MyoKardia (MYOK) Reports Positive Six-Month Safety, Efficacy Data

MyoKardia, Inc. (Nasdaq: MYOK), a clinical-stage biopharmaceutical company pioneering a precision medicine approach for the treatment of serious cardiovascular diseases, today announced positive 12- and 24-week safety, efficacy and biomarker data for its investigational drug, mavacamten, from the company’s PIONEER open-label extension (OLE) study. The PIONEER-OLE study enrolled a total of thirteen patients who previously completed the Phase 2 PIONEER-HCM trial. These data reflect the longest duration of mavacamten treatment reported to date.

Mavacamten is being developed for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy (oHCM), in an ongoing Phase 3 pivotal trial, known as EXPLORER-HCM. Patients with oHCM are at an increased risk of heart failure, atrial fibrillation, stroke and death, including sudden cardiac death. There are an estimated 410,000 people with oHCM in the United States, with approximately 65,000 currently diagnosed and seeking medical attention. The EXPLORER-HCM trial is enrolling 220 patients with oHCM. Patients will be randomized 1:1 to receive mavacamten or placebo for 30 weeks with a primary endpoint of clinical response. EXPLORER-HCM topline data is expected in the second half of 2020, with the completion of enrollment expected in the second half of this year.

https://www.streetinsider.com/Corporate+News/MyoKardia+%28MYOK%29+Reports+Positive+Six-Month+Safety%2C+Efficacy+Data+from+PIONEER-OLE+Study+of+Mavacamten+for+Treatment+of+Symptomatic%2C+Obstructive+HCM/15209954.html