Decision highlights the promise of BGB-16673, an investigational and potentially first-in-class BTK degrader designed to overcome resistance and deepen responses in B-cell malignancies

BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) designation to the Company’s investigational Bruton’s tyrosine kinase (BTK) degrader, BGB-16673, for the treatment of patients with Waldenstrom’s macroglobulinemia (WM) previously treated with a BTK inhibitor.

“This is the Company’s first PRIME designation, marking a milestone for BeOne and providing early and enhanced interaction with the EMA as we advance BGB-16673,” said Julie Lepin, Senior Vice President, Chief Regulatory Affairs Officer at BeOne. “PRIME allows us to align early with the EMA on key evidence requirements and potentially accelerate our path to marketing authorization of BGB-16673 for patients with relapsed or refractory Waldenstrom’s macroglobulinemia.”

In addition to the PRIME designation, the EMA’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on the EU Orphan Drug Designation (ODD) application for BGB-16673 in WM. A final decision is anticipated in the coming weeks. The U.S. Food and Drug Administration (FDA) also granted Fast Track Designation to BGB-16673 for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and adult patients with R/R mantle cell lymphoma (MCL).

The EMA’s CHMP granted PRIME designation to BGB-16673 based on data demonstrating its novel mechanism and anti-tumor activity in B-cell malignancies. The CHMP recognized the limited treatment options available for WM patients post-BTK inhibitor therapy and acknowledged the strong biological rationale and promising clinical data for BGB-16673 in this setting, thereby demonstrating the potential to address the unmet medical need.

The PRIME initiative, launched by the EMA in 2016, provides early, proactive, and enhanced regulatory support to developers of promising medicines. It is designed to optimize development plans and accelerate evaluation, helping innovative therapies reach patients with unmet medical needs faster.

About BGB-16673

BGB-16673 is an orally available Bruton’s tyrosine kinase (BTK) targeting protein degrader from BeOne’s chimeric degradation activation compound (CDAC) platform. BGB-16673 is designed to promote the degradation, or breakdown, of both wild-type and mutant forms of BTK, including those that commonly result in resistance to BTK inhibitors in patients who experience progressive disease. BGB-16673 is the most advanced BTK protein degrader in the clinic, with an extensive global clinical development program.

About BeOne Medicines

BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more affordable and 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 more than 11,000 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 BeOne’s advancement of, and anticipated clinical development, regulatory milestones and commercialization of BGB-16673; the potential of BGB-16673 to significantly address the unmet medical need; 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 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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