American Heart Association

Gene therapy for Danon disease improved or stabilized heart structure and function in 6/7 patients over 24-54 months, with no major safety concerns. The treatment, RP-A501, involved a single dose of AAV9 vector carrying the LAMP2B transgene, showing promising results in halting disease progression and improving quality of life.

Arrowhead Pharmaceuticals presents new data on plozasiran from PALISADE Phase 3 and MUIR/SHASTA-2 open-label extension studies at AHA24, showing significant reductions in triglycerides and favorable lipid profile changes, supporting its development for FCS, severe hypertriglyceridemia, and mixed hyperlipidemia.

Linear ablation combined with ethanol infusion via the vein of Marshall (EIVOM) significantly improves freedom from atrial arrhythmia recurrence without antiarrhythmic drugs (70.7% vs 61.5%; hazard ratio, 0.73) in patients with persistent atrial fibrillation (AF) undergoing first-time ablation, according to a randomized clinical trial involving 498 patients.

Cardiol Therapeutics announced positive Phase II MAvERIC-Pilot study results for CardiolRx in treating pericarditis, with sustained pain and inflammation reduction over 26 weeks, supporting upcoming Phase III trials.

Cardiol Therapeutics reports positive Phase II MAvERIC-Pilot study results showing CardiolRx™ maintained improvements in pericarditis pain and inflammation over 26 weeks. These findings support the initiation of Phase III trials, MAVERIC-3 and MAVERIC-2, to assess CardiolRx™ for pericarditis treatment and recurrence prevention.

Cardiol Therapeutics reported positive Phase II MAvERIC-Pilot results for CardiolRx™ in treating recurrent pericarditis, showing marked improvements in pain and inflammation maintained throughout the 26-week study, with key results including pain reduction from 5.8 to 1.5 at week 26, CRP normalization in 80% of patients at week 8, and reduction in pericarditis episodes from 5.8 to 0.9 per year, supporting advancement to Phase II/III MAVERIC-2 and Phase III MAVERIC-3 trials.

Intellia Therapeutics reports early-stage data indicating its gene editing treatment, NTLA-2001, may stabilize or improve outcomes for ATTR-CM patients, showing a median improvement in the six-minute walk test and 92% maintaining or improving NYHA class. The treatment uses CRISPR to reduce TTR production, with a 90% mean reduction observed. Intellia is now in Phase III trials for NTLA-2001, with potential revenue of $1.2bn by 2030.

Boston Scientific announced positive three-year results for its Watchman FLX device in a clinical trial, demonstrating superiority to oral anticoagulation for stroke risk reduction in non-valvular atrial fibrillation patients post-cardiac ablation. The OPTION trial met primary safety and efficacy endpoints, with Watchman FLX showing non-inferiority and potential to expand its indication as a frontline therapy.