Cover
Cover - USD ($) | 12 Months Ended | ||
Mar. 31, 2021 | Jun. 07, 2021 | Sep. 30, 2020 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Mar. 31, 2021 | ||
Current Fiscal Year End Date | --03-31 | ||
Document Transition Report | false | ||
Entity File Number | 001-37418 | ||
Entity Registrant Name | Sio Gene Therapies Inc. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 85-3863315 | ||
Entity Address, Address Line One | 130 West 42nd St. | ||
Entity Address, Address Line Two | 26th Floor | ||
Entity Address, City or Town | New York | ||
Entity Address, State or Province | NY | ||
Entity Address, Postal Zip Code | 10036 | ||
City Area Code | 877 | ||
Local Phone Number | 746-4891 | ||
Title of 12(b) Security | Common Stock, par value $0.00001 per share | ||
Trading Symbol | SIOX | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Interactive Data Current | Yes | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | false | ||
Entity Shell Company | false | ||
Entity Public Float | $ 131,913,331 | ||
Entity Common Stock, Shares Outstanding | 69,558,434 | ||
Entity Central Index Key | 0001636050 | ||
Amendment Flag | false | ||
Document Fiscal Year Focus | 2021 | ||
Document Fiscal Period Focus | FY |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2021 | Mar. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 118,986 | $ 80,752 |
Receivable from sale of long-term investment | 4,343 | 0 |
Prepaid expenses and other current assets | 7,348 | 2,971 |
Income tax receivable | 1,656 | 1,707 |
Total current assets | 132,333 | 85,430 |
Long-term investment | 0 | 5,871 |
Other non-current assets | 0 | 46 |
Long-term restricted cash | 1,184 | 0 |
Operating lease right-of-use assets | 1,152 | 1,532 |
Property and equipment, net | 478 | 801 |
Total assets | 135,147 | 93,680 |
Current liabilities: | ||
Accounts payable | 1,341 | 4,412 |
Accrued expenses | 9,196 | 11,319 |
Current portion of operating lease liabilities | 311 | 889 |
Current portion of long-term debt | 0 | 15,423 |
Total current liabilities | 10,848 | 32,043 |
Operating lease liabilities, net of current portion | 932 | 79 |
Total liabilities | 11,780 | 32,122 |
Commitments and contingencies | ||
Shareholders’ equity: | ||
Common stock, par value $0.00001 per share, 1,000,000,000 shares authorized, 69,377,567 and 39,526,299 issued and outstanding at March 31, 2021 and March 31, 2020, respectively | 1 | 0 |
Accumulated other comprehensive income (loss) | 335 | (55) |
Additional paid-in capital | 914,100 | 820,257 |
Accumulated deficit | (791,069) | (758,644) |
Total shareholders’ equity | 123,367 | 61,558 |
Total liabilities and shareholders’ equity | $ 135,147 | $ 93,680 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2021 | Mar. 31, 2020 |
Common stock | ||
Common shares par value (in dollars per share) | $ 0.00001 | $ 0.00001 |
Common shares authorized (in shares) | 1,000,000,000 | 1,000,000,000 |
Common shares issued (in shares) | 69,377,567 | 39,526,299 |
Common shares outstanding (in shares) | 69,377,567 | 39,526,299 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Operating expenses: | ||
Research and development expenses | $ 24,903 | $ 47,110 |
General and administrative expenses | 17,294 | 22,061 |
Total operating expenses | 42,197 | 69,171 |
Interest expense | 799 | 4,377 |
Other income | (10,359) | (1,358) |
Loss before income tax (benefit) expense | (32,637) | (72,190) |
Income tax (benefit) expense | (212) | 438 |
Net loss | $ (32,425) | $ (72,628) |
Net loss per share of common stock — basic and diluted (in dollars per share) | $ (0.62) | $ (2.93) |
Weighted average shares of common stock outstanding — basic and diluted (in shares) | 52,181,398 | 24,812,536 |
Consolidated Statements of Op_2
Consolidated Statements of Operations (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Research and Development Expense | ||
Share-based compensation expense | $ 1,583 | $ 2,772 |
General and Administrative Expense | ||
Share-based compensation expense | $ 2,909 | $ 5,123 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (32,425) | $ (72,628) |
Other comprehensive income (loss): | ||
Foreign currency translation adjustment | 390 | (966) |
Total other comprehensive income (loss) | 390 | (966) |
Comprehensive loss | $ (32,035) | $ (73,594) |
Consolidated Statements of Shar
Consolidated Statements of Shareholders’ Equity - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) |
Opening balance (in shares) at Mar. 31, 2019 | 22,779,891 | ||||
Opening balance at Mar. 31, 2019 | $ 56,213 | $ 0 | $ 741,318 | $ (686,016) | $ 911 |
Increase (Decrease) in Shareholders' Equity (Deficit) | |||||
Shares issued upon exercise of stock options (in shares) | 11,778 | 11,778 | |||
Issuance of shares upon exercise of stock options | $ 86 | 86 | |||
Shares issued upon settlement of restricted stock units (in shares) | 103,294 | ||||
Shares and pre-funded warrants sold in public offering, net of underwriting discounts and commissions and offering expenses (in shares) | 16,631,336 | ||||
Shares and pre-funded warrants sold in public offering, net of underwriting discounts and commissions and offering expenses | 70,810 | 70,810 | |||
Stock-based compensation expense | 7,895 | 7,895 | |||
Capital contribution received from Roivant Sciences, Inc. | 148 | 148 | |||
Foreign currency translation adjustment | (966) | (966) | |||
Net loss | (72,628) | (72,628) | |||
Ending balance (in shares) at Mar. 31, 2020 | 39,526,299 | ||||
Ending balance at Mar. 31, 2020 | $ 61,558 | $ 0 | 820,257 | (758,644) | (55) |
Increase (Decrease) in Shareholders' Equity (Deficit) | |||||
Shares issued upon exercise of stock options (in shares) | 0 | ||||
Shares issued upon settlement of restricted stock units (in shares) | 187,741 | ||||
Shares and pre-funded warrants sold in public offering, net of underwriting discounts and commissions and offering expenses (in shares) | 29,663,527 | ||||
Shares and pre-funded warrants sold in public offering, net of underwriting discounts and commissions and offering expenses | $ 89,231 | $ 1 | 89,230 | ||
Stock-based compensation expense | 4,492 | 4,492 | |||
Capital contribution received from Roivant Sciences, Inc. | 121 | 121 | |||
Foreign currency translation adjustment | 390 | 390 | |||
Net loss | (32,425) | (32,425) | |||
Ending balance (in shares) at Mar. 31, 2021 | 69,377,567 | ||||
Ending balance at Mar. 31, 2021 | $ 123,367 | $ 1 | $ 914,100 | $ (791,069) | $ 335 |
Consolidated Statements of Sh_2
Consolidated Statements of Shareholders’ Equity (Parenthetical) - USD ($) $ in Millions | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Public Offering | ||
Underwriting discounts, commissions, and offering expenses | $ 4 | $ 3.9 |
Consolidated Statement of Cash
Consolidated Statement of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Cash flows from operating activities: | ||
Net loss | $ (32,425) | $ (72,628) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Unrealized foreign currency translation adjustment | 390 | (966) |
Amortization of operating lease right-of-use assets | 1,521 | 1,617 |
Stock-based compensation expense | 4,492 | 7,895 |
Depreciation and non-cash amortization | 945 | 1,477 |
Gains on long-term investment | (11,256) | 0 |
Change in operating lease liabilities | (866) | (1,582) |
Other | 84 | 19 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | (4,377) | 2,892 |
Income tax receivable | 51 | 19 |
Other non-current assets | 46 | 340 |
Accounts payable | (3,071) | 2,710 |
Accrued expenses | (2,123) | (9,266) |
Net cash used in operating activities | (46,589) | (67,473) |
Cash flows from investing activities: | ||
Cash proceeds from sale of long-term investment | 12,784 | 0 |
Purchases of property and equipment | (398) | (255) |
Net cash provided by (used in) investing activities | 12,386 | (255) |
Cash flows from financing activities: | ||
Payments on long-term debt | (15,731) | (29,563) |
Capital contribution received from Roivant Sciences, Inc. | 121 | 148 |
Cash proceeds from stock option exercises | 0 | 86 |
Cash proceeds from issuance of shares of common stock and pre-funded warrants, net of issuance costs | 89,231 | 70,810 |
Net cash provided by financing activities | 73,621 | 41,481 |
Net change in cash and cash equivalents and long-term restricted cash | 39,418 | (26,247) |
Cash and cash equivalents—beginning of year | 80,752 | 106,999 |
Cash and cash equivalents—end of year | 120,170 | 80,752 |
Cash and cash equivalents | 118,986 | 80,752 |
Long-term restricted cash | 1,184 | 0 |
Non-cash operating activities: | ||
Operating lease right-of-use assets recognized upon and since the adoption of ASC 842, Leases, on April 1, 2019 | 1,141 | 3,103 |
Operating lease liabilities recognized upon and since the adoption of ASC 842, Leases, on April 1, 2019 | 1,141 | 2,517 |
Amounts reclassified from other non-current assets to operating lease right-of-use assets upon the adoption of ASC 842, Leases, on April 1, 2019 | 0 | 586 |
Supplemental disclosure of cash paid: | ||
Income taxes | 40 | 562 |
Interest | $ 465 | $ 4,012 |
Description of Business
Description of Business | 12 Months Ended |
Mar. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Description of Business | Description of Business Sio Gene Therapies Inc. ("Sio"), together with its wholly owned subsidiaries (the "Company"), is a clinical-stage company focused on developing gene therapies for neurodegenerative diseases. The Company is developing a pipeline of innovative product candidates for the treatment of these debilitating diseases, including GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs and Sandhoff diseases) and Parkinson's disease. The Company is dedicated to realizing the potential of gene therapies to offer transformative patient outcomes in areas of high unmet medical need. Sio is a Delaware corporation, which was originally an exempted limited company incorporated under the laws of Bermuda in October 2014 and was named Axovant Gene Therapies Ltd. ("AGT") from March 2019 until November 2020. During November 2020, the Company completed a corporate transformation, changing its jurisdiction of incorporation from Bermuda to the State of Delaware, changing its name to Sio Gene Therapies Inc., and changing its ticker symbol on The Nasdaq Global Select Market (“Nasdaq”) to “SIOX” (collectively, these events comprise the “Domestication”). The Company continues to be subject to the reporting requirements of the Securities Exchange Act of 1934, as amended, and applicable rules of Nasdaq. Since its initial public offering in 2015, the Company has devoted substantially all of its efforts to raising capital, acquiring product candidates and advancing its product candidates into clinical development. The Company has determined that it has one operating and reporting segment as it allocates resources and assesses financial performance on a consolidated basis. The Company does not expect to generate revenue unless and until it successfully completes development and obtains regulatory approval for one of its product candidates. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies (A) Basis of Presentation: The Company’s fiscal year ends on March 31, and its fiscal quarters end on June 30, September 30, and December 31. These consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States ("U.S. GAAP"). Any reference in these notes to applicable guidance is meant to refer to the authoritative U.S. GAAP as found in the Accounting Standards Codification ("ASC") and as amended by Accounting Standards Updates ("ASU") issued by the Financial Accounting Standards Board ("FASB"). These consolidated financial statements and accompanying notes include the accounts of the Company and its wholly owned subsidiaries. The Company has no unconsolidated subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Certain prior period balances have been reclassified to conform to the current period presentation. During November 2020, the historical financial statements and subsidiaries of AGT became the historical financial statements and subsidiaries of Sio upon consummation of the Domestication. As a result, these consolidated financial statements and accompanying notes reflect (i) the historical operating results of AGT and its subsidiaries prior to the Domestication; (ii) the operating results of the Company following the Domestication; and (iii) the Company’s equity structure for all periods presented. A 1-for-8 reverse stock split of the Company's outstanding common stock was effected in May 2019 as approved by the Company's Board of Directors and a majority of its shareholders. As such, all references to share and per share amounts in these financial statements and accompanying notes have been retroactively restated to reflect the 1-for-8 reverse stock split, except for the authorized number of shares of the Company's common stock and the par value per share, which were not affected. (B) Going Concern and Management's Plans: The Company assesses and determines its ability to continue as a going concern in accordance with the provisions of ASC Subtopic 205-40, " Presentation of Financial Statements—Going Concern " ("ASC Subtopic 205-40"), which requires the Company to evaluate whether there are conditions or events that raise substantial doubt about its ability to continue as a going concern within one year after the date that its annual and interim consolidated financial statements are issued. Certain additional financial statement disclosures are required if such conditions or events are identified. If and when an entity’s liquidation becomes imminent, financial statements should be prepared under the liquidation basis of accounting. Determining the extent, if any, to which conditions or events raise substantial doubt about the Company’s ability to continue as a going concern, or the extent to which mitigating plans sufficiently alleviate any such substantial doubt, as well as whether or not liquidation is imminent, requires judgment by management. The Company has evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date the consolidated financial statements are issued. The Company is currently a development stage company, and thus, has not yet achieved profitability. The Company expects to continue to incur significant operating and net losses, as well as negative cash flows from operations, for the foreseeable future as it continues to develop its gene therapy product candidates and prepares for potential future regulatory approvals and commercialization of its products. The Company has not generated any revenue to date and does not expect to generate product revenue unless and until it successfully completes development and obtains regulatory approval for at least one of its gene therapy product candidates. The Company's current cash and cash equivalents balance will also not be sufficient to complete all necessary development activities and commercially launch its products. For the years ended March 31, 2021 and March 31, 2020, the Company incurred net losses of $32.4 million and $72.6 million, respectively. As of March 31, 2021, the Company's cash and cash equivalents totaled $119.0 million and its accumulated deficit was $791.1 million. The Company estimates that its significantly increased current cash and cash equivalents balance, which includes cash proceeds received from its sales agreement with SVB Leerink LLC to sell shares of common stock from time to time through an at-the-market equity offering program, as well as cash proceeds received in connection with the sale of its long-term investment in Arvelle Therapeutics B.V. ("Arvelle"), is sufficient to support operations beyond the twelve month period following the date that these consolidated financial statements were issued, including beyond the expected dates of major upcoming milestones for the Company's AXO-AAV-GM1 gene therapy program for the treatment of GM1 gangliosidosis. As such, the Company has determined that there is no longer substantial doubt about its ability to continue as a going concern for the one-year period following the date that these consolidated financial statements and footnotes were issued. These estimates are based on assumptions that may prove to be wrong, and the Company could use its available capital resources sooner than it currently expects. In order to meet the Company's long-term operating requirements, the Company will need, among other things, additional capital resources. The Company continually assesses multiple options to obtain additional funding to support its operations, including proceeds from offerings of its equity securities or debt, or transactions involving product development, technology licensing or collaboration arrangements, or other sources of capital to complete its currently planned development programs. Management can provide no assurances that it can raise a sufficient amount of financing for the Company on favorable terms, if at all. (C) Use of Estimates: The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. The Company regularly evaluates estimates and assumptions related to certain assets and liabilities, including its research and development accruals, as well as assumptions used to estimate the fair value of its stock option awards, estimate its income tax expense and estimate its ability to continue as a going concern. Specifically, the Company’s assessment of the completeness of the information for research and development accruals is subject to variability and uncertainty. In addition, in certain circumstances, the determination of the nature and amount of research and development services that have been received during the reporting period requires judgment as the timing and pattern of vendor invoicing does not correspond to the level of services provided. The Company estimates the grant date fair value of stock option awards with only time-based vesting requirements using a Black-Scholes valuation model and uses a Monte Carlo Simulation method under the income approach to estimate the grant date fair value of stock option awards with market-based performance conditions. The Company bases its estimates and assumptions on historical experience and on various other factors that it believes to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates. Additionally, the Company assessed the impact that the COVID-19 pandemic has had on its operations and financial results as of March 31, 2021 and through the date of issuance of these audited consolidated financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and expenses. (D) Risks and Uncertainties: The Company is subject to risks common to companies in the pharmaceutical industry including, but not limited to, uncertainties related to commercialization of products, regulatory approvals, dependence on key products, dependence on key customers and suppliers, and protection of intellectual property rights. (E) Concentrations of Credit Risk: Financial instruments that potentially subject the Company to concentration of credit risk include cash and cash equivalents. At March 31, 2021, substantially all of the cash balances are deposited in 2 banking institutions, each in excess of insured levels. (F) 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 money market funds. (G) Property and Equipment: Property and equipment, consisting of leasehold improvements, furniture and fixtures, computers, software and other office and laboratory equipment, is recorded at cost. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred. Upon disposal, retirement or sale, the related cost and accumulated depreciation is removed from the accounts and any resulting gain or loss is included in the results of operations. Depreciation is recorded for property and equipment using the straight-line method over the estimated useful lives of the respective assets, generally three The Company reviews the recoverability of all long-lived assets, including the related useful lives, whenever events or changes in circumstances indicate that the carrying amount of a long-lived asset might not be recoverable. Recoverability is measured by comparison of the book values of the assets to future net undiscounted cash flows that the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book value of the assets exceed their fair value, which is measured based on the projected discounted future net cash flows arising from the assets. (H) Debt Issuance Costs and Debt Discount: Debt issuance costs related to a recognized debt liability are presented on the balance sheet as a direct deduction from the carrying amount of that debt liability, consistent with debt discounts, and are amortized to interest expense over the term of the related debt using the effective interest method. Further, debt discounts created as a result of the allocation of proceeds received from a debt issuance to warrants issued in conjunction with the debt issuance are amortized to interest expense under the effective interest method over the life of the recognized debt liability. (I) Research and Development Expense: Research and development costs are expensed as incurred. Clinical study costs are accrued over the service periods specified in the contracts and adjusted as necessary based upon an ongoing review of the level of effort and costs actually incurred. Payments for a product license prior to regulatory approval of the product and payments for milestones achieved prior to regulatory approval of the product are expensed in the period incurred as research and development. Milestone payments made in connection with final regulatory approvals are capitalized and amortized to cost of revenue over the remaining useful life of the asset. Research and development costs primarily consist of intellectual property and research and development materials acquired under license and license and collaboration agreements (see Note 3) and expenses from third parties who conduct research and development activities on behalf of the Company. The Company expenses in-process research and development projects acquired as asset acquisitions which have not reached technological feasibility, and which have no alternative future use. (J) Income Taxes: Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and the respective tax bases. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in income in the period that includes the enactment date. A valuation allowance is recorded when, after consideration of all positive and negative evidence, it is not more likely than not that the Company's deferred tax assets will be realizable. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. When and if the Company were to recognize interest and penalties related to unrecognized tax benefits, they would be reported in tax expense in the consolidated statement of operations. (K) Stock-Based Compensation: Stock-based awards to employees and directors with only time-based vesting requirements are valued at fair value on the date of grant and that fair value is recognized on a straight-line basis over the requisite service period of the entire award and is included in research and development expense and general and administrative expense in the Company's consolidated statements of operations. The Company values such time-based stock options using the Black-Scholes option pricing model. Certain assumptions are made with respect to utilizing the Black-Scholes option pricing model, including the expected life of the award, the volatility of the underlying shares and the risk-free interest rate. The expected life of such time-based stock options is calculated using the simplified method (based on the mid-point between the vesting date and the end of the contractual term), and the risk-free interest rate is based on the rates paid on securities issued by the U.S. Treasury with a term approximating the expected life of the equity award. The expected share price volatility for such time-based stock option awards was estimated partially using weighted average measures of implied volatility and using the average historical price volatility for industry peers. The Company estimates the grant date fair value of stock option awards to employees with market-based performance conditions using a Monte Carlo Simulation method under the income approach. Certain assumptions are made with respect to utilizing the Monte Carlo Simulation method, including the volatility of the underlying shares and the drift rate, or estimated cost of equity. The expected share price volatility for such market-based performance stock option awards was estimated by taking the median historical price volatility for industry peers over the contractual term of the options. The drift rate, or estimated cost of equity, for such market-based performance stock option awards is based on various financial and risk-associated metrics of industry peers, as well as estimated factors specific to us. The Company accounts for stock-based payments to nonemployees issued in exchange for services in accordance with ASU No. 2018-07, " Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting " based upon the fair value of the equity instruments issued. Compensation expense for stock options issued to nonemployees is calculated using the Black-Scholes option pricing model on the grant date and is recorded over the service performance period. The Company recognizes forfeitures of awards when they occur. (L) Net Loss per Share of Common Stock: Basic net loss per share of common stock is computed by dividing the net loss applicable to shareholders of common stock by the weighted-average number of shares of common stock and 3,301,998 pre-funded warrants (see Note 9(B)) outstanding during the period, without further consideration for potentially dilutive securities. In accordance with ASC Topic 260, Earnings Per Share , the pre-funded warrants are included in the computation of basic net loss per share because the exercise price is negligible and they are fully vested and exercisable at any time after the original issuance date. Diluted net loss per share of common stock is computed by dividing the net loss applicable to shareholders of common stock by the diluted weighted-average number of shares of common stock outstanding during the period calculated in accordance with the treasury stock method. In periods in which the Company reports a net loss, all share of common stock equivalents are deemed anti-dilutive such that basic net loss per share of common stock and diluted net loss per share of common stock are equivalent. Potentially dilutive shares of common stock have been excluded from the diluted net loss per share of common stock computations in all periods presented because such securities have an anti-dilutive effect on net loss per share of common stock due to the Company’s net loss. There are no reconciling items used to calculate the weighted-average number of total shares of common stock outstanding for basic and diluted net loss per share of common stock data. Restricted Stock Units ("RSUs") and stock options outstanding for a total of 3.1 million and 2.3 million shares of common stock were not included in the calculation of diluted weighted-average shares of common stock outstanding for the years ended March 31, 2021 and 2020, respectively, because they were anti-dilutive given the net loss of the Company. (M) Financial Instruments and Fair Value Measurement: The Company utilizes fair value measurement guidance prescribed by accounting standards to value its financial instruments. The guidance establishes a fair value hierarchy for instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and the Company's own assumptions (unobservable inputs). Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company's assumptions about the inputs that market participants would use in pricing the asset or liability and are developed based on the best information available in the circumstances. Fair value is defined as the exchange price, or exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As a basis for considering market participant assumptions in fair value measurements, the guidance establishes a three-tier fair value hierarchy that distinguishes among the following: • Level 1-Valuations are based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access. • Level 2-Valuations are based on quoted prices for similar assets or liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active and models for which all significant inputs are observable, either directly or indirectly. • Level 3-Valuations are based on inputs that are unobservable (supported by little or no market activity) and significant to the overall fair value measurement. To the extent the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument's level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The Company's financial instruments include cash and cash equivalents, a receivable from the sale of a long-term investment and long-term restricted cash. Cash consists of non-interest-bearing deposits denominated in the U.S. dollar, Swiss franc and Euro, while cash equivalents consists of interest-bearing money market fund deposits denominated in the U.S. dollar, which are invested in debt securities issued or guaranteed by the U.S. government and repurchase agreements fully collateralized by U.S. Treasury and U.S. government securities. Cash and the receivable from the sale of a long-term investment are stated at their respective historical carrying amounts, which approximate fair value due to their short-term nature. Long-term restricted cash is stated at its historical carrying amount, which approximates fair value. The carrying value of the Company's money market fund included in cash and cash equivalents of $114.0 million at March 31, 2021 approximated fair value, which is based on quoted prices in active markets for identical securities. The following table summarizes the fair value of the Company's money market fund included in cash equivalents based on the inputs used at March 31, 2021 in determining such values (in thousands): Fair Value Price Quotations (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Money market fund $ 114,000 $ 114,000 $ — $ — (N) Recent Accounting Pronouncements: In February 2016, the FASB issued ASU No. 2016-02, " Topic 842 — Leases " ("Topic 842"), which requires lessees to recognize on their consolidated balance sheets a liability to make lease payments and a right-of-use asset representing their right to use the underlying asset for the lease term for both finance and operating leases with lease terms greater than twelve months. Topic 842 is effective for annual periods beginning after December 15, 2018, and interim periods within fiscal years beginning after December 15, 2018. Early adoption is permitted. A modified retrospective transition approach is required, applying the new standard to all leases existing at the date of initial application. Topic 842 allows entities to choose to use either (i) the effective date or (ii) the beginning of the earliest comparative period presented in the financial statements as the date of initial application. Topic 842 provides a number of optional practical expedients in transition. The Company adopted Topic 842 on April 1, 2019 using the optional modified retrospective transition method. Comparative periods were not restated. For leases that commenced prior to April 1, 2019, the Company elected the following package of practical expedients when assessing the transition impact: (1) not to reassess whether any expired or existing contracts are or contain leases; (2) not to reassess the lease classification for any expired or existing leases; and (3) not to reassess initial direct costs for any existing leases. The Company also elected to: (1) use the total lease term in its initial incremental borrowing rate calculation; (2) combine its lease and non-lease components and account for them as a single lease component; and (3) not apply the use of hindsight in determining the lease term when considering lessee options to extend or terminate the lease and to purchase the underlying asset. Upon adoption, the Company recorded corresponding aggregate operating lease right-of-use assets and operating lease liabilities of $3.0 million and $2.4 million, respectively, including $0.6 million of prepaid rent previously classified within other non-current assets in the Company's consolidated balance sheet that was reclassified to operating lease right-of-use assets. The adoption of the new standard did not materially impact the Company's consolidated results of operations and cash flows and did not have an impact on the Company's beginning accumulated deficit balance. For additional information regarding the Company's leases, see Note 5 for further information regarding the impact of the Company's adoption of Topic 842. In June 2016, the FASB issued ASU No. 2016-13, “ Financial Instruments — Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments " ("ASU No. 2016-13"). ASU 2016-13 requires that financial assets measured at amortized cost, such as loans, accounts and trade receivables and investments, be represented net of expected credit losses, which may be estimated based on relevant information such as historical experience, current conditions, and future expectation for each pool of similar financial asset. ASU No. 2016-13 requires enhanced disclosures related to trade receivables and associated credit losses. In May 2019, the FASB issued ASU No. 2019-05, “ Financial Instruments — Credit Losses (Topic 326): Targeted Transition Relief ", which allows for a transition election on certain instruments and is effective for Small Reporting Companies for fiscal years beginning after December 15, 2022 and interim periods in those fiscal years. In November 2019, the FASB issued ASU No. 2019-11, " Codification Improvements to Topic 326, Financial Instruments — Credit Losses ", which amends certain aspects of ASU NO. 2016-13, including transition relief for trouble debt restructuring, among other topics. While the Company does not expect the adoption of this guidance to materially impact the Company's consolidated financial statements and related disclosures because it does not currently have any investments or trade receivables and the receivable from the sale of a long-term investment outstanding at March 31, 2021 was collected in May 2021, the impact on the Company's consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. In December 2019, the FASB issued ASU No. 2019-12, " Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes " ("ASU No. 2019-12"). ASU No. 2019-12 simplifies the accounting for income taxes, eliminates certain exceptions within Income Taxes (Topic 740) , and clarifies certain aspects of the current guidance to promote consistency among reporting entities, and is effective for fiscal years, and for interim periods within those fiscal years, beginning after December 15, 2020, with early adoption permitted. Most amendments within ASU No. 2019-12 are required to be applied on a prospective basis, while certain amendments must be applied on a retrospective or modified retrospective basis. The Company early adopted the provisions of ASU No. 2019-12 on April 1, 2020, which did not have a material impact on its consolidated financial statements and related disclosures. In August 2020, the FASB issued ASU No. 2020-06, " Debt — Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging — Contracts in Entity's Own Equity (Subtopic 815-40) " ("ASU No. 2020-06"). ASU No. 2020-06 simplifies the accounting for convertible debt instruments by removing the beneficial conversion and cash conversion separation models for convertible instruments. Under ASU No. 2020-06, the embedded conversion features are no longer separated from the host contract for convertible instruments with conversion features that are