ROCKET PHARMACEUTICALS, INC. 10-Q

Item 1. Financial Statements Rocket Pharmaceuticals, Inc. Consolidated Balance Sheets ( $ in thousands, except shares and per share amounts ) March 31 , December 31, 2023 2022 (unaudited) Assets Current assets: Cash and cash equivalents $ 64,579 $ 140,517 Investments 266,505 215,877 Prepaid expenses and other current assets 6,949 7,666 Total current assets 338,033 364,060 Property and equipment, net 30,588 29,009 Goodwill 39,154 39,154 Intangible assets 25,724 25,724 Restricted cash 1,340 1,340 Deposits 459 608 Investments 28,957 43,276 Operating lease right-of-use assets 4,369 1,972 Finance lease right-of-use asset 46,133 46,664 Total assets $ 514,757 $ 551,807 Liabilities and stockholders’ equity Current liabilities: Accounts payable and accrued expenses $ 28,609 $ 36,660 Operating lease liabilities, current 849 773 Finance lease liability, current 1,748 1,736 Total current liabilities 31,206 39,169 Operating lease liabilities, non-current 3,506 1,088 Finance lease liability, non-current 19,294 19,269 Other liabilities 1,875 2,595 Total liabilities 55,881 62,121 Commitments and contingencies (Note 12) Stockholders’ equity: Preferred stock, $ 0.01 par value, authorized 5,000,000 shares: Series A convertible preferred stock; 300,000 shares designated as Series A; 0 shares issued and outstanding - - Series B convertible preferred stock; 300,000 shares designated as Series B; 0 shares issued and outstanding - - Common stock, $ 0.01 par value, 120,000,000 shares authorized; 80,412,194 and 79,123,312 shares issued and 80,409,623 and 79,120,741 shares outstanding at March 31, 2023 and December 31, 2022, respectively 804 791 Treasury stock, at cost, 2,571 common shares at March 31, 2023 and December 31, 2022, respectively ( 47 ) ( 47 ) Additional paid-in capital 1,230,319 1,203,074 Accumulated other comprehensive loss ( 90 ) ( 357 ) Accumulated deficit ( 772,110 ) ( 713,775 ) Total stockholders’ equity 458,876 489,686 Total liabilities and stockholders’ equity $ 514,757 $ 551,807 The accompanying notes are an integral part of these consolidated financial statements. 4 Index Rocket Pharmaceuticals, Inc. Consolidated Statements of Operations ( $ in thousands, except shares and per share amounts ) (unaudited) Three Months Ended March 31, 2023 2022 Revenue $ - $ - Operating expenses: Research and development 46,371 30,794 General and administrative 15,823 11,770 Total operating expenses 62,194 42,564 Loss from operations ( 62,194 ) ( 42,564 ) Interest expense ( 468 ) ( 464 ) Interest and other income, net 1,908 623 Accretion of discount and amortization of premium on investments, net 2,419 ( 577 ) Net loss $ ( 58,335 ) $ ( 42,982 ) Net loss per share - basic and diluted $ ( 0.73 ) $ ( 0.67 ) Weighted-average common shares outstanding - basic and diluted 79,453,519 64,509,721 The accompanying notes are an integral part of these consolidated financial statements. 5 Index Rocket Pharmaceuticals, Inc. Consolidated Statements of Comprehensive Loss (in thousands) (unaudited) Three Months Ended March 31, 2023 2022 Net loss $ ( 58,335 ) $ ( 42,982 ) Other comprehensive loss Net unrealized gain (loss) on investments 272 ( 468 ) Total comprehensive loss $ ( 58,063 ) $ ( 43,450 ) The accompanying notes are an integral part of these consolidated financial statements. 6 Index Rocket Pharmaceuticals, Inc. Consolidated Statements of Stockholders’ Equity For the Three Months Ended March 31, 2023 and 2022 (in thousands except share amounts) (unaudited) Accumulated Additional Other Total Common Stock Treasury Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Stock Capital Income/(Loss) Deficit Equity Balance at December 31, 2022 79,123,312 $ 791 $ ( 47 ) $ 1,203,074 $ ( 357 ) $ ( 713,775 ) $ 489,686 Issuance of common stock pursuant to exercise of stock options 88,429 1 - 1,113 - - 1,114 Issuance of common stock pursuant to vesting of restricted stock units 126,060 1 - ( 1 ) - - - Issuance of common stock pursuant to exercise of warrants 126,093 1 - 6 - - 7 Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs 948,300 10 - 17,212 - - 17,222 Unrealized comprehensive gain on investments - - - - 267 - 267 Stock-based compensation - - - 8,915 - - 8,915 Net loss - - - - - ( 58,335 ) ( 58,335 ) Balance at March 31, 2023 80,412,194 $ 804 $ ( 47 ) $ 1,230,319 $ ( 90 ) $ ( 772,110 ) $ 458,876 Accumulated Additional Other Total Common Stock Treasury Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Stock Capital (Loss) Deficit Equity Balance at December 31, 2021 64,505,889 $ 645 $ - $ 946,152 $ ( 161 ) $ ( 491,912 ) $ 454,724 Issuance of common stock pursuant to exercise of stock options 16,168 - - 76 - - 76 Unrealized comprehensive loss on investments - - - - ( 468 ) - ( 468 ) Stock-based compensation - - - 6,270 - - 6,270 Net loss - - - - - ( 42,982 ) ( 42,982 ) Balance at March 31, 2022 64,522,057 $ 645 $ - $ 952,498 $ ( 629 ) $ ( 534,894 ) $ 417,620 The accompanying notes are an integral part of these consolidated financial statements. 7 Index Rocket Pharmaceuticals, Inc. Consolidated Statements of Cash Flows (in thousands) (unaudited) Three Months Ended March 31, 2023 2022 Operating activities: Net loss $ ( 58,335 ) $ ( 42,982 ) Adjustments to reconcile net loss to net cash used in operating activities: Depreciation and amortization of property and equipment 1,135 767 Amortization of finance lease right of use asset 538 535 Write down of property and equipment, net - 40 Stock-based compensation 8,915 6,270 Amortization of premium and accretion of discount on investments, net ( 2,343 ) 577 Changes in operating assets and liabilities: Prepaid expenses and other assets 866 ( 3,936 ) Accounts payable and accrued expenses ( 7,750 ) ( 491 ) Operating lease liabilities 97 ( 33 ) Finance lease liability 37 45 Other liabilities ( 720 ) ( 15 ) Net cash used in operating activities ( 57,560 ) ( 39,223 ) Investing activities: Purchases of investments ( 96,034 ) ( 