Latest news with #Alnylam
Yahoo
08-07-2025
- Health
- Yahoo
Market Barriers Continue to Challenge PNH Treatment Optimization Despite Emerging Options, According to Spherix Global Insights
New research highlights significant insurance-driven hurdles, increasing second-line uptake of Fabhalta (Novartis), and strong physician interest in pipeline therapies including zaltenibart (Omeros), pozelimab-cemdisiran (Regeneron/Alnylam), and ruxaprubart (NovelMed). EXTON, PA, July 08, 2025 (GLOBE NEWSWIRE) -- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder that can affect individuals of any age, race, or gender, though it is most commonly diagnosed in adults in their 30s and 40s. In the United States, an estimated 400 to 500 new cases are identified annually. The clinical presentation of PNH varies widely—some patients experience only mild symptoms, while others face life-threatening complications requiring interventions such as immunosuppressive therapy or blood transfusions. ¹ Hematologists are actively adapting their prescribing strategies in paroxysmal nocturnal hemoglobinuria (PNH), with future pipeline entrants poised to further reshape the treatment landscape. According to Spherix Global Insights' Q2 2025 update from RealTime Dynamix™: PNH (US), the availability and adoption of biosimilars are expected to significantly expand the overall treated PNH population—bringing more patients onto complement inhibitor therapy than ever before. The Spherix research projects that biosimilar eculizumab products—Bkemv (Amgen) and Epysqli (Teva/Samsung Bioepis)—will continue to gain traction, capturing share from both branded Soliris and Ultomiris (Alexion/AstraZeneca), as hematologists grow increasingly confident in the clinical utility and cost-effectiveness of biosimilar alternatives. Use of novel entrants in the second line is growing as well, with therapies such as Fabhalta (Novaratis) and Voydeya (AstraZeneca) capturing an increasing share of recent prescribing. These agents are selected for patients who progress or are sub-optimally controlled on C5 inhibitors. Despite these gains, access remains a major commercial hurdle. Many patients with PNH are believed to be suboptimally managed due to persistent insurance-related barriers. Nearly all prescribers report significant challenges navigating the prior authorization process, citing inconsistent payer requirements, delays in drug procurement, and a growing reliance on artificial intelligence by insurers to further restrict coverage. This evolving dynamic has become a major source of frustration for hematologists, often delaying timely access to appropriate therapies and complicating clinical decision-making. The competitive landscape in PNH continues to intensify, with future therapies gaining interest among both academic and community hematologists. The latest Spherix study incorporates target product profiles and prescriber expectations for emerging agents, including zaltenibart (Omeros), a MASP-3 inhibitor that has captured strong physician interest. Most hematologists anticipate zaltenibart will be adopted alongside existing first- and second-line therapies upon approval, driven by high efficacy expectations and its potential to address persistent unmet needs. Other late-stage candidates—such as pozelimab-cemdisiran (Regeneron/Alnylam) and ruxaprubart (NovelMed)—also register meaningful commercial and clinical potential among surveyed specialists. 'These results underscore that physicians aren't just passively watching the market evolve – they're preparing to act,' said Sarah Hendry, Hematology Franchise Head at Spherix. 'With rising expectations for access support and guideline alignment, manufacturers that combine clinical value with commercial enablement will be best positioned for success.' As was seen in Paris at the IPIG international conference when Spherix presented data from their Patient Chart Dynamix™, membership in critical professional societies is growing as hematologists seek the guidance in PNH treatments and care. The most recent release offers a comprehensive look at the U.S. PNH market, including prescribing patterns, brand dynamics, access challenges, and future projections. Delivered quarterly, RealTime Dynamix™ helps commercial teams anticipate shifts, benchmark against competitors, and support strategic planning ahead of key market events. RealTime Dynamix™ is an independent service providing strategic guidance through quarterly or semiannual reports, which include market trending and a fresh infusion of event-driven and variable content with each wave. The reports provide an unbiased view of the competitive landscape within rapidly evolving specialty markets, fueled by robust HCP primary research and our in-house team of experts. Patient Chart Dynamix™ is an independent service that includes robust patient chart audits and integrated specialist surveys fielded biannually. This research provides an in-depth, real-world view of treatment practices by combining verified patient data with attitudinal insights from physicians. The series highlights clinical decision-making, treatment sequencing, and outcomes for targeted patient populations across key therapeutic areas. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. CONTACT: Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 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
Time Business News
24-06-2025
- Health
- Time Business News
RNAi Crucial Aspects and Innovations in the RNAi Technology Market
RNA interference (RNAi) is biological process where RNA molecules inherit gene expression or translation by neutralizing targeted mRNA molecules. RNAi is crucial in regulating genes and virus protection in eukaryotic organisms. A growing prevalence of genetic disorders, rising need for therapies that target cancer, growing emphasis on neurological disorders, growing RNA interference usage in viral and infectious disease treatments, increased research and development and innovation in delivery technologies regarding RNAi technology market. Developmental regulators occur naturally as hormones or proteins which foster cellular differentiation, development, proliferation, and healing. These cells are essential for immune system control, tissue repair, and development. Key Growth Drivers and Opportunities Genetic Disorder Cases Spike: As genetic disorders become more prevalent, there is increasing demand for enhanced and specialized treatments. One of the most promising methods in this area is RNA interference (RNAi) technology. With given factors the RNAi technology will possibly be surged, generating investments, research and development and end-up with more extensive use for genetic disorder treatment. Advancement in Delivery Methods: Delivery method advancements include nanotechnology-based delivery systems and non-transgenic delivery technologies, and are estimated to mitigate delivery issues of RNAi therapeutics. Ongoing research is focusing on studying the mechanisms OD siRNA delivery and catalytic RNA vehicle development to make RNAi based cancer gene therapy having more impact. Challenges The Off-target implications which result when RNAi treatments accidently alter genes other than the intended target, restrict growth in markets. Off target effects or phenotypic results are caused by an RNAi reagent's elimination of unintended genes rather that the gene of interest. Accidental gene suppression is often referred to as an off-target effect. It develops when the RNAi molecule attaches itself to something that is not intended. Innovation and Expansion F FDA Approves Qfitlia (fitusiran), the First siRNA (RNAi Therapeutic) for the Treatment of Hemophilia A or B In March 2025, Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, highlighted the significance of the U.S. Food and Drug Administration's (FDA) approval of Qfitlia (fitusiran), the sixth Alnylam-discovered RNAi therapeutic approved in the U.S., and the first and only therapeutic to lower antithrombin (AT), a protein that inhibits blood clotting, with the goal of promoting thrombin generation to rebalance hemostasis and prevent bleeds. Alnylam's vutrisiran shows positive Phase 3 results in ATTR cardiomyopathy In June 2024, RNAi drug vutrisiran was announced by Alnylam that reduces mortality and recurrent cardiovascular events by up to 33% in patients with transthyretin amyloidosis–related cardiomyopathy, clearing the way for an upcoming NDA submission. RNAi Therapies emerge for Hereditary Transthyretin Amyloidosis (ATTR) In April 2025, The Yuzu Matcha Martini has become a new ready-to-drink bottle that was introduced by world-renowned bartender Hidetsugu Ueno in collaboration with On the Rocks Cocktails and The House of Suntory. The bottle is matched with the Japanese Boulevardier, a traditional whiskey cocktail with an Eastern touch, so it's not simply a matcha martini to add to the family's bar. Four RNAi drugs show >80% Reduction in Lipoprotein (a) In August 2024, Early-phase trials of four RNAi-based therapeutics have managed to lower plasma Lp(a) by more than 80%, and Phase 3 studies are now being conducted to assess their impact on cardiovascular outcomes. Combination mRNA+RNAi Nanoparticle Therapy for Complex Diseases In December 2024, Codedeliry of mRNA and siRNA via nanoparticles can simultaneously boost positive gene expression and mute detrimental genes, revealed to research from the University of Ottawa. This opens up fresh therapies for a range of ailments, notably cancer and heart disorders. Inventive Sparks, Expanding Markets Companies' actives in Rnai technology market includes Alnylam Pharmaceuticals, Inc., ARROWHEAD PHARMACEUTICALS, INC., Silence Therapeutics, Thermo Fisher Scientific Inc., Revvity Discovery Limited, Company, and among others. Companies for RNAi technology aim on developing safe, precise, and efficient gene-silencing therapies for broad range of rare and genetic diseases. Their goals involve getting regulatory clearances for additional indications, refining delivery methods, minimizing off-target effects, and expanding the application of RNA interference beyond liver-targeted therapies. About Author: Prophecy is a specialized market research, analytics, marketing and business strategy, and solutions company that offer strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high value opportunities in the target business area. Also, we help our client to address business challenges and provide best possible solutions to overcome them and transform their business. TIME BUSINESS NEWS
Yahoo
19-06-2025
- Business
- Yahoo
Alnylam names Pushkal Garg as EVP, chief research and development officer
