Poziotinib for Patients With HER2 Exon 20 Mutant Non–Small-Cell Lung Cancer: Results From a Phase II Trial

Author(s): Yasir Y Elamin 1, Jacqulyne P Robichaux 1, Brett W Carter 2, Mehmet Altan 1, Don L Gibbons 1, Frank V Fossella 1, Vincent K Lam 1 3, Anisha B Patel 4, Marcelo V Negrao 1, Xiuning Le 1, Frank E Mott 1, Jianjun Zhang 1, Lei Feng 5, George Blumenschein Jr 1, Anne S Tsao 1, John V Heymach 1
Source: J Clin Oncol. 2022 Mar 1;40(7):702-709. doi: 10.1200/JCO.21.01113. Epub 2021 Sep 22.
Lucio Gordan MD

Dr. Gordan's Thoughts

Another trial corroborating the activity of poziotinib in this setting.


Targeted therapies against non–small-cell lung cancer (NSCLC) harboring HER2 mutations remain an unmet need. In this study, we assessed the efficacy and safety of poziotinib in patients with HER2 exon 20 mutant advanced NSCLC in a single-arm, open-label, phase II study.


Patients with advanced HER2 exon 20 mutant NSCLC were enrolled to receive poziotinib at a dose of 16 mg/d for 28-day cycles. The primary end point was objective response rate per RECIST version 1.1. Confirmatory scans were performed at least 28 days from initial radiologic response.


Thirty patients received poziotinib treatment. At baseline, 90% of patients received prior platinum-based chemotherapy and 53% had two lines or more prior systemic therapies. As of data cutoff on March 1, 2021, the confirmed objective response rate was 27% (95% CI, 12 to 46). Responses were observed across HER2 exon 20 mutation subtypes. The median duration of response was 5.0 months (95% CI, 4.0 to not estimable). The median progression-free survival was 5.5 months (95% CI, 4.0 to 7.0). The median overall survival was 15 months (95% CI, 9.0 to not estimable). The most common grade 3 treatment-related adverse events were skin rash (47%) and diarrhea (20%). There was one possible treatment-related death because of pneumonitis.


Poziotinib showed promising antitumor activity in patients with HER2 exon 20 mutant NSCLC including patients who had previously received platinum-based chemotherapy.

Author Affiliations

1Department of Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX.2Department of Thoracic Imaging, The University of Texas MD Anderson Cancer Center, Houston, TX.3Department of Medicine, Johns Hopkins Sidney Kimmel Cancer Center, Baltimore, MD.4Department of Dermatology, The University of Texas MD Anderson Cancer Center, Houston, TX.5Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX.

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