BeOne Presents Compelling Data in Hard-to-Treat Liver and Lung Cancers at ESMO 2026

Potential first-in-class GPC3 x 4-1BB bispecific achieves a 36% confirmed response rate at Phase 3 dose and favorable safety profile in heavily pretreated liver cancer

Brain-penetrant PRMT5 inhibitor shows promising antitumor and CNS activity in the largest publicly shared MTAP-deficient dataset

First clinical data of CEA-targeting ADC demonstrates encouraging and sustained responses in CEA^high NSCLC

SAN CARLOS, Calif.--(BUSINESS WIRE)-- BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced new data from multiple differentiated investigational medicines across its priority solid tumor disease areas at the European Society for Medical Oncology (ESMO) Congress 2026 (October 23–27, Madrid). The presentations span gastrointestinal, lung, and breast cancers, and reflect the growing momentum of BeOne’s solid tumor franchise.

Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeOne, said:

“At ESMO, we are showing how our strategy of matching the right modality to disease biology is translating into meaningful clinical progress. Our GPC3 x 4-1BB bispecific antibody continues to be well tolerated and demonstrates durable activity with longer follow up in patients with heavily pre-treated hepatocellular carcinoma, while the first clinical data from our brain-penetrant PRMT5 inhibitor and CEA-targeting ADC expand our innovation in non-small cell lung cancer. Alongside the global Phase 3 KANDELA-302 study of our CDK4 inhibitor in breast cancer, these advances mark a clear step forward as BeOne’s solid tumor pipeline moves from promising science toward medicines with the potential to improve outcomes in difficult-to-treat cancers.”

Potential first-in-class GPC3×4-1BB bispecific continues to demonstrate compelling and durable activity in heavily pretreated HCC (Proffered Paper: 1679O; October 24, 10:15–11:45 CET)

Patients with advanced HCC who progress after immunotherapy face a high unmet need, with no established standard of care and five-year survival of only approximately 20%^1,2. BGB-B2033 is an investigational bispecific antibody designed to address this Gap by activating 4-1BB on T cells in the presence of GPC3-expressing tumor cells, concentrating immune activation at the tumor site, and limiting systemic toxicity, a key consideration for HCC patients who frequently suffer from underlying liver disease.

Updated Phase 1 results include the first disclosure of dose-optimization data and longer follow-up from dose escalation. Among 94 patients, 93.6% had received prior PD-(L)1 therapy and 61.7% had received both PD-(L)1 therapy and a tyrosine kinase inhibitor (TKI). Highlights include:

• At the recommended Phase 3 dose of 450 mg every three weeks, the confirmed objective response rate (ORR) was 36.0%, a result that exceeds efficacy observed with TKIs, which have shown an ORR of approximately 5% in late-line HCC.³
• At the selected Phase 3 dose, the estimated six-month confirmed duration-of-response rate was 74.1%, with responses observed across all clinically relevant patient subgroups.
• Across dose levels, Grade 3 or higher treatment-related adverse events occurred in 5.3% of patients, and treatment-related adverse events led to discontinuation in only 2.1%.

BGB-B2033 received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in December 2025, as well as Orphan Drug Designation from the FDA in March 2026 and the European Commission in October 2026 for hepatocellular carcinoma (HCC). BeOne plans to initiate global registrational clinical trials in late-line HCC by the end of 2026, while also developing plans for registrational studies in earlier treatment settings.

Initial first-in-human data from two differentiated assets advance BeOne’s emerging NSCLC portfolio

At ESMO 2026, BeOne is presenting initial clinical data in NSCLC from two biomarker-led programs with distinct mechanisms: the oral, brain-penetrant PRMT5 inhibitor, BGB-58067, and the CEA-targeting antibody-drug conjugate (ADC), BG-C477. Based on these promising data, both programs are intended to advance toward late-stage development in 2027, continuing to build BeOne’s footprint in lung cancer alongside its established program with TEVIMBRA (tislelizumab).

Brain-penetrant PRMT5 inhibitor, BGB-58067, demonstrates systemic and intracranial activity in MTAP-deficient NSCLC (Poster: 1043P; October 23, 3:15–4:00 PM CET)

BGB-58067 is a highly potent, brain-penetrant methylthioadenosine (MTA)-cooperative PRMT5 inhibitor designed to exploit the vulnerability created by MTAP deficiency. In this, the largest publicly shared data set in MTAP-deficient solid tumors, initial Phase 1 findings from 182 patients, including 83 with NSCLC, showed:

• Among 75 efficacy-evaluable patients with MTAP-deficient NSCLC, confirmed ORR was 25.3% and disease control rate (DCR) was 76.0%. In the EGFR wild-type subgroup, confirmed ORR was 38.5% and DCR was 82.1%.
• Median progression-free survival was 7.7 months in the overall NSCLC cohort and 8.1 months in the EGFR wild-type subgroup.
• In the NSCLC cohort, an intracranial objective response rate of 30.8% was observed in patients with untreated, asymptomatic brain metastases as well as a low rate of central nervous system (CNS) progression in 2L+ NSCLC.
• At daily doses of 200–400 mg (n=87), Grade 3 or higher treatment-related adverse events occurred in 27.6%, and treatment-related adverse events led to discontinuation in 5.7%. The most common treatment-related events were nausea, anemia and fatigue.

