A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)

Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age.
Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.
This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.

Start Date12 May 2025

Sponsor / Collaborator

Eidos Therapeutics, Inc.

ISRCTN11628974

/ CompletedPhase 1

A multiple dose study to evaluate the pharmacokinetics of an acoramidis (AG10) modified release tablet formulation in healthy subjects

Start Date30 Aug 2022

Sponsor / Collaborator

Eidos Therapeutics, Inc.

NCT04988386

/ Active, not recruitingPhase 3

An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Start Date27 Oct 2021

Sponsor / Collaborator

Eidos Therapeutics, Inc.

100 Clinical Results associated with Acoramidis

100 Translational Medicine associated with Acoramidis

100 Patents (Medical) associated with Acoramidis

Literatures (Medical) associated with Acoramidis

01 Dec 2025·Current heart failure reports

Etiological Treatment of Cardiac Amyloidosis: Standard of Care and Future Directions

Review

Author: Morfino, Paolo ; Emdin, Michele ; Vergaro, Giuseppe ; Chubuchna, Olena ; Aimo, Alberto ; Castiglione, Vincenzo ; Buda, Gabriele ; Ferrari Chen, Yu Fu ; Fabiani, Iacopo

Abstract:

Purpose of Review:

Cardiac amyloidosis (CA) is a condition caused by interstitial infiltration of misfolded proteins structured into amyloid fibrils. Transthyretin (ATTR) and immunoglobulin light chain (AL) amyloidosis represent the most common forms of CA. CA was traditionally perceived as a rare and incurable disease, but diagnostic and therapeutic advances have undermined the conventional paradigm.

Recent Findings:

The standard of care for ATTR-CA include agents capable of selectively stabilizing the precursor protein (e.g., tafamidis), whereas the plasma cell clone is the main target of chemotherapy for AL-CA. For long, tafamidis represented the only drug approved for patients with ATTR-CA. Recent data from ATTRibute-CM led to the approval of acoramidis, whereas patisiran received refusal based on the APOLLO-B trial. Novel CRISPR-Cas9-based drugs (i.e., NTLA-2001) hold great potential in the setting of ATTR-CA. Several hematological regimens are available to treat AL-CA. The main limit of current therapies is their inability to trigger removal of amyloid from tissues. However, the investigation of monoclonal antibodies targeting misfolded ATTR (e.g., PRX004, NI301A) or AL (e.g., birtamimab, anselamimab) has led to encouraging results.

Summary:

Various cutting-edge strategies are being tested for treatment of CA and may change the prognostic landscape of this condition in the next years.

01 Dec 2025·MOLECULAR BIOLOGY REPORTS

Transthyretin as a therapeutic target: the future of disease-modifying therapies for Alzheimer’s disease

Review

Author: Shaw, Swetaleena ; Aran, Khadga Raj ; Porel, Pratyush

BACKGROUND:

Alzheimer's disease (AD) is the most common neurodegenerative disease for causing memory deficits and primarily characterized by extracellular deposition of amyloid-β (Aβ) plaques, intracellular neurofibrillary tangles (NFTs), and hyperphosphorylation of tau protein, all are pathological hallmarks for AD. Transthyretin (TTR) is a highly conserved homo-tetrameric protein, primarily synthesized in liver and choroid plexus, and most importantly involved in transport of T₃-T₄ hormones and retinol.

OBJECTIVES:

This review explores the dual role of TTR, with a greater emphasis on its neuroprotective action, particularly in AD.

METHODS:

Based on the available literature, TTR's potential as a biomarker in the central nervous system (CNS), focusing its role in stabilizing Aβ aggregation and the senile plaque formation during neurodegeneration. Additionally, TTR's dual roles, in neurodegeneration and neuroprotection are studied, emphasizing its potential for improving AD diagnosis and treatment strategies.

RESULTS:

Recent research has revealed that TTR is gradually showcasing its promise in neuroprotection and neuronal viability in AD by binding with Aβ and mitigating its neurotoxic effects. Current preclinical and clinical studies also support that TTR is actively involved in maintaining the blood-brain barrier (BBB) integrity and maintain neurotransmitter balance, all of which offer significant therapeutic promise through TTR stabilizers, such as Tafamidis, Acoramidis, and Vutrisiran, highlighting their potential in AD treatment CONCLUSION: This review concludes that TTR plays bidirectional role and gaining interest as a potential biomarker, though several challenges must be addressed before it can be established a novel therapeutic target in AD management in the modern era of drug discovery.

