A Phase 2 Study of Encorafenib in Combination with Binimetinib in Patients with Metastatic BRAF -Mutated Thyroid Cancer in Japan
An erratum to this article was published (Thyroid, Oct 2025 ↗)
Authors' conclusion
Treatment with encorafenib plus binimetinib met the primary endpoint criteria and demonstrated clinical benefit in patients with BRAFV600E-mutated thyroid cancer regardless of its histological type, such as DTC or ATC, with no new safety concerns identified.
Study context
- Population
- Patients in Japan with BRAF V600-mutated locally advanced or distant metastatic thyroid cancer not amenable to curative treatment, refractory/intolerant to ≥1 previous VEGF receptor-targeted regimen or ineligible for those; 17 differentiated thyroid cancer (DTC), 5 anaplastic thyroid cancer (ATC)17 DTC, 5 ATC
- Participants
- 22 patients
- Outcome
- Centrally assessed objective response rate (ORR)
Methodological detailsopen-label, uncontrolled, 10 institutions
- Open-label
- Uncontrolled
- 10 institutions
Main finding
As reported in the abstract
Centrally assessed ORR · data cutoff October 26, 2022; median follow-up 11.5 (range 3.4-19.0) months
Centrally assessed objective response rate (ORR) (overall)
54.5%
95% CI 32.2–75.6
Partial response
12 patients
Stable disease
10 patients
Secondary outcomes
ORR by histological subgroup
- ORR, DTC
- 47.1% (8 of 17)
- ORR, ATC
- 80.0% (4 of 5)
Duration of response (DOR), progression-free survival (PFS) and overall survival (OS) · 12 months
- Ongoing response rate at 12 months
- 90.9%
- PFS rate at 12 months
- 78.8%
- OS rate at 12 months
- 81.8%
- Medians for DOR, PFS and OS not reached (overall, DTC and ATC)
Safety
Adverse events
- ≥1 AE
- All patients
- Grade 3 adverse events (AEs)
- 6 patients (27.3%)
- Grade 4-5 AEs
- 0 patients
- Most common grade 3 AE: lipase increased
- 4 patients [18.2%]
- Toxicities mostly manageable with monitoring and dose adjustment
Original publication
A Phase 2 Study of Encorafenib in Combination with Binimetinib in Patients with Metastatic BRAF -Mutated Thyroid Cancer in Japan. Thyroid. Published 14 March 2024. doi:10.
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