Cue Biopharma, Inc. announced the presentation of new positive data from its ongoing Phase 1 trial evaluating its lead interleukin 2 (IL-2)-based biologic, CUE-101 in combination with pembrolizumab (KEYTRUDA), as a first line therapy for patients with recurrent/metastatic HPV+ head and neck squamous cell carcinoma (HNSCC). The data will be presented in a poster at the Society for Immunotherapy of Cancer's 37th Annual Meeting (SITC 2022), to be held in Boston, Massachusetts and virtually on November 8-12, 2022. The poster will also provide an update from the Company's ongoing fully enrolled Phase 1b trial evaluating CUE-101 monotherapy as third line and beyond therapy in the same patient population.

Additionally, the Company will present two posters discussing the design of its ongoing Phase 1 trial evaluating its second IL-2 based candidate, CUE-102, for the treatment of Wilms' Tumor 1 positive (WT1+) malignancies, and preclinical data regarding its mechanistic effect in vitro and in vivo. Key data highlights from the CUE-101 dose escalation and patient expansion portion of the Phase 1 trial in combination with pembrolizumab with 16 evaluable patients to date, include: Overall response rate (ORR) of 40% and a clinical benefit rate (CBR) of 70% in the first 10 evaluable patients treated at the recommended Phase 2 dose (RP2D) of 4 mg/kg of CUE-101 and 200 mg of pembrolizumab administered every three weeks – This includes four confirmed partial responses (cPR) in addition to three durable stable disease (DSD) of = 12 weeks. These responses include patients with low PD-L1 expression (combined positive score (CPS) less than 20).

At the CUE-101 2 mg/kg dose plus pembrolizumab (n=3), one patient experienced a cPR and one DSD, for a CBR of 67%. At the CUE-101 1 mg/kg dose plus pembrolizumab (n=3), one patient experienced DSD, for a CBR of 33%. Notably, tumor reduction from baseline in the five patients with confirmed PRs was between 35% and 69%.

Key data highlights from the CUE-101 expansion portion of the Phase 1b trial at the RP2D as a monotherapy to date, include: 42% overall clinical benefit rate, including one PR of > 42 weeks duration and seven DSD. Median overall survival approaching greater than 12 months in third line and beyond (3L+) patients treated with C E-101 monotherapy, which is 50% greater than current standard of care with anti-PD-1 therapies in second line (2L) patients. CUE-101 continued to show a favorable tolerability profile both as monotherapy and in combination with pembrolizumab.

Ken Pienta, M.D., acting chief medical officer of Cue Biopharma, added, “This new data from combination study demonstrates early evidence of complementary mechanistic activity of CUE-101 with pembrolizumab. In addition, the sustained clinical benefit rate with CUE-101 as monotherapy is very encouraging and has continued to demonstrate proof-of-concept of CUE-101 as a single agent. Overall, the data shows the potential of CUE-101 to provide patients with an improved clinical benefit rate and with lower toxicity than current standard of care.

The company looks forward to continuing to evaluate data from the trial in addition to defining the potential registrational trial for CUE-101, which the company anticipates in mid-2023.” Key data highlights from CUE-102 preclinical study to date include: CUE-102 selectively activated and expanded WT1-specific CD8+ T cells from peripheral blood mononuclear cells (PBMC) of healthy donors. CUE-102 elicited and expanded WT1-specific CD8+ T cells from naïve mice without significantly altering the frequencies of other immune lineages. The WT1-specific CD8+ T cells expanded in vivo exhibited polyfunctionality and selectively killed WT1-presenting target cells in vitro and in vivo.