What changed in this filing

/stocks — the workspaceLOADING

Item 2. Management’s Discussion and Analysis

2024-05-09 compared with 2024-02-06 · 113 added, 58 removed, 137 unchanged (56% of the section changed)

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The Company continues to develop other clinical candidates for future clinical trials.

Clinical candidates are tested internally and through GLP toxicology studies at outside laboratories.

Clinical candidates are tested internally and through Good Laboratory Practice (GLP) toxicology studies at outside laboratories.

Drug materials for such studies and clinical trials are either manufactured internally or contracted to third-party manufacturers.

The Company engages third-party contract research organizations (CROs) to manage clinical trials and works cooperatively with such organizations on all aspects of clinical trial management, including plan design, patient recruiting, and follow up.

These outside costs, relating to 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 Quarter of Fiscal 2024 Business Highlights

Key recent developments during the first quarter of fiscal 2024 included the following:

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 2024 Business Highlights

Key recent developments through fiscal 2024 included the following:

• Completed enrollment in Amgen’s Phase 3 OCEAN(a) - outcome trial of olpasiran, triggering a $50.0 million milestone payment to the Company, which was paid in the third quarter of fiscal 2024;

• Presented final data from the double-blind treatment period of the Company’s Phase 2 SHASTA-2 study of investigational plozasiran in patients with severe Hypertriglyceridemic.

Results from the SHASTA-2 study showed dramatic, consistent, and sustained reductions in Apolipoprotein C-III (APOC3) and triglycerides and improvement in multiple atherogenic lipoprotein levels;

• Announced an Expanded Access Program (EAP) to make investigational plozasiran available outside of a clinical trial for qualifying patients with familial chylomicronemia syndrome (FCS);

• Initiated a Phase 1/2a clinical trial of ARO-DM1, being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy;

• Filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-CFB, being developed as a potential treatment for complement mediated renal disease;

• Entered into an underwriting agreement with Jefferies LLC, BofA Securities, Inc., and Cowen and Company, LLC, as representatives of the several underwriters.

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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;

• Filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-CFB, being developed as a potential treatment for complement mediated renal disease;

• Filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy;

• Entered into an Amended and Restated License Agreement with GSK, pursuant to which GSK received a worldwide, exclusive license to develop and commercialize JNJ-3989 (formerly ARO-HBV).

JNJ-3989 had previously been licensed to Janssen Pharmaceuticals, Inc.

Net loss was $132.9 million for the three months ended December 31, 2023 as compared to $41.3 million for the three months ended December 31, 2022.

Net loss per share – diluted was $1.24 for the three months ended December 31, 2023 as compared to $0.39 for the three months ended December 31, 2022.

The changes in net loss for the three months ended December 31, 2023 were due to the combination of a decrease in revenue and an increase in 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 $58.2 million of cash, cash equivalents and restricted cash, $162.1 million in available-for-sale securities, and $626.3 million of total assets as of December 31, 2023, as compared to $110.9 million of cash, cash equivalents and restricted cash, $292.7 million in available-for-sale securities and $765.6 million of total assets as of September 30, 2023.

Based upon the Company’s current cash and investment resources, operating plan, and factoring in the $429.0 million in net proceeds from the January 2024 stock offering, the Company expects to have sufficient liquidity to fund operations for at least the next twelve months.

See Note 2 - Collaboration and License Agreements to Consolidated Financial Statements of Part I, “Item 1.

Financial Statements.”

Net loss attributable to the Company was $125.3 million for the three months ended March 31, 2024 as compared to net income attributable to the Company of $48.7 million for the three months ended March 31, 2023.

Net loss attributable to the Company was $258.2 million for the six months ended March 31, 2024 as compared to net income attributable to the Company of $7.4 million for the six months ended March 31, 2023.

Net loss per share – diluted was $1.02 for the three months ended March 31, 2024 as compared to net income per share – diluted of $0.45 for the three months ended March 31, 2023.

Net loss per share – diluted was $2.24 for the six months ended March 31, 2024 as compared to net income per share – diluted of $0.07 for the six months ended March 31, 2023.

The changes in net loss attributable to the Company for the three and six months ended March 31, 2024 were mainly due to a decrease in revenue from the Company’s license and collaboration agreements, in conjunction with increased

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 $127.7 million of cash, cash equivalents and restricted cash, $395.4 million in available-for-sale securities, and $955.2 million of total assets as of March 31, 2024, as compared to $110.9 million of cash, cash equivalents and restricted cash, $292.7 million in available-for-sale securities and $765.6 million of total assets as of September 30, 2023.

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.

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 December 31,

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

2024 2023 2024 2023

(in thousands, except per share amounts)

Revenue $ — $ 146,267 $ 3,551 $ 208,813

Operating (loss) income

$ (126,191) $ 48,165 $ (262,736) $ 6,031

Operating loss $ (136,545) $ (42,134)

Net loss attributable to Arrowhead Pharmaceuticals, Inc.

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

$ (125,300) $ 48,675 $ (258,164) $ 7,350

Net loss per share-diluted $ (1.24) $ (0.39)

Total revenue for the three months ended December 31, 2023 decreased by $59.0 million, or 94.3% from the same period of 2022.

The revenue for the three months ended December 31, 2023 were mainly driven by the revenue recognition associated with GSK and Takeda, as discussed below.

Net (loss) income per share-diluted

$ (1.02) $ 0.45 $ (2.24) $ 0.07

Total revenue for the three months ended March 31, 2024 decreased by $146.3 million or 100.0% from the same period of 2023.

Total revenue for the six months ended March 31, 2024 decreased by $205.3 million, or 98.3% from the same period of 2023.

The changes were primarily driven by decreased revenue recognition associated with the Company’s license and collaboration agreements during the first half of fiscal 2024.

The revenue for the six months ended March 31, 2024 was mainly driven by the revenue recognition associated with Takeda and GSK, as discussed below.

The Company has evaluated each agreement in accordance with FASB Topic 808– Collaborative Arrangements and Topic 606- Revenue for Contracts from Customers .

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

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

The Company has 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.

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

During the six months ended March 31, 2023, the Company recorded $132.5 million revenue, including $40.0 million milestone payment by dosing the first patient in the Phase 3 REDWOOD clinical study of fazirsiran.

On December 11, 2023, GSK and the Company entered into the GSK HBV Agreement.

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

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

The Company has 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 is 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 Company previously expected these clinical trials to extend to September 2025 in order to demonstrate long term safety and efficacy in the open label extension (OLE) part of the studies;

however, in August 2023, Takeda initiated a Phase 3 OLE study, concluding the Phase 2 study visits for patients in the SEQUOIA and AROAAT2002 studies by December 31, 2023.

Consequently, the Company adjusted its revenue recognition estimates in the fiscal 2023 to align with the revised performance period and the remaining $0.9 million deferred revenue was recognized for the three months ended December 31, 2023.

The Company recognized $16.3 million revenue for the three months ended December 31, 2022.

During the six months ended March 31, 2023, the Company recorded a $30.0 million milestone payment by dosing the first patient in a Phase 2b trial under GSK License Agreement.

Horizon/Amgen :

During the three months ended December 31, 2022, Horizon recorded $6.7 million revenue of the total $40.0 million upfront payment received in July 2021, which was recognized on a straight-line basis over the timeframe for completing the Horizon R&D Services, concluding in the first quarter of 2023.

Horizon also enrolled the first subject in December 2022 in a Phase 1 randomized, placebo-controlled trial to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of HZN-457, triggering a $15.0 million milestone payment to the Company which was paid in the second quarter of fiscal 2023.

During the six months ended March 31, 2023, the Company recorded $6.7 million revenue of the total $40.0 million upfront payment received in July 2021, which was recognized on a straight-line basis over the

timeframe for completing the Horizon R&D Services, concluding in the first quarter of 2023.

Horizon enrolled the first subject in December 2022 in a Phase 1 randomized, placebo-controlled trial to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of HZN-457, triggering a $15.0 million milestone payment to the Company which was paid in the second quarter of fiscal 2023.

Further, Amgen enrolled the first subject in its Phase 3 trial of olpasiran, which triggered a $25.0 million milestone payment to the Company which was paid in the second quarter of fiscal 2023.

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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 months ended December 31, 2023 and 2022 are shown in the tables below.

For purposes of comparison, the amounts for the three and six months ended March 31, 2024 and 2023 are shown in the tables below.

Research and Development (R&D) Expenses

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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 R&D expenses by individual research and development project, or by individual drug candidate.

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

December 31, 2023 % of

March 31, 2024 % of

Three Months Ended

December 31, 2022 % of

March 31, 2023 % of

Increase (Decrease)

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Total research and development expense $ 101,122 100 % $ 74,881 100 % $ 26,241 35 %

Candidate costs increased $3.0 million, or 7%, for the three months ended December 31, 2023 compared to the same period of 2022.

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 increased $16.5 million, or 130%, for the three months ended December 31, 2023 compared to the same period of 2022.

(in thousands) Six Months Ended

March 31, 2024 % of

Six Months Ended

March 31, 2023 % of

Increase (Decrease)

Candidate costs $ 79,179 36 % $ 68,870 43 % $ 10,309 15 %

R&D discovery costs 54,008 25 % 30,124 19 % 23,884 79 %

Salaries 47,516 22 % 31,093 20 % 16,423 53 %

Facilities related 12,465 6 % 6,791 4 % 5,674 84 %

Total research and development expense, excluding non-cash expense $ 193,168 89 % $ 136,878 86 % $ 56,290 41 %

Stock compensation 16,494 7 % 17,147 11 % (653) (4) %

Depreciation/amortization 7,951 4 % 4,551 3 % 3,400 75 %

Total research and development expense $ 217,613 100 % $ 158,576 100 % $ 59,037 37 %

Candidate costs increased $7.3 million, or 28%, for the three months ended March 31, 2024 and $10.3 million, or 15%, for the six months ended March 31, 2024 compared to the same period of 2023.

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 increased $7.4 million, or 42%, for the three months ended March 31, 2024 and $23.9 million, or 79%, for the six months ended March 31, 2024 compared to the same period of 2023.

This increase was primarily driven by the growth of the Company’s discovery efforts and continued advancement into novel therapeutic areas and tissue types, particularly due to an increase in labor along with rising costs associated with CNS studies and lab supplies.

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

The increases in salaries and stock comp expenses for the three months ended December 31, 2023 were 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.

The increase in salaries for the three and six months ended March 31, 2024 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.

Stock compensation expense was based upon the valuation of stock options and restricted stock units granted to employees and directors.

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

Facilities-related costs increased $3.2 million, or 96%, for the three months ended December 31, 2023 compared to the same period of 2022.

This increase was mainly due to the additional lease expense in San Diego, California as the Company expands discovery efforts to identify new drug candidates.

Depreciation and amortization expense, a non-cash expense, increased $1.6 million, or 68% for the three months ended December 31, 2023 compared to the same period of 2022.

The decrease in stock compensation expense for the three and six months ended March 31, 2024 was primarily due to the cancelled awards upon the departure of employees.

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

Facilities-related costs increased $2.5 million, or 72%, for the three months ended March 31, 2024 and $5.7 million, or 84%, for the six months ended March 31, 2024 compared to the same period of 2023.

This increase was mainly due to the ATIAs on the lease in San Diego, California.

See Note 8 — Leases of Notes to Consolidated Financial Statements of Part I, “Item 1.

Financial Statements.”

Depreciation and amortization expense, a non-cash expense, increased $1.8 million, or 81% for the three months ended March 31, 2024 and $3.4 million, or 75%, for the six months ended March 31, 2024, compared to the same period of 2023.

The increase was primarily attributed to higher leasehold improvements, due to completion of the development of the San Diego facility.

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(in thousands) Three Months Ended

December 31, 2023 % of

March 31, 2024 % of

Category Three Months Ended

December 31, 2022 % of

March 31, 2023 % of

Category Increase (Decrease)

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Facilities related 1,015 4 % 1,020 4 % (5) — %

Total general & administrative expense, excluding non-cash expense $ 12,506 53 % $ 9,595 46 % $ 2,911 30 %

Total general & administrative expense, excluding non-cash expenses

$ 14,381 57 % $ 10,948 47 % $ 3,433 31 %

Stock compensation 10,263 41 % 11,868 51 % (1,605) (14) %

Depreciation and amortization 425 2 % 405 2 % 20 5 %

Total general & administrative expense $ 23,605 100 % $ 20,985 100 % $ 2,620 12 %

Salaries expense increased $2.1 million, or 49%, for the three months ended December 31, 2023 compared to the same period of 2022.

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

Total general & administrative expenses

$ 25,069 100 % $ 23,221 100 % $ 1,848 8 %

(in thousands) Six Months Ended

March 31, 2024 % of

Category Six Months Ended

March 31, 2023 % of

Category Increase (Decrease)

Salaries $ 13,347 27 % $ 9,212 21 % $ 4,135 45 %

Professional, outside services, and other 11,500 24 % 9,306 21 % 2,194 24 %

Facilities related 2,040 4 % 2,025 4 % 15 1 %

Total general & administrative expense, excluding non-cash expenses

$ 26,887 55 % $ 20,543 46 % $ 6,344 31 %

Stock compensation 20,950 43 % 22,855 52 % (1,905) (8) %

Depreciation and amortization 837 2 % 808 2 % 29 4 %

Total general & administrative expenses

$ 48,674 100 % $ 44,206 100 % $ 4,468 10 %

Salaries expense increased $2.1 million, or 42%, for the three months ended March 31, 2024 and $4.1 million, or 45%, for the six months ended March 31, 2024 compared to the same period of 2023.

