PRINCETON, NJ – (BUSINESS WIRE) – October 1, 2021–
Bristol Myers Squibb (NYSE: BMY) today announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for mavacamten, a leading experimental cardiac myosin inhibitor class, intended for the treatment of patients with obstructive hypertrophy cardiomyopathy (obstructive HCM). The validation of the application confirms that the submission is complete and that the centralized procedure of the EMA with the evaluation of the Committee for Medicinal Products for Human Use (CHMP) begins.
“Despite the global prevalence of obstructive CMH, its debilitating symptoms and cardiac dysfunction, there is as yet no approved treatment that targets the underlying cause of this devastating disease. Most of the medications currently prescribed only relieve symptoms. Mavacamten could potentially provide a treatment option that addresses the unmet needs of people living with obstructive HCM worldwide, ”said Roland Chen, MD, senior vice president, Cardiovascular Development, Bristol Myers Squibb. “Today’s acceptance of the dossier by the EMA is a step forward in bringing this important targeted therapeutic approach to patients and physicians in Europe, and we thank the patients and researchers who were involved in the EXPLORER trial -HCM.
The request is based on the results of the pivotal phase 3 EXPLORER-HCM trial, which evaluated mavacamten in patients with symptomatic obstructive HCM versus placebo. The results of the trial showed that the mavacamten demonstrated a clear therapeutic effect, with clinically significant improvements in symptoms, functional status and quality of life, as well as the ability to relieve obstruction of the pathway. left ventricular ejection. In the EXPLORER-HCM study, all primary and secondary endpoints were met with statistical significance.
About the EXPLORER-HCM Phase 3 trial
The Phase 3 EXPLORER-HCM trial enrolled a total of 251 patients with symptomatic obstructive hypertrophic cardiomyopathy (NYHA class II or III). All participants had a measurable left ventricular outflow pathway (LVOT) gradient (at rest and / or provoked) ≥50 mmHg at baseline.
The primary endpoint for EXPLORER-HCM was a composite functional analysis designed to capture the effect of mavacamten on symptoms and function. Secondary endpoints were changes from baseline at week 30 in post-exercise LVOT gradient, pVO2, proportion of patients with at least NYHA class improvement, and measurements of patient-reported results. Additional endpoints included changes from baseline at week 30 in echocardiographic indices, circulating biomarkers, heart rhythm patterns, and accelerometry.
About Obstructive Hypertrophic Cardiomyopathy
Obstructive Hypertrophic Cardiomyopathy (Obstructive HCM), the most common type of HCM, is a chronic, progressive disease in which the heart muscle becomes abnormally enlarged or thick, causing obstruction of the left ventricular outflow tract (LVOT) where blood leaves the heart to become blocked by enlarged heart muscle. As a result, obstructive HCM can lead to debilitating symptoms for patients and has also been associated with increased risks of atrial fibrillation, stroke, heart failure, and sudden cardiac death.
The most common cause of obstructive HCM is mutations in sarcomere heart muscle proteins and up to 50% of patients have an inherited predisposition to the disease. Obstructive HCM is estimated to affect 400,000 to 600,000 people worldwide, but many patients remain undiagnosed and / or asymptomatic.
Mavacamten is a first-class oral allosteric modulator of cardiac myosin being investigated for the treatment of conditions caused by excessive cardiac contractility and impaired diastolic filling of the heart, including hypertrophic cardiomyopathy (HCM) and heart failure. with preserved ejection fraction (HFpEF).
In clinical studies, mavacamten has been shown to be significantly effective in reducing cardiac muscle contractility by reducing excess actin-myosin crossbridges, leading to less hypercontractility and better relaxation.
In obstructive HCM in particular, it is the first and only selective cardiac myosin inhibitor that has the potential to address the underlying pathophysiology of the disease.
Mavacamten is an investigational treatment and it is not approved for use in any country.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company with a mission to discover, develop and deliver innovative medicines that help patients overcome serious illnesses. For more information on Bristol Myers Squibb, visit us on BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Caution regarding forward-looking statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 concerning, among other things, the research, development and marketing of pharmaceutical products. All statements that are not statements of historical fact are, or may be considered, forward-looking statements. These forward-looking statements are based on historical performance and current expectations and projections regarding our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, deflect or modify one of them. over the next few years, which are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed or implied in the statements. These risks, assumptions, uncertainties and other factors include, among others, the possibility that mavacamten does not receive regulatory approval for the indication described in this release within the currently planned timeframe or not at all, any marketing authorization. , if granted, may have significant limitations on their use and, if approved, whether this product candidate for such an indication described in this release will be commercially successful. No forward-looking statement can be guaranteed. The forward-looking statements contained in this press release should be weighed together with the many risks and uncertainties that affect the business and the market of Bristol Myers Squibb, particularly those identified in the disclaimer and discussion of risk factors in the Bristol Myers Squibb annual report on Form 10-K for the fiscal year ended December 31, 2020, as updated by our subsequent quarterly reports on Form 10-Q, current reports on Form 8-K and other documents filed with the Securities and Exchange Commission. Forward-looking statements included in this document are made only as of the date of this document and except as otherwise provided by applicable law, Bristol Myers Squibb assumes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, change in circumstances or otherwise.
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CONTACT: Bristol Myers Squibb
KEYWORD: EUROPE UNITED STATES NORTH AMERICA NEW JERSEY
INDUSTRY KEYWORD: CARDIOLOGY SCIENCE BIOTECHNOLOGY PHARMACEUTICAL RESEARCH GENERAL HEALTH HEALTH CLINICAL TRIALS
SOURCE: Bristol Myers Squibb
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PUB: 01/10/2021 06:59 / DISC: 01/1021 06:59