Neuren Launches First US Site for Phelan-McDermid Phase 3 Trial
Neuren Pharmaceuticals has initiated the first US investigational site for its pivotal Phase 3 trial of NNZ-2591 targeting Phelan-McDermid syndrome, marking a significant milestone in addressing this rare neurodevelopmental disorder.
- First US site initiated for NNZ-2591 Phase 3 trial in Phelan-McDermid syndrome
- Trial to enroll approximately 160 children aged 3-12 with no approved treatments
- Randomized, double-blind, placebo-controlled design with 13-week treatment period
- Open-label extension for 12 months available for all participants
- Trial design aligned with FDA following Phase 2 meetings; fully funded by Neuren’s cash reserves
Neuren Advances Clinical Development in Rare Disorder
Neuren Pharmaceuticals has taken a decisive step forward in its mission to develop treatments for rare neurodevelopmental disorders by initiating the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591. This trial targets Phelan-McDermid syndrome (PMS), a debilitating genetic condition with no currently approved therapies.
The Phase 3 study is designed as a randomized, double-blind, placebo-controlled trial involving approximately 160 children aged between 3 and 12 years. Participants will undergo 13 weeks of treatment, with the option to continue receiving NNZ-2591 for an additional 12 months in an open-label extension. This approach not only aims to rigorously evaluate efficacy and safety but also offers ongoing access to the investigational drug for families affected by PMS.
Addressing a Critical Unmet Need
Phelan-McDermid syndrome arises from deletions or mutations in the SHANK3 gene on chromosome 22, leading to severe developmental delays, intellectual disabilities, and a spectrum of neurological symptoms including autism-like behaviors and epilepsy. Affecting an estimated 1 in 8,000 to 1 in 15,000 individuals, PMS represents a significant unmet medical need with profound impacts on patients and their families.
Neuren’s NNZ-2591 has shown promising results in earlier Phase 2 trials not only for PMS but also for other rare neurodevelopmental disorders such as Pitt Hopkins and Angelman syndromes. The company’s engagement with the US Food and Drug Administration has resulted in alignment on the Phase 3 trial design, including primary efficacy endpoints, underscoring regulatory confidence in the program’s scientific rigor.
Fully Funded and Strategically Positioned
The trial is fully funded from Neuren’s existing cash reserves, highlighting the company’s financial preparedness to advance this critical program without immediate capital raises. This financial stability is particularly notable given the orphan drug designation granted to NNZ-2591, which offers incentives to support development for rare diseases.
Neuren’s CEO, Jon Pilcher, emphasized the importance of this milestone, noting that the initiation of the first US site marks a pivotal moment in the company’s efforts to bring a much-needed therapy to children living with PMS. The company is progressing other US sites through the initiation process, aiming for robust enrollment and data collection.
With no approved treatments currently available, the success of this Phase 3 trial could transform the therapeutic landscape for PMS and related disorders, offering hope to families who have long faced limited options.
Bottom Line?
Neuren’s Phase 3 trial launch signals a critical advance in rare disease treatment, with eyes now on enrollment progress and upcoming data readouts.
Questions in the middle?
- How quickly will Neuren complete enrollment across all US sites?
- What interim data might emerge during the 13-week treatment period?
- Could positive Phase 3 results accelerate regulatory approvals beyond the US?
