RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Breakthrough Therapy (China), Orphan Drug (Japan), Orphan Drug (Australia), Priority Review (Australia)

Structure/Sequence

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Sequence Code 319466156

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Sequence Code 1190395904

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Clinical Trials associated with Plozasiran

NCT06880770

/ Not yet recruitingPhase 3

Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults With Severe Hypertriglyceridemia at High Risk of Acute Pancreatitis (SHASTA-5 Study)

This study will evaluate the efficacy and safety of plozasiran in approximately 140 adult participants with severe hypertriglyceridemia (SHTG) and history of at least two prior acute pancreatitis (AP) events not attributed to other etiologies, with at least one occurring within the last 12 months prior to screening. Eligible participants will be randomly assigned in a double-blind manner to either receive plozasiran 25 mg by subcutaneous (SC) injection every three months (Q3M) or matching placebo. Enrolled participants will be counseled to remain on the specified low-fat diet and background medications throughout the study. Following completion of the double-blind treatment period, or if the participant has a positively adjudicated AP event (whichever occurs first), participants will transition to the 12-month Open-Label Extension (OLE) treatment period receiving plozasiran 25 mg by SC injection Q3M.

Start Date01 Apr 2025

Sponsor / Collaborator

Arrowhead Pharmaceuticals, Inc.

NCT06822790

/ RecruitingPhase 3

A Phase 3 Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Plozasiran in Adults With Hypertriglyceridemia (SHASTA-10 Study)

This is an open-label study to be conducted in adults with hypertriglyceridemia (HTG) and severe hypertriglyceridemia (SHTG). Each participant must have completed all required visits per protocol in the parent study AROAPOC3-3001 (NCT05089084), AROAPOC3-3003 (NCT06347003), AROAPOC3-3004 (NCT06347016) or AROAPOC3-3009 (NCT06347133). All eligible participants will receive plozasiran administered subcutaneously (SC) approximately every 3 months for 24 months. Participants will be counseled to remain on the specified low-fat diet throughout the study in accordance with local standard of care.

Start Date09 Apr 2025

Sponsor / Collaborator

Arrowhead Pharmaceuticals, Inc.

NCT06347016

/ Active, not recruitingPhase 3

Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozsiran in Adults With Severe Hypertriglyceridemia

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After Month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

Start Date23 Jul 2024

Sponsor / Collaborator

Arrowhead Pharmaceuticals, Inc.

100 Clinical Results associated with Plozasiran

100 Translational Medicine associated with Plozasiran

100 Patents (Medical) associated with Plozasiran

Literatures (Medical) associated with Plozasiran

01 Dec 2025·Current Atherosclerosis Reports

Novel Therapeutics for Familial Chylomicronemia Syndrome

Review

Author: Izar, Maria Cristina ; Fonseca, Francisco Antonio Helfenstein

PURPOSE OF REVIEW:

This review discusses new treatment approaches for familial chylomicronemia syndrome (FCS), a rare disorder affecting triglyceride metabolism. The focus is on antisense oligonucleotides (ASO) and small-interfering RNA (siRNA) therapies targeting APOC3 and angiopoietin-like protein 3 (ANGPTL3).

RECENT FINDINGS:

Volanesorsen, an ASO targeting APOC3, has shown effectiveness in managing FCS, multifactorial chylomicronemia, and familial partial lipodystrophy, but its use is limited by thrombocytopenia. Emerging therapies, Olezarsen (ASO anti-APOC3) and Plozasiran (siRNA anti-APOC3), both conjugated with GalNAc, show promise in reducing acute pancreatitis risk without platelet concerns. ANGPTL3 inhibition requires residual lipoprotein lipase (LPL) activity, with only siRNA-based therapies-zodasiran and solbinsiran-under investigation. Suppressing APOC3 expression and targeting ANGPTL3 via siRNA offer significant potential, but long-term studies are needed to confirm their efficacy and safety. Future research may explore gene-editing strategies using lipid nanoparticle-based CRISPR-Cas9 delivery for more durable treatment outcomes.

