CG0070 Plus Pembrolizumab Yields Positive Preliminary Data in Non-Muscle Invasive Bladder Cancer

Patients with non-muscle invasive bladder cancer unresponsive to Bacillus Calmette-Guerin may benefit from treatment with CG0070 plus pembrolizumab.

Treatment with CG0070 and pembrolizumab (Keytruda) resulted in promising preliminary findings in a small population of patients with non-muscle invasive bladder cancer who were unresponsive to Bacillus Calmette-Guerin (BCG), according to a press release on the phase 2 CORE1 trial (NCT04387461).

The preliminary findings indicated that the combination was well-tolerated with promising efficacy in the study’s population of 9 patients. As of the data cut off in November 2021 and at the initial 3-month timepoint, investigators reported that all patients achieved a complete response (CR). All patients who reached additional timepoints (n = 6) maintained a CR at 6 months and 3 maintained a CR at 9 months.

“These preliminary results are exciting,” Roger Li, MD, lead study investigator and urologic oncologist at Moffitt Cancer Center, said in a press release. “If similar trends hold moving forward, we may have a game changer to combat BCG-unresponsive bladder cancer for patients with significant unmet medical need.”

The open label trial has an estimated enrollment of 37 patients. The study’s primary end point is CR with key secondary end points including safety, duration of response, overall survival, and progression-free survival.

To enroll on the trial, patients were required to have an ECOG performance status of 0 or 1, pathologically confirmed disease, and be unresponsive to BCG therapy. Additionally, patients needed to be ineligible or have refused radical cystectomy and have adequate organ function. Patients who were immunodeficient because of treatment with chronic steroids or immunosuppressants, human immunodeficiency virus, or organ transplant were not eligible for the study. Other exclusion criteria included previous treatment with adenovirus-based therapy or checkpoint inhibitors, clinically significant or active cardiac disease, or active autoimmune disease.

In terms of safety, adverse effects (AEs) were limited to grade 1/2 local genitourinary symptoms and immune-related AEs. These included urinary frequency, bladder spasm, fatigue, chills, autoimmune thyroiditis, blood discharge, dysuria, and flu-like symptoms. Investigators did not report any grade 3 or higher AEs.

“We are excited to announce these preliminary results toward CG0070’s safety, tolerability, and clinical efficacy in patients with bladder cancer unresponsive to BCG who have limited treatment options. We hope to see continued responses as the study progresses, as CG0070’s dual mechanism of action has shown to be highly effective in this difficult-to-treat patient population,” Arthur Kuan, chief executive officer at CG Oncology, concluded.


CG Oncology presents preliminary phase 2 data with CG0070 in combination with KEYTRUDA® (pembrolizumab) in non-Muscle-invasive bladder cancer unresponsive to Bacillus Calmette-Guerin. News release. CG Oncology. November 13, 2021. Accessed November 23, 2021.

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