ATTR-CM is a rare, life-threatening manifestation of transthyretin amyloidosis in which transthyretin amyloid deposits in the heart and causes restrictive cardiomyopathy and heart failure. It can occur in wild-type, hereditary, or mixed ATTR phenotypes.
Alnylam Pharmaceuticals entered a $1.23 billion discovery collaboration with Tenaya Therapeutics to identify up to 15 novel genetic targets for heart disease. The agreement includes $10 million upfront and up to $1.13 billion in milestone payments.
Alnylam reported full-year 2025 profitability and $2.987 billion in global net product revenues, driven by U.S. approval and growth of AMVUTTRA. The company also outlined 2026 pipeline goals and additional HELIOS-B analyses for vutrisiran.
BridgeBio Pharma reported long term acoramidis data in transthyretin amyloid cardiomyopathy and submitted a US FDA NDA for BBP-418 in LGMD2I/R9. BBP-418 could become the first approved therapy for the disease if cleared.
Prothena reported Q4 and full year 2025 financial results with $308.4 million in cash as partners Roche and Novo Nordisk initiated Phase 3 trials for prasinezumab and coramitug, both expected to complete in 2029.
The cardiovascular biologics market is projected to reach $4.23 billion by 2033, while the cell therapy market is expected to reach $5.38 billion by 2032, driven by RNA-based therapies and regulatory approvals.
The FDA removed the clinical hold on Intellia Therapeutics' MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran for ATTRv-PN, allowing patient enrollment and dosing to resume with enhanced safety monitoring.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07338942 |
Implementation of Standardized Early Identification and Diagnosis for Transthyretin Amyloidosis (ATTR) in High-Risk Populations |
NOT_YET_RECRUITING | NA |
| NCT06836011 |
A Multicenter Study on Atrioventricular Regurgitation in Transthyretin Amyloid Cardiomyopathy: Definition, Prevalence, and Prognostic Impact. |
RECRUITING | |
| NCT06465810 |
Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis |
RECRUITING | |
| NCT06029452 |
A Retrospective Validation Study To Identify Chart-Based Clinical Diagnosis Of Wild-Type Transthyretin Amyloid Cardiomyopathy (Attrwt-CM) And Non-Amyloid Heart Failure Among Patients With Heart Failure (HF). |
COMPLETED | |
| NCT05776212 |
Quantitative-imaging in Cardiac Transthyretin Amyloidosis |
RECRUITING | |
| NCT00935012 |
Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy |
COMPLETED | PHASE3 |
