ARO-APOC3
- Generic Name
- ARO-APOC3
- Drug Type
- Biotech
- Background
ARO-APOC3 is an investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3). It received an Orphan Drug designation by the FDA for the treatment of familial chylomicronemia syndrome.
Plozasiran Shows Promise in Reducing Cholesterol and Triglycerides in Mixed Hyperlipidemia Patients
• Plozasiran, an investigational hepatocyte-targeted APOC3 small interfering RNA developed by Arrowhead Pharmaceuticals, demonstrated significant reductions in triglyceride levels and improved lipid profiles in patients with mixed hyperlipidemia.
• Multiple clinical trials including MUIR, SHASTA-2, and PALISADE have shown plozasiran's effectiveness in reducing harmful blood fats across various patient populations, addressing unmet needs for those who cannot tolerate statins.
• Based on positive clinical results, Arrowhead Pharmaceuticals plans to advance plozasiran into the phase 3 CAPITAN cardiovascular outcomes trial, potentially offering a novel treatment option for patients with residual atherosclerotic cardiovascular disease risk.
Arrowhead's Plozasiran for Familial Chylomicronemia Syndrome Fast-Tracked for EU Approval
• Arrowhead Pharmaceuticals' plozasiran, a novel treatment for familial chylomicronemia syndrome (FCS), has been filed for review by the European Medicines Agency and is being fast-tracked for potential EU marketing approval.
• The accelerated review process highlights the significant unmet medical need for effective therapies targeting FCS, a rare genetic disorder characterized by severe hypertriglyceridemia and risk of life-threatening pancreatitis.
• Plozasiran joins a cohort of 15 new drug applications under EMA review, including Novo Nordisk's semaglutide for the treatment of cirrhotic metabolic dysfunction-associated steatohepatitis (MASH).
Oncotelic Therapeutics Completes Phase 1 Trial of OT-101/IL-2 Combination for Advanced Solid Tumors
• Oncotelic Therapeutics has successfully completed a Phase 1 trial evaluating OT-101, an antisense therapeutic targeting TGFβ2, in combination with IL-2 for advanced or metastatic solid tumors.
• The combination demonstrated a tolerable safety profile with no unexpected safety signals, positioning the company to advance into further clinical studies exploring synergies with checkpoint inhibitors.
• CEO Dr. Vuong Trieu presented findings at the SWCR 2025 Conference, highlighting TGFβ2's central role in immune suppression and OT-101's ongoing development in pancreatic cancer, gliomas, and combination immunotherapy regimens.
FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months
• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration.
• Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients.
• Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.
2024 Advances in Lipid Management: A Year of Progress and Setbacks
2024 marked significant progress in lipid management, with notable advancements in the treatment of familial chylomicronemia syndrome (FCS) and lipoprotein(a) (Lp(a)) management. However, the year also saw challenges to the 'HDL hypothesis', highlighting the complexity of lipid-related cardiovascular disease prevention. Key developments included FDA approvals of novel therapies and promising results from clinical trials targeting triglycerides and Lp(a).
Arrowhead Pharmaceuticals CFO Kenneth Myszkowski Sells $893,095 in Stock
Kenneth Myszkowski, CFO of Arrowhead Pharmaceuticals, has sold $893,095 worth of company stock, retaining 455,433 shares. The sales, part of a pre-established trading plan, were made to cover tax obligations. Arrowhead is advancing in pharmaceutical developments and strategic partnerships, with significant clinical trials underway.
Ionis' Tryngolza Approved for FCS as Arrowhead's Plozasiran Awaits FDA Review
• The FDA has approved Ionis Pharmaceuticals' Tryngolza (olezarsen) as the first treatment for adults with familial chylomicronemia syndrome (FCS).
• Tryngolza, an RNA-targeted therapy, significantly reduces triglyceride levels and the risk of acute pancreatitis in FCS patients when used with a low-fat diet.
• Arrowhead Pharmaceuticals' plozasiran, another RNA interference therapeutic for FCS, has been accepted for FDA review, with a decision expected by November 2025.
