DOI: 10.1158/1078-0432.ccr-23-1030 ISSN:

A Phase 2 Trial of Rivoceranib, an Oral Vascular Endothelial Growth Factor Receptor 2 Inhibitor, for Recurrent or Metastatic Adenoid Cystic Carcinoma

Glenn J. Hanna, Myung-Ju Ahn, Jameel Muzaffar, Bhumsuk Keam, Daniel W. Bowles, Deborah J. Wong, Alan L. Ho, Sung-Bae Kim, Francis Worden, Tak Yun, Xianzhang Meng, Jan M. Van Tornout, Maureen G. Conlan, Hyunseok Kang
  • Cancer Research
  • Oncology


Purpose: This open-label, single-arm, phase 2 study evaluated the vascular endothelial growth factor receptor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) rivoceranib in patients with recurrent or metastatic (R/M) adenoid cystic carcinoma (ACC). Patients and Methods: Eligible patients had confirmed disease progression per Response Evaluation Criteria in Solid Tumors (RECIST) with ≥20% increase in radiologically or clinically measurable lesions or appearance of new lesions within the preceding 6 months. Patients received oral rivoceranib 700 mg once daily. Primary outcomes were objective response rate (ORR) by investigator review and by blinded independent review committee (BIRC). Results: Eighty patients were enrolled and 72 were efficacy evaluable. Seventy-four patients had distant metastases and 49 received prior systemic treatment (14 received VEGFR TKIs). Per investigator and BIRC, respectively, ORR was 15.3% (95% CI, 7.9–25.7) and 9.7% (95% CI, 4.0–19.0); median duration of response was 14.9 months (95% CI, 4.9–17.3) and 7.2 months (95% CI, 3.5–8.4); and median progression-free survival was 9.0 months (95% CI, 7.3–11.5) and 9.0 months (95% CI, 7.7–11.5). Grade ≥3 treatment-related adverse events occurred in 56 patients (70.0%); the most common were hypertension (34, 42.5%) and stomatitis (6, 7.5%). Four grade 5 events occurred with one attributed to rivoceranib (epistaxis). Sixty-eight patients (85.0%) had ≥1 dose modifications and 16 patients (20.0%) discontinued rivoceranib for toxicity. Conclusions: In patients with progressing R/M ACC, rivoceranib demonstrated anti-tumor activity and a manageable safety profile consistent with other VEGFR TKIs.

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