Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2018 | Jul. 31, 2018 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2018 | |
Document Fiscal Year Focus | 2,018 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | AVRO | |
Entity Registrant Name | AVROBIO, INC. | |
Entity Central Index Key | 1,681,087 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Common Stock, Shares Outstanding | 23,936,838 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Jun. 30, 2018 | Dec. 31, 2017 |
Current assets: | ||
Cash and cash equivalents | $ 155,015 | $ 5,963 |
Prepaid expenses and other current assets | 1,132 | 345 |
Total current assets | 156,147 | 6,308 |
Property and equipment, net | 2,050 | 349 |
Other assets | 533 | 365 |
Total assets | 158,730 | 7,022 |
Current liabilities: | ||
Accounts payable | 4,193 | 527 |
Accrued expenses and other current liabilities | 5,438 | 2,098 |
Total current liabilities | 9,631 | 2,625 |
Warrant to purchase redeemable convertible preferred stock | 35 | |
Derivative liability | 371 | |
Deferred rent, net of current portion | 781 | 126 |
Other long-term liability | 500 | |
Total liabilities | 10,412 | 3,657 |
Commitments and contingencies (Note 13) | ||
Redeemable convertible preferred stock (Note 8) | 26,500 | |
Stockholders' equity (deficit): | ||
Common stock, $0.0001 par value; 150,000,000 and 51,000,000 shares authorized as of June 30, 2018 and December 31, 2017, respectively; 23,936,838 and 2,581,474 shares issued as of June 30, 2018 and December 31, 2017, respectively; 23,722,049 and 2,305,173 shares outstanding as of June 30, 2018 and December 31, 2017, respectively | 2 | |
Additional paid-in capital | 192,443 | 339 |
Accumulated deficit | (44,127) | (23,474) |
Total stockholders' equity (deficit) | 148,318 | (23,135) |
Total liabilities, redeemable convertible preferred stock and stockholders' equity (deficit) | $ 158,730 | $ 7,022 |
Condensed Consolidated Balance3
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 2018 | Dec. 31, 2017 |
Statement of Financial Position [Abstract] | ||
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, authorized | 150,000,000 | 51,000,000 |
Common stock, issued | 23,936,838 | 2,581,474 |
Common stock, outstanding | 23,722,049 | 2,305,173 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2018 | Jun. 30, 2017 | Jun. 30, 2018 | Jun. 30, 2017 | |
Operating expenses: | ||||
Research and development | $ 7,407 | $ 1,881 | $ 13,054 | $ 3,315 |
General and administrative | 2,140 | 661 | 4,281 | 1,271 |
Total operating expenses | 9,547 | 2,542 | 17,335 | 4,586 |
Loss from operations | (9,547) | (2,542) | (17,335) | (4,586) |
Other income (expense): | ||||
Interest income | 234 | 11 | 392 | 15 |
Change in fair value of preferred stock warrant liability | (150) | (162) | ||
Change in fair value of derivative liability | (1,042) | (3) | (1,629) | (35) |
Other expense | (2) | (15) | (5) | |
Total other income (expense), net | (960) | 8 | (1,414) | (25) |
Net loss | (10,507) | (2,534) | (18,749) | (4,611) |
Comprehensive loss | (10,507) | (2,534) | (18,749) | (4,611) |
Reconciliation of net loss to net loss attributable to common stockholders: | ||||
Net loss | (10,507) | (2,534) | (18,749) | (4,611) |
Accretion of issuance costs on redeemable convertible preferred stock | (2,243) | (47) | ||
Net loss attributable to common stockholders-basic and diluted | $ (10,507) | $ (2,534) | $ (20,992) | $ (4,658) |
Net loss per share attributable to common stockholders - basic and diluted (Note 12) | $ (2.98) | $ (1.15) | $ (7.16) | $ (2.12) |
Weighted-average number of common shares used in computing net loss per share attributable to common stockholders-basic and diluted | 3,529,269 | 2,202,735 | 2,930,358 | 2,192,283 |
Condensed Consolidated Stateme5
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2018 | Jun. 30, 2017 | |
Cash flows from operating activities: | ||
Net loss | $ (18,749) | $ (4,611) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 566 | 64 |
Depreciation and amortization expense | 66 | 15 |
Impairment loss of property and equipment | 235 | |
Amortization of deferred offering costs | 22 | 2 |
Deferred rent expense | (23) | 135 |
Change in fair value of preferred stock warrant liability | 162 | |
Change in fair value of derivative liability | 1,629 | 35 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | (768) | (176) |
Other assets | (300) | (3) |
Accounts payable | 3,029 | (545) |
Accrued expenses and other current liabilities | (98) | 626 |
Net cash used in operating activities | (14,229) | (4,458) |
Cash flows from investing activities: | ||
Change in restricted cash | 24 | |
Purchases of property and equipment | (424) | (62) |
Net cash used in investing activities | (400) | (62) |
Cash flows from financing activities: | ||
Proceeds from issuance of redeemable convertible preferred stock, net of issuance costs | 58,258 | 3,468 |
Payments of issuance costs on debt facility | (32) | |
Proceeds from issuance of common shares upon completion of initial public offering, net of offering costs | 105,423 | |
Net cash provided by financing activities | 163,681 | 3,436 |
Net increase (decrease) in cash and cash equivalents | 149,052 | (1,084) |
Cash and cash equivalents at beginning of period | 5,963 | 5,357 |
Cash and cash equivalents at end of period | 155,015 | 4,273 |
Supplemental disclosure of non-cash investing and financing activities: | ||
Purchases of property and equipment included in accounts payable | 756 | 240 |
Purchases of property and equipment paid for by landlord | 842 | |
Deferred offering costs included in accrued expenses | 1,241 | 11 |
Accretion of issuance costs related to redeemable convertible preferred stock | 2,240 | 47 |
Issuance of warrants associated with debt facility | $ 18 | |
Property and equipment held for sale | $ 19 |
Nature of the Business and Basi
Nature of the Business and Basis of Presentation | 6 Months Ended |
Jun. 30, 2018 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of the Business and Basis of Presentation | 1. Nature of the Business and Basis of Presentation AVROBIO, Inc. (the “Company” or “AVROBIO”) is a clinical stage gene therapy company focused on developing potentially curative ex vivo The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including but not limited to, risks associated with completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize revenue from product sales. Basis of Presentation The accompanying condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification (ASC) and Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB). The unaudited condensed consolidated interim financial statements have been prepared on the same basis as the audited annual consolidated financial statements as of and for the year ended December 31, 2017, and, in the opinion of management, reflect all adjustments, consisting of normal recurring adjustments, necessary for the fair presentation of the Company’s financial position as of June 30, 2018, and the results of its operations and its cash flows for the three and six months ended June 30, 2018 and 2017. The results for the six months ended June 30, 2018 are not necessarily indicative of the results to be expected for the year ending December 31, 2018, any other interim periods, or any future year or period. These interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2017, and the notes thereto, which are included elsewhere in the Company’s final prospectus for its initial public offering (“IPO”), filed with the Securities and Exchange Commission (“SEC”) pursuant to Rule 424(b)(4) under the Securities Act of 1933, as amended, on June 21, 2018 (the “Prospectus”). The accompanying condensed financial statements reflect the application of certain significant accounting policies as described below and elsewhere in these notes to the financial statements. As of June 30, 2018, the Company’s significant accounting policies and estimates, which are detailed in the Company’s Prospectus, have not changed. Reverse Stock Split On June 1, 2018, the Board approved a 1-for-4.312 Initial Public Offering On June 20, 2018, the Company’s registration statement on Form S-1 |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2018 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Subsequent Event Considerations The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. See Note 15. Use of Estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual for research and development expenses, stock-based compensation expense, the valuation of equity and derivative instruments and the recoverability of the Company’s net deferred tax assets and related valuation allowance. 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 materially from those estimates. Cash and Cash Equivalents The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in interest-bearing money market accounts. Cash equivalents are carried at cost, which approximates their fair market value. Concentrations of Credit Risk The Company has no significant off-balance Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP (see Note 3). Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1—Quoted prices in active markets for identical assets or liabilities. • Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3—Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision making group, in making decisions on how to allocate resources and assess performance. The Company’s chief operating decision maker is the chief executive officer (“CEO”). The Company and the CEO view the Company’s operations and manage its business as one operating segment. All material long-lived assets of the Company reside in the United States. Research and Development Costs Research and development costs are expensed as incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries, stock-based compensation and benefits, facilities costs, depreciation, third-party license fees, and external costs of outside vendors engaged to conduct preclinical development activities and clinical trials as well as to manufacture research and development materials. Non-refundable The Company has entered into various research and development related contracts with parties both inside and outside of the United States. The payments to these agreements are recorded as research and development expenses as incurred. The Company records accrued liabilities for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs. Stock-Based Compensation For stock-based awards issued to employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of grant and recognizes compensation expense for those awards over the requisite service period, which is generally the vesting period of the respective award. The Company issues stock-based awards with only service-based vesting conditions and records the expense for these awards using the straight-line method. The Company has not issued any stock-based awards with performance- or market-based vesting conditions. The Company accounts for forfeitures as they occur. Stock-based awards issued to non-employees non-employees The Company classifies stock-based compensation expense in its consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s cash compensation costs are classified. Given the absence of an active market for the Company’s common stock prior to the IPO, the Company and the Board, the members of which the Company believes have extensive business, finance, and venture capital experience, were required to estimate the fair value of the Company’s common stock at the time of each grant of a stock-based award. The Company and the Board determined