Neuren Pharmaceuticals achieves breakthrough in Pitt Hopkins syndrome treatment


Neuren Pharmaceuticals (ASX: NEU) has announced promising top-line results from its Phase 2 clinical trial of NNZ-2591, aimed at treating children with Pitt Hopkins syndrome (PTHS), a neurodevelopmental disorder with no approved treatments to date. The trial demonstrated statistically significant improvements across all four specifically designed efficacy measures, much to the optimism of clinicians and caregivers involved.

Results from Pitt Hopkins Syndrome Trial

The Phase 2 trial, conducted across five hospitals in the United States, focused on children aged 3 to 17 years. It tested NNZ-2591’s safety, tolerability, and efficacy over 13 weeks, with a dosage adjustment period in the first six weeks. Results indicated a statistically significant mean improvement from baseline in all four PTHS-specific efficacy measures, both for the subjects who completed the trial and including those who discontinued.

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Jon Pilcher, CEO of Neuren Pharmaceuticals, expressed enthusiasm about the trial’s success, stating, “This first clinical trial in Pitt Hopkins patients marks a significant milestone. We are now one step closer to providing a first approved treatment for this underserved community, addressing a critical unmet need.”

Expert Insights and Future Directions

Elliott Sherr, M.D., Ph.D., Professor of Neurology and Pediatrics at the University of California San Francisco, and an investigator in the trial, shared his optimism regarding the results. “The response observed in PTHS-specific measures and the underlying mechanism of action of NNZ-2591 suggest its potential applicability in other significant neurodevelopmental disorders,” commented Dr. Sherr.

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Dr. Nancy Jones, Vice President of Clinical Development at Neuren, emphasized the importance of syndrome-specific measurements in severe neurodevelopmental disorders. “The consistent results affirm the need for tailored approaches in clinical trials for such conditions,” she noted.

Safety and Efficacy Highlights

The study also underscored NNZ-2591’s favorable safety profile. Treatment-emergent adverse events were mild to moderate, and no serious adverse events were linked to the drug. This highlights its potential as a safe treatment option for PTHS and possibly other related disorders.

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Commitment to Neurodevelopmental Disorder Research

The trial’s success is a testament to the collaborative efforts between Neuren Pharmaceuticals, the Pitt-Hopkins Research Foundation, and numerous clinical experts. This partnership has been pivotal in advancing research and potentially offering new hope to families affected by Pitt Hopkins syndrome.

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