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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549
_____________________________________
FORM 10-Q
_____________________________________
(Mark One)
xQUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the quarterly period ended June 30, 2023
oTRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from          to          .
Commission file number 001-38042
_____________________________________
ARROWHEAD PHARMACEUTICALS, INC.
(Exact name of registrant as specified in its charter)
_____________________________________
Delaware46-0408024
(State or other jurisdiction of incorporation or organization)(I.R.S. Employer Identification No.)
177 E. Colorado Blvd, Suite 700
Pasadena, California 91105
(626) 304-3400
(Address and telephone number of principal executive offices)
Former name, former address, and former fiscal year, if changed since last report: N/A
_____________________________________
Securities registered pursuant to Section 12(b) of the Exchange Act:
Title of each classTrading Symbol(s)Name of each exchange on which registered
Common Stock, par value $0.001 per share
ARWR
The Nasdaq Global Select Market
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes x No o
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes x No o
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large Accelerated Filer
xAccelerated Filer
o
Non-Accelerated Filer
o
Smaller Reporting Company
o
Emerging Growth Company
o
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes o No x
The number of shares of the registrant’s common stock outstanding as of July 31, 2023 was 107,192,901.



Page(s)
Consolidated Statements of Stockholders’ Equity



PART I. FINANCIAL INFORMATION
ITEM 1.    FINANCIAL STATEMENTS
Arrowhead Pharmaceuticals, Inc.
Consolidated Balance Sheets
(In thousands, except per share amounts)
June 30, 2023
September 30, 2022
(unaudited)
ASSETS
Current assets:
Cash, cash equivalents and restricted cash$105,334 $108,005 
Accounts receivable1,247 1,410 
Short term investments346,369 268,391 
Prepaid expenses10,053 7,289 
Other current assets7,162 20,204 
Total current assets470,165 405,299 
Property and equipment, net231,369 110,297 
Intangible assets, net10,687 11,962 
Long-term investments42,758 105,872 
Right-of-use assets40,667 58,291 
Other assets210 218 
Total Assets$795,856 $691,939 
LIABILITIES, NONCONTROLLING INTEREST AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable$7,874 $2,868 
Accrued expenses38,191 46,856 
Accrued payroll and benefits4,434 12,251 
Lease liabilities2,823 2,776 
Deferred revenue16,905 74,099 
Total current liabilities70,227 138,850 
Long-term liabilities:
Lease liabilities, net of current portion79,911 78,800 
Deferred revenue, net of current portion 55,950 
Liability related to the sale of future royalties263,064  
Other liabilities669  
Total long-term liabilities343,644 134,750 
Commitments and contingencies (Note 7)
Noncontrolling interest and stockholders’ equity:
Common stock, $0.001 par value:
Authorized 290,000 and 145,000 shares; issued and outstanding 107,102 and 105,960 shares
199 198 
Additional paid-in capital1,281,393 1,219,213 
Accumulated other comprehensive loss(411)(136)
Accumulated deficit(916,351)(820,755)
Total Arrowhead Pharmaceuticals, Inc. stockholders’ equity364,830 398,520 
Noncontrolling interest17,155 19,819 
Total noncontrolling interest and stockholders’ equity381,985 418,339 
Total Liabilities, Noncontrolling Interest and Stockholders’ Equity$795,856 $691,939 
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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Arrowhead Pharmaceuticals, Inc.
Consolidated Statements of Operations and Comprehensive Loss
(In thousands, except per share amounts)
(unaudited)
Three Months Ended June 30,Nine Months Ended June 30,
2023202220232022
Revenue$15,825 $32,412 $224,638 $211,656 
Operating expenses:
Research and development94,757 72,180 253,333 213,930 
General and administrative23,771 33,141 67,977 92,403 
Total operating expenses118,528 105,321 321,310 306,333 
Operating loss(102,703)(72,909)(96,672)(94,677)
Other income (expense):
Interest income4,172 1,240 11,414 3,450 
Interest expense(5,158) (13,064) 
Other, net306 (377)821 675 
Total other (loss) income(680)863 (829)4,125 
Loss before income tax expense and noncontrolling interest(103,383)(72,046)(97,501)(90,552)
Income tax expense742  759  
Net loss including noncontrolling interest(104,125)(72,046)(98,260)(90,552)
Net loss attributable to noncontrolling interest, net of tax(1,179) (2,664) 
Net loss attributable to Arrowhead Pharmaceuticals, Inc.$(102,946)$(72,046)$(95,596)$(90,552)
Net loss per share attributable to Arrowhead Pharmaceuticals, Inc.:
Basic$(0.96)$(0.68)$(0.90)$(0.86)
Diluted$(0.96)$(0.68)$(0.90)$(0.86)
Weighted-average shares used in calculating
Basic107,004 105,753 106,597 105,273 
Diluted107,004 105,753 106,597 105,273 
Other comprehensive loss, net of tax:
Foreign currency translation adjustments(79)(33)(275)(71)
Comprehensive loss$(104,204)$(72,079)$(98,535)$(90,623)
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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Arrowhead Pharmaceuticals, Inc.
Consolidated Statements of Stockholders’ Equity
(in thousands)
(unaudited)
Common
Stock
Amount ($)
Additional
Paid-In
Capital
Accumulated
Other
Comprehensive
Loss
Accumulated
Deficit
Non-
controlling Interest
Totals
Balance at September 30, 2022105,960 $198 $1,219,213 $(136)$(820,755)$19,819 $418,339 
Stock-based compensation— — 19,390 — — — 19,390 
Exercise of stock options82 — 576 — — — 576 
Common stock - restricted stock units vesting98 1 (1)— — —  
Foreign currency translation adjustments— — — (122)— — (122)
Interest in joint venture— — — — — (486)(486)
Net loss for the three months ended December 31, 2022
— — — — (41,325)— (41,325)
Balance at December 31, 2022106,140 $199 $1,239,178 $(258)$(862,080)$19,333 $396,372 
Stock-based compensation— — 20,612 — — — 20,612 
Exercise of stock options64 — 520 — — — 520 
Common stock - restricted stock units vesting665 — — — — —  
Foreign currency translation adjustments— — — (74)— — (74)
Interest in joint venture— — — — — (999)(999)
Net income for the three months ended March 31, 2023
— — — — 48,675 — 48,675 
Balance at March 31, 2023106,869 $199 $1,260,310 $(332)$(813,405)$18,334 $465,106 
Stock-based compensation— — 19,947 — — — 19,947 
Exercise of stock options198 — 1,136 — — — 1,136 
Common stock - restricted stock units vesting35 — — — — —  
Foreign currency translation adjustments— — — (79)— — (79)
Interest in joint venture— — — — — (1,179)(1,179)
Net loss for the three months ended June 30, 2023
— — — — (102,946)— (102,946)
Balance at June 30, 2023107,102 $199 $1,281,393 $(411)$(916,351)$17,155 $381,985 
Common
Stock
Amount ($)
Additional
Paid-In
Capital
Accumulated
Other
Comprehensive
Loss
Accumulated
Deficit
Non-
controlling Interest
Totals
Balance at September 30, 2021104,327 $197 $1,053,386 $(69)$(644,692)$ $408,822 
Stock-based compensation— — 24,504 — — — 24,504 
Exercise of stock options208 — 2,145 — — — 2,145 
Common stock - restricted stock units vesting263 — — — — —  
Foreign currency translation adjustments— — — (39)— — (39)
Net loss for the three months ended December 31, 2021
— — — — (62,872)— (62,872)
Balance at December 31, 2021104,798 $197 $1,080,035 $(108)$(707,564)$ $372,560 
Stock-based compensation— — 33,802 — — — 33,802 
Exercise of stock options237 — 1,537 — — — 1,537 
Common stock - restricted stock units vesting667 1 (1)— — —  
Foreign currency translation adjustments— — — 1 — — 1 
Interest in joint venture— — — — — —  
Net income for the three months ended March 31, 2022
— — — — 44,366 — 44,366 
Balance at March 31, 2022105,702 $198 $1,115,373 $(107)$(663,198)$ $452,266 
Stock-based compensation— — 33,391 — — — 33,391 
Exercise of stock options53 — 599 — — — 599 
Common stock - restricted stock units vesting40 — — — — —  
Foreign currency translation adjustments— — — (33)— — (33)
Interest in joint venture— — 39,750 — — 20,250 60,000 
Net loss for the three months ended June 30, 2022
— — — — (72,046)— (72,046)
Balance at June 30, 2022105,795 $198 $1,189,113 $(140)$(735,244)$20,250 $474,177 
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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Arrowhead Pharmaceuticals, Inc.
