Australia-based Neuren Pharmaceuticals has confirmed the primary goals for its double-blind, single Phase III pivotal trial of its drug NNZ-2591 in treating Phelan-McDermid syndrome (PMS) for 13 weeks.
The company’s decision is based on a productive Type C meeting with the US Food and Drug Administration (FDA).
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
This placebo-controlled trial’s co-primary endpoints will focus on change from baseline in the receptive communication sub-domain of the Vineland Adaptive Behaviour Scales, Third Edition (VABS-3 Receptive-Raw Score), and the overall PhelanMcDermid Syndrome Assessment of Change (PMSA-C) score.
These measures showed improvement in the open-label Phase II trial.
According to the company, 16 out of 18 paediatric patients exhibited improvements as indicated by the VABS-3 Receptive-Raw Score, with a 7.5 mean improvement from a baseline average of 29.0.
Additionally, 16 subjects demonstrated enhancement from the baseline measurements by the PMSA-C, achieving a mean score of 2.4.
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataThe endpoints combine the caregiver’s evaluation of change in a key symptom area with the clinician’s assessment of change in various PMS aspects.
Caregivers reported that one of the most impactful PMS health concerns is communication.
Neuren noted that the VABS-3 Receptive-Raw Score gauges an individual’s capacity of receiving and understanding both non-verbal and verbal forms of communication.
This skill is crucial as it lays the groundwork for social interaction, speech and learning.
The alignment with the US regulator on the important features of the Phase III trial was established during a Phase II meeting end.
Neuren Pharmaceuticals CEO Jon Pilcher said: “We are very pleased with the outcome of another constructive discussion with the FDA and are now excited to be able to move forward as planned with the first ever Phase III trial in children with PMS.”
Neuren noted that it is on track to start the Phase III trial by the middle of this year, pending the agency’s review of the trial protocol’s final version.
The VABS-3 and PMSA-C are essential tools in assessing adaptive behaviour and changes in core symptoms of PMS, respectively.
PMS is associated with deletion or any changes in the 22q13 region of chromosome 22.
NNZ-2591, which is being developed for several neurodevelopmental disorders, has shown positive outcomes in Phase II trials for Pitt-Hopkins syndrome, Angelman syndrome, and PMS.
