Bristol Myers Squibb Announces Positive Topline Results for Mavacamten


The treatment for hypertrophic cardiomyopathy met its primary endpoint at week 6 in the phase 3 VALOR-HCM trial, the pharmaceutical company says.

The VALOR-HCM, a phase 3, double-blind, placebo-controlled, randomized study evaluating mavacamten in adults with systematic obstructive hypertrophic cardiomyopathy (HCM) who are eligible for septal reduction therapy (SRT) met its primary endpoint at week 16, Bristol Myers Squibb said in a statement.

Furthermore, the safety of mavacamten was consistent with previous studies.

“We are encouraged by the findings from this important study, which add to the growing body of clinical evidence that supports the promise of mavacamten for patients living with obstructive HCM,” Roland Chen, MD, senior vice president of Cardiovascular Development at Bristol Myers Squibb, said in the statement. “We look forward to sharing the results from VALOR-HCM at the American College of Cardiology 71st Annual Scientific Session & Expo taking place in April.”

Mavacamten, a first-in-class allosteric modulator of cardiac myosin for the treatment of obstructive HCM, targets the underlying pathophysiology of obstructive HCM.

It has been shown to reduce cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy, and reduced compliance, according to the statement.

The company plans to share these data with regulatory agencies.

The study included more than 100 individuals randomized on a 1-on-1 basis to receive mavacamten or a placebo.

Additionally, VALOR-HCM included 3 treatment periods over 128 weeks, including a 16-week active treatment period where all the individuals received mavacamten, a 16-week placebo-controlled period, and a 96-week extension period where all individuals continued to receive mavacamten.

The primary endpoint of VALOR-HCM was a composite of the number of individuals who decided to proceed with SRT prior to or at week 16 and the number of individuals who remained SRT-guideline eligible at week 16 in the mavacamten group compared with the placebo group.

Additionally, the key secondary endpoints were the impact on exercise gradient VOT, NYHA Class and Kansas City Cardiomyopathy Questionnaire and biomarkers at week 16, according to the statement.

Obstructive HCM is a chronic progressive disease where excessive contraction of the heart muscles reduces the ability of the left ventricle to fill can and make it difficult for blood to circulate to the rest of the body. This leads to the development of cardiac dysfunction and debilitating symptoms.

Up to 50% of individuals with obstructive HCM have a hereditary disposition.

The disease can develop at any age but is typically diagnosed between aged 40 and 50 years, according to the statement.

Obstructive HCM has been associated with increased risk of atrial fibrillation, heart failure, stroke, and sudden cardiac death. The most frequent cause of HCM is mutations in the heart muscle proteins of sarcomere.

It is estimated that 400,000 to 600,000 individuals worldwide have obstructive HCM, though many are asymptomatic or are undiagnosed, according to the statement.


Bristol Myers Squibb announces positive topline results from phase 3 VALOR-HCM trial, evaluating mavacamten in patients with obstructive hypertrophic cardiomyopathy who are eligible for septal reduction therapy. Bristol Myers Squibb. News release. February 16, 2022. Accessed February 17, 2022.

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