Consolidated Statements of Cash Flows
(in thousands)
(unaudited)
Nine Months Ended June 30,
20232022
CASH FLOWS FROM OPERATING ACTIVITIES:
Net loss$(98,260)$(90,552)
Adjustments to reconcile net loss to net cash flow from operating activities
Stock-based compensation59,949 91,697 
Depreciation and amortization8,634 7,761 
(Accretion) amortization of note premiums/discounts(1,030)2,013 
Non-cash interest expense on liability related to the sale of future royalties13,064  
Unrealized losses on marketable securities 5,755 
Changes in operating assets and liabilities:
Accounts receivable164 10,016 
Prepaid expenses and other current assets27,913 (8,867)
Accounts payable5,001 (3,563)
Accrued expenses(32,082)1,713 
Deferred revenue(113,144)(87,100)
Operating lease liabilities1,158 3,733 
Net cash used in operating activities(128,633)(67,394)
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property and equipment(112,830)(20,066)
Purchases of investments(233,984)(223,391)
Proceeds from maturities of investments220,150 201,595 
Net cash used in investing activities(126,664)(41,862)
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from the exercises of stock options2,232 4,331 
Proceeds from the sale of future royalties250,000  
Proceeds from investment in joint venture 60,000 
Proceeds from additional tenant improvement allowance669  
Net cash provided by financing activities252,901 64,331 
Net decrease in cash, cash equivalents and restricted cash(2,396)(44,925)
Effect of exchange rate on cash, cash equivalents and restricted cash(275)(70)
CASH, CASH EQUIVALENTS AND RESTRICTED CASH:
BEGINNING OF PERIOD108,005 184,434 
END OF PERIOD$105,334 $139,439 
Supplementary disclosures:
Interest paid$ $ 
Income taxes (paid) refunded$ $ 
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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Arrowhead Pharmaceuticals, Inc.
Notes to Consolidated Financial Statements
(unaudited)
NOTE 1. ORGANIZATION AND SIGNIFICANT ACCOUNTING POLICIES
General and Recent Developments
Arrowhead Pharmaceuticals, Inc. and its subsidiaries (referred to herein collectively as the “Company”) are primarily engaged in developing medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, the Company’s therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference (“RNAi”) is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. The Company’s RNAi-based therapeutics may leverage this natural pathway of gene silencing to target and shut down specific disease-causing genes.
The following table presents the Company’s current pipeline:
Therapeutic AreaNameStageProduct Rights
CardiometabolicARO-APOC3Phase 2b and Phase 3Arrowhead
ARO-ANG3Phase 2bArrowhead
OlpasiranPhase 3Amgen
PulmonaryARO-ENAC2Pre-ClinicalArrowhead
ARO-RAGEPhase 1/2Arrowhead
ARO-MUC5ACPhase 1/2aArrowhead
ARO-MMP7Phase 1/2aArrowhead
Liver
GSK-4532990 (formerly ARO-HSD)
Phase 2GSK
FazirsiranPhase 3Takeda and Arrowhead
JNJ-3989Phase 2Janssen
HZN-457 (formerly ARO-XDH)Phase 1Horizon
ARO-C3Phase 1/2Arrowhead
ARO-PNPLA3 (formerly JNJ-75220795)Phase 1Arrowhead
MuscleARO-DUX4Pre-ClinicalArrowhead
Central Nervous System (CNS)
ARO-SOD1Pre-ClinicalArrowhead
The Company operates lab facilities in San Diego, California and Madison, Wisconsin, where its research and development activities, including the development of RNAi therapeutics, take place. The Company’s principal executive offices are located in Pasadena, California.
During the first three quarters of fiscal 2023, the Company continued to develop and advance its pipeline and partnered candidates. Several key recent developments include:
hosted a Research & Development (R&D) Day on June 1, 2023 to discuss progress of the Company's pipeline of RNAi Therapeutics, at which the following updates were discussed:
ARO-RAGE showed continued dose response with single inhaled dose of 184 mg achieving mean knockdown of 90% and max of 95%;
adipose delivery platform achieved single dose target gene silencing of greater than 90% with six months of duration in non-human primates;
improved hepatic dimer platform achieved equivalent or better knockdown of two target genes with longer duration than monomer mixture in non-human primates;
TRiM™ platform now has potential to address multiple cell types including liver, solid tumors, lung, central nervous system, skeletal muscle, and adipose;
announced progress towards the Company's "20 in 25" goal to grow its pipeline of RNAi therapeutics that leverage the proprietary Targeted RNAi Molecule (TRiM™) platform to a total of
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20 clinical stage or marketed products in the year 2025;
presented updated data from the Phase 2 SEQUOIA study of investigational RNAi therapy Fazirsiran in patients with alpha-1 antitrypsin deficiency liver disease which included:
Fazirsiran reduced serum Z-AAT concentration in a dose-dependent manner;
Fazirsiran significantly reduced liver Z-AAT;
Fazirsiran consistently reduced hepatic globule burden;
Fazirsiran treatment reduced histological signs of hepatic inflammation;
50% of the pooled Fazirsiran treated patients showed at least a one-point improvement in METAVIR liver fibrosis versus 38% in the placebo group;
Fazirsiran has been well tolerated to date;
pulmonary function test results (FEV1 and DLCO) for both Fazirsiran and placebo were stable over time with no apparent dose-dependent effects;
updated Phase 2 clinical data were presented at the European Association for the Study of the Liver (EASL) Congress 2023 in an oral presentation titled, “Fazirsiran reduces liver Z-alpha-1 antitrypsin synthesis, decreases globule burden and improves histological measures of liver disease in adults with alpha-1 antitrypsin deficiency: a randomized placebo-controlled phase 2 study”;
presented interim data from the ongoing Phase 2 GATEWAY clinical study of ARO-ANG3 which included:
mean reduction in LDL-C of 48.1% (200mg) and 44.0% (300mg);
ANPTL3 inhibition with ARO-ANG3 also reduced HDL-C, non-HDL-C, and triglycerides, consistent with published human genetic data;
safety and tolerability;
completed enrollment of the Phase 3 PALISADE clinical trial evaluating ARO-APOC3 for treatment of familial chylomicronemia syndrome;
announced interim results from ARO-RAGE administration in Part 1 of the ongoing Phase 1/2 study in normal healthy volunteers which included:
reductions in soluble RAGE (sRAGE) as measured in serum after two doses on Day 1 and Day 29;
duration of pharmacologic effect persisted for at least 6 weeks after the second administration of the 92 mg does with further follow up ongoing;
reduction in sRAGE as measured in bronchoalveolar lavage fluid (BALF) at Day 31 after a single dose;
reduction in in serum sRAGE was observed after a single dose;
the pooled placebo groups experienced a mean sRAGE increase of 8% in BALF and a mean decrease of 1% serum;
safety and tolerability;
expanded TRiMTM platform to include an optimized intrathecal administration for CNS delivery with distribution throughout the brain and in all relevant brain cell types. The first development candidate to utilize this new delivery platform is ARO-SOD1. In June 2023, the Company filed a clinical trial application (CTA) for approval to initiate a Phase 1 clinical study. In preclinical studies, ARO-SOD1 achieved 95% spinal cord tissue mRNA knockdown after a single intrathecal dose in human SOD1 transgenic rats and maintained greater than 80% spinal cord tissue mRNA knockdown three months after a single intrathecal dose in non-human primates;
dosed the first patient in Takeda’s Phase 3 REDWOOD clinical study of Fazirsiran for the treatment of alpha-1 antitrypsin deficiency associated liver diseases, triggering a $40.0 million milestone payment to the Company which was paid in the third quarter of fiscal 2023;
dosed the first patient in GSK’s Phase 2b trial of GSK4532990, formerly called ARO-HSD, an investigational RNAi therapeutic for the treatment of patients with non-alcoholic steatohepatitis (NASH), triggering a $30.0 million milestone payment to the Company which was paid in the third quarter of fiscal 2023;
