What changed in this filing

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Item 9A. Controls and Procedures

2025-11-25 compared with 2024-11-26 · 492 added, 312 removed, 1106 unchanged (42% of the section changed)

20 unchanged sentences

There has been no change in the Company’s internal control over financial reporting during the Company’s most recent fiscal quarter that has materially affected, or is reasonably likely to materially affect, the Company’s internal control over financial reporting.

The Company regularly evaluates its controls and procedures and makes improvements in the design and effectiveness of established controls and procedures and the remediation of any deficiencies which may be identified during this process.

The Company regularly evaluates its controls and procedures and makes improvements in the

design and effectiveness of established controls and procedures and the remediation of any deficiencies which may be identified during this process.

OTHER INFORMATION

(a) License and Collaboration Agreement

On November 25, 2024, the Company entered into an Exclusive License and Collaboration Agreement (the “Collaboration Agreement”) with Sarepta Therapeutics, Inc.

(“Sarepta”) for the co-development and commercialization of multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system, and the lungs.

Under the Collaboration Agreement, Sarepta has received an exclusive worldwide license to the Company’s ARO-DUX4, ARO-DM1, ARO-MMP7, and ARO-ATXN2 clinical stage programs.

Sarepta has also received an exclusive sublicensable worldwide license to the Company’s ARO-HTT, ARO-ATXN1, and ARO-ATXN3 preclinical stage programs.

Pursuant to the Collaboration Agreement, Sarepta will be able to select up to six new targets for which the Company will perform discovery, optimization and preclinical development.

Upon completion of the Company’s preclinical activities, Sarepta will receive an exclusive license to the Company’s product-specific intellectual property rights covering those compounds and be wholly responsible for clinical development and commercialization of each compound.

Under the terms of the Collaboration Agreement, the Company expects to receive $500.0 million as an upfront payment and $250.0 million to be paid in annual installments of $50.0 million over 5 years.

The Company is also eligible to receive $300.0 million in near-term payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study, which the Company is on track to achieve.

Further, for each of the 13 programs, the Company is eligible to receive development milestone payments between $110.0 million and $180.0 million per program and sales milestone payments between $500.0 million and $700.0 million per program.

The Company is also eligible to receive tiered royalties on net sales of licensed products of up to the low double digits.

Closing of the Collaboration Agreement is subject to clearance under the Hart-Scott Rodino Antitrust Improvements Act.

The foregoing description of the Collaboration Agreement does not purport to be complete and is qualified in its entirety by reference to the Collaboration Agreement, a copy of which will be filed with the Company’s Quarterly Report on Form 10-Q for the quarter ended December 31, 2024.

Stock Purchase Agreement

In connection with the Collaboration Agreement, on November 25, 2024, the Company entered into a Stock Purchase Agreement (the “Stock Purchase Agreement”) with an affiliate of Sarepta (the “Purchaser”) for a private placement of shares of common stock of the Company (the “Private Placement”).

Pursuant to the Stock Purchase Agreement, the Company sold 11,926,301 shares of common stock (the “Shares”), at a price per Share of $27.2507, for an aggregate value of approximately $325.0 million.

The Private Placement is expected to close concurrently with the Collaboration Agreement (the “Closing”).

The Stock Purchase Agreement contains customary representations and warranties of the Company, on the one hand, and the Purchaser, on the other hand, and customary conditions to closing.

The Stock Purchase Agreement provides that at any time following the Closing, the Purchaser may elect to exchange any or all of its Shares for pre-funded warrants to purchase shares of common stock of the Company, substantially in the form attached to the Stock Purchase Agreement.

At the Closing, the Company will enter into an Investor Rights Agreement (the “Investor Rights Agreement”) with the Purchaser, which provides that the Company will appoint Doug Ingram to the board of directors of the Company effective as of the Closing.

In addition, the Company will register the resale of the Shares pursuant to the Investor Rights Agreement.

The Company is required to prepare and file a registration statement with the Securities and Exchange Commission no later than 30 days following the Closing.

from certain liabilities and pay certain fees and expenses incident to the Company’s obligations under the Investor Rights Agreement.

The securities to be issued and sold to Purchaser under the Stock Purchase Agreement will not be registered under the Securities Act of 1933, as amended (the Securities Act) in reliance on the exemption from registration provided by Section 4(a)(2) of the Securities Act and/or Rule 506 of Regulation D promulgated thereunder, or under any state securities laws.

The Company relied on this exemption from registration based in part on representations made by the Purchaser.

The securities may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements.

This Annual Report on Form 10-K is not an offer to sell or the solicitation of an offer to buy the securities described herein.

The foregoing descriptions of the Stock Purchase Agreement and the form of Investor Rights Agreement do not purport to be complete and are qualified in their entirety by reference to the Stock Purchase Agreement and the form of Investor Rights Agreement, copies of which are filed as Exhibits 10.48 and 4.6 to this Annual Report on Form 10-K, respectively, and are incorporated by reference herein.

Amendment to Credit Facility

Also on November 26, 2024, the Company entered into an amendment to the Credit Facility (the “Amendment”) to modify, subject to certain conditions, amongst other things, the requirements to make prepayments of the loans under the Credit Facility with respect to the transactions contemplated by the Collaboration Agreement and the Stock Purchase Agreement.

The foregoing description of the Amendment does not purport to be complete and is qualified in its entirety by reference to the Amendment, a copy of which will be filed with the Company’s Quarterly Report on Form 10-Q for the quarter ending December 31, 2024.

Securities Purchase Agreement

On November 25, 2024, the Company entered into a Securities Purchase Agreement (the “Securities Purchase Agreement”) with an institutional and accredited investor (the “Warrant Purchaser”) for a private placement of pre-funded warrants to purchase shares of common stock with an exercise price of $0.001 per share.

Pursuant to the Securities Purchase Agreement, the Company sold pre-funded warrants to purchase up to 917,441 shares of common stock at a purchase price of $27.2497 per pre-funded warrant, for an aggregate value of approximately $25.0 million.

The transaction is expected to close on or about November 27, 2024 (the “Warrant Closing”).

The Securities Purchase Agreement contains customary representations and warranties of the Company, on the one hand, and the Purchasers, on the other hand, and customary conditions to closing.

At the Warrant Closing, the Company will enter into a Registration Rights Agreement (the Registration Rights Agreement) with the Warrant Purchaser, which provides that the Company will register the resale of the shares of common stock underlying the pre-funded warrants pursuant to the Registration Rights Agreement.

The Company is required to prepare and file a registration statement with the Securities and Exchange Commission no later than 30 days following the Warrant Closing.

The Company has also agreed to, among other things, indemnify the Purchaser, their officers, directors, members, employees, partners, managers, stockholders, affiliates, investment advisors and agents under the registration statement from certain liabilities and pay certain fees and expenses incident to the Company’s obligations under the Registration Rights Agreement.

The securities to be issued and sold to Warrant Purchaser under the Securities Purchase Agreement will not be registered under the Securities Act in reliance on the exemption from registration provided by Section 4(a)(2) of the Securities Act and/or Rule 506 of Regulation D promulgated thereunder, or under any state securities laws.

The Company relied on this exemption from registration based in part on representations made by the Warrant Purchaser.

The securities may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements.

This Annual Report on Form 10-K is not an offer to sell or the solicitation of an offer to buy the securities described herein.

The foregoing descriptions of the Securities Purchase Agreement, the form of Registration Rights Agreement and the form of Pre-Funded Warrant do not purport to be complete and are qualified in their entirety by reference to the Securities Purchase Agreement, the form of Registration Rights Agreement and the form of Pre-Funded Warrant, copies of which are filed as Exhibits 10.49, 4.7 and 4.8 to this Annual Report on Form 10-K, respectively, and are incorporated by reference herein.

(b) Trading Plans

During the fiscal quarter ended September 30, 2025, the following directors and officers (as defined in Exchange Act Rule 16a-1(f)) adopted certain trading plans intended to satisfy Rule 10b5-1(c):

Name Title Adoption or Termination Date Plan Start Date Plan End Date Shares Vesting and Subject to Sell-To-Cover (1)

Name Title Adoption or Termination Date Plan End Date Shares Vesting and Subject to Sell-To-Cover (1)

Other Shares Being Sold (Subject to Certain Conditions)

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Christopher Anzalone President and Chief Executive Officer 09/19/2025 12/24/2026 50,000 n/a

Christopher Anzalone President and Chief Executive Officer 08/22/2024 12/04/2024 12/31/2026 2,082,892 n/a

Christopher Anzalone President and Chief Executive Officer 09/16/2024 01/02/2025 12/31/2026 96,566 n/a

Douglass Given Board Member 09/12/2024 12/16/2024 12/31/2024 n/a 5,547

James Hamilton Chief of Discovery and Translational Medicine 08/19/2024 12/04/2024 11/28/2025 n/a 30,000

Daniel Apel Chief Financial Officer 09/16/2025 04/30/2026 25,000 n/a

James Hamilton Chief Medical Officer, Head of R&D 09/03/2025 01/16/2026 72,500 n/a

Mauro Ferrari Board Member 09/25/2025 12/31/2025 n/a 7,530

Patrick O'Brien Chief Operating Officer & General Counsel

09/03/2025 01/07/2026 77,500 20,000

Victoria Vakiener Board Member 09/03/2025 12/31/2025 n/a 10,040

William Waddill Board Member 09/03/2025 12/31/2025 n/a 8,367

(1) This column indicates the total number of shares vesting, but the 10b5-1 Plan provides for the sale of only those shares necessary to satisfy payment of applicable withholding taxes.

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Number Description Form Date

1.1 Open Market Sale Agreement, dated as of December 2, 2022, by and between Arrowhead Pharmaceuticals, Inc.

and Jefferies LLC

Current Report on Form 8-K as Exhibit 1.1 December 2, 2022

2.1† Stock and Asset Purchase Agreement between Arrowhead Research Corporation and Roche entities, dated October 21, 2011

2 unchanged sentences

Quarterly Report on Form 10-Q, as Exhibit 2.1 May 11, 2015

3.1 Amended and Restated Certificate of Incorporation (incorporated by reference from Exhibit 3.3 of the Company’s Form 8-K filed on April 6, 2016)

3.1 Amended and Restated Certificate of Incorporation

Current Report on Form 8-K as Exhibit 3.3 April 6, 2016

3.2 Certificate of Amendment to the Amended and Restated Certificate of Incorporation of Arrowhead Pharmaceuticals, Inc.

(incorporated by reference from Exhibit 3.2 of the Company’s Form 10-Q filed on May 2, 2023)

Quarterly Report on Form 10-Q, as Exhibit 3.2 May 2, 2023

3.3 Second Amended and Restated Bylaws (incorporated by reference from Exhibit 3.1 of the Company’s Form 8-K filed on January 30, 2023)

3.3 Second Amended and Restated Bylaws

Current Report on Form 8-K as Exhibit 3.2 January 30, 2023

7 unchanged sentences

Annual Report on Form 10-K, as Exhibit 4.4 November 25, 2019

Incorporated by Reference Herein

Number Description Form Date

4.5 Registration Rights Agreement by and between Arrowhead Pharmaceuticals, Inc.

1 unchanged sentence

Quarterly Report on Form 10-Q, as Exhibit 10.4 February 7, 2019

Form of Investor Rights Agreement by and between Company and Sarepta Therapeutics Investments, Inc.

(included as Exhibit A in Exhibit 10.4 8 )

Incorporated by Reference Herein

Number Description Form Date

4.6 Form of Registration Rights Agreement by and between Company and Avoro Life Sciences Fund LLC (included as Exhibit B in Exhibit 10.49)

Annual Report on Form 10-K, as Exhibit 4.7

November 26, 2024

4.7 Form of Pre-Funded Warrant for Avoro Life Sciences Fund LLC

Annual Report on Form 10-K, as Exhibit 4.8

November 26, 2024

10.1** Arrowhead Research Corporation 2004 Equity Incentive Plan, as amended

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Annual Report on Form 10-K, as Exhibit 10.11 December 14, 2006

A rrowhead Pharmaceuticals, Inc.

Arrowhead Pharmaceuticals, Inc.

Inducement Plan

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Quarterly Report on Form 10-Q, as Exhibit 10.1 August 12, 2014

Incorporated by Reference Herein

Number Description Form Date

Second Collaboration and Licensing Agreement between Arrowhead Pharmaceuticals, Inc.

1 unchanged sentence

Annual Report on Form 10-K, as Exhibit 10.19 December 14, 2016

Incorporated by Reference Herein

Number Description Form Date

10.20 Common Stock Purchase Agreement between the Company and Amgen Inc., dated September 28, 2016

12 unchanged sentences

Annual Report on Form 10-K, as Exhibit 10.20 November 25, 2019

Amended and Restated License Agreement by an d between Arrowhead Pharmaceuticals, Inc.

Amended and Restated License Agreement by and between Arrowhead Pharmaceuticals, Inc.

and GlaxoSmithKline Intellectual Property (No.

