ROCKET PHARMACEUTICALS INC
For the period ended Sep 30, 2024. Filed Nov 7, 2024.
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Sep 30, 2024 · Filed Nov 7, 2024
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Notes to Consolidated Financial Statements (unaudited)
Notes to Consolidated Financial Statements
($ in thousands, except share and per share data) (Unaudited)
Nature of Business
Rocket Pharmaceuticals, Inc. is a fully integrated, late-stage 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 hasthree clinical-stageex vivo lentiviral vector programs, which include programs for:
Fanconi Anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102);
Leukocyte Adhesion Deficiency-I, a genetic disorder that causes the immune system to malfunction (RP-L201); and
Pyruvate Kinase Deficiency, a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301).
In September 2023, the FDA accepted the BLA and granted priority review for RP-L201 for the treatment of severe LAD-I. On June 28, 2024, the Company announced that the FDA had issued a CRL in response to the BLA wherein the FDA requested limited additional CMC information to complete its review. The Company is in the process of providing the additional requested information and approval is expected in 2025. Treatments in the FA Phase 2 studies were completed in 2023 and submission of a BLA on a rolling review basis was initiated on September 26, 2024. In April 2024, the EMA accepted our MAA for RP-L102. Additional work on a gene therapy program for the less common FA subtypes C and G is ongoing.
The Company also hastwo clinical stage andone pre-clinical stagein vivo adeno-associated virus programs, which include programs for:
Danon Disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. The DD program is currently in an ongoing Phase 2 trial (RP-A501);
Plakophilin-2 Arrhythmogenic Cardiomyopathy, 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 (RP-A601); and
BAG3 Dilated Cardiomyopathy, 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 utilizes recombinant AAV9-based gene therapy designed to slow or halt progression of BAG3-DCM.
In September 2023, the Company announced its alignment with the FDA on its pivotal study design for RP-A501 in Danon Disease and completion of enrollment in this study was announced in September 2024, dosing and follow-up is ongoing. The Company has received FDA clearance of an IND for RP-A601, and has initiated a Phase 1 study for this program. For the BAG3 program, nonclinical and IND enabling studies are ongoing. Submission of the IND is anticipated in the first half of 2025.
The Company has global commercialization and development rights to all of these product candidates under royalty-bearing license agreements.
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 $1.16 billion as of September 30, 2024. As of September 30, 2024, the Company had $235.7 million of cash, cash equivalents and investments. The Company expects that in accordance with the current operating plan, which includes cost optimization and savings, such resources will be sufficient to fund the Company’s operating expenses and capital expenditure requirements into 2026.
On February 28, 2022, the Company entered into a sales agreement (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 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. On September 12, 2023, the Company and Cowen entered into an amendment (the “Amended Sales Agreement”) pursuant to which the aggregate offering amount available under the at-the-market offering program was reduced to $180.0 million. Through September 30, 2024, 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”). The Company did not sell any shares under the at-the-market offering program during thenine months ended September 30, 2024.
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.
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, 2023 included in the Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 27, 2024. 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 September 30, 2024 and the results of its operations and its cash flows for the nine months ended September 30, 2024. The financial data and other information disclosed in these consolidated notes related to the three and nine months ended September 30, 2024 and 2023 are unaudited. The results for the three and nine months ended September 30, 2024 are not necessarily indicative of results to be expected for the year ending December 31, 2024 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 and nine months ended September 30, 2024 are consistent with those disclosed in Note 3 to the consolidated financial statements in the 2023 Form 10-K with the 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 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 R&D expenses, and 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, 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 “Leases” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit cards. Cash, cash equivalents and restricted cash consist of the following:
| September 30, 2024 | December 31, 2023 | |
|---|---|---|
| Cash and cash equivalents | $65,599 | $55,904 |
| Restricted cash | 1,362 | 1,372 |
| Total cash, cash equivalents and restricted cash | $66,961 | $57,276 |
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 and Corporate 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-/Aa3 rated, thereby reducing credit risk exposure.
