Neuren Pharmaceuticals (ASX:NEU) has announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome (PMS).
The company said significant improvement was observed by both clinicians and caregivers from treatment across multiple efficacy measures. Improvements were consistently seen across many of the core PMS characteristics.
The open-label Phase 2 trial in up to 20 children aged 3 to 12 at four hospitals in the US examined safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ2591.
NNZ-2591 was administered to all subjects as an oral liquid dose twice daily, with escalation in two stages up to the target dose during the first six weeks of treatment, subject to independent review of safety and tolerability data.
The company said NNZ-2591 was well tolerated and demonstrated a good safety profile. Most Treatment Emergent Adverse Events (TEAEs) were mild to moderate, it said.
PMS has severe quality-of-life impacts on patients, and no approved treatments exist.
Neuren CEO Jon Pilcher said, "The strength and consistency of these results has exceeded our expectations and gives us high confidence as we strive to accelerate the development of a potential first therapy to address the overwhelming unmet medical need of PMS. We are very grateful to all the people at the trial sites in the United States and in the PMS community who enabled this groundbreaking trial to be completed successfully."
Elizabeth Berry-Kravis, MD, PhD, Professor of the Department of Pediatrics at Rush University Medical Center in Chicago and an Investigator in the study, said, "Although the Phase 2 trial was an open-label study, I am very encouraged that both clinicians and caregivers observed pervasive improvements across multiple, clinically important features of PMS including communication, cognition, learning, socialisation and behaviour. Improvements typically considered clinically meaningful were achieved in the clinician rated CGI-I as well as the caregiver rated CIC. I look forward to seeing this program advance to the next stage of development for the PMS community."
