July 1, 2026 – NeuExcell Therapeutics, a clinical-stage biotechnology company pioneering in vivo cell reprogramming, announced a landmark milestone in its first-in-human study of NXL-004 for recurrent malignant glioma. All six patients in the high-dose cohort have survived beyond 12 months post-treatment, achieving a 100% one-year survival rate—a result unprecedented in recent clinical literature for this aggressive disease.
The final patient in the high-dose cohort completed the 12-month follow-up in late June 2026. This patient, treated one year ago with tumor resection followed by intracavitary injection of NXL-004, has remained progression-free without any additional anti-cancer therapy and continues to maintain a good functional status.
Among the six subjects, two have achieved sustained disease control exceeding 12 months, with one patient demonstrating a Complete Response (CR).
Recurrent malignant glioma carries a dismal prognosis, with median overall survival (OS) typically ranging from 6 to 9 months. Despite therapeutic advances, published one-year survival rates for existing regimens generally remain below 40%. To our knowledge, no publicly available clinical report has previously documented a 100% one-year survival rate in this patient population.
"These data represent a significant step forward in our quest to transform the treatment paradigm for malignant glioma," said Dr. Gong Chen, founder and CSO of NeuExcell Therapeutics. "The durability of the response and the quality of life observed in these patients are highly encouraging and support further clinical development of NXL-004. We are continuing our development of NXL-004 and exploring the potential of this approach for other neurological disorders."
While these results are from an exploratory study and require validation in larger, more rigorous clinical trials, they provide strong clinical proof-of-concept for NXL-004’s therapeutic potential.
A Novel Mechanism of Action: Reprogramming, Not Killing
NXL-004 is a first-in-class gene therapy that employs an AAV6 vector to deliver the NeuroD1 gene directly into glioma cells. Unlike conventional therapies—such as radiotherapy, chemotherapy, oncolytic viruses, or CAR-T—which rely on direct cytotoxicity, NXL-004 works through a unique in vivo reprogramming approach:
This synergistic mechanism—combining suppressed proliferation, induced cell death, and immune activation—offers a novel therapeutic avenue for patients with few remaining options.
The success of NXL-004 also provides critical clinical validation for NeuExcell’s proprietary in vivo reprogramming platform, underscoring its broader potential to address other severe neurological conditions, including Alzheimer’s disease and stroke.
About NeuExcell Therapeutics
NeuExcell Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative therapies for neural injury and neurodegenerative diseases. Built on pioneering research from Dr. Gong Chen’s team, NeuExcell’s platform enables targeted in vivo cell conversion within the central nervous system, including astrocyte-to-neuron conversion and glioma cell reprogramming.
NeuExcell is advancing a robust pipeline in glioma, Alzheimer’s disease, and stroke, with the mission of delivering transformative treatments to patients suffering from severe neurological disorders.