Recommendation based on results of three Phase 3 clinical trials demonstrating benefit of tislelizumab as a first- and second-line treatment for patients with NSCLC

BASEL, Switzerland & BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending approval of tislelizumab as a treatment for non-small cell lung cancer (NSCLC) across three indications:

• In combination with carboplatin and either paclitaxel or nab-paclitaxel for the first-line treatment of adult patients with squamous NSCLC who have locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
• In combination with pemetrexed and platinum-containing chemotherapy for the first-line treatment of adult patients with non-squamous NSCLC whose tumors have PD-L1 expression on ≥50% of tumor cells with no EGFR or ALK positive mutations and who have locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
• As monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy. Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab.

“Through three Phase 3 clinical trials enrolling nearly 1,500 patients across the world including in the European Union, tislelizumab has been shown to be an effective therapy for patients with treatment-naïve and treatment-resistant NSCLC,” said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. “Today's positive CHMP opinion brings us one step closer to providing an important treatment option to patients in Europe with lung cancer, which is among the most common cancers and a leading cause of cancer death in the region.”

The Marketing Authorization Application (MAA) for NSCLC is based on results from three Phase 3 studies that enrolled 1,499 patients. First-line combination therapy results from RATIONALE 307 evaluating tislelizumab in advanced squamous NSCLC and from RATIONALE 304 evaluating tislelizumab in locally advanced or metastatic non-squamous NSCLC were published in JAMA Oncology and in the Journal of Thoracic Oncology, respectively. Second-line monotherapy results from RATIONALE 303 evaluating tislelizumab in previously treated advanced NSCLC were published in the Journal of Thoracic Oncology.

Dr. Lanasa added, “As we strengthen our global portfolio in solid tumors, this positive CHMP opinion marks another significant milestone in the European Union for tislelizumab only a few months after it was approved for the treatment of advanced esophageal squamous cell carcinoma. We will continue to follow the science and data to advance tislelizumab as a monotherapy and combination treatment to address unmet needs of patients across the world.”

Tislelizumab, under the brand name TEVIMBRA®, received approval from the European Commission for advanced or metastatic ESCC after prior chemotherapy in 2023 and is currently under review with the U.S. Food and Drug Administration. Tislelizumab is also under review by the FDA as a first-line treatment for patients with unresectable, recurrent, locally advanced, or metastatic ESCC. BeiGene has launched more than 17 potentially registration-enabling trials with tislelizumab with over 13,000 patients enrolled to-date, of which 15 have already reported positive readouts. In these clinical studies, tislelizumab has consistently demonstrated its ability deliver clinically meaningful improvements in survival and quality of life with a positive benefit-risk balance for cancer patients across a range of tumor types – in many cases, regardless of PD-(L)1 status – both as monotherapy and in combination with other regimens. More than 900,000 patients have been prescribed tislelizumab to date.

About RATIONALE 307 RATIONALE 307 (NCT03594747) is an open-label, randomized Phase 3 trial that enrolled 360 patients with advanced squamous NSCLC. The study met its primary endpoint with first-line tislelizumab in combination with chemotherapy resulting in statistically significant improvement in progression free survival (PFS), as well as higher objective response rates (ORRs) and a manageable safety/tolerability profile, regardless of PD-L1 expression. The median PFS was 7.7 months for tislelizumab in combination with paclitaxel and carboplatin (hazard ratio, HR: 0.45 [95% CI: 0.326-0.619]; P