biostudies-literatureUnknown13(15)Coats CJ<h4>Background</h4>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).<h4>Methods and results</h4>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 titratiJournal of the American Heart Associatione035993https://www.ebi.ac.uk/biostudies/studies/S-EPMC11964075biostudies-literatureDosing and Safety Profile of Aficamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM.PMC11964075Bundgaard HGarcia-Pavia PGeske JToste ACooper RMarian AEnciso JSBach RVeselka JMasri AShu YSeidler TEsteban MTTWever-Pinzon OYuan ZLyle MMitrovic VZeng QNassif MEKnackstedt CNielsen CSherrid MHagege AAWatkins HAlvarez AGOwens ATHeitner SBMa CSHabib GMusumeci BKramer CCollado ESolomon SDClaggett BArad MStoerk SFermin DDesai MTfelt-Hansen JReant PVera TVRMerkely BZemanek DChoudhury LCheng XJanuzzi JLAmin ATower-Rader AShao CBilen OTurer AKelly JBastos JMMahmod MOwens DYu ZWohltman ADungen HDWong THeart AHalabi MSaberi SHong KMetra MMichels MEmdin MHeart PMeder BMitter SMalik FIHealth OHeart LPiltz XMcGrew FMeng LAsher CHeart SLakdawala NMaurer MWang AHussain ZSEQUOIA‐HCM Investigators *Elliott PWang JLhermusier TLutz JDBarriales-Villa RMogensen JSharma ARassaf TAhmad FWang YBekfani THannawi BYang HDarlington AThune JChen MZfat-Zwas DGerman PCharron PEdelmann FCoats CJNagueh SSen SBlanes JRGCanepa MGlasgow NGLi XRaymer DMarkowitz JOlivotto IMartinez MKupfer SPantazis AUrbano RJHMaron MSSarswat NJensen MJani SLee MMYNaidu SSpertus JALogeart DMiao ZMEmani Svan Sinttruije MOreziak ADudek DAbraham TPTao LCardim NDiaz LMRBering PJacoby DLSymanski JTrochu JNWojakowski WPaz OLewis GDSchulze PCZhang YRader FBrinkley MfalseDosing and Safety Profile of Aficamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM.<h4>Background</h4>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).<h4>Methods and results</h4>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 titrati2024-01-01T00:00:00Z2024 Aug2026-06-01T14:32:58.935Z2026-04-08T13:22:59.397ZS-EPMC119640753905634910.1161/JAHA.124.035993