NNZ-2591

Experimental drug for neurodevelopmental disorders

NNZ-2591
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NNZ-2591 is an experimental drug being investigated for the treatment of various neurodevelopmental disorders. It is a synthetic analog of the naturally occurring neuropeptide cyclo-glycyl-proline (cGP), which is known to have neuroprotective and cognitive-enhancing properties.

Mechanism of Action

NNZ-2591 is believed to exert its effects by modulating the activity of insulin-like growth factor 1 (IGF-1) and its receptor. IGF-1 is a hormone similar in molecular structure to insulin and plays an important role in childhood growth and continues to have anabolic effects in adults. By influencing IGF-1 signaling pathways, NNZ-2591 may help to restore normal function in neural circuits that are disrupted in neurodevelopmental disorders.

Potential Therapeutic Uses

NNZ-2591 is being studied for its potential to treat several neurodevelopmental disorders, including:

• Phelan-McDermid syndrome
• Angelman syndrome
• Rett syndrome
• Pitt-Hopkins syndrome

These conditions are characterized by intellectual disability, developmental delay, and other neurological symptoms. NNZ-2591 aims to improve cognitive function and reduce the severity of these symptoms.

Clinical Trials

As of the latest updates, NNZ-2591 is undergoing various stages of clinical trials to evaluate its safety and efficacy. Early-phase trials have shown promising results in terms of tolerability and potential therapeutic benefits. Further studies are needed to confirm these findings and to determine the optimal dosing regimen.

Research and Development

The development of NNZ-2591 is spearheaded by Neuren Pharmaceuticals, a company focused on developing therapies for neurological disorders. The research involves collaboration with academic institutions and clinical research organizations to advance the understanding of NNZ-2591's effects and its potential as a treatment option.

Related Pages

• Neurodevelopmental disorder
• Neuropeptide
• Insulin-like growth factor 1
• Phelan-McDermid syndrome
• Angelman syndrome
• Rett syndrome
• Pitt-Hopkins syndrome