MyoKardia presents cardiac imaging data from Mavacamten’s 30-week EXPLORER-HCM study


EXPLORER-HCM Cardiac Imaging Data Presented at AHA 2020 Scientific Session with Concurrent Publication in Circulation

Mavacamten T.reatment Caused Favorable effect on Cardiac structure – Significantly reduced Hypertrophy in patients with hypertrophic cardiomyopathy

BRISBANE, Calif., November 15, 2020 (GLOBE NEWSWIRE) – MyoKardia, Inc. (MYOK) announced the results of its mavacamten cardiac magnetic resonance imaging (CMR) substudy for the potential treatment of hypertrophic cardiomyopathy (HCM) demonstrating that 30-treatment weekly with mavacamten has a favorable effect on cardiac structure, maintaining contractile function within the normal range. These data were shared today in an oral presentation titled “Mavacamten Favorably Impacts Cardiac Structure in Obstructive Hypertrophic Cardiomyopathy: EXPLORER-HCM CMR Substudy” (Oral session 18654), at the American Heart Association Scientific Sessions 2020 during the High-profile clinical sciences in CVD session. Data from the EXPLORER-HCM CMR substudy was also published simultaneously in Circulation.

The CMR substudy was conducted as part of the pivotal phase 3 clinical trial EXPLORER-HCM of MyoKardia in patients with symptomatic and obstructive HCM to evaluate the impact of once daily treatment with mavacamten on cardiac structure and function parameters. Thirty-five patients were enrolled in the CMR substudy and randomized to mavacamten (n = 17) or placebo (n = 18) and had valid CMR assessments at day 1 and week 30 (end of treatment) which were centrally blinded. Statistically significant changes from baseline to Week 30 were observed in the mavacamten group compared to placebo for the primary endpoint of the left ventricular mass index (p <0.0001), as well as the exploratory endpoints of the absolute intracellular myocardial mass index. , maximum wall thickness and left atrial volume index (all p <0.001 for the difference from placebo). From baseline to week 30, there was no worsening of myocardial fibrosis, another common feature of HCM.

“Hypertrophic cardiomyopathy is defined by thickening of the heart muscle, so seeing favorable remodeling of the heart indicating a decrease in hypertrophy within 30 weeks of treatment is a highly encouraging finding for the many patients struggling with HCM,” he said. Sara Saberi, MD, assistant professor in the division of cardiovascular medicine and member of the hereditary cardiomyopathy program at the University of Michigan Medical School and lead author of the study. “The CMR substudy is the first randomized controlled clinical trial in HCM patients to demonstrate that a once daily oral therapeutic agent can reduce left ventricular wall thickness and mass and positively impact many other structure parameters. and heart function. Left ventricular hypertrophy and left atrium volumes in particular are predictive of poor prognosis in HCM patients, so it will be exciting to see how mavacamten affects heart function and disease progression as we continue to follow patients in time “.

Changes in left ventricular hypertrophy and left atrium volumes were observed concurrently with reductions in plasma biomarker levels of stress and myocardial injury, consistent with echocardiographic observations of the overall EXPLORER-HCM population. Importantly, the reduction in left ventricular mass index correlates with a reduction in cardiac troponin I.

“The data from the CMR substudy complement and extend our observations on the positive benefits of mavacamten on cardiac function and symptom relief, providing us with encouraging evidence that treatment with mavacamten can change the course of adverse cardiac remodeling inherent in HCM. In directly targeting heart muscle proteins that lead to excessive contraction and compromised relaxation underlying HCM, we have a growing body of data demonstrating that mavacamten reduces hyper-contractility and hypertrophy and reduces damage. to the heart muscle by keeping contractile function within the normal range, “said Jay Edelberg, MD, Ph.D., Chief Medical Officer of MyoKardia.

As previously reported, the EXPLORER-HCM results demonstrated that patients treated with mavacamten experienced statistically significant and clinically significant improvements in symptoms, functional status and key aspects of quality of life. In addition to meeting the primary and secondary endpoints, mavacamten was well tolerated with a similar safety profile to placebo. MyoKardia plans to file a new drug application for mavacamten with the U.S. Food and Drug Administration (FDA) in the first quarter of 2021.

