Drug to Treat Mitochondrial-related Heart Failure Shows Promise in Clinical Trial, Company Reports

Stealth BioTherapeutics presented new clinical trial data during the recent American College of Cardiology’s 65th Annual Scientific Session in Chicago, showing that its lead candidate elamipretide is both safe and well-tolerated in patients with heart failure.

At the April 2–4 meeting, the company also reported demonstrated improvements in major secondary efficacy study measures, and that future trials in this patient population are planned.

Stealth’s sole focus is the development of drugs for the treatment of mitochondrial dysfunction — targeting both rare and common diseases. Their lead drug candidate, elamipretide, previously known under the name Bendavia, has undergone several clinical trials investigating its properties in healthy volunteers and its effects on a variety of diseases.

The Phase 1 trial — named PREVIEW — was a randomized, double-blind, placebo-controlled study exploring dose escalation in 36 heart failure patients with ejection fraction of 35 percent or less. Patients received either a single four-hour infusion of elamipretide in three ascending, weight-adjusted doses, or placebo.

The study’s main aim was to assess the safety and tolerability of elamipretide, while secondary endpoints were improvements in cardiac function measured by echocardiography.

Elamipretide treats conditions linked to mitochondrial dysfunction by preserving energetics, restoring energy production, and decreasing oxidative stress in the mitochondria.

“The heart requires a significant amount of energy to both contract and relax, and targeting the mitochondrial dysfunction affecting the heart’s energy supply in this population is a new approach to treating the disease,” Melissa Daubert of the Duke University Health System, the trial’s primary investigator for echocardiography, said in a press release. “Based on the improvement in heart function seen after just one high-dose treatment, we are very excited to explore the benefits of elamipretide after repeated dosing.”

“The promising findings in the PREVIEW trial support our ongoing development of elamipretide for heart failure patients with significant unmet treatment needs,” Stealth’ chief executive officer, Reenie McCarthy, added. “These findings compel us to study this investigational drug in additional heart failure trials in the coming months for patients with either reduced or preserved ejection fraction, including a study for patients hospitalized due to worsening heart failure. These trials will help further our understanding of elamipretide’s potential to treat the failing heart.