Cover
Cover - shares | 3 Months Ended | |
Jun. 30, 2021 | Aug. 10, 2021 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Jun. 30, 2021 | |
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 $0.00001 per share | |
Trading Symbol | SIOX | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 72,941,507 | |
Entity Central Index Key | 0001636050 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --03-31 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q1 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Jun. 30, 2021 | Mar. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 110,976 | $ 118,986 |
Receivable from sale of long-term investment | 0 | 4,343 |
Prepaid expenses and other current assets | 6,276 | 7,348 |
Income tax receivable | 1,681 | 1,656 |
Total current assets | 118,933 | 132,333 |
Long-term restricted cash | 1,184 | 1,184 |
Operating lease right-of-use assets | 1,103 | 1,152 |
Property and equipment, net | 595 | 478 |
Total assets | 121,815 | 135,147 |
Current liabilities: | ||
Accounts payable | 937 | 1,341 |
Accrued expenses | 6,389 | 9,196 |
Current portion of operating lease liabilities | 269 | 311 |
Total current liabilities | 7,595 | 10,848 |
Operating lease liabilities, net of current portion | 921 | 932 |
Total liabilities | 8,516 | 11,780 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Common stock, par value $0.00001 per share, 1,000,000,000 shares authorized, 69,639,509 and 69,377,567 issued and outstanding at June 30, 2021 and March 31, 2021, respectively | 1 | 1 |
Additional paid-in capital | 915,900 | 914,100 |
Accumulated deficit | (802,939) | (791,069) |
Accumulated other comprehensive income | 337 | 335 |
Total stockholders’ equity | 113,299 | 123,367 |
Total liabilities and stockholders’ equity | $ 121,815 | $ 135,147 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 2021 | Mar. 31, 2021 |
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) | 69,639,509 | 69,377,567 |
Common shares outstanding (in shares) | 69,639,509 | 69,377,567 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Operating expenses: | ||
Research and development expenses | $ 8,058 | $ 5,194 |
General and administrative expenses | 3,859 | 4,640 |
Total operating expenses | 11,917 | 9,834 |
Other (income) expenses: | ||
Interest expense | 1 | 796 |
Other income | (20) | (2,066) |
Loss before income tax (benefit) expense | (11,898) | (8,564) |
Income tax (benefit) expense | (28) | 30 |
Net loss | $ (11,870) | $ (8,594) |
Net loss per share of common stock — basic (in dollars per share) | $ (0.16) | $ (0.20) |
Net loss per share of common stock — diluted (in dollars per share) | $ (0.16) | $ (0.20) |
Weighted-average shares of common stock outstanding — basic (in shares) | 72,861,870 | 43,287,222 |
Weighted-average shares of common stock outstanding — diluted (in shares) | 72,861,870 | 43,287,222 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Operations (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Research and development expenses | ||
Share-based compensation expense | $ 432 | $ 563 |
General and administrative expenses | ||
Share-based compensation expense | $ 889 | $ 1,027 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (11,870) | $ (8,594) |
Other comprehensive income: | ||
Foreign currency translation adjustment | 2 | 44 |
Total other comprehensive income | 2 | 44 |
Comprehensive loss | $ (11,868) | $ (8,550) |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Stockholders’ Equity - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) |
Beginning balance (in shares) at Mar. 31, 2020 | 39,526,299 | ||||
Beginning balance at Mar. 31, 2020 | $ 61,558 | $ 0 | $ 820,257 | $ (758,644) | $ (55) |
Increase (Decrease) in Shareholders' Equity (Deficit) | |||||
Shares issued upon settlement of restricted stock units (in shares) | 53,653 | ||||
Shares sold in connection with at-the-market offering, net of brokerage fees and offering expenses (in shares) | 1,393,428 | ||||
Shares sold in connection with at-the-market offering, net of brokerage fees and offering expenses of $0.5 million | 3,930 | 3,930 | |||
Stock-based compensation expense | 1,590 | 1,590 | |||
Capital contribution received from affiliate | 53 | 53 | |||
Foreign currency translation adjustment | 44 | 44 | |||
Net loss | (8,594) | (8,594) | |||
Ending balance (in shares) at Jun. 30, 2020 | 40,973,380 | ||||
Ending balance at Jun. 30, 2020 | 58,581 | $ 0 | 825,830 | (767,238) | (11) |
Beginning balance (in shares) at Mar. 31, 2020 | 39,526,299 | ||||