the estimated fair value of the Company’s equity instruments based on a number of factors, including external market conditions affecting the biotechnology industry sector. The Company and the Board utilized various valuation methodologies in accordance with the framework of the American Institute of Certified Public Accountants’ Technical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation arm’s-length The fair value of each stock option award is estimated on the date of grant using the Black-Scholes option pricing model. As there was no public market for its common stock prior to June 21, 2018, which was the first day of trading, and as the trading history of the Company’s common stock was limited through June 30, 2018, the Company determined the volatility for awards granted based on an analysis of reported data for a group of guideline companies that issued options with substantially similar terms. The expected volatility has been determined using a weighted-average of the historical volatility measures of this group of guideline companies. The Company expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded stock price. The expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. The Company has not paid, and does not anticipate paying, cash dividends on its common stock; therefore, the expected dividend yield is assumed to be zero. See Note 10 for the assumptions used by the Company in determining the grant date fair value of stock-based awards granted, as well as a summary of the stock-based award activity under the Company’s stock-based compensation plan for the six months ended June 30, 2018. Warrant to Purchase Preferred Stock Prior to the IPO, the Company classified the warrant for the purchase of shares of its redeemable convertible preferred stock (see Note 7) as a liability on its consolidated balance sheets as the warrant is a free-standing financial instrument that may require the Company to transfer assets upon exercise. The preferred stock warrant liability was initially recorded at fair value upon the date of issuance and is subsequently remeasured to fair value at each reporting date. Changes in the fair value of the warrant to purchase preferred stock are recognized as a component of other income (expense), net in the consolidated statements of operations and comprehensive loss. Changes in the fair value of the warrant to purchase preferred stock will continue to be recognized until the warrant is exercised, expires or qualifies for equity classification. The Company utilizes the Black-Scholes option-pricing model, which incorporates assumptions and estimates, to value the warrant. The Company assesses these assumptions and estimates on a quarterly basis as additional information impacting the assumptions is obtained. Estimates and assumptions impacting the fair value measurement include the fair value per share of the underlying redeemable convertible preferred stock issuable upon exercise of the warrant, the remaining contractual term of the warrant, the risk-free interest rate, the expected dividend yield and the expected volatility of the price of the underlying redeemable convertible preferred stock. Upon the IPO, the warrant to purchase preferred stock was converted to a warrant to purchase common stock. The carrying amount of warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital. Comprehensive Income (Loss) Comprehensive income (loss) is defined as the change in stockholders’ equity of a business enterprise during a period from transactions and other events and circumstances from non-owner Leases The Company categorizes leases at their inception as either operating or capital leases. On certain lease arrangements, the Company may receive rent holidays or other incentives. The Company recognizes lease costs on a straight-line basis once control of the space is achieved, without regard to deferred payment terms, such as rent holidays, that defer the commencement date of required payments or escalating payment amounts. The difference between required lease payments and rent expense has been recorded as deferred rent and other accrued expenses and other current liabilities in the accompanying consolidated balance sheets. Additionally, incentives received are treated as a reduction of costs over the term of the agreement, as they are considered an inseparable part of the lease agreement. Net Income (Loss) per Share Attributable to Common Stockholders Net income (loss) per share attributable to common stockholders is determined using the two-class Under the two-class two-class if-converted Emerging Growth Company Status The Company is an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act, or JOBS Act, and may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies. The Company may take advantage of these exemptions until the Company is no longer an “emerging growth company.” Section 107 of the JOBS Act provides that an “emerging growth company” can take advantage of the extended transition period afforded by the JOBS Act for the implementation of new or revised accounting standards. The Company has elected to use the extended transition period for complying with new or revised accounting standards and as a result of this election, its consolidated financial statements may not be comparable to companies that comply with public company effective dates. The Company may take advantage of these exemptions up until the last day of the fiscal year following the fifth anniversary of an offering or such earlier time that it is no longer an “emerging growth company”. Recently Issued Accounting Pronouncements In June 2018, the FASB issued ASU No. 2018-07, “Compensation—Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting” non-employees 505-50. 505-50 non-employee non-employees, non-employee non-employee non-public 2018-07 In July 2017, the FASB issued ASU 2017-11, Earnings Per Share (Topic 260), Distinguishing Liabilities from Equity (Topic 480), Derivatives and Hedging (Topic 815) I. Accounting for Certain Financial Instruments with Down Round Features II. Replacement of the Indefinite Deferral for Mandatorily Redeemable Financial Instruments of Certain Nonpublic Entities and Certain Mandatorily Redeemable Noncontrolling Interests with a Scope Exception . 2017-11 In May 2017, the FASB issued ASU No. 2017-09, Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting 2017-09”), 2017-09 In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash 2016-18”), 2016-18 In August 2016, the FASB issued ASU No. 2016-15, Statement of Cash Flows: Classification of Certain Cash Receipts and Cash Payments 2016-15”), 2016-15 In February 2016, the FASB issued ASU No. 2016-02 , Leases (Topic 842) 2016-02”), right-of-use 2016-02 Leases not-for-profit In September 2017, the FASB issued ASU No. 2017-13, Revenue Recognition (Topic 605), Revenue from Contracts with Customers (Topic 606), Leases (Topic 840), and Leases (Topic 842) ASU 2016-02 2016-02. ASU 2016-02 |
Fair Value of Financial Assets
Fair Value of Financial Assets and Liabilities | 6 Months Ended |
Jun. 30, 2018 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Assets and Liabilities | 3. Fair Value of Financial Assets and Liabilities The following table presents information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values as of June 30, 2018 and December 31, 2017, respectively: Fair Value Measurements as of June 30, 2018 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 154,549 $ — $ — $ 154,549 Fair Value Measurements as of December 31, 2017 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 5,684 $ — $ — $ 5,684 Restricted cash 24 — — 24 $ 5,708 $ — $ — $ 5,708 Liabilities: Derivative liability $ — $ — $ 371 $ 371 Warrant to purchase redeemable convertible preferred stock — — 35 35 $ — $ — $ 406 $ 406 During the six months ended June 30, 2018 and 2017, there were no transfers between Level 1, Level 2 and Level 3. Valuation of the Warrant to Purchase Preferred Stock The warrant to purchase preferred stock liability in the table above is composed of the fair value of a warrant to purchase shares of Series A Preferred Stock that was issued to a lender in connection with a loan and security agreement in 2017. The fair value of the warrant to purchase preferred stock was determined based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy. In order to determine the fair value of the warrant to purchase preferred stock, the Company utilized available facts and circumstances to estimate the number of shares of Series A Preferred Stock for which the warrant will ultimately be exercisable. The Company then used the Black-Scholes option-pricing model, which incorporates assumptions and estimates, to value the warrant to purchase preferred stock. Estimates and assumptions impacting the fair value measurement include the fair value of the underlying shares of Series A Preferred Stock, the remaining contractual term of the warrant, risk-free interest rate, expected dividend yield and expected volatility of the price of the underlying preferred stock. The Company determined the fair value of the underlying preferred stock based on various valuation methodologies. The Company lacks company-specific historical and implied volatility information of its stock. Therefore, it estimates its expected stock volatility based on the historical volatility of publicly traded guideline companies for a term equal to the remaining contractual term of the warrant. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve for time periods approximately equal to the remaining contractual term of the warrant. The Company estimated no expected dividend yield based on the fact that the Company has never paid or declared dividends and does not intend to do so in the foreseeable future. Upon the IPO, the warrant to purchase preferred stock was converted to a warrant to purchase common stock. The carrying amount of warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital. The following table sets forth a summary of changes in the fair value of the Company’s preferred stock warrant liability for which fair value was determined by Level 3 inputs: Warrant Liability Balance as of December 31, 2017 $ 35 Change in fair value 162 Conversion of preferred stock warrant to common stock warrant (197 ) Balance as of June 30, 2018 $ — Valuation of Derivative In January 2016, in connection with a license agreement entered into with University Health Network (“UHN”), and as part of the initial consideration for the license, the Company issued 1,161,665 shares of common stock to UHN pursuant to a stock purchase agreement (the “Stock Purchase Agreement”). The Stock Purchase Agreement contains a provision requiring the Company to make a cash payment to UHN of up to $2,000 if UHN’s fully diluted ownership is reduced within specified percentages as part of an IPO by the Company. The Company concluded the anti-dilution feature represented a derivative instrument and should be measured at fair value, with changes in fair value recognized as a gain or loss to other income (expense), net in the consolidated statements of operations and comprehensive loss. The initial fair value of the derivative of $49 was recorded as research and development expense in January 2016. On June 30, 2017, the Company remeasured the fair value of the derivative, using current assumptions, resulting in an increase in fair value of $3 and $35, which was recorded in other expense in the accompanying condensed consolidated statements of operations and comprehensive loss for the three months and six months ended June 30, 2017, respectively. On June 21, 2018, in connection with the Company’s IPO, the Company remeasured the fair value of the derivative to $2,000 as the Company was required to pay the dilution payment as mentioned above, which the Company paid in July 2018. An increase in fair value of $1,042 and $1,629 was recorded in other expense in the accompanying condensed consolidated statements of operations and comprehensive loss for the three and six months ended June 30, 2018, respectively. The following table sets forth a summary of changes in the fair value of the Company’s derivative liability for which fair value is determined by Level 3 inputs: Derivative Liability Balance as of December 31, 2017 $ 371 Change in fair value 1,629 Transferred to accrued expenses (2,000 ) Balance as of June 30, 2018 $ — |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 6 Months Ended |