not required to be accounted for as derivatives or that do not result in substantial premiums accounted for as paid-in capital. ASU No. 2020-06 also amends the accounting for certain contracts in an entity’s own equity that are currently accounted for as derivatives because of specific settlement provisions. In addition, the new guidance modifies how particular convertible instruments and certain contracts that may be settled in cash or shares impact the computation of diluted earnings or loss per share. The provisions of ASU No. 2020-06 are effective for fiscal years beginning after December 15, 2021, and interim periods within those fiscal years, with early adoption permitted, but no earlier than fiscal years beginning after December 15, 2020, including interim periods within those fiscal years. While the Company does not expect the adoption of ASU No. 2020-06 to materially impact the Company's consolidated financial statements and related disclosures because it does not currently maintain any debt instruments accounted for in accordance with ASC Subtopic 470-20, " Debt — Debt with Conversion and Other Options " or instruments accounted for as derivatives in accordance with ASC Subtopic 815-40, " Derivatives and Hedging — Contracts in Entity's Own Equity ", and the Company also currently includes outstanding pre-funded warrants in the computation of basic net loss per share (see Note 2(L)), the impact on the Company's consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. In May 2021, the FASB issued ASU No. 2021-04, " Earnings Per Share (Topic 260), Debt — Modifications and Extinguishments (Subtopic 470-50), Compensation — Stock Compensation (Topic 718), and Derivatives and Hedging — Contracts in Entity’s Own Equity (Subtopic 815-40): Issuer’s Accounting for Certain Modifications or Exchanges of Freestanding Equity-Classified Written Call Options " ("ASU No. 2021-04"). ASU No. 2021-04 provides a principles-based framework for issuers to account for a modification or exchange of freestanding equity-classified written call options. To the extent applicable, issuers first reference other U.S. GAAP to account for the effect of the modification. In the absence of other U.S. GAAP, ASU No. 2021-04 clarifies whether to account for the effect as an adjustment to equity, and the related EPS implications, or as an expense, and if so the manner and pattern of recognition. The provisions of ASU No. 2021-04 are effective for annual periods beginning after December 15, 2021, including interim periods within those fiscal years. Early adoption is permitted. While the Company does not expect the adoption of ASU No. 2021-04 to materially impact the Company's consolidated financial statements and related disclosures because it does not currently anticipate modifications to its outstanding equity-classified written call options, the impact on the Company's consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. Other recent accounting pronouncements issued by the FASB (including its Emerging Issues Task Force), the American Institute of Certified Public Accountants, and the SEC did not, or are not believed by management to, have a material impact on the Company’s present or future consolidated financial position, results of operations or cash flows. (O) Foreign Currency: The Company has operations in the United States, the United Kingdom, Ireland and Switzerland. The results of its non-U.S. dollar based functional currency operations are translated to U.S. dollars at the average exchange rates during the year. The Company’s assets and liabilities are translated using the current exchange rate as of the balance sheet date and shareholders’ equity is translated using historical rates. Adjustments resulting from the translation of the financial statements of the Company’s foreign functional currency subsidiaries into U.S. dollars are excluded from the determination of net loss and are accumulated in a separate component of shareholders’ equity. Foreign exchange transaction gains and losses are included in other (income) expense in the Company’s results of operations. |
License and Collaboration Agree
License and Collaboration Agreements | 12 Months Ended |
Mar. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
License and Collaboration Agreements | License and Collaboration Agreements (A) The University of Massachusetts Medical School Exclusive License Agreement: In December 2018, the Company entered into an exclusive license agreement (the "UMMS Agreement"), with the University of Massachusetts Medical School ("UMMS") pursuant to which the Company received a worldwide, royalty-bearing, sub-licensable license under certain patent applications and any patents issuing therefrom, biological materials and know-how controlled by UMMS to develop and commercialize gene therapy product candidates, including AXO-AAV-GM1 and AXO-AAV-GM2, for the treatment of GM1 gangliosidosis and GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), respectively. This license is exclusive with respect to patents and biological materials and non-exclusive with respect to know-how and is subject to UMMS' retained rights for academic research, teaching and non-commercial patient care purposes, as well as to certain pre-existing rights of the U.S. government. Under the UMMS Agreement, the Company is solely responsible, at its expense, for the research, development and commercialization of the licensed gene therapy product candidates. The Company will reimburse UMMS for payments made by UMMS for the manufacture of clinical trial materials for the Company, up to a specified amount. The Company is obligated to use diligent efforts to develop and commercialize the licensed gene therapy product candidates and is required to achieve certain development and commercial milestones in accordance with the timeline set forth in the agreement. In addition, the Company could be obligated to make payments to UMMS totaling up to $24.5 million upon the achievement of specified development and regulatory milestones, including development and regulatory milestones of $1.0 million and $1.0 million that were achieved in February 2019 and October 2019, respectively, and that were paid during the fiscal year ended March 31, 2020, and up to $39.8 million upon the achievement of specified commercial milestones. The Company is also obligated to pay UMMS tiered mid-single digit royalties based on yearly net sales of the licensed products, subject to a specified annual minimum amount. Additionally, the Company will pay UMMS a percentage of any revenues it receives from any third-party sublicenses to licensed products at rates ranging in the mid-single digits to mid-teens. Excluding development and regulatory milestones achieved, the Company incurred a total of $6.9 million and $8.8 million of program-specific costs related to its AXO-AAV-GM1 and AXO-AAV-GM2 programs within research and development expenses in its consolidated statements of operations during the years ended March 31, 2021 and March 31, 2020, respectively. The Company paid a total of $29 thousand and $3.0 million to UMMS during the years ended March 31, 2021 and March 31, 2020, respectively, including payments for development and regulatory milestones achieved. The UMMS Agreement expires upon the expiration of the Company's obligations to make royalty payments to UMMS, which continues until the later of the expiration of licensed patents and any applicable orphan designation exclusivity and 10 years after the first commercial sale of the licensed products. Upon such expiration, the licenses granted to the Company by UMMS will automatically convert to perpetual, irrevocable, worldwide royalty-free licenses. The Company has the right to terminate the UMMS Agreement at any time upon 90 days' advance written notice to UMMS. Either party may terminate the UMMS Agreement for the other party's uncured material breach upon 60 days' advance written notice, including in the event that UMMS reasonably determines the Company has not fulfilled its diligence obligations. (B) Oxford Biomedica License Agreement: In 2018, the Company entered into an exclusive license agreement (the "Oxford Agreement") with Oxford Biomedica (UK) Ltd. ("Oxford"), pursuant to which the Company received a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Oxford to develop and commercialize AXO-Lenti-PD and related gene therapy products for all diseases and conditions. Under the terms of the Oxford Agreement, the Company could be obligated to make payments to Oxford totaling up to $55.0 million upon the achievement of specified development milestones, including certain development milestones that were achieved in April 2019 that resulted in a $13.0 million net payment due to Oxford that was paid during the year ended March 31, 2020, and up to $757.5 million upon the achievement of specified regulatory and sales milestones. The Company will also be obligated to pay Oxford a tiered royalty from 7% to 10%, based on yearly aggregate net sales of the underlying gene therapy products, subject to specified reductions upon the occurrence of certain events as set forth in the Oxford Agreement. These royalties are required to be paid, on a product-by-product and country-by-country basis, until the latest to occur of the expiration of the last to expire valid claim of a licensed patent covering such product in such country, the expiration of regulatory exclusivity for such product in such country, or 10 years after the first commercial sale of such product in such country. The Company is solely responsible, at its expense, for all activities related to the development and commercialization of the gene therapy products underlying the Oxford Agreement. Pursuant to the Oxford Agreement, the Company is required to use commercially reasonable efforts to develop, obtain regulatory approval of, and commercialize a gene therapy product underlying the Oxford Agreement in the United States and at least one major market country in Europe. In addition, the Company is required to meet certain diligence milestones and to include at least one U.S.-based clinical trial site in a pivotal study of a gene therapy product underlying the Oxford Agreement. If the Company fails to meet any of these specified development milestones, it may cure such failure by paying Oxford certain fees, which range from $0.5 million to $1.0 million. Excluding development milestones achieved, the Company incurred $5.7 million and $9.2 million of AXO-Lenti-PD program-specific costs within research and development expenses in its consolidated statement of operations during the years ended March 31, 2021 and March 31, 2020, respectively. The Company paid a total of $3.5 million and $15.2 million to Oxford during the years ended March 31, 2021 and March 31, 2020, respectively, including payments for development milestones achieved. The Company has the right to terminate the Oxford Agreement at any time upon two months' advance written notice prior to the first commercial sale of a product, or for a specified period of advance written notice after the first commercial sale of a product. Either party may terminate the Oxford Agreement for the other party's uncured material breach or with respect to a failure to make a required payment. (C) Benitec Biopharma License and Collaboration Agreement: In July 2018, the Company entered into a license and collaboration agreement (the "Benitec Agreement") with Benitec Biopharma Limited ("Benitec"), pursuant to which the Company received a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Benitec to develop and commercialize investigational gene therapy AXO-AAV-OPMD and related gene therapy products (collectively, the "AXO-AAV-OPMD Program") for all diseases and conditions. The Company terminated the Benitec Agreement in its entirety, effective September 3, 2019. The Company incurred $1.8 million of AXO-AAV-OPMD Program-specific costs within research and development expenses in its consolidated statement of operations and paid a total of $2.8 million to Benitec during the year ended March 31, 2020. |
Investment in Arvelle Therapeut
Investment in Arvelle Therapeutics B.V | 12 Months Ended |
Mar. 31, 2021 | |
Investments, Debt and Equity Securities [Abstract] | |
Investment in Arvelle Therapeutics B.V. | Investment in Arvelle Therapeutics B.V. On February 13, 2019, the Company entered into a share subscription agreement (the "Subscription Agreement") to purchase up to approximately 8.1 million shares of nonredeemable convertible preferred stock of Arvelle Therapeutics B.V. in exchange for €0.00001 per share paid in cash, as well as certain goods and services provided by the Company to Arvelle. The Company accounted for its investment in Arvelle in accordance with the provisions of ASC 321, " Investments - Equity Securities ", and elected to use the measurement alternative therein. The first closing under the Subscription Agreement occurred on February 25, 2019 with the Company purchasing approximately 5.9 million nonredeemable convertible preferred shares of Arvelle, which was initially recorded at a fair value of $5.9 million and was capitalized as a long-term investment in the Company's consolidated balance sheet and recorded to other non-operating income in its consolidated statement of operations. The Company also received the right to purchase up to approximately 2.2 million additional nonredeemable convertible preferred shares of Arvelle at a price of €0.00001 per share upon a potential future second closing under the Subscription Agreement. In May 2020, the Company fully exercised this right and purchased the approximately 2.2 million additional nonredeemable convertible preferred shares upon the closing of the second financing under the Subscription Agreement, which was recorded at a fair value of $2.2 million and was capitalized as a long-term investment in the Company's consolidated balance sheet and recorded to other non-operating income in the Company's consolidated statement of operations. In February 2021, the Company sold its investment in Arvelle to a third party as part of that third party's cash acquisition of all of the outstanding equity of Arvelle. In exchange, the Company received an upfront payment of approximately $11.6 million,in addition to a future payment to be received of approximately $1.2 million that is being held in escrow until August 2022 and that was recorded to long-term restricted cash in the Company's consolidated balance sheet at March 31, 2021, as well as the right to receive up to an additional total of $7.0 million in potential future regulatory and sales milestone payments (collectively, the "Arvelle Sale"). The Company recorded a net gain of approximately $4.7 million to other non-operating income in the Company's consolidated statement of operations upon the closing of the Arvelle Sale in February 2021, as well as a gain of approximately $4.3 million recorded to other non-operating income in the Company's consolidated statement of operations and to receivable from sale of long-term investment in its consolidated balance sheet upon the achievement of a regulatory milestone in March 2021. |
Leases
Leases | 12 Months Ended |
Mar. 31, 2021 | |
Leases [Abstract] | |
Leases | Leases The Company adopted the provisions of Topic 842 on April 1, 2019 using the effective date as its date of initial application and applied the optional modified retrospective transition method. Comparative periods were not restated. For leases that commenced prior to April 1, 2019, the Company elected the following package of practical expedients when assessing the transition impact: (i) not to reassess whether any expired or existing contracts are or contain leases; (ii) not to reassess the lease classification for any expired or existing leases; and (iii) not to reassess initial direct costs for any existing leases. The Company also elected to: (i) use the total lease term in its initial incremental borrowing rate calculation; (ii) combine its lease and non-lease components and account for them as a single lease component; and (iii) not apply the use of hindsight in determining the lease term when considering lessee options to extend or terminate the lease and to purchase the underlying asset. Upon adoption, the Company recorded corresponding aggregate operating lease right-of-use assets and operating lease liabilities of $3.0 million and $2.4 million, respectively, including $0.6 million of prepaid rent previously classified within other non-current assets in the Company’s consolidated balance sheet that was reclassified to operating lease right-of-use assets. Operating right-of-use assets and obligations were recognized based on the present value of remaining lease payments over the lease term using an incremental borrowing rate of 12.9% upon adoption. As the Company’s operating leases do not provide an implicit rate, estimated incremental borrowing rates were used based on the information available at the adoption date in determining the present value of lease payments. Operating lease expense is recognized on a straight-line basis over the lease term. Variable lease costs such as common area costs and other operating costs are expensed as incurred. Leases with an initial term of 12 months or less are not recorded within the Company's balance sheet. In addition, the Company reviews agreements at inception to determine if they include a lease, and when they do, uses its incremental borrowing rate or implicit interest rate to determine the present value of the future lease payments. In August 2020, the Company entered into a lease agreement for an office facility in New York, New York that commenced in December 2020 and ends in June 2026. Upon commencement of this lease, the Company recorded operating lease right-of-use assets and operating lease liabilities of approximately $1.1 million, net of expected abatement, based on the present value of payments over the lease term using an incremental borrowing rate of 8.9%. The Company also leases an office facility in Durham, North Carolina with a lease term ending in November 2022, and the Company had leased other office facilities in New York, New York and Princeton, New Jersey with lease terms that ended in January 2021 and October 2020, respectively. Each of these leases are or were, as applicable, classified as an operating lease in accordance with the provisions of Topic 842. The aggregate weighted-average remaining payment term, aggregate weighted-average remaining lease term and aggregate weighted-average discount rate were 5.0 years, 5.1 years and 9.2%, respectively, for the Company's contractual rent obligations for its operating leases as of March 31, 2021. During the years ended March 31, 2021 and March 31, 2020, the Company incurred $1.6 million and $1.8 million, respectively, of rent expense associated with contractual rent obligations for its operating leases. During the years ended March 31, 2021 and March 31, 2020, the Company paid $0.9 million and $1.8 million, respectively, related to its contractual rent obligations. The following table provides a reconciliation of the Company's remaining undiscounted contractual rent obligations due within each respective fiscal year ending March 31 to the operating lease liabilities recognized as of March 31, 2021 (in thousands): Year Ending March 31, Amount 2022 $ 327 2023 272 2024 295 2025 303 2026 311 Thereafter 53 Total undiscounted payments 1,561 Less: present value adjustment (318) Present value of future payments $ 1,243 |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Mar. 31, 2021 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | Accrued Expenses As of March 31, 2021 and 2020, accrued expenses consisted of the following (in thousands): March 31, 2021 March 31, 2020 Research and development expenses $ 6,091 $ 6,951 Bonuses and other compensation expenses 2,331 2,521 Legal expenses 15 704 Other expenses 759 1,143 Total accrued expenses $ 9,196 $ 11,319 |
