Positive Data on Primary Mitochondrial Myopathy Drug Candidate Elamipretide (Bendavia) Presented by Stealth BioTherapeutics

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Positive data on primary mitochondrial myopathy drug elamipretide

Stealth BioTherapeutics recently reported positive data from its Phase 2 MMPOWER clinical trial, a double-blind, placebo-controlled study that evaluated the safety, tolerability, and efficacy of elamipretide (Bendavia) in patients with mitochondrial myopathy (MM).

The MMPOWER study investigated Bendavia for the treatment of myopathy (muscle weakness) in patients with genetic mitochondrial diseases. These diseases are a diverse group of rare inherited disorders characterized by systemic mitochondrial dysfunction that impairs patient health and well-being.

Stealth BioTherapeutics’ Bendavia is an investigational drug that targets the inner mitochondrial membrane, and developed for the treatment of several conditions, including cardiorenal diseases and orphan mitochondrial diseases. The drug has the potential to preserve energetics and restore normal energy production in mitochondria while reducing oxidative stress.

The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to Bendavia in January 2016 as a treatment option for primary mitochondrial myopathy in patients with genetic mitochondrial diseases.

Data from the MMPOWER trial will be presented on June 17 at the Mitochondrial Medicine 2016, the United Mitochondrial Disease Foundation (UMDF) symposium June 15-18 in Seattle.

The company recently presented results from the MMPOWER trial at the BIO International Convention at the Moscone Center in San Francisco, where researchers and biotechnology and pharma leaders discussed the opportunities and challenges inherent to mitochondrial diseases, the current state of drug development, as well as the mitochondrial research potential for other conditions, including aging.

At the convention, Mark Bamberger, the company’s chief scientific officer, spoke during the session “Drug Development for Mitochondrial Disease: Examining the Current Landscape and Scientific/Regulatory Gaps.

“Mitochondrial medicine has the potential to address numerous rare and common diseases, and as a leader in the space, we are thrilled to participate in these meetings to help bring this conversation to the broader healthcare community and to share updates with those already in the field,” said Stealth BioTherapeutics’ CEO, Reenie McCarthy, according to a recent press release.

“We especially look forward to presenting positive results from the MMPOWER trial and our ongoing development plans for elamipretide in rare mitochondrial diseases, for which there are currently no FDA-approved treatments.”

Stealth BioTherapeutics plans to begin the MMPOWER-2 clinical trial in patients who took part in the MMPOWER study in the second half of 2016.