Solid Biosciences Inc. 10-Q

Item 1. Financial Statements (unaudited) SOLID BIOSCIENCES INC. CONDENSED CONSOLIDATED BALANCE SHEETS (unaudited, in thousands, except share and per share data) March 31, December 31, 2022 2021 Assets Current assets: Cash and cash equivalents $ 129,711 $ 119,136 Available-for-sale securities 50,339 88,643 Prepaid expenses and other current assets 18,544 14,723 Accounts receivable - related party 14 110 Total current assets 198,608 222,612 Property and equipment, net 6,097 6,462 Operating lease, right-of-use assets 722 1,142 Other non-current assets 59 94 Restricted cash 2,070 2,070 Total assets $ 207,556 $ 232,380 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 4,761 $ 4,463 Accrued expenses 9,440 9,528 Operating lease liabilities 984 1,263 Finance lease liabilities 239 232 Other current liabilities 150 35 Deferred revenue - related party 6,170 8,080 Total current liabilities 21,744 23,601 Operating lease liabilities, excluding current portion 70 275 Finance lease liabilities, excluding current portion 231 293 Total liabilities 22,045 24,169 Commitments and contingencies Preferred stock, $ 0.001 par value; 10,000,000 shares authorized at March 31, 2022 and December 31, 2021; no shares issued and outstanding at March 31, 2022 and December 31, 2021 — — Common stock, $ 0.001 par value; 300,000,000 shares authorized at March 31, 2022 and December 31, 2021; 112,781,291 shares issued and outstanding at March 31, 2022 and 110,340,281 shares issued and outstanding at December 31, 2021; no pre-funded warrants outstanding at March 31, 2022 and 2,158,329 pre-funded warrants outstanding at December 31, 2021 112 112 Additional paid-in capital 687,535 684,901 Accumulated other comprehensive loss ( 51 ) ( 45 ) Accumulated deficit ( 502,085 ) ( 476,757 ) Total stockholders’ equity 185,511 208,211 Total liabilities and stockholders’ equity $ 207,556 $ 232,380 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 2 SOLID BIOSCIENCES INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited, in thousands, except share and per share data) Three Months Ended March 31, 2022 2021 Collaboration revenue - related party $ 1,925 $ 3,335 Operating expenses: Research and development 19,945 14,206 General and administrative 7,352 6,015 Total operating expenses 27,297 20,221 Loss from operations ( 25,372 ) ( 16,886 ) Other income (expense), net 44 ( 14 ) Net loss $ ( 25,328 ) $ ( 16,900 ) Net loss per share attributable to common stockholders, basic and diluted $ ( 0.22 ) $ ( 0.19 ) Weighted average shares of common stock outstanding, basic and diluted 112,607,322 89,267,194 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 3 SOLID BIOSCIENCES INC. CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (unaudited, in thousands) Three Months Ended March 31, 2022 2021 Net loss $ ( 25,328 ) $ ( 16,900 ) Other comprehensive loss: Unrealized loss on available-for-sale securities ( 6 ) — Comprehensive loss $ ( 25,334 ) $ ( 16,900 ) The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 4 SOLID BIOSCIENCES INC. CONDENSED CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS’ EQUITY (unaudited, in thousands, except share data) For the Three Months Ended March 31, 2022 Common Stock Amount Additional Paid in Capital Accumulated Other Comprehensive Loss Accumulated Deficit Total Stockholders' Equity Balance at December 31, 2021 112,498,610 $ 112 $ 684,901 $ ( 45 ) $ ( 476,757 ) $ 208,211 Equity-based compensation — — 2,612 — — 2,612 Vesting of restricted stock units 282,681 — — — — — Exercise of pre-funded warrants — — 22 — — 22 Unrealized loss on available-for-sale securities — — — ( 6 ) — ( 6 ) Net loss — — — — ( 25,328 ) ( 25,328 ) Balance at March 31, 2022 112,781,291 $ 112 $ 687,535 $ ( 51 ) $ ( 502,085 ) $ 185,511 For the Three Months Ended March 31, 2021 Common Stock Amount Additional Paid in Capital Accumulated Other Comprehensive Income (Loss) Accumulated Deficit Total Stockholders' Equity Balance at December 31, 2020 87,052,323 $ 87 $ 536,568 $ — $ ( 404,569 ) $ 132,086 Equity-based compensation — — 2,907 — — 2,907 Sale of common stock, net of issuance costs of $ 8,872 25,000,000 25 134,853 — 134,878 Exercise of stock options 8,150 — 29 — 29 Vesting of restricted stock units 412,931 — — — — — Forfeiture of restricted stock awards ( 26,508 ) — — — — — Net loss — — — — ( 16,900 ) ( 16,900 ) Balance at March 31, 2021 112,446,896 $ 112 $ 674,357 $ — $ ( 421,469 ) $ 253,000 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 5 SOLID BIOSCIENCES INC. CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited, in thousands) Three Months Ended March 31, 2022 2021 Cash flows from operating activities: Net loss $ ( 25,328 ) $ ( 16,900 ) Adjustments to reconcile net loss to net cash used in operating activities: Amortization of premium on available-for-sale securities 371 — Equity-based compensation expense 2,612 2,907 Depreciation expense 709 734 Loss on disposal of property and equipment — 3 Changes in operating assets and liabilities: Prepaid expenses and other current and non-current assets ( 3,366 ) ( 769 ) Accounts receivable - related party 96 ( 280 ) Accounts payable 71 ( 90 ) Accrued expenses and other current and non-current liabilities ( 445 ) ( 3,916 ) Deferred revenue- related party, current and non-current ( 1,910 ) ( 3,055 ) Net cash used in operating activities ( 27,190 ) ( 21,366 ) Cash flows from investing activities: Purchases of property and equipment ( 184 ) ( 35 ) Proceeds from sale and maturities of available-for-sale securities 46,808 — Purchases of available-for-sale securities ( 8,881 ) — Net cash provided by (used in) investing activities 37,743 ( 35 ) Cash flows from financing activities: Proceeds from issuance of common stock, net of issuance costs — 135,125 Proceeds from exercise of warrants 22 — Proceeds from exercise of stock options — 29 Net cash provided by financing activities 22 135,154 Net increase in cash, cash equivalents and restricted cash 10,575 113,753 Cash, cash equivalents, and restricted cash at beginning of period 121,206 155,071 Cash, cash equivalents, and restricted cash at end of period $ 131,781 $ 268,824 Supplemental disclosure