RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Overseas New Drugs Urgently Needed in Clinical Settings (China)

Structure/Sequence

Molecular FormulaC21H24Cl2N2O8

InChIKeyDQJDBUPLRMRBAB-WZTVWXICSA-N

CAS Registry951395-08-7

Clinical Trials associated with Tafamidis Meglumine

CTRI/2026/05/111381

/ Not yet recruitingNot Applicable

An open-labeled, balanced, randomized, two-treatment, single-period, parallel, truncated, single-dose, oral bioequivalence study of test product (T) Tafamidis Tablets 61 mg of Dr. Reddys Laboratories Limited, India comparing with reference product (R) Tafamidis tablets 61 mg of Pfizer labs, Division of Pfizer INC. NY, USA in normal, healthy, adult, human trial participants under fed condition. - Nil

Start Date09 Jun 2026

Sponsor / Collaborator

Dr. Reddy's Laboratories Ltd.

CTRI/2026/06/111647

/ Not yet recruitingNot Applicable

An open label balanced randomized two treatment two period two sequence truncated single dose crossover oral bioequivalence study of Test product T Tafamidis Tablets 61 mg of DrReddys Laboratories Limited India comparing with Reference product R Tafamidis Tablets 61 mg of Pfizer labs Division of Pfizer Inc NY USA in healthy adult human trial participants under fed condition - Nil

Start Date09 Jun 2026

Sponsor / Collaborator

Dr. Reddy's Laboratories Ltd.

CTRI/2026/05/111030

/ Not yet recruitingNot Applicable

An open-labeled, balanced, randomized, two-treatment, single-period, parallel, truncated, single-dose, oral bioequivalence study of test product (T) Tafamidis Tablets 61 mg of Dr. Reddy s Laboratories Limited, India comparing with reference product (R) Tafamidis Tablets 61 mg of Pfizer labs, Division of Pfizer Inc. NY, USA in normal, healthy, adult, human trial participants under fasting condition. - Nil

Start Date08 Jun 2026

Sponsor / Collaborator

Dr. Reddy's Laboratories Ltd.

100 Clinical Results associated with Tafamidis Meglumine

100 Translational Medicine associated with Tafamidis Meglumine

100 Patents (Medical) associated with Tafamidis Meglumine

203

Literatures (Medical) associated with Tafamidis Meglumine

01 Feb 2026·NEPHROLOGY

A Case of Amyloid Arthritis With Increased Amyloid Deposition of Both β2‐Microglobulin and Transthyretin Before Reinitiating Dialysis Long After Kidney Transplantation

Article

Author: Tsuneyoshi, Shoji ; Torisu, Kumiko ; Mito, Miki ; Tasaki, Masayoshi ; Fujino, Takeo ; Ueda, Mitsuharu ; Kitazono, Takanari ; Ueki, Kenji ; Taniguchi, Midori ; Narutomi, Fumiya ; Oda, Yoshinao ; Tateishi, Yuki ; Nakano, Toshiaki ; Yamada, Shunsuke ; Minami, Yuki

ABSTRACT:

We herein present the first reported case of a 65‐year‐old man on haemodialysis who developed amyloid arthritis involving both β2‐microglobulin and transthyretin. He had begun haemodialysis 46 years prior, and 22 years later, he developed carpal tunnel syndrome due to dialysis‐related amyloidosis. After 29 years on haemodialysis, he received a deceased‐donor kidney transplant, allowing him to discontinue dialysis. Fourteen years post‐transplant, he was diagnosed with wild‐type transthyretin cardiac amyloidosis. Seventeen years after the kidney transplant, he had to resume haemodialysis because of kidney dysfunction and presented with persistent fever. Computed tomography revealed increased amyloid deposits in the joints compared with the time of his transplant. A biopsy of the shoulder joint confirmed the presence of amyloid deposits positive for both β2‐microglobulin and transthyretin as determined by immunostaining and mass spectrometry. The patient was treated with glucocorticoids, tafamidis meglumine and β2‐microglobulin adsorption therapy, leading to improvement in his symptoms. This case highlights that amyloid deposition can progress due to kidney dysfunction, even during periods without dialysis following a kidney transplant, and underscores the need to consider transthyretin amyloid deposition in patients undergoing dialysis.

01 Jan 2026·Annales Pharmaceutiques Francaises

Optimization of UV spectrophotometric techniques for tafamidis meglumine detection in pharmaceutical formulations and biological samples: A green chemistry perspective

Article

Author: Shirkhedkar, Atul A ; Raut, Shubham R ; Khan, Md Mojeeb G ; Khan, Zamir G ; Alam, Md Shamsher ; Usman, Md Rageeb Md

OBJECTIVES:

The primary objective was to develop and validate four novel UV/visible spectrophotometric methods for the quantification of tafamidis meglumine in bulk drug, proprietary capsules, and spiked urine samples, ensuring accuracy, sensitivity, and environmental sustainability for pharmaceutical analysis.

METHODS:

Four spectrophotometric approaches were established using absorbance and area under the curve (AUC) measurements, employing both zero-order and first-order derivative techniques. Method validation followed ICH guidelines, assessing linearity, accuracy, precision, sensitivity (LOD, LOQ), and greenness. Methanol was used as a green solvent, and eco-friendliness was evaluated using AGREE and ComplexGAPI metrics.

RESULTS:

All methods exhibited excellent linearity (R²=0.9980-0.9995) over a 3-18μg/mL range. Accuracy was confirmed with recovery rates between 99.00% and 100.57%. Precision studies yielded %RSD values below 2%, indicating high reproducibility. Sensitivity was demonstrated with LOD and LOQ values from 0.27μg/mL to 2.3μg/mL. The use of methanol minimized environmental impact, and high AGREE and ComplexGAPI scores validated the methods' eco-friendly nature.

CONCLUSION:

The developed spectrophotometric methods are simple, rapid, sensitive, and environmentally sustainable for quantifying tafamidis meglumine in various matrices. These validated approaches set a new standard for green analytical chemistry in pharmaceutical quality control, ensuring both regulatory compliance and reduced environmental footprint.

01 Sep 2025·Brain and Behavior

Effects of Tafamidis Meglumine on Transient Focal Neurological Episodes and Meningeal Contrast Enhancement in Hereditary Transthyretin‐Related Meningeal Amyloidosis: Report of Two Patients Carrying the c.265T>C (p.Y89H) Variant

Article

Author: Ishiyama, Ayaka ; Takahashi, Tetsuya ; Kuroha, Yasuko ; Hasegawa, Arika ; Saito, Natsumi ; Tokutake, Takayoshi ; Wakasugi, Takahiro

ABSTRACT:

Background:

Y69H (p.Y89H) variant hereditary transthyretin (ATTRv) amyloidosis causes meningeal amyloidosis, with mutant TTR deposits localized to the leptomeninges and vitreous body.

Methods:

The effect of tafamidis meglumine on neurological disorders, such as the frequency of transient focal neurological episodes (TFNEs), magnetic resonance imaging (MRI) findings, and TTR levels in cerebrospinal fluid, was investigated in two patients diagnosed with Y69H ATTRv mutation.

Results:

The initial symptoms in both patients were TFNEs, such as aphasia, sensory disturbance, motor paralysis, ataxia, and drop attacks. Neither epileptic drugs nor antiplatelet therapy decreased the frequency of attacks. The patients exhibited diffuse leptomeningeal enhancement on brain and spinal MRI. Tafamidis meglumine was initiated at a dose of 20 mg/day and was found to be partially effective. The TFNEs nearly resolved, and meningeal enhancement on brain MRI improved; however, the neurological deficits progressed over the following 2 years.

Conclusions:

Tafamidis had a partial effect on TFNEs and meningeal contrast enhancement on MRI; however, cerebellar ataxia and cognitive decline continued to progress.

198

News (Medical) associated with Tafamidis Meglumine

23 Sep 2026

·www.globenewswire.com

Acoramidis Demonstrates Real-World Benefit Versus Tafamidis in ATTR-CM, Reducing Risk of Clinical Worsening

