Latest news with #AlnylamPharmaceuticals
Yahoo
23-05-2025
- Business
- Yahoo
Exploring High Growth Tech Stocks In The US This May 2025
Over the last 7 days, the United States market has experienced a 1.4% drop, yet it remains up by 11% over the past year with earnings projected to grow by 14% annually. In such a dynamic environment, identifying high growth tech stocks requires evaluating their potential for innovation and scalability amidst these fluctuating market conditions. Name Revenue Growth Earnings Growth Growth Rating Super Micro Computer 26.38% 39.09% ★★★★★★ Ardelyx 20.78% 59.46% ★★★★★★ Travere Therapeutics 26.41% 64.47% ★★★★★★ Blueprint Medicines 21.36% 61.45% ★★★★★★ TG Therapeutics 26.46% 38.75% ★★★★★★ Alnylam Pharmaceuticals 23.65% 61.11% ★★★★★★ AVITA Medical 27.28% 60.66% ★★★★★★ Alkami Technology 20.54% 76.67% ★★★★★★ Ascendis Pharma 35.16% 60.26% ★★★★★★ Lumentum Holdings 21.59% 110.32% ★★★★★★ Click here to see the full list of 237 stocks from our US High Growth Tech and AI Stocks screener. Below we spotlight a couple of our favorites from our exclusive screener. Simply Wall St Growth Rating: ★★★★☆☆ Overview: Bilibili Inc. offers online entertainment services targeted at young audiences in China, with a market capitalization of $7.78 billion. Operations: Bilibili Inc. generates revenue primarily through mobile games, value-added services, advertising, and e-commerce. The company focuses on engaging the young demographic in China by offering a diverse range of online entertainment content. Bilibili, a key contender in the interactive media and services sector, is navigating its path toward profitability with an expected earnings growth of 47.78% annually. Despite current unprofitability, the company's revenue trajectory is promising with an 8.6% annual increase, outpacing the general US market rate. Recent financial disclosures reveal substantial progress: Q1 sales surged to CNY 1.73 billion from CNY 982.81 million year-over-year, alongside a dramatic reduction in net loss to CNY 9.1 million from CNY 748.55 million, underscoring significant operational improvements and efficient cost management strategies. Dive into the specifics of Bilibili here with our thorough health report. Evaluate Bilibili's historical performance by accessing our past performance report. Simply Wall St Growth Rating: ★★★★★☆ Overview: CoreWeave, Inc. operates a cloud platform focused on scaling, support, and acceleration for GenAI with a market cap of $51.54 billion. Operations: The company generates revenue primarily from its data processing services, amounting to $2.71 billion. CoreWeave has recently demonstrated a robust strategic approach to scaling its AI and cloud computing capabilities. With a notable 37.3% annual revenue growth, the company is outperforming the broader US market significantly. This growth trajectory is complemented by an aggressive expansion into European markets with a $2.2 billion investment aimed at enhancing AI cloud infrastructure, powered entirely by renewable energy sources. Moreover, CoreWeave's recent $4 billion deal with OpenAI to boost cloud computing capacity underscores its pivotal role in powering next-generation AI technologies, positioning it well for future industry demands despite its current unprofitability forecasted to pivot within three years with an expected earnings surge of 67.5% annually. Click here and access our complete health analysis report to understand the dynamics of CoreWeave. Assess CoreWeave's past performance with our detailed historical performance reports. Simply Wall St Growth Rating: ★★★★★★ Overview: Legend Biotech Corporation is a biopharmaceutical company engaged in the discovery, development, manufacture, and commercialization of novel cell therapies for oncology and other indications across the United States, China, and Europe with a market cap of approximately $5.22 billion. Operations: Legend Biotech focuses on developing and commercializing innovative cell therapies, generating revenue primarily from its biotechnology segment, which amounts to $728.30 million. Amidst a dynamic biotech landscape, Legend Biotech is making notable strides with its innovative CAR-T therapies, as evidenced by recent data presentations at major oncology conferences. The company's aggressive R&D focus is highlighted by a significant increase in its research budget, aligning with its strategic expansion into solid tumor treatments. Despite a current unprofitable status, Legend Biotech's revenue surged to $195 million in Q1 2025 from $94 million the previous year, demonstrating a robust growth trajectory. This performance is underpinned by substantial investments in new facilities like their upcoming global headquarters in New Jersey, ensuring sustained growth and innovation in cutting-edge biotechnological research and therapies. Take a closer look at Legend Biotech's potential here in our health report. Gain insights into Legend Biotech's past trends and performance with our Past report. Reveal the 237 hidden gems among our US High Growth Tech and AI Stocks screener with a single click here. Are you invested in these stocks already? Keep abreast of every twist and turn by setting up a portfolio with Simply Wall St, where we make it simple for investors like you to stay informed and proactive. Join a community of smart investors by using Simply Wall St. It's free and delivers expert-level analysis on worldwide markets. Explore high-performing small cap companies that haven't yet garnered significant analyst attention. Fuel your portfolio with companies showing strong growth potential, backed by optimistic outlooks both from analysts and management. Find companies with promising cash flow potential yet trading below their fair value. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include NasdaqGS:BILI NasdaqGS:CRWV and NasdaqGS:LEGN. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@
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.
