Seelos Therapeutics provides update on top-line data of Phase 2/3 trial

Seelos Therapeutics provided an update on top-line data of the Phase 2/3 HEALEY ALS Platform trial. The company plans to request a meeting with the FDA to discuss potential next steps for the programThe study was designed to evaluate SLS-005, a low molecular weight disaccharide that stabilizes misfolded proteins and activates autophagy, in decreasing the slope of the ALS Functional Rating Scale and separation from placebo in Function and Mortality in an all-comers population of Persons with ALS. While the study did not meet statistical significance in the primary and secondary endpoint in the Full Analysis Set, by showing a 13% improvement in Function and Mortality with an 88% success probability, vs. the pre-specified 98%, it showed a potential signal of efficacy in a pre-specified subgroup. In the pre-specified subgroup of PALS treated with SLS-005, without Relyvrio, the top-line data favored SLS-005 versus placebo in efficacy measures in the Efficacy Relyvrio Free data set, including: a 22% improvement in slope of change in ALSFRS-R assessment adjusted for mortality, with an 89% success probability, at 24 weeks the rate of decline in ALSFRS-R slope also favored the SLS-005 treatment group versus placebo over 6 months; a 25% slowing of Slow Vital Capacity decline versus placebo at 24 weeks. Seelos plans to run additional analyses, including biomarkers of neurodegeneration, neurofilament light chain, exploratory efficacy results, subgroups and post-hoc analyses. SLS-005 was generally well-tolerated and comparable to placebo in safety. There was an imbalance of deaths/death equivalents observed in the study, with more events seen in the SLS-005 group compared to placebo, all were considered unrelated to the study drug.