A Phase I Study of B-Cell Maturation Antigen (BCMA)-Specific Chimeric Antigen Receptor T Cells in Combination With JSMD194, a Small Molecule Inhibitor of Gamma Secretase, in Patients With Relapsed or Persistent Multiple Myeloma

This phase I trial determines the side effects and best dose of B-cell maturation antigen (BCMA)-chimeric antigen receptor (CAR) T-cells when combined with gamma-secretase inhibitor LY3039478 (JSMD194), cyclophosphamide, and fludarabine in treating participants with multiple myeloma that that has come back or remains despite treatment. Placing genes added in the laboratory into immune T-cells may make the T-cells recognize BCMA, a protein on the surface of cancer cells. JSMD194 may enhance the killing of cancer cells by increasing the BCMA expression on multiple myeloma cells, making the targeted BCMA CAR-T treatment more effective. JSMD194 also decreases the amount of BCMA found in the circulation (called soluble BCMA) that is not bound to the myeloma cells. JSMD194 can therefore reduce the potential for soluble BCMA to act as a decoy. Drugs used in chemotherapy, such as cyclophosphamide and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving BCMA CAR T therapy with JSMD194, cyclophosphamide, and fludarabine may work better in treating participants with relapsed or persistent multiple myeloma.

Start Date23 May 2018

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

[+2]

NCT03436771

/ TerminatedNot Applicable

Long-Term Follow-up Protocol for Subjects Treated With a CAR T-Cell Product on a Juno-Sponsored Clinical Trial

This study will provide long-term follow-up for patients who have received treatment with a Juno CAR T-cell product in a Juno-sponsored clinical trial. In this study, patients will be followed for up to 15 years after their last dose of Juno CAR T cells for evaluation of delayed adverse events, presence of persisting CAR T-cell vector sequences, presence of replication-competent retrovirus (RCR) or lentivirus (RCL), and survival.

Start Date19 Feb 2018

Sponsor / Collaborator

Juno Therapeutics, Inc.

NCT03430011

/ CompletedPhase 1/2

Protocol H125001: An Open-Label Phase 1/2 Study of JCARH125, BCMA-targeted Chimeric Antigen Receptor (CAR) T Cells, in Subjects With Relapsed or Refractory Multiple Myeloma

This is an open-label, multicenter, Phase 1/2 study to determine the safety and efficacy of JCARH125, a CAR T-cell product that targets B-cell maturation antigen (BCMA), in adult subjects with relapsed and/or refractory multiple myeloma. The study will include a Phase 1 part to determine the recommended dose of JCARH125 in subjects with relapsed and/or refractory multiple myeloma, followed by a Phase 2 part to further evaluate the safety and efficacy of JCARH125 at the recommended dose. The safety and tolerability of JCARH125 in subjects who receive prophylactic treatment with anakinra will be evaluated in a separate Phase 1 cohort. The antitumor activity of JCARH125 in subjects who have been previously treated with BCMA-directed therapy will be evaluated in separate Phase 2a cohorts.

Start Date01 Feb 2018

Sponsor / Collaborator

Juno Therapeutics, Inc.

100 Clinical Results associated with Orvacabtagene Autoleucel

100 Translational Medicine associated with Orvacabtagene Autoleucel

100 Patents (Medical) associated with Orvacabtagene Autoleucel

Literatures (Medical) associated with Orvacabtagene Autoleucel

01 Sep 2026·Molecular Therapy Advances

Epitope mapping of humoral immunogenicity of orvacabtagene autoleucel shows an IgM response with minimal impact on CAR T cellular kinetics

Article

Author: Stoevesandt, Oda ; Stadler, Volker ; Pazos, Michael ; Jawa, Vibha ; Liu, Xianghong ; Mora, Johanna ; Hu, Hongxiang ; Ogasawara, Ken ; Dai, Yanshan ; Gokemeijer, Jochem