01 Dec 2025·Current Atherosclerosis Reports

Highlights of Cardiovascular Disease Prevention Studies Presented at the 2024 American Heart Association Scientific Sessions

Review

Author: Khoja, Adeel ; Inam, Maha ; Murthy, Nikitha ; Epstein, Elizabeth ; Virani, Salim S ; Vaughan, Elizabeth ; Sheikh, Sana ; Mehta, Sandeep ; Minhas, Abdul Mannan Khan ; Slipczuk, Leandro ; Junaid, Vashma ; Chai, Zohar ; Hermel, Melody ; Hinkamp, Colin

PURPOSE OF REVIEW:

Focused review highlighting ten select studies presented at the 2024 American Heart Association (AHA) Scientific Sessions.

RECENT FINDING:

Included studies assessed effects of intensive blood pressure control in patients with type 2 diabetes (BPROAD); decision support system for physicians to optimize early lipid lowering therapies after acute coronary syndrome (ZODIAC); efficacy and safety of zerlasiran, a short interfering RNA targeting lipoprotein(a) (ALPACAR); efficacy and safety of muvalaplin an oral disrupter of the assembly of lipoprotein(a) particles (KRAKEN); safety and efficacy of obicetrapib in patients with heterozygous familial hypercholesterolemia (BROOKLYN); efficacy and safety of lerodalcibep, a third generation PCSK9 inhibitor in heterozygous familial hypercholesterolemia subjects (LIBerate-HeFH_OLE); personalized app-based coaching to improve physical activity in patients with HFpEF compared to standard care (MyoMobile); semaglutide to improve cardiovascular outcomes in patients with a history of coronary artery bypass surgery and overweight or obesity (the SELECT trial); efficacy and safety of plozasiran in adults with genetically or clinically defined familial chylomicronemia syndrome at high risk of acute pancreatitis (PALISADE); and transcriptomic signatures and predictors of evolocumab added to maximum statin therapy based on intra-coronary plaque characteristics (YELLOW III). Research presented at the 2024 AHA Scientific Sessions emphasized innovative strategies in cardiovascular disease prevention and management.

18 Jul 2025·Current Atherosclerosis Reports

ApoC-III as Therapeutic Target: Is it Primetime for Clinical Use?

Review

Author: Tosin, Paola ; Simioni, Paolo ; Bertocco, Sandra ; Galimberti, Federica ; Previato, Lorenzo ; Zambon, Alberto ; Casula, Manuela ; Biolo, Marta ; Portinari, Camilla ; Zambon, Sabina

PURPOSE OF REVIEW:

Apolipoprotein C-III (ApoC-III) plays a pivotal role in triglyceride (TG) metabolism by inhibiting lipoprotein lipase and hepatic clearance of TG-rich lipoproteins, contributing to hypertriglyceridaemia and elevated cardiovascular risk, as well as a high risk of acute pancreatitis. This review aims to summarize current evidence on ApoC-III inhibition strategies.

RECENT FINDINGS:

Current treatments targeting Apo C-III include two antisense oligonucleotides (ASOs) (volanesorsen and olezarsen), and a small interfering RNA (siRNA) (plozasiran). Volanesorsen, a second-generation ASO, has shown effectiveness in reducing TG and preventing acute pancreatitis, especially in patients with familial chylomicronemia syndrome (FCS). However, its use is limited by the risk of thrombocytopenia, likely related to its chemical structure rather than ApoC-III inhibition itself. Olezarsen, a third-generation ASO with GalNAc conjugation for targeted liver delivery, offers an improved safety profile and strong efficacy in lowering TG and atherogenic lipoproteins levels, making it a promising candidate for a broader clinical use. Plozasiran, a GalNAc-conjugated siRNA, has shown robust and sustained TG reductions with a favorable safety profile, and early data suggest it may also reduce acute pancreatitis risk. ApoC-III inhibition represents an innovative and effective approach in managing hypertriglyceridaemia and its complications. Further outcome-driven trials are essential to define its role in cardiovascular risk reduction.

