Get the Latest Investment Ideas Delivered Straight to Your Inbox. Subscribe

TICKERS:

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

Share on Stocktwits

Source:

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%).

[NLINSERT]

Disclosure:
1) Stephen Hytha compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.
2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.
3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.
4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the interview or the decision to write an article until three business days after the publication of the interview or article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.
6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.





Want to read more about Biotechnology / Pharmaceuticals investment ideas?
Get Our Streetwise Reports Life Sciences Report Newsletter Free and be the first to know!

A valid email address is required to subscribe