The following discussion and analysis of our financial condition and results of operations should be read in conjunction with the audited consolidated financial statements and the related notes thereto included elsewhere in this Annual Report on Form 10-K.
Overview
Historically, we were a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases. We previously had three clinical-stage programs: AXO-AAV-GM1 for the treatment of GM1 gangliosidosis, AXO-AAV-GM2 for the treatment of GM2 gangliosidosis (including Tay-Sachs and Sandhoff diseases) and AXO-Lenti-PD for the treatment of Parkinson's disease.
Currently, we are winding down these three clinical-stage programs while also working on one pre-clinical program.
InJune 2018 , we entered into the Oxford Agreement with Oxford pursuant to which we received a worldwide, 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. InFebruary 2022 , we provided notice to Oxford to terminate the Oxford Agreement to develop and commercialize AXO-Lenti-PD and related gene therapy product candidates. We determined to terminate the Oxford Agreement and redirect resources to our AXO-AAV-GM1 and -GM2 programs, as well as other strategic initiatives, due to several factors, including the resource requirements and development timelines to reach meaningful value inflection for the program and an increasingly challenging market and regulatory environment for Parkinson's disease. We will continue to incur immaterial expenses in connection with the Oxford Agreement until its termination becomes effective. InDecember 2018 , we entered into the UMMS Agreement with UMMS pursuant to which we 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). InApril 2022 , we provided notice to UMMS to terminate the UMMS Agreement. The UMMS Agreement will be formally terminated following the 90-day wind-down/termination notice period. We will continue to conduct clinical operations for the AXO-AAV-GM1 and AXO-AAV-GM2 programs under the UMMS Agreement during the 90-day wind-down/termination period. In parallel with our decision to terminate the AXO-AAV-GM1 and -GM2 programs, inApril 2022 , our board of directors approved and we announced the strategic decision to explore and review a range of strategic alternatives focused on maximizing stockholder value from our existing cash and cash equivalents, including a potential sale, merger, business combination or similar transaction. In connection with these actions, and as approved by our board of directors, we began implementing a significant headcount reduction, which we expect to conclude inJune 2022 . As part of these strategic decisions, we expect to incur aggregate costs estimated to range from approximately$0.9 million to$1.5 million relating to the reduction in headcount, all to be incurred during the fiscal quarter endingJune 30, 2022 . While we continue to conduct certain pre-clinical research and development initiatives in gene therapy, we expect to devote substantial time and resources to exploring strategic alternatives. Despite devoting significant efforts to identify and evaluate potential strategic alternatives, there can be no assurance that this strategic review process will result in us pursuing any transaction or that any transaction, if pursued, will be completed on attractive terms or at all. We have not set a timetable for completion of this strategic review process, and our board of directors has not approved a definitive course of action. Additionally, there can be no assurances that any particular course of action, business arrangement or transaction, or series of transactions, will be pursued, successfully consummated or lead to increased stockholder value or that we will make any additional cash distribution to our stockholders. In addition, we expect to incur additional operating expenses associated with the wind-down of the UMMS Agreement and the Oxford Agreement, including clinical trial activities that we will continue to conduct during the wind-down period. 33 --------------------------------------------------------------------------------
The Domestication
We have substantially completed our previously disclosed corporate transformation to align corporate structure and governance with current and future business activity, including significantly reducing the number of our subsidiaries. OnNovember 12, 2020 ,Axovant Gene Therapies Ltd. ("AGT") discontinued as aBermuda exempted company pursuant to Section 132G of the Companies Act 1981 ofBermuda , and pursuant to Section 388 of the General Corporation Law of theState of Delaware (the "DGCL"), continued its existence under the DGCL as a corporation namedSio Gene Therapies Inc. ("Sio") organized in theState of Delaware (the "Domestication"). The Domestication effected a change in our jurisdiction of incorporation, and other changes of a legal nature, including changes in our organizational documents. Our consolidated business, operations, assets and liabilities did not change upon effectiveness of the Domestication. However, following the Domestication, the principal executive offices and registered offices of Sio are located at130 West 42nd St , 26th Floor,New York, New York 10036, and the telephone number for Sio at its principal executive offices is 1-877-746-4891. The fiscal year end ofSio Gene Therapies Inc. following the Domestication remains atMarch 31 . In addition, our directors and executive officers immediately after the Domestication were the same individuals who were directors and executive officers, respectively, immediately prior to the Domestication. In the Domestication, each of our currently issued and outstanding common shares automatically converted by operation of law, on a one-for-one basis, into shares of Sio common stock. Consequently, upon the effectiveness of the Domestication, each holder of an AGT common share instead holds a share of Sio common stock representing the same proportional equity interest in Sio as that stockholder held in AGT and representing the same class of shares. The number of shares of Sio common stock outstanding immediately after the Domestication is the same as the number of common shares of AGT. outstanding immediately prior to the Domestication. In connection with the Domestication, we adopted a new certificate of incorporation, bylaws and form of common stock certificate, copies of which were filed as Exhibits 3.1, 3.2 and 4.1, respectively, to our Report on Form 8-K12G3 filed with theSEC onNovember 13, 2020 .
COVID-19 Business Update
We are continuing to closely monitor the impact of the global COVID-19 pandemic on our business and operations. We believe that the measures we have previously implemented are appropriate, reflecting both regulatory and public health guidance, to maintain business continuity. We will continue to closely monitor and seek to comply with guidance from governmental authorities and adjust our activities as appropriate. In the conduct of our business activities during the pandemic, we took actions designed to protect the safety and well-being of patients, healthcare workers and employees. For patients previously enrolled in our clinical trials, we worked closely with clinical trial investigators and site staff to continue treatment in compliance with trial protocols and to uphold trial integrity, while working to observe government and institutional guidelines designed to safeguard the health and safety of patients, clinical trial investigators and site staff. While the COVID-19 pandemic has not resulted in a significant delay to our prior clinical development timelines to-date and has not had a significant impact to our historical operations, the COVID-19 pandemic continues to evolve, including as a result of variants, and could materially impact our strategic goals to explore and review a range of strategic alternatives focused on maximizing stockholder value from our existing cash and cash equivalents, including a potential sale, merger, business combination or similar transaction. The COVID-19 pandemic and related impacts (including inflationary pressures) could result in significant and prolonged disruption of global financial markets, which has negatively impacted and may continue to reduce our ability to access capital, limiting the financial resources available to us. We do not yet know the full extent of potential impacts on our business, operations, strategic goals, or the global economy as a whole. However, these effects could harm our operations, and we will continue to monitor the COVID-19 situation closely. For additional information about risks and uncertainties related to the COVID-19 pandemic that may impact our business, financial condition and results of operations, see the section titled "Risk Factors" under Part I, Item 1A in this Annual Report on Form 10-K.
Financial Operations Overview
Revenue
We have not generated any revenue from the sale of any products, and we do not expect to generate any revenue unless and until we obtain regulatory approval of and begin to commercialize any product candidates.
Research and Development Expense
Since our inception, our operations have historically primarily been focused on organizing and staffing our company, raising capital, and acquiring, preparing for and advancing our prior product candidates into clinical development. Our research and development expenses include program-specific costs, as well as unallocated internal costs. 34 --------------------------------------------------------------------------------
Program-specific costs include:
•direct third-party costs, which include expenses incurred under agreements with CROs and contract manufacturing organizations, the cost of consultants who assist with the development of our product candidates on a program-specific basis, investigator grants, sponsored research, manufacturing costs in connection with producing materials for use in conducting nonclinical and clinical studies, and any other third-party expenses directly attributable to the development of our prior product candidates; and
•payments for research and development milestones, which include costs incurred under our agreements with UMMS and Oxford.
Unallocated internal costs include:
•stock-based compensation expense for research and development personnel;
•personnel-related expenses, which include employee-related expenses, such as salaries, benefits and recruiting expenses, for research and development personnel; and
•other expenses, which include research and development software costs, travel expenses, laboratory facility rental costs and research and development equipment depreciation expenses, as well as the cost of consultants who assist with our research and development but are not allocated to a specific program. Our research and development expenses are expected to decrease substantially in the near term, following the previously announced discontinuation of our AXO-AAV-GM1, AXO-AAV-GM2 and AXO-Lenti-PD programs, as well as the significant reduction in workforce that we implemented subsequent toMarch 31, 2022 . These programs are expected to be wound down byJune 30, 2022 after which our research and development activities will be concentrated on one preclinical program.
