Pilot Study of the Infusion of Differentiated Autologous T-cells From Peripheral Blood, Expanded and Transduced With a Lentivirus to Express a Chimeric Antigen Receptor With Anti-BCMA (TNFRSF17) Specificity Humanized Conjugated With the Co-stimulatory Region 4-1BB and Signal-transduction CD3z (ARI0002h) in Patients With Relapsed/Refractory Multiple Myeloma With Previous Treatment With Proteasome Inhibitor, Immunomodulatory Drug and Anti-CD38 Monoclonal Antibody

To assess the safety and efficacy of CARTBCMA ARI0002h in patients with relapsed/refractory multiple myeloma who have received treatment with proteasome inhibitor, immunomodulatory drug and anti-CD38 monoclonal antibody.

Start Date27 May 2020

Sponsor / Collaborator

Institut d'Investigacions Biomèdiques August Pi I Sunyer

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100 Clinical Results associated with Cesnicabtagene Autoleucel

100 Translational Medicine associated with Cesnicabtagene Autoleucel

100 Patents (Medical) associated with Cesnicabtagene Autoleucel

Literatures (Medical) associated with Cesnicabtagene Autoleucel

01 Aug 2023·The Lancet. Oncology

Fractionated initial infusion and booster dose of ARI0002h, a humanised, BCMA-directed CAR T-cell therapy, for patients with relapsed or refractory multiple myeloma (CARTBCMA-HCB-01): a single-arm, multicentre, academic pilot study.

Article

Author: Jiménez, Raquel ; Reguera, Juan Luis ; González-Calle, Verónica ; Oliver-Caldés, Aina ; Inogés, Susana ; López-Parra, Miriam ; Zabaleta, Aintzane ; Rodríguez-Otero, Paula ; Cabañas, Valentín ; Tovar, Natalia ; López-Corral, Lucía ; Varea, Sara ; Delgado, Julio ; López-Díaz de Cerio, Ascensión ; Olesti, Eulàlia ; Prósper, Felipe ; Pérez-Simón, José Antonio ; Martin-Antonio, Beatriz ; Fernández de Larrea, Carlos ; Urbano-Ispizua, Alvaro ; Calvo-Orteu, Maria ; Moraleda, José M ; Rodríguez-Lobato, Luis Gerardo ; Sáez-Peñataro, Joaquín ; Juan, Manel ; Paiva, Bruno ; Pascal, Mariona ; Español-Rego, Marta ; Sánchez-Salinas, Andrés ; Rosiñol, Laura ; Mateos, María-Victoria

BACKGROUNDChimeric antigen receptor (CAR) T-cell therapy is a promising option for patients with heavily treated multiple myeloma. Point-of-care manufacturing can increase the availability of these treatments worldwide. We aimed to assess the safety and activity of ARI0002h, a BCMA-targeted CAR T-cell therapy developed by academia, in patients with relapsed or refractory multiple myeloma.METHODSCARTBCMA-HCB-01 is a single-arm, multicentre study done in five academic centres in Spain. Eligible patients had relapsed or refractory multiple myeloma and were aged 18-75 years; with an Eastern Cooperative Oncology Group performance status of 0-2; two or more previous lines of therapy including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody; refractoriness to the last line of therapy; and measurable disease according to the International Myeloma Working Group criteria. Patients received an initial fractionated infusion of 3 × 10⁶ CAR T cells per kg bodyweight in three aliquots (0·3, 0·9, and 1·8 × 10⁶ CAR-positive cells per kg intravenously on days 0, 3, and 7) and a non-fractionated booster dose of up to 3 × 10⁶ CAR T cells per kg bodyweight, at least 100 days after the first infusion. The primary endpoints were overall response rate 100 days after first infusion and the proportion of patients developing cytokine-release syndrome or neurotoxic events in the first 30 days after receiving treatment. Here, we present an interim analysis of the ongoing trial; enrolment has ended. This study is registered with ClinicalTrials.gov, NCT04309981, and EudraCT, 2019-001472-11.FINDINGSBetween June 2, 2020, and Feb 24, 2021, 44 patients were assessed for eligibility, of whom 35 (80%) were enrolled. 30 (86%) of 35 patients received ARI0002h (median age 61 years [IQR 53-65], 12 [40%] were female, and 18 [60%] were male). At the planned interim analysis (cutoff date Oct 20, 2021), with a median follow-up of 12·1 months (IQR 9·1-13·5), overall response during the first 100 days from infusion was 100%, including 24 (80%) of 30 patients with a very good partial response or better (15 [50%] with complete response, nine [30%] with very good partial response, and six [20%] with partial response). Cytokine-release syndrome was observed in 24 (80%) of 30 patients (all grade 1-2). No cases of neurotoxic events were observed. Persistent grade 3-4 cytopenias were observed in 20 (67%) patients. Infections were reported in 20 (67%) patients. Three patients died: one because of progression, one because of a head injury, and one due to COVID-19.INTERPRETATIONARI0002h administered in a fractioned manner with a booster dose after 3 months can provide deep and sustained responses in patients with relapsed or refractory multiple myeloma, with a low toxicity, especially in terms of neurological events, and with the possibility of a point-of-care approach.FUNDINGInstituto de Salud Carlos III (co-funded by the EU), Fundación La Caixa, and Fundació Bosch i Aymerich.

British Journal of Haematology

Serum mass spectrometry for treatment monitoring in patients with multiple myeloma receiving ARI0002h CAR T‐cells

Article

Author: Español‐Rego, Marta ; Reguera, Juan Luis ; Uribe‐Herranz, Mireia ; Pascal, Mariona ; Benítez‐Ribas, Daniel ; Puig, Noemí ; Inogés, Susana ; Tréboles, Karen ; Fernández de Larrea, Carlos ; Urbano‐Ispizua, Álvaro ; López‐Diaz de Cerio, Ascensión ; Cabañas, Valentín ; Tovar, Natalia ; Aróstegui, Juan Ignacio ; Agulló, Cristina ; Moraleda, José María ; Zabaleta, Aintzane ; Zugasti, Inés ; Mora, Génesis ; Ortiz de Landazuri, Iñaki ; Paiva, Bruno ; Rodríguez Otero, Paula ; Contreras, María Teresa ; Rodríguez‐Lobato, Luis Gerardo ; González‐Calle, Verónica ; González, Europa Azucena ; Juan, Manel ; Navarro, Sergio ; Martin‐Antonio, Beatriz ; Mateos, María Victoria ; Oliver‐Caldés, Aina ; Rosiñol, Laura ; Yagüe, Jordi ; Charry, Paola

SummaryChimeric antigen receptor (CAR) T‐cell therapies have increased the patients with relapsed/refractory multiple myeloma (RRMM) in whom standard electrophoretic techniques fail to detect the M‐protein. Quantitative immunoprecipitation mass spectrometry (QIP‐MS) can accurately measure serum M‐protein with high sensitivity, and identify interferences caused by therapeutic monoclonal antibodies. Here, we investigate the outcome of QIP‐MS in 33 patients treated with the academic BCMA‐directed CAR T‐cell ARI0002h (Cesnicabtagene Autoleucel). QIP‐MS offered more detailed insights than serum immunofixation (sIFE), identifying glycosylated M‐proteins and minor additional peaks. Moreover, the potential interferences owing to daratumumab or tocilizumab treatments were successfully detected. When analysing different assay platforms during patient's monitoring after ARI0002h administration, we observed that QIP‐MS showed a high global concordance (78.8%) with sIFE, whereas it was only moderate (55.6%) with bone marrow (BM)‐based next‐generation flow cytometry (NGF). Furthermore, QIP‐MS consistently demonstrated the lowest negativity rate across the different timepoints (27.3% vs. 60.0% in months 1 and 12, respectively). Patients with QIP‐MS(+)/BM‐based NGF(−) showed a non‐significant shorter median progression free survival than those with QIP‐MS(−)/BM‐based NGF(−). In summary, we show the first experience to our knowledge demonstrating that QIP‐MS could be particularly useful as a non‐invasive technique when evaluating response after CAR T‐cell treatment in MM.

100 Deals associated with Cesnicabtagene Autoleucel

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Plasma cell myeloma refractory	Phase 2	ES	Instituto De SAlud Carlos Iii De Madrid	27 May 2020
Relapse multiple myeloma	Phase 2	ES	Instituto De SAlud Carlos Iii De Madrid	27 May 2020

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EHA2024	Not Applicable	Multiple Myeloma sBCMA \| circulating EVs	19	PHE885	rcvnzdmmth(kidmoxjbfz) = The presence of BCMA was observed and quantified in circulating EVs (these EVs were characterized by canonical EV-associated proteins). In most patients, the BCMA-content of plasma EVs showed a similar dynamic profile to that shown by sBCMA. qvegpxscfp (bcbfyfgtws ) View more	-	14 May 2024
EHA2024	Not Applicable	Multiple Myeloma sBCMA \| circulating EVs	19	ARI0002h		-	14 May 2024
NCT04309981 (CARTBCMA-HCB-01, ASH2023)	Phase 1/2	Relapse multiple myeloma	60	ARI0002h	(yjmdlbrhuw) = gtpckwgkfs ttwoapzxfk (gktzbpaaqd ) View more	-	11 Dec 2023
NCT04309981 (CARTBCMA-HCB-01, Pubmed)	Phase 1/2	Plasma cell myeloma refractory	35	ARI0002h	(umrribcwex) = kpvcblmals gpmoszaaqu (izxozwgpmq ) View more	-	03 Jul 2023
NCT04309981 (CARTBCMA-HCB-01, EHA2022) Manual	Phase 1/2	Relapse multiple myeloma	35	Cyclophosphamide+fludarabine+ARI0002H	(wvtehmspdp) = pehqpfrbln djdwwhqhfn (naroaolaak ) View more	Positive	12 May 2022
NCT04309981 (CARTBCMA-HCB-01, ASH2021) Manual	Phase 1/2	Relapse multiple myeloma	35	ARI0002h	(ppafrupceb) = ilbjpcutch xnjhzrgvhx (adcyimjevq ) View more	Positive	05 Nov 2021

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