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Last update 09 Dec 2025
Carfilzomib
Last update 09 Dec 2025
Overview
Basic Info
Drug Type Synthetic peptide |
Synonyms Carfilzomib (JAN/USAN/INN), NSC-758252, ONO-7057 + [5] |
Target |
Action inhibitors |
Mechanism Proteasome inhibitors |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
License Organization |
Drug Highest PhaseApproved |
First Approval Date United States (20 Jul 2012), |
RegulationFast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Special Review Project (China), Conditional marketing approval (China), Orphan Drug (Japan) |
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Structure/Sequence
Molecular FormulaC40H57N5O7 |
InChIKeyBLMPQMFVWMYDKT-NZTKNTHTSA-N |
CAS Registry868540-17-4 |
Sequence Code 16182110
Related
270
Clinical Trials associated with CarfilzomibNCT07082270
Phase I Trial of GB1211 (Selvigaltin), an Oral Galectin-3 (Gal-3) Inhibitor, Combined With Standard of Care Treatment in Relapsed/Refractory Multiple Myeloma (RRMM)
This phase I trial studies the side effects and best dose of selvigaltin when given together with standard of care treatment (daratumumab-hyaluronidase, carfilzomib, dexamethasone) in treating patients with multiple myeloma that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). Selvigaltin works by blocking the activity of a protein called galectin-3. Galectin-3 is involved in various cellular processes, including inflammation and tissue scarring, which is associated with worse outcomes in several forms of cancer. By blocking the activity of galectin-3, selvigaltin may help reduce inflammation and tissue scarring. Daratumumab-hyaluronidase is a drug composed of daratumumab and hyaluronidase. Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells, including myeloma cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Hyaluronidase helps deliver the daratumumab to CD38-expressing cancer cells. Carfilzomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Giving selvigaltin with standard of care treatment may be safe, tolerable, and/or effective in treating patients with relapsed or refractory multiple myeloma.
Start Date01 Feb 2026 |
Sponsor / Collaborator |
NCT07222761
An Open-Label, Randomized Phase 3 Study of Linvoseltamab Monotherapy and Linvoseltamab Plus Carfilzomib Versus Standard of Care Combination Regimens in Patients With Relapsed/Refractory Multiple Myeloma
This study is researching a drug called linvoseltamab (also called "study drug") either given alone or in combination with another anti-myeloma drug called carfilzomib, compared to several standard treatments for progressive Multiple Myeloma (MM) after at least 1 but no more than 3 prior therapies.
The aim of this study is to see if the safety and efficacy of linvoseltamab alone or in combination with carfilzomib can deliver better outcomes (deeper and longer responses that help extend life) than standard treatment options.
The study is looking at several other research questions, including:
* What side effects may happen from taking the study drug
* How much study drug is in the blood at different times
* Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
The aim of this study is to see if the safety and efficacy of linvoseltamab alone or in combination with carfilzomib can deliver better outcomes (deeper and longer responses that help extend life) than standard treatment options.
The study is looking at several other research questions, including:
* What side effects may happen from taking the study drug
* How much study drug is in the blood at different times
* Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
Start Date29 Jan 2026 |
Sponsor / Collaborator |
NCT07227311
A Phase 2, Multicenter, Open Label, Non-randomized Study to Evaluate the Efficacy and Safety of Extended Dosing of Belantamab Mafodotin in Different Combinations With Standard of Care Regimens in Participants With Relapsed-refractory Multiple Myeloma (DREAMM-15)
This study is for adults with multiple myeloma (a type of blood cancer) that has come back after being treated earlier or isn't responding to the current treatment.
The main goal is to find out if the study drug, belantamab mafodotin, given less often (on an extended schedule) with other cancer medicines, can still treat the cancer effectively while causing fewer side effects, especially those affecting the eyes. The study will also look at how well the treatment works overall and how safe it is when administered to the participants.
The main goal is to find out if the study drug, belantamab mafodotin, given less often (on an extended schedule) with other cancer medicines, can still treat the cancer effectively while causing fewer side effects, especially those affecting the eyes. The study will also look at how well the treatment works overall and how safe it is when administered to the participants.
Start Date14 Jan 2026 |
Sponsor / Collaborator |
100 Clinical Results associated with Carfilzomib
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100 Translational Medicine associated with Carfilzomib
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100 Patents (Medical) associated with Carfilzomib
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2,377
Literatures (Medical) associated with Carfilzomib31 Dec 2025·Hematology
Pharmacovigilance and signal detection of adverse drug events associated with proteasome inhibitors in multiple myeloma: a real-world analysis using the FAERS database
Article
Author: Gao, Yao ; Zhang, Meiling ; Luo, JunYun ; Zeng, Yingjian ; Chen, Shupeng
BACKGROUND:
Proteasome inhibitors (PIs) such as Bortezomib, Carfilzomib, and Ixazomib have significantly improved outcomes in multiple myeloma (MM), but their real-world safety profiles require further exploration.
OBJECTIVE:
To assess adverse drug events (ADEs) associated with Bortezomib, Carfilzomib, and Ixazomib in MM patients using the FDA Adverse Event Reporting System (FAERS), and to identify new ADE signals not listed in product labels.
METHODS:
A total of 47,310 ADE reports (Bortezomib: 23,843; Carfilzomib: 9,835; Ixazomib: 13,632) were analyzed from FAERS (2004 - Q4 2024). Signal detection was conducted using Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma Poisson Shrinker (MGPS). Onset time was evaluated using Weibull survival analysis.
RESULTS:
All three PIs were associated with significant ADE signals across multiple system organ classes, notably in the hematologic, nervous, cardiovascular, hepatobiliary, and gastrointestinal systems. Newly identified ADEs included muscular weakness (Bortezomib), pancytopenia (Carfilzomib), and cognitive disorder (Ixazomib). Onset time analysis showed that most ADEs occurred within the first 30 days of treatment.
CONCLUSION:
This study highlights both known and newly detected ADEs linked to PIs, emphasizing the importance of early-phase monitoring and ongoing pharmacovigilance. The findings provide crucial real-world evidence for clinical risk management and future label updates.
01 Nov 2025·Blood neoplasia
Minimal residual disease measurement in blood by mass spectrometry identifies long-term responders in multiple myeloma
Article
Author: Derman, Benjamin A ; Kubicki, Tadeusz ; Cooperrider, Jennifer H ; Dytfeld, Dominik ; Jakubowiak, Andrzej J ; Barnidge, David ; Jiang, Ken ; Puła, Anna
Modern multiple myeloma treatment enables deep and sustained responses, necessitating assessment of minimal residual disease (MRD) in the bone marrow to refine response categorization. Recently, mass spectrometry (MS)-based methods have emerged as highly sensitive tools for measuring MRD in the peripheral blood. However, the role specific MS techniques play in response categorization has yet to be established. We pooled data from 97 patients treated in 3 prospective phase 2 trials evaluating carfilzomib-based triplets and quadruplets, with or without autologous stem cell transplantation. MRD was assessed in the bone marrow using next-generation sequencing (NGS) and in the peripheral blood with 2 MS methods: matrix-assisted laser desorption ionization-time of flight (EXENT) and the more sensitive liquid chromatography-MS (LC-MS). EXENT negativity was associated with superior progression-free survival (PFS) and overall survival. LC-MS negativity identified patients with long-term responses. EXENT complemented NGS MRD, with patients with double negativity experiencing longer PFS than those negative in only 1 modality. Patients negative by both LC-MS and NGS MRD at 10-6 had a 5-year PFS rate of 89%. These findings support incorporating MS into MRD response assessment and in prognostic algorithms in myeloma. In addition, our results indicate that LC-MS can provide valuable end point in future studies aiming for functional cure.