General and Administrative Expense
General and administrative expenses consist primarily of employee-related expenses such as salaries, benefits and travel expenses for our general and administrative personnel; stock-based compensation, including stock-based compensation allocated to us from our affiliate, Roivant Sciences Ltd. ("RSL"), for certain RSL equity instruments granted to certain of our employees (primarily our former CEO (the "RSL Equity Instruments"), who resigned as our CEO inJanuary 2022 ); non-employee benefit insurance premiums; third-party legal and accounting fees; information technology costs; office rent, fixed asset depreciation and other overhead costs; and consulting services. During the fiscal year endingMarch 31, 2023 , we anticipate that our general and administrative expenses will decrease primarily as a result of stock-based compensation expense largely associated with the RSL Equity Instruments, for which expensing commenced upon the liquidity event vesting condition being met upon the closing of RSL's business combination withMontes Archimedes Acquisition Corp. ("MAAC") onSeptember 30, 2021 .
Results of Operations for the Years Ended
The following table summarizes our results of operations for the years ended
Years Ended March 31, 2022 2021 Operating expenses: Research and development expenses (includes$1,286 and$1,583 of stock-based compensation expense for the years ended March 31, 2022 and 2021, respectively)$ 53,399 $ 24,903 General and administrative expenses (includes$6,139 and$2,909 of stock-based compensation expense for the years ended March 31, 2022 and 2021, respectively) 18,163 17,294 Total operating expenses 71,562 42,197 Interest expense 27 799 Other expense (income) 39 (10,359) Income tax expense (benefit) 259 (212) Net loss$ (71,887) $ (32,425) 35
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Research and Development Expenses
For the years ended
Years Ended March 31, 2022 2021 Change Program-specific costs: AXO-AAV-GM1$ 17,021 $ 4,676 $ 12,345 AXO-AAV-GM2 10,725 2,231 8,494 AXO-Lenti-PD 10,571 5,668 4,903
Unallocated internal costs:
Personnel-related 9,439 7,058 2,381 Stock-based compensation 1,286 1,583 (297) Other 4,357 3,687 670 Total research and development expenses$ 53,399 $
24,903
Research and development expenses were
(i) a$10.8 million increase in AXO-AAV-GM1 program expenses primarily related to clinical trial material manufacturing expenses (not including a milestone payment disclosed in (iii) below) ;
(ii) a
(iii)
General and Administrative Expenses
General and administrative expenses were$18.2 million for the year endedMarch 31, 2022 and$17.3 million for the year endedMarch 31, 2021 . The increase of$0.9 million was primarily related to an increase of$3.9 million of stock-based compensation expense associated with the RSL Equity Instruments, for which expensing commenced upon the liquidity event vesting condition being met upon the closing of RSL's business combination with MAAC onSeptember 30, 2021 . These increases were partially offset by decreases of (i)$1.7 million for rent, depreciation and facility expenses primarily due to the downsizing of ourNew York office footprint, (ii)$0.7 million for tax, auditing and accounting fees resulting primarily from the simplification of our corporate structure and the domestication ofSio Gene Therapies Inc. fromBermuda toDelaware that was completed inNovember 2020 , and (iii)$0.7 million for stock-based compensation expense unrelated to the RSL Equity Instruments.
Interest Expense
Interest expense was$27 thousand and$0.8 million for the years endedMarch 31, 2022 and 2021, respectively. The decrease in interest expense during the current year was due to theApril 2020 prepayment in full of the$15.7 million outstanding principal balance on our loan and security agreement with Hercules Capital, Inc. ("Hercules"). Other Expense (Income) Other expense (income) was$39 thousand and$(10.4) million for the years endedMarch 31, 2022 and 2021, respectively. Other expense for the year endedMarch 31, 2022 consisted primarily of foreign exchange losses. Other income for the year endedMarch 31, 2021 included income of approximately$11.3 million associated with gains on our investment inArvelle Therapeutics B.V. ("Arvelle") that was sold inFebruary 2021 , which was partially offset by foreign exchange losses. 36
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Liquidity and Capital Resources
Sources of Liquidity
Since our initial public offering inJune 2015 , our operations have been financed primarily through sales of common stock and pre-funded warrants, as well as borrowings under our credit facilities. As ofMarch 31, 2022 , we had$63.7 million of cash and cash equivalents available to us, and inApril 2020 , we prepaid the remaining outstanding principal balance, equal to$15.7 million , together with$0.3 million of accrued interest, fees and other amounts due under our loan and security agreement with Hercules.
Capital Requirements
We have not yet achieved profitability and expect to continue to incur operating and net losses, as well as negative cash flows from operations, for the foreseeable future. We have not generated any revenue to date. Until such time, if ever, as we can generate product substantial revenue, and subject to our pursuit of strategic alternatives, we expect to primarily finance our cash needs using our existing cash. We expect that our existing cash and cash equivalents of$63.7 million atMarch 31, 2022 will enable us to fund our current operating plan beyond the twelve-month period following the date that the accompanying consolidated financial statements and footnotes were issued. In order to meet longer operating requirements, including as we continue to explore and pursue strategic alternatives, we will need additional capital resources. We have based these estimates on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect. Our principal operating focus is currently on pursuing a range of strategic alternatives. We believe we have sufficient cash resources, net of costs which we estimate to incur in relation to such a transaction, to complete a strategic transaction. If we do not complete a strategic transaction, we may consider dissolving the Company and liquidating the assets. In that case, we believe that our cash resources are sufficient to satisfy estimated liabilities and costs of such a process. However, the achievement of a strategic transaction and the associated costs and timing thereof is uncertain and the time, cost and reserves which may be required to be held back for future claims is uncertain so our estimates may prove incorrect. Our future funding requirements, both near and long-term, will depend on many factors, including, but not limited to, the timing and outcome of our exploration of, and execution upon any, potential strategic alternatives, the cost of obtaining necessary intellectual property and defending potential intellectual property disputes, realization of the anticipated benefits of our headcount reduction, and the costs of operating as a public company. For the years endedMarch 31, 2022 andMarch 31, 2021 , we incurred net losses of$71.9 million and$32.4 million , respectively. As ofMarch 31, 2022 , our cash and cash equivalents totaled$63.7 million and our accumulated deficit was$863.0 million . We estimate that our current cash and cash equivalents balance is sufficient to support operations beyond the twelve-month period following the date that the accompanying consolidated financial statements were issued. This estimate is based on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect. We expect to primarily finance our cash needs using our existing cash. We do not currently have any committed external source of funds. We continually assess multiple options to obtain additional funding to support our operations, including proceeds from offerings of our equity securities or debt financings. To the extent that we raise additional capital through the sale of equity or convertible debt securities, our stockholders' ownership interests will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect our stockholders' rights. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. Sources of a sufficient amount of financing may not be available to us on favorable terms, if at all, and our ability to raise additional capital has been, and may continue to be, adversely impacted by, among other things, potentially worsening global economic conditions and the recent disruptions to and volatility in the credit and financial markets inthe United States and worldwide resulting from the ongoing COVID-19 pandemic. In addition, extreme price and volume fluctuations in the stock market in general, and the Nasdaq Global Select Market, in particular, have resulted in volatile and sometimes decreased stock prices for many companies, including us. Broad market and industry factors, including worsening economic conditions and other adverse effects or developments relating to the evolving effects of the COVID-19 pandemic, may negatively affect the market price of our common stock, regardless of our actual operating performance, and impact our ability to raise sufficient additional capital on acceptable terms, if at all. 37 --------------------------------------------------------------------------------
At-the-Market Equity Offering Program
We have engagedSVB Securities LLC as our agent to sell shares of our common stock from time to time through an at-the-market equity offering program.SVB Securities LLC is entitled to compensation for its services in an amount equal to 3% of the gross proceeds of any of our shares of common stock sold. As ofMarch 31, 2022 , we have sold approximately 30.4 million shares of common stock for total proceeds of approximately$92.0 million , net of brokerage fees, under the sales agreement sinceApril 2020 .
Cash Flows
The following table sets forth a summary of our cash flows for each of the periods shown (in thousands):
Years EndedMarch 31, 2022 2021
Net cash used in operating activities
Net cash provided by investing activities 3,648 12,386 Net cash provided by financing activities 1,441 73,621 Operating Activities Cash flows from operating activities consist of net loss adjusted for non-cash items, including depreciation and stock-based compensation expenses, as well as the effect of changes in working capital and other activities. For the year endedMarch 31, 2022 , net cash used in operating activities was$60.3 million and was primarily attributable to a net loss of$71.9 million , which includes costs incurred for research and development activities, including CRO fees, manufacturing, regulatory and other clinical trial costs, as well as our general and administrative expenses, in addition to a decrease of$1.0 million in accrued expenses, which were partially offset by$7.4 million of non-cash stock-based compensation expense, an increase of$2.6 million in accounts payable, as well as a decrease of$2.1 million in prepaid expenses and other current assets. For the year endedMarch 31, 2021 , net cash used in operating activities was$46.6 million and was primarily attributable to a net loss of$32.4 million , which includes costs incurred for research and development activities, including CRO fees, manufacturing, regulatory and other clinical trial costs, as well as our general and administrative expenses, in addition to other income of$11.3 million associated with gains on our investment in Arvelle, an increase of$4.4 million in prepaid expenses and other current assets and decreases of$3.1 million in accounts payable and$2.1 million in accrued expenses, which were partially offset by$4.5 million of non-cash stock-based compensation expense and$1.5 million of operating lease right-of-use asset amortization expense.
