See highlights from the Global Science Summit

Current Pipeline
Small Molecule

Neuren • NNZ-2591

Neuren Pharmaceuticals is exploring an investigational drug called NNZ-2591, a synthetic analog related to Insulin-Like Growth Factor-1 (IGF-1), as a potential treatment for Angelman syndrome.

Discovery & DevPre-clinicalPhase 1Phase 2Phase 3To Patients

About the Program

NNZ-2591 is a synthetic analog of a peptide that is related to IGF-1, a growth factor that is stimulated by growth hormone. IGF-1 in the brain is thought to be critical for normal brain development and to maintain biological balance by reducing inflammation, restoring normal cell function of the cells that surround neurons of the brain, and improve the function at the junction of the neurons.

Investigational Drug Name
NNZ-2591
Company Website
Neuren Pharmaceuticals

Program News

Neuren Pharmaceuticals announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS).

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COMPLETED: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome (AS-001)

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