We believe that to treat cardiomyopathies you have to understand the root cause. We’re focused on restoring cardiac contractility by targeting the key proteins that drive sarcomeric function.

The sarcomere is the basic contractile unit within muscle fibers, made up of thick and thin protein filaments that slide past one another. Within the sarcomere, myosin (the motor protein attached to the thick filament) binds to actin (a protein bound to the thin filament), forming cross-bridges that regulate the contraction and relaxation of the heart in a tightly regulated cycle. Thousands of sarcomeres acting in concert provide the ensemble force for muscle contraction.

sacromere

MyoKardia's Precision Medicine Approach
path connecting approaches path connecting approaches path connecting approaches
heart

understand
the cause

Mutations in cardiac sarcomere proteins are known to give rise to a few common biomechanical defects underlying numerous cardiovascular diseases. Our research has linked these biomechanical defects to three distinct cardiac muscle disruptions -- excessive contraction, impaired relaxation and/or insufficient contraction -- that reduce the ability of the heart to pump blood to meet the needs of the body.
cardiac muscle protiens

Target
cardiac muscle
proteins

Our targeted therapies are intended to correct or offset the downstream disruption in cardiac muscle function that drives disease progression. Our initial focus has been on myosin and actin, which drive the cycle of contraction (systole) and relaxation (diastole) as they bind to form cross bridges and then release.
group of people

Segment
patient
populations

We focus on the treatment of relatively small patient subsets where the mechanism of disease and the shared characteristics of the patients are known. Using clinical feedback on patient genetics, response to therapy and disease presentation, we are able to refine our understanding of which patients are most likely to benefit from our product candidates.
MyoKadia logo, stylised heart

Apply
learnings

Our integrated research and development efforts enables the iterative generation of new disease targets and clinical development candidates designed to establish early clinical proof-of-concept. Data from preclinical studies informs clinical development objectives, and data generated from our experience in the clinic is guiding discovery, optimization and development choices for therapeutics in our emerging pipeline.
researchers
pipeline
patients

featured publication

Long-term Safety and Effectiveness of Mavacamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy Patients, PIONEER-Open Label Extension Study (PIONEER-OLE)

American College of Cardiology Scientific Sessions 2019 / Heitner, et al

Mar 2019

see all publications
grants & funding

I had made a great choice in becoming a MyoKardian.

The MyoKardia on-boarding experience was energizing. It focused on understanding of MyoKardia's values and included conversations with members of the Executive Committee to allow me to quickly understand the company's strategic approach and priorities. As I had moved cross-country to join MyoKardia, my colleagues’ friendliness, curiosity and attentiveness further confirm that I had made a great choice in becoming a MyoKardian.

Mondira B ⁠— VP, Pharmacovigilance

Recharge and take time for self-enrichment

In 2018, we introduced a sabbatical program for employees who achieve their six-year anniversary with the company. Our sabbatical program provides a great opportunity for employees to recharge, spend quality time with family and friends or take time for self-enrichment

Ingrid Boyes — SVP, Human Resources

Right drug to the right patient at the right time.

Precision medicine involves integrating genetic and molecular information, as well as clinical and environmental factors, to discover and develop treatments tailored to the underlying causal factors or shared characteristics of a patient’s disease. In short, I like to say that our goal is to get the right drug to the right patient at the right time.

Bob McDowell, PhD — Chief Scientific Officer