SAN CARLOS, Calif. - Thursday, 14. May 2026
BEQALZI is a
foundational BCL2 inhibitor designed for greater potency and
selectivity, with potential to improve efficacy, tolerability, and
convenience versus others in the class
Approval of BEQALZI marks
the first new BCL2 inhibitor approved in a decade in the U.S. and the
only BCL2 inhibitor approved in MCL, aiming to set a new standard of
innovation
(BUSINESS WIRE)--BeOne Medicines Ltd.
(“BeOne”) (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology
company, today announced that the U.S. Food and Drug Administration
(FDA) has granted accelerated approval to BEQALZI™ (bee-KAHL-zee;
sonrotoclax), a foundational, next-generation BCL2 inhibitor, for the
treatment of adult patients with relapsed or refractory (R/R) mantle
cell lymphoma (MCL), after at least two lines of systemic therapy,
including a Bruton’s tyrosine kinase (BTK) inhibitor. BEQALZI was
designed to enhance BCL2 inhibition—with greater potency, selectivity,
and a pharmacologic profile with potential to improve efficacy,
tolerability, and convenience over others in the class.
Michael
Wang, M.D., Global Principal Investigator, the Puddin Clarke Endowed
Professor, Department of Lymphoma and Myeloma, The University of Texas
MD Anderson Cancer Center, said:
“The data supporting the
approval of sonrotoclax in the U.S. confirm its role as a foundational
therapy for mantle cell lymphoma in the post-BTK inhibitor setting, and
demonstrate that it can deliver robust disease control when treatment
choices are limited and outcomes are poor. From a clinical perspective,
this provides physicians with an important new option grounded in both
efficacy and tolerability, fundamentally changing how we think about
sequencing therapy in this disease.”
Data supporting approval
The
accelerated approval of BEQALZI is supported by efficacy and safety
data from the Phase 1/2 study, BGB-11417-201 (NCT05471843), which was
presented at the 67th American Society of Hematology (ASH) Annual
Meeting & Exposition. The study included an independent review of
efficacy data and demonstrated:
Overall response rate (ORR): 52% (95% CI, 42-62)
Complete response (CR) rate: 16% (95% CI, 9.1-24.0)
Median time to response (TTR): 1.9 months
Median duration of response (DOR): 15.8 months (95% CI, 7.4
months-NE) at a median response follow-up of 11.9 months (has yet to
reach full maturity)
Safety: treatment with sonrotoclax monotherapy was generally well tolerated
Continued
approval for this indication is contingent upon confirmation of
clinical benefit in the confirmatory CELESTIAL-RRMCL trial
(NCT06742996), which is underway. The U.S. FDA granted Breakthrough
Therapy Designation (BTD) for sonrotoclax in this indication, as well as
Fast Track Designation and Orphan Drug Designation.
Amit Agarwal, M.D., Ph.D., Chief Medical Officer, Hematology, BeOne Medicines, said:
“BeOne
is leading the advancement and enhancement of BCL2 inhibition to
revolutionize how we treat patients living with B-cell malignancies.
Today’s approval of BEQALZI represents critical progress for patients
with mantle cell lymphoma and reinforces our strategy of building
foundational medicines designed to raise the standard of care in B-cell
malignancies.”
A new BCL2 option for a challenging R/R MCL post–BTK inhibitor setting
MCL
is a rare and often aggressive subtype of non-Hodgkin lymphoma. In the
United States, approximately 3,300 new cases of MCL are diagnosed each
year.1 While many patients respond to initial therapy, relapse is
common, and outcomes after progression can be poor, particularly after
prior treatment with a BTK inhibitor. The accelerated approval of
BEQALZI introduces a new targeted mechanism to the MCL treatment
landscape and reinforces the importance of expanding therapeutic choices
for patients as the disease evolves.
Meghan Gutierrez, Chief Executive Officer, Lymphoma Research Foundation, said:
“For
people living with relapsed or refractory mantle cell lymphoma, each
progression can bring uncertainty and questions regarding remaining
treatment options. The FDA approval of sonrotoclax represents
significant progress for the U.S. mantle cell lymphoma community,
offering renewed hope for patients and families who have exhausted other
available therapies. Advances like this underscore why continued
research and innovation in this disease remain so critical.”
Additional regulatory and development updates
BEQALZI
is also approved in China for the treatment of R/R MCL, as well as
adult patients with chronic lymphocytic leukemia (CLL)/small lymphocytic
lymphoma (SLL) who have previously received at least one systemic
therapy, including a BTK inhibitor. Data from the Phase 1/2 study of
sonrotoclax in R/R MCL is also under review by the European Medicines
Agency and other regulatory agencies.
The U.S. FDA also granted
sonrotoclax Fast Track Designation for Waldenström macroglobulinemia
(WM), as well as Orphan Drug Designation for the treatment of adult
patients with WM, multiple myeloma, acute myeloid leukemia, and
myelodysplastic syndrome.
Additionally, sonrotoclax is currently
being studied in combination with other therapeutics, including
zanubrutinib, as a potential treatment for CLL, with updated data
expected to be presented at the 2026 American Society of Clinical
Oncology (ASCO) Annual Meeting.
About BEQALZI™ (sonrotoclax)
BEQALZI™
(sonrotoclax) is a foundational, next-generation and potentially
best-in-class B-cell lymphoma 2 (BCL2) inhibitor with a unique
pharmacokinetic and pharmacodynamic profile. Preclinical and clinical
studies in 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, including chronic lymphocytic
leukemia (CLL), and is in development as a monotherapy and in
combination with other therapeutics, including zanubrutinib. To date,
more than 2,200 patients have been enrolled across the broad sonrotoclax
global development program.
INDICATION
BEQALZI™
(sonrotoclax) is a BCL-2 inhibitor indicated for the treatment of adult
patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL)
after at least two lines of systemic therapy, including a Bruton’s
tyrosine kinase (BTK) inhibitor.
This indication is approved
under accelerated approval based on response rate and duration of
response. Continued approval for this indication may be contingent upon
verification and description of clinical benefit in a confirmatory
trial(s).
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
BEQALZI
is contraindicated with strong CYP3A inhibitors at initiation and
during the ramp-up phase due to the potential for an increased risk of
tumor lysis syndrome (TLS).
WARNINGS & PRECAUTIONS
Tumor Lysis Syndrome (TLS): BEQALZI can cause rapid tumor reduction and
changes in blood chemistries consistent with TLS, which may be serious
or life-threatening and require prompt management. TLS may occur as
early as 4 hours after the first dose, with dose increases, or upon
reinitiation following treatment interruption. Laboratory or clinical
TLS occurred in 7% of patients who followed the recommended dose
ramp-up. Assess all patients for TLS risk and initiate prophylaxis,
including adequate hydration and antihyperuricemics. For patients at
high risk of TLS, consider hospitalization with intravenous hydration
and employ frequent monitoring. Monitor blood chemistries closely and
manage abnormalities promptly. Interrupt BEQALZI for TLS; upon
reinitiation, follow dose modification guidance in the Prescribing
Information.
Serious Infections: BEQALZI can cause fatal or
serious infections. Serious infections occurred in 14% of patients;
Grade 3-4 occurred in 17% (fatal: 2.6%). The most common Grade 3 or
greater infection was pneumonia (10%). Monitor for signs and symptoms of
infection and treat appropriately. Consider prophylactic antimicrobials
and immunoglobulins. Interrupt, reduce dose, or permanently discontinue
BEQALZI based on severity.
Neutropenia: BEQALZI can cause
serious or severe cytopenias, including neutropenia. Grade 3 or 4
decreases in neutrophils occurred in 18% of patients (Grade 4: 6%);
febrile neutropenia occurred in 1.7% of all patients. Monitor complete
blood counts throughout treatment. Interrupt treatment, reduce the dose,
or permanently discontinue BEQALZI based on severity.
Embryo-Fetal Toxicity: BEQALZI can cause fetal harm when administered to
pregnant women. Advise patients of the potential risk to a fetus.
Verify pregnancy status prior to initiation. Advise females to use
effective contraception and males with female partners of reproductive
potential to use effective contraception during treatment and for 1 week
after the last dose.
ADVERSE REACTIONS
The most common
adverse reactions (≥15%) are pneumonia (16%) and fatigue (16%). The most
common Grade 3-4 laboratory abnormalities (≥15%) are decreases in
lymphocytes (29%) and neutrophils (18%).
DRUG INTERACTIONS
Strong or Moderate CYP3A Inhibitors: Concomitant use increases
BEQALZI exposure. Avoid use of strong CYP3A inhibitors during BEQALZI
initiation and ramp-up. Avoid use of moderate CYP3A inhibitors at the 1
mg and 2 mg doses; for all other doses, reduce the BEQALZI dose with
concomitant use. See approved labeling for dose modifications.
Strong or Moderate CYP3A Inducers: Concomitant use decreases BEQALZI exposure. Avoid use.
SPECIAL POPULATIONS
Lactation: Advise women not to breastfeed during treatment with BEQALZI and for 1 week after the last dose.
To
report SUSPECTED ADVERSE REACTIONS, contact BeOne Medicines at
1-877-828-5596 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full U.S. Prescribing Information.
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 benefits
of sonrotoclax; BeOne’s expectations regarding sonrotoclax’s clinical
development, regulatory milestones, submissions and approvals; 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 periodic report, 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.
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Contacts
Investor Contact
Liza Heapes
+1 857-302-5663
ir@beonemed.com
Media Contact
Kyle Blankenship
+ 1 667-351-5176
media@beonemed.com