Phase 2 Trial on Therapy for Hospitalized Patients with Congestive Heart Failure Begun

Phase 2 Trial on Therapy for Hospitalized Patients with Congestive Heart Failure Begun

Stealth Bio, a company that develops drugs to treat mitochondrial dysfunction and genetic mitochondrial diseases, recently launched Phase 2 of the clinical trial IDDEA-HF assessing elamipretide in patients hospitalized with congestive heart failure.

Heart failure affects about 21 million people in the U.S. and Europe with cases expected to rise as populations continue to age. Heart failure is the reduced ability of the heart muscle to relax and contract in order to pump blood through the body. It is usually associated with mitochondrial dysfunction and its resulting lack of energy.

“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,” said Jim Carr, Stealth’s vice president of clinical development, in a press release. “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.”

Elamipretide is an investigational drug that could modify disease by preserving energetics and restoring normal energy production in mitochondria while diminishing oxidative stress. Energy from mitochondria sustains health physiology and prevents disease.

The IDDEA-HF clinical trial (NCT02914665) is one of three Phase 2 studies recently initiated to evaluate elamipretide in heart failure. The other two are: PROGRESS-HF (NCT02788747), evaluating the effects of elamipretide in patients with stable heart failure with reduced ejection fraction; and RESTORE-HF (NCT02814097), evaluating the effects of elamipretide in patients with stable heart failure with preserved ejection fraction.

IDDEA-HF will recruit 300 patients. They will be randomized within 72 hours of presentation to receive 20 mg of elamipretide daily or a placebo administered intravenously for up to seven days. Researchers will seek primarily to determine change in a cardiac biomarker that reflects level of congestion (NT-proBNP). The study’s secondary endpoints include safety, tolerability, and change in clinical status.

Stealth’s CEO Reenie McCarthy said the study is a “key step” in developing therapies for common diseases that occur with 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,” McCarthy said. “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.”

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