Scientific Publications | 15 Jan 2024

European Journal of Cancer: Surufatinib plus toripalimab in patients with advanced neuroendocrine tumours and neuroendocrine carcinomas: An open-label, single-arm, multi-cohort phase II trial

Surufatinib plus toripalimab in patients with advanced neuroendocrine tumours and neuroendocrine carcinomas: An open-label, single-arm, multi-cohort phase II trial

Panpan Zhang, Si Shi, Jianming Xu, Zhendong Chen, Lijie Song, Xing Zhang, Ying Cheng, Yanqiao Zhang, Feng Ye, Zhiping Li, Fei Yin, Dongmei Ji, Heli Gao, Yi Li, Wei Chen, Minjie Yang, Desheng Weng, Chunjiao Wu, Yue Ma, Wang Sheng, Yaqin Zhao, Xiaolei Yin, Weina Shen, Weiguo Su, Michael Shi, Songhua Fan, Panfeng Tan, Qian Xu, Ming Lu and Lin Shen

Highlights

Treatment options are limited for patients with neuroendocrine neoplasm.
Here, surufatinib plus toripalimab show tolerable safety in neuroendocrine neoplasm.
The regimen shows potential synergistic interaction in neuroendocrine carcinoma.
Data warrant phase III study in patients with neuroendocrine carcinoma.

Abstract

Background

The programmed death 1 inhibitor toripalimab plus the angio-immuno kinase inhibitor surufatinib revealed a tolerable safety profile and preliminary efficacy in patients with advanced solid tumours in a phase I study.

Patients and Methods

This was an open-label, single-arm, multi-cohort phase II study in China. Patients with advanced neuroendocrine tumours (NETs) or neuroendocrine carcinomas (NECs) or mixed neuroendocrine non-neuroendocrine neoplasms (MiNENs) who had failed or were intolerable of standard treatment were given surufatinib (250 mg orally, once daily) plus toripalimab (240 mg intravenously, once every 3 weeks). Primary end-point was investigator-assessed objective response rate (ORR) per Response Evaluation Criteria in Solid Tumors version 1.1. Secondary end-points included duration of response (DoR), disease control rate, progression-free survival (PFS), overall survival (OS), and safety.

Results

Forty patients were enrolled into two cohorts by tumour types (NET, n = 19; NEC-MiNEN, n = 21). ORRs (95% CIs) were 21.1% (6.1–45.6) and 23.8% (8.2–47.2) in the NET and NEC-MiNEN cohorts, respectively. Median DoR was 7.1 months (6.9–not evaluable [NE]) and 4.1 months (3.0–NE), respectively. Median PFS was 9.6 months (4.1–NE) and 4.1 months (1.5–5.5); median OS was 27.3 (15.3–NE) and 10.9 months (9.1–14.6), respectively. Overall, grade ≥ 3 treatment-related adverse events occurred in 18 (45.0%) patients.

Conclusions

Surufatinib plus toripalimab showed antitumour activity and a tolerable safety profile in patients with previously treated NETs/NECs/MiNENs. Further study of this combination regimen is ongoing for advanced NECs, for which current therapeutic options remain limited.

Trial Registration

ClinicalTrials.gov Identifier: NCT04169672

Citations and Links

Please follow the link below to access the publication:

DOI: https://doi.org/10.1016/j.ejca.2024.113539

Link to article: https://www.ejcancer.com/article/S0959-8049(24)00015-7