Open-label Phase 1b Dose Escalation/Dose Expansion study exploring the safety and efficacy of NXC-201 in patients with relapsed or refractory light chain amyloidosis (AL).

开始日期2024-06-05

申办/合作机构

Nexcella, Inc.

[+1]

NCT04720313 / Recruiting临床1期IIT

A Phase 1 Dose Escalation and Safety Study of NXC-201 (Formerly HBI0101) CART in BCMA-Expressing Multiple Myeloma Patients

It is a phase one study with dose escalation and safety CART in BCMA- Expressing Multiple Myeloma and AL amyloidosis Patients

开始日期2021-01-01

申办/合作机构

Hadassah University Hospital

[+1]

100 项与 NXC-201 相关的临床结果

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100 项与 NXC-201 相关的转化医学

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100 项与 NXC-201 相关的专利（医药）

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项与 NXC-201 相关的文献（医药）

2024-08-13·Blood Advances

Clinical evaluation and determinants of response to HBI0101 (BCMA CART) therapy in relapsed/refractory multiple myeloma

Article

作者: Kenett, Ron S ; Shehadeh, Alaa ; Zelmanovich, Veronica ; Lebel, Eyal ; Kfir-Erenfeld, Shlomit ; Stepensky, Polina ; Grisariu, Sigal ; Elias, Shlomo ; Roziner, Ilan ; Gatt, Moshe E ; Ishtay, Aseel ; Pick, Marjorie ; Alexander-Shani, Rivka ; Asherie, Nathalie ; Assayag, Miri ; Zimran, Eran ; Avni, Batia ; Cohen, Yael ; Cohen, Cyrille J ; Bessig, Nomi ; Dubnikov Sharon, Tatyana ; Vainstein, Vladimir ; Avivi, Irit

AbstractHBI0101 is an academic chimeric antigen receptor T-cell (CART)–targeted to B-cell maturation antigen (BCMA) for the treatment of relapsed and refractory multiple myeloma (R/RMM) and light chain amyloidosis. Herein, we present the phase 1b/2 results of 50 heavily pretreated patients with R/RMM dosed with 800 × 106 CART cells. Inclusion criteria were relatively permissive (i.e., performance status and baseline organ function) and consequently, approximately half of the enrolled patients would have been ineligible for pivotal clinical trials. The median time elapsed from patient enrollment until CART delivery was 25 days (range, 14-65). HBI0101-related toxicities included grade 1 to 3 cytokine release syndrome, grade 3 to 4 hematologic toxicities, and grade 1 to 2 immune effector cell–associated neurotoxicity syndrome. Responses were achieved in 90% of the patients, 56% achieved stringent and complete response, and 70% reached a minimal residual disease negativity. Within a median follow-up of 12.3 months, the median progression-free survival (PFS) was 11.0 months (95% confidence interval [CI], 6.2-14.6), and the overall survival was not reached (95% CI, 13.3 to not reached). Multivariable analysis on patient/disease and CART-related characteristics revealed that high-risk cytogenetic, extramedullary disease, and increased number of effector-memory T cells in CART products were independently associated with inferior PFS. In conclusion, comprehensive analyses of the parameters affecting the response to CART therapy are essential for improving patients’ outcome. This trial was registered at www.ClinicalTrials.gov as #NCT04720313.

Haematologica

Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial

Article

作者: Lebel, Eyal ; Cohen, Yael ; Kfir-Erenfeld, Shlomit ; Dubnikov, Tatyana ; Pick, Marjorie ; Assayag, Miri ; Cohen, Cyrille ; Grisariu, Sigal ; Stepensky, Polina ; Avivi, Irit ; Asherie, Nathalie ; Zalcman, Nomi ; Shaulov, Adir ; Gatt, Moshe E ; Avni, Batia ; Zimran, Eran

