of 20 licensed products. The Company was formed in 2008 in the State of Delaware and is headquartered in Rockville, Maryland. As of September 30, 2021, the Company had generated an accumulated deficit of $ 455.3 million since inception. As the Company has incurred cumulative losses since inception, transition to recurring profitability is dependent upon achieving a level of revenues adequate to support the Company’s cost structure, which depends heavily on the successful development, approval and commercialization of its product candidates. The Company may never achieve recurring profitability, and unless and until it does, the Company will continue to need to raise additional capital, to the extent possible. As of September 30, 2021, the Company had cash, cash equivalents and marketable securities of $ 533.5 million, which management believes is sufficient to fund operations for at least the next 12 months from the date these consolidated financial statements were issued. 2. Summary of Significant Accounting Policies Basis of Presentation and Principles of Consolidation The accompanying consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). The interim unaudited consolidated financial statements have been prepared on the same basis as the annual audited consolidated financial statements as of and for the year ended December 31, 2020 included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the SEC on March 1, 2021. Certain information and footnote disclosures required by GAAP which are normally included in the Company’s annual consolidated financial statements have been omitted pursuant to SEC rules and regulations for interim reporting. In the opinion of management, the accompanying consolidated financial statements reflect all adjustments, which are normal and recurring in nature, necessary for fair financial statement presentation. The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year, any other interim periods, or any future year or period. These interim consolidated financial statements should be read in conjunction with the audited consolidated financial statements as of and for the year ended December 31, 2020, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. The accompanying consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Use of Estimates The preparation of the consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses and related disclosure of contingent assets and liabilities for the periods presented. Management bases its estimates on historical experience and on various other factors that it believes are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities, and other reported amounts, that are not readily apparent from other sources. Actual results may differ materially from these estimates. Significant estimates are used in the following areas, among others: license and royalty revenue, the allowance for credit losses, accrued research and development expenses and other accrued liabilities, stock-based compensation expense, non-cash interest expense, income taxes and the fair value of financial instruments. 8 Table of Contents The Company is actively monitoring the impact of the COVID-19 pandemic on its business, results of operations and financial condition. The full extent to which the COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition in the future is unknown at this time and will depend on future developments that are highly unpredictable . The most significant estimates affecting the Company’s consolidated financial statements that may be impacted by the COVID-19 pandemic are related to the Company’s assessment of credit losses on accounts receivable, contract assets and available-for-sale debt securities. Restricted Cash Restricted cash includes money market mutual funds used to collateralize irrevocable letters of credit as required by the Company’s lease agreements. The following table provides a reconciliation of cash and cash equivalents and restricted cash as reported on the consolidated balance sheets to the total of these amounts as reported at the end of the period in the consolidated statements of cash flows (in thousands): September 30, 2021 September 30, 2020 Cash and cash equivalents $ 228,387 $ 93,220 Restricted cash 1,330 1,330 Total cash and cash equivalents and restricted cash $ 229,717 $ 94,550 Accounts Receivable Accounts receivable primarily consist of consideration due to the Company resulting from its license agreements with NAV Technology Licensees. Accounts receivable include amounts invoiced to licensees as well as rights to consideration which have not yet been invoiced, including unbilled royalties, and for which payment is conditional solely upon the passage of time. If a licensee elects to terminate a license prior to the end of the license term, the licensed intellectual property is returned to the Company and any accounts receivable from the licensee which are not contractually payable to the Company are charged off as a reduction of license revenue in the period of the termination. Accounts receivable which are not expected to be received by the Company within 12 months from the reporting date are stated net of a discount to present value and recorded as non-current assets on the consolidated balance sheets. The present value discount is recognized as a reduction of revenue in the period in which the accounts receivable are initially recorded and is accreted as interest income from licensing over the term of the receivables. Accounts receivable are stated net of an allowance for credit losses, if deemed necessary based on the Company’s evaluation of collectability and potential credit losses. Management assesses the collectability of its accounts receivable using the specific identification of account balances, and considers the credit quality and financial condition of its significant customers, historical information regarding credit losses and the Company’s evaluation of current and expected future economic conditions. If necessary, an allowance for credit losses is recorded against accounts receivable such that the carrying value of accounts receivable reflects the net amount expected to be collected. Accounts receivable balances are written off against the allowance for credit losses when the potential for collectability is considered remote. Please refer to Note 8 for further information regarding the allowance for credit losses related to accounts receivable. Fair Value of Financial Instruments The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. Accounting Standards Codification (ASC) 820, Fair Value Measurements and Disclosures , establishes a hierarchy of inputs used in measuring fair value that maximizes the use of observable inputs and minimizes the use of unobservable inputs by requiring that the observable inputs be used when available. Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability, and are developed based on the best information available in the circumstances. The fair value hierarchy applies only to the valuation inputs used in determining the reported fair value of the investments and is not a measure of the investment credit quality. The three levels of the fair value hierarchy are described below: • Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. 9 Table of Contents • Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly. • Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The fair values of the Company’s Level 2 instruments are based on quoted market prices or broker or dealer quotations for similar assets. These investments are initially valued at the transaction price and subsequently valued utilizing third party pricing providers or other market observable data. Please refer to Note 4 for further information on the fair value measurement of the Company’s financial instruments. Net Income (Loss) Per Share Basic net income (loss) per share is calculated by dividing net income (loss) applicable to common stockholders by the weighted-average common shares outstanding during the period, without consideration for common stock equivalents. Diluted net income (loss) per share is calculated by adjusting the weighted-average common shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury-stock method. Contingently convertible shares in which conversion is based on non-market-priced contingencies are excluded from the calculations of both basic and diluted net income (loss) per share until the contingency has been fully met. For purposes of the diluted net income (loss) per share calculation, common stock equivalents are excluded from the calculation of diluted net income (loss) per share if their effect would be anti-dilutive. 3. Marketable Securities The following tables present a summary of the Company’s marketable securities, which consist solely of available-for-sale debt securities (in thousands): Amortized Cost Unrealized Gains Unrealized Losses Fair Value September 30, 2021 U.S. government and federal agency securities $ 25,003 $ — $ ( 23 ) $ 24,980 Certificates of deposit 2,939 10 ( 1 ) 2,948 Corporate bonds 274,592 41 ( 452 ) 274,181 Municipal securities 3,004 — — 3,004 $ 305,538 $ 51 $ ( 476 ) $ 305,113 Amortized Cost Unrealized Gains Unrealized Losses Fair Value December 31, 2020 U.S. government and federal agency securities $ 12,782 $ 22 $ — $ 12,804 Certificates of deposit 1,956 34 — 1,990 Corporate bonds 165,850 497 ( 55 ) 166,292 Municipal securities 3,035 2 — 3,037 $ 183,623 $ 555 $ ( 55 ) $ 184,123 As of September 30, 2021 and December 31, 2020, no available-for-sale debt securities had remaining maturities greater than three years. The amortized cost of marketable debt securities is adjusted for amortization of premiums and accretion of discounts to maturity, or to the earliest call date for callable debt securities purchased at a premium. 10 Table of Contents As of September 30, 2021 and December 31, 2020 , the balance in the Company’s accumulated other comprehensive loss consisted solely of unrealized gains and losses on available-for-sale debt securities, net of reclassification adjustments for realized gains and losses and income tax effects. The Company uses the aggregate portfolio approach to release the tax effects of unrealized gains and losses on available-for-sale debt securities in accumulated other comprehensive loss. Realized gains and losses from the sale or maturity of marketable securities are based on the specific identification method and are included in results of operations as investment income (loss) . Unrealized gain ( loss ) on available-for-sale securities, net, as presented in the statements of operations and comprehensive income ( loss ) consisted of the following (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Unrealized gain (loss) before reclassifications $ ( 30 ) $ ( 457 ) $ ( 918 ) $ 108 Realized gains reclassified to investment income (loss) — ( 30 ) ( 7 ) ( 50 ) Income tax expense — — — — Unrealized gain (loss) on available-for-sale securities, net $ ( 30 ) $ ( 487 ) $ ( 925 ) $ 58 The following tables present the fair values and unrealized losses of available-for-sale debt securities held by the Company in an unrealized loss position for less than 12 months and 12 months or greater (in thousands): Less than 12 Months 12 Months or Greater Total Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses September 30, 2021 U.S. government and federal agency securities $ 17,444 $ ( 23 ) $ — $ — $ 17,444 $ ( 23 ) Certificates of deposit 489 ( 1 ) — — 489 ( 1 ) Corporate bonds 238,835 ( 452 ) — — 238,835 ( 452 ) $ 256,768 $ ( 476 ) $ — $ — $ 256,768 $ ( 476 ) Less than 12 Months 12 Months or Greater Total Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses December 31, 2020 Corporate bonds $ 55,507 $ ( 55 ) $ — $ — $ 55,507 $ ( 55 ) $ 55,507 $ ( 55 ) $ — $ — $ 55,507 $ ( 55 ) As of September 30, 2021, available-for-sale debt securities held by the Company in an unrealized loss position consisted of 47 investment grade security positions. The Company has the intent and ability to hold such securities until recovery, and based on the credit quality of the issuers and low severity of each unrealized loss position relative to its amortized cost basis, the Company did not identify any credit losses associated with its available-for-sale debt securities. The Company did no t record an allowance for credit losses on its available-for-sale debt securities as of September 30, 2021 or December 31, 2020. The Company did no t recognize any impairment or credit losses on available-for-sale debt securities during the three and nine months ended September 30, 2021 and 2020. During the three and nine months ended September 30, 2020, the Company recognized total net realized and unrealized losses of $ 7.5 million and $ 8.3 million, respectively, related to its marketable equity securities of Prevail Therapeutics Inc. (Prevail), which were acquired as consideration for a license to the NAV Technology Platform granted to Prevail in August 2017. As of December 31, 2020, the Company had sold all of its Prevail equity securities. 11 Table of Contents 4. Fair Value of Financial Instruments Financial instruments reported at fair value on a recurring basis include cash equivalents and marketable securities. The following tables present the fair value of cash equivalents and marketable securities in accordance with the hierarchy discussed in Note 2 (in thousands): Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total September 30, 2021 Cash equivalents: Money market mutual funds $ — $ 187,123 $ — $ 187,123 Total cash equivalents — 187,123 — 187,123 Marketable securities: U.S. government and federal agency securities — 24,980 — 24,980 Certificates of deposit — 2,948 — 2,948 Corporate bonds — 274,181 — 274,181 Municipal securities — 3,004 — 3,004 Total marketable securities — 305,113 — 305,113 Total cash equivalents and marketable securities $ — $ 492,236 $ — $ 492,236 Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total December 31, 2020 Cash equivalents: Money market mutual funds $ — $ 96,307 $ — $ 96,307 Total cash equivalents — 96,307 — 96,307 Marketable securities: U.S. government and federal agency securities — 12,804 — 12,804 Certificates of deposit — 1,990 — 1,990 Corporate bonds — 166,292 — 166,292 Municipal securities — 3,037 — 3,037 Total marketable securities — 184,123 — 184,123 Total cash equivalents and marketable securities $ — $ 280,430 $ — $ 280,430 Management estimates that the carrying amounts of its current accounts receivable, accounts payable and accrued expenses and other current liabilities approximate fair value due to the short-term nature of those instruments. Accounts receivable which contain non-current portions are recorded at their present values using a discount rate that is based on prevailing market rates and the credit profile of the licensee on the date the amounts are initially recorded. Management does not believe there have been any significant changes in market conditions or credit quality that would cause the discount rates initially used to be materially different from those that would be used as of September 30, 2021 to determine the present value of the receivables. Accordingly, management estimates that the carrying value of its non-current accounts receivable approximates the fair value of those instruments. Non-marketable Equity Securities Non-marketable equity securities are measured at cost less impairment, adjusted for observable price changes for identical or similar investments of the same issuer. As of September 30, 2021, the Company did no t hold any non-marketable equity securities. As of December 31, 2020, non-marketable equity securities had a carrying value of $ 1.1 million and were included in other assets on the consolidated balance sheet. The Company did not identify any observable price changes or changes in circumstances that would have had an adverse effect on the fair value of the securities as of December 31, 2020. No remeasurements or impairment losses were recorded on non-marketable equity securities during the three and nine months ended September 30, 2021 and 2020. 12 Table of Contents The Company’s non-marketable equity securities as of December 31, 2020 consisted solely of equity securities of Corlieve Therapeutics SAS (Corlieve), which were acquired in June 2020 as consideration under a license and collaboration agreement with Corlieve. In July 2021, Corlieve was acquired by uniQure N.V. (uniQure). In exchange for its ownership in Corlieve, the Company received proceeds of € 4.8 million ($ 5.6 million) from uniQure and is entitled to receive additional proceeds of € 0.6 million ($ 0.6 million as of September 30, 2021) by July 2022. During the three and nine months ended September 30, 2021, the Company recorded a realized gain of $ 5.2 million as a result of the acquisition of its Corlieve securities by uniQure, which is included in investment income (loss) in the consolidated statements of operations and comprehensive income (loss). In connection with the acquisition, the Company is also eligible to receive payments of up to € 37.1 million ($ 43.2 million as of September 30, 2021) from uniQure contingent upon the achievement of various development and regulatory milestones, none of which have been recognized in the consolidated financial statements as of September 30, 2021. Proceeds contingent upon the achievement of these milestones will be recognized as investment income in the period in which any uncertainty regarding realization is substantially resolved, which may not occur until the achievement of the underlying milestones. It is at least reasonably possible that some or all of the proceeds contingent upon these milestones will not be realized by the Company. 5. Property and Equipment, Net Property and equipment, net consists of the following (in thousands): September 30, 2021 December 31, 2020 Laboratory and manufacturing equipment $ 49,951 $ 26,306 Computer equipment and software 4,070 3,764 Furniture and fixtures 6,515 4,114 Leasehold improvements 88,131 44,957 Total property and equipment 148,667 79,141 Accumulated depreciation and amortization ( 26,436 ) ( 22,674 ) Property and equipment, net $ 122,231 $ 56,467 6. Liability Related to Sale of Future Royalties In December 2020, the Company entered into a royalty purchase agreement (the Royalty Purchase Agreement) with entities managed by Healthcare Royalty Management, LLC (collectively, HCR). Under the agreement, HCR purchased the Company’s rights to a capped amount of Zolgensma royalty payments under the Company’s license agreement with Novartis Gene Therapies, Inc. (formerly AveXis, Inc.) (Novartis Gene Therapies), including $ 4.0 million of royalty payments received by the Company in the fourth quarter of 2020 (the Pledged Royalties). In consideration for these rights, HCR paid the Company $ 200.0 million (the Purchase Price), less $4.0 million representing the payment of the Pledged Royalties to HCR. Beginning upon the effective date of the agreement, Zolgensma royalty payments, up to a specified threshold, will be