Bitter setback for Seelos’ injectable sugar in mid-stage ALS study

2024-03-20
Firstwordpharma
临床2期临床结果细胞疗法
Seelos Therapeutics’ shares plunged as much as 51% on Tuesday after a mid-stage study of its investigational amyotrophic lateral sclerosis (ALS) therapy met with failure. However, the stock recovered some of its losses, ending the day down 29%, with the company hoping that results seen in a subgroup of patients offer a path forward.
SLS-005, an intravenous formulation of the low molecular weight disaccharide trehalose, failed to meet key primary and secondary endpoints of the study, but signalled potential efficacy within patients not treated with Amylyx Pharmaceuticals' Relyvrio (sodium phenylbutyrate/taurursodiol). “We believe the observed signal and success probability is competitive to other recently FDA-approved therapies for ALS which also failed…on similar primary and efficacy endpoints," remarked CEO Raj Mehra.
Relyvrio use muddies data
Seelos’ therapy was evaluated as part of the Phase II/III HEALEY ALS Platform trial, whose primary endpoint was the change in ALS Functional Rating Scale (ALSFRS-R) slope, a measure of disease severity. Top-line findings showed that SLS-005 failed to reach statistical significance in both primary and secondary endpoints versus placebo, demonstrating a 13% improvement in function and mortality with an 88% success probability, well below the pre-specified 98%. The company suggested that 31 patients received Relyvrio, “confounding” results from this population.
However, in a prespecified subgroup of subjects who were not treated with Relyvrio, SLS-005 was associated with a 22% improvement on the ALSFRS-R slope change and a 25% reduction in respiratory function decline compared with placebo. Seelos noted that while SLS-005 was well tolerated in the study, an imbalance in deaths or death-related events was observed, although this was determined to be unrelated to the drug.
Mehra indicated that Seelos intends to meet with the FDA to discuss the future programme for SLS-005, and engage with potential partners.
ALS field struck by setbacks
Several defeats have rattled the ALS arena in the near past, the most recent one being Relvyrio’s impending market exit following failure in the confirmatory PHOENIX study. For more, see Physician Views Results: Relyvrio setback leaves ALS docs on uncertain ground.
Meanwhile, in February, Denali and Sanofi’s experimental RIPK1 inhibitorRIPK1 inhibitor also failed to meet the primary endpoint in the Phase II HIMALAYA study. Additionally, BrainStorm Cell Therapeutics' ALS cell therapy NurOwn was downvoted by an FDA advisory panel in September last year, owing to lack of sufficient evidence.
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