Long Term Debt
Long Term Debt | 12 Months Ended |
Mar. 31, 2021 | |
Debt Disclosure [Abstract] | |
Long Term Debt | Long-term DebtIn February 2017, the Company and certain of its subsidiaries (the "Borrowers") entered into a loan and security agreement (as amended in May 2017, September 2017 and November 2019) with Hercules Capital, Inc. ("Hercules") (the "Loan Agreement"), under which the Borrowers borrowed an aggregate of $55.0 million (the "Term Loan"). The Term Loan had a scheduled maturity date of March 1, 2021. The Term Loan initially bore interest at a variable per annum rate calculated for any day as the greater of either (i) the prime rate plus 6.80%, and (ii) 10.55%, which was subsequently changed in connection with the November 2019 amendment of the Loan Agreement to a variable per annum rate calculated for any day as the greater of either (i) the prime rate plus 6.80%, and (ii) 11.55%. The Borrowers were initially obligated to make monthly payments of accrued interest under the Loan Agreement until September 2018, followed by monthly installments of principal and interest from October 2018 until March 2021. Subsequent to the November 2019 amendment of the Loan Agreement, the Borrowers were obligated to make monthly payments of accrued interest from December 2019 until August 2020, followed by monthly installments of principal and interest from September 2020 until March 2021. Prepayment of the Term Loan was subject to penalty. The Company prepaid 50%, or approximately $15.7 million, of outstanding principal due without penalty in connection with the November 2019 amendment of the Loan Agreement, which was accounted for as a modification of debt in accordance with applicable accounting guidance. In April 2020, the Company prepaid $15.7 million of outstanding principal, together with $0.3 million of accrued interest, fees and other amounts, due under the Loan Agreement with Hercules, which was accounted for as an extinguishment of debt with a corresponding loss of approximately $0.5 million recorded to interest expense during the year ended March 31, 2021. In connection with the prepayment, the credit facility and the Loan Agreement with Hercules were terminated, and all obligations, liens and security interests under the Loan Agreement were released, discharged and satisfied. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Mar. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | Related Party Transactions (A) Services Agreements: The Company has entered into services agreements with Roivant Sciences, Inc. ("RSI") and Roivant Sciences GmbH (collectively, the "Service Providers"), each a wholly owned subsidiary of RSL, pursuant to which the Service Providers provide the Company with services in relation to the identification of potential product candidates and project management of clinical trials, as well as other services related to the Company's development, administrative and financial functions (the "Services Agreements"). Under the terms of the Services Agreements, the Company is obligated to pay or reimburse the Service Providers for the costs they, or third parties acting on their behalf, incur in providing services to the Company, including administrative and support services as well as research and development services. In addition, the Company is obligated to pay to the Service Providers at a predetermined mark-up on any general and administrative and research and development services incurred directly by the Service Providers. Under the terms of the Services Agreements, the Service Providers have agreed to indemnify the Company and its officers, employees and directors against all losses arising out of, due to or in connection with the provision of services (or the failure to provide services) under the Services Agreements, subject to certain limitations set forth in the Service Agreements. In addition, the Company has agreed to indemnify the Service Providers and their respective affiliates and officers, employees and directors against all losses arising out of, due to or in connection with the receipt of services under the Services Agreements, subject to certain limitations set forth in the Services Agreements. Such indemnification obligations will not exceed the payments made by the Company under the Services Agreements for the specific service that allegedly caused or was related to the losses during the period in which such alleged losses were incurred. The term of each of the services agreements will continue until terminated upon 90 days’ written notice by any party with respect to the services such party provides or receives thereunder. For the years ended March 31, 2021 and 2020, the Company incurred expenses of $0.1 million and $0.1 million, respectively, under the Services Agreements, inclusive of the mark-up, which have been treated as capital contributions (see Note 9(B)). (B) Information Sharing and Cooperation Agreement: In March 2015, the Company entered into an information sharing and cooperation agreement with RSL, as amended and restated in June 2018 (the "Restated Cooperation Agreement") in connection with a share purchase placement agreement with RSL (the "Private Placement"), for which the amendments became effective in July 2018 upon the closing of the Private Placement. The Restated Cooperation Agreement, among other things, obligates the Company to deliver periodic financial statements and other financial information to RSL and comply with other specified financial reporting requirements, and requires the Company to implement and observe certain policies and procedures related to applicable laws and regulations. The Company agreed to indemnify RSL and its affiliates and their respective officers, employees and directors against all losses arising out of, due to or in connection with RSL’s status as a shareholder under the Restated Cooperation Agreement and the operations of or services provided by RSL or its affiliates or their respective officers, employees or directors to the Company or any of its subsidiaries, subject to certain limitations set forth in the Restated Cooperation Agreement. Subject to specified exceptions, the Restated Cooperation Agreement will terminate at such time as RSL is no longer required (a) under U.S. GAAP to consolidate the Company's results of operations and financial position, (b) under U.S. GAAP to account for its investment in the Company under the equity method of accounting, or (c) otherwise to include separate financial statements of the Company in its filings with the SEC pursuant to any SEC rule. In addition, the Cooperation Agreement may be terminated upon mutual written consent of the parties or upon written notice from RSL to the Company in the event of the Company's bankruptcy, liquidation, dissolution or winding-up. (C) RSL Financing Participation: In February 2020, the Company issued and sold 16,631,336 shares of common stock and pre-funded warrants to purchase up to 3,301,998 shares of common stock in a follow-on public offering, including 2,600,000 shares of common stock sold pursuant to the exercise of the underwriters’ option to purchase additional shares and also including 5,333,333 shares of common stock issued and sold to RSL, at an offering price of $3.75 per share of common stock and $3.74999 per pre-funded warrant. The net proceeds to the Company were approximately $70.8 million, after deducting underwriting discounts and commissions and offering expenses incurred (see Note 9(B)). |
Stockholders_ Equity
Stockholders’ Equity | 12 Months Ended |
Mar. 31, 2021 | |
Stockholders' Equity Note [Abstract] | |
Stockholders’ Equity | Stockholders’ Equity (A) Overview: Sio's Certificate of Incorporation filed with the State of Delaware on November 12, 2020 authorizes the issuance of up to a total of 1,010,000,000 shares, of which 1,000,000,000 shares are common stock with a par value of $0.00001 per share and 10,000,000 shares are preferred stock with a par value of $0.00001 per share. A 1-for-8 reverse stock split of the Company's outstanding common stock was effected in May 2019 as approved by the Company's Board of Directors and a majority of its shareholders. As such, all references to share and per share amounts in these consolidated financial statements and accompanying notes have been retroactively restated to reflect the 1-for-8 reverse stock split, except for the authorized number of shares of the Company's common stock and the par value per share, which were not affected. The reverse stock split reduced the number of the Company's shares of common stock issued and outstanding from approximately 182.2 million to 22.8 million as of March 31, 2019. (B) Transactions: During the years ended March 31, 2021 and March 31, 2020, expenses of $0.1 million and $0.1 million, respectively, of expenses were incurred by RSI on behalf of the Company and were recorded as capital contributions (see Note 8(A)). In February 2020, the Company issued and sold 16,631,336 shares of common stock and pre-funded warrants to purchase up to 3,301,998 shares of common stock in a follow-on public offering, including 2,600,000 shares of common stock sold pursuant to the exercise of the underwriters’ option to purchase additional shares and also including 5,333,333 shares of common stock issued and sold to RSL, at an offering price of $3.75 per share of common stock and $3.74999 per pre-funded warrant. The net proceeds to the Company were approximately $70.8 million, after deducting underwriting discounts and commissions and offering expenses incurred (see Note 8(C)). The pre-funded warrants do not expire and are immediately exercisable except that the pre-funded warrants cannot be exercised by the holder if, after giving effect thereto, the holder would beneficially own more than 9.99% of the Company’s common stock, subject to certain exceptions. The pre-funded warrants are classified as equity in accordance with ASC 480, " Distinguishing Liabilities from Equity ", and the fair value of the pre-funded warrants was recorded as a credit to additional paid-in capital and is not subject to remeasurement. As of March 31, 2021, none of the pre-funded warrants had been exercised. During the year ended March 31, 2021, the Company engaged SVB Leerink LLC as its agent to sell shares of the Company's common stock from time to time through an at-the-market equity offering program. SVB Leerink LLC receives compensation for its services in an amount equal to 3% of the gross proceeds of any of the Company's common stock sold. As of March 31, 2021, the Company sold approximately 29.7 million shares of its common stock for total proceeds of approximately $90.5 million, net of brokerage fees, under this program, and subsequent to March 31, 2021, the Company has sold approximately 0.2 million shares of its common stock for total proceeds of approximately $0.5 million, net of brokerage fees (see Note 14). |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Mar. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Stock-Based Compensation | Stock-Based Compensation (A) Amended and Restated 2015 Equity Incentive Plan: In March 2015, the Company adopted its 2015 Equity Incentive Plan, which was amended and restated in June 2017 by its Board of Directors and became effective upon shareholder approval in August 2017 and was further amended and restated by the Company's Board of Directors in October 2020 (the "2015 Plan"). A 1-for-8 reverse stock split of the Company's outstanding common stock was effected on in 2019 as approved by the Company's Board of Directors and a majority of its shareholders. As such, all references to share and per share amounts in these consolidated financial statements and accompanying notes have been retroactively restated to reflect the 1-for-8 reverse stock split, except for the authorized number of shares of the Company's common stock and the par value per share, which were not affected. The reverse stock split reduced each of (i) the number of shares authorized for issuance from 24.8 million to 3.1 million, (ii) the number of shares available for issuance from 8.4 million to 1.0 million, and (iii) the number of options outstanding from 15.4 million to 1.9 million under the 2015 Plan as of March 31, 2019. In April 2019 and April 2020, the number of shares of common stock authorized for issuance under the 2015 Plan increased automatically by 0.9 million and 1.6 million, respectively, in accordance with the terms of the 2015 Plan. At March 31, 2021, totals of 5.6 million shares of common stock were authorized for issuance and 2.0 million shares of common stock were available for future issuance under the 2015 Plan. (B) Stock Options: Stock options granted under the 2015 Plan provide option holders, if approved by the Board of Directors, the right to exercise their options prior to vesting. In the event that an option holder exercises the unvested portion of any option, such unvested portion will be subject to a repurchase option held by the Company at the lower of (i) the fair market value of its common stock on the date of repurchase and (ii) the exercise price of the options. Any shares of common stock underlying such unvested portion will continue to vest in accordance with the original vesting schedule of the option. Time-based stock options granted to the Company's employees vest over a period of four years with 25% of the shares of common stock underlying the option vesting on the first anniversary of the vesting commencement date and the remainder vesting in 12 quarterly installments thereafter, subject to continuing service. Initial stock options granted to the Company's non-employee directors vest in equal installments on the first, second and third anniversaries of the vesting commencement date, and stock options subsequently granted annually to the Company's non-employee directors vest fully on the first anniversary of the vesting commencement date, each subject to continuous service. Stock options granted to employees during the year ended March 31, 2020 include options with market-based performance conditions to purchase 0.4 million shares of common stock with a weighted average exercise price of $8.07 per share and a corresponding total estimated grant date fair value of $1.2 million, which was estimated using Monte Carlo Simulation method under the income approach. The Company did not grant any market-based performance stock options during the year ended March 31, 2021. At March 31, 2021, options with market-based performance conditions to purchase 0.4 million shares of common stock at a weighted average exercise price of $8.87 per share were outstanding. The market-based performance options vest based on the trading price for the Company's shares of common stock exceeding certain closing price thresholds. The Company estimated the fair value of each time-based stock option on the date of grant using the Black-Scholes closed-form option-pricing model applying the weighted average assumptions during the years ended March 31, 2021 and March 31, 2020, as follows: Years Ended March 31, 2021 2020 Expected stock price volatility 109.5 % 78.9 % Expected risk free interest rate 0.43 % 2.30 % Expected term, in years 6.11 6.13 Expected dividend yield — % — % The Company estimated the grant date fair value of each market-based performance stock option granted during the year ended March 31, 2020 using a Monte Carlo Simulation method under the income approach by applying the following assumptions: Expected share price volatility 73.6 - 74.3% Contractual term, in years 10 The following table presents a summary of stock option activity and data under the 2015 Plan: Number of Options Weighted Average Exercise Price Weighted Average Grant Date Fair Value Weighted Average Remaining Contractual Life (in years) Aggregate Intrinsic Value Options outstanding at March 31, 2019 1,919,158 $ 34.09 $ 49.35 6.86 $ 