NNZ-2591
Also known as: cGP analogue, cyclo-glycine-proline analog, NNZ2591
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Summary
NNZ-2591 is an orally bioavailable small cyclic dipeptide being developed by Neuren Pharmaceuticals for multiple pediatric neurodevelopmental disorders including Phelan-McDermid syndrome, Pitt-Hopkins syndrome, Angelman syndrome, and Rett syndrome. It has demonstrated neuroprotective and pro-cognitive effects in preclinical models and has entered Phase 2 clinical trials.
Mechanism of Action
NNZ-2591 is a synthetic analog of the endogenous cyclic dipeptide cyclo-glycine-proline (cGP), a metabolite of IGF-1. It modulates IGF-1 signaling, promotes neuroprotection and synaptic plasticity, reduces neuroinflammation, and supports myelin maintenance by acting on IGF-1-dependent pathways in the CNS without directly binding the IGF-1 receptor.
Routes of Administration
Goals & Uses
- NeuroprotectionNeurologyModerate
- Cognitive and behavioral improvementNeurological / FunctionalLow
- Treatment of Phelan-McDermid syndromeNeurodevelopmental DisorderModerate
- Treatment of Pitt-Hopkins syndromeNeurodevelopmental DisorderModerate
- Treatment of Rett syndromeNeurodevelopmental DisorderModerate
- Treatment of Angelman syndromeNeurodevelopmental DisorderModerate
Contraindications
- Severe hepatic impairmentOrganModerateLiver function concerns
- Known hypersensitivity to NNZ-2591 or diketopiperazine componentsAllergyHigh
Adverse Effects
- HeadacheNeurologicUnknownPain in the head or upper neck
- Gastrointestinal discomfortGastrointestinalUnknown
- IrritabilityPsychiatricUnknownIncreased agitation or short temper
- SomnolenceNeurologicUnknownExcessive sleepiness
Drug Interactions
- IGF-1 / insulin pathway modulatorsModerate
Population Constraints
- PregnancyReproductive SafetyRelative
- Renal impairmentOrgan ImpairmentRelative
- Pediatric patients (primary population)AgeRelative
Regulatory Status
- European UnionUnknownNo known EU regulatory designation at this time
- United StatesInvestigationalFDA Orphan Drug Designation for Phelan-McDermid syndrome and Pitt-Hopkins syndrome; Phase 2 trials ongoing
- United KingdomUnknownNo known UK regulatory designation at this time
NNZ-2591 has received Orphan Drug Designation from the FDA for several rare neurodevelopmental conditions including Phelan-McDermid syndrome and Pitt-Hopkins syndrome. It is not yet approved in any jurisdiction. Phase 2 trials are ongoing in the US and Australia.
Evidence & Sources
No sources recorded yet.
Frequently Asked Questions
What is NNZ-2591?
NNZ-2591 is an orally bioavailable small cyclic dipeptide being developed by Neuren Pharmaceuticals for multiple pediatric neurodevelopmental disorders including Phelan-McDermid syndrome, Pitt-Hopkins syndrome, Angelman syndrome, and Rett syndrome. It has demonstrated neuroprotective and pro-cognitive effects in preclinical models and has entered Phase 2 clinical trials.
What is NNZ-2591 used for?
NNZ-2591 is educationally associated with: Neuroprotection, Cognitive and behavioral improvement, Treatment of Phelan-McDermid syndrome, Treatment of Pitt-Hopkins syndrome, Treatment of Rett syndrome, Treatment of Angelman syndrome. Educational only — not medical advice.
How is NNZ-2591 administered?
Recorded routes of administration: Oral.
What are the potential side effects of NNZ-2591?
Reported adverse effects include: Headache, Gastrointestinal discomfort, Irritability, Somnolence. This list is not exhaustive — consult a qualified clinician.
Who should avoid NNZ-2591?
Recorded contraindications: Severe hepatic impairment, Known hypersensitivity to NNZ-2591 or diketopiperazine components. Consult a qualified clinician before use.