Latest news with #HomozygousFamilialHypercholesterolemia
Business Wire
08-07-2025
- Business
- Business Wire
Arrowhead Pharmaceuticals Initiates Phase 3 YOSEMITE Study of Investigational Zodasiran for the Treatment of Homozygous Familial Hypercholesterolemia
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subject in the YOSEMITE Phase 3 clinical trial of zodasiran, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease. Zodasiran is the fourth investigational RNAi-based candidate developed by Arrowhead to reach late-stage pivotal studies, after investigational drugs plozasiran, fazirsiran (licensed to Takeda) and olpasiran (licensed to Amgen). As an RNAi-based therapeutic targeting ANGPTL3, investigational zodasiran has the potential to treat HoFH in a fundamentally different manner from traditional LDL-C–lowering therapies. Share 'Patients living with HoFH are difficult to adequately treat and have a very high risk of developing atherosclerotic cardiovascular disease due to severely elevated LDL-C, often exceeding 500 mg/dL. As an RNAi-based therapeutic targeting ANGPTL3, investigational zodasiran has the potential to treat HoFH in a fundamentally different manner from traditional LDL-C–lowering therapies,' said James Hamilton, M.D., Chief Medical Officer and head of R&D at Arrowhead. 'In Phase 2 clinical studies, patients with HoFH receiving zodasiran achieved reductions from baseline in LDL-C, ApoB, non-HDL-C, and triglycerides, supporting its potential therapeutic role for the treatment of HoFH patients.' About Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia (HoFH) is an ultra-rare treatment‐resistant genetic condition characterized by elevated low density lipoprotein cholesterol (LDL-C) and early-onset cardiovascular disease. Most cases of HoFH are due to mutations in the low-density lipoprotein receptor gene (LDLR) coding for the LDL receptor (LDLR). Thus, HoFH represents a unique case where LDL-C lowering therapies not requiring functional LDL receptors may have benefit. If left untreated, individuals with HoFH can have median LDL-C levels above 500 mg/dL (13 mmol/L), leading to early clinical manifestations of coronary artery disease 1. Patients with HoFH may also have cholesterol deposits under the skin (xanthomas), around the eyes (xanthelasmas), or around the cornea (corneal arcus), but physical signs are not always present, particularly in children. HoFH remains challenging to treat and currently only patients with the more severe HoFH phenotypes get diagnosed and treated early. The estimated prevalence of HoFH globally is between 1:360,000 and 1:250,000 1. About YOSEMITE Phase 3 Study YOSEMITE (NCT07037771) is a Phase 3 multicenter, randomized, placebo-controlled study to evaluate the efficacy and safety of zodasiran in adolescent and adult patients with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) on maximally tolerated lipid lowering therapy. Approximately 60 subjects over the age of 12 will be randomized (2:1) to receive 4 doses (once every 3 months) of 200 mg zodasiran or placebo. The primary endpoint is the percent change from baseline to month 12 in fasting LDL-C. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension. About Zodasiran Zodasiran, previously called ARO-ANG3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of angiopoietin-like protein (ANGPTL3), which is a hepatocyte expressed regulator of lipid and lipoprotein metabolism with multiple potential modes of action, including inhibition of lipoprotein lipase (LPL) and endothelial lipase (EL) 5,6. ANGPTL3 is an emerging therapeutic target with relevance to hypercholesterolemia, hypertriglyceridemia, and mixed hyperlipidemia. Genetic studies suggest that individuals with ANGPTL3 loss-of-function variants have enhanced lipoprotein lipase and endothelial lipase activity, resulting in lower levels of atherogenic lipoproteins and a reduced risk of ASCVD 2-4. In prior clinical studies, investigational zodasiran was associated with dose-dependent reductions in triglycerides, triglyceride rich lipoprotein remnants, and total atherogenic lipoproteins, including LDL-C, in patients with homozygous (HoFH) and heterozygous (HeFH) familial hypercholesterolemia and mixed hyperlipidemia. Zodasiran also showed a favorable safety profile. In the Phase 2 GATEWAY study in patients with HoFH, there were no drug discontinuations, drug-related serious adverse events, or deaths. The most frequent adverse events were COVID-19, nasopharyngitis, upper respiratory tract infection, and dizziness. About Arrowhead Pharmaceuticals, Inc. Arrowhead Pharmaceuticals, Inc. develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit 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,' 'might,' 'will,' 'expect,' 'believe,' 'anticipate,' 'goal,' 'endeavor,' 'strive,' 'hope,' 'intend,' 'plan,' 'project,' 'could,' 'estimate,' 'potential,' '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 regulatory approval for and commercial launch of plozasiran; 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. ________________ 1. Cuchel, et al. Eur Heart J. 2023;44(25):2277-91 2. Dewey, et al. N Engl J Med. 2017;377 (3):211-21. 3. Minicocci, et al. J Lipid Res. 2013;54(12): 3481-90 4. Musunuru, et al. N Engl J Med. 2010; 363(23):2220-7 5. Adam, et al. J Lipid Res. 2020;61(9): 1271-86. 6. Rosenson. J Lipid Res. 2021:62:100060.
