Efficacy and Tolerability of Zenocutuzumab in Advanced NRG1 Fusion-Positive Cholangiocarcinoma: Results From the eNRGy Phase II Trial.
James M Cleary, Christoph Springfeld, Dirk Arnold et al.
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In 30 seconds
This single-arm, multicenter phase II trial evaluated zenocutuzumab in previously treated or standard-therapy-ineligible adults with advanced NRG1 fusion-positive cholangiocarcinoma. Among 19 patients included in the efficacy analysis, 7 responded, for an investigator-assessed overall response rate of 36.8% (95% CI, 16.3 to 61.6). Median duration of response was 7.4 months and median progression-free survival was 9.2 months (95% CI, 3.9 to 11.1). Most treatment-related adverse events were grade 1 or 2; no patient stopped treatment because of an adverse event.
Key findings
- Seven of 19 patients responded; the overall response rate was 36.8% (95% CI, 16.3 to 61.6).
- Median duration of response was 7.4 months, and median progression-free survival was 9.2 months (95% CI, 3.9 to 11.1).
- The clinical benefit rate was 57.9% (95% CI, 33.5 to 79.7).
- Most treatment-related adverse events were grade 1 or 2; one grade 3 event was anemia, and no patient discontinued because of an adverse event.
Why it matters
NRG1 fusion-positive cholangiocarcinoma has no approved targeted therapy, according to the study. The observed responses and response duration provide clinical evidence of antitumor activity for zenocutuzumab in this molecularly defined population, alongside reported tolerability. These results add to evidence for targeted treatment in advanced disease, while the single-arm design limits comparative conclusions.
What to keep in mind
The study was single-arm, and the efficacy analysis included 19 patients, limiting comparative inference and precision.