Společnost Arrowhead Pharmaceuticals ve studii fáze I/II po úvodní dávce ARO-MAPT snížila hladinu proteinu tau v mozkomíšním moku zdravých dobrovolníků v průměru až o 73 %.
Want Diversified Upside in Biotechnology? Check out LABUArrowhead Pharmaceuticals NASDAQ: ARWR presented interim Phase I/II data for ARO-MAPT, an investigational small interfering RNA therapy designed to reduce tau protein expression in the central nervous system, alongside initial clinical results for its TRiM CNS delivery platform.
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The company said the platform uses a transferrin receptor 1-targeted Fab linked to an siRNA payload, with the goal of enabling subcutaneous administration and delivery across the blood-brain barrier. ARO-MAPT targets the MAPT gene, which produces tau, a protein implicated in Alzheimer’s disease and primary tauopathies including progressive supranuclear palsy, corticobasal degeneration and some forms of frontotemporal dementia.
Early healthy-volunteer data show tau reductions
James Hamilton, Arrowhead’s Head of R&D and Chief Medical Officer, said interim data from single-ascending-dose healthy-volunteer cohorts showed mean reductions in cerebrospinal fluid, or CSF, total tau of up to 73% following an initial loading dose. The largest individual reduction was up to 85%, he said.
Hamilton said the data showed evidence of a dose response, while placebo recipients showed no reductions in CSF tau. The company characterized the response as generally consistent across treated individuals.
Available safety findings supported continued dosing and further development, according to Hamilton. He said ARO-MAPT had been well tolerated to date, with no reported serious treatment-emergent adverse events, no neurobehavioral events such as confusion, and no persistent anemia.
The ongoing ARO-MAPT-SC-1001 study is designed to assess safety, pharmacokinetics and pharmacodynamic effects in healthy volunteers and people with early Alzheimer’s disease. Healthy-volunteer enrollment has been completed, while enrollment in Alzheimer’s patient cohorts is ongoing at sites in Canada, the U.K., Australia and New Zealand.
Arrowhead plans to provide additional data from multiple-ascending-dose healthy-volunteer cohorts in 2027 and expects an update from its Alzheimer’s patient cohorts that year as well.
Company highlights subcutaneous CNS delivery
Chief Executive Officer Chris Anzalone said the results represent Arrowhead’s first clinical proof of concept for delivery of siRNA to the central nervous system through a subcutaneous injection. He said the company believes the approach could potentially support dosing as infrequently as quarterly and, if developed successfully, could potentially be administered at home.
In preclinical monkey studies, Arrowhead said subcutaneous administration of ARO-MAPT reduced MAPT expression by more than 75% across multiple brain regions, including the hippocampus. The company also reported that repeat dosing in monkeys maintained gene silencing and supported up to 60% CSF tau knockdown four months after the last dose.
Hamilton said Arrowhead’s preclinical work suggested more homogeneous brain distribution from the subcutaneous platform than from intrathecal administration, which delivers drugs directly into spinal fluid. He said broader distribution could avoid high concentrations in the spinal cord and potentially improve silencing in disease-relevant brain regions.
Anzalone said the company has one wholly owned and one partnered CNS candidate in clinical development using the TRiM CNS platform, and expects another partnered candidate to enter the clinic in 2027. Arrowhead also plans to introduce at least three additional wholly owned CNS candidates into the clinic over the next 12 months, according to his remarks.
Tau remains an emerging therapeutic target
Adam Boxer, a professor of neurology at the University of California, San Francisco, reviewed the rationale for reducing tau in neurodegenerative diseases. He said the amount and location of insoluble tau in the brain are closely correlated with symptoms in Alzheimer’s disease and several primary tauopathies.
Boxer noted that two approved anti-amyloid therapies have shown modest slowing of cognitive decline in symptomatic Alzheimer’s disease, but said tau-directed treatments may provide another way to address disease progression. He discussed prior studies of tau-targeting antisense therapies, including results suggesting reductions in CSF tau and signals of slowed cognitive decline or neurodegeneration in certain settings.
However, Boxer said important questions remain regarding the optimal degree and duration of tau reduction. Tau has normal physiological roles, and while animal studies and human genetic evidence have not linked tau loss of function to disease, prior clinical findings with another tau-lowering therapy raised questions about whether adverse effects could result from the treatment approach or potentially from on-target tau reduction.
Potential next studies in PSP and FTD
Arrowhead said it has begun regulatory discussions regarding potential later-stage studies in MAPT-variant frontotemporal dementia and progressive supranuclear palsy. Hamilton said the company aims to finalize study designs following additional regulatory discussions over the next one to two quarters, with a goal of starting studies in 2027.
Boxer said PSP and certain forms of frontotemporal dementia may offer potential development advantages because they are rare, severe diseases with no approved disease-modifying therapies and generally faster clinical progression than Alzheimer’s disease. He added that clinical studies in these conditions can potentially be smaller and shorter, although he did not provide a specific regulatory outlook for ARO-MAPT.
In discussing future dose selection, Hamilton said the 6.4 mg/kg and 12.8 mg/kg regimens appeared broadly comparable after the initial loading dose. He said Arrowhead may favor the lower end of the range if higher doses do not show added benefit, though patient data and results after subsequent doses will be needed to guide that decision.