Jun. 30, 2018 | |
Text Block [Abstract] | |
Prepaid Expenses and Other Current Assets | 4. Prepaid Expenses and Other Current Assets Prepaid expenses and other current assets consisted of the following: June 30, 2018 December 31, 2017 Prepaid development costs $ 762 $ 163 Prepaid rent 76 122 Other current assets 294 60 $ 1,132 $ 345 |
Property and Equipment, Net
Property and Equipment, Net | 6 Months Ended |
Jun. 30, 2018 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net consisted of the following: June 30, 2018 December 31, 2017 Laboratory and office equipment $ 926 $ 126 Leasehold improvements 1,112 240 Computer equipment and software 83 28 2,121 394 Less: Accumulated depreciation and amortization (71 ) (45 ) $ 2,050 $ 349 Depreciation and amortization expense for the three months ended June 30, 2018 and 2017 was $45 and $13, respectively. Depreciation and amortization expense |
Accrued Expenses
Accrued Expenses | 6 Months Ended |
Jun. 30, 2018 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 6. Accrued Expenses Accrued expenses and other current liabilities consisted of the following: June 30, 2018 December 31, 2017 Dilution payment (Note 3) $ 2,000 $ — Compensation and benefit costs 846 794 Short-term deferred rent 171 9 Research and development costs 753 831 IPO issuance cost 693 — Property and equipment 667 — Other liabilities 308 464 $ 5,438 $ 2,098 |
Loan Agreement and Warrant to P
Loan Agreement and Warrant to Purchase Preferred Stock | 6 Months Ended |
Jun. 30, 2018 | |
Statement of Stockholders' Equity [Abstract] | |
Loan Agreement and Warrant to Purchase Preferred Stock | 7. Loan Agreement and Warrant to Purchase Preferred Stock On June 23, 2017, the Company entered into the Loan Agreement with a lender, which provided for the issuance of term loans of up to $10,000, subject to the achievement of various development and corporate milestones. Any outstanding principal amounts under the Loan Agreement will accrue interest at a floating per annum rate equal to the greater of 1% and the “prime rate,” as defined in the Loan Agreement, minus 3%. Payments on the Loan Agreement are interest only, payable monthly in arrears, until November 1, 2018, which can be extended by six months if the third tranche is drawn. Thereafter, principal and interest amounts are repayable over a 30-month 24-month The Loan Agreement contains customary indemnification obligations and customary events of default. In the event of default by the Company under the Loan Agreement, the lender would be entitled to exercise their remedies thereunder, including the right to accelerate the debt, upon which the Company may be required to repay all amounts then outstanding under the Loan Agreement or the lender may take possession of the collateral securing the Loan Agreement. No events of default had occurred through June 30, 2018. The Loan Agreement also includes certain restrictions on, among other things, the Company’s ability to incur additional indebtedness, change the name or location of its business, merge with or acquire other entities, pay dividends or make other distributions to holders of the Company’s capital stock, make certain investments, engage in transactions with affiliates, create liens, open new deposit accounts, sell assets or pay subordinated debt. In connection with the Loan Agreement, the Company agreed to issue a warrant to the lender for the purchase of up to 188,702 shares of the Company’s Series A Preferred Stock with an exercise price of $0.7949 per share. The warrant expires on June 22, 2027. The warrant is initially exercisable for the purchase of up to 28,305 shares of Series A Preferred Stock and can become exercisable for up to an additional 160,397 shares of Series A Preferred Stock based on the amounts drawn under the Loan Agreement. On the issuance date of the warrant, the Company recorded a deferred financing cost and a liability for the warrant to purchase preferred stock in the Company’s consolidated balance sheet equal to the issuance-date fair value of the warrant. Upon closing of the IPO, the carrying amount of the warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital (Note 3). The Company recognized a loss of $150 and $162 in the condensed consolidated statements of operations and comprehensive loss for the three and six months ended June 30, 2018, respectively, related to the change in fair value of the warrant. |
Redeemable Convertible Preferre
Redeemable Convertible Preferred Stock | 6 Months Ended |
Jun. 30, 2018 | |
Text Block [Abstract] | |
Redeemable Convertible Preferred Stock | 8. Redeemable Convertible Preferred Stock Prior to the IPO, the authorized capital stock of the Company included 63,491,857 shares of $0.0001 par value preferred stock, of which 3,333,333 shares have been designated as Series Seed Preferred Stock, 31,639,202 shares have been designated as Series A Preferred Stock and 28,519,322 shares have been designated as Series B Preferred Stock. In January 2018, the Company issued and sold 28,519,322 shares of Series B Preferred Stock, at a price of $2.1389 per share, for total proceeds of $58,757, net of issuance costs of $2,243. As of December 31, 2017, the Preferred Stock consisted of the following: December 31, 2017 Preferred Shares Authorize Preferred Shares Issued and Outstanding Carrying Value Liquidation Preference Common Stock Issuable Upon Conversion Series Seed preferred stock 3,333,333 3,333,333 $ 1,500,000 $ 1,500,000 806,711 Series A preferred stock 31,639,202 31,450,499 25,000,000 25,000,000 7,611,438 34,972,535 34,783,832 $ 26,500,000 $ 26,500,000 8,418,149 Upon closing of the IPO, the Preferred Stock as of December 31, 2017, together with Series B Preferred Stock issued in January 2018, were converted to 15,320,213 shares of common stock. The holders of the Company’s Preferred Stock had certain voting, dividend, and redemption rights, as well as liquidation preferences and conversion privileges. All rights, preferences, and privileges associated with the preferred stock were terminated at the time of the Company’s IPO in conjunction with the conversion of all outstanding shares of Preferred Stock into shares of common stock. |
Common Stock
Common Stock | 6 Months Ended |
Jun. 30, 2018 | |
Federal Home Loan Banks [Abstract] | |
Common Stock | 9. Common Stock As of June 30, 2018, the authorized capital stock of the Company included 150,000,000 shares of common stock, $0.0001 par value, respectively. At June 30, 2018 and December 31, 2017, the Company has reserved the following shares of common stock for future issuance: June 30, 2018 December 31, 2017 Shares reserved for Series Seed Preferred Stock outstanding — 806,711 Shares reserved for Series A Preferred Stock outstanding — 7,611,438 Shares reserved for issuance under the 2018 Stock Option and Incentive Plan 533,911 — Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 223,200 — Shares reserved for vesting of restricted stock awards 214,789 276,301 Shares reserved for exercise of outstanding stock options 1,871,139 1,034,961 Shares reserved for issuance under the 2015 Stock Option and Grant Plan — 143,717 Total shares of authorized common stock reserved for future issuance 2,843,039 9,873,128 |
Stock-Based Compensation
Stock-Based Compensation | 6 Months Ended |
Jun. 30, 2018 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock-Based Compensation | 10. Stock-Based Compensation Amended and Restated 2015 Stock Option and Grant Plan The Company’s Amended and Restated 2015 Stock Option and Grant Plan, (the “2015 Plan”) provides for the Company to issue restricted stock awards and restricted stock units, or to grant incentive stock options or non-statutory non-statutory The total number of common shares that may be issued under the 2015 Plan was 2,008,564 shares. Following the IPO, no further grants will be made under 2015 plan. Shares that expire, are terminated, surrendered or canceled under the 2015 Plan without having been fully exercised will be available for future awards under the 2018 Plan (as defined below). In addition, shares of common stock that are tendered to the Company by a participant to exercise an award are added to the number of shares of common stock available for future awards. The 2015 Plan is administered by the Board. Equity awards granted to employees and members of the Board typically vest over four years. 2018 Stock Option and Incentive Plan The Company’s 2018 Stock Option and Incentive Plan (the “2018 Plan”) was adopted by the Board June 1, 2018 and approved by stockholders on June 7, 2018 and became effective upon the effectiveness of the Company’s Registration Statement on Form S-1. The Company initially reserved 616,300 shares of its common stock for the issuance of awards under the 2018 Plan. The 2018 Plan provides that the number of shares reserved and available for issuance under the plan will automatically increase each January 1, beginning on January 1, 2019, by 4% of the outstanding number of shares of our common stock on the immediately preceding December 31, or such lesser number of shares as determined by its compensation committee. This number is subject to adjustment in the event of a stock split, stock dividend or other change in its capitalization. The total number of common shares that may be issued under the 2018 Plan was 616,300 shares as of June 30, 2018, of which 533,911 shares remained available for future grant. During the six months ended June 30, 2018 and 2017, the Company granted options to purchase 836,178 and 196,872 shares of common stock to employees and members of the Board, respectively. 2018 Employee Stock Purchase Plan The Company’s 2018 Employee Stock Purchase Plan (the “ESPP”) was adopted by the Board June 1, 2018 and approved by stockholders on June 7, 2018 and became effective upon the effectiveness of the Company’s Registration Statement on Form S-1. The total number of common shares that may be issued under the ESPP was 223,200 shares as of June 30, 2018, of which 223,200 shares remained available for future grant. As of June 30, 2018, the initial purchase period under the ESPP has not yet commenced. Stock Option Valuation The assumptions that the Company used to determine the grant-date fair value of stock options granted to employees and members of the Board were as follows, presented on a weighted-average basis: Six Months Ended June 30, 2018 2017 Expected option life (years) 6.06 6.08 Risk-free interest rate 2.73 % 1.90 % Expected volatility 83.86 % 85.50 % Expected dividend yield — % — % The following table summarizes the Company’s stock option activity for the six months ended June 30, 2018: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2017 1,034,961 $ 0.78 8.94 $ 3,427 Granted 836,178 $ 6.38 9.74 Exercised — Cancelled or forfeited — Outstanding as of June 30, 2018 1,871,139 $ 3.29 9.02 $ 47,228 Exercisable as of June 30, 2018 309,594 $ 0.72 8.13 $ 8,619 Unvested as of June 30, 2018 1,559,125 $ 3.80 9.20 $ 38,609 The aggregate intrinsic value of stock options is calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock for those stock options that had exercise prices lower than the estimated fair value of the Company’s common stock at June 30, 2018. The weighted-average grant-date fair value of the Company’s stock options granted during the six months ended June 30, 2018 and 2017 was $7.63 and $0.66, respectively. Restricted Common Stock The following table summarizes the Company’s restricted common stock activity for the six months ended June 30, 2018: Number of Shares Weighted-Average Grant Date Fair Value Issued and unvested as of December 31, 2017 276,301 $ 0.42 Vested (61,512 ) 0.42 Forfeited, canceled or expired — — Issued an unvested as of June 30, 2018 214,789 0.42 The total fair value of restricted common stock vested during the six months ended June 30, 2018 and 2017 was $26 and $17, respectively. Stock-Based Compensation Stock-based compensation expense was allocated as follows: Six Months Ended June 30, 2018 2017 Research and development $ 187 $ 35 General and administrative 379 29 Total stock-based compensation expense $ 566 $ 64 As of June 30, 2018, total unrecognized compensation cost related to the unvested stock-based awards was $6,776, which is expected to be recognized over a weighted-average period of 3.61 years. |