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announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ARO-APOC3 for reducing triglycerides in adult patients with familial chylomicronemia syndrome (FCS). ARO-APOC3 was previously granted Orphan Drug designation by the FDA and the European Union;
announced interim results from Part 1 of AROC3-1001, an ongoing Phase 1/2 clinical study of ARO-C3, which included:
a dose-dependent reduction in serum C3, with 88% mean reduction at highest dose tested;
a dose-dependent reduction in AH50, a marker of alternative complement pathway hemolytic activity, with 91% mean reduction at highest dose tested;
duration of pharmacologic effect supportive of quarterly or less frequent subcutaneous dose administration;
safety and tolerability;
received notice from Janssen of its decision to voluntarily terminate the Research Collaboration and Option Agreement (the “Janssen Collaboration Agreement”) between the Company and Janssen. The Company regained full rights to ARO-PNPLA3, formerly called JNJ-75220795, upon termination of the Janssen Collaboration Agreement, which took effect on April 7, 2023. ARO-PNPLA3 is in Phase 1 clinical trials that are now being developed by the Company;
initiated dosing in ARO-MMP7-1001 (NCT05537025), a Phase 1/2a single ascending dose and multiple ascending dose clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-MMP7, an investigational RNAi therapeutic designed to reduce expression of matrix metalloproteinase 7 (MMP7) as a potential treatment for idiopathic pulmonary fibrosis (IPF), in up to 56 healthy volunteers and in up to 21 patients with IPF;
enrolled the first subject in a Phase 1 randomized, placebo-controlled trial to assess the safety tolerability, pharmacokinetics and pharmacodynamics of a development-stage medicine, HZN-457 (previously known as ARO-XDH), which is out-licensed to Horizon, triggering a $15.0 million milestone payment to the Company which was paid in the second quarter of fiscal 2023;
enrolled the first subject in Amgen’s Phase 3 trial of Olpasiran, triggering a $25.0 million milestone payment to the Company which was paid in the second quarter of fiscal 2023;
entered into a Royalty Purchase Agreement (the “Royalty Pharma Agreement”) with Royalty Pharma Investments 2019 ICAV (“Royalty Pharma”) on November 9, 2022, pursuant to which Royalty Pharma paid $250.0 million upfront (See Note 11 — Liability Related to the Sale of Future Royalties of Notes to Consolidated Financial Statements of Part I, “Item 1. Financial Statements.”);
announced top line results from the SEQUOIA Phase 2 Study of Fazirsiran in patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease in which:
fibrosis regression was observed in 50% of patients receiving Fazirsiran;
median reductions of 94% of Z-AAT accumulation in the liver and mean reductions of 68% in histologic globule burden were observed;
treatment emergent adverse events were generally well balanced between Fazirsiran and placebo groups;
results were consistent with AROAAT-2002 open-label study previously published in The New England Journal of Medicine.
Consolidation and Basis of Presentation
The interim Consolidated Financial Statements include the accounts of Arrowhead Pharmaceuticals, Inc. 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. 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.
The interim Consolidated Financial Statements have been prepared in conformity with U.S. generally accepted accounting principles (“GAAP”). The financial data of the Company included herein are unaudited. In the opinion of management, all material adjustments of a normal recurring nature have been made to present fairly the Company’s
7


financial position at June 30, 2023 and the results of operations and cash flows for the periods presented. All intercompany transactions and balances have been eliminated. Certain prior period amounts have been reclassified to conform with the current period presentation.
Certain financial information that is normally included in annual financial statements prepared in accordance with GAAP, but that is not required for interim reporting purposes, has been omitted from the accompanying interim consolidated financial statements and related notes. Readers are urged to review the Company’s Annual Report on Form 10-K for the year ended September 30, 2022 for more complete descriptions and discussions. Operating results and cash flows for the nine months ended June 30, 2023 are not necessarily indicative of the results that may be expected for the fiscal year ending September 30, 2023.
Liquidity
The Company’s primary sources of financing have been through the sale of its securities, revenue from its licensing and collaboration agreements and the sale of certain future royalties. Research and development activities have required significant capital 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 significantly. Additionally, significant capital investment will be required as the Company’s pipeline matures into later stage clinical trials and as the Company plans to increase its internal manufacturing capabilities.
At June 30, 2023, the Company had $105.3 million in cash and cash equivalents (including $7.3 million in restricted cash), $346.4 million in short-term investments and $42.8 million in long-term investments to fund operations. During the nine months ended June 30, 2023, the Company’s cash and cash equivalents and investments balance increased by $12.2 million which was primarily due to the $250.0 million upfront payment received from Royalty Pharma (Note 11) and $110.0 million in milestone payments from the Company’s collaboration and license agreements, partially offset by cash used to fund its operations.
In total, the Company is eligible to receive up to $3.4 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.
Summary of Significant Accounting Policies
There have been no changes to the significant accounting policies disclosed in the Company’s most recent Annual Report on Form 10-K for the fiscal year ended September 30, 2022.
Recent Accounting Pronouncements
There have been no recent accounting pronouncements that have significantly impacted this Quarterly Report on Form 10-Q, beyond those disclosed in the Company’s most recent Annual Report on Form 10-K for the fiscal year ended September 30, 2022.
NOTE 2. COLLABORATION AND LICENSE AGREEMENTS
The following table provides a summary of revenue recognized:
Three Months Ended June 30,Nine Months Ended June 30,
2023202220232022
(in thousands)
GSK$277 $ $29,600 $120,000 
Horizon1,539 6,666 23,206 21,251 
Takeda14,009 25,507 146,477 67,100 
Janssen 239 355 3,305 
Amgen  25,000  
Total$15,825 $32,412 $224,638 $211,656 
The following table summarizes the balance of receivables and contract liabilities related to the Company’s
8


collaboration and license agreements:
June 30, 2023September 30, 2022
(in thousands)
Receivables included in accounts receivable$1,539 $6,174 
Contract liabilities included in deferred revenue$16,905 $130,049 
Glaxosmithkline Intellectual Property (No. 3) Limited (“GSK”)
On November 22, 2021, GSK and the Company entered into an Exclusive License Agreement (the “GSK License Agreement”). Under the GSK License Agreement, GSK has received an exclusive license for GSK-4532990 (formerly ARO-HSD). The exclusive license is worldwide with the exception of greater China. The Company completed its Phase 1/2 study of GSK-4532990, and GSK is wholly responsible for all clinical development and commercialization of GSK-4532990 in its territory. Under the terms of the agreement, the Company has received an upfront payment of $120.0 million and recognized an additional $30.0 million at the start of a Phase 2 trial. The Company is also eligible for an additional payment of $100.0 million upon achieving a successful Phase 2 trial readout and 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. The Company is further eligible to receive tiered royalties on net product sales in a range of mid-teens to twenty percent.