Globe and Mail
12-06-2025
- Business
- Globe and Mail
Homozygous Familial Hypercholesterolemia Market to Reach New Heights in Growth by 2034, DelveInsight Predicts
Key Homozygous Familial Hypercholesterolemia Companies in the market include - Arrowhead Pharmaceutical, Novartis, Alnylam Pharmaceutical, LIB Therapeutics, Amryt Pharma, Novartis, Ultragenyx Pharmaceutical, Arrowhead Pharmaceuticals, Sanofi, Cerenis Therapeutics, Aegerion Pharmaceuticals, The Medicines Company, Akeso, Aegerion Pharmaceuticals, and others. DelveInsight's 'Homozygous Familial Hypercholesterolemia Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Homozygous Familial Hypercholesterolemia, historical and forecasted epidemiology as well as the Homozygous Familial Hypercholesterolemia market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan. To Know in detail about the Homozygous Familial Hypercholesterolemia market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Homozygous Familial Hypercholesterolemia Market Forecast Some of the key facts of the Homozygous Familial Hypercholesterolemia Market Report: The Homozygous Familial Hypercholesterolemia market size was valued approximately USD 108 million in 2022 and is anticipated to grow with a significant CAGR during the study period (2020-2034) In March 2025, Esperion (NASDAQ: ESPR) has announced that, following discussions with the U.S. Food and Drug Administration (FDA), it has received regulatory alignment to proceed with Phase 3 clinical trials of bempedoic acid—both as a standalone therapy and in combination with ezetimibe—in pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH). The company intends to launch these trials later this year. Notably, the FDA has previously granted orphan drug designation to bempedoic acid for the treatment of HoFH. In January 2025, LIB Therapeutics Inc. (LIB), a privately held, late-stage biopharmaceutical company developing Lerodalcibep—a next-generation PCSK9 inhibitor—announced that results from its Phase 3 LIBerate-HoFH trial have been published in The Lancet Diabetes & Endocrinology (Raal, F.J. et al., 2025). The study evaluated Lerodalcibep in a globally diverse population with homozygous familial hypercholesterolemia (HoFH). DelveInsight's 2022 analysis indicates that there were approximately 2,845 diagnosed prevalent cases of HoFH in the 7MM. This number is projected to rise significantly by 2034, with a notable CAGR throughout the study period (2020–2034). Estimates from DelveInsight's epidemiology model suggest that there were around 1,349 diagnosed prevalent cases of HoFH in the US in 2022. This number is projected to increase by 2034 over the study period (2020–2034). In the 7MM, the EU4 and the UK accounted for about 38% of the total diagnosed prevalent cases of HoFH in 2022, with expectations for this figure to rise further during the study period. According to DelveInsight's analysis, the United States alone represented approximately 47% of all diagnosed prevalent cases of HoFH among the 7MM in 2022. Key Homozygous Familial Hypercholesterolemia Companies: Arrowhead Pharmaceutical, Novartis, Alnylam Pharmaceutical, LIB Therapeutics, Amryt Pharma, Novartis, Ultragenyx Pharmaceutical, Arrowhead Pharmaceuticals, Sanofi, Cerenis Therapeutics, Aegerion Pharmaceuticals, The Medicines Company, Akeso, Aegerion Pharmaceuticals, and others Key Homozygous Familial Hypercholesterolemia Therapies: ARO-ANG3, LEQVIO (inclisiran/KJX839), Lerodalcibep (LIB003), Lomitapide, Inclisiran, Evinacumab, ARO-ANG 3 Injection, Alirocumab, CER-001, lomitapide, ALN-PCSSC, AK102, AEGR-733, and others The Homozygous Familial Hypercholesterolemia epidemiology based on mutation-specific cases analyzed that a mutation in the LDLR gene accounted for the highest diagnosed prevalent cases in the US The Homozygous Familial Hypercholesterolemia market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Homozygous Familial Hypercholesterolemia pipeline products will significantly revolutionize the Homozygous Familial Hypercholesterolemia market dynamics. Homozygous Familial Hypercholesterolemia Overview Homozygous Familial Hypercholesterolemia (HoFH) is a rare and severe genetic disorder characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) from birth. It is caused by mutations in both alleles of genes involved in the clearance of LDL-C from the bloodstream, typically the LDL receptor gene. This condition leads to early and aggressive atherosclerosis, increasing the risk of heart attacks, strokes, and other cardiovascular diseases at a young age. Get a Free sample for the Homozygous Familial Hypercholesterolemia Market Forecast, Size & Share Analysis Report: Homozygous Familial Hypercholesterolemia Epidemiology The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends. Homozygous Familial Hypercholesterolemia Epidemiology Segmentation: The Homozygous Familial