Securities registered pursuant to Section 12(b) of the Act: Securities registered pursuant to Section 12(g) of the Act:None Indicate by check mark if the Registrant is not required to file reports pursuant to Section 13 or 15(d) of the Act. YES☐NO☒ Indicate by check mark whether the Registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding12 months (or for such shorter period that the Registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. YES☒NO☐ Indicate by check mark whether the Registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T(§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the Registrant was required to submit such files). YES☒NO☐ Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company, or an emerging growthcompany. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the ExchangeAct. If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financialaccounting standards provided pursuant to Section 13(a) of the Exchange Act.☐ Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financialreporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report.☐ If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect thecorrection of an error to previously issued financial statements.☐ Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of theregistrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b).☐ Indicate by check mark whether the Registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). YES☐NO☒ The number of shares of Registrant’s Common Stock outstanding as of March 3, 2026 was 1,372,043. Portions of the Registrant’s definitive Proxy Statement for its 2026 Annual Meeting of Stockholders to be filed with the Securities and Exchange Commission pursuant toRegulation 14A not later than 120 days after the end of the fiscal year covered by this Annual Report on Form 10-K are incorporated by reference into Part III of this AnnualReport on Form 10-K. TABLE OF CONTENTS SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTSSUMMARY OF RISKS ASSOCIATED WITH OUR BUSINESS PART I Item 1.BusinessItem 1A.Risk FactorsItem 1B.Unresolved Staff CommentsItem 1C.CybersecurityItem 2.PropertiesItem 3.Legal ProceedingsItem 4.Mine Safety Disclosures PART II Item 5.Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of EquitySecurities88Item 6.[Reserved]88Item 7.Management’s Discussion and Analysis of Financial Condition and Results of Operations89Item 7A.Quantitative and Qualitative Disclosures About Market Risk98Item 8.Financial Statements and Supplementary Data99Item 9.Changes in and Disagreements With Accountants on Accounting and Financial Disclosure127Item 9A.Controls and Procedures127Item 9B.Other Information128Item 9C.Disclosure Regarding Foreign Jurisdictions that Prevent Inspections128 PART III Item 10.Directors, Executive Officers and Corporate Governance129Item 11.Executive Compensation129Item 12.Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters129Item 13.Certain Relationships and Related Transactions, and Director Independence129Item 14.Principal Accountant Fees and Services130 PART IVItem 15. Exhibit and Financial Statement SchedulesItem 16Form 10-K Summary 131134 SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS This Annual Report on Form 10-K (the “Annual Report”) contains forward-looking statements within the meaning of thefederal securities laws made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Allstatements other than statements of historical facts contained in this Annual Report, including statements regarding our futureresults of operations and financial position, business strategy, research and development costs; the ability to seek acceleratedapproval of tralesinidase alfa enzyme replacement therapy (“TA-ERT”) for Sanfilippo Syndrome Type B, orMucopolysaccharidosis Type IIIB (“MPS IIIB”) based on existing clinical data; the ti