US FDA approves Baiyueze® (Zebutinib) for the treatment of patients with Waldenstrom's macroglobulinemia

This is the second approval of Baiyueze® by the FDA and the third approval for Fahrenheit's macroglobulinemia globally.

This approval is based on the results of the Phase 3 ASPEN clinical trial of Baiyueze® vs.
Ibrutinib

Beijing time September 2, 2021, BeiGene (Nasdaq: BGNE; Hong Kong Stock Exchange: 06160) is a global biopharmaceutical company focusing on the development and commercialization of innovative drugs worldwide
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Dr.
Huang Weijuan, Chief Medical Officer of BeiGene Hematology, said: "We are very pleased that the FDA has approved the second indication of Baiyueze® in the United States today
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Dr.
Huang Weijuan continued to comment: “In less than two years, Baiyueze® has obtained 11 regulatory approvals worldwide, including 2 approvals in the United States.
This shows that Baiyueze® is gradually becoming a target A fruitful treatment option for B-cell malignancies and expanding its global footprint, it is expected to benefit more patients
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Dr.
Steven Treon, Director of the Fahrenheit Macroglobulinemia Research Bing Center and Professor of Medicine at Harvard Medical School at the Dana-Farber Cancer Institute in the United States, commented: A highly active inhibitor for the treatment of patients with Waldenstrom's macroglobulinemia, compared with the first-generation BTK inhibitor, it has shown better tolerance in a series of clinically important side effects
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Pete DeNardis, Chairman of the International Fahrenheit Macroglobulinemia Foundation (IWMF), said: "Baiyueze® was approved for the treatment of Fahrenheit macroglobulinemia, bringing the second approval for this rare type of lymphoma.
The treatment of drugs is a boon for patients, which means that patients with Fahrenheit macroglobulinemia have more treatment options.
In particular, drugs such as Baiyueze® can be administered orally and treated with a single agent.
It can improve the treatment experience of patients
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The FDA's approval of Baiyueze® in WM is mainly based on the effectiveness of the multi-center, open phase 3 ASPEN trial (NCT03053440) comparing Baiyueze® and Ibrutinib in the treatment of WM patients
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The primary efficacy endpoint of the ASPEN trial is the very good partial response (VGPR) rate assessed by the Independent Review Committee (IRC) in the overall intention-to-treat (ITT) population
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In the FDA-approved Baiyueze® label, the main effectiveness result is reflected in the partial remission (PR) or better remission rate assessed by the IRC
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The most common adverse reactions of Baiyueze® (found in ≥20% of patients) are based on the safety data of 779 patients, including neutropenia, upper respiratory tract infection, thrombocytopenia, rash, bleeding, musculoskeletal pain, hemoglobin Decrease, bruising, diarrhea, infectious pneumonia and cough
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The recommended dosage of Baiyueze® is 160 mg twice daily or 320 mg once daily, either on an empty stomach or after meals
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About Baiyueze®

BRUKINSA® (BRUKINSA®, Zebutinib) is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) independently developed by BeiGene scientists.
It is currently undergoing extensive clinical trials worldwide.
As a single agent and combined with other therapies to treat a variety of B-cell malignancies
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Since the new BTK will be continuously synthesized in the human body, Baiyueze® is designed to achieve complete and continuous inhibition of BTK protein by optimizing bioavailability, half-life and selectivity
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With its differentiated pharmacokinetics from other approved BTK inhibitors, Baiyueze® can inhibit the proliferation of malignant B cells in multiple disease-related tissues
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Baiyueze® has been approved for the following indications in the following regions:

In November 2019, Baiyueze® was approved in the United States for the treatment of adult mantle cell lymphoma (MCL) patients who have received at least one treatment in the past*

In June 2020, Baiyueze® was approved in China for the treatment of adult mantle cell lymphoma (MCL) patients who have received at least one treatment in the past**

In June 2020, Baiyueze® was approved in China for the treatment of adult patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) who have received at least one treatment in the past**

In February 2021, Baiyueze® was approved in the United Arab Emirates for the treatment of patients with relapsed or refractory MCL

In March 2021, Baiyueze® was approved in Canada for the treatment of adult WM patients

In April 2021, Baiyueze® was registered in Israel and included in the local medical insurance reimbursement list for the treatment of adult MCL patients who have received at least one treatment in the past*

In June 2021, Baiyueze® was approved in China for the treatment of adult WM patients who have received at least one treatment in the past**

In July 2021, Baiyueze® was approved in Canada for the treatment of adult MCL patients who had received at least one treatment in the past**

In August 2021, Baiyueze® was approved in Brazil for the treatment of adult MCL patients who have received at least one treatment in the past

In August 2021, Baiyueze® was approved in the United States for the treatment of adult WM patients

So far, more than 30 marketing applications for multiple indications have been submitted, covering the United States, China, the European Union and more than 20 other countries or regions
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*This indication is based on the accelerated approval of the overall response rate (ORR)
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Subsequent approval for this indication will depend on the verification and description of clinical benefits in confirmatory trials
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**This indication was approved with conditions
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Full approval for this indication will depend on the results of ongoing confirmatory randomized, controlled clinical trials
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About BeiGene Oncology

BeiGene continues to promote the research and development of best-in-class or first-in-class clinical drug candidates through independent research and development or with like-minded partners, and is committed to providing influential, accessible and affordable drugs to patients around the world
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The company's global clinical research and development team has reached approximately 2,300 people, and the size of the team is still expanding
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The team is currently supporting more than 90 clinical studies worldwide and has recruited more than 13,000 patients and healthy subjects
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BeiGene's own clinical development team plans and leads the development and expansion of the company's product pipeline, providing support and guidance for clinical trials covering more than 40 countries/regions around the world
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The company pays special attention to the targeted therapy of hematological tumors and solid tumors and tumor immunotherapy, and focuses on single-drug and combination therapies
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At present, three drugs independently researched and developed by BeiGene have been approved for marketing: Baiyueze® (BTK inhibitor, which has been approved for marketing in the United States, China, Canada and other international markets), Baizean® (which can effectively avoid Fc -γ receptor-binding anti-PD-1 antibody, has been approved for marketing in China) and Baihuize® (PARP inhibitor, has been approved for marketing in China)
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At the same time, BeiGene is also cooperating with other innovative companies to jointly promote the research and development of innovative therapies to meet global health needs
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In China, BeiGene is selling a variety of oncology drugs authorized by Amgen and Bristol-Myers Squibb
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The company has also cooperated with many companies including Amgen, Biotech, EUSA Pharma, Mirati Therapeutics, Seagen and Zymeworks to meet the current global unmet medical needs to a greater extent
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BeiGene has also reached a cooperation with Novartis, authorizing Novartis to develop, produce and commercialize Bezeran® in North America, Europe and Japan
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About BeiGene

BeiGene is a science-based global biopharmaceutical company that focuses on the development of innovative and affordable drugs to improve treatment outcomes and access to drugs for patients around the world
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The company's broad drug portfolio currently includes more than 40 clinical drug candidates
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The company accelerates the development of diversified and innovative drug pipelines by strengthening independent research and development capabilities and cooperation
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We are committed to comprehensively improving access to medicines for more than 2 billion people around the world by 2030
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BeiGene has built a team of more than 7,000 people on five continents around the world
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For more information, please visit
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Forward-looking statement

This press release contains forward-looking statements as defined under the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including regarding the progress of BeiGene’s plan, BeiGene’s comments on BeiGene’s The anticipated clinical development, pharmaceutical milestones and commercialization progress of Zee®, the potential of BaiGene to provide patients with better clinical benefits and the mention under the sub-headings of "BeiGene Oncology" and "About BeiGene" BeiGene plans, commitments, ambitions and goals
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Due to various important factors, actual results may differ materially from forward-looking statements
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These factors include: BeiGene’s ability to prove the efficacy and safety of its drug candidates; the clinical results of the drug candidates may not support further development or marketing approval; the actions of the drug regulatory authority may affect the initiation, schedule and progress of clinical trials And drug marketing approval; BeiGene's marketed drugs and drug candidates (if approved) have the ability to achieve commercial success; BeiGene's ability to obtain and maintain the intellectual property protection of its drugs and technologies; BeiGene's ability to rely on the first The three parties’ drug development, production and other services; BeiGene’s limited experience in obtaining regulatory approval and commercialization of pharmaceutical products, and its ability to obtain further working capital to complete the development of drug candidates and realize and maintain profitability; COVID-19 Global The impact of the pandemic on BeiGene's clinical development, supervision, commercial operations and other businesses; the various risks discussed more fully in the "Risk Factors" section of Form 10-Q in the most recent quarterly report of BeiGene; and The discussion of potential risks, uncertainties and other important factors in BeiGene’s subsequent submission to the US Securities and Exchange Commission
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All information in this press release is only as of the date of the press release, and BeiGene has no responsibility to update such information unless required by law
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