143,023 ) Proceeds from maturities of investments 62,335 81,983 Payments made to acquire right of use asset ( 7 ) - Purchases of property and equipment ( 3,015 ) ( 1,955 ) Net cash used in investing activities ( 36,721 ) ( 62,995 ) Financing activities: Issuance of common stock, pursuant to exercise of stock options 1,114 76 Exercise of warrants 7 - Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs 17,222 - Net cash provided by financing activities 18,343 76 Net change in cash, cash equivalents and restricted cash ( 75,938 ) ( 102,142 ) Cash, cash equivalents and restricted cash at beginning of period 141,857 234,037 Cash, cash equivalents and restricted cash at end of period $ 65,919 $ 131,895 Supplemental disclosure of non-cash financing and investing activities: Accrued purchases of property and equipment, ending balance $ 1,794 $ 1,635 Unrealized gain (loss) on investments $ 267 $ ( 468 ) The accompanying notes are an integral part of these consolidated financial statements. 8 Index ROCKET PHARMACEUTICALS, INC. Notes to Consolidated Financial Statements ($ in thousands, except share and per share data) (Unaudited) 1. Nature of Business Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) is a clinical-stage, multi-platform biotechnology company focused on the development of first, only and best in-class gene therapies, with direct on-target mechanism of action and clear clinical endpoints, for rare and devastating diseases. The Company has three clinical-stage ex vivo lentiviral vector (“LV”) programs. These include programs for Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I (“LAD-I”), a genetic disorder that causes the immune system to malfunction and Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-I program produced data read outs in 2022 and regulatory filings in the United States (“U.S.”) and Europe (“EU”) are anticipated in 2023. Additional work on a gene therapy program for the less common FA subtypes C and G is ongoing. In the U.S., the Company also has a clinical stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. The Danon program is currently in an ongoing Phase 1 trial and pivotal Phase 2 study initiation expected in the second quarter of 2023. Additionally, the Company has an AAV vector program targeting Plakophilin-2 Arrhythmogenic Cardiomyopathy (“PKP2-ACM”), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death. This program, also referred to as Pegasus, will be approaching IND submission in the second quarter of 2023. As a result of the Company’s acquisition of Renovacor Inc. (“Renovacor”) (see Note 14 “Renovacor Acquisition”), the Company is able to utilize recombinant AAV9-based gene therapy designed to slow or halt progression of BAG3 Dilated Cardiomyopathy (“DCM”), which is the most common form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart resulting in enlarged heart chambers that are unable to pump blood. The Company has global commercialization and development rights to all of these product candidates under royalty-bearing license agreements. 2. Risks and Liquidity The Company has not generated any revenue and has incurred losses since inception. Operations of the Company are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, having no commercial manufacturing experience, marketing or sales capability or experience, dependency on key personnel, compliance with government regulations and the need to obtain additional financing. Drug candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure, and extensive compliance-reporting capabilities. The Company’s product candidates are in the development and clinical stage. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies. The Company’s consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. The Company has experienced negative cash flows from operations and had an accumulated deficit of $ 772.1 million as of March 31, 2023. As of March 31, 2023, the Company had $ 360.0 million of cash, cash equivalents and short-term and long-term investments. The Company expects such resources will be sufficient to fund the Company’s operating expenses and capital expenditure requirements into the first half of 2025. On February 28, 2022, the Company entered into a sales agreement (the “Sales Agreement”), with Cowen and Company, LLC (“Cowen”), with respect to an at-the-market offering program pursuant to which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock, par value $ 0.01 per share, having an aggregate offering price of up to $ 200 million (the “Shares”) through Cowen as its sales agent. Through March 31, 2023, the Company has sold 4.2 million shares of common stock for net proceeds of $ 63.8 million pursuant to the at-the-market offering program (see Note 8 “Stockholders’ Equity”), including 0.9 million shares for net proceeds of $ 17.2 million during the three months ended March 31, 2023. In the longer term, the future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. The Company’s failure to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies. 9 Index 3. Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited interim consolidated financial statements should be read in conjunction with the Company’s consolidated financial statements for the year ended December 31, 2022 included in the Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on February 28, 2023 (“2022 Form 10-K”). The unaudited interim consolidated financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s consolidated financial position as of March 31, 2023 and the results of its operations and its cash flows for the three months ended March 31, 2023. The financial data and other information disclosed in these consolidated notes related to the three months ended March 31, 2023 and 2022 are unaudited. The results for the three months ended March 31, 2023 are not necessarily indicative of results to be expected for the year ending December 31, 2023 and any other interim periods or any future year or period. Significant Accounting Policies The significant accounting policies used in the preparation of these consolidated financial statements for the three months ended March 31, 2023 are consistent with those disclosed in Note 3 to the consolidated financial statements in the 2022 Form 10-K with most significant policies also being listed here. Principles of Consolidation The consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiaries in conformity with accounting principles generally accepted in the United States (“U.S. GAAP”). All intercompany accounts have been eliminated in consolidation. Use of Estimates The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Significant estimates and assumptions reflected in these consolidated financial statements include but are not limited to goodwill and intangible asset impairments, the accrual of research and development (“R&D”) expenses, the valuation of equity transactions and stock-based awards. Changes in estimates and assumptions are reflected in reported results in the period in which they become known. Actual results could differ from those estimates. Cash, Cash Equivalents and Restricted Cash Cash, cash equivalents and restricted cash consists of bank deposits, certificates of deposit and money market accounts with financial institutions. Cash equivalents are carried at cost which approximates fair value due to their short-term nature and which the Company believes do not have a material exposure to credit risk. The Company considers all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, may exceed federally insured limits. The Company has not experienced any losses in such accounts. Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s operating leases (see Note 12 “Commitments and Contingencies” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit card. Cash, cash equivalents and restricted cash consist of the following: March 31, December 31, 2023 2022 Cash and cash equivalents $ 64,579 $ 140,517 Restricted cash 1,340 1,340 $ 65,919 $ 141,857 Concentrations of credit risk and off-balance sheet risk Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities consist of U.S. Treasury Securities, Commercial Paper and Corporate and Agency Bonds. The Company’s investment policy limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA+/Aa1 rated, thereby reducing credit risk exposure. 10 Index Investments Investments consist of investments in U.S. Treasury Securities, Commercial Paper and Corporate and Agency Bonds. Management determines the appropriate classification of these securities at the time they are acquired and evaluates the appropriateness of such classifications at each balance sheet date. The Company classifies its investments as available-for-sale pursuant to Financial Accounting Standards Board (“FASB”) Accounting Standard Codification (“ASC”) 320, Investments—Debt and Equity Securities. Investments are recorded at fair value, with unrealized gains and losses included as a component of accumulated other comprehensive income (loss) in stockholders’ equity and a component of total comprehensive loss in the consolidated statements of comprehensive loss, until realized. Realized gains and losses are included in investment income on a specific-identification basis. For the three months ended March 31, 2023, there were net unrealized gains on investments of $ 0.3 million. For the three months ended March 31, 2022, there were net unrealized losses on investments of $ 0.5 million. Intangible Assets Intangible assets related to in process research and development (“IPR&D”) projects are considered to be indefinite-lived until the completion or abandonment of the associated R&D efforts. If and when development is complete, which generally occurs if and when regulatory approval to market a product is obtained, the associated assets would be deemed finite-lived and would then be amortized based on their respective estimated useful lives at that point in time. IPR&D intangible assets which are determined to have had a drop in their fair value are adjusted downward and an expense is recognized in R&D expenses in the Consolidated Statements of Operations. These IPR&D intangible assets are tested at least annually or when a triggering event occurs that could indicate a potential impairment based on indicators including progress of R&D activities, changes in projected development of assets, and changes in regulatory environment and future commercial markets. Fair Value Measurements 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. FASB ASC 820, Fair Value Measurements and Disclosures, establishes a hierarchy of inputs 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. • 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 value of the Company’s financial instruments, including cash and cash equivalents, restricted cash, deposits, accounts payable and accrued expenses approximate their respective carrying values due to the short-term nature of most of these instruments. Warrants The Company accounts for stock warrants as either equity instruments, liabilities or derivative liabilities in accordance with ASC Topic 480, Distinguishing Liabilities from Equity (”ASC 480”) and/or ASC Topic 815, Derivatives and Hedging (”ASC 815”), depending on the specific terms of the warrant agreement. Liability-classified warrants are recorded at their estimated fair values at each reporting period until they are exercised, terminated, reclassified or otherwise settled. Changes in the estimated fair value of liability-classified warrants are included in interest and other income in the Company’s consolidated statement of operations. 