Information on EXPLORER-HCM
The Phase 3 EXPLORER-HCM study enrolled a total of 251 patients with symptomatic hypertrophic obstructive cardiomyopathy (NYHA class II or III). All participants had a measurable (resting and / or provoked) LVOT gradient ≥50 mmHg at baseline. Patients were randomized on a 1: 1 basis to receive an individualized daily dose of mavacamten or placebo. Patients started with a dose of 5 mg, with a maximum of two possibilities for dose adjustments (at doses of 2.5 mg – 15 mg) based on a combination of residual LVOT gradient, plasma drug concentration and levels of left ventricular ejection fraction (LVEF).

The primary endpoint for EXPLORER-HCM was a composite functional assay designed to capture the effect of mavacamten on both symptoms and function. The composite functional endpoint is defined as (1) the achievement of a ≥1.5 mL / kg / min improvement in peak VO2 accompanied by an improvement of ≥1 NYHA functional class or (2) the achievement of ≥3, 0 ml / kg / min improvement in VO2 peak without worsening of NYHA functional class. In addition to the endpoints reported today, the EXPLORER-HCM study also evaluated the effect of mavacamten on patient-reported outcomes, health-related quality of life, and assessments of symptom severity, changes from baseline to Week 30 in echocardiographic indices. , circulating biomarkers, rhythmic patterns and accelerometry.

Of MyoKardia
MyoKardia is a clinical stage biopharmaceutical company that discovers and develops targeted therapies for the treatment of severe cardiovascular disease. The company is pioneering a precision medicine approach to its discovery and development efforts by 1) understanding the biomechanical basis of disease; 2) target the proteins that modulate a given condition; 3) identify patient populations with shared disease characteristics; and 4) apply the lessons learned from research and clinical trials to inform and guide pipeline growth and product progress. The initial focus of MyoKardia is on heart protein-targeted small molecule therapies that modulate heart muscle contraction to address diseases driven by excessive contraction, reduced relaxation, or insufficient contraction. Among his discoveries there are three therapies in the clinical phase: mavacamten (former MYK-461); danicamtiv (formerly MYK-491) and MYK-224.

MyoKardia’s mission is to change the world for people with severe cardiovascular disease through bold and innovative science.

Forward-Looking Statements
The statements we make in this press release may include statements that are not historical facts and are considered forward-looking under Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which they are usually identified by the use of words such as “anticipate”, “believe”, “estimate”, “expect”, “intend”, “may”, “plan”, “projects”, “seek”, “should,” “will” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the Safe Harbor provisions for forward-looking statements in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and we are making this statement in order to comply with these Safe Harbor provisions. These forward-looking statements, including statements regarding the clinical and therapeutic benefit and future potential of mavacamten, the ability of our long-term studies to provide further evidence of how mavacamten affects heart function and disease progression over time, ability of our term study to provide further evidence of the potential of mavacamten to have a positive impact on many other parameters of cardiac structure and function, that treatment with mavacamten can change the course of adverse cardiac remodeling inherent in HCM and the ability to mavacamten to reduce damage to the heart muscle by keeping contractility functions within the normal range, reflect our current views on our plans, intentions, expectations, strategies and perspectives, which are based on the information currently available to us and the assumptions we have done. While we believe that our plans, intentions, expectations, strategies and perspectives reflected or suggested by such forward-looking statements are reasonable, we cannot guarantee that any plans, intentions, expectations or strategies will be achieved or implemented. In addition, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks and factors that are beyond our control including, without limitation, the risks associated with the development and regulation of our candidate products. and any ongoing effects of the COVID-19 pandemic, as well as those noted in our quarterly report on Form 10-Q for the quarter ended September 30, 2020, and our other SEC filings. Except as required by law, we undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.

Michelle Corral
Executive Director, Corporate Communications and Investor Relations
MyoKardia, Inc.
[email protected]

Hannah Deresiewicz (investors)
Stern Investor Relations, Inc.
[email protected]

Julie Normant (media)
[email protected]

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