paid to HCR, net of upstream royalties payable by the Company to certain licensors in accordance with existing license agreements. Pursuant to the Royalty Purchase Agreement, the total amount of royalty payments to be received by HCR under the agreement is subject to an increasing cap (the Cap Amount) equal to (i) $ 260.0 million applicable for the period from the effective date of the agreement through November 7, 2024, and (ii) $ 300.0 million applicable for the period from November 8, 2024 through the effective date of termination of the license agreement with Novartis Gene Therapies. If, on or prior to the defined dates for each Cap Amount, the total amount of royalty payments received by HCR equals or exceeds the Cap Amount applicable to such date, the Royalty Purchase Agreement will automatically terminate and all rights to the Zolgensma royalty payments will revert back to the Company. The Company has no obligation to repay any amounts to HCR if total future Zolgensma royalty payments are not sufficient to achieve the applicable Cap Amount prior to the termination of the license agreement with Novartis Gene Therapies. The Company has a call option to repurchase its rights to the purchased royalties from HCR for a repurchase price equal to, as of the option exercise date, $ 300.0 million minus the total amount of royalty payments received by HCR; provided, however, that with respect to a call option exercised on or before November 7, 2024, in the event that the then applicable Cap Amount minus the total amount of royalty payments received by HCR is less than $ 1.0 million, the repurchase price shall equal such difference. The proceeds received from HCR of $ 196.0 million were recorded as a liability, net of transaction costs of $ 3.5 million, which is amortized over the estimated life of the arrangement using the effective interest method. In order to determine the amortization of the liability, the Company is required to estimate the total amount of future royalty payments to be received by HCR, subject to the Cap Amount, over the life of the arrangement. The total amount of royalty payments received by HCR under the agreement, less the net proceeds received by the Company of $ 192.5 million, is recorded as non-cash interest expense over the life of the arrangement using the effective interest method. Due to its continuing involvement in the underlying license agreement with Novartis Gene Therapies, the Company continues to recognize royalty revenue on net sales of Zolgensma and records the royalty payments to HCR as a reduction of the liability when paid. As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement. 13 Table of Contents The Company estimates the effective interest rate used to record non-cash interest expense under the Royalty Purchase Agreement based on its estimate of future royalty payments to be received by HCR. As of September 30, 2021, the estimated effective interest rate under the agreement was 14.9 %. Over the life of the arrangement, the actual effective interest rate will be affected by the amount and timing of the royalty payments received by HCR and changes in the Company’s forecasted royalties. At each reporting date, the Company reassesses its estimate of total future royalty payments to be received by HCR at the applicable Cap Amount, and prospectively adjusts the effective interest rate and amortization of the liability, as necessary. The following table presents the changes in the liability related to the sale of future royalties under the Royalty Purchase Agreement with HCR (in thousands): Nine Months Ended September 30, 2021 Liability related to sale of future royalties, beginning balance $ 193,298 Zolgensma royalties paid to HCR ( 33,252 ) Non-cash interest expense 19,777 Liability related to sale of future royalties, ending balance 179,823 Current portion of liability related to sale of future royalties ( 35,508 ) Liability related to sale of future royalties, non-current $ 144,315 7. Capitalization In January 2021, the Company completed a public offering of 4,899,000 shares of its common stock (inclusive of 639,000 shares pursuant to the full exercise by the underwriters of their option to purchase additional shares) at a price of $ 47.00 per share. The aggregate net proceeds received by the Company from the offering, inclusive of the underwriters’ option exercise, were $ 216.1 million, net of underwriting discounts and commissions and offering expenses payable by the Company. 8. License and Royalty Revenue As of September 30, 2021, the Company’s NAV Technology Platform was being applied by NAV Technology Licensees in one commercially available product, Zolgensma, and in the development of 20 other licensed products. Consideration to the Company under its license agreements may include: (i) up-front and annual fees, (ii) option fees to acquire additional licenses, (iii) milestone payments based on the achievement of certain development and sales-based milestones by licensees, (iv) sublicense fees and (v) royalties on sales of licensed products. Sublicense fees vary by license and range from a mid-single digit percentage to a low-double digit percentage of license fees received by licensees as a result of sublicenses. Royalties on net sales of commercialized products vary by license and range from a mid-single digit percentage to a low double-digit percentage of net sales by licensees. Development milestone payments are evaluated each reporting period and are only included in the transaction price of each license and recognized as license revenue to the extent the milestones are considered probable of achievement. Sales-based milestones are excluded from the transaction price of each license agreement and recognized as royalty revenue in the period of achievement. As of September 30, 2021, the Company’s license agreements, excluding additional licenses that could be granted upon the exercise of options by licensees, contained unachieved milestones which could result in aggregate milestone payments to the Company of up to $ 194.8 million, including (i) $ 23.3 million upon the commencement of various stages of clinical trials, (ii) $ 21.0 million upon the submission of regulatory approval filings, (iii) $ 93.5 million upon the approval of commercial products by regulatory agencies and (iv) $ 57.0 million upon the achievement of specified sales targets for licensed products. To the extent the milestone payments are realized by the Company, the Company will be obligated to pay sublicense fees to licensors based on a specified percentage of the fees earned by the Company. The achievement of milestones by licensees is highly dependent on the successful development and commercialization of licensed products and it is at least reasonably possible that some or all of the milestone fees will not be realized by the Company. 