Beginning balance at Mar. 31, 2020 | 61,558 | $ 0 | 820,257 | (758,644) | (55) |
Increase (Decrease) in Shareholders' Equity (Deficit) | |||||
Net loss | (32,400) | ||||
Ending balance (in shares) at Mar. 31, 2021 | 69,377,567,000 | ||||
Ending balance at Mar. 31, 2021 | 123,367 | $ 1 | 914,100 | (791,069) | 335 |
Increase (Decrease) in Shareholders' Equity (Deficit) | |||||
Shares issued upon settlement of restricted stock units (in shares) | 82,542,000 | ||||
Shares sold in connection with at-the-market offering, net of brokerage fees and offering expenses (in shares) | 179,400,000 | ||||
Shares sold in connection with at-the-market offering, net of brokerage fees and offering expenses of $0.5 million | 479 | 479 | |||
Stock-based compensation expense | 1,321 | 1,321 | |||
Foreign currency translation adjustment | 2 | 2 | |||
Net loss | (11,870) | (11,870) | |||
Ending balance (in shares) at Jun. 30, 2021 | 69,639,509,000 | ||||
Ending balance at Jun. 30, 2021 | $ 113,299 | $ 1 | $ 915,900 | $ (802,939) | $ 337 |
Condensed Consolidated Statem_5
Condensed Consolidated Statements of Stockholders’ Equity (Parenthetical) - USD ($) $ in Millions | 3 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Statement of Stockholders' Equity [Abstract] | ||
Brokerage fees and offering expenses | $ 0 | $ 0.5 |
Condensed Consolidated Statem_6
Condensed Consolidated Statement of Cash Flows - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | Mar. 31, 2021 | |
Cash flows from operating activities: | |||
Net loss | $ (11,870) | $ (8,594) | $ (32,400) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Unrealized foreign currency translation adjustment | 2 | 44 | |
Amortization of operating lease right-of-use assets | 49 | 427 | |
Stock-based compensation expense | 1,321 | 1,590 | |
Depreciation and non-cash amortization | 61 | 492 | |
Gain on long-term investment | 0 | (2,184) | |
Change in operating lease liabilities | (53) | (432) | |
Other | 2 | 3 | |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other current assets | 1,072 | (1,027) | |
Income tax receivable | (25) | (10) | |
Accounts payable | (404) | (1,442) | |
Accrued expenses | (2,807) | (2,370) | |
Net cash used in operating activities | (12,652) | (13,503) | |
Cash flows from investing activities: | |||
Cash proceeds from sale of long-term investment | 4,343 | 0 | |
Purchases of property and equipment | (180) | (19) | |
Net cash provided by (used in) investing activities | 4,163 | (19) | |
Cash flows from financing activities: | |||
Payments on long-term debt | 0 | (15,731) | |
Capital contributions received from affiliate | 0 | 53 | |
Cash proceeds from issuance of shares of common stock, net of issuance costs | 479 | 3,930 | |
Net cash provided by (used in) financing activities | 479 | (11,748) | |
Net change in cash and cash equivalents and long-term restricted cash | (8,010) | (25,270) | |
Total cash and cash equivalents and long-term restricted cash—beginning of period | 120,170 | 80,752 | 80,752 |
Total cash and cash equivalents and long-term restricted cash—end of period | 112,160 | 55,482 | 120,170 |
Cash and cash equivalents —beginning of period | 118,986 | 80,752 | 80,752 |
Restricted cash included in long-term assets—beginning of period | 1,184 | 0 | 0 |
Cash and cash equivalents—end of period | 110,976 | 55,482 | 118,986 |
Restricted cash included in long-term assets—end of period | $ 1,184 | $ 0 | $ 1,184 |
Description of Business
Description of Business | 3 Months Ended |
Jun. 30, 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 | 3 Months Ended |
Jun. 30, 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 unaudited condensed consolidated financial statements and accompanying notes have been prepared in accordance with accounting principles generally accepted in the United States ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. These unaudited condensed consolidated financial statements and accompanying notes should be read in conjunction with the Company’s audited consolidated financial statements and notes thereto included in the Company’s Annual Report on Form 10-K for the fiscal year ended March 31, 2021 (the "Annual Report"), filed with the SEC on June 9, 2021. In the opinion of management, all adjustments (consisting of normal recurring adjustments) considered necessary to present fairly the financial position of the Company and its results of operations and cash flows for the periods presented have been included. Operating results for the three months ended June 30, 2021 are not necessarily indicative of the results that may be expected for the year ending March 31, 2022, for any other interim period, or for any other future year. 