About Arrowhead Pharmaceuticals (NASDAQ:ARWR)Arrowhead Pharmaceuticals, Inc is a biopharmaceutical company focused on developing medicines that silence disease-causing genes. Its research is based primarily on RNA interference (RNAi), a biological process that reduces the production of specific proteins by targeting messenger RNA before it can be used to make those proteins.
The company uses its proprietary Targeted RNAi Molecule (TRiM) platform to design and deliver RNAi therapeutics to selected tissues, including the liver and lungs.
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].
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FDA zařadila plozasiran společnosti Arrowhead do programu STAR, což může zkrátit lhůtu pro posouzení žádosti o schválení léčby těžké hypertriglyceridemie na šest měsíců.
- FDA STAR pilot program aims to shorten the time from complete supplement submission to the action date to allow earlier patient access to therapies that address an unmet medical need
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the United States Food and Drug Administration (FDA) has granted plozasiran entry in the Split Real Time Application Review (STAR) pilot program in connection with Arrowhead’s upcoming supplemental new drug application (sNDA) for severe hypertriglyceridemia (sHTG). SHTG is a condition that significantly increases the risk of acute pancreatitis, which is associated with repeated hospital admissions and can be fatal.
We are committed to pursuing every potential opportunity to accelerate the process of bringing plozasiran to patients, and the FDA STAR Pilot program provides important benefits to sponsors with efficacy supplements.
Share The FDA STAR pilot program aims to shorten the time from the date of a complete sNDA submission to the action date for certain supplemental indications for already-approved drugs. To be granted entry in the STAR program, FDA must determine that clinical evidence from adequate and well-controlled investigation(s) indicates that the drug may demonstrate substantial improvement on a clinically relevant endpoint(s) over available therapies.
Once accepted into the STAR program, a supplemental application qualifies for a 6-month Priority Review compared to a 10-month standard review. In addition, FDA will generally target an “expedited review” for efficacy supplements in the STAR program, meaning an action at least 1 month before the Priority Review goal date is possible. The use of a Priority Review Voucher (PRV) is not necessary for programs that are granted entry in the STAR program, so Arrowhead retains the PRV previously purchased in August 2026.
“We are committed to pursuing every potential opportunity to accelerate the process of bringing plozasiran to patients, and the FDA STAR Pilot program provides important benefits to sponsors with efficacy supplements,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. “In addition, earning Priority Review for plozasiran without the need to use a PRV gives us the option to redeploy our voucher for a future application or monetize it.”
Arrowhead is on schedule to submit a sNDA for plozasiran in sHTG to the FDA by year-end 2026 and plans to seek regulatory approval with additional global regulatory authorities thereafter. Plozasiran has received regulatory approval, as REDEMPLO®, in the United States, the European Union, China, Australia, and Canada as an adjunct to diet to reduce triglycerides in adults with genetically confirmed or clinically diagnosed familial chylomicronemia syndrome (FCS), which is the most severe form of sHTG.
About Severe Hypertriglyceridemia
Severe hypertriglyceridemia (sHTG) is characterized by triglyceride (TG) levels greater than 500 mg/dL, with the most severe form being familial chylomicronemia syndrome (FCS) where TGs typically exceed 880 mg/dL. SHTG significantly increases the risk of acute pancreatitis (AP), which can often include recurrent attacks requiring repeat hospital admissions and worsening outcomes. AP risk is proportional to the number, characteristics, and concentration of triglyceride rich lipoproteins (TRLs), particularly chylomicrons, and increases as TGs rise. Elevated TGs can also increase the risk of atherosclerotic cardiovascular disease (ASCVD). Limited treatment options exist to sustainably reduce TGs below guideline directed risk thresholds.
About REDEMPLO® (plozasiran)
REDEMPLO (plozasiran) is currently approved by the U.S. Food and Drug Administration, Health Canada, China’s National Medical Products Administration, the Australian Therapeutic Goods Administration, and by the European Commission as an adjunct to diet to reduce triglycerides for adults with FCS. REDEMPLO is the first and only siRNA treatment approved in these countries to be studied in both clinically diagnosed and genetically confirmed patients living with FCS.
REDEMPLO is designed to suppress the production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting APOC3 with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is self-administered via subcutaneous injection once every three months.
REDEMPLO has been granted Orphan Medicinal Product Designation by the EMA for the treatment of patients with FCS, and Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation by the U.S. FDA for the treatment of patients with FCS and was also granted Breakthrough Therapy designation by the U.S. FDA in severe hypertriglyceridemia.
Sanofi acquired the rights to develop and commercialize REDEMPLO in Greater China, with Arrowhead retaining rights to REDEMPLO in all geographies, outside of Greater China.
For more information about REDEMPLO, visit Our Medicines.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM™) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.
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, products or product candidate or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates; and our beliefs and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products, demand for our products, 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, 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 assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.
Arrowhead Pharmaceuticals plánuje do konce roku podat žádost o rozšíření registrace plozasiranu pro léčbu těžké hypertriglyceridémie. Ve studiích SHASTA-3 a SHASTA-4 snížil triglyceridy asi o 80 %.
Want Diversified Upside in Biotechnology? Check out LABUArrowhead Pharmaceuticals NASDAQ: ARWR is expanding its commercial and clinical footprint around RNA interference therapies, with its recently launched triglyceride-lowering treatment REDEMPLO and several pipeline programs targeting cardiometabolic disease, obesity and central nervous system disorders.
