SINGAPORE, Dec. 18, 2025 (GLOBE NEWSWIRE) — NeuExcell Therapeutics announced encouraging clinical results of NXL-004, the world’s first in situ conversion gene therapy for malignant glioma. The data were presented at the ESMO Asia Congress 2025, December 5–7 in Singapore.
Recurrent malignant glioma carries an extremely poor prognosis, with median survival under nine months. NXL-004 is a first-in-class gene therapy using an adeno-associated virus (AAV) vector to deliver the neural transcription factor NeuroD1 directly into glioma cells, reprogramming tumor cells into non-dividing neurons or inducing apoptosis, representing a novel therapeutic paradigm.
This first-in-human study evaluated the safety and efficacy of intracranial NXL-004 administration in patients with recurrent malignant glioma. The trial was jointly conducted by the Fourth Affiliated Hospital of Soochow University and NeuExcell Therapeutics.
Eleven patients with recurrent malignant glioma following surgery and chemoradiotherapy were enrolled. Ten patients received surgical tumor resection followed by intracavitary NXL-004 injection, and one patient received intratumoral NXL-004 injection following biopsy.
Key study findings include:
- Safety: NXL-004 showed a favorable safety profile, with no drug-related serious adverse events or dose-limiting toxicities observed.
- Efficacy: Following NXL-004 treatment, the estimated median overall survival (OS) exceeded 12 months, well above historical controls of 6–9 months. The first patient enrolled survived over 18 months post-surgery and treatment.
- Tumor Response: The patient receiving intratumoral injection demonstrated a clear therapeutic effect, with significant regression of target lesion.
- Complete Response: In one patient receiving tumor resection followed by intracavitary NXL-004 injection, postoperative imaging showed progressive tumor reduction, ultimately resulting in complete radiographic disappearance, achieving a complete response (CR).
The results show that NXL-004 has a favorable safety profile and promising clinical efficacy, supporting the feasibility and therapeutic potential of in situ conversion therapy for the treatment of glioma.
About ESMO Asia:
ESMO Asia is the premier oncology conference in the Asia-Pacific region, convening global experts to disseminate breakthrough research and clinical innovations.
About NeuExcell Therapeutics:
NeuExcell is a clinical-stage biotech company focusing on the development of innovative treatments for neurological diseases. NeuExcell has developed a core platform to reprogram internal glial cells directly into functional neurons via in situ conversion approach, targeting a variety of neurological disorders including glioma, Alzheimer’s, Parkinson’s, and stroke.
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