Seelos Therapeutics has dosed the first subject in a Phase II/III clinical trial of its investigational treatment SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for spinocerebellar ataxia, with a focus on type3 patients (SCA3; Machado-Joseph disease).

The randomised, double-blind, placebo-controlled, registrational trial intends to enrol up to 245 SCA3 patients across the globe.

These subjects will be categorised into one of two dosage arms to receive either SLS-005 or placebo.

Evaluating the variation from baseline in the Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52 is the trial’s primary efficacy endpoint.

The variation from baseline in a blood-based biomarker for neurodegeneration, clinical global impression of severity, subject global impression of severity and a day-to-day living activities score will comprise the secondary endpoints.

Additionally, the trial will analyse the safety and tolerability of SLS-005 in SCA3 patients.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

A low molecular weight disaccharide, SLS-005 can cross the blood-brain barrier.

It could potentially stabilise proteins and activate autophagy by activating Transcription Factor EB (TFEB), an evolving treatment target for various ailments with pathologic storage material build-up. 

The product demonstrated to lower the build-up of misfolded proteins and cut down pathologic material accumulation in animal models of various ailments linked to abnormal cellular protein accumulation or storage of pathologic material.

Earlier, the US Food and Drug Administration (FDA) and the European Medicines Agency granted Orphan Drug Designation for SLS-005 to treat SCA type 3.

A serious ailment, spinocerebellar ataxia is caused by cerebellum degeneration beginning typically in adult life. 

In July this year, the company dosed the first participant in an open-label basket study of SLS-005 to treat patients with amyotrophic lateral sclerosis in Australia.