NNZ-2591 is under clinical development by Neuren Pharmaceuticals and currently in Phase I for Prader-Willi Syndrome (PWS). According to GlobalData, Phase I drugs for Prader-Willi Syndrome (PWS) have a 93% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how NNZ-2591’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

NNZ-2591 overview

NNZ-2591 is under development for the treatment of Phelan-McDermid syndrome, Angelman Syndrome, Pitt Hopkins syndrome (genetic disorders), Fragile X syndrome, stroke, peripheral neuropathy, memory impairment, cognitive impairment, traumatic brain injury, multiple sclerosis, Prader-Willi syndrome and Parkinson’s disease. The drug candidate is administered orally. It is a synthetic analog of the neurotrophic peptide cyclic glycine proline, a naturally occurring neuropeptide. It belongs to the diketopiperazine family.

Neuren Pharmaceuticals overview

Neuren Pharmaceuticals (Neuren Pharma) is a biopharmaceutical company that develops drugs for brain injury, neurodevelopmental and neurodegenerative disorders. The company pipeline products includes trofinetide, NNZ-2591 and NNZ-2566. Its trofinetide has a pharmacokinetic profile, which is suitable for intravenous infusion and oral delivery. Neuren Pharma undertakes clincal trials for several drugs. The company’s trofinetide is a clinical stage drug candidate it provides molecule which is derived from IGF-1 and occurs naturally in the brain. Its drugs are also used in neurodevelopmental disorders such as angelman syndrome and idiopathic autism, or neurodegenerative disorders such as Parkinson’s disease and multiple sclerosis. Neuren Pharma is headquartered in Camberwell, Victoria, Australia.

For a complete picture of NNZ-2591’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.