On Monday, Denali Therapeutics Inc. DNLI revealed topline outcomes from an evaluation of Routine G of the Part 2/3 HEALEY ALS Platform Trial evaluating DNL343 for amyotrophic lateral sclerosis.
In comparison with placebo, the research didn’t meet the first endpoint of efficacy in slowing illness development.
The first endpoint was evaluated as a change in illness severity over time as measured by the ALS Practical Score Scale-Revised (ALSFRS-R) and survival by week 24.
Additionally Learn: Denali Therapeutics Neurodegenerative Illness Platform Sparks Analyst Optimistic
Key secondary endpoints, measuring muscle power and respiratory operate, have been additionally not statistically completely different between the lively and placebo teams at week 24.
For the first evaluation, 186 members who have been randomized to obtain DNL343 remedy have been in comparison with 139 members randomized to obtain placebo on this routine (n=63) or shared from a concurrently enrolling routine (n=76).
Total, DNL343 was discovered to be secure and well-tolerated. Additional analyses are anticipated later in 2025, together with neurofilament mild (NfL) and different fluid biomarkers, information from pre-specified subgroups, and prolonged findings from the lively remedy extension interval.
William Blair famous that whereas the replace is disappointing for sufferers and buyers, it isn’t completely surprising. That is because of the quick remedy interval and the complexity of the illness, which has a historical past of medical trial challenges.
Biogen Inc. BIIB and Ionis Prescription drugs, Inc. IONS confronted an analogous state of affairs with Qalsody, which did not present vital enchancment in ALSFRS-R scores over six months. Nonetheless, it will definitely acquired accelerated approval based mostly on lowered NfL ranges and indications of profit with early remedy over 52 weeks.
Analyst Sarah Schram believes it’s too quickly to desert this system and is awaiting a extra detailed evaluation, anticipated in late 2025.
Worth Motion: DNLI inventory is up 7.29% at $21.27 on the final verify on Tuesday.
Learn Subsequent:
Market Information and Knowledge delivered to you by Benzinga APIs
© 2025 Benzinga.com. Benzinga doesn’t present funding recommendation. All rights reserved.