RegulationBreakthrough Therapy (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), Fast Track (United States)

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

/ 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 288 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 Date24 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 AROAPOC3-2003 (USA and Canada participants only; NCT# 05413135), AROAPOC3-3001(Canada and Japan participants only; NCT05089084), AROAPOC3-3003 (NCT06347003), AROAPOC3-3004 (NCT06347016) or AROAPOC3-3009 (Argentina, Italy, South Africa, and Spain; NCT06347133).
Subjects who previously met all eligibility requirements for AROAPOC3-3003 or AROAPOC3-3004 and were not permitted to proceed to randomization per the Sponsor's direction in order to prevent excessive over-enrollment may also be enrolled in this trial. The subjects must meet all other applicable eligibility criteria prior to enrollment and have an HbA1c results of <=10% within 30 days prior to Day 1.
Subjects entering this OLE from AROAPOC3-2003 must meet the following additional criteria to be considered for enrollment in addition to applicable eligibility criteria:
1. HbA1c ≤10% within 30 days prior to Day 1
2. Completed AROAPOC3-2001 prior to entry into AROAPOC3-2003 AND fulfill either (c) or (d)
3. Baseline fasting TG level of ≥500 mg/dL and prior history of acute pancreatitis at the time of enrollment into AROAPOC3-2001
4. Baseline fasting TG level of ≥1000 mg/dL at the time of enrollment into AROAPOC3-2001
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 Apr 2026·Current Opinion in Endocrinology Diabetes and Obesity

Lessons from recent clinical trials for the prevention of acute pancreatitis in chylomicronemia syndromes

Article

Author: Arca, Marcello ; Tramontano, Daniele ; D'Erasmo, Laura

Purpose of review:

Familial chylomicronemia (FCS) and multifactorial or persistent chylomicronemia syndromes (MCS, pCS) are rare, severe disorders characterized by extreme hypertriglyceridemia and a high risk of recurrent, potentially life-threatening acute pancreatitis. Most patients do not achieve adequate triglyceride control with lifestyle interventions or conventional lipid-lowering therapies, leaving them exposed to persistent complications. This review critically examines emerging therapeutic strategies aimed at improving triglyceride control and reducing acute pancreatitis risk.

Recent findings:

Advances targeting key molecular regulators of triglyceride metabolism have shown substantial promise. APOC3 inhibitors, including volanesorsen, olezarsen, and plozasiran, achieve up to 80% reductions in triglycerides and markedly lower AP incidence, with favorable safety profiles. ANGPTL3 inhibition via evinacumab may benefit patients with residual lipoprotein lipase activity, including polygenic or mixed chylomicronemia, and could be used during acute sHTG episodes. Lomitapide, acting independently of LPL, is effective in selected FCS patients but requires careful hepatic monitoring. FGF21 analogs, such as pegozafermin, are in early development and show potential for metabolic dysfunction-associated steatotic liver disease, though their impact on acute pancreatitis prevention remains to be established.

Summary:

These emerging mechanism-based therapies are reshaping the management of severe hypertriglyceridemia, offering targeted approaches to reduce triglycerides and acute pancreatitis risk. Ongoing studies will clarify long-term safety, durability of response, and optimal patient selection, providing a framework for improved clinical outcomes.

01 Mar 2026·VACCINE

VSA-2-, a novel plant-derived adjuvant for SARS-CoV-2 subunit vaccine

Article

Author: Zou, Jing ; Adam, Awadalkareem ; Jones, Madison C ; Lee, Christy ; Peng, Bi-Hung ; Xie, Xuping ; Wang, Tian ; Wang, Pengfei ; Harrington, Brinley R

QS-21, a key component of several licensed vaccines is facing limited supply, dose-limiting toxicity and other drawbacks which together limit its broader usage. Development of saponin alternatives to QS-21 that retain its desirable adjuvant activity without its drawbacks is in high need. Incorporating an amide side chain into the more sustainable Momordica saponins (MS) I and II led to the recent discovery of two semisynthetic immunostimulatory adjuvants VSA-1 and VSA-2. Here, we showed that SARS-CoV-2 receptor-binding protein (RBD) adjuvanted with VSA-2 induced high titers of SARS-CoV-2 specific humoral and T helper-1 prone immune responses in mice comparable to that triggered by QS-21-RBD vaccination. Vaccination with VSA-2-RBD provided strong protection against SARS-CoV-2 and variants infection and the virus-induced lung inflammation and pathology similarly as QS-21-RBD vaccination. Overall, our results suggest that VSA-2 adjuvant can potentially complement the clinically proven saponin adjuvant QS-21 in vaccines against infectious diseases.

