FDA Grants Fast Track Designation to CUE-101 for Recurrent/Metastatic HNSCC


Building on data showing its preclinical anti-tumor activity, CUE-101 will be examined in a phase 1b study and has been granted FDA fast track designation.

The FDA granted fast track designation to the interleukin 2 biologic CUE-101, for the treatment of patients with human papillomavirus (HPV16+) recurrent/metastatic head and neck squamous cell carcinoma (HNSCC), according to an announcement by Cue Biopharma.1

CUE-101 activates and expands HPV16 tumor-specific T cells. Across multiple tumor types, proof-of-concept for the agent has been established. Currently, CUE-101 is being investigated for the treatment of HPV16+ driven recurrent or metastatic HNNSCC as a single-agent and in combination with pembrolizumab (Keytruda).

We are very pleased to have received Fast Track designation from the FDA for CUE-101. This designation not only underscores the large unmet need for patients with R/M head and neck cancer who currently rely on available non-targeted therapies, but also highlights the potential of CUE-101 to provide a significant clinical benefit,” said Matteo Levisetti, MD, senior vice president, Clinical Development of Cue Biopharma, in a press release. “To date in its Phase 1b clinical trials, CUE-101 has demonstrated a favorable tolerability profile and single-agent anti-tumor activity in monotherapy as well as encouraging anti-tumor clinical activity in combination with pembrolizumab, supporting the potential to improve overall survival (OS) for these patients.”

In the phase 1b first-in-human, open-label, dose escalation and expansion study, the safety, anti-tumor effect, and immunogenicity of CUE-101 will be assessed. The coprimary end points of the study are dose-limiting toxicity and pharmacokinetics. The secondary end point being explored is the overall response rate.2

Part A and B will be the dose-escalation and dose-expansion phases during which CUE-101 will be administered via intravenous (IV) infusion. In parts C and D, CUE-101 will be administered IV at the recommended phase 2 dose in combination with pembrolizumab 200 mg by injection.

The study is actively recruiting patients aged 18 years or older with recurrent of metastatic HNSCC, an ECOG performance status of 0 or 1, a life expectancy ≥ 12 weeks, measurable disease per RECIST 1.1., histologically and/or cytologically proven HPV16-positive tumors, and adequate laboratory results. Patients must also have the HLA A*0201 genotype.

“We look forward to providing periodic updates and remain committed to advancing the development of CUE-101 to provide patients with a potentially more effective and better tolerated treatment option. We anticipate initiating a registrational trial for CUE-101 monotherapy by mid-2023,” said Levisetti.1


1. Cue Biopharma granted FDA fast track designation for CUE-101 for the treatment of recurrent/metastatic head and neck squamous cell carcinoma. News release. Cue Biopharma. October 4, 2022. Accessed October 4, 2022. https://bit.ly/3SSm66j

2. A phase 1 study in patients with HPV16+ recurrent/ metastatic head and neck squamous cell carcinoma. Clinicaltrials.gov. Updated August 4, 2022. Accessed October 4, 2022. https://clinicaltrials.gov/ct2/show/NCT03978689

Related Videos
Related Content