NeuExcell Therapeutics Announces Global First-in-Human Dosing of AAV-NeuroD1 Gene Therapy NXL-004 for Recurrent Malignant Glioma

Recently, the AAV-NeuroD1 gene therapy product NXL-004, independently developed by NeuExcell Therapeutics, was successfully administered to its first patient at the Fourth Affiliated Hospital of Soochow University (Suzhou Dushu Lake Hospital) by a team led by Professor Huang Yulun, Director of the Department of Neurosurgery, Chief Physician, and Doctoral Supervisor.

The subject was a patient with recurrent glioma. The surgical procedure and drug administration proceeded smoothly, the patient’s postoperative condition remained stable, no significant adverse reactions were observed, recovery was favorable, and the patient was discharged uneventfully one week after surgery. This marks the first clinical administration of AAV-NeuroD1 gene therapy worldwide, signifying that this novel gene therapy has officially entered the clinical research phase.

Malignant brain gliomas are characterized by high mortality rates, with the majority being glioblastoma (GBM). Current standard treatments for malignant gliomas include surgery, radiotherapy, and chemotherapy. Despite significant advances in the understanding of malignant gliomas in recent years, therapeutic efficacy remains limited. Furthermore, recurrence is nearly inevitable in patients with malignant gliomas, and survival after recurrence is short; therefore, there is an urgent need for more effective treatment strategies.

NXL-004 is an innovative therapeutic approach developed based on the pioneering in situ transdifferentiation technology platform established by Professor Gong Chen’s team. It is also the first AAV gene therapy product for malignant glioma to receive FDA Orphan Drug Designation. Its mechanism of action involves overexpressing the neural transcription factor NeuroD1 within glioma cells, thereby inhibiting tumor cell division and proliferation while promoting their transdifferentiation into neuronal cells. Compared with radiotherapy and chemotherapy, NeuroD1 gene therapy acts intrinsically within tumor cells by altering their transcriptomic profile of gene expression, resulting in fewer side effects on surrounding tissues or cells in other organs. The objective of this clinical study is to evaluate the safety and tolerability of NXL-004 injection in patients with malignant glioma, and to preliminarily explore its efficacy.

Professor Huang Yulun, the Principal Investigator, stated: “Glioblastoma is highly malignant and associated with a high mortality rate; concerted efforts are required to explore its treatment. NXL-004 has demonstrated favorable efficacy and safety data in preclinical glioma models and animal studies. The first clinical subject has been dosed with good safety outcomes, which is a promising signal. We will continue to monitor the patient and look forward to observing favorable therapeutic effects.”

Professor Gong Chen, Founder of NeuExcell Therapeutics, stated: “We extend our sincere gratitude to Professor Yulun Huang and his team for their dedicated collaboration, meticulous preoperative preparation, and exceptional surgical expertise, which ensured the successful administration of the investigational product to the first patient. The successful intracerebral delivery of AAV-NeuroD1 gene therapy in the world’s first glioma patient marks a significant milestone, signifying NeuExcell Therapeutics’ transition from preclinical animal studies to clinical trials in patients. This achievement opens new avenues for individuals suffering from neurological disorders, and we hope that our years of dedication will ultimately benefit patients.”

Dear Patients and Their Families:

The Fourth Affiliated Hospital of Soochow University (Suzhou Dushu Lake Hospital) is currently conducting“An Exploratory Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of NXL-004 Injection in Patients with Malignant Glioma”This study was investigator-initiated and approved by the Ethics Committee of our hospital.

If you or your family and friends meet the following main criteria:

1. Age ≥18 and ≤70 years.

2. Previously diagnosed malignant glioma (WHO Grade 3-4) confirmed by biopsy or histopathology.

3. Recurrence or progression after chemoradiotherapy, with one measurable lesion on contrast-enhanced MRI (tumor diameter ≥1.0 cm).

If you are interested in learning more about this study, please consult the physicians listed below. You will be eligible to participate if, based on the physician’s comprehensive assessment, you meet the study’s inclusion criteria. During the study, specialist physicians will provide regular consultations and conduct periodic assessments and monitoring of your physical condition and disease status.

Contact: Dr. Zhu Haiping

Phone: 13962123202

Mailing Address: No. 9 Chongwen Road, Suzhou Industrial Park, Suzhou Dushu Lake Hospital