20 Feb 2026·JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Approach to the Adult Patient with Chylomicronemia

Review

Author: Hegele, Robert A

Abstract:

Chylomicronemia, defined by fasting triglycerides ≥10 mmol/L (≥885 mg/dL), has diverse etiologies. When clinical features such as abdominal pain, lipemia retinalis, eruptive xanthomas, hepatosplenomegaly, pancreatitis, or visibly lipemic plasma accompany the biochemical disturbance, the condition is called chylomicronemia syndrome. Subtypes include rare monogenic familial chylomicronemia syndrome (FCS), the more common multifactorial chylomicronemia syndrome (MCS), autoimmune chylomicronemia, and lipodystrophy-associated chylomicronemia. Patients are at risk for acute pancreatitis and sometimes atherosclerotic cardiovascular disease. Accurate diagnosis includes medical history, physical exam, laboratory testing (including plasma apolipoprotein B and the ratio of triglyceride to total cholesterol), clinical scoring systems, as well as selective use of genetic testing when FCS is suspected. In adults, the overwhelming majority of patients with chylomicronemia have MCS and not FCS. Treatment centers on dietary fat restriction, total alcohol avoidance, management of secondary factors, and traditional triglyceride-lowering therapies such as fibrates and omega-3 fatty acids. Acute pancreatitis management requires stabilization, analgesia, supportive care, and preventive management of hypertriglyceridemia. Emerging RNA-based therapies targeting apolipoprotein C-III (eg, volanesorsen, olezarsen, and plozasiran) offer transformative potential for FCS and for some refractory patients with other chylomicronemia subtypes. A multidisciplinary approach—integrating clinical, biochemical, and genetic assessment—guides therapy and reduces pancreatitis risk.

107

News (Medical) associated with Plozasiran

30 Mar 2026

·www.biospace.com

Arrowhead Pharmaceuticals Presents New Long-Term Efficacy and Safety Data for Plozasiran Across a Spectrum of Hypertriglyceridemia at the American College of Cardiology’s 75th Annual Scientific Session and Expo

