HLTX-001 / The Children’s Hospital of Philadelphia, Myrio Therap, Hula Therapeutics - LARVOL DELTA

PHOX2B PC-CAR T Cells for Relapsed Neuroblastoma (clinicaltrials.gov) - P1 | N=38 | Active, not recruiting | Sponsor: Stephan Grupp MD PhD | Recruiting ➔ Active, not recruiting

Enrollment closed • First-in-human • Neuroblastoma • Oncology • Solid Tumor

Durable remission of high-risk and refractory neuroblastoma by PHOX2B derived peptide-HLA directed CAR T cells: An IND enabling study (AACR 2026) - P1 | "Here, we report IND-enabling studies of second-generation PHOX2B PC-CAR T cells incorporating a 4-1BB costimulatory domain...PHOX2B-directed PC-CAR T cells show stringent specificity, potent cytotoxicity, and curative efficacy in preclinical patient-derived NB models. These IND-enabling data supported the ongoing first-in-human/child Phase 1 trial (NCT07007117), validating PHOX2B as a lineage-restricted immunotherapeutic target in high-risk neuroblastoma."

CAR T-Cell Therapy • Clinical • First-in-human • IO biomarker • Neuroblastoma • Oncology • Solid Tumor • CD8 • HOXB6 • IFNG • PHOX2B

PHOX2B PC-CAR T Cells for Relapsed Neuroblastoma (clinicaltrials.gov) - P1 | N=38 | Recruiting | Sponsor: Stephan Grupp MD PhD | Not yet recruiting ➔ Recruiting

Enrollment open • Neuroblastoma • Oncology • Solid Tumor

U.S. Food & Drug Administration (FDA) Approves Investigational New Drug (IND) Application for Myrio's Lead Product (PHOX2B PC-CAR T) for the Treatment of Neuroblastoma (PRNewswire) - "On 7 May 2025, the FDA approved an IND application enabling Myrio's lead product (PHOX2B PC-CAR T) to enter human trials. Myrio, in collaboration the leading children's hospital in Philadelphia have co-developed a Chimeric Antigen Receptor (CAR-T) therapy for the treatment of neuroblastoma....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."

IND • Trial status • Neuroblastoma