45 unchanged sentences

November 23, 2020

Incorporated by Reference Herein

Number Description Form Date

10.37 Amendment No.

2 unchanged sentences

Quarterly Report on Form 10-Q, as Exhibit 10.3 February 4, 2021

Incorporated by Reference Herein

Number Description Form Date

10.38 Amendment No.

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and University Research Park, dated September 13, 2024

10.43 Amendment No.

1 2 to Lease Agreement by and between Arrowhead Madison, Inc.

and University Research Park, dated June 11, 2025

Quarterly Report on Form 10-Q, as Exhibit 10.6

August 7, 2025

10.44 Office Lease by and between 177 Colorado Owner LLC and Arrowhead Pharmaceuticals, Inc., dated April 17, 2019

12 unchanged sentences

November 29, 2023

10.48 Open Market Sale Agreement, dated as of December 2, 2022, by and between Arrowhead Pharmaceuticals, Inc.

and Jefferies LLC

Current Report on Form 8-K as Exhibit 1.1 December 2, 2022

Financing Agreement by and between Company and Sixth Street Lending Partners, dated August 7, 2024

Stock Purchase Agreement by and between Company and Sarepta Therapeutics Investments, Inc., dated November 25, 2024

Securities Purchase Agreement by and between Company and Avoro Life Sciences Fund LLC, dated November 25, 2024

16.1 Letter from Rose, Snyder & Jacobs LLP, dated December 4, 2023

Current Report on Form 8-K, as Exhibit 16.1

December 5, 2023

Annual Report on Form 10-K, as Exhibit 10.47

November 26, 2024

First Amendment to Financing Agreement by and between Company and Sixth Street Lending Partners, dated November 26, 2024

Quarterly Report on Form 10-Q, as Exhibit 10.4 February 10, 2025

Exclusive License and Collaboration Agreement by and between the Company and Sarepta Therapeutics, Inc., dated November 25, 2024

Quarterly Report on Form 10-Q, as Exhibit 10.3 February 10, 2025

Exclusive License and Collaboration Agreement by and between Arrowhead Pharmaceuticals, Inc.

and Novartis Pharma AG, dated August 29, 2025

Severance and Change of Control Agreement by and between Company and Christopher Anzalone, dated May 9, 2025

Quarterly Report on Form 10-Q, as Exhibit 10.1 May 12, 2025

10.54 Severance and Change of Control Agreement by and between Company and Daniel Apel, dated May 8, 2025

Quarterly Report on Form 10-Q, as Exhibit 10.2 May 12, 2025

10.55 Severance and Change of Control Agreement by and between Company and Patrick O'Brien, dated May 9, 2025

Quarterly Report on Form 10-Q, as Exhibit 10.3 May 12, 2025

10.56 Severance and Change of Control Agreement by and between Company and James Hamilton, dated May 8, 2025

Quarterly Report on Form 10-Q, as Exhibit 10.4 May 12, 2025

10.57 CFO Retirement Letter by and between Company and Ken Myszkowski, dated May 9, 2025

Quarterly Report on Form 10-Q, as Exhibit 10.5 May 12, 2025

19.1 Arrowhead Pharmaceuticals, Inc.

Insider Trading Policy

Annual Report on Form 10-K, as Exhibit 19.1

November 26, 2024

21.1* List of Subsidiaries

Incorporated by Reference Herein

Number Description Form Date

23.1* Consent of Independent Public Registered Accounting Firm

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101.INS* Inline XBRL Taxonomy Extension Instance Document

Incorporated by Reference Herein

Number Description Form Date

101.SCH* Inline XBRL Taxonomy Extension Schema Document

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Chief Executive Officer

POWER OF ATTORNEY

KNOW ALL PERSONS BY THESE PRESENTS, that each person whose signature appears below constitutes and appoints Christopher Anzalone and Daniel Apel, and each of them, as true and lawful attorneys-in-fact and agents, with full powers of substitution and resubstitution, for them and in their name, place and stead, in any and all capacities, to sign in any and all capacities (including, without limitation, the capacities listed below), this Annual Report on Form 10-K, any and all amendments thereto, and to file the same, with all exhibits thereto, and all other documents in connection therewith, with the Securities and Exchange Commission, and hereby grants to such attorneys-in-fact and agents, and each of them, full power and authority to do and perform each and every act and anything necessary to be done to enable the registrant to comply with the provisions of the Securities Exchange Act and all the requirements of the Securities and Exchange Commission, as fully to all intents and purposes as the undersigned might or could do in person, hereby ratifying and confirming all that said attorneys-in-fact and agents, or any of them, or their or his or her substitute, or substitutes, may lawfully do or cause to be done by virtue hereof.

Pursuant to the requirements of the Securities Exchange Act of 1934, this report on Form 10-K has been signed below by the following persons on behalf of the Registrant and in the capacities and on the dates indicated:

Signature Title Date

/s/ Christopher Anzalone Chief Executive Officer, President and Director (Principal Executive Officer) November 26, 2024

/s/ Christopher Anzalone Chief Executive Officer, President and Director, Chairman of the Board of Directors (Principal Executive Officer) November 25, 2025

Christopher Anzalone

/s/ Kenneth A.

Myszkowski Chief Financial Officer (Principal Financial and Accounting Officer) November 26, 2024

/s/ Douglass Given Director, Chairman of the Board of Directors November 26, 2024

Douglass Given

/s/ Daniel Apel Chief Financial Officer (Principal Financial and Accounting Officer) November 25, 2025

/s/ Mauro Ferrari Director November 25, 2025

Mauro Ferrari

/s/ Michael S.

Perry Director November 26, 2024

/s/ William Waddill Director November 26, 2024

William Waddill

/s/ Douglass Ingram Director November 25, 2025

Douglass Ingram

/s/ Hongbo Lu Director November 25, 2025

/s/ Adeoye Olukotun Director November 25, 2025

Adeoye Olukotun

/s/ Michael S.

Perry Director November 25, 2025

/s/ Victoria Vakiener Director November 25, 2025

Victoria Vakiener

/s/ Hongbo Lu Director November 26, 2024

/s/ William Waddill Director November 25, 2025

William Waddill

INDEX TO FINANCIAL STATEMENTS AND SCHEDULE

Reports of Independent Registered Public Accounting Firm (PCAOB ID:

Reports of Independent Registered Public Accounting Firm

Report of Independent Registered Public Accounting Firm

Consolidated Balance Sheets as of September 30, 202 5 and 202 4

Consolidated Statements of Operations and Comprehensive Loss for the years ended September 30, 2024, 2023 and 2022

Consolidated Statements of Operations and Comprehensive Income ( Loss ) for the years ended September 30, 202 5 , 202 4 and 202 3

Consolidated Statements of Stockholders’ Equity for the years ended September 30, 202 5 , 202 4 and 202 3

2 unchanged sentences

Report of Independent Registered Public Accounting Firm

To the Board of Directors and

Stockholders of Arrowhead Pharmaceuticals, Inc.

To the Board of Directors and Stockholders

Arrowhead Pharmaceuticals, Inc.:

Opinion on the Consolidated Financial Statements

We have audited the accompanying consolidated balance sheet of Arrowhead Pharmaceuticals, Inc.

and subsidiaries (the Company) as of September 30, 2024, the related consolidated statement of operations and comprehensive loss, stockholders’ equity, and cash flows for the year then ended, and the related notes (collectively, the consolidated financial statements).

In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company as of September 30, 2024, and the results of its operations and its cash flows for the year ended, in conformity with U.S.

We have audited the accompanying consolidated balance sheets of Arrowhead Pharmaceuticals, Inc.

and subsidiaries (the Company) as of September 30, 2025 and 2024, the related consolidated statements of operations and comprehensive income (loss), stockholders’ equity, and cash flows for each of the years in the two-year period ended September 30, 2025, and the related notes (collectively, the consolidated financial statements).

In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company as of September 30, 2025 and 2024, and the results of its operations and its cash flows for each of the years in the two-year period ended September 30, 2025, in conformity with U.S.

generally accepted accounting principles.

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These consolidated financial statements are the responsibility of the Company’s management.

Our responsibility is to express an opinion on these consolidated financial statements based on our audit.

Our responsibility is to express an opinion on these consolidated financial statements based on our audits.

We are a public accounting firm registered with the PCAOB and are required to be independent with respect to the Company in accordance with the U.S.

federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.

We conducted our audit in accordance with the standards of the PCAOB.

We conducted our audits in accordance with the standards of the PCAOB.

Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to error or fraud.

Our audit included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks.

Our audits included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks.

Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the consolidated financial statements.

Our audit also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the consolidated financial statements.

We believe that our audit provides a reasonable basis for our opinion.

Critical Audit Matter

The critical audit matter communicated below are matters arising from the current period audit of the consolidated financial statements that were communicated or required to be communicated to the audit committee and that:

Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the consolidated financial statements.

We believe that our audits provide a reasonable basis for our opinion.

Critical Audit Matters

The critical audit matters communicated below are matters arising from the current period audit of the consolidated financial statements that were communicated or required to be communicated to the audit committee and that:

(1) relate to accounts or disclosures that are material to the consolidated financial statements and (2) involved our especially challenging, subjective, or complex judgments.

The communication of critical audit matter does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing separate opinions on the critical audit matter or on the accounts or disclosures to which they relate.

The communication of critical audit matters does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matters below, providing separate opinions on the critical audit matters or on the accounts or disclosures to which they relate.

Sufficiency of audit evidence over valuation of future royalty sales liability

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The estimate of the carrying value of the liability related to the sale of future royalties is derived from the estimate of future sales of olpasiran and the probability of success assumption.

The estimate of future sales of olpasiran is based on key assumptions such as patient population, market penetration, olpasiran

sales price, and the comparable guideline drug.

The estimate of future sales of olpasiran is based on key assumptions such as patient population, market penetration, olpasiran sales price, and the comparable guideline drug.

The liability related to the sale of future royalties was $367,397 thousand as of September 30, 2025.

8 unchanged sentences

We evaluated the sufficiency of audit evidence obtained by assessing the cumulative results of the audit procedures performed and potential bias in the accounting estimate, including the appropriateness of the nature and extent of such evidence.

Evaluation of distinct performance obligations related to the license and collaboration agreement with Sarepta Therapeutics, Inc

As discussed in Notes 1 and 2 to the consolidated financial statements, the Company entered into an Exclusive License and Collaboration Agreement with Sarepta Therapeutics, Inc.

At contract inception, the Company assesses whether the goods or services promised within the contract are distinct and, therefore, represent a separate performance obligation, or whether they are not distinct and are combined with other goods and services until a distinct bundle is identified.

The Company then determines the transaction price and allocates it to each performance obligation.

The Company identified 17 performance obligations under the license and collaboration agreement with Sarepta.

Fixed consideration of $833.6 million and an estimated variable consideration of $71.2 million were allocated to all performance obligations based on their relative standalone selling price.

We identified the evaluation of distinct performance obligations, including understanding the nature and significance of the contractual obligations and their standalone selling prices, related to the license and collaboration agreement with Sarepta as a critical audit matter.

Subjective and complex auditor judgment was required to assess the Company’s identification of distinct performance obligations, including evaluating the rights and obligations described in the agreement, their benefit to the customer, and the level of modification or customization among the performance obligations.

In addition, subjective auditor judgment was required to evaluate certain significant assumptions in the discounted cash flow model used by management to determine the standalone selling prices of the distinct performance obligations, including the discount rate and certain forecasted expenses.

The audit effort associated with assessing the discount rate assumption required specialized skills and knowledge.

The following are the primary procedures we performed to address this critical audit matter.

We evaluated the design and tested the operating effectiveness of certain internal controls related to the Company’s revenue recognition process, including management’s identification of distinct performance obligations in the license and collaboration agreement and determination of the standalone selling prices.

We evaluated management’s identification of distinct performance obligations and the significance of the contractual obligations by obtaining and reading the license and collaboration agreement to gain an understanding of the contractual terms and conditions and the commitments being made in the agreement.

We conducted inquiries with personnel responsible for clinical development to understand the nature of the research and development activities specific to the clinical, preclinical, and discovery stage programs to evaluate the nature of the commitments made to the customer.

We evaluated certain forecasted expenses within the discounted cash flow model by comparing such amounts to external market and industry data.

We involved valuation professionals with specialized skills and knowledge, who assisted in the assessment of the discount rate within the discounted cash flow model by comparing it to a discount rate that was independently developed using publicly available market data for comparable companies.

We have served as the Company’s auditor since 2024.

San Diego, CA

San Diego, California

November 25, 2025

Report of Independent Registered Public Accounting Firm

To the Board of Directors and

Stockholders of Arrowhead Pharmaceuticals, Inc.

To the Board of Directors and Stockholders

Arrowhead Pharmaceuticals, Inc.:

Opinion on Internal Control Over Financial Reporting

2 unchanged sentences

In our opinion, the Company maintained, in all material respects, effective internal control over financial reporting as of September 30, 2025, based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission.

We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States) (PCAOB), the consolidated balance sheet of the Company as of September 30, 2024, the related consolidated statement of operations and comprehensive loss, stockholders’ equity, and cash flows for the year then ended, and the related notes (collectively, the consolidated financial statements), and our report dated November 26, 2024 expressed an unqualified opinion on those consolidated financial statements.

We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States) (PCAOB), the consolidated balance sheets of the Company as of September 30, 2025 and 2024, the related consolidated statements of operations and comprehensive income (loss), stockholders’ equity, and cash flows for each of the years in the two-year period ended September 30, 2025, and the related notes (collectively, the consolidated financial statements), and our report dated November 25, 2025 expressed an unqualified opinion on those consolidated financial statements.

Basis for Opinion

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Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.

San Diego, CA

San Diego, California

November 25, 2025

3 unchanged sentences

Opinion on the Financial Statements

We have audited the accompanying consolidated balance sheet of Arrowhead Pharmaceuticals, Inc., and Subsidiaries (the Company) as of September 30, 2023, and the related consolidated statements of operations and comprehensive income (loss), stockholders’ equity, and cash flows for each of the years in the two-year period ended September 30, 2023, and the related notes (collectively referred to as the financial statements).

In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company as of September 30, 2023, and the results of its operations and its cash flows for each of the years in the two-year period ended September 30, 2023, in conformity with accounting principles generally accepted in the United States of America.

We have audited the accompanying consolidated statements of operations and comprehensive income (loss), stockholders’ equity, and cash flows for period ended September 30, 2023 of Arrowhead Pharmaceuticals, Inc.

and Subsidiaries (the Company), and the related notes (collectively referred to as the financial statements).

In our opinion, the consolidated financial statements present fairly, in all material respects, the consolidated results of the Company’s operations and cash flows for the period ended September 30, 2023, in conformity with accounting principles generally accepted in the United States of America.

