Stealth BioTherapeutics Corp (NASDAQ: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.