At the inception of the GSK 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 responsibility to complete the Phase 1/2 study (the “GSK R&D Services”). Due to the specialized and unique nature of the GSK R&D Services and their direct relationship with the license, the Company determined that these deliverables represented one distinct bundle and, thus, one performance obligation. Beyond the GSK R&D Services, which are the responsibility of the Company, GSK will be responsible for managing future clinical development and commercialization 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 excluded any future estimated milestones or royalties from this transaction price to date. The Company has allocated the total $120.0 million initial transaction price to its one distinct performance obligation for the GSK-4532990 license and the associated GSK R&D Services. As the Company has completed its performance obligation related to this agreement, the upfront payment of $120.0 million was fully recognized during the six months ended March 31, 2022. Further, GSK dosed the first patient in a Phase 2 trial in March 2023, triggering a $30.0 million milestone payment to the Company which was paid in the third quarter of fiscal 2023. There were no contract assets and liabilities recorded as of June 30, 2023.
Horizon Therapeutics Ireland DAC (“Horizon”)
On June 18, 2021, Horizon and the Company entered into a collaboration and license agreement (the “Horizon License Agreement”). 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. The Company conducted all activities through the preclinical stages of development of, and Horizon is now wholly responsible for clinical development and commercialization of, HZN-457. The Company received $40.0 million as an upfront payment in July 2021 and an additional $15.0 million upon Horizon’s initiation of a Phase 1 clinical trial in January 2023, and is eligible to receive up to $645.0 million in additional potential development, regulatory and sales milestones. The Company is also eligible to receive royalties in the low- to mid-teens range on net product sales.
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”). Due to the specialized and unique nature of these Horizon R&D Services and their direct relationship with the license, the Company determined that these deliverables represented one distinct bundle and, thus, one performance obligation. Beyond the Horizon R&D Services, which are the responsibility of the Company, Horizon is responsible for managing future clinical development and commercialization of HZN-457.
The Company determined the initial transaction price totaled $40.0 million, including the upfront payment. The Company has excluded any future estimated milestones or royalties from this transaction price to date. The Company allocated the total $40.0 million initial transaction price to its one distinct performance obligation for the HZN-457 license and the associated Horizon R&D Services. Revenue was recognized on a straight-line basis over the timeframe for
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completing the Horizon R&D Services. The Company determined that the straight-line basis was appropriate as its efforts were expended evenly over the course of completing its performance obligation. Further, 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. There was $1.5 million in contract assets recorded as accounts receivable and $0 contract liabilities as of June 30, 2023.
Takeda Pharmaceutical Company Limited (“Takeda”)
On October 7, 2020, Takeda and the Company entered into an Exclusive License and Co-Funding Agreement (the “Takeda License Agreement”). Under the Takeda License Agreement, Takeda and the Company will co-develop its Fazirsiran program, the Company’s second-generation subcutaneously administered RNAi therapeutic candidate being developed as a treatment for liver disease associated with alpha-1 antitrypsin deficiency. Within the United States, Fazirsiran, if approved, will be co-commercialized under a 50/50 profit sharing structure. Outside the United States, Takeda will lead the global commercialization strategy and received an exclusive license to commercialize Fazirsiran, while the Company will be eligible to receive tiered royalties of 20% to 25% on net sales. The Company received $300.0 million as an upfront payment in January 2021, recognized an additional $40.0 million upon Takeda’s initiation of a Phase 3 clinical study in March 2023, and is eligible to receive potential development, regulatory and commercial milestones of up to $527.5 million.
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”). 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. 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.
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 a proportional performance 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, Takeda now intends to initiate a new OLE study available to patients participating in these Phase 2 studies that will initiate as early as the fourth quarter of fiscal 2023. Based on this new information, patients enrolled in the SEQUOIA and AROAAT2002 studies are expected to complete their Phase 2 study visits between June 2023 and June 2024, shortening the Company’s performance obligation. As a result, effective the second quarter of fiscal 2023, the Company changed its estimates of the revenue recognition to better reflect this newly estimated performance period. The effect of these changes in estimates resulted in accelerated revenue by $61.4 million, or $0.58 per share (diluted) for each of the three and nine months ended June 30, 2023. There were $16.9 million of contract liabilities recorded as deferred revenue, of which $16.9 million was classified as current as of June 30, 2023.
In March 2023, Takeda dosed the first patient in the Phase 3 REDWOOD clinical study of Fazirsiran, triggering a $40.0 million milestone payment to the Company which was paid in the third quarter of fiscal 2023. The Company also recorded $1.4 million as accrued expenses as of June 30, 2023 that was primarily driven by co-development and co-commercialization activities.
Janssen Pharmaceuticals, Inc. (“Janssen”)
On October 3, 2018, Janssen, part of the Janssen Pharmaceutical Companies of Johnson & Johnson, and the Company entered into a License Agreement (the “Janssen License Agreement”) and the Janssen Collaboration Agreement. The Company also entered into a stock purchase agreement with JJDC, Inc. (“JJDC”), Johnson & Johnson’s venture capital arm (the “JJDC Stock Purchase Agreement”).
Under the Janssen License Agreement, Janssen received a worldwide, exclusive license to the Company’s JNJ-3989 (ARO-HBV) program, the Company’s third-generation subcutaneously administered RNAi therapeutic candidate being
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developed as a potential therapy for patients with chronic hepatitis B virus infection. Beyond the Company’s Phase 1/2 study of JNJ-3989 (ARO-HBV), which the Company was responsible for completing, Janssen is wholly responsible for clinical development and commercialization of JNJ-3989 (ARO-HBV). Under the terms of the Janssen License Agreement, the Company has received $175.0 million as an upfront payment, $75.0 million in the form of an equity investment by JJDC in the Company’s common stock under the JJDC Stock Purchase Agreement, and milestone and option payments totaling $73.0 million, and the Company may receive up to $0.8 billion in development and sales milestone payments for the Janssen License Agreement. The Company is further eligible to receive tiered royalties on product sales up to mid-teens under the Janssen License Agreement.
In May 2021, Janssen exercised its option right for JNJ-75220795 (ARO-JNJ1) which resulted in a $10.0 million milestone payment to the Company. This $10.0 million milestone payment was recognized entirely as of September 30, 2021. The Company conducted its discovery, optimization and preclinical research and development of JNJ-75220795 (ARO-JNJ1), ARO-JNJ2, and ARO-JNJ3 under the Janssen Collaboration Agreement. All costs and labor hours spent by the Company have been entirely funded by Janssen. On April 7, 2023, Janssen voluntarily terminated the Janssen Collaboration Agreement. Upon termination, the Company regained full rights to ARO-PNPLA3, formerly called JNJ-75220795, the only candidate for which Janssen had exercised its option.
At the inception of the Janssen License Agreement, the Company determined that the key deliverables included the license and certain R&D services including the Company’s responsibility to complete the Phase 1/2 study of JNJ-3989 (ARO-HBV) and the Company’s responsibility to ensure certain manufacturing of JNJ-3989 (ARO-HBV) drug product is completed and delivered to Janssen (the “Janssen R&D Services”). Due to the specialized and unique nature of these Janssen 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.
The Company determined the transaction price totaled approximately $252.7 million, which includes the upfront payment, the premium paid by JJDC for its equity investment in the Company, two $25.0 million milestone payments related to JNJ-3989 (ARO-HBV), and estimated payments for reimbursable Janssen R&D Services to be performed. The Company has allocated the total $252.7 million initial transaction price to its one distinct performance obligation for the JNJ-3989 (ARO-HBV) license and the associated Janssen R&D Services. The Company recognized this transaction price in its entirety as of September 30, 2021, as its performance obligations were substantially completed. Future milestones and royalties achieved will be recognized in their entirety when earned. There were no contract assets and liabilities recorded as of June 30, 2023.