Basis for Opinion

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Cash, cash equivalents and restricted cash $ 88,706 $ 76,208

Cash at variable interest entity 137,842 26,477

Accounts receivable 6,824 —

Available-for-sale securities, at fair value 692,818 578,276

14 unchanged sentences

Deferred revenue 2,399 —

Credit facility 40,000 —

Other liabilities 10,811 432

3 unchanged sentences

Liability related to the sale of future royalties 367,397 341,361

Credit facility 393,183 —

Credit facility, net of current portion 214,883 393,183

Total long-term liabilities 686,392 845,571

3 unchanged sentences

Authorized 290,000 shares;

issued and outstanding 124,376 and 107,312 shares

issued and outstanding 135,702 and 124,376 shares at September 30, 2025 and 2024, respectively

Additional paid-in capital 2,139,725 1,806,000

Accumulated other comprehensive income (loss) 4,750 ( 3,222 )

Accumulated other comprehensive income 6,443 4,750

Accumulated deficit ( 1,627,154 ) ( 1,625,523 )

Treasury stock;

2,661 and 0 shares of common stock at September 30, 2025 and 2024, respectively

Total Arrowhead Pharmaceuticals, Inc.

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Arrowhead Pharmaceuticals, Inc.

Consolidated Statements of Operations and Comprehensive Loss

Consolidated Statements of Operations and Comprehensive Income (Loss)

(in thousands, except per share amounts)

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Total operating expenses 731,102 604,631 445,737

Operating loss ( 601,080 ) ( 205,002 ) ( 178,507 )

Other income (expense):

Operating income (loss) 98,346 ( 601,080 ) ( 205,002 )

Other (expense) income:

Interest income 37,289 22,720 15,299

1 unchanged sentence

Other, net 5,259 ( 1,748 ) 1,538

Total other (expense) income ( 11,380 ) ( 1,489 ) 5,798

Loss before income tax expense and noncontrolling interest ( 612,460 ) ( 206,491 ) ( 172,709 )

Income tax (benefit) expense ( 2,767 ) 2,784 3,785

Net loss including noncontrolling interest ( 609,693 ) ( 209,275 ) ( 176,494 )

Net loss attributable to noncontrolling interest, net of tax ( 10,200 ) ( 4,000 ) ( 431 )

Total other expense ( 46,813 ) ( 11,380 ) ( 1,489 )

Income (loss) before income tax expense and noncontrolling interest 51,533 ( 612,460 ) ( 206,491 )

Income tax expense (benefit) 21,419 ( 2,767 ) 2,784

Net income (loss) including noncontrolling interest 30,114 ( 609,693 ) ( 209,275 )

Net income (loss) attributable to noncontrolling interest, net of tax 31,745 ( 10,200 ) ( 4,000 )

Net loss attributable to Arrowhead Pharmaceuticals, Inc.

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Foreign currency translation adjustments ( 432 ) 4,197 ( 122 )

Comprehensive loss $ ( 601,721 ) $ ( 212,361 ) $ ( 176,561 )

Comprehensive income (loss) attributed to noncontrolling interest 31,745 ( 10,200 ) ( 4,000 )

Other comprehensive income (loss) $ 31,807 $ ( 601,721 ) $ ( 212,361 )

The accompanying notes are an integral part of these consolidated financial statements.

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Loss Accumulated

Deficit Common Stock in Treasury Amount ($) Non-

Interest Totals

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Common stock - restricted stock units vesting 913 1 ( 1 ) — — — — — —

Unrealized losses on available-for-sale securities — — — ( 2,964 ) — — — — ( 2,964 )

Foreign currency translation adjustments — — — ( 122 ) — — — — ( 122 )

Interest in joint venture — — 39,750 — — 20,250 60,000

Net loss — — — — ( 205,275 ) — — ( 4,000 ) ( 209,275 )

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Comprehensive

Loss Accumulated

(Loss) Income Accumulated

Deficit Common Stock in Treasury Amount ($) Non-

Interest Totals

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Common stock - restricted stock units vesting 1,048 1 ( 1 ) — — — — — —

Unrealized losses on available-for-sale securities — — — ( 2,964 ) — — ( 2,964 )

Common stock issued, net of offering costs 15,790 16 429,249 — — — — — 429,265

Unrealized gains on available-for-sale securities — — — 3,775 — — — — 3,775

Foreign currency translation adjustments — — — 4,197 — — — — 4,197

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Comprehensive

Loss Accumulated

Income Accumulated

Deficit Common Stock in Treasury Amount ($) Non-

Interest Totals

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Common stock - restricted stock units vesting 1,526 2 — — — — — — 2

Common stock issued, net of offering costs 15,790 16 429,249 — — — 429,265

Common stock issued 11,926 12 241,376 — — — — — 241,388

Common stock - repurchase — — — — — ( 2,661 ) ( 53,193 ) — ( 53,193 )

Issuance of pre-funded warrants — — 25,000 — — — — — 25,000

Unrealized gains on available-for-sale securities — — — 2,125 — — — — 2,125

Foreign currency translation adjustments — — — ( 432 ) — — — — ( 432 )

Net loss — — — — ( 599,493 ) ( 10,200 ) ( 609,693 )

Net (loss) income — — — — ( 1,631 ) — — 31,745 30,114

Balance at September 30, 2025 138,363 $ 231 $ 2,139,725 $ 6,443 $ ( 1,627,154 ) ( 2,661 ) $ ( 53,193 ) $ 37,364 $ 503,416

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CASH FLOWS FROM OPERATING ACTIVITIES:

Net loss $ ( 609,693 ) $ ( 209,275 ) $ ( 176,494 )

Adjustments to reconcile net loss to net cash flow from operating activities:

Net income (loss) $ 30,114 $ ( 609,693 ) $ ( 209,275 )

Adjustments to reconcile net income (loss) to net cash flow from operating activities:

Stock-based compensation 63,366 73,968 78,130

Depreciation and amortization 23,928 18,595 12,493

(Accretion) Amortization of note premiums/discounts ( 3,244 ) ( 2,017 ) 2,910

Accretion of note premiums/discounts ( 5,789 ) ( 3,244 ) ( 2,017 )

Non-cash interest expense on liability related to the sale of future royalties 26,036 23,035 18,326

Non-cash interest expense on credit facility 63,325 9,317 —

Non-cash gain on treasury stock received ( 3,193 ) — —

Realized loss on investments — 80 —

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Other 3,094 ( 1,200 ) —

Net cash used in operating activities ( 462,851 ) ( 153,890 ) ( 136,131 )

Net cash provided by (used in) operating activities 179,552 ( 462,851 ) ( 153,890 )

CASH FLOWS FROM INVESTING ACTIVITIES:

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CASH FLOWS FROM FINANCING ACTIVITIES:

Proceeds from the exercises of stock options 3,983 2,389 3,053

Proceeds from the issuance of common stock, net of offering costs — 429,265 —

Proceeds from the sale of future royalties — 50,000 250,000

Proceeds from credit facility 392,000 — —

Proceeds from the issuance of warrants 25,000 — —

Payment of debt issuance costs ( 5,000 ) ( 3,134 ) —

Proceeds from the exercises of stock options 2,389 3,053 5,186

Proceeds from investment in joint venture — — 60,000

Proceeds from the issuance of common stock 241,388 — —

Proceeds from credit facility — 392,000 —

Repayments of credit facility ( 201,625 ) — —

Proceeds from Visirna credit agreement 10,260 — —

Net cash provided by financing activities 74,006 870,520 253,053

Net (decrease) increase in cash, cash equivalents and restricted cash ( 12,403 ) 3,008 ( 76,362 )

Net increase (decrease) in cash, cash equivalents and restricted cash 124,264 ( 12,403 ) 3,008

Effect of exchange rate on cash, cash equivalents and restricted cash ( 401 ) 4,197 ( 122 )

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Supplementary disclosure of cash flows:

Income Taxes Paid $ ( 3,744 ) $ — $ ( 2 )

Interest paid $ ( 19 ) $ — $ —

Income taxes (refund) paid $ 814 $ ( 3,744 ) $ —

Treasury stock received to settle accounts receivable $ 50,000 $ — $ —

Supplementary disclosure of non-cash investing activities:

Capital expenditures included in accounts payable and accrued expenses $ 4,206 $ 14,044 $ 17,578

Supplementary disclosure of non-cash financing activities:

Debt issuance costs included in accrued expenses $ 5,000 $ — $ —

Capital expenditures included in accrued expenses $ 277 $ 4,206 $ 14,044

The accompanying notes are an integral part of these consolidated financial statements.

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Phase 3 Arrowhead (1)

Phase 2b Arrowhead

zodasiran Phase 3 Arrowhead

olpasiran Phase 3 Amgen

Pulmonary ARO-RAGE Phase 1/2a

ARO-MUC5AC Phase 1/2a Arrowhead

ARO-MMP7 Phase 1/2a

Liver GSK-4532990 Phase 2b GSK

fazirsiran Phase 3 Takeda and Arrowhead

ARO-PNPLA3 Phase 1 Arrowhead

ARO-INHBE Phase 1/2a Arrowhead

ARO-ALK7 Phase 1/2a Arrowhead

Phase 1/2a Arrowhead

Pulmonary ARO-RAGE Phase 1/2a Arrowhead

SRP-1002 (ARO-MMP7) Phase 1/2a Sarepta

Liver fazirsiran Phase 3 Takeda and Arrowhead

daplusiran/tomligisiran

ARO-PNPLA3 Phase 1

ARO-C3 Phase 1/2a

ARO-CFB Phase 1/2a

ARO-DUX4 Phase 1/2a

ARO-DM1 Phase 1/2a

Central Nervous System (CNS)

ARO-ATXN2 Phase 1/2a

Neuromuscular SRP-1001 (ARO-DUX4) Phase 1/2a Sarepta

SRP-1003 (ARO-DM1) Phase 1/2a Sarepta

SRP-1004 (ARO-ATXN2) Phase 1/2a Sarepta

Other ARO-C3 Phase 1/2a Arrowhead

ARO-CFB Phase 1/2a Arrowhead

(1) Greater China rights for plozasiran are out-licensed to Sanofi.

The Company operates lab facilities in California and Wisconsin, where its research and development activities, including the development of RNAi therapeutics, take place.

The Company also operates an active pharmaceutical ingredient manufacturing and supporting laboratory facility in Verona, Wisconsin.

The Company’s principal executive offices are located in Pasadena, California.

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and its subsidiaries (wholly-owned subsidiaries and a variable interest entity for which the Company is the primary beneficiary).

Subsidiaries refer to Arrowhead Madison, Inc., Visirna Therapeutics, Inc.

(“Visirna”), and Arrowhead Australia Pty Ltd.

Subsidiaries refer to Arrowhead Madison, Inc., Arrowhead Australia Pty Ltd., Arrowhead Pharmaceuticals Ireland Limited, Arrowhead Pharmaceuticals NZ Limited, and Visirna Therapeutics, Inc.

For subsidiaries in which the Company owns or is exposed to less than 100% of the economics, the Company records net loss attributable to noncontrolling interests in its consolidated statements of operations equal to the percentage of the economic or ownership interests retained in such entity by the respective noncontrolling party.

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All intercompany transactions and balances have been eliminated.

Certain prior period amounts have been reclassified to conform with the current period presentation.

The Company operates as a single segment as the chief operating decision maker, or CODM, reviews operating results on an aggregate basis and manages the operations as a single operating segment.

Refer to Note 16, Segment Reporting , for further details on the segment information.

The Company’s primary sources of financing have been through the sale of its equity securities, credit facility, revenue from its licensing and collaboration agreements and the sale of certain future royalties.

Research and development activities have required significant investment since the Company’s inception and are expected to continue to require significant cash expenditure in the future, particularly as the Company’s pipeline of drug candidates and its headcount have

both expanded.

Additionally, significant capital investment will be required as the Company’s pipeline matures into later stage clinical trials, including commercialization efforts.

At September 30, 2024, the Company had $ 102.7 million in cash, cash equivalents and restricted cash ($ 3.5 million in restricted cash) and $ 578.3 million in available-for-sale securities to fund operations.

During the year ended September 30, 2024, the Company’s cash, cash equivalents and restricted cash and investments balance increased by $ 277.3 million.

This increase was primarily driven by net proceeds from the credit facility and the underwritten offering as discussed below, offset by ongoing expenses related to the Company’s research and development programs, general and administrative costs, and capital expenditures.

On August 7, 2024, the Company entered into a financing agreement (the “Financing Agreement”) with Sixth Street Lending Partners, as representatives of several lenders.

The Financing Agreement provides for a senior secured term loan facility of $ 500 million, which includes $ 400.0 million funded on the closing date with an additional $ 100.0 million at the Company’s option during the seven-year term.

The Company received net proceeds of $ 388.9 million, after issuance costs as of September 30, 2024.

On January 2, 2024, the Company entered into an underwriting agreement with Jefferies LLC, BofA Securities, Inc., and Cowen and Company, LLC, as representatives of the several underwriters.

The Company issued 15,790,000 shares of common stock at an offering price of $ 28.50 per share.

The aggregate purchase price paid by investors was $ 450.0 million, and the Company received net proceeds of $ 429.3 million after deducting advisory fees and offering expenses.

In total, the Company is eligible to receive up to $ 2.7 billion in developmental, regulatory and sales milestones, and may receive various royalties on net sales from its licensing and collaboration agreements, subject to the terms and conditions of those agreements.

The revenue recognition for these collaboration agreements is discussed further in Note 2.

Additionally, significant investment will be required as the Company’s pipeline matures into later stage clinical trials and commercialization efforts.

As of September 30, 2025, the Company had $ 226.5 million in cash, cash equivalents and restricted cash ($ 1.9 million in restricted cash) and $ 692.8 million in available-for-sale securities to fund operations.

During the year ended September 30, 2025, the Company’s cash, cash equivalents and restricted cash and investments balance increased by $ 238.4 million, which was primarily due to the $ 500.0 million as an upfront payment under the Sarepta agreement and $ 325.0 million in the form of an equity investment under the Sarepta agreement, $ 100.0 million relating to the achievement of partnership milestone achievement of which $ 50.0 million was settled in cash and the remaining $ 50.0 million was settled through the repurchase of Company's common stock , $ 37.3 million interest income earned on investments, and $ 25.0 million in the form of pre-funded warrants, partially offset by ongoing expenses related to the Company’s research and development programs, $ 201.6 million payments on its credit facility and $ 22.7 million relating to capital expenditure.

In total, the Company is eligible to receive up to $ 13.4 billion in additional developmental, regulatory and sales milestones, and may receive various royalties on net sales from its licensing and collaboration agreements, subject to the terms and conditions of those agreements.