Speaking at H.C. Wainwright's 28th Annual Global Investment Conference, Chief Executive Officer Chris Anzalone said the company has evolved from its initial focus on liver-targeted RNAi therapies into a broader platform company. Arrowhead now has approximately 21 or 22 drug candidates in clinical studies, he said, with clinical candidates representing five different cell types.
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“The first tissue that we could address is the liver, hepatocytes,” Anzalone said. “But we decided early on that in order to extract proper value from this technology and to treat as many patients as we could, we needed to get outside the liver.”
REDEMPLO Launch and Severe Hypertriglyceridemia Filing REDEMPLO, also known as plozasiran, is an APOC3 inhibitor designed to lower triglycerides. The drug is approved for familial chylomicronemia syndrome, or FCS, an ultra-orphan condition. Anzalone said the launch has progressed well, although contracting with payers required several quarters.
The company is now able to serve patients with both genetic FCS and what it describes as clinically defined FCS. Anzalone estimated there could be 15,000 to 20,000 clinically defined FCS patients. Arrowhead has expanded its commercial organization from an initial sales force of roughly 20 representatives and expects another phase of sales-force growth in January and February as it prepares for a potential severe hypertriglyceridemia, or SHTG, launch in the second quarter of 2027.
Arrowhead plans to submit a supplemental new drug application for plozasiran in SHTG by year-end. The company acquired a priority review voucher, which Anzalone said could reduce review timing by four months.
Chief Medical Officer and Head of R&D James Hamilton said results from the Phase 3 SHASTA-3 and SHASTA-4 trials showed about an 80% reduction from baseline in triglycerides among patients with severe hypertriglyceridemia, defined in the studies as triglycerides above 500. The triglyceride reduction was associated with statistically significant reductions in acute pancreatitis, with risk reductions ranging from 78% to more than 80%, depending on the population, he said.
In a subset of patients with triglycerides above 880 and a history of pancreatitis, Hamilton said the pooled SHASTA-3 and SHASTA-4 data showed a 100% risk reduction, with no events among patients receiving active treatment.
Hamilton also described the safety profile as “pretty clean,” citing no evidence of thrombocytopenia or hypersensitivity reactions and no clinically meaningful or statistically significant increase in liver fat versus placebo. The company’s MUIR study is complete and its database is locked. Hamilton said the study, which enrolled about 1,000 hypertriglyceridemia patients, will contribute to a safety database of at least 1,500 patients treated for one year and will be included in the supplemental application.
Anzalone said the company sees SHTG as an education-driven market that may develop gradually rather than produce an immediate rapid launch. He said Arrowhead estimates plozasiran could become a $3 billion to $4 billion annual U.S. drug at peak.
Additional Cardiometabolic Programs Arrowhead’s zodasiran program in homozygous familial hypercholesterolemia, or HoFH, has completed enrollment in its Yosemite study, with data expected around the middle of next year, according to Hamilton. He pointed to Phase 3 data presented by Visirna, which holds Chinese rights to zodasiran and is partially funded and owned by Arrowhead. Those data showed a greater than 40% reduction in LDL cholesterol, including a reported 44% reduction in an HoFH population.
Anzalone said he expects zodasiran to be Arrowhead’s next launch after a potential SHTG launch, describing it as a relatively straightforward addition for a sales force already calling on endocrinologists, cardiologists and lipidologists. He characterized HoFH as a small, ultra-orphan indication.
The company also recently reported first-in-human data for ARO-DIMER-PA, a therapy using two linked siRNAs as a single molecule to target PCSK9 and APOC3. Hamilton said top-line single-dose data showed APOC3 reductions greater than 80%, PCSK9 reductions greater than 70%, a 54% reduction in LDL cholesterol, triglyceride reductions above 70%, and an approximately 50% reduction in ApoB.
Arrowhead plans to complete the ongoing study and review data after a second dose before selecting two or three dose levels for a potential Phase 2b study in mixed hyperlipidemia. Hamilton said the company’s goal would be to pursue approval based on LDL reduction in a biomarker-driven Phase 3 program, potentially alongside a cardiovascular outcomes trial.
Obesity and CNS Pipeline Updates In obesity, Arrowhead is studying activin E and ALK7, targets in the same pathway. The company expects to provide additional data toward the end of the year, with an update focused primarily on ALK7. Hamilton said planned data include weight-loss measures across subpopulations, including patients with Type 2 diabetes, as well as MRI-based measurements of body composition, visceral fat, total fat and lean mass.
Arrowhead is also advancing ARO-MAPT, a subcutaneously administered siRNA designed to silence the MAPT gene and reduce tau, a protein implicated in Alzheimer’s disease and other tauopathies. Hamilton said the company expects to present healthy-volunteer data within the next month, focused mainly on safety and cerebrospinal fluid total tau knockdown. Enrollment of Alzheimer’s patients is ongoing, with those data expected next year.
Anzalone said Arrowhead expects several additional wholly owned CNS targets to enter the clinic over the next 18 months. He emphasized that the company’s broader pipeline remains central to its strategy even as investors focus on its first commercial product.
