Neuren Pharmaceuticals Announces FDA Orphan Drug Designation for NNZ-2591 in Genetic Neurological Disorders

On October 21, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biopharmaceutical company Neuren Pharmaceuticals (“Neuren”) announced that its NNZ-2591 had received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Angelman syndrome (AS), Phelan-McDermid syndrome (PMS), and Pitt-Hopkins syndrome (PTHS).

AS, PMS, and PTHS are all rare genetic neurological disorders. AS is caused by gene deletions or mutations on chromosome 15, while PMS results from deletions in the 22q13 region of chromosome 22 or other structural abnormalities. Patients primarily present with intellectual developmental delay, hypotonia, and motor retardation, and may develop epilepsy and autism. Currently, there are no specific treatments available for PMS.

NNZ-2591 is a class of drugs developed based on cyclic glycine-proline (cGP). cGP is naturally present in the human brain and plays a crucial role in neuronal development. Treatment with NNZ-2591 can restore normal pathways and signal transduction between neurons in the human brain. Studies in various animal models have demonstrated that this drug has neuroprotective and memory-enhancing effects.

Neuren conducted trials using a Shank3 knockout mouse model for PMS, administering NNZ-2591 to both wild-type and affected mice over a three-week period. The study demonstrated that the seizure frequency in treated knockout mice was reduced by 60%, with concomitant alleviation of symptoms including anxiety, repetitive behaviors, and motor deficits.

Neuren, established in 1993 and headquartered in Camberwell, Australia, is a biopharmaceutical company. The company primarily focuses on developing innovative drugs for brain injuries, neurodevelopmental disorders, and neurodegenerative diseases, providing new therapies for patients with hereditary neurological conditions. Neuren was listed on the Australian Securities Exchange in February 2005, with the stock code NEU.

In addition to NNZ-2591, Neuren has also developed Trofinetide (NNZ-2566), a patented synthetic analog derived from the human insulin-like growth factor 1 (IGF-1) protein. IGF-1 is produced by neurons and glial cells and can be cleaved into the glycine-proline-glutamate (GPE) peptide fragment, which is crucial for the normal development and protection of the central nervous system. However, this mechanism has not yet been fully elucidated.

Unlike traditional GPE drugs administered via injection, Trofinetide can be taken orally, offering superior efficacy, easier storage, and a longer duration of action in the human bloodstream. Previously, Neuren conducted double-blind, placebo-controlled trials of Trofinetide to evaluate its clinical efficacy in treating neurodevelopmental disorders, specifically Rett syndrome and Fragile X syndrome.

Neuren Executive Director Richard Treagus commented, “The FDA’s orphan drug designation for NNZ-2591 is a milestone in our development journey, which will advance the clinical trials and commercialization of this drug in 2020.”

(Compiled by Xu Xiaoxue)