On December 7, 2025 BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported new data on sonrotoclax, a next-generation investigational BCL2 inhibitor, demonstrating meaningful clinical benefit as monotherapy and in combination across B-cell malignancies. These data were featured at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in Orlando, Florida. The five presentations highlight durable responses in heavily pretreated patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL) and additional studies showing deep, rapid, and sustained undetectable minimal residual disease (uMRD) rates with sonrotoclax-based combinations in patients with treatment-naive chronic lymphocytic leukemia (CLL), highlighting the foundational potential of this medicine.

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"The data we’re presenting at ASH (Free ASH Whitepaper) 2025 are redefining what physicians can expect from sonrotoclax as a next-generation BCL2 inhibitor," said Amit Agarwal, M.D., Ph.D., Chief Medical Officer, Hematology, BeOne. "Our data demonstrates that sonrotoclax has succeeded where others have failed, achieving deep and durable responses as a monotherapy in both R/R CLL and MCL and notably fast kinetics as a combination therapy in treatment-naïve CLL. With these results, we believe sonrotoclax will become a foundational medicine in B-cell malignancies, potentially transforming outcomes for patients worldwide."

Sonrotoclax could become the first BCL2 inhibitor indicated for R/R MCL in the U.S., based on data showing an overall response rate (ORR) of 52.4%. (Oral Presentation: 663; December 7 from 5:00-5:15 PM EST)

In this Phase 1/2, global, multicenter, single-arm, open-label study (NCT05471843), ORR by IRC was 52.4% (95% CI, 42.4-62.4) with a complete response (CR) rate of 15.5% (95% CI, 9.1-24.0) in patients with R/R MCL post-treatment with anti-CD20 therapy and a BTK inhibitor treated with 320 mg of sonrotoclax (n=103). Notably, ORR by IRC benefit was consistent across patients with high-risk disease subtypes, including patients with TP53 mutation, a key prognostic marker for MCL. In this patient group, ORR by IRC was 59.1% (95% CI, 36.3-79.3).

At a median study follow-up of 14.2 months (range, 0.3-24.9 months), the median duration of response (DOR) by IRC was 15.8 months (95% CI, 7.4 months-NE) and has yet to reach full maturity. The median time to response (TTR) was 1.9 months (range 1.6-6.5 months), and the median progression-free survival (PFS) was 6.5 months (95% CI: 4.0-10.4).

Treatment with sonrotoclax monotherapy was generally well tolerated, and adverse events were manageable. The most common grade ≥3 treatment-emergent adverse events (TEAEs) in greater than 10% of patients were neutropenia (19.1%), infections (16.5%), and pneumonia (10.4%).

These data are under Priority Review by the U.S Food and Drug Administration for potential accelerated approval.

"Achieving deep and durable responses in relapsed or refractory mantle cell lymphoma after BTK inhibitor therapy has been a long-standing challenge," said Michael Wang, M.D., Professor, Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, and presenting author of the study. "In this analysis, sonrotoclax monotherapy demonstrated meaningful and lasting responses in heavily pretreated patients, including those with high-risk disease. These findings are highly encouraging and suggest this next-generation BCL2 inhibitor could play a foundational role in improving outcomes for patients with limited treatment options."

Sonrotoclax combinations demonstrate fast responses with unmatched uMRD rates and notably better kinetics than current options

BGB-11417-101 (NCT04277637) is an ongoing, phase 1/1b, dose-escalation/expansion study in patients with B-cell malignancies. Results presented at ASH (Free ASH Whitepaper) showcase data from sonrotoclax combinations in patients with treatment-naive (TN) CLL/SLL. Notable highlights include:

Sonrotoclax plus zanubrutinib (Poster Presentation: 3891)
In 135 efficacy-evaluable patients, ORR was 100%, with CR/CR with incomplete count recovery (CRi) in 55% of the 320-mg cohort. Median TTR was 2.6 months (range, 1.5-10.8 months).
At 48 weeks of combination treatment, the uMRD4 rate in the 320-mg cohort was 91% and uMRD rates continue to increase over time, with 98% of patients achieving uMRD4 by 96 weeks.
Median time from the initiation of the combination to uMRD4 was 4.5 months.
With a median study follow-up of 30.9 months, no progression events have been observed in the 320-mg cohort, including in the 40% of patients (34 patients) who had electively discontinued treatment.
The combination was generally well tolerated, with no TEAEs leading to death, or clinical or laboratory tumor lysis syndrome (TLS).
Sonrotoclax plus obinutuzumab (Oral Presentation: 793)
In the 320 mg efficacy-evaluable cohort (n=30), the ORR was 93%, with CR/CRi in 43% of patients.
The median time from reaching sonrotoclax target dose to uMRD was 2.3 months (range, 1.4-5.6 months) in the 320-mg cohort.
The combination was generally well tolerated, with no sonrotoclax discontinuations or deaths due to TEAEs.
Updated efficacy and safety data will be presented on Monday, December 8, 10:30 AM–10:45 AM EST.
Sonrotoclax plus zanubrutinib and obinutuzumab (Poster Presentation: 3890)
In 15 efficacy-evaluable patients, the ORR was 100%, with a CR/CRi rate of 40%.
Of the MRD-evaluable patients (n=10), 100% achieved uMRD4, discontinued treatment as mandated by the protocol, and remain in remission; 80% of patients achieved uMRD6.
With a median study follow-up of approximately 18.0 months, no PFS events have occurred.
The combination was generally well tolerated, and no deaths or discontinuations of any study drug due to TEAEs were observed.
Sonrotoclax monotherapy achieves an ORR by IRC of 76%, with a CR/CRi of 19%, in patients with R/R CLL/SLL, demonstrating rapid and deep responses (Poster Presentation: 5666)

