Latest news with #RNAi
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
Yahoo
28-05-2025
- Business
- Yahoo
Biogen Inc. (BIIB) Taps RNAi Therapy in City Therapeutics Deal
Biogen Inc. (NASDAQ:BIIB)'s recent partnership with City Therapeutics, Inc. to develop RNAi therapies for central nervous system diseases has fueled a 6% rise in its share price over the past month, reflecting renewed investor optimism. This move comes amid a broader rally in the tech sector driven by favorable policy shifts and positive market sentiment, as major indices have also trended upward. A scientist studying a petri dish with magnifying glass in a laboratory setting. The collaboration underscores Biogen Inc. (NASDAQ:BIIB)'s strategic pivot toward innovative treatments, expanding its pipeline beyond established Alzheimer's therapies like LEQEMBI. Analysts suggest this could strengthen BIIB's long-term growth prospects and help offset challenges such as declining multiple sclerosis revenues and mounting competition from generics and biosimilars. Despite the recent uptick, Biogen Inc. (NASDAQ:BIIB)'s three-year total shareholder return remains down 37.09%, significantly underperforming the broader US biotech industry, which gained 12.9% over the past year. The company's current share price still trades at a 36.67% discount to the analyst consensus target of $171.95, highlighting persistent investor caution regarding future earnings and revenue growth. While the RNAi collaboration may gradually shift market perceptions, BIIB faces ongoing pressure to deliver on its innovation-driven strategy and regain industry momentum. While we acknowledge the potential of BIIB to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than BIIB and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
21-05-2025
- Business
- Yahoo
RNAi Technology Market Report 2025: Global Industry Size, Shares, Trends, Opportunities, and Growth Forecast to 2030
The RNAi Technology Market offers growth opportunities in drug development, gene therapy, and agriculture due to RNAi's precision in gene silencing. Key drivers include the rising demand for RNAi therapeutics for complex diseases, personalized medicine, and advances in delivery systems, despite challenges like off-target effects. Dublin, May 21, 2025 (GLOBE NEWSWIRE) -- The "RNAi Technology Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2020-2030F" has been added to RNAi Technology Market was valued at USD 1.19 Billion in 2024 and is projected to reach USD 2.14 Billion by 2030, rising at a CAGR of 10.28%. RNA interference (RNAi) has emerged as a transformative tool in the biotechnology and pharmaceutical sectors, enabling targeted gene silencing through small RNA molecules. This mechanism has reshaped the landscape of gene regulation and therapeutic innovation, finding applications across drug development, gene therapy, agriculture, and molecular research. With its ability to selectively inhibit disease-related genes, RNAi is increasingly being integrated into drug pipelines for treating complex conditions such as cancer, genetic disorders, and viral infections. Pharmaceutical companies are prioritizing RNAi-based approaches due to their high precision, adaptability, and potential for developing personalized treatments. The continuous advancements in delivery systems, coupled with growing investments in RNAi therapeutics and diagnostics, are expected to drive robust market expansion through Market Driver: Growing Demand for RNAi-based TherapeuticsThe rising demand for RNAi-based therapeutics plays a critical role in propelling the growth of the global RNAi technology market. RNAi enables highly targeted treatment by silencing genes that contribute to the progression of chronic and genetic diseases, such as certain cancers, viral infections, and neurodegenerative conditions. As the global prevalence of such disorders continues to rise, there is a growing need for precise and less invasive treatment drugs support a personalized medicine approach, tailoring therapies based on individual genetic profiles and minimizing off-target effects. The successful commercialization of RNAi therapies, such as Onpattro, has strengthened confidence in the clinical viability of these treatments, encouraging more companies to invest in research and development. Furthermore, RNAi's utility in functional genomics and target validation makes it a valuable tool in drug discovery, reinforcing its expanding role across both therapeutic and research Market Challenge: Off-Target EffectsOff-target gene silencing presents a major obstacle to the widespread adoption of RNAi technology. Although RNAi is known for its precision, unintended interactions with non-target genes can lead to undesired cellular responses, including toxicity or immune reactions. These safety concerns require extensive preclinical and clinical validation, increasing development time and costs. Designing RNAi molecules with high specificity remains a complex task, particularly when targeting genes in complex biological systems. Off-target effects not only compromise therapeutic outcomes but also affect research accuracy in functional genomics. Addressing this challenge involves improving RNA sequence design, refining delivery mechanisms, and enhancing regulatory oversight to ensure the safe application of RNAi in both clinical and research Market Trend: Precision Medicine and Personalized TherapiesThe integration of RNAi technology into precision medicine is a significant trend shaping the future of the healthcare industry. As personalized therapies become increasingly important, RNAi's ability to silence specific genetic mutations makes it an ideal platform for individualized treatment strategies. In oncology, for instance, RNAi-based therapies are being developed to target genetic mutations unique to different tumor types, offering greater efficacy and reduced side effects compared to conventional therapies. This alignment with personalized medicine is also driving partnerships and R&D efforts focused on developing tailored RNAi-based solutions. By enabling gene-specific modulation, RNAi supports the broader shift toward customized, data-driven healthcare Players Profiled in this RNAi Technology Market Report Alnylam Pharmaceuticals, Inc Ionis Pharmaceuticals, Inc. Atalanta Therapeutics Phio Pharmaceuticals Benitec Biopharma Novartis AG AstraZeneca Silence Therapeutics Arbutus Biopharma Sylentis S.A. Report Scope RNAi Technology Market, by Type: Micro RNA Small interfering RNA Others RNAi Technology Market, by Application: Treatment Type Therapeutics Drug Delivery Agriculture Others RNAi Technology Market, by End User: Research and Academic Laboratories Diagnostic Laboratories Others RNAi Technology Market, by Region: North America Europe Asia-Pacific South America Middle East & Africa Key Attributes Report Attribute Details No. of Pages 185 Forecast Period 2024-2030 Estimated Market Value (USD) in 2024 $1.19 Billion Forecasted Market Value (USD) by 2030 $2.14 Billion Compound Annual Growth Rate 10.2% Regions Covered Global For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
15-05-2025
- Business
- Yahoo
AbbVie stakes $335M on a startup's RNAi drugs
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. AbbVie is staking hundreds of millions of dollars on RNA interference, agreeing on Wednesday to work with biotechnology startup ADARx Pharmaceuticals to develop experimental medicines for neurological conditions, immune diseases and cancer. Under the terms of the deal, AbbVie will pay ADARx $335 million in cash upfront. The startup could receive 'several billion dollars' more in the form of option-related fees and downstream payments, though that money isn't guaranteed. ADARx would also get a share of royalties on any marketed products to emerge from the collaboration. ADARx is part of a new generation of startups trying to take RNAi interference, a way of silencing disease-causing gene mutations, in new directions. While the technology has been used to make a handful of drugs now on the market, it's still largely focused on rare conditions and disease targets in the liver. ADARx and several others are trying to break beyond those limitations. The company has drugs in testing for rare as well as common conditions, and diseases of the eye and kidney. It also claims to be able to deliver RNAi drugs to the brain, an area of focus in its collaboration with AbbVie. The partners didn't disclose which specific diseases they're targeting. But ADARx's pipeline page shows 'multiple' programs underway for central nervous system disorders, along with the planned work in immunology and cancer. RNAi is 'a promising genetic medicine approach for silencing disease-causing genes, but challenges still remain in targeting and delivering [it] effectively," said Jonathon Sedgwick, AbbVie's senior vice president and global head of discovery research, in a statement. 'Together, we're committed to developing innovative solutions for difficult-to-treat diseases across neuroscience, immunology and oncology." ADARx is backed by more than a dozen investment firms and, last year, raised a $200 million Series C round that included several 'crossover' investors that back private and publicly traded companies. Alongside its RNAi work, the company is also pursuing RNA editing, another fast-growing area of drug research. It hasn't publicly disclosed any RNA editing programs yet, however.
Yahoo
13-05-2025
- Business
- Yahoo
Arrowhead Pharmaceuticals Inc (ARWR) Q2 2025 Earnings Call Highlights: Strong Financials and ...
Net Income: $370.4 million or $2.75 per share for the quarter ended March 31, 2025. Revenue: $542.7 million for the quarter ended March 31, 2025. Operating Expenses: $161.5 million for the quarter ended March 31, 2025. Cash and Investments: $1.1 billion as of March 31, 2025. Common Shares Outstanding: 138.1 million as of March 31, 2025. Cash Flow from Operating Activities: $460.1 million provided during the quarter ended March 31, 2025. Sarepta Agreement Revenue Recognition: $542.7 million recognized during the quarter ended March 31, 2025. Future Revenue Guidance: $90 million to $125 million expected over the next 12 months from initial fixed contract revenue. Warning! GuruFocus has detected 5 Warning Signs with ARWR. Release Date: May 12, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR) is on track to launch its first commercial product, plozasiran, this year, pending regulatory approval. The company has a strong financial position, with $1.1 billion in cash and investments, and is funded into 2028. Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR) has a robust pipeline with multiple Phase III studies and potential launches in the coming years. The company has secured a significant partnership with Sarepta Therapeutics, bringing in $500 million upfront and additional potential milestones. Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR) is making progress in expanding its RNAi technology to new areas, including CNS and obesity treatments. The biotech market remains uncertain, which could impact future funding and partnerships. Regulatory approval for plozasiran is still pending, with a PDUFA date set for November 18, 2025. There is competition in the triglyceride-lowering market, which could impact the commercial success of plozasiran. The company faces challenges in patient identification and market education for its rare disease treatments. Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR) has not yet finalized its commercialization strategy for plozasiran outside the U.S., which could delay international market entry. Q: For INHIBNY and ALK7, how are you setting expectations for initial monotherapy and potential combo data, including changes on weight loss, body composition, and relevant biomarkers? A: James Hamilton, Chief of Discovery and Translational Medicine, stated that they are not providing guidance on expectations as this is a first-in-human study. The animal data were compelling, showing good weight loss both as a monotherapy and in combination with tirzepatide. They are particularly interested in the quality of weight loss, such as the loss of visceral fat and retention of lean muscle mass, which was observed in animal models. Initial data is expected towards the end of the year. Q: Ahead of the plozasiran FDA review decision, how do you think about the robustness of your pancreatitis data and potential label differentiation? A: Bruce Given, Chief Operating Officer and Head of Research and Development, mentioned that they haven't had labeling negotiations with the FDA yet. They are confident in their data, which focused on confirmed pancreatitis using the AtLAA criteria, unlike Ionis, which included possible and probable cases. The focus is on achieving low triglyceride levels, which correlates with reduced pancreatitis risk. Q: For plozasiran in SHTG, what is the expected baseline rate of acute pancreatitis in your population for SHASTA-3 and 4, and what magnitude of effect do you expect to show? A: Christopher Anzalone, President and CEO, explained that they expect to replicate Phase II results, with mean triglycerides around 850 to 900 and a placebo-adjusted change of 53% reduction. For the pancreatitis study, the baseline might be closer to the PALISADE study, around 2,000, as patients with a history of pancreatitis are more susceptible even at lower triglyceride levels. Q: How are you thinking about advancing both ARO-ALK7 and ARO-INHBE through clinical development, and do you have interest or capacity to advance both? A: James Hamilton stated that both programs are being advanced through Phase I to assess safety, PD, and efficacy data. ARO-INHBE uses the well-vetted GalNAc technology, while ARO-ALK7 uses a new platform. They plan to choose one to move forward after reviewing the data, with potential for partnership opportunities. Q: How are you thinking about commercialization of plozasiran ex-U.S.? Are you planning to do that by yourself or looking for a partner? A: Christopher Anzalone mentioned that they are preparing for commercialization in Europe and are open to finding partners. They believe the European markets, particularly the large four European markets and the U.K., are well-suited for commercialization of a rare disease like FCS, with limited infrastructure and resources needed to reach centers of excellence. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus.
