Mavacamten is currently being developed for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults. Hypertrophic cardiomyopathy (HCM) is a genetic condition whereby areas of heart muscle become thickened and stiff. Blood decreases in the left ventricular volume and narrowing of the left ventricular outflow tract (LVOT) is classified as obstructive HCM (oHCM). HCM is a genetic condition that is caused by a change or fault (or mutation) in one or more genes. The most common symptoms are shortness of breath, palpitations, chest pain and light-headedness. Patients with oHCM can develop serious complications such as atrial fibrillation, heart failure, malignant ventricular arrhythmias, and sudden cardiac death (SCD).
Mavacamten, intended for oral administration, is the first therapeutic candidate in a new class of direct myosin (protein involved in muscle contraction) inhibitors. It targets excessive contractility and impaired relaxation, heart muscle energetics and compliance, with the intent of correcting the abnormal function of the HCM heart. If licensed, mavacamten in addition to the current standard of care would offer an additional treatment options for adult patients with symptomatic oHCM.
Treprostinil is in clinical development for patients with chronic thromboembolic pulmonary hypertension (CTEPH). CTEPH is a progressive disease caused by blood clots forming in the lungs that do not dissolve. These clots restrict blood flow through the lungs and cause scar tissue to form on the inside of the pulmonary arteries (arteries that supply the lungs) resulting in pulmonary hypertension. This means the heart has to work harder to pump the blood and can weaken the heart muscles. Some patients may be offered surgery to remove the scar tissue but in patients who are inoperable or who have persistent/recurrent CTEPH after surgery there is a need for additional treatment options.