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.
About the Webinar
Representatives from Medpace and MyoKardia will be discussing important topics including:
- 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