01 Jun 2025·DRUGS

Acoramidis: First Approval

Review

Author: Lee, Arnold

Disassociation of transthyretin (TTR) has been identified as a key step in the pathology of TTR amyloid cardiomyopathy, which is characterised by the presence of amyloid fibrils in cardiac tissue. Acoramidis (ATTRUBY™) is a small molecule TTR stabiliser being developed by BridgeBio Pharma, Inc. and is the first drug to demonstrate near-complete (> 90%) stabilisation of TTR. This article summarizes the milestones in the development of acoramidis leading to its first approval in the USA for the treatment of the cardiomyopathy of wild-type or variant TTR-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization. In the EU, a positive opinion has been adopted for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy.

171

News (Medical) associated with Acoramidis

01 Aug 2025

·www.biospace.com

‘Very Impressive’ Amvuttra ATTR-CM Sales Send Alnylam Soaring

iStock, Deagreez In its first commercial quarter for ATTR-cardiomyopathy, Alnylam’s Amvuttra reached roughly 1,400 patients and made more than $490 million. In its first commercial quarter in the ATTR-CM market, Alnylam’s siRNA therapy Amvuttra carried the biotech’s revenues to a comfortable beat over consensus estimates and gave the company enough confidence to substantially raise its full-year 2025 guidance. Amvuttra made $491.9 million in the quarter, a 112% increase from the same period last year. Those sales are from both of its indications: polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTR), for which Amvuttra was initially approved in 2022 , and ATTR-cardiomyopathy (CM), its newest indication that was granted in March . Some 1,400 ATTR-CM patients were on Amvuttra at the end of its first quarter of availability for the condition, according to Alnylam’s second-quarter earnings report on Thursday. Writing to investors on Thursday, analysts at Stifel called Amvuttra’s Q2 performance “very impressive,” noting that its total revenues deliver a “significant beat versus Street expectations” of $342 million. Investors also seemed impressed by Alnylam’s execution of Amvuttra’s ATTR-CM launch. The company was up more than 15% at market close. “Even in the backdrop of high Street expectations, Amvuttra performance’s here clearly aligns with the bull case and makes us more optimistic on peak sales potential,” Stifel added. BMO Capital Markets wrote in their own note late Thursday that Amvuttra’s sales “reflect strong patient demand,” given a growing prescriber base, robust uptake without price adjustments and apparent penetration of the drug into first and second line settings.Given Amvuttra’s strong launch, Alnylam on Thursday lifted its full-year sales guidance from $2.05 billion to $2.25 billion, as previously forecasted, to $2.65 billion to $2.8 billion, a 27% increase. The company also now expects the growth of its net product sales to hit 59% to 68%, year-on-year at constant exchange rates, versus its prior guidance of 26% to 39%. The biotech made $773.7 million in Q2, representing a 17% year-on-year growth and a substantial beat over the consensus revenue forecast of $651 million. In the ATTR-CM market, Alnylam is facing off against Pfizer, which owns the transthyretin stabilizer tafamidis. That drug was approved in 2019 as Vyndaqel and Vyndamax and has used its first-to-market advantage well: In 2024, Pfizer logged $5.45 billion in sales for its tafamidis products, a hefty 60% year-on-year growth. Pfizer remains confident that it can protect its slice of the ATTR-CM market, with CEO Albert Bourla telling investors at a TD Cowen event in March that as long as treatment is going well, doctors “will never switch a patient to something different.” Pfizer “will maintain a lion’s market share” for new ATTR-CM prescriptions, Bourla added, noting the pharma’s extensive network and deep understanding of physician prescription patterns. Also competing with Alnylam is BrdigeBio, which owns Attruby, another transthyretin stabilizer. Since its approval in November 2024 , Attruby had delivered 1,028 unique prescriptions as of Feb. 17. As of April 25, more than 2,000 unique patients had been treated with Attruby, resulting in $36.7 million in sales during the drug’s first commercial quarter.

