Latest news with #AMVUTTRA
Yahoo
13-06-2025
- Business
- Yahoo
The European Commission Approves Alnylam's AMVUTTRA
Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is one of the 13 Biotech Stocks with Huge Upside Potential. It declared on June 9, 2025, that AMVUTTRA® (vutrisiran) has been approved by the European Commission to treat wild-type or hereditary ATTR amyloidosis with cardiomyopathy (ATTR-CM). A researcher studying genetic medicines under a microscope in a biopharmaceutical laboratory. As a result, AMVUTTRA is the first and only RNAi treatment approved in the EU for transthyretin amyloidosis symptoms that show as cardiomyopathy and polyneuropathy. The HELIOS-B Phase 3 research, which revealed a 36% mortality reduction over 42 months and a 28% decrease in all-cause mortality and recurrent cardiovascular events, served as the basis for the approval. More than 6,000 patient-years of safety data support the quarterly administration of the medication. The drug is already authorized in Brazil and the United States. Up to 100,000 individuals in Europe have ATTR amyloidosis, mostly as cardiomyopathy. An RNAi treatment called vutrisiran provides continuous knockdown by stopping TTR production at its source with quarterly injections. Functional and quality-of-life gains were validated by the HELIOS-B trial, which involved patients on SGLT2 inhibitors and tafamidis. Elevations of liver enzymes and injection site responses are frequent adverse effects. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is an innovator in the investigation of RNA interference medicines. While we acknowledge the potential of ALNY as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. 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
11-06-2025
- Business
- Yahoo
The European Commission Approves Alnylam's AMVUTTRA
Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is one of the 13 Biotech Stocks with Huge Upside Potential. It declared on June 9, 2025, that AMVUTTRA® (vutrisiran) has been approved by the European Commission to treat wild-type or hereditary ATTR amyloidosis with cardiomyopathy (ATTR-CM). A researcher studying genetic medicines under a microscope in a biopharmaceutical laboratory. As a result, AMVUTTRA is the first and only RNAi treatment approved in the EU for transthyretin amyloidosis symptoms that show as cardiomyopathy and polyneuropathy. The HELIOS-B Phase 3 research, which revealed a 36% mortality reduction over 42 months and a 28% decrease in all-cause mortality and recurrent cardiovascular events, served as the basis for the approval. More than 6,000 patient-years of safety data support the quarterly administration of the medication. The drug is already authorized in Brazil and the United States. Up to 100,000 individuals in Europe have ATTR amyloidosis, mostly as cardiomyopathy. An RNAi treatment called vutrisiran provides continuous knockdown by stopping TTR production at its source with quarterly injections. Functional and quality-of-life gains were validated by the HELIOS-B trial, which involved patients on SGLT2 inhibitors and tafamidis. Elevations of liver enzymes and injection site responses are frequent adverse effects. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is an innovator in the investigation of RNA interference medicines. While we acknowledge the potential of ALNY as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. 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
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. DISCLOSURES: (None) All opinions expressed by the CNBC Pro contributors are solely their opinions and do not reflect the opinions of CNBC, NBC UNIVERSAL, their parent company or affiliates, and may have been previously disseminated by them on television, radio, internet or another medium. THE ABOVE CONTENT IS SUBJECT TO OUR TERMS AND CONDITIONS AND PRIVACY POLICY . THIS CONTENT IS PROVIDED FOR INFORMATIONAL PURPOSES ONLY AND DOES NOT CONSITUTE FINANCIAL, INVESTMENT, TAX OR LEGAL ADVICE OR A RECOMMENDATION TO BUY ANY SECURITY OR OTHER FINANCIAL ASSET. THE CONTENT IS GENERAL IN NATURE AND DOES NOT REFLECT ANY INDIVIDUAL'S UNIQUE PERSONAL CIRCUMSTANCES. THE ABOVE CONTENT MIGHT NOT BE SUITABLE FOR YOUR PARTICULAR CIRCUMSTANCES. BEFORE MAKING ANY FINANCIAL DECISIONS, YOU SHOULD STRONGLY CONSIDER SEEKING ADVICE FROM YOUR OWN FINANCIAL OR INVESTMENT ADVISOR. INVESTING INVOLVES RISK. EXAMPLES OF ANALYSIS CONTAINED IN THIS ARTICLE ARE ONLY EXAMPLES. THE VIEWS AND OPINIONS EXPRESSED ARE THOSE OF THE CONTRIBUTORS AND DO NOT NECESSARILY REFLECT THE OFFICIAL POLICY OR POSITION OF RITHOLTZ WEALTH MANAGEMENT, LLC. JOSH BROWN IS THE CEO OF RITHOLTZ WEALTH MANAGEMENT AND MAY MAINTAIN A SECURITY POSITION IN THE SECURITIES DISCUSSED. ASSUMPTIONS MADE WITHIN THE ANALYSIS ARE NOT REFLECTIVE OF THE POSITION OF RITHOLTZ WEALTH MANAGEMENT, LLC" TO THE END OF OR OUR DISCLOSURE. Click here for the full disclaimer.