14 Table of Contents Changes in Accounts Receivable, Contract Assets and Deferred Revenue The following table presents changes in the balances of the Company’s net accounts receivable, contract assets and deferred revenue, as well as other information regarding revenue recognized during the periods presented (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Accounts receivable, net, current and non-current: Balance, beginning of period $ 47,202 $ 46,494 $ 46,266 $ 42,303 Additions 30,385 100,243 71,544 137,220 Deductions ( 28,840 ) ( 21,057 ) ( 69,063 ) ( 53,843 ) Balance, end of period $ 48,747 $ 125,680 $ 48,747 $ 125,680 Contract assets: Balance, beginning of period $ 702 $ 350 $ 350 $ — Additions 407 — 1,109 350 Deductions — — ( 350 ) — Balance, end of period $ 1,109 $ 350 $ 1,109 $ 350 Deferred revenue, current and non-current: Balance, beginning of period $ 4,025 $ 4,457 $ 4,232 $ 3,333 Additions — — — 1,124 Deductions ( 99 ) ( 113 ) ( 306 ) ( 113 ) Balance, end of period $ 3,926 $ 4,344 $ 3,926 $ 4,344 Revenue recognized during the period from: Amounts included in deferred revenue at beginning of period $ 99 $ 113 $ 306 $ — Performance obligations satisfied in previous periods $ 30,256 $ 98,799 $ 70,603 $ 125,555 Additions to accounts receivable during the periods presented consisted primarily of receivables recorded related to royalties on net sales of Zolgensma, new licenses granted by the Company, the achievement of development and sales-based milestones by licensees and interest income from licensing recognized during the period. Deductions to accounts receivable during the periods presented consisted primarily of amounts collected from licensees and increases in the allowance for credit losses, as discussed further below. Additions to contract assets during the periods presented consisted primarily of development milestones deemed probable of achievement by licensees during the period. Deductions to contract assets during the periods presented consisted of the achievement of such milestones and billing of the associated milestone payments by the Company. As of September 30, 2021, the Company had recorded deferred revenue of $ 3.9 million which represents consideration received from licensees for performance obligations that have not yet been satisfied by the Company. Unsatisfied performance obligations consisted of (i) options granted to licensees that provide material rights to the licensee to acquire additional licenses from the Company, which will be satisfied upon the exercise or expiration of the options and (ii) research and development services to be performed by the Company related to licensed products, which will be satisfied as the research and development services are performed. Revenue recognized from performance obligations satisfied in previous periods was primarily attributable to Zolgensma royalty revenues, the achievement of sales-based milestones for net sales of Zolgensma, sublicense fees earned from licensees and changes in the transaction prices of the Company’s license agreements. Changes in transaction prices were primarily attributable to development milestones achieved or deemed probable of achievement during the periods, which were previously not considered probable of achievement. 15 Table of Contents Accounts Receivable, Contract Assets and the Allowance for Credit Losses Accounts receivable, net consisted of the following (in thousands): September 30, 2021 December 31, 2020 Current accounts receivable: Billed to customers $ 30,248 $ 30,573 Unbilled 28,979 20,104 Allowance for credit losses ( 13,210 ) ( 7,678 ) Current accounts receivable, net 46,017 42,999 Non-current accounts receivable: Unbilled 2,730 3,267 Allowance for credit losses — — Non-current accounts receivable, net 2,730 3,267 Total accounts receivable, net $ 48,747 $ 46,266 The following table presents the changes in the allowance for credit losses related to accounts receivable and contract assets for the nine months ended September 30, 2021 (in thousands): Accounts Receivable Contract Assets Balance at December 31, 2020 $ 7,678 $ — Provision for credit losses 5,532 — Write-offs — — Balance at September 30, 2021 $ 13,210 $ — The Company’s allowance for credit losses as of September 30, 2021 and December 31, 2020 was related solely to accounts receivable from Abeona Therapeutics Inc. (Abeona). Please refer to the section below, Abeona Therapeutics Inc., for further information regarding amounts due from Abeona and the associated allowance for credit losses. The Company’s provision for credit losses was $ 5.0 million and $ 5.5 million for the three and nine months ended September 30, 2021, respectively, and $ 7.7 million and $ 7.7 million for the three and nine months ended September 30, 2020, respectively. The Company’s provision for credit losses for the three and nine months ended September 30, 2021 and 2020 was related solely to changes in estimates regarding the collectability of the accounts receivable due from Abeona. Novartis Gene Therapies, Inc. In March 2014, the Company entered into an exclusive license agreement, as amended, (the March 2014 License) with Novartis Gene Therapies (formerly AveXis, Inc.). Under the March 2014 License, the Company granted Novartis Gene Therapies an exclusive, worldwide commercial license, with rights to sublicense, to the NAV Technology Platform, as well as other certain rights, for the treatment of spinal muscular atrophy (SMA) in humans by in vivo gene therapy. Novartis Gene Therapies launched commercial sales of Zolgensma, a licensed product under the March 2014 License, in the second quarter of 2019, upon which the Company began recognizing royalty revenue on net sales of the licensed product. Pursuant to the March 2014 License, Novartis Gene Therapies was obligated to pay a sales-based milestone fee of $ 80.0 million to the Company upon the achievement of $ 1.0 billion in cumulative net sales of licensed products. Novartis Gene Therapies achieved cumulative net sales of Zolgensma of $ 1.0 billion in the third quarter of 2020, upon which the Company recognized revenue of $ 80.0 million related to the sales-based milestone fee. The $ 80.0 million milestone fee was recorded as accounts receivable as of September 30, 2020, and the Company received payment of the $ 80.0 million milestone fee from Novartis Gene Therapies in October 2020. 