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 unaudited condensed 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 unaudited condensed 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. There have been no significant changes in the Company’s accounting policies from those disclosed in its Annual Report. (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 three months ended June 30, 2021 and the fiscal year ended March 31, 2021, the Company incurred net losses of $11.9 million and $32.4 million, respectively. As of June 30, 2021, the Company’s cash and cash equivalents totaled $111.0 million and its accumulated deficit was $802.9 million. The Company estimates that its current cash and cash equivalents balance is sufficient to support operations beyond the twelve month period following the date that these unaudited condensed 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 does not have substantial doubt about its ability to continue as a going concern for the one-year period following the date that these unaudited condensed 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 June 30, 2021 and through the date of issuance of these unaudited condensed 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) 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 7(B) and Note 10) 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 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. Restricted Stock Units ("RSUs") and stock options outstanding for a total of 5.6 million and 3.2 million shares of common stock were not included in the calculation of diluted weighted-average shares of common stock outstanding for the three months ended June 30, 2021 and June 30, 2020, respectively, because they were anti-dilutive given the net loss of the Company. (E) 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 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 is stated at its historical carrying amount, which approximates fair value due to its short-term nature. Long-term restricted cash is stated at its historical carrying amount, which approximates fair value. The carrying values of the Company's money market fund included in cash and cash equivalents of $107.0 million and $114.0 million at June 30, 2021 and March 31, 2021, respectively, approximated their fair values, which are 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 June 30, 2021 and March 31, 2021 in determining such values (in thousands): As of June 30, 2021 As of March 31, 2021 Fair Value Price Quotations (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Fair Value Price Quotations (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Money market fund $ 107,000 $ 107,000 $ — $ — $ 114,000 $ 114,000 $ — $ — (F) Recent Accounting Pronouncements: 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 outstanding, 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. |
License and Collaboration Agree
License and Collaboration Agreements | 3 Months Ended |
Jun. 30, 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. The Company incurred a total of $3.4 million and $0.4 million of program-specific costs related to its AXO-AAV-GM1 and AXO-AAV-GM2 programs within research and development expenses in its unaudited condensed consolidated statements of operations during the three months ended June 30, 2021 and June 30, 2020, respectively. The Company paid a total of $1.6 million and zero to UMMS during the three months ended June 30, 2021 and June 30, 2020, respectively. (B) Oxford Biomedica License Agreement: In June 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. The Company incurred $0.6 million and $1.7 million of AXO-Lenti-PD program-specific costs within research and development expenses in its unaudited condensed consolidated statement of operations during the three months ended June 30, 2021 and June 30, 2020, respectively. The Company paid Oxford a total of $22 thousand and $0.5 million during the three months ended June 30, 2021 and June 30, 2020, respectively. |
Investment in Arvelle Therapeut
Investment in Arvelle Therapeutics B.V. | 3 Months Ended |
Jun. 30, 2021 | |
Investments, All Other Investments [Abstract] | |