License Agreements
License Agreements | 6 Months Ended |
Jun. 30, 2018 | |
Text Block [Abstract] | |
License Agreements | 11. License Agreements Agreements with UHN Fabry License Agreement— On January 27, 2016, the Company entered into an agreement with UHN, pursuant to which UHN granted the Company an option to enter into an exclusive license under the UHN intellectual property related to Fabry disease in accordance with the pre-negotiated non-exclusive know-how, Under this agreement, the Company paid an option fee of CAD $20, an upfront license fee of CAD $75, plus the annual license maintenance fee for the first year. Thereafter, the Company is also required to pay UHN future annual license maintenance fees until the first sale of a licensed product in certain markets. The Company is also obligated to make future milestone payments in an aggregate amount of up to CAD $2,450 upon the achievement of specified milestones as well as royalties on a country-by-country mid-single UHN may terminate the agreement if the Company fails to meet these performance milestones despite using commercially reasonable efforts and the Company is unable to reach agreement with UHN on revised timeframes. The Company’s royalty obligation expires on a licensed product-by-licensed country-by-country Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all licensed products. UHN can terminate the agreement if the Company fails to make any payments within a specified period after receiving written notice of such failure, or in the event that the Company fails to obtain or maintain insurance. Either the Company or UHN may terminate the license agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. The Company can voluntarily terminate the agreement with prior notice to UHN. For the six months ended June 30, 2018 and 2017, no upfront fee and maintenance fees were incurred related to Fabry license agreement. Interleukin 12 License Agreement— On January 27, 2016, the Company entered into an exclusive license agreement with UHN, pursuant to which UHN granted the Company a license to certain patent rights for the commercial development, manufacture, distribution and use of any products or processes resulting from development of those patent rights related to Interleukin 12. Upon execution of this agreement, the Company paid an upfront license fee of CAD $264. In addition, as part of the initial consideration for the license, the Company issued to UHN 1,161,665 shares of the Company’s common stock. The fair value of the shares issued to UHN of $480 and the upfront fee was expensed upon the execution of the agreement. In addition, the Company agreed to pay UHN up to $2,000 upon the closing of an IPO if certain criteria are met. This obligation is considered a derivative instrument and was initially recorded at fair value of $49. The Company is also required to pay UHN future annual license maintenance fees of CAD $50 on each anniversary of the effective date of the license agreement prior to expiration or termination and potential future milestone payments of up to CAD $19,275 upon the achievement of specified clinical and regulatory milestones. The Company also agreed to pay UHN royalties of a low single digit percentage of net sales of licensed products sold by the Company. If the Company grants any sublicense rights under the license agreement, the Company has agreed to pay UHN a low double-digit royalty percentage of any sublicense income received by the Company. The agreement requires the Company to meet certain due diligence requirements based upon specified milestones. The agreement expires on the later of the date the last patent rights expire in the last country or ten years from the date of first sale. UHN can terminate the agreement if the Company fails to make any payments within a specified period after receiving written notice of such failure, or in the event that the Company fails to obtain or maintain insurance. The Company can voluntarily terminate the agreement with prior notice to UHN. Either the Company or UHN may terminate the license agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. For the six months ended June 30, 2018 and 2017, the Company recorded research and development expense related to this agreement with UHN of $41 and $151 (nil for the three months ended June 30, 2018 and 2017), respectively, which consists of upfront fees and license maintenance fees and development milestone payments. Agreement with BioMarin Pharmaceutical Inc. (“BioMarin”) On August 31, 2017, the Company entered into a license agreement with BioMarin, pursuant to which BioMarin granted the Company an exclusive worldwide license under certain intellectual property rights owned or controlled by BioMarin to develop, commercialize and sell products for use in the treatment of Pompe disease. As consideration for this agreement, the Company paid an upfront license fee of $500 in cash and issued 233,765 shares of Series B Preferred Stock to BioMarin at the time of our Series B Preferred Stock financing in January 2018. Both the upfront cash payment of $500 and the value of the shares Series B Preferred Stock issued of $500 were recorded as research and development expense during the year ended December 31, 2017. The Company is also obligated to make future milestone payments of up to $13,000 upon the achievement of certain specified milestones and agreed to pay BioMarin royalties of a low single-digit percentage of net sales of licensed products sold by the Company or its affiliates covered by patent rights in a relevant country. No upfront fees related to the license were recorded for the six months ended June 30, 2018. Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all licensed products throughout the world. BioMarin and the Company can terminate the agreement in the event of a material breach by the other party and failure to cure such breach within a certain period of time. The Company may terminate the agreement at will upon written notice to BioMarin. BioMarin has the right to terminate the agreement upon the Company’s bankruptcy or insolvency, or in the event of any challenge or opposition to the licensed patent rights or related actions brought by the Company or its affiliates or sublicensees, or if the Company, its affiliates or sublicensees knowingly assist a third-party in challenging or otherwise opposing the licensed patent rights, except as required under a court order or subpoena. Agreement with GenStem Therapeutics, Inc. (“GenStem”) On October 2, 2017, the Company entered into a license agreement with GenStem, pursuant to which GenStem granted the Company an exclusive worldwide license, subject to certain retained rights, under certain intellectual property rights owned or controlled by GenStem to develop, commercialize and sell products for use in the treatment of cystinosis. Under this agreement, the Company paid an upfront license fee of $1,000 and is required to make payments upon completion of certain milestones up to an aggregate of $16,000. The Company also agreed to pay GenStem a tiered mid to high single-digit royalty percentage on annual net sales of licensed products as well as a low double-digit percentage of sublicense income received from certain third party licensees. The Company’s royalty obligation expires on a licensed product-by-licensed country-by-country Agreement with Lund University Rights Holders On November 17, 2016, the Company entered into a license agreement with affiliates of Lund University, along with certain other relevant rights holders that may be added from time to time, pursuant to which such rights holders granted to the Company an exclusive worldwide license, subject to certain retained rights, under certain intellectual property rights to develop, commercialize and sell products in any and all uses relevant to Gaucher disease. As consideration for the license, the Company is required to make payments in connection with the achievement of certain milestones up to an aggregate of $550. The agreement expires on the latest of (i) the twentieth anniversary of the end of a certain research project the Company is funding pursuant to an agreement with Lund University, (ii) the expiration of the term of any patent filed on the licensed rights that covers a licensed product, (iii) the expiration of any applicable marketing exclusivity right and (iv) such time that neither the Company nor any sublicensees, partners or contractors are commercializing a licensed product. Either the Company or the rights holders acting together may terminate the license agreement if the other such party commits a material breach and fails to cure such breach within a certain period of time, or if the other party enters into liquidation, becomes insolvent, or enters into composition or statutory reorganization proceedings. No upfront fees related to the license were recorded for the six months ended June 30, 2018 and 2017. |
Net Loss per Share
Net Loss per Share | 6 Months Ended |