– Patients with severe hypertriglyceridemia (sHTG) achieved an 83% median reduction in triglycerides (TG), with 96% of patients achieving TG levels below 500 mg/dL, a threshold associated with increased risk of acute pancreatitis – No adjudicated acute pancreatitis events occurred in any patient receiving plozasiran during the 2-year Phase 2b Open-Label Expansion (OLE) Study – Favorable and durable improvements in atherogenic lipoproteins, including remnant cholesterol, non-HDL cholesterol, and ApoB, were observed, with a safety pro with earlier trials PASADENA, Calif.--(BUSINESS WIRE)-- $arwr -- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced long-term efficacy and safety data from a two-year long open-label extension (OLE) study of investigational plozasiran supporting its potential as therapeutic solution for a diverse spectrum of patients with hypertriglyceridemia (HTG). The data were presented by Dr. Christie M. Ballantyne, MD, professor at Baylor College of Medicine, member of the Texas Heart Institute at Baylor, and Principal Investigator of the 2-year OLE study, during an oral presentation at the American College of Cardiology 75 th Annual Scientific Session & Expo (ACC.26) in New Orleans and published in the American Journal of Preventive Cardiology. “With a new therapy, it is always important to see longer term data to better understand both efficacy and safety. In a long-term open-label extension study, plozasiran treatment was associated with dramatic and meaningful reductions in TGs. In addition, the lack of any acute pancreatitis events during two years of treatment in a patient population that includes those at high risk was particularly reassuring,” said Dr. Ballantyne. “These data reinforce the importance of APOC3 silencing as a central mechanism in promoting triglyceride clearance. The study provides promising evidence that plozasiran treatment may lead to durable, sustained reductions in triglycerides and atherogenic lipoproteins across the hypertriglyceridemia spectrum.” Plozasiran is designed to reduce the hepatic production of apolipoprotein C-III (APOC3) through targeted RNA interference. APOC3 is a key regulator of triglyceride metabolism that inhibits both lipoprotein lipase (LPL)-dependent and LPL-independent pathways involved in triglyceride catabolism and clearance, leading to elevated triglyceride levels. Individuals with genetic loss-of-function variants in APOC3 typically have markedly lower triglyceride levels and a reduced risk of atherosclerotic cardiovascular disease. The ACC.26 presentation builds on positive findings from two Phase 2b double-blind, placebo-controlled studies of plozasiran: SHASTA-2, conducted in adults with severe hypertriglyceridemia (sHTG), and MUIR, which enrolled patients with hypertriglyceridemia, both of which demonstrated short-term efficacy and safety. With the same patient populations, the company initiated an extension phase in which both groups received plozasiran 25 mg quarterly via subcutaneous injections. During the two-year OLE, patients saw median reductions in their triglycerides by -83% in sHTG patients from SHASTA-2 and -67% in HTG patients from MUIR with favorable reductions in remnant cholesterol and non-HDL-cholesterol. In both studies, the majority of patients achieved TG levels below thresholds for acute pancreatitis (AP) risk or below normal thresholds. 96% of sHTG patients achieved TGs below 500 mg/dL and 63% achieved TGs below 150 mg/dL, a threshold associated with increased risk of ASCVD. 93% of HTG patients achieved TGs below 150 mg/dL. These findings support the potential of plozasiran as a promising approach for managing patients with moderate to severe HTG phenotypes who are at risk of AP and potentially other cardiometabolic comorbidities. Plozasiran demonstrated a consistent long-term safety and tolerability pro both sHTG and HTG patient populations, with stable glycemic parameters, no clinically meaningful differences in routine clinical laboratory measurements, and no new safety signals. Common treatment-emergent adverse events included diabetes, COVID-19, upper respiratory tract infection, and back pain, consistent with prior studies; HbA1c levels remained stable. In SHASTA-2, mean placebo-adjusted liver fat content, as assessed by MRI-PDFF, did not change significantly over time with 25 mg plozasiran treatment. “We’re pleased with the consistent, positive clinical results we’re seeing with plozasiran across the spectrum of hypertriglyceridemias and we’re excited about what this could mean for patients,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. “In the four months since its FDA approval for treating FCS, plozasiran is already making a meaningful difference for the FCS community, and we believe it has the potential to become an important therapy for people living with severe hypertriglyceridemia as well. These advances, enabled by our innovative TRiM™ platform, reflect our commitment to developing targeted RNAi therapeutics to silence genes associated with cardiometabolic disorders as well as serious diseases in numerous tissue types throughout the body.” Arrowhead is on schedule to complete the SHASTA-3, SHASTA-4, and MUIR-3 Phase 3 clinical studies, the company’s global Phase 3 clinical studies designed to support regulatory submissions for marketing approval of plozasiran for the treatment of severe hypertriglyceridemia, in mid-2026 and intends to submit a supplemental New Drug Application (sNDA) to the U.S. FDA by year-end 2026. Arrowhead also plans to seek regulatory approval with additional global regulatory authorities thereafter. The ACC.26 presentation can be accessed on the Events and Presentations page under the Investors section of the Arrowhead website and the data have also been published in the American Journal of Preventive Cardiology. 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 FCS Familial chylomicronemia syndrome (FCS) is a severe and rare disease leading to extremely high triglyceride (TG) levels, typically over 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 Plozasiran Plozasiran is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (apoC-III) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. ApoC-III increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. The goal of treatment with plozasiran is to reduce the level of apoC-III, thereby reducing triglycerides and restoring lipids to more normal levels. Plozasiran is being investigated in the SHASTA-3, SHASTA-4, and SHASTA-5 Phase 3 studies in patients with severe hypertriglyceridemia and the MUIR Phase 3 study in patients with mixed hyperlipidemia. About REDEMPLO ® (plozasiran) REDEMPLO (plozasiran) is approved by the U.S. Food and Drug Administration, Health Canada, and the Chinese National Medical Products Administration as an adjunct to diet to reduce triglycerides for adults with Familial Chylomicronemia Syndrome (FCS). REDEMPLO is an siRNA therapeutic designed to suppress the production of apoC-III, a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting apoC-III with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is the first and only siRNA FDA-approved treatment studied in both genetically confirmed and clinically diagnosed patients living with FCS. 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 , 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 . 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, 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; 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 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 assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc. Contacts 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: Jacqueline Wickwire 617-901-2854 Jacqueline.wickwire@havasred.com

Clinical ResultPhase 3Drug ApprovalPhase 2

18 Feb 2026

·www.biospace.com

Ionis Paves Its Own Path as Initial Tryngolza Launch Defies Expectations

iStock, shinpanu thamvisead Long an R&D company that partnered off assets, RNAi biotech Ionis Pharmaceuticals shifted in 2025 to bring two medicines to market alone. Analysts are already impressed—and there’s more to come in 2026. Investors were not exactly expecting Ionis’ GSK-partnered antisense oligonucleotide to achieve a functional cure in chronic hepatitis B. CEO Brett Monia, however, was not surprised. “I think we made our point. I don’t think it’s seeping in well enough yet, but it will,” Monia told BioSpace on the sidelines of the J.P. Morgan Healthcare Conference in January. Kicking off a catalyst-rich year for Ionis, partner GSK on January 7 revealed that bepirovirsen achieved a “statistically significant and clinically meaningful functional cure rate” in a Phase 3 trial. GSK did not reveal the specific figures, only saying that the trial met its primary endpoint. The Big Pharma partner will release more details from the trial at the European Association for the Study of the Liver meeting this spring, while a new drug application will be filed in the first quarter. Monia explained that the trial measured a functional cure rate of about 15–20%, while existing treatments achieve maybe 1–3%. And last month’s bepirovirsen readout just scratches the surface of what Ionis has in store for 2026. Last year, the RNA interference biotech launched two products independently—Dawnzera for the prevention of hereditary angioedema attacks and Tryngolza for familial chylomicronemia syndrome (FCS). “Ionis has been historically an R&D organization. Now we built the commercial capabilities to support the launches that occurred,” Monia said. Cardiovascular disease Ionis’ Tryngolza ‘Game-Changing’ in Lowering Triglycerides, Cementing Case for FDA Filing Ionis is planning a supplemental submission by the end of the year to expand Tryngolza into severe hypertriglyceridemia. If granted, William Blair expects the antisense drug to be “transformational” for this indication. November 10, 2025 · 2 min read · Tristan Manalac Read More Tryngolza took home approximately $102 million in 2025 while Ionis has yet to report sales for Dawnzera. But the launch of the former has impressed analysts so far. William Blair in January predicted that Tryngolza could reach $2 billion in sales in 2034. Ionis also had two successful Phase 3 readouts last year, including one for Tryngolza in severe hypertriglyceridemia (sHTG). Monia said the data showed “remarkable reductions” in triglycerides. The trial, which could help support bringing the treatment into a larger disease population, helped underpin an FDA application at the end of the year. The initial launch success in FCS is “a sign of the company’s impressive salesforce in cardiometabolic disease and auspicious for a potential launch of Tryngolza in sHTG,” William Blair wrote in a January note. While Ionis has now launched medicines independently, the potential sHTG indication would be the first time the company has forayed into more common diseases. In this space, Ionis is up against Arrowhead Pharmaceuticals, another mid-sized biotech that is rising as an independent commercial company. Plozasiran was approved for FCS in November 2025 and a Phase 3 readout for sHTG is expected in the third quarter. With the FCS approval, Monia said Ionis priced the drug for a rare indication with a cost of $595,000. The CEO promised the price would come down as it moves into a more common indication. “Let’s face the facts: the FCS market—we pretty much created it, and we’re doing extremely well. There’s not much left for a competitor to come in,” Monia said. The specific sHTG price has not been announced, although William Blair put the wholesale acquisition cost between $10,000 to $20,000 per year—much lower than Arrowhead’s planned $60,000 cost for the rival medicine. Ionis also has zilganersen, which showed a disease-modifying benefit in a Phase 3 trial of patients with the rare neurodegenerative condition Alexander disease last year. Monia said that children with the devastating disorder typically do not survive past age four. Ionis plans to approval application for zilganersen this quarter. Elsewhere, Ionis’ partnered pipeline is advancing as well, with four Phase 3 readouts expected this year. In addition to GSK, the company has partnerships with AstraZeneca, Roche and Novartis, as well as a handful of smaller companies. “It seems like [2025] was the greatest year in our history, based on all of the achievements we made and how we transformed the company,” Monia said. “And I expect to make the same claim in December of this year.” Ionis will report earnings and provide company highlights on February 25. .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to BioPharm Executive! 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Phase 3Clinical ResultAcquisitionDrug ApprovalOligonucleotide

