{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["13(15)"],"submitter":["Coats CJ"],"pubmed_abstract":["Background
Aficamten, a novel cardiac myosin inhibitor, reversibly reduces cardiac hypercontractility in obstructive hypertrophic cardiomyopathy. We present a prespecified analysis of the pharmacokinetics, pharmacodynamics, and safety of aficamten in SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM).Methods and results
A total of 282 patients with obstructive hypertrophic cardiomyopathy were randomized 1:1 to daily aficamten (5-20 mg) or placebo between February 1, 2022, and May 15, 2023. Aficamten dosing targeted the lowest effective dose for achieving site-interpreted Valsalva left ventricular outflow tract gradient <30 mm Hg with left ventricular ejection fraction (LVEF) ≥50%. End points were evaluated during titrati"],"journal":["Journal of the American Heart Association"],"pagination":["e035993"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11964075"],"repository":["biostudies-literature"],"pubmed_title":["Dosing and Safety Profile of Aficamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM."],"pmcid":["PMC11964075"],"pubmed_authors":["Bundgaard H","Garcia-Pavia P","Geske J","Toste A","Cooper R","Marian A","Enciso JS","Bach R","Veselka J","Masri A","Shu Y","Seidler T","Esteban MTT","Wever-Pinzon O","Yuan Z","Lyle M","Mitrovic V","Zeng Q","Nassif ME","Knackstedt C","Nielsen C","Sherrid M","Hagege AA","Watkins H","Alvarez AG","Owens AT","Heitner SB","Ma CS","Habib G","Musumeci B","Kramer C","Collado E","Solomon SD","Claggett B","Arad M","Stoerk S","Fermin D","Desai M","Tfelt-Hansen J","Reant P","Vera TVR","Merkely B","Zemanek D","Choudhury L","Cheng X","Januzzi JL","Amin A","Tower-Rader A","Shao C","Bilen O","Turer A","Kelly J","Bastos JM","Mahmod M","Owens D","Yu Z","Wohltman A","Dungen HD","Wong T","Heart A","Halabi M","Saberi S","Hong K","Metra M","Michels M","Emdin M","Heart P","Meder B","Mitter S","Malik FI","Health O","Heart L","Piltz X","McGrew F","Meng L","Asher C","Heart S","Lakdawala N","Maurer M","Wang A","Hussain Z","SEQUOIA‐HCM Investigators *","Elliott P","Wang J","Lhermusier T","Lutz JD","Barriales-Villa R","Mogensen J","Sharma A","Rassaf T","Ahmad F","Wang Y","Bekfani T","Hannawi B","Yang H","Darlington A","Thune J","Chen M","Zfat-Zwas D","German P","Charron P","Edelmann F","Coats CJ","Nagueh S","Sen S","Blanes JRG","Canepa M","Glasgow NG","Li X","Raymer D","Markowitz J","Olivotto I","Martinez M","Kupfer S","Pantazis A","Urbano RJH","Maron MS","Sarswat N","Jensen M","Jani S","Lee MMY","Naidu S","Spertus JA","Logeart D","Miao ZM","Emani S","van Sinttruije M","Oreziak A","Dudek D","Abraham TP","Tao L","Cardim N","Diaz LMR","Bering P","Jacoby DL","Symanski J","Trochu JN","Wojakowski W","Paz O","Lewis GD","Schulze PC","Zhang Y","Rader F","Brinkley M"],"additional_accession":[]},"is_claimable":false,"name":"Dosing and Safety Profile of Aficamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM.","description":"Background
Aficamten, a novel cardiac myosin inhibitor, reversibly reduces cardiac hypercontractility in obstructive hypertrophic cardiomyopathy. We present a prespecified analysis of the pharmacokinetics, pharmacodynamics, and safety of aficamten in SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM).Methods and results
A total of 282 patients with obstructive hypertrophic cardiomyopathy were randomized 1:1 to daily aficamten (5-20 mg) or placebo between February 1, 2022, and May 15, 2023. Aficamten dosing targeted the lowest effective dose for achieving site-interpreted Valsalva left ventricular outflow tract gradient <30 mm Hg with left ventricular ejection fraction (LVEF) ≥50%. End points were evaluated during titrati","dates":{"release":"2024-01-01T00:00:00Z","publication":"2024 Aug","modification":"2026-06-01T14:32:58.935Z","creation":"2026-04-08T13:22:59.397Z"},"accession":"S-EPMC11964075","cross_references":{"pubmed":["39056349"],"doi":["10.1161/JAHA.124.035993"]}}