NXL-004 / NeuExCell Therap - LARVOL DELTA

First-in-human study of AAV6-NeuroD1 trans-differentiation therapy in recurrent malignant glioma. (ASCO 2026) - P=N/A | "To our knowledge, this is the first clinical evidence of in vivo AAV-based trans-differentiation gene therapy for cancer treatment. NXL-004 showed favorable safety and encouraging efficacy, indicating that AAV-based cell reprogramming approach may represent a new modality for the treatment of glioma."

First-in-human • P1 data • Anaplastic Astrocytoma • Astrocytoma • Brain Cancer • CNS Disorders • Epilepsy • Gene Therapies • Glioblastoma • Glioma • High Grade Glioma • Infectious Disease • Solid Tumor • MGMT • NEUROD1

NeuExcell Therapeutics Announces Encouraging Data of NXL-004 in Recurrent Malignant Glioma at 2026 ASCO (GlobeNewswire) - "Median overall survival (mOS) of all treated recurrent glioma patients was over 13.2 months, comparing favorably with historical benchmarks of 6–9 months. The observed one-year survival rate reached 77.9%. All five patients enrolled between February and June 2025 were in high-dose group and remained alive at the time of this report: Among these five patients, four already achieved OS exceeding 12 months. One complete response (CR) and one durable stable disease (SD) were observed in the high-dose group, with ongoing progression-free survival (PFS) of 13.1 and 11.0 months."

First-in-human • P1 data • Glioma

Safety and efficacy of AAV-NeuroD1 based gene therapy for patients with recurrent malignant glioma from a first-In-human study (ESMO Asia 2025) - P=N/A | "This is the first clinical study of AAV-based in situ trans-differentiation gene therapy for glioma. NXL-004 showed good safety and preliminary efficacy, warranting further development."

Clinical • First-in-human • Gene therapy • P1 data • Anaplastic Astrocytoma • Astrocytoma • Brain Cancer • Glioblastoma • Glioma • High Grade Glioma • Oncology • Solid Tumor • NEUROD1

NXL-004: A novel trans-differentiation therapy for glioblastoma treatment. (ASCO 2024) - "Preclinical studies of NXL-004 have demonstrated that this intervention effectively reprograms tumor cells, downregulating glial cell- and tumor-related gene expression, while concurrently upregulating neuron-related gene expression. The induced differentiation towards non-dividing neurons results in significant inhibition of tumor cell proliferation, substantial reductions in tumor volume and prolonged survival in GBM-bearing mice. Additionally, NXL-004 has exhibited a favorable safety profile in toxicity studies in rats and non-human primates."

Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • NEUROD1

NeuExcell Therapeutics Announces Successful Dosing of First Patient by NeuroD1 Gene Therapy (PRNewswire) - "NeuExcell Therapeutics...announces the successful dosing of the first patient with the first-in-class NeuroD1 gene therapy product NXL-004. This milestone represents significant progress in the development of new treatment for malignant glioma patients. Led by Professor Yulun Huang and his team at the Fourth Affiliated Hospital of Soochow University in Suzhou, China, the investigator-initiated trial (IIT) will evaluate the safety and tolerability of NeuExcell's AAV-NeuroD1 gene therapy product NXL-004 in patients with glioblastoma."

Trial status • Glioblastoma

NeuExcell’s AAV Gene Therapy for Malignant Glioma Granted Orphan Drug Designation by FDA (PRNewswire) - "On December 7, 2023, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation ('ODD') to NXL-004, an investigational AAV gene therapy product being developed by NeuExcell Therapeutics Inc. ('NeuExcell'), for the treatment of malignant glioma. This is the world's first AAV gene therapy product for malignant glioma to receive FDA ODD designation....NeuExcell's lead clinical programs include...GBM, all expected to enter the clinic in 2024..."

New trial • Orphan drug • Brain Cancer • CNS Tumor • Glioma • Oncology • Solid Tumor