Latest news with #JamesHamilton
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
25-06-2025
- Business
- Globe and Mail
Besra Gold Announces Results of Shareholders' Special Meeting
Melbourne, Victoria--(Newsfile Corp. - June 25, 2025) - Besra Gold Inc (ASX: BEZ) (Besra or the Company) Dear Shareholder, Please follow the link below to the results of the meeting on 24 June 2025 (Australian Time): Results of Meeting 24 June 2025 Michael Higginson Company Secretary North America Contact: James Hamilton M +1-416-471-4494
Miami Herald
21-06-2025
- Health
- Miami Herald
Econometer: Should the US ban drug advertising to consumers?
The U.S. is rare among Western nations because it allows pharmaceutical advertising. But a new effort aims to stop it. A bill was introduced in Congress recently that would ban pharmaceutical manufacturers from using direct-to-consumer advertising, from TV to social media, to promote their products. Prescription drug advertising employs a lot of people, directly and indirectly. Billions are spent on advertising each year, employing advertising workers, and 24.4% of ad minutes were for prescription drugs across evening news programs on ABC, CBS, CNN, Fox News, MSNBC and NBC this year through May, according to data from iSpot analyzed by The Wall Street Journal. Proponents of the bill say advertising drives up the cost of prescription goods. Pharmaceutical trade groups have said advertisements serve public health by increasing disease awareness and educating consumers about treatment options. Question:Should the U.S. ban drug advertising to consumers? Economists Alan Gin, University of San Diego YES: Advertising is supposed to give consumers more information about products, but are consumers really in a position to make an informed decision about pharmaceuticals? Those decisions are best left to physicians, who probably have more knowledge about the effectiveness of medications. Consumers can be swayed by slick and repetitive ads into wanting products that might not be the best for them. The money spent on the ads will add to the already high price of the drugs. James Hamilton, UC San Diego NO: Proponents of a ban argue that ads cause people to request unnecessary drugs. But advertisements helped several of my friends learn about options that they didn't know were available. I'm also concerned any time the government dictates what companies are allowed to talk about. It's appropriate to ensure ads do not make inaccurate claims. And doctors should always say no if patients request a prescription that the doctor does not believe is going to help them. Caroline Freund, UC San Diego School of Global Policy and Strategy YES: Advertising specific drugs leads to overprescribing, higher drug and insurance prices, and creates bad incentives, like promoting the most profitable drugs. Because insurance limits consumer costs, more prescription drugs are purchased than needed or used. If the goal is to share important information, industry groups can promote a range of treatments for a condition, leaving discussions of individual products to medical professionals. Drugs also carry risks that are not easily captured in 30 seconds. Kelly Cunningham, San Diego Institute for Economic Research NO: Firms do not advertise to raise costs but engage in marketing to inform the public (especially doctors writing prescriptions) of the drug's usefulness. Without marketing, firms would be unable to get information out necessary to make a drug salable in the first place. The drug's value is decided by the marketplace with consumers driving the entire process. Value of advertising is derived from the value consumers place on the drug, not the other way around. Norm Miller, University of San Diego NO: While most physicians try to keep up on the latest drug research, some do not, thus the need for public information about new drugs. What should be mandatory in ads are their efficacy, side effects and potential for addiction, using FDA verified stats. Lies and exaggerations should be illegal. It should also be illegal for drug manufacturers to incentivize or pay doctors for prescribing any drug, and physicians that take such gifts should lose their license. Ray Major, economist YES: Every ad starts with or ends with "ask your doctor if this drug is right for you." Prescription drug advertisement targets consumers hoping they ask their doctor for a specific brand of drug. Consumers are not qualified to self diagnose symptoms and prescribe drugs to themselves based on information from a commercial. Doctors should be prescribing drugs based on a patients' needs and not influenced by patients who have seen an ad for a prescription drug. David Ely, San Diego State University NO: Commercial speech by pharmaceutical companies that is truthful and informative should be protected. A ban on drug advertising goes too far. A better option is enhanced regulation by the FDA and FTC to ensure that the risks and effectiveness of prescription drugs are accurately communicated in advertising to the public. Under a ban, resources would be shifted to increased promotional efforts targeting health care providers so the cost of prescription goods may not decline. Executives Gary London, London Moeder Advisors NO: I am not a big fan of drug advertisements, but unlike cigarette ads, which clearly promoted sickness for generations, at least drugs are lifesaving. The government should not get involved. However, I have never fully understood why pharmaceutical companies promote directly