At the 70th annual American College of Cardiology (ACC) 2021 meeting, positive data from the EXPLORE-HCM trial was presented for Bristol-Myers Squibb’s mavacamten, a targeted inhibitor of cardiac-myosin that is currently at the pre-registration stage of development in the US for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). According to GlobalData, a leading data and analytics company, the data demonstrates that mavacamten provides a potentially novel strategy for the treatment of oHCM, with significant improvements observed in the health status of trial participants,and increases its chance of FDA approval. The expected strong uptake of the drug is also expected to lead to mavacamten gaining a significant portion of the oHCM market.

Akash Patel, Healthcare Analyst at GlobalData, comments: “Mavacamten stands out from the current crowd of therapies for oHCM as it is a targeted inhibitor of cardiac myosin that leads to a reduction in the number of myosin-actin cross bridges and decreases contractility. Mavacamten is the first therapy to offer this MoA, that is specifically designed to treat the underlying pathophysiology that results in oHCM. Further, Mavacamten is the first disease-specific therapy for oHCM, with the current therapeutic regimen consisting of beta-blockers, anti-arrhythmia and anti-hypertensive drugs that lead to poor long-term outcomes and a reduced quality of life for many oHCM patients.”

The EXPLORE-HCM trial included patients with hemodynamically significant oHCM, however, whether these benefits would be observed in other patient HCM populations requires further long-term studies.

Patel adds: “EXPLORE-HCM demonstrates that mavacamten markedly improves the health of patients with symptomatic oHCM compared with placebo. Mavacamten provides a novel strategy to patients treating oHCM and achieving the primary goals of improving symptoms, physical and social function, and quality of life leading to significant uptake of the drug by physicians and patients”.