Tuesday, Seelos Therapeutics Inc SEEL provided an update on top-line data of the Phase 2/3 HEALEY ALS Platform trial.
The study was designed to evaluate SLS-005 (IV trehalose), a low molecular weight disaccharide that stabilizes misfolded proteins and activates autophagy, in decreasing the slope of the ALS Functional Rating Scale (ALSFRS-R) and separation from placebo in Function and Mortality in an all-comers population of Persons with ALS (PALS).
The company said that while the study did not meet statistical significance in the primary and secondary endpoint in the Full Analysis Set (FAS), it showed a 13% improvement in Function and Mortality with an 88% success probability (versus the pre-specified 98%), a potential signal of efficacy in a pre-specified subgroup (ERF).
In the pre-specified subgroup of PALS treated with SLS-005, without Amylyx Pharmaceuticals Inc AMLX Relyvrio, the top-line data favored SLS-005 versus placebo in efficacy measures in the Efficacy RELYVRIO Free (ERF) data set (n=130), including:
- 22% improvement in the 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 (points per month) also favored the SLS-005 treatment group versus placebo over six months (-0.80 and -1.07 points per month, respectively).
- 25% slowing of Slow Vital Capacity (SVC) decline versus placebo (-11.5% for SLS-005 and -15.4% for placebo) at 24 weeks.
Seelos awaits receiving full data sets and it 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.
Price Action: SEEL shares are down 54.2% at $0.42 on the last check Tuesday.
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