What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-08-07 compared with 2025-05-12 · 93 added, 88 removed, 161 unchanged (53% of the section changed)

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• Facioscapulohumeral muscular dystrophy - SRP-1001 (formerly ARO-DUX4, out-licensed to Sarepta);

• Dystrophia myotonica protein kinase (DMPK) - SRP1003 (formerly ARO-DM1 out-licensed to Sarepta;

• Myotonic Dystrophy Type 1 - SRP1003 (formerly ARO-DM1 out-licensed to Sarepta;

• Spinocerebellar ataxia 2 - SRP-1004 (formerly ARO-ATXN2, out-licensed to Sarepta).

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These outside costs, including toxicology/efficacy testing and manufacturing costs, as well as the preparation for and administration of clinical trials, are referred to as “candidate costs.” As clinical candidates progress through clinical development, candidate costs will increase.

The First Half of Fiscal 2025 Business Highlights

Key recent developments through fiscal 2025 included the following:

The First Three Quarters of Fiscal 2025 Business Highlights

Key recent developments through the first three quarters of fiscal 2025 included the following:

• Triggered a $100.0 million milestone payment from Sarepta Therapeutics, Inc., which was triggered on July 27, 2025, when the Company reached the first of two prespecified enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 clinical study of ARO-DM1, an investigational RNAi therapeutic for the treatment of type 1 myotonic dystrophy (DM1);

• Announced the signing of an asset purchase agreement between Sanofi and Visirna Therapeutics, a majority-owned subsidiary of the Company, created to develop and commercialize four of the Company’s investigational cardiometabolic candidates in Greater China.

Under the terms of the agreement, Sanofi will acquire rights to develop and commercialize investigational plozasiran, the Company's first-in-class RNAi therapeutic candidate designed to reduce production of apolipoprotein C-III (APOC3) as a potential treatment for familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG), in Greater China;

• Initiated and dosed the first subject in the YOSEMITE Phase 3 clinical trial of zodasiran, the Company’s investigational RNAi therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease;

• Completed enrollment of SHASTA-3, SHASTA-4, and MUIR-3 Phase 3 clinical trials of plozasiran.

The Company’s global Phase 3 clinical studies are designed to support regulatory submissions for approval of investigational plozasiran in the treatment of severe hypertriglyceridemia.

The Company previously submitted a New Drug Application to the U.S.

Food and Drug Administration (“FDA”) on November 16, 2024 for plozasiran based on positive Phase 3 PALISADE study results in patients with familial chylomicronemia syndrome, which the FDA accepted on January 17, 2025, with a Prescription Drug User Fee Act (PDUFA) action date of November 18, 2025, and indicated it is not currently planning to hold an advisory committee meeting;

• Initiated a Phase 1/2a clinical trial of ARO-ALK7 for the treatment of obesity.

ARO-ALK7 is the first RNAi-based therapy designed to silence adipocyte expression of the ACVR1C gene to reduce the production of Activin receptor-like kinase 7 (ALK7), which acts as a receptor in a pathway that regulates energy homeostasis in adipose tissue;

• Announced Topline results from Part 2 of a Phase 1/2 clinical study of ARO-C3, the Company’s investigational RNAi therapeutic designed to reduce liver production of complement component 3 (C3) as a potential therapy for various complement mediated diseases.

ARO-C3 achieved reductions in alternative pathway complement activity and proteinuria;

• Showcased preclinical data supporting the advancement of two first-in-class clinical stage, RNAi-based investigational therapeutics being developed by the Company for the treatment of obesity and metabolic diseases;

• Announced preclinical results on ARO-ALK7, which is the first RNAi-based therapy designed to silence adipocyte expression of the ACVR1 to reduce the production of Activin receptor-like kinase 7 (ALK7), which acts as a receptor in a pathway that regulates energy homeostasis in adipose tissue.

Arrowhead received regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-ALK7 in New Zealand, which the company anticipates will begin dosing in the second quarter of 2025;

• Entered into a global licensing and collaboration agreement with Sarepta Therapeutics, Inc ( “ Sarepta ” ) on November 25, 2024, which closed on February 7, 2025.

• Entered into a global licensing and collaboration agreement with Sarepta on November 25, 2024, which closed on February 7, 2025.

Closing of the transaction was subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.

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Additionally, the Company is eligible to receive royalties on commercial sales and up to approximately $10.0 billion in future potential milestone payments;

• Submitted a New Drug Application (NDA) to the U.S.

