- Stealth BioTherapeutics Corp MITO presented new SBT-272 preclinical data demonstrating functional improvement in upper motor neurons with TDP-43 pathology.
- TDP-43 pathology plays a significant role in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
- The data were presented at the Keystone Neurodegeneration Symposium.
- A Phase 1 study to evaluate the safety and tolerability of SBT-272 in healthy volunteers is underway.
- Read Next: Stealth Bio Posts Disappointing Elamipretide Data From Mid-Stage Geographic Atrophy Study.
- The data demonstrated that SBT-272 significantly improved mitochondrial structural integrity and motility in TDP-43 mutant (A315T)-expressing upper motor neurons.
- These enhancements in mitochondrial health were associated with improved axon outgrowth, a key indicator of improved neuronal health.
- The effect of SBT-272 on axon outgrowth was superior to edaravone (approved for the treatment of ALS) and Amylyx Pharmaceuticals Inc AMLX AMX0035 (under FDA review).
- Chronic in vivo administration of SBT-272 reduced upper motor neuron degeneration and neuroinflammation in the motor cortex of the prp‐hTDP‐43A315T‐UeGFP mouse model of ALS.
- Price Action: MITO shares are up 6.60% at $0.27 on the last check Wednesday.
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