About Arrowhead Pharmaceuticals (NASDAQ:ARWR)Arrowhead Pharmaceuticals, Inc NASDAQ: ARWR is a biopharmaceutical company focused on developing medicines that silence disease-causing genes. The company uses RNA interference (RNAi), a biological process that reduces the production of specific proteins, to target conditions that may be difficult to treat with conventional drugs.
Arrowhead's proprietary TRiM platform is designed to deliver RNAi-based therapies to different tissues, including the liver, lungs and other organs. Its investigational pipeline has included candidates for cardiovascular and metabolic diseases, liver disorders, pulmonary conditions and inflammatory diseases.
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].
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Arrowhead Pharmaceuticals uvedla, že na konci srpna představila plné 12měsíční výsledky studií SHASTA-3 a SHASTA-4 u těžké hypertriglyceridémie. Firma plánuje do konce roku podat doplňující žádost o registraci (NDA) s využitím priority review voucheru.
Arrowhead Pharmaceuticals, Inc. (ARWR) H.C. Wainwright 28th Annual Global Investment Conference September 16, 2026 9:00 AM EDT
Company Participants
Dr. Christopher Anzalone - Chairman, CEO & President
James Hamilton - Chief Medical Officer and Head of R&D
Conference Call Participants
Patrick Trucchio - H.C. Wainwright & Co, LLC, Research Division
Presentation
Patrick Trucchio
H.C. Wainwright & Co, LLC, Research Division
Okay. Perfect. Hello, everyone. Good morning, and welcome back to H.C. Wainwright's 20th Annual Global Investment Conference. I'm Patrick Trucchio, senior health care analyst at H.C. Wainwright. It's my pleasure to welcome our next speakers, CEO, Chris Anzalone; and CMO and Head of R&D, James Hamilton at Arrowhead Pharmaceuticals.
Arrowhead is a commercial stage pharmaceutical company developing RNA interference or RNAi therapeutics with broad pipeline across cardiometabolic indications and CNS. Arrowhead REDEMPLO, plozasiran, was approved by the U.S. FDA in 2025 as an adjunct to diet to treat triglycerides in adults with familial chylomicronemia syndrome or FCS. At the end of August, Arrowhead presented the full 12-month results of the pivotal Phase III SHASTA-3 and SHASTA-4 trials in severe hypertriglyceridemia, or sHTG, in a hotline late-breaking science session at the European Society of Cardiology Congress and plan to file a supplemental NDA by year-end in sHTG using a priority review voucher. So it's a huge year for Arrowhead. Pleasure to have you with us.
Question-and-Answer Session
Patrick Trucchio
H.C. Wainwright & Co, LLC, Research Division
Just first, it's been a transformational last few months, FCS launch, 2 positive pivotal trials, priority review voucher. For those who are new to the Arrowhead story, can you give us an overview of the RNAi platform, why subcutaneous RNAi to the liver became such a robust franchise?
Dr. Christopher Anzalone
Chairman, CEO & President
Sure. First, thanks very much for having us. It's a pleasure to be
Arrowhead Pharmaceuticals oznámila, že její ARO-DIMER-PA v rané studii snížil PCSK9 až o 72 % a APOC3 až o 88 %. Současně klesly LDL-C o 54 % a triglyceridy o 73 %.
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced interim topline data from a Phase 1/2a clinical trial of ARO-DIMER-PA, the company’s first investigational RNA interference (RNAi) therapeutic dimer being developed as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia. ARO-DIMER-PA is designed to silence expression of both proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes. Additional results will be submitted for presentation at an upcoming medical congress.
“With these interim topline results, Arrowhead’s innovative and proprietary Targeted RNAi Molecule (TRiM™) platform has achieved clinical validation of its ability to target and silence two genes simultaneously in one molecule. This represents an important step forward and a first for the field of RNAi therapeutics. We look forward to presenting additional details on this groundbreaking advance at an upcoming medical congress,” said Chris Anzalone, Ph.D., President and CEO at Arrowhead Pharmaceuticals. “This validation potentially expands the number of diseases and patients worldwide that may be helped by Arrowhead’s RNAi therapies. ARO-DIMER-PA also fits well strategically with Arrowhead’s growing focus on cardiometabolic diseases. It is complemented by our existing portfolio, which includes our first commercial product REDEMPLO® (plozasiran), now approved in the U.S., European Union, Canada, Australia, and China for the treatment of familial chylomicronemia syndrome (FCS), and investigational zodasiran, which is currently being studied in the global Phase 3 YOSEMITE clinical trial in patients with homozygous familial hypercholesterolemia (HoFH).”
James Hamilton, M.D., MBA, Chief Medical Officer and head of R&D at Arrowhead, added, "The relationship between low-density lipoprotein-cholesterol (LDL-C) and Apolipoprotein B (ApoB) reduction and cardiovascular risk is one of the most consistently replicated findings in modern medicine. Over the past three decades, numerous clinical studies have shown that lowering these markers translates into fewer heart attacks, strokes, and cardiovascular deaths. The early single dose data with ARO-DIMER-PA in patients with mixed hyperlipidemia give us a high degree of confidence that the robust reductions in LDL-C and ApoB that we have seen, along with robust reductions in additional atherogenic lipoproteins, have the potential to translate into a reliable clinical benefit in later stage studies.”