News (Medical) associated with Plozasiran

12 Aug 2025

·www.biospace.com

Caught In Sarepta ‘Downdraft,’ Arrowhead Becomes RNAi Knight in Shining Armor

iStock, Afry Harvy Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior. Arrowhead Pharmaceuticals’ shares have fallen 12% in the past month, but it had little to do with work going on at the RNA interference specialist. Instead, it was Arrowhead’s association with Sarepta Therapeutics—with which it has a partnership worth as much as $11 billion —that sowed the chaos at the markets. “We have been caught in that downdraft as well, unfortunately,” Arrowhead CEO Chris Anzalone told BioSpace , speaking to Sarepta’s gene therapy safety drama that has been playing out over the past month. “And it’s not unreasonable. I think that people were curious about Sarepta’s ability to perform on the partnership.” For the record, Anzalone isn’t worried about Sarepta dropping out of the alliance. He thinks the deal is too sweet for both sides. H.C. Wainwright Research agreed in a July 31 note, calling the partnership “symbiotic and mutually beneficial.” Annalee Armstrong for BioSpace “[Sarepta] have said quite clearly that—and I don’t want to put words in their mouth, but my interpretation is—the future of their company is based on the assets they got from us,” Anzalone said. Indeed, Sarepta last month announced a strategic restructuring and pipeline pivot away from its legacy gene therapies and toward a handful of siRNA platform assets that are partnered with Arrowhead. Even so, Anzalone understands why investors have been nervous. “Cash from Sarepta is a big part of our financial model over the next two years,” he said. HCW pointed out that the non-dilutive capital provided by Sarepta is key to Arrowhead’s plans to reach cash flow–positive operations by 2030. With some explaining of what happens if Sarepta doesn’t pay, Anzalone said that investors have been reaching the place he is at right now. “I’m almost at equivoce. The deal goes as planned. Great. The deal goes under, we get our assets back and we keep the cash. That’s also fine.” The Future of Sarepta Is Here Sarepta’s pipeline pivot should have been good news for Arrowhead—it was a vote of confidence for the biotech’s platform and a sign that Sarepta saw the massive partnership as its future. But a day later, the company’s months-long safety drama came to a head. On July 18, Sarepta revealed that a third patient had died after taking one of its gene therapies. This spurred a market tumble for both companies. While Arrowhead kept its head down, Sarepta became wrapped up in a very public dispute with the FDA. Analysts began to wonder if Sarepta could keep up its commitments to Arrowhead. The deal, signed in November 2024, is worth $825 million upfront, with as much as $10 billion possible later on through a series of consistent payments tied to enrollment and other clinical development milestones. The partnership is focused on rare genetic diseases of the muscle, central nervous system and lung, with two key Phase I muscular disease programs: ARO DM1 for type 1 myotonic dystrophy (DM1) and ARO-DUX4 for facioscapulohumeral muscular dystrophy type 1 (FSHD1). The deal also includes two other clinical-stage therapies and three preclinical programs. Arrowhead has been working swiftly behind the scenes on the Sarepta programs, already meeting one of the Phase I enrollment targets for ARO DM1 that would trigger a $100 million milestone. This payment is due by the end of the fourth quarter under the terms of the agreement. Anzalone doesn’t anticipate any issues with that payout. “I expect [Sarepta] to continue to perform, and we’ve seen no evidence that they will not,” Anzalone said. “I would read into that that they’re going to spend their last dollar to make sure that [the deal] stays intact, because it’s so important for them strategically.” But if they don’t, Anzalone said the contract has solid termination framework, which HCW also noted. If Sarepta stops paying, the assets fall back to Arrowhead and no cash has to be returned. “Look, I’m okay with either of those things—again, I don’t think the latter is going to happen. I think they’re going to perform,” Anzalone said. “The fail-safe here is that we get a bunch of really good assets back and move forward ourselves.” The CEO also expressed an openness to work with Sarepta in the future to tweak the deal if