- Neuren Pharmaceuticals announces encouraging outcomes in its Phase 2 clinical trial of NNZ-2591 for Phelan-McDermid syndrome
- Clinicians and caregivers report improvements across key areas
- The company is conducting Phase 2 trials for NNZ-2591 in children
- NEU last traded at $17.14 at 10:00am AEDT
Melbourne-based Neuren Pharmaceuticals (NEU) has unveiled findings from its Phase 2 clinical trial of NNZ-2591 in treating Phelan-McDermid syndrome (PMS).
The trial, conducted over 13 weeks with up to 20 children aged 3 to 12 years, demonstrated notable improvements in various aspects of the syndrome.
Advancements were observed by both clinicians and caregivers, covering critical areas such as communication, behaviour, and cognition/learning.
The results displayed a level of improvement considered clinically meaningful, Neuren wrote on Monday.
Expectations beaten
“The strength and consistency of these results have exceeded our expectations, giving us high confidence as we strive to accelerate the development of a potential first therapy for PMS.” Neuren CEO Jon Pilcher said
Dr. Elizabeth Berry-Kravis, a partner in the study, added, “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.”
The open-label trial focused on safety, tolerability, pharmacokinetics, and efficacy. NNZ-2591, administered orally twice daily, demonstrated good safety and tolerability, with most adverse events being mild to moderate.
Safety data strong
The trial reported only one serious adverse event (gastroenteritis), unrelated to the study drug.
The results have prompted Neuren to advance NNZ-2591 into the next stage of development.
The company is also conducting Phase 2 trials for NNZ-2591 in children with Pitt Hopkins syndrome, Angelman syndrome, and Prader-Willi syndrome.
NEU last traded at $17.14 at 10:00am AEDT