to patients rather than physicians. They complicate medical care. Be that as it may, these advertisements certainly prop up the cable channels, who need the revenue. Bob Rauch, R.A. Rauch & Associates YES: The U.S. and New Zealand are the only countries that allow pharmaceutical companies to advertise directly to consumers. Drug ads often downplay the risks, leading to uninformed decisions. Ads can push consumers toward brand-name drugs, even when cheaper alternatives exist. Also, patients may request unnecessary medications, pressuring doctors to prescribe them. Sure, ads can educate, lead to earlier diagnosis, and boost the economy! But let's limit ads during the first few years of release. Phil Blair, Manpower NO: They are a product like any other. With artificial intelligence, clients and patients can educate themselves on various options just like they do with other products. Of course, they should heed their doctors' advice. Austin Neudecker, Weave Growth YES: Drugmakers spent $10 billion on direct-to-consumer ads last year. These costs are ultimately reflected in the world's highest per-capita health care bill, with relatively poor health outcomes. Slick spots encourage viewers to "ask your doctor" for brands even when cheaper generics accomplish the same goal. Treatment decisions should be based on clinical evidence, not marketing budgets. Pharma could shift a fraction of this outreach to physician education so that patients will still learn about therapies from an informed source. Chris Van Gorder, Scripps Health YES: Absolutely. The cost of pharmaceuticals has become prohibitive to patients and providers like hospitals, and the huge cost of advertising is wrapped into those costs. While we want informed patients, pharmaceutical education should be handled by patients' physicians, not a jingle on TV. Advertising also can be misleading and increase the cost of drugs to taxpayers - which is why many countries prohibit advertising. Jamie Moraga, Franklin Revere NO: While I don't enjoy watching the litany of drug advertisements consistently shown on family programming, I don't support a blanket ban. Instead, drug advertising should follow the model currently allowed to cigarette advertising: prohibit ads on TV and radio but allow other forms of advertising with appropriate limitations and regulations. While raising awareness of available treatments can be beneficial, the current barrage of drug advertising is excessive and likely leads to over prescription and increased health care costs. Copyright (C) 2025, Tribune Content Agency, LLC. Portions copyrighted by the respective providers.
Business Wire
02-06-2025
- Business
- Business Wire
Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-ALK7 for the Treatment of Obesity
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity. ARO-ALK7 is designed to intervene in a known pathway that signals the body to store fat in adipose tissue. The study initiates in otherwise healthy obese subjects using single and multiple escalating doses of ARO-ALK7 monotherapy and is expected to progress rapidly to investigate combinations of ARO-ALK7 with tirzepatide in obese patients with and without type 2 diabetes. ARO-ALK7 is designed to intervene in a known pathway that signals the body to store fat in adipose tissue. Share 'Arrowhead's two clinical stage RNAi-based obesity programs, ARO-ALK7 and ARO-INHBE, intervene in a known pathway that signals the body to store fat in adipose tissue. Both programs have strong genetic validation and promising results in preclinical studies, which suggest that silencing the respective genes may lead to reduced body weight and potentially preserve lean muscle mass resulting in improved body composition,' said James Hamilton, M.D., Chief Medical Officer and Head of R&D. 'This ongoing Phase 1/2a clinical study will evaluate single and multiple ascending doses of ARO-ALK7 as monotherapy in otherwise healthy obese volunteers as well as multiple doses in obese patients with or without type 2 diabetes in combination with incretin therapy.' About ARO-ALK7 ARO-ALK7 is designed to silence adipocyte expression of the ACVR1C gene to reduce production of Activin receptor-like kinase 7 (ALK7), which acts as a receptor in a pathway that regulates energy homeostasis in adipose tissue. In large genetic datasets, reduced ACVR1C expression has been associated with healthier adipose distribution and reduced risk of obesity-related metabolic complications. In preclinical animal studies, ALK7 silencing in adipose tissue led to reduced body weight and fat mass with preservation of lean muscle. Treatment with investigational ARO-ALK7 has the potential to reduce visceral adiposity and improve lipid and glycemic parameters. About the AROALK7-1001 Phase 1/2 Study AROALK7-1001 (NCT06937203) is a Phase 1/2a first-in-human dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-ALK7 in up to 90 adult volunteers with obesity. Part 1 of the study is designed to assess single and multiple doses of ARO-ALK7 monotherapy, and Part 2 of the study is designed to assess ARO-ALK7 in combination with tirzepatide, a subcutaneously administered GLP-1/GIP receptor co-agonist that has been approved in the United States and the European Union for management type 2 diabetes mellitus since 2022 and weight management since 2023/2024 respectively. About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit 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 impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc.