CEA-targeting ADC BG-C477 achieves encouraging response rate in previously-treated CEA^high NSCLC (Poster: 2567P; October 26, 12:00–12:45 PM CET)

BG-C477 is the first ADC to emerge from BeOne’s internal drug discovery organization. With a proprietary CEA (or CEACAM5) targeting antibody, a highly stable conjugator, and a cleavable linker, BG-C477 is designed to deliver a potent topoisomerase I inhibitor payload directly to CEA-expressing tumor cells.

As of April 9, 2026, preliminary Phase 1 monotherapy findings from 43 heavily pretreated patients with NSCLC, including 25 with high CEA expression, showed:

• Confirmed ORR was 56.0% in patients with CEA^high NSCLC, with a disease control rate of 88.0%; confirmed ORR in the overall NSCLC population was 34.9%.
• Among patients with CEA^high NSCLC, median duration of response had not been reached. The majority of responders, as well as two patients with unconfirmed responses, remained on treatment at data cutoff.
• Grade 3 or higher treatment-emergent adverse events (TEAEs) were predominantly hematologic events (anemia 14.0%, neutropenia 14.0%, thrombocytopenia 2.3%).

Updated results, with a more recent data cut-off, will be presented in the Locally Advanced NSCLC poster session on Monday, October 26 at 12:00 CET. BG-C477 received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in October 2026.

Pivotal-study update: Phase 3 KANDELA-302 trial of CDK4 inhibitor BGB-43395 now enrolling in first-line HR+/HER2-negative breast cancer (1885TiP; October 24, 12:00–12:45 PM CEST)

Building on previously reported Phase 1 activity and tolerability findings, BeOne is advancing BGB-43395, an investigational selective CDK4 inhibitor, in the global Phase 3 KANDELA-302 study (NCT07492641). The study compares BGB-43395 plus letrozole with investigator’s choice of abemaciclib, palbociclib or ribociclib plus letrozole in first-line HR+/HER2-negative advanced or metastatic breast cancer.

Enrollment began in May 2026, with patients planned across North and South America, Europe, Asia and Australia. The primary endpoint is progression-free survival by blinded independent central review, with overall survival as the key secondary endpoint.

About BGB-B2033
BGB-B2033 is a bispecific antibody targeting GPC3 (glypican 3), a tumor-specific antigen highly expressed in HCC⁴, and 4-1BB, a co-stimulatory receptor associated with T-cell activation and tumor reactivity in HCC.⁵ The molecule has been designed with reduced antibody-dependent cellular cytotoxicity (ADCC) to prevent systemic toxicity.

About BGB-58067
BGB-58067 is an investigational, oral small molecule designed to inhibit protein arginine methyltransferase 5 (PRMT5) in an MTA-dependent manner, enabling selective targeting of MTAP-deleted cancer cells. Preclinical data demonstrate enhanced selectivity compared with MTAP wild-type cells, as well as strong potency and brain penetration.

About BG-C477
BG-C477 is an investigational ADC designed to Target CEA, also known as CEACAM5, a protein highly expressed in several epithelial cancers, including certain lung and gastrointestinal cancers. The molecule is designed to work as a targeted delivery system, using an antibody that specifically recognizes CEA to deliver a potent anti-cancer payload to CEA-expressing tumor cells. BG-C477 incorporates a highly stable conjugator and cleavable linker, aiming for a strong bystander effect and manageable toxicity.

About BGB-43395
BGB-43395 is an investigational cyclin-dependent kinase (CDK) 4 inhibitor being studied in a global clinical development program that includes the KANDELA-302 (NCT07492641) Phase 3 clinical trial, in hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) breast cancer. BGB-43395 is a highly potent and selective CDK4 inhibitor that may reduce dose-limiting hematologic toxicities associated with the current CDK4/6 standard of care and improve tolerability, thereby enabling deeper CDK4 inhibition.

About BeOne
BeOne Medicines is a global oncology company that is discovering and developing innovative treatments for 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. The Company has a growing global Team spanning six continents who are driven by scientific excellence and exceptional speed to reach more patients than ever before. 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, BGB-58067, BG-C477 and BGB-43395 to improve outcomes for patients; BeOne’s plans to initiate global registrational clinical trials of BGB-B2033 in late-line HCC by the end of 2026 and to develop registrational studies in earlier treatment settings; the expected advancement of BGB-58067 and BG-C477 toward late-stage development in 2027; the conduct and enrollment of the KANDELA-302 study; 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.

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Source: BeOne Medicines Ltd.