01 Nov 2025·MOLECULAR CARCINOGENESIS
Noncanonical Pathways of Proteasome Inhibition in HPV‐Negative Head & Neck Cancer
Article
Author: Wang, Zhiyong ; Amornphimoltham, Panomwat ; Gutkind, J. Silvio ; Lee, Hye‐Yeon ; Jeong, Woo‐Jin ; Kim, Ji‐Yeong
ABSTRACT:
Carfilzomib, a second‐generation proteasome inhibitor approved for the treatment of multiple myeloma, is a highly potent and selective inhibitor of the CT‐L activity of the i20S proteasome. Several studies have shown that carfilzomib (CFZ) can bypass resistance to bortezomib; however, its impact on squamous cell carcinoma of the head and neck is not well understood. This study aimed to evaluate the anticancer potential of CFZ in head and neck cancer cells (HNSCC) by examining its effects on proliferation, apoptosis, and the underlying mechanisms in both HPV‐positive and HPV‐negative HNSCC. In Vitro validation of CFZ showed an IC50 that was more than fourfold lower in HPV‐negative CAL‐27 than other HNSCC cell lines. In addition, CFZ inhibited p‐Akt and p‐S6 and activated p21, which increased growth inhibition and apoptosis in CAL‐27 cells. In mice bearing xenografted HPV‐negative CAL‐27 cells, we confirmed that CFZ reduced tumor growth. Collectively, the cytotoxic effects induced by CFZ involve cell growth inhibition and apoptosis via the PI3K/AKT/mTOR and p21 signaling pathways. This suggests that CFZ is a novel therapeutic agent that can overcome the existing cisplatin resistance used in the treatment of HPV‐negative HNSCC.
256
News (Medical) associated with Carfilzomib07 Dec 2025
All three dose groups (50 mg, 100 mg and 200 mg) showed impressive monotherapy efficacy, with VGPR+ (very good partial response or better) of ≥70% despite 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 evaluable VGPR+ patients achieved minimal residual disease negative status
Data featured in an ASH oral presentation; LINKER-MM4 is the first clinical trial to evaluate a BCMAxCD3 bispecific monotherapy in NDMM and is part of a broad clinical development program evaluating Lynozyfic-based regimens in earlier lines of treatment
Regeneron to host virtual ‘Regeneron Roundtable’ investor event to discuss its multiple myeloma development program on Wednesday, December 10 at 8:30 a.m. ET
TARRYTOWN, N.Y. , Dec. 07, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced 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) 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.
“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 https://investor.regeneron.com/events-and-presentations. 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 MyelomaAs 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 LynozyficLynozyfic 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 clinicaltrials@regeneron.com 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.
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.
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
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
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
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.
About Regeneron 's VelocImmune Technology Regeneron 's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes Dupixent® (dupilumab), Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb), Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz® (pozelimab-bbfg). In addition, REGEN-COV® (casirivimab and imdevimab) had been authorized by the FDA during the COVID-19 pandemic until 2024. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases.
Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.
For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X.Forward-Looking Statements and Use of Digital Media This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation Lynozyfic™ (linvoseltamab-gcpt); the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products, such as Lynozyfic as a monotherapy and/or in combination with standard-of-care agents across different lines of therapy in multiple myeloma (“MM”) and plasma cell precursor disorders, including the treatment of adults with newly diagnosed MM as discussed in this press release; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products (such as Lynozyfic) and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates and risks associated with tariffs and other trade restrictions; safety issues resulting from the administration of Regeneron’s Products (such as Lynozyfic) and Regeneron’s Product Candidates in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement or copay assistance for Regeneron’s Products from third-party payors and other third parties, including private payor healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payors and other third parties and new policies and procedures adopted by such payors and other third parties; changes to drug pricing regulations and requirements and Regeneron’s pricing strategy; other changes in laws, regulations, and policies affecting the healthcare industry; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates (including biosimilar versions of Regeneron’s Products); the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics on Regeneron 's business; and risks associated with litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts ), risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2024 and its Form 10-Q for the quarterly period ended September 30, 2025 . Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron . Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise.
Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron 's media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals).
Source: Regeneron Pharmaceuticals, Inc.
Clinical ResultPhase 3Phase 2Drug ApprovalPhase 1
20 Nov 2025
SAN CARLOS, Calif.--(BUSINESS WIRE)--BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, advances its vision to become the world’s leading oncology company with extensive new data from its differentiated hematology portfolio at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida, December 6-9. Nearly 50 abstracts have been accepted, including six oral presentations, featuring the company’s three transformative approved and investigational hematology assets – BTK inhibitor BRUKINSA® (zanubrutinib), BCL2 inhibitor sonrotoclax, and BTK degrader BGB-16673.
Key presentations include:
SEQUOIA: BRUKINSA demonstrated sustained overall survival (84%; 88% after COVID adjustment) and landmark progression-free survival (PFS) superiority vs bendamustine + rituximab with an estimated 74% PFS at 6 years in treatment-naïve chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) (Poster Presentation: 2129)
ALPINE: Post-hoc analysis from Phase 3 study of BRUKINSA versus ibrutinib in patients with relapsed/refractory (R/R) CLL/SLL, using longitudinal patient-reported outcomes (PRO) (Oral Presentation: 711)
BGB-11417-201: Phase 1/2 study of sonrotoclax in patients with R/R mantle cell lymphoma (MCL) previously treated with a BTK inhibitor (Oral Presentation: 663)
BGB-11417-101: Updated safety and efficacy results, including undetectable minimal residual disease (uMRD) data, from ongoing Phase 1/1b study of sonrotoclax plus BRUKINSA in treatment-naïve CLL/SLL (Poster Presentation: 3891)
CaDAnCe-101: Updated efficacy and safety results of BGB-16673 in patients with R/R CLL/SLL and R/R Waldenström macroglobulinemia (WM) (Oral Presentation: 85; Poster Presentation: 3583)
“Our data at ASH 2025 raises the bar for patient and physician expectations of what’s possible. Our long-term data drives confidence in duration of response in CLL treatment decisions,” said Lai Wang, Ph.D., Global Head of R&D at BeOne. “Six-year SEQUOIA and long-term extension data from patients originally enrolled in ALPINE cement BRUKINSA’s role as the backbone of CLL therapy, and our three innovative B-cell treatment modalities of BTK inhibition, BCL2 inhibition, and BTK degradation have the potential to advance options that evolve with patient needs across all stages of disease.”
Additional highlights include:
Never-before-presented clinical data from BeOne’s emerging pipeline will also be shared at the meeting, including in new combinations and disease areas.
BGB-11417-101: Results from Phase 1/1b study: MRD-guided therapy of sonrotoclax plus obinutuzumab in patients with treatment-naïve CLL/SLL (Oral Presentation: 793) Initial results of treatment with sonrotoclax plus BRUKINSA plus obinutuzumab in patients with treatment-naïve CLL/SLL (Poster Presentation: 3890)
MRD-guided therapy of sonrotoclax plus obinutuzumab in patients with treatment-naïve CLL/SLL (Oral Presentation: 793)
Initial results of treatment with sonrotoclax plus BRUKINSA plus obinutuzumab in patients with treatment-naïve CLL/SLL (Poster Presentation: 3890)
BGB-11417-202: Phase 2 study of sonrotoclax monotherapy in patients with R/R CLL/SLL (Poster Presentation: 5666)
BGB-11417-105: Initial results from Phase 1b/2 study of sonrotoclax plus carfilzomib and dexamethasone in patients with t(11;14)-positive R/R multiple myeloma (Oral Presentation: 102)
CaDAnCe-101 Preliminary results from the ongoing Phase 1 study of BGB-16673 in patients with R/R Richter’s transformation (Poster Presentation: 3895)
Ongoing clinical data from BRUKINSA continue to demonstrate clinically meaningful benefit for patients with CLL/SLL.
SEQUOIA Arm D: Single-arm study of BRUKINSA plus venetoclax in patients with first-line CLL/SLL, with del(17p) and/or TP53 mutation or without both (Poster Presentation: 5669)
ALPINE thru LTE1: Up to 6 years of follow-up of patients with R/R CLL/SLL who were originally randomized to receive BRUKINSA as part of the ALPINE study and continued BRUKINSA treatment in a long-term extension study (LTE-1) (Poster Presentation: 2123)
Presentations also include data leveraging real-world evidence and validated modeling approaches to refine understanding of real-world experience and outcomes achieved with covalent BTK inhibitors.
A model analysis of number needed to treat (NNT) estimates that treating patients with BRUKINSA instead of ibrutinib for CLL could potentially prevent approximately 255 cardiac deaths in the second-line or later (2L+) setting and 266 in the first-line (1L) setting over a 10-year period. (Abstract Number: 13636)
Model evaluating BRUKINSA vs other covalent BTK inhibitors in R/R CLL and the number of patients needed to treat to avoid progression or death (Poster Presentation: 4553)
Observational study examining patient-reported outcomes in U.S. patients with CLL/SLL and treated with BRUKINSA or acalabrutinib in the community oncology setting (Poster Presentation: 2768)
“In CLL, selecting the right therapy for the right patient at the right time is essential, and continuous treatment with BTK inhibitors like BRUKINSA has become central to achieving enduring disease control,” said Dany Habr, M.D., Senior Vice President and Head of Medical Affairs, North America & International Markets at BeOne. “Emerging data from real-world settings suggest that BRUKINSA may offer a more manageable side effect profile, including for symptoms such as fatigue, pain, headache – further supporting its role as the BTKi of choice.”
BeOne Presentations at ASH 2025 (organized chronologically by asset)
BRUKINSA: The backbone of the hematology franchise
Abstract Title
Presentation Details
Final analysis of the randomized Phase 2 ROSEWOOD study of zanubrutinib + obinutuzumab vs obinutuzumab monotherapy in patients with R/R follicular lymphoma
Oral Presentation: 227
Session Title: Mantle Cell, Follicular, Waldenstrom's, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: FL and WM
Session Date/Time: December 6, 2025, 2:00-3:30 PM EST
Pier Luigi Zinzani
Sustained efficacy of zanubrutinib vs bendamustine + rituximab in treatment- naïve CLL/SLL with continued favorable survival in non-randomized patients with del(17p): 6-year follow-up in the Phase 3 SEQUOIA study
Poster Presentation: 2129
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Constantine S. Tam
Long-term results of patients receiving zanubrutinib in the Phase 3 ALPINE study confirm sustained benefit of zanubrutinib in patients with R/R CLL/SLL: Up to 6 years of follow-up with the long-term extension
Poster Presentation: 2123
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Constantine S. Tam
Symptom-based progression-free survival as a clinically relevant and patient-centric endpoint in CLL/SLL: Results from the ALPINE trial
Oral Presentation: 711
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Quality of Life and Supportive Care in Lymphoid Malignancies
Session Date/Time: December 7, 2025, 4:30-6:00 PM EST
Jennifer R. Brown
Progression-free survival in patients with low health-related quality of life treated with zanubrutinib versus ibrutinib monotherapy: Post-hoc analysis of the ALPINE trial
Poster Presentation: 6275
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Quality of Life and Supportive Care in Lymphoid Malignancies
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Loic Ysebaert
Zanubrutinib + venetoclax for treatment-naïve CLL/SLL, including patients with del(17p) and/or TP53 mutation and unmutated immunoglobulin heavy-chain variable status: 3-year results from SEQUOIA arm D
Poster Presentation: 5669
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Mazyar Shadman
Evaluation of factors from established prognostic models in patients with CLL treated with zanubrutinib: A post-hoc analysis of two Phase 3 studies (SEQUOIA and ALPINE)
Poster Presentation: 5681
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Inhye Ahn
Zanubrutinib is well tolerated and effective in acalabrutinib-intolerant patients with B-cell malignancies: A long-term follow-up
Poster Presentation: 5663
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Mazyar Shadman
Single-arm, open-label, multicenter study of the BTKi zanubrutinib in patients with CD79B-mutated R/R diffuse large B-cell lymphoma
Poster Presentation: 3684
Session Title: Aggressive Lymphomas: Targeted and Pharmacologic Therapies: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Li Wang
Sonrotoclax: Potential best-in-class next-generation BCL2 inhibitor
Abstract Title
Presentation Details
Lead Author
Initial Phase 1b/2 study results with sonrotoclax in combination with carfilzomib and dexamethasone in patients with t(11;14)-positive R/R multiple myeloma
Oral Presentation: 102
Session Title: Multiple Myeloma: Pharmacologic Therapies: Advancing the Standard: Improving Myeloma Treatment through Diagnosis, Maintenance and Relapse
Session Date/Time: December 6, 2025, 10:45-11:00 AM EST
Hang Quach
A Phase 3, randomized, open-label, multicenter study of sonrotoclax plus anti-CD20 antibody therapies vs venetoclax plus rituximab in patients with R/R CLL/SLL (CLL-RR1/CELESTIAL-RRCLL)
Trial-in-progress Poster Presentation: 2137
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Othman Al-Sawaf
Sonrotoclax monotherapy in patients with relapsed/refractory mantle cell lymphoma previously treated with BTKi: Early results from a Phase 1/2 study
Oral Presentation: 663
Session Title: Mantle Cell, Follicular, Waldenstrom's, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological - Novel Treatments for and Insights into Mantle Cell Lymphoma
Session Date/Time: December 7, 2025, 4:30-6:00 PM EST
Michael Wang
Frontline treatment of sonrotoclax and zanubrutinib for CLL/SLL demonstrates high undetectable minimal residual disease rates with favorable tolerability: Updated data from BGB-11417-101, an ongoing Phase 1/1b study
Poster Presentation: 3891
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Constantine S. Tam
Zanubrutinib + obinutuzumab + sonrotoclax in patients with treatment-naïve CLL/SLL: Initial results from an ongoing Phase 1/1b study, BGB-11417-101
Poster Presentation: 3890
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Jacob D. Soumerai
MRD-guided therapy of sonrotoclax + obinutuzumab in patients with treatment-naive CLL: Initial results from an ongoing Phase 1/1b study, BGB-11417-101