Summary of Significant Accounting Policies

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All highly liquid interest-bearing investments are classified as cash equivalents.

These investments mainly include commercial paper with maturities of three months or less when purchased.

These investments mainly include term deposits, money market instruments, corporate debt securities, and certificate of deposits with maturities of three months or less when purchased.

The carrying value of these cash equivalents approximate fair value.

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The Company classified all of its investments in debt securities as available-for-sale and as current assets as they represent the investment of funds available for current operations as of September 30, 2025 and 2024.

The available-for-sale investments may consist of investment-grade interest bearing instruments, primarily money market accounts, government-sponsored enterprise securities, corporate bonds and/or commercial paper, which are accounted for at fair value.

The available-for-sale investments may consist of investment-grade interest bearing instruments, primarily corporate debt securities, U.S.

government and agency securities, commercial notes, certificate of deposits, and municipal securities, which are accounted for at fair value.

Dividends from these funds were automatically re-invested.

Changes in fair values are reported as unrealized gains or losses and are recorded in the Company’s consolidated statement of operations and comprehensive loss.

The Company evaluates its investments for impairment.

If an unrealized loss is determined to be other-than-temporary, it is written off as a realized loss through the consolidated statements of operations and comprehensive loss.

The Company’s methodology of assessing other-than-temporary impairments is based on security-specific analysis as of the balance sheet date and considers various factors, including the length of time to maturity and the extent to which the fair value has been less than the cost, recoverability of future cash flows as compared to carrying value of the security, the financial condition and the near-term prospects of the issuer, and the Company’s ability and intent to hold the security.

If a decline in fair value of investments is determined to be other-than-temporary, the securities are written down to fair value

as the new cost basis and the amount of the write down is accounted for as realized losses.

The Company did not recognize any other-than-temporary impairments of its investment for the years ended September 30, 2024, 2023, and 2022.

Changes in fair values are reported as unrealized gains or losses and are recorded in the Company’s consolidated statement of operations and comprehensive income (loss).

The Company evaluates its investments for impairment based on a security-specific analysis as of each balance sheet date.

If the fair value of a security is below its amortized cost, the Company first assesses whether it intends to sell the security or is more likely than not required to sell it before recovery of its amortized cost.

If neither condition is met, the Company evaluates whether a portion of the decline is attributable to credit loss.

Any credit-related impairment is recorded as an allowance for credit losses through earnings, with non-credit-related unrealized losses recorded in other

comprehensive income (loss).

The Company did not recognize any credit loss relating to its investment for the years ended September 30, 2025, 2024, and 2023.

Concentration of Credit Risk

Financial instruments that potentially expose the Company to concentration of credit risk primarily consist of cash, cash equivalents and restricted cash and investments.

As of September 30, 2024 and 2023, the Company’s investments were primarily invested in money market funds, commercial paper, and corporate debt securities through highly rated financial institutions.

As of September 30, 2025 and 2024, the Company’s investments were primarily invested in money market funds, U.S.

Government, commercial paper, and corporate debt securities and term deposit through highly rated financial institutions.

The Company has established guidelines relative to diversification and maturities that maintain safety and liquidity.

The Company periodically reviews and modifies these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity.

The Company also maintains several bank accounts primarily at two financial institutions for its operations.

The Company also maintains several bank accounts at two financial institutions for its operations.

These accounts are insured by the Federal Deposit Insurance Corporation (FDIC) for up to $250,000 per institution.

In addition, Visirna maintains several deposit and term accounts in mainland China, Hong Kong, and Singapore.

Cash balances are held with various local and international financial institutions, which are subject to their respective jurisdictional deposit insurance programs.

The Deposit Insurance Fund Management Corporation in China provides coverage of up to RMB 500,000 per depositor per institution, and the Hong Kong Deposit Protection Scheme provides protection of up to HKD 800,000 per depositor per bank, and the Singapore Deposit Insurance Corporation provides coverage of up to SGD 100,000 per depositor per member bank.

Property, Plant and Equipment

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Depreciation expense is recorded on a straight-line basis over the estimated useful lives of the assets.

Leasehold improvements are amortized over the lesser of the expected useful life or the remaining lease term.

Leasehold improvements are amortized over the shorter of the asset life or lease term.

Construction in progress reflects amounts incurred for construction or improvements of property, plant and equipment that have not been placed in service.

Upon disposition, the cost and accumulated depreciation of assets retired or sold are removed from the respective asset category, and any gain or loss is recognized in the Company’s consolidated statement of operations and comprehensive loss.

The estimated useful lives of property, plant and equipment are as follows (in years):

Estimated Useful Lives

Research equipment 5 to 10

Research equipment 5

Manufacturing equipment

Computers and software 3 to 5

Leasehold improvements 3 to 15

Leasehold improvements Shorter of asset life or lease term

The Company periodically assesses long-lived assets or asset groups, including property, plant and equipment, for recoverability when events or changes in circumstances indicate that their carrying amounts may not be recoverable.

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Accrual estimates may be based on vendor communications to obtain pending invoices and/or estimates for services performed during the period.

In some cases, these estimates require judgment, drawing on an understanding of research and development programs, services provided during the period, prior experience, and, where applicable, the expected duration of third-party contracts.

In some cases, these estimates require significant judgment, drawing on an understanding of research and development programs, services provided during the period, prior experience, and, where applicable, the expected duration of third-party contracts.

Actual costs upon settlement may differ significantly from the accrued amounts in the Company’s consolidated financial statements, though historical estimates have not differed materially from actual costs.

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At the end of each subsequent reporting period, the Company re-evaluates the probability of a significant reversal of the cumulative revenue recognized for its milestones and royalties, and, if necessary, adjusts its estimate of the overall transaction price.

Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and net income in the Company’s consolidated statements of operations and comprehensive loss.

Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and net income in the Company’s consolidated statements of operations and comprehensive income (loss).

Typically, milestone payments and royalties are achieved after the Company’s performance obligations associated with the collaboration agreements have been completed and after the customer has assumed responsibility for the respective clinical or preclinical program.

Milestones or royalties achieved after the Company’s performance obligations have been completed are recognized as revenue in the period the milestone or royalty was achieved.

If a milestone payment is achieved during the performance period, the milestone payment would be recognized as revenue to the extent performance had been completed at that point, and the remaining balance would be recorded as deferred revenue.

If a milestone payment is achieved during the performance period, the milestone payment would be

recognized as revenue to the extent performance had been completed at that point, and the remaining balance would be recorded as deferred revenue.

The revenue standard requires the Company to assess whether a significant financing component exists in determining the transaction price.

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If other observable transactions in which the Company has sold the same performance obligation separately are not available, the Company estimates the standalone selling price of each performance obligation.

Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success.

Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines and costs, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success.

Whenever the Company determines that goods or services promised in a contract should be accounted for as a combined performance obligation over time, the Company determines the period over which the performance obligations will be performed and revenue will be recognized.

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Research and Development Expenses

Costs and expenses that can be clearly identified as research and development are charged to expense as incurred.

Research and development costs are charged to expense as incurred.

Included in research and development costs are operating costs, facilities, supplies, external services, clinical trial and manufacturing costs, overhead directly related to the Company’s research and development operations, and costs to acquire technology licenses.

Stock-Based Compensation

Share-based compensation expenses for all stock grants are based on their estimated grant-date fair value.

Share-based compensation expense for all stock grants are based on their estimated grant-date fair value.

The fair value of stock option awards is estimated using the Black-Scholes option valuation model which requires the input of subjective assumptions to calculate the value of stock options.

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For restricted stock units, the value of the award is based on the Company’s stock price at the grant date.

For performance-based restricted stock unit awards, the value of the award is based on the Company’s stock price at the grant date, with consideration given to the probability of the performance condition being achieved.

For performance-based restricted stock unit awards, the value of the award is based on the Company’s stock price at the grant date, with consideration given to the

probability of the performance condition being achieved.

Expense is recognized over the vesting period for all awards and commences at the grant date for time-based awards and upon the Company’s determination that the achievement of such performance conditions is probable for performance-based awards.

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the design of Visirna, including its capitalization structure, subordination of interests, payment priority, and the reasons why the interests are held by the Company.

At Visirna’s inception, the Company determined whether it was the primary beneficiary and if Visirna should be consolidated based on the facts and circumstances.

The Company performs ongoing reassessments of the VIE based on reconsideration events and reevaluates whether a change to the consolidation is required.

At Visirna’s inception, the Company determined it was the primary beneficiary and that Visirna should be consolidated based on the facts and circumstances.

The Company performs ongoing reassessments of the VIE based on reconsideration events and reevaluates whether a change to the consolidation conclusion is required.

As of September 30, 2025, there were no events to be reconsidered in the consolidation.

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Foreign Currency Translation Adjustments

One of the Company’s wholly-owned subsidiaries’ functional currencies is not the United States dollar, which is the Company’s reporting currency.

Three of the Company’s wholly-owned subsidiaries’ functional currencies is not the United States dollar, which is the Company’s reporting currency.

Assets and liabilities are translated at the exchange rate in effect at the balance sheet date.

Revenues and expenses are translated at the average rate of exchange prevailing during the reporting period.

Translation adjustments arising from the use of different exchange rates from period to period are included in the accumulated other comprehensive loss.

Translation adjustments arising from the use of different exchange rates from period to period are included in the accumulated other comprehensive income (loss).

Segment Information

The Company operates as a single segment because its chief decision makers review operating results on an aggregate basis and manage its operations as a single operating segment.

The Company operates as a single segment because its CODM reviews operating results on an aggregate basis and manages its operations as a single operating segment.

Recent Accounting Pronouncements

In January 2025, the Financial Accounting Standards Board (“FASB”) issued Accounting Standard Update (“ASU”) 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures (Subtopic 220-40):

Disaggregation of Income Statement Expenses, in November 2024, and ASU 2025-01, Clarifying the Effective Date .

These updates require entities to provide disaggregated disclosures of income statement expenses.

The ASUs do not affect the expense captions presented on the face of the income statement but instead require the disaggregation of certain expense captions into specified categories within the footnotes to the financial statements.

The ASUs will become effective for the Company beginning October 1, 2027, and the Company is currently evaluating the impact on its consolidated financial statements and related disclosures.

In December 2023, the FASB issued Accounting Standard Update (“ASU”) 2023-09, Income Taxes (Topic 740):

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Under the guidance, entities must annually (1) disclose specific categories in the rate reconciliation and (2) provide additional information for reconciling items that meet a quantitative threshold.

This guidance will become effective for the Company beginning on October 1, 2025.

This guidance became effective for the Company beginning on October 1, 2025.

The Company does not expect any material impact on its consolidated financial statements and related disclosures resulting from applying this ASU.

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The amendments should be applied retrospectively to all prior periods presented in the financial statements.

The Company does not expect any material impact on its consolidated financial statements and related disclosures resulting from applying this ASU.

This ASU is applicable to the Company’s Annual Report on Form 10-K for the year ending September 30, 2025, and subsequent interim periods.

Refer to Note 16, Segment Reporting , for further details on segment information.

On July 4, 2025, the One Big Beautiful Bill Act (“OBBBA”) was enacted in the U.S.

The OBBBA includes significant provisions, such as the permanent extension of certain expiring provisions of the Tax Cuts and Jobs Act, modifications to the international tax framework and the restoration of favorable tax treatment for certain business provisions.

The legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented through 2027.

ASC 740, "Income Taxes", requires the effects of changes in tax rates and laws to be recognized in the period in which the legislation is enacted.

The Company has implemented OBBBA in the fourth quarter of fiscal 2025.

Refer to Note 11, Income Taxes , for further details.

COLLABORATION AND LICENSE AGREEMENTS

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Amgen — — 25,000

Sarepta 696,803 — —

Sanofi $ 130,000 $ — $ —

Total $ 829,448 $ 3,551 $ 240,735

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GSK is wholly responsible for all clinical development and commercialization of GSK-4532990 in its territory.

The Company determined the initial transaction price totaled $ 120.0 million, including the upfront payment, which was collected in January 2022.

The Company has completed its performance obligation related to this agreement, and the upfront payment of $ 120.0 million was fully recognized in the year ended September 30, 2022.

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The Company is eligible for an additional payment of $ 100.0 million upon achieving the first patient dosed in a Phase 3 trial.

Furthermore, should the Phase 3 trial read out positively, and the potential new medicine receives regulatory

approval in major markets, the deal provides for commercial milestone payments to the Company of up to $ 190.0 million at first commercial sale, and up to $ 590.0 million in sales-related milestone payments.

Furthermore, should the Phase 3 trial read out positively, and the potential new medicine receives regulatory approval in major markets, the deal provides for commercial milestone payments to the Company of up to $ 190.0 million at first commercial sale, and up to $ 590.0 million in sales-related milestone payments.

The Company is further eligible to receive tiered royalties on net product sales in a range of mid-teens to twenty percent.

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On December 11, 2023, the Company entered into an Amended and Restated License Agreement with GSK (the “GSK-HBV Agreement”) pursuant to which GSK received a worldwide, exclusive license to develop and commercialize daplusiran/tomligisiran (GSK5637608, formerly JNJ-3989), the Company’s third-generation subcutaneously administered RNAi therapeutic candidate being developed as a potential therapy for patients with chronic hepatitis B virus infection.

GSK5637608 had previously been licensed to Janssen in October 2018.

Under the terms of the GSK-HBV Agreement, the Company received $ 2.7 million in December 2023, upon signing the amended GSK-HBV Agreement.

Further, GSK dosed the fifth patient in a Phase 2 trial in December 2024, triggering a $ 2.5 million milestone payment to the Company which was paid in the second quarter of fiscal 2025.

The Company is eligible to receive up to $ 830.0 million in development and sales milestone payments under the GSK-HBV Agreement.

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Under the terms of the Horizon License Agreement, Horizon received a worldwide exclusive license for HZN-457, a clinical-stage medicine being developed by Horizon as a potential treatment for people with uncontrolled gout.

At the inception of the Horizon License Agreement, the Company identified one distinct performance obligation.