Associated Press
02-06-2025
- Business
- Associated Press
Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-ALK7 for the Treatment of Obesity
PASADENA, Calif.--(BUSINESS WIRE)--Jun 2, 2025-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity. ARO-ALK7 is designed to intervene in a known pathway that signals the body to store fat in adipose tissue. The study initiates in otherwise healthy obese subjects using single and multiple escalating doses of ARO-ALK7 monotherapy and is expected to progress rapidly to investigate combinations of ARO-ALK7 with tirzepatide in obese patients with and without type 2 diabetes. 'Arrowhead's two clinical stage RNAi-based obesity programs, ARO-ALK7 and ARO-INHBE, intervene in a known pathway that signals the body to store fat in adipose tissue. Both programs have strong genetic validation and promising results in preclinical studies, which suggest that silencing the respective genes may lead to reduced body weight and potentially preserve lean muscle mass resulting in improved body composition,' said James Hamilton, M.D., Chief Medical Officer and Head of R&D. 'This ongoing Phase 1/2a clinical study will evaluate single and multiple ascending doses of ARO-ALK7 as monotherapy in otherwise healthy obese volunteers as well as multiple doses in obese patients with or without type 2 diabetes in combination with incretin therapy.' About ARO-ALK7 ARO-ALK7 is designed to silence adipocyte expression of the ACVR1C gene to reduce production of Activin receptor-like kinase 7 (ALK7), which acts as a receptor in a pathway that regulates energy homeostasis in adipose tissue. In large genetic datasets, reduced ACVR1C expression has been associated with healthier adipose distribution and reduced risk of obesity-related metabolic complications. In preclinical animal studies, ALK7 silencing in adipose tissue led to reduced body weight and fat mass with preservation of lean muscle. Treatment with investigational ARO-ALK7 has the potential to reduce visceral adiposity and improve lipid and glycemic parameters. About the AROALK7-1001 Phase 1/2 Study AROALK7-1001 ( NCT06937203 ) is a Phase 1/2a first-in-human dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-ALK7 in up to 90 adult volunteers with obesity. Part 1 of the study is designed to assess single and multiple doses of ARO-ALK7 monotherapy, and Part 2 of the study is designed to assess ARO-ALK7 in combination with tirzepatide, a subcutaneously administered GLP-1/GIP receptor co-agonist that has been approved in the United States and the European Union for management type 2 diabetes mellitus since 2022 and weight management since 2023/2024 respectively. About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit 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:Source: Arrowhead Pharmaceuticals, Inc. View source version on CONTACT: Arrowhead Pharmaceuticals, Inc. Vince Anzalone, CFA 626-304-3400 [email protected]: LifeSci Advisors, LLC Brian Ritchie 212-915-2578 [email protected]: LifeSci Communications, LLC Kendy Guarinoni, Ph.D. 724-910-9389 [email protected] KEYWORD: CALIFORNIA UNITED STATES NORTH AMERICA INDUSTRY KEYWORD: HEALTH DIABETES GENETICS CLINICAL TRIALS PHARMACEUTICAL BIOTECHNOLOGY SOURCE: Arrowhead Pharmaceuticals, Inc. Copyright Business Wire 2025. PUB: 06/02/2025 07:30 AM/DISC: 06/02/2025 07:28 AM