Food and Drug Administration (FDA) on November 16, 2024, which was accepted for filing on January 17, 2025.

The FDA provided a Prescription Drug User Fee Act (PDUFA) action date of November 18, 2025, and indicated it is not currently planning to hold an advisory committee meeting;

• GSK dosed its 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;

• Announced that the Company has dosed the first subjects in a Phase 1/2a clinical trial of ARO-INHBE;

• Announced that the Company dosed the first subjects in a Phase 1/2a clinical trial of ARO-INHBE;

• Presented interim results from a Phase 1/2a clinical study of ARO-CFB at the 8th Complement-Based Drug Development Summit.

The study resulted in multiple promising findings including:

The study resulted in multiple findings including:

(1) ARO-CFB led to dose dependent reductions in circulating CFB protein by up to 90% with greater than 3 months duration, (2) single and multiple doses of ARO-CFB led to near complete inhibition of alternative pathway activity based on Wieslab AP, and (3) single and multiple doses of ARO-CFB led to near complete inhibition of alternative pathway hemolytic activity, measured by AH50.

Net income attributable to Arrowhead Pharmaceuticals, Inc.

was $370.4 million for the three months ended March 31, 2025 and net loss attributable to Arrowhead Pharmaceuticals, Inc.

was $125.3 million for the three months ended March 31, 2024.

Net income attributable to Arrowhead Pharmaceuticals, Inc.

was $197.4 million for the six months ended March 31, 2025 and net loss attributable to Arrowhead Pharmaceuticals, Inc.

was $258.2 million for the six months ended March 31, 2024.

Net income per diluted share was $2.75 for the three months ended March 31, 2025 and net loss per diluted share was $1.02 for the three months ended March 31, 2024.

Net income per diluted share was $1.52 for the six months ended March 31, 2025 and net loss per diluted share was $2.24 for the six months ended March 31, 2024.

The increase in net income for the three and six months ended March 31, 2025 was due to an increase in revenue from the Company's Sarepta Collaboration Agreement partially offset by research and development expenses, which have continued to increase as the Company's pipeline of candidates has expanded and progressed through clinical trial phases.

The Company had $185.7 million of cash, cash equivalents and restricted cash and $911.7 million in available-for-sale securities as of March 31, 2025, as compared to $102.7 million of cash, cash equivalents and restricted cash and $578.3 million in available-for-sale securities as of September 30, 2024.

Based upon the Company’s current cash and investment resources and operating plan, the Company expects to have sufficient liquidity to fund operations for at least the next twelve months from the date of the issuance of these unaudited consolidated financial statements.

Critical Accounting Estimates

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The following data summarizes the Company’s results of operations for the following periods indicated:

Three Months Ended March 31, Six Months Ended March 31,

Three Months Ended June 30, Nine Months Ended June 30,

2025 2024 2025 2024

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Revenue $ 27,767 $ — $ 572,976 $ 3,551

Operating income (loss) $ 381,202 $ (126,191) $ 219,790 $ (262,736)

Net income (loss) attributable to Arrowhead Pharmaceuticals, Inc.

Operating (loss) income $ (165,550) $ (176,141) $ 54,240 $ (438,877)

Net (loss) income attributable to Arrowhead

$ (175,241) $ (170,793) $ 22,119 $ (428,957)

Net income (loss) per diluted share attributable to Arrowhead Pharmaceuticals, Inc.

Net (loss) income per diluted share attributable to Arrowhead

$ (1.26) $ (1.38) $ 0.17 $ (3.63)

Total revenue for the three months ended March 31, 2025 increased by $542.7 million from the same periods of 2024.

Total revenue for the six months ended March 31, 2025 increased by $541.7 million from the same periods of 2024.

Total revenue for the three and nine months ended June 30, 2025 increased by $27.8 million and $569.4 million, respectively, from the same periods of 2024.

The change was primarily driven by the revenue recognition associated with GSK and Sarepta license agreements as discussed below.

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Financial Statements” for more information on revenue recognized under the collaboration and license agreements.

On December 11, 2023, the Company entered into 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.

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

During the six months ended March 31, 2025, the Company recorded $2.5 million revenue.

In October 2020, Takeda and the Company entered into the Takeda License Agreement.

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

During the six months ended March 31, 2024, the Company recorded $0.9 million revenue.

During the nine months ended June 30, 2024, the Company recorded $0.9 million revenue.