Hypercholesterolemia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of HoFH in the 7MM Mutation-specific Diagnosed Prevalent Cases of HoFHin the 7MM Download the report to understand which factors are driving Homozygous Familial Hypercholesterolemia epidemiology trends @ Homozygous Familial Hypercholesterolemia Epidemiology Forecast Homozygous Familial Hypercholesterolemia Drugs Uptake and Pipeline Development Activities The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Homozygous Familial Hypercholesterolemia market or expected to get launched during the study period. The analysis covers Homozygous Familial Hypercholesterolemia market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share. The report also covers the Homozygous Familial Hypercholesterolemia Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies. Homozygous Familial Hypercholesterolemia Therapies and Key Companies Homozygous Familial Hypercholesterolemia Market Strengths The clarity in disease understanding and pathogenesis has driven the development of novel pharmacological options like PCSK9 inhibitors, ANGPTL3 inhibitors, and MTP inhibitor Updated international diagnostic and clinical guidelines for FH and HoFH enable evidence-based therapeutic approaches and screening strategies for early identification Advances in research have led to the discovery of novel molecules like siRNA and recombinant fusion protein that may offer novel options to lower LDL significantly Homozygous Familial Hypercholesterolemia Market Opportunities Preclinical studies have yielded gene therapy and CRISPR-based gene editing approaches; conducting further research and clinical trials may offer curative therapy. Current therapies do not impart sufficient cholesterol-lowering allowing pharma players to bring newer, more potent, and better LDL-C lowering therapies and prevent ASCVD in HoFH patients. Advancements in healthcare technologies and digital health solutions can potentially enhance early detection and personalized management of HoFH. Scope of the Homozygous Familial Hypercholesterolemia Market Report Study Period: 2020–2034 Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan] Key Homozygous Familial Hypercholesterolemia Companies: Arrowhead Pharmaceutical, Novartis, Alnylam Pharmaceutical, LIB Therapeutics, Amryt Pharma, Novartis, Ultragenyx Pharmaceutical, Arrowhead Pharmaceuticals, Sanofi, Cerenis Therapeutics, Aegerion Pharmaceuticals, The Medicines Company, Akeso, Aegerion Pharmaceuticals, and others Key Homozygous Familial Hypercholesterolemia Therapies: ARO-ANG3, LEQVIO (inclisiran/KJX839), Lerodalcibep (LIB003), Lomitapide, Inclisiran, Evinacumab, ARO-ANG 3 Injection, Alirocumab, CER-001, lomitapide, ALN-PCSSC, AK102, AEGR-733, and others Homozygous Familial Hypercholesterolemia Therapeutic Assessment: Homozygous Familial Hypercholesterolemia current marketed and Homozygous Familial Hypercholesterolemia emerging therapies Homozygous Familial Hypercholesterolemia Market Dynamics: Homozygous Familial Hypercholesterolemia market drivers and Homozygous Familial Hypercholesterolemia market barriers Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies Homozygous Familial Hypercholesterolemia Unmet Needs, KOL's views, Analyst's views, Homozygous Familial Hypercholesterolemia Market Access and Reimbursement Table of Contents 1. Homozygous Familial Hypercholesterolemia Market Report Introduction 2. Executive Summary for Homozygous Familial Hypercholesterolemia 3. SWOT analysis of Homozygous Familial Hypercholesterolemia 4. Homozygous Familial Hypercholesterolemia Patient Share (%) Overview at a Glance 5. Homozygous Familial Hypercholesterolemia Market Overview at a Glance 6. Homozygous Familial Hypercholesterolemia Disease Background and Overview 7. Homozygous Familial Hypercholesterolemia Epidemiology and Patient Population 8. Country-Specific Patient Population of Homozygous Familial Hypercholesterolemia 9. Homozygous Familial Hypercholesterolemia Current Treatment and Medical Practices 10. Homozygous Familial Hypercholesterolemia Unmet Needs 11. Homozygous Familial Hypercholesterolemia Emerging Therapies 12. Homozygous Familial Hypercholesterolemia Market Outlook 13. Country-Wise Homozygous Familial Hypercholesterolemia Market Analysis (2020–2034) 14. Homozygous Familial Hypercholesterolemia Market Access and Reimbursement of Therapies 15. Homozygous Familial Hypercholesterolemia Market Drivers 16. Homozygous Familial Hypercholesterolemia Market Barriers 17. Homozygous Familial Hypercholesterolemia Appendix 18. Homozygous Familial Hypercholesterolemia Report Methodology 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight About DelveInsight DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach. Media Contact Company Name: DelveInsight Contact Person: Gaurav Bora Email: Send Email Phone: +14699457679 Address: 304 S. 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