needed. While the situation is a little uncertain, Anzalone said he’s confident in one thing: “These are good assets.” Looking back at the deal process, Anzalone said he would still sign the same one today. Sarepta—which is known for its Duchenne muscular dystrophy therapies—was a natural fit for ARO-DUX4, an RNA interference (RNAi) conjugate in development for the progressive genetic muscle disease FSHD1. Anzalone remembers the scope of the partnership growing as discussions went on with Sarepta CEO Doug Ingram. Sarepta at that time was in the initial stages of commercializing Elevidys after its June 2023 approval—before it ran into the safety problems it faces today—but had a fairly empty pipeline. “They appear to be good at regulatory, commercial and late-stage development, but they didn’t have much of a pipeline. And we are nothing but pipeline,” Anzalone said. “So it just kind of grew. And next thing you knew, we were doing what was going to be a very large deal.” The final terms eventually work out to somewhere around $11 billion. Besides the $100 million ARO DM1 enrollment milestone, Anzalone expects to trigger another $200 million by the end of the year for the same program, plus a $50 million milestone in February 2026 for an R&D milestone. “We didn’t pick anybody’s pocket. This was, I think, as good a deal for Sarepta as it was for us,” Anzalone said. “This is one of those funny deals that I would be glad to take either side of the transaction.” Data from the first Sarepta-partnered programs ARO DM1 and ARO DUX4 are expected by the end of the year, and while there’s no firm date, Anzalone suspects Sarepta’s leadership will be eager to make the results public once they’re ready. He said those data are expected to be interpretable—which is to say, investors should get their first true assessment of the programs’ likelihood of success. “Too early data that doesn’t tell a story . . . can be confusing, but as soon as there is a nugget of a story, I’d like to put that out,” Anzalone said. Pizza and Beer Money The modality itself, RNAi, is derisked thanks to larger peer Alnylam Pharmaceuticals, and Arrowhead is largely seen as the next in line biopharma to capitalize on the technology. Besides Sarepta, Arrowhead is also partnered with Amgen, GSK and Takeda and is awaiting its first FDA approval this fall for the lead candidate in its wholly owned pipeline. Plozasiran is set for a Nov. 18 decision date for possible approval in familial chylomicronemia syndrome (FCS), a rare genetic metabolic disorder that prevents the body from breaking down fats. “[Arrowhead’s] RNAi pipeline is steadily advancing toward commercial-stage maturity, independent of partnership support,” HCW said. With such a focus on partnering, Anzalone said it was important to the team to take plozasiran all the way alone, even though Arrowhead could have continued as just an R&D company. With that kind of model, “you can stay capitalized and stay in what I call pizza and beer, but you can’t really make that leap to be an independent, longer-term valuable biotech company that say Vertex, Regeneron and the like have translated into,” the CEO said. To ensure success, Anzalone said the team has bulked up with regulatory experts and is focusing on the small FCS market first. “We, like any company who goes from an R&D-only organization to R&D plus commercial, are going to make a lot of dumb mistakes,” Anzalone admitted. “But it’s an ultra-rare indication and [has] allowed us to kind of get our feet wet and learn how to be a commercial organization.” Afterward, Arrowhead is targeting the much larger severe hypertriglyceridemia indication with plozasiran. The therapy is currently being tested in a trio of Phase III clinical trials with completion expected in mid-2026. These readouts could pave the way for Arrowhead’s second FDA nod. Besides the lead plozasiran program, Arrowhead has caught the obesity craze and is working on two early assets. Of the two, Anzalone is most excited about ARO-ALK7, which is meant to silence the ACVR1C gene, in turn reducing the risk of obesity-related metabolic complications. These two programs are not up for partnering, Anzalone says. Arrowhead is specifically focused on providing better weight loss and improving tolerability. “This is the health need of our time in western economies and so the GLP-1s are a very good start, but there’s a ton of white space there.”