BGB-11417-202 (NCT05479994) is an open-label, phase 2, and a potential registrational study evaluating the efficacy and safety of sonrotoclax in 100 heavily pretreated patients with R/R CLL/SLL. At a median follow-up of 14.4 months (range, 0.2-27.5 months), primary analysis results show:

Similar ORR and CR responses were seen in patients with unmutated IGHV, del(17p) and/or TP53 mutation, and BTK mutation. Median TTR was 3.7 months (range, 1.3-11.1 months).
The best blood uMRD rate was 49.0% (n=49/100). Median time to blood uMRD4 was 5.8 months (range, 3-12 months).
Sonrotoclax monotherapy was well tolerated, and toxicities were manageable and no clinical TLS occurred.
Updated data from the abstract will be presented on Monday, December 8, 6:00-8:00 PM EST. Additionally, these data are under review by China’s National Medical Products Administration (NMPA) for potential accelerated approval.
For more information about our presence at the 2025 ASH (Free ASH Whitepaper) Annual Meeting and Exposition, please visit our meeting hub: congress.beonemedicines.com.

About Mantle Cell Lymphoma

Mantle cell lymphoma (MCL) is a rare subtype of aggressive B-cell non-Hodgkin lymphoma (NHL)1 that develops 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 Chronic Lymphocytic Leukemia

Chronic lymphocytic leukemia (CLL) is a life-threatening cancer of adults. It is a type of mature B-cell malignancy in which abnormal leukemic B lymphocytes (a type of white blood cells) arise from the bone marrow and flood peripheral blood, bone marrow, and lymphoid tissues.7,8 CLL is the most common type of leukemia in adults, accounting for about one-third of new cases.2,9

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.

The information provided in this press release is intended for a global audience.

(Press release, BeOne Medicines, DEC 7, 2025, View Source [SID1234661215])

On December 7, 2025 Kite, a Gilead Company (Nasdaq: GILD), reported a new analysis demonstrating that second-line Yescarta (axicabtagene ciloleucel) therapy offers consistent benefits in patients with relapsed/refractory large B-cell lymphoma (R/R LBCL), even among those ineligible for the previous standard of care, high-dose chemotherapy followed by an autologous stem cell transplant (ASCT).

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Results were shared from the combined analysis of four-year data from the landmark ZUMA-7 Phase 3 pivotal study of Yescarta for R/R LBCL and two-year data from the Phase 2 ALYCANTE study, designed by French collaborative group LYSA and sponsored by LYSARC, for transplant-ineligible patients. The findings were presented (Abstract #3714) during the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

"Patients with large B-cell lymphoma who are ineligible for stem cell transplants face limited treatment options and poor outcomes due to age, co-morbidities and other factors," said Professor Roch Houot, Head of Haematology Department, University Hospital of Rennes, France and coordinator of the ALYCANTE study. "This analysis reinforces that Yescarta should be considered earlier in a patient’s treatment paradigm given its curative potential as a one-time treatment and further establishes CAR T as the new standard of care for second-line treatment of large B-cell lymphoma."

The efficacy analyses included 178 and 69 patients from the ZUMA-7 and ALYCANTE trials, respectively.

After two years:

Overall survival (OS) rate, meaning the percentage of patients in the trials who were still alive, was 64.9% in the pooled analysis, 62.8% in ZUMA-7, and 70.8% in ALYCANTE. Historically, the prognosis for R/R LBCL was very poor; prior to the introduction of new therapies like CAR Ts, the two-year survival rate was only about 20%.
Event-free survival (EFS) rate, meaning the percentage of patients who were still alive and had not seen their disease worsen or experienced other major complications, was 45.2% in the pooled analysis, 45.4% in ZUMA-7, and 44.7% in ALYCANTE.
Progression-free survival (PFS) rate, meaning the percentage of patients who were still living without their disease getting worse, was 47.4% in the pooled analysis, 47.6% in ZUMA-7, and 46.8% in ALYCANTE.
Additionally:

After three months, 55.6% of the patients in the pooled analysis showed a complete metabolic response (CMR), meaning their disease was barely, if at all, detectable. CMR was 51.2% in ZUMA-7 and 67.7% in ALYCANTE.
After one year after treatment, overall response rate (ORR), meaning patients saw their cancer significantly shrink or completely disappear, was 46.6% in the pooled analysis, 46.5% in ZUMA-7, and 46.8% in ALYCANTE.
For those patients who responded well to treatment, 61% saw that positive response continue after a full year. Pooled 12-month duration of response (DOR) was 61.0%, 60.6% in ZUMA-7, and 62.1% in ALYCANTE.
In the safety analysis, which included 170 ZUMA-7 and 62 ALYCANTE Yescarta-infused patients, safety outcomes were comparable between the two studies. The incidence of grade ≥3 treatment-emergent adverse events (TEAEs) was similar between the ZUMA-7 and ALYCANTE patients, 91.2% and 88.7% respectively, with a pooled incidence of 90.5%. Pooled incidence of grade ≥3 neurologic events, neutropenias, and anemia were 19.8% (ZUMA-7 21.2%, ALYCANTE 16.1%), 64.7% (ZUMA-7 70.0%, ALYCANTE 50.0%), and 27.6% (ZUMA-7 30.0%, ALYCANTE 21.0%), respectively.

Across both studies, patients generally experienced similar long-term improvements in their quality of life after initial treatment challenges. At Day 50 after treatment, patients in both trials showed a transient decline (-7.2 in ZUMA-7 and -6.3 in ALYCANTE) for EORTC QLQ-C30 global health status, and -12.9 for physical function in both. However, patients in ALYCANTE reported a meaningful improvement in their overall well-being (EQ-5D-5L VAS) by Day 100 (+9.9), while ZUMA-7 patients reached a similar meaningful improvement (+9.9) at Day 150, which continued through Month 12. By month 24, both ALYCANTE and ZUMA-7 showed continued improvements of global health status.