Orvacabtagene autoleucel (orva-cel) is a fully human B cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T cell therapy evaluated in a phase 1/2 study in patients with relapsed or refractory multiple myeloma (RRMM). To assess treatment-related immunogenicity, anti-CAR therapeutic domain-specific antibodies (ATAs) were monitored in 157 treated patients. The ATAs were detected in 44.6% of patients over the course of study, with titers and incidence increasing over time. The goal of this study was to further characterize the observed immune response. The ATA status did not affect CAR T cell expansion or patient survival outcomes, though reduced persistence was observed in ATA-positive patients. Comprehensive immune profiling-including isotype analysis and B cell epitope mapping-identified five immunodominant consensus peptide sequences within the CAR domain. These epitopes were targeted by both Immunoglobulin G (IgG) and Immunoglobulin M (IgM) isotypes, with a persistent IgM response detected in most ATA-positive individuals. Despite the presence of ATAs, no adverse impact on cellular expansion was observed, potentially due to lymphodepletion and baseline immune suppression characteristic of B cell malignancies. These data suggest that the limited functional T- and B-cell capacity in RRMM may attenuate the clinical consequences of ATA development. The in vitro immunogenicity risk assessment and epitope mapping identified immunogenic hotspots within the CAR structure, which could have led to the high incidence of immune response observed in the patients. However, the analysis from this study points to a weak clinically non-relevant nature of the response that could be attributed to the patient's immune status and diseased state.

01 Dec 2025·Transplantation and Cellular Therapy

Impact of Gamma-Secretase Inhibition on Outcomes Following BCMA CAR-T Therapy in Multiple Myeloma: A Comparison of Two Phase 1 Trials

Article

Author: Pont, Margot J ; Riddell, Stanley R ; Blake, Michelle ; Works, Melissa G ; Shadman, Mazyar ; Green, Damian J ; Wu, Vicky Q ; Wood, Brent L ; Sather, Blythe D ; Liang, Emily C ; Gooley, Ted A ; Cowan, Andrew J ; Till, Brian G ; Song, Xiaoling ; Turtle, Cameron J ; Libby, Edward N ; Portuguese, Andrew J ; Chapuis, Aude G ; Coffey, David G ; Voutsinas, Jenna M ; Cole, Gabriel O ; Maloney, David G ; Milano, Filippo ; Tuazon, Sherilyn A ; Thomas, Sushma

BACKGROUND:

BCMA-directed chimeric antigen receptor (CAR)-T cell therapy represents a major therapeutic breakthrough for relapsed/refractory multiple myeloma (RRMM), offering deep and durable responses in heavily pretreated patients. However, a subset of patients experiences early relapse or fail to respond, highlighting the need for strategies to enhance efficacy. Gamma-secretase inhibitors (GSIs) have been shown to increase surface BCMA expression on malignant plasma cells and may potentiate the activity of BCMA CAR-T cells, particularly in patients with low baseline BCMA antigen density. Two contemporaneous phase 1 trials (FH9952 and FH9762) evaluated the fully human BCMA-targeted CAR-T product FCARH143, with FH9952 incorporating GSI co-administration.

OBJECTIVE:

To determine whether GSI use improves clinical outcomes following FCARH143 CAR-T therapy in RRMM, with particular focus on overall survival (OS), progression-free survival (PFS), and subgroup differences based on prior BCMA-targeted therapy and baseline tumor BCMA levels.

STUDY DESIGN:

This retrospective analysis compared outcomes from two single-arm, single-center phase 1 trials of the fully human BCMA-targeted CAR-T cell product FCARH143 in RRMM: FH9952 (n = 18), which incorporated the GSI crenigacestat, and FH9762 (n = 25), which did not. Both studies had extended follow-up beyond previously published reports. Eligible patients had measurable RRMM with ≥10% bone marrow plasma cell involvement and confirmed BCMA expression. BCMA-naïve patients were defined as those without prior exposure to BCMA-targeted therapies (e.g., BCMA CAR-T, bispecific antibodies, or antibody-drug conjugates); BCMA-exposed patients had received at least one such therapy. All participants received lymphodepletion with fludarabine and cyclophosphamide followed by infusion of FCARH143. In FH9952, crenigacestat (25 mg orally, three times weekly) was administered from day 0 through day +18. Outcomes included response rates, adverse events, OS, and PFS. Cox proportional hazards models were used for survival analysis.

