Stealth BioTherapeutics Corp, a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today presented new data demonstrating the potential synergistic relationship between elamipretide and an exon skipping phosphorodiamidate morpholino oligomer (PMO) in the X-linked muscular dystrophy (mdx) mouse model. The data were presented at the 2022 Muscular Dystrophy Association and Clinical Scientific Conference, held in Nashville, TN.
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Mitochondrial dysfunction has been observed early in the progression of Duchenne muscular dystrophy (DMD) and contributes to impaired energy homeostasis and inflammatory signaling in DMD. As PMO efficacy has been shown to be energy-dependent, the study tested the hypothesis that improving mitochondrial function with the mitochondria-targeting investigational new product elamipretide would enhance PMO efficacy. Results from the 7-week study demonstrated that the combination of weekly PMO and daily elamipretide therapies produced more than double the mean level of dystrophin protein in muscles of affected mice compared to treatment with PMO alone. These data highlight combination therapy of a PMO and elamipretide as a potential treatment for patients with DMD.
“We believe that improved mitochondrial health may lead to better outcomes in DMD, where dystrophin deficiency and mitochondrial dysfunction contribute to the devastating and progressive decline in muscle and cardiac function for young patients,” said Reenie McCarthy, Chief Executive Officer of Stealth. “These data demonstrating the promise of combined elamipretide and PMO therapies offer early support for this premise, energizing our efforts to develop improved therapeutic options for DMD patients.”
We are a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body’s main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases and are involved in many common age-related diseases, typically involving organ systems with high energy demands such as the eye, the neuromuscular system, the heart and the brain. We believe our lead product candidate, elamipretide, has the potential to treat ophthalmic diseases entailing mitochondrial dysfunction, such as dry age-related macular degeneration, rare neuromuscular disorders, such as primary mitochondrial yopathy and DMD, and rare cardiomyopathies, such as Barth syndrome