Stealth BioTherapeutics, a biopharmaceutical company developing treatments for rare, mitochondria-related diseases, recently announced the initiation of a new clinical study of Bendavia for the treatment of Mitochondrial Myopathy (MM).
Mitochondrial Myopathy (MM) is rare genetic condition that affects the mitochondria, a cell’s “center” responsible for maintaining the body’s energy, causing progressive muscle weakness. The disease leads to significant patient health and wellbeing impairments.
“There are more than 270 orphan mitochondrial diseases and no FDA-approved treatments. The impact these diseases have on quality of life and patient health is very real,” said Dr. Bruce H. Cohen, Director of Pediatric Neurology at Akron Children’s Hospital in a recent news release “By targeting mitochondrial myopathy, we are addressing an important component of these disorders: muscle loss and impaired exercise capacity accompanied by extraordinary fatigue. Mitochondrial myopathy impairs patients’ ability to perform simple daily activities, greatly diminishing their quality of life.”
The trial will investigate Bendavia as a candidate drug that targets the internal mitochondrial membrane, treating both common and rare conditions such as cardio-renal diseases and orphan mitochondrial diseases.
Multiple Phase 1 and phase 2 trials studying Bendavia in mitochondrial conditions have found that the drug is well tolerated. In other non-clinical trials, the drug was shown to modify mitochondrial dysfunction, targeting internal mitochondrial membrane and restoring cellular energy and function. Stealth BioTherapeutics’ MM clinical trial will examine increased doses of the drug in MM patients in several clinical settings in the United States.
“We are aggressively focused on pursuing our orphan MM program in the hopes of providing patients with the first FDA-approved therapy for inherited mitochondrial diseases,” said Chief Executive Officer Travis Wilson in a news release. “We look forward to initiating this trial and evaluating its benefits in this underserved patient population.”
Participants must meet clinical diagnosis for genetic mitochondrial disease with MM and be aged between 16 to 65 years. The trials will assess Bendavia’s safety, tolerability and efficacy.
“This trial is an important step in our pursuit of therapies for children and adults affected by mitochondrial diseases, including MELAS, Leigh’s, MERRF and many other disorders,” said Cristy Balcells, Executive Director at Mitochondrial disease advocacy group MitoAction,
The company has another lead candidate drug called Ocuvia that also has the potential to modify MM through mitochondrial protection. Both Bendavia and Ocuvia are being developed for diseases for which there are no FDA-approved treatment options.
“The current treatment options are limited mainly to vitamins and supplements, so the patient community’s participation in clinical trials is critical. Without trials like this one, there will be no effective patient therapies,” added Charles Mohan, Executive Director of UMDF.
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