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor T-cell (CAR T) therapy shows remarkable efficacy in patients with relapsed and/or refractory (R/R) multiple myeloma (MM). HBI0101, a novel second generation optimized anti- BCMA CAR T-cell therapy, was developed in an academic setting. We conducted a phase I dose-escalation study of HBI0101 (cohort 1: 150x106 CAR T cells, n=6; cohort 2: 450x106 CAR T cells, n=7; cohort 3: 800x106 CAR T cells, n=7) in 20 heavily pre-treated R/R MM patients. Grade 1-2 cytokine release syndrome (CRS) was reported in 18 patients (90%). Neither grade 3-4 CRS nor neurotoxicity of any grade were observed. No dose-limiting toxicities were observed in any cohort. The overall response rate (ORR), (stringent) complete response (CR/sCR), and very good partial response rates were 75%, 50%, and 25%, respectively. Response rates were dose-dependent with 85% ORR, 71% CR, and 57% minimal residual disease negativity in the high-dose cohort 3. Across all cohorts, the median overall survival (OS) was 308 days (range 25-466+), with an estimated OS of 55% as of June 27th (data cut-off). The median progression-free survival was 160 days, with 6 subjects remaining progression free at the time of data cut-off. Our findings demonstrate the manageable safety profile and efficacy of HBI0101. These encouraging data support the decentralization of CAR T production in an academic setting, ensuring sufficient CAR T supply to satisfy the increasing local demand. Clinicaltrials.gov NCT04720313.

129

项与 NXC-201 相关的新闻（医药）

2024-11-13

·PRNewswire

75+ Key Companies Charting New Frontiers in Multiple Sclerosis Therapeutic Space | DelveInsight

Multiple sclerosis (MS) is a chronic neurological disorder affecting the central nervous system (CNS), particularly the brain, spinal cord, and optic nerves. Continuous research has led to new drug developments, particularly disease-modifying therapies (DMTs) that slow MS progression. Monoclonal antibodies, oral therapies, and infusion treatments are among the most promising options and drives the multiple sclerosis market. LAS VEGAS, Nov. 13, 2024 /PRNewswire/ -- DelveInsight's ' Multiple Sclerosis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline multiple sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the multiple sclerosis pipeline domain. Key Takeaways from the Multiple Sclerosis Pipeline Report DelveInsight's multiple sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for multiple sclerosis treatment. Key multiple sclerosis companies such as Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Promising multiple sclerosis pipeline therapies such as GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. In October 2024, Immunic, Inc. reported favorable results from the non-binding interim futility analysis of its Phase 3 ENSURE trial, which is evaluating its lead candidate, vidofludimus calcium (IMU-838), a nuclear receptor-related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS). In October 2024, Immunic, Inc. announced a positive outcome of the non-binding, interim futility analysis of its Phase III ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned. In September 2024, Roche, secured FDA approval for an injectable form of its blockbuster multiple sclerosis treatment, Ocrevus, reducing patient treatment time and potentially helping the company stay ahead of increasing competition. In September 2024, Sanofi announced the outcomes of Phase III clinical trials of its investigational oral brain-penetrant BTK inhibitor, tolebrutinib, aimed at treating multiple sclerosis (MS). These studies, namely HERCULES, GEMINI 1, and GEMINI 2, have provided mixed results, with the HERCULES trial meeting its primary endpoint while the GEMINI trials did not. The HERCULES study focused on the efficacy and safety of tolebrutinib in individuals with non-relapsing secondary progressive MS (nrSPMS) compared to a placebo. In August 2024, Abata Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The FDA recently cleared ABA-101's Investigational New Drug (IND) application, and initiation of a first-in-human (FIH) Phase I study is imminent. In August 2024, TG Therapeutics announced that the FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). In January 2024, Kyverna Therapeutics, Inc. announced it received fast-track designation by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of multiple sclerosis (MS). Request a sample and discover the recent advances in multiple sclerosis