Investments
Investments consist of U.S. Treasury Securities and Corporate 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 FASB 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. The Company estimates expected credit losses for investments when unrealized losses exist. Unrealized losses that are credit related are recognized in the Company’s Consolidated Statements of Operations and unrealized losses that are not credit related are recognized in accumulated other comprehensive income (loss). For the three and nine months ended September 30, 2024 and 2023, there wereno unrealized losses that were credit related. For thethree and nine months ended September 30, 2024, there was$0.4 million of net unrealized gains on investments and$0.1 million of net unrealized losses on investments, respectively. For the three and nine months ended September 30, 2023, there was $0.2 million of net unrealized gains on investments and $0.2 million of net unrealized losses on investments, respectively.
Intangible Assets
Intangible assets related to in process research and development 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 decrease 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. If a triggering event occurs that would indicate a potential impairment, the Company will perform a quantitative analysis to determine whether it is more likely than not that the fair value is below carrying amount.
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 FASB ASC 480, Distinguishing Liabilities from Equity and/or FASB ASC 815, Derivatives and Hedging, 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 Statements of Operations.
Stock-Based Compensation
The Company issues stock-based awards to employees and non-employees, generally in the form of stock options, RSUs and PSUs.
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 of stock options and RSUs is recognized over the requisite service period of the awards on a straight-line basis with forfeitures recognized as they occur. The vesting condition for PSUs is performance based and the cost of PSUs is recognized when it is likely that the performance goals associated with the PSUs will be achieved and the awards will vest.
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.
Recent Accounting Pronouncements
Accounting Pronouncements Not Adopted as of September 30, 2024
ASU 2023-09: Income Taxes Topic 740 - Improvements to Income Tax Disclosures. This update standardizes categories for the effective tax rate reconciliation, requires disaggregation of income taxes and additional income tax-related disclosures. This update is required to be effective for the Company for fiscal periods beginning after December 15, 2024. As this accounting standard is not expected to change the Company’s accounting, it is not expected to have a material impact on the Company’s consolidated financial statements.
ASU 2023-07: Segment Reporting Topic 280 - Improvements to Reportable Segment Disclosures. This update requires expanded annual and interim disclosures for significant segment expenses that are regularly provided to the chief operating decision maker and included within each reported measure of segment profit or loss. This update will be effective for fiscal years beginning after December 15, 2023, and is to be applied retrospectively to all periods presented in the financial statements. Early adoption is permitted. As this accounting standard is not expected to change the Company’s accounting, it is not expected to have a material impact on the Company’s consolidated financial statements.
Fair Value of Financial Instruments
Items measured at fair value on a recurring basis are the Company’s investments and warrant liability. 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 September 30, 2024 Using: | |||||||
|---|---|---|---|---|---|---|---|
| Level 1 · Level 2 · Level 3 · Total | |||||||
| Assets: | |||||||
| Cash equivalents: | |||||||
| Money market mutual funds | $53,993 | $ | - | $ | - | $53,993 | |
| 53,993 | - | - | 53,993 | ||||
| Investments: | |||||||
| U.S. Treasury securities | - | 157,563 | - | 157,563 | |||
| Corporate bonds | - | 12,500 | - | 12,500 | |||
| - | 170,063 | - | 170,063 | ||||
| Total assets | $53,993 | $170,063 | $ | - | $224,056 | ||
| Liabilities: | |||||||
| Warrant liability | $ | - | $ | - | $1 | $1 | |
| Total liabilities | $ | - | $ | - | $1 | $1 | |
| Fair Value Measurements as of December 31, 2023 Using: | |||||||
| Level 1 · Level 2 · Level 3 · Total | |||||||
| Assets: | |||||||
| Cash equivalents: | |||||||
| Money market mutual funds | $50,737 | $ | - | $ | - | $50,737 | |
| U.S. Treasury securities | - | 2,487 | - | 2,487 | |||
| 50,737 | 2,487 | - | 53,224 | ||||
| Investments: | |||||||
| U.S. Treasury securities | - | 312,696 | - | 312,696 | |||
| Corporate bonds | - | 38,895 | - | 38,895 | |||
| - | 351,591 | - | 351,591 | ||||
| Total assets | $50,737 | $354,078 | $ | - | $404,815 | ||
| Liabilities: | |||||||
| Warrant liability | $ | - | $ | - | $1,876 | $1,876 | |
| Total liabilities | $ | - | $ | - | $1,876 | $1,876 | |
The Company classifies its money market mutual funds 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 U.S. Treasury Securities and Corporate 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, 2023 | $1,876 |
| Fair value adjustments | (1,875) |
| Balance, September 30, 2024 | $1 |
The Company utilizes a Black-Scholes option pricing 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 a Black-Scholes option 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 its common stock, 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:
| September 30, 2024 | December 31, 2023 | |
|---|---|---|
| Stock price | $18.47 | $29.50 |
| Exercise price | $65.23 | $65.23 |
| Expected volatility | 50.38% | 68.83% |
| Risk-free interest rate | 4.33% | 4.70% |
| Expected dividend yield | - | - |
| Expected life (years) | 0.56 | 1.31 |
| Fair value per warrant | $0.00 | $3.04 |
Property and Equipment, Net
The Company’s property and equipment consisted of the following:
| September 30, 2024 | December 31, 2023 | |
|---|---|---|
| Laboratory equipment | $32,044 | $29,232 |
| Machinery and equipment | 12,902 | 12,325 |
| Computer equipment | 1,015 | 244 |
| Furniture and fixtures | 2,777 | 2,777 |
| Leasehold improvements | 7,304 | 6,723 |
| Internal use software | 1,903 | 1,903 |
| 57,945 | 53,204 | |
| Less: accumulated depreciation and amortization | (19,431) | (14,032) |
| Total property and equipment, net | $38,514 | $39,172 |
During the three and nine months ended September 30, 2024, the Company recognized $1.9 million and $5.4 million of depreciation and amortization expense, respectively. During the three and nine months ended September 30, 2023, the Company recognized $1.2 million and $3.5 million of depreciation and amortization expense, respectively.
Intangible Assets and Goodwill
The Company’s indefinite lived intangible assets consist of an acquired IPR&D asset received in the acquisition of Renovacor on December 1, 2022. Intangible assets as of September 30, 2024 and December 31, 2023 are summarized as follows:
| September 30, 2024 | December 31, 2023 | |
|---|---|---|
| Gross carrying value | $25,150 | $25,150 |
| Accumulated amortization | - | - |
| Total intangible assets | $25,150 | $25,150 |
The carrying value of Goodwill as of September 30, 2024 and December 31, 2023 was $39.2 million.
Accounts Payable and Accrued Expenses
The Company’s accounts payable and accrued expenses consisted of the following:
| September 30, 2024 | December 31, 2023 | |
|---|---|---|
| Research and development | $19,074 | $13,867 |
| Investment payable | - | 13,137 |
| Employee compensation | 10,316 | 9,930 |
| Property and equipment | 265 | 1,077 |
| Professional fees | 4,708 | 6,006 |
| Other | 2,770 | 1,772 |
| Total accounts payable and accrued expenses | $37,133 | $45,789 |
The $13.1 million investment payable at December 31, 2023 was related to investment purchases of available-for-sale securities in 2023 that settled in 2024.
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 has reimbursed Cowen for certain expenses incurred in connection with the Sales Agreement. On September 12, 2023, the Company and Cowen entered into the Amended Sales Agreement pursuant to which the aggregate offering amount available under the at-the-market offering program was reduced to $180.0 million. Through September 30, 2024, 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. The Company didnot sell any shares under the at-the-market offering program during thenine months ended September 30, 2024.
