The US FDA has approved an Investigational New Drug (IND), enabling the Melbourne-based company Myrio to progress its lead product into human clinical trials.
Myrio said it is collaborating in the trials with the leading children’s hospital in Philadelphia to study the CAR-T therapy for the treatment of neuroblastoma.
The functionally relevant and highly specific protein, called PHOX2B, was identified in neuroblastoma cells, and the team elucidated that a peptide from PHOX2B could serve as an excellent target for an immunotherapy to treat this devastating disease.
Myrio developed a highly specific binder to the PHOX2B peptide-major histocompatibility complex target using its ReD technology. In collaboration with the hospital, the Myrio binder has been engineered into a CAR-T product. Importantly, unlike other binders which target a single HLA-allotype, Myrio said its binders are capable of recognising the peptide in multiple HLA-allotypes, a phenomenon referred to as breaking HLA restriction, which offers the opportunity to treat a broader population of patients using the same immunotherapy.
This is the first time a binder developed by Myrio will enter a human trial.
The Phase 1 clinical trial entitled 'PHOX2B Peptide-Centric Chimeric Antigen Receptor Autologous T cells (PHOX2B PC-CAR T) for Relapsed Neuroblastoma' will be conducted under the leadership of Professor John Maris, and it is anticipated that the first patient will be enrolled mid-year 2025.
Myrio CEO Dr Graeme Wald said, “That this is a major step forward for Myrio. It is the culmination of many years of work at Myrio in developing bispecific binders to Human Leukocyte Antigens for the treatment of solid tumours.”