Jun. 30, 2018 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | 12. Net Loss per Share For purposes of the diluted net loss per share calculation, stock options, unvested restricted stock, the warrant to purchase shares of Series A Preferred Stock and Preferred Stock were considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, the weighted-average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The following potentially dilutive common stock equivalents, presented based on amounts outstanding at each period end, were excluded 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: Six Months Ended June 30, 2018 2017 Options to purchase common stock 1,871,139 796,311 Restricted common stock 214,789 368,062 Redeemable convertible preferred stock (as converted to common stock) — 4,155,742 Warrants to purchase redeemable convertible preferred stock (as converted to common stock) — 6,850 Warrants to purchase common stock 6,850 — |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2018 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 13. Commitments and Contingencies Lease Agreements On January 12, 2018, the Company entered into a lease agreement for office space located in Cambridge, Massachusetts. The lease agreement expires in January 2023, with a landlord who is an affiliate of the landlord of the Company’s prior lease facility. The annual lease payments are subject to a 3% increase each year. The Company recognizes rent expense on a straight-line basis over the lease period and has recorded deferred rent for rent expense incurred but not yet paid. The Company received a tenant incentive allowance of $842 in 2018. Such incentive allowance is being amortized as a reduction of rent expense on a straight-line basis over the lease period. In accordance with the lease agreement, the Company is required to maintain a security deposit of $209, which was recorded in other assets. In contemplation of this agreement, the Company terminated its prior lease agreement. Legal Proceedings The Company, from time to time, may be party to litigation arising in the ordinary course of business. The Company was not subject to any material legal proceedings during the six months ended June 30, 2018 and 2017, and, to the best of its knowledge, no material legal proceedings are currently pending or threatened. Other The Company is also party to various agreements, principally relating to licensed technology, that require future payments relating to milestones not met at June 30, 2018 and December 31, 2017, or royalties on future sales of specified products. No milestone or royalty payments under these agreements are expected to be payable in the immediate future. The Company enters into standard indemnification agreements in the ordinary course of business. Pursuant to the agreements, the Company agrees to indemnify, hold harmless, and to reimburse the indemnified party for losses suffered or incurred by the indemnified party, generally the Company’s business partners, in connection with any U.S. patent or any copyright or other intellectual property infringement claim by any third-party with respect to the Company’s products. The term of these indemnification agreements is generally perpetual any time after execution of the agreement. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is unlimited. The Company has never incurred costs to defend lawsuits or settle claims related to these indemnification agreements. |
Related Party Transactions
Related Party Transactions | 6 Months Ended |
Jun. 30, 2018 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 14. Related Party Transactions UHN In connection with the Company’s entry into a license agreement with UHN on January 27, 2016, the Company issued UHN 1,161,665 shares of its common stock. As a result of the issuance of common stock, UHN owned 4.64% and 9.65% of the Company’s fully diluted equity as of June 30, 2018 and December 31, 2017, respectively. Upon the closing of the IPO, as UHN’s fully-diluted percentage ownership of the Company was reduced within a range of specified percentages, the Company is obligated to pay UHN an amount up to $2,000, which was recorded in accrued expenses as of June 30, 2018. See Note 3 for further discussion on the accounting treatment for this provision. During the six months ended June 30, 2018 and 2017, the Company recognized $41 and $151 (nil for three months ended June 30, 2018 and 2017), respectively, of research and development expense related to the license agreements with UHN. For the six months ended June 30, 2018 and 2017 the Company recorded expenses of $40 and $48 ($2 and $24 for the three months ended June 30, 2018 and 2017), respectively, related to consulting services provided by an entity affiliated with an officer of the Company and a member of the Board. The entity is also a shareholder of the Company and owned 0.48% and 1.56% of the Company’s fully diluted equity as of June 30, 2018 and 2017, respectively. Others For the three and six months ended June 30, 2017, the Company recorded expenses of $15 and $15, respectively, related to services provided by an entity affiliated with a member of the Board and the use of office space. The lease was terminated in February 2017 and the services were no longer being provided as of June 30, 2017. |
Subsequent Events
Subsequent Events | 6 Months Ended |
Jun. 30, 2018 | |
Subsequent Events [Abstract] | |
Subsequent Events | 15. Subsequent Events On July 13, 2018, the lender of the Loan Agreement exercised its common stock warrants to purchase 6,850 shares of common stock on a net basis, resulting in the issuance of 6,091 shares of common stock. |
Nature of the Business and Ba21
Nature of the Business and Basis of Presentation (Policies) | 6 Months Ended |
Jun. 30, 2018 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Basis of presentation | Basis of Presentation The accompanying condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification (ASC) and Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB). The unaudited condensed consolidated interim financial statements have been prepared on the same basis as the audited annual consolidated financial statements as of and for the year ended December 31, 2017, and, in the opinion of management, reflect all adjustments, consisting of normal recurring adjustments, necessary for the fair presentation of the Company’s financial position as of June 30, 2018, and the results of its operations and its cash flows for the three and six months ended June 30, 2018 and 2017. The results for the six months ended June 30, 2018 are not necessarily indicative of the results to be expected for the year ending December 31, 2018, any other interim periods, or any future year or period. These interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2017, and the notes thereto, which are included elsewhere in the Company’s final prospectus for its initial public offering (“IPO”), filed with the Securities and Exchange Commission (“SEC”) pursuant to Rule 424(b)(4) under the Securities Act of 1933, as amended, on June 21, 2018 (the “Prospectus”). The accompanying condensed financial statements reflect the application of certain significant accounting policies as described below and elsewhere in these notes to the financial statements. As of June 30, 2018, the Company’s significant accounting policies and estimates, which are detailed in the Company’s Prospectus, have not changed. |
Reverse Stock Split | Reverse Stock Split On June 1, 2018, the Board approved a 1-for-4.312 |
Subsequent Event Considerations | Subsequent Event Considerations The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. See Note 15. |
Use of Estimates | Use of Estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, the accrual for research and development expenses, stock-based compensation expense, the valuation of equity and derivative instruments and the recoverability of the Company’s net deferred tax assets and related valuation allowance. 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 materially from those estimates. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in interest-bearing money market accounts. Cash equivalents are carried at cost, which approximates their fair market value. |
Concentrations of Credit Risk | Concentrations of Credit Risk The Company has no significant off-balance |
Fair Value Measurements | Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP (see Note 3). Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1—Quoted prices in active markets for identical assets or liabilities. • Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3—Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. |
Segment Information | Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker, or decision making group, in making decisions on how to allocate resources and assess performance. The Company’s chief operating decision maker is the chief executive officer (“CEO”). The Company and the CEO view the Company’s operations and manage its business as one operating segment. All material long-lived assets of the Company reside in the United States. |
Research and Development Costs | Research and Development Costs Research and development costs are expensed as incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries, stock-based compensation and benefits, facilities costs, depreciation, third-party license fees, and external costs of outside vendors engaged to conduct preclinical development activities and clinical trials as well as to manufacture research and development materials. Non-refundable The Company has entered into various research and development related contracts with parties both inside and outside of the United States. The payments to these agreements are recorded as research and development expenses as incurred. The Company records accrued liabilities for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs. |
Stock-Based Compensation | Stock-Based Compensation For stock-based awards issued to employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of grant and recognizes compensation expense for those awards over the requisite service period, which is generally the vesting period of the respective award. The Company issues stock-based awards with only service-based vesting conditions and records the expense for these awards using the straight-line method. The Company has not issued any stock-based awards with performance- or market-based vesting conditions. The Company accounts for forfeitures as they occur. Stock-based awards issued to non-employees non-employees The Company classifies stock-based compensation expense in its consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s cash compensation costs are classified. Given the absence of an active market for the Company’s common stock prior to the IPO, the Company and the Board, the members of which the Company believes have extensive business, finance, and venture capital experience, were required to estimate the fair value of the Company’s common stock at the time of each grant of a stock-based award. The Company and the Board determined the estimated fair value of the Company’s equity instruments based on a number of factors, including external market conditions affecting the biotechnology industry sector. The Company and the Board utilized various valuation methodologies in accordance with the framework of the American Institute of Certified Public Accountants’ Technical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation arm’s-length The fair value of each stock option award is estimated on the date of grant using the Black-Scholes option pricing model. As there was no public market for its common stock prior to June 21, 2018, which was the first day of trading, and as the trading history of the Company’s common stock was limited through June 30, 2018, the Company determined the volatility for awards granted based on an analysis of reported data for a group of guideline companies that issued options with substantially similar terms. The expected volatility has been determined using a weighted-average of the historical volatility measures of this group of guideline companies. The Company expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded stock price. The expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. The Company has not paid, and does not anticipate paying, cash dividends on its common stock; therefore, the expected dividend yield is assumed to be zero. See Note 10 for the assumptions used by the Company in determining the grant date fair value of stock-based awards granted, as well as a summary of the stock-based award activity under the Company’s stock-based compensation plan for the six months ended June 30, 2018. |
Warrant to Purchase Preferred Stock | Warrant to Purchase Preferred Stock Prior to the IPO, the Company classified the warrant for the purchase of shares of its redeemable convertible preferred stock (see Note 7) as a liability on its consolidated balance sheets as the warrant is a free-standing financial instrument that may require the Company to transfer assets upon exercise. The preferred stock warrant liability was initially recorded at fair value upon the date of issuance and is subsequently remeasured to fair value at each reporting date. Changes in the fair value of the warrant to purchase preferred stock are recognized as a component of other income (expense), net in the consolidated statements of operations and comprehensive loss. Changes in the fair value of the warrant to purchase preferred stock will continue to be recognized until the warrant is exercised, expires or qualifies for equity classification. The Company utilizes the Black-Scholes option-pricing model, which incorporates assumptions and estimates, to value the warrant. The Company assesses these assumptions and estimates on a quarterly basis as additional information impacting the assumptions is obtained. Estimates and assumptions impacting the fair value measurement include the fair value per share of the underlying redeemable convertible preferred stock issuable upon exercise of the warrant, the remaining contractual term of the warrant, the risk-free interest rate, the expected dividend yield and the expected volatility of the price of the underlying redeemable convertible preferred stock. Upon the IPO, the warrant to purchase preferred stock was converted to a warrant to purchase common stock. The carrying amount of warrant to purchase preferred stock as of the date of IPO was transferred to the account of additional paid in capital. |
Comprehensive Income (Loss) | Comprehensive Income (Loss) Comprehensive income (loss) is defined as the change in stockholders’ equity of a business enterprise during a period from transactions and other events and circumstances from non-owner |
Leases | Leases The Company categorizes leases at their inception as either operating or capital leases. On certain lease arrangements, the Company may receive rent holidays or other incentives. The Company recognizes lease costs on a straight-line basis once control of the space is achieved, without regard to deferred payment terms, such as rent holidays, that defer the commencement date of required payments or escalating payment amounts. The difference between required lease payments and rent expense has been recorded as deferred rent and other accrued expenses and other current liabilities in the accompanying consolidated balance sheets. Additionally, incentives received are treated as a reduction of costs over the term of the agreement, as they are considered an inseparable part of the lease agreement. |
Net Income (Loss) per Share Attributable to Common Stockholders | Net Income (Loss) per Share Attributable to Common Stockholders Net income (loss) per share attributable to common stockholders is determined using the two-class Under the two-class two-class if-converted |
Emerging Growth Company Status | Emerging Growth Company Status The Company is an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act, or JOBS Act, and may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies. The Company may take advantage of these exemptions until the Company is no longer an “emerging growth company.” Section 107 of the JOBS Act provides that an “emerging growth company” can take advantage of the extended transition period afforded by the JOBS Act for the implementation of new or revised accounting standards. The Company has elected to use the extended transition period for complying with new or revised accounting standards and as a result of this election, its consolidated financial statements may not be comparable to companies that comply with public company effective dates. The Company may take advantage of these exemptions up until the last day of the fiscal year following the fifth anniversary of an offering or such earlier time that it is no longer an “emerging growth company”. |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In June 2018, the FASB issued ASU No. 2018-07, “Compensation—Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting” non-employees 505-50. 505-50 non-employee non-employees, non-employee non-employee non-public 2018-07 In July 2017, the FASB issued ASU 2017-11, Earnings Per Share (Topic 260), Distinguishing Liabilities from Equity (Topic 480), Derivatives and Hedging (Topic 815) I. Accounting for Certain Financial Instruments with Down Round Features II. Replacement of the Indefinite Deferral for Mandatorily Redeemable Financial Instruments of Certain Nonpublic Entities and Certain Mandatorily Redeemable Noncontrolling Interests with a Scope Exception . 2017-11 In May 2017, the FASB issued ASU No. 2017-09, Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting 2017-09”), 2017-09 In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash 2016-18”), 2016-18 In August 2016, the FASB issued ASU No. 2016-15, Statement of Cash Flows: Classification of Certain Cash Receipts and Cash Payments 2016-15”), 2016-15 In February 2016, the FASB issued ASU No. 2016-02 , Leases (Topic 842) 2016-02”), right-of-use 2016-02 Leases not-for-profit In September 2017, the FASB issued ASU No. 2017-13, Revenue Recognition (Topic 605), Revenue from Contracts with Customers (Topic 606), Leases (Topic 840), and Leases (Topic 842) ASU 2016-02 2016-02. ASU 2016-02 |
Fair Value of Financial Asset22
Fair Value of Financial Assets and Liabilities (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Summary of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis | The following table presents information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values as of June 30, 2018 and December 31, 2017, respectively: Fair Value Measurements as of June 30, 2018 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 154,549 $ — $ — $ 154,549 Fair Value Measurements as of December 31, 2017 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ 5,684 $ — $ — $ 5,684 Restricted cash 24 — — 24 $ 5,708 $ — $ — $ 5,708 Liabilities: Derivative liability $ — $ — $ 371 $ 371 Warrant to purchase redeemable convertible preferred stock — — 35 35 $ — $ — $ 406 $ 406 |
Warrants to Purchase Redeemable Convertible Preferred Stock [Member] | |
Summary of Changes in Fair Value Determined by Level 3 Inputs | The following table sets forth a summary of changes in the fair value of the Company’s preferred stock warrant liability for which fair value was determined by Level 3 inputs: Warrant Liability Balance as of December 31, 2017 $ 35 Change in fair value 162 Conversion of preferred stock warrant to common stock warrant (197 ) Balance as of June 30, 2018 $ — |
Derivative Liabilities [Member] | |
Summary of Changes in Fair Value Determined by Level 3 Inputs | The following table sets forth a summary of changes in the fair value of the Company’s derivative liability for which fair value is determined by Level 3 inputs: Derivative Liability Balance as of December 31, 2017 $ 371 Change in fair value 1,629 Transferred to accrued expenses (2,000 ) Balance as of June 30, 2018 $ — |
Prepaid Expenses and Other Cu23
Prepaid Expenses and Other Current Assets (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Text Block [Abstract] | |
Summary of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following: June 30, 2018 December 31, 2017 Prepaid development costs $ 762 $ 163 Prepaid rent 76 122 Other current assets 294 60 $ 1,132 $ 345 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Property, Plant and Equipment [Abstract] | |
Summary of Property and Equipment, Net | Property and equipment, net consisted of the following: June 30, 2018 December 31, 2017 Laboratory and office equipment $ 926 $ 126 Leasehold improvements 1,112 240 Computer equipment and software 83 28 2,121 394 Less: Accumulated depreciation and amortization (71 ) (45 ) $ 2,050 $ 349 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Payables and Accruals [Abstract] | |
Summary of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consisted of the following: June 30, 2018 December 31, 2017 Dilution payment (Note 3) $ 2,000 $ — Compensation and benefit costs 846 794 Short-term deferred rent 171 9 Research and development costs 753 831 IPO issuance cost 693 — Property and equipment 667 — Other liabilities 308 464 $ 5,438 $ 2,098 |
Redeemable Convertible Prefer26
Redeemable Convertible Preferred Stock (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Text Block [Abstract] | |
Summary of Preferred Stock | As of December 31, 2017, the Preferred Stock consisted of the following: December 31, 2017 Preferred Shares Authorize Preferred Shares Issued and Outstanding Carrying Value Liquidation Preference Common Stock Issuable Upon Conversion Series Seed preferred stock 3,333,333 3,333,333 $ 1,500,000 $ 1,500,000 806,711 Series A preferred stock 31,639,202 31,450,499 25,000,000 25,000,000 7,611,438 34,972,535 34,783,832 $ 26,500,000 $ 26,500,000 8,418,149 |
Common Stock (Tables)
Common Stock (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Federal Home Loan Banks [Abstract] | |
Summary of Common Stock Reserved for Future Issuance | At June 30, 2018 and December 31, 2017, the Company has reserved the following shares of common stock for future issuance: June 30, 2018 December 31, 2017 Shares reserved for Series Seed Preferred Stock outstanding — 806,711 Shares reserved for Series A Preferred Stock outstanding — 7,611,438 Shares reserved for issuance under the 2018 Stock Option and Incentive Plan 533,911 — Shares reserved for issuance under the 2018 Employee Stock Purchase Plan 223,200 — Shares reserved for vesting of restricted stock awards 214,789 276,301 Shares reserved for exercise of outstanding stock options 1,871,139 1,034,961 Shares reserved for issuance under the 2015 Stock Option and Grant Plan — 143,717 Total shares of authorized common stock reserved for future issuance 2,843,039 9,873,128 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Summary of Assumptions that Used to Determine Grant-Date Fair Value of Stock Options Granted to Employees and Members of Board | The assumptions that the Company used to determine the grant-date fair value of stock options granted to employees and members of the Board were as follows, presented on a weighted-average basis: Six Months Ended June 30, 2018 2017 Expected option life (years) 6.06 6.08 Risk-free interest rate 2.73 % 1.90 % Expected volatility 83.86 % 85.50 % Expected dividend yield — % — % |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity for the six months ended June 30, 2018: Number of Options Weighted- Average Exercise Price Weighted- Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2017 1,034,961 $ 0.78 8.94 $ 3,427 Granted 836,178 $ 6.38 9.74 Exercised — Cancelled or forfeited — Outstanding as of June 30, 2018 1,871,139 $ 3.29 9.02 $ 47,228 Exercisable as of June 30, 2018 309,594 $ 0.72 8.13 $ 8,619 Unvested as of June 30, 2018 1,559,125 $ 3.80 9.20 $ 38,609 |
Summary of Restricted Stock Unit Activity | The following table summarizes the Company’s restricted common stock activity for the six months ended June 30, 2018: Number of Shares Weighted-Average Grant Date Fair Value Issued and unvested as of December 31, 2017 276,301 $ 0.42 Vested (61,512 ) 0.42 Forfeited, canceled or expired — — Issued an unvested as of June 30, 2018 214,789 0.42 |