RESULTS:

With extended follow-up (median 5.8 years), the overall cohort (n = 43) had a median OS of 2.7 years (95% CI 1.9-4.8) and median PFS of 1.1 years (95% CI 0.72-2.2). Baseline characteristics were generally similar, though BCMA-exposed status was more frequent in FH9952 (39% versus 8%). Rates of cytokine release syndrome, immune effector cell-associated neurotoxicity, and early immune effector cell-associated hematotoxicity were comparable between trials. Among BCMA-naïve patients (n = 34), GSI use was associated with improved OS (not reached versus 2.3 years; adjusted HR 0.30, 95% CI 0.10-0.88, P = .028) and a trend toward improved PFS (2.6 versus 1.3 years; adjusted HR 0.47, 95% CI 0.22-1.04, P = .062). No benefit was observed among BCMA-exposed patients. Exploratory spline modeling suggested GSI mitigated the adverse impact of low tumor BCMA levels, with inferior outcomes at low BCMA expression observed only in the non-GSI cohort.

CONCLUSIONS:

Co-administration of a GSI with BCMA CAR-T therapy was associated with improved survival in BCMA-naïve RRMM patients, particularly those with low baseline tumor BCMA levels. These findings suggest GSI modulation of BCMA surface expression may enhance CAR-T efficacy in select patients and support further prospective investigation.

31 Jul 2025·BLOOD

A phase 1 trial of fully human BCMA CAR-T therapy for relapsed/refractory multiple myeloma with 5-year follow-up

Article

Author: Green, Damian J. ; Cole, Gabriel O. ; Maloney, David G. ; Wood, Brent L. ; Song, Xiaoling ; Tuazon, Sherilyn A. ; Voutsinas, Jenna M. ; Cowan, Andrew J. ; Gooley, Ted A. ; Coffey, David G. ; Sather, Blythe D. ; Portuguese, Andrew J. ; Wu, Qian V. ; Shadman, Mazyar ; Works, Melissa G. ; Till, Brian G. ; Blake, Michelle ; Pont, Margot J. ; Turtle, Cameron J. ; Riddell, Stanley R. ; Thomas, Sushma ; Liang, Emily C.

Abstract:

FCARH143, an autologous B-cell maturation antigen (BCMA)–targeted chimeric antigen receptor (CAR) T-cell (CAR-T) therapy, which incorporates a fully human BCMA-specific single chain variable fragment and 4-1BB costimulatory domain, was evaluated in a phase 1 trial for relapsed/refractory multiple myeloma (RRMM). Patients were stratified by bone marrow plasma cell involvement (10%-30% or >30%) and received lymphodepleting chemotherapy followed by escalating CAR-T doses (50 × 106 to 450 × 106). The primary end point was safety; secondary end points were overall response rate (ORR), duration of response, and progression-free survival (PFS). Among 28 enrolled patients, all underwent leukapheresis and successful CAR-T manufacturing, although 3 (11%) did not proceed to infusion. The 25 treated patients (median age, 64 years) had a median of 8 prior therapies, 80% were triple-class refractory, and 44% had extramedullary disease. Cytokine release syndrome occurred in 84% (8% grade 3-4 and no grade 5), and neurotoxicity in 24% (12% grade 3 and no grade 4-5). No treatment-related deaths occurred. At a median follow-up of 67.3 months, treated patients had an ORR of 100%, including a stringent complete response in 64%. Median PFS and overall survival (OS) were 15.5 and 32.1 months, respectively. In an intention-to-treat analysis (median follow-up, 69.6 months), the ORR was 89.3%, and OS was 30.2 months. FCARH143 demonstrated potent antimyeloma activity, with a 100% response rate and manageable toxicity, independent of disease burden or cytogenetic risk. Further evaluation in high-risk RRMM is warranted. This trial was registered at www.clinicaltrials.gov as #NCT03338972.