Amgen Inc. (“Amgen”)
On September 28, 2016, Amgen and the Company entered into two collaboration and license agreements and a common stock purchase agreement. Under the Second Collaboration and License Agreement (the “Olpasiran Agreement”), Amgen received a worldwide, exclusive license to the Company’s novel RNAi Olpasiran program. These RNAi molecules are designed to reduce elevated lipoprotein(a), which is a genetically validated, independent risk factor for atherosclerotic cardiovascular disease. Under the first collaboration and license agreement (the “First Collaboration and License Agreement” or the “ARO-AMG1 Agreement”), Amgen received an option to a worldwide, exclusive license to ARO-AMG1, an RNAi therapy for an undisclosed genetically validated cardiovascular target. Under both agreements, Amgen is wholly responsible for clinical development and commercialization.
Under the Olpasiran Agreement and the ARO-AMG1 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 additional an $55.0 million in milestone payments; $10.0 million upon Amgen’s initiation of 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 and the ARO-AMG1 Agreement. There were no contract assets and liabilities recorded as of June 30, 2023.
In November 2022, Royalty Pharma and the Company entered into the Royalty Pharma Agreement. 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 $535.0 million in remaining development, regulatory and sales milestone payments payable from Amgen and Royalty Pharma. See Note 11.
Joint Venture and License Agreement with Visirna Therapeutics, Inc. (“Visirna”)
On April 25, 2022, Visirna and the Company entered into a License Agreement (the “Visirna License Agreement”), pursuant to which Visirna received an exclusive license to develop, manufacture and commercialize four of the Company’s
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RNAi-based investigational cardiometabolic medicines in Greater China (including the People’s Republic of China, Hong Kong, Macau and Taiwan). Pursuant to a Share Purchase Agreement (the “Visirna SPA”) entered into simultaneously with the Visirna License Agreement, the Company acquired a majority stake in Visirna as partial consideration for the Visirna License Agreement. Under the Visirna SPA, entities affiliated with Vivo Capital also acquired a minority stake in Visirna in exchange for $60.0 million in upfront capital to support the operations of Visirna. As further consideration under the Visirna License Agreement, the Company is also eligible to receive potential royalties on commercial sales.
During the nine months ended June 30, 2023, the Company performed manufacturing and development work pursuant to a Clinical Supply Agreement between the parties contemplated by the Visirna License Agreement. The Company received $0.9 million as consideration for this manufacturing and development work, and there were no contract assets and liabilities recorded as of June 30, 2023.
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NOTE 3. PROPERTY AND EQUIPMENT
The following table summarizes the Company’s major classes of property and equipment:
June 30, 2023September 30, 2022
(in thousands)
Computers, software, office equipment and furniture$2,198 $2,182 
Land2,996 2,996 
Research equipment50,472 38,283 
Leasehold improvements96,344 42,017 
Construction in progress118,279 56,373 
270,289 141,851 
Less: Accumulated depreciation and amortization(38,920)(31,554)
Property and equipment, net$231,369 $110,297 
Depreciation and amortization expense for property and equipment for the three months ended June 30, 2023 and 2022 was $2.9 million and $2.2 million, respectively. Depreciation and amortization expense for property and equipment for the nine months ended June 30, 2023 and 2022 was $7.4 million and $6.5 million, respectively.
The increase in the construction in progress during the nine months ended June 30, 2023 was mainly due to the continuing developments of manufacturing, laboratory and office facilities in Verona, Wisconsin as well as a new laboratory and office facility in San Diego, California. In May 2023, the Company completed the development of the San Diego facility, which resulted in the reclassification of construction in progress as leasehold improvements as of June 30, 2023. See Note 7.

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NOTE 4. INVESTMENTS
The Company’s investments consisted of the following:
As of June 30, 2023
(in thousands)
Adjusted BasisGross
Unrealized Gains
Gross
Unrealized Losses
Fair Value
Short-term investments (due within one year)
Held to maturity debt securities$346,369 $ $(4,204)$342,165 
Held to maturity certificate of deposit    
Total short-term investments$346,369 $ $(4,204)$342,165 
Long-term investments (due within one through three years)
Held to maturity debt securities$42,758 $ $(294)$42,464 
Total long-term investments$42,758 $ $(294)$42,464 
As of September 30, 2022
(in thousands)
Adjusted BasisGross
Unrealized Gains
Gross
Unrealized Losses
Fair Value
Short-term investments (due within one year)
Held to maturity debt securities$218,391 $ $(3,661)$214,730 
Held to maturity certificate of deposit50,000   50,000 
Total short-term investments$268,391 $ $(3,661)$264,730 
Long-term investments (due within one through three years)
Held to maturity debt securities$105,872 $ $(5,569)$100,303 
Total long-term investments$105,872 $ $(5,569)$100,303 
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NOTE 5. INTANGIBLE ASSETS
Intangible assets subject to amortization include patents and a license agreement capitalized as part of the Novartis RNAi asset acquisition in March 2015. The following table presents the components of intangible assets:
Gross Carrying AmountAccumulated AmortizationImpairmentNet Carrying AmountUseful Lives
(in thousands)(in years)
As of June 30, 2023
Patents$21,728 $12,933 $ $8,795 14
License3,129 1,237  1,892 21
Total intangible assets, net$24,857 $14,170 $ $10,687 
As of September 30, 2022
Patents$21,728 $11,770 $ $9,958 14
License3,129 1,125  2,004 21
Total intangible assets, net$24,857 $12,895 $ $11,962 
Intangible assets are reviewed annually for impairment and more frequently if potential impairment indicators exist. No impairment indicators were identified during the nine months ended June 30, 2023 and 2022.
Intangible assets with definite useful lives are amortized on a straight-line basis over their useful lives. Intangible assets amortization expense was $0.4 million for each of the three months ended June 30, 2023 and 2022, and $1.3 million and for each of the nine months ended June 30, 2023 and 2022. None of the intangible assets with definite useful lives are anticipated to have a residual value.
The following table presents the estimated future amortization expense related to intangible assets as of June 30, 2023:
Amortization Expense
Year Ending September 30, (in thousands)
2023 (remainder)$425 
20241,700 
20251,700 
20261,700 
20271,700 
Thereafter3,462 
Total$10,687 

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NOTE 6. STOCKHOLDERS’ EQUITY
The following table summarizes the Company’s shares of common stock and preferred stock:
Shares
Par ValueAuthorizedIssuedOutstanding
(in thousands)
As of June 30, 2023
Common stock$0.001 290,000 107,102 107,102 
Preferred stock$0.001 5,000   
As of September 30, 2022
Common stock$0.001 145,000 105,960 105,960 
Preferred stock$0.001 5,000   
On March 16, 2023, the Company’s stockholders approved an increase in authorized common shares, par value 0.001 per share, from 145,000,000 to 290,000,000. The amendment to the Amended and Restated Certificate of Incorporation was filed on April 27, 2023.
As of June 30, 2023 and September 30, 2022, respectively, 12,914,571 and 14,000,392 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 2004 Equity Incentive Plan, 2013 Incentive Plan, and 2021 Incentive Plan, as well as for inducement grants made to new employees under Rule 5635(c)(4) of the Nasdaq Listing Rules.
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”). The Company is not required to sell shares under the Open Market Sale Agreement. The Company will pay Jefferies LLC a commission of up to 3.0% of the aggregate gross proceeds received from all sales of the common stock under the Open Market Sale Agreement. Unless otherwise terminated, the ATM Offering shall terminate upon the earlier of (i) the sale of all shares of common stock subject to the Sales Agreement and (ii) the termination of the Sales Agreement as permitted therein. The Company and Jefferies may each terminate the Open Market Sale Agreement at any time upon prior notice. As of June 30, 2023, no shares have been issued under the Open Market Sale Agreement.
NOTE 7. COMMITMENTS AND CONTINGENCIES
Litigation
From time to time, the Company may be subject to various claims and legal proceedings in the ordinary course of business. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount is reasonably estimable, the Company will accrue a liability for the estimated loss. There were no contingent liabilities recorded as of June 30, 2023.