Steven Nissen, M.D., Chief Academic Officer for the Heart and Vascular Institute at the Cleveland Clinic, the Lewis and Patricia Dickey Chair in Cardiovascular Medicine and Professor of Medicine at the Lerner College of Medicine, added, “Mixed hyperlipidemia is not adequately addressed by treating LDL-C alone. Even with intensive statin therapy and PCSK9 inhibitors, substantial ASCVD risk remains, and triglyceride-rich remnant lipoproteins may be an important part of that residual risk. This medication represents a new approach to targeting multiple lipid abnormalities with a single therapy.”
Select ARO-DIMER-PA Phase 1/2a Interim Single Dose Results
In participants with mixed hyperlipidemia, ARO-DIMER-PA single doses achieved dose-dependent mean maximal reductions in serum PCSK9 of 72% and APOC3 of 88%Silencing of both the PCSK9 and APOC3 genes led to favorable changes in lipids and lipoproteins including the following mean maximal reductions:LDL-C reduction of 54%Triglyceride (TG) reduction of 73%Non-high density lipoprotein (non-HDL) cholesterol reduction of 61%Apolipoprotein B (ApoB) reduction of 50%Interim Safety and Tolerability
Single dose escalation has been completed through 400 mg. The most commonly reported treatment emergent adverse events (TEAE) were injection site events and headaches. No drug related SAEs were reported. The study is ongoing to assess safety and tolerability of multiple doses.
About Mixed Hyperlipidemia
Mixed hyperlipidemia is a highly prevalent disorder characterized by elevated levels of both LDL-C and TGs. It is a major risk factor for ASCVD, which is the leading cause of mortality worldwide and associated with substantial morbidity and healthcare costs. Despite the efficacy of LDL-C-lowering therapies in reducing ASCVD risk, there remains substantial residual risk in patients with mixed hyperlipidemia.
About ARO-DIMER-PA
ARO-DIMER-PA is a dual-functional RNAi molecule designed to silence expression of both PCSK9 and APOC3 genes in hepatocytes. Prior clinical experience with other investigational and approved agents suggests that PCSK9 and APOC3 inhibition may lead to robust reductions in LDL-C, TGs, triglyceride rich lipoprotein remnants, and total atherogenic lipoproteins.
ARO-DIMER-PA is the first clinical candidate designed to selectively silence the expression of two genes with a single RNAi molecule.
About the ARODIMER-PA-1001 Phase 1/2a Study
ARO-DIMER-PA-1001 (NCT07223658) is a Phase 1/2a placebo-controlled dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and effects on LDL-C and TGs of single-dose ARO-DIMER-PA (part 1) and multiple doses of ARO-DIMER-PA (part 2) in up to 78 adult subjects with mixed hyperlipidemia.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM™) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.
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, products or product candidate or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates; and our beliefs and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products, demand for our products, 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, 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 assume no obligation 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/20260915705822/en/
Arrowhead Pharmaceuticals uvedla, že plozasiran ve 3. fázi snížil triglyceridy zhruba o 80 % a v souhrnu o asi 80 % omezil akutní pankreatitidu oproti placebu. Firma plánuje podat žádost o schválení před koncem roku.
Want Diversified Upside in Biotechnology? Check out LABUArrowhead Pharmaceuticals NASDAQ: ARWR presented 12-month results from its Phase 3 SHASTA-3 and SHASTA-4 trials evaluating plozasiran in adults with severe hypertriglyceridemia, reporting substantial reductions in triglycerides and acute pancreatitis events versus placebo. The company said the data were presented at the European Society of Cardiology Congress in Munich and have been accepted for publication in a major medical journal.
Plozasiran, marketed as REDEMPLO for familial chylomicronemia syndrome, or FCS, is approved to reduce triglycerides in FCS patients in the U.S., European Union, Canada, Australia and China. The severe hypertriglyceridemia indication remains investigational and has not been reviewed or approved by regulators, Arrowhead said.
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Phase 3 results Gerald Watts, Winthrop Professor at the University of Western Australia, presented the findings from the two pivotal trials. SHASTA-3 and SHASTA-4 enrolled adults with severe hypertriglyceridemia, defined as triglyceride levels above 500 milligrams per deciliter. Participants received 25 milligrams of plozasiran or placebo through four subcutaneous injections over 12 months, alongside background dietary and conventional lipid-lowering treatment.
Watts said plozasiran produced triglyceride reductions of about 80% from baseline at three months, with reductions sustained through 12 months. More than 90% of plozasiran-treated patients reached triglyceride levels below 500 milligrams per deciliter, a threshold associated with elevated acute pancreatitis risk. More than half of treated patients achieved normal fasting triglyceride levels below 150 milligrams per deciliter.
Secondary measures also showed reductions in remnant cholesterol, non-HDL cholesterol and apolipoprotein C3, or ApoC3, according to the presentation. Plozasiran is designed to reduce hepatic production of ApoC3, a regulator of triglyceride metabolism.
In a pooled analysis of the two studies, plozasiran reduced cumulative acute pancreatitis events by roughly 80% relative to placebo, Watts said. The presentation cited a 44.1% absolute risk reduction and a number needed to treat of 24 over one year for the overall trial population.
In exploratory analyses of higher-risk groups, the company reported more than a 90% relative reduction in acute pancreatitis among patients with a prior history of the condition. In a smaller subgroup with triglycerides above 880 milligrams per deciliter and prior acute pancreatitis, Arrowhead reported no acute pancreatitis events among plozasiran-treated patients. Watts cautioned that the subgroup involved small numbers and was exploratory.