Investing Activities
For the year endedMarch 31, 2022 , net cash provided by investing activities was$3.6 million , consisting of proceeds of$4.3 million from the sale of our long-term investment in Arvelle that was partially offset by purchases of fixed assets. For the year endedMarch 31, 2021 , net cash provided by investing activities was$12.4 million , consisting of proceeds of$12.8 million from the sale of our long-term investment in Arvelle that was partially offset by purchases of fixed assets. Financing Activities For the year endedMarch 31, 2022 , net cash provided by financing activities was$1.4 million and consisted of net proceeds from the issuance and sale of our shares of common stock under our share sales agreement withSVB Securities LLC . For the year endedMarch 31, 2021 , net cash provided by financing activities was$73.6 million and consisted primarily of$89.2 million of net proceeds from the issuance and sale of our shares of common stock under our share sales agreement withSVB Securities LLC , partially offset by$15.7 million of principal payments made on long-term debt. 38
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Contractual Obligations
InOctober 2019 , we entered into an agreement with a third-party to lease office space inDurham, North Carolina under a lease agreement expiring inNovember 2022 , and inAugust 2020 , we entered into a lease agreement with a third-party for an office facility inNew York, New York that commenced inDecember 2020 and expires inJune 2026 . InNovember 2021 , we entered into a lease agreement for a research and development facility and related office space inDurham, North Carolina with an initial term expiring inDecember 2024 . For the years endedMarch 31, 2022 andMarch 31, 2021 , we incurred$0.6 million and$1.6 million , respectively, in rent expense under these agreements.. As ofMarch 31, 2022 , our real property lease obligations were$2.9 million with$0.8 million expected to be paid within 12 months and the remainder thereafter. In addition, we have 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 us with 30- or 60-days' written notice, unless otherwise indicated. These cancellable contracts are not included in the total obligations in the preceding paragraph. Further, certain of our manufacturing agreements could require early termination and wind-down payments due from us as a result of the recent termination of our clinical trials.
Recent Accounting Pronouncements
For detailed information regarding recently issued accounting pronouncements and the expected impact on our financial statements, refer to Note 2 "Summary of Significant Accounting Policies," in the accompanying notes to our audited consolidated financial statements included elsewhere in this Annual Report on Form 10-K.
Critical Accounting Policies and Significant Judgments and Estimates
Our management's discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which have been prepared in accordance withU.S. generally accepted accounting principles ("GAAP"). The preparation of these consolidated financial statements and accompanying notes requires us to make estimates, judgments and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the dates of the balance sheets and the reported amounts of expenses during the reporting periods. In accordance withU.S. GAAP, we evaluate our estimates and judgments on an ongoing basis. Significant estimates include research and development accruals. We base our estimates on historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. We define our critical accounting policies as those underU.S. GAAP that require us to make subjective estimates and judgments about matters that are uncertain and are likely to have a material impact on our financial condition and results of operations, as well as the specific manner in which we apply those principles. Our significant accounting policies are more fully described in Note 2, "Summary of Significant Accounting Policies," to our audited consolidated financial statements included elsewhere in this Annual Report on Form 10-K. Not all of these significant accounting policies, however, require that we make estimates and assumptions that we believe are "critical accounting estimates." We believe that our estimates relating to research and development accruals have the greatest potential impact on our consolidated financial statements and consider these to be our critical accounting policies and estimates and are "critical accounting estimates."
Research and Development Accruals
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. The Company's assessment of the completeness of the information is subject to variability and uncertainty. In addition, in certain circumstances, the determination of the nature and amount of 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. 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 are charged to expense when incurred and currently primarily consist of the development and regulatory milestones achieved for our AXO-AAV-GM1, AXO-AAV-GM2 and AXO-Lenti-PD gene therapy programs, as well as research and development materials acquired from UMMS and Oxford and expenses from third parties who conduct research and development activities on our behalf. We expense in-process research and development projects acquired as asset acquisitions which have not reached technological feasibility, and which have no alternative future use. 39
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