Latest news with #ATTR-CM
Yahoo
20-05-2025
- Health
- Yahoo
Acoramidis Reduced Incidence of Atrial Fibrillation Events in Patients with ATTR-CM
- In a post-hoc analysis of ATTRibute-CM, acoramidis reduced the annual frequency of CVH due to AF/AFL by 43% compared to placebo and reduced the incidence of new-onset AF/AFL by 17% in the subgroup with no prior history of AF compared to placebo - In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRwt-CM patients: - In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placebo- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 - Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA® by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency and UK Medicines and Healthcare Products Regulatory Agency PALO ALTO, Calif., May 20, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a new type of biopharmaceutical company focused on genetic diseases, presented data from ATTRibute-CM, highlighting the reduced incidence of atrial fibrillation (AF) events in the overall ATTR-CM population. These data were presented in a moderated ePoster at the Annual Congress of the Heart Failure Association of the ESC (Heart Failure 2025), taking place in Belgrade, Serbia from May 17 - 20, 2025. Acoramidis is a selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. 'The reduction in new-onset atrial fibrillation and AF-related hospitalizations represents an important finding for the ATTR-CM community. I am encouraged by the growing body of data from the ATTRibute-CM study, which adds to the understanding of acoramidis and its potential impact on clinical outcomes for patients. Furthermore, the observed reductions in hospitalizations and mortality, along with improvements in functional capacity and quality of life, suggest that acoramidis may offer benefit to both variant and wild-type ATTR-CM patients who have limited treatment options,' said Kevin Alexander, M.D. of Stanford University School of Medicine, USA. 'These data support further consideration of acoramidis as a promising front-line therapy for ATTR-CM, particularly for patients with the hereditary form of the disease, who often face rapid and severe progression.' Details from the post-hoc analysis on incidence of AF in ATTRibute-CM included: Acoramidis Treatment Is Associated with a Lower Incidence of Atrial Fibrillation-related Events in Patients with ATTR-CM: A Post-hoc Analysis of the ATTRibute-CM Trial, presented by Dr. Alexander AF is a common complication of ATTR-CM, observed in up to 70% of patients, and the onset of AF is associated with an increased risk of cardiovascular-related hospitalizations (CVH) In ATTRibute-CM, a 43% relative risk reduction in the annual frequency of CVH due to AF/atrial flutter (AFL) was observed with acoramidis relative to placebo. In the subgroup who had no prior history of AF, a 17% lower incidence of new-onset AF/AFL was reported with acoramidis compared to placebo These findings show the potential of acoramidis to reduce both disease progression, as indicated by a lower incidence of new-onset AF/AFL, and CVH morbidity caused by AF/AFL, in patients with ATTR-CM In addition to the moderated ePoster, three analyses were shared on the strong clinical outcomes in ATTRv-CM versus placebo. ATTRv-CM is associated with early age of disease onset with more advanced heart failure symptoms, which often leads to a poorer prognosis than those with wild-type ATTR-CM (ATTRwt-CM). These findings included: Acoramidis Improves Serum TTR Levels in Patients with Wild-type or Variant Transthyretin Amyloid Cardiomyopathy: Results from ATTRibute-CM, presented by Anique Ducharme, M.D. of Université de Montréal, CAN In both subgroups of ATTRv-CM and ATTRwt-CM, acoramidis treatment induced a rapid increase in serum TTR levels, a measure of TTR stability, by Day 28, with comparable serum TTR levels achieved in both subgroups from Day 28 through Month 30. Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature1 Effect of Acoramidis on Functional Capacity and Quality of Life in Patients with Variant ATTR-CM: Results from ATTRibute-CM, presented by Marianna Fontana, M.D. of University College London, UK Data from ATTRibute-CM showed that when acoramidis was administered for 30 months, participants with ATTRv-CM had a clinically significant slower decline in functional capacity and quality of life compared with placebo, consistent with the overall results in both ATTRv-CM and ATTRwt-CM At Month 30, the mean difference between acoramidis and placebo treatment groups in the change from baseline in 6-minute walk distance was 86.7 meters (p = 0.0048) in favor of acoramidis and in the change from baseline in KCCQ-OS at Month 30, was 20.3 points (p = 0.0019) in favor of acoramidis, in patients with ATTRv-CM Effect of Acoramidis on All-cause Mortality, Cardiovascular Hospitalization and NT-proBNP in Variant ATTR-CM: Results from ATTRibute-CM, presented by Marianna Fontana, M.D. of University College London, UK In ATTRibute-CM, acoramidis treatment administered for 30 months led to a substantial reduction (>50%) in the composite of all-cause mortality (ACM)/CVH, ACM and CVH in participants with ATTRv-CM compared to placebo. This improvement was accompanied by favorable effects on N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels 'Based on the findings from ATTRibute-CM, we believe that acoramidis has the potential to enable patients with both variant and wild-type ATTR-CM to live longer, healthier lives, especially for those with variant ATTR-CM who typically have a poorer prognosis. We observed both a striking reduction in the frequency of cardiovascular hospitalizations (which included clinic or emergency department visits for urgent heart failure management) and a clinically important and statistically significant reduction in all-cause mortality in the important subgroup. These clinical outcomes were further mirrored in robust improvements in functional capacity, quality of life, and biomarkers of heart failure severity,' said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. 