On May 31, 2026 NeuExcell Therapeutics reported that Dr. Yulun Huang will present the first-in-human clinical study of NXL-004 for recurrent malignant glioma as Rapid Oral presentation at the 2026 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (ASCO 2026). ASCO (Free ASCO Whitepaper) is one of the leading global oncology conferences. With over 8,500 submissions, abstracts selected for oral presentation represents studies of significant scientific and clinical interests.

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"We are honored that our NXL-004 program has been selected for Rapid Oral Presentation at ASCO (Free ASCO Whitepaper) 2026," said Dr. Gong Chen, Founder and Chief Scientific Officer of NeuExcell. "This recognition underscores the potential of our in vivo cell conversion platform to address significant unmet needs in recurrent malignant glioma and potentially other devastating neurological diseases."

The study was conducted in collaboration with Dr. Yulun Huang and his team at the Department of Neurosurgery at the Fourth Affiliated Hospital of Soochow University.

Presentation Details

Title:
First-in-human study of AAV6-NeuroD1 trans-differentiation therapy in recurrent malignant glioma

Presenter:
Dr. Yulun Huang

Session:
Rapid Oral Abstract Session – Central Nervous System Tumors

Date & Time:
May 31, 2026 | 4:30 PM–6:00 PM CT

Abstract Number:
2012

Favorable Safety and Encouraging Efficacy

Recurrent malignant glioma remains one of the most difficult cancers to treat, with median overall survival (OS) at approximately 6-9 months. After recurrence, there is currently no established standard of care, and no therapy has demonstrated a clear survival benefit in randomized controlled studies.

NXL-004 is a first-in-class in vivo cell conversion therapy using an adeno-associated virus (AAV) vector to deliver neural transcription factor NeuroD1. The therapy is designed to reprogram proliferating glioma cells toward non-proliferating neuron-like cells or induce tumor cell apoptosis.

Results from the first-in-human study in recurrent malignant glioma showed a favorable safety profile and encouraging efficacy:

Baseline and Treatment
Eleven patients were enrolled, with a median age of 48.0 years; 81.8% male.
All patients had recurrent WHO grade 4 astrocytoma, including 10/11 with IDH-wildtype tumors and 6/11 with MGMT-unmethylated tumors.
Ten patients received repeat tumor resection plus intracavitary injection of NXL-004, and one received biopsy plus intra-tumoral injection.
Safety:
No drug-related serious adverse events (SAEs) were reported.
All AEs related to NXL-004 were grade 1–2. The most common treatment-related AEs included fever and headache.
Efficacy:
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.
"This study represents the first clinical evaluation of an AAV-NeuroD1 based gene therapy for glioma," said Dr. Yulun Huang, the principal investigator of the study. "The findings provide encouraging evidence supporting both the safety and therapeutic potential of this novel approach."

About NXL-004

NXL-004 is NeuExcell’s investigational gene therapy candidate based on its proprietary in vivo cell conversion platform. The therapy utilizes AAV-mediated delivery of NeuroD1 to reprogram tumor cells directly within the brain microenvironment.

(Press release, NeuExcell Therapeutics, MAY 31, 2026, View Source [SID1234666286])