Phase III EXPLORER-HCM trial of MYK 461 in obstructive hypertrophic cardiomyopathy published in The Lancet.- MyoKardia

MyoKardia announced that 30-week results from its pivotal Phase III EXPLORER-HCM clinical trial of MYK 461 (mavacamten) for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy (HCM) were presented during a live Hot Line session at the European Society of Cardiology Congress 2020 and were simultaneously published in The Lancet. MyoKardia previously announced topline data from the EXPLORER-HCM study showing that patients treated with mavacamten experienced statistically significant and clinically meaningful improvements in symptoms, functional status and key aspects of quality of life. In addition to meeting the primary and all secondary endpoints, mavacamten was well tolerated with a safety profile similar to placebo. The data presented and reported in the published manuscript also include mavacamten’s positive impact on reducing cardiac biomarkers associated with poor prognosis and demonstrate the consistency of its therapeutic benefit across multiple prespecified subgroups. Data presented and published in The Lancet reveal that mavacamten’s benefit versus placebo for the primary and all secondary endpoints extended across all prespecified subgroups. Specifically, the reduction in obstruction and the improvements in exercise capacity and clinician (NYHA) and patient (KCCQ and HCMSQ) assessments favoring mavacamten over placebo were highly consistent regardless of age, gender, genetic status, body mass index, background use of beta blockers, or baseline measures of ejection fraction, NYHA class and NT-proBNP. Mavacamten was well tolerated and demonstrated safety results similar to placebo, with no new findings observed from those previously reported. Ninety-seven percent of patients enrolled completed the study treatment period. Overall rates of adverse events, serious adverse events, and cardiac adverse events, including atrial fibrillation, were comparable for patients treated with mavacamten and placebo.See: "Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial" Iacopo Olivotto et al. The Lancet Published:August 29, 2020 DOI:https://doi.org/10.1016/S0140-6736(20)31792-X