Stock-Based Compensation
Stock Option Valuation
The weighted average assumptions that the Company used in a Black-Scholes pricing model to determine the fair value of stock options granted to employees, non-employees and directors were as follows:
| Nine Months Ended September 30, | ||
|---|---|---|
| 2024 | 2023 | |
| Risk-free interest rate | 4.85% | 3.98% |
| Expected term (in years) | 5.82 | 5.84 |
| Expected volatility | 73.70% | 73.35% |
| Expected dividend yield | 0.00% | 0.00% |
| Exercise price | $27.24 | $20.15 |
| Fair value of common stock | $27.24 | $20.15 |
The following table summarizes stock option activity for thenine months ended September 30, 2024:
| Weighted · Weighted | |||||
|---|---|---|---|---|---|
| Average · Average · Aggregate | |||||
| Number of · Exercise · Contractual · Intrinsic | |||||
| Shares · Price · Term (Years) · Value | |||||
| Outstanding as of December 31, 2023 | 14,863,996 | $15.07 | 5.16 | $250,602 | |
| Granted | 1,725,627 | 27.24 | 9.37 | ||
| Exercised | (246,208) | 11.74 | 3,340 | ||
| Cancelled or forfeited | (195,395) | 26.73 | |||
| Outstanding as of September 30, 2024 | 16,148,020 | $16.28 | 4.94 | $104,704 | |
| Options vested and exercisable as of September 30, 2024 | 13,012,785 | $14.52 | 3.98 | $103,807 | |
| Options unvested as of September 30, 2024 | 3,135,235 | $23.57 | 8.93 | $897 | |
The weighted average grant-date fair value per share of stock options granted during the nine months ended September 30, 2024, and 2023 was$18.34 and$13.32, respectively.
The total fair value of options vested during nine months ended September 30, 2024 and 2023 was$23.6 million and$26.9 million, respectively.
Restricted Stock Units
The following table summarizes the Company’s RSU activity for thenine months ended September 30, 2024:
| Weighted Average | ||
|---|---|---|
| Number of | Grant Date | |
| Shares | Fair Value | |
| Unvested as of December 31, 2023 | 1,490,357 | $18.53 |
| Granted | 743,022 | 27.73 |
| Vested | (588,217) | 18.75 |
| Forfeited | (79,718) | 20.03 |
| Unvested as of September 30, 2024 | 1,565,444 | $22.73 |
Performance Stock Units
The following table summarizes the Company’s PSU activity fornine months ended September 30, 2024:
| Weighted Average | ||
|---|---|---|
| Number of | Grant Date | |
| Shares | Fair Value | |
| Unvested as of December 31, 2023 · - · $ · - | ||
| Granted | 156,738 | 27.93 |
| Vested | - | - |
| Forfeited | (34,831) | 28.71 |
| Unvested as of September 30, 2024 | 121,907 | $27.71 |
PSU vesting and expense recognition is based on achievement of specific performance goals within certain time periods. PSU awards that are not achieved within specific time periods are forfeited. No performance goals were probable of achievement and the time period for one performance goal had expired as of September 30, 2024.
Stock-Based Compensation Expense
Stock-based compensation expense recognized by award type was as follows:
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |
| Stock options | $7,005 | $7,482 | $21,105 | $21,922 |
| Restricted stock units | 4,243 | 2,834 | 11,727 | 7,554 |
| Total stock-based compensation expense | $11,248 | $10,316 | $32,832 | $29,476 |
Stock-based compensation expense by classification included within the Consolidated Statements of Operations and Comprehensive Loss was as follows:
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |
| Research and development | $4,789 | $4,673 | $14,311 | $13,130 |
| General and administrative | 6,459 | 5,643 | 18,521 | 16,346 |
| Total stock-based compensation expense | $11,248 | $10,316 | $32,832 | $29,476 |
As of September 30, 2024, the Company had an aggregate of$72.0 million of unrecognized stock-based compensation expense related to stock options, RSU and PSU grants. The stock options and RSU grants had an aggregate of$68.6 million of unrecognized stock-based compensation expense, which is expected to be recognized over a weighted average period of 1.72 years.
Warrants
A summary of the warrants outstanding as of September 30, 2024 is as follows:
| Exercise Price | Outstanding | Grant/Assumption Date | Expiration Date |
|---|---|---|---|
| $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 |
| $0.01 | 3,126,955 | September 15, 2023 | N/A |
| Total | 5,715,078 | ||
There were no changes in warrants to purchase common stock for the nine months ended September 30, 2024.
Warrants Issued in Public Offering
On September 15, 2023, the Company completed a public offering that included pre-funded warrants to purchase 3,126,955 shares of common shares at a price of $0.01 per share (see Note 16 “Related Party Transactions”).
Assumed Renovacor Public Warrants
In conjunction with the acquisition of Renovacor, 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 acquisition, the Public Warrants were recorded as a component of additional paid-in capital of $3.4 million.
Assumed Renovacor Private Warrants
In conjunction with the acquisition of Renovacor, 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 “Fair Value of Financial Instruments” for discussion of fair value measurement of the warrant liability.
Net Loss Per Share
Basic and diluted net loss per share attributable to common stockholders was calculated as follows:
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |
| Numerator: | ||||
| Net loss attributable to common stockholders | $(66,719) | $(61,899) | $(198,419) | $(185,935) |
| Denominator: | ||||
| Weighted-average common shares outstanding - basic and diluted | 94,158,491 | 82,636,120 | 93,893,729 | 80,865,658 |
| Net loss per share attributable to common stockholders - basic and diluted | $(0.71) | $(0.75) | $(2.11) | $(2.30) |
For the nine months ended September 30, 2024, the Company included the 3,126,955 shares of common stock issuable upon the exercise of pre-funded warrants acquired by funds affiliated with RTW in the weighted average shares outstanding as it was determined that these met the definition for equity classification as the holder is only required to pay $0.01 per share upon exercise of the pre-funded warrants.
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 and Nine Months Ended September 30, | ||
|---|---|---|
| 2024 | 2023 | |
| Warrants exercisable for common shares | 2,588,123 | 2,588,123 |
| Restricted stock units | 1,565,444 | 1,606,104 |
| Performance stock units | 121,907 | - |
| Options to purchase common shares | 16,148,020 | 14,889,669 |
| Total potential shares excluded from diluted net loss per share | 20,423,494 | 19,083,896 |
Leases
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 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 fortwo 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 as part of deposits in the Consolidated Balance Sheets as of September 30, 2024 and December 31, 2023.
Operating Leases
On June 7, 2018, the Company entered into athree-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 $0.8 million. On March 26, 2021, the Company entered into 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. On March 29, 2024, the Company entered into Amendment No. 2 to the ESB Lease Agreement that extended the term of the lease agreement to July 31, 2027. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary and expires September 30, 2027.
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 September 30, 2024 and December 31, 2023.
On November 15, 2022, the Company entered into a lease agreement with a lease term until October 31, 2024, for laboratory space in Madrid, Spain. The lease commenced on April 1, 2023 and the Company recognized a right-of-use asset and corresponding lease liability of approximately $0.2 million each.
On December 1, 2022, in connection with the acquisition of Renovacor, the Company acquired the Renovacor operating leases for space at facilities in Hopewell, New Jersey and Cambridge, Massachusetts with remaining lease terms of approximately 10.3 and 1.3 years, respectively. The Company recognized total right-of-use assets of $3.8 million with corresponding total lease liabilities of $3.6 million at lease commencement dates. The Company signed an agreement to sublease one of these facilities in January 2024 and intends to sublease the other remaining facilities. Rental income received under the sublease agreement totaled $0.1 million and $0.3 million for the three and nine months ended September 30, 2024, respectively.
Rent expense under operating leases was $0.6 million and $2.0 million for the three and nine months ended September 30, 2024. Rent expense under operating leases was $0.7 million and $1.7 million for thethree and nine months ended September 30, 2023, respectively.
The total restricted cash balance for the Company’s operating and finance leases as of September 30, 2024 and December 31, 2023 was $0.8 million.
Operating lease cost was $0.3 million and $1.0 million for thethree and nine months ended September 30, 2024, respectively. Operating lease cost was $0.3 million and $1.0 million for thethree and nine months ended September 30, 2023, respectively.