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

During the nine months ended June 30, 2025, the Company recorded $2.6 million revenue.

On November 25, 2024, the Company entered into the Sarepta Collaboration Agreement and Stock Purchase 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.

During the six months ended March 31, 2025, the Company recorded $542.7 million in revenue.

As of March 31, 2025, no revenue was recorded for the milestone payments or royalties, as none had been achieved.

During the nine months ended June 30, 2025, the Company recorded $570.3 million in revenue.

As of June 30, 2025, no revenue was recorded for the milestone payments or royalties, as none had been achieved.

Operating Expenses

The analysis below details the operating expenses and discusses the expenditures of the Company within the major expense categories.

For purposes of comparison, the amounts for the three and six months ended March 31, 2025 and 2024 are shown in the tables below.

For purposes of comparison, the amounts for the three and nine months ended June 30, 2025 and 2024 are shown in the tables below.

Research and Development (“R&D”) Expenses

R&D expenses are related to the Company’s research and development discovery efforts and related candidate costs, which are comprised primarily of outsourced costs related to the manufacturing of clinical supplies, toxicity/efficacy studies and clinical trial expenses.

Research and development expenses are related to the Company’s research and development discovery efforts and related candidate costs, which are comprised primarily of outsourced costs related to the manufacturing of clinical supplies, toxicity/efficacy studies and clinical trial expenses.

Internal costs primarily relate to discovery operations at the Company’s research facilities in California and Wisconsin, including facility costs and laboratory-related expenses.

The Company does not separately track R&D expenses by individual research and development projects, or by individual drug candidates.

The Company does not separately track research and development expenses by individual research and development projects, or by individual drug candidates.

The Company operates in a cross-functional manner across projects and does not separately allocate facilities-related costs, candidate costs, discovery costs, compensation expenses, depreciation and amortization expenses, and other expenses related to research and development activities.

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(in thousands) Three Months Ended

March 31, 2025 % of

June 30, 2025 % of

Three Months Ended

March 31, 2024 % of

June 30, 2024 % of

Increase (Decrease)

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Total research and development expense $ 162,368 100 % $ 152,431 100 % $ 9,937 7 %

(in thousands) Six Months Ended

March 31, 2025 % of

Six Months Ended

March 31, 2024 % of

(in thousands) Nine Months Ended

June 30, 2025 % of

Nine Months Ended

June 30, 2024 % of

Increase (Decrease)

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Total research and development expense $ 432,472 100 % $ 370,044 100 % $ 62,428 17 %

Candidate costs increased $30.1 million, or 73%, for the three months ended March 31, 2025 and $54.0 million, or 57%, for the six months ended March 31, 2025 compared to the same periods of 2024.

This increase was primarily due to the additional progression of the Company’s pipeline of candidates into and through clinical trials, which resulted in higher manufacturing, outsourced clinical trial, and toxicity study costs.

R&D discovery costs decreased $2.8 million, or 16%, for the three months ended March 31, 2025 and $11.4 million, or 29%, for the six months ended March 31, 2025 compared to the same periods of 2024.

This decrease was primarily driven by strategic shifts toward clinical development and commercial launch.

Candidate costs increased $3.4 million, or 4%, for the three months ended June 30, 2025 compared to the same period of 2024, and $57.4 million, or 31%, for the nine months ended June 30, 2025 compared to the same period of 2024.

The increase for both periods was primarily due to the additional progression of the Company’s pipeline of candidates into and through clinical trials, which resulted in higher manufacturing, outsourced clinical trial, and toxicity study costs.

R&D discovery costs increased $3.4 million, or 19%, for the three months ended June 30, 2025 compared to the same period of 2024, primarily driven by animal studies and system support costs.

R&D discovery costs decreased $8.0 million, or 14%, for the nine months ended June 30, 2025 compared to the same period of 2024, primarily driven by strategic shifts toward clinical development and commercial launch.

R&D discovery costs are influenced by the Company’s ongoing discovery efforts, continued advancements into novel therapeutic areas and tissue types, and increasing costs related to CNS studies and lab supplies.

Salaries consist of salary, bonuses, payroll taxes, and related benefits for the Company’s R&D personnel.

Salaries expense increased $2.0 million, or 8%, for the three months ended March 31, 2025 and $6.5 million, or 14%, for the six months ended March 31, 2025 compared to the same periods of 2024.