Oral Presentation: 793
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: MRD Guided Therapy and Emergence of Resistance
Session Date/Time: December 8, 2025, 10:30 AM-12:00 PM EST
Marc S. Hoffmann
Primary analysis of a multicenter, open-label, Phase 2 study of sonrotoclax monotherapy in patients with R/R CLL/SLL
Poster Presentation: 5666
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Shuhua Yi
BGB-16673: Potential first-in-class BTK protein degrader
Abstract Title
Presentation Details
Lead Author
Updated efficacy and safety results of the BTK degrader BGB-16673 in patients with R/R CLL/SLL from the ongoing Phase 1 CaDAnCe-101 study
Oral Presentation: 85
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Treatment of CLL in Relapse and in Richter Transformation
Session Date/Time: December 6, 2025, 9:30-11:00 AM EST
Inhye E. Ahn
CaDAnCe-104, an ongoing, open-label, Phase 1b/2 master protocol study of BTK degrader BGB-16673 in combination with other agents in patients with R/R B-cell malignancies
Trial-in-Progress Poster Presentation: 1839
Session Title: Mantle Cell, Follicular, Waldenstrom's, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Chan Y. Cheah
Updated efficacy and safety results of the BTK degrader BGB-16673 in patients with R/R indolent non-Hodgkin lymphoma from the ongoing Phase 1 CaDAnCe-101 study
Poster Presentation: 3584
Session Title: Mantle Cell, Follicular, Waldenstrom's, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Romain Guièze
Preliminary efficacy and safety of the BTK BGB-16673 in patients with R/R Richter transformation: Results from the ongoing Phase 1 CaDAnCe-101 study
Poster Presentation: 3895
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Meghan C. Thompson
Updated efficacy and safety results of the BTK BGB-16673 in patients with R/R Waldenström macroglobulinemia from the ongoing Phase 1 CaDAnCe-101 study
Poster Presentation: 3583
Session Title: Mantle Cell, Follicular, Waldenstrom's, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Constantine S. Tam
CaDAnCe-304, a Phase 3, open-label, randomized study to evaluate the safety and efficacy of BTK degrader BGB-16673 compared with pirtobrutinib in patients with R/R CLL/SLL
Trial-in-progress Poster Presentation: 5691
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Meghan C. Thompson
Other hematology assets: BGB-21447, next-generation BCL2 inhibitor
Abstract Title
Presentation Details
Lead Author
Preliminary results from a Phase 1/1b first-in-human study of BGB-21447, a next-generation BCL2 inhibitor, in patients with B-cell non-Hodgkin lymphoma
Poster Presentation: 1910
Session Title: Aggressive Lymphomas: Targeted and Pharmacologic Therapies: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Fei Li
Integrative evidence generation and health economics related to BRUKINSA
Abstract Title
Presentation Details
Lead Author
Matching-Adjusted Indirect Comparison
Indirect comparison of efficacy of zanubrutinib vs ibrutinib for the treatment of R/R MCL
Poster Presentation: 5365
Session Title: Mantle Cell, Follicular, Waldenstrom's, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Toby A. Eyre
A matching-adjusted indirect comparison of zanubrutinib vs venetoclax + ibrutinib in treatment-naive CLL
Abstract Number: 7756
Talha Munir
Health Economic and Outcomes Research
Outcomes during BTKi treatment for CLL: Insights from remote therapeutic monitoring
Poster Presentation: 2768
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Gurjyot Doshi
Number needed to treat to avoid progression or death: Zanubrutinib vs other covalent BTKis in R/R CLL
Poster Presentation: 4553
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Mazyar Shadman
Estimated cardiac deaths associated with treating CLL with ibrutinib versus zanubrutinib in the United States
Abstract Number: 13636
Jennifer R. Brown
Number of patients needed to treat to prevent one atrial fibrillation event with zanubrutinib versus ibrutinib and acalabrutinib in B-cell malignancies
Abstract Number: 14445
Talha Munir
Number of cardiac deaths associated with ibrutinib versus zanubrutinib for the treatment of CLL: A European risk-based estimation
Abstract Number: 14028
Talha Munir
Real-World Evidence
Mediators of racial and ethnic inequities in access to front-line therapies for CLL in the United States: A real-world evidence study
Poster Presentation: 2720
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Jacqueline C. Barrientos
Real-world treatment patterns and biomarker utilization among patients aged ≥65 years with CLL/SLL from 2020 to 2024
Poster Presentation: 2723
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Paul Hampel
Chemo ± immunotherapy remains utilized for CLL in the real-world practice: Unmet needs, treatment patterns, and age disparities in the United States
Poster Presentation: 2762
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster I
Session Date/Time: December 6, 2025, 5:30-7:30 PM EST
Javier Pinilla-Ibarz
Impact of testing for genetic markers on treatment selection and clinical outcomes among patients with CLL
Poster Presentation: 3894
Session Title: Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Brian Koffman
Treatment patterns and outcomes among patients treated with second-generation BTK inhibitors in CLL
Poster Presentation: 4528
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Aryan Ayat
Changes in real-world treatment patterns over time by patient characteristics and time burden of treatment in CLL/SLL
Poster Presentation: 6283
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Mengyang Di
Real-world treatment patterns and patient characteristics of venetoclax combination time-limited therapy for CLL
Poster Presentation: 6317
Session Title: Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster III
Session Date/Time: December 8, 2025, 6:00-8:00 PM EST
Jing-Zhou Hou
Real-world treatment utilization, sequencing, and outcomes in mantle cell lymphoma: Emerging treatment patterns in the United States
Abstract Number: 13378
Alvaro Alencar
Real-world zanubrutinib treatment patterns in CLL/SLL among a curated sample of US community oncology patients with prior acalabrutinib therapy
Abstract Number: 8798
Jing-Zhou Hou
Incidence of cardiac-related deaths among patients aged ≥65 years with B-cell malignancies treated with ibrutinib
Abstract Number: 7341
Ryan Jacobs
Real-world CLL-specific biomarker testing patterns and frontline treatment patterns in patients with CLL/small lymphocytic lymphoma
Abstract Number: 7773
Timothy Reynolds
Real-world CLL/SLL treatment patterns at Florida Cancer Specialists & Research Institute among patients receiving zanubrutinib immediately following prior BTKi therapy
Abstract Number: 13894
Amanda Warner
Patient Preference
Patient preferences and factors affecting patient treatment decisions for CLL in Japan
Poster Presentation: 4406
Session Title: Health Services and Quality Improvement: Lymphoid Malignancies: Poster II
Session Date/Time: December 7, 2025, 6:00-8:00 PM EST
Sikander Ailawadhi
Evaluating patient preferences for CLL in Korea: A discrete choice experiment
Abstract Number: 4019
Byung Woo Yoon
Systemic Literature Review
Patients with high-risk features in mantle cell lymphoma: A systematic literature review of clinical trials and real-world studies
Abstract Number: 14130
Christine E. Ryan
For additional information about our presence at the 2025 ASH Annual Meeting and Exposition, please visit our meeting hub: congress.beonemedicines.com.
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. Studies in the lab and 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 undetectable minimal residual disease (uMRD) rates in treatment-naïve patients with CLL. To date, more than 2,200 patients have been enrolled across the broad global development program.
