NeuExcell Therapeutics' NXL-004 Gene Therapy Shows Positive Early Results in Treating Recurrent Malignant Glioblastoma

​SUZHOU, China – [June 2025]​​ – NeuExcell Therapeutics and the Fourth Affiliated Hospital of Soochow University announced the completion of enrollment and positive early results from an investigator-initiated trial (IIT) evaluating NXL-004, an innovative AAV-based gene therapy product, in the treatment of recurrent malignant glioblastoma patients.

Professor HUANG Yulun, the Principal Investigator and the Director of Neurosurgery at the Fourth Affiliated Hospital of Soochow University, led this first-in-human study to evaluate the safety and preliminary efficacy of NXL-004. This trial represents the world's first clinical application of in situ trans-differentiation technology in human brain, and the first clinical study of an AAV-based gene therapy to treat glioma.

Malignant gliomas, particularly glioblastoma (GBM), remain among the most aggressive and difficult-to-treat cancers, with limited treatment options available. Despite scientific advances, current standard therapies provide only modest survival benefits. Recurrence is almost inevitable among GBM patients, with median survival ranging from 6 to 9 months. There is an urgent need for novel and more effective therapeutic approaches.

Key Findings from the IIT:​

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1. ​Safety & Tolerability:​​ The study enrolled 10 patients with recurrent or progressive malignant glioma following prior surgery, radiation, and chemotherapy. NXL-004 was administered via intracranial injection after tumor resection or biopsy. The therapy demonstrated a favorable safety profile, with no drug-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs). NXL-004 was well-tolerated.
2. ​Survival Data:​​ To date, the majority of patients remain under survival follow-up. Among patients enrolled in 2024, the median follow-up time exceeds 8.5 months. Notably:
   Two patients have now surpassed 12 months of survival follow-up.
   The longest follow-up duration is >15 months.
   The estimated mean overall survival (OS) for the cohort currently exceeds 11 months.
3. ​Early Efficacy Signal:​​ Significant shrinkage of the target tumor lesion was observed in some patients following administration of NXL-004.

About NXL-004:​​

NXL-004 is a cutting-edge gene therapy product, utilizing an adeno-associated virus (AAV) vector to deliver the neural transcription factor NeuroD1 into glioma cells. This approach is designed to reprogram tumor cells, inducing cell death or trans-differentiating tumor cells into non-dividing neurons, thereby inhibiting tumor growth. NXL-004 has demonstrated promising efficacy and safety profile in rigorous preclinical studies. It is the ​first AAV-based gene therapy product to treat malignant glioma​ patients and has received the ​Orphan Drug Designation (ODD)​​ from the ​U.S. Food and Drug Administration (FDA).

About NeuExcell Therapeutics:

NeuExcell Therapeutics is a clinical-stage biotechnology company dedicated to developing next-generation therapies to treat neural injuries and neurodegenerative diseases through its proprietary in situ neural regeneration platform AtN ASTROCYTES-TO-NEURONS^TM technology. By leveraging this cutting-edge technology, NeuExcell aims to provide transformative solutions for the treatment of neurological disorders such as Alzheimer’s disease, stroke, and glioma.

For more information, please visit www.neuexcell.com.