"This analysis offers compelling evidence of Yescarta’s consistent, durable efficacy and safety profile across a broad range of patients, including those with difficult-to-treat relapsed or refractory disease who historically faced very limited options and a poor prognosis," said Gallia Levy, MD, PhD, Senior Vice President and Global Head of Development, Kite. "These robust data further reinforce Yescarta’s potential as a treatment with curative intent, reflecting Kite’s deep commitment to transforming patient outcomes."

About LBCL

Globally, LBCL is the most common type of non-Hodgkin lymphoma. In the United States, more than 18,000 people are diagnosed with LBCL each year. About 30-40% of patients with LBCL will need second-line treatment, as their cancer will either relapse (return) or become refractory (not respond) to initial treatment.

About ALYCANTE Study

ALYCANTE (NCT04531046) is a Phase 2 study evaluating the efficacy and safety of Yescarta in patients with R/R LBCL after one prior line of therapy who were deemed ineligible for high-dose chemotherapy and ASCT, sponsored by the LYSA/LYSARC collaborative group. The primary endpoint was the complete metabolic response at three months from Yescarta infusion. The study was funded by Kite, a Gilead Company, and carried out with Yescarta manufactured by Kite.

About LYSA and LYSARC

The Lymphoma Study Association (LYSA) is an independent, multidisciplinary network and an international leader in clinical lymphoma and CLL/WM research. With over 500 experts and a network of 90 centers across France, Belgium, and Portugal, it conducts clinical trials ranging from early-stage treatment evaluation to the development of new therapeutic strategies.

The Lymphoma Academic Research Organization (LYSARC), based in France, is the largest European academic organization dedicated to clinical lymphoma and CLL/WM research. As the operational arm of LYSA, LYSARC sponsors and leads research initiatives and operates specialized platforms in pathology, biology, and imaging. It manages and coordinates numerous clinical trials (phases 1 to 4) each year, as well as non-interventional and data reuse studies. LYSARC is a key research expert, leading innovative projects on an international scale.

About ZUMA-7 Study

Based on the primary efficacy endpoint results of ZUMA-7, the U.S. Food & Drug Administration approved Yescarta as initial treatment of R/R LBCL in April 2022. The EU granted approval in October 2022, followed by approvals in several other countries including Australia, Canada, Great Britain, Israel, Japan and Switzerland.

ZUMA-7 is a randomized, open-label, global, multicenter, Phase 3 study evaluating the safety and efficacy of Yescarta versus standard of care (SOC) for second-line therapy in adult patients with relapsed or refractory LBCL within 12 months of first-line therapy. The SOC for initial treatment of R/R LBCL has been a multi-step process involving platinum-based salvage combination chemotherapy regimen, and for responders, HDT and ASCT. In the study, 359 patients in 77 centers around the world were randomized (1:1) to receive a single infusion of Yescarta or SOC second-line treatment. The primary endpoint was EFS as determined by blinded central review and defined as the time from randomization to the earliest date of disease progression per Lugano Classification, commencement of new lymphoma therapy, or death from any cause. Key secondary endpoints include objective response rate and OS. Additional secondary endpoints included patient-reported outcomes (PROs) and safety. Per hierarchical testing of primary and key secondary endpoints and group sequential testing of OS, an interim analysis of OS occurred at the time of the primary EFS.

About Yescarta

Please see full Prescribing Information, including BOXED WARNING below and Medication Guide.

YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy.
Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Limitations of Use: YESCARTA is not indicated for the treatment of patients with primary central nervous system lymphoma.

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, and SECONDARY HEMATOLOGICAL MALIGNANCIES

Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving YESCARTA. Do not administer YESCARTA to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids.
Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving YESCARTA, including concurrently with CRS or after CRS resolution. Monitor for neurologic toxicities after treatment with YESCARTA. Provide supportive care and/or corticosteroids, as needed.
T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including YESCARTA.
CYTOKINE RELEASE SYNDROME (CRS)

CRS, including fatal or life-threatening reactions, occurred following treatment with YESCARTA. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL), including ≥ Grade 3 CRS in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including ≥ Grade 3 in 9%. Among patients with LBCL who died after receiving YESCARTA, 4 had ongoing CRS events at the time of death. For patients with LBCL in ZUMA-1, the median time to onset of CRS was 2 days following infusion (range: 1-12 days) and the median duration was 7 days (range: 2-58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1-10 days) and the median duration was 7 days (range: 2-43 days).

CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including ≥ Grade 3 CRS in 8%. Among patients with iNHL who died after receiving YESCARTA, 1 patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1-20 days) and median duration was 6 days (range: 1-27 days) for patients with iNHL.

Key manifestations of CRS (≥ 10%) in all patients combined included fever (85%), hypotension (40%), tachycardia (32%), chills (22%), hypoxia (20%), headache (15%), and fatigue (12%). Serious events that may be associated with CRS include, cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), renal insufficiency, cardiac failure, respiratory failure, cardiac arrest, capillary leak syndrome, multi-organ failure, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS).

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events, CRS occurred in 93% (38/41), including 2% (1/41) with Grade 3 CRS; no patients experienced a Grade 4 or 5 event. The median time to onset of CRS was 2 days (range: 1-8 days) and the median duration of CRS was 7 days (range: 2-16 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Thirty-one of the 39 patients (79%) developed CRS and were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing ≥ Grade 3 CRS. The median time to onset of CRS was 5 days (range: 1-15 days) and the median duration of CRS was 4 days (range: 1-10 days). Although there is no known mechanistic explanation, consider the risk and benefits of prophylactic corticosteroids in the context of pre-existing comorbidities for the individual patient and the potential for the risk of Grade 4 and prolonged neurologic toxicities.