The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for sonrotoclax for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL). In addition, the FDA has granted sonrotoclax Fast Track Designation for MCL and Waldenström macroglobulinemia, as well as Orphan Drug Designation for the treatment of adult patients with MCL, WM, multiple myeloma, acute myeloid leukemia, and myelodysplastic syndrome.
About BGB-16673
BGB-16673 is a potential first-in-class Bruton’s tyrosine kinase (BTK) protein degrader and is the most advanced protein degrader in the clinic, with nearly 800 patients dosed to date in an extensive global clinical development program. This program includes three randomized Phase 3 trials in R/R CLL, including the head-to-head Phase 3 trial versus pirtobrutinib which began enrolling in Q4 2025. Originating from BeOne’s chimeric degradation activation compound (CDAC) platform, BGB-16673 is designed to promote the degradation, or breakdown, of both wildtype and mutant forms of BTK, including those that commonly result in resistance to BTK inhibitors in patients who experience progressive disease.
The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to BGB-16673 for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and adult patients with R/R mantle cell lymphoma (MCL). Additionally, the European Medicines Agency (EMA) granted BGB-16673 PRIority MEdicines (PRIME) designation for the treatment of patients with Waldenstrom’s macroglobulinemia (WM) previously treated with a BTK inhibitor.
About BRUKINSA® (zanubrutinib)
BRUKINSA is an orally available, small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.
BRUKINSA has the broadest label globally of any BTK inhibitor and is the only BTK inhibitor to provide the flexibility of once or twice daily dosing. BRUKINSA is also the only BTK inhibitor to demonstrate superiority to another BTK inhibitor in a Phase 3 study.
The global BRUKINSA clinical development program includes about 7,100 patients enrolled in 30 countries and regions across more than 35 trials. BRUKINSA is approved in more than 75 markets, and more than 247,000 patients have been treated globally.
Select Important Safety Information
Serious adverse reactions, including fatal events, have occurred with BRUKINSA, including hemorrhage, infections, cytopenias, second primary malignancies, cardiac arrhythmias, and hepatotoxicity (including drug-induced liver injury).
In the pooled safety population (N=1729), the most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received BRUKINSA were neutrophil count decreased (51%), platelet count decreased (41%), upper respiratory tract infection (38%), hemorrhage (32%), and musculoskeletal pain (31%).
Please see full U.S. Prescribing Information including U.S. Patient Information.
The information provided in this press release is intended for a global audience. Product indications vary by region.
About BeOne
BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more accessible to cancer patients worldwide. With a portfolio spanning hematology and solid tumors, BeOne is expediting development of its diverse pipeline of novel therapeutics through its internal capabilities and collaborations. With a growing global team of nearly 12,000 colleagues spanning six continents, the Company is committed to radically improving access to medicines for far more patients who need them.
To learn more about BeOne, please visit www.beonemedicines.com and follow us on LinkedIn, X, Facebook and Instagram.
Forward-Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the potential benefits of BeOne’s product and product candidates; bringing innovative and transformative medicines to patients; and BeOne’s plans, commitments, aspirations and goals under the caption “About BeOne.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeOne’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeOne’s ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeOne's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeOne’s reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeOne’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products; BeOne’s ability to obtain additional funding for operations and to complete the development of its drug candidates and achieve and maintain profitability; and those risks more fully discussed in the section entitled “Risk Factors” in BeOne’s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeOne’s subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeOne undertakes no duty to update such information unless required by law.
To access BeOne media resources, please visit our Newsroom.
Clinical ResultPhase 1Phase 3Phase 2ASH
06 Nov 2025
Phase 3 AQUILA study showed DARZALEX FASPRO® significantly reduced the risk of progression to active multiple myeloma or death by 51 percent compared to active monitoring
Landmark approval supports earlier intervention and disease interception of multiple myeloma for the first time
HORSHAM, Pa., Nov. 6, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) today announced the U.S. Food and Drug Administration (FDA) approved DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) as a single agent treatment for adult patients with high-risk smoldering multiple myeloma (HR-SMM).1 DARZALEX FASPRO® is the first and only approved treatment for HR-SMM, enabling earlier intervention before the disease progresses to active multiple myeloma.
FDA approval is based on findings from the AQUILA study (NCT03301220), which evaluated the efficacy and safety of DARZALEX FASPRO® compared to active monitoring (or "Watch and Wait") in the largest Phase 3 trial in patients with HR-SMM. The AQUILA study demonstrated a significant improvement in the primary endpoint of progression-free survival (PFS), with DARZALEX FASPRO® reducing the risk of disease progression to active multiple myeloma or death by 51 percent compared to active monitoring, according to the International Myeloma Working Group (IMWG) diagnostic criteria for multiple myeloma. Today's milestone follows the May 2025 vote by the U.S. FDA Oncologic Drugs Advisory Committee (ODAC) in favor of the benefit-risk profile of DARZALEX FASPRO® as a single agent treatment for patients with HR-SMM.
Smoldering multiple myeloma (SMM) is an asymptomatic malignancy that is genomically the same as active multiple myeloma and where these abnormal cells can be detected in the bone marrow.2,3,4 In 2025, it is estimated that more than 36,000 people will be diagnosed with multiple myeloma in the U.S., and approximately 15 percent of those are classified as smoldering.5,6 An estimated 50 percent of patients diagnosed with HR-SMM are likely to progress to active disease within two years of diagnosis.6 Currently, the standard of care for HR-SMM is active monitoring to track signs of biochemical progression and/or end-organ damage. Recent evidence suggests that people at high-risk of progressing to active multiple myeloma could benefit from earlier therapeutic intervention.6
"Until now, patients diagnosed with smoldering multiple myeloma only have the option to watch and wait for any active signs of progression to active disease," said Peter Voorhees, M.D., Atrium Health/Levine Cancer Institute, Charlotte, N.C.* "Results from AQUILA demonstrated DARZALEX FASPRO significantly delayed disease progression, underscoring the role of early disease intervention for patients with high-risk smoldering multiple myeloma."
The Phase 3 AQUILA study showed after a median follow-up of 65.2 months, 63.1 percent of patients who received DARZALEX FASPRO® had not progressed to active myeloma at 5 years (60 months) versus 40.7 percent in the active monitoring group (hazard ratio [HR], 0.49; 95 percent confidence interval [CI], 0.36-0.67; P<0.001). Today, most physicians use the Mayo 2018 criteria (20/2/20) to assess risk status in patients with smoldering myeloma. In a post hoc analysis of AQUILA, 41 percent of patients met the Mayo 2018 HR-SMM classification. Among these patients, median PFS was not reached in the DARZALEX FASPRO® arm and was 22.1 months in the active monitoring arm (HR, 0.36; 95 percent CI, 0.23-0.58).1
Beyond the primary endpoint of PFS, patients in AQUILA who received DARZALEX FASPRO® saw a higher response rate of 63.4 percent compared to 2.0 percent with active monitoring (P<0.001). The median time to patients receiving first-line multiple myeloma treatment was delayed for patients receiving DARZALEX FASPRO® compared to active monitoring, with median time to first treatment NR vs 50.2 months for the active monitoring group (HR, 0.46; 95 percent CI, 0.33-0.62).1
"DARZALEX FASPRO is a foundational therapy in multiple myeloma and illustrates our commitment to improve outcomes for patients at every stage of their disease," said Jordan Schecter, M.D., Vice President, Research & Development, Multiple Myeloma, Oncology, Johnson & Johnson Innovative Medicine. "Data from the AQUILA study reinforce the significant impact DARZALEX FASPRO continues to have for patients. With today's approval, patients with HR-SMM will now be able to receive this treatment before they progress to active multiple myeloma, giving us the opportunity to shift the treatment paradigm and bring hope to people who are impacted by this disease."