The following table summarizes lease cost for thenine months ended September 30, 2024 and 2023:
| Nine Months Ended September 30, | ||
|---|---|---|
| Lease cost | 2024 | 2023 |
| Operating lease cost | $956 | $1,026 |
| Finance lease cost: | ||
| Amortization of right of use assets | 1,615 | 1,615 |
| Interest on lease liabilities | 1,413 | 1,405 |
| Total lease cost | $3,984 | $4,046 |
The following table summarizes the future lease payments of the Company’s operating lease liabilities on an undiscounted cash flow basis:
| Fiscal Year Ending December 31, | September 30, 2024 |
|---|---|
| 2024 (three months) | $257 |
| 2025 | 1,001 |
| 2026 | 1,005 |
| 2027 | 759 |
| 2028 | 522 |
| Thereafter | 2,420 |
| Total lease payments | $5,964 |
| Less: interest | (1,546) |
| Total operating lease liabilities | $4,418 |
The following table summarizes the future lease payments of the Company’s finance lease liability on an undiscounted cash flow basis:
| Fiscal Year Ending December 31, | September 30, 2024 |
|---|---|
| 2024 (three months) | $457 |
| 2025 | 1,856 |
| 2026 | 1,911 |
| 2027 | 1,969 |
| 2028 | 2,028 |
| Thereafter | 41,003 |
| Total lease payments | $49,224 |
| Less: interest | (28,000) |
| Total finance lease liability | $21,224 |
The following table summarizes the operating and financing lease liabilities and right-of-use assets as of September 30, 2024 and December 31, 2023:
| Leases | September 30, 2024 | December 31, 2023 |
|---|---|---|
| Operating right-of-use assets | $4,349 | $3,901 |
| Operating current lease liabilities | $1,005 | $925 |
| Operating noncurrent lease liabilities | 3,413 | 2,973 |
| Total operating lease liabilities | $4,418 | $3,898 |
| Finance right-of-use assets | $42,902 | $44,517 |
| Finance current lease liability | $1,842 | $1,791 |
| Finance noncurrent lease liability | 19,382 | 19,353 |
| Total finance lease liability | $21,224 | $21,144 |
| Nine Months Ended September 30, | ||
|---|---|---|
| Other Information | 2024 | 2023 |
| Cash paid for amounts included in the measurement of lease liabilities: | ||
| Operating cash flows from operating leases | $829 | $894 |
| Cash flows from finance lease | $1,333 | $1,296 |
| Weighted-average remaining lease term - operating leases | 6.7 years | 8.0 years |
| Weighted-average remaining lease term - finance lease | 19.9 years | 20.9 years |
| Weighted-average discount rate - operating leases | 8.82% | 8.34% |
| Weighted-average discount rate - finance lease | 8.96% | 8.96% |
Commitments and Contingencies
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.
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 R&D activities has no alternative future uses.
CIRM Grants
LAD-I CIRM Grant
On April 30, 2019, 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, RP-L201. Proceeds from the grant helped 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 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. As of September 30, 2024, the Company has received $5.9 million in total RP-L201 grants from CIRM. The Company received a final milestone grant of $0.05 million on January 2, 2024 andno additional payments are available under the grant awards program.
Danon CIRM Grant
On August 18, 2024, CIRM awarded the Company up to $5.8 million under a CLIN2 grant award to support the clinical development of its AAV-based gene therapy, RP-A501 for the treatment of DD. Proceeds from the grant would help fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients. As of September 30, 2024, the Company has received an initial RP-A501 grant of $1.7 million from CIRM, which was recorded as a reduction of R&D expenses for the RP-A501 program.
Related Party Transactions
In June 2023, the Company entered into a consulting agreement with the spouse of one of the Company’s executive officers for information technology advisory services. The Company incurred expenses of approximately $0.002 million for thenine months ended September 30, 2024, relating to services provided under this agreement.
In September 2023, in connection with a public offering, the Company sold approximately 3.1 million pre-funded warrants to purchase shares of the Company’s common stock to funds affiliated with RTW, the Company’s largest shareholder (see Note 10 “Warrants”).
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 thethree and nine months ended September 30, 2024, was$0.5 and$1.3 million, respectively. The Company’s matching contribution for the three and nine months ended September 30, 2023, was $0.4 and $1.1 million, respectively.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.