The Company determined that the key deliverables included the license and certain R&D services, including the Company’s responsibilities to conduct all activities through the preclinical stages of development of HZN-457 (the “Horizon R&D Services”).

The Company received a $ 40.0 million upfront payment in July 2021.

Revenue was recognized on a straight-line basis, which corresponded to the timeframe for completing the Horizon R&D Services, concluding in the first quarter of 2023.

Further, the Company received an additional $ 15.0 million upon Horizon’s initiation of a Phase 1 clinical trial in January 2023.

On October 6, 2023, Amgen completed its acquisition of Horizon and subsequently notified the Company of Amgen’s intent to terminate the HZN-457 license.

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Outside the United States, Takeda received an exclusive license to commercialize fazirsiran and will lead the global commercialization strategy, while the Company will be eligible to receive tiered royalties of 20 % to 25 % on net sales.

At the inception of the Takeda License Agreement, the Company identified one distinct performance obligation.

The Company determined that the key deliverables included the license and certain R&D services including the Company’s responsibilities to complete the initial portion of the SEQUOIA study, to complete the ongoing Phase 2 AROAAT2002 study and to ensure certain manufacturing of fazirsiran drug product is completed and delivered to Takeda (the “Takeda R&D Services”).

The Company determined that the key deliverables included the license and certain research and development services including the Company’s responsibilities to complete the initial portion of the SEQUOIA study, to complete the ongoing Phase 2 AROAAT2002 study, and to ensure certain manufacturing of fazirsiran drug product is completed and delivered to Takeda (the “Takeda R&D Services”).

Due to the specialized and unique nature of these Takeda R&D Services and their direct relationship with the license, the Company determined that these deliverables represent one distinct bundle and, thus, one performance obligation.

Beyond the Takeda R&D Services, which are the responsibility of the Company, Takeda will be responsible for managing future clinical development and commercialization outside the United States.

Within the United States, the Company will also participate in co-development and co-commercialization efforts and will co-fund these efforts with Takeda as part of the 50/50 profit sharing structure within the United States.

Takeda is responsible for managing clinical development and commercialization outside the United States.

Within the United States, the Company and Takeda are responsible in the co-development and co-commercialization efforts.

The Company considers the collaborative activities, including the co-development and co-commercialization, to be a separate unit of account within Topic 808, and as such, these co-funding amounts are recorded as research and development expenses or general and administrative expenses, as appropriate.

in March 2023, and is eligible to receive up to $ 527.5 million in additional potential development, regulatory and commercial milestones.

Under the terms of the Takeda License Agreement, the Company received $ 300.0 million as an upfront payment in January 2021 and an additional $ 40.0 million upon Takeda’s initiation of a Phase 3 REDWOOD clinical study of fazirsiran in March 2023, and is eligible to receive up to $ 527.5 million in additional potential development, regulatory and commercial milestones.

The Company allocated the total $ 300.0 million initial transaction price to its one distinct performance obligation for the fazirsiran license and the associated Takeda R&D Services.

Revenue was recognized using the input method (based on actual patient visits completed versus total estimated visits completed for the ongoing SEQUOIA and AROAAT2002 clinical studies).

The Phase 2 study visits for patients in the SEQUOIA and AROAAT2002 studies concluded by December 31, 2023, and the Company has substantially completed its performance obligation under the Takeda License Agreement.

The Company has substantially completed its performance obligation under the Takeda License Agreement by December 31, 2023.

As such, all revenue has been fully recognized as of December 31, 2023.

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On April 7, 2023, Janssen voluntarily terminated its collaboration agreement with the Company and the Company regained full rights to ARO-PNPLA3, formerly called JNJ-75220795.

ARO-PNPLA3 is in Phase 1 clinical trials, which are now being developed by the Company.

Further, on December 11, 2023, the Company entered into the GSK-HBV Agreement, as discussed above, pursuant to which GSK received an exclusive license for daplusiran/tomligisiran (GSK5637608, formerly JNJ-3989).

Daplusiran/tomligisiran had previously been licensed to Janssen in October 2018.

There are no currently active trials for ARO-PNPLA3.

Further, on December 11, 2023, the Company entered into the GSK-HBV Agreement, as discussed above, pursuant to which GSK received an exclusive license for JNJ-3989 (formerly ARO-HBV).

JNJ-3989 had previously been licensed to Janssen in October 2018.

In September 2016, Amgen and the Company entered into two collaboration and license agreements and a common stock purchase agreement.

2 unchanged sentences

Under the Olpasiran Agreement, Amgen is wholly responsible for clinical development and commercialization.

Under the Olpasiran Agreement, the Company has received $ 35.0 million in upfront payments and $ 21.5 million in the form of an equity investment by Amgen in the Company’s common stock.

Further, the Company received an additional $ 55.0 million in milestone payments;

$ 10.0 million upon Amgen’s initiation of a Phase 1 study in September 2018, $ 20.0 million upon its initiation of a Phase 2 clinical study in July 2020, and $ 25.0 million upon its first subject enrollment in a Phase 3 trial in December 2022.

The Company has substantially completed its performance obligations under the Olpasiran Agreement.

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In November 2022, Royalty Pharma Investments 2019 ICAV (“Royalty Pharma”) and the Company entered into a Royalty Purchase Agreement with Royalty Pharma (the “Royalty Pharma Agreement”).

Pursuant to the Royalty Pharma Agreement, Royalty Pharma paid an upfront amount of $ 250.0 million during the first quarter of fiscal 2023.

An additional milestone payment of $ 50.0 million was paid during the third quarter of fiscal 2024 due to the completed enrollment of the Phase 3 OCEAN(a) outcomes trial for olpasiran.

In consideration for the payments under the Royalty Pharma Agreement, Royalty Pharma is entitled to receive all royalties otherwise payable by Amgen to the Company under the Olpasiran Agreement.

The Company remains eligible to receive up to an additional $ 485.0 million in remaining development, regulatory and sales milestone payments payable from Amgen and Royalty Pharma.

Sarepta Therapeutics, Inc.

On November 25, 2024, the Company entered into an Exclusive License and Collaboration Agreement (the “Sarepta Collaboration Agreement”) with Sarepta for the development and commercialization of multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system, and lungs.

The Company concurrently entered into a Stock Purchase Agreement (the “Stock Purchase Agreement”) with Sarepta (see Note 6).

Under the Sarepta Collaboration Agreement, Sarepta received an exclusive sublicensable worldwide license to SRP-1001 (formerly ARO-DUX4), SRP-1003 (formerly ARO-DM1), SRP-1002 (formerly ARO-MMP7), and SRP-1004 (formerly ARO-ATXN2) clinical stage programs (the “C1” programs).

Sarepta also received an exclusive sublicensable

worldwide license to the Company’s ARO-HTT, ARO-ATXN1, and ARO-ATXN3 preclinical stage programs (the “C2” programs).

The Company will perform certain research and development activities for the C1 and C2 programs.

Further, Sarepta may select up to six gene targets for which the Company will perform discovery, optimization and preclinical development activities to identify RNAi compounds against each selected target (the “C3” programs).

Upon target acceptance, Sarepta will receive an exclusive license to the Company’s intellectual property rights to exploit compounds directed to those targets and is wholly responsible for clinical development and commercialization of each compound after the Company delivers a Clinical Trial Application ready data package (the "CTA package").

The Company identified 17 performance obligations under the Sarepta Collaboration Agreement.

The four C1 licenses are distinct performance obligations from the four C1 research and development performance obligations since the customer can use and benefit from the licenses separately.

The performance obligations for the licenses were satisfied in the second quarter of fiscal 2025 upon delivery and the research and development performance obligations will be satisfied as the work is performed.

The remaining nine performance obligations include three C2 preclinical stage program licenses and research and development activities, and six C3 unidentified discovery target licenses and research and development activity.

Each of the three C2 programs and the six C3 programs were determined to represent one performance obligation, as the customer cannot benefit from the use of the product license at the point of transfer until the specified research and development activities are performed.

As such, each of the C2 and C3 product licenses and respective research and development work will be combined to form one performance obligation.

For these nine performance obligations, revenue is recognized over time as the work is performed.

For performance obligations recognized over time, the estimated performance period over which revenue will be recognized is determined to be the period over which the Company estimates it will perform the research and development activities.

The Company determined that the most appropriate method of measuring progress for these performance obligations is an input method based on research and development costs in the program budget.

Accordingly, the Company has estimated the total cost required to complete its obligation and recognized an amount of revenue equal to the proportion of services performed, which is reassessed on an ongoing basis as the program progresses.

In the period an agreement expires or is terminated, remaining deferred revenue, if any, is recognized as revenue.

Under the terms of the Sarepta Collaboration Agreement, the Company received an upfront payment of $ 500.0 million on February 14, 2025.

In addition, on February 7, 2025, the Company received $ 325.0 million in the form of an equity investment under the Stock Purchase Agreement.

Based upon the Company's share price on February 7, 2025, (the “Closing Date”), the difference between the $ 325.0 million and the fair value of the shares on the Closing date resulted in a premium of $ 83.6 million.

The premium is included as part of the total consideration of the Sarepta Collaboration Agreement for revenue recognition purposes.

The Company is entitled to receive $ 250.0 million to be paid in annual installments of $ 50.0 million over the first five years of the agreement.

The Company is also eligible receive reimbursement of certain costs related to carrying out the research and development activities for the C1 programs.

The fixed consideration of $ 833.6 million and an estimated variable consideration of $ 71.2 million for a total of $ 904.9 million were allocated to all performance obligations based on their relative standalone selling price.

Standalone selling prices for the product licenses were determined using an adjusted market-based approach through the net present value of the expected future cash flows for each program.

The standalone selling prices for the research and development work were determined based on an expected cost plus margin approach.

The Company estimates the stand-alone selling price for each distinct performance obligation, which involves assumptions that may require significant judgment.

The Company’s estimates of the stand-alone selling price for license-related performance obligations includes forecasted revenues and expenses, phase dates, probability of success, development timelines, and the discount rate.

The estimates of the stand-alone selling price for research and development performance obligations generally include forecasting the expected costs of satisfying a performance obligation at market rates.

The Company identified a discount based on the difference between the aggregate stand-alone selling price and the transaction price for accounting revenue recognition purposes.

The Company allocated the discount proportionally to each of the performance obligations based upon their standalone selling price.

The Company receives reimbursement of certain costs related to carrying out the research and development activities for the C1 programs and may receive development milestone payments of up to $ 300.0 million.

Further, for each of the 13 programs, the Company is eligible to receive regulatory milestone payments between $ 110.0 million and $ 180.0 million per program.

Variable consideration associated with the milestones that may be achieved will be allocated to the performance obligation to which it is determined to be related, which will be the respective development work that is being reimbursed and the respective programs to which the milestones relate.

ARO-DM1 development milestones were allocated between the license and development work based on the allocation of the standalone selling price.

The Company will recognize the ARO-DM1 development milestones and other development milestones as revenue in the periods the underlying milestone events are achieved as achievement of the milestone events are highly susceptible to factors outside of the entity's influence and therefore there is a possibility that the milestone event will not be achieved.

The Company is also eligible to receive sales milestone payments between $ 500.0 million and $ 700.0 million per program as well as tiered royalties on net sales of licensed products of up to the low double digits, subject to the terms and conditions of the Sarepta Collaboration Agreement.

The Company has applied the sales-based scope exception to the sales milestones and the royalty-based payments.

The Sarepta Collaboration Agreement commenced in February 2025 and may be terminated by either party in the event of a material breach as defined therein.

In addition, Sarepta may voluntarily terminate the Sarepta Collaboration Agreement with 30 days' written notice to the Company if terminated prior to any regulatory approval of a licensed product.

Unless earlier terminated, the Sarepta Collaboration Agreement expires on a product-by-product and country-by-country basis, upon the date of expiration of the relevant royalty term for such product in such country.

In August 2025, the Company repurchased 2,660,989 shares of its common stock from Sarepta in connection with the $ 100.0 million DM1 first development milestone under the Sarepta Collaboration Agreement.

The repurchase satisfied $ 50.0 million of the milestone payment through delivery of the Company’s common stock, with the remaining $ 50.0 million settled in cash.

The shares were recorded as treasury stock at their fair value of $ 53.2 million, resulting in a $ 3.2 million gain on settlement.

The repurchased shares are presented as a reduction to total stockholders’ equity in accordance with ASC 505-30.

As of September 30, 2025, the Company recorded $ 696.8 million in revenue from Sarepta and $ 6.8 million in accounts receivable.

The recognition of the remaining revenue for the performance obligations is dependent upon the time it takes to complete the respective research and development activities and in consideration of the timing of the selection of the C3 programs.

Visirna Therapeutics Inc.

(“Visirna”) and Genzyme Corporation (“Sanofi”)

On August 1, 2025, Visirna Therapeutics HK Limited (“Visirna HK”), a wholly owned subsidiary of Visirna Therapeutics, Inc, a majority owned subsidiary of the Company, entered into an Asset Purchase Agreement (the “Asset Purchase Agreement”) with Genzyme Corporation (“Sanofi”), a wholly owned subsidiary of Sanofi S.A., pursuant to which Visirna HK sold all of its assets and rights in investigational plozasiran to Sanofi, which included an assignment of Visirna HK’s rights (as successor by assignment from Visirna) to develop and commercialize investigational plozasiran in Greater China pursuant to that certain License Agreement by and between the Company and Visirna dated, April 25, 2022 (the “Visirna License Agreement”).

In connection with the Asset Purchase Agreement, the Company consented to the partial assignment of the Visirna License Agreement by Visirna HK to Sanofi (as so assigned, the “Sanofi License Agreement”), amongst other agreements between the Company and Visirna, effective as of the closing of the Asset Purchase Agreement.

This agreement was not deemed a legal sale of intellectual property from the consolidated perspective of the Company.

After giving effect to the Asset Purchase Agreement, Visirna HK retains rights to develop and commercialize in Greater China three other cardiometabolic drugs licensed to it pursuant to the Visirna License Agreement.

Upon closing of the Asset Purchase Agreement, Visirna received an upfront payment of $ 130.0 million from Sanofi and is eligible to receive further development milestone payments of up to $ 265.0 million upon approval of plozasiran across various indications in mainland China.

The Company is also eligible to receive royalties from Sanofi on net commercial product sales in Greater China under the Sanofi License Agreement.

During the year ended September 30, 2025, the Company recorded $ 130.0 million in revenue.

Visirna identified the licenses as defined in the agreement as the performance obligations under the Asset Purchase Agreement.

The performance obligations for the licenses was satisfied in the fourth quarter of fiscal 2025 upon delivery.

The fixed consideration of $ 130.0 million was allocated to the performance obligations.

The Company will recognize the development milestones as revenue in the periods the underlying milestone events are achieved as achievement of the milestone events are highly susceptible to factors outside of the entity's influence and therefore there is a possibility that the milestone events will not be achieved.

The Company has also applied the sales-based scope exception to the royalty-based payments.

The Sanofi License Agreement may be terminated by either party in the event of a material breach as defined therein.

Unless earlier terminated, the Sanofi License Agreement expires on a product-by-product basis, upon the date of expiration of the relevant royalty term for such product in Greater China.

BALANCE SHEET ACCOUNTS

4 unchanged sentences

Land $ 2,996 $ 2,996

Building 75,988 —

251,317 75,988

Research equipment 62,758 65,353

Manufacturing equipment

Furniture 5,594 5,594

6 unchanged sentences

Depreciation and amortization expense for property, plant and equipment for the years ended September 30, 2025, 2024, and 2023 was $ 22.2 million, $ 16.9 million and $ 10.7 million, respectively.

During the first quarter of fiscal 2024, the Company completed the build out of one of its laboratory and office facilities in Verona, Wisconsin, which resulted in the reclassification of $ 76.0 million from construction in progress to building as of September 30, 2024.

Further, the Company commenced depreciation on the newly completed facility over a 39 -year period.

During the first quarter of fiscal 2025, the Company substantially completed the build out of its manufacturing facility in Verona, Wisconsin, leading to the reclassification of $ 162.7 million from construction in progress to buildings and $ 2.6 million from construction in progress to manufacturing equipment.

The Company subsequently incurred and capitalized $ 10.6 million to buildings and $ 16.0 million to manufacturing equipment during the remainder of fiscal 2025.

Furthermore, the Company began depreciating the newly completed manufacturing facility over a 39 -year period and the manufacturing equipment over 7 - or 10 -year periods.

During the first quarter of fiscal 2024, the Company completed the build out of its laboratory and office facilities in Verona, Wisconsin, which resulted in the reclassification of $ 71.8 million from construction in progress to buildings.

The Company subsequently incurred and capitalized $ 4.2 million from construction in progress to buildings in fiscal 2024.

Accrued Expenses

Accrued expenses consist of the following:

Accrued expenses consisted of the following:

September 30,

(in thousands)

Accrued R&D expenses $ 28,069 $ 12,826

Accrued R&D expenses;

Accrued research and development expenses

$ 30,330 $ 28,069

Accrued research and development expenses;

co-development

31,296 23,351

Accrued capital expenditures 277 4,206

Accrued income taxes (benefits)

Other 7,717 7,391

Total accrued expenses $ 90,419 $ 63,017

As of September 30, 2025, the Company’s accrued research and development expenses was primarily attributable to ongoing clinical trial operations, preclinical animal studies, and associated toxicology assessments.

In addition, accrued research and development expenses;

co-development relates to the co-development and co-commercialization activities under the Takeda License Agreement (see Note 2).

The Company’s investments consisted of the following:

14 unchanged sentences

Total current investments $ 577,465 $ 837 $ ( 26 ) $ 578,276

As of September 30, 2024, the gross unrealized losses were immaterial, and all of the Company’s available-for-sale investments were due within one year or less.

On September 30, 2023, the Company changed the classification of its investment securities from held-to-maturity to available-for-sale.

At the date of the transfer, the carrying value of the Company’s held-to-maturity securities was $ 295.7 million, and net unrealized losses of $ 3.0 million were recognized in accumulated other comprehensive loss.

The following table summarizes the contract maturity of the available-for-sale securities as of:

September 30, 2025 September 30, 2024

(in thousands)

Within one year $ 224,328 $ 578,276

After one to two years 468,490 —

After two to three years — —

Total $ 692,818 $ 578,276

As of September 30, 2025, the gross unrealized losses were immaterial.

The Company has determined that the available-for-sale securities that were in an unrealized loss position did not have any credit loss impairment as of September 30, 2025 and 2024.

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Intangible assets are reviewed annually for impairment and more frequently if potential impairment indicators exist.

No impairment indicators were identified during 2024 and 2023.

No impairment indicators were identified during fiscal 2025 and 2024.

Intangible assets with definite useful lives are amortized on a straight-line basis over their useful lives.

Intangible assets amortization expense in each of 2024, 2023, and 2022 was $ 1.7 million.

Intangible assets amortization expense in each of fiscal 2025, 2024, and 2023 was $ 1.7 million.

None of the intangible assets with definite useful lives are anticipated to have a residual value.

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Common stock (1)

$ 0.001 290,000 135,702 135,702

Preferred stock $ 0.001 5,000 — —

2 unchanged sentences

Preferred stock $ 0.001 5,000 — —

(1) Does not include shares of common stock into which the Avoro Pre-Funded Warrants may be exercised.

As of September 30, 2025 and 2024, respectively, 9,851,400 and 11,492,293 shares of common stock were reserved for issuance upon exercise of options and vesting of restricted stock units granted or available for grant under the Company’s 2013 and 2021 Incentive Plans, as well as for other inducement grants made to new employees under Rule 5635(c)(4) of the Nasdaq Listing Rules.

On January 2, 2024, the Company entered into an underwriting agreement with Jefferies LLC, BofA Securities, Inc., and Cowen and Company, LLC, as representatives of the several underwriters.

The Company issued 15,790,000 shares of common stock at an offering price of $ 28.50 per share.

The aggregate purchase price paid by investors was $ 450.0 million and the Company received net proceeds of $ 429.3 million after deducting advisory fees and offering expenses.

On November 25, 2024, the Company entered into a Securities Purchase Agreement (the “Securities Purchase Agreement”) with an institutional and accredited investor for a private placement of pre-funded warrants to purchase shares of common stock with an exercise price of $ 0.001 per share (“Avoro Pre-Funded Warrants”).

Pursuant to the Securities Purchase Agreement, the Company sold pre-funded warrants to purchase up to 917,441 shares of common stock at a purchase price of $ 27.25 per pre-funded warrant, for an aggregate value of approximately $ 25.0 million.

The outstanding Avoro Pre-Funded Warrants are exercisable at any time and do not have an expiration date.

The Company determined that the Avoro Pre-Funded Warrants are freestanding financial instruments because they (i) are immediately exercisable, (ii) do not embody an obligation for the Company to repurchase its shares, (iii) permit the holders to receive a fixed number of shares of common stock upon exercise, and (iv) are indexed to the Company’s common stock.

As such, the Company evaluated the Avoro Pre-Funded Warrants to determine whether they represent instruments that require liability classification pursuant to the guidance in ASC 480.

However, the Company concluded that the Avoro Pre-Funded Warrants are not a liability within the scope of ASC 480 due to their characteristics.

Further, the Company determined that the Avoro Pre-Funded Warrants do not meet the definition of a derivative under ASC 815 because they do not meet the criteria regarding no or little initial net investment.

Accordingly, the Company assessed the Avoro Pre-Funded Warrants relative to the guidance in ASC 815-40, Contracts in Entity's Own Equity, to determine the appropriate treatment.

The Company concluded that the Avoro Pre-funded Warrants are both indexed to its own stock and meet all other conditions for equity classification.

Accordingly, the Company has classified the Avoro Pre-funded Warrants as permanent equity.

As of September 30, 2025, no shares underlying the Avoro Pre-Funded Warrants had been exercised.

In connection with the Sarepta Collaboration Agreement, on November 25, 2024, the Company entered into the Stock Purchase Agreement with an affiliate of Sarepta for a private placement of shares of common stock of the Company (the “Private Placement”).

Pursuant to the Stock Purchase Agreement, the Company sold 11,926,301 shares of common stock, at a price per share of $ 27.25 , for an aggregate value of approximately $ 325.0 million.

The Private Placement closed on February 7, 2025.

On August 13, 2025, the Company subsequently entered into an agreement with Sarepta to repurchase 2,660,989 common stock of the Company from Sarepta at a price per share of $ 18.79 for an aggregate value of approximately $ 50.0 million and approximately $ 50.0 million in cash to satisfy the milestone payment of $ 100.0 million due from Sarepta.

The shares were recorded as treasury stock at their fair value of $ 53.2 million, resulting in a $ 3.2 million gain on settlement.

As of the end of fiscal 2025, Sarepta no longer holds an equity position in the Company.

On December 2, 2022, the Company entered into an open market sale agreement (the “Open Market Sale Agreement”), pursuant to which the Company may, from time to time, sell up to $ 250,000,000 in shares of the Company’s common stock through Jefferies LLC, acting as the sales agent and/or principal, in an at-the-market offering (“ATM Offering”).

8 unchanged sentences

There were no contingent liabilities recorded as of September 30, 2025 and 2024.

The Company owns land in the Verona Technology Park in Verona, Wisconsin, which has been developed into an approximately 160,000 square foot drug manufacturing facility and an approximately 140,000 square foot laboratory and office facility which will support the Company’s manufacturing process development and analytical activities.

During the first quarter of fiscal 2024, the Company completed the build out of one of its laboratory and office facilities.

As of September 30, 2024, the Company has incurred $ 285.7 million and intends to spend an additional $ 8.0 million to complete the build out of the facilities.

On September 10, 2025, the Company filed a Complaint for Declaratory Judgment in the United States District Court for the District of Delaware against Ionis Pharmaceuticals, Inc.

(“Ionis”) to declare that the United States Patent No.

9,593,333 (“the ’333 patent”) is invalid and not infringed by the Company’s planned commercialization of investigational plozasiran.

On September 11, 2025, Ionis filed a Complaint for Patent Infringement against the Company in the United States District Court for the Central District of California alleging patent infringement of the ’333 patent by the Company’s planned commercialization of investigational plozasiran and seeking damages.

There were no contingent liabilities recorded related to this litigation.

The Company owns land in the Verona Technology Park in Verona, Wisconsin, where it has constructed an approximately 160,000 square foot drug manufacturing facility and an approximately 140,000 square foot laboratory and office facility to support the Company’s manufacturing, process development, and analytical activities.

As of September 30, 2025, the build-out of these facilities was substantially completed, with total costs incurred of $ 296.4 million.

These costs included $ 173.4 million capitalized to building related to the drug manufacturing facility, $ 78.0 million capitalized to building related to the laboratory and office facility, $ 18.6 million capitalized to manufacturing equipment, $ 15.9 million in construction in progress and $ 7.9 million capitalized to research equipment and $ 2.6 million capitalized to furniture.

Pasadena, California :

5 unchanged sentences

The Company leases 144,000 square feet of office and research and development laboratory space located at 10102 Hoyt Park from 11404 & 11408 Sorrento Valley Owner, LLC, which lease expires on April 30, 2038.

Pursuant to the lease, within twelve months of the expiration of the initial 15 -year term, the Company has the option to

extend the lease for up to one additional ten-year term, with certain annual increases in base rent.

Pursuant to the lease, within twelve months of the expiration of the initial 15 -year term, the Company has the option to extend the lease for up to one additional ten-year term, with certain annual increases in base rent.

The Company is not reasonably certain that it will exercise this option to renew and therefore it is not included in right-of-use assets and liabilities as of September 30, 2025.

The lease agreement grants the Company the right to receive an Additional Tenant Improvement Allowance (“ATIA”) funded by the lessor, with a maximum amount of $ 7.2 million, subject to a 7 % interest per annum over the base term.

Further, on September 25, 2023, the Company executed the first amendment to the lease, which grants a second ATIA with a maximum amount of $ 23.6 million, bearing interest at a rate of 9 % per annum over the base term.

The Company received $ 3.1 million and $ 27.7 million in ATIA from the lessor during fiscal years 2024 and 2023, respectively.

The lease agreement, as amended, granted the Company the right to receive an Additional Tenant Improvement Allowance (“ATIA”) funded by the lessor.

The Company received $ 30.8 million in ATIA, including a final payment of $ 3.1 million during the first quarter of fiscal 2024.

As a result, the Company remeasured its lease liability and right-of-use assets to reflect these additional allowances and the related increased lease payments.

The Company has further concluded that these ATIAs have no effects on the classification of the lease.

The Company previously subleased additional research and development space in San Diego, California, which sublease ended during the fiscal year of 2023.

Madison, Wisconsin :

The Company leases 107,000 square feet space located at 502 South Rosa Road for its office and laboratory facilities from University Research Park, Inc., which lease expires on September 30, 2031.

The Company leases 110,956 square feet space, which it increased from 107,000 square feet on June 30, 2025, located at 502 South Rosa Road for its office and laboratory facilities, which lease expires on September 30, 2031.

The lease contains options to renew for two terms of five years .

17 unchanged sentences

(1) Variable lease cost is primarily related to operating expenses associated with the Company’s operating leases.

There was $ 0 , $ 1.4 million and $ 0.3 million short-term lease cost during the years ended September 30, 2024, 2023, and 2022, respectively.

There was $ 0 , $ 0 and $ 1.4 million in short-term lease cost during the years ended September 30, 2025, 2024, and 2023, respectively.

The following table presents maturities of operating lease liabilities on an undiscounted basis as of September 30, 2025:

18 unchanged sentences

The Company has three plans that provide for equity-based compensation.

Under the 2013 Incentive Plan (the “2013 Plan”), 2,899,230 shares of the Company’s common stock are reserved for grants of stock options and restricted stock awards to employees and directors as of September 30, 2024.

Under the 2021 Incentive Plan (the “2021 Plan”), 8,000,000 shares (subject to certain adjustments) of the Company’s common stock are reserved for grants of stock options, stock appreciation rights, restricted and unrestricted stock, performance awards, cash awards and other awards convertible into or otherwise based on shares of the Company’s common stock.