of non-cash investing and financing activities: Offering costs in accruals $ — $ 247 Property and equipment included in accounts payable and accruals $ 264 $ 10 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 6 SOLID BIOSCIENCES INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (unaudited, amounts in thousands, except share and per share data) 1. Nature of the Business and Basis of Presentation Nature of Business Solid Biosciences Inc. was organized in March 2013 under the name SOLID Ventures Management, LLC and operated as a Delaware limited liability company until immediately prior to the effectiveness of its registration statement on Form S-1 on January 25, 2018, at which time it completed a statutory corporate conversion into a Delaware corporation and changed its name to Solid Biosciences Inc. (the “Company”). The Company’s mission is to cure Duchenne muscular dystrophy (“Duchenne”), a genetic muscle-wasting disease predominantly affecting boys. It is caused by mutations in the dystrophin gene, which result in the absence or near-absence of dystrophin protein. Dystrophin protein works to strengthen muscle fibers and protect them from daily wear and tear. Without functioning dystrophin and certain associated proteins, muscles suffer excessive damage from normal daily activities and are unable to regenerate, leading to the build-up of fibrotic, or scar, and fat tissue. The Company’s lead product candidate, SGT-001, is a gene transfer candidate under investigation for its ability to drive functional dystrophin protein expression in patients’ muscles and improve the course of the disease. SGT-001 has been granted Rare Pediatric Disease Designation and Fast Track Designation in the United States and Orphan Drug Designations in both the United States and European Union. The Company filed an Investigational New Drug application (“IND”) in September 2017 and initiated a Phase I/II clinical trial for SGT-001 in the United States during the fourth quarter of 2017 , which is called IGNITE DMD. In March 2022, the Company reported two-year interim safety and efficacy data from the first three patients treated with SGT-001 in the 2E14 vg/kg dose cohort of IGNITE DMD, which results suggested durable benefit compared with natural history trajectories 24 months post-administration of SGT-001 across functional, pulmonary and patient reported outcome measures. In addition, no new drug-related safety findings have been identified in patients treated with SGT-001 in IGNITE DMD in post-dosing periods of 90 days to approximately four years. In April 2022, the Company announced that it had concluded enrollment in IGNITE DMD. In May 2021, the Company announced the advancement of a next-generation Duchenne microdystrophin gene transfer program, SGT-003, a preclinical candidate that combines a novel and rationally designed capsid candidate with the Company’s proprietary neuronal Nitric Oxide Synthase (“nNOS”) containing microdystrophin construct. The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, dependence on licenses, protection of proprietary technology, dependence on key personnel, compliance with government regulations and the need to obtain additional financing to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical studies and clinical trials and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure and extensive compliance and reporting capabilities. The Company’s product candidates are in development. 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 regulatory 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, among others, other pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, partners and consultants. Liquidity The accompanying condensed consolidated financial statements have been prepared on a basis that assumes the Company will continue as a going concern and which contemplates the realization of assets and satisfaction of liabilities and commitments in the ordinary course of business. Through March 31, 2022, the Company has funded its operations primarily with the proceeds from the sale of redeemable preferred units and member units, the sale of common stock and prefunded warrants to purchase shares of its common stock in private placements, the sale of common stock in its initial public offering and follow-on public offering in March 2021 and sales under an at-the-market sales agreement, dated March 13, 2019, as amended on August 16, 2021 (the “ATM Sales Agreement”), by and between the Company and Jefferies LLC (“Jefferies”). 7 In accordance with Accounting Standards Codification (“ASC”) 205-40, Going Concern, the Company has evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date the financial statements are issued. As of March 31, 2022, the Company had an accumulated deficit of $ 502,085 . During the three months ended March 31, 2022, the Company incurred a net loss of $ 25,328 , and the Company used $ 27,190 of cash in operations for the three months ended March 31, 2022. The Company expects to continue to generate operating losses in the foreseeable future. Based upon its current operating plan, the Company expects that its cash, cash equivalents and available-for-sale securities of $ 180,050 as of March 31, 2022, will be sufficient to fund its operating expenses and capital expenditure requirements for at least twelve months from the date of issuance of these financial statements. However, the Company has based this estimate on assumptions that may prove to be wrong, and its operating plan may change as a result of many factors currently unknown to it. As a result, the Company could deplete its capital resources sooner than it currently expects. The Company expects it may finance its future cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances or licensing arrangements. If the Company is unable to obtain funding, the Company would be forced to delay, reduce or eliminate some or all of its research and development programs, preclinical and clinical testing or commercialization efforts, which could adversely affect its business prospects. The accompanying condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”). The accompanying