Aims: The phase 1b KEYNOTE-028 trial showed durable activity with pembrolizumab monotherapy in previously treated HR+/HER2−, PD-L1–positive (combined positive score [CPS] ≥1) advanced breast cancer. KEYNOTE-B49 (NCT04895358) is a phase 3, double-blind study of pembrolizumab+chemotherapy vs placebo+chemotherapy in PD-L1–positive, HR+/HER2− locally recurrent inoperable or mBC after progression on prior endocrine therapy.
Methods: ⁓800 patients with HR+/HER2− locally recurrent inoperable or mBC who are candidates for chemotherapy (no prior chemotherapy for metastatic disease) with PD-L1 CPS ≥1 and documented PD on prior endocrine therapy will be enrolled. Prior endocrine therapy comprises ≥2 lines (≥1 in combination with a CDK4/6 inhibitor) in the metastatic setting (group 1); or 1 line with CDK4/6 inhibitor treatment for mBC in patients with relapse ≤24 months of primary surgery while on adjuvant endocrine therapy (group 2a); or PD ≤12 months of starting endocrine therapy with CDK4/6 inhibitor for metastatic disease (group 2b). Patients without prior CDK4/6 inhibitor treatment may enroll if they had PD ≤6-months of starting endocrine therapy for metastatic disease and previously relapsed ≤24-months of primary tumor surgery while on adjuvant endocrine therapy (group 3). Patients are randomized 1:1 to pembrolizumab 200 mg IV or placebo Q3W, each in combination with investigator’s choice of chemotherapy: paclitaxel 90 mg/m2 IV on days (D) 1/8/15 Q4W; nab-paclitaxel 100 mg/m2 IV on D1/8/15 Q4W; liposomal doxorubicin 50 mg/m2 IV on D1 Q4W; or capecitabine 1000 mg/m2 PO BID on D1–14 Q3W. Treatment continues until PD, unacceptable toxicity, withdrawal, or, for pembrolizumab/placebo, completion of 35 cycles (~2y); chemotherapy can continue per investigator discretion. Radiologic assessments are performed Q9W for 54 weeks and Q12W thereafter. Primary endpoints are PFS per RECIST v1.1 by BICR and OS in patients with PD-L1 CPS ≥10 and ≥1 tumors, separately. Enrollment is ongoing at 204 international sites.