Neuren Pharmaceuticals (ASX:NEU) has reached a regulatory milestone with Health Canada approving its Clinical Trial Application for the Koala Phase 3 clinical trial of NNZ-2591, a drug candidate under development for Phelan-McDermid syndrome (PMS).
The approval clears the way for the inclusion of clinical trial sites in Canada, further expanding the global reach of the first-ever Phase 3 trial for this rare neurodevelopmental disorder.
The Koala study is evaluating NNZ-2591 in children aged three to twelve years. PMS is a condition for which no approved treatments currently exist.
The trial had previously received Investigational New Drug approval from the US Food and Drug Administration, enabling enrolment at US sites to commence. The second clinical site in California is now activated and actively enrolling participants. Additional US sites are progressing through the activation process, broadening access for families on both the East and West coasts.
Neuren's Chief Executive Officer, Jon Pilcher, described the Health Canada decision as a validation of the program and a meaningful step forward for the PMS community. He noted that regulatory clearance from a second authority underscores the significance of the Koala trial and reinforces the company’s commitment to advancing NNZ-2591 as a potential therapy for children with this severe condition.
NNZ-2591 represents Neuren’s second major development program and has already demonstrated encouraging results in Phase 2 trials across multiple neurodevelopmental disorders, including Phelan-McDermid syndrome, Pitt Hopkins syndrome and Angelman syndrome.