condensed consolidated financial statements include the accounts of Solid Biosciences Inc. and its wholly owned or controlled subsidiaries. All intercompany accounts and transactions have been eliminated. In the opinion of management, the Company’s accompanying unaudited condensed consolidated financial statements include all adjustments, consisting of normal recurring accruals, necessary for a fair statement of the Company’s financial statements for interim periods in accordance with GAAP. The information included in this quarterly report on Form 10-Q should be read in conjunction with the Company’s consolidated financial statements and the accompanying notes included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021. The year-end condensed consolidated balance sheet data presented for comparative purposes was derived from the Company’s audited financial statements but does not include all disclosures required by GAAP. The results of operations for the three months ended March 31, 2022 are not necessarily indicative of the operating results for the full year or for any other subsequent interim period. 2. Summary of Significant Accounting Policies The Company’s accounting policies are described in the “Notes to Consolidated Financial Statements” in its Annual Report on Form 10-K for the year ended December 31, 2021 and updated, as necessary, in this report. Use of Estimates The preparation of the Company’s condensed consolidated financial statements in conformity with GAAP requires management to make estimates, judgments and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of revenues and expenses during the reporting periods. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, estimates related to revenue recognition, the recognition of research and development expenses and equity-based compensation. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from the Company’s estimates. The full extent to which the COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition, including clinical trials and employee-related amounts, will depend on future developments that are highly uncertain, including new information that may emerge concerning COVID-19 and the actions taken to contain it or treat its impact. The Company has made estimates of the impact of COVID-19 within its financial statements and there may be changes to those estimates in future periods. Actual results could differ from the Company’s estimates. Cash Equivalents The Company considers all short-term, highly liquid investments with original maturities of 90 days or less at acquisition date to be cash equivalents. 8 Restricted Cash The Company held restricted cash of $ 2,070 in separate restricted bank accounts as security deposits for leases of the Company’s facilities as of March 31, 2022 and December 31, 2021. As of March 31, 2022 and December 31, 2021, the Company classified $ 2,070 as a non-current asset. A reconciliation of the amounts of cash and cash equivalents and restricted cash from the cash flow statement to the balance sheet is as follows: March 31, 2022 December 31, 2021 March 31, 2021 December 31, 2020 Cash and cash equivalents as presented on balance sheet $ 129,711 $ 119,136 $ 268,497 $ 154,744 Restricted cash, as presented on balance sheet 2,070 2,070 327 327 Cash and cash equivalents and restricted cash as presented on cash flow statement $ 131,781 $ 121,206 $ 268,824 $ 155,071 Leases At inception of a contract, the Company determines if a contract meets the definition of a lease. A lease is a contract, or part of a contract, that conveys the right to control the use of identified property, plant, or equipment (an identified asset) for a period of time in exchange for consideration. The Company determines if the contract conveys the right to control the use of an identified asset for a period of time. The Company assesses throughout the period of use whether the Company has both of the following: (1) the right to obtain substantially all of the economic benefits from use of the identified asset and (2) the right to direct the use of the identified asset. This determination is reassessed if the terms of the contract are changed. Leases are classified as operating or finance leases based on the terms of the lease agreement and certain characteristics of the identified asset. Right-of-use assets and lease liabilities are recognized at the lease commencement date based on the present value of the minimum future lease payments. The Company’s policy is to not record leases with an original term of twelve months or less on the consolidated balance sheets. The Company recognizes lease expense for these short-term leases on a straight-line basis over the lease term. Certain lease agreements include rental payments that are adjusted periodically for inflation or other variables. In addition to rent, the leases may require the Company to pay additional amounts for taxes, insurance, maintenance and other expenses, which are generally referred to as non-lease components. Such adjustments to rental payments and variable non-lease components are treated as variable lease payments and recognized in the period in which the obligation for these payments was incurred. Variable lease components and variable non-lease components are not measured as part of the right of use asset and liability. Only when lease components and their associated non-lease components are fixed are they accounted for as a single lease component and recognized as part of a right of use asset and liability. Total contract consideration is allocated to the combined fixed lease and non-lease components. In June 2021, the Company entered into a lease with Hood Park LLC (“Landlord”), pursuant to which the Company will lease approximately 49,869 square feet of office, laboratory, research and development and manufacturing space located in Charlestown, Massachusetts (“Premises”). The Company expects to relocate its corporate headquarters to the Premises in May 2022. The term of the lease commences on the later of (i) the date the Landlord delivers the Premises to the Company or (ii) the earlier of (a) the date the Company’s work on the Premises is substantially completed, (b) the date the Company commences business operations in the Premises, or (c) the one hundred twentieth (120th) day following the Landlord’s satisfaction of item (i) above. The lease commencement date is anticipated to be in the second quarter of 2022. The initial term of the lease will be for a ten-year period commencing on the lease commencement date, unless earlier terminated. The lease provides the Company with an option to extend the lease for an additional five-year term. The Company and the Landlord are each obligated to undertake certain improvements prior to the commencement of the lease, and significant improvements were still in progress as of March 31, 2022. The lease will commence when the construction of the lessor assets is substantially complete, which is expected to be in May 2022. The monthly lease payment is approximately $ 305 with annual escalation of approximately 3 %. The lease includes a $ 10,223 construction allowance. The Company was required to post a customary letter of credit in the amount of $ 1,833 , subject to decrease on a set schedule, as a security deposit pursuant to the lease. As of March 31, 2022, the Company recorded approximately $ 6,171 of tenant improvement allowance receivable, reimbursable by the landlord which is included in other current assets on the accompanying balance sheet. In April 2022, the Company terminated a lease for lab space in Cambridge, Massachusetts early. The lease will terminate in June 2022 . The Company is currently evaluating the impact of the termination agreement to the financial statements including the right of use asset and lease liability. 9 Segment Data The Company manages its operations as a single segment for the purposes of assessing performance and making operating decisions. The Company’s singular focus is on developing treatments through gene therapy and other means for patients with Duchenne. All of the Company’s tangible assets are held in the United States. Related Parties In October 2020, the Company entered into a collaboration and license agreement (the “Collaboration Agreement”) with Ultragenyx Pharmaceutical Inc. (“Ultragenyx”). In connection with the Collaboration Agreement, Ultragenyx also purchased 7,825,797 shares of the Company’s common stock, which resulted in Ultragenyx becoming a related party of the Company. In November 2020, the Company entered into a consulting agreement with Danforth Advisors, LLC, or Danforth, an affiliate of Stephen DiPalma, the Company’s interim chief financial officer. Pursuant to the consulting agreement, Danforth provides the Company with the chief financial officer services of Mr. DiPalma, and other services, including financial planning, offering support and accounting services, in exchange for fees payable to Danforth based on hourly rates. The Company has paid Danforth approximately $ 235 and $ 85 for the three months ended March 31, 2022, and March 31, 2021, respectively. In accordance with the consulting agreement, in November 2020, the Company issued to Danforth a warrant to purchase 30,000 shares of the Company’s common stock at an exercise price per share of $ 3.29 . As of March 31, 2022, the shares had vested in full. The consulting agreement may be terminated by either party without cause upon 60 days’ prior written notice to the other party and with cause upon 30 days’ prior written notice to the other party. Recently Issued Accounting Pronouncements Not Yet Adopted In August 2020, the Financial Accounting Standards Board (the “FASB”) issued Accounting Standards Update (“ASU”) No. 2020-06, Debt, Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging—Contracts in Entity’s Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity’s Own Equity , which, among other things, provides guidance on how to account for contracts on an entity’s own equity. This ASU simplifies the accounting for certain financial instruments with characteristics of liabilities and equity. Specifically, the ASU eliminated the need for the Company to assess whether a contract on the entity’s own equity (1) permits settlement in unregistered shares, (2) whether counterparty rights rank higher than shareholder’s rights, and (3) whether collateral is required. In addition, the ASU requires incremental disclosure related to contracts on the entity’s own equity and clarifies the treatment of certain financial instruments accounted for under this ASU on earnings per share. The ASU also simplifies the accounting for convertible instruments by removing the beneficial conversion feature and cash conversion feature separation models. This ASU may be applied on a full retrospective or modified retrospective basis. This ASU is effective for smaller reporting companies for fiscal years beginning after December 15 , 2023, with early adoption permitted. The Company does not expect the adoption to materially impact its financial position and results of operations. In June 2016, the FASB issued ASU 2016-13, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments , which was subsequently modified by several ASU’s issued in 2018 and 2019. The standard introduces a new current expected credit loss (“CECL”) model for measuring expected credit losses for certain types of financial instruments measured at amortized cost and replaces the incurred loss model. The CECL model requires an entity to recognize an allowance for credit losses for the difference between the amortized cost basis of a financial instrument and the amount the entity expects to collect over the instrument’s contractual life after consideration of historical experience, current conditions, and reasonable and supportable forecasts. The standard eliminates the concept of other-than-temporary impairment and requires an entity to determine whether any impairment is the result of a credit loss or other factors. ASU 2016-13 is effective for the Company on January 1, 2023. The Company is currently evaluating the potential impact that this standard may have on its financial statements and related disclosures. 3. Collaborations Ultragenyx Collaboration Collaboration Agreement On October 22, 2020 (the “Effective Date”), the Company entered into the Collaboration Agreement with Ultragenyx to focus on the development and commercialization of new gene therapies for Duchenne. The Company granted Ultragenyx an exclusive worldwide license for any pharmaceutical product that expresses the Company’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for the treatment of Duchenne and other diseases resulting from the lack of functional dystrophin (the “Licensed Products”). The Company retains exclusive rights to all other uses of its microdystrophin proteins, including under its existing SGT-001 program. 10 The Company is conducting certain research and development activities with respect to the development of the Licensed Products. Ultragenyx is reimburs ing the Company for personnel and out-of-pocket costs that the Company incurs in conducting such development activities. In addition, Ultragenyx granted to the Company an exclusive Development Option or Income Share Option (each as defined and described below) exercisable in the Company’s sole discretion one time per Licensed Product. After the date of first achievement of clinical proof of concept, Ultragenyx will provide to the Company a data package with respect to the relevant Licensed Product. The Company will use the data package to determine whether to exercise the corresponding Development Option or Income Share Option with respect to such Licensed Product. With respect to each Licensed Product for which the Company has not exercised the Development Option or Income Share Option the Company will be entitled to milestone payments of up to $ 25,000 in the aggregate for each such Licensed Product that achieves specified development milestones and $ 65,000 in the aggregate for each such Licensed Product that achieves specified regulatory milestones. With respect to each Licensed Product for which the Company has not exercised the Income Share Option, the Company will also be entitled to milestone payments of up to $ 165,000 in the aggregate for each Licensed Product that achieves specified annual worldwide net sales milestones. For Licensed Products for which the Company has not exercised the Development Option or Income Share Option, Ultragenyx will pay the Company tiered royalties on a Licensed Product-by-Licensed Product and country-by-country basis ranging from a low double-digit percentage to a mid-teens percentage based on Ultragenyx’s annual worldwide net sales of such Licensed Products. For each Licensed Product for which Ultragenyx decides to initiate a registrational trial in humans, the Company will have the option to fund 30 % of the development costs in the United States and European Union for such Licensed Product and forgo the development and regulatory milestones (the “Development Option”) and receive tiered royalties on a Licensed Product-by-Licensed Product and country-by-country basis ranging from a mid-teens percentage to a low twenties percentage based on Ultragenyx’s annual worldwide net sales of each such Licensed Product. For each Licensed Product for which the Company exercises the Development Option, the Company may also elect to share 30 % of the net income and net losses on net sales of such Licensed Product in the United States and European Union (the “Income Share Option”). For Licensed Products for which the Company has exercised the Income Share Option, the Company will not be entitled to milestone payments and Ultragenyx will pay the Company tiered royalties on a Licensed Product-by-Licensed Product and country-by-country basis ranging from a mid-teens percentage to a low twenties percentage based on Ultragenyx’s annual net sales of each such Licensed Product outside of the United States and European Union. The Company may only exercise an Income Share Option if neither the Company nor any of its affiliates is then developing or commercializing a product that is competitive with the Licensed Product that is subject to such option. If the Company or any of its affiliates subsequently develops or commercializes a product that is competitive with a Licensed Product for which the Company has exercised an Income Share Option, then the Company and Ultragenyx will no longer share the net income and net losses on net sales of such Licensed Product and such Licensed Product will be treated as if the Company had exercised the Development Option with respect to such Licensed Product. Following the Company’s exercise of the Development Option or Income Share Option with respect to a Licensed Product, the Company also has the right to cease participation in the sharing of development costs and sharing in net income and net losses on net sales, as applicable, for such Licensed Product by written notice to Ultragenyx. Upon such notice, the Company will no longer share in the development costs and net income and net losses on net sales of such Licensed Product, as applicable, and will be eligible to receive payments on milestones achieved after the opt-out for such Licensed Product and royalties at the rates applicable to Licensed Products for which the Company has not exercised the Development Option or Income Share Option, as described above. The Collaboration Agreement continues on a country-by-country and Licensed Product-by-Licensed Product basis until the expiration of all payment obligations under the agreement. With respect to any Licensed Product for which the Company has exercised an Income Share Option, the Collaboration Agreement continues until there are no longer sales of such Licensed Product in the United States or Europe. Either party has the right to terminate the agreement if the other party has materially breached in the performance of its obligations under the agreement and such breach has not been cured within the applicable cure period. Ultragenyx may also terminate the Collaboration Agreement in its sole discretion upon 90 days’ prior written notice to the Company. Stock Purchase Agreement In connection with the execution of the Collaboration Agreement, Ultragenyx and the Company also entered into a stock purchase agreement (the “Stock Purchase Agreement”) on the Effective Date, pursuant to which the Company issued and sold 7,825,797 shares of its common stock (the “Shares”) to Ultragenyx at a price of $ 5.1113 per share for an aggregate purchase price of approximately $ 40,000 . The Stock Purchase Agreement contains customary representations, warranties and covenants of each of the parties thereto. Following the sale of the Shares, Ultragenyx beneficially owned approximately 14.45 % of the Company’s outstanding common stock . As of March 31, 2022 , Ultragenyx beneficially owned less than 10 % of the Company’s outstanding common stock. 