10 Feb 2026

·www.prnewswire.com

Vanscoy Rare Pharmacy Selected as Exclusive Distribution Pharmacy for Redemplo® (plozasiran) for adults with Familial Chylomicronemia Syndrome (FCS)

PITTSBURGH, Feb. 10, 2026 /PRNewswire/ -- Vanscoy Rare Pharmacy, a leading specialty pharmacy focused on supporting people living with rare conditions, announced today that it has been selected as the exclusive specialty pharmacy provider for Redemplo® (plozasiran), a small interfering RNA (siRNA) medicine, indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). FCS is a severe, rare disease, with an estimated 6,500 people in the U.S. living with genetic or clinical FCS, characterized by triglyceride levels that can be 10 to 100 times higher than normal leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis. With the FDA approval of Redemplo, patients will have access to a targeted treatment option designed to address the underlying cause of FCS. Through this agreement, Vanscoy Rare Pharmacy will provide patients with comprehensive support at each stage of their treatment journey with Redemplo. "We are honored to be selected as the exclusive Specialty Pharmacy dispensing Redemplo and to play a role in improving access for individuals and families affected by FCS," said Dr. Gordon Vanscoy, CEO, Vanscoy Rare Pharmacy. "Our team is deeply committed to ensuring that patients receive optimal support throughout their treatment journey — from initiation to long-term management." "Arrowhead Pharmaceuticals is proud to collaborate with Vanscoy Rare Pharmacy, whose experience in rare metabolic disorders and patient-centered approach align with our mission to treat diseases with significant unmet needs," said Chris Anzalone, CEO, Arrowhead Pharmaceuticals. INDICATION REDEMPLO® (plozasiran) is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS: None ADVERSE REACTIONS: Most common adverse reactions in REDEMPLO treated patients (incidence ≥10% of patient treated with REDEMPLO and >5% more frequently than with placebo) are hyperglycemia, headache, nausea, and injection site reaction. Prescribing Information Redemplohcp.com About Familial Chylomicronemia Syndrome (FCS) Familial chylomicronemia syndrome (FCS) is a severe and rare disease leading to extremely high triglyceride (TG) levels, typically over 880 mg/dl. Such severe elevations can lead to various 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 Vanscoy Rare Pharmacy Vanscoy Rare Pharmacy is a triple accredited, national specialty pharmacy dedicated to providing high-touch care for patients with rare conditions. Through customized clinical programs, expert pharmacist support, and collaborative partnerships with manufacturers and providers, Vanscoy Rare Pharmacy ensures optimal access and outcomes for the patients it serves. Learn more at 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, visit SOURCE Vanscoy Rare Pharmacy 21% more press release views with Request a Demo

Drug ApprovalLicense out/in

100 Deals associated with Plozasiran

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Familial chylomicronaemia syndrome	United States	Arrowhead Pharmaceuticals, Inc.	18 Nov 2025