Business Wire
09-06-2025
- Business
- Business Wire
Alnylam Receives European Commission Approval for AMVUTTRA ® (vutrisiran) for the Treatment of ATTR Amyloidosis with Cardiomyopathy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the European Commission (EC) has granted approval for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) as an additional indication for its orphan RNAi therapeutic AMVUTTRA ® (vutrisiran). The approval broadens the indication for AMVUTTRA, which now becomes the first and only RNAi therapeutic approved by the EC for the treatment of the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. Alnylam Receives European Commission Approval for AMVUTTRA® (vutrisiran) for the Treatment of ATTR Amyloidosis with Cardiomyopathy Share 'Estimates show approximately 100,000 people are affected by ATTR amyloidosis across Europe, most with cardiomyopathy, so this approval marks a critical step toward addressing this underserved patient population,' said Pushkal Garg, M.D., Chief Medical Officer at Alnylam. 'AMVUTTRA is supported by a well-established efficacy and safety profile, with over 6,000 patient-years of global experience in the treatment of hATTR with polyneuropathy. By delivering rapid and sustained knockdown of TTR through convenient, quarterly dosing, it offers a clinically differentiated approach with the potential to transform outcomes for patients living with this debilitating and potentially fatal disease. We now look forward to securing access to AMVUTTRA for eligible patients across the EU as quickly as possible.' The EC decision is based on positive results from the pivotal HELIOS-B Phase 3 study – a randomized, double-blind, placebo-controlled, multicenter, global trial that enrolled a diverse group of patients reflective of the contemporary ATTR-CM population, including those receiving substantial concurrent use of available standard-of-care therapies such as tafamidis and SGLT2 inhibitors. AMVUTTRA met all 10 pre-specified primary and secondary endpoints across both the overall and monotherapy populations. These included statistically significant reductions in all-cause mortality and recurrent cardiovascular events, as well as significant improvements in functional capacity (6-minute walk test), health status and quality of life (Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (NYHA class). In the overall population, AMVUTTRA achieved a 28% reduction in the primary composite of all-cause mortality and recurrent cardiovascular events as compared to placebo. Mortality in this population was significantly reduced by 36% through 42 months in a pre-specified secondary endpoint analysis which included up to 36 months of the double-blind period plus six months of open-label extension. In HELIOS-B, rates of adverse events (AEs), serious AEs, severe AEs and AEs leading to study drug discontinuation were similar between the AMVUTTRA and placebo arms. Adverse drug reactions of AMVUTTRA include injection site reactions and increase in blood alkaline phosphatase and alanine transaminase. Detailed results from the HELIOS-B study were published in The New England Journal of Medicine. 1 'The HELIOS-B findings provide compelling evidence to support the use of vutrisiran as a first-line treatment option for patients with ATTR-CM,' said Marianna Fontana, M.D., Ph.D., HELIOS-B investigator, Professor of Cardiology, University College London, National Amyloidosis Center, Royal Free Hospital, London. 'As a physician, it is a privilege to see the true impact on patients in the clinic. The trial enrolled a broad population reflective of real-world clinical practice, and that's what makes the results so meaningful. This is a milestone for patients, who now have a new treatment option that has the potential to significantly improve outcomes of this disease.' ATTR-CM is caused by the deposition of misfolded TTR fibrils, which drive progressive and irreversible cardiovascular damage and premature death. AMVUTTRA is an RNAi therapeutic that works upstream by delivering sustained knockdown of disease-causing TTR at its source. In the EU, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers, offering flexibility in treatment delivery. 'Amyloidosis is a serious and progressive disease that significantly impacts not only patients' physical health, but also their quality of life and independence. I am thrilled by the news of a new therapy for people in the EU living with ATTR-CM who often face delayed diagnosis. Having a new treatment option available marks a welcome development for the amyloidosis community,' said Giovanni d'Alessio, President of the Amyloidosis Alliance. In May 2025, the European Medicines Agency's Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on the maintenance of the EU Orphan Designation for AMVUTTRA in ATTR amyloidosis. AMVUTTRA was approved in March 2025 by the U.S. Food and Drug Administration (FDA) and the Brazilian Health Regulatory Agency (ANVISA) for the treatment of the cardiomyopathy of wild-type or hereditary ATTR amyloidosis in adults. Alnylam continues to pursue additional global submissions to bring vutrisiran to patients worldwide. Indications In the EU, AMVUTTRA ® (vutrisiran) is indicated for the treatment of: hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase. For additional information about vutrisiran, please see the full Summary of Product Characteristics. About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of variant and wild‑type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. It is also approved in the U.S. and Brazil for the treatment of wild-type or hereditary ATTR amyloidosis in adult patients with cardiomyopathy (ATTR-CM). In the EU, AMVUTTRA is administered once every three months, either by a healthcare professional or through self-administration by patients or their caregivers. About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000-300,000 people worldwide. 2-5 About RNAi 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. 6 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. 7 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. 6 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 P 5 x25 ' 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 expectations regarding the safety and efficacy of AMVUTTRA for the treatment of ATTR-CM; the potential for AMVUTTRA to be used as a first-line treatment for ATTR-CM; the potential for AMVUTTRA to address the underserved ATTR-CM patient population and to improve outcomes for ATTR-CM patients; and Alnylam's ability to secure access to AMVUTTRA for eligible patients across the EU and the timing of such access 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.
