Cue Biopharma Announces Positive Results for CUE-221 in Chronic Urticaria Study

Cue Biopharma, a clinical-stage biopharmaceutical company, announced positive topline results from the Phase 2 clinical trial of CUE-221 in chronic spontaneous urticaria (CSU). The trial, conducted in China by Genesis Life Sciences, a related company of Ascendant Health Limited, involved 145 participants with moderate to severe CSU, a condition characterized by chronic hives and recurring itch. The study aimed to evaluate the efficacy and safety of CUE-221 compared to placebo and omalizumab, a current standard treatment for CSU.

The primary endpoint of the study, the proportion of patients who achieved a Histamine Skin Scratch Test (HSS) score of 0 at week 12, was met with high statistical significance. Additionally, a key secondary endpoint, the proportion of patients who achieved a Urticaria Activity Score (UAS) of 0 at week 12, was dose-responsive and met statistical significance at the highest dose level of 4 mg/kg. The results showed that the percentage of participants who achieved HSS7=0 further increased beyond the Week 12 primary endpoint, peaking at Week 22 across all CUE-221 dose groups. After the last dose was administered at Week 16, the clinically meaningful benefit was maintained for up to 12 weeks off treatment (through week 28) at the 4 mg/kg dose level. A higher rate of complete resolution of hives was observed for the 4 mg/kg dose group relative to the lower dose groups and placebo, with a post hoc analysis at Week 28 demonstrating a statistically significant difference versus omalizumab (delta = 36%).

CUE-221 demonstrated a favorable safety profile, with no treatment-related serious adverse events and no cases of hypersensitivity reactions, including anaphylaxis. Injection site reactions were infrequent, with only one reaction graded above 1, and none led to study discontinuation. The study was designed to test superiority over placebo, with omalizumab included for comparative efficacy without planned statistical testing.

The results of the Phase 2 CUE-221 study in patients with CSU are particularly notable, according to Dale Umetsu, M.D., Ph.D., a clinical professor of medicine and former chief of the Allergy and Immunology Division at Stanford University. Dr. Umetsu stated, "First, the results after 12 weeks of treatment, after three doses, appear to indicate that CUE-221 was better than XOLAIR for CSU at all dose levels tested. Moreover, there was persistent improvement with the 4 mg/kg dose at week 28, which was 12 weeks off treatment, significantly better than that off of standard XOLAIR dosing. Finally, the safety data analyzed so far, show no major safety issues, which is not unexpected from an anti-IgE mAb."

Dr. Umetsu added, "These results suggest that CUE-221, like XOLAIR, is designed to prevent IgE from binding to FceR1 but unlike XOLAIR, allows IgE to bind to CD23, may represent a critical advancement over XOLAIR. The results support that the effects of CUE-221 on IgE function could result in substantial efficacy in CSU that persists for several months, even after dosing ends. Since XOLAIR has represented the clear standard for all other CSU therapies, the possibility that CUE-221 may represent a clear improvement above that of XOLAIR is most impressive."

Cue Biopharma will host a conference call and webcast on Monday, September 21, at 8:00 a.m. EDT to present the results. The event will be webcast live and can be accessed via the attached link: edge.media-server.com/mmc/p/mjexxy37

The positive results from the Phase 2 study of CUE-221 in chronic spontaneous urticaria highlight the potential of this novel anti-IgE antibody with a dual mechanism of action. The company plans to advance CUE-221 in a Phase 2b/3 study in chronic spontaneous urticaria and in a Phase 2 study in food allergy.