• Clinical trials showed Tryngolza achieved a 57% placebo-adjusted mean reduction in triglycerides at 12 months, while plozasiran demonstrated an 80% median reduction.
Arrowhead Pharmaceuticals' FDA Acceptance of Plozasiran NDA Marks Milestone in RNAi Therapeutics
• Arrowhead Pharmaceuticals announced FDA acceptance of its New Drug Application for plozasiran to treat familial chylomicronemia syndrome (FCS), with a PDUFA date of November 18, 2025.
• The company secured significant funding through strategic deals, including an $825 million agreement with Sarepta Therapeutics, extending its cash runway into 2028 to support pipeline development.
• Arrowhead is advancing multiple clinical programs, including two obesity candidates (ARO-INHBE and ARO-ALK7) and expanding into CNS applications with its proprietary TRiM platform technology.
Sarepta Therapeutics and Arrowhead Pharmaceuticals Announce RNAi Collaboration to Treat Rare Genetic Diseases
• Sarepta Therapeutics and Arrowhead Pharmaceuticals have entered into a global licensing and collaboration agreement to develop RNAi-based treatments for rare genetic diseases.
• Sarepta will gain rights to several of Arrowhead’s clinical-stage programs, including treatments for facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, idiopathic pulmonary fibrosis, and spinocerebellar ataxia 2.
• Arrowhead will receive an upfront payment of $500 million, a $325 million equity investment, and is eligible for up to $10 billion in milestone payments and royalties.
• The collaboration diversifies Sarepta’s portfolio, complementing its gene therapy and gene editing efforts with Arrowhead's RNAi technology, while extending Arrowhead's cash runway into 2028.
Arrowhead Submits FDA Application for Plozasiran in Familial Chylomicronemia Syndrome
• Arrowhead Pharmaceuticals has submitted a new drug application to the FDA for plozasiran for the treatment of familial chylomicronemia syndrome (FCS).
• Plozasiran demonstrated sustained reductions in apolipoprotein C-III and triglycerides in the PALISADE Phase 3 study and open-label extensions.
• The drug was generally well-tolerated, with the most frequently reported adverse events being abdominal pain, COVID-19, nasopharyngitis, and nausea.
• The FDA previously granted breakthrough therapy designation to plozasiran for FCS in September 2024, expediting its potential approval.
Plozasiran Shows Promise in Reducing Triglycerides in Phase 3 and Extension Studies
• Plozasiran significantly reduced triglycerides (TG) in patients with familial chylomicronemia syndrome (FCS), regardless of genetic confirmation, according to Phase 3 PALISADE study data.
• In the PALISADE study, plozasiran achieved approximately -80% reduction in triglycerides and greater than -90% reduction in apolipoprotein C-III (APOC3) at the 25 mg dose.
• Open-label extension (OLE) data from MUIR and SHASTA-2 studies showed sustained TG reductions up to -73% and -86%, respectively, with favorable changes in other lipid parameters.
• Plozasiran was generally well-tolerated, with common adverse events including abdominal pain, COVID-19, nasopharyngitis, and headache.
Arrowhead Submits Plozasiran NDA to FDA for Familial Chylomicronemia Syndrome
• Arrowhead Pharmaceuticals has submitted an NDA to the FDA for plozasiran, targeting familial chylomicronemia syndrome (FCS), a rare genetic disease with no approved treatments.
• The NDA is based on positive Phase 3 PALISADE study results, which showed significant reductions in triglycerides, apolipoprotein C-III, and acute pancreatitis incidence.
• Plozasiran demonstrated an 80% median reduction in triglycerides and an 83% reduction in acute pancreatitis risk compared to placebo in the PALISADE trial.
• Arrowhead plans to submit applications for plozasiran approval to additional regulatory authorities in 2025, expanding its reach to patients beyond the U.S.
Plozasiran Shows Promise in Reducing Triglycerides in Familial Chylomicronemia Syndrome and Severe Hypertriglyceridemia
• Phase 3 PALISADE study reveals plozasiran significantly reduces triglycerides in patients with Familial Chylomicronemia Syndrome (FCS), regardless of genetic confirmation.
