Neuren Publishes Promising Phase 2 Data as Phase 3 Koala Trial Recruits PMS Patients
Neuren Pharmaceuticals has published promising Phase 2 trial results for NNZ-2591 in Phelan-McDermid syndrome, underpinning its ongoing Phase 3 Koala trial recruiting in the US.
- Phase 2 trial results published in Neurology® Genetics
- NNZ-2591 well-tolerated with meaningful symptom improvements
- Phase 3 Koala trial recruiting children aged 3 to 12 in the US
- FDA granted Fast Track, Rare Pediatric Disease, and Orphan Drug designations
- Phelan-McDermid syndrome remains an unmet medical need with no approved treatments
Neuren Publishes Phase 2 Results in Leading Neurology Journal
Neuren Pharmaceuticals (ASX – NEU) has reached a significant milestone with the publication of its Phase 2 clinical trial results for NNZ-2591 in children and adolescents with Phelan-McDermid syndrome (PMS). The study, featured in the respected peer-reviewed journal Neurology® Genetics, highlights that the drug was well-tolerated and led to meaningful improvements in key symptoms as reported by clinicians and caregivers.
Building on Phase 2 Success – The Koala Phase 3 Trial
The Phase 2 trial, which spanned 13 weeks and involved patients aged 3 to 12, has directly informed the design of Neuren’s ongoing Phase 3 Koala trial. This pivotal study is currently recruiting approximately 160 participants in the United States, maintaining the same age range, treatment duration, and dosing regimen as its predecessor. The Koala trial is a randomized, double-blind, placebo-controlled study aimed at rigorously evaluating the safety and efficacy of NNZ-2591.
Addressing a Rare and Challenging Disorder
Phelan-McDermid syndrome is a rare genetic condition caused by deletions or mutations affecting the SHANK3 gene, critical for brain synapse function. With an estimated prevalence between 1 in 8,000 and 1 in 15,000 people, PMS presents with severe developmental delays, intellectual impairment, autism-like symptoms, and other neurological challenges. Currently, no approved therapies exist, leaving a substantial unmet medical need for affected families.
Regulatory Support and Future Prospects
NNZ-2591 has been granted Fast Track, Rare Pediatric Disease, and Orphan Drug designations by the US Food and Drug Administration, recognising its potential to address this unmet need. Neuren’s CEO Jon Pilcher expressed optimism about the Phase 3 recruitment and the prospect of delivering a much-needed treatment option for the PMS community. The company’s broader portfolio also includes DAYBUE® for Rett syndrome, underscoring its focus on neurodevelopmental disorders.
Looking Ahead
While the Phase 2 results provide a strong scientific foundation, the Phase 3 Koala trial will be critical in confirming NNZ-2591’s efficacy and safety profile. Success in this trial could pave the way for regulatory approval and commercialisation, offering hope to families affected by PMS and potentially transforming the treatment landscape for this rare disorder.
Bottom Line?
Neuren’s Phase 3 trial progress will be pivotal in translating promising early results into a viable treatment for PMS.
Questions in the middle?
- When will interim or final data from the Phase 3 Koala trial be available?
- How might NNZ-2591’s efficacy compare to emerging therapies for PMS or related disorders?
- What are Neuren’s plans for commercialisation or partnerships if Phase 3 results are positive?
