On May 13, 2026 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on developing innovative antibody-based therapeutics for the treatment of cancer, reported financial results for the quarter ended March 31, 2026, and highlighted its recent corporate progress.

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"We are very pleased to report a strong start to the year, building on the momentum generated in 2025. These results reflect our team’s disciplined execution of a strategy designed to sharpen our focus, maximize the value of our pipeline, and strengthen our financial position. As part of this effort, we recently announced the sale of our GMP manufacturing operations to Bora Pharmaceuticals and the monetization of additional ZYNYZ royalties with Sagard Healthcare Partners. Subject to the closing of the manufacturing operations divestiture, these transactions are expected to provide significant non-dilutive capital to support growth opportunities in 2026 and beyond," said Eric Risser, President and CEO of MacroGenics. "We look forward to providing multiple updates during the remainder of the year, including key programmatic milestones for MGC026, MGC028, and MGC030. We believe our increased focus on discovering and developing breakthrough medicines has the potential to enhance patients’ lives while creating meaningful value for our shareholders."

Focus and Realignment Across Our Business

MacroGenics recently took a series of significant steps designed to focus resources on the Company’s innovative oncology programs. These steps include:

Divestiture of Manufacturing Operations. As announced earlier this week, MacroGenics entered into a definitive agreement with Bora Pharmaceuticals Co., Ltd. and Bora Biologics USA, LLC (collectively, Bora) to sell its manufacturing operations, inclusive of drug substance manufacturing, development and quality services. Subject to customary closing conditions, MacroGenics is expected to receive an upfront payment of $122.5 million, before transaction fees and expenses. As part of this transaction, MacroGenics’ headquarters and warehouse sites in Maryland will transfer to Bora. The transaction is expected to close in the third quarter of this year. At closing, MacroGenics will enter into a supply agreement with Bora to support development and production of clinical drug substance for current and future pipeline programs. The Company has historically leveraged both internal manufacturing capabilities as well as those of external contract manufacturing partners. Through this transaction, the Company will transition to a fully outsourced model, which is expected to provide increased flexibility and cost advantages.

Expanded ZYNYZ Royalty Monetization. Earlier this month, MacroGenics announced that it had entered into an amended royalty purchase agreement with Sagard Healthcare Partners (Sagard) in exchange for a revised capped royalty interest in future global net sales of ZYNYZ. MacroGenics received a $60.0 million cash payment from Sagard with the potential to receive an additional milestone, based on 2026 ZYNYZ sales performance, of up to $20.0 million.

Corporate Restructuring. As part of the manufacturing divestiture transaction, approximately 140 employees are expected to transfer to Bora. With some additional reductions across the company, MacroGenics is expected to have approximately 135 employees at closing, enabling a more agile organization that is focused on the research and clinical development of novel therapeutics.

Advancement of Innovative Pipeline

MacroGenics is developing potential best-in-class or first-in-class antibody-drug conjugates (ADCs) and T-cell engagers (TCEs). MacroGenics’ two clinical-stage ADC programs, MGC026 and MGC028, continue to demonstrate acceptable safety profiles to date, with no observations of interstitial lung disease, as well as evidence of anti-tumor activity by Response Evaluation Criteria in Solid Tumors (RECIST).

MGC026 is a novel ADC that targets B7-H3, which is overexpressed in multiple solid tumors. The Company completed enrollment of the dose escalation portion of a Phase 1 study in late 2025 and is currently enrolling patients in the dose expansion portion of the study in selected solid tumor indications. The Company anticipates reporting initial MGC026 clinical data in mid-2026.

MGC028 is a first-in-class ADC that targets ADAM9, which is overexpressed in multiple solid tumors. MGC028 is currently being evaluated in the dose escalation portion of a Phase 1 study in patients with advanced solid tumors. The Company anticipates reporting initial MGC028 clinical data in the second half of 2026.

MGC030 is a first-in-class preclinical ADC that targets an undisclosed antigen expressed across several solid tumors. An Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MGC030 is planned for the third quarter of 2026.

Future Pipeline. MacroGenics is advancing additional preclinical programs that incorporate proprietary platforms for next-generation TCEs and ADCs with novel payloads. The company expects to nominate two additional product candidates by the end of 2026.

