Clinical Trials

In September 2019, we initiated a first-in-human, multi-center, open-label, Phase 1 dose-escalation and dose-expansion study of CUE-101, our lead drug candidate in eligible HNSCC patients. CUE-101 is a novel fusion protein designed to activate and expand tumor-specific T cells that target Human Papilloma Virus 16 (HPV16)-driven malignancies. HPV16 causes multiple tumor types including head and neck squamous cell carcinoma (HNSCC), cervical and anal cancers. Cue Biopharma has engaged a network of nationally recognized clinical investigators and 13 Phase 1 sites are actively recruiting patients.

The primary objectives of this Phase 1 trial are to assess the safety and tolerability, preliminary antitumor activity and potential for an antitumor immune response following CUE-101 treatment in patients suffering with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC).

Eligible patients with HNSCC will have progressive disease after at least one prior systemic therapy, including a platinum-based chemotherapy, and have been offered checkpoint immunotherapy. The requirement for prior systemic therapy may be waived if a patient was intolerant of or refused standard first or second-line therapies. In addition, patients must have:

  • histologically and/or cytologically confirmed HPV16+ tumors expressing 16INK4A, a known biomarker of HPV+ malignancies, by a central laboratory, and
  • an HLA-A*0201 genotype as determined by genomic testing performed at a central laboratory

Abbreviations used in the above table: 2L+, two or more prior lines of therapy; BED, best effective dose; H&N, head and neck; HPV, human papilloma virus; MTD, maximum-tolerated dose; PK/PD, pharmacokinetics/pharmacodynamics; Q3W, dosing once every 3 weeks; R/M, recurrent/metastatic; RP2D, recommended Phase 2 dose; TCR, T cell receptor

HPV-driven cancers account for more than 20,000 deaths each year in the U.S. and Europe. The majority of these cancers are driven by HPV 16 which carries the E7 antigen targeted by CUE-101. Despite treatment with current standards of care, approximately 50% of patients with advanced disease will experience recurrence and a significant quality of life impact. Patients with HPV-related head and neck, cervical and genitoanal cancers represent an important unmet clinical need and underscore the opportunity for promising new therapeutics.

For more information on the CUE-101 study, please visit (NCT03978689).