Bristol Myers Squibb Announces Phase 3 Trial Failure of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy

▎Armstrong

On April 14, 2025, Bristol-Myers Squibb announced that the Phase III clinical trial of Mavacamten for the treatment of non-obstructive hypertrophic cardiomyopathy (nHCM) did not meet the dual primary endpoints of KCCQ-23 CSS and pVO2. Mavacamten is a new drug acquired by Bristol-Myers Squibb through the $13.1 billion acquisition of MyoKardia and has already been approved for the treatment of obstructive hypertrophic cardiomyopathy (oHCM).

Patients with non-obstructive HCM (nHCM, without LVOTO), accounting for about one-quarter of HCM patients, mainly present with clinical symptoms such as dyspnea, exercise intolerance, and chest discomfort.

Summary

Mavacamten was approved for marketing in 2022, with sales reaching $602 million in 2024, a year-on-year increase of 161%. It has become the standard therapy for oHCM and is in a phase of rapid market expansion. Although it failed in nHCM this time, the proportion is relatively low at only one-fourth, which does not significantly impact the market potential of Mavacamten.