Lorigerlimab Update

MacroGenics continues its LINNET Phase 2 monotherapy study of lorigerlimab, a PD-1 × CTLA-4 bispecific DART molecule, in patients with gynecological cancers.

As of a data cut-off on May 7, 2026, 17 patients with clear cell gynecologic cancer (CCGC) were treated at 6 mg/kg every three weeks (Q3W). Of the 16 evaluable patients with CCGC, 4 (25%) had objective responses, including 4 with confirmed partial responses (PR), of which 1 patient subsequently had an unconfirmed complete response (CR). Of the 17 CCGC patients evaluable for safety, Grade ≥3 treatment-related adverse events (TRAEs) occurred in 8 patients (47%) and 2 patients (12%) discontinued treatment due to AEs. No treatment-related fatalities were reported in these patients.

Going forward, MacroGenics intends to enroll an additional 20 CCGC patients at a lower dose of 3 mg/kg Q3W, which was selected based on pharmacokinetic and pharmacodynamic modeling, with the goal of improving safety while maintaining clinical benefit. The Company anticipates completing enrollment of these 20 patients by year-end 2026 and reporting updated study results in the first half of 2027.

Based on an assessment of results from the high-grade serous and platinum-resistant ovarian cancer (PROC) cohort of the LINNET study, the predetermined response rate was not achieved, and the Company no longer intends to pursue development in this indication.

Current Partnerships

MacroGenics maintains partnerships with Incyte Corporation, Sanofi, and Gilead Sciences, which span multiple commercial, clinical and preclinical programs. These include ZYNYZ (retifanlimab-dlwr), TZIELD (teplizumab-mzwv), and MGD024, a clinical-stage CD123 × CD3 bispecific DART molecule. Across these collaborations, the Company remains eligible to receive up to approximately $2.5 billion in aggregate future milestones, in addition to royalties on partnered products.

First Quarter 2026 Financial Results

Cash Position: Cash, cash equivalents and marketable securities balance as of March 31, 2026, was $154.2 million, compared to $189.9 million as of December 31, 2025. The balance as of March 31, 2026, did not include the $60.0 million received from Sagard earlier this month. In addition, the Company is expected to receive $122.5 million proceeds from Bora, less related transaction fees and expenses, in connection with the manufacturing operations divestiture, which is expected to close in the third quarter of this year, subject to customary closing conditions.

Revenue: Total revenue was $20.8 million for the quarter ended March 31, 2026, compared to $13.2 million for the quarter ended March 31, 2025. The increase was due to higher contract manufacturing revenue from higher production volume for external clients and royalty revenue recognized from higher sales of ZYNYZ, offset by decreased collaborative revenue.

Cost of Manufacturing Services: Cost of manufacturing services was $9.5 million for the quarter ended March 31, 2026, compared to $5.4 million for the quarter ended March 31, 2025. The increase was due to increased production for external clients.

R&D Expenses: Research and development expenses were $35.0 million for the quarter ended March 31, 2026, compared to $39.7 million for the quarter ended March 31, 2025. The decrease is primarily due to the discontinuation of further development of vobramitamab duocarmazine. This was partially offset by increased development costs related to MGC028 and the preclinical TCE programs.

G&A Expenses: General and administrative expenses were $9.7 million for the quarter ended March 31, 2026, compared to $10.7 million for the quarter ended March 31, 2025.

Net Loss: Net loss was $36.8 million for the quarter ended March 31, 2026, compared to $41.0 million for the quarter ended March 31, 2025.
Shares Outstanding: Shares of common stock outstanding as of March 31, 2026, were 63,560,068.
Cash Runway Guidance: MacroGenics anticipates that its cash, cash equivalents and marketable securities balance of $154.2 million as of March 31, 2026, in addition to projected and anticipated future payments from partners, including $60.0 million received from Sagard earlier this month plus anticipated sale proceeds of $122.5 million, less related transaction fees and expenses, from Bora related to divestiture of the Company’s manufacturing operations, is expected to support its cash runway through 2028.

(Press release, MacroGenics, MAY 13, 2026, View Source [SID1234665634])

On May 13, 2026 Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd., reported that the U.S. Food and Drug Administration (FDA) has approved INQOVI (decitabine and cedazuridine) plus venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older or who are ineligible for intensive induction chemotherapy. INQOVI in combination with venetoclax is the first and only all-oral combination treatment regimen approved for this patient population, offering an alternative to parenteral hypomethylating agent–based regimens that require frequent clinic visits.

