Neuren (ASX:NEU) initiates Phase 2 trials of NNZ-2591

Be the First to Comment Read

Neuren (ASX:NEU) initiates Phase 2 trials of NNZ-2591

 Neuren (ASX:NEU) initiates Phase 2 trials of NNZ-2591
Image source: © Alfaphoto9 |


  • Neuren Pharmaceuticals (ASX:NEU) has began recruiting subjects for Phase 2 clinical trials of its second drug candidate, NNZ-2591.
  • The trials will be conducted in children each for Pitt Hopkins syndrome (PTHS) and Phelan-McDermid syndrome (PMS).
  • PTHS and PMS are neurological disorders affecting both sexes, emerging in early childhood.

Neuren Pharmaceuticals (ASX:NEU) has initiated enrolment for Phase 2 clinical trials of NNZ-2591 for Phelan-McDermid syndrome (PMS) and Pitt Hopkins syndrome (PTHS) in the United States. The trials will be conducted in children under approved Investigational New Drug applications (INDs) with the US Food and Drug Administration.

Neuren is focused on developing two new drug therapies for treating serious early childhood neurological disorders. Neuren’s lead compound is trofinetide, which is being developed for Rett syndrome, while NNZ-2591 is the second drug candidate of Neuren.


Image Source: © Mjbowra |

PMS and PTHS are serious neurological disorders that affects both sexes. These disorders appear in early childhood and impact almost every part of life.   Further, they do not have any approved medicines.

PMS characteristics include speech problems, intellectual disability, epilepsy, etc. PTHS is a neurodevelopmental condition with symptoms including behavioural differences, developmental delay, gastrointestinal problems, sleep disturbance, etc.

Safety, tolerability, pharmacokinetics, and efficacy

The first subjects of the trial are expected to enter the trial imminently at Rush University Medical Center in Chicago, with results anticipated in H1 2023. Both these Phase 2 trials are aimed at generating information to update the design of a successive registration trial.

Besides tolerability and safety, the primary results of the trial reflect standard pharmacokinetic elements as well as severity, frequency and incidence of adverse events.

Secondary outcome measures are different kinds of exploratory efficiency measures for each syndrome. Recently, Neuren initiated a Phase 2 trial of NNZ-2591 in Angelman syndrome (AS) and is also implementing the preliminary work to prepare for Phase 3 development in different indications. 

Image Source: © 2022 Kalkine Media®

Stock information

Shares of Neuren were trading at AU$5.710 (as on 03:23 PM AEST on 08 August 2022). The company has a market capitalisation of AU$702.86 million.


Speak your Mind

Featured Articles

We use cookies to ensure that we give you the best experience on our website. If you continue to use this site we will assume that you are happy with it. OK