Positive opinion for first-line treatment of extensive-stage small cell lung cancer based on results of RATIONALE-312 study demonstrating statistically significant overall survival benefit for TEVIMBRA in combination with chemotherapy

BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that intends to 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 etoposide and platinum chemotherapy, as a first-line treatment for adult patients with extensive-stage small cell lung cancer (ES-SCLC).

“The aggressive nature of extensive-stage small cell lung cancer makes it an extremely difficult type of lung cancer to treat, and currently available treatments may not adequately control disease progression,” said Prof. Silvia Novello, M.D., Ph.D., President Women Against Lung Cancer in Europe (WALCE) and Head of Medical Oncology Unit of San Luigi Hospital in Orbassano, Italy. “The compelling data from the RATIONALE-312 study demonstrates the potential of TEVIMBRA plus chemotherapy as a further first-line treatment option to extend overall survival for patients with ES-SCLC.”

The extension of indication for ES-SCLC is based on results from BeiGene’s RATIONALE-312 (NCT04005716), a randomized, double-blind, placebo-controlled, multicenter, Phase 3 study to evaluate the efficacy and safety of TEVIMBRA, in combination with platinum (investigator’s choice of cisplatin or carboplatin) plus etoposide, as first-line treatment in adult patients with ES-SCLC. The study, which randomized 457 patients, met its primary endpoint, exhibiting a statistically significant and clinically meaningful improvement in overall survival (OS) with TEVIMBRA in combination with chemotherapy, compared with placebo plus chemotherapy in the intent-to-treat (ITT) population. As reported in the Journal of Thoracic Oncology, at the protocol-defined final analysis, the median OS was 15.5 months for TEVIMBRA with chemotherapy versus 13.5 months for placebo plus chemotherapy (HR 0.75 [95% CI: 0.61–0.93]; one-sided p = 0.0040), resulting in a 25% reduction in the risk of death. TEVIMBRA plus chemotherapy was generally well tolerated, with no new safety signals identified.

“Today's positive CHMP opinion marks another important step for TEVIMBRA to potentially expand its indications in a fourth disease area in Europe to reach more patients affected by cancer,” said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. “TEVIMBRA is the cornerstone of our solid tumor portfolio with 58 regulatory approvals in 18 months and is being studied in combination with multiple novel molecules with the potential to herald the next wave of cancer therapeutics.”

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 unresectable esophageal squamous cell carcinoma (ESCC) and gastric or gastroesophageal junction (G/GEJ) adenocarcinoma in combination with chemotherapy, as a second line treatment in unresectable, locally advanced or metastatic ESCC after prior platinum-based chemotherapy, and for three non-small lung cancer (NSCLC) indications covering both the first- and second-line settings.

The Company recently announced its intent to change its name to BeOne Medicines, reaffirming its commitment to develop innovative medicines to eliminate cancer by partnering with the global community to serve as many patients as possible.

About Extensive-Stage Small Cell Lung Cancer (ES-SCLC)

Lung cancer is the leading cause of cancer-related deaths worldwide.¹ SCLC is an aggressive, high-grade cancer that accounts for 15% of all lung cancers,² and is typically classified as limited-stage or extensive-stage disease.³ Approximately 70% of SCLC patients are diagnosed with extensive-stage disease,⁴ defined as cancer that has spread throughout or beyond the lungs, or exceeding an area that can be treated with radiation alone.⁵ In Europe, the estimated prevalence of SCLC is 1-5 per 10,000 people.⁶ ES-SCLC is associated with a very poor prognosis with a median OS of 8 to 13 months and an expected 2-year survival rate of only 5%.⁷

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 45 countries, and more than 1.3 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 plans to 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 extend overall survival for patients with ES-SCLC; the ability of TEVIMBRA to further expand its indications and reach more patients affected by cancer; TEVIMBRA’s ability to herald the next wave of cancer therapeutics; 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 annual report on Form 10-K, 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. To access BeiGene media resources, please visit our News & Media site.

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