11 Index Stock-Based Compensation The Company measures the compensation expense of employee and non-employee services received in exchange for an award of equity instruments based on the fair value of the award on the grant date. That cost is recognized over the requisite service period of the awards on a straight-line basis with forfeitures recognized as they occur. The Company classifies stock-based compensation expense in its consolidated statements of operations in the same manner in which the award recipient’s payroll costs and services are classified or in which the award recipient’s service payments are classified. Income Taxes In May 2022, the Company received a notice from the New York City Department of Finance regarding an audit of the NYC Biotechnology Credit for the tax periods ended December 31, 2018 through December 31, 2020, which is ongoing as of March 31, 2023. Recent Accounting Pronouncements There were no recent accounting pronouncements that impacted the Company, or which had a significant effect on the consolidated financial statements. 4. Fair Value of Financial Instruments Items measured at fair value on a recurring basis are the Company’s investments. The following table sets forth the Company’s financial instruments that were measured at fair value on a recurring basis by level within the fair value hierarchy: Fair Value Measurements as of March 31, 2023 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market mutual funds $ 31,353 $ - $ - $ 31,353 Corporate Bonds - 3,778 - 3,778 United States Treasury securities 7,670 - - 7,670 39,023 3,778 - 42,801 Investments: Commercial Paper - 5,147 - 5,147 United States Treasury securities 228,443 - - 228,443 Corporate Bonds - 54,159 - 54,159 Agency Bonds - 7,713 - 7,713 228,443 67,019 - 295,462 Total assets $ 267,466 $ 70,797 $ - $ 338,263 Liabilities: Warrant liability $ - $ - $ 815 $ 815 Total liabilities $ - $ - $ 815 $ 815 Fair Value Measurements as of December 31, 2022 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents: Money market mutual funds $ 90,527 $ - $ - $ 90,527 Commercial Paper - 3,899 - 3,899 United States Treasury Securities 3,848 - - 3,848 Corporate Bonds - 8,618 - 8,618 94,375 12,517 - 106,892 Investments: Commercial Paper - 1,151 - 1,151 United States Treasury securities 189,444 - - 189,444 Corporate Bonds - 60,905 - 60,905 Agency Bonds - 7,653 - 7,653 189,444 69,709 - 259,153 Total assets $ 283,819 $ 82,226 $ - $ 366,045 Liabilities: Warrant liability $ - $ - $ 1,512 $ 1,512 Total liabilities $ - $ - $ 1,512 $ 1,512 12 Index The Company classifies its money market mutual funds and U.S. Treasury securities as Level 1 assets under the fair value hierarchy, as these assets have been valued using quoted market prices in active markets without any valuation adjustment. The Company classifies its Commercial Paper and Corporate and Agency Bonds as Level 2 assets as these assets are not traded in an active market and have been valued through a third-party pricing service based on quoted prices for similar assets. The reconciliation of the Company’s warrant liability, which is recorded as part of Other Liabilities in the consolidated balance sheets, measured at fair value on a recurring basis using unobservable inputs (Level 3) is as follows: Warrant Liability Balance, December 31, 2022 $ 1,512 Fair value adjustments ( 697 ) Balance, March 31, 2023 $ 815 The Company utilizes a Black-Scholes model to value the warrant liability (see Note 10 “Warrants”) at each reporting period, with changes in fair value recognized in the consolidated statements of operations. The estimated fair value of the warrant liability is determined using Level 3 inputs. Inherent in an options pricing model are assumptions related to expected share-price volatility, expected life, risk-free interest rate and dividend yield. The Company estimates the expected volatility of its common stock based on historical volatility of a peer group, considering the expected remaining life of the warrants. The risk-free interest rate is based on the U.S. Treasury zero-coupon yield curve on the valuation date for a maturity similar to the expected remaining life of the warrants. The expected life of the warrants is assumed to be equivalent to their remaining contractual term. The dividend rate is based on the historical rate, which the Company anticipates will remain at zero . The fair value of the warrant liability has been estimated with the following assumptions: March 31, 2023 December 31, 2022 Stock price $ 17.13 $ 18.39 Exercise price $ 65.23 $ 65.23 Expected volatility 67.38 % 71.25 % Risk-free interest rate 4.04 % 4.14 % Expected dividend yield - - Expected life (years) 2.07 2.39 Fair value per warrant $ 1.32 $ 2.45 5. Property and Equipment, Net The Company’s property and equipment consisted of the following: March 31, December 31, 2023 2022 Laboratory equipment $ 23,317 $ 21,905 Machinery and equipment 11,443 11,326 Computer equipment 244 244 Furniture and fixtures 2,216 2,135 Leasehold improvements 1,694 589 Internal use software 1,903 1,903 40,817 38,102 Less: accumulated depreciation and amortization ( 10,229 ) ( 9,093 ) $ 30,588 $ 29,009 During the three months ended March 31, 2023 and 2022, the Company recognized $ 1.1 million and $ 0.8 million of depreciation and amortization expense, respectively. 13 Index 6. Intangible Assets and Goodwill The Company’s indefinite lived intangible assets consists of acquired IPR&D asset and a mice colony model received from the acquisition of Renovacor. Intangible assets as of March 31, 2023 and December 31, 2022 are summarized as follows: Gross Carrying Value Accumulated Amortization Intangible Assets, Net In process research & development $ 25,150 $ - $ 25,150 Mice colony model 574 - 574 Total intangible assets $ 25,724 $ - $ 25,724 The gross carrying value of intangible assets was due to the acquisition of Renovacor (see Note 14 “Renovacor Acquisition”). The carrying value of Goodwill was $ 39.2 million as of March 31, 2023 and included $ 8.3 million as a result of the acquisition of Renovacor (see Note 14 “Renovacor Acquisition”). 7. Accounts Payable and Accrued Expenses As of March 31, 2023 and December 31, 2022, the Company’s accounts payable and accrued expenses consisted of the following: March 31 , December 31, 2023 2022 Research and development $ 17,536 $ 19,100 Employee compensation 4,199 10,006 Property and equipment 1,794 2,095 Professional fees 2,813 1,436 Acquisition related expenses - 1,153 Government grant payable 597 597 Other 1,670 2,273 $ 28,609 $ 36,660 8. Stockholders’ Equity At-the-Market Offering Program On February 28, 2022, the Company entered into the Sales Agreement with Cowen with respect to an at-the-market offering program pursuant to which the Company may offer and sell, from time to time at its sole discretion, shares through Cowen as its sales agent. The shares to be offered and sold under the Sales Agreement, if any, will be offered and sold pursuant to the Company’s shelf registration statement on Form S- 3. The Company filed a prospectus supplement with the SEC on February 28, 2022 in connection with the offer and sale of the shares pursuant to the Sales Agreement. The Company will pay Cowen a cash commission of 3.0 % of gross proceeds from the sale of the shares pursuant to the Sales Agreement. The Company has provided Cowen with customary indemnification and contribution rights. The Company reimbursed Cowen for certain expenses incurred in connection with the Sales Agreement. Through March 31, 2023, the Company sold 4.2 million shares under the at-the-market offering program for gross proceeds of $ 65.8 million, less commissions of $ 2.0 million for net proceeds of $ 63.8 million. During the three months ended March 31, 2023, the Company sold 0.9 million shares under the at-the-market offering program for gross proceeds of $ 17.8 million, less commission of $ 0.6 million for net proceeds of $ 17.2 million. 14 Index 9. Stock Based Compensation Stock Option Valuation The weighted average assumptions that the Company used in the Black-Scholes pricing model to determine the fair value of the stock options granted to employees, non-employees and directors were as follows: Three Months Ended March 31, 2023 2022 Risk-free interest rate 4.02 % 1.88 % Expected term (in years) 5.88 5.86 Expected volatility 73.54 % 74.07 % Expected dividend yield 0.00 % 0.00 % Exercise price $ 20.17 $ 17.85 Fair value of common stock $ 20.17 $ 17.85 The following table summarizes stock option activity for the three months ended March 31, 2023, under the Second Amended and Restated 2014 Stock Option and Incentive Plan: Weighted Weighted Average Average Aggregate Number of Exercise Contractual Intrinsic Shares Price Term (Years) Value Outstanding as of December 31, 2022 13,138,870 $ 14.52 5.46 $ 118,767 Granted 1,792,097 20.36 6.86 Exercised ( 88,429 ) 12.59 631 Cancelled ( 191,148 ) 33.99 Outstanding as of March 31, 2023 14,651,390 $ 14.99 5.85 $ 97,263 Options vested and exercisable as of March 31, 2023 10,586,141 $ 12.60 4.52 $ 92,808 Options unvested as of March 31, 2023 4,065,249 $ 21.24 9.32 $ 4,455 The weighted average grant-date fair value per share of stock options granted during the three months ended March 31, 2023, and 2022 was $ 13.50 and $ 11.60 , respectively. The total fair value of options vested during the three months ended March 31, 2023 and 2022 was $ 11.4 million and $ 12.5 million, respectively. Restricted Stock Units (“RSU”) The following table summarizes the Company’s RSU activity for the three months ended March 31, 2023: Weighted Average Number of Grant Date Shares Fair Value Unvested as of December 31 , 2022 992,874 $ 16.49 Granted 764,204 20.23 Vested (1) ( 126,145 ) 17.37 Forfeited ( 8,476 ) 17.19 Unvested as of March 31 , 2023 1,622,457 $ 18.18 (1) Common stock issued is net of 85 shares related to taxes. 15 Index Stock-based Compensation Stock-based compensation expense recognized by award type was as follows: Three Months Ended March 31, 2023 2022 Stock options $ 6,985 $ 5,961 Restricted stock units 1,930 309 Total stock-based compensation expense $ 8,915 $ 6,270 Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows : Three Months Ended March 31, 2023 2022 Research and development $ 3,819 $ 2,318 General and administrative 5,096 3,952 Total stock-based compensation expense $ 8,915 $ 6,270 As of March 31, 2023, the Company had an aggregate of $ 75.4 million of unrecognized stock-based compensation expense related to both stock options and RSU grants, which is expected to be recognized over the weighted average period of 1.52 years. 10 . Warrants A summary of the warrants outstanding as of March 31, 2023 is as follows: Exercise Price Outstanding Grant/Assumption Date Expiration Date 24.42 7,051 June 28, 2013 June 28, 2023 57.11 603,386 December 21, 2020 December 21, 2030 33.63 301,291 August 9, 2021 August 9, 2031 22.51 153,155 December 17, 2021 December 17, 2031 22.51 153,155 December 17, 2021 December 17, 2031 65.23 617,050 December 1, 2022 April 23, 2025 65.23 760,086 December 1, 2022 December 1, 2026 Total 2,595,174 The following table below is a summary of changes in warrants to purchase common stock for the three months ended March 31, 2023: Number of Warrant Shares Outstanding and Exercisable Exercise Price per Share Balance as of December 31, 2022 2,721,267 Granted - Exercised ( 126,093 ) $ 0.06 Balance as of March 31, 2023 2,595,174 Assumed Renovacor Public Warrants In conjunction with the Renovacor acquisition (see Note 14 “Renovacor Acquisition”), Rocket assumed pre-acquisition public warrants (“Public Warrants”) that were converted into Rocket warrants with a right to purchase 760,086 of Rocket common shares at an exercise price of $ 65.23 per share. The Company determined that the Public Warrants met all of the criteria for equity classification. Accordingly, upon closing of the Merger, the Public Warrants were recorded as a component of additional paid-in capital of $ 3.4 million. 