Denali Therapeutics Inc. announced that its eIF2B agonist DNL343 failed to meet the primary endpoint in Regimen G of the Phase 2/3 HEALEY ALS Platform Trial for amyotrophic lateral sclerosis (ALS). The study did not demonstrate efficacy in slowing disease progression compared to placebo.
The primary endpoint, which evaluated the change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and survival through week 24, was not met. Key secondary endpoints, assessing muscle strength and respiratory function, also showed no statistically significant difference between the active treatment and placebo groups at week 24.
A total of 186 participants were randomized to receive DNL343, while 139 participants received placebo in this regimen or were shared from a concurrently enrolling regimen. Despite the lack of efficacy, DNL343 was found to be safe and generally well tolerated. Further analyses, including neurofilament light (NfL) and other fluid biomarkers, pre-specified subgroups, and extended findings from the active treatment extension period, are anticipated later in 2025.
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