News (Medical) associated with Orvacabtagene Autoleucel

12 Sep 2023

·www.biospace.com

CARGO Therapeutics Announces Appointment of Michael Ports, PhD, as Chief Scientific Officer

– Scientific leader with multi-company cell therapy drug development experience spanning early discovery initiatives through late-stage development – SAN MATEO, Calif., Sept. 12, 2023 (GLOBE NEWSWIRE) -- CARGO Therapeutics, Inc. (CARGO), a clinical-stage biotechnology company uniquely positioned to advance next generation, potentially curative cell therapies for cancer patients, today announced the appointment of Michael Ports, PhD, as Chief Scientific Officer. Dr. Ports will be responsible for advancing CARGO's proprietary platform technologies and discovery-stage programs. “I am delighted to welcome Michael, an experienced leader in cell therapy, to CARGO as our Chief Scientific Officer. Michael brings strong scientific leadership and a deep understanding of cell therapy to this role and will be an inspiring thought partner to our research and development teams,” said Gina Chapman, CARGO's President and Chief Executive Officer. “We look forward to partnering with Michael to further progress our platform technologies and pipeline programs to the clinic as we strive to deliver more cures to patients.” “I am pleased to join the exceptional team at CARGO and am inspired by their dedication, scientific excellence and vision. Throughout my career, I have sought opportunities to develop differentiated therapeutics and treatment strategies to significantly improve outcomes for patients,” said Michael Ports, Chief Scientific Officer, CARGO Therapeutics. “I have a passion for bringing new cell therapy options to the clinic, and I am eager to contribute to CARGO’s important work to bring new CAR T-cell therapies to patients.” Prior to joining CARGO, Dr. Ports was the Vice President and Head of Cell Therapy Discovery at Janssen Pharmaceuticals, overseeing the strategy and management of teams to advance both autologous and allogeneic cell therapy development candidates. Before joining Janssen, Dr. Ports held roles of increasing responsibility at Juno Therapeutics (acquired by Celgene Corporation) and Celgene (acquired by Bristol-Myers Squibb) where he served as head of late-stage research. In this role, he led preclinical efforts that enabled two investigational new drug (IND) applications in multiple myeloma for the anti-BCMA CAR T, JCARH125, and five IND/IND amendments and documentation for a BLA filing in support of the anti-CD19 CAR T, Breyanzi®. Breyanzi® is a CAR T-cell therapy approved in the United States and the European Union to treat certain types of lymphoma including large B cell lymphoma (LBCL). Dr. Ports began his industry career at Gilead Sciences where he explored the use of Zydelig® and other small molecules as immuno-oncology agents for use in solid tumors. Dr. Ports was a Postdoctoral Fellow at the Netherlands Cancer Institute and Fred Hutchinson Cancer Research Center. He received his PhD in Cancer Biology from the University of Arizona and his BS in Molecular, Cellular and Developmental Biology from University of California, Santa Barbara. About CARGO Therapeutics CARGO Therapeutics is a clinical-stage biotechnology company uniquely positioned to advance next generation, potentially curative cell therapies for cancer patients. CARGO’s programs, platform technologies, and manufacturing strategy are designed to directly address the limitations of approved cell therapies, including limited durability of effect, safety concerns and unreliable supply. CARGO is currently evaluating its lead program, CRG-022, an autologous CD22 chimeric antigen receptor (CAR) T-cell therapy candidate, in a potentially pivotal Phase 2 clinical trial in patients with large B-cell lymphoma (LBCL) whose disease relapsed or was refractory (R/R) to CD19 CAR T-cell therapy. CARGO also plans to evaluate CRG-022 in patients at earlier stages of disease, including LBCL and other hematologic malignancies. Beyond its lead program, CARGO is leveraging its proprietary cell engineering platform technologies to develop a pipeline of programs that incorporate multiple transgene therapeutic “cargo” designed to enhance CAR T-cell persistence and trafficking to tumor lesions, as well as to help safeguard against tumor resistance and T-cell exhaustion. CARGO’s founders are pioneers and world-class experts in CAR T-cell therapy, and its team has significant experience and success developing, manufacturing, launching and commercializing oncology and cell therapy products. For more information, please visit the CARGO Therapeutics website at . Find more information at cargo-tx.com Follow us on LinkedIn: CARGO Therapeutics Follow us on Twitter: @CARGOTx Media Contact: Maura Gavaghan maura@redhousecomms.com Investor Contact: Laurence Watts laurence@gilmartinir.com

Cell TherapyExecutive ChangePhase 2Immunotherapy

19 May 2021

·www.globenewswire.com

Multiple Myeloma Pipeline Analysis Demonstrates Stunning Growth With A Gamut of Pharma Companies Involved Worldwide

