STEALTH BIOTHERAPEUTICS CORP Stealth BioTherapeutics Corp. announced the presentation of new SBT-272 preclinical data demonstrating functional improvement in upper motor neurons with TDP-43 pathology, which plays a significant role in both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The data were presented at the Keystone Neurodegeneration Symposium in Keystone, CO held June 5-9, 2022.
A Phase 1 study to evaluate the safety and tolerability of SBT-272 in healthy volunteers is underway. 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 AMX0035 (NDA 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. Together, these data support further investigation of SBT-272 as a potential treatment for ALS with TDP-43 pathology.
