RegulationOrphan Drug (US), Breakthrough Therapy (CN), Conditional marketing approval (CN), Accelerated Approval (US), Priority Review (CN), Orphan Drug (EU)

Gene Sequence

Sequence Code 9718ScFv

Sequence Code 170963ScFv

Sequence Code 13042446CAR construct

Clinical Trials associated with Axicabtagene Ciloleucel

NCT06609304 / RecruitingPhase 4IIT

Prospective Clinical Study of Axicabtagene Ciloleucel for Consolidation After First-line Treatment of High-risk Large B-cell Lymphoma

The goal of this clinical trial is to learn if Axicabtagene Ciloleucel (axi-cel) works for consolidation after first-line treatment of high-risk Large B-cell Lymphoma (LBCL). It will also learn about the safety of axi-cel treatment. The main questions it aims to answer are:
* Does axi-cel treatment result in prolonged clinical benefit to patients with high-risk LBCL after first-line treatment?
* What medical problems do participants have when receiving axi-cel treatment?
In this investigator-initiated, single-arm clinical trial, participants will:
* Receive atezolizumab treatment at 2.0×10^6 cells/Kg as a one-time therapy.
* Visit the clinic as instructed for checkups and tests.

Start Date30 Sep 2024

Sponsor / Collaborator

Zhengzhou University

NCT06550141 / Not yet recruitingPhase 2IIT

A Phase 2 Trial of Emapalumab for the Prevention of CAR-T Cell Associated Toxicities

This research study involves assessing the impact of emapalumab as preventative management of CAR-T related cytokine release syndrome in participants with Non-Hodgkin's lymphoma (NHL).
The research study involves the following study interventions:
* Fludarabine and cyclophosphamide (Lymphodepleting Chemotherapy)
* Axicabtagene Ciloleucel
* Emapalumab

Start Date01 Aug 2024

Sponsor / Collaborator

The General Hospital Corp.

[+1]

ChiCTR2400086809 / SuspendedNot ApplicableIIT

A single-arm, multicenter, open clinical study to evaluate the efficacy and safety of Axicabtagene Ciloleucel injection as second -line treatment for adult patients with relapsed/refractory large B-cell lymphoma (r/r LBCL)

Start Date31 Jul 2024

Sponsor / Collaborator-

100 Clinical Results associated with Axicabtagene Ciloleucel

100 Translational Medicine associated with Axicabtagene Ciloleucel

100 Patents (Medical) associated with Axicabtagene Ciloleucel

544

Literatures (Medical) associated with Axicabtagene Ciloleucel

31 Dec 2024·Journal of medical economics

Cost-effectiveness of axicabtagene ciloleucel versus tisagenlecleucel for the treatment of 3L + relapsed/refractory large B-cell lymphoma in the United States: incorporating longer survival results

Article

Author: Davies, Neil ; Patel, Anik R ; Bradford, Rory ; Jones, Calum ; Ray, Markqayne D ; Oluwole, Olalekan O ; Locke, Frederick L

AIMS:

To provide an update on the cost-effectiveness of the chimeric antigen receptor (CAR) T-cell therapies axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) for the treatment of relapsed/refractory (r/r) large B-cell lymphoma (LBCL) among patients who have previously received ≥2 lines of systemic therapy using more mature clinical trial data cuts (60 months for axi-cel overall survival [OS] and 45 months for tisa-cel OS and progression-free survival [PFS]).

METHODS:

A partitioned survival model consisting of three health states (pre-progression, post-progression and death) was used to estimate quality-adjusted life years (QALYs) and costs associated with axi-cel and tisa-cel over a lifetime horizon. PFS and OS inputs for axi-cel and tisa-cel were based on a previously published matching-adjusted indirect treatment comparison (MAIC). Long-term OS and PFS were extrapolated using parametric survival mixture cure models (PS-MCMs). Costs of CAR-T cell therapy drug acquisition and administration, conditioning chemotherapy, apheresis, CAR T-specific monitoring, stem cell transplant, hospitalization, adverse events, routine care, and terminal care were sourced from US cost databases. Health state utilities were derived from previous publications. Model inputs were varied using a range of sensitivity and scenario analyses.

RESULTS:

Compared with tisa-cel, axi-cel resulted in 2.51 additional QALYs and $50,185 additional costs (an incremental cost-effectiveness ratio [ICER] of $19,994 per QALY gained). In probabilistic sensitivity analysis (PSA), the ICER for axi-cel versus tisa-cel was ≤$50,000/QALY in 99.4% of simulations and ≤$33,500 in 99% of simulations. Axi-cel remained cost-effective versus tisa-cel (assuming a willingness-to-pay threshold of $150,000 per QALY) across a range of scenarios.

CONCLUSIONS:

With longer-term survival data, axi-cel continues to represent a cost-effective option versus tisa-cel for treatment of r/r LBCL among patients who have previously received ≥2 lines of systemic therapy, from a US payer perspective.

31 Dec 2024·Journal of medical economics

An updated cost-effectiveness analysis of axicabtagene ciloleucel in second-line large B-cell lymphoma patients in the United States

Article

Author: Johnston, Patrick B ; Dickinson, Michael ; Patel, Anik R ; Vadgama, Sachin ; Oluwole, Olalekan O ; Perales, Miguel-Angel ; Feng, Chaoling ; Smith, Nathaniel J ; Blissett, Rob

AIMS:

This economic evaluation of axicabtagene ciloleucel (axi-cel) versus previous standard of care (SOC; salvage chemotherapy followed by high-dose therapy with autologous stem cell rescue) in the second line (2L) large B-cell lymphoma population is an update of previous economic models that contained immature survival data.

METHODS:

This analysis is based on primary overall survival (OS) ZUMA-7 clinical trial data (median follow-up of 47.2 months), from a United States (US) payer perspective, with a model time horizon of 50 years. Mixture cure models were used to extrapolate updated survival data; subsequent treatment data and costs were updated. Patients who remained in the event-free survival state by 5 years were assumed to have achieved long-term remission and not require subsequent treatment.

RESULTS:

Substantial survival and quality of life benefits were observed despite 57% of patients in the SOC arm receiving subsequent cellular therapy: median model-projected (ZUMA-7 trial Kaplan-Meier estimated) OS was 78 months (median not reached) for axi-cel versus 25 months (31 months) for SOC, resulting in incremental quality-adjusted life year (QALY) difference of 1.63 in favor of axi-cel. Incrementally higher subsequent treatment costs were observed in the SOC arm due to substantial crossover to cellular therapies, thus, when considering the generally accepted willingness to pay threshold of $150,000 per QALY in the US, axi-cel was cost-effective with an incremental cost-effectiveness ratio of $98,040 per QALY.

CONCLUSIONS:

Results remained consistent across a wide range of sensitivity and scenario analysis, including a crossover adjusted analysis, suggesting that the mature OS data has significantly reduced the uncertainty of axi-cel's cost-effectiveness in the 2L setting in the US. Deferring treatment with CAR T therapies after attempting a path to transplant may result in excess mortality, lower quality of life and would be an inefficient use of resources relative to 2L axi-cel.

31 Dec 2024·JOURNAL OF MEDICAL ECONOMICS

Cost-effectiveness analysis of mosunetuzumab for treatment of relapsed or refractory follicular lymphoma after two or more lines of systemic therapy in the United States

Article

Author: Sanchez Alvarez, Javier ; Lin, Shih-Wen ; Parisé, Hélène ; Di Maio, Danilo ; Zuk, Eric ; Kim, Eunice ; Shapouri, Sheila ; Matasar, Matthew

AIMS:

Mosunetuzumab has received accelerated approval by the US Food and Drug Administration for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. We evaluated the cost-effectiveness of mosunetuzumab for the treatment of R/R FL from a US private payer perspective.

MATERIALS AND METHODS:

A partitioned survival model simulated lifetime costs and outcomes of mosunetuzumab against seven comparators: axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), tazemetostat (taz, EZH2 wild-type only), rituximab plus lenalidomide (R-Len) or bendamustine (R-Benda), obinutuzumab plus bendamustine (O-Benda), and a retrospective real-world cohort (RW) based on current patterns of care derived from US electronic health records (Flatiron Health). Efficacy data for mosunetuzumab were from the pivotal Phase II GO29781 trial (NCT02500407). Relative treatment efficacy was estimated from indirect treatment comparisons (ITCs). Costs included were related to treatment, adverse events, routine care, and terminal care. Except for drug costs (March 2023), all costs were inflated to 2022 US dollars. Costs and quality-adjusted life-years (QALYs) were used to calculate incremental cost-effectiveness ratios (ICERs). Net monetary benefit (NMB) was calculated using a willingness-to-pay (WTP) threshold of $150,000/QALY.

RESULTS:

Mosunetuzumab dominated taz, tisa-cel, and axi-cel with greater QALYs and lower costs. Mosunetuzumab was projected to be cost-effective against R-Benda, O-Benda, and RW with ICERs of $78,607, $42,731, and $21,434, respectively. Mosunetuzumab incurred lower costs but lower QALYs vs. R-Len. NMBs showed that mosunetuzumab was cost-effective against comparators except R-Len.

LIMITATIONS:

Without head-to-head comparative data, the model had to rely on ITCs, some of which were affected by residual bias. Model inputs were obtained from multiple sources. Extensive sensitivity analyses assessed the importance of these uncertainties.

CONCLUSION:

Mosunetuzumab is estimated to be cost-effective compared with approved regimens except R-Len for the treatment of adults with R/R FL.

267

News (Medical) associated with Axicabtagene Ciloleucel

30 Oct 2024

·www.prnewswire.com

Fosun Pharma Announces 2024Q3 Financial Results

Revenue Exceed RMB30.9 Billion with R&D Expenditure RMB3.9 Billion Continuing to Consolidate its Dominant Position In The fields of Breast Cancer and Lung Cancer SHANGHAI, Oct. 29, 2024 /PRNewswire/ -- On October 29, Shanghai Fosun Pharmaceutical (Group) Co., Ltd. ("Fosun Pharma" or "the Group"; Stock Code: 600196.SH; 02196.HK), announced its operating performance for the first three quarters of 2024. From January to September 2024, Fosun Pharma achieved revenue of RMB30.91 billion, an increase of about 5.74% YoY after excluding COVID-related products. The net profit attributable to owners of the parent of the Group after deducting extraordinary gain or loss amounted to RMB1.84 billion, up by 24.58% YoY. In 2024, Fosun Pharma will further focus on innovative drugs and high-value devices, and continue to promote lean operations, cost reduction, efficiency improvement, and asset rationalization to optimize assets and financial structure, actively promote supply chain management and operational efficiency, and achieve healthy operating cash flow. In the first three quarters of 2024, the net cash flow from operating activities of Fosun Pharma was RMB2.99 billion, a year-on-year increase of 21.33%; the management expense ratio decreased by 0.15 percentage points YoY; excluding the impact of newly acquired companies, management expenses decreased by approximately RMB300 million on the same basis. Focusing on advantageous therapeutic areas with innovative R&D pipelines continue to advance In terms of innovative R&D, Fosun Pharma continues to focus on advantageous pipelines to achieve efficient results and continuous implementation of innovative products. In the first three quarters of 2024, Fosun Pharma's R&D expenditure totaled RMB3.92 billion. In particular, R&D expenses were RMB2.65 billion. In addition to independent R&D, the Group fully implemented an open R&D model, and incubated and invested in R&D projects by initiating/managing industrial funds and other diversified ways, so as to ensure the sustainability of innovation and R&D. Fosun Pharma's innovative drug business mainly covers the core therapeutic areas of solid tumors, hematological tumors and immuno-inflammatory diseases, with emphasis on the enhancement of the core technology platforms of antibody/ADCs, cellular therapy and small molecules, to create an open and global innovative R&D system, continuously enhance pipeline value, and promote the development and commercialization of its products. In the field of tumor immunotherapy, on September 13, 2024, Fosun Pharma announced it will acquire the 50% stake in Fosun Kite and will wholly own Fosun Kite (now renamed as "Fosun Kairos"). In the future, Fosun Kairos will serve as the core platform of Fosun Pharma's cell therapy, continue to focus on the field of tumor immunotherapy, as well as to promote the development and commercialization of existing licensed products, Axi-Cel (i.e. Fosun Kite's marketed product "Yi Kai Da") and Brexu-Cel (i.e. Fosun Kite's pipeline project FKC889), in Chinese mainland, Hong Kong SAR and Macau SAR with Kite Pharma. In the third quarter of 2024, Fosun Pharma's multiple independently developed innovative products and pipeline clinical trial results were announced in industry conferences and journals, further consolidating its dominant position in the fields of hematological tumors, breast cancer, lung cancer and other tumors. Particularly, the interim analysis results of the Phase III study of Foritinib Succinate (SAF-189s), an innovative drug independently developed to treat ALK-positiveb advanced non-small cell lung cancer (NSCLC), were released during the 2024 World Conference on Lung Cancer ("WCLC"). The above-mentioned studies have found that the overall efficacy of Foritinib Succinate is good. Compared with crizotinib treatment, it can significantly improve PFS and reduce the risk of CNS progression. Its safety is controllable, and no new safety signals appeared after treatment. Faritinib Succinate is expected to break through the current clinical difficulties faced in the treatment of ALK-positive NSCLC and bring new treatment options to NSCLC patients. The multicenter real-world study of Fosun Pharma's self-developed serplulimab-based immunochemotherapy for extensive-stage small cell lung cancer was released at the 2024 WCLC. The ASTRUM-005R study provides additional empirical evidence to support the therapeutic value of serplulimab plus chemotherapy and complements the pivotal data from the ASTRUM-005 clinical trial. Additionally, during the reporting period, the Committee for Medicinal Products for Human Use ("CHMP") of the European Medicines Agency (EMA) issued a positive opinion recommending approval for Fosun Pharma's self-developed serplulimab injection in combination with carboplatin and etoposide for the first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC). The CHMP's opinion will be submitted to the European Commission (EC) as a reference for marketing authorization approval. Results of the phase 2 study of HLX22, an innovative anti-HER2 monoclonal antibody (mAb), in combination with Han Qu You (trastuzumab) and chemotherapy for the first-line treatment of HER2-positive advanced gastric/gastroesophageal junction (G/GEJ) cancer were presented on 2024 ESMO Gastrointestinal Cancers Congress (ESMO GI) and MED, a flagship clinical and translational research monthly journal by Cell Press. The results showed that add HLX22 to HLX02 (trastuzumab) and chemotherapy as first-line therapy improved efficacy in HER2-positive G/GEJ cancer patients with manageable safety. In September 2024, Fosun Pharma announced the Biologics License Application ("BLA") for the licensed product RT002 (DaxibotulinumtoxinA-lanm, Chinese trademark: 达希斐®) for the temporary improvement in the appearance of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adult patients, was approved by the National Medical Products Administration ("NMPA"), becoming the first DaxibotulinumtoxinA-lanm approved for marketing in Chinese mainland. Additionally, the results of a Phase III multicenter, double-blind, placebo-controlled study conducted in China for the treatment of moderate to severe glabellar lines were published in the Journal of Plastic, Reconstructive & Aesthetic Surgery (JPRAS). The study demonstrates that the product provides durable efficacy and high safety in Chinese patients with moderate to severe glabellar lines. Actively implementing share repurchases and increasing holdings with continuous efforts on enhancing ESG Fosun Pharma, a company listed on both the A-share and H-share markets, and its controlling shareholder Fosun High Tech have actively engaged in share repurchases and increased holdings this year, demonstrating confidence in the Company's future development and further boosting market confidence. In the first three quarters of 2024, Fosun Pharma spent approximately RMB127 million and approximately HKD66.9 million to repurchase around 5.68 million A shares and 5.47 million H shares respectively. As of now, the H Share Repurchase Plan is still valid. In addition, the controlling shareholder Fosun High Tech, has spent a total of approximately RMB101 million to increase its holdings of around 4.30 million A shares of Fosun Pharma, under the latest Shareholding Increase Plan. Founded in 1994 and with three decades of development, Fosun Pharma has grown into a global innovation-driven pharmaceutical and healthcare industry group. The Company always regards innovation as its primary social responsibility, consistently focusing on unmet clinical needs, prioritizing innovative R&D, advancing the launch of innovative products, promoting drug accessibility and affordability, and driving high-quality corporate growth. Fosun Pharma maintained an A grade rating in MSCI ESG Ratings for three consecutive years and an A- rating in HSI ESG, ranking among the top tier in the healthcare industry for pharmaceuticals and biotechnology. It was also selected as a constituent of the Hang Seng (China A) Corporate Sustainability Benchmark Index ("HSCASUSB"), Hang Seng (China A) Corporate Sustainability Index ("HSCASUS"), and Hang Seng (Mainland and HK) Corporate Sustainability Index ("HSMHSUS"). With its sustained excellence in innovation and global operational capabilities, Fosun Pharma has been recognized by multiple authoritative institutions in innovative R&D, ESG and other areas. The Company ranked in the top four of China's 2023 Top 100 Pharmaceutical Industry, was included in the "China Best Managed Companies" List (BMC) for the second consecutive year and was listed in Forbes 2024 China ESG 50. In addition, the Company has actively introduced its independently developed artemisinin-based innovative products to Africa, providing a Chinese solution to the global fight against malaria. This initiative highlights the inclusive, diverse, and open nature of ESG and earned Fosun Pharma recognition as an "Inspirational ESG Case", serving as a model for corporate ESG practices. *** About Fosun Pharma Founded in 1994, Shanghai Fosun Pharmaceutical (Group) Co., Ltd.* ("Fosun Pharma"; stock code: 600196.SH, 02196.HK) is a global innovation-driven pharmaceutical and healthcare industry group. Fosun Pharma directly operates businesses including pharmaceuticals, medical devices, medical diagnosis, and healthcare services. As a shareholder of Sinopharm Co., Ltd., Fosun Pharma expands its areas in the pharmaceutical distribution and retail business. Fosun Pharma is patient-centered and unmet clinical needs-oriented. Through diversified and multi-level cooperation models such as independent research and development, cooperative development, license-in, and industrial investment, the company continues to enrich its innovative product pipeline and focus on differentiated product R&D with high-tech barriers, to continuously enhance the value of its pipeline. Fosun Pharma's innovative products focus on core therapeutic areas such as solid tumors, hematologic tumors and immunity inflammation. It also strengthens core technology platforms such as antibodies/ADC, cell therapy, and small molecules. Guided by the 4IN strategy (Innovation, Internationalization, Intelligentization, and Integration), Fosun Pharma adheres to the business philosophy of "Innovation for Good Health", continues to promote innovative transformation, actively deploys internationalization, strengthens business focus by product lines, promotes integrated operations and efficiency improvement, and is dedicated to being the global leading integrator of pharmaceutical and healthcare innovation. For more information, please visit the official website: SOURCE Fosun Pharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyDrug ApprovalPhase 2Phase 3

30 Oct 2024

·www.echemi.com

Controversy Erupts Over South Korea's New Drug Compensation Price Reform: 68 Drugs Face Refund Risks!

The Ministry of Health and Welfare (MHW) recently announced an implementation plan to improve the compensation price system for innovative new drugs, which aims to revise the current regulations on drug compensation and price setting. The proposal is currently under public consultation until Dec 16, 2024. Overall, the new reforms covered by the latest proposal could provide manufacturers with greater flexibility to handle drug reimbursement and pricing negotiations with South Korean authorities. Under the proposal, the Ministry of Health and Welfare plans to introduce two new risk-sharing agreements, namely fixed-cost initial treatment refunds and performance-based refunds, on top of four existing mechanisms. Under the fixed-cost initial treatment refund RSA, drug manufacturers would be required to return a certain amount of money for their products upon each patient receiving their first dose. As for the performance-based refund RSA, if the patient fails to achieve a certain clinical benefit after receiving the product, the manufacturer will need to repay the government. In addition to the two newly introduced RSA types, the existing RSA types in Korea are mandatory economic evaluation/spending caps, rebates, patient-level usage limits/usage caps, and a conditional continuing treatment type plus a money-back guarantee. Risk sharing agreements (RSAs) are a commonly used pricing and reimbursement strategy for insurance payers to control the future financial impact resulting from treatments with a high degree of uncertainty about clinical benefits or economic effects. Overall, the implementation of RSAs has had a positive impact on the pharmaceutical industry, implying that high-cost new therapies with highly uncertain cost-benefit outcomes can gain reimbursement status at an earlier stage under more flexible price negotiation strategies. GlobalData has identified at least 25 markets worldwide that use RSA mechanisms, both developed and emerging. South Korea introduced the RSA system in 2013. A total of 68 drugs have been introduced into South Korea's RSAs reimbursement list as of August 2023, according to an official at the National Health Insurance Service (NHIS), the pricing agency. In the Asia-Pacific region, several markets with comprehensive healthcare systems and established reimbursement systems for drugs already use RSAs under their national health insurance schemes. Australia, New Zealand, and Taiwan Province of China (along with South Korea) are prominent examples of widespread use of RSA in drug reimbursement decision-making processes. All four markets have formal RSA systems that can support access to national health insurance benefits for a drug at an early stage. There are seven RSA mechanisms in New Zealand, with rebates being the most commonly used RSA.NHIA also has the flexibility to accept other types of RSA. However, the two largest markets in Asia Pacific, China and Japan, have yet to implement official RSAs in their drug reimbursement decision-making process. Japan reimburses almost all prescription drugs under its national health insurance program and thus uses its strict repricing rules to control pharmaceutical spending. In the case of China, the coverage of the National Reimbursement Drug List (NRDL) is highly dependent on confidential price negotiations, in which case RSAs are less likely to be used. However, Chinese commercial firms use the RSA mechanism to reimburse for new high-cost treatments. Recently, Chinese drug developer Fosun Cate Biotech (China; A joint venture between Fosun Pharmaceutical and Kite Pharma) launched RSA of Yescarta (axicabtagene ciloleucel), a performance-based chimeric antigen receptor T-cell (CAR-T) therapy, This scheme was not included in the NRDL. As the RSA system in Korea is expected to be expanded under this reform, manufacturers with high-cost therapeutic products will have greater flexibility in reaching price agreements with NHIS. This will open up more opportunities for the pharmaceutical industry, while also providing patients with more treatment options.

ImmunotherapyCell Therapy

25 Oct 2024

·www.pharmaceutical-technology.com

Lyell goes all in on CAR-T therapies, buys ImmPACT Bio and trims pipeline

Lyell intends to focus all its resources on CAR T-cell clinical programmes, with LYL119 and PmmPACT’s IMPT-314 being the lead candidates. Image credit: Andrii Yalanskyi / Shutterstock. Lyell Immunopharma has entered a definitive agreement to acquire ImmPACT Bio, a privately-owned clinical-stage biotechnology company specialising in chimeric antigen receptor (CAR)-T cell therapies. Concurrently, the company has discontinued the development of two programmes – LYL797, its ROR1-targeted CAR-T cell product; and LYL845, a tumour-infiltrating lymphocyte (TIL) therapy. Lyell noted that clinical data for LYL845 “did not meet our rigorous pre-determined criteria for continued development”, adding that the company plans to discontinue all preclinical TIL and rejuvenation programmes. Lyell is expected to pay $30m in cash and 37.5 million shares of Lyell common stock for the acquisition. The ImmPACT shareholders will also be in line to receive milestone-based payments of contingent consideration consisting of 12.5 million shares of Lyell common stock and a “low single-digit” royalty on future US net sales of IMPT-314. The deal is expected to close by the end of the year. Lyell noted that it expects its cash reserves to last into 2027 after closing the acquisition. ImmPACT’s lead therapy is IMPT-314, a bispecific CAR therapy targeting CD19 and CD20. It is being evaluated as a treatment for relapsed/refractory (r/r) aggressive B cell non-Hodgkin lymphoma in a Phase I/II trial (NCT05826535) . See Also: Pharma companies must adapt to keep pace with AI developments, say experts AF stroke patients could benefit from earlier anticoagulant administration The company intends to present data from the trial later this year, with Lyell planning to start a pivotal trial for IMPT-314 as a third-line treatment for patients who have not yet been exposed to CAR T-cell therapy in 2025. Lyell intends to focus all its resources on CAR-T clinical programmes. GlobalData estimates the cell and gene therapy market to be worth more than $81bn by 2029. Multiple CAR-T cell therapies have been approved in the US , the most successful of these is Gilead Sciences ’ Yescarta (axicabtagene ciloleucel) generating $1.5bn in sales last year. GlobalData is the parent company of Pharmaceutical Technology . Lyell noted that instead of LYL797, which was being evaluated as a treatment for advanced solid tumours in a Phase I trial (NCT05274451), it will focus on another ROR1-targeted CAR-T cell therapy, LYL119. The company plans to start a Phase I trial evaluating LYL119 in patients with platinum-resistant ovarian cancer or r/r endometrial cancer this year or early next year. Lyell had a rough start with LYL797, with the company reporting cases of pneumonitis in patients with lung metastases enrolled in a Phase I study. The initial dataset consisted of 20 patients, 16 with triple-negative breast cancer (TNBC) and four with non-small cell lung cancer (NSCLC). The company noted that the adverse event of Grade 3 or higher pneumonitis occurred only in patients with TNBC and lung metastases. As a result, Lyell modified the trial’s dosing schedule to separate dose escalation into two cohorts based on lung involvement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Phase 1Cell TherapyImmunotherapyAcquisitionGene Therapy

100 Deals associated with Axicabtagene Ciloleucel

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	L01	DB13915

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
High grade B-cell lymphoma	CA	Gilead Sciences Canada, Inc.	13 Feb 2019
Diffuse Large B-Cell Lymphoma	EU	Kite Pharma EU BV	23 Aug 2018
Diffuse Large B-Cell Lymphoma	IS	Kite Pharma EU BV	23 Aug 2018
Diffuse Large B-Cell Lymphoma	LI	Kite Pharma EU BV	23 Aug 2018
Diffuse Large B-Cell Lymphoma	NO	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	EU	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	IS	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	LI	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	NO	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	EU	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	IS	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	LI	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	NO	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	EU	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	IS	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	LI	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	NO	Kite Pharma EU BV	23 Aug 2018
Follicular Lymphoma	US	Kite Pharma, Inc.	18 Oct 2017
Large B-cell lymphoma	US	Kite Pharma, Inc.	18 Oct 2017

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Non-Hodgkin Lymphoma	Phase 2	US	Kite Pharma, Inc.	08 Nov 2023
Indolent Non-Hodgkin Lymphoma	Phase 2	CN	Fosun Kite Biotechnology Co., Ltd.Startup	10 May 2022
Marginal Zone B-Cell Lymphoma	Phase 2	US	Kite Pharma, Inc.	20 Jun 2017
Marginal Zone B-Cell Lymphoma	Phase 2	FR	Kite Pharma, Inc.	20 Jun 2017
Recurrent Indolent Non-Hodgkin Lymphoma	Phase 2	US	Kite Pharma, Inc.	20 Jun 2017
Recurrent Indolent Non-Hodgkin Lymphoma	Phase 2	FR	Kite Pharma, Inc.	20 Jun 2017
Refractory Indolent Non-Hodgkin Lymphoma	Phase 2	US	Kite Pharma, Inc.	20 Jun 2017
Refractory Indolent Non-Hodgkin Lymphoma	Phase 2	FR	Kite Pharma, Inc.	20 Jun 2017
Diffuse large B-cell lymphoma recurrent	Phase 2	US	Kite Pharma, Inc.	21 Apr 2015
Diffuse large B-cell lymphoma recurrent	Phase 2	CA	Kite Pharma, Inc.	21 Apr 2015

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Pubmed \| Blood Adv Manual	Not Applicable	Large B-cell lymphoma Third line	179	2L SOC; 3L cellular immunotherapy	fkdgyrwmck(sqdihabvgl) = gshxljelwb fztmsvikkw (iitgihemid ) View more	Positive	11 Jun 2024
				2L SOC; did not 3L therapy	fkdgyrwmck(sqdihabvgl) = jueegtgbkm fztmsvikkw (iitgihemid ) View more
NCT04608487 (ASCO2024) Manual	Phase 1	Secondary Central Nervous System Lymphoma CD19	18	Axicabtagene ciloleucel (axi-cel)	bciaeixlfg(qjwnddhvoz) = cjkiepolei ibvmajftwf (jkvqobyuws ) View more	Positive	24 May 2024
CART-SIE (EHA2024) Manual	Not Applicable	Diffuse Large B-Cell Lymphoma CD19	485	Axicabtagene Ciloleucel	bslnsqinkz(usuggrkhib) = nfejskftox ovbtqnpdrd (uyfdrahtdp ) View more	Positive	14 May 2024
				Tisagenlecleucel	bslnsqinkz(usuggrkhib) = lnsdhdvamx ovbtqnpdrd (uyfdrahtdp ) View more
NCT05459571 (ZUMA-24, EHA2024) Manual	Phase 2	Large B-cell lymphoma	23	Axicabtagene ciloleucel	xzhnkjbqze(premsktfuc) = nsafynvpkb kiwutspruy (aodmxqruhy ) View more	Positive	14 May 2024
EHA2024 Manual	Not Applicable	Non-Hodgkin's lymphoma refractory	155	axi-cel or brexu-cel (early mgmt period [EMP])	iqeodawfiz(goqzynhbwd) = hwhjrucatz iahlcxotsu (qektlzzsol ) View more	Positive	14 May 2024
				axi-cel or brexu-cel (late mgmt period [LMP])	iqeodawfiz(goqzynhbwd) = hhuzvapguy iahlcxotsu (qektlzzsol ) View more
NCT03391466 \| NCT02348216 (ZUMA-1, EHA2024) Manual	Phase 2/3	Large B-cell lymphoma Second line \| Third line CD19	4,087	Axicabtagene ciloleucel in 2L	xyrkewxsqx(xpgrxyjpsd) = cfvtzctrbc ravnnfoypr (kqmhcpqory ) View more	Positive	14 May 2024
				Axicabtagene ciloleucel in 3L+	xyrkewxsqx(xpgrxyjpsd) = xxuqsekoap ravnnfoypr (kqmhcpqory ) View more
EHA2024	Not Applicable	Large B-cell lymphoma CD19	95	Axicabtagene ciloleucel	tmqxsscilx(iuoregqqxe) = tyzojmiici jvczhldrwq (lskeooxccv, 42 - 63) View more	-	14 May 2024
ALYCANTE (EHA2024)	Phase 2	Large B-cell lymphoma [18F]FDG positron emission tomography (PET)	62	axicabtagene ciloleucel	rojvxdfvyf(brvhmewgia) = uqyptpizit oquorcvrfd (dhsfebndix ) View more	Positive	14 May 2024
				axicabtagene ciloleucel	rojvxdfvyf(brvhmewgia) = jswudqukyy oquorcvrfd (dhsfebndix ) View more
NCT03391466 (ZUMA-7, AACR2024) Manual	Phase 3	Large B-cell lymphoma Second line CD19 \| CD45RA \| CCL22 ... View more	134	axicabtagene ciloleucelgene ciloleucel	-	Positive	05 Apr 2024
				axicabtagene ciloleucely
NCT02348216 (ZUMA-1, AACR2024)	Phase 1/2	Large B-cell lymphoma CD19	-	Axicabtagene ciloleucel (axi-cel)	emyfgmafrp(zqehccotyd) = lxszqozpmh emnvroelem (zgktnafebe ) View more	-	05 Apr 2024

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis