From Promise to Reality: MyoKardia and Medpace Talk Precision Medicine in Cardiovascular Disease

Life Sciences, Clinical Trials, Pharmaceutical, Drug Discovery & Development,
  • Tuesday, December 15, 2020 | 12pm EST (NA) / 5pm GMT (UK) / 6pm CET (EU-Central)
  • 60 min

Despite advances in medical therapeutics, cardiovascular disease (CVD) remains the leading cause of death worldwide. Notably, heart failure and coronary disease continue to present an exorbitant burden on patients and health care. Recently, breakthroughs in precision medicine are creating optimism for a field that has historically witnessed few long-term successes in targeted therapeutic approaches. Indeed, precision-medicine approaches target the subsets of patients that are most likely to benefit from drug, including approaches based on mechanism of disease and shared patient characteristics. By matching mechanism of a drug action with specific disease etiology, precision-medicine approaches are starting to turn the page in CVD trials.

Investigational drugs like mavacamten, developed by MyoKardia, offer a new hope for patients with hypertrophic cardiomyopathy (HCM), an inherited and often lethal primary myocardial disorder without effective pharmacological options. Indeed, the striking positive results found in obstructive and non-obstructive HCM patients are expected to support a New Drug Application (NDA) filing, thus commencing a new promising chapter in the precision-medicine approach to CVD drug discovery.

Join Medpace and MyoKardia as they discuss the following topics:

  • An overview of CV precision-medicine approach and its role in clinical trials
  • Case study: MyoKardia’s MAVERICK-HCM trial
  • Operational precision medicine challenges, best practices, and lessons learned
  • Future directions, including mitigations during a pandemic



Richard Lee, MD, Senior Medical Director, Medical Department, Medpace

Dr. Richard S. Lee is a board-certified cardiothoracic surgeon with an extensive background in a full range of adult cardiac surgery, including valve repair and replacement, coronary artery bypass grafting, aortic aneurysm surgery, surgery for atrial fibrillation, and transcatheter aortic valve replacement. Dr. Lee graduated summa cum laude from Yale University with a degree in Molecular Biophysics and Biochemistry. After graduating, he earned his medical degree, cum laude, from Harvard Medical School in Boston, Massachusetts. He completed a residency in general surgery at Massachusetts General Hospital as well as a residency in cardiothoracic surgery from Stanford University Medical Center in California. He has solid research experience in transplantation immunology. Dr. Lee has been board certified in cardiothoracic surgery since 2007.

Message Presenter

Jeff Douglas, PharmD, Head of Clinical Operations, MyoKardia

Jeff is a drug development leader with >25 years of global clinical research experience, diversified across clinical settings, including large and small biopharma, contract research organization (CRO), pharmacy and health care institutions.

Message Presenter

Kathy Loxterkamp, RN, BSN, Senior Director, Clinical Trial Management, Medpace

Ms. Loxterkamp, has 27 years of nursing experience in addition to 12 years in program management as a part of Clinical Operations at Medpace.  Kathy received her BSN degree from the University of Cincinnati, College of Nursing and Health.  In her role as Trial Manager at Medpace, Kathy partners in the conduct of clinical trials with small to mid-sized Biopharma in complex cardiovascular programs, metabolic disorders, rare disease and other endocrine disorders. She provides strategic oversight and expert guidance throughout all stages of study planning and execution.

Message Presenter

Who Should Attend?

This webinar will be of interest to clinical trials professionals working within the following areas:

  • Clinical Affairs
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Outsourcing
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

What You Will Learn

  • An overview of the precision-medicine approach and its role in CVD clinical trials
  • Case study: MyoKardia’s MAVERICK-HCM trial
  • Operational precision medicine challenges, best practices, and lessons learned
  • Future directions, including mitigations during a pandemic

Xtalks Partners


Medpace is a scientifically driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages regulatory and therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective. Headquartered in Cincinnati, Ohio, Medpace employs approximately 3,400 people across 39 countries as of September 30, 2020.


MyoKardia is a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases. The company is pioneering a precision medicine approach to its discovery and development efforts by 1) understanding the biomechanical underpinnings of disease; 2) targeting the proteins that modulate a given condition; 3) identifying patient populations with shared disease characteristics; and 4) applying learnings from research and clinical studies to inform and guide pipeline growth and product advancement. MyoKardia’s 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. Among its discoveries are three clinical-stage therapeutics: mavacamten (formerly MYK-461); danicamtiv (formerly MYK-491) and MYK-224. MyoKardia’s mission is to change the world for people with serious cardiovascular disease through bold and innovative science.

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