The increase was primarily due to an increase in R&D headcount that has occurred as the Company has expanded its pipeline of candidates, in addition to annual salary increases.

expense increased $2.0 million, or 8%, for the three months ended June 30, 2025 and $8.5 million, or 12%, for the nine months ended June 30, 2025 compared to the same periods of 2024.

The increase for both periods was primarily due to an increase in headcount that has occurred as the Company has expanded its pipeline of candidates, in addition to annual salary increases.

Facilities-related expense includes lease costs for the Company’s research and development facilities in San Diego, California and in Madison and Verona, Wisconsin.

These expenses increased $0.8 million, or 14%, for the three months ended March 31, 2025 and $2.0 million, or 16%, for the six months ended March 31, 2025 compared to the same periods of 2024.

This increase was primarily due to property taxes charged to the laboratory and office facilities in Verona, Wisconsin, which completed their build out during the first quarter of fiscal 2024.

Stock compensation expense, a non-cash expense, is based up the valuation of stock options and restricted stock units granted to employees.

Stock compensation expense increased $0.4 million, or 5%, for the three months ended March 31, 2025, which was primarily due to an increase in R&D headcount.

Stock compensation decreased $1.1 million, or 6%, for the six months ended March 31, 2025 compared to the same periods of 2024.

The decrease was primarily due to the cancellation of awards upon the departure of employees.

These expenses decreased $0.7 million, or 9%, for the three months ended June 30, 2025 compared to the same period of 2024, due to refunds received related to the San Diego and Madison buildings.

These expenses increased $1.3 million, or 7%, for the nine months ended June 30, 2025 compared to the same period of 2024, primarily due to property taxes charged to the laboratory and office facilities in Verona, Wisconsin, which completed their build out during the first quarter of fiscal 2024.

Stock compensation expense, a non-cash expense, is primarily based on the valuation of the restricted stock units granted to employees, which is based on the closing stock price on the grant date.

Stock compensation expense increased $0.4 million, or 5%, for the three months ended June 30, 2025 compared to the same period of 2024, primarily due to an increase in headcount.

Stock compensation decreased $0.7 million, or 3%, for the nine months ended June 30, 2025 compared to the same period of 2024, primarily due to the cancellation of awards upon the departure of employees.

Depreciation and amortization expense, a non-cash expense, relates to depreciation on buildings, lab equipment and leasehold improvements.

These expenses increased $1.5 million, or 36% for the three months ended March 31, 2025 and $2.3 million, or 30%, for the six months ended March 31, 2025 compared to the same periods of 2024.

The increase was primarily attributed to completion of the build out of facilities in Verona, Wisconsin, and the commencement of depreciation.

The Company anticipates these R&D expenses to continue to increase as its pipeline of candidates grows and progresses to later phase clinical trials, in addition to inflationary pressure on goods and services and the labor market.

These expenses increased $1.4 million, or 34% for the three months ended June 30, 2025 and $3.8 million, or 31%, for the nine months ended June 30, 2025 compared to the same periods of 2024.

The increase was primarily attributable to completion of the build out of facilities in Verona, Wisconsin, and the commencement of depreciation.

General & Administrative Expenses

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(in thousands) Three Months Ended

March 31, 2025 % of

June 30, 2025 % of

Three Months Ended

March 31, 2024 % of

June 30, 2024 % of

Increase (Decrease)

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$ 30,949 100 % $ 23,710 100 % $ 7,239 31 %

(in thousands) Six Months Ended

March 31, 2025 % of

Category Six Months Ended

March 31, 2024 % of

(in thousands) Nine Months Ended

June 30, 2025 % of

Category Nine Months Ended

June 30, 2024 % of

Category Increase (Decrease)

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Total general & administrative expense $ 86,264 100 % $ 72,384 100 % $ 13,880 19 %

Salaries expense increased $0.8 million, or 11%, for the three months ended March 31, 2025 and $1.8 million, or 14%, for the six months ended March 31, 2025 compared to the same periods of 2024.

The increase was driven by the combination of annual salary increases and increased headcount required to support the Company’s growth.

Salaries expense increased $1.2 million, or 18%, for the three months ended June 30, 2025 and $3.1 million, or 15%, for the nine months ended June 30, 2025 compared to the same periods of 2024.

The increase was driven by the combination of annual salary increases and an increase in headcount required to support the Company’s growth as the Company prepares for commercialization.

Professional, outside services, and other expenses include costs related to legal, audit, consulting, patent filings, business insurance, other external services, as well as travel, communication, and technology expenses.

