The U.S. Food and Drug Administration (FDA) has approved Myrio Therapeutics’ Investigational New Drug (IND) application for its lead product, PHOX2B PC-CAR T, paving the way for human trials in treating neuroblastoma. This milestone follows collaborative research with a leading children’s hospital in Philadelphia. The therapy targets a unique protein, PHOX2B, present in neuroblastoma cells, identified by Prof. John Maris. Myrio’s ReDTM technology was instrumental in developing a highly specific binder to the PHOX2B peptide-major histocompatibility complex (p-HLA). This binder breaks HLA restriction, broadening the potential patient population that can benefit from the immunotherapy.
Prof. Maris highlighted the urgent need for new treatments, stating that current options for high-risk neuroblastoma have low response rates and significant toxicities. 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 Prof. Maris and it is anticipated that the first patient will be enrolled mid-year 2025.
The incidence of neuroblastoma in the United States is approximately 800 cases, accounting for 15% of paediatric cancer-related deaths. Despite improvements in overall survival rates, challenges remain in treating high-risk forms of the disease. Dr. Graeme Wald, CEO of Myrio Therapeutics, hailed this approval as a major step forward for the company and a validation of its technology in developing bispecific binders to Human Leukocyte Antigens for solid tumor treatment.
Myrio’s ReDTM platform facilitates the discovery of stable, human scFv binders against peptides on solid cancer cells. These binders, used in bispecific T-Cell Engagers or CAR-T formats, direct cytotoxicity against specific tumor cells. This approval marks the first time a Myrio-developed binder will enter human trials.
