Rimeporide, a first in class NHE-1 inhibitor, is repositioned as a novel target for patients with Duchenne. Rimeporide was developed for the treatment of advanced congestive heart failure (CHF) and was dosed in more than 150 adult subjects. Rimeporide is an orally available drug shown to be cardioprotective and to prolong survival in dystrophic hamsters with dilated cardiomyopathy. In mdx mice Rimeporide demonstrated promising data on skeletal muscle function, fibrosis and inflammation in skeletal, cardiac and diaphragm muscles.
A Phase 1b study to establish the safety of Rimeporide in young boys with Duchenne has been completed. A Phase 2 trial is currently under preparation.
This program is sponsored by EspeRare Foundation.