NS 065

Drug Profile

NS 065

Alternative Names: Exon 53 skipping antisense oligonucleotide - Nippon Shinyaku/National Center of Neurology and Psychiatry; Exon 53 skipping DMD therapeutic - Nippon Shinyaku/National Center of Neurology and Psychiatry; NCNP-01; NS-065/NCNP-01; NS065

Latest Information Update: 10 Sep 2017

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At a glance

  • Originator National Center of Neurology and Psychiatry; Nippon Shinyaku
  • Class Antisense oligonucleotides
  • Mechanism of Action Dystrophin expression stimulants
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Duchenne muscular dystrophy

Most Recent Events

  • 06 Jul 2017 NS Pharma initiates enrolment in an extension phase II trial for Duchenne muscular dystrophy (In children) in Canada and USA (NCT03167255)
  • 29 May 2017 NS Pharma plans a phase II trial for Duchenne muscular dystrophy(In children) in Canada and USA (NCT03167255)
  • 01 Feb 2017 NS 065 receives rare paediatric disease designation for Duchenne muscular dystrophy in USA
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