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Trehalose - Seelos Therapeutics

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Drug Profile

Trehalose - Seelos Therapeutics

Alternative Names: Cabaletta; SLS-005; Trehalose dihydrate

Latest Information Update: 08 May 2024

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At a glance

  • Originator Bioblast Pharma
  • Developer Bioblast Pharma; Mashhad University of Medical Sciences; Massachusetts General Hospital; Seelos Therapeutics; Team Sanfilippo Foundation
  • Class Antidementias; Disaccharides; Glucans; Pyrrolidines; Small molecules
  • Mechanism of Action Autophagy stimulants; Protein aggregation inhibitors; Transcription factor stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Machado-Joseph Disease; Spinocerebellar ataxias; Oculopharyngeal muscular dystrophy; Amyotrophic lateral sclerosis; Mucopolysaccharidosis III
  • New Molecular Entity No

Highest Development Phases

  • Phase III Oculopharyngeal muscular dystrophy
  • Phase II/III Amyotrophic lateral sclerosis; Spinocerebellar ataxias
  • Phase II Huntington's disease; Machado-Joseph Disease; Mucopolysaccharidosis III
  • Preclinical Alzheimer's disease; Neurodegenerative disorders
  • Discontinued X-linked bulbo-spinal atrophy

Most Recent Events

  • 22 Apr 2024 Sellos Therapeutics completes an expanded-access programme in Amyotrophic lateral sclerosis in USA (IV) (NCT05597436)
  • 19 Mar 2024 Seelos Therapeutics plans to request a meeting with US FDA to discuss steps following failure to meet phase II/III HEALEY ALS trial's primary and secondary endpoint
  • 19 Mar 2024 Efficacy data from a phase II/III trial HEALEY ALS trial in Amytrophic lateral scelrosis released by Seelos

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