China's First! BeiGene's PD-1 Inhibitor Baizean? Approved for 14th Indication

On October 21, BeiGene announced that its PD-1 inhibitor Baizean? (Tislelizumab) has been officially approved by the National Medical Products Administration of China. The drug is approved for use in combination with platinum-based chemotherapy as a neoadjuvant treatment, followed by Baizean? monotherapy as adjuvant treatment after surgery, for the treatment of patients with resectable stage II or IIIA non-small cell lung cancer (NSCLC).

As the first PD-1 drug approved in China for neoadjuvant and adjuvant immunotherapy in stage II-III NSCLC, Tislelizumab provides a new option for perioperative treatment of operable lung cancer patients, helping them achieve longer survival benefits.

This approval is based on clinical trial data from RATIONALE 315 (NCT04379635). The study is a multicenter, randomized, placebo-controlled, double-blind phase 3 trial evaluating the efficacy and safety of Tislelizumab combined with platinum-based doublet chemotherapy as neoadjuvant treatment, followed by adjuvant Tislelizumab therapy in patients with resectable stage II or IIIA NSCLC.

RATIONALE-315 study design

Study results showed that both primary endpoints of event-free survival (EFS) and major pathological response (MPR) rate were achieved, confirming that perioperative treatment with Tislelizumab combined with neoadjuvant chemotherapy significantly reduces the risk of perioperative progression, recurrence, or metastasis.

Compared to placebo combined with neoadjuvant chemotherapy, Tislelizumab combined with neoadjuvant chemotherapy significantly increased the MPR rate (56.2% vs 15.0%) and pathological complete response (pCR) rate (40.7% vs 5.7%), and significantly prolonged EFS (HR: 0.56, 95% CI: 0.40~0.79). A positive trend in overall survival (OS) has also been observed (HR: 0.62, 95% CI: 0.39~0.98). The overall tolerability of this treatment regimen was good, with no new safety signals identified. The results of this study were presented at the European Society for Medical Oncology (ESMO) plenary session in February 2024.

EFS results of ITT and subgroup populations in the RATIONALE-315 study

Summary of EFS results of phase III study on perioperative immunotherapy

Currently, Tislelizumab has been approved by China's National Medical Products Administration for 14 indications, 11 of which have been included in the National Reimbursement Drug List, making it the PD-1 inhibitor with the most approved indications included in the national insurance list.

Globally, Tislelizumab has been approved in the European Union, the United Kingdom, the United States, South Korea, Switzerland, Australia, and other countries and regions, including approval by the European Commission in April this year for three first- and second-line NSCLC indications. Applications for new indications for first-line treatment of gastric or gastroesophageal junction cancer and first-line treatment of esophageal squamous cell carcinoma are under review by the U.S. Food and Drug Administration (FDA) and the European Commission (EC).

Lung cancer is a common malignancy worldwide, with the highest mortality rate globally. In China, lung cancer is also the leading type of cancer, with both incidence and mortality rates ranking first among malignant tumors. NSCLC accounts for approximately 85% of all lung cancer cases. Despite curative surgical resection options for early- and mid-stage NSCLC, more than half of patients experience local recurrence or distant metastasis within five years after surgery. Previous perioperative chemotherapy has offered limited survival improvement for surgical patients, with the five-year survival rate increasing by only 5%.