Los Angeles, USA, May 18, 2021 (GLOBE NEWSWIRE) -- Multiple Myeloma Pipeline Analysis Demonstrates Stunning Growth with a Gamut of Pharma Companies Involved Worldwide The Multiple Myeloma pipeline is majorly thriving owing to the several different companies developing novel and sophisticated blockbuster drugs, increasing R&D, increasing global prevalence, better awareness and appropriate diagnosis in patients. DelveInsight’s ‘Multiple Myeloma Pipeline Insights’ report provides a holistic view of the pipeline therapies that are under development in preclinical as well as clinical stages of development, and growth prospects across the Multiple Myeloma domain. Some of the key takeaways from the Multiple Myeloma Pipeline report: Got queries? Want to know more? Request for Sample @ Multiple Myeloma Pipeline Insights Multiple Myeloma is a malignant disorder characterized by uncontrolled proliferation of clonal plasma cells causing a wide variety of complications leading to organ dysfunction and eventually death. It is the second most prevalent hematological malignancy worldwide, with a median onset of 60 years. This incurable malignancy develops from an accumulation of terminally differentiated monoclonal plasma cells (PC) in the bone marrow. Reach out @ Multiple Myeloma Pipeline: Novel therapies and emerging technologies The Multiple Myeloma pipeline report proffers a holistic view of the business opportunities, threats, prospective collaborations and agreements, strong competitors, growth strategies, failed as well as discontinued drugs. Multiple Myeloma Pipeline: Drug Portfolio The report lays down a complete coverage of the therapeutics by development stage, product type, route of administration, molecule type, and MOA type for Multiple Myeloma across the complete product development cycle, including all clinical and non-clinical stages. Multiple Myeloma Therapeutic Assessment By Product Type By Stage By Molecule Type By Route of Administration By Mechanism of Action By Targets By Stage and Route of AdministrationBy Stage and Product Type Scope of the Report Coverage: GlobalKey Players: Kiadis Pharma, CASI Pharmaceuticals, NOXXON Pharma, MorphoSys), Sana Biotechnology, Ayala Pharmaceuticals, Cellectar Biosciences, Bristol-Myers Squibb, Poseida Therapeutics, AbbVie/Genentech, Janssen Research and Development, Nanjing Legend Biotech, Genenta Science, Biotest AG, Teneobio, iCell Gene Therapeutics, Juno Therapeutics, Arch Oncology, Regeneron Pharmaceuticals, Incyte Corporation, NexImmune Inc., Chongqing Precision Biotech, Novartis, CASI Pharmaceuticals, Ionis Pharmaceuticals, AgenTus Therapeutics, Biohaven Pharmaceuticals, CRISPR Therapeutics, Pfizer, GlaxoSmithKline, Seagen Inc., Heidelberg Pharma, Bluebird Bio, C4 Therapeutics, Inc., Hoffmann-La Roche, Phosplatin Therapeutics, Arcellx, Inc., HitGen Inc., Boehringer Ingelheim, I-Mab Biopharma, Celyad Oncology, Amgen and othersKey Multiple Myeloma Pipeline Therapies: TNB-383B, ONC201, AO-176, JCARH125, REGN5458, INCB001158, TAK-573, NEXI-002 T Cells, PHE885, CID-103, ION251, EMB-06, agenT-797, CTX120, PF-06863135, SEA-BCMA, AEVI-007, HDP-101, bb21217, CFT7455, RO7425781, PT-112, HG146, CYAD-211, AMG 701, Nivolumab, ventoclax, RAPA-201 autologous T cells, K NK004, CID 103, NOX-A12, TJ202, APG-2575, AL 102, CLR 131 and others. Reach out @ Multiple Myeloma Pipeline: Novel therapies and emerging technologies Table of Contents Visit to know more of what’s covered @ Multiple Myeloma Emerging Therapies Related Reports Multiple Myeloma MarketDelveInsight’s ‘Multiple Myeloma (MM)-Market Insights, Epidemiology and Market Forecast—2030’ report. Multiple Myeloma Epidemiology ForecastDelveInsight’s ‘Multiple Myeloma (MM)- Epidemiology Forecast to 2030’ report. Car T Cell Therapy For Multiple Myeloma MarketDelveInsight’s ‘CAR T-Cell Therapy for Multiple Myeloma-Market Insights and Market Forecast-2030’ report. Refractory Multiple Myeloma MarketDelveInsight's "Refractory Multiple Myeloma - Market Insights, Epidemiology, and Market Forecast-2030" report. Relapsed Refractory Multiple Myeloma MarketDelveInsight's "Relapsed/Refractory Multiple Myeloma (RRMM) - Market Insights, Epidemiology, and Market Forecast-2030" report. Triple Refractory Multiple Myeloma MarketDelveInsight's "Triple-refractory Multiple Myeloma - Market Insights, Epidemiology, and Market Forecast-2030" report. Higher Risk Chronic Myelomonocytic Leukemia MarketDelveInsight's "Higher-Risk Chronic Myelomonocytic Leukemia Market Insights, Epidemiology, and Market Forecast-2030" report. Related Posts Multiple Myeloma Market Insights Multiple Myeloma Therapies MarketRare Cancer Market Outlook About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. For more insights, visit Pharma, Healthcare, and Biotech News

