(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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