16 Table of Contents The Company recognized the following amounts under the March 2014 License with Novartis Gene Therapies (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Royalties on net sales of Zolgensma $ 30,254 $ 18,799 $ 66,946 $ 40,723 Achievement of sales-based milestone for Zolgensma — 80,000 — 80,000 Other license revenue — — — 3,500 Total license and royalty revenue $ 30,254 $ 98,799 $ 66,946 $ 124,223 Interest income from licensing $ 5 $ 6 $ 17 $ 20 As of September 30, 2021 and December 31, 2020, the Company had recorded total accounts receivable of $ 28.4 million and $ 19.6 million, respectively, from Novartis Gene Therapies under the March 2014 License, which consisted primarily of unbilled receivables for Zolgensma royalties. Zolgensma royalties receivable recorded as of September 30, 2021 included $ 13.8 million expected to be paid to HCR in accordance with the Royalty Purchase Agreement discussed in Note 6. The Company recognizes royalty revenue from net sales of Zolgensma in the period in which the underlying products are sold by Novartis Gene Therapies, which in certain cases may require the Company to estimate royalty revenue for periods of net sales which have not yet been reported to the Company. Estimated royalties are reconciled to actual amounts reported in subsequent periods and royalty revenues are adjusted, as necessary. Abeona Therapeutics Inc. In November 2018, the Company entered into a license agreement with Abeona (as amended, the November 2018 License), for the treatment of various diseases using the NAV Technology Platform. Pursuant to the November 2018 License, Abeona was required to pay a license fee of $ 8.0 million to the Company no later than April 1, 2020. Abeona failed to make this payment, and in April 2020, the Company delivered to Abeona a notice of its breach of the license agreement and written demand for payment. Upon expiration of the applicable cure period in May 2020, the license agreement terminated. As a result of the termination, Abeona was required to pay an additional $ 20.0 million license fee to the Company within 15 days of the termination date, which otherwise would have been due to the Company in November 2020. As of September 30, 2021, the Company had not received any portion of the $ 28.0 million in license fees due from Abeona under the license agreement. Unpaid balances due under the November 2018 License accrue interest at 1.5 % per month. In May 2020, after the termination of the November 2018 License, Abeona filed a claim in arbitration alleging that the Company had breached certain responsibilities to communicate with Abeona regarding the Company’s prosecution of licensed patents under the November 2018 License. The Company disputed Abeona’s claim and filed a counterclaim in arbitration demanding payment of the $ 28.0 million of unpaid fees from Abeona, plus accrued interest. A binding arbitration was held in March 2021. In July 2021, the arbitration tribunal issued its ruling, which denied Abeona’s claim and upheld the Company’s counterclaim. The arbitration tribunal’s ruling, which was subsequently amended to reflect a minor adjustment in the computation of accrued interest, awarded the Company a total of $33.6 million, which consisted of $ 28.0 million in damages and $ 5.6 million in accrued interest payable to the Company by Abeona. As of October 28, 2021, the Company had not received any portion of the $ 33.6 million arbitration award from Abeona. The Company has filed a petition to confirm the arbitration award and to enter judgment on it in the Supreme Court of the State of New York for New York County. The Company cannot be certain of the precise timing or amount of recovery and will continue to pursue enforcement of the award against Abeona. Abeona has filed an additional claim in a second arbitration to enforce a purported settlement relating to the unpaid fees, which the Company disputes. As of September 30, 2021 and December 31, 2020, the Company had recorded gross accounts receivable of $ 30.1 million from Abeona under the November 2018 License, which consisted of the $ 8.0 million fee due April 1, 2020, the $ 20.0 million fee due within 15 days of the termination of the license agreement in May 2020 and accrued interest on the outstanding balances. While the Company anticipates taking appropriate measures to enforce the aforementioned arbitration award if Abeona does not comply with the tribunal’s ruling, the Company assessed the collectability of the $ 30.1 million due from Abeona as it relates to credit risk. In performing this assessment, the Company evaluated Abeona’s credit profile and financial condition, as well its expectations regarding Abeona’s future cash flows and ability to satisfy this obligation. As a result of its analyses, the Company recorded an allowance for credit losses of $ 13.2 million and $ 7.7 million as of September 30, 2021 and December 31, 2020, respectively, related to the accounts receivable due from Abeona. The Company recorded a provision for credit losses of $ 5.0 million and $ 5.5 million for the three and nine months ended September 30, 2021, respectively, as a result of changes in estimates regarding the allowance during the periods. 17 Table of Contents As of September 30, 2021 and December 31, 2020, the Company had recognized interest income from licensing of $ 2.1 million related to the unpaid license fees from Abeona under the November 2018 License, which is included in the gross accounts receivable balance of $ 30.1 million. In accordance with its interest accrual policy, the Company ceased the recognition of interest income accrued under the license agreement subsequent to the establishment of the allowance for credit losses in the third quarter of 2020. The arbitration tribunal’s ruling in July 2021, as subsequently adjusted, awarded the Company $ 5.6 million in accrued interest payable by Abeona, including $ 3.5 million of interest earned subsequent to the receivable being placed on non-accrual status which has not been recognized in the consolidated financial statements. As of September 30, 2021, the Company had continued to maintain the accounts receivable due from Abeona on non-accrual status and will not recognize any further interest income associated with the accounts receivable unless and until such amounts are deemed to be collectable. Collaboration and License Agreement with AbbVie In September 2021, the Company entered into a Collaboration and License Agreement with AbbVie Global Enterprises Ltd. (AbbVie), a subsidiary of AbbVie Inc., to develop and commercialize RGX-314, the Company’s product candidate for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal diseases (the AbbVie Collaboration and License Agreement). The transaction is contingent upon the satisfaction of customary closing conditions, including the expiration or termination of the applicable waiting or suspension period under the Hart-Scott Rodino Antitrust Improvements Act of 1976, as amended, and any other applicable competition laws. Pursuant to the AbbVie Collaboration and License Agreement, the parties will conduct certain activities for the development of products containing RGX-314 under a development plan determined in accordance with the AbbVie Collaboration and License Agreement. The Company and AbbVie will develop licensed products in the United States, and AbbVie will be responsible for the development of licensed products in specified markets outside the United States. Through December 31, 2022, the Company will be responsible for development expenses for certain ongoing trials of RGX-314 and the parties will share additional development expenses related to RGX-314. Beginning on January 1, 2023, AbbVie will be responsible for the majority of all RGX-314 development expenses. The Company will lead the manufacturing of RGX-314 for clinical development and U.S. commercial supply, and AbbVie will lead manufacturing of RGX-314 for commercial supply outside the United States. Manufacturing expenses will be allocated between the parties in accordance with the terms of the AbbVie Collaboration and License Agreement and mutually agreed supply agreements. In the United States, the Company shall participate in commercialization of licensed products under a commercialization plan to be determined in accordance with the AbbVie Collaboration and License Agreement, and the parties will equally share net profits and net losses associated with commercialization of licensed products in the United States. Outside the United States, AbbVie will be responsible, at its sole cost, for the commercialization of licensed products. The Company will receive an upfront payment of $ 370.0 million from AbbVie in connection with the closing of the AbbVie Collaboration and License Agreement. Additionally, the Company will be eligible to receive up to $ 1.38 billion in development, regulatory and commercial milestone payments, in the aggregate, for the achievement of specified milestones for the licensed products, of which $ 782.5 million are based on development and regulatory milestones, with the remainder based on commercial milestones. The Company will also be eligible to receive tiered royalties on net sales by AbbVie of licensed products outside the United States at percentages in the mid-teens to low twenties, subject to specified offsets and reductions. Royalties will be payable on a product-by-product and country-by-country basis outside the United States commencing on the date of first commercial sale of each licensed product, and ending on the later of (a) expiration of all valid claims of specified licensed patents in such country, (b) expiration of regulatory exclusivity in such country and (c)(x) if such country is in the European Union, 12 years following first commercial sale of such product in such country, or (y) if such country is outside the European Union, 10 years following the first commercial sale of such product in such country (the Royalty Term). The AbbVie Collaboration and License Agreement will remain in effect, unless earlier terminated, on a country-by-country basis until (a) in the case of the United States, the later of (i) the 120th day after any quarter in which no licensed product is being developed or commercialized under such agreement, and (ii) the date that specified licensed patents for a licensed product expire in the United States, and (b) in the case of any country outside the United States, the date the Royalty Term for a licensed product expires in such country. The AbbVie Collaboration and License Agreement contains provisions for termination, including termination for convenience by AbbVie. Contemporaneously with entering into the AbbVie Collaboration and License Agreement, in September 2021, the Company entered into a Sublicense Agreement with AbbVie (the AbbVie Sublicense Agreement) pursuant to which the Company granted AbbVie an exclusive sublicense to exploit licensed products in connection with the AbbVie Collaboration and License Agreement 18 Table of Contents under specified patents licensed to the Company from The Trustees of the University of Pennsylvania. The AbbVie Sublicense Agreement will be coterminous with the AbbVie Collaboration and License Agreement. 9. Stock-based Compensation In January 2021, the Board of Directors authorized an additional 1,499,037 shares to be issued under the 2015 Equity Incentive Plan (the 2015 Plan). As of September 30, 2021, the total number of shares of common stock authorized for issuance under the 2015 Plan and the 2014 Stock Plan (the 2014 Plan) was 13,911,954 , of which 2,392,917 remained available for future grants under the 2015 Plan. Stock-based Compensation Expense The Company’s stock-based compensation expense by award type was as follows (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Stock options $ 8,920 $ 7,880 $ 27,010 $ 23,745 Restricted stock units 698 — 2,093 — Employee stock purchase plan 116 155 543 623 $ 9,734 $ 8,035 $ 29,646 $ 24,368 As of September 30, 2021, the Company had $ 75.7 million of unrecognized stock-based compensation expense related to stock options, restricted stock units and the 2015 Employee Stock Purchase Plan (the 2015 ESPP), which is expected