Phase 3Phase 1Drug Approval

07 Aug 2025

·arrowheadpharma.com

Arrowhead Pharmaceuticals Reports Fiscal 2025 Third Quarter Results

Arrowhead Pharmaceuticals Reports... – Conference Call and Webcast Today, August 7, 2025, at 4:30 p.m. ET PASADENA, Calif.–(BUSINESS WIRE)–Aug. 7, 2025– Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced financial results for its fiscal 2025 third quarter ended June 30, 2025. The Company is hosting a conference call today, August 7, 2025, at 4:30 p.m. ET to discuss the results. “Arrowhead continues to achieve strong execution in discovery, clinical and regulatory, and business development. Our pipeline has become quite mature, with four Arrowhead discovered candidates currently in pivotal Phase 3 studies. In addition, our commercial buildout is designed to make us launch ready on day one, should plozasiran receive regulatory approval on the November 18, 2025, PDUFA date,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. “We continue to maintain a strong balance sheet, which we believe gives us the financial resources to move multiple innovative new medicines through the clinical and regulatory process and ultimately get them to the patients who need them.” Selected Recent Events Announced the signing of an asset purchase agreement between Sanofi and Visirna Therapeutics, a majority-owned subsidiary of Arrowhead created to develop and commercialize four of Arrowhead’s investigational cardiometabolic candidates in Greater China. Summary terms of the agreement include: Visirna will receive an upfront payment of $130 million from Sanofi. In addition, Visirna will be eligible to receive further milestone payments of up to $265 million upon approval of plozasiran across various indications in mainland China. Sanofi will receive an exclusive license to develop and commercialize investigational plozasiran in Greater China from Visirna Therapeutics, offering potential treatment to people living with elevated triglycerides. Earned a $100 million milestone payment from Sarepta Therapeutics when Arrowhead reached the first of two prespecified enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 clinical study of ARO-DM1, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy. Arrowhead currently expects to achieve the second enrollment target by the end of 2025, which would trigger an additional $200 million milestone payment from Sarepta. Completed enrollment in investigational plozasiran clinical studies SHASTA-3, SHASTA-4, and MUIR-3, the company’s global Phase 3 clinical studies designed to support regulatory submissions for marketing approval in the treatment of severe hypertriglyceridemia. Arrowhead anticipates completing the primary portion of these studies in mid-2026 with topline data expected shortly thereafter and planned submissions for regulatory review and potential approval to follow. The company previously submitted a New Drug Application (NDA) for plozasiran based on positive Phase 3 PALISADE study results in patients with familial chylomicronemia syndrome, which the U.S. FDA has accepted with a Prescription Drug User Fee Act (PDUFA) action date set for November 18, 2025. Dosed the first subject in the YOSEMITE Phase 3 clinical trial of zodasiran, the company’s investigational RNAi therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease. Zodasiran is the fourth investigational RNAi-based candidate developed by Arrowhead to reach late-stage pivotal studies, after investigational drugs plozasiran, fazirsiran (licensed to Takeda) and olpasiran (licensed to Amgen). Dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, the company’s investigational RNAi therapeutic being developed as a potential treatment for obesity. ARO-ALK7 is designed to intervene in a known pathway that signals the body to store fat in adipose tissue with a novel mechanism of action that may better preserve lean muscle mass compared to currently approved obesity therapies. The study initiates in otherwise healthy obese subjects using single and multiple escalating doses of ARO-ALK7 monotherapy and is expected to progress rapidly to investigate combinations of ARO-ALK7 with tirzepatide in obese patients with and without type 2 