Non-obstructive Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disease with an estimated prevalence in the general population of 1:200 – 1:500. It is characterized by the abnormal thickening of the heart muscle, which can lead to various complications.

One of the key issues in HCM is a deficiency in cardiac energy, resulting from increased energy demands during contraction, and inefficient energy utilization by the cardiac muscle. This energy deficiency has a significant impact on the functioning of the heart, impairing the relaxation and filling of the heart, and leading to symptoms such as breathlessness and reduced exercise capacity. Impaired energetics occurs early in the progression of HCM, even before the development of heart muscle thickening. Within HCM, a third of patients have no left ventricular outflow tract obstruction at rest or after provocation and are referred to as having non-obstructive disease (nHCM). Patients with nHCM experience a high burden of symptoms of heart failure and are at risk for adverse disease complications yet have no proven pharmacotherapies.

Our Phase 2 trial in nHCM provided data on functional measure assessed by cardiopulmonary exercise test as well as patient symptoms with the potential to directly support advancement into Phase 3 clinical development.

References: C. Semsarian et al. J Am Coll Cardiol, 65 (2015), pp. 1249-1254; Maron MS et al. Circulation. 2006 Nov 21;114(21):2232-9.