to be recognized over a weighted-average period of 2.5 years. The Company recorded aggregate stock-based compensation expense in the consolidated statements of operations and comprehensive income (loss) as follows (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Research and development $ 4,868 $ 4,110 $ 14,999 $ 12,442 General and administrative 4,866 3,925 14,647 11,926 $ 9,734 $ 8,035 $ 29,646 $ 24,368 Stock Options The following table summarizes stock option activity under the 2014 Plan and 2015 Plan (in thousands, except per share data): Weighted- average Weighted- Remaining average Contractual Aggregate Exercise Life Intrinsic Shares Price (Years) Value (a) Outstanding at December 31, 2020 6,361 $ 31.21 7.2 $ 101,356 Granted 1,373 $ 43.47 Exercised ( 325 ) $ 11.21 Cancelled or forfeited ( 232 ) $ 45.79 Outstanding at September 30, 2021 7,177 $ 33.99 7.0 $ 74,141 Exercisable at September 30, 2021 4,408 $ 28.74 6.0 $ 67,896 Vested and expected to vest at September 30, 2021 7,177 $ 33.99 7.0 $ 74,141 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at the dates reported. 19 Table of Contents The weighted-average grant date fair value per share of options granted during the nine months ended September 30, 2021 was $ 26.28 . During the nine months ended September 30, 2021, the total number of stock options exercised was 325,273 , resulting in total proceeds of $ 3.3 million. The total intrinsic value of options exercised during the nine months ended September 30, 2021 was $ 10.1 million. Restricted Stock Units The following table summarizes restricted stock unit activity under the 2015 Plan (in thousands, except per share data): Weighted- average Grant Date Shares Fair Value Unvested balance at December 31, 2020 — $ — Granted 277 $ 44.17 Vested — $ — Forfeited ( 12 ) $ 44.68 Unvested balance at September 30, 2021 265 $ 44.15 No restricted stock units vested during the three and nine months ended September 30, 2021 and 2020. Employee Stock Purchase Plan In January 2021, the Board of Directors authorized an additional 374,759 shares to be issued under the 2015 ESPP. As of September 30, 2021, the total number of shares of common stock authorized for issuance under the 2015 ESPP was 998,683 , of which 769,174 remained available for future issuance. During the nine months ended September 30, 2021, 53,596 shares of common stock were issued under the 2015 ESPP. 10. Income Taxes The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. Based on the Company’s history of operating losses, including three-year cumulative loss positions as of September 30, 2021 and December 31, 2020, the Company concluded that it is more likely than not that the benefit of its deferred tax assets will not be realized. Accordingly, the Company provided a full valuation allowance for its net deferred tax assets as of September 30, 2021 and December 31, 2020. 11. Related Party Transactions FOXKISER LLP Since 2016, the Company has been party to professional services agreements with FOXKISER LLP (FOXKISER), an affiliate of certain stockholders of the Company and an affiliate of a member of the Company’s Board of Directors, pursuant to which the Company pays a fixed monthly fee in consideration for certain strategic services provided by FOXKISER. Effective January 2019, the Company entered into a new professional services agreement with FOXKISER with similar terms and conditions as the previous agreements. The agreement was amended effective June 2019 to expand the scope of the services provided and increase the monthly fee. Effective August 2020, the agreement was further amended to extend the term of the agreement by two years through December 2022. The agreement may be terminated by either party with six months’ advanced written notice. Expenses incurred under the agreement with FOXKISER were $ 1.2 million and $ 3.6 million for the three and nine months ended September 30, 2021, respectively, and $ 1.2 million and $ 3.6 million for the three and nine months ended September 30, 2020, respectively, and were recorded as research and development expenses in the consolidated statements of operations and comprehensive income (loss). 20 Table of Contents 12. Net Income (Loss) Per Share The computations of basic and diluted net income (loss) per share were as follows (in thousands, except per share data): Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Basic net income (loss) per share: Net income (loss) $ ( 58,405 ) $ 8,791 $ ( 166,183 ) $ ( 65,009 ) Shares used in computation: Weighted-average common shares outstanding 42,629 37,342 42,324 37,234 Basic net income (loss) per share $ ( 1.37 ) $ 0.24 $ ( 3.93 ) $ ( 1.75 ) Diluted net income (loss) per share: Net income (loss) $ ( 58,405 ) $ 8,791 $ ( 166,183 ) $ ( 65,009 ) Shares used in computation: Weighted-average common shares outstanding 42,629 37,342 42,324 37,234 Stock options — 1,529 — — Employee stock purchase plan — 6 — — Weighted-average diluted common shares 42,629 38,877 42,324 37,234 Diluted net income (loss) per share $ ( 1.37 ) $ 0.23 $ ( 3.93 ) $ ( 1.75 ) For periods in which the Company incurred net losses, common stock equivalents were excluded from the calculation of diluted net loss per share as their effect would be anti-dilutive. Accordingly, basic and diluted net loss per share were the same for such periods. The following potentially dilutive common stock equivalents outstanding at the end of the period were excluded from the computations of weighted-average diluted common shares for the periods indicated as their effects would be anti-dilutive (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Stock options issued and outstanding 7,177 4,072 7,177 6,409 Unvested restricted stock units outstanding 265 — 265 — Employee stock purchase plan 15 — 15 21 7,457 4,072 7,457 6,430 13. Supplemental Disclosures Accrued expenses and other current liabilities consisted of the following (in thousands): September 30, 2021 December 31, 2020 Accrued sublicense fees and royalties $ 15,869 $ 12,160 Accrued personnel costs 14,034 13,155 Accrued purchases of property and equipment 8,303 7,853 Accrued external research and development expenses 8,069 9,738 Accrued external general and administrative expenses 2,974 2,865 Accrued income taxes payable — 3,135 Other accrued expenses and current liabilities 445 176 $ 49,694 $ 49,082 21 Table of Contents