Seelos Therapeutics Doses First Patient in A Registrational Phase II/III Study of SLS-005 in Spinocerebellar Ataxia
CT. Seelos' Phase II/III trial (NCT05490563) plans to enroll up to 245 participants globally with spinocerebellar ataxia type-3 in a double-blind, randomized, placebo-controlled trial. Eligible participants will be randomized to treatment
with SLS-005 or placebo in 1 of 2 dosage arms and assessed with a primary efficacy endpoint measuring change from baseline in the Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52. Secondary endpoints include change from baseline in a blood-based biomarker for neurodegeneration, clinical global impression of severity, patient global impression of severity and an activities of daily living score. Safety and tolerability of SLS-005 will be monitored and assessed throughout the trial. About SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) SLS-005 is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier and is thought to stabilize proteins and activate autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material. In animal models of several diseases associated with abnormal cellular protein aggregation or storage of pathologic material, SLS-005 has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. SLS-005 has previously received Orphan Drug Designation for the treatment of SCA type 3 from the U.S. Food and Drug Administration and from the European Medicines Agency in the EU. SLS-005 is an investigational treatment and is not currently approved by any health authority for medicinal use.