Schedule of Stock-based Compensation Expense | Stock-based compensation expense was allocated as follows: Six Months Ended June 30, 2018 2017 Research and development $ 187 $ 35 General and administrative 379 29 Total stock-based compensation expense $ 566 $ 64 |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 6 Months Ended |
Jun. 30, 2018 | |
Earnings Per Share [Abstract] | |
Schedule of Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share | The following potentially dilutive common stock equivalents, presented based on amounts outstanding at each period end, were excluded 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: Six Months Ended June 30, 2018 2017 Options to purchase common stock 1,871,139 796,311 Restricted common stock 214,789 368,062 Redeemable convertible preferred stock (as converted to common stock) — 4,155,742 Warrants to purchase redeemable convertible preferred stock (as converted to common stock) — 6,850 Warrants to purchase common stock 6,850 — |
Nature of the Business and Ba30
Nature of the Business and Basis of Presentation - Additional Information (Detail) $ / shares in Units, $ in Thousands | Jun. 25, 2018USD ($)shares | Jun. 20, 2018USD ($)$ / sharesshares | Jun. 01, 2018 | Jun. 30, 2018USD ($)shares |
Nature Of Organization And Operations [Line Items] | ||||
Reverse stock split ratio | 0.2420135 | |||
Net proceeds from IPO, after deducting underwriting discounts and commissions and other offering expenses | $ 105,423 | |||
IPO [Member] | ||||
Nature Of Organization And Operations [Line Items] | ||||
Common shares issued and sold in initial public offering | shares | 787,193 | 5,247,958 | ||
Public offering price | $ / shares | $ 19 | |||
Net proceeds from IPO, after deducting underwriting discounts and commissions and other offering expenses | $ 13,910 | $ 90,271 | $ 104,182 | |
Underwriting discounts and commissions | $ 1,047 | 6,980 | ||
Initial public offering expenses | $ 2,459 | |||
Preferred stock converted into common stock | shares | 15,320,213 |
Summary of Significant Accoun31
Summary of Significant Accounting Policies - Additional Information (Detail) | Jun. 30, 2018 |
Expected Dividend Yield [Member] | |
Significant Of Accounting Policies [Line Items] | |
Expected cash dividend yield | 0 |
Fair Value of Financial Asset32
Fair Value of Financial Assets and Liabilities - Summary of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - Fair Value on Recurring Basis [Member] - USD ($) | Jun. 30, 2018 | Dec. 31, 2017 |
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 5,708 | |
Liabilities Fair Value Disclosure | ||
Liabilities Fair Value Disclosure | 406 | |
Money Market Funds [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 154,549 | 5,684 |
Restricted Cash [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 24 | |
Derivative Liabilities [Member] | ||
Liabilities Fair Value Disclosure | ||
Liabilities Fair Value Disclosure | 371 | |
Warrants to Purchase Redeemable Convertible Preferred Stock [Member] | ||
Liabilities Fair Value Disclosure | ||
Liabilities Fair Value Disclosure | 35 | |
Level 1 [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 5,708 | |
Level 1 [Member] | Money Market Funds [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | $ 154,549 | 5,684 |
Level 1 [Member] | Restricted Cash [Member] | ||
Assets Fair Value Disclosure | ||
Assets Fair Value Disclosure | 24 | |
Level 3 [Member] | ||
Liabilities Fair Value Disclosure | ||
Liabilities Fair Value Disclosure | 406 | |
Level 3 [Member] | Derivative Liabilities [Member] | ||
Liabilities Fair Value Disclosure | ||
Liabilities Fair Value Disclosure | 371 | |
Level 3 [Member] | Warrants to Purchase Redeemable Convertible Preferred Stock [Member] | ||
Liabilities Fair Value Disclosure | ||
Liabilities Fair Value Disclosure | $ 35 |
Fair Value of Financial Asset33
Fair Value of Financial Assets and Liabilities - Additional Information (Detail) - USD ($) | Jan. 27, 2016 | Jun. 30, 2018 | Jun. 30, 2017 | Jun. 30, 2018 | Jun. 30, 2017 | Jul. 31, 2018 |
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||||
Fair value input transfer between levels | $ 0 | $ 0 | $ 0 | $ 0 | ||
Increase in fair value of derivative | 1,042,000 | $ 3,000 | 1,629,000 | $ 35,000 | ||
Dilution payment | $ 2,000,000 | $ 2,000,000 | ||||
IPO [Member] | Subsequent Event [Member] | ||||||
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||||
Dilution payment | $ 2,000,000 | |||||
License Agreement [Member] | ||||||
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||||
Provision for maximum cash payment | $ 2,000,000 | |||||
License Agreement [Member] | Research and Development Expense [Member] | ||||||
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||||
Initial fair value of the derivative | $ 49,000 | |||||
License Agreement [Member] | Stock Purchase Agreement [Member] | ||||||
Fair Value of Assets and Liabilities Measured on Non Recurring Basis [Line Items] | ||||||
Common stock issued for initial consideration for license | 1,161,665 |
Fair Value of Financial Asset34
Fair Value of Financial Assets and Liabilities - Summary of Changes in Fair Value of Preferred Stock Warrant Liability for Fair Value Determined by Level 3 Inputs (Detail) - Level 3 [Member] - Warrants to Purchase Redeemable Convertible Preferred Stock [Member] $ in Thousands | 6 Months Ended |
Jun. 30, 2018USD ($) | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning balance | $ 35 |
Change in fair value | 162 |
Conversion of preferred stock warrant to common stock warrant | $ (197) |
Fair Value of Financial Asset35
Fair Value of Financial Assets and Liabilities - Summary of Changes in Fair Value of Derivative Liability for Fair Value Determined by Level 3 Inputs (Detail) - Level 3 [Member] - Derivative Liabilities [Member] $ in Thousands | 6 Months Ended |
Jun. 30, 2018USD ($) | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning balance | $ 371 |
Change in fair value | 1,629 |
Transferred to accrued expenses | $ (2,000) |
Prepaid Expenses and Other Cu36
Prepaid Expenses and Other Current Assets - Summary of Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands | Jun. 30, 2018 | Dec. 31, 2017 |
Prepaid Expense and Other Assets, Current [Abstract] | ||
Prepaid development costs | $ 762 | $ 163 |
Prepaid rent | 76 | 122 |
Other current assets | 294 | 60 |
Total | $ 1,132 | $ 345 |
Property and Equipment, Net - S
Property and Equipment, Net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Jun. 30, 2018 | Dec. 31, 2017 |
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 2,121 | $ 394 |
Less: Accumulated depreciation and amortization | (71) | (45) |
Property plant and equipment, net | 2,050 | 349 |
Laboratory and Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 926 | 126 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | 1,112 | 240 |
Computer Equipment and Software [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property plant and equipment, gross | $ 83 | $ 28 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2018 | Jun. 30, 2017 | Jun. 30, 2018 | Jun. 30, 2017 | |
Property, Plant and Equipment [Abstract] | ||||
Depreciation and amortization expense | $ 45 | $ 13 | $ 66 | $ 15 |
Accrued Expenses - Summary of A
Accrued Expenses - Summary of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Jun. 30, 2018 | Dec. 31, 2017 |
Accrued Liabilities, Current [Abstract] | ||
Dilution payment (Note 3) | $ 2,000 | |
Compensation and benefit costs | 846 | $ 794 |
Short-term deferred rent | 171 | 9 |
Research and development costs | 753 | 831 |
IPO issuance cost | 693 | |
Property and equipment | 667 | |
Other liabilities | 308 | 464 |
Accrued expenses | $ 5,438 | $ 2,098 |
Loan Agreement and Warrant to40
Loan Agreement and Warrant to Purchase Preferred Stock - Additional Information (Detail) - USD ($) | Jun. 23, 2017 | Jun. 30, 2018 | Jun. 30, 2018 |
Class of Warrant or Right [Line Items] | |||
Maximum issuance of term loans | $ 10,000,000 | ||
Available facility | $ 10,000,000 | $ 10,000,000 | |
Loss related to change in fair value of warrant | $ 150,000 | $ 162,000 | |
Prime Rate [Member] | |||
Class of Warrant or Right [Line Items] | |||
Floating interest rate per annum | 3.00% | ||
Series A Preferred Stock [Member] | |||
Class of Warrant or Right [Line Items] | |||
Warrant to purchase shares | 188,702 | ||
Warrant exercise price | $ 0.7949 | ||
Warrant expiration date | Jun. 22, 2027 | Jun. 22, 2027 | |
Shares purchase upon exercise of warrant | 28,305 | ||
Additional shares purchase upon exercise of warrant | 160,397 | ||
Minimum [Member] | |||
Class of Warrant or Right [Line Items] | |||
Floating interest rate per annum | 1.00% |
Redeemable Convertible Prefer41
Redeemable Convertible Preferred Stock - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 6 Months Ended | |||
Jan. 31, 2018 | Jun. 30, 2018 | Jun. 30, 2017 | Jun. 20, 2018 | Dec. 31, 2017 | |
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 63,491,857 | 34,972,535 | |||
Redeemable convertible preferred stock, par value | $ 0.0001 | ||||
Redeemable convertible preferred stock, shares issued and sold | 34,783,832 | ||||
Proceeds from issuance of redeemable convertible preferred stock | $ 58,258 | $ 3,468 | |||
IPO [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, sales price per share | $ 19 | ||||
Preferred stock converted into common stock | 15,320,213 | ||||
Series Seed Redeemable Convertible Preferred Stock [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 3,333,333 | 3,333,333 | |||
Redeemable convertible preferred stock, shares issued and sold | 3,333,333 | ||||
Series A Redeemable Convertible Preferred Stock [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 31,639,202 | 31,639,202 | |||
Redeemable convertible preferred stock, shares issued and sold | 31,450,499 | ||||
Series B Redeemable Convertible Preferred Stock [Member] | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, shares authorized | 28,519,322 | ||||
Redeemable convertible preferred stock, shares issued and sold | 28,519,322 | ||||
Redeemable convertible preferred stock, sales price per share | $ 2.1389 | ||||
Proceeds from issuance of redeemable convertible preferred stock | $ 58,757 | ||||
Redeemable convertible preferred stock, net of issuance costs | $ 2,243 |
Redeemable Convertible Prefer42
Redeemable Convertible Preferred Stock - Summary of Preferred Stock (Detail) - USD ($) | Jun. 30, 2018 | Dec. 31, 2017 |
Temporary Equity [Line Items] | ||
Preferred Shares Authorized | 63,491,857 | 34,972,535 |
Preferred Shares Issued | 34,783,832 | |
Preferred Shares Outstanding | 34,783,832 | |
Carrying Value | $ 26,500,000 | |
Liquidation Preference | $ 26,500,000 | |
Common Stock Issuable Upon Conversion | 8,418,149 | |
Series Seed Redeemable Convertible Preferred Stock [Member] | ||
Temporary Equity [Line Items] | ||
Preferred Shares Authorized | 3,333,333 | 3,333,333 |
Preferred Shares Issued | 3,333,333 | |
Preferred Shares Outstanding | 3,333,333 | |
Carrying Value | $ 1,500,000 | |
Liquidation Preference | $ 1,500,000 | |
Common Stock Issuable Upon Conversion | 806,711 | |
Series A Redeemable Convertible Preferred Stock [Member] | ||
Temporary Equity [Line Items] | ||
Preferred Shares Authorized | 31,639,202 | 31,639,202 |
Preferred Shares Issued | 31,450,499 | |
Preferred Shares Outstanding | 31,450,499 | |
Carrying Value | $ 25,000,000 | |
Liquidation Preference | $ 25,000,000 | |
Common Stock Issuable Upon Conversion | 7,611,438 |
Common Stock - Additional Infor
Common Stock - Additional Information (Detail) - $ / shares | Jun. 30, 2018 | Dec. 31, 2017 |
Equity [Abstract] | ||