treatment drugs @ Multiple Sclerosis Pipeline Report The multiple sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage multiple sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the multiple sclerosis clinical trial landscape. Multiple Sclerosis Overview Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, leading to the deterioration or permanent damage of myelin, the protective sheath surrounding nerve fibers. While the exact cause of multiple sclerosis remains unclear, it is believed to involve a combination of genetic predisposition, environmental factors, and possibly viral infections. Common symptoms of multiple sclerosis include fatigue, difficulty walking, numbness or tingling in the limbs, muscle weakness, and vision problems, which can vary significantly among individuals and may evolve over time. Diagnosis typically involves a combination of neurological examinations, magnetic resonance imaging (MRI) to detect lesions in the brain and spinal cord, and sometimes lumbar puncture to analyze cerebrospinal fluid for inflammatory markers. While there is no cure for multiple sclerosis, treatment options aim to manage symptoms, reduce the frequency and severity of relapses, and slow disease progression. This may include disease-modifying therapies (DMTs), corticosteroids for acute exacerbations, and symptomatic treatments such as physical therapy, pain management, and lifestyle modifications. Early diagnosis and a tailored treatment plan are crucial for improving the quality of life for those living with multiple sclerosis. Find out more about multiple sclerosis treatment drugs @ Drugs for Multiple Sclerosis Treatment A snapshot of the Multiple Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging multiple sclerosis pipeline therapies @ Multiple Sclerosis Clinical Trials Multiple Sclerosis Therapeutics Assessment The multiple sclerosis pipeline report proffers an integral view of the multiple sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Multiple Sclerosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Immunomodulators, MHC class II gene modulators, Agammaglobulinaemia tyrosine kinase inhibitors, Dihydroorotate dehydrogenase inhibitors, Nuclear receptor subfamily 4 group A member 2 agonists, CD20 antigen inhibitors, Energy metabolism stimulants; Immunomodulators, Lysine-specific demethylase 1 inhibitors, Monoamine oxidase B inhibitors, Protein-arginine deiminase inhibitors, Immunostimulants Key Multiple Sclerosis Companies: Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Key Multiple Sclerosis Pipeline Therapies:GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. Dive deep into rich insights for new drugs for multiple sclerosis treatment, visit @ Multiple Sclerosis Drugs Table of Contents For further information on the multiple sclerosis pipeline therapeutics, reach out @ Multiple Sclerosis Treatment Drugs Related Reports Multiple Sclerosis Market Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key multiple sclerosis companies including Immune Response BioPharma, Inc., Clene Nanomedicine, Genzyme, ImStem Biotechnology, Rho, Inc., Bristol-Myers Squibb, TG Therapeutics, Inc., Hoffmann-La Roche, Atara Biotherapeutics, Immunic AG, Celgene, Anokion SA, Sanofi, Actelion, Biogen, Supernus Pharmaceuticals, Inc., GeNeuro SA, HuniLife Biotechnology, Inc., Emerald Health Pharmaceuticals, RemeGen Co., Ltd., Antisense Therapeutics, Biocad, AB Science, Genentech, Novartis, Pipeline Therapeutics, ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, Mapi Pharma, f5 Therapeutics, Autobahn Therapeutics, AstraZeneca, ZyVersa Therapeutics, RedHill Biopharma, Gossamer Bio, Sarepta Therapeutics, BrainStorm Cell Limited, CytoDyn, Pear Therapeutics, Solstice Neurosciences, ProJenX, among others. Multiple Sclerosis Epidemiology Forecast Multiple Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Relapsing Multiple Sclerosis Pipeline Relapsing Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key relapsing multiple sclerosis companies, including ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, among others. Relapsing-Remitting Multiple Sclerosis Pipeline Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, Cinnagen, Polpharma Biologics, Immunic, Mapi Pharma, Biocad, Apimeds, Genentech (Roche), Merck, Immune Response BioPharma, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, Emerald Health Pharmaceuticals, GlaxoSmithKline, RedHill Biopharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期临床结果快速通道细胞疗法免疫疗法