Business Wire
19-05-2025
- Business
- Business Wire
Alnylam to Share Progress Across its Transthyretin Amyloidosis Franchise Including Additional Analyses of the HELIOS-B Phase 3 Study Results at Heart Failure 2025 Congress
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present the latest data from its flagship transthyretin amyloidosis (TTR) franchise at the upcoming Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia. The latest analyses of the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM), including further outcomes data on cardiovascular hospitalizations and urgent heart failure visits up to 42-months, will be presented as a late-breaking abstract in the 'Hottest Trials and Trial Updates 1' session. Data from the HELIOS-B study supported the recent approvals of AMVUTTRA ® (vutrisiran) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults in the U.S. and Brazil. These data also supported the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending approval of vutrisiran for the same indication. AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of transthyretin, addressing the disease at its source, with four subcutaneous doses per year. Additional updates to be presented include the design and rationale of the TRITON-CM Phase 3 study of nucresiran (ALN-TTRsc04), an investigational next-generation TTR silencer, in patients with ATTR-CM, as well as an additional analysis from the HELIOS-B study of vutrisiran in patients with ATTR-CM who experienced disease progression while being treated with tafamidis. Presentation Details Vutrisiran Reduces All-Cause Mortality, Cardiovascular Mortality, and Cardiovascular Events in Patients with Transthyretin Amyloid Cardiomyopathy: Analysis from the HELIOS-B Trial Session: Hottest Trials and Trial Updates 1 Saturday, May 17, 11:50 – 11:58 CEST, 5:50 – 5:58 A.M. EST Presenting Author: Marianna Fontana, United Kingdom Clinical Presentation and Treatment Landscape of Patients with Transthyretin Amyloidosis With Cardiomyopathy: A Real-world Study in Five European Countries and Japan Session: Novel Insights into Heart Failure Therapeutics Sunday, May 18, 13:00 – 13:45 CEST, 7:00 – 7:45 A.M. EST Presenter: Caroline Morbach, Germany Utility of Genetic Testing For Diagnosing hATTR Patients: Results from a European and Middle East Genetic Testing Program Session: Novel Insights into Heart Failure Therapeutics Monday, May 19, 9:00 – 10:00 CEST, 3:00 – 4:00 A.M. EST Presenter: Antoine Bondue, Belgium Design and Rationale of a Phase 3 Study to Evaluate Efficacy and Safety of Nucresiran (ALN-TTRsc04) in Patients with Transthyretin Amyloidosis with Cardiomyopathy Session: Research Methodology Monday, May 19, 14:00 – 15:00 CEST, 8:00 – 9:00 A.M. EST Presenter: Marianna Fontana, United Kingdom Vutrisiran In Patients With Transthyretin Amyloidosis with Cardiomyopathy In HELIOS-B Who Had Progressed On Tafamidis Session: Evolving Treatment Paradigms in Heart Failure: SGLT2 Inhibition to TTR Stabilisation and Beyond Tuesday, May 20, 9:21 – 9:30 CEST, 3:21 – 3:30 A.M. EST Presenter: Jose Gonzalez-Costello, Spain About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of variant and wild-type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. In Europe, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide. 1-4 About RNAi 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 P 5 x25 ' 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. 1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638. 2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112. 3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst 4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.