Confirm that 2 doses of tocilizumab are available prior to infusion of YESCARTA. Monitor patients at least daily for 7 days following infusion for signs and symptoms of CRS. Monitor patients for signs or symptoms of CRS for 2 weeks after infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES

Neurologic toxicities including immune effector cell-associated neurotoxicity syndrome (ICANS) that were fatal or life-threatening occurred following treatment with YESCARTA. Neurologic toxicities occurred in 78% (330/422) of patients with NHL receiving YESCARTA, including ≥ Grade 3 in 25%.

Neurologic toxicities occurred in 87% (94/108) of patients with LBCL in ZUMA-1, including ≥ Grade 3 in 31% and in 74% (124/168) of patients in ZUMA-7 including ≥ Grade 3 in 25%. The median time to onset was 4 days (range: 1-43 days) and the median duration was 17 days for patients with LBCL in ZUMA-1. The median time to onset for neurologic toxicity was 5 days (range: 1-133 days) and median duration was 15 days in patients with LBCL in ZUMA-7. Neurologic toxicities occurred in 77% (112/146) of patients with iNHL, including ≥ Grade 3 in 21%. The median time to onset was 6 days (range: 1-79 days) and the median duration was 16 days. Ninety-eight percent of all neurologic toxicities in patients with LBCL and 99% of all neurologic toxicities in patients with iNHL occurred within the first 8 weeks of YESCARTA infusion. Neurologic toxicities occurred within the first 7 days of infusion in 87% of affected patients with LBCL and 74% of affected patients with iNHL.

The most common neurologic toxicities (≥ 10%) in all patients combined included encephalopathy (50%), headache (43%), tremor (29%), dizziness (21%), aphasia (17%), delirium (15%), and insomnia (10%). Prolonged encephalopathy lasting up to 173 days was noted. Serious events, including aphasia, leukoencephalopathy, dysarthria, lethargy, and seizures occurred. Fatal and serious cases of cerebral edema and encephalopathy, including late-onset encephalopathy, have occurred.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of neurologic toxicities was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received corticosteroids at the onset of Grade 1 toxicities, neurologic toxicities occurred in 78% (32/41) and 20% (8/41) had Grade 3 neurologic toxicities; no patients experienced a Grade 4 or 5 event. The median time to onset of neurologic toxicities was 6 days (range: 1-93 days) with a median duration of 8 days (range: 1-144 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Of those patients, 85% (33/39) developed neurologic toxicities; 8% (3/39) developed Grade 3 and 5% (2/39) developed Grade 4 neurologic toxicities. The median time to onset of neurologic toxicities was 6 days (range: 1-274 days) with a median duration of 12 days (range: 1-107 days). Prophylactic corticosteroids for management of CRS and neurologic toxicities may result in higher grade of neurologic toxicities or prolongation of neurologic toxicities, delay the onset, and decrease the duration of CRS.

Monitor patients for signs and symptoms of neurologic toxicities following infusion at least daily for 7 days; and for 2 weeks thereafter and treat promptly. Advise patients to avoid driving for at least 2 weeks following infusion.

HYPERSENSITIVITY REACTIONS

Allergic reactions may occur with the infusion of YESCARTA. Serious hypersensitivity reactions, including anaphylaxis, may be due to dimethyl sulfoxide (DMSO) or residual gentamicin in YESCARTA.

SERIOUS INFECTIONS

Severe or life-threatening infections occurred after YESCARTA infusion. Infections (all grades) occurred in 45% of patients with NHL. Grade 3 or higher infections occurred in 17% of patients, including ≥ Grade 3 infections with an unspecified pathogen in 12%, bacterial infections in 5%, viral infections in 3%, and fungal infections in 1%. YESCARTA should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 36% of patients with NHL and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, including those who have received YESCARTA, life-threatening and fatal opportunistic infections including disseminated fungal infections (e.g., candida sepsis and aspergillus infections) and viral reactivation (e.g., human herpes virus-6 [HHV-6] encephalitis and JC virus progressive multifocal leukoencephalopathy [PML]) have been reported. The possibility of HHV-6 encephalitis and PML should be considered in immunosuppressed patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, has occurred in patients treated with drugs directed against B cells, including YESCARTA. Perform screening for HBV, HCV, and HIV and management in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS

Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and YESCARTA infusion. Grade 3 or higher cytopenias not resolved by Day 30 following YESCARTA infusion occurred in 39% of all patients with NHL and included neutropenia (33%), thrombocytopenia (13%), and anemia (8%). Monitor blood counts after infusion.

HYPOGAMMAGLOBULINEMIA

B-cell aplasia and hypogammaglobulinemia can occur in patients receiving YESCARTA. Hypogammaglobulinemia was reported as an adverse reaction in 14% of all patients with NHL. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement.

The safety of immunization with live viral vaccines during or following YESCARTA treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during YESCARTA treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES

Patients treated with YESCARTA may develop secondary malignancies. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including YESCARTA. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes.

Monitor life-long for secondary malignancies. In the event that a secondary malignancy occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

ADVERSE REACTIONS

The most common adverse reactions (incidence ≥ 20%) in:

patients with LBCL in ZUMA-7 included fever, CRS, fatigue, hypotension, encephalopathy, tachycardia, diarrhea, headache, musculoskeletal pain, nausea, febrile neutropenia, chills, cough, infection with unspecified pathogen, dizziness, tremor, decreased appetite, edema, hypoxia, abdominal pain, aphasia, constipation, and vomiting.
patients with LBCL in ZUMA-1 included CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections with pathogen unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.
Please see full Prescribing Information, including BOXED WARNING and Medication Guide.