Commitments
On December 20, 2021, the Company completed a purchase of 13 acres of land in the Verona Technology Park in Verona, Wisconsin, which is being 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 process development and analytical activities. As of June 30, 2023, the Company has incurred $102.7 million and intends to spend an additional $160.0 million to $180.0 million to complete the build out of the facilities. As part of this acquisition, the Company entered into a development agreement with the City of Verona to construct certain infrastructure improvements within the tax incremental district and will be reimbursed up to $16.0 million by the City of Verona by future tax increment revenue generated from the developed property. The total amount of funding that the City of Verona will pay under the Tax Incremental Financing program is not guaranteed and will depend on future tax revenues generated from the developed property. The Company will also receive up to $2.5 million of refundable Wisconsin state income tax credits from the Wisconsin Economic Development Corporation (WEDC) as incentives to invest in the local community and create new jobs.
Technology License Commitments
The Company has licensed from third parties the rights to use certain technologies for its research and development activities, as well as in any products it may develop using these licensed technologies. These agreements and other similar
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agreements often require the Company to make milestone and royalty payments. Milestone payments, for example, may be required as the research and development process progresses through various stages of development, such as when clinical candidates enter or progress through clinical trials, upon NDA and/or certain sales level milestones. During the three and nine months ended June 30, 2023 and 2022, the Company did not reach any milestones.
NOTE 8. LEASES
On November 19, 2021, the Company entered into a 15-year lease for approximately 144,000 square feet of office and research and development laboratory space in San Diego, California. This new facility accommodates increased personnel for its expanding pipeline of current and future drug candidates. The lease payments, which began on April 19, 2023, the rent commencement date, will be approximately $119.0 million over the initial 15-year term. The Company also estimates annual operating expenses to be approximately $3.0 million for the first year of the lease, and these payments will continue throughout the initial 15-year term. The Company expects to pay approximately $32.0 million for leasehold improvements, net of tenant improvement allowances. 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.
Further, the lease agreement grants the Company the right to receive an Additional Tenant Improvement Allowance (“ATIA”) funded by the lessor. The maximum amount of ATIA is $7.2 million, and as of June 30, 2023, the Company has received approximately $0.7 million, which has been recorded as other liabilities on its consolidated balance sheets. The Company will repay the ATIA through equal monthly payments, including 7% interest per annum over the base term, starting from the rent commencement date. Interest begins accruing on the date the lessor first disburses the ATIA.
Other Significant Leases
Pasadena, California: The Company leases 49,000 square feet of office space located at 177 Colorado Blvd. for its corporate headquarters from 177 Colorado Owner, LLC, which lease expires on April 30, 2027. The lease contains an option to renew for one term of five years.
San Diego, California: The Company subleased space from Halozyme, Inc. for additional research and development space in San Diego, California. The term of this sublease commenced on April 1, 2020 and ended on January 14, 2023. On December 23, 2022, the Company entered into a new six-month lease agreement with 11404 & 11408 Sorrento Valley Owner (DE) LLC, effective January 15, 2023. The lease ended on July 15, 2023.
Madison, Wisconsin: The Company leases space for office and laboratory facilities, which expires on September 30, 2031. The lease contains options to renew for two terms of five years. After accounting for additional rental square feet added pursuant to amendments to the lease agreement in 2019 and 2020, the Company currently leases a total of 111,000 square feet.
The components of lease assets and liabilities along with their classification on the Company’s consolidated balance sheets were as follows:
Lease Assets and LiabilitiesClassificationJune 30, 2023September 30, 2022
(in thousands)
Operating lease assetsRight-of-use assets$40,667 $58,291 
Current operating lease liabilitiesLease liabilities2,823 2,776 
Non-current operating lease liabilitiesLease liabilities, net of current portion79,911 78,800 
Three Months Ended June 30,Nine Months Ended June 30,
Lease CostClassification2023202220232022
(in thousands)
Operating lease costResearch and development$3,323 $2,974 $7,735 $4,757 
General and administrative expense509 448 1,542 1,288 
Variable lease cost (1)
Research and development257 179 627 519 
General and administrative expense    
Total $4,089 $3,601 $9,904 $6,564 
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(1) Variable lease cost is primarily related to operating expenses associated with the Company’s operating leases.
There was $0.6 million and $0.2 million short-term lease cost during the three months ended June 30, 2023, and 2022, respectively. There was $1.2 million and $0.7 million short-term lease cost during the nine months ended June 30, 2023, and 2022, respectively.
The following table presents payments of operating lease liabilities on an undiscounted basis as of June 30, 2023:
YearAmounts
(in thousands)
2023 (remainder of fiscal year)$1,143 
20248,094 
202511,800 
202612,148 
202711,320 
2028 and thereafter102,812 
Total$147,317 
Less imputed interest$(64,583)
Total operating lease liabilities (includes current portion)$82,734 
Supplemental cash flow and other information related to leases was as follows:
Nine Months Ended June 30,
20232022
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases (in thousands)4,430 3,398 
June 30,
20232022
Weighted-average remaining lease term (in years)13.47.3
Weighted-average discount rate8.0 %8.5 %
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NOTE 9. STOCK-BASED COMPENSATION
The Company has three plans that provide for equity-based compensation. Under the 2004 Equity Incentive Plan (the “2004 Plan”) and 2013 Incentive Plan (the “2013 Plan”), 68,555 and 3,440,076 shares, respectively, of the Company’s common stock are reserved for grants of stock options and restricted stock awards to employees and directors as of June 30, 2023.
On March 18, 2021, the Company’s Board of Directors approved the Arrowhead Pharmaceuticals, Inc. 2021 Incentive Plan (the “2021 Plan”), which authorized 8,000,000 shares (subject to certain adjustments) available 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 June 30, 2023, the total number of shares reserved for issuance was 6,186,644 shares, which included 197,596 shares that were forfeited under the 2013 Plan, and 1,977,114 shares have been granted under the 2021 Plan.
In addition, there were 712,454 shares reserved for options and 746,175 shares reserved for restricted stock units issued as inducement grants to new employees granted outside of the Company’s equity-based compensation plans under Rule 5635(c)(4) of the Nasdaq Listing Rules.
The following table presents a summary of awards outstanding:
As of June 30, 2023
2004 Plan2013 Plan2021 PlanInducement AwardsTotal
Granted and outstanding awards:
Options68,555 1,550,951 33,838 712,454 2,365,798 
Restricted stock units 1,889,125 1,686,766 746,175 4,322,066 
Total68,555 3,440,076 1,720,604 1,458,629 6,687,864 
The following table summarizes stock-based compensation expenses included in operating expenses:
Three Months Ended June 30,Nine Months Ended June 30,
2023202220232022
(in thousands)
Research and development8,982 8,098 26,129 23,958 
General and administrative10,965 25,292 33,820 67,739 
Total$19,947 $33,390 $59,949 $91,697 
Stock Option Awards
The following table presents a summary of the stock option activity for the nine months ended June 30, 2023:
SharesWeighted-
Average
Exercise
Price
Per Share
Weighted-
Average
Remaining
Contractual
Term (Years)
Aggregate
Intrinsic
Value
Outstanding at September 30, 2022
2,721,384$20.73 
Granted32,15133.03 
Cancelled or expired(43,936)62.31 
Exercised(343,801)6.48 
Outstanding at June 30, 2023
2,365,798$22.20 4.4 years$43,813,599 
Exercisable at June 30, 2023
2,188,690$20.28 4.2 years$43,480,339 
The aggregate intrinsic values represents the amount by which the market price of the underlying stock exceeds the exercise price of the option. The total intrinsic value of the options exercised during the three months ended June 30, 2023 and
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2022 was $6.5 million and $1.6 million, respectively. The total intrinsic value of the options exercised during the nine months ended June 30, 2023 and 2022 was $10.1 million and $24.9 million, respectively.