Safety observations Watts said patient retention exceeded 90% and adherence to the four-dose regimen was nearly 100%. Treatment discontinuations due to adverse events were low and similar between treatment groups, according to the presentation.
The company reported no anaphylaxis, systemic hypersensitivity or meaningful changes in platelet counts, liver enzymes or liver fat fraction relative to placebo. Investigators observed a small increase in measures related to glycemic control in some analyses, though Watts described the change in glycated hemoglobin, or HbA1c, as small and not clinically meaningful at the end of treatment.
Børge Nordestgaard, professor and chief physician at Copenhagen University Hospital, said the glycemic-control observation appeared consistent with the broader ApoC3 inhibitor class. He described the safety profile presented for plozasiran as promising while noting that longer-term follow-up will be needed.
Market focus and regulatory plans Jennifer Hellawell, Arrowhead’s head of clinical development in cardiometabolic disease, said severe hypertriglyceridemia affects approximately 1% of the population, or more than 3 million people in the U.S. The company defines a high-risk segment of about 1 million U.S. patients as those with triglycerides at or above 880 milligrams per deciliter, or those above 500 milligrams per deciliter with a prior history of acute pancreatitis.
Andy Davis, Arrowhead’s head of cardiometabolic commercial operations, said the company intends to initially focus a potential severe hypertriglyceridemia launch on those high-risk patients. Arrowhead has identified more than 20,000 healthcare professionals across lipidology, endocrinology, preventive cardiology, internal medicine and primary care who treat these patients, Davis said.
Vince Anzalone, Arrowhead’s senior vice president of finance and investor relations, said the company remains on schedule to file a supplemental new drug application seeking approval for plozasiran in severe hypertriglyceridemia before year-end. Arrowhead recently purchased a priority review voucher, which Anzalone said could accelerate the potential U.S. review timeline. Subject to a positive regulatory review, the company is targeting a potential launch in 2027.
Arrowhead estimates the U.S. severe hypertriglyceridemia opportunity could represent $3 billion to $4 billion in annual sales over the long term. The company also highlighted other cardiometabolic programs, including zodasiran for homozygous familial hypercholesterolemia and ARO-DIMER-PA, an investigational dual-targeting therapy for mixed hyperlipidemia.
About Arrowhead Pharmaceuticals (NASDAQ:ARWR)Arrowhead Pharmaceuticals, Inc is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Since its founding in 2008, Arrowhead has leveraged its proprietary delivery platform—known internally as the Advanced RNAi Compound (ARC) technology—to silence disease-causing genes in patients suffering from genetically defined diseases. The company's approach aims to offer durable, targeted treatments across a range of therapeutic areas.
The company's pipeline includes multiple candidates in various stages of development.
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Arrowhead Pharmaceuticals oznámila pozitivní výsledky fáze III pro plozasiran u těžké hypertriglyceridémie a chystá podání žádosti sNDA do konce roku 2026. Tržby ve 3. čtvrtletí vzrostly na zhruba 75 milionů USD.
Want Diversified Upside in Biotechnology? Check out LABUArrowhead Pharmaceuticals NASDAQ: ARWR reported positive Phase III results for plozasiran in severe hypertriglyceridemia (SHTG), accelerating plans for a supplemental New Drug Application and highlighting continued growth in the launch of its REDEMPLO therapy for familial chylomicronemia syndrome (FCS).
For the fiscal third quarter ended June 30, 2026, Arrowhead recorded a net loss of $194.3 million, or $1.36 per diluted share, compared with a loss of $175.2 million, or $1.26 per share, a year earlier. Revenue rose to approximately $75 million from $28 million in the prior-year period, driven by collaboration agreements and commercial REDEMPLO sales.
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Phase III SHTG Results and Regulatory Plans President and Chief Executive Officer Dr. Chris Anzalone said the company’s Phase III SHASTA-3 and SHASTA-4 studies both met their primary endpoint and all prespecified secondary endpoints in patients with SHTG.
Median triglyceride reductions from baseline were 79% in SHASTA-3 and 81% in SHASTA-4. In the studies’ placebo groups, reductions were approximately 27%, according to Andy Davis, senior vice president and head of the Global Cardiometabolic Franchise.
Arrowhead also reported a statistically significant reduction in acute pancreatitis events in a prespecified pooled analysis of the trials. Davis said plozasiran reduced cumulative acute pancreatitis events by 78% versus placebo across the broader SHTG population. In a subgroup of patients with triglyceride levels above 880 milligrams per deciliter and a prior history of acute pancreatitis, the company reported a 100% reduction in events versus placebo.
The company said safety and tolerability findings were consistent with prior plozasiran studies. Anzalone said Arrowhead observed no new safety signals, no clinically meaningful adverse changes in liver enzymes, no hypersensitivity cases and no thrombocytopenia signal. In a prespecified MRI-PDFF subgroup, there was no statistically significant difference in mean liver fat content between plozasiran and placebo.
Detailed SHASTA results are scheduled for presentation at the European Society of Cardiology Congress on Aug. 30, followed by an Arrowhead webcast on Aug. 31. Chief Medical Officer and Head of R&D Dr. James Hamilton said the company remained under embargo regarding further trial details ahead of the conference.
Arrowhead intends to submit an sNDA to the Food and Drug Administration before the end of 2026. The company acquired an FDA priority review voucher that could shorten the agency’s review period from 10 months to six months. Chief Financial Officer Dan Apel said Arrowhead expects to pay $215 million for the voucher during its fiscal fourth quarter, following Hart-Scott-Rodino clearance.
Management said the initial focus of a potential SHTG launch would likely be patients at the highest risk, while emphasizing that the clinical data support treatment across patients with triglyceride levels above 500 milligrams per deciliter. Anzalone said the company expects SHTG to be an education-driven market because physicians have historically had limited options for substantially reducing triglycerides.
REDEMPLO Launch Expands in FCS REDEMPLO prescription volume more than doubled during the fiscal third quarter and that momentum continued into the current quarter, Davis said. Arrowhead has supported more than 400 unique prescribers, led by preventive cardiologists and endocrinologists.
Davis said the company was receiving approximately 20 to 30 new prescriptions per week, consistent with its previously disclosed run rate. Arrowhead is working to improve the progression of prescriptions through prior authorization and appeals processes and expects an additional wave of field personnel to enter the market during August.
REDEMPLO has favorable policies in place with the most significant U.S. payers, according to Davis, and the company expects remaining coverage gaps to narrow in the coming months. Nearly all published payer policies allow clinicians to diagnose FCS through clinical criteria rather than genetic confirmation alone.
The therapy’s U.S. wholesale acquisition cost remains $45,000 per patient annually. Management said it does not intend to change the price following the SHASTA results, citing REDEMPLO’s efficacy, safety profile, quarterly dosing schedule and 25-milligram fixed dose.
REDEMPLO is approved for FCS in the United States, Canada, China, Australia and the European Union. Arrowhead said reimbursement processes in Europe will proceed country by country over approximately 12 months, beginning with Germany. Sanofi leads commercialization in Greater China.
Pipeline Readouts Ahead Arrowhead expects several clinical data releases before year-end. The company plans to report top-line Phase I data for ARO-DIMER-PA in September. The candidate is designed to silence both APOC3 and PCSK9 to reduce LDL cholesterol and triglycerides in patients with mixed hyperlipidemia.
The company also expects September data from its Phase I healthy-volunteer study of ARO-MAPT, a subcutaneously administered RNA interference therapy targeting tau. Hamilton said the release will focus on safety and total tau knockdown, with Arrowhead targeting approximately 50% to 60% knockdown. The study’s second phase in Alzheimer’s disease patients is actively enrolling.
For obesity and metabolic dysfunction-associated steatohepatitis, Arrowhead plans to provide an update primarily focused on ARO-ALK7 in the fourth quarter. It has submitted an ARO-INHBE Phase IIb protocol to regulators. Earlier data showed a placebo-adjusted 44% reduction in liver fat in a small subgroup of patients with obesity and elevated baseline liver fat receiving at least a 200-milligram dose as monotherapy.
Separately, Arrowhead completed enrollment in the Phase III YOSEMITE trial of its therapy for homozygous familial hypercholesterolemia, enrolling 70 patients compared with a planned 60. The company expects study completion in mid-2027 and data in the second half of 2027.
Financial Position and Collaboration Revenue Apel said the quarter’s revenue included approximately $26 million from the Sarepta collaboration, about $20 million from the Novartis collaboration and the full $25 million upfront payment from Madrigal for the ARO-PNPLA3 license and technology transfer.
Commercial REDEMPLO revenue was approximately $2.4 million for the quarter, more than double the roughly $1 million reported in the prior fiscal quarter. Arrowhead said it does not plan to separately highlight product sales until they become a more meaningful financial contributor.
Operating expenses increased to approximately $245 million from $193 million a year earlier, including research and development expense of $198 million and selling, general and administrative expense of $47 million. The increases reflected clinical development, manufacturing activity and commercialization investment.
Arrowhead ended the quarter with approximately $1.6 billion in cash and investments. Apel said the company believes its balance sheet provides flexibility to fund pipeline development, commercial activities and longer-term strategic priorities.
About Arrowhead Pharmaceuticals (NASDAQ:ARWR)Arrowhead Pharmaceuticals, Inc is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Since its founding in 2008, Arrowhead has leveraged its proprietary delivery platform—known internally as the Advanced RNAi Compound (ARC) technology—to silence disease-causing genes in patients suffering from genetically defined diseases. The company's approach aims to offer durable, targeted treatments across a range of therapeutic areas.
The company's pipeline includes multiple candidates in various stages of development.
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].
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Should You Invest $1,000 in Arrowhead Pharmaceuticals Right Now?Before you consider Arrowhead Pharmaceuticals, you'll want to hear this.
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Arrowhead Pharmaceuticals získala v EU schválení pro REDEMPLO® (plozasiran) u dospělých se syndromem familiární chylomikronémie. Lék snižuje triglyceridy a je prvním i jediným schváleným siRNA přípravkem pro FCS v EU.
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the European Commission (EC) has formally granted marketing authorization for REDEMPLO® (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS). REDEMPLO is the first and only siRNA medicine authorized by the EC for adults with FCS, diagnosed either by the presence of clinical criteria or genetic testing.
Importantly, the ability to diagnose and treat without requiring a genetic test could enable earlier treatment, which is particularly relevant in rare diseases such as FCS.
Share“FCS drives an elevated risk of recurrent and potentially fatal episodes of acute pancreatitis. Results from the PALISADE study demonstrate that plozasiran can achieve significant and sustained reductions in triglycerides for these patients,” said Professor Børge Nordestgaard, Department of Clinical Medicine, University of Copenhagen and President, European Atherosclerosis Society. “Importantly, the ability to diagnose and treat without requiring a genetic test could enable earlier treatment, which is particularly relevant in rare diseases such as FCS.”
Announcing its recommendation for the approval of REDEMPLO, the European Medicines Agency noted, "Although other authorised medicines can help people with FCS confirmed by genetic testing, REDEMPLO does not require genetic confirmation of the condition, thus providing a treatment option for more adults with FCS and addressing the unmet medical need in these patients."
“Today's approval marks a pivotal moment for people living with familial chylomicronemia syndrome. As a patient myself and having spoken with countless others living with FCS through leading our organization, I know firsthand how devastating the burden of FCS is on every dimension of daily life. The constant uncertainty, the worry, the fear of acute pancreatitis, the chronic pain and fatigue are challenges the FCS community faces every single day, on top of the long and often frustrating journey to receiving a diagnosis,” added Rosa Pérez Jiménez, President of Familial Chylomicronemia Association (Asociación de Quilomicronemia Familiar) Spain. “This new therapeutic option gives renewed hope to patients who have waited far too long to be seen, understood, and treated.”
Harnessing Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform, REDEMPLO is designed to suppress production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by inhibiting their breakdown and clearance.
“We are pleased to have received EC approval for REDEMPLO as a new treatment option for people living with genetically or clinically confirmed FCS. With this approval secured, we are engaging with relevant national authorities and healthcare communities across the European Union to bring REDEMPLO to people living with FCS as quickly and efficiently as possible,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead Pharmaceuticals. “This ongoing cadence of regulatory approvals around the world reflects the strength of our clinical data and the real progress being made across our diverse pipeline of siRNA-based therapies that leverage our proprietary TRiM™ platform.”
EC regulatory approval was supported by clinical data from the Phase 3 PALISADE study, a randomized, double-blind, placebo-controlled trial in 75 adults with clinically diagnosed or genetically confirmed FCS.1,2 The PALISADE study met its primary endpoint and all multiplicity-controlled key secondary endpoints. In PALISADE, 25 mg REDEMPLO reduced triglycerides by a median of 80% from baseline versus a 17% reduction with placebo. Additionally, the combined doses of 25 mg and 50 mg plozasiran significantly reduced the incidence of acute pancreatitis (odds ratio, 0.169; p=0.0292). The odds of acute pancreatitis were 83% lower in the pooled plozasiran groups compared with the placebo group. The most common adverse reactions were hyperglycaemia (12.8%), headache (6.8%), nausea (4.7%), and injection site reaction (4.7%).1,2
About Familial Chylomicronemia Syndrome (FCS)
Familial chylomicronemia syndrome is a severe and rare disease leading to extremely high triglyceride (TG) levels, typically over 10 mmol/L (880 mg/dL). Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, there are limited therapeutic options to adequately treat FCS.
About REDEMPLO® (plozasiran)
REDEMPLO (plozasiran) is currently approved by the U.S. Food and Drug Administration, Health Canada, China’s National Medical Products Administration, the Australian Therapeutic Goods Administration, and by the European Commission as an adjunct to diet to reduce triglycerides for adults with FCS. REDEMPLO is the first and only siRNA treatment approved in these countries to be studied in both clinically diagnosed and genetically confirmed patients living with FCS.
REDEMPLO is designed to suppress the production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting APOC3 with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is self-administered via subcutaneous injection once every three months.
REDEMPLO has been granted Orphan Medicinal Product Designation by the EMA for the treatment of patients with FCS, and Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation by the U.S. FDA for the treatment of patients with FCS. In December 2025, plozasiran was also granted Breakthrough Therapy designation by the U.S. FDA in severe hypertriglyceridemia.
Plozasiran is also being investigated in the SHASTA-3 (NCT06347003), SHASTA-4 (NCT06347016), and SHASTA-5 (NCT06880770) Phase 3 studies in adults with severe hypertriglyceridemia and the MUIR-3 (NCT06347133) Phase 3 study in adults with hypertriglyceridemia.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM™) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.
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, products or product candidates or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts of REDEMPLO (plozasiran) on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates if approved; and our beliefs and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products if approved, demand for our products, 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, 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 assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.
References
Watts GF, Rosenson RS, Hegele RA, Goldberg IJ, Gallo A, Mertens A, Baass A, Zhou R, Muhsin M, Hellawell J, et al. Plozasiran for managing persistent chylomicronemia and pancreatitis risk. N Engl J Med. 2024;392:127–137. https://doi.org/10.1056/nejmoa2409368 PMID: 39225259.Watts GF, Hegele RA, Rosenson RS et al. Temporal Effects of Plozasiran on Lipids and Lipoproteins in Persistent Chylomicronemia. Circulation. 2025:151(10); 733-736; https://doi.org/10.1161/CIRCULATIONAHA.124.072860 PMID:39549263.Source: Arrowhead Pharmaceuticals, Inc.