Investment in Arvelle Therapeutics B.V. | Investment in Arvelle Therapeutics B.V.In February 2021, the Company sold its investment of 8.1 million shares of nonredeemable preferred stock (the "Arvelle Shares") of Arvelle Therapeutics B.V. ("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 unaudited condensed consolidated balance sheet at June 30, 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 originally purchased its Arvelle Shares in February 2019 and May 2020 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 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 that was collected during the three months ended June 30, 2021. |
Accrued Expenses
Accrued Expenses | 3 Months Ended |
Jun. 30, 2021 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | Accrued Expenses As of June 30, 2021, and March 31, 2021, accrued expenses consisted of the following (in thousands): June 30, 2021 March 31, 2021 Research and development expenses $ 4,336 $ 6,091 Bonuses and other compensation expenses 1,253 2,331 Other expenses 800 774 Total accrued expenses $ 6,389 $ 9,196 |
Long Term Debt
Long Term Debt | 3 Months Ended |
Jun. 30, 2021 | |
Debt Disclosure [Abstract] | |
Long Term Debt | Long-term DebtIn April 2020, the Company fully prepaid $15.7 million of outstanding principal, together with $0.3 million of accrued interest, fees and other amounts, due under its loan and security agreement (the "Loan Agreement") with Hercules Capital, Inc. ("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 three months ended June 30, 2020. 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. |
Stockholders' Equity
Stockholders' Equity | 3 Months Ended |
Jun. 30, 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 (see Note 1). (B) Transactions: In February 2020, as part of a follow-on public offering, the Company issued and sold pre-funded warrants to purchase up to 3,301,998 shares of common stock at an offering price of $3.74999 and an exercise price of $0.00001 per pre-funded warrant, which were fully exercised in July 2021 (see Note 10). The pre-funded warrants did not expire and were immediately exercisable except that the pre-funded warrants could not be exercised by the holder if, after giving effect thereto, the holder would have beneficially owned more than 9.99% of the Company’s common stock, subject to certain exceptions. The pre-funded warrants were 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 was not subject to remeasurement. The Company has 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. During the three months ended June 30, 2020, which was the inception of this program, the Company sold approximately 1.4 million shares of its common stock for total proceeds of approximately $4.3 million, net of brokerage fees. During the three months ended June 30, 2021, the Company sold approximately 0.2 million shares of its common stock for total proceeds of approximately $0.5 million, net of brokerage fees, under this program. As of June 30, 2021, the Company sold a total of approximately 29.8 million shares of its common stock for aggregate proceeds of approximately $90.9 million, net of brokerage fees, under and since the inception of this program. |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Jun. 30, 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 its Board of Directors in October 2020 (the "2015 Plan"). In April 2021 and April 2020, the number of shares of common stock authorized for issuance under the 2015 Plan increased automatically by 2.8 million and 1.6 million, respectively, in accordance with the terms of the 2015 Plan. At June 30, 2021, totals of 8.4 million shares of common stock were authorized for issuance and 2.3 million shares of common stock were available for future issuance under the 2015 Plan. (B) Stock Options: Time-based stock options granted to the Company's employees vest over a period of either (i) 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 equal quarterly installments thereafter for such stock options granted prior to April 2021, or (ii) three years with one-third of the shares of common stock underlying the stock option vesting on the first anniversary of the vesting commencement date and the remainder vesting in 8 equal quarterly installments thereafter for such stock options granted since April 2021, each 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. Options with market-based performance conditions vest based on the trading price for the Company's shares of common stock exceeding certain closing price thresholds. 