Lisocabtagene maraleucel
- Generic Name
- Lisocabtagene maraleucel
- Brand Names
- Breyanzi
- Drug Type
- Biotech
- Unique Ingredient Identifier
- 7K2YOJ14X0
- Background
Lisocabtagene maraleucel is a chimeric antigen receptor (CAR) T-cell therapy, similar to brexucabtagene autoleucel and axicabtagene ciloleucel. Lisocabtagene maraleucel is a genetically modified autologous T-cell therapy that targets CD19, the B-lymphocyte surface antigen B4.
CAR T-cell therapy has changed the treatment of B-cell lymphomas, significantly increasing survival rates over standard therapy. However, data on the efficacy of CAR T-cell therapies on less severe forms of B-cell lymphoma are lacking. Despite the adverse reactions, the majority of patients given lisocabtagene maraleucel reported an overall increase in quality of life over a 1 year period.
Lisocabtagene maraleucel was granted FDA approval on 5 February 2021 and EC approval on 5 April 2022. It was later granted Health Canada approval on 6 May 2022.
- Indication
Lisocabtagene maraleucel is indicated to treat adults with relapsed or refractory large B-cell lymphoma after ≥2 systemic therapies, diffuse large B-cell lymphoma, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and grade 3B follicular lymphoma.
- Associated Conditions
- Grade 3b Follicular Lymphoma, High-grade B Cell Lymphoma (HGBCL), Primary Mediastinal (Thymic) Large B Cell Lymphoma (PMBCL), Refractory Diffuse Large B Cell Lymphoma (DLBCL), Refractory Large B-cell Lymphoma, Relapsed Diffuse Large B-cell Lymphoma (DLBCL)
CAR-T Cell Therapies Drive Growth in Adult T-Cell Leukemia-Lymphoma Market Despite Access Challenges
• The global Adult T-cell Leukemia-Lymphoma (ATLL) market is projected to experience significant growth through 2035, driven by increasing HTLV-1 infections and advancements in immunotherapy technologies.
• CAR-T cell therapies from companies like Gilead Sciences, Bristol Myers Squibb, and Novartis are revolutionizing ATLL treatment, though high costs—approximately $525,000 per infusion—create substantial access barriers.
• Pharmaceutical companies are working to reduce CAR-T manufacturing turnaround time by up to 50%, potentially improving outcomes for patients with aggressive forms of ATLL where timely intervention is critical.
Artiva's AlloNK Shows Promising Durability in B-Cell Non-Hodgkin Lymphoma, Matching CAR-T Efficacy
• Artiva Biotherapeutics' AlloNK in combination with rituximab demonstrated a 64% complete response rate in heavily pretreated B-cell non-Hodgkin lymphoma patients, comparable to approved autologous CAR-T therapies.
• The median duration of response has not been reached and extends beyond 19.4 months, with a well-tolerated safety profile showing no cytokine release syndrome or neurotoxicity events.
• Results suggest AlloNK's potential for deep B-cell depletion in autoimmune diseases, with a safety profile compatible with outpatient administration in community settings.
Long-Term Follow-Up of TRANSFORM Trial Shows Sustained Benefits of Liso-Cel CAR T-Cell Therapy in Relapsed LBCL
• Three-year follow-up data from the phase 3 TRANSFORM trial demonstrates lisocabtagene maraleucel (liso-cel) significantly improved event-free survival with a median of 29.5 months versus 2.4 months with standard of care in relapsed large B-cell lymphoma.
• Liso-cel showed impressive efficacy with an 87% overall response rate and 74% complete response rate, while maintaining a favorable safety profile with lower rates of cytokine release syndrome and neurotoxicity compared to axicabtagene ciloleucel.
• The study revealed that patients who received liso-cel as second-line therapy had substantially better outcomes than those who crossed over after standard chemotherapy, emphasizing the importance of early CAR T-cell intervention.
Pfizer to Showcase Breakthrough Cancer Therapies at ASCO 2025 with Over 60 Presentations
• Pfizer will present data from more than 60 abstracts at ASCO 2025, including 15 oral presentations highlighting advancements across breast, genitourinary, hematologic, thoracic, and colorectal cancers.
• Two late-breaking presentations will be featured in ASCO's press program, including pivotal survival data for BRAFTOVI in colorectal cancer and progression-free survival results for vepdegestrant in metastatic breast cancer.
• The company will showcase novel antibody-drug conjugate combinations with immunotherapy and present five-year survival data for XTANDI in prostate cancer, supporting Pfizer's goal of delivering eight breakthrough cancer medicines by 2030.
Liso-cel Combined With Ibrutinib Shows Promise for Relapsed/Refractory CLL and Richter Transformation
• Phase 1/2 Transcend CLL 004 Study demonstrates promising efficacy of lisocabtagene maraleucel (liso-cel) combined with ibrutinib in patients with relapsed/refractory CLL/SLL, achieving high response rates.
• A case series of six patients with Richter transformation reported an 83.3% overall response rate to liso-cel, with all four surviving patients receiving concurrent ibrutinib and achieving MRD-negative complete responses.
• Researchers suggest ibrutinib may enhance CAR T-cell therapy efficacy by overcoming T-cell anergy in CLL, potentially offering new hope for patients with limited treatment options and poor prognosis.
European Commission Approves BMS's Breyanzi for Relapsed or Refractory Follicular Lymphoma
• The European Commission has granted expanded approval to Bristol Myers Squibb's CAR T cell therapy Breyanzi (lisocabtagene maraleucel) for treating adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.
• In the Phase II TRANSCEND FL trial, Breyanzi demonstrated impressive efficacy with a 97.1% overall response rate and 94.2% complete response rate in third-line and beyond treatment settings.
• The approval extends across all European Union member states plus Norway, Liechtenstein, and Iceland, addressing significant unmet needs for follicular lymphoma patients with limited treatment options.
Bristol Myers Squibb Acquires 2seventy bio for $286 Million to Gain Full Control of CAR-T Therapy Abecma
• Bristol Myers Squibb has agreed to acquire 2seventy bio for $286 million, ending the profit-sharing arrangement for the BCMA-targeted CAR-T therapy Abecma used in multiple myeloma treatment.
• The acquisition represents an 88% premium over 2seventy's closing share price but marks the end of a challenging journey for the cell therapy company, which was spun out from bluebird bio in 2021.
• BMS's strategic move aligns with its cost-cutting initiatives aimed at saving $3.5 billion through 2027, as the company faces patent expirations for key products including Opdivo, Yervoy, and Eliquis.
CAR T-cell Therapy Pipeline Surges with 180+ Companies Advancing Novel Cancer Treatments
• The CAR T-cell therapy pipeline has experienced significant growth, with over 180 companies actively developing more than 200 innovative cell therapy candidates across various stages of clinical development.
• Recent breakthroughs include Hemogenyx's first human administration of HG-CT-1 for acute myeloid leukemia and NICE's approval of lisocabtagene maraleucel for large B-cell lymphoma treatment.
• Strategic industry developments are accelerating progress, with companies like CARsgen Therapeutics forming alliances to advance allogeneic CAR-T products and multiple firms reporting successful trial milestones.
Verastem Oncology to Present New LGSOC Treatment Data at SGO 2025 Annual Meeting
• Verastem Oncology will present additional analyses from the Phase 2 RAMP 201 trial evaluating avutometinib plus defactinib in recurrent low-grade serous ovarian cancer at SGO 2025, building on their FDA Priority Review submission.
• The presentations include new subgroup analyses by KRAS mutational status, supporting the company's ongoing efforts to advance treatment options for RAS/MAPK pathway-driven cancers.
• The company's NDA for the avutometinib-defactinib combination therapy has received Priority Review from the FDA with a PDUFA date of June 30, 2025, for KRAS mutant LGSOC patients.
NHS Approves Breakthrough CAR T-Cell Therapy Liso-cel for Advanced Lymphoma Patients
• A groundbreaking CAR T-cell therapy, liso-cel, has been approved by NHS for treating large B-cell lymphoma patients who haven't responded to initial treatment or experienced relapse within 12 months.
• Clinical trials demonstrate that liso-cel significantly extends progression-free survival, with patients living more than twice as long without disease worsening compared to standard care.
• The treatment, priced at £297,000 per patient, is expected to benefit approximately 600 patients annually and can be administered in an outpatient setting, reducing hospital stays.
Nivolumab Plus Ipilimumab Shows Promise in Advanced Hepatocellular Carcinoma
• The combination of nivolumab and ipilimumab has shown significantly improved overall survival in patients with unresectable hepatocellular carcinoma (HCC).
• The CheckMate 9DW trial demonstrated a median overall survival of 23.7 months with nivolumab/ipilimumab compared to 20.6 months with lenvatinib or sorafenib.
• The combination therapy also resulted in a higher objective response rate of 36% versus 13% with lenvatinib/sorafenib, indicating better tumor control.
• The FDA has accepted the application for nivolumab plus ipilimumab as a first-line treatment for unresectable HCC, with a decision expected by April 2025.
Breyanzi Receives Positive CHMP Opinion for Follicular Lymphoma Treatment in EU
• The CHMP has recommended Breyanzi for treating relapsed or refractory follicular lymphoma (FL) in adults after two or more prior systemic therapies.
• The recommendation is based on the Phase 2 TRANSCEND FL study, which showed a 97.1% overall response rate and a 94.2% complete response rate.
• Breyanzi demonstrated rapid and durable responses, with 75.7% of patients in response at 18 months, and a consistent safety profile in clinical trials.
• The European Commission will review the CHMP recommendation, with a final decision expected within approximately two months.
J&J's Carvykti Shows Promise in Earlier Myeloma Treatment
• The CARTITUDE-4 study indicates that Carvykti (ciltacabtagene autoleucel) significantly improves progression-free survival in multiple myeloma patients with 1-3 prior lines of therapy.
• Carvykti, a BCMA-directed CAR-T therapy, may soon be used earlier in the treatment pathway, potentially leapfrogging Bristol-Myers Squibb's Abecma.