Adverse reactions observed in the pivotal AQUILA study were generally consistent with previous DARZALEX FASPRO® studies. The most common adverse reactions (≥20%) in patients with HR-SMM who received DARZALEX FASPRO® monotherapy are upper respiratory tract infection, musculoskeletal pain, fatigue, diarrhea, rash, sleep disorder, sensory neuropathy, and injection site reactions.1
Results from AQUILA were first presented at the 2024 American Society of Hematology (ASH) Annual Meeting and simultaneously published in The New England Journal of Medicine. A subgroup analysis from the AQUILA study, evaluating the efficacy and safety of DARZALEX FASPRO® monotherapy in patients with HR-SMM using IMWG 2020 and IMWG 2020 plus cytogenetic risk models, will be presented at the 2025 ASH Annual Meeting in Orlando from December 6-9.
About the AQUILA Study
AQUILA (NCT03301220) is a randomized, multicenter Phase 3 study comparing treatment with DARZALEX FASPRO® to active monitoring in patients with SMM. Patients received single agent DARZALEX FASPRO® as a fixed-duration treatment for up to 36 months. The primary endpoint is progression-free survival (PFS), defined as progression to active multiple myeloma (MM) as assessed by an independent review committee, according to IMWG diagnostic criteria for MM (SLiM-CRAB), or death. Major secondary endpoints included overall response rate, PFS on first-line MM treatment (PFS2), and overall survival. Forty-one percent of patients had 2 or more of the following criteria for high-risk smoldering multiple myeloma: serum monoclonal protein level >2 g/dL, involved-to-uninvolved serum-free light chain ratio >20, and bone marrow plasma cells >20%. DARZALEX FASPRO® is only indicated for patients with high-risk smoldering multiple myeloma and is not indicated for other risk categories.
About Multiple Myeloma
Multiple myeloma is a blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.7 In multiple myeloma, these malignant plasma cells proliferate and replace normal cells in the bone marrow.8 Multiple myeloma is the second most common blood cancer worldwide and remains an incurable disease.9 In 2025, it is estimated that more than 36,000 people will be diagnosed with multiple myeloma in the U.S. and more than 12,000 will die from the disease.5 People with multiple myeloma have a 5-year survival rate of 59.8 percent.10 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.2,10
About Smoldering Multiple Myeloma
Smoldering multiple myeloma (SMM) is an asymptomatic precursor disease state of multiple myeloma where abnormal cells can be detected in the bone marrow.11 People living with SMM do not show signs or symptoms typically associated with active myeloma, such as bone pain, bone fractures, kidney problems, or anemia. However, as abnormal plasma cells are present, organ damage may begin and progress asymptomatically.4,6 Approximately fifteen percent of all cases are classified as SMM, and half of those diagnosed with high-risk SMM are estimated to progress to active multiple myeloma within two years.6
About DARZALEX FASPRO® and DARZALEX®
DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) received U.S. FDA approval in May 2020 and is approved for ten indications in multiple myeloma, four of which are for frontline treatment in newly diagnosed patients who are transplant eligible or ineligible.1 It is the only subcutaneous CD38-directed antibody approved to treat patients with multiple myeloma. DARZALEX FASPRO® is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology.
DARZALEX® (daratumumab) received U.S. FDA approval in November 2015 and is approved in eight indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant-eligible and ineligible.12 DARZALEX® is the first CD38-directed antibody approved to treat multiple myeloma.12 DARZALEX®-based regimens have been used in the treatment of more than 618,000 patients worldwide and more than 68,000 patients in the U.S. alone.
In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab
For more information, visit .
DARZALEX FASPRO® INDICATIONS AND IMPORTANT SAFETY INFORMATION
INDICATIONS
DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) is indicated for the treatment of adult patients with multiple myeloma:
In combination with bortezomib, lenalidomide, and dexamethasone for induction and consolidation in newly diagnosed patients who are eligible for autologous stem cell transplant
In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant
In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy
In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant
In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor (PI)
In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy
In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy
As monotherapy in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double refractory to a PI and an immunomodulatory agent
DARZALEX FASPRO® as monotherapy is indicated for the treatment of adult patients with high-risk smoldering multiple myeloma.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
DARZALEX FASPRO® is contraindicated in patients with a history of severe hypersensitivity to daratumumab, hyaluronidase, or any of the components of the formulation.
WARNINGS AND PRECAUTIONS
Hypersensitivity and Other Administration Reactions
Both systemic administration-related reactions, including severe or life-threatening reactions, and local injection-site reactions can occur with DARZALEX FASPRO®. Fatal reactions have been reported with daratumumab-containing products, including DARZALEX FASPRO®.
Systemic Reactions
In a pooled safety population of 1446 patients with multiple myeloma (N=1235) or light chain (AL) amyloidosis (N=193) who received DARZALEX FASPRO® as monotherapy or in combination, 7% of patients experienced a systemic administration-related reaction (Grade 2: 3%, Grade 3: 0.8%, Grade 4: 0.1%). In patients with high-risk smoldering multiple myeloma (N=193), systemic administration-related reactions occurred in 17% of patients in AQUILA (Grade 2: 7%, Grade 3: 1%).
In all patients (N=1639), systemic administration-related reactions occurred in 7% of patients with the first injection, 0.5% with the second injection, and cumulatively 1% with subsequent injections. The median time to onset was 3.2 hours (range: 4 minutes to 3.5 days). Of the 283 systemic administration-related reactions that occurred in 135 patients, 240 (85%) occurred on the day of DARZALEX FASPRO® administration. Delayed systemic administration-related reactions have occurred in 1% of the patients.
Severe reactions included hypoxia, dyspnea, hypertension, tachycardia, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Other signs and symptoms of systemic administration-related reactions may include respiratory symptoms, such as bronchospasm, nasal congestion, cough, throat irritation, allergic rhinitis, and wheezing, as well as anaphylactic reaction, pyrexia, chest pain, pruritus, chills, vomiting, nausea, hypotension, and blurred vision.
Pre-medicate patients with histamine-1 receptor antagonist, acetaminophen, and corticosteroids. Monitor patients for systemic administration-related reactions, especially following the first and second injections. For anaphylactic reaction or life-threatening (Grade 4) administration-related reactions, immediately and permanently discontinue DARZALEX FASPRO®. Consider administering corticosteroids and other medications after the administration of DARZALEX FASPRO® depending on dosing regimen and medical history to minimize the risk of delayed (defined as occurring the day after administration) systemic administration-related reactions.
Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with daratumumab-containing products. If ocular symptoms occur, interrupt DARZALEX FASPRO® and seek immediate ophthalmologic evaluation prior to restarting DARZALEX FASPRO®.
