European Commission Approves TEVIMBRA® in Combination with Chemotherapy as a First-Line Treatment for Nasopharyngeal Carcinoma

New indication based on results of RATIONALE-309 study demonstrating statistically significant improvement in progression-free survivalSecond approval for TEVIMBRA in the European Union this year

BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the European Commission has approved TEVIMBRA^® (tislelizumab), in combination with gemcitabine and cisplatin, for the first-line treatment of adult patients with metastatic or recurrent nasopharyngeal carcinoma (NPC), not amenable to curative surgery or radiotherapy. Nasopharyngeal carcinoma is a rare cancer in which malignant cells form in the nasopharynx, the upper part of the throat located behind the nose.¹

“The approval of TEVIMBRA combined with chemotherapy in Europe marks an important advancement for people with recurrent or metastatic nasopharyngeal carcinoma—a rare and challenging disease,” said Prof. Lisa Licitra, Chief of the Head and Neck Cancer Medical Oncology Department at Fondazione IRCCS Istituto Nazionale dei Tumori in Milan, Italy. “Thanks to the compelling results from the RATIONALE-309 study, we now have a powerful new treatment that not only delays disease progression but also helps patients live longer. This approval brings new hope and a clinically proven option to patients who urgently need better care.”

The approval in NPC is based on results of RATIONALE-309 (NCT03924986), a double-blind, placebo-controlled, multicenter, Phase 3 study, which randomized 263 treatment-naïve patients who received either TEVIMBRA in combination with gemcitabine plus cisplatin or placebo in combination with gemcitabine plus cisplatin. The primary endpoint was met at the first prespecified interim analysis in which TEVIMBRA significantly prolonged progression-free survival (PFS) in the intent-to-treat (ITT) population (HR 0.52 [95% CI:0.38, 0.73] p<0.0001), showing a 48% reduction in the risk of disease progression or death. The median PFS in the TEVIMBRA plus chemotherapy arm was 9.2 months compared to 7.4 months in the placebo plus chemotherapy arm.

An updated analysis with an additional 12 months of follow-up showed efficacy results consistent with the interim analysis. Clinically meaningful and sustained improvement in overall survival (OS) was observed, with a median OS of 45.3 months for TEVIMBRA plus chemotherapy compared to 31.8 months for placebo plus chemotherapy.

TEVIMBRA plus chemotherapy was generally well tolerated, and no new safety signals were identified. Pooled safety data included over 3,900 patients who received TEVIMBRA, either as 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 (≥ 10%) associated with TEVIMBRA given in combination with chemotherapy were neutropenia, anemia, and thrombocytopenia.

“Following our recent EU approval of TEVIMBRA for extensive-stage small cell lung cancer, this new authorization in nasopharyngeal carcinoma reflects strong momentum in broadening access to our immunotherapy across solid tumors,” said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeOne. “With a comprehensive EU label spanning lung and gastrointestinal cancers, and more than 100 regulatory approvals globally, we are delivering on our ambition to bring innovative therapies to more patients around the world.”

TEVIMBRA was previously 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), and for three non-small cell lung cancer (NSCLC) indications covering both the first- and second-line settings.

About Nasopharyngeal Cancer (NPC)

Nasopharyngeal cancer, also known as nasopharyngeal carcinoma, is a type of head and neck cancer that starts in the nasopharynx, the upper throat passage that connects the nose to the lungs.² NPC, which can be categorized into different pathological subtypes (keratinizing squamous, non-keratinizing, and basaloid squamous)³, is often diagnosed at advanced stages due to its deep anatomical location and mild early symptoms, making early detection challenging.⁴ In 2020, NPC accounted for approximately 133,000 new cancer cases and 80,000 deaths per year globally and exhibits a unique geographical pattern, with its prevalence notably concentrated in Asia.⁵ While the overall 5-year survival rate for NPC is approximately 63%, in advanced disease the survival rate decreases to 49%.⁶

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 BeOne’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 availability and approved indications vary by country. Please refer to local prescribing information for complete details.

About BeOne

BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more affordable and accessible to cancer patients worldwide. With a portfolio spanning hematology and solid tumors, BeOne is expediting development of its diverse pipeline of novel therapeutics through its internal capabilities and collaborations. With a growing global team of more than 11,000 colleagues spanning six continents, the Company is committed to radically improving access to medicines for far more patients who need them. To learn more about BeOne, please visit www.beonemedicines.com and follow us on LinkedIn, X, Facebook and Instagram.

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; BeOne’s ability to expand patient access to cancer treatments; and BeOne’s plans, commitments, aspirations, and goals under the heading “About BeOne.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeOne'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; BeOne's ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeOne's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeOne's reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeOne’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 BeOne’s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeOne'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 BeOne undertakes no duty to update such information unless required by law.

To access BeOne media resources, please visit ourNewsroom.

¹ National Cancer Institute at the National Institutes of Health. Nasopharyngeal Cancer Treatment (PDQ®)–Patient Version. https://www.cancer.gov/types/head-and-neck/patient/adult/nasopharyngeal-treatment-pdq
² Cleveland Clinic. Nasopharynx.https://my.clevelandclinic.org/health/body/22376-nasopharynx
³ American Cancer Society. What Is Nasopharyngeal Cancer?https://www.cancer.org/cancer/types/nasopharyngeal-cancer/about/what-is-nasopharyngeal-cancer.html
⁴ Wei, X., et al. (2024) Nasopharyngeal cancer risk assessment by country or region worldwide from 1990 to 2019. BMC Public Health, 24, 1931. https://doi.org/10.1186/s12889-024-19228-9
⁵ Yang, Y., et al. (2023) Tislelizumab plus chemotherapy as first-line treatment for recurrent or metastatic nasopharyngeal cancer: A multicenter phase 3 trial (RATIONALE-309). Cancer Cell, 41(7): 1061–1072.
⁶ American Cancer Society. Survival Rates for Nasopharyngeal Cancer. https://www.cancer.org/cancer/types/nasopharyngeal-cancer/detection-diagnosis-staging/survival-rates.html

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