2024-10-02

·GlobeNewswire-Health Care

Immix Biopharma Advances CAR-T NXC-201 to Expansion Cohort Dose Level in U.S. AL Amyloidosis Trial NEXICART-2

Now dosing at dose expansion level of 450 million NXC-201 CAR+T cells. First cohort at 150 million CAR+T cells already successfully completed Each of these two doses have produced complete responses in prior clinical studiesLead site Memorial Sloan Kettering Cancer Center (MSKCC); 4 disclosed clinical sites so farNXC-201 is the only one-time CAR-T treatment option being studied for relapsed/refractory AL Amyloidosis in U.S. clinical trials AL Amyloidosis, a disorder of plasma cells that leads to progressive damage to the heart, kidney and liver, affects approximately 33,000 patients in the U.S. LOS ANGELES, CA, Oct. 02, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us”, “IMMX”) (Nasdaq: IMMX), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and select immune-mediated diseases, today announced advances in the NEXICART-2 clinical study of its CAR-T NXC-201 for relapsed/refractory AL Amyloidosis, a plasma cell disorder that leads to progressive damage to the heart, kidney and liver, which currently affects approximately 33,000 patients in the U.S. Immix Biopharma’s sterically-optimized CAR-T NXC-201 is the only one-time CAR-T treatment option being studied for relapsed/refractory AL Amyloidosis in U.S. clinical trials. “We are pleased to report that the NEXICART-2 study is making excellent progress. We are now one step closer to providing a new treatment option for patients with relapsed/refractory AL Amyloidosis, where no FDA drugs are approved today,” said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. “Robust enrollment reflects the enthusiasm of clinical investigators for CAR-T NXC-201.” “Positive data from our ex-US study of NXC-201, the largest CAR-T clinical study in relapsed/refractory AL Amyloidosis to-date, showed a 92% overall response rate (12/13) and a 28.0 month duration of response (best responder), presented at ASGCT 2024,” said Gabriel Morris, Chief Financial Officer of Immix Biopharma. “We credit the resolute efforts of our investigators, sites, and team as we continue on track for interim and final read-outs.” The NEXICART-2 study is intended to evaluate the safety and efficacy of NXC-201 in relapsed/refractory AL Amyloidosis patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. Advancement to the second dosing cohort of three patients at 450 million CAR-T cells will dose one patient with NXC-201 every 28-days, subsequently enabling multiple patients to be dosed per month in NEXICART-2. Each of the NEXICART-2 dose levels have produced complete responses in relapsed/refractory AL Amyloidosis patients in the ex-U.S. NEXICART-1 study presented at ASGCT 2024. NXC-201 is the only CAR-T therapy currently in development in AL Amyloidosis, mentioned in a review article entitled “Systemic Light Chain Amyloidosis” published in June, 2024 New England Journal of Medicine. About NEXICART-2 NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site U.S. Phase 1b/2 dose expansion clinical trial of CAR-T NXC-201 in relapsed/refractory AL Amyloidosis. NEXICART-2 is expected to enroll 40 patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The study is designed with a standard 6 patient safety run-in to evaluate two doses (three patients each at 150 million CAR+T cells and 450 million CAR+T cells), with dose expansion at 450 million CAR+T cells following. During dose expansion, multiple patients are allowed to be dosed per month. The NXC-201 dosing levels being evaluated in NEXICART-2 study have been evaluated in the NEXICART-1 study and have produced complete responses in relapsed/refractory AL Amyloidosis patients. The study aims to evaluate the safety and efficacy of NXC-201 in this patient population. Primary endpoints are complete response rate and overall response rate, according to consensus recommendations (Palladini et al. 2012). About NXC-201 NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. Initial NXC-201 clinical data in relapsed/refractory AL Amyloidosis from Phase 1b/2 ex-U.S. study NEXICART-1 has demonstrated a high overall and complete response rate, as well as no neurotoxicity