Cue Biopharma Initiates Investigator-Sponsored Trial of CUE-102 in Recurrent Glioblastoma Multiforme, First Patient Dosed at Dana-Farber Cancer Institute.

Cue Biopharma has initiated an investigator-sponsored trial of CUE-102 in recurrent glioblastoma multiforme (rGBM) at Dana-Farber Cancer Institute. The trial aims to evaluate the tolerability and clinical activity of CUE-102 in patients with GBM at first recurrence. CUE-102 targets Wilms' Tumor 1 protein (WT1) expressing cancers and has shown anti-tumor activity with a favorable tolerability profile in previous trials. The trial's principal investigator is David A. Reardon, MD, a leader in immunotherapy for brain cancer.

Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company, has announced the initiation of an investigator-sponsored trial (IST) of CUE-102 for recurrent glioblastoma multiforme (rGBM) at Dana-Farber Cancer Institute (DFCI). The Phase 1b open-label study, led by Dr. David A. Reardon, Clinical Director of DFCI's Center for Neuro-Oncology, aims to evaluate the tolerability and clinical activity of CUE-102 in patients with GBM at first recurrence [1].

CUE-102 is designed to activate Wilms' Tumor 1 (WT1)-specific T cells against glioblastoma, which typically presents challenges for standard immunotherapies due to its immunologically 'cold' tumor nature but exhibits high levels of WT1 protein [2]. The trial, identified as NCT06917885, is a significant clinical development for Cue Biopharma, expanding its clinical pipeline to target a difficult-to-treat brain cancer with high unmet need.

The principal investigator, Dr. Reardon, is a leader in the field of immunotherapy for brain cancer. He stated, "Glioblastoma remains one of the most aggressive and hard-to-treat cancers, and as a result, there is a pressing need for more effective therapies. Investigational treatments targeting WT1 in GBM have shown a potential correlation between expansion of antigen-specific T cells and survival" [1].

Matteo Levisetti, MD, chief medical officer at Cue Biopharma, added, "Glioblastoma is an immunologically ‘cold’ tumor representing a disadvantage for treatment with standard immunotherapies such as checkpoint inhibitors, but is known to express high levels of the Wilms’ Tumor 1 oncofetal protein. We believe the mechanism of action of CUE-102, to preferentially activate and expand WT1 tumor-specific T cells, has the potential to activate and generate an enhanced anti-tumor immune response against glioblastoma" [1].

CUE-102 has demonstrated anti-tumor activity and a favorable tolerability profile with no dose-limited toxicities observed in a Phase 1 open-label, dose escalation and expansion trial (NCT05360680) for patients with late-stage colorectal, gastric/gastroesophageal junction, pancreatic, and ovarian cancers that express WT1 [1].

Cue Biopharma is developing a novel class of injectable biologics to selectively engage and modulate disease-specific T cells directly within the patient’s body. The company’s proprietary platform, Immuno-STAT® (Selective Targeting and Alteration of T cells), and biologics are designed to harness the curative potential of the body’s intrinsic immune system without the adverse effects of broad systemic immune modulation [1].

References:
[1] https://www.globenewswire.com/news-release/2025/08/13/3132505/0/en/Cue-Biopharma-Announces-Initiation-of-Investigator-Sponsored-Trial-of-CUE-102-in-Recurrent-Glioblastoma-Multiforme.html
[2] https://www.stocktitan.net/news/CUE/cue-biopharma-announces-initiation-of-investigator-sponsored-trial-sv46v2wqgk0b.html