Stock-based compensation expense related to stock options outstanding for the three months ended June 30, 2023 and 2022, was $2.1 million and $2.6 million, respectively. Stock-based compensation expense related to stock options for the nine months ended June 30, 2023 and 2022 was $6.7 million and $8.3 million, respectively.
As of June 30, 2023, the pre-tax compensation expense for all outstanding unvested stock options in the amount of $5.3 million will be recognized in the Company’s results of operations over a weighted average period of 12 months.
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 option 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.
The following table provides the assumptions used in the calculation of grant-date fair values of these stock options based on the Back-Scholes option pricing model:
Nine Months Ended June 30,
2023
2022 (5)
Expected dividend yield(1)
 N/A
Risk-free interest rate(2)
3.69 %N/A
Expected volatility(3)
86.4 %N/A
Expected term (in years)(4)
6.25N/A
Weighted average grant date fair value per share of options granted$24.80 N/A
(1) The dividend yield is zero as the Company currently does not pay a dividend.
(2) The risk-free interest rate is based on that of the U.S. Treasury yields with equivalent terms in effect at the time of the grant.
(3) Volatility is estimated based on volatility average of the Company’s common stock price.
(4) The expected term represents the period of time that stock options granted are expected to be outstanding, by using historical exercise patterns and post-vesting termination behavior.
(5) No options were granted during the nine months ended June 30, 2022.
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 and as inducements grants 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. RSU awards generally vest subject to the satisfaction of service requirements or the satisfaction of both service requirements and achievement of certain performance targets.
The following table summarizes the activity of the Company’s RSUs:
Number of
RSUs
Weighted-
Average
Grant
Date
Fair Value
Per Share
Outstanding at September 30, 2022
4,069,431$62.96 
Granted1,144,59434.27 
Vested(798,271)53.72 
Forfeited(93,688)54.58 
Outstanding at June 30, 2023
4,322,066$57.45 
The fair value of RSUs was determined based on the closing price of the Company’s common stock on the grant date, with consideration given to the probability of achieving service and/or performance conditions for awards.
For the three months ended June 30, 2023 and 2022, the Company recorded $17.8 million and $33.7 million of expense
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related to RSUs, respectively. For the nine months ended June 30, 2023 and 2022, the Company recorded $53.2 million and $83.4 million of expense related to RSUs, respectively. As of June 30, 2023, there was $131.6 million of total unrecognized compensation cost related to RSUs that is expected to be recognized over a weighted-average period of 2.3 years.

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NOTE 10. FAIR VALUE MEASUREMENTS
The Company employs a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The fair value of a financial instrument is the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date using the exit price. Accordingly, when market observable data are not readily available, the Company’s own assumptions are used to reflect those that market participants would be presumed to use in pricing the asset or liability at the measurement date.
Assets and liabilities recorded at fair value on the consolidated balance sheets are categorized based on the level of judgment associated with inputs used to measure their fair values and the level of market price observability, as follows:

Level 1    Unadjusted quoted prices are available in active markets for identical assets or liabilities as of the reporting date.
Level 2    Pricing inputs are other than quoted prices in active markets, which are based on the following:
• Quoted prices for similar assets or liabilities in active markets;
• Quoted prices for identical or similar assets or liabilities in non-active markets; or
• Either directly or indirectly observable inputs as of the reporting date.
Level 3    Pricing inputs are unobservable and significant to the overall fair value measurement, and the determination of fair value requires significant management judgment or estimation.
In certain cases, inputs used to measure fair value may fall into different levels of the fair value hierarchy. In such cases, the level in the fair value hierarchy within which the fair value measurement in its entirety falls has been determined based on the lowest level input that is significant to the fair value measurement in its entirety. Thus, a Level 3 fair value measurement may include inputs that are observable (Level 1 or Level 2) and unobservable (Level 3). The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and consideration of factors specific to the asset or liability.
The Company uses prices and inputs that are current as of the measurement date, including during periods of market disruption. In periods of market disruption, the ability to observe prices and inputs may be reduced for many instruments. This condition could cause an instrument to be reclassified from Level 1 to Level 2, or from Level 2 to Level 3. The Company recognizes transfers between levels at either the actual date of the event or a change in circumstances that caused the transfer. At June 30, 2023 and September 30, 2022, 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 indicates the fair value hierarchy of the valuation techniques utilized by the Company:
June 30, 2023
Level 1Level 2Level 3Total
(in thousands)
Financial assets:
U.S. government bonds$27,092 $ $ $27,092 
Municipal securities 7,033  7,033 
Commercial notes 62,755  62,755 
Corporate debt securities 287,749  287,749 
Certificate of deposits    
Money market instruments49,199   49,199 
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September 30, 2022
Level 1Level 2Level 3Total
(in thousands)
U.S. government bonds$1,973 $ $ $1,973 
Commercial notes 41,727  41,727 
Corporate debt securities 271,333  271,333 
Certificate of deposits50,000   50,000 
Money market instruments39,262   39,262 
NOTE 11. LIABILITY RELATED TO THE SALE OF FUTURE ROYALTIES
On November 9, 2022, the Company and Royalty Pharma entered into the Royalty Pharma Agreement, pursuant to which Royalty Pharma agreed to pay up to $410.0 million in cash to the Company in consideration for the Company’s future royalty interest in Olpasiran, a small interfering RNA (siRNA) originally developed by the Company and licensed to Amgen in 2016 under the Olpasiran Agreement.
Pursuant to the Royalty Pharma Agreement, Royalty Pharma paid $250.0 million upfront and agreed to pay up to an additional $160.0 million in aggregate one-time milestone payments due if and when the following milestone events occur: (i) $50.0 million on completion of enrollment in the OCEAN Phase 3 clinical trial for Olpasiran, (ii) $50.0 million upon receipt of FDA approval of Olpasiran for an approved indication (reduction in the risk of myocardial infarction, urgent coronary revascularization, or coronary heart disease death in adults with established cardiovascular disease and elevated Lp(a)), and (iii) $60.0 million upon Royalty Pharma’s receipt of at least $70.0 million of royalty payments under the Royalty Pharma Agreement in any single calendar year.
In consideration for the payment of the foregoing amounts 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 any milestone payments potentially payable by Amgen under the Olpasiran Agreement.
The Company has evaluated the terms of the Royalty Pharma Agreement and concluded in accordance with the relevant accounting guidance that the Company accounted for the transaction as debt and the funding of $250.0 million from Royalty Pharma was recorded as a liability related to the sale of future royalties on its consolidated balance sheets. The Company is not obligated to repay this upfront funding received under the Royalty Pharma Agreement. This liability is amortized over the expected repayment term using an effective interest rate method. The effective interest rate is calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement. The interest rate may vary during the term of the agreement depending on a number of factors, including the amount and timing of forecasted net revenues which affects the repayment timing and ultimate amount of repayment. The Company will evaluate the effective interest rate periodically based on its current revenue forecasts utilizing the prospective method. For the three and nine months ended June 30, 2023, the Company recognized non-cash interest expense of $5.2 million and $13.1 million, respectively, on the consolidated statements of operations and comprehensive loss.
NOTE 12. EARNINGS PER SHARE
The following table presents the computation of basic and diluted earnings per share for the nine months ended
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June 30, 2023 and 2022.
Three Months Ended June 30,Nine Months Ended June 30,
2023202220232022
(in thousands, except per share amounts)
Numerator:
Net loss$(102,946)$(72,046)$(95,596)$(90,552)
Denominator:
Weighted-average basic shares outstanding107,004 105,753 106,597 105,273 
Effect of dilutive securities    
Weighted-average diluted shares outstanding107,004 105,753 106,597 105,273 
Basic earnings per share$(0.96)$(0.68)$(0.90)$(0.86)
Diluted earnings per share$(0.96)$(0.68)$(0.90)$(0.86)
Potentially dilutive securities representing approximately 3,467,000 and 4,024,000 shares of common stock were excluded from the computation of diluted earnings per share for the three and nine months ended June 30, 2023, respectively, because their effect would have been anti-dilutive.

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ITEM 2.    MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and we intend that such forward-looking statements be subject to the safe harbors created thereby. For this purpose, any statements contained in this Quarterly Report on Form 10-Q except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “intend,” “plan,” “project,” “could,” “estimate,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify forward-looking statements. In addition, any statements that refer to projections of the Company’s future financial performance, trends in its business, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of the Company’s preclinical studies and clinical trials, and its research and development programs; its expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions the Company has entered into or may enter into in the future; its beliefs and expectations regarding the amount and timing of future milestone, royalty or other payments that could be due to or from third parties under existing agreements; and its estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties.
The forward-looking statements included herein are based on current expectations of the Companys management based on available information and involve a number of risks and uncertainties, all of which are difficult or impossible to predict accurately, and many of which are beyond the Company’s control. As such, the Company’s actual results and timing of certain events may differ materially from the results discussed, projected, anticipated or indicated in any forward-looking statements. Forward-looking statements are not guarantees of future performance and the Company’s actual results of operations, financial condition and cash flows may differ materially. Factors that may cause or contribute to such differences include, but are not limited to, those discussed in more detail in “Item 1. Business” and Item 1A. Risk Factors” of Part I and “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations” of Part II of the Company’s most recent Annual Report on Form 10-K. Readers should carefully review these risks, as well as the additional risks described in other documents the Company files from time to time with the Securities and Exchange Commission (the “SEC”). In light of the significant risks and uncertainties inherent in the forward-looking information included herein, the inclusion of such information should not be regarded as a representation by the Company or any other person that such results will be achieved, and readers are cautioned not to place undue reliance on such forward-looking information. Statements made herein are as of the date of the filing of this Quarterly Report on Form 10-Q with the SEC and should not be relied upon as of any subsequent date. Except as may be required by law, the Company disclaims any intent to revise the forward-looking statements contained herein to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.
OVERVIEW
The Company develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, the Company’s therapies trigger the RNAi mechanism to induce rapid, deep and durable knockdown of target genes. RNAi is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. RNAi-based therapeutics may leverage this natural pathway of gene silencing to target and shut down specific disease-causing genes.
The Company has focused its resources on therapeutics that exclusively utilize its high levels of pharmacologic activity in multiple animal models spanning several therapeutic areas. The Company believes that TRiMTM enabled therapeutics offer several potential advantages over prior generation and competing technologies, including: simplified manufacturing and reduced costs; multiple routes of administration including subcutaneous injection and inhaled administration; the ability to target multiple tissue types including liver, lung, muscle, CNS and others; and the potential for improved safety and reduced risk of intracellular buildup, because there are fewer metabolites from smaller, simpler molecules.
The Company’s pipeline includes:
Hypertriglyceridemia - ARO-APOC3
Dyslipidemia - ARO-ANG3
Cardiovascular disease - Olpasiran (formerly AMG 890 or ARO-LPA, out-licensed to Amgen)
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Cystic fibrosis - ARO-ENAC2
Muco-obstructive or inflammatory pulmonary conditions - ARO-MUC5AC and ARO-RAGE
Idiopathic pulmonary fibrosis - ARO-MMP7
Non-alcoholic steatohepatitis (NASH) - GSK-4532990 (formerly ARO-HSD, out-licensed to GSK)
Alpha-1 antitrypsin deficiency (AATD) - Fazirsiran (formerly ARO-AAT, a collaboration with Takeda)
Chronic hepatitis B virus - JNJ-3989 (formerly ARO-HBV, out-licensed to Janssen)
Uncontrolled gout - HZN-457 (formerly ARO-XDH, out-licensed to Horizon)
Complement mediated diseases - ARO-C3
Non-alcoholic steatohepatitis (NASH) - ARO-PNPLA3 (formerly JNJ-75220795 or ARO-JNJ1)
Facioscapulohumeral muscular dystrophy - ARO-DUX4
Amyotrophic lateral sclerosis “ALS” (CNS) - ARO-SOD1
The Company operates lab facilities in San Diego, California and Madison, Wisconsin, where its research and development activities, including the development of RNAi therapeutics, take place. The Company’s principal executive offices are located in Pasadena, California.
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. 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 Three Quarters of Fiscal 2023 Business Highlights
Key recent developments during the first three quarters of fiscal 2023 included the following:
hosted a Research & Development (R&D) Day on June 1, 2023 to discuss progress of the Company's pipeline of RNAi Therapeutics, at which the following updates were discussed:
ARO-RAGE showed continued dose response with single inhaled dose of 184 mg achieving mean knockdown of 90% and max of 95%;
adipose delivery platform achieved single dose target gene silencing of greater than 90% with six months of duration in non-human primates;
improved hepatic dimer platform achieved equivalent or better knockdown of two target genes with longer duration than monomer mixture in non-human primates;
TRiM™ platform now has potential to address multiple cell types including liver, solid tumors, lung, central nervous system, skeletal muscle, and adipose;
announced progress towards the Company's "20 in 25" goal to grow its pipeline of RNAi therapeutics that leverage the proprietary Targeted RNAi Molecule (TRiM™) platform to a total of 20 clinical stage or marketed products in the year 2025;
presented updated data from the Phase 2 SEQUOIA study of investigational RNAi therapy Fazirsiran in patients with alpha-1 antitrypsin deficiency liver disease which included:
Fazirsiran reduced serum Z-AAT concentration in a dose-dependent manner;
Fazirsiran significantly reduced liver Z-AAT;
Fazirsiran consistently reduced hepatic globule burden;
Fazirsiran treatment reduced histological signs of hepatic inflammation;
50% of the pooled Fazirsiran treated patients showed at least a one-point improvement in METAVIR liver fibrosis versus 38% in the placebo group;
Fazirsiran has been well tolerated to date;
pulmonary function test results (FEV1 and DLCO) for both Fazirsiran and placebo were stable over time with no apparent dose-dependent effects;
updated Phase 2 clinical data were presented at the European Association for the Study of the Liver
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(EASL) Congress 2023 in an oral presentation titled, “Fazirsiran reduces liver Z-alpha-1 antitrypsin synthesis, decreases globule burden and improves histological measures of liver disease in adults with alpha-1 antitrypsin deficiency: a randomized placebo-controlled phase 2 study”;
presented interim data from the ongoing Phase 2 GATEWAY clinical study of ARO-ANG3 which included:
mean reduction in LDL-C of 48.1% (200mg) and 44.0% (300mg);
ANPTL3 inhibition with ARO-ANG3 also reduced HDL-C, non-HDL-C, and triglycerides, consistent with published human genetic data;
safety and tolerability;
completed enrollment of the Phase 3 PALISADE clinical trial evaluating ARO-APOC3 for treatment of familial chylomicronemia syndrome;
secured stockholder approval to increase authorized common shares to 290,000,000 from 145,000,000 to provide the Company with additional flexibility to issue common stock for a variety of general corporate purposes;
announced interim results from ARO-RAGE administration in Part 1 of the ongoing Phase 1/2 study in normal healthy volunteers which included:
reductions in soluble RAGE (sRAGE) as measured in serum after two doses on Day 1 and Day 29;
duration of pharmacologic effect persisted for at least 6 weeks after the second administration of the 92 mg does with further follow up ongoing;
reduction in sRAGE as measured in bronchoalveolar lavage fluid (BALF) at Day 31 after a single dose;
reduction in in serum sRAGE was observed after a single dose;
the pooled placebo groups experienced a mean sRAGE increase of 8% in BALF and a mean decrease of 1% serum;
safety and tolerability;