This expense increased $4.5 million, or 72%, for the three months ended March 31, 2025 and $9.4 million, or 82%, for the six months ended March 31, 2025 compared to the same periods of 2024.

The increase was mainly due to professional services associated with commercialization and business development efforts.

These expenses

increased $9.5 million, or 175%, for the three months ended June 30, 2025 and $18.9 million, or 112%, for the nine months ended June 30, 2025 compared to the same periods of 2024.

The increase for both periods were mainly due to professional services associated with commercialization and business development efforts as the Company prepares for a product launch, including costs for data analytics, marketing and commercial launch support.

Facilities related expense primarily includes rental costs and other facilities-related costs for the Company’s corporate headquarters in Pasadena, California.

Stock compensation expense, a non-cash expense, is based on the valuation of stock options and restricted stock units granted to employees.

This expense decreased $2.1 million, or 21%, for the three months ended March 31, 2025 and $5.2 million, or 25%, for the six months ended March 31, 2025 compared to the same periods of 2024.

These expenses increased $0.9 million, or 76%, for the three months ended June 30, 2025 and $1.3 million, or 39%, for the nine months ended June 30, 2025 compared to the same periods of 2024.

The increase was primarily driven by higher common area maintenance charges, increased staff amenities expenses, and an increase in headcount.

Stock compensation expense, a non-cash expense, is based on the valuation of the restricted stock units granted to employees, which is based on the closing stock price on the grant date.

These expenses decreased $4.4 million, or 45%, for the three months ended June 30, 2025 and $9.5 million, or 31%, for the nine months ended June 30, 2025 compared to the same periods of 2024.

The decrease was primarily due to lower compensation costs related to performance awards, as the timing of these expenses can vary based on the achievement of related performance targets.

Depreciation and amortization expense, a noncash expense, was primarily related to amortization of leasehold improvements for the Company’s corporate headquarters.

The Company anticipates these general and administrative expenses to continue to increase as its pipeline of candidates grows and progresses to later phase clinical trials including commercialization efforts, in addition to inflationary pressure on goods and services and the labor market.

Other Income (Expense)

Other income (expense) is primarily related to interest income and expense.

Other expense increased $10.8 million and $22.3 million for the three and six months ended March 31, 2025 compared to the same periods of 2024.

The increase was primarily due to non-cash interest expense associated with the liability related to the sale of future royalties and the Credit Facility, partially offset by higher income from increased investment yields due to higher average cash balance.

Other (Expense) Income

Other (expense) income is primarily related to interest income and expense.

Other expense increased $15.7 million and $38.0 million for the three and nine months ended June 30, 2025 compared to the same periods of 2024.

Net loss attributable to Arrowhead Pharmaceuticals, Inc.

was $175.2 million and $170.8 million for the three months ended June 30, 2025 and 2024, respectively.

Net income attributable to Arrowhead Pharmaceuticals, Inc.

was $22.1 million for the nine months ended June 30, 2025 compared to a net loss attributable to Arrowhead Pharmaceuticals, Inc.

of $429.0 million for the same period of 2024.

Net loss per diluted share was $1.26 and $1.38 for the three months ended June 30, 2025 and 2024, respectively.

Net income per diluted share was $0.17 for the nine months ended June 30, 2025 compared to net loss per diluted share of $3.63 for the same period of 2024.

The increase in net loss attributable to Arrowhead Pharmaceuticals, Inc.

for the three months ended June 30, 2025 compared to the same period of 2024 was primarily due to higher research and development expenses as the Company's pipeline of candidates has expanded and progressed through clinical trial phases, as well as higher interest expense related to the Financing Agreement.

The increase in net income for the nine months ended June 30, 2025 compared to the same period of 2024 was primarily due to an increase in revenue from the Sarepta Collaboration Agreement, partially offset by higher research and development expenses, which have continued to increase as the Company's pipeline of candidates has expanded and progressed through clinical trial phases.

LIQUIDITY AND CAPITAL RESOURCES

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Research and development activities have required significant capital investment since the Company’s inception and are expected to continue to require significant cash expenditure as the Company’s pipeline continues to expand and matures into later stage clinical trials, including commercialization efforts.

The Company’s cash, cash equivalents and restricted cash was $185.7 million as of March 31, 2025 compared to $102.7 million as of September 30, 2024.

Cash invested in available-for-sale securities was $911.7 million as of March 31, 2025 compared to $578.3 million as of September 30, 2024.