11 Investor Agreement In connection with the consummation of the transactions contemplated by the Stock Purchase Agreement, the Company and Ultragenyx entered into an Investor Agreement (the “Investor Agreement”) on the Effective Date. Pursuant to the terms of the Investor Agreement, Ultragenyx agreed that the Shares will be subject to a lock-up restriction, such that Ultragenyx will not, and will also cause its affiliates not to, without the prior approval of the Company and with certain exceptions, sell, transfer or otherwise dispose of the Shares until the earliest to occur of (i) 18 months after the Effective Date, (ii) the termination of the Collaboration Agreement or (iii) other specified events. Pursuant to the terms of the Investor Agreement, Ultragenyx agreed that, so long as it holds at least 10 % of the Company’s outstanding common stock, the Shares will be subject to a voting agreement, such that until the earliest to occur of certain specified events, and subject to specified conditions, Ultragenyx will, and will cause its permitted transferees to, vote in accordance with the recommendation of the Company’s Board of Directors with respect to specified matters. Accounting Treatment The Company concluded that the Collaboration Agreement and the Stock Purchase Agreement should be combined and treated as a single arrangement for accounting purposes as the agreements were entered into contemporaneously and in contemplation of one another. The Company assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, Ultragenyx, is a customer. The Company identified the following promises in the Collaboration Agreement that were evaluated under the scope of ASC 606: (1) an exclusive worldwide license to the Licensed Products; (2) an obligation to perform research and development services; and (3) an obligation to participate in a joint steering committee. The Company assessed the promised goods and services to determine if they are distinct. Based on this assessment, the Company determined that Ultragenyx cannot benefit from the promised goods and services separately from the others as they are highly interrelated and therefore not distinct. Due to the early stage of the Licensed Products, the research and development services could not be performed by another party. The Company’s skill-set, knowledge and expertise are required to conduct the research and development services and the research and development services are expected to involve significant further development of the Licensed Products. Accordingly, the promised goods and services represent one combined performance obligation and the entire transaction price will be allocated to that single combined performance obligation. The Company determined the transaction price under ASC 606 at the inception of the Collaboration Agreement to be $ 22,513 , which represents the excess proceeds from the equity investment under the Stock Purchase Agreement, when measured at fair value after taking into consideration a discount for lack of marketability, plus the estimated reimbursement of research and development costs, which represents variable consideration. The Company included the estimated reimbursement of research and development costs in the transaction price at the inception of the arrangement because the Company is required to perform research and development services and the contract requires Ultragenyx to reimburse the Company for costs incurred. Also, since the related revenue would be recognized only as the costs are incurred, the Company determined it is not probable that a significant reversal of cumulative revenue would occur. The Company evaluated how much variable consideration related to development and regulatory milestones, and the Company’s potential exercise of its Development Option or Income Share Option per Licensed Product, to include in the transaction price using the most likely amount approach and concluded that no amount should be included in the transaction price due to the high degree of uncertainty and risk associated with these potential payments. The Company also determined that royalties and sales milestones relate solely to the license of intellectual property and are therefore excluded from the transaction price under the sales- or usage-based royalty exception of ASC 606. Revenue related to these royalties and sales milestones will only be recognized when the associated sales occur, and relevant thresholds are met. The Company determined that revenue under the Collaboration Agreement should be recognized over time as Ultragenyx simultaneously receives the benefit from the Company as the Company performs under the single performance obligation over time. The Company will recognize revenue for the single performance obligation using a cost-to-cost input method as the Company has concluded it best depicts the research and development and joint steering committee participation services performed. Under this method, the transaction price is recognized over the contract’s entire performance period, using costs incurred relative to total estimated costs to determine the extent of progress towards completion. During the three months ended March 31, 2022 and March 31, 2021, the Company recognized $ 1,925 and $ 3,335 , respectively, of related party collaboration revenue, associated with its collaboration with Ultragenyx related to research and development services performed during the period and the corresponding cost reimbursement receivable. 