of any kind and short duration of cytokine release syndrome, presented at the American Society of Hematology (ASH) and the American Society of Gene and Cell Therapy (ASGCT) annual meetings. NXC-201 is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, with the potential to expand into select immune mediated diseases. The NXC-201 NEXICART-2 U.S. clinical trial builds on an existing robust clinical dataset. NXC-201 has been awarded Orphan Drug Designation (ODD) in the US by the FDA and in the EU by the EMA in AL Amyloidosis. About AL Amyloidosis AL amyloidosis is caused by abnormal plasma cells in the bone marrow, which produce misfolded amyloid proteins that build-up in the heart, kidney, liver, and other organs. This build-up causes progressive and widespread damage to multiple organs, including heart failure, and leads to high mortality rates. The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at 12% per year according to Staron, et al Blood Cancer Journal, to approximately 33,277 patients in 2024. The Amyloidosis market was $3.6 billion in 2017, and is expected to reach $6 billion in 2025, according to Grand View Research. About Immix Biopharma, Inc. Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX) is a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and select immune-mediated diseases. Our lead candidate is sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201. NXC-201 is being evaluated in the U.S. Phase 1b/2 trial NEXICART-2 (NCT06097832) as well as the ex-U.S. study NEXICART-1 (NCT04720313). NXC-201 has demonstrated a high overall and complete response rate, as well as no neurotoxicity of any kind and short duration of cytokine release syndrome, presented at the American Society of Hematology (ASH) and the American Society of Gene and Cell Therapy (ASGCT) annual meetings. NXC-201 has been awarded Orphan Drug Designation (ODD) in the U.S. by the FDA and in the EU by the EMA in AL Amyloidosis. Learn more at www.immixbio.com and www.BeProactiveInAL.com. Forward Looking StatementsThis press release contains forward-looking statements regarding Immix Biopharma, Inc., including the potential benefits of our product candidate CAR-T NXC-201. Forward-looking statements include, but are not limited to, our plans, objectives, expectations and intentions and other statements that contain words such as “expects”, “contemplates”, “anticipates”, “plans”, “intends”, “believes”, “estimates”, “potential”, and variations of such words or similar expressions that convey the uncertainty of future events or outcomes, or that do not relate to historical matters. Those forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause actual results to differ materially. Among those factors are: (i) the risk that the further data from the ongoing Phase 1b/2a clinical trials for CAR-T NXC-201 will not be favorably consistent with the initial data initial data readouts, (ii) the risk that the Company may not be able to advance to registration-enabling studies for CAR-T NXC-201 or other product candidates, (iii) that success in early phases of pre-clinical and clinicals trials do not ensure later clinical trials will be successful; (iv) that no drug product developed by the Company has received FDA pre-market approval or otherwise been incorporated into a commercial drug product, (v) the risk that the Company may not be able to obtain additional working capital with which to continue the clinical trials for CAR-T NXC-201, or advance to the initiation of registration-enabling studies, for such product candidates as and when needed and (vii) those other risks disclosed in the section “Risk Factors” included in the Company’s Annual Report on Form 10-K filed with the SEC on March 29, 2024 and other periodic reports subsequently filed with the Securities and Exchange Commission. Immix Biopharma cautions readers not to place undue reliance on any forward-looking statements. Immix Biopharma does not undertake, and specifically disclaims, any obligation to update or revise such statements to reflect new circumstances or unanticipated events as they occur, except as required by law. Contacts Mike Moyer LifeSci Advisors mmoyer@lifesciadvisors.com Company Contact irteam@immixbio.com