1,569,809 Granted 1,541,672 8.12 4.96 Exercised (11,778) 7.20 114.40 2,972 Forfeited (1,440,317) 33.99 50.32 Options outstanding at March 31, 2020 2,008,735 $ 14.39 $ 13.87 8.33 $ — Granted 434,775 3.72 3.07 Exercised — — — — Forfeited (347,967) 13.92 15.84 Options outstanding at March 31, 2021 2,095,543 $ 12.26 $ 11.30 7.90 $ — Options vested and expected to vest at March 31, 2021 2,095,543 $ 12.26 $ 11.30 7.90 $ — Options exercisable at March 31, 2021 1,571,191 $ 14.77 $ 13.78 7.59 $ — During the years ended March 31, 2021 and March 31, 2020, the total grant date fair values of options that vested under the 2015 Plan were $3.5 million and $3.8 million, respectively. At March 31, 2021 under the 2015 Plan, vested options to purchase a total of 0.9 million shares of common stock were outstanding, with no options with market-based performance conditions vested and outstanding. (C) Restricted Stock Units: Of the total number of RSUs granted in September 2019 representing approximately 0.3 million shares of the Company's common stock, one-half vested on January 31, 2020 and the remaining one-half vested on July 31, 2020, subject to continuing service. All other RSUs granted during the years ended March 31, 2021 and March 31, 2020 vest in three equal annual installments commencing on the first anniversary of the vesting commencement date, subject to continuing service. The following table presents a summary of restricted stock unit activity and data under the 2015 Plan: Number of RSUs Weighted Average Grant Date Fair Value RSUs outstanding at March 31, 2019 — $ — Granted 369,577 7.50 Vested and settled (103,294) 7.77 Forfeited (18,420) 7.77 RSUs outstanding at March 31, 2020 247,863 $ 7.37 Granted 1,247,850 3.37 Vested and settled (187,741) 7.68 Forfeited (281,756) 3.95 RSUs outstanding at March 31, 2021 1,026,216 $ 3.39 During the years ended March 31, 2021 and March 31, 2020, the total grant date fair values of RSUs that vested under the 2015 Plan were $1.0 million and $1.2 million, respectively. All 1.0 million of the RSUs outstanding at March 31, 2021 were unvested, and approximately 0.2 million of the RSUs outstanding at March 31, 2020 were unvested. (D) Stock-based Compensation Expense: The Company recorded total stock-based compensation expense of $4.4 million and $7.0 million for the years ended March 31, 2021 and March 31, 2020, respectively, related to options and RSUs granted to its employees and directors, excluding stock-based compensation expense allocated to the Company from RSL (see Note 10(E)). At March 31, 2021, total unrecognized compensation expense for unvested outstanding option and RSU equity awards of the Company's common stock granted to its employees and directors under the 2015 Plan was $6.2 million, which is expected to be recognized over the remaining weighted-average service period of 2.16 years. (E) RSL Common Share Awards and Options: Certain employees of the Company, as well as certain employees of RSL and certain of its subsidiaries, have been granted RSL common share awards and options for which stock-based compensation expense is allocated to the Company from RSL. The Company recorded such total allocated stock-based compensation expense of $0.1 million and $0.9 million during the years ended March 31, 2021 and March 31, 2020, respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Mar. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes The loss before income taxes and the related tax expense are as follows (in thousands): Year ended March 31, 2021 Year ended March 31, 2020 Loss before income taxes: United States $ (17,382) $ (4,403) Switzerland (1) (11,978) (62,520) Bermuda (2) (3,177) (5,216) Other (3) (100) (51) Total loss before income taxes $ (32,637) $ (72,190) Current taxes: United States $ (212) $ 403 Switzerland (1) — — Bermuda (2) — — Other (4) — 35 Total current tax expense (212) 438 Deferred taxes: United States — — Switzerland (1) — — Bermuda (2) — — Other (3) — — Total deferred tax expense — — Total income tax expense $ (212) $ 438 (1) Wholly-owned subsidiary, Axovant Sciences GmbH. (2) Bermuda entity was centrally controlled and managed in the United Kingdom. (3) Wholly-owned subsidiaries, Axovant Holdings Limited (United Kingdom) and Sio Europe Limited (Ireland). (4) Includes state income tax expense. A reconciliation of income tax benefit computed at the U.S/Bermuda statutory rate to income tax (benefit) expense reflected in the financial statements is as follows (in thousands): Year Ended Year Ended March 31, 2021 March 31, 2020 $ % $ % Income tax benefit at federal statutory rate $ (6,854) 21.00 % $ — — % Foreign rate differential (1) 1,118 (3.43) (17,268) 23.92 Nondeductible/nontaxable items (1,823) 5.59 766 (1.06) Valuation allowance 10,749 (32.94) 16,759 (23.21) Research and development credit (914) 2.80 (615) 0.85 Research and development true-up (301) 0.92 (285) 0.39 Deferred adjustments 2,139 (6.55) 1,068 (1.48) Restructuring (4,108) 12.59 — — Other (218) 0.67 13 (0.02) Total income tax (benefit) expense $ (212) 0.65 % $ 438 (0.61) % (1) Primarily related to current tax on United States operations including permanent and temporary differences, Swiss net operating losses and United Kingdom taxation of the parent company. Until November 12, 2020, the Company was not subject to taxation under the laws of Bermuda since AGT was organized as a Bermuda Exempted Limited Company, for which there is no current tax regime. Since November 12, 2020, when Sio was incorporated in the State of Delaware in connection with the Domestication (see Note 1), the Company is subject to taxation under the laws of the United States of America. The Company's provision for income taxes is primarily federal, state and local taxes in the United States. The Company's effective tax rates of 0.65% and (0.61)% for the years ended March 31, 2021 and March 31, 2020, respectively, differ from the U.S. federal statutory rate of 21% and the Bermuda federal statutory rate of 0% primarily due to the U.S. permanent unfavorable tax differences, stock compensation deductions and a valuation allowance that effectively eliminates the Company's net deferred tax assets. On March 27, 2020, the United States government enacted the Coronavirus Aid, Relief, and Economic Security Act (the "CARES Act") which includes numerous modifications to income tax provisions, including a limitation on business interest expense and net operating loss provisions and the acceleration of alternative minimum tax credits. Given the Company's history of losses, the CARES Act is not expected to have a material impact on its income tax positions. As of March 31, 2021, the Company had an aggregate tax receivable of $1.7 million from various federal, state and local jurisdictions. Deferred taxes reflect the tax effects of the differences between the amounts recorded as assets and liabilities for financial reporting purposes and the comparable amounts recorded for income tax purposes. Significant components of the deferred tax assets (liabilities) at March 31, 2021 and 2020 are as follows (in thousands): March 31, 2021 March 31, 2020 Deferred tax assets: Net operating loss $ 154,877 $ 160,319 Research tax credits 11,798 10,782 Stock-based compensation 8,341 10,738 Intangibles 8,747 6,738 Lease liability 257 206 Other 51 78 Subtotal 184,071 188,861 Valuation allowance (183,703) (187,762) Deferred tax liabilities: Right of use (250) (334) Other (118) (765) Total net deferred tax assets $ — $ — The Company had net operating losses in the United States, Switzerland, the United Kingdom and Ireland in the amounts of $3.2 million, $1.18 billion, $150 thousand and $77 thousand, respectively, as of March 31, 2021. The United States federal net operating loss can be carried forward indefinitely with utilization limited to 80% of future taxable income for tax years beginning after January 1, 2021. The Switzerland net operating loss will begin to expire as of March 31, 2025. The United Kingdom net operating loss can be carried forward indefinitely with an annual limitation on utilization. As of March 31, 2021, the Company has federal research and development carryforwards of approximately $11.8 million. If not utilized, the research and development credit carryforwards will start to expire in 2038. The Company assesses the realizability of the deferred tax assets at each balance sheet date based on available positive and negative evidence in order to determine the amount which is more likely than not to be realized and record a valuation allowance as necessary. Due to the Company's cumulative loss position which provides significant negative evidence difficult to overcome, the Company has recorded a full valuation allowance of $183.7 million as of March 31, 2021, representing the portion of the deferred tax asset that is not more likely than not to be realized. The Company will continue to assess the realizability of deferred tax assets at each balance sheet date in order to determine the proper amount, if any, required for a valuation allowance. As of March 31, 2021, the Company does not have unremitted earnings from foreign subsidiaries. The potential tax implications of the repatriation of unremitted earnings are driven by the facts at the time of distribution; however, due to U.S. tax reform and the Company’s current accumulated earnings deficit, the incremental cost to repatriate earnings is not expected to be material. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Mar. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Commitments and Contingencies As of March 31, 2021, the Company had entered into commitments under the UMMS Agreement (see Note 3(A)), the Oxford Agreement (see Note 3(B)), the Services Agreements with certain of RSL's wholly owned subsidiaries (see Note 8(A)) and agreements to rent office space (see Note 5). In addition, the Company has entered into services agreements with third parties for pharmaceutical manufacturing and research activities in the normal course of business, which can generally be terminated by the Company with 30- or 60-days' written notice, unless otherwise indicated. Further, certain of the Company's manufacturing agreements could require early termination and wind-down payments due from the Company upon either the termination of its clinical trials or if the Company terminates such agreements for convenience. The Company has the right to terminate the UMMS Agreement at any time upon 90 days' advance written notice to UMMS. Either party may terminate the UMMS Agreement for the other party's uncured material breach upon 60 days' advance written notice, including in the event that UMMS reasonably determines the Company has not fulfilled its diligence obligations. |
Selected Quarterly Financial Da
Selected Quarterly Financial Data (Unaudited) | 12 Months Ended |
Mar. 31, 2021 | |
Quarterly Financial Information Disclosure [Abstract] | |
Selected Quarterly Financial Data (Unaudited) | Selected Quarterly Financial Data (Unaudited) The following table presents selected quarterly financial data for the years ended March 31, 2021 and March 31, 2020 (in thousands, except per share amounts): First Quarter Ended Second Quarter Ended Third Quarter Ended Fourth Quarter Ended First Quarter Ended Second Quarter Ended Third Quarter Ended Fourth Quarter Ended June 30, September 30, December 31, March 31, June 30, September 30, December 31, March 31, 2020 2020 2020 2021 2019 2019 2019 2020 Research and development expenses $ 5,194 $ 5,058 $ 6,407 $ 8,244 $ 21,090 $ 6,833 $ 8,267 $ 10,920 General and administrative expenses 4,640 4,491 4,198 3,965 6,468 5,051 5,409 5,133 Total operating expenses 9,834 9,549 10,605 12,209 27,558 11,884 13,676 16,053 Net loss (8,594) (9,984) (10,516) (3,331) (28,057) (13,884) (14,039) (16,648) Net loss per share attributable to shareholders of common stock - basic and diluted $ (0.20) $ (0.21) $ (0.20) $ (0.05) $ (1.23) $ (0.61) $ (0.62) $ (0.54) |
Subsequent Events
Subsequent Events | 12 Months Ended |
Mar. 31, 2021 | |
Subsequent Events [Abstract] | |
Subsequent Events | Subsequent EventsSubsequent to March 31, 2021, the Company has sold approximately 0.2 million shares of its common stock for total proceeds of approximately $0.5 million, net of brokerage fees, through SVB Leerink LLC as placement agent. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation: The Company’s fiscal year ends on March 31, and its fiscal quarters end on June 30, September 30, and December 31. These consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States ("U.S. GAAP"). Any reference in these notes to applicable guidance is meant to refer to the authoritative U.S. GAAP as found in the Accounting Standards Codification ("ASC") and as amended by Accounting Standards Updates ("ASU") issued by the Financial Accounting Standards Board ("FASB"). These consolidated financial statements and accompanying notes include the accounts of the Company and its wholly owned subsidiaries. The Company has no unconsolidated subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Certain prior period balances have been reclassified to conform to the current period presentation. During November 2020, the historical financial statements and subsidiaries of AGT became the historical financial statements and subsidiaries of Sio upon consummation of the Domestication. As a result, these consolidated financial statements and accompanying notes reflect (i) the historical operating results of AGT and its subsidiaries prior to the Domestication; (ii) the operating results of the Company following the Domestication; and (iii) the Company’s equity structure for all periods presented. A 1-for-8 reverse stock split of the Company's outstanding common stock was effected in May 2019 as approved by the Company's Board of Directors and a majority of its shareholders. As such, all references to share and per share amounts in these financial statements and accompanying notes have been retroactively restated to reflect the 1-for-8 reverse stock split, except for the authorized number of shares of the Company's common stock and the par value per share, which were not affected. |
Going Concern and Management's Plans | Going Concern and Management's Plans: The Company assesses and determines its ability to continue as a going concern in accordance with the provisions of ASC Subtopic 205-40, " Presentation of Financial Statements—Going Concern " ("ASC Subtopic 205-40"), which requires the Company to evaluate whether there are conditions or events that raise substantial doubt about its ability to continue as a going concern within one year after the date that its annual and interim consolidated financial statements are issued. Certain additional financial statement disclosures are required if such conditions or events are identified. If and when an entity’s liquidation becomes imminent, financial statements should be prepared under the liquidation basis of accounting. Determining the extent, if any, to which conditions or events raise substantial doubt about the Company’s ability to continue as a going concern, or the extent to which mitigating plans sufficiently alleviate any such substantial doubt, as well as whether or not liquidation is imminent, requires judgment by management. The Company has evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date the consolidated financial statements are issued. The Company is currently a development stage company, and thus, has not yet achieved profitability. The Company expects to continue to incur significant operating and net losses, as well as negative cash flows from operations, for the foreseeable future as it continues to develop its gene therapy product candidates and prepares for potential future regulatory approvals and commercialization of its products. The Company has not generated any revenue to date and does not expect to generate product revenue unless and until it successfully completes development and obtains regulatory approval for at least one of its gene therapy product candidates. The Company's current cash and cash equivalents balance will also not be sufficient to complete all necessary development activities and commercially launch its products. For the years ended March 31, 2021 and March 31, 2020, the Company incurred net losses of $32.4 million and $72.6 million, respectively. As of March 31, 2021, the Company's cash and cash equivalents totaled $119.0 million and its accumulated deficit was $791.1 million. The Company estimates that its significantly increased current cash and cash equivalents balance, which includes cash proceeds received from its sales agreement with SVB Leerink LLC to sell shares of common stock from time to time through an at-the-market equity offering program, as well as cash proceeds received in connection with the sale of its long-term investment in Arvelle Therapeutics B.V. ("Arvelle"), is sufficient to support operations beyond the twelve month period following the date that these consolidated financial statements were issued, including beyond the expected dates of major upcoming milestones for the Company's AXO-AAV-GM1 gene therapy program for the treatment of GM1 gangliosidosis. As such, the Company has determined that there is no longer substantial doubt about its ability to continue as a going concern for the one-year period following the date that these consolidated financial statements and footnotes were issued. These estimates are based on assumptions that may prove to be wrong, and the Company could use its available capital resources sooner than it currently expects. In order to meet the Company's long-term operating requirements, the Company will need, among other things, additional capital resources. The Company continually assesses multiple options to obtain additional funding to support its operations, including proceeds from offerings of its equity securities or debt, or transactions involving product development, technology licensing or collaboration arrangements, or other sources of capital to complete its currently planned development programs. Management can provide no assurances that it can raise a sufficient amount of financing for the Company on favorable terms, if at all. |
