Seelos Therapeutics Inc. (NASDAQ: SEEL) has announced the dosing of its first participants in the registrational phase 2/3 study evaluating the experimental treatment SLS-005 for amyotrophic lateral sclerosis treatment on the HEALEY ALS Platform Trial.
Seelos initiates HEALEY ALS Platform Study
CEO and Chairman of Seelos Raj Mehra stated, “Initiating this trial is a major achievement for Seelos, and we are honored to be part of the HEALEY ALS Platform Trial. We look forward to offering this investigational therapy to people suffering from this debilitating disease.”
In Seelos’ Phase 2/3 trial (NCT05136885), 160 people with familial or sporadic ALS will be enrolled in a double-blind placebo-controlled study. Subjects will be randomized 3:1 (drug: placebo), and the primary outcome will be the change in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score from baseline after 24 weeks. In addition, at 24 weeks, secondary outcomes such as baseline values in slow vital function, muscular endurance, quality of life assessments, and further indicators of disease progression will be assessed.
Principal HEALEY ALS Platform Trial Investigator Merit Cudkowicz stated, “We are thankful to all the patients with ALS who participate in the HEALEY ALS Platform Trial and help develop new treatments in a much faster and more efficient approach. Partnering with Seelos to determine the efficacy of SLS-005 in this platform trial will give answers sooner because of the sharing of data and infrastructure with other regimens in the platform trial.”
SLS-005 crosses the brain barrier to stabilize proteins and stimulate autophagy
SLS-005 is a low molecular weight disaccharide that passes the blood-brain barrier. It is thought to normalize proteins and stimulate autophagy by activating Transcription Factor EB (TFEB), a crucial component in lysosomal and autophagy gene regulation. Stimulation of TFEB is a promising therapeutic option for various disorders characterized by pathologic buildup of storage material. In addition, SLS-005 has been found to diminish misfolded protein agglomeration and pathologic material buildup. SLS-005 was previously granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency for the treatment of ALS.