Associated Press
17-05-2025
- Health
- Associated Press
AMVUTTRA® (vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 17, 2025-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today presented the most contemporary analysis of the HELIOS-B Phase 3 study of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) as a late-breaking abstract at the Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia. The results demonstrate that vutrisiran, which rapidly knocks down transthyretin, reduces key cardiovascular (CV) events such as CV hospitalizations, and heart failure (HF) hospitalizations. Additionally, in the analysis, urgent HF visits were reduced by 46% (95% CI: 0.30, 0.98; p = 0.041) in the overall population during the double-blind period, compared to placebo. These CV events often precede all-cause mortality (ACM) and are key indicators of disease progression. Importantly, results from the November 2024 data cut, including further follow up through up to 42 months, reinforce the primary HELIOS-B analysis showing vutrisiran's effect on ACM, and further demonstrate that vutrisiran reduces CV mortality. Through 42 months, the risk of ACM was reduced by 36% (95% CI: 0.46, 0.88; p = 0.007) and the risk of CV mortality was reduced by 33% (95% CI: 0.47, 0.96; p = 0.038) in the overall population, compared to placebo. For both the primary analysis and the current analysis, vital status through 42 months was ascertained for over 99% of all randomized patients from the HELIOS-B study, underscoring the robustness of the results. The study was conducted in a contemporary patient population with patients receiving robust background therapy, inclusive of treatment with a TTR stabilizer and SGLT2 inhibitors. The analysis of the HELIOS-B Phase 3 study, including mortality data through up to 42 months, was simultaneously published in JACC. 'From the primary analysis of HELIOS-B, we know that AMVUTTRA profoundly impacts all-cause mortality, while preserving patients' functional capacity and quality of life,' said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. 'These new data—including the impact on mortality, on cardiovascular events and on urgent heart failure visits, the latter of which was reduced by nearly half—add to the story of consistency and magnitude of benefit. I remain impressed by the HELIOS-B results, which are noteworthy given the substantial use of heart failure treatments in the study population, and I believe they continue to reinforce AMVUTTRA as a clinically differentiated, first-line option for patients with ATTR-CM.' The results from the analysis underscore the rapid and sustained benefits of vutrisiran in treating ATTR-CM across key endpoints: In a separate presentation on Tuesday, May 20, Alnylam will share findings from a subgroup analysis of HELIOS-B evaluating the impact of vutrisiran on ACM and recurrent CV events among patients identified by investigators as having experienced disease progression while being treated with tafamidis. Also at the Heart Failure 2025 Congress, Alnylam will present the study design and rationale for TRITON-CM, a Phase 3, randomized, double-blind, study of nucresiran in patients with ATTR-CM. Nucresiran is an investigational next-generation RNAi therapeutic targeting TTR that has been shown to deliver rapid knockdown of TTR greater than 95% with twice-annual dosing in a Phase 1 study. TRITON-CM is an event-driven CV outcomes trial with a primary endpoint of composite ACM and CV events. The study is on track to initiate in the first half of 2025 and will enroll approximately 1,200 patients with wild-type or variant TTR and confirmed cardiomyopathy, including those receiving background stabilizer therapy. Additional details of the study's secondary endpoints and key inclusion and exclusion criteria will be shared on Monday, May 19. AMVUTTRA ® (vutrisiran) was approved by the U.S. Food and Drug Administration (FDA) and the Brazilian Health Regulatory Agency (ANVISA) for treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of vutrisiran for the same indication. A formal regulatory decision by the European Commission of the EMA is expected by the third quarter of 2025. Vutrisiran is currently under review for the treatment of ATTR-CM by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025 and beyond. For additional information on Alnylam's presentations at the Heart Failure 2025 Congress, please visit Capella. Indications and Important Safety Information AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). In a study of patients with ATTR-CM, no new safety issues were identified. For additional information about AMVUTTRA, please see the full Information(revised March 2025) 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 discover and develop novel drug candidates and delivery approaches and 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 and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; 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 other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 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. 2016;21:5-9. 4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. View source version on CONTACT: Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom (Investors and Media) +1-617-682-4340 Josh Brodsky (Investors) +1-617-551-8276 KEYWORD: SERBIA EUROPE UNITED STATES NORTH AMERICA MASSACHUSETTS INDUSTRY KEYWORD: HEALTH CLINICAL TRIALS RESEARCH SCIENCE PHARMACEUTICAL CARDIOLOGY BIOTECHNOLOGY SOURCE: Alnylam Pharmaceuticals, Inc. Copyright Business Wire 2025. PUB: 05/17/2025 05:50 AM/DISC: 05/17/2025 05:51 AM