diabetes. Visirna will receive an upfront payment of $130 million from Sanofi. In addition, Visirna will be eligible to receive further milestone payments of up to $265 million upon approval of plozasiran across various indications in mainland China. Sanofi will receive an exclusive license to develop and commercialize investigational plozasiran in Greater China from Visirna Therapeutics, offering potential treatment to people living with elevated triglycerides. Selected Fiscal 2025 Third Quarter Financial Results ARROWHEAD PHARMACEUTICALS, INC. CONSOLIDATED CONDENSED FINANCIAL INFORMATION (in thousands, except per share amounts) Three Months Ended June 30, OPERATING SUMMARY 2025 2024 (Unaudited) Revenue $ 27,767 $ — Operating Expenses: Research and development 162,368 152,431 General and administrative expenses 30,949 23,710 Total operating expenses 193,317 176,141 Operating loss (165,550 ) (176,141 ) Total other (expense) income (13,539 ) 2,164 Loss before income tax expense and noncontrolling interest (179,089 ) (173,977 ) Income tax (benefit) expense (437 ) — Net loss including noncontrolling interest (178,652 ) (173,977 ) Net loss attributable to noncontrolling interest, net of tax (3,411 ) (3,184 ) Net loss attributable to Arrowhead Pharmaceuticals, Inc. $ (175,241 ) $ (170,793 ) Net loss per share attributable to Arrowhead Pharmaceuticals, Inc. – Diluted $ (1.26 ) $ (1.38 ) Weighted-average shares used in calculating – Diluted 139,039 124,199 FINANCIAL POSITION SUMMARY June 30, 2025 September 30, 2024 (unaudited) Cash, cash equivalents and restricted cash $ 129,793 $ 102,685 Available-for-sale securities, at fair value 770,579 578,276 Total cash resources (Cash, cash equivalents and restricted cash and Available-for-sale securities, at fair value) 900,372 680,961 Other current and long-term assets 480,240 458,841 Total Assets $ 1,380,612 $ 1,139,802 Liability related to the sale of future royalties $ 360,254 $ 341,361 Credit Facility 240,332 393,183 Deferred revenue 22,979 — Other liabilities 237,241 214,195 Total Liabilities $ 860,806 $ 948,739 Total Arrowhead Pharmaceuticals, Inc. Stockholders’ Equity 522,313 185,444 Noncontrolling Interest (2,507 ) 5,619 Total Noncontrolling Interest and Stockholders’ Equity $ 519,806 $ 191,063 Total Liabilities, Noncontrolling Interest and Stockholders’ Equity $ 1,380,612 $ 1,139,802 Shares Outstanding 138,144 124,376 Webcast and Conference Call and Details Investors may access a live audio webcast on the Events and Presentations page under the Investors section of the Arrowhead website. A replay of the webcast will be available approximately two hours after the conclusion of the call. For analysts that wish to participate in the conference call, please register at https://register-conf.media-server.com/register/BI3955ca6732974874b0bc0ac323811a7f. Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call. About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals 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, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company’s email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts. Safe Harbor Statement under the Private Securities Litigation Reform Act: This news release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release 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,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, 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 our preclinical studies and clinical trials, including the timing of achievement of the next ARO-DM1 milestone, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements, including our ability to achieve such milestones on projected timelines (if at all) and receive timely payment if milestones are achieved; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We disclaim any intention to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20250807601354/en/ Arrowhead Pharmaceuticals, Inc. Vince Anzalone, CFA 626-304-3400 ir@arrowheadpharma.com Investors: LifeSci Advisors, LLC Brian Ritchie 212-915-2578 britchie@lifesciadvisors.com Media: LifeSci Communications, LLC Kendy Guarinoni, Ph.D. 724-910-9389 kguarinoni@lifescicomms.com Source: Arrowhead Pharmaceuticals, Inc.

License out/inPhase 3Financial StatementNDA

01 Aug 2025

·pipelinereview.com

Arrowhead Subsidiary Visirna Sells Rights to Hypertriglyceridemia Candidate Plozasiran in Greater China to Sanofi

– Sanofi to pay $130 million upfront and up to $265 million in potential regulatory milestones to Visirna Therapeutics, a majority-owned subsidiary of Arrowhead, which was previously granted rights to investigational plozasiran in Greater China – Sanofi will receive an exclusive license to develop and commercialize investigational plozasiran in Greater China from Visirna Therapeutics, offering potential treatment to people living with elevated triglycerides PASADENA, CA, USA I August 1, 2025 I Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced the signing of an asset purchase agreement between Sanofi and Visirna Therapeutics, a majority-owned subsidiary of Arrowhead created to develop and commercialize four of Arrowhead’s investigational cardiometabolic candidates in Greater China. Under the terms, Sanofi will acquire rights to develop and commercialize investigational plozasiran, Arrowhead’s first-in-class RNA interference (RNAi) therapeutic candidate designed to reduce production of apolipoprotein C-III (APOC3) as a potential treatment for familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG), in Greater China. Visirna has completed a Phase 3 clinical trial (CTR20231418/NCT05902598) of investigational plozasiran in Chinese patients with familial chylomicronemia syndrome (FCS), which successfully met its primary efficacy endpoint and all key secondary endpoints. Visirna subsequently submitted a New Drug Application (NDA) for plozasiran to the National Medical Products Administration (NMPA) in China for the treatment of FCS and received official acceptance in January 2025. Plozasiran has also been granted Breakthrough Therapy Designation in the treatment of patients with FCS and Priority Review Designation by the China NMPA. “When we founded Visirna in 2022, we believed that Greater China represented an important potential future market for multiple programs in Arrowhead’s pipeline of investigational RNAi-based candidates for cardiometabolic diseases,” said Christopher Anzalone, Ph.D., Arrowhead’s President and CEO. “The team at Visirna understand the intricacies of China’s clinical, regulatory, and commercial environment and have done impressive work moving plozasiran through clinical studies and into the regulatory submission and review process. We now look forward to working with Sanofi and believe they are extremely well positioned as a global company with a strong presence in China.” Wayne Shi, President, Sanofi Greater China, added, “We are pleased to have concluded this agreement to enable us to advance plozasiran in Greater China where Sanofi has deep roots and a proud heritage of serving people living with a wide range of diseases. Plozasiran has shown considerable potential in studies across diverse patient populations where serious illness is caused by elevated triglycerides. With our strong presence in China’s cardiometabolic field, we now look forward to bringing it forward to address unmet need.” Upon closing of the Asset Purchase Agreement, Visirna will receive an upfront payment of $130 million from Sanofi. In addition, Visirna will be eligible to receive further milestone payments of up to $265 million upon approval of plozasiran across various indications in mainland China. Arrowhead is further eligible to receive royalties on net commercial product sales in Greater China as part of the Arrowhead-Visirna license which was assigned in part to Sanofi. Gibson, Dunn & Crutcher LLP and Sidley Austin LLP are serving as legal advisors to Arrowhead. About Plozasiran Plozasiran, previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (APOC3) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by receptors in the liver. The goal of treatment with plozasiran is to reduce the level of APOC3, thereby reducing triglycerides and restoring lipids to more normal levels. In multiple clinical studies, investigational plozasiran has demonstrated reductions in triglycerides and multiple atherogenic lipoproteins in patients with familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (SHTG), and mixed hyperlipidemia. Plozasiran has been generally well tolerated to date with treatment emergent adverse events reported that generally reflect the comorbidities and underlying conditions of the study populations. Across clinical studies and study populations, the most frequently reported treatment emergent adverse events for the 25 mg dose that is proposed for marketing approval were COVID-19, upper respiratory tract infection, headache, Type 2 diabetes mellitus, and abdominal pain. Plozasiran is being investigated in the SUMMIT program of clinical studies, which includes the PALISADE Phase 3 study in patients with FCS, the SHASTA Phase 2 and Phase 3 studies in patients with SHTG, and the MUIR Phase 2 and Phase 3 studies in patients with mixed hyperlipidemia. Plozasiran in the treatment of patients with FCS has been granted Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation by the U.S. Food and Drug Administration and Orphan Medicinal Product Designation by the European Medicines Agency. Investigational plozasiran has been submitted for marketing authorization in treatment of FCS to multiple global regulatory authorities but has not been reviewed or approved to treat any disease. About Visirna Therapeutics Visirna Therapeutics was founded in 2022 as a majority owned subsidiary of Arrowhead Pharmaceuticals. Headquartered in China with a global perspective, Visirna strives to emerge as a frontrunner in the advancement of siRNA therapeutics. The existing product portfolio comprises clinical stage siRNA candidates with a focus on cardiovascular/metabolic, and auto-immune ailments. About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals 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, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit www.arrowheadpharma.com , or follow us on X (formerly Twitter) at @ArrowheadPharma , LinkedIn , Facebook , and Instagram . To be added to the Company’s email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts . SOURCE: Arrowhead Pharmaceuticals