'Given these compelling results, acoramidis should be considered as first-line treatment for newly diagnosed patients, and those currently on other therapies could be switched to acoramidis to maximize their potential to achieve such benefits.' Additional acoramidis moderated ePosters at Heart Failure 2025 included: Disease Progression Among Patients Receiving Tafamidis for ATTR-CM in a Real-world Setting, presented by Daniel P. Judge, M.D. of Medical University of South Carolina, USA This analysis suggests disease progression despite treatment with tafamidis in ATTR-CM. CVH was frequent, with approximately 1 in 5 tafamidis-treated patients hospitalized in the first six months of therapy. As more therapeutic options become available, measuring the clinical effectiveness of therapies in a real-world setting will be important to help inform physicians and patients when making treatment decisions Cause of Death in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Findings from the ATTRibute-CM Study, presented by Laura Obici, M.D. of University of Pavia, ESP In the ATTRibute-CM study, total deaths were numerically lower with acoramidis compared with placebo. The relative risk reduction of 30% in cardiovascular-related mortality by Month 30 was driven predominantly by a reduction in heart failure-related deaths Time from First Recorded Clinical Manifestation to Diagnosis of Transthyretin Amyloid Cardiomyopathy: A Retrospective Cohort Study Using U.S. Claims Data, presented by Joshua Mitchell, M.D., Washington University School of Medicine in St. Louis, USA The median time from the first documented clinical manifestation to ATTR-CM diagnosis was almost 5 years, and over 2 years from the first heart failure diagnosis. This demonstrates that the patient journey to an ATTR-CM diagnosis can be prolonged and challenging, which potentially leads to more severe disease at diagnosis. Understanding the factors contributing to diagnostic delays is important to improving diagnostic pathways and patient outcomes Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency with all labels specifying near-complete stabilization of TTR. More data on the benefit of Attruby for ATTR-CM patients is planned for future medical meetings. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. About Attruby™ (acoramidis) INDICATION Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATION Adverse Reactions Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBioBridgeBio Pharma (BridgeBio; NASDAQ:BBIO) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit and follow us on LinkedIn, Twitter, Facebook, and YouTube. BridgeBio Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the 'Securities Act'), and Section 21E of the Securities Exchange Act of 1934, as amended (the 'Exchange Act'), which are usually identified by the use of words such as 'anticipates,' 'believes,' 'continues,' 'could,' 'estimates,' 'expects,' 'hopes,' 'intends,' 'may,' 'plans,' 'projects,' 'potential,' 'seeks,' 'should,' 'will,' and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements regarding the potential impact of acoramidis on clinical outcomes for patients, the possibility that acoramidis may offer benefit to both variant and wild-type ATTR-CM patients, the consideration of acoramidis as a promising front-line therapy for ATTR-CM, the potential of acoramidis to reduce disease progression and cardiovascular hospitalization morbidity caused by AF/AFL, BridgeBio's belief that acoramidis could help patients live longer, healthier lives—especially those with variant ATTR-CM—and the view that acoramidis should be considered as a first-line treatment or as a replacement for current therapies to maximize patient benefit, reflect BridgeBio's current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions it has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies as reflected in or suggested by these forward-looking statements are reasonable, it can give no assurance that such plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties, and assumptions, including, but not limited to: the risks associated with BridgeBio's dependence on third parties for development; regulatory authorities requiring additional studies or data to support the continued or expanded commercialization of acoramidis; whether data and results meet regulatory requirements or are sufficient for continued development, review, or approval; and whether other regulatory agencies agree with BridgeBio's strategies or data interpretations. These risks also include impacts from global health emergencies, such as delays in regulatory reviews and other activities, manufacturing and supply chain interruptions, adverse effects on healthcare systems, and disruption of the global economy; and the impacts of macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing inflation rates, and fluctuating interest rates on BridgeBio's operations and expectations. Additional risks are described in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio's management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in these statements. Except as required by applicable law, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@ (650)-789-8220 BridgeBio Investor Contact:Chinmay Shukla, VP Strategic Financeir@ 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
19-05-2025
- Health
- Yahoo
Early and Sustained Increase in Serum TTR Levels by Acoramidis Independently Predicted Improved Survival in the ATTRibute-CM Study
- For each 5-mg/dL increase in serum TTR level within 28 days of starting treatment, the relative risk reduction of mortality was up to 31.6% through Month 30, confirming the hypothesis that ever better levels of stabilization achieved by treatment with acoramidis, a near-complete (≥90%) TTR stabilizer, lead to ever better clinical outcomes - ATTRibute-CM is the only study to demonstrate a direct association between a prompt, sustained increase in serum TTR and survival in patients with ATTR-CM - The open-label extension data for all ATTRibute-CM participants at Month 42 showed that rapid, sustained TTR stabilization from acoramidis demonstrated statistically significant reductions in ACM and CVH (including urgent outpatient treatment for heart failure exacerbations) - In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRwt-CM patients: - In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placebo- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 - Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA® by the European Commission, the Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency PALO ALTO, Calif., May 19, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a new type of biopharmaceutical company focused on genetic diseases, published data showing that an early, sustained increase in serum transthyretin (TTR) levels predicted improved survival in ATTRibute-CM, its Phase 3 trial of acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM). These findings were published in Journal of the American College of Cardiology (JACC) in the Special Focus Issue: Amyloid. Acoramidis is a selective, small molecule, orally administered, near-complete (≥90%) TTR stabilizer. These findings further support the thesis that ever better increases in serum TTR lead to ever better clinical outcomes and that early elevations in serum TTR are an important prognostic marker to inform treatment selection. 'Patients with ATTR-CM have progressive amyloid accumulation in the heart, which when untreated manifests as progressive heart failure, arrhythmias, and eventually can result in death. Increases in serum TTR seen with acoramidis therapy within 28 days of initiation and that were sustained with therapy, were associated with a decrease in all-cause mortality independent of baseline risk among subjects in the ATTRibute-CM trial. The increase in serum TTR is hypothesized to be due to a leftward shift in amyloidogenic TTR to a more stable tetrameric TTR. This is the first concrete evidence that there is a link between this rapid increase in serum TTR and survival. Such data may inform clinical practice, as early and sustained increases in serum TTR could represent a new potential ATTR disease-specific and prognostic biomarker that may further inform clinical decisions in optimizing care for ATTR-CM patients,' said Mathew Maurer, M.D. of Columbia University Irving Medical Center. In patients with ATTR-CM, aging or an inherited variant can cause tetrameric TTR to destabilize and misfold, causing buildup of amyloid fibrils in the organs, specifically in the heart. These data demonstrate that by using acoramidis, a selective near-complete TTR stabilizer to bind serum TTR, a rapid and sustained increase in tetrameric TTR was independently associated with an improvement in overall survival, even after adjustment for known predictors like TTR variant status, baseline New York Heart Association (NYHA) functional class, baseline National Amyloidosis Centre (NAC) stage, and baseline serum TTR levels. Findings from the analysis included: Treatment with acoramidis resulted in a sharp, significant early rise in serum TTR levels (mean 9.1 mg/dL) within 28 days which was sustained throughout the 30-month treatment period For every 5-mg/dL increase in serum TTR level, the Cox proportional hazards model predicted a relative risk reduction of mortality of 26.6% and the logistic model predicted a relative reduction of 31.6% in odds of death through Month 30 An early increase in serum TTR levels on Day 28 of dosing was associated with reduced all-cause mortality (ACM) in univariate analysis, an association which persisted in multivariate analysis independent of TTR variant status, baseline NYHA functional class, baseline NAC stage, and baseline serum TTR levels Logistic modeling demonstrated that among participants treated with acoramidis, the early increase in serum TTR was associated with reduced ACM, whereas there was no prompt and sustained increase in serum TTR observed in participants treated with placebo Causal mediation analysis showed evidence that the acoramidis treatment effect on ACM probability through month 30 was fully mediated by the observed prompt and sustained increase in serum TTR 'This landmark analysis adds an incredibly important proof point to acoramidis's repository of compelling data that higher serum TTR levels are directly correlated with and mediate a reduction in mortality risk.' said Jonathan Fox, M.D., Ph.D., Chief Medical Officer of BridgeBio Cardiorenal. 'We believe that this will be an important measure for physicians treating ATTR-CM to consider and will be encouraging information for new and existing patients when reviewing options to treat their condition.' Data from 42 months sustained treatment in the open-label extension study from ATTRibute-CM showed that rapid and sustained TTR stabilization from acoramidis demonstrated statistically significant reductions in both ACM and cardiovascular-related hospitalizations (CVH, which included urgent outpatient treatment for heart failure exacerbations). The continued curve separation of the composite endpoint of ACM and CVH emphasizes the importance of early and continuous treatment resulting in early and sustained clinical benefits. These data further underscore the hypothesis that ever better levels of stabilization lead to ever better clinical outcomes and emphasizes the importance of a prognostic biomarker, serum TTR, to inform decision making for patient care. Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and UK Medicines and Healthcare Products Regulatory Agency with all labels specifying near-complete stabilization of TTR. About Attruby™ (acoramidis) INDICATION Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATION Adverse Reactions Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBio Pharma, Pharma, Inc. (BridgeBio) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit and follow us on LinkedIn, Twitter, Facebook, and YouTube. BridgeBio Forward-Looking StatementsThis press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the 'Securities Act'), and Section 21E of the Securities Exchange Act of 1934, as amended (the 'Exchange Act'), which are usually identified by the use of words such as 'anticipates,' 'believes,' 'continues,' 'could,' 'estimates,' 'expects,' 'hopes,' 'intends,' 'may,' 'plans,' 'projects,' 'potential,' 'seeks,' 'should,' 'will,' and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements regarding the potential for sustained increases in serum TTR to serve as a disease-specific and prognostic biomarker in ATTR-CM, the belief that early elevations in serum TTR are an important prognostic marker to inform treatment selection, the hypothesis that improved serum TTR stabilization is correlated with better clinical outcomes, and expectations about how these findings may guide physician decision-making and patient care, reflect BridgeBio's current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions it has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies as reflected in or suggested by these forward-looking statements are reasonable, it can give no assurance that such plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties, and assumptions, including, but not limited to: the risks associated with BridgeBio's dependence on third parties for development; regulatory authorities requiring additional studies or data to support the continued or expanded commercialization of acoramidis; whether data and results meet regulatory requirements or are sufficient for continued development, review, or approval; and whether other regulatory agencies agree with BridgeBio's strategies or data interpretations. These risks also include impacts from global health emergencies, such as delays in regulatory reviews and other activities, manufacturing and supply chain interruptions, adverse effects on healthcare systems, and disruption of the global economy; and the impacts of macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing inflation rates, and fluctuating interest rates on BridgeBio's operations and expectations. Additional risks are described in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio's management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in these statements. Except as required by applicable law, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, Executive Vice Presidentcontact@ BridgeBio Investor Contact:Chinmay Shukla, VP Strategic Financeir@
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.
Business Insider
18-05-2025
- Health
- Business Insider
Alnylam presents contemporary analysis of HELIOS-B Phase 3 study of vutrisiran
Alnylam (ALNY) Pharmaceuticals 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 events such as CV hospitalizations, and heart failure hospitalizations. Additionally, in the analysis, urgent HF visits were reduced by 46% 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% and the risk of CV mortality was reduced by 33% 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.
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.