(Press release, Gilead Sciences, DEC 7, 2025, View Source [SID1234661214])

On December 7, 2025 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics ("Molecular Partners" or the "Company"), reported it will present updated data from a Phase 1/2a trial of the multispecific T-cell engager MP0533 in patients with acute myeloid leukemia (AML) in a poster at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 6-9, 2025, in Orlando, Florida.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The poster outlines the latest results of this multicenter, open-label study evaluating MP0533 for the treatment of patients with relapsed/refractory (R/R) AML and myelodysplastic syndrome (MDS)/AML (ClinicalTrials.gov: NCT05673057). Data from cohorts 8 and 9 show that densified MP0533 dosing appears tolerable and leads to markedly improved serum exposure in treatment cycle 1, with encouraging preliminary antitumor activity.

"The results in patients with higher frequency dosing regimens of MP0533 are very encouraging. I am happy to see the clinical benefit of MP0533 in a mutation-agnostic manner in R/R AML patients, in particular those with lower disease burden. The data indicate that MP0533 has the potential to significantly improve treatment options for patients with AML and I support investigating MP0533 in a Phase 2 setting to confirm its safety and activity in earlier lines as consolidation addition to existing backbones," said Prof. Courtney DiNardo, M.D., Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, and co-chair of the MP0533 dose escalation review committee (DERC).

As of the data cut-off of September 1, 2025, 54 patients had been treated with MP0533. Eight of 48 evaluable patients achieved a response, with five reaching composite complete responses (3 complete responses, CR, and 2 CR with partial hemotologic recovery) and three reaching morphologic leukemia-free state (MLFS). Six of the 8 responders, and 11 of 14 patients who achieved a reduction in bone marrow blasts of more than 50%, presented with less than 20% bone marrow blast at baseline, indicating that patients with low disease burden are most likely to benefit from MP0533. One patient in cohort 8 has been in complete remission for over a year, and one patient in cohort 9 is on the trial since 4 months. Cohort 10, which aims at reaching the same target dose as cohort 8 while exposing patients to more drug over time, is ongoing with data expected in 2026.

"The Phase 1/2a trial with MP0533 is making good progress, with the densified dosing regimen showing to be feasible, with an acceptable safety profile, and resulting in clinical benefit. The results warrant further exploration of MP0533’s potential, both in the R/R and front-line AML settings in combination with standard of care, and several consortia have approached us expressing interest in conducting such studies. We are actively engaging with potential partners and continue our dialogue with key opinion leaders and regulators to shape the next phase of development of our innovative T-cell engager for patients," said Philippe Legenne, M.D., CMO of Molecular Partners.

MP0533 is a novel tetra-specific T cell-engaging DARPin designed for selective and broad killing of AML cells in a mutation-agnostic manner. MP0533 simultaneously targets three tumor-associated antigens CD33, CD123, and CD70 on AML cells as well as the immune activator CD3 on T cells. AML cells commonly co-express at least two of the three target antigens, whereas most healthy cells only express one or none. MP0533 binds with increasing avidity as the number of its target antigens present increases, thereby preferentially binding to AML cells over healthy cells. This unique mode of action is designed to enable T cell-mediated killing of AML cells while preserving a therapeutic window that minimizes damage to healthy cells.

Details of the poster presentation:

Title: Phase 1/2 study of MP0533, a tetra-specific T-cell engager (CD33 x CD123 x CD70 x CD3), in patients with relapsed/refractory AML or MDS/AML: Initial results from optimized treatment regimen including densified MP0533 dosing and adapted premedication
Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster II
Session Date: December 7, 2025
Presentation Time & Location: 6:00– 8:00 PM ET; OCCC, West Halls B3–B4
Publication Number: 3419

(Press release, Molecular Partners, DEC 7, 2025, View Source [SID1234661213])

On December 7, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported updated data from its ongoing Phase 1b study evaluating R2891, an oral prodrug of R835, a potent and selective dual inhibitor of interleukin receptor-associated kinases 1 and 4 (IRAK1/4), in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). The data are being presented today in an oral session by Dr. Guillermo Garcia-Manero at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 6-9, in Orlando, Florida and virtually.

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"New therapies are needed for patients with transfusion dependent lower-risk MDS. We’re pleased to share these updated study results, which underscore the potential of R289 to become a treatment option for these patients," said Lisa Rojkjaer, M.D., Rigel’s chief medical officer. "We look forward to concluding the dose expansion phase of the study and anticipate selection of the recommended Phase 2 dose for future clinical studies in the second half of 2026."

Rigel’s open-label Phase 1b study of R289 is evaluating the safety, tolerability, pharmacokinetics and preliminary efficacy in patients with R/R lower-risk MDS (NCT05308264). Enrollment in the dose escalation phase of the study was completed in July 2025. In October 2025, the first patient was dosed in the dose expansion phase where up to 40 patients will be randomized to either 500 mg once or twice daily to determine the recommended Phase 2 dose for future clinical trials.

Key highlights from the updated data as of October 28, 2025, include:

33 patients were enrolled, representing a difficult-to-treat population. The median age was 75. The median number of prior therapies was 3 (range: 1-8); 76% (25) of patients had received luspatercept, 73% (24) had received an erythropoiesis stimulating agent (ESA), 67% (22) had received an hypomethylating agent (HMA) and 6% (2) had received imetelstat. 61% (20) of patients were high transfusion burden (HTB) at baseline. 67% (22) of patients were ring sideroblast (RS) negative.
Median duration of treatment was 5.5 months (range: 0.9 – 27.7 months). R289 was generally well tolerated across all dose groups in this heavily pre-treated lower-risk MDS patient population, the majority of whom were HTB at baseline. The most common Grade 1/2 treatment-emergent adverse events (TEAEs) (in ≥18% of patients) were diarrhea (n=10, 30%), constipation and fatigue (each n=9, 27%), and creatinine increased and cough (each n=7, 21%). The most frequent Grade 3/4 TEAEs were anemia (n=6, 18%), neutrophil count decreased and pneumonia (each n=5, 15%), and alanine aminotransferase (ALT) increased and aspartate aminotransferase (AST) increased (each n=3, 9%). One (1) dose limiting toxicity (DLT) (Grade 4 AST increased/Grade 3 ALT increased) was reported in the 750 mg dose group.
For evaluable transfusion dependent (TD) patients (≥16 weeks follow up) at dose levels of at least 500 mg QD and higher, 6/18 (33%) patients achieved durable red blood cell transfusion independence (RBC-TI) of >8 weeks [500 mg QD (1/3), 750 mg QD (2/5), 500/250 mg QD (1/5), 500 mg BID (2/5)]. Duration of RBC-TI was >16 weeks in 4 patients and >24 weeks in 3 patients. The median time to onset of RBC-TI was 1.9 months and the median duration of RBC-TI was 22.9 weeks. Peak hemoglobin increases of 2.9 to 6.1 g/dL compared to baseline occurred in patients achieving RBC-TI.
Of the 6 patients achieving RBC-TI, 5 had received an HMA.
At doses ≥500 mg QD, steady state R835 plasma concentrations reached or exceeded those associated with 50-90% inhibition of lipopolysaccharide (LPS)-induced cytokine release previously observed in healthy volunteers.
Oral presentation details are as follows:

Sunday, December 7, 2025, 9:45am to 10:00am ET
Publication #: 489
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML
Presentation Title: An Update of Safety and Efficacy Results from a Phase 1b Study of R289, a Dual IRAK 1/4 Inhibitor, in Patients with Relapsed/Refractory (R/R) Lower Risk Myelodysplastic Syndrome (LR-MDS)
Presenter: Guillermo Garcia-Manero, M.D.

R289 was previously granted Orphan Drug designation for the treatment of myelodysplastic syndromes and granted Fast Track designation for the treatment of previously-treated transfusion dependent lower-risk MDS by the FDA.

About R289
R289 is a prodrug of R835, an IRAK1/4 dual inhibitor, which has been shown in preclinical studies to block inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to various inflammatory conditions. Chronic stimulation of both these receptor systems is thought to cause the pro-inflammatory environment in the bone marrow responsible for persistent cytopenias in lower-risk MDS patients.

(Press release, Rigel, DEC 7, 2025, View Source [SID1234661208])

On December 7, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported encouraging data from the Phase 1/2 LINKER-MM4 trial evaluating Lynozyfic (linvoseltamab) in adults with newly diagnosed multiple myeloma (NDMM) who were transplant eligible or ineligible were shared in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. These data build on results from a broad clinical development program evaluating Lynozyfic in early lines of treatment, including precursor conditions, as monotherapy and in combination with standard-of-care or novel agents.

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"The treatment of newly diagnosed multiple myeloma often relies on complicated combinations of quadruplet or triplet regimens, each with its own toxicities, in order to achieve rapid and durable responses, which can be incredibly burdensome for these patients," said Robert Orlowski, M.D., Ph.D., Deputy Chair, Professor of Medicine, and Director of Translational Myeloma Research in the Departments of Lymphoma/Myeloma and Experimental Therapeutics at The University of Texas MD Anderson Cancer Center and the lead investigator for the LINKER-MM4 trial. "As the first to evaluate a BCMAxCD3 bispecific monotherapy in this setting, LINKER-MM4 seeks to understand whether frontline intervention with a single agent can deliver strong efficacy, enabling the simplification and potentially greater tolerability of these regimens. Lynozyfic monotherapy is already achieving MRD negativity rates comparable to quadruplet regimens but earlier in the treatment course, and these compelling results are expected to deepen with longer follow up. These results underscore Lynozyfic’s potential as a foundational component of frontline treatment regimens for multiple myeloma – or even a monotherapy regimen – for both transplant-eligible and transplant-ineligible patients."

LINKER-MM4 is an ongoing, open-label Phase 1/2 trial investigating Lynozyfic in adults with NDMM. During a Phase 1A (dose escalation) cohort, patients were treated with a step-up dosing regimen followed by 50 mg, 100 mg or 200 mg doses of Lynozyfic. The lowest (50 mg) and highest (200 mg) tolerated doses were selected for further evaluation in the Phase 1B (dose-expansion) cohort. Among the 45 treated patients in both Phase 1A and 1B, 28 were transplant eligible, and 17 were transplant ineligible.

Across all dose levels (n=45), there was a 1.2 months median time to onset of response (range: 1-4.5 months). All three dose groups (50 mg, 100 mg and 200 mg) showed impressive efficacy, with a VGPR+ (very good partial response or better) of ≥70% with limited follow-up. Evidence shows that these responses are expected to deepen over time. Across all dose groups, 95% (19 of 20 patients) of all minimum residual disease (MRD) evaluable VGPR+ patients achieved MRD negative status at 10-5 sensitivity.

Across all dose levels, the most common treatment-emergent adverse events (TEAEs) were cytokine release syndrome (CRS; all Grade 1: 44%) and neutropenia (any Grade: 38%; Grade 3/4: 33%). Among other adverse events of special interest, one patient in the 50 mg cohort experienced Grade 1 immune effector cell-associated neurotoxicity syndrome (ICANS). Infections occurred in 84% of patients (Grade 1/2: 51%; Grade 3: 33%) with the majority occurring within the first three months of treatment and the rate of infections decreased over time. There were no ≥Grade 4 infections, Grade 5 TEAEs or dose-limiting toxicities. Ten patients elected to undergo an autologous stem cell transplant, all of whom had an acceptable CD34+ stem cell yield post-induction (range: 2.5-11.5 x 106/kg).

