U.S. FDA Grants Priority Review to Sonrotoclax for the Treatment of Relapsed or Refractory Mantle Cell Lymphoma

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 accepted and granted Priority Review to a New Drug Application (NDA) for sonrotoclax, a next-generation BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), following treatment with a Bruton’s tyrosine kinase (BTK) inhibitor.

“Sonrotoclax is advancing with remarkable speed, from Breakthrough Therapy Designation to Priority Review, all within a short window,” said Lai Wang, Ph.D., Global Head of R&D at BeOne. “That pace reflects both the strength of the data and the urgency of the need for patients with R/R MCL. With rapid, deep, and durable responses and a manageable safety profile, sonrotoclax is emerging as a potential best-in-class BCL2 inhibitor, alongside our two other transformative hematology assets – BTK inhibitor BRUKINSA, and investigational BTK degrader BGB-16673.”

The NDA is supported by data from the global, multicenter, single-arm, open-label, Phase 1/2 study, BGB-11417-201 (NCT05471843), which enrolled 125 adult patients with R/R MCL who received prior treatment with a BTK inhibitor. Sonrotoclax achieved its primary endpoint of overall response rate (ORR) as assessed by an independent review committee (IRC), demonstrating clinically meaningful responses in this heavily pretreated population. The study also showed promising results across several secondary efficacy endpoints, including complete response (CR) rate, duration of response (DOR), and progression-free survival (PFS). The treatment was well-tolerated, and the risks were manageable.

BeOne will present the full results for the first time at the 67^th American Society of Hematology (ASH) Annual Meeting and Exposition, December 6-9, in Orlando, Florida. (Oral Presentation: 663; December 7 from 5:00-5:15 PM EST).

Supporting efforts to rapidly advance global review and potential access, BeOne will participate in the FDA’s Project Orbis for sonrotoclax, an initiative that provides a framework for concurrent submission and review of oncology products among international partners. BeOne also intends to submit the Phase 1/2 data to other global regulatory bodies for the potential approval of sonrotoclax in R/R MCL, including the European Medicines Agency.

New drug applications for sonrotoclax for the treatment of R/R MCL and R/R chronic lymphocytic leukemia / small lymphocytic lymphoma (CLL/SLL) have also been accepted and are under review by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for potential accelerated approval.

About Mantle Cell Lymphoma

Mantle cell lymphoma (MCL) is a rare subtype of aggressive non-Hodgkin lymphoma (NHL)¹ that originates in B-cells located in the mantle zone of the lymph nodes. MCL accounts for approximately 5% of all NHL cases globally², affecting an estimated 28,000 people³. MCL is often diagnosed at advanced stages⁴ and nearly all MCL patients will eventually develop refractory or relapsed (R/R) disease.⁵ The five-year survival rate for MCL is approximately 50%, reflecting the urgent need for new therapeutic options.⁶

About Sonrotoclax (BGB-11417)

Sonrotoclax is a next-generation and potentially best-in-class investigational B-cell lymphoma 2 (BCL2) inhibitor with a unique pharmacokinetic and pharmacodynamic profile. Laboratory studies during early drug development have shown that sonrotoclax is a highly potent and specific BCL2 inhibitor with a short half-life and no drug accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies and is in development as a monotherapy and in combination with other therapeutics, including BRUKINSA. Notably, in early clinical trials, sonrotoclax plus BRUKINSA has demonstrated rapid and unprecedented rates of undetectable minimal residual disease (uMRD) in treatment-naïve patients with CLL. To date, more than 2,200 patients have been enrolled across the broad sonrotoclax global development program.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for sonrotoclax for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL). In addition, the FDA has granted sonrotoclax Fast Track Designation for MCL and Waldenström macroglobulinemia, as well as Orphan Drug Designation for the treatment of adult patients with MCL, WM, multiple myeloma, acute myeloid leukemia, and myelodysplastic syndrome.

About BeOne

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 benefits of sonrotoclax; BeOne’s expectations regarding sonrotoclax’s clinical development, regulatory milestones, submissions and approvals; BeOne’s plans to present the full data at an upcoming medical meeting; and BeOne’s plans, commitments, aspirations and goals under the caption “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; BeOne’s 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 ourNewsroom.

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1 Jain, P., and Wang, M. L. (2022). Mantle cell lymphoma: 2022 update on diagnosis, risk stratification, and clinical management. American Journal of Hematology, 97(5), 638–656. https://doi.org/10.1002/ajh.26523

2 Ferlay, J., et al. (2024). Global Cancer Observatory: Non-Hodgkin Lymphoma. International Agency for Research on Cancer. https://gco.iarc.who.int/media/globocan/factsheets/cancers/34-non-hodgkin-lymphoma-fact-sheet.pdf

3 Association of Community Cancer Centers. Relapsed/Refractory Mantle Cell Lymphoma. https://www.accc-cancer.org/home/learn/cancer-types/hematologic-malignancies/mcl

4 Cencini, E., et al. (2024). Survival outcomes of patients with mantle cell lymphoma: A retrospective, 15-year, real-life study. Hematology Reports, 16(1), 50–62. https://doi.org/10.3390/hematolrep16010006

5 Burkart, M., and Karmali R. (2022). Relapsed/refractory mantle cell lymphoma: Beyond BTK inhibitors. Journal of Personalized Medicine, 12(3), 376. https://doi.org/10.3390/jpm12030376

6 Cleveland Clinic. (2023). Mantle cell lymphoma. https://my.clevelandclinic.org/health/diseases/24030-mantle-cell-lymphoma

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