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

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< 0.001) and 9.6 months for tislelizumab in combination with nab-paclitaxel and carboplatin (HR: 0.43 [95% CI: 0.308-0.60]; P< 0.001) versus 5.5 months for paclitaxel and carboplatin alone, at a median study follow-up of 8.6 months. The most common grade ≥3 treatment emergent adverse events were decreased neutrophil levels, neutropenia and leukopenia.

About RATIONALE 304

RATIONALE 304 (NCT03663205) is an open-label, randomized Phase 3 trial that enrolled 334 patients with locally advanced or metastatic non-squamous NSCLC. The study met its primary endpoint, with first-line tislelizumab in combination with chemotherapy resulting in statistically significant improvement in PFS compared to chemotherapy (HR: 0.65 [95% CI: 0.47-0.91]; P=0.0054) along with higher response rates and longer response duration. The median PFS in the overall and in the PD-L1≥50% populations was 9.7 months for tislelizumab in combination with platinum (carboplatin or cisplatin) and pemetrexed versus 7.6 months for platinum and pemetrexed alone and 14.6 months with tislelizumab in combination with chemotherapy vs. 4.6 months with chemotherapy alone (stratified HR: 0.31 [95% CI: 0.178-0.547]) respectively, at a median study follow-up of 9.8 months. The most common grade ≥3 treatment emergent adverse events were associated with chemotherapy and included neutropenia and leukopenia.

About RATIONALE 303

RATIONALE 303 (NCT03358875) is an open-label, randomized Phase 3 trial with tislelizumab versus docetaxel that enrolled 805 patients with advanced NSCLC who progressed on prior platinum-based chemotherapy. The study met its primary endpoint, with second- or third-line tislelizumab resulting in statistically significant and clinically meaningful improvement in overall survival (OS) compared with docetaxel in the intent-to-treat population (HR: 0.66 [95% CI: 0.56-0.79]; P<0.0001), regardless of PD-L1 expression. The median OS was 16.9 months for tislelizumab versus 11.9 months for docetaxel. At the final analysis, OS in the PD-L1 positive population was also significantly improved in favor of tislelizumab (median 19.3 versus 11.5 months, respectively; HR: 0.53 [95% CI: 0.41-0.70]; P<0.0001). The most commonly reported grade ≥3 treatment emergent adverse events were pneumonia, anemia and dyspnea.

About NSCLC

Lung cancer is the second most common type of cancer and the leading cause of cancer-related death worldwide.¹ Lung cancer is the third most common cancer in Europe; NSCLC represents 85–90% of all lung cancers.² In 2020, the number of new cases of lung cancer diagnosed in Europe was estimated at 477,534.³

About Tislelizumab

Tislelizumab 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.

About BeiGene

BeiGene 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 10,000 colleagues spans five continents, with administrative offices in Basel, Beijing, and Cambridge, U.S. To learn more about BeiGene, please visit www.beigene.com and follow us on LinkedIn and X (formerly known as Twitter).

Forward-Looking Statements

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 effectiveness of tislelizumab as a treatment for patients with treatment-naïve and treatment-resistant NSCLC; the future advancement of tislelizumab as a therapy to address unmet needs of patients across the world; the ability of tislelizumab to consistently deliver clinically meaningful improvements in survival and quality of life for cancer patients; 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.

To access BeiGene media resources, please visit our News & Media site.

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¹ Globocan 2020. 900-world-fact-sheets.pdf (iarc.fr).

² European Society of Medical Oncology. What is Non-Small-Cell Lung Cancer? https://www.esmo.org/content/download/7252/143219/file/en-non-small-cell-lung-cancer-guide-for-patients.pdf.

³ Sung H, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2021;71(3):209-49.

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