Seelos Shares Preliminary ALS Study Results for SLS-005

Seelos Therapeutics, a biopharmaceutical firm at the clinical stage, is dedicated to crafting remedies for central nervous system disorders and uncommon diseases. The company has recently shared preliminary findings from its Phase 2/3 HEALEY ALS Platform trial, conducted in partnership with The Sean M. Healey and AMG Center, known for their significant contributions to ALS research and support.

The research aimed to assess the effects of SLS-005 (IV trehalose), a low molecular weight disaccharide with the potential to stabilize misfolded proteins and stimulate autophagy, on the ALS Functional Rating Scale (ALSFRS-R) and its ability to differentiate from a placebo in terms of functionality and mortality among a diverse ALS patient population.

Although the study did not achieve statistical significance for its primary and secondary endpoints within the Full Analysis Set (FAS), it did reveal a 13% improvement in Function and Mortality with a success probability of 88%, falling short of the expected 98%. However, a promising indication of efficacy was observed within a pre-defined subgroup (ERF).

Among the ERF subgroup of ALS patients treated exclusively with SLS-005, the data showed a 22% improvement in the ALSFRS-R assessment's slope adjusted for mortality after 24 weeks, with an 89% success probability. Additionally, the rate of decline in ALSFRS-R slope over six months favored the SLS-005 group, with a 25% reduction in the decline of Slow Vital Capacity (SVC) compared to the placebo group.

Raj Mehra, Ph.D., CEO of Seelos, expressed that the HEALEY platform is intended to identify efficacy signals, and the observed signal and success probability are competitive with other recent ALS therapies approved by the FDA, which also did not achieve statistical significance on similar primary endpoints.

Seelos is yet to receive the complete data set, and upon doing so, intends to conduct further analyses, including neurodegeneration biomarkers and other exploratory results. SLS-005 demonstrated a good safety profile, with tolerability comparable to that of a placebo. However, there was noted an imbalance in deaths/death equivalents during the study, with a higher number in the SLS-005 group, though these were deemed unrelated to the study drug.

The study's populations were categorized into ERF1, FAS2, and ERO3, with the ERF population representing those who adhered strictly to the study protocol. SLS-005, an investigational treatment, has received Orphan Drug Designation for ALS treatment from both the U.S. and European regulatory authorities but is not yet approved for medical use.

ALS is a collection of rare neurological disorders that primarily affect the neurons controlling voluntary muscle movement. The disease is progressive, with most cases being sporadic. Despite the severity of ALS, there is currently no cure or treatment capable of stopping or reversing the disease's progression. A slight change in the ALSFRS-R score can significantly reflect a patient's ability to perform daily activities independently.

Seelos Therapeutics is advancing novel therapeutics to meet unmet medical needs, with a portfolio that includes late-stage clinical assets for conditions like Major Depressive Disorder, ALS, and spinocerebellar ataxia, alongside early-stage research into Huntington's, Alzheimer's, and Parkinson's diseases.