Drug ApprovalBiosimilarClinical Result

31 Jul 2025

·firstwordpharma.com

Amvuttra's stellar performance pushes Alnylam's guidance, stock higher

The year's second quarter financial report — the first full three-month period since Amvuttra (vutrisiran) won a key label expansion for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) — was a major test of whether Alnylam Pharmaceuticals' RNAi therapeutic could break into a market long dominated by Pfizer. Amvuttra didn't just meet expectations, it blew past them. The drug raked in $491.95 million in sales, more than double its revenues from the year-ago period and exceeding analyst estimates of about $365 million. Amvuttra was first approved for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in 2022.Overall, Alnylam reported $672.21 million in net revenues for the second quarter, a year-over-year increase of 64% — coming in ahead of consensus figures of about $540 million.The stellar beat sent Alnylam's shares soaring up more than 15% on Thursday. It also prompted the biopharma to raise its full-year guidance for its TTR portfolio — which also includes Onpattro (patisiran) — as well as its overall net sales projection for 2025. Amvuttra is the major contributor to the company's TTR portfolio, however. Onpattro brought in only $52.54 million in sales for the quarter, down 33% from the same three-month period in 2024.Guiding for a steep sales trajectoryThe FDA approved Amvuttra in March to treat ATTR-CM, setting it up to compete with Pfizer's Vyndaqel/Vyndamax franchise — which had been the sole treatment option for about five years until the November 2024 approval of BridgeBio's Attruby (acoramidis). Now, Alnylam and BridgeBio are seeing who can wrestle away the biggest piece of the ATTR-CM pie from Pfizer, whose Vyndaqel products brought in $5.5 billion in global sales last year (see – Physician Views Poll Results: Amvuttra another compelling treatment option for ATTR-CM, say majority of cardiologists and Physician Views Results: Cardiologist feedback suggests Alnylam well placed to take on Pfizer in ATTR-CM field).And so far, Alnylam is liking what it's seeing from Amvuttra. The drug's second-quarter results gave the biopharma the confidence to raise its full-year revenue projections for its TTR portfolio to a range of $2.18 billion to $2.28 billion, up from a May estimate of $1.6 billion to $1.73 billion.Alnylam also boosted its total net product revenue guidance to between $2.65 billion and $2.8 billion; it had earlier predicted a full-year revenue range of $2.05 billion to $2.25 billion.

Drug ApprovalFinancial StatementBiosimilarClinical ResultIPO

30 Jun 2025

·www.globenewswire.com

BridgeBio Raises $300 Million Through Partial Capped Monetization of BEYONTTRA® European Royalty

BridgeBio has received a $300 million upfront payment, strengthening the Company’s balance sheet, and supporting the launch of Attruby® and ongoing late-stage pipeline programsTransaction monetizes 60% of BridgeBio’s European royalties on the first $500 million of annual BEYONTTRA net sales, with total payments to the investors subject to an initial cap of 1.45xIn 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 placeboA 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30A 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., June 30, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced it has sold a portion of royalties due to the Company from sales of BEYONTTRA in Europe to HealthCare Royalty (“HCRx”) and funds managed by Blue Owl Capital (“Blue Owl”) for $300 million. This royalty financing agreement monetizes select anticipated royalties and provides immediate less-dilutive capital to the Company. “We’re excited to partner with HCRx and Blue Owl to strengthen our balance sheet in support of the launch of Attruby and our pipeline of first and best-in-class genetic medicines. This transaction preserves significant upside for our shareholders, with careful structuring that limits annual as well as total payments made to the royalty investors. This financing highlights the strong early start and large global potential of acoramidis,” said Chinmay Shukla, Senior Vice President of Strategic Finance at BridgeBio. Clarke Futch, Chairman and Chief Executive Officer of HCRx commented: “We have been following the progress of acoramidis for years and strongly believe in its potential to positively impact the lives of patients living with ATTR-CM. This investment exemplifies HCRx’s commitment to supporting innovation in the biopharmaceutical industry and we are pleased to collaborate with BridgeBio on this transaction.” “We are pleased to continue our support of BridgeBio through this royalty monetization transaction,” said Sandip Agarwala, Managing Director and Head of Life Sciences at Blue Owl. “This investment reflects our confidence in BEYONTTRA commercial potential and our commitment to providing flexible capital solutions that help advance life-saving therapies.” Under the terms of the agreement, BridgeBio has received $300 million from HCRx and Blue Owl managed funds in exchange for 60% of royalties on the first $500 million of annual BEYONTTRA net sales in Europe. The agreement includes an initial cap of 1.45x. Once the applicable cap is met, no further payments will be owed to the investors. In March 2024, BridgeBio entered into an exclusive licensing agreement with Bayer Consumer Care AG to commercialize BEYONTTRA in Europe for the treatment for ATTR‑CM. To date, BridgeBio has received $210 million in upfront and regulatory milestones, and anticipates receiving a further $75 million in near-term milestone payments, along with tiered royalties starting in the low‑30% range on net sales of BEYONTTRA in Europe. Acoramidis is approved in the U.S. as Attruby by the FDA and in Europe as BEYONTTRA by the European Commission. It is also approved as BEYONTTRA by the Japanese Pharmaceuticals and Medical Devices Agency, and UK Medicines and Healthcare Products Regulatory Agency with all labels specifying near-complete stabilization of TTR. Latham & Watkins LLP served as legal advisor to BridgeBio. Gibson, Dunn & Crutcher LLP served as legal advisor to HCRx and Blue Owl. About BEYONTTRABEYONTTRA is an orally administered near-complete (≥90%) stabilizer of transthyretin (TTR) indicated for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). For full prescribing information, please refer to the Summary of Product Characteristics (SmPC) on the Medicines and Healthcare products Regulatory Agency website at https://products.mhra.gov.uk/. 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 BridgeBio Pharma, Inc.BridgeBio 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 bridgebio.com and follow us on LinkedIn, Twitter, Facebook, and YouTube. About HealthCare RoyaltyHealthCare Royalty (HCRx) is a leading royalty acquisition company focused on commercial or near-commercial biopharmaceutical products. With offices in Stamford, Conn., San Francisco, Boston, London and Miami, HCRx has invested $5+ billion in over 90 biopharmaceutical products since inception. For more information, visit https://www.hcrx.com. HEALTHCARE ROYALTY® and HCRx® are registered trademarks of HealthCare Royalty Management, LLC. About Blue OwlBlue Owl (NYSE: OWL) is a leading asset manager that is redefining alternatives®.With $273 billion in assets under management as of March 31, 2025, we invest across three multi-strategy platforms: Credit, Real Assets, and GP Strategic Capital. Anchored by a strong permanent capital base, we provide businesses with private capital solutions to drive long-term growth and offer institutional investors, individual investors, and insurance companies differentiated alternative investment opportunities that aim to deliver strong performance, risk-adjusted returns, and capital preservation. Together with over 1,200 experienced professionals globally, Blue Owl brings the vision and discipline to create the exceptional. To learn more, visit www.blueowl.com. BridgeBio Pharma, Inc. 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,” “could,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “potential,” “projects,” “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 commercial performance of BEYONTTRA in Europe and other global markets; the anticipated benefits of the royalty monetization agreement with HCRx and Blue Owl Capital; BridgeBio’s expectations for the launch and market uptake of Attruby and BEYONTTRA; the belief in acoramidis’ ability to positively impact the lives of patients with ATTR-CM; and the expected use of proceeds to support BridgeBio’s pipeline of genetic medicines, reflect BridgeBio’s current views about its plans, intentions, expectations, and strategies, which are based on information currently available and 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: BridgeBio’s dependence on third parties for development or commercialization activities; regulatory authorities requiring additional studies or data to support the continued or expanded commercialization of acoramidis; whether data and results meet applicable 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 highly competitive and rapidly evolving industry in which new risks may 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.com (650)-789-8220 BridgeBio Investor Contact:Chinmay Shukla, Senior VP Strategic Financeir@bridgebio.com

Drug ApprovalLicense out/inPhase 3Acquisition

100 Deals associated with Acoramidis

External Link

KEGG	Wiki	ATC	Drug Bank
D11972	-	-	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Amyloidosis, Hereditary, Transthyretin-Related	European Union	Bayer AG	10 Feb 2025
Amyloidosis, Hereditary, Transthyretin-Related	Iceland	Bayer AG	10 Feb 2025
Amyloidosis, Hereditary, Transthyretin-Related	Liechtenstein	Bayer AG	10 Feb 2025
Amyloidosis, Hereditary, Transthyretin-Related	Norway	Bayer AG	10 Feb 2025
Transthyretin Amyloid Cardiomyopathy	United States	BridgeBio Pharma, Inc.	22 Nov 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Polyneuropathies	Phase 3	United States	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Bulgaria	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	France	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Germany	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Italy	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Malaysia	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Netherlands	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Portugal	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Singapore	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	South Korea	Eidos Therapeutics, Inc.	12 May 2025

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03860935 (ATTRibute-CM, Pubmed \| J Am Coll Cardiol)	Phase 3	Transthyretin Amyloid Cardiomyopathy sTTR levels	557	Acoramidis	bvqprxtpzl(dfocitryfk) = was associated with reduced ACM in univariate analysis (HR: 0.96 per 1 mg/dL increase in early ΔTTR; 95% CI: 0.93-0.98; P = 0.002). In the multivariate analysis, after adjusting for TTR variant status, baseline New York Heart Association functional class, baseline National Amyloidosis Centre stage, and baseline sTTR level, early ΔTTR remained independently associated with reduced ACM (P < 0.001). lsmgdnwmvx (ciwpsmrjbk ) View more	Positive	01 May 2025
NCT03860935 (ATTRibute-CM, Pubmed \| J Am Coll Cardiol)	Phase 3	Transthyretin Amyloid Cardiomyopathy \| Senile cardiac amyloidosis N-terminal pro-B-type natriuretic peptide level	632	Acoramidis hydrochloride 800 mg twice daily	isdggllbrz(gianxovnvr): relative risk ratio = 50 (95% CI, 0.36 - 0.7), P-Value = < 0.0001 View more	Positive	01 Mar 2025
				Placebo
NCT04622046 (CTgov)	Phase 3	Transthyretin Amyloid Cardiomyopathy	25	ALXN2060	yxtqgazpum(jraetndeys) = wxnyfdwlcd lmhmxcnzsq (gzaaqcoyst, 9.182) View more	-	16 Dec 2024
NCT03860935 (FDA_CDER) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	611	ATTRUBY 712 mg	vruwjtgpdk(bgvearlfmp): P-Value = 0.018 View more	Positive	22 Nov 2024
				Placebo
NCT03860935 (ATTRibute-CM, Company_Website) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	389	Acoramidis	rftowlksme(owuxwrarhn) = cjkvigyawz coaxawrgah (gkgyxzqypj ) View more	Positive	18 Nov 2024
				Placebo	-
NCT03860935 (ATTRibute-CM, GlobeNewswire \| PipelineReview) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	-	Acoramidis	cyviobfshc(ncxpfvuzgt) = koptqftpra lhswkoodmj (dfqqvjkrwd )	Positive	27 Sep 2024
				Placebo	-
NCT03860935 (ATTRibute-CM, ESC2024)	Phase 3	Transthyretin Amyloid Cardiomyopathy serum TTR levels	611	Acoramidis	rlieyukymd(dspkfirtmc) = mnbmczzwho ziltielnwg (umgvzycnes )	Positive	30 Aug 2024
				Tafamidis	rlieyukymd(dspkfirtmc) = wkkfyauejj ziltielnwg (umgvzycnes, 0.4 (4.92))
NCT03860935 (ATTRibute-CM, CTgov)	Phase 3	Transthyretin Amyloid Cardiomyopathy \| Senile cardiac amyloidosis	632	acoramidis (Acoramidis HCl 800 mg)	qwsvarxqyh = htorcislwm frrsqcfyre (sirsbuwzvd, lnqtkihqlv - svsentxtde) View more	-	27 Jun 2024
				Placebo Oral Tablet (Placebo)	qwsvarxqyh = wrxzgehuop frrsqcfyre (sirsbuwzvd, eeswbuybqu - hifrpveghh) View more
NCT03860935 (ATTRibute-CM, Company_Website) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	-	Acoramidis	loecyawtlr(vrblbedkyi) = every 5mg/dL increase in serum TTR level at day 28 after treatment initiation, the risk of death through Month 30 was reduced by 30.9% (by the logistic model) and 26.1% (by the Cox proportional hazards model) psazyqvgnb (xtnxxixfbw ) View more	Positive	29 May 2024
				Placebo
NCT03860935 (ATTRibute-CM, ESC2024)	Phase 3	NT-proBNP	-	Acoramidis	awiyhddktf(rgbarqpufb) = wvdazqcnhz cuutthctit (mhhrlkqzzg )	Positive	13 May 2024

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