The Food and Drug Administration (FDA) has granted Orphan Drug designation to SLS-005 (trehalose; Seelos Therapeutics) for the treatment of amyotrophic lateral sclerosis (ALS).

SLS-005 contains the active ingredient trehalose, a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and activates autophagy. In preclinical studies, SLS-005 was found to increase clearance of TDP-43, decrease SOD1 and SQSM1/p62 aggregates and monomers, preserve ventral horn motor neurons, and increase muscle fiber size.

A double-blind, placebo-controlled phase 2b/3 trial will investigate SLS-005 in approximately 160 patients with either familial or sporadic ALS. Patients will be randomized 3:1 to receive SLS-005 or placebo. The change from baseline on Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score at 24 weeks has been designated as the primary outcome measure. Secondary end points include the change from baseline in slow vital capacity, muscle strength, quality of life measurements, and additional signs of disease progression.

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals. The designation was previously granted to SLS-005 for Sanfilippo syndrome, spinocerebellar ataxia type 3 and oculopharyngeal muscular dystrophy.


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References

  1. Seelos Therapeutics receives US Orphan Drug designation for SLS-005 (trehalose) in amyotrophic lateral sclerosis. [press release]. New York, NY: Seelos Therapeutics, Inc; November 19, 2020. 
  2. Seelos Therapeutics receives FDA may proceed notice to initiate a phase IIb/III trial of SLS-005 in amyotrophic lateral sclerosis. [press release]. New York, NY: Seelos Therapeutics, Inc; August 10, 2020.