MyoKardia: Mavacamten treatment produced obstruction reductions in trial

MyoKardia announced the publication of results from the company’s Phase 2 PIONEER-HCM study of mavacamten in an upcoming issue of the Annals of Internal Medicine. In the open-label Phase 2 PIONEER-HCM dose-ranging study, treatment with mavacamten for twelve weeks resulted in significant improvements across the primary and secondary endpoints measured. Among the most noteworthy findings were that mavacamten could be dosed to eliminate the LVOT pressure gradient below the guideline-based definition of obstruction or below the level used to recommend invasive intervention, as measured using echocardiography. Along with LVOT gradient reductions, patients experienced improvements in New York Heart Association functional classification and exercise capacity as measured by peak VO2 levels. Reductions in shortness of breath and in levels of NT-proBNP, a well-established marker of ventricular wall stress, were also observed. Researchers noted that improvements were seen in participants with and without background beta-blocker therapy, with and without sarcomeric genetic variants, and over a wide age range. “The positive findings from the PIONEER-HCM study guided our dosing approach in the pivotal Phase 3 EXPLORER-HCM trial of mavacamten for the treatment of oHCM, and recently reported results from the PIONEER open-label extension study have further confirmed this approach. Patients start at a daily dose of 5mg and, based on echocardiographic assessment, may be titrated up to eliminate any residual LVOT gradient. In this way, each patient’s dose is individualized, and we believe this approach will reflect real-world usage,” said June Lee, M.D., Chief Development Officer.

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