Myrio Therapeutics Pty Ltd. is set to advance lead product PHOX2B PC-CAR T (PHOX2B peptide-centric chimeric antigen receptor autologous T cells) into clinical trials for relapsed neuroblastoma following IND clearance by the FDA. The first patient will be enrolled around mid-year.
Neuroblastoma is a pediatric solid tumor characterized by dysregulation of the CDK-RB-E2F axis, leading to elevated E2F activity, uncontrolled progression through the G1/S transition and an aggressive tumor phenotype. Researchers from Circle Pharma Inc. and collaborators presented preclinical data on CID-078, a first-in-class oral macrocycle with dual cyclin A and B RxL inhibitory activity, in models of neuroblastoma.
Neuroblastoma, an aggressive malignancy originating from neural crest cells, accounts for 15% of cancer-related deaths in children. Treatment strategies include systemic chemotherapy, radiation or immunotherapy with anti-GD2 antibodies, all with severe side effects and long-term toxicity. Retinoic acid (RA) has been shown to promote neuroblastoma growth inhibition while suppressing MYCN oncogene expression. However, its effect is reversible, and tumor regrowth may occur.
Researchers at Children’s Hospital of Fudan University, Shanghaitech University and Suzhou Zhongke New Drug Incubator Biomedical Technology Co. Ltd. have identified WD repeat-containing protein 5 (WDR5; BIG3)/c-Myc interaction inhibitors reported to be useful for the treatment of cancer.
Neuroblastoma is the most common extracranial solid tumor in children and accounts for up to 15% of childhood cancer-related deaths worldwide. Neuroblastoma tumors are characterized by hypoxia, which represents a major challenge in its treatment.
Researchers from the Children's Hospital of Philadelphia presented data from a study that aimed to identify novel biologically relevant cell surface immunotherapeutic targets for neuroblastoma.
Invenra Inc.’s bispecific antibody, INV-724, developed for the treatment of neuroblastoma, has been awarded orphan drug and rare pediatric disease designations by the FDA.
The interaction between the transcriptional co-repressor runt-related transcription factor 1 translocation partner 1 (Runx1t1) and MYCN, a member of the MYC proto-oncogene family, plays a crucial role in the development of neuroblastoma, according to a study led by the Children's Cancer Institute at Lowy Cancer Centre, UNSW Sydney.
Ligand Pharmaceuticals Inc. is adding one more income stream to its portfolio, with the $100 million acquisition of Apeiron Biologics AG, owner of royalty rights to Qarziba, a treatment for high risk neuroblastoma that is currently marketed in 35 countries and is en route to approval in the U.S.
Day One Biopharmaceuticals Inc. has entered into an exclusive licensing agreement with Mabcare Therapeutics for MTX-13, a novel antibody-drug conjugate (ADC) targeting protein-tyrosine kinase 7 (PTK7). Under the agreement, Day One has exclusive rights to develop, manufacture and commercialize MTX-13 (now DAY-301), worldwide, excluding Greater China.
