Stealth BioTherapeutics Showcases New Nonclinical Data From Duchenne Muscular Dystrophy Program

Stealth BioTherapeutics Corp MITO 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) animal model. 

• The data were presented at the 2022 Muscular Dystrophy Association and Clinical and Scientific Conference.
• Mitochondrial dysfunction has been observed early in the progression of Duchenne muscular dystrophy (DMD).
• It contributes to impaired energy homeostasis and inflammatory signaling in DMD. 
• Read Next: Stealth Bio Stock Falls On FDA Refusal to File Letter For Barth Syndrome Candidate.
• As PMO efficacy has been shown to be energy-dependent, the study tested the hypothesis that improving mitochondrial function with the mitochondria-targeting 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 compared to PMO alone. 
• The data highlight the PMO/elamipretide combo therapy as a potential treatment for patients with DMD. 
• In December, Stealth Bio concluded the pre-IND meeting for the DMD development program, including aligning on Stealth's proposal to assess the progression of myocardial fibrosis as a surrogate endpoint for its planned clinical trial.
• Price Action: MITO shares closed 11.1% at $0.64 during after-hours trading on Wednesday.