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Shares of Neuren Pharmaceuticals Ltd (ASX:NEU) jumped over 10% on Thursday after the company announced a key development in its rare disease pipeline. The U.S. Patent and Trademark Office has approved Neuren’s patent application for NNZ-2591, a promising treatment targeting Pitt Hopkins syndrome (PTHS), a rare genetic neurological disorder with no approved therapies. The newly granted patent is valid until April 2040, giving Neuren long-term protection for the drug's use in treating PTHS.
This milestone adds to the momentum already behind NNZ-2591, which has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA). These regulatory advantages are designed to speed up the development and review process for treatments addressing serious or life-threatening conditions.
In a recent Phase 2 clinical trial, NNZ-2591 demonstrated significant potential, with 9 out of 11 children showing improvements in communication, cognitive function, and motor skills. Neuren is currently in discussions with the FDA to determine the next steps and is preparing to initiate a Phase 3 trial for a separate neurodevelopmental disorder, further expanding its pipeline.
As of 01:09 GMT, Neuren shares surged 11% to A$13.93 on the ASX, reaching their highest level in two weeks, reflecting strong investor confidence in the company’s R&D trajectory. Neuren already has one FDA-approved therapy—DAYBUE—for Rett syndrome, another rare neurodevelopmental disorder, reinforcing its position as a leading biotech in the field of pediatric neurology.
The patent approval for NNZ-2591 marks a significant advancement in Neuren’s efforts to bring novel treatments for underserved rare diseases to market, enhancing both its scientific credibility and market value.
