Australia’s Neuren Pharma surges over 10% on US patent win for rare disease drug

Published 26/06/2025, 02:20
©  Reuters

Investing.com-- Shares of Neuren Pharmaceuticals Ltd (ASX:NEU) soared more than 10% on Thursday after the Australian biotech firm said the U.S. Patent and Trademark Office allowed its patent application for NNZ-2591, a treatment for the rare genetic disorder Pitt Hopkins syndrome (PTHS).

The patent, set to expire in April 2040, covers the use of NNZ-2591 for PTHS, a condition with no approved therapies, the company said in a statement.

The U.S. Food and Drug Administration has already granted the drug Orphan Drug and Fast Track designations, accelerating its development.

Sydney-listed shares of the company surged 11% to A$13.93 as of 01:09 GMT, reaching their highest level in two weeks.

In a Phase 2 trial, NNZ-2591 showed improvements in communication, cognition, and motor skills in 9 out of 11 children. Neuren plans to discuss next steps with the FDA and is preparing a Phase 3 trial for another neurodevelopmental disorder.

The company’s lead drug, DAYBUE, is already approved for Rett syndrome.

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