Small molecular drugCell TherapyCollaborate

04 Dec 2018

·www.biospace.com

ASH’s Hot Ticket: BCMA-Targeted CAR-T for B-cell Cancers

Janssen Pharmaceutical, a Johnson & Johnson company, along with Legend Biotech, reported results from their LEGEND-2 Phase I/II clinical trial of CAR-T therapy LCAR-B38M. The therapeutic was being investigated in patients with advanced relapsed or refractory (r/r) multiple myeloma. Janssen Pharmaceutical , a Johnson & Johnson company, along with Legend Biotech , reported results from their LEGEND-2 Phase I/II clinical trial of CAR-T therapy LCAR-B38M. The therapeutic was being investigated in patients with advanced relapsed or refractory (r/r) multiple myeloma. The findings were presented at the 2018 American Society of Hematology (ASH) Annual Meeting and Exposition. The findings in the oral presentation were built on data from one of four independent institutional studies, the Second Affiliated Hospital of Xi’an Jiaotong University . That data was originally presented at the 2017 American Society of Clinical Oncology (ASCO) Meeting and the 2017 European Hematology Association (EHA) Meeting. The CAR-T therapy, LCAR-B38M, was directed at the B-cell maturation antigen (BCMA). In the trial, the therapy showed deep and durable responses and a decent safety pro patients who had failed a median of three previous therapies. In the study, 57 patients received treatment with LCAR-B38M. The median age of patients was 54 years, and as noted above, the median number of previous therapies was three, ranging from one to nine. About 74 percent of patients had Stage III disease by Durie-Salmon staging. The companies reported an 88 percent overall response rate (ORR), and a complete response (CR) of 74 percent. Four percent of patients had a “very good partial response” and 11 percent had a partial response. Among the 42 patients with CR, 68 percent (39) were minimal residual disease (MRD) negative in the bone marrow. The median duration of response with a median follow-up of 12 months, was 16 months. Median progression-free survival (PFS) was 15 months for all patients. “CAR-T science has led to the approval of much-needed therapeutic interventions for certain blood cancers, and it is our hope that the results we are seeing in multiple myeloma will yield another much-needed option for patients,” stated Wan-Hong Zhao , associate director of Hematology at The Second Affiliated Hospital of Xi’an Jiaotong University in Xi’an, China, and lead study investigator. “We are excited about these data and the fact that they demonstrate notable responses in heavily pre-treated patients with multiple myeloma, a population that traditionally has been difficult to treat.” They were not the only companies to present data on BCMA-targeted CAR-T therapies. Celgene and bluebird bio presented initial data from their ongoing Phase I clinical trial of bb21217, a next-generation anti-B-cell maturation antigen (BCMA) CAR-T therapy for relapsed/refractory multiple myeloma. The patient population had received at least three prior treatments, including a proteasome inhibitor and immunomodulatory agent, or are double refractory. Twelve patients in the study received bb21217. Of those 12, 83 percent had an objective clinical response by International Myeloma Working Group (IMWG) criteria. At the time of the data analysis, there were ongoing responses in nine out of 10 patients, including three with a complete response (CR) or stringent complete response (sCR), and two reported a very good partial response (VGPR) and four had a partial response (PR). On its own, Celgene also announced safety data from its ongoing proof-of-concept trial of JCARH125 in patients with r/r multiple myeloma. JCARH125 is an investigational BCMA-targeting CAR-T therapy developed by Juno Therapeutics , a Celgene company. At the time of the data cut off, 44 patients had received JCARH125 in three dose escalation cohorts. They were heavily pretreated. Of those, 71 percent experienced grade 1 and 2 cytokine release syndrome (CRS) with 9 percent experience grade 3 or 4 CRS. Also, 18 percent experienced grade 1 and 2 neurological events with 7 percent having a grade 3 or 4 event. “We believe that cellular therapies targeting BCMA will play an important role in the future treatment of patients with multiple myeloma,” stated Mark Gilbert , chief medical officer of Juno. “These data from 44 patients in the EVOLVE trial further support our commitment to innovation in multiple myeloma clinical research.”