Yahoo
17-05-2025
- Health
- Yahoo
AMVUTTRA® (vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B
– Analysis Presented at the Heart Failure 2025 Congress Supports Primary Findings, Highlighting Impact of AMVUTTRA, which Delivers Rapid Knockdown of Transthyretin – – Additional 42-Month Data Reinforce Vutrisiran's Impact on the Risk of All-Cause Mortality and Further Underscore the Effect on Cardiovascular Mortality – – Vutrisiran Demonstrated Substantial Benefit Across a Range of Cardiovascular Events, Notably Reducing Urgent Heart Failure Visits by 46% in the Overall Patient Population During the Double-Blind Period – – Findings Simultaneously Published in JACC – – Details of TRITON-CM Phase 3 Study Evaluating Alnylam's Investigational Next-Generation TTR Silencer, Nucresiran, to be Presented at Congress – CAMBRIDGE, Mass., May 17, 2025--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today presented the most contemporary analysis of the HELIOS-B Phase 3 study of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) as a late-breaking abstract at the Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia. The results demonstrate that vutrisiran, which rapidly knocks down transthyretin, reduces key cardiovascular (CV) events such as CV hospitalizations, and heart failure (HF) hospitalizations. Additionally, in the analysis, urgent HF visits were reduced by 46% (95% CI: 0.30, 0.98; p = 0.041) in the overall population during the double-blind period, compared to placebo. These CV events often precede all-cause mortality (ACM) and are key indicators of disease progression. Importantly, results from the November 2024 data cut, including further follow up through up to 42 months, reinforce the primary HELIOS-B analysis showing vutrisiran's effect on ACM, and further demonstrate that vutrisiran reduces CV mortality. Through 42 months, the risk of ACM was reduced by 36% (95% CI: 0.46, 0.88; p = 0.007) and the risk of CV mortality was reduced by 33% (95% CI: 0.47, 0.96; p = 0.038) in the overall population, compared to placebo. For both the primary analysis and the current analysis, vital status through 42 months was ascertained for over 99% of all randomized patients from the HELIOS-B study, underscoring the robustness of the results. The study was conducted in a contemporary patient population with patients receiving robust background therapy, inclusive of treatment with a TTR stabilizer and SGLT2 inhibitors. The analysis of the HELIOS-B Phase 3 study, including mortality data through up to 42 months, was simultaneously published in JACC. "From the primary analysis of HELIOS-B, we know that AMVUTTRA profoundly impacts all-cause mortality, while preserving patients' functional capacity and quality of life," said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. "These new data—including the impact on mortality, on cardiovascular events and on urgent heart failure visits, the latter of which was reduced by nearly half—add to the story of consistency and magnitude of benefit. I remain impressed by the HELIOS-B results, which are noteworthy given the substantial use of heart failure treatments in the study population, and I believe they continue to reinforce AMVUTTRA as a clinically differentiated, first-line option for patients with ATTR-CM." The results from the analysis underscore the rapid and sustained benefits of vutrisiran in treating ATTR-CM across key endpoints: Impact of Vutrisiran Versus Placebo Overall PopulationN=654 Reduction in Urgent HF Visits through 36 Months 46% RR: 0.54 P = 0.041 Reduction in HF Hospitalizations through 36 Months 33% RR: 0.67 P = 0.002 Reduction in Risk of CV Events through 36 Months 27% RR: 0.73 P = 0.001 Reduction in CV Hospitalizations through 36 Months 25% RR: 0.75 P = 0.004 Reduction in Risk of ACM through 42 Months 36% HR: 0.64 P = 0.007 Reduction in CV Mortality through 42 Months 33% HR: 0.67 P = 0.038 In a separate presentation on Tuesday, May 20, Alnylam will share findings from a subgroup analysis of HELIOS-B evaluating the impact of vutrisiran on ACM and recurrent CV events among patients identified by investigators as having experienced disease progression while being treated with tafamidis. Also at the Heart Failure 2025 Congress, Alnylam will present the study design and rationale