MyoKardia (MYOK) Commences PIONEER-HCM Trial Substudy in oHCM Patients

November 11, 2016 8:08 AM EST
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MyoKardia, Inc. (Nasdaq: MYOK) announced an exploratory digital health substudy that is under way as part of the Company’s PIONEER-HCM trial of MYK-461 in symptomatic, obstructive hypertrophic cardiomyopathy (oHCM) patients.

The digital health exploratory objective of the trial assesses the potential effects of MYK-461 on the arterial pulse wave using an investigational photoplethysmography (PPG) biosensor. Data collected with the biosensor will be compared with traditional measures of disease. Abnormal arterial flow patterns have been previously observed in HCM patients using conventional tools such as echocardiography. This substudy will investigate whether a non-invasive investigational PPG wristband, similar to commercially available digital health wearables, could provide information to supplement standard measures and support development of a digital biomarker for treating oHCM patients.

Use of the investigational PPG wristband is part of a partnership between MyoKardia and Wavelet Health, a wearable technology platform for health research. The wristband shines beams of light onto the skin to measure patterns of blood flow in the wrist with each heartbeat. Data from the PPG signal will be analyzed with Wavelet’s proprietary algorithms to characterize the blood flow patterns of the oHCM patients in the study.

“Wearable devices will likely play a transformative role in how we gather, analyze and interpret medical information,” said Steven Steinhubl, M.D., director of digital medicine for Scripps Translational Science Institute. “Early investigational research in this field is an important first step in seeking validation of their considerable potential.”

MyoKardia anticipates that the technology in this study, while investigational, could be applied in longitudinal studies to provide insight into patient physiology during daily activities as well as potential response to treatment.

“One of MyoKardia’s core values is Imagine and Innovate, which extends beyond the discovery of much needed therapies and includes novel applications of digital health to our target disease areas,” said Tassos Gianakakos, chief executive officer. “The digital health substudy is aligned with our precision medicine approach and may provide important insights for clinicians and researchers by deepening our understanding of the HCM patient’s journey.”

PIONEER-HCM is a Phase 2 study assessing the effect of multiple doses of MYK-461 in symptomatic oHCM patients. The primary endpoint is the level of reduction in post-exercise left ventricular outflow tract (LVOT) gradient over 12 weeks of drug treatment. PIONEER-HCM will also explore the relationship between reduction in contractility and LVOT gradient, endpoints measuring functional capacity (i.e., exercise) and clinical symptoms in addition to gathering safety and tolerability data on MYK-461 in an outpatient setting. Topline data from PIONEER-HCM are expected in the second half of 2017.

About MYK-461 and PIONEER-HCM

MYK-461 is an orally administered small molecule designed to reduce left ventricular contractility by allosterically modulating the function of cardiac myosin, the motor protein that drives heart muscle contraction. MyoKardia has evaluated MYK-461 in three Phase 1 clinical trials, primarily designed to evaluate safety and tolerability of oral doses of MYK-461, as well as provide pharmacokinetic and pharmacodynamic data. In April 2016, the U.S. FDA granted Orphan Drug Designation for MYK-461 for the treatment of symptomatic oHCM, a subset of HCM.

MyoKardia is currently studying MYK-461 in PIONEER-HCM, a Phase 2 open-label single-arm study to evaluate safety, tolerability and efficacy of MYK-461 in patients with symptomatic oHCM. The primary endpoint of PIONEER-HCM is the level of reduction in post-exercise left ventricular outflow tract (LVOT) gradient over 12 weeks of drug treatment. PIONEER-HCM will also explore the relationship between reduction in contractility and LVOT gradient, endpoints measuring functional capacity (i.e., exercise) and clinical symptoms in addition to gathering safety and tolerability data on MYK-461 in an outpatient setting.



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