Seelos Therapeutics has dosed the first subject in an open-label basket study of an investigational therapy, SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion), to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) patients in Australia.
A low molecular weight disaccharide (0.342 kDa), SLS-005 can cross the blood-brain barrier.
It is believed to stabilise proteins and trigger autophagy by activating Transcription Factor EB (TFEB), a crucial factor in lysosomal and autophagy gene expression.
The open-label basket study in Australia will analyse SLS-005’s impact on disease progression and severity.
It will also assess the safety and tolerability of the therapy in subjects with ALS, spinocerebellar ataxia and Huntington’s disease.
Furthermore, a study of SLS-005 on the HEALEY ALS Platform is anticipated to conclude subject enrolment in the current quarter of this year with top-line data expected in the middle of next year.
Seelos Therapeutics chairman and CEO Raj Mehra said: “We hope to gain valuable insights into the activity of SLS-005 in several devastating neurodegenerative diseases and the open-label design provides a degree of transparency not possible in a blinded study.
“Today we are also very excited to share that the registrational study in the HEALEY platform trial is actively enrolling and at its current pace of enrolment, we currently expect it to be fully enrolled by the end of this quarter.”
SLS-005 demonstrated to lower misfolded protein and pathologic material buildup in animal models of various ailments linked to abnormal cellular protein accumulation or storage of pathologic material.
A group of rare neurological diseases, amyotrophic lateral sclerosis (ALS) chiefly impacts neurons that control voluntary muscle movement.
In January last year, Seelos dosed the first subjects in its registrational ‘Proof of Concept’ study to analyse SLS-002 (intranasal racemic ketamine) for patients with acute suicidal ideation and behaviour in major depressive disorder.