The Food and Drug Administration (FDA) has accepted the Investigational New Drug Application and granted Fast Track designation to SLS-005 (trehalose) for the treatment of spinocerebellar ataxia (SCA).
SLS-005 contains the active ingredient trehalose, a low molecular weight disaccharide (0.342 kDa) that has been shown to penetrate muscle and cross the blood brain barrier. SCA is a rare, genetic disorder caused by degeneration of the cerebellum and characterized by a loss of coordination, abnormal speech, involuntary eye movement, and cognitive dysfunction.
The designation is supported by data from an exploratory, randomized, parallel-group, dose-escalation and dose-controlled phase 2a study (ClinicalTrials.gov Identifier: NCT02147886), which evaluated 14 patients with SCA type 3 over a 6-month period. Patients were randomly assigned 1:1 to receive either 15g or 30g of trehalose via intravenous infusion once weekly for 24 weeks.
Results found that on average, the Scale for Assessment and Rating of Ataxia (SARA) remained stable in all patients at 6 months; 6 patients who received treatment for 12 months continued to maintain stable SARA scores. Normally, during the natural progression of the disease, patients with SCA3 would see an increase on SARA within 12 months, indicating worsening symptoms.
The Company expects to initiate a global phase 2b/3 placebo-controlled study evaluating SLS-005 in patients with SCA in early 2022.
“SLS-005 has already displayed encouraging open label human data in SCA3, the most common type of SCA, and our team has taken that experience and knowledge into the design and plans for our global phase 2b/3 placebo-controlled study,” said Raj Mehra, PhD, Chairman and CEO of Seelos.
The FDA previously granted Orphan Drug designation to SLS-005 for SCA type 3.
Seelos Therapeutics announces FDA acceptance of Investigational New Drug Application and grant of Fast Track designation for SLS-005 (IV trehalose) for the treatment of spinocerebellar ataxia. News release. Seelos Therapeutics. Accessed November 8, 2021. https://www.prnewswire.com/news-releases/seelos-therapeutics-announces-fda-acceptance-of-investigational-new-drug-application-and-grant-of-fast-track-designation-for-sls-005-iv-trehalose-for-the-treatment-of-spinocerebellar-ataxia-301417731.html