Exhibit 99.1
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MyoKardia to Evaluate Mavacamten as an Alternative to Septal Reduction Therapy
in Obstructive Hypertrophic Cardiomyopathy Patients
Planned Study Will Assess Effect of Mavacamten on the Need for SRT Among
HCM Patients Referred for Invasive Procedures
SOUTH SAN FRANCISCO, Calif., September 10, 2019 – MyoKardia, Inc. (Nasdaq: MYOK), today announced that it will study mavacamten as a therapeutic alternative to septal reduction therapy (SRT). The study will be conducted at leading HCM centers that regularly perform surgical myectomy or alcohol septal ablation procedures, with support from the established referral networks at those centers. The randomized, double-blind study will enroll symptomatic, obstructive HCM patients referred for SRT, and MyoKardia anticipates that enrollment will begin in the first half of 2020.
The study’s Executive Committee is being chaired by Steve Nissen, M.D., Chief Academic Officer at the Cleveland Clinic Heart and Vascular Institute. Milind Desai, M.D., Director, Clinical Operations, Cleveland Clinic Heart and Vascular Institute, is serving as Principal Investigator. The study is being run by Cleveland Clinic C5 Research
“This study is designed to address the question of whether mavacamten, which addresses the underlying cause of HCM, can be an effective alternative to surgical procedures which can only address removal of the physical obstruction of oHCM,” said Jay Edelberg, Senior Vice President of Clinical Development at MyoKardia. “This septal reduction therapy trial will allow us to focus on obstructive HCM patients who have been referred for SRT and to expand our knowledge for the broader HCM community. The data from this blinded, controlled study will supplement the clinical data from our ongoing Phase 3 EXPLORER pivotal trial in understanding the potential of mavacamten in treatment of these patients.”
HCM is a chronic, progressive condition in which the heart muscle thickens due to excess contraction. In the obstructive form of HCM, the wall of the septum thickens and may block the flow of blood from the left ventricle to the aorta. Each year, approximately 1,500 patients with obstructive HCM undergo septal reduction therapy in the United States. SRT is performed as either an open-heart surgical procedure, known as a myectomy, or injection of alcohol into the heart muscle, which causes the heart muscle cells in the thickened area to die. Both procedures are intended to reduce the thickness of the septal wall and alleviate obstruction.
“HCM can be a debilitating disease, interfering with the ability of patients to go about the activities of daily living and putting them at long-term risk of atrial fibrillation, stroke and heart failure,” said Dr. Desai.
MyoKardia is developing mavacamten for the treatment of HCM. Mavacamten was specifically developed to correct the abnormal mechanics of the HCM heart by normalizing the number of myosin-actin cross-bridges that drive the excessive contractility, left ventricular hypertrophy and reduced compliance characteristic of HCM. In the company’s Phase 2PIONEER-HCM clinical trial of patients with obstructive HCM, mavacamten reduced or eliminated the obstruction of the left ventricular outflow tract, resulting in improvements in how patients feel (as measured by New York Heart Association classification), and how their hearts are functioning (based on peak VO2 measured by cardiopulmonary exercise testing).
“MyoKardia is committed to leadership within the broader HCM community, including the development of robust evidence regarding treatment alternatives,” commented Tassos Gianakakos, president and CEO at MyoKardia. “We look forward to results from this and other potential studies outside of our registration program to further inform referral and treatment decisions for patients across the full spectrum of this potentially debilitating disease.”
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