BeiGene Receives Positive CHMP Opinion for TEVIMBRA® as a First-Line Treatment for Nasopharyngeal Cancer

Positive opinion for TEVIMBRA in combination with chemotherapy as a first-line treatment of nasopharyngeal cancer (NPC) based on results of RATIONALE-309 study demonstrating statistically significant improvement in progression-free survival

BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that will change its name to BeOne Medicines Ltd., today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion recommending approval of TEVIMBRA^® (tislelizumab), in combination with gemcitabine and cisplatin, for the first-line treatment of adult patients with recurrent, not amenable to curative surgery or radiotherapy, or metastatic NPC.

“Chemotherapy has been the standard treatment for metastatic NPC; however, distant metastasis continues to be a significant cause of treatment failure and death, making new treatment options essential,” said Prof. Lisa Licitra, Chief of Head and Neck Cancer Medical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. “Results from the RATIONALE-309 study support the robust clinical benefit of TEVIMBRA plus chemotherapy, which has the potential to significantly reduce the risk of disease progression or death for patients with recurrent or metastatic NPC.”

The extension of indication for NPC is based on results from BeiGene’s RATIONALE-309 (NCT03924986), a randomized, double-blind, placebo-controlled, multicenter, Phase 3 study to evaluate the efficacy and safety of TEVIMBRA, in combination with gemcitabine plus cisplatin, as first-line treatment in adult patients with recurrent or metastatic NPC. At the time of the prespecified interim analysis, the study, which randomized 263 treatment-naïve patients, met its primary endpoint, significantly prolonging progression-free survival (PFS) in the intent-to-treat (ITT) population (HR 0.52 [95% CI:0.38, 0.73] p<0.0001). The median PFS in the TEVIMBRA with chemotherapy arm was 9.2 months compared to 7.4 months in the placebo with chemotherapy arm. An updated analysis showed consistent efficacy results with the interim analysis. The median overall survival (OS) was 45.3 months for TEVIMBRA plus chemotherapy versus 31.8 months for placebo plus chemotherapy. TEVIMBRA plus chemotherapy was generally well tolerated, with no new safety signals identified.

“Today’s announcement marks a second positive CHMP opinion for TEVIMBRA in 2025, signifying the potential to expand into yet another disease area in the European Union and to support even more patients living with cancer,” said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. “As the foundation of our solid tumor portfolio, we are encouraged by TEVIMBRA’s momentum in achieving more than 100 regulatory approvals for a range of cancer indications across the world, including major markets such as the US, the EU, China, and Japan, demonstrating the strength of evidence across a range of indications.”

The pooled safety data in this extension of indication included more than 3,900 patients who received TEVIMBRA as either monotherapy (n=1,952) or in combination with chemotherapy (n=1,950) at the approved dosing regimen. The most common Grade 3 or 4 adverse reactions (≥ 2%) for TEVIMBRA given in combination with chemotherapy were neutropenia, anemia, thrombocytopenia, hyponatremia, hypokalemia, fatigue, pneumonia, lymphopenia, rash, decreased appetite, increased aspartate aminotransferase, and increased alanine aminotransferase.

TEVIMBRA is currently approved in the EU as a first-line treatment for eligible patients with gastric or gastroesophageal junction (G/GEJ) adenocarcinoma, as a first-line treatment for unresectable esophageal squamous cell carcinoma (ESCC), as a second-line treatment in ESCC after prior platinum-based chemotherapy, as a first-line treatment for extensive-stage small cell lung cancer (ES-SCLC) in combination with chemotherapy, and for three non-small lung cancer (NSCLC) indications covering both the first- and second-line settings.

About Nasopharyngeal Cancer (NPC)

Nasopharyngeal cancer is a type of head and neck cancer that starts in the nasopharynx,¹ the upper throat passage that connects the nose to the lungs.² NPC is often diagnosed at advanced stages due to its deep anatomical location and mild early symptoms, making early detection challenging.³ Globally, NPC accounts for approximately 133,000 new cancer cases and 80,000 deaths per year and exhibits a unique geographical pattern, with its prevalence notably concentrated in Asia.⁴ While the overall 5-year survival rate for NPC is approximately 63%, in advanced disease the survival rate decreases to 49%.⁵

About TEVIMBRA (Tislelizumab)

TEVIMBRA is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1(PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors.

TEVIMBRA is the foundational asset of BeiGene’s solid tumor portfolio and has shown potential across multiple tumor types and disease settings. The global TEVIMBRA clinical development program includes almost 14,000 patients enrolled to date in 35 countries and regions across 70 trials, including 21 registration-enabling studies. TEVIMBRA is approved in 46 countries, and more than 1.5 million patients have been treated globally.

Important Safety Information

The current European Summary of Product Characteristics (SmPC) for TEVIMBRA is available from the European Medicines Agency.

This information is intended for a global audience. Product indications vary by region.

About BeiGene

BeiGene, which will change its name to BeOne Medicines Ltd., is a global oncology company that is discovering and developing innovative treatments that are more affordable and accessible to cancer patients worldwide. With a broad portfolio, we are expediting development of our diverse pipeline of novel therapeutics through our internal capabilities and collaborations. We are committed to radically improving access to medicines for far more patients who need them. Our growing global team of more than 11,000 colleagues spans six continents. To learn more about BeiGene, please visit www.beigene.com.

Forward-Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the ability of TEVIMBRA plus chemotherapy to significantly reduce the risk of disease progression or death for patients with recurrent or metastatic NPC; the ability of BeiGene to support even more patients living with cancer;; and BeiGene’s plans, commitments, aspirations, and goals under the heading “About BeiGene.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing, and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene's reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeiGene’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products and its ability to obtain additional funding for operations and to complete the development of its drug candidates and achieve and maintain profitability; and those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

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