The increase was driven by the

combination of annual salary increases and increased headcount required to support the Company’s growth.

Professional, outside services, and other expense includes legal, consulting, patent expenses, business insurance expenses, other outside services, travel, and communication and technology expenses.

This expense increased $0.8 million, or 19%, for the three months ended December 31, 2023 compared to the same period of 2022.

The increase was mainly due to legal services associated with new patent applications and intellectual property matters.

This expense increased $1.4 million, or 28%, for the three months ended March 31, 2024 and $2.2 million, or 24%, for the six months ended March 31, 2024 compared to the same period of 2023.

The increase was mainly due to legal services associated with new patent applications and intellectual property matters, as well as other professional services.

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, was based upon the valuation of stock options and restricted stock units granted to employees.

This expense decreased $1.6 million, or 14%, for the three months ended March 31, 2024 and $1.9 million, or 8%, for the six months ended March 31, 2024 compared to the same period of 2023.

The decrease was mainly due to the decreased compensation costs related to performance awards.

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

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

Other expense increased $2.5 million for the three months ended December 31, 2023 compared to the same periods of 2022.

The increase was primarily due to the non-cash interest expense on the liability related to the sale of future royalties.

Other expense increased $0.3 million and $2.8 million for the three and six months ended March 31, 2024, respectively, compared to the same periods of 2023.

The increase was primarily due to the non-cash interest expense on the liability related to the sale of future royalties, partially offset by higher yields on investments due to increased interest rates.

LIQUIDITY AND CAPITAL RESOURCES

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Each of these expansions is designed to increase the Company’s internal manufacturing and discovery capabilities and requires significant capital investment.

The Company’s cash, cash equivalents and restricted cash decreased to $58.2 million at December 31, 2023 compared to $110.9 million at September 30, 2023.

Cash invested in available-for-sale debt securities was $162.1 million at December 31, 2023 compared to $292.7 million at September 30, 2023.

The Company’s cash, cash equivalents and restricted cash increased to $127.7 million at March 31, 2024 compared to $110.9 million at September 30, 2023.

Cash invested in available-for-sale securities was $395.4 million at March 31, 2024 compared to $292.7 million at September 30, 2023.

On December 2, 2022, the Company entered into 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 December 31, 2023, no shares have been issued under the Open Market Sale Agreement.

As of March 31, 2024, no shares have been issued under the Open Market Sale Agreement.

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.

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The following table presents a summary of cash flows:

Three Months Ended December 31,

Six Months Ended March 31,

(in thousands)

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Financing activities 431,044 251,096

Net (decrease) increase in cash, cash equivalents and restricted cash $ (52,734) $ 94,366

Net increase in cash, cash equivalents and restricted cash

$ 16,729 $ 27,150

Cash, cash equivalents and restricted cash at end of period $ 127,704 $ 134,959

During the three months ended December 31, 2023, cash flow used by operating activities was $117.8 million, which was primarily due to the ongoing expenses related to the Company’s research and development programs and general and administrative expenses.

Cash provided by investing activities was $64.8 million, which was primarily related to sales and maturities of investments of $133.5 million, offset by capital expenditures, $68.7 million of construction in progress.

Cash provided by financing activities of $0.3 million was primarily related to cash received from stock option exercises.

During the three months ended December 31, 2022, cash flows used by operating activities was $75.5 million, which was primarily due to the ongoing expenses related to the Company’s research and development programs and general and administrative expenses, partially offset by the receipt of $6.0 million from GSK and Horizon.

Cash used in investing activities was $80.7 million, which was primarily related to capital expenditures, primarily construction in progress, of $38.9 million and investments of $111.2 million, partially offset by net sales and maturities of investments of $69.4 million.

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.

Cash used in investing activities amounted to $204.1 million, which was primarily attributable to capital expenditures of $102.7 million and investment purchases of $310.0 million, offset by proceeds from sales and maturities of investments of $208.6 million.

Cash provided by financing activities of $431.0 million was primarily related to cash received from the issuance of common stock as well as 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, 2023, cash flows used by operating activities was $107.2 million, which was primarily due to the ongoing expenses related to the Company’s research and development programs and general and administrative expenses, partially offset by the receipt of $40.0 million from Amgen and Horizon.

Cash used in investing activities was $116.8 million, which was primarily related to capital expenditures, $66.2 million of construction in progress and investment purchases of $192.5 million, offset by proceeds from sales and maturities of investments of $142.0 million.

Cash provided by financing activities of $251.1 million was primarily related to the $250.0 million payment from Royalty Pharma as well as cash received from stock option exercises.

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Financial Statements.”

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 2023.

QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

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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