Phase 1 Trial of CUE-102 in WT1+ Solid Tumors is Now in Motion

Certain patients with Wilm's tumor-expressing solid tumors are eligible to be treated with an investigational agent in a phase 1 clinical trial.

The first patient was dosed in a phase 1 dose escalation study (NCT05360680) which is evaluating CUE-102, of the interleukin 2 (IL-2) series, as monotherapy for the treatment of patients with Wilms’ Tumor 1 (WT1)-positive recurrent/metastatic cancers. The study will focus on colorectal, gastric, pancreatic, and ovarian cancers, according to a press release from Cue Biopharma.1

“CUE-102 has the potential to activate the patient’s immune system against numerous WT1-expressing cancers, including solid tumors and hematologic malignancies, and has demonstrated selective and significant activation of WT1-specific T cells in preclinical studies. We believe that CUE-102 can play an important role in changing the treatment landscape for patients with WT1-positive cancers, by potentially delivering higher efficacy and lower toxicities than current available treatments, said Ken Pienta, MD, acting chief medical officer of Cue Biopharma, in a press release.

About the Phase 1 Study of CUE-102

Trial Name: A Phase 1, Open-Label, Dose Escalation and Expansion Study of CUE-102 Monotherapy in HLA-A*0201 Positive Patients With WT1 Positive Recurrent/Metastatic Cancers Indentifier: NCT05360680

Sponsor: Cue Biopharma

Recruitment Contact: steven Margossian, MD, PhD 857-228-0636 and Reena Lynam 617-949-2637

Completion Date: December 2026

In a preclinical study of CUE-102 tested humans with peripheral blood mononuclear cells (PBMC) for cellular activity and specificity, while in vivo CUE-102 is testing in mice who are human leukocyte antigen HLA-A2 transgenic. Investigators found that CUE-102 both activates and expands WT1-specific CD8-positive T cells from PBMC of healthy donors. Similar to results seen with CUE-101, CUE-102 showed significant functional reduction of the IL-2 components.The in vivo study found CUE-102 expands polyfunctional WT1-specific CD8-positive T cells from mice who were naïve and previously immunized, but the treatment did not alter the frequencies of other immune lineages.The in vivo study also found the WT1-specific CD8-positive T cells had a polyfunctional response to peptide-loaded target cells and selectively killed WT1-presenting target cells.These results, along with a tolerable safety profile, support the initiation of the phase 1 trial of CUE-102.2

This phase 1 study aims to find the dose-limiting toxicity and maximum-tolerated dose of CUE-102. The secondary end points include safety and tolerability, antitumor response rate, antitumor duration of response, antitumor clinical benefit rate, progression-free survival, and overall survival. The dose escalation portion of the trial will administer CUE-102 in intravenous doses of 1 mg/kg, 2 mg/kg, 4 mg/kg, 8 mg/kg, and an experimental recommended phase 2 dose every 3 weeks for up to 2 years.3

Eligible patients in this phase 1 study will include patients who have colorectal, gastric, pancreatic, or ovarian cancer who have an ECOG performance status of 0 or 1, a life expectancy of at least 12 weeks, have a human leukocyte antigen (HLA)-A*0201 genotype as determined by genomic testing, and tumors who are WT1-positive. Patients will be deemed ineligible if patients with central nervous system metastases have been treated and be asymptomatic, have a history of prior allogeneic bone marrow, stem cell, or solid organ transplant, treatment with radiation therapy within 14 days before the first dose of CUE-102, and a history of clinically significant cardiovascular disease.3

“Initiating this phase 1 clinical study of CUE-102 at a starting dose of 1mg/kg, a clinically active dose in our Phase 1 CUE-101 clinical trial for HPV+ head and neck cancer, is an important step forward in demonstrating the modularity of our Immuno-STAT™ platform and the broader clinical potential of our CUE-100 series of biologics,” said Dan Passeri, chief executive officer of Cue Biopharma, in the press release. “We believe, given the preservation of the core molecular framework between CUE-102 and CUE-101 with the primary exception of the tumor-specific epitope, initiating the dose escalation trial at 1 mg/kg will result in reduced time and cost to evaluate tolerability at therapeutically active doses.”


1. Cue biopharma doses first patient in phase 1 study of CUE-102 for Wilms’ Tumor 1 (WT1) - expressing cancers. Press release. Cue Biopharma; August 22, 2022. Accessed August 23, 2022.

2. Zhang C, Girgis N, Merazga Z, et al. 720 CUE-102 selectively activates and expands WT1-specific T cells for the treatment of patients with WT1+ malignancies. Journal for ImmunoTherapy of Cancer. 2021;9:doi: 10.1136/jitc-2021-SITC2021.720

3. A phase 1 in patients with HLA-A*0201+ and WT1+ recurrent/metastatic cancers. Updated July 7, 2022. Accessed August 23, 2022.

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