Faculty, Staff and Student Publications

Publication Date

3-1-2025

Journal

Nature Medicine

DOI

10.1038/s41591-024-03443-3

PMID

39863775

PMCID

PMC11922750

PubMedCentral® Posted Date

1-25-2025

PubMedCentral® Full Text Version

Post-print

Abstract

Encorafenib + cetuximab (EC) is approved for previously treated BRAF V600E-mutant metastatic colorectal cancer (mCRC) based on the BEACON phase 3 study. Historically, first-line treatment of BRAF V600E-mutant mCRC with chemotherapy regimens has had limited efficacy. The phase 3 BREAKWATER study investigated EC+mFOLFOX6 versus standard of care (SOC) in patients with previously untreated BRAF V600E mCRC. The dual primary endpoint of progression-free survival is event driven; data were not mature at data cutoff. BREAKWATER met the other dual primary endpoint of objective response rate, demonstrating significant and clinically relevant improvement in objective response rate (EC+mFOLFOX6: 60.9%; SOC: 40.0%; odds ratio, 2.443; 95% confidence interval (CI): 1.403-4.253; 99.8% CI: 1.019-5.855; one-sided P = 0.0008). Median duration of response was 13.9 versus 11.1 months. At this first interim analysis of overall survival, the hazard ratio was 0.47 (95% CI: 0.318-0.691; repeated CI: 0.166-1.322). Serious adverse event rates were 37.7% versus 34.6%. The safety profiles were consistent with those known for each agent. BREAKWATER demonstrated a significantly improved response rate that was durable for first-line EC+mFOLFOX6 versus SOC in patients with BRAF V600E mCRC. ClinicalTrials.gov identifier: NCT04607421 .

Keywords

Adult, Aged, Female, Humans, Male, Middle Aged, Antineoplastic Combined Chemotherapy Protocols, Carbamates, Cetuximab, Colorectal Neoplasms, Fluorouracil, Leucovorin, Mutation, Organoplatinum Compounds, Progression-Free Survival, Proto-Oncogene Proteins B-raf, Sulfonamides

Published Open-Access

yes

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