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Monepantel - PharmAust

Drug Profile

Monepantel - PharmAust

Alternative Names: NUZ-001; PPL-1; S-Monepantel

Latest Information Update: 08 Oct 2025

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At a glance

  • Originator Pitney Pharmaceuticals
  • Developer Neurizon Therapeutics; PharmAust
  • Class Acetonitriles; Anthelmintics; Antineoplastics
  • Mechanism of Action Cyclin-dependent kinase 2 modulators; Cyclin-dependent kinase 4 modulators; TDP-43 protein inhibitors
  • Orphan Drug Status

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

    Yes - Amyotrophic lateral sclerosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Solid tumours
  • Phase I Motor neuron disease
  • Preclinical Amyotrophic lateral sclerosis; Huntington's disease

Most Recent Events

  • 06 Oct 2025 US FDA lifts the clinical hold for monepantel for Amyotrophic lateral sclerosis in USA
  • 06 Oct 2025 Neurizon Therapeutics plans the phase II/III HEALEY ALS trial for Amyotrophic lateral sclerosis in USA (PO), in the fourth quarter of 2025
  • 20 Aug 2025 Efficacy and adverse events data from a phase I trial in Motor neuron disease released by PharmAust

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