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MyoKardia Shares Get a Jolt from Positive Phase 3 HCM Clinical Trial Results

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Shares of MyoKardia traded 60% higher, setting a new 52-week high price, after the company reported that it met all primary and secondary endpoints in the Phase 3 EXPLORER study of mavacamten in treatment of obstructive hypertrophic cardiomyopathy.

Clinical-stage biopharmaceutical company MyoKardia Inc. (MYOK:NASDAQ), which is focused on developing targeted therapies for the treatment of serious cardiovascular diseases, today announced it achieved "positive topline data from the company's Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM)." The firm further stated that MYK-461 (mavacamten) demonstrated a robust, well-tolerated and safe treatment effect and that it met all primary and secondary endpoints in the study that produced statistically significant results including reduction or elimination in obstruction of the left ventricle in treated patients.

Iacopo Olivotto, M.D., of Careggi University Hospital and lead clinical investigator for the EXPLORER-HCM clinical trial commented, "The extraordinary data from the EXPLORER pivotal trial confirm mavacamten's ability to relieve dynamic outflow obstruction, control symptoms and improve quality of life in patients with hypertrophic cardiomyopathy...HCM is the most common inherited cardiovascular disease, and patients face an uncertain journey that all too frequently includes debilitating symptoms, as well as serious complications, such as heart failure, stroke and cardiac arrest. Mavacamten is the first drug developed to target the specific molecular defect of the disease. EXPLORER represents a major achievement toward a precision-medicine approach in cardiomyopathies and should provide great hope to a community painfully aware of the lack of disease-specific treatment options."

The company's CEO Tassos Gianakakos remarked, "The resoundingly positive data from EXPLORER bring us a significant step closer to improving the lives of people with serious cardiovascular conditions, starting with HCM, a debilitating disease estimated to affect one in every 500 people...The activity and tolerability profile observed for mavacamten in this pivotal study underscores the profound impact and potential for therapeutics that target the underlying biology of disease. We look forward to the submission of MyoKardia's first New Drug Application and, importantly, to serving the many patients that stand to benefit from mavacamten."

The firm indicated that the EXPLORER-HCM clinical trial enrolled a total of 251 patients and is part of MyoKardia's pivotal program studying mavacamten as a treatment for symptomatic, obstructive hypertrophic cardiomyopathy. The company stated that it plans to submit a New Drug Application to the U.S. Food and Drug Administration (FDA) in Q1/21.

The company explained that "HCM is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to the development of debilitating symptoms and cardiac dysfunction and it is estimated to affect one in every 500 people." The firm noted that the most frequent cause of HCM is mutations in the heart muscle proteins of the sarcomere; however, it has additionally been linked to increased risks of atrial fibrillation, stroke, heart failure and sudden cardiac death.

MyoKardia is a clinical-stage biopharmaceutical company based in Brisbane, Calif., that concentrates on developing targeted therapies for cardiovascular diseases. The firm stated that "its initial focus is on small molecule therapeutics aimed at the proteins of the heart that modulate cardiac muscle contraction to address diseases driven by excessive contraction, impaired relaxation, or insufficient contraction."

MyoKardia began the day with a market capitalization of around $2.9 billion with approximately 46.68 million shares outstanding and a short interest of about 10.6%. MYOK shares opened nearly 67% higher today at $101.91 (+$40.82, +66.82%) over Friday's $61.09 closing price and reached a new 52-week high price this morning of $104.26. The stock has traded today between $98.02 to $104.26 per share and is currently trading at $100.06 (+$38.97, +63.79%).


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