- In the first peer-reviewed, real-world, contemporary, comparative effectiveness study of TTR stabilizers in ATTR-CM using U.S. claims data, acoramidis was associated with a statistically and clinically significant 34% reduction in a composite of clinical worsening events (diuretic intensification, heart failure hospitalization, and mortality) versus tafamidis (p=0.046), supporting improved clinical stability of acoramidis over tafamidis - Acoramidis was also associated with a significant 43% reduction in risk of diuretic intensification versus tafamidis (p=0.021); diuretic intensification is an early marker of worsening heart failure and worse clinical outcomes - Separation in clinical outcomes emerged within weeks of treatment initiation and increased over time, reinforcing a consistent early treatment effect and durable clinical stabilization - Findings underscore acoramidis’s differentiated profile, with potential to inform frontline treatment decisions and support switching individuals to acoramidis - An additional, independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data is expected to be released at an upcoming 2026 medical meeting PALO ALTO, Calif., Sept. 23, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, today announced results published in Cardiology and Therapy from the first peer-reviewed, contemporary real-world comparative effectiveness retrospective study of transthyretin (TTR) stabilizers in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM), demonstrating that acoramidis was associated with significantly lower risk of early clinical worsening compared to tafamidis. This represents the first peer-reviewed, real-world evidence differentiating clinical outcomes between approved TTR stabilizers, reinforcing acoramidis’s differentiated clinical profile in present-day practice. “These findings further position acoramidis as a differentiated TTR stabilizer in contemporary clinical practice,” said Richard Wright, M.D., M.A.C.C. of the Pacific Heart Institute, U.S. “The significant reduction in diuretic intensification, an early indicator of worsening heart failure, points to improved disease control and clinical stability. The progressive nature of ATTR-CM is evident in all Phase 3 trials and is seen again here. This demonstrates the need for clinicians to proactively mitigate disease progression, and these data have the potential to meaningfully inform treatment selection in newly diagnosed patients and in patients currently on other treatments.” In this analysis of newly treated individuals living with ATTR-CM, acoramidis showed early, consistent, and clinically meaningful advantages compared to tafamidis across key measures of disease progression during a mean follow-up of 4.6 months. The key findings from the study comparing acoramidis benefit versus tafamidis in individuals with ATTR-CM: 34% statistically and clinically significant reduction in risk of composite clinical worsening (HR 0.66; p=0.046), including diuretic intensification, heart failure hospitalization, and all-cause mortality43% reduction in risk of diuretic intensification (HR 0.57; p=0.021), an established early marker of worsening heart failure and predictor of hospitalization and mortality; diuretic intensification was rigorously defined as initiation or dose-equivalent escalation of oral loop diuretics, parenteral loop diuretics use, or addition of a thiazide-type diuretic48% reduction in initiation or dose-equivalent escalation of oral loop diuretics (HR=0.52; p=0.014)Rapid benefit, with separation of Kaplan-Meier curves observed within weeks of treatment initiation and increasing over timeFewer individuals with acoramidis initiated alternative ATTR-CM therapy as compared to those treated with tafamidis (4.6% vs 7.7%, respectively), consistent with findings of improved clinical stability with acoramidisBackground heart failure therapies in this study were well balanced across arms and reflect contemporary practice: 43-44% SGLT2i, 36-39% MRAMultiple falsification analyses testing for residual bias were nonsignificant The study leveraged a retrospective, longitudinal, new-user, active-comparator design using U.S. claims data and incorporated a unique dataset linking Komodo Healthcare Map U.S. claims with Claritas hub and specialty pharmacy data. After weighting, the analysis included 170 individuals with acoramidis and 448 individuals with tafamidis newly initiating treatment between Dec 2024-April 2025 and followed through July 2025, with a mean follow-up of approximately 4.6 months. Acoramidis is approved as Attruby® by the U.S. FDA and is approved as BEYONTTRA® by the European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency, Swissmedic, the Swiss Agency for Therapeutic Products, the UK Medicines and Healthcare Products Regulatory Agency, and the Brazilian Health Regulatory Agency (ANVISA) with all labels specifying near-complete stabilization of TTR. Additional data on the real-world benefit of acoramidis versus tafamidis in individuals living with ATTR-CM is planned for fall medical meetings and scientific publications, including an independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data, expected to be released at an upcoming 2026 medical meeting. As with all real-world evidence studies, these findings should be interpreted in the context of inherent limitations of administrative claims data, including limited capture of cardiac biomarkers and other clinical measures of disease severity. Follow-up was limited given the recent approval of acoramidis, and longer-term data are needed to further add to these findings. About Attruby® (acoramidis)INDICATIONAttruby 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 INFORMATIONAdverse ReactionsDiarrhea (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 exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit bridgebio.com and follow us on LinkedIn, X, Facebook, Instagram, YouTube, and TikTok. 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,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “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 include statements regarding the potential clinical significance and therapeutic implications of the real-world data regarding acoramidis, including the potential for the findings to inform frontline treatment decisions and treatment selection for newly diagnosed patients and patients currently receiving other treatments, and to support switching individuals to acoramidis; and BridgeBio’s plans and expectations regarding the presentation and publication of additional real-world data regarding acoramidis, including an independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data expected to be released at an upcoming 2026 medical meeting. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the 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 risk that results from retrospective, observational, real-world evidence studies, including studies based on administrative claims data, may be subject to confounding, selection bias, residual bias, incomplete or inaccurate data, limited follow-up or other limitations and may not be predictive of future clinical outcomes or treatment effects; that the observed associations and differences between acoramidis and tafamidis may not be replicated in additional analyses, independent studies or longer-term data or may not translate into improved long-term clinical outcomes; that the findings may not meaningfully inform treatment selection or support switching patients to acoramidis; that additional real-world studies, including the independent study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data, may yield results that differ from or do not confirm the findings described in this press release; that plans or timing for future medical meeting presentations or scientific publications may change; the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Middle East, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company 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 the Company’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 the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact:Kaitlyn Reilly, Director, Communicationscontact@bridgebio.com (650) 789-8220 BridgeBio Investor Contact:Kristen Kelleher, Director, Investor Relationsir@bridgebio.com

Drug ApprovalClinical ResultAHAPhase 3

20 Sep 2026

·www.biospace.com

HFSA’s 2026 Annual Scientific Meeting to Feature Late-Breaking Trials That Could Reshape Heart Failure Treatment

October 9-12, 2026 | Phoenix, Arizona -- New data spans pharmacotherapy, imaging and diagnostics, and device therapy for patients with cardiomyopathy WASHINGTON , Sept. 18, 2026 / PRNewswire / -- The Heart Failure Society of America ( HFSA ) today announced the Late-Breaking Clinical Research slate for its Annual Scientific Meeting ( ASM ), October 9-12, 2026, at the Phoenix Convention Center in Phoenix, Arizona. This year's late-breaking program spans pharmacotherapy, imaging and diagnostics, and device-based therapy - including new data on whether a novel oral therapy could establish an additional treatment pillar for both HFrEF and HFpEF , a first-of-its-kind imaging approach designed to detect cardiac amyloidosis without an invasive biopsy, and real-world outcomes from established device therapies. View all late-breaking science below and online . These trials ... represent some of the most anticipated data in ... medicine for patients with cardiomyopathy this year. "This year's Late-Breaking Science program reflects the pace of innovation transforming heart failure care," said Jennifer Thibodeau , MD, MSCS , 2026 HFSA Annual Scientific Meeting Program Committee Chair. "These trials showcase advances in pharmacotherapy, imaging and diagnostics, as well as device therapy, and represent some of the most anticipated data in cardiovascular medicine for patients with cardiomyopathy this year." Late-breaking clinical research to be featured during two plenary sessions includes results and updates from these major randomized trials: Sunday, October 11 Plenary Session | 9:00 AM – 10:30 AM Late-Breaking Evidence from Novel Therapies to Real-World Outcomes Cycle-1 REF A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Assess the Safety and Effectiveness of Tovinontrine in Patients With Chronic Heart Failure With Reduced Ejection Fraction: The Cycle-1 REF Trial Cycle-2 PEF A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Clinical Study to Assess the Safety and Effectiveness of Tovinontrine in Patients With Chronic Heart Failure With Preserved Ejection Fraction: The Cycle-2 PEF Trial CARDIO- TTRansform Effect of Eplontersen on Health Status in Patients with Transthyretin Amyloid Cardiomyopathy Enrolled in the CARDIO- TTRansform Trial 124I- Evuzamitide Amyloid-targeted PET/CT Scan: Potential for Detection of Early Cardiac Amyloidosis in a Prospective Multicenter Phase 3 Study Cohort Cardiac Contractility Modulation Therapy One-Year Real-World Clinical Outcomes Following Cardiac Contractility Modulation Therapy from the U.S. Post-Approval Study REBALANCE The REBALANCE Registry: Real World Outcomes Following Baroreflex Activation Therapy Monday, October 12 Plenary Session | 9:00 AM – 10:30 AM Late-Breaking Evidence from Novel Therapies to Real-World Outcomes ACACIA- HCM Global Efficacy Of Aficamten In Nonobstructive Hypertrophic Cardiomyopathy: Results From ACACIA- HCM Aficamten Improves Comprehensive Measures of Exercise Performance in Non-Obstructive Hypertrophic Cardiomyopathy: A Prespecified Analysis of ACACIA- HCM POSEIDON Prevalence of Cardiovascular Inflammation and Associated Patient Characteristics and Biomarker Profiles Across the Heart Failure Spectrum in the United States: Results From the POSEIDON Study C-MIC III & C-MIC II Effects of Cardiac Microcurrent Therapy on Exercise Capacity: Independent Core Laboratory CPET Analysis of the C-MIC II Trial Six-Month Outcomes of Subcutaneous Cardiac Microcurrent Therapy: Comparative Analysis of the C-MIC III Pilot Study with the C-MIC II Randomized Trial Late-breaking clinical research showcases novel results of major randomized trials and rapidly evolving research. Due to the exceptional volume and quality of research submitted this year, HFSA will also feature late-breaking research through Rapid Fire Oral Sessions. These Rapid Fire sessions are fast-paced oral presentations spotlighting key data from late breaking clinical research. Sessions take place on Sunday and Monday with Q&A and new topics each day. Sunday, October 11 | Rapid Fire 1 | 10:45 AM - 11:45 AM Ratio of Serum Transthyretin to NT- proBNP is a Novel Prognostic Measure for Transthyretin Amyloid Cardiomyopathy: Insights From ATTRibute -CM Colchicine In Patients With Heart Failure And Preserved Ejection Fraction: A Crossover Trial The ReDS -SAFE HF II: Remote Dielectric Sensing-Guided Decongestion Strategy In Patients Hospitalized For Heart Failure. A Trial Design Early eGFR Decline Following Acoramidis Initiation is Associated With Reduced Risk of Recurrent Cardiovascular Hospitalizations and Mortality Gator SCALES- WHF : Sub-Cutaneous Administration of Lasix to Eliminate Symptoms of Worsening Heart Failure Real World Comparative Effectiveness Study of Acoramidis Versus Tafamidis in Transthyretin Amyloid Cardiomyopathy Using the Epic COSMOS Data Platform Dosing And Safety Of Aficamten For Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy In The ACACIA- HCM Trial Sunday, October 11 | Rapid Fire 2 | 2:30 PM - 3:30 PM Ambulatory Upright Pulmonary Artery Pressure and Risk of HF Hospitalization and Death in Patients with Chronic Heart Failure Hyperbaric Oxygen Therapy and EECP as Adjuvant Therapy in Heart Failure : A Prospective Interventional Study Baroreflex Activation Therapy in Heart Failure with Mildly Reduced Ejection Fraction: First Real-World Evidence of Reduced Hospitalization Burden Cost-Effectiveness Of ReDS -Guided Heart Failure Management Pulmonary Artery Pressure Response to Loop and Thiazide Diuretic Intensification in Heart Failure: Impact of Diuretic Class and Patient Characteristics Ambient Home Monitoring of Peripheral Oedema Significantly Reduces Heart Failure Events: Final Results from the Randomized HF-TRACK Trial Therapeutic Sequencing Is Projected to Improve Quality-Adjusted Survival in Heart Transplant Candidates with Advanced Heart Failure: A Risk-Selected Left Ventricular Assist Device-First Strategy Sunday, October 11 | Rapid Fire 3 | 5:00 PM - 6:00 PM Impact of Eplontersen on Cardiac Biomarkers in Participants with Transthyretin Amyloid Cardiomyopathy in the CARDIO- TTRansform Phase III Trial Weight Loss Surgery Impact on Comprehensive Metabolic Responses to Exercise in Heart Failure with Preserved Ejection Fraction Primary Results Trajectories of Dyspnea After Heart Failure Hospitalization: A Prospective Longitudinal Cohort Study Achieving Near Target-Dose Quadruple Guideline-Directed Medical Therapy Before Hospital Discharge After Acute Heart Failure: The STRONG-HF Japan Study Association of Quadruple Therapy Achievement and Biochemically Verified Medication Adherence with Outcomes in HFrEF : Insights from the POLY-HF Trial The Impact Of An Adaptive Patient-Centered Web Application on Medication Optimization In Patients With HFrEF : A Multicenter Randomized Trial Monday, October 12 | Rapid Fire 4 | 12:15 PM – 1:15 PM TAILORD Electronic Intervention to Improve Therapy in a Diverse Cohort of Patients with Heart Failure Post-ARIES Antithrombotic Implementation and Safety Evaluation in HeartMate 3 (PRAISE HM-3) EDG -15400, A Cardiac Sarcomere Modulator, Demonstrated Favorable Pharmacokinetics , Pharmacodynamics , and Tolerability in Healthy Adult Participants Rapid Non-invasive Sensors-Derived Assessment of Left Ventricular Filling Pressure in Patients Presenting With Acute Dyspnea Three-Year Results of PROACTIVE-HF Trial Stratified by Sex The meeting kicks off in just three weeks. A full schedule-at-a-glance is available online , as well as information on the science and research available at the meeting. Learn more about the meeting and register at hfsa .org/ asm 2026 . Media Registration & Embargo Policy Credentialed and accredited media may register for complimentary press access to HFSA ASM 2026 by contacting Laura Poko at lpoko @ hfsa .org. As part of HFSA's 24-Hour Press Access Window, credentialed and accredited media will receive embargoed access to press materials beginning 24 hours before each embargo lifts. During this period, media may review embargoed press releases, abstracts, and presenter details via a secure, password-protected folder, and schedule pre -embargo interviews with late-breaking science presenters. All interviews and materials remain strictly embargoed until the time of presentation listed above. Follow # HFSA 2026 for real-time coverage from the meeting. About the Heart Failure Society of America The Heart Failure Society of America, Inc. ( HFSA ) represents the first organized effort by heart failure experts from the Americas to provide a forum for all those interested in heart function, heart failure, and congestive heart failure ( CHF ) research and patient care. The mission of HFSA is to provide a platform to improve and expand heart failure care through collaboration, education, innovation, research, and advocacy. HFSA members include physicians, scientists, nurses, nurse practitioners, pharmacists, trainees, other healthcare workers and patients. For more information, visit hfsa .org . Media Contact : Laura Poko , 301-798-4493, ext. 226, lpoko @ hfsa .org View original content to download multimedia: SOURCE Heart Failure Society of America

Phase 3Phase 2

31 Aug 2026

·www.fiercebiotech.com

ESC: Alnylam's next-gen silencer sees silver linings after fallout of AZ, Ionis trial in ATTR-CM

As AstraZeneca appears to close the book on a combo approach following the phase 3 failure of its Ionis-partnered Wainua, several analysts contend it’s too early to count out Alnylam’s silencing drugs in ATTR-CM patients also taking standard-of-care stabilizers. Meanwhile, Alnylam itself has stepped up with new data in defense of its approved cardiomyopathy med Amvuttra, wading into the emerging monotherapy vs. silencer-plus-stabilizer debate. That line of questioning was sparked in early July on the topline fumble of AZ and Ionis’ CARDIO-TTRansform trial. The trial’s surprise results went on to become one of the most anticipated presentations at the European Society of Cardiology Congress in Munich this past weekend. The issue on doctors' and industry watchers’ minds wasn’t just what the results mean for AZ and Ionis’ antisense oligonucleotide Wainua, but the follow-on implications for the silencing approach more generally in a heavily stabilizer-dependent patient population. Wainua (eplontersen), Alnylam’s approved Amvuttra (vutrisiran) and the late-stage RNA interference asset nucresiran are all considered silencers. Signals earlier this summer suggested that Wainua might have functioned better on its own, whereas more than half of patients—exceeding 80% by the study’s end—in CARDIO-TTRansform were also on a stabilizer drug, a class that includes Pfizer’s juggernaut tafamidis franchise. That common use of stabilizers is not an aberration among patients with transthyretin amyloid cardiomyopathy (ATTR-CM), in which misfolded TTR deposits form in the heart muscle, impairing cardiac function and worsening symptoms of heart failure and recurrent cardiovascular events. AZ seems to think that layering such treatments on top of one another is a dead end. CARDIO-TTRansform was designed to answer whether ATTR-CM patients might benefit from the addition of a second mechanism, “[a]nd I think what we’ve realized through this is unfortunately, there isn’t,” Mina Makar, AZ’s SVP of global cardiovascular, renal and metabolism, said in an interview ahead of the company’s ESC presentation. But to hear Alnylam’s R&D chief Pushkal Garg, M.D., tell it, the Wainua study and Amvuttra’s positive Helios-B trial “reinforce that not all TTR silencers or clinical trial designs are the same.” Garg espoused continued confidence in both Amvuttra and nucresiran for ATTR-CM in an emailed statement to Fierce. With new evidence from ESC in hand, several analysts agreed Alnylam’s next-gen siRNA prospect nucresiran could still succeed in its own ongoing phase 3 test, TRITON-CM . “There is a case that TRITON-CM could be in okay shape to still succeed,” analysts from Stifel wrote in an Aug. 30 note to investors, if Alnylam is focusing more on patients with earlier stages of ATTR-CM. Wainua did beat placebo in patients with stage 1 disease in the CARDIO-TTRansform trial, the analysts noted, even though about 60% of those patients were also taking Pfizer's stabilizer drug Vyndamax (tafamidis). “This is a notable data point and suggests that there was probably benefit on top of tafamidis for eplontersen in the right patients who are milder,” the analysts wrote. “Which would make sense given the progressive nature of TTR-CM.” Garg also highlighted the nominal benefit observed in NAC stage 1 patients in CARDIO-TTRansform, which appeared to have enrolled “a substantially higher proportion of patients with more advanced disease” compared with Amvuttra’s Helios-B study. That difference, he said, “underscores the importance of understanding which patients are most likely to benefit from TTR silencing.” Unlike transthyretin stabilizers, which bind to the protein and prevent it from breaking apart into its toxic form, silencers like Wainua, Amvuttra and nucresiran operate upstream to destroy the mRNA blueprint used to make transthyretin in the first place. Wainua’s antisense approach is also different from Amvuttra and nucresiran’s RNA-interference method in their enzymatic mechanisms and mRNA-targeting efficiency. Nucresiran’s chances are also bolstered by its more thorough knockdown of transthyretin, analysts from Citi pointed out in a note Monday. Nucresiran reduced the protein’s levels by more than 92% in phase 1, surpassing Wainua’s 70% and Amvuttra’s 81% marks. Alnylam currently anticipates a 95% or greater knockdown for the newer silencer, according to its R&D chief. “In view of full CARDIO-TTRansform results at ESC, we believe the outlook for Alnylam’s TTR franchise looks incrementally brighter on both fronts,” the Citi analysts wrote, “with eplontersen more conclusively emerging as weaker on TTR knockdown and CARDIO-TTRansform potentially hurt by enrolling more advanced disease.” Analysts from Evercore ISI agreed, adding in an Aug. 30 note that experts in the field are “receptive to” the growing idea that RNA interference approaches like Amvuttra and nucresiran are “meaningfully differentiated” from an antisense oligonucleotide. “Data and physician feedback this weekend do indeed seem to lend support to a more credible path to TRITON success,” they wrote. Overall, given those differences between the drugs and their studies, “we don’t see the silencer vs stabilizer clinical debate undermined post ESC in a way that would challenge” Alnylam’s current growth outlook, the Citi team suggested. Still, the mood was markedly more diminished over at Jefferies, where analysts wrote Sunday that a few “incremental positives” for Amvuttra and nucresiran can’t help the fact that the value proposition of silencers has been “somewhat diminished,” and that the probability of the TRITON-CM study’s success remains “in question.” To the Jefferies team, silencers simply “don’t add much CV benefit to stabilizers” in light of the data. Analysts there argued that a lot hinges now on the belief that TTR knockdown depth matters, both in terms of Amvuttra’s market positioning and the odds of TRITON-CM serving up a win. Addressing that point directly, Garg stressed to Fierce that at Alnylam, “we believe that the degree of TTR reduction achieved by a silencer matters,” describing the difference in knockdown between Helios-B and CARDIO-TTRansform as “clinically meaningful and an important factor in understanding differences in results across these trials.” Meanwhile, all-cause mortality (ACM) results in patients on background stabilizers in both companies’ trials do point “to a real benefit,” the Jefferies team wrote, and a TRITON-CM endpoint inclusive of ACM could bolster the study’s chances. Still, the overall risk around the trial “remains hard to handicap,” they cautioned. On the topic of the trial plan, Alnylam’s Garg said that “[w]e remain confident in TRITON-CM as currently designed,” while adding that his company will “continue to evaluate the CARDIO-TTRansform data to assess whether any changes are warranted.” He clarified that the study’s design already reflects goals around “deeper TTR knockdown, an event-driven design focused on all-cause mortality and enrichment of enrollment” to find patients most likely to benefit. He also agreed that, despite the CARDIO-TTRansform miss in the overall population on the primary cardiovascular mortality endpoint, ACM “appeared to favor eplontersen as was the case for Amvuttra in Helios-B.” “We think these findings highlight the complexity of measuring outcomes in a heterogeneous, multisystemic disease like ATTR-CM,” Garg explained. As for Alnylam’s marketed med Amvuttra (vutrisiran), approved in ATTR-CM patients last March, the company itself used the ESC Congress to trumpet a subgroup analysis in its pivotal Helios-B trial focused on patients according to baseline use of Pfizer’s tafamidis. In the results, which also went live in the Journal of the American College of Cardiology Sunday, Amvuttra’s performance on the primary composite endpoint of all-cause mortality and recurrent cardiovascular events “was consistent irrespective of baseline tafamidis use,” according to Alnylam, which said suggests “clinical benefits across broad patient populations, including those receiving stabilizers.” That said, the company did caveat that its Helios-B trial, which helped earn Amvuttra its cardiomyopathy nod, was not powered to weigh the benefit of the drug specifically in patients receiving background tafamidis from the start. On the commercial front, Jefferies analysts said they believe the CARDIO-TTRansform results have “cast a cloud over the role of silencers in stabilizer-controlled patients,” noting this could challenge share gains enjoyed by silencers. “[I]t’s unclear to us how strong the Amvuttra-stabilizer argument will be to physicians,” they added. Alnylam appears to contest that thinking, too. Across the company’s ESC data, Garg said, “vutrisiran, unlike eplontersen, delivered consistent efficacy results across all sub-groups in the study, including in patients on background stabilizers, as well as an independent meta-analysis that showed that vutrisiran was associated with better clinical outcomes than eplontersen across every endpoint evaluated.”

Phase 3Clinical ResultPhase 1Oligonucleotide

100 Deals associated with Tafamidis Meglumine

External Link

KEGG	Wiki	ATC	Drug Bank
D09674	Tafamidis Meglumine	N07XX08	DB11644

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Amyloid Neuropathies	China	Pfizer Europe MA EEIG	05 Feb 2020
Cardiomyopathies	United States	FoldRx Pharmaceuticals LLC	03 May 2019
Transthyretin Amyloid Cardiomyopathy	South Korea	Pfizer Pharmaceuticals Korea Ltd.	28 Apr 2015
Amyloid Neuropathies, Familial	Japan	Pfizer Japan, Inc.	20 Sep 2013
Amyloidosis, Hereditary, Transthyretin-Related	European Union	Pfizer Europe MA EEIG	16 Nov 2011
Amyloidosis, Hereditary, Transthyretin-Related	Iceland	Pfizer Europe MA EEIG	16 Nov 2011
Amyloidosis, Hereditary, Transthyretin-Related	Liechtenstein	Pfizer Europe MA EEIG	16 Nov 2011
Amyloidosis, Hereditary, Transthyretin-Related	Norway	Pfizer Europe MA EEIG	16 Nov 2011

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Poikiloderma With Neutropenia	Phase 3	Argentina	Pfizer Inc.	01 Jul 2008
Poikiloderma With Neutropenia	Phase 3	Brazil	Pfizer Inc.	01 Jul 2008
Poikiloderma With Neutropenia	Phase 3	France	Pfizer Inc.	01 Jul 2008
Poikiloderma With Neutropenia	Phase 3	Germany	Pfizer Inc.	01 Jul 2008
Poikiloderma With Neutropenia	Phase 3	Portugal	Pfizer Inc.	01 Jul 2008
Poikiloderma With Neutropenia	Phase 3	Sweden	Pfizer Inc.	01 Jul 2008

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	158	Tafamidis therapy	jtihqirqdj(vneeerdjmp) = lmrwnqtiny mifcbsgokj (sjxihefxmd ) View more	Positive	30 Aug 2026
ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	2,494	Vutrisiran	tclpscxhnp(muwfzqbstq) = nflnhsvomd yvrzumjitt (puvpzrczhz, 0.24 - 0.82) View more	Positive	30 Aug 2026
				Tafamidis	kelxiovbfd(jguwrqubji) = xkedpteaji unsqpuhpig (dauckbqbkj, 0.43 - 0.82) View more
ESC2026	Not Applicable	Senile cardiac amyloidosis NT-proBNP \| troponin T	40	Tafamidis	repaxdrzae(rdbgyuhshj) = baviecadjg awedsndqeq (wqtvnqxzor, 68.9 - 119.6) View more	Positive	29 Aug 2026
ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	163	Tafamidis	manlnvyiiv(mdxcfkxrss) = ypnapnwahq gniqfcqpbj (uqivoflubk ) View more	Positive	28 Aug 2026
SWISS-CARE (ESC2026)	Not Applicable	Transthyretin Amyloid Cardiomyopathy	452	Tafamidis 61 mg daily	zcpcajqbjx(fjyxlurqws) = Patients who experienced MACE were significantly older, had a higher NYHA class, increased NT-proBNP, reduced renal function and LVEF (all p<0.001). Adjusted multivariable analysis identified independent MACE predictors: age (years) (HR 1.31, 95% CI: 1.03-1.65, p<0.05), arterial hypertension (HR 1.61, 95% CI: 1.07-2.41, p<0.05), NT-proBNP (pg/mL) (HR 1.31, 95% CI: 1.03-1.68, p<0.05), and moderate-to-severe tricuspid valve disease (HR 1.61, 95% CI: 1.08-2.41, p<0.05). Higher LVEF was independently associated with lower risk (HR 0.81, 95% CI: 0.67-0.98, p<0.05). fgjrlsmqro (kmtsnzgicf ) View more	Positive	28 Aug 2026
ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	45	Tafamidis therapy	wxqljzgpzj(smkbcbvjpo) = cpotcymaft ldpstquvob (ifyapgbkva ) View more	Positive	11 May 2026
ATTR-ACT (ESC2026)	Phase 3	Transthyretin Amyloid Cardiomyopathy	264	Tafamidis	kqjbeedowh(qvacdaaiqe) = hbwluelqpv gsuslsyakv (jozighpatj ) View more	Positive	11 May 2026
				Tafamidis + Tafamidis	kqjbeedowh(qvacdaaiqe) = xdvcrvichk gsuslsyakv (jozighpatj ) View more
ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	278	Tafamidis 61mg	jmpmrfwsmp(hnnwfetwbn) = rzbbyxyrsv nnkvxarxft (pjmebowgjo ) View more	Positive	11 May 2026
				stand-alone HF treatment	mvbrkhvstn(zzrfcfoexq) = uzzlufpumd slznelyjmk (jycczjyufz ) View more
ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	207	Tafamidis	dezhwycgrt(aemqyjfyqx) = tafamidis vs acoramidis: 27.5 vs 43.8, p<0.0001 gwhmyqowyq (aaknkjqxao ) View more	Positive	11 May 2026
				Acoramidis
ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	180	Tafamidis 61mg id	bzcjfxkabq(rlczcngdrf) = The acoramidis cohort had a 100% event-free survival, with no new reported adverse events, although interpretation is limited by the short follow-up. wrdobjusat (vowfcowhwn )	Positive	11 May 2026
				Acoramidis 712mg bid

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Hiro LS Researcher

The AI Search Engine Built to Accelerate Biopharma Decisions

Search across billion-scale life sciences data to uncover signals, validate evidence, and act with confidence.

Ask any biopharma research question→

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, Patsnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Discover Synapse Data Servers

Synapse data is now integrated into the PatSnap LS Model Context Protocol (MCP) service. Customize your LLM agent now using our MCP server!

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis