BeOne Medicines Granted U.S. FDA Fast Track Designation for BGB-B2033 as Treatment for Hepatocellular Carcinoma

Key Terms

bispecific antibody medical

A bispecific antibody is a specially designed protein that can attach to two different targets at the same time. Think of it as a custom-made connector that brings two things together—such as a disease cell and an immune system component—helping the body fight illnesses more effectively. For investors, understanding bispecific antibodies is important because they represent innovative therapies that could lead to new treatments and potentially lucrative market opportunities.

fast track designation regulatory

A "fast track designation" is a process that speeds up the review and approval of a product or project, allowing it to reach the market or be completed more quickly than usual. For investors, it can signal that a product may become available sooner, potentially leading to earlier revenue or benefits, and indicating a priority status that might influence company performance and market opportunities.

hepatocellular carcinoma medical

Hepatocellular carcinoma is the most common form of primary liver cancer, arising from the main functional cells of the liver. For investors it matters because its serious health impact drives demand for diagnostic tests, treatments and long-term care; progress in trials or approvals can change a drugmaker’s revenue outlook much like a successful product launch can reshape a company’s future.

phase 1 medical

Phase 1 is the first stage of testing a new drug or medical treatment in people, focused primarily on safety, how the body handles the product, and finding a tolerated dose. Think of it as a short, tightly controlled experiment with a small group to check for dangerous side effects before wider testing; for investors it is an early milestone that reduces some uncertainty but still carries high risk and potential for both big value changes and setbacks.

pd-1 inhibitor medical

A PD-1 inhibitor is a type of medicine that helps the immune system recognize and attack cancer cells by blocking the PD-1 checkpoint—a molecular “off switch” that cancer can use to hide from immune defenses. For investors, these drugs matter because they can transform treatment options, drive sales or partnerships, and affect a company’s valuation if clinical trials, approvals, or competitive positioning change; think of them as unlocking a locked door to let the body’s defenses work.

BGB-B2033 is a bispecific antibody directed at GPC3 and 4-1BB; key targets in the most common liver cancer

FDA Fast Track Designation reflects the potential of BGB-B2033 in an area of high unmet need

SAN CARLOS, Calif.--(BUSINESS WIRE)-- BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted the Company Fast Track Designation for BGB-B2033, its GPC3x4-1BB bispecific antibody for the treatment of adult patients with hepatocellular carcinoma (HCC) with disease progression on or after prior systemic treatment.

“The FDA awards Fast Track Designation to therapies that show potential to address an unmet medical need in serious or life-threatening conditions. The FDA’s decision reflects the encouraging profile of BGB-B2033 in advanced hepatocellular carcinoma, where patients continue to face limited treatment options,” said Julie Lepin, Senior Vice President and Chief Regulatory Affairs Officer at BeOne.

BeOne is currently conducting a global, multi-center Phase 1 clinical trial (NCT06427941) to explore the safety and anti-tumor activity of BGB-B2033, both alone and in combination with PD-1 inhibitor TEVIMBRA^® (tislelizumab).

About Hepatocellular Carcinoma

Hepatocellular Carcinoma (HCC) is the sixth most common cancer worldwide and the fourth leading cause of cancer-related death.¹ HCC accounts for 80% of all primary liver cancers, with the number of new cases expected to double between 2022 and 2050.² The rising burden of HCC is primarily attributed to the high prevalence of the hepatitis B and hepatitis C viruses (HBV/HBC) and lifestyle factors such as obesity, tobacco, and alcohol consumption.³ With approximately 80% of patients diagnosed in advanced stages and five-year survival rates for this patient population lower than 20%, new treatment options are needed beyond currently available systemic therapy.⁴

About BGB-B2033

BGB-B2033 is a bispecific antibody targeting GPC3 (glypican 3), a tumor-specific antigen highly expressed in HCC⁵, and 4-1BB, a co-stimulatory receptor associated with T-cell activation and tumor reactivity in HCC.⁶ The molecule has been designed with reduced antibody-dependent cellular cytotoxicity (ADCC) to prevent systemic toxicity.

About BeOne Medicines

BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more 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 nearly 12,000 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 potential of BGB-B2033 to address an unmet medical need in HCC; BeOne’s clinical development plan for BGB-B2033; and BeOne’s plans, commitments, aspirations, and goals under the heading “About BeOne Medicines.” 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 our Newsroom site.

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1 Samant H, Amiri HS, Zibari GB. Addressing the worldwide hepatocellular carcinoma: epidemiology, prevention and management. J Gastrointest Oncol. 2021 Jul;12(Suppl 2):S361-S373. doi: 10.21037/jgo.2020.02.08. PMID: 34422400; PMCID: PMC8343080.

2 Chan, Stephen Lam et al The Lancet Commission on addressing the global hepatocellular carcinoma burden: comprehensive strategies from prevention to treatment. . The Lancet, Volume 406, Issue 10504, 731 - 778

3 Kim DY. Changing etiology and epidemiology of hepatocellular carcinoma: Asia and worldwide. Journal of Liver Cancer 2024;24(1):62-70. DOI: https://doi.org/10.17998/jlc.2024.03.13

4 Li D, Sedano S, Allen R, Gong J, Cho M, Sharma S. Current Treatment Landscape for Advanced Hepatocellular Carcinoma: Patient Outcomes and the Impact on Quality of Life. Cancers (Basel). 2019 Jun 18;11(6):841. doi: 10.3390/cancers11060841. PMID: 31216701; PMCID: PMC6627588.

5 Fanching Lin et al. Novel GPC3-targeting radiopharmaceutical therapy for hepatocellular carcinoma. J Clin Oncol 42, 525-525(2024).DOI:10.1200/JCO.2024.42.3_suppl.525

6 Kim DY et al. 4-1BB Delineates Distinct Activation Status of Exhausted Tumor-Infiltrating CD8+ T Cells in Hepatocellular Carcinoma. Hepatology, VOL. 71, NO. 3, 2020. https://onlinelibrary.wiley.com/doi/pdf/10.1002/hep.30881

 

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Source: BeOne Medicines Ltd.