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The approval was supported by results from the Phase 2 ASCERTAIN-V study of INQOVI plus venetoclax in adult patients with newly diagnosed AML who were ineligible for intensive induction chemotherapy.1

Efficacy was established based on complete remission (CR) and the duration of CR (DoCR). Duration of remission was defined as the time from first CR until disease relapse or death from any cause, whichever occurred first. In combination with venetoclax, 42 patients achieved a CR (41.6%, 95% CI: 31.9, 51.8) with a median time to CR of two months (range: 0.4 to 15.3 months). The median duration of CR was not reached (range: 0.5 to 16.3 months).

The Prescribing Information contains Warnings and Precautions for myelosuppression and embryo-fetal toxicity. Please see the Important Safety Information and Prescribing Information for more details.

INQOVI is an orally administered hypomethylating regimen previously approved in the U.S. and Canada for the treatment of adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).2

"This FDA approval represents a significant milestone for patients with newly diagnosed acute myeloid leukemia who are not candidates for intensive induction chemotherapy," said Peter Melnyk, President and Chief Executive Officer of Taiho Oncology. "With the approval of an all-oral regimen, INQOVI in combination with venetoclax brings a new treatment option to this patient population and underscores our commitment to advancing innovative, patient-focused therapies in hematologic malignancies."

In 2026, an estimated 22,720 people in the U.S. will be diagnosed with AML, a cancer of the blood and bone marrow.3 More than half of those patients are likely to be ineligible for intensive induction chemotherapy due to advanced age or health concerns.4

"As a leader in the development of oral anti-cancer regimens, we are proud that INQOVI in combination with venetoclax will now be available for newly diagnosed patients with acute myeloid leukemia in the U.S.," said Harold Keer, MD, PhD, Chief Medical Officer of Taiho Oncology. "This approval marks an important step forward in expanding how treatment can be delivered for this patient population, offering an all‑oral option that can potentially reduce the overall treatment burden associated with receiving treatment in hospitals or infusion centers. We believe this approach has the potential to make a meaningful impact for patients and caregivers."

*ASCERTAIN-V Study: AStx727-07: decitabine + CEdazuRidine TreAtment IN AML, adding Venetoclax

About INQOVI

INQOVI is an orally administered, fixed-dose combination of the DNA hypomethylating agent decitabine together with cedazuridine,5 an inhibitor of cytidine deaminase.6 By inhibiting cytidine deaminase in the gut and the liver, the fixed dose combination is designed to allow for the oral delivery of decitabine to achieve comparable systemic exposure to that of IV decitabine.

Indications and Important Safety Information

INDICATIONS

INQOVI is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

INQOVI is indicated in combination with venetoclax for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Myelosuppression

INQOVI as Monotherapy for MDS or CMML

In patients with MDS or CMML, INQOVI can cause severe myelosuppression, including fatal adverse reactions. Based on laboratory values, new or worsening thrombocytopenia occurred in 82% of patients, with Grade 3 or 4 occurring in 76%. Neutropenia occurred in 73% of patients, with Grade 3 or 4 occurring in 71%. Anemia occurred in 71% of patients, with Grade 3 or 4 occurring in 55%. Febrile neutropenia occurred in 33% of patients, with Grade 3 or 4 occurring in 32%. Thrombocytopenia, neutropenia, anemia, and febrile neutropenia are the most frequent cause of INQOVI dose reduction or interruption, occurring in 36% of patients. Permanent discontinuation due to myelosuppression (febrile neutropenia) occurred in 1% of patients. Myelosuppression and worsening neutropenia may occur more frequently in the first or second treatment cycles and may not necessarily indicate progression of underlying MDS.

Fatal and serious infectious complications can occur with INQOVI. Pneumonia occurred in 21% of patients, with Grade 3 or 4 occurring in 15%. Sepsis occurred in 14% of patients, with Grade 3 or 4 occurring in 11%. Fatal pneumonia occurred in 1% of patients, fatal sepsis in 1%, and fatal septic shock in 1%.

Obtain complete blood cell counts prior to initiation of INQOVI, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors and anti‑infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.

