American Heart Association

Intellia Therapeutics announces positive Phase 1 data for nex-z, a CRISPR-based gene editing therapy for ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction and favorable safety profile. Data presented at the 2024 AHA Scientific Sessions and published in the New England Journal of Medicine.

46 polygenic risk scores (PRSs) for coronary heart disease (CHD) with similar population-level performance produced discordant individual-level risk estimates, with 20% of participants having PRSs in both the top and bottom 5th percentile at least once. This variability suggests that CHD PRSs with similar population-level characteristics may not provide consistent individual risk estimates, emphasizing the need for refined statistical methods and strategies to communicate uncertainty in clinical implementation.

Risk prediction for primary prevention of cardiovascular disease is universally recommended, with a focus on aligning prevention efforts with absolute risk. The heritability of coronary heart disease (CHD) has led to interest in integrating genetic risk via polygenic risk scores (PRSs), despite lack of guideline recommendations. A study by Abramowitz et al. examined the discordance in predicted risk from 46 CHD PRSs across diverse participants, finding significant variability in risk estimates for individuals. The analysis highlights the need for caution in clinical use of PRSs due to heterogeneity in risk estimates and uncertainty in clinical utility.

A CRISPR-Cas9 gene-editing therapy reduced transthyretin levels in cardiac amyloidosis patients, halting disease progression in most at 1 year.

Fluctuating cholesterol levels without medication changes may indicate higher dementia risk, according to a study using ASPREE trial data. High variability in total cholesterol and LDL-C was linked to increased dementia and cognitive decline, while triglyceride and HDL-C fluctuations were not. The study suggests that maintaining stable cholesterol levels is crucial for brain health.

Idorsia Pharmaceuticals U.S. Inc. announces aprocitentan (TRYVIO™) availability for hypertension treatment in the US, targeting inadequately controlled blood pressure in adults. Presented at the AHA Annual Scientific Sessions 2024, aprocitentan showed significant blood pressure reductions in the PRECISION trial, with mild-to-moderate edema as the most frequent adverse event. Idorsia highlights aprocitentan as the first FDA-approved treatment targeting the endothelin system in over three decades.

Intellia Therapeutics announces positive Phase 1 data for nex-z in ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction, disease stabilization or improvement, and favorable safety profile. Data presented at 2024 AHA Scientific Sessions and published in NEJM.

SUMMIT Phase 3 trial shows tirzepatide significantly reduces heart failure risk in HFpEF and obesity patients, improving symptoms and physical limitations, with Lilly initiating regulatory submissions globally.

Cytokinetics announced new post-hoc analyses from GALACTIC-HF, showing omecamtiv mecarbil's potential efficacy in severe heart failure patients, irrespective of age. The treatment reduced the risk of the primary composite endpoint, with similar effects across age groups. Additionally, omecamtiv mecarbil did not increase ventricular arrhythmias and appeared to reduce the risk for composite outcomes in patients with lower left ventricular ejection fraction (LVEF).

Cytokinetics presented new post-hoc analyses of GALACTIC-HF, showing omecamtiv mecarbil reduced risk of adverse cardiac outcomes in severe heart failure patients across age groups, with significant risk reductions in both <65 and ≥65 years, and potential benefits in reducing ventricular arrhythmia risk.