Monepantel - PharmAust
Alternative Names: NUZ-001; PPL-1; S-MonepantelLatest Information Update: 08 Oct 2025
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
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Orphan Drug Status
Yes - Amyotrophic lateral sclerosis
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- 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