Common stock, shares authorized | 150,000,000 | 51,000,000 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common Stock - Summary of Commo
Common Stock - Summary of Common Stock Reserved for Future Issuance (Detail) - shares | Jun. 30, 2018 | Dec. 31, 2017 |
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 2,843,039 | 9,873,128 |
Series Seed Redeemable Convertible Preferred Stock [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 806,711 | |
Series A Redeemable Convertible Preferred Stock [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 7,611,438 | |
2018 Stock Option and Incentive Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 533,911 | |
2018 Employee Stock Purchase Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 223,200 | |
Restricted Stock [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 214,789 | 276,301 |
Employee Stock Option [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 1,871,139 | 1,034,961 |
2015 Stock Option and Grant Plan [Member] | ||
Class of Stock [Line Items] | ||
Total shares of authorized common stock reserved for future issuance | 143,717 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 6 Months Ended | |
Jun. 30, 2018 | Jun. 30, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of options granted | 836,178 | |
Options Granted, Weighted-average grant-date fair value | $ 7.63 | $ 0.66 |
Total fair value of restricted common stock vested | $ 26 | $ 17 |
Unrecognized stock-based compensation expenses | $ 6,776 | |
Unrecognized stock-based compensation expense, period for recognition | 3 years 7 months 10 days | |
2018 Stock Option and Incentive Plan [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of common shares that may be issued under the plan | 616,300 | |
Common shares available for future grant | 533,911 | |
Number of shares of common stock outstanding, increase, percentage | 4.00% | |
2015 Stock Option and Grant Plan [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of common shares that may be issued under the plan | 2,008,564 | |
Common shares available for future grant | 0 | |
Option vesting period | 4 years | |
Number of options granted | 836,178 | 196,872 |
2018 Employee Stock Purchase Plan [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of common shares that may be issued under the plan | 223,200 | |
Common shares available for future grant | 223,200 | |
Employee Stock Purchase Plan, description | The ESPP provides that the number of shares reserved and available for issuance will automatically increase each January 1, beginning on January 1, 2019 and each January 1 thereafter through January 1, 2028, by the least of (i) 1% of the outstanding number of shares of our common stock on the immediately preceding December 31; (ii) 1,115,700 shares or (iii) such number of shares as determined by the ESPP administrator. | |
2018 Employee Stock Purchase Plan [Member] | Maximum [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of common shares that may be issued under the plan | 223,200 | |
Number of shares of common stock outstanding, increase, percentage | 1.00% | |
Number of common shares that may increase under the plan | 1,115,700 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Assumptions that Used to Determine Grant-Date Fair Value of Stock Options Granted to Employees and Members of Board (Detail) | 6 Months Ended | |
Jun. 30, 2018 | Jun. 30, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award, Fair Value Assumptions and Methodology [Abstract] | ||
Expected option life (years) | 6 years 22 days | 6 years 29 days |
Risk-free interest rate | 2.73% | 1.90% |
Expected volatility | 83.86% | 85.50% |
Expected dividend yield | 0.00% | 0.00% |
Stock-Based Compensation - Su47
Stock-Based Compensation - Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward] | ||
Number of options, Outstanding beginning balance | 1,034,961 | |
Number of options, Granted | 836,178 | |
Number of options, Exercised | 0 | |
Number of options, Cancelled or forfeited | 0 | |
Number of options, Outstanding ending balance | 1,871,139 | 1,034,961 |
Number of options, Exercisable | 309,594 | |
Number of options, Unvested | 1,559,125 | |
Weighted average exercise price, Outstanding beginning balance | $ 0.78 | |
Weighted average exercise price, Granted | 6.38 | |
Weighted average exercise price, Exercised | 0 | |
Weighted average exercise price, Cancelled or forfeited | 0 | |
Weighted average exercise price, Outstanding ending balance | 3.29 | $ 0.78 |
Weighted average exercise price, Exercisable | 0.72 | |
Weighted average exercise price, Unvested | $ 3.80 | |
Weighted average remaining contractual term, Granted | 9 years 8 months 26 days | |
Weighted average remaining contractual term, Outstanding balance | 9 years 7 days | 8 years 11 months 8 days |
Weighted average remaining contractual term, Exercisable | 8 years 1 month 16 days | |
Weighted average remaining contractual term, Unvested | 9 years 2 months 12 days | |
Aggregate intrinsic value, Outstanding balance | $ 47,228 | $ 3,427 |
Aggregate intrinsic value, Exercisable | 8,619 | |
Aggregate intrinsic value, Unvested | $ 38,609 |
Stock-Based Compensation - Su48
Stock-Based Compensation - Summary of Restricted Stock Unit Activity (Detail) | 6 Months Ended |
Jun. 30, 2018$ / sharesshares | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Number of shares, Issued and unvested beginning balance | 276,301 |
Number of shares, Vested | (61,512) |
Number of shares, Forfeited, cancelled or expired | 0 |
Number of shares, Issued and unvested ending balance | 214,789 |
Weighted average grant date fair value, Issued and unvested, beginning balance | $ / shares | $ 0.42 |
Weighted average grant date fair value, Vested | $ / shares | 0.42 |
Weighted average grant date fair value, Issued and unvested, ending balance | $ / shares | $ 0.42 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Stock-based Compensation Expense (Detail) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2018 | Jun. 30, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock based compensation expense | $ 566 | $ 64 |
Research and Development Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock based compensation expense | 187 | 35 |
General and Administrative Expense [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Total stock based compensation expense | $ 379 | $ 29 |
License Agreements - Additional
License Agreements - Additional Information (Detail) $ in Thousands | Oct. 02, 2017USD ($) | Nov. 17, 2016USD ($) | Jan. 27, 2016USD ($)shares | Jan. 27, 2016CAD ($) | Aug. 31, 2017USD ($)shares | Jun. 30, 2018USD ($)shares | Jun. 30, 2017USD ($) | Jun. 30, 2018USD ($)shares | Jun. 30, 2017USD ($) | Dec. 31, 2017USD ($)shares | Jan. 27, 2016CAD ($)shares |
License Agreement [Line Items] | |||||||||||
Upfront license fee | $ 0 | $ 0 | |||||||||
Upfront fee | 0 | 0 | |||||||||
Annual maintenance fees | $ 0 | 0 | |||||||||
Common stock, shares issued | shares | 23,936,838 | 23,936,838 | 2,581,474 | ||||||||
Research and development | $ 7,407,000 | $ 1,881,000 | $ 13,054,000 | 3,315,000 | |||||||
UHN Agreement [Member] | Fabry License Agreement [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Option fee | $ 20 | ||||||||||
Upfront license fee | $ 75 | ||||||||||
Milestone payments | 2,450 | ||||||||||
UHN Agreement [Member] | Interleukin 12 License Agreement [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Upfront license fee | 264 | ||||||||||
Milestone payments | 19,275 | ||||||||||
Annual maintenance fees | $ 50 | ||||||||||
Common stock, shares issued | shares | 1,161,665 | 1,161,665 | |||||||||
Fair value of shares issued | $ 480 | ||||||||||
Payments upon closing of an initial public offering | 2,000,000 | ||||||||||
Fair value of derivative liability | $ 49,000 | ||||||||||
Research and development | $ 0 | $ 0 | 41,000 | $ 151,000 | |||||||
BioMarin Pharmaceutical Inc [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Upfront license fee | $ 500,000 | 0 | |||||||||
Milestone payments | 13,000,000 | ||||||||||
Research and development | $ 500,000 | ||||||||||
Upfront cash payment | $ 500,000 | ||||||||||
GenStem Therapeutics Inc [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Upfront license fee | $ 1,000,000 | $ 0 | |||||||||
Milestone payments | $ 16,000,000 | ||||||||||
Lund University Rights Holders Agreement [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Milestone payments | $ 550,000 | ||||||||||
Series B Preferred Stock [Member] | BioMarin Pharmaceutical Inc [Member] | |||||||||||
License Agreement [Line Items] | |||||||||||
Preferred stock issued | shares | 233,765 |
Net Loss per Share - Schedule o
Net Loss per Share - Schedule of Dilutive Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Detail) - shares | 6 Months Ended | |
Jun. 30, 2018 | Jun. 30, 2017 | |
Employee Stock Option [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 1,871,139 | 796,311 |
Restricted Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 214,789 | 368,062 |
Warrant [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 6,850 | |
Common Stock Warrant [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 6,850 | |
Redeemable Convertible Preferred Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share amount | 4,155,742 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Thousands | Jan. 12, 2018 | Jun. 30, 2018 |
Commitments and Contingencies Disclosure [Abstract] | ||
Lease agreement expiration date | Jan. 31, 2023 | Feb. 28, 2017 |
Percentage of annual increase in rent | 3.00% | |
Tenant incentive allowance | $ 842 | |
Security deposit in connection with lease | $ 209 |
Related Party Transactions - Ad
Related Party Transactions - Additional information (Detail) - USD ($) | Jan. 12, 2018 | Jan. 27, 2016 | Jun. 30, 2018 | Jun. 30, 2017 | Jun. 30, 2018 | Jun. 30, 2017 | Dec. 31, 2017 |
Related Party Transaction [Line Items] | |||||||
Research and development expense related to license agreements | $ 7,407,000 | $ 1,881,000 | $ 13,054,000 | $ 3,315,000 | |||
Lease termination date | Jan. 31, 2023 | Feb. 28, 2017 | |||||
Officers and Board Members [Member] | Consulting Services Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Issuance of common stock ownership percentage | 0.48% | 1.56% | 0.48% | 1.56% | |||
Expense related to related party | $ 2,000 | $ 24,000 | $ 40,000 | $ 48,000 | |||
Board Member [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Expense related to related party | 15,000 | 15,000 | |||||
License Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Provision for maximum cash payment | $ 2,000,000 | ||||||
Research and development expense related to license agreements | $ 0 | $ 0 | 41,000 | $ 151,000 | |||
License Agreement [Member] | Accrued Expenses [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Provision for maximum cash payment | $ 2,000,000 | ||||||
License Agreement [Member] | Stock Purchase Agreement [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Common stock issued for initial consideration for license | 1,161,665 | ||||||
Issuance of common stock ownership percentage | 4.64% | 4.64% | 9.65% |
Subsequent Events - Additional
Subsequent Events - Additional Information (Detail) - shares | Jul. 13, 2018 | Jun. 30, 2018 | Dec. 31, 2017 |
Subsequent Event [Line Items] | |||
Warrants to purchase common stock | 150,000,000 | 51,000,000 | |
Warrants to purchase common stock, issued | 23,936,838 | 2,581,474 | |
Warrant [Member] | Subsequent Event [Member] | |||
Subsequent Event [Line Items] | |||
Warrants to purchase common stock | 6,850 | ||
Warrants to purchase common stock, issued | 6,091 |