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hypertriglyceridemia	NDA/BLA	Canada	Arrowhead Pharmaceuticals, Inc.	01 Jul 2025
Hyperlipoproteinemia Type I	Phase 3	United States	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	Japan	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	Argentina	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	Australia	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	Austria	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	Belgium	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	Canada	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	Croatia	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021
Hyperlipoproteinemia Type I	Phase 3	France	Arrowhead Pharmaceuticals, Inc.	14 Dec 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04720534 (SHASTA-2, CTgov)	Phase 2	Hypertriglyceridemia	229	Placebo (Placebo, Day 1 and Week 12)	jjsgdangim(rpyxrcieac) = nmibrxxlhc scsnbiqmkl (hjvfqjmssd, 5.27) View more	-	24 Mar 2026
				ARO-APOC3 (ARO-APOC3 10 mg)	jjsgdangim(rpyxrcieac) = worgdnricg scsnbiqmkl (hjvfqjmssd, 5.61) View more
NCT05902598 (CTgov)	Phase 3	Familial chylomicronaemia syndrome	37	Plozasiran (Plozasiran 25 mg)	xkwwkwxemc(gjryxgzleo) = obgfqltfzs mjbwboywaz (avhykkanjp, gtevjafoff - lxcnnkfted) View more	-	27 Feb 2026
				Plozasiran (Plozasiran 50 mg)	xkwwkwxemc(gjryxgzleo) = rztkdpcfna mjbwboywaz (avhykkanjp, cpthdieqyv - pfnwcyznzl) View more
NCT04998201 (MUIR, CTgov)	Phase 2	Dyslipidemias	353	Placebo (Placebo)	cprqiyzgnl(ruujtqeugv) = lkobunmjba oznjkfezwt (ydzlkdeujq, 3.07) View more	-	24 Feb 2026
				ARO-APOC3 (ARO-APOC3 10 mg Q12W)	cprqiyzgnl(ruujtqeugv) = lgkybqngwd oznjkfezwt (ydzlkdeujq, 3.54) View more
NCT05089084 (PALISADE, CTgov)	Phase 3	Hyperlipoproteinemia Type I	75	Plozasiran (ARO-APOC3 (Plozasiran) 25 mg)	utlpaquqlt(utrvahwfze) = liebsfxfjz gwjyksvvhz (vyrhfiqbxy, ryxlhaultg - ceyxolgvsg) View more	-	04 Feb 2026
				Plozasiran (ARO-APOC3 (Plozasiran) 50 mg)	utlpaquqlt(utrvahwfze) = faipzuawdr gwjyksvvhz (vyrhfiqbxy, jjvzkuijtc - heavwlapvc) View more
NCT05089084 (FDA_CDER) Manual	Phase 3	Familial chylomicronaemia syndrome	51	REDEMPLO 25 mg	sasadmftkd(cotpamgghv) = uuprmmiikj lguridmvjp (qlpuirdjbp ) View more	Positive	18 Nov 2025
				Placebo (pooled)	sasadmftkd(cotpamgghv) = mrrbvbrrov lguridmvjp (qlpuirdjbp ) View more
NCT05757596 (Pubmed \| Cardiovasc Diabetol) Manual	Phase 1	-	24	Plozasiran 25 mg	etgiafhbpi(pncewrcymg) = ishqtdsvog thaucrsgzs (irinculdur ) View more	Positive	15 Oct 2025
				Plozasiran 50 mg	etgiafhbpi(pncewrcymg) = atyvxuceiz thaucrsgzs (irinculdur ) View more
NCT05089084 (PALISADE, Corporate Publications \| ESC2025) Manual	Phase 3	Familial chylomicronaemia syndrome	-	Plozasiran (switched fr placebo)	uvkzkkzvzb(yqqmmsepuu) = afbaierxtb mralerueyj (jtqtdnrtqb ) View more	Positive	30 Aug 2025
				Plozasiran (continuous treated)	uvkzkkzvzb(yqqmmsepuu) = qkglljpchk mralerueyj (jtqtdnrtqb ) View more
NCT05902598 \| CTR20231418 (PRNewswire) Manual	Phase 3	Familial chylomicronaemia syndrome	37	VSA001 25 mg	mpbqsvdcme(bmsnqnehde) = wpxrismewh tfdrbehafh (eprwjucvkm ) Met View more	Positive	17 Mar 2025
				VSA001 50 mg	mpbqsvdcme(bmsnqnehde) = zbkzrnsxsp tfdrbehafh (eprwjucvkm ) Met View more
NCT04998201 \| NCT04720534 (MUIR, Company_Website) Manual	Phase 2	Hypertriglyceridemia \| Hyperlipidemia, Familial Combined	418	Plozasiran (MUIR)	fypknlulct(ffsmgjiigi) = nngfafiovd eikcairepa (alhuvizqlx )	Positive	18 Nov 2024
				Plozasiran (SHASTA-2)	fypknlulct(ffsmgjiigi) = mifoaeahfl eikcairepa (alhuvizqlx )
NCT05089084 (PALISADE, Company_Website) Manual	Phase 3	Familial chylomicronaemia syndrome	75	Plozasiran 25 mg	ahhnjcwull(wynyhuzhve) = hidsjjyjfl lskicxnvln (hnxegjrhvu ) View more	Positive	18 Nov 2024
				Plozasiran 50 mg	ahhnjcwull(wynyhuzhve) = pbmetqiseu lskicxnvln (hnxegjrhvu ) View more

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