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. During the three months ended June 30, 2021 and 2020, the Company granted options to purchase a total of 1.6 million shares and 0.4 million shares, respectively, of its common stock, with weighted-average exercise prices of $2.47 and $3.45, respectively, and estimated grant date fair values of $3.3 million and $1.1 million, respectively, under the 2015 Plan. There were no options with market-based performance conditions granted during the three months ended June 30, 2021 and June 30, 2020 under the 2015 Plan. At June 30, 2021, options to purchase a total of 3.6 million shares of common stock were outstanding under the 2015 Plan with a weighted-average exercise price of $8.00 per share, including 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. At June 30, 2021, vested options to purchase a total of 1.0 million shares of common stock were outstanding under the 2015 Plan, with no options with market-based performance conditions vested and outstanding. During the three months ended June 30, 2021 and June 30, 2020, the total grant date fair values of stock options that vested under the 2015 Plan were $0.9 million and $1.7 million, respectively. (C) Restricted Stock Units: RSUs granted during the three months ended June 30, 2021 and June 30, 2020 vest in three equal annual installments commencing on the first anniversary of the vesting commencement date, subject to continuing service. 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. During the three months ended June 30, 2021 and June 30, 2020, the Company granted RSUs for a total of 1.1 million and 0.7 million shares of common stock, respectively, with an aggregate grant date fair value of $2.8 million and $2.6 million, respectively, to its employees under the 2015 Plan. At June 30, 2021, RSUs for approximately 1.9 million shares of common stock were outstanding, of which approximately 0.1 million were vested. During the three months ended June 30, 2021 and June 30, 2020, the total grant date fair values of RSUs that vested under the 2015 Plan were $0.6 million and zero, respectively. (D) Stock-based Compensation Expense: The Company recorded total stock-based compensation expense of $1.3 million and $1.6 million for the three months ended June 30, 2021 and 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 8(E)). The stock-based compensation expense was included in research and development and general and administrative expenses in the Company's unaudited condensed consolidated statements of operations. At June 30, 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 $10.5 million, which is expected to be recognized over the remaining weighted-average service period of 2.36 years. (E) RSL Common Share Awards and Options: Certain employees of the Company 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 $6 thousand and $29 thousand during the three months ended June 30, 2021 and 2020, respectively. |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Jun. 30, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Commitments and Contingencies As of June 30, 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 and agreements to rent office space. 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- to 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. |
Subsequent Events
Subsequent Events | 3 Months Ended |
Jun. 30, 2021 | |
Subsequent Events [Abstract] | |
Subsequent Events | Subsequent EventsIn July 2021, pre-funded warrants to purchase up to 3,301,998 shares of the Company's common stock were fully exercised at an exercise price of $0.00001 per share, which were originally issued and sold in February 2020 as part of a follow-on public offering (see Note 7(B)). |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Jun. 30, 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 unaudited condensed consolidated financial statements and accompanying notes have been prepared in accordance with accounting principles generally accepted in the United States ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. These unaudited condensed consolidated financial statements and accompanying notes should be read in conjunction with the Company’s audited consolidated financial statements and notes thereto included in the Company’s Annual Report on Form 10-K for the fiscal year ended March 31, 2021 (the "Annual Report"), filed with the SEC on June 9, 2021. In the opinion of management, all adjustments (consisting of normal recurring adjustments) considered necessary to present fairly the financial position of the Company and its results of operations and cash flows for the periods presented have been included. Operating results for the three months ended June 30, 2021 are not necessarily indicative of the results that may be expected for the year ending March 31, 2022, for any other interim period, or for any other future year. 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 unaudited condensed 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 unaudited condensed 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. There have been no significant changes in the Company’s accounting policies from those disclosed in its Annual Report. |
