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Additional Relevant Details Data from the trials suggest favorable tolerability of single doses of MYK-461 in healthy volunteers and HCM patients. Additionally, repeat daily doses of MYK-461 were well tolerated for up to 28 days in healthy volunteers across a pharmacologically active dose range, with reductions in cardiac contractility sustained throughout the study period. The data also indicated a dose-dependent reduction in contractility consistent with the magnitude of reduction that the scientific literature indicates may confer a meaningful clinical benefit.Based on these data, MyoKardia plans to initiate its PIONEER-HCM study of MYK-461 in the second half of this year. The Phase 2 study will focus on symptomatic, obstructive HCM (oHCM), for which the U.S. Food and Drug Administration (FDA) has granted MYK-461 Orphan Drug Designation.

Excess left ventricular contractility is responsible for the clinical consequences of HCM, including reduced effort tolerance and symptoms that include shortness of breath and chest pain. HCM substantially increases the risk of atrial fibrillation that can lead to stroke, or malignant ventricular arrhythmias that can cause sudden cardiac death. oHCM, a subset of HCM, is a physiological complication in which abnormally thickened heart muscle obstructs the left ventricular outflow tract (LVOT). In severe and symptomatic cases of oHCM, invasive interventions, including open-heart surgery, may be indicated. The study findings announced today include single-dose data from two patients with LVOT obstruction that support MYK-461’s potential to reduce LVOT pressure gradients.

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Clinical Data Pharmacodynamic Data
Clinical proof of mechanism was observed overall as a dose-dependent reduction in cardiac contractility following single oral doses, both in healthy volunteers and HCM patients. All 15 HCM patients in our single ascending dose study demonstrated reductions in excess cardiac contractility. Furthermore, we observed achievement of stable reductions in contractility following repeat dosing to steady state in healthy volunteers in the two highest-dose cohorts of our multiple ascending dose study, confirming proof of mechanism for multiple doses. As previously noted, the scientific literature and MyoKardia’s research suggest that an agent that produces a modest reduction of cardiac contractility (e.g. 5 percent to 10 percent relative reduction as measured by left ventricular ejection fraction, or LVEF) has potential as a treatment for HCM patients.HCM Patient Subgroup with LVOT Obstruction
In our Phase 1 single dose study, two HCM patients, both with a history of LVOT obstruction, had provocable gradients before administration of a single 96 mg dose of MYK-461 (28 mmHg and 42 mmHg, respectively). In both patients, their gradients were reduced following dosing (to 5 mmHg and 9 mmHg, respectively). Relief of LVOT obstruction is associated with lower mortality, improved exercise capacity and symptom relief in oHCM patients. MyoKardia plans to explore obstruction relief as a primary endpoint in the Phase 2 PIONEER-HCM study.R&D Day; Submission to AHA 2016
MyoKardia plans to host an R&D Day in the fall and will present further details on these Phase 1 studies and the planned Phase 2 PIONEER-HCM study in symptomatic, obstructive HCM. Additionally, MyoKardia intends to discuss the broader registration program for MYK-461.

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