Local Reactions
In this pooled safety population of 1446 patients with multiple myeloma (N=1253) or light chain amyloidosis (N=193), injection-site reactions occurred in 8% of patients, including Grade 2 reactions in 1.1%. The most frequent (>1%) injection-site reaction were injection site erythema and injection site rash. In patients with high-risk smoldering multiple myeloma (N=193), injection-site reactions occurred in 28% of patients, including Grade 2 reactions in 3%. These local reactions occurred a median of 6 minutes (range: 0 minutes to 6.5 days) after starting administration of DARZALEX FASPRO®. Monitor for local reactions and consider symptomatic management.
Infections
DARZALEX FASPRO® can cause serious, life-threatening, or fatal infections. In patients who received DARZALEX FASPRO® in a pooled safety population including patients with smoldering multiple myeloma and light chain (AL) amyloidosis (N=1639), serious infections, including opportunistic infections, occurred in 24% of patients, Grade 3 or 4 infections occurred in 22%, and fatal infections occurred in 2.5%. The most common type of serious infection reported was pneumonia (8.5%).
Monitor patients for signs and symptoms of infection prior to and during treatment with DARZALEX FASPRO® and treat appropriately. Administer prophylactic antimicrobials according to guidelines.
Neutropenia
Daratumumab may increase neutropenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX FASPRO® until recovery of neutrophils. In lower body weight patients receiving DARZALEX FASPRO®, higher rates of Grade 3-4 neutropenia were observed.
Thrombocytopenia
Daratumumab may increase thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Consider withholding DARZALEX FASPRO® until recovery of platelets.
Embryo-Fetal Toxicity
Based on the mechanism of action, DARZALEX FASPRO® can cause fetal harm when administered to a pregnant woman. DARZALEX FASPRO® may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX FASPRO® and for 3 months after the last dose.
The combination of DARZALEX FASPRO® with lenalidomide, thalidomide, or pomalidomide is contraindicated in pregnant women because lenalidomide, thalidomide, and pomalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, thalidomide, or pomalidomide prescribing information on use during pregnancy.
Interference With Serological Testing
Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab administration. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient's serum. The determination of a patient's ABO and Rh blood type are not impacted.
Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX FASPRO®. Type and screen patients prior to starting DARZALEX FASPRO®.
Interference With Determination of Complete Response
Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some DARZALEX FASPRO®-treated patients with IgG kappa myeloma protein.
ADVERSE REACTIONS
In multiple myeloma, the most common adverse reaction (≥20%) with DARZALEX FASPRO® monotherapy is upper respiratory tract infection. The most common adverse reactions with combination therapy (≥20% for any combination) include fatigue, nausea, diarrhea, dyspnea, insomnia, headache, rash, pyrexia, cough, muscle spasms, back pain, vomiting, hypertension, musculoskeletal pain, upper respiratory tract infection, peripheral neuropathy, peripheral sensory neuropathy, constipation, pneumonia, edema, peripheral edema, and anemia.
The most common adverse reactions (≥20%) in patients with high-risk smoldering multiple myeloma who received DARZALEX FASPRO® monotherapy are upper respiratory tract infection, musculoskeletal pain, fatigue, diarrhea, rash, sleep disorder, sensory neuropathy, and injection site reactions.
The most common hematology laboratory abnormalities (≥40%) with DARZALEX FASPRO® are decreased leukocytes, decreased lymphocytes, decreased neutrophils, decreased platelets, and decreased hemoglobin.
Please
click here
to read the full Prescribing Information for DARZALEX FASPRO®.
DARZALEX® INDICATIONS AND IMPORTANT SAFETY INFORMATION
INDICATIONS
DARZALEX® (daratumumab) is indicated for the treatment of adult patients with multiple myeloma:
In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant
In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy
In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant
In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor
In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy
In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy
As monotherapy in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent
CONTRAINDICATIONS
DARZALEX® is contraindicated in patients with a history of severe hypersensitivity (eg, anaphylactic reactions) to daratumumab or any of the components of the formulation.
WARNINGS AND PRECAUTIONS
Infusion-Related Reactions
DARZALEX® can cause severe and/or serious infusion-related reactions including anaphylactic reactions. These reactions can be life threatening, and fatal outcomes have been reported. In clinical trials (monotherapy and combination: N=2066), infusion-related reactions occurred in 37% of patients with the Week 1 (16 mg/kg) infusion, 2% with the Week 2 infusion, and cumulatively 6% with subsequent infusions. Less than 1% of patients had a Grade 3/4 infusion-related reaction at Week 2 or subsequent infusions. The median time to onset was 1.5 hours (range: 0 to 73 hours). Nearly all reactions occurred during infusion or within 4 hours of completing DARZALEX®. Severe reactions have occurred, including bronchospasm, hypoxia, dyspnea, hypertension, tachycardia, headache, laryngeal edema, pulmonary edema, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Signs and symptoms may include respiratory symptoms, such as nasal congestion, cough, throat irritation, as well as chills, vomiting, and nausea. Less common signs and symptoms were wheezing, allergic rhinitis, pyrexia, chest discomfort, pruritus, hypotension and blurred vision.
When DARZALEX® dosing was interrupted in the setting of ASCT (CASSIOPEIA) for a median of 3.75 months (range: 2.4 to 6.9 months), upon re-initiation of DARZALEX®, the incidence of infusion-related reactions was 11% for the first infusion following ASCT. Infusion-related reactions occurring at re-initiation of DARZALEX® following ASCT were consistent in terms of symptoms and severity (Grade 3 or 4: <1%) with those reported in previous studies at Week 2 or subsequent infusions. In EQUULEUS, patients receiving combination treatment (n=97) were administered the first 16 mg/kg dose at Week 1 split over two days, ie, 8 mg/kg on Day 1 and Day 2, respectively. The incidence of any grade infusion-related reactions was 42%, with 36% of patients experiencing infusion-related reactions on Day 1 of Week 1, 4% on Day 2 of Week 1, and 8% with subsequent infusions.
Pre-medicate patients with antihistamines, antipyretics, and corticosteroids. Frequently monitor patients during the entire infusion. Interrupt DARZALEX® infusion for reactions of any severity and institute medical management as needed. Permanently discontinue DARZALEX® therapy if an anaphylactic reaction or life-threatening (Grade 4) reaction occurs and institute appropriate emergency care. For patients with Grade 1, 2, or 3 reactions, reduce the infusion rate when re-starting the infusion.
To reduce the risk of delayed infusion-related reactions, administer oral corticosteroids to all patients following DARZALEX® infusions. Patients with a history of chronic obstructive pulmonary disease may require additional post-infusion medications to manage respiratory complications. Consider prescribing short- and long-acting bronchodilators and inhaled corticosteroids for patients with chronic obstructive pulmonary disease.
Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with DARZALEX® infusion. If ocular symptoms occur, interrupt DARZALEX® infusion and seek immediate ophthalmologic evaluation prior to restarting DARZALEX®.
Interference With Serological Testing
Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab infusion. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient's serum. The determination of a patient's ABO and Rh blood type is not impacted. Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX®. Type and screen patients prior to starting DARZALEX®.
Neutropenia and Thrombocytopenia
DARZALEX® may increase neutropenia and thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX® until recovery of neutrophils or for recovery of platelets.
Interference With Determination of Complete Response
Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some patients with IgG kappa myeloma protein.