The Company’s cash, cash equivalents and restricted cash was $129.8 million as of June 30, 2025 compared to $102.7 million as of September 30, 2024.

Cash invested in available-for-sale securities was $770.6 million as of June 30, 2025 compared to $578.3 million as of September 30, 2024.

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 $250.0 million 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.

As of March 31, 2025, no shares have been issued under the Open Market Sale Agreement.

As of June 30, 2025, no shares have been issued under the Open Market Sale Agreement.

In August 2024, the Company entered into the Credit Facility, which provides for a senior secured term loan facility of $500.0 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 of the agreement.

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Upon closing, the Company received $325.0 million for the purchase of 11,926,301 shares of common stock, at a price per share of $27.25, and received $500.0 million as an upfront payment on February 24, 2025.

The Company believes its current financial resources are sufficient to fund its operations through at least the next twelve months from the date of the issuance of these unaudited consolidated financial statements.

The Company is eligible to receive additional milestones of up to $350.0 million over the 12 months from the date of this report.

In the event of Sarepta's termination of the licensing and collaboration agreement for convenience, the Company would remain entitled to receive milestone payments totaling $300.0 million in the aggregate minus any milestone previously paid.

Based upon the Company's current cash and investment resources and operating plan, the Company expects to have sufficient liquidity to fund its operations through at least the next twelve months from the date of the issuance of these unaudited consolidated financial statements.

The following table presents a summary of cash flows:

Six Months Ended March 31,

Nine Months Ended June 30,

(in thousands)

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Financing activities 70,337 481,431

Net increase in cash, cash equivalents and restricted cash $ 83,494 $ 16,729

Net increase (decrease) in cash, cash equivalents and restricted cash $ 27,485 $ (41,353)

Cash, cash equivalents and restricted cash at end of period $ 129,793 $ 69,399

During the six months ended March 31, 2025, cash flow provided by operating activities was $313.8 million, which was primarily due to $500.0 million of cash received as part of the Sarepta agreement, partially offset by ongoing expenses related to the Company's research and development programs and general and administrative expenses.

Cash used in investing activities amounted to $343.3 million, which was primarily attributable to capital expenditures of $12.8 million and investment purchases of $677.9 million, offset by proceeds from maturities of investments of $347.4 million.

During the nine months ended June 30, 2025, cash flow provided by operating activities was $159.1 million, which was primarily due to $500.0 million of cash received as part of the Sarepta agreement, partially offset by ongoing expenses related to the Company's research and development programs and general and administrative expenses.

Cash used in investing activities amounted to $201.9 million, which was primarily attributable to capital expenditures of $15.2 million and investment purchases of $774.6 million, partially offset by proceeds from maturities of investments of $587.9 million.

Cash provided by financing activities of $70.3 million was primarily related to cash received from the issuance of common stock in the Sarepta agreement and pre-funded warrants and stock option exercises (See Note 6 — Stockholders’ Equity of Notes to Consolidated Financial Statements of Part I, “Item 1.

Financial Statements”).

During the six months ended March 31, 2024, cash flow used in operating activities was $210.2 million, which was primarily due to the ongoing expenses related to the Company’s research and development programs and general and administrative expenses.

During the nine months ended June 30, 2024, cash flow used in operating activities was $325.6 million, which was primarily due to the ongoing expenses related to the Company’s research and development programs and general and administrative expenses.

Cash used in investing activities was $197.1 million, which was primarily attributable to capital expenditures of $117.2 million and investment purchases of $428.6 million, partially offset by proceeds from sales and maturities of investments of $348.6 million.

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Contractual Obligations

There has been no material change in the Company’s contractual obligations from that described in Item 7 of its Annual Report on Form 10-K for the year ended September 30, 2024.

The Company entered into an amendment to the Financing Agreement with Sixth Street Lending Partners on November 24, 2024 (see Note 12).

There has been no other material change in the Company’s contractual obligations from that described in Item 7 of its Annual Report on Form 10-K for the fiscal year ended September 30, 2024.

QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

There has been no material change in the Company ’ s exposure to market risk from that described in Item 7A of its Annual Report on Form 10-K for the year ended September 30, 2024.

There has been no material change in the Company ’ s exposure to market risk from that described in Item 7A of its Annual Report on Form 10-K for the fiscal year ended September 30, 2024.

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-Q

Item 2. Management’s Discussion and Analysis