Alnylam (ALNY) Pharmaceuticals announced that Pushkal Garg, M.D., has been appointed Executive Vice President, Chief Research and Development Officer to oversee an integrated R&D organization. 'I could not be more excited for Pushkal to take the helm of the combined R&D organization, given his strategic vision, operational excellence and impressive track record building and steering an industry-leading, innovative drug development organization that has delivered a new class of medicines to the world,' said Yvonne Greenstreet, M.D., CEO. Garg joined Alnylam in 2014 to build and lead the company's Development group. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on ALNY: Disclaimer & DisclosureReport an Issue Alnylam price target raised to $338 from $330 at JPMorgan Cautious Outlook on Alnylam Pharma: Hold Rating Amid Incremental EU Approval and Promising US Sales Projections Alnylam receives European Commission approval of AMVUTTRA Alnylam Pharma's Strong Execution and Innovation in ATTR-CM Space Drives Positive Outlook Promising Market Outlook and Strong Product Pipeline Drive Buy Rating for Alnylam Pharma 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
Business Wire
18-06-2025
- Business
- Business Wire
Alnylam Announces Promotion of Pushkal Garg to Chief Research and Development Officer
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that Pushkal Garg, M.D., has been appointed Executive Vice President, Chief Research and Development Officer to oversee an integrated R&D organization. Building on the launch of AMVUTTRA ® in transthyretin amyloidosis with cardiomyopathy (ATTR-CM), Alnylam is entering a new phase of growth, and a combined R&D organization will accelerate pipeline progress by fostering earlier collaboration, faster decision-making, and more agile responses to both scientific opportunity and patient need. 'I could not be more excited for Pushkal to take the helm of the combined R&D organization, given his strategic vision, operational excellence and impressive track record building and steering an industry-leading, innovative drug development organization that has delivered a new class of medicines to the world,' said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. 'In recent years, we've made remarkable progress across all facets of our innovation engine, achieving numerous drug approvals and positive Phase 3 readouts, and rapidly increasing the size of our clinical pipeline. As our science has advanced, so has the need for tighter alignment across Research and Development to ensure that we continue to scale our impact and transform the lives of patients across a growing number of diseases.' 'As we begin this exciting new chapter, I am energized about what lies ahead for Alnylam R&D and for patients,' said Dr. Garg. 'I believe we are just beginning to scratch the surface of what RNAi therapeutics can do to improve human health. Our ambition is to deliver RNAi therapeutics to every major tissue in the body and thereby address many additional diseases that lack effective treatments. It's a privilege for me to have the opportunity to work with and lead an integrated R&D organization that has already validated a new class of medicines so that we can imagine the next generation of treatments, drive them from the lab to approval, and help patients live healthier lives.' Dr. Garg joined Alnylam in 2014 to build and lead the Company's Development group. He previously held leadership roles at Bristol-Myers Squibb and Millennium Pharmaceuticals. He received a B.A. with high honors in Biochemistry from the University of California, Berkeley, and an M.D. from the University of California, San Francisco (UCSF). He completed residency training in Internal Medicine at UCSF, was a fellow in the Robert Wood Johnson Clinical Scholars Program at Johns Hopkins University, and served on the faculty of Harvard Medical School and the Brigham & Women's Hospital in Boston prior to joining the industry. About RNAi Therapeutics RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as 'a major scientific breakthrough that happens once every decade or so,' and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its 'Alnylam P5x25' strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Alnylam Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam's expectation that a combined R&D organization will accelerate pipeline progress; Alnylam's ability to scale its impact and transform the lives of patients across a growing number of diseases; the potential for RNAi therapeutics to improve human health; Alnylam's ability to deliver RNAi therapeutics to every major tissue in the body and to address many additional diseases that lack effective treatments; and Alnylam's ability to imagine the next generation of treatments, achieve regulatory approval for additional treatments and to help patients live healthier lives should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam's ability to successfully execute on its 'Alnylam P 5 x25' strategy; Alnylam's ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; and any delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; as well as those risks more fully discussed in the 'Risk Factors' filed with Alnylam's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam's subsequent Quarterly Reports on Form 10-Q and in its other SEC filings. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
CNBC
09-06-2025
- Business
- CNBC