Use of Estimates | Use of Estimates: The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. The Company regularly evaluates estimates and assumptions related to certain assets and liabilities, including its research and development accruals, as well as assumptions used to estimate the fair value of its stock option awards, estimate its income tax expense and estimate its ability to continue as a going concern. Specifically, the Company’s assessment of the completeness of the information for research and development accruals is subject to variability and uncertainty. In addition, in certain circumstances, the determination of the nature and amount of research and development services that have been received during the reporting period requires judgment as the timing and pattern of vendor invoicing does not correspond to the level of services provided. The Company estimates the grant date fair value of stock option awards with only time-based vesting requirements using a Black-Scholes valuation model and uses a Monte Carlo Simulation method under the income approach to estimate the grant date fair value of stock option awards with market-based performance conditions. The Company bases its estimates and assumptions on historical experience and on various other factors that it believes to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates. Additionally, the Company assessed the impact that the COVID-19 pandemic has had on its operations and financial results as of March 31, 2021 and through the date of issuance of these audited consolidated financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and expenses. |
Risks and Uncertainties | Risks and Uncertainties:The Company is subject to risks common to companies in the pharmaceutical industry including, but not limited to, uncertainties related to commercialization of products, regulatory approvals, dependence on key products, dependence on key customers and suppliers, and protection of intellectual property rights. |
Concentrations of Credit Risk | Concentrations of Credit Risk:Financial instruments that potentially subject the Company to concentration of credit risk include cash and cash equivalents. At March 31, 2021, substantially all of the cash balances are deposited in 2 banking institutions, each in excess of insured levels. |
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 money market funds. |
Property and Equipment | Property and Equipment: Property and equipment, consisting of leasehold improvements, furniture and fixtures, computers, software and other office and laboratory equipment, is recorded at cost. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred. Upon disposal, retirement or sale, the related cost and accumulated depreciation is removed from the accounts and any resulting gain or loss is included in the results of operations. Depreciation is recorded for property and equipment using the straight-line method over the estimated useful lives of the respective assets, generally three The Company reviews the recoverability of all long-lived assets, including the related useful lives, whenever events or changes in circumstances indicate that the carrying amount of a long-lived asset might not be recoverable. Recoverability is measured by comparison of the book values of the assets to future net undiscounted cash flows that the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book value of the assets exceed their fair value, which is measured based on the projected discounted future net cash flows arising from the assets. |
Debt Issuance Costs and Debt Discount | Debt Issuance Costs and Debt Discount:Debt issuance costs related to a recognized debt liability are presented on the balance sheet as a direct deduction from the carrying amount of that debt liability, consistent with debt discounts, and are amortized to interest expense over the term of the related debt using the effective interest method. Further, debt discounts created as a result of the allocation of proceeds received from a debt issuance to warrants issued in conjunction with the debt issuance are amortized to interest expense under the effective interest method over the life of the recognized debt liability. |
Research and Development Expense | Research and Development Expense:Research and development costs are expensed as incurred. Clinical study costs are accrued over the service periods specified in the contracts and adjusted as necessary based upon an ongoing review of the level of effort and costs actually incurred. Payments for a product license prior to regulatory approval of the product and payments for milestones achieved prior to regulatory approval of the product are expensed in the period incurred as research and development. Milestone payments made in connection with final regulatory approvals are capitalized and amortized to cost of revenue over the remaining useful life of the asset. Research and development costs primarily consist of intellectual property and research and development materials acquired under license and license and collaboration agreements (see Note 3) and expenses from third parties who conduct research and development activities on behalf of the Company. The Company expenses in-process research and development projects acquired as asset acquisitions which have not reached technological feasibility, and which have no alternative future use. |
Income Taxes | Income Taxes:Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and the respective tax bases. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in income in the period that includes the enactment date. A valuation allowance is recorded when, after consideration of all positive and negative evidence, it is not more likely than not that the Company's deferred tax assets will be realizable. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. When and if the Company were to recognize interest and penalties related to unrecognized tax benefits, they would be reported in tax expense in the consolidated statement of operations. |
Stock-Based Compensation | Stock-Based Compensation: Stock-based awards to employees and directors with only time-based vesting requirements are valued at fair value on the date of grant and that fair value is recognized on a straight-line basis over the requisite service period of the entire award and is included in research and development expense and general and administrative expense in the Company's consolidated statements of operations. The Company values such time-based stock options using the Black-Scholes option pricing model. Certain assumptions are made with respect to utilizing the Black-Scholes option pricing model, including the expected life of the award, the volatility of the underlying shares and the risk-free interest rate. The expected life of such time-based stock options is calculated using the simplified method (based on the mid-point between the vesting date and the end of the contractual term), and the risk-free interest rate is based on the rates paid on securities issued by the U.S. Treasury with a term approximating the expected life of the equity award. The expected share price volatility for such time-based stock option awards was estimated partially using weighted average measures of implied volatility and using the average historical price volatility for industry peers. The Company estimates the grant date fair value of stock option awards to employees with market-based performance conditions using a Monte Carlo Simulation method under the income approach. Certain assumptions are made with respect to utilizing the Monte Carlo Simulation method, including the volatility of the underlying shares and the drift rate, or estimated cost of equity. The expected share price volatility for such market-based performance stock option awards was estimated by taking the median historical price volatility for industry peers over the contractual term of the options. The drift rate, or estimated cost of equity, for such market-based performance stock option awards is based on various financial and risk-associated metrics of industry peers, as well as estimated factors specific to us. The Company accounts for stock-based payments to nonemployees issued in exchange for services in accordance with ASU No. 2018-07, " Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting " based upon the fair value of the equity instruments issued. Compensation expense for stock options issued to nonemployees is calculated using the Black-Scholes option pricing model on the grant date and is recorded over the service performance period. |
Net Loss per Share of Common Stock | Net Loss per Share of Common Stock: Basic net loss per share of common stock is computed by dividing the net loss applicable to shareholders of common stock by the weighted-average number of shares of common stock and 3,301,998 pre-funded warrants (see Note 9(B)) outstanding during the period, without further consideration for potentially dilutive securities. In accordance with ASC Topic 260, Earnings Per Share |
Financial Instruments and Fair Value Measurement | Financial Instruments and Fair Value Measurement: The Company utilizes fair value measurement guidance prescribed by accounting standards to value its financial instruments. The guidance establishes a fair value hierarchy for instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and the Company's own assumptions (unobservable inputs). Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company's assumptions about the inputs that market participants would use in pricing the asset or liability and are developed based on the best information available in the circumstances. Fair value is defined as the exchange price, or exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As a basis for considering market participant assumptions in fair value measurements, the guidance establishes a three-tier fair value hierarchy that distinguishes among the following: • Level 1-Valuations are based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access. • Level 2-Valuations are based on quoted prices for similar assets or liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active and models for which all significant inputs are observable, either directly or indirectly. • Level 3-Valuations are based on inputs that are unobservable (supported by little or no market activity) and significant to the overall fair value measurement. To the extent the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument's level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. |
Recently Accounting Pronouncements | Recent Accounting Pronouncements: In February 2016, the FASB issued ASU No. 2016-02, " Topic 842 — Leases " ("Topic 842"), which requires lessees to recognize on their consolidated balance sheets a liability to make lease payments and a right-of-use asset representing their right to use the underlying asset for the lease term for both finance and operating leases with lease terms greater than twelve months. Topic 842 is effective for annual periods beginning after December 15, 2018, and interim periods within fiscal years beginning after December 15, 2018. Early adoption is permitted. A modified retrospective transition approach is required, applying the new standard to all leases existing at the date of initial application. Topic 842 allows entities to choose to use either (i) the effective date or (ii) the beginning of the earliest comparative period presented in the financial statements as the date of initial application. Topic 842 provides a number of optional practical expedients in transition. The Company adopted Topic 842 on April 1, 2019 using the optional modified retrospective transition method. Comparative periods were not restated. For leases that commenced prior to April 1, 2019, the Company elected the following package of practical expedients when assessing the transition impact: (1) not to reassess whether any expired or existing contracts are or contain leases; (2) not to reassess the lease classification for any expired or existing leases; and (3) not to reassess initial direct costs for any existing leases. The Company also elected to: (1) use the total lease term in its initial incremental borrowing rate calculation; (2) combine its lease and non-lease components and account for them as a single lease component; and (3) not apply the use of hindsight in determining the lease term when considering lessee options to extend or terminate the lease and to purchase the underlying asset. Upon adoption, the Company recorded corresponding aggregate operating lease right-of-use assets and operating lease liabilities of $3.0 million and $2.4 million, respectively, including $0.6 million of prepaid rent previously classified within other non-current assets in the Company's consolidated balance sheet that was reclassified to operating lease right-of-use assets. The adoption of the new standard did not materially impact the Company's consolidated results of operations and cash flows and did not have an impact on the Company's beginning accumulated deficit balance. For additional information regarding the Company's leases, see Note 5 for further information regarding the impact of the Company's adoption of Topic 842. In June 2016, the FASB issued ASU No. 2016-13, “ Financial Instruments — Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments " ("ASU No. 2016-13"). ASU 2016-13 requires that financial assets measured at amortized cost, such as loans, accounts and trade receivables and investments, be represented net of expected credit losses, which may be estimated based on relevant information such as historical experience, current conditions, and future expectation for each pool of similar financial asset. ASU No. 2016-13 requires enhanced disclosures related to trade receivables and associated credit losses. In May 2019, the FASB issued ASU No. 2019-05, “ Financial Instruments — Credit Losses (Topic 326): Targeted Transition Relief ", which allows for a transition election on certain instruments and is effective for Small Reporting Companies for fiscal years beginning after December 15, 2022 and interim periods in those fiscal years. In November 2019, the FASB issued ASU No. 2019-11, " Codification Improvements to Topic 326, Financial Instruments — Credit Losses ", which amends certain aspects of ASU NO. 2016-13, including transition relief for trouble debt restructuring, among other topics. While the Company does not expect the adoption of this guidance to materially impact the Company's consolidated financial statements and related disclosures because it does not currently have any investments or trade receivables and the receivable from the sale of a long-term investment outstanding at March 31, 2021 was collected in May 2021, the impact on the Company's consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. In December 2019, the FASB issued ASU No. 2019-12, " Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes " ("ASU No. 2019-12"). ASU No. 2019-12 simplifies the accounting for income taxes, eliminates certain exceptions within Income Taxes (Topic 740) , and clarifies certain aspects of the current guidance to promote consistency among reporting entities, and is effective for fiscal years, and for interim periods within those fiscal years, beginning after December 15, 2020, with early adoption permitted. Most amendments within ASU No. 2019-12 are required to be applied on a prospective basis, while certain amendments must be applied on a retrospective or modified retrospective basis. The Company early adopted the provisions of ASU No. 2019-12 on April 1, 2020, which did not have a material impact on its consolidated financial statements and related disclosures. In August 2020, the FASB issued ASU No. 2020-06, " Debt — Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging — Contracts in Entity's Own Equity (Subtopic 815-40) " ("ASU No. 2020-06"). ASU No. 2020-06 simplifies the accounting for convertible debt instruments by removing the beneficial conversion and cash conversion separation models for convertible instruments. Under ASU No. 2020-06, the embedded conversion features are no longer separated from the host contract for convertible instruments with conversion features that are not required to be accounted for as derivatives or that do not result in substantial premiums accounted for as paid-in capital. ASU No. 2020-06 also amends the accounting for certain contracts in an entity’s own equity that are currently accounted for as derivatives because of specific settlement provisions. In addition, the new guidance modifies how particular convertible instruments and certain contracts that may be settled in cash or shares impact the computation of diluted earnings or loss per share. The provisions of ASU No. 2020-06 are effective for fiscal years beginning after December 15, 2021, and interim periods within those fiscal years, with early adoption permitted, but no earlier than fiscal years beginning after December 15, 2020, including interim periods within those fiscal years. While the Company does not expect the adoption of ASU No. 2020-06 to materially impact the Company's consolidated financial statements and related disclosures because it does not currently maintain any debt instruments accounted for in accordance with ASC Subtopic 470-20, " Debt — Debt with Conversion and Other Options " or instruments accounted for as derivatives in accordance with ASC Subtopic 815-40, " Derivatives and Hedging — Contracts in Entity's Own Equity ", and the Company also currently includes outstanding pre-funded warrants in the computation of basic net loss per share (see Note 2(L)), the impact on the Company's consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. In May 2021, the FASB issued ASU No. 2021-04, " Earnings Per Share (Topic 260), Debt — Modifications and Extinguishments (Subtopic 470-50), Compensation — Stock Compensation (Topic 718), and Derivatives and Hedging — Contracts in Entity’s Own Equity (Subtopic 815-40): Issuer’s Accounting for Certain Modifications or Exchanges of Freestanding Equity-Classified Written Call Options " ("ASU No. 2021-04"). ASU No. 2021-04 provides a principles-based framework for issuers to account for a modification or exchange of freestanding equity-classified written call options. To the extent applicable, issuers first reference other U.S. GAAP to account for the effect of the modification. In the absence of other U.S. GAAP, ASU No. 2021-04 clarifies whether to account for the effect as an adjustment to equity, and the related EPS implications, or as an expense, and if so the manner and pattern of recognition. The provisions of ASU No. 2021-04 are effective for annual periods beginning after December 15, 2021, including interim periods within those fiscal years. Early adoption is permitted. While the Company does not expect the adoption of ASU No. 2021-04 to materially impact the Company's consolidated financial statements and related disclosures because it does not currently anticipate modifications to its outstanding equity-classified written call options, the impact on the Company's consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. |
Foreign Currency | Foreign Currency:The Company has operations in the United States, the United Kingdom, Ireland and Switzerland. The results of its non-U.S. dollar based functional currency operations are translated to U.S. dollars at the average exchange rates during the year. The Company’s assets and liabilities are translated using the current exchange rate as of the balance sheet date and shareholders’ equity is translated using historical rates. Adjustments resulting from the translation of the financial statements of the Company’s foreign functional currency subsidiaries into U.S. dollars are excluded from the determination of net loss and are accumulated in a separate component of shareholders’ equity. Foreign exchange transaction gains and losses are included in other (income) expense in the Company’s results of operations. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Fair Value of Cash Equivalents | The following table summarizes the fair value of the Company's money market fund included in cash equivalents based on the inputs used at March 31, 2021 in determining such values (in thousands): Fair Value Price Quotations (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Money market fund $ 114,000 $ 114,000 $ — $ — |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Mar. 31, 2021 | |
Leases [Abstract] | |
Schedule of Leases Remaining Contractual Rent Obligations | The following table provides a reconciliation of the Company's remaining undiscounted contractual rent obligations due within each respective fiscal year ending March 31 to the operating lease liabilities recognized as of March 31, 2021 (in thousands): Year Ending March 31, Amount 2022 $ 327 2023 272 2024 295 2025 303 2026 311 Thereafter 53 Total undiscounted payments 1,561 Less: present value adjustment (318) Present value of future payments $ 1,243 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Mar. 31, 2021 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Liabilities | As of March 31, 2021 and 2020, accrued expenses consisted of the following (in thousands): March 31, 2021 March 31, 2020 Research and development expenses $ 6,091 $ 6,951 Bonuses and other compensation expenses 2,331 2,521 Legal expenses 15 704 Other expenses 759 1,143 Total accrued expenses $ 9,196 $ 11,319 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Mar. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Schedule of Weighted Average Assumptions Used for Estimating the Fair Value of Stock Options | The Company estimated the fair value of each time-based stock option on the date of grant using the Black-Scholes closed-form option-pricing model applying the weighted average assumptions during the years ended March 31, 2021 and March 31, 2020, as follows: Years Ended March 31, 2021 2020 Expected stock price volatility 109.5 % 78.9 % Expected risk free interest rate 0.43 % 2.30 % Expected term, in years 6.11 6.13 Expected dividend yield — % — % The Company estimated the grant date fair value of each market-based performance stock option granted during the year ended March 31, 2020 using a Monte Carlo Simulation method under the income approach by applying the following assumptions: Expected share price volatility 73.6 - 74.3% Contractual term, in years 10 |
Schedule of Stock Option Activity | The following table presents a summary of stock option activity and data under the 2015 Plan: Number of Options Weighted Average Exercise Price Weighted Average Grant Date Fair Value Weighted Average Remaining Contractual Life (in years) Aggregate Intrinsic Value Options outstanding at March 31, 2019 1,919,158 $ 34.09 $ 49.35 6.86 $ 1,569,809 Granted 1,541,672 8.12 4.96 Exercised (11,778) 7.20 114.40 2,972 Forfeited (1,440,317) 33.99 50.32 Options outstanding at March 31, 2020 2,008,735 $ 14.39 $ 13.87 8.33 $ — Granted 434,775 3.72 3.07 Exercised — — — — Forfeited (347,967) 13.92 15.84 Options outstanding at March 31, 2021 2,095,543 $ 12.26 $ 11.30 7.90 $ — Options vested and expected to vest at March 31, 2021 2,095,543 $ 12.26 $ 11.30 7.90 $ — Options exercisable at March 31, 2021 1,571,191 $ 14.77 $ 13.78 7.59 $ — |
Schedule of Restricted Stock Unit Activity | The following table presents a summary of restricted stock unit activity and data under the 2015 Plan: Number of RSUs Weighted Average Grant Date Fair Value RSUs outstanding at March 31, 2019 — $ — Granted 369,577 7.50 Vested and settled (103,294) 7.77 Forfeited (18,420) 7.77 RSUs outstanding at March 31, 2020 247,863 $ 7.37 Granted 1,247,850 3.37 Vested and settled (187,741) 7.68 Forfeited (281,756) 3.95 RSUs outstanding at March 31, 2021 1,026,216 $ 3.39 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Mar. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Schedule of Components of Income Tax Expense (Benefit) | The loss before income taxes and the related tax expense are as follows (in thousands): Year ended March 31, 2021 Year ended March 31, 2020 Loss before income taxes: United States $ (17,382) $ (4,403) Switzerland (1) (11,978) (62,520) Bermuda (2) (3,177) (5,216) Other (3) (100) (51) Total loss before income taxes $ (32,637) $ (72,190) Current taxes: United States $ (212) $ 403 Switzerland (1) — — Bermuda (2) — — Other (4) — 35 Total current tax expense (212) 438 Deferred taxes: United States — — Switzerland (1) — — Bermuda (2) — — Other (3) — — Total deferred tax expense — — Total income tax expense $ (212) $ 438 (1) Wholly-owned subsidiary, Axovant Sciences GmbH. (2) Bermuda entity was centrally controlled and managed in the United Kingdom. (3) Wholly-owned subsidiaries, Axovant Holdings Limited (United Kingdom) and Sio Europe Limited (Ireland). (4) Includes state income tax expense. |
Reconciliation of Income Tax Benefit to Statutory Rate | A reconciliation of income tax benefit computed at the U.S/Bermuda statutory rate to income tax (benefit) expense reflected in the financial statements is as follows (in thousands): Year Ended Year Ended March 31, 2021 March 31, 2020 $ % $ % Income tax benefit at federal statutory rate $ (6,854) 21.00 % $ — — % Foreign rate differential (1) 1,118 (3.43) (17,268) 23.92 Nondeductible/nontaxable items (1,823) 5.59 766 (1.06) Valuation allowance 10,749 (32.94) 16,759 (23.21) Research and development credit (914) 2.80 (615) 0.85 Research and development true-up (301) 0.92 (285) 0.39 Deferred adjustments 2,139 (6.55) 1,068 (1.48) Restructuring (4,108) 12.59 — — Other (218) 0.67 13 (0.02) Total income tax (benefit) expense $ (212) 0.65 % $ 438 (0.61) % |
Schedule of Deferred Tax Assets and Liabilities | Significant components of the deferred tax assets (liabilities) at March 31, 2021 and 2020 are as follows (in thousands): March 31, 2021 March 31, 2020 Deferred tax assets: Net operating loss $ 154,877 $ 160,319 Research tax credits 11,798 10,782 Stock-based compensation 8,341 10,738 Intangibles 8,747 6,738 Lease liability 257 206 Other 51 78 Subtotal 184,071 188,861 Valuation allowance (183,703) (187,762) Deferred tax liabilities: Right of use (250) (334) Other (118) (765) Total net deferred tax assets $ — $ — |
Selected Quarterly Financial _2
Selected Quarterly Financial Data (Unaudited) (Tables) | 12 Months Ended |
Mar. 31, 2021 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Information | The following table presents selected quarterly financial data for the years ended March 31, 2021 and March 31, 2020 (in thousands, except per share amounts): First Quarter Ended Second Quarter Ended Third Quarter Ended Fourth Quarter Ended First Quarter Ended Second Quarter Ended Third Quarter Ended Fourth Quarter Ended June 30, September 30, December 31, March 31, June 30, September 30, December 31, March 31, 2020 2020 2020 2021 2019 2019 2019 2020 Research and development expenses $ 5,194 $ 5,058 $ 6,407 $ 8,244 $ 21,090 $ 6,833 $ 8,267 $ 10,920 General and administrative expenses 4,640 4,491 4,198 3,965 6,468 5,051 5,409 5,133 Total operating expenses 9,834 9,549 10,605 12,209 27,558 11,884 13,676 16,053 Net loss (8,594) (9,984) (10,516) (3,331) (28,057) (13,884) (14,039) (16,648) Net loss per share attributable to shareholders of common stock - basic and diluted $ (0.20) $ (0.21) $ (0.20) $ (0.05) $ (1.23) $ (0.61) $ (0.62) $ (0.54) |
Description of Business (Detail
Description of Business (Details) | 12 Months Ended |
Mar. 31, 2021segment | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Number of operating segments | 1 |
Number of reporting segments | 1 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Basis of Presentation (Details) | May 08, 2019 | Mar. 31, 2020 |
Class of Stock [Line Items] | ||
Stock split ratio | 0.125 | 0.125 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Going Concern and Management's Plans (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Mar. 31, 2021 | Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2021 | Mar. 31, 2020 | |
Accounting Policies [Abstract] | ||||||||||
Net (loss) income | $ (3,331) | $ (10,516) | $ (9,984) | $ (8,594) | $ (16,648) | $ (14,039) | $ (13,884) | $ (28,057) | $ (32,425) | $ (72,628) |
Cash and cash equivalents | 118,986 | 80,752 | 118,986 | 80,752 | ||||||
(Accumulated deficit) retained earnings | $ (791,069) | $ (758,644) | $ (791,069) | $ (758,644) |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Concentration Risk (Details) | Mar. 31, 2021bank |
Credit Concentration Risk | |
Concentration Risk [Line Items] | |
Number of banking institutions | 2 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Property and Equipment (Details) | 12 Months Ended |
Mar. 31, 2021 | |
Minimum | |
Property, Plant and Equipment [Line Items] | |
Useful life | 3 years |
Maximum | |
Property, Plant and Equipment [Line Items] | |
Useful life | 5 years |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Net Loss per Share of Common Stock (Details) - shares | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Net Loss per Common Share | ||
Pre-funded warrants outstanding (in shares) | 3,301,998 | |
Stock options and warrants | ||
Net Loss per Common Share | ||
Anti-dilutive securities not included in calculation of common shares outstanding (in shares) | 3,100,000 | |
Stock options and RSUs | ||
Net Loss per Common Share | ||
Anti-dilutive securities not included in calculation of common shares outstanding (in shares) | 2,300,000 |
Summary of Significant Accoun_9
Summary of Significant Accounting Policies - Financial Instruments and Fair Value Measurement (Details) $ in Thousands | Mar. 31, 2021USD ($) |
Accounting Policies [Abstract] | |
Money market fund | $ 114,000 |
Summary of Significant Accou_10
Summary of Significant Accounting Policies - Fair Value of Cash Equivalents (Details) $ in Thousands | Mar. 31, 2021USD ($) |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | |
Money market fund | $ 114,000 |
Price Quotations (Level 1) | |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | |
Money market fund | 114,000 |
Significant Other Observable Inputs (Level 2) | |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | |
Money market fund | 0 |
Significant Unobservable Inputs (Level 3) | |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | |
Money market fund | $ 0 |
Summary of Significant Accou_11
Summary of Significant Accounting Policies - Recently Issued Accounting Policies (Details) - USD ($) $ in Thousands | Mar. 31, 2021 | Apr. 01, 2020 | Mar. 31, 2020 |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | $ 1,152 | $ 1,532 | |
Lease liability | 1,243 | ||
Other non-current assets | 0 | $ 46 | |
Accounting Standards Update 2016-02 | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | 3,000 | $ 3,000 | |
Lease liability | $ 2,400 | 2,400 | |
Other non-current assets | $ 600 |
License and Collaboration Agr_2
License and Collaboration Agreements (Details) - USD ($) | Apr. 30, 2019 | Dec. 07, 2018 | Jun. 05, 2018 | Mar. 31, 2021 | Mar. 31, 2020 | Oct. 31, 2019 | Feb. 28, 2019 |
License Agreement [Line Items] | |||||||
Payments for License Agreement | $ 13,000,000 | ||||||
The University of Massachusetts Medical School | |||||||
License Agreement [Line Items] | |||||||
Payments for license and collaboration agreement, development and regulatory milestones, total | $ 24,500,000 | ||||||
Research and development arrangement | $ 1,000,000 | $ 1,000,000 | |||||
Payments for license and collaboration agreement, commercial milestones, total | $ 39,800,000 | ||||||
License agreement costs | $ 6,900,000 | $ 8,800,000 | |||||
Payments for License Agreement | $ 29,000 | 3,000,000 | |||||
License Agreement, Agreement Term After Commercial Sale | 10 years | ||||||
Termination notice term | 90 days | 90 days | |||||
Termination notice term, uncured material breach | 60 days | 60 days | |||||
Oxford BioMedica (UK) Ltd. | |||||||
License Agreement [Line Items] | |||||||
License agreement costs | $ 5,700,000 | 9,200,000 | |||||
Payments for License Agreement | $ 3,500,000 | 15,200,000 | |||||
License Agreement, Agreement Term After Commercial Sale | 10 years | ||||||
Payments for license agreement, developmental milestones | $ 55,000,000 | ||||||
Payments for license agreement, regulatory and sales milestones | $ 757,500,000 | ||||||
License agreement, termination notice prior term | 2 months | ||||||
Oxford BioMedica (UK) Ltd. | Minimum | |||||||
License Agreement [Line Items] | |||||||
Royalty percentage | 7.00% | ||||||
Fee if developmental milestones are not me | $ 500,000 | ||||||
Oxford BioMedica (UK) Ltd. | Maximum | |||||||
License Agreement [Line Items] | |||||||
Royalty percentage | 10.00% | ||||||
Fee if developmental milestones are not me | $ 1,000,000 | ||||||
Benitec Biopharma Limited | |||||||
License Agreement [Line Items] | |||||||
Payments for License Agreement | 2,800,000 | ||||||
Benitec Biopharma Limited | Other than research and development expenses | |||||||
License Agreement [Line Items] | |||||||
License agreement costs | $ 1,800,000 |
Investment in Arvelle Therape_2
Investment in Arvelle Therapeutics B.V (Details) $ / shares in Units, $ in Thousands, shares in Millions | Feb. 25, 2019shares | Feb. 13, 2019USD ($)shares | Mar. 31, 2021USD ($) | Feb. 28, 2021USD ($) | May 31, 2020USD ($)shares | Nov. 12, 2020$ / shares | Mar. 31, 2020USD ($) | Feb. 13, 2019€ / shares |
Equity Securities without Readily Determinable Fair Value [Line Items] | ||||||||
Maximum shares to be purchased under subscription agreement (in shares) | shares | 8.1 | |||||||
Par value (in EUR per share) | $ / shares | $ 0.00001 | |||||||
Number of shares acquired during period (in shares) | shares | 5.9 | 2.2 | ||||||
Long-term investment | $ 5,900 | $ 0 | $ 5,871 | |||||
Arvelle Therapeutics | ||||||||
Equity Securities without Readily Determinable Fair Value [Line Items] | ||||||||
Equity Securities (in shares) | shares | 2.2 | |||||||
Capitalized as a long-term investment | $ 2,200 | |||||||
Proceeds from sale of equity method investments | $ 11,600 | |||||||
Received held in escrow | 1,200 | |||||||
Contingent consideration expected to be received | 7,000 | |||||||
Realized gain (loss) on disposal | $ 4,300 | $ 4,700 | ||||||
Long-term Investee | ||||||||
Equity Securities without Readily Determinable Fair Value [Line Items] | ||||||||
Par value (in EUR per share) | € / shares | € 0.00001 |
Leases - Narrative (Details)
Leases - Narrative (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Mar. 31, 2021 | Mar. 31, 2020 | Aug. 31, 2020 | Apr. 01, 2020 | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Operating lease right-of-use assets | $ 1,152 | $ 1,532 | ||
Lease liability | $ 1,243 | |||
Lessee, operating lease, discount rate | 12.90% | |||
Payment term | 5 years | |||
Operating lease, weighted average remaining lease term | 5 years 1 month 6 days | |||
Operating lease, weighted average discount rate, percent | 9.20% | |||
Rent expense | $ 1,600 | 1,800 | ||
Payments for contractual rent obligations | 900 | $ 1,800 | ||
Office Lease Facility In New York New York | ||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Operating lease right-of-use assets | $ 1,100 | |||
Lessee, operating lease, discount rate | 8.90% | |||
Accounting Standards Update 2016-02 | ||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Operating lease right-of-use assets | 3,000 | $ 3,000 | ||
Lease liability | 2,400 | $ 2,400 | ||
Prepaid rent | $ 600 |
Leases - Remaining Contractual
Leases - Remaining Contractual Rent Obligations (Details) $ in Thousands | Mar. 31, 2021USD ($) |
Leases [Abstract] | |
2021 | $ 327 |
2023 | 272 |
2024 | 295 |
2025 | 303 |
2026 | 311 |
Thereafter | 53 |
Total undiscounted payments | 1,561 |
Less: present value adjustment | (318) |
Present value of future payments | $ 1,243 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Mar. 31, 2021 | Mar. 31, 2020 |
Payables and Accruals [Abstract] | ||
Research and development expenses | $ 6,091 | $ 6,951 |
Bonuses and other compensation expenses | 2,331 | 2,521 |
Legal expenses | 15 | 704 |
Other expenses | 759 | 1,143 |
Total accrued expenses | $ 9,196 | $ 11,319 |
Long Term Debt (Details)
Long Term Debt (Details) - USD ($) $ in Millions | Nov. 27, 2019 | Feb. 02, 2017 | Apr. 30, 2020 | Mar. 31, 2021 |
Debt Instrument [Line Items] | ||||
Accrued interest, fees and other amounts | $ 0.3 | |||
Extinguishment of debt, amount | $ 0.5 | |||
Term Loan | Loan and Security Agreement with Hercules Capital, Inc. | ||||
Debt Instrument [Line Items] | ||||
Amount borrowed | $ 55 | |||
Debt instrument, redemption price, percentage of principal amount redeemed | 50.00% | |||
Repayments of debt | $ 15.7 | $ 15.7 | ||
Term Loan | Loan and Security Agreement with Hercules Capital, Inc. | Minimum | ||||
Debt Instrument [Line Items] | ||||
Fixed interest rate | 11.55% | 10.55% | ||
Term Loan | Loan and Security Agreement with Hercules Capital, Inc. | Prime Rate | ||||
Debt Instrument [Line Items] | ||||
Variable interest rate | 6.80% | 6.80% |
Related Party Transactions (Det
Related Party Transactions (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 12 Months Ended | |
Feb. 29, 2020 | Mar. 31, 2021 | Mar. 31, 2020 | |
Public Offering | |||
Related Party Transaction [Line Items] | |||
Number of shares issued and sold (in shares) | 16,631,336 | ||
Warrants issued (in shares) | 3,301,998 | ||
Price of shares issued and sold (in dollars per share) | $ 3.75 | ||
Warrants issued (in dollars per share) | $ 3.74999 | ||
Net proceeds from common stock issued | $ 70.8 | ||
Public Offering | Roivant Sciences, Ltd. | |||
Related Party Transaction [Line Items] | |||
Number of shares issued and sold (in shares) | 5,333,333 | ||
Underwriter's Option | |||
Related Party Transaction [Line Items] | |||
Number of shares issued and sold (in shares) | 2,600,000 | ||
RSI | |||
Related Party Transaction [Line Items] | |||
Expense under service agreement | $ 0.1 | $ 0.1 |
Stockholders_ Equity (Details)
Stockholders’ Equity (Details) $ / shares in Units, $ in Thousands | May 08, 2019 | Feb. 29, 2020USD ($)$ / sharesshares | Jun. 09, 2021USD ($)shares | Mar. 31, 2020$ / sharesshares | Mar. 31, 2021USD ($)$ / sharesshares | Mar. 31, 2020USD ($)$ / sharesshares | Nov. 12, 2020$ / sharesshares | Mar. 31, 2019shares |
Class of Stock [Line Items] | ||||||||
Common shares authorized (in shares) | 1,000,000,000 | 1,000,000,000 | 1,000,000,000 | 1,010,000,000 | ||||
Common stock issued (in shares) | 39,526,299 | 69,377,567 | 39,526,299 | 1,000,000,000 | ||||
Common shares par value (in dollars per share) | $ / shares | $ 0.00001 | $ 0.00001 | $ 0.00001 | $ 0.00001 | ||||
Preferred stock authorized (in dollars per share) | 10,000,000 | |||||||
Par value (in EUR per share) | $ / shares | $ 0.00001 | |||||||
Stock split ratio | 0.125 | 0.125 | ||||||
Capital contribution | $ | $ 121 | $ 148 | ||||||
Public Offering | ||||||||
Class of Stock [Line Items] | ||||||||
Common stock issued during period (in shares) | 16,631,336 | |||||||
Warrants issued (in shares) | 3,301,998 | |||||||
Common stock issued (in dollars per share) | $ / shares | $ 3.75 | |||||||
Warrants issued (in dollars per share) | $ / shares | $ 3.74999 | |||||||
Net proceeds from common stock issued | $ | $ 70,800 | |||||||
Percent ownership threshold to no longer exercise | 9.99% | |||||||
Underwriter's Option | ||||||||
Class of Stock [Line Items] | ||||||||
Common stock issued during period (in shares) | 2,600,000 | |||||||
Private Placement | ||||||||
Class of Stock [Line Items] | ||||||||
Percentage of gross proceeds from common stock issuance paid for services | 3.00% | |||||||
Roivant Sciences, Ltd. | Public Offering | ||||||||
Class of Stock [Line Items] | ||||||||
Common stock issued during period (in shares) | 5,333,333 | |||||||
SVB Leerink LLC | Private Placement | ||||||||
Class of Stock [Line Items] | ||||||||
Common stock issued during period (in shares) | 29,700,000 | |||||||
Net proceeds from common stock issued | $ | $ 90,500 | |||||||
SVB Leerink LLC | Private Placement | Subsequent Event | ||||||||
Class of Stock [Line Items] | ||||||||
Common stock issued during period (in shares) | 200,000 | |||||||
Net proceeds from common stock issued | $ | $ 500 | |||||||
RSL | ||||||||
Class of Stock [Line Items] | ||||||||
Capital contribution | $ | $ 100 | $ 100 | ||||||
Common Stock | ||||||||
Class of Stock [Line Items] | ||||||||
Shares outstanding before split (in shares) | 182,200,000 | |||||||
Shares outstanding (in shares) | 39,526,299 | 69,377,567 | 39,526,299 | 22,779,891 |
Stock-Based Compensation - Narr
Stock-Based Compensation - Narrative (Details) $ / shares in Units, $ in Millions | May 08, 2019 | Sep. 30, 2019shares | Mar. 31, 2020$ / sharesshares | Mar. 31, 2021USD ($)installment$ / sharesshares | Mar. 31, 2020USD ($)$ / sharesshares | Apr. 30, 2020shares | Apr. 30, 2019shares | Mar. 31, 2019$ / sharesshares |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Stock split ratio | 0.125 | 0.125 | ||||||
Number of options outstanding (in shares) | 2,008,735 | 2,095,543 | 2,008,735 | 1,919,158 | ||||
Number of options granted (in shares) | 434,775 | 1,541,672 | ||||||
Options granted, weighted average exercise price (in dollars per share) | $ / shares | $ 3.72 | $ 8.12 | ||||||
Exercise price of options (in dollars per share) | $ / shares | $ 14.39 | $ 12.26 | $ 14.39 | $ 34.09 | ||||
Fair value of options vested | $ | $ 3.5 | $ 3.8 | ||||||
Grants To Directors And Employees | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Share-based compensation expense | $ | 4.4 | 7 | ||||||
Unrecognized compensation expense related to options | $ | $ 6.2 | |||||||
Remaining weighted-average service period | 2 years 1 month 28 days | |||||||
Employees | RSL | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Share-based compensation expense | $ | $ 0.1 | $ 0.9 | ||||||
Time-Based Stock Options | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Vesting period | 4 years | |||||||
Vesting percentage | 25.00% | |||||||
Number of installments | installment | 12 | |||||||
Number of options vested (in shares) | 900,000 | |||||||
Market-Based Performance Options | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Number of options outstanding (in shares) | 400,000 | |||||||
Number of options granted (in shares) | 400,000 | |||||||
Options granted, weighted average exercise price (in dollars per share) | $ / shares | $ 8.07 | |||||||
Options granted, estimated grant date fair value | $ | $ 1.2 | |||||||
Exercise price of options (in dollars per share) | $ / shares | $ 8.87 | |||||||
Number of options vested (in shares) | 0 | |||||||
Restricted Stock Units (RSUs) | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Vesting percentage | 33.33% | 33.33% | ||||||
Number of installments | installment | 3 | |||||||
Number of shares from vesting (in shares) | 300,000 | |||||||
Fair value of awards vested | $ | $ 1 | $ 1.2 | ||||||
Outstanding unvested (in shares) | 247,863 | 1,026,216 | 247,863 | 0 | ||||
Restricted Stock Units (RSUs) | Vested January 31, 2020 | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Vesting percentage | 50.00% | |||||||
Restricted Stock Units (RSUs) | Vested July 31, 2020 | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Vesting percentage | 50.00% | |||||||
2015 Equity Incentive Plan | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Shares authorized for issuance before split (in shares) | 24,800,000 | |||||||
Shares authorized for issuance (in shares) | 5,600,000 | 1,600,000 | 900,000 | 3,100,000 | ||||
Number of shares available for future issuance (in shares) | 8,400,000 | |||||||
Number of shares available for future issuance (in shares) | 2,000,000 | 1,000,000 | ||||||
Number of options outstanding, before stock split (in shares) | 15,400,000 |
Stock-Based Compensation - Stoc
Stock-Based Compensation - Stock Option, Fair Value Assumptions (Details) | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Time-Based Stock Options | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected stock price volatility | 109.50% | 78.90% |
Expected risk free interest rate | 0.43% | 2.30% |
Expected term, in years | 6 years 1 month 9 days | 6 years 1 month 17 days |
Expected dividend yield | 0.00% | 0.00% |
Market-Based Performance Options | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term, in years | 10 years | |
Market-Based Performance Options | Minimum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected stock price volatility | 73.60% | |
Market-Based Performance Options | Maximum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected stock price volatility | 74.30% |
Stock-Based Compensation - St_2
Stock-Based Compensation - Stock Option Activity (Details) - USD ($) | 12 Months Ended | ||
Mar. 31, 2021 | Mar. 31, 2020 | Mar. 31, 2019 | |
Number of Options | |||
Beginning balance (in shares) | 2,008,735 | 1,919,158 | |
Granted (in shares) | 434,775 | 1,541,672 | |
Exercised (in shares) | 0 | (11,778) | |
Forfeited (in shares) | (347,967) | (1,440,317) | |
Ending balance (in shares) | 2,095,543 | 2,008,735 | 1,919,158 |
Weighted Average Exercise Price | |||
Beginning balance (in dollars per share) | $ 14.39 | $ 34.09 | |
Granted (in dollars per share) | 3.72 | 8.12 | |
Exercised (in dollars per share) | 0 | 7.20 | |
Forfeited (in dollars per share) | 13.92 | 33.99 | |
Ending balance (in dollars per share) | 12.26 | 14.39 | $ 34.09 |
Weighted Average Grant Date Fair Value | |||
Beginning balance (in dollars per share) | 13.87 | 49.35 | |
Granted (in dollars per share) | 3.07 | 4.96 | |
Exercised (in dollars per share) | 0 | 114.40 | |
Forfeited (in dollars per share) | 15.84 | 50.32 | |
Ending balance (in dollars per share) | $ 11.30 | $ 13.87 | $ 49.35 |
Additional Disclosures | |||
Options outstanding, weighted average contractual term | 7 years 10 months 24 days | 8 years 3 months 29 days | 6 years 10 months 9 days |
Options outstanding, aggregate intrinsic value | $ 0 | $ 0 | $ 1,569,809 |
Options granted, aggregate intrinsic value | $ 0 | $ 2,972 | |
Options exercisable, number of options (in shares) | 1,571,191 | ||
Options exercisable, weighted average exercise price (in dollars per share) | $ 14.77 | ||
Options exercisable, weighted average grant date fair value (in dollars per share) | $ 13.78 | ||
Options exercisable, weighted average contractual term | 7 years 7 months 2 days | ||
Options exercisable, aggregate intrinsic value | $ 0 | ||
Options Vested and Expected to Vest | |||
Number of options (in shares) | 2,095,543 | ||
Weighted average exercise price (in dollars per share) | $ 12.26 | ||
Weighted average grant date fair value (in dollars per share) | $ 11.30 | ||
Weighted Average Remaining Contractual Life (in years) | 7 years 10 months 24 days | ||
Aggregate Intrinsic Value | $ 0 |
Stock-Based Compensation - Rest
Stock-Based Compensation - Restricted Stock Unit Activity (Details) - Restricted Stock Units (RSUs) - $ / shares | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Number of RSUs | ||
Beginning balance (in shares) | 247,863 | 0 |
Granted (in shares) | 1,247,850 | 369,577 |
Vested and settled (in shares) | (187,741) | (103,294) |
Forfeited (in shares) | (281,756) | (18,420) |
Ending balance (in shares) | 1,026,216 | 247,863 |
Weighted Average Grant Date Fair Value | ||
Beginning balance (in dollars per share) | $ 7.37 | $ 0 |
Granted (in dollars per share) | 3.37 | 7.50 |
Vested and settled (in dollars per share) | 7.68 | 7.77 |
Forfeited (in dollars per share) | 3.95 | 7.77 |
Ending balance (in dollars per share) | $ 3.39 | $ 7.37 |
Income Taxes- Summary of Income
Income Taxes- Summary of Income Tax Expense by Jurisdiction (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Loss before income taxes: | ||
Total loss before income taxes | $ (32,637) | $ (72,190) |
Current taxes: | ||
Total current tax expense | (212) | 438 |
Deferred taxes: | ||
Bermuda | 0 | 0 |
Total deferred tax expense | 0 | 0 |
Total income tax (benefit) expense | (212) | 438 |
United States | ||
Loss before income taxes: | ||
Foreign | (17,382) | (4,403) |
Current taxes: | ||
Foreign | (212) | 403 |
Deferred taxes: | ||
Foreign | 0 | 0 |
Switzerland | ||
Loss before income taxes: | ||
Foreign | (11,978) | (62,520) |
Current taxes: | ||
Foreign | 0 | 0 |
Deferred taxes: | ||
Foreign | 0 | 0 |
Bermuda | ||
Loss before income taxes: | ||
Bermuda | (3,177) | (5,216) |
Current taxes: | ||
Bermuda | 0 | 0 |
Other | ||
Loss before income taxes: | ||
Foreign | (100) | (51) |
Current taxes: | ||
Foreign | 0 | 35 |
Deferred taxes: | ||
Foreign | $ 0 | $ 0 |
Income Taxes - Reconciliation o
Income Taxes - Reconciliation of Income Tax Benefit to Statutory Rate (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
$ | ||
Income tax benefit at federal statutory rate | $ (6,854) | $ 0 |
Foreign rate differential | 1,118 | (17,268) |
Nondeductible/nontaxable items | (1,823) | 766 |
Valuation allowance | 10,749 | 16,759 |
Research and development credit | (914) | (615) |
Research and development true-up | (301) | (285) |
Deferred adjustments | 2,139 | 1,068 |
Restructuring | (4,108) | 0 |
Other | (218) | 13 |
Total income tax (benefit) expense | $ (212) | $ 438 |
% | ||
Income tax benefit at federal statutory rate | 21.00% | 0.00% |
Foreign rate differential | (3.43%) | 23.92% |
Nondeductible/nontaxable items | 5.59% | (1.06%) |
Valuation allowance | (32.94%) | (23.21%) |
Research and development credit | 2.80% | 0.85% |
Research and development true-up | 0.92% | 0.39% |
Deferred adjustments | (6.55%) | (1.48%) |
Restructuring | 12.59% | 0.00% |
Other | 0.67% | (0.02%) |
Total income tax (benefit) expense | 0.65% | (0.61%) |
Income Taxes- Narrative (Detail
Income Taxes- Narrative (Details) - USD ($) | 12 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Operating Loss Carryforwards [Line Items] | ||
Effective income tax rate | 0.65% | (0.61%) |
Income tax receivable | $ 1,656,000 | $ 1,707,000 |
Valuation allowance on deferred tax assets | 183,703,000 | $ 187,762,000 |
Unrecognized tax benefits | $ 0 | |
Bermuda | ||
Operating Loss Carryforwards [Line Items] | ||
Effective income tax rate | 0.65% | (0.61%) |
United States | ||
Operating Loss Carryforwards [Line Items] | ||
Net operating losses | $ 3,200,000 | |
SWITZERLAND | ||
Operating Loss Carryforwards [Line Items] | ||
Net operating losses | 1,180,000,000 | |
United Kingdom | ||
Operating Loss Carryforwards [Line Items] | ||
Net operating losses | 150,000 | |
Ireland | ||
Operating Loss Carryforwards [Line Items] | ||
Net operating losses | 77,000 | |
Research and Development Tax Credit | ||
Operating Loss Carryforwards [Line Items] | ||
Tax credit carryforward | $ 11,800,000 |
Income Taxes- Schedule of Defer
Income Taxes- Schedule of Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2021 | Mar. 31, 2020 |
Deferred tax assets: | ||
Net operating loss | $ 154,877 | $ 160,319 |
Research tax credits | 11,798 | 10,782 |
Stock-based compensation | 8,341 | 10,738 |
Intangibles | 8,747 | 6,738 |
Lease liability | 257 | 206 |
Other | 51 | 78 |
Subtotal | 184,071 | 188,861 |
Valuation allowance | (183,703) | (187,762) |
Deferred tax liabilities: | ||
Right of use | (250) | (334) |
Other | (118) | (765) |
Total net deferred tax assets | $ 0 | $ 0 |
Commitments and Contingencies (
Commitments and Contingencies (Details) | Dec. 07, 2018 | Mar. 31, 2021 |
Third Parties for Pharmaceutical Manufacturing and Research Activities | Minimum | ||
Other Commitments [Line Items] | ||
Termination notice term | 30 days | |
Third Parties for Pharmaceutical Manufacturing and Research Activities | Maximum | ||
Other Commitments [Line Items] | ||
Termination notice term | 60 days | |
The University of Massachusetts Medical School | ||
Other Commitments [Line Items] | ||
Termination notice term | 90 days | 90 days |
Termination notice term, uncured material breach | 60 days | 60 days |
Oxford BioMedica (UK) Ltd. | ||
Other Commitments [Line Items] | ||
License agreement, termination notice prior term | 2 months |
Selected Quarterly Financial _3
Selected Quarterly Financial Data (Unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Mar. 31, 2021 | Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2021 | Mar. 31, 2020 | |
Quarterly Financial Information Disclosure [Abstract] | ||||||||||
Research and development expenses | $ 8,244 | $ 6,407 | $ 5,058 | $ 5,194 | $ 10,920 | $ 8,267 | $ 6,833 | $ 21,090 | $ 24,903 | $ 47,110 |
General and administrative expenses | 3,965 | 4,198 | 4,491 | 4,640 | 5,133 | 5,409 | 5,051 | 6,468 | 17,294 | 22,061 |
Total operating expenses | 12,209 | 10,605 | 9,549 | 9,834 | 16,053 | 13,676 | 11,884 | 27,558 | 42,197 | 69,171 |
Net loss | $ (3,331) | $ (10,516) | $ (9,984) | $ (8,594) | $ (16,648) | $ (14,039) | $ (13,884) | $ (28,057) | $ (32,425) | $ (72,628) |
Net loss per share attributable to shareholders of common stock - basic and diluted (in dollars per share) | $ (0.05) | $ (0.20) | $ (0.21) | $ (0.20) | $ (0.54) | $ (0.62) | $ (0.61) | $ (1.23) | $ (0.62) | $ (2.93) |
Subsequent Events (Details)
Subsequent Events (Details) - Private Placement - SVB Leerink LLC - USD ($) shares in Millions, $ in Millions | 2 Months Ended | 12 Months Ended |
Jun. 09, 2021 | Mar. 31, 2021 | |
Subsequent Event [Line Items] | ||
Common stock issued during period (in shares) | 29.7 | |
Net proceeds from common stock issued | $ 90.5 | |
Subsequent Event | ||
Subsequent Event [Line Items] | ||
Common stock issued during period (in shares) | 0.2 | |
Net proceeds from common stock issued | $ 0.5 |