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 three months ended June 30, 2021 and the fiscal year ended March 31, 2021, the Company incurred net losses of $11.9 million and $32.4 million, respectively. As of June 30, 2021, the Company’s cash and cash equivalents totaled $111.0 million and its accumulated deficit was $802.9 million. The Company estimates that its current cash and cash equivalents balance is sufficient to support operations beyond the twelve month period following the date that these unaudited condensed 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 does not have substantial doubt about its ability to continue as a going concern for the one-year period following the date that these unaudited condensed 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 June 30, 2021 and through the date of issuance of these unaudited condensed 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. |
Net Loss per Common Share | 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 7(B) and Note 10) 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 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. Restricted Stock Units ("RSUs") and stock options outstanding for a total of 5.6 million and 3.2 million shares of common stock were not included in the calculation of diluted weighted-average shares of common stock outstanding for the three months ended June 30, 2021 and June 30, 2020, respectively, because they were anti-dilutive given the net loss of the Company. |
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. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements: 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 outstanding, 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. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Jun. 30, 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 June 30, 2021 and March 31, 2021 in determining such values (in thousands): As of June 30, 2021 As of March 31, 2021 Fair Value Price Quotations (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Fair Value Price Quotations (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Money market fund $ 107,000 $ 107,000 $ — $ — $ 114,000 $ 114,000 $ — $ — |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 3 Months Ended |
Jun. 30, 2021 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | As of June 30, 2021, and March 31, 2021, accrued expenses consisted of the following (in thousands): June 30, 2021 March 31, 2021 Research and development expenses $ 4,336 $ 6,091 Bonuses and other compensation expenses 1,253 2,331 Other expenses 800 774 Total accrued expenses $ 6,389 $ 9,196 |
Description of Business (Detail
Description of Business (Details) | 3 Months Ended |
Jun. 30, 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 - Narrative (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | ||||
Jun. 30, 2021 | Jun. 30, 2020 | Mar. 31, 2021 | Jul. 31, 2021 | Mar. 31, 2020 | Feb. 29, 2020 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||||
Net loss | $ (11,870) | $ (8,594) | $ (32,400) | |||
Cash and cash equivalents | 110,976 | $ 55,482 | 118,986 | $ 80,752 | ||
(Accumulated deficit) retained earnings | $ (802,939) | (791,069) | ||||
Employee Stock Option and Warrant | ||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||||
Anti-dilutive securities not included in calculation of common shares outstanding (in shares) | 5,600,000 | |||||
Stock Options and RSUs | ||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||||
Anti-dilutive securities not included in calculation of common shares outstanding (in shares) | 3,200,000 | |||||
Money Market Funds | ||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||||
Money market funds | $ 107,000 | $ 114,000 | ||||
Public Offering | ||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||||
