Non-obstructive Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiomyopathy with a prevalence in the general population of 1:200. HCM is a disease of profound cardiac energy deficiency due to energy wasting by the sarcomere. This energy deficit impairs cardiac filling and drives the development of cardiac hypertrophy.

Ischemia is a core pathophysiological feature of HCM, leading to shortness of breath, chest pain/angina typically in connection with exercise and in more severe cases, arrythmias with risk of sudden death. Diastolic dysfunction appears to be one of the most important determinants of heart failure symptoms in this group and the risk of developing progressive heart failure symptoms for non-obstructive HCM (nHCM) patients is high, with approximately 30% of patients at any one point in time symptomatic with NYHA class II or III symptoms.

Our Phase 2 trial in nHCM will provide data on cardiac energetics and function, patient symptoms and maximal functional capacity (peak VO2) to directly support a Phase 3 registrational trial.

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.