INQOVI in Combination with Venetoclax for AML

In patients with AML, INQOVI can cause severe myelosuppression, including fatal adverse reactions, when given in combination with venetoclax. Based on laboratory values in Study ASTX727-07 Phase 2 new or worsening thrombocytopenia occurred in 70% of patients, with Grade 3 or 4 occurring in 69%. Neutropenia occurred in 48% of patients, with Grade 3 or 4 occurring in 48%. Anemia occurred in 54% of patients, with Grade 3 or 4 occurring in 50%. Febrile neutropenia occurred in 52% of patients, with Grade 3 or 4 occurring in 52%. Thrombocytopenia, neutropenia, anemia, and febrile neutropenia were a frequent cause of INQOVI and/or venetoclax dose reduction or interruption. Dose reductions of INQOVI due to neutropenia and thrombocytopenia occurred in 4% and 1% of patients, respectively. Dose interruptions of INQOVI due to neutropenia, febrile neutropenia, thrombocytopenia, and anemia occurred in 40%, 11%, 8%, and 2% of patients, respectively.

Fatal and serious infectious complications can occur during treatment with INQOVI and venetoclax. Pneumonia occurred in 25% of patients, with Grade 3 or 4 occurring in 20%. Sepsis occurred in 28% of patients, with Grade 3 or 4 occurring in 18%. Fatal pneumonia occurred in 2% of patients and fatal sepsis in 8%.

Obtain complete blood cell counts prior to initiation of INQOVI with venetoclax, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.

Embryo-Fetal Toxicity

Advise patients of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with INQOVI and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with INQOVI and for 3 months after the last dose.

ADVERSE REACTIONS

INQOVI as Monotherapy for MDS or CMML

Serious adverse reactions occurred in 68% of patients who received INQOVI. Serious adverse reactions in > 5% of patients included febrile neutropenia (30%), pneumonia (14%), and sepsis (13%). Fatal adverse reactions occurred in 6% of patients. These included sepsis (1%), septic shock (1%), pneumonia (1%), respiratory failure (1%), and one case each of cerebral hemorrhage and sudden death.

The most common adverse reactions (≥ 20%) were fatigue (55%), constipation (44%), hemorrhage (43%), myalgia (42%), mucositis (41%), arthralgia (40%), nausea (40%), dyspnea (38%), diarrhea (37%), rash (33%), dizziness (33%), febrile neutropenia (33%), edema (30%), headache (30%), cough (28%), decreased appetite (24%), upper respiratory tract infection (23%), pneumonia (21%), and transaminase increased (21%). The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were leukocytes decreased (81%), platelet count decreased (76%), neutrophil count decreased (71%), and hemoglobin decreased (55%).

INQOVI in Combination with Venetoclax for AML

Serious adverse reactions occurred in 82% of patients who received INQOVI+VEN. Serious adverse reactions in > 5% of patients included febrile neutropenia (31%), sepsis (22%), pneumonia (15%), infection (bacterial/viral) (10%), hemorrhage (9%), and dyspnea (6%). Fatal adverse reactions occurred in 8% of patients who received INQOVI+VEN. These included sepsis (5%), dyspnea (2%), myocardial infarction (1%), hemolytic anemia (1%), and tumor lysis syndrome (1%).

The most common adverse reactions (≥ 20%) were neutropenia (60%), febrile neutropenia (52%), thrombocytopenia (52%), hemorrhage (42%), anemia (41%), infection (bacterial/viral) (40%), diarrhea (38%), fatigue (36%), mucositis (36%), constipation (36%), arthralgia (35%), decreased appetite (31%), edema (31%), nausea (31%), dyspnea (30%), white blood cell count decreased (28%), sepsis (28%), pneumonia (25%), rash (25%), transaminitis (24%), myalgia (23%), arrhythmia (21%), and abdominal pain (21%). The most common Grade 3 or 4 laboratory abnormalities (≥ 20%) were decreased leukocytes (91%), decreased lymphocytes (81%), decreased platelets (69%), decreased hemoglobin (50%), and decreased neutrophils (48%).

USE IN SPECIFIC POPULATIONS

Lactation

Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with INQOVI and for 2 weeks after the last dose.