Embryo-Fetal Toxicity
Based on the mechanism of action, DARZALEX® can cause fetal harm when administered to a pregnant woman. DARZALEX® may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX® and for 3 months after the last dose.
The combination of DARZALEX® with lenalidomide, pomalidomide, or thalidomide is contraindicated in pregnant women because lenalidomide, pomalidomide, and thalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, pomalidomide, or thalidomide prescribing information on use during pregnancy.
ADVERSE REACTIONS
The most frequently reported adverse reactions (incidence ≥20%) were: upper respiratory infection, neutropenia, infusion related reactions, thrombocytopenia, diarrhea, constipation, anemia, peripheral sensory neuropathy, fatigue, peripheral edema, nausea, cough, pyrexia, dyspnea, and asthenia. The most common hematologic laboratory abnormalities (≥40%) with DARZALEX® are: neutropenia, lymphopenia, thrombocytopenia, leukopenia, and anemia.
Please click here to read the full Prescribing Information for DARZALEX®.
About Johnson & Johnson
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity.
Learn more at or at .
Janssen Research & Development, LLC and Janssen Biotech, Inc. are both Johnson & Johnson companies.
Cautions Concerning Forward-Looking Statements
This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj). The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments.
* Peter Voorhees, M.D., Atrium Health/Levine Cancer Institute, Charlotte, N.C., has provided consulting, advisory, and speaking services to Johnson & Johnson; he has not been paid for any media work.
1 DARZALEX FASPRO® U.S. Prescribing Information
2 American Cancer Society. What is Multiple Myeloma? Accessed November 2025. Available at:
3 Oben, B, Froyen, G, Maclachlan, K.H, et al. Whole-genome sequencing reveals progressive versus stable myeloma precursor conditions as two distinct entities. Nat Commun 2021;12(1861). doi:10.1038/s41467-021-22140-0
4 Maura, F, Bergsagel PL. Targeting the Tumor and the Immune System in Smoldering Multiple Myeloma. N Engl J Med. 2025;392:1858-1860. doi: 10.1056/NEJMe2504273
5 American Cancer Society. Myeloma Cancer Statistics. Accessed November 2025. Available at:
6 M.A. Dimopoulos, et al. Phase 3 Randomized Study of Daratumumab Monotherapy Versus Active Monitoring in Patients With High-risk Smoldering Multiple Myeloma: Primary Results of the AQUILA Study. Presented at the December 2024 ASH Annual Meeting & Exposition. Abstract JJD-78127.
7 Rajkumar SV. Multiple Myeloma: 2020 Update on Diagnosis, Risk-Stratification and Management. Am J Hematol. 2020;95(5):548-5672020;95(5):548-567.
8 National Cancer Institute. Plasma Cell Neoplasms. Accessed November 2025. Available at:
9 Multiple Myeloma. City of Hope, 2022. Multiple Myeloma: Causes, Symptoms & Treatments. Accessed November 2025. Available at:
10 American Cancer Society. Multiple Myeloma Early Detection, Diagnosis, and Staging. Accessed November 2025. Available at:
11 WebMD. Smoldering Multiple Myeloma. Accessed November 2025. Available at:
12 DARZALEX® U.S. Prescribing Information
SOURCE Johnson & Johnson
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Clinical ResultPhase 3Drug ApprovalASHLicense out/in
100 Deals associated with Carfilzomib
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External Link
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| D08880 | Carfilzomib |
R&D Status
Approved
10 top approved records. to view more data
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| Indication | Country/Location | Organization | Date |
|---|---|---|---|
| Multiple Myeloma | United States | 20 Jul 2012 |
Developing
10 top R&D records. to view more data
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Refractory Multiple Myeloma | Phase 3 | United States | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Japan | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Austria | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Bulgaria | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Czechia | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Finland | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | France | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Germany | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Greece | 08 May 2019 | |
| Refractory Multiple Myeloma | Phase 3 | Lithuania | 08 May 2019 |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
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| Study | Phase | Population | Analyzed Enrollment | Group | Results | Evaluation | Publication Date |
|---|
Phase 1/2 | 29 | (Dose Level 1: Carfilzomib 45mg/m2/Dose) | kdzotiebee = uusfqszkei nbtzzqesdl (bnadgxfeyt, veeniwexrs - smhsvyvhme) View more | - | 20 Nov 2025 | ||
(Dose Level 2: Carfilzomib 56mg/m2/Dose) | kdzotiebee = jqilknbvet nbtzzqesdl (bnadgxfeyt, jswudytihz - ockqmheqqt) View more | ||||||
Phase 2 | 46 | zprctpxgzi = lzzokcpdwh uwyjfxnebl (kvpwuppjwm, etoxwukonf - bwlbpvkoqm) View more | - | 10 Sep 2025 | |||
Phase 3 | 1,087 | gdiidmeyhi(icqjinsrrz) = sfdqlnpewh nujsavpbnq (eelshrqzfa ) | Positive | 30 May 2025 | |||
gdiidmeyhi(icqjinsrrz) = swnopcgliu nujsavpbnq (eelshrqzfa ) | |||||||
Not Applicable | - | gplgyevrci(joxxnqmocq): aOR = 1.61 (95% CI, 1.48 - 1.77), P-Value = < 0.001 View more | Negative | 30 May 2025 | |||
No Carfilzomib | |||||||
Phase 2 | Multiple Myeloma minimal residual disease (MRD) positive | 306 | yvaenidlkq(ielpjzkmnq): adjusted OR = 2.5 (95% CI, 1.5 - 4.2) View more | Positive | 30 May 2025 | ||
Phase 2 | Multiple Myeloma HR cytogenetic aberration (CA) (del(17p), t(4;14), t(14;16), ≥3 copies 1q21) | 219 | jmrxbqjdtv(zogvtpefzy) = mOS has not been reached nqqgeecolb (xvyyheymlw ) View more | Positive | 30 May 2025 | ||
Phase 3 | Multiple Myeloma Maintenance | 81 | Carfilzomib, lenalidomide, and dexamethasone (KRd) | vpjdgmozcm(hbhmphbnxq) = zqilpxtmav jungcwjeiy (mmxobsvego ) View more | Positive | 30 May 2025 | |
vpjdgmozcm(hbhmphbnxq) = omcueycdmp jungcwjeiy (mmxobsvego ) View more | |||||||
Phase 2 | 38 | muuftqgjmn(aecfzcyysk) = ivbecvtehu pozolnpbzv (nsljvmbcjy ) View more | Positive | 30 May 2025 | |||
Phase 2 | 306 | DKRd | jqbcyyualb(txqvsxgzxw) = fmkricumwf juhjbbobxd (nysuonnyvt ) View more | Positive | 14 May 2025 | ||
KRd | jqbcyyualb(txqvsxgzxw) = bgrukftgzw juhjbbobxd (nysuonnyvt ) View more | ||||||
Phase 2 | Consolidation | Maintenance t4;14 | t(14;16) | del17 ... View more | 474 | (KRd-ASCT) | kjekxnbbyw(amdyvcuosn) = 8-years OS was similar in the 3 arms (75% vs. 72% vs. 68%, respectively) edwmqstckp (vadswcqcyl ) View more | Positive | 14 May 2025 | |
(KRd12) |
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