for TRITON-CM, a Phase 3, randomized, double-blind, study of nucresiran in patients with ATTR-CM. Nucresiran is an investigational next-generation RNAi therapeutic targeting TTR that has been shown to deliver rapid knockdown of TTR greater than 95% with twice-annual dosing in a Phase 1 study. TRITON-CM is an event-driven CV outcomes trial with a primary endpoint of composite ACM and CV events. The study is on track to initiate in the first half of 2025 and will enroll approximately 1,200 patients with wild-type or variant TTR and confirmed cardiomyopathy, including those receiving background stabilizer therapy. Additional details of the study's secondary endpoints and key inclusion and exclusion criteria will be shared on Monday, May 19. AMVUTTRA® (vutrisiran) was approved by the U.S. Food and Drug Administration (FDA) and the Brazilian Health Regulatory Agency (ANVISA) for treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of vutrisiran for the same indication. A formal regulatory decision by the European Commission of the EMA is expected by the third quarter of 2025. Vutrisiran is currently under review for the treatment of ATTR-CM by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025 and beyond. For additional information on Alnylam's presentations at the Heart Failure 2025 Congress, please visit Capella. Indications and Important Safety Information Indications Approved by the U.S. FDAAMVUTTRA® (vutrisiran) is indicated for the treatment of the: cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Important Safety InformationReduced Serum Vitamin A Levels and Recommended Supplementation AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse ReactionsIn a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%). In a study of patients with ATTR-CM, no new safety issues were identified. For additional information about AMVUTTRA, please see the full U.S. Prescribing Information (revised March 2025) 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 ATTRTransthyretin 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 RNAiRNAi (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 PharmaceuticalsAlnylam (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 StatementsThis 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 vutrisiran for the treatment of ATTR-CM, including the ability of vutrisiran to reduce mortality and cardiovascular events in ATTR-CM patients and to preserve patients' functional capacity and quality of life; the potential for vutrisiran to become a first-line therapy for ATTR-CM; the timing of the initiation of the TRITON-CM study and the number of patients who will be enrolled in that study; the timing of additional global regulatory submissions for vutrisiran; the timing or receipt of any additional regulatory approvals for vutrisiran for ATTR-CM; Alnylam's ability to execute on its "Alnylam P5x25" strategy and to deliver transformative medicines in both rare and common diseases benefit patients around the world through sustainable innovation and exceptional financial performance; and Alnylam's ability to have a leading biotech profile 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 P5x25" strategy; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and 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 and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; 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 other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 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. 2016;21:5-9.4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. View source version on Contacts Alnylam Pharmaceuticals, Regan Lindenboom(Investors and Media)+1-617-682-4340Josh Brodsky(Investors)+1-617-551-8276
Business Wire
17-05-2025
- Health
- Business Wire
(vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today presented the most contemporary analysis of the HELIOS-B Phase 3 study of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) as a late-breaking abstract at the Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia. The results demonstrate that vutrisiran, which rapidly knocks down transthyretin, reduces key cardiovascular (CV) events such as CV hospitalizations, and heart failure (HF) hospitalizations. Additionally, in the analysis, urgent HF visits were reduced by 46% (95% CI: 0.30, 0.98; p = 0.041) in the overall population during the double-blind period, compared to placebo. These CV events often precede all-cause mortality (ACM) and are key indicators of disease progression. Importantly, results from the November 2024 data cut, including further follow up through up to 42 months, reinforce the primary HELIOS-B analysis showing vutrisiran's effect on ACM, and further demonstrate that vutrisiran reduces CV mortality. Through 42 months, the risk of ACM was reduced by 36% (95% CI: 0.46, 0.88; p = 0.007) and the risk of CV mortality was reduced by 33% (95% CI: 0.47, 0.96; p = 0.038) in the overall population, compared to placebo. For both the primary analysis and the current analysis, vital status through 42 months was ascertained for over 99% of all randomized patients from the HELIOS-B study, underscoring the robustness of the results. The study was conducted in a contemporary patient population with patients receiving robust background therapy, inclusive of treatment with a TTR stabilizer and SGLT2 inhibitors. The analysis of the HELIOS-B Phase 3 study, including mortality data through up to 42 months, was simultaneously published in JACC. 'From the primary analysis of HELIOS-B, we know that AMVUTTRA profoundly impacts all-cause mortality, while preserving patients' functional capacity and quality of life,' said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. 'These new data—including the impact on mortality, on cardiovascular events and on urgent heart failure visits, the latter of which was reduced by nearly half—add to the story of consistency and magnitude of benefit. I remain impressed by the HELIOS-B results, which are noteworthy given the substantial use of heart failure treatments in the study population, and I believe they continue to reinforce AMVUTTRA as a clinically differentiated, first-line option for patients with ATTR-CM.' The results from the analysis underscore the rapid and sustained benefits of vutrisiran in treating ATTR-CM across key endpoints: In a separate presentation on Tuesday, May 20, Alnylam will share findings from a subgroup analysis of HELIOS-B evaluating the impact of vutrisiran on ACM and recurrent CV events among patients identified by investigators as having experienced disease progression while being treated with tafamidis. Also at the Heart Failure 2025 Congress, Alnylam will present the study design and rationale for TRITON-CM, a Phase 3, randomized, double-blind, study of nucresiran in patients with ATTR-CM. Nucresiran is an investigational next-generation RNAi therapeutic targeting TTR that has been shown to deliver rapid knockdown of TTR greater than 95% with twice-annual dosing in a Phase 1 study. TRITON-CM is an event-driven CV outcomes trial with a primary endpoint of composite ACM and CV events. The study is on track to initiate in the first half of 2025 and will enroll approximately 1,200 patients with wild-type or variant TTR and confirmed cardiomyopathy, including those receiving background stabilizer therapy. Additional details of the study's secondary endpoints and key inclusion and exclusion criteria will be shared on Monday, May 19. AMVUTTRA ® (vutrisiran) was approved by the U.S. Food and Drug Administration (FDA) and the Brazilian Health Regulatory Agency (ANVISA) for treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of vutrisiran for the same indication. A formal regulatory decision by the European Commission of the EMA is expected by the third quarter of 2025. Vutrisiran is currently under review for the treatment of ATTR-CM by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025 and beyond. For additional information on Alnylam's presentations at the Heart Failure 2025 Congress, please visit Capella. Indications and Important Safety Information Indications Approved by the U.S. FDA AMVUTTRA ® (vutrisiran) is indicated for the treatment of the: cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions In a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%). In a study of patients with ATTR-CM, no new safety issues were identified. For additional information about AMVUTTRA, please see the full U.S. Prescribing Information (revised March 2025) 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. 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 vutrisiran for the treatment of ATTR-CM, including the ability of vutrisiran to reduce mortality and cardiovascular events in ATTR-CM patients and to preserve patients' functional capacity and quality of life; the potential for vutrisiran to become a first-line therapy for ATTR-CM; the timing of the initiation of the TRITON-CM study and the number of patients who will be enrolled in that study; the timing of additional global regulatory submissions for vutrisiran; the timing or receipt of any additional regulatory approvals for vutrisiran for ATTR-CM; Alnylam's ability to execute on its ' Alnylam P 5 x25 ' strategy and to deliver transformative medicines in both rare and common diseases benefit patients around the world through sustainable innovation and exceptional financial performance; and Alnylam's ability to have a leading biotech profile 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 discover and develop novel drug candidates and delivery approaches and 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 and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; 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 other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 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.