Renal Impairment

No dosage modification of INQOVI is recommended for patients with mild or moderate renal impairment (creatinine clearance [CLcr] of 30 to 89 mL/min based on Cockcroft-Gault). Due to the potential for increased adverse reactions, monitor patients with moderate renal impairment (CLcr 30 to 59 mL/min) frequently for adverse reactions. INQOVI has not been studied in patients with severe renal impairment (CLcr 15 to 29 mL/min) or end-stage renal disease (ESRD: CLcr <15 mL/min).

(Press release, Taiho, MAY 13, 2026, View Source [SID1234665650])

On May 13, 2026 Takeda reported Financial Report For the Quarter Ended March 31, 2026.

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(Presentation, Takeda, MAY 13, 2026, View Source [SID1234666404])

On May 13, 2026 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported that it is approaching the first unblinding of data from its pivotal Phase 2B/3 "MIRACLE" trial evaluating Annamycin in combination with cytarabine for the treatment of subjects that have been relapsed or refractory to their primary line of treatment for acute myeloid leukemia (R/R AML). The trial incorporates two arms of Annamycin at different doses plus cytarabine compared to the control arm of cytarabine plus a placebo. The Company continues to expect this unblinding to occur prior to June 30, 2026, as previously communicated.

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The Company also reported that preliminary blinded efficacy data for the 45 subjects continue to approximate previously disclosed results, including a composite complete remission rate (CRc) exceeding 40% and a complete remission (CR) rate of approximately 30%. These results compare favorably to historical CR rates from two major trials of approximately 17-18% observed with cytarabine alone in similar patient populations. Cytarabine is a currently approved standard of care for second line treatment of AML. Based on preliminary data, the median age for subjects enrolled is in the mid-60’s, with over 30% entering the trial after becoming relapsed from or refractory (R/R) to a prior venetoclax regimen as first line therapy, which is considered a particularly challenging patient group. As of May 1st, a total of 56 subjects have been recruited in the MIRACLE trial to date keeping the Company on track to recruit the 90th subject in Part A in Q3 2026.

"The data coming from MIRACLE is encouraging" said Walter Klemp, Chairman and CEO of Moleculin. "For decades, cytarabine monotherapy regimens, although one of the current standards of care, have set a relatively low bar in relapsed or refractory AML, with historical complete remission rates hovering around 17-18% in previously referenced clinical trials. With our trial comparing two arms of Annamycin in combination with cytarabine compared to cytarabine with a placebo, we are looking forward to the opportunity of showing Annamycin is additive to cytarabine alone in a head to head trial. Against this backdrop, the data emerging from MIRACLE highlight what could be a substantial advancement, suggesting that we may finally be moving beyond the limitations that have defined standard-of-care outcomes."

MIRACLE Trial Progress and Next Steps

The MIRACLE study (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled, adaptive designed clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) portion for purposes of measuring its primary efficacy endpoint. Part A of the MIRACLE trial is designed to evaluate the effectiveness of Annamycin in two dosing arms (190 mg/m² and 230 mg/m²) in combination with cytarabine (also referred to as Ara-C) as compared to a control arm of cytarabine plus placebo. The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data of the three arms at 45 subjects in Part A, in addition to the conclusion of Part A (at 90 total subjects). The first early unblinding should yield approximately 30 subjects treated with Annamycin (190 mg/m2 and 230 mg/m2) in combination with cytarabine and 15 subjects treated with just cytarabine plus placebo. The MIRACLE trial is being offered only to AML patients who have had a single prior induction therapy (2nd line patients or 2L) including subjects that were treated with a Venetoclax regimen.

The unblinding of the first 45 subjects is on track to occur in late Q2 2026, as the data are subject to final data entry and audit. The second group of 45 subjects in Part A are expected to be fully recruited in the third quarter of 2026 with unblinding expected late in the second half of 2026. Recruitment of the second group of 45 subjects in Part A will continue uninterrupted while the first 45 subjects with efficacy are being unblinded. Unblinding for the full 90 subjects in Part A may require more time than for the first 45 as it involves more data to support the transition from Part A to Part B.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU can be found on euclinicaltrials.eu and the reference identifier is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

(Press release, Moleculin, MAY 13, 2026, View Source [SID1234665635])

On May 13, 2026 BeOne Medicines Ltd. ("BeOne") (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported 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.

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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) (Free ASH Whitepaper) 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) (Free ASCO Whitepaper) 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.

(Press release, BeOne Medicines, MAY 13, 2026, View Source [SID1234665651])