Best Stocks: Three healthcare names to ponder including a biotech back to levels not seen in a decade
(This is The Best Stocks in the Market , brought to you by Josh Brown and Sean Russo of Ritholtz Wealth Management.) Josh here — The healthcare sector has entered the chat. There are 12 healthcare names now on our Best Stocks list as of the end of last week. I'll show you a few of these set-ups below and then Sean's going to share some fundamentals for these healthcare firms. Sector Leaderboard As of 6/9/2025 morning, there are 114 names on The Best Stocks in the Market list Top Sector Ranking: Top Industries: Top 5 Best Stocks by Relative Strength: Sector Spotlight: Healthcare Josh — Alnylam Pharma (ALNY) just broke out above prior resistance at $300. There was company-specific news about new indications for one of their most important drugs that led to multiple price target raises on The Street. Alnylam is named for Alnilam, the brightest star in the Belt of Orion constellation, which ancient mariners used for navigation. The founders of the company believed their trailblazing work in the field of RNA interference (RNAi) would chart a brand new course of drug discovery and development by which the next generation of scientists would be guided. So far, so good as ALNY grew revenue to $2.25 billion in revenue last year and the company's market cap swelled to a respectable $40 billion. Wall Street's median price target is ten percent higher than today's price while the most bullish analyst, H.C. Wainwright, just published a target of $500 this past week. In Q1 2025, revenue surged by over 28% year-over-year to approximately $594 million, driven largely by the strength of the TTR franchise. Looking ahead, analysts project revenue growth of 24–33% through 2025–2026, supported by improving EPS and an expected annual revenue of $2.9 billion. The company also maintains a strong balance sheet, with around $223 million in free cash flow, a quick ratio of approximately 2.7×, and increasing support from major institutional investors who are adding to their positions. (data via Reuters) Sean — AMVUTTRA (vutrisiran) was recently FDA‑approved for cardiomyopathy, expanding its market from neuropathy, which is a major catalyst for the stock — it significantly expands its addressable market beyond its original use for polyneuropathy. This approval allows Alnylam to target patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a much larger population than those with ATTR polyneuropathy. The drug's unique RNA interference mechanism and dosing schedule (quarterly or biannual injections) give it a competitive edge over existing therapies like Pfizer's Vyndaqel. This dual-indication approval not only boosts Alnylam's revenue potential but also strengthens its path toward profitability, making AMVUTTRA a key growth driver for the company and a pivotal reason for recent momentum. Josh — Allow for short-term consolidation in the $300 area, await the next catalyst. 50-day crossing over 200-day important signal that a new uptrend could be forming. Cardinal Health Josh — All three major Pharmaceutical Wholesalers made the list - McKesson (MCK) , Cencora (COR) and Cardinal Health (CAH) , only Cardinal looks good technically right now: CAH held its 50-day on a weekly closing basis during the April bloodbath, I'd use that area as a stop and update it each week. If the uptrend breaks, there's no reason to be long. Sean — CAH is a $37 billion wholesaler, sourcing and distributing branded, generic, and specialty pharmaceutical products to pharmacies, hospitals, and healthcare providers. All three names on our list, Cardinal, Cencora, and McKesson, hold well over 90% of the US pharmaceutical wholesale industry. CAH trades at an 18x forward PE and a 15x P/FCF - this thing is generating cash for investors. It has a 1.3% dividend yield and is growing earnings 8% this year, and is expected to grow its earnings 12% next year. Gilead Sciences Josh — Gilead (GILD) , as you can see in the chart above, is not done going up. This name has been on the list for most of the year so far. At the market lows this spring, it never violated its upward-sloping 200-day moving average — didn't even pay it a visit. And just for fun, below is the "forever" chart back to the company's IPO. I am a believer that price has memory, but I'm not sure there are still a lot of shareholders hanging around here from the last time it traded near the $120 level a decade ago. That said, I wouldn't be surprised to see a battle at that old high as new shareholders buy from sellers who are just thrilled to be getting out break-even. That's what makes a market. Sean — Gilead Sciences (GILD) has been on a run. Its experimental HIV prevention shot, lenacapavir, showed near-complete effectiveness in trials and is expected to generate $2–4 billion in peak annual sales—potentially transforming the HIV prevention market. The company has also posted solid financial results, with 2024 revenue and earnings rising due to strong performance in its HIV, liver disease, and oncology franchises. Analysts have raised price targets across the board, reflecting increased confidence in the company's pipeline and execution. GILD has a meaningful run rate of free cash flow (~$9.6B) and a growing pipeline of drugs for the future. 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