Date: 2016-02-04
Type of information: Publication of results in a medical journal
phase: preclinical
Announcement: publication of results in Science
Company: Myokardia (USA - CA)
Product: MYK-461
Action
mechanism: MYK-461 is a first-in-class, oral, selective, small molecule allosteric modulator of cardiac myosin designed to treat patients with hypertrophic cardiomyopathy caused by one of the most common molecular mechanisms of disease. MyoKardia is currently evaluating MYK-461 in three Phase 1 clinical trials, which are primarily designed to evaluate the safety and tolerability of MYK-461 and are expected to provide data on its pharmacokinetic and pharmacodynamic profile.
Disease: hypertrophic cardiomyopathy
Therapeutic area: Cardiovascular diseases
Country:
Trial details:
Latest
news: * On February 4, 2016, MyoKardia announced the publication of an article in Science. The article demonstrates the ability of its lead candidate MYK-461 to prevent and reverse development of disease in multiple genetic mouse models of hypertrophic cardiomyopathy. The published research represents the product of collaboration among scientists from MyoKardia, Harvard Medical School, the University of Colorado and Stanford University. These data add to a growing body of laboratory and clinical research demonstrating the potential of MYK-461 as an important and novel approach to treating hypertrophic cardiomyopathy. The study is titled “A Small-Molecule Inhibitor of Sarcomere Contractility Suppresses Hypertrophic Cardiomyopathy in Mice". To study the role of sarcomere mutations in the development of HCM, MyoKardia used previously generated mouse models of HCM, which recapitulate key morphologic and functional features of human HCM. To quantify the level of left ventricular hypertrophy, the cardinal manifestation of HCM, the researchers noninvasively measured left ventricular wall thickness (LVWT). MyoKardia researchers and their collaborators demonstrated that early and chronic administration of MYK-461 could prevent the development of disease. Compared to the characteristic increase in LVWT observed in untreated mutant mice, no increase in LVWT was observed in mutant mice treated with MYK-461. The research also showed that MYK-461 promoted partial reversal of disease, as shown by a measurable decline, upon administration of MYK-461, of LVWT in HCM mice that had already developed hypertrophy. Furthermore, the research showed that MYK-461 could prevent the development of left ventricular fibrosis, which is an important histopathological feature of HCM and causally implicated in other potentially dangerous heart conditions.