• Open-label extension of Phase 2 MUIR and SHASTA-2 studies demonstrates sustained triglyceride reductions with plozasiran in severe hypertriglyceridemia patients over 15 months.
• Plozasiran treatment led to favorable changes in multiple atherogenic lipoproteins and did not worsen HbA1c levels, suggesting a favorable long-term safety profile.
• Arrowhead Pharmaceuticals is advancing plozasiran as a potential therapy for FCS, severe hypertriglyceridemia, and mixed hyperlipidemia based on these promising results.
Arrowhead Submits FDA Application for Plozasiran in Familial Chylomicronemia Syndrome
• Arrowhead Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for plozasiran, targeting familial chylomicronemia syndrome (FCS).
• The NDA is based on Phase 3 PALISADE study results, which demonstrated significant triglyceride reductions and decreased acute pancreatitis risk.
• Plozasiran aims to address the unmet need in FCS, a severe genetic disease with currently no FDA-approved treatments.
• The company plans to submit further applications to other regulatory authorities in 2025, expanding plozasiran's potential availability.
Arrowhead Seeks FDA Approval for Plozasiran in Familial Chylomicronemia Syndrome
• Arrowhead Pharmaceuticals has submitted an NDA to the FDA for plozasiran, a potential treatment for familial chylomicronemia syndrome (FCS).
• The application is based on Phase 3 PALISADE study results, which showed significant triglyceride reduction and decreased acute pancreatitis risk.
• Plozasiran, utilizing Arrowhead's TRiM™ platform, targets apolipoprotein C-III to regulate triglyceride metabolism in FCS patients.
• The FDA has granted plozasiran Breakthrough Therapy, Orphan Drug, and Fast Track Designations, highlighting its potential to address unmet needs.
Arrowhead Submits FDA Application for Plozasiran in Familial Chylomicronemia Syndrome
• Arrowhead Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for plozasiran, a potential treatment for familial chylomicronemia syndrome (FCS).
• The application is based on Phase 3 PALISADE study results, which demonstrated an 80% median reduction in triglycerides with the 25 mg dose.
• Plozasiran also significantly reduced the risk of acute pancreatitis by 83% compared to placebo in pooled plozasiran groups in the PALISADE trial.
• If approved, plozasiran would be the first FDA-approved therapy for FCS, addressing a significant unmet need for this rare genetic disease.
Arrowhead Pharma Focuses on Familial Chylomicronemia Syndrome with Awareness Campaign and Plozasiran Development
• Arrowhead Pharmaceuticals has initiated the "We'll Get There Soon" campaign to raise awareness for Familial Chylomicronemia Syndrome (FCS), a rare and potentially life-threatening disease.
• The FDA granted Breakthrough Therapy designation to Arrowhead's Plozasiran, a treatment for FCS, highlighting its potential to address the unmet needs of FCS patients.
• Arrowhead secured $500 million in strategic financing to support the commercial launch of Plozasiran, anticipated in 2025, and to fund further pipeline growth and innovation.
• The company aims to amplify the voices of those affected by FCS, with patient insights informing their efforts and driving hope within the rare disease community.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
Regulatory Updates in Cardiometabolic Health: Hypertension, Lipids, and Diabetes Therapies
• The FDA approved an expanded indication for subcutaneous furosemide injection for heart failure patients, offering a convenient self-administration option.
• Aprocitentan received FDA approval for hypertension, providing a new treatment option for adults with uncontrolled blood pressure despite other medications.
• The FDA cleared a continuous glucose monitoring (CGM) system featuring an implantable sensor lasting up to one year for diabetes management.
FDA Approves Hearing Aid Software for AirPods, Tremfya for Ulcerative Colitis, and Grants Breakthrough Therapy Designation to Plozasiran
• The FDA has approved over-the-counter hearing aid software for certain Apple AirPods Pro models, demonstrated to provide comparable performance to professionally fitted hearing aids.
• Guselkumab (Tremfya) has received FDA approval for treating moderate to severe active ulcerative colitis in adults, based on Phase 2b/3 QUASAR study results showing significant clinical remission.
• Plozasiran, an investigational RNAi therapy, was granted Breakthrough Therapy designation by the FDA for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS).
Drug Development Updates
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