Number of securities called by warrants (in shares) | 3,301,998 | |||||
Public Offering | Subsequent Event | ||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||||
Number of securities called by warrants (in shares) | 3,301,998 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Money Market Fund (Details) - Money market fund - USD ($) $ in Thousands | Jun. 30, 2021 | Mar. 31, 2021 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents, fair value disclosure | $ 107,000 | $ 114,000 |
Price Quotations (Level 1) | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents, fair value disclosure | 107,000 | 114,000 |
Significant Other Observable Inputs (Level 2) | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents, fair value disclosure | 0 | 0 |
Significant Unobservable Inputs (Level 3) | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents, fair value disclosure | $ 0 | $ 0 |
License and Collaboration Agr_2
License and Collaboration Agreements (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
License Agreement [Line Items] | ||
Research and development expenses | $ 8,058 | $ 5,194 |
The University of Massachusetts Medical School | Collaborative Arrangement, Transaction with Party to Collaborative Arrangement | ||
License Agreement [Line Items] | ||
Research and development expenses | 3,400 | 400 |
Payments for license agreement | 1,600 | 0 |
Oxford BioMedica (UK) Ltd. | Collaborative Arrangement, Transaction with Party to Collaborative Arrangement | ||
License Agreement [Line Items] | ||
Research and development expenses | 600 | 1,700 |
Payments for license agreement | $ 22 | $ 500 |
Investment in Arvelle Therape_2
Investment in Arvelle Therapeutics B.V. (Details) $ / shares in Units, shares in Millions, $ in Millions | 1 Months Ended | ||||
Mar. 31, 2021USD ($) | Feb. 28, 2021USD ($)shares | Nov. 12, 2020$ / shares | May 30, 2020€ / shares | Feb. 28, 2019€ / shares | |
Schedule of Equity Method Investments [Line Items] | |||||
Par value (in EUR per share) | $ / shares | $ 0.00001 | ||||
Arvelle Therapeutics | |||||
Schedule of Equity Method Investments [Line Items] | |||||
Maximum shares to be purchased under subscription agreement (in shares) | shares | 8.1 | ||||
Proceeds from sale of investment | $ 11.6 | ||||
Future payment held in escrow | 1.2 | ||||
Potential future regulatory and sales milestone payments | 7 | ||||
Par value (in EUR per share) | € / shares | € 0.00001 | € 0.00001 | |||
Net gain on sale of investment | $ 4.3 | $ 4.7 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Mar. 31, 2021 |
Payables and Accruals [Abstract] | ||
Research and development expenses | $ 4,336 | $ 6,091 |
Bonuses and other compensation expenses | 1,253 | 2,331 |
Other expenses | 800 | 774 |
Total accrued expenses | $ 6,389 | $ 9,196 |
Long Term Debt (Details)
Long Term Debt (Details) - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended |
Apr. 30, 2020 | Jun. 30, 2020 | |
Debt Instrument [Line Items] | ||
Extinguishment of debt amount | $ 0.5 | |
Loan and Security Agreement with Hercules Capital, Inc. | Secured Debt | ||
Debt Instrument [Line Items] | ||
Prepayment of outstanding principal due | $ 15.7 | |
Accrued interest, fees and other amounts | $ 0.3 |
Stockholders' Equity (Details)
Stockholders' Equity (Details) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | 17 Months Ended | ||||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Mar. 31, 2021 | Nov. 12, 2020 | Feb. 29, 2020 | |
Stockholders' Equity | ||||||
Common shares authorized (in shares) | 1,000,000,000 | 1,000,000,000 | 1,000,000,000 | 1,010,000,000 | ||
Common shares issued (in shares) | 69,639,509 | 69,639,509 | 69,377,567 | 1,000,000,000 | ||
Common shares par value (in dollars per share) | $ 0.00001 | $ 0.00001 | $ 0.00001 | $ 0.00001 | ||
Preferred stock shares authorized (in shares) | 10,000,000 | |||||
Preferred Stock shares par value (in dollars per share) | $ 0.00001 | |||||
Stock Offering | ||||||
Stockholders' Equity | ||||||
Number of securities called by warrants (in shares) | 3,301,998 | |||||
Warrants issued (in dollars per share) | $ 3.74999 | |||||
Exercise price (in dollars per share) | $ 0.00001 | |||||
Percent ownership threshold to no longer exercise | 9.99% | |||||
Private Placement | SVB Leerink LLC | ||||||
Stockholders' Equity | ||||||
Percentage of gross proceeds from common stock issuance paid for services | 3.00% | |||||
Shares sold in common stock issuance (in shares) | 200,000 | 1,400,000 | 29,800,000 | |||
Net proceeds from common stock issued | $ 0.5 | $ 4.3 | $ 90.9 |
