Denali Therapeutics announces completion of enrollment for regimen G evaluating eIF2B agonist DNL343 in the phase 2/3 HEALEY ALS platform trial. Amyotrophic lateral sclerosis (ALS) causes progressive nerve degeneration in the spinal cord and brain, representing a large unmet medical need. The activation of integrated stress response (ISR) is documented in ALS patients, resulting in the suppression of eukaryotic translation initiation factor 2B (eIF2B) activity, and impaired protein synthesis.
Denali's ALS therapy DNL343 is an investigational small molecule eIF2B activator. It is designed to restore protein synthesis and inhibit ISR pathway in ALS. The compound is found to be generally well-tolerated and support once-daily dosing.