Seelos Updates on ALS Trial with SLS-005

Seelos Therapeutics, a biopharmaceutical firm dedicated to developing treatments for central nervous system conditions and rare diseases, recently shared preliminary findings from its Phase 2/3 HEALEY ALS Platform trial. The research, conducted in partnership with The Sean M. Healey and AMG Center, a prominent ALS research and patient care entity, evaluated SLS-005 (IV trehalose), a treatment designed to stabilize misfolded proteins and stimulate autophagy.

The trial aimed to assess the drug's impact on the ALS Functional Rating Scale (ALSFRS-R) slope and its differentiation from a placebo in terms of functionality and survival rates among a diverse ALS patient population. Although the study did not achieve statistical significance for its primary and secondary endpoints, it indicated a potential therapeutic effect in a specific subgroup.

Among patients treated with SLS-005, excluding those on RELYVRIO®, the data showed a 22% improvement in ALSFRS-R assessment slope adjusted for mortality after 24 weeks, with a success probability of 89%. Additionally, the rate of decline in ALSFRS-R was slower in the SLS-005 group compared to the placebo over a six-month period. The drug also demonstrated a 25% reduction in the decline of Slow Vital Capacity (SVC) compared to a placebo at 24 weeks.

Raj Mehra, CEO of Seelos, noted that the platform's design is to identify efficacy signals, and the observed results are competitive with other recently approved ALS therapies that also did not reach statistical significance in similar primary efficacy endpoints. Seelos intends to engage with the FDA to discuss future steps for the program and is considering potential partnerships.

The company has yet to receive the complete data set but plans to conduct further analyses once it is obtained. These will include assessments of neurodegeneration biomarkers, neurofilament light chain (NfL), exploratory efficacy, subgroups, and post-hoc analyses. SLS-005 was found to be well-tolerated and safe, with no significant difference from the placebo. However, there was a noted imbalance in deaths between the SLS-005 group and the placebo group, which were deemed unrelated to the drug.

The study's populations were categorized into Full Analysis Set (FAS), Efficacy Regimen Only (ERO), and Efficacy RELYVRIO® Free (ERF) groups. The ERF group, which excluded participants who started RELYVRIO® during the study, is considered to represent the protocol's intended participants.

SLS-005 is a disaccharide that can cross the blood-brain barrier and is hypothesized to stabilize proteins and activate autophagy by triggering Transcription Factor EB (TFEB), a crucial element in lysosomal and autophagy gene expression. It has received Orphan Drug Designation for ALS treatment from both the U.S. FDA and the European Medicines Agency, though it is still under investigation and not yet approved for medical use.

Amyotrophic Lateral Sclerosis (ALS) is a rare group of neurological disorders affecting motor neurons, leading to muscle weakness, twitching, and atrophy. The disease is progressive, with most cases being sporadic and without a clear risk factor or family history. The majority of ALS patients die from respiratory failure within 3 to 5 years of symptom onset, although a small percentage survive for a decade or more. Currently, there is no cure or effective treatment to stop or reverse ALS progression. A change of one point in the ALSFRS-R score can significantly affect a patient's independence in daily activities.

Seelos Therapeutics is in the clinical stage of development, focusing on novel treatments for CNS disorders and other rare diseases. Their portfolio includes assets in late-stage clinical trials for conditions such as Acute Suicidal Ideation and Behavior in Major Depressive Disorder, ALS, and spinocerebellar ataxia, as well as early-stage research for Huntington's disease, Alzheimer's disease, and Parkinson's disease.