16 Index Assumed Renovacor Private Warrants In conjunction with the Renovacor acquisition (see Note 14 “Renovacor Acquisition”), Rocket assumed pre-acquisition private warrants (“Private Warrants”) that were converted into Rocket warrants with a right to purchase 617,050 of Rocket common shares at an exercise price of $ 65.23 per share. The Company determined that the Private Warrants did not meet all of the criteria for equity classification. Accordingly, the Company classifies the Private Warrants as derivative liabilities in its consolidated balance sheets. The Company measures the fair value of the warrants at the end of each reporting period and recognizes changes in the fair value from the prior period in the Company’s operating results for the current period. See Note 4 for discussion of fair value measurement of the warrant liabilities. Assumed Renovacor Pre-Funded Warrants In conjunction with the Renovacor acquisition (see Note 14 “Renovacor Acquisition”), Rocket assumed pre-funded warrants (“Pre-Funded Warrants”) that were converted into Rocket warrants with a right to purchase 126,093 of Rocket common shares at an exercise price of $ 0.06 per share. These warrants were exercised in January 2023. 11. Net Loss Per Share Basic and diluted net loss per share attributable to common stockholders was calculated as follows: Three Months Ended March 31, 2023 2022 Numerator: Net loss attributable to common stockholders $ ( 58,335 ) $ ( 42,982 ) Denominator: Weighted-average common shares outstanding - basic and diluted 79,453,519 64,509,721 Net loss per share attributable to common stockholders - basic and diluted $ ( 0.73 ) $ ( 0.67 ) The Company excluded the following potential shares of common stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three Months Ended March 31, 2023 2022 Warrants exercisable for common shares 2,595,174 1,218,038 Restricted stock units convertible for common shares 1,622,457 457,709 Options to purchase common shares 14,651,390 12,047,299 18,869,021 13,723,046 12. Commitments and Contingencies The Company determines if an arrangement is a lease at inception. Operating and finance leases are presented in the Company’s consolidated balance sheet as right-of-use assets from leases, current lease liabilities and long-term lease liabilities. Certain of the Company’s lease agreements contain renewal options; however, the Company does not recognize right-of-use assets or lease liabilities for renewal periods unless it is determined that the Company is reasonably certain of renewing the lease at inception or when a triggering event occurs. As the Company’s leases do not provide an implicit rate, the Company estimated the incremental borrowing rate in calculating the present value of the lease payments using an estimate of the Company’s collateralized borrowing rate for debt with a similar term. The Company has utilized its incremental borrowing rate based on the long-term borrowing costs of comparable companies in the biotechnology industry. Since the Company elected to account for each lease component and its associated non-lease components as a single combined lease component, all contract consideration was allocated to the combined lease component. Some of the Company’s lease agreements contain rent escalation clauses (including index-based escalations). For operating leases, the Company recognizes the minimum rental expense on a straight-line basis based on the fixed components of a lease arrangement. The Company will amortize this expense over the term of the lease beginning with the lease commencement date. Variable lease components represent amounts that are not fixed in nature and are not tied to an index or rate and are recognized as incurred. 17 Index Finance Lease The Company has a lease for a facility in Cranbury, New Jersey, consisting of 103,720 square feet of space including areas for offices, process development, research, and development laboratories and 50,000 square feet dedicated to AAV Current Good Manufacturing Practice (“cGMP”) manufacturing facilities to support the Company’s pipeline (such lease, as amended, the “NJ Lease Agreement”). The NJ Lease Agreement has a 15 -year term from September 1, 2019, with an option to renew for two consecutive five-year renewal terms. Estimated rent payments for the NJ Lease Agreement are $ 1.2 million per annum, payable in monthly installments, depending upon the nature of the leased space, and subject to annual base rent increases of 3 %. The total commitment under the lease is estimated to be approximately $ 29.3 million over the 15 -year term of the lease. The Company paid a cash security deposit of $ 0.3 million to the landlord in connection with the NJ Lease Agreement which has been reflected in deposits in the consolidated balance sheets as of March 31, 2023 and December 31, 2022. Operating Leases On June 7, 2018, the Company entered into a three-year lease agreement for office space in the Empire State Building in New York, NY (the “ESB Lease Agreement”). In connection with the ESB Lease Agreement, the Company established an irrevocable standby letter of credit (the “Empire LOC”) for $ 0.9 million. On March 26, 2021, the Company entered in Amendment No. 1 to the ESB Lease Agreement (“ESB Lease Amendment”) that extended the term of the lease agreement to June 30, 2024 , reduced the rent payments going forward, and reduced the Empire LOC to $ 0.8 million. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary and expires August 29, 2024 . The Company has a certificate of deposit of $ 0.8 million with a bank as collateral for the Empire LOC which is classified as part of restricted cash in the consolidated balance sheets as of March 31, 2023 and December 31, 2022 . On January 4, 2018, in connection with the Reverse Merger with Inotek , the Company assumed an operating lease for Inotek’s former headquarters in Lexington, Massachusetts, with a term which ended on February 28, 2023 . In July 2018, the Company signed an agreement to sublease a portion of the Lexington, Massachusetts space and in September 2018, the Company signed an agreement to sublease the remaining portion of the Lexington, Massachusetts space. Rental income received under the sublease agreement totaled $ 0.1 million for the three months ended March 31, 2023 and 2022. These amounts are netted against rent expense in the consolidated statements of operations for the three months ended March 31, 2023 and 2022. A security deposit of $ 0.2 million was returned to the Company in April 2023 and is reflected in other current assets as of March 31, 2023. On December 1, 2022, in connection with the Renovacor acquisition (see Note 14 “Renovacor Acquisition ”), Rocket acquired the Renovacor operating leases for space at facilities in Hopewell, New Jersey and Cambridge, Massachusetts with remaining lease terms of approximately 10.25 and 1.3 years, respectively. As of March 31, 2023, lease commencement dates have occurred for all leases and the Company recognized total right-of-use assets of $ 3.8 million with corresponding total lease liabilities of $ 3.6 million. The Company intends to sublease both premises through the remainder of their lease terms. Rent expense was $ 0.4 million and $ 0.3 million for the three months ended March 31, 2023 and 2022, respectively. The total restricted cash balance for the Company’s operating and finance leases as of each of March 31, 2023 and December 31, 2022 was $ 0.8 million . Lease cost March 31, 2023 Operating lease cost $ 358 Finance lease cost Amortization of right of use assets 538 Interest on lease liabilities 468 Total lease cost $ 1,364 The following table summarizes the future lease payments of the Company’s operating and finance lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating and finance lease liabilities as of March 31, 2023: Fiscal Year Ending December 31, March 31, 2023 2023 (nine months) 869 2024 798 2025 538 2026 545 2027 506 Thereafter 2,941 Total lease payments $ 6,197 Less: interest ( 1,842 ) Total operating lease liabilities $ 4,355 18 Index Fiscal Year Ending December 31, March 31, 2023 2023 (nine months) 1,305 2024 1,791 2025 1,856 2026 1,912 2027 1,969 Thereafter 43,032 Total lease payments $ 51,865 Less: interest ( 30,823 ) Total finance lease liability $ 21,042 Leases March 31, 2023 Operating right-of-use assets $ 4,369 Operating current lease liabilities 849 Operating noncurrent lease liabilities 3,506 Total operating lease liabilities $ 4,355 Finance right-of-use assets $ 46,133 Finance current lease liability 1,748 Finance noncurrent lease liability 19,294 Total finance lease liability $ 21,042 Other information Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 261 Cash flows from finance lease $ 431 Weighted-average remaining lease term - operating leases 8.2 years Weighted-average remaining lease term - finance lease 21.4 years Weighted-average discount rate - operating leases 8.08 % Weighted-average discount rate - finance lease 8.96 % Litigation From time to time, the Company may be subject to various legal proceedings and claims that arise in the ordinary course of its business activities. Although the results of litigation and claims cannot be predicted with certainty, the Company does not believe it is party to any other claim or litigation the outcome of which, if determined adversely to the Company, would individually or in the aggregate be reasonably expected to have a material adverse effect on its business. Regardless of the outcome, litigation can have an adverse impact on the Company because of defense and settlement costs, diversion of management resources and other factors. Indemnification Arrangements Pursuant to its bylaws and as permitted under Delaware law, the Company has indemnification obligations to directors, officers, employees or agents of the Company or anyone serving in these capacities. The maximum potential amount of future payments the Company could be required to pay is unlimited. The Company has insurance that reduces its monetary exposure and would enable it to recover a portion of any future amounts paid. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal. Throughout the normal course of business, the Company has agreements with vendors that provide goods and services required by the Company to run its business. In some instances, vendor agreements include language that requires the Company to indemnify the vendor from certain damages caused by the Company’s use of the vendor’s goods and/or services. The Company has insurance that would allow it to recover a portion of any future amounts that could arise from these indemnifications. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal. 19 Index 13. Agreements Related to Intellectual Property The Company, directly and through its subsidiary Spacecraft Seven, LLC, has various license and research and collaboration arrangements. The transactions principally resulted in the acquisition of rights to intellectual property which is in the preclinical phase and has not been tested for safety or feasibility. In all cases, the Company did not acquire tangible assets, processes, protocols, or operating systems. The Company expenses the acquired intellectual property rights as of the acquisition date on the basis that the cost of intangible assets purchased from others for use in research and development activities has no alternative future uses. 14. Renovacor Acquisition On September 19, 2022, the Company entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Renovacor, a Delaware corporation pursuant to which, on December 1, 2022, the Company acquired Renovacor (the “Renovacor Acquisition”). On December 1, 2022, pursuant to the terms of the Merger Agreement, (i) Merger Sub I merged with and into the Company (the “First Merger”) and (ii) the Company, as the surviving company of the First Merger merged with and into Merger Sub II, with Merger Sub II surviving the Second Merger. Subject to the terms and conditions of the Merger Agreement, at the closing of the Renovacor Acquisition each share of Renovacor’s common stock outstanding immediately prior to the effective time of the First Merger were canceled and converted into the right to receive 0.1763 (the “Exchange Ratio”) of fully paid and non-assessable shares of the Company common stock, which was determined on the basis of the exchange formula set forth in the Merger Agreement that was subject to adjustment depending on the level of the Renovacor’s net cash at the closing. Prior to the market opening on December 1, 2022, Renovacor shares ceased to trade on NYSE and upon the closing of the acquisition, Renovacor’s outstanding common stock were converted into 3,391,976 shares of Rocket common stock. Total consideration for the Renovacor Acquisition was $ 72.3 million, consisting of $ 62.4 million for common stock outstanding, $ 2.7 million for the portion of equity compensation attributable to the pre-combination service period, and $ 7.2 million for assumed warrants. The consideration was based on the estimated fair values on the acquisition date of (i) 3,391,976 common shares issued for shares outstanding for common shares of Renovacor, (ii) estimated fair value of employee stock options to acquire 367,852 common shares of the Company, (iii) 28,798 common shares issued for employee time-vesting RSUs, and (iv) warrants to acquire 1,503,229 common shares (see Note 10 “Warrants”). The total consideration for the acquisition of Renovacor of $ 72.3 million consisted of the following: Shares Value Total Stock consideration 3,391,976 $ 18.39 $ 62,378 Cash consideration (1) 29 Stock options 367,852 2,163 Time-vesting RSUs 28,798 512 Assumed warrants (2) 1,503,229 7,183 Total consideration 5,291,855 $ 72,265 (1) Represents consideration paid for cash in lieu of fractional shares. (2) Assumed Renovacor Warrants of $ 7,183 with $ 5,671 classified as equity and $ 1,512 classified as liabilities. The acquisition has been accounted for as a business combination using the acquisition method of accounting which requires that assets acquired and liabilities assumed be recognized at their fair values as of the acquisition date and that the fair value of acquired IPR&D assets are classified as indefinite-life assets until the successful completion or abandonment of the associated research and development efforts. The preliminary purchase price allocation resulted in the following amounts being allocated to the assets acquired and liabilities assumed at the acquisition date based on their respective preliminary fair values summarized below: Working capital (1) $ ( 5,210 ) Cash and cash equivalents 42,755 Property and equipment 1,414 Operating lease right-of-use assets 1,161 Other non-current assets 113 IPR&D 25,150 Other intangible asset 574 Operating lease liability ( 970 ) Deferred tax liability ( 1,061 ) Net assets acquired 63,926 Goodwill 8,339 Purchase consideration $ 72,265 (1) Includes other receivables, prepaid expenses, account payable and accrued liabilities. 20 Index The fair value assigned to acquired IPR&D was based on the present value of expected after-tax cash flows attributable to Renovacor’s most advanced AAV-based gene therapy targeting BAG3-DCM. The present value of expected after-tax cash flows was determined by estimating the after-tax costs to complete development into a commercially viable product, estimating future revenue and ongoing expenses to produce, and discounting the resulting net cash flows to present value. The cost and revenue projections used were reduced based on the assessed probabilities of different stages of development. Acquired IPR&D will be accounted for as an indefinite-lived intangible asset until regulatory approval in a major market or discontinuation of development. The excess of purchase price over the fair value of amounts assigned to identifiable assets acquired and liabilities assumed represents the goodwill amount of $ 8.3 million resulting from the acquisition. The goodwill recorded as part of the acquisition is primarily attributable to the broadening of the Company’s portfolio and research capabilities, deferred taxes and the assembled workforce. The goodwill attributable to the acquisition has been recorded as a non-current asset in the Company’s consolidated balance sheet and is not amortized, but subject to review for impairment annually. 15. CIRM Grants LAD-1 CIRM Grant On April 30, 2019, the California Institute for Regenerative Medicine (“CIRM”) awarded the Company up to $ 7.5 million under a CLIN2 grant award to support the clinical development of its LV-based gene therapy for RP-L201. Proceeds from the grant will help fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients enrolled at the U.S. clinical site, University of California, Los Angeles (“UCLA”) Mattel Children’s Hospital, led by principal investigator Donald Kohn, M.D., UCLA Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. Through March 31, 2023, the Company has received $ 5.9 million in total RP-L201 grants from CIRM. No additional milestones were achieved as of March 31, 2023. 16. Related Party Transactions In October 2020 , the Company entered into a consulting agreement with the spouse of one of the Company’s executive officers for information technology advisory services. In exchange for the services provided under the agreement, the Company granted 10,000 restricted stock units which vest over a three-year period. 17. 401(k) Savings Plan The Company has a defined contribution savings plan (the “Plan”) under Section 401(k) of the Internal Revenue Code of 1986. This Plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. Company contributions to the Plan may be made at the discretion of the Company’s Board of Directors. The Company has elected the safe harbor match of 4 % of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the three months ended March 31, 2023 and 2022, was $ 0.3 million and $ 0.2 million, respectively. 21 Index