Association Between Objective Response and Overall Survival in BRAF V600e-Mutant Metastatic Colorectal Cancer: An Analysis of the BEACON CRC Trial

Author(s)

Maria Cecilia Vieira, MS1, Xiaosong Zhang, MD, PhD1, Benjamin Li, PhD1, Mu Cheng, MPH2, Kalé Kponee-Shovein, ScD, MS, MPH2, James Signorovitch, PhD2, Jingyi Liu, MBI2, Aparna Parikh, MD3.
1Pfizer Inc., New York, NY, USA, 2Analysis Group, Inc., Boston, MA, USA, 3Massachusetts General Hospital, Boston, MA, USA.
OBJECTIVES: To quantify the association between objective tumor response and overall survival (OS) in patients with BRAF V600E-mutant metastatic colorectal cancer (mCRC) receiving encorafenib + cetuximab (EC)-based therapy. Clarifying this association may inform the relevance of tumor response in clinical, regulatory, and reimbursement decision-making for this high-risk population.
METHODS: This retrospective analysis used data from the intent-to-treat population of the phase 3 BEACON CRC trial (data cutoff: Nov 10, 2022). Patients in BEACON CRC were randomized to EC + binimetinib (BINI), EC alone, or control (cetuximab + irinotecan/FOLFIRI). Landmark analyses compared OS between responders (complete or partial response) and non-responders, based on RECIST v1a.1 criteria, at 3, 6, and 9 months post-randomization. Adjusted hazard ratios (aHRs) and 95% confidence intervals (CIs) were estimated using Cox models, adjusting for prior lines of therapy. Analyses were conducted in the pooled EC arms (with or without BINI) and within each arm.
RESULTS: Among patients receiving EC-based therapy (N=444; median age: 61.0 years; 65.8% with 1 prior line of therapy), the objective response rate was 23.6% (95% CI: 19.8-27.9), and median OS was 9.6 months (95% CI: 8.5-10.8). Compared with non-responders, responders at 3, 6 and 9 months had a lower risk of death (aHR): 0.59 (95% CI: 0.46-0.76), 0.61 (95% CI: 0.46-0.80), and 0.64 (95% CI: 0.48-0.87), respectively. Results were consistent across treatment arms.
CONCLUSIONS: Achieving a tumor response was associated with reduced mortality risk in patients with BRAF V600E-mutant mCRC receiving EC-based therapy. This association was robust across time points and treatment arms, underscoring the potential relevance of tumor response in this population, particularly when OS data are limited, requiring much longer time for follow-up.

Conference/Value in Health Info

2025-11, ISPOR Europe 2025, Glasgow, Scotland

Value in Health, Volume 28, Issue S2

Code

CO23

Topic

Clinical Outcomes

Topic Subcategory

Relating Intermediate to Long-term Outcomes

Disease

Gastrointestinal Disorders, Oncology

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