Myrio’s Novel PHOX2B PC-CAR T Therapy for Neuroblastoma Receives FDA IND Clearance

MELBOURNE, Australia – June 13, 2025 – Myrio, an Australian biotech company, announced FDA Investigational New Drug (IND) clearance for its lead product, PHOX2B PC-CAR T, to enter human trials. Co-developed with a leading children’s hospital, this novel CAR T-cell therapy targets neuroblastoma, a devastating pediatric cancer.

The therapy targets PHOX2B, a specific protein identified in neuroblastoma cells. Myrio’s unique ReD™ technology developed a highly specific binder to the PHOX2B peptide-major histocompatibility complex (p-HLA) target. Crucially, this binder is capable of recognizing the peptide in multiple HLA-allotypes, “breaking HLA restriction” to potentially treat a broader patient population.

Professor John Maris, a key collaborator, emphasized the urgent need for new neuroblastoma treatments, stating this immunotherapy “has the potential to be a major advance.” The Phase 1 clinical trial for PHOX2B PC-CAR T is anticipated to enroll its first patient mid-2025. This IND clearance marks a significant validation of Myrio’s technology and its potential to address the high unmet medical need in pediatric neuroblastoma.