A broad clinical development program investigating Lynozyfic in early stages of the disease is underway. This includes the Phase 2 portion of the LINKER-MM4 trial evaluating Lynozyfic at the recommended 200 mg dose, as well as LINKER-MM6 (EMN39), a trial evaluating a combination of daratumumab, lenalidomide and dexamethasone (DRd) followed by Lynozyfic monotherapy compared with continued DRd in transplant-ineligible NDMM.

The use of Lynozyfic described above is investigational, and its safety and efficacy has not been evaluated by any regulatory authority for this indication.

About the ‘Regeneron Roundtable’ Investor Event
Regeneron will host a virtual investor event to discuss its multiple myeloma program on Wednesday, December 10 at 8:30 a.m. ET. This is the next webcast in a new investor event series called the ‘Regeneron Roundtable,’ intended to highlight programs from the company’s innovative investigational pipeline.
 
Links to the webcast and to register via telephone may be accessed from the ‘Investors and Media’ page of Regeneron’s website at View Source Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the company’s website for at least 30 days.

About Multiple Myeloma
As the second most common blood cancer, there are over 187,000 new cases of MM diagnosed globally every year, with more than 36,000 diagnosed and 12,000 deaths anticipated in the U.S. in 2025. The disease is characterized by the proliferation of cancerous plasma cells (MM cells) that crowd out healthy blood cells in the bone marrow, infiltrate other tissues and cause potentially life-threatening organ injury. Despite treatment advances, MM is not curable, and while current treatments are able to slow progression of the cancer, most patients will ultimately experience cancer progression and require additional therapies.

About Lynozyfic
Lynozyfic was invented using Regeneron’s VelocImmune technology and is a fully human BCMAxCD3 bispecific antibody designed to bridge B-cell maturation antigen (BCMA) on MM cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing. Lynozyfic is approved to treat certain adults with R/R MM: in the U.S. after four lines of therapy and in the European Union after at least three prior therapies.

In the U.S., the generic name for Lynozyfic in its approved indications is linvoseltamab-gcpt, with gcpt as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. FDA. Outside of the U.S., the generic name of Lynozyfic in its approved indications is linvoseltamab.

Lynozyfic is being investigated in a broad clinical development program exploring its use as a monotherapy as well as in combination regimens across different lines of therapy in MM, including earlier lines of treatment, as well as plasma cell precursor disorders. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority. 

In addition to LINKER-MM4, ongoing trials include:

LINKER-MM1: Phase 1/2 dose-escalation and dose-expansion trial evaluating the safety, tolerability, dose-limiting toxicities and anti-tumor activity of Lynozyfic monotherapy in R/R MM
LINKER-MM2: Phase 1b, open-label trial evaluating Lynozyfic in combination with other cancer treatments in patients with R/R MM
LINKER-MM3: Phase 3 confirmatory trial evaluating Lynozyfic monotherapy compared to the combination of elotuzumab, pomalidomide and dexamethasone in R/R MM
LINKER-MM5: Phase 3 trial evaluating Lynozyfic monotherapy or in combination with carfilzomib compared to standard of care combination regimens in patients with R/R MM
LINKER-MM6 (EMN39): Phase 3 trial, in collaboration with the European Myeloma Network, evaluating daratumumab, lenalidomide and dexamethasone induction followed by Lynozyfic monotherapy compared to continued daratumumab, lenalidomide, and dexamethasone in NDMM who are transplant-ineligible
Phase 1 trial evaluating Lynozyfic in combination with a Regeneron CD38xCD28 costimulatory bispecific in R/R MM
LINKER-SMM1: Phase 2 trial evaluating Lynozyfic monotherapy in high-risk smoldering MM
LINKER-MGUS1: Phase 2 dose-ranging trial evaluating Lynozyfic monotherapy in high-risk monoclonal gammopathy of unknown significance and non-high-risk SMM
LINKER-AL2: Phase 1/2 trial evaluating Lynozyfic monotherapy in R/R systemic light chain amyloidosis
For more information on Regeneron’s clinical trials in blood cancer, visit the clinical trials website, or contact via [email protected] or 844-734-6643.

IMPORTANT SAFETY INFORMATION FOR U.S. PATIENTS

What is the most important information I should know about LYNOZYFIC?
LYNOZYFIC may cause serious or life-threatening side effects, including Cytokine Release Syndrome (CRS) and infusion-related reactions (IRR), or neurologic problems.

Cytokine Release Syndrome (CRS) and infusion related reactions (IRR). CRS is common during treatment with LYNOZYFIC and can also be serious or life-threatening. Tell your healthcare provider or get medical help right away if you develop any signs or symptoms of CRS or IRR, including:

fever of 100.4ºF (38ºC) or higher
fast heartbeat
chills or shaking
dizziness or light-headedness
trouble breathing

Neurologic problems. LYNOZYFIC can cause neurologic problems that can be serious or life-threatening. Tell your healthcare provider or get medical help right away if you develop any signs or symptoms of neurologic problems, including:

headache
agitation, trouble staying awake, confusion or disorientation, seeing or hearing things that are not real (hallucinations)
trouble speaking, writing, thinking, remembering things, paying attention, or understanding things
problems walking, muscle weakness, shaking (tremors), loss of balance, or muscle spasms
numbness and tingling (feeling like "pins and needles")
burning, throbbing, or stabbing pain
changes in your handwriting
seizures
Due to the risk of CRS and neurologic problems, you will receive LYNOZYFIC on a "step-up dosing schedule" and should be hospitalized for 24 hours after the first and second "step-up" doses.

During the "step-up dosing schedule":
For your first dose, you will receive a smaller "step-up" dose of LYNOZYFIC on Day 1 of your treatment.
For your second dose, you will receive a larger "step-up" dose of LYNOZYFIC, which is usually given on Day 8 of your treatment.
For your third dose, you will receive the first treatment dose of LYNOZYFIC, which is usually given on Day 15 of your treatment.
Your healthcare provider may repeat one or both of the "step-up" doses depending on side effects or if your treatment is delayed.
Before the "step-up" doses and the first two treatment doses of LYNOZYFIC, you will receive medicines to help reduce your risk of CRS and IRR. Your healthcare provider will decide if you need to receive medicine to help reduce your risk of side effects with future doses.
LYNOZYFIC is available only through the LYNOZYFIC Risk Evaluation and Mitigation Strategy (REMS) due to the risk of side effects of CRS and neurologic problems. You will receive a Patient Wallet Card from your healthcare provider. Carry the LYNOZYFIC Patient Wallet Card with you at all times and show it to all of your healthcare providers. The LYNOZYFIC Patient Wallet Card lists signs and symptoms of CRS and neurologic problems. Get medical help right away if you develop any of the signs and symptoms listed on the LYNOZYFIC Patient Wallet Card. You may need to be treated in a hospital.

Your healthcare provider will monitor you for signs and symptoms of CRS and neurologic problems during treatment with LYNOZYFIC, as well as other side effects, and may treat you in a hospital if needed. Your healthcare provider may temporarily stop or completely stop your treatment with LYNOZYFIC if you develop CRS, neurologic problems, or any other severe side effects.

If you have any questions about LYNOZYFIC, ask your healthcare provider.

Before receiving LYNOZYFIC, tell your healthcare provider about all of your medical conditions, including if you:

have an infection.
are pregnant or plan to become pregnant. LYNOZYFIC may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with LYNOZYFIC.
Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with LYNOZYFIC.
You should use an effective form of birth control (contraception) during treatment with LYNOZYFIC and for 3 months after your last dose of LYNOZYFIC.
are breastfeeding or plan to breastfeed. It is not known whether LYNOZYFIC passes into your breast milk. Do not breastfeed during treatment with LYNOZYFIC and for 3 months after your last dose of LYNOZYFIC.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How will I receive LYNOZYFIC?

LYNOZYFIC will be given to you by your healthcare provider by infusion through a needle placed in a vein (intravenous infusion).
After the "step-up dosing schedule", the treatment dose of LYNOZYFIC is usually given 1 time each week for 11 doses, and then 1 time every other week for 5 doses. After these doses and based on how your disease responds, your healthcare provider will decide if you are able to receive LYNOZYFIC less often (every 4 weeks) or will continue to have every other week treatment.
Your healthcare provider will decide how long you will receive treatment with LYNOZYFIC.
If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. It is important for you to be monitored closely for side effects during treatment with LYNOZYFIC.
What should I avoid while receiving LYNOZYFIC?
Do not drive, or operate heavy or potentially dangerous machinery, or do other dangerous activities for 48 hours after completing each of your "step-up" doses or at any time during treatment with LYNOZYFIC if you develop new neurologic symptoms, until the symptoms go away.

What are the possible side effects of LYNOZYFIC?
LYNOZYFIC may cause serious side effects, including:

Infections. LYNOZYFIC can cause bacterial, viral, or fungal infections that are serious, life-threatening, or that may lead to death. Upper respiratory tract infections and pneumonia are common during treatment with LYNOZYFIC.
Your healthcare provider will monitor you for signs and symptoms of infection before and during treatment with LYNOZYFIC.
Your healthcare provider may prescribe medicines for you to help prevent infections and treat you as needed if you develop an infection during treatment with LYNOZYFIC.
Tell your healthcare provider right away if you develop any signs or symptoms of infection during treatment with LYNOZYFIC, including:
fever of 100.4 °F (38 °C) or higher
chills
cough
shortness of breath
chest pain
sore throat
pain during urination
feeling weak or generally unwell
Decreased white blood cell counts. Decreased white blood cell counts are common during treatment with LYNOZYFIC and can also be severe. Fever can happen with low white blood cell counts and may be a sign that you have an infection. Your healthcare provider will check your blood cell counts before you start treatment and during treatment with LYNOZYFIC, and will treat you as needed.
Liver problems. LYNOZYFIC can cause increased liver enzymes and bilirubin in your blood. These increases can happen with or without you also having CRS. Your healthcare provider will do blood tests to check your liver before starting and during treatment with LYNOZYFIC. Tell your healthcare provider if you develop any of the following signs or symptoms of liver problems:
tiredness
loss of appetite
pain in your right upper stomach-area (abdomen)
dark urine yellowing of your skin or the white part of your eyes
The most common side effects of LYNOZYFIC include:

muscle and bone pain
nausea
cough
headache
diarrhea
tiredness or weakness
shortness of breath
The most common severe abnormal blood test results with LYNOZYFIC include: low white blood cell counts and low red blood cell counts.

These are not all of the possible side effects of LYNOZYFIC.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see full Prescribing Information, including Boxed WARNING, and Medication Guide for LYNOZYFIC.

What is LYNOZYFIC?
LYNOZYFIC is a prescription medicine used to treat adults with multiple myeloma who:

have already received at least 4 treatment regimens, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody to treat their multiple myeloma, and
their cancer has come back or did not respond to prior treatment.
It is not known if LYNOZYFIC is safe and effective in children.

About Regeneron in Hematology
At Regeneron, we’re applying more than three decades of biology expertise with our proprietary VelociSuite technologies to develop medicines for patients with diverse blood cancers and rare blood disorders.

Our blood cancer research is focused on bispecific antibodies that are being investigated both as monotherapies and in various combinations and emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop customized and potentially synergistic cancer treatments.

Our research and collaborations to develop potential treatments for rare blood disorders include explorations in antibody medicine, gene editing and gene-knockout technologies, and investigational RNA-approaches focused on depleting abnormal proteins or blocking disease-causing cellular signaling.

(Press release, Regeneron, DEC 7, 2025, View Source [SID1234661207])