Neuren Advances NNZ-2591 Program as Phase 2 PMS Data Published and Phase 3 Koala Trial Recruits in US

An announcement from Neuren Pharmaceuticals Limited ( (AU:NEU) ) is now available.

Neuren Pharmaceuticals has announced that results from its Phase 2 clinical trial of NNZ-2591 in children and adolescents with Phelan-McDermid syndrome have been published in the peer-reviewed journal Neurology Genetics, with the study reporting that the treatment was well tolerated and associated with meaningful improvements in key symptoms as observed by clinicians and caregivers. These Phase 2 data underpin the design of Neuren’s ongoing Koala Phase 3 trial in the United States, a randomized, placebo-controlled study targeting about 160 pediatric patients with Phelan-McDermid syndrome, a rare disorder with no approved therapies and a significant unmet medical need, and the program benefits from US FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations, reinforcing Neuren’s position in the rare neurodevelopmental disease space.

The most recent analyst rating on (AU:NEU) stock is a Buy with a A$21.00 price target. To see the full list of analyst forecasts on Neuren Pharmaceuticals Limited stock, see the AU:NEU Stock Forecast page.

More about Neuren Pharmaceuticals Limited

Neuren Pharmaceuticals is an Australian biopharmaceutical company focused on developing novel drug therapies for serious neurological and neurodevelopmental disorders that emerge in early childhood and have few or no approved treatments. Its portfolio includes DAYBUE (trofinetide), approved in the United States for Rett syndrome and licensed globally to Acadia Pharmaceuticals, and its second drug candidate NNZ-2591, which is being developed for multiple rare neurodevelopmental syndromes and has received orphan drug designations in the US and EU.

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