Stock-Based Compensation (Detai
Stock-Based Compensation (Details) $ / shares in Units, $ in Thousands, shares in Millions | 1 Months Ended | 3 Months Ended | |||
Sep. 30, 2019shares | Jun. 30, 2021USD ($)installment$ / sharesshares | Jun. 30, 2020USD ($)installment$ / sharesshares | Apr. 30, 2021shares | Apr. 30, 2020shares | |
Stock-Based Compensation | |||||
Fair value of options vested | $ | $ 900 | $ 1,700 | |||
Number of shares from vesting (in shares) | 0.3 | ||||
Employees | RSL | |||||
Stock-Based Compensation | |||||
Share-based compensation expense | $ | $ 6 | 29 | |||
Time-Based Stock Options | |||||
Stock-Based Compensation | |||||
Number of options vested, outstanding (in shares) | 1 | ||||
Time-Based Stock Options | Tranche One | |||||
Stock-Based Compensation | |||||
Vesting period | 4 years | ||||
RSUs vesting (as a percent) | 25.00% | ||||
Number of installments | installment | 12 | ||||
Time-Based Stock Options | Tranche Two | |||||
Stock-Based Compensation | |||||
Vesting period | 3 years | ||||
RSUs vesting (as a percent) | 33.33% | ||||
Number of installments | installment | 8 | ||||
Stock Options | |||||
Stock-Based Compensation | |||||
Total unrecognized compensation expense, stock options | $ | $ 10,500 | ||||
Remaining weighted-average service period | 2 years 4 months 9 days | ||||
Stock Options | Grants to Directors and Employees | |||||
Stock-Based Compensation | |||||
Share-based compensation expense | $ | $ 1,300 | $ 1,600 | |||
Market-Based Performance Stock Options | |||||
Stock-Based Compensation | |||||
Number of options granted (in shares) | 0 | 0 | |||
Number of options outstanding (in shares) | 0.4 | ||||
Weighted average exercise price of options outstanding (in dollars per share) | $ / shares | $ 8.87 | ||||
Number of options vested, outstanding (in shares) | 0 | ||||
Restricted Stock Units (RSUs) | |||||
Stock-Based Compensation | |||||
Number of installments | installment | 3 | 3 | |||
Fair value of options vested | $ | $ 600 | $ 0 | |||
RSUs granted (in shares) | 1.1 | 0.7 | |||
Aggregate grant date fair value of RSUs | $ | $ 2,800 | $ 2,600 | |||
RSUs outstanding (in shares) | 1.9 | ||||
RSUs vested (in shares) | 0.1 | ||||
Restricted Stock Units (RSUs) | Tranche One | |||||
Stock-Based Compensation | |||||
RSUs vesting (as a percent) | 50.00% | ||||
Restricted Stock Units (RSUs) | Tranche Two | |||||
Stock-Based Compensation | |||||
RSUs vesting (as a percent) | 50.00% | ||||
2015 Equity Incentive Plan | |||||
Stock-Based Compensation | |||||
Shares authorized for issuance (in shares) | 8.4 | 2.8 | 1.6 | ||
Number of shares available for future grant (in shares) | 2.3 | ||||
2015 Equity Incentive Plan | Stock Options | |||||
Stock-Based Compensation | |||||
Number of options granted (in shares) | 1.6 | 0.4 | |||
Exercise price of options granted (in dollars per share) | $ / shares | $ 2.47 | $ 3.45 | |||
Estimated grant date fair value of options granted | $ | $ 3,300 | $ 1,100 | |||
Number of options outstanding (in shares) | 3.6 | ||||
Weighted average exercise price of options outstanding (in dollars per share) | $ / shares | $ 8 |
Commitments and Contingencies (
Commitments and Contingencies (Details) - Collaborative Arrangement, Transaction with Party to Collaborative Arrangement | 3 Months Ended |
Jun. 30, 2021 | |
Third Parties for Pharmaceutical Manufacturing and Research Activities | Minimum | |
Long-term Purchase Commitment [Line Items] | |
Termination notice term, for uncured material breach from other party | 30 days |
Third Parties for Pharmaceutical Manufacturing and Research Activities | Maximum | |
Long-term Purchase Commitment [Line Items] | |
Termination notice term, for uncured material breach from other party | 60 days |
The University of Massachusetts Medical School | |
Long-term Purchase Commitment [Line Items] | |
Termination notice term | 90 days |
Oxford BioMedica (UK) Ltd. | |
Long-term Purchase Commitment [Line Items] | |
Termination notice term, prior to first commercial sale of product | 2 months |
Subsequent Events (Details)
Subsequent Events (Details) - Public Offering - $ / shares | Jul. 31, 2021 | Feb. 29, 2020 |
Subsequent Event [Line Items] | ||
Number of securities called by warrants (in shares) | 3,301,998 | |
Exercise price (in dollars per share) | $ 0.00001 | |
Subsequent Event | ||
Subsequent Event [Line Items] | ||
Number of securities called by warrants (in shares) | 3,301,998 | |
Exercise price (in dollars per share) | $ 0.00001 |