Study highlights need for new options within heart failure treatment paradigm

Stealth BioTherapeutics' phase 2 study examines whether treatment for myocardial dysfunction will relieve congestive heart failure.

Elamipretide is intended for the treatment of mitochondrial dysfunction. The phase 2 study—IDDEA-HF—is a randomized, double-blind, placebo-controlled study to evaluate the cardiac and renal effects of daily treatment with elamipretide in patients who have been hospitalized with congestive heart failure.

"In heart failure, mitochondrial dysfunction may not only be a causative factor but may also contribute to the progression of the disease and the associated fluid build-up that causes congestion, due to muscle weakness from a lack of energy production," Stealth Vice President of Clinical Development Jim Carr said in a statement. "In line with our findings in elderly patients enrolled in the MOTION trial, we hope to demonstrate the ability of elamipretide to increase energy production to help the heart muscle work better, subsequently relieving congestion in the body."

Up to 300 patients will be randomized within 72 hours of presenting congestive heart failure symptoms. They will be given 20 mg daily elamipretide or placebo intravenously for up to seven days.

The primary endpoint is change in NT-proBNP, a cardiac biomarker reflecting the level of congestion. Secondary endpoints include change in clinical status and safety and tolerability.

"IDDEA-HF is a key step in our development of therapies for common diseases of aging. These patients have failed current therapies, experiencing an episode of acute decompensation, which highlights the intense need for new options within the heart failure treatment paradigm where we believe elamipretide can have a significant impact," said Reenie McCarthy, Stealth CEO. "The data from this study, together with our other ongoing trials in heart failure, will help inform our projected phase 3 heart failure program as well as our approach to other common diseases of aging for which elamipretide may have therapeutic potential."

Heart failure currently affects approximately 21 million people in the U.S. and Europe, with those numbers expected to grow as the population continues to age.