12 The following table presents changes in the balances of the Company’s related party collaboration receivables and contract liabilities during the three months ended March 31, 2022: Balance as of December 31, 2021 Additions Deductions Balance as of March 31, 2022 Related party collaboration receivable $ 110 $ 14 $ ( 110 ) $ 14 Contract liabilities: Deferred revenue 8,080 — ( 1,910 ) 6,170 The changes in the related party collaboration receivables balance during the three months ended March 31, 2022 are driven by amounts owed to the Company for research and development services provided, partially offset by the collections received from Ultragenyx during the three months ended March 31, 2022. As of March 31, 2022 and December 31, 2021, there was $ 6,170 and $ 8,080 , respectively, of deferred revenue related to the Collaboration Agreement, which is classified as either current or non-current in the accompanying condensed consolidated balance sheet based on the period the services are expected to be delivered. Additionally, as of March 31, 2022 and December 31, 2021, there was $ 14 and $ 110 , respectively, of related party collaboration receivables related to reimbursable costs expected to be received from Ultragenyx for research and development services performed. Costs incurred relating to the Collaboration Agreement consist of internal and external research and development costs, which primarily include salaries and benefits, lab supplies, preclinical research studies, clinical studies, consulting services, and commercial development. These costs are included in research and development expenses in the Company’s condensed consolidated statement of operations during the three months ended March 31, 2022. 4. Fair Value of Financial Assets and Liabilities The following tables present information about the Company’s assets and liabilities that are measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair values: Fair Value Measurements as of March 31, 2022 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ — $ 71,287 $ — $ 71,287 Available-for-sale securities $ — $ 50,339 $ — $ 50,339 $ — $ 121,626 $ — $ 121,626 Fair Value Measurements as of December 31, 2021 Using: Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ — $ 75,224 $ — $ 75,224 Available-for-sale securities — 88,643 — 88,643 $ — $ 163,867 $ — $ 163,867 As of March 31, 2022 and December 31, 2021, the fair values of the Company’s available-for-sale securities, which consisted of corporate bond securities as of March 31, 2022 and treasury bills, commercial paper, and corporate bond securities as of December 31, 2021, were determined using Level 2 inputs. During the three months ended March 31, 2022 and the year ended December 31, 2021, there were no transfers between Level 1, Level 2 and Level 3. The fair value of the Company’s cash, restricted cash, accounts payable, and accrued expenses and other current liabilities approximate their carrying value due to their short-term maturities. 5. Available-for-Sale Securities As of March 31, 2022, the fair value of available-for-sale securities by type of security was as follows: 13 March 31, 2022 Amortized Cost Gross Unrealized Gain Gross Unrealized Loss Fair Value Investments: Corporate bond securities 50,390 — ( 51 ) 50,339 $ 50,390 $ — $ ( 51 ) $ 50,339 As of December 31, 2021, the fair value of available-for-sale securities by type of security was as follows: December 31, 2021 Amortized Cost Gross Unrealized Gain Gross Unrealized Loss Fair Value Investments: Treasury bill $ 2,800 $ — $ — $ 2,800 Corporate bond securities 83,889 — ( 45 ) 83,844 Commercial paper 1,999 — — 1,999 $ 88,688 $ — $ ( 45 ) $ 88,643 The estimated fair value and amortized cost of the Company’s available-for-sale securities as of March 31, 2022, by contractual maturity are summarized as follows: March 31, 2022 Amortized Cost Fair Value Due in one year or less $ 50,390 $ 50,339 Total available-for-sale securities $ 50,390 $ 50,339 The weighted average maturity of the Company’s available-for-sale securities as of March 31, 2022 was approximately 0.7 years . The estimated fair value and amortized cost of the Company’s available-for-sale securities as of December 31, 2021 by contractual maturity are summarized as follows: December 31, 2021 Amortized Cost Fair Value Due in one year or less $ 88,688 $ 88,643 Total available-for-sale securities $ 88,688 $ 88,643 The weighted average maturity of the Company’s available-for-sale securities as of December 31, 2021 was approximately 0.7 years . 14 6. Property and Equipment Property and equipment consists of the following: March 31, 2022 December 31, 2021 Furniture and fixtures $ 213 $ 212 Laboratory equipment 10,800 10,719 Leasehold improvements 4,713 4,713 Computer equipment 436 436 Computer software 553 553 Construction in process 1,753 1,490 18,468 18,123 Less accumulated depreciation 12,371 11,661 $ 6,097 $ 6,462 Depreciation expense was $ 709 and $ 734 for the three months ended March 31, 2022 and March 31, 2021, respectively. 7. Prepaid Expenses and Other Current Assets Prepaid expenses and other current assets consist of the following: March 31, 2022 December 31, 2021 Prepaid research and development expenses $ 7,697 $ 6,015 Prepaid expenses and other assets 10,847 8,708 $ 18,544 $ 14,723 8. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consist of the following: March 31, 2022 December 31, 2021 Accrued research and development $ 3,437 $ 1,507 Accrued compensation 2,270 3,084 Accrued other 3,733 4,937 $ 9,440 $ 9,528 9. Stockholders’ Equity In July 2019, the Company issued and sold in a private placement (i) 10,607,525 shares of its common stock at a price per share of $ 4.65 and (ii) 2,295,699 pre-funded warrants to purchase shares of its common stock at a price per warrant of $ 4.64 . Each pre-funded warrant was exercisable for one share of common stock at an exercise price of $ 0.01 and the pre-funded warrants had no expiration date. In October 2020, 137,370 of these pre-funded warrants were exercised. During the three months ended March 31, 2022, 2,158,329 of these pre-funded warrants were exercised. No warrants were exercised during the three months ended March 31, 2021. In March 2021, the Company issued and sold in a public offering 25,000,000 shares of its common stock at a price to the public of $ 5.575 per share. The Company received net proceeds of $ 134,878 after deducting underwriting discounts and commissions and offering expenses. 10. Equity-Based Compensation In connection with the closing of the Company’s initial public offering, the Board of Directors and stockholders approved the 2018 Omnibus Incentive Plan (the “2018 Plan”), which provides for the reservation of 5,001,000 shares of common stock for equity awards. On June 16, 2020, the Company’s stockholders approved the 2020 Equity Incentive Plan (“2020 Plan”) which consists of (i) 3,000,000 shares of common stock and (ii) additional shares of common stock (up to 4,879,025 ) as is equal to (i) the number of shares reserved under the 2018 Plan that remain available for grant under the 2018 Plan as of immediately prior to the date the 2020 Plan was approved by the Company’s stockholders and (ii) the number of shares subject to awards granted under the 2018 Plan which awards 15 expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right. In June 2021, the Company’s stockholders approved an amendment to the 2020 Plan to reserve an additional 7,000,000 shares of common stock for issuance under the plan. As of March 31, 2022 , 4,901,412 shares remained available for future issuance under the 2020 Plan. Under the 2020 Plan, stock options may not be granted at less than fair value on the date of grant. In June 2021, the Company’s stockholders also approved the 2021 Employee Stock Purchase Plan (“ESPP”) , which provides for 1,102,885 shares to be available for purchase by eligible employees according to its terms . The first offering period under the ESPP commenced on September 1, 2021. As of March 31, 2022 , 1,058,204 shares remained available for future issuance under the ESPP. During the three months ended March 31, 2022 and March 31, 2021, the Company granted options for the purchase of 2,621,075 and 2,397,495 shares of common stock, respectively. During the three months ended March 31, 2022, the Company granted 1,451,495 restricted stock units. The Company did not grant restricted stock units for the three months ended March 31, 2021. The Company recorded equity-based compensation expense related to all of its share-based awards to employees and non-employees in the following captions within its condensed consolidated statements of operations: Three Months Ended March 31, 2022 2021 Research and development $ 1,180 $ 1,490 General and administrative 1,432 1,417 Total $ 2,612 $ 2,907 11. Income Taxes During the three months ended March 31, 2022 and 2021, the Company recorded no income tax benefits for the net operating losses incurred or for the research and development tax credits and orphan drug credits generated in each year due to its uncertainty of realizing a benefit from those items. The Company has provided a valuation allowance for the full amount of its net deferred tax assets because, at March 31, 2022 and December 31, 2021, it was more likely than not that any future benefit from deductible temporary differences and net operating loss and tax credit carryforwards would not be realized. As of March 31, 2022 and December 31, 2021, the Company had no t recorded any amounts for unrecognized tax benefits. The Company files income tax returns as prescribed by the tax laws of the jurisdictions in which it operates. In the normal course of business, the Company is subject to examination by federal and state jurisdictions, where applicable. There are currently no pending income tax examinations. The Company’s C-Corporation tax years beginning with the year ended December 31, 2019 are open under statute. Any tax credit or net operating loss carryforward can be adjusted in future periods after the respective year of generation’s statute of limitation has closed. 12. Commitments and Contingencies Legal Proceedings The Company may periodically become subject to legal proceedings and claims arising in connection with ongoing business activities, including claims or disputes related to patents that have been issued or that are pending in the field of research on which the Company is focused. The Company is not aware of any material legal proceedings or claims as of March 31, 2022. 13. Net Loss per Share Basic and diluted net loss per share attributable to common stockholders were calculated as follows: The numerator for basic and diluted net loss per share attributable to common stockholders is as follows: Three Months Ended March 31, 2022 2021 Net loss attributable to common stockholders $ ( 25,328 ) $ ( 16,900 ) 16 The denominator is as follows: Three Months Ended March 31, 2022 2021 Weighted average shares of common stock outstanding, basic and diluted 112,607,322 87,108,865 Weighted average shares of pre-funded warrants to purchase common stock — 2,158,329 Total 112,607,322 89,267,194 Net loss per share attributable to common stockholders, basic and diluted is as follows: Three Months Ended March 31, 2022 2021 Net loss per share attributable to common stockholders $ ( 0.22 ) $ ( 0.19 ) The following potential common stock equivalents, presented based on amounts outstanding at each period end, were excluded from the calculation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect for the three months ended March 31: 2022 2021 Options to purchase shares of common stock 8,407,375 5,076,747 Unvested shares of common stock — 22,804 Unvested restricted stock units 1,643,558 322,901 10,050,933 5,422,452 14. Subsequent Events In April 2022, the Company implemented changes to its corporate strategy to prioritize the advancement of its key programs, SGT-001 and SGT-003. In connection with the changes to corporate operations, the Company reduced headcount by approximately 35 percent. The Company expects to substantially complete the restructuring in the second quarter of 2022. The Company estimates total restructuring costs of approximately $ 1,700 related to severance and other employee termination benefits. The Company expects that approximately $ 500 would be paid during the three months ended June 30, 2022 and approximately $ 1,200 would be paid during the remainder of 2022. 17