• The study compared Carvykti to standard three-drug regimens, showing a significant benefit that led to unblinding of the trial.
• Expansion of Carvykti's use is a key component of J&J's strategy in multiple myeloma, alongside other therapies like Darzalex and bispecific antibodies.
Kymriah Receives EU Approval as First CAR-T Therapy for Follicular Lymphoma
• Novartis' Kymriah is the first CAR-T therapy approved in the EU for relapsed/refractory follicular lymphoma, offering a new option for patients after multiple treatments.
• The approval was based on the ELARA trial, demonstrating an 86% response rate and 69% complete response rate in patients treated with Kymriah.
• Kymriah provides durable treatment effects, with 87% of complete responders remaining in remission for at least nine months after initial response.
• This approval expands Kymriah's indications in the EU, adding to its existing approvals for diffuse large B-cell lymphoma and acute lymphoblastic leukemia.
FDA Approves Subcutaneous Opdivo Qvantig for Multiple Solid Tumors
• The FDA approved Opdivo Qvantig, a subcutaneous injection of nivolumab, for adult solid tumors previously approved for intravenous Opdivo.
• CheckMate-67T trial data showed non-inferior pharmacokinetic exposures, similar efficacy, and comparable safety to intravenous Opdivo.
• Opdivo Qvantig offers a faster 3-5 minute administration time, providing convenience and flexibility for patients.
• The approval helps Bristol Myers Squibb maintain market share amid patent expirations and increasing biosimilar competition.
CAR T-Cell Therapy Shows Promise in Treating Autoimmune Diseases: Clinical Trials and Future Directions
• UChicago Medicine launched a Phase 2 clinical trial to explore CAR T-cell therapy for systemic lupus erythematosus, inflammatory myositis, and systemic sclerosis.
• CAR T-cell therapy, initially for blood cancers, is being adapted to autoimmune diseases, potentially offering a new solution for resistant cases.
• BMS reported promising Phase 1 trial results using CD19 CAR T-cells for severe autoimmune diseases, showing significant disease activity reduction.
• Researchers are optimistic about CAR T-cell therapy's potential to 'reset' the immune system, but emphasize the need for long-term data and safety monitoring.
Lisocabtagene Maraleucel Shows Promise in Relapsed/Refractory Lymphomas
• Lisocabtagene maraleucel (liso-cel) demonstrates efficacy and safety in second-line treatment of relapsed/refractory large B-cell lymphoma (LBCL), aligning with pivotal trial outcomes.
• The TRANSCEND FL trial indicates liso-cel achieved a significant overall response rate in relapsed/refractory marginal zone lymphoma (MZL), meeting the primary endpoint.
• Five-year data from TRANSCEND-NHL-001 underscore the curative potential of liso-cel in third-line LBCL, showing a 38% overall survival rate.
• Real-world data supports liso-cel's effectiveness across diverse LBCL patient subgroups, reinforcing its role as a standard-of-care treatment option.
Acalabrutinib Plus Venetoclax Significantly Improves PFS in Untreated CLL
• The phase 3 AMPLIFY trial demonstrated that acalabrutinib plus venetoclax, with or without obinutuzumab, significantly improved progression-free survival (PFS) in treatment-naive CLL patients.
• The doublet and triplet regimens reduced the risk of disease progression or death by 35% and 58%, respectively, compared to chemoimmunotherapy.
• The highest rates of undetectable minimal residual disease (uMRD) were observed in patients treated with the triplet regimen of acalabrutinib, venetoclax, and obinutuzumab.
• The combination provides a chemotherapy-free, fixed-duration option for previously untreated CLL, offering improved outcomes and flexibility in managing the disease.
FDA Accepts sBLA for Glofitamab Plus Chemotherapy in Relapsed/Refractory DLBCL
• The FDA has accepted Roche's sBLA for glofitamab combined with gemcitabine and oxaliplatin (GemOx) for relapsed/refractory DLBCL patients ineligible for autologous stem cell transplant.
• The sBLA is based on the phase 3 STARGLO trial, which demonstrated a statistically significant and clinically meaningful improvement in overall survival compared to rituximab plus GemOx.
• The FDA is expected to make a decision on the approval of glofitamab in combination with GemOx by July 20, 2025, offering a potential new treatment option.
• The safety profile of glofitamab plus GemOx was consistent with the known safety profiles of the individual agents, with cytokine release syndrome being a common adverse event.
Cytokinetics' Aficamten Advances in Regulatory Review for Obstructive Hypertrophic Cardiomyopathy
• The EMA has validated Cytokinetics' Marketing Authorization Application (MAA) for aficamten, a cardiac myosin inhibitor, for treating obstructive hypertrophic cardiomyopathy (HCM).
• The FDA has accepted the New Drug Application (NDA) for aficamten with a PDUFA target action date of September 26, 2025, and no advisory committee meeting is planned.
• Aficamten significantly improved exercise capacity and clinical outcomes in the SEQUOIA-HCM Phase 3 trial, supporting regulatory submissions in the U.S., Europe, and China.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath