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Monepantel - PharmAust

Drug Profile

Monepantel - PharmAust

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

Latest Information Update: 01 Sep 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

  • 20 Aug 2025 Efficacy and adverse events data from a phase I trial in Motor neuron disease released by PharmAust
  • 15 Aug 2025 The US FDA decision on the Clinical Hold Complete Response (CHCR) to the IND application for NUZ 001, for Amyotrophic lateral sclerosis is expected by October 3, 2025
  • 28 Jul 2025 No recent reports of development identified for phase-I development in Amyotrophic-lateral-sclerosis in Australia (PO, Tablet)

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