NNZ-2591

Cyclic Dipeptide (diketopiperazine) / Neuroprotective Peptide AnalogRx: InvestigationalCompound: Investigational

Also known as: cGP analogue, cyclo-glycine-proline analog, NNZ2591

Educational Only — Not medical advice. Consult a qualified clinician before using any peptide.

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

Oral

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.

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