Phase 3License out/inBreakthrough TherapyOrphan DrugNDA

100 Deals associated with Plozasiran

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Familial chylomicronaemia syndrome	NDA/BLA	United States	Arrowhead Pharmaceuticals, Inc.	18 Nov 2024
Hypertriglyceridemia	Phase 3	United States	Arrowhead Pharmaceuticals, Inc.	26 Apr 2024
Hypertriglyceridemia	Phase 3	United States	Wei Ya Zhen Sheng Wu Ji Shu （ Su Zhou ） You Xian Gong Si	26 Apr 2024
Hypertriglyceridemia	Phase 3	United States	Ajinomoto Althea, Inc.	26 Apr 2024
Hypertriglyceridemia	Phase 3	Argentina	Ajinomoto Althea, Inc.	26 Apr 2024
Hypertriglyceridemia	Phase 3	Argentina	Wei Ya Zhen Sheng Wu Ji Shu （ Su Zhou ） You Xian Gong Si	26 Apr 2024
Hypertriglyceridemia	Phase 3	Argentina	Arrowhead Pharmaceuticals, Inc.	26 Apr 2024
Hypertriglyceridemia	Phase 3	Australia	Wei Ya Zhen Sheng Wu Ji Shu （ Su Zhou ） You Xian Gong Si	26 Apr 2024
Hypertriglyceridemia	Phase 3	Australia	Arrowhead Pharmaceuticals, Inc.	26 Apr 2024
Hypertriglyceridemia	Phase 3	Australia	Ajinomoto Althea, Inc.	26 Apr 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05902598 \| CTR20231418 (PRNewswire) Manual	Phase 3	Familial chylomicronaemia syndrome	37	VSA001 25 mg	thxeeyvsgs(cuvozxasac) = igsslwdtdj paynbozmqw (jjnccqxgla ) Met View more	Positive	17 Mar 2025
				VSA001 50 mg	thxeeyvsgs(cuvozxasac) = iimudeujsx paynbozmqw (jjnccqxgla ) Met View more
NCT05089084 (PALISADE, Company_Website) Manual	Phase 3	Familial chylomicronaemia syndrome	75	Plozasiran 25 mg	hdbxjdrupz(pcpuceoigv) = xuqreopnyd gxghjdvnvv (hauofgwqoi ) View more	Positive	18 Nov 2024
				Plozasiran 50 mg	hdbxjdrupz(pcpuceoigv) = pbszbzwfhf gxghjdvnvv (hauofgwqoi ) View more
NCT04998201 \| NCT04720534 (MUIR, Company_Website) Manual	Phase 2	Hypertriglyceridemia \| Hyperlipidemia, Familial Combined	418	Plozasiran (MUIR)	tvyqhsaesk(hsjmhwakfn) = tbgdohrqqm wvfuilchlv (mwqybctflu )	Positive	18 Nov 2024
				Plozasiran (SHASTA-2)	tvyqhsaesk(hsjmhwakfn) = qsnydnphfw wvfuilchlv (mwqybctflu )
NCT05089084 (PALISADE, BusinessWire) Manual	Phase 3	Familial chylomicronaemia syndrome	75	Plozasiran 25 mg	lexquyyjip(zsxympcxwx) = wympkjojma nfnlyzyayz (zaqhqormea ) View more	Positive	02 Sep 2024
				Plozasiran 50 mg	lexquyyjip(zsxympcxwx) = arufzmrtte nfnlyzyayz (zaqhqormea ) View more
NCT05089084 (PALISADE, Corporate Publications) Manual	Phase 3	Familial chylomicronaemia syndrome	75	Plozasiran 25 mg	unwjczsspv(wdxttzigfa) = ecnnlvjshq rufvexylom (wmcqbgkocl ) Met View more	Positive	03 Jun 2024
				Plozasiran 50 mg	unwjczsspv(wdxttzigfa) = kgpoatyrlv rufvexylom (wmcqbgkocl ) Met View more
NCT04998201 (MUIR, BusinessWire) Manual	Phase 2	Hyperlipidemias	-	Plozasiran 10mg	otkfyzxmko(onjluhiqjj) = juuvmkaleb xyvpztjirl (jhllrxeyfb ) View more	Positive	28 May 2024
				Plozasiran 25mg	otkfyzxmko(onjluhiqjj) = odxoisbjhu xyvpztjirl (jhllrxeyfb ) View more
NCT04998201 (MUIR, Pubmed) Manual	Phase 2	Hyperlipidemia, Familial Combined	353	Plozasiran 10 mg (quarterly doses)	zdyhdkrltg(aqldhozhec) = pmrbcrjulh tfaukuykib (sxtalfdaje, -59.0 to -40.6) View more	Positive	28 May 2024
				Plozasiran 25 mg (quarterly doses)	zdyhdkrltg(aqldhozhec) = xsbpziiixz tfaukuykib (sxtalfdaje, -65.1 to -46.8) View more
NCT04720534 (SHASTA-2, Biospace \| JAMA Cardiol \| Pubmed)	Phase 2	Hypertriglyceridemia	226	Plozasiran	qyuafehbek(byemmqigyo): Difference (%) = -57 (95% CI, -71.9 to -42.1), P-Value = <0.001 View more	Positive	08 Apr 2024
				Placebo
NCT04720534 (SHASTA-2, Corporate Publications) Manual	Phase 2	Hypertriglyceridemia	226	Plozasiran 10 mg	vgeemcboon(wuidigxjhj) = uzoslgkicc xjpozptlyt (jcxqpegaxk ) View more	Positive	13 Nov 2023
				Plozasiran 25 mg	vgeemcboon(wuidigxjhj) = eepiniiabr xjpozptlyt (jcxqpegaxk ) View more
NCT04998201 (MUIR, AHA2023) Manual	Phase 2	Dyslipidemias	353	ARO-APOC3 10mg Q12W	pdcliunhcg(rnbkxcpbvi) = xqobrxjcvu usqtpvfafw (mhcqkcmmrf, 3.55) View more	Positive	06 Nov 2023
				ARO-APOC3 25mg Q12W	pdcliunhcg(rnbkxcpbvi) = cgvzohqmtc usqtpvfafw (mhcqkcmmrf, 3.51) View more

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