Clinical ResultCell TherapyPhase 1ImmunotherapyASH

100 Deals associated with Orvacabtagene Autoleucel

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Refractory Multiple Myeloma	Phase 2	United States	Juno Therapeutics, Inc.	01 Feb 2018
Relapse multiple myeloma	Phase 2	United States	Juno Therapeutics, Inc.	01 Feb 2018
Acute Lymphoblastic Leukemia	Phase 2	-	Juno Therapeutics, Inc.	-
BCMA Positive Multiple Myeloma	Phase 1	United States	Juno Therapeutics, Inc.	29 Nov 2017
Recurrent Multiple Myeloma	Phase 1	United States	Juno Therapeutics, Inc.	29 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03430011 (H125001 \| EVOLVE, CTgov)	Phase 1/2	Relapse multiple myeloma \| Refractory Multiple Myeloma	165	CD3+CAR+T cells+JCARH125 (Phase 1 50 x 10^6 Cells)	oysvdwqelf = vuatchzixl ylfvcjzfsd (aamrwqqriy, kuqngnyreu - eyekzebjgr) View more	-	24 May 2024
				CD3+CAR+T cells+JCARH125 (Phase 1 150 x 10^6 Cells)	oysvdwqelf = lmemjmzulh ylfvcjzfsd (aamrwqqriy, slpxztxzow - tyambxcfop) View more
NCT03338972 (CTgov)	Phase 1	Recurrent Multiple Myeloma \| BCMA Positive Multiple Myeloma \| Refractory Multiple Myeloma	28	Leukapheresis+Fludarabine+Cyclophosphamide+Autologous Anti-BCMA-CAR-expressing CD4+/CD8+ T-lymphocytes FCARH143 (Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 1)	dxcbpgzsrk = yiulmldyjm lvocavrvxi (tcibnwmzjm, lmscsuxtlc - gmucpzsuhm) View more	-	05 Aug 2022
				Leukapheresis+Fludarabine+Cyclophosphamide+Autologous Anti-BCMA-CAR-expressing CD4+/CD8+ T-lymphocytes FCARH143 (Treatment (Chemotherapy, BCMA CAR-T Cells) at Dose Level 2)	dxcbpgzsrk = xfpuqpxobc lvocavrvxi (tcibnwmzjm, yjoodqdbxw - zigxgaqusn) View more
NCT03430011 (EVOLVE, EHA2020)	Phase 1/2	Multiple Myeloma	51	Orva-cel 300 × 10^6 CAR+ T cells	uisytzwbco(nzhqgzgxsy) = rlqhaffjib ykhlqjujll (sjhjjpwhvy ) View more	Positive	14 May 2020
				Orva-cel 450 × 10^6 CAR+ T cells	csmfihnhqp(wccvlkwltd) = wwiqtxllwt mcegfahpmd (uuialhfknb )
NCT03430011 (H125001, ASH 12018 \| ASH 22018) Manual	Phase 1/2	Relapse multiple myeloma	19	JCARH125	ckcnssywbu(qyeybvnyqc) = 75% (Cytokine release syndrome) jdymmdvmfx (lbimqpguue ) View more	Positive	29 Nov 2018

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