Under the 2013 Incentive Plan (the “2013 Plan”), 2,276,279 awards are granted and outstanding, relating to stock options and restricted stock awards to employees and directors as of September 30, 2025.

Under the 2021 Incentive Plan (the “2021 Plan”), 8,000,000 shares (subject to certain adjustments) of the Company’s common stock are authorized for grants of stock options, stock appreciation rights, restricted and unrestricted stock, performance awards, cash awards and other awards convertible into or otherwise based on shares of the Company’s common stock.

The maximum number of shares authorized under the 2021 Plan will be (i) reduced by any shares subject to awards made under the 2013 Plan after January 1, 2021, and (ii) increased by any shares subject to outstanding awards under the 2013 Plan as of January 1, 2021 that, after January 1, 2021, are canceled, expired, forfeited or otherwise not issued under such awards (other than as a result of being tendered or withheld to pay the exercise price or withholding taxes in connection with any such awards) or settled in cash.

As of September 30, 2024, the total number of shares available for issuance was 4,600,465 shares, which includes 159,678 and 190,627 shares that were forfeited under the 2013 and 2021 Plans, respectively, and 3,749,840 shares have been granted under the 2021 Plan.

Under the Inducement Plan (the “Inducement Plan”), 832,950 shares of the Company’s common stock are authorized for issuance pursuant to grants of stock options, stock appreciation rights, restricted and unrestricted stock, stock units (including restricted stock units), performance awards, cash awards, and other awards convertible into or otherwise based on shares of the Company’s common stock.

As of September 30, 2025, 6,231,559 shares have been granted under the 2021 Plan.

The total number of shares available for issuance was 2,378,770 shares, which includes 170,898 and 439,431 shares that were forfeited under the 2013 and 2021 Plans, respectively.

Under the Company’s Inducement Plan (the “Inducement Plan”), 832,950 shares of the Company’s common stock are authorized for issuance pursuant to grants of stock options, stock appreciation rights, restricted and unrestricted stock, stock units (including restricted stock units), performance awards, cash awards, and other awards convertible into or otherwise based on shares of the Company’s common stock.

Awards under the Inducement Plan may only be granted to new employees of the Company in accordance with the provisions of Rule 5635(c)(4) of the Nasdaq Listing Rules.

As of September 30, 2024, the total number of shares remaining available for issuance was 510,600 shares, and 362,050 shares have been granted under the Inducement Plan.

As of September 30, 2025, 660,020 shares have been granted under the Inducement Plan.

The total number of shares remaining available for issuance was 255,244 shares.

In addition, prior to adoption of the Inducement Plan, the Company previously granted stand-alone inducement awards in the form of stock options and restricted stock units outside of the Company’s equity plans to new employees under Rule 5635(c)(4) of the Nasdaq Listing Rules.

As of September 30, 2025, there were 598,605 and 53,713 shares underlying outstanding stand-alone inducement options and restricted stock units, respectively.

The following table presents a summary of awards outstanding:

The following table presents a summary of awards outstanding attributable to Arrowhead Pharmaceuticals, Inc.:

As of September 30, 2025

4 unchanged sentences

Total 2,276,279 3,782,222 1,158,885 7,217,386

The following table summarizes stock-based compensation expenses included in operating expenses:

The following table summarizes stock-based compensation expenses included in operating expenses attributable to Arrowhead Pharmaceuticals, Inc.:

Year Ended September 30,

17 unchanged sentences

Stock-based compensation expense related to stock options outstanding for the years ended September 30, 2025, 2024, and 2023 was $ 0.1 million, $ 2.8 million and $ 8.4 million, respectively.

As of September 30, 2024, the pre-tax compensation expense for all outstanding unvested stock options in the amount of $ 0.1 million will be recognized in the Company’s results of operations over a weighted average period of 2 months.

As of September 30, 2025, the pre-tax compensation expense for all outstanding unvested stock options is considered nominal.

The fair value of each stock option award is estimated on the date of grant using the Black-Scholes option pricing model.

The Black-Scholes pricing valuation model was developed for use in estimating the fair value of traded options, which do not have vesting restrictions and are fully transferable.

The Black-Scholes option pricing valuation model was developed for use in estimating the fair value of traded options, which do not have vesting restrictions and are fully transferable.

The determination of the fair value of each stock option is affected by the Company’s stock price on the date of grant, as well as assumptions regarding a number of highly complex and subjective variables.

Because the Company’s employee stock options have characteristics significantly different from those of traded options, and because changes in the subjective input assumptions can materially affect the fair value estimate, the existing models do not necessarily provide a reliable single measure of the fair value of its employee stock options.

No options were granted during the years ended September 30, 2025 and 2024.

The following table provides the assumptions used in the calculation of grant-date fair values of these stock options based on the Black-Scholes option pricing model:

Year Ended September 30,

2023 2022 (5)

Expected dividend yield (1)

Risk-free interest rate (2)

N/A 3.69 – 4.57 %

N/A N/A 3.69 % – 4.57 %

Expected volatility (3)

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Visirna ESOP :

On October 1, 2023, Visirna, a subsidiary of the Company, granted 7,500,000 stock options to its employees from the Employee Stock Option Plan (the “Visirna ESOP”), which authorizes 20,000,000 shares for issuance.

As of September 30, 2025, Visirna, a subsidiary of the Company, granted 14,612,000 stock options to its employees from the Employee Stock Option Plan (the “Visirna ESOP”), which authorizes 20,000,000 shares for issuance.

The Visirna ESOP is independently managed by Visirna, including the valuation process.

As of September 30, 2024, stock-based compensation expense related to the Visirna ESOP was $ 6.9 million.

For the years ended September 30, 2025 and 2024, stock-based compensation expense related to the Visirna ESOP was $ 8.5 million and $ 6.9 million, respectively.

Restricted Stock Units

Restricted Stock Units (“RSUs”), including market-based, time-based and performance-based awards, have been granted under the Company’s 2013 and 2021 Plans, the Inducement Plan and as inducements awards granted outside of the Company’s equity-based compensation plans.

At vesting, each outstanding RSU will be exchanged for one share of the Company’s common stock.

At vesting, each outstanding RSU will be exchanged for one share of the

Company’s common stock.

RSU awards generally vest subject to the satisfaction of service requirements or the satisfaction of both service requirements and achievement of certain performance targets.

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At September 30, 2025 and 2024, the Company did not have any financial assets or financial liabilities based on Level 3 measurements.

The following table presents information about the Company’s assets and liabilities measured at fair value on a recurring basis, and indicate the fair value hierarchy of the valuation techniques utilized by the Company:

The following tables present information about the Company’s assets and liabilities measured at fair value on a recurring basis, and indicate the fair value hierarchy of the valuation techniques utilized by the Company:

September 30, 2025

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government and agency securities $ — $ 150,695 $ — $ 150,695

Certificate of deposits — 12,019 — 12,019

Municipal securities — 7,046 — 7,046

Commercial notes — 13,801 — 13,801

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Total available-for-sale securities — 692,818 — 692,818

— 578,276 — 578,276

Cash equivalents

Money market instruments 64,460 — — 64,460

Term deposit — 134,357 — 134,357

Certificate of deposits — 3,001 — 3,001

Corporate debt securities — 16,182 — 16,182

Total cash equivalents 64,460 153,540 — 218,000

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Available-for-sale securities

government bonds $ 31,553 $ — $ — $ 31,553

Municipal securities — 7,093 — 7,093

government and agency securities $ — $ 160,723 $ — $ 160,723

Commercial notes — 179,714 — 179,714

1 unchanged sentence

Total available-for-sale securities — 578,276 — 578,276

31,553 261,182 — 292,735

Cash equivalents

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Total financial assets $ 66,966 $ 578,276 $ — $ 645,242

Income Tax Provision

The components of the loss before income tax expense and noncontrolling interest are as follows:

Income Tax Provision (Benefit)

The components of the income (loss) before income tax expense and noncontrolling interest are as follows:

Year Ended September 30,

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$ 51,533 $ ( 612,460 ) $ ( 202,491 )

The provision for income taxes consisted of the following components:

Income tax provision (benefit) consisted of the following components:

Year Ended September 30,

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Income tax provision $ 21,419 $ ( 2,767 ) $ 2,784

The following table presents a reconciliation of the tax expense based on the statutory rate to the Company’s actual tax expense in the consolidated statements of operations and comprehensive loss.

The following table presents a reconciliation of the tax expense based on the statutory rate to the Company’s actual tax expense in the consolidated statements of operations and comprehensive income (loss).

A notional 21% tax rate was applied as follows:

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Foreign-derived intangible income deduction ( 16.2 ) % — % 1.2 %

Other income 11.1 % — % — %

Stock compensation 10.7 % ( 0.7 ) % ( 1.1 ) %

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Unrealized gains ( 630 ) ( 194 )

Original Issue Discount ( 24,586 ) —

Total gross deferred tax liability $ ( 65,143 ) $ ( 24,141 )

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On the basis of this evaluation at September 30, 2025 and 2024, a valuation allowance of $ 497.5 million and $ 448.9 million, respectively, has been recorded.

As of September 30, 2024, the Company had accumulated federal, state, and foreign net operating loss (“NOL”) carry forwards of $ 223.1 million, $ 693.2 million and $ 38.3 million, respectively.

As of September 30, 2025, the Company had accumulated federal, state, and foreign net operating loss (“NOL”) carryforwards of $ 20.6 million, $ 815.5 million and $ 46.3 million, respectively.

Of the $ 20.6 million in federal NOL carryforwards, $ 20.6 million was generated before January 1, 2018, and is subject to a 20-year carryforward period (“pre-Tax Act losses”), with expiration beginning in 2031.

The remaining $ 199.8 million (“post-Tax Act losses”) can be carried forward indefinitely but is subject to an 80% taxable income limitation.

Of the $ 815.5 million in state NOL carryforwards, $ 2.7 million can be carried forward indefinitely, while the remaining balance begins to expire in 2031.

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Ending balance of unrecognized tax benefits $ 22,152 $ 16,613 $ 14,536

The Company has recorded income tax benefit of $ 3.3 million for the year ended September 30, 2024, and income tax expense of $ 0 and $ 3.5 million for the years ended September 30, 2023 and 2022, respectively, related to uncertain tax positions inclusive of interest and penalties.

The Company has recorded income tax (benefit) expense of $ 0 and $ 3.3 million for the years ended September 30, 2025 and 2024, respectively, related to uncertain tax positions inclusive of interest and penalties.

The Company’s policy is to recognize potential interest and penalties related to unrecognized tax benefits associated with uncertain tax positions, if any, in the income tax provision.

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and various states along with other foreign countries.

Due to the presence of NOL carryforwards, all of the income tax years remain open for examination domestically.

The Company has not been notified that it is under audit by the Internal Revenue Service or foreign taxing authorities;

however, the Company has been notified of an income tax examination by the state of California.

Due to the presence of NOL carryforwards, all of the income tax years remain open for examination.

The Company is currently under audit by the IRS for September 30, 2023.

California income tax examination has been closed.

There are no other audits in any other jurisdictions.

Deferred income taxes have not been provided for undistributed earnings of the Company’s consolidated foreign subsidiaries because of the Company’s intent to reinvest such earnings indefinitely in active foreign operations.

At September 30, 2024, the Company had $ 0 in unremitted earnings that were permanently reinvested related to its consolidated foreign subsidiaries.

The Company analyzes undistributed earnings of each foreign subsidiary and has determined that no withholding taxes are applicable to earnings which are currently available for distribution.

No additional deferred tax liability has been recorded as the parent entity would not be required to include the distribution into income under the current law.

The Tax Cuts and Jobs Act subjects a U.S.

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The Company has elected to account for GILTI in the year the tax is incurred.

On July 4, 2025, the One Big Beautiful Bill Act (“OBBBA”) was enacted in the U.S.

The legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented through 2027.

ASC 740, "Income Taxes", requires the effects of changes in tax rates and laws to be recognized in the period in which the legislation is enacted.

The Company has implemented OBBBA in the fourth quarter of the current year.

EMPLOYEE BENEFIT PLANS

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FINANCING AGREEMENT

On August 7, 2024 (the “Closing Date”), the Company entered into the Financing Agreement with the guarantors party thereto, the lenders party thereto (the “Lenders”), and Sixth Street Lending Partners (“Sixth Street”), as the administrative agent and collateral agent for the Lenders.

The Financing Agreement establishes a senior secured term loan facility of $ 500.0 million (the “Credit Facility”), consisting of $ 400.0 million funded on the Closing Date and an additional $ 100.0 million available at the Company’s option, subject to mutual agreement with Sixth Street, over the seven-year term.

The outstanding principal balance of this Credit Facility, along with the accrued but unpaid interest, is due and payable on August 7, 2031 and bears interest at an annual rate of 15.0 %.

On the Closing Date, the Company received net proceeds of

$ 390.7 million, after issuance costs.

Additional fees related to third parties have been paid or accrued as of September 30, 2024.

On August 7, 2024 (the “Closing Date”), the Company entered into a Financing Agreement with the guarantors party thereto, the lenders party thereto (the “Lenders”), and Sixth Street Lending Partners (“Sixth Street”), as the administrative agent and collateral agent for the Lenders (the “Financing Agreement”).

The loans under the Credit Facility bear interest at an annual rate of 15.0 %, which is paid in kind and added to the outstanding principal balance of the Credit Facility each period.

The outstanding principal balance of this Credit

Facility, including amounts representing accrued but unpaid interest previously paid in kind, is due and payable on August 7, 2031.

The Company is permitted to use the net proceeds for working capital, capital expenditures and general corporate purposes of the Company and its subsidiaries.

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The Company is required to partially repay loans under the Credit Facility with proceeds from certain asset sales, condemnation events and extraordinary receipts, subject, in some cases, to reinvestment rights.

If the Company repays in full the aggregate principal outstanding under the Credit Facility and such payment in full occurs on or prior to August 7, 2028, the Company will be required to make an additional payment to the lenders under the Credit Facility on such date in an amount necessary for the lenders to achieve a multiple of two times on invested capital of the aggregate principal amount funded on the Closing Date.

If such payment in full occurs after August 7, 2028, the Company will be required to make an additional payment to the lenders under the Credit Facility on such date in an amount necessary for the lenders to achieve the greater of the multiple of two times on invested capital of the aggregate principal amount funded on the Closing Date and the present value of all interest payments that would have been payable from such date through the maturity date of the Credit Facility.

All obligations under the Financing Agreement will be secured on a first-priority basis, subject to certain exceptions, by security interests in substantially all assets of the Company and material subsidiaries of the Company, including its intellectual property, and will be guaranteed by material subsidiaries of the Company, including foreign subsidiaries, subject to certain exceptions.

The Financing Agreement contains customary covenants, including, without limitation, a financial covenant to maintain liquidity (cash, cash equivalents and investments) of at least $ 100.0 million if the Company ’ s market capitalization is above $ 1.5 billion, and negative covenants that, subject to certain exceptions, restrict indebtedness, liens, investments (including acquisitions), fundamental changes, asset sales and licensing transactions, dividends, modifications to material agreements, payment of subordinated indebtedness, and other matters customarily restricted in such agreements.

If the Company repays in full the aggregate principal outstanding under the Credit Facility and such payment in full occurs on or prior to August 7, 2028, the Company will be required to make an additional payment to the lenders under the Credit Facility on such date in an amount necessary for the lenders to achieve a two times multiple of invested capital (“MOIC”) of the aggregate principal amount funded on the Closing Date (the “MOIC Payment”).

If such payment in full occurs after August 7, 2028, the Company will be required to make a payment to the lenders under the Credit Facility on such date in an amount necessary for the lenders to achieve the greater of the MOIC Payment and the present value of all interest payments that would have been payable from such date through the maturity date of the Credit Facility discounted at the Treasury Rate (as defined in the Financing Agreement) plus 0.5 %;

provided that such payment amount in this instance will not exceed the amount necessary for the lenders to achieve a 2.5 times MOIC.

On November 26, 2024, the Company entered into an amendment to the Financing Agreement (the "Amendment") to modify, amongst other things, some of the prepayment terms of the loans under the Credit Facility, including, the prepayment terms related to the Sarepta Collaboration Agreement.

The Amendment was effective on February 14, 2025, following the closing of the Sarepta Collaboration Agreement and receipt of the $ 500.0 million upfront payment from Sarepta.

The Amendment added an additional prepayment clause that requires certain contractual prepayments of principle and MOIC payments throughout the life of the loans under the Credit Facility.

Additionally, any prepayment will be split with 50 % of any such prepayment paying down the principle balance of the loans under the Credit Facility and the other 50 % being applied to prepay the MOIC Payment.

In the event the prepayment amounts result in fees being prepaid in excess of the actual amounts required to be paid, the excess fees shall be reallocated and applied to reduce the amount of the principal balance upon repayment in full of the loans under the Credit Facility.

As of September 30, 2025, the Company has paid $ 100.0 million in MOIC payments of which $ 25.3 million is expected to be applied to principal upon repayment in full.

To date, the Company has paid $ 201.6 million of the loans under the Credit Facility during fiscal 2025.

The Amendment was accounted for as a debt modification under ASC 470-50, “Debt—Modification and extinguishments” since the Amendment did not result in substantially different terms.

In connection with the Amendment, the Company did not incur significant third-party fees.

All obligations under the Financing Agreement are secured on a first-priority basis by security interests in substantially all assets of the Company and material subsidiaries of the Company, including its intellectual property, subject to certain exceptions, and is guaranteed by material subsidiaries of the Company, including foreign subsidiaries, subject to certain exceptions.

The Financing Agreement contains customary covenants, including, without limitation, a financial covenant to maintain liquidity (cash, cash equivalents and investments) of at least $ 100.0 million if the Company’s market capitalization is above $ 1.5 billion, and negative covenants that, subject to certain exceptions, restrict indebtedness, liens, investments (including acquisitions), fundamental changes, asset sales and licensing transactions, dividends, modifications to material agreements, payment of subordinated indebtedness, distributions from certain parties, and other matters customarily restricted in such agreements.

The Company is subject to restrictions on sales and licensing transactions with respect to certain core intellectual property, subject to certain exceptions, including certain transactions related to areas outside the United States, United Kingdom, European Union, Japan and China.

The Financing Agreement contains certain embedded features that were identified and evaluated as not material to the consolidated financial statements.

On August 13, 2025, the Company entered into second amendment to the Financing Agreement (the "Second Amendment") that permitted the share repurchase of the Company's common stock from Sarepta and required the Company to pay a nominal administrative fee.

The outstanding balance of the Credit Facility consisted of the following:

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$ 400,000 $ 400,000

Interest on the Initial Term Loan

Unamortized debt discount and issuance costs

Net carrying amount $ 393,183 $ —

Accumulated interest on the Initial Term Loan 66,942 9,000

Accumulated accretion of the MOIC Payment

Unamortized debt issuance costs ( 13,912 ) ( 15,817 )

Current portion of credit facility ( 40,000 ) —

Payments ( 201,625 ) —

Credit facility, net of current portion $ 214,883 $ 393,183

The following table sets forth total interest expense recognized related to the Credit Facility:

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$ 1,906 $ 317 $ —

Accretion of the MOIC Payment 3,478 — —

Contractual interest expense

57,941 9,000 —

Total interest expense

$ 63,325 $ 9,317 $ —

The amounts shown in the table below, related to the Credit Facility, represent the maximum payments the Company is obligated to make to the Lenders during the indicated periods.

A principal repayment of $ 400.0 million is scheduled for the fifth year, in line with the contractual terms of the Credit Facility.

Actual payments may vary and could be lower than the amounts presented in the table.

The amounts shown in the table below, related to the Credit Facility, represent the expected repayments of principle and accrued interest balance as of September 30, 2025 as well as any mandatory prepayments that the Company is obligated to make to the Lenders during the indicated periods.

The principal balance will increase from accrued paid in kind interest and the table does not include MOIC payments beyond those contractually determined.

Actual payments on current principal may vary from the amounts presented in the table.

(in thousands)

2026 $ 40,000

In May 2025, Visirna entered into the Revolving Credit Agreement with Bank of Zhejiang.

The maximum aggregate credit facility is 72.9 million Chinese Yuan ($ 10.3 million) bearing an annual interest rate of 4.1 %.

The term of each loan is twelve months .

The amount outstanding as of September 30, 2025 was 72.9 million Chinese Yuan ($ 10.3 million) on the credit facility which was classified as other current liabilities.

NET LOSS PER SHARE

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Weighted-average basic shares outstanding (1)

133,758 119,784 106,750

Effect of dilutive securities — — —

Weighted-average diluted shares outstanding (1)

133,758 119,784 106,750

Basic net loss per share $ ( 0.01 ) $ ( 5.00 ) $ ( 1.92 )

Diluted net loss per share $ ( 0.01 ) $ ( 5.00 ) $ ( 1.92 )

(1) Includes shares of common stock into which the Avoro Pre-Funded Warrants may be exercised.

The following table sets forth the potentially dilutive securities that have been excluded from the calculation of diluted net loss per share because to include them would be anti-dilutive.

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Total 5,020 4,737 4,053

SEGMENT INFORMATION

We operate in a single segment dedicated to the discovery, development, manufacturing and commercialization of RNAi therapeutics.

The Company's RNAi therapeutics are comprised of siRNAs that function upstream of conventional medicines by potently silencing messenger RNA (“mRNA”) that encode for proteins implicated in the cause or pathway of disease, thus preventing them from being made.

Consistent with our operational structure, our Chief Executive Officer (“CEO”), as the CODM, manages and allocates resources on a consolidated basis at the global corporate level.

Our global research and development and technical operations and quality organizations are responsible for the discovery, development, and supply of products.

Commercial efforts that coordinate the marketing, sales and distribution of these products are organized by geographic region and therapeutic area.

All of these activities are supported by corporate staff functions.

Managing and allocating resources at the corporate level enables our CEO to assess the overall level of resources available and how to best deploy these resources in line with our overarching long-term, corporate-wide strategic goals.

The determination of a single segment is consistent with the consolidated financial information regularly reviewed by the CODM for the purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets.

Consistent with our management reporting, results of our operations are reported on a consolidated basis for purposes of segment reporting.

The CEO evaluates performance and decides how to allocate resources based on consolidated net loss that is reported on the consolidated statements of operations and comprehensive income (loss).

The measure of segment assets is reported on the consolidated balance sheets as total assets.

The CEO uses consolidated net loss to evaluate income generated from the Company’s business activities in deciding how to allocate company resources (such as pursuing clinical development or entering a strategic collaboration), monitoring budget versus actual results, and establishing management’s compensation.

Please refer to the consolidated financial statements for further information related to these measures of segment performance.

In addition, research and development and selling, general and administrative expenses are significant segment expenses regularly provided to the CEO with the following categories:

Research and Development

Year Ended September 30,

2025 2024 2023

(in thousands)

Candidate costs $ 347,571 $ 259,280 $ 162,459

R&D discovery costs 66,788 74,150 55,586

Salaries 109,085 96,418 73,668

Facilities related 29,233 25,782 16,267

Total research and development expense, excluding non-cash expense $ 552,677 $ 455,630 $ 307,980

Stock compensation 32,582 33,586 34,332

Depreciation and amortization 21,900 16,654 10,876

Total research and development expense $ 607,159 $ 505,870 $ 353,188

General & Administrative

Year Ended September 30,

2025 2024 2023

(in thousands)

Salaries $ 31,916 $ 27,589 $ 22,999

Professional, outside services, and other 53,589 24,733 20,720

Facilities related 5,625 4,116 3,415

Total general and administrative expense, excluding non-cash expense $ 91,130 $ 56,438 $ 47,134

Stock compensation 30,785 40,382 43,798

Depreciation/amortization 2,028 1,941 1,617

Total general and administrative expense $ 123,943 $ 98,761 $ 92,549

SUBSEQUENT EVENTS

On November 25, 2024, the Company entered into an Exclusive License and Collaboration Agreement (the “Collaboration Agreement”) with Sarepta Therapeutics, Inc.

(“Sarepta”) for the co-development and commercialization of multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system, and the lungs.

Under the Collaboration Agreement, Sarepta has received an exclusive worldwide license to the Company’s ARO-DUX4, ARO-DM1, ARO-MMP7, and ARO-ATXN2 clinical stage programs.

Sarepta has also received an exclusive sublicensable worldwide license to the Company’s ARO-HTT, ARO-ATXN1, and ARO-ATXN3 preclinical stage programs.

Pursuant to the Collaboration Agreement, Sarepta will be able to select up to six new targets for which the Company will perform discovery, optimization and preclinical development.

Upon completion of the Company’s preclinical

activities, Sarepta will receive an exclusive license to the Company’s product-specific intellectual property rights covering those compounds and be wholly responsible for clinical development and commercialization of each compound.

Closing of the Collaboration Agreement is subject to clearance under the Hart-Scott Rodino Antitrust Improvements Act.

In connection with the Collaboration Agreement, on November 25, 2024, the Company entered into a Stock Purchase Agreement (the “Stock Purchase Agreement”) with an affiliate of Sarepta for a private placement of shares of common stock of the Company (the “Private Placement”).

Pursuant to the Stock Purchase Agreement, the Company sold 11,926,301 shares of common stock, at a price per share of $ 27.25 , for an aggregate value of approximately $ 325.0 million.

The Private Placement is expected to close concurrently with the Collaboration Agreement.

Under the terms of the agreements taken together, the Company expects to receive $ 500.0 million as an upfront payment under the Collaboration Agreement, $ 325.0 million in the form of an equity investment under the Stock Purchase Agreement, and $ 250.0 million to be paid in annual installments of $ 50.0 million over 5 years.

The Company is also eligible to receive $ 300.0 million in near-term payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study, which the Company is on track to achieve.

On August 29, 2025, the Company entered into an Exclusive License and Collaboration Agreement (the “Novartis Collaboration Agreement”) with Novartis Pharma AG (“Novartis”) for the co-development and commercialization of multiple preclinical programs in rare, genetic diseases.

Under the Novartis Collaboration Agreement, Novartis has received an exclusive worldwide license to the Company's ARO-SNCA preclinical stage program.

The Novartis Collaboration Agreement closed on October 17, 2025 subsequent to clearance under the Hart-Scott-Rodino Antitrust Improvement Act.

Under the terms of the Novartis Collaboration Agreement, the Company received $ 200.0 million as an upfront payment.

The Company is also eligible to receive $ 30.0 million associated with certain target nominations.

Further, for each of the 4 programs, the Company is eligible to receive development milestone payments between $ 175.0 million and $ 245.0 million per program and sales milestone payments between $ 285.0 million and $ 370.0 million per program.

The Company is also eligible to receive tiered royalties on net sales of licensed products of up to the low double digits.

On November 26, 2024, the Company also entered into an amendment to the Credit Facility to modify, subject to certain conditions, amongst other things, the requirements to make prepayments of the loans under the Credit Facility with respect to the transactions contemplated by the Collaboration Agreement and the Stock Purchase Agreement.

The transaction is expected to close on or about November 27, 2024.

Sarepta DM1 Milestone

On November 20, 2025, the Company earned a $ 200.0 million milestone payment from Sarepta.

The milestone was earned when Arrowhead achieved the second development milestone event in a Phase 1/2 clinical study of ARO-DM1, also called SRP-1003, an investigational RNAi therapeutic for the treatment of type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.

The second milestone event included the achievement of a patient enrollment target, drug safety committee review and subsequent authorization to dose escalate and proceed, and completion of day 105 study visit by at least one patient in the clinical trial.

REDEMPLO Commercial Launch

The FDA approved the Company's New Drug Application (NDA) for REDEMPLO (plozasiran) injection for Familial Chylomicronemia Syndrome (FCS), on November 18, 2025.

This approval, which was based on the results of the Phase 3 PALISADE clinical trial, was completed within the Prescription Drug User Fee Act (PDUFA) VI timeframe.

This approval is a significant milestone for the Company, and the commercial launch of REDEMPLO is in progress.

We expect to begin generating revenue from sales of REDEMPLO in the upcoming fiscal year.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 9A. Controls and Procedures