细胞疗法临床结果ASH会议临床研究免疫疗法

2024-09-19

·GlobeNewswire-Health Care

Immix Biopharma Announces Dr. Raymond Comenzo, Internationally Recognized AL Amyloidosis Expert, Director of the Myeloma and Amyloid Program at Tufts Medical Center, Joins Scientific Advisory Board

Dr. Comenzo was the senior author of the landmark Andromeda trial results published in the New England Journal of Medicine in 2021, resulting in the first FDA-approved therapy for AL amyloidosis Dr. Comenzo was the lead author on the landmark 2012 publication of consensus guidelines, including response criteria, for conduct and reporting of clinical trials in AL Amyloidosis LOS ANGELES, CA, Sept. 19, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“Immix Biopharma”, “Company”, “We” or “Us”, Nasdaq: IMMX), announced that effective today, Dr. Raymond Comenzo, internationally recognized AL Amyloidosis expert and Director of the Myeloma and Amyloid Program at Tufts Medical Center, joined ImmixBio subsidiary Nexcella Scientific Advisory Board. Dr. Comenzo is internationally recognized for his pioneering work in developing consensus guidelines for clinical trials and response criteria in AL Amyloidosis, and was the senior author of the landmark Andromeda trial results published in the New England Journal of Medicine in 2021, resulting in the first FDA-approved therapy for AL amyloidosis. “CAR-T represents a novel approach to treat AL Amyloidosis. I am thrilled to see focus on developing treatment options for relapsed/refractory AL Amyloidosis, where no drugs are approved today. I am excited to join the Nexcella team’s efforts to advance CAR-T NXC-201," commented Dr. Comenzo. "We are thrilled that Dr. Comenzo has joined our scientific advisory board. We believe we will benefit greatly from Dr. Comenzo’s counsel," stated Ilya Rachman, MD, PhD, Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, “Dr. Comenzo’s foundational contributions to the AL Amyloidosis field are internationally recognized. We are excited to be working with Dr. Comenzo.” Dr. Comenzo is Director of the Myeloma and Amyloid Program; Director, the Cell Therapy Laboratory at Tufts Medical Center; and Professor of Medicine at Tufts University School of Medicine. He is principally responsible for the clinical care of all plasma cell disease patients and for clinical research for these diseases at Tufts Medical Center. Dr. Comenzo completed his residency at Boston City Hospital and his fellowship in hematology/oncology and transfusion medicine at Tufts Medical Center. Prior to coming to Tufts MC, Dr. Comenzo was medical director of the cell therapy laboratory, and an attending member at Memorial Sloan Kettering Cancer Center; a professor of medicine at Weil-Cornell School of Medicine; and an attending physician in hematology, the founder of the Stem Cell Transplant Program, and the director of the Blood Bank at Boston Medical Center. Dr. Comenzo is board certified in Internal Medicine and Hematology. About Immix Biopharma, Inc.Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX) is a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and immune-mediated diseases. Our lead candidate is sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201. NXC-201 is being evaluated in the U.S. Phase 1b/2 trial NEXICART-2 (NCT06097832) as well as the ex-U.S. study NEXICART-1 (NCT04720313). NXC-201 has demonstrated no neurotoxicity of any kind in AL Amyloidosis and short duration of cytokine release syndrome (CRS), supporting expansion into immune-mediated diseases. NXC-201 has been awarded Orphan Drug Designation (ODD) in AL Amyloidosis by the US FDA and in the EU by the EMA. Learn more at www.immixbio.com and www.BeProactiveInAL.com. About Nexcella, Inc.Nexcella, Inc. is a wholly-owned subsidiary of, and the cell therapy division of, Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX). Forward Looking StatementsThis press release contains forward-looking statements regarding Immix Biopharma, Inc., its results of operations, prospects, future business plans and operations and the matters discussed above, including, but not limited to, potential benefits of our product candidate CAR-T NXC-201. These statements involve risks and uncertainties, and actual results may differ materially from any future results expressed or implied by the forward-looking statements. Forward-looking statements also include, but are not limited to, our plans, objectives, expectations and intentions and other statements that contain words such as “expects”, “contemplates”, “anticipates”, “plans”, “intends”, “believes”, “estimates”, “potential”, and variations of such words or similar expressions that convey the uncertainty of future events or outcomes, or that do not relate to historical matters. Those forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause actual results to differ materially. Among those factors are: (i) the risk that the further data from the ongoing Phase 1b/2 clinical trials for CAR-T NXC-201 will not be favorably consistent with the initial data initial data readouts, (ii) the risk that the Company may not be able to advance to registration-enabling studies for CAR-T NXC-201 or other product candidates, (iii) that success in early phases of pre-clinical and clinicals trials do not ensure later clinical trials will be successful; (iv) that no drug product developed by the Company has received FDA pre-market approval or otherwise been incorporated into a commercial drug product, (v) the risk that the Company may not be able to obtain additional working capital with which to continue the clinical trials for CAR-T NXC-201, or advance to the initiation of registration-enabling studies, for such product candidates as and when needed and (vii) those other risks disclosed in the section “Risk Factors” included in the Company’s Annual Report on Form 10-K filed with the SEC on March 29, 2024 and other periodic reports subsequently filed with the Securities and Exchange Commission. These reports are available at www.sec.gov. Immix Biopharma cautions that the foregoing list of important factors is not complete. Immix Biopharma cautions readers not to place undue reliance on any forward-looking statements. Immix Biopharma does not undertake, and specifically disclaims, any obligation to update or revise such statements to reflect new circumstances or unanticipated events as they occur, except as required by law. If we update one or more forward-looking statements, no inference should be drawn that we will make additional updates with respect to those or other forward-looking statements. Contacts Mike Moyer LifeSci Advisors mmoyer@lifesciadvisors.com Company Contactirteam@immixbio.com

细胞疗法孤儿药高管变更上市批准

100 项与 NXC-201 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
免疫球蛋白轻链淀粉样变性	临床2期	-	Immix Biopharma, Inc.	-
多发性骨髓瘤	临床2期	美国	Immix Biopharma, Inc.	-
自身免疫性疾病	临床前	以色列	Nexcella, Inc.	2024-03-12

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04720313 (NEXICART-1, Biospace) 人工标引	临床1期	免疫球蛋白轻链淀粉样变性	13	NXC-201	(齋網簾艱簾廠淵遞憲積) = 壓築遞艱範鑰獵繭鹽淵顧窪夢鑰鏇壓夢鏇繭鬱 (壓壓鏇糧構壓鬱齋鏇鬱 ) 更多	积极	2024-05-10
NCT04720313 (NEXICART-1, Biospace) 人工标引	临床1期	免疫球蛋白轻链淀粉样变性	13	NXC-201 (pts not exposed to prior BCMA-targeted bispecific)	(齋網簾艱簾廠淵遞憲積) = 鹽獵築鹽鑰鹽淵築醖範顧窪夢鑰鏇壓夢鏇繭鬱 (壓壓鏇糧構壓鬱齋鏇鬱 ) 更多	积极	2024-05-10
ASH2023	临床1/2期	复发性多发性骨髓瘤	51	HBI0101	鹽願觸構糧襯製壓壓願(憲醖蓋膚鏇窪製遞壓蓋) = 繭願齋膚遞衊觸襯鹹淵淵範遞鹽壓觸繭餘積範 (廠網鑰醖鏇觸網遞糧醖 ) 更多	-	2023-12-11
NCT04720313 (ASH2023)	临床1/2期	免疫球蛋白轻链淀粉样变性	9	HBI0101 therapy	(鹹衊膚窪襯衊鑰遞製簾) = 選鑰鑰艱繭鑰網襯鹹壓選顧艱艱簾顧鏇醖憲鬱 (廠齋願顧遞築餘選餘艱 ) 更多	积极	2023-12-10
NCT04720313 (NEXICART-1, Corporate Publications) 人工标引	临床1/2期	免疫球蛋白轻链淀粉样变性	9	NXC-201 (heavily pre-treated patients with daratumumab relapsed/refractory AL Amyloidosis)	(範餘襯鹽餘繭構繭鹽窪) = 窪願選鹹獵憲襯鹽蓋憲網襯願膚醖積餘簾簾餘 (壓壓獵簾壓憲衊構艱遞 ) 更多	积极	2023-10-03
NCT04720313 (NEXICART-1, Corporate Publications) 人工标引	临床1/2期	免疫球蛋白轻链淀粉样变性	9	NXC-201 (t(11;14) relapsed/refractory AL Amyloidosis)	(範餘襯鹽餘繭構繭鹽窪) = 鏇鹽壓襯糧廠鹹憲壓選網襯願膚醖積餘簾簾餘 (壓壓獵簾壓憲衊構艱遞 ) 更多	积极	2023-10-03
NCT04720313 (IMS2023)	临床1期	复发性多发性骨髓瘤	50	HBI0101	構觸憲窪鹽鹽齋糧憲簾(鹹觸憲醖繭齋鏇淵窪艱) = 獵憲廠壓範醖鹹膚築壓製蓋築艱齋範艱構襯壓 (繭簾衊膚鏇艱築鏇夢膚 ) 更多	-	2023-09-26
NCT04720313 (GlobeNewswire) 人工标引	临床1期	多发性骨髓瘤	42	NXC-201	(襯觸製憲範廠構窪膚鑰) = 艱積築願膚艱遞顧壓選襯築餘壓糧選廠獵獵鑰 (鏇鏇窪淵繭淵鹹範壓遞 ) 更多	积极	2023-02-09
NCT04720313 (GlobeNewswire) 人工标引	临床1期	多发性骨髓瘤	42	NXC-201 (800 million CAR+T cells)	(襯觸製憲範廠構窪膚鑰) = 鑰繭窪醖鏇蓋窪鑰蓋蓋襯築餘壓糧選廠獵獵鑰 (鏇鏇窪淵繭淵鹹範壓遞 ) 更多	积极	2023-02-09
NCT04720313 (Pubmed) 人工标引	临床1期	多发性骨髓瘤	20	HBI0101	製憲範鑰窪鹽襯餘糧膚(衊膚齋觸願衊鏇憲艱憲) = 鬱廠廠憲壓鑰願醖淵廠齋艱襯壓製獵遞窪壓蓋 (蓋壓襯夢鏇襯鹹觸獵醖 ) 更多	积极	2022-10-06
NCT04720313 (Pubmed)	临床1期	免疫球蛋白轻链淀粉样变性	4	HBI0101	(鏇鹽顧壓蓋鹽壓範構蓋) = 顧鏇遞觸艱糧鑰蓋積鏇壓鬱網廠構構餘觸獵鹹 (鹽夢夢網簾網鬱淵鹹餘 )	积极	2022-09-15
NCT04720313 (EHA2022) 人工标引	临床1期	浆细胞骨髓瘤难治	20	HBI0101 (cohort 1: 150 x 10^6 cells)	(餘膚膚獵糧鹽觸選憲餘) = 壓範選窪餘鹹繭衊積網繭簾遞襯築鹹餘構餘網 (窪顧衊夢壓繭範範鏇夢 ) 更多	积极	2022-05-12
NCT04720313 (EHA2022) 人工标引	临床1期	浆细胞骨髓瘤难治	20	HBI0101 (cohort 2: 450 x 10^6 cells)	(餘膚膚獵糧鹽觸選憲餘) = 構蓋襯憲構願簾願廠願繭簾遞襯築鹹餘構餘網 (窪顧衊夢壓繭範範鏇夢 ) 更多	积极	2022-05-12