Cue Biopharma to Launch CUE-101 Immunotherapy Trial for Head and Neck Cancer Caused by HPV

The U.S. Food and Drug Administration (FDA) has accepted Cue Biopharma‘s investigational new drug (IND) application for its immunotherapy candidate CUE-101, clearing the way for the first-in-human Phase 1 trial in people with head and neck squamous cell carcinoma (HNSCC) caused by human papillomavirus (HPV).

CUE-101 is an immuno-oncology biologic therapy that is based on Cue’s proprietary platform immuno-STAT, which causes the selective targeting and alteration of T-cells.  This novel therapy was specifically designed to activate and expand a population of anti-cancer-specific T-cells to fight HPV-driven tumors.

It combines an IL-2 signaling molecule — a potent activator of immune cells — with a fragment of E7 protein, which is carried by malignant types of HPV. This particular cocktail is expected to boost anti-tumor, E7-driven T-cell responses without the typical side effects associated with many immunotherapies, which can affect immune cells other than just T-cells.

In preclinical studies, CUE-101 showed it could selectively bind to T-cells, and aid in the activation and expansion of E7-specific targeting cells. It also demonstrated the potential to stop tumor growth when used as a single therapy, and also in combination with a PD-1 inhibitor.

Encouraged by these results, Cue Biopharma filed an IND to push the therapy forward to clinical studies in people, which was now cleared by the FDA.

“The ability to offer a therapeutic with the potential to activate and amplify cancer-specific T cells directly in a patient’s body differentiates CUE-101 from other immunotherapies in development,” Ken Pienta, MD, chief medical officer of Cue Biopharma, said in a press release. “We are pleased to enter the clinic with CUE-101 and provide patients suffering from HPV-driven cancers with this promising new clinical drug candidate.”

The first planned trial will be an open-label, multi-center Phase 1 study aimed at assessing the safety and tolerability of CUE-101 in people with recurrent or metastatic (spread) HNSCC — a type of cancer that can be caused by HPV.

Researchers will determine the maximum tolerated dose, and establish the recommended dose of CUE-101 to be used in future Phase 2 studies, based on markers of biological activity. Other trial aims including measuring the therapy’s pharmacokinetics — its absorption and distribution in the body — anti-tumor immune response, and preliminary anti-tumor effectiveness.

This trial will be conducted in the U.S. and is expected to enroll approximately 50 patients.

“Our clinical trial of CUE-101 aims to replicate promising pre-clinical studies that have shown the ability to expand specific T cell populations exhibiting polyfunctionality, which is characteristic of potent anti-tumor activity,” said Dan Passeri, president and CEO of Cue Biopharma. “We believe CUE-101 will enhance anti-tumor immunity in patients with HPV 16-driven malignancies.”

With the IND acceptance, Cue Biopharma earned $2.5 million from LG Chem Life Sciences, the life sciences division of LG Chem Ltd.. The milestone payment was part of the companies’ licensing agreement to develop multiple Immuno-STAT biologics in the field of oncology.

HPV cancers lead to over 20,000 deaths each year in the U.S. and Europe, mostly due to HPV 16. Approximately 50% of patients with advanced disease will see their cancer come back after standard treatment. People with HPV-related head and neck, cervical, and genitoanal cancers “represent an important unmet clinical need and underscore the opportunity for promising new therapeutics,” the company said.