--Application based on positive results from Phase 3 EXPLORER-HCM trial
Bristol Myers Squibb (NYSE: BMY) today announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for mavacamten, an investigational, first-in-class cardiac myosin inhibitor, for the treatment of patients with obstructive hypertrophic cardiomyopathy (obstructive HCM). Validation of the application confirms the submission is complete, and the EMA's centralized procedure with Committee for Medicinal Products for Human Use (CHMP)'s assessment begins.
"Despite the global prevalence of obstructive HCM and its debilitating symptoms and cardiac dysfunction, there is yet to be an approved therapy that targets the underlying cause of this devastating disease. Currently prescribed medicines largely provide only symptom relief. Mavacamten could potentially provide a treatment option that addresses the unmet needs of people living with obstructive HCM around the world," said Roland Chen, M.D., 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 investigators who have been involved in the EXPLORER-HCM trial."
The application is based on the results of the pivotal Phase 3 EXPLORER-HCM trial, which evaluated mavacamten in patients with symptomatic obstructive HCM versus placebo. Results from the trial showed mavacamten demonstrated a clear treatment effect, with clinically meaningful improvements in symptoms, functional status and quality of life, as well as the ability to relieve left ventricular outflow tract obstruction. In the EXPLORER-HCM study all primary and secondary endpoints were met with statistical significance.
About the Phase 3 EXPLORER-HCM Trial
The EXPLORER-HCM Phase 3 trial enrolled a total of 251 patients with symptomatic (NYHA Class II or III) obstructive hypertrophic cardiomyopathy. All participants had measurable left ventricular outflow tract (LVOT) gradient (resting and/or provoked) greater-than or equal to50 mmHg at baseline.
The primary endpoint for EXPLORER-HCM was a composite functional analysis designed to capture mavacamten's effect on both symptoms and function. Secondary endpoints were changes from baseline to week 30 in postexercise LVOT gradient, pVO2, proportion of patients with at least one NYHA class improvement, and measures of patient reported outcomes. Additional endpoints included changes from baseline to Week 30 in echocardiographic indices, circulating biomarkers, cardiac 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 the left ventricular outflow tract (LVOT) where blood leaves the heart to become obstructed by the 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 frequent cause of obstructive HCM is mutations in the heart muscle proteins of the sarcomere and as many as 50% of patients have a hereditary predisposition to the disease. Obstructive HCM is estimated to affect 400,000-600,000 people worldwide, however many patients remain undiagnosed and/or asymptomatic.
Mavacamten is a first-in-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 demonstrated significant efficacy in reducing cardiac muscle contractility by reducing excess actin-myosin cross-bridging, leading to less hypercontractility and improved relaxation.
In obstructive HCM specifically, it is the first and only selective cardiac myosin inhibitor that has the potential to treat the underlying pathophysiology of the disease.
Mavacamten is an investigational therapy and is not approved for use in any country.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Cautionary Statement Regarding Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that 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 in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, the possibility that mavacamten may not receive regulatory approval for the indication described in this release in the currently anticipated timeline or at all, any marketing approvals, if granted, may have significant limitations on their use, and, if approved, whether such product candidate for such indication described in this release will be commercially successful. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb's business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb's Annual Report on Form 10-K for the year ended December 31, 2020, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.
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SOURCE: Bristol Myers Squibb
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