Denali Therapeutics’ ALS misses pivotal trial, but analysts say that’s expected – Denali Therapeutics (NASDAQ:DNLI)

on Monday, Denali Therapeutics DNLI Revealing key results from the Protocol G analysis of the Phase 2/3 HEALEY ALS platform trial evaluating DNL343 in amyotrophic lateral sclerosis.

The study did not meet its primary efficacy endpoint of slowing disease progression compared with placebo.

The primary endpoints were change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and survival at week 24.

Also Read: Denali Therapeutics Neurodegenerative Disease Platform Sparks Analyst Optimism

There were also no statistical differences between the active and placebo groups at week 24 in key secondary endpoints measuring muscle strength and respiratory function.

In the primary analysis, 186 participants randomized to receive DNL343 were compared with 139 participants randomized to receive placebo in the regimen (n=63) or 139 participants who shared concurrent enrollment (n=76) Compare.

Overall, DNL343 was found to be safe and well-tolerated. Further analyzes are expected later in 2025, including neurofilament light (NfL) and other fluid biomarkers, data from pre-specified subgroups, and expanded findings from the active treatment extension phase.

William Blair noted that while the update was disappointing to patients and investors, it wasn’t entirely unexpected. This is due to the short treatment duration and complex disease, which have historically faced clinical trial challenges.

Biogen BIIB and Ioannis Pharmaceuticals ion Qalsody faced a similar situation, with the company failing to show significant improvements in ALSFRS-R scores over six months. However, the drug ultimately received accelerated approval based on reduced NfL levels and signs of benefit from early treatment at 52 weeks.

Analyst Sarah Schram believes it is too early to abandon the plan and is waiting for a